Study to Evaluate Using JSP191 With ARU-1801 as Gene Therapy

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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A new research collaboration between Jasper Therapeutics and Aruvant Sciences will evaluate the use of JSP191, Jasper’s anti-CD117 monoclonal antibody, as a conditioning agent for ARU-1801, Aruvant’s experimental gene therapy for sickle cell disease (SCD).

“The unique attributes of ARU-1801 enable us to bring a potentially curative one-time therapy to individuals with sickle cell disease that can be delivered in the safest way possible. By partnering with Jasper to evaluate the use of JSP191 with ARU-1801, we are one step closer to developing a next-generation definitive therapy with an even more patient-friendly conditioning regimen,” Will Chou, MD, CEO of Aruvant, said in a press release.

ARU-1801 is designed to genetically modify a patients’ blood stem cells so they give rise to red blood cells capable of producing fetal hemoglobin — a form of hemoglobin produced early in life that is more effective at transporting oxygen than its adult counterpart. Increasing  fetal hemoglobin levels is expected to restore red blood cell function and improve oxygen transport, thereby lessening SCD symptoms.

Recently announced data from an ongoing Phase 1/2 clinical trial called MOMENTUM (NCT02186418) showed that ARU-1801 was able to trigger the production of fetal hemoglobin in red blood cells and lower the number of vaso-occlusive crises in three people with SCD.

When a person undergoes treatment with ARU-1801, blood stem cells must first be harvested from the bone marrow, then modified in a lab, and finally given back to the patient.

Before cells can be put back, however, the patient must undergo a process called conditioning. Basically, this involves receiving treatment that aims to destroy faulty blood stem cells already in the bone marrow, in order to “make room” for those that have been engineered via gene therapy.

Standard conditioning regimens generally involve chemotherapy and/or radiation therapy. While these treatments are effective at killing blood stem cells, they carry substantial safety risks, such as cancer, infections, and infertility. Notably, the conditioning regimen being used in the MOMENTUM trial consists of a relatively low

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chemotherapy dose, which has a more limited side effect profile than standard regimens.

JSP191 is designed to target CD117, a protein found on the surface of blood stem cells that helps control their growth and survival. The overall aim of using JSP191 as a conditioning regimen is to more precisely target these cells for destruction, thereby boosting treatment’s effectiveness while minimizing its potential side effects.

“We believe that this combination may be able to further expand the number of patients who can benefit from ARU-1801 in the future, including potentially those with more moderate disease,” Chou said.

Kevin N. Heller, MD, executive vice president of research and development at Jasper, said that combining JSP191 with ARU-1801 presents “a clear advantage by moving beyond the harsh conditioning agents currently used for gene therapy and establishing this next-generation potentially curative treatment as a leader in sickle cell disease.”

Last year, Jasper raised more than $50 million to fund the development of JSP191. Heller said the company intends “to establish JSP191 as a potential new standard of care conditioning agent” for both gene therapies and stem cell transplants.