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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.

Allena Pharmaceuticals, Inc., a biopharmaceutical company, discovers, develops, and commercializes oral enzyme therapeutics to treat patients with rare and severe metabolic, and kidney disorders in the United States. Its lead product candidate is ALLN-346, a novel and urate degrading enzyme for patients with hyperuricemia and gout in the setting of advanced chronic kidney disease. The company was incorporated in 2011 and is headquartered in Newton, Massachusetts.

Curis, Inc., a biotechnology company, engages in the discovery and development of drug candidates for the treatment of human cancers in the United States. Its clinical stage drug candidates include Emavusertib, an oral small molecule IRAK4 kinase inhibitor, which is in a Phase 1/2 open-label, single arm expansion trial in patients with relapsed or refractory, or R/R, AML and high-risk myelodysplastic syndromes. The company's pipeline also includes Fimepinostat, an oral dual inhibitor of HDAC and PI3K enzymes for the treatment of patients with relapsed or refractory diffuse large B-cell lymphoma; CA-170, an oral, small molecule antagonist designated as CA-170 that selectively targets PD-L1 and VISTA; and CA-327, an oral, small molecule, TIM3/PD-L1, which is a molecule antagonist of PD-L1 and TIM3. It has collaboration agreement with Genentech Inc., or Genentech and F. Hoffmann-La Roche Ltd, or Roche, for the commercialization of Erivedge, an orally-administered small molecule hedgehog signaling pathway antagonist for the treatment of advanced basal cell carcinoma, or BCC; Aurigene Discovery Technologies Limited for the discovery, development, and commercialization of small molecule compounds in the areas of immuno-oncology and precision oncology; and also licensed four programs under the Aurigene collaboration, including emavusertib. Curis, Inc. was incorporated in 2000 and is headquartered in Lexington, Massachusetts.

Aceragen, Inc. is a clinical stage biopharmaceutical company, which focuses on the clinical development, and commercialization of drug candidates for both oncology and rare disease indications. The company was founded by Paul C. Zamecnik, Sudhir A. Agrawal, and James B. Wyngaarden on May 25, 1989 and is headquartered in Exton, PA.

Pluristem Therapeutics Inc. operates as a bio-technology company. It focuses on the research, development, clinical trial, and manufacture of cell therapeutic products and related technologies for the treatment of various ischemic, inflammatory, and hematologic conditions, as well as autoimmune disorders. The company develops placental expanded (PLX) based cell therapy products, including PLX-PAD that is in Phase III clinical trial for the recovery after surgery for hip fracture; in Phase II clinical trail for the treatment of acute respiratory distress syndrome associated with COVID-19; and in Phase I/II clinical trial for treatment of steroid-refractory graft versus host disease. It also develops PLX-R18, which has completed Phase I clinical trial incomplete hematopoietic recovery following hematopoietic cell transplantation, as well as conducts various animal studies for the evaluation of PLX-R18 for the treatment of acute radiation syndrome. The company has a license agreement for conducting clinical trials of PLX-PAD product in South Korea. It also has a collaborative project nTRACK, that examines gold nano particles labeling of stem cells; and has collaboration agreement with the NASA's Ames Research Center to evaluate the potential of PLX cell therapies in preventing and treating medical conditions caused during space missions. Pluristem Therapeutics Inc. was incorporated in 2001 and is based in Haifa, Israel.