The Phase III NIMBLE trial met its primary and key secondary endpoints, with cemdisiran monotherapy showing robust efficacy ...

For Sirius, the collaboration marks a major milestone in its mission to deliver innovative RNA-based therapies globally, leveraging deep expertise in siRNA design and delivery. About CTX211 ...

Gene therapy addresses genetic disorders by correcting defective genes, but delivery remains challenging. Viral vectors, while efficient, pose risks ...

Total revenues were $15.3 million for the second quarter of 2025 compared to $8.0 million in the second quarter of 2024. The increase was primarily due to variability in customers’ manufacturing ...

With effective cost control measures in place, Brii Bio remains well-capitalized, maintaining a cash position of US$289.9 million, which is sufficient to support its late-stage development plans for ...

Preliminary data are expected this year for a Phase 1/2 clinical trial testing the investigational therapy SRP-1003 in DM1.

Sarepta is facing a number of issues currently, including platform-level safety concerns, management credibility issues, etc.

Postmitotic myotubes cannot complete DNA replication following forced cell cycle reactivation. Comparative genomic ...

Sarepta Therapeutics Announces Advancement of siRNA Collaboration and Sale of Arrowhead Equity Investment - Safety review leading to dose escalation and achievement of first predetermined ...

This study presents useful findings on the molecular mechanisms driving female-to-male sex reversal in the ricefield eel (Monopterus albus) during aging, which would be of interest to biologists ...