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Eton Pharmaceuticals, Inc., a specialty pharmaceutical company, focuses on developing, acquiring, and commercializing pharmaceutical products for rare diseases. The company offers ALKINDI SPRINKLE, a replacement therapy for adrenocortical insufficiency in children under 17 years of age; Carglumic Acid for the treatment of acute and chronic hyperammonemia due to N-acetylglutamate Synthase deficiency; Betaine Anhydrous for the treatment of homocystinuria; and Nitisinone for the treatment of tyrosinemia type 1. It also provides Zeneo hydrocortisone autoinjector for the treatment of adrenal crisis. Eton Pharmaceuticals, Inc. was incorporated in 2017 and is based in Deer Park, Illinois.

Medicenna Therapeutics Corp., an immunotherapy company, engages in the development and commercialization of Superkines and empowered Superkines for the treatment of cancer and other diseases. It develops MDNA55, an interleukin- 4 (IL-4) EC for the treatment of recurrent glioblastoma, as well as for brain tumors. The company also develops MDNA11, an enhanced version of IL-2 to activate and proliferate the immune cells needed to fight cancer; MDNA209, an IL-2 antagonist for autoimmune diseases, such as multiple sclerosis and graft versus host disease; MDNA413, a dual IL-4/IL-13 antagonist to treat cancer immunotherapies; and MDNA132, an IL-13 Superkine for solid tumors. In addition, it provides BiSKITs platform to develop designer Superkines by fusing them to other proteins, antibodies, cytokines, or other Superkines. The company is headquartered in Toronto, Canada.

NeuBase Therapeutics, Inc., a preclinical-stage biopharmaceutical company, focuses on the development of therapies to treat rare genetic diseases and cancers caused by mutant genes. The company's proprietary modular peptide-nucleic acid antisense oligo (PATrOL) platform focuses on addressing Huntington's disease (HD) and myotonic dystrophy, as well as other genetic disorders, and cancer and oncology applications. Its pipeline includes NT0100 for Huntington's disease; NT0200 for myotonic dystrophy type 1; and NT0300 treatment targets the mutated KRAS gene. NeuBase Therapeutics, Inc. is headquartered in Pittsburgh, Pennsylvania.

Ritter Pharmaceuticals, Inc. develops and sells novel therapeutic products that modulate the human gut microbiome to treat gastrointestinal diseases. Its lead product candidate is RP-G28, a novel microbiome modulator, which has completed Phase 2b clinical trial for the reduction of symptoms associated with lactose intolerance. The company's product, RP-G28 is designed to stimulate the growth of lactose-metabolizing bacteria in the colon, thereby adapting the gut microbiome to assist in digesting the lactose that reaches the large intestine. The company was formerly known as Ritter Natural Sciences, LLC and changed its name to Ritter Pharmaceuticals, Inc. in September 2008. Ritter Pharmaceuticals, Inc. was founded in 2004 and is headquartered in Los Angeles, California.