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Lysogene’s most advanced gene therapy product candidate is LYS-SAF302 (international nonproprietary name [INN]: olenasufligene relduparvovec), currently the subject of AAVance, a global Phase II/III clinical trial for the treatment of Mucopolysaccharidosis type IIIA (MPS IIIA), or Sanfilippo syndrome type A.
LYS-SAF302 delivers a functional copy of the human SGSH gene directly to brain cells using the adeno-associated virus carrier, AAVrh.10, which has a particular tropism for cells of the CNS.
The treatment is administered through a one-time procedure comprising infusion at six intracerebral sites. By providing the functional version of SGSH gene, the treatment may provide a permanent source of functional enzyme in the brain. This in turn may help stabilize, or even reverse, the damage to patients’ brain cells.
MPS IIIA Gene Therapy
1- A normal SGSH gene is inserted into the AAVrh.10 virus
2- The gene therapy is administered to the child
3 – The gene therapy is taken into the brain cells
4- Gene therapy spreads to other brain cells, which will then be able to produce the enzyme
In February 2020, LYS-SAF302 received Fast Track designation by the US FDA. The program has received Orphan Drug Designations in the European Union (2014) and the US (2015) for MPS IIIA, and also has Rare Pediatric Disease Designation in the US.
VIDEO
Watch our video to learn more about the AAVance clinical study of LYS-SAF302 through one family’s journey with MPS IIIA and to find out more about the disease and procedure.
Mucopolysaccharidosis type IIIA
MPS IIIA is a rare inherited neurodegenerative lysosomal storage that primarily affects the brain of infants and young children. It is caused by mutations in the N-sulfoglucosamine sulfohydrolase (SGSH) gene that encodes the lysosomal enzyme heparan-N-sulfamidase. The primary function of this enzyme is to break down a substance called heparan sulfate in cells. Heparan sulfate regulates a range of important biological processes, but when it accumulates as a result of dysfunctional SGSH activity, this leads to progressive, severe neurodegeneration.
This damage to the nervous system results in delayed development, behavioral disturbances, seizures and a range of other cognitive and physical symptoms.
