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Annexon, Inc., a clinical-stage biopharmaceutical company, discovers and develops medicines for treating inflammatory-related diseases. Its lead candidate is ANX005, an investigational full-length monoclonal antibody, which is in Phase 3 clinical trial for the treatment of patients with guillain-barré syndrome; completed Phase II clinical trial for treating Huntington's disease; and in Phase II clinical trial for the treatment of amyotrophic lateral sclerosis. The company is also developing ANX007, an antigen-binding fragment (Fab) that is in Phase 3 program for the treatment of patients with geographic atrophy; and ANX1502, a novel oral small molecule inhibitor, which is in Phase 1 clinical trials for autoimmune indications. In addition, it develops ANX009, a C1q-blocking Fab that is in Phase I clinical trial for treating patients with lupus nephritis. The company was incorporated in 2011 and is headquartered in Brisbane, California.

Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy is under phase III clinical trial; and pociredir, a fetal hemoglobin inducer for the treatment of sickle cell disease and beta-thalassemia is under phase I clinical trial. The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases. Fulcrum Therapeutics, Inc. has collaboration and license agreement with Acceleron Pharma Inc. to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; and MyoKardia, Inc. to discover and develop therapies for the treatment of genetic cardiomyopathies. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Selecta Biosciences, Inc., a clinical-stage biopharmaceutical company, engages in the research and development of nanoparticle immunomodulatory drugs for the treatment and prevention of human diseases. The company's lead therapeutic gene therapy program is SEL-302 that is in Phase I clinical trial to enhance the treatment of methylmalonic acidemia. It is also developing biologic therapies, such as SEL-212 that is in Phase III clinical trials for the treatment of chronic refractory gout; and product candidates to treat IgA-mediated diseases, including IgA nephropathy, linear IgA bullous dermatitis, IgA pemphigus, and Henoch-Schonlein purpura. In addition, the company is developing gene therapies comprising for the treatment of pompe disease, duchenne muscular dystrophy, limb-girdle muscular dystrophy, lysosomal storage disorder, and other autoimmune diseases. Further, it develops tolerogenic therapies to treat autoimmune diseases. The company has license and collaboration agreements with Ginkgo Bioworks Holdings, Inc.; Genovis AB (publ.); Cyrus Biotechnology, Inc.; IGAN Biosciences, Inc.; Astellas Therapeutics, Inc.; Takeda Pharmaceuticals USA, Inc.; Swedish Orphan Biovitrum AB (publ.); Sarepta Therapeutics, Inc.; Asklepios Biopharmaceutical, Inc.; Massachusetts Institute of Technology; and Shenyang Sunshine Pharmaceutical Co., Ltd. Selecta Biosciences, Inc. was incorporated in 2007 and is headquartered in Watertown, Massachusetts.

Zevra Therapeutics, Inc., a rare disease company melding science, discovers and develops various proprietary prodrugs to treat serious medical conditions in the United States. The company utilizes its Ligand Activated Therapy technology to generate improved prodrug versions of FDA-approved drugs, as well as to generate prodrug versions of existing compounds that may have applications for new disease indications. Its prodrug product candidate pipeline is focused on the high need areas of attention deficit hyperactivity disorder, stimulant use disorder, and CNS rare diseases, including idiopathic hypersomnia (IH). The company's lead product candidate KP1077, which is under Phase II clinical trial for the treatment of IH and narcolepsy, is based on its prodrug of d-methylphenidate, known as serdexmethylphenidate. It is also developing KP879, a prodrug product candidate for the treatment of stimulant use disorder and is under Phase II clinical trial. In addition, the company has received FDA approval for AZSTARYS, a once-daily treatment for attention deficit hyperactivity disorder in patents age six years and older, and for APADAZ, an immediate-release combination product containing benzhydrocodone, a prodrug of hydrocodone, and acetaminophen. The company's product pipeline include, arimoclomol It has collaboration and license agreement with KVK-Tech, Inc. and Commave Therapeutics SA. The company was formerly known as KemPharm, Inc. and changed its name to Zevra Therapeutics, Inc. in February 2023. Zevra Therapeutics, Inc. was incorporated in 2006 and is headquartered in Celebration, Florida.