Gene Therapy at Codexis

Gene Therapy

Delivering superior enzymes systemically

Disorders arising from genetic loss of function affect hundreds of thousands of people worldwide. Many of these conditions are debilitating or lethal, and very few have any treatment options. Delivering therapeutic proteins by recombinant enzyme replacement or gene therapy has shown promise for some of these disorders, but issues related to safety, lack of efficacy, and very high dosing have limited the success of many programs. At Codexis, we use our proprietary CodeEvolver® technology platform to engineer proteins that may offer improved targeting, expression, and potency when administered as gene therapies, offering potentially improved therapeutic benefit as compared to current options.

Gene therapy programs

In 2020, we entered a strategic collaboration and licensing agreement with Takeda Pharmaceuticals to research and develop engineered proteins to deliver as transgenes via gene therapy. As part of this collaboration, we have engineered protein variants for three lysosomal storage disorders, including fabry disease and pompe disease, and a blood factor disorder, for further development by Takeda as gene therapies. For each program, we identified protein variants with superior expression, stability, and potency, with similar or superior preclinical immunogenicity risk profiles as compared to the respective wild-type proteins. Additionally, we have several wholly-owned early research programs aimed at delivering superior enzymes for potentially treating lysosomal storage diseases by AAV gene therapy.

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New capsids for enhanced transgene delivery

In addition to our therapeutic protein engineering programs in gene therapy, we are also exploring applications of our CodeEvolver® technology platform toward engineering AAV capsids with enhanced properties such as more efficient tissue targeting or de-targeting, and neutralizing antibody avoidance.

Scientific Highlights

In vitro modeling of GM1 Gangliosidosis using iPSC-derived cellular and organoid CNS models

Christopher Schmitt, Sr. Scientist II at Codexis, presented a poster at the 2023 WORLDSymposium™ highlighting work being done in the GM1 Gangliosidosis gene therapy program. The objective of the work presented was to develop a suite of cell, spheroid, and organoid-based central nervous system (CNS) models for use in drug development, including enzyme replacement and gene therapy modalities.

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