Targeted Gene Therapy for Hunter Syndrome (MPS II)
Delivering the IDS gene to where it is needed most – in both body and brain.
Understanding Hunter Syndrome
Hunter syndrome (MPS II) is a rare, X-linked lysosomal storage disorder caused by deficiency of the enzyme iduronate-2-sulfatase (IDS).
Without sufficient IDS activity, glycosaminoglycans (GAGs) accumulate in cells and tissues, leading to progressive damage across multiple organ systems.
X-linked inheritance
Affects 1 in 162,000 males
IDS Enzyme Pathway
GAG accumulation disrupts cellular function when IDS enzyme is deficient
Symptoms and Impact
Neurological
- Developmental delay
- Behavioral changes
- Cognitive regression
Cardiac
- Heart valve dysfunction
- Cardiomyopathy
- Reduced cardiac output
Musculoskeletal
- Joint stiffness
- Short stature
- Skeletal deformities
Current Treatment Landscape
Enzyme Replacement Therapy
Systemic enzyme delivery via IV infusions
Stem Cell Transplantation
Hematopoietic stem cell transplantation
Crossing Barriers, Changing Outcomes
CNS-targeted gene delivery for neurological symptom control
Systemic enzyme restoration for multi-organ health
Single-dose potential for long-term benefit
Targeted Delivery System
Our proprietary platform delivers functional IDS genes directly to both CNS and systemic tissues
Research and Development Progress
Supporting Patients & Advocacy
Dawn Therapeutics recognizes the importance of holistic support for patients and families affected by Hunter syndrome.
- Genetic counselling for affected families and carriers
- Clinical trial navigation for eligible patients
- Educational resources for families and healthcare providers
Hunter Syndrome Foundation
MPS Society
National MPS Society
Rare Disease Advocacy
Partner With Us
Join us in advancing gene therapy solutions for rare diseases. Together, we can make a difference in patients’ lives.