CGTLive®’s Weekly Rewind – November 1, 2024
Review top news and interview highlights from the week ending November 1, 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. BridgeBio Pharma’s Gene Therapy BBP-812 Effects Progressive Improvement in Motor Function in Patients With Canavan Disease
The majority of patients treated with the gene therapy also showed improved myelination as measured by T-2-weighted MRI.
2. Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy
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.
3. Abeona Therapeutics Resubmits BLA to FDA for Epidermolysis Bullosa Gene Therapy Pz-Cel
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.
4. R. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, on Gene Therapy for APOE4 Homozygous Alzheimer Disease
The 3 experts discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease.
5. Intellia’s CRISPR Gene Editing Therapy NTLA-2002 Appears to Eliminate Hereditary Angioedema Attacks in Some Patients Following One-Time Treatment
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.
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.
