Our Vision
Deliver curative RNA and gene medicines precisely where they’re needed.
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 programs—built for precision and scale.
Tissue-targeted vectors
Engineered for CNS, joint/cartilage, muscle, and liver with tunable tropism and expression profiles.
Modality flexibility
AAV gene transfer, LNP-mRNA, RNAi/ASO, and gene-editing strategies selected per disease biology.
Design → Make → Test loop
In-house design, rapid prototyping, automated analytics, and iterative optimization.
Manufacturability by design
CMC built in from day one for consistent quality and global scalability.
Modalities We Use
The right tool for each target.
AAV Gene Transfer
Durable expression of therapeutic genes for LSDs and select CNS indications.
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).
Gene Editing (where appropriate):
Precise correction or knock-down with safety-first controls.
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
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.
1. Adaptive trials: 
Seamless phase designsresponse-adaptive randomization where applicable.
2. Precision eligibility: Genotype/phenotype stratification and enrichment for those most likely to benefit.
3. Meaningful endpoints: Function quality-of-life and disease-specific biomarkers selected with KOL input.
4. Access & equity: Sites and materials localized for language culture, and geography; travel support options.
Data Science, AI & Analytics
- 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.
🔹Strong translational package enabling rapid, responsible trials.