Dna compositions and related methods
AssigneeFlagship Pioneering Innovations Vii, LLC
InventorsAlexandra Rachael Sneider, Jacob Rosenblum Rubens, Narahari Subbanna Pujar, Molly Krisann Gibson, Benjamin Andrew Portney, Geoffrey A. von Maltzahn, Camilo Ayala Breton
Life of this patent
Estimating the term…
Abstract
The disclosure provides, for example, single stranded, covalently closed DNA that does not form a double stranded structure longer than 100 base pairs. The ssDNA may encode an effector sequence, for instance a therapeutic protein. The ssDNA may comprise a nuclear targeting sequence (NTS). In some embodiments, the ssDNA shows decreased activation of the innate immune system compared to an otherwise similar dsDNA.
Claims26
A pharmaceutical formulation comprising a lipid nanoparticle (LNP) comprising a single stranded DNA (ssDNA), wherein the ssDNA:
- (a) encodes a therapeutic protein,
- (b) is covalently closed,
- (c) does not comprise a region of intramolecular complementarity longer than 100 base pairs or does not form a double stranded structure longer than 100 base pairs,
- (d) is at least 1000 nucleotides in length,
- (e) does not comprise a protelomerase target sequence, and
- (f) comprises an EBV oriP site;
- wherein:
- (i) at least 95% by mass of total DNA in the pharmaceutical formulation is the ssDNA that is covalently closed, and
- (ii) the pharmaceutical formulation is free of viral protein.
Dependencies are inferred from the claim text.