CGTLive’s Weekly Rewind – January 19, 2024
Review top news and interview highlights from the week ending January 19, 2024.
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.
Bendamustine Is an Effective Alternative to Fludarabine-Based Lymphodepletion in LBCL
December 7th 2024In the wake of fludarabine shortages, lemphodepletion with bendamustine was found to be an effective alternative compared for patients with large B-cell lymphoma being treated with a CD19-directed CAR T-cell therapy.
