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 ...
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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 therapy-focused pipeline and discuss recent regulatory interactions around its lead ...
PepGen (PEPG) announced that based on the levels of dystrophin protein measured in the 10 mg/kg cohort of its CONNECT1-EDO51 study investigating PGN-EDO51 in Duchenne muscular dystrophy patients ...
PBGENE-DMD is Precision's wholly-owned, first-in-class in vivo gene editing program utilizing a gene excision approach intended to permanently correct the dystrophin gene in patients with mutations ...
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 ...
Capricor Therapeutics, Inc. (CAPR) has a significant catalyst that is rapidly approaching, which could change the scope of this biotech. This would be regarding the release of top-line data from ...
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, ...
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