Sharif Tabebordbar, PhD, on Improving In Vivo Gene Editing for DMD


The cofounder and chief executive officer of Kate Therapeutics discussed research that awarded him an Outstanding New Investigator at the ASGCT 2024 meeting.

“The dose that we're using [in nonhuman primate studies] is about at least 3 times lower compared to what is currently being tested in the clinic, and we have benchmarked our development candidate for Duchenne compared to an FDA approved-DMD gene therapy... We have significantly higher levels of protein expression, both in skeletal and protein, while being detargeted from the liver, so a better safety margin as well.”

Kate Therapeutics is developed muscle- and heart-targeted, liver de-targeted (MyoAAV-LD) capsids to improve gene therapy techniques for treating muscular dystrophies (MD), with KT809 as its lead MD candidate for the potential treatment of Duchenne muscular dystrophy (DMD). Promising nonhuman primate data from the KT809 program were presented by Sharif Tabebordbar, PhD, cofounder and chief executive officer, Kate Therapeutics, at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland, in a keynote lecture as recipient of the 2024 Outstanding Investigator Award for contributions to the field of gene therapy.

CGTLive® spoke with Tabebordbar to learn more about Kate’s programs and his work in improving in vivo gene editing. He shared potential advantages of KT809 over other investigational and approved therapies for DMD, notably its improved precision of targeting and expression. He also gave an overview of Kate’s gene therapy program for facioscapulohumeral dystrophy.

1. Kate Therapeutics Unveils Platform and Pipeline Progress at American Society of Gene & Cell Therapy 2024 Annual Meeting. News release. Kate Therapeutics. May 9, 2024.
2. Congratulations to Our 27th Annual Meeting Award Winners. News release. ASGCT. April 10, 2024.
Related Videos
Luke Roberts, MBBS, PhD, on Challenges in Developing Gene Therapy for Heart Failure
Steve Kanner, PhD, the chief scientific officer of Caribou Biosciences
Paul Y. Song, MD, the chairman and chief executive officer of NKGen
Lisa Nieland on Slowing Tumor Growth in Glioblastoma With Novel AAV Therapy
Manali Kamdar, MD, on Acclimating to Routine CAR T Practice in the Field
Paul Y. Song, MD, the chairman and chief executive officer of NKGen
Jennifer Taylor-Cousar, MD, MSCS, on Early Signals of Efficacy With Cystic Fibrosis Gene Therapy
Leigh Ramos-Platt, MD, on Looking Forward to Gene Therapy’s Growth
Paul Y. Song, MD, the chairman and chief executive officer of NKGen
Manali Kamdar, MD, on Evaluating Liso-Cel in Mantle Cell Lymphoma by Lines of Therapy, Prior BTKi
© 2024 MJH Life Sciences

All rights reserved.