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Rett In The News

Taysha Gene Therapies
August 24, 2023
Taysha announced the FDA has granted Fast Track Designation (FTD) to TSHA-102 for Rett. FTD is designed to help treatments reach patients faster by expediting the review of therapies with potential to address unmet medical needs for a serious condition. Benefits of FTD to programs include early ...
Taysha Gene Therapies
May 19, 2023
New preclinical data after neonatal administration in wild-type mice showed no detectable impact on survival, neurobehavioral functions and overall health, suggesting TSHA-102, engineered with novel miRARE technology, avoided toxic overexpression of MeCP2 within cells already expressing MeCP2
Emory University
March 6, 2023
What can heart rate tell us about Rett? A lot! In an RSRT-funded study, Emory researchers were able to associate Rett syndrome severity with variations in overnight heart rate. Using heart rate to understand symptom severity provides a new way to measure symptoms directly and objectively, and ...
UMass Chan Medical School
February 2, 2023
David Keener, a PhD candidate in the Morningside Graduate School of Biomedical Sciences Interdisciplinary Graduate Program, has received a Ruth L. Kirschstein National Research Service Award Individual Predoctoral Fellowship from the National Institute for Neurological Diseases and Stroke. The ...
Taysha Gene Therapies
January 31, 2023
Taysha provides an update on their lead program as well as their Rett syndrome program, TSHA-102
Business Wire
January 26, 2023
Herophilus announced today that in vivo studies underway of its lead candidate HRP-12975 are funded in part by the Rett Syndrome Research Trust (RSRT). HRP-12975 is the first small molecule therapy for Rett Syndrome with the potential to reverse the root cause of the disease, MECP2 deficiency.
November 18, 2022
Taysha's gene replacement trial for Rett, TSHA-102, is now recruiting. The first site is in Montreal, Canada. This program got its start through RSRT's Gene Therapy Consortium and MECP2 Consortium.
Endpoints News
September 28, 2022
Unravel Biosciences plans to take the first drug validated in their new platform, a liquid reformulation of vorinostat for Rett syndrome, into the clinic in a one-patient trial outside of the US by the end of this year.
Genetic Engineering & Biotechnology News
June 20, 2022
Researchers from UMASS working on Rett have created a more compact genome editor that can be more easily delivered into cells.
Neurogene Inc.
May 18, 2022
Neurogene Inc., a company founded on the vision to push the boundaries of genetic medicine to address complex and devastating neurological diseases, announced the addition of NGN-401 for Rett syndrome to the Company’s development pipeline.
Alcyone Therapeutics
May 18, 2022
Alcyone Therapeutics, a biotechnology company pioneering next-gen precision gene-based therapies for complex neurological conditions, today presents preclinical data showing the Company’s partnered novel AAV9 gene therapy vector ACTX-101 safely and effectively reactivates the inactive X ...
Taysha Gene Therapies
May 10, 2022
An update from Taysha on their Rett gene replacement program that got its start with RSRT funding.
May 5, 2022
As it stands there is no nationally or internationally defined registry for Rett syndrome. With the valuable data and collective knowledge that parents have related to their children with Rett Syndrome, it is critical to have a research-ready, clinical-trial-grade database that combines ...
Alcyone Therapeutics
May 2, 2022
Alcyone will be providing an update on its lead program. ACTX-101, which got its start through RSRT funding.
March 31, 2022
In a fourth-quarter corporate update, Taysha Gene Therapies announced that it will focus its efforts on clinical programs targeting Rett Syndrome and giant axonal neuropathy (GAN). Homing in on these programs will result in the pausing of other clinical research and development and a 35% ...
Taysha Gene Therapies
March 29, 2022
Taysha Gene Therapies announces that Health Canada has given clearance to begin a gene replacement trial for Rett Syndrome.