In the CRL, the FDA stated that the statutory requirement for “substantial evidence of effectiveness” was not met by the BLA and that more clinical data will be needed.

SGT-501 is intended to address CPVT by providing a full-length, codon-optimized copy of the cardiac calsequestrin gene to the muscle cells of the heart.

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed how comprehensive care for DMD patients involves early diagnosis, steroid treatment, consideration of mutation-specific therapies, and more.

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

The vice president of cell & gene therapy product development and the vice president of genetic medicine platforms at Pacira discussed the company’s symposium at ASGCT 2025.

Jeffrey Chamberlain, PhD, McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed how gene therapy shows promise for DMD treatment.

The vice president of cell & gene therapy product development at Pacira BioSciences discussed findings related to the immunogenicity of the therapy’s high-capacity adenovirus vector.

Alongside the new data from REGEN-007, ProKidney also announced that it will be attending a Type B meeting with the FDA later in the summer.

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.