Oral administration of therapeutic agent coupled to transporting agent

• US 20040014698A1
• Filed: 07/18/2002
• Published: 01/22/2004
• Est. Priority Date: 07/18/2002
• Status: Abandoned Application

• Current Assignee: NeoX, Inc.
• Original Assignee: NeoX, Inc.
• Inventors: Gomez Vargas, Andrew, Hortelano, Gonzalo
• Application Number:
  US10/199,914
• Publication Number:
  US 20040014698A1
• Time in Patent Office: Days
• US Class Current: 514/44
• CPC Class Codes: A61K 47/61 the organic macromolecular compound being a polysaccharide or a derivative thereof
• CPC Class Codes: A61K 47/645 Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
• CPC Class Codes: A61K 47/6927 the form being a solid microparticle having no hollow or gas-filled cores
• CPC Class Codes: A61K 47/6939 the polymer being a polysaccharide, e.g. starch, chitosan, chitin, cellulose or pectin
• CPC Class Codes: A61K 48/0025 wherein the non-active part clearly interacts with the delivered nucleic acid
• CPC Class Codes: A61K 48/0041 the non-active part being polymeric
• CPC Class Codes: A61K 48/0075 characterised by an aspect of the delivery route, e.g. oral, subcutaneous
• CPC Class Codes: A61P 11/00 Drugs for disorders of the respiratory system
• CPC Class Codes: A61P 21/00 Drugs for disorders of the muscular or neuromuscular system
• CPC Class Codes: A61P 21/04 for myasthenia gravis
• CPC Class Codes: A61P 3/10 for hyperglycaemia, e.g. antidiabetics
• CPC Class Codes: A61P 31/00 Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
• CPC Class Codes: A61P 31/18 for HIV
• CPC Class Codes: A61P 35/00 Antineoplastic agents
• CPC Class Codes: A61P 37/02 Immunomodulators
• CPC Class Codes: A61P 43/00 Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
• CPC Class Codes: A61P 7/04 Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
• CPC Class Codes: B82Y 5/00 Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery

Abstract

The present invention is directed toward a composition for widespread distribution, systemic expression and sustained delivery of a therapeutic agent and to a process for administration of a therapeutic agent via a natural gastrointestinal pathway. More particularly, the invention discloses a composition for the administration of oral gene therapy and a process for its production and use.

Claims

1. A composition for administration of a therapeutic agent to a host via a natural gastrointestinal pathway comprising:

at least one compound including an amine group; and

at least one genetic material;

said at least one compound and at least one genetic material coupled in a manner effective to enable widespread distribution, systemic expression and sustained delivery via said gastrointestinal path;

whereby a desirable biological effect is obtained.

4. The composition in accordance with any one of claims 1 or 2 or 3 wherein said transporting agent is a compound containing an amine group which facilitates widespread in vivo distribution of said genetic material upon coupling therewith.

5. The composition in accordance with any one of claims 1 or 2 or 3 wherein said transporting agent is a polypeptide.

6. The composition in accordance with claim 1 or 2 or 3 wherein said genetic material comprises a complete transcriptional unit.

7. The composition in accordance with claim 6 wherein said complete transcriptional unit is effective for ubiquitous expression of said therapeutic agent.

8. The composition in accordance with claim 6 wherein said complete transcriptional unit is effective for tissue specific expression.

2. A composition for administration of a therapeutic agent to a targeted cell via a natural gastrointestinal pathway comprising:

at least one transporting agent effective for transporting a genetic material via said natural gastrointestinal pathway; and

at least one genetic material effective for instigating a desirable biological effect;

said transporting agent and said at least one genetic material coupled in a manner effective to enable widespread distribution, systemic expression and sustained delivery as a result of cellular uptake subsequent to passage via said natural gastrointestinal pathway.

3. A composition for administration of a genetic material to a host via a natural gastrointestinal pathway thereby enabling intracellular expression of a therapeutic agent, comprising in combination:

at least one compound effective for protecting said genetic material within said natural gastrointestinal pathway;

at least one transporting agent effective for transporting a genetic material via said natural gastrointestinal pathway; and

at least one genetic material effective for intracellular expression of a therapeutic agent;

said transporting agent and said at least one genetic material coupled in a manner effective to enable widespread distribution, systemic delivery and sustained expression as a result of cellular uptake subsequent to passage via said natural gastrointestinal pathway;

whereby intracellular expression of said therapeutic agent occurs subsequent to said cellular uptake.

9. A process for expressing a therapeutic agent in a host by way of a natural gastrointestinal pathway comprising:

providing at least one transporting agent effective for enabling widespread distribution, systemic expression and sustained delivery of a genetic material coupled thereto via said natural gastrointestinal pathway;

providing at least one genetic material constructed and arranged to provide intracellular expression of said therapeutic agent upon cellular uptake thereof;

forming a distributable moiety by coupling said transporting agent and said at least one genetic material in a manner effective to produce widespread distribution, systemic delivery and sustained expression upon intracellular absorption via said natural gastrointestinal pathway;

administering said distributable moiety;

transporting said distributable moiety in vivo via said natural gastrointestinal pathway, whereby said moiety is included within essentially all cells of said subject; and

expressing said therapeutic agent subsequent to intracellular absorption,

wherein a desirable biological effect is instigated.

10. The process in accordance with claim 9 wherein said step of expressing is ubiquitous.

12. The process in accordance with claim 9 further including providing at least one compound effective for protecting said genetic material within said natural gastrointestinal pathway.

13. The process in accordance with claim 9 wherein said genetic material comprises at least one complete transcriptional unit.

14. The process in accordance with claim 9 wherein said transporting agent is a polypeptide.

15. The process in accordance with claim 9 wherein said transporting agent is a compound containing an amine group and constructed and arranged to couple with said genetic material to enable widespread distribution, systemic expression and sustained delivery of said therapeutic.

16. The process in accordance with claim 9 wherein said coupling is via electrostatic binding.

11. The process in accordance with clam 9 wherein said step of expressing is tissue specific.