Rare Diseases

Founded in 2018 by Professor Steve Jackson in partnership with Ahren Innovation Capital, Adrestia Therapeutics was a Cambridge-based biotech that pioneered a novel approach to…

Alcresta Therapeutics develops enzyme-based products targeting fat malabsorption in patients with rare diseases who rely on enteral feeding for nutrition. The company focuses on…

Formed to bring the portfolio of its Italian parent directly to North American prescribers, Alfasigma USA is the dedicated US commercial subsidiary of Alfasigma S.p.A., the…

AMO Pharma is building a portfolio of new medicines targeting serious and debilitating diseases, with a particular focus on rare and orphan genetic disorders. The privately held…

ANI Pharmaceuticals is a NASDAQ-listed integrated specialty pharmaceutical company (ticker: ANIP) focused on building a leading rare disease business. Operating across branded and…

Europe's pioneer in integrated therapies for rare diseases and critical care, AOP Health (trading as AOP Orphan) brings together scientific rigour, clinical development and…

AstraZeneca is one of the world's largest biopharmaceutical companies , with its global headquarters at the Cambridge Biomedical Campus in England. Founded in 1999 through the…

Pioneering second-generation complement therapeutics , Invizius is a Scottish biotech company dedicated to improving outcomes for patients with autoimmune and inflammatory…

Founded in 2010 and headquartered in Cambridge, Massachusetts, Moderna is a clinical-stage biotechnology company listed on the Nasdaq (ticker: MRNA) that has built its entire…