Taysha Gene Therapies has announced that pharmaceutical company Astellas Gene Therapies, a division of Astellas Pharma, has made a strategic investment to enhance development of Taysha’s two lead programs: one for Rett syndrome and one for giant axonal neuropathy (GAN).
Astellas paid $50 million to acquire 15% of Taysha, and the agreement gives Astellas the option to license Taysha’s Rett and GAN programs after there is data from adult and/or pediatric clinical trials. Both programs remain under Taysha’s control for now.
Taysha’s Rett program is an MECP2 gene replacement program called TSHA-102. This gene replacement program is designed to deliver a shortened version of the MECP2 gene (referred to as a “mini-gene”) and a novel platform technology called miRARE, which should control the level of MECP2 expression. The mini-gene was developed by Sir Professor Adrian Bird and funded by RSRT as part of the MECP2 Consortium. The miRARE platform was developed by Dr. Steve Gray and funded by RSRT as part of our Gene Therapy Consortium. Taysha recently announced they have received regulatory approval from Health Canada to begin a clinical trial. This first trial will be in individuals with Rett 18 years of age and older.
You can find more information about this announcement through the following resources: