CRISPR Therapeutics
NASDAQ:CRSPCRISPR Therapeutics is a gene-editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene-editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit www.crisprtx.com.
Denali Therapeutics
NASDAQ:DNLIDenali Therapeutics Inc., a biopharmaceutical company, develops a portfolio of product candidates engineered to cross the blood-brain barrier for neurodegenerative diseases and lysosomal storage diseases in the United States. The company's transport vehicle (TV)-enabled programs include DNL310 ETV, an IDS enzyme replacement therapy program for MPS II; TAK-594/DNL593 which is in Phase 1/II for frontotemporal dementia-granulin; DNL126 program for MPS IIIA; and DNL622 for MPS I, as well as other preclinical programs that target amyloid beta and HER2. Its brain-penetrant small molecule programs comprise BIIB122/DNL151 LRRK2 inhibitor program for Parkinson's disease; SAR443820/DNL788 RIPK1 inhibitor program for CNS disease; DNL343 eIF2B Activator program for amyotrophic lateral sclerosis; and SAR443122/DNL758 RIPK1 inhibitor program for peripheral inflammatory diseases. It also provides early stage program include TAK-594/DNL593 program for FTD-GRN; DNL126 program for MPS IIIA, a Sanfilippo Syndrome A; DNL622 for MPS I which is Hurler syndrome; Antibody Transport Vehicle Amyloid beta program; Oligonucleotide Transport Vehicle platform, a novel class of biotherapeutics to address the root cause of diseases through modulation of gene expression; and other TV-enabled discovery programs. The company has collaboration agreements with Biogen MA Inc. and Biogen International GmbH; Genzyme Corporation; Takeda Pharmaceutical Company Limited; F-star Gamma Limited, F-star Biotechnologische Forschungs-und Entwicklungsges m.b.H, and F-star Biotechnology Limited; and Genentech, Inc. The company was formerly known as SPR Pharma Inc. and changed its name to Denali Therapeutics Inc. in March 2015. Denali Therapeutics Inc. was incorporated in 2013 and is headquartered in South San Francisco, California.
Neurocrine Biosciences
NASDAQ:NBIXNeurocrine Biosciences, Inc. discovers, develops, and markets pharmaceuticals for neurological, neuroendocrine, and neuropsychiatric disorders in the United States and internationally. The company's products include INGREZZA for tardive dyskinesia and chorea associated with Huntington's disease; ALKINDI for adrenal insufficiency; Efmody capsules for classic congenital adrenal hyperplasia; Orilissa tablets for endometriosis; and Oriahnn capsules to treat uterine fibroids. Its product candidates in clinical development include valbenazine to treat dyskinetic cerebral palsy in pediatrics and adults; NBI-921352 to treat developmental and epileptic encephalopathy syndrome in pediatrics and adults; NBI-827104 to treat epileptic encephalopathy with continuous spike-and-wave during sleep; NBI-1076986 to treat movement disorders; crinecerfront to treat congenital adrenal hyperplasia in adults and children; EFMODY to treat congenital adrenal hyperplasia and adrenal insufficiency in adults; valbenazine for the adjunctive treatment of schizophrenia; NBI-1065845 for the treatment of inadequate response to treatment in major depressive disorder; luvadaxistat to treat cognitive impairment related to schizophrenia; NBI-1117568 for the treatment of schizophrenia; NBI-1070770 to treat major depressive disorder; NBI-1117570 for the treatment of symptoms of psychosis and cognition in neurological and neuropsychiatric conditions; and NBI-1117569, NBI-1117567, and NBI-1065890 to treat CNS indications. The company also has license and collaboration agreements with Heptares Therapeutics Limited; Takeda Pharmaceutical Company Limited; Idorsia Pharmaceuticals Ltd; Xenon Pharmaceuticals Inc.; Voyager Therapeutics, Inc.; BIAL Portela & Ca, S.A.; Mitsubishi Tanabe Pharma Corporation; and AbbVie Inc. The company was incorporated in 1992 and is headquartered in San Diego, California.
Qiagen
NYSE:QGENQIAGEN N.V. offers sample to insight solutions that transform biological materials into molecular insights worldwide. The company provides primary sample technology consumables, such as nucleic stabilization and purification kits for primary sample materials, manual and automated processing for genotyping, gene expression, and viral and bacterial analysis, as well as silica membranes and magnetic bead technologies; secondary sample technology consumables, including kits and components for purification of nucleic acids from secondary sample materials; and instruments for nucleic acid purification and accessories. It also provides interferon-gamma release assay for TB testing, and assays for post-transplant testing and viral load monitoring; assays for prenatal testing and detection of sexually transmitted diseases and HPV, as well as assays for analysis of genomic variants, such as mutations, insertions, deletions, and fusions; and sample to insight instruments, including one-step molecular analysis of hard-to-diagnose syndromes, and integrated PCR testing. In addition, it offers PCR consumables, such as quantitative PCR, reverse transcription, and combinations kits for analysis of gene expression, genotyping and gene regulation, and running on QIAGEN instruments and technologies; human ID and forensics assay consumables, including STR assays for human ID, and assays for food contamination; PCR instruments consist of digital PCR and qPCR solutions; and developed and configured enzymes and PCR solutions. Further, the company provides predefined and custom NGS gene panels, library prep kits and components, and whole genome amplification; QIAGEN consumables and instruments, as well as bioinformatics solutions; and custom laboratory and genomic services. It serves molecular diagnostics, academia, pharmaceutical, and applied testing customers. The company was founded in 1984 and is headquartered in Venlo, the Netherlands.
Repligen
NASDAQ:RGENRepligen Corporation develops and commercializes bioprocessing technologies and systems for use in biological drug manufacturing process in North America, Europe, the Asia Pacific, and internationally. It offers Protein A ligands that are the binding components of Protein A affinity chromatography resins; and cell culture growth factor products. The company's chromatography products include OPUS pre-packed chromatography columns, which are used in the purification of biologics; and OPUS smaller-scale columns that are used in the high throughput process development screening, viral clearance validation studies, and scale down validation of chromatography processes. It also offers ELISA test kits; and chromatography resins under the CaptivA brand. In addition, the company provides filtration products, such as XCell Alternating Tangential Flow systems that are filtration devices used in upstream perfusion and cell culture processing; TangenX flat sheet cassettes, which are used in downstream biologic drug concentration, buffer exchange, and formulation processes; KrosFlo tangential flow filtration and tangential flow depth filtration systems; Spectra/Por laboratory and process dialysis products, and ProConnex TFDF flow paths. Further, it provides process analytics products, such as slope spectroscopy systems under the SoloVPE, FlowVPE, and FlowVPX brands. The company sells its products to life sciences, biopharmaceutical, and diagnostics companies; laboratory researchers; and contract manufacturing organizations. Repligen Corporation has collaboration agreements with Navigo Proteins GmbH to develop multiple affinity ligands. The company was incorporated in 1981 and is headquartered in Waltham, Massachusetts.