Neurology

Patients treated at Dose 2 in the dose-escalation phase of Part 1 of the study showed a persistent 68% reduction in CK levels over 2 years.

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

OAV101 IT is a version of Zolgensma that is delivered directly to the spine.

Acute liver injury is known to be a possible adverse event associated with AAV vector-based gene therapies such as Elevidys.

The findings were presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference

Adverse events related to the gene therapy itself were mild or moderate, with most occurring in the first 90 days posttreatment.

Review top news and interview highlights from the week ending March 14, 2025.

John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, offered insights into the obstacles the clinical community is facing around integrating gene therapies into clinical practice.

The study included 48 patients, 36 of whom received laromestrocel and 12 of whom received a placebo.

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

In Episode 2 of ImmunoLogic, Bruce Levine, PhD, discussed the current the risk-benefit-ratio for CAR-T therapy.

Review top news and interview highlights from the week ending March 7, 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.

The agency has set the PDUFA action date for the BLA as August 31, 2025.

Following up on Rare Disease Day, observed on February 28, Mary Schroth, MD, FAAP, FCCP, chief medical officer at Cure SMA, shared insights on the advances and promise for treating spinal muscular atrophy.

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 February 28, 2025.

In honor of Rare Disease Day, observed this year on February 28, Amber Freed, the founder of SLC6A1 Connect, shared thoughts on advocacy and efforts to raise awareness for rare genetic disease SLC6A1.

The company intends to pursue a full IND application for troculeucel in FTD.

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

The FDA’s decision was based on a review of findings from a phase 1b clinical trial.

Review top news and interview highlights from the week ending February 21, 2025.

The PDUFA action date for the BLA has been set at August 18, 2025, and no advisory committee meeting is currently planned.

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