News & Press Releases: Breaking News

ISCT NA LRA Regulatory WatchDog Update: FDA Releases 6 Guidances on Gene Therapy

Thursday, July 12, 2018  
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ISCT NA Legal and Regulatory Affairs (LRA) Update

On July 11, the 
FDA released a total of 6 guidance documents intended to help advance the field of gene therapy. This includes three new draft guidance documents that address the development of gene therapy products for specific disease categories (Hemophilia, Retinal Disorders and Rare Diseases) and updates to three existing guidances that address manufacturing issues related to gene therapy.
Statement from FDA Commissioner Scott Gottlieb, M.D. on agency’s efforts to advance development of gene therapies - Click here to view

Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs); Draft Guidance for Industry (PDF - 486KB)
Click here to view

Long Term Follow-up After Administration of Human Gene Therapy Products; Draft Guidance for Industry (PDF - 294KB)
Click here to view

Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up; Draft Guidance for Industry (PDF - 124KB)
Click here to view

Human Gene Therapy for Hemophilia; Draft Guidance for Industry (PDF - 371KB)
Click here to view

Human Gene Therapy for Rare Diseases; Draft Guidance for Industry (PDF - 136KB)
Click here to view

Human Gene Therapy for Retinal Disorders; Draft Guidance for Industry (PDF - 172KB)
Click here to view

View the most recent NA LRA Regulatory WatchDog Report from the June Telegraft issue.

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