IP Library Granted Patent US 8,900,621
Granted Patent B2
US 8,900,621 · App. 13/790,980 · Granted Dec 2, 2014

Polyamine derivatives

Inventors: Gregory Slobodkin (Huntsville, AL); Richard Congo (Huntsville, AL); Majed Matar (Madison, AL); Jason Fewell (Madison, AL); Khursheed Anwer (Madison, AL); Brian Jeffery Sparks (Huntsville, AL)
Assignee: CLSN Laboratories, Inc.
A61K31/713C07C233/38C07C235/10C07C235/60A61K47/16A61K47/48061A61K47/48946A61K9/1272C07D475/04C07H15/10C07K7/23C07K7/64A61K47/48815
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Quick Facts
Patent No.
US 8,900,621
App. No.
13/790,980
Granted
Dec 2, 2014
Kind
B2
Abstract

Disclosed are compounds, compositions and methods for systemic and local delivery of biologically active molecules.

Claims (18)

1. A compound of the formula:

wherein

n1, n2, n3, and n4 are independently 1, 2, 3, or 4;

X and X′ are independently a bond, oxygen, or nitrogen;

R 1 and R 2 are independently C 8 -C 25 hydrocarbon groups optionally containing from 1-4 double or triple bonds; and

T 1 and T 2 are independently hydrogen or a targeting ligand;

G 1 and G 2 are independently bond or a polymer moiety,

where at least one of T 1 and T 2 is a targeting ligand.

2. A compound according to claim 1 , wherein n1, n2, n3, and n4 are all 1, and both X and X′ are bonds.

3. A compound according to claim 1 , wherein R 1 and R 2 are independently C 8 -C 25 hydrocarbon groups containing 1 or 2 double bonds.

4. A compound according to claim 1 , wherein at least one of G 1 and G 2 is a polyoxyalkylene, polyvinylpyrrolidone, polyacrylamide, polydimethylacrylamide, polyvinyl alcohol, dextran, poly(L-glutamic acid), styrene maleic anhydride, poly-N-(2-hydroxypropyl)methacrylamide, or polydivinylether maleic anhydride.

5. A compound according to claim 4 , wherein the polymer comprises at least one linker group between polymer units.

6. A compound according to claim 5 , wherein the polymer is a polyoxyalkylene where the oxyalkylene groups are independently straight or branched chain polyoxyalkylene groups having from 2-5 carbon atoms in their repeating units.

7. A formulation comprising a compound of claim 1 and a molecule selected from the group consisting of:

(a) ribosomal RNA; antisense polynucleotides of RNA or DNA; ribozymes; siRNA; shRNA; miRNA; and polynucleotides of genomic DNA, cDNA, or mRNA that encode for a therapeutically useful protein;

(b) an aptamer; or

(c) proteins, peptides, cholesterol, hormones, small molecule pharmaceutical compounds, vitamins, and co-factors.

8. A method for in vivo delivery of a molecule, said method comprising administering to a mammalian subject a therapeutically effective amount of a formulation of claim 7 .

Assignments (2)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Sep 22, 2014
From: EGEN, INC.
To: CLSN LABORATORIES, INC.
Reel/Frame 033788/0539 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Apr 25, 2014
From: SLOBODKIN, GREGORY; CONGO, RICHARD; MATAR, MAJED; FEWELL, JASON; ANWER, KHURSHEED; SPARKS, BRIAN JEFFREY
To: EGEN, INC.
Reel/Frame 032761/0640 →
Continuity (3)
Division 12727987 · Mar 19, 2010
Provisional Application 61161828 · Mar 20, 2009
Related Publication 20140050775A1 · Feb 20, 2014