Compositions of oral gene therapy and methods of using same
AssigneeThe Johns Hopkins University
InventorsKam Leong, Grace Okoli, Gonzalo Hortelano
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Abstract
The present invention provides nanoparticle compositions comprising a cationic biopolymer and at least one biologically active substance, pharmaceutical compositions comprising such nanoparticles and methods for the oral administration of biologically active molecules which are susceptible to degradation in the gastro-intestinal tract using nanoparticle. The present invention further provides compositions and methods for the oral administration of gene therapy.
Claims53
A method for oral administration of a biologically active substance which is susceptible to degradation in the gastro-intestinal tract, the method comprising the steps of:
- providing an orally deliverable nanoparticle composition comprising
- at least one biologically active substance susceptible to degradation in the gastro-intestinal tract;
- and
- at least one cationic biopolymer selected from optionally substituted chitin, optionally substituted chitosan, or a derivative thereof;
- and
- orally administering the nanoparticle composition to a patient such that at least a portion of the biologically active substance present in the nanoparticle composition is taken up by the patient without degradation in the gastrointestinal tract.
A method for oral administration of a gene therapy, the method comprising the steps of:
- providing an orally deliverable nanoparticle composition comprising
- at least a portion of at least one gene;
- and
- at least one cationic biopolymer selected from optionally substituted chitin,
- optionally substituted chitosan, or a derivative thereof;
- and
- administering the nanoparticle composition to a patient orally such that at least a portion of gene or gene fragment present in the nanoparticle composition is delivered to a biological fluid, cell or tissue such that gene therapy occurs without degradation of the gene or gene fragment in the gastro-intestinal tract.
A nanoparticle composition for the oral delivery of a biologically active substance which is susceptible to degradation in the gastro-intestinal tract to a patient, the composition comprising:
- at least one biologically active substance susceptible to degradation in the gastro-intestinal tract;
- and
- at least one cationic biopolymer according to Formula II:
- wherein
- R is independently selected at each occurrence from the group consisting of hydrogen, optionally substituted alkyl, C(O)R′, steroid derivatives, and cellular recognition ligands;
- R′ is independently selected at each occurrence from the group consisting of optionally substituted alkyl, steroid derivatives and cellular recognition ligands;
- X is a pharmaceutically acceptable anion;
- n is an integer from about 10 to about 20,000;
- and
- y is 1 or 2.
A method of preparing a nanoparticle composition, the method comprising the steps of:
- providing at least one cationic biopolymer selected from optionally substituted chitin, optionally substituted chitosan, or a derivative thereof and at least one biologically active substance;
- combining the cationic biopolymer and the biologically active substance in a homogeneous solution;
- inducing phase separation of the homogeneous solution under conditions conducive to the formation of a nanoparticle composition comprising the cationic biopolymer and the biologically active substance.
Dependencies are inferred from the claim text.