Autologous lentiviral ARSA gene therapy

Unassigned

New Medicines

Pre-symptomatic early onset (late infantile and early juvenile) metachromatic leukodystrophy (MLD)

Information

Advanced therapy medicinal product (ATMP)
Orchard Therapeutics
Orchard Therapeutics

Development and Regulatory status

None
Phase III Clinical Trials
None
Yes

Category

Autologous CD34+ hematopoietic stem cells transduced ex vivo with lentiviral vector encoding for the human ARSA gene. Given as a single infusion.
MLD is a rare and life threatening metabolic disorder caused by a mutation in the arylsulfatase-A (ARSA) gene [1]. This leads to a deficiency in the ARSA enzyme, the accumulation of sulfatides and the progressive destruction of the myelin sheath in nerve cells. UK incidence is approximately 1 in 40,000 [2].
Pre-symptomatic early onset (late infantile and early juvenile) metachromatic leukodystrophy (MLD)
Intravenous infusion