Vertex Pharmaceuticals Incorporated, a biotechnology company, engages in developing and commercializing therapies for treating cystic fibrosis (CF). It markets TRIKAFTA/KAFTRIO for people with CF with at least one F508del mutation for 2 years of age or older; SYMDEKO/SYMKEVI for people with CF for 6 years of age or older; ORKAMBI for CF patients 1 year or older; and KALYDECO for the treatment of patients with 1 year or older who have CF with ivacaftor. The company's pipeline includes VX-522, a CFTR mRNA therapeutic designed to treat the underlying cause of CF, which is in Phase 1 clinical trial; VX-548, a non-opioid medicine for the treatment of acute and neuropathic pain which is in Phase 3 clinical trial; Exa-cel, for the treatment of sickle cell disease and transfusion-dependent beta thalassemia which is in Phase 2/3 clinical trial. In addition, it provides inaxaplin for the treatment of APOL1-mediated focal segmental glomerulosclerosis and co-morbidities, such as hypertension which is in single Phase 2/3; VX- 880 and VX-264, treatment for Type 1 Diabetes which is in Phase 1/2 clinical trial; VX-970, which is in Phase 2 clinical trial for the treatment of cancer; and VX-803 and VX-984 for treatment of cancer in Phase 1 clinical trial. Further, it sells the products to specialty pharmacy and specialty distributors in the United States, as well as retail pharmacies, hospitals, and clinics. Additionally, the company has collaborations with CRISPR Therapeutics AG.; Moderna, Inc.; Entrada Therapeutics, Inc.; Arbor Biotechnologies, Inc.; Mammoth Biosciences, Inc.; and Verve Therapeutics., as well as collaborations with Tevard Biosciences to develop novel tRNA-based therapies for duchenne muscular dystrophy. Vertex Pharmaceuticals Incorporated was founded in 1989 and is headquartered in Boston, Massachusetts.

Xenon Pharmaceuticals Inc., a neuroscience-focused biopharmaceutical company, engages in the development of therapeutics to treat patients with neurological disorders in Canada. Its clinical development pipeline includes XEN1101, a novel and potent Kv7 potassium channel opener, which is in Phase 3 clinical trials for the treatment of epilepsy and other neurological disorders. The company has a license and collaboration agreement with the Neurocrine Biosciences, Inc. for the development of NBI-921352, a selective Nav1.6 sodium channel inhibitor that is in Phase 2 clinical trials for the treatment of SCN8A developmental and epileptic encephalopathy, and other indications, including adult focal epilepsy. Xenon Pharmaceuticals Inc. was incorporated in 1996 and is headquartered in Burnaby, Canada.

Aurinia Pharmaceuticals Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies to treat various diseases with unmet medical need in the United States. It offers LUPKYNIS for the treatment of adult patients with active lupus nephritis. It has a collaboration and license agreement with Otsuka Pharmaceutical Co., Ltd. The company was incorporated in 1993 and is headquartered in Edmonton, Canada.

Akebia Therapeutics, Inc., a biopharmaceutical company, focuses on the development and commercialization of therapeutics for patients with kidney diseases. The company's lead product investigational product candidate is Vafseo (vadadustat), an oral hypoxia-inducible factor prolyl hydroxylase, which is in Phase III development for the treatment of anemia due to chronic kidney disease (CKD) in dialysis-dependent and non-dialysis dependent patients. It offers Auryxia, a ferric citrate that is used to control the serum phosphorus levels in adult patients with DD-CKD on dialysis; and the treatment of iron deficiency anemia in adult patients with CKD not on dialysis. The company's product pipeline includes AKB-9090, a drug targeting critical-care indications; and AKB-10108, a drug targeting conditions related to premature birth. It has collaboration agreements with Mitsubishi Tanabe Pharma Corporation for the development and commercialization of vadadustat in Japan and other Asian countries, as well as research and license agreement with Janssen Pharmaceutica NV for the development and commercialization of hypoxia-inducible factor prolyl hydroxylase targeted compounds worldwide. Akebia Therapeutics, Inc. was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.

Xencor, Inc., a clinical stage biopharmaceutical company, focuses on the discovery and development of engineered monoclonal antibody and cytokine therapeutics to treat patients with cancer and autoimmune diseases. The company provides Sotrovimab that targets the SARS-CoV-2 virus; Ultomiris for the treatment of patients with paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome; and Monjuvi for the treatment of patients with relapsed or refractory diffuse large B-cell lymphoma. It develops Plamotamab, a bispecific antibody, which is in Phase I clinical trial to treat non-Hodgkin lymphoma; Vudalimab, a bispecific antibody, which is in Phase II clinical trial to treat metastatic castration-resistant prostate cancer and other solid tumor types. The company is also developing XmAb306, which is in Phase I clinical trial to treat solid tumors; XmAb104, which is in Phase II clinical trial to treat patients with selected solid tumors; XmAb564 that is in Phase Ia clinical trial to treat autoimmune diseases; AMG 509, which is in Phase I clinical trial to treat prostate cancer; XmAb819 for patients with renal cell carcinoma; XmAb541 for the treatment of ovarian cancer; and XmAb662 which is in Phase I clinical trial to treat patients with solid tumors. In addition, the company develops VIR-3434, which is in Phase II clinical trial for patients with hepatitis B virus infection; and VIR-2482 that is in Phase 2 clinical trial to trat hepatitis B virus. The company develops AIMab7195 to reduce blood serum levels of IgE that mediates allergic responses and allergic disease; Obexelimab to treat autoimmune disease; and Xpro1595 to treat patients with Alzheimer's disease, and depression. It has a license agreement with Caris Life Sciences. Xencor, Inc. was incorporated in 1997 and is headquartered in Pasadena, California.

Unicycive Therapeutics, Inc., a biotechnology company, develops treatments for various medical conditions in the United States. Its drug candidates include UNI 494, a pro-drug of Nicorandil that is being developed as a treatment for acute kidney injury; and Renazorb (Oxylanthanum Carbonate), which is being developed for the treatment of hyperphosphatemia in patients with chronic kidney disease. Unicycive Therapeutics, Inc. has license agreements with Shilpa Medicare Ltd; Sphaera Pharma Pte. Ltd.; Syneos Health LLC; Inotiv; Lee's Pharmaceutical (HK) Limited; Celerion; and Lotus International Pte Ltd. The company was incorporated in 2016 and is based in Los Altos, 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.

ZyVersa Therapeutics, Inc., a clinical stage biopharmaceutical company, develops and commercializes products for the treatment of renal and inflammatory diseases. The company develops drug development platforms, including Cholesterol Efflux Mediator VAR 200, an injectable drug, which is in Phase 2a clinical trial to the treatment of renal indications, such as focal segmental glomerulosclerosis, alport syndrome, and diabetic kidney diseases; and Inflammasome ASC Inhibitor IC 100, a humanized monoclonal antibody that is in preclinical stage for treatment of inflammatory diseases comprising acute respiratory syndrome, multiple sclerosis, IgA neuropathy, pancreatic cancer, Parkinson's and Huntington's disease, atherosclerosis, Alzheimer's disease, and obesity. ZyVersa Therapeutics, Inc. was founded in 2014 and is headquartered in Weston, Florida.