Curis
NASDAQ:CRISCuris, Inc., a biotechnology company, engages in the discovery and development of drug candidates for the treatment of human cancers in the United States. Its clinical stage drug candidates include Emavusertib, an oral small molecule IRAK4 kinase inhibitor, which is in a Phase 1/2 open-label, single arm expansion trial in patients with relapsed or refractory, or R/R, AML and high-risk myelodysplastic syndromes. The company's pipeline also includes Fimepinostat, an oral dual inhibitor of HDAC and PI3K enzymes for the treatment of patients with relapsed or refractory diffuse large B-cell lymphoma; CA-170, an oral, small molecule antagonist designated as CA-170 that selectively targets PD-L1 and VISTA; and CA-327, an oral, small molecule, TIM3/PD-L1, which is a molecule antagonist of PD-L1 and TIM3. It has collaboration agreement with Genentech Inc., or Genentech and F. Hoffmann-La Roche Ltd, or Roche, for the commercialization of Erivedge, an orally-administered small molecule hedgehog signaling pathway antagonist for the treatment of advanced basal cell carcinoma, or BCC; Aurigene Discovery Technologies Limited for the discovery, development, and commercialization of small molecule compounds in the areas of immuno-oncology and precision oncology; and also licensed four programs under the Aurigene collaboration, including emavusertib. Curis, Inc. was incorporated in 2000 and is headquartered in Lexington, Massachusetts.
DBV Technologies
NASDAQ:DBVTDBV Technologies S.A., a clinical-stage biopharmaceutical company, engages in the research and development of epicutaneous immunotherapy products. Its product pipeline comprising Viaskin Peanut, an immunotherapy product, which has completed Phase 3 clinical trial for the treatment of peanut allergies; and Viaskin Milk which is in Phase 1/2 clinical trial for the treatment of immunoglobulin E (IgE) mediated or cow's milk protein allergy and eosinophilic esophagitis. The company's earlier stage research programs include vaccine for the respiratory syncytial virus, potential treatments for inflammatory bowel disease, celiac disease, and type I diabetes. In addition, it develops Viaskin technology platform, a platform to potentially treat food allergy. The company has a collaboration with Nestlé Health Science to develop MAG1C, a ready-to-use atopy patch test for the diagnosis of non-IgE mediated CMPA in infants and toddlers. DBV Technologies S.A. was incorporated in 2002 and is headquartered in Montrouge, France.
Precision BioSciences
NASDAQ:DTILPrecision BioSciences, Inc., an advanced gene editing company, develops in vivo gene editing therapies for gene edits, including gene elimination, insertion, and excision in the United States. The company offers ARCUS, a genome editing platform to DNA genome insertion, deletion, and repair. It also provides PBGENE-HBV for the treatment of chronic hepatitis B virus (HBV) to eliminate covalently closed circular DNA with direct cuts and edits as well as to inactivate integrated HBV DNA with the goal of long-lasting reductions in hepatitis B surface antigen; PBGENE-PMM for the treatment of m.3243 associated primary mitochondrial myopathy (PMM) which is expected to submit an IND and/or CTA. In addition, it develops PBGENE-NVS for sickle cell disease/beta thalassemia for insertion; PBGENE-DMD (excision) for duchenne muscular dystrophy; PBGENE-LL2 (insertion), a liver directed target; PBGENE-LL3, a central nervous system directed target; and iECURE-OTC (insertion) for ornithine transcarbamylase deficiency. The company has license and collaboration agreement with Caribou Biosciences, Inc.; license agreement with TG Cell Therapy, Inc. to develop, manufacture, and commercialize azer-cel for autoimmune diseases and other indications outside of cancer; development and license agreement with Eli Lilly and Company for the research and development of potential in vivo therapies for genetic disorders; Cellectis S.A.; iECURE, Inc. to develop ARCUS-based gene-insertion therapies; Duke University; and Novartis Pharma AG to discover and develop in vivo gene editing products. Precision BioSciences, Inc. was incorporated in 2006 and is headquartered in Durham, North Carolina.
MeiraGTx
NASDAQ:MGTXMeiraGTx Holdings plc, a clinical stage gene therapy company, focusing on developing treatments for patients with serious diseases. The company develops various therapies for ocular diseases, including inherited retinal diseases and large degenerative ocular diseases, neurodegenerative diseases, and xerostomia. Its programs in clinical development include Phase I/II clinical stage programs in achromatopsia, X-linked retinitis pigmentosa, and RPE65-deficiency; Phase I clinical trials for radiation-induced xerostomia; and Parkinson's program that has completed a Phase II trial. It has a research collaboration agreement with Janssen Pharmaceuticals, Inc. for the research, development, and commercialization of gene therapies for the treatment of inherited retinal disease. The company was incorporated in 2015 and is based in New York, New York.
Solid Biosciences
NASDAQ:SLDBSolid Biosciences Inc., a life science company, develops therapies for neuromuscular and cardiac diseases in the United States. The company's lead product candidate is SGT-003, a gene transfer candidate for the treatment of Duchenne muscular dystrophy; and SGT-501 to treat Catecholaminergic polymorphic ventricular tachycardia. It is also developing AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy; SGT-601 to treat a rare cardiac disease characterized by mutations in the gene that codes for cardiac troponin T protein, which helps coordinate contraction of the heart muscle; SGT-701 for treatment of rare inherited disease characterized by mutations in the RBM20 gene, a cardiac splicing factor that regulates alternative splicing, and codes for RNA binding motif protein 20; and AVB-202-TT to treat Friedreich's ataxia, as well as other drugs for the treatment of cardiac and other diseases. In addition, the company is involved in developing platform technologies, including capsid libraries and dual gene expression, a technology for packaging multiple transgenes into one capsid. The company has collaboration and license agreement with Ultragenyx Pharmaceutical Inc. to develop and commercialize new gene therapies for Duchenne; and a license agreement with the University of Washington, University of Missouri, and University of Florida. Solid Biosciences Inc. was incorporated in 2013 and is headquartered in Charlestown, Massachusetts.