Nitto-denko Corporation

Mohammad Ahmadian

Joseph E Payne

Richard P Witte

Victor Knopov

Violetta Akopian

Yoshiro Niitsu

Yasunobu Tanaka

Loren A Perelman

John Gaudette

Zheng Hou

Wenbin Ying

Priya Prakash Karmali

Jun Zhang

Sridhar C Nagarajan

Elena Feinstein

Igor Mett

Hagar Kalinski

Kenjiro Minomi

Sharon Avkin-Nahum

Yun Liu

Kenjirou Minomi

Jens Harborth

Hirokazu Takahashi

Erika Terada

Lei Yu

Neda Safarzadeh

Sharon Avkin-nachum

John A Gaudette

C Nagarajan Sridhar

Yun Lui

This invention provides compounds, compositions and methods for modulating the expression of human GST-&#x3c0; using RNA interference. The RNA interference molecules can be used in methods for preventing or treating diseases such as malignant tumor. Provided are a range of siRNA structures, having one or more of nucleotides being modified or chemically-modified. Advantageous structures include siRNAs with 2'-deoxy nucleotides located in the seed region, as well as other nucleotide modifications.

RNA interference agents for GST-PI gene modulation

The description is directed to ionizable lipids useful for enhancing the delivery of therapeutic agents in liposomes.

Lipids for therapeutic agent delivery formulations

Here described is a composition comprising a compound of formula I: wherein Rand Ris independently selected from a group consisting of Cto Calkyl, Cto Calkenyl, and oleoyl group; Rand Rare independently selected from a group consisting of Cto Calkyl, and Cto Calkanol; X is selected from a group consisting of &#x2014;CH&#x2014;, &#x2014;S&#x2014;, and &#x2014;O&#x2014; or absent; Y is selected from &#x2014;(CH), &#x2014;S(CH), &#x2014;O(CH)&#x2014;, thiophene, &#x2014;SO(CH)&#x2014;, and ester; n=1-4; a=1-4; b=1-4; c=1-4; and Zis a counterion.

Cationic lipids for therapeutic agent delivery formulations

RNA interference agents for GST-&#x3c0; gene modulation

Here described are compounds consisting of the structure(targeting molecule)-linker-(targeting molecule),wherein the targeting molecule is a retinoid or a fat soluble vitamin having a specific receptor on the target cell; wherein m and n are independently 0, 1, 2 or 3; and wherein the linker comprises a polyethylene glycol (PEG) or PEG-like molecule, as well as compositions and pharmaceutical formulations including these compounds which are useful for the targeting and delivery of therapeutic agents; and methods of using these compositions and pharmaceutical formulations.

Compounds for targeting drug delivery and enhancing siRNA activity

Here described is a method of delivery of a drug to a stellate cell using a composition comprising a compound of formula I: wherein Rand Rare independently selected from Cto Calkyl, Cto Calkenyl, and oleoyl; Rand Rare independently selected from Cto Calkyl and Cto Calkanol; X is selected from &#x2014;CH&#x2014;, &#x2014;S&#x2014;, and &#x2014;O&#x2014;, or X is absent; Y is selected from &#x2014;(CH), &#x2014;S(CH)&#x2014;, &#x2014;O(CH)&#x2014;, -thiophene-, &#x2014;SO(CH)&#x2014;, and ester; n&#x2550;1-4; a&#x2550;1-4; b&#x2550;1-4; c&#x2550;1-4; and Zis a counterion.

What is described is a method for treating a fibrotic disease by administering a pharmaceutical composition comprising a drug carrier, which comprises a lipid and a retinoid, and a double-stranded nucleic acid molecule, which comprises an antisense sequence to mRNA encoding human hsp47.

Method for treating fibrosis using siRNA and a retinoid-lipid drug carrier

What is described is a pharmaceutical composition for treating a fibrotic disease comprising a drug carrier, which comprises a lipid and a retinoid, and a double-stranded nucleic acid molecule, which comprises an antisense sequence to mRNA encoding human hsp47.

Retinoid-lipid drug carrier

Here described are compounds consisting of the structure

This invention provides compounds, compositions and methods for modulating the expression of human GST-&#x3c0; using RNA interference. The RNA interference molecules can be used in methods for preventing or treating diseases such as malignant tumor. A nucleic acid molecule can have a) a polynucleotide sense strand and a polynucleotide antisense strand; b) each strand of the molecule being from 15 to 30 nucleotides in length; c) a contiguous region of from 15 to 30 nucleotides of the antisense strand being complementary to a sequence of an mRNA encoding GST-&#x3c0;; and d) at least a portion of the sense strand can be complementary to at least a portion of the antisense strand, and the molecule has a duplex region of from 15 to 30 nucleotides in length.

RNA agents for GST-Pi gene modulation

Here described are compounds of formula I: wherein Rand Rare independently selected from Cto Calkyl, Cto Calkenyl, and oleoyl; Rand Rare independently selected from Cto Calkyl and Cto Calkanol; X is selected from &#x2014;CH&#x2014;, &#x2014;S&#x2014;, and &#x2014;O&#x2014;, or X is absent; Y is selected from &#x2014;(CH), &#x2014;S(CH)&#x2014;, &#x2014;O(CH)&#x2014;, -thiophene-, &#x2014;SO(CH)&#x2014;, and ester; n=1-4; a=1-4; b=1-4; c=1-4; and Zis a counterion. Also described herein are compositions and pharmaceutical formulations including compounds of formula I which are useful for the delivery of therapeutic agents, and methods of using these compositions and formulations.

Growing community of inventors

Carlsbad, CA, United States of America

Mohammad Ahmadian