NGN-401, a new gene therapy in the pipeline from Neurogene, extended the lifespan and reduced the disease burden in mice with a form of Rett syndrome, the company announced. Researchers also observed that the therapy was safe when delivered to mice or early in the life of…

A gene therapy aimed at slowing disease progression and improving movement, coordination and communication in children with Rett syndrome, is being developed. Mostly seen in girls, Rett affects brain development, causing impairments such as progressive loss of motor skills and speech. “It’s often a full-time job as a parent,…

Taysha Gene Therapies and Catalent have established a partnership to advance Taysha’s gene therapies for neurological disorders, including Rett syndrome. The partnership allows Taysha to use Catalent’s gene therapy facilities in Maryland to expand the company’s manufacturing capacity. The gene therapy for Rett, called TSHA-102, uses a modified,…

Taysha Gene Therapies received $95 million in funding that will support the clinical development of its gene therapy program for neurological disorders that include Rett syndrome. The gene therapy for Rett is called TSHA-102 and is one of 17 such treatments being developed by Taysha. All use…

For the first time, scientists have repaired a mutation in mice that underlies Rett syndrome, partially restoring lost protein function, a recent study reports. The findings provide proof of principle that a technique called RNA editing might prove an effective therapeutic strategy in humans, according to the researchers involved…

Taysha Gene Therapies has raised $30 million and established a partnership with UT Southwestern Medical Center to expand its gene therapy program for neurological disorders caused by mutations in a single gene, called monogenic diseases, including Rett syndrome. The funding will allow the company to expand its…

A form of gene therapy based on providing an unstable version of the MECP2 gene extended survival and improved motor function in a mouse model of Rett syndrome, a study reports. The study, “Whole brain delivery of an instability-prone Mecp2 transgene improves behavioral and molecular pathological…

Sarepeta Therapeutics and StrideBio will collaborate to advance novel gene therapies for four genetic neurological disorders, including Rett syndrome, the companies have announced. Under the agreement, StrideBio, which specializes in viral-based delivery systems for gene therapy, will conduct initial research, development, and manufacturing for the first…