Hey GP2C and Rett U Community! Check out this super-encouraging news from our friends at RSRT!

Taysha Rett Program Updates March 2024

On March 19 Taysha Gene Therapies provided clinal updates on its gene therapy clinical trials for Rett syndrome. Here is a summary of the updates:

  1. Data from a 35-week assessment of the first adult patient in the REVEAL study showed that the TSHA-102 gene therapy low dose was well tolerated with sustained improvement across various efficacy measures.
  2. Data from a 19-week assessment of the second adult patient, also given a low dose, showed that the gene therapy was well-tolerated and indicated improvement in various efficacy measures.
  3. An Independent Data Monitoring Committee gave approval to proceed to the higher dose in the adolescent and adult study and to dose a second pediatric patient at the lower dose.
  4. Initial data from the first cohort (low dose) of the pediatric trial is expected in mid-2024. And initial data from the second cohort (high dose) in both pediatric and adolescent/adult trials is expected in the second half of the year.

The Principal Investigator… stated: “Both adult patients treated with TSHA-102 showed sustained and new improvements across key areas of disease impacting activities of daily living, including multiple aspects of autonomic function, social communication, motor skills, and experienced stabilized or significantly reduced seizures.”

READ MORE HERE: https://ir.tayshagtx.com/news-releases/news-release-details/taysha-gene-therapies-reports-full-year-2023-financial-results

Neurogene Announces Updates to Pediatric Gene Therapy Clinical Trial

Neurogene announced the following updates today to their pediatric gene therapy trial of NGN-401.

  1. Three patients have been dosed to date in the low dose cohort and NGN-401 has been well tolerated
  2. The size of the trial has been expanded from 5 patients to 16 with 8 patients expected to be enrolled in the low dose cohort and 8 patients in the high dose cohort
  3. Age range is 4 to 10 years old
  4. Patients will be recruited from the US and UK
  5. The remaining 5 patients in the low dose cohort can now be dosed concurrently with no waiting period between patients
  6. The first 3 patients in the high dose cohort will be dosed in a staggered manner with the first patient expected to be dosed in 2nd quarter of this year

READ MORE HERE: https://reverserett.org/site/assets/files/8847/our-letter-to-rett-syndrome-community-march-2024.pdf