Rett In The News

An informative article on the recent and tragic death of a young child in a gene therapy trial for Rett syndrome.

"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 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 ...

Taysha reports financial results as well as updates on their pediatric and adult clinical trials.

Neurogene announced initial safety and tolerability data from its Rett gene therapy clinical. NGN-401 was generally well-tolerated by all three patients dosed, with follow-up of approximately 9, 6 and 3 months post-dosing. These data were presented during the American Society of Gene and Cell ...

Taysha announced the FDA has granted RMAT designation to TSHA-102. Companies receiving RMAT designation can benefit from increased interactions with the FDA with the goal of expediting drug development.

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

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.

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.

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