Allogene Therapeutics
NASDAQ:ALLOAllogene Therapeutics, Inc., a clinical stage immuno-oncology company, develops and commercializes genetically engineered allogeneic T cell therapies for the treatment of cancer. It develops, manufactures, and commercializes UCART19, an allogeneic chimeric antigen receptor (CAR) T cell product candidate for the treatment of pediatric and adult patients with R/R CD19 positive B-cell acute lymphoblastic leukemia (ALL). The company also develops cemacabtagene ansegedleucel, an engineered allogeneic CAR T cell product candidate that targets CD19 for the treatment of large B-cell lymphoma; and is in Phase 1b clinical trial for the treatment of chronic lymphocytic leukemia. In addition, it is developing ALLO-715, an allogeneic CAR T cell product candidate that is in a Phase 1 clinical trial for treating R/R multiple myeloma; ALLO-605, an allogeneic CAR T cell product candidate that is in a Phase I clinical trial for the treatment of multiple myeloma; ALLO-647, an anti-CD52 monoclonal antibody; CD70 to treat renal cell cancer; ALLO-316, an allogeneic CAR T cell product candidate that is in Phase 1 clinical trial for the treatment of advanced or metastatic RCC; ALLO-329 for the treatment of certain autoimmune diseases; DLL3 for the treatment of small cell lung cancer and other aggressive neuroendocrine tumors; and Claudin 18.2 for the treatment of gastric and pancreatic cancer. The company has license and collaboration agreements with Pfizer Inc.; Servier; Cellectis S.A.; and Notch Therapeutics Inc. It also has a strategic collaboration agreement with The University of Texas MD Anderson Cancer Center for the preclinical and clinical investigation of allogeneic CAR T cell product candidates; and a strategic partnership with Foresight Diagnostics to develop MRD-based In-Vitro Diagnostic for use in ALPHA3. The company was incorporated in 2017 and is headquartered in South San Francisco, California.
bluebird bio
NASDAQ:BLUEbluebird bio, Inc., a biotechnology company, researches, develops, and commercializes gene therapies for severe genetic diseases. Its product candidates for severe genetic diseases include ZYNTEGLO (betibeglogene autotemcel) for the treatment of transfusion-dependent ß-thalassemia; lovotibeglogene autotemcel for the treatment of sickle cell disease (SCD); and SKYSONA (elivaldogene autotemcel) to treat cerebral adrenoleukodystrophy. The company's clinical development programs include HGB-205, HGB-206, and HGB-210 to evaluate the safety and efficacy of lovo-cel in the treatment of patients with SCD; and HGB-204, HGB-205, HGB-207, and HGB-212 to evaluate the safety and efficacy of beti-cel in the treatment of patients with ß-thalassemia. It has license agreement with Orchard Therapeutics Limited. The company was formerly known as Genetix Pharmaceuticals, Inc., and changed its name to bluebird bio, Inc. in September 2010. bluebird bio, Inc. was incorporated in 1992 and is headquartered in Somerville, Massachusetts.
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.
Halozyme Therapeutics
NASDAQ:HALOHalozyme Therapeutics, Inc., a biopharma technology platform company, researches, develops, and commercializes proprietary enzymes and devices in the United States, Switzerland, Belgium, Japan, and internationally. The company's products are based on the patented recombinant human hyaluronidase enzyme (rHuPH20) that enables delivery of injectable biologics, such as monoclonal antibodies and other therapeutic molecules, as well as small molecules and fluids. It offers Hylenex recombinant, a formulation of rHuPH20 to facilitate subcutaneous (SC) fluid administration for achieving hydration to enhance the dispersion and absorption of other injected drugs in SC urography and to enhance resorption of radiopaque agents; rilpivirine, cabotegravir, and N6LS BNAB for the treatment of HIV; ocrelizumab for multiple sclerosis; XYOSTED, an injection for SC administration of testosterone replacement therapy; and ATRS-1902, a proprietary drug device combination product. The company also provides Herceptin (trastuzumab), Herceptin Hylecta, and Phesgo to treat breast cancer; Mabthera SC for the treatment of chronic lymphocytic leukemia; HYQVIA to treat primary immunodeficiency disorders; and DARZALEX for patients with amyloidosis, smoldering myeloma, and multiple myeloma. In addition, it offers Epinephrine Injection to treat allergic reactions; nivolumab+relatlimab and ANTI-TIM3 for the treatment of solid tumors; ARGX-117 for multifocal motor neuropathy; atezolizumab; nivolumab; afgartigimod; teriparatide injections; and OTREXUP, a SC methotrexate injection for adults with severe active rheumatoid arthritis and severe recalcitrant psoriasis, as well as children with active polyarticular juvenile idiopathic arthritis. Further, the company provides ATRS-1902 for adrenal crisis rescue; ARGX-113; and ARGX-117 to treat severe autoimmune diseases in multifocal motor neuropathy. Halozyme Therapeutics, Inc. was founded in 1998 and is headquartered in San Diego, California.