Neurology

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.

The findings come from patients with DMD44 treated in the phase 1/2 EXPLORE44 clinical trial and the phase 2 EXPLORE44OLE clinical trial.

Capricor stated that it was not notified in advance by the FDA that the latter would be publishing the CRL publicly on its website.

Review top news and interview highlights from the week ending September 5, 2025.

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

The rAAVrh8 gene therapy delivers HEXA and HEXB genes and has posted positive phase 1/2 results in infantile GM2 gangliosidosis, with enhancements in enzymatic activity and neurological outcomes.

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

Review top news and interview highlights from the week ending August 29, 2025.

Annaiz Grimm, BS, a research scientist at Seattle Children's Research Institute, discussed the potential use of EngTregs to treat autoimmune disease.

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

Review top news and interview highlights from the week ending August 22, 2025.