Taysha Gene Therapies has received FDA alignment on key elements of the TSHA-102 pivotal trial design for Rett syndrome, with the agency advising direct submission of trial protocol as an IND amendment, potentially expediting study initiation.
A comprehensive 2025 pipeline analysis identifies over 20 companies developing more than 20 therapeutic candidates for Rett syndrome, highlighting significant industry investment in this rare neurological disorder.
Peter Marks, head of FDA's Center for Biologics Evaluation and Research since 2016, was forced to resign after refusing to support HHS Secretary Robert F. Kennedy Jr.'s vaccine safety claims.
Neurogene has suspended enrollment in the high-dose arm of its NGN-401 gene therapy trial for Rett syndrome after a patient developed life-threatening systemic hyperinflammatory syndrome, a known risk with AAV vector therapies.
Two companies, Neurogene and Taysha Gene Therapies, are in Phase 1/2 clinical trials with gene therapies targeting the MECP2 gene for Rett syndrome.
A patient with Rett syndrome died after receiving a high dose of Neurogene's experimental gene therapy, NGN-401, triggering a safety review.
Taysha Gene Therapies reported positive updates on its TSHA-102 program for Rett syndrome, with high dose treatment showing good tolerability in trials.
Neurogene's stock plummeted after disclosing a serious adverse event in a patient receiving a high dose of NGN-401, its gene therapy for Rett syndrome.
The gene therapy market is experiencing substantial growth, fueled by technological advancements, increased investments, and a deeper understanding of genetic diseases.
Taysha Gene Therapies is evaluating TSHA-102, an AAV-based gene therapy, in Phase 1/2 REVEAL trials for both adolescent/adult and pediatric patients with Rett syndrome.