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.

The hematologist/oncologist at the University of Pittsburgh Medical Center Hillman Cancer Center in Pennsylvania discussed updates to NCCN guidelines and research to pay attention to in hematologic malignancies.

AAV2-BDNF gene therapy shows promise in preventing neuronal loss and cognitive decline in early Alzheimer disease, according to trial results.

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.

The therapy is intended to restore hearing and support early speech development in children with OTOF-related congenital deafness.

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

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.

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 August 1, 2025.

The opinion recommends against conditional marketing authorization for patients who are ambulatory.

Notably, the BLA resubmission was accepted with priority review and the PDUFA target action date has been set for January 10, 2026.

The FDA also stated that it has concluded its investigation of the death an 8-year-old boy who was treated with Elevidys in Brazil.

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

In episode 5 of ImmunoLogic, Michael T. Lotze, MD, discusses the evolution and future of tumor-infiltrating lymphocyte therapies.

According to Rocket, the RMAT designation was granted based on results from a phase 1 clinical trial (NCT05885412).

The patient, an 8-year-old boy, was treated with Elevidys in Brazil.

Sven Moller-Tank, PhD, a senior director at Regeneron, also discussed his thoughts on trends in the gene therapy field in general.