On January 28th, Boston-based Akcea Therapeutics and Ionis Pharmaceuticals, headquartered in Carlsbad, CA,  announced positive Phase 2 study results of an oligonucleotide treatment of hypertriglyceridemia, type 2 diabetes and non-alcoholic fatty liver disease (NAFLD). The oligonucleotide, AKCEA-ANGPTL3-LRx, functions by an antisense-mediated reduction of angiopoietin-like 3 (ANGPTL3) protein in the liver. The antisense oligonucleotide is a […]

Acceleron Pharma has announced that its Phase 2 trial of sotatercept met its primary and key secondary endpoints in patients with pulmonary arterial hypertension (PAH). “We’re thrilled to report such positive topline results…” said Habib Dable, President and Chief Executive Officer of Acceleron. “PAH is a debilitating disease of high unmet medical need, so we’re […]

Biogen has announced an agreement to acquire a novel drug from Pfizer for the potential treatment of patients with behavioral and neurological symptoms across various psychiatric and neurological diseases including Alzheimer’s and Parkinson’s. The purchase of the drug, a CNS-penetrant small molecule inhibitor of casein kinase 1 (CK1), will include an upfront payment of $75 […]

Blueprint Medicines, a Cambridge biotech focused on genomically defined cancers, rare diseases and cancer immunotherapy, has announced FDA approval for Ayvakit (avapritinib), its kinase inhibitor for the treatment of a genomically defined population of patients with gastrointestinal stromal tumor (GIST), a rare, sarcoma of the GI tract. “The full approval of Ayvakit…is an incredibly exciting […]

Sarepta Therapeutics and Roche have entered into a licensing agreement providing Roche exclusive commercial rights to SRP-9001, Sarepta’s investigational gene therapy for Duchenne muscular dystrophy (DMD), outside the United States. Under the agreement, Sarepta will receive $1.15 billion in an upfront payment and an equity investment; up to $1.7 billion in regulatory and sales milestones; […]

Flagship Pioneering has launched Ring Therapeutics, an early-stage biotech developing first-in-class gene therapies using a new viral vector platform based on its groundbreaking work on the human commensal virome. Ring’s technology promises to address many of the limitations of current DNA and gene therapies, such as limited access to diverse tissues, inability to redose, risk […]