On December 23, 2025, GT307 injection , a next-generation gene-knockout tumor-infiltrating lymphocyte (TIL) product independently developed by Sapphire Biotech , received Investigational New Drug (IND) approval from the U.S. Food and Drug Administration (FDA) . This marks another significant milestone for Sapphire Biotech in the field of high-safety gene-edited TILs, following the completion of clinical trial applications for its GT201 product in China and the United States. It also signifies that multiple cell therapy pipelines of the company have entered the clinical stage in the United States.

GT307 is a next-generation dual-gene knockout TIL product based on CRISPR gene editing technology, designed to address key bottlenecks in traditional TILs, such as functional exhaustion and insufficient persistence in the tumor microenvironment. Leveraging the ImmuT Finder® high-throughput immunomodulatory target screening platform independently developed by SandGrey Biotech, the company systematically discovered and identified key immunomodulatory factors that inhibit the anti-tumor function of TILs, and precisely knocked them out using CRISPR technology, thereby significantly enhancing the anti-tumor function and in vivo persistence of TILs.

In terms of gene editing strategy, GT307 adopts a high-fidelity CRISPR/AaCas12b MAX nuclease system, which shows significant advantages over the classic SpCas9 nuclease in terms of editing safety, product phenotype, amplification and function. The relevant research results have been published in " Molecular Therapy ", providing key scientific evidence for the clinical application of AaCas12b MAX nuclease in the field of cell therapy (DOI: 10.1016/j.ymthe.2025.09.009) .

Based on this gene-editing technology, SandGrey Biotech established a systematic safety assessment framework compliant with FDA guidelines , covering key dimensions such as off-target risk and chromosomal structural variations. This assessment framework systematically supported the preclinical safety studies of GT307 injection. The results showed that GT307 injection achieved zero off-target effects and an extremely low frequency of chromosomal structural variations, providing strong data support for its gene-editing safety. Safety studies, as a crucial component of the application materials, together with core modules such as CMC and clinical studies, constituted a complete application, supporting the successful acquisition of FDA IND approval for GT307 injection. During the FDA's review and communication process, the safety studies did not trigger further supplementary research requirements, demonstrating the reliability and applicability of this safety assessment protocol in actual applications, and providing a valuable practical reference for international clinical applications of gene-edited cell products.

The FDA IND approval for GT307 injection fully demonstrates Sapphire Biotech's comprehensive strength in the design and development of next-generation TIL products, gene editing safety control, process development and quality control, and the construction of an international regulatory compliance system. As the world's only TIL product with proven efficacy in cold tumors, GT307 validates the clinical feasibility of its innovative target selection and product strategy, establishing its differentiated competitive advantage in the field of solid tumor TILs.

Currently, the IND application for GT307 injection in China is also progressing actively. In the future, SandGrey Biotech will leverage its dual clinical research platforms in China and the United States to accelerate the clinical research of GT307 in various advanced or metastatic solid tumors, providing more cancer patients with cell therapy options with breakthrough potential.

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