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Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company, focuses on developing and delivering gene therapy products for severe and life-threatening rare diseases. The company's lead programs are EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB); ABO-102, which are AAV based gene therapies for Sanfilippo syndrome type A; and ABO-101, an adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome type B. It is also developing ABO-201 gene therapy for juvenile Batten disease; ABO-202 gene therapy for treatment of infantile Batten disease; EB-201 for for epidermolysis bullosa (EB); ABO-301 for Fanconi anemia disorder; and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy program for rare blood diseases. Further, it is involved in marketing MuGard, a mucoadhesive oral wound rinse for the management of mucositis, stomatitis, aphthous ulcers, and traumatic ulcers. Abeona Therapeutics Inc. has collaborations with EB Research Partnership and Epidermolysis Bullosa Medical Research Foundation that focus on gene therapy treatments for EB; and Brammer Bio for commercial translation of ABO-102. The company was formerly known as PlasmaTech Biopharmaceuticals, Inc. and changed its name to Abeona Therapeutics Inc. in June 2015. Abeona Therapeutics Inc. was incorporated in 1989 and is based in Dallas, Texas.

Eledon Pharmaceuticals, Inc. operates as a clinical stage biotechnology company. The company uses its immunology expertise in targeting the CD40 Ligand (CD40L, also called CD154) pathway to develop therapies to protect transplanted organs and prevent rejection, and to treat amyotrophic lateral sclerosis (ALS). The company's lead compound in development is tegoprubart, an anti-CD40L antibody with high affinity for CD40 Ligand, a biological target with therapeutic potential. It has a collaboration agreement with eGenesis, Inc. The company was formerly known as Novus Therapeutics, Inc. and changed its name to Eledon Pharmaceuticals, Inc. in January 2021. Eledon Pharmaceuticals, Inc. is headquartered in Irvine, California.

Galecto, Inc., a clinical-stage biotechnology company, develops molecules for the treatment of fibrosis, cancer, inflammation, and other related diseases. The company's lead product candidate is GB2064, which is in Phase IIa for the treatment of myelofibrosis. It also develops GB0139, an inhaled small molecule inhibitor of galectin-3 that is in Phase IIb clinical trial for the treatment of fibrotic lung diseases, such as idiopathic pulmonary fibrosis, a life-threatening progressive fibrotic disease of the lung; GB2064, a selective oral small molecule inhibitor of LOXL2 that is in Phase 2a clinical trial for the treatment of myelofibrosis; and GB1211, a selective oral galectin-3 inhibitor that is in Phase IIa for the treatment of cancer, as well as in Phase Ib/IIa for fibrosis. Galecto, Inc. was founded in 2011 and is headquartered in Copenhagen, Denmark.

Genprex, Inc., a clinical-stage gene therapy company, focuses on developing therapies for patients with cancer and diabetes. Its lead product candidate is REQORSA (GPX-001) that is in Phase 1/2 and 2 clinical trials to treat non-small cell lung cancer and small cell lung cancer. The company is also developing GPX-002 for the treatment of type 1 diabetes, and GPX-003 for the treatment of type 2 diabetes. Genprex, Inc. was incorporated in 2009 and is headquartered in Austin, Texas.

Ocuphire Pharma, Inc., a clinical-stage ophthalmic biopharmaceutical company, focuses on developing and commercializing therapies for the treatment of unmet needs of patients with refractive and retinal eye disorders. The company offers Phentolamine Ophthalmic Solution for reversal of mydriasis, as well as is in Phase III clinical trials for presbyopia and dim light or night vision disturbances. Its lead retinal product candidate is APX3330, a small-molecule inhibitor of reduction oxidation effector factor-1 protein that has completed Phase II clinical trial for the treatment of diabetic retinopathy. The company also develops APX2009 and APX2014 that are preclinical product candidates for retina indications. The company was founded in 2018 and is headquartered in Farmington Hills, Michigan.