Clinical trials · Georgia
Gene Therapy clinical trials in Georgia
We’re tracking 10 recruiting gene therapy studies with a site in Georgia — including Phase 1, Phase 1/Phase 2, Phase 2, Phase 2/Phase 3, Phase 3 and Phase 4 studies, each written for real people, not researchers.
Recruiting studies in Georgia
- NCT07075185RecruitingPhase 1
Gene therapy study for hard-to-treat multiple myeloma
This study tests a new gene therapy for people with multiple myeloma that has come back or stopped responding to at least 3 prior treatments. The therapy is designed to help your immune system fight the cancer cells more effectively.
Atlanta, GeorgiaAges 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.
Atlanta, GeorgiaAges birth–5 years - 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.
Atlanta, GeorgiaAges 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.
Atlanta, GeorgiaAges 3 months–2 years - 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.
Atlanta, GeorgiaAges birth–17 years - NCT06817382RecruitingPhase 1
Gene therapy for young boys with Duchenne muscular dystrophy
This study tests a one-time gene therapy given by injection into the spinal fluid (intrathecal) for boys with Duchenne muscular dystrophy. It aims to see if the treatment is safe and how it spreads in the body, and may offer a new way to slow the disease.
Atlanta, GeorgiaAges 2–4 - 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.
Atlanta, GeorgiaAges 18 years+ - NCT06285643RecruitingPhase 2
Gene therapy for moderate Parkinson's disease
This trial tests a gene therapy (AAV2-GDNF) to see if it can slow or improve symptoms of Parkinson's disease. It is for people who have had moderate Parkinson's for 4 to 10 years and still respond to levodopa therapy.
Atlanta, GeorgiaAges 45–75 - NCT04628585Enrolling by invitation
Follow-up for people with sickle cell treated with gene therapy
This study is a long-term follow-up for people who already received gene therapy for sickle cell disease in a specific sponsor’s clinical trial. It helps researchers watch for long-term safety and outcomes over time.
Atlanta, GeorgiaAges 2–53 - NCT03435796RecruitingPhase 2/Phase 3
Follow-up for people treated with gene-modified T cells
This study is a long-term check-in for people who previously received gene-modified T-cell infusions in a related Celgene/Juno/BMS trial. It helps researchers track safety and long-term effects over time.
Atlanta, GeorgiaAges Any age
Where these studies are running in Georgia
Institutions with a site for the recruiting gene therapy studies listed above.
- Children's Healthcare of Atlanta · 4 studies
- Emory University · 4 studies
- Rare Disease Research · 2 studies
- Northside Hospital
What taking part in a gene therapy study involves
A screening visit first
Before anything else, the study team checks whether you fit — usually a visit with some tests. You can stop at any point, and screening is typically free.
Care at a nearby site
Study visits happen at a clinic or hospital taking part. Many studies cover the cost of the study treatment and related visits, and some reimburse travel.
You stay in control
Taking part is voluntary and you can leave a study at any time, for any reason, without affecting your regular care.
The team decides eligibility
Our fit check is a helpful first read, not a decision. The study team makes the final call after reviewing your health history.
Gene Therapy trials in other states
Common questions
- Are there gene therapy clinical trials in Georgia?
- Yes. We're currently tracking 10 recruiting gene therapy studies with a site in Georgia, each rewritten in plain language so you can see what it's testing and who it's for.
- How do I find out if I qualify for a gene therapy study in Georgia?
- Each study lists its eligibility rules — age, diagnosis, prior treatments. On every trial page we explain these in plain language and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Do I have to live in Georgia to take part?
- Not necessarily. These studies have a site in Georgia, but eligibility is about your health, not your address — some people travel to take part, and a few studies reimburse travel. The study team can tell you what's required.
- Does it cost anything to join a gene therapy trial?
- Using Clin2 is always free. Many trials cover the study treatment and related visits; some reimburse travel. The study team explains exactly what's covered before you decide.
Not the right time?
New gene therapy studies open in Georgia regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.
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.