StemCell Therapy

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review.

If approved, Lenti-D (elivaldogene autotemcel or eli-cel) could transform the prospects of people with CALD, the most severe form of the neurodegenerative disease ALD that usually emerges in boys during early childhood and causes physical and mental disabilities as well as behavioural problems.

Around 40% of patients develop the cerebral form of ALD, which in turn affects around one in 17,000 live births.

A few weeks ago, Bluebird reported new data from the phase 2/3 STARBEAM trial of Lenti-D which showed that 87% of CALD patients were still alive and free of major functional disabilities after at least two years follow-up.

The EU filing comes ahead of a filing for eli-cel in the US, which Bluebird says should take place sometime towards the middle of next year, having been delayed by the coronavirus pandemic.

If approved, eli-cel would provide a one-shot treatment for CALD, holding back the progressive breakdown in the protective myelin that sheathes neurons.

It would be the first alternative to a stem cell transplant to treat the disease, a therapy that can provide significant improvements and even halt progression in some patients if given early enough.

However it requires high-dose chemotherapy to destroy the bone marrow, and that poses significant risks to patients in its own right, and can also lead to graft-versus-host disease, a potentially life-threatening complication in which the bone marrow donors immune cells attack the recipients cells and tissues.

CALD is caused by mutations in the ABCD1 gene located on the X chromosome, which provides instructions for the production of the ALD protein.

ALD protein is needed to clear toxic molecules called very long-chain fatty acids (VLCFAs) in the brain, and if mutated causes the VLCFAs to accumulate and damage the myelin sheath.

Using eli-cel, the patients own stem cells are modified in the lab to produce a working version of the ABCD1 gene, producing functional ALD protein that can help to flush VLCFAs from the body.

CALD is a devastating disease, often marked by rapid neurodegeneration, the development of major functional disabilities, and eventual death, said Gary Fortin, head of severe genetic disease programmes at Bluebird.

If approved, eli-cel would represent the first therapy for CALD that uses a patients own haematopoietic stem cells, potentially mitigating the risk of life-threatening immune complications associated with transplant using cells from a donor, he added.

Aside from STARBEAM, which will follow treated patients for up to 15 years, Bluebird is also conducting the phase 3 ALD-104 trial of eli-cel in CALD, which is due to generate results in 2024.

The EU filing for eli-cel comes shortly after Bluebirds development partner received a 27 March 2021 FDA review date for anti-BCMA CAR-T cell therapy ide-cel, a potential therapy for multiple myeloma.

The biotech already has approval in Europe for Zynteglo, a gene therapy for haematological disease beta thalassaemia, and is due to file its related therapy LentiGlobin for sickle cell disease next year. The two therapies have been tipped to generate $1.5 billion-plus in peak sales by some analysts.

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EMA starts rapid review of Bluebird's gene therapy for rare disease CALD - - pharmaphorum

The FDA has lifted the clinical hold on a phase 1/2 clinical trial of Solid Biosciences gene therapy treatment for Duchenne muscular dystrophy (DMD). Solid Bio secured clearance to resume dosing in the trial after making manufacturing changes to cut the number of viral particles given to patients.

SGT-001, the adeno-associated viral (AAV) vector-mediated gene transfer therapy being tested in the phase 1/2 trial, has suffered a series of setbacks since entering the clinic, most recently when the FDA put the study on hold in response to a case of acute kidney injury. The FDA imposed the hold 11 months ago. In July, Solid Bio said the FDA wanted to see more data before lifting the hold.

The request led Solid Bio to share further information on its gene therapy manufacturing process and its latest safety and efficacy data. The additional information proved sufficient to persuade the FDA to lift the clinical hold.

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Solid Bio will resume dosing using a gene therapy made under a revised manufacturing process. The new process is intended to remove most empty viral capsids, thereby enabling Solid Bio to cut total viral load without reducing the dose. The focus on viral load reflects concerns systemic delivery of AAV vectors can damage organs and cause inflammation.

To get the FDA to lift the hold, Solid Bio shared data from a quantitative, in vitro microdystrophin expression assay designed to show the comparability of SGT-001 manufactured under the old and new processes. Solid Bio shared those results in response to the FDAs request for information in July.

The biotech is taking other precautions to manage the potential risk posed by SGT-001. Solid Bio has capped the maximum weight of the first two patients to receive SGT-001 after the hold lifts at 18 kg. As the dose of SGT-001 is determined by weight, heavier patients receive more vector genomes. The adverse events seen in some gene therapy trials, such as the deaths in Audentes Therapeutics trial, have happened in patients who were heavier and therefore received a higher viral load.

Solid Bio is further mitigating the potential for SGT-001 to cause harm by amending the protocol to include the prophylactic use of eculizumab, the anti-complement inhibitor sold by Alexion as Soliris, and C1 esterase inhibitor, while also increasing the prednisone dose in the month after treatment.

The protocol changes position Solid Bio to resume its pursuit of DMD gene therapy leader Sarepta Therapeutics, which suffered a setback of its own last month when the FDA asked it to use an extra potency assay in a planned clinical trial. Pfizer is also in the race but, like Solid Bio, has run into safety issues that could give Sarepta an edge.

Shares in Solid Bio, which had slumped to $2 apiece, rose 70% in response to the end of the hold.

Link:
FDA lifts clinical hold on Solid Bio gene therapy trial - FierceBiotech

ALDERSON, W.Va. (WVVA) A Greenbrier County teen is about to become one of the first five patients in history to receive an experimental gene therapy for Hurler's Syndrome.

The Alderson native is in many ways your typical 13-year-old. "I like to help my dad feed cows....and donkeys," said Kendra Goins.

But life hasn't always been easy for Kendra. The extremely rare condition makes it impossible for her body to breakdown certain sugars. In addition to causing damage to her organs, the condition makes it difficult for her body to grow.

But whenever anyone has anything to say about it, her sister, Kiristen is always the first time stand up.

"Me with my big head is always the one to jump in," said Goins, who said she has gotten into quite a few quarrels over the issue at school in defense of her sister.

She worries though that she won't be able to jump in when Kendra heads to California soon for a clinical trial. Kendra is heading into the treatment with her first bone marrow transplant nearing the end of its course.

"I've spoken with experts across the country from the chemists who made the drug to the doctor who has used it. This looks like her golden ticket," said Kendra's mother Sheryl Goins.

Administered through the brain, the goal of the gene therapy is to help her body produce the enzymes she needs to survive.

While the cost of the clinical trial and airfare is covered, the family said they need help with expenses they will incur during their three-month stay.

To learn more about how you can help, visit https://www.gofundme.com/f/kendra039s-gene-therapy?utm_source=facebook&utm_medium=social&utm_campaign=p_cp%20share-sheet&fbclid=IwAR3yzHCexch5a_awjaYy06ijB28zMXJ-72WkfQ-SPEwMQgy5s8x_carlz34

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Greenbrier County teen to be among first in nation to receive gene therapy for Hurler's Syndrome - WVVA TV

Oct. 5, 2020 08:09 UTC

HARLOW, England--(BUSINESS WIRE)-- Diamond Pharma Services (Diamond), a leading technical services and regulatory affairs consulting group, has announced that it provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight Biologics (GenSight), including the preparation, authoring support, agency communication and submission of GenSights first Marketing Authorisation Application to the European Medicines Agency (EMA), for its novel ocular gene therapy LUMEVOQ. The EMA decision is expected in H2 2021.

LUMEVOQ (Lenadogene nolparvovec) is a gene therapy to treat vision loss due to the rare, mitochondrial genetic disease, Leber Hereditary Optic Neuropathy (LHON) caused by mutation in the ND4 mitochondrial gene. LHON mainly affects young males, and the ND4 mutation results in the worst visual outcomes, with most patients becoming legally blind. There is a high unmet medical need for LHON patients, of which there are 800-1200 in the EU and the US annually.

Headquartered in Paris, France, GenSight is a biopharma company focused on developing and commercialising innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. Diamond has provided GenSight with regulatory, pharmacovigilance, quality and compliance support for LUMEVOQ leading up to the MAA assessment.

Maureen Graham, Managing Director, Regulatory, Diamond Pharma Services, said: We are pleased GenSight Biologics chose to work with our team of experts at Diamond to provide European Regulatory, Pharmacovigilance, Quality and Compliance support for LUMEVOQ, its first MAA submission, and the first for a gene therapy treating a mitochondrial disease. It has been a personal ambition of mine to have the opportunity to work on a gene therapy within the ophthalmic arena, and GenSight has allowed Diamond that opportunity and that privilege.

This submission adds to Diamonds broad experience in providing support to companies developing advanced therapy medicinal products (ATMPs), which includes over 50 programmes at various stages of development, and two MAA approvals - Glybera and Yescarta.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201005005339/en/

Original post:
Diamond Pharma Services Supports GenSight Biologics in Submitting Its First Marketing Authorisation Application, for Ocular Gene Therapy LUMEVOQ -...

The Food and Drug Administration (FDA) has granted Fast Track designation to the investigational gene therapy candidate, PF-06939926 (Pfizer), for the treatment of Duchenne muscular dystrophy (DMD).

PF-06939926 is a recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of human dystrophin gene under the control of a human muscle-specific promoter. The Company has chosen the rAAV9 capsid due to its potential to target muscle tissue.

The designation was based on data from an ongoing phase 1b study evaluating the safety and tolerability of a single intravenous infusion of PF-06939926 in 9 ambulatory boys with DMD aged 6 to 12 years. Preliminary results showed that PF-06939926 was well tolerated during the infusion period and dystrophin expression levels were sustained over a 12-month period.

The Company plans to launch a double-blind, placebo-controlled phase 3 study to evaluate the efficacy and safety of PF-06939926 in boys with DMD. The study will include patients who are at least 4 years old and less than 8 years old; all participants will need to be on a daily dose of glucocorticoids for at least 3 months prior to enrolling and to stay on daily glucocorticoids for the first 2 years of the study. The primary outcome of the study (change from baseline in North Star Ambulatory Assessment) will be assessed at 52 weeks; patients will be followed for 5 years after treatment.

The FDAs decision to grant our investigational gene therapy PF-06939926 Fast Track designation underscores the urgency to address a significant unmet treatment need for Duchenne muscular dystrophy, said Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development. We are working to advance our planned phase 3 program as quickly as possible.

The FDAs Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to drugs that are expected to have an impact on factors such as survival and daily functioning.

For more information visit pfizer.com.

Pfizer receives FDA Fast Track designation for Duchenne muscular dystrophy investigational gene therapy. https://www.businesswire.com/news/home/20201001005382/en/Pfizer-Receives-FDA-Fast-Track-Designation-for-Duchenne-Muscular-Dystrophy-Investigational-Gene-Therapy. Accessed October 2, 2020.

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Investigational Gene Therapy Fast Tracked for Duchenne Muscular Dystrophy - Monthly Prescribing Reference

NEW YORK, Oct. 05, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that the Company will present at upcoming conferences and will announce six-month safety and efficacy data from the second cohort of its AXO-Lenti-PD program the morning of Tuesday, October 6, 2020. Additionally, as previously announced the Company will hold a Parkinsons Disease R&D Day on Friday, October 30.

Information on the upcoming presentations can be found below:

Chardan 4th Annual Genetic Medicines Conference

2020 ARM Virtual Cell and Gene Meeting on the Mesa

AXO-Lenti-PD Parkinsons Disease R&D Day

A live webcast of the presentations will be available in the Events section of Axovant's website at http://www.axovant.com. Replays will be available for approximately 30 days following the conferences.

About Axovant Gene Therapies

Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit http://www.axovant.com.

Contacts:

Media & Investors

Parag MeswaniAxovant Gene Therapies Ltd.(212) 547-2523media@axovant.cominvestors@axovant.com

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Axovant to Participate in Upcoming October Conferences and Provide AXO-Lenti-PD Program Update the Morning of October 6th - BioSpace

Global Cancer Gene Therapy Market: Overview

Cancer could be defined as uncontrolled cell growth in the body leading to organ malfunction. If untreated, it can lead to death. Uncontrolled growth of cell is managed by the body in several ways, one of them is by deploying white blood cells to detect and eradicate these cancerous cells. It has been discovered that the immune system could be manipulated to influence cancerous cells to destroy itself.

Read Report Overview https://www.transparencymarketresearch.com/cancer-gene-therapy-market.html

Radiation and chemotherapy therapy have consistent and reliable effects to decrease cancerous cells in the body. Recently, immunotherapy for hematological cancers has experienced a recognition and is of interest for many researchers Scientists have developed methods to isolate, replicate, and develop cancer-destroying cells from the patients blood cancer and injecting those cells back for the destruction of their cancers, with durable remissions.

New options for the treatment is needed to be developed if order to achieve elimination of cancer suffering and death by 2020. According to NCI, 5-year survival rate for cancers such as lung (15%), glioblastoma (5%), pancreatic (4%), and liver (7%) remains very low. Current available treatments have several side effects, the systemic toxicity due to chemotherapy results in nausea, mild cognitive impairments, and mouth ulcerations, in addition to long-term side effects such as increasing risk of developing other types of cancers. Therefore, new and innovative treatment methods are required to reduce the suffering of cancer patients.

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Global Cancer Gene Therapy Market: Drivers and Restraints

The emerging field of cancer Gene Therapy offers varied potential treatments. Gene therapy involves a range of treatment types, which use genetic material to alter cells (either in vivo or in vitro) to help cure the disease. Cancer Gene Therapy shown efficacy in various in vitro and preclinical testing. Preclinical testing for cancer gene therapy has been performed on glioma, pancreatic cancer, liver cancer, and many other cancers.

Increase in prevalence of cancer, rise in government funding and initiatives, growth in pipeline of cancer gene therapy products, and collaborations to develop and launch gene-therapy products are some factors driving the market. According to NCBI researchers, development of genetically-modified T-cell therapies for treatment of cancer has had maximum clinical impact among other gene therapies. However, high treatment cost is a major limitation in the cancer gene therapy market. The reason behind the huge cost for cancer gene therapy is the necessity of rigorous, exhaustive clinical trials; also treatment by cancer gene therapy differs from person to person depending upon the genetic acceptance of every patient, unlike other drugs thereby limiting the market growth.

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Global Cancer Gene Therapy Market: Key Segments

Based on type, the cancer gene therapy market is segmented into gene transfer immunotherapy and oncolytic virotherapy. Immunotherapy uses genetically modified cells and viral particles to stimulate the immune system to destroy cancer cells. Immunotherapy include treatment with either cytokine gene delivery or tumor antigen gene delivery. Oncolytic virotherapy uses viral particles, which replicate within the cancer cell causing the death of the cell. It is an emerging treatment modality that is expected to shows great promise, particularly in metastatic cancer treatment. It includes treatment with adenovirus, retrovirus, lentivirus, herpes simplex virus, adeno-associated virus, simian virus, alphavirus, and vaccinia virus. Gene transfer is the newest treatment modality that is expected to introduce new modified genes into cancerous cell or associated tissue for destruction of cell or to slow down cancer growth. This technique is flexible as a wide variety of vectors and genes are used for clinical trials with positive outcomes. As gene therapy advance, they could be used alone or in combination with other treatments to control the disease. Gene transfer or gene replacement is performed using naked/plasmid vectors, electroporation, sonoporation, magnetofection, and gene gun.

Pre Book Cancer Gene Therapy Market Report at https://www.transparencymarketresearch.com/checkout.php?rep_id=40985&ltype=S

Based on region, the global cancer gene therapy market is segmented into North America, Europe, Asia Pacific, Latin America and Middle East & Africa. North America is anticipated to hold the largest market share. The U.S. dominates the cancer gene therapy market owing to its increase in funding for research & development and other government initiatives. Key players in the biotech industry are engaging in research & development of gene therapy products. Moreover, rising demand for DNA vaccines and growing interest of venture capitalists to investment in commercialization of gene-based cancer therapies are likely to propel the market. The cancer gene therapy market in Asia Pacific is anticipated to expand at a rapid pace as in China cancer gene therapy is anticipated to attribute for largest revenue, due to the recent launch of Gendicine and rising healthcare expenditure with strong R&D facilities.

Global Cancer Gene Therapy Market: Key Players

Key players operating in the global cancer gene therapy market are Adaptimmune, ZioPharm Oncology Altor Bioscience, MolMed, bluebird bio, Shanghai Sunway Biotech company limited , MultiVir, Shenzhen SiBiono GeneTech, Corporation.

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Cancer Gene Therapy Market is Expected to Expand at an Impressive Rate by 2025 - The Daily Chronicle

CAMBRIDGE, Mass., Oct. 05, 2020 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, announced today that Phillip Samayoa, vice president of strategy and portfolio development, will present a company overview at the annual Cell & Gene Meeting on the Mesa. The presentation will be available for registered participants to view on demand throughout the conference, to be held virtually Oct. 12-16, at meetingonthemesa.com.

Geoff McDonough, M.D., Generation Bios president and chief executive officer, will participate in a panel on the future of gene delivery. That discussion will be available to view on demand on the conference website starting Oct. 13.

About Generation Bio

Generation Biois an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases. The companys non-viral platform incorporates a proprietary, high-capacity DNA construct called closed-ended DNA, or ceDNA; a cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed. The ctLNP is designed to deliver large genetic payloads, including multiple genes, to specific tissues to address a wide range of indications. The companys efficient, scalable manufacturing process supports Generation Bios mission to extend the reach of gene therapy to more people, living with more diseases, in more places around the world.For more information, please visitgenerationbio.com.

Contact:

InvestorsChelcie ListerTHRUST Strategic Communicationschelcie@thrustsc.com910-777-3049

MediaStephanie SimonTenBridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

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Generation Bio to Present at 2020 Virtual Cell & Gene Meeting on the Mesa - GlobeNewswire

Nearly five months after snagging a conditional approval for its spinal muscular atrophy (SMA) gene therapy in Europe, Novartis is rolling out fresh data to support a launch that has seen its share of challenges.

The company unveiled interim data from a phase 3 trial of the gene therapy in babies born with the genetic disease. Of the 33 patients treated so far in the European trial, 21 had achieved milestones in motor skills during a mean follow-up period of 10.6 months that the disease would normally prevent, the company said. For example, six could sit without assistance for more than 10 seconds and 20 could control their head movements.

Most of the children in the study who entered it without requiring ventilation remained free of ventilatorsupport, and 67% were able to eat without help, Novartis said during the World Muscle Society 2020 Virtual Congress. Most of the children received a higher score on a widely used test of neuromuscular functioning than untreated SMA patients typically do, the company added.

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Shephard Mpofu, M.D., chief medical officer of Novartis Gene Therapies, said in a statement that the data boosts previous clinical trial results supporting the use of Zolgensma in patients with type 1 SMA, the most common form of the disease. With more than 600 patients now treated, including some more than five years post-treatment and more than five years old, these data further reinforce the transformative benefit a one-time dose of Zolgensma has on SMA patients, Mpofu said.

RELATED: New Zolgensma 'inflection point' is here as Novartis snags EU nod for SMA gene therapy

Novartis effort to win approval for Zolgensma in Europe ran into one hurdle back in April 2019, when an infant in the trial died of brain damage and respiratory issues. An autopsy later revealed that the death was unrelated to Zolgensma.

But then, in October of last year, Novartis was hit with questions from regulators in Europe and Japan about the Zolgensma manufacturing process. Resolving the queries from the EU required inspections, and it pushed back thedecision from the EUs Committee for Medicinal Products for Human Use (CHMP).

Now that Zolgensma is finally approved in Europe, though, Novartis has an opportunity to steal market share from Biogens SMA drug Spinraza. Thats because the European approval allows the Zolgensma to be used in children weighing up to 21 kilograms, which basically covers any child under the age of 5.

Thats a significant difference from the FDA approval, which only covers children under 2 years of ageand it could allow Novartis to offer its therapy to older children currently taking Spinraza.

RELATED: Roche touts 2-year Evrysdi data as oral SMA drug drives early interest

Novartis has an ambitious expansion plan for Zolgensma in the U.S., too, though that has hit some obstacles recently. Its working on a new intrathecal formulation of the drug, in the hopes that the FDA will approve that version for children up to 5 years old. But last week, the company said the FDA will require it to run a phase 3 study of the intrathecal formulation, rather than considering it for approval based on a smaller phase 1/2 trial. That could push the filing to 2023.

Analysts deemed the FDAs decision a major setback. Jefferies analysts had estimated the gene therapy would peak at $2.8 billion in worldwide sales, with the intrathecal version claiming $1 billion of that.

Meanwhile, Novartis is facing potential competition from more than just Spinraza. Roche introduced an oral drug for SMA, Evrysdi, last month. And it just revealed new data from a pivotal trial of that drug that intensify Novartis marketing challenge.

In the Evrysdi study, 88% of infants with type 1 SMA were alive without needing full-time ventilation after two years. Their motor functioning also improved in the second year, with more patients able to sit, stand, turn over or maintain head control.

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Novartis cites 'transformative' data on Zolgensma as it rolls out SMA gene therapy in Europe - FiercePharma

SAN RAFAEL, Calif., Oct. 2, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a pioneer in developing treatments for phenylketonuria (PKU) and gene therapies, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation toBMN 307, an investigational gene therapy for the treatment of individuals with PKU.

Fast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fulfill an unmet medical need, enabling drugs to reach patients earlier. Clinical programs with Fast Track designation may benefit from early and frequent communication with the FDA throughout the regulatory review process. These clinical programs may also be eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met, as well as Rolling Review, which means that completed sections of the Biologic License Application can be submitted for review before the entire FDA application is complete. Both the FDA and European Medicines Agency have granted BMN 307 Orphan Drug Designation.

"Fast Track designation combined with our ability to conduct our clinical studies incorporating material manufactured using a commercial-ready process will further facilitate rapid clinical development of BMN 307 gene therapy," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "We are looking forward to working closely with the FDA, as well as other health agencies, to evaluate the safety and efficacy of this promising investigational gene therapy as we continue our unwavering 15-year commitment to advance the standard of care for people with PKU."

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down Phe, an amino acid that is commonly found in many foods. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychological issues, affecting a person's ability to think and problem solve, and can lead to depression, anxiety, and behavior disturbance impacting quality of life. Due to the seriousness of these symptoms, in many countries, infants are screened at birth to ensure early diagnosis and treatment to avoid intellectual disability and other complications. According to treatment guidelines, PKU patients should maintain lifelong control of their Phe levels.

BMN 307 Clinical Program

Last week, BioMarin announced that it had dosed the first participant in the global Phearless Phase 1/2 study with BMN 307, an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells. BMN 307 will be evaluated to determine safety and whether a single dose of treatment can restore natural Phe metabolism, increase plasma Phe levels, and enable a normalization of diet in patients with PKU.BioMarin is conducting this study with material manufactured with a commercial-ready process to facilitate rapid clinical development and potentially support approval. BMN 307 represents a potential third PKU treatment option in BioMarin's PKU franchise and a second gene therapy development program.

BioMarin's clinical program is composed of two key studies. Phearless, a Phase 1/2 study, will evaluate the safety, efficacy, and tolerability of a single intravenous administration of BMN 307 in patients with PKU. The study consists of a dose-escalation phase, followed by a cohort expansion phase once an initially efficacious dose has been demonstrated. In addition, BioMarin is sponsoring an observational study, Phenom, which includes patients with PKU to measure both established and new markers of disease and clinical outcomes over time.

BioMarin's 15-Plus Year Commitment to PKU Research

For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients. BioMarin has developed therapies that have been used to treat approximately 7,000 PKU patients around the world. The company has two approved PKU therapies, and the investigational gene therapy BMN 307 is currently in development. BioMarin has conducted 41 clinical studies in PKU and has sponsored 44 external clinical studies. BioMarin researchers have authored 65 publications in medical and scientific journals on PKU and supported another 57 publications by external researchers.

About Gene Therapy

Gene therapy is a form of treatment designed to address a genetic problem by adding a normal copy of the defective gene. The functional gene is inserted into a vector containing a small DNA sequence that acts as a delivery mechanism, providing the ability to deliver the functional gene to targeted cells. The cells can then use the information from the normal gene to build the functional proteins that the body needs, potentially reducing or eliminating the cause of the disease.

Gene Therapy Manufacturing

BioMarin has leveraged its knowledge and experience in manufacturing complex biological products to design, construct and validate a state-of-the-art vector production facility in Novato, California. This facility is the site of production for both valoctocogene roxaparvovec and BMN 307, investigational gene therapies. Manufacturing capabilities are an essential driver for BioMarin's gene therapy programs and allows the Company to control quality, capacity, costs and scheduling enabling rapid development. Production of BMN 307 with a commercial ready process at scale reduces risk associated with making process changes later in development and may speed overall development timelines significantly.

Ongoing process development efforts and experience gained at commercial scale have led to improvements in productivity and operational efficiency. The ability to scale out the facility with additional equipment combined with the improvements in productivity result in a doubling of overall potential capacity to 10,000 doses per year, combined for both products, depending on final dose and product mix. This improvement in productivity is anticipated to meet potential commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well into the future.

About Phenylketonuria

PKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic disorder affecting approximately 70,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.

To learn more about PKU and PAH deficiency, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases.The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.For additional information, please visitwww.biomarin.com. Information on such website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the Company's BMN 307 program being eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met, as well as Rolling Review, the development of BioMarin's BMN 307 program generally, including the impact on the timing and process for regulatory interactions and decisions, BioMarin's gene therapy manufacturing capabilities and the anticipation that the current manufacturing capabilities will meet potential commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well into the future and the impact of using material manufactured at commercial scale in a clinical trial on reducing risk and speeding up overall development timelines. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others:the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities; uncertainties inherent in research and development, including unfavorable new clinical data and additional analyses of existing clinical data; the results and timing of current and future clinical trials related to BMN 307; our ability to reproducibly and consistently manufacture sufficient quantities of BMN 307, the possibility that changes may be required to the current manufacturing process; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

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BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

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SOURCE BioMarin Pharmaceutical Inc.

https://www.biomarin.com/

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BioMarin, Pioneer in Phenylketonuria (PKU) and Gene Therapy, Receives FDA Fast Track Designation for PKU Investigational Gene Therapy, BMN 307 -...

Gene therapy is no longer an approach for the future. It's a technique used now.

As of January 2020, the FDA has approved four gene therapies for use in the United States and has received more than 900 investigational new drug (IND) applications for clinical studies. At present, more than 3,400 active gene therapy trials are taking place worldwide, according to ClinicalTrials.gov.

None of these trials would have launched without valid preclinical research. One of the key requirements during preclinical research is selection of the appropriate animal model.

Selecting animal models that reflect the population studied and reproduce the target disease state increases the likelihood that studies will meet strict regulatory expectations. Further, strong preclinical research, with accurate data and clinically relevant biomarkers, helps ensure that clinical trials get to the finish line on time and on budget.

"If you choose a too simplistic model or a model that does not accurately recapitulate the disease state you're trying to address, your preclinical data doesn't amount to much,"said Anjli Venkateswaran, PhD, spokesperson for Biomere, a preclinical CRO based in Worcester, Massachusetts. "Selection becomes even more important in gene therapy research, and it's something scientists grapple with daily."

Gene therapy researchers rely on animal models to assess variables such as safety, efficacy, dosage and localization of transgene expression. Traditional inbred and outbred mice the most common laboratory mice are suitable for most pharmaceutical research. However, when the goal is to alter a human genetic defect, scientists need to test their approach in models that contain the human target sequences. This led to the development of genetically engineered mouse models (GEMMs) of which there are many.

"Any mouse that is genetically changed or altered to be an appropriate model for disease falls under this umbrella,"Dr. Venkateswaran said. "These models express the gene target and recapitulate some, if not all, of the disease pathophysiology."

As animal model providers gain access to next-generation sequencing, genome-engineering tools and other technology, they can develop more customized models. "Older technology was based on homologous recombination,"said Tom Pack, PhD, senior scientist for Axovant, a New York City-based clinical-stage gene therapy company. "Now we've moved into an era of CRISPR-Cas9, where you have more efficient and sophisticated technology for manipulating DNA, and you can more closely mimic human mutations. You can also focus on specific organs or tissue types with DNA recombinase-based and intersectional genetics approaches to design more targeted therapies more rapidly."

For all the technological advances, certain studies may require a different type of model. "There are limitations as to how much you can humanize mice,"Dr. Venkateswaran said. "Also, mice behave very differently physiologically from humans, and they have a different blood-brain barrier, which can result in differences in gene therapy delivery to the brain. Because of their anatomical differences, large-animal models are emerging in gene therapy."

Large-animal models, including nonhuman primates, may suit certain studies more specifically than humanized mice. For example, pig models have been used effectively in cystic fibrosis studies because their respiratory is more similar to humans than mouse models.

However, large-animal models have limitations. They are more expensive than mouse models and require specialized scientists and technicians and adequate lab space. That's why Dr. Venkateswaran recommends that, generally, researchers should start small and move up.

"You need to get some confidence in your gene therapy's performance,"she said. "When data looks good in in vitro models and in mouse models, then, depending on the therapy, consider testing in relevant large animal models."

Axovant has two pediatric rare-disease studies in the pipeline: both fatal diseases with no other treatment options available. These delicate situations require Axovant to be especially thoughtful about model selection.

"You not only want to mimic the genetic condition, you want to be able to practice the same surgical techniques you would use to deliver the therapy as well as look at some of the same clinical biomarkers,"Dr. Pack said. "With a larger-animal model, you can optimize everything clinically before you start clinical trials."

To determine the appropriate animal model(s) for preclinical gene therapy studies, researchers must weigh both scientific and practical considerations.

Scientific considerations include:

Practical considerations include:

Gene therapy researchers have a lot to consider when designing preclinical research. Appropriate animal model selection is one of the first and most important steps to help move that research into clinical trials.

Fortunately, a select group of CROs have preclinical research expertise to help expedite the process. That includes access to complex genetically modified mouse models and other animal models, as well as scientific experts that understand the biology of these models. Many CROs, including Biomere, also have housing and breeding programs and experience with testing multiple types of therapies (CRISPR-mediated gene editing, AAV and other viruses, RNA therapeutics (RNAi andmiRNA), ceDNA, antisense oligos etc.).

"When you get it right in preclinical studies, you improve the success rate in the clinic,"Dr. Venkateswaran said. "A more thoughtful approach will lead to more robust therapies, which ultimately leads to more patient lives saved." To learn more about model selection for gene therapy studies, download Biomere's white paper.

Need a preclinical partner for a gene therapy study? Visit Biomere.com to contact one of its scientists about your preclinical study today.

Meyerholz DK. Lessons learned from the cystic fibrosis pig.Theriogenology. 2016;86(1):427-432. doi:10.1016/j.theriogenology.2016.04.057

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Preclinical done right: The importance of using relevant animal models in gene therapy studies - BioPharma Dive

Gene therapy - which aims to treat diseases by essentially inserting a gene into a patients cells rather than via drugs or surgery - was a hot trend in the biotech space, but investor interest in the sector appears to have died down considerably, on account of high R&D spending and delays companies have seen in launching revenue-generating products. Our indicative theme of Gene-Based Therapy Stocks is down by about -23% year-to-date, underperforming the S&P 500 which is up by about 2%. However, with valuations declining, these companies could be attractive bets for investors as years of investments potentially start to pay off. These companies could also be acquisition candidates for big pharma. Below is a bit more about these companies and how they have fared this year.

Sarepta Therapeutics is a commercial-stage biopharmaceutical company that develops RNA-targeted therapeutics and gene therapy products. The company recently provided some positive data on its investigational gene therapy for Duchenne muscular dystrophy. The stock is up 11% year-to-date.

SRPT

Voyager Therapeutics is a clinical-stage biotech company that is developing gene therapies for Parkinsons disease, Huntingtons disease, and other conditions. The stock is down by about -20% year-to-date.

REGENXBIO Inc.: is a clinical-stage biotechnology company working on gene-based therapies for Retinal diseases, Hunter and Hurler syndromes. The stock is down by about -33% this year.

uniQure is primarily focused on gene-based therapy for Hemophilia and is currently in the late-stage of clinical trials and another program focuses on Huntingtons disease. The stock is down by about -49% year-to-date.

What if youre looking for a more balanced portfolio instead? Heres a high-quality portfolio to beat the market, with over 100% return since 2016, versus 50% for the S&P 500. Comprised of companies with strong revenue growth, healthy profits, lots of cash, and low risk, it has outperformed the broader market year after year, consistently.

See allTrefis Price EstimatesandDownloadTrefis Datahere

Whats behind Trefis? See How Its Powering New Collaboration and What-Ifs ForCFOs and Finance Teams |Product, R&D, and Marketing Teams

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Gene Therapy Stocks Continue To Underperform. Are They Worth A Look? - Forbes

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A question hanging over gene therapies, which attempt a once-and-done fix for genetic diseases, is will their benefits endure? Monday, Sarepta Therapeutics told a scientific conference that the first four children who got its investigational gene therapy, for Duchenne muscular dystrophy, all continued to show better-functioning muscles after two years.

Durability is an important consideration for a onetime treatment, said Sareptas head of gene therapy, Louise Rodino-Klapac. She spoke on a conference call, part of an online version of the annual congress of the World Muscle Society, where scientists reported their progress against the often-deadly muscular dystrophies.

Sarepta (ticker: SRPT) said that a battery of tests that measure daily functions like walking and fatigue showed the children doing even better than they had at the one-year mark. The assessment is an average of 17 tests, and not every child showed improvement on every measure.

Perhaps that is why Sarepta stock slipped 0.5% on the day, to $143.20, in a rising stock market.

Analysts may have also been anxious to hear whether Sarepta had resolved questions raised by the U.S. Food and Drug Administration about the production process to be used in a Phase 3 study of the gene therapy. Agency demands have slowed the timetables of other gene- therapy developers, and its questions for Sarepta are delaying the launch of its pivotal trial.

Chief executive Doug Ingram said Sarepta was ready with a process to produce therapies for a clinical trial and subsequent commercial demand. Were working with the agency right now to resolve any questions that they might have, said Ingram, get their blessing and be starting that trial as soon as is possible.

RBC Capital analyst Brian Abrahams found the data encouraging. He rates Sarepta stock at Outperform, with a price target of $200 a share.

We view the results as continuing to support true functional benefits from the companys gene therapies, Abrahams said in a Monday note. These studies werent controlled, he cautioned, so investors are looking forward to Sareptas results from a randomized, controlled study for which results should be available in the first quarter of 2021.

On Sareptas heels is Pfizer (PFE), with clinical trials of a gene therapy for muscular dystrophy whose benefits it has measured out to one year.

At the conference, Sarepta also showed data from early-stage trials of a gene therapy for another group of inherited muscle diseases, known as Limb-girdle muscular dystrophies. Functional tests of the first three patients showed them maintaining their improvement over 18 months.

CEO Ingram reminded the audienceand perhaps any listening regulatorsthat families are impatient for these muscular dystrophy trials conclusions.

This is truly -- Im not being hyperbolic, but I say in a very real sense, at least from our perspective, a matter of life and death, he said. We have to get this trial started. Kids are waiting for this therapy.

Write to Bill Alpert at william.alpert@barrons.com

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Sareptas First Gene-Therapy Test Patients Look Good, 2 Years Out - Barron's

SlateXpace (pronounced "Slate Space") is a CRB solution that breaks from conventional facility design by providing unparalleled flexibility, speed to market and budget control, allowing companies to quickly deliver multiple novel therapies to patients. Clients get a customizable, time and cost-efficient solution that accommodates rapidly evolving multi-modal pipelines while future-proofing their capital investments. They also get peace of mind as SlateXpace leverages the expertise and capabilities of a team that manages every detail end to end from facility fabrication and delivery, equipment selection and installation, to start-up, qualification and operator training.

The agility at the heart of SlateXpace's mission heralds a critical turning point in the story of advanced therapy medicinal product (ATMP) manufacturing. The 1980s biotech explosion, the advent of stainless-steel facilities, and the arrival of closed processing technology and eventually ballroom manufacturing concepts each ushered periods of uncomfortable adjustment followed soon by untapped potential. Today, biotechnology companies are navigating the next scientific breakthrough with the promises brought on by the rise of cell and gene therapy products. However, many operators remain constrained by technologies and processes ill-suited for a fast-shifting market.

SlateXpace seizes on that potential by giving clients full operational turnkey delivery, upfront cost and schedule confidence and infinite flexibility, allowing clients to capture new markets or take advantage of emerging technologies. Implementing SlateXpace facilities allows you to constantly transform and grow with your business to address rapidly evolving market demands.

Drawing on CRB's more than 35 years of life sciences experience, SlateXpace's unique suite-based platform moves ATMP manufacturing beyond off-the-shelf cleanroom boxes and into a new era that empowers clients to shift flexibly, quickly and seamlessly between therapies. Using SlateXpace, an operator could run a product campaign in one modality, decontaminate that space, swap out single-use and mobile equipment and run a new batch in the same space in a different modality all within a few weeks.

Modalities currently test-fitted for SlateXpace include vaccines, monoclonal antibodies (mAbs), viral vectors, plasmids, allogeneic cell therapies and autologous cell therapies as well as the potential to accommodate other future biotech modalities an "X" factor that can accommodate bespoke client designs, new technologies or unforeseen scientific breakthroughs.

"SlateXpace meets the market need for a truly essential and efficient facility design, one that makes many, highly-tailored processes possible for clients," said Ryan Schroeder, President of CRB. "We started SlateXpace with the goal of delivering a facility that would never be idle. Manufacturers have the means to rapidly shift their operations as technologies change, business expands, and patient needs evolve. That will minimize downtime and maximize growth, no matter what comes next."

"Whether it's an established biotech company or contract manufacturing organization (CMO) looking for maximum operational flexibility to support complex product combinations across their global network, or a startup company looking for scalability and cost control so they can focus on development, SlateXpace provides a holistic solution with unparalleled advantages over today's manufacturing environments" said Noel Maestre, Director of SlateXpace based in San Diego. "The term 'disruptive' gets thrown around a lot in our industry. But for our clients, SlateXpace is exactly that a game-changing turnkey facility that delivers the kind of flexibility once considered impossible for customized and novel processes."

To learn more about SlateXpace and how CRB can provide your business a future-proof facility beating all industry benchmarks for speed, flexibility and adaptability visit our website at http://www.slatexpace.com. We can also be found on LinkedInand Twitter.

About SlateXpace:

SlateXpaceTM (pronounced "Slate Space"), is a CRB forward-looking solution that provides configurable and equipment-agnostic facilities to the ever-adapting life sciences industry. It allows clients to rapidly shift operations as technologies, business objectives, and patient needs evolve. Find us at http://www.slatexpace.com and on social media.

About CRB:

CRB is a leading provider of sustainable engineering, architecture, construction and consulting solutions to the global life sciences and advanced technology industries. Our more than 1,300 employees provide best-in-class solutions that drive success and positive change for our clients, our people and our communities. CRB is a privately held company with a rich history of serving clients throughout the world, consistently striving for the highest standard of technical knowledge, creativity and execution.

MEDIA CONTACT INFORMATION:Chris Clark: 816-200-5234

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CRB's SlateXpace gives cell and gene therapy clients new flexibility, control over manufacturing needs to support highly complex product pipelines -...

Pfizer snagged Fast Track designation from the U.S. Food and Drug Administration (FDA) for its Duchenne muscular dystrophy (DMD) gene therapy treatment, PF-06939926.

PF-06939926 is currently being evaluated to determine its safety and efficacy in boys with DMD. In May, the company reported promising preliminary results from a Phase Ib study of the gene therapy asset. Data from nine boys with DMD between the ages of six and 12 showed encouraging efficacy and manageable safety events, Pfizer said. As BioSpace reported at the time, PF-06939926 demonstrated durable and statistically significant improvements in multiple efficacy endpoints measured 12 months following infusion. These included sustained levels of mini-dystrophin expression and improvements on the North Star Ambulatory Assessment (NSAA) rating scale.

Although the efficacy is promising, there were three serious adverse events in the study. Two appeared to be immune reactions related to complement activation. While they were severe, all three events were fully resolved within two weeks.

DMD causes a progressive loss of muscle strength attributable to a loss of a protein called dystrophin, which normally protects muscle fibers from breaking down. Approximately 15,000 U.S. patients are affected with DMD, with a total of about 300,000 patients worldwide.

The Fast Track designation awarded by the FDA was based on data from that Phase Ib study. With the designation, Pfizer will be able to have an expedited review of PF-06939926 when, and if, it is submitted for potential regulatory approval.

The FDAs decision to grant our investigational gene therapy PF-06939926 Fast Track designation underscores the urgency to address a significant unmet treatment need for Duchenne muscular dystrophy, Brenda Cooperstone, Chief Development Officer of Rare Disease at Pfizer Global Product Development said in a statement. DMD is a devastating condition and patients, and their parents, are waiting desperately for treatment options. We are working to advance our planned Phase 3 program as quickly as possible.

PF-06939926 is an investigational, recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor. The rAAV9 capsid was chosen as the delivery vector because of its potential to target muscle tissue, Pfizer said.

Pfizer announced the Fast Track designation the same day Solid Biosciences announced the FDA lifted a nearly year-long clinical hold on its gene therapy treatment for DMD, SGT-001. The treatment delivers microdystrophin, a synthetic dystrophin gene, which encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins.

Currently, there are two FDA-approved DMD treatments. Sarepta Therapeutics Exondys 51 was approved in 2016 for DMD patients amenable to skipping exon 51. Last year, Sarepta secured another FDA approval for Vyondys 53, which was greenlit for patients amenable to skipping exon 53.

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FDA Grants Fast Track Designation to Pfizer's DMD Gene Therapy - BioSpace

With ongoing advances in science and technology, the cell and gene therapy pipeline has grown especially robust over the past few years. At present, ClinicalTrials.gov shows more than 4,500 active gene therapy trials globally. In the United States, McKinsey experts expect to see 10 to 20 cell and gene therapy approvals per year over the next five years.

This rise in supply has created a heightened demand for contract development and manufacturing organizations (CDMOs) with biotech expertise. CDMOs typically supply materials and handle production and manufacturing, allowing life sciences companies to focus on innovation and marketing.

The bottleneck stems from a shortage of CDMOs with gene therapy expertise and resources. Considering the critical need for safe, effective gene therapies and the rapid pace of development, it's important for pharma and biopharma to find a CDMO with both gene therapy capabilities and availability to take on new partners nownot 18 months from now.

"Full-service CDMOs that can assist with both development and manufacturing are in highest demand,"said Richard Welch, PhD, vice president, development services for Emergent BioSolutions, a global CDMO and specialty life sciences company headquartered in Gaithersburg, Maryland. "As pharma and biopharma companies move from early phase to late phase, CDMOs need experience with process characterization and process validation as well as commercial production and supply chain."

"The supply chain is much more complex,"added Tarek Abdel-Gawad, senior director of commercial strategy for Emergent BioSolutions. "You aren't just growing cells. You're ensuring viruses, helper viruses, and plasmid DNA work together to produce the molecule of choice. Few companies have the capabilities, equipment, and GMP expertise."

Much of the gene therapy development as of late has stemmed from smaller biotech companies or research universities according to a McKinsey report. Large pharmaceutical companies may partner with these organizations on rare disease or oncology treatments two therapeutic areas where much of the research lies.

Many small to midsize companies have the idea and investor support, but do not have the employees, infrastructure or manufacturing space. "A CDMO is a good partner in those cases,"said Mukesh Mayani, PhD, principal scientist, gene therapy at Sanofi. "You can test your hypotheses and work with a CDMO that has the platform, the people, and the preclinical models. This arrangement speeds up the timeline and allows these innovative companies to focus on other modalities and molecules."

Pharma and biopharma companies of all sizes can learn from this "single-source"approach. Partnering with a CDMO earlier in the processfrom preclinical development through packagingfrees up resources to focus on innovation and communication.

"It is neither simple nor cheap to develop and manufacture gene therapies,"said Dr. Welch. "A CDMO has the built-in skill set to grow viruses at the densities necessary to meet early-phase studies while hitting safety margins. With the clinical trial failure rate as high as it is, working with a CDMO that has experience in different technologies and products makes for a more efficient, cost-effective process."

Although there is a high demand now for CDMOs with gene therapy expertise, the market is quickly growing. According to Grand View Research, the CDMO market is expected to grow from $115.6 billion in 2020 to $157.7 billion in 2025, outpacing the pharmaceutical industry as a whole. New cell and gene therapy CDMOs are emerging and established CDMOs are expanding capabilities.

Before you start your CDMO search, consider the following two factors:

When vetting CDMOs for your gene therapy studies, consider the strengths and weaknesses of your company as well as your potential CDMO partner. A few points to consider include:

As gene therapy research continues to expand, innovators in this space will need CDMOs with highly specific expertise, facilities, and equipment. Choose a partner that can assist from the earliest phases of product development all the way to commercialization.

Capra, Emily, et al. "Gene therapy coming of age: Opportunities and challenges to getting ahead."McKinsey, October 2, 2019

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Gene therapy solution: The value of a CDMO as your end-to-end partner - BioPharma Dive

BOTHELL, Wash., Oct. 1, 2020 /PRNewswire/ --BioLife Solutions, Inc. (NASDAQ: BLFS)("BioLife" or the "Company"), a leading developer and supplier of a portfolio of class-defining bioproduction tools for cell and gene therapies, today announced it has closed the acquisition of SciSafe, a privately held multi-facility provider of biological materials storage to the cell and gene therapy and pharmaceutical industries.

SciSafe had 2019 unaudited revenue of $6 million and positive EBITDA and is anticipated to be accretive during 2021. Fourth quarter 2020 revenue is estimated at$1.8 million.

About SciSafe

Founded in 2010, SciSafe offers dedicated pharmaceutical and biological specimen storage in its four fully cGMP-compliant state-of-the-art sample management facilities. SciSafe has built flourishing relationships with over 300 of the world's leading and most admired organizations. Clients have repeatedly chosen to store their most valued and irreplaceable biological samples because they trust SciSafe to care for them as if they were their own. SciSafe values and respects its long-term client relationships. With over 60 years combined experience specifically in life sciences, SciSafe personnel fully appreciate the vital requirements of all areas of specimen storage and cold chain management. For more information, please visit http://www.scisafe.com.

About BioLife Solutions

BioLife Solutions is a leading supplier of a portfolio of class-defining cell and gene therapy bioproduction tools and services. Our tools portfolio includes our proprietaryCryoStorfreeze media and HypoThermosolshipping and storage media, ThawSTARfamily of automated, water-free thawing products, evocold chain management system, and Custom Biogenic Systemshigh capacity storage freezers. Services include SciSafe biologic and pharmaceutical materials storage. For more information, please visit http://www.biolifesolutions.com, and follow BioLife on Twitter.

Cautions Regarding Forward Looking Statements

Except for historical information contained herein, this press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, statements concerning the expected financial performance of the company following the completion of its acquisition of SciSafe, the expected synergies between the company and SciSafe, the company's ability to realize all or any of the anticipated benefits associated with the acquisition of SciSafe, the company's ability to implement its business strategy and anticipated business and operations, including following the acquisition of SciSafe, the potential utility of and market for the company's and SciSafe's products and services, guidance for financial results for 2020 and 2021, including regarding SciSafe's revenue, and potential revenue growth and market expansion, including with consideration to our acquisition of SciSafe. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. These statements are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and assumptions that could cause actual results to differ materially from those described in the forward-looking statements, including among other things, uncertainty regarding unexpected costs, charges or expenses resulting from the company's acquisition of SciSafe or the 2019 acquisitions, charges or expenses resulting from the acquisition of SciSafe; market adoption of the company's products (including the company's recently acquired products) or SciSafe's products; the ability of the SciSafe acquisition to be accretive on the company's financial results; the ability of the company to implement its business strategy; uncertainty regarding third-party market projections; market volatility; competition; litigation; the impact of the COVID-19 pandemic; and those other factors described in our risk factors set forth in our filings with the Securities and Exchange Commission from time to time, including our Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. We undertake no obligation to update the forward-looking statements contained herein or to reflect events or circumstances occurring after the date hereof, other than as may be required by applicable law.

Media & Investor Relations

Roderick de Greef

Chief Financial Officer

(425) 686-6002

[emailprotected]

SOURCE BioLife Solutions, Inc.

http://www.biolifesolutions.com

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BioLife Solutions Closes Acquisition of SciSafe, a High-Growth Biostorage Service Provider to the Cell and Gene Therapy Industry - PRNewswire

The Advanced Therapies Innovation Centre will be located in College Station, Texas, US. Credit: FUJIFILM Diosynth Biotechnologies. The new centre will house dedicated process development and innovation laboratories. Credit: Gorodenkoff/Shutterstock. The Advanced Therapies Innovation Centre is scheduled to be operational by fall 2021. Credit: CI Photos/Shutterstock.

Fujifilm Diosynth Biotechnologies (FDB) began the construction of its Advanced Therapies Innovation Centre in College Station, Texas, US.

The facility will triple the FDBs gene therapy development capabilities with the addition of dedicated process and analytical development laboratories. It will support the manufacturing of Covid-19 vaccine under the Operation Warp Speed, a US governments initiative to begin delivery of safe and effective Covid-19 vaccine in the US.

The $55m Advanced Therapies Innovation Centre is a part of the Fujifilms plan announced in November 2019 to invest $120m (13bn) in gene therapy.

FDB held a virtual groundbreaking ceremony for the facility in August 2020. The facility should be operational by fall 2021 and will add approximately 100 jobs to the Texas campus.

Advanced Therapies Innovation Centre is being constructed adjacent to the Flexible Biomanufacturing Facility (FBF), FDBs existing state-of-the-art cGMP gene therapy manufacturing facility in College Station, Texas, US.

The building will occupy an area of 60,000ft2 and be a part of a 22-acre land acquired from Lake Walk by FDB in June 2020. The facility will be equipped with multiple 500L and 2000L bioreactors. It will house designated laboratories with BSL-2 capabilities including state-of-the-art technologies for upstream, downstream and analytical development.

Gene Therapy Innovation Centre will help customers create gene therapy drugs for the treatment of genetic disorders such as cancer and muscular dystrophy.

FDF was subcontracted for the manufacturing of Covid-19 vaccine candidate by Centre for Innovation in Advanced Development & Manufacturing (CIADM) under the task order issued by the US Biomedical Advanced Research and Development Authority (BARDA) in July 2020.

Expansion of the Texas facility will enhance vaccine production at the campus. The transfer of technologies from North Carolina to Texas for the mass production of NVX-CoV2373, Novavax Covid-19 vaccine candidate, will start at the end of 2020 with the mass production beginning in early 2021.

State-of-the-art facilities of the FDB include over 50 bioreactors ranging from 3l to 200l and support technologies such as Cyto-Mine and ambr 250 screening for both flexibility and capacity.

FDB offers both single-use (200l-2,000l) and stainless steel (20,000l) manufacturing platforms, cell culture systems, QdB, process transfer-in and development process monitoring in upstream and resin screening, UF or DF development, process characterisation, intermediate stability, pegylation, hapten conjugation and enzymatic cleavage in downstream.

Expansion of the Texas facility will enhance vaccine production at the campus.

The new UV-vis spectroscopy technology, SoloVPE, is utilised to deliver precise measures of concentration in less than a minute. It allows less time spent on in-process processing and more time spent producing the product.

The technology uses calculations of variable-path length to define the linear absorbance spectrum that is connected to the path length.

FDB is a contract development and manufacturing organisation (CDMO) with offices in College Station and Research Triangle Park in the US, Teesside in the UK, and Hillerod in Denmark. It focusses on the development and manufacturing of microbial, mammalian, and viral therapies, gene therapy and vaccines.

Core FDB services include process development, analytical development and current good manufacturing practice (cGMP) manufacturing.

The company offers an extensive list of premium services including process development, cell-line development, analytical development, clinical and FDA-approved commercial manufacturing, using its proprietary microbial pAVEway microbial and Apollo cell line systems technologies.

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Fujifilm Diosynth Biotechnologies' Advanced Therapies Innovation Centre - pharmaceutical-technology.com

Although the therapeutic options for sickle cell anemia have expanded over the past years, the clinical complications of the condition as well as the limitations of these pharmacotherapies have called for an urgent need to implement a personalized treatment strategy for patients that is based on risk stratification.

In a recent article, Emily Meier, MD, of the Indiana Hemophilia and Thrombosis Center, elucidated on the key considerations in the prescribing of such therapies as well as the current barriers that preclude healthcare providers from achieving an optimal treatment strategy for these patients.

With increasing therapeutic options, the ideal scenario for children with SCA would be one similar to childhood acute lymphoblastic leukemia (ALL) risk stratification: treatment intensity varies with risk level, Meier wrote.

Thus, children who are at low risk for sickle cell anemia complications would receive less intense therapies, which includes a continuation of hydroxyurea. On the other hand, those with the highest risk would be recommended to immediately receive one or more curative therapies, such as hematopoietic stem cell transplant, gene therapy, transfusion therapy, voxelotor, and/or crizanlizumab.

Of course, as Meier noted, there are certain limitations that must be considered before implementing such a strategy.

For one, crizanlizumab and voxelotor are approved for ages 16 and 12 years, respectively. According to the risk based therapy model, high-risk patients should only use both therapies once age appropriate.

Similarly, patients with medium risk of complications should only use L-glutamine once they reach the appropriate age of 5 years.

Additionally, there is no validated predictor for the overall severity of the disease prior to the onset of associated complications. Currently available predictors of a severe outcome is an abnormal velocity on transcranial Doppler ultrasonography. These predictors identity children at highest risk for stroke.

Meier noted that there are no predictors for vaso-occlusive episodes or acute chest syndrome.

Furthermore, there is no unanimous agreement of what constitutes severe sickle cell anemia. However, the inclusion criteria for hematopoietic stem cell transplant is considered a promising start.

Overall, Meier suggested that hydroxyurea should be the standard of care in pediatric and adult patients, regardless of disease severity.

In adults with sickle cell anemia, a risk-based strategy should still be utilized, but the end organ injury makes such an approach more challenging to implement.

She suggested that the additional FDA-approved treatments should be based on clinical and laboratory complications that are still present even after hydroxyurea dosing has been maximized.

According to the seminal trials in support of these agents, L-glutamine and crizanlizumab should be considered as additional therapy in patients who continue to experience vaso-occlusive episodes. Meier also encouraged the addition of voxelotor to hydroxyurea for those adults who continue to have significant anemia.

Hopefully, as the number of SCA modifying and curative therapies increase, more innovative treatment strategies will be tested and lead to improved quality of life and increased life expectancy for individuals with SCA, she concluded.

The opinion piece, What are the key considerations when prescribing pharmacotherapy for sickle cell anemia? was published online at Taylor & Francis Online.

Originally posted here:
Risk Based Therapy Approach for Sickle Cell Anemia Patients is Ideal Scenario - MD Magazine

CAMBRIDGE, Mass., Oct. 02, 2020 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today announced that Jennifer Ziolkowski, Chief Financial Officer, Joel Schneider, Chief Technology Officer and Cathryn Clary, Interim Chief Medical Officer, will participate in a virtual fireside chat at the Chardan 4th Annual Genetic Medicines Conference on Tuesday, October 6, 2020 at 4:45 pm ET.

A live webcast of the fireside chat will be available on the Events page of the Investors section of the Company website or by clicking here. A webcast replay will be archived for approximately 30 days on the Events page.

About Solid BiosciencesSolid Biosciences is a life sciences company focused on advancing transformative treatments to improve the lives of patients living with Duchenne. Disease-focused and founded by a family directly impacted by Duchenne, our mandate is simple yet comprehensive work to address the disease at its core by correcting the underlying mutation that causes Duchenne with our lead gene therapy candidate, SGT-001. For more information, please visitwww.solidbio.com.

Investor Contact:David CareyFINN Partners212-867-1768David.Carey@finnpartners.com

Media Contact:Erich SandovalFINN Partners917-497-2867Erich.Sandoval@finnpartners.com

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Solid Biosciences to Participate in Virtual Fireside Chat at the Chardan 4th Annual Genetic Medicines Conference - GlobeNewswire