Nitto-denko Corporation

Richard P Witte

Victor Knopov

Violetta Akopian

Joseph E Payne

Mohammad Ahmadian

Yoshiro Niitsu

Yasunobu Tanaka

Loren A Perelman

John Gaudette

Zheng Hou

Priya Prakash Karmali

Wenbin Ying

Sridhar C Nagarajan

Elena Feinstein

Igor Mett

Hagar Kalinski

Kenjiro Minomi

Sharon Avkin-Nahum

Yun Liu

Priya Karmali

Jun Zhang

Lei Yu

David Webb

Robin Lee

Neda Safarzadeh

Yun Lui

C Nagarajan Sridhar

Sharon Avkin-nachum

John A Gaudette

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

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

What is described is a method for preparing a liposome that efficiently encapsulates a negatively charged therapeutic polymer, e.g., siRNA. The process involves preparing a lipid mixture comprising a cationic lipid in a water miscible organic solvent, such as ethanol, at a concentration of 2.3 mg/ml, and adding this solution to the polymer dissolved in water to a final concentration of 35% ethanol in water. The final charge ratio of drug:lipid is 1:2.5. The resulting nanoparticles have a mean size of 50 to 150 nm.

Method of producing lipid nanoparticles for drug delivery

Here described are compounds consisting of the structure

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.

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San Diego, CA, United States of America

Richard P Witte