CBER Flags Planned Gene Therapy Guidances for 2019

July 8, 2019

CBER released an updated list of guidances it plans to publish this year, including draft  recommendations on the following gene therapy topics:

  • Human gene therapies for hemophilia, retinal disorders and rare diseases;
  • Chemistry, manufacturing and control (CMC) information for human gene therapy IND applications;
  • Interpreting the sameness of gene therapy products under orphan drug regulations;
  • The testing of retroviral vector-based human gene therapy products for Replication Competent Retrovirus (RCR) during product manufacturing and patient follow-up;
  • Long term follow-up after human gene therapy product administration; and
  • Working with the FDA on complex and innovative clinical trial designs for drugs and biological products.

The updated guidance agenda also notes several draft guidances that may be finalized following the agency’s review of public comments, including Expedited Programs for Regenerative Medicine Therapies for Serious Conditions, released in February, and Standards Development and their Use in Regulatory Submissions Reviewed in CBER, issued in March.

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