Krystof Bankiewicz, MD, PhD, on GDNF Gene Therapy for Parkinson’s and AADC Deficiency


The professor at Ohio State University and University of California – San Francisco discussed investigations with different delivery methods of GDNF gene therapy.

“We never thought that we'd be able to help patients (with AADC deficiency) that are older. Our oldest patient is 27 years old, and that man responded to treatment. But there’s also a lot of patients that are a little bit older, they're not 5, 6 or 8 years of age, that are also responding quite dramatically. So that was unexpected.We've now been treating patients around the world... There’s only maybe 250 of these patients around the world.”

Krystof Bankiewicz, MD, PhD, professor and vice chair of research, department of neurological surgery, Ohio State University, and professor emeritus and vice chair of research, University of California – San Francisco, and cofounder, Brain Neuropathy Bio, presented updated data from a trial evaluating glial cell-derived neurotrophic factor (GDNF) gene therapy for Parkinson disease (PD) at the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting, held May 16-20, in Los Angeles, California.

CGTLive spoke with Bankiewicz to learn more about the GDNF gene therapy and the phase 1b trial evaluating it in patients with PD. He also discussed investigations with the gene therapy in patients with aromatic L-amino acid decarboxylase (AADC) deficiency, data observed in a trial, and progress of the trial so far. He shared his surprise at the efficacy seen with gene therapy in older patients with AADC deficiency in addition to younger patients.He explained how GDNF gene therapy may work for both of these indications and how different delivery methods can change its use.

Click here to read more coverage of ASGCT 2023.

Bankiewicz K. Developing AAV Gene Therapies for Movement Disorders. Presented at: ASGCT 2023 Annual Meeting; May 16-20; Los Angeles, California.
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