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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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Entrada Reports Positive Topline Results from Phase 1/2 ELEVATE-44-201 Study in Duchenne

Entrada Therapeutics has announced positive topline data from Cohort 1 of the double-blind, placebo-controlled, multiple ascending dose portion of the Phase 1/2 ELEVATE-44-201 study evaluating ENTR-601-44 in ambulatory participants ages 4–20 living with Duchenne muscular…

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Sarepta Announces Plans to Submit sNDAs for AMONDYS 45 and VYONDYS 53

Today, Sarepta Therapeutics announced plans to submit supplemental New Drug Applications (sNDAs) to the U.S. Food and Drug Administration (FDA) for AMONDYS 45 and VYONDYS 53, the company’s exon-skipping therapies for the treatment of individuals…

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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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Capricor Therapeutics Announces Positive Data from HOPE-3 Study of Deramiocel

Capricor Therapeutics has announced additional analyses and new functional outcomes data from the Phase 3 HOPE-3 clinical trial of Deramiocel in Duchenne. Deramiocel is the company’s investigational cell therapy for the treatment of Duchenne cardiomyopathy….

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Solid Biosciences Shares Interim Positive Update on Phase 1/2 INSPIRE DUCHENNE Trial

Solid Biosciences Inc. has shared updated positive interim data from the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial of SGT-003. SGT-003 is a gene therapy candidate for the treatment of Duchenne that delivers a microdystrophin…

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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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Edgewise Announces Positive Long-Term Sevasemten Data

Edgewise Therapeutics, Inc. has shared long-term data from its MESA open-label extension study of sevasemten in Becker muscular dystrophy. Sevasemten is an orally administered small molecule inhibitor designed to protect muscle against contraction-induced damage in…

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