Cambridge-based Amylyx Pharmaceuticals has submitted a New Drug Application (NDA) to the FDA for AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). “We will continue to work closely with the FDA throughout the review process to move AMX0035 toward a potential regulatory approval as quickly and efficiently as possible,” said Tammy Sarnelli, Global Head […]

Vertex Pharmaceuticals has joined forces with California-based Mammoth Biosciences, co-founded by CRISPR pioneer and Nobel Laureate Jennifer Doudna, to develop in vivo gene-editing therapies for two genetic diseases using Mammoth’s next-generation CRISPR systems. Driven by its unique protein discovery engine, Mammoth’s CRISPR platform consists of a proprietary toolbox of novel, ultracompact Cas enzymes, including Cas14 and […]

Gavi, the Vaccine Alliance has exercised its option to purchase an additional 176.5 million doses of the Moderna COVID-19 vaccine for the COVAX Facility. Of these additional doses, 116.5 million doses are expected to be delivered in the first quarter of 2022 and 60 million doses are expected to be delivered in the second quarter of 2022. […]

Watertown biotech Neumora Therapeutics, a clinical-stage company pioneering precision medicines for brain diseases, has announced its launch. Neumora was founded as a response to the lack of targeted, effective medicines for brain diseases and the high neuroscience drug development failure rate and has raised over $500 million, including a $100 million equity investment from Amgen. […]