RNA, Small Interfering
Novartis Again Partners with Argo Biopharma in $5.2B+ RNAi Cardiovascular Deal
Novartis; Argo Biopharma; RNA interference (RNAi); siRNA; cardiovascular pipeline; ANGPTL3; dyslipidemia; biotech collaboration; drug development; milestone payments
Regeneron Reports Phase 3 Success for Cemdisiran in Rare Disease Generalized Myasthenia Gravis
Regeneron; Phase 3 trial; cemdisiran; generalized myasthenia gravis; rare disease; RNA drug; siRNA; Alnylam Pharmaceuticals; U.S. regulatory submission
Sarepta Shifts Focus to siRNA Platform, Lays Off 500 After Elevidys Receives Black Box Warning
Sarepta Therapeutics; siRNA platform; Elevidys; Duchenne muscular dystrophy; layoffs; black box warning; pipeline shift; acute liver injury; gene therapy; restructuring
CRISPR Therapeutics Partners with Sirius for siRNA Therapies, Expanding Beyond Gene Editing
CRISPR Therapeutics; Sirius Therapeutics; siRNA; gene editing; SRSD107; cardiovascular diseases; thrombotic disease; biotech partnerships; Factor XI; anticoagulant therapies
AbbVie Commits $335M Upfront in Landmark siRNA Deal With ADARx
AbbVie; ADARx Pharmaceuticals; siRNA therapeutics; gene silencing; neuroscience; immunology; oncology; biotech partnerships; RNA technology; drug development
Solbinsiran Demonstrates Significant Lipid Reductions in Phase 2 Trial for Mixed Dyslipidemia
Solbinsiran, siRNA, mixed dyslipidemia, Eli Lilly, PROLONG-ANG3, apolipoprotein B, LDL-C, triglycerides
Solbinsiran: Eli Lilly’s Promising ANGPTL3 Inhibitor for Lipid Management
ANGPTL3 inhibition, solbinsiran, dyslipidemia, lipid reduction, siRNA therapy
Voyager Therapeutics Halts ALS Gene Therapy Development Due to Off-Target Effects
SOD1 ALS gene therapy, Off-target effects, VY9323, siRNA payload, TRACER capsid, Preclinical data, Drug development delay
Sarepta Therapeutics and Arrowhead Pharmaceuticals Enter $825M Global Licensing and Collaboration Agreement for Multiple siRNA Programs
Sarepta Therapeutics, Arrowhead Pharmaceuticals, siRNA programs, Global Licensing and Collaboration Agreement, Rare genetic diseases, Gene therapy, RNA interference
Rona’s Sanofi-Sourced siRNA Demonstrates Early Efficacy in Dyslipidaemia Treatment
siRNA, dyslipidaemia, Rona, Sanofi, RNA interference, lipid disorders, therapeutic development