CGTLive’s Weekly Rewind – September 29, 2023


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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Alfonso Sabater, MD, PhD, on Bringing Gene Therapy to Ophthalmology
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Thomas Crawford, MD, on Helping Patients With SMA Left Behind by Gene Therapy
J. Andrew Livingston, MD
Shankar Musunuri, PhD
Pat Furlong, BSN, RN, on Future Areas of Research in DMD
Michael Kelly, PhD, on Continuing Progress in Muscular Dystrophy in 2024
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