Clinical trials · Massachusetts
Gene Therapy clinical trials in Massachusetts
We’re tracking 19 recruiting gene therapy studies with a site in Massachusetts — 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 Massachusetts
- NCT06662188RecruitingPhase 1/Phase 2
Gene therapy for children with SHANK3-related condition
This trial tests a gene therapy called JAG201 for children with a SHANK3 mutation or 22q13.3 deletion, which causes Phelan-McDermid syndrome. The goal is to see if it can help with development and daily function.
Boston, MassachusettsAges 2–9 - 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.
Boston, MassachusettsAges 18 years+ - NCT06696456Enrolling by invitation
Long-term follow-up of gene therapy for otoferlin hearing loss
This study follows up with people who received a gene therapy (AAVAnc80-hOTOF) in a previous clinical trial. It checks how well the treatment works over time and how safe it is.
Boston, MassachusettsAges Any age - NCT05821959RecruitingPhase 3
Gene therapy for otoferlin-related hearing loss
This trial tests a gene therapy that aims to restore hearing in people with a specific genetic form of hearing loss. The therapy is given as a one-time injection into the inner ear and may help you hear without needing a cochlear implant.
Boston, MassachusettsAges 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.
Boston, MassachusettsAges 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.
Boston, MassachusettsAges Any age - 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.
Worcester, MassachusettsAges 18–79 - NCT07148089RecruitingPhase 1
Gene therapy study for CPVT heart condition
This trial tests a new gene therapy called SGT-501 for people with a genetic heart condition called CPVT, which can cause dangerous fast heartbeats. The goal is to see if the treatment can reduce these episodes.
Boston, MassachusettsAges 7 years+ - NCT07052929RecruitingPhase 1/Phase 2
Gene therapy study for young boys with XLMTM on ventilators
This study tests a new gene therapy called ASP2957 in young boys with X-linked myotubular myopathy who are dependent on a ventilator. The goal is to see if it can help improve their breathing and muscle strength.
Boston, MassachusettsAges Up to 3 years - NCT06826612RecruitingPhase 1/Phase 2
Gene therapy trial for early Huntington's disease
This trial tests a gene therapy called SPK-10001 for people with early-stage Huntington's disease. It aims to slow the progression of symptoms by targeting the genetic cause of the disease.
Boston, MassachusettsAges 25–65 - 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.
Boston, MassachusettsAges Up to 17 years - 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.
Boston, MassachusettsAges 18 years+ - NCT06092034RecruitingPhase 2
Gene therapy for Danon disease in males
This trial tests a gene therapy called RP-A501 for males with Danon disease, a genetic condition that affects the heart. The treatment aims to fix the faulty gene to help improve heart function and slow disease progression.
Boston, MassachusettsAges 8 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.
Boston, MassachusettsAges 18–75 - NCT05878860RecruitingPhase 3
Gene therapy for X-linked retinoschisis
This trial tests a gene therapy called ATSN-201 for people with X-linked retinoschisis (XLRS), a condition that affects the retina and can cause vision loss. The therapy aims to fix the faulty gene that causes the condition, potentially halting vision loss and improving vision. Participants receive a one-time injection into the eye, and the study monitors safety and effectiveness.
Boston, MassachusettsAges 5 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.
Boston, MassachusettsAges Up to 2.5 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.
Boston, MassachusettsAges Any age - NCT07215416RecruitingPhase 1/Phase 2
Gene therapy for ataxia-telangiectasia with specific ATM mutation
This trial tests a new genetic medicine designed for people with A-T who have a specific ATM gene change. It aims to see if the treatment is safe and if it can improve symptoms.
Boston, MassachusettsAges birth–17 years - NCT07341867RecruitingPhase 1
Dose changes for cancer treatment in people with Duffy null
This trial tests if changing the dose of certain chemotherapy drugs can help people with a genetic trait called Duffy null avoid side effects like low white blood cell counts. It is for people with multiple myeloma or triple-negative breast cancer starting specific treatments.
Boston, MassachusettsAges 18 years+
Gene Therapy trials by city in Massachusetts
Where these studies are running in Massachusetts
Institutions with a site for the recruiting gene therapy studies listed above.
- Boston Children's Hospital · 10 studies
- Dana-Farber Cancer Institute · 2 studies
- Massachusetts General Hospital · 2 studies
- Beth Israel Deaconess Medical Center
- Boston Children's Hospital - Main
- Local Institution - 01106
- Local Institution - 01107
- Massachusetts Eye and Ear
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 Massachusetts?
- Yes. We're currently tracking 19 recruiting gene therapy studies with a site in Massachusetts, 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 Massachusetts?
- 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 Massachusetts to take part?
- Not necessarily. These studies have a site in Massachusetts, 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 Massachusetts 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.