Search clinical trials for: AAV Vector
Total 3138 results
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Tenaya TherapeuticsMedical University of South Carolina; Johns Hopkins University; University of... and other collaboratorsRecruitingArrhythmogenic Right Ventricular CardiomyopathyUnited States, Italy, United Kingdom, France, Sweden, Germany
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Grace Science, LLCRecruitingNGLY1 DeficiencyUnited States
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PfizerRecruitingHemophilia A | Hemophilia BAustralia, United States, Turkey
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SwanBio Therapeutics, Inc.RecruitingAMN | AMN Gene Mutation | X-ALDNetherlands, United States
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Weill Medical College of Cornell UniversityNational Heart, Lung, and Blood Institute (NHLBI)RecruitingMyocardial Fibrosis | Cardiomyopathies | Friedreich Ataxia | Cardiac HypertrophyUnited States
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UniQure Biopharma B.V.RecruitingHuntington DiseasePoland, United Kingdom, Germany
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BioMarin PharmaceuticalRecruitingHemophilia A With Inhibitor | Hemophilia A With Anti Factor VIIIUnited States, United Kingdom, Taiwan, Israel, Korea, Republic of, South Africa, Brazil, Italy, Germany
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PfizerRecruitingHousehold ContactsUnited States
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UniQure Biopharma B.V.RecruitingHuntington's DiseaseUnited States
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National Human Genome Research Institute (NHGRI)Sio Gene TherapiesRecruitingGangliosidosis | Lysosomal Diseases | GM1United States
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GenSight BiologicsRecruitingNon-syndromic Retinitis PigmentosaUnited States, France, United Kingdom
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CSL BehringActive, not recruitingHemophilia BUnited States, Belgium, Netherlands, Denmark, Germany, Ireland, Sweden, United Kingdom
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National Institute of Neurological Disorders and...Completed
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VegaVect, Inc.National Eye Institute (NEI)Active, not recruiting
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Spark Therapeutics, Inc.Children's Hospital of Philadelphia; University of IowaActive, not recruitingLeber Congenital Amaurosis | Inherited Retinal Dystrophy Due to RPE65 MutationsUnited States
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Nanoscope Therapeutics Inc.CompletedEye Diseases | Retinal Degeneration | Retinal Diseases | Retinitis Pigmentosa | Retinitis | Eye Diseases, Hereditary | Retinal DystrophiesUnited States, Puerto Rico
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Spark TherapeuticsCompletedHematologic Diseases | Blood Coagulation Disorders, Inherited | Coagulation Protein Disorders | Hemorrhagic Disorders | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Gene Therapy | Blood Coagulation Disorder | Gene Transfer | Adeno-Associated Virus (AAV) | Factor VIII (FVIII) | Factor VIII (FVIII)... and other conditionsUnited States
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GenSight BiologicsCompletedLeber Hereditary Optic NeuropathyUnited States, France, Italy, United Kingdom, Germany
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Spark TherapeuticsCompletedChoroideremia | CHM (Choroideremia) Gene MutationsUnited States
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Spark TherapeuticsActive, not recruitingConfirmed Biallelic RPE65 Mutation-associated Retinal DystrophyUnited States
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Spark TherapeuticsCompletedHemophilia AUnited States, Australia, Canada, Israel, Thailand
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Adverum Biotechnologies, Inc.ParexelActive, not recruitingNeovascular Age-related Macular DegenerationUnited States, France, United Kingdom
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GenSight BiologicsNo longer availableLeber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)
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BioMarin PharmaceuticalActive, not recruiting
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Nanoscope Therapeutics Inc.Enrolling by invitationEye Diseases | Retinal Degeneration | Retinal Diseases | Retinitis Pigmentosa | Retinitis | Eye Diseases, Hereditary | Retinal DystrophiesUnited States, Puerto Rico
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Byron LamNational Eye Institute (NEI)Active, not recruitingLeber's Hereditary Optic NeuropathyUnited States
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Adverum Biotechnologies, Inc.CompletedAlpha 1-Antitrypsin DeficiencyUnited States
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Spark TherapeuticsActive, not recruitingLeber Congenital AmaurosisUnited States
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MeiraGTx, LLCNational Institute of Dental and Craniofacial Research (NIDCR)Active, not recruitingSquamous Cell Head and Neck Cancer | Radiation Induced Xerostomia | Salivary HypofunctionUnited States
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Adverum Biotechnologies, Inc.CompletedDiabetic Retinopathy | Diabetic Macular EdemaUnited States, Puerto Rico
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Adverum Biotechnologies, Inc.CompletedNeovascular Age-related Macular Degeneration | Wet Age-related Macular DegenerationUnited States
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Nanoscope Therapeutics Inc.Enrolling by invitationRetinal Degeneration | Retinitis Pigmentosa | Retinal DiseaseIndia
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Brainvectis, a subsidiary of Asklepios BioPharmaceutical...Active, not recruitingHuntington DiseaseFrance
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Baxalta now part of ShireTakeda Development Center Americas, Inc.; Baxalta Innovations GmbH, now part...Active, not recruitingHemophilia AUnited States, Spain, Hungary, Italy, Germany, Austria, France
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Adverum Biotechnologies, Inc.Active, not recruitingNeovascular Age-related Macular Degeneration | Wet Age-related Macular DegenerationUnited States
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National Taiwan University HospitalPTC TherapeuticsCompletedAromatic L-amino Acid Decarboxylase (AADC) DeficiencyTaiwan
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BiocadTerminated
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MeiraGTx UK II LtdJanssen Research & Development, LLCCompleted
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Adverum Biotechnologies, Inc.Enrolling by invitationDiabetic Retinopathy | Diabetic Macular EdemaUnited States, Puerto Rico
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GenSight BiologicsActive, not recruitingLeber Hereditary Optic NeuropathyBelgium, Spain, United States, France, United Kingdom, Taiwan, Italy
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GenSight BiologicsCompletedOptic, Atrophy, Hereditary, LeberUnited Kingdom, United States, France, Germany, Italy
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Baxalta now part of ShireTerminatedHemophilia BTurkey, Spain
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Adverum Biotechnologies, Inc.Completed
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Neurocrine BiosciencesVoyager TherapeuticsCompletedBrain Diseases | Central Nervous System Diseases | Nervous System Diseases | Parkinson's Disease | Parkinsonian Disorders | Movement Disorders | Neurodegenerative Diseases | Idiopathic Parkinson's Disease | Basal Ganglia DiseaseUnited States
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Institut National de la Santé Et de la Recherche...Assistance Publique - Hôpitaux de Paris; European Leukodystrophy AssociationActive, not recruitingMetachromatic LeukodystrophyFrance
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Nanoscope Therapeutics Inc.CompletedRetinal Degeneration | Retinal Diseases | Retinitis PigmentosaIndia
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GenSight BiologicsCompletedLeber Hereditary Optic NeuropathyUnited States, France, Italy, Spain, United Kingdom
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Weill Medical College of Cornell UniversityNational Institutes of Health (NIH)CompletedBatten Disease | Late-Infantile Neuronal Ceroid LipofuscinosisUnited States
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Weill Medical College of Cornell UniversityNathan's Battle FoundationCompletedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States