Therapeutic circular DNA forms
AssigneeFlagship Pioneering Innovations Vii, LLC
InventorsEdward Matthew Kennedy, Camilo Ayala Breton, Carl Wayne Brown, III, Brian Patrick Mead
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Abstract
The disclosure provides, for example, double stranded DNA (dsDNA) molecules comprising one or more chemically modified nucleobases. In some embodiments, the dsDNA molecule is circular and comprises a first strand and a second strand, wherein the first strand comprises one or more chemically modified nucleobases, and the second strand is free of chemically modified nucleobases. In some embodiments, the dsDNA molecule comprises a promoter sequence and an effector sequence that encodes an effector.
Claims22
A pharmaceutical composition comprising a lipid nanoparticle (LNP) that comprises a double stranded DNA (dsDNA) molecule, wherein:
- (a) the dsDNA molecule is circular;
- (b) the dsDNA molecule comprises a first strand and a second strand, wherein:
- at least 50% of thymine or uracil positions in the first strand of the dsDNA molecule comprise 5-hydroxymethyluracil, and
- the second strand is free of chemically modified nucleobases;
- and
- (c) the dsDNA molecule comprises an effector sequence that encodes an effector, wherein the effector is selected from the group consisting of:
- a transcription factor, a chromatin remodeling factor, an antigen, a peptide, a hormone, an enzyme, an antibody, a receptor ligand, a receptor, a clotting factor, and a membrane protein.
Dependencies are inferred from the claim text.