IP Library › Granted Patent US 12,247,047
Granted Patent B2
US 12,247,047 · App. 18/792,393 · Granted Mar 11, 2025

Arnatar compounds and methods for enhanced cellular uptake

Inventors: Derek Steiner (Del Mar, CA); Xuehai Liang (Del Mar, CA); Yangfeng Wang (Carlsbad, CA)
Assignee: ARNATAR THERAPEUTICS, INC
C07H21/02A61K47/549C07H15/08
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Quick Facts
Patent No.
US 12,247,047
App. No.
18/792,393
Granted
Mar 11, 2025
Kind
B2
Abstract

This invention generally relates to the field of compounds and methods for enhanced cellular uptake. In particular, the invention relates to N-acetylgalactosamine compounds and their conjugates. Also provided are methods for the preparation of these molecules and possible uses thereof, in particular in medicine.

Claims (20)

1. A compound having the structure of formula (0a), (Ia) or (IIa):

Wherein

X is a hydroxy-protecting group or an H atom,

Y is a hydroxy-protecting group, an H atom, a payload, or a solid support,

Oligo represents a modified and/or unmodified oligonucleotide

R 1 is selected from any of hydroxy-protecting groups and H atom,

R 6 represents a hydroxy-protecting group, and

R 7 represents a moiety of formula (VI):

wherein in R7

R represents a solid support, an amino-protecting group, a hydroxy-protecting group,

or an H atom,

Z represents NH or O, and

R 8 represents a linear or branched alkylene having from 1 to 8 carbon atoms and/or a cycloalkylene having from 5 to 8 carbon atoms.

2. The compound of claim 1 , wherein when Y is a payload or a solid support it is connected to the compound via a spacer.

3. The compound of claim 1 , wherein in R 6 , the hydroxy-protecting group is a DMT or MMT.

4. The compound of claim 3 , wherein the hydroxy-protecting group of R 6 is MMT.

5. The compound of claim 1 , wherein when R represents a solid support, it is connected to the compound via a spacer.

6. The compound of claim 1 , wherein in R 8 one or more carbon atoms are substituted by an O, S, NH and/or N—(C 1 -C 3 alkyl) and/or a cycloalkylene having from 5 to 8 carbon atoms.

7. The compound of claim 6 , wherein the cycloalkylene having from 5 to 8 carbon atoms is any of ethylene, 1,1-dimethyl ethylene, propylene, n-butylene or 1,2-cyclohexylene.

8. A pharmaceutical composition comprising the compound of claim 1 and a pharmaceutically acceptable carrier, excipient, and/or diluent.

Continuity (5)
Continuation PCTUS2023084692 · Dec 18, 2023
Provisional Application 63433730 · Dec 19, 2022
Provisional Application 63533273 · Aug 17, 2023
Provisional Application 63602245 · Nov 22, 2023
Related Publication 20240417422A1 · Dec 19, 2024
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