CGTLive®’s Weekly Rewind – March 22, 2024

Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.

Arsa-cel, approved under the name Lenmeldy, is set to be priced at $4.25 million.

The associate professor of clinical pediatrics at Cincinnati Children's discussed the latest data from the phase 1/2 STAAR study of isaralgagene civaparvovec.

The gene-edited cell therapy has been approved as Lenmeldy by the FDA.

The head of the early neurodevelopment clinic at Monash Children's Hospital discussed preclinical work in the context of neonatal cell therapy research.

Many surveyed participants were content with current treatments or wary of gene therapy’s adverse events and durability.