PRESS RELEASE
Preclinical insights into dosing for first-in-human in vivo liver-directed gene therapy for MMA delivered in an oral presentation at the ASGCT Annual Meeting
Milan, ITALY – 14 May 2025 – Genespire, a biotechnology company developing off-the-shelf gene therapies for pediatric patients affected by genetic diseases, presented positive preclinical insights into dosing for the first-in-human in vivo liver-directed gene therapy for methylmalonic acidemia (MMA) in an oral presentation late yesterday at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) taking place in Baltimore, MD, USA.
In the study presented at ASGCT, intravenous administration of an immune-shielded lentiviral vector (ISLV) encoding a human MUT transgene was…