BioMarin Pharmaceutical Inc. engages in the development and commercialization of therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease. The company's commercial products also comprise Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia; and Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. In addition, it develops Roctavian, an adeno associated virus vector, for the treatment of patients with severe hemophilia A. The company serves specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. It has license and collaboration agreements with Sarepta Therapeutics; Ares Trading S.A.; Catalyst Pharmaceutical Partners, Inc.; and CAMP4 Therapeutics Corporation. The company was incorporated in 1996 and is headquartered in San Rafael, California.

Ascendis Pharma A/S, a biopharmaceutical company, focuses on developing therapies for unmet medical needs. It offers SKYTROFA for treating patients with growth hormone deficiency (GHD). The company is also developing a pipeline of three independent endocrinology rare disease product candidates in clinical development, as well as focuses on advancing oncology therapeutic candidates. The company was incorporated in 2006 and is headquartered in Hellerup, Denmark.

Zai Lab Limited develops and commercializes therapies to treat oncology, autoimmune disorders, infectious diseases, and neuroscience. Its commercial products include Zejula, an orally administered poly polymerase 1/2 inhibitor; Optune, a cancer therapy that uses electric fields tuned to specific frequencies to kill tumor cells; NUZYRA for acute bacterial skin and skin structure infections, and community acquired bacterial pneumonia; Qinlock to treat gastrointestinal stromal tumors, and VYVGART, a human IgG1 antibody fragment for myesthenia gravis. The company also develops Tumor Treating Fields, a portable device for delivery of electric fields; Repotrectinib, a tyrosine kinase inhibitor (TKI) to target ROS1 and TRK A/B/C in TKI-naïve- or -pretreated cancer patients; Tisotumab vedotin, an antibody drug conjugate; Adagrasib for treating KRAS-G12C-mutated NSCLC, colorectal cancer, and pancreatic cancer; and Bemarituzumab to treat gastric and gastroesophageal junction cancer patients. In addition, it develops Sulbactam/durlobactam, a combination of a beta-lactam antibiotic and a beta-lactamase inhibitor for the treatment of serious infections caused by Acinetobacter; KarXT for the treatment of psychiatric and neurological conditions. It has license and collaboration agreement with Tesaro, Inc. to develop, manufacture, and commercialize niraparib; NovoCure to develop and commercialize Tumor Treating Fields; Deciphera to develop and commercialize ripretinib; Paratek Bermuda Ltd. to develop, manufacture, and commercialize omadacycline; argenx, to develop and commercialize efgartigimod; BMS to develop and commercialize tisotumab vedotin and repotrectinib; Mirati to research, develop, manufacture, and commercialize adagrasib; Amgen to develop and commercialize bemarituzumab; and Innoviva to develop and commercialize Sulbactam-Durlobactam; Karuna to develop and commercialize KarXT; and strategic collaboration with Pfizer for XACDURO. The company was incorporated in 2013 and is headquartered in Shanghai, China.

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.

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.

Theratechnologies Inc., a biopharmaceutical company, focuses on the development and commercialization of various therapies to address the unmet medical needs in the United States, Canada, and Europe. The company offers EGRIFTA SV for the reduction of excess abdominal fat in human immunodeficiency virus (HIV)-infected patients with lipodystrophy; and Trogarzo for the treatment of HIV-1 infection in heavily treatment-experienced adults with multidrug-resistant HIV-1 infection failing their current antiretroviral regimen. Its pipeline products include F8 Formulation that is in Phase 2b/3 clinical trials for studying tesamorelin for the treatment of nonalcoholic steatohepatitis; multi -dose pen injector for the administration of tesamorelin; SORT1+ Technology Platform for the development of proprietary peptides for cancer drug development targeting SORT1 receptors; Sudocetaxel Zendusortide, which is in Phase 1 Clinical Trial for various solid tumor types, including HR+ breast, triple negative breast, ovarian, endometrial, melanoma, thyroid, small cell lung, and prostate cancers. The company was incorporated in 1993 and is headquartered in Montreal, Canada.

Grace Therapeutics, Inc. engages in the development and commercialization of pharmaceutical products for rare and orphan diseases in Canada. The company's lead product candidate is the GTX-104, an intravenous infusion to treat subarachnoid hemorrhage. It also develops GTX-102, an oral mucosal betamethasone spray for the treatment of ataxia-telangiectasia; and GTX-101, a topical bioadhesive film-forming bupivacaine spray for postherpetic neuralgia. The company was formerly known as Acasti Pharma Inc. and changed its name to Grace Therapeutics, Inc. in October 2024. The company was incorporated in 2002 and is headquartered in Princeton, New Jersey.

COSCIENS Biopharma Inc., a specialty biopharmaceutical company, engages in developing and commercializing therapeutics and diagnostic tests in Canada, Switzerland, Ireland, Denmark, Germany, the United States, and internationally. Its lead product is Macrilen (macimorelin), an orally available peptidomimetic ghrelin receptor (GHSR-1a) agonist that stimulates the secretion of growth hormone by binding to the GHSR-1a for the diagnosis of patients with adult growth hormone deficiency and childhood-onset growth hormone deficiency, as well as endocrinology and oncology indications; AEZS-150, a delayed clearance parathyroid hormone fusion polypeptide that is in preclinical trial for the treatment of chronic hypoparathyroidism; and AEZS-130 that is in preclinical trial for the treatment of amyotrophic lateral sclerosis. The company also has a license and research agreement with University of Wuerzburg to develop, manufacture, and commercialize AIM biologicals for the treatment of neuromyelitis optica spectrum disorder; and for pre-clinical development towards the potential treatment of Parkinson's disease. In addition, it has a license agreement with Consilient Health Ltd. and NK MEDITECH Ltd. for the development and commercialization of macimorelin; a distribution and commercialization agreement with Er-Kim Pharmaceuticals Bulgaria Eood for the commercialization of macimorelin for the diagnosis of growth hormone deficiency in children and adults; as well as The University of Sheffield, the United Kingdom for the development, manufacture, and commercialization of parathyroid hormone fusion polypeptides for the treatment of chronic hypoparathyroidism. The company was formerly known as Aeterna Zentaris Inc. and changed its name to COSCIENS Biopharma Inc. in August 2024. COSCIENS Biopharma Inc. was incorporated in 1990 and is headquartered in Toronto, Canada.