CGTLive’s Weekly Rewind – October 27, 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. Mark Walters, MD, on Long-Term Risks of Blood Cancer in Children Treated With Gene-Edited Cell Therapy

The professor in residence of pediatrics at University of California San Francisco discussed a potential long-term consequence of gene therapy and myeloablative conditioning.

2. REGENXBIO’s ABBV-RGX-314 Trials Aim to Reduce Treatment Burden in Inherited Retinal Diseases

The company expects that pivotal ATMOSPHERE and ASCENT trials will support a BLA filing in 2024 for wet AMD, while the AAVIATE trial evaluates the therapy in diabetic retinopathy.

3. David Rawlings, MD, on the Potential of Engineered Treg Cells to Treat Autoimmune Diseases

The director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute discussed preclinical research on overcoming limitations of Treg cells through genetic engineering.

4. Focal Epilepsy Cell Therapy NRTX-10 Shows Efficacy Signals in First Patient Cohort

Neurona Therapeutics also reported that this first patient cohort has completed enrollment in addition to the early efficacy data from the first 2 patients treated.

5. Positive Preclinical Data Support FTD-GRN Gene Therapy Study Initiation

AviadoBio announced the initiation of the phase 1/2 ASPIRE-FTD clinical trial earlier in October.