Google
Trusted Resources: News & Events
Latest announcements and gatherings
CTX001 Continues to Show Promise in Severe SCD
A single dose of CTX001, an experimental gene-editing cell therapy, rapidly increases the levels of hemoglobin and prevents vaso-occlusive crises (VOCs) for up to nearly two years in people with severe sickle cell disease (SCD), according to interim data from the Phase 1/2 CLIMB-SCD-121 clinical trial.
CRISPR Therapeutics and Vertex Pharmaceuticals are jointly developing CTX001 for the treatment of hemoglobin-associated diseases, including SCD and transfusion-dependent beta thalassemia (TDT).
These preliminary findings, along with positive interim results from the CLIMB-Thal-111 study (NCT03655678) testing the therapy in TDT patients, were presented at the European Hematology Association 2021 Virtual Congress, held June 9–17.
“We are excited about these results and look forward to additional longer-term data and to moving this investigational medicine forward for a larger population of patients with these two devastating diseases,” Samarth Kulkarni, PhD, CRISPR Therapeutics’ CEO, said in a press release.
+myBinderRelated Content
-
A novel approach to reducing admissions for children with sickle cell disease in pain crisis through individualizati...BACKGROUND:Vaso-occlusive crisis (VOC) i...
-
New Research Suggests SANGUINATE™ Reduces the Number of Sickled Red Blood Cells in Patients with Vaso-Occlusive Cr...SANGUINATE™, the only investigational ...
-
Nociceptors Protect Sickle Cell Disease Mice From Vaso-Occlusive Episodes and Chronic Organ DamageSickle cell disease (SCD) is a common he...
-
Step toward gene therapy for sickle cell diseaseA team of researchers at the Stanford Un...
-
Sequence Replacement to Cure Sickle Cell DiseaseNext-generation gene editing is already ...
-
Gene therapy targets sickle-cell diseaseElliott Vichinsky estimates that at leas...
-
Virtual Reality Helps Reduce Pain Among Patients With Sickle Cell DiseaseImmersive virtual reality appeared effec...
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.