CGTLive’s Weekly Rewind – September 29, 2023

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Review top news and interview highlights from the week ending September 29, 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. Alternative Polyadenylation May Play a Role in ALS Disease Pathogenesis

Sebastian Michels, MD, a postdoctoral fellow and member of the La Spada Lab at University of California, Irvine, discussed how his lab’s findings may open the door to developing RNA therapeutics for ALS.

2. Shankar Musunuri, PhD, on Gene Modifier Therapy vs Gene Replacement Therapy for IRDs

The chairman, chief executive officer, and cofounder of Ocugen discussed unmet needs within retinitis pigmentosa and the potential of OCU400 gene therapy.

3. Precigen Trying Its Hand at CAR-T for Ovarian Cancer in Phase 1/1b Trial

Notably, the trial is assessing intravenous administration of PRGN-3005 against intraperitoneal administration in 2 separate cohorts.

4. J. Andrew Livingston, MD, on Forging Forward With Novel Sarcoma Trials

The associate professor at MD Anderson Cancer Center discussed the upcoming trial of NY-ESO-1 TCR/IL-15 NK cell therapy for SS and MRCLS.

5. Abeona's RDEB Cell Therapy Up for Review

Abeona has submitted a BLA for its EB-101 cell therapy.

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Nirav Shah, MD, MSHP, associate professor of medicine, at the Medical College of Wisconsin
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Mark Hamilton, MD, PhD, a hematology-oncology and bone marrow transplant (BMT) cell therapy fellow at Stanford University
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Sarah Larson, MD, the medical director of the Immune Effector Cell Therapy Program in the Division of Hematology/Oncology at David Geffen School of Medicine at University of California, Los Angeles (UCLA)
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
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