CGTLive’s Weekly Rewind – December 2, 2022
Review top news and interview highlights from the week ending December 2, 2022.
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. CAR-T Outcome Measures Unaffected by Socioeconomic Status and Neighborhood Opportunity
There was no significant difference in OS and CR rate for patients based on household poverty and neighborhood opportunity.
2. Arshad Khanani, MD, on Reducing Treatment Burden in Wet AMD With Gene Therapy
The director of clinical research at Sierra Eye Associates discussed unmet needs in patients with wet AMD.
3. GM1 Gangliosidosis Gene Therapy Trial Safe to Continue
Lysogene’s MPS IIIA gene therapy program recently failed its primary endpoint in the phase 2/3 AAVANCE trial.
4. Hemgenix Approval May Pave Way for Val-Rox in Hemophilia A
The FDA has decided not to hold an advisory committee meeting for Roctavian after all.
5. Maria Escolar, MD, on Addressing Unmet Needs in Krabbe Disease With Gene Therapy
The chief medical officer of Forge Biologics discussed clinical data presented at ESGCT.
News and Expert Insights in Hemophilia Gene Therapy for Bleeding Disorders Awareness Month 2025
March 9th 2025In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest news and expert insights in gene therapy for hemophilia, which is among the most common of these disorders.
