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Akouos, Inc., a precision genetic medicine company, developing gene therapies to restore, improve, and preserve physiologic hearing for individuals. The company's precision genetic medicine platform incorporates a proprietary adeno-associated viral vector library and a novel delivery approach. Its lead product candidate is AK-OTOF, a gene therapy for the treatment of hearing loss due to mutations in the OTOF gene. The company is also developing AK-CLRN1 for the auditory manifestations of Usher syndrome 3A, or USH3A; and AK-antiVEGF for vestibular schwannoma. In addition, its precision genetic medicine platform addresses hearing loss related to genes needed for supporting cell function. The company was incorporated in 2016 and is based in Boston, Massachusetts.

Amryt Pharma plc, a commercial-stage biopharmaceutical company, focuses on acquiring, developing, and commercializing various treatments to help improve the lives of patients with rare and orphan diseases. The company provides metreleptin, an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy; oral octreotide, a long-term maintenance therapy in acromegaly patients; and lomitapide, an adjunct to a low-fat diet and other lipid-lowering medicinal products for adults with the Homozygous familial Hypercholesteraemia. Its development candidates include Oleogel-S10 for the cutaneous manifestations of Junctional and Dystrophic Epidermolysis Bullosa (EB), a rare and distressing genetic skin disorder affecting young children and adults; and AP103, a pre-clinical gene therapy for patients with dystrophic EB. The company sells its products in the Americas, Europe, and the Middle East. Amryt Pharma plc was founded in 2015 and is headquartered in Dublin, Ireland.

Marinus Pharmaceuticals, Inc., a pharmaceutical company, focuses on development and commercialization of therapeutic products for patients suffering from rare genetic epilepsies and other seizure disorders. It offers ZTALMY (ganaxolone), an oral suspension for the treatment of seizures associated with cyclin-dependent kinase-like 5 deficiency disorder for adult and pediatric patient populations in acute and chronic care, and in-patient and self-administered settings. The company's ZTALMY product candidate acts at synaptic and extrasynaptic GABAA receptors, a target for its anti-seizure, antidepressant, and anxiolytic potential. It is developing ganaxolone for treating genetic epilepsy disorders, such as PCDH19-related epilepsy, and tuberous sclerosis complex. Marinus Pharmaceuticals, Inc. has license agreement with Purdue Neuroscience Company and CyDex Pharmaceuticals, Inc.; and collaboration agreement with Orion Corporation and Tenacia Biotechnology (Shanghai) Co., Ltd. Marinus Pharmaceuticals, Inc. was incorporated in 2003 and is headquartered in Radnor, Pennsylvania.

Provention Bio, Inc. is a clinical-stage biopharmaceutical company, which engages in the development and commercialization of novel therapeutics and solutions. Its products include PRV-031 for the interception of type 1 diabetes (T1D), PRV-015 for the treatment of gluten-free diet non-responding celiac disease, PRV-6527 for Crohn's disease, PRV-3279 for the treatment of lupus, and PRV-101 for the prevention of acute coxsackie virus B (CVB), and the prevention of type 1 diabetes (T1D) onset. The company was founded by Francisco Leon and Ashleigh Palmer on October 4, 2016 and is headquartered in Red Bank, NJ.

XBiotech Inc., a biopharmaceutical company, discovers, develops, and commercializes True Human monoclonal antibodies for treating various diseases. The company focuses on developing a pipeline of product candidates targeting both inflammatory and infectious diseases. It is also developing interleukin-1 alpha therapies to treat variety of medical conditions, such as cancer, stroke, heart attack, or arthritis; and mediates tissue breakdown, angiogenesis, the formation of blood clots, malaise, muscle wasting, and inflammation. The company was incorporated in 2005 and is headquartered in Austin, Texas.