Our Approach to Transformative Medicines
At Dawn Therapeutics, our medicines are built on a simple but powerful principle: deliver the right therapy, to the right place, at the right time. By combining tissue-targeted delivery systems with cutting-edge RNA and gene therapies, we are reimagining how complex diseases can be treated — from the brain to cartilage, muscle, and beyond.
Our focus is on life-changing treatments for CNS disorders, lysosomal storage diseases, musculoskeletal conditions, and other high-impact rare genetic disorders
How Our Medicines Work
Each of our therapies is carefully designed to:
- Address the root cause of disease, not just the symptoms.
- Precisely target the tissues and cells affected.
- Minimise off-target effects for improved safety.
- Deliver lasting benefit — in some cases, with a single treatment.
Therapeutic Modalities We Use:
- AAV Gene Transfer – Long-lasting therapeutic protein expression for diseases caused by missing or faulty genes.
- LNP-mRNA Therapies – Flexible, repeat-dose solutions where transient expression is preferred.
- RNA Interference (RNAi) – Silencing harmful gene products.
- Antisense Oligonucleotides (ASOs) – Correcting splicing or blocking toxic RNA.
- Gene Editing – Correcting or disabling disease-causing genes at the DNA level.
Therapeutic Areas
Our medicines are designed for patients with:
- CNS Disorders – Alzheimer’s, Parkinson’s, Huntington’s, Autism Spectrum Disorder, Addiction Disorders, Spinal Cord Injury, and more.
- Lysosomal Storage Disorders – MPS I, MPS II, MPS III, MPS IV, MPS VI, MPS VII, MPS IX, and related conditions.
- Musculoskeletal Disorders – Osteoarthritis, Duchenne Muscular Dystrophy, and rare skeletal disorders.
- Other Rare Genetic Diseases – Including ultra-rare indications with high unmet need.
Our Development Stages
Our medicines progress through a clear and disciplined development pathway:
Discovery & Preclinical
We identify disease targets, select the most effective therapeutic modality, and validate our approach in preclinical models.
Clinical Trials
We design biomarker-led clinical trials to ensure patients most likely to benefit are enrolled, while collecting high-quality safety and efficacy data.
Regulatory Review
We work closely with global regulators to meet the highest safety and efficacy standards.
Patient Access
We collaborate with healthcare systems, advocacy groups, and payers to ensure equitable access worldwide.
Commitment to Safety
Safety is at the core of every Dawn Therapeutics medicine. We use:
- Advanced biodistribution profiling to confirm targeted delivery.
- Long-term follow-up protocols to track durability and late-onset effects.
- Global compliance with ICH, GCP, and GMP standards.
Making Medicines Accessible
We are committed to:
- Supporting early diagnosis through newborn screening advocacy.
- Providing patient support programmes to guide families through treatment.
- Partnering globally to make medicines affordable and accessible.