Noah Stansfield

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.

Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital, discussed results she presented at the 21st Annual WORLDSymposium.

The adult metabolic consultant at Salford Royal Hospital discussed results she presented at the 21st Annual WORLDSymposium.

AlloHeme utilizes detection of increased mixed chimerism (iMC) at a threshold of 0.2% or greater in the recipient’s chimerism between 2 consecutive readings to predict relapse.

Liso-cel showed a statistically significant and clinically meaningful overall response rate in the cohort.

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

Troculeucel is currently being evaluated in a phase 1/2a trial (NCT06189963) for moderate AD.

The transplant, which took place at Massachusetts General Hospital MGH, was part of an FDA-authorized Expanded Access pathway.

The company stated that at 1 year posttreatment AlloStem showed an 80% overall efficacy rate in decreasing insulin dependency.

Specifically, the recommendation pertains to a potential indication for adults with r/r FL who have received 2 or more prior lines of systemic therapy.

The therapy is currently being evaluated for AL amyloidosis in the phase 1b/2 NEXICART-2 clinical trial (NCT06097832) in the United States.

Mark Hamilton, MD, PhD, a hematology-oncology and BMT cell therapy fellow at Stanford University, discussed implications of his institution’s findings on treatment-related secondary malignancies.

A positive mean eGFR slope was seen in 23 patients who reached 1 year of follow-up in the phase 1/2 STAAR trial.

With regard to safety, Ultragenyx characterized the gene therapy as “generally well-tolerated."

The product, also known as Vigil, is being evaluated in the ongoing randomized, placebo-controlled phase 2b VITAL clinical trial.