Aclaris Therapeutics
NASDAQ:ACRSAclaris Therapeutics, Inc. a clinical-stage biopharmaceutical company, engages in the development of novel drug candidates for immune-inflammatory diseases in the United States. The company operates through two segments, Therapeutics and Contract Research. The Therapeutics segment is involved in identifying and developing therapies to address significant unmet needs for immuno-inflammatory diseases. The Contract Research segment provides laboratory services. It develops Zunsemetinib (ATI-450), an MK2 inhibitor which is under Phase 1b/2 trials for the treatment of metastatic breast and pancreatic cancer; ATI-1777, a soft JAK 1/3 inhibitor, completed Phase 2b trails for the treatment of moderate to severe atopic dermatitis and other dermatologic conditions; and ATI-2138, an oral covalent inhibitor of ITK and JAK3 inhibitor under Phase 1 trials as a treatment for T cell-mediated autoimmune diseases. The company was incorporated in 2012 and is headquartered in Wayne, Pennsylvania.
Chinook Therapeutics
NASDAQ:KDNYChinook Therapeutics, Inc., a clinical-stage biotechnology company, focuses on the discovery, development, and commercialization of precision medicines for kidney diseases. The company's lead clinical program is atrasentan, a Phase III endothelin receptor antagonist for the treatment of IgA nephropathy and other proteinuric glomerular diseases. Its product candidates also include BION-1301, an anti-APRIL monoclonal antibody is being evaluated in a Phase I/II trial for IgA nephropathy; and CHK-336, an oral small molecule LDHA inhibitor for the treatment of primary hyperoxaluria, as well as research programs for other rare and severe chronic kidney diseases. Chinook Therapeutics, Inc. is headquartered in Seattle, Washington. As of August 11, 2023, Chinook Therapeutics, Inc. operates as a subsidiary of Novartis AG.
Kura Oncology
NASDAQ:KURAKura Oncology, Inc., a clinical-stage biopharmaceutical company, develops medicines for the treatment of cancer. The company's pipeline consists of small molecule product candidates that target cancer. Its lead product candidates are ziftomenib, an orally bioavailable small molecule inhibitor of the menin-KMT2A interaction for the treatment of genetically defined subsets of acute leukemias, including acute myeloid leukemia and acute lymphoblastic leukemia; tipifarnib, an orally bioavailable farnesyl transferase inhibitor combination with alpelisib for patients with PIK3CA-dependent HNSCC; and KO-2806, a farnesyl transferase inhibitor for the treatment of solid tumors. It has a clinical collaboration with Novartis Pharma AG to evaluate the combination of tipifarnib and alpelisib in patients with head and neck squamous cell carcinoma whose tumors have HRAS overexpression and/or PIK3CA mutation and/or amplification. The company was founded in 2014 and is headquartered in San Diego, California.
Rocket Pharmaceuticals
NASDAQ:RCKTRocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inheritable cardiac disorder; and BAG3 Dilated Cardiomyopathy. It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.