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 bCap 1 capsid product, a breakthrough CNS-targeted AAV gene delivery vector with best-in-class potential, in a keynote address at the company’s Scientific Symposium at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting. The Dyno bCap 1 vector provides dramatically improved CNS delivery and liver detargeting compared to leading natural capsids and stronger all-around characteristics relative to other engineered CNS-IV capsids.
“Safe and effective gene delivery to the brain is a primary factor limiting the treatment of CNS diseases with gene therapy. We believe effective delivery to all cells throughout the brain will unlock the potential to treat patients affected by a variety of genetic diseases, including amyotrophic lateral sclerosis, Angelman syndrome, Parkinson’s disease and Alzheimer’s disease,” said Adrian Veres, M.D., Ph.D., CSO and Co-founder of Dyno. “We look forward to further exploring the transformative potential of Dyno bCap 1, as well as our growing line of capsid products, by partnering with leading developers of gene therapies.”
To create high-performing capsids, Dyno has pioneered the application of state-of-the-art methods in deep learning and generative artificial intelligence (AI) to protein sequence design, while also leveraging large, internally collected in vivo datasets that provide high-resolution insights into the many therapeutically relevant capsid delivery properties. By combining AI and high-throughput biology, Dyno’s platform is capable of more fully exploring the AAV capsid sequence space in search of capsids that are optimized across multiple dimensions, such as CNS targeting, liver detargeting, and production efficiency.
Key Data on Dyno bCap 1 Technology
- Relative to the commonly used AAV9 capsid, Dyno bCap1 exhibits 100-fold better pan-brain CNS transduction upon crossing of the blood-brain barrier and 10-fold better liver detargeting.
- Dyno bCap1 improvements in transduction and targeting specificity are conserved across NHP species in both cynomolgus monkey (Macaca fascicularis) and African green monkey (Chlorocebus sabaeus), increasing confidence that the breakthrough CNS delivery capabilities of Dyno bCap 1 could be relevant for applications in human therapeutics.
- Whereas naturally-derived capsids such as AAV9 transduce only a small fraction of brain cells in NHPs, with a low IV injected dose of 1e13vg/kg, payloads delivered by the Dyno bCap 1 capsid transduced between 4-14% of cells in the brain, and 5-20% of neurons across pan-brain regions and the spinal cord.
SOURCE: Businesswire