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Aurinia Pharmaceuticals Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies to treat various diseases with unmet medical need in the United States. It offers LUPKYNIS for the treatment of adult patients with active lupus nephritis. It has a collaboration and license agreement with Otsuka Pharmaceutical Co., Ltd. The company was incorporated in 1993 and is headquartered in Edmonton, Canada.

Generation Bio Co. develops non-viral genetic medicines for the treatment of rare and prevalent diseases. The company develops cell-targeted lipid nanoparticle (ctLNP) platform, a modular delivery system for nucleic acids to avoid off-target clearance by the liver and spleen that enables ctLNPs to persist in systemic circulation, which allows for highly selective and potent ligand-driven targeting to specific tissues and cell types; and novel immune-quiet DNA (iqDNA) to enable long-lasting high levels of gene expression from non-integrating episomes and avoids innate immune sensors that have long prevented DNA from use in non-viral systems. It uses its platform for developing a portfolio of programs for treating cancer, autoimmune, hematologic disorders, prioritizing sickle cell, beta-thalassemia, and hemophilia A diseases, as well as for other tissues and cell types, including retina, skeletal muscle, and central nervous system. The company was formerly known as Torus Therapeutics, Inc. and changed its name to Generation Bio Co. in November 2017. Generation Bio Co. was incorporated in 2016 and is headquartered in Cambridge, Massachusetts.

Harmony Biosciences Holdings, Inc., a commercial-stage pharmaceutical company, focuses on developing and commercializing therapies for patients with rare and other neurological diseases in the United States. The company offers WAKIX (pitolisant), a molecule with a novel mechanism of action for the treatment of excessive daytime sleepiness in adult patients with narcolepsy. It also offers HBS-102, a melanin-concentrating hormone receptor 1 for MCH neurons. The company was formerly known as Harmony Biosciences II, Inc. and changed its name to Harmony Biosciences Holdings, Inc. in February 2020. Harmony Biosciences Holdings, Inc. was incorporated in 2017 and is headquartered in Plymouth Meeting, Pennsylvania.

uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases. It offers HEMGENIX that has completed Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also develops AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington's disease. In addition, it is developing AMT-162, which is in preclinical trial for the treatment of superoxide dismutase 1- amyotrophic lateral sclerosis; AMT-260 that is in preclinical trial to treat temporal lobe epilepsy; AMT-191, which is in preclinical trial for the treatment of fabry disease; AMT-161 that is in preclinical trial to treat amyotrophic lateral sclerosis caused by mutations; AMT-240, which is in preclinical trial to for the treatment of autosomal dominant Alzheimer's disease; and AMT-210 that is in preclinical trial to treat Parkinson's disease. The company was founded in 1998 and is headquartered in Amsterdam, the Netherlands.