Our Science

The Encoded Platform

Our platform is advancing genomics-driven discovery and unlocking new opportunities for viral gene therapies. Using this novel platform, Encoded is developing precision therapies for a broad range of genetic disorders, including those not addressable with existing gene therapy approaches.

Unlocking Opportunities for Viral Gene Therapy

At Encoded, we are focused on moving viral gene therapy beyond the limitations that exist today. By incorporating human regulatory elements into therapeutic viral vectors, we can impart new functionality, such as superior cell-type selectivity and potency, and endogenous gene control.

Our initial programs use adeno-associated viral (AAV) vectors, a clinically-validated gene therapy modality, and offer the potential to address disorders outside the scope of existing gene therapy technology.

Cell-Type Selectivity

Current viral gene therapies are limited in their ability to selectively target disease-relevant cells, and widespread gene expression may lead to negative effects. The Encoded platform enables identification of cell-type selective regulatory elements for precise gene expression, ultimately resulting in potentially superior efficacy and improved safety.

 

Potency

Many genetic disorders require high levels of gene expression for a therapeutic effect. By utilizing regulatory elements that drive robust gene expression, the Encoded platform can dramatically increase the potency of viral gene therapy. This creates opportunities to treat disorders that require high levels of therapeutic gene expression at doses that are well-tolerated.

 

Endogenous Gene Control

Deciphering the genome’s regulatory code creates tremendous opportunity for precise control over endogenous gene expression. Endogenous gene control has far-reaching potential for disorders involving large genes, dominant negative gene species, epigenetically-silenced genes, or genes that produce multiple protein isoforms. The Encoded platform has demonstrated the ability to upregulate or downregulate endogenous genes using clinically-validated viral delivery systems.

 

Our diverse and impactful gene therapies aim to address devastating genetic and acquired disorders spanning multiple disease pathways, including: neurocircuitry, liver and metabolic disorders, neurodegeneration, and cardiovascular disease.