UniQure from the Netherlands has promising results for its enzyme replacement therapy phase Ib/II trial targeting a rare Lysosomal disease. This has granted fast-track approval to launch its phase III trial for children with Sanfilippo Type B (MPS-III) in collaboration with France’s Institut Pasteur.
UniQure is the Amsterdam gene-therapy biotech with a lot going on. Last year they secured a eye-watering €1.1M prescription deal for their drug Glybera (which treats the 150-200 individuals in the EU with the ultra-rare lipoprotein lipase deficiency). Now they’ve developed a clinical-phase Ib/II gene therapy for another rare genetic disorder, Sanfilippo disease type B.
Sanfilippo (also known as Mucopolysaccharidosis III) is an autosomal recessive disease group characterized by enzyme deficiencies, affecting around 1 in 70,000 births in the EU. There are 4 subtypes (A-D) and Sanfilippo B, in particular, is the subtype resulting from the N-acetyl-alpha-D-glucosaminidase (NaGLU) enzyme. NaGLU hydrolyses particular complex sugars responsible for the break down of heparin sulfate.
If allowed to accumulate, heparin sulfates severely affect multiple tissues (e.g.