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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.

Chiasma, Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing oral medications using transient permeability enhancer technology platform for the treatment of rare and serious chronic disease worldwide. The company offers MYCAPSSA, an oral octreotide capsule for the long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide. It also develops MPOWERED, an oral octreotide capsule, which has completed Phase III clinical trials for the maintenance treatment of adult patients with acromegaly. Chiasma, Inc. was incorporated in 2001 and is headquartered in Needham, Massachusetts.

Checkpoint Therapeutics, Inc., a clinical-stage immunotherapy and targeted oncology company, focuses on the acquisition, development, and commercialization of novel treatments for patients with solid tumor cancers in the United States and internationally. The company's lead antibody product candidate is Cosibelimab, which is in Phase 1 clinical trial to treat patients with selected recurrent or metastatic cancers; and CK-302, a product candidate in preclinical trials for hematological malignancies and solid tumors. It is also developing Olafertinib, which is in Phase 3 clinical trials for the treatment of adult patients with metastatic non-small cell lung cancer; CK-103 for the treatment of various advanced and metastatic solid tumor cancers; and Anti-Carbonic Anhydrase IX (CAIX) antibody, a product candidate in preclinical trials to recognize CAIX expressing cells and kill them via antibody-dependent cellular cytotoxicity and complement-dependent cytotoxicity. The company has licensing and collaboration agreements with Adimab, LLC for the development of Cosibelimab; NeuPharma, Inc., to develop and commercialize Olafertinib; and Jubilant Biosys Limited for the development and commercialization of novel compounds that inhibit BET bromodomains. The company was incorporated in 2014 and is based in Waltham, Massachusetts. Checkpoint Therapeutics, Inc. is a subsidiary of Fortress Biotech, Inc.

Milestone Pharmaceuticals Inc., a biopharmaceutical company, focuses on the development and commercialization of cardiovascular medicines. The company's lead product candidate is etripamil, a novel and potent calcium channel blocker, which is in Phase III clinical trial for the treatment of paroxysmal supraventricular tachycardia in the United States and Canada; and Phase II clinical trial for the treatment of atrial fibrillation and rapid ventricular rate. It has a license and collaboration agreement with Ji Xing Pharmaceuticals to develop and commercialize etripamil prophylactic and therapeutic uses in humans, as well as with the Montreal Heart Institute, the WCN network, and other research centers. The company was incorporated in 2003 and is headquartered in Montréal, Canada.

NeuBase Therapeutics, Inc., a preclinical-stage biopharmaceutical company, focuses on the development of therapies to treat rare genetic diseases and cancers caused by mutant genes. The company's proprietary modular peptide-nucleic acid antisense oligo (PATrOL) platform focuses on addressing Huntington's disease (HD) and myotonic dystrophy, as well as other genetic disorders, and cancer and oncology applications. Its pipeline includes NT0100 for Huntington's disease; NT0200 for myotonic dystrophy type 1; and NT0300 treatment targets the mutated KRAS gene. NeuBase Therapeutics, Inc. is headquartered in Pittsburgh, Pennsylvania.