The FDA has approved Alnylam’s Givlaari (givosiran), an RNAi therapeutic for treatment of adults with acute hepatic porphyria (AHP), a family of ultra-rare, genetic diseases. Long-term complications of AHP can include chronic neuropathic pain, hypertension, chronic kidney disease and liver disease. “AHP can have a profound impact on the lives of patients and their families. […]

Novo Ventures and the Broad Institute of MIT and Harvard have announced the launch of the Novo Broad Greenhouse, a joint initiative to discover and propel transformative new therapies from academic science into the clinic. To drive the development of the next generation of molecular medicines, the Novo Broad Greenhouse unites three core ingredients: deep […]

A new study from the Wyss Institute at Harvard University and Harvard Medical School (HMS) reports that a single administration of an adeno-associated virus (AAV)-based gene therapy delivering combinations of three longevity-associated genes dramatically improved or completely reversed multiple age-related diseases in mice, suggesting that a systems-level approach to treating such diseases could improve overall […]

Biogen and Alkermes have announced that the FDA has approved Vumerity (diroximel fumarate) for the treatment of relapsing forms of multiple sclerosis. Biogen holds the exclusive, worldwide license to commercialize Vumerity and intends to make it available in the U.S. in the near future. The FDA approval was based on a New Drug Application (NDA) […]

Dicerna Pharmaceuticals has announced a research collaboration and licensing agreement with Roche to develop novel therapies for the treatment of chronic hepatitis B virus (HBV) infection using Dicerna’s proprietary GalXC RNAi platform technology. The collaboration will focus on worldwide development and commercialization of DCR-HBVS, Dicerna’s investigational therapy in Phase 1 clinical development. The collaboration also […]

After consulting with the FDA, Biogen plans to pursue regulatory approval for aducanumab, an investigational treatment for early Alzheimer’s disease (AD). The Phase 3 EMERGE Study met its primary endpoint showing a significant reduction in clinical decline, and Biogen believes that results from a subset of patients in the Phase 3 ENGAGE Study who received […]

CRISPR Therapeutics and Bayer today announced proposed plans whereby Casebia Therapeutics, a joint venture between CRISPR and Bayer, would operate under the direct management of CRISPR. Upon closing of the transaction, Casebia would focus on the development of its lead programs in hemophilia, ophthalmology and autoimmune diseases, with Bayer having opt-in rights for two products at […]

The FDA has approved Vertex’s Trikafta (elexacaftor/tezacaftor/ivacaftor) for the treatment of cystic fibrosis (CF) in people ages 12 years and older who have the most common CF-causing mutation. With this approval, for the first time, approximately 6,000 people with CF ages 12 years and older who have one F508del mutation and one minimal function mutation […]

Boston-based Alexion Pharmaceuticals is set to acquire Achillion, a clinical-stage biopharma focused on the development of oral small molecule Factor D inhibitors to treat complement alternative pathway-mediated rare diseases. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, […]

Cambridge-based Ra Pharmaceuticals is being acquired by UCB, a global biopharma focused on diseases of the immune system or the central nervous system. Under the terms of the agreement, Ra Pharma shareholders will receive $48 in cash for each Ra Pharma share at closing. The Boards of Directors of both companies have unanimously approved the […]