CGTLive®’s Weekly Rewind – November 8, 2024
Review top news and interview highlights from the week ending November 8, 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. RiboX's Circular RNA Therapy RXRG001 Cleared for Phase 1/2a Trial in Radiation-Induced Xerostomia and Hyposalivation
RiboX stated that this is the first IND clearance the FDA has granted for any circular RNA therapy.
2. Caroline Diorio, MD, FRCPC, FAAP, on Noninflammatory Neurotoxicity Events in Patients Receiving CAR-T
The attending physician at the Cancer Center at Children's Hospital of Philadelphia discussed a study she coauthored that was recently published in Blood.
3. Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients With Alzheimer Disease
LX1001's safety profile was characterized as well-tolerated with no amyloid-related imaging abnormalities reported.
4. Nanoscope Therapeutics’ Retinitis Pigmentosa Gene Therapy MCO-010 Improves Visual Acuity in Randomized Controlled Clinical Trial
The randomized, controlled clinical trial treated patients with either a high dose of MCO-010, a low dose of MCO-010, or a sham–control.
5. FDA Removes Clinical Holds Previously Placed on 3 of CARsgen's Oncology Cell Therapies
The clinical holds on the 3 products were originally placed by the FDA in December 2023 based on findings from an inspection of CARsgen’s manufacturing facility.
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.
