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Top CureDuchenne News

Capricor says FDA may not convene expert panel for cell therapy
United States · United States

Capricor says FDA may not convene expert panel for cell therapy

100% Left coverage: 1 sources
Capricor Therapeutics said on Tuesday the U.S. Food and Drug Administration may not seek its outside experts' advice when deciding on the company's cell therapy for a heart condition associated with Duchenne muscular dystrophy.See the Story
Kymera Therapeutics Announces Pricing of $250 Million Public Offering
United States · United States

Kymera Therapeutics Announces Pricing of $250 Million Public Offering

WATERTOWN, Mass., June 26, 2025 (GLOBE NEWSWIRE) — Kymera Therapeutics, Inc. (NASDAQ: KYMR), a clinical-stage biopharmaceutical company advancing a new class of oral small molecule degrader medicines for immunological diseases, today announced the pricing of its underwritten public offering of $250.8 million of shares of its common stock and, in lieu of common stock to […] The post Kymera Therapeutics Announces Pricing of $250 Million Public Off…See the Story

Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne

60% Center coverage: 5 sources
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun 15, 2025-Artificial IntelligenceSee the Story

FDA Meeting Gives Window Into Gene Therapy Field’s Angst

100% Center coverage: 2 sources
Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a regulatory forum Thursday.FDA · United StatesSee the Story

REGENXBIO REPORTS NEW POSITIVE FUNCTIONAL DATA FROM PHASE I/II AFFINITY DUCHENNE® TRIAL OF RGX-202

100% Left coverage: 1 sources
RGX-202 demonstrating consistent evidence of positively changing disease trajectory for DuchenneAll dose level 2 participants exceeded external natural history controls on all functional measuresBiomarker data demonstrate consistent, robust microdystrophin expression and transduction levels across all treated agesOne new participant aged…See the Story

PepGen to discontinue development of experimental Duchenne therapy; shares drop

100% Center coverage: 1 sources
(Reuters) -Drug developer PepGen said on Wednesday it will discontinue development of its experimental therapy for Duchenne muscular dystrophy (DMD) after it failed to boost production of a protein in a mid-stage study. Shares of the company were down 9.5% at $1.43 in extended trading. The therapy PGN-EDO51 did not achieve target levels of dystrophin production. Dystrophin is a protein whose absence leads to muscle weakening in patients with DMD…See the Story

Satellos’ DMD treatment shows efficacy signals in adult patients

Satellos said that following the Phase I data, it will be advancing its DMD small molecule to a Phase II trial.The post Satellos’ DMD treatment shows efficacy signals in adult patients appeared first on Clinical Trials Arena.Irvine, California · IrvineSee the Story

CureDuchenne Ventures Invests in Entos Pharmaceuticals

Entos Pharmaceuticals, an Edmonton, Canada-based biotech company that develops genetic medicines, received an investment from CureDuchenne Ventures. The amount of the deal was not disclosed. The company intends to use the funds to create a muscle-targeting therapeutic to deliver full-length dystrophin for the treatment of Duchenne muscular dystrophy (DMD).  Led by CEO John Lewis, Entos […] The post CureDuchenne Ventures Invests in Entos Pharmace…Technology · Newport BeachSee the Story

CureDuchenne Appoints Brenda Wong, MD, as Chief Medical Advisor to Advance Research and Improve Care for Individuals with Duchenne and Becker

100% Center coverage: 3 sources
Newport Beach, Calif., May 21, 2025 — CureDuchenne, a global nonprofit committed to finding and funding a cure for Duchenne muscular dystrophy, proudly announces the appointment of Brenda Wong, MD, as its new Chief Medical Advisor, effective January 5, 2026. A nationally recognized authority in neuromuscular medicine and clinical research, Dr. Wong has devoted her career to compassionate, patient-centered care for those living with Duchenne and …Maryland · MarylandSee the Story

Entrada receives authorization to start exon 44 skipping trial in Europe

CureDuchenne provided early funding to Entrada Therapeutics.  Entrada has received regulatory authorization in Europe to initiate a Phase 1/2 clinical trial of ENTR-601-44 in ambulatory individuals with Duchenne amenable to skipping exon 44.  The study, which will run in the UK and the EU, will start in the 2nd quarter of 2025.  Entrada is also planning a Phase 1/2 for ambulatory individuals with Duchenne amenable to skipping exon 45, which will…United Kingdom · United KingdomRead Article

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