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JUST-AAV-PPT1
- Development stage
- Preclinical
- Lead developer
- JCR Pharmaceuticals
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
JUST-AAV-PPT1 is an investigational gene therapy developed by JCR Pharmaceuticals for the treatment of Neuronal Ceroid Lipofuscinosis Type 1 (CLN1), also known as infantile Batten disease. It utilizes the proprietary JUST-AAV platform, which features an adeno-associated virus (AAV) capsid engineered to display a miniaturized anti-transferrin receptor (TfR) antibody. This modification enables the vector to bind to transferrin receptors on the blood-brain barrier, facilitating efficient transcytosis and widespread delivery of the palmitoyl-protein thioesterase-1 (PPT1) transgene to the central nervous system following systemic (intravenous) administration. In preclinical models, JUST-AAV-PPT1 has demonstrated superior survival benefits, reduced neuroinflammation, and preserved cognitive and motor functions compared to conventional AAV9 vectors and enzyme replacement therapies.
- Other names
- JUST-AAV-CLN1JUST-AAV-CLN-1JUST-AAV-CLN 1
02
Targets
TFRC (Transferrin Receptor)
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