Technology Platform


APEX Technology & Manufacturing Platform Highlights

Our APEX Technology & Manufacturing Platform is what makes Frontera a leader in gene therapy.

We focus on rapidly transitioning manufacturing processes across the product development lifecycle with our APEX Technology & Manufacturing Platform. Our manufacturing capabilities make it possible to efficiently advance new AAV therapeutics - turning research and early development into clinical programs, and then therapeutics that address patients in need.


Design and Engineering

To target each specific indication, Frontera designs AAV gene therapy vectors accordingly using various approaches including gene replacement, 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 have achieved 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

Screening, 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 Modeling

Engineering, Differentiation, and Culturing

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.

GMP Manufacturing

AAV Product Control and Flexibility

Our GMP facilities are designed to meet global clinical and commercial regulatory standards. Our leading-edge, in-house GMP manufacturing facility gives us full control of product supply and quality at all stages of AAV gene therapy development. Our in-house platform also provides the flexibility needed to support a wide range of preclinical, clinical and commercial needs. With facilities in the United States and GMP Manufacturing in China, our infrastructure is proven to increase product development speed, AAV yield, and product purity.