Our Mission in Motion
At Dawn Therapeutics, our pipeline reflects where cutting-edge science meets patient need. Every programme is grounded in tissue-targeted delivery technology, robust preclinical evidence, and a commitment to transformative impact in CNS, lysosomal storage, and musculoskeletal disorders.
We prioritise diseases with:
- A clear genetic cause or molecular pathway.
- High unmet medical need.
- Feasible, targeted delivery to affected tissues.
- Measurable biomarkers for efficacy and safety.
Pipeline Overview
Our development programmes span discovery to clinical stage and use a variety of modalities, including:
- AAV Gene Therapies
- LNP-mRNA Therapeutics
- RNAi and ASO Therapies
- Gene Editing Solutions
Current Programmes
Lysosomal Storage Disorders (LSDs)
MPS I – Hurler Syndrome
MPS II – Hunter Syndrome
MPS III – Sanfilippo Syndrome
MPS IV – Morquio Syndrome
MPS VI – Maroteaux–Lamy Syndrome
MPS VII – Sly Syndrome
MPS IX – Natowicz Syndrome
Lysosomal Storage Disorders – Umbrella Programme
Musculoskeletal Disorders
Osteoarthritis (OA)
Musculoskeletal Disorders – Umbrella Programme
Duchenne Muscular Dystrophy (DMD)
Friedreich’s Ataxia
Amyotrophic Lateral Sclerosis (ALS)
CNS Disorders
Alzheimer’s Disease
Parkinson’s Disease
Huntington’s Disease
Autism Spectrum Disorder
Addiction Disorders
Spinal Cord Injury (SCI)
Our Development Philosophy
Precision First
Each programme undergoes detailed biodistribution and biomarker profiling before entering the clinic.
Patient-Centric Trials
Eligibility criteria ensure that those most likely to benefit are prioritised.
Regulatory Engagement
Early consultation with agencies to align on safety, endpoints, and trial design.
Scalable Manufacturing
CMC processes established early for seamless scale-up.
Strategic Partnerships
We actively seek partnerships to accelerate programmes through:
• Co-development agreements
• Licensing opportunities
• Technology access collaborations
Recent Milestones
Preclinical studies initiated
Initiated preclinical studies for MPS I & II gene therapy programmes.
CNS portfolio expansion
Expanded CNS portfolio to include targeted mRNA therapy for Autism Spectrum Disorder.
Scalable CMC process
Established scalable CMC process for lead AAV vector platform.
Academic partnerships
Partnered with academic research centres for biomarker validation.
