Business:
Gene Therapy
Drug notes:
Undisclosed programs RD pediatric CNS disorders
About:
Encoded Therapeutics is creating next-generation molecular therapies that leverage the regulatory regions of DNA. Encoded’s name derives from the epigenetic code that influences when genes are expressed within a cell. Using genomics-driven screening and machine learning algorithms, Encoded is identifying and optimizing synthetic human regulatory elements to modulate the function of diseased cells by changing gene expression. Their current platform utilizes AAV vectors so that they can deliver the human regulatory elements specifically to disease-relevant cell types. Encoded’s precision gene therapies therefore have the potential to target specific cell types to address a variety of disease mechanisms. Currently, Encoded is focusing on pediatric central nervous system disorders.