Clinical trials · California
Gene Therapy clinical trials in California
We’re tracking 35 recruiting gene therapy studies with a site in California — including Phase 1, Phase 1/Phase 2, Phase 2, Phase 2/Phase 3 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in California
- NCT06876363RecruitingPhase 1/Phase 2
Gene therapy study for X-linked chronic granulomatous disease
This study tests a new gene therapy called EN-374 for people with X-linked chronic granulomatous disease (X-CGD). It aims to fix the genetic cause of the disease to prevent severe infections and inflammation.
Los Angeles, CaliforniaAges 3 months+ - NCT07137338RecruitingPhase 1
Gene therapy for BAG3-related dilated cardiomyopathy
This trial tests a one-time gene therapy called RP-A701 for people with dilated cardiomyopathy caused by a BAG3 gene mutation. The goal is to see if it is safe and can improve heart function.
San Diego, CaliforniaAges 18–65 - 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 - 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+ - 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.
Palo Alto, CaliforniaAges 18–79 - 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.
San Francisco, CaliforniaAges 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.
Oakland, CaliforniaAges 2–53 - NCT06379789RecruitingPhase 1/Phase 2
Gene therapy for severe hemophilia B
This trial tests a new gene therapy (REGV131-LNP1265) that aims to let your body produce its own factor IX, reducing or stopping the need for regular infusions. It is for children and adults with severe hemophilia B who are already using factor IX replacement therapy.
Los Angeles, CaliforniaAges 2 years+ - NCT04626674RecruitingPhase 1
Gene therapy safety study for non-ambulatory Duchenne
This Phase 1 trial studies a one-time gene therapy treatment (SRP-9001) to see if it is safe and whether it can produce the needed gene activity in people with Duchenne muscular dystrophy (DMD) who are not walking. It may help researchers understand whether this approach could be useful for DMD patients, especially in later stages.
Sacramento, CaliforniaAges 2 years+ - 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.
Duarte, CaliforniaAges Any age - NCT05694715RecruitingPhase 1
Test combo treatment for cancers with DNA repair gene mutations
This Phase 1 study tests a combination of medicines (including niraparib and irinotecan) for advanced solid tumors that have specific DNA repair gene changes, such as BRCA1/2 or related defects. It aims to see whether the treatment can shrink tumors and is safe enough to continue studying.
San Francisco, CaliforniaAges 18 years+
Gene Therapy trials by city in California
Where these studies are running in California
Institutions with a site for the recruiting gene therapy studies listed above.
- University of California, San Francisco · 6 studies
- University of California, Los Angeles (UCLA) · 4 studies
- University of California, Davis Medical Center · 2 studies
- Children's Hospital Los Angeles
- City of Hope Comprehensive Cancer Center
- Kaiser Permanente Los Angeles Medical Center
- Local Institution
- Loma Linda University (Neurology)
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 California?
- Yes. We're currently tracking 35 recruiting gene therapy studies with a site in California, 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 California?
- 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 California to take part?
- Not necessarily. These studies have a site in California, 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 California 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.