Regeneron has reported a phase 3 win in generalized myasthenia gravis, positioning the biotech to file for FDA approval of its siRNA candidate in the increasingly competitive neuromuscular disease.

Cemdisiran monotherapy, dosed subcutaneously every three months, met the primary and key secondary endpoints, showing a 2.3-point ...

The candidates are sourced from Arnatar’s next-gen RNA discovery engine, dubbed DARGER for dual-functional antisense RNA for ...

Arnatar Therapeutics, a biotechnology company pioneering RNA-based therapies for severe and underserved diseases, emerges from stealth today and announces that the U.S. Food and Drug Administration ...

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

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it ...

The milestone payment is partially funded through Sarepta’s Arrowhead stock sale as both companies push forward with ...

AbbVie has signed a collaboration and licensing agreement with US-based ADARx Pharmaceuticals, committing $335m upfront to access its small interfering RNA (siRNA) platform. The partnership gives ...

siRNA holds significant potential for gene-specific therapies, particularly in inhibiting oncogenes, but its clinical application has been hampered by delivery obstacles. To overcome these ...