Avrobio has licensed a Hunter syndrome lentiviral gene therapy from the University of Manchester in the U.K. The deal positions Avrobio to join Regenxbio and Sangamo Therapeutics in the race to develop gene therapies for use in patients with the rare disorder.
Hunter syndrome, also known as mucopolysaccharidosis Type II, is driven by a mutation that limits a patient’s ability to break down sugar molecules generated as a byproduct of cell activity. The buildup of the molecules causes progressive damage to organs such as the brain, affecting the development of children with the lysosomal disorder.
Takeda’s approved drug Elaprase gives Hunter syndrome patients the enzyme they need to break the molecules down, but its inability to cross the blood-brain barrier renders it ineffective against the CNS manifestations of the disease.