• Rare Disease News
  • [email protected]
Facebook-f Instagram Linkedin-in Pinterest Twitter
  • About
    • Meet Our Contributors
    • Meet Our Partners
    • Meet Our Team
    • Collaborative Content On Patient Worthy
  • Diseases
  • Share Your Story
    • Patient Worthy Content Submission Guidelines
    • Patient Worthy Writing Prompts
    • Submit Your Story
    • Patient Stories
  • Resources
    • Library
    • Memes
    • Events
    • Patient Worthy FAQs
  • Listen
  • Languages
    • Spanish / Español
    • Russian / русский
    • French / Français
  • Contact
Menu
  • About
    • Meet Our Contributors
    • Meet Our Partners
    • Meet Our Team
    • Collaborative Content On Patient Worthy
  • Diseases
  • Share Your Story
    • Patient Worthy Content Submission Guidelines
    • Patient Worthy Writing Prompts
    • Submit Your Story
    • Patient Stories
  • Resources
    • Library
    • Memes
    • Events
    • Patient Worthy FAQs
  • Listen
  • Languages
    • Spanish / Español
    • Russian / русский
    • French / Français
  • Contact
  • Join PW

Blog

Home » Hemophilia A » Shire Looking at New Gene Therapies for Hemophilia A and B
Home » Hemophilia A » Shire Looking at New Gene Therapies for Hemophilia A and B

Shire Looking at New Gene Therapies for Hemophilia A and B

Ronald Ledsen

  • August 18, 2017
  • Hemophilia A, Hemophilia B, Rare Disease

Gene therapy is rapidly shaping up to be the next stage in the evolution of treatment for a wide array of conditions. And it makes sense: rather than treat the symptoms of the disease, gene therapy is all about going straight to the source and correcting the faulty genes themselves.

While the approach is still very much in its infancy, more forward-thinking companies are looking at how they can treat diseases on a genetic level. You can add to that list of innovators Shire Pharmaceuticals, who are looking at how to use gene therapy to treat hemophilia A and hemophilia B.
The venerable global company already has a portfolio of factor-replacement products, but in July they presented findings from some of their recent hemophilia studies at the 26th Biennial International Society on Thrombosis and Haemostasis Congress in Berlin.

The primary goal of these studies was to go beyond just replacing factor: It was to integrate a new gene that would encourage higher levels of Factor VIII and Factor IX production to boost the body’s natural protection against bleeds. The challenge with introducing anything that encourages cell growth is the risk of growing too much of a certain type of cell, which raises the risk for cancer. But in a Factor VIII study, researchers were able to boost the production of factor in mice without also growing the cells that could lead to liver cancer. Likewise, a Factor IX trial in mice boosted factor production in mice by as much as five times without triggering an adverse immune system response.

As is always the case in science, these results are not the end of the story, only the beginning. The results seen in mice need to be duplicated in humans, and more observation is needed to see how it holds up in the long-term. But in terms of changing the way patients are impacted by bleeds on a daily basis, it could be the start of a bold new age of living with hemophilia: An age where the emphasis is on “living.”


Read more about Shire’s research here. What do you think of gene therapy? Is it the way of the future or just a stepping stone to something greater? Sound off in the comments below!
Ronald Ledsen

Ronald Ledsen

After emigrating from his native Sweden, Ronald spent a stint in the Merchant Marines while trying to work out what he wanted to do with his life. He discovered a love of writing while helping a friend write anonymous Harry Potter fan-fiction online; he discovered meaning to his writing when he began journaling after an anxiety disorder diagnosis. Ronald is most relaxed when spending quiet time with his wife, two sons, and hyperactive cat.

Share this post

Share on facebook
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email

OF INTEREST


Picture of the family


Lila’s Journey: Staying Positive While Living With Pediatric Low-Grade Glioma (pLGG)

Patient Stories

STUDY OF THE WEEK

Welcome to Study of the Week. We go in-depth and select a study we think is of particular interest, discussing details, explaining its importance, who may be impacted and lots more!

READ MORE

RARE CLASSROOM

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most mysterious diseases and conditions. This series is an opportunity to learn the basics about some of the diseases that almost no one hears much about.

READ MORE
We believe rare disease patients are people, not a diagnosis. Through education, awareness and some humor, we help patients, caregivers and support persons by providing relevant and often inspirational news and stories.
Our goals are to share stories, cultivate strong community, provide the latest medical findings, connect people and pioneer production of patient worthy information. Help us attain these goals by telling us a little bit about yourself!

Let’s Work Together!

Partner With Us
Submit a Story

Keep Up to Date

Subscribe to Our Newsletter
Check Out Rare Events
Get Inspired By Our Memes

Learn More

About Us
Rare Diseases and Conditions
Terms of Use
Privacy Policy
Privacy Policy for CA Residents
EU Privacy Notice
Privacy Shield Policy

© Copyright Patient Worthy

Sign Up With a Patient Worthy Account and Share Your Rare Story

- OR -

Sign Up For Our Patient Panel

Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.

More Info

We're Happy You're Here!

What best describes you when it comes to rare disease? (check all that apply)

What rare disease(s)/conditions are most important to you?

Visit Home Page or

Thank you for signing up for a Patient Worthy Account!

Have a rare disease story to share? Let us know

Share Story

- OR -

Sign Up For Our Patient Panel

Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.

More Info