GeneTherapyLive’s Weekly Rewind – February 11, 2022

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. Assessing EtranaDez for Hemophilia B in HOPE-B: Wolfgang Miesbach, MD, PhD

The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed results of the final analysis of the HOPE-B study. He gave an overview of the study, discussing the positive data and outcomes.

2. Stargardt Disease Gene Therapy IND Cleared to Proceed

Nanoscope Therapeutics has received approval from the FDA for its investigational new drug application for MCO-010, a gene therapy for the potential treatment of Stargardt disease, a type of macular degeneration.

3. The Importance of Collaboration in Developing Next Generation Cell Therapies

Dolores Schendel, PhD, chief executive and chief scientific officer, Medigene, discussed the company’s collaboration with 2seventy bio to develop TCR T-cell therapies as well as future collaborations and research that Medigene is pursuing.

4. CAR T-Cell Therapy Efficacious at 10 Years in Chronic Lymphocytic Leukemia

New findings, from a recent study evaluating 10-year follow-up of patients with complete responses to chronic lymphocytic leukemia (CLL) chimeric antigen receptor (CAR) T-cell treatment, have elucidated the long-term role of CD19+ CAR T-cells in the body.

5. Treating Solid Tumors With Personalized Immunotherapy: Julian Molina, MD, PhD

The hematologist/oncologist from Mayo Clinic discussed targeting HLA loss in solid tumors and developing personalized immunotherapies for solid tumors.