Taysha announcement: Key takeaways


On 10 January 2024, Taysha Gene Therapies announced a press release sharing the news that the UK regulator, the MHRA has approved their Clinical Trial Application (CTA) for a UK clinical trial of Taysha’s product TSHA-102 in children with Rett syndrome. Please see below for the full press release, key takeaways and Taysha’s community letter.

Key takeaways:

The first child with Rett syndrome has been dosed with TSHA-102 in the USA.

The MHRA has authorised a CTA to expand the ongoing US children’s trial to the UK.

There are two parts to the study.

The first part will include patients age 5-8. Three patients will receive the low dose. The next three patients will receive a higher dose.

The second part of the study will expand to include up to 21 patients age 3-8, dose is yet to be confirmed.

Full press release here

Download here

Did you know?

Through our longstanding partnership with the US based RSRT, Reverse Rett has provided a significant level of funding for the MECP2 and Gene Therapy Consortia whose work led to the development of TSHA-102.  

The MECP2 and MECP2 Gene Therapy Consortia received over $2,220,000 from Reverse Rett over the last fourteen years which helped enable this work to move from the laboratory bench to human clinical trials

We are delighted for Sir Professor Adrian Bird and Dr Steve Gray and send them and all their colleagues at the University of Edinburgh, UT Southwestern (previously University of North Carolina, Chapel Hill) and Taysha Gene Therapies our deepest thanks and congratulations!

In the relentless pursuit of life-changing treatments for Rett syndrome, it’s essential that we continue to contribute to research projects focused on developing better, less risky, more effective treatments for everyone with Rett syndrome. You can help by starting a fundraiser today. Email [email protected] to find out how you can help.