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BEIJING

EdiGene, Inc., a clinical-stage biotechnology company focused on developing transformative gene-editing therapies, announced an oral presentation on the preclinical proof-of-concept (POC) data in non-human primate (NHP) model, including NHP disease model, for LEAPER^TM 2.0-based in vivo RNA editing therapies at the 26th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) to be held on May 16-20, 2023 in Los Angeles, California. LEAPER^TM 2.0 is our proprietary exogenous protein-free RNA base editing technology that uses engineered circular ADAR-recruiting RNAs.

Data from specific NHP disease model have demonstrated the preclinical safety and efficacy of LEAPER^TM 2.0-based in vivo therapeutic approach with outstanding editing efficiency. With endogenous ADAR, LEAPER^TM has achieved precise and highly efficient RNA base editing using only one guide RNA (arRNA). Additionally, the exogenous protein-free feature significantly reduces delivery difficulty and minimizes the potential for immune response induced by foreign proteins. Algorithms are also developed to limit bystander effects, ensuring the high specificity of LEAPER^TM 2.0. These results represent a significant step forward in the development of novel therapies.

“We are thrilled about the tremendous potential of our in vivo RNA editing therapeutic approach, which may have the capability to address significant unmet medical needs by enabling A-to-G editing, and these needs are not limited to correcting G-to-A mutations.” said Pengfei Yuan, Ph.D., Chief Technology Officer of EdiGene. “Our team is relentlessly working to advance our leading programs into life-changing therapies for patients, with an initial focus on ophthalmic, neuromuscular, and CNS diseases, through both our internal efforts and external collaborations.”

Details of the presentation:
Title: Efficient and Safe RNA Editing in Non-Human Primates Using AAV Delivered LEAPER Agents
Session Date/Time: Thursday, May 18, 2023, 3:45 PM – 5:30 PM
Session title: Gene Targeting and Gene Correction: New Technologies
Room: Room 515 AB
Presentation Time: 5:00 pm – 5:15 pm
Final abstract number: 233

About EdiGene, Inc.

EdiGene Inc. is a clinical-stage company focusing on translating gene-editing technologies into transformative genetic medicines for patients with significant unmet medical needs including neuromuscular diseases, ophthalmology, CNS diseases, hematology, and oncology. Anchored by its proprietary technologies of gene editing, bioinformatics, and high throughput genome-editing screening, EdiGene advances the pipeline on therapeutic platforms of LEAPER^TM-based in vivo RNA base editing, ex vivo gene-editing hematopoietic stem cells, and ex vivo gene-editing T cells for allogeneic CAR-T. More information can be found at https://www.EdiGene.com.

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