REGENXBIO announced last month that the Investigational New Drug application (IND) is active for a Phase I/II clinical trial of RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II) in children.
MPS II, also known as Hunter syndrome, is a progressively debilitating disorder that affects many areas of the body. It primarily occurs in males (very rarely is a female child affected). Progression varies from patient to patient. Those with a severe form of Hunter syndrome typically decline very quickly, while those with a milder form are living longer thanks to improved treatments for respiratory and cardiac health. To read more about this rare disease, click here.
RGX-121 has received orphan drug designation as well as rare pediatric disease designation from the FDA.
“The goal of the RGX-121 program is to develop a single-dose treatment for MPS II that can prevent the progression of neurocognitive decline experienced by children with the disease, which addresses one of the shortcomings of the current standard of care, enzyme replacement therapy,” said Stephen Yoo, M.D., Chief Medical Officer of REGENXBIO. “We expect to commence trial enrollment in the first half of 2018, and we look forward to working with leading gene therapy researchers and the broader MPS II community on this novel clinical program.”
Though this is still in its early phase, this news is heartening for a community where children are affected. This renews hope that treatment – and perhaps a cure, considering REGENXBIO’s gene therapy work – is very well on the horizon!