# US 11,999,683 B2 · Lipocationic dendrimers and uses thereof

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- Number: US 11,999,683 B2
- Document ID: US11999683B2
- Status: Granted
- Kind: B2
- Application number: 18/335,960
- Filed: 2023-06-15
- Published: 2024-02-08
- Granted: 2024-06-04
- Earliest claimed: 2015-09-14
- Assignees: Board of Regents, The University of Texas System
- Applicants: The Board of Regents of The University of Texas System
- Inventors: Daniel J. Siegwart; Kejin Zhou
- Web page: https://patentgrep.com/patent/US11999683B2

## CPC

- C07C 321/14: of an acyclic saturated carbon skeleton
- A61K 9/513: {Organic macromolecular compounds; Dendrimers}
- A61K 9/5146: {obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides}
- A61K 31/7105: Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
- A61K 31/7125: Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
- A61K 47/20: containing sulfur, e.g. dimethyl sulfoxide [DMSO], docusate, sodium lauryl sulfate or aminosulfonic acids
- A61K 47/22: Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
- A61K 47/59: obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
- A61K 47/60: the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
- A61K 47/6931: {the material constituting the nanoparticle being a polymer}
- A61K 48/0041: {the non-active part being polymeric}
- A61P 35/00: Antineoplastic agents
- A61P 37/08: Antiallergic agents (antiasthmatic agents A61P11/06; ophthalmic antiallergics A61P27/14)
- C07C 323/52: the carbon skeleton being acyclic and saturated
- C07D 295/15: to an acyclic saturated chain
- C07C: ACYCLIC OR CARBOCYCLIC COMPOUNDS
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- C07D: HETEROCYCLIC COMPOUNDS (macromolecular compounds C08)

## Abstract

Modular dendrimers with cationic groups and lipophilic groups are provided herein. In some aspects, the dendrimers provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.

## Claims

### Claim 1

A compound that is a compound of Formula (I):
Core-Repeating Unit-Terminating Group (I),
or a pharmaceutically acceptable salt thereof, wherein the core is linked to four repeating units and each repeating unit is linked to a nitrogen of the core, wherein the core has the following structure:
wherein each repeating unit is a degradable diacyl, having the formula
wherein in Formula (VII),
A1 and A2 are each —O—;
Y3 is alkanediyl(C≤12);
R9 is alkanediyl(C≤8); and
the terminating group is:
wherein Y4 is alkanediyli(C≤18) and R10 is H.

### Claim 2 (depends on 1)

The compound of claim 1, wherein the terminating group is selected from the group consisting of:

### Claim 3 (depends on 1)

The compound of claim 1, wherein the terminating group is selected from the group consisting of:

### Claim 4 (depends on 1)

The compound of claim 1, wherein the terminating group is:

### Claim 5 (depends on 1)

The compound of claim 1, wherein Y3 is —CH2CH2— and R9 is —CH3.

### Claim 6 (depends on 5)

The compound of claim 5, wherein the terminating group is selected from the group consisting of:

### Claim 7 (depends on 5)

The compound of claim 5, wherein the terminating group is selected from the group consisting of:

### Claim 8 (depends on 1)

The compound of claim 1, wherein the compound is the compound of Formula (I).

### Claim 9 (depends on 8)

The compound of claim 8, wherein the terminating group is selected from the group consisting of:

### Claim 10 (depends on 8)

The compound of claim 8, wherein the terminating group is selected from the group consisting of:

### Claim 11 (depends on 8)

The compound of claim 8, wherein the terminating group is:

### Claim 12 (depends on 8)

The compound of claim 8, wherein Y3 is —CH2CH2— and R9 is —CH3.

### Claim 13 (depends on 12)

The compound of claim 12, wherein the terminating group is selected from the group consisting of:

### Claim 14 (depends on 12)

The compound of claim 12, wherein the terminating group is selected from the group consisting of:

### Claim 15

A compound that is a compound of Formula (I):
Core-Repeating Unit-Terminating Group (I),
or a pharmaceutically acceptable salt thereof, wherein the core is linked to four repeating units and each repeating unit is linked to a nitrogen of the core, wherein the core has the following structure:
wherein the repeating unit is a degradable diacyl, having the formula:
wherein in Formula (VII),
A1 and A2 are each —O—;
Y3 is —CH2CH2—;
R9 is —CH3; and
the terminating group is:

### Claim 16 (depends on 15)

The compound of claim 15, wherein the terminating group is:

### Claim 17 (depends on 15)

The compound of claim 15, wherein the terminating group is:

### Claim 18

A compound of Formula (I):
Core-Repeating Unit-Terminating Group (I),
wherein the core is linked to four repeating units and each repeating unit is linked to a nitrogen of the core, wherein the core has the following structure:
wherein the repeating unit is a degradable diacyl, having the formula
wherein in Formula (VII),
A1 and A2 are each —O—;
Y3 is —CH2CH2—;
R9 is —CH3; and
the terminating group is:

### Claim 19 (depends on 18)

The compound of claim 18, wherein the terminating group is:

### Claim 20 (depends on 18)

The compound of claim 18, wherein the terminating group is: