March 22, 2022
The US FDA has set a 15 year follow-up period for therapeutic gene editing and edited products in new draft guidelines.
The guidance – available here – covers therapeutic use of gene editing techniques like ZFN, and CRISPR, as well as gene therapy products made using those techniques.
The aim, according to the FDA, is “to assist in the translation of these products from the bench to clinical trials, this guidance includes recommendations for how to assess the safety and quality of these products and address the potential risks of these products.”
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One of the key recommendations is that developers use a design platform that is most applicable to their genomic target and the type of intended modification.
The agency also states that the design, screening processes and rationale for using a particular approach – as well as the genetic sequences of the genome editing components – be included in the investigational new drug dossier (IND) submitted for each product.
“We recommend sponsors optimize the [genome editing] components to reduce the potential for off-target genome modification, to the extent possible,” the FDA wrote.
The draft guideline should not be concerning for the gene therapy sector, according to Luca Issi, analyst at RBC Capital Markets.
He wrote in a note to investors that “The FDA gene editing guidance released Tuesday reads benign with 15 years follow-up cited in the document mirroring prior guidance for gene therapy.”
Issi pointed out that although the requirement that first-in-human studies of gene therapy products “should enroll only subjects with no other treatment options” is prescriptive, elsewhere there is still room for maneuver.
“The door remains open to less severe indications in some instances and importantly the FDA is silent on indication prioritization beyond first-in-human,” he wrote.
The requirement that “patients treated with gene editing should be followed for at least 15 years,” is also likely to have minimal impact on the industry according to Issi.
“This mirrors prior guidance for gene therapy, which cites follow-up as long as 15 years for integrating approaches like lenti. While we find it interesting that gene editing guidance cites follow-up of “at least 15 years” vs “as long as 15 years” for gene therapy guidance, we don’t read too much into it as the former document directly cites the latter.
“Overall, we think this is net positive for the field as the FDA is clearly not breaking new ground by asking for longer term follow-up.”
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