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2025
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P/S
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Pharmaceutical preparations for the treatment of bone, genetic, joint, metabolic, musculoskeletal... |
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P/S
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Pharmaceutical, scientific, and medical research and development in the field of one, genetic, jo... |
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P/S
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Providing medical services in the field of bone, genetic, joint, metabolic, musculoskeletal, oste... |
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2024
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P/S
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Pharmaceutical, scientific, and medical research and development in the field of bone, genetic, j... |
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P/S
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Providing medical services in the field of genetic, central nervous system, and lysosomal storage... |
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P/S
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Pharmaceutical preparations for the treatment of genetic, central nervous system, and lysosomal s... |
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P/S
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Pharmaceutical, scientific, and medical research and development in the field of genetic, central... |
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Invention
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Methods of producing cdkl5 proteins and uses of the same. The present disclosure provides novel m... |
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Invention
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Compositions and methods for treating conditions associated with bsep deficiency. This applicatio... |
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Invention
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Compositions and methods for reducing host cell proteins in recombinant adeno-associated virus pr... |
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Invention
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Formulations comprising recombinant aav and methods of administering the same. e.g.e.g., rare gen... |
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Invention
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Engineered producer cell lines and methods of making and using the same.
This application relate... |
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Invention
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Human fgf23-binding antibodies with improved affinity and efficacy. The disclosure provides impro... |
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Invention
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Methods of heat inactivation of adenovirus.
The present disclosure generally relates to methods ... |
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Invention
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Crystalline forms of sialic acid derivatives and methods for their use. The present disclosure re... |
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2023
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Invention
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Method of producing soluble microdystrophin protein and uses of the same. The present disclosure ... |
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Invention
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Sialylated glycoprotein compositions and uses thereof.
The present application relates to sialyl... |
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Invention
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Method for determination of double-stranded rna impurity. e.ge.g., a sample solution that compris... |
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P/S
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Pharmaceutical preparations and substances for the treatment
of metabolic and rare diseases and ... |
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P/S
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Pharmaceutical preparations and substances for the treatment of metabolic and rare diseases and d... |
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Invention
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Novel anion-exchange chromatography methods for separation of empty from full recombinant adeno-a... |
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Invention
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Effective and efficient control of serum phosphate for optimal bone formation.
The present inven... |
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Invention
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Creatine prodrugs, compositions and methods of use thereof.
The present disclosure provides crea... |
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P/S
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Printed publications, namely, books, brochures, white papers, informational pamphlets, informatio... |
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P/S
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Compiling of data for research purposes in the field of rare diseases and disorders; medical and ... |
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P/S
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Organizing business networking events in the field of rare diseases and disorders; organizing bus... |
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P/S
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Education and training services, namely, seminars, classes, workshops, and providing non-download... |
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Invention
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Modified batch aav production systems and methods. e.g.e.g., intensified perfusion) for increasin... |
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Invention
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Modified batch aav production systems and methods. This application provides modified batch syste... |
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P/S
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Pharmaceutical preparations and substances for the treatment of metabolic and rare metabolic and ... |
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Invention
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Certain dux4 inhibitors and methods of use thereof. This application relates to double-stranded s... |
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Invention
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Solid compositions of triglycerides and uses thereof.
The present invention includes solid compo... |
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P/S
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Chemical, biological, and biochemical products for use in gene delivery technology, namely, nucle... |
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P/S
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printed publications, namely, books, brochures, informational pamphlets, informational cards, and... |
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P/S
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Research and development in the field of gene therapy manufacturing technology; biomedical and sc... |
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P/S
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Pharmaceutical preparations for the treatment of liver, hepatological, cardiovascular, central ne... |
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P/S
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providing medical services, namely, gene therapy design and delivery services being gene therapy ... |
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P/S
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Licensing of intellectual property |
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2022
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P/S
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Chemical, biological, and biochemical preparations for use in gene delivery technology, namely, n... |
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Invention
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Engineered cell lines for increased production of recombinant adeno-associated virus (raav).
Thi... |
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Invention
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Engineered cell lines for increased production of recombinant adeno-associated virus (raav). This... |
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Invention
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Methods of using anti-sclerostin antibodies in treatment of osteogenesis imperfecta.
Disclosed a... |
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Invention
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Methods of using anti-sclerostin antibodies in treatment of osteogenesis imperfecta. Disclosed ar... |
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Invention
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Methods and compositions for the treatment of amyloidosis.
Methods and compositions for the trea... |
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Invention
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Recombinant aav formulations.
This present disclosure provides pharmaceutical compositions for d... |
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Invention
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Recombinant aav formulations. This present disclosure provides pharmaceutical compositions for de... |
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Invention
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Anion-exchange chromatography methods for purification of recombinant adeno-associated viruses.
... |
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Invention
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Anion-exchange chromatography methods for purification of recombinant adeno-associated viruses. P... |
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Invention
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Compositions and methods for treating a muscular dystrophy.
The present disclosure provides nove... |
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Invention
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Compositions and methods for treating a muscular dystrophy. The present disclosure provides novel... |
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Invention
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Therapeutics for glycogen storage disease type iii.
This invention provides a range of translata... |
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Invention
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Creatine prodrugs, compositions and methods of use thereof. The present disclosure provides creat... |
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P/S
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Providing medical services, namely, gene therapy design and delivery services being gene therapy ... |
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2021
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Invention
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Methods for treating tumor-induced osteomalacia. The present invention provides compositions and ... |
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Invention
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Sialylated glycoprotein compositons and uses thereof.
The present application relates to sialyla... |
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Invention
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Dux4 inhibitors and methods of use thereof.
This application relates to double-stranded small in... |
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Invention
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Dux4 inhibitors and methods of use thereof. This application relates to double-stranded small int... |
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P/S
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Clinical diagnostic reagents for medical and veterinary use,
including in vitro diagnostic reage... |
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Invention
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Methods of heat inactivation of adenovirus. The present disclosure generally relates to methods o... |
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Invention
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Gene therapy for treating propionic acidemia.
This present disclosure provides recombinant adeno... |
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Invention
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Gene therapy for treating propionic acidemia. This present disclosure provides recombinant adeno-... |
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Invention
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Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant a... |
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Invention
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Methods for enhancing recombinant adeno-associated virus yield.
The invention provides methods f... |
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Invention
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Methods for enhancing recombinant adeno-associated virus yield. The invention provides methods fo... |
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Invention
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Gene therapy for treating cdkl5 deficiency disorder.
This present disclosure provides adeno-asso... |
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Invention
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Chaotropic agents for reducing formation of double-stranded rna.
The present disclosure provides... |
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2020
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P/S
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pharmaceuticals for use in treating genetic disorders, autosomal disorders, lysosomal storage dis... |
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Invention
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Methods for improved therapeutic use of recombinant aav.
Provided herein are methods for managin... |
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P/S
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Compiling data into computer databases for research purposes
in the field of rare diseases and d... |
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P/S
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Compiling data into computer databases for research purposes in the field of rare diseases and di... |
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Invention
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Compositions and methods for treatment of hemochromatosis.
This application provides polynucleot... |
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P/S
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Pharmaceuticals for use in treating genetic disorders, autosomal disorders, lysosomal storage dis... |
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Invention
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Gene therapy constructs for treating wilson disease.
This application relates to adeno-associate... |
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2019
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Invention
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Methods and compositions for treating glycogen storage diseases.
This invention provides a varie... |
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P/S
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Providing medical research and scientific research information in the field of hypophosphatemia; ... |
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P/S
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Pharmaceuticals for use in treating genetic disorders,
autosomal disorders, lysosomal storage di... |
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P/S
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Downloadable computer software for use in inputting, gathering, monitoring, sharing, and analyzin... |
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P/S
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providing medical research and scientific research information in the field of hypophosphatemia; ... |
