CRISPR Therapeutics AG
CRISPR Therapeutics AG, a gene editing company, focuses on developing gene-based medicines for serious human diseases using its Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) platform. The company's CRISPR/Cas9 is a revolutionary technology for gene editing which is the process of precisely altering specific sequences of genomic DNA. It has a portfolio of therapeutic programs across a range of disease areas, including hemoglobinopathies, CAR T cell therapies, in vivo, and type 1 diabetes, as well as develops investigational CAR T programs, including an autologous, gene-edited CAR T program targeting glypican-3 (GPC3) for the potential treatment of solid tumors. The company's lead product candidate is CASGEVY, an ex vivo CRISPR/Cas9 gene-edited cell therapy for treating patients suffering from transfusion-dependent beta-thalassemia, severe sickle cell disease (SCD), and hemoglobinopathies in which a patient's hematopoietic stem and progenitor cells are edited to produce high levels of fetal hemoglobin in red blood cells. It also develops CAR T cell therapies, including CTX112 targeting cluster of differentiation 19 (CD19) and CTX131 targeting CD70 for oncology and autoimmune indications; CTX310 and CTX320, in vivo gene editing to address the cardiovascular disease by disrupting the validated targets angiopoietin-like protein 3 and lipoprotein; and CTX211, an allogeneic, gene-edited, hypoimmune stem cell-derived product candidate for the treatment of T1D. It has strategic partnerships with Vertex Pharmaceuticals Incorporated, Nkarta, Inc., and Capsida Biotherapeutics. CRISPR Therapeutics AG was incorporated in 2013 and is headquartered in Zug, Switzerland.
Open CRSP dashboard ↗Jazz Pharmaceuticals, Inc.
Jazz Pharmaceuticals plc identifies, develops, and commercializes pharmaceutical products in the United States, Europe, and internationally. The company offers Xywav to treat cataplexy or excessive daytime sleepiness (EDS) with narcolepsy and idiopathic hypersomnia (IH); Xyrem for the treatment of cataplexy or EDS with narcolepsy; Epidiolex for seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC); Rylaze for the treatment of acute lymphoblastic leukemia or lymphoblastic lymphoma; Enrylaze to treat acute lymphoblastic leukemia and lymphoblastic lymphoma; Zepzelca for the treatment of metastatic small cell lung cancer with disease progression on or after platinum-based chemotherapy; Ziihera to treat HER2-positive biliary tract cancers; Defitelio to treat severe veno-occlusive disease; and Vyxeos for the treatment of newly-diagnosed therapy-related acute myeloid leukemia. It also develops Zanidatamab in Phase 3 trial to treat HER2-expressing gastroesophageal adenocarcinoma (GEA), and patients with HER2-expressing metastatic GEA; Epidiolex in Phase 3 trial to treat LGS, DS, and TSC; Suvecaltamide in Phase 2 trial to treat Parkinson's disease tremor; JZP815, a pan-RAF kinase inhibitor in Phase 1 trial that targets components of the mitogen-activated protein kinase; JZP898, a conditionally activated interferon alpha molecule in Phase 1 trial; JZP441 in Phase 1 trial to treat narcolepsy, IH, and other sleep disorders; JZP324, an extended-release low sodium oxybate formulation in Phase 1 trial for patients with sleep disorders; and JZP150 to treat post-traumatic stress disorder. The company has licensing and collaboration agreements with Redx Pharma plc, Autifony Therapeutics Limited, Zymeworks Inc., Sumitomo Pharma Co., Ltd., and Werewolf Therapeutics, Inc. Jazz Pharmaceuticals plc was founded in 2003 and is headquartered in Dublin, Ireland.
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