Patent classifications
A61K38/30
DEVICE, SYSTEM AND METHODS FOR THE ORAL DELIVERY OF THERAPEUTIC COMPOUNDS
Embodiments of the invention provide swallowable devices, preparations and methods for delivering drugs and other therapeutic agents within the GI tract. Particular embodiments provide a swallowable device such as a capsule for delivering drugs into the intestinal wall or other GI lumen. Embodiments also provide various drug preparations that are configured to be contained within the capsule, advanced from the capsule into the intestinal wall and degrade within the wall to release the drug to produce a therapeutic effect. The preparation can be coupled to a delivery mechanism having one or more balloons or other expandable devices which are expandable responsive to a condition in the small intestine or other GI lumen to advance the preparation out of the capsule into the intestinal wall. Embodiments of the invention are particularly useful for the delivery of drugs which are poorly absorbed, tolerated and/or degraded within the GI tract.
PROANGIOGENIC PROTEIN COCKTAILS DELIVERED IN CUSTOM BIOMATERIALS TO REVASCULARIZE ISCHEMIC TISSUE
The invention provides a highly versatile system to promote vascularization in ischemic tissue. The system is composed of a fully defined, customizable hydrogel loaded with a potent cocktail of proangiogenic growth factors. The hydrogel's mechanical, degradation, and factor release behavior can be tailored to the specifications of any given target tissue or ischemic disease state. The growth factor cocktail can be optimized for maximal vessel density or size to meet the perfusion specifications required by the tissue. The embodiments of the disclosure concern methods and compositions of the system, with examples of preparation for both injectable and implantable delivery modes.
PROANGIOGENIC PROTEIN COCKTAILS DELIVERED IN CUSTOM BIOMATERIALS TO REVASCULARIZE ISCHEMIC TISSUE
The invention provides a highly versatile system to promote vascularization in ischemic tissue. The system is composed of a fully defined, customizable hydrogel loaded with a potent cocktail of proangiogenic growth factors. The hydrogel's mechanical, degradation, and factor release behavior can be tailored to the specifications of any given target tissue or ischemic disease state. The growth factor cocktail can be optimized for maximal vessel density or size to meet the perfusion specifications required by the tissue. The embodiments of the disclosure concern methods and compositions of the system, with examples of preparation for both injectable and implantable delivery modes.
Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain.
Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain.
Lyophilized pharmaceutical compositions for naked DNA gene therapy
The present invention provides a novel lyophilized pharmaceutical composition that maintains the stability of a DNA plasmid while forming a uniform and elegant cake during lyophilization. The novel lyophilization formulation further allows uniform reconstitution of the DNA plasmid in a pharmaceutically acceptable solution, enabling complete recover of the active ingredients, minimizing partial loss of potency and allowing administration of the active ingredients in an accurate and consistent manner. Additionally provided herein include methods of making the lyophilized pharmaceutical composition and methods of administering the composition for treatment of various diseases.
Lyophilized pharmaceutical compositions for naked DNA gene therapy
The present invention provides a novel lyophilized pharmaceutical composition that maintains the stability of a DNA plasmid while forming a uniform and elegant cake during lyophilization. The novel lyophilization formulation further allows uniform reconstitution of the DNA plasmid in a pharmaceutically acceptable solution, enabling complete recover of the active ingredients, minimizing partial loss of potency and allowing administration of the active ingredients in an accurate and consistent manner. Additionally provided herein include methods of making the lyophilized pharmaceutical composition and methods of administering the composition for treatment of various diseases.
Heparin-Associated Polypeptides and Uses Thereof
Described herein are polypeptides comprising an IGF2 amino acid sequence and an amino acid sequence from a heterologous polypeptide useful for the treatment of soft-tissue and muscle diseases, disorders, and injuries. Mutations within the IGF2 amino acid sequence improved the stability of the molecule by reducing backbone cleavage. Also described herein are synergistic combinations of an Insulin-like Growth Factor 1 Receptor (IGF1R) agonist and a short chain fatty acid. Also described are methods of treating muscle and soft-tissue diseases comprising administering the polypeptides and/or synergistic compositions.
Heparin-Associated Polypeptides and Uses Thereof
Described herein are polypeptides comprising an IGF2 amino acid sequence and an amino acid sequence from a heterologous polypeptide useful for the treatment of soft-tissue and muscle diseases, disorders, and injuries. Mutations within the IGF2 amino acid sequence improved the stability of the molecule by reducing backbone cleavage. Also described herein are synergistic combinations of an Insulin-like Growth Factor 1 Receptor (IGF1R) agonist and a short chain fatty acid. Also described are methods of treating muscle and soft-tissue diseases comprising administering the polypeptides and/or synergistic compositions.
APPLICATION OF NON-IGF1R-BINDING SUBSTANCE IN PREVENTION AND/OR TREATMENT OF INFLAMMATORY DISEASES
Provided is an application of a non-IGF1R-binding substance in the prevention and/or treatment of inflammatory diseases. Specifically, provided is use of a non-IGF1R-binding substance. The non-IGF1R-binding substance is used for preparing a composition or formulation, and the composition or formulation is used for preventing and/or treating inflammatory diseases.