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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.

Metacrine, Inc., a clinical-stage biopharmaceutical company, focuses on discovering and developing therapies for patients with gastrointestinal diseases. It is developing MET642, which has completed Phase I clinical trial for the treatment of ulcerative colitis. Metacrine, Inc. was incorporated in 2014 and is headquartered in San Diego, California.

Regulus Therapeutics Inc., a clinical stage biopharmaceutical company, focuses on discovery and development of drugs that targets microRNAs to treat a range of diseases in the United States. Its product candidates include RG-012, an anti-miR targeting miR-21 that is in Phase II clinical trial for the treatment of Alport syndrome, a life-threatening kidney disease; and RGLS8429, an anti-miR targeting miR-17, which is in Phase 1b clinical study for the treatment of autosomal dominant polycystic kidney disease. The company is also developing a pipeline of preclinical drug products for target organ-selective delivery strategies. Regulus Therapeutics Inc. was incorporated in 2007 and is headquartered in San Diego, California.

Soligenix, Inc., a late-stage biopharmaceutical company, focuses on developing and commercializing products to treat rare diseases in the United States. The company operates through two segments, Specialized BioTherapeutics and Public Health Solutions. The Specialized BioTherapeutics segment develops SGX301 (HyBryte), a novel photodynamic therapy, which has completed Phase III clinical trial for the treatment of cutaneous T-cell lymphoma; SGX942, an innate defense regulator technology that is in Phase III clinical trial for the treatment of inflammatory diseases, including oral mucositis in head and neck cancer; SGX302, an IDR technology which is in Phase IIa study to treat mil-to-moderate Psoriasis; and SGX945 and IDR technology that is in Phase IIa protocol for the treatment of Aphthous Ulcers in Behçet's Disease. The Public Health Solutions segment is involved in the development of RiVax, a ricin toxin vaccine candidate, which has completed Phase Ia, Ib, and Ic clinical trials; SGX943, a therapeutic candidate that is in pre-clinical stage for the treatment of antibiotic-resistant and emerging infectious diseases; ThermoVax, a technology in pre-clinical development for thermostabilizing vaccines; and CiVax, a vaccine candidate for the prevention of COVID-19. The company was formerly known as DOR BioPharma, Inc. and changed its name to Soligenix, Inc. in 2009. Soligenix, Inc. was incorporated in 1987 and is headquartered in Princeton, New Jersey.

Vallon Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel medications for central nervous system disorders with a focus on abuse-deterrent medications. Its lead investigational product candidate is ADAIR, an abuse-deterrent oral formulation of immediate-release dextroamphetamine for the treatment of attention-deficit/hyperactivity disorder (ADHD) and narcolepsy. The company is also developing ADMIR, an abuse deterrent formulation of methylphenidate for the treatment of ADHD. Vallon Pharmaceuticals, Inc. was incorporated in 2018 and is based in Philadelphia, Pennsylvania.