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adeno-associated virus serotype 9 (AAV9)

Showing 1 - 25 of >10,000
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Danon Disease Trial in La Jolla, Philadelphia (RP-A501)

Recruiting
  • Danon Disease
  • RP-A501
  • La Jolla, California
  • +1 more
Oct 17, 2023

Canavan Disease Trial in Boston (AAV9 BBP-812)

Recruiting
  • Canavan Disease
  • AAV9 BBP-812
  • Boston, Massachusetts
    Massachusetts General Hospital (MGH); Center for Rare Neurologic
Mar 12, 2022

Danon Disease Trial in La Jolla, Aurora, Philadelphia (RP-A501)

Recruiting
  • Danon Disease
  • RP-A501
  • La Jolla, California
  • +2 more
Aug 24, 2021

Idiopathic Parkinson's Disease Trial in United States (CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]), Sham

Completed
Has Results
  • Idiopathic Parkinson's Disease
  • CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN])
  • Sham Surgery
  • Birmingham, Alabama
  • +8 more
Nov 8, 2022

Hypertrophic Cardiomyopathy Trial in United States (TN-201)

Not yet recruiting
  • Hypertrophic Cardiomyopathy
  • TN-201
  • Boston, Massachusetts
  • +6 more
Apr 18, 2023

Hemophilia B Trial (CSL222 (AAV5-hFIXco-Padua))

Not yet recruiting
  • Hemophilia B
  • CSL222 (AAV5-hFIXco-Padua)
  • (no location specified)
Aug 14, 2023

Hemophilia A Trial (Biospecimen Collection)

Not yet recruiting
  • Hemophilia A
  • Biospecimen Collection
  • (no location specified)
Oct 11, 2022

Pompe Disease Trial in Gainesville (genetic, drug, other)

Completed
  • Pompe Disease
  • Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase
  • +7 more
  • Gainesville, Florida
    Clinical and Translational Research Building (CTRB), University
Mar 24, 2022

Parkinson's Disease Trial run by the National Institute of Neurological Disorders and Stroke (NINDS) (Convection enhanced

Completed
  • Parkinson's Disease
  • Convection enhanced delivery/AAV2-GDNF
  • Bethesda, Maryland
    National Institutes of Health Clinical Center
Jan 26, 2023

Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy Trial in New York (AAVrh.10hFXN, serotype rh.10 adeno-associated virus

Recruiting
  • Friedreich Ataxia
  • +3 more
  • AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN
  • Prednisone
  • New York, New York
    Weill Cornell Medicine
Jul 15, 2022

Seroprevalence and Seroconversion of Antibodies to

Recruiting
  • Hemophilia A
  • Blood sample collection
  • Madison, Wisconsin
    Covance Inc
Sep 16, 2022

Parkinson's Disease Trial in San Francisco, Chicago (CERE-120: AAV2-NTN)

Completed
  • Parkinson's Disease
  • CERE-120: AAV2-NTN
  • San Francisco, California
  • +1 more
Nov 8, 2022

GSD1 Trial in Worldwide (DTX401)

Completed
Has Results
  • GSD1
  • DTX401
  • steroid regimen
  • Farmington, Connecticut
  • +5 more
Oct 25, 2022

Infantile-onset Pompe Disease Trial in Beijing (Genetic: GC301)

Recruiting
  • Infantile-onset Pompe Disease
  • Genetic: GC301
  • Beijing, Beijing, China
    Bayi Children's Hospital, Seventh Medical Center, PLA general ho
Sep 30, 2022

Achromatopsia Trial in Ann Arbor, London (adeno-associated virus vector AAV- CNGA3)

Completed
Has Results
  • Achromatopsia
  • adeno-associated virus vector AAV- CNGA3
  • Ann Arbor, Michigan
  • +1 more
Feb 10, 2022

Hemophilia B Trial in United States (AAV5-hFIXco-Padua (AMT-061))

Active, not recruiting
Has Results
  • Hemophilia B
  • AAV5-hFIXco-Padua (AMT-061)
  • Los Angeles, California
  • +3 more
May 17, 2022

Long Term Follow-Up Gene Therapy Study for XLRP RPGR

Recruiting
  • X-Linked Retinitis Pigmentosa
  • AAV-RPGR
  • Boston, Massachusetts
  • +3 more
Dec 5, 2022

Ornithine Transcarbamylase (OTC) Deficiency Trial in Worldwide (scAAV8OTC, Oral prednisone)

Completed
Has Results
  • Ornithine Transcarbamylase (OTC) Deficiency
  • scAAV8OTC
  • +2 more
  • Aurora, Colorado
  • +8 more
Jan 23, 2023

Glycogen Storage Disease Type IA Trial in Worldwide (genetic, other, drug)

Recruiting
  • Glycogen Storage Disease Type IA
  • DTX401
  • +3 more
  • Orange, California
  • +16 more
Jan 13, 2023

Adeno-Associated Virus Antibody Study in Subjects OTC

Recruiting
  • Ornithine Transcarbamylase Deficiency
  • +2 more
    • Akron, Ohio
    • +2 more
    Jul 22, 2022

    Hemophilia A, Gene Therapy Trial in Tianjin (Injection of GS001)

    Recruiting
    • Hemophilia A
    • Gene Therapy
    • Injection of GS001
    • Tianjin, Tianjin, China
      Chinese Academy of Medical Science and Blood Disease Hospital
    Sep 19, 2022

    Squamous Cell Head Neck Cancer, Radiation Induced Xerostomia, Salivary Hypofunction Trial run by the (AAV2hAQP1)

    Recruiting
    • Squamous Cell Head and Neck Cancer
    • +2 more
    • AAV2hAQP1
    • Bethesda, Maryland
      National Institutes of Health Clinical Center, 9000 Rockville Pi
    May 3, 2022

    Hemophilia B Trial in Worldwide (AAV5-hFIXco-Padua)

    Active, not recruiting
    Has Results
    • Hemophilia B
    • AAV5-hFIXco-Padua
    • Factor IX (FIX)
    • Phoenix, Arizona
    • +32 more
    Sep 26, 2022

    Congenital Adrenal Hyperplasia Trial in United States (AAV BBP-631)

    Recruiting
    • Congenital Adrenal Hyperplasia
    • AAV BBP-631
    • Los Angeles, California
    • +6 more
    Jul 29, 2022

    Determine Eligibility for CAH-301 [NCT04783181])

    Recruiting
    • Congenital Adrenal Hyperplasia
    • Pre-Screening
    • Los Angeles, California
      Science37
    Mar 1, 2022