Alnylam Pharmaceuticals has announced FDA approval for lumasiran (brand name Oxlumo), a RNAi therapeutic for PH1 (primary hyperoxaluria type 1), an ultra-rare genetic disease characterized by oxalate overproduction. The excess production of oxalate results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful […]

Agios Pharmaceuticals has announced that its global Phase 3 ACTIVATE trial of mitapivat met its primary endpoint. ACTIVATE is a double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of mitapivat as a potential treatment for adults with pyruvate kinase (PK) deficiency, a chronic, lifelong hemolytic anemia, who do not receive regular transfusions. Treatment with […]

Biogen and Sage Therapeutics have executed a global collaboration and license agreement to jointly develop and commercialize zuranolone (SAGE-217) for major depressive disorder (MDD), postpartum depression (PPD) and other psychiatric disorders and SAGE-324 for essential tremor and other neurological disorders. “We are excited about the potential to bring together Biogen’s leading capabilities in neuroscience with Sage’s […]

Vedere Bio, a stealth-stage company focused on advancing optogenetic therapies that are delivered to the retina to restore functional vision, has been acquired by Novartis. Shareholders in Vedere Bio received $150 million upfront and will be eligible for up to $130 million in milestone payments, for a total of $280 million. Vedere Bio was formed […]

Sartorius is expanding its presence in Massachusetts with a 40,000-square-foot North American Customer Interaction Center in Marlborough that will open in early 2021. The Customer Interaction Center initiative will enable Sartorius to scale up its operations and capabilities in the Boston biotech hub. The new site in Marlborough also will provide the capacity to support […]