Companies in #Genetic Diseases

Be Biopharma (Be Bio) is a private clinical-stage biotechnology company pioneering engineered B cell medicines (BCMs), a new modality that uses genetically modified B cells to deliver durable, redosable protein therapeutics. Its lead clinical program was in hemophilia B but was discontinued in June 2026 after a strategic pipeline review; the company is refocusing its platform on other genetic disease and oncology indications beyond CAR-T.

Swedish Orphan Biovitrum (Sobi) is a global specialty biopharmaceutical company focused on rare diseases, with a portfolio spanning hematology, immunology, and specialty care. The company markets hemophilia treatments (Eloctate, Alprolix), immunology products (Kineret, Gamifant), and specialty therapeutics, and is expanding its rare-disease pipeline through both internal R&D and acquisitions.