News

SNUG01 is intended to deliver a copy of the human TRIM72 gene to neurons via an rAAV9 vector.

The NDA is supported primarily by data from the phase 2 CT041-ST-01 randomized controlled clinical trial.

Alexey Danilov, MD, PhD, discussed the importance of reassessing sequencing stratagems in the face of a growing toolbox of targeted therapies in DLBCL.

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington discusssed how gene therapy shows promise for DMD treatment, though challenges remain with delivery efficiency and determining which patients will benefit most.

Several experts weighed in on the various strategies being explored to tackle solid tumors with cell therapy, expressing optimism despite the plethora of obstacles that remain.

Review top news and interview highlights from the week ending July 4, 2025.

The FDA made the move with the view that the REMS are no longer necessary to ensure a favorable risk-benefit-ratio for the products and that dropping the requirement may increase accessibility to these therapies.

Tami John, MD, a clinical associate professor at Stanford Medicine, emphasized the need to build community among patients with TDT.

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

The IND clearance enables the company to start a first-in-human phase 1 clinical trial for the gene therapy.

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

Tami John, MD, a clinical associate professor at Stanford Medicine, discussed considerations for using beti-cel and exa-cel for TDT in the clinic.

Tami John, MD, a clinical associate professor at Stanford Medicine, also provided background about the current state of care in TDT.

The 3 FDA-approved CAR T-cell therapies showed comparable survival outcomes in DLBCL over a 3-year period.

Review top news and interview highlights from the week ending June 27, 2025.

The clinical associate professor at Stanford Medicine emphasized the need to build community among patients with TDT.

The clinical associate professor at Stanford Medicine discussed considerations for using beti-cel and exa-cel for TDT in the clinic.

The gene therapy is currently being evaluated in the phase 1/2 LEGEND clinical trial.

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

The FDA stated that it is assessing the risk of ALF that results in hospitalization or death after treatment and whether additional regulatory measures will be necessary.

The clinical associate professor at Stanford Medicine also provided background about the current state of care in TDT.

In a mid-cycle review meeting, the agency also noted that the review committee found no major deficiencies with the BLA package.

The study, which may enroll from 20 to 200 participants in total, will administer the DC-CTL cells 1 to 2 days after the patients have received conventional antitumor therapy.

Peter Cook, PhD, a senior research scientist at Seattle Children’s Research Institute, discussed preclinical work on using dimerizing agent-regulated immune-receptor complex T-cells to target plasma cells.

Brian Kim, MBA, the chief executive officer of Mission Bio, also discussed the company’s plans for collaboration.

The patients also showed cessation of severe hypoglycemia events and hit recommended glycemic control targets.

Review top news and interview highlights from the week ending June 20, 2025.

Brian Kim, MBA, the chief executive officer of Mission Bio, discussed the company’s single cell assay platform in the context of the broader developing field cell and gene therapy field.

In honor of World Sickle Cell Day, observed annually on June 19, we took a look back at news and expert insights from the past year in cell and gene therapy for SCD.