EdiGene and Peking Union Medical College Hospital Collaborate on LEAPER™-Based Gene Editing Therapy for Inherited Retinal Dystrophy

On November 23, 2021, Boya jiyin (Beijing) Biotechnology Co., Ltd. reached a research collaboration with Professor Rui Fang Sui's team from Peking Union Medical College Hospital to explore targeted in vivo gene editing therapies based on the genetic mutation characteristics of China's inherited retinal degeneration (IRD) population. Boya jiyin is a global biopharmaceutical company at the clinical stage, focusing on the translation of gene editing technologies and committed to developing innovative therapies for difficult-to-treat genetic diseases and cancers.

Professor RuiFang Sui's team focuses on the clinical and pathogenic gene research of eye genetic diseases. Through large-scale studies, they have obtained a unique gene variation spectrum for various IRD patients in China. According to the cooperation agreement, specific pathogenic gene variations will be selected as targets to evaluate the efficacy and safety of Boya jiyin(Beijing)Biotechnology Co., Ltd.'s proprietary LEAPER™ RNA base editing technology, in order to advance the development process of in vivo gene editing therapy for this genetic disease.

"LEAPER™ technology does not require the introduction of exogenous proteases but achieves precise RNA editing by recruiting endogenous ADAR proteins through specially designed arRNA, showing broad application prospects in various disease fields, including genetic disorders," said Dr. Pengfei Yuan, Chief Technology Officer of Boya jiyin (Beijing) Biotechnology Co., Ltd. "The collaboration with Professor Sui’s team will accelerate the transformation of LEAPER™ technology into in vivo gene-editing therapies, thereby helping patients as soon as possible."

"Many current studies and data on genes and phenotypes of genetic diseases are based on reports from Europe and the U.S. However, the mutation sites and characteristics of many genetic diseases in the Chinese population differ from those in Europe and the U.S. We have already identified the genetic variation characteristics of multiple eye genetic diseases in the Chinese population, providing targets for the clinical translational research of LEAPER™, a cutting-edge gene-editing technology in China," said Professor Rui Fang Sui from Peking Union Medical College Hospital. "We will fully leverage our team’s years of research and practical experience to actively explore innovative therapies suitable for Chinese patients."

"LEAPER™ technology was invented by the laboratory of Professor Weisheng Wei, the scientific founder of Boya jiyin(Beijing)Biotechnology Co., Ltd. and a professor at Peking University. This collaboration with Peking Union Medical College Hospital is based on the characteristics of China's specific genetic disease population. Through our independent research and development efforts, we are transforming LEAPER™ technology into an innovative therapy to help these patients, which represents a practical implementation of advancing cutting-edge technology from the laboratory to medical institutions and enterprises through industry-academia-research cooperation," said Dr. Dong Wei, CEO of Boya jiyin. "As our internal R&D capabilities continue to grow and more external research collaborations are established, we will accelerate the transformation of gene-editing technologies and explore more treatment solutions in China, allowing innovative therapies to benefit patients as soon as possible."

Boya Jiyin (Beijing) Biotechnology Co., Ltd. has four major R&D platforms based on gene editing technology, developing ex vivo gene editing therapies, in vivo gene editing therapies, and targeted therapies. Currently, the company’s gene-edited hematopoietic stem cell research product ET-01 for transfusion-dependent β-thalassemia is undergoing a multi-center Phase I registrational clinical trial.

Editor: Liuli

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