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

• AAV Vector Modification and Screening

  We engineer AAV capsids from various serotypes for enhanced tissue and cell targeting, as well as higher transduction efficiency. Additionally, we optimize recombinant baculovirus vector design to produce highly potent, full AAV particles in Sf9 cells.

  Our integrated in vitro and in vivo screening platform develops novel AAV capsid variants with strong tissue tropism, high infection efficiency, and low immunogenicity.

• Gene Expression Cassette Optimization

  We optimize gene expression regulatory elements (such as enhancers, promoters, introns, and poly(A) signals) and modified codon sequences to achieve optimal protein expression level and desired functional activity in target cells.

  By lowering CpG motifs, we decrease immunogenicity, which can prolong gene expression and enhance the durability and efficacy of treatments.

Production Technology Platform

• 

  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.

• 

  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.

• 

  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.