CGTLive’s Weekly Rewind – December 22, 2023

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Review top news and interview highlights from the week ending December 22, 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. Excision Seeks to Suppress HIV Viral Replication With EBT-101 CRISPR Gene Therapy

The company has released positive safety data from the first 3 participants with HIV-1 so far, with no evidence of vector shedding in sexual organ tissue.

2. Franck Morschhauser, MD, PhD, on Next Steps for Follicular Lymphoma and Liso-Cel

The professor of hematology at University of Lille and chair of LYSA also discussed what the group will be focusing on in 2024.

3. Gene-Edited Cell Therapy Improves Sickle Cell, Transfusion-Dependent Thalassemia Phenotypes

Rabi Hanna, MD, presented updated data from the RUBY and EdiThal trials at ASH 2023.

4. Veit Bücklein, Dr Med, on CAR-T Outcomes Between Male and Female Patients With LBCL

The postdoctoral researcher at Laboratory for Translational Cancer Immunology, Ludwig-Maximilians-Universität München, discussed the need for research into how CAR T-cells act differently between sexes.

5. More Work Will Be Needed to Harness the Full Potential of Patient Reported Outcomes Measurements

Akshay Sharma, MBBS, a bone marrow transplant physician at St. Jude Children’s Research Hospital, also gave his thoughts on the recent FDA approval of Vertex and CRISPR’s exa-cel for sickle cell disease.

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Ben Samelson-Jones, MD, PhD, assistant professor pediatric hematology, Perelman School of Medicine, University of Pennsylvania and Associate Director, Clinical In Vivo Gene Therapy, Children’s Hospital of Philadelphia
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Steven W. Pipe, MD, a professor of pediatric hematology/oncology at CS Mott Children’s Hospital
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
Chun-Yu Chen, PhD, a research scientist at Seattle Children’s Research Institute
Michael Severino on In Vivo Gene Editing With RNA Gene Writers
Chris Wright, MD, PhD, on Annelloviruses, a Potential Alternative to AAV for Gene Therapy
Carol Miao, PhD, a principal investigator at Seattle Children’s Research Institute
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