Clinical trials · California
Gene Therapy clinical trials in California
We’re tracking 32 recruiting gene therapy studies with a site in California — including Phase 1, Phase 1/Phase 2, Phase 2/Phase 3 and Phase 4 studies, each written for real people, not researchers.
Recruiting studies in California
- 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.
Madera, CaliforniaAges 3 months–2 years - 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 - 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 - 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+ - 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 for non-ambulatory Duchenne muscular dystrophy
This study tests a gene therapy that aims to help muscles produce a protein they are missing. It is for people with Duchenne muscular dystrophy who are no longer able to walk, and it may help slow the progression of the disease.
Palo Alto, 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.
- UCSF Benioff Children's Hospital Oakland · 2 studies
- University of California, Davis · 2 studies
- Children's Hospital Los Angeles
- City of Hope
- Local Institution - 01043
- Local Institution - 01206
- Orthopaedic Hemophilia Treatment Center
- Stanford Cancer Center
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 32 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.