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JUST-AAV-TPP1
- Development stage
- Preclinical
- Lead developer
- JCR Pharmaceuticals
- Modality
- Gene Therapies
- Administration
- Intrathecal
01
Overview
JUST-AAV-TPP1 is an investigational adeno-associated virus (AAV) gene therapy being developed by Just-Evotec Biologics, a subsidiary of Evotec, for the treatment of CLN2 disease (late infantile neuronal ceroid lipofuscinosis). CLN2 is a rare, fatal neurodegenerative lysosomal storage disorder caused by mutations in the TPP1 gene, which leads to a deficiency in the tripeptidyl peptidase 1 (TPP1) enzyme. JUST-AAV-TPP1 utilizes the company's proprietary Just-AAV platform to deliver a functional copy of the TPP1 gene to affected cells, aiming to restore enzyme production and mitigate the accumulation of lysosomal storage material. The program leverages Evotec's end-to-end "Access" platform for optimized design and manufacturing of AAV vectors and is currently in the preclinical stage of development.
- Other names
- AAV-TPP1AAV-TPP-1AAV-TPP 1
02
Targets
Adeno-associated virus receptor and glycan complexTPP1 (Tripeptidyl peptidase 1)
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