REGENXBIO has completed dosing in the confirmatory study of RGX-202, an AAV-delivered microdystrophin gene therapy for the treatment of Duchenne. The company is on track to initiate a Biologics License […]
Solid Biosciences reports that 33 children have now been dosed in their SGT-003 gene therapy trial for Duchenne. So far, the treatment has been generally well tolerated with signs of improved muscle […]
REGENXBIO announced new, positive 18-month functional data from patients treated with the pivotal dose in the Phase I/II portion of the AFFINITY DUCHENNE® trial. They expect to share pivotal topline data […]
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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 […]
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:
Regenxbio shared positive data from the AFFINITY DUCHENNE trial of RGX-202. In addition to robust microdystrophin expression, Regenxbio reported functional benefits at 9 and 12 months after treatment for all […]
Newport Beach, Calif. and Edmonton, Canada (May 22, 2025) – CureDuchenne Ventures announced an initial $1M investment in Entos Pharmaceuticals Inc (Entos), a biotech company that develops genetic medicines utilizing […]
