DRAFT Questions for October 25:
General Questions on Lentivirus Vectors
1. What safety data should be available prior to initial use of HIV-based lentivirus vectors in phase 1 clinical trials? Please consider the following:
2. What should be the appropriate species for in vivo, preclinical safety and toxicology evaluation of lentivirus vectors? Please consider the following:
3. Given the limitations of the available animal models for study of vector safety and mobilization, please comment on whether in vitro assays are sufficient to address the safety issues of recombination, RCL generation, and rescue and/or mobilization of lentiviral vectors, assuming such assays were accompanied by limited safety data from in vivo preclinical proof-of-concept studies?