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:
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 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 Investigational Modalities
At Dawn Therapeutics, we are developing platform-agnostic modalities tailored for precision delivery. All are currently in preclinical stages and under active research.
LNP-mRNA Therapies
We harness lipid nanoparticle delivery systems to transport messenger RNA payloads effectively into target cells. Our CNS and bone/cartilage platforms are designed to localize mRNA expression in specific tissues, minimizing off-target exposure.
RNA Interference (RNAi)
Through small interfering RNA (siRNA) or short hairpin RNA (shRNA) strategies, we aim to downregulate aberrant gene expression in disease models. Our targeting frameworks may enable selective suppression with reduced systemic toxicity.
Antisense Oligonucleotides (ASOs)
ASOs designed to modulate splicing, mRNA stability, or transcript levels are under exploration. Our delivery approaches seek to enhance efficacy in specific tissues historically difficult to reach.
AAV Gene Transfer
Adeno-associated virus (AAV) vectors remain a powerful option for gene therapy. We are engineering vector and capsid designs to optimize targeting, expression control, and safety when combined with our delivery platforms.
Commitment to Safety
Safety is at the core of every Dawn Therapeutics programme. We use:
- Advanced biodistribution profiling to confirm targeted delivery.
- Long-term follow-up plans in preclinical and future clinical stages to track durability and late-onset effects.
- Alignment with global standards including ICH, GCP and GMP, appropriate to each stage of development.
Making Medicines Accessible
As programmes advance, we are committed to:
- Supporting early diagnosis through newborn screening advocacy and awareness with partners.
- Developing patient support programmes to guide families through evaluation and potential treatment when trials or approvals allow.
- Partnering globally to improve affordability and access in line with regulatory pathways and local health-system frameworks.
Our Therapeutic Philosophy
Precision First
We emphasize exact delivery over systemic exposure — enabling lower doses with improved safety margins.
Modular & Adaptable
Payloads are selected to suit disease biology, with flexibility to pivot across modalities as scientific evidence evolves.
Safety-Centric Design
All constructs and delivery systems are evaluated in parallel for tolerability, immunogenicity, and off-target risk.
Translational Readiness
Our development strategy is mapped for future IND-enabling validation, aligning payloads, targeting, and manufacturing early in the research cycle.
Pipeline Snapshot
| Modality | Lead Focus | Status / Goal |
|---|---|---|
| LNP-mRNA | IDUA expression in CNS & skeletal tissues | Preclinical optimization & biodistribution |
| RNAi | Gene knockdown in neurodegenerative or inflammatory settings | Feasibility studies & delivery evaluation |
| ASO | Transcript modulation in CNS or cartilage | Exploratory design & targeting validation |
| AAV | Long-term gene transfer for durable expression | Vector design & safety assessment |
All programmes are investigational and not approved for clinical use. Inclusion in this list does not guarantee a pipeline product or human trial.
Recent Advances in Our Medicines
Stay informed on our latest clinical milestones, regulatory updates, and real-world impact stories.
