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rAAV

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STUDY
CONDITIONS
INTERVENTIONS
LOCATIONS

Hemophilia B Without Inhibitor Trial (Low dose KL001, Middle dose KL001, High dose KL001)

Not yet recruiting
  • Hemophilia B Without Inhibitor
  • Low dose KL001
  • +2 more
  • (no location specified)
Nov 5, 2023

Hemophilia B Without Inhibitor Trial (Low dose KL001, Middle dose KL001, High dose KL001)

Not yet recruiting
  • Hemophilia B Without Inhibitor
  • Low dose KL001
  • +2 more
  • (no location specified)
Nov 5, 2023

HIV Trial (Low dose KL-HIV-Tri01, Middle dose KL-HIV-Tri01, High dose KL-HIV-Tri01)

Not yet recruiting
  • HIV Infections
  • Low dose KL-HIV-Tri01
  • +2 more
  • (no location specified)
Nov 5, 2023

Bietti's Crystalline Dystrophy Trial in Beijing (ZVS101e)

Recruiting
  • Bietti's Crystalline Dystrophy
  • Beijing, Beijing, China
    Peking University Third Hospital
Feb 2, 2023

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

Achromatopsia Trial in Tuebingen (rAAV.hCNGA3)

Recruiting
  • Achromatopsia
  • Tuebingen, Germany
    University Hospital Tuebingen, Center for Ophthalmology
Dec 7, 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

Bietti's Crystalline Dystrophy Trial in China (ZVS101e)

Recruiting
  • Bietti's Crystalline Dystrophy
  • Beijing, Beijing, China
  • +3 more
Apr 16, 2023

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

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

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

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

Retinitis Pigmentosa Trial in Tuebingen (subretinal injection of rAAV.hPDE6A)

Active, not recruiting
  • Retinitis Pigmentosa
  • subretinal injection of rAAV.hPDE6A
  • Tuebingen, Germany
    Universitätsklinikum Tübingen, Department für Augenheilkunde
Dec 7, 2022

Retinal Disease, Retinitis Pigmentosa Trial in Riyadh (Subretinal administration of rAAV2-VMD2-hMERTKRecombinant

Completed
  • Retinal Disease
  • Retinitis Pigmentosa
  • Subretinal administration of rAAV2-VMD2-hMERTKRecombinant Adeno-Associated Virus
  • Riyadh, Saudi Arabia
    King Khaled Eye Specialist Hospital
Jan 25, 2022

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

MPS I Trial in Oakland (SB-318)

Terminated
  • MPS I
  • SB-318
  • Oakland, California
    UCSF Benioff Children's Hospital Oakland
Jan 6, 2023

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

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

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

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

Osteoarthritis, Knee, Osteo Arthritis Knee, Knee Osteoarthritis Trial (GNSC-001, transient immune-modulation, Placebo)

Not yet recruiting
  • Osteoarthritis, Knee
  • +9 more
  • GNSC-001
  • +2 more
  • (no location specified)
Apr 19, 2023

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

Bietti's Crystalline Dystrophy Trial in Beijing (Gene Replacement Therapy by rAAV2/8-hCYP4V2)

Recruiting
  • Bietti's Crystalline Dystrophy
  • Gene Replacement Therapy by rAAV2/8-hCYP4V2
  • Beijing, Beijing, China
    Beijing Tongren Hospital
May 6, 2021

Study of Patients With MPS IIIB Treated With ABO-101

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

Choroideremia Trial in Edmonton (rAAV2.REP1 vector)

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

X-Linked Retinitis Pigmentosa Trial in United States (rAAV2tYF-GRK1-RPGR)

Recruiting
  • X-Linked Retinitis Pigmentosa
  • rAAV2tYF-GRK1-RPGR
  • Jacksonville, Florida
  • +6 more
Jul 13, 2021

Mucopolysaccharidosis II, MPS II Trial in United States (SB-913)

Terminated
  • Mucopolysaccharidosis II
  • MPS II
  • SB-913
  • Oakland, California
  • +4 more
Oct 21, 2022

Huntington Disease Trial in United States (intra-striatal rAAV5-miHTT, Imitation (sham) surgery)

Recruiting
  • Huntington Disease
  • intra-striatal rAAV5-miHTT
  • Imitation (sham) surgery
  • Birmingham, Alabama
  • +11 more
Dec 2, 2021