Google
Trusted Resources: News & Events
Latest announcements and gatherings
CTX001 Earns FDA’s Fast Track Status for Treating Sickle Cell Disease
The U.S. Food and Drug Administration (FDA) has awarded fast track status to investigational therapy CTX001 to treat sickle cell disease.
A Phase 1/2 clinical trial (NCT03745287) is recruiting patients.
CTX001 uses the CRISPR gene-editing technology to increase the production of fetal hemoglobin in patients’ red blood cells.
Fetal hemoglobin is a form of hemoglobin that naturally exists when one is born and later is replaced by the adult form of hemoglobin.
For the treatment, doctors collect a patient’s own hematopoietic stem cells (cells from the bone marrow) and modify them in the laboratory so they can produce high levels of fetal hemoglobin.
+myBinderRelated Content
-
Prodigy’s death shines light on slow progress against sickle cell diseaseThe death of the rap artist Prodigy (Alb...
-
bluebird bio Announces Publication of Case Study on First Patient with Severe Sickle Cell Disease Treated with Gene ...Patient treated with LentiGlobinTM drug ...
-
Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA u...Advances in allogeneic hematopoietic cel...
-
Crizanlizumab lowers pain crises in at-risk sickle cell patients, ad-hoc trial data showNovartis’ investigational th...
-
Scientists edit genome to cure sickle cell anemiahttps://www.youtube.com/watch?v=4LKzM3G6...
-
Accelerating the Science of SCD Therapies—Is a Cure Possible?Nearly 7 decades have passed since semin...
-
1st Patients to Get CRISPR Gene-Editing Treatment Continue to ThriveThe last thing a lot of people want to d...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Powered by
Support for this site is provided by
This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.