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SPK-10001
- Development stage
- Unknown
- Lead developer
- Roche
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intraparenchymal
01
Overview
**SPK-10001** is an investigational adeno-associated virus (AAV)-based gene therapy developed for Huntington's disease (HD). It is designed to deliver a transgene encoding a microRNA into brain cells, specifically targeting the caudate and putamen via bilateral intraparenchymal infusion. This microRNA reduces both normal and mutant huntingtin (HTT) mRNA and protein in a nonallele-specific manner, aiming to decrease mutant HTT accumulation responsible for neurodegeneration in HD. The therapy is intended for durable, long-lasting HTT reduction after a single dose, with preclinical data indicating a dose-dependent effect, minimal off-target distribution, and neuronal safety in non-human primates. SPK-10001 is currently undergoing early-phase clinical trials to assess its safety, tolerability, and efficacy in humans with genetically confirmed Huntington's disease[1][2][3][4][5].
- Brand names
- SPK-10001SPK10001SPK 10001
- Other names
- SPK-10001SPK10001SPK 10001
02
Targets
HTT mRNA (HTT / Huntingtin gene)
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