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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.

AcelRx Pharmaceuticals, Inc., a specialty pharmaceutical company, focuses on the development and commercialization of therapies for the treatment of acute pain. Its lead product candidate is DSUVIA and DZUVEO, a 30 mcg sufentanil sublingual tablet for the treatment of moderate-to-severe acute pain. The company develops ZALVISO, a patient-controlled analgesia system that allows hospital patients with moderate-to-severe acute pain to self-dose with sufentanil sublingual tablets to manage their pain; ARX-02, which is in Phase 2 clinical trial for the treatment of cancer breakthrough pain in opioid-tolerant patients; and ARX-03, which is in Phase 2 clinical trial for the treatment of procedural anxiety and acute pain. Its product candidates also include Niyad, a regional anticoagulant for the extracorporeal circuit; and LTX-608, a nafamostat formulation for direct IV infusion being explored as an investigational product for antiviral treatment of COVID, acute respiratory distress syndrome, disseminated intravascular coagulation, and acute pancreatitis. The company also develops Fedsyra, an ephedrine pre-filled syringe that contains 10 ml of a solution of 3 mg/ml ephedrine hydrochloride for injection; and phenylephrine, a phenylephrine pre-filled syringe, which contains 10 ml of a solution of 50 mcg/ml phenylephrine for injection. The company was formerly known as SuRx, Inc. and changed its name to AcelRx Pharmaceuticals, Inc. in August 2006. AcelRx Pharmaceuticals, Inc. was incorporated in 2005 and is headquartered in Hayward, California.

Kindred Biosciences, Inc. is a development-stage biopharmaceutical company, which focuses on saving and improving the lives of pets. The company was founded by Richard Chin and Denise M. Bevers on September 25, 2012 and is headquartered in Burlingame, CA.

9 Meters Biopharma, Inc., a clinical-stage biopharmaceutical company, focuses on treatments for people with rare digestive diseases, gastrointestinal conditions with unmet needs, and debilitating disorders in North America. Its lead product candidate is Vurolenatide, a long-acting injectable glucagon-like-peptide-1 that is in Phase 3 clinical trial to treat short bowel syndrome. The company is developing NM-136, a humanized monoclonal antibody for the treatment of obesity and obesity-related disorders; NM-102, a small molecule peptide to prevent antigens from trafficking into systemic circulation; and NM-003, a proprietary long-acting glucagon-like peptide-2 receptor agonist for prevention of acute graft versus host disease. Its partnered product is Larazotide, an 8-amino acid peptide, which is in Phase 2 clinical trial for treatment of multi-system inflammatory syndrome. The company has licensing agreements with Amunix, MHS Care Innovation LLC, Alba Therapeutics Corporation, and EBRIS. 9 Meters Biopharma, Inc. is headquartered in Raleigh, North Carolina. On July 17, 2023, 9 Meters Biopharma, Inc. filed a voluntary petition for liquidation under Chapter 7 in the U.S. Bankruptcy Court for the Eastern District of North Carolina.

Strongbridge Biopharma Plc operates as a commercial-stage biopharmaceutical company, which focuses on the development and commercialization of therapies for rare diseases. Its pipeline is comprised of Keveyis, Macrilen, Recorlev, and Veldoreotide. The company was founded in 1996 and is headquartered in Trevose, PA.