CGTLive’s Weekly Rewind – January 12, 2024

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Review top news and interview highlights from the week ending January 12, 2024.

CGTLive’s Weekly Rewind – January 12, 2024

CGTLive’s Weekly Rewind – January 12, 2024

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. FDA Activity Recap: December 2023 Features 2 Major Approvals, Meetings, and Clinical Holds

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive™ team.

2. Mazyar Shadman, MD, on Investigating Treatment Choices With CAR-T and Auto-HSCT in LBCL

The associate professor at Fred Hutch Cancer Center discussed data from a retrospective analysis of patients treated with tisa-cel, axi-cel, or liso-cel.

3. Myasthenia Gravis mRNA CAR-T Demonstrates Clinically Meaningful Improvements in Phase 2a Trial

In terms of safety, Descartes-08 was characterized as well-tolerated during the study and long-term follow-up period.

4. Tami John, MD, on the FDA Approvals of Exa-cel and Lovo-cel

The clinical associate professor at Stanford Medicine also discussed ongoing trends in sickle cell disease research.

5. Vertex and CRISPR’s Ongoing Trial for Sickle Cell Disease Gene Therapy Seeks to Gather Patient Reported Outcomes

Exa-cel was recently approved for the treatment of SCD in the US, but data from the pivotal clinical trial continues to be collected.

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Chun-Yu Chen, PhD, a research scientist at Seattle Children’s Research Institute
William Chou, MD, on Targeting Progranulin With Gene Therapy for Frontotemporal Dementia
Alexandra Collin de l’Hortet, PhD, the head of therapeutics at Epic Bio
Joshua M. Hare, MD, on Working to Address Unmet Needs in Alzheimer Disease With Lomecel-B Cell Therapy
William Chou, MD, on Expanding Frontotemporal Dementia Gene Therapy to Both GRN and C9orf72 Mutations
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
Joshua M. Hare, MD, on Promising Data on Lomecel-B for Alzheimer Disease
Carol Miao, PhD, a principal investigator at Seattle Children’s Research Institute
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