hematology
(5)
Kyowa Kirin, Inc. - U.S.
A Japanese fermentation lineage became a U.S. rare-disease business by following biology that larger markets often overlook. Now Kyowa Kirin is testing whether patient-scale focus can coexist with manufacturing-scale ambition.
Cayuga Biotech
Cayuga Biotech is a clinical-stage biotech developing first-in-class therapeutics that stop life-threatening bleeding by accelerating the body's own clotting cascade. Its lead candidate, CAY001, is a synthetic polyphosphate-silica nanoparticle (polyP-SNP) designed to work where conventional hemostatic agents fall short - non-compressible hemorrhage from trauma, surgery, blood thinners and inherited bleeding disorders. Founded on research from UC Santa Barbara, the company harnesses polyphosphate, a natural cofactor stored in platelets, to normalize thrombin production and speed clot formation without slowing clot breakdown.
Nurix Therapeutics
Nurix Therapeutics is a clinical-stage biopharmaceutical company in San Francisco pioneering targeted protein degradation - medicines that destroy disease-causing proteins rather than merely blocking them. Built on its DELigase discovery engine that harnesses the cell's ubiquitin-proteasome system and DNA-encoded library screening, Nurix is advancing a pipeline of degraders and degrader antibody conjugates for cancer and autoimmune disease, led by the BTK degrader bexobrutideg (NX-5948). The company is partnered with Roche, Sanofi, Gilead, and Pfizer (via Seagen) and trades on Nasdaq under NRIX.
Protagonist Therapeutics
Protagonist Therapeutics is a Newark, California clinical-stage biopharmaceutical company that turns peptides - short chains of amino acids long dismissed as too fragile to be drugs - into stable, often orally available medicines. Its proprietary peptide discovery and engineering platform powers a pipeline spanning hematology, inflammation/immunology and metabolic disease, anchored by rusfertide (a hepcidin mimetic for polycythemia vera under FDA Priority Review) and icotrokinra, an oral IL-23 receptor blocker partnered with Johnson & Johnson that reached the U.S. market in 2026. Publicly traded on Nasdaq as PTGX, the company pairs internal discovery with high-value pharma partnerships.
Star Therapeutics
Star Therapeutics is a South San Francisco clinical-stage biotech building first-in-class antibody medicines for serious diseases, with a 'pipeline-in-a-product' approach. Its lead asset VGA039, developed through subsidiary Vega Therapeutics, is a once-monthly subcutaneous monoclonal antibody targeting Protein S, now in Phase 3 for von Willebrand disease.