Update from Taysha Gene Therapies
Taysha Gene Therapies have provided an update on their Rett syndrome clinical trials today along with financial results.
Top line news;
Interim safety data from the first 12+ patient who received the high dose level of TSHA-102 showed that the gene therapy was well-tolerated with no safety issues at 6 weeks after dosing.
After reviewing those data, the Independent Data Monitoring Committee (IDMC) approved dosing of;
2nd 12+ participant in the higher dose cohort
1st paediatric participant in higher dose cohort
These participants have been identified, enrolled and dosing is scheduled.
Taysha also announced today that the Canadian regulator, Health Canada has authorized the clinical trial application (CTA) for TSHA-102 in pediatric patients with Rett syndrome, enabling expansion of the ongoing children’s trial into Canada.
Read Taysha’s press release HERE
Read the community letter HERE
In January 2024, the UK regulator the MHRA authorised both Taysha Gene Therapies and Neurogene to initiate a UK paediatric clinical trial of TSHA-102 in children with Rett syndrome.
These clinical trials have not yet started. However, we do know that the UK clinical trial of TSHA-102 is going to start at the Evelina Children’s Hospital under Dr Dan Lumsden.
As soon as we have more information about either gene therapy clinical trial we will update our community.
We do need to remind families that the number of patients needed for these first Rett syndrome gene therapy trials will be veryb small and it is likely that the doctors involved will recruit patients local to them.
As the number of patients needed may increase, it is likely that doctors involved with these clinical trials will request patients from the Rett Registry UK
If you are not already signed up to the Registry, you can sign up here
You can also email us directly to flag up your interest in gene therapy trials by emailing [email protected]
You can learn more about gene therapy trials by watching the following videos on YouTube;
Reverse Rett Bitesize: Emerging gene therapies – Episode 2 with Rachael Stevenson
Reverse Rett Bitesize: Emerging Gene Therapies – Episode 3 with Dr Dan Lumsden
Reverse Rett Bitesize: Emerging Gene Therapies – Episode 4 with Taysha Gene Therapies
‘An introduction to gene therapies for Rett Syndrome’ Dr Daniel Lumsden: LEAP Day Talks