Home » News » Biotech Stock Spikes On Promising First In-Human CRISPR Gene-Editing Drug


Shares of Crispr Therapeutics (CRSP) jumped Tuesday after the biotech company’s gene-editing approach showed early promise. During the regular session on the stock market today, Crispr stock popped 17%, to 68.46, in above-average volume.

At nine months following infusion, the beta thalassemia patient didn’t need a blood transfusion. Four months after treatment, the sickle cell patient didn’t experience any vaso-occlusive crises, a painful complication of the disease.

CRISPR Gene-Editing Drug Shows Promise

Prior to receiving the CRISPR gene-editing treatment, the patient with beta thalassemia needed 16.5 blood transfusions each year, at an annualized rate for two years. Since the last infusion in the first quarter of 2019, that patient hasn’t required a transfusion.

The sickle cell patient experienced seven vaso-occlusive crises per year prior to infusion, at an annualized rate for two years. Since receiving the drug in mid-2019, the patient hasn’t experienced any of these painful complications.

The beta thalassemia patient showed sustained levels of white blood cells after 33 days. Platelets were sustained at 37 days post-infusion. The sickle cell patient hit those levels at 30 days following the CRISPR gene-editing treatment.

Both patients are approaching normal levels of hemoglobin, a protein used to transport oxygen in the bloodstream.

The pair also showed high levels of fetal hemoglobin, which is beneficial because it binds to oxygen with greater affinity than adult hemoglobin. In the beta thalassemia patient, 99.8% of certain red blood cells showed fetal hemoglobin. The sickle cell patient was at 94.7%.

“Management noted that the levels of fetal hemoglobin in seen in these patients exceeded their expectations (noting they were hoping for least 30% fetal hemoglobin), which has been shown to ameliorate disease in (blood diseases),” RBC’s Abrahams said.

Side Effects Are Likely To Continue

Although there were side effects, none were related to the CRISPR gene-editing drug. The beta thalassemia patient experienced pneumonia and a liver complication.

The liver complication was related to the use of the drug busulfan. Surgeons use busulfan to prepare a patient for a bone-marrow transplant. This is important because Crispr and Vertex’s drug is a gene-edited stem cell transplant. So, patients will need busulfan prior to receiving it.

The sickle cell patient developed sepsis, gallstones and abdominal pain.

“Both patients developed infections (sepsis and pneumonia), likely related to the conditioning regimen required for the transplant, reaffirming that the one-time procedure — while potentially curative — is not benign,” RBC’s Abrahams said.

Still, he expects the biotech companies to accelerate enrollment in the study. Crispr and Vertex say they plan to study up to 45 patients in each disease for roughly two years.


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