Noah Stansfield

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

CGTLive® took a closer look at the single-center, open-label study, which aims to assess the safety and efficacy of the CAR-T therapy.

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.

David-Alexandre Gros, MD, the chief executive officer of Eledon Pharmaceuticals, discussed the company’s collaboration with EGenesis.

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

David Barrett, JD, the chief executive officer of ASGCT, broke down the highlights of the organization’s final report for 2024.

Neurotech expects that the therapy will become available in the United States in June 2025.

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

Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital, discussed areas of interest for future study for the Gaucher disease gene therapy product.

The chief executive officer of Eledon Pharmaceuticals discussed the company’s collaboration with EGenesis.

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

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

The chief executive officer of ASGCT broke down the highlights of the organization’s final report for 2024.

Out of 11 treated children who had at least 1 posttreatment assessment, 10 showed improved hearing at various decibel hearing levels.

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

Alfred L. Garfall, MD, MS, discussed data from the phase 2 BMT CTN 1902 trial.

The PDUFA action date for the BLA has been set at August 27, 2025.

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

The adult metabolic consultant at Salford Royal Hospital discussed areas of interest for future study for the Gaucher disease gene therapy product.

The data monitoring committee reviewed safety data from the trial’s first cohort, which included 3 patients, and deemed the findings favorable.

The company made the decision based on a lack of interest from the patient and clinician communities.

BRTX-100 is currently being evaluated in a prospective, randomized, and controlled phase 2 clinical trial.

Nirav Shah, MD, an associate professor of medicine at Medical College of Wisconsin, discussed efficacy data presented at the 2025 Tandem Meetings.

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.

Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center, discussed new results presented at Tandem 2025.

With regard to safety, the company characterized SGT-003 as well-tolerated.

CGTLive® took a closer look at the phase 3 study, which is tracking patients for up to 15 years after their treatment with GLPG CAR-T products.