CGTLive’s Weekly Rewind – 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.