The mAb, JSP191, will be tested as a targeted conditioning agent option for patients with Fabry disease and/or Gaucher disease type 1 who are being treated with one of Avrobio's investigational ex vivo lentiviral gene therapies.

JSP191 is a conditioning agent that blocks stem cell factor receptor signaling, which clears hematopoietic stem cells from bone marrow and leaves an empty space for donor or genetically modified transplanted stem cells to engraft. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients.

In addition, Jasper is launching two clinical trials to test JSP191 in patients with myelodysplastic syndromes/acute myeloid leukemia and severe combined immunodeficiency.

Avrobio gets orphan drug nod for Fabry disease gene therapy
Avrobio has been granted orphan drug designation by the European Commission for AVR-RD-01, an ex vivo lentiviral gene therapy that consists of a patient's...

Avrobio expands Hunter syndrome gene therapy program
Avrobio has signed a research and licensing agreement with the University of Manchester in the U.K. to investigate the use of lentiviral gene therapy...

Avrobio gets orphan drug nod for Gaucher disease gene therapy
Avrobio has been granted orphan drug designation for AVR-RD-02, an investigational gene therapy for treatment of Gaucher disease, by the European Commission.

Avrobio moves forward with gene therapy candidate
Avrobio released preclinical data for AVR-RD-03, lentiviral gene therapy for Pompe disease, at the American Society of Gene and Cell Therapy annual meeting...