METHODS AND COMPOSITIONS FOR INHIBITING THE FUNCTION OF POLYNUCLEOTIDE SEQUENCES

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United States of America Patent

APP PUB NO 20160208261A1
SERIAL NO

14946223

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Abstract

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A therapeutic composition for inhibiting the function of a target polynucleotide sequence in a mammalian cell includes an agent that provides to a mammalian cell an at least partially double-stranded RNA molecule comprising a polynucleotide sequence of at least about 200 nucleotides in length, said polynucleotide sequence being substantially homologous to a target polynucleotide sequence. This RNA molecule desirably does not produce a functional protein. The agents useful in the composition can be RNA molecules made by enzymatic synthetic methods or chemical synthetic methods in vitro; or made in recombinant cultures of microorganisms and isolated therefrom, or alternatively, can be capable of generating the desired RNA molecule in vivo after delivery to the mammalian cell. In methods of treatment of prophylaxis of virus infections, other pathogenic infections or certain cancers, these compositions are administered in amounts effective to reduce or inhibit the function of the target polynucleotide sequence, which can be of pathogenic origin or produced in response to a tumor or other cancer, among other sources.

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Patent Owner(s)

Patent OwnerAddress
ALNYLAM PHARMACEUTICALS INC675 WEST KENDALL STREET HENRI A TERMEER SQUARE CAMBRIDGE MA 02142 02142

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Pachuk, Catherine J Cambridge, US 47 592
Satishchandran, Chandrasekhar Cambridge, US 36 157

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