Genfit
NASDAQ:GNFTGenfit S.A., a late-stage biopharmaceutical company, discovers and develops drug candidates and diagnostic solutions for metabolic and liver-related diseases. The company develops Elafibranor, which is in Phase III clinical trial to treat patients with primary biliary cholangitis. It also engages in the development of NIS4 technology for the diagnosis of nonalcoholic steatohepatitis (NASH) and fibrosis; VS-01 for the treatment of Urea Cycle Disorder (UCD) and Organic Acidemia Disorder (OAD); GNS561, which is in Phase 1b/2a trial to treat patients with cholangiocarcinoma (CCA); VS-01-ACLF and Nitazoxanide (NTZ), which is in Phase 1 trial to treat acute-on-chronic liver failure, as well as VS-02-HE, which is in preclinical trial for the treatment of Reduction of Hyperammonemia and the Stabilization of Blood Ammonia; CML-022; SRT-015, an ASK1 inhibitor targets the inhibition of cellular apoptosis, inflammation, and fibrosis. The company has a licensing agreement with Labcorp for the commercialization of NASHnext, a blood-based molecular diagnostic test; and Genoscience Pharma to develop and commercialize the investigational treatment GNS561 for CCA. The company was incorporated in 1999 and is headquartered in Loos, France.
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.
Nightstar Therapeutics
NASDAQ:NITENightstar Therapeutics plc, a clinical-stage gene therapy company, focuses on developing and commercializing novel one-time treatments for patients suffering from rare inherited retinal diseases in the United Kingdom. The company's lead product candidate is NSR-REP1, a candidate that is in phase 3 clinical development for the treatment of patients with choroideremia. It is also developing NSR-RPGR, a candidate that is in Phase 1/2 clinical trials for the treatment of patients with X-linked retinitis pigmentosa, an inherited X-linked recessive retinal disease; and NSR-BEST1, a candidate that is in preclinical development stage for the treatment of best vitelliform macular dystrophy. In addition, the company is developing NSR-ABCA4, a candidate that is in preclinical development stage for the treatment of Stargardt disease. The company was founded in 2013 and is headquartered in London, the United Kingdom.
NanoString Technologies
NASDAQ:NSTGNanoString Technologies, Inc. develops, manufactures, and sells technology for scientific and clinical information in the fields of genomics and proteomics in the Americas, Europe, the Middle East, and the Asia Pacific. The company offers nCounter Analysis System, an automated, multi-application, digital detection, and counting system. It provides nCounter Pro and FLEX systems that include Prep Station, an automated liquid handling component that processes and prepares samples for data collection; and nCounter Digital Analyzer, which collects data from samples by taking images of the immobilized fluorescent reporters in the sample cartridge and processing the data into output files. The company also offers nCounter SPRINT Profiler, an instrument that provides liquid handling steps and the digital analysis through use of a microfluidic cartridge; and nSolver Analysis Software, a data analysis program that enables researchers to check, normalize, and analyze their data. In addition, it provides custom CodeSets; panels with oncology, immunology and infectious disease, and neuroscience applications. Further, the company offers Master Kits, cartridges, ancillary reagents, tips and reagent plates to setup and process samples in instruments; and Prosigna in vitro diagnostic kits. The company is also developing GeoMx DSP system to enable the field of spatial biology. It has collaboration with Lam Research Corporation for the development of NGS sequencing platform and related assays; and Parker Institute for Cancer Immunotherapy to develop CAR-T regimens that enhances patient outcomes for various types of cancer, as well as collaboration with Stanford Medicine, Acrobat Genomics, and Illumina Accelerator to discover new drug targets for gene editing-based therapeutics. NanoString Technologies, Inc. was incorporated in 2003 and is headquartered in Seattle, Washington. On February 4, 2024, NanoString Technologies, Inc., along with its affiliates, filed a voluntary petition for reorganization under Chapter 11 in the U.S. Bankruptcy Court for the District of Delaware.
Vericel
NASDAQ:VCELVericel Corporation, a commercial-stage biopharmaceutical company, engages in the research, development, manufacture, and distribution of cellular therapies for sports medicine and severe burn care markets in North America. The company markets autologous cell therapy products comprising MACI, an autologous cultured chondrocytes on porcine collagen membrane for the repair of symptomatic, and single or multiple full-thickness cartilage defects of the knee; Epicel, a permanent skin replacement humanitarian use device for the treatment of adult and pediatric patients with deep-dermal or full-thickness burns; and NexoBrid, a biological orphan product for eschar removal in adults with deep partial-thickness and/or full-thickness thermal burns. The company was formerly known as Aastrom Biosciences, Inc. Vericel Corporation was incorporated in 1989 and is headquartered in Cambridge, Massachusetts.