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▎Editor of WuXi AppTec content team On June 11, 2021, Vertex Pharmaceuticals and CRISPR Therapeutics jointly announced 22 patients with transfusion-dependent β-thalassemia (TDT) or severe sickle cell anemia (SCD).
The latest clinical trial data of CRISPR/Cas9 gene editing therapy CTX001 treatment
.
The results of the trial showed that all 15 TDT patients were no longer dependent on blood transfusions at the latest follow-up
.
All 7 patients with SCD had no vascular occlusive crisis (VOCs)
.
The patient's follow-up time ranged from 4 months to 26 months
.
Among them, 5 TDT patients and 2 SCD patients were followed up for more than one year, showing a stable and long-lasting response to treatment
.
These data further support that CTX001 is expected to become a potential one-time cure for TDT and SCD
.
Both TDT and SCD are blood diseases caused by mutations in the gene encoding hemoglobin
.
Severe patients usually need to receive red blood cell transfusion frequently for treatment, which not only brings inconvenience to the patient, but also long-term red blood cell transfusion will bring side effects such as iron overload
.
CTX001 is an autologous CRISPR/Cas9 gene editing therapy under development
.
By modifying the patient's hematopoietic stem cells in vitro, high levels of fetal hemoglobin (HbF) are produced in the red blood cells
.
HbF is a form of hemoglobin that carries oxygen and exists naturally at birth.
As the baby grows up, the hemoglobin in the blood is converted to the adult form of hemoglobin
.
Through CTX001 treatment, HbF levels can be increased, it is possible to alleviate the blood transfusion needs of TDT patients, and reduce the pain and debilitating vascular occlusive crisis of SCD patients
.
CTX001 has been granted the Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of TDT and SCD by the US FDA, as well as fast track and orphan drug designation
.
▲ Mechanism of action of CTX001: A, BCL11A is a transcription factor that down-regulates the expression of fetal hemoglobin; B, CTX001 up-regulates the expression of fetal hemoglobin through gene editing of BCL11A (Source: Reference [2]) CLIMB- In 111 clinical trials, 15 patients all showed rapid and continuous increases in total hemoglobin, fetal hemoglobin, and blood transfusion independence
.
At the last follow-up, all patients were independent of blood transfusion, and the total hemoglobin level (8.
9 g/dL-16.
9 g/dL) and the proportion of fetal hemoglobin (67.
3%-99.
6%) showed clinically significant improvements
.
In the CLIMB-121 clinical trial for the treatment of patients with severe SCD, all 7 patients showed rapid and continuous increases in total hemoglobin and fetal hemoglobin, and eliminated VOCs
.
The follow-up time ranged from 5 to 22 months, and the hemoglobin level (11 g/dL-15.
9 g/dL) and the proportion of fetal hemoglobin (39.
6%-49.
6%) at the last follow-up showed clinically significant improvements
.
Dr.
Samarth Kulkarni, CEO of CRISPR Therapeutics, said: "The continuous progress of CTX001 verifies the possible role of CRISPR gene editing technology in future treatments
.
We are excited about these results and look forward to obtaining more long-term data to promote the development of this research therapy and benefit more patients with these two serious diseases
.
"References: [1] Vertex and CRISPR Therapeutics Present New Data in 22 Patients With Greater Than 3 Months Follow-Up Post-Treatment With Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at European Hematology Association Annual Meeting.
Retrieved June 11 , 2021, from https:// Frangoul et al.
, (2020).
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
NEJM, DOI: 10.
1056/NEJMoa2031054.
Note: This article aims to introduce the progress of medical and health research, not to recommend treatment plans
.
If you need guidance on treatment plans, please go to a regular hospital
.
The latest clinical trial data of CRISPR/Cas9 gene editing therapy CTX001 treatment
.
The results of the trial showed that all 15 TDT patients were no longer dependent on blood transfusions at the latest follow-up
.
All 7 patients with SCD had no vascular occlusive crisis (VOCs)
.
The patient's follow-up time ranged from 4 months to 26 months
.
Among them, 5 TDT patients and 2 SCD patients were followed up for more than one year, showing a stable and long-lasting response to treatment
.
These data further support that CTX001 is expected to become a potential one-time cure for TDT and SCD
.
Both TDT and SCD are blood diseases caused by mutations in the gene encoding hemoglobin
.
Severe patients usually need to receive red blood cell transfusion frequently for treatment, which not only brings inconvenience to the patient, but also long-term red blood cell transfusion will bring side effects such as iron overload
.
CTX001 is an autologous CRISPR/Cas9 gene editing therapy under development
.
By modifying the patient's hematopoietic stem cells in vitro, high levels of fetal hemoglobin (HbF) are produced in the red blood cells
.
HbF is a form of hemoglobin that carries oxygen and exists naturally at birth.
As the baby grows up, the hemoglobin in the blood is converted to the adult form of hemoglobin
.
Through CTX001 treatment, HbF levels can be increased, it is possible to alleviate the blood transfusion needs of TDT patients, and reduce the pain and debilitating vascular occlusive crisis of SCD patients
.
CTX001 has been granted the Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of TDT and SCD by the US FDA, as well as fast track and orphan drug designation
.
▲ Mechanism of action of CTX001: A, BCL11A is a transcription factor that down-regulates the expression of fetal hemoglobin; B, CTX001 up-regulates the expression of fetal hemoglobin through gene editing of BCL11A (Source: Reference [2]) CLIMB- In 111 clinical trials, 15 patients all showed rapid and continuous increases in total hemoglobin, fetal hemoglobin, and blood transfusion independence
.
At the last follow-up, all patients were independent of blood transfusion, and the total hemoglobin level (8.
9 g/dL-16.
9 g/dL) and the proportion of fetal hemoglobin (67.
3%-99.
6%) showed clinically significant improvements
.
In the CLIMB-121 clinical trial for the treatment of patients with severe SCD, all 7 patients showed rapid and continuous increases in total hemoglobin and fetal hemoglobin, and eliminated VOCs
.
The follow-up time ranged from 5 to 22 months, and the hemoglobin level (11 g/dL-15.
9 g/dL) and the proportion of fetal hemoglobin (39.
6%-49.
6%) at the last follow-up showed clinically significant improvements
.
Dr.
Samarth Kulkarni, CEO of CRISPR Therapeutics, said: "The continuous progress of CTX001 verifies the possible role of CRISPR gene editing technology in future treatments
.
We are excited about these results and look forward to obtaining more long-term data to promote the development of this research therapy and benefit more patients with these two serious diseases
.
"References: [1] Vertex and CRISPR Therapeutics Present New Data in 22 Patients With Greater Than 3 Months Follow-Up Post-Treatment With Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at European Hematology Association Annual Meeting.
Retrieved June 11 , 2021, from https:// Frangoul et al.
, (2020).
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
NEJM, DOI: 10.
1056/NEJMoa2031054.
Note: This article aims to introduce the progress of medical and health research, not to recommend treatment plans
.
If you need guidance on treatment plans, please go to a regular hospital
.
