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Capricor Therapeutics, Inc. (NASDAQ:CAPR) is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Capricor's lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy. Capricor has also established itself as one of the leading companies investigating the field of extracellular vesicles and is exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders.

Clearside Biomedical, Inc., a biopharmaceutical company, focuses on the revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space. It offers XIPERE, a triamcinolone acetonide suprachoroidal injectable suspension for the treatment of uveitis macular edema. It also develops CLS-AX, an axitinib injectable suspension for suprachoroidal injection, which is in Phase IIb clinical trial to treat wet AMD. The company has a collaboration with Bausch Health, Arctic Vision, REGENXBIO, Inc., BioCryst Pharmaceuticals, Inc., and Aura Biosciences. Clearside Biomedical, Inc. was incorporated in 2011 and is headquartered in Alpharetta, Georgia.

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.

HOOKIPA Pharma Inc., a clinical-stage biopharmaceutical company, develops immunotherapeutics targeting infectious diseases and cancers based on its proprietary arenavirus platform. The company's lead infectious disease product candidate is HB-101, which is in a randomized double-blinded Phase 2 clinical trial in cytomegalovirus-negative patients awaiting kidney transplantation from cytomegalovirus-positive donors. Its lead oncology product candidates are HB-201 and HB-202 that are in preclinical studies for the treatment of human papillomavirus-positive cancers. The company has a collaboration with Gilead Sciences, Inc. to develop arenavirus based therapeutics to treat hepatitis B virus and human immunodeficiency virus infections. HOOKIPA Pharma Inc. was founded in 2011 and is headquartered in New York, New York.

Synlogic, Inc., a clinical-stage biopharmaceutical company, engages in the discovery and development of synthetic biotics to treat metabolic and immunological diseases in the United States. Its pipeline include SYNB1618, an orally administered, non-systemically absorbed drug candidate to treat phenylketonuria; SYNB1934, an orally administered, non-systemically absorbed drug candidate, which is in Phase III clinical trial to treat phenylketonuria; SYNB1353, an orally administered, non-systemically absorbed drug candidate, which is in Phase II clinical to treat homocystinuria; SYNB8802, an orally administered, non-systemically absorbed drug candidate that is in Phase II clinical trial for the treatment of enteric hyperoxaluria; and SYNB2081 to lower uric acid for the potential treatment of gout. The company is also developing SYNB1891, an intratumorally administered synthetic biotic medicine to treat solid tumors and lymphoma. It has a collaboration agreement with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. for the research and pre-clinical development of a synthetic biotic medicine for the treatment of inflammatory bowel disease; and Ginkgo Bioworks, Inc. Synlogic, Inc. is based in Cambridge, Massachusetts.