US 12,473,568 B2 · Non-immunogenic circular, non-viral DNA vectors

Abstract

The present disclosure relates to circular, non-viral DNA vectors, compositions including one or more of the disclosed vectors, and methods for delivering and/or expressing one or more therapeutic genes (e.g., proteins) in mammals, e.g., human patients. In some embodiments, the present disclosure is directed to circular, non-viral DNA vectors, such as circular non-viral DNA vectors including at least two inverted repeat sequences, where the at least two inverted repeat sequences are separated by a non-repeated nucleotide sequence which is not part of the at least two inverted repeat sequences. In some embodiments, the disclosed circular, non-viral DNA vectors do not include a “DD element.” In some embodiments, the disclosed circular, non-viral DNA vectors do not include a “DD element,” but include at least a portion of a bacterial origin of replication.

Claims27

Dependent claims

A pharmaceutical composition for administration to a human comprising:

(i) a circular, non-viral, non-integrating DNA vector comprising:
(a) a first portion comprising an expression cassette comprising a nucleic acid sequence encoding a therapeutic protein, wherein the nucleic acid sequence encoding the therapeutic protein is operably linked to a promoter;
and
(b) a second portion comprising a nucleic acid sequence comprising a first inverted repeat sequence, a bacterial origin of replication (Ori) sequence, and a second inverted repeat sequence;
wherein the first inverted repeat sequence, bacterial Ori sequence, and second inverted repeat sequence are contiguous, and wherein the Ori sequence is flanked by, and in between, the first inverted repeat sequence and the second inverted repeat sequence;
and
(ii) a lipid nanoparticle.

4 more elements

Dependencies are inferred from the claim text.

Non-immunogenic circular, non-viral DNA vectors

Life of this patent

Non-immunogenic circular, non-viral DNA vectors

Life of this patent

Abstract

Claims27