• Metachromatic Leukodystrophy, commonly known as MLD, is a genetic disorder that affects the white matter, or myelin, of the brain and the central nervous system. The life expectancy is usually less than 10 years after diagnosis.
• MLD is an autosomal recessive genetic defect caused by mutations of the arylsulfatase A (ARSA) gene.
• There is no cure for MLD at present. Management of most MLD patients is usually limited to supportive care.

This invention provides an advanced hematopoietic stem cell (HSC)-based gene therapy to treat MLD, including the following:

• A modified version of lentivirus vector containing the human ARSA gene for MLD treatment
• Protocols of mass production of Lenti-ARSA for clinical application in compliance with GMP requirements
• Protocols for transduction of Lenti-ARSA into HSC
• Culturing medium and methods to maintain the multipotency of the transfected stem cells
• Clinical assessment system to determine disease severity subjected to HSC-gene therapy

Benefits

• Successful treatment for 9 patients including treatment for late-stage MLD cases which has not been achieved by other methods
• No obvious side effects
• No increased risk of tumorigenesis

Therapeutics for treating MLD

Stage of Development

• Clinically proven in 9 patients

Patents

• PCT Application No. PCT/CN2020/077088 filed on 28 Feb 2020

IP Status

• Patent application submitted