News

Cells lacking Mecp2 harm healthy neurons in Rett mouse study

New research supports how the lack of Mecp2 protein impairs the function of healthy nerve cells in a mouse study of Rett syndrome. Specifically, the function of nerve cells was affected when the cells were grown alongside nerve-cell supporting astrocytes lacking the Mecp2 protein, mimicking the genetic defect that…

Rett gene therapy NGN-401 trial expanding, accelerating

Neurogene is expanding its ongoing Phase 1/2 clinical trial testing gene therapy candidate NGN-401 in girls with Rett syndrome and making some changes to the trial’s structure to accelerate enrollment and dosing. Changes include adding more patients and removing dose staggering (one-at-a-time dosing) in the trial’s first low-dose…

Taysha gets green light for dose escalation in TSHA-102 trial

Taysha Gene Therapies has received the needed approval to move forward with dose escalation in its Phase 1/2 trial testing TSHA-102, its gene therapy candidate for Rett syndrome, in adolescents and adults, the company announced. That green light from the study’s independent data monitoring committee (IDMC) — a group…

Intrathecal TSHA-102 leads to improvements in Rett mice

An injection of Taysha Gene Therapies’ experimental gene therapy TSHA-102 into the spinal canal of young mice modeling Rett syndrome can improve weight, survival, breathing, and motor skills, a preclinical study suggests. The data support using the intrathecal route in ongoing Phase 1/2…

Neurologists appear to be more comfortable managing Rett: Survey

Among experienced physicians, neurologists are reportedly more comfortable than pediatricians in treating people with Rett syndrome, according to a survey conducted in the U.S. Neurologists also consider more tools than pediatricians do when diagnosing patients with Rett, data show. These findings highlight the need for “better education and support…

1st child dosed with gene therapy TSHA-102 in REVEAL trial

The first girl with Rett syndrome has been given the experimental gene therapy TSHA-102 in a clinical trial, according to the treatment’s developer, Taysha Gene Therapies. “Dosing the first pediatric patient with Rett syndrome marks an important step forward in our efforts to broaden the clinical evaluation…