Vutrisiran receives Fast Track Designation from FDA
Alnylam has submitted a new drug application (NDA) to the FDA for vutrisiran, which could bring another treatment option to patients with ATTR amyloidosis. Vutrisiran received “Fast Track” Designation, which is a process designed to expedite the review of drugs...
First treatment for AL Amyloidosis approved in US
Daratumumab has just been granted approval by the FDA for the treatment of AL Amyloidosis in the United States. Today marks a major milestone and an achievement that has been years in the making. Janssen applied for FDA approval for...
Early Findings from Novel CRISPR Therapy Show Promise for hATTR Amyloidosis
July 7, 2021 CRISPR technology offers a cutting-edge approach to gene-editing. New clinical findings from Intellia’s Phase 1 trial have demonstrated for the first time that CRISPR can be administered by infusion to target specific cells within the body. (more…)