gene therapy

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

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

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

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

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

Astellas Gene Therapies Announces plans to discontinue development of Duchenne gene therapy programs

Last night, based on recent preclinical data, Astellas Gene Therapies announced that the research and development of gene therapy programs AT702, AT751 and AT753 for Duchenne Muscular Dystrophy have been terminated. • AT702 was designed…

Learn More

Solid Biosciences Reports 2-Year Efficacy and Safety Data from Ongoing Phase I/II IGNITE DMD Clinical Trial of SGT-001

Solid Biosciences has shared an update from IGNITE DMD, an ongoing Phase I/II clinical trial evaluating its gene therapy candidate SGT-001 in participants with Duchenne. In the announcement, Solid reports positive two-year efficacy and safety…

Learn More

January 13, 2022

Solid Provides Updates on IGNITE DMD Study and SGT-003 Pre-Clinical Data

Solid Biosciences has shared an update from IGNITE DMD, an ongoing Phase I/II clinical trial evaluating its gene therapy candidate SGT-001 in participants with Duchenne, as well as developments for the company’s next generation gene…

Learn More

January 11, 2022

Sarepta Announces Topline Results from Part 2 of Study SRP-9001-102 Evaluating Duchenne Gene Therapy Candidate

Sarepta Therapeutics has shared an update from Part 2 of Study SRP-9001-102, an ongoing clinical trial evaluating Sarepta’s gene therapy candidate SRP-9001 in participants with Duchenne. SRP-9001 is an investigational gene transfer therapy intended to…

Learn More

January 6, 2022

REGENXBIO Announces FDA Clearance of IND for Clinical Trial of RGX-202 Gene Therapy Candidate for Duchenne

PPMD is excited to share that REGENXBIO has announced that they have received clearance from the FDA on their Investigational New Drug (IND) application for RGX-202, a micro-dystrophin gene therapy product. With this clearance REGENXBIO…

Learn More

Join Our Mailing List