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Hematopoietic Stem Cell-Based Gene Therapy for Metachromatic Leukodystrophy (MLD)

Hematopoietic Stem Cell-Based Gene Therapy for Metachromatic Leukodystrophy (MLD)
Therapeutic Biologics

Key Problem and Market Opportunity

  • 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.

Key Advantages of the Technology

Technology Section Slider 1

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


  • 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

Potential Product and Services

Therapeutics for treating MLD

Development Status and IP Strength

Stage of Development
  • Clinically proven in 9 patients
  • PCT Application No. PCT/CN2020/077088 filed on 28 Feb 2020
IP Status
  • Patent application submitted

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