Neurology

The patient showed grade 4 liver transaminases and increased total bilirubin and was hospitalized.

Deborah Phippard, PhD, and Renier Brentjens, MD, PhD, discussed the unpredictability of "living drugs" in the human body and the ongoing development of understanding.

Deborah Phippard, PhD, and Renier Brentjens, MD, PhD, shared their thoughts on how streamlined collaboration can help move therapies to patients faster.

Deborah Phippard, PhD, and Renier Brentjens, MD, PhD, discussed strategies used to streamline development in cell and gene therapy.

Deborah Phippard, PhD, and Renier Brentjens, MD, PhD, discussed the major friction points in cell and gene therapy development and uptake.

Review top news and interview highlights from the week ending October 24, 2025.

Deborah Phippard, PhD, and Renier Brentjens, MD, PhD, discussed their thoughts on major moments in the past 25 years of cell and gene therapy research.

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

The primary end point of the study was met.

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.