BIO comments on FDA draft guidance for human gene therapy products incorporating human genome editing

By Rachel Arthur

- Last updated on GMT


Related tags BIO Gene therapy Gene therapy candidate genome editing

BIO, the world’s largest trade association for the biotech industry, has responded to the US Food and Drug Administration's draft guidance on developing human gene therapy products that incorporate genome editing (GE) of human somatic cells.

The organization – which represents biotech companies, academic institutions, state biotechnology centers and related organizations in the US and beyond – welcomes the development of the guidance: but wants to see the scope of the guidance extended to help sponsors see how the rapidly evolving field is likely to shape up from a regulatory viewpoint.

'A well-written resource'

The FDA’s draft guidance was published on March 15,​ providing recommendations to sponsors developing human gene therapy products incorporating GE of human somatic cells.

Specifically, the guidance provides recommendations for information that should be provided in an Investigational New Drug application: including product design, product manufacturing, product testing, preclinical safety assessment and clinical trial design.

“Overall, BIO found the guidance to be a well-written resource that will be useful once further experience is gained with these therapies,”​ said BIO, having submitted its official comments to the FDA on June 14 during the in-person return of the BIO International Convention in San Diego.

“However, given the field is rapidly advancing, BIO recommended the Agency revise certain sections of the guidance to make it more informative and practical for sponsors to implement. For example, expanding the scope of this guidance beyond human somatic cells to include cells derived from adult and pluripotent stem cells, would provide valuable insight to sponsors on FDA’s current thinking.

“Additionally, the guidance would be enhanced by a more comprehensive discussion of how safety, efficacy, durability, and quality of a GE product are factoring into FDA’s regulatory decisions. In particular, given the field of GE products is rapidly maturing, BIO believes the guidance should more fully discuss the benefit-risk considerations at marketing application review, including discussion of how the perspectives of patients will be incorporated.

“Furthermore, BIO challenged FDA’s implication that sponsors should be incorporating the best available technology in each GE product application, as newly discovered and yet untested and unproven technologies, should not distract from the objective and focused review of existing technologies that have matured to the point of being incorporated into GE products.”

BIO's comments on the FDA's draft guidance can be found in full here.

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