Updated results from the Alta trial show that a single infusion with the highest dose of SB-525, an investigational gene therapy, yields dose-dependent and durable increases in clotting factor VIII (FVIII). The trial, in adults with severe hemophilia A , found no bleeding episodes up to 24 weeks following the infusion.
That highest dose of SB-525 31013 vector genomes, vg/kilogram, kg led patients to reach normal FVIII activity. Participants no longer needed replacement therapy following a short preventive course post-SB-525-administration.
With these promising results, Pfizer has initiated a lead-in study (NCT03587116) to support SB-525 advancement to a Phase 3 registrational clinical trial. The six-month study will evaluate the current efficacy and safety of preventive replacement therapy in the usual care setting. It is currently recruiting participants worldwide.
The Alta trials most recent findings will be shared at the upcoming 61st Annual Meeting of the American Society of Hematology (ASH), to be held Dec. 7-10 in Orlando, Fla.
Data will be featured in a poster titled Updated Follow-up of the Alta Study, a Phase 1/2, Open Label, Adaptive, Dose-Ranging Study to Assess the Safety and Tolerability of SB-525 Gene Therapy in Adult Patients with Severe Hemophilia A.
SB-525 is a gene therapy candidate to treat hemophilia A thats being developed by Sangamo Therapeutics in collaboration with Pfizer. It consists of a DNA sequence coding for the production of a working FVIII the clotting factor missing in hemophilia A. That FVIII is carried and delivered to liver cells, where clotting factors are produced, using a harmless adeno-associated viral (AAV) vector.
The goal of the therapy is for patients to regain the ability to continuously produce the coagulation factor, and reduce or eliminate the need for FVIII replacement therapy.
The therapys safety and effectiveness for the treatment of adults with severe hemophilia A are currently being evaluated in the open-label Phase 1/2 Alta trial (NCT03061201).
The study is testing a single infusion into the vein (intravenous) of one of four ascending doses of SB-525: 91011 vg/kg; 21012 vg/kg; 11013 vg/kg; and 31013 vg/kg. Two people have been dosed per group, except for the highest dose group, which was expanded to five patients.
Updated trial data now released show the results for the two patients dosed in the third group those given 11013 vg/kg and the five individuals receiving the highest dose of 31013 vg/kg.
In the third group, a single infusion of SB-525 resulted in stable and clinically relevant increases in FVIII activity.
Stronger results were seen with SB-525s highest dose. Of the five patients treated, data were available for four. For these participants, a single infusion with the highest dose of SB-525 led to normal FVIII levels with no bleeding events reported up to 24 weeks post-administration. These individuals no longer needed replacement therapy after the initial prophylactic period of up to about three weeks after SB-525 dosing.
In addition, preliminary tests from the high-dose group indicate similar activity of SB-525-derived FVIII and the clotting factor provided by Xyntha, Pfizers recombinant therapy for hemophilia A.
As to safety, one patient had treatment-related serious adverse events, namely low blood pressure and fever, occurring about six hours after infusion. These effects resolved with treatment within 24 hours, with no loss of FVIII expression.
Some patients also showed elevated blood levels of liver enzymes(ALT, alanine aminotransferase). However, these were reported to be mild and temporary increases, which were treated in a timely manner with corticosteroids.
Dosing in the fourth group is ongoing. At the upcoming meeting, Sangamo will disclose additional analyses of the trial data, including a follow-up of approximately 4 to 11 months after treatment.
The rapid kinetics of Factor VIII expression, durability of response, and the relatively low intra-cohort variability in the context of a complete cessation of bleeding events and elimination of exogenous Factor VIII usage continues to suggest SB-525 is a differentiated hemophilia A gene therapy, Bettina Cockroft, MD, MBA, chief medical officer of Sangamo said in a press release.
We are pleased with the progress of the program toward a registrational Phase 3 study led by Pfizer, who announced it has enrolled its first patient in the 6-month Phase 3 lead-in study. We have recently completed the manufacturing technology transfer to Pfizer and initiated the transfer of the IND [investigational new drug].
Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.
Total Posts: 121
Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.
View original post here:
- Gene Therapies Make it to Clinical Trials - Discover Magazine - December 31st, 2019
- Experimental Gene Therapy Shows Promise for Preventing and Treating Lou Gehrig's Disease in Mice - BioSpace - December 31st, 2019
- 2019: The year gene therapy came of age - INQUIRER.net - December 31st, 2019
- BLA Submitted for Gene Therapy to Treat Hemophilia A - Monthly Prescribing Reference - December 31st, 2019
- First Alzheimer's Disease Gene Therapy Human Study Provided by Maximum Life Foundation, Offers 10 Free Therapies for Qualifying Patients - Benzinga - December 31st, 2019
- Year in Review: Gene Therapy Technology and a Milestone 2019 for Medical Research - News18 - December 31st, 2019
- Gene therapy to conquering hepatitis C: A decade of medical breakthroughs - Business Standard - December 31st, 2019
- What were the biggest biopharma stories of 2019? - MedCity News - December 31st, 2019
- Global Gene Therapy Market 2019 Revenue, Opportunity, Forecast and Value Chain 2025 - Market Research Sheets - December 31st, 2019
- Manufacturing: the next breakthrough in gene therapy - STAT - December 23rd, 2019
- Novartis in talks with patients upset about lottery-like gene therapy giveaway - Reuters - December 23rd, 2019
- Ring Therapeutics Launches to Expand Gene Therapy Viral Vector Options - Xconomy - December 23rd, 2019
- Gene Therapy for Sickle-Cell Anemia Looks Promisingbut It's Riddled With Controversy - Singularity Hub - December 23rd, 2019
- Making advanced therapies takes industrializing personalization - STAT - December 23rd, 2019
- Dyno Therapeutics Launches to Improve Viral Vectors for Gene Therapy - BioSpace - December 23rd, 2019
- New Gene Therapy Method May Open BRAVE New World in Parkinson's - Parkinson's News Today - December 23rd, 2019
- Pharma's gene and cell therapy ambitions will kick into high gear in 2020despite some major hurdles - FiercePharma - December 23rd, 2019
- Viewpoint: EU should take a lead in enforcing the corporate social responsibility of gene therapy manufacturers - Science Business - December 23rd, 2019
- Takeda Presents Data for Hemophilia A and B Gene Therapy Optimization - Hemophilia News Today - December 23rd, 2019
- GenScript Biotech to Host Global Forum on Cell and Gene Therapy and the Booming China Market During JPM Week - PRNewswire - December 23rd, 2019
- The gene therapy research that could save a family of four - News - The University of Sydney - December 23rd, 2019
- Waning treatment is a warning for all 'one-and-done' therapies - STAT - December 23rd, 2019
- Would you pay $1M to partake in an anti-aging gene therapy trial? - Fox Business - December 13th, 2019
- Pfizer to bring gene therapy production in-house - BioPharma-Reporter.com - December 13th, 2019
- Buyer beware of this $1 million gene therapy for aging - MIT Technology Review - December 13th, 2019
- Dr. James Wilson, a scientific pioneer, on the future of gene therapy - STAT - December 13th, 2019
- The Rise Of Patent Wars In Europe's Gene Therapy Space - Law360 - December 13th, 2019
- Pfizer cites competitiveness of inhouse gene therapy - Bioprocess Insider - BioProcess Insider - December 13th, 2019
- Investment in UK cell and gene therapy manufacturing set to continue - European Pharmaceutical Review - December 13th, 2019
- Biomanufacturing and Supply Chain Standardization Key to Success in Cell and Gene Therapy Industry Boom - BioBuzz - December 13th, 2019
- Research targets gene therapy for exudative AMD patients - Modern Retina - December 13th, 2019
- Magenta Therapeutics Demonstrates First-ever Successful Gene Therapy Transplant Without Chemotherapy in Primates Using a Single Dose of Antibody-drug... - December 13th, 2019
- GenEdit and Editas Medicine Enter into Exclusive License and Collaboration Agreement for Nanoparticle Gene Therapy Delivery - Business Wire - December 13th, 2019
- First 2 Adults with Severe Hemophilia A Respond Well to Gene Therapy BAY 2599023 in Clinical Trial - Hemophilia News Today - December 13th, 2019
- New Anti-Aging Clinical Trial Begins. For $1 Million, You Can Be a Participant. - Livescience.com - December 13th, 2019
- Watch out, Keytruda. Ferring's bladder cancer gene therapy rival has new dataand they look competitive - FiercePharma - December 13th, 2019
- Solid Biosciences Stock Crashes Over Safety Concerns About Its Gene Therapy - Barron's - November 13th, 2019
- Solid's Duchenne gene therapy trial halted after patient suffers toxicity - STAT - November 13th, 2019
- Gene Therapy in Neurology: 2019 Overview & Forecast Report - Yahoo Finance - November 13th, 2019
- How Gene Therapy Is Evolving to Tackle Complex... - Labiotech.eu - November 13th, 2019
- Lonza taps Cryoport to bolster cell and gene therapy delivery - BioPharma Dive - November 13th, 2019
- Pfizer's 'brainstorming' payment deals as gene therapies advance, exec tells Bloomberg - FiercePharma - November 13th, 2019
- Next generation cell and gene therapies: fine tuning the promise - Business Weekly - November 13th, 2019
- Vertex invests in gene therapy manufacturing - BioPharma-Reporter.com - November 13th, 2019
- Scientists are using gene therapy to treat a heart disease in dogs. Could humans be next? - 10News - November 13th, 2019
- Sangamo Announces Gene Therapy and Ex Vivo Gene-Edited Cell Therapy Data Presentations at the American Society of Hematology Annual Meeting - Business... - November 13th, 2019
- Modified Protein Enhances the Accuracy of CRISPR Gene Therapy - DocWire News - November 13th, 2019
- Triple-Gene Announces Completion of Enrollment and Dosing in Phase 1 Trial of INXN4001, First Multigenic Investigational Therapeutic Candidate for... - November 13th, 2019
- Five benefits of gene therapies - Echo Live - September 19th, 2019
- Reprogramming the Human Computer: Silicon Valley Meets Cell and Gene Therapy - BioBuzz - September 19th, 2019
- FDA approves 2nd gene therapy cancer drug from Durham's Precision Bio for clinical trial - WRAL Tech Wire - September 19th, 2019
- 7-Year-Old Receives New FDA-Approved Retina Gene Therapy - University of Michigan Health System News - September 19th, 2019
- First Patient Dosed in Trial of Fabry Gene Therapy Candidate FLT190 - Fabry Disease News - September 19th, 2019
- Bluebird bio reveals further encouraging data for CALD gene therapy - PMLiVE - September 19th, 2019
- Joseph M. Sanzari Childrens Hospital and John Theurer Cancer Center Launch Clinical Trial Evaluating Gene Therapy for Severe Sickle Cell Disease in... - September 19th, 2019
- Personalised medicine: developing gene therapies - AOP - September 19th, 2019
- A Child's Shattered Chromosomes Illustrate the Value of Supportive Therapies | DNA Science Blog - PLoS Blogs - September 19th, 2019
- Novartis says Pharmalittle: Pelosi unveils aggressive drug pricing plan - STAT - September 19th, 2019
- TrakCel partners with McKesson - BioPharma-Reporter.com - September 19th, 2019
- CMTA Grants $335,000 to Projects that Advance Therapy Development - Charcot-Marie-Tooth News - September 19th, 2019
- First patient dosed in Fabry Disease gene therapy trial - PharmaTimes - September 19th, 2019
- PPMD Awards University of Florida $1 Million for Novel Gene Therapy Approach Targeting the Heart - PRNewswire - September 19th, 2019
- Early snapshot of Adverum's eye gene therapy sparks concern about vision loss - Endpoints News - September 19th, 2019
- Type 2 Gaucher Trial of Gene Therapy PR001 May Open with Higher Dose - Gaucher Disease News - September 19th, 2019
- Therapy creates new neurons for faster stroke recovery - Futurity: Research News - September 19th, 2019
- Bluebird gene therapy shows sustained benefit in CALD study - Seeking Alpha - September 19th, 2019
- Ripple Price Forecast: XRP vs SWIFT, SEC Updates, and More - May 25th, 2019
- Cryptocurrency News: This Week on Bitfinex, Tether, Coinbase, & More - May 25th, 2019
- Cryptocurrency News: Looking Past the Bithumb Crypto Hack - May 25th, 2019
- Cryptocurrency News: XRP Validators, Malta, and Practical Tokens - May 25th, 2019
- Cryptocurrency News: Bitcoin ETFs, Andreessen Horowitz, and Contradictions in Crypto - May 25th, 2019
- Cryptocurrency News: Bitcoin ETF Rejection, AMD Microchip Sales, and Hedge Funds - May 25th, 2019
- Cryptocurrency News: What You Need to Know This Week - May 25th, 2019
- Cryptocurrency News: New Exchanges Could Boost Crypto Liquidity - May 25th, 2019
- Cryptocurrency News: Vitalik Buterin Doesn’t Care About Bitcoin ETFs - May 25th, 2019
- Bitcoin Rise: Is the Recent Bitcoin Price Surge a Sign of Things to Come or Another Misdirection? - May 25th, 2019
- Gene therapy - Wikipedia - May 14th, 2019
- Gene therapy - Mayo Clinic - May 14th, 2019
- Gene therapy | medicine | Britannica.com - May 14th, 2019
- What is Gene Therapy? - Learn.Genetics - May 14th, 2019