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Update on Pfizer’s DAYLIGHT Gene Therapy Trial for Duchenne
The Duchenne muscular dystrophy community recently faced a profound loss—a young participant in Pfizer’s Phase 2 DAYLIGHT study (for boys 2 years to less than 4 years of age) passed away due to cardiac arrest, over a year after receiving an investigational micro-dystrophin gene therapy aimed at treating Duchenne.
Decoding Gene Therapy for Duchenne
While there is currently no cure for Duchenne muscular dystrophy, gene therapy has the potential to target the underlying genetic cause of many mono genetic diseases and could potentially benefit […]
CureDuchenne Ventures Invests in hC Bioscience and its Novel tRNA-Based Therapeutic Approach for Duchenne Muscular Dystrophy
CureDuchenne Ventures Invests in hC Bioscience and its Novel tRNA-Based Therapeutic Approach for Duchenne Muscular Dystrophy
CureDuchenne Ventures Commits $500,000 to 4D Molecular Therapeutics to Advance Next-Generation AAV Delivery of Gene Therapy
NEWPORT BEACH, Calif. – (BUSINESS WIRE) – CureDuchenne, a national venture philanthropy nonprofit focused on finding a cure for Duchenne muscular dystrophy (DMD), announced today a $500,000 investment into 4D […]
CureDuchenne Shares Pfizer’s Update Regarding Initiation of Gene Therapy Trial
Today, Pfizer announced that the first patient was dosed in their mini-dystrophin gene therapy trial. This is an exciting day for the Duchenne community. Pfizer has the experience to conduct […]
Phase 1b clinical research study of mini-dystrophin for DMD
CureDuchenne is proud that another one of our funding projects is entering clinical trial. Pfizer Inc. is planning a Phase 1b, Multicenter, Open-label, Single Ascending Dose Study to Evaluate the […]
CureDuchenne Ventures Seed Funding to Exonics Helped Advance CRISPR Research and Attract Series A Financing to Accelerate Development of Lead Gene Editing Program in Duchenne
Dr. Eric Olson to Provide CRISPR/Cas9 and Gene Therapy Update During November 16 Webinar We are delighted that Exonics Therapeutics has closed a $40 million Series A financing with […]
Sarepta Therapeutics Announces its Partner, Genethon, Published New Micro-Dystrophin Gene Therapy Data in Nature Communications
Data show for the first time a systemic therapeutic effect in DMD dogs using a rAAV2/8 micro-dystrophin gene therapy approach without immunosuppressive treatment CAMBRIDGE, Mass., July 27, 2017 (GLOBE NEWSWIRE) […]