COMPOSITIONS AND METHODS FOR MODULATING COMPLEMENT FACTOR EXPRESSION B Russian patent published in 2019 - IPC C12N5/00 A61K48/00 

Abstract RU 2701645 C2

FIELD: biotechnology.

SUBSTANCE: invention relates to the field of biotechnology. Described is a group of inventions comprising a compound for inhibiting complement factor B (CFB) expression (embodiments), a pharmaceutical composition for treating a disease associated with deregulation of an alternative complement pathway in a subject, a method of treating a disease associated with deregulation of an alternative complement pathway in a subject, use of said compound or composition for treating a disease associated with dysregulation of an alternative complement path, using said compound or composition when preparing a drug for treating a disease associated with deregulation of an alternative complement pathway. In one embodiment, the compound contains a modified oligonucleotide and a conjugate group.

EFFECT: invention extends the range of agents for inhibiting complement factor B (CFB) expression.

42 cl, 232 tbl, 135 ex

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RU 2 701 645 C2

Authors

Prakash Thazha P.

Seth Punit P.

Swayze Eric E.

Grossman Tamar R.

Mccaleb Michael L.

Watt Andrew T.

Freier Susan M.

Dates

2019-09-30Published

2015-05-01Filed