CureDuchenne was an early investor in Avidity Biosciences in 2018, and congratulates the company for receiving Breakthrough Therapy Designation from the US FDA for delpacibart zotadirsen (abbreviated as del-zota), their exon 44-skipping agent […]
Precision BioSciences is developing a novel gene editing approach for individuals with dystrophin mutations in the “hot spot” region of exons 45-55, with the goal of submitting applications later this […]
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 […]
Capricor Therapeutics announced that it has received a Complete Response Letter indicating that the FDA has completed the review of Deramiocel for Duchenne cardiomyopathy, but would be unable to approve […]
Edgewise Therapeutics, which received early funding from CureDuchenne, has reported positive results in its sevasemten program for Becker and Duchenne muscular dystrophies. New open-label data in Becker from the MESA trial demonstrated […]
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. […]
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 […]
Letter to the Community:
