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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.

ADVANZ PHARMA Corp. Limited, a pharmaceutical company, through its subsidiaries, owns or licenses a portfolio of branded and generic prescription products worldwide. The company operates through two segments: ADVANZ PHARMA International and ADVANZ PHARMA North America. Its ADVANZ PHARMA International segment offers a portfolio of branded and generic prescription products to wholesalers, distributors, hospitals, and pharmacies. This segment's products include Brinavess for the conversion of onset atrial fibrillation to sinus rhythm in adults; Aggrastat, a reversible GP IIb/IIIa inhibitor indicated for use in patients with acute coronary syndrome; Xydalba, a semi-synthetic lipoglycopeptide for the treatment of acute bacterial skin and skin structure infections in adults; and Zevtera/Mabelio, a cephalosporin antibiotic for the treatment of community-acquired and hospital-acquired pneumonia. The company's ADVANZ PHARMA North America segment provides Donnatal for the treatment of irritable bowel syndrome; Zonegran for the treatment of partial seizures in adults with epilepsy; Nilandron for the treatment of metastatic prostate cancer; Lanoxin for the treatment of mild to moderate heart failure and atrial fibrillation; Plaquenil for the treatment of lupus and rheumatoid arthritis; and Photofrin for the treatment of certain types of cancer. It also offers Prostaglandin E1 formulations for the treatment of erectile dysfunction and peripheral arterial occlusive disease under the Prostavasin, Viridal, Vasaprostan, and Edex brands, as well as holds licensed commercialization rights to a pre-registration drug/device combination product, Trevyent for the treatment of pulmonary arterial hypertension. It sells its products through direct sales and local distribution relationships. The company was formerly known as ADVANZ PHARMA Corp. and changed its name to ADVANZ PHARMA Corp. Limited in December 2019. ADVANZ PHARMA Corp. Limited is headquartered in London, the United Kingdom.

Mirum Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases. Its lead product candidate is LIVMARLI (maralixibat), an orally administered and minimally absorbed ileal bile acid transporter (IBAT) inhibitor that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States and internationally. The company is also involved in the commercialization of Cholbam, a cholic acid capsule, which is approved as treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for adjunctive treatment of patients with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder and Smith-Lemli-Opitz syndrome; and Chenodal, a tablet, which is approved for the treatment of radiolucent stones in the gallbladder, and under Phase 3 development for the treatment cerebrotendinous xanthomatosis. In addition, it develops Volixibat, an oral and minimally absorbed agent designed to inhibit IBAT, currently under Phase 2b clinical trial for the treatment of adult patients with cholestatic liver diseases. The company was incorporated in 2018 and is headquartered in Foster City, California.

Proteostasis Therapeutics, Inc., a clinical stage biopharmaceutical company, engages in the discovery and development of novel therapeutics to treat cystic fibrosis. The company's lead product candidates for the treatment of CF include PTI-801, a cystic fibrosis transmembrane conductance regulator (CFTR) corrector agent; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. It has collaboration agreements with Cystic Fibrosis Foundation, Inc. to research, develop, and commercialize products for the treatment of CF, non-classical CF, and other pulmonary diseases in the United States or the European Union; and Genentech, Inc. for licensing the technology and materials relating to therapeutic small molecule modulators. The company was formerly known as Proteoguard, Inc. and changed its name to Proteostasis Therapeutics, Inc. in September 2007. Proteostasis Therapeutics, Inc. was founded in 2006 and is headquartered in Boston, Massachusetts.

Spero Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on identifying, developing, and commercializing novel treatments for multi-drug resistant (MDR) bacterial infections and rare diseases in the United States. The company's product candidates include tebipenem pivoxil hydrobromide (HBr), an oral carbapenem-class antibiotic to treat complicated urinary tract infections, including pyelonephritis for adults; SPR206, an intravenous-administered antibiotic against MDR Gram-negative pathogens comprising carbapenem-resistant enterobacterales (CRE), acinetobacter baumannii, and pseudomonas aeruginosa, as well as negative bacterial infections in the hospital setting; and SPR720, a novel oral antibiotic agent for the treatment of non-tuberculous mycobacterial pulmonary disease. It has license agreement with Meiji Seika Pharma Co., Ltd. to support the development of tebipenem HBr; Everest Medicines to develop, manufacture, and commercialize SPR206 in Greater China, South Korea, and Southeast Asian countries; and Vertex Pharmaceuticals Incorporated for patents relating to SPR720, as well as SPR719, an active metabolite. Spero Therapeutics, Inc. was founded in 2013 and is headquartered in Cambridge, Massachusetts.