EdiGene Presents Preclinical Data of LEAPER™ 2.0-Based In Vivo RNA Editing Therapy in Non-Human Primate Models at ASGCT 2023

Recently, Boya jiyin (Beijing) Biotechnology Co., Ltd. announced that at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), the company will present preclinical proof-of-concept data of its in vivo RNA editing therapy in non-human primate (NHP) models, including disease models, in the form of an oral presentation. This therapy is developed based on the company's proprietary LEAPER™ 2.0 technology, which does not require the introduction of exogenous proteins and achieves RNA editing through specially designed circular RNA. The conference will be held in Los Angeles, California, USA, from May 16 to 20, 2023. Boya jiyin is a clinical-stage biopharmaceutical company focused on the translation of gene editing technologies, dedicated to developing transformative gene editing therapies to meet unmet clinical needs.

Data from specific non-human primate (NHP) disease models demonstrate the preclinical safety and efficacy of LEAPER™ 2.0-based in vivo RNA editing therapeutic candidates, showcasing excellent editing efficiency. LEAPER™ utilizes specially designed arRNA (ADAR-recruiting RNA) to recruit endogenous ADAR proteins (Adenosine Deaminase Acting on RNA), achieving highly efficient and precise RNA editing. Moreover, the feature of not requiring exogenous protein significantly reduces delivery complexity and avoids related immunogenicity issues. We have also developed algorithms to limit bystander effects, ensuring the high specificity of LEAPER™ 2.0. These achievements indicate that our efforts to translate LEAPER™ 2.0 into a novel therapy have made substantial progress.

"The tremendous potential of this in vivo RNA editing therapy is exciting, as it has the potential to address many unmet therapeutic needs through A-to-G editing, particularly not limited to correcting G-to-A mutations," said Dr. Pengfei Yuan, Chief Technology Officer of Boya jiyin (Beijing) Biotechnology Co., Ltd. "Our team is consolidating internal resources and expanding external collaborations to advance leading projects, with the goal of developing innovative therapies that can truly benefit patients and make a difference. We are currently focusing primarily on areas such as ophthalmology, neuromuscular, and central nervous system diseases."

▌Oral Report Details

• Title: Safe and Efficient RNA Editing in Non-Human Primates (NHP) Achieved by Delivering LEAPER™ via AAV

• Meeting Time: Friday, May 19, 2023, 3:45 am – 5:30 am (Beijing Time)

• Conference Theme: Gene Targeting and Gene Correction: New Technologies
  

• Venue: 515 AB
  

• Report Time: 5:00 am - 5:15 am (Beijing Time)
  

• Abstract Number: 233

▌About Boya jiyin

EdiGene, Inc. is a clinical-stage biopharmaceutical company focused on the development of gene-editing therapies. It is dedicated to bringing transformative treatments to patients in disease areas such as blood disorders, oncology, ophthalmology, and the central nervous system. Leveraging cutting-edge technological innovation in gene editing, high-throughput genomic editing screening, and bioinformatics, the company advances internationally leading ex vivo and in vivo gene-editing therapy development. EdiGene has established multiple therapeutic platforms, including an ex vivo hematopoietic stem cell platform, an ex vivo universal CAR-T platform, and an in vivo RNA base editing platform. For more information, please visit the company’s official website: www.EdiGene.com.