FDA Eases Uncertainty in Gene Therapy Regulations... Green Light for Korean Companies Like Toolgen to Enter Global Markets

The U.S. Food and Drug Administration (FDA) has released a draft of safety guidelines for customized gene therapies targeting patients with ultra-rare diseases. With the establishment of standardized evaluation criteria for the risk of unintended cuts (off-target events) that can occur during gene editing, uncertainty caused by related regulations is expected to decrease. As a result, domestic companies in this field are expected to accelerate their entry into the global market.

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According to industry sources on April 16, the draft recently issued by the Center for Biologics Evaluation and Research (CBER) under the FDA focuses on evaluating unintended editing outcomes of therapies utilizing next-generation sequencing (NGS) technology. The guidelines apply to both ex vivo and in vivo editing products and define the scope of non-clinical research data required for submission with Investigational New Drug (IND) and Biologics License Application (BLA) filings. The draft also contains detailed recommendations on sequencing analysis strategies, sample selection, and analytical parameters.

The urgency for establishing such safety guidelines stems from the characteristics of gene editing technology and the unpredictable regulatory environment. Genome editing therapies, such as those using CRISPR, directly cut or correct the DNA blueprint within cells that cause disease, addressing the root cause of the condition. While considered an alternative for treating rare genetic diseases, there is a risk of off-target side effects, where the technology mistakenly cuts unintended sequences. Such errors can lead to unexpected genetic changes or even new diseases, making rigorous validation essential. However, due to the lack of standardized evaluation criteria, new drug developers have had to accept uncertainty from the clinical design stage. In particular, the FDA has faced criticism recently, as unexpected regulatory decisions have occurred during the review of new rare disease drugs, or advisory committee meetings have not been convened.

With the evaluation criteria now clarified, Korean companies possessing gene-editing scissors technology can collect data that meets global standards. Toolgen, which holds the original patent for CRISPR-Cas9, is preparing for U.S. clinical trials of a treatment for Charcot-Marie-Tooth disease type 1A (CMT1A), a rare genetic disorder. Jincore, which owns a micro-sized gene scissors platform, can also apply the new guidelines in its drug development process to demonstrate safety from the preclinical stage.

Gplus Life Sciences, which has developed an improved CRISPR gene scissors platform, can use NGS-based assessments as an objective indicator to validate the targeting accuracy of its technology. Etgen, which specializes in mitochondrial gene correction technology, has also secured a foundation for preparing clinical trials in line with U.S. regulatory standards. An industry official commented, "The gene therapy sector is gaining attention as a next-generation modality, with major investments from companies like Eli Lilly. As evaluation criteria become clearer, Korean companies will be able to proactively secure safety data for their pipelines and enhance their negotiating power for technology exports."

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