Project Summary/Abstract
The Pediatric Gene Therapy and Medical Ethics Working Group (PGTME)—housed in NYU Grossman School
of Medicine’s Division of Medical Ethics—is planning to host a one-day hybrid conference in Washington D.C.
in mid-May entitled, “Antibodies to Viral Vectors in Gene Therapy Research: Seeking Best Practices for
Sponsor Policies and Communications.” We are researching this topic in pursuit of possible policy solutions
addressing standards of and communication around antibody (AB) testing to viral vectors used in gene therapy
(GT) research. In 2021, PGTME held two listening sessions and one closed-door meeting on this topic.
Participants from these meetings raised a number of potential educational and/or structural interventions;
however, there remain questions around which are feasible and effective. PGTME is convening a diverse
group of experts to explore possible policy recommendations and communication standardization during this
conference.
Gene therapy (GT) interventions cannot currently be reversed or “turned off,” should they cause harm to the
recipient. Furthermore, GTs administered by means of a viral vector result in immunologic sequelae which
preclude recipients’ participation in future GT clinical trials. Thus, at present, research subjects in these studies
are only able to receive one potentially life-altering candidate GT. Given this unique set of facts about GT
research, it is essential that patients and/or surrogate decision-makers have the best possible understanding of
the potential risks and benefits of participating in a GT clinical trial, as well as the implications of such trial
participation in terms of time, expense, effort, and opportunity costs. While there are numerous ethical issues
surrounding GT clinical trials, the proposed conference focuses on only two. First, patients and/or caregivers
of individuals with rare diseases for which GT clinical trials are currently underway or are currently
anticipated often receive inconsistent messaging from sponsors and other entities about the antibody
level testing done as part of screening a potential participant for eligibility in a GT clinical trial.
Secondly, these patients and/or caregivers often do not receive appropriate or consistent
communication from clinicians or trial sponsors about immunogenic implications of participating in
GT research.
We fully anticipate that the panel sessions and other informal interactions between the participants of this
conference will contribute to advancing practices and policies that improve messaging from GT trial sponsors
and clinicians about antibody level testing utility and will set the basis for collaborative approaches aimed at
more ethical and more efficient GT clinical trials.
