Disclosed herein are viral vector production systems secreting nuclease for degradation of residual nucleic acid during viral vector production and methods of the same. Such a viral vector production system comprises a viral vector production cell comprising nucleic acid sequences encoding: 1) viral vector components; and 2) a nuclease, wherein the nuclease is expressed in the production cell and secreted in cell culture thereby degrading residual nucleic acid during viral vector production. Another such viral vector production system comprises 1) a viral vector production cell comprising nucleic acid sequences encoding viral vector components; and 2) a nuclease helper cell comprising a nucleic acid sequence encoding a nuclease, wherein the nuclease is expressed and secreted in co-culture of the production cell of 1) and the helper cell of 2), thereby degrading residual nucleic acid during viral vector production.
A method of purifying a viral vector preparation is described. The method comprises: passing a viral vector preparation through a cation exchange column, contacting flow-through from the cation exchange column with an anion exchanger that binds the viral vector, and eluting the bound viral vector from the anion 5 exchanger as a viral vector eluate.
B01D 15/18 - Adsorption sélective, p. ex. chromatographie caractérisée par des caractéristiques de structure ou de fonctionnement relatives aux différents types d'écoulement
B01D 15/36 - Adsorption sélective, p. ex. chromatographie caractérisée par le mécanisme de séparation impliquant une interaction ionique, p. ex. échange d'ions, paire d'ions, suppression d'ions ou exclusion d'ions
B01D 15/42 - Adsorption sélective, p. ex. chromatographie caractérisée par le mode de développement, p. ex. par déplacement ou par élution
The invention relates to the production of lentiviral vectors. More specifically, the present invention relates to nucleotide sequences encoding a lentiviral vector genome which comprises any one or more of a modified 3′ LTR; a modified 5′ LTR; a vector intron; at least one cis-acting sequence; and/or an interfering RNA. Methods and uses of such nucleotide sequences are also encompassed by the invention.
The invention relates to a viral vector production system. More specifically, the present invention relates to a set of nucleotide sequences comprising sequences encoding heterologous envelope proteins that facilitates the production of mixed envelopes, wherein the mixed envelopes comprise a mixed species of envelope proteins. Viral vectors, methods and uses of such viral vector production systems are also encompassed by the invention.
C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
G01N 33/50 - Analyse chimique de matériau biologique, p. ex. de sang ou d'urineTest par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligandsTest immunologique
A nucleotide sequence encoding the RNA genome of a lentiviral vector, wherein the major splice donor site in the RNA genome of the lentiviral vector is inactivated, and wherein the cryptic splice donor site 3′ to the major splice donor site is inactivated.
The invention relates to the preparation of a solution of polymer/nucleic acid complexes, and the use of such a solution in methods for the transfection of cells.
C12N 15/88 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation utilisant la micro-encapsulation, p. ex. utilisant des vésicules liposomiques
C12N 5/00 - Cellules non différenciées humaines, animales ou végétales, p. ex. lignées cellulairesTissusLeur culture ou conservationMilieux de culture à cet effet
C12N 5/071 - Cellules ou tissus de vertébrés, p. ex. cellules humaines ou tissus humains
The present invention provides a novel method for detecting replication competent virus in a test sample. The method comprises culturing and diluting a plurality of individual cell culture aliquots comprising virus-permissive cells and a portion of the test sample, followed by testing for the presence of replication competent virus. The method may be used in parallel with a positive control, which is also provided herein.
A method of purifying a viral vector preparation is described. The method comprises: passing a viral vector preparation through a cation exchange column, contacting flow-through from the cation exchange column with an anion exchanger that binds the viral vector, and eluting the bound viral vector from the anion 5 exchanger as a viral vector eluate.
C12N 5/00 - Cellules non différenciées humaines, animales ou végétales, p. ex. lignées cellulairesTissusLeur culture ou conservationMilieux de culture à cet effet
C12N 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
A cell for producing retroviral vectors comprising nucleic acid sequences encoding: i) gag-pol; ii) env; iii) the RNA genome of the retroviral vector; and iv) optionally rev, or a functional substitute thereof, wherein at least two nucleic acid sequences are located at the same genetic locus; and wherein the at least two nucleic acid sequences are in reverse and/or alternating orientations.
The present invention provides a lentiviral vector genome comprising at least one modified viral cis-acting sequence, wherein at least one internal open reading frame (ORF) in the viral cis-acting sequence is disrupted.
The invention relates to production of lentiviral vectors. More specifically, the present invention relates to nucleotide sequences comprising a lentiviral genome expression cassette. The expression cassette comprises a rev/RRE-independent lentiviral vector genome which comprises an intron. Methods and uses of such nucleotide sequences are also encompassed by the invention.
The invention relates to the production of lentiviral vectors. More specifically, the present invention relates to a nucleotide sequence encoding a lentiviral vector genome, wherein the 3' LTR of the lentiviral vector genome comprises a modified polyadenylation sequence, and wherein the modified polyadenylation sequence comprises a polyadenylation signal which is 5' of the 3' LTR R region. The invention also relates to a nucleotide sequence encoding a lentiviral vector genome, wherein the lentiviral vector genome comprises a modified 5' LTR, and wherein the R region of the modified 5' LTR comprises at least one polyadenylation downstream enhancer element (DSE). Methods and uses of such nucleotide sequences are also encompassed by the invention.
The invention relates to the production of lentiviral vectors. More specifically, the present invention relates to nucleotide sequences encoding a lentiviral vector genome which comprises any one or more of a modified 3' LTR; a modified 5' LTR; a vector intron; at least one cis-acting sequence; and/or an interfering RNA. Methods and uses of such nucleotide sequences are also encompassed by the invention.
The present invention relates to novel nucleotide sequences, and to viral vectors or cells comprising such nucleotide sequences. The invention also relates to viral vector production systems, and methods for producing viral vectors using the nucleotide sequences, viral vectors, or cells described herein. Methods for identifying sequences that improve transgene expression in a target cell are also provided herein.
The invention relates to the production of retroviral vectors. More specifically, the present invention relates to a set of nucleic acid sequences for producing a retroviral vector comprising (i) a nucleic acid sequence encoding the retroviral vector genome, wherein the retroviral vector genome comprises a transgene expression cassette; and (ii) at least one nucleic acid sequence encoding an interfering RNA. Methods and uses of the set of nucleic acid sequences are also encompassed by the invention.
The present invention relates to a nucleic acid sequence comprising a nucleotide of interest and a tryptophan RNA-binding attenuation protein (TRAP) binding site, and optionally a Kozak sequence, wherein said TRAP binding site overlaps the Kozak sequence and/or the ATG start codon of the nucleotide of interest. The present invention further relates to a nucleic acid sequence comprising a nucleotide of interest and a Kozak sequence, wherein said Kozak sequence comprises a portion of a tryptophan RNA-binding attenuation protein (TRAP) binding site. The present invention further relates to a nucleic acid sequence comprising a nucleotide of interest and TRAP binding site wherein the TRAP binding site comprises a portion of the start codon ATG of said nucleotide of interest or wherein the ATG start codon comprises a portion of the TRAP binding site. The present invention further relates to a nucleic acid sequence comprising a nucleotide of interest, a binding site for tryptophan RNA-binding attenuation protein (TRAP), a multiple cloning site and a Kozak sequence, wherein said multiple cloning site is overlapping with or located downstream to the 3′ KAGN2-3 repeat of the TRAP binding site and upstream of the Kozak sequence.
C12N 15/67 - Méthodes générales pour favoriser l'expression
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A modified U1 snRNA, wherein said modified U1 snRNA has been modified to bind to a nucleotide sequence within the packaging region of a lentiviral vector genome sequence.
A method of improving motor function and reducing dyskinesia in a subject suffering from a neurodegenerative disease or a disease where endogenous dopamine levels are reduced in the subject comprising administering an effective amount of a viral vector comprising a nucleic acid construct comprising (i) a nucleotide sequence which encodes tyrosine hydroxylase (TH), (ii) a nucleotide sequence which encodes GTP-cyclohydrolase I (CH1), (iii) a nucleotide sequence which encodes Aromatic Amino Acid Dopa Decarboxylase (AADC), or any combination thereof to the subject.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
The invention relates to the preparation of a solution of polymer/nucleic acid complexes, and the use of such a solution in methods for the transfection of cells.
C12N 15/87 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
The present invention provides a lentiviral vector genome comprising at least one modified viral cis-acting sequence, wherein at least one internal open reading frame (ORF) in the viral cis-acting sequence is disrupted.
The present invention provides a novel method for detecting replication competent virus in a test sample. The method comprises culturing and diluting a plurality of individual cell culture aliquots comprising virus-permissive cells and a portion of the test sample, followed by testing for the presence of replication competent virus. The method may be used in parallel with a positive control, which is also provided herein.
C12Q 1/70 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions faisant intervenir des virus ou des bactériophages
C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
A nucleotide sequence encoding the RNA genome of a lentiviral vector, wherein the major splice donor site in the RNA genome of the lentiviral vector is inactivated, and wherein the cryptic splice donor site 3' to the major splice donor site is inactivated.
The present invention relates to a nucleic acid sequence comprising a nucleotide of interest and a tryptophan RNA-binding attenuation protein (TRAP) binding site, and optionally a Kozak sequence, wherein said TRAP binding site overlaps the Kozak sequence and/or the ATG start codon of the nucleotide of interest. The present invention further relates to a nucleic acid sequence comprising a nucleotide of interest and a Kozak sequence, wherein said Kozak sequence comprises a portion of a tryptophan RNA-binding attenuation protein (TRAP) binding site. The present invention further relates to a nucleic acid sequence comprising a nucleotide of interest and TRAP binding site wherein the TRAP binding site comprises a portion of the start codon ATG of said nucleotide of interest or wherein the ATG start codon comprises a portion of the TRAP binding site. The present invention further relates to a nucleic acid sequence comprising a nucleotide of interest, a binding site for tryptophan RNA-binding attenuation protein (TRAP), a multiple cloning site and a Kozak sequence, wherein said multiple cloning site is overlapping with or located downstream to the 3' KAGN2-3 repeat of the TRAP binding site and upstream of the Kozak sequence.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
C12N 15/69 - Augmentation du nombre de copies du vecteur
Disclosed herein are viral vector production systems secreting nuclease for degradation of residual nucleic acid during viral vector production and methods of the same. Such a viral vector production system comprises a viral vector production cell comprising nucleic acid sequences encoding: 1) viral vector components; and 2) a nuclease, wherein the nuclease is expressed in the production cell and secreted in cell culture thereby degrading residual nucleic acid during viral vector production. Another such viral vector production system comprises 1) a viral vector production cell comprising nucleic acid sequences encoding viral vector components; and 2) a nuclease helper cell comprising a nucleic acid sequence encoding a nuclease, wherein the nuclease is expressed and secreted in co-culture of the production cell of 1) and the helper cell of 2), thereby degrading residual nucleic acid during viral vector production.
A modified U1 snRNA, wherein said modified U1 snRNA has been modified to bind to a nucleotide sequence within the packaging region of a lentiviral vector genome sequence.
A method of improving motor function and reducing dyskinesia in a subject suffering from a neurodegenerative disease or a disease where endogenous dopamine levels are reduced in the subject comprising administering an effective amount of a viral vector comprising a nucleic acid construct comprising (i) a nucleotide sequence which encodes tyrosine hydroxylase (TH), (ii) a nucleotide sequence which encodes GTP-cyclohydrolase I (CH1), (iii) a nucleotide sequence which encodes Aromatic Amino Acid Dopa Decarboxylase (AADC), or any combination thereof to the subject.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61K 38/00 - Préparations médicinales contenant des peptides
A61P 25/14 - Médicaments pour le traitement des troubles du système nerveux pour traiter les mouvements anormaux, p. ex. chorée, dyskinésie
The present invention relates to a nucleic acid sequence comprising a binding site operably linked to a nucleotide of interest, wherein the binding site is capable of interacting with an RNA-binding protein such that translation of the nucleotide of interest is repressed in a viral vector production cell.
Provided is a construct comprising (i) a nucleotide sequence which encodes tyrosine hydroxylase (TH), (ii) a nucleotide sequence which encodes GTP-cyclohydrolase I (CH1) and (iii) a nucleotide sequence which encodes Aromatic Amino Acid Dopa Decarboxylase (AADC) wherein the nucleotide sequence encoding TH is linked to the nucleotide sequence encoding CH1 such that they encode a fusion protein TH-CH1. Also provided is a construct comprising (i) a nucleotide sequence which encodes tyrosine hydroxylase (TH), (ii) a nucleotide sequence which encodes GTP-cyclohydrolase I (CH1) and (iii) a nucleotide sequence which encodes Aromatic Amino Acid Dopa Decarboxylase (AADC) wherein the nucleotide sequence encoding AADC is linked to the nucleotide sequence encoding TH such that they encode a fusion protein AADC-TH or TH-AADC. Further provided is a viral vector comprising such nucleotide sequences and its use in the treatment and/or prevention of Parkinson's disease.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
The present invention relates to immunotherapeutic approaches to treating haematological cancers. In particular the invention relates to a method for treating a haematological cancer by targeting the 5T4 antigen. As such, the invention provides a method for treating haematological cancers comprising administering to a subject a 5T4-targeting agent. The invention also provides a 5T4-specific chimeric antigen receptor (CAR) and uses thereof in treating cancers.
C07K 16/30 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire provenant de cellules de tumeurs
A61K 35/17 - LymphocytesLymphocytes BLymphocytes TCellules tueuses naturellesLymphocytes activés par un interféron ou une cytokine
A61K 47/68 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un anticorps, une immunoglobuline ou son fragment, p. ex. un fragment Fc
A61P 35/02 - Agents anticancéreux spécifiques pour le traitement de la leucémie
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
Disclosed herein are viral vector production systems secreting nuclease for degradation of residual nucleic acid during viral vector production and methods of the same. Such a viral vector production system comprises a viral vector production cell comprising nucleic acid sequences encoding: 1) viral vector components; and 2) a nuclease, wherein the nuclease is expressed in the production cell and secreted in cell culture thereby degrading residual nucleic acid during viral vector production. Another such viral vector production system comprises 1) a viral vector production cell comprising nucleic acid sequences encoding viral vector components; and 2) a nuclease helper cell comprising a nucleic acid sequence encoding a nuclease, wherein the nuclease is expressed and secreted in co-culture of the production cell of 1) and the helper cell of 2), thereby degrading residual nucleic acid during viral vector production.
The present invention relates to immunotherapeutic approaches to treating haematological cancers. In particular the invention relates to a method for treating a haematological cancer by targeting the 5T4 antigen. As such, the invention provides a method for treating haematological cancers comprising administering to a subject a 5T4-targeting agent. The invention also provides a 5T4-specific chimeric antigen receptor (CAR) and uses thereof in treating cancers.
C07K 16/30 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire provenant de cellules de tumeurs
A process for producing a retroviral or lentiviral vector formulation comprising a filter-sterilisation step wherein the filter-sterilisation step is not the final step in the purification process.
C12N 15/00 - Techniques de mutation ou génie génétiqueADN ou ARN concernant le génie génétique, vecteurs, p. ex. plasmides, ou leur isolement, leur préparation ou leur purificationUtilisation d'hôtes pour ceux-ci
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
05 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
[ Chemical compositions containing nucleic acids for use in the manufacture of pharmaceuticals and retroviral vector preparations; Chemicals for use in industry and science; enzymes for scientific and research purposes; biochemicals, namely, precursors for in vitro genetic engineering use; polypeptides for in vitro research use, monoclonal antibodies for in vitro scientific or research use; chemicals for use in the purification of proteins for in vitro use; diagnostic reagents for in vitro use in biochemistry, clinical chemistry and microbiology; laboratory chemicals, namely, an antibody reagent used for the detection of antigens in cell and tissue analysis for in vitro diagnostic use; micro-organisms for laboratory and scientific research use, viruses for laboratory and scientific research use, cell cultures for laboratory and scientific research use, plasmids and cultures of micro-organisms for laboratory and scientific research use, nucleic acid sequences for laboratory and scientific research use, cloning vehicles in the nature of plasmids, microbes and chemical reagents for DNA cloning for laboratory and scientific research use, chemicals for laboratory, scientific and biotechnology research and development and recombinant nucleic acid variations thereof for laboratory and scientific research use, namely, in vitro and in vivo use ] [ Clinical medical reagents for use in gene therapy; pharmaceutical preparations for use in gene therapy; therapeutic compositions containing retroviral vector preparations and/or nucleic acid suitable for gene therapy; retroviral vector preparations for use in gene therapy; pharmaceutical preparations containing a retroviral vector delivering genes to cells for use in the treatment of viral and bacterial infections, cancer, HIV and AIDS, neurodegenerative diseases, diseases of the eye, leukemia and bodily conditions associated with impaired immunosystems; Gene therapy and prophylactic products, namely, gene delivery pharmaceuticals; small molecules, vaccines, biologics, enzymes and enzyme preparations all for medical treatment purposes; pharmaceuticals for medical purposes for the delivery of genes to cells; viral, retroviral and non-viral vectors, all for medical purposes in gene therapy; pharmaceutical and biochemical preparations for the treatment or prevention of cancer, infectious disease, fungal diseases, diseases caused by parasites, namely, hookworms, pinworms, roundworms, tapeworms, flukes, giardia, entameoba, cryptosporidium, toxoplasmosis, sleeping sickness, trichuriasis, isosporiasis, malaria, parasitic skin disease and leishmaniasis, eye diseases, diseases of the central and peripheral nervous systems, namely, ataxia, dementia, epilepsy, Alzheimer's, cerebral palsy, stroke, motor neuron disease, Parkinson's disease, spinal injury, avulsion injury and peripheral neuropathies, cardiovascular disease, diseases affecting the immune system, namely, cancer, Crohn's disease, AIDS, HIV, lupus, celiac disease, diabetes, Grave's disease, Guillain-Barré syndrome, Addison's disease, Hashimoto's disease, myasthenia gravis, narcolepsy, pernicious anaemia, rheumatoid arthritis, psoriasis, ulcerative colitis, immunodeficiency and inflammatory diseases, inherited diseases, namely, genetic disorders, hemophilia, cystic fibrosis and Huntingdon's, single gene disorders, bone and blood diseases and reproductive system disorders; pharmaceutical and biochemical preparations for contraceptive purposes; pre-filled vials for medical purposes and for pharmaceutical and biochemical preparations, for use in gene therapy and for the treatment or prevention of cancer, infectious disease, fungal diseases, diseases caused by parasites, namely, hookworms, pinworms, roundworms, tapeworms, flukes, giardia, entameoba, cryptosporidium, toxoplasmosis, sleeping sickness, trichuriasis, isosporiasis, malaria, parasitic skin disease and leishmaniasis, eye diseases, diseases of the central and peripheral nervous systems, namely, ataxia, dementia, epilepsy, Alzheimer's, cerebral palsy, stroke, motor neuron disease, Parkinson's disease, spinal injury, avulsion injury and peripheral neuropathies, cardiovascular disease, diseases affecting the immune system, namely, cancer, Crohn's disease, AIDS, HIV, lupus, celiac disease, diabetes, Grave's disease, Guillain-Barré syndrome, Addison's disease, Hashimoto's disease, myasthenia gravis, narcolepsy, pernicious anaemia, rheumatoid arthritis, psoriasis, ulcerative colitis, immunodeficiency and inflammatory diseases, inherited diseases, namely, genetic disorders, hemophilia, cystic fibrosis and Huntingdon's, single gene disorders, bone and blood diseases and reproductive system disorders ] Scientific, pharmaceutical and medical research and development; genetic engineering services, biotechnology services, namely, research and development for new products in the field of biotechnology, advisory services relating to gene therapy products and clinical trials, conducting clinical trials [ Medical services; ] advisory services relating to vaccination regimes; advisory services relating to the treatment, relief and/or prevention of cancer, infectious diseases, fungal diseases, diseases caused by parasites, namely, hookworms, pinworms, roundworms, tapeworms, flukes, giardia, entameoba, cryptosporidium, toxoplasmosis, sleeping sickness, trichuriasis, isosporiasis, malaria, parasitic skin disease and leishmaniasis, eye diseases, diseases of the central and peripheral nervous systems, namely, ataxia, dementia, epilepsy, Alzheimer's, cerebral palsy, stroke, motor neuron disease, Parkinson's disease, spinal injury, avulsion injury and peripheral neuropathies, cardiovascular disease, diseases affecting the immune system, namely, cancer, Crohn's disease, AIDS, HIV, lupus, celiac disease, diabetes, Grave's disease, Guillain-Barré syndrome, Addison's disease, Hashimoto's disease, myasthenia gravis, narcolepsy, pernicious anaemia, rheumatoid arthritis, psoriasis, ulcerative colitis, immunodeficiency and inflammatory diseases, inherited diseases, namely, genetic disorders, hemophilia, cystic fibrosis and Huntingdon's, single gene disorders, bone and blood diseases and reproductive system disorders and contraceptives
The present invention relates to a nucleic acid sequence comprising a binding site operably linked to a nucleotide of interest, wherein the binding site is capable of interacting with an RNA-binding protein such that translation of the nucleotide of interest is repressed in a viral vector production cell.
The present invention relates to a nucleic acid sequence comprising a binding site operably linked to a nucleotide of interest, wherein the binding site is capable of interacting with an RNA-binding protein such that translation of the nucleotide of interest is repressed in a viral vector production cell.
Provided is a construct comprising (i) a nucleotide sequence which encodes tyrosine hydroxylase (TH), (ii) a nucleotide sequence which encodes GTP-cyclohydrolase I (CH1) and (iii) a nucleotide sequence which encodes Aromatic Amino Acid Dopa Decarboxylase (AADC) wherein the nucleotide sequence encoding TH is linked to the nucleotide sequence encoding CH1 such that they encode a fusion protein TH-CH1. Also provided is a construct comprising (i) a nucleotide sequence which encodes tyrosine hydroxylase (TH), (ii) a nucleotide sequence which encodes GTP-cyclohydrolase I (CH1) and (iii) a nucleotide sequence which encodes Aromatic Amino Acid Dopa Decarboxylase (AADC) wherein the nucleotide sequence encoding AADC is linked to the nucleotide sequence encoding TH such that they encode a fusion protein AADC-TH or TH-AADC. Further provided is a viral vector comprising such nucleotide sequences and its use in the treatment and/or prevention of Parkinson's disease.
A61K 38/54 - Mélanges d'enzymes ou de proenzymes couverts par plus d'un seul des groupes ou
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 9/02 - Oxydoréductases (1.), p. ex. luciférase
C12N 9/78 - Hydrolases (3.) agissant sur les liaisons carbone-azote autres que les liaisons peptidiques (3.5)
The present invention provides a construct comprising (i) a nucleotide sequence which encodes tyrosine hydroxylase (TH), (ii) a nucleotide sequence which encodes GTP- cyclohydrolase I (CH1) and (iii) a nucleotide sequence which encodes Aromatic Amino Acid Dopa Decarboxylase (AADC) wherein the nucleotide sequence encoding TH is linked to the nucleotide sequence encoding CHI such that they encode a fusion protein TH-CH1. The invention also provides a viral vector comprising such a nucleotide sequence and its use in the treatment and/or prevention of Parkinson's disease.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 15/62 - Séquences d'ADN codant pour des protéines de fusion
A method for determining a prognosis for benefit for a cancer patient receiving immunotherapy treatment involving (a) measuring a level of haematocrit and haemoglobin in a sample from the cancer patient, and (b) comparing the level of haematocrit in the sample to a reference level of platelets and comparing the level of haemoglobin in the sample to a reference level of haemoglobin, wherein a lower level of haematocrit and higher level of haemoglobin in the sample correlates with increased benefit to the patient.
The present invention provides a lentiviral vector for delivery to the brain for use in treating a neurological condition, wherein the lentiviral vector is delivered directly to the brain by delivering the lentiviral vector via six or fewer tracts per hemisphere, at a single deposit point per tract.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A method for determining a prognosis for benefit for a cancer patient receiving immunotherapy treatment involving (a) measuring a level of platelets and haemoglobin in a sample from the cancer patient, and (b) comparing the level of platelets in the sample to a reference level of platelets and comparing the level of haemoglobin in the sample to a reference level of haemoglobin, wherein a lower level of platelets and higher level of haemoglobin in the sample correlates with increased benefit to the patient.
The present invention provides a method for detecting a 5T4-positive cancer in a subject, which comprises the following steps: (i) identifying and/or isolating exosomes in a sample from the subject; and (ii) detecting exosome-associated 5T4.
The present invention provides methods for: (i) treating and/or preventing Parkinson's disease in a subject without causing cognitive impairment by using dopamine replacement gene therapy to maintain or restore constant physiological dopaminergic tone in both the dorsal and ventral striatum of the subject; (ii) normalising neuronal electrical activity in basal ganglia and/or subthalamic nucleus in a Parkinson's disease subject; and (iii) treating and/or preventing dyskinesias associated with oral L-dopa administration in a Parkinson's disease subject by administration of a vector system for dopamine replacement gene therapy to the subject.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
The present invention provides a method of monitoring the efficacy of an immunotherapy in a mammalian subject, wherein the subject has been administered an immunotherapy, wherein the immunotherapy comprises a viral vector containing a polynucleotide encoding an antigen, wherein the viral vector is capable of transducing cells in the mammalian subject to cause the cells to express the antigen; the method comprising: (b) measuring, from a biological sample isolated from the subject, an immune response of the subject to the antigen and comparing the immune response of the subject to the antigen to a reference measurement of immune response to the antigen; (c) measuring, from a biological sample isolated from the subject, an immune response of the subject to the viral vector and comparing the immune response of the subject to the viral vector to a reference measurement of immune response to the viral vector; and (d) determining efficacy based on the comparisons of (b) and (c), wherein an elevated immune response to the antigen and a reduced immune response to the viral vector are indicative of an effective immunotherapy.
G01N 33/68 - Analyse chimique de matériau biologique, p. ex. de sang ou d'urineTest par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligandsTest immunologique faisant intervenir des protéines, peptides ou amino-acides
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
C12N 15/863 - Vecteurs poxviraux, p. ex. virus de la vaccine
G01N 33/574 - Tests immunologiquesTests faisant intervenir la formation de liaisons biospécifiquesMatériaux à cet effet pour le cancer
A process for producing a retroviral or lentiviral vector formulation comprising a filter-sterilization step wherein the filter-sterilization step is not the final step in the purification process.
C12N 15/00 - Techniques de mutation ou génie génétiqueADN ou ARN concernant le génie génétique, vecteurs, p. ex. plasmides, ou leur isolement, leur préparation ou leur purificationUtilisation d'hôtes pour ceux-ci
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A process for producing a retroviral or lentiviral vector formulation comprising a filter-sterilisation step wherein the filter-sterilisation step is not the final step in the purification process.
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
05 - Produits pharmaceutiques, vétérinaires et hygièniques
10 - Appareils et instruments médicaux
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Chemical products for use in industry and science; enzymes; chemical products for in-vitro use in laboratories and for analysis; micro-organisms, viruses, cell cultures, plasmids and cultures of micro-organisms, nucleic acid sequences, cloning vehicles, chemical products for use in biotechnology, research and development and recombinant nucleic acid variations thereof, all for in-vivo use. Therapeutic and prophylactic products; gene therapy products; pharmaceutical substances and preparations; small molecules, vaccines, biologics, enzymes and enzyme preparations, pharmaceuticals for the delivery of genes to cells, all for medical purposes; viral , retroviral and non-viral vectors; viral, retroviral and non-viral vector manufacturing preparations; biochemical and chemical products for use in medical science; pharmaceutical and biochemical preparations for the treatment or prevention of cancer, infectious disease, fungal diseases, disease caused by parasites, eye disease, diseases of the central and peripheral nervous systems, cardiovascular disease, diseases affecting the immune system, inflammatory diseases, inherited disease, single gene disorders, diseases of the mouth, teeth, skin, hair and ear, bone and blood diseases and reproductive system disorders; pharmaceutical and biochemical preparations for contraceptive purposes. Instruments and apparatus for the administration of pharmaceutical, gene therapy and/or vaccine preparations and substances, pre-filled vials; syringes and injectors for medical purposes. Scientific, pharmaceutical and medical research and development; genetic engineering services, biotechnology services, advisory services relating to gene therapy products and clinical trials, conducting clinical trials. Medical services; advisory services relating to vaccination regimes; advisory services relating to the treatment, relief and/or prevention of cancer, infectious diseases, fungal diseases, disease caused by parasites, eye disease, diseases of the central and peripheral nervous systems, cardiovascular disease, diseases affecting the immune system, inflammatory diseases, inherited disease, single gene disorders, diseases of the mouth, teeth, skin, hair and ear, bone and blood diseases, reproductive system disorders, and pharmaceutical and biochemical preparations for contraceptive purposes, in humans and animals.
Provided is a lentiviral expression system to deliver potential therapeutic agents for treating motor neuron diseases in a tissue-specific manner. Vector constructs are provided that comprise a rabies G-protein and a muscle cell-specific microRNA sequence.
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
51.
METHODS AND COMPOSITION FRO T CELL RECEPTORS WHICH RECOGNIZE 5T4 ANTIGEN
The present invention relates to the use of peptide epitopes of 5T4 antigen in the identification and isolation of T cell receptors which recognize 5T4 antigen.
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
An integration defective retroviral vector particle for gene therapy comprising a viral genome wherein said vector particle is capable of infecting a mammalian target cell.
C07K 14/15 - Rétroviridae, p. ex. virus de la leucémie bovine, virus de la leucémie féline, virus de la leucémie lymphoïde des lymphocytes-T humains
C07K 14/155 - Lentiviridae, p. ex. virus du déficit immunitaire humain [HIV], virus visna-maedi ou virus de l'anémie infectieuse équine
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A polynucleotide comprising a nucleotide sequence encoding a retroviral gag protein wherein the gag protein comprises a heterologous RNA binding domain capable of recognising a corresponding sequence in an RNA genome to facilitate packaging of the RNA genome into a retroviral vector particle.
The present invention provides a construct comprising (i) a nucleotide sequence which encodes tyrosine hydroxylase (TH), (ii) a nucleotide sequence which encodes GTP- cyclohydrolase I (CH1) and (iii) a nucleotide sequence which encodes Aromatic Amino Acid Dopa Decarboxylase (AADC) wherein the nucleotide sequence encoding TH is linked to the nucleotide sequence encoding CHI such that they encode a fusion protein TH-CH1. The invention also provides a viral vector comprising such a nucleotide sequence and its use in the treatment and/or prevention of Parkinson's disease.
The present invention relates to immunotherapeutic approaches to treating haematological cancers. In particular the invention relates to a method for treating a haematological cancer by targeting the 5T4 antigen. As such, the invention provides a method for treating haematological cancers comprising administering to a subject a 5T4-targeting agent. The invention also provides a 5T4-specific chimeric antigen receptor (CAR) and uses thereof in treating cancers.
C07K 16/30 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire provenant de cellules de tumeurs
56.
GENE THERAPY COMPOSITIONS AND METHODS FOR TREATING PARKINSON'S DISEASE
A method of improving motor function and reducing dyskinesia in a subject suffering from a neurodegenerative disease or a disease where endogenous dopamine levels are reduced in the subject comprising administering an effective amount of a viral vector comprising a nucleic acid construct comprising (i) a nucleotide sequence which encodes tyrosine hydroxylase (TH), (ii) a nucleotide sequence which encodes GTP-cyclohydrolase I (CH1), (iii) a nucleotide sequence which encodes Aromatic Amino Acid Dopa Decarboxylase (AADC), or any combination thereof to the subject.
A61K 38/00 - Préparations médicinales contenant des peptides
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 25/14 - Médicaments pour le traitement des troubles du système nerveux pour traiter les mouvements anormaux, p. ex. chorée, dyskinésie
The present invention provides a construct comprising (i) a nucleotide sequence which encodes tyrosine hydroxylase (TH), (ii) a nucleotide sequence which encodes GTP-cyclohydrolase I (CH1) and (iii) a nucleotide sequence which encodes Aromatic Amino Acid Dopa Decarboxylase (AADC) wherein the nucleotide sequence encoding TH is linked to the nucleotide sequence encoding CH1 such that they encode a fusion protein TH- CH1. The invention also provides a viral vector comprising such a nucleotide sequence and its use in the treatment and/or prevention of Parkinson's disease.
