Encoded harnesses nature’s solutions to gene regulation to design precision therapies
Nature as a Source of Medical Discovery
The human genome is a masterful regulator of gene expression, providing a rich resource for medical discovery. Gene regulation is achieved through several mechanisms, including chromatin structure, transcriptional regulation, and RNA processing.
At Encoded, we have characterized millions of human DNA sequences, known as regulatory elements, that control gene expression through these pathways.
A Genomics-Powered Product Engine
The Encoded platform identifies and optimizes regulatory elements within the human genome to develop gene therapies with unprecedented selectivity and potency.
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.
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 superior efficacy and improved safety.
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.
The Encoded platform is advancing genomics-driven discovery, 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.