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rAAV

Showing 26 - 50 of 79
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X-Linked Retinitis Pigmentosa Trial (rAAV2tYF-GRK1-hRPGRco)

Not yet recruiting
  • X-Linked Retinitis Pigmentosa
  • rAAV2tYF-GRK1-hRPGRco
  • (no location specified)
May 19, 2021

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

Withdrawn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

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

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

Leber Hereditary Optic Neuropathy Trial in Wuhan (rAAV2-ND4)

Active, not recruiting
  • Leber Hereditary Optic Neuropathy
  • Wuhan, Hubei, China
    Department of Ophthalmology，Tongji Hospital, Tongji Medical Coll
Sep 15, 2020

Choroideremia Trial in Tuebingen (rAAV2.REP1)

Completed
  • Choroideremia
  • rAAV2.REP1
  • Tuebingen, Germany
    University Hospital Tuebingen, Center for Ophthalmology
Oct 26, 2020

Duchenne Muscular Dystrophy Trial in Columbus (rAAV1.CMV.huFollistin344)

Completed
Has Results
  • Duchenne Muscular Dystrophy
  • rAAV1.CMV.huFollistin344
  • Columbus, Ohio
    Nationwide Children's Hospital
Apr 1, 2020

Acute LHON, Onset Within Three Months, Onset Between 3 to 6 Months Trial in Wuhan (rAAV2-ND4)

Unknown status
  • Acute LHON
  • +6 more
  • Wuhan, Hubei, China
    Department of Ophthalmology ，Tongji Hospital, Tongji Medical Col
Jul 27, 2020

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

Alpha 1-Antitrypsin Deficiency Trial in Gainesville, Worcester (rAAV2-CB-hAAT Gene Vector)

Completed
  • Alpha 1-Antitrypsin Deficiency
  • rAAV2-CB-hAAT Gene Vector
  • Gainesville, Florida
  • +1 more
Apr 8, 2020

Leber Hereditary Optic Neuropathy Trial in Paris (GS010)

Completed
  • Leber Hereditary Optic Neuropathy
  • GS010
  • Paris, France
    CIC du CHNO DES QUINZE-VINGTS
Jun 30, 2020

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

Dravet Syndrome Trial in Melbourne (ETX101)

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

Sanfilippo Syndrome B Trial in Paris (rAAV2/5-hNAGLU)

Completed
  • Sanfilippo Syndrome B
  • Paris, Le Kremlin-Bicetre Cedex, France
    Hopitaux Universitaires Paris-Sud
Nov 28, 2019

Neovascular (Wet) Age-related Macular Degeneration (nAMD) Trial (EXG102-031)

Not yet recruiting
  • Neovascular (Wet) Age-related Macular Degeneration (nAMD)
  • EXG102-031
  • (no location specified)
Jun 13, 2023

Alpha-1 Antitrypsin Deficiency Trial in Ireland, United States (rAAV1-CB-hAAT)

Completed
Has Results
  • Alpha-1 Antitrypsin Deficiency
  • Denver, Colorado
  • +3 more
Mar 18, 2019

Amyotrophic Lateral Sclerosis Trial (AMT-162)

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

Wilson's Disease Trial in Denmark, Germany, United States (VTX-801)

Recruiting
  • Wilson's Disease
  • VTX-801
  • Sacramento, California
  • +7 more
Dec 23, 2022

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

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

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

Dravet Syndrome Trial (ETX101)

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

Methylmalonic Acidemia Trial in United States (hLB-001)

Recruiting
  • Methylmalonic Acidemia
  • hLB-001
  • Atlanta, Georgia
  • +3 more
Aug 16, 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