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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Last year, we announced our comprehensive policy framework for regenerative medicine, including a draft guidance that describes the expedited programs, such as the breakthrough therapy designation, and the regenerative medicine advanced therapy (RMAT) designation, that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.
news & eventsMARAC Advisory Statement: Gene Therapy & Bone Marrow TherapiesPlease note: A previous version of this ...
news & eventsFDA Clears Graphite Bio to Begin Trial for Gene Therapy in Sickle Cell DiseaseIn response to Graphite Bio’s investig...
education & researchGene Therapy: An Experimental Technique to Treat Genetic DiseasesGene therapy is an experimental techniqu...
videos & visualsAlabama Man Free of Sickle Cell After Gene Therapyhttps://www.youtube.com/watch?v=E9vYON8D...
Community CenterSuccessful Gene Therapy for Sickle Cell DiseasePrior to this report, the only curative ...
news & eventsStatement on NHLBI Decision to Pause the Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Si...bluebird bio, Inc. suspended its clinica...
education & researchBiologic and Clinical Efficacy of LentiGlobin for Sickle Cell DiseaseBackground: Sickle cell disease is char...
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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.