• Rare Disease News
  • contribute@patientworthy.com
Facebook-f
Instagram
Linkedin-in
Pinterest
Twitter
  • About
    • Meet Our Contributors
    • Meet Our Partners
    • Meet Our Team
    • Collaborative Content On Patient Worthy
    • Contact Us
  • Diseases
  • Share Your Story
    • Patient Worthy Content Submission Guidelines
    • Patient Worthy Writing Prompts
    • Submit Your Story
  • Resources
    • Library
    • Memes
    • Events
    • Patient Worthy FAQs
  • Listen
  • Languages
    • Spanish / Español
    • Russian / русский
    • French / Français
  • Log In
Menu
  • About
    • Meet Our Contributors
    • Meet Our Partners
    • Meet Our Team
    • Collaborative Content On Patient Worthy
    • Contact Us
  • Diseases
  • Share Your Story
    • Patient Worthy Content Submission Guidelines
    • Patient Worthy Writing Prompts
    • Submit Your Story
  • Resources
    • Library
    • Memes
    • Events
    • Patient Worthy FAQs
  • Listen
  • Languages
    • Spanish / Español
    • Russian / русский
    • French / Français
  • Log In
  • Join PW

Blog

Home » Sickle Cell Disease » Fast Track Designation Granted to Potential Therapy for Sickle Cell Disease
Home » Sickle Cell Disease » Fast Track Designation Granted to Potential Therapy for Sickle Cell Disease

Fast Track Designation Granted to Potential Therapy for Sickle Cell Disease

Trudy Horsting

  • January 7, 2019
  • Beta Thalassemia, Rare Disease, Sickle Cell Disease

CRISPR Therapeutics and Vertex Pharmaceuticals have been collaborating to develop a new treatment option for Sickle Cell Disease. It’s called CTX001, a type of stem cell therapy.

This drug has just received Fast Track Designation by the FDA, bringing even more hope about its potential as a therapy for this population of patients.

Fast Track Designation

The FDA offers Fast Track Designation to help accelerate the development of new drugs which may help serve patient populations whose needs are currently unmet. There are various benefits that may accompany this designation. Perhaps most importantly, it gives the drug developers increased communication with the FDA, helping to expedite review. It also increases the chances that the drug will receive Priority Review or Accelerated Approval.

CTX001

CTX001 is an ex vivo gene-edited therapy. CRISPR and Vertex believe that this therapy has the potential to help the body produce more fetal hemoglobin, or HbF (a form of oxygen carrying hemoglobin), in the red blood cells. HbF normally is depleted after infancy, but for Sickle Cell Disease, high levels of this form of hemoglobin could significantly improve the symptoms of the disease and ultimately the quality of life of patients.

Back in October of 2018, CTX001’s Investigational New Drug application was accepted by the FDA as a potential new treatment option for Sickle Cell disease.

CRISPR and Vertex are also working on CTX001 as a potential treatment option for β-thalassemia and they are currently enrolling a Phase 1/2 trial for the condition in Europe.

You can read more about CTX001’s development and the implications of its new Fast Track Designation here.


What are your thoughts on this new potential therapy for Sickle Cell Disease? Share your stories, thoughts, and hopes with the Patient Worthy community!

Trudy Horsting

Trudy Horsting

Share this post

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

GET INVOLVED

Make a difference, share your experiences and get paid. Opt-in & join Patient Worthy’s panel for paid opportunities such as Surveys, Market Research, Patient Advisory Panels & much more.

JOIN NOW

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
SIGN UP FOR OUR NEWSLETTER
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
Become a Contributor

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

© 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