Company secures technology to greatly reduce ‘off-target organ toxicity risk,’ a critical weakness of AAV-based 바카라사이트 제작 therapy
[by Yu, Suin] ENCell, a contract development and manufacturing organization (CDMO) specializing in advanced biopharmaceuticals and new drug development, announced on July 24 that it has recently filed a patent application with the Korean Intellectual Property Office (KIPO) for its core platform technology related to adeno-associated virus (AAV)-based gene therapy targeting muscle-related genetic disorders, titled ‘Chimeric promoter for muscle-specific expression and AAV-based gene expression system using the same.’
According to ENCell, the ‘muscle-specific expression control technology’ described in the patent, developed through the company’s own research, constitutes a core platform capable of addressing key limitations of existing AAV-based gene therapies. The company emphasized that this technology significantly reduces the risk of toxicity by simultaneously targeting skeletal muscle and cardiac muscle while suppressing gene expression in non-target organs such as the liver and testis, thereby overcoming one of the critical drawbacks associated with conventional AAV-based gene therapies.
ENCELL reported that the platform technology was validated using an in vitro muscle cell differentiation model and an in vivo animal model. The research team observed high expression efficiency in skeletal and cardiac muscle following systemic administration of the AAV vector, while expression in non-target organs such as the liver and reproductive organs remained markedly low. These findings suggest that the platform offers better safety in vivo. The company plans to advance development efforts to further confirm its practical potential as a 바카라사이트 제작 therapy platform for treating 바카라사이트 제작tic disorders of the muscular system.
Notably, the occurrence of consecutive patient deaths caused by ‘liver toxicity’ following administration of AAV-based gene therapy developed by the U.S. biotech firm Sarepta Therapeutics raised significant concerns in the industry, to the extent that ensuring the safety of AAV gene therapies became regarded as the most critical challenge. In this context, ENCell emphasized that its newly developed platform technology offers a promising solution. The company noted that the technology’s ability to suppress gene expression in non-target organs, especially those with a high risk of toxicity, while achieving robust expression in therapeutic muscle tissue, positions it as a promising advancement capable of addressing the primary safety issues associated with existing AAV-based gene therapies.
“The muscle-specific expression control technology is significant in that it not only enhances therapeutic efficacy but also addresses the critical safety issue that has been a major concern in AAV-based gene therapy,” an ENCell official said. “Having secured a platform technology capable of simultaneously resolving the dual challenges of efficacy and safety, we will continue to develop this field as a new engine for future growth.”
Conversely, ENCell has also filed a patent application for an AAV platform verification model technology designed to support the development of ocular disease treatments. In addition, the company recently signed a KRW 5.7 billion (approximately USD 4.1 million) contract with the Korea Research Institute of Bioscience & Biotechnology (KRIBB) for the development of an AAV-based 바카라사이트 제작 therapy platform and associated CDMO services. Looking ahead, ENCell plans to further strengthen its technological competitiveness on a global scale, with the strategic goal of establishing itself as a leading platform company in the field of cell and 바카라사이트 제작 therapy (CGT).