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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Last year, we announced our comprehensive policy framework for regenerative medicine, including a draft guidance that describes the expedited programs, such as the breakthrough therapy designation, and the regenerative medicine advanced therapy (RMAT) designation, that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.
news & eventsGene therapy may help cure sickle cell disease, study saysA gene therapy that could provide a perm...
videos & visualsResearchers Develop Effective Treatment for Sickle Cell Anemiahttps://www.youtube.com/watch?v=7zceTJzH...
videos & visualsGene Therapy: Your Questions Answeredhttps://www.youtube.com/watch?v=5ChXI6cS...
videos & visualsAdvances in the Treatment of Sickle Cell Disease: L-Glutamine, Crizanlizumab & Gene Therapyhttps://www.youtube.com/watch?v=wkm4axJ3...
news & eventsSickle Cell Patient Receives CRISPR Gene TherapyMany human diseases can be traced back t...
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...
news & eventsGene Therapy for Blood DisordersIn the context of intense scrutiny over ...
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