Disclosed herein are methods for the treatment of Pompe Disease comprising administering a recombinant AAV (rAAV) vector comprising a rAVV genome comprising a heterologous nucleic acid encoding an acid alpha-glucosidase (GAA) polypeptide operatively linked to a liver-specific promoter, wherein the subject is withdrawn or not administered enzyme replacement therapy (ERT).
The present invention relates to regulatory nucleic acid sequences, in particular short liver-specific cis-regulatory elements, cis-regulatory modules, promoters and other such nucleic acid sequences, that are short and capable of enhancing liver-specific expression of genes. The invention also relates to expression constructs, vectors and cells comprising such short liver-specific regulatory nucleic acid sequences, and to methods of their use. The liver-specific regulatory nucleic acid sequences are of particular utility for gene therapy applications, but also find utility in other areas such as bioprocessing and biotechnology.
A61K 47/61 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule the organic macromolecular compound being a polysaccharide or a derivative thereof
A61K 47/60 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
The present invention relates to regulatory nucleic acid sequences, in particular muscle- specific promoters, elements thereof, and other such nucleic acid sequences, that are capable of enhancing muscle-specific expression of genes. In particular, the invention relates to regulatory nucleic acid sequences capable of enhancing muscle-specific expression but having low liver-specific expression. The invention also relates to expression constructs, vectors and cells comprising such muscle-specific regulatory nucleic acid sequences, and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications, but also find utility in other areas such as bioprocessing and biotechnology.
09 - Scientific and electric apparatus and instruments
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Non-downloadable software for patients, caregivers and
medical professionals for use in managing pharmaceutical
treatments and administering pharmaceutical preparations;
downloadable mobile applications for patients, caregivers,
and medical professionals for use in managing pharmaceutical
treatments and administering pharmaceutical preparations. Pharmaceutical patient support program providing guidance
and information on pharmaceutical treatment and
administration via non-downloadable software and a
downloadable mobile application.
09 - Scientific and electric apparatus and instruments
42 - Scientific, technological and industrial services, research and design
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Downloadable software in the nature of a mobile application for patients, caregivers, and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations Providing online non-downloadable software for patients, caregivers and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations Pharmaceutical patient support program, namely, providing guidance and information to patients, caregivers, and medical professionals on pharmaceutical treatment and administration of pharmaceutical preparations via non-downloadable software and a downloadable mobile application
09 - Scientific and electric apparatus and instruments
42 - Scientific, technological and industrial services, research and design
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Downloadable software in the nature of a mobile application for patients, caregivers, and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations Providing online non-downloadable software for patients, caregivers and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations Pharmaceutical patient support program, namely, providing guidance and information to patients, caregivers, and medical professionals on pharmaceutical treatment and administration of pharmaceutical preparations via non-downloadable software and a downloadable mobile application
09 - Scientific and electric apparatus and instruments
42 - Scientific, technological and industrial services, research and design
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Downloadable software in the nature of a mobile application for patients, caregivers, and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations Providing online non-downloadable software for patients, caregivers and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations Pharmaceutical patient support program, namely, providing guidance and information to patients, caregivers, and medical professionals on pharmaceutical treatment and administration of pharmaceutical preparations via non-downloadable software and a downloadable mobile application
16 - Paper, cardboard and goods made from these materials
Goods & Services
Educational materials and guides for school staff and administration to educate them on the unique needs and medical nuances of students living with bleeding conditions
09 - Scientific and electric apparatus and instruments
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Non-downloadable software for patients, caregivers and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations; downloadable mobile applications for patients, caregivers, and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations. Pharmaceutical patient support program providing guidance and information on pharmaceutical treatment and administration via non-downloadable software and a downloadable mobile application.
20.
ADENOVIRUS-BASED NUCLEIC ACIDS AND METHODS THEREOF
A reactor includes a support base, at least one wall, a top cover and a moveable plate assembly. The top cover, the support base and the at least one wall form an internal chamber therein. The moveable plate assembly includes a moveable plate located within the internal chamber. The moveable plate being configured to move in the x-direction, y-direction, and the z-direction. The temperature of the moveable plate is controllable to a desired temperature. The internal chamber is accessible via at least one of the top cover, the support base, and the at least one wall.
01 - Chemical and biological materials for industrial, scientific and agricultural use
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Chemical preparations, enzymes, DNA vectors, viral vectors, rAAV vectors, and DNA extracts for the preparation of nucleic acid, for scientific and research purposes; chemicals for use in the preparation and creation of DNA vectors, viral vectors, rAAV vectors, DNA extracts and cassettes for scientific and industrial purposes; enzymes for use in the preparation and creation of DNA extracts and cassettes for scientific and industrial purposes; DNA vectors, viral vectors, rAAV vectors, and DNA extracts and cassettes for scientific and research use; DNA vectors, viral vectors, rAAV vectors, DNA extracts for scientific and research purposes in the fields of agri-biotechnology and nanotechnology; Biological and biochemical reagents being DNA plasmid vectors, DNA vectors, and rAAV vectors, viral vectors that form a gene analysis system not for medical purposes; Diagnostic reagents for scientific or research purposes, namely, a diagnostic kit consisting primarily of DNA vectors, rAAV vectors, or DNA plasmid vectors. Vaccines; DNA based vaccines; DNA extracts, namely, nucleic acid sequences and chemical reagents for medical use; Therapeutic DNA, namely, nucleic acid sequences and chemical reagents for medical and veterinary purposes; Biotechnological products, namely, viral DNA vectors, DNA vectors, and rAAV vectors, for the delivery of genes and other materials, in the nature of DNA, RNA, or transcriptional targeting promoters, to a biological cell for medical purposes; Biological preparations, namely, viral vectors, rAAV vectors, and DNA extracts for production of DNA vectors and viral vectors, for medical purposes; Recombinant viruses. Scientific and technological services, namely, research and design in the field of DNA research and development; chemical and biological analysis and research services; scientific research in the field of DNA; scientific research and development in the field of creating DNA extracts; scientific and technological services, namely, scientific research, analysis, design and development in the field of DNA based pharmaceutical drugs and therapies; scientific research and development in the fields of gene therapy, diagnostics, agri-biotechnology and nanotechnology; scientific consultancy services in the fields of gene therapy, diagnostics, agri-biotechnology, nano technology and DNA analysis.
01 - Chemical and biological materials for industrial, scientific and agricultural use
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
(1) Chemical preparations, enzymes, DNA vectors, viral vectors, rAAV vectors, and DNA extracts for the preparation of nucleic acid, for scientific and research purposes; chemicals for use in the preparation and creation of DNA vectors, viral vectors, rAAV vectors, DNA extracts and cassettes for scientific and industrial purposes; enzymes for use in the preparation and creation of DNA extracts and cassettes for scientific and industrial purposes; DNA vectors, viral vectors, rAAV vectors, and DNA extracts and cassettes for scientific and research use; DNA vectors, viral vectors, rAAV vectors, DNA extracts for scientific and research purposes in the fields of agri-biotechnology and nanotechnology; Biological and biochemical reagents being DNA plasmid vectors, DNA vectors, and rAAV vectors, viral vectors that form a gene analysis system not for medical purposes; Diagnostic reagents for scientific or research purposes, namely, a diagnostic kit consisting primarily of DNA vectors, rAAV vectors, or DNA plasmid vectors.
(2) Vaccines; DNA based vaccines; DNA extracts, namely, nucleic acid sequences and chemical reagents for medical use; Therapeutic DNA, namely, nucleic acid sequences and chemical reagents for medical and veterinary purposes; Biotechnological products, namely, viral DNA vectors, DNA vectors, and rAAV vectors, for the delivery of genes and other materials, in the nature of DNA, RNA, or transcriptional targeting promoters, to a biological cell for medical purposes; Biological preparations, namely, viral vectors, rAAV vectors, and DNA extracts for production of DNA vectors and viral vectors, for medical purposes; Recombinant viruses. (1) International Class 042: Scientific and technological services, namely, research and design in the field of DNA research and development; chemical and biological analysis and research services; scientific research in the field of DNA; scientific research and development in the field of creating DNA extracts; scientific and technological services, namely, scientific research, analysis, design and development in the field of DNA based pharmaceutical drugs and therapies; scientific research and development in the fields of gene therapy, diagnostics, agri-biotechnology and nanotechnology; scientific consultancy services in the fields of gene therapy, diagnostics, agri-biotechnology, nano technology and DNA analysis.
25.
FORSKOLIN-INDUCIBLE PROMOTERS AND HYPOXIA-INDUCIBLE PROMOTERS
Disclosed herein are codon-optimized nucleic acids encoding a Factor VIII polypeptide. Also disclosed are expression cassettes and expression vectors (e.g., recombinant AAV (rAAV) vectors) that contain the codon-optimized nucleic acids in expressible form. Methods for the treatment of Hemophilia A comprising administering expression vector comprising the codon-optimized nucleic acids (e.g., a recombinant AAV (rAAV) vector) are also disclosed.
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
The present invention relates to regulatory nucleic acid sequences, in particular NS-specific promoters, cell specific promoters, multi-cell specific promoters, and elements thereof. The invention also relates to expression constructs, vectors, virions, pharmaceutical compositions and cells comprising such promoters and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications.
42 - Scientific, technological and industrial services, research and design
45 - Legal and security services; personal services for individuals.
Goods & Services
Promoting collaboration between scientists, technicians, clinical specialists, patients, and their families to achieve advances in therapeutics, technology, regulatory requirements, and public policy in the field of healthcare, genetic disease, gene therapy, and clinical trials Providing medical and scientific research information in the field of gene therapy, and clinical trials; consulting services for others in the field of designing clinical trials; medical and scientific research, namely, designing clinical trials for others Providing patient advocate services in the field of genetic disease, gene therapy, and potential curative therapies; providing personal support services for patients, namely, emotional counseling and emotional support by providing personal emotional support information in the fields of genetic disease, gene therapy, and potential curative therapies; providing patient advocacy and personal emotional support services to potential participants and participants in clinical trials for genetic diseases and gene therapy; providing information in the field of personal emotional support of gene therapy patients and patients' families
29.
METHODS AND COMPOSITIONS FOR THE TREATMENT OF PARKINSON'S DISEASE
Aspects of the disclosure relate to compositions and methods useful for treating Parkinson's disease. In some embodiments, the disclosure provides a method for treating Parkinson's disease comprising administration of a viral vector comprising a GDNF nucleic acid sequence. In some embodiments, administration is locally to the subject putamen. In some embodiments, administration is systemically, e.g., via the viral vector comprising a modified viral capsid, such as for preferentially targeting cells in the CNS or PNS.
THERAPEUTIC ADENO-ASSOCIATED VIRUS USING CODON OPTIMIZED NUCLEIC ACID ENCODING ALPHA-GLUCOSIDASE (GAA) FOR TREATING POMPE DISEASE, WITH SIGNAL PEPTIDE MODIFICATIONS
Disclosed herein is a method for the treatment of Pompe Disease comprising administering a recombinant AAV (rAAV) vector comprising a rAVV genome comprising a heterologous nucleic acid encoding a GAA signal peptide or portion thereof, a heterologous signal peptide, and an acid alpha-glucosidase (GAA) polypeptide, or N-terminal truncation thereof, where the heterologous nucleic acid is operatively linked to a liver-specific promoter, where the nucleic acid encoding GAA polypeptide can be wild type nucleic acid sequence, or modified nucleic acid sequence, or a codon optimized nucleic acid sequence, and can optionally be modified to reduce or completely eliminate CG and CpG dinucleotides and, optionally eliminated alternative reading frames (ARF) content.
09 - Scientific and electric apparatus and instruments
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Downloadable mobile application for patients, caregivers, and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations. Pharmaceutical patient support program providing guidance and information on pharmaceutical treatment and administration via a downloadable mobile application.
09 - Scientific and electric apparatus and instruments
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Downloadable mobile application for patients, caregivers, and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations. Pharmaceutical patient support program providing guidance and information on pharmaceutical treatment and administration via a downloadable mobile application.
33.
THERAPEUTIC ADENO-ASSOCIATED VIRUS FOR TREATING POMPE DISEASE WITH LONG TERM CESSATION OF GAA ENZYME REPLACEMENT THERAPY
Disclosed herein are methods for the treatment of Pompe Disease comprising administering a recombinant AAV (rAAV) vector comprising a rAVV genome comprising a heterologous nucleic acid encoding an acid alpha-glucosidase (GAA) polypeptide operatively linked to a liver-specific promoter, wherein the subject is withdrawn or not administered enzyme replacement therapy (ERT).
Aspects of the disclosure relate to compositions and methods useful for treating neurological diseases and disorders. In some embodiments, the disclosure provides a method for treating a neurological disease or disorder comprising administration of both a viral vector comprising interfering nucleic acids (e.g., artificial miRNAs) and a viral vector comprising a CYP46A1 protein. In some embodiments, the disclosure provides a method for treating Huntington's disease comprising administration of both a viral vector comprising interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and a viral vector comprising a CYP46A1 protein. In some embodiments, the viral vector comprises a modified viral capsid, such as for preferentially targeting cells in the CNS or PNS.
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
A61P 25/00 - Drugs for disorders of the nervous system
The present invention relates to regulatory nucleic acid sequences, in particular muscle-specific promoters, elements thereof, and other such nucleic acid sequences, that are capable of enhancing muscle-specific expression of genes. The invention also relates to expression constructs, vectors and cells comprising such muscle-specific regulatory nucleic acid sequences, and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications, but also find utility in other areas such as bioprocessing and biotechnology.
16 - Paper, cardboard and goods made from these materials
Goods & Services
Printed educational materials and guides for school staff and administration to educate them on the unique needs and medical nuances of students living with bleeding conditions
38.
FORSKOLIN-INDUCIBLE PROMOTERS AND HYPOXIA-INDUCIBLE PROMOTERS
42 - Scientific, technological and industrial services, research and design
Goods & Services
Scientific and technological services, namely, research and design in the field of DNA research and development; chemical and biological analysis and research services; scientific research in the field of DNA; scientific research and development in the field of creating DNA extracts; scientific and technological services, namely, scientific research, analysis, design and development in the field of DNA based pharmaceutical drugs and therapies; scientific research and development in the fields of gene therapy, diagnostics, agri-biotechnology and nanotechnology; scientific consultancy services in the fields of gene therapy, diagnostics, agri-biotechnology, nano technology and DNA analysis
01 - Chemical and biological materials for industrial, scientific and agricultural use
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Enzymes, DNA vectors, viral vectors, rAAV vectors, and DNA extracts for the preparation of nucleic acid, for scientific and research purposes; enzymes for use in the preparation and creation of DNA extracts and DNA cassettes for scientific and industrial purposes; DNA vectors, viral vectors, rAAV vectors, and DNA extracts and DNA cassettes for scientific and research use; DNA vectors, viral vectors, rAAV vectors, DNA extracts for scientific and research purposes in the fields of agri-biotechnology and nanotechnology; Biological and biochemical reagents being DNA plasmid vectors, DNA vectors, and rAAV vectors, viral vectors that form a gene analysis system not for medical purposes; diagnostic reagents for scientific or research purposes, namely, a diagnostic kit consisting primarily of DNA vectors, rAAV vectors, and DNA plasmid vectors for scientific and research purposes Biotechnological products, namely, viral DNA vectors, DNA vectors, and rAAV vectors, for the delivery of genes and other materials, in the nature of DNA, RNA, and transcriptional targeting promoters, to a biological cell for medical purposes; biological preparations, namely, viral vectors, rAAV vectors, and DNA extracts for production of DNA vectors and viral vectors, for medical purposes
01 - Chemical and biological materials for industrial, scientific and agricultural use
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
DNA vectors, viral vectors, rAAV vectors, and DNA extracts for the preparation of nucleic acid for scientific and research purposes; chemicals for use in the preparation and creation of DNA vectors, viral vectors, rAAV vectors, DNA extracts and cassettes for scientific and industrial purposes; enzymes for use in the preparation and creation of DNA vectors, rAAV vectors, DNA extracts and cassettes for scientific and industrial purposes; DNA vectors, viral vectors, rAAV vectors, and DNA extracts and cassettes for scientific and research use; DNA vectors, viral vectors, rAAV vectors, and DNA extracts for scientific and research purposes in the fields of agri-biotechnology and nanotechnology; Biological and biochemical reagents being RNA polymerase, DNA polymerase, DNA plasmid vectors, DNA vectors, viral vectors, and rAAV vectors, and RNA markers that form a gene analysis system not for medical purposes; Diagnostic reagents for scientific or research purposes, namely, a diagnostic kit consisting primarily of DNA vectors, rAAV vectors, or DNA plasmid vectors. Vaccines; DNA based vaccines; DNA extracts, namely, nucleic acid sequences and chemical reagents for medical use; Therapeutic DNA, namely, nucleic acid sequences and chemical reagents for medical and veterinary purposes; Biotechnological products, namely, viral DNA vectors, DNA vectors, and rAAV vectors, for the delivery of genes and other materials, in the nature of DNA, RNA, or transcriptional targeting promoters, to a biological cell for medical purposes; Biological preparations, namely, viral vectors, rAAV vectors, and DNA extracts for production of DNA vectors and viral vectors for medical purposes; Recombinant viruses. Scientific and technological services, namely, research and design in the field of DNA research and development; chemical and biological analysis and research services; scientific research in the field of DNA; scientific research and development in the field of creating DNA extracts; scientific and technological services, namely, scientific research, analysis, design and development in the field of DNA based pharmaceutical drugs and therapies; scientific research and development in the fields of gene therapy, diagnostics, agri-biotechnology and nanotechnology; scientific consultancy services in the fields of gene therapy, diagnostics, agri-biotechnology, nano technology, and DNA analysis.
01 - Chemical and biological materials for industrial, scientific and agricultural use
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Chemical preparations, enzymes, DNA vectors, viral vectors, rAAV vectors, and DNA extracts for the preparation of nucleic acid for scientific and research purposes; chemicals for use in the preparation and creation of DNA vectors, viral vectors, rAAV vectors, DNA extracts and cassettes for scientific and industrial purposes; enzymes for use in the preparation and creation of DNA vectors, rAAV vectors, DNA extracts and cassettes for scientific and industrial purposes; DNA vectors, viral vectors, rAAV vectors, and DNA extracts and cassettes for scientific and research use; DNA vectors, viral vectors, rAAV vectors, and DNA extracts for scientific and research purposes in the fields of agri-biotechnology and nanotechnology; Biological and biochemical reagents being RNA polymerase, DNA polymerase, DNA plasmid vectors, DNA vectors, viral vectors, and rAAV vectors, and RNA markers that form a gene analysis system not for medical purposes; Diagnostic reagents for scientific or research purposes, namely, a diagnostic kit consisting primarily of DNA vectors, rAAV vectors, or DNA plasmid vectors. Vaccines; DNA based vaccines; DNA extracts, namely, nucleic acid sequences and chemical reagents for medical use; Therapeutic DNA, namely, nucleic acid sequences and chemical reagents for medical and veterinary purposes; Biotechnological products, namely, viral DNA vectors, DNA vectors, and rAAV vectors, for the delivery of genes and other materials, in the nature of DNA, RNA, or transcriptional targeting promoters, to a biological cell for medical purposes; Biological preparations, namely, viral vectors, rAAV vectors, and DNA extracts for production of DNA vectors and viral vectors for medical purposes; Recombinant viruses. Scientific and technological services, namely, research and design in the field of DNA research and development; chemical and biological analysis and research services; scientific research in the field of DNA; scientific research and development in the field of creating DNA extracts; scientific and technological services, namely, scientific research, analysis, design and development in the field of DNA based pharmaceutical drugs and therapies; scientific research and development in the fields of gene therapy, diagnostics, agri-biotechnology and nanotechnology; scientific consultancy services in the fields of gene therapy, diagnostics, agri-biotechnology, nano technology, and DNA analysis.
The present invention relates to regulatory nucleic acid sequences, in particular CNS—specific promoters, and elements thereof. The invention also relates to expression constructs, vectors, virions, pharmaceutical compositions and cells comprising such promoters and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Vaccines; DNA based vaccines; DNA extracts, namely, nucleic acid sequences and chemical reagents for medical use; Therapeutic DNA, namely, nucleic acid sequences and chemical reagents for medical and veterinary purposes; Biotechnological products, namely, viral DNA vectors, DNA vectors, and rAAV vectors, for the delivery of genes and other materials, in the nature of DNA, RNA, and transcriptional targeting promoters, to a biological cell for medical purposes; Biological preparations, namely, viral vectors, rAAV vectors, and DNA extracts for production of DNA vectors and viral vectors for medical purposes
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Vaccines; DNA based vaccines; DNA extracts, namely, nucleic acid sequences and chemical reagents for medical use; Therapeutic DNA, namely, nucleic acid sequences and chemical reagents for medical and veterinary purposes; Biotechnological products, namely, viral DNA vectors, DNA vectors, and rAAV vectors, for the delivery of genes and other materials, in the nature of DNA, RNA, and transcriptional targeting promoters, to a biological cell for medical purposes; Biological preparations, namely, viral vectors, rAAV vectors, and DNA extracts for production of DNA vectors and viral vectors for medical purposes
Recombinant AAV (rAAV) vectors comprising a rAVV genome comprising a heterologous nucleic acid encoding a lysosomal protein, e.g., acid alpha-glucosidase (GAA) polypeptide, and optionally a signal peptide and/or optionally a targeting sequence, e.g., IGF2 targeting peptide, operatively linked to a liver-specific promoter (LSP), enabling the GAA polypeptide to be secreted from the liver and targeted to the lysosomes. Particular embodiments relate to a recombinant AAV (rAAV) vector encoding an alpha-glucosidase (GAA) polypeptide, having a liver secretory signal peptide and a IGF2 targeting peptide that binds human cation-independent mannose-6-phosphate receptor (CI-MPR) or to the IGF2 receptor, permitting proper subcellular localization of the GAA polypeptide to lysosomes. Also encompassed are cells, and methods to treat a lysosomal disease, for example, a glycogen storage disease type II (GSD II) disease and/or Pompe Disease with the rAAV vector.
The present invention relates to regulatory nucleic acid sequences, in particular muscle- specific promoters, elements thereof, and other such nucleic acid sequences, that are capable of enhancing muscle-specific expression of genes. The invention also relates to expression constructs, vectors and cells comprising such muscle-specific regulatory nucleic acid sequences, and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications, but also find utility in other areas such as bioprocessing and biotechnology.
The present invention relates to regulatory nucleic acid sequences, in particular muscle- specific promoters, elements thereof, and other such nucleic acid sequences, that are capable of enhancing muscle-specific expression of genes. The invention also relates to expression constructs, vectors and cells comprising such muscle-specific regulatory nucleic acid sequences, and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications, but also find utility in other areas such as bioprocessing and biotechnology.
A cannula assembly includes a cannula, polymeric support material and a hub. The cannula has a proximal end and a distal end. The polymeric support material substantially surrounds a portion of the cannula at or near the proximal end. The hub is configured to be attached to a syringe. The polymeric support material is located between the cannula and the hub. The cannula, the polymeric support material and the hub are adhesively attached.
A cannula assembly includes a cannula, polymeric support material and a hub. The cannula has a proximal end and a distal end. The polymeric support material substantially surrounds a portion of the cannula at or near the proximal end. The hub is configured to be attached to a syringe. The polymeric support material is located between the cannula and the hub. The cannula, the polymeric support material and the hub are adhesively attached.
The present invention relates to a substantially homogenous population of a rational polyploid adeno-associated virus (AAV) virons that cross the blood brain barrier (BBB), where the rational polyploid comprises a VP3 viral structural protein from any AAV serotype that cross the BBB. In some embodiments, the rational polyploid crosses the BBB upon systemic or intrathecal administration to a subject. In some embodiments, a rational polyploid AAV virion comprises at least one VP1 and/or VP2 viral structural protein in addition to the VP3 protein. In some embodiments, the VP3 capsid protein is from a non-human primate, and in some embodiments the VP3 capsid protein is a AAV rhesus monkey serotype. In specific embodiments, rational polyploid AAV virion comprises a VP1 capsid protein from AAV8, and at least a VP3 capsid protein from any AAV serotype that cross the BBB.
The present invention relates to a substantially homogenous population of a rational polyploid adeno-associated virus (AAV) virons that cross the blood brain barrier (BBB), where the rational polyploid comprises a VP3 viral structural protein from any AAV serotype that cross the BBB. In some embodiments, the rational polyploid crosses the BBB upon systemic or intrathecal administration to a subject. In some embodiments, a rational polyploid AAV virion comprises at least one VP1 and/or VP2 viral structural protein in addition to the VP3 protein. In some embodiments, the VP3 capsid protein is from a non-human primate, and in some embodiments the VP3 capsid protein is a AAV rhesus monkey serotype. In specific embodiments, rational polyploid AAV virion comprises a VP1 capsid protein from AAV8, and at least a VP3 capsid protein from any AAV serotype that cross the BBB.
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
(1) Produits pharmaceutiques et vétérinaires ainsi que produits hygiéniques, à savoir, préparations pharmaceutiques pour la prévention et le traitement des saignements causés par des troubles sanguins; produits diététiques pour enfants et malades, à savoir, suppléments alimentaires pour l'état général de la santé et le bien-être; emplâtres en tant que matériel pour pansements, matériel pour pansements, à savoir, pansements médicaux; matières pour plomber les dents et pour empreintes dentaires; désinfectants multi usages; préparations pour détruire les mauvaises herbes et les animaux nuisibles.
Provided herein are purification, production and manufacturing methods for recombinant viral vector particles such as recombinant adeno-associated viral (rAAV) vector particles substantially free of empty viral particles; a population of recombinant adeno-associated virus (rAAV) particles purified using the method described herein, and a pharmaceutical composition comprising the purified rAAV.
B01D 15/16 - Selective adsorption, e.g. chromatography characterised by constructional or operational features relating to the conditioning of the fluid carrier
B01D 15/36 - Selective adsorption, e.g. chromatography characterised by the separation mechanism involving ionic interaction, e.g. ion-exchange, ion-pair, ion-suppression or ion-exclusion
B01D 15/38 - Selective adsorption, e.g. chromatography characterised by the separation mechanism involving specific interaction not covered by one or more of groups , e.g. affinity, ligand exchange or chiral chromatography
B01D 15/42 - Selective adsorption, e.g. chromatography characterised by the development mode, e.g. by displacement or by elution
Provided herein are purification, production and manufacturing methods for recombinant viral vector particles such as recombinant adeno-associated viral (rAAV) vector particles substantially free of empty viral particles; a population of recombinant adeno-associated virus (rAAV) particles purified using the method described herein, and a pharmaceutical composition comprising the purified rAAV.
B01D 15/38 - Selective adsorption, e.g. chromatography characterised by the separation mechanism involving specific interaction not covered by one or more of groups , e.g. affinity, ligand exchange or chiral chromatography
B01D 15/36 - Selective adsorption, e.g. chromatography characterised by the separation mechanism involving ionic interaction, e.g. ion-exchange, ion-pair, ion-suppression or ion-exclusion
B01D 15/16 - Selective adsorption, e.g. chromatography characterised by constructional or operational features relating to the conditioning of the fluid carrier
B01D 15/42 - Selective adsorption, e.g. chromatography characterised by the development mode, e.g. by displacement or by elution
01 - Chemical and biological materials for industrial, scientific and agricultural use
Goods & Services
Chemical preparations, enzymes, DNA vectors, and DNA extracts for the preparation of nucleic acid for scientific and research purposes; chemicals for use in the preparation and creation of DNA extracts and cassettes for scientific and industrial purposes; enzymes for use in the preparation and creation of DNA extracts and cassettes for scientific and industrial purposes; DNA extracts and cassettes for scientific and research use; DNA extracts for scientific and research purposes in the fields of agri-biotechnology and nanotechnology; biological and biochemical reagents being RNA polymerase, DNA polymerase, DNA plasmid vectors, DNA vectors, rAAV vectors, and RNA markers that form a gene analysis system not for medical purposes; diagnostic reagents for scientific or research purposes, namely, a diagnostic kit consisting primarily of DNA plasmid vectors
01 - Chemical and biological materials for industrial, scientific and agricultural use
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Chemical preparations, enzymes, DNA vectors, and DNA extracts for the preparation of nucleic acid for scientific and research purposes; chemicals for use in the preparation and creation of DNA extracts and cassettes for scientific and industrial purposes; enzymes for use in the preparation and creation of DNA extracts and cassettes for scientific and industrial purposes; DNA extracts and cassettes for scientific and research use; DNA extracts for scientific and research purposes in the fields of agri-biotechnology and nanotechnology; Biological and biochemical reagents being RNA polymerase, DNA polymerase, DNA plasmid vectors, DNA vectors, rAAV vectors, and RNA markers that form a gene analysis system not for medical purposes; Diagnostic reagents for scientific or research purposes, namely, a diagnostic kit consisting primarily of DNA plasmid vectors Vaccines; DNA based vaccines; DNA extracts, namely, nucleic acid sequences and chemical reagents for medical use; Therapeutic DNA, namely, nucleic acid sequences and chemical reagents for medical and veterinary purposes; Biotechnological product, namely, viral DNA vectors, for the delivery of genes and other materials in the nature of RNA and transcriptional targeting promoters, to a biological cell for medical purposes; biological preparations, namely, DNA extracts for production of viral vectors for medical purposes Scientific and technological services, namely, research and design in the field of DNA research and development; chemical and biological analysis and research services; scientific research in the field of DNA; scientific research and development in the field of creating DNA extracts; scientific and technological services, namely, scientific research, analysis, design and development in the field of DNA based pharmaceutical drugs and therapies; scientific research and development in the fields of gene therapy, diagnostics, agri-biotechnology and nanotechnology; scientific consultancy services in the fields of gene therapy, diagnostics, agri-biotechnology, nano technology, and DNA analysis
THE UNIVERSITY OF NORTH CAROLINA CHAPEL HILL (USA)
Inventor
Childers, Martin K.
Yanez-Cuna, Jorge Omar
Iglesias Gonzalez, Juan Manuel
Cooper, Sinclair
Evripioti, Antonia
Roberts, Michael L.
Tretiakova, Anna
Suarez, Lester
Nguyen, Anh
Low, Siewhui
Xiao, Xiao
Abstract
Disclosed herein are various optimized nucleic acids encoding the fukutin-related protein (FKRP). Recombinant vectors comprising the optimized nucleic acid (e.g. operatively linked to a muscle specific promoter), such as recombinant adeno-associated virus vectors, for expressing the protein (e.g. in skeletal and cardiac muscle), and therapeutic compositions contains the vectors are also disclosed. Therapeutic methods of administration of the vectors to a subject for the treatment of a subject with a dystroglycanopathy disorder (e.g., limb-girdle muscular dystrophy 2I) are also disclosed.
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
71.
THERAPEUTIC ADENO-ASSOCIATED VIRUS DELIVERY OF FUKUTIN RELATED PROTEIN (FKRP) FOR TREATING DYSTROGLYCANOPATHY DISORDERS INCLUDING LIMB GIRDLE 2I (LGMD2I)
THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL (USA)
ASKLEPIOS BIOPHARMACEUTICAL, INC. (USA)
Inventor
Childers, Martin K.
Yanez-Cuna, Jorge Omar
Iglesias Gonzalez, Juan Manuel
Cooper, Sinclair
Evripioti, Antonia
Roberts, Michael L.
Tretiakova, Anna
Suarez, Lester
Nguyen, Anh
Low, Siewhui
Xiao, Xiao
Abstract
Disclosed herein are various optimized nucleic acids encoding the fukutin-related protein (FKRP). Recombinant vectors comprising the optimized nucleic acid (e.g. operatively linked to a muscle specific promoter), such as recombinant adeno-associated virus vectors, for expressing the protein (e.g. in skeletal and cardiac muscle), and therapeutic compositions contains the vectors are also disclosed. Therapeutic methods of administration of the vectors to a subject for the treatment of a subject with a dystroglycanopathy disorder (e.g., limb-girdle muscular dystrophy 2I) are also disclosed.
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
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
Medical devices, namely, vial access system consisting primarily of a docking tray that facilitates direct access to contents of multiple vials containing pharmaceutical preparations for patient infusion.
Medical devices, namely, vial access system consisting primarily of a docking tray that facilitates direct access to contents of multiple vials containing pharmaceutical preparations for patient infusion
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Medical services, namely, cellular starter material collection services featuring human blood-derived biological raw materials that are eventually processed for biopharmaceutical uses and disease treatments
Aspects of the disclosure relate to compositions and methods useful for treating neurological diseases and disorders. In some embodiments, the disclosure provides a method for treating a neurological disease or disorder comprising administration of both a viral vector comprising interfering nucleic acids (e.g., artificial miRNAs) and a viral vector comprising a CYP46A1 protein. In some embodiments, the disclosure provides a method for treating Huntington's disease comprising administration of both a viral vector comprising interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and a viral vector comprising a CYP46A1 protein. In some embodiments, the viral vector comprises a modified viral capsid, such as for preferentially targeting cells in the CNS or PNS.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61P 25/00 - Drugs for disorders of the nervous system
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
Aspects of the disclosure relate to compositions and methods useful for treating neurological diseases and disorders. In some embodiments, the disclosure provides a method for treating a neurological disease or disorder comprising administration of both a viral vector comprising interfering nucleic acids (e.g., artificial miRNAs) and a viral vector comprising a CYP46A1 protein. In some embodiments, the disclosure provides a method for treating Huntington's disease comprising administration of both a viral vector comprising interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and a viral vector comprising a CYP46A1 protein. In some embodiments, the viral vector comprises a modified viral capsid, such as for preferentially targeting cells in the CNS or PNS.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61P 25/00 - Drugs for disorders of the nervous system
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
The present invention relates to regulatory nucleic acid sequences, in particular NS-specific promoters, cell specific promoters, multi-cell specific promoters, and elements thereof. The invention also relates to expression constructs, vectors, virions, pharmaceutical compositions and cells comprising such promoters and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications.
The present invention relates to regulatory nucleic acid sequences, in particular NS-specific promoters, cell specific promoters, multi-cell specific promoters, and elements thereof. The invention also relates to expression constructs, vectors, virions, pharmaceutical compositions and cells comprising such promoters and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications.
The present invention relates to methods of administration of rAAV vectors in a single administration method comprising a series of sub-administrations of sub-doses of rAAV vectors. The present invention also relates to rAAV vectors comprising cardiac-specific promoters, cardiac-cell specific promoters, multi-cell cardiac specific promoters, and elements thereof. The invention also relates to rAAV vectors, pharmaceutical compositions and uses thereof in methods for treating cardiovascular disease, heart diseases and heart failure in subjects in need thereof.
The present invention relates to methods of administration of rAAV vectors in a single administration method comprising a series of sub-administrations of sub-doses of rAAV vectors. The present invention also relates to rAAV vectors comprising cardiac-specific promoters, cardiac-cell specific promoters, multi-cell cardiac specific promoters, and elements thereof. The invention also relates to rAAV vectors, pharmaceutical compositions and uses thereof in methods for treating cardiovascular disease, heart diseases and heart failure in subjects in need thereof.
A liquid handling robot has a worktable that supports a rack holding a set of pipette tips. The liquid handling robot also has an arm that is operably suspended above the worktable, where the arm includes a tip receiver that is configured to engage the set of pipette tips. A controller of the liquid handling robot is configured to raise the tip receiver away from the worktable to withdraw the engaged set of pipette tips from the rack. A sensor is fixed relative to the worktable and is operable to emit a beam. A microcontroller monitors the sensor with the arm in a checking position to determine if the rack interrupts in the beam to indicate that the rack stuck to the pipette tips, which is autonomously resolved by the liquid handling robot performing a corrective action.
The present invention relates to regulatory nucleic acid sequences, in particular liver-specific cis-regulatory elements, cis-regulatory modules, promoters and other such nucleic acid sequences, that are capable of enhancing liver-specific expression of genes. The invention also relates to expression constructs, vectors and cells comprising such liver-specific regulatory nucleic acid sequences, and to methods of their use. The liver-specific regulatory nucleic acid sequences are of particular utility for gene therapy applications, but also find utility in other areas such as bioprocessing and biotechnology.
A filter press device includes a supporting frame and elongated side rails disposed parallel and horizontally on the supporting frame. A following head assembly is disposed on an end portion of the supporting frame, and a following head assembly is movably disposed on another end portion of the supporting frame. The elongated side rails support a combination of filter plates of varying sizes through one or more adapters. An adapter plate interposes between adjacent filter plates of different sizes, providing fluidic communication between the different sized filter plates.
A medicinal fluid pooling device may be used to pool multiple containers of medicinal fluid to facilitate administration of the medicinal fluid to a patient. A medicinal pooling device may include spikes covered by spike sheaths which are pierced when a container of medicinal fluid is inserted into the medicinal pooling device. The medicinal pooling device may also include a cover configured to cover the spikes. The medicinal pooling device may also include a fluidic interface which may be used to fluidly connect the medicinal pooling device to an infusion pump or syringe.
The present invention relates to regulatory nucleic acid sequences, in particular CNS- specific promoters, and elements thereof. The invention also relates to expression constructs, vectors, virions, pharmaceutical compositions and cells comprising such promoters and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications.
The present invention relates to regulatory nucleic acid sequences, in particular CNS- specific promoters, and elements thereof. The invention also relates to expression constructs, vectors, virions, pharmaceutical compositions and cells comprising such promoters and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications.
Described is a method for preparing an Immunoglobulin G (IgG) enriched fraction from a Cl- INH depleted plasma supernatant. Isolation of Immunoglobulin G (IgG) enriched fraction from a Cl-INH depleted plasma supernatant provided an alternative starting material for the manufacturing process. In the present invention, Cl-INH depleted plasma supernatant is treated with heparin before further processing.
Aspects described herein relate to stable cell line for recombinant viral vector (e.g., recombinant adeno associated viral vector) production comprising at least one inducible promoter operatively linked to a heterologous gene that encodes a toxic protein. Methods for making stable cell lines, and methods for viral vector production are further provided herein.
Aspects described herein relate to stable cell line for recombinant viral vector (e.g., recombinant adeno associated viral vector) production comprising at least one inducible promoter operatively linked to a heterologous gene that encodes a toxic protein. Methods for making stable cell lines, and methods for viral vector production are further provided herein.
In some embodiments, a reconstitution or medicinal fluid delivery device includes a transfer engine including two fluidly connected spikes, each configured to pierce a container. A check valve may be disposed between the two spikes to allow unidirectional flow from one container to the other. Physical access to a fluid outlet may be obstructed by a housing until the reconstitution or medicinal fluid delivery device is actuated, whereupon physical access to the fluid outlet is permitted. The reconstitution or medicinal fluid delivery device may be placed on a flat surface and actuated with force applied in a single direction toward the flat surface.
Disclosed herein are methods of treating a toxicity in a subject receiving recombinant viral vector, such as a recombinant adeno-associated viral AAV (rAAV) vector comprising co-administration of an antibiotic and a viral vector. In one embodiment, the antibiotic is a tetracycline or macrolide family member. Also provided herein are compositions comprising an antibiotic and a viral vector.
The present invention relates to a method for treatment of menorrhagia in a subject with von Willebrand Disease (VWD) comprising administering a therapeutic amount of recombinant von Willebrand Factor (rVWF) to the subject.
The present invention provides, among other aspects, methods for the treatment of Alzheimer's disease in a subject in need thereof, the method including administration of a therapeutically effective amount of a pooled human immunoglobulin G (IgG) composition to a subject with moderately severe Alzheimer's disease, a subject carrying an ApoE4 allele, or both, where the amount of pooled human IgG is from 300 mg/kg to 800 mg/kg body weight of the subject per two week period, and where the amount is administered in one or more doses during the two week period after initiation of a therapeutic regimen. Also provided, are methods for selecting a treatment regimen for a subject with Alzheimer's disease, including diagnosing the severity of the Alzheimer's disease, determining if the subject carries an APOE4 allele, or both, and assigning a treatment regimen including administration of pooled human immunoglobulin G and/or an anti-beta amyloid monoclonal antibody.
C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
A61K 38/47 - Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
C07K 16/06 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies from serum
C07K 16/00 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
