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.
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Supernus Pharmaceuticals, Inc., a biopharmaceutical company, develops and commercializes products for the treatment of central nervous system (CNS) diseases in the United States. The company offers Qelbree, a non-stimulant indicated for the treatment of attention-deficit hyperactivity disorder (ADHD); GOCOVRI for the treatment of dyskinesia and levodopa/carbidopa in patients with Parkinson's Disease (PD); Oxtellar XR, an extended-release oxcarbazepine product indicated for the monotherapy treatment of partial onset epilepsy seizures; and APOKYN for the acute and intermittent treatment of hypomobility or off episodes in patients with advanced PD. It also provides Trokendi XR, an extended release topiramate product indicated for the treatment of epilepsy, as well as for the prophylaxis of migraine headache; XADAGO for the treatment of levodopa/carbidopa in patients with PD experiencing off episodes; and MYOBLOC, a Type B toxin product indicated for the treatment of cervical dystonia and chronic sialorrhea. In addition, the company develops ONAPGO, which received FDA approval, for the treatment of motor fluctuations in adults with advanced PD; SPN-817, a first-in-class selective acetylcholinesterase inhibitor, which is in Phase 2 clinical trial, for the treatment of epilepsy, partial seizures, Dravet syndrome, and Lennox-Gaustaut syndrome; SPN-820, a small molecule in Phase 2 clinical trial for treating resistant depression; SPN-443, a stimulant in Phase 1 trial for the treatment of ADHD/CNS; and SPN-446, a preclinical product for CNS. It markets and sells its products through pharmaceutical wholesalers, specialty pharmacies, and distributors. It has a development agreement with Navitor Inc. for the conduct of Phase 2 clinical program of SPN-820. Supernus Pharmaceuticals, Inc. was incorporated in 2005 and is based in Rockville, Maryland.
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