CGTLive®’s Weekly Rewind – October 4, 2024

CGTLive®’s Weekly Rewind

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. Cilta-cel Boosts Overall Survival in New Early Line Multiple Myeloma Data

The data, from the CARTITUDE-4 clinical trial, compared outcomes to patients treated with standard of care options.

2. Arun Upadhyay, PhD, on Results from IRD Gene Therapy OCU400’s Phase 1/2 Trial

The chief scientific officer and head of research, development, and Medical at Ocugen, also discussed future plans for the gene therapy, which is now in a phase 3 study.

3. Anixa Biosciences Seeks Protocol Change to Enable Repeat Dosing in Trial for Ovarian Cancer CER-T

Anixa noted that it had previously obtained clearance of a single-patient investigational new drug application to redose a patient in the trial.

4. CRISPR-Mediated In Vivo Epigenomic Activation

Daniel Hart, PhD, the senior director and head of technology development at Epic Bio, discussed potential applications of the new technology.

5. Myrtelle’s Canavan Disease Gene Therapy rAAV-Olig001-ASPA Decreases N-Acetylaspartate Levels in Phase 1/2 Study

CSF analyses from 7 patients treated in the study showed reductions of more than 80% in NAA levels from baseline.