GeneTherapyLive’s Weekly Rewind – December 9, 2021

Welcome to GeneTherapyLive’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. Importance of Screening and Early Treatment in Genetic Disorders: Paul Wuh-Liang Hwu, MD, PhD

The professor from National Taiwan University Hospital discussed the importance of early treatment in genetic disorders. He discussed gene therapy trials in AADC deficiency and Parkinson disease as well as newborn screening studies he is investigating in spinal muscular atrophy and Pompe disease.

2. CAR T-Cell Therapy Shows Efficacy and Safety in B-Cell Malignancies

Treatment with the chimeric antigen receptor (CAR) T-cell therapy ADI-001 elicited complete and near complete responses in patients with B-cell Non-Hodgkin’s Lymphoma, according to interim data from the phase 1 GLEAN study (NCT04735471) announced by Adicet Bio.

3. Reprogramming the Immune System for Kidney Transplant and Scleroderma

Scott Requadt, chief executive officer, Talaris Therapeutics, discussed the company’s goal to reprogram the immune system. He discussed the company’s approach and how the COVID-19 pandemic has exacerbated the unmet need in these indications.

4. Hemophilia B Gene Therapy Meets Primary End Points in HOPE-B Trial

The phase 3 HOPE-B trial (NCT03569891) of the investigational adeno-associated virus 5 gene therapy etranacogene dezaparvovec (uniQure; CSL Behring) has met its primary end point of reduction in annualized bleeding rate (ABR) post-treatment compared with baseline Factor IX prophylactic therapy in hemophilia B.

5. Gene Therapy for Sanfilippo Syndrome: Latest Updates

Ralph Laufer, PhD, chief scientific officer, Lysogene, discussed LYS-SAF302, its mechanism of action, and trial data. He also discussed further research being conducted with the therapy including a real-world experience video study.