StemCell Therapy

A researcher at the University of San Diego, California (USCD) made an accidental discovery several years back after he silenced a gene called PTB in mouse fibroblasts, cells in connective tissue. Within weeks, almost all of the fibroblasts were gone, and the rest had transformed into neurons.

Now, researchers in the same lab are applying that discovery to Parkinsons disease in the hopes of creating a one-time gene therapy to replace the dopamine-producing neurons that are lost to the disease.

The UCSD team developed a gene therapy technique that cripplesthe ability of the PTB gene to produce a functioning protein. In mouse models of Parkinsons, the gene therapy turned supportive cells called astrocytes into dopamine-producing neurons, erasing symptoms of the disease, the researchers reported in the journal Nature.

White Paper: Keep Your GI Trials Moving During COVID-19

Clinical Inks intimate knowledge of and experience with GI trials enables a better deployment experience and improved trial conduct. Learn how our GI-specific data capture solutions can support virtual and hybrid trials during COVID-19.

The gene therapy created by the UCSD team consists of a viral vector that carries a piece of DNA called an antisense oligonucleotide into cells. The DNA binds to the RNA that codes for PTB, shutting off the production of the PTB protein and allowing neurons to form.

"Researchers around the world have tried many ways to generate neurons in the lab, using stem cells and other means, said Xiang-Dong Fu, Ph.D., professor of cellular and molecular medicine at UCSD School of Medicine, in a statement. "The fact that we could produce so many neurons in such a relatively easy way came as a big surprise."

RELATED: Mount Sinai researchers uncover new genetic drivers of Parkinson's disease

The UCSD team delivered the gene therapy to the midbrain of the mice and compared them with animals that got a sham procedure. The population of neurons in the brains of mice that received the treatment jumped 30%, and their dopamine levels returned to normal. The control mice saw no improvements, the researchers reported.

Within three months of the gene therapy, the treated mice were able to move their limbs normally, and they remained free of Parkinsons symptoms for the rest of their lives, the team added.

Antisense oligonucleotides are already being used in neurodegenerative diseases. Biogens Spinraza to treat spinal muscular atrophy is an antisense oligonucleotide drug, as are many experimental medicines. Earlier this month, Eli Lilly formed a $20 million deal with Evox Therapeutics, which is using the approach to develop treatments for Duchenne muscular dystrophy and several neurological disorders.

Meanwhile, some research groups are exploring other gene-based approaches to Parkinsons. Last year, a team at Mount Sinai knocked out the gene STMN2 in mice and discovered that nine other genes related to Parkinsons were activateda finding that could lead to new treatment pathways. Startup Prevail Therapeutics is developing a gene therapy to treat Parkinsons thats caused by mutations in the GBA1 gene.

The next step for the UCSD team is to optimize the gene therapy and test it in mouse models of genetic Parkinsons. Theyve patented the technique with plans tofurther optimizeit for testing in people.

See more here:
One-and-done gene therapy wipes out Parkinson's symptoms in mouse models - FierceBiotech

Emergent BioSolutions, a contract development and manufacturing organization (CDMO) currently focused on biologics, plans to widen the range of its offerings by adding viral vector and gene therapy services.

According to the company, this will involve a molecule-to-market service, once it has completed the $75m (67m) investment into its Canton, Massachusetts site.

The capital will be put towards buying a property adjacent to its existing live viral drug substance facility, with the expanded area being a multi-suite operation holding capacity up to the 1,000L scale.

Once the work is completed, the CDMO expects to be able to offer advanced therapy drug substance manufacturing services by the beginning of 2023.

Complementing the Canton site are Emergents additional locations in the US, as the company offers development services out of its Gaithersburg location and drug product manufacturing from Rockville, with the latter site also being expanded and with a completion date set for the end of 2021.

Alongside a growing manufacturing footprint, the company has found its services in demand during the novel coronavirus outbreak.

The US government provided the company with a $628m contract to provide CDMO services for COVID-19 programs, with Emergent set to use part of this funding towards expanding drug product fill/finish capacity.

Prior to this, Emergent had already sealed a manufacturing partnership with Johnson & Johnson to develop its lead COVID-19 vaccine candidate as the latter company worked towards securing a supply of one billion doses for its potential vaccine.

Follow this link:
Emergent in $75m expansion into viral vectors and gene therapy - BioPharma-Reporter.com

On February 67, 2020, the Simons Foundation Autism Research Initiative (SFARI) convened a two-day workshop to explore the possibility of gene therapies for autism spectrum disorder (ASD), a neurodevelopmental condition associated with changes in over 100 genes. Inspired by the recent, stunning successes of gene therapy for the fatal neuromuscular disorder spinal muscular atrophy (SMA)1, and by the accumulation of genes confidently associated with ASD2, SFARI welcomed a diverse collection of researchers to begin to think about whether a similar approach could be taken for ASD. Because gene therapy attempts to fix what is broken at the level of a causative gene, it would offer a more direct and imminent strategy than mitigation of the many and as yet mostly unclear downstream effects of a damaged gene.

The workshop was organized in 20 talks and several discussion panels, which tackled many outstanding issues, including how to choose candidate target genes and predict outcomes; how to optimize vectors for gene delivery; how to decide when to intervene; which animal models to develop; how to find appropriate endpoints for clinical trials and understand the available regulatory pathways. SFARI also raised the question of how its funding might best propel gene therapy efforts amid the emerging, complex ecosystem of academic laboratories, biotech companies, and pharmaceutical industries.

Even the opportunity to have this discussion is very rewarding, said SFARI Investigator Matthew State of the University of California, San Francisco (UCSF), one of the investigators who directed teams of geneticists to analyze the Simons Simplex Collection (SSC).

These efforts have offered up multiple potentially feasible therapeutic targets. Though rare, de novo disruptive mutations in the highest confidence ASD genes often result in severe impairment characterized not only by social difficulties, but also by intellectual disability and seizures. The combination of a single gene mutation of large effect coupled with particularly severe outcomes that include ASD are likely to offer the most immediate targets for gene therapy. For now, this leaves out a large number of individuals with autism for whom genetic causes are not yet known and are likely the result of a combination of many small effect alleles across a large number of genes.

Highlights from talks and discussion panel, chaired by Rick Lifton of Rockefeller University

In the first talk of the workshop, State brought the group up to speed on ASD genomics. The most recent tally from exome-sequencing in simplex cases of ASD highlighted 102 genes in which rare mutations confer individually large risks2. In contrast, the task of identifying common variants carrying very small risks remains quite challenging, with less than a half dozen alleles so far identified with confidence3. The rare, disruptive mutations that result in loss of function of one gene copy are an attractive focus for gene therapy because of the tractability of targeting a single spot in the genome per individual and because, in the vast majority of cases, there remains a single unchanged allele. This points to ways to boost gene and/or protein expression back toward the normal state by leveraging the unaffected copy. But both the limited number of cases known so far combined with the possibility that different mutations to the same gene may have different effects complicate thinking about how to prioritize targets for gene therapy.

State made several points that were continually touched on throughout the workshop. Many ASD genes are highly expressed during midfetal development in the cortex, and additional experiments will need to determine whether and how long a window of opportunity may be present for successful gene therapy postnatally. Given the relatively small number of people with these conditions, new clinical trial designs are needed that dont rely on comparisons between large control and intervention groups (see also Bryan Kings talk below).

Beyond the gene-crippling mutations found in the exome, disruptions to transcription may also dramatically raise risk for autism and may be corrected with a type of gene therapy using ASOs. SFARI Investigator Stephan Sanders of UCSF focused on the role of splicing, the process by which an initial transcript is turned into messenger RNA by removal of introns and joining together of exons. Splicing is disrupted in at least 1.5 percent of individuals with ASD4, and possibly many more, as suggested by transcript irregularities found in postmortem autism brain5. Sanders described Illuminas Splice AI project in which machine-learning helps predict noncoding variants that can alter splicing, including those beyond typical splice sites found near a gene6. As a result of incorporating sequence information around and between splice sites, this computational tool detected more mutations with predicted splice-altering consequences in people with ASD and intellectual disability than in those without the condition.

An ASO designed to bind specific portions of RNA could conceivably correct errors in transcription. ASOs have already been approved for use in other disorders in order to skip exons, retain exons or to degrade mRNA. Unlike other forms of gene therapy, ASOs do not permanently alter the genome, making it a kind of gene therapy lite. This reversibility has both disadvantages (having to re-infuse the ASO every few months) and advantages (multiple opportunities to optimize the dose and target; serious adverse effects are not permanent).

Jonathan Weissman of UCSF discussed the available toolbox for controlling gene expression developed by many different laboratories. To turn genes on or off, he has developed a method to combine CRISPR with an enzymatically inactive (dead) Cas9, which can then be coupled with a transcriptional activator (CRISPRa) or repressor (CRISPRi)7 (Figure 2). In the case of loss-of-function mutations, Weissman outlined strategies to make the remaining good allele work harder: increase transcription via CRISPRa, decrease mRNA turnover, increase translation of a good transcript via modification of upstream open reading frames (uORFs) or increase a proteins stability, possibly through small molecules acting on the ubiquitin system8. That said, the effects on a cell may be complicated. Using Perturb-Seq screens, Weissman described genetic interaction manifolds that show nonlinear mapping between genotype and single cell transcriptional phenotypes9. Additionally, Weissman summarized recent work from his laboratory that has identified large numbers of uORFs that result in polypeptides, some of which affect cellular function.

SFARI Investigator Michael Wigler of Cold Spring Harbor Laboratories echoed the idea of a gene-therapy strategy that increases expression of the remaining good copy of a gene, especially given that in his estimate, 45 percent of simplex cases of autism carried a de novo, likely disrupting variant. He also called attention to the uterine environment, especially the challenge posed by expression of paternally derived antigens in the fetus and the impact of a potential maternal immune response, and the need to understand how it interacts with de novo genetic events.

Highlights from talks and discussion panel, chaired by Arnon Rosenthal of Alector

The discussion turned to finding ways of getting genes into the central nervous system. The AAV is the darling of gene therapy, given that it does not replicate and is not known to cause disease in humans. A version that can cross the blood-brain barrier (AAV9) was used to deliver a gene replacement to children with SMA intravenously; though this effectively delivered the genetic cargo to ailing motor neurons in the spinal cord, it does not work that well at delivering genes throughout the brain.

Ben Deverman of the Stanley Center at the Broad Institute of MIT and Harvard detailed his efforts to optimize AAV for efficient transduction of brain cells through a targeted evolution process: his team engineers millions of variants in the capsid of the virus, then screens them for entry into the nervous system and transduction of neurons and glia. This has yielded versions (called AAV-PHP.B and AAV-PHP.eB) that more efficiently enter the brain10,11. One successfully delivered the MECP2 gene to the brain of a Rett syndrome mouse model, resulting in ameliorated symptoms and an extended lifespan12. Unfortunately, these viruses dont work in human cells or in all mouse strains. A quick mouse genome-wide association study (GWAS) revealed that the Ly6a gene mediates efficient blood-brain barrier crossing of AAV-PHP.B and AAV-PHP.eB13. Now his group has identified Ly6a-independent capsids that may translate better to humans. He also noted that the PHP.B vectors have tissue specificity for brain and liver.

With an estimated 87 percent of autism-associated genes raising risk through haploinsufficiency (having only one functional gene copy out of the two), SFARI Investigator Nadav Ahituv of UCSF made the case for approaches that boost expression of the remaining good copy of a gene through endogenous mechanisms a strategy he called cis-regulation therapy. This method also provides a way to work around the small four kb payload of AAV, which strains to contain cDNA of many autism genes. A recent study by his group used CRISPRa targeted at an enhancer or promoter of SIM1 and promoter of MC4R, both obesity genes, in mice. Using one AAV vector for a dCas9 joined to a transcription activator, and another AAV vector having a guide RNA targeting either a promoter or an enhancer, and a guide RNA targeting a promoter, the researchers injected the vectors together into the hypothalamus, which resulted in increased SIM1 or MC4R transcription and reversed the obesity phenotype brought on by loss of these genes14. Targeting regulatory elements had the added benefit of tissue specificity, and there seemed to be a ceiling effect for SIM1 expression, which suggested an endogenous safeguard against overexpression at work. He is now collaborating with SFARI Investigator Kevin Bender, also at UCSF, to apply this approach to the autism gene SCN2A.

Botond Roska of the Institute of Molecular and Clinical Ophthalmology in Basel, Switzerland pointed out that getting genes to the cells where they are needed is crucial when treating eye diseases. Off-target effects there can induce degeneration of healthy cells. For this reason, Roska and his group have created AAVs that target specific cell types in the retina by developing synthetic promoters that efficiently promote expression of the viruss cargo15. The promoters they designed were educated guesses based on four approaches: likely regulatory elements close to genes expressed with cell-type specificity in the retina, conserved elements close to cell typespecific genes, binding sites for cell typespecific transcription factors and open chromatin close to cell typespecific genes. Screening a library of these in mouse, macaque and human retina revealed some with high cell-type specificity (Figure 3). Importantly, macaque data predicted success in human retina much better than did mouse data. In preliminary experiments, and more relevant to gene therapy for ASD, these cell-specific vectors also had some success in mouse cortex, for example lighting up parvalbumin neurons or an apparently new type of astrocyte.

Roska also described new methods for delivery, in which nanoparticles are coated with AAV, then drawn into the brain using magnets16. This magnetophoresis technique allows a library of experimental AAVs to be tested at the same time in one monkey. Steering nanoparticles with magnets gives more control of vector placement and gene delivery. He argued that these in the future could access even deep structures of the brain.

Highlights from talks and discussion panel, chaired by Steven Hyman of the Broad Institute at MIT and Harvard

Kathy High of Spark Therapeutics reviewed the story of gene therapy for spinal muscular atrophy (SMA) type 1. Though she was not directly involved in that research, she is well aware of the regulatory atmosphere surrounding gene therapy, given that Spark Therapeutics developed the first approved AAV-delivered gene for a form of retinal dystrophy. The SMA story is a useful case study in that an ASO-based therapy (nusinersen, marketed as Spinraza), approved in 2016, set the stage for a gene-replacement therapy, marketed as Zolgensma (onasemnogene abeparvovec). Ultimately, the amount of data supporting Zolgensmas approval was modest: a Phase one dose study of 15 infants1, and an ongoing Phase three trial of 21 infants and safety data from 44 individuals. Yet the approval was helped by the dramatic results and clear endpoints: those receiving a single intravenous infusion of an AAV9 vector containing a replacement gene all remained alive at 20 months of age, whereas only 8 percent survived to that age in the natural history data, which compiles the diseases untreated course. High mentioned that maintaining product quality for gene therapeutics may prove trickier than for typical medications.

The attractive, highly customizable nature of gene therapy might have a regulatory downside in that different vector payloads, even when designed to do the same thing, could invite separate approval processes. Though not knowing how regulatory agencies would view this, High said that their perspectives are bound to evolve as more gene therapy trials are completed.

Getting to ASD-related syndromes, Bender talked about SCN2A, which encodes the sodium channel Nav1.2. SCN2A mutations in humans can be gain of function or loss of function; gain-of-function mutations are associated with early onset epilepsy, and loss-of-function mutations with intellectual disability and ASD. In a mouse model missing one copy of SCN2A, Bender and his group have discovered a role for SCN2A in action potential generation in the first week after birth, and in synaptic function and maturation afterward through regulation of dendritic excitability18 (Figure 4). Using AAV containing CRISPRa constructs developed with the Ahituv lab, the researchers successfully increased SCN2A expression, and recovered synapse function and maturity, even when done several weeks postnatally. Getting the appropriate dosage is critical since gain-of-function mutations are linked to epilepsy. However, Bender reported even when SCN2A expression increased to double normal levels, no hints of hyperexcitability appeared. We might be able to overdrive this channel as much as we want and actually may not have risk of producing an epileptic insult, he said. Next steps are to figure out the developmental windows for intervention, evaluate changes in seizure sensitivity and extend this kind of cis-regulatory approach to other ASD genes.

Angelman syndrome is another condition that attracts interest for gene therapy, in part because neurons already harbor an appropriate replacement gene. Angelman syndrome stems from mutations to the maternally inherited UBE3A gene, which is particularly damaging to neurons because they only express the maternal allele, while the paternal allele is silenced by an antisense transcript. SFARI Investigator Mark Zylka of the University of North Carolina and colleagues showed in 2011 that this paternal allele could be unsilenced with a cancer drug in a mouse model of Angelman syndrome19. Since then, three companies have built ASOs to do the same thing, and these are going into clinical trials. To get a more permanent therapeutic, Zylka has been developing CRISPR/Cas9 systems to reactivate paternal UBE3A, and preliminary experiments show that injecting this construct into the brains of embryonic mice, and then again at birth, results in brain-wide expression of paternal UBE3A and is long-lasting (at least 17 months). Zylka is now making human versions of these constructs. He later noted rare cases of mosaicism for the Angelman syndrome mutation people with 10 percent normal cells in blood have a milder phenotype20, which suggests that even inefficient transduction of a gene vector could help.

Zylka also made a case for prenatal interventions in Angelman syndrome: studies of mouse models indicate that early reinstatement of UBE3A expression in mouse embryos rescues multiple Angelman syndrome-related phenotypes, whereas later postnatal interventions rescue fewer of these21; for humans, a diagnostic, cell-based, noninvasive prenatal test will be available soon22; ultrasound-guided injections into fetal brain of nonhuman primates have been developed23; prenatal surgeries are now standard of care for spinal bifida; and intervening prenatally decreases the risk of an immunogenic response to an AAV vector or its cargo. During the discussion, it was noted that another benefit of acting early was that less AAV would be needed to transduce a much smaller brain; however, a drawback is the lack of data on Angelman syndrome development from birth to one year of age. This natural history would be necessary for understanding whether a prenatal therapy is more effective than treatment of neonates.

SFARI Investigator Guoping Feng of the Massachusetts Institute of Technology has been investigating SHANK3, a high-confidence autism risk gene linked to a severe neurodevelopmental condition called Phelan-McDermid syndrome, which is marked by intellectual disability, speech impairments, as well as ASD. SHANK3 is a scaffold protein important for organizing post-synaptic machinery in neurons. Mouse studies by Feng have shown that SHANK3 re-expression in adult mice that have developed without it can remedy some, but not all, of their phenotypes, including dendritic spine densities, neural function in the striatum and social interaction24. Furthermore, early postnatal re-expression rescued most phenotypes. This makes SHANK3 a potential candidate for gene therapy; however, it is a very large gene 5.2kb as a cDNA that is difficult to fit into a viral vector. To get around this, Fengs group has designed a smaller SHANK3 mini-gene as a substitute for the full-sized version. Preliminary experiments show that AAV delivery of the mini-gene can rescue phenotypes like anxiety, social behavior and corticostriatal synapse function in SHANK3 knockout mice. Feng also discussed his success in editing the genome in marmosets and macaques using CRISPR/Cas9 technology and showed data from a macaque model of SHANK3 dysfunction25. These models may help test gene therapy approaches and identify biomarkers of brain development closely related to the human disorder.

For people with rare conditions brought on by even rarer mutations, individualized gene therapies can provide a pathway for treatment. SFARI Investigator Timothy Yu of Boston Childrens Hospital/Harvard described his N-of-1 study in treating a girl with Batten disease, a recessive disorder in which a child progressively loses vision, speech and motor control while developing seizures. In a little over a year, an ASO that targeted her unusual splice-site mutation in the CLN7 gene was designed, developed and given intrathecally to the girl26. The lift was in negotiating with the FDA and working with private organizations, not just in the science, Yu said. After a year of treatment with the ASO (dubbed milasen after the girl, Mila), there were no serious adverse events; seizure frequency and duration had decreased (Figure 5); and possibly her decline had slowed. Though she remains blind, without intelligible speech and unable to walk on her own, she was still attentive and could respond happily to her familys voices. The highly personalized framework for this drugs approval is completely different from how medications meant for populations are approved, and it opens a regulatory can of worms, Yu said, though he added that the regulators were willing to countenance drug approval for an individuals clinical benefit.

Rett syndrome is a neurodevelopmental condition caused by mutations to the MECP2 gene that has a substantial research base in mouse models. Over 10 years ago, mouse models highlighted the possibility for therapeutics in this condition when Rett-associated phenotypes were rescued by adding back MECP2, even in adulthood27. This reversibility has spurred interest in gene therapy for Rett syndrome, but getting the MECP2 dose right is critical, said Stuart Cobb of the University of Edinburgh and Neurogene: just as too little MECP2 leads to Rett syndrome, too much also results in severe phenotypes. For this reason, it would be nice to package a replacement MECP2 gene with other regulatory elements to control its expression, but this results in constructs that do not fit into viral vectors. To make more room, Cobb and his colleagues have been able to chop away two-thirds of the MECP2, reserving two domains that interact to make a complex on DNA (Figure 6). Mice with this mini-gene are viable and have near normal phenotypes; likewise, injecting this mini-gene into MECP2-deficient mice extended their survival28. Doubling the dose, however, substantially lowered survival. Putting in safety valves to prevent overexpression is going to be quite important, he said. One idea is to add back a construct containing only the last two exons of MECP2, which is where most Rett mutations land. These would then be spliced into native transcripts (called trans-splicing), and thus their expression controlled by endogenous regulatory elements.

Underscoring the double-edged sword of MECP2 dosage, Yingyao Shao from Huda Zoghbis lab at Baylor described an MECP2 duplication syndrome (MDS) in humans, which features hypotonia, intellectual disability, epilepsy and autism. Experiments in an MDS mouse model, which carries one mouse version and one human version of MECP2, recapitulates some of the phenotypes of the human condition and can be rescued by an ASO targeting the human allele29. Shao described work to optimize the ASO for translation into humans, which involved developing a more humanized MDS model that carries two human MECP2 alleles. An acute injection of the ASO was able to knock down MECP2 expression in a dose-dependent manner in these mice, and RNA levels dropped a week after injection, with protein levels falling a week later. MECP2 target genes also normalized their expression level, and one maintained this for at least 16 weeks post-injection. The ASO also rescued behavioral phenotypes of motor coordination and fear conditioning, but not of anxiety; these corrections followed the molecular effects, and these timelines would be important to keep in mind while designing clinical trials. Shao also noted that overtreatment with the ASO resulted in Rett-associated phenotypes, but that this was reversible, which suggests that some fine-tuning of dosing in humans might be possible.

To avoid overtreatment and toxicity of any MDS-directed therapy, Mirjana Maletic-Savatic, also at Baylor, is leaving no stone unturned in a hunt for MDS biomarkers that can predict, in each individual, the safety of a particular dose and regimen. Such biomarkers would also help monitor individuals during treatment, give information about target engagement and identify candidates for a particular treatment. Anything found to be sensitive to expression levels of MECP2 could also be useful for Rett, though she noted that MECP2 levels measured in blood do not track linearly with gene copy number. Thus, because of interindividual variability, her approach is to collect a kitchen sink of data deriving composite biomarkers that accurately reflect the stage and severity of disease in a given case. She and her colleagues are collecting clinical, genetic, neurocircuitry (such as EEG and sleep waves), immunology and molecular data detected in blood, urine and CSF. These measures are also being explored in induced neurons derived from skin samples of people with MDS. She highlighted two interrelated potential biomarkers in the blood of those with this condition; both measures are downstream targets of MECP2 and are responsive to ASO treatment.

Highlights from Early detection and clinical trial issues talks and panel discussion, chaired by Paul Wang of SFARI

Coming up with objective measures of a persons status either their eligibility for a treatment, or whether the treatment has engaged with its target or even whether the treatment is effective is a real necessity in autism-related conditions, which comprise multiple interrelated behaviors. Eye-tracking methodology may provide such a marker, argued SFARI Investigator Ami Klin of Emory University. Focusing on the core social challenges of autism, Klin, Warren Jones and colleagues have been studying children as they view naturalistic social scenes to quantify their social attention patterns. This has revealed how remarkably early in development social visual learning begins and that this process is disrupted in infants later diagnosed with ASD prior to features associated with the condition appearing. By missing social cues, autism in many ways creates itself, moment by moment, Klin said. In considering gene therapy, it may be useful to know that eye looking (how much a subject looks at a persons eyes, an index of social visual engagement) in particular and social visual engagement in general are under genetic control30; that eye-tracking differences emerge as early as 26 months of age; and that homologies in social visual engagement exist between human babies and nonhuman infant primates.

In getting to a point to test gene therapies, identifying those who need them is essential. Wendy Chung of Columbia University and the Simons Foundation illustrated how diagnosis is yoked closely to therapy. To illustrate this, she described her pilot study of newborn blood spots to screen for SMA; at the start, no treatment was available, but the screen identified newborns for a clinical trial of nusinersin. Notably, the screen only cost an additional 11 cents per baby. In the three years since her pilot screen began, the FDA approved two gene therapies for SMA and the SMA screen was adopted for nationwide newborn screening. Currently she is piloting a screen for Duchenne muscular dystrophy and plans to develop a platform that will allow researchers to add other conditions. In prioritizing genetic conditions for gene therapy, she outlined some ideas for focus, such as genes resulting in phenotypes that would not be identified early without screening, those that are relatively frequent, those that are lethal or neurodegenerative, those with a treatment in clinical trials or with FDA-approved medications, and those conditions that are reversible.

In the meantime, Chung also outlined SFARIs involvement in establishing well-characterized cohorts of individuals with autism, which can help lay a groundwork for gene therapy. People with an ASD diagnosis can join SPARK (Simons Foundation Powering Autism Research for Knowledge), which collects medical, behavioral and genetic information (through analysis of DNA from saliva, at no cost to the participant). If a de novo genetic variant is found in one of ~150 genes, that person is referred to Simons Searchlight, which fosters rare conditions communities and which is also compiling natural history data on people with these mutations.

Bryan King of UCSF discussed how current trial designs for ASD were inadequate for gene therapy trials. As ASD prevalence has grown, parallel design trials with one group receiving an experimental medicine and the other a placebo are the standard, but these wont be possible for the rare conditions that are candidates for gene therapy. Also, change is hard to capture, given the malleable nature of ASD: with no intervention, diagnosis can shift between ASD and pervasive developmental disorder-not otherwise specified (PDD-NOS) in 1284 months (as defined by the DSM-IV). Current scales are subjective and may miss specific items of clinical significance. (Last year, SFARI funded four efforts to develop more sensitive outcome measures.) King outlined other pitfalls in ASD clinical trials, including significant placebo responses, inadequate sample sizes and not being specific enough when asking about adverse effects. King also mentioned improvements that may arise from just enrolling in a study, which could prompt previously housebound families to venture out with their child, which could kick off a cascade of positive effects. He reiterated how, for gene therapy, a natural history comparison group may be more appropriate, combined with solid outcome measures.

SFARI Investigator James McPartland of Yale University then underlined the need for objective biomarkers for clinical trials, for which there are currently none that are FDA qualified for ASD. As the director of the Autism Biomarkers Consortium for Clinical Trials (ABC-CT), he works with other scientists to develop reliable biomarkers that can be scaled for use in large samples across different sites. McPartland noted a biomarker studied in the ABC-CT: an event-related potential (N170) to human faces, which is on average slower in ASD than in typically developing children. He is working on ways to make it easier for people with ASD and intellectual disabilities to participate in biomarker studies and to make them more socially naturalistic. In discussion, he mentioned he thought it would be possible to look for these kinds of biomarkers in younger children.

SFARI Investigator Shafali Jeste of the University of California, Los Angeles recounted her experience in working with children with genetic syndromes associated with neurodevelopmental conditions. Though she is asked to participate in clinical trials for these conditions, she senses the field has some work to do to be ready for these trials, particularly in those with additional challenges such as epilepsy and intellectual disability. Meaningful and measurable clinical endpoints are still insufficient, and there needs to be more ways to improve accessibility of these trials for these rare conditions. This means developing new measures, such as gait-mat technology that senses walking coordination, or EEG measures in waking and sleep, which have been applied to people with chromosome 15q11.2-13.1 duplication (dup15q) syndrome, who have severe intellectual disability and motor impairments. Jeste also emphasized that increasing remote access to some measures can make a big difference for a trial; for example, a trial of a behavioral intervention for tuberous sclerosis complex that required weekly lab visits was disappointingly under-enrolled until researchers revamped it so most of the intervention could be done remotely31.

By grappling with the challenges to gene therapy for ASD, the workshop marked out a faint road map of a way forward. As the scientific questions are answered, the regulatory and clinical trial infrastructure will need to develop apace, and coordination between private, academic and advocacy sectors will be essential. But as gene therapy for diverse human conditions continues to be explored and gene discovery in ASD continues, there is reason to believe that some forms of ASD can eventually benefit from this strategy.This workshop provided a terrific discussion about the challenges in developing targeted gene interventions and their potentially transformative effects as therapies, said John Spiro, Deputy Scientific Director of SFARI. We are grateful to all theparticipants, and SFARI looks forward to translating these discussions into focused funding decisions in the near future.

Back to Top

Go here to see the original:
SFARI | SFARI workshop explores challenges and opportunities of gene therapies for autism spectrum disorder - SFARI News

Dive Brief:

Known for its muscular dystrophy drugs Exondys 51 and Vydonys 53, Sarepta has quickly become a leader in gene therapy as well.

The last couple of years have seen the Cambridge, Massachusetts-based biotech ink gene therapy manufacturing and development deals, including a partnership with Roche that could be worth more than $3 billion.

Sarepta now has six gene therapies in clinical testing and another half dozen in preclinical stages. Most recently, the company announced positive, albeit early, results from a study of its experimental treatment for limb-girdle muscular dystrophy, a potentially deadly genetic disease.

With gene therapy set to become a cornerstone of its business, Sarepta is trying to avoid some the challenges presented by current technologies.

For example, when the company teamed up with North Carolina-based StrideBio late last year, one of the main goals was to use the partner's technology to "address re-dosing challenges in patients who have received AAV-delivered gene therapy." The deal came just days after a study testing an experimental gene therapy from Solid Biosciences, one of Sarepta's rivals, was paused due to a patient experiencing an immune response and organ complications.

"If successful, the ability to re-dose will be an enormous leap forward in the science of gene therapy and provide invaluable benefits to patients beyond those we anticipate with one-time dosing," said Doug Ingram, Sarepta's CEO, in a June 18 statement announcing the Selecta deal.

Under terms of that deal, Sarepta will pay Selecta an initial, undisclosed amount. Selecta is eligible to receive pre-clinical milestone payments, and could take home additional development, regulatory and commercial milestones should Sarepta exercise its options to enter a licensing agreement.

Specifically, the deal with Selecta centers on gene therapies for Duchenne muscular dystrophy and certain limb-girdle muscular dystrophies.

With Codiac, Sarepta has offered up $72.5 million in upfront and near-term license payments plus research funding. In addition, Codiak is eligible for "significant" milestone payments, according to Sarepta.

The two-year deal gives Sarepta the option to license Codiak's technology for up to five neuromuscular targets. The companies said they will collaborate on the design of exosomes that can deliver and release gene therapy, gene editing or RNA payloads. If Sarepta exercises an option, Codiak will then be in charge of research until right before the candidate goes into in-human testing. Sarepta is responsible for clinical development and commercial activities.

Here is the original post:
Sarepta tries to fine-tune its gene therapy approach - BioPharma Dive

Dive Brief:

Huntington's disease is marked by physical and cognitive decline caused by cellular production of an abnormal version of a protein called huntingtin, which is essential for nerve cell health. About 30,000 people in the U.S. have the condition.

No treatments exist for the underlying disease, but Lundbeck's Xenazine and Teva's Austedo can help control the abnormal movements associated with Huntington's.

AMT-130, by contrast, uses a common virus to deliver strips of nucleic acid that can block the mutated genes that cause production of abnormal huntingtin. It is the first gene therapy to be tested in Huntington's disease patients.

UniQure plans to enroll 26 patients in this trial, with two groups receiving different doses of AMT-130 and a placebo arm that involves a partial surgery to make it appear patients have received the injection deep in the brain.

The main goal of the trial will be to determine if the therapy is safe, but as a secondary goal researchers will want to see how persistent AMT-130 is in the brain. Researchers will also be looking for signs that patients receiving the gene therapy decline more slowly than those who didn't, but the small trial may not be able to definitively answer that question.

UniQure likely won't be alone in this chase for long. Voyager, which has already advanced a Parkinson's disease gene therapy into the clinic in partnership with Neurocrine Biosciences, is nearing a decision on when to advance VY-HTT01 into the clinic.

At its first quarter corporate update, Voyager said it is "engaged in the ongoing conduct and review of preclinical studies" and would provide an update at mid-year.

View post:
UniQure gets out the gate first in race for Huntington's gene therapy - BioPharma Dive

XconomyPhiladelphia

The science underpinning the latest investigational cell and gene therapies is complex enough. But the rapid advance of technologies that support development of these kinds of drugs presents logistical considerations, too.

Drug development is an interesting process because it takes five to 10 years to get a drug to market and invariably, somewhere along that line, the technologys a little outdated before it even is approved, and theres new things out there that are better, said Jeffrey Castelli, chief portfolio officer and head of gene therapy at Amicus Therapeutics (NASDAQ: FOLD). You look at some of the gene therapies that are now just ready coming to market and you think, wow, thats a pretty outdated gene therapy approachbut you really get locked in as youre moving your product toward development.

Castelli was among the executives who spoke as part of a panel at Xconomys online Xcelerating Life Sciences Philadelphia forum last week.

Amicus, headquartered in Cranbury Township, NJ, operates a gene therapy R&D center in Philadelphia.To keep its therapies from becoming stale prior to commercialization, the company keeps an eye on new technologies that arise, Castelli said. But some innovations invariably surface too late to incorporate.

We really try to implement them early on, and then make sure that when we have our second wave of programs and products weve incorporated some of the innovation, he said. But you do get locked into your approach along the way, and theres more and more switching costs as you move along that pathway.

To guide that process Tom Wilton, chief business officer at Philadelphia-based cell therapy company Carisma Therapeutics, says its essential to collect and analyze data early on to guide the path of drug development.

As a relatively small company youve really got to focus the majority of your resource on pushing forward that lead program and getting it into the clinic, he said. Later this year well get a lot of data back from that first-in-human clinical study, the first time anyones ever taken an engineered macrophage into the clinic. What that should give us then is a set of criteria and priorities around what we need to bring forward in a next-generator program.

To date the companys consideration of new technologies has been perhaps a bit more opportunisticeverything from gene editing the macrophages to novel binders to different combination strategiesbut once Carisma has the initial clinical data in hand, that will narrow its focus, Wilton said.

Theres always a tendency [with a first-generation product] to say we could do this to this, we could do this to this, but you have to lock it down, you have to get into the clinic and get that data set to really understand what your priorities are and where you need to focus, and thats what were planning to do.

As experimental products move into later stages of development, another consideration arises, the panelists said: Producing them at scale.

For cell and gene therapy companies, figuring out when to lock in the space needed to make these drugs so it is available when needed for clinical trials or commercialization is difficult to determine precisely, especially given the shortage of manufacturers versed in some of the advanced technologies needed to do so.

There is a capacity shortage right now in cell and gene therapy manufacturing, said Audrey Greenberg, co-founder and executive managing director of Discovery Labs, an MLP Ventures-backed biotech coworking and incubator space in King of Prussia, PA. Theres estimates that its five times [current cumulative bioreactor volumetric capacity] now and will be 50 times in five years given the FDA pipeline and the dollars flowing into these companies.

Discovery Labs is building out a $1.1 billion gene and cell treatment manufacturing operation plus developing a contract development manufacturing organization to provide services to companies in the sector.

Image: iStock/f11photo

Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.

More here:
Timing Is an Essential Element of Cell & Gene Therapy Product Development - Xconomy

On the heels of research deals with two small biotechs, shares of Sarepta Therapeutics Inc. (NASDAQ:SRPT) hit their all-time high of more than $172 on June 23.

The share price of the Cambridge, Massachusetts-based biopharmaceutical company has doubled since mid-March. It has a market cap of more than $13 billion.

Sarepta has made several deals in the past several years that have made gene therapy a key part of its business, which had been dominated by drugs for muscular dystrophy. One of those pacts, with Roche (RHHBY), could be worth more than $3 billion.

Sarepta currently has a half dozen gene therapies in clinical trials, with another six waiting in the wings, according to BioPharma Dive. One of the company's compounds has shown promise in treating a type of muscular dystrophy, a genetic disease that causes weakness and wasting of the muscles in the arms and legs.

A major challenge of gene therapy is immune system reactions. Sarepta, like other developers, uses a type of virus in its therapy that, while effective, may not be able to given more than once because patients can create antibodies to it.

That's a problem Sarepta is trying to address in its latest deals with privately held Codiak Biosciences and Selecta Biosciences Inc. (NASDAQ:SELB). In both cases, Sarepta has an option to license the biotechs' technology to develop and commercialize its therapies.

The Selecta deal focuses on gene therapies for Duchene muscular dystrophy and certain limb-girdle muscular dystrophies.

A Zion Market Research report said the global demand for the Duchenne muscular mystrophy therapeutics market was valued at approximately $2.4 billion in 2018 and is expected to grow to more than $20 billion by the end of 2025, a compound annual growth rate of more than 36% between 2019 and 2025.

Given the size of the opportunity, it's no surprise Sarepta has plenty of competitors vying for a share of the business, including Pfizer Inc. (NYSE:PFE), PTC Therapeutics (NASDAQ:PTCT), FirbroGen Inc. (NASDAQ:FGEN), Roche and Bristol-Myers Squibb Co. (NYSE:BMY).

14b4c0913859c7da3fa2339160bde942.png

The two-year deal with Codiak gives Sarepta the right to license its technology for up to five neuromuscular diseases. If Sarepta exercises an option, Codiak will then handle research until right before the candidate goes into human testing. Sarepta will then be responsible for clinical development and commercializing the drug.

In April, the company announced it has resurrected its antiviral program in response to Covid-19. It has a therapy that is meant to block the coronavirus' ability to replicate. The treatment will be tested at the U.S. Army Medical Research Institute of Infectious Diseases.

"If it works, it will reduce the ability of the virus to replicate" and its ability to infect other cells, Sarepta CEO Doug Ingram told Forbes. He cautioned that the drug is at an "early stage."

According to CNN Money, the 21 analysts offering 12-month price forecasts for Sarepta have a median target of $192, with a high estimate of $260 and a low $152. The stock is rated a buy.

Disclosure: The author hold positions in Pfizer and Bristol-Myers Squibb.

Read more here:

Not a Premium Member of GuruFocus? Sign up for a free 7-day trial here.

This article first appeared on GuruFocus.

Read the original post:
Sarepta Addressing Gene Therapy Issue With Two Acquisitions - Yahoo Finance

A group of collaborating scientists received a $13.65 million federal grant to study and develop a CAR-T therapy that will genetically modify immune cells and potentially cure HIV, according to a press release from the University of California, Los Angeles (UCLA).

The National Institutes of Health (NIH) funds the five-year grant as part of its effort to support HIV cure research. Participating researchers are affiliated with UCLA, the University of WashingtonFred Hutchinson Cancer Research Center and CSL-Behring, a biotech company based in the United States and Australia.

The overarching goal of our proposed studies is to identify a newgene therapy strategy to safely and effectively modify a patients own stem cells to resist HIV infection andsimultaneously enhance their ability to recognize and destroy infected cells in the body in hopes of curing HIV infection, said UCLAs Scott Kitchen, PhD, an associate professor of medicine in the division of hematology and oncology, in the press release. Kitchen will colead the research with Irvin Chen, PhD, director of the UCLA AIDS Institute at the David Geffen School of Medicine.

Transplantation ofHIV-resistant stem cells is the only approach that has ever led to a known cure for HIV(andlikely a second such cure). But stem cell transplants are risky and can only be done in people with HIVwho need them for cancer treatment. Using gene therapy tomodify an individuals own stem cells might be a safer way toachieve the same result.

The Food and Drug Administration first approved CAR-T therapywhich stands for chimeric antigen receptor T-cell therapyin 2017. Its used to treat some forms of cancer, but as POZs sister publication Cancer Health has reported, it hasnt been commonly used because it is expensive and must be custom made for each patient.

In the case of cancer treatment, CAR-T therapy involves taking a patients T cells and sending them to alab where they are genetically modified to recognize and attack the cancer. The resulting cells are then infused back into the individual after the person has received strong chemotherapy to kill off some of their existing immune cells to make room for the new ones.

In CAR-T therapy for HIV, blood-forming stem cells would be genetically engineered togive rise to T cells that would seek out and destroy cells infected with HIV.

In a recent early study of the approach, the UCLA scientists found that engineered CAR T cells destroyed HIV-infected cellsand lived for more than two years.

Our work under the NIH grant will provide a great deal of insight into ways the immune response can be modified to better fight HIV infection, said Chen, a professor of medicine and of microbiology, immunology and molecular genetics at the Geffen School of Medicine. The development of this unique strategy that allows the body to develop multiple ways to attack HIV could have an impact on other diseases as well, including the development of similar approaches targeting other types of chronic viral infections and cancers.

View post:
$14M Federal Grant to Research CAR-T Gene Therapy to Cure HIV - POZ

SOUTH SAN FRANCISCO, Calif., June 19, 2020 (GLOBE NEWSWIRE) -- Catalyst Biosciences, Inc. (NASDAQ: CBIO), today presented data from preclinical studies of its hemophilia B gene therapy CB 2679d-GT at the WorldFederation of Hemophilia Virtual Summit, taking place from June 14 -19, 2020.

The oral presentation, entitled: Combination of a Novel Chimeric AAV Capsid and Potency Enhanced FIX Variant for Hemophilia B Gene Therapy, given by Dr. Grant Blouse, senior vice president of translational research, provided preclinical results of CB 2679d-GT, the companys novel FIX gene therapy. CB 2679d-GT was designed to achieve clinically relevant FIX levels at a reduced viral load by combining engineered AAV capsids with Catalysts novel high potency FIX transgene.

The preclinical data from our constructs demonstrated a strong dose response and improved reduction in bleeding relative to the Padua variant, said Nassim Usman, Ph.D., president and chief executive officer of Catalyst. The enhanced FIX activity and reduced viral dose may offer advantages over current AAV-based gene therapies in clinical development.

Studies of CB 2679d-GT in hemophilia B mice have demonstrated a 4-fold reduction in blood loss and an 8-fold reduction in bleeding time when compared with the same dose of the Padua variant of FIX. Furthermore, when packaged in a proprietary chimeric AAV capsid, CB 2679d-GT demonstrated a clear dose response of high stable FIX levels across the three dose levels in hemophilia B mice.

A pilot non-human primate study compared the expression and tolerability of CB 2679d-GT in the novel chimeric capsid KP1 with the LK03 capsid. The study demonstrated that CB 2679d-GT was well tolerated with high FIX expression that stabilized to approximately 25% to 50% FIX above baseline levels at the 6-week interim data cutoff. The novel chimeric capsid had differentiated and superior response to anti-capsid neutralizing antibodies than that observed for the LK03 comparator during the screening of non-human primates for the study.

A copy of the presentation slides can be accessed on the Events and Presentations section of the Catalyst website.

About Catalyst BiosciencesCatalyst is a research and clinical development biopharmaceutical company focused on addressing unmet needs in rare hematologic and systemic complement-mediated disorders. Our protease engineering platform includes development programs in hemophilia, a research program on subcutaneous (SQ) systemic complement inhibitors and a partnered preclinical development program with Biogen for dry age-related macular degeneration (AMD). One of our key competitive advantages is that the product candidates generated by our protease engineering platform have improved functionality and potency. These characteristics allow for improved dosing of our candidates including SQ systemic administration of recombinant coagulation factors and complement inhibitors, low-dose, high activity gene therapy constructs, and less frequently dosed intravitreal therapeutics. Our most advanced asset, SQ MarzAA has successfully completed Phase 2 development in prophylaxis, significantly reducing the annualized bleed rate (ABR) in individuals with Hemophilia A or B with inhibitors. Following regulatory guidance from the U.S. Food and Drug Administration and European Medicines Agency, we recently announced the design of a Phase 3 registration study that is planned for late 2020. Subcutaneous dalcinonacog alfa (DalcA) is being developed for the treatment of Hemophilia B and has demonstrated efficacy and safety in a Phase 2b clinical trial. We have a discovery stage Factor IX gene therapy construct - CB 2679d-GT - for Hemophilia B, that has demonstrated superiority compared with the Padua variant in preclinical models. Finally, we have a global license and collaboration agreement with Biogen for the development and commercialization of anti-complement Factor 3 (C3) pegylated CB 2782 for the potential treatment of geographic atrophy-associated dry AMD.

Forward-Looking StatementsThis press release contains forward-looking statements that involve substantial risks and uncertainties. Forward-looking statements include statements about the superiority of CB 2679d-GT over the Padua variant, enhanced FIX activity of CB 2679d-GT, which may reduce viral dose and maintain high FIX activity levels while potentially decreasing liver toxicity, the chimeric capsid, which may have lower neutralization by pre-existing AAV antibodies, as well as plans for a Phase 3 trial of MarzAA in late 2020 and the Companys collaboration with Biogen for the development and commercialization of pegylated CB 2782 for the potential treatment of geographic atrophy-associated dry age-related macular degeneration. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially, including, but not limited to, the risk that trials and studies may be delayed as a result of the COVID-19 virus and other factors, that trials may not have satisfactory outcomes, that additional human trials will not replicate the results from animal trials or earlier human trials, that potential adverse effects may arise from the testing or use of DalcA or MarzAA, including the generation of neutralizing antibodies, which has been observed in patients treated with DalcA, the risk that costs required to develop or manufacture the Companys products will be higher than anticipated, including as a result of delays in development and manufacturing resulting from COVID-19 and other factors, the risk that Biogen will terminate Catalysts agreement, competition and other risks described in the Risk Factors section of the Companys quarterly report filed with the Securities and Exchange Commission on May 11, 2020, and in other filings with the Securities and Exchange Commission. The Company does not assume any obligation to update any forward-looking statements, except as required by law.

Contact:Ana KaporCatalyst Biosciences, Inc.investors@catbio.com

Read more from the original source:
Catalyst Biosciences Presents Preclinical FIX Gene Therapy Data in an Oral Presentation at the World Federation of Hemophilia Virtual Summit 2020 -...

Washington, DC, June 24, 2020 (GLOBE NEWSWIRE) -- via NEWMEDIAWIRE -- The Alliance for Regenerative Medicine (ARM), an international multi-stakeholder advocacy organization representing the regenerative medicine and advanced therapy sector, today announced a new virtual format for its annual Cell & Gene Meeting on the Mesa, which will take place October 12-16, 2020.

ARM's leadership and Board of Directors arrived at the decision to host the 2020 Cell & Gene Meeting on the Mesa virtually after consulting with numerous ARM members and gathering information from various health authorities. Ultimately, hosting the meeting virtually will be the safest and most inclusive solution, allowing attendees to connect with potential partners globally while avoiding any risk to safety.

This year's Cell & Gene Meeting on the Mesa will bring together senior executives from leading cell therapy, gene therapy, and tissue engineering companies worldwide, large pharma and biotech, institutional investors, academic research institutions, patient foundations and disease philanthropies, life science media, and more.

During this unprecedented period of social distancing, ARM continues to provide members with avenues to engage and connect, said ARM CEO Janet Lambert. Our virtual Meeting on the Mesa is an invaluable opportunity for stakeholders from across the sector to convene, to network, and to continue to work to ensure innovative cell and gene therapies reach patients in need.

The conference, which will now take place over five days, includes a virtual form of the meetings signature partnering system, expected to facilitate more than 3,000 one-to-one meetings between industry leaders. The program will include 15+ digital panels and workshops featuring key industry leaders discussing issues and trends in the regenerative medicine and advanced therapy sector, from market access to the latest discoveries in gene editing. Representatives from more than 80 prominent public and private companies will deliver on-demand presentations highlighting their clinical and commercial progress to interested partners and investors.

Additional event details will be updated regularly on the event website http://www.meetingonthemesa.com.

Registration is currently open, with discounted early-bird rates available through July 24. Registration is complimentary for investors and credentialed members of the media. To learn more and to register, please visitwww.meetingonthemesa.com. For members of the media interested in attending, please contact Kaitlyn (Donaldson) Dupont atkdonaldson@alliancerm.org.

For interested organizations looking to increase exposure to this fields top decision-makers via sponsorship, please contact Laura Stringham at lparsons@alliancerm.org for additional information.

About the Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory, and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors, and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 350 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visithttp://www.alliancerm.org.

Kaitlyn (Donaldson) Dupont

803-727-8346

kdonaldson@alliancerm.org

Continue reading here:
The Alliance for Regenerative Medicine Announces Its 2020 Cell & Gene Meeting on the Mesa Goes Virtual - GlobeNewswire

Prophecy Market Insights Global Cell and Gene Therapy Market market research report focuses on the market structure and various factors affecting the growth of the market. The research study encompasses an evaluation of the market, including growth rate, current scenario, and volume inflation prospects, based on DROT and Porters Five Forces analyses. The market study pitches light on the various factors that are projected to impact the overall market dynamics of the Global Cell and Gene Therapy Market market over the forecast period (2019-2029).

The data and information required in the market report are taken from various sources such as websites, annual reports of the companies, journals, and others and were validated by the industry experts. The facts and data are represented in the Global Cell and Gene Therapy Market report using diagrams, graphs, pie charts, and other clear representations to enhance the visual representation and easy understanding the facts mentioned in the report.

Get Sample Copy of This Report @ https://www.prophecymarketinsights.com/market_insight/Insight/request-sample/4053

The Global Cell and Gene Therapy Market research study contains 100+ market data Tables, Pie Chat, Graphs & Figures spread through Pages and easy to understand detailed analysis. The predictions mentioned in the market report have been derived using proven research techniques, assumptions and methodologies. This Global Cell and Gene Therapy Market market report states the overview, historical data along with size, share, growth, demand, and revenue of the global industry.

All the key players mentioned in the Global Cell and Gene Therapy Market market report are elaborated thoroughly based on R&D developments, distribution channels, industrial penetration, manufacturing processes, and revenue. Also, the report examines, legal policies, and competitive analysis between the leading and emerging and upcoming market trends.

Global Cell and Gene Therapy MarketMarket Key Companies:

Segmentation Overview:

Global Cell and Gene Therapy Market, By-Products:

Global Cell and Gene Therapy Market, By Distribution Channel Type:

Global Cell and Gene Therapy Market, By End-Users:

Apart from key players analysis provoking business-related decisions that are usually backed by prevalent market conditions, we also do substantial analysis on market segmentation. The report provides an in-depth analysis of the Global Cell and Gene Therapy Market market segments. It highlights the latest trending segment and major innovations in the market. In addition to this, it states the impact of these segments on the growth of the market.

Request [emailprotected] https://www.prophecymarketinsights.com/market_insight/Insight/request-discount/4053

Regional Overview:

The survey report includes a vast investigation of the geographical scene of the Global Cell and Gene Therapy Market market, which is manifestly arranged into the localities. The report provides an analysis of regional market players operating in the specific market and outcomes related to the target market for more than 20 countries.

Australia, New Zealand, Rest of Asia-Pacific

Key Questions Answered in Report:

Stakeholders Benefit:

About us:

Prophecy Market Insights is specialized market research, analytics, marketing/business strategy, and solutions that offers strategic and tactical support to clients for making well-informed business decisions and to identify and achieve high-value opportunities in the target business area. We also help our clients to address business challenges and provide the best possible solutions to overcome them and transform their business.

Contact Us:

Mr. Alex (Sales Manager)

Prophecy Market Insights

Phone: +1 860 531 2701

Email: [emailprotected]

VISIT MY BLOG:- https://medium.com/@varshashirode0000/global-voice-user-interface-market-analysis-and-forecast-till-2029-7f7bd61f113

See the original post:
Global Cell and Gene Therapy Market: Revenue Growth and Applications Insights - Cole of Duty

A new business intelligence report released by CMI with title Global Cell and Gene Therapy Market Insights, Forecast to 2027 that targets and provides comprehensive market analysis with future prospects to 2024. The analysts of the study have garnered extensive research methodologies and data sources (i.e. Secondary & Primary Sources) in order to generate collective and useful information that delivers latest market undercurrents and industry trends.

If you are involved in the Global Cell and Gene Therapy industry or intend to be, then this study will provide you comprehensive outlook. Its vital you keep your market knowledge up to date segmented by major players. If you have a different set of players/manufacturers according to geography or needs regional or country segmented reports we can provide customization according to your requirement.

We Do Offer Sample of this report. Kindly go through the follow information in order to access sample copy.

Note- This report sample includes:

Brief Introduction to the research report.

Table of Contents (Scope covered as a part of the study)

Top players in the market

Research framework (structure of the report)

Research methodology adopted by Coherent Market Insights

Request Sample Report @ https://www.coherentmarketinsights.com/insight/request-sample/2475

This study categorizes the global Cell and Gene Therapy breakdown data by manufacturers, region, type and application, also analyzes the market status, market share, growth rate, future trends, market drivers, opportunities and challenges, risks and entry barriers, sales channels, distributors and Porters Five Forces Analysis. Global Cell and Gene Therapy market size will increase to Million US$ by 2027, from Million US$ in 2017, at a CAGR of during the forecast period. In this study, 2017 has been considered as the base year and 2020 to 2027 as the forecast period to estimate the market size for Cell and Gene Therapy. This report focuses on the top manufacturers Cell and Gene Therapy capacity, production, value, price and market share of Cell and Gene Therapy in global market. The following manufacturers are

Competition Analysis:

Some of key competitors or manufacturers included in the study are Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST.

Detailed Segmentation:

By Therapy Type:Cell TherapyStem CellsT CellsDendritic CellsNK CellsTumor CellsGene TherapyGlobal Cell and Gene Therapy Market, By Indication:Cardiovascular DiseaseCancerGenetic DisorderInfectious DiseaseNeurological DisordersOthers

Market Analysis by Geographies:

This report is segmented into key Regions to identify significant trends and factors driving or inhibiting the market growth. To analyze the opportunities in the market for stakeholders by identifying the high growth segments., To strategically analyze each submarket with respect to individual growth trend and their contribution to the market. & to analyze competitive developments such as expansions, agreements, new product launches, and acquisitions in the market. With Production Development, Sales, and Regional Trade & Forecast.

Stay up-to-date with Global Cell and Gene Therapy market research offered by CMI. Check how key trends and emerging drivers are shaping this industry growth as the study avails you with market characteristics, size and growth, segmentation, regional breakdowns, competitive landscape, shares, trend and strategies for this market. In the Cell and Gene Therapy Market Analysis & Forecast 2020-2027, the revenue is valued at USD XX million in 2017 and is expected to reach USD XX million by the end of 2027, growing at a CAGR of XX% between 2020 and 2027. The production is estimated at XX million in 2017 and is forecasted to reach XX million by the end of 2027, growing at a CAGR of XX% between 2020 and 2027.

Get PDF Brochure Report @https://www.coherentmarketinsights.com/insight/request-pdf/2475

Some of the Points cover in Global Cell and Gene Therapy Market Research Report is:

Chapter 1: Overview of Global Cell and Gene Therapy Market (2020-2027)

Chapter 2: Market Competition by Players/Suppliers 2015 and 2020

Chapter 3: Sales (Volume) and Revenue (Value) by Region (2015-2020)

Chapter 4, 5 and 6: Global Cell and Gene Therapy Market by Type, Application & Players/Suppliers Profiles (2015-2020)

Chapter 7, 8 and 9: Global Cell and Gene Therapy Manufacturing Cost, Sourcing & Marketing Strategy Analysis

Chapter 10 and 11: Cell and Gene Therapy Market Effect Factors Analysis and Market Size (Value and Volume) Forecast (2020-2027)

Chapter 12, 13, 14 and 15: Global Cell and Gene Therapy Market Research Findings and Conclusion, appendix and data source

Some of the key questions answered in this report:

Note: Access insightful study with over 150+ pages, list of tables & figures, profiling 20+ companies.

Buy This Complete A Business Report @https://www.coherentmarketinsights.com/insight/buy-now/2475

Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, Europe or Asia.

About Us:

Coherent Market Insights is a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity.

Contacts US:

Mr. ShahEmail:sal[emailprotected]Phone: US +1-206-701-6702/UK +44-020 8133 4027

Follow this link:
Impact Analysis of Covid-19 On Cell and Gene Therapy Market Key Players, Share, Trend, Segmentation and Forecast to 2027 | Amgen, Biogen, BioMarin...

June 24 (Reuters) - Uniqure NV:

* UNIQURE ANNOUNCES LICENSE AGREEMENT WITH CSL BEHRING TO COMMERCIALIZE HEMOPHILIA B GENE THERAPY

* UNIQURE NV - UNIQURE ELIGIBLE TO RECEIVE MORE THAN $2 BILLION, INCLUDING $450 MILLION IN UPFRONT CASH

* UNIQURE NV - CO ELIGIBLE TO RECEIVE $1.6 BILLION IN MILESTONE PAYMENTS, AND DOUBLE-DIGIT ROYALTIES RANGING UP TO A LOW-TWENTIES PERCENTAGE OF NET SALES

* UNIQURE NV - LICENSING AGREEMENT PROVIDES CSL BEHRING EXCLUSIVE GLOBAL RIGHTS TO ETRANACOGENE DEZAPARVOVEC

* UNIQURE NV - CLINICAL DEVELOPMENT AND REGULATORY ACTIVITIES PERFORMED BY UNIQURE UNDER AGREEMENT WILL BE REIMBURSED BY CSL BEHRING

* UNIQURE NV - CSL BEHRING WILL BE RESPONSIBLE FOR REGULATORY SUBMISSIONS & COMMERCIALIZATION OF ETRANACOGENE DEZAPARVOVEC

* UNIQURE NV - CO PLANS TO DE-PRIORITIZE ITS RESEARCH PROGRAM OF AMT-180 FOR PATIENTS WITH HEMOPHILIA A

* UNIQURE NV - EXPECT TRANSACTION TO PROVIDE CO WITH SIGNIFICANT FINANCIAL RESOURCES TO ADVANCE & EXPAND PIPELINE OF GENE THERAPY CANDIDATES Source text for Eikon: Further company coverage:

See original here:
BRIEF-Uniqure Announces License Agreement With CSL Behring To Commercialize Hemophilia B Gene Therapy - Reuters

- Alliance to explore the utility of engineered exosomes developed with Codiaks engEx Platform to deliver gene therapy, gene editing and RNA technologies -

- Two-year, global research and option agreement covers up to five neuromuscular targets -

- Codiak is eligible to receive up to$72.5 millionin upfront and near-term license payments plus research funding -

CAMBRIDGE, Mass., June 22, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc.(NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, and Codiak BioSciences, Inc., a company at the forefront of advancing engineered exosomes as a new class of biologic medicines, today announced a global research and option agreement to design and develop engineered exosome therapeutics to deliver gene therapy, gene editing and RNA technologies for neuromuscular diseases. The engineered exosome approach offers the potential to effectively deliver genetic therapeutics without triggering the adaptive immune response.

The two-year agreement includes up to five neuromuscular targets. Codiak is eligible to receive up to$72.5 millionin upfront and near-term license payments plus research funding. Sarepta is granted an option to any of the candidates developed pursuant to the research alliance.

Exosomes are natural nanoparticles that serve as the bodys intercellular communication system, facilitating the transfer of a wide variety molecular payloads between cells. As they are derived from human cells, exosomes provide a unique advantage as a targeted delivery system for genetic medicines because they are inherently non-immunogenic. Through its proprietary, engEx Platform, Codiak can engineer exosomes with specific cargos and guide tropism to cell types of interest. The collaboration will leverage Codiaks exosome engineering capabilities with Sareptas expertise in precision genetic medicine to develop next generation therapeutics for patients with neuromuscular diseases that have few or no treatment options.

As Sarepta expands its leadership position in precision genetic medicine, this alliance with Codiak furthers our goal to deliver the most advanced therapies to patients. Codiaks engEx technology could potentially address some of the limitations of current treatment approaches and offers broad utility across Sareptas therapeutic modalities -- gene therapies, gene editing and RNA. Codiaks exosomes are engineered for precise tissue targeting and offer a non-viral delivery approach with non-immunogenic potential, thus opening up avenues for more efficient delivery and potential re-dosing, said Doug Ingram, president and chief executive officer, Sarepta Therapeutics.

The development of targeted delivery systems that enable repeat-dosing to select cell types has been a challenge for the emerging field of genetic medicine, especially in diseases of the muscle, said Douglas E. Williams, President and CEO of Codiak BioSciences. Engineered exosomes may offer a solution through their potential to selectively target muscle cells. We are excited to work with the world-class team at Sarepta to further build the engEx Platform and evaluate the potential of exosomes as next generation constructs incorporating gene therapy, gene editing and other nucleic acid payload modalities.

Under the terms of the agreement, Sarepta has the exclusive option to license Codiaks technology to develop and commercialize engineered exosome therapeutics for up to five neuromuscular targets. Sarepta and Codiak will collaborate to design exosomes that can deliver and functionally release select payloads, such as nucleic acids and gene therapy and gene editing constructs, in neuromuscular indications. If Sarepta elects to exercise its option on a target, Codiak will be responsible for research and preclinical development through IND preparation, and Sarepta will be responsible for clinical development and commercial activities. In addition to upfront, research funding and license payments, Codiak will be eligible to receive significant development and regulatory milestone payments and tiered royalties on future sales.

About engEx PlatformThe engEx Platform is Codiaks proprietary exosome therapeutic engine for engineering and manufacturing novel exosome product candidates designed to target multiple pathways throughout the body. Using this platform, Codiak can design exosomes with precisely engineered properties, incorporate various types of biologically active molecules and direct them to specific cell types and tissues. These exosomes engage targets by cellular uptake, membrane-to-membrane interaction or a combination of both mechanisms and are designed to change the biological functioning of the recipient cells in order to produce the intended biological effect. Codiak is building a broad pipeline of engEx product candidates that may have a transformative impact on the treatment of many diseases.

About Codiak BioSciencesCodiak BioSciences is harnessing exosomesnatural intercellular messengersto develop a new class of biologic medicines, exosome therapeutics. Utilizing our proprietary and versatile exosome engineering and manufacturing platform (engEx Platform), Codiak is developing exosomes with precisely engineered properties to engage pathways and deliver potent therapeutics to specific cell targets. We are building a broad pipeline of therapeutic candidates that may have a transformative impact on the treatment of many diseases, including in the areas of oncology, immune-based diseases, fibrotic disorders, neurological disorders and rare diseases. For more information, visithttp://www.codiakbio.com.

About Sarepta Therapeutics At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Sarepta Forward Looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the parties obligations and responsibilities under the agreement, potential payments and fees, potential neuromuscular targets and Sareptas option to any of the candidates developed pursuant to the research alliance; the potential benefits of the engineered exosome approach, including effectively delivering genetic therapeutics without triggering the adaptive immune response and selectively targeting muscle cells; the potential of exosomes to provide a unique advantage as a targeted delivery system for genetic medicines; and the potential benefits of the collaboration between Sarepta and Codiak, including the development of next generation therapeutics for patients with neuromuscular diseases that have few or no treatment options.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: the expected benefits and opportunities related to the collaboration between Sarepta and Codiak BioSciences may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development. In particular, the collaboration may not result in any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement; success in preclinical trials does not ensure that later clinical trials will be successful; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates and the COVID-19 pandemic; and even if Sareptas programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect Sareptas business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Contacts:

Sarepta TherapeuticsInvestors: Ian Estepan, 617-274-4052, iestepan@sarepta.comMedia: Tracy Sorrentino, 617-301-8566, tsorrentino@sarepta.com

Codiak BioSciencesKate Niazi-Sai, 617-949-5696, media@codiakbio.com

Link:
Sarepta Therapeutics and Codiak BioSciences Collaborate to Research and Develop Exosome-Based Therapeutics for Rare Diseases - GlobeNewswire

Andre Watson

SAN FRANCISCO (PRWEB) June 18, 2020

Vaccines currently in development face the dual challenge of overcoming the temporary immunity presented by coronavirus infection, and the SARS-CoV-2 viruss highly effective cloaking mechanism that effectively renders it invisible to the immune system.

Ligandals technology has been designed around the unique genetic signature of the virus, which has informed the development of a peptide nanoscaffold. This peptide prevents the virus from binding with human cells, halting infection. The peptide also simultaneously disables the viral cloaking mechanism, making the virus vulnerable to an immune response.

Andre Watson, CEO and Founder of Ligandal, says, I started Ligandal to create practical genetic medicine technology that solves the worlds most pressing health problems. We had been developing a way of training T-cells to attack cancer tumours, but when the COVID-19 pandemic began, I realised our technology would be effective at neutralising this single, virulent pathogen without requiring a gene therapy component. The beauty of our solution is that it can be used as treatment and vaccine. In infected people, the peptide will prevent viral entry and replication, while bolstering the immune response and formation of neutralising antibodies that can eliminate the virus. Other approaches may neutralise the virus, but many alternatives such as antibody therapies and viral-neutralising compounds will leave the body vulnerable to repeat infection. In those who havent been infected, the peptide will display critical immunoepitopes for antibody and T cell responses against the key parts of the virus necessary for forming a neutralising response. The peptide can also be used in conjunction with other treatments and vaccinesand may bolster the efficacy of spike protein vaccines in particularalthough that shouldnt be necessary if we achieve in vivo results suggested by the in silico modelling.

Adam Hamdy, a medical consultant and author, who recently joined the companys advisory board, says, The microbiological characteristics of SARS-CoV-2 make it extremely unlikely that current vaccine approaches, which rely on training the immune system but present key challenges with this virus, will offer anything more than partial protection at best. It was clear to me that any effective response to this virus had to target it directly while also bolstering immune response.

Importantly, Ligandals peptide has advantages over other technologies in development because it is room temperature stable, meaning it represents a genuinely global solution, both in terms of logistics and the storage of the medicine. It also has a low cost per dose once at mass production scale, which means global producton is entirely feasible. It can also move rapidly through preclinical and clinical studies given the accelerated global regulatory environment and simple at scale manufacturing process.

Andre Watson says, As hostile actors take note of the havoc caused by the SARS-CoV-2 virus, it is clear we need better ways to ramp up our biodefense capabilities. A rapid four-year conventional vaccine cycle to trial a single solution isnt going to be sufficient to meet future threats. Ligandal modelled its peptide within five hours of receiving a genetic sequence of the virus, and was ready to test its response in two months. We anticipate being ready to commence clinical studies later this year or very early next year. Our only limitation in getting this to the general public is how quickly we can move through the clinical studies.

Adam Hamdy says, A long term solution to the COVID-19 problem lies in genetic medicine. Ligandals peptide is an exciting leap forward in our ability to address and neutralise the virus directly. Unlike conventional vaccines, which only get one shot at proving efficacy, Ligandals peptide can be improved by rewriting its genetic code in response to novel viral variants, giving us the opportunity to rapidly iterate and keep at the problem until we get the answer absolutely right.

About Ligandal

Based in San Francisco, Ligandal is a genetic medicine company that uses nanotechnology to develop targeted and personalised therapies.

For more information visit http://www.ligandal.com

Share article on social media or email:

Go here to read the rest:
Genetic Medicine will be the Solution to COVID-19, and Ligandal is Leading the way - PR Web

PALO ALTO, Calif., June 24, 2020 (GLOBE NEWSWIRE) -- BridgeBio LLC Pharma, Inc. (NASDAQ: BBIO), a clinical-stage biopharmaceutical company focused on genetic diseases, today announced that it has added three new independent directors to its board: former Allergan CEO and biopharma deal-maker, Brent Saunders; genomics pioneer and entrepreneur, Randy Scott, Ph.D.; and renowned economist and BridgeBio co-founder, Andrew Lo, Ph.D.

We are privileged to welcome these world-class company builders, innovators, and thought leaders to our board where they can help us construct and advance a vast pipeline of meaningful medicines for patients with genetic disease, said BridgeBio CEO and founder Neil Kumar, Ph.D. Brent has a remarkable track record of leading companies to growth across many therapeutic areas. Randy is a giant in genomic medicine who has used his entrepreneurial skills to connect genetic information to patients in profound ways. Andrews groundbreaking financial engineering work led to the founding of BridgeBio and inspires us to find new ways to accelerate the drug development process. I look forward to working with and learning from these leaders as we seek to discover, develop, and deliver life-changing medicines at scale. Its day one at BridgeBio and we are ready to go.

Brent Saunders

Mr. Saunders will bring his considerable experience to bear as he advises BridgeBio on scaling its business across new product and therapeutic areas, expanding into new geographies, developing commercial expertise, and utilizing new corporate structures.

Mr. Saunders most recently served as chairman, president and chief executive officer of Allergan. In this capacity, he led the company to launch more than 15 products and achieve 9.4% revenue growth until its merger with AbbVie in 2020. He previously served as president and CEO at Actavis, where he led a $15 billion global pharmaceutical business until its merger with Allergan. He initially joined Actavis as part of the companys acquisition of Forest Laboratories, where he served as president and CEO. Before joining Forest Laboratories, Mr. Saunders served as president and CEO of Bausch & Lomb. Mr. Saunders received his bachelors degree in economics and East Asian studies from the University of Pittsburgh, a Juris Doctor degree from Temple University School of Law and his Master of Business Administration from Temple University School of Business.

I appreciate BridgeBios ability to accelerate the development of therapeutics for patients in need and I have been impressed with their unheard-of progress in pipeline growth in such a short time. As the company moves toward potential commercialization of its lead products, Im eager to bring my experience leading and growing global pharmaceutical companies to help guide BridgeBio in its game-changing efforts, said Mr. Saunders.

Randy Scott, Ph.D.

Dr. Scott will bring his deep expertise to BridgeBio and advise the company on how best to utilize the broad ecosystem of genomic medicine (beyond the pill) so that a wider universe of patients can be served. BridgeBios decentralized business model will also benefit from Dr. Scotts guidance on developing and maintaining a strong culture of excellence.

Dr. Scott pioneered the introduction of genetics into everyday medical care as the founder of multiple cutting-edge biotech companies. Dr. Scott recently served as chief executive officer and executive chairman at Invitae, a company he co-founded to bring genetic information into routine medical practice. Prior to Invitae, he founded Genomic Health and served as the companys CEO and later executive chairman leading the company to develop and launch genomic diagnostic tests for breast, colon, and prostate cancer. Earlier in his career, Dr. Scott served as the president and chief scientific officer for Incyte, one of the first geneticinformationcompanies. He co-founded and currently serves as chairman of Genome Medical, a telegenomics-based clinical care company. Dr. Scott earned his bachelors degree in chemistry from Emporia State University and his doctorate in biochemistry from the University of Kansas.

For the last 30 years, Ive focused on building genomics companies to better understand the fundamental basis of human disease and improve the quality of treatment decisions through genomic diagnosis.It is with great excitement that I now join the BridgeBioboard to take the next step inbringing multipletherapies for genetic disorders to market, said Dr. Scott.Genetic disorders are much more common than previously thought, and BridgeBio has the unique opportunity toefficiently bring multiple life-saving products to the clinic at an accelerating pace.With my experience in building and scaling companies, I plan to focus on helping the BridgeBio team to scale the organization and build a world-class pharmaceutical company focused on solving genetic disease."

Andrew Lo, Ph.D.

Dr. Lo brings his vast knowledge and understanding of economics and financial engineering to BridgeBio and will advise the company on how to continue accessing financing from a range of markets to broaden its work for patients and pursue more groundbreaking scientific innovation. As a famously innovative and iconoclastic thinker, he will also challenge the company to continue to innovate in areas as diverse as R&D process, clinical trial statistics, and talent management.

Dr. Lo is the Charles E. and Susan T. Harris Professor at the MIT Sloan School of Management, director of the MIT Laboratory for Financial Engineering, a principal investigator at the MIT Computer Science and Artificial Intelligence Laboratory, and an affiliated faculty member of the MIT Department of Electrical Engineering and Computer Science. His research spans several areas of financial economics, but his most recent focus is on developing new statistical tools for predicting clinical trial outcomes, incorporating patient preferences into the drug approval process and accelerating biomedical innovation through novel financing structures. His work formed the foundation for BridgeBios business model, and he is one of the companys co-founders. Dr. Lo earned his bachelors degree in economics from Yale University and a masters degree and doctorate in economics from Harvard University.

Its remarkable to see the significant progress that BridgeBio has made in the five years since its founding to develop new medicines for genetically driven diseases, said Dr. Lo. "I'm excited and honored to join the BridgeBio board and look forward to contributing in whatever ways I can to help them bring new therapies to patients who have no other alternatives."

About BridgeBio PharmaBridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio's pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, please visitbridgebio.com.

Contact:Grace RauhBridgeBio Pharma, Inc.Grace.rauh@bridgebio.com(917) 232-5478

Source: BridgeBio Pharma, Inc.

See the rest here:
BridgeBio Pharma, Inc. Appoints Biotech Trailblazers Brent Saunders and Randy Scott and Renowned Economist Andrew Lo to Board of Directors - BioSpace

Application of ImmTOR plus Sareptas investigational gene therapies will be evaluated for Duchenne Muscular Dystrophy and Limb-Girdle Muscular Dystrophies

CAMBRIDGE, Mass. and WATERTOWN, Mass., June 18, 2020 (GLOBE NEWSWIRE) --Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, and Selecta Biosciences, Inc. (NASDAQ: SELB) today announced that they have entered into a Research License and Option agreement granting Sarepta an option to license the rights to develop and commercialize Selectas immune tolerance platform, ImmTOR, for use in Duchenne muscular dystrophy (DMD) and certain limb-girdle muscular dystrophies (LGMDs). In advance of exercising its option, Sarepta will conduct research and evaluate the utility of ImmTOR to minimize or prevent the formation of neutralizing antibodies (NAb) to adeno-associated virus (AAV) in connection with the administration of Sareptas DMD and LGMD gene therapy candidates.

Sareptas late-stage gene therapy candidates are delivered using AAV in particular, AAVrh74. AAVrh74 was selected because of its safety profile, superior muscle tropism, empirical demonstration of high expression, and low screen-out rate for pre-existing antibodies. Currently, however, all systemic AAV-delivered constructs are one-time therapies that cannot be re-dosed due to the robust post-administration development of NAbs specific to the AAV vector. Selecta is a leader in immune tolerance and has generated strong preclinical evidence to support the potential for re-dosing patients receiving gene therapy.Selecta has reported that in preclinical studies, when used in combination with AAV gene therapy vectors,Selectas ImmTOR immune tolerance platform inhibits the development of NAbs to the vector, permitting re-dosing of the gene therapy. i

As we build our enduring gene therapy engine, we intend not only to rapidly advance treatments for rare, life-ending diseases, but at the same time, to advance the state of genetic medicine science by continually improving the utility of gene therapy. If successful, the ability to re-dose will be an enormous leap forward in the science of gene therapy and provide invaluable benefits to patients beyond those we anticipate with one-time dosing. We are encouraged by the data generated on the ImmTOR platform and excited to join with Selecta to explore the possibility of unlocking the opportunity to safely and effectively re-dose AAV-mediated gene therapies in patients with DMD and LGMDs, if needed, said Doug Ingram, President and Chief Executive Officer, Sarepta Therapeutics.

We are pleased to build on our already strong foundation of strategic partnerships and expand the clinical application of the ImmTOR platform into neuromuscular diseases. The ability to re-dose gene therapy addresses one of the major challenges of one-time therapies today, said Carsten Brunn Ph.D., President and Chief Executive Officer of Selecta Biosciences. We are excited to collaborate with a leader in genetic medicine like Sarepta, and are confident that their expertise in rare diseases combined with our immune tolerance platform has the potential to enhance the long-term therapeutic benefit to patients with these debilitating conditions.

DMD is a rare, degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. LGMDs are a group of over 30 distinct diseases that cause weakness and wasting of the muscles around the hips and shoulders, eventually progressing to the arms and legs. LGMD can be caused by a single gene defect that affects specific proteins within the muscle cell, including those responsible for keeping the muscle membrane intact.

Under the terms of the research license and option agreement, Sarepta will make an initial payment to Selecta, and Selecta is eligible to receive certain pre-clinical milestone fees. If Sarepta exercises its options to enter any commercial license agreements, Selecta will be eligible for additional development, regulatory, and commercial milestone payments, as well as tiered royalties on net product sales. Additional financial details are not being disclosed.

About Selecta Biosciences, Inc.

Selecta Biosciences, Inc. is a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its pioneering immune tolerance platform (ImmTOR). Selecta is committed to utilizing ImmTOR to potentially improve the efficacy of biologics, enable re-dosing of life-saving gene therapy, and create novel immunotherapies for autoimmune diseases. Selectas late-stage product candidate, SEL-212, is designed to be a monthly treatment for chronic refractory gout, a debilitating rare disease with a significant unmet medical need. SEL-212 consists of a combination of our ImmTOR platform co-administered with pegadricase, an enzyme designed to treat patients with symptomatic gout, refractory to standard uric acid lowering treatment. Selectas proprietary gene therapy product candidates are in development for certain rare inborn errors of metabolism and incorporate our ImmTOR platform with the goal of addressing barriers to repeat administration. In addition to our own pipeline of core discovery and clinical candidates, Selecta has established collaborative relationships with leading biopharmaceutical companies, including Asklepios BioPharmaceutical (AskBio) for gene therapy, and Swedish Orphan Biovitrum AB (Sobi) for SEL-212. Selecta is based in Watertown, Massachusetts. For more information, please visit http://www.selectabio.com.

Selecta Forward-Looking Statements:

SelectaBiosciences, Inc. (the company), including without limitation, the companys actions regarding the monitoring and assessment of COVID-19 on the companys operations, clinical trials and manufacturing, Sareptas plans to evaluate its gene therapies in combination with the companys ImmTOR technology, the possibility of Sarepta exercising an option to enter into a commercial license agreement, the unique proprietary technology platform of the company and the unique proprietary platform of its partners, the potential of ImmTOR to enable re-dosing of AAV gene therapy, the ability of the companys ImmTOR platform to unlock the full potential of biologic therapies, the potential treatment applications for product candidates utilizing the ImmTOR platform in areas such as enzyme therapy and gene therapy, the novelty of treatment paradigms that Sarepta is able to develop in combination with the companys ImmTOR technology, the potential of any therapies developed by Sarepta in combination with the companys ImmTOR technology to fulfill unmet medical needs, the companys plan to apply its ImmTOR technology platform to a range of biologics for rare and serious diseases, the ability of Sareptas existing therapies to target the heart and skeletal muscle, expected payments to be made to the company under the Research License and Option Agreement, the potential of the ImmTOR technology platform generally and the companys ability to grow its strategic partnerships, the sufficiency of the companys cash, cash equivalents and short-term investments, and other statements containing the words anticipate, believe, continue, could, estimate, expect, hypothesize, intend, may, plan, potential, predict, project, should, target, would, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including their uncertain outcomes, the effect of the COVID-19 outbreak on any of the companys planned or ongoing clinical trials, manufacturing activities, supply chain and operations, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, the unproven approach of the companys ImmTOR technology, Sareptas ability to research and develop therapeutic candidates using the companys ImmTOR technology, undesirable side effects of the companys product candidates, its reliance on third parties to manufacture its product candidates and to conduct its clinical trials as well as the impact of the COVID-19 outbreak on those third parties and their ability to continue their operations, the companys inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, managements ability to perform as expected, potential delays in regulatory approvals, Sareptas ability to make up-front and milestone payments, the companys business development strategy, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the companys recurring losses from operations and negative cash flows from operations raise substantial doubt regarding its ability to continue as a going concern, substantial fluctuation in the price of its common stock including stock market fluctuations that occur as a result of the COVID-19 outbreak, and other important factors discussed in the Risk Factors section of the companys most recent Quarterly Report on Form 10-Q, and in other filings that the company makes with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the companys views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any intention to update any forward-looking statements included in this press release.

AboutSarepta Therapeutics

At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.comor follow us onTwitter,LinkedIn,InstagramandFacebook.

Sarepta Forward-Looking Statement

This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the parties undertakings under the agreement and potential payments and fees; the potential benefits of Sareptas gene therapy product candidates; the potential of ImmTOR to enable re-dosing of AAV gene therapy; Sareptas intention to rapidly advance treatments for rare, life-ending diseases, and to advance the state of the genetic medicine science by continually improving the utility of gene therapy; the potential of re-dosing to provide invaluable benefits to patients beyond those Sarepta anticipates with one-time dosing;the possibility of unlocking the opportunity to safely and effectively re-dose AAV-mediated gene therapies in patients with DMD and LGMDs, if needed; and the potential of the collaboration between Sarepta and Selecta to enhance the long-term therapeutic benefit to patients with these debilitating conditions.

These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: the expected benefits and opportunities related to the collaboration between Sarepta and Selecta may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development. In particular, the collaboration may not result in any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement; success in preclinical trials does not ensure that later clinical trials will be successful; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates and the COVID-19 pandemic; and even if Sareptas programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect Sareptas business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Selecta Contacts:For Media:Joshua R. MansbachSolebury Trout+1-646-378-2964jmansbach@soleburytrout.com

For Investors:Lee M. SternSolebury Trout+1-646-378-2922lstern@soleburytrout.com

Sarepta Contacts: Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

_____________________________iNature Communications,October 2018.

More:
Sarepta Therapeutics and Selecta Biosciences Enter into Research License and Option Agreement for Selecta's ImmTOR Immune Tolerance Platform in...

Image: Prof. Dr. David Ellinghaus and Frauke Degenhardt, both first authors of the study (Photo courtesy of UKSH Kiel)

The world's first large-scale genome-wide study conducted by scientists at the University Medical Center Schleswig-Holstein (UKSH Kiel, Germany) and the Kiel University (CAU Kiel, Germany), in cooperation with a research group from Norway, has found gene variants that significantly influence the course of the disease-one of them concerns the gene for the blood group trait. This suggests that different blood groups may be responsible for why some people become severely ill with COVID-19 while others show hardly any symptoms.

Doctors from several hospitals of the corona epicenters in Northern Italy and Spain, sent blood samples of a total of 1,980 intensive care COVID-19 patients who had to be treated with oxygen or connected to a ventilator. For the control group, 2,205 randomly selected women and men from the population of these countries were obtained. Within only three weeks, DNA was isolated from the blood samples and 8.5 million positions of the genetic material from each individual were measured with so-called biochips (SNP arrays). The study showed that people with blood group A had an approximately 50% higher risk of severe COVID-19 progression than people with other blood groups. In contrast, people with type 0 blood groups were almost 50% better protected against serious COVID-19 disease. Thus, the study confirmed for the first time by means of a comprehensive genome-wide analysis two earlier studies by international researchers who had already described a possible correlation between blood group characteristics and the disease using the blood serum of COVID-19 patients.

In addition to the significant abnormality in the AB0 blood group locus, the gene locus by which the individual blood group is determined, the researchers found an even higher effect strength for a genetic variation on chromosome 3. Which of the several candidate genes located in this locus is responsible for this cannot be determined precisely at present, but the analysis was able to show that carriers of the gene are at a twofold higher risk of contracting severe COVID-19 than people who do not carry this variation. Among the Italian and Spanish patients who were so ill that they not only had to be supplied with oxygen but also connected to a ventilator, a particularly high number carried this genetic disposition. A result that was also evident in the distribution of blood groups: Among the particularly seriously ill, there were also a particularly large number of people with blood group A.

The results were very exciting and surprising for us. The region on chromosome 3 in particular had not previously been associated with COVID-19 by scientists. In other regions of the genome for which an effect on the disease had been suspected, no statistically significant differences were found between the healthy volunteers and the patients; neither in the chromosome section 6p21, which is associated with the immune system and many infectious diseases, nor in the gene IFITM3, which is associated with influenza, said Prof. Dr. Andre Franke, Director of the Institute of Clinical Molecular Biology (IKMB) and member of the steering committee of the cluster of excellence "Precision Medicine in Chronic Inflammation" (PMI). With chromosome 3 and the AB0 blood group locus we describe real causes for a severe course of COVID-19. Our results, therefore, create an excellent basis for the development of active substances that can target the candidate genes found. It has been proven that a clinical study in which a drug is tested has twice as much success if genetic evidence for the target is already available. The results could also contribute to an improved risk assessment for a severe course of COVID-19 in patients.

Related Links:University Medical Center Schleswig-Holstein (UKSH)UKSH Kiel

View post:
Genes and Blood Type Determine Risk of COVID-19 Severity - HospiMedica

The Global Precision Medicine market report presents market dynamics focusing on all the important factors market movements depend on. It includes current market trends with a record from historic year and prediction of the forecast period. This report is a comprehensive market analysis of the Precision Medicine market done on a basis of regional and global level. Important market analysis aspects covered in this report are market trends, revenue growth patterns market shares and demand and supply along with business distribution.

Get Research Insights @Precision Medicine Market 2019-2025 (USD Bn)

Precision medicines are about bringing the right treatment to the right patient at the right time. It is one of the key vehicles by which the healthcare system of tomorrow can achieve better outcomes for patients and financial efficiency.

Global Precision Medicine market size is estimated based on the population genetic screening data, disease prevalence rate and adoption of the precision medicines across the globe. Global Precision Medicine manufacturers have varying products, depending on consumer genetic screening data. The report also provides various key strategies adopted by the leading players as they mainly target prevalent diseases in the developed and developing economies. Precision Medicine market share in various diseases has also been derived on the basis of thorough understanding of the forecast and dynamics of various products under pipeline in various geographies. Also, the global Precision Medicine market size has been laid down based on an extensive value chain analysis and patent analysis.

Browse Complete Research Insights @https://www.adroitmarketresearch.com/industry-reports/precision-medicine-market

The global precision medicine market size is projected to be valued USD 119.90 billion by 2025. Advancement in genomic and proteomic sequencing with reduced costs, is bringing in the so-called genomic revolution. By identifying the optimal point of intervention for treatment, healthcare providers massively improve the quality of care. Additionally, these technologies offer optimized cost by effectively timing treatment intervention and eliminating the need for insufficient or excessive treatment regimens.

Next generation sequencing has shifted the paradigm of genomics by accomplishing entire genome sequencing in a matter of hours. The enhanced speed and ease of DNA sequencing has drastically reduced the cost. The cost of deciphering the entire human genome has dropped by an order of magnitude from USD 10,000 in 2011 to about USD 1,000 today. As genome sequencing costs are declining rapidly healthcare providers and patients are likely to be more inclined to seek treatments that are targeted to a particular illness.

Get Infographics @https://www.adroitmarketresearch.com/infographics/precision-medicine-industry

Cost is a major issue for precision medicines. Precision medicines may overburden many healthcare systems which are currently under significant financial constraints. It is a major factor hindering the patients access to treatment. The new targeted drugs that are launched in the market, are so highly priced that the patients can hardly afford them unless the cost is fully covered by the payers.

Geographically, Asia Pacific led the precision medicine market share in the global market in 2017, growing with a CAGR of 14.38% from 2018 to 2025. Globally, 40 countries have their individual versions of precision medicine ingenuities. United States and China being one of them. China, is spending $43 for every $1 the United States is spending on its precision medicine initiatives. The Beijing Genome Institute has the worlds largest sequencer and repository of genetic material. Additionally, developments in computational power and artificial intelligence are also in progress to discover new drugs, treatment and delivery methods. Wuxi Nextcode and Huawei are collaboratively developing cloud computing infrastructure to store and compute enormous amounts of data for precision medicines.

Furthermore, the Korean government is strategizing framework for implementation of precision medicine into clinical practice. The Korean Ministry of Food and Drug Safety, conducted a survey in 2012-2013, in order to explore the public awareness and attitude towards precision medicine. Results of the survey demonstrated low levels of public awareness regarding precision medicine, however, family income showed a positive correlation with precision medicine knowledge, thus reducing potential health disparities will increase the access to precision medicine.

Some of the leading players operating in the market include Novartis International AG, F. Hoffmann-La Roche AG, AstraZeneca plc, Eli Lilly and Company, Pfizer Inc., Teva Pharmaceutical Industries Ltd., Abbott Laboratories, Merck & Co. and others.

Make an Enquire to buy this report @https://www.adroitmarketresearch.com/contacts/enquiry-before-buying/831

Key segments of the global precision medicine market

Application Overview, 2015-2025 (USD Million)

Regional Overview, 2015-2025 (USD Million)

Grasp advance knowledge on Precision Medicine Industry through in-depth Blogs, White Paper & Infographics:

Access the White Paper Precision Medicine And The Future Of Health Technology

Read the in depth blog titled How Does Precision Medicine Benefit An Individuals Health?

What does the report include?

About Adroit Market Research:

Adroit Market Research provide quantified B2B research on numerous opportunistic markets, and offer customized research reports, consulting services, and syndicate research reports. We assist our clients to strategize business decisions and attain sustainable growth in their respective domain. Additionally, we support them with their revenue planning, marketing strategies, and assist them to make decisions before the competition so that they remain ahead of the curve.

Read the original here:
Precision Medicine Market 2020 | Increasing Demand, Current Trend, Scope, Business Strategies, Challenges, New Innovations, Future Projections and...

If you were to read many of the adverts for stem cell therapy that you can find online, you would be forgiven for believing that stem cell therapy is nothing short of a panacea. It is, according to those ads, able to improve all sorts of conditions, from knee pain and osteoarthritis, hair loss to heart disease, diabetes and even autism.

Theres just one problem theres little science behind many of the claims.

Stem cells are only approved for use in treating certain cancers and blood disorders, yet a search for the term on Facebook or Google will return details of a large number of clinics offering treatments for many other conditions.

The harsh reality is that while there is a lot of promising research being undertaken in this area, nobody should be parting with large sums of money for what may be currently no more than snake oil treatments, according to Noel Caplice, who is professor of cardiovascular sciences in the department of medicine at University College Cork and a consultant cardiologist.

Caplice, who has more than 20 years experience monitoring stem cell research as part of his studies into heart disease, told The Irish Times that we should all be suspicious about the range of different ailments clinics are willing to treat with stem cells.

There should be red lights flashing and alarm bells ringing. No therapy treats everything from Parkinsons disease to multiple sclerosis to heart disease to knee pain thats idiotic. Medicine just doesnt work like that.

True stem cell therapy is extremely complex because you have to refine the type of cell youre going to give to the organ it will be used in, and there are different challenges in different organs. Legitimate scientists are working on these things, but they are not there yet. Its an incredibly difficult area of research.

Stem cells have long been considered a great hope of medicine. They are the bodys building blocks, the cells from which other types of cells develop. Under the right conditions they can be encouraged to become any other type of cell found in the body, such as blood cells, brain cells, heart muscle cells and so on.

At its simplest, stem cell therapy involves cultivating stem cells in the lab, guiding them to grow into specific types of other cells, and then injecting those healthy cells into diseased parts of the body where in certain circumstances they have been shown to help the bodys own cells to fight disease.

This effect was first shown around 30 years ago in experiments on mice. However, things have not been all plain sailing since then.

The initial promise of stem cells has not been fulfilled, and whats happened in the meantime is that commercial clinics offering treatments have gotten ahead of the science, said Caplice.

The first trials in mice showed incredible regeneration, but their progress turned out not to be so straightforward. When the initial trials were replicated, the researchers couldnt reproduce the same early data.

While research is ongoing and there have been a few significant breakthroughs using stem cells, notably in the case of combined stem cell and gene therapy for thalassemia and leukaemia, that has not stopped unscrupulous clinics from marketing all sorts of treatments under the banner of stem cell therapy.

In the private world anything goes. There are people spinning this therapy for multiple sclerosis, Parkinsons, solid organ deterioration a whole range of problems. Ten years ago there was even a boat operating off the west Cork coast that was treating people for multiple sclerosis using stem cells. This has been going on for decades in this parallel world, and its mostly driven by money, Caplice said.

According to Frank Barry, professor of cellular therapy at the regenerative medicine institute with NUI Galway, a negative side effect of the off-label use of stem cells is that it makes it harder for researchers to raise money for research.

It damages our reputation to have people doing this. We all get painted with the same brush, and it makes it much harder to raise money. When these maverick clinics are exposed for their bad practices, there is a blow-back effect on us even though were completely unconnected, he said.

The sad thing is that there are genuinely quite exciting applications of stem cell therapy that will be possible in the future. All of these are undergoing scientifically-designed clinical trials that are carefully done, carefully managed, are placebo-controlled, double blind the works. Some of these trials are going quite well and suggest that the outcome will be good.

The biopharmaceutical company Takeda Ireland, for example, is currently developing a treatment for inflammatory bowel disease using the results of a trial that was conducted into stem cells.

Thats a dreadful condition that blights peoples lives. This is a new treatment so thats very exciting. That project achieved market authorisation because of careful work done over many years in high quality clinical trials, said Barry.

My own work is in the treatment of arthritis with stem cell therapy, and thats also going well. Were in the middle of a big trial thats been running in a number of clinical sites around Europe, and we think that when its finished itll be positive.

Running trials like these takes a lot of time and a lot of money. In the meantime bad actors are stepping into the gap that exists between promising early results and actual rigorous and robust science.

The harsh reality is that you cant recommend that a patient has stem cell therapy for anything that isnt directly authorised. If someone does that now theyre getting it off-label, so to speak, and basically theyre taking their chances, said Barry.

I can understand why someone might want to do that, but its not authorised. I would hold out a great deal of hope that when all the work is done there will be strong proof supporting this kind of treatment. But at the moment you can spend a huge amount of money essentially for nothing because there isnt the evidence to support treatment.

Original post:
Stem cell therapy: why we need to be suspicious about cure-all claims - The Irish Times