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Donald Kohn, MD, on the Trajectory of Gene Therapy

The renowned professor from University of California Los Angeles reflected on receiving the Outstanding Achievement Award from the ASGCT.

“Our signature work was the ADA-SCID work. We had our first-generation approach, we did a trial, we saw the deficiencies, we went back to the lab, improved the vectors, improved the cell processing, did another trial. As technology continued to improve, we changed our approach. Now we have up to 50 children [who had ADA-SCID] who are alive and well because of gene therapy. That's our biggest accomplishment.”

The American Society of Gene & Cell Therapy (ASGCT) presented its highest honor, the Outstanding Achievement Award, to renowned physician scientist Donald Kohn, MD, professor of microbiology, immunology and molecular genetics; and pediatric hematology/oncology, University of California Los Angeles (UCLA), during the 25th Annual Meeting, which took place May 16-19, 2022, in Washington, DC.

Kohn served as president of ASGCT from 2003-2004, president of the Clinical Immunology Society from 2013-2014, and was chair of the Recombinant DNA Advisory Committee for the NIH Office of Biotechnology from 2013-2015.

CGTLive spoke with Kohn about his career and his achievements along the way in reflection of the award. He discussed his work at UCLA developing a lentiviral stem cell gene therapy for adenosine deaminase severe combined immunodeficiency (SCID), conducting the first trial of its kind. He also discussed the ever-evolving technology of cell and gene therapies, hurdles that remain in the field, and the importance of collaboration.

1. Congratulations to the 2022 Award Recipients! News release. ASGCT. April 11, 2022. https://asgct.org/research/news/april-2022/2022-annual-meeting-award-winners
2. Kohn DB, Booth C, Shaw KL, et al. Autologous ex vivo lentiviral gene therapy for adenosine deaminase deficiency. N Engl J Med. 2021; 384:2002-2013. doi: 10.1056/NEJMoa2027675