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Trial Shows Gene Therapy Results with Sickle Cell Disease

Sickle cells illustration

Sickle cells illustration (Open Stax College, wikimedia commons,

16 May 2022. Early results from a clinical trial show an experimental gene transfer therapy largely reduces painful events from sickle cell disease for one to two years. Findings from the trial sponsored by the therapy’s developer Aruvant Sciences are scheduled for delivery today at a meeting of American Society of Gene and Cell Therapy in Washington, D.C.

Sickle cell disease is a genetic blood disorder where hemoglobin, a protein in blood that delivers oxygen to cells in the body, causes blood cells to become crescent or sickle shaped instead of their normal round form. The abnormal shape causes blood cells to break down, become less flexible, and accumulate in tiny blood vessels, leading to anemia and periodic painful episodes. Sickle cell disease is prevalent worldwide, and affects some 100,000 people in the U.S., including about 1 in 500 people of African descent, and 1 in 1,000 to 1,400 people of Hispanic descent.

Aruvant Sciences is a four year-old biotechnology enterprise in New York developing gene therapies for rare diseases, beginning with the blood disorders sickle cell disease and beta-thalassemia where people produce lower levels of hemoglobin in their blood. The company is part of collection of businesses created by Roivant Sciences in Basel, Switzerland to develop biomedical technologies. Roivant calls the individual companies “vants” with each of the companies focusing on a particular disease or technology type. Science & Enterprise reported on formation of Aruvant Sciences in Nov. 2018.

Lower chemotherapy doses

The clinical trial is evaluating Aruvant Sciences’ lead product code-named ARU-1801 that uses benign lentiviruses to deliver a modified healthy fetal hemoglobin gene to a patient’s own blood-forming stem cells. In this case, a sample of the patient’s stem cells are extracted from bone marrow, then cultured and transformed into healthy blood-forming cells with functioning genes producing fetal hemoglobin, and infused back into the patient. This process was first developed by Punam Malik, a hematologist at Cincinnati Children’s Hospital studying gene therapies for blood disorders, and licensed to Aruvant Sciences.

The early- and mid-stage trial, led by Malik, is enrolling 10 adults with severe sickle cell disease, or SCD, in the U.S. and Jamaica. Participants first receive a form of chemotherapy to reduce immune-system interference with the ARU-1801 infusions. The company says it uses lower doses of chemotherapy to reduce chances of adverse effects. The study team tracks participants for two years looking mainly for severe or life-threatening adverse effects from the gene therapy, as well as efficacy indicators, such as fetal hemoglobin measures and number of painful episodes from sickle cells blocking blood vessels, called vaso-occlusive events. The study has no control or comparison group.

Results presented today report on four participants in the trial, age 19 to 34, receiving ARU-1801, and followed for 12 to 24 months. Researchers say participants experienced temporary low white blood cell and platelet cell counts for a median period of seven days, but otherwise no serious adverse effects from the chemotherapy or ARU-1801 treatment. In addition, two participants report no painful episodes in the 12 to 18 months following treatment, while two others report 80 and 93 percent fewer events in the 24 months after receiving ARU-1801.

“The data demonstrate,” says Malik in an Aruvant Sciences statement, “that the ARU-1801 gene therapy may not only be able to reduce severe vaso-occlusive events, but also reduce days in the hospital for SCD patients, which could provide a clinically meaningful benefit for patients and help reduce health care costs.”

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