Approach
Our proven approach bridges the gap between innovation and a potential cure.
Our proven approach bridges the gap between innovation and a potential cure.
With our gene therapy approach, we’re changing the way we speak about disease.
Gene therapies have proven to deliver transformational benefit to patients who suffer from devastating diseases. Our mission at Taysha is to build upon these advancements to eradicate monogenic CNS diseases for the thousands affected. Through our proven gene therapy strategy and our unrivaled partnership with UT Southwestern, we are creating an engine for new cures.
We are developing next-generation technologies to optimize key components of our AAV-based gene therapies, including redosing, transgene regulation and capsid development.
In a number of disorders, including Rett syndrome and FOXG1 syndrome, the expression of a therapeutic transgene needs to be regulated. High doses of the engineered gene may be harmful, while low doses may not be effective. For disorders that require replacement of dose-sensitive genes, we have combined high-throughput microRNA (miRNA), profiling and genome mining to create miRARE, our novel miRNA target panel. This approach is designed to enable our product candidates to maintain safe transgene expression levels in the brain.