Deborah Phippard, PhD, on the Future of Gene Therapy in Neurology

“Everyone's well aware that Alzheimer disease and Parkinson disease trials have often been very disappointing with small molecules and even with some of the biological treatments we have now, so gene therapy is poised to be useful... It’s a very rich, promising field.”

Gene therapy is rapidly becoming a source of hope for patients and families of patients with diseases that have often been seen as untreatable with other modalities. As more and more gene therapy products enter clinical trials, and new tools such as artificial intelligence (AI) provide promise for overcoming the currently existing limitations of gene therapy as a modality, efforts are increasing for expanding it beyond single-gene disorders, such as lysosomal storage diseases, and into more complex indications like Alzheimer disease and Parkinson disease.

In her role as the chief scientific officer of the clinical research services organization Precision for Medicine, Deborah Phippard, PhD, has become familiar with many of the forward trends in the field of gene therapy for neurological indications. In an interview with CGTLive™, she discussed her thoughts on the future of gene therapy and gave her view on the indications where gene therapy has the most potential to make an impact.

Phippard emphasized that the future of gene therapy is dependent not just on the further development of gene therapy technology itself, but also on related medical innovations such as diagnostic tools and methods for measuring efficacy. She also discussed the potential of AI tools to aid the discovery process for novel gene therapies, especially when it comes to the innovative delivery methods necessary for crossing the blood–brain barrier to treat central nervous system indications. Phippard additionally noted that although innovation is exciting, there is a need to move forward with caution so that gene therapy projects do not get derailed by unexpected adverse events.