First CRISPR Drug Coming Soon
July 6, 2022| |
Vertex and CRISPR Therapeutics presented impressive results of clinical trials for exagamglogene autotemcel (exa-cel), which can help patients with beta thalassemia and sickle cell disease (SCD). Exa-cel is the first gene editing therapy of its kind and will be submitted for regulatory approval in the US, UK, and Europe by the end of 2022.
Beta thalassemia affects 1 in 100,000 people globally. It is characterized by damages or missing genes that cause the body to produce less hemoglobin, which can lead to the enlargement of vital organs. Regular blood transfusion is necessary for patients with this disease. On the other hand, SCD affects millions globally and the sickle-shaped hemoglobin blocks the healthy blood cells from transporting oxygen around the body.
The latest results of the clinical trials of exa-cel were revealed at the European Hematology Association Congress in Switzerland. It was reported that 75 patients with beta thalassemia or SCD given who underwent the gene editing therapy had very minimal to zero blood transfusions or incidences of life-threatening blockages.
Read the original story in Fast Company.
| |
You might also like:
- Putting Breaks on Gene Editing with Anti-CRISPRs
- Experts Broaden Target Range of CRISPR-Cas Systems
- CRISPR Experts Introduce Twin Prime Editing
Biotech Updates is a weekly newsletter of ISAAA, a not-for-profit organization. It is distributed for free to over 22,000 subscribers worldwide to inform them about the key developments in biosciences, especially in biotechnology. Your support will help us in our mission to feed the world with knowledge. You can help by donating as little as $10.
-
See more articles:
-
Gene Editing Supplement (July 6, 2022)
-
Research and Tools
- OsPP65 Negatively Regulates Osmotic and Salt Stress Responses in Rice
- Designer TALE Successfully Produces Plant Growth Regulator to Increase Rice Yield
- No Difference in Meat and Milk Nutritional Values of Genome-edited and Conventional Cattle
- ‘Softer' Form of CRISPR Edits Genes More Accurately
- Danish Research Project Refines CRISPR; A Step Towards More Precise and Effective Use of Genetic Scissors
- First CRISPR Drug Coming Soon
-
Read the latest: - Biotech Updates (March 12, 2025)
- Gene Editing Supplement (March 12, 2025)
- Gene Drive Supplement (February 22, 2023)
-
Subscribe to BU: - Share
- Tweet