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LTS-101

Development stage: Preclinical
Lead developer: Latus Bio
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration: Intracerebroventricular

Overview

LTS-101 is an investigational gene therapy being developed by Latus Bio for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease. This rare, inherited neurodegenerative disorder is characterized by a deficiency in the tripeptidyl peptidase 1 (TPP1) protein, leading to lysosomal dysfunction and progressive neurodegeneration. LTS-101 utilizes an adeno-associated virus (AAV) vector delivered intracerebroventricularly to introduce a functional copy of the TPP1 gene into patients' central nervous system cells, aiming to restore TPP1 activity and slow or halt disease progression[1][4]. The therapy is currently in preclinical or early clinical development.

Targets

TPP1 (Tripeptidyl peptidase 1)

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