Clinical trials
Gene Therapy clinical trials
Below are recruiting gene therapy clinical trials, each written for real people, not researchers. We’re tracking 191 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06973356Recruiting
CAR-T therapy for severe, hard-to-treat ITP
This trial tests a new type of immunotherapy called CAR-T cells that target two proteins (CD19 and BCMA) on immune cells. It is for adults with severe ITP that has not improved with other treatments. The goal is to reset the immune system to stop attacking platelets.
Changsha, HunanAges 18 years+ - NCT03311503RecruitingPhase 1/Phase 2
Gene therapy for X-linked SCID in young children
This trial tests a gene therapy using a lentivirus to give missing immune system instructions in children with X-linked SCID (SCID-X1). It also uses a low dose of targeted chemotherapy (busulfan) to help the new genes work, and it requires long-term follow-up for safety.
Los Angeles, CaliforniaAges birth–5 years - NCT05788536RecruitingPhase 1/Phase 2
Gene therapy study for OTOF-related hearing loss
This trial tests a one-time gene therapy delivered into the ear to improve hearing in people with certain OTOF gene changes. It studies safety and how well the treatment can work, especially in children and infants, as doctors monitor hearing tests for up to 48 weeks.
Los Angeles, CaliforniaAges Any age - NCT05302271RecruitingPhase 1
Gene therapy for the heart problems in Friedreich's ataxia
This trial tests a one-time gene therapy that delivers a corrected copy of the frataxin gene directly to the heart. It aims to improve or stabilize heart function in people with Friedreich's ataxia who have early or advanced heart involvement.
New York, New YorkAges 12–50 - NCT07775313Enrolling by invitationPhase 1/Phase 2
Gene therapy for WHIM syndrome
This trial tests a one-time gene therapy treatment for WHIM syndrome, a rare immune disorder. It aims to fix the genetic cause to improve your body's ability to fight infections. Your stem cells will be collected, edited in a lab, and given back to you after some chemotherapy to prepare your body.
Bethesda, MarylandAges 3–75 - NCT04912843RecruitingPhase 2/Phase 3
Gene therapy for inherited optic nerve vision loss
This trial tests a gene therapy injection into the eye to improve vision in people with a specific type of inherited optic nerve disease called Leber’s hereditary optic neuropathy (LHON). It is aimed at people who have a particular genetic change and a history of vision loss lasting long enough to measure change, while checking for safety first.
BeijingAges 12–75 - NCT04676048RecruitingPhase 1/Phase 2
Gene therapy study for adults with hemophilia A
This Phase 1/2 study tests a one-time gene therapy (ASC618) to help the body make clotting factor for people with hemophilia A. It may reduce bleeding and the need for factor infusions, but you must meet several safety and eligibility checks.
Little Rock, ArkansasAges 18 years+ - NCT03466463Recruiting
Gene therapy for severe Crigler-Najjar liver jaundice
This trial studies a gene therapy for people with severe Crigler-Najjar syndrome, a condition that causes high bilirubin (jaundice) due to a UGT1A1 gene change. It may help reduce the need for constant bilirubin-lowering treatment like phototherapy.
ClamartAges 9 years+ - NCT05207657RecruitingPhase 1/Phase 2
Gene therapy for p47 form of chronic granulomatous disease
This trial tests an individualized gene therapy made from your own stem cells to help the immune system work better in p47-related chronic granulomatous disease (CGD). It’s designed for children who have serious infections or inflammation despite usual treatments and who don’t have a fully matched bone marrow donor.
LondonAges 1.9 years+ - NCT05858983RecruitingPhase 1/Phase 2
Gene therapy for certain RPE65 inherited eye conditions
This early-stage trial tests a gene therapy aimed at treating inherited retinal dystrophy caused by changes in the RPE65 gene on both copies. It may help slow vision loss or improve eye function, but the study is mainly about safety and learning how the treatment works.
Beijing, Beijing MunicipalityAges 8–45 - NCT05874310RecruitingEarly Phase 1
Gene therapy trial for RPGR-related vision loss
This early study tests a gene therapy designed for people whose vision problems are caused by an RPGR genetic change. It may help slow or improve the eye disease by treating the underlying cause.
Beijing, Beijing MunicipalityAges 8–45 - NCT05916391RecruitingPhase 1
Gene therapy for diabetic macular swelling affecting vision
This early-stage (Phase 1) study tests a new gene therapy to improve vision for adults with diabetic macular edema. It mainly looks at whether the treatment can be safely given and may help your vision over time.
Tianjin, Tianjin,ChinaAges 18 years+ - NCT07826767RecruitingPhase 1/Phase 2
Gene therapy for inherited immune disease
This trial tests a new gene therapy (SGX-001) for chronic granulomatous disease caused by a specific genetic change. The treatment tries to fix the gene in your immune cells so they can fight infections better, and the trial will follow participants for up to 15 years to make sure it's safe and effective.
UlmAges 1.5 years+ - NCT06207552RecruitingEarly Phase 1
Gene therapy study for children with Fabry disease
This study tests a new gene therapy for children aged 7 to 17 with Fabry disease. The treatment aims to fix the faulty gene that causes the condition, which may help reduce symptoms and prevent organ damage.
ShanghaiAges 7–18 - NCT06492863RecruitingPhase 1/Phase 2
Gene therapy (FT-003) for wet AMD
This trial tests a new gene therapy, FT-003, for people with wet AMD. It aims to see if a single treatment can help stop vision loss and may reduce the need for frequent eye injections.
Tianjin, Tianjin,ChinaAges 50–80 - NCT06492876RecruitingPhase 1/Phase 2
Gene therapy trial for diabetic macular edema (DME)
This trial tests a gene therapy for diabetic macular edema (DME), a condition where fluid builds up in the central part of the eye's retina. It aims to improve vision and reduce the need for frequent eye injections.
Tianjin, Tianjin,ChinaAges 18–74 - NCT07807111RecruitingPhase 1/Phase 2
Gene therapy for advanced rod-cone dystrophy
This study tests a new gene therapy for people with advanced rod-cone dystrophy, a genetic condition that causes progressive vision loss. The treatment is given as an injection into the eye, and the study aims to see if it is safe and if it can help improve or preserve vision.
GhentAges 18 years+ - NCT03996824Recruiting
Gene therapy study for certain vestibular schwannoma surgeries
This trial tests delivering gene therapy into the inner-ear hearing balance nerve cells during surgery for vestibular schwannoma. It may help by improving how the inner ear functions after tumor removal, but you must meet several specific surgery and safety requirements.
Le Kremlin-Bicêtre, Île-de-France RegionAges 18 years+ - NCT07721025RecruitingPhase 2
Gene therapy trial for Friedreich ataxia heart disease
This trial tests a one-time gene therapy called LX2006 for heart problems caused by Friedreich ataxia. The goal is to see if it can improve heart function and structure.
Tampa, FloridaAges 6 years+ - NCT07690527Enrolling by invitationPhase 1
Long-term follow-up for RB001 gene therapy in children with Phelan-McDermid syndrome
This study follows children who completed the RB001 gene therapy trial for Phelan-McDermid syndrome (caused by changes in the SHANK3 gene) to monitor their health and progress over a longer period of time.
Beijing, ChinaAges Any age - NCT07673809RecruitingPhase 1/Phase 2
Gene therapy trial for Duchenne muscular dystrophy in young boys
This trial tests a gene therapy called GNR-097 for boys with Duchenne muscular dystrophy (DMD). The therapy aims to help improve muscle function, and the study will check how safe and effective it is.
MinskAges 4–9 - NCT06399107RecruitingPhase 1/Phase 2
Gene therapy trial for sickle cell disease using BAH243
This trial tests a gene therapy that uses a modified virus to deliver a healthy gene to your blood stem cells. It aims to help people with sickle cell disease produce normal red blood cells and reduce painful crises.
Beijing, Beijing MunicipalityAges 2–90 - NCT06141460RecruitingPhase 1/Phase 2
Gene therapy trial for wet age-related macular degeneration
This trial tests a new gene therapy called RRG001 for people with the 'wet' form of age-related macular degeneration. It aims to treat leaky blood vessels in the eye that can cause vision loss, possibly reducing the need for frequent eye injections.
Tianjin, Tianjin MunicipalityAges 50 years+ - NCT07643844RecruitingPhase 1
Gene therapy for propionic acidemia in infants
This trial tests a new gene therapy treatment designed to help the body process certain amino acids better in infants with propionic acidemia. The therapy uses a modified virus to deliver a working copy of a faulty gene, potentially reducing dangerous acid buildups and hospital visits.
Rochester, MinnesotaAges 6 months–2 years
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Common questions
- Are there clinical trials for gene therapy?
- Yes. Clin2 currently lists 191 recruiting gene therapy studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a gene therapy trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a gene therapy trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.