Novel AAV Capsid Takes Gene Editing to the Brain

Following intravenous administration in non-human primates (NHPs), the novel capsid, STAC-BBB, showed robust penetration of the blood-brain barrier (BBB) and strong transgene expression throughout the central nervous system (CNS).

This breakthrough marks a significant leap in treating neurological disorders, as it addresses the longstanding challenge of achieving effective CNS drug delivery. The STAC-BBB capsid, born out of Sangamo's SIFTER capsid platform, excelled in brain delivery and showed 700-fold higher transgene expression compared to the benchmark capsid AAV9.

Its capability to deliver zinc finger epigenetic regulators led to the potent repression of disease-related genes, hinting at a transformative impact on treating prion diseases and tauopathies. This capsid variant's precision in targeting the CNS, coupled with its de-targeting of peripheral tissues like the liver, underscores its potential in refining neurotherapy with a favourable safety profile.

Sangamo is poised to integrate STAC-BBB into its neurology-focused endeavours, eyeing IND submissions for various programs by the end of 2025. The firm's strategic shift towards neurology, leveraging this capsid's capabilities, could rejuvenate paused projects and forge new collaborations, offering hope for advancements in genomic medicines for neurological conditions.

The new preclinical data was shared yesterday in a press release from Sangamo. You can read it here.