Abrégé

Provided herein are adeno-associated virus (AAV) compositions that can express an arylsulfatase A (ARSA) polypeptide in a cell, thereby restoring the ARSA gene function. Also provided are methods of using the AAV compositions, and packaging systems for making the AAV compositions.

Classes IPC  ?

• C12N 15/864 - Vecteurs parvoviraux
• 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 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
• C12N 9/16 - Hydrolases (3.) agissant sur les liaisons esters (3.1)

Abrégé

Provided herein are recombinant adeno-associated virus (AAV) capsid proteins, compositions (e.g., rAAV) comprising the capsid proteins, nucleic acids encoding the capsid proteins, and methods of making and using the capsid proteins.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus

Abrégé

Provided herein are polynucleotides comprising novel bidirectional dual expression cassettes, recombination adeno-associated virus (rAAV) comprising these polynucleotides, and methods of making and using the polynucleotides and rAAV. Also provided are novel transcriptional control elements (e.g., promoters, enhancers, introns, polyadenylation sequences, and combinations thereof), and novel antibody coding sequences. The compositions and methods disclosed herein are particularly advantageous in that they allow for the efficient expression of two different polypeptides (e.g., an antibody heavy chain and an antibody light chain) in a cell. In particular, they allow for the efficient expression of antibodies (e.g., anti-C5 antibodies) in a subject, for the treatment of diseases (e.g., C5-mediated diseases, such as PNH).

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• C07K 16/18 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains
• 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 37/06 - Immunosuppresseurs, p. ex. médicaments pour le traitement du rejet de greffe

Abrégé

Provided herein are recombinant adeno-associated virus (AA V) capsid proteins, compositions (e.g., rAAV) comprising the capsid proteins nucleic acids encoding the capsid proteins, and methods of making and using the capsid proteins. The recombinant AAV capsid proteins provided herein are engineered to comprise non-wild type amino acids at positions corresponding to amino acid positions 346, 501, 505, and/or 706 of SEQ ID NO: 14.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• A61K 35/761 - Adénovirus
• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• C12N 15/90 - Introduction stable d'ADN étranger dans le chromosome

Abrégé

Provided herein are methods for the production of recombinant adeno-associated virus (rAAV) particles. These methods are particularly useful for the large-scale production of AAV particles.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux

Abrégé

Provided herein are polynucleotides comprising novel bidirectional dual expression cassettes, recombination adeno-associated virus (rAAV) comprising these polynucleotides, and methods of making and using the polynucleotides and rAAV. Also provided are novel transcriptional control elements (e.g., promoters, enhancers, introns, polyadenylation sequences, and combinations thereof), and novel antibody coding sequences. The compositions and methods disclosed herein are particularly advantageous in that they allow for the efficient expression of two different polypeptides (e.g., an antibody heavy chain and an antibody light chain) in a cell. In particular, they allow for the efficient expression of antibodies (e.g., anti-C5 antibodies) in a subject, for the treatment of diseases (e.g., C5-mediated diseases, such as PNH).

Classes IPC  ?

• 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 15/52 - Gènes codant pour des enzymes ou des proenzymes
• C12N 15/86 - Vecteurs viraux

Abrégé

Provided herein are methods for the production of recombinant adeno-associated virus (rAAV) particles. These methods are particularly useful for the large-scale production of AAV particles.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• C07K 14/015 - Parvoviridae, p. ex. virus de l'aleucémie féline, parvovirus humain
• C07K 14/435 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
• C12N 7/02 - Isolement ou purification

Abrégé

Provided herein are methods and compositions for the separation of an adeno-associated virus (AAV) particle from a mixture of the AAV and at least one contaminant using anion exchange chromatography. These methods and compositions allow for improved purification of complete AAV particles from contaminants such as AAV particles that lack a complete genome (e.g., empty capsids) and AAV degradation products.

Classes IPC  ?

• C12N 7/02 - Isolement ou purification
• C12N 15/864 - Vecteurs parvoviraux
• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus

Abrégé

Provided herein are methods and compositions for the separation of an adeno-associated virus (AAV) particle from a mixture of the AAV and at least one contaminant using anion exchange chromatography. These methods and compositions allow for improved purification of complete AAV particles from contaminants such as AAV particles that lack a complete genome (e.g., empty capsids) and AAV degradation products.

Classes IPC  ?

• 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 15/38 - Adsorption sélective, p. ex. chromatographie caractérisée par le mécanisme de séparation impliquant une interaction spécifique non couverte par un ou plusieurs des groupes , p. ex. chromatographie d'affinité, chromatographie d'échange par ligand ou chromatographie chirale
• B01D 15/42 - Adsorption sélective, p. ex. chromatographie caractérisée par le mode de développement, p. ex. par déplacement ou par élution
• C12N 15/86 - Vecteurs viraux

Abrégé

Provided herein are polynucleotides and vectors comprising non-naturally occurring polyadenylation (polyA) sequences, and methods of making and using these polynucleotides and vectors.

Classes IPC  ?

• C12N 15/67 - Méthodes générales pour favoriser l'expression

Abrégé

Provided herein are pharmaceutical compositions (e.g., formulations) that can provide for the long-term stability of AAV vectors. Also provided herein are methods of making and using the pharmaceutical compositions. The pharmaceutical compositions provided by the present disclosure generally comprise an AAV, histidine, a stabilizing agent, a salt, and a surfactant.

Classes IPC  ?

• 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 15/86 - Vecteurs viraux
• A61K 38/44 - Oxydoréductases (1)
• A61K 38/46 - Hydrolases (3)
• A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
• A61K 47/22 - Composés hétérocycliques, p. ex. acide ascorbique, tocophérol ou pyrrolidones
• A61K 47/26 - Hydrates de carbone, p. ex. polyols ou sucres alcoolisés, sucres aminés, acides nucléiques, mono-, di- ou oligosaccharidesLeurs dérivés, p. ex. polysorbates, esters d’acide gras de sorbitan ou glycyrrhizine
• A61K 47/02 - Composés inorganiques

Abrégé

Provided herein are polynucleotides and vectors comprising non-naturally occurring polyadenylation (poly A) sequences, and methods of making and using these polynucleotides and vectors. Where the polyA signal comprises a first intervening nucleic acid sequence derived from a naturally occurring poly A sequence of a first gene, wherein said naturally occurring poly A sequence of a first gene comprises a poly A signal, a GT rich region, and a nucleic acid sequence positioned between said poly A signal and said GT rich region.

Classes IPC  ?

• C12N 15/62 - Séquences d'ADN codant pour des protéines de fusion
• A61K 38/27 - Hormone de croissance [GH], c.-à-d. somatotropine
• C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
• C12N 15/861 - Vecteurs adénoviraux

Abrégé

Provided herein are pharmaceutical compositions (e.g., formulations) that can provide for the long-term stability of AAV vectors. Also provided herein are methods of making and using the pharmaceutical compositions. The pharmaceutical compositions provided by the present disclosure generally comprise an AAV, histidine, a stabilizing agent, a salt, and a surfactant.

Classes IPC  ?

• C12N 15/09 - Technologie d'ADN recombinant
• C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
• C12N 15/79 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes
• C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
• C12N 15/86 - Vecteurs viraux

Abrégé

Provided herein are novel adeno-associated virus (AAV) capsids, compositions (e.g., rAAV) comprising the capsids, and nucleic acids encoding the capsids. Also provided are methods of making and using the capsids and compositions disclosed herein. The rAAV disclosed herein are particularly useful for gene transfer applications where high transduction efficiency is required (e.g., gene therapy).

Classes IPC  ?

• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus

Abrégé

Provided herein is a dual vector transfection system for the production of recombinant adeno-associated virus (rAAV). The dual vector transfection system generally comprises: (1) a first nucleic acid vector comprising a first nucleotide sequence encoding an AAV Rep protein, a second nucleotide sequence comprising an rAAV genome comprising a transgene, and a third nucleotide sequence encoding an AAV capsid protein; and (2) a second nucleic acid vector comprising a helper virus gene.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• C12N 9/02 - Oxydoréductases (1.), p. ex. luciférase
• C12N 9/16 - Hydrolases (3.) agissant sur les liaisons esters (3.1)
• C07K 16/18 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains

Abrégé

Provided herein is a dual vector transfection system for the production of recombinant adeno-associated virus (rAAV). The dual vector transfection system generally comprises: (1) a first nucleic acid vector comprising a first nucleotide sequence encoding an AAV Rep protein, a second nucleotide sequence comprising an rAAV genome comprising a transgene, and a third nucleotide sequence encoding an AAV capsid protein; and (2) a second nucleic acid vector comprising a helper virus gene.

Classes IPC  ?

• C12N 15/09 - Technologie d'ADN recombinant
• A61K 35/761 - Adénovirus

Abrégé

Provided herein, inter alia, are compositions and methods related to correcting intron 22 inversion in the F8 gene using AAVHSC-mediated nuclease-free genome-editing.

Classes IPC  ?

• C12N 15/90 - Introduction stable d'ADN étranger dans le chromosome
• C12N 15/09 - Technologie d'ADN recombinant
• A61K 35/76 - VirusParticules sous-viralesBactériophages
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique

Abrégé

Provided herein are adeno-associated virus (AAV) compositions that can express an arylsulfatase A (ARSA) polypeptide in a cell, thereby restoring the ARSA gene function. Also provided are methods of using the AAV compositions, and packaging systems for making the AAV compositions.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• C12N 9/16 - Hydrolases (3.) agissant sur les liaisons esters (3.1)
• A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique

Abrégé

Provided are methods for reducing the risk of occurrence and/or the severity of pathogenic diseases (e.g., diseases associate with varicella zoster virus reactivation, influenza infection, and/or S. pneumoniae infection) in a subject that is receiving a gene therapy and an accompanying immunosuppressant regimen.

Classes IPC  ?

• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• C12N 15/86 - Vecteurs viraux

Abrégé

Provided are methods for reducing the risk of occurrence and/or the severity of transaminitis in a subject that is receiving a gene therapy.

Classes IPC  ?

• A61K 31/573 - Composés contenant des systèmes cycliques du cyclopenta[a]hydrophénanthrèneLeurs dérivés, p. ex. stéroïdes substitués en position 17 bêta par une chaîne à deux atomes de carbone, p. ex. prégnane ou progestérone substitués en position 21, p. ex. cortisone, dexaméthasone, prednisone ou aldostérone
• C12N 15/86 - Vecteurs viraux
• A61K 38/44 - Oxydoréductases (1)
• A61K 39/245 - Herpetoviridae, p. ex. virus de l'Herpès simplex
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• A61P 31/22 - Antiviraux pour le traitement des virus ADN des virus de l'herpès
• A61K 39/09 - Streptococcus
• A61K 39/145 - Orthomyxoviridae, p. ex. virus de l'influenza
• A61P 37/06 - Immunosuppresseurs, p. ex. médicaments pour le traitement du rejet de greffe
• A61P 31/04 - Agents antibactériens
• A61P 31/16 - Antiviraux pour le traitement des virus ARN de la grippe ou des rhinovirus

Abrégé

Provided are methods for reducing the risk of occurrence and/or the severity of transaminitis in a subject that is receiving a gene therapy.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus

Abrégé

e.g.S. pneumoniae S. pneumoniae infection) in a subject that is receiving a gene therapy and an accompanying immunosuppressant regimen.

Classes IPC  ?

• A61K 39/12 - Antigènes viraux
• A61K 39/25 - Herpesvirus varicellae
• A61K 39/145 - Orthomyxoviridae, p. ex. virus de l'influenza

Abrégé

e.g.e.g., Paroxysmal Nocturnal Hemoglobinuria)). Also provided are compositions, systems and methods for making the rAAV compositions.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux

Abrégé

e.ge.g., PKU). Also provided are nucleic acids, vectors, packaging systems, and methods for making the adeno-associated virus compositions.

Classes IPC  ?

• C12N 15/90 - Introduction stable d'ADN étranger dans le chromosome
• A61K 35/761 - Adénovirus
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification

Abrégé

e.g.e.g., Hunter syndrome). Also provided are compositions, systems and methods for making the AAV compositions.

Classes IPC  ?

• A61K 38/46 - Hydrolases (3)
• C12N 5/10 - Cellules modifiées par l'introduction de matériel génétique étranger, p. ex. cellules transformées par des virus
• C12N 9/16 - Hydrolases (3.) agissant sur les liaisons esters (3.1)
• C12N 15/09 - Technologie d'ADN recombinant
• C12N 15/52 - Gènes codant pour des enzymes ou des proenzymes
• C12N 15/861 - Vecteurs adénoviraux

Produits et services

Pharmaceuticals, excluding pharmaceuticals for the treatment of genetic diseases and disorders.

Abrégé

Provided herein are methods of treating a subject having a disease or disorder associated with a PAH gene mutation. The methods generally comprise administering to the subject a therapeutically effective does of a recombinant adeno-associated virus (rAAV) that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell and thereby restore PAH gene function in the subject.

Classes IPC  ?

• C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
• C12N 15/86 - Vecteurs viraux

Abrégé

Provided herein are novel adeno-associated virus (AAV) capsids, compositions (e.g., rAAV) comprising the capsids, and nucleic acids encoding the capsids. Also provided are methods of making and using the capsids and compositions disclosed herein. The rAAV disclosed herein are particularly useful for gene transfer applications where high transduction efficiency is required (e.g., gene therapy).

Classes IPC  ?

• A61K 35/761 - Adénovirus
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• C07K 14/075 - Adénoviridae

Produits et services

Pharmaceuticals for human use.

Produits et services

(1) Pharmaceutical preparations and compositions for the treatment of human genetic diseases and disorders.

Abrégé

Provided herein are adeno-associated virus (AAV) compositions that can express an arylsulfatase A (ARSA) polypeptide in a cell, thereby restoring the ARSA gene function. Also provided are methods of using the AAV compositions, and packaging systems for making the AAV compositions.

Classes IPC  ?

• 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
• 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 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
• A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
• C12N 15/86 - Vecteurs viraux
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification

Produits et services

House mark for pharmaceuticals

Produits et services

House mark for pharmaceuticals

Abrégé

Provided herein are adeno-associated virus (AAV) compositions that can restore F8 gene function in cell. Also provide are packaging systems for making the adeno-associated virus compositions.

Classes IPC  ?

• 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
• C12N 15/09 - Technologie d'ADN recombinant
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique

Abrégé

Provided herein are adeno-associated virus (AAV) compositions that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell, thereby restoring the PAH gene function. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.

Classes IPC  ?

• A61K 38/43 - EnzymesProenzymesLeurs dérivés
• A61K 35/761 - Adénovirus
• 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 15/864 - Vecteurs parvoviraux
• C12N 9/02 - Oxydoréductases (1.), p. ex. luciférase
• C12N 5/10 - Cellules modifiées par l'introduction de matériel génétique étranger, p. ex. cellules transformées par des virus
• A61P 3/00 - Médicaments pour le traitement des troubles du métabolisme

Abrégé

Provided herein are adeno-associated virus (AAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cell. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.

Classes IPC  ?

• A61K 35/76 - VirusParticules sous-viralesBactériophages
• A61K 35/761 - Adénovirus
• 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 3/00 - Médicaments pour le traitement des troubles du métabolisme
• C12N 15/86 - Vecteurs viraux
• C12N 15/861 - Vecteurs adénoviraux

Abrégé

Provided herein are adeno-associated virus (AAV) compositions for correcting a mutation in a beta globin gene (HBB) gene and methods of using the same to correct an HBB gene mutation in a cell. Also provided are packaging systems for making the adeno-associated virus compositions.

Classes IPC  ?

• 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 7/04 - Inactivation ou atténuationProduction de parties élémentaires de virus
• C12N 15/864 - Vecteurs parvoviraux
• A61K 35/76 - VirusParticules sous-viralesBactériophages
• C12N 15/90 - Introduction stable d'ADN étranger dans le chromosome

Produits et services

Pharmaceuticals. Pharmaceutical research and development services.

Produits et services

House mark for pharmaceuticals

Produits et services

Pharmaceuticals. Pharmaceutical research and development services.

Produits et services

(1) Pharmaceutical preparations and compositions for the treatment of human genetic disorders; pharmaceutical preparations and compositions for the treatment and prevention of cancer; pharmaceutical preparations and substances for the treatment of gastrointestinal diseases; pharmaceutical preparations and compositions for regulating the immune system; pharmaceutical preparations and compositions for suppressing tumours; pharmaceutical preparations and compositions for the prevention of osteoporosis; pharmaceutical preparations and compositions for the treatment of autoimmune diseases; pharmaceutical preparations and compositions for the treatment of bacterial skin infections, fungal skin infections, viral skin infections and parasitic skin infections; pharmaceutical preparations and compositions for the treatment of blood disorders; pharmaceutical preparations and compositions for the treatment of bone diseases; pharmaceutical preparations for the treatment of bone fractures; pharmaceutical preparations and compositions for the treatment of cardiovascular diseases; pharmaceutical preparations and compositions for the treatment of carpal tunnel syndrome; pharmaceutical preparations and compositions for the treatment of chronic pain; pharmaceutical preparations and compositions for the treatment of cranial or facial nerve disorders; pharmaceutical preparations and compositions for the treatment of degenerative nerve diseases and disorders, namely, Alzheimer's disease, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis ("ALS"), spinal muscular atrophy ("SMA") and multiple sclerosis; pharmaceutical preparations and compositions for the treatment of dental and oral diseases; pharmaceutical preparations and compositions for the treatment of diseases, disorders and infections of the endocrine system, namely growth and thyroid disorders; pharmaceutical preparations and compositions for the treatment of diseases, disorders and infections of the spinal cord systems and related cartilage and tissue; pharmaceutical preparations and compositions for the treatment of drug and alcohol abuse; pharmaceutical preparations and compositions for the treatment of erectile dysfunction; pharmaceutical preparations and compositions for the treatment of genito-urinary and pelvic diseases, disorders and infections, namely infertility, sexually transmitted diseases, incontinence and sexual dysfunction; pharmaceutical preparations and compositions for the treatment of genitourinary diseases, namely, urological diseases, infertility, sexually transmitted diseases, inflammatory pelvic diseases; pharmaceutical preparations and compositions for the treatment of glaucoma; pharmaceutical preparations and compositions for the treatment of headaches; pharmaceutical preparations and compositions for the treatment of heart rhythm disorders; pharmaceutical preparations and compositions for the treatment of hypercholesteremia; pharmaceutical preparations and compositions for the treatment of hypercholesterolemia; pharmaceutical preparations and compositions for the treatment of hyperlipidemia; pharmaceutical preparations and compositions for the treatment of immunologic diseases, namely, autoimmune diseases, immunologic deficiency syndromes; pharmaceutical preparations and compositions for the treatment of infectious diseases, namely respiratory infections, eye infections; pharmaceutical preparations and compositions for the treatment of inflammatory connective tissue diseases and injuries; pharmaceutical preparations and compositions for the treatment of inflammatory diseases, namely, inflammatory bowel diseases, inflammatory connective tissue diseases; pharmaceutical preparations and compositions for the treatment of inflammatory muscle diseases and disorders; pharmaceutical preparations and compositions for the treatment of metabolic diseases and disorders, namely rare or orphan metabolic diseases of the liver, namely phenylketonuria, intrahepatic cholestasis, glycogen storage disease, hemophilia type A, hemophilia type B, alpha-1 antitrypsin deficiency, citrullinemia, tyrosinemia, Wilson disease, Wolman disease, Crigler-Najjar syndrome; pharmaceutical preparations and compositions for the treatment of myalgia and neuralgia; pharmaceutical preparations and compositions for the treatment of neurological diseases, namely, Alzheimer's, Huntington's Disease, cerebral palsy; pharmaceutical preparations and compositions for the treatment of neurological disorders, namely, brain injury, spinal cord injury, seizure disorders; pharmaceutical preparations and compositions for the treatment of neuromuscular diseases and disorders; pharmaceutical preparations and compositions for the treatment of oncological diseases and disorders; pharmaceutical preparations and compositions for the treatment of ophthalmological diseases and disorders; pharmaceutical preparations and compositions for the treatment of physical and psychological addictions, namely, gambling addiction, eating disorders and addictions, nicotine addiction, Internet addiction, drug addiction; pharmaceutical preparations and compositions for the treatment of psychiatric and other brain diseases and disorders, namely, anxiety, mood disorders, schizophrenia, cognitive and bipolar disorders, epilepsy, Alzheimer's, cerebral palsy, Parkinson's disease, multiple sclerosis; pharmaceutical preparations and compositions for the treatment of psychiatric diseases, namely, mood disorders, anxiety disorders, cognitive disorders, schizophrenia; pharmaceutical preparations and compositions for the treatment of respiratory diseases, disorders and infections; pharmaceutical preparations and compositions for the treatment of skin irritations, namely bee stings, sunburn, rashes, sores, corns, calluses, and acne; pharmaceutical preparations and compositions for the treatment of the diseases, disorders and infections of the central nervous system, namely central nervous system infections, central nervous system movement disorders, Friedreich's ataxia, Rett syndrome, Huntington's disease, lysosomal storage diseases with central nervous system manifestations, metachromatic leukodystrophy, mucopolysaccharidoses, Hunter syndrome, neuromuscular disorders, spinal muscular atrophy, spinal cord injury, Parkinson's disease, Alzheimer's disease, ocular disorders, ocular motility disorders, encephalitis, motility disorders; pharmaceutical preparations and compositions for the treatment of viral diseases, namely, herpes, hepatitis, Acquired Immune Deficiency Syndrome (AIDS); pharmaceutical preparations and compositions for use in dermatology, namely, dermatitis, skin pigmentation diseases, sexually transmitted diseases; pharmaceutical preparations and compositions for use in oncology; pharmaceutical products and compositions for treating respiratory diseases; pharmaceutical preparations and compositions for the treatment of disorders of the peripheral nervous system; pharmaceutical preparations and compositions for the treatment of disorders of hematopoietic stem cells; pharmaceutical preparations and compositions for the treatment of disorders of CD34+ cells; pharmaceutical preparations and compositions for the treatment of disorders of musculoskeletal system, namely rare or orphan diseases of the musculoskeletal or neuromuscular structures namely motor neuron disease, monomelic amyotrophy, O'Sullivan-McLeod Syndrome, amyotrophic lateral sclerosis, primary lateral sclerosis, progressive muscular atrophy, spinal muscular atrophy, muscular channelopathy, myotonic syndrome, myotonic dystrophy, myotubular myopathy, skeletal muscle disease, Duchenne muscular dystrophy, Becker muscular dystrophy, limb-girdle muscular dystrophy, congenital muscular dystrophy; pharmaceutical preparations and compositions for the treatment of disorders of neuromuscular system; pharmaceutical preparations and compositions for the treatment of letabolic disorders; pharmaceutical preparations and compositions for the treatment of liver disorders; pharmaceutical preparations and compositions for the treatment of lung diseases; pharmaceutical preparations and compositions for the treatment of Cystic fibrosis; pharmaceutical preparations and compositions for the treatment of Phenylketonuria; pharmaceutical preparations and compositions for the treatment of Sickle cell disease; pharmaceutical preparations and compositions for the treatment of Beta-thalassemia; pharmaceutical preparations and compositions for the treatment of Duchenne Muscular Dystrophy ("DMD"); pharmaceutical preparations and compositions for the treatment of Friedreich's ataxia; pharmaceutical preparations and compositions for the treatment of metachromatic leukodystrophy; pharmaceutical preparations and compositions for the treatment of mucopolysaccharidoses; pharmaceutical preparations and compositions for the treatment of Hunter syndrome; pharmaceutical preparations and compositions for the treatment of Gaucher disease; pharmaceutical preparations and compositions for the treatment of Pompe disease; pharmaceutical preparations and compositions for the treatment of Fabry disease (1) Pharmaceutical research and development services

Produits et services

(1) Pharmaceutical preparations and compositions for the treatment of human genetic disorders; pharmaceutical preparations and compositions for the treatment and prevention of cancer; pharmaceutical preparations and substances for the treatment of gastrointestinal diseases; pharmaceutical preparations and compositions for regulating the immune system; pharmaceutical preparations and compositions for suppressing tumours; pharmaceutical preparations and compositions for the prevention of osteoporosis; pharmaceutical preparations and compositions for the treatment of autoimmune diseases; pharmaceutical preparations and compositions for the treatment of bacterial skin infections, fungal skin infections, viral skin infections and parasitic skin infections; pharmaceutical preparations and compositions for the treatment of blood disorders; pharmaceutical preparations and compositions for the treatment of bone diseases; pharmaceutical preparations for the treatment of bone fractures; pharmaceutical preparations and compositions for the treatment of cardiovascular diseases; pharmaceutical preparations and compositions for the treatment of carpal tunnel syndrome; pharmaceutical preparations and compositions for the treatment of chronic pain; pharmaceutical preparations and compositions for the treatment of cranial or facial nerve disorders; pharmaceutical preparations and compositions for the treatment of degenerative nerve diseases and disorders, namely, Alzheimer's disease, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis ("ALS"), spinal muscular atrophy ("SMA") and multiple sclerosis; pharmaceutical preparations and compositions for the treatment of dental and oral diseases; pharmaceutical preparations and compositions for the treatment of diseases, disorders and infections of the endocrine system, namely growth and thyroid disorders; pharmaceutical preparations and compositions for the treatment of diseases, disorders and infections of the spinal cord systems and related cartilage and tissue; pharmaceutical preparations and compositions for the treatment of drug and alcohol abuse; pharmaceutical preparations and compositions for the treatment of erectile dysfunction; pharmaceutical preparations and compositions for the treatment of genito-urinary and pelvic diseases, disorders and infections, namely infertility, sexually transmitted diseases, incontinence and sexual dysfunction; pharmaceutical preparations and compositions for the treatment of genitourinary diseases, namely, urological diseases, infertility, sexually transmitted diseases, inflammatory pelvic diseases; pharmaceutical preparations and compositions for the treatment of glaucoma; pharmaceutical preparations and compositions for the treatment of headaches; pharmaceutical preparations and compositions for the treatment of heart rhythm disorders; pharmaceutical preparations and compositions for the treatment of hypercholesteremia; pharmaceutical preparations and compositions for the treatment of hypercholesterolemia; pharmaceutical preparations and compositions for the treatment of hyperlipidemia; pharmaceutical preparations and compositions for the treatment of immunologic diseases, namely, autoimmune diseases, immunologic deficiency syndromes; pharmaceutical preparations and compositions for the treatment of infectious diseases, namely respiratory infections, eye infections; pharmaceutical preparations and compositions for the treatment of inflammatory connective tissue diseases and injuries; pharmaceutical preparations and compositions for the treatment of inflammatory diseases, namely, inflammatory bowel diseases, inflammatory connective tissue diseases; pharmaceutical preparations and compositions for the treatment of inflammatory muscle diseases and disorders; pharmaceutical preparations and compositions for the treatment of metabolic diseases and disorders, namely rare or orphan metabolic diseases of the liver, namely phenylketonuria, intrahepatic cholestasis, glycogen storage disease, hemophilia type A, hemophilia type B, alpha-1 antitrypsin deficiency, citrullinemia, tyrosinemia, Wilson disease, Wolman disease, Crigler-Najjar syndrome; pharmaceutical preparations and compositions for the treatment of myalgia and neuralgia; pharmaceutical preparations and compositions for the treatment of neurological diseases, namely, Alzheimer's, Huntington's Disease, cerebral palsy; pharmaceutical preparations and compositions for the treatment of neurological disorders, namely, brain injury, spinal cord injury, seizure disorders; pharmaceutical preparations and compositions for the treatment of neuromuscular diseases and disorders; pharmaceutical preparations and compositions for the treatment of oncological diseases and disorders; pharmaceutical preparations and compositions for the treatment of ophthalmological diseases and disorders; pharmaceutical preparations and compositions for the treatment of physical and psychological addictions, namely, gambling addiction, eating disorders and addictions, nicotine addiction, Internet addiction, drug addiction; pharmaceutical preparations and compositions for the treatment of psychiatric and other brain diseases and disorders, namely, anxiety, mood disorders, schizophrenia, cognitive and bipolar disorders, epilepsy, Alzheimer's, cerebral palsy, Parkinson's disease, multiple sclerosis; pharmaceutical preparations and compositions for the treatment of psychiatric diseases, namely, mood disorders, anxiety disorders, cognitive disorders, schizophrenia; pharmaceutical preparations and compositions for the treatment of respiratory diseases, disorders and infections; pharmaceutical preparations and compositions for the treatment of skin irritations, namely bee stings, sunburn, rashes, sores, corns, calluses, and acne; pharmaceutical preparations and compositions for the treatment of the diseases, disorders and infections of the central nervous system, namely central nervous system infections, central nervous system movement disorders, Friedreich's ataxia, Rett syndrome, Huntington's disease, lysosomal storage diseases with central nervous system manifestations, metachromatic leukodystrophy, mucopolysaccharidoses, Hunter syndrome, neuromuscular disorders, spinal muscular atrophy, spinal cord injury, Parkinson's disease, Alzheimer's disease, ocular disorders, ocular motility disorders, encephalitis, motility disorders; pharmaceutical preparations and compositions for the treatment of viral diseases, namely, herpes, hepatitis, Acquired Immune Deficiency Syndrome (AIDS); pharmaceutical preparations and compositions for use in dermatology, namely, dermatitis, skin pigmentation diseases, sexually transmitted diseases; pharmaceutical preparations and compositions for use in oncology; pharmaceutical products and compositions for treating respiratory diseases; pharmaceutical preparations and compositions for the treatment of disorders of the peripheral nervous system; pharmaceutical preparations and compositions for the treatment of disorders of hematopoietic stem cells; pharmaceutical preparations and compositions for the treatment of disorders of CD34+ cells; pharmaceutical preparations and compositions for the treatment of disorders of musculoskeletal system, namely rare or orphan diseases of the musculoskeletal or neuromuscular structures namely motor neuron disease, monomelic amyotrophy, O'Sullivan-McLeod Syndrome, amyotrophic lateral sclerosis, primary lateral sclerosis, progressive muscular atrophy, spinal muscular atrophy, muscular channelopathy, myotonic syndrome, myotonic dystrophy, myotubular myopathy, skeletal muscle disease, Duchenne muscular dystrophy, Becker muscular dystrophy, limb-girdle muscular dystrophy, congenital muscular dystrophy; pharmaceutical preparations and compositions for the treatment of disorders of neuromuscular system; pharmaceutical preparations and compositions for the treatment of letabolic disorders; pharmaceutical preparations and compositions for the treatment of liver disorders; pharmaceutical preparations and compositions for the treatment of lung diseases; pharmaceutical preparations and compositions for the treatment of Cystic fibrosis; pharmaceutical preparations and compositions for the treatment of Phenylketonuria; pharmaceutical preparations and compositions for the treatment of Sickle cell disease; pharmaceutical preparations and compositions for the treatment of Beta-thalassemia; pharmaceutical preparations and compositions for the treatment of Duchenne Muscular Dystrophy ("DMD"); pharmaceutical preparations and compositions for the treatment of Friedreich's ataxia; pharmaceutical preparations and compositions for the treatment of metachromatic leukodystrophy; pharmaceutical preparations and compositions for the treatment of mucopolysaccharidoses; pharmaceutical preparations and compositions for the treatment of Hunter syndrome; pharmaceutical preparations and compositions for the treatment of Gaucher disease; pharmaceutical preparations and compositions for the treatment of Pompe disease; pharmaceutical preparations and compositions for the treatment of Fabry disease (1) Pharmaceutical research and development services

Produits et services

(1) Pharmaceutical research and development services

Produits et services

Pharmaceuticals for the treatment of genetic diseases and disorders