Companies in #Hematology

Apellis Pharmaceuticals is a commercial-stage biopharmaceutical company developing targeted C3 therapies to inhibit the complement cascade in diseases of hematology, ophthalmology, and nephrology. The company has two FDA-approved C3 inhibitors: Empaveli (pegcetacoplan) for paroxysmal nocturnal hemoglobinuria (PNH), and Syfovre for geographic atrophy (GA) secondary to age-related macular degeneration.

Biotechnology company pioneering CRISPR base editing to create a new class of precision genetic medicines for serious diseases, including sickle cell disease and alpha-1 antitrypsin deficiency.

BeOne Medicines is a global oncology company that discovers and develops innovative medicines intended to improve the lives of patients worldwide. The company markets BRUKINSA (zanubrutinib), a BTK inhibitor approved for B-cell malignancies.

Global biotechnology company developing and commercializing innovative therapies for rare genetic diseases.

Bristol Myers Squibb (NYSE: BMY) is a global biopharmaceutical company with a strong oncology franchise (Opdivo, Yervoy, Reblozyl, Pomalyst, Empliciti), hematology, cardiovascular (Eliquis), and immunology (Sotyktu, Zeposia, deucravacitinib). BMS is investing in cell therapy, radiopharmaceuticals, and next-generation immuno-oncology platforms.

Global biopharmaceutical company (NYSE: BMY) focused on oncology, hematology, immunology, cardiovascular, neuroscience and fibrotic disease; marketed assets include Opdivo (nivolumab), Yervoy, Revlimid, Eliquis (apixaban with Pfizer), Pomalyst, Sprycel, Reblozyl, Sotyktu, deucravacitinib and the cardio-failure drug Camzyos; major acquisitions: Celgene ($74B, 2019), MyoKardia ($13B, 2020), Turning Point Therapeutics ($4.1B, 2022), Karuna Therapeutics ($14B, 2024), RayzeBio ($4.1B, 2024).

Late-stage biopharmaceutical company (NASDAQ: CVKD) developing tecarfarin, a novel oral reversible anticoagulant for patients with implanted cardiac devices, and CAD-1005, a 12-LOX inhibitor for heparin-induced thrombocytopenia.

Clinical-stage biopharmaceutical company developing genome-edited allogeneic cell therapies for hematologic malignancies, built on its CRISPR platform.

Disc Medicine is a clinical-stage biopharmaceutical company developing novel hematology therapeutics for patients with serious and rare blood diseases. The company's pipeline includes bitopertin (a glycine transport-1 inhibitor) for erythropoietic protoporphyria (EPP) and other hematologic disorders.

Global biopharmaceutical company specializing in plasma-derived products for rare and serious diseases.

Phase 3-stage pharmaceutical development company advancing therapies for hard-to-treat tumors and viruses. Its lead candidate, Annamycin (naxtarubicin), is a next-generation anthracycline designed to avoid multidrug-resistance mechanisms and to eliminate the cardiotoxicity common to currently prescribed anthracyclines, and is in development for relapsed or refractory acute myeloid leukemia and soft tissue sarcoma lung metastases.

Swiss-headquartered plasma products company specializing in coagulation factor therapies (wilate, Nuwiq) for hemophilia and von Willebrand disease, with operations across 40+ countries.

Orca Bio is a clinical-stage cell therapy company developing high-precision, allogeneic cell therapies for cancer and autoimmune diseases.

A biopharmaceutical company focused on discovering and developing peptide-based novel therapeutics for hematology and hematology-related conditions.

Rigel Pharmaceuticals is a commercial-stage biotechnology company focused on hematologic disorders and cancer, marketing TAVALISSE (fostamatinib) for chronic immune thrombocytopenia, REZLIDHIA (olutasidenib) for relapsed or refractory AML with an IDH1 mutation, and GAVRETO (pralsetinib) for RET fusion-positive cancers.

South San Francisco biotech built on a hub-and-spoke model that incubates antibody companies in bleeding disorders and immunology, most notably Vega Therapeutics (VGA039, sold to Incyte in 2026) and ELECTRA Therapeutics.