Shape Therapeutics to Present Breakthrough RNA Editing Data for CNS Therapies at ASGCT Annual Meeting
核心洞察
Shape Therapeutics (搜索) will showcase multiple presentations at ASGCT 2025, including data demonstrating up to 88% targeted RNA editing (搜索) in primate brain using systemically delivered AAV5 (搜索)-based capsid variants.
The company's innovative approach combines programmable RNA editing (搜索) technology (RNAfix (搜索)®) with targeted AAV.ai (搜索) delivery systems and TruStable (搜索)™ manufacturing platforms to advance gene therapy development.
Encoded Therapeutics (搜索) will also present at ASGCT, highlighting progress in their CNS gene therapy programs for Angelman syndrome (搜索), chronic pain (搜索), and Alzheimer's disease (搜索) using engineered AAV vectors.
Shape Therapeutics (搜索) announced multiple presentations at the upcoming American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, showcasing significant advancements in RNA-based gene therapy. The meeting, scheduled for May 13-17, 2025, in New Orleans, will feature two oral presentations and multiple poster sessions from the company.
Groundbreaking RNA Editing Technology Shows Promise for CNS Disorders
Shape Therapeutics (搜索) will present compelling data demonstrating the potential of its programmable RNA editing (搜索) technology for central nervous system (CNS) applications. Most notably, an oral presentation by Dr. Rick Sullivan will highlight how systemically delivered AAV5 (搜索)-based capsid variants achieved up to 88% targeted RNA editing in primate brain—a significant milestone for CNS-targeted gene therapies.
"We look forward to sharing a bolus of data at this year's ASGCT as it builds on our scientific body of evidence that support the development of our programmable RNA medicines to shape the future of gene therapy through precise RNA editing (搜索), targeted AAV.ai (搜索) delivery and scalable manufacturing," stated David Huss, Ph.D., CSO and acting CEO of Shape Therapeutics (搜索).
The company's approach integrates three key technological platforms: RNAfix (搜索)® for programmable RNA editing (搜索), AAV.ai (搜索) for targeted delivery, and TruStable (搜索)™ for scalable manufacturing. This comprehensive strategy aims to overcome traditional limitations in gene therapy development.
Manufacturing Innovations Address Production Challenges
A second oral presentation by Dr. Sandhya Pande will focus on "Mechanistic Insights Enhance Multi-Serotype Production in the TruStable (搜索)™ AAV Producer Cell Line," addressing one of the industry's most significant challenges—reliable and scalable manufacturing of AAV vectors.
Ken Prentice will present a poster comparing the TruStable (搜索)™ system with transient production systems for AAV vector manufacturing, potentially offering solutions to production bottlenecks that have historically limited gene therapy commercialization.
Encoded Therapeutics Also Showcasing CNS Gene Therapy Advancements
In parallel developments, Encoded Therapeutics (搜索) will present new data for their CNS gene therapy programs at the same ASGCT meeting. Their presentations will focus on vector engineering for Angelman syndrome (搜索) (ETX201), chronic pain (搜索), and Alzheimer's disease (搜索)/tauopathies (搜索).
"Our vector engineering platform continues to demonstrate its potential to bring life-changing therapies to patients," commented Stephanie Tagliatela, Chief Scientific Officer and co-founder of Encoded Therapeutics (搜索). "Specifically, in our Angelman syndrome (搜索) and Alzheimer's/tauopathies (搜索) programs, new findings further validate the ability of our engineered vectors to modulate disease-relevant genes in bulk NHP CNS tissue with AAV9 (搜索)."
Encoded's approach differs slightly, utilizing AAV9 (搜索)-based vectorized miRNA therapeutic candidates. Their poster presentations will showcase data on ETX201 for Angelman syndrome (搜索), reduction of microtubule-associated protein Tau (MAPT (搜索)) in non-human primates for Alzheimer's applications, and development of gene therapy for chronic pain (搜索) through SCN9A (搜索) knockdown.
Industry Implications and Future Directions
These presentations collectively highlight the rapid advancement of RNA-based and AAV-delivered gene therapies for neurological conditions with significant unmet needs. The ability to achieve high editing efficiency in the CNS through systemic delivery represents a potential paradigm shift for treating neurological disorders.
The full schedule of Shape Therapeutics (搜索)' presentations includes:
- Poster: "Programmable RNA Editing Enables Precise Protein Modulation in the Central Nervous System" by Dr. Alison VanSchoiack (May 14)
- Poster: "Comprehensive Evaluation of TruStable™ vs Transient Production Systems of AAV Vector Manufacturing" by Ken Prentice (May 15)
- Oral Presentation: "Systemically Delivered AAV5-Based Capsid Variants Enable up to 88% Targeted RNA Editing in Primate Brain" by Dr. Rick Sullivan (May 16)
- Oral Presentation: "Mechanistic Insights Enhance Multi-Serotype Production in the TruStable™ AAV Producer Cell Line" by Dr. Sandhya Pande (May 16)
As both companies advance their platforms toward clinical applications, the gene therapy field watches closely for validation of these novel approaches that could potentially address the limitations of current gene therapy technologies, particularly for CNS disorders that have proven challenging to treat with conventional methods.