Dyno Therapeutics, Inc., a techbio company pioneering applications of artificial intelligence to engineer AAV capsids that can expand the potential of genetic medicine, announced the launch of its Dyno eCap 1™ capsid product, a leading eye AAV gene delivery vector with best-in-class potential, in a keynote address at the company’s Scientific Symposium at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT). The Dyno eCap 1 vector provides significantly improved delivery to the eye compared to other externally engineered capsids, including transduction throughout multiple layers of the retina.
“Enabling effective delivery to all cells in the eye, and in particular across the entire retina, is crucial to enable gene therapies to treat ocular diseases such as wet age-related macular degeneration (wAMD), geographic atrophy, and retinitis pigmentosa,” said Adrian Veres, M.D., Ph.D., Chief Scientific Officer and Co-founder of Dyno. “The Dyno eCap 1 vector is accelerating the next generation of ocular gene therapies for both genetic and non-genetic diseases, joining our growing suite of leading-edge capsids ready to help our partners bring the world’s best gene therapies into the clinic to help more patients.”
Dyno creates highly optimized delivery vectors by deploying a pioneering machine-guided approach. This approach combines state-of-the-art methods in machine learning and generative artificial intelligence (AI) for protein sequence design, in combination with large, internally collected in vivo datasets that provide high-resolution insights into numerous relevant capsid delivery properties. To engineer its state-of-the-art capsids for ocular gene delivery, Dyno conducted robust measurement of transduction as well as spatial localization across hundreds of thousands of capsid sequences, mapping the multi-modal properties of the ocular capsid sequence space from which the Dyno eCap 1 vector could be identified. This field-leading capsid displays consistent high performance for retinal transduction across retinal layers and cell types when delivered via intravitreal injection, a common non-surgical route of administration.
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Key Data on Dyno eCap 1 Technology
- The Dyno eCap 1 vector exhibits 80-fold better transduction to the retina compared to the commonly used AAV2 capsid.
- The Dyno eCap 1 vector transduces the retina better than a cohort of external engineered IVT capsids, supporting a field-leading profile.
- The Dyno eCap 1 vector consistently transduces cell types broadly across retinal layers, including rod cel, bipolar cells and retinal ganglion cells.
- The Dyno eCap 1 vector was validated in cynomolgus monkey (Macaca fascicularis), the most relevant NHP model for predicting translatability to human clinical trials. Improved performance is highly consistent across bulk RNA-seq, single-cell RNA-seq and histology.
“At Dyno, we are dedicated to realizing the full potential of gene therapy by helping our partners solve the challenge of delivering genes to every cell, every organ and every patient,” said Dyno Chief Executive Officer and Co-founder Eric Kelsic, Ph.D. “The trailblazing performance of our AI-designed capsids, as demonstrated by Dyno eCap 1 technology, continues to draw leading gene therapy developers to Dyno, so they can succeed in bringing transformative gene therapies to patients who await better treatments.”
SOURCE: BusinessWire
