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:
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:
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:
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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