Taysha Gene Therapies Rett Program Update
On November 13th, Taysha Gene Therapies reported an update on their Rett gene therapy program TSHA-102.
This was primarily a financial and corporate update, however, the company did provide information about the current status of the clinical trial.
Key takeaways:
- High dose TSHA-102 has been well tolerated with no Serious Adverse Events in two adolescent/adult patients and one paediatric patient as of the cut off date.
- The Independent Data Monitoring Committee has approved continued enrolment in cohort 2 (high dose) across both the adolescent/adult and children’s trial.
- The low dose part of the trial is now considered complete. Going forward, participants in both the adult/adolescents’ trial and the children’s trial will receive high dose TSHA-102.
- The UK clinical trial site at the Evelina Children’s Hospital is now scheduled to be activated in 2025.
Read Taysha’s full press release HERE
Read Taysha’s community letter HERE
Next steps:
Taysha plan to share clinical data from the high dose cohorts and an update on clinical data from the low dose cohorts in both adolescent/adults trial and children’s trial in the first half of 2025.
A total of 8 participants have now received TSHA-102 gene therapy to date.
This includes:
2 low dose adolescents/adults and 3 high dose adolescents/adults.
2 low dose children and 1 high dose child.
The children’s Taysha trial has an estimated enrolment of 20 children worldwide over 10 trial sites. This means that the number of children needed in the UK trial is currently very small.
As the number of patients for this and other Rett trials are likely to increase over time, we are calling for all UK patients to register on the Rett Registry UK so that we are able to effectively support doctors with recruitment.
Whether you want your loved one to participate in clinical trials or not, it is very important that we have an accurate measure of the number of UK patients to demonstrate the need for treatment to the relevant authorities as needed.
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 4 with Taysha Gene Therapies
‘An introduction to gene therapies for Rett Syndrome’ Dr Daniel Lumsden: LEAP Day Talks