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
-
American Society of Gene & Cell Therapy: Blood DisordersBlood is very important to our body. The...
-
The FDA Encourages New Treatments for Sickle Cell DiseaseThe FDA continues to work with stakehold...
-
FDA grants CRISPR gene therapy fast track designation for sickle cell diseaseThe FDA granted fast track designation t...
-
Prolong Pharma’s Sanguinate Shows Promise in Reverting Shape of Red Blood Cells in SCDProlong Pharmaceuticals’ investigation...
-
A multiple drug approach to preventing sickle cell crisisSickle cell disease is characterized by ...
-
CRISPR deployed to combat sickle-cell anaemiaA mutation in a single DNA letter causes...
-
Stem cell gene-editing method may be breakthrough for sickle cell researchResearchers have cured mice with a genet...
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.