CRISPR Therapeutics AG, a gene editing company, focuses on developing transformative gene-based medicines for serious human diseases. It develops its products using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9), a gene editing technology that allows for precise directed changes to genomic DNA. It has a portfolio of therapeutic programs in a range of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases. The company's lead product candidate is CTX001, an ex vivo CRISPR gene-edited therapy for treating patients suffering from transfusion-dependent beta thalassemia or severe sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells. It is also developing CTX110, a donor-derived gene-edited allogeneic CAR-T therapy targeting cluster of differentiation 19 positive malignancies. In addition, the company is developing allogeneic CAR-T programs comprising CTX120 targeting B-cell maturation antigen for the treatment of multiple myeloma; and CTX130 for the treatment of solid tumors and hematologic malignancies. Further, it engages in developing regenerative medicine programs in diabetes; and in vivo and other genetic disease programs to treat glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. The company has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland.
CRISPR Therapeutics AG
Editas Medicine, Inc. operates as a clinical stage genome editing company. The company focuses on developing transformative genomic medicines to treat a range of serious diseases. The company develops a proprietary genome editing platform based on CRISPR technology, which includes CRISPR/Cas9, CRISPR/Cas12a, and engineered forms of both of these CRISPR systems to target genetically addressable diseases and therapeutic areas. It also develops EDIT-101, which is in Phase 1/2 clinical trial for the treatment of Leber Congenital Amaurosis type 10, a genetic form of vision loss that leads to blindness in childhood. In addition, the company develops EDIT-102 for the treatment of Usher Syndrome 2A, which is a form of retinitis pigmentosa that also includes hearing loss; autosomal dominant retinitis pigmentosa 4, a progressive form of retinal degeneration; and EDIT-301 treat sickle cell disease and beta-thalassemia. It has a research collaboration with Juno Therapeutics, Inc. to develop engineered T cells for cancer; strategic alliance and option agreement with Allergan Pharmaceuticals International Limited to discover, develop, and commercialize new gene editing medicines for a range of ocular disorders; strategic research collaboration Asklepios BioPharmaceutical, Inc. to develop a therapy to treat a neurological disease; and research collaboration with Sandhill Therapeutics, Inc. to develop allogeneic healthy donor derived NK cell medicines for the treatment of solid tumors. The company was formerly known as Gengine, Inc. and changed its name to Editas Medicine, Inc. in November 2013. Editas Medicine, Inc. was founded in 2013 and is headquartered in Cambridge, Massachusetts.
Editas Medicine, Inc.
Vertex Pharmaceuticals Incorporated engages in developing and commercializing therapies for treating cystic fibrosis. The company markets SYMDEKO/SYMKEVI, ORKAMBI, and KALYDECO to treat patients with cystic fibrosis who have specific mutations in their cystic fibrosis transmembrane conductance regulator gene; and TRIKAFTA for the treatment of patients with CF 12 years of age or older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator, or CFTR, gene. Its pipeline includes Pipeline for Alpha-1 antitrypsin deficiency that is in Phase 2 clinical trial; VX-864, a second investigational small molecule corrector for the treatment of AAT deficiency, which is in Phase 1 clinical trial; and VX-147 that completed a Phase 1 clinical trial for the treatment of APOL1-mediated focal segmental glomerulosclerosis, or FSGS, and other serious kidney diseases. The company sells its products primarily to specialty pharmacy and specialty distributors in the United States, as well as specialty distributors and retail chains, and hospitals and clinics internationally. Vertex Pharmaceuticals Incorporated has collaborations with CRISPR Therapeutics AG; Arbor Biotechnologies, Inc.; Moderna, Inc.; Genomics plc; Merck KGaA; Darmstadt, Germany; X-Chem, Inc.; Janssen Pharmaceuticals, Inc.; Merck KGaA; Kymera Therapeutics; Ribometrix, Inc.; Molecular Templates, Inc.; and Affinia Therapeutics. The company was founded in 1989 and is headquartered in Boston, Massachusetts.
Vertex Pharmaceuticals, Inc.
Intellia Therapeutics, Inc., a genome editing company, focuses on the development of therapeutics. It utilizes a biological tool known as the Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. The company develops in vivo programs focusing on liver diseases, including transthyretin amyloidosis and hereditary angioedema, as well as other research programs comprising primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Its ex vivo pipeline includes proprietary programs focused on developing engineered cell therapies to treat various oncological and autoimmune diseases; partnered programs focused on chimeric antigen receptor T cells and hematopoietic stem cells; and acute myeloid leukemia. Intellia Therapeutics, Inc. has license and collaboration agreements with Novartis Institutes for BioMedical Research, Inc.; Regeneron Pharmaceuticals, Inc.; Ospedale San Raffaele; and GEMoaB Monoconals GmbH. The company was formerly known as AZRN, Inc. and changed its name to Intellia Therapeutics, Inc. in July 2014. Intellia Therapeutics, Inc. was founded in 2014 and is headquartered in Cambridge, Massachusetts.
Intellia Therapeutics, Inc.
Repare Therapeutics Inc., a precision oncology company, discovers and develops novel therapeutics by using its synthetic lethality approach in Canada. The company use its SNIPRx, a proprietary, genome-wide, and CRISPR-enabled platform to systematically discover and develop highly targeted cancer therapies focused on genomic instability, including DNA damage repair. Its lead product candidate is RP-3500, an oral small molecule inhibitor for the treatment of solid tumors with specific DNA damage repair-related genomic alterations. It is also developing CCNE1-SL inhibitor, a proprietary drug discovery program for tumors with amplification of CCNE1; and Polymerase Theta program, a SL target associated with BRCA mutations and other genomic alterations. The company was founded in 2016 and is headquartered in MontrÃ©al, Canada.
Repare Therapeutics, Inc.
CureVac N.V., a clinical-stage biopharmaceutical company, engages in developing various transformative medicines based on messenger ribonucleic acid (mRNA). Its lead proprietary programs include CV8102 that is in a Phase 1 dose escalating clinical trials for four types of cancers as a monotherapy and in combination with anti-PD-1; and CV7202, which is in a Phase 1 clinical trials as a vaccine candidate against rabies. It is also advancing mRNA vaccine program against coronavirus (SARS-CoV-2), which is in Phase 1 clinical trial. The company's partnered programs comprise BI1361849, a therapeutic vaccine candidate that is in a Phase 1/2 clinical trials to elicit antigen-specific immune responses against tumor-associated antigens frequently overexpressed in patients with non-small cell lung cancer; Cas9 mRNA constructs for use in gene editing therapeutics; mRNA based novel therapeutic antibodies; prophylactic vaccines to prevent picornaviruses, influenza, malaria, and rotavirus; and programs against SARS-CoV-2, Lassa virus, and yellow fever. It has strategic partnerships with Genmab, Arcturus, Acuitas, CRISPR Therapeutics, Boehringer Ingelheim, GlaxoSmithKline, the Bill & Melinda Gates Foundation, CEPI, Tesla Grohmann, and others. The company was founded in 2000 and is headquartered in TÃ¼bingen, Germany.