- The patient with TDT has the β0/IVS-I-110 genotype and required 16.5 transfusions per year before enrolling in the clinical study. The patient achieved neutrophil engraftment 33 days after the CTX001 infusion and platelet engraftment 37 days after infusion.
- Two serious adverse events (SAEs) occurred, neither of which the principal investigator (PI) considered related to CTX001: pneumonia in the presence of neutropenia and veno-occlusive liver disease attributed to busulfan conditioning, both subsequently resolved.
- At nine months after the CTX001 infusion the patient was transfusion independent and had total hemoglobin levels of 11.9 g/dL, 10.1 g/dL fetal hemoglobin and 99.8% F-cells (erythrocytes expressing fetal hemoglobin).
- The patient with SCD experienced seven VOCs per year before enrolling in the clinical study. The patient achieved neutrophil and platelet engraftment 30 days after the CTX001 infusion.
- Three SAEs occurred, none of which the PI considered related to CTX001: sepsis in the presence of neutropenia, cholelithiasis and abdominal pain, all of which resolved.
- At four months after the CTX001 infusion the patient was free of VOCs and had total hemoglobin levels of 11.3 g/dL, 46.6% fetal hemoglobin and 94.7% F-cells (erythrocytes expressing fetal hemoglobin).
CRISPR and Vertex Comments on the Results
Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics said, “We are very encouraged by these preliminary data, the first such data to be reported for patients with beta-thalassemia and sickle cell disease treated with our CRISPR/Cas9 edited autologous hematopoietic stem cell candidate, CTX001. These data support our belief in the potential of our therapies to have meaningful benefit for patients following a one-time intervention. We continue to enroll these studies as we drive forward to develop CRISPR/Cas9 therapies as a new class of transformative medicines to treat serious diseases.”
Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer of Vertex said, “The data we announced today are remarkable and demonstrate that CTX001 has the potential to be a curative CRISPR/Cas9-based gene-editing therapy for people with sickle cell disease and beta-thalassemia. While the data are exciting, we are still in the early phase of this clinical program. We look forward to continuing to work with physicians, patients, caregivers, and families over the coming months and years to bring forward the best possible therapy for these two serious diseases and to continue to accelerate our gene-editing programs for other serious diseases such as Duchenne muscular dystrophy and type 1 myotonic dystrophy.”
About CTX001
CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells.
HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients.
Vertex and CRISPR Therapeutics Collaboration
CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. This strategic research collaboration was signed in 2015 and focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease.
CTX001 is the first treatment to emerge from the joint research program.
CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.
Prohost Observations
After two years of opposing opinions about the new gene-editing CRISPR surfaced, which has led to the yo-yoing of the gene-editing stocks, we are hearing extremely promising results about TDT and SCD.
In TDT, at nine months, after the CTX001 infusion, the transfusion-dependent patient became independent with total hemoglobin levels of 11.9 g/dL, 10.1 g/dL fetal hemoglobin, and 99.8% F-cells (erythrocytes expressing fetal hemoglobin).
In SCD, four months after the CTX001 infusion, the patient was free of VOCs and had total hemoglobin levels of 11.3 g/dL, 46.6% fetal hemoglobin, and 94.7% F-cells (erythrocytes expressing fetal hemoglobin).
This is undeniable good new that offers foretaste about future treatments of genetic hereditary diseases using CRISPR gene editing.
At this hour:
VRTX is trading at $213.51,UP $3.51.
CRSP is trading at $66.86,UP $8.33.
That’s Good News. Isn’t It?
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Why Vertex and CRISPR Rallied Today
Vertex Pharmaceuticals and CRISPR Therapeutics Rallied Today
Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP) announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials.
A patient with transfusion-dependent beta-thalassemia (TDT) received CTX001 in the first quarter of 2019. Data for this patient reflect nine months of safety and efficacy follow-up.
A patient with severe sickle cell disease (SCD) received CTX001 in mid-2019. Data for this patient reflect four months of safety and efficacy follow-up.
These studies are ongoing and patients will be followed for approximately two years post-infusion.
Several additional patients have been enrolled and have had drug products manufactured across the two studies.
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