COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)

Title:

COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)

Document Type and Number:

WIPO Patent Application WO/2017/214518

Kind Code:

A8

Abstract:

The invention relates to methods for treating subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria, using iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and anti-C5 antibodies, e.g., eculizumab.

Inventors:

NAJAFIAN NADER (US)
KIM JAE (US)
VAROGLU MUSTAFA (US)
ROBBIE GABRIEL (US)

Application Number:

PCT/US2017/036775

Publication Date:

January 11, 2018

Filing Date:

June 09, 2017

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Assignee:

ALNYLAM PHARMACEUTICALS INC (US)

International Classes:

C12N15/113; A61K31/713; A61K39/395; C07H21/00

Attorney, Agent or Firm:

ZACHARAKIS, Maria Laccotripe et al. (US)

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