HIV-1-infected subjects treated with the proprietary retrovirus-based vector showed long-term presence of the engineered CD34+ stem cells as well as a long-term presence of engineered CD4+ immune cells in circulation.
The phase I clinical trial took place at the University of California San Francisco. There were no treatment-related adverse events during the study, or any evidence of leukemia seen by standard hematology, the report said.
Antisense RNA was present in all five subjects available at 12 months post treatment. At the 48th month, four subjects were available and antisense RNA was present in three of the four, and antisense RNA survived to month 60 in one of the subjects.
“These results are a significant, perhaps unique, example of the survival of engineered CD34+ stem cells in nonablated adult human subjects. That these engineered cells have produced progeny CD4+ immune cells is evidenced by the presence of antisense RNA in these cells in circulation,” the researchers said.