Edgewise Therapeutics
NASDAQ:EWTXEdgewise Therapeutics, Inc., a biopharmaceutical company, discovers, develops, and commercializes therapies for the treatment of muscle disorders. Its lead product candidate, EDG-5506, an orally administered small molecule that is in Phase II clinical trials, designed to address the root cause of dystrophinopathies including Duchenne muscular dystrophy and Becker muscular dystrophy. The company develops EDG-7500, a small molecule for the treatment of hypertrophic cardiomyopathy and other severe cardiac disorders that is in Phase I clinical trials. In addition, it develops a pipeline of precision medicine product candidates that target key muscle proteins and modulators to address genetically defined muscle disorders. Edgewise Therapeutics, Inc. was incorporated in 2017 and is headquartered in Boulder, Colorado.
Seres Therapeutics
NASDAQ:MCRBSeres Therapeutics, Inc., a microbiome therapeutics company, develop microbiome therapeutics to treat the modulation of the colonic microbiome. It develops a novel class of biological drugs that are designed to treat by modulating the microbiome to restore health by repairing the function of a disrupted microbiome to a non-disease state. The company's lead product candidate is VOWST, an oral microbiome therapeutic that has completed Phase III clinical trial for the treatment of recurrent Clostridioides difficile infection. Its product pipeline also includes SER-155, an investigational oral fermented microbiome therapeutic which is in Phase 1b clinical trials for the treatment of gastrointestinal infections, bacteremia, and graft versus host disease in immunocompromised patients including patients receiving allogeneic hematopoietic stem cell transplantation. In addition, the company engages in the development of SER-287 which is in Phase 2b and SER-301 that is in Phase 1b to treat ulcerative colitis. Further, it has license Agreement with NHSc Rx License GmbH for the therapeutic products based on the microbiome technology, which includes VOWST product candidate, which is developed for the treatment of CDI and recurrent CDI; and collaboration license agreement with Société des Produits Nestlé S.A. (Nestlé) for the development and commercialization of certain product candidates for the treatment and management of CDI and inflammatory bowel disease including UC and Crohn's disease. The company was formerly known as Seres Health, Inc. and changed its name to Seres Therapeutics, Inc. in May 2015. Seres Therapeutics, Inc. was incorporated in 2010 and is headquartered in Cambridge, Massachusetts.
Precigen
NASDAQ:PGENPrecigen, Inc. operates as a discovery and clinical-stage biopharmaceutical company that develops gene and cell therapies using precision technology to target diseases in therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. It operates through two segments, Biopharmaceuticals and Exemplar. The company offers therapeutic platforms consisting of UltraCAR-T to provide chimeric antigen receptor T cell therapies for cancer patients; AdenoVerse immunotherapy, which utilizes a library of proprietary adenovectors for gene delivery of therapeutic effectors, immunomodulators, and vaccine antigen; and ActoBiotics for specific disease modification. It also develops programs based on the UltraCAR-T platform, including PRGN-3005 in Phase 1b clinical trial to treat advanced ovarian, fallopian tube, or primary peritoneal cancer; PRGN-3006 in Phase 1b trial for patients with relapsed or refractory acute myeloid leukemia and high-risk myelodysplastic syndromes; and PRGN-3007 in Phase 1/1b trial for the treatment of advanced receptor tyrosine kinase-like orphan receptor 1-positive, hematologic, and solid tumors. In addition, the company is developing programs based on the AdenoVerse immunotherapy platform comprising PRGN-2009 in Phase 2 trial for patients with HPV-associated cancer; and PRGN-2012 in Phase ½ trial to treat recurrent respiratory papillomatosis, as well as AG019, which is based on the ActoBiotics platform and in Phase 1b/2a trial, to treat type 1 diabetes mellitus. Further, it provides UltraPorator, a proprietary electroporation device; and develops research models and services for healthcare research applications. The company was formerly known as Intrexon Corporation and changed its name to Precigen, Inc. in February 2020. Precigen, Inc. was founded in 1998 and is headquartered in Germantown, Maryland.
Phathom Pharmaceuticals
NASDAQ:PHATPhathom Pharmaceuticals, Inc., biopharmaceutical company, focuses on developing and commercializing treatments for gastrointestinal diseases. The company has the rights in the United States, Europe, and Canada for an investigational potassium-competitive acid blocker (P-CAB) that blocks acid secretion in the stomach. It is also developing vonoprazan for the treatment of erosive gastroesophageal reflux disease; and in combination with antibiotics for the treatment of Helicobacter pylori infection. The company was incorporated in 2018 and is headquartered in Florham Park, New Jersey.
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.