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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.

Assembly Biosciences, Inc., a biopharmaceutical company, discovers and develops therapeutic candidates for the treatment of hepatitis B virus (HBV) infection in the United States. The company's next-generation HBV core inhibitors include ABI-4334, which is in Phase 1a studies for the treatment of HBV; and ABI-H3733, which is in Phase 1b clinical study. It is also involved in novel small molecule approaches for HBV and hepatitis delta virus (HDV) comprising an orally bioavailable HBV/HDV entry inhibitor and liver-focused interferon-a receptor agonist. In addition, the company develops Herpesvirus programs, such as ABI-5366 HSV-2, a long-acting helicase inhibitor; and pan-herpes non-nucleoside polymerase inhibitors for transplant-associated infections. Further, Assembly Biosciences, Inc. has collaboration agreement with BeiGene, Ltd. to develop and commercialize the novel core inhibitor product candidates for chronic HBV infection in the People's Republic of China, Hong Kong, Taiwan, and Macau; and clinical trial collaboration agreement with Arbutus Biopharma Corporation to conduct a randomized, multi-center, and open-label clinical trials, as well as agreement with Antios Therapeutics, Inc. for triple combination therapy. The company also has license agreement with Indiana University Research and Technology Corporation for HBV research, as well as a partnership with Gilead Sciences, Inc. to develop next-generation therapeutics for serious viral diseases. The company was formerly known as Ventrus Biosciences, Inc. and changed its name to Assembly Biosciences, Inc. in June 2014. Assembly Biosciences, Inc. was incorporated in 2005 and is headquartered in South San Francisco, California.

Kadmon is a clinical-stage biopharmaceutical company that discovers, develops and delivers transformative therapies for unmet medical needs. Its clinical pipeline includes treatments for immune and fibrotic diseases as well as immuno-oncology therapies.

Kura 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.

Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops medicines to treat the underlying causes of severe genetic diseases in the United States. The company utilizes its proprietary targeted augmentation of nuclear gene output to develop antisense oligonucleotides to selectively restore protein levels. Its lead clinical candidate is STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy. The company also develops STK-001, which is in phase I/II clinical trial to treat Dravet syndrome; and programs focused on multiple targets, including haploinsufficiency diseases of the central nervous system and eye. The company has a license and collaboration agreement with Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was incorporated in 2014 and is headquartered in Bedford, Massachusetts.