AVROBIO announce collaborative research agreement with Manchester university for MPS II gene therapy

AVROBIO, a leading clinical-stage gene therapy company today announced an exclusive, worldwide license agreement and a collaborative research funding agreement with The University of Manchester for an investigational lentiviral gene therapy for mucopolysaccharidosis type II (MPS II), or Hunter syndrome.


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AVROBIO said they are proud to add this program to their lysosomal disorder pipeline and are excited about the opportunity to advance research and support the Hunter community. The investigational lentiviral gene therapy for Hunter syndrome, which will be called AVR-RD-05, is the fifth therapy in AVROBIO’s pipeline to target lysosomal disorders. It aims to address both the physical and CNS manifestations of Hunter syndrome. An investigator-sponsored Phase 1/2 clinical trial will be conducted by The University of Manchester team and is expected to begin in the second half of 2021.


"If we treat children early, before their symptoms arise, we hope to prevent the tragic complications that rob these young children of their futures."

Geoff MacKay, AVROBIO’s president and CEO


"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. 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."

Professor Brian Bigger, The University of Manchester


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