Poly(phosphoesters) for delivery of nucleic acids
AssigneeTranslate Bio, Inc.
InventorsFrank DeRosa, Michael Heartlein, Shrirang Karve, Yi Zhang
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Abstract
Disclosed are polymers comprising the moiety A, which is a moiety of formula I: and pharmaceutically acceptable salts thereof, wherein R, R1, R2, L, n1 and n2 are as defined herein. These polymers are useful for delivering nucleic acids to subject. These polymers and pharmaceutically acceptable compositions comprising such polymers and nucleic acids can be useful for treating various diseases, disorders and conditions.
Claims22
A polymer comprising a repeating unit that is
- or a pharmaceutically acceptable salt thereof, wherein:
- each instance of R1 independently is —H or a C1-C20 aliphatic group;
- each instance of R2 independently is —H or a C1-C20 aliphatic group;
- each instance of R3 independently is —H or a C1-C20 aliphatic group;
- X is independently S or O;
- n1 is an integer from 1 to 6;
- n2 is an integer from 1 to 6;
- R is N or CH;
- and
- L is C1-6 alkyl or a protonatable group provided that when R is CH then L is a protonatable group, wherein the protonatable group comprises at least one 1° amino, 2° amino, 3° amino or imidazolyl group.
A polymer comprising a repeating unit that is
- or a pharmaceutically acceptable salt thereof, wherein:
- each instance of R1 independently is —H or a C1-C20 aliphatic group;
- each instance of R2 independently is —H or a C1-C20 aliphatic group;
- each instance of R3 independently is —H or a C1-C20 aliphatic group;
- X is independently S or O;
- n2 is an integer from 1 to 6;
- R is N or CH;
- and
- L is C1-6 alkyl or a protonatable group provided that when R is CH then L is a protonatable group, wherein the protonatable group comprises at least one 1° amino, 2° amino, 3° amino or imidazolyl group.
A homopolymer comprising a repeating group selected from the group consisting of:
Dependencies are inferred from the claim text.