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SLP-2 AAV
- Development stage
- Preclinical
- Lead developer
- Université Laval
- Modality
- Gene Therapies
- Administration
- Intracerebral
01
Overview
SLP-2 AAV is a preclinical gene therapy candidate designed to treat Parkinson's disease by overexpressing Stomatin-like protein 2 (SLP-2) in dopaminergic neurons. SLP-2 is a protein localized to the inner mitochondrial membrane where it acts as a membrane scaffold, regulating mitochondrial integrity, function, and bioenergetics. Research indicates that SLP-2 levels are reduced in the substantia nigra of Parkinson's patients, contributing to mitochondrial dysfunction and increased vulnerability to alpha-synuclein toxicity. By utilizing an adeno-associated virus (AAV) vector to deliver the SLP-2 gene directly into the substantia nigra pars compacta, the therapy aims to rescue mitochondrial respiration and protect neurons from neurodegeneration. The program is primarily being developed within academic and research institutions in Quebec, Canada.
- Other names
- Adeno-associated virus-Stomatin-like protein 2
02
Targets
HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))Sia (N-linked sialic acid)SLP-2 (Stomatin-like protein 2)
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