About rAAV

• Mechanism of Action
  Recombination adeno-associated virus (rAAV) vectors carry a target gene of interest (GOI) to transduce specific tissues and cells. This enables continuous expression of functional proteins, repair of damaged cells, restoration of tissue function, improvement of clinical symptoms, and reversal of disease progression.
• Clinical Value
  For hereditary diseases where no effective treatments currently exist, AAV gene therapies provide promising approaches with the potential to achieve lasting cures
  For chronic diseases requiring lifelong medication, AAV vectors provide a "single-dose, long-term benefit" regimen. This significantly reduces medical burdens on patients while offering strong pharmacoeconomic advantages.

R&D Technology Platform (EXACTE™)

• Capsid Engineering

  Engineer and screen optimized capsids for efficient delivery of therapeutic transgenes to target tissues, including the eye, liver, and heart.

  Design proprietary RepCap constructs tailored for the baculovirus/Sf9 production system, enabling the generation of highly potent, full AAV particles.

• Payload Engineering

  Optimize codon sequences, including codon adaptation index, cryptic splice sites, CpG motifs, repetitive elements, and unfavorable RNA structures, to improve transgene expression.

  Refine regulatory elements, including enhancers, promoters, introns, Kozak sequences, and poly(A) signals, to enhance transcriptional and translational efficiency.

  Engineer payload constructs to improve vector yield, purity, and consistency during manufacturing.

• Candidate Screening

  Develop comprehensive potency, activity, and functional assays to evaluate capsid performance and transgene expression.

  Establish and select appropriate in vitro and in vivo models for robust efficacy screening.

  Conduct multi-model toxicity evaluations to assess safety and de-risk lead candidate selection.

Production Technology Platform (AAVANCE™)

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  Industry Leading AAV Production Platform

  Large-Scale： Utilizing the Sf9 insect cell expression system, we have established GMP production capacities of 200L and 500L.

  High Yield： bioreactor yields consistently exceed 1E15 vg/L, reflecting an industry-leading level.

  High Potency： Our vector designs, optimized for Sf9 cells, yield highly active AAV viruses.

  Low Cost： Streamlined processes significantly reduce direct production costs, improving affordability for patients.

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  Innovative Purification Process

  We maintain empty capsid levels below 5%, greatly enhancing product safety and creating a strong competitive advantage.

  Downstream purification yields reach 50%, setting another industry benchmark.

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  Comprehensive Quality System

  We have established a full-spectrum AAV quality control and assurance system, ensuring robust and consistent product quality.

  Our platform meets regulatory standards across the United States, European Union, and China for gene therapy products.