BIO Comments on FDA Guidance: Human Gene Therapy Products Incorporating Human Genome Editing
June 10, 2022
On Friday, June 10th, BIO submitted comments in response to the FDA’s draft guidance on developing human gene therapy products that incorporate genome editing (GE) of human somatic cells. Overall, BIO found the guidance to be a well-written resource that will be useful once further experience is gained with these therapies. 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.
Download Full Comments Below
BIO Comment Letter Gene Editing Guidance FDA 2021 D 0398
On Tuesday, January 3rd, BIO submitted comments in response to the Food and Drug Administration’s (FDA) recent draft guidance containing recommendations for sponsors on monitoring growth and evaluating pubertal development in clinical trials which…
BIO and its members appreciate the opportunity to work with FDA to develop and align on approaches that are robust, practical, and expedite patient focused drug development. To further enhance the Guidance, we believe that a few areas would benefit…
On Wednesday, June 22nd, BIO submitted comments on the FDA’s Quality Metrics (QM) Reporting Program. In the comments submitted, BIO stated appreciation for FDA’s intent to support a general shift toward a risk-based approach to regulation by…
.png){kind=logo}
On Friday, June 10th, BIO submitted comments in response to the FDA’s draft guidance on developing human gene therapy products that incorporate genome editing (GE) of human somatic cells. Overall, BIO found the guidance to be a well-written resource that will be useful once further experience is gained with these therapies. 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.