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 ...
St. Thomas Source
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.
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
Taysha Gene Therapies Announces Publication of Preclinical Data for TSHA-102 in Rett Syndrome in Brain
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.
Behavioral Training Could Help Babies with Rett Syndrome, Mouse Study Suggests
In mouse models of Rett Syndrome intensive training in young mice staved off symptoms of the disorder suggesting that perhaps the same might be true in people.
University of Connecticut
UConn professor of physiology and neurobiology Daniel Mulkey has received a $2.1 million grant from the NIH to investigate the cellular and molecular basis for disordered breathing in Rett syndrome. This project is part of a collaboration with Michelle Olsen, associate professor of neurobiology ...
UVA’s Sanchita Bhatnagar, PhD, discovered that tiny bits of RNA, called microRNAs, play an important role in Rett, a rare genetic disorder that can impair children’s ability to speak, move and even breathe.
Texas Children's Hospital
ASO as a feasible therapy to treat MECP2 duplication disorder
A study by Dr. Zoghbi provides experimental evidence that supports the use of antisense oligonucleotides as a feasible strategy to treat MECP2 Duplication Syndrome. Funding for this work came from the MECP2 Duplication Syndrome Fund at RSRT.
Rett Syndrome Awareness on Anderson Coooper's Full Circle
Four girls with Rett Syndrome and their moms discuss what they are grateful for in the "For Goods" section of "Full Circle"
Altering a protein linked to Rett syndrome so that it cannot bind to a particular type of DNA tag may explain the syndrome’s delayed onset in people.
The Washington Post
They thought they’d be near the front of the line for the vaccine. Now, they don’t know where they stand.
The long wait for vaccination has left many people across the country feeling anxious, desperate even. But for the young and disabled, and for the people who care about them, the realization of where they fall on priority lists has also left them feeling disregarded and discarded.
Taysha Gene Therapies, Inc. has highlighted its strategic priorities and provided a business outlook for 2021.
Neurogene and University of Edinburgh Announce Research Collaboration to Advance Next Generation Gene Therapies
Neurogene Inc. has announced a collaboration to advance development of multiple platform approaches designed to enable next generation gene therapies.
Two unpublished studies detail improved methods for delivering gene therapies to the brain: One involves a type of stem cell that can produce gene-altering proteins on-site; the other taps an engineered virus to target neurons efficiently and noninvasively.
The New York Times
Clusters of living brain cells are teaching scientists about diseases like autism. With a new finding, some experts wonder if these organoids may become too much like the real thing.
A gene therapy for Angelman syndrome stands at the forefront of efforts to treat autism-linked conditions that stem from single genes.
October is Rett Syndrome Month. It's a rare and very challenging disease that affects just one in 10,000 girls. It's a tough go, but there is hope. Tim Freeman from the Rett Syndrome Research Trust tells us more.