Dyno Therapeutics to Showcase Advanced AAV Capsids for CNS, Eye, and Muscle Gene Delivery at ASGCT Annual Meeting

Dyno Therapeutics, a biotechnology company focused on gene delivery, will present groundbreaking data on its AAV (adeno-associated virus) capsid designs for the central nervous system (CNS), eye, and neuromuscular conditions at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in New Orleans, Louisiana, from May 13 to 17, 2025. The presentations will showcase how Dyno's AI and high-throughput in vivo data collection are optimizing AAV capsids for better efficiency, precise targeting, and manufacturability, ultimately improving the viability and accessibility of gene therapies. During the meeting, Dyno’s co-founder and CEO, Eric Kelsic, Ph.D., will lead a Scientific Symposium titled "Dyno Therapeutics: Leveling Up Genetic Medicine with Frontier AI and AAV Vectors for CNS, Eye, and Muscle." This session, scheduled for Wednesday, May 14, from 12:15 to 1:15 PM CT in Room 391-392, will detail the development of three novel AAV capsids designed for enhanced delivery to the CNS, eye, and neuromuscular system. Dr. Kelsic will also discuss the advanced AI models that facilitated the discovery of these capsids and outline Dyno’s strategy to bring these therapies into clinical trials, focusing on adding new treatments where there is significant unmet medical need. The symposium is complemented by three oral presentations: Widespread CNS Delivery with Best-in-Class Liver Detargeting Following Intravenous Injection of a Novel AAV Presenter: Mugdha Deshpande, Ph.D. Session: AAV Gene Transfer (A): Crossing the Blood-Brain Barrier Date and Time: Wednesday, May 14, 2025, 1:30 PM - 1:45 PM CT Location: New Orleans Theater C Abstract Number: 2220 This presentation will highlight a new AAV capsid designed to efficiently deliver therapeutic genes to the CNS while minimizing liver uptake, a common issue in gene therapy that can lead to toxicity and reduced efficacy. Selective Improvement in Retinal Bipolar Cell Targeting with Intravitreal Injection of a Novel AAV Capsid in Mouse and Non-Human Primate (NHP) Models Presenter: Amanda Miles, Ph.D. Session: AAV Gene Transfer (B): Ocular, Neurological & Immune Cell Systems Date and Time: Friday, May 16, 2025, 2:45 PM - 3:00 PM CT Location: New Orleans Theater A Abstract Number: 2175 Dr. Miles will present data showing a novel AAV capsid that selectively targets retinal bipolar cells, a critical cell type in the eye, after intravitreal injection. The research demonstrates significant improvements in targeting accuracy compared to current vectors. A Novel Neuromuscular AAV Capsid Combines Efficient Systemic Muscle and CNS Delivery with Liver Detargeting in Non-Human Primates (NHP) Presenter: Megan Cramer, Ph.D. Session: AAV Gene Transfer (C): Antibody Evasion, Cardiac & Neuromuscular Targets Date and Time: Saturday, May 17, 2025, 11:45 AM - 12:00 PM CT Location: New Orleans Theater A Abstract Number: 2208 This talk will focus on a new AAV capsid that achieves efficient systemic muscle and CNS delivery while effectively detargeting the liver. The data from NHP models suggests robust therapeutic potential for neuromuscular disorders such as muscular dystrophy. Dyno’s innovative approach combines cutting-edge AI with high-throughput in vivo experiments to identify and optimize AAV capsids. By leveraging these technologies, Dyno aims to overcome the primary hurdle in gene therapy—delivering genetic payloads safely and efficiently to target cells. This capability is crucial for advancing gene therapies for various diseases, including those affecting the CNS, eyes, and muscles, which are often challenging to treat due to their specific anatomical and physiological requirements. The company has partnered with leading gene therapy developers such as Astellas, Roche, and Sarepta, as well as technology giants like NVIDIA, to enhance its platform and broaden its impact. These collaborations underscore Dyno’s commitment to pushing the boundaries of genetic medicine and making advanced therapies more accessible to patients. Industry insiders view Dyno’s work as a significant step forward in the field of gene therapy. The ability to achieve precise and efficient gene delivery could revolutionize the treatment of complex diseases, particularly those where traditional methods fall short. Dyno’s strategic partnerships and its emphasis on AI-driven solutions position it as a key player in the rapidly evolving landscape of genetic and cellular therapeutics. Dyno Therapeutics is headquartered in Watertown, Massachusetts, and is dedicated to transforming patient health through innovative gene delivery technologies. For more information about the company and its upcoming presentations at ASGCT, visit their website at www.dynotx.com.