Sukumar Nagendran, MD, on Improving Rett Syndrome Symptoms in Adult Patients With Gene Therapy
The president and head of R&D at Taysha Gene Therapies discussed positive early results from the first 2 patients treated with gene therapy TSHA-102.
This is the second part of an interview with Sukumar Nagendran, MD. For the first part,
“The first patient that we dosed was 20 years of age and had severe Rett syndrome in stage 4, and most of us are very surprised of because of the clinical response that we saw. Most of us, including the experts said, ‘You're not going to see a clinical response, get the safety data and go and move into the pediatric population.’ But what we noticed in the first patient was within 7 to 10 days there was a positive response on motor function and developmental milestones.”
In mid-2023, Taysha Gene Therapies
In December 2023, about a month before Taysha announced
REFERENCES
1. Taysha Gene Therapies announces first patient dosed with TSHA-102 in the reveal phase 1/2 trial under investigation for the treatment of Rett syndrome. News release. Taysha Gene Therapies, Inc. June 5, 2023. Accessed January 12, 2024. https://ir.tayshagtx.com/news-releases/news-release-details/taysha-gene-therapies-announces-first-patient-dosed-tsha-102
2. Taysha Gene Therapies reports third quarter 2023 financial results and provides corporate and clinical updates. News release. Taysha Gene Therapies, Inc.. November 14, 2023. Accessed January 12, 2024. https://ir.tayshagtx.com/news-releases/news-release-details/taysha-gene-therapies-reports-third-quarter-2023-financial
3. Taysha Gene Therapies announces first pediatric patient dosed with TSHA-102 in REVEAL phase 1/2 pediatric trial in Rett syndrome. News release. Taysha Gene Therapies, Inc. January 10, 2024. Accessed January 12, 2024. https://ir.tayshagtx.com/news-releases/news-release-details/taysha-gene-therapies-announces-first-pediatric-patient-dosed
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