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AVROBIO, Inc., a clinical-stage gene therapy company, develops hematopoietic stem cell gene therapies to treat rare diseases in the United States. Its gene therapies employ hematopoietic stem cells that are harvested from the patient and then modified with a lentiviral vector to insert the equivalent of a functional copy of the gene that is mutated in the target disease. The company's pipeline includes AVR-RD-02, which is in phase 1/2 clinical trial for the treatment of Gaucher disease type 1 and 3; AVR-RD-01, which is in phase 2 clinical trials for the treatment of Fabry disease; and AVR-RD-03 that is in preclinical stage for the treatment of Pompe disease. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

ERYTECH Pharma S.A., a biopharmaceutical company, focus on development of red blood cell-based therapeutics for cancer and orphan diseases in France and the United States. It develops eryaspase, which is in Phase 3 clinical development for the treatment of second-line pancreatic cancer, and in Phase 2 stage for the treatment of triple-negative breast cancer and second-line acute lymphoblastic leukemia patients. The company also engages in developing erymethionase, a preclinical product candidate that consists of methionine-?-lyase in red blood cells to target the amino acid metabolism of cancer cells and induce tumor starvation. ERYTECH Pharma S.A. was incorporated in 2004 and is headquartered in Lyon, France.

Protara Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in advancing transformative therapies for the treatment of cancer and rare diseases. The company's lead program is TARA-002, an investigational cell therapy for the treatment of lymphatic malformations. It also develops intravenous choline chloride, an investigational phospholipid substrate replacement therapy for the treatment of intestinal failure associated liver disease. The company was formerly known as ArTara Therapeutics, Inc. and changed its name to Protara Therapeutics, Inc. in May 2020. The company is headquartered in New York, New York.

TRACON Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel targeted therapeutics for cancer in the United States. Its clinical stage products include envafolimab (KN035), a PD-L1 single-domain antibody for the treatment of refractory soft tissue sarcoma; and YH001, an investigational humanized cytotoxic T-lymphocyte-associated protein 4 IgG1 monoclonal antibody that is in Phase I clinical trial for the treatment of various cancer indications. The company's clinical stage products also include TRC102, a small molecule that is in Phase II clinical trial for the treatment of mesothelioma, and in Phase I clinical trial to treat solid tumors, lung cancer, and glioblastoma; and TJ004309, a CD73 antibody that is in Phase I clinical development for the treatment of solid tumors. In addition, it is developing bispecific antibodies, which are in preclinical stage. It has collaboration and license agreements with 3D Medicines Co., Ltd. and Jiangsu Alphamab Biopharmaceuticals Co., Ltd. for the development of envafolimab; I-Mab Biopharma for the development of CD73 antibody TJ004309 and bispecific antibodies; and cooperative research and development agreement with National Cancer Institute. The company was formerly known as Lexington Pharmaceuticals, Inc. and changed its name to TRACON Pharmaceuticals, Inc. in March 2005. TRACON Pharmaceuticals, Inc. was incorporated in 2004 and is headquartered in San Diego, California.

X4 Pharmaceuticals, Inc., a late-stage clinical biopharmaceutical company, focuses on the research, development, and commercialization of novel therapeutics for the treatment of rare diseases. Its lead product candidate is mavorixafor, a small molecule inhibitor of the chemokine receptor C-X-C chemokine receptor type 4 (CXCR4), which is in Phase III clinical trial for the treatment of patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome; and Phase II clinical trial to treat congenital, idiopathic, or cyclic neutropenia. The company is also developing X4P-003, a CXCR4 antagonist for the treatment of CXCR4 dependent disorders and primary immunodeficiencies; and X4P-002, a CXCR4 antagonist for the treatment of blood-brain barrier diseases. It has a license agreement with Abbisko Therapeutics Co Ltd. to develop, manufacture, and commercialize mavorixafor in combination with checkpoint inhibitors or other agents in oncology indications. The company is headquartered in Boston, Massachusetts.