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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.
Who We Are - CRISPR Therapeutics
At CRISPR Therapeutics, our aim is to find cures for people suffering from serious diseases through transformative gene-based medicines.
Pipeline - CRISPR Therapeutics
Exa-cel, a CRISPR/Cas9 gene-edited therapy arising out of our collaboration with Vertex Pharmaceuticals Incorporated, has now been approved in some countries for certain eligible patients with sickle cell disease or transfusion-dependent beta thalassemia.
Gene Editing - CRISPR Therapeutics
As a gene-editing tool, CRISPR/Cas9 has revolutionized biomedical research and enabled medical breakthroughs. CRISPR/Cas9 edits genes by precisely cutting DNA and then harnessing natural DNA repair processes to modify the gene in the desired manner.
Therapies | CRISPR Therapeutics
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.
Therapeutic Approach | CRISPR Therapeutics
With CRISPR/Cas9, we can potentially engineer the genomes of cellular therapies to make them more efficacious, safer and available to a broader group of patients. At CRISPR Therapeutics, we aim to develop transformative gene-based medicines based on CRISPR/Cas9 gene editing.
CRISPR Therapeutics Highlights Strategic… | CRISPR Therapeutics
Jan 8, 2024 · CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease.
CRISPR Therapeutics Presents Data at the 2024… | CRISPR …
Dec 9, 2024 · CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic in 2018 to investigate the treatment of sickle cell disease or transfusion-dependent beta thalassemia, and beginning in late 2023, CASGEVY™ (exagamglogene autotemcel [exa-cel]) was approved in some countries to treat eligible patients with either of ...
CRISPR Therapeutics Proposes New Appointment to… | CRISPR …
Jan 7, 2025 · CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic in 2018 to investigate the treatment of sickle cell disease or transfusion-dependent beta thalassemia, and beginning in late 2023, CASGEVY™ (exagamglogene autotemcel [exa-cel]) was approved in some countries to treat eligible patients with either of ...
Current Clinical Research | CRISPR Therapeutics
CRISPR Therapeutics is focused on the development of novel therapies to treat hemoglobinopathies (inherited blood disorders), cancer, autoimmune diseases, cardiovascular diseases, diabetes, and other diseases.