ToolGen

Therapeutics

Diverse Pipelines of Cell and Gene therapies

Therapeutics Development

ToolGen is committed to bringing a healthier life to humanity by developing therapeutics, based on CRISPR/Cas system.
Therapeutics under way in ToolGen is as below:

In vivo: CNS/PNS

Program(Disease)	Modeling	In vitro	In vivo	Lead Optimization	Pre- Clinical	IND	Phase 1
TGT-001 Charcot-Marie-Tooth Disease
TGT-001is a therapeutic genome editing program for CMT1A. CMT1A, from which more than 1 million patients around the world are suffering, is a genetic disease caused by the duplication of genomic locus containing PMP22 gene. This duplication and subsequent overexpression of PMP22 in Schwann cell cause the demyelination in peripheral nervous system. There are no therapeutics for CMT1A in market yet. TGT-001 is a gene therapy targeting the fundamental cause of CMT1A. By normalizing PMP22 expression in Schwann cell by genome editing, TGT-001 can be the first cure for CMT1A. TGT-001 is currently in an IND-enabling stage.

In vivo: Eye

Program(Disease)	Modeling	In vitro	In vivo	Lead Optimization	Pre- Clinical	IND	Phase 1
TGT-101 Age-related Macular Degeneration
TGT-101is genome editing therapy for ocular disease involving neovascularization such as wet AMD and diabetic retinopathy. VEGF is a well-established target for wet AMD and diabetic retinopathy. Current therapeutic intervention uses protein-based inhibitors for VEGF. These drugs require repeated use every several weeks creating a need for therapeutics with long-term effect. TGT-101 targets VEGF with CRISPR/Cas9 delivered by adeno-associated virus, which could establish a very long-term therapeutic window. TGT-101 is currently in a late discovery stage to confirm its efficacy in large animals.

In vivo: Liver

Program(Disease)	Modeling	In vitro	In vivo	Lead Optimization	Pre- Clinical	IND	Phase 1
TG-LBP Hemophilia B
Liver biofactory platform (LBP)is a gene therapy approach to establish a long-term, potentially permanent, expression of therapeutic protein in patients with genetic/incurable diseases requiring a life-long and repeated use of protein drugs. By delivering therapeutic gene with CRISPR/Cas9 system to the liver of patient, the stable and safe insertion of therapeutic gene into the liver cell can be achieved. Such therapeutic gene inserted into a genomic locus can produce protein drug in patient body possibly without a gradual loss of therapeutic gene by liver cell regeneration or growth of pediatric patients. LBP is initially applied to hemophilia B program, which is currently late discovery stage with therapeutic efficacy validated in animal models.
TG-HBV Hepatitis B
TG-HBVaims to provide a cure for chronic hepatitis B infection by targeting viral DNA in infected liver cells. Chronic HBV infection is cause of many liver diseases including liver cancer and cirrhosis The genome of HBV in infected cell exist in a form called cccDNA whose elimination is not achieved with current anti-HBV drugs. By using a combination of multiple sgRNA for HBV genome, Cas9 mRNA and a lipid nanoparticle delivery, TG-HBV are expected to efficiently target multiple HBV strains in patient liver cells.

Ex vivo: CAR-T

Program(Disease)	Modeling	In vitro	In vivo	Lead Optimization	Pre- Clinical	IND	Phase 1
Next-generation CAR-T
Genome editing can be used to establish a better immune cell therapy platform in various ways by stably modifying gene function related to the efficacy and safety of immune cells. One of such effort is our Styx immune cell platform which involves the inhibition of DGK genes in immune cell. DGKs are well-established negative regulator of immune cells including T and NK cells. Therapeutic modulation of DGK genes, however, is difficult for their location in cytoplasm and also there multifaceted functions in many different cell types. CAR-T with knockout of DGK genes show improved efficacy and persistency in cellular and animal models to various targets (CARs) and target cancer cells. With our expertise in immune cell genome editing and Styx immune cell platform, we aim to establish multiple partnerships with cell therapy companies to provide more potent immune cell therapeutics for patients suffering from various tumors.

Address . (08501) #1204, Byucksan Digital Valley 6-cha, 219 Gasan Digital 1-ro, Geumcheon-gu, Seoul, South Korea

Business registration number . 108-81-18555

E-mail . info@toolgen.com TEL . 02-873-8168 FAX . 02-873-8169

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