AVROBIO Expands Lentiviral Gene Therapy Pipeline with Program for Hunter Syndrome - Seite 2

“We feel an enormous urgency to bring forward a treatment that may halt this deadly disease in its tracks, before symptoms emerge and before children lose their physical and cognitive skills,” said Prof. Bigger. “We are delighted to be working with AVROBIO on this program. Both of our teams have deep experience running international clinical trials in other lysosomal disorders. AVROBIO also has a leading gene therapy platform, plato, which is designed to optimize the consistency, predictability and efficacy of its gene therapies and to enable efficient scaling for worldwide commercialization. Both these factors make AVROBIO an excellent partner for our investigational Hunter syndrome gene therapy. By working together, we believe we can greatly accelerate development of this important program.”

The investigational gene therapy, which will be called AVR-RD-05, involves ex vivo transduction of the patient’s own hematopoietic stem cells with a therapeutic transgene designed to express functional enzyme the patient needs to maintain cellular health, coupled to a proprietary protein tag that is designed to improve stability of the enzyme in the bloodstream and facilitate uptake by tissues from head to toe. When reinfused into the patient, the gene-modified stem cells are expected to engraft in the bone marrow and produce generations of daughter cells, each carrying the transgene. Those daughter cells are then expected to differentiate into macrophages, microglia and other components of the immune system and circulate throughout the body and central nervous system, potentially enabling widespread distribution of functional enzyme.

AVROBIO’s other investigational gene therapies for lysosomal disorders are being evaluated in a Phase 1 and Phase 2 clinical trial for Fabry disease, a Phase 1/2 trial for cystinosis and a Phase 1/2 trial for Gaucher disease. In addition, the company is advancing a preclinical program in Pompe disease. Further details on the new Hunter syndrome program will be discussed at AVROBIO’s upcoming virtual R&D Day on Tuesday, Nov. 17, 2020.

Financial terms of the agreement

The University of Manchester’s technology transfer office, the University of Manchester Innovation Factory and AVROBIO have negotiated an exclusive, worldwide license agreement to the technology and a collaborative research funding agreement. Under the license agreement, AVROBIO will pay The University of Manchester an upfront cash payment and additional payments based on development and regulatory milestones. The company will also pay The University of Manchester a mid-single digit percentage royalty on annual net sales of licensed products. Additionally, under the collaborative research funding agreement, AVROBIO will cover budgeted Phase 1/2 clinical trial costs.