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
- 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+ - 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 - 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 - 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 - 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+ - 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 - 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+ - 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 - NCT06473740RecruitingPhase 4
Gene therapy plus angioplasty for severe leg blockages
This study tests if adding gene therapy to a standard leg artery-opening procedure (angioplasty or stenting) helps people with severe leg pain or non-healing wounds due to blocked arteries. It may improve blood flow and save the leg from amputation.
Blagoveshchensk, Amur OblastAges 18 years+ - NCT05821959RecruitingPhase 3
Gene therapy for otoferlin-related hearing loss
This trial tests a gene therapy that aims to restore hearing in people with a specific genetic form of hearing loss. The therapy is given as a one-time injection into the inner ear and may help you hear without needing a cochlear implant.
San Francisco, CaliforniaAges Any age - NCT05377307Recruiting
Long-term follow-up for gene-edited immune cell therapy
This study checks how people are doing years after receiving a specific company’s gene-edited immune cell treatment. It helps researchers learn about long-term safety and health outcomes.
Kaohsiung, TaiwanAges Any age - NCT07121777Recruiting
CAR T cell therapy for autoimmune diseases
This study tests a new type of cell therapy (CAR T cells) that targets certain immune cells to treat autoimmune diseases like rheumatoid arthritis, lupus, Sjögren's syndrome, and systemic sclerosis. It may help people whose disease is not well controlled with standard treatments.
Hefei, AnhuiAges 18 years+ - NCT07432867RecruitingPhase 1/Phase 2
Gene therapy for severe sickle cell disease in teens and adults
This trial tests a new gene therapy for sickle cell disease. It uses your own blood stem cells (collected from bone marrow) that are modified in a lab to help produce healthy red blood cells, then given back to you after a short chemotherapy. The goal is to reduce or stop severe complications like pain crises and lung problems.
Paris, Île-de-France RegionAges 12–35 - NCT07211048Recruiting
Gene therapy for B-cell lymphoma that hasn't responded to standard treatment
This trial tests a new gene therapy that re-engineers your own immune cells to attack CD19-positive lymphoma. It is for people whose B-cell lymphoma has come back or not responded to at least two standard treatments.
Hefei, AnhuiAges 18 years+
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Gene Therapy trials by city
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Gene Therapy trials by state
Studies with a site anywhere in these states.
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.