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EdiGene Enters Research Collaboration to Develop Gene-editing Therapies for Inherited Retinal Degeneration with Peking Union Medical College Hospital

Time:21-11-23 09:00 Form: EdiGene Views:475

BEIJING and CAMBRIDGE, MASS– (November 23, 2021) – EdiGene, Inc., a global biotechnology company focused on translating gene-editing technologies into transformative therapies for patients with serious genetic diseases and cancer, has entered into a research collaboration with Professor Sui Ruifang’s team at Peking Union Medical College Hospital to promote the development of in vivo gene-editing therapies based on the genetic characteristics of inherited retinal degeneration (IRD) in China.

Professor Sui’s team focuses on the pathogenic mechanisms of inherited retinal diseases. Through large sampling studies and clinical research, the team has obtained multiple, unique gene variant characteristics of IRD patients in China. By selecting specific pathogenic genes as targets, the collaborative research will evaluate the efficacy and safety of LEPAER™, EdiGene’s proprietary RNA base editing technology, to facilitate the development of in vivo gene-editing therapies to treat the condition.

“LEAPER™ employs uniquely engineered ADAR-recruiting RNAs (arRNAs) to enable precise RNA editing with broad therapeutic applicability in multiple diseases,” said Pengfei Yuan, Ph.D., Chief Technology Officer of EdiGene. “This collaboration allows us to accelerate the translation of LEAPER™ technology into in vivo gene-editing therapies with the potential to benefit patients in the near future.”

“Most current genetic and phenotypic studies and data of inherited diseases are from the United States and European countries. The mutation loci and characteristics of many genetic diseases of the Chinese population are not the same as those of the U.S. and Europe,” said Professor Sui. “We will leverage our extensive experience and expertise in translational research and practice to actively develop innovative therapies for Chinese patients.”

“LEAPER™ is developed by Professor Wensheng Wei’s lab at Peking University. Professor Wensheng Wei is the Scientific Founder of EdiGene. Through this industry-university-research collaboration, we will be able to address a significant gap in the development of genetic medicines by focusing on mutations that drive inherited retinal disease in China,” said Dong Wei, Ph.D., CEO of EdiGene. “Our R&D capabilities and knowledge of the characteristics of patients with genetic diseases in China will speed the translation of EdiGene’s proprietary LEAPER™ technology into innovative therapeutic options for patients.”


EdiGene is developing ex vivo gene-editing therapies, in vivo gene-editing therapies, and targeted therapies on its four, genome editing-based platforms. ET-01, EdiGene’s gene-editing hematopoietic stem cell therapy for transfusion-dependent β-thalassemia, is in a multicenter Phase I clinical trial.

About Prof. Ruifang Sui

Prof. Ruifang Sui, Ph.D. is the Director of the Department of Ophthalmology at Peking Union Medical College Hospital. She is also the Vice-President of the Chinese Medical Doctor Association Ophthalmology Committee, and the Councilor of the Chinese Ophthalmology Genetics Alliance. Prof. Sui has been engaged in clinical and applied research for genetic and congenital retinal diseases for more than 20 years. Her team was the first to publish a cohort study and retinal genetic disease cases of multiple Chinese inherited retinal degenerations, identified multiple clinical and genetic variant characteristics of patients in China, and discovered eight new inherited retinal degeneration genes, as well as a new high myopia causative gene. She has pioneered gene therapy research on inherited retinal degeneration in China and has led more than ten national- and provincial-level projects. She is the only Chinese scholar to have won the Career Development Award and Individual Award from the American Foundation for the Blind. Her leading Project on Innovation, Research and Application of Precision Diagnosis and Treatment for Genetic Retinal Diseases won the China Medical Science and Technology Award.


LEAPER™ (Leveraging endogenous ADAR for programmable editing of RNA) is an RNA base editing technology. It employs short engineered ADAR-recruiting RNAs (arRNAs) which recruits endogenous ADAR to change a specific adenosine to inosine. LEAPER™ is co-developed by EdiGene and the lab led by Professor Wensheng Wei, Founder of EdiGene, in 2019. As a single-molecule system, LEAPER™ enables precise, efficient RNA editing with broad applicability for therapy and basic research.

About EdiGene, Inc

EdiGene is a global, clinical-stage biotechnology company focused on translating gene editing technologies into transformative therapies for patients with serious genetic diseases and cancer. The company has established its proprietary ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies. Founded in 2015, EdiGene is headquartered in Beijing, with offices in Guangzhou and Shanghai, China and Cambridge, Massachusetts, USA. More information can be found at www.EdiGene.com.