Therapeutics

For a healthier life without diseases, we seek to combine genome editing technology with gene/cell therapy to develop novel therapies.

Overview

Therapeutics Division aims to develop ‘Genomic Cure’ for incurable diseases. Based on CRISPR-Cas9 application and deep expertise of therapeutic development, we seek to provide novel solution for both genetic and non-genetic diseases.

By delivering CRISPR-Cas9 directly into the body with the help of clinically translatable delivery vehicles, we established in vivo therapeutic pipelines. On the other hand, by transferring CRISPR-Cas9 treated cells isolated from patients or healthy donors, we established ex vivo therapeutic pipelines.

..

Pipeline

In Vivo

TGT-001 | Charcot-Marie-Tooth Disease

PNS

Program(Disease)

In vitro POC

In vivo POC

Lead Optimization

Pre-Clinical

IND

Phase 1

TG-LBP | Hemophilia B

Liver

Program(Disease)

In vitro POC

In vivo POC

Lead Optimization

Pre-Clinical

IND

Phase 1

Ex Vivo

Next-generation CAR-T

Immune cell

Program(Disease)

In vitro POC

In vivo POC

Lead Optimization

Pre-Clinical

IND

Phase 1

CREAM

Engineered MSC

Program(Disease)

In vitro POC

In vivo POC

Lead Optimization

Pre-Clinical

IND

Phase 1