AAV Vector Design Service for Age related Macular Degeneration

Introduction

Age-related macular degeneration (AMD) is a progressive retinal disease and top cause of irreversible blindness. At Creative Biolabs, we aim to replace burdensome frequent injections with our custom rAAV vector service for AMD. Leveraging advanced recombinant DNA and protein engineering, we provide single-administration vectors enabling sustained protein expression, delivering durable efficacy to transform wet and dry AMD treatment.

Age Related Macular Degeneration (AMD)

Fig.1 Schematic diagram of healthy eyes with retinal, choroidal and retinal pigment epithelial (RPE) cells, as well as the impact of AMD.¹

Pathogenesis

Dry AMD (Atrophic AMD)

As the predominant form of AMD (accounting for 85%-90% of cases), its core pathological mechanism is closely linked to chronic oxidative stress and inflammatory responses. Metabolic waste products (known as drusen) gradually accumulate beneath the retina, triggering the progressive atrophy and functional degeneration of retinal pigment epithelial (RPE) cells and photoreceptors (key cells responsible for light sensing). This ultimately leads to the gradual decline of central vision.

Wet AMD (Neovascular AMD)

Although it accounts for only 10%-15% of AMD cases, it poses a much higher risk of blindness. Its pathological hallmark is choroidal neovascularization: after oxidative stress and inflammation disrupt the blood-retinal barrier, abnormal new blood vessels grow beneath the retina. These fragile vessels are prone to leaking fluid or blood, causing macular edema and structural damage, which results in rapid and severe vision loss.

Ttherapeutic Approaches

Treatments for Dry AMD

• Basic interventions: Antioxidant supplementation (e.g., vitamin C/E, lutein, zeaxanthin) plus lifestyle adjustments (blood pressure/glucose control, avoiding strong light, quitting smoking).
• Emerging directions: AAV gene therapy (delivers complement system-regulating genes or uses gene-editing for neuroprotection) and retinal cell transplantation (under clinical exploration).

Treatments for Wet AMD

• Core goal: Inhibit neovascularization and reduce leakage.

Traditional therapy: Regular intravitreal anti-VEGF injections (e.g., ranibizumab, aflibercept; monthly/bi-monthly) with/without photodynamic therapy. Frequent injections may lower compliance.

Breakthrough therapy: AAV gene therapy (single administration) delivers anti-VEGF-encoding genes to enable continuous intraocular protein expression. Leverages the eye's immune privilege and AAV's low immunogenicity/long-term expression.

Workflow

Required Starting Materials: To initiate a project, clients typically provide a target gene sequence (e.g., an anti-VEGF construct, a neuroprotective factor, or a complement inhibitor), the desired expression profile (e.g., constitutive or inducible expression), and information on the target cell type (e.g., retinal pigment epithelial cells or photoreceptors).

Key Steps Involved:

1. Initial Consultation & Design: Discuss therapeutic goals, then develop customized vector strategies—including serotype selection and promoter optimization for precise ocular targeting and appropriate expression.
2. Vector Construction & Engineering: Synthesize therapeutic genes and expression cassettes; use capsid engineering/rational design to boost tropism and reduce immune responses (backed by research).
3. Vector Production & Purification: Produce high-titer, high-purity rAAV with low empty capsids via advanced, scalable manufacturing (consistent for preclinical/clinical use).
4. Characterization & QC: Conduct rigorous testing (titer, sterility, identity) plus in vitro/in vivo studies to confirm transduction efficiency and sustained transgene expression.
5. Final Deliverables: Provide fully characterized, lead-ready rAAV, comprehensive process/QC reports, and stability analysis for downstream research.
6. Estimated Timeframe: The typical timeframe for this service ranges from 8 to 16 weeks, depending on the complexity of the vector design, the scope of optimization, and the specific QC requirements.

What We Can Offer

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Customer Reviews

"Using Creative Biolabs' AAV design service in our preclinical research has significantly improved the transduction efficiency of our therapeutic gene. The quality of the final vector was exceptional, and it directly translated to robust, long-term expression in our animal models."

"We were struggling with a high immune response in our initial gene therapy candidates. Creative Biolabs provided a custom-engineered AAV that successfully evaded neutralizing antibodies, allowing us to proceed with our study. Their team's knowledge of vector immunology was invaluable."

"The detailed characterization report from Creative Biolabs gave us the confidence to move forward. The data on vector purity and stability was crucial for our regulatory submission, and their support team was always available to answer our questions."

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FAQs

At Creative Biolabs, we pioneer Age-Related Macular Degeneration gene therapy. Leveraging AAV vector engineering expertise and a client-centric approach, we offer end-to-end services from custom vector design to production and characterization, supporting the development of durable, single-administration therapies that preserve vision.

Contact Our Team for More Information and to Discuss Your Project

Reference

1. Rastoin, Olivia, Gilles Pagès, and Maeva Dufies. "Experimental models in neovascular age related macular degeneration." International journal of molecular sciences 21.13 (2020): 4627. https://doi.org/10.3390/ijms21134627. Distributed under Open Access license CC BY 4.0, without modification.