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AAV-mediated factor VIII (FVIII) gene transfer

Showing 1 - 25 of >10,000
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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

Liver Biopsy Following Gene Therapy For Hemophilia

Not yet recruiting
  • Hemophilia A
  • Hemophilia B
  • Liver Biopsy
  • Memphis, Tennessee
    St. Jude Children's Research Hospital
Jun 27, 2023

Hemophilia A Trial in Worldwide (BAY2599023 (DTX201))

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

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

Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII Trial in United Kingdom, United States (Valoctocogene

Recruiting
  • Hemophilia A With Inhibitor
  • Hemophilia A With Anti Factor VIII
  • Valoctocogene roxaparvovec
  • Los Angeles, California
  • +4 more
Aug 2, 2022

Hemophilia A Trial in Milwaukee (Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human

Recruiting
  • Hemophilia A
  • Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII
  • Milwaukee, Wisconsin
    Froedtert Hospital and the Medical College of Wisconsin
Dec 21, 2022

Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited Trial in United States

Recruiting
  • Adeno-Associated Virus (AAV)
  • +16 more
  • SPK-8016
  • Los Angeles, California
  • +13 more
Feb 3, 2022

Severe Haemophilia A Trial in United Kingdom (valoctocogene roxaparvovec)

Active, not recruiting
  • Severe Haemophilia A
  • valoctocogene roxaparvovec
  • Basingstoke, United Kingdom
  • +5 more
Feb 22, 2022

Multiple System Atrophy Trial in Irvine, Columbus (AAV2-GDNF gene therapy, Sham (Placebo) Surgery)

Recruiting
  • Multiple System Atrophy
  • AAV2-GDNF gene therapy
  • Sham (Placebo) Surgery
  • Irvine, California
  • +1 more
Jan 18, 2023

Wilson Disease Trial in Spain, United States (genetic, other, drug)

Recruiting
  • Wilson Disease
  • UX701
  • +3 more
  • Los Angeles, California
  • +11 more
Jul 22, 2022

Severe Hemophilia A Subjects Who Received BMN 270 inPrior

Enrolling by invitation
  • Hemophilia A
    • London, United Kingdom
      Royal London Hospital, Barts and the London Hemophilia Center
    Mar 13, 2023

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

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

    Melanoma Trial in Boston (Autologous, lethally irradiated melanoma cells)

    Active, not recruiting
    • Melanoma
    • Autologous, lethally irradiated melanoma cells
    • Boston, Massachusetts
    • +1 more
    Feb 2, 2022

    Hemophilia A Trial in Worldwide (SPK-8011)

    Recruiting
    • Hemophilia A
    • SPK-8011
    • Sacramento, California
    • +18 more
    Jul 15, 2022

    Hemophilia A Trial in Worldwide (BAX 888)

    Active, not recruiting
    • Hemophilia A
    • BAX 888
    • Phoenix, Arizona
    • +15 more
    Nov 23, 2021

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

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

    GSD1 Trial in Worldwide (DTX401)

    Completed
    Has Results
    • GSD1
    • DTX401
    • steroid regimen
    • Farmington, Connecticut
    • +5 more
    Oct 25, 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

    Hemophilia A Trial in Tianjin (Single dose intravenous injection of BBM-H803)

    Recruiting
    • Hemophilia A
    • Single dose intravenous injection of BBM-H803
    • Tianjin, Tianjin, China
      Institute of Hematology & Blood Diseases Hospital Chinese Academ
    Oct 31, 2023

    Hemophilia A Trial in Worldwide (PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy)

    Active, not recruiting
    • Hemophilia A
    • PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
    • Mountain View, California
    • +34 more
    Jan 26, 2022

    Hemophilia B Trial in Washington (SB-FIX)

    Terminated
    • Hemophilia B
    • SB-FIX
    • Washington, District of Columbia
      Georgetown University Medical Center
    Mar 8, 2022

    Hemophilia B, Hemophilia A Trial in Worldwide (Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement

    Recruiting
    • Hemophilia B
    • Hemophilia A
    • Standard of Care FIX Replacement therapy
    • Standard of Care FVIII Replacement therapy
    • Palo Alto, California
    • +77 more
    Dec 29, 2022

    Phenylketonuria (PKU) Trial in United Kingdom, United States (BMN 307)

    Active, not recruiting
    • Phenylketonuria (PKU)
    • BMN 307
    • Tampa, Florida
    • +3 more
    Oct 6, 2021

    Ornithine Transcarbamylase (OTC) Deficiency Trial in Worldwide (scAAV8OTC, Oral prednisone)

    Completed
    Has Results
    • Ornithine Transcarbamylase (OTC) Deficiency
    • scAAV8OTC
    • +2 more
    • Aurora, Colorado
    • +8 more
    Jan 23, 2023

    Hereditary Angioedema, HAE Trial in United States (Dose 1 of BMN 331, Dose 2 of BMN 331, Dose 3 of BMN 331)

    Recruiting
    • Hereditary Angioedema
    • HAE
    • Dose 1 of BMN 331
    • +4 more
    • Birmingham, Alabama
    • +11 more
    Jan 26, 2023