01
Programme design
Refinement of core programme architecture, target rationale, and development assumptions to support a coherent preclinical plan.
Pipeline
Investing in Next-Generation Precision Delivery Programmes
Dawn Therapeutics is building a preclinical pipeline anchored by a lead Hurler syndrome programme and supported by a dual-platform delivery strategy. Our pipeline is designed to progress through milestone-based scientific, manufacturing, and regulatory decision gates, with a focus on translational readiness and platform leverage across future indications.
Explore pipeline
01 / Our lead programme
A focused pipeline built around DAWN-01
Dawn Therapeutics is advancing a disciplined, preclinical pipeline led by DAWN-01, our lead programme focused on Hurler syndrome (MPS-1H). The programme is designed to align precision delivery science with scalable development planning, with a clear emphasis on translational readiness.
- Indication
- Hurler syndrome (MPS-1H)
- Therapeutic cargo
- Functional IDUA gene
- Delivery platform
- In vivo stealth lentiviral vector
- Development stage
- Preclinical
02 / The path forward
DAWN-01 development roadmap
Five decision gates.
One focused programme.
02
Development framework
Preclinical optimisation and characterisation
Advance preclinical work to strengthen design quality and generate programme-supporting evidence.
Timelines and milestones shown on this page are presented as a development framework and may be updated as preclinical work progresses.
03 / Science into development
Preclinical workstreams for DAWN-01
02
Preclinical characterisation
Preclinical studies designed to characterise performance, tissue delivery behaviour, and key translational signals relevant to programme progression.
03
Safety-oriented planning
Early safety-focused design and risk assessment integrated into the preclinical pathway to support disciplined decision-making.
04
Manufacturing readiness
Development planning for scalable production strategy, process consistency, and future CMC alignment as the programme matures.
05
Translational modelling
Use of structured data and modelling workflows to improve programme design quality and support evidence-based milestone decisions.
06
Milestone governance
Stage-gated progression with defined review points to maintain focus, resource discipline, and partnership readiness.
04 / The reason we advance
Why DAWN-01 matters
High unmet need in MPS-1H
Hurler syndrome (MPS-1H) remains a severe rare genetic disease area where improved treatment strategies and delivery approaches are urgently needed.
Patients & familiesThe next conversation
Connect with Dawn Therapeutics
If you are interested in DAWN-01, Dawn can share additional programme information through the appropriate channel for partnership, diligence, or strategic discussions.
DAWN-01 is a preclinical programme. This page is for company, partnership, and investor information.
Discover more