Gene Therapy
A New Option for Adults with Hemophilia
Gene therapy offers a one-time treatment option for adults with severe hemophilia A or moderate/severe hemophilia B. This innovative therapy is provided as an outpatient service at our infusion center.
Gene Therapy works by delivering a functional gene to your liver, where it begins its mission to help your body produce clotting factors naturally.
Once the therapy reaches your liver, the functional gene provides instructions for your cells to produce higher levels of factor VIII or IX – the proteins your body needs to improve blood clotting. This means your body could start making these factors on its own.
Comprehensive Care
Our gene therapy team is dedicated to providing education, answering questions, helping navigate insurance, and delivering support from start to finish. Visits are completed at CIBD or via telehealth, and assessments are arranged with assistance from our team at CIBD or your local lab.
Treatment is provided with a single outpatient infusion, completed in one day at our Infusion Center. Follow-up care continues at CIBD after treatment.
Hemgenix® Administration Site
Our team is trained in the preparation and administration of Hemgenix, and CIBD is a designated Hemgenix Administration Site. We have also completed Hemgenix treatments for patients at our Infusion Center.
The Gene Therapy Journey
Interest in gene therapy
Antibody testing
Education visit
Labs & imaging
Insurance authorization
Infusion day
Ongoing follow up & outcome review
If you are interested in learning more about gene therapy, please fill out our contact form and select the ‘Gene Therapy’ option from the dropdown menu.
Frequently Asked Questions
Is gene therapy a cure?
Gene therapy is not considered a cure, but some gene therapies can have significant, potentially transformative effects for people living with certain rare diseases. Depending on the treatment and condition, gene therapy may help address the underlying cause of a disease and change how it affects the body.
Is gene therapy a new field?
No. Scientists began developing early concepts for gene therapy in the 1960s. Since then, researchers around the world have continued studying and advancing gene therapy and our understanding of how it can be used to treat disease.
Who is eligible for gene therapy?
Eligibility depends on several factors and we carefully evaluate each individual to determine whether gene therapy may be a treatment option. This workup includes type and severity of hemophilia, age, liver health, medical history, and specialized laboratory testing. Current FDA-approved gene therapies have been studied primarily in adults with hemophilia, and current FDA approved gene therapy is available to individuals over the age of 18 and with moderate or severe hemophilia B.
If commercially available gene therapy is not appropriate for you, CIBD may be able to discuss research studies or future treatment opportunities that could be available.
How long does the treatment take?
Gene therapy is given as a one-time intravenous (IV) infusion. The infusion itself typically takes a few hours and is performed on-site at CIBD’s Infusion Center. Patients should plan to spend most of the day at CIBD on infusion day because we monitor during and after the infusion to ensure they are doing well.
It’s important to note that though the treatment is delivered in a single visit, the process includes multiple evaluations before treatment and ongoing follow-up afterward.
Are there any risks or side effects?
Like all medical treatments, gene therapy has potential risks and side effects. The most common side effect is an increase in liver enzymes, which may indicate that the body’s immune system is reacting to the treatment. Some people may need medications, such as steroids, to help manage this response. Other possible side effects include headache, fatigue, nausea, flu-like symptoms, or infusion-related reactions.
At CIBD, we closely monitor patients before, during, and after treatment and discuss the potential benefits and risks to help individuals and families make informed decisions. Because gene therapy is still a relatively new treatment approach, researchers continue to study its long-term effects.
Will gene therapy replace my current treatment?
Gene therapy may reduce or eliminate the need for regular factor replacement therapy for some people, but it is not considered a guaranteed cure. Results vary from person to person. Some individuals experience significant increases in clotting factor levels and fewer bleeding episodes, while others may continue to need factor concentrate or other hemophilia treatments over time. Factor levels may also change in the years following treatment.
Our team will work closely with you to monitor your response to treatment and determine whether any changes to your current treatment plan are appropriate.
What kind of follow-up care is needed?
Follow-up care is an important part of the gene therapy journey. After treatment, CIBD will closely monitor your liver health, clotting factor levels, and overall response through regular telehealth and in-clinic visits and blood tests. Monitoring is most frequent during the first few months after treatment and then becomes less frequent over time. Long-term follow-up continues for several years to help ensure safety and assess how well the therapy is working.
Our team is committed to supporting patients and families throughout the process and will continue to provide guidance, education, and comprehensive hemophilia care before and after gene therapy.
We understand that deciding whether gene therapy is right for you or your family is an important and personal decision. Our team is available to answer questions, review treatment options, and help you understand both currently available therapies and emerging research opportunities so you can make an informed choice about your care.
Who do I contact if I'm interested in learning more?
If you are interested in learning more about gene therapy, please fill out our contact form and select the ‘Gene Therapy’ option from the dropdown menu.
In the News
Center for Inherited Blood Disorders Administers Country's First Gene Therapy Infusion to Treat Hemophilia A
The Center for Inherited Blood Disorders (CIBD) administered the country’s first successful gene therapy infusion to treat Hemophilia A outside of the clinical trial setting. The landmark infusion, administered by CIBD’s skilled medical team, is a testament to the center’s commitment to advancing cutting-edge treatments for inherited blood disorders.