ENCell said Wednesday that it has filed a patent application for its adeno-associated virus (AAV) platform technology for treating inherited retinal pigmentary disease (IRD).

IRD is a genetically inherited condition that causes photoreceptors in the eye to perish, leading to the progressive destruction of optic nerve cells and retinal pigment epithelial cells. It affects one in every 2,500 to 3,000 people worldwide, with an estimated 20,000 patients in Korea.

ENCell has identified a candidate AAV vector that can be delivered specifically to photoreceptor cells in the eye. This patented technology enables precise gene delivery to ocular cells and tissues and improves expression efficiency.

AAV is a key vector for gene therapeutics due to its low immunogenicity and lack of integration into the genome. ENCell's proprietary AAV production technology and efficient viral vector production system have enabled the company to reduce production costs and improve productivity.

ENCell plans to develop AAV-based gene therapy products as the next growth engine. The company is also developing the original technology for muscle-related AAV platforms beyond eye diseases. In addition, it recently completed developing an efficient mass production process for AAV-based gene therapies in collaboration with Sartorius Korea.

“As a leading contract development and manufacturing organization (CDMO) for advanced biopharmaceuticals, we have intensively fostered the field of AAV-based therapeutics,” an ENCell official said. “Based on our excellent technology and world-class GMP capabilities, the field of AAV gene therapy will become a new growth engine for the company.”