Crinetics Pharmaceuticals
NASDAQ:CRNXCrinetics 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.
Praxis Precision Medicines
NASDAQ:PRAXPraxis Precision Medicines, Inc., a clinical-stage biopharmaceutical company, engages in the development of therapies for central nervous system disorders characterized by neuronal excitation-inhibition imbalance. It is developing ulixacaltamide, a small molecule inhibitor of T-type calcium channels that is in Phase III clinical trial for the treatment of essential tremor; PRAX-562 for the treatment of pediatric patients with developmental and epileptic encephalopathies (DEE); and PRAX-628 to treat focal epilepsy. The company also develops PRAX-222 for the treatment of pediatric patients with early-onset SCN2A-DEE; PRAX-020 to treat KCNT1 related epilepsies; PRAX-080 for the treatment of PCDH19; and PRAX-090 and PRAX-100 for SYNGAP1 and SCN2A-LoF. It has a license agreement with RogCon Inc.; a research collaboration and license agreement with Ionis Pharmaceuticals, Inc.; a strategic collaboration and license agreement with UCB Biopharma SRL; and collaboration with The Florey Institute to develop three novel ASOs. The company was incorporated in 2015 and is based in Boston, Massachusetts.
Silence Therapeutics
NASDAQ:SLNSilence Therapeutics plc, a biotechnology company, focuses on the discovery and development novel molecules incorporating short interfering ribonucleic acid (siRNA) to inhibit the expression of specific target genes in hematology, cardiovascular, and rare diseases. The company's mRNAi GalNAc Oligonucleotide Discovery platform consists of precision engineered product candidates designed to target specific disease-associated genes in the liver. The company develops Zerlasiran (SLN360), which is in phase 2 clinical trial for cardiovascular disease associated with elevated lipoprotein(a); and Divesiran (SLN124), an siRNA molecule that is in Phase I clinical trials for the treatment of genetic hematological conditions, including polycythemia vera. It is also developing SLN-COMP-1 and SLN-COMP-2 for complement-mediated diseases; and SLN-HAN-1 and SLN-HAN-2. It has collaboration agreements with AstraZeneca PLC to discover, develop, and commercialize siRNA therapeutics for the treatment of cardiovascular, renal, metabolic, and respiratory diseases; Hansoh Pharmaceutical Group Company Limited to develop siRNAs; and Mallinckrodt Pharma IP Trading DAC to develop and commercialize RNAi drug targets designed to silence the complement cascade in complement-mediated disorders. The company was formerly known as SR Pharma plc and changed its name to Silence Therapeutics plc in May 2007. Silence Therapeutics plc was incorporated in 1994 and is headquartered in London, the United Kingdom.
Xenon Pharmaceuticals
NASDAQ:XENEXenon Pharmaceuticals Inc., a clinical-stage biopharmaceutical company, engages in developing therapeutics to treat patients with neurological disorders in Canada. Its clinical development pipeline includes XEN496, a Kv7 potassium channel opener that is Phase III clinical trials for the treatment of KCNQ2 developmental and epileptic encephalopathy; and XEN1101, a Kv7 potassium channel opener, which is in Phase II clinical trial for the treatment of epilepsy and other neurological disorders. The company's product candidates also comprise NBI-921352, a selective Nav1.6 sodium channel inhibitor that is in Phase II clinical trials for the treatment of SCN8A developmental and epileptic encephalopathy, and other indications, including adult focal epilepsy. It has a license and collaboration agreement with the Neurocrine Biosciences, Inc. to develop treatments for epilepsy. The company was incorporated in 1996 and is headquartered in Burnaby, Canada.
Zomedica
NYSEAMERICAN:ZOMZomedica Corp., a veterinary health company, focuses on the unmet needs of clinical veterinarians by developing products for companion animals. The company offers diagnostic products comprising TRUFORMA Bulk Acoustic Wave point of care diagnostic platform; TRUVIEW, a digital cystoscopy platform that offers automated slide preparation within the instrument; and VetGuardian, a zero-touch vital signs remote monitoring system. It also provides therapeutic products, including PulseVet electrohydraulic shockwave therapy platform; and Assisi Loop line of products, including the Assisi Loop, Assisi Loop Lounge, and DentaLoop devices that treat pain and inflammation through delivery of targeted pulsed electromagnetic field focused energy. The company has collaboration agreements with Celsee, Inc. for the development and commercialization of liquid biopsy assays and related consumables for the detection of cancer in companion animals; and Seraph Biosciences, Inc. for the development of a novel pathogen detection system. The company was formerly known as Zomedica Pharmaceuticals Corp. and changed its name to Zomedica Corp. in October 2020. Zomedica Corp. is headquartered in Ann Arbor, Michigan.