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.
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
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 […]
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 […]
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 […]
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 […]
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) […]
Today, we are very pleased to announce that Pfizer has acquired Bamboo Therapeutics, a gene therapy company we invested in earlier this year through CureDuchenne Ventures. The deal is valued […]