CGTLive’s Weekly Rewind – September 23, 2022

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. Eli-Cel Approved for CALD, Marking Second Gene Therapy Win for bluebird bio

The approval follows unanimous support of eli-cel's benefits in CALD from an FDA AdComm meeting and an approval for beti-cel in beta-thalassemia.

2. Roberto Giugliani, MD, PhD, on Reducing GAGs in MPS Type 2 With Gene Therapy

The professor of genetics at Federal University of Rio Grande do Sul discussed updated data from the CAMPSIITE trial.

3. Zevor-Cel Yields 100% ORR in R/R Multiple Myeloma

Eleven patients in the LUMMICAR-2 trial were evaluable for preliminary efficacy analyses.

4. David S. Hong, MD, on the SURPASS Clinical Trial and TCR T-cell Therapy in Solid Tumors

Hong presented data from the SURPASS clinical trial at the 2022 European Society for Medical Oncology (ESMO) Congress.

5. Intellia Nets Positive Data Across In Vivo CRISPR Therapies for ATTR Amyloidosis Cardiomyopathy, Angioedema

Administration of NTLA-2001 led to rapid and deep reductions in serum TTR by day 28.