Clinical trials
Gene Therapy clinical trials
Below are recruiting gene therapy clinical trials, each written for real people, not researchers. We’re tracking 208 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT04912843RecruitingPhase 2/Phase 3
Gene therapy for inherited optic nerve vision loss
This trial tests a gene therapy injection into the eye to improve vision in people with a specific type of inherited optic nerve disease called Leber’s hereditary optic neuropathy (LHON). It is aimed at people who have a particular genetic change and a history of vision loss lasting long enough to measure change, while checking for safety first.
BeijingAges 12–75 - NCT04998396RecruitingPhase 1/Phase 2
Gene therapy trial for Canavan disease in young children
This Phase 1/2 trial tests an AAV9 gene therapy to treat Canavan disease. It aims to improve disease-related findings in children whose genetic test confirms ASPA gene changes.
Oakland, CaliforniaAges Up to 2.5 years - NCT05454774RecruitingEarly Phase 1
Gene therapy for adults with severe hemophilia A
This early-phase study tests an FVIII gene therapy to help your body make more of the missing clotting factor in hemophilia A. It’s aimed at adults with severe low FVIII levels who have already used FVIII replacement products for a long time and have not developed an immune blocker (an inhibitor).
Tianjin, Tianjin MunicipalityAges 18 years+ - NCT05672121RecruitingPhase 1/Phase 2
Gene therapy safety study for neovascular macular degeneration
This trial tests a new gene therapy drug (KH631) to see if it is safe and tolerated in people with neovascular age-related macular degeneration (nAMD). It may help your retina problems, but the early goal is mainly to learn about side effects and how the treatment behaves in the eye.
BeijingAges 50–85 - NCT05686798RecruitingPhase 1
Gene therapy plus radiation for recurring brain tumor
This early-phase study tests a “suicide gene” therapy delivered by a virus along with radiation for adults whose high-grade brain tumor has come back or grown again. The goal is to see if the treatment is safe and can help control the tumor.
Detroit, MichiganAges 18 years+ - NCT05709288RecruitingPhase 1
Gene therapy for teens with hemophilia B
This Phase 1 study tests a one-time gene therapy treatment (BBM-H901) for boys ages 12 to 18 with hemophilia B. It aims to see if the treatment is safe and can help the body make more clotting factor over time.
Tianjin, Tianjin MunicipalityAges 12–18 - NCT05791864RecruitingPhase 1/Phase 2
Gene therapy for vision loss in CLN2, tested for safety
This early-phase trial studies a gene therapy medicine called TTX-381 to see if it is safe and tolerable in people with CLN2 disease that affects the eyes. The study also looks at how well the treatment fits into existing eye enzyme therapy care.
HamburgAges 1–7 - 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 - 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 - NCT05861999RecruitingPhase 4
Risdiplam for young children with SMA after gene therapy plateau
This trial studies whether risdiplam is safe and helpful for children under 2 with spinal muscular atrophy (SMA) whose function has leveled off or declined after gene therapy. It may be an option if you saw benefits from gene therapy but then noticed a stop or setback in key abilities.
Little Rock, ArkansasAges 3 months–2 years - NCT05861986RecruitingPhase 4
Risdiplam for very young children after gene therapy for SMA
This trial studies whether risdiplam, given early after gene therapy, is safe and helps children with spinal muscular atrophy (SMA) keep improving or stay stable. It focuses on children under 2 years old who already received gene therapy and need to meet timing and health requirements.
Little Rock, ArkansasAges 3 months–2 years - 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 - NCT06092034RecruitingPhase 2
Gene therapy for Danon disease in males
This trial tests a gene therapy called RP-A501 for men with Danon disease, a heart condition. It aims to replace the faulty gene that causes the disease to improve heart function.
La Jolla, CaliforniaAges 8 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 (NGGT006) to treat a severe form of inherited high cholesterol that doesn't respond well to standard medications. It aims to lower cholesterol and reduce the risk of heart disease.
Xi'an, ShaanxiAges 12–55 - 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 - NCT06207552RecruitingEarly Phase 1
Gene therapy study for children with Fabry disease
This study tests a new gene therapy for children aged 7 to 17 with Fabry disease. The treatment aims to fix the faulty gene that causes the condition, which may help reduce symptoms and prevent organ damage.
ShanghaiAges 7–18 - 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 - NCT06237790Recruiting
Comparing speech understanding: gene therapy vs. cochlear implant
This study compares how well people with severe hearing loss from birth understand speech after either gene therapy (which fixes a faulty gene) or a cochlear implant (a device that helps you hear). It includes healthy people with normal hearing as a comparison group, and it aims to see which treatment works better for understanding spoken language.
Beijing, Beijing MunicipalityAges 6 months+ - 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 - 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 - NCT06345898RecruitingEarly Phase 1
Gene therapy for X-linked retinoschisis in young males
This trial tests a single gene therapy injection under the retina for boys with X-linked retinoschisis, a condition that causes vision loss. It aims to see if the treatment is safe and can improve vision.
Chengdu, SichuanAges 5–18 - 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 - 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+
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Common questions
- Are there clinical trials for gene therapy?
- Yes. Clin2 currently lists 208 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.