Califf: Decades Of Follow-Up Necessary For Gene Therapy
FDA Commissioner Califf acknowledges that regulatory pathways for gene therapies are still being developed and that many factors outside of the modality impact agency requirements as he emphasized the need for long-term follow up due to the unknowns of gene modification.
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The year-end package of US FDA reforms reflects the impact of one now-retired US Senator: North Carolina’s Richard Burr’s objections helped take out a number of ‘anti-innovation’ provisions – while still securing inclusion of a ‘Platform Technologies’ pathway that might otherwise not have been enacted.
Duchenne Doldrums Continue As Entrada Hit With IND Clinical Hold
The US firm’s lead oligonucleotide asset, ENTR-601-44, has received a clinical hold notice after IND submission, adding to a growing list of companies in the Duchenne space facing setbacks.
Requiring FDA Guidances Does Not Always Advance The Science, Gene Therapy Director Says
OTAT Director Wilson Bryan says important cell and gene therapy guidances came from clear community needs, not user fee program requirements.