Compositions and methods for delivery of nucleic acids
AssigneesPoseida Therapeutics, Inc., University of San Diego
InventorsJoshua Rychak, Jivan Yewle, Sean Essex, Nicholas Cam, Peter M. Iovine, Jake R. Hughes, Gopi Nath Vemuri
Life of this patent
Estimating the term…
Abstract
The present disclosure provides compositions and methods for the genetic modification of cells, including, but not limited to, resting T-cells and hepatocytes. The compositions and methods can comprise lipid nanoparticles, wherein the lipid nanoparticles comprise at least one multivalent cationic bolaform amphiphilic lipid, at least one structural lipid, at least one phospholipid and at least one PEGylated lipid.
Claims20
A composition comprising at least one lipid nanoparticle comprising at least one compound of Formula (I):
- or a salt thereof, wherein:
- each A is independently:
- each Z is —S—R1;
- each R1 is independently:
- each R2 or R3 is independently selected from hydrogen, C1-C6 alkyl, C1-C6 alkenyl, aralkyl, or hydroxyalkyl;
- each R4, R6 or R7 is independently selected from C1-C6 alkyl, C1-C6 alkenyl, or hydroxyalkyl;
- each R5 is independently hydrogen or C1-C6 alkyl;
- each X is independently halogen;
- each m is independently an integer independently selected from 1-10;
- each q is independently an integer independently selected from 1-200;
- each B is independently selected from:
- wherein each R is independently selected from hydrogen or C1-C6 alkyl;
- and C is:
- a. a C2-C100 hydrocarbon chain optionally containing one or more S atoms,
- b. CycA-L-CycB, wherein CycA and CycB are each independently a 5-8 membered cycloalkyl and L is C1-C3 alkylene,
- c. a polyalkylene oxide or polyalkylene oxide block copolymers of 200-5,000 molecular weight,
- d. an aliphatic or aromatic polyester of 200-5,000 molecular weight, or
- e. an aliphatic or aromatic polyurethane of 200-5,000 molecular weight;
- or
- wherein
- together form:
- wherein R8 is selected from C2-C6 alkyl, aryl,
- wherein t is an integer selected from 1-10;
- and
- wherein the at least one lipid nanoparticle further comprises at least one nucleic acid molecule.
Dependencies are inferred from the claim text.