Noah Stansfield

The research scientist at Seattle Children's Research Institute discussed a potential alternative to standard of care immunosuppressive therapy.

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

Prerna Mewawalla, MD, the medical director of Apheresis and a hematologist-oncologist in the Division of Hematology and Cellular Therapy at Allegheny Health Network, spoke on unmet needs in patients who receive early-line CAR-T for R/R MM.

Notably, the FDA granted Papzimeos a full approval, without a requirement for a confirmatory trial.

Ultragenyx anticipates that the full BLA will have been submitted in the fourth quarter of this year.

The new PDUFA date is set for February 8, 2026.

Surbhi Sidana, MD, the assistant professor of medicine, bone marrow transplantation, and cellular therapy at Stanford discussed the benefit of second-line cilta-cel in PFS and OS.

Review top news and interview highlights from the week ending August 22, 2025.

The hold was originally placed on the trial by the FDA on May 23, 2025, following the death of a patient treated in the study.

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

Damien Maura, PhD, a senior scientist at Voyager, discussed the company’s work on developing AAV capsids capable of overcoming patients’ preexisting immunity.

Lifileucel has been approved in the US since February 2024.

The nonrandomized, open-label clinical trial for pCHIM-p47, which launched on March 20, 2023, is taking place at Great Ormond Street Hospital in London.

The senior scientist at Voyager Therapeutics discussed the company’s work on developing AAV capsids capable of overcoming patients’ preexisting immunity.

The postdoctoral research fellow at Brigham and Women's Hospital discussed research on focused ultrasound aimed at enhancing AAV delivery across the blood-brain barrier.

Review top news and interview highlights from the week ending August 15, 2025.

Notably, the first patient was dosed in the GARDian3 in July 2025.

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

The RMAT designation was granted based on data from the phase 1/2 ATALANTA-1 clinical trial, which includes a subset of patients with R/R MCL.

Rajeev Sivasankaran, PhD, vice president of neuroscience at Voyager Therapeutics, discussed preclinical data on the company’s AAV-delivered RNA interference therapy, VY-1706.

AAVB-039 is intended to deliver a functional copy of the disease-targeted ABCA4 gene.

The move was based on updated safety data from an FDA probe into the risk of hematologic malignancies in patients treated with eli-cel.

Review top news and interview highlights from the week ending August 8, 2025.

The company noted that as a result of the meeting’s scheduling it has rescheduled its release of financial results for the second quarter to August 11, 2025.

INmune Bio reported that INKmune was well-tolerated at the 3 dose levels used in the trial.

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

The company noted that it plans to submit the meeting request by the fourth quarter of this year and that the meeting will focus on the approvability of the product based on available clinical data.

The Prescription Drug User Fee Act goal date for the sBLA has been set for December 5, 2025.

The patient’s death was attributed to anti-CD52 monoclonal antibody ALLO-647 rather than to cema-cel itself.

In observance of Spinal Muscular Atrophy Awareness Month, held annually in August, we took a look back at the past year's news in gene and cell therapy for SMA.