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
- 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+ - 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 - NCT07135050RecruitingPhase 1/Phase 2
Gene therapy study for Pitt Hopkins syndrome
This trial tests a new gene therapy called MZ-1866 for people with Pitt Hopkins syndrome. The therapy is given as a single injection into the fluid around the brain to help replace the missing or faulty TCF4 gene.
Oakland, CaliforniaAges 2–25 - NCT07102524RecruitingPhase 1/Phase 2
Gene therapy for SLC13A5 citrate transporter disorder
This trial tests a new gene therapy (TSHA-105) given through a spinal tap to treat SLC13A5 citrate transporter disorder, a genetic condition that affects the brain and body. The goal is to see if it can help improve symptoms by delivering a working copy of the gene.
Dallas, TexasAges 2–20 - 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+ - NCT07050160Enrolling by invitation
Long-term follow-up after gene therapy for arrhythmogenic cardiomyopathy
This study follows participants who have already received the gene therapy LX2020 for a genetic heart condition called arrhythmogenic cardiomyopathy. It tracks their long-term safety and health outcomes, with no new treatment given.
Ann Arbor, MichiganAges 18–67 - NCT07052929RecruitingPhase 1/Phase 2
Gene therapy study for young boys with XLMTM on ventilators
This study tests a new gene therapy called ASP2957 in young boys with X-linked myotubular myopathy who are dependent on a ventilator. The goal is to see if it can help improve their breathing and muscle strength.
Chicago, IllinoisAges Up to 3 years - NCT07053358RecruitingEarly Phase 1
Gene therapy for wet AMD
This trial tests a new gene therapy (LX111) for people with wet age-related macular degeneration (AMD) who have already been helped by standard anti-VEGF injections. The goal is to see if one dose of LX111 can reduce the need for frequent eye injections.
ShanghaiAges 50 years+ - NCT07023965Enrolling by invitation
Long-term follow-up study for gene therapy in critical limb ischemia
This study follows up with people who previously took part in a clinical trial testing an experimental gene therapy for critical limb ischemia (severe leg pain or sores due to blocked arteries). It aims to see how well the treatment works over time. If you were in that earlier trial and received at least one dose, joining this follow-up could help researchers learn more about the therapy's lasting effects.
Beijing, Beijing MunicipalityAges 20–85 - NCT07024524Recruiting
Hearing evaluation and rehab system for congenital hearing loss
This study is developing a system to evaluate and improve hearing for people with congenital hearing loss. It compares participants who have hearing loss (and have used hearing aids, cochlear implants, or gene therapy) with healthy controls, to better understand and treat hearing challenges.
ShanghaiAges 1–35 - 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+ - NCT06996756RecruitingPhase 1
Gene therapy for alpha-1 antitrypsin deficiency
This trial tests a gene therapy for people with alpha-1 antitrypsin deficiency who have mild to moderate lung disease. The aim is to see if the treatment can improve lung health and is safe.
New York, New YorkAges 18–70 - 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 - 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 - 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 - 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 - 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 - NCT06885424Enrolling by invitation
Long-term follow-up for A2 Bio gene therapy patients
This study follows up with people who previously received a gene therapy product from A2 Biotherapeutics in an earlier trial. The goal is to monitor their health over time and check for any long-term effects.
Gilbert, ArizonaAges 18 years+ - NCT05860569RecruitingPhase 1
Gene therapy safety study for inherited high triglycerides and pancreatitis
This Phase 1 trial tests the safety of a gene therapy for people with inherited, hard-to-control high triglycerides (fat in the blood) who have had pancreatitis episodes. It may help by targeting the genes involved in triglyceride control, but the main goal here is to check safety and tolerability.
Beijing, Beijing MunicipalityAges 18–60 - 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+ - NCT05824169RecruitingPhase 1/Phase 2
Gene therapy for babies with severe spinal muscular atrophy
This early-stage study tests whether a one-time gene therapy can safely improve outcomes for babies with the most severe type of spinal muscular atrophy (SMA type 1). It focuses on safety and early signs of benefit, and participation requires specific genetic and health criteria.
BeijingAges birth–6 months - 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+ - NCT06851767Enrolling by invitationPhase 1/Phase 2
Gene therapy for X-linked severe combined immunodeficiency
This study tests a gene therapy to treat X-linked severe combined immunodeficiency (X-SCID) without a stem cell transplant from a matched sibling. It aims to improve immune function and reduce infections using your own stem cells modified to carry the correct gene.
Bethesda, MarylandAges 3–99 - 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
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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.