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 3 and Phase 4 studies, each written for real people, not researchers.
Recruiting studies in California
- NCT04049084Enrolling by invitation
Follow-up study for people treated with gene therapy for ADA-SCID
This is a follow-up (no new treatment) study for people who previously received a gene therapy made from their own cells for ADA-SCID. It mainly tracks long-term safety and whether a marker showing the gene change can still be found.
Los Angeles, CaliforniaAges Any age - 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.
San Francisco, CaliforniaAges 18 years+ - NCT07282847RecruitingPhase 1/Phase 2
Gene therapy for adults with Pompe disease
This study tests a one-time gene therapy (AB-1009) for adults with late-onset Pompe disease. It aims to see if the treatment is safe and improves muscle and breathing function.
Irvine, CaliforniaAges 18–65 - NCT05821959RecruitingPhase 1/Phase 2
Gene therapy for hearing loss caused by otoferlin gene changes
This early-stage study tests a one-time gene therapy to help patients with profound hearing loss linked to otoferlin gene mutations. It may help hearing by delivering a working version of the otoferlin gene to the inner ear, followed by long-term monitoring.
San Francisco, CaliforniaAges Any age - 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.
Los Angeles, CaliforniaAges birth–5 years - NCT05788536RecruitingPhase 1/Phase 2
Gene therapy study for OTOF-related hearing loss
This trial tests a one-time gene therapy delivered into the ear to improve hearing in people with certain OTOF gene changes. It studies safety and how well the treatment can work, especially in children and infants, as doctors monitor hearing tests for up to 48 weeks.
Los Angeles, CaliforniaAges Any age - NCT06581146Recruiting
Liver health study for boys with XLMTM
This study looks at how the liver works in boys with a serious muscle condition called XLMTM. It checks whether their liver is healthy, which may help doctors better understand the condition and guide future treatments.
Santa Monica, CaliforniaAges Up to 17 years - NCT06003387RecruitingPhase 3
Gene therapy for hemophilia B with AAV5 antibodies
This trial tests a gene therapy called CSL222 for adults with hemophilia B who have antibodies against the AAV5 virus used to deliver the therapy. It aims to see if the treatment is safe and effective even when these antibodies are present.
San Diego, CaliforniaAges 18 years+ - NCT06533579RecruitingPhase 1/Phase 2
Gene therapy for CD19-positive blood cancers
This trial tests a new gene therapy treatment for people with certain types of leukemia or lymphoma that have the CD19 marker and have not responded to other treatments. The therapy uses a virus to deliver a gene to target and kill cancer cells.
Duarte, CaliforniaAges 13–90 - NCT05432310RecruitingPhase 1/Phase 2
Gene therapy for ADA-SCID using blood and a special treatment shot
This Phase 1/2 trial studies a one-time gene therapy approach for children and adults with ADA-SCID, a rare immune deficiency caused by low ADA enzyme activity. The goal is to use the person’s own cells to deliver a corrected gene and help the immune system work better.
Los Angeles, CaliforniaAges 1 month+ - 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+ - 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.
Stanford, CaliforniaAges 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.
La Jolla, CaliforniaAges 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+ - 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+ - 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 - 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 - NCT06063850RecruitingPhase 1/Phase 2
Gene therapy for adults with hard-to-control epilepsy
This trial tests a one-time gene therapy (AMT-260) for adults whose seizures start in one area of the brain (the temporal lobe) and aren't controlled by medication. The goal is to see if it's safe and reduces seizure frequency.
Palo Alto, CaliforniaAges 18–75 - NCT06092034RecruitingPhase 2
Gene therapy for Danon disease in males
This trial tests a gene therapy called RP-A501 for men with Danon disease, a heart condition. It aims to replace the faulty gene that causes the disease to improve heart function.
La Jolla, CaliforniaAges 8 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.
Los Angeles, CaliforniaAges birth–17 years - NCT06332807RecruitingPhase 1/Phase 2
Gene therapy trial for classic PKU
This trial tests a one-time gene therapy called NGGT002 for adults with classic PKU who cannot take or do not respond well to current treatments. The goal is to see if it can safely lower blood phenylalanine levels.
Orange, CaliforniaAges 18–55 - NCT06731933RecruitingPhase 2
Gene therapy for skin cancer in recessive dystrophic epidermolysis bullosa
This trial tests a gene therapy called BVEC to see if it can prevent squamous cell carcinoma (a type of skin cancer) from coming back in people with a severe form of epidermolysis bullosa (fragile skin). It is for adults who have had a confirmed SCC and are willing to use the gene therapy.
Redwood City, CaliforniaAges 18 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.
Davis, CaliforniaAges 2–4
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.
- Stanford University · 5 studies
- University of California, Los Angeles (UCLA) · 4 studies
- University of California, San Francisco · 4 studies
- City of Hope Comprehensive Cancer Center · 2 studies
- UCSF Benioff Children's Hospital Oakland · 2 studies
- Valkyrie Clinical Trials · 2 studies
- Center for Inherited Blood Disorders
- Children's Hospital Los Angeles
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.