Astellas Licenses Dyno Therapeutics’ AI-Designed AAV Capsid, Advancing Gene Delivery for Muscle Disorders

Dyno Therapeutics announced that Astellas Pharma Inc. will license an engineered adeno-associated virus (AAV) capsid designed for skeletal muscle–targeted gene delivery. The agreement originated from a 2021 research collaboration¹ to develop next-generation AAV vectors for gene therapy directed to both skeletal and cardiac muscle using Dyno's CapsidMap™ platform.

CapsidMap represents a potentially transformative approach applying in vivo experimental data and machine learning to create novel AAV capsids—the cell-targeting protein shells of viral vectors—designed to optimize tissue targeting and immune-evading properties. The arrangement includes a $15 million upfront payment and potential downstream milestone and royalty components. Astellas will be responsible for subsequent preclinical, clinical, and commercialization activities.

AI-Designed Capsids Address Longstanding Delivery Challenges

Efficient delivery of gene therapies to skeletal muscle remains a technical limitation in the field. Wild-type AAV serotypes, including AAV9, generally require high systemic doses to achieve therapeutic levels of transduction in

muscle tissue, which may increase the risk of dose-dependent toxicities and

complicate manufacturing scalability.

The capsid licensed in this agreement was generated using machine learning models trained on large-scale in vivo datasets. These models are designed to predict sequence-function relationships and enable exploration of capsid sequence space beyond the scope of conventional approaches such as directed evolution or rational design. The objective of this strategy is to identify variants with improved tissue tropism and transduction efficiency.

Preclinical data reported by the company indicate that the engineered capsid demonstrates enhanced skeletal muscle targeting in nonhuman primates relative to benchmark AAV capsids. The construct is also reported to be compatible with existing AAV9-based production systems, which may facilitate translation to established manufacturing workflows.

Second Major Licensing Milestone for Dyno

This represents the second instance in which Dyno Therapeutics has licensed an AI-designed AAV capsid. A prior agreement involved a capsid intended for central nervous system applications, licensed by Roche in 2025.

The current agreement extends the application of AI-derived capsid engineering to muscle-targeted gene delivery. Together, these transactions suggest that machine learning guided approaches are being explored across multiple therapeutic contexts, including both neuromuscular and central nervous system indications.

This deal is a meaningful proof point for the broader field of AI-designed biologics, according to the company. The fact that Dyno has now had AI-engineered capsids licensed for both central nervous system and muscle applications signals that AI-designed gene delivery vectors are moving from theoretical promise to clinical application. Muscle-targeting AAVs are especially relevant to diseases such as Duchenne muscular dystrophy and other genetic muscle disorders, which have long lacked effective delivery tools.

Implications for Gene Therapy Development

Advances in capsid engineering are a central focus in improving the therapeutic index of in vivo gene therapies. Enhanced tissue specificity and transduction efficiency could enable lower vector doses, which may mitigate immune-mediated adverse effects and reduce vector-related toxicity.

As Astellas Pharma Inc. advances development of gene therapy candidates incorporating the licensed capsid, forthcoming studies may provide additional insight into the clinical utility and safety profile of AI-engineered delivery vectors.

The use of AI-based design frameworks introduces a data-driven approach to capsid optimization, potentially increasing the efficiency of candidate identification. However, further validation in clinical settings will be required to determine whether preclinical improvements in biodistribution and expression translate into meaningful therapeutic benefit.

References

1. Kelsic et al. “Virus (AAV) Capsids for Advanced Gene Therapy” Wyss Institute
   https://wyss.harvard.edu/technology/synthetic-aav-virus-capsids-for-advanced-gene-therapy/
2. “Astellas and Dyno Therapeutics Announce Research Collaboration to Develop Next-Generation AAV Gene Therapy Vectors for Skeletal and Cardiac Muscle” PR Newswire, December1, 2021.
   https://www.prnewswire.com/news-releases/astellas-and-dyno-therapeutics-announce-research-collaboration-to-develop-next-generation-aav-gene-therapy-vectors-for-skeletal-and-cardiac-muscle-301435696.html
3. Maddox, W. “Astellas unearths Dyno muscle disorder AAV capsid with $15M option agreement” Fierce Biotech Apr 8, 2026.
   https://www.fiercebiotech.com/biotech/astellas-pharma-and-dyno-therapeutics-unearth-15m-muscle-disorder-capsid-ongoing