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US 2023/0310652 A1Published application
Vlp for the treatment of leukodystrophies
AssigneeNEUWAY Pharma GmbH
InventorsVictoria DEMINA, Oliver ERNST, Anke BONSE, Markus STAPF
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AssigneeNEUWAY Pharma GmbH
InventorsVictoria DEMINA, Oliver ERNST, Anke BONSE, Markus STAPF
Estimating the term…
The invention relates to virus like particles (VLP) associated with an enzyme abnormally expressed in particular leukodystrophies or an expression vector encoding the enzyme or an mRNA encoding the enzyme or a combination thereof which are used in a method for the treatment of the particular leukodystrophies in a subject in the need thereof, preferably a human. The invention also relates to a pharmaceutical composition for use in a method for the treatment of the particular leukodystrophies, to an expression vector encoding the abnormally expressed enzyme and to a method of associating a VLP with the enzyme, an expression vector encoding the enzyme or an mRNA encoding the enzyme or a combination thereof.
VLP associated with an enzyme or an expression vector encoding said enzyme or an mRNA encoding said enzyme or a combination thereof for use in a method for the treatment of a leukodystrophy in a subject, wherein the enzyme is aspartoacylase or galactocerebrosidase.
Expression vector having a coding region encoding an enzyme, a promoter selected from the group consisting of CAG and CMV, and having a size of less than 7 kb, preferably less than 6 kb, more preferably less than 5 kb, most preferably less than 4 kb, wherein the enzyme is aspartoacylase or galactocerebrosidase.
Method of associating a VLP with an enzyme, or an expression vector encoding an enzyme or an mRNA encoding an enzyme or a combination thereof, wherein the enzyme is aspartoacylase or galactocerebrosidase, and wherein the method comprises the following steps:
Dependencies are inferred from the claim text.