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This is the first time CRISPR/Cas9 gene-editing technology has been used in human trials to treat genetic diseases.
CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals (NASDAQ:VRTX) released positive data from their combined gene-editing clinical trials on Tuesday (November 19). This is the first time CRISPR/Cas9 technology has been used in human trials.
Two patients with genetic blood disorders — one with transfusion-dependent beta thalassemia and the other with sickle cell disease — were treated with one time gene-editing therapy CTX001.
At the four and nine month follow ups, both patients indicated that the treatment was safe and effective, highlighting that there were no flare ups. Neither needed a blood cell transfusion.
“These data support our belief in the potential of our therapies to have meaningful benefit for patients following a one-time intervention,” Samarth Kulkarni, PhD, CEO of CRISPR Therapeutics, said in a release. “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.”
CRISPR technology has been making advances as a treatment for a range of ailments, including cancer. The technology edits the human genome and allows modifications to be made to DNA sequences.
In the case of the two trials from CRISPR Therapeutics and Vertex Pharmaceuticals, patients’ blood cells were edited and then infused back into the patients, with CTX001 as part of the stem cell transplant. The trials are ongoing, and each will see 45 patients participate for two years after being treated with CTX001.
The first study is now enrolling at six sites split between Canada, the US and Europe, while the latter is enrolling patients at 12 sites in the same regions.
“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,” Jeffrey Leiden, MD, PhD, chairman, president and CEO of Vertex, said in the joint release.
As the US National Library of Medicine describes it, beta thalassemia is a blood disorder that prevents hemoglobin from being produced. Hemoglobin is an iron-producing protein found in red blood cells that helps oxygen flow in the body.
When patients have beta thalassemia, most often they have a low amount of red blood cells, causing weakness, fatigue and pale skin, and are at risk of developing blood clots.
Sickle cell disease encompasses multiple disorders that impact hemoglobin and usually begin presenting early in life. Symptoms include a low red blood cell count, painful episodes and repeat infections. When red blood cells sickle, it can lead to anemia due to the cells breaking down too early.
Tuesday’s announcement comes following the release of CRISPR Therapeutics’ Q3 financial results for this year in late October.
“We … began treating patients in our clinical trial for CTX110, our allogeneic CAR-T therapy, and are advancing additional CAR-T candidates toward clinical development,” Kulkarni said.
In addition to its ongoing trials of CTX110 in beta thalassemia and sickle cell disease, the company said it has started a Phase 1/2 trial of CTX110 in CD19+ malignancies, which is a protein that typically diagnoses B-cell lymphomas, acute lymphoblastic leukemia and chronic lymphocytic leukemia.
In this trial, the company is currently enrolling patients across five sites in the US and Australia.
Shares of CRISPR Therapeutics were up almost 17 percent from its previous close to end Tuesday’s trading session at US$68.47. Vertex Pharmaceuticals rose marginally in comparison by 2.38 percent to close Tuesday at an even US$215.
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Securities Disclosure: I, Jocelyn Aspa, hold no direct investment interest in any company mentioned in this article.