gene therapy

October 22, 2026

Fellowship Update: Universal Antibody Screening Test for AAV Gene Therapy

As gene therapy research continues to advance, understanding who may be eligible for a particular treatment remains an important challenge for the Duchenne and Becker community. PPMD is committed to supporting research that addresses these…

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Solid Biosciences Shares Updates on SGT-003

Solid Biosciences has shared a new letter to the Duchenne community providing updates on the development of SGT-003, the company’s investigational gene therapy for Duchenne. Solid shared that the ongoing INSPIRE DUCHENNE Phase 1/2 clinical…

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GEMMABio Announces Duchenne Muscular Dystrophy Program, Shares Preclinical Data for Investigational Gene Therapy for Duchenne

Today, Gemma Biotherapeutics (GEMMABio), a clinical‑stage, global, genetic therapeutics company, shared information about the company’s Duchenne muscular dystrophy program, presenting preclinical data supporting candidate declaration for GB703, a novel, investigational gene therapy for the treatment…

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REGENXBIO Shares Topline Results from Pivotal Phase III AFFINITY DUCHENNE® Study of RGX-202

REGENXBIO Inc. has announced positive topline and interim functional data from the pivotal Phase III portion of the Phase I/II/III AFFINITY DUCHENNE® trial of RGX-202, an investigational gene therapy being developed for individuals with Duchenne…

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Solid Biosciences Doses First Participant in Phase 3 IMPACT DUCHENNE Clinical Trial Evaluating SGT-003 in Duchenne

Solid Biosciences Inc. has shared that the first participant has been dosed in IMPACT DUCHENNE, the company’s multi-country, placebo-controlled, randomized, double-blind, Phase 3 clinical trial investigating SGT-003 for the treatment of Duchenne muscular dystrophy. SGT-003…

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Sarepta Announces Initiation of Screening and Enrollment for ENDEAVOR Cohort 8 in Non-Ambulatory Individuals Living with Duchenne

Sarepta Therapeutics, Inc. has announced screening and enrollment are underway in Cohort 8 of the company’s ENDEAVOR study. ENDEAVOR is an open-label, Phase 1b study assessing the expression and safety of ELEVIDYS in multiple cohorts…

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REGENXBIO Shares Positive Interim Data from Phase I/II AFFINITY DUCHENNE Trial of RGX-202

REGENXBIO has announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202, an investigational gene therapy being developed for individuals with Duchenne muscular dystrophy. RGX-202 is designed to deliver microdystrophin via…

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January 26, 2026

Sarepta Shares Topline Three-Year EMBARK Data for ELEVIDYS in Ambulatory Duchenne Patients

Sarepta Therapeutics, Inc. has announced positive topline three-year functional results from Part 1-treated patients in EMBARK, the company’s global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals living with Duchenne. According…

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November 25, 2025

Sarepta Announces FDA Approval to Begin ENDEAVOR Cohort 8 Dosing in Non-Ambulatory Individuals Living with Duchenne

Sarepta Therapeutics, Inc. has shared that the U.S. Food and Drug Administration (FDA) has approved dosing in Cohort 8 of ENDEAVOR, an open-label, Phase 1b study assessing the expression and safety of ELEVIDYS in multiple…

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November 14, 2025

FDA Approves New Safety Warning and Revised Indication for ELEVIDYS

Today the FDA announced a significant update to the labeling and indication of ELEVIDYS for the treatment of individuals living with Duchenne following reports of death based on acute liver failure (ALF) in non-ambulatory patients…

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