CGTLive’s Weekly Rewind – January 19, 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.

1. Vertex Nets Early Casgevy Approval for Transfusion-Dependent Thalassemia

The therapy’s original PDUFA date was March 30, 2024, but the TDT approval followed only a month after exa-cel's sickle cell approval.

2. David Shyr, MD, on Surprising Findings With CRISPR Cell Therapy Nula-Cel in Sickle Cell Disease

The clinical assistant professor at Stanford Medicine discussed outcomes of the first patient that received KMAU-011.

3. Passage Bio’s Gene Therapy PBFT02 Positively Impacts Biomarker Levels in Patients With Frontotemporal Dementia

Among 3 patients in the study’s first cohort, a 3.6 to 6.6-fold increase in CSF PGRN over baseline was observed at 30 days posttreatment.

4. Tom E. Howard, MD, PhD, on Bringing Together Knowledge of Genetic Diversity and HLA Molecules for Gene Editing Strategies

The clinical professor in the Department of Human Genetics at University of Texas Rio Grande Valley discussed research on racial differences in the Factor VIII gene that can impact treatment efficacy for hemophilia A.

5. MultiStem Cell Therapy Not Superior to Placebo in Patients With Ischemic Stroke

Further research is needed to determine if cell therapy may be beneficial to certain subgroups of patients.