Crispr's Gene-Editing Therapy Found Effective In Treating Blood Disorders In Early-Stage Study

Crispr Therapeutics AG CRSP shares are ripping higher after the company focused on developing gene therapies using its proprietary CRISPR/Cas9 platform said CTX001, a gene-editing therapy it's co-developing with Vertex Pharmaceuticals Incorporated VRTX, produced positive results in human trials.

Gene editing is a technology that aims at altering the DNA, which involves addition, removal and alteration of genetic material at a particular location in the genome. This helps treat or prevent genetic diseases by correcting mutations that lead to the disease.

CRISPR is a type of gene editing technology that allows rapid and accurate cutting of genetic material.

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Promising Outcome For Crispr, Vertex

Crispr and Vertex released interim data from the first two patients with severe hemoglobinopathies treated with CTX001 in ongoing Phase 1/2 trials, with the first patient with transfusion-dependent beta thalassemia (TDT) receiving the therapy in the first quarter of 2019 and the second person with sickle cell disease (SCD) receiving the therapy in mid-2019.

The companies noted that at nine months after CTX001 infusion, the patient with TDT, who required 16.5 transfusions per year ahead of the infusion, was transfusion independent and had total hemoglobin levels of 11.9 g/dL, 10.1 g/dL fetal hemoglobin, and 99.8% F-cells. The patient achieved neutrophil engraftment 33 days after the infusion and platelet engraftment four days later.

Four months after CTX001 infusion, the patient with SCD was free of vaso-occlusive crises and had total hemoglobin levels of 11.3 g/dL, 46.6% fetal hemoglobin, and 94.7% F-cells. Neutrophil and platelet engraftment were achieved 30 days after the infusion.

"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," said Vertex CEO Jeffrey Leiden. "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 myotonic dystrophy type 1."

Crispr shares traded higher by 12.5% to $65.86 at time of publication, while Vertex shares added 1.6% to $213.32.