Gene Therapy Clinical Trial for MPS II
Homology is conducting the juMPStart clinical trial with investigational one-time gene therapy HMI-203 in adults with Hunter syndrome.
HMI-203 is designed to deliver functioning IDS genes to the peripheral organs and the central nervous system.
What does participation in the juMPStart trial involve?
Screening Period
The trial will include a screening period to ensure participants meet the eligibility criteria to enroll in the study.
Study Period
Following a one time I.V. dose of HMl-203, participants will be observed periodically for 52 weeks.
Follow-Up Period
Participants will be seen less frequently for another 4 years.
How could HMI-203 potentially work in the body?
Step 1
A patient could receive a one-time intravenous (I.V.) administration of HMI-203.
Step 1
A patient could receive a one-time intravenous (I.V.) administration of HMI-203.
Step 2
HMI-203 could reach organs throughout the body (peripheral organs) and the nervous system (by crossing the blood-brain and blood-nerve barriers) where I2S protein is required to eliminate the build-up of GAGs.
Step 3
HMI-203 could enter the cells, where it would deliver the functional gene and promoter, which would help “activate” the gene.
Step 4
HMI-203 could result in I2S protein expression and systematic reduction in GAGs.
Step 5
Goal to lead to cross-correction.
To learn more about juMPStart:
juMPStart Fact Sheet (English)
juMPStart Fact Sheet (Spanish)
Hunter Syndrome Gene Therapy Fact Sheet (English)
Hunter Syndrome Gene Therapy Fact Sheet (Spanish)
HMI-203 is not approved by the U.S. Food & Drug Administration (FDA) or any other regulatory agency.