siRNA
Voyager Therapeutics Exploring Alternate Payload in SOD1-ALS Gene Therapy Program
New three-month data from a study of nonhuman primates underpins the biotech firm's decision to amend the small interfering RNA program.
FDA Clears Aro Biotherapeutics' siRNA Drug IND for Late-Onset Pompe Disease
The company has already kicked off an open-label Phase I trial of ABX1100 in Canada following positive results from a study of healthy volunteers.
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.
Apr 4, 2024
Jan 7, 2021