• Join Today!

Become a member and connect with:

  • An Active Online Community
  • Articles and Advice on SCD
  • Help Understanding Clinical Trials
https://www.onescdvoice.com/wp-content/uploads/2019/09/Annual.jpg
happenings

10th Annual Sickle Cell Disease Therapeutics Conference – Virtual

The Annual Sickle Cell Disease Therapeutics Conference is a forum to discuss the latest advancements and future trends for treating patients with sickle cell disease. Conference attendees will hear presentations from healthcare companies, key opinion leaders, patients, policy makers and others.

 

When:
September 15, 2021
9:00 AM EST

Where:
Virtual Conference

 

Sep 15, 2021
https://www.onescdvoice.com/wp-content/uploads/2021/07/CTX001.jpg
clinical trials and research

CTX001 Continues to Show Promise in Severe SCD

A single dose of CTX001, an experimental gene-editing cell therapy, rapidly increases the levels of hemoglobin and prevents vaso-occlusive crises (VOCs) for up to nearly two years in people with severe sickle cell disease (SCD), according to interim data from the Phase 1/2 CLIMB-SCD-121 clinical trial.

CRISPR Therapeutics and Vertex Pharmaceuticals are jointly developing CTX001 for the treatment of hemoglobin-associated diseases, including SCD and transfusion-dependent beta thalassemia (TDT).

These preliminary findings, along with positive interim results from the CLIMB-Thal-111 study (NCT03655678) testing the therapy in TDT patients, were presented at the European Hematology Association 2021 Virtual Congress, held June 9–17.

“We are excited about these results and look forward to additional longer-term data and to moving this investigational medicine forward for a larger population of patients with these two devastating diseases,” Samarth Kulkarni, PhD, CRISPR Therapeutics’ CEO, said in a press release.

 

Jul 8, 2021

To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More information

The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.

Close