Consider Gene Therapy as a Potential Hemophilia Approach

Considering Gene Therapy as a Hemophilia Treatment

You’ve already reviewed the science of gene therapy and now wonder if gene therapy may be a potential option for managing your hemophilia. The decision to consider gene therapy (transfer or addition) as a hemophilia treatment option is a very personal choice that you will make with your doctor. It may not be for everyone. There will be several important criteria to take into account before determining whether gene therapy may be an appropriate option for you. As always, talk to your doctor about any questions you may have about gene therapy, including safety or other considerations.

Am I a good candidate for hemophilia gene therapy?

Some of the factors below may help determine whether someone may be a candidate for hemophilia gene therapy. Sign up through the HemHorizon form to find out more.

Hemophilia
Severity

Gene therapy for hemophilia may be limited to adult patients with moderately severe to severe hemophilia.

Inhibitors

Gene therapy may not be an appropriate option for people with a history of inhibitors to factor VIII or IX.

Coexisting
conditions

An individual’s overall health, including conditions other than hemophilia, may be considered before receiving gene therapy. For example, hemophilia gene therapy aims to target the liver and may not be an appropriate option for people with other conditions that impact liver health.

Antibodies to rAAV
Vector

Before administering gene therapy, healthcare providers may check to see if a person has neutralizing antibodies (NAbs) against a particular vector. In some cases, patients with pre-existing NAbs may be excluded from certain gene therapies.

Age

Currently, hemophilia gene therapy research has been conducted in adults only (18 years of age or older).

Hemophilia
Severity

Gene therapy for hemophilia may be limited to adult patients with moderately severe to severe hemophilia.

Inhibitors

Gene therapy may not be an appropriate option for people with a history of inhibitors to factor VIII or IX.

Coexisting
conditions

Antibodies to rAAV
Vector

Age

Currently, hemophilia gene therapy research has been conducted in adults only (18 years of age or older).

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Is gene therapy safe?

Many people have questions or concerns about the safety of gene therapy, and that’s perfectly normal. Clinical trials are done to ensure that gene therapy, or any other treatment, is safe and effective.

The FDA rigorously reviews results from clinical trials before approving a potential therapy. The FDA also often requires long-term monitoring for new treatments like gene therapy, which is intended to evaluate safety data over time.

Hemophilia Gene Therapy Safety Considerations

If gene therapy is something you may be considering, it’s important to understand some potential safety considerations:

Immune response

Even if a person does not have NAbs, the immune system might still recognize the vector used in gene therapy as a foreign body. Some people may develop an immune response after receiving gene therapy, such as inflammation. In such instances, corticosteroids may be used to help suppress the immune response. The frequency, severity, and treatment plan for any adverse event often depends on the specific gene therapy, disease, and individual patient profile. Your doctor will discuss the possible risks and side effects with you before you consider gene therapy.

Neutralizing Antibodies (NAbs)

Exposure to the AAV vector through gene therapy may cause the body to produce NAbs to the AAV vector, which may impact future dosing.

Increased factor levels

The goal of gene therapy for hemophilia is to help increase blood clotting factor levels. If clotting factor levels become too high, however, there may be a risk of blood clots, also known as thrombosis. It is important to have clotting factor levels monitored after gene therapy.

Inhibitors

To date, no participants in gene therapy (transfer or addition) research have developed inhibitors, but researchers continue to monitor for this possibility.

Viral shedding

Viral shedding is how the body releases the vector over time, once it is no longer needed. This can be done through the patient's blood, urine, semen, feces and/or saliva. No events of transmission have been observed in research to date.

Vector integration

Integration means that a part of the healthy or functioning gene is inserted into the patient’s own DNA after receiving gene therapy. So far in human and animal research, vector integration occurs <1% of the time. Research is ongoing to understand any potential impact of vector integration.

Please speak with your doctor about any questions or concerns you may have about the safety considerations associated with gene therapy.

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How long does gene therapy last?

Clinical trials are helping us learn more about gene therapy, across multiple therapeutic areas, including potential length of effect. So far, research suggests that gene therapy may last up to several years in some patients.

The length of time may vary depending on the type of genetic disease, the specific gene therapy, and the specific patient characteristics, and gene therapy may not work for all patients.

Discussing gene therapy as a hemophilia treatment

The potential of gene therapy as a treatment option for people with hemophilia is exciting, but it may not be appropriate for some people. Because hemophilia gene therapy is a one-time treatment, you and your doctor will need to determine which hemophilia gene therapy, if any, is right for you.

Evolving hemophilia treatment

As the science of gene therapy in hemophilia continues to evolve, so do people’s questions.
Learn about hemophilia through the answers to frequently asked questions.

This site will be updated to include new information over time.

As the science of gene therapy in hemophilia continues to evolve, answers to frequently asked questions can be found here.

This site will be updated to include new
information over time.

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Gene Therapy Considerations