REGENXBIO Inc. (RGNX) has made remarkable progress in being able to advance its pipeline with various types of gene therapies. The last time I wrote about this article it was in a Seeking Alpha ...
Sarepta Therapeutics says its RNA therapy for Duchenne muscular dystrophy (DMD) could be more effective than its existing drugs, but a dose-escalation study also dug up some new—and serious—adverse ...
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Solid Biosciences Highlights FDA Path for SGT-003 DMD Study, Shares Early Friedreich’s Ataxia Update
Solid Biosciences (NASDAQ:SLDB) used a presentation at the 2026 Emerging Outlook Biotech Summit to outline its gene ...
DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing ...
Sarepta Therapeutics SRPT posted third-quarter 2024 adjusted earnings per share (EPS) of 62 cents, which beat the Zacks Consensus Estimate of a loss of 15 cents. Earnings rose 99% year over year, ...
– U.S. FDA Study May Proceed notification enables initiation of clinical trial site activation for the FUNCTION-DMD Phase 1/2 clinical study in patients with Duchenne muscular dystrophy (DMD) – – ...
- PBGENE-DMD is a first-in-class in vivo gene editing approach for the majority of Duchenne Muscular Dystrophy patients impacted by dystrophin mutations in the most common ‘hot spot’ region between ...
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