CGTLive®’s Weekly Rewind – November 22, 2024

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Review top news and interview highlights from the week ending November 22, 2024.

CGTLive®’s Weekly Rewind

CGTLive®’s Weekly Rewind

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. Atamyo Therapeutics' Gene Therapy ATA-200 Cleared for US Trial in LGMD Type 2C/R5

In addition to the IND clearance, the FDA also granted ATA-200 orphan drug designation.

2. Bhagirathbhai R. Dholaria, MD, on Evaluating Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma

The associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center discussed interim data from the phase 1/1b clinical trial evaluating Poseida's CAR-T.

3. Patient Treated With Neurogene’s Investigational Rett Syndrome Gene Therapy NGN-401 in Critical Condition After Developing Life-Threatening Immune Response

The patient was treated in the trial’s high-dose cohort, which Neurogene will now discontinue in favor of the low-dose cohort.

4. David Barrett, JD, on ASGCT’s Q3 2024 Landscape Report

The chief executive officer of ASGCT discussed the latest trends in the field of cell and gene therapy.

5. First Patient Dosed in Trial for Adicet's T-Cell Therapy ADI-100 in Autoimmune Disease

Adicet is also presenting biomarker data at ACR Convergance from a trial evaluating ADI-001 in B-cell malignancies that may indicate its potential to treat autoimmune diseases.



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Haydar Frangoul, MD, the medical director of pediatric hematology/oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial
Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center
Caroline Diorio, MD, FRCPC, FAAP, an attending physician at the Cancer Center at Children's Hospital of Philadelphia
R. Nolan Townsend; Sandi See Tai, MD; Kim G. Johnson, MD
Paul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
Barry J. Byrne, MD, PhD, the chief medical advisor of Muscular Dystrophy Association (MDA) and a physician-scientist at the University of Florida
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
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