Rett In The News

Researchers from UMASS working on Rett have created a more compact genome editor that can be more easily delivered into cells.

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, 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 ...

An update from Taysha on their Rett gene replacement program that got its start with RSRT funding.

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 will be providing an update on its lead program. ACTX-101, which got its start through RSRT funding.

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 announces that Health Canada has given clearance to begin a gene replacement trial for Rett Syndrome.

A therapeutic molecule that makes the brain less likely to seize in mice with severe seizures also reduces symptoms in mouse models of Rett syndrome.

A new mouse model may accelerate the discovery of gene-activating therapies to treat Rett syndrome, a condition related to autism, its makers say.

Spectrum, a leading source of news on autism research, spoke to Dr. von Hehn, RSRT's senior director of research and clinical strategy, about the Rett Syndrome Global Registry which will be launching soon.

BioNews reports on RSRT's 2021 research awards in support of CURE 360.

Technology Networks speaks with Saul Kato, the CEO of Herophilus, about how the company plans to reinvent neuro drug discovery, by combining human brain models, scaled biology and machine learning to decode brain disease, including Rett syndrome.

RSRT has invested $8 million in RNA editing and the investment is paying off! Four of the seven companies mentioned in this article have Rett programs. Read on....

Increasing expression of the gene that is faulty in Rett syndrome lowers repetitive behaviors in a mouse model of a different autism-linked condition called Pitt-Hopkins syndrome, a new unpublished study demonstrates.

Taysha Gene Therapies today announced that it has been granted orphan drug designation from the European Commission for TSHA-102, an AAV9-based gene replacement therapy in development for Rett syndrome. An orphan designation allows a pharmaceutical company to benefit from incentives such as ...