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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.

Flexion Therapeutics, Inc. operates as a biopharmaceutical company, which engages in the development and commercialization of novel and local therapies. It specializes in the treatment of patients with musculoskeletal conditions including osteoarthritis. It offers products under the Zilretta brand, an extended-release corticosteroid approved to manage osteoarthritis knee pain. The company was founded by Michael D. Clayman and Neil Bodick on November 5, 2007 and is headquartered in Burlington, MA.

Frequency Therapeutics, Inc., a clinical-stage biotechnology company, focuses on developing therapeutics to activate a person's innate regenerative potential to restore function. Its Progenitor Cell Activation approach uses small molecules to activate progenitor cells within the body to create functional tissue. The company's lead product candidate is FX-322, which is in Phase IIb clinical trial to treat the underlying cause of sensorineural hearing loss. It is also developing medicines for patients across a range of degenerative conditions, including multiple sclerosis, and diseases of the muscle, gastrointestinal tract, skin, and bone. The company has a license and collaboration agreement with Astellas Pharma Inc. for the development and commercialization of FX-322, as well as collaboration and licensing agreements with Massachusetts Eye and Ear, the Massachusetts Institute of Technology, and The Scripps Research Institute. Frequency Therapeutics, Inc. was incorporated in 2014 and is headquartered in Lexington, Massachusetts.

Tonix Pharmaceuticals Holding Corp., a biopharmaceutical company, focuses on developing, discovering, commercializing, and licensing therapeutics to treat and prevent human disease and alleviate suffering. It markets Zembrace SymTouch and Tosymra for the treatment of acute migraine with or without aura in adults. Its development portfolio focuses on central nervous system disorders. The company's priority is to submit a New Drug Application (NDA) to the FDA for TNX-102 SL (cyclobenzaprine HCl sublingual tablet), which has completed two positive Phase 3 studies for the management of fibromyalgia. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition. Its TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted breakthrough therapy designation by the FDA. The company's rare disease development portfolio comprises TNX-2900, an intranasal potentiated oxytocin for the treatment of Prader-Willi syndrome (PWS). Its immunology development portfolio includes biologics to address organ transplant rejection, autoimmunity, and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. The company's infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases, including TNX-1800, in development as a vaccine to protect against COVID-19. Its infectious disease development portfolio also comprises TNX-3900 and TNX-4000, which are classes of broad-spectrum small molecule oral antivirals. The company was founded in 2007 and is headquartered in Chatham, New Jersey.

Verona Pharma plc, a clinical stage biopharmaceutical company, focuses on development and commercialization of therapies for the treatment of respiratory diseases with unmet medical needs. The company's product candidate is ensifentrine, an inhaled and dual inhibitor of the phosphodiesterase (PDE) 3 and PDE4 enzymes that acts as both a bronchodilator and an anti-inflammatory agent in a single compound, which is in Phase 3 clinical trials for the treatment of chronic obstructive pulmonary disease, asthma, and cystic fibrosis. It is developing ensifentrine in three formulations, including nebulizer, dry powder inhaler, and pressurized metered-dose inhaler. The company was incorporated in 2005 and is headquartered in London, the United Kingdom.