Google
Trusted Resources: News & Events
Latest announcements and gatherings
Early clinical trial data show gene therapy reversing sickle cell anemia
After over a decade of preclinical research and development, a new gene therapy treatment for sickle cell anemia (SCA) is reversing disease symptoms in two adults and showing early potential for transportability to resource-challenged parts of the world where SCA is most common.
Preliminary data from a pilot Phase 1-2 clinical trial testing the gene-addition therapy were presented on Dec. 3 at the American Society of Hematology’s (ASH) annual meeting in San Diego by principal investigator Punam Malik, MD. She is a physician-scientist at the Cincinnati Children’s Cancer and Blood Diseases Institute and director of its Comprehensive Sickle Cell Center. Malik called the data promising.
One year after treatment of our first patient, and six months after treatment of our second patient, both have seen a remarkable improvement in the quality of life due to remarkable reduction in disease symptoms.
+myBinderRelated Content
-
bluebird bio Announces Publication of Case Study on First Patient with Severe Sickle Cell Disease Treated with Gene ...Patient treated with LentiGlobinTM drug ...
-
Jennifer Doudna on ethics of gene editinghttps://www.youtube.com/watch?v=8Ijr1ccY...
-
Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell DiseaseBackground: Sickle cell disease is char...
-
Gene Therapy: What You Need to Know (Warrior FAQs)Two gene therapies were recently approve...
-
Bluebird bio acquires Durham, NC, manufacturing site for lentiviral vector productionbluebird bio said today it has acquired ...
-
Experimental Gene Therapy Reverses Sickle Cell Disease for YearsA study of an investigational gene thera...
-
SCDAA News Advisory: SCDAA Statement on Exa-cel Gene TherapyOn October 31, 2023, the Food and Drug A...
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.