The juMPStart study is investigating the safety and efficacy of an investigational one-time gene therapy, HMI-203 in adult patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome. In Hunter syndrome, the iduronate-2-sulfatase (IDS) gene is missing or malfunctioning and the body is unable to produce the iduronate-2-sulfatase (I2S) enzyme that breaks down glycosaminoglycans (GAGs), which are large sugar molecules or cellular waste. The resulting accumulation of GAGs in patients with MPS II causes symptoms such as joint stiffness, carpal tunnel syndrome, sleep apnea and loss of hearing.
Currently, there are no available treatments targeting the genetic cause of MPS II; standard of care treatments only address the symptoms. HMI-203 is an investigational gene therapy designed to deliver the IDS gene with a one-time intravenous (I.V.) infusion.
In the juMPStart study, investigational HMI-203 is designed to deliver functional copies of IDS gene throughout the body, including to peripheral organs and the central nervous system, using an adeno-associated virus (AAV). Participation in this clinical trial may include enzyme replacement therapy (ERT) discontinuation.
You may be eligible to participate in the juMPStart study if you:
- Are a male between the ages of 18-30 years
- Have a confirmed diagnosis of MPS II
- Are currently receiving ERT as treatment for MPS II
Additional eligibility criteria will apply.