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 us in the clinic or at your local lab.
Treatment is provided with a single outpatient infusion, completed in one day at our Infusion Center. Follow-up care is also continued at our clinic.
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?
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How long does the treatment take?
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Are there any risks or side effects?
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Will gene therapy replace my current treatment?
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What kind of follow-up care is needed?
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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.