Gene therapy involves delivering genetic material to cells, often using a specialized carrier. Viruses are frequently used as carriers for gene therapies because of their natural ability to transport genetic material into cells. Viruses are modified in the laboratory to ensure they do not cause disease when used in humans.iii For Hunter syndrome, the investigational therapy uses a viral carrier to deliver a functional copy of the IDS gene to cells to the central nervous system.iv
The IDS gene codes for an important enzyme called iduronate-2-sulfatase (I2S). People with Hunter syndrome have a mutation in this gene, leading to a deficiency of the I2S enzyme and widespread symptoms affecting many body systems.v The investigational gene therapy for Hunter syndrome is designed to deliver a copy of the IDS gene to cells of the central nervous system, potentially enabling long-term production of the I2S enzyme.iv Initial findings from clinical trials have shown evidence of increased I2S activity following administration of this gene therapy.iv Currently, the U.S. Food and Drug Administration (FDA) is reviewing comprehensive safety and efficacy data to evaluate the 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.
Hunter syndrome, also known as mucopolysac-charidosis type II (MPS II), is a rare genetic condition that impacts many parts of the body.
Exon-skipping has the potential to treat Duchenne muscular dystrophy.
JAK1 inhibition is being developed to treat EGPA, a type of vasculitis.
Hunter syndrome, also known as mucopolysac-charidosis type II (MPS II), is a rare genetic condition that impacts many parts of the body.
Exon-skipping has the potential to treat Duchenne muscular dystrophy.
JAK1 inhibition is being developed to treat EGPA, a type of vasculitis.
