Tralesinidase Alfa Pre-Clinical Publications

Allievex obtained an exclusive worldwide license for tralesinidase alfa from BioMarin (BMN 250) in October 2019 for the treatment of patients with Sanfilippo syndrome type B. Review pre-clinical publications that support our clinical development program below.

Winner of the Best Paper Award, 2020 Drug Delivery and Translational Research Journal:

• Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B, Drug Delivery and Translational Research (2020)

Other Notable Publications:

• An exonic insertion in the NAGLU gene causing Mucopolysaccharidosis IIIB in Schipperke dogs, Scientific Reports (2020)

• Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B, Molecular Therapy: Methods & Clinical Development Vol. 14 (2019)

• BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different patterns of cellular uptake into critical cell types of Sanfilippo syndrome B disease pathogenesis, PLoS ONE (2019)

• Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice, Molecular Therapy: Methods & Clinical Development Vol. 6 (2017)

• Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB, PNAS Vol. 111 (2014)