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Important Update: Sarepta Therapeutics Announces Strategic Restructuring and Pipeline Prioritization Plan to Maintain Long-term, Sustainable Growth and Provides Update on ELEVIDYS Label
CureDuchenne is sharing a community letter from Sarepta announcing a strategic company restructuring and pipeline prioritization plan. Additionally, Sarepta shared that the U.S. FDA has requested and Sarepta has agreed to include a […]
Craft Your Story
Crafting Your Story for Awareness, Connection, and Change Webinar. Watch HERE
Regulatory Update on Capricor’s Deramiocel for Duchenne
Capricor’s Deramiocel for Duchenne: Capricor Therapeutics, which received early funding from CureDuchenne, has indicated that the FDA has decided that an Advisory Committee meeting is not warranted at this time. […]
CureDuchenne’s latest investment aims to address the limitations of current AAV-delivered gene therapy treatments for Duchenne muscular dystrophy (DMD).
Our recent investment in Entos Pharmaceuticals (https://entospharma.com) aims to explore the potential of their non-viral, redosable Fusogenix PLV platform to deliver full-length dystrophin protein to all muscle groups in a […]
Sarepta pauses Duchenne gene therapy for non-ambulatory individuals after second death due to acute liver failure
Letter to the Community:
REGENXBIO WEBINAR
AFFINITY DUCHENNE®: New Phase 1/2 Interim Functional Data. Watch HERE
Capricor’s Advisory Committee meeting with FDA to be held July 30, 2025
Capricor Therapeutics, which received early funding from CureDuchenne, announced regulatory updates for their Deramiocel program to treat Duchenne. The FDA informed Capricor of its intent to hold an Advisory Committee […]
US leadership emphasizes support for rare disease drug development
CureDuchenne is pleased that one of the main themes at the recent US FDA Cell and Gene Therapy Roundtable was preserving incentives and leveraging regulatory flexibility and innovation to remove […]