Treatment of neuronal ceroid lipofuscinosis
AssigneeOrfoneuro Aps
InventorsJens Morten Fogh, Paul Saftig, Friederike Zunke, André Rosa Alcalde Marques
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Abstract
The invention provides for novel treatments of lysosomal storage diseases, in particular of Neuronal Ceroid Lipofuscinosis (NCL) or synucleinopathy or a disease characterized by block of autophagic flow. The treatment comprises administration of human pro-Cathepsin, in particular human pro-Cathpsin D, B or L to patients in need thereof.
Claims18
A method of therapeutic and/or prophylactic treatment of a human being for a Neuronal Ceroid Lipofuscinosis (NCL) or synucleinopathy or disease characterized by block of autophagic flow comprising administration to the human being a pharmaceutically effective and acceptable amount of a pro-cathepsin selected from the group consisting of pro-cathepsin D (proCTSD), pro-cathepsin B (proCTSB), and pro-cathepsin L (proCTSL), or any combination thereof,
- wherein said proCTSD comprises an amino acid sequence comprising a total of at most 20 amino acid substitutions and amino acid deletions compared SEQ ID NO:
- 3, and
- wherein said proCTSB comprises an amino acid sequence comprising a total of at most 20 amino acid substitutions and amino acid deletions compared to SEQ ID NO:
- 9, and
- wherein said proCTSL comprises an amino acid sequence comprising a total of at most 20 amino acid substitutions and amino acid deletions compared to SEQ ID NO:
- 14, and
- wherein said proCTSD or proCTSB or proCTSL is co-administered or formulated with an agent that facilitates passage of the proCTSD/proCTSB/proCTSL across the blood-brain barrier, or
- wherein the proCTSD/proCTSB/proCTSL is coupled to a moiety, which facilitates passage of the proCTSD/proCTSB/proCTSL across the blood-brain barrier.
Dependencies are inferred from the claim text.