Roundtable Discussion: If Re-Dosing Is a Reality, Then What Must The Field Be Prepared to Face?

Time: 4:30 pm
day: Day Two


The immune response to systemic use of gene therapy is challenge that looms over the field, particularly as questions regarding durability of gene therapy products means
that re-administration seems to look like an increasingly likely reality for many diseases targeted by this drug.

Re-dosing or re-administration is limited by a unique set of challenges itself. The biggest of which being the formation of an anti-AAV and anti-transgene immune response. Consequently, in cases where re-dosing is desirable or unavoidable, immunogenicity stands to be the biggest challenge that the field must work to overcome.

As such this roundtable discussion will set out to address:

  • Durability of gene therapy products and its implications for re-dosing
  • Immune modulation and immunosuppression strategies as an approach to dampen and mitigate immune response
  • If re-dosing is a reality, then what must we be prepared to face?
  • What effect does repeat exposure have on the immune response?
  • The challenges of patient safety in re-dosing
  • What can we expect the need and feasibility of re-dosing to be in the next 10 years?