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Applied Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in the development of a pipeline of novel product candidates against validated molecular targets in indications of high unmet medical need in the United States. The company's lead product candidate is AT-007 (also called govorestat) that has completed phase 3 for the treatment of galactosemia in healthy volunteers and adults, in pediatric clinical study for the treatment of galactosemia in kids, for treating enzyme sorbitol dehydrogenase, and for the treatment of phosphomannomutase enzyme-CDG. It also develops AT-001 (also called caficrestat) that is in phase 3 clinical trials to treat diabetic cardiomyopathy, as well as for the treatment of diabetic peripheral neuropathy; and AT-003, which is in preclinical studies for the treatment diabetic retinopathy. The company has exclusive license and supply agreement with Mercury Pharma Group Limited to commercialize drug products containing AT-007. Applied Therapeutics, Inc. was incorporated in 2016 and is headquartered in New York, New York.

Cerecor, Inc. is a biopharmaceutical company, which engages in the development and commercialization of treatments for rare and orphan diseases. Its lead product candidates include CERC-801, CERC-802, and CERC-803, which are therapies for inherited metabolic disorders known as congenital disorders of glycosylation. The company was founded by Blake M. Paterson, Isaac Blech, Barbara S. Slusher, and Solomon H. Snyder on January 31, 2011 and is headquartered in Rockville, MD.

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.

Mustang Bio, Inc., a clinical-stage biopharmaceutical company, focuses on translating medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors, and rare genetic diseases. Its pipeline focuses on gene therapy programs for rare genetic disorders, chimeric antigen receptor (CAR) engineered T cell (CAR T) therapies for hematologic malignancies, and CAR T therapies for solid tumors. The company develops MB-117 and MB-217, a gene therapy program for X-linked severe combined immunodeficiency, a rare genetic immune system condition in which affected patients do not live beyond infancy without treatment. The company also develops MB-106 CAR T cell program for B cell non-hodgkin lymphoma and chronic lymphocytic leukemia; MB-101 CAR T cell program for glioblastoma; MB-108, a next-generation oncolytic herpes simplex virus. It has license agreements with Nationwide Children's Hospital, CSL Behring; Mayo Clinic; Leiden University Medical Centre; SIRION Biotech GmbH. The company was incorporated in 2015 and is headquartered in Worcester, Massachusetts.