Hematopoietic Recovery After Myeloablative Transplantation May Be Entirely Driven by Hematopoietic Stem Cells

Findings from a preclinical study, which are in contrast with the current biphasic recovery model, have implications for both HSC transplantation and gene therapy.

Yara Abdou, MD, on the Advantages of CAR Macrophages in Solid Tumors

The assistant professor of oncology at UNC School of Medicine discussed the first-in-human study being conducted with CT-0508.

Homology-Directed Repair-Developed TCR Therapy Shows Potential in Type 1 Diabetes

Investigators from Seattle Children’s Hospital found that engineered T-cell regulatory products demonstrated significant suppression of effector T cells.

Florian Eichler, MD, on How Novel Gene Therapy Delivery Improved Targeting in GM2 Gangliosidosis

The director of the leukodystrophy service at Mass Gen discussed the phase 1/2 study of AXO-AAV-GM2.

Final Analysis of Phase 3 HOPE-B Trial: Durability of Etranacogene Dezaparvovec and QoL Improvements

The trial met its primary efficacy end point, and participants reported improvements in quality-of-life factors such as outlook on the future and feelings about having hemophilia.

Lentiviral Gene Therapy for Infants With X-Linked SCID Helps Build ‘Functional’ Immune System

The interim study results presented at ASGCT 2022 likely represent the longest follow-up data to date for the largest cohort of infants with XSCID treated with lentiviral vector gene therapy.