CGTLive®’s Weekly Rewind – March 1, 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.
CGTLive®'s Weekly Rewind
1. mHA-Specific Donor Tregs Show Potential in Preventing GvHD in Donor HCT
The expanded donor Treg clones maintained lineage fidelity and were persistent through 1 year post-HCT.
2. Paul Harmatz, MD, on Harnessing Accelerated Approval for LSDs, Rare Disease Treatment Advancements
The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed challenges in investigating therapies for rare diseases.
3. First Patient With Esophageal Cancer Receives TAC101-CLDN18.2 Cell Therapy
The patient is doing well so far and may receive a booster dose of the TAC cell therapy.
4. Paul Melmeyer, MPP, on Advocacy and Progress in Rare Neuromuscular Diseases From a Policy Standpoint
In observance of Rare Disease Day 2024, the vice president of public policy and advocacy at the Muscular Dystrophy Association discussed advocacy and policy progress regarding rare neuromuscular diseases.
5. Latest News in Cell and Gene Therapy for Rare Disease Day 2024
In observance of Rare Disease Day, held this year on February 29, catch up on some of the latest data updates from clinical trials for rare diseases.
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Jeffrey Chamberlain, PhD, on the Import of Early Intervention in DMD
July 10th 2025The McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed how comprehensive care for DMD patients involves early diagnosis, steroid treatment, consideration of mutation-specific therapies, and more.
