Clinical trials
Gene Therapy clinical trials
Below are recruiting gene therapy clinical trials, each written for real people, not researchers. We’re tracking 211 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT05991336Recruiting
Growth and development after gene therapy for beta-thalassemia in children
This study looks at how children with transfusion-dependent beta-thalassemia grow and develop after gene therapy. It compares children who have had gene therapy with those who have not, and also with healthy children of the same age and gender.
Tianjin, Tianjin MunicipalityAges 3–14 - NCT04728841Recruiting
Gene therapy trial for Chinese hemophilia A
This trial tests a one-time gene therapy treatment (GS001) to help people with hemophilia A make more of the clotting factor they lack. If it works and is safe for you, it could improve how well blood clots form over time.
Tianjin, Tianjin MunicipalityAges 18 years+ - NCT07285629RecruitingEarly Phase 1
Klotho and follistatin gene therapy safety study
This trial tests a one-time gene therapy that may help slow or reverse signs of aging. It is for healthy adults aged 50 to 80 who are open to possible visible body changes.
Austin, TexasAges 50–80 - NCT07173153Enrolling by invitationPhase 1/Phase 2
Gene therapy study for SLC6A1 genetic disorder
This trial tests a gene therapy for people with a specific mutation (S295L) in the SLC6A1 gene, which causes a neurodevelopmental disorder. The therapy aims to correct the genetic issue, and participants will be monitored closely for safety and effectiveness.
Columbus, OhioAges Any age - NCT05152823Enrolling by invitationPhase 1/Phase 2
Gene therapy for IGHMBP2-related nerve conditions
This early-phase study tests a gene therapy meant to treat people who have IGHMBP2 gene changes. It aims to improve the way the nervous system works and to see if the treatment is safe, especially in young children.
Columbus, OhioAges 2 months–14 years - NCT07216781RecruitingPhase 1
Klotho gene therapy safety trial
This trial tests a new gene therapy that uses a harmless piece of DNA (called a plasmid) to help your body make more Klotho, a protein linked to healthy aging. The goal is to see if it's safe and whether it might slow aging or improve health.
Austin, TexasAges 23–90 - NCT03000244Recruiting
Long-term follow-up after stem cell or gene therapy
This study checks how people do long-term after stem cell transplant, cellular therapy, or gene therapy. It may help doctors track longer-term health effects and recovery and improve future care.
Bethesda, MarylandAges 4–120 - NCT07443826RecruitingPhase 1/Phase 2
Gene therapy for age-related muscle loss and heart health
This trial tests a gene therapy that may help rebuild muscle and improve blood vessel growth in people ages 35 to 75. It's designed for those with stable health conditions and no history of cancer, heart disease, blood clots, or diabetes.
Coxen Hole, Bay IslandsAges 35–75 - NCT07424157Recruiting
Long-term gene therapy follow-up study
This study follows patients who received gene therapy at Seattle Children's Hospital to check on their health and safety over time. It helps doctors understand how well the treatment works in the long run.
Seattle, WashingtonAges Any age - NCT03725670Recruiting
Gene therapy injection for metachromatic leukodystrophy (MLD)
This trial tests a direct gene-therapy injection into the body to treat MLD, a genetic brain condition. It may help replace a faulty gene so the disease process slows or changes.
Shenzhen, GuangdongAges 1 month–50 years - NCT03727555Recruiting
Lentiviral gene therapy for childhood cerebral X-ALD
This trial studies an experimental gene therapy given through infusion (IV) and into the spinal fluid (injection) for people with X-ALD affecting the brain. It aims to improve or slow down damage in the nervous system, and you must be able to do MRI scans and meet specific health and infection requirements.
Shenzhen, GuangdongAges 1–60 - NCT05810181Recruiting
Interviews to improve choices about gene therapy for rare diseases
This study uses one-on-one interviews to learn what families and clinicians need to make decisions about gene therapy for rare diseases. The goal is to create or improve decision tools that make gene therapy information easier to understand and act on.
Memphis, TennesseeAges 8 years+ - NCT07313618RecruitingEarly Phase 1
Gene therapy for children with oculocutaneous albinism type 1
This trial tests a single injection of JWK010 gene therapy for children aged 5 to 12 with OCA1A albinism. The therapy is given into the eye to try to improve vision, and is meant for those who have not had gene therapy before.
Chengdu, SichuanAges 5–12 - NCT04049084Enrolling by invitation
Follow-up study for people treated with gene therapy for ADA-SCID
This is a follow-up (no new treatment) study for people who previously received a gene therapy made from their own cells for ADA-SCID. It mainly tracks long-term safety and whether a marker showing the gene change can still be found.
Los Angeles, CaliforniaAges Any age - NCT06662188RecruitingPhase 1/Phase 2
Gene therapy for children with SHANK3-related condition
This trial tests a gene therapy called JAG201 for children with a SHANK3 mutation or 22q13.3 deletion, which causes Phelan-McDermid syndrome. The goal is to see if it can help with development and daily function.
Chicago, IllinoisAges 2–9 - NCT07480564RecruitingPhase 3
Gene therapy trial for girls ages 2-4 with Rett syndrome
This trial tests a new gene therapy called TSHA-102 for girls with classic Rett syndrome caused by a specific MECP2 gene mutation. It aims to see if the treatment is safe and can help improve symptoms.
Boston, MassachusettsAges 2–3 - NCT07075185RecruitingPhase 1
Gene therapy study for hard-to-treat multiple myeloma
This study tests a new gene therapy for people with multiple myeloma that has come back or stopped responding to at least 3 prior treatments. The therapy is designed to help your immune system fight the cancer cells more effectively.
Duarte, CaliforniaAges 18 years+ - NCT07282847RecruitingPhase 1/Phase 2
Gene therapy for adults with Pompe disease
This study tests a one-time gene therapy (AB-1009) for adults with late-onset Pompe disease. It aims to see if the treatment is safe and improves muscle and breathing function.
Phoenix, ArizonaAges 18–65 - NCT05472649Enrolling by invitation
Long-term follow-up after gene-modified cell therapy
This study checks your health over time after you previously received gene-modified cell therapy in a Sorrento-sponsored trial. It helps researchers understand long-term safety and outcomes and may lead to better care for future patients.
Philadelphia, PennsylvaniaAges Any age - NCT07270549RecruitingPhase 1/Phase 2
Gene therapy trial for children with CTNNB1 syndrome
This trial tests a one-time gene replacement therapy given into the fluid around the brain to help children with CTNNB1 neurodevelopmental syndrome. The goal is to see if replacing the faulty gene can improve development and reduce symptoms.
LjubljanaAges 2–12 - NCT05529342Enrolling by invitation
Long-term follow-up for a prior gene-cell treatment
This study follows people long-term after receiving a specific gene-cell treatment called AGT103-T. It helps researchers check long-term safety and outcomes, which can guide future care for others.
Washington D.C., District of ColumbiaAges 18 years+ - NCT06492850RecruitingPhase 1/Phase 2
Gene therapy for RPGR-related retinitis pigmentosa
This trial tests a gene therapy for people with a specific genetic form of retinitis pigmentosa (caused by changes in the RPGR gene). It aims to improve vision and slow disease progression.
Beijing, Beijing MunicipalityAges 8–45 - NCT06722170Recruiting
Gene therapy study for hearing loss caused by OTOF gene mutation
This trial tests a gene therapy called EH002 for people with severe or profound hearing loss due to changes in the OTOF gene. The treatment aims to help the ear hear better by replacing the faulty gene.
Zhengzhou, HenanAges 6 months+ - NCT06696456Enrolling by invitation
Long-term follow-up of gene therapy for otoferlin hearing loss
This study follows up with people who received a gene therapy (AAVAnc80-hOTOF) in a previous clinical trial. It checks how well the treatment works over time and how safe it is.
Iowa City, IowaAges Any age
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Gene Therapy trials by city
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Gene Therapy trials by state
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Common questions
- Are there clinical trials for gene therapy?
- Yes. Clin2 currently lists 211 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.