[Edaily Reporter NA EUN-KYUNG ] ToolGen Incorporated(199800)is set to begin Good Laboratory Practice (GLP) toxicity testing for TGT-002 (GEB-200), an in vivo gene-editing therapy targeting atherosclerotic cardiovascular disease (ASCVD).

ToolGen Incorporated announced on the 3rd that it is conducting the preclinical development of TGT-002 in collaboration with its co-development partner, GenEditBio, and has now fully entered the GLP toxicity testing phase in preparation for clinical trials.

TGT-002 is a gene-editing therapy currently under development with the goal of lowering lipoprotein(a) (Lp(a)), a risk factor for atherosclerotic cardiovascular disease. Lp(a) is a complex formed by the binding of apolipoprotein (a) to a particle similar to low-density lipoprotein (LDL); it is known that higher blood concentrations of Lp(a) increase the risk of cardiovascular diseases such as atherosclerosis, thrombosis, and aortic valve calcification.

Data released by ToolGen Incorporated also shows a positive correlation: as Lp(a) concentrations rise, the risk ratio for atherosclerotic cardiovascular disease increases accordingly. Since Lp(a) concentrations are significantly influenced by genetic factors, the development goal of TGT-002 is to continuously lower Lp(a) by directly correcting the causative gene in vivo.

GenEdit Bio is a clinical-stage biotech company headquartered in Hong Kong that specializes in lipid nanoparticle (LNP) delivery technology for in vivo gene-editing therapeutics. GenEdit Bio’s LNP delivery technology is also being applied to TGT-002. According to ToolGen Incorporated, preliminary evaluations in animal models and non-human primates (NHPs) confirmed the editing efficiency for the target gene. Based on these results, GeneEditBio has completed efficacy evaluations in the preclinical phase. However, specific dosage, gene editing rates, and Lp(a) reduction rates were not disclosed at this time.

Work on pharmaceutical manufacturing and quality control (CMC) is also proceeding in parallel. GLP-level production and quality verification for key raw materials and formulations have been completed, and the company plans to proceed with the production of clinical trial materials in accordance with Good Manufacturing Practice (GMP) standards.

Meanwhile, ToolGen Incorporated signed a strategic cross-licensing agreement with JinEditBio in November of last year. The agreement involves the joint development of TGT-002 by combining ToolGen Incorporated’s CRISPR-Cas9 gene-editing technology with JinEditBio’s LNP delivery technology. The two companies are conducting preclinical and clinical development and plan to jointly pursue global technology transfer in the future.

ToolGen Incorporated official stated, “We are proceeding with development with the goal of submitting an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in 2027, by ensuring the smooth execution of the ongoing preclinical trials and the establishment of a manufacturing development package.”