A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61K 9/00 - Medicinal preparations characterised by special physical form
Methods of analyzing adeno associated virus (AAV) genomes and use thereof are provided herein. The methods and uses include the methods of preparing an AAV genomes for sequenc-ing, and methods of analyzing and characterizing AAV genomes. For example, the AAV analy-sis methods include steps of aligning reference nucleotide sequences to sequence reads of AAV genomes; determining alignment scores of each reference nucleotide sequence to the sequence reads and identifying strand types of the AAV genomes based on the alignment scores; determin-ing first and second coordinates within the sequence reads based on alignments with the refer-ence nucleotide sequences used to identify strand type; and analyzing sequences adjacent to the first and second coordinates to identify ITR configurations of the AAV genomes.
3.
HIGH THROUGHPUT SCREEN FOR GENETIC VARIANTS ASSOCIATED WITH SHORT STATURE
The present disclosure, relates, in general, to use of a high throughput screening method for identifying genetic mutations in genes, e.g., the natriuretic peptide receptor 2 (NPR2) gene, associated with CNP dysfunction and short stature disorders, and methods of treatment of short stature disorders.
C12Q 1/6897 - Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids involving reporter genes operably linked to promoters
C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
The disclosure provides various compositions comprising novel adeno-associated virus (AAV) capsid sequences and functional fragments thereof. Also provided, are methods of delivery, treatment and manufacture using the compositions provided by the disclosure.
C12N 7/00 - Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/005 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
The disclosure relates to methods of treating hemophilia patients with Factor VIII (FVIII) adeno-associated virus (AAV), wherein the patients have pre-existing AAV antibodies or require re-administration or redosing of a gene therapy vector. The disclosure also provides for methods of treating hemophilia patients with active FVIII inhibitors or patients that have undergone seroconversion to AAV5 capsid proteins.
A61K 38/16 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61K 31/573 - Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
The present disclosure provides for use of variants of C-type natriuretic peptide (CNP), and novel pharmaceutical compositions and formulations comprising CNP variant peptides for the treatment of skeletal dysplasias, one or more symptoms of skeletal dysplasias, such as long bone growth or growth velocity, and other disorders having a skeletal dysplasia and/or CNP-associated symptom or component.
A61K 47/12 - Carboxylic acids; Salts or anhydrides thereof
A61K 47/18 - Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
A61K 47/20 - Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing sulfur, e.g. dimethyl sulfoxide [DMSO], docusate, sodium lauryl sulfate or aminosulfonic acids
A61K 47/26 - Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
Provided herein are antisense oligonucleotides (ASOs) that induce skipping of exon 53 of human dystrophin pre-mRNA, and pharmaceutically acceptable derivatives thereof. Also provided are pharmaceutical compositions containing the ASOs and methods of using the ASOs and compositions for treating a subject with Duchenne muscular dystrophy.
The disclosure provides various compositions comprising novel adeno-associated virus (AAV) capsid sequences and functional fragments thereof. Also provided, are methods of delivery, treatment and manufacture using the compositions provided by the disclosure.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/005 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
C12N 7/00 - Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
The disclosure provides various compositions comprising novel adeno-associated virus (AAV) capsid sequences and functional fragments thereof. Also provided, are methods of delivery, treatment and manufacture using the compositions provided by the disclosure.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/005 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
Provided herein are antisense oligonucleotides (ASOs) that induce skipping of exon 53 of human dystrophin pre-mRNA, and pharmaceutically acceptable derivatives thereof. Also provided are pharmaceutical compositions containing the ASOs and methods of using the ASOs and compositions for treating a subject with Duchenne muscular dystrophy.
Provided herein are antisense oligonucleotides (ASOs) and pharmaceutically acceptable derivatives thereof that modulate expression of STXBP1 and have activity in treating STXBP1 disorders. Also provided are pharmaceutical compositions containing the ASOs and methods of using the ASOs for treating a subject with an STXBP1 disorder.
09 - Scientific and electric apparatus and instruments
Goods & Services
Downloadable computer application software for mobile
phones, namely, software for patients to access information
related to and maintain compliance with treatment of genetic
diseases and disorders and the use of enzyme therapy.
09 - Scientific and electric apparatus and instruments
Goods & Services
Downloadable computer application software for mobile
phones, namely, software for patients to access information
related to and maintain compliance with treatment of genetic
diseases and disorders and the use of enzyme therapy.
09 - Scientific and electric apparatus and instruments
Goods & Services
Downloadable computer application software for mobile
phones, namely, software for patients to access information
related to and maintain compliance with treatment of genetic
diseases and disorders and the use of enzyme therapy.
09 - Scientific and electric apparatus and instruments
Goods & Services
Downloadable computer application software for mobile
phones, namely, software for patients to access information
related to and maintain compliance with treatment of genetic
diseases and disorders and the use of enzyme therapy.
16.
COMPOSITIONS OF BETA-HEXOSAMINIDASE VARIANTS AND USES THEREOF
Disclosed herein are recombinant β-hexosaminidase variant a subunits that form a β-hexosaminidase variant a subunit homodimer that have optimized properties for use in treating Tay-Sachs disease or Sandhoff Disease.
The invention relates to novel adeno-associated virus (AAV) capsid proteins, AAV particles comprising a novel capsid protein, polynucleotides encoding these capsid proteins and AAV vectors expressing these capsid proteins. The invention also relates to methods of making the herein described AAV vectors expressing the novel capsid proteins of the invention and associated therapeutic uses of thereof.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
The present disclosure provides for use of variants of C-type natriuretic peptide (CNP), and novel pharmaceutical compositions and formulations comprising CNP variant peptides for the treatment of skeletal dysplasias, one or more symptoms of skeletal dysplasias, such as long bone growth or growth velocity, and other disorders having a skeletal dysplasia and/or CNP-associated symptom or component.
A61K 47/12 - Carboxylic acids; Salts or anhydrides thereof
A61K 47/18 - Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
A61K 47/20 - Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing sulfur, e.g. dimethyl sulfoxide [DMSO], docusate, sodium lauryl sulfate or aminosulfonic acids
A61K 47/26 - Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
19.
STABLE EXPRESSION OF AAV VECTORS IN JUVENILE SUBJECTS
The invention relates to the use of adeno-associated virus (AAV) vectors to achieve long term expression of a transgene in the liver of a juvenile subject. The invention includes the stable long-term amelioration of disease symptoms of the subjection following a single administration of an AAV vector to a juvenile subject, wherein the AAV vector delivers the transgene to the subject's liver.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61K 9/00 - Medicinal preparations characterised by special physical form
The present disclosure relates in general to therapeutic lysosomal enzyme fusion proteins useful for treating lyso-somal storage diseases, liquid formulations comprising such fusion proteins and associated methods useful for treating lysosomal storage diseases in mammals.
A61K 47/26 - Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
A61K 47/65 - Peptidic linkers, binders or spacers, e.g. peptidic enzyme-labile linkers
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
The disclosure provides for methods of administering an epigenetic modifier, such as a histone deacetylase (HDAC) inhibitors, to improve transgene expression and durability of adeno-associated virus gene therapy.
C07K 14/47 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from humans from vertebrates from mammals
A61K 31/165 - Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide
A61K 31/167 - Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide having the nitrogen atom of a carboxamide group directly attached to the aromatic ring, e.g. lidocaine, paracetamol
The present disclosure relates, in general, to methods for readministering, or redosing, a subject having undergone a first gene therapy regimen with a second, or subsequent, administration of a gene therapy regimen, wherein the first gene therapy vector and second gene therapy vector comprise different AAV capsids but carry a transgene or polynucleotide useful to treat the same disease or disorder.
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
45 - Legal and security services; personal services for individuals.
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Medical information; Providing health information; Providing medical information in the field of rare diseases; Providing on-line information, news and commentary in the field of health and wellness relating to rare diseases; Maintaining patient medical records and files; Providing information to patients in the field of administering medications; Prescription refill reminder services Providing patient advocate and case management services, namely, coordinating the procurement and administration of medication; Investigation services related to insurance claims; Providing live, one-on-one personal support services for patients and families of patients with rare diseases, namely, providing counseling and support, and making personal arrangements for health care Pharmaceutical preparations for use in enzyme therapy; pharmaceutical preparations for use in treatment of genetic disorders
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
45 - Legal and security services; personal services for individuals.
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Medical information; Providing health information; Providing medical information in the field of rare diseases; Providing on-line information, news and commentary in the field of health and wellness relating to rare diseases; Maintaining patient medical records and files; Providing information to patients in the field of administering medications; and Prescription refill reminder services Providing patient advocate and case management services, namely, coordinating the procurement and administration of medication; and Investigation services related to insurance claims; Providing live, one-on-one personal support services for patients and families of patients with rare diseases, namely, providing counseling and support, and making personal arrangements for health care Pharmaceutical preparations for use in enzyme therapy; pharmaceutical preparations for use in treatment of genetic disorders
25.
Use of C-Type Natriuretic Peptide Variants to Treat Skeletal Dysplasia
The present disclosure provides for use of variants of C-type natriuretic peptide (CNP), and novel pharmaceutical compositions and formulations comprising CNP variant peptides for the treatment of skeletal dysplasias, one or more symptoms of skeletal dysplasias, such as long bone growth or growth velocity, and other disorders having a skeletal dysplasia and/or CNP-associated symptom or component.
A61K 47/12 - Carboxylic acids; Salts or anhydrides thereof
A61K 47/18 - Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
A61K 47/20 - Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing sulfur, e.g. dimethyl sulfoxide [DMSO], docusate, sodium lauryl sulfate or aminosulfonic acids
A61K 47/26 - Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
26.
USE OF HISTIDINE RICH PEPTIDES AS A TRANSFECTION REAGENT FOR rAAV AND rBV PRODUCTION
The present invention provides methods, compositions, and kits for preparing and using adeno associated virus and baculovirus. The methods for producing adeno associated virus and baculovirus particles include using histidine rich peptides and other cationic peptides as transfection reagents. The adeno associated virus are pseudotyped with capsids, in particular for use in gene therapy and/or diagnostics. The baculovirus are also used to prepare adeno associated virus.
The present invention provides modified alphaviruses and compositions, methods, and kits for preparing and using, in particular AAV viral particles pseudotyped with capsids, in particular for use in gene therapy and/or diagnostics.
Provided herein are gene therapy compositions and methods of treating reduced levels of functional cardiac myosin binding protein C in a subject having hypertrophic cardiomyopathy.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61P 9/00 - Drugs for disorders of the cardiovascular system
C07K 14/47 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from humans from vertebrates from mammals
29.
TREATMENT OF ARRHYTHMOGENIC CARDIOMYOPATHY WITH AAV GENE THERAPY VECTORS
Provided herein are gene therapy compositions and methods of treating loss of cardiomyocyte alignment due to reduced levels of functional plakophilin-2 protein in a subject having arrhythmogenic cardiomyopathy. Also provided herein are gene therapy vector components and methods to be used in gene therapy for improving cardiac expression of gene therapy products.
C07K 14/44 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from humans from protozoa
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
09 - Scientific and electric apparatus and instruments
Goods & Services
Downloadable computer application software for mobile phones, namely, software for patients to access information related to and maintain compliance with treatment of genetic diseases and disorders and the use of enzyme therapy
09 - Scientific and electric apparatus and instruments
Goods & Services
Downloadable computer application software for mobile phones, namely, software for patients to access information related to and maintain compliance with treatment of genetic diseases and disorders and the use of enzyme therapy
09 - Scientific and electric apparatus and instruments
Goods & Services
Downloadable computer application software for mobile phones, namely, software for patients to access information related to and maintain compliance with treatment of genetic diseases and disorders and the use of enzyme therapy
09 - Scientific and electric apparatus and instruments
Goods & Services
Downloadable computer application software for mobile phones, namely, software for patients to access information related to and maintain compliance with treatment of genetic diseases and disorders and the use of enzyme therapy
34.
COMPOSITIONS AND METHODS FOR TREATING LONG QT SYNDROME
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.
A61K 47/26 - Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C12N 7/00 - Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
G01N 33/68 - Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
Provided herein are phenylalanine ammonia-lyase (PAL) variants produced by prokaryotes, wherein such prokaryotic PAL variant has a greater phenylalanine-converting activity and/or a reduced immunogenicity as compared to a wild-type PAL. Further provided are compositions of prokaryotic PAL and biologically active fragments, mutants, variants or analogs thereof, as well as methods for the production, purification, formulation, and use of such compositions for industrial and therapeutic purposes, e.g., treating hyperphenylalaninemia, including phenylketonuria, and other disorders, including cancer.
The present invention provides process for enriching adeno-associated virus particles using anion exchange chromatography and zonal ultracentrifugation.
Provided herein are antisense oligonucleotides (AONs) that induce skipping of exon 51 of human dystrophin pre-mRNA, and pharmaceutically acceptable derivatives thereof. Also provided are pharmaceutical compositions containing the AONs and methods of using the AONs and compositions for treating a subject with Duchenne muscular dystrophy.
Provided herein are compositions and methods for treating a Cl esterase inhibitor deficiency by normalizing levels of the Cl esterase inhibitor protein in a subject having HAE.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
The invention relates to novel adeno-associated virus (AAV) capsid proteins, AAV particles comprising a novel capsid protein, polynucleotides encoding these capsid proteins and AAV vectors expressing these capsid proteins. The invention also relates to methods of making the herein described AAV vectors expressing the novel capsid proteins of the invention and associated therapeutic uses of thereof.
Provided herein are antisense oligonucleotides (AONs) that induce skipping of exon 51 of human dystrophin pre-mRNA, and pharmaceutically acceptable derivatives thereof. Also provided are pharmaceutical compositions containing the AONs and methods of using the AONs and compositions for treating a subject with Duchenne muscular dystrophy.
The present invention provides further improved compositions and methods for efficient lysosomal targeting based on the GILT technology. Among other things, the present invention provides methods and compositions for targeting lysosomal enzymes to lysosomes using furin-resistant lysosomal targeting peptides. The present invention also provides methods and compositions for targeting lysosomal enzymes to lysosomes using a lysosomal targeting peptide that has reduced or diminished binding affinity for the insulin receptor.
A61K 47/54 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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 compound
A61P 19/08 - Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
49.
C-TYPE NATRIURETIC PEPTIDE THERAPY OF BONE-RELATED DISORDERS
The present disclosure relates, in general, to measures of efficacy in patients receiving C-type natriuretic peptide (CNP) therapy to treat a skeletal dysplasia, short stature or bone-related disorders.
The present disclosure relates, in general, to measures of efficacy in patients receiving C-type natriuretic peptide (CNP) therapy to treat a skeletal dysplasia, short stature or bone-related disorders.
The present disclosure provides for use of variants of C-type natriuretic peptide (CNP), for the treatment of skeletal dysplasias in children, and improvement in one or more symptoms of skeletal dysplasias, such as long bone growth or growth velocity, and use in other disorders having a skeletal dysplasia and/or CNP-associated symptom or component.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
The disclosure provides various compositions comprising novel adeno-associated virus (AAV) capsid sequences and functional fragments thereof. Also provided, are methods of delivery, treatment and manufacture using the compositions provided by the disclosure.
The disclosure provides various compositions comprising novel adeno-associated virus (AAV) capsid sequences and functional fragments thereof. Also provided, are methods of delivery, treatment and manufacture using the compositions provided by the disclosure.
The disclosure provides various compositions comprising novel adeno-associated virus (AAV) capsid sequences and functional fragments thereof. Also provided, are methods of delivery, treatment and manufacture using the compositions provided by the disclosure.
The invention is directed to methods of removing adventitious virus from cells and cell lines and virus-free cells and cell lines obtainable by such methods.
The disclosure provides various compositions comprising novel adeno-associated virus (AAV) capsid sequences and functional fragments thereof. Also provided, are methods of delivery, treatment and manufacture using the compositions provided by the disclosure.
The disclosure provides various compositions comprising novel adeno-associated virus (AAV) capsid sequences and functional fragments thereof. Also provided, are methods of delivery, treatment and manufacture using the compositions provided by the disclosure.
The disclosure provides various compositions comprising novel adeno-associated virus (AAV) capsid sequences and functional fragments thereof. Also provided, are methods of delivery, treatment and manufacture using the compositions provided by the disclosure.
The present invention provides processes for producing and characterizing adenoassociated virus particles and baculovirus particles. The present invention is directed to methods of improving adeno associated virus (AAV) production. The present invention addresses the problems associated with the production of rAAV using baculovirus infected Sf9 cells and achieves an improved method for producing rAAV. The present invention developed different methods for producing recombinant baculovirus (rBV) and recombinant adeno-associated virus (rAA V). These methods address issues such as genome instability and also result in improved production of rAA V, as well as produce rAA V with improved properties.
C12N 5/10 - Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
64.
LARGE SCALE ADENO-ASSOCIATED VIRUS PRODUCTION SYSTEMS
The present invention provides processes for producing and characterizing adenoassociated virus particles and baculovirus particles. The present invention is directed to methods of improving adeno associated virus (AAV) production. The present invention addresses the problems associated with the production of rAAV using baculovirus infected Sf9 cells and achieves an improved method for producing rAAV. The present invention developed different methods for producing recombinant baculovirus (rBV) and recombinant adeno-associated virus (rAA V). These methods address issues such as genome instability and also result in improved production of rAA V, as well as produce rAA V with improved properties.
C12N 5/10 - Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
The present disclosure provides for use of variants of C-type natriuretic peptide (CNP), for the treatment of skeletal dysplasias in children, and improvement in one or more symptoms of skeletal dysplasias, such as long bone growth or growth velocity, and use in other disorders having a skeletal dysplasia and/or CNP-associated symptom or component.
A61K 47/52 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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 inorganic compound, e.g. an inorganic ion that is complexed with the active ingredient
A61P 19/08 - Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
66.
C-TYPE NATRIURETIC PEPTIDE VARIANTS TO TREAT SKELETAL DYSPLASIA IN CHILDREN
The present disclosure provides for use of variants of C-type natriuretic peptide (CNP), for the treatment of skeletal dysplasias in children, and improvement in one or more symptoms of skeletal dysplasias, such as long bone growth or growth velocity, and use in other disorders having a skeletal dysplasia and/or CNP-associated symptom or component.
A61K 47/52 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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 inorganic compound, e.g. an inorganic ion that is complexed with the active ingredient
A61P 19/08 - Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
67.
CELL CULTURE FEED FOR RECOMBINANT ADENO-ASSOCIATED VIRUS PRODUCTION IN MAMMALIAN CELLS
Various recombinant gene therapy virus production culture mediums are disclosed having a nicotinamide concentration of 1 mM or more or components that increase nicotinamide adenine dinucleotide biosynthesis or decrease nicotinamide adenine dinucleotide degradation. Various feed formulations are disclosed that are used to supplement cell culture mediums such that the cell culture mediums have a nicotinamide concentration of 1 mM or more for a predetermined time or have components that increase nicotinamide adenine dinucleotide biosynthesis or decrease nicotinamide adenine dinucleotide degradation. Additionally, methods of generating gene therapy viruses such as recombinant adeno-associated virus using the various cell culture mediums and feed formulations are disclosed.
C12N 1/00 - Microorganisms, e.g. protozoa; Compositions thereof; Processes of propagating, maintaining or preserving microorganisms or compositions thereof; Processes of preparing or isolating a composition containing a microorganism; Culture media therefor
Disclosed herein are recombinant β-hexosaminidase variant α subunits that form a β-hexosaminidase variant α subunit homodimer that have optimized properties for use in treating Tay-Sachs disease or Sandhoff Disease.
C12N 9/24 - Hydrolases (3.) acting on glycosyl compounds (3.2)
A61K 38/47 - Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
The invention relates to novel adeno-associated virus (AAV) capsid proteins, AAV particles comprising a novel capsid protein, polynucleotides encoding these capsid proteins and AAV vectors expressing these capsid proteins. The invention also relates to methods of making the herein described AAV vectors expressing the novel capsid proteins of the invention and associated therapeutic uses of thereof.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
Phenylalanine ammonia-lyase (PAL) variants with a greater phenylalanine-converting activity and/or a reduced immunogenicity as compared to a wild-type PAL for therapeutic uses, including the treatment of adolescent subjects having PKU.
Containers are provided herein for facilitating freezing, thawing, and/or storage of products that may be perishable, such as pharmaceuticals. The containers can include shells that enclose the product, and that include apertures permitting flow of a fluid through the containers' internal volume. The temperature of the fluid can be selected so as to freeze or thaw the product. The container optionally can include pressure building members that control the flow of the fluid through the containers' inner volume, e.g., increase the fluid flow rate and thus increase the rate with which the fluid is frozen or thawed.
F25D 25/00 - Charging, supporting, or discharging the articles to be cooled
F25D 15/00 - Devices associated with refrigerating machinery not covered by group or , e.g. non-self-contained movable devices
F25D 17/06 - Arrangements for circulating cooling fluids; Arrangements for circulating gas, e.g. air, within refrigerated spaces for circulating gas, e.g. by natural convection by forced circulation
F25D 3/10 - Devices using other cold materials; Devices using cold-storage bodies using liquefied gases, e.g. liquid air
B65D 1/38 - Baskets or like containers of skeleton or apertured construction
72.
Compositions of prokaryotic phenylalanine ammonia-lyase and methods of treating adolescent subjects
Phenylalanine ammonia-lyase (PAL) variants with a greater phenylalanine-converting activity and/or a reduced immunogenicity as compared to a wild-type PAL for therapeutic uses, including the treatment of adolescent subjects having PKU.
The present invention provides further improved compositions and methods for efficient lysosomal targeting based on the GILT technology. Among other things, the present invention provides methods and compositions for targeting lysosomal enzymes to lysosomes using furin-resistant lysosomal targeting peptides. The present invention also provides methods and compositions for targeting lysosomal enzymes to lysosomes using a lysosomal targeting peptide that has reduced or diminished binding affinity for the insulin receptor.
This disclosure relates to the use of size exclusion chromatography and/or size exclusion chromatography with multi-angle light scattering technology to characterize viral particles such as adeno-associated virus and lentivirus particles. The disclosed methods are also useful for estimating the titer of viral particles, determining the integrity of the viral particles and estimating the amount of DNA encapsidated in the viral particle.
The present disclosure, relates, in general, to hydrophobic salts of hydrophilic peptides that form low solubility materials in aqueous solutions and are capable of extended or sustained release of the peptide component when administered to a subject. Hydrophobic salts of C-type natriuretic peptides and uses thereof are also disclosed.
A61K 47/52 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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 inorganic compound, e.g. an inorganic ion that is complexed with the active ingredient
A61K 47/54 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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 compound
A61K 9/00 - Medicinal preparations characterised by special physical form
Provided herein are methods of treating Neuronal Ceroid Lipofuscinosis (CLN2) disease in a subject less than 3 years old. In exemplary embodiments, the method comprises administering to the subject a formulation comprising recombinant human tripeptidyl peptidase-1 (rhTPP1) in an amount effective to treat the CLN2 disease in the subject. Also provided are methods of delaying the onset of CLN2 disease, or a symptom thereof, in a subject less than 3 years old. In exemplary embodiments, the method comprises administering to the subject a formulation comprising recombinant human tripeptidyl peptidase-1 (rhTPP1) in an amount effective to delay the onset of the CLN2 disease or symptom in the subject.
A61K 9/00 - Medicinal preparations characterised by special physical form
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
77.
USE OF HISTIDINE RICH PEPTIDES AS A TRANSFECTION REAGENT FOR RAAV AND RBV PRODUCTION
The present invention provides methods, compositions, and kits for preparing and using adeno associated virus and baculovirus. The methods for producing adeno associated virus and baculovirus particles include using histidine rich peptides and other cationic peptides as transfection reagents. The adeno associated virus are pseudotyped with capsids, in particular for use in gene therapy and/or diagnostics. The baculovirus are also used to prepare adeno associated virus.
The present invention provides methods, compositions, and kits for preparing and using adeno associated virus and baculovirus. The methods for producing adeno associated virus and baculovirus particles include using histidine rich peptides and other cationic peptides as transfection reagents. The adeno associated virus are pseudotyped with capsids, in particular for use in gene therapy and/or diagnostics. The baculovirus are also used to prepare adeno associated virus.
The present invention provides modified alphaviruses and compositions, methods, and kits for preparing and using, in particular AAV viral particles pseudotyped with capsids, in particular for use in gene therapy and/or diagnostics.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
The disclosure relates to the use of variants of C-type natriuretic peptide (CNP) to treat osteoarthritis, to ameliorate one or more symptoms of osteoarthritis, and to treat disorders having an osteoarthritis component.
A61P 19/02 - Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
A61K 9/19 - Particulate form, e.g. powders lyophilised
A61K 31/573 - Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
The present invention provides process for enriching adeno-associated virus particles using anion exchange chromatography and zonal ultracentrifugation.
The present invention provides process for enriching adeno-associated virus particles using anion exchange chromatography and zonal ultracentrifugation.
This disclosure relates to the use of size exclusion chromatography and/or size exclusion chromatography with multi-angle light scattering technology to characterize viral particles such as adeno-associated virus and lentivirus particles. The disclosed methods are also useful for estimating the titer of viral particles, determining the integrity of the viral particles and estimating the amount of DNA encapsidated in the viral particle.
C12Q 1/70 - Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving virus or bacteriophage
C12N 7/00 - Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
C12Q 1/68 - Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
85.
USE OF REGULATORY PROTEINS FOR THE PRODUCTION OF ADENO-ASSOCIATED VIRUS
The present disclosure provides method and host cell constructs for the production of infectious virions of adeno-associated virus (AAV) in host cells modified to include a nucleotide sequence encoding a regulatory protein and an expression control element operably linked to the nucleotide sequence. The host cells are transformed with recombinant polynucleotide constructs so that the cells produce AAV capsid proteins, large and small AAV Rep proteins, one or more regulatory protein proteins, and helper proteins. The disclosure further provides for the production and formulation of insect-cell-derived AAV into a pharmaceutical composition and the medical use of the composition to achieve gene-therapy.
A61K 47/26 - Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
A61K 47/34 - Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
Provided herein are compositions and methods of treating an α-galactosidase A deficiency by normalizing levels of α-galactosidase A protein in a subject having Fabry Disease.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C12N 9/40 - Hydrolases (3.) acting on glycosyl compounds (3.2) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase
The invention is directed to methods of removing adventitious virus from cells and cell lines and virus-free cells and cell lines obtainable by such methods.
This invention provides compositions of active highly phosphorylated lysosomal sulfatase enzymes, their pharmaceutical compositions, methods of producing and purifying such lysosomal sulfatase enzymes and compositions and their use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the lysosomal sulfatase enzyme.
Described herein are compounds, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or disorders associated with the enzyme glycolate oxidase (GO). Such diseases or disorders include, for example, disorders of glyoxylate metabolism, including primary hyperoxaluria, that are associated with production of excessive amounts of oxalate.
C07D 417/12 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group containing two hetero rings linked by a chain containing hetero atoms as chain links
C07D 409/12 - Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing two hetero rings linked by a chain containing hetero atoms as chain links
C07D 405/12 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a chain containing hetero atoms as chain links
C07D 403/12 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing two hetero rings linked by a chain containing hetero atoms as chain links
C07D 401/12 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings linked by a chain containing hetero atoms as chain links
C07D 401/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
91.
SECONDARY CONTAINER WITH FLOW-THROUGH APERTURES FOR FREEZING, THAWING, AND SHIPPING PRODUCTS, AND ASSOCIATED METHODS
Containers are provided herein for facilitating freezing, thawing, and/or storage of products that may be perishable, such as pharmaceuticals. The containers can include shells that enclose the product, and that include apertures permitting flow of a fluid through the containers' internal volume. The temperature of the fluid can be selected so as to freeze or thaw the product. The container optionally can include pressure building members that control the flow of the fluid through the containers' inner volume, e.g., increase the fluid flow rate and thus increase the rate with which the fluid is frozen or thawed.
F25D 25/00 - Charging, supporting, or discharging the articles to be cooled
F25D 15/00 - Devices associated with refrigerating machinery not covered by group or , e.g. non-self-contained movable devices
F25D 17/06 - Arrangements for circulating cooling fluids; Arrangements for circulating gas, e.g. air, within refrigerated spaces for circulating gas, e.g. by natural convection by forced circulation
F25D 3/10 - Devices using other cold materials; Devices using cold-storage bodies using liquefied gases, e.g. liquid air
B65D 1/38 - Baskets or like containers of skeleton or apertured construction
B65D 81/26 - Adaptations for preventing deterioration or decay of contents; Applications to the container or packaging material of food preservatives, fungicides, pesticides or animal repellants with provision for draining away, or absorbing, fluids, e.g. exuded by contents; Applications of corrosion inhibitors or desiccators
92.
TREATMENT OF HEREDITARY ANGIOEDEMA WITH LIVER-SPECIFIC GENE THERAPY VECTORS
Provided herein are compositions and methods for treating a Cl esterase inhibitor deficiency by normalizing levels of the Cl esterase inhibitor protein in a subject having HAE.
Provided herein are compositions and methods for treating a Cl esterase inhibitor deficiency by normalizing levels of the Cl esterase inhibitor protein in a subject having HAE.
This disclosure relates to the use of size exclusion chromatography and/or size exclusion chromatography with multi-angle light scattering technology to characterize viral particles such as adeno-associated virus and lentivirus particles. The disclosed methods are also useful for estimating the titer of viral particles, determining the integrity of the viral particles and estimating the amount of DNA encapsidated in the viral particle.
C12N 7/00 - Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
C12Q 1/70 - Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving virus or bacteriophage
G01N 15/02 - Investigating particle size or size distribution
A61K 47/54 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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 compound
A61K 38/17 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from humans
A61P 19/08 - Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
A61K 47/54 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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 compound
Provided herein are methods of treating Neuronal Ceroid Lipofuscinosis (CLN2) disease in a subject less than 3 years old. In exemplary embodiments, the method comprises administering to the subject a formulation comprising recombinant human tripeptidyl peptidase-1 (rhTPPl) in an amount effective to treat the CLN2 disease in the subject. Also provided are methods of delaying the onset of CLN2 disease, or a symptom thereof, in a subject less than 3 years old. In exemplary embodiments, the method comprises administering to the subject a formulation comprising recombinant human tripeptidyl peptidase-1 (rhTPPl) in an amount effective to delay the onset of the CLN2 disease or symptom in the subject.
A61K 38/48 - Hydrolases (3) acting on peptide bonds (3.4)
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
C12N 9/64 - Proteinases derived from animal tissue, e.g. rennin
99.
METHODS FOR TREATING CLN2 DISEASE IN PEDIATRIC SUBJECTS
Provided herein are methods of treating Neuronal Ceroid Lipofuscinosis (CLN2) disease in a subject less than 3 years old. In exemplary embodiments, the method comprises administering to the subject a formulation comprising recombinant human tripeptidyl peptidase-1 (rhTPPl) in an amount effective to treat the CLN2 disease in the subject. Also provided are methods of delaying the onset of CLN2 disease, or a symptom thereof, in a subject less than 3 years old. In exemplary embodiments, the method comprises administering to the subject a formulation comprising recombinant human tripeptidyl peptidase-1 (rhTPPl) in an amount effective to delay the onset of the CLN2 disease or symptom in the subject.
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
