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AAV9

Showing 1 - 25 of 176
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Dravet Syndrome Trial in Melbourne (ETX101)

Not yet recruiting
  • Dravet Syndrome
  • Melbourne, Australia
    The Royal Children's Hospital
Oct 30, 2023

AAV9 Neutralizing Antibody Seroconversion in Household Contacts.

Recruiting
  • Household Contacts
  • Blood Draw
  • Baltimore, Maryland
    Kennedy Krieger Institute
Oct 4, 2022

CLN7 Trial in Dallas (AAV9/CLN7)

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

Lysosomal Diseases, Gangliosidosis, GM1 Trial run by the NHGRI (biological, drug, diagnostic test, other, procedure, behavioral)

Recruiting
  • Lysosomal Diseases
  • +2 more
  • AAV9-GLB1
  • +20 more
  • Bethesda, Maryland
    National Institutes of Health Clinical Center
Jan 19, 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, Philadelphia (RP-A501)

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

Rett Syndrome Trial (TSHA-102)

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

Mesial Temporal Lobe Epilepsy Trial in Columbus (AAV9-hSyn1-miGRIK2)

Not yet recruiting
  • Mesial Temporal Lobe Epilepsy
  • AAV9-hSyn1-miGRIK2
  • Columbus, Ohio
    Ohio State University
Sep 25, 2023

Rett Syndrome Trial in Montréal (TSHA-102)

Recruiting
  • Rett Syndrome
  • TSHA-102
  • MontrĂ©al, Canada
    Taysha Study Site
Nov 8, 2022

Neuronal Ceroid Lipofuscinosis Type 2 Trial in London (RGX-381)

Not yet recruiting
  • Neuronal Ceroid Lipofuscinosis Type 2
  • RGX-381
  • London, United Kingdom
    Greater Ormond Street Hospital
Mar 17, 2023

CLN3, Batten Disease Trial in Columbus (AT-GTX-502)

Active, not recruiting
  • CLN3
  • Batten Disease
  • AT-GTX-502
  • Columbus, Ohio
    Nationwide Children's Hospital
Jul 18, 2022

AMN, AMN Gene Mutation, X-ALD Trial in Worcester (SBT101, Imitation Procedure)

Recruiting
  • AMN
  • +2 more
  • SBT101
  • Imitation Procedure
  • Worcester, Massachusetts
    University of Massachusetts Chan Medical School
Jun 1, 2022

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

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

Dravet Syndrome Trial (ETX101)

Not yet recruiting
  • Dravet Syndrome
  • (no location specified)
Jun 10, 2022

Duchenne Muscular Dystrophy Trial in Columbus (scAAV9.U7.ACCA)

Active, not recruiting
  • Duchenne Muscular Dystrophy
  • scAAV9.U7.ACCA
  • Columbus, Ohio
    Nationwide Children's Hospital
Nov 25, 2021

Hypertrophic Cardiomyopathy Trial in United States (TN-201)

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

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

Variant Late-Infantile Neuronal Ceroid Lipofuscinosis Trial in Columbus (AT-GTX-501)

Completed
  • Variant Late-Infantile Neuronal Ceroid Lipofuscinosis
  • AT-GTX-501
  • Columbus, Ohio
    Nationwide Children's Hosptial
Jan 21, 2022

Gene Editing as a Therapeutic Approach for Rett Syndrome

Recruiting
  • Rett Syndrome
  • Gene editing in vitro
  • Siena, Italy
    University of Siena
Feb 13, 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

Choroideremia, X-Linked Retinitis Pigmentosa Trial in Worldwide (BIIB111, BIIB112)

Enrolling by invitation
  • Choroideremia
  • X-Linked Retinitis Pigmentosa
  • BIIB111
  • BIIB112
  • Los Angeles, California
  • +22 more
Apr 20, 2022

Hemophilia A Trial in Worldwide (BAY2599023 (DTX201))

Active, not recruiting
  • Hemophilia A
  • BAY2599023 (DTX201)
  • Little Rock, Arkansas
  • +12 more
Jan 5, 2023

X-Linked Retinitis Pigmentosa Trial in United Kingdom, United States (AAV2/5-RPGR)

Completed
  • X-Linked Retinitis Pigmentosa
  • AAV2/5-RPGR
  • Boston, Massachusetts
  • +4 more
Dec 15, 2021

Choroideremia Trial in London, Oxford (AAV-mediated REP1 gene replacement)

Completed
  • Choroideremia
  • AAV-mediated REP1 gene replacement
  • London, United Kingdom
  • +1 more
Aug 4, 2021

Choroideremia Trial in Worldwide (BIIB111)

Completed
  • Choroideremia
  • BIIB111
  • Los Angeles, California
  • +17 more
Dec 1, 2021