IP Library › Granted Patent US 11,407,775
Granted Patent B2
US 11,407,775 · App. 16/084,556 · Granted Aug 9, 2022

Compositions and methods for phosphoramidite and oligonucleotide synthesis

Inventors: David Charles Donnell Butler (Medford, MA); Subramanian Marappan (Acton, MA); Ik-Hyeon Paik (Needham, MA)
Assignee: WAVE LIFE SCIENCES LTD.
C07F9/65844C07H1/06C07H19/06C07H19/16
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Quick Facts
Patent No.
US 11,407,775
App. No.
16/084,556
Granted
Aug 9, 2022
Kind
B2
Abstract

The present disclosure, among other things, provides technologies for preparing and purifying phosphoramidites for oligonucleotide synthesis.

Claims (426)

1. A method for purifying a phosphoramidite, comprising steps of:

a) contacting a purification medium with a hygroscopic solvent system comprising at least 50% (v/v) acetonitrile or methanol;

b) contacting the purification medium with a phosphoramidite; and

c) using the purification medium to purify a phosphoramidite;

wherein the phosphoramidite has the structure of formula I,

wherein:

BA is R, or an optionally substituted group selected from a 3-30 membered cycloaliphatic ring, a 6-30 membered aryl ring, a 3-30 membered heterocyclyl having 1-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon, a 5-30 membered heteroaryl ring having 1-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon, a natural nucleobase moiety and a modified nucleobase moiety;

SU is a sugar moiety, a modified sugar moiety, -L-O— or

wherein SU is connected to the phosphorus atom in formula I through the oxygen atom;

L is a covalent bond, or a bivalent, optionally substituted, linear or branched group selected from C 1-30 aliphatic and C 1-30 heteroaliphatic group having 1-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon, wherein one or more methylene units are optionally and independently replaced by an optionally substituted C 1-6 alkylene, C 1-6 alkenylene, —C≡C—, —C(R′) 2 —, -Cy-, —O—, —S—, —S—S—, —N(R′)—, —C(O)—, —C(S)—, —C(NR′)—, —C(O)N(R′)—, —N(R′)C(O)N(R′)—, —N(R′)C(O)—, —N(R′)C(O)O—, —OC(O)N(R′)—, —S(O)—, —S(O) 2 —, —S(O) 2 N(R′)—, —N(R′)S(O) 2 —, —SC(O)—, —C(O)S—, —OC(O)—, or —C(O)O—;

R 5s is R′ or —OR′;

R 2s is —F, —CN, —N 3 , —NO, —NO 2 , —R′, —OR′, —SR′, —N(R′) 2 , -L-R′, —O-L-OR′, —O-L-SR′, or —O-L-N(R′) 2 , or R 2s is L connecting C2 with C1, C2, C3, C4 or C5;

-Cy- is an optionally substituted bivalent ring selected from 3-30 membered carbocyclylene, 6-30 membered arylene, 5-30 membered heteroarylene having 1-10 heteroatoms independently selected from oxygen, nitrogen and sulfur, and 3-30 membered heterocyclylene having 1-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon;

each of R 1 , R 2 , and R 3 is independently R′, or two or three of R 1 , R 2 , and R 3 are taken together with their intervening atoms to form:

Ring A is an optionally substituted multivalent, monocyclic, bicyclic or polycyclic, saturated, partially unsaturated, or aryl 3-30 membered ring having, in addition to the intervening atoms, 0-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon;

each R s is independently R′ or -L-R′;

t is 0-5;

each R′ is independently —R, —C(O)R, —CO 2 R, or —SO 2 R, or:

two or more R′ are taken together with their intervening atoms to form an optionally substituted monocyclic, bicyclic or polycyclic, saturated, partially unsaturated, or aryl 3-30 membered ring having, in addition to the intervening atoms, 0-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon; and

each R is independently hydrogen, or an optionally substituted group selected from C 1-30 aliphatic, C 1-30 heteroaliphatic having 1-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon, a 6-30 membered aryl ring, a 5-30 membered heteroaryl ring having 1-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon, and a 3-30 membered heterocyclic ring having 1-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon.

2. The method of claim 1 , wherein the hygroscopic solvent system consists of one hygroscopic solvent.

3. The method of claim 1 , wherein the hygroscopic solvent system comprises methanol.

4. The method of claim 1 , wherein the hygroscopic solvent system comprises acetonitrile.

5. The method of claim 4 , wherein the hygroscopic solvent system further comprises a modifier, wherein the modifier is trimethylamine up to 50% (v/v).

6. The method of claim 1 , wherein the hygroscopic solvent system is acetonitrile.

7. The method of claim 1 , wherein the acetonitrile contains no more than 0.5%, 0.4%, 0.3%, 0.2%, or 0.1% (v/v) water.

8. The method of claim 1 , wherein SU is

9. The method of claim 8 , wherein R 5S is —ODMTr.

10. The method of claim 8 , wherein R 2s is selected form —H, —F, —OMe, and —OCH 2 CH 2 OMe.

11. The method of claim 1 , wherein

wherein one or more R s is not hydrogen.

12. The method of claim 1 , wherein

13. The method of claim 1 , wherein

14. The method of claim 1 , wherein the phosphoramidite is selected from:

or is selected from:

R′ is H and R is H,

R′ is H, R is OMe,

R′ is H and R is OCH 2 CH 2 OMe,

R′ is H and R is F,

R′ is Me and R is H,

R′ is Me, R is OMe,

R′ is Me and R is OCH 2 CH 2 OMe, or

R′ is Me and R is F;

R′ is H and R is H,

R′ is H, R is OMe,

R′ is H and R is OCH 2 CH 2 OMe,

R′ is H and R is F,

R′ is Me and R is H,

R′ is Me, R is OMe,

R′ is Me and R is OCH 2 CH 2 OMe, or

R′ is Me and R is F;

R′ is H and R is H,

R′ is H, R is OMe,

R′ is H and R is OCH 2 CH 2 OMe,

R′ is H and R is F,

R′ is Me and R is H,

R′ is Me, R is OMe,

R′ is Me and R is OCH 2 CH 2 OMe, or

R′ is Me and R is F;

R′ is H and R is H,

R′ is H, R is OMe,

R′ is H and R is OCH 2 CH 2 OMe,

R′ is H and R is F,

R′ is Me and R is H,

R′ is Me, R is OMe,

R′ is Me and R is OCH 2 CH 2 OMe, or

R′ is Me and R is F;

R′ is Ac and R is H,

R′ is Ac, R is OMe,

R′ is Ac and R is OCH 2 CH 2 OMe,

R′ is Ac and R is F,

R′ is iBu and R is H,

R′ is iBu, R is OMe,

R′ is iBu and R is OCH 2 CH 2 OMe, or

R′ is iBu and R is F;

R′ is Ac and R is H,

R′ is Ac, R is OMe,

R′ is Ac and R is OCH 2 CH 2 OMe,

R′ is Ac and R is F,

R′ is iBu and R is H,

R′ is iBu, R is OMe,

R′ is iBu and R is OCH 2 CH 2 OMe, or

R′ is iBu and R is F;

R′ is Ac and R is H,

R′ is Ac, R is OMe,

R′ is Ac and R is OCH 2 CH 2 OMe,

R′ is Ac and R is F,

R′ is iBu and R is H,

R′ is iBu, R is OMe,

R′ is iBu and R is OCH 2 CH 2 OMe, or

R′ is iBu and R is F;

R′ is Ac and R is H,

R′ is Ac, R is OMe,

R′ is Ac and R is OCH 2 CH 2 OMe,

R′ is Ac and R is F,

R′ is iBu and R is H,

R′ is iBu, R is OMe,

R′ is iBu and R is OCH 2 CH 2 OMe, or

R′ is iBu and R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F; and

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F.

15. The method of claim 14 , wherein the phosphoramidite is selected from:

16. The method of claim 14 , wherein the phosphoramidite is selected from:

R′ is H and R is H,

R′ is H, R is OMe,

R′ is H and R is OCH 2 CH 2 OMe,

R′ is H and R is F,

R′ is Me and R is H,

R′ is Me, R is OMe,

R′ is Me and R is OCH 2 CH 2 OMe, or

R′ is Me and R is F;

R′ is H and R is H,

R′ is H, R is OMe,

R′ is H and R is OCH 2 CH 2 OMe,

R′ is H and R is F,

R′ is Me and R is H,

R′ is Me, R is OMe,

R′ is Me and R is OCH 2 CH 2 OMe, or

R′ is Me and R is F;

R′ is Ac and R is H,

R′ is Ac, R is OMe,

R′ is Ac and R is OCH 2 CH 2 OMe,

R′ is Ac and R is F,

R′ is iBu and R is H,

R′ is iBu, R is OMe,

R′ is iBu and R is OCH 2 CH 2 OMe, or

R′ is iBu and R is F;

R′ is Ac and R is H,

R′ is Ac, R is OMe,

R′ is Ac and R is OCH 2 CH 2 OMe,

R′ is Ac and R is F,

R′ is iBu and R is H,

R′ is iBu, R is OMe,

R′ is iBu and R is OCH 2 CH 2 OMe, or

R′ is iBu and R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F; and

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F.

17. The method of claim 14 , wherein the phosphoramidite is selected from:

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F; and

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F.

18. A method for preparing a phosphoramidite, comprising:

1) forming a phosphoramidite from a nucleoside and a chiral auxiliary; and

2) purifying the phosphoramidite obtained in step 1), comprising:

a) contacting a purification medium with a hygroscopic solvent system comprising at least 50% (v/v) acetonitrile or methanol;

b) contacting the purification medium with the phosphoramidite; and

c) using the purification medium to purify a phosphoramidite;

wherein the phosphoramidite has the structure of formula I,

wherein:

BA is R, or an optionally substituted group selected from a 3-30 membered cycloaliphatic ring, a 6-30 membered aryl ring, a 3-30 membered heterocyclyl having 1-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon, a 5-30 membered heteroaryl ring having 1-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon, a natural nucleobase moiety and a modified nucleobase moiety;

SU is

wherein SU is connected to the phosphorus atom in formula I through the oxygen atom;

L is a covalent bond, or a bivalent, optionally substituted, linear or branched group selected from C 1-30 aliphatic and C 1-30 heteroaliphatic group having 1-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon, wherein one or more methylene units are optionally and independently replaced by an optionally substituted C 1-6 alkylene, C 1-6 alkenylene, —C≡C—, —C(R′) 2 —, -Cy-, —O—, —S—, —S—S—, —N(R′)—, —C(O)—, —C(S)—, —C(NR′)—, —C(O)N(R′)—, —N(R′)C(O)N(R′)—, —N(R′)C(O)—, —N(R′)C(O)O—, —OC(O)N(R′)—, —S(O)—, —S(O) 2 —, —S(O) 2 N(R′)—, —N(R′)S(O) 2 —, —SC(O)—, —C(O)S—, —OC(O)—, or —C(O)O—;

R 5s is R′ or —OR′;

R 2s is —F, —CN, —N 3 , —NO, —NO 2 , —R′, —OR′, —SR′, —N(R′) 2 , -L-R′, —O-L-OR′, —O-L-SR′, or —O-L-N(R′) 2 , or R 2s is L connecting C2 with C1, C2, C3, C4 or C5;

-Cy- is an optionally substituted bivalent ring selected from 3-30 membered carbocyclylene, 6-30 membered arylene, 5-30 membered heteroarylene having 1-10 heteroatoms independently selected from oxygen, nitrogen and sulfur, and 3-30 membered heterocyclylene having 1-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon;

has the structure of

wherein one or more R s is not hydrogen;

each R s is independently R′ or -L-R′;

each R′ is independently —R, —C(O)R, —CO 2 R, or —SO 2 R, or:

two or more R′ are taken together with their intervening atoms to form an optionally substituted monocyclic, bicyclic or polycyclic, saturated, partially unsaturated, or aryl 3-30 membered ring having, in addition to the intervening atoms, 0-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon; and

each R is independently hydrogen, or an optionally substituted group selected from C 1-30 aliphatic, C 1-30 heteroaliphatic having 1-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon, a 6-30 membered aryl ring, a 5-30 membered heteroaryl ring having 1-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon, and a 3-30 membered heterocyclic ring having 1-10 heteroatoms independently selected from oxygen, nitrogen, sulfur, phosphorus and silicon.

19. The method of claim 18 , wherein

is

20. The method of claim 18 , wherein the

21. The method of claim 20 , comprising reacting

with a protected nucleoside.

22. The method of claim 18 , wherein the phosphoramidite is selected from:

or is selected from:

R′ is H and R is H,

R′ is H, R is OMe,

R′ is H and R is OCH 2 CH 2 OMe,

R′ is H and R is F,

R′ is Me and R is H,

R′ is Me, R is OMe,

R′ is Me and R is OCH 2 CH 2 OMe, or

R′ is Me and R is F;

wherein X is

and

R′ is H and R is H,

R′ is H, R is OMe,

R′ is H and R is OCH 2 CH 2 OMe,

R′ is H and R is F,

R′ is Me and R is H,

R′ is Me, R is OMe,

R′ is Me and R is OCH 2 CH 2 OMe, or

R′ is Me and R is F;

R′ is H and R is H,

R′ is H, R is OMe,

R′ is H and R is OCH 2 CH 2 OMe,

R′ is H and R is F,

R′ is Me and R is H,

R′ is Me, R is OMe,

R′ is Me and R is OCH 2 CH 2 OMe, or

R′ is Me and R is F;

R′ is H and R is H,

R′ is H, R is OMe,

R′ is H and R is OCH 2 CH 2 OMe,

R′ is H and R is F,

R′ is Me and R is H,

R′ is Me, R is OMe,

R′ is Me and R is OCH 2 CH 2 OMe, or

R′ is Me and R is F;

R′ is Ac and R is H,

R′ is Ac, R is OMe,

R′ is Ac and R is OCH 2 CH 2 OMe,

R′ is Ac and R is F,

R′ is iBu and R is H,

R′ is iBu, R is OMe,

R′ is iBu and R is OCH 2 CH 2 OMe, or

R′ is iBu and R is F;

R′ is Ac and R is H,

R′ is Ac, R is OMe,

R′ is Ac and R is OCH 2 CH 2 OMe,

R′ is Ac and R is F,

R′ is iBu and R is H,

R′ is iBu, R is OMe,

R′ is iBu and R is OCH 2 CH 2 OMe, or

R′ is iBu and R is F;

R′ is Ac and R is H,

R′ is Ac, R is OMe,

R′ is Ac and R is OCH 2 CH 2 OMe,

R′ is Ac and R is F,

R′ is iBu and R is H,

R′ is iBu, R is OMe,

R′ is iBu and R is OCH 2 CH 2 OMe, or

R′ is iBu and R is F;

R′ is Ac and R is H,

R′ is Ac, R is OMe,

R′ is Ac and R is OCH 2 CH 2 OMe,

R′ is Ac and R is F,

R′ is iBu and R is H,

R′ is iBu, R is OMe,

R′ is iBu and R is OCH 2 CH 2 OMe, or

R′ is iBu and R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F; and

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F.

23. The method of claim 18 , wherein the phosphoramidite is selected from:

R′ is H and R is H,

R′ is H, R is OMe,

R′ is H and R is OCH 2 CH 2 OMe,

R′ is H and R is F,

R′ is Me and R is H,

R′ is Me, R is OMe,

R′ is Me and R is OCH 2 CH 2 OMe, or

R′ is Me and R is F;

R′ is H and R is H,

R′ is H, R is OMe,

R′ is H and R is OCH 2 CH 2 OMe,

R′ is H and R is F,

R′ is Me and R is H,

R′ is Me, R is OMe,

R′ is Me and R is OCH 2 CH 2 OMe, or

R′ is Me and R is F;

R′ is Ac and R is H,

R′ is Ac, R is OMe,

R′ is Ac and R is OCH 2 CH 2 OMe,

R′ is Ac and R is F,

R′ is iBu and R is H,

R′ is iBu, R is OMe,

R′ is iBu and R is OCH 2 CH 2 OMe, or

R′ is iBu and R is F;

R′ is Ac and R is H,

R′ is Ac, R is OMe,

R′ is Ac and R is OCH 2 CH 2 OMe,

R′ is Ac and R is F,

R′ is iBu and R is H,

R′ is iBu, R is OMe,

R′ is iBu and R is OCH 2 CH 2 OMe, or

R′ is iBu and R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F; and

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F.

24. The method of claim 18 , wherein the phosphoramidite is selected from:

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F;

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F; and

R is H,

R is OMe,

R is OCH 2 CH 2 OMe, or

R is F.

25. The method of claim 1 , wherein

wherein one R s is hydrogen, and the other R s is -L-R′, wherein R′ is —SO 2 R and L is a bivalent, optionally substituted C 1-30 aliphatic group.

26. The method of claim 18 , wherein

wherein one R s is hydrogen, and the other R s is -L-R′, wherein R′ is —SO 2 R and L is a bivalent, optionally substituted C 1-30 aliphatic group.

Assignments (2)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Apr 12, 2022
From: PAIK, IK-HYEON
To: WAVE LIFE SCIENCES LTD.
Reel/Frame 059690/0106 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Dec 12, 2018
From: BUTLER, DAVID CHARLES DONNELL; MARAPPAN, SUBRAMANIAN
To: WAVE LIFE SCIENCES LTD.
Reel/Frame 047756/0239 →
Continuity (2)
Provisional Application 62307542 · Mar 13, 2016
Related Publication 20190077817A1 · Mar 14, 2019
Cited By (11)
US 12,391,942 US 12,403,156 US 12,435,105 US 12,473,321 US 12,486,505 US 12,552,743 US 12,583,883 US 12,590,115 US 12,637,672 US 12,674,168 US 12,735,703