IP Library › Granted Patent US 12,319,643
Granted Patent B2
US 12,319,643 · App. 17/658,075 · Granted Jun 3, 2025

Ionizable compounds and compositions and uses thereof

Inventors: Roger Adami (Carlsbad, CA); Hao Bai (San Diego, CA); John Gaudette (Poway, CA); Bharat Majeti (San Diego, CA); Seiji Nukui (San Diego, CA); Kwok Yin Tsang (Irvine, CA); Hai Wang (San Diego, CA); Haiqing Yin (San Marcos, CA); Wenbin Ying (San Diego, CA)
Assignee: Nitto Denko Corporation
C07C271/16A61K9/127A61K9/5123A61K31/7105A61K31/713A61K47/16A61K47/22A61K47/24C07C237/08C07C237/16C07C237/50C07D205/04C07D207/09C07D207/12C07D211/74C07D211/82C07D211/86C07F9/06C07F9/106
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Quick Facts
Patent No.
US 12,319,643
App. No.
17/658,075
Granted
Jun 3, 2025
Kind
B2
Abstract

Ionizable compounds, and compositions and methods of use thereof. The ionizable compounds can be used for making nanoparticle compositions for use in biopharmaceuticals and therapeutics. More particularly, the compounds, compositions and methods are to provide nanoparticles to encapsulate active agents, such as nucleic acid agents, and to deliver and distribute the active agents to cells, tissues, organs, and subjects.

Claims (24)

1. A compound comprising the structure shown in Formula IV

wherein R 1 and R 2 are

R 1 ═C(═O)OR 4

R 2 ═C(═O)OR 5

wherein R 4 and R 5 are independently for each occurrence a C(12-20) alkyl group, or a C(12-20) alkenyl group:

wherein R 3 is selected from

wherein

each R 6 is independently selected from H, alkyl, hydroxyl, hydroxyalkyl, alkoxy, alkoxyalkoxy, and aminoalkyl;

each R 7 is independently selected from H, alkyl, hydroxyalkyl, and aminoalkyl;

each R 8 is independently selected from H, alkyl, hydroxyalkyl, and aminoalkyl, and any two R 8 may form a ring;

q is from zero to four;

Q is O or NR 7 ;

p is from 1 to 4.

2. The compound of claim 1 , having a structure selected from

3. A composition comprising an ionizable compound of claim 1 and a pharmaceutically acceptable carrier.

4. The composition of claim 3 , wherein the composition comprises nanoparticles.

5. A pharmaceutical composition comprising an ionizable compound of claim 1 , an active agent, and a pharmaceutically acceptable carrier.

6. The composition of claim 5 , wherein the ionizable compound is from 15 mol % to 40 mol % of the lipids of the composition.

7. The composition of claim 5 , wherein the composition comprises nanoparticles.

8. The composition of claim 5 , wherein the active agent is one or more RNAi molecules.

9. The composition of claim 5 , wherein the active agent is one or more RNAi molecules selected from small interfering RNAs (siRNA), double stranded RNAs (dsRNA) that are Dicer substrates, microRNAs (miRNA), short hairpin RNAs (shRNA), DNA-directed RNAs (ddRNA), Piwi-interacting RNAs (piRNA), repeat associated siRNAs (rasiRNA), and modified forms thereof.

10. A composition for use in distributing an active agent for treating a condition or disease in a subject, the composition comprising an ionizable compound of claim 1 , a structural lipid, a stabilizer lipid, and a lipid for reducing immunogenicity of the composition.

11. The composition of claim 10 , wherein the active agent is one or more RNAi molecules and the composition comprises nanoparticles that encapsulate the RNAi molecules.

12. The composition of claim 11 , wherein the active agent is one or more RNAi molecules selected from small interfering RNAs (siRNA), double stranded RNAs (dsRNA) that are Dicer substrates, microRNAs (miRNA), short hairpin RNAs (shRNA), DNA-directed RNAs (ddRNA), Piwi-interacting RNAs (piRNA), repeat associated siRNAs (rasiRNA), and any modified forms thereof.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Dec 19, 2024
From: ADAMI, ROGER; BAI, HAO; GAUDETTE, JOHN; MAJETI, BHARAT; NUKUI, SEIJI; TSANG, KWOK YIN; WANG, HAI; YIN, HAIQING; YING, WENBIN
To: NITTO DENKO CORPORATION
Reel/Frame 069644/0620 →
Continuity (4)
Continuation 16228054 · Dec 20, 2018
Continuation 15191511 · Jun 23, 2016
Provisional Application 62184188 · Jun 24, 2015
Related Publication 20220242820A1 · Aug 4, 2022
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