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Recent Articles

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June 30, 2025
Neurogene Announces Trial Design for Embolden™ Study of NGN-401 Gene Therapy for Rett Syndrome
Ben Deverman, PhD | Senior Director of Vector Engineering at the Broad Institute of MIT and Harvard
April 30, 2025
Breaking Barriers: New Pathways Into the Brain
Ben Deverman, PhD | Senior Director of Vector Engineering at the Broad Institute of MIT and Harvard
April 18, 2025
How Will Cuts to FDA and NIH Impact Gene Therapy Clinical Trials and Rett Syndrome Research?
Ben Deverman, PhD | Senior Director of Vector Engineering at the Broad Institute of MIT and Harvard
April 15, 2025
Rewriting RNA: RSRT Funds Genetic Engineers in the AbuGoot Lab
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March 19, 2025
NYU Seizure Study Needs Your Videos
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February 11, 2025
3900 Trials and Counting: The Global Outlook on Genetic Medicines
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Press Releases

April 30, 2025
Apertura Gene Therapy and Rett Syndrome Research Trust Collaborate to Pioneer Advanced Genetic Medicines for Rett Syndrome Using TfR1-Targeted AAV Capsid
New York, NY and Trumbull, CT, April 30, 2025 – Apertura Gene Therapy, a biotechnology company focused on innovative gene therapy solutions, ...
March 13, 2025
RSRT Appoints Amy Gilliland to its Board of Trustees
Trumbull, CT  – Rett Syndrome Research Trust (RSRT), the organization working to cure Rett syndrome, is pleased to announce that Amy ...
June 5, 2024
RSRT Launches "Roadmap to Cures" to Advance Next-Generation Genetic Medicines into Clinical Trials
Trumbull, CT Rett Syndrome Research Trust (RSRT), the organization working to cure Rett syndrome, announced the launch of a bold new initiative: ...
April 10, 2024
RSRT Launches the MECP2 Editing Consortium With Goal to Advance Programs to Clinical Trials
Trumbull, CT - The Rett Syndrome Research Trust (RSRT) has taken a significant stride in the pursuit of innovative genetic medicines for Rett ...
March 28, 2024
PMD Solutions Supports RSRT's Biosensor VIBRANT Study for Rett Syndrome with Donation of FDA-cleared RespiraSense Devices and Analytics
Trumbull, CT - PMD Solutions, a leading provider of innovative healthcare technologies, is proud to announce its partnership with the Rett ...
January 31, 2024
Rett Syndrome Research Trust Awards a Record $10.3 Million in 2023 to Advance a Cure for Rett Syndrome
The Rett Syndrome Research Trust (RSRT) continues to spearhead groundbreaking research in its pursuit of a cure for Rett syndrome. RSRT has ...
January 16, 2024
The Rett Syndrome Research Trust Strengthens Research Team with Addition of Robert Deans as Chief Technology Officer
The Rett Syndrome Research Trust (RSRT) is excited to announce the addition of Robert Deans, PhD, as Chief Technology Officer, Head of Research. ...
November 9, 2023
Rett Syndrome Research Trust Launches the Rett Syndrome Global Registry: A Pioneering Parent-Reported Platform Designed to Expedite the Development of Genetic Medicines
The Rett Syndrome Research Trust (RSRT) is excited to announce the launch of the Rett Syndrome Global Registry, a fully-remote platform for ...
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Rett In The News

Innovative Genomics Institute
IGI is Awarded New Funding to Develop CRISPR Tools for Editing the Brain
January 6, 2025
A new project co-led by IGI founder Jennifer Doudna and Doudna lab researcher Kai Chen has been awarded $1.25 million from the Rett Syndrome Research Trust (RSRT) to develop CRISPR tools to edit the brain. The ultimate aim of this research is to create CRISPR-based therapies for Rett syndrome, ...
The Transmitter
What’s next for brain-directed gene therapy after death in Neurogene trial
November 26, 2024
An informative article on the recent and tragic death of a young child in a gene therapy trial for Rett syndrome.
Science Magazine
One of the Most Widely Read Science Journals Highlights Rett Syndrome
October 24, 2024
"Like other parents of children with Rett syndrome, Coenraads wishes scientists would hurry up and develop treatments. But as the founder and CEO of the Rett Syndrome Research Trust, she’s in a position to do something about it.”
Neurogene, Inc.
Neurogene Announces RMAT Designation for NGN-401 Investigational Gene Therapy for Rett Syndrome
August 7, 2024
Neurogene announced that NGN-401 received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA for the treatment of Rett syndrome. The designation was based on preliminary clinical evidence from the ongoing Phase 1/2 clinical trial with NGN-401 that shows potential to ...
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