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Acasti Pharma Inc. engages in the development and commercialization of pharmaceutical products for rare and orphan diseases in Canada. The company's lead product candidate is the GTX-104, an intravenous infusion to treat subarachnoid hemorrhage. It also develops GTX-102, an oral mucosal spray for the treatment of ataxia-telangiectasia; and GTX-101, a topical bioadhesive film-forming bupivacaine spray for postherpetic neuralgia. The company was incorporated in 2002 and is headquartered in Laval, Canada.

Capricor Therapeutics, Inc. (NASDAQ:CAPR) is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Capricor's lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy. Capricor has also established itself as one of the leading companies investigating the field of extracellular vesicles and is exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders.

Citius Pharmaceuticals, Inc., a late-stage pharmaceutical company, engages in the development and commercialization of critical care products focusing on oncology products, anti-infectives products in adjunct cancer care, prescription products, and stem cell therapy. It is developing five proprietary products comprising LYMPHIR, an engineered IL-2 diphtheria toxin fusion protein for the treatment of patients with persistent or recurrent cutaneous T-cell lymphoma, which is in Phase 3 clinical trial; Mino-Lok, an antibiotic lock solution to treat patients with catheter-related bloodstream infections by salvaging the infected catheter, which is in Phase 3 clinical trial; Halo-Lido, a corticosteroid-lidocaine topical formulation that intends to provide anti-inflammatory and anesthetic relief to persons suffering from hemorrhoids that is in clinical Phase 2b trial; Mino-Wrap, a liquifying gel-based wrap for reduction of tissue expander infections following breast reconstructive surgeries; and NoveCite, a mesenchymal stem cell therapy for the treatment of acute respiratory disease syndrome. The company was founded in 2007 and is headquartered in Cranford, New Jersey.

Stealth BioTherapeutics Corp, a clinical-stage biotechnology company, focuses on the discovery, development, and commercialization of therapies for diseases involving mitochondrial dysfunction. Its lead product candidate is Elamipretide that is in phase 3 clinical trial to treat barth syndrome, phase 2/3 clinical trial for the treatment of duchenne cardiomyopathy, phase 2a clinical trial to treat friedreich's ataxia, phase 2 clinical trial for the treatment of dry age-related macular degeneration, phase 2 clinical trial to treat leber's hereditary optic neuropathy, and phase 3 clinical trial for the treatment of replisome myopathies. The company's product candidates also include SBT-20 to treat complications of diabetes, renal diseases, ocular diseases, neurodegenerative, Parkinson's, Alzheimer's, Huntington's, and ALS diseases; SBT-550 for rare neurological indications; and SBT-272, a peptidomimetic that targets the mitochondria and stabilizing mitochondrial function under conditions of oxidative stress, as well as SBT 550. Stealth BioTherapeutics Corp was incorporated in 2006 and is based in George Town, the Cayman Islands.

Verona Pharma plc, a clinical stage biopharmaceutical company, focuses on development and commercialization of therapies for the treatment of respiratory diseases with unmet medical needs. The company's product candidate is ensifentrine, an inhaled and dual inhibitor of the phosphodiesterase (PDE) 3 and PDE4 enzymes that acts as both a bronchodilator and an anti-inflammatory agent in a single compound, which is in Phase 3 clinical trials for the treatment of chronic obstructive pulmonary disease, asthma, and cystic fibrosis. It is developing ensifentrine in three formulations, including nebulizer, dry powder inhaler, and pressurized metered-dose inhaler. The company was incorporated in 2005 and is headquartered in London, the United Kingdom.