Hunter syndrome affects the entire body, but its severity can range from attenuated (less severe) to neuronopathic (more severe) depending on the degree of brain involvement and cognitive impairment.vi
People with the attenuated form typically live into adulthood with minimal cognitive impairment, whereas people with the neuronopathic form experience severe neurodevelopmental decline and have a shortened life expectancy.vi
Treatment options for Hunter syndrome aim to address the deficiency of the I2S enzyme.vii,viii In the U.S., the only currently approved treatment for Hunter syndrome is enzyme replacement therapy (ERT). However, ERT is unable to reach the brain and treat symptoms affecting the central nervous system (CNS).vii,viii
Our pipeline includes an investigational gene therapy which has been developed to address this unmet need.iv The therapy is designed to deliver a version of the gene responsible for I2S directly to CNS cells, potentially enabling long-term I2S production.iv Currently, the U.S. Food and Drug Administration (FDA) is reviewing comprehensive safety and efficacy data to evaluate this gene therapy for potential regulatory approval.iv
We go beyond symptom management, providing patients with potentially disease-modifying
treatment options that could improve and extend quality time with loved ones.
Gene therapy has the potential to address critical genetic mutations, like those causing Hunter syndrome.
Exon-skipping has the potential to treat Duchenne muscular dystrophy.
JAK1 inhibition is being developed to treat EGPA, a type of vasculitis.
Gene therapy has the potential to address critical genetic mutations, like those causing Hunter syndrome.
Exon-skipping has the potential to treat Duchenne muscular dystrophy.
JAK1 inhibition is being developed to treat EGPA, a type of vasculitis.
