Drug intelligence / Profile preview

Neoclease LRRK2 editor

Development stage
Preclinical
Lead developer
Neoclease
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration
Intrathecal
01

Overview

Neoclease's LRRK2 editor is an investigational, preclinical-stage gene editing therapy designed for the treatment of Parkinson's disease. Developed using the company's proprietary AI-driven platform for designing novel nucleases, the therapy specifically targets the Leucine-rich repeat kinase 2 (LRRK2) gene. Mutations in LRRK2, which lead to increased kinase activity, are one of the most common genetic contributors to both familial and sporadic Parkinson's disease. This gene editing approach aims to modify or disable the LRRK2 gene at the genomic level to modulate its function, reduce pathogenic activity, and potentially offer a disease-modifying treatment that slows or stops neurodegeneration.

Other names
LRRK2 gene editorLRRK-2 gene editorLRRK 2 gene editorLRRK2 nuclease editorLRRK-2 nuclease editorLRRK 2 nuclease editor
02

Targets

LRRK2 (Leucine-rich repeat serine/threonine-protein kinase 2)

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