September 14, 2022

WATCH: SRP-9001 Data Update from Studies 101, 102, and 103 (August 2022)

On August 24, 2022, PPMD was joined by Sarepta Therapeutics for a community webinar to present a SRP-9001 gene therapy data update from Studies 101, 102, and 103, following their recent presentation at a medical…

Learn More

September 1, 2022

Pfizer Shares Update on DAYLIGHT Phase 2 Gene Therapy Trial

Pfizer has issued a community letter announcing that its Phase 2 DAYLIGHT clinical trial evaluating the company’s investigational mini-dystrophin gene therapy product (fordadistrogene movaparvovec) has opened a first trial site in Australia. Pfizer shares that…

Learn More

July 29, 2022

Sarepta Announces Intent to Submit an Accelerated Approval Biologics License Application for SRP-9001

Sarepta Therapeutics today announced its intention to file for FDA approval of SRP-9001 this fall using the FDA’s accelerated approval pathway. SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle…

Learn More

July 6, 2022

Sarepta Shares New Functional Data From SRP-9001 Duchenne Gene Therapy Clinical Development Program

Sarepta Therapeutics has shared new functional data demonstrating functional improvements across multiple studies from the clinical development program for SRP-9001 including one-year functional results compared to an external control group, from Study SRP-9001-103 (ENDEAVOR) in…

Learn More

June 24, 2022

Breaking News! PPMD Awards $2 Million to ACTION Network to Revolutionize Duchenne Cardiac Care

PPMD is pleased to announce a $2 million grant to be awarded to the Advanced Cardiac Therapies Improving Outcomes Network (ACTION), a learning health system focused on children and adult congenital heart disease patients at…

Learn More

May 17, 2022

Nationwide Children’s Announces Restoration of Full-Length Dystrophin Using Dup 2 Gene Therapy Approach

PPMD is very excited to see promising results shared from Nationwide Children’s scAAV9.U7.ACCA gene therapy trial, a Phase 1/2 study aiming to induce exon skipping for individuals with a duplication of exon 2 to restore full-length…

Learn More

May 16, 2022

WATCH: PPMD & Pfizer Discuss Updates on CIFFERO Phase 3 Gene Therapy Trial

Recently Pat Furlong, PPMD’s founder and CEO, sat down with Dr. Beth Belluscio, Pfizer’s Global Clinical Lead of Rare Neurological Disorders, to discuss Pfizer’s learnings from previous gene therapy studies and updates on the CIFFERO…

Learn More

May 4, 2022

Regenxbio Announces Delay in Timeline for Initiation of First Participant Dosed in Affinity Duchenne Trial

REGENXBIO today announced that it will delay the initiation of dosing in the Phase I/II AFFINITY DUCHENNETM trial studying RGX-202 in participants with Duchenne, due to an unexpected observation in the final stages of manufacturing…

Learn More

April 28, 2022

Pfizer Announces Clinical Hold Lifted for its Mini-Dystrophin Gene Therapy Program

Pfizer has issued a community letter sharing that the FDA has lifted the clinical hold on the company’s Phase 3 ambulatory trial (CIFFREO) for their investigational mini-dystrophin gene therapy product for Duchenne. Pfizer has addressed…

Learn More

April 27, 2022

Solid Biosciences Shares Update on Duchenne Gene Therapy Pipeline Programs

Today Solid Bio shared an update on their gene therapy programs for SGT-001 and SGT-003. SGT-001 is Solid’s AAV9 microdystrophin gene therapy investigational product that is currently being evaluated in boys with Duchenne. SGT-003 is…

Learn More