Clinical trials · Los Angeles, CA
Gene Therapy clinical trials in Los Angeles
We’re tracking 20 recruiting gene therapy studies with a site in or near Los Angeles — 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 near Los Angeles
- 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 - 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.
Los Angeles, CaliforniaAges 13–90 - 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+ - 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 - 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.
Los Angeles, CaliforniaAges Any age - 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 - 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+ - 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 - 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 - 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.
Los Angeles, CaliforniaAges 6 years+ - 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+ - 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.
Los Angeles, CaliforniaAges 2–4 - 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+ - 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+ - 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 - 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.
Duarte, CaliforniaAges 18 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+ - 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 - 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.
Irvine, CaliforniaAges 45–75 - 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
Hear when a new Gene Therapy trial opens near Los Angeles, CA
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Gene Therapy trials in other cities
Where these studies are running near Los Angeles
Institutions with a site for the recruiting gene therapy studies listed above.
- University of California, Los Angeles (UCLA) · 8 studies
- City of Hope Comprehensive Cancer Center · 3 studies
- Children's Hospital Los Angeles · 2 studies
- Valkyrie Clinical Trials · 2 studies
- Center for Inherited Blood Disorders
- Children's Hospital of Orange County
- Kaiser Permanente Los Angeles Medical Center
- Local Institution
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.
Looking wider? See all recruiting gene therapy studies in California.
Common questions
- Are there gene therapy clinical trials near Los Angeles?
- Yes. We're currently tracking 20 recruiting gene therapy studies with a site near Los Angeles, 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 near Los Angeles?
- 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 Los Angeles to take part?
- Not necessarily. These studies have a site near Los Angeles, 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 near Los Angeles regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.