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CLN2 Disease - Ocular program
- Development stage
- Preclinical
- Lead developer
- Latus Bio
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies
- Administration
- Ophthalmic
01
Overview
CLN2 Disease - Ocular program is an investigational adeno-associated virus (AAV) gene therapy being developed by Latus Bio for the treatment of ocular manifestations associated with CLN2 disease (late infantile neuronal ceroid lipofuscinosis). CLN2 disease is caused by mutations in the TPP1 gene, leading to a deficiency in the tripeptidyl-peptidase 1 enzyme, which results in progressive neurodegeneration and vision loss. This program utilizes Latus Bio's proprietary AAV.Ep+ capsid family to deliver a functional TPP1 gene specifically to the eye, aiming to restore enzyme activity and prevent or slow the progression of retinal degeneration. It is currently in the discovery/preclinical stage of development.
- Other names
- Latus Bio-TPP1-AAV gene therapy-CLN2 Disease (Ocular)
02
Targets
TPP1 (Tripeptidyl peptidase 1)
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