We are pleased to share that initial data from the first adult patient with Rett Syndrome on the Taysha Gene Therapies clinical trial in Canada have been shared today in a press release and community letter from the company.

“For TSHA-102 in Rett Syndrome, we believe the initial safety profile and significant clinical improvements seen in the first adult patient with severe disease four weeks post-treatment reinforces the transformative potential of our gene therapy to address the root cause of Rett syndrome.”

Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. 

Read the full press release here 

Read the community letter here 

Top line information: 

  • There have been no serious adverse events related to TSHA-102 in the first patient dosed as of six weeks following administration.
  • The first patient dosed has shown improvements in a number of areas based on data collected from several assessments at 4 weeks post-administration. 
  • Taysha has received clearance from the US regulator, the FDA, for a clinical trial in children ages 5-8. 
  • Taysha has submitted a Clinical Trial Application to the UK regulator, the MHRA for a clinical trial in children. No information has been shared regarding age group at this time. 
  • Taysha has received clearance for the second adult patient to be dosed. This is projected to happen in Q3, 2023. 

At Reverse Rett, we are delighted by today’s news and proud of the contribution we have made to this milestone moment. From funding the work of Steve Gray Ph.D., and Professor Sir Adrian Bird whose lab research is implicit in the Taysha gene therapy product, to developing the Rett Registry UK, to providing UK clinical trial support including recruitment, screening and retention services, all of these pieces have helped to bring us to where we are today, awaiting CTA approval for a UK clinical trial of gene therapy for Rett.

None of this progress would have been possible without the dedication, commitment and support of everyone who has been behind the work of Reverse Rett over the last 13 years. We thank all of you from the bottom of our hearts. We look forward to being able to share more good news with you in the coming months.

In the meantime, we send our deep gratitude and all good wishes to patient #1, patient #2, their families and all the patients and families who have come forward to participate in screenings for this clinical trial. We will be thinking of you in the days, weeks and months to come and sending you all our love and thanks.

We look forward to the next update from Taysha Gene Therapies and send our love, gratitude and all best wishes to patients #1 and #2, their families, and all the patients and families coming forward for screenings for these vital clinical trials.