CGTLive®’s Weekly Rewind – November 22, 2024
Review top news and interview highlights from the week ending November 22, 2024.
CGTLive®’s Weekly Rewind
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. Atamyo Therapeutics' Gene Therapy ATA-200 Cleared for US Trial in LGMD Type 2C/R5
In addition to the IND clearance, the FDA also granted ATA-200 orphan drug designation.
2. Bhagirathbhai R. Dholaria, MD, on Evaluating Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma
The associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center discussed interim data from the phase 1/1b clinical trial evaluating Poseida's CAR-T.
3. Patient Treated With Neurogene’s Investigational Rett Syndrome Gene Therapy NGN-401 in Critical Condition After Developing Life-Threatening Immune Response
The patient was treated in the trial’s high-dose cohort, which Neurogene will now discontinue in favor of the low-dose cohort.
4. David Barrett, JD, on ASGCT’s Q3 2024 Landscape Report
The chief executive officer of ASGCT discussed the latest trends in the field of cell and gene therapy.
5. First Patient Dosed in Trial for Adicet's T-Cell Therapy ADI-100 in Autoimmune Disease
Adicet is also presenting biomarker data at ACR Convergance from a trial evaluating ADI-001 in B-cell malignancies that may indicate its potential to treat autoimmune diseases.
Sickle Cell Disease Gene Therapy Exa-Cel's Ability to Prevent VOCs
December 12th 2024Haydar Frangoul, MD, the medical director of pediatric hematology/oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial, discussed the latest data update from the CLIMB SCD-121 trial evaluating exa-cel.
10-Year Data Show Allogeneic Stem Cell Transplant Benefits for Sickle Cell Anemia
December 10th 2024A long-term follow-up to the DREPAGREFFE-1 trial suggest that children with sickle cell anemia may benefit long-term on risk of cerebral injury, cognitive functions, and quality of life over standard care transfusions.
Autologous HCT Shows No Benefit for Patients With MCL in First Complete Remission
December 10th 2024Among those who had undetectable minimal residual disease, autologous hematopoietic cell transplantation showed signs of benefit only for those who remained MRD-positive following induction therapy.
