Dyno Therapeutics to Present Cutting-Edge AAV Gene Delivery Data for CNS, Eye, and Muscle at 2025 ASGCT Annual Meeting
Dyno Therapeutics, Inc., a pioneer in genetic technologies harnessing the power of artificial intelligence (AI) to revolutionize in vivo gene delivery, announced today its upcoming participation at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), to be held from May 13 to 17, 2025, at the Ernest N. Morial Convention Center in New Orleans, Louisiana.
At this high-profile scientific gathering, Dyno will unveil a comprehensive dataset demonstrating the design and functional performance of novel adeno-associated virus (AAV) capsids specifically optimized for gene delivery to three vital organ systems: the central nervous system (CNS), the eye, and the neuromuscular system. The presentations comprising a dedicated Scientific Symposium and three oral presentations underscore Dyno’s leadership in redefining the capabilities of AAV-based gene therapy platforms using frontier AI tools.
Transforming Gene Therapy Delivery Through AI
Gene therapy holds extraordinary promise for treating a wide range of inherited and acquired diseases, from neurodegenerative disorders to muscular dystrophies and ocular diseases. However, a critical challenge persists: delivering therapeutic genetic material precisely and safely to the correct cells and tissues within the human body.
Dyno Therapeutics is tackling this bottleneck head-on. By applying advanced AI-driven design processes and high-throughput in vivo experimentation, Dyno engineers custom AAV capsids that overcome the limitations of naturally occurring viral vectors. These engineered capsids offer dramatically improved targeting specificity, enhanced efficiency of delivery, and scalable manufacturability—three essential pillars for the successful clinical deployment of gene therapies.
“Our mission is to unlock the full potential of gene therapy by solving the in vivo delivery problem,” said Eric Kelsic, Ph.D., cofounder and Chief Executive Officer of Dyno Therapeutics. “Thanks to our AI-powered platform, we’re able to design capsids that outperform natural variants and first-generation engineered vectors, opening the door to transformative treatments for patients in need.”
A Spotlight on Innovation at ASGCT 2025
At the ASGCT meeting, Dr. Kelsic will lead a Scientific Symposium that offers a deep dive into Dyno’s latest discoveries. Among the highlights will be three next-generation AAV capsids engineered for enhanced delivery to:
- The Central Nervous System (CNS): Effective gene delivery across the blood-brain barrier remains a formidable challenge. Dyno’s new capsids show significantly improved CNS transduction, offering potential breakthroughs in treating neurodegenerative diseases such as Parkinson’s, Huntington’s, and ALS.
- The Eye: Dyno’s ocular-targeting capsids demonstrate robust delivery across key structures of the eye, with implications for treating inherited retinal diseases and age-related macular degeneration. Notably, the designs enable lower doses, which may reduce immune responses and improve safety.
- The Neuromuscular System: For disorders such as Duchenne muscular dystrophy and spinal muscular atrophy, muscle tissue targeting is crucial. Dyno’s new capsids exhibit high specificity and expression in skeletal and cardiac muscle while minimizing off-target effects.
These advances are made possible through Dyno’s CapsidMap™ platform, a proprietary AI-guided system that integrates massive datasets of in vivo functional readouts with machine learning models. This platform enables iterative design cycles that rapidly converge on optimal capsid sequences tailored for specific therapeutic goals.
AI at the Heart of Capsid Discovery
Traditional approaches to capsid development often rely on directed evolution and random mutagenesis, methods that can be time-consuming, resource-intensive, and limited in scope. In contrast, Dyno’s use of AI accelerates the pace of innovation and expands the design space dramatically.
The AI models used by Dyno are trained on rich biological datasets collected through high-throughput in vivo screening experiments, where thousands of capsid variants are tested in animal models for tissue tropism, transduction efficiency, immune evasion, and other properties. Machine learning algorithms then analyze these datasets to predict novel capsid sequences with superior performance metrics.
“Frontier AI is a force multiplier in our discovery process,” said Kelsic. “By continuously learning from experimental results, our models evolve to become better predictors of real-world outcomes, guiding us toward the most promising capsid candidates with unmatched speed and accuracy.”
A Path Toward the Clinic
In addition to presenting breakthrough data, Dr. Kelsic will share Dyno’s strategic roadmap for translating these scientific advances into clinical impact. The company is rapidly moving to validate its capsids in preclinical disease models and is actively engaging with partners to co-develop gene therapies that can enter the clinic within the next few years.
Dyno operates a partnership-driven model that extends its capsid design capabilities to leading gene therapy developers worldwide. By integrating its capsids into partner programs, Dyno accelerates the development of therapies across multiple disease areas while expanding access to cutting-edge delivery tools.
“Our goal is to make next-generation gene therapies a reality for as many patients as possible,” said Kelsic. “Through collaborations and partnerships, we can scale the impact of our technology far beyond what any single company could achieve alone.”
Expanding Access to Transformative Medicines
A key priority for Dyno is to address the inequities in access to advanced genetic medicines. Today, many gene therapies are prohibitively expensive, partly due to the inefficiencies of existing delivery systems. By designing capsids that enable lower dosing, broader tissue targeting, and more efficient manufacturing, Dyno aims to lower the barriers to gene therapy development and distribution.
“We believe that better capsids lead to better therapies not just scientifically, but economically and logistically,” Kelsic noted. “When delivery becomes more efficient, therapies become more accessible.”
As the field of gene and cell therapy continues to mature, innovation in delivery technology will remain a defining factor in therapeutic success. Dyno Therapeutics is positioned at the forefront of this evolution, combining AI, synthetic biology, and deep domain expertise to create a new generation of AAV vectors that redefine what’s possible in gene therapy.
With a compelling slate of presentations at ASGCT 2025, the company is set to showcase not only its scientific achievements but also its vision for the future—one in which gene therapy is safer, more effective, and accessible to patients worldwide.
For more information about Dyno Therapeutics, its participation in ASGCT 2025, or its collaboration opportunities, please visit www.dynotx.com.
