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Amicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering medicines for rare diseases. Its commercial product and product candidates include Galafold, an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant; and Pombiliti + Opfolda, for the treatment of late onset. It has collaboration and license agreements with the University of Pennsylvania to research and develop parvovirus gene therapy products; and GlaxoSmithKline. Amicus Therapeutics, Inc. was incorporated in 2002 and is headquartered in Princeton, New Jersey.

Karuna Therapeutics, Inc., a clinical-stage biopharmaceutical company, creates and delivers transformative medicines for people living with psychiatric and neurological conditions. Its lead product candidate includes KarXT, an oral modulator of muscarinic receptors for the treatment of acute psychosis in patients with schizophrenia; and for the treatment of central nervous system disorders, such as negative and cognitive symptoms of schizophrenia and psychosis, as well as for the treatment of various peripheral tissues and dementia-related psychosis, including Alzheimer's disease. The company also focuses on developing other muscarinic-targeted drug candidates; and intends to develop lead candidature TRPC4/5 and KAR-2618 for the treatment of depressive disorder. Karuna Therapeutics, Inc. has a license agreement with Eli Lilly and Company, and Zai Lab (Shanghai) Co., Ltd; patent license agreement with PureTech Health LLC; and drug discovery partnership with Charles River Laboratories, as well as drug discovery collaboration with PsychoGenics, Inc. The company was formerly known as Karuna Pharmaceuticals, Inc. and changed its name to Karuna Therapeutics, Inc. in March 2019. Karuna Therapeutics, Inc. was incorporated in 2009 and is headquartered in Boston, Massachusetts.

Ra Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops therapeutics for the treatment of diseases caused by excessive or uncontrolled activation of the complement system. The company's peptide chemistry platform enables the production of synthetic macrocyclic peptides that combine the diversity and specificity of antibodies with the pharmacological properties of small molecules. Its lead product candidate is Zilucoplan, an injection into the tissue under the skin that has completed Phase II clinical trial for the treatment of generalized myasthenia gravis (gMG); has completed Phase II clinical trial for treating paroxysmal nocturnal hemoglobinuria (PNH); and has completed Phase Ib clinical trial to treat patients with renal impairment. The company's pre-clinical programs include Factor D inhibition for treating C3 glomerulonephritis and dense deposit disease; and inhibitors of other complement factors for renal, autoimmune, and central nervous system diseases. It has collaboration and license agreement with Merck & Co., Inc. to identify orally available cyclic peptides for non-complement program targets, and provide research and development services. Ra Pharmaceuticals, Inc. was founded in 2008 and is headquartered in Cambridge, Massachusetts.

Synthorx, Inc., a biopharmaceutical company, focuses on the development of cytokine Synthorin programs for the treatment of cancer and autoimmune disorders in the United States. The company's lead product candidate is THOR-707, a variant of recombinant human IL-2 that is in the development in various solid tumor types as a single agent and in combination with an immune checkpoint inhibitor. It also develops IL-2 Synthorin for autoimmune indications; IL-10 Synthorin, a naturally occurring immune cell growth factor in humans for the treatment of immuno-oncology (IO); and IL-15 Synthorins, an immunoregulatory cytokine to treat IO. In addition, the company develops other Synthorin programs targeting undisclosed cytokines that play critical roles in the orchestration of anti-tumor responses by innate and adaptive immune cells. The company was formerly known as Alinos, Inc. and changed its name to Synthorx, Inc. in March 2014. Synthorx, Inc. was founded in 2014 and is headquartered in La Jolla, California.

Zogenix, Inc. is a pharmaceutical company. The firm engages in the development and commercialization of central nervous system (CNS) therapies and products for the treatment orphan diseases and other CNS disorders. Its products include Fintepla and MT1621. The company was founded by Stephen James Farr, Cam L. Garner, Roger L. Hawley, Bret E. Megargel, Jonathan M. N. Rigby, Scott L. Glenn, and John J. Turanin on May 11, 2006 and is headquartered in Emeryville, CA.