Albireo Pharma
NASDAQ:ALBOAlbireo Pharma, Inc. is a clinical stage biopharmaceutical company, which engages in the development and commercialization of novel bile acid modulators for the treatment of orphan pediatric liver diseases and gastrointestinal disorders. Its pipeline products include Odevixibat, A3384, and Elobixibat. The company was founded by Jan Peter Mattsson and Per-Göran Gillberg on December 8, 2003 and is headquartered in Boston, MA.
Centessa Pharmaceuticals
NASDAQ:CNTACentessa Pharmaceuticals plc, a clinical-stage pharmaceutical company, discovers, develops, and delivers medicines to patients. Its pipeline products include SerpinPC, an activated protein C inhibitor, which is in Phase IIa clinical development for the treatment of hemophilia A and B; and ORX750, an orally administered OX2R agonist for the treatment of NT1 with potential expansion into other sleep disorders. The company also develops LB101, a PD-L1xCD47 LockBody, which is designed to selectively drive potent CD47 and CD3 effector function activity while avoiding systemic toxicity; ZF874 in alpha-1 antitrypsin deficiency, a dual-STAT3/5 degrader program in acute myeloid leukemia (AML); MGX292, a protein-engineered variant of human bone morphogenetic protein 9 (BMP9) for the treatment of pulmonary arterial hypertension (PAH); and OX2R Agonists compounds are currently in development for the treatment of narcolepsy, including TAK-861. In addition, its products pipeline comprises CBS001, a neutralizing therapeutic mAb to the inflammatory membrane form of LIGHT for inflammatory / fibrotic diseases; and CBS004, a therapeutic mAb targeting BDCA-2 for the potential treatment of autoimmune diseases, as well as earlier-stage preclinical assets, including ORX750, an orally administered, selective orexin receptor-2 (OX2R) agonist for the treatment of narcolepsy and other sleep disorders; and discovery-stage programs in certain other disease areas. Centessa Pharmaceuticals plc was incorporated in 2020 and is based in Altrincham, the United Kingdom.
IDEAYA Biosciences
NASDAQ:IDYAIDEAYA Biosciences, Inc., a synthetic lethality-focused precision medicine oncology company, discovers and develops targeted therapeutics for patient populations selected using molecular diagnostics in the United States. The company's products in development include IDE196, a protein kinase C inhibitor that is in Phase 2/3 clinical trials for genetically defined cancers having GNAQ or GNA11 gene mutations; IDE397, a methionine adenosyltransferase 2a inhibitor that is in Phase 1/2 clinical trials for patients with solid tumors having methylthioadenosine phosphorylase gene deletions, such as non-small cell lung, bladder, gastric, and esophageal cancers; IDE161, a poly ADP-ribose glycohydrolase inhibitor that is in Phase 1 clinical trial to treat tumors with homologous recombination deficiency (HRD), and other genetic or molecular signatures; GSK101, a Pol Theta Helicase inhibitor that is in Phase 1 clinical trial for the treatment of tumors with BRCA or other homologous recombination, and HRD mutations; and Werner Helicase inhibitors for tumors with high microsatellite instability. It offers preclinical research programs focused on pharmacological inhibition; DECIPHER dual CRISPR library for synthetic lethality target and biomarker discovery; and INQUIRE chemical library and HARMONY machine-learning engines to enhance its drug discovery platform. The company has strategic alliances with GlaxoSmithKline, Pfizer Inc., Novartis International Pharmaceuticals Ltd., Amgen Inc., Gilead Sciences, Inc., Cancer Research UK, and the University of Manchester. IDEAYA Biosciences, Inc. was incorporated in 2015 and is headquartered in South San Francisco, California.
Nkarta
NASDAQ:NKTXNkarta, Inc., a clinical-stage biopharmaceutical company, develops and commercializes natural killer cell therapies for cancer and autoimmune disease treatment. The company's lead product candidate is NKX019, a chimeric antigen receptor-natural killer (CAR NK) targeting the CD19 antigen that is in Phase 1 clinical trial for the treatment of relapsed/refractory (r/r) non-hodgkin lymphoma, as well as for lupus nephritis. It also develops NKX101, a CAR NK product candidate targeting cells that display NKG2D ligands, which is in Phase I clinical trial for the treatment of r/r acute myeloid leukemia or higher risk myelodysplastic syndromes, as well as for solid tumors. In addition, the company develops NKX070, targeting the CD70 tumor antigen to treat solid and liquid tumors; and NK+T cell therapy for use in the treatment of oncology, autoimmune disease, or infectious disease. It has a research collaboration agreement with CRISPR Therapeutics AG. Nkarta, Inc. was incorporated in 2015 and is based in South San Francisco, California.
Rallybio
NASDAQ:RLYBRallybio Corporation, a clinical-stage biotechnology company, engages in development and commercialization of life-transforming therapies for patients suffering from severe and rare diseases. Its lead product candidate is RLYB212, a monoclonal anti-HPA-1a antibody that has completed Phase I clinical trial for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT); and RLYB211 for the prevention of FNAIT. The company is also developing RLYB114, a pegylated complement factor 5 (C5)-targeted Affibody molecule in preclinical development for the treatment of complement-mediated ophthalmic diseases; RLYB116, an inhibitor of complement component 5 (C5) to treat several diseases of complement dysregulation which has completed phase 1 trial; and RLYB331, a preclinical antibody, for the treatment of severe anemia with ineffective erythropoiesis and iron overload. It entered into a strategic alliance with AbCellera to discover, develop, and commercialize novel antibody-based therapeutics for rare diseases. Rallybio has collaboration with Exscientia for the development of small molecule therapeutics for rare diseases; and collaboration agreement with Johnson & Johnson to provide pregnant individuals therapeutic solutions at risk of fetal and neonatal alloimmune thrombocytopenia. The company was founded in 2018 and is headquartered in New Haven, Connecticut.