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rAAV9

Showing 1 - 25 of 44
STUDY
CONDITIONS
INTERVENTIONS
LOCATIONS
LAST UPDATED

Mucopolysaccharidosis Type 3 B Trial in Worldwide (rAAV9.CMV.hNAGLU)

Terminated
  • Mucopolysaccharidosis Type 3 B
  • rAAV9.CMV.hNAGLU
  • Columbus, Ohio
  • +3 more
Apr 29, 2022

Canavan Disease Trial in Gainesville (rAAV9-CB6-AspA)

Available
  • Canavan Disease
  • Gainesville, Florida
    University of Florida
Apr 7, 2022

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

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

Study of Patients With MPS IIIB Treated With ABO-101

Terminated
  • Mucopolysaccharidosis III-B
  • ABO-101
  • Columbus, Ohio
  • +2 more
May 25, 2022

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

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

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

Dravet Syndrome Trial in Melbourne (ETX101)

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

X-linked Retinoschisis Trial in Xiaogan (ZM-01-L, ZM-01-H)

Recruiting
  • X-linked Retinoschisis
  • Xiaogan, Hubei, China
    Wuhan University Renmin Hospital affiliated with Hanchuan Hospit
Sep 29, 2023

X-linked Retinoschisis Trial in United States (rAAV2tYF-CB-hRS1)

Active, not recruiting
Has Results
  • X-linked Retinoschisis
  • rAAV2tYF-CB-hRS1
  • San Francisco, California
  • +8 more
Jul 18, 2022

Dravet Syndrome Trial (ETX101)

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

Canavan Disease Trial in Dayton (rAAV-Olig001-ASPA, Levetiracetam, Prednisone)

Enrolling by invitation
  • Canavan Disease
  • Dayton, Ohio
    Dayton Children's Hospital
Jun 23, 2022

Gaucher Disease Trial in Durham, Fairfax (LY3884961, Methylprednisolone, Sirolimus)

Not yet recruiting
  • Gaucher Disease
  • LY3884961
  • +3 more
  • Durham, North Carolina
  • +1 more
Aug 1, 2022

Retinitis Pigmentosa Trial (ZVS203e)

Not yet recruiting
  • Retinitis Pigmentosa
  • (no location specified)
Mar 27, 2023

Non-syndromic Retinitis Pigmentosa Trial in Pittsburgh, Paris, London (Gene therapy: GS030-DP AND Medical device: GS030-MD)

Recruiting
  • Non-syndromic Retinitis Pigmentosa
  • Gene therapy: GS030-DP AND Medical device: GS030-MD
  • Pittsburgh, Pennsylvania
  • +2 more
Jul 25, 2022

Amyotrophic Lateral Sclerosis Trial (AMT-162)

Not yet recruiting
  • Amyotrophic Lateral Sclerosis
  • (no location specified)
Oct 20, 2023

Choroideremia Trial in Edmonton (rAAV2.REP1 vector)

Completed
  • Choroideremia
  • rAAV2.REP1 vector
  • Edmonton, Alberta, Canada
    University of Alberta
May 16, 2022

Duchenne Muscular Dystrophy Trial in Chengdu (JWK007 Single intravenous infusion administration)

Recruiting
  • Duchenne Muscular Dystrophy
  • JWK007 Single intravenous infusion administration
  • Chengdu, Sichuan, China
    West China Hospital, Sichuan University
Oct 29, 2023

Huntington Disease Trial (Intraparenchymal rAAV1 - (mi)RNA HTT)

Withdrawn
  • Huntington Disease
  • Intraparenchymal rAAV1 - (mi)RNA HTT
  • (no location specified)
Aug 9, 2021

Leber Hereditary Optic Neuropathy Trial in Worldwide (GS010, Placebo)

Active, not recruiting
  • Leber Hereditary Optic Neuropathy
  • GS010
  • Placebo
  • Pasadena, California
  • +12 more
Aug 2, 2022

Spinal Muscular Atrophy Type I Trial in Shanghai, Hangzhou (EXG001-307 injection)

Recruiting
  • Spinal Muscular Atrophy Type I
  • EXG001-307 injection
  • Shanghai, Shanghai, China
  • +1 more
Nov 7, 2022

Amaurosis of Leber, Retinal Diseases Trial in Gainesville, Philadelphia (rAAV2-CBSB-hRPE65)

Active, not recruiting
  • Amaurosis of Leber
  • Retinal Diseases
  • rAAV2-CBSB-hRPE65
  • Gainesville, Florida
  • +1 more
Dec 13, 2021

Huntington Disease Trial in Germany, Poland, United Kingdom (intra-striatal rAAV5-miHTT)

Recruiting
  • Huntington Disease
  • intra-striatal rAAV5-miHTT
  • Düsseldorf, Germany
  • +5 more
Feb 17, 2022

AADC Deficiency Trial in San Francisco, Columbus (AAV2-hAADC)

Recruiting
  • AADC Deficiency
  • San Francisco, California
  • +2 more
Jan 14, 2022