Coave Therapeutics to Present at Upcoming Conferences Yahoo Finance UK
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Coave Therapeutics to Present at Upcoming Conferences - Yahoo Finance UK
4DMT Announces Update on Regulatory Interactions and Development Path for 4D-710 for Treatment of Cystic Fibrosis Yahoo Finance
Advancements in Biotechnology: Navigating the Future of Genetic Medicine FinSMEs
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Advancements in Biotechnology: Navigating the Future of Genetic Medicine - FinSMEs
People Who Are Genetically Prone to Obesity Need to Work Out Harder to Avoid Weight Gain Yahoo Movies Canada
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People Who Are Genetically Prone to Obesity Need to Work Out Harder to Avoid Weight Gain - Yahoo Movies Canada
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6 Best Genomics Stocks to Buy in 2024 The Motley Fool
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Gene therapy hailed as medical magic wand for hereditary swelling disorder The Guardian
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Gene therapy hailed as medical magic wand for hereditary swelling disorder - The Guardian
BridgeBio Pharma Secures up to $1.25 Billion of Capital from Blue Owl and CPP Investments to Accelerate the ... GlobeNewswire
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BridgeBio Pharma Secures up to $1.25 Billion of Capital from Blue Owl and CPP Investments to Accelerate the ... - GlobeNewswire
Boston-area companies usher in 'the age of genomic medicines' - Boston Business Journal The Business Journals
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Boston-area companies usher in 'the age of genomic medicines' - Boston Business Journal - The Business Journals
If you have family members with Parkinsons disease, or if you yourself have the disease and are concerned about your childrens chances of developing it, youve probably already wondered: Is there a gene that causes Parkinsons disease? How direct is the link?
About 15 percent of people with Parkinsons disease have a family history of the condition, and family-linked cases can result from genetic mutations in a group of genes LRRK2, PARK2, PARK7, PINK1 or the SNCA gene (see below). However, the interaction between genetic changes, or mutations, and an individuals risk of developing the disease is not fully understood, says Ted Dawson, M.D., Ph.D., director of the Institute for Cell Engineering at Johns Hopkins.
Heres what you need to know:
Theres a long list of genes known to contribute to Parkinsons, and there may be many more yet to be discovered. Here are some of the main players:
SNCA: SNCA makes the protein alpha-synuclein. In brain cells of individuals with Parkinsons disease, this protein gathers in clumps called Lewy bodies. Mutations in the SNCA gene occur in early-onset Parkinsons disease.
PARK2: The PARK2 gene makes the protein parkin, which normally helps cells break down and recycle proteins.
PARK7: Mutations in this gene cause a rare form of early-onset Parkinsons disease. The PARK7 gene makes the protein DJ-1, which protects against mitochondrial stress.
PINK1: The protein made by PINK1 is a protein kinase that protects mitochondria (structures inside cells) from stress. PINK1 mutations occur in early-onset Parkinsons disease.
LRRK2: The protein made by LRRK2 is also a protein kinase. Mutations in the LRRK2 gene have been linked to late-onset Parkinsons disease.
Among inherited cases of Parkinsons, the inheritance patterns differ depending on the genes involved. If the LRRK2 or SNCA genes are involved, Parkinsons is likely inherited from just one parent. Thats called an autosomal dominant pattern, which is when you only need one copy of a gene to be altered for the disorder to happen.
If the PARK2, PARK7 or PINK1 gene is involved, its typically in an autosomal recessive pattern, which is when you need two copies of the gene altered for the disorder to happen. That means that two copies of the gene in each cell have been altered. Both parents passed on the altered gene but may not have had any signs of Parkinsons disease themselves.
Our major effort now is understanding how mutations in these genes cause Parkinsons disease, says Dawson. SNCA, the gene responsible for making the protein that clumps in the brain and triggers symptoms, is particularly interesting.
Our research is trying to understand how alpha-synuclein works, how it travels through the brain, says Dawson. The latest theory is that it transfers from cell to cell, and our work supports that idea. Weve identified a protein that lets clumps of alpha-synuclein into cells, and we hope a therapy can be developed that interferes with that process.
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The Genetic Link to Parkinson's Disease - Hopkins Medicine
Ovid Therapeutics has struck a deal with young biotechnology company Gensaic, hoping the startups method of delivering genetic medicines can yield new brain drugs.
Under the deal, the partners will develop up to three gene-based treatments for neurological conditions Ovid is targeting. The New York biotech will get rights to license any gene therapies that emerge from the deal, so long as the two can agree on terms. Ovid also invested $5 million in the startup and committed to participate in future financing rounds.
The deal is the latest step in a rebuilding plan for Ovid, a biotech former Teva and Bristol Myers Squibb executive Jeremy Levin formed seven years ago.
Levins plan in starting Ovid was to grab medicines overlooked elsewhere, license them and develop them for rare brain diseases. That strategy led Ovid to two medicines the company developed for Angelmans syndrome and rare forms of epilepsy, and helped the biotech to go public in 2017.
Ovid hasnt been successful, however. The Angelmans drug failed a Phase 3 trial in 2020, erasing more than half of the companys value. One year later, Ovid, aiming to bolster its dwindling cash reserves, sold rights to the epilepsy drug back to Takeda. Though Ovid can still receive milestone payments and royalties from the drug, which is now in late-stage testing, its only remaining in-house programs are in preclinical testing. At just over $2 apiece, shares trade near all-time lows.
Recently, Ovid has taken steps to restock its pipeline. One experimental medicine for treatment-resistant epileptic seizures could start human trials later this year, while a licensing deal with AstraZeneca and a related partnership with Tufts University could yield other drug candidates that might follow in 2024.
The alliance with Gensaic adds up to three more prospects, while pushing Ovid into the field of gene therapy.
Gensaic was seeded in 2021 as M13 Therapeutics and is currently housed in Cambridge, Massachusetts biotech startup incubator LabCentral. Over the past two years, the company has won awards in multiple startup competitions for its research into a method of gene therapy delivery designed to overcome the limitations of standard approaches.
Many gene therapies rely on modified viruses to send genetic instructions into the bodys cells. Those delivery vehicles are used in multiple products approved for rare inherited diseases, but they also come with weaknesses, too. One commonly used tool, the adeno-associated virus, can only carry a relatively small amount of genetic cargo and is sometimes shut down by the body. Another, the lentivirus, also has limited packaging capacity and has been linked in rare cases to the development of cancers.
Gensaic instead aims to use tiny particles derived from phages, the viruses that infect bacteria, to deliver genetic material. Gensaic claims these particles can be engineered to target multiple tissue types among them the lung and brain and can carry much larger genes. Gensaic believes they may have the potential to be administered more than once, too, though that hasnt yet been proven.
In a statement, Levin said the approach appears to be optimal for carrying substantial genetic cargo across the blood-brain barrier, a filtering mechanism the body uses to keep foreign substances out of the brain.
We believe it may hold the potential to treat a broad continuum of diseases in the brain, Levin said.
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Ovid turns to gene therapy startup to restock drug pipeline - BioPharma Dive
CRISPR-Cas9 genome editing has changed the game for gene therapy, but carries safety risks when cutting DNA. The new U.S. firm Epic Bio aims to reduce these risks by targeting epigenetic controls on gene expression.
The development of the genome-editing tool CRISPR-Cas9 caused a paradigm shift in the biotech industry because it made it easier than ever to make small edits to the genetic code. The tool is also being tested in clinical trials to see if it can form the basis of gene and cell therapies for conditions including genetic blindness, cancer and blood disorders.
However, CRISPR-Cas9 gene editing also has its limitations. One is that the Cas9 protein used to cut DNA molecules can also make permanent cuts in unexpected parts of the genome, which could be dangerous for the cell. Another is that the CRISPR-Cas9 machinery is too large to deliver into the patients body using adeno-associated viral (AAV) vectors, the most common delivery method for gene therapies.
To overcome the obstacles for CRISPR-Cas9 gene editing, the startup Epic Bio was launched in July 2022 with an impressive Series A round worth $55 million. The firm, based in San Francisco, U.S., is developing gene therapies based on editing the epigenome, a biological system that cells use to control which genes become proteins.
Epic Bios therapies involve fusing together a protein that binds to DNA with a so-called modulator protein that can make epigenetic changes to the DNA molecule. This construct is directed to a target site in the genome using a customized guide RNA molecule. Epic Bios technology is dubbed Gene Expression Modulation System, or GEMS for short.
CRISPR-Cas9 binds and cuts the DNA whereas GEMS binds and modifies the chemistry of DNA without changing the genetic code, explained Amber Salzman, CEO of Epic Bio. This allows fine-tuning gene expression and avoids the risks of cutting DNA.
Epic Bio is deploying its epigenome-editing therapies in a range of rare diseases such as facioscapulohumeral muscular dystrophy, heterozygous familial hypercholesterolemia, and forms of retinitis pigmentosa. In each case, the therapy is designed to correct harmful epigenetic changes to genes that are linked to the disease.
Epic Bio aims to prepare for clinical testing by the end of 2023. According to Salzman, earlier generations of gene therapy technology have struggled to treat these diseases as they arent precise enough to hit the target site in the genome.
By leveraging CRISPR and sequence-specific guide RNAs to home to target sequences, Epic Bio can address limitations of specificity, said Salzman. Similarly, robust and durable activators and suppressors are needed to drive desired target gene behaviors. Epic Bio has the largest library of such precise epigenetic modulators to address this challenge.
Another problem with gene editing therapies is that its tough to deliver them to the patient in vivo because AAV vectors can only carry a small amount of genetic cargo. To get around this problem, Epic Bio licensed a tiny DNA-binding protein called CasMINI from Stanford University, which allows the companys gene therapies to fit on a single AAV vector.
Today, AAV is the most validated vector to deliver genetic medicine in vivo, and our therapies can fit in an AAV, explained Salzman. She added that the main alternative delivery method, via lipid nanoparticles, is currently limited to targeting the liver.
Because of the small size of CasMINI, that leaves more room for guide RNAs and multiple modulators that could perhaps regulate multiple genes at a time.
Epic Bio is one of several biotech players that have kicked off in the epigenome editing space. Chroma Medicine launched in late 2021 with a neat $125 million investment. This was swiftly followed by Tune Therapeutics, which debuted with $40 million. As it launched, Chroma Medicine also acquired another epigenome editing specialist, the Italian firm Epsilen Bio.
Epigenome editing remains an emerging therapeutic field with a lot of challenges. For example, its crucial to make sure the target sequence is verified when making epigenetic changes, and companies need to avoid the bodys own DNA repair systems reversing the edits. Nonetheless, the technology has a lot of potential to treat conditions that have been out of reach of traditional gene therapies.
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Epic Bio makes gene therapies by editing the epigenome - Labiotech.eu
Clinical features of patients
Between 2015 and 2017, a total of 177 patients (81 males; median [range] age, 4 [030] years) from 169 families were referred to the TOKAI-IRUD program. All patients registered in this study were new patients, i.e., those who had not been previously analyzed for comprehensive genomic variants; however, several patients have been included in a few subsequent investigations19,20,21,22.
The TOKAI-IRUD program is open to the possibility of accepting any patient. The clinical symptoms of the applicants were global developmental delay (HP: 0001263; n=95, 54%), seizures (HP: 0001250; n=40, 23%), intellectual disability (HP: 0001249; n=29, 16%), muscular hypotonia (HP: 0001252; n=24, 14%), dysmorphic facial features (HP: 0001999; n=17, 9.6%), short stature (HP: 0004322; n=14, 7.9%), microcephaly (HP: 0000252; n=11, 6.2%), and others (n=38, 21%) (Table 1, Supplementary Table S2, and Supplementary Table S3).
In accordance with ACMG guidelines, pathogenic SNVs were identified in 36 (20%) patients. Furthermore, 30 (17%) patients carried SNVs classified as likely pathogenic based on clinical validity assessment and consistency in clinical information and phenotypes with applicable diseases. Among 66 patients with pathogenic or likely pathogenic SNVs, 47 had autosomal dominant genetic disorders, seven had autosomal recessive genetic disorders, eight had X-linked dominant genetic disorders, and four had X-linked recessive genetic disorders (Fig.1).
Patient characteristics and information on detected variants. Each column indicates one patient. SNV single-nucleotide variant, CNV copy number variant, UPD uniparental disomy, AD autosomal dominant, AR autosomal recessive, XLD X-linked dominant, XLR X-linked recessive.
Copy number analysis identified diagnostic duplication/deletion in 11 (6.2%) patients, and these included a 10q26.3 deletion (TOKAI-IRUD-1135 and TOKAI-IRUD-1273), 22q11.2 duplication (TOKAI-IRUD-1236), 5q14.3 deletion (TOKAI-IRUD-1252), 47,XXY (TOKAI-IRUD-1297), 1p36 deletion (TOKAI-IRUD-1301), 7q11.23 duplication (TOKAI-IRUD-1321), 19p13.13 deletion (TOKAI-IRUD-1335), 16p13.3 duplication (TOKAI-IRUD-1337), 17p11.2 duplication (TOKAI-IRUD-1343), and 4p16.3 deletion (TOKAI-IRUD-1475).
ROH analysis identified homozygous regions larger than 10Mb in 105 cases; this included a diagnostic upd(15)pat in 1 patient (0.6%) who was diagnosed with Angelman syndrome (TOKAI-IRUD-1290, OMIM #105830). Furthermore, UPD of a whole chromosome was identified in 2 (1.1%) patients [upd(2)pat; TOKAI-IRUD-1249 and upd(3)pat; TOKAI-IRUD-1180] with no diagnostic SNVs or CNVs. Thus, genetic diagnoses were obtained for 78 of 177 (44%) patients, and of these, 10 (13%) cases were diagnosed with diseases recognized after 2015, i.e., when this project was initiated. A considerable number of patients showed a milder phenotype (26 [33%]), a more severe phenotype (9 [12%]), or an atypical complex phenotype (17 [22%]) compared to conventional clinical presentation of the respective disease.
TOKAI-IRUD-1290 with upd(15)pat: The patient, a 2-year-old boy at the time of sample submission, was the third of three children of healthy non-consanguineous parents (Fig.2b). Gyrus dysplasia, suspected since the fetal period, was confirmed by magnetic resonance imaging (MRI) after birth (Fig.2a). He was tube fed due to difficulties with oral intake and a tracheostomy was performed after repeated aspiration pneumonia. He also had congenital hydronephrosis, congenital hypothyroidism, gastroesophageal reflux disease, developmental delay, epilepsy, deafness, and laryngotracheomalacia. ROH analysis identified a paternal UPD region over the entire length of the long arm of chromosome 15 [upd(15)pat], covering the region of the UBE3A gene, which led to a diagnosis of Angelman syndrome (OMIM#105830) (Fig.2b). Additionally, 11 homozygous rare variants were identified in a paternally derived UPD region, which included a DUOX2 (c.G1560C, p.E520D) variant. DUOX2 is a known causative gene for congenital hypothyroidism, but this particular variant has not been previously reported.
Clinical features and results of UPD analysis of TOKAI-IRUD-1290. (a) Brain MRI at the age of 2years showing cortical dysplasia of the temporal lobes (arrowheads) and corpus callosum dysgenesis (arrow). (b) Results of UPD analysis. A paternally inherited UPD region over the entire length of the long arm of chromosome 15 [upd(15)pat] was identified, which covers the region of the UBE3A gene. (c) H2O2-producing capacity of the DUOX2 proteins was measured with Amplex Red reagent in the presence of co-expressed DUOXA2-FLAG. The activity of the mutants were standardized based on those of the WT (100%) and mock-transfected control (0%). Data are representative of three independent experiments (each performed in triplicate) with similar results. T-bars indicate standard errors of the mean.*p<0 05 vs. WT (Welchs t-test). (d) Subcellular localization analysis using HA-tagged DUOX2 constructs (WT or E520D; green fluorescence). (e) Fluorescence immunostaining under permeabilized conditions revealed that the localization of E520D-DUOX2 was consistent with DUOXA2.
To verify the pathogenicity of the DUOX2 p.E520D missense substitution detected in this case, expression experiments were conducted using HEK293 cells wherein the H2O2-producing capacity of the E520D mutant in the presence of co-expressed DUOXA2-FLAG was evaluated. We show that the E520D mutant showed complete loss of H2O2-producing activity (Fig.2c). Visualization of subcellular localization using immunofluorescence revealed substantial differences in membrane expression levels between the WT and E520D mutant (Fig.2d,e), indicating that protein localization was affected by the missense substitution.
TOKAI-IRUD-1180 with upd(3)pat: This patient, a 3-year-old girl at the time of sample submission, was the only child of healthy non-consanguineous parents. She suffered seizures beginning on day 1 after birth and symptomatic epilepsy was suspected based on abnormalities detected on an electroencephalogram. However, the seizures ceased from day 14, when oral administration of phenobarbital was initiated. She was unable to sit and had poor language understanding at the time of sample submission. ROH analysis revealed a full-length UPD of chromosome 3 [upd(3)pat], and although 40 homozygous rare missense variants were identified on chromosome 3, it was not possible to arrive at a genetic diagnosis by WES analysis.
TOKAI-IRUD-1249 with upd(2)pat: The patient, a 4-month-old girl at the time of sample submission, was the only child of healthy non-consanguineous parents. A prenatal MRI confirmed hydrocephalus. She was born by scheduled cesarean section at gestational week 34 and suffered from deafness, bilateral club feet, bilateral hip dislocation, multiple joint contractures, congenital hydrocephalus, ventricular septal defect, developmental delay, short and mildly curved femurs, a bell-shaped rib cage, and a vagina without an external opening. ROH analysis revealed a full-length UPD of chromosome 2 [upd(2)pat]. She died of aspiration pneumonia at the age of 10months, and although 34 rare homozygous missense variants and one nonsense variant were identified on chromosome 2, WES analysis did not lead to a genetic diagnosis.
One pathogenic variant of a gene included in the ACMG recommendations for reporting incidental findings was detected in one patient (TOKAI-IRUD-1150), viz, c.C6952T in BRCA2. Additionally, discordant parentchild relationships were identified in three families.
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Whole-exome analysis of 177 pediatric patients with undiagnosed diseases | Scientific Reports - Nature.com
First Gene Therapy for Adults with Severe Hemophilia A, BioMarin's ROCTAVIAN (valoctocogene roxaparvovec), Approved by European Commission (EC)
Maintains Orphan Drug Designation (ODD) in the EU Providing 10-years of Market Exclusivity
Significant BenefitOver Existing Therapies for Patients with Severe Hemophilia A in EU Based on EMA Determination of ODD
Conference Call and Webcast to be Held Wed., Aug. 24th at 8:00 pm Eastern
SAN RAFAEL, Calif., Aug. 24, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the European Commission (EC) has granted conditional marketing authorization (CMA) to ROCTAVIAN (valoctocogene roxaparvovec) gene therapy for the treatment of severe hemophilia A (congenital Factor VIII deficiency) in adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5). The EC also endorsed EMA's recommendation for Roctavian to maintain orphan drug designation, thereby granting a 10-year period of market exclusivity. The EMA recommendation noted that, even in light of existing treatments, Roctavian may potentially offer a significant benefit to those affected with severe Hemophilia A. The one-time infusion is the first approved gene therapy for hemophilia A and works by delivering a functional gene that is designed to enable the body to produce Factor VIII on its own without the need for continued hemophilia prophylaxis, thus relieving patients of their treatment burden relative to currently available therapies. People with hemophilia A have a mutation in the gene responsible for producing Factor VIII, a protein necessary for blood clotting.
It is estimated that more than 20,000 adults are affected by severe hemophilia A across more than 70 countries in Europe, the Middle East, and Africa. Of the 8,000 adults with severe hemophilia A in the 24 countries within BioMarin's footprint covered by today's EMA approval, there are an estimated 3,200 patients who will be indicated for Roctavian. BioMarin anticipates additional access to ROCTAVIAN for patients outside of the EU through named patient sales based on the European Medicines Agency (EMA) approval in countries in the Middle East, Africa and Latin America and expects additional market registrations to be facilitated by the EMA license.
"This approval in the EU represents a medical breakthrough in the treatment of patients with severe hemophilia A that expands the conversation between a patient and physician on treatment choices to now include a one-time infusion that protects from bleeds for several years," said Professor Johannes Oldenburg, Director of the Institute of Experimental Haematology and Transfusion Medicine and the Haemophilia Centre at the University Clinic in Bonn, Germany. "It is exciting to imagine the possibilities of this approved gene therapy, which has demonstrated a substantial and sustained reduction in bleeding for patients, who potentially could be freed from the burden of regular infusions."
"Roctavian approval in Europe is a historic milestone in medicine and is built upon almost four decades of scientific discovery, innovation, and perseverance. We thank the European Commission for recognizing Roctavian's value as the first gene therapy for hemophilia A, a feat that we believe will transform how healthcare professionals and the patient community think about caring for bleeding disorders," said Jean-Jacques Bienaim, Chairman and Chief Executive Officer of BioMarin. "We are grateful to the patients, investigators and community, who dedicated their time and effort to this achievement and whose aspirations provided the driving force behind making this one-time therapy a reality."
The EC based its decision on a significant body of data from the Roctavian clinical development program, the most extensively studied gene therapy for hemophilia A, including two-year outcomes from the global GENEr8-1 Phase 3 study. The GENEr8-1 Phase 3 study demonstrated stable and durable bleed control, including a reduction in the mean annualized bleeding rate (ABR) and the mean annualized Factor VIII infusion rate. In addition, the data included five and four years of follow-up from the 6e13 vg/kg and 4e13 vg/kg dose cohorts, respectively, in the ongoing Phase 1/2 dose escalation study. BioMarin has committed to continue working with the broader community and the EMA to monitor the long-term effects of treatment. The Product Information will be available shortly on the EMA website under the Medicines tab. Search for "ROCTAVIAN" and select "Human medicine European public assessment report (EPAR): Roctavian. Then select "Product Information" in the Table of Contents and then select "Roctavian: EPAR Product Information."
A Conditional Marketing Authorization (CMA) recognizes that the medicine fulfils an unmet medical need based on a positive benefit-risk assessment, and that the benefit to public health of the immediate availability on the market outweighs the uncertainties inherent to the fact that additional data are still required. BioMarin will provide further data from ongoing studies within defined timelines to confirm that the benefits continue to outweigh the risks, building on what already constitutes the largest clinical data package for gene therapy in hemophilia A. Conversion to a standard marketing authorization will be contingent on the provision of additional data from currently ongoing Roctavian clinical studies, including longer-term follow up of patients enrolled in the pivotal trial GENEr8-1, as well as a study investigating efficacy and safety of ROCTAVIAN with prophylactic use of corticosteroids (Study 270-303), for which enrollment is now complete.
Orphan drug designation is reserved for medicines treating rare (affecting not more than five in 10,000 people in the EU), life-threatening or chronically debilitating diseases. Authorized orphan medicines benefit from ten years of market exclusivity, protecting them from competition with similar medicines with the same therapeutic indication, which cannot be marketed during the exclusivity period.
BioMarin remains committed to bringing Roctavian to eligible patients with severe hemophilia A in the United States and is targeting a Biologics License Application (BLA) resubmission for Roctavian by the end of September 2022. Typically, BLA resubmissions are followed by a six-month review procedure. However, the Company anticipates three additional months of review may be necessary based on the number of data read-outs that will emerge during the procedure.
Robust Clinical Program
BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of hemophilia A. In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is also conducting a Phase 3B, single arm, open-label study to evaluate the efficacy and safety of Roctavian at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with hemophilia A (Study 270-303). Also ongoing are a Phase 1/2 Study with the 6e13 vg/kg dose of Roctavian in people with hemophilia A with pre-existing AAV5 antibodies (Study 270-203) and aa Phase 1/2 Study with the 6e13 vg/kg dose of Roctavian in people with hemophilia A with active or prior Factor VIII inhibitors (Study 270-205).
Safety Summary
Overall, single 6e13 vg/kg dose of Roctavian has been well tolerated with no delayed-onset treatment related adverse events. The most common adverse events (AE) associated with Roctavian occurred early and included transient infusion associated reactions and mild to moderate rise in liver enzymes with no long-lasting clinical sequelae. Alanine aminotransferase (ALT) elevation (113 participants, 80%), a laboratory test of liver function, remained the most common adverse drug reaction. Other adverse reactions included aspartate aminotransferase (AST) elevation (95 participants, 67%), nausea (52 participants, 37%), headache (50 participants, 35%), and fatigue (42 participants, 30%). No participants developed inhibitors to Factor VIII, thromboembolic events or malignancy associated with Roctavian.
About Hemophilia A
People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (Factor VIII levels <1%) often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population. People with hemophilia A with moderate (Factor VIII 1-5%) or mild (Factor VIII 5-40%) disease show a much-reduced propensity to bleed. Individuals with severe hemophilia A are treated with a prophylactic regimen of intravenous Factor VIII infusions administered 2-3 times per week (100-150 infusions per year) or a bispecific monoclonal antibody that mimics the activity of Factor VIII administered 1-4 times per month (12-48 infusions per year). Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.
Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A.
Conference Call and Webcast to be Held Wed., Aug. 24th at 8:00 pm Eastern
BioMarin will host a conference call and webcast to discuss the EC approval today, Wed., Aug. 24th at 8:00 pm Eastern. This event can be accessed in the investor section of the BioMarin website at https://investors.biomarin.com/events-presentations.
U.S./Canada Dial-in Number: 800-831-4163
Replay Dial-in Number: 800-645-7964
International Dial-in Number: 213-992-4616
Replay International Dial-in Number: 757-849-6722
(No ID required for live call)
Playback ID: 9184
About BioMarin
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening genetic diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of eight commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases. For additional information, please visit http://www.biomarin.com.
Forward-Looking Statements
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the number of adults across Europe, the Middle East, and Africa who are affected by severe hemophilia A; the number of adults in the countries within BioMarin's footprint covered by the EMA approval who have severe hemophilia A and are indicated for Roctavian; BioMarin anticipating additional access to Roctavian for patients outside of the EU through named patient sales based on the EMA approval in countries in the Middle East, Africa and Latin America and the expectation that additional market registrations will be facilitated by the EMA license; the potential for Roctavian to be a one-time infusion protecting patients from bleeds for several years and freeing them from the burden of regular infusions; Roctavian potentially offering a significant benefit to those affected with severe hemophilia A; Roctavian potentially transforming how healthcare professionals and the patient community think about caring for bleeding disorders; BioMarin's plans to provide further data from ongoing studies within defined timelines to confirm that the benefits of Roctavian continue to outweigh the risks; conversion of Roctavian's CMA to a standard marketing authorization; BioMarin's plans to re-submit a BLA for Roctavian to the FDA by the end of September 2022; and the duration of the FDA's review procedure of BioMarin's BLA resubmission for Roctavian. 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 results and timing of current and planned preclinical studies and clinical trials of Roctavian; additional data from the continuation of the clinical trials of Roctavian, any potential adverse events observed in the continuing monitoring of the participants in the clinical trials; the content and timing of decisions by the FDA, the EC and other regulatory authorities, including decisions to grant additional marketing registrations based on an EMA license; the content and timing of decisions by local and central ethics committees regarding the clinical trials; our ability to successfully manufacture Roctavian for the clinical trials and commercially; our ability to provide the additional data from currently ongoing Roctavian clinical studies to support the conversion from a CMA to a standard marketing authorization; and those 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, 2022 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 and ROCTAVIAN is a trademark of BioMarin Pharmaceutical Inc.
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First Gene Therapy for Adults with Severe Hemophilia A, BioMarin's ROCTAVIAN (valoctocogene roxaparvovec), Approved by European Commission (EC) -...
CAMBRIDGE, Mass., Aug. 25, 2022 (GLOBE NEWSWIRE) --Arbor Biotechnologies, Inc. a biotechnology company discovering and developing the next generation of genetic medicines, today announced that it has entered into an agreement with Acuitas Therapeutics, a leader in the development of lipid nanoparticles (LNP).
As part of the agreement, the companies will combine the optimized delivery of Acuitas' highly validated LNP technology with Arbors differentiated, proprietary CRISPR gene editing technology designed for use in vivo in patients with rare liver diseases.
We are building a robust, proprietary portfolio of genomic medicines, beginning with severe liver diseases, for which LNPs are known to provide an optimal delivery approach with their ability to efficiently target hepatocytes, limit off target toxicity and have minimal immunogenicity. We are looking forward to working with Acuitas, a leading global developer of clinically-validated LNP technology, said Devyn Smith, Ph.D., CEO, Arbor Biotechnologies. Importantly, we believe this partnership accelerates our path to the clinic, with an ability to leverage established and scalable manufacturing.
Commented Dr. Thomas Madden, President & CEO of Acuitas Therapeutics: We are excited to collaborate with Arbor on the development of novel genomic medicines for patients who currently have few, if any, therapeutic options. Arbors commitment to addressing this unmet clinical need resonates with Acuitas. We look forward to supporting their advance into the clinic.
About Arbor BiotechnologiesArbor Biotechnologies is a next-generation gene editing company focused on discovering and developing potentially curative genomic medicines. Founded by Feng Zhang, David Walt, David Scott, and Winston Yan, our proprietary discovery engine is focused on discovering genetic editing capabilities spanning knockdowns to whole gene insertions, which has enabled us to generate the most extensive toolbox of proprietary genomic editors in the industry to date. Leveraging our wholly-owned nucleases as the chassis for genetic modification, we can work backward from disease pathology to choose the optimal editing approach that specifically addresses the underlying cause of disease, resulting in a potentially curative medicine for a wider range of genetic disorders. As Arbor continues to advance its pipeline toward the clinic with an initial focus in liver and CNS disease, the Company has also secured several partnerships around gene editing and ex vivo cell therapy programs to broaden the reach of its novel nuclease technology. For more information, visit arbor.bio.
About Acuitas TherapeuticsFounded in February 2009, Vancouver-based Acuitas Therapeutics (www.acuitastx.com) is a private biotechnology company that specializes in the development of delivery systems for nucleic acid therapeutics based on lipid nanoparticles. The company partners with pharmaceutical and biotechnology companies, as well as non-governmental organizations and academic institutes to advance nucleic acid therapeutics into clinical trials and to the marketplace. The team works with partners to develop new therapies to address unmet clinical needs based on its internationally recognized capabilities in delivery technology. Acuitas Therapeutics has agreements in place with several partners to use its proprietary lipid nanotechnology in the development of COVID-19 vaccines. These include Pfizer/BioNTech for COMIRNATY, which has received full approval in the U.S. and Canada and is authorized for Emergency Use in Europe, the UK and many other countries. The Acuitas team is currently working on therapeutics focused on addressing cancer, HIV/AIDS, tuberculosis, malaria, rabies, and other serious diseases.
Contacts
MediaAmy Bonanno, Solebury Troutabonanno@soleburytrout.com914-450-0349
Investor RelationsAlexandra Roy, Solebury Troutaroy@soleburytrout.com617-221-9197
WALTHAM, Mass.--(BUSINESS WIRE)--ElevateBio, LLC (ElevateBio), a technology-driven company focused on powering transformative cell and gene therapies, today announced that it has partnered with the California Institute for Regenerative Medicine (CIRM) to advance the discovery and development of regenerative medicine as part of CIRMs Industry Alliance Program. Through the partnership, ElevateBio will provide access to high quality, well-characterized iPSC lines to academic institutions and biopharmaceutical companies that are awarded CIRM Discovery and Translational Grants. ElevateBio will also offer access to its viral vector technology, process development, analytical development, and Good Manufacturing Practice (GMP) manufacturing capabilities that are part of its integrated ecosystem built to power the cell and gene therapy industry.
This exciting partnership with CIRM reflects the novelty of our iPSC platform and recognition of our next-generation cell lines that address industry challenges and could potentially save time and costs for partners developing iPSC-derived therapeutics, said David Hallal, Chairman and Chief Executive Officer of ElevateBio. We are setting a new standard with iPSCs that can streamline the transition from research to clinical development and commercialization and leveraging our unique ecosystem of enabling technologies and expertise to help strategic partners harness the power of regenerative medicines.
With $5.5 billion in funding from the state of California, CIRM has funded 81 clinical trials and currently supports over 161 active regenerative medicine research projects spanning candidate discovery through phase III clinical trials. As part of CIRMs expansion of its Industry Alliance Program to incorporate Industry Resource Partners, this partnership will provide CIRM Awardees the option to license ElevateBios iPSC lines produced in xeno-free, feeder-free conditions using non-integrating technologies and have the ability to gain access to other enabling technologies, including gene editing, cell and vector engineering, and end-to-end services within ElevateBios integrated ecosystem, which are essential for driving the development of regenerative medicines.
About ElevateBio:
ElevateBio is a technology-driven company built to power the development of transformative cell and gene therapies today and for many decades to come. The company has assembled industry-leading talent, built state-of-the-art facilities, and integrated diverse technology platforms, including gene editing, induced pluripotent stem cells (iPSCs), and protein, vector, and cellular engineering, necessary to drive innovation and commercialization of cellular and genetic medicines. In addition, BaseCamp is a purpose-built facility offering process innovation, process sciences, and current Good Manufacturing Practice (cGMP) manufacturing capabilities. Through BaseCamp and its enabling technologies, ElevateBio is focused on growing its collaborations with industry partners while also developing its own portfolio of cellular and genetic medicines. ElevateBio's team of scientists, drug developers, and company builders are redefining what it means to be a technology company in the world of drug development, blurring the line between technology and healthcare.
ElevateBio is located in Waltham, Mass. For more information, visit us at http://www.elevate.bio, or follow Elevate on LinkedIn, Twitter, or Instagram.
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ElevateBio Partners with the California Institute for Regenerative Medicine to Accelerate the Development of Regenerative Medicines - Business Wire
PITTSBURGH--(BUSINESS WIRE)--ElevateBio, LLC (ElevateBio) and the University of Pittsburgh today announced that they have entered into a long-term strategic partnership to accelerate the development of highly innovative cell and gene therapies. Through this agreement, ElevateBio will locate one of its next BaseCamp process development and Good Manufacturing Practice (GMP) manufacturing facilities in Pittsburgh, fully equipped with its enabling technologies, including gene editing, induced pluripotent stem cell (iPSC) and cell, vector, and protein engineering capabilities. The University of Pittsburgh has long been a research powerhouse and is consistently among the top U.S. institutions in National Institutes of Health research funding.
The Richard King Mellon Foundation announced a $100 million grant to the University of Pittsburgh in November 2021 to create the Pitt BioForge BioManufacturing Center at Hazelwood Green. The grant was the largest single-project grant in the Foundation's 75-year history. The University of Pittsburgh and ElevateBio BaseCamp intend to locate the new technology-enabled process development and GMP manufacturing facility at Pitt BioForge at Hazelwood Green to further innovation in the Pittsburgh region. The new facility is expected to generate more than 170 permanent full-time jobs, 900 construction jobs, and 360 off-site support jobs.
This announcement supports the region's rise as a leader in cell and gene therapy and advances our vision of bringing an entirely new commercial manufacturing sector to the area," says Patrick Gallagher, Chancellor of the University of Pittsburgh. "The University of Pittsburgh is proud to partner with ElevateBio in this work, which will see us leveraging lessons from the labin new and exciting waysfor the benefit of human health.
To realize our vision of transforming the cell and gene therapy field for decades to come, broadening our footprint across metropolitan areas is a key priority for us, and we are thrilled that the University of Pittsburgh will be home to one of our BaseCamp facilities, said David Hallal, Chairman and Chief Executive Officer of ElevateBio. Weve identified Pittsburgh as an ideal location to extend our BaseCamp presence as it sits at the intersection of science, technology, and talent. We are grateful for the support of the Governor and County Executive as we bring the first-of-its-kind offering we have built at ElevateBio BaseCamp to advance the work of the entire biopharmaceutical industry.
Pitt Senior Vice Chancellor for the Health Sciences, Dr. Anantha Shekhar, continued by saying, We have some exceptional emerging research coming out of the University of Pittsburgh. However, the missing ingredient has been access to high-quality process science and manufacturing capabilities. As we position ourselves to become the next global hub for life sciences and biotech, we were in search of the right partner to help us realize our vision, and ElevateBios expertise and reputation in cell and gene therapy made them the perfect partner to accelerate our ability to build our biomanufacturing center of excellence.
This partnership between two national life-science powerhouses the University of Pittsburgh and ElevateBio - is a consequential step forward in realizing our shared vision to make Pittsburgh a national and international biomanufacturing destination, said Sam Reiman, Director of the Richard King Mellon Foundation. Pitt BioForge is a generational opportunity to bring extraordinary economic-development benefits to our region, and life-changing cell and gene therapies to patients - distribution that will be accelerated and enhanced by Pitts partnership with UPMC. ElevateBio could have chosen to locate its next biomanufacturing hub anywhere in the world; the fact they are choosing to come to Pittsburgh is another powerful validation of our region, and the Pitt BioForge project at Hazelwood Green.
We are excited that Pitt, working with UPMC Enterprises, has attracted ElevateBio to this region, said Leslie Davis, President and Chief Executive Officer of UPMC (University of Pittsburgh Medical Center). The companys expertise and manufacturing capabilities, combined with Pitt research and UPMCs clinical excellence, are essential to delivering the life-changing therapies that people depend on UPMC to deliver.
In addition, the Commonwealth of Pennsylvania and the County of Allegheny have provided incentive grants to ElevateBio in support of this partnership to build a biomanufacturing center and establish Pittsburgh as a premier biomanufacturing destination.
This announcement is continued verification of Pittsburgh's ability to attract new and emerging companies that provide economic opportunities in the life sciences field. The University of Pittsburgh and its medical school are a magnet for that ecosystem and along with this region's quality of life and investment in innovation, we continue to see businesses choosing Pittsburgh, said County Executive Rich Fitzgerald. The creation of the Innovation District, and the many companies that call it home, continue to provide great opportunities for talent to fill jobs across the ecosystem's pipeline. We welcome ElevateBio to our region and look forward to all that you will do here as part of this great ecosystem.
About ElevateBio:
ElevateBio is a technology-driven company built to power the development of transformative cell and gene therapies today and for many decades to come. The company has assembled industry-leading talent, built state-of-the-art facilities, and integrated diverse technology platforms, including gene editing, induced pluripotent stem cells (iPSCs), and protein, vector, and cellular engineering, necessary to drive innovation and commercialization of cellular and genetic medicines. In addition, BaseCamp in Waltham, MA, is a purpose-built facility offering process innovation, process sciences, and current Good Manufacturing Practice (cGMP) manufacturing capabilities. It was designed to support diverse cell and gene therapy products, including autologous, allogeneic, and regenerative medicine cell products such as induced pluripotent stem cells, or iPSC, and viral vector manufacturing capabilities.
Through BaseCamp and its enabling technologies, ElevateBio is focused on growing its collaborations with industry partners while also developing its own portfolio of cellular and genetic medicines. ElevateBio's team of scientists, drug developers, and company builders are redefining what it means to be a technology company in the world of drug development, blurring the line between technology and healthcare.
For more information, visit us at http://www.elevate.bio, or follow ElevateBio on LinkedIn, Twitter, or Instagram.
About the University of Pittsburgh:
Founded in 1787, the University of Pittsburgh is an internationally renowned leader in health sciences learning and research. A top 10 recipient of NIH funding since 1998, Pitt has repeatedly been ranked as the best public university in the Northeast, per The Wall Street Journal/Times Higher Education. Pitt consists of a campus in Pittsburghhome to 16 undergraduate, graduate and professional schools and four regional campuses located throughout Western Pennsylvania. Pitt offers nearly 500 distinct degree programs, serves more than 33,000 students, employs more than 14,000 faculty and staff, and awards 9,000 degrees systemwide.
For more information, please visit http://www.pitt.edu and http://www.health.pitt.edu.
About the Richard King Mellon Foundation:
Founded in 1947, the Richard King Mellon Foundation is the largest foundation in Southwestern Pennsylvania, and one of the 50 largest in the world. The Foundations 2021 year-end net assets were $3.4 billion, and its Trustees in 2021 disbursed $152 million in grants and Program-Related Investments. The Foundation focuses its funding on six primary program areas, delineated in its 2021-2030 Strategic Plan.
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ElevateBio and the University of Pittsburgh Announce Creation of Pitt BioForge BioManufacturing Center at Hazelwood Green to Accelerate Cell and Gene...
When all else fails, some patients trying to overcome alcoholism, severe depression or anxiety, and even cluster headaches, turn to psychedelic drugs, which clinical research has shown can help treat individuals with these conditions, sometimes with dramatically positive results.
But sometimes, as with any therapy, the psychedelic treatment does not work. It just takes a patient on a long strange trip.
Now, UNC School of Medicine researchers led by Dr. Bryan Roth, the Michael Hooker Distinguished Professor of Pharmacology, report that one reason for treatment disparity could be common genetic variations in one serotonin receptor.
Dr. Bryan L. Roth
Published in the journal ACS Chemical Neuroscience, the lab research in cells shows that seven variants uniquely and differentially impact the receptors response to four psychedelic drugs psilocin, LSD, 5-methoxy-N,N-dimethyltryptamine (5-MeO-DMT) and mescaline.
Based on our study, we expect that patients with different genetic variations will react differently to psychedelic-assisted treatments, said Roth, who leads the NIH Psychotropic Drug Screening Program. We think physicians should consider the genetics of a patients serotonin receptors to identify which psychedelic compound is likely to be the most effective treatment in future clinical trials.
After decades of taboo regarding potential therapeutic benefit of psychotropic drugs, there has been renewed interest and research in using such compounds to treat neuropsychiatric disorders, such as major depression disorder, because the drugs stimulate serotonin receptors in the brain. These receptors bind the neurotransmitter serotonin and other similar amine-containing molecules, helping regulate peoples mood and emotions, as well as their appetite. In particular, the 5-hydroxytryptamine receptor known as 5-HT2A is responsible for mediating how a person reacts to psychedelic drugs. However, there are several naturally occurring, random genetic variations, known as single nucleotide polymorphisms, or SNPs, that can affect the function and structure of the 5-HT2A receptor.
Site of the 5-HT2A serotonin receptor.
Roth and colleagues wanted to explore how variation in this one serotonin receptor changes the activity of four psychedelic therapies.
Graduate student Gavin Schmitz and postdoctoral researchers Manish Jain and Samuel Slocum used a series of experimental assays to measure the effect that seven different SNPs had onin vitro binding and signaling of the 5-HT2A serotonin receptor when in the presence of one of the four drugs. Their results indicated that some gene variations even ones far from the exact location where the drug binds to the receptor alter the way that the receptor interacts with the psychedelic drugs.
For example, the SNP Ala230Th had decreased response to one of the four drugs (psilocin the active metabolite of psilocybin) while the Ala447Val mutation showed only reduced effects to two of the drugs.
This is another piece of the puzzle we must know when deciding to prescribe any therapeutic with such dramatic effect aside from the therapeutic effect, Roth said. Further research will help us continue to find the best ways to help individual patients.
The National Institutes of Health and the Defense Advanced Research Projects Agency (DARPA) funded this research.
Read more here:
Genetic variants cause different reactions to psychedelic therapy - The Well : The Well - The Well
Medical treatment is shifting from a traditional symptom-based approach to one thats personalized for you.
This is especially true for cancer care, where personalized medicine is often the first step in treatment decision-making.
Prostate cancer is among the cancer types most impacted by the personalization of medicine. For prostate cancer, special disease markers are used to decide whether treatment is needed before it even begins.
Weve partnered with the Prostate Cancer Foundation (PCF) to learn more about how personalized, or precision, medicine is used for prostate cancer.
Precision medicine is used across the spectrum of prostate cancer care, from screening to treatment.
Precision medicine, or personalized medicine, is an innovative approach to tailoring disease prevention or treatment to account for differences unique to a specific patient or tumor, explains Dr. Rana McKay, a medical oncologist at the University of California San Diego and PCF-funded researcher.
For example, blood tests that detect a protein known as prostate-specific antigen (PSA) are used to screen for early signs of prostate cancer.
Cancer cells tend to release more PSA than healthy prostate cells, so elevated levels in the blood may suggest that more regular or additional types of testing are needed.
PSA can be high even if you dont have cancer, though. Observing trends in PSA levels over time is most helpful.
Taking your age and other personal characteristics into account, doctors can understand when a person with high PSA levels may have cancer versus another condition, such as prostate enlargement (benign prostatic hyperplasia) or prostatitis.
The best age to begin screening for prostate cancer can be personalized based on your risk factors. The PCF recommends:
The role of precision medicine becomes even more important during treatment. It helps doctors match the right treatment to the exact cancer that you have.
The goal of precision medicine is to target the right treatments to the right patients at the right time, McKay says.
This is important because there are several treatments and clinical trials that are [designed for] people with specific molecular changes in their tumor.
Oncologists and their teams may consider a variety of factors to evaluate the unique characteristics of a persons prostate cancer type, such as:
Some types of tests that may be used to evaluate these factors include:
Results from these tests can help healthcare professionals understand:
For instance, tumors that contain mutations in certain DNA damage repair genes may be more likely to respond to a poly adenosine diphosphate-ribose polymerase (PARP) inhibitor, such as rucaparib (Rubraca) or olaparib (Lynparza).
On the other hand, tumors that contain mutations in mismatch repair genes are more likely to respond to pembrolizumab (Keytruda).
Knowing which medication is likely to work for a specific tumor helps doctors avoid treatments that are unlikely to be effective and minimize potentially unpleasant and unnecessary side effects.
Doctors will also consider things like age and other health conditions when tailoring treatment plans to individuals.
For example, prostate cancer is known to be more aggressive and can be fatal when diagnosed in younger men, whereas men over 70 can live with the disease for many years.
However, men who are younger and otherwise healthy have the potential to live for a long time after treatment, which may also influence treatment decisions.
Understanding these factors and taking a personalized approach helps your care team determine how aggressive to be with different cancer therapies.
Personalized medicine relies on doctors finding a specific feature in a persons tumor thats known to predict response to a specific treatment.
While many advancements have been made in the field of precision medicine for prostate cancer, theres a lot left to learn.
Currently, there are only a handful of gene alterations (mutations or abnormalities) in prostate cancer that can help guide clinical decision-making and predict response to treatment.
However, if we were to actually take all possible alterations that we can target with a drug, the majority of patients likely have a genomic alteration that could potentially be targeted with a specific drug or combination of drugs, McKay estimates.
A 2015 study reported that samples from almost 90% of prostate cancer cells contained clinically actionable disease markers meaning the researchers could predict response to treatment or use the information to understand a persons diagnosis or prognosis.
The study only included tumor samples from people with advanced prostate cancer. These individuals are at the highest risk of fatal cancer and may benefit a lot from a personalized approach to treatment.
Lifestyle absolutely plays a tremendous role in prostate cancer treatment and also overcoming side effects of therapy, says McKay.
Recently, experts have started to wonder whether guiding lifestyle changes is the next step in precision medicine for various diseases and conditions.
Understanding how certain genetic features affect the likelihood that a person will develop prostate cancer can help them take steps to prevent cancer from developing in the first place.
For example, its known that diet and physical activity affect your chances of developing prostate cancer. These could be factored into a personalized prevention plan.
During treatment for prostate cancer, lifestyle plans tailored to individuals could someday help people deal with different responses to therapy and side effects.
While research hasnt yet advanced to the point that a personalized lifestyle plan can be used to help prevent or treat prostate cancer, such a future may not be far off.
Research on precision medicine for prostate cancer is continually growing.
McKay notes that there are many exciting studies on treatments, biomarkers, imaging, and other approaches on the horizon.
Shes particularly excited about the PREDICT study through the Alliance for Clinical Trials, which will launch in January of 2023.
This is a novel phase 2 biomarker-based umbrella study that uses DNA and RNA tumor profiling to guide therapy selection, she explains.
There are several other areas of prostate cancer research that one day will be used to guide personalized treatment approaches. Some of the remaining questions include:
McKay adds that having enough people from diverse backgrounds to conduct studies is what helps advance prostate cancer research and the field of precision medicine.
Participation in clinical research is really paramount for helping optimize treatment for patients, she says.
Prostate cancer care has been revolutionized by a personalized approach to treatment.
These advancements can help improve outcomes, reduce the occurrence of unnecessary side effects, and set people on the path to recovery sooner.
If you or a loved one has prostate cancer, your doctor should discuss the testing options available to help guide your personalized treatment decision-making.
Its important to engage with your healthcare clinicians to optimize treatment and ensure the best outcomes, recommends McKay.
Excerpt from:
Personalized Medicine for Prostate Cancer: What It Is and How It Works - Healthline
The fertility watchdog is pushing for the biggest overhaul of fertility laws in 30 years and discussing how to future proof any new fertility laws to make sure they can deal with current and future radical scientific advances.
Here are four of the new reproductive treatments that scientists say could be just a few years away from the clinic.
Scientists are making significant advances in the ability to grow eggs and sperm in the laboratory. The ultimate goal is to take adult skin cells, transform them into induced pluripotent stem cells that have the ability to turn into other cell types and then, using a cocktail of chemicals, coax these cells along the developmental pathway to becoming either eggs or sperm cells.
This may sound biologically improbable, but scientists have already achieved the feat in mice, producing healthy pups. In theory, a female skin cell could be used to produce a sperm cell and vice versa, which would be revolutionary.
Translating this work into human cells is not straightforward. There are still big scientific hurdles to overcome, and demonstrating safety would be a lengthy process. But there is growing confidence that this will eventually be possible and already there are companies, such the US-based Conception, aiming to bring the most recent advances to the clinic.
Genome editing is a method for making specific changes to the DNA of a cell or organism. Gene therapy, where new genes are added or faulty genes disabled in specific cells, is already used in medicine to treat genetic diseases.
Changing the DNA of an embryo goes a step further because the genetic changes would occur in every cell in the body, meaning the edits would be passed on to subsequent generations. The technique could allow people to avoid passing on heritable diseases.
However, in many cases, pre-implantation screening of embryos can achieve this goal and research has shown that gene editing tools risk producing off target changes. So there will be a very high bar for demonstrating that the technology is safe enough to be medically justified.
The last big amendment to UK fertility law came in 2015 when MPs voted for an amendment to allow a technique called mitochondrial transfer, designed to eliminate certain incurable genetic diseases. The technique involves swapping the eggs mitochondrial DNA (a tiny fraction of the total DNA, which sits outside the eggs nucleus) with that of a healthy donor.
At present, only two specific techniques are permitted, but many people would like the law made more flexible so that new techniques with the same objective could be licensed.
It is possible that in future the technique could have wider applications, for instance if faulty mitochondria were identified as a cause of infertility.
UK fertility laws regulate the use of embryos in research, and place a 14-day limit on how far into development embryos can be cultivated in the lab. However, the HFEA has no remit over so-called synthetic embryos.
This month, two teams of scientists report creating these embryo-like structures, featuring a beating heart and primitive brain, from mouse cells. The synthetic embryos look essentially the same as real embryos but do not require an egg or sperm to produce. The same scientists are trying to replicate the work in human cells, and some think new legal guidelines are required.
Separately, many scientists would like to see the 14-day rule relaxed to allow them to get a better understanding of human development, including why many pregnancies fail at an early stage.
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Four radical new fertility treatments just a few years away from clinics - The Guardian