Agios Pharmaceuticals
NASDAQ:AGIOAgios Pharmaceuticals, Inc., a biopharmaceutical company, discovers and develops medicines in the field of cellular metabolism in the United States. Its lead product includes PYRUKYND (mitapivat), an activator of wild-type and mutant pyruvate kinase (PK), enzymes for the treatment of hemolytic anemias. The company develops AG-946, a PK activator for treating lower-risk myelodysplastic syndrome and hemolytic anemias; and AG-181, a phenylalanine hydroxylase stabilizer for the treatment of phenylketonuria. Its preclinical product is siRNA for the treatment of polycythemia vera, a rare blood disorder. Agios Pharmaceuticals, Inc. was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.
Arrowhead Pharmaceuticals
NASDAQ:ARWRArrowhead Pharmaceuticals, Inc. develops medicines for the treatment of intractable diseases in the United States. The company's products in pipeline includes Plozasiran, which is in Phase 2b and one Phase 3 clinical trial to treat hypertriglyceridemia, mixed dyslipidemia, and chylomicronemia syndrome; Zodasiran that is in Phase 2b clinical trial for the treatment of dyslipidemia and hypertriglyceridemia; ARO-PNPLA3, which is in Phase 1 clinical trial to treat patients with non-alcoholic steatohepatitis; ARO-RAGE that is in Phase 1/2a clinical trial to treat inflammatory pulmonary conditions; and ARO-MUC5AC, which is in Phase 1/2a clinical trial to treat muco-obstructive pulmonary diseases. It also develops ARO-MMP7 that is in Phase 1/2a clinical trial for treatment of idiopathic pulmonary fibrosis; ARO-DUX4 for the treatment of facioscapulohumeral muscular dystrophy; ARO-SOD1 for the potential treatment of amyotrophic lateral sclerosis; and ARO-C3, which is in Phase 1/2a clinical trial for the treatment of patients with various complement mediated or complement associated renal diseases. In addition, the company is involved in the development of JNJ-3989, which is in Phase 2 clinical trial to treat chronic hepatitis B virus infection; Olpasiran that is in Phase 3 clinical trial to reduce the production of apolipoprotein A; GSK-4532990 that is in phase 2 clinical trial to treat liver diseases; HZN-457, which is in phase 1 clinical trial to treat uncontrolled gout; and Fazirsiran that is in Phase 3 clinical trial for the treatment for liver disease associated with alpha-1 antitrypsin deficiency. Arrowhead Pharmaceuticals, Inc. has license and research collaboration agreements with Janssen Pharmaceuticals, Inc.; Takeda Pharmaceutical Company Limited; Horizon Therapeutics Ireland DAC; Amgen Inc.; and Glaxosmithkline Intellectual Property (No. 3) Limited. The company was founded in 2003 and is headquartered in Pasadena, California.
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.
PTC Therapeutics
NASDAQ:PTCTPTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to patients with rare disorders in the United States and internationally. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder; Tegsedi and Waylivra for the treatment of rare diseases; and Evrysdi to treat spinal muscular atrophy (SMA) in adults and children. Its development pipeline products include Sepiapterin for the treatment of phenylketonuria; PTC518 splicing platform, which is being developed for the treatment of Huntington's disease; and ferroptosis and inflammation platforms, including vatiquinone to treat Friedreich ataxia and utreloxastat for the treatment of amyotrophic lateral sclerosis. The company distributes its products through third-party distributors. It has collaborations with F. Hoffman-La Roche Ltd, Hoffman-La Roche Inc., the SMA Foundation, National Taiwan University, Akcea Therapeutics, Inc., and Shiratori Pharmaceutical Co., Ltd. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in South Plainfield, New Jersey.
Schrödinger
NASDAQ:SDGRSchrödinger, Inc., together with its subsidiaries, develops physics-based computational platform that enables discovery of novel molecules for drug development and materials applications. The company operates in two segments, Software and Drug Discovery. The Software segment is focused on licensing its software to transform molecular discovery for life sciences and materials science industries. The Drug Discovery segment focuses on building a portfolio of preclinical and clinical programs, internally and through collaborations. The company serves biopharmaceutical and industrial companies, academic institutions, and government laboratories worldwide. Schrödinger, Inc. was incorporated in 1990 and is based in New York, New York.