CRISPR-Cas9 Gene Editing for Beta Thalassemia
What You Need to Know Before You Apply
What is the purpose of this trial?
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. Please consult with the trial team for guidance.
Is CRISPR-Cas9 gene editing safe for treating beta thalassemia?
How is the treatment CTX001 for beta thalassemia different from other treatments?
What data supports the effectiveness of the treatment CTX001 for beta thalassemia?
Research shows that CRISPR/Cas9 gene editing, which is part of the CTX001 treatment, has been successful in correcting mutations in the beta-globin gene, leading to restored normal gene function in cells from beta thalassemia patients. This suggests that CTX001 could be an effective treatment for beta thalassemia by fixing the genetic issues causing the disease.12368
Are You a Good Fit for This Trial?
This trial is for individuals with transfusion-dependent β-thalassemia, which means they need regular blood transfusions due to their condition. They should have a history of significant blood transfusion needs and be suitable for an autologous stem cell transplant. People with certain genetic variations of thalassemia or active infections, low white blood cell or platelet counts can't participate.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single infusion of CTX001 through a central venous catheter
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- CTX001
CTX001 is already approved in European Union, United States for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
Vertex Pharmaceuticals Incorporated
Lead Sponsor
Dr. David Altshuler
Vertex Pharmaceuticals Incorporated
Chief Medical Officer since 2020
MD, PhD
Dr. Reshma Kewalramani
Vertex Pharmaceuticals Incorporated
Chief Executive Officer since 2020
MD, trained in internal medicine and nephrology
CRISPR Therapeutics
Industry Sponsor