Review top news and interview highlights from the week ending November 1, 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.
The majority of patients treated with the gene therapy also showed improved myelination as measured by T-2-weighted MRI.
Melissa Spencer, PhD, spoke about the emerging role of adeno-associated virus as a delivery system for gene therapies targeting muscular dystrophies, and the challenges associated with them.
The company made the resubmission after reaching an accord with the FDA in a Type A meeting with the agency that took place in August 2024.
The 3 experts discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease.
Participants in the trial received either a single dose of 25 mg of NTLA-2002, a single dose of 50 mg of NTLA-2002, or a placebo.
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.