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Condition: Gene Therapy Synced from ClinicalTrials.gov · July 29, 2026

Recruiting studies: Gene Therapy

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

A Gaucher Disease Gene Therapy Trial With FLT201
Phase 3 · 33 location(s) · Spur Therapeutics · NCT07223944Research of Therapeutic Targets in the Frame of Nephronophthisis and Renal Associated Ciliopathies
NA · 1 location(s) · Imagine Institute · NCT06648044Phase 1/2 Trial of S241656 in Selected RAS/MAPK Mutation- Positive Malignancies
Phase 1/Phase 2 · 27 location(s) · Institut de Recherches Internationales Servier · NCT05786924Immunotherapy Based on Tumor Associated Antigen-specific Immune Effector Cells
Phase 1/Phase 2 · 1 location(s) · Shenzhen Geno-Immune Medical Institute · NCT03535246Multi-center Study of TBI-1301 (INN: Mipetresgene Autoleucel; Mip-cel) in Patients With NY-ESO-1 Positive Synovial Sarco
Phase 3 · 2 location(s) · Takara Bio Inc. · NCT07174427Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic Patients With Recurrent Pancreat
Phase 1 · 1 location(s) · GeneCradle Inc · NCT05860569Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
Phase 2 · 1 location(s) · Lexeo Therapeutics · NCT07721025A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease
Phase 1 · 14 location(s) · CRISPR Therapeutics · NCT06925542Clinical Efficacy of Tumour Treatment Vaccine (TTV) Combined With PD-1 in the Treatment of Relapsed Refractory Advanced
NA · 1 location(s) · Wuxi People's Hospital · NCT06430502Markers to Evaluate the Efficacy of PH-based Regimen as a Neoadjuvant Therapy for Operable HER2 Positive Breast Cancer
NA · 1 location(s) · Fudan University · NCT04281641Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodefic
Phase 1/Phase 2 · 1 location(s) · National Institute of Allergy and Infectious Diseases (NIAID · NCT01306019Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
EARLY_Phase 1 · 1 location(s) · Shanghai Siponuoyin Biotechnology Co Ltd · NCT07188012Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa
Phase 1/Phase 2 · 1 location(s) · Frontera Therapeutics · NCT06492850Gene Therapy for Diabetic Macular Edema
Phase 1 · 1 location(s) · Frontera Therapeutics · NCT05916391Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
NA · 1 location(s) · Shenzhen Geno-Immune Medical Institute · NCT03645460Phase 2 Trial of RGX-314 in Adults With Center Involved - Diabetic Macular Edema (CI - DME)
Phase 2 · 1 location(s) · Sierra Eye Associates · NCT06942520Effectiveness of Cognitive Stimulation Treatment in Patients With Parkinson's Disease
Phase N/A · 1 location(s) · Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico · NCT06323278HSV-tk and XRT and Chemotherapy for Newly Diagnosed GBM
Phase 1/Phase 2 · 1 location(s) · The Methodist Hospital Research Institute · NCT03603405Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated A
Phase 1 · 7 location(s) · Tenaya Therapeutics · NCT06228924Etiology and Treatment of Neonatal Seizure
Phase N/A · 1 location(s) · Children's Hospital of Fudan University · NCT03822741A Study to Evaluate the Efficacy and Safety of TL118 in Solid Tumors Patients
Phase 2 · 19 location(s) · Teligene US · NCT06010342Safety and Efficacy of RRG001 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)
Phase 1/Phase 2 · 1 location(s) · Shanghai Refreshgene Technology Co., Ltd. · NCT06141460A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265)
Phase 1/Phase 2 · 44 location(s) · Regeneron Pharmaceuticals · NCT06379789Adoptive T Cell Immunotherapy for Advanced Melanoma Using Engineered Lymphocytes
Phase 1 · 1 location(s) · Loyola University · NCT02870244Safety and Tolerability Study of ST-503 for Refractory Pain Due to Peripheral Neuropathy (Small Fiber Predominant, SFN)
Phase 1/Phase 2 · 11 location(s) · Sangamo Therapeutics · NCT06980948The HOPE Biobank Resource (BMT CTN 2402 HOPE)
Phase N/A · 6 location(s) · Medical College of Wisconsin · NCT07227155Study of Autophagy and the Effects of GALIG Gene Products in HIV-1 Infected Patients Who Are Under Antiretroviral Therap
Phase N/A · 1 location(s) · Centre Hospitalier Régional d'Orléans · NCT04160455Gene Therapy for Chinese Hemophilia A
NA · 1 location(s) · Institute of Hematology & Blood Diseases Hospital, China · NCT04728841Treatment Strategies and Survival Outcome for Non-small Cell Lung Cancer With Oncogenic Mutation
Phase 2 · 1 location(s) · Hunan Province Tumor Hospital · NCT04322890A Study to Evaluate the Safety and Tolerability of SCTB14 as First-Line Therapy in Non-Small Cell Lung Cancer.
Phase 3 · 1 location(s) · Sinocelltech Ltd. · NCT07362459
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.