Are you ready to elevate your ophthalmic research with AAV gene therapy?
Cyagen’s AAV gene therapy ophthalmology
contract research organization (CRO) platform can be your partner in reshaping the landscape of ocular
genetic
interventions. With the unique combination of our highly-effective artificial intelligence (AI)-powered
AAV vector
design and production capabilities and our range of murine ocular models, our comprehensive CRO platform
aims to
help improve translational research for your ophthalmology studies.
Empowering Breakthroughs in Ophthalmology Gene Therapy with
Cyagen's AI-Powered AAV Discovery
At Cyagen, we are a pioneer in the field of AAV gene therapy and ophthalmic research. Our cutting-edge technology utilizes the power of large language AI models to revolutionize discovery of novel Adeno-Associated Virus (AAV) vector designs, reshaping the way we target and treat ocular disorders.
Compared to traditional directed evolution, our AI-powered directed evolution utilizes the most advanced large-language models enabling a much shorter AAV discovery cycle with higher efficiency and multi-dimensional prediction capabilities.
Figure 1. AAV Discovery Workflow.
(A) Workflow for traditional directed evolution.
(B) Workflow for AI-powered directed evolution demonstrates a shorter AAV discovery cycle.
Unleash the Power of Multi-Dimensional
AI-Guided AAV Vector Design
Dimension #1
Dimension #2
Superior Treatment
Efficacy
High-Titer AAV Production
Our AI model demonstrates exceptional prediction accuracy in AAV productivities and is able to produce novel AAV designs at high-titer yields, as validated by wet-lab experiments (Fig. 2), significantly lowering AAV manufacturing costs.
Figure 2. AI guides the design of novel AAVs with higher productivity.
(A) AAV productivity prediction by AI model. (B) AI-predicted viral production for AAV mutants
closely
matches experimental results.
Enhanced Ocular Transduction & Penetration
With exceptional prediction accuracy, our AI model successfully designed novel AAV vectors that outperform the clinically validated AAV2-7m8 in retinal penetration and whole eye transduction efficiency, providing better targeting AAVs for basic research and a platform for identifying more translatable gene therapy products for clinical use.
Figure 3. AI-designed AAVs demonstrate superior retinal penetration and whole eye transduction
efficiency.
(A) AAV transduction efficiency prediction by AI model. Left: AAV whole eye transduction; Right:
AAV rod
cell transduction. (B-C) Comparisons of whole eye transduction and ocular penetration between WT,
AAV2-7M8
and AI-designed novel AAVs (ROD8,12,13,16) by GFP expression at 21 days post intravitreal injection
(3E9
vg/eye). (D) Comparisons of whole eye transduction between WT, AAV2-7M8 and AI-designed novel AAVs
by
luciferase assay at 21 days post intravitreal injection (3E9 vg/eye).
Superior Treatment Efficacy
AAV-based gene therapy using our AI-designed novel AAV vector has shown remarkable efficacy in treating wet Age-Related Macular Degeneration (AMD) compared to both the WT AAV2 and to the FDA-approved Ranibizumab (Lucentis) in our humanized VEGF (hVEGF) mouse model.
Figure 4. AI-designed AAV exhibits remarkable efficacy in a hVEGF model.
(A) Comparison of fluorescein leakage by fluorescein angiography (FFA) between PBS, Lucentis, wild
type
AAV2-CAG-anti-VEGF and AAV2-Novel-CAG-anti-VEGF (AI-designed), at different days post intravitreal
injection
(1E9 vg/eye). (B) Quantification and statistics of recovery percentage post treatment.
Unveiling Novel AAVs with
Whole Eye and Rod Cell Transduction in NHPs
Our commitment to innovation does not stop at rodents. We have taken a step further
by
identifying novel
AAVs effective in non-human primate (NHP) research. With the first-round large-library screening and
the
second-round small library screening, we have identified candidates with superior whole eye and
rod cell
transduction compared to the NHP-validated AAV2-7m8, AAV2-GL-1 and AAV2-NN-2. This
breakthrough reaffirms our
dedication to pushing boundaries and achieving excellence in advancing AAV gene therapies for
ophthalmic
research.
Figure 5. AI-predicted AAVs exhibit high efficiency in whole eye and rod cell transduction in NHPs.
(A) AAV transduction efficiency prediction by AI model and validated by Next-Generation Sequencing
(NGS) in
macaques at 21 days post intravitreal injection (3E9 vg/eye). Upper: AAV whole eye transduction; Bottom:
AAV rod
cell transduction. (B) AI model Identified high-confidence novel AAV candidates with better whole-eye
and/or rod
cell transduction than AAV2-7m8, AAV2-GL-1 and AAV2-NN-2.
Our Milestones
At Cyagen, our journey towards excellence in advancing ophthalmic gene therapy
research
has been marked by extraordinary milestones, including groundbreaking collaborations with leading
pharmaceutical companies worldwide. Together with our esteemed partners, we are redefining the
landscape of
gene therapy using our multi-dimensional AI-powered AAV discovery platform. Our shared vision and
combined
expertise have not only accelerated the development of cutting-edge treatments, but have also
illuminated new
avenues of exploration in the realm of ocular health. As we continue to forge ahead, these pivotal
collaborations remain as pillars of inspiration, driving us to unlock further advancements and
deliver
unprecedented breakthroughs in ophthalmology gene therapy on a global scale.
Join the Movement
Cyagen invites companies and research institutions focusing on AAV gene therapies for ophthalmic research to join us in shaping the future medicine for patients with genetic ocular diseases. Our mission is to empower your research and therapeutic development with the unmatched potential of AI-powered AAV vector discovery platform, redefining the possibilities for ophthalmology.
Experience Excellence Today:
Ready to collaborate with the leaders in AI-powered AAV discovery for gene therapy? Contact us now to learn more about how our breakthrough CRO platform can accelerate your research journey towards revolutionary ocular treatments.
Accelerate Ophthalmic Gene Therapy with Cyagen:
Transforming Vision, Transforming Lives.
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