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Applied Genetic Technologies Corporation, a clinical-stage biotechnology company, develops transformational genetic therapies for patients suffering from rare and debilitating diseases. Its advanced product candidates include three ophthalmology development programs across two targets, including X-linked retinitis pigmentosa, which is in the Phase 1/2 clinical trials; and achromatopsia that is in Phase 1/2 clinical trials. It is also developing an optogenetic product candidate to treat advanced retinal disease. In addition, the company has initiated one preclinical program in otology; preclinical program in dry age-related macular degeneration; and two preclinical programs in targeting central nervous system disorders, including frontotemporal dementia and amyotrophic lateral sclerosis. It has collaboration agreements with University of Florida; Bionic Sight, Inc.; and Otonomy, Inc. The company was incorporated in 1999 and is headquartered in Alachua, Florida.

CASI Pharmaceuticals, Inc., a biopharmaceutical company, develops and commercializes therapeutics and pharmaceutical products in the People's Republic of China, the United States, and internationally. It offers EVOMELA, an intravenous formulation of melphalan for use as a conditioning treatment prior to stem cell transplantation, and as a palliative treatment for patients with multiple myeloma. The company's other hematology/oncology assets in pipeline include CNCT 19, an autologous CD19 CAR-T investigative product; BI-1206, novel anti-Fc?RIIB antibody, for the treatment of solid tumors and relapsed/refractory non-hodgkin lymphoma; CB-5339, a novel VCP/p97 inhibitor that focuses on valosin-containing protein (VCP)/p97 as a novel target in protein homeostasis, DNA damage response, and other cellular stress pathways for therapeutic use in the treatment of patients with various malignancies; and CID-103, a human IgG1 anti-CD38 monoclonal antibody. The company has licensing agreements with Juventas Biotechnology (Tianjin) Co., Ltd.; BioInvent International AB; Black Belt Therapeutics Limited; Cleave Therapeutics, Inc.; and Acrotech Biopharma L.L.C. to develop and commercialize its commercial product EVOMELA. It also has distribution agreements with China Resources Pharmaceutical Commercial Group International Trading Co., Ltd; Pharmathen Global BV; and Riemser Pharma GmbH. CASI Pharmaceuticals, Inc. was incorporated in 1991 and is based in Beijing, the People's Republic of China.

Codiak BioSciences, Inc., a clinical-stage biopharmaceutical company, focuses on developing exosome-based therapeutics for the treatment of oncology, neuro-oncology, neurology, neuromuscular diseases, infectious diseases, and other diseases. Its lead product candidates are exoSTING and exoIL-12 exosome therapeutic candidates for the treatment of various solid tumors. The company is also developing exoASO-STAT6, an antisense oligonucleotide targeting the STAT6 transcription factor; and exoVACC, a modular platform for constructing precision vaccines. It has a collaboration agreement with the Jazz Pharmaceuticals Ireland Limited, Sarepta Therapeutics, Inc., Kayla Therapeutics S.A.S., Washington University, Ragon Institute of MGH, MIT, and Harvard. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Quince Therapeutics, Inc., a preclinical stage biopharmaceutical company, develops precision therapeutics for debilitating and rare diseases. Its lead compound is NOV004, a systemically administered bone anabolic peptide engineered to target and concentrate at bone fracture sites. The company was formerly known as Cortexyme, Inc. and changed its name to Quince Therapeutics, Inc. in August 2022. Quince Therapeutics, Inc. was incorporated in 2012 and is headquartered in South San Francisco, California.