Our Vision
Deliver curative RNA and gene medicines precisely where they’re needed in rare and common diseases.
Dawn Therapeutics integrates tissue-targeted delivery, advanced RNA/gene modalities, and rigorous translational science to tackle CNS, lysosomal, and musculoskeletal diseases that remain underserved by conventional therapies
The Dawn Platform
One platform, many programmess—built for precision and scale.
Tissue-targeted vectors
Engineered for CNS, joint/cartilage, muscle, and liver with tunable tropism and expression profiles.
Modality flexibility
LNP-mRNA, RNAi/ASO, AAV gene transfer (in the near future), and gene-editing strategies selected per disease biology.
Design → Make → Test loop
Ability to design, rapid prototyping, automated analytics, and iterative optimization.
Manufacturability by design
Dawn has technology expertise to help design CMC rules from day one.
Modalities We Use
The right tool for each target.
LNP-mRNA
Rapid, repeat-dose capable expression when transient production is preferred.
RNAi & ASO
Post-transcriptional silencing for toxic gain-of-function genes (e.g., neurodegeneration).
AAV Gene Transfer (in Progress)
Durable expression of therapeutic genes for LSDs and select CNS indications.
Gene Editing (in the Near Future):
Precise correction or knock-down with safety-first controls.
Future Plans: Translational Blueprint
Bench to bedside—disciplined and data-driven.
Disease modelling
Human iPSC systems and validated in vivo models for efficacy and dose projection.
PK/PD & biomarkers
qPCR, ddPCR, proteomics, neurofilament/light chains, GAG reduction, imaging readouts.
Dose translation
Allometric scaling with exposure–response modelling to set first-in-human starting doses.
Clinical-grade analytics
Qualified assays for vector genomes, potency, impurities, and durability.
CMC & Manufacturing
Our Future Plan is to include Quality and scalability from the start.
1. Process
🔹 Scalable upstream production
🔹 Robust purification
🔹 Platform analytics (capsid ratio, empty/full)
2. Release & Stability
🔹 ICH-aligned testing
🔹 Forced-degradation studies
🔹 Real-time stability
3. Supply Chain
🔹 Redundant raw-material vendors
🔹 Tech-transfer-ready documentation for global sites
4. Sustainability
🔹 Process intensification
🔹 Reduce footprint & cost of goods
Clinical Development Philosophy
Patient-centred, biomarker-anchored, regulator-ready.
At Dawn Therapeutics, our development strategy is built on scientific precision and ethical responsibility, ensuring that every programme moves toward the clinic with translational clarity and purpose. Our philosophy aligns innovation with patient impact and global regulatory standards.
Our Focus Areas
- Rare Genetic Disorders (Brain and Bone):
Our primary focus is on rare, life-limiting genetic diseases where current therapeutic options are minimal or non-existent. Dawn’s lead in-vivo programme for Hurler syndrome (MPS-I H) demonstrates the full translational concept — from gene delivery to tissue-specific expression — and will be followed by additional candidates targeting both neurological and musculoskeletal manifestations of rare genetic disorders. - Diseases with Unmet Medical Need:
We prioritise conditions for which no effective therapies exist, applying our delivery platforms to enable new treatment possibilities for neurodegenerative and musculoskeletal diseases with substantial patient burden and limited clinical solutions. - Enhancement of Existing Therapeutics:
Our precision delivery systems are designed to make existing therapeutics safer, more targeted, and more effective, reducing off-target toxicity while increasing tissue-specific efficacy. These platforms can be adapted to improve a wide range of nucleic acid and small-molecule therapies.
Our Guiding Principles
- Adaptive and Data-Driven Trial Design
- Seamless, flexible designs that allow efficient progression between study phases once regulatory approval for human trials is granted.
- Incorporation of response-adaptive methodologies and real-time analytics to refine dosing and safety assessments.
- Precision Eligibility
- Genotype- and phenotype-based stratification to identify subpopulations most likely to benefit.
- Integration of preclinical biomarker data to define evidence-based inclusion criteria.
- Meaningful and Quantifiable Endpoints
- Endpoints reflecting real functional outcomes, quality of life, and validated disease-specific biomarkers.
- Developed in collaboration with key opinion leaders and patient representatives to ensure clinical relevance.
- Access and Equity
- Commitment to inclusivity by localising study materials and site engagement for language, culture, and geography.
- Development of frameworks to support equitable participation through logistical and travel assistance for eligible participants.
Data Science, AI & Analytics
Faster insights, smarter design
- Sequence & capsid design: ML-guided libraries to optimize tropism and manufacturability.
- Imaging & digital endpoints: Quantify motor function, gait, and cognition with validated digital tools.
- Real-world evidence: Registries and natural-history cohorts to contextualize effect size and durability.
Safety, Bioethics & Compliance
- Independent safety advisory board and DSMBs.
- Long-term follow-up frameworks for durability and delayed events.
- Informed consent designed for clarity and accessibility.
- Adherence to global guidelines (ICH, GCP, GMP) and local regulations.
IP & Partnerships
1. Collaborate, don’t duplicate
🔹 Balanced portfolio of proprietary vectors, delivery chemistries, and manufacturing know-how.
🔹 Academic, foundation, and biotech partnerships to accelerate programs and expand access.
🔹 Open to co-development, regional licensing, and platform collaborations.
Publications & Resources
🔹 Explore our latest conference posters, peer-reviewed publications,
🔹, and technical white papers covering delivery,
🔹 biomarkers, and clinical methodology.
How We Choose Indications
🔹 High unmet need and clear genetic driver.
🔹 Feasible delivery route with quantifiable biomarkers.
🔹Ability to design strong translational package enabling rapid, responsible trials.
