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
- 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 - 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+ - NCT06219239Recruiting
Gene therapy for transfusion-dependent beta-thalassemia
This trial tests a new gene therapy for people with beta-thalassemia who need regular blood transfusions. It uses a virus to deliver a working gene into your own stem cells, then those cells are put back into your body to help your body make healthy red blood cells.
Tianjin, Tianjin MunicipalityAges 3–35 - NCT01166009Recruiting
CIBMTR research database for transplant and bone marrow injury patients
This study collects health and treatment information in a large research database. It may help researchers better understand outcomes after blood stem cell transplants or bone marrow injury from treatment like radiation.
Minneapolis, MinnesotaAges Any age - NCT06664541Enrolling by invitation
Gene therapy decisions and quality of life
This study looks at how people with blood diseases and their families make decisions about gene therapy, and how it affects their quality of life. It involves interviews or surveys to understand your experience.
Washington D.C., District of ColumbiaAges 18 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 - 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 - NCT06544798Enrolling by invitationPhase 2
Long-term follow-up of gene therapy for dry mouth
This study follows patients who previously received gene therapy for dry mouth caused by radiation. It aims to track long-term safety and any lasting benefits.
Gilbert, ArizonaAges 18 years+ - 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 - 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+ - 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 - NCT06364774RecruitingPhase 1/Phase 2
Gene therapy for transfusion-dependent beta thalassemia
This gene therapy trial uses a modified virus to deliver a healthy gene into your own stem cells. The goal is to help your body make normal red blood cells so you may need fewer or no transfusions.
Philadelphia, PennsylvaniaAges 18–40 - NCT06332807RecruitingPhase 1/Phase 2
Gene therapy trial for classic PKU
This trial tests a one-time gene therapy called NGGT002 for adults with classic PKU who cannot take or do not respond well to current treatments. The goal is to see if it can safely lower blood phenylalanine levels.
Orange, CaliforniaAges 18–55 - NCT06292650RecruitingEarly Phase 1
Gene therapy ZM-02 for advanced retinitis pigmentosa
This experimental gene therapy (ZM-02) is designed for people with advanced retinitis pigmentosa who have very poor vision (counting fingers or worse). It aims to see if the treatment is safe and can improve vision by replacing a faulty gene in the retina.
Beijing, Beijing MunicipalityAges 18–65 - NCT06237790Recruiting
Gene therapy versus cochlear implants for congenital hearing loss
This study looks at how well gene therapy or cochlear implants help restore hearing in people with severe hearing loss from birth. It compares the outcomes of these two treatments to see which is more effective.
Beijing, Beijing MunicipalityAges 6 months+ - NCT06138639RecruitingPhase 1/Phase 2
Gene therapy SGT-003 for Duchenne muscular dystrophy
This trial tests a new gene therapy called SGT-003 for children with Duchenne muscular dystrophy. It aims to help the body produce a working version of dystrophin, the protein missing in Duchenne.
Little Rock, ArkansasAges birth–17 years - NCT06116110Recruiting
Long-term follow-up after gene therapy treatment
This study is a long-term follow-up for people who previously received a gene therapy treatment called zamtocabtagene autoleucel in a Miltenyi Biomedicine-sponsored clinical study. It helps researchers monitor your health over time after the treatment.
Gilbert, ArizonaAges 18 years+ - NCT06111638RecruitingPhase 2/Phase 3
Gene therapy for severe hemophilia A
This trial tests a gene therapy to help your body produce more of the clotting protein (factor VIII) it's missing. It may reduce or stop the need for regular factor infusions.
Beijing, Beijing MunicipalityAges 18 years+ - NCT06125847RecruitingEarly Phase 1
Gene therapy for inherited high cholesterol
This trial tests a one-time gene therapy to lower extremely high cholesterol in people with a rare inherited condition. It may help reduce heart attack risk in those who haven't responded to standard treatments.
Xi'an, ShaanxiAges 12–55 - NCT06092034RecruitingPhase 2
Gene therapy for Danon disease in males
This trial tests a gene therapy called RP-A501 for males with Danon disease, a genetic condition that affects the heart. The treatment aims to fix the faulty gene to help improve heart function and slow disease progression.
La Jolla, CaliforniaAges 8 years+ - 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 - NCT06063850RecruitingPhase 1/Phase 2
Gene therapy for adults with hard-to-control epilepsy
This trial tests a one-time gene therapy (AMT-260) for adults whose seizures start in one area of the brain (the temporal lobe) and aren't controlled by medication. The goal is to see if it's safe and reduces seizure frequency.
Birmingham, AlabamaAges 18–75 - 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+ - NCT05921162Enrolling by invitation
Long-term follow-up after a vision gene eye injection
This study checks how people who previously received a gene therapy eye injection (vMCO-I) are doing over the long term. It may help researchers understand long-lasting effects and safety.
Cuttack, OdishaAges 18 years+
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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.