Black Diamond Therapeutics
NASDAQ:BDTXBlack Diamond Therapeutics, Inc., a clinical-stage oncology medicine company, focuses on the discovery and development of MasterKey therapies for patients with genetically defined tumors. The company's lead product candidate is BDTX-1535, a brain-penetrant epidermal growth factor receptor MasterKey inhibitor, which is in phase 2 clinical trial for the treatment of epidermal growth factor receptor mutant non-small cell lung cancer, as well as phase 1 clinical trial to treat glioblastoma. It is also developing BDTX-4933, a brain-penetrant RAF MasterKey inhibitor targeting KRAS, NRAS, and BRAF alterations in solid tumors that is in phase 1 clinical trial. In addition, the company is developing BDTX-4876, a MasterKey inhibitor of oncogenic FGFR2/3 mutations with selectivity versus FGFR1/4, which is in preclinical stage. The company was formerly known as ASET Therapeutics, Inc. and changed its name to Black Diamond Therapeutics, Inc. in January 2018. Black Diamond Therapeutics, Inc. was incorporated in 2014 and is headquartered in Cambridge, Massachusetts.
Krystal Biotech
NASDAQ:KRYSKrystal Biotech, Inc., a commercial-stage biotechnology company, discovers, develops, and commercializes genetic medicines for patients with rare diseases in the United States. It commercializes VYJUVEK (beremagene geperpavec-svdt, or B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB). The company also develops KB105, which is in Phase 1/2 clinical trials for treating patients with deficient autosomal recessive congenital ichthyosis; KB104 for treating netherton syndrome; KB407 that is in Phase 1 clinical trials for treating cystic fibrosis; KB707 that is in Phase 1 clinical trials for the treatment of anti-PD-1 relapsed/refractory; KB408, which is in Phase 1 clinical trials for treating Alpha-1 antitrypsin deficiency; and KB301 that is in Phase 2 clinical trials for treating aesthetic skin conditions, as well as in open label study with ophthalmic B-VEC for treating for ocular complications of deb. Krystal Biotech, Inc. was founded in 2016 and is headquartered in Pittsburgh, Pennsylvania.
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.
Orchard Therapeutics
NASDAQ:ORTXOrchard Therapeutics plc, a gene therapy company, research, develops, and commercialize hematopoietic stem cell and gene therapies in the United Kingdom, Italy, France, and Germany. It offers OTL-200, an ex vivo autologous hematopoietic stem cell gene therapy for the treatment of patients with metachromatic leukodystrophy under the Libmeldy brand; and Strimvelis, a gammaretroviral vector-based gene therapy for the treatment of adenosine deaminase deficiency. The company's program for neurodegenerative disorders comprises clinical proof of concept-stage program, which includes OTL-203 for the treatment of mucopolysaccharidosis type I and OTL-201 for mucopolysaccharidosis type IIIA, or MPS-IIIA; and pre-clinical program, OTL-204 for the treatment of frontotemporal dementia with progranulin mutations. In addition, it develops program for immunological disorders consist of pre-clinical programs, which includes OTL-104 for Crohn's disease with mutations in the nucleotide-binding oligomerization domain-containing protein 2; and OTL-105 for the treatment of hereditary angioedema. The company was formerly known as Orchard Rx Limited. Orchard Therapeutics plc was founded in 2015 and is headquartered in London, the United Kingdom. As of January 24, 2024, Orchard Therapeutics plc operates as a subsidiary of Kyowa Kirin Co., Ltd..
SpringWorks Therapeutics
NASDAQ:SWTXSpringWorks Therapeutics, Inc., a commercial-stage biopharmaceutical company, engages in the development and commercialization of medicines for underserved patient populations suffering from rare diseases and cancer. Its lead product candidate is OGSIVEO (nirogacestat), an oral small molecule gamma secretase inhibitor that is in Phase III DeFi trial for the treatment of desmoid tumors; and Nirogacestat, is also in Phase 2 clinical development as a monotherapy for the treatment of ovarian granulosa cell tumors (GCT), a subtype of ovarian cancer. The company is also involved in the development of mirdametinib, an oral small molecule MEK inhibitor that is in Phase 2b clinical trials for the treatment of neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN); mirdametinib + lifirafenib, a combination therapy that is in Phase 1b clinical trial in patients with advanced or refractory solid tumors; and mirdametinib in monotherapy and combination approaches to treat other genetically defined solid tumors, including Phase 1/2 clinical trial for the treatment of pediatric and young adult patients with low-grade gliomas. In addition, it develops Brimarafenib (BGB-3245), an oral selective small molecule inhibitor of monomeric and dimeric forms of activating BRAF mutations. The company has collaborations with BeiGene, Ltd. and GlaxoSmithKline LLC; and license agreements with Pfizer Inc. for nirogacestat and mirdametinib. It also has a license agreement with Katholieke Universiteit Leuven and the Flanders Institute for Biotechnology for a portfolio of novel small molecule inhibitors of the TEA Domain; and Dana-Farber Cancer Institute for a portfolio of novel small molecule inhibitors of Epidermal Growth Factor Receptor. The company was founded in 2017 and is headquartered in Stamford, Connecticut.