Anellovirus compositions and methods of use
AssigneeFlagship Pioneering Innovations V, Inc.
InventorsErica Gabrielle Weinstein, Avak Kahvejian, Simon Delagrave, Nathan Lawrence Yozwiak, Kevin James Lebo, Fernando Martin Diaz, Dhananjay Maniklal Nawandar, Ryan D. Tedstone, Jared David Pitts
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Abstract
This invention relates generally to viral vectors and viral particles based on Anelloviruses, which can be used to deliver an agent (e.g., an exogenous effector or an endogenous effector, e.g., a therapeutic effector) to a cell (e.g., a cell in a subject to be treated therapeutically). Described herein are anellosomes, anellovectors, and compositions and uses thereof.
Claims66
A method of delivering a therapeutic polypeptide or nucleic acid to a subject, comprising administering to the subject a pharmaceutical composition comprising an anellosome comprising:
- (i) a proteinaceous exterior comprising an Anellovirus ORF1 polypeptide, and
- (ii) a genetic element encapsulated by the proteinaceous exterior;
- wherein the genetic element:
- (a) comprises a promoter operably linked to a heterologous sequence encoding the therapeutic polypeptide or nucleic acid;
- (b) comprises an Anellovirus 5′ UTR;
- (c) does not comprise any sequence encoding Anellovirus ORF1;
- (d) does not comprise any sequence encoding Anellovirus ORF2;
- and
- (e) does not comprise any sequence encoding Anellovirus ORF3.
A method of delivering a therapeutic polypeptide or nucleic acid to a subject, comprising administering to the subject a pharmaceutical composition comprising an anellosome comprising:
- (i) a proteinaceous exterior comprising an Anellovirus ORF1 polypeptide, and
- (ii) a genetic element encapsulated by the proteinaceous exterior;
- wherein the genetic element:
- (a) comprises a promoter operably linked to a heterologous sequence encoding the therapeutic polypeptide or nucleic acid;
- (b) comprises a nucleic acid sequence comprising at least 90% sequence identity to about 60 consecutive nucleotides of an Anellovirus 5′ UTR;
- (c) does not comprise any sequence encoding Anellovirus ORF1;
- (d) does not comprise any sequence encoding Anellovirus ORF2;
- and
- (e) does not comprise any sequence encoding Anellovirus ORF3.
A method of delivering a therapeutic polypeptide or nucleic acid to a subject, comprising administering to the subject a pharmaceutical composition comprising an anellosome comprising:
- (i) a proteinaceous exterior comprising an Anellovirus ORF1 polypeptide, and
- (ii) a genetic element encapsulating the proteinaceous exterior;
- wherein the genetic element:
- (a) comprises a promoter operably linked to a heterologous sequence encoding the therapeutic polypeptide or nucleic acid;
- (b) comprises a nucleic acid sequence comprising at least 90% sequence identity to about 60 consecutive nucleotides of an Anellovirus 5′ UTR;
- (c) does not comprise any sequence encoding Anellovirus ORF1 or comprises a nucleic acid sequence encoding nonfunctional Anellovirus ORF1;
- (d) does not comprise any sequence encoding Anellovirus ORF2 or comprises a nucleic acid sequence encoding nonfunctional Anellovirus ORF2;
- and
- (e) does not comprise any sequence encoding Anellovirus ORF3 or comprises a nucleic acid sequence encoding nonfunctional Anellovirus ORF3.
Dependencies are inferred from the claim text.