Amy Pooler, PhD, on Improving Gene Delivery to the CNS


The vice president of neuroscience at Sangamo Therapeutics discussed identifying novel capsids with improved neuronal transduction to the CNS.

“There are some advantages based on the fact that you could use a lower dose... it may also be able to evade some of the new neutralizing antibodies that we know are circulating in the in the bloodstream and target directly the CNS rather than increasing exposure in some of the peripheral organs.”

While gene and cell therapies have the potential to treat many rare, neurological diseases, efficient gene delivery to the central nervous system (CNS) remains a barrier to developing these therapies. One possible avenue of gene delivery lies in the cerebrospinal fluid; however, more work is needed to make this approach feasible for CNS disorders.

Sangamo Therapeutics’ new platform, SIFTER (Selecting In vivo for Transduction and Expression of RNA), is designed to engineer capsids with improved neuronal transduction after CSF administration. Preclinical data on this platform was presented at American Society of Gene & Cell Therapy 25th Annual Meeting (ASGCT), held in Washington, DC, and virtually May 16-19, 2022, by David S. Ojala, PhD, senior scientist, AAV engineering, Sangamo Therapeutics.

CGTLive spoke with Amy Pooler, PhD, vice president of neuroscience, Sangamo Therapeutics, to learn more about the platform and how it combats challenges with gene delivery to the CNS. She discussed novel capsids identified with SIFTER that showed improved neuronal transduction.

Ojala DS, Andrews L, Nanjaraj A, et al. Engineered AAV capsids exhibit improved transduction of the central nervous system after CSF administration in adult cynomolgus macaques. Presented at: ASGCT 25th Annual Meeting, May 16-19, 2022, Washington DC. Abstract #441.
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