Sung-Yun Pai, MD, on Further Research With Lentiviral Gene Therapy for X-SCID

“We are using low dose busulfan, which is still chemotherapy. These are very young infants; they are generally less than 6 months old at the time of treatment. And so, we are exposing them to chemotherapy that they don't necessarily need to have. So, the other advance, of course, would be to pair this very successful lentiviral vector with a nontoxic form of conditioning to eliminate enough of the stem cell compartment for the gene therapy transduced cells to engraft.”

Updated data from a phase 1/2 clinical trial (NCT03311503) demonstrated 100% survival and robust T-cell recovery more rapid than after standard hematopoietic stem cell transplantation (HCT) in patients with X-linked severe combined immunodeficiency (SCID-X1) that received lentiviral gene therapy delivering codon optimized IL2RG.

The data were presented by investigator Sung-Yun Pai, MD, chief, immune deficiency cellular therapy program, and tenured senior investigator, National Cancer Institute Center for Cancer Research, at the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting, held May 16-20, in Los Angeles, California. CGTLive spoke with Pai to learn more about the next steps she would like to take in researching the lentiviral gene therapy. She also discussed the need for nontoxic conditioning, especially for diseases in which patients are very young, as in this study.

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REFERENCE

Pai SY, Booth C, Kohn DB, et al. Universal survival and superior immune reconstitution after lentiviral gene therapy with low dose conditioning for X-linked SCID (SCID-X1). Presented at: ASGCT 2023 Annual Meeting; May 16-20; Los Angeles, California.