US-based biopharmaceutical company Abeona Therapeutics has obtained the European regulatory approval for its Phase I/II gene therapy clinical trial of ABO-102 (AAV-SGSH) to treat Sanfilippo syndrome type A (MPS IIIA).
ABO-102 has been designed as a gene therapy based on an adeno-associated viral (AAV) indicated to be used to treat MPS IIIA (Sanfilippo syndrome).
It is delivered as a normal copy of the defective gene (SGSH) through an intravenous injection for a single time to deliver to the cells of the central nervous system (CNS) and peripheral organs, in order to correct the genetic errors triggering the disease.
The Phase I/II gene therapy clinical trial is planning to involve 25 patients with MPS III.
It has been designed to assess the neurocognitive evaluations, biochemical assays and MRI data produced by the gene therapy after observing the subjects for a year.
Spain's Cruces University Hospital department of paediatrics paediatrician Luis Aldámiz-Echevarría said: "Sanfilippo syndromes are devastating and progressive lysosomal storage diseases that affect children around the world.
“These gene therapies are delivered as a single, non-invasive intravenous injection and offer a new and promising treatment paradigm for patients with this relentless disease.
The preclinical trial earned the approval, during which a single dose of ABO-102 released cells in the CNS and peripheral organs to produce the missing enzyme, and also repaired the cell pathology that had caused the disease.
It also demonstrated safety and early biopotency signals in two low dose patients during an ongoing US trial.