医療従事者の為の最新医療ニュースや様々な情報・ツールを提供する医療総合サイト

EdiGene to Unveil Promising Preclinical POC Data for LEAPER™ 2.0-based in vivo RNA Editing Therapies in NHP Model at the 26th Annual Meeting of ASGCT via Oral Presentation

2023年05月03日 AM05:30
このエントリーをはてなブックマークに追加


 

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 LEAPERTM 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. LEAPERTM 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 LEAPERTM 2.0-based in vivo therapeutic approach with outstanding editing efficiency. With endogenous ADAR, LEAPERTM 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 LEAPERTM 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 LEAPERTM-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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20230502005658/en/

CONTACT

Xiaomeng Zhang

EdiGene, Inc.

+86 10-80733899

media@edigene.com

同じカテゴリーの記事 

  • Exscientiaがゴールドマン・サックスの第44回年次国際医療カンファレンスに出席
  • Vulcan FHIR® Accelerator Connects CDISC, HL7, and ICH M11 in a Project to Digitize Exchange of Clinical Research Protocols
  • Exscientia将出席Goldman Sachs第44届全球医疗保健年会
  • Sequoia India & Southeast Asia Is Now Peak XV Partners
  • LUNARピボタル試験で、プラチナ製剤治療後の進行したステージ4のNSCLC患者の全生存期間に統計的に有意で臨床的に意義のある延長を確認