Massachusetts-based AVROBIO announced today that it has entered an exclusive global license agreement, as well as a collaborative research funding agreement, with The University of Manchester. Together, the university and AVROBIO will look into an investigational lentiviral gene therapy for mucopolysaccharidosis type II (MPS II), or Hunter syndrome.

The condition, which impacts an estimated one in 100,000 males worldwide, causes complications throughout the body and brain. Children with severe cases typically show symptoms beginning in their toddler years. At the moment, the standard of care is weekly enzyme replacement therapy, but it does not halt progression of the disease or address cognitive issues that may arise.

We believe a lentiviral gene therapy approach is well suited to treat a progressive and pervasive disease such as Hunter syndrome, which affects organs throughout the body and severely impairs cognitive function. If we treat children early, before their symptoms arise, we hope to prevent the tragic complications that rob these young children of their futures, said Geoff MacKay, AVROBIOs president and CEO. We believe our deep experience with investigational gene therapies for lysosomal disorders will enable us to efficiently move the program through clinical development in collaboration with Prof. Brian Bigger, who has done tremendous work to develop and optimize this investigational gene therapy. Were proud to add this program to our leading lysosomal disorder pipeline and excited about its potential to change the lives of patients and families living with Hunter syndrome.

The investigational gene therapy, titled AVR-RD-05, includes ex vivo transduction of the patients own hematopoietic stem cells with a therapeutic transgene. The transgene is meant to express functional enzymes that the patient needs to maintain cellular health. When reinfused back into the patient, the modified stem cells are designed to engraft in the bone marrow and produce generations of daughter cells, each carrying the transgene.

This is just one company looking toward making an impact in the MPS II realm as of late. REGENXBIO announced at the end of September that it was expanding its RGX-121 program, looking into the treatment of MPS II. RGX-121 is an investigational one-time gene therapy that uses the AAV9 vector to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme directly to the central nervous system.

An ongoing Phase I/II study is evaluating a single intracisternal administration of RGX-121 in severe instances of MPS II in patients under the age of five. As of Sept. 16, RGX-121 was reported to be well-tolerated in patients and there were no drug-related serious adverse events.

"MPS II is a serious and debilitating lysosomal disease that affects 1 in 100,000 children, and available treatments are inadequate to treat the neurodegenerative manifestations of the disease, said Terri Klein, President and Chief Executive Officer of the National MPS Society. Initiating a natural history study will increase the understanding of neurocognitive effects and key biomarkers of severe MPS II, and is critical to advancing the development of new treatment options. We are grateful for REGENXBIO's dedication to MPS and commitment to share the learnings from this observational study with the community.

REGENXBIO has also announced that the U.S. Food and Drug Administration cleared an Investigational New Drug application. The company plans on initiating a second Phase I/II multicenter, open-label trial of RGX-121 for the treatment of pediatric patients with severe MPS II between the ages of five and 18.

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AVROBIO and University of Manchester Enter Agreement for MPS II Research - BioSpace

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