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Avadel Pharmaceuticals plc operates as a biopharmaceutical company in the United States. Its lead product candidate is LUMRYZ, a formulation of sodium oxybate, which is in a Phase 3 clinical trial for the treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy. The company was formerly known as Flamel Technologies SA and changed its name to Avadel Pharmaceuticals plc in January 2017. Avadel Pharmaceuticals plc was incorporated in 2015 and is headquartered in Dublin, Ireland.

Homology Medicines, Inc., a genetic medicines company, focuses on transforming the lives of patients suffering from rare genetic diseases in the United States. The company offers proprietary platform is designed to utilize its human hematopoietic stem cell derived adeno-associated virus vectors (AAVHSCs) to deliver genetic medicines in vivo either through a nuclease-free gene editing mobility, gene therapy or for various genetic disorders. Its earlier stage product candidates comprising HMI-202, an investigational gene therapy which completed IND-enabling studies for the treatment of metachromatic leukodystrophy; and HMI-104 which completed IND-enabling studies. Homology Medicines, Inc. was incorporated in 2015 and is headquartered in Bedford, Massachusetts.

Inozyme Pharma, Inc., a clinical-stage rare disease biopharmaceutical company, focuses on developing novel therapeutics for the treatment of vasculature, soft tissue, and skeleton diseases. The company's lead product candidate is INZ-701, a soluble, recombinant, genetically engineered, and fusion protein for the treatment of ENPP1 and ABCC6 deficiencies, as well as patients with end-stage kidney disease receiving hemodialysis. It has a license agreement with Yale University for specified therapeutic and prophylactic products. The company was incorporated in 2015 and is headquartered in Boston, Massachusetts.

SIGA Technologies, Inc., a commercial-stage pharmaceutical company, focuses on the health security related markets in the United States. Its lead product is TPOXX, an oral formulation antiviral drug for the treatment of human smallpox disease caused by variola virus. The company was incorporated in 1995 and is headquartered in New York, New York.

Spruce Biosciences, Inc., a biopharmaceutical company, focuses on developing and commercializing novel therapies for rare endocrine disorders. The company engages in developing tildacerfont, a non-steroidal therapy to enhance disease control and reduce steroid burden for patients suffering from congenital adrenal hyperplasia (CAH), which is in Phase 2b clinical trial; and to evaluate glucocorticoid reduction in adult patients with classic CAH that is Phase 2b clinical trial. It is also developing tildacerfont for the treatment of pediatric classic congenital adrenal hyperplasia in children that is in Phase 2 clinical trial; and for females with polycystic ovary syndrome, which is in Phase 2 clinical trial. Spruce Biosciences, Inc. has a license agreement with Eli Lilly and Company to research, develop, and commercialize compounds for various pharmaceutical uses; and collaboration and license agreement with Kaken Pharmaceutical Co. Ltd. to develop, manufacture, and commercialize tildacerfont for the treatment of CAH in Japan. The company was incorporated in 2014 and is headquartered in South San Francisco, California.