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First-Ever Approved CRISPR-Based Therapy. CASGEVY™ (exagamglogene autotemcel), a CRISPR/Cas9 gene-edited therapy arising out of our collaboration with Vertex Pharmaceuticals Incorporated, is approved in some countries for certain eligible patients with sickle cell disease or transfusion-dependent beta thalassemia.
Pipeline. Description: Autologous, ex vivo CRISPR/Cas9 gene-edited therapy in which aims to edit a patient’s own hematopoietic stem cells to produce fetal hemoglobin in red blood cells.
At CRISPR Therapeutics, our aim is to find cures for people suffering from serious diseases through transformative gene-based medicines. Creating an impact. The first-ever CRISPR-based therapy is a direct reflection of our patient-forward philosophy.
Jan 18, 2024 · What does the first approved CRISPR therapy treat? The MHRA approved Casgevy to treat sickle-cell disease (SCD) and transfusion-dependent beta-thalassemia. These are lifelong, genetic disorders...
Dec 8, 2023 · The U.K. had already approved the CRISPR treatment, called exa-cel (brand name: Casgevy), from Vertex Pharmaceuticals and CRISPR Therapeutics (which was co-founded by Charpentier), to treat...
Nov 21, 2023 · The UK’s regulator has approved the world’s first CRISPR–Cas9 gene editing therapy, which aims to cure sickle cell disease and transfusion-dependent β-thalassemia. Casgevy (exagamglogene...
Jun 13, 2024 · The future of CRISPR-based therapeutics is undeniably bright, yet it is not without challenges. These range from the technical and biological constraints of the technology to clinical...
Jan 16, 2024 · CRISPR Therapeutics Announces U.S. Food and Drug Administration (FDA) Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia. - Approximately 1,000 patients in the U.S. 12 years of age and older are now eligible for this one-time treatment-.
The Investor Relations website contains information about CRISPR Therapeutics's business for stockholders, potential investors, and financial analysts.
Jan 16, 2023 · Abstract. Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases...
