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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No AdComm Puts Sarepta On Path To Blockbuster Success With Duchenne Gene Therapy
The US FDA’s waiving of an advisory committee is another promising signal that Sarepta's Duchenne gene therapy SRP-9001 could be approved on 29 May and set it up to become a bigger earner than Novartis’s Zolgensma.
Sen. Burr’s Parting Gifts For US FDA Include ‘Platform’ Pathway
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.