Scientific symposium
Wednesday, May 8 (3:45 – 4:15 p.m.)
Room 337-338 | Third floor
“To administer or not to administer? The benefits and challenges to the incorporation of immunosuppressive regimens during nonclinical safety evaluations”
Moderated by Brian E. McIntosh, PhD
Director of Science Experts and Global Preclinical Lead of Cell and Gene Therapy, Safety Assessment/Toxicology
The age of advanced therapies has introduced exciting gene therapies to treat, and even cure, debilitating monogenetic diseases. With this introduction, viral systems, such as adeno-associated virus (AAV), have initially emerged as the payload delivery vector of choice. While non-replicating viral capsids have evolved to efficiently infect mammalian cells, human innate and adaptive immune responses to viral proteins remain consistent and pose a potential liability with respect to both safety and efficacy. Treatment with co-immunosuppressive regimens is often sought in the clinic to mitigate immunotoxicity and/or allow the gene therapy to remain active. However, in the evaluation of product safety during the nonclinical phase, a conundrum is continuously at play in relation to the use of immunosuppressives in this setting.
This expert panel will present and discuss a variety of opportunities and challenges associated with the use of immunosuppressive countermeasures during the discovery and/or the nonclinical safety evaluations of viral delivery systems used for gene therapies. Our discussion will provide you with a better understanding of the pros and cons, and how to assess your program considering this insight.