gene therapy

Duchenne UK and PPMD Announce $1 Million Joint Research Grant Call for Gene Therapy

PPMD is thrilled to announce our partnership with Duchenne UK to launch a joint 2020 Research Grant Call, inviting research proposals for submission and review. Up to $1 million (US) is committed to a project…

Learn More

Pfizer Shares New Phase 1b Results of Gene Therapy in Ambulatory Boys With Duchenne

PROGRESS! Pfizer announced updated Phase 1b clinical data on PF-06939926, their investigational gene therapy being developed to treat Duchenne. The preliminary data from 9 ambulatory boys with Duchenne, aged 6 to 12 indicate that the…

Learn More

Sarepta and Dyno Therapeutics Announce Agreement to Develop Next-Generation Gene Therapy Vectors

Sarepta Therapeutics and Dyno Therapeutics announced an agreement to develop next-generation gene therapy vectors for muscle diseases. PPMD is pleased to see Sarepta’s continued investment and contribution in enhancing the future of gene therapy technologies…

Learn More

Solid Biosciences Shares Update on SGT-001 Phase I/II Clinical Hold for IGNITE DMD Program

Solid Biosciences shared today that their IGNITE DMD Phase I/II clinical trial will remain on clinical hold. Solid will continue to work with the FDA to provide further data and analyses relating to their manufacturing…

Learn More

Sarepta Therapeutics Shares COVID-19 Resources

Sarepta Therapeutics has shared a new COVID-19 resource page on its website, along with a new video providing contact phone numbers for those whom may have questions about continuity of access to Sarepta sponsored clinical…

Learn More

Audentes Therapeutics Shares Community Update on Duchenne Program & COVID-19

Audentes Therapeutics has shared an update with the community regarding their Duchenne program and COVID-19. Read the Update from Audentes Dear Duchenne Patient Leaders, On behalf of the team at Audentes, we hope that you…

Learn More

Solid Biosciences Shares Letter to the Duchenne Community: COVID-19 Update

Solid Biosciences Inc. has shared an update with the community regarding the IGNITE DMD clinical hold and COVID-19. Solid’s Letter to the Duchenne Community: Letter to the Duchenne Community: COVID-19 Update Dear Duchenne Community, During…

Learn More

Pfizer Shares Update on PF-06939926 Gene Therapy Trial and COVID-19

Pfizer has shared an update regarding the PF-06939926 gene therapy trial and COVID-19. Read Pfizer’s update: Our Clinical Trials UPDATE: March 25 —To avoid adding to the demands on the healthcare system during this unprecedented…

Learn More

Sarepta Therapeutics Shares Community Update on COVID-19

Sarepta Therapeutics has provided the community with an update addressing questions from the community regarding COVID-19, including updates on current clinical trials and accessing approved therapies. Read Sarepta’s Update March 19, 2020 Dear Duchenne and…

Learn More

Solid Biosciences Provides Letter to the Duchenne Community: Data and IGNITE DMD Clinical Trial Update

Solid Biosciences Inc. provided an update on the IGNITE DMD phase I/II microdystrophin gene therapy clinical program. Solid shares that biomarker data from all three patients dosed in the 2E14 vg/kg cohort of IGNITE DMD…

Learn More

Join Our Mailing List