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AAV

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CLN7 Trial in Dallas (AAV9/CLN7)

Recruiting
  • CLN7
  • AAV9/CLN7
  • Dallas, Texas
    Children' Health
May 16, 2022

AAV2hAQP1 for Radiation Induced Xerostomia

Enrolling by invitation
  • Radiation-induced Xerostomia
    • Stanford, California
    • +3 more
    Dec 22, 2022

    Phenylketonurias Trial in Bengbu (NGGT002)

    Recruiting
    • Phenylketonurias
    • NGGT002
    • Bengbu, Anhui, China
      The First Affiliated Hospital of Bengbu Medical College
    Sep 25, 2023

    Hemophilia A, Gene Therapy, Clotting Disorders Trial in Johannesburg, London, Southampton (Valoctocogene Roxaparvovec)

    Recruiting
    • Hemophilia A
    • +3 more
    • Valoctocogene Roxaparvovec
    • Seoul, Korea, Republic of
    • +8 more
    Jan 6, 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

    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

    Cocaine Dependence, in Remission Trial in Rochester (AAV8-hCocH)

    Recruiting
    • Cocaine Dependence, in Remission
    • Rochester, Minnesota
      Mayo Clinic in Rochester
    Oct 8, 2021

    Parkinson Disease Trial in United States (AAV-GAD)

    Enrolling by invitation
    • Parkinson Disease
    • AAV-GAD
    • West Bloomfield, Michigan
    • +4 more
    Jun 7, 2023

    Male Hemophilia B Previously Treated With Etranacogene

    Not yet recruiting
    • Hemophilia B
    • AAV5-hFIXco-Padua
    • (no location specified)
    Jul 31, 2023

    Rett Syndrome Trial (TSHA-102)

    Not yet recruiting
    • Rett Syndrome
    • TSHA-102
    • (no location specified)
    Nov 21, 2023

    Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF) Trial (DB-OTO)

    Not yet recruiting
    • Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
    • DB-OTO
    • (no location specified)
    Mar 28, 2023

    Long Term Follow-Up Gene Therapy Study for XLRP RPGR

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

    X-Linked Retinitis Pigmentosa Trial in Worldwide (Genetic: AAV5-RPGR 4e11, Genetic: AAV5-RPGR 2e11)

    Recruiting
    • X-Linked Retinitis Pigmentosa
    • Genetic: AAV5-RPGR 4e11
    • Genetic: AAV5-RPGR 2e11
    • Los Angeles, California
    • +9 more
    Aug 16, 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 A Trial in Tianjin (Single dose intravenous injection of BBM 002)

    Not yet recruiting
    • Hemophilia A
    • Single dose intravenous injection of BBM 002
    • Tianjin, Tianjin, China
      Institute of Hematology & Blood Diseases Hospital
    Jul 7, 2022

    Pompe Disease Trial in Saint Louis, New York, Durham (ACTUS-101)

    Recruiting
    • Pompe Disease
    • ACTUS-101
    • Saint Louis, Missouri
    • +2 more
    May 25, 2022

    Parkinson Disease Trial in West Bloomfield, Philadelphia, Morgantown (AAV-GAD, Sham Surgery)

    Recruiting
    • Parkinson Disease
    • AAV-GAD
    • Sham Surgery
    • West Bloomfield, Michigan
    • +2 more
    Jan 2, 2023

    Granulomatosis With Polyangiitis, Microscopic Polyangiitis, ANCA Associated Vasculitis Trial in Boston, Rochester, Philadelphia

    Not yet recruiting
    • Granulomatosis With Polyangiitis
    • +2 more
    • Boston, Massachusetts
    • +2 more
    Dec 29, 2022

    Aromatic Amino Acid Decarboxylase Deficiency Trial in Taipei (AAV2-hAADC)

    Active, not recruiting
    • Aromatic Amino Acid Decarboxylase Deficiency
    • Taipei, Taiwan
      National Taiwan University Hospital
    Nov 15, 2021

    Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)

    Active, not recruiting
    • Inherited Retinal Dystrophy Due to RPE65 Mutations
    • AAV2-hRPE65v2
    • (no location specified)
    Dec 9, 2021

    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

    OTC Deficiency Trial in Worldwide (genetic, other, drug)

    Recruiting
    • OTC Deficiency
    • DTX301
    • +4 more
    • Aurora, Colorado
    • +19 more
    Jul 29, 2022

    LCA5 Trial (AAV8.hLCA5)

    Not yet recruiting
    • LCA5
    • AAV8.hLCA5
    • (no location specified)
    Nov 22, 2022

    Parkinson's Disease Trial in Irvine, San Francisco, Columbus (AAV2-GDNF)

    Recruiting
    • Parkinson's Disease
    • AAV2-GDNF
    • Irvine, California
    • +2 more
    Feb 8, 2022

    Alzheimer's Disease Trial in San Diego, Cleveland (AAV2-BDNF Gene Therapy)

    Recruiting
    • Alzheimer's Disease
    • AAV2-BDNF Gene Therapy
    • San Diego, California
    • +1 more
    Sep 1, 2021