Clinical trials
Gene Therapy clinical trials
Below are recruiting gene therapy clinical trials, each written for real people, not researchers. We’re tracking 210 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- 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 1/Phase 2
Gene therapy for hearing loss caused by otoferlin gene changes
This early-stage study tests a one-time gene therapy to help patients with profound hearing loss linked to otoferlin gene mutations. It may help hearing by delivering a working version of the otoferlin gene to the inner ear, followed by long-term monitoring.
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 - 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+ - 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+ - 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
Testing gene therapy for Friedreich’s ataxia heart disease
This Phase 1 trial tests an AAV gene therapy that aims to improve heart problems caused by Friedreich’s ataxia. You may be eligible if you have genetically confirmed Friedreich’s ataxia with measurable heart weakening but still enough heart function to safely receive treatment.
New York, New YorkAges 12–50 - NCT07160634RecruitingPhase 3
Gene therapy for ambulant males with Duchenne muscular dystrophy
This trial tests a new gene therapy called SGT-003 in boys and men with Duchenne muscular dystrophy who are still able to walk. The treatment aims to deliver a working version of the dystrophin gene to help muscle cells produce the protein they are missing.
Little Rock, ArkansasAges 7–11 - NCT06064890RecruitingPhase 1/Phase 2
Gene therapy trial for genetic frontotemporal dementia
This trial tests a new gene therapy called AVB-101 for people with a specific genetic cause of frontotemporal dementia (FTD). The therapy is given as a one-time injection into the brain to potentially slow or stop the disease.
Columbus, OhioAges 30–75 - NCT06581146Recruiting
Liver health study for boys with XLMTM
This study looks at how the liver works in boys with a serious muscle condition called XLMTM. It checks whether their liver is healthy, which may help doctors better understand the condition and guide future treatments.
Santa Monica, CaliforniaAges Up to 17 years - NCT06003387RecruitingPhase 3
Gene therapy for hemophilia B with AAV5 antibodies
This trial tests a gene therapy called CSL222 for adults with hemophilia B who have antibodies against the AAV5 virus used to deliver the therapy. It aims to see if the treatment is safe and effective even when these antibodies are present.
San Diego, CaliforniaAges 18 years+ - 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 - NCT07317934RecruitingPhase 3
Gene therapy for wet age-related macular degeneration
This study tests a one-time gene therapy called LX102 for wet age-related macular degeneration (AMD). It may reduce the need for frequent eye injections and help preserve vision.
Hefei, AnhuiAges 50–80 - NCT06533579RecruitingPhase 1/Phase 2
Gene therapy for CD19-positive blood cancers
This trial tests a new gene therapy treatment for people with certain types of leukemia or lymphoma that have the CD19 marker and have not responded to other treatments. The therapy uses a virus to deliver a gene to target and kill cancer cells.
Duarte, CaliforniaAges 13–90 - 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 - NCT05432310RecruitingPhase 1/Phase 2
Gene therapy for ADA-SCID using blood and a special treatment shot
This Phase 1/2 trial studies a one-time gene therapy approach for children and adults with ADA-SCID, a rare immune deficiency caused by low ADA enzyme activity. The goal is to use the person’s own cells to deliver a corrected gene and help the immune system work better.
Los Angeles, CaliforniaAges 1 month+ - 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+ - 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+ - NCT03645460Recruiting
Gene therapy for ADA-SCID using a modified virus vector
This trial tests a gene therapy made to help people with ADA-SCID (an inherited immune condition caused by a faulty ADA gene). The goal is to improve immune function and reduce severe infections by delivering a working ADA gene into the patient’s own cells.
Shenzhen, GuangdongAges 1 month+ - 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+ - 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
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Common questions
- Are there clinical trials for gene therapy?
- Yes. Clin2 currently lists 210 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.