Pioneering Targeted Gwnw Therapy For Herler Syndrome (MPS I)
Delivering corrective genes directy to the brain, cartilage, and bone to address the root cause of this rare , live-limiting condition.
What is Hurler Syndrome?
Hurler Syndrome, or Mucopolysaccharidosis Type I (MPS I), is a rare lysosomal storage disorder caused by a deficiency in the enzyme α-L-iduronidase. This enzyme is critical for breaking down glycosaminoglycans (GAGs) — complex sugars found in connective tissue.
- Genetics: Autosomal recessive; mutations in the IDUA gene
- Onset: Symptoms typically appear in infancy or early childhood
- Prevalence: Approx. 1 in 100,000 live births worldwide
How Does Hurler Syndrome Affect Patients?
Neurological
Developmental delay, cognitive decline, hydrocephalus
Musculoskeletal
Skeletal deformities, joint stiffness, short stature
Cardiac
Heart valve disease, cardiomyopathy
Respiratory
Airway obstruction, sleep apnoea
Limitations of Existing Therapies
Enzyme Replacement Therapy
Can reduce some systemic symptoms but does not cross the blood–brain barrier, leaving neurological disease untreated.
Stem Cell Transplantation
May slow progression but carries significant procedural risks and is most effective if performed early.
Supportive Care
Physical therapy, surgeries, and symptom management remain necessary but don’t address root cause.
Delivering a Corrective Solution Where It Matters Most
At Dawn Therapeutics, we are developing a tissue-targeted gene therapy designed to address both neurological and systemic symptoms by delivering a functional copy of the IDUA gene directly to affected tissues.
- Cross the blood–brain barrier to treat neurological disease
- Reach cartilage and bone to address musculoskeletal complications
- Sustain enzyme production long-term to minimise treatment burden
Where We Are in the Pipeline
Preclinical Data
Successful vector delivery demonstrated
IND-Enabling Studies
Currently optimizing dosing & safety
Phase 1 Trial
Preparing for human studies
Future Expansion
Broader patient population
Support for Families Affected by Hurler Syndrome
We collaborate with advocacy organisations to ensure patients and caregivers have access to educational resources, clinical trial information, and patient navigation services.
MPS Society
Rare Disease Foundation
Patient Advocates
Global Alliance
Collaboration for Impact
We welcome partnerships with researchers, biotech and pharma companies, and advocacy organisations to accelerate the development of transformative therapies for MPS I.
