RNAi therapeutics company Alnylam has announced positive topline results from its Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), a rare heart condition that is both progressive and fatal. Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by […]

Sarepta Therapeutics has received FDA approval of an expansion to the labeled indication for Elevidys, a single-dose, adeno-associated virus (AAV)-based gene transfer therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene […]

Moderna has announced that the FDA has approved mRESVIA (mRNA-1345), an mRNA respiratory syncytial virus (RSV) vaccine, to protect adults aged 60 years and older from lower respiratory tract disease caused by RSV infection. The approval was granted under a breakthrough therapy designation and marks the second approved mRNA product from Moderna which has filed […]

Biogen has agreed to acquire Human Immunology Biosciences (HI-Bio) for $1.15 billion upfront and up to $650 million in potential milestone payments. HI-Bio is a privately-held clinical-stage biotech focused on targeted therapies for patients with severe immune-mediated diseases. The transaction is subject to customary closing conditions, including receipt of necessary regulatory approvals and is currently […]

Vaccine developer Novavax has entered into an agreement with Sanofi that includes: a co-exclusive license to co-commercialize Novavax’s current COVID-19 vaccine worldwide; a sole license to Novavax’s COVID-19 vaccine for use in combination with Sanofi’s flu vaccines; and a non-exclusive license to use Novavax’s COVID-19 vaccine in combination with non-flu vaccines. Outside of the collaboration, each […]

Mariana Oncology of Watertown has announced it will be acquired by Novartis for $1 billion upfront and up to $750 million in potential milestone payments. The acquisition brings together Mariana Oncology’s innovative radiopharmaceutical pipeline and platform with the proven clinical development and commercialization expertise of Novartis. Mariana Oncology has developed a robust portfolio of novel […]

Waltham-based Deciphera Pharmaceuticals has announced it is being acquired by Japan’s ONO Pharmaceutical Company for $2.4 billion. Deciphera is focused on discovering, developing, and commercializing new cancer medicines and brings specialized R&D and development capabilities in kinase drug discovery, well-established commercial and sales platforms in the United States and Europe, and global clinical development capabilities. The acquisition is […]

Vertex has announced it plans to acquire Seattle biotech Alpine Immune Sciences for $65 per share or approximately $4.9 billion in cash. Alpine is focused on discovering and developing innovative, protein-based immunotherapies. The transaction was unanimously approved by both the Vertex and Alpine Boards of Directors and is anticipated to close later this quarter. Alpine’s […]

Watertown clinical-stage biopharma Neumora Therapeutics’ Phase 1 trial of NMRA-266 has been placed on clinical hold by the FDA based on pre-clinical data showing convulsions in rabbits. Approximately 30 participants have been dosed in the Phase 1 study, with no evidence of convulsions observed in any participant. “We are disappointed with the unanticipated safety findings […]

Amylyx Pharmaceuticals has started a process with the FDA and Health Canada to voluntarily discontinue the marketing authorizations for its ALS drug and immediately remove the product from the market in both the U.S. and Canada. The drug is sold under the name Relyvrio in the U.S.and is also known as AMX0035. The decision is […]