CGTLive’s Weekly Rewind – March 24, 2023

Welcome to CGTLive’s Weekly Rewind! This week’s coverage highlights the recent 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference the team covered live on site, through expert interviews and session coverage.

1. Death Prompts CAR-NKT Cell Therapy Hold On Neuroblastoma Trial

The patient was treated at the new fifth dose level and had concomitant metapneumovirus infection.

2. Courtney Young, PhD, on Gene Editing Approaches to Treating Neuromuscular Disease

The co-founder and chief executive officer of MyoGene Bio spoke about the company's gene editing therapy for Duchenne muscular dystrophy at MDA’s 2023 conference.

3. Sarepta’s SRP-9001 Shows Functional Improvements in DMD Integrated Analysis

Presented at MDA 2023, the gene therapy (also known as delandistrogene moxeparvovec) showed significant improvements on NSAA total score, 10-meter walk/run scores, and time to rise scores relative to an external control cohort.

4. Angela Genge, MD, FRCPC, eMBA, on Targeting STMN2 to Rescue Neurons in ALS

The chief medical officer of QurAlis discussed the antisense oligonucleotide therapy QRL-201.

5. Nusinersen Well-Tolerated After Zolgensma in SMA

Data from the phase 4 RESPOND trial were presented at the 2023 MDA Conference.