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TG-LBP
- Development stage
- Preclinical
- Lead developer
- ToolGen
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intravenous
01
Overview
TG-LBP is a preclinical-stage CRISPR-based gene therapy being developed by ToolGen for the treatment of Hemophilia B. The therapy utilizes ToolGen's proprietary CRISPR/Cas9 genome editing technology to target hepatocytes in the liver, aiming to restore the production of functional Coagulation Factor IX (FIX). By providing a durable genetic correction or insertion, TG-LBP seeks to offer a long-term therapeutic solution that could potentially eliminate the need for frequent factor replacement therapy in patients with this bleeding disorder.
02
Targets
ATIII (Antithrombin III)APOC3 (Apolipoprotein C-III)
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