News

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

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.

The associate professor of medicine at Medical College of Wisconsin discussed efficacy data presented at the 2025 Tandem Meetings.

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

The associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center discussed results from a phase 1 clinical trial.

The success of cell and gene therapies relies not just on approvability but on affordability and accessibility. Innovative manufacturing, automation, and digital tools are key to expanding patient access and ensuring commercial viability.

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

The data, which comes from a phase 2 trial, were presented at the 2025 Tandem Meetings.

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.

In observance of International Epilepsy Day, held annually on the second Monday of February, we took a look back at the past year's news in cell and gene therapy for epilepsy indications.

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

Review top news and interview highlights from the week ending February 7, 2025.

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."