Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.
Type:
Grant
Filed:
July 31, 2015
Date of Patent:
November 13, 2018
Assignees:
ALYNYLAM PHARMACEUTICALS, INC., ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
Inventors:
Brian Bettencourt, Kevin Fitzgerald, William Querbes, Robert J. Desnick, Makiko Yasuda
Abstract: The invention relates to an isolated RNA that mediates RNA interference of an mRNA to which it corresponds and a method of mediating RNA interference of mRNA of a gene in a cell or organism using the isolated RNA.
Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting one or more EGLN genes, EGLN1, EGLN2 and/or EGLN3 and methods of using such dsRNA compositions to inhibit expression of these genes.
Type:
Grant
Filed:
December 9, 2011
Date of Patent:
November 24, 2015
Assignees:
Alynylam Pharmaceuticals, Inc., Dana-Farber Cancer Institute
Inventors:
William G. Kaelin, Jr., Victor Kotelianski, William Querbes, Brian Bettencourt
Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Factor V Leiden mutant gene (Factor V Leiden mutant gene), comprising an antisense strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially complementary to at least a part of the Factor V Leiden mutant gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the Factor V Leiden mutant gene using the pharmaceutical composition; and methods for inhibiting the expression of the Factor V Leiden mutant gene in a cell.
Type:
Grant
Filed:
January 20, 2011
Date of Patent:
February 25, 2014
Assignee:
Alynylam Pharmaceuticals, Inc.
Inventors:
Hans-Peter Vornlocher, John M. Maraganore
Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Huntingtin gene (HD gene), comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the HD gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the HD gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.
Type:
Grant
Filed:
October 4, 2011
Date of Patent:
November 20, 2012
Assignee:
Alynylam Pharmaceuticals, Inc.
Inventors:
Dinah Wen-Yee Sah, Philipp Hadwiger, Ingo Roehl, Birgit Bramlage, Pamela Tan, Hans-Peter Vornlocher, David Bumcrot