Alnylam Pharmaceuticals, Inc.

Alnylam Europe Ag

Giuliani International Limited

Roland Kreutzer

Stefan Limmer

Hans-Peter Vornlocher

Matthias John

Philipp Hadwiger

Sylvia Limmer

Olaf Heidenreich

Anke Geick

Detlef Schuppan

Andreas Steinbrecher

Gerhard Giegerich

Ingo Kleiter

Markus Horn

Rainer Apfel

Matthias Ocker

Christoph Herold

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.

Method and medicament for inhibiting the expression of a given gene

The present invention relates to a double-stranded ribonucleic acid (dsRNA) having a nucleotide sequence which is substantially identical to at least a part of a target gene and which is no more than 49, preferably less than 25, nucleotides in length, and which comprises a complementary (antisense) RNA strand having a 1 to 4 nucleotide overhang at the 3'-end and a blunt 5&#x2032;-end. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target gene, as well as for treating diseases caused by expression of the target gene, at low dosages (i.e., less than 5 milligrams, preferably less than 25 micrograms, per kg body weight per day). The invention also relates to methods for inhibiting the expression of a target gene, as well as methods for treating diseases caused by the expression of the gene.

Compositions and methods for inhibiting expression of a target gene

The present invention relates to the specific inhibition of expression of a target gene in mammals using a short double stranded RNA. The dsRNA is less than 49 nucleotides in length and has a nucleotide sequence which is complementary to at least a part of the target gene. The dsRNAs of the present invention are useful for treating diseases, for example, cancer, viral diseases or neurodegenerative diseases.

The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a mutant gene, comprising a first strand that has a complementary region that is complementary to at least a portion of an RNA transcript of at least part of the mutant target gene and a second strand of the dsRNA complementary or substantially complementary to the first strand. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target mutant gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene.

Compositions and methods for inhibiting expression of a mutant gene

The present invention relates to a double-stranded ribonucleic acid (dsRNA) having a nucleotide sequence which is less that 30 nucleotides in length and which is substantially identical to at least a part of a 3'-untranslated region (3&#x2032;-UTR) of a (+) strand RNA virus, such as HCV, as well as pharmaceutical compositions comprising the dsRNA, together with a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for treating infections and diseases caused by the replication or activity of the (+) strand RNA virus, as well as methods for inhibiting viral replication.

Compositions and methods for inhibiting viral replication

The present invention provides methods and compositions for inhibiting gene expression using double stranded RNA molecules that are between 15 and 21 nucleotides in length and are complementary to a target gene sequence.

Method for making a 21 nucleotide double stranded RNA chemically linked at one end

The present invention provides methods and compositions for inhibiting gene expression using double stranded RNA molecules that are between 15 and 24 nucleotides in length and are complementary to a target gene sequence.

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Weidenberg, Germany

Roland Kreutzer