Stephanie Tagliatela on Researching AAV for Lennox-Gastaut, Alzheimer Disease, SCN9a Pain
The cofounder and chief scientific officer at Encoded Therapeutics shared preclinical research with the company’s AAV and miRNA platforms.
“There's been a lot of exciting developments in the CNS field, especially over the past few years, with some pivotal approvals in Alzheimer disease and a lot of exciting biomarker work. So, we think that the CNS space is primed for AAV gene therapy to move into, and by establishing this knockdown platform, I think we have an opportunity to go after targets that have a lot of clinical and biological validation, but have been limited by, for various reasons, alternate modalities.”
Encoded Therapeutics is hoping to tackle central nervous system (CNS) disorders with its adeno-associated virus (AAV) gene regulation therapy platform. Included in the company’s pipeline are programs for Dravet syndrome (the company’s lead candidate), Angelman syndrome, Lennox-Gastaut syndrome (LGS), Alzheimer disease (AD), and chronic pain. Encoded presented multiple posters of preclinical research on these programs at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland.
CGTLive® spoke with Stephanie Tagliatela, cofounder and chief scientific officer, Encoded Therapeutics, during the ASGCT meeting to learn more about the progress and findings of these programs. In this clip, Tagliatela discussed the posters on LGS, Alzheimer disease, and chronic pain. The LGS demonstrated improvements in incidence and severity in 2 distinct seizure models at relatively low doses of AAV gene therapy. The poster on AD and pain showcased an early use of Encoded’s AAV-micro RNA platform knocking down SC9NA for pain and MAPT for AD.
REFERENCES
1. Babineau B, Sears S, Su J, et al. GABA Selective AAV-Mediated Gene Therapy Provides Durable Seizure Protection in Multiple Refractory Epilepsy Models. Presented at: ASGCT 27th Annual Meeting, May 7-10; Baltimore, Maryland. Abstract #19
2. Tai C, Tatavarty T, Chen SY, et al. Identification of Potent and Selective AAV-miRNA Candidates to Knockdown Non-Monogenic Neurological Targets SCN9A (Pain) and MAPT (Tauopathies). Presented at: ASGCT 27th Annual Meeting, May 7-10; Baltimore, Maryland. Abstract #1601
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