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Gene therapy

Showing 1 - 25 of >10,000
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Transfusion-dependent Beta-thalassemia After Gene Therapy

Recruiting
  • Transfusion-dependent Beta-Thalassemia
  • Gene Therapy
  • Gene therapy
  • Tianjin, Tianjin, China
    Regenerative Medicine Center
Aug 6, 2023

Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)

Not yet recruiting
  • Sickle Cell Disease
  • Interview
  • Memphis, Tennessee
    St. Jude Children's Research Hospital
Mar 30, 2023

To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)

Not yet recruiting
  • To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)
  • (no location specified)
Aug 29, 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

Age-Related Macular Degeneration Trial in Toronto (NG101 AAV gene therapy)

Not yet recruiting
  • Age-Related Macular Degeneration
  • NG101 AAV gene therapy
  • Toronto, Ontario, Canada
    Vitreous Retina Macula Specialists of Toronto
Aug 2, 2023

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

Lysosomal Storage Disease, Metachromatic Leukodystrophy Trial in Milan (OTL-200 Gene Therapy)

Active, not recruiting
  • Lysosomal Storage Disease
  • Metachromatic Leukodystrophy
  • OTL-200 Gene Therapy
  • Milan, Italy
    Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR
Nov 4, 2022

Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC Trial (LX2020)

Not yet recruiting
  • Arrhythmogenic Cardiomyopathy
  • +2 more
  • LX2020
  • (no location specified)
Oct 25, 2023

Sensorineural Hearing Loss, Bilateral Trial in Philadelphia (AAVAnc80-hOTOF via Akouos Delivery Device)

Recruiting
  • Sensorineural Hearing Loss, Bilateral
  • AAVAnc80-hOTOF via Akouos Delivery Device
  • Philadelphia, Pennsylvania
    Children's Hospital of Philadelphia
Apr 18, 2023

Leber Congenital Amaurosis Trial in Shanghai (HG004)

Recruiting
  • Leber Congenital Amaurosis
  • HG004
  • Shanghai, Shanghai, China
    Xinhua Hospital affiliated with Shanghai Jiao Tong UniversitySch
Oct 12, 2023

Leber Congenital Amaurosis Trial in Shanghai (HG004)

Recruiting
  • Leber Congenital Amaurosis
  • HG004
  • Shanghai, Shanghai, China
    Xinhua Hospital affiliated with Shanghai Jiao Tong University Sc
Sep 26, 2023

To Evaluate the Safety and Tolerability of Intravitreal Injection of LX109 in Patients With nAMD Trial (LX109)

Not yet recruiting
  • To Evaluate the Safety and Tolerability of Intravitreal Injection of LX109 in Patients With nAMD
  • (no location specified)
Sep 1, 2023

X-linked Retinoschisis Trial (ATSN-201)

Not yet recruiting
  • X-linked Retinoschisis
  • ATSN-201
  • (no location specified)
May 26, 2023

Hemophilia A, Gene Therapy Trial in Tianjin (Injection of GS001)

Recruiting
  • Hemophilia A
  • Gene Therapy
  • Injection of GS001
  • Tianjin, Tianjin, China
    Chinese Academy of Medical Science and Blood Disease Hospital
Sep 19, 2022

Amyotrophic Lateral Sclerosis Trial (AMT-162)

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

Hemophilia B Trial (BBM-H901)

Not yet recruiting
  • Hemophilia B
  • (no location specified)
Feb 6, 2023

Hypertriglyceridemia, Familial Trial in Nanjing (GC304)

Not yet recruiting
  • Hypertriglyceridemia, Familial
  • GC304
  • Nanjing, China
    Affiliated Jinling Hospital, Medical School, Nanjing University
May 5, 2023

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

Dravet Syndrome Trial in Melbourne (ETX101)

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

Retinitis Pigmentosa Trial in Pittsburgh, Paris (SPVN06)

Not yet recruiting
  • Retinitis Pigmentosa
  • Pittsburgh, Pennsylvania
  • +1 more
Feb 17, 2023

Gaucher Disease, Type 3 Trial (Gene therapy, Enzyme Replacement Agent)

Not yet recruiting
  • Gaucher Disease, Type 3
  • Gene therapy
  • Enzyme Replacement Agent
  • (no location specified)
Apr 14, 2023

Age-Related Macular Degeneration Trial (KH631)

Not yet recruiting
  • Age-Related Macular Degeneration
  • (no location specified)
Dec 10, 2022

Leber Hereditary Optic Neuropathy (LHON) Trial (NFS-02 Injection)

Not yet recruiting
  • Leber Hereditary Optic Neuropathy (LHON)
  • NFS-02 Injection
  • (no location specified)
Apr 6, 2023

Muscular Atrophy, Spinal Trial (risdiplam)

Not yet recruiting
  • Muscular Atrophy, Spinal
  • (no location specified)
May 8, 2023

Drug Resistant Epilepsy Trial (lentiviral gene therapy)

Not yet recruiting
  • Drug Resistant Epilepsy
  • lentiviral gene therapy
  • (no location specified)
May 17, 2022