Clinically Proven Gene Therapy for Late-Stage MLD Treatment
Hematopoietic Stem Cell-Based Gene Therapy for Metachromatic Leukodystrophy (MLD)
Tags: The University of Hong Kong, Hong Kong, Healthcare & Lifesciences
This hematopoietic stem cell (HSC)-based gene therapy offers a breakthrough treatment for Metachromatic Leukodystrophy (MLD), a fatal genetic disorder caused by ARSA gene mutations. Using a modified lentivirus vector to deliver the human ARSA gene, the therapy has shown clinical success in treating nine patients, including late-stage cases, with no significant side effects or tumorigenesis risks. The technology also includes GMP-compliant protocols for mass production, transduction, and maintenance of multipotent stem cells. Applications extend to therapeutics for MLD, offering hope where only supportive care existed. A patent application (PCT/CN2020/077088) has been filed to secure its intellectual property.
IP Type or Form Factor: Patent Pending; Process & Method; Platform
TRL: 7 - product operates in intended environment with industrial standards
Industry or Tech Area: Biotechnology; Pharmaceutical Engineering
