TPP1 PREPARATIONS AND METHODS OF TREATING CLN2 DISEASE Russian patent published in 2020 - IPC A61K9/08 A61K49/14 A61P25/28 

FIELD: medicine.

SUBSTANCE: group of inventions is intended for treating neuronal ceramide lipofuscinosis (CLN2). Preparation for treating CLN2-disease contains recombinant human tripeptidyl peptidase-1 (rhTPP1), wherein rhTPP1 comprises the amino acid sequence SEQ ID NO: 1 or its fragment. Said preparation contains rhTPP1 in concentration of 25 mg/ml to 35 mg/ml, potassium chloride in concentration of 0.01 mg/ml to 1 mg/ml, magnesium chloride hexahydrate in concentration of 0.01 mg/ml to 1 mg/ml and calcium chloride dihydrate in concentration of 0.01 mg/ml to 1 mg/ml, pH is 6.5. Said preparation is intended for preparing a drug for treating neuronal ceramide lipofuscinosis (CLN2). Also provided is a composition for treating a CLN2-disease, comprising a dose of rhTPP1, effective for maintaining the physiological function or slowing down or reducing the degree of deterioration of the physiological function in the individual, wherein the physiological function is a speech function, a motor function, vision or nutrition function, wherein rhTPP1 contains the amino acid sequence SEQ ID NO: 1 or its fragment. Said composition contains rhTPP1 in concentration of 25 mg/ml to 35 mg/ml, potassium chloride in concentration of 0.01 mg/ml to 1 mg/ml, magnesium chloride hexahydrate in concentration of 0.01 mg/ml to 1 mg/ml and calcium chloride dihydrate in concentration of 0.01 mg/ml to 1 mg/ml, and where pH of composition is 6.5.

EFFECT: use of the group of inventions provides effective arrest of the progression of CLN2 disease in treating subjects with CLN2 or in whom there is a family history of CLN2.

13 cl, 10 dwg, 6 tbl, 3 ex