US2021115101A1PendingUtilityA1
Modified polynucleotides for the production of proteins associated with human disease
Est. expiryApr 2, 2032(~5.7 yrs left)· nominal 20-yr term from priority
Inventors:Stephane BancelTirtha ChakrabortyAntonin De FougerollesSayda M. ElbashirMatthias JohnAtanu RoySusan WhoriskeyKristy M. WoodPaul HatalaJason P. SchrumKenechi EjebeJeff Lynn EllsworthJustin Guild
A61K 48/0066C07K 14/535A61K 48/0033C07K 14/4713C07K 14/745C07K 14/475C07K 14/47C12N 15/85C12N 2840/85C07K 14/4705A61K 38/1816C12N 9/93A61K 38/193C12P 21/005C07K 14/435C07K 14/565A61K 9/0019A61K 9/1272C12N 15/67A61K 38/363A61K 38/212C07K 14/005A61K 38/45C07K 14/75C12N 15/87A61K 31/7088C07K 14/56A61K 38/4846C12P 21/00C12N 15/88A61K 38/191A61K 47/543A61K 38/215C07K 14/505A61K 9/1271C12N 9/644C07K 16/32A61K 38/17C12Y 113/12007C12Y 603/02019C12N 9/2445C12N 15/11A61K 9/5031C07K 14/485A61K 38/177A61K 38/36C12P 13/04A61K 39/3955C07K 14/62A61K 48/005C12N 2840/00C07K 14/515C07K 14/705A61K 47/542C12N 9/6451A61K 31/7115C12Y 304/21022C07K 16/00A61K 48/00C12N 9/88C07K 14/525C12N 9/2402C12N 9/0069C07K 16/2887A61K 38/4833A61K 9/1277A61K 48/0075A61K 9/5146A61K 47/34A61K 38/1866A61K 38/1767C12N 15/52C12Y 304/21005A61K 47/10A61K 9/14C12N 9/1051A61K 47/54A61K 38/44A61K 9/145C07K 19/00A61K 38/1891C12N 9/16A61K 9/5153A61K 38/00A61K 9/5123
81
PatentIndex Score
0
Cited by
0
References
0
Claims
Abstract
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.
Claims
exact text as granted — not AI-modified1 - 19 . (canceled)
20 . A method of expressing a protein in a mammalian subject, the method comprising administering a composition comprising plurality of lipid nanoparticles having a mean particle size of between 80 nm and 160 nm, wherein the plurality of lipid nanoparticles encapsulate a polynucleotide comprising:
(a) an open reading frame encoding a polypeptide, wherein the open reading frame consists of modified uridine, cytidine, adenosine, and guanosine nucleotides; (b) a 5′-UTR; (c) a 5′ cap structure; (d) a 3′-UTR; and (e) a 3′ tailing sequence of linked nucleosides.
21 . The method of claim 20 , wherein the open reading frame consists of nucleotides selected from 1-methyl-pseudouridine, cytidine, adenosine, and guanosine.
22 . The method of claim 20 , wherein the plurality of lipid nanoparticles comprise a cationic lipid, a PEGlyated lipid, cholesterol, and a phospholipid.
23 . The method of claim 22 , wherein the cationic lipid is DLin-DMA, DLin-K-DMA, DLin-KC2-DMA, 98N12-5, C12-200, DLin-MC3-DMA, DODMA, DSDMA, or DLenDMA.
24 . The method of claim 20 , wherein the plurality of lipid nanoparticles in the pharmaceutical composition have a mean PDI of between 0.02 and 0.2, and a mean lipid to polynucleotide ratio (wt/wt) of between 10 and 20.
25 . The method of claim 20 , wherein the 3′-tailing sequence of linked nucleosides is selected from the group consisting of a poly-A tail of approximately 160 nucleotides and a polyA-G quartet.
26 . The method of claim 20 , wherein the at least one 5′ terminal cap is selected from the group consisting of Cap0, Cap1, ARCA, inosine, N1-methyl-guanosine, 2′fluoro-guanosine, 7-deaza-guanosine, 8-oxo-guanosine, 2-amino-guanosine, LNA-guanosine, and 2-azido-guanosine.
27 . The method of claim 20 , wherein the 3′-UTR comprises a miR binding site.
28 . The method of claim 20 , wherein the 5′-UTR comprises a Kozak sequence.Join the waitlist — get patent alerts
Track US2021115101A1 — get alerts on status changes and closely related new filings.
We store only your email — no account needed. See our privacy policy.