IP Library Granted Patent US 9,850,269
Granted Patent B2
US 9,850,269 · App. 14/696,140 · Granted Dec 26, 2017

Methods for purification of messenger RNA

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Quick Facts
Patent No.
US 9,850,269
App. No.
14/696,140
Granted
Dec 26, 2017
Kind
B2
Abstract

The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of (a) precipitating mRNA from an impure preparation; (b) subjecting the impure preparation comprising precipitated mRNA to a purification process involving membrane filtration such that the precipitated mRNA is captured by a membrane; and (c) eluting the captured precipitated mRNA from the membrane by re-solubilizing the mRNA, thereby resulting in a purified mRNA solution. In some embodiments, a purification process involving membrane filtration suitable for the present invention is tangential flow filtration.

Claims (26)

1. A method of large scale purification of messenger RNA (mRNA), comprising:

providing an impure preparation comprising in vitro synthesized mRNA greater than 1 gram and prematurely aborted RNA sequences;

treating the impure preparation with a denaturing agent under conditions that facilitate separation of the prematurely aborted RNA sequences from the mRNA;

precipitating the mRNA from the treated impure preparation;

subjecting a heterogeneous suspension comprising the precipitated mRNA to tangential flow filtration such that the precipitated mRNA is captured by a filtration membrane; and

eluting the captured precipitated mRNA from the membrane by re-solubilizing the mRNA, thereby resulting in a purified mRNA solution.

2. The method of claim 1 , wherein the step of precipitating mRNA comprises treating the impure preparation with a solution comprising a reagent selected from the group consisting of lithium chloride, potassium chloride, guanidinium chloride, guanidinium thiocyanate, guanidinium isothiocyanate, and ammonium acetate, or a combination thereof.

3. The method of claim 1 , wherein the method further comprises a step of dialyzing the purified mRNA solution.

4. The method of claim 3 , wherein the purified mRNA solution is dialyzed with 1 mM sodium citrate using a 100 kDa molecular weight cut-off (MWCO) membrane.

5. The method of claim 1 , wherein the impure preparation comprises an in vitro mRNA synthesis reaction mixture.

6. The method of claim 5 , wherein the impure preparation further comprises enzyme reagents used in in vitro synthesis.

7. The method of claim 1 , wherein the purified mRNA solution contains less than 1% of prematurely aborted RNA sequences.

8. The method of claim 1 , wherein the in vitro synthesized mRNA is greater than 10 gram, 100 gram, 1 kg, 10 kg, or 100 kg of mRNA.

9. The method of claim 1 , wherein the mRNA is or greater than about 1 kb, 1.5 kb, 2 kb, 2.5 kb, 3 kb, 3.5 kb, 4 kb, 4.5 kb, 5 kb, 6 kb, 7 kb, 8 kb, 9 kb, 10 kb, 11 kb, 12 kb, 13 kb, 14 kb, 15 kb, or 20 kb in length.

10. The method of claim 1 , wherein the purified mRNA has an integrity of or greater than 95%.

11. A method for manufacturing messenger RNA (mRNA) comprising:

synthesizing mRNA in vitro; and

purifying the in vitro synthesized mRNA using a method according to claim 1 .

12. A purified mRNA composition comprising 5 grams or more of a single mRNA species purified using a method according to claim 1 and suitable for administration to a human subject.

13. A composition comprising messenger RNA purified using a method according to claim 1 .

14. A composition comprising in vitro synthesized messenger RNA, wherein the composition contains less than 1% of prematurely aborted RNA sequences and/or enzyme reagents used in in vitro synthesis.

15. The method of claim 1 , further comprising a step of adding a cap and/or a polyA tail to the mRNA in the purified solution, thereby producing polyA tailed and capped mRNA.

16. The method of claim 15 , further comprising a step of purifying the polyA tailed and capped mRNA.

17. The method of claim 10 , wherein the purified mRNA has the integrity of or greater than 96%.

18. The method of claim 10 , wherein the purified mRNA has the integrity of or greater than 98%.

19. The method of claim 10 , wherein the purified mRNA has the integrity of or greater than 99%.

Assignments (3)
CHANGE OF NAME Recorded Jul 20, 2017
From: RANA THERAPEUTICS, INC.
To: TRANSLATE BIO, INC.
Reel/Frame 043267/0165 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Apr 6, 2017
From: SHIRE HUMAN GENETIC THERAPIES, INC.
To: RANA THERAPEUTICS, INC.
Reel/Frame 042177/0032 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Sep 29, 2015
From: DEROSA, FRANK; DIAS, ANUSHA; KARVE, SHRIRANG; HEARTLEIN, MICHAEL
To: SHIRE HUMAN GENETIC THERAPIES, INC.
Reel/Frame 036681/0939 →