Crinetics Pharmaceuticals, Inc., a clinical-stage pharmaceutical company, focuses on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors. The company's lead product candidate is paltusotine, an oral selective nonpeptide somatostatin receptor type 2 agonist, which is in Phase 3 trial for the treatment of acromegaly; and Phase 2 trial for treating carcinoid syndrome associated with neuroendocrine tumors. It is also developing CRN04894, an investigational oral nonpeptide product candidate to antagonize the adrenocorticotrophic hormone (ACTH) receptor that has completed a Phase 1 study for the treatment of diseases caused by excess ACTH, including congenital adrenal hyperplasia and Cushing's disease. In addition, the company is developing antagonists of the parathyroid hormone (PTH) receptor for the treatment of primary hyperparathyroidism and humoral hypercalcemia of malignancy, and other diseases of excess PTH; identified investigational orally available somatostatin receptor type 3 targeted nonpeptide agonists for the treatment of autosomal dominant polycystic kidney disease; and developing thyroid-stimulating hormone receptor antagonists for the treatment of graves' disease and thyroid eye disease, as well as Oral GLP-1 and GIP nonpeptides for the treatment of diabetes and obesity. Crinetics Pharmaceuticals, Inc. was incorporated in 2008 and is headquartered in San Diego, California.

X4 Pharmaceuticals, Inc., a late-stage clinical biopharmaceutical company, focuses on the research, development, and commercialization of novel therapeutics for the treatment of rare diseases. Its lead product candidate is mavorixafor, a small molecule inhibitor of the chemokine receptor C-X-C chemokine receptor type 4 (CXCR4), which is in Phase III clinical trial for the treatment of patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome; and Phase II clinical trial to treat congenital, idiopathic, or cyclic neutropenia. The company is also developing X4P-003, a CXCR4 antagonist for the treatment of CXCR4 dependent disorders and primary immunodeficiencies; and X4P-002, a CXCR4 antagonist for the treatment of blood-brain barrier diseases. It has a license agreement with Abbisko Therapeutics Co Ltd. to develop, manufacture, and commercialize mavorixafor in combination with checkpoint inhibitors or other agents in oncology indications. The company is headquartered in Boston, Massachusetts.

Mereo BioPharma Group plc, a biopharmaceutical company, develops and commercializes therapeutics for the treatment of oncology and rare diseases in the United Kingdom, the United States, and internationally. It develops Etigilimab (MPH-313), an antibody T-cell immunoreceptor with Ig and ITIM domains, which is in Phase 1b clinical trial for the treatment of tumors. The company is also developing Navicixizumab (OMP-305B83), which has completed Phase 1b clinical trials for the treatment of the late line ovarian cancer; Acumapimod (BCT-197), a p38 MAP kinase inhibitor that is in Phase II clinical trials to treat acute exacerbations of chronic obstructive pulmonary disease; and Leflutrozole (BGS-649), an oral aromatase inhibitor for the treatment of hypogonadotropic hypogonadism which is in Phase 2 trials. In addition, it develops rare disease product candidates, including Setrusumab (BPS-804), an antibody for the treatment of osteogenesis imperfecta; and Alvelestat (MPH-966), an oral small molecule that is in Phase II clinical trial to treat Alpha-1 anti-trypsin deficiency. The company has a license agreement with Feng Biosciences for the development and commercialization of navicixizumab; license agreement with ReproNovo for the development and commercialization of leflutrozole; licensing agreement with AstraZeneca; and license agreement with Ultragenyx Pharmaceutical Inc. to develop and commercialize setrusumab. Mereo BioPharma Group plc was incorporated in 2015 and is headquartered in London, the United Kingdom.

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. It 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; MyoKardia, Inc. to discover and develop therapies for the treatment of genetic cardiomyopathies; and Sanofi for the development and commercialization of losmapimod, an oral small molecule being investigated for the treatment of facioscapulohumeral muscular dystrophy. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, 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.

Lumos Pharma, Inc., a clinical-stage biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of products and therapies for people with rare diseases. Its primary product candidate is LUM-201, an oral growth hormone secretagogue ibutamoren, which is in Phase III clinical trial for the treatment of idiopathic pediatric growth hormone deficiency and other rare endocrine disorders. The company has a clinical collaboration with Massachusetts General Hospital to evaluate oral LUM-201 in nonalcoholic fatty liver disease. Lumos Pharma, Inc. is headquartered in Austin, Texas. As of December 12, 2024, Lumos Pharma, Inc. was taken private.

MetaVia Inc., a clinical-stage biotechnology company focuses on developing and commercializing novel pharmaceuticals to treat cardiometabolic diseases. It develops DA-1241, a novel G-Protein-Coupled Receptor 119 agonist with development optionality as a standalone and/or combination therapy that is in Phase 2a clinical trial for metabolic dysfunction-associated steatohepatitis (MASH), and has completed Phase 1 clinical trial for the treatment of type 2 diabetes mellitus (T2DM); and DA-1726, a novel oxyntomodulin analogue functioning as a GLP-1 receptor and glucagon receptor dual agonist, which is in preclinical development for the treatment of obesity. The company's therapeutic programs include ANA001, a proprietary oral niclosamide formulation for the treatment of patients with moderate COVID-19; NB-01 for the treatment of painful diabetic neuropathy; NB-02 for the treatment of cognitive impairment; and Gemcabene for the treatment of dyslipidemia. It has a license agreement with Pfizer Inc. for the research, development, manufacture, and commercialization of Gemcabene; and joint research agreement with Dong-A ST and ImmunoForge for the development of DA-1726. The company was formerly known as NeuroBo Pharmaceuticals, Inc. and changed its name to MetaVia Inc. in November 2024. MetaVia Inc. is headquartered in Cambridge, Massachusetts.

MetaVia Inc., a clinical-stage biotechnology company focuses on developing and commercializing novel pharmaceuticals to treat cardiometabolic diseases. It develops DA-1241, a novel G-Protein-Coupled Receptor 119 agonist with development optionality as a standalone and/or combination therapy that is in Phase 2a clinical trial for metabolic dysfunction-associated steatohepatitis (MASH), and has completed Phase 1 clinical trial for the treatment of type 2 diabetes mellitus (T2DM); and DA-1726, a novel oxyntomodulin analogue functioning as a GLP-1 receptor and glucagon receptor dual agonist, which is in preclinical development for the treatment of obesity. The company's therapeutic programs include ANA001, a proprietary oral niclosamide formulation for the treatment of patients with moderate COVID-19; NB-01 for the treatment of painful diabetic neuropathy; NB-02 for the treatment of cognitive impairment; and Gemcabene for the treatment of dyslipidemia. It has a license agreement with Pfizer Inc. for the research, development, manufacture, and commercialization of Gemcabene; and joint research agreement with Dong-A ST and ImmunoForge for the development of DA-1726. The company was formerly known as NeuroBo Pharmaceuticals, Inc. and changed its name to MetaVia Inc. in November 2024. MetaVia Inc. is headquartered in Cambridge, Massachusetts.