CGTLive’s Weekly Rewind – September 1, 2023


Review top news and interview highlights from the week ending September 1, 2023.

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. FDA Approves Bristol Myers Squibb’s Reblozyl for Expanded Indication in Myelodysplastic Syndromes

The FDA’s decision was based on data from the randomized, open-label phase 3 COMMANDS clinical trial.

2. Robert J. Hariri, MD, PhD, on the Unique Potential of Chimeric Therapy

The founder, chairman, and CEO of Celularity shared his view on the concept of “chimeric vigor” and the potential benefits arising from it in the cell therapy field.

3. BlueRock Therapeutics' Parkinson Disease Cell Therapy Bemdaneprocel Shows Initial Efficacy in Phase 1 Trial

In light of the promising results, BlueRock is preparing for a phase 2 trial.

4. Taking a Second Look at Placental-Derived Mesenchymal-Like Adherent Stromal Cell Therapy for Crohn Disease

Adrian Kilcoyne, MD, MPH, MBA, the chief medical officer of Celularity, discussed a new analysis of gene and protein signatures from patients treated in the company’s legacy clinical trials.

5. Investigational AAV8 Gene Therapy GNT0003 Shows Safety in Crigler–Najjar Syndrome

In a small study, administration of the Généthon therapy encoding UGT1A1 resulted in no serious adverse events. It is also being evaluated in a larger pivotal trial.

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George Tachas, PhD
Alexandra Gomez-Arteaga, MD
Pietro Genovese, PhD, the principal investigator at the Gene Therapy Program of Dana-Farber/Boston Children’s Cancer and Blood Disorder Center
Akshay Sharma, MBBS, a bone marrow transplant physician at St. Jude Children’s Research Hospital
Caspian Oliai, MD, MS, the medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center
Frederick “Eric” Arnold, PhD
Genovefa (Zenia) Papanicolaou, MD, an infectious diseases specialist at Memorial Sloan Kettering Cancer Center
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