The Administrators of the Tulane Educational Fund (USA)
Inventor
Hughes, Michael
Dhall, Sandeep
Wu, Hongju
Fonseca, Vivian A.
Abstract
Provided are recombinant adeno-associated virus (rAAV) vectors comprising a transgene to express Pax4; virions comprising said vectors (rAAV virions); methods of their production; methods of their use, including methods for treating diabetes, increasing insulin production and transdifferentiating α-cells to β-cells; pharmaceutical compositions and kits including same.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61P 3/10 - Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
Provided are recombinant adeno-associated virus (r.AAV) vectors comprising a transgene to express galactose-1-phosphate uridylyl transferase (GALT); virions comprising said vectors (r.AAV virions); methods of their production; methods of their use, including methods for treating galactosemia, GALT-deficiency, symptoms therefrom: and kits. These recombinant adeno-associated virus (rAAV) vectors comprise a method of treating galactosemia in a subject in need.
Provided are recombinant adeno-associated virus (rAAV) vectors comprising a transgene to express galactose-1-phosphate uridylyl transferase (GALT); virions comprising said vectors (rAAV virions); methods of their production; methods of their use, including methods for treating galactosemia, GALT-deficiency, symptoms therefrom; and kits.
Aspects of the disclosure relate to expression cassettes encoding miniShank3 proteins, AAV vectors comprising the expression cassettes, and gene therapy methods. In particular, the expression cassettes is formulated to further comprise AAV9 capsids, and the gene therapy is useful for treating neuroveleopmental disorders such as autism spectrum disorder (ASD) and Phelan-McDermid syndrome.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 31/711 - Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61P 25/00 - Drugs for disorders of the nervous system
40 - Treatment of materials; recycling, air and water treatment,
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biomanufacturing for others, namely, manufacturing of pharmaceuticals using biological organism in the manufacturing process; custom manufacture for others of products relating to biotechnology, namely, recombinant proteins for use in the biotechnology industries; custom manufacture for others of cell therapy products; custom manufacture for others of gene therapy products; custom manufacture of pharmaceuticals in accordance with specific standards GMP regulations Contract development and manufacturing organization (CDMO) services, namely, pharmaceutical drug development services; consulting and advisory services in the field of analytical development and quality control testing of pharmaceutical, gene therapy, and cell therapy products; consulting and advisory services in the field of manufacturing process development for pharmaceutical, gene therapy and cell therapy products; consulting in the field of chemistry manufacturing and controls (CMC) activities; testing of pharmaceutical, gene therapy, and cell therapy products and materials; quality control testing of pharmaceutical products and materials, namely, biopharmaceutical products and materials in accordance with specifications or requirements of regulatory standards applicable in target countries; testing and validation services of biopharmaceutical products to assure compliance with global regulatory requirements, specifications, and industry standards for pharmaceutical, cell therapy and gene therapy methods of development; technical consulting for regulatory compliance in the field of chemistry manufacturing and controls (CMC)
40 - Treatment of materials; recycling, air and water treatment,
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biomanufacturing for others, namely, manufacturing of pharmaceuticals using biological organism in the manufacturing process; custom manufacture for others of products relating to biotechnology, namely, recombinant proteins for use in the biotechnology industries; custom manufacture for others of cell therapy products; custom manufacture for others of gene therapy products; custom manufacture of pharmaceuticals in accordance with specific standards GMP regulations Contract development and manufacturing organization (CDMO) services, namely, pharmaceutical drug development services; consulting and advisory services in the field of analytical development and quality control testing of pharmaceutical, gene therapy, and cell therapy products; consulting and advisory services in the field of manufacturing process development for pharmaceutical, gene therapy and cell therapy products; consulting in the field of chemistry manufacturing and controls (CMC) activities; testing of pharmaceutical, gene therapy, and cell therapy products and materials; quality control testing of pharmaceutical products and materials, namely, biopharmaceutical products and materials in accordance with specifications or requirements of regulatory standards applicable in target countries; testing and validation services of biopharmaceutical products to assure compliance with global regulatory requirements, specifications, and industry standards for pharmaceutical, cell therapy and gene therapy methods of development; technical consulting for regulatory compliance in the field of chemistry manufacturing and controls (CMC)
40 - Treatment of materials; recycling, air and water treatment,
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biomanufacturing for others, namely, manufacturing of pharmaceuticals using biological organism in the manufacturing process; custom manufacture for others of products relating to biotechnology, namely, recombinant proteins for use in the biotechnology industries; custom manufacture for others of cell therapy products; custom manufacture for others of gene therapy products; custom manufacture of pharmaceuticals in accordance with specific standards GMP regulations Contract development and manufacturing organization (CDMO) services, namely, pharmaceutical drug development services; consulting and advisory services in the field of analytical development and quality control testing of pharmaceutical, gene therapy, and cell therapy products; consulting and advisory services in the field of manufacturing process development for pharmaceutical, gene therapy and cell therapy products; consulting in the field of chemistry manufacturing and controls (CMC) activities; testing of pharmaceutical, gene therapy, and cell therapy products and materials; quality control testing of pharmaceutical products and materials, namely, biopharmaceutical products and materials in accordance with specifications or requirements of regulatory standards applicable in target countries; testing and validation services of biopharmaceutical products to assure compliance with global regulatory requirements, specifications, and industry standards for pharmaceutical, cell therapy and gene therapy methods of development; technical consulting for regulatory compliance in the field of chemistry manufacturing and controls (CMC)
40 - Treatment of materials; recycling, air and water treatment,
42 - Scientific, technological and industrial services, research and design
Goods & Services
Biomanufacturing for others, namely, manufacturing of pharmaceuticals using biological organism in the manufacturing process; custom manufacture for others of products relating to biotechnology, namely, recombinant proteins for use in the biotechnology industries; custom manufacture for others of cell therapy products; custom manufacture for others of gene therapy products; custom manufacture of pharmaceuticals in accordance with specific standards GMP regulations Contract development and manufacturing organization (CDMO) services, namely, pharmaceutical drug development services; consulting and advisory services in the field of analytical development and quality control testing of pharmaceutical, gene therapy, and cell therapy products; consulting and advisory services in the field of manufacturing process development for pharmaceutical, gene therapy and cell therapy products; consulting in the field of chemistry manufacturing and controls (CMC) activities; testing of pharmaceutical, gene therapy, and cell therapy products and materials; quality control testing of pharmaceutical products and materials, namely, biopharmaceutical products and materials in accordance with specifications or requirements of regulatory standards applicable in target countries; testing and validation services of biopharmaceutical products to assure compliance with global regulatory requirements, specifications, and industry standards for pharmaceutical, cell therapy and gene therapy methods of development; technical consulting for regulatory compliance in the field of chemistry manufacturing and controls (CMC)
THE ADMINISTRATORS OF THE TULANE EDUCATIONAL FUND (USA)
Inventor
Hughes, Michael
Dhall, Sandeep
Wu, Hongju
Fonseca, Vivian A.
Abstract
Provided are recombinant adeno-associated virus (rAAV) vectors comprising a transgene to express Pax4; virions comprising said vectors (rAAV virions); methods of their production; methods of their use, including methods for treating diabetes, increasing insulin production and transdifferentiating ?-cells to ?-cells; pharmaceutical compositions and kits including same.
THE ADMINISTRATORS OF THE TULANE EDUCATIONAL FUND (USA)
Inventor
Hughes, Michael
Dhall, Sandeep
Wu, Hongju
Fonseca, Vivian, A.
Abstract
Provided are recombinant adeno-associated virus (rAAV) vectors comprising a transgene to express Pax4; virions comprising said vectors (rAAV virions); methods of their production; methods of their use, including methods for treating diabetes, increasing insulin production and transdifferentiating α-cells to β-cells; pharmaceutical compositions and kits including same.
Provided are recombinant adeno-associated virus (rAAV) vectors comprising a transgene to express galactose- 1 -phosphate uridylyl transferase (GALT); virions comprising said vectors (rAAV virions); methods of their production; methods of their use, including methods for treating galactosemia, GALT -deficiency, symptoms therefrom; and kits.
Provided are recombinant adeno-associated virus (rAAV) vectors comprising a transgene to express galactose- 1 -phosphate uridylyl transferase (GALT); virions comprising said vectors (rAAV virions); methods of their production; methods of their use, including methods for treating galactosemia, GALT-deficiency, symptoms therefrom; and kits.
A61K 38/16 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
Provided are recombinant adeno-associated virus (rAAV) vectors comprising a transgene to express galactose- 1 -phosphate uridylyl transferase (GALT); virions comprising said vectors (rAAV virions); methods of their production; methods of their use, including methods for treating galactosemia, GALT-deficiency, symptoms therefrom; and kits.
Provided are recombinant adeno-associated virus (r.AAV) vectors comprising a transgene to express galactose-1 -phosphate uridylyl transferase (GALT); virions comprising said vectors (r.AAV virions); methods of their production; methods of their use, including methods for treating galactosemia, GALT-deficiency, symptoms therefrom: and kits. These recombinant adeno-associated virus (rAAV) vectors comprise a method of treating galactosemia in a subject in need.
A61K 38/16 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
C12N 15/00 - Mutation or genetic engineeringDNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purificationUse of hosts therefor
Technologies are provided for automated extraction of a substance from a tube. The technologies comprise a system that includes an aseptic extraction chamber and a transfer arm configured to position an ultracentrifuge (UC) tube within the aseptic extraction chamber. The UC tube contains a liquid solution that has particulate matter embedded therein, where the particulate matter forms multiple layers. The system also includes an extractor arm configured to extract a particular layer of the multiple layers.
