siRNA
Sarepta Licenses Clinical, Preclinical siRNA Candidates From Arrowhead, Enters Discovery Deal
Sarepta will pay Arrowhead Pharmaceuticals $500 million upfront, make a $325 million equity investment, plus pay $250 million over five years, and more.
Silence Therapeutics Selects Dose, Regimen for Registrational Phase III Trial of Lp(a) siRNA Drug
The investigational cardiovascular drug, zerlasiran, reduced lipoprotein(a) levels by 90 percent or greater in a Phase II trial, the company said.
Ionis is preparing to submit a new drug application for an investigational treatment for chylomicronemia syndrome that targets APOC3 mRNA.
If Novartis and others show that lowering Lp(a) improves cardiovascular outcomes, it may spur a new class of drugs that change treatment and testing paradigms.
Voyager Therapeutics Selects SOD1-ALS siRNA Development Candidate
The agent, for which the firm will file an IND in 2025, is designed to decrease SOD1 expression via an intravenously delivered capsid carrying an siRNA.
Jan 7, 2021