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Dyno Therapeutics Launches Three Breakthrough Capsid Delivery Vectors for Next-Generation Eye, Muscle and CNS Gene Therapies at the 2025 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

via Business Wire
  • Dyno’s AI-designed capsids improved therapeutic gene delivery to eye, muscle and CNS compared to commonly used AAV capsids, showcasing improved delivery efficiency, target-organ specificity and manufacturability
  • Data showing effective delivery in non-human primates demonstrates high potential for advanced gene therapies incorporating the Dyno capsids to benefit patients with ophthalmological, neuromuscular and neurological disorders
  • The new Dyno capsids are available for licensing to partners developing optimized gene therapies

Dyno Therapeutics, Inc., a genetic technologies company applying artificial intelligence (AI) to solve the grand challenge of in vivo gene delivery, today announced the launch of three new adeno-associated virus (AAV) capsid gene delivery vectors targeting the eye, musculoskeletal system and central nervous systems (CNS), each with best-in-class potential. Key data from the capsids were presented in a Scientific Symposium at the 28th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting and in three oral abstract presentations.

“I’m excited for Dyno to expand our offerings for our gene delivery partners with these new optimized capsids that more efficiently and more specifically deliver therapeutic genes to the eye, muscle and brain,” said Eric Kelsic, Ph.D., CEO and Cofounder of Dyno. “These new capsids advance the frontiers of gene delivery, bringing Dyno’s partners and the field one key step closer to realizing the full potential of genetic medicine to transform patient lives.”

Ineffective delivery of therapeutic genes to specific organs and cells in vivo is the most limiting challenge for gene therapy today. Dyno’s platform applies advanced AI models trained on data from high-throughput in vivo measurements in animal models to design AAV capsids that are optimized for delivery efficiency, precision targeting, and manufacturability. By sharing these improved gene delivery technologies with partners, Dyno aims to improve the effectiveness of genetic medicines, reducing delivery costs and accelerating the development of optimized therapies that address a wide range of human health challenges via genetic perturbations that address the root cause of each disorder.

Each Dyno capsid was optimized and validated for in vivo delivery to cynomolgus monkey (Macaca fascicularis), the most relevant non-human primate (NHP) model for predicting translation potential for human therapy. Dyno’s presentations highlighted the optimized performance, translatability, and manufacturability of the Dyno-4z2 capsid for eye delivery, Dyno-3hv capsid for neuromuscular delivery, and Dyno-ahq capsid for CNS delivery:

Dyno-4z2 for Eye Delivery

The Dyno-4z2 capsid is optimized for delivery to the retina upon intravitreal (IVT) injection and shows exceptional efficiency transducing bipolar cells in the inner nuclear layer (INL), supporting its use in optogenetic ocular gene therapies to restore vision for patients with retinal degeneration:

  • Greater than 40-fold improvement in broad retinal transduction compared to AAV2 at doses of 1e10-1e11 vg/eye in NHP.
  • A 5-fold improvement in transduction of bipolar cells and across the INL in NHP compared to a reference externally engineered capsid, an improvement also observed when both capsids were tested in mice.
  • Easy to administer via IVT injection, enabling broader patient access compared to more invasive subretinal injections.
  • Manufacturable at large scales and compatible with AAV2-based processes.

Dyno-3hv for Neuromuscular Delivery

Dyno-3hv is the first AAV capsid to demonstrate highly-efficient and enhanced delivery to the multiple tissues affected by neuromuscular disorders, enabling high efficient delivery to heart, skeletal muscle and brain with a single drug product:

  • Transduces 53-90% of skeletal myofibers and multiple regions of the heart and CNS with IV injection at a dose of 4e12 vg/kg in NHPs.
  • Potential for safer IV dosing through liver detargeting, with <10 vg/dg observed in liver in NHP after IV dosing at 4e12vg/kg and low biodistribution to other off-target organs.
  • Easy to administer via IV injection, enabling broader patient access compared to more invasive intracranial and intramuscular injections.
  • Identification of a novel receptor for crossing the blood-brain-barrier (BBB) and demonstration of conserved Dyno-3hv binding to both NHP and human orthologues supports translation for human therapies.
  • Easily manufactured at large scale and compatible with AAV9-based processes.

Dyno-ahq for CNS Delivery

Dyno-ahq is optimized for widespread and efficient delivery to the brain and throughout the CNS upon IV injection. With enhanced CNS delivery efficiencies compared to prior Dyno capsids, exceptional increases in liver detargeting, and a known mechanism for crossing the BBB that is conserved between NHPs and humans, Dyno-ahq has exceptional utility for delivering gene therapies addressing neurological diseases:

  • Reaches up to 30% of neurons in NHP after crossing the BBB at a dose of 3e13 vg/kg, a 280-fold increase in efficiency compared to the AAV9.
  • Liver detargeting in NHP by greater than 50-fold compared to AAV9, with more neurons being transduced in the brain than hepatocytes in the liver.
  • Easy to administer via IV injection, enabling broader CNS transduction and broader patient access compared to more invasive intraparenchymal and intracisternal injections.
  • Identification of a novel receptor for crossing the BBB and demonstration of conserved Dyno-ahq binding to both NHP and human orthologues supports translation for human therapies.
  • Easily manufactured at large scale and compatible with AAV9-based processes.

In addition to unveiling the new capsids, the Dyno Scientific Symposium, “Leveling up genetic medicine with frontier AI and AAV vectors for CNS, eye, and muscle,” provided updates on Dyno’s frontier AI algorithms and strategy to rapidly advance next-generation gene therapies incorporating these capsids into human clinical trials by expanding partnerships with gene therapy developers, towards approval of new treatments in areas of high unmet patient need.

Licensing Dyno Capsid Technology

Alongside the previously released Dyno-86m (also known as Dyno eCap™ 1) optimized for eye delivery, and Dyno-hc9 (also known as Dyno bCap™ 1) capsid optimized for brain delivery, the Dyno-4z2, Dyno-3hv and Dyno-ahq capsids and additional proprietary platform technologies are available for licensing to Dyno’s gene therapy partners.

“By solving the grand challenge of in vivo delivery with optimized capsids, our goal is to enable Dyno’s partners to transform patient lives with breakthrough genetic medicines,” says Kelsic. “With breakthrough new capsids like Dyno-4z2, Dyno-3hv, and Dyno-ahq, we are advancing the frontiers of delivery across therapeutic areas. These innovations reflect the quality and depth of our in vivo data, the power of our AI algorithms for sequence design, and our commitment to helping partners develop exceptional gene therapies that are safe, highly-effective and accessible to patients worldwide.”

About Dyno Therapeutics

Dyno Therapeutics’ mission is to build high-performance genetic technologies that transform patient lives. Dyno is creating better technologies for gene delivery and sequence design to increase “Genetic Agency”—the capacity for patients to take action and improve their health at a genetic level—enabling individuals to live the life they choose through safe, effective and widely accessible genetic treatments. With frontier AI models and high-throughput in vivo experimentation, Dyno designs optimized AAV delivery vectors that solve gene delivery challenges across a wide range of therapeutic applications. Dyno partners across industries to ensure these life-transforming technologies can help as many patients as possible, including strategic collaborations with leading gene therapy developers Astellas, Roche and Sarepta, and with technology companies including NVIDIA. Visit www.dynotx.com for more information.

Forward-Looking Statements: The capsids described are investigational and intended for future clinical development. References to delivery performance or therapeutic potential are based on preclinical data and do not imply regulatory approval or clinical validation. Licensing is subject to agreement terms.

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