Aerie Pharmaceuticals
NASDAQ:AERIAerie Pharmaceuticals, Inc., a pharmaceutical company, focuses on the discovery, development, and commercialization of ophthalmic therapies for open-angle glaucoma, dry eye, diabetic macular edema, and wet age-related macular degeneration in the United States. Its products include Rhopressa, a once-daily eye drop to reduce elevated intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension; and Rocklatan, a once-daily fixed-dose combination of Rhopressa and latanopros to reduce IOP to treat patients with open-angle glaucoma or ocular hypertension. The company also develops AR-15512 to treat signs and symptoms of dry eye; and AR-1105 and AR-14034 SR sustained-release implants focused on retinal diseases. It has a collaborative research, development, and licensing agreement with DSM Biomedical. Aerie Pharmaceuticals, Inc. was incorporated in 2005 and is headquartered in Durham, North Carolina.
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.
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.
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.