Neurology

Review top news and interview highlights from the week ending October 17, 2025.

Headaches, falls, and nausea constituted the common treatment-emergent AEs, and were deemed mild-to-moderate in terms of severity.

Tr1X intends to go forward with plans for a phase 1/2a dose-escalation clinical trial, which it expects to launch early next year.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

With regard to safety, MYR-101 was characterized as “well tolerated” with a “favorable safety profile.”

Review top news and interview highlights from the week ending October 10, 2025.

Neurogene also relayed new preclinical findings on the use of intracerebroventricular and intrathecal lumbar delivery of NGN-401.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Taysha also announced that the FDA has aligned with the company on the pivotal trial protocol and statistical analysis plan.

Review top news and interview highlights from the week ending October 3, 2025.

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive® team.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

MVX-220 is intended to provide a functional copy of UBE3A, the disease-targeted gene, to the neurons.

Review top news and interview highlights from the week ending September 26, 2025.

The study also met a key secondary end point.

Jainu Jogani, the cofounder of Child’s Cure Genetic Research, discussed his daughter’s rare genetic disease and the need for new treatment options.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

In honor of Duchenne Action Month and World Duchenne Day, held every year in September, CGTLive is taking a closer look at this ongoing study.

Review top news and interview highlights from the week ending September 19, 2025.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

In observance of Duchenne Action Month and World Duchenne Day, held annually in September, we took a look back at recent news in gene and cell therapy for DMD.

Review top news and interview highlights from the week ending September 12, 2025.

Capsida has made the move to pause the trial voluntarily.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.