Rett In The News

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lowers repetitive behaviors in a mouse model of a different autism-linked condition called Pitt-Hopkins syndrome, a new unpublished study demonstrates.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 ...

A new class of therapies based on transfer RNA could treat forms of cystic fibrosis, muscular dystrophy, genetic epilepsies, and more

UCLA researchers identify an experimental drug that improves abnormal neural oscillations found in Rett brain organoids.

Taysha Gene Therapies announces ambitious timeline of starting Rett syndrome gene replacement clinical trial by year end and reporting clinical data by end of 2022.

Olympic bronze medal winner Gabby Thomas studied neurobiology at Harvard and worked on Rett syndrome in a lab at Boston Children’s Hospital.

Shape Therapeutics, a biotech focusing on RNA editing, has secured $112 million in financing to advance their research programs. They have two Rett Syndrome programs one of which is their lead program. We’re proud that RSRT has facilitated Shape taking on Rett.

Two years after spinning out of CRISPR pioneer Prashant Mali’s lab, Shape Therapeutics has a lot more cash and a slightly new mission. They have lead programs in Rett syndrome and Parkinson’s.

Scientists in France published a paper showing that the MECP2 protein binds certain repeating sequences in strands of DNA thereby changing its structure. Mutated MECP2 could not bind and therefore no change in DNA strucuture was seen. If and how this finding is relevant to developing treatments ...

Ella Gaspard, a Virgin Islander, is diagnosed with Rett Syndrome. Ella’s Hope invites the public to join it on the first STT Cross-Island Challenge Ride to be held on July 3. One hundred percent of all proceeds raised from the STT Cross-Island Challenge will be donated to Rett Syndrome ...

The Massachusetts biotech unveiled itself Wednesday with $23 million in funding from RTW Investments with a pipeline of 12 adeno-associated virus (AAV) gene therapy programs for severe CNS disorders. Alcyone lead programs include ACTX-101, a Rett syndrome gene therapy in pre-IND enabling ...