Our Science

Our science focuses on gene therapy programs with the potential to transform care for patients with central nervous system (CNS) diseases – starting with Rett syndrome.

Our approach aims to address unique challenges in gene therapy by bringing together advanced genetic medicine technologies with established tools.

What is Gene Therapy?

Monogenic diseases are caused by harmful changes – or mutations – to a single gene in an individual’s genetic code.

Gene therapy is intended to treat the root cause of these diseases by replacing, changing or inactivating a gene that is causing a disease. One strategy involves introducing a healthy version of a gene into the types of cells where a disease process occurs. Most gene therapy approaches rely on the use of modified or engineered viruses used as a delivery vehicle, or vector, to bring the new gene into a cell, where it is transcribed and translated to produce a functional protein.

The Taysha Strategy: De-risk Development

The fundamental components of our approach are based on recent success in gene therapy development and commercialization to enable rapid clinical and commercial development: a clinically and commercially proven adeno-associated virus serotype 9 (AAV9) capsid, intrathecal delivery and an efficient manufacturing process.

AAV9 Capsid

We use an AAV9 capsid to deliver therapeutic genes engineered to replace a mutated gene, enhance the expression of a silenced gene or decrease the expression of a gene. AAV9 is the most studied and well-characterized, with demonstrated clinical activity and tolerability across multiple CNS indications.

Intrathecal Delivery

We use intrathecal administration, which directly delivers our gene therapies to the cerebrospinal fluid to facilitate biodistribution and cell transduction within key regions of the CNS. The procedure is routinely performed in an outpatient setting, and compared to intravenous administration, it allows for a lower dose of the therapy.

Efficient Manufacturing

Our flexible manufacturing processes allow us to produce our gene therapy products efficiently at scale. We leverage a highly scalable and commercial-viable HEK293 triple transfection suspension process that strikes the right balance of favorable yield, rapid development and a harmonized platform process across our programs.