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 […]

Flagship Pioneering has unveiled Cambridge-based Cygnal Therapeutics which was founded in 2017 in Flagship Labs. Cygnal is the first company to develop drugs in the field of exoneural biology, a new understanding of how nerves and neural signaling drive human health and disease. The company has discovered biologically relevant pathways and advanced starting points for […]

Vertex Pharmaceuticals and RNA therapeutics developer Ribometrix, headquartered in Durham, North Carolina, have entered into a strategic collaboration to discover and develop novel RNA-targeted small molecule therapeutic candidates for serious diseases. The collaboration will combine Ribometrix’s proprietary discovery platform for identifying and optimizing small molecule therapeutics that modulate RNA function by targeting three-dimensional (3D) RNA […]