Google
Trusted Resources: News & Events
Latest announcements and gatherings
Statement from FDA Commissioner on agency’s efforts to advance development of gene therapies
Once just a theory, gene therapies are now a therapeutic reality for some patients. These platforms may have the potential to treat and cure some of our most intractable and vexing diseases. The policy framework we construct for how these products should be developed, reviewed by regulators, and reimbursed, will help set the stage for the continued advancement of this new market.
Last year, we announced our comprehensive policy framework for regenerative medicine, including a draft guidance that describes the expedited programs, such as the breakthrough therapy designation, and the regenerative medicine advanced therapy (RMAT) designation, that may be available to sponsors of these therapies. Today, we’re unveiling a complementary framework for the development, review and approval of gene therapies.
+myBinderRelated Content
-
SCDAA Gene Therapy Town Hall – Webinarhttps://www.youtube.com/watch?v=J77Gq9yI...
-
Gene-Edited ‘Supercells’ Make Progress In Fight Against Sickle Cell DiseaseDoctors are reporting the first evidence...
-
Successful Gene Therapy for Sickle Cell DiseasePrior to this report, the only curative ...
-
NIH Launches new Collaboration to Develop Gene-Based Cures for Sickle Cell Disease and HIV on Global ScaleThe National Institutes of Health plans ...
-
Alabama Man Free of Sickle Cell After Gene Therapyhttps://www.youtube.com/watch?v=E9vYON8D...
-
These patients had sickle-cell disease. Experimental therapies might have cured themScientists have long known what causes s...
-
Statement on NHLBI Decision to Pause the Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Si...bluebird bio, Inc. suspended its clinica...
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.