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TECHNOLOGY PLATFORM

Frontera adopts industrial leading insect cell culture technique to produce AAV. Through process development and optimization, a high yield, high purity and scalable manufacturing platform is established.

 

  • Vectorology – vector design and engineering

       To target each specific indication, Frontera designs AAV gene therapy vectors accordingly using various approaches including gene replacement/ Veritas BE Agents/augmentation, gene silencing, gene editing, antibody expression, or a combination of multiple approaches. Frontera’s vector biology research team optimizes various elements of the recombinant AAV vector genome in order to increase expression, enhance activity, and/or reduce immunogenicity. Through gene expression cassette optimization, we may achieve high expression of transgene which could potentially enhance gene therapy efficacy and/or reduce vector dose which in turn improve the safety of our gene therapy products. 

 

  • Capsid selection – screen, engineering and discovery

       The AAV capsid is the primary interface between the virus containing the payload and the target cells. The effectiveness of AAV is largely determined by the molecular interactions between the capsid and target cell surface receptors and subsequent downstream events following particle internalization. AAV capsid structures can be engineered to efficiently target certain cell types or even specific cell organelles such as mitochondria.

At Frontera, we design and select the optimal capsids based on tissue specificity, transduction efficiency, safety, and manufacturing scalability to ensure successful delivery of the genes of interest and minimize potential adverse responses.

 
  • Assay and disease model development

       In vitro cell-based models and animal models are critical model systems for us to screen our vector designs and evaluate efficacy and safety of our gene therapies. At Frontera we use several state-of-the-art technologies including genetic engineering, stem cell differentiation, and organoid culture to establish in vitro disease models such as primary cells from human patients, organoids, iPSC-derived disease relevant cells, and genetic engineered cell lines.

       We employ in vivo models that mimic the human disease pathogenesis and phenotype to study the effects of our AAV-based GT. Through using these in vitro and in vivo studies we can select candidates to proceed to clinical-stage development.

 

  • AAV production process

       The ability to produce and manufacture high-quality AAV vectors at low cost is a critical success factor in AAV gene therapy. Frontera team consists of many pioneers and experts in AAV gene therapy, and together we are building our production platform that can achieve high yields, high purity and scalability.

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High Yield

Through cell line development and process development, our production platform reaches the high level of production yield among those reported in the AAV industry. The high yield leads to low manufacturing cost, and competitive drug pricing to benefit patients and payors across the world. 

高产率

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High Purity

Our production platform produces high percentage of full AAV vectors containing the therapeutic DNA, and thus reduces the generation of empty capsids. In addition, our platform produces AAVs with low DNA impurities and low protein impurities. The high level of empty capsids, and/or the high impurity DNA or proteins, in an AAV drug are likely associated with reported Serious Adverse Event (SAE) in various AAV clinical trials. With the high AAV purity, we improve the drug safety profile.

高纯度

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Scalability

Our process is reproducible at different scales from 2L to 500L in single-use bioreactors. The lower levels of empty capsids produced in our upstream process enables all downstream purification steps to be scalable and removes bottlenecks (e.g. ultracentrifugation) present in other production systems. The good scalability shortens the development time from research to manufacturing, and removes uncertainty of the AAV productivity in the program planning and execution. 

高纯度

  • Manufacturing Platform

     

       Frontera built a state of art GMP manufacturing facility at Suzhou Biobay, China to produce clinical and future commercial recombinant AAV products. Our fully disposable manufacturing platform reduces contamination risk while improved operational efficiency. Our capability to produce AAV at vastly different scales can meet the needs of different therapeutic areas. Our ability to build robust platforms for AAV processes and analytics enables us to move from small scale to large scale GMP productions in shortest time.

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Time of issue:2021-12-29 15:36:18

Assay and disease model development
In vitro cell-based models and animal models are critical model systems for us to screen our vector designs and evaluate efficacy and safety of our gene therapies. At
Frontera we use several state-of-the-art technologies including genetic engineering, stem cell differentiation, and organoid culture to establish in vitro disease models such as primary cells from human patients, organoids, iPSC-derived disease relevant cells, and genetic engineered cell lines.
We employ in vivo models that mimic the human disease pathogenesis and phenotype to study the effects of our AAV-based GT. Through using these in vitro and in vivo studies we can select candidates to proceed to clinical-stage development.
 

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