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Homology Medicines, Inc., a genetic medicines company, focuses on transforming the lives of patients suffering from rare genetic diseases in the United States. The company offers proprietary platform is designed to utilize its human hematopoietic stem cell derived adeno-associated virus vectors (AAVHSCs) to deliver genetic medicines in vivo either through a nuclease-free gene editing mobility, gene therapy or for various genetic disorders. Its earlier stage product candidates comprising HMI-202, an investigational gene therapy which completed IND-enabling studies for the treatment of metachromatic leukodystrophy; and HMI-104 which completed IND-enabling studies. Homology Medicines, Inc. was incorporated in 2015 and is headquartered in Bedford, Massachusetts.

Omeros Corporation, a clinical-stage biopharmaceutical company, discovers, develops, and commercializes small-molecule and protein therapeutics, and orphan indications targeting inflammation, complement-mediated diseases, cancers, and addictive and compulsive disorders. The company's clinical programs include Narsoplimab (OMS721/MASP-2) that has completed pivotal studies for hematopoietic stem-cell transplant-associated thrombotic microangiopathy (HSCT-TMA); that is in Phase III clinical trial for immunoglobulin A nephropathy (IgAN); and Phase II clinical trial to treat COVID-19. It also develops OMS1029 that is in phase I clinical trials for long-acting second-generation antibody targeting lectin pathway disorders; OMS906 that is in phase Ib clinical trials for Paroxysmal nocturnal hemoglobinuria, complement 3 glomerulopathy, and other alternative pathway disorders; OMS527 that is in phase I clinical trials for addictions and compulsive disorders, and movement disorders; and OMS405 that is in phase II clinical trials for opioid and nicotine addiction. In addition, the company develops MASP-2-small-molecule inhibitors for the treatment of aHUS, HSCT-TMA, and age-related macular degeneration; GPR174 inhibitors and chimeric antigen receptor T-Cell and adoptive T-Cell therapies for various cancers; and G protein-coupled receptors for treating immunologic, immuno-oncologic, metabolic, CNS, cardiovascular, musculoskeletal, and other disorders. Omeros Corporation was incorporated in 1994 and is headquartered in Seattle, Washington.

Principia Biopharma Inc., a late-stage biopharmaceutical company, focuses on developing novel therapies for immunology and oncology. The company is developing PRN1008, an inhibitor that is in Phase III clinical trial for the treatment of pemphigus, a chronic skin disease, as well as in Phase II clinical trial to treat immune thrombocytopenic purpura. It is also developing PRN2246/SAR442168, an inhibitor, which has completed Phase I clinical trial for treating multiple sclerosis (MS) and other central nervous system (CNS) diseases; PRN1371, a drug candidate that is in Phase I clinical trial for the treatment of solid tumors; and oral small molecule inhibitors of the immunoproteasome. The company has a collaboration agreement with Genzyme Corporation to develop relapsing and progressive MS and other diseases of the CNS. Principia Biopharma Inc. was incorporated in 2008 and is headquartered in South San Francisco, California.

Rocket 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.

Travere Therapeutics, Inc. is a biopharmaceutical company. It engages in the identification, development, commercialization, and distribution of therapies to people living with rare diseases. Its products include Chenodal, Cholbam, and Thiola. The company was founded by Martin Shkreli on February 8, 2008 and is headquartered in San Diego, CA.