What may be on the horizon for hemophilia treatment?

What may be on the horizon for hemophilia treatment?

Pfizer gene therapies are currently not FDA approved.

Gene therapy research continues to evolve. As more information becomes available, the below may help you better understand gene therapy and shared decision making within an evolving treatment landscape.

The Potential of Gene Therapy

Gene therapy aims to address the cause of a genetic disease. It can potentially help treat certain diseases by introducing a healthy (working or functioning) gene. 

  • In hemophilia A or B, the introduced gene is intended to inform the body to produce the factor VIII or factor IX protein involved in blood clotting
  • Gene therapy could potentially provide a long-term treatment benefit to people living with genetic disease with a single dose, as a one-time treatment option.
  • It may help some people manage their hemophilia without frequent infusions or injections

It’s important to note gene therapy may not be appropriate for all people with hemophilia.

The Science of Gene Therapy

Gene Therapy Transfer or Addition is the transfer or addition of genetic material into the cell. A carrier shell delivers a healthy (working or functioning) gene, which is introduced into the cells of a person to treat a specific medical condition.

  • While gene therapy introduces a healthy gene to a person’s body, it does not replace or edit the mutated gene

Gene Regulation is the process of turning genes on or off.

  • Gene regulation is accomplished by a variety of mechanisms, by targeting RNA, the product of DNA transcription

Gene/Genome Editing’s goal is to remove or correct pieces of DNA within a person’s gene rather than replace the gene in the cell with a healthy gene, as gene transfer or addition would do.

  • Gene editing can be used to correct, introduce, or remove pieces of DNA in different types of cells 

Shared Decision Making: Discussing Gene Therapy With Your Care Team

Shared decision making is a partnership between a person with hemophilia and their health care providers. Choices about treatment options such as gene therapy may be made with the help of shared decision making in order to meet one’s treatment goals.

Being prepared for a discussion with your provider or care team is an important step of the shared decision making process. 

If you’d like to discuss treatment options like gene therapy, you may want to bring a list of questions, such as:

  • Is it a good fit for me?
  • What are the risks and benefits?
  • What is the process like?

If you’re considering a different hemophilia treatment like gene therapy, talk to your care team to find out what options may be a good fit for you.

Information available at HEMHORIZON may help you learn more. Visit HEMHORIZON.com to get started, and sign up for emails to be among the first to know when new information is available from Pfizer.


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April 2024