MODIFIED SULPHAMIDASE AND METHOD FOR PRODUCTION THEREOF Russian patent published in 2019 - IPC C12N9/14 C07K14/435 

FIELD: biotechnology.

SUBSTANCE: group of inventions relates to biotechnology. Modified sulfamidase comprises a polypeptide consisting of the amino acid sequence given in SEQ ID NO 1, or a polypeptide having at least 95 % sequence identity with the amino acid sequence given in SEQ ID NO 1. Disclosed sulphamidase essentially does not include epitopes recognized by glycan-recognizing receptors, wherein said epitopes are absent on at least four of five N-glycosylation sites: N in position 21 (N(21)), N in position 122 (N(122)), N in position 131 (N(131)), N in position 244 (N(244)) and N in position 393 (N(393)) of SEQ ID NO 1. Composition of sulphamidase for treating a mammal suffering from lysosomal accumulation disease includes said sulfamidase. Said sulfamidase has a C ratioα-formylglycine (FGly) to serine (Ser) on an active site is greater than 1. Method of producing modified sulphamidase involves steps of subjecting glycosylated sulphamidase to reaction with a periodate of an alkali metal and reacting with an alkali metal borohydride for a period of time of not more than 2 h to modify glycation groups of sulphamidase and reduced activity of sulphamidase towards receptors recognizing glycans, preserving its catalytic activity. Method of treating a mammal suffering lysosomal accumulation disease, such as IIIA (MPS IIIA), involves administering to a mammal a therapeutically effective amount of modified sulphonidase selected from said modified sulphonidase and said sulphonidase composition.

EFFECT: group of inventions provides penetration of said sulphamidase through a blood-brain barrier of a mammal and to exhibit catalytic activity in its brain.

31 cl, 18 dwg, 6 tbl, 11 ex