News

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…

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…