Rett In The News

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 ...

In downtown Lowell, MA, tucked into one of the country’s oldest surviving textile mill complexes is a new biotech toiling away on next-gen CNS therapies for severe neurological disorders. And it’s starting with Rett syndrome — a rare neurodevelopmental condition that almost exclusively ...

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 ...

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 ...

“This is the way to do it, to discover drugs for neurological diseases,” says Sharath Hegde “And it’s not going to be easy, but this is the way to do it.” And the company's lead program is Rett Syndrome.

Herophilus appoints a new Chief Scientific Officer and sets its sight on Rett Syndrome.

RNA used to be considered a bit player. DNA was the start. Today it's a different story....

I’m proud of the work we have done at my production company, Peak Curiosity, and with the charitable advocacy work we do at Magnolia’s Hope on behalf of my daughter who suffers from Rett Syndrome.

Taysha Gene Therapies announces the publication of new preclinical data for their Rett Syndrome program, TSHA-102.

Farmingdale pharmaceutical firm DepYmed is no stranger to capital investments – but a new infusion might be enough to push the rare-disease innovator over the commercialization line.