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Epicrispr GEMS SORL1 activator
- Development stage
- Preclinical
- Lead developer
- Epicrispr Biotechnologies
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Classical Binding Small Molecules → Small Molecules, Covalent Small Molecules → Small Molecules
01
Overview
Epicrispr GEMS SORL1 activator is an experimental epigenetic editing therapy developed by Epicrispr Biotechnologies for the treatment of Alzheimer's disease. The therapy utilizes the company's proprietary Gene Expression Modulation System (GEMS), a non-editing CRISPR-based platform designed to precisely and durably upregulate the endogenous expression of the SORL1 gene. SORL1 encodes SORLA, an endosomal trafficking receptor that plays a critical role in regulating amyloid precursor protein (APP) processing; its deficiency is linked to both familial and sporadic Alzheimer's disease. By increasing SORLA protein levels, the GEMS activator aims to restore proper APP trafficking and significantly reduce the production of neurotoxic beta-amyloid (Aβ40 and Aβ42) and tau peptides. Preclinical data in human iPSC-derived neurons have demonstrated robust transcriptional induction and downstream suppression of amyloid pathology, positioning the asset as a potential disease-modifying strategy for Alzheimer's.
- Other names
- SORL1 activatorSORL-1 activatorSORL 1 activator
02
Targets
SORL1 (Sortilin-related receptor 1)
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