RNA constructs and uses thereof
AssigneeBioNTech SE
InventorsUgur Sahin, Gábor Boros, Azita Josefine Mahiny, Jonas Reinholz, Katalin Karikó
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Abstract
Disclosed herein are RNA polynucleotides comprising a 5′ Cap, a 5′ UTR comprising a cap proximal sequence disclosed herein, and a sequence encoding a payload. Also disclosed herein are compositions and medical preparations comprising the same, and methods of making and using the same.
Claims22
A composition or medical preparation comprising an RNA polynucleotide comprising:
- (a) a 5′ cap comprising a Cap1 structure;
- (b) a cap proximal sequence comprising positions +1, +2, +3, +4, and +5 of the RNA polynucleotide;
- (c) a 5′ UTR sequence comprising the nucleotide sequence of SEQ ID NO:
- 27;
- (d) a sequence encoding a payload;
- (e) a 3′ UTR sequence comprising the nucleotide sequence of SEQ ID NO:
- 13;
- and
- (f) a polyA sequence comprising the nucleotide sequence of SEQ ID NO:
- 14;
- wherein:
- (i) the Cap1 structure comprises m7(3′ OMeG)(5′)ppp(5)(2′OMeA1)pG2, wherein A1 is position +1 of the RNA polynucleotide, and G2 is position +2 of the RNA polynucleotide;
- (ii) the cap proximal sequence comprises A1 and G2 of the Cap1 structure, and a sequence comprising:
- A3A4U5 (SEQ ID NO:
- 5) at positions +3, +4 and +5 respectively of the RNA polynucleotide;
- and
- (iii) the RNA polynucleotide optionally comprises a modified uridine in place of one or more uridines.
A pharmaceutical composition comprising an RNA polynucleotide formulated in lipid nanoparticles (LNPs) comprising a cationically ionizable lipid, a neutral lipid, a sterol, and a polymer-conjugated lipid, the RNA polynucleotide comprising:
- (a) a 5′ cap comprising a Cap1 structure;
- (b) a cap proximal sequence comprising positions +1, +2, +3, +4, and +5 of the RNA polynucleotide;
- (c) a 5′ UTR sequence comprising the nucleotide sequence of SEQ ID NO:
- 27;
- (d) a sequence encoding a payload;
- (e) a 3′ UTR sequence comprising the nucleotide sequence of SEQ ID NO:
- 13;
- and
- (f) a polyA sequence comprising the nucleotide sequence of SEQ ID NO:
- 14;
- wherein:
- (i) the Cap1 structure comprises m7(3′ OMeG)(5′)ppp(5)(2′OMeA1)pG2, wherein A1 is position +1 of the RNA polynucleotide, and G2 is position +2 of the RNA polynucleotide;
- (ii) the cap proximal sequence comprises A1 and G2 of the Cap1 structure, and a sequence comprising:
- A3A4U5 (SEQ ID NO:
- 5) at positions +3, +4 and +5 respectively of the RNA polynucleotide;
- and
- (iii) the RNA polynucleotide optionally comprises a modified uridine in place of one or more uridines.
Dependencies are inferred from the claim text.