Angela Mastronuzzi: Yet another impactful and giant step in clinical research
Angela Mastronuzzi shared on LinkedIn:
“Also out with The NEW ENGLAND JOURNAL of MEDICINE of the NEJM Group: Exagamglogene Autotemcel for Severe Sickle Cell Disease.
Part of the study was conducted at Ospedale Pediatrico Bambino Gesù, led by Franco Locatelli, and is yet another impactful and giant step in clinical research.
They conducted a phase 3, single-group, open-label study of exa-cel in patients 12 to 35 years of age with sickle cell disease who had had at least two severe vaso-occlusive crises in each of the 2 years before screening. CD34+ HSPCs were edited with the use of CRISPR-Cas9.
Before the exa-cel infusion, patients underwent myeloablative conditioning with pharmacokinetically dose-adjusted busulfan.
Treatment with exa-cel eliminated vaso-occlusive crises in 97% of patients with sickle cell disease for a period of 12 months or more.”
Source: Angela Mastronuzzi/LinkedIn
Angela Mastronuzzi is pediatric hematologist-oncologist and the Head of Neuro-Oncology Unit at Bambino Gesù Children’s Hospital (Italy). She is also a professor at the UniCamillus International Medical University, and a Board Member of AIEOP (Associazione Italiana Ematologia Oncologia Pediatrica).
Her main fields of interest are CNS tumors and innovative therapies. She is the author of more than 180 scientific publications in peer-reviewed international journals.