CGTLive’s Weekly Rewind – August 12, 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. Zolgensma Gene Therapy Linked to 2 Deaths in SMA Patients, Novartis Reports

The 2 deaths, due to acute liver failure, occurred in patients treated in Kazakhstan and Russia.

2. Ide-Cel Shows Benefit in Earlier Use in R/R Multiple Myeloma

A recent comparison of health-related quality of life scores also favored ide-cel over belantamab mafodotin treatment.

3. UniQure Pauses High Dose of AMT-130 Huntington Disease Gene Therapy for Severe Adverse Events

No such adverse events have occurred in the lower-dose group.

4. First Patient Dosed in Sickle Cell Gene Therapy Clinical Trial From Graphite Bio

Nula-cel is intended to directly correct the mutation that causes sickle cell disease.

5. Frontotemporal Dementia Gene Therapy Trial Initiates Dosing

PBFT02, developed by Passage Bio, is delivered via intra cisterna magna administration to the brain.