Alnylam Pharmaceuticals
NEWS
After first reporting that its investigational RNAi drug vutrisiran hit its 9-month Phase III endpoints, Alnylam announced it continues to demonstrate positive results at the 18-month mark.
Alnylam Chief Executive Officer Dr. John Maraganore, who has guided the company’s RNAi therapies from concept to commercialization, is handing over the reins of the company at the end of the year.
The experimental drug, NTLA-2001, is being developed for the treatment of transthyretin (ATTR) amyloidosis.
The top three clusters from last year’s list - Greater Boston, San Francisco Bay Area and San Diego - all stood their ground and maintained their rankings from the previous year.
While the study hit its primary efficacy endpoints, investors are likely concerned about reports of “inconsistent results” regarding reducing Uox excretion in a subtype of patients with the rare disease.
Tadataka “Tachi” Yamada, a former GlaxoSmithKline and Takeda Pharmaceutical executive, early gene therapy backer, and philanthropist, passed away on Wednesday of natural causes at the age of 76.
The FDA has already given its go-ahead to use lumasiran to treat PH1 and lower the urinary oxalate levels in pediatric and adult patients under the brand name OXLUMO.
Interim results from the Phase I study presented at the Peripheral Nerve Society Annual Meeting demonstrated that the investigational therapy, NTLA-2001, greatly reduced the disease-causing protein after a single infusion.
The U.S. FDA has had a mix of announcements this week, from drug approvals to the acceptance of NDAs and INDs. Here’s a look.
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