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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.

Concert Pharmaceuticals, Inc. is a clinical stage biopharmaceutical company, which discovers and develops novel small molecule drugs through the use of deuterium technology. Its product candidates target a range of diseases including autoimmune disorders, inflammation, and central nervous system disorders. The company was founded by Richard H. Aldrich, Roger D. Tung and Christoph H. Westphal on April 12, 2006 and is headquartered in Lexington, MA.

Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy is under phase III clinical trial; and pociredir, a fetal hemoglobin inducer for the treatment of sickle cell disease and beta-thalassemia is under phase I clinical trial. The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases. Fulcrum Therapeutics, Inc. has collaboration and license agreement with Acceleron Pharma Inc. to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; and MyoKardia, Inc. to discover and develop therapies for the treatment of genetic cardiomyopathies. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

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.

Sol-Gel Technologies Ltd., together with its subsidiary Sol-Gel Technologies Inc., develops topical dermatological drugs for patients with severe skin conditions in Israel. The company offers Twyneo, a once-daily, non-antibiotic topical cream for the treatment of acne vulgaris; and Epsolay, a once-daily topical cream for the treatment of papulopustular (subtype II) rosacea. It also develops SGT-610 that is in Phase 3 clinical trials for the treatment of Gorlin Syndrome; and SGT-210, which has completed Phase I clinical trial, to treat rare hyperkeratinization disorders, such as Darier, PC, PPK, Olmsted, etc. In addition, the company is also involved in the development of generic topical dermatological drug products. It has collaboration with Padagis Israel Pharmaceuticals Ltd; and license agreements with Galderma Holding SA and Searchlight Pharma Inc. Sol-Gel Technologies Ltd. was incorporated in 1997 and is headquartered in Ness Ziona, Israel.