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
- NCT06141460RecruitingPhase 1/Phase 2
Gene therapy trial for wet age-related macular degeneration
This trial tests a new gene therapy called RRG001 for people with the 'wet' form of age-related macular degeneration. It aims to treat leaky blood vessels in the eye that can cause vision loss, possibly reducing the need for frequent eye injections.
Tianjin, Tianjin MunicipalityAges 50 years+ - NCT07024524Recruiting
Hearing evaluation and rehab system for congenital hearing loss
This study is developing a system to evaluate and improve hearing for people with congenital hearing loss. It compares participants who have hearing loss (and have used hearing aids, cochlear implants, or gene therapy) with healthy controls, to better understand and treat hearing challenges.
ShanghaiAges 1–35 - NCT07102524RecruitingPhase 1/Phase 2
Gene therapy for SLC13A5 citrate transporter disorder
This trial tests a new gene therapy (TSHA-105) given through a spinal tap to treat SLC13A5 citrate transporter disorder, a genetic condition that affects the brain and body. The goal is to see if it can help improve symptoms by delivering a working copy of the gene.
Dallas, TexasAges 2–20 - NCT07362927RecruitingEarly Phase 1
Gene therapy trial for diabetic macular edema
This study tests a new gene therapy called LX111 for diabetic macular edema (DME) that has not improved enough with previous injections. It aims to see if a single treatment can reduce swelling and improve vision.
ShanghaiAges 18 years+ - NCT06492863RecruitingPhase 1/Phase 2
Gene therapy (FT-003) for wet AMD
This trial tests a new gene therapy, FT-003, for people with wet AMD. It aims to see if a single treatment can help stop vision loss and may reduce the need for frequent eye injections.
Tianjin, Tianjin,ChinaAges 50–80 - NCT07192432RecruitingPhase 1/Phase 2
Gene therapy for HER2-positive cancer
This trial tests a new gene therapy for people with HER2-positive cancer. It is for two groups: those with advanced cancer that has worsened after treatment, and those with early-stage cancer at high risk of coming back. The goal is to see if the therapy can safely help shrink tumors or prevent return.
Los Angeles, CaliforniaAges 18 years+ - NCT05874310RecruitingEarly Phase 1
Gene therapy trial for RPGR-related vision loss
This early study tests a gene therapy designed for people whose vision problems are caused by an RPGR genetic change. It may help slow or improve the eye disease by treating the underlying cause.
Beijing, Beijing MunicipalityAges 8–45 - NCT06492876RecruitingPhase 1/Phase 2
Gene therapy trial for diabetic macular edema (DME)
This trial tests a gene therapy for diabetic macular edema (DME), a condition where fluid builds up in the central part of the eye's retina. It aims to improve vision and reduce the need for frequent eye injections.
Tianjin, Tianjin,ChinaAges 18–74 - NCT07293546Enrolling by invitationPhase 1/Phase 2
Gene therapy trial for FOXG1 syndrome
This trial tests a gene therapy called FRF-001 for people with FOXG1 syndrome. It aims to see if the treatment is safe and can help improve symptoms.
Houston, TexasAges 2–20 - NCT07135050RecruitingPhase 1/Phase 2
Gene therapy study for Pitt Hopkins syndrome
This trial tests a new gene therapy called MZ-1866 for people with Pitt Hopkins syndrome. The therapy is given as a single injection into the fluid around the brain to help replace the missing or faulty TCF4 gene.
Oakland, CaliforniaAges 2–25 - 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+ - NCT05878860RecruitingPhase 3
RS1-related retinoschisis gene therapy for vision improvement
This Phase 3 trial tests a gene therapy (ATSN-201) to improve or preserve vision in people with RS1-associated X-linked retinoschisis (XLRS), a genetic eye condition. You may qualify if you have a certain level of vision and specific eye “schisis” changes seen on a scan.
Phoenix, ArizonaAges 6 years+ - NCT07226206RecruitingPhase 1/Phase 2
Gene therapy for adults with severe hemophilia A
This trial tests a new gene therapy called SPK-8011QQ for adults with severe or moderately severe hemophilia A. The goal is to see if a single infusion can help your body produce its own factor VIII, reducing or stopping the need for regular factor VIII injections.
Orange, CaliforniaAges 18 years+ - NCT07721025RecruitingPhase 2
Gene therapy trial for Friedreich ataxia heart disease
This trial tests a one-time gene therapy called LX2006 for heart problems caused by Friedreich ataxia. The goal is to see if it can improve heart function and structure.
Tampa, FloridaAges 6 years+ - NCT03466463Recruiting
Gene therapy for severe Crigler-Najjar liver jaundice
This trial studies a gene therapy for people with severe Crigler-Najjar syndrome, a condition that causes high bilirubin (jaundice) due to a UGT1A1 gene change. It may help reduce the need for constant bilirubin-lowering treatment like phototherapy.
ClamartAges 9 years+ - NCT03538899RecruitingPhase 1/Phase 2
Gene therapy for children with an Artemis SCID immune problem
This early-phase study tests an “autologous” gene therapy, meaning they use the child’s own cells, adjusted with a working gene, to help restore immune function. It is for babies and children with Artemis-deficient SCID, aiming to improve T-cell immunity and reduce the need for lifelong infection risk management.
San Francisco, CaliforniaAges 2 months+ - NCT03596086RecruitingPhase 1/Phase 2
Study drug plus radiation and chemo for recurrent brain cancer
This Phase 1/2 study tests whether combining an HSV-tk “gene switch” treatment, valacyclovir tablets, focused radiation (SBRT), and chemotherapy can help people with recurrent glioblastoma or anaplastic astrocytoma. It may help shrink tumors or slow growth after standard treatments have already been tried.
Houston, TexasAges 18 years+ - NCT03603405RecruitingPhase 1/Phase 2
Test vaccine and radiation with chemotherapy for new brain tumor
This trial studies a gene-based treatment (HSV-tk) given with radiation and chemotherapy for people newly diagnosed with glioblastoma or anaplastic astrocytoma. It may help the new brain tumor respond better to standard treatments, though risks and benefits are still being tested.
Houston, TexasAges 18 years+ - NCT03601286RecruitingPhase 1
Gene therapy for X-linked SCID using a modified virus
This early-stage study tests a one-time lentiviral gene therapy to help children with X-linked severe combined immunodeficiency (SCID-X1) make infection-fighting T cells. It may reduce the need for a matching donor transplant, but it requires long-term follow-up for safety over many years.
London, Greater LondonAges 2 months–5 years - NCT03645486Recruiting
Gene therapy for chronic granulomatous disease (CGD)
This trial tests a lentiviral gene therapy to improve immune function in people with CGD, a condition where the body can’t fight certain infections well. It may help by addressing the underlying genetic problem, but it also requires a procedure to collect immune cells and special “conditioning” medicines beforehand.
Shenzhen, GuangdongAges Any age - NCT03961243RecruitingPhase 1
Gene therapy for hemophilia B using a FIX gene treatment
This trial tests a one-time (or limited) gene therapy meant to help the body make more factor IX (FIX), which can reduce bleeding in people with hemophilia B. You may be able to qualify if you have significant bleeding history, no factor IX “blockers” (inhibitors), and can follow strict safety and pregnancy-prevention rules.
Shenzhen, GuangdongAges 2–65 - NCT03996824Recruiting
Gene therapy study for certain vestibular schwannoma surgeries
This trial tests delivering gene therapy into the inner-ear hearing balance nerve cells during surgery for vestibular schwannoma. It may help by improving how the inner ear functions after tumor removal, but you must meet several specific surgery and safety requirements.
Le Kremlin-Bicêtre, Île-de-France RegionAges 18 years+ - NCT04416178Recruiting
Survey and interviews for families affected by sickle cell disease
This study collects feedback through surveys, interviews, and focus groups from people affected by sickle cell disease. Your input helps the research team improve how genomic and gene-therapy information and needs are understood for patients and caregivers.
Memphis, TennesseeAges 13 years+ - NCT04676048RecruitingPhase 1/Phase 2
Gene therapy study for adults with hemophilia A
This Phase 1/2 study tests a one-time gene therapy (ASC618) to help the body make clotting factor for people with hemophilia A. It may reduce bleeding and the need for factor infusions, but you must meet several safety and eligibility checks.
Little Rock, ArkansasAges 18 years+
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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.