Vertex Pharmaceuticals has announced positive results from a small, Phase 2 proof-of-concept (POC) study of investigational drug VX-147 in patients with APOL1-mediated kidney disease. Based on these data, Vertex plans to advance VX-147 into pivotal development in Q1 2022 as a potential first-in-class therapy for the more than 100,000 patients with kidney disease mediated by […]

Dicerna Pharmaceuticals, a biopharmaceutical company focused on the development of investigational ribonucleic acid interference (RNAi) therapeutics, is being acquired by Danish drug giant Novo Nordisk for $38.25 per share in cash, which represents a total equity value of $3.3 billion and a premium of 80% to Dicerna’s closing price on November 17, 2021. Dicerna’s RNAi technology platform enables access to […]

Pfizer is seeking Emergency Use Authorization (EUA) of its investigational oral antiviral candidate, Paxlovid (PF-07321332; ritonavir), for the treatment of mild to moderate COVID-19 in patients at increased risk of hospitalizations or death. If authorized or approved, Paxlovid would be the first oral antiviral of its kind, a 3CL protease inhibitor specifically designed to combat […]

Moderna and Metagenomi, have entered into a strategic R&D collaboration focused on advancing new gene editing systems for in vivo human therapeutic applications. The collaboration will utilize Metagenomi’s novel gene editing tools and leverage Moderna’s mRNA platform, as well as lipid nanoparticle (LNP) delivery technologies, with the goal of developing curative therapies for patients with serious genetic diseases. […]

Cambridge-based Generate Biomedicines has raised $370 Million in a Series B financing to advance the development of its machine learning-powered drug generation platform. The Generate platform can rapidly generate antibodies, peptides, enzymes, cell and gene therapies to meet any therapeutic need. This approach enables Generate to create previously impossible therapeutics and to do so with unprecedented speed and […]

Flagship Pioneering has entered into a strategic partnership with the Cystic Fibrosis Foundation and Flagship’s Pioneering Medicines to develop therapeutics for cystic fibrosis. Through this agreement, the Cystic Fibrosis Foundation has committed up to $110 million to Pioneering Medicines to bring forward multiple development candidates and develop these candidates to human proof of concept. “Despite recent advances, approximately 10% […]

Bluebird Bio has signed a long-term lease to establish a biotech hub at Assembly Row in Somerville with a 61,000 square foot facility to be located at 455 Grand Union Blvd.  The company expects to move approximately 425 employees to its new headquarters in spring of 2022 where the workspace will be designed to enable a distributed […]

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. […]