CGTLive’s Weekly Rewind – December 2, 2022

Article

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.

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Shahzad Raza, MD, a hematologist/oncologist at the Cleveland Clinic
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Shahzad Raza, MD, a hematologist/oncologist at the Cleveland Clinic
Laura Aguilar MD, PhD, the chief medical officer of Diakonos Oncology
Jamie Jacobs, PhD, the program director of the center for psychiatric oncology & behavioral sciences at Mass General Cancer Center
Laura Aguilar MD, PhD, the chief medical officer of Diakonos Oncology
Sarah Hein, PhD, the chief executive officer and cofounder of March Biosciences
Kiran Musunuru, MD, PhD, a physician-scientist and Barry J. Gertz Professor for translational research in the Perelman School of Medicine at the University of Pennsylvania and CHOP and Rebecca Ahrens-Nicklas, MD, PhD, a physician-scientist and director of the Gene Therapy for Inherited Metabolic Disorders Frontier Program at CHOP
Kiran Musunuru, MD, PhD, a physician-scientist and Barry J. Gertz Professor for translational research in the Perelman School of Medicine at the University of Pennsylvania and CHOP and Rebecca Ahrens-Nicklas, MD, PhD, a physician-scientist and director of the Gene Therapy for Inherited Metabolic Disorders Frontier Program at CHOP
Kiran Musunuru, MD, PhD, a physician-scientist and Barry J. Gertz Professor for translational research in the Perelman School of Medicine at the University of Pennsylvania and CHOP and Rebecca Ahrens-Nicklas, MD, PhD, a physician-scientist and director of the Gene Therapy for Inherited Metabolic Disorders Frontier Program at CHOP
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