CGTLive’s Weekly Rewind – September 8, 2023
Review top news and interview highlights from the week ending September 8, 2023.
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. uniQure's Mesial Temporal Lobe Epilepsy Gene Therapy AMT-260 Cleared for US Trial
In light of the IND clearance, uniQure announced its intention to carry out a phase 1/2a clinical trial, with screening of potential participants with refractory MTLE anticipated to begin in the last 3 months of 2023.
2. Pamela Garzone, PhD, on Exploring Unknowns in CAR-T for Solid Tumors
The chief development officer of Anixa Biosciences discussed the company’s trial of a CAR-T in ovarian cancer that is looking at the effect of regional administration and lymphodepletion on efficacy.
3. Nusinersen Show Benefits in Other SMA Populations
New data shows the ASO therapy’s benefit in older populations, following beneficial findings in patients previously treated with gene therapy.
4. Fiona Freeman, PhD, on Investigating miRNA-29b in Osteosarcoma Models
The assistant professor at University College Dublin discussed challenges and different approaches to using microRNA in preclinical models of osteosarcoma.
5. Arsa-cel Continues to Show Benefit Up To 12 Years of Follow-up in Metachromatic Leukodystrophy
The data come from 30 patients treated across 2 clinical trials and 9 patients treated in expanded access frameworks who had follow-up times ranging from 0.64 years to 12.19 years.
Evaluating Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma
November 21st 2024Bhagirathbhai R. Dholaria, MD, an 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.
World Pancreatic Cancer Day 2024: Looking Back at Progress in Cell and Gene Therapy
November 21st 2024In observance of World Pancreatic Cancer Day, held on the third Thursday of November each year, we took a look back at the past year's news in cell and gene therapy for pancreatic cancer indications.
