Findings from a preclinical study, which are in contrast with the current biphasic recovery model, have implications for both HSC transplantation and gene therapy.
The assistant professor of oncology at UNC School of Medicine discussed the first-in-human study being conducted with CT-0508.
Investigators from Seattle Children’s Hospital found that engineered T-cell regulatory products demonstrated significant suppression of effector T cells.
The director of the leukodystrophy service at Mass Gen discussed the phase 1/2 study of AXO-AAV-GM2.
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.
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.