Rett In The News

UMASS Chan Medical School announces the $2.3 million award from RSRT to develop genetic medicines for Rett syndrome.

ProQR, a biotech company in The Netherlands, and RSRT have initiated a collaboration to develop RNA editiing therapeutics for Rett syndrome. RSRT has made an initial $1 million award to ProQR.

Key highlights from Neurogene's update include approval to dose patient #3; clearance to initiate a trial in the UK; plans to expand the US trial to more patients and to test a higher dose; expect to share data on US trial in Q4 2024.

Anavex Life Sciences, a biotech company that has come under fire for trying to spin its clinical trial results, wants to claim partial success on a late-stage trial in kids with Rett syndrome, a rare genetic neurological disorder. However, the biotech’s own ad-hoc analysis says otherwise.

Neurogene has announced that their merger with Neoleukin is finalized. The company has sufficient funds to advance their gene therapy programs into the second half of 2026.

Texas Children's Hospital are the first to delivery a novel gene therapy to treat pediatic patients with Rett syndrome with a gene therapy product developed by the biotech company Neurogene. Two female patients with Rett syndrome were the first children worldwide to receive this treatment.

Neurogene, a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, announced today the dosing of the first two female pediatric patients with Rett syndrome in its ongoing Phase 1/2 trial of NGN-401. To date, ...

Focused on revolutionizing regulatory documentation and reporting for clinical trials, RSRT is bringing together artificial intelligence, data analytics, insights and state-of-the-art technology to super-charge it’s clinical research process through a joint partnership with Narrativa, a ...

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 ...

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

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 ...

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 provides an update on their lead program as well as their Rett syndrome program, TSHA-102

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.

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.

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.