NORTHERN BIO BLOG: POSTERS

SPK-10001, an AAV-based gene therapy for Huntington’s disease (HD), targets Huntingtin protein (HTT) accumulation in the brain’s basal ganglia. Administered via bilateral intraparenchymal (IP) infusion using MRIguided ClearPoint SmartFlow cannula, SPK-10001 aims for a durable, dose-dependent reduction of HTT protein while minimizing brain tissue disruption and ensuring neuronal tolerance.

In cynomolgus macaques, increasing doses of SPK-10001 demonstrated lasting HTT reduction and safety over 12 months, with detectable transgene in the cerebrospinal fluid (CSF) serving as a durability biomarker. MRI scans showed resolution of surgery-related injuries within two months post-injection, while CSF protein analysis and histopathology confirmed minimal neuronal damage. Overall, SPK-10001’s preclinical data support its further development for Huntington’s disease.