Clinical Trials Directory

Trials / Conditions / Gene Therapy

Gene Therapy

17 registered clinical trials studyying Gene Therapy — 10 currently recruiting.

StatusTrialSponsorPhase
Not Yet RecruitingA Clinical Study of the Safety and Efficacy of Chemogenetics Therapy in the Treatment of Parkinson's Disease
NCT07085195
Ruijin HospitalEARLY_Phase 1
Not Yet RecruitingSafety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type
NCT06948019
BlackfinBio LtdPhase 1 / Phase 2
RecruitingA Study to Evaluate a Novel Gene Therapy in Patients With Relapsed and Refractory Multiple Myeloma
NCT07075185
Kelonia Therapeutics, Inc.Phase 1
RecruitingPerceptual Evaluation and Rehabilitation System Development for Congenital Hearing Loss
NCT07024524
Eye & ENT Hospital of Fudan UniversityN/A
Not Yet RecruitingA Prospective, Single-center, Open-label Clinical Study to Evaluate the Safety and Efficacy of Inhaled BMD003(
NCT06963008
Peking Union Medical College HospitalEARLY_Phase 1
RecruitingGrowth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene The
NCT05991336
Institute of Hematology & Blood Diseases Hospital, China—
CompletedNeeds Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy
NCT05044845
St. Jude Children's Research Hospital—
Enrolling By InvitationLong-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With nAMD
NCT05210803
AbbVie—
WithdrawnIntravitreal AAVCAGsCD59 for Advanced Dry Age-related Macular Degeneration (AMD) With Geographic Atrophy (GA)
NCT04358471
Hemera BiosciencesPhase 2
RecruitingGene Therapy for Chinese Hemophilia A
NCT04728841
Institute of Hematology & Blood Diseases Hospital, ChinaN/A
UnknownGene Therapy for X Linked Severe Combined Immunodeficiency
NCT04286815
Children's Hospital of Chongqing Medical UniversityN/A
CompletedDose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals
NCT03734588
Spark Therapeutics, Inc.Phase 1 / Phase 2
TerminatedGene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
NCT03520712
BioMarin PharmaceuticalPhase 1 / Phase 2
RecruitingPhase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
NCT03311503
David WilliamsPhase 1 / Phase 2
CompletedGene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
NCT03306277
Novartis Gene TherapiesPhase 3
CompletedSafety and Tolerability Study of AAV2-sFLT01 in Patients With Neovascular Age-Related Macular Degeneration (AM
NCT01024998
Genzyme, a Sanofi CompanyPhase 1
RecruitingCIBMTR Research Database
NCT01166009
Center for International Blood and Marrow Transplant Research—