Clinical trials
Gene Therapy clinical trials
Below are recruiting gene therapy clinical trials, each written for real people, not researchers. We’re tracking 207 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06391736RecruitingPhase 1/Phase 2
Gene therapy for late-onset Pompe disease
This trial tests a new gene therapy for late-onset Pompe disease. The therapy aims to help your body produce the enzyme needed to break down glycogen, which may improve muscle and breathing function.
BeijingAges 6 years+ - 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 - NCT06421831RecruitingPhase 1/Phase 2
Gene therapy trial for spinal muscular atrophy type 3
This trial is testing a one-time gene therapy drug for people with SMA type 3. The goal is to see if it can safely improve muscle function and strength.
Beijing, Beijing MunicipalityAges 2 years+ - NCT06458595RecruitingPhase 1/Phase 2
Gene therapy for wet age-related macular degeneration (nAMD)
This trial tests a gene therapy (KH658) as a one-time treatment for wet macular degeneration. It's for people whose condition has been stabilized with anti-VEGF injections, and aims to see if it is safe and can reduce the need for frequent eye injections.
TianjinAges 50–85 - NCT06508775Recruiting
Long-term checkup for Miltenyi cell therapy patients
This study is a long-term check-in for people who received a special type of immunotherapy (CAR T cells) made by Miltenyi. It helps researchers see how you're doing over time after your treatment.
BerlinAges Any age - NCT06517888RecruitingPhase 1/Phase 2
Gene therapy for hearing loss from vestibular schwannoma
This trial tests a gene therapy injected into the ear to slow tumor growth and prevent further hearing loss in people with a vestibular schwannoma that is getting worse. It's for those who already have severe hearing loss in the affected ear.
Baltimore, MarylandAges 18 years+ - NCT06641154RecruitingPhase 1/Phase 2
Gene therapy for severe Crigler-Najjar syndrome
This trial tests a gene therapy for children with severe Crigler-Najjar syndrome type I. It aims to reduce the need for daily phototherapy by fixing the faulty gene.
MoscowAges 3 months–10 years - NCT06646640Recruiting
Long-term follow-up for hemoglobin disorder treatments
This study tracks people with sickle cell disease or thalassemia who have had a bone marrow transplant or gene therapy in the past 15 years. It aims to monitor their health over time to understand long-term effects.
Memphis, TennesseeAges Any age - NCT06731933RecruitingPhase 2
Gene therapy for skin cancer in recessive dystrophic epidermolysis bullosa
This trial tests a gene therapy called BVEC to see if it can prevent squamous cell carcinoma (a type of skin cancer) from coming back in people with a severe form of epidermolysis bullosa (fragile skin). It is for adults who have had a confirmed SCC and are willing to use the gene therapy.
Redwood City, CaliforniaAges 18 years+ - NCT06817382RecruitingPhase 1
Gene therapy for young boys with Duchenne muscular dystrophy
This study tests a one-time gene therapy given by injection into the spinal fluid (intrathecal) for boys with Duchenne muscular dystrophy. It aims to see if the treatment is safe and how it spreads in the body, and may offer a new way to slow the disease.
Little Rock, ArkansasAges 2–4 - NCT06826612RecruitingPhase 1/Phase 2
Gene therapy trial for early Huntington's disease
This trial tests a gene therapy called SPK-10001 for people with early-stage Huntington's disease. It aims to slow the progression of symptoms by targeting the genetic cause of the disease.
Boston, MassachusettsAges 25–65 - NCT06844214RecruitingPhase 1/Phase 2
Gene therapy trial for myotonic dystrophy type 1
This trial tests a one-time gene therapy (SAR446268) for people with non-congenital myotonic dystrophy type 1. It aims to see if the treatment is safe and can improve muscle symptoms like stiffness and weakness.
Gainesville, FloridaAges 10–55 - NCT06914479RecruitingPhase 1
Gene therapy and valacyclovir for recurrent brain tumors in young people
This trial tests a new gene therapy that uses two special viruses and an oral antiviral drug (valacyclovir) to treat brain tumors that have come back in children and young adults. The therapy is given right after surgery to remove the tumor, with the goal of helping your immune system fight the cancer.
Ann Arbor, MichiganAges 3–39 - NCT06921317RecruitingPhase 1/Phase 2
Gene therapy for advanced glaucoma
This trial tests a gene therapy treatment called GVB-2001 for people with primary open angle glaucoma. It involves a one-time injection into the eye to help lower eye pressure, and could be an option if you have tried other treatments that haven't worked well enough.
Suzhou, JiangsuAges 18–75 - NCT06922890RecruitingPhase 1/Phase 2
Gene therapy for chronic spinal cord injury (AIS-A/B)
This trial tests a new gene therapy called STUP-001 that aims to repair spinal cord damage directly. It is for people who have had a traumatic spinal cord injury with complete or near-complete paralysis (AIS-A or B) for at least 6 months.
SeoulAges 19–60 - NCT06959771RecruitingPhase 1/Phase 2
Gene therapy for CD40L deficiency (Hyper-IgM syndrome) - single patient study
This trial tests a new gene therapy that uses a person's own blood stem cells and T cells, which are corrected with a gene-editing tool called base editing. It is designed for people with a specific CD40L gene mutation who also have liver problems and portal hypertension. The goal is to see if this treatment can improve their immune system and liver health.
Bethesda, MarylandAges 37–120 - NCT06971094RecruitingPhase 3
Gene therapy for type 2 spinal muscular atrophy
This trial tests a new gene therapy called GC101 given as a one-time injection into the spinal fluid for people with type 2 spinal muscular atrophy (SMA). It may help improve motor function.
BeijingAges 2–12 - NCT06996756RecruitingPhase 1
Gene therapy for alpha-1 antitrypsin deficiency
This trial tests a gene therapy to fix the genetic cause of alpha-1 antitrypsin deficiency, aiming to stop or slow emphysema. It is for people with the most common harmful gene variants who have mild to moderate lung damage and are otherwise healthy enough to take steroids safely.
New York, New YorkAges 18–70 - NCT07004361Recruiting
Ethics of gene therapy for wet AMD
This study explores the ethical questions around using gene therapy for wet age-related macular degeneration (AMD). It is not a treatment trial, but a discussion study for patients whose doctors might consider gene therapy as an option.
Nantes, NantesAges 18 years+ - NCT07046338Recruiting
Gene therapy for MLD using the patient's own stem cells
This trial tests a new gene therapy that uses a patient's own blood stem cells to treat MLD (metachromatic leukodystrophy), a rare genetic disorder that damages the nervous system. The goal is to see if this treatment can stop or slow the disease.
Shenzhen, GuangdongAges 1 month–50 years - NCT07063030RecruitingEarly Phase 1
Gene therapy trial for AIPL1-related eye disease
This trial tests a gene therapy called LX107 for people with vision loss caused by changes in the AIPL1 gene. It aims to see if the treatment is safe and can improve vision.
ShanghaiAges 4 years+ - NCT07137338RecruitingPhase 1
Gene therapy for BAG3-related dilated cardiomyopathy
This trial tests a one-time gene therapy called RP-A701 for people with dilated cardiomyopathy caused by a BAG3 gene mutation. The goal is to see if it is safe and can improve heart function.
San Diego, CaliforniaAges 18–65 - NCT07148089RecruitingPhase 1
Gene therapy study for CPVT heart condition
This trial tests a new gene therapy called SGT-501 for people with a genetic heart condition called CPVT, which can cause dangerous fast heartbeats. The goal is to see if the treatment can reduce these episodes.
Boston, MassachusettsAges 7 years+ - NCT07180355RecruitingPhase 1
Gene therapy trial for Friedreich's ataxia
This trial tests a gene therapy called SGT-212 for people with Friedreich's ataxia. It aims to help treat the condition by delivering a working copy of the gene.
Los Angeles, CaliforniaAges 18–40
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Common questions
- Are there clinical trials for gene therapy?
- Yes. Clin2 currently lists 207 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.