BridgeBio Pharma
NASDAQ:BBIOBridgeBio Pharma, Inc., a commercial-stage biopharmaceutical company, discovers, creates, tests, and delivers transformative medicines to treat patients who suffer from genetic diseases and cancers. Its products in development programs include AG10, a next-generation oral small molecule near-complete TTR stabilizer that is in Phase 3 clinical trial for the treatment of TTR amyloidosis, or transthyretin amyloid cardiomyopathy (ATTR-CM); low-dose infigratinib, an oral FGFR1-3 selective tyrosine kinase inhibitor, which is in Phase 3 double-blinded, placebo-controlled pivotal study for the treatment option for children with achondroplasia; and BBP-631, an AAV5 gene transfer product candidate that is in Phase 1/2 clinical trial for the treatment of congenital adrenal hyperplasia, or CAH, driven by 21-hydroxylase deficiency, or 21OHD. The company also develops Encaleret, a small molecule antagonist of the calcium sensing receptor, or CaSR, which is in phase 3 clinical trial for treating autosomal dominant hypocalcemia type 1, or ADH1; and BBP-418, a glycosylation substrate pro-drug that is in Phase 3 clinical trial for treating limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). In addition, it engages in developing products for mendelian, oncology, and gene therapy diseases. BridgeBio Pharma, Inc. has license and collaboration agreements with the Leland Stanford Junior University; and Leidos Biomedical Research, Inc. The company was founded in 2015 and is headquartered in Palo Alto, California.
Travere Therapeutics
NASDAQ:TVTXTravere Therapeutics, Inc., a biopharmaceutical company, identifies, develops, and delivers therapies to people living with rare kidney and metabolic diseases. Its products include FILSPARI (sparsentan), a once-daily, oral medication designed to target two critical pathways in the disease progression of IgA Nephropathy (endothelin 1 and angiotensin-II); and Thiola and Thiola EC (tiopronin tablets) for the treatment of cystinuria, a rare genetic cystine transport disorder that causes high cystine levels in the urine and the formation of recurring kidney stones. The company's clinical-stage programs consist of Sparsentan, a novel investigational product candidate, which has been granted Orphan Drug Designation for the treatment of focal segmental glomerulosclerosis in the U.S. and Europe; and Pegtibatinase (TVT-058), a novel investigational human enzyme replacement candidate being evaluated for the treatment of classical homocystinuria. It has a cooperative research and development agreement with National Institutes of Health's National Center for Advancing Translational Sciences and Alagille Syndrome Alliance for the identification of potential small molecule therapeutics for Alagille syndrome. The company was formerly known as Retrophin, Inc. and changed its name to Travere Therapeutics, Inc. in November 2020. Travere Therapeutics, Inc. was incorporated in 2008 and is headquartered in San Diego, California.
Verve Therapeutics
NASDAQ:VERVVerve Therapeutics, Inc., a clinical stage genetic medicines company, engages in developing gene editing medicines for patients to treat cardiovascular diseases in the United States. The company's lead product candidate is VERVE-101, a single-course gene editing treatment that permanently turns off the PCSK9 gene in the liver; and VERVE-102, a product candidate that targets the PCSK9 gene for the treatment of HeFH. It engages in the development of ANGPTL3 program to permanently turn off the ANGPTL3 gene in the liver. It has a collaboration and license agreement with Beam Therapeutics Inc.; a development and option agreement with Acuitas Therapeutics, Inc.; and a Cas9 license agreement with The Broad Institute and the President and Fellows of Harvard College. The company was formerly known as Endcadia, Inc. and changed its name to Verve Therapeutics, Inc. in January 2019. Verve Therapeutics, Inc. was incorporated in 2018 and is headquartered in Cambridge, Massachusetts.
Zentalis Pharmaceuticals
NASDAQ:ZNTLZentalis Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on discovering and developing small molecule therapeutics for the treatment of various cancers. Its products candidatures include the ZN-c3, an inhibitor of WEE1, a protein tyrosine kinase for the treatment of inhibitor for advanced solid tumors and hematological malignancies; in Phase 2 clinical trial as a monotherapy for the treatment of uterine serous carcinoma indications; Phase 2 clinical trial in Cyclin E1 driven high-grade serous ovarian cancer, fallopian tube, or primary peritoneal cancer; Phase 1/2 clinical trial in combination with PARPi for platinum-resistant ovarian cancer; Phase 1b clinical trial in combination with chemotherapy in platinum-resistant ovarian, and peritoneal or fallopian tube cancer; Phase 1/2 clinical trial in combination with chemotherapy in relapsed or refractory osteosarcoma; Phase 1/2 clinical trial with encorafenib and cetuximab for mutant metastatic colorectal cancer; and Phase 1/2 clinical trial in combination with chemotherapy in pancreatic cancer. The company's products pipeline also includes ZN-d5, an oral small molecule inhibitor of B-cell lymphoma 2, for hematological malignancies and related disorders; and BCL-xL heterobifunctional degrader, a member of the anti-apoptotic BCL-2 proteins, for solid tumors and hematological malignancies. Zentalis Pharmaceuticals, Inc. has licensing agreements and strategic collaborations with Recurium IP Holdings, LLC; Pfizer, Inc.; Zentera Therapeutics; Dana-Farber; and GlaxoSmithKline plc. The company was incorporated in 2014 and is headquartered in New York, New York.