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
- NCT07629115Recruiting
Long-term follow-up study for severe Parkinson's gene therapy
This study follows patients who previously received IPS101A, an experimental gene therapy treatment for severe Parkinson's disease. Researchers want to check how you're doing over time and gather information about the treatment's long-term effects.
SeoulAges 50–80 - 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 - NCT06458595RecruitingPhase 1/Phase 2
Gene therapy for wet age-related macular degeneration (nAMD)
This trial tests a gene therapy (KH658) as a one-time treatment for wet macular degeneration. It's for people whose condition has been stabilized with anti-VEGF injections, and aims to see if it is safe and can reduce the need for frequent eye injections.
TianjinAges 50–85 - NCT04817462Recruiting
Liver biopsy in people with haemophilia gene therapy
This study takes a small liver tissue sample (a liver biopsy) in men who previously received haemophilia gene therapy. The goal is to learn more about how the gene therapy works and how safe it is for the liver.
LondonAges 18–80 - NCT07503613Enrolling by invitation
Long-term follow-up for AAVAnc80 gene therapy
This study follows people who already received the experimental AAVAnc80-antiVEGF gene therapy for vestibular schwannoma. It monitors long-term safety and effects of the treatment.
Dallas, TexasAges 18 years+ - NCT06641154RecruitingPhase 1/Phase 2
Gene therapy for severe Crigler-Najjar syndrome
This trial tests a gene therapy for children with severe Crigler-Najjar syndrome type I. It aims to reduce the need for daily phototherapy by fixing the faulty gene.
MoscowAges 3 months–10 years - NCT07426419RecruitingPhase 1/Phase 2
Gene therapy for heart failure caused by BAG3 mutation
This trial tests an experimental gene therapy (AFTX-201) designed to address the genetic cause of heart failure in people with a specific BAG3 gene mutation. It aims to improve heart function and reduce symptoms.
Miami, FloridaAges 18–70 - 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 - NCT07401121RecruitingPhase 1
Study of CTx1000 for people with ALS
This trial tests a new drug, CTx1000, for people with ALS. It aims to see if the drug is safe and tolerable, and may offer a new treatment option.
Sydney, New South WalesAges 18–80 - 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+ - 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 - 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+ - NCT07364357RecruitingPhase 1
Study of CREPT-618 for advanced liver cancer
This phase 1 trial tests a new drug called CREPT-618 for people with advanced liver cancer that hasn't responded to standard treatments. The drug is designed to target cancer cells with specific markers (CREPT and ASGPR), so only patients whose tumors have these markers can join.
BeijingAges 18–75 - NCT07346144RecruitingPhase 1/Phase 2
Gene therapy study for high-grade glioma
This trial tests a new gene therapy given directly into the brain tumor. It's for people with high-grade glioma, either newly diagnosed or after one recurrence, to see if it can help control the tumor.
Columbus, OhioAges 18–70 - 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 - 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.
Oakland, CaliforniaAges 2–20 - NCT07290062RecruitingPhase 1
Gene therapy injection for ALS safety study
This trial tests a single injection of a gene therapy called INS1202 into the spinal fluid. It is for people with ALS, including those with a specific genetic form (SOD1 mutation). The goal is to see if it is safe and how it affects the body.
La Jolla, CaliforniaAges 18–79 - NCT07264166Enrolling by invitation
Gene therapy trial for cerebral palsy and HIE
This trial tests a gene therapy given as a nose spray for people with cerebral palsy or HIE. It aims to see if the treatment is safe and helps improve movement over two years.
Monterrey, Nuevo LeónAges 2–65 - 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+ - NCT07223944RecruitingPhase 3
Gene therapy trial for Gaucher disease type 1
This trial tests a gene therapy called FLT201 for adults with Gaucher disease type 1. It aims to provide a long-term treatment option for people who have been stable on enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least two years.
Los Angeles, CaliforniaAges 18 years+ - 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+ - NCT07180355RecruitingPhase 1
Gene therapy trial for Friedreich's ataxia
This trial tests a gene therapy called SGT-212 for people with Friedreich's ataxia. It aims to help treat the condition by delivering a working copy of the gene.
Los Angeles, CaliforniaAges 18–40 - NCT07159581Enrolling by invitation
Gene therapy for Wilson disease study with PET scans
This trial uses a special PET scan with a copper tracer to see how well gene therapy is working in people with Wilson disease. It may help doctors understand if the treatment is helping your body handle copper properly.
Aarhus, Aarhus NAges 18 years+ - 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
Hear when a new Gene Therapy trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.
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.