Jasper Therapeutics, Avrobio Team Up to Assess JSP191

Yedida Y Bogachkov PhD avatar

by Yedida Y Bogachkov PhD |

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Jasper and Avrobio | Fabry Disease News | JSP191 | illustration of handshake

Jasper Therapeutics and Avrobio have entered into a non-exclusive research collaboration to evaluate the use of JSP191 in patients with Fabry disease who are being treated with one of Avrobio’s investigational gene therapies.

Under this collaboration, JSP191 — an anti-CD117 monoclonal antibody — also will be evaluated in Gaucher disease type 1, another disease that is caused by lysosomal enzyme mutations, for which Avrobio is developing a gene therapy treatment.

“This collaboration with Avrobio further expands the potential of JSP191 for use with lentiviral gene therapies,” Bill Lis, executive chairman and CEO of Jasper Therapeutics, said in a press release.

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Fabry disease is a lysosomal storage disorder characterized by the buildup of toxic materials within cells. The disease mainly affects the heart, nervous system, and kidneys and can lead to life-threatening symptoms.

It is caused by mutations in the GLA gene, located on the X chromosome, which provides instructions for producing the alpha-galactosidase A (AGA) enzyme. This enzyme is responsible for the breakdown of fatty molecules into components that cells can use.

JSP191 blocks CD117, a stem cell factor receptor required for the cells’ survival, thus causing the stem cells to die. This is important in therapeutic strategies for which stem cells in the bone marrow — responsible for creating blood cells and immune cells — need to be cleared so they can be replaced, via transplant, by healthy donor or genetically modified stem cells.

According to Jasper Therapeutics, JSP191’s use as a conditioning agent — a way of getting a patient ready for stem cell transplant — may be favored over the currently used radiation and chemotherapy agents because it may have fewer side effects. It already has been evaluated in more than 90 healthy volunteers and patients.

With the use of AVR-RD-01, Avrobio’s gene therapy protocol for Fabry disease, the GLA mutation can be fixed. The protocol uses lentiviruses that can integrate the healthy form of the GLA gene into stem cells, which then can be used to repopulate the cleared-out niche left by JSP191 so that a functional AGA enzyme can be produced.

“We believe the use of JSP191 in AVROBIO’s Fabry disease and/or Gaucher disease type 1 investigational gene therapy programs offers patients and doctors a compelling new option when it comes to matching a patient’s disease with a conditioning agent meeting their therapeutic goals and requirements,” Lis said.

Despite the collaboration, each company will retain the commercial rights to its respective technology.