DAWN-01
Hurler syndrome (MPS I-H)
A clear starting point: develop a genetic medicine for a rare disease with significant remaining needs.
Explore the programmeInvestors
Back the science.
Build the next dawn.
A focused rare disease programme. Two complementary delivery platforms. A disciplined path from laboratory evidence towards clinical readiness.
Explore the investment storyRequest investor materials01 / The idea, made simple
Good medicine needs
a way to get there.
The challenge is delivery. Dawn’s ambition is to help genetic medicines reach places in the body that are difficult to treat.
Explore the journey · 1 of 3
Genetic instructions
Genetic instructions
Think of a genetic medicine as a set of instructions for a cell. For DAWN-01, the aim is to deliver a working IDUA gene—the instructions for an enzyme involved in breaking down certain complex sugars.
Explore the science02 / One focus. Wider potential.
A programme to advance.
Platforms to build on.
Dawn combines a lead medicine in development with delivery technologies that could support additional programmes and partner projects.
Route 01
Develop the lead programme
Build evidence, manufacturing capability and the regulatory package for DAWN-01.
Route 02
Extend the delivery platforms
Explore additional applications as tissue-targeting evidence develops.
Route 03
Work with partners
Pursue licensing, co-development and specialist execution collaborations.
These are potential development and partnering routes. All programmes remain investigational; future outcomes and commercial agreements are not assured.
03 / Evidence with context
Understand the signal.
Ask the next question.
Select a finding to see what it means—and what still needs to be established.
Preclinical research · conceptual illustration
A biological signal in the lead programme.
GAGs are complex sugars that can accumulate in Hurler syndrome. Dawn reports an average 42% reduction in toxic GAG accumulation in preclinical studies. This is an animal-model result, not a demonstrated benefit in people.
Read the sLV evidence04 / What the next stage enables
Capital behind
clear decision points.
A planned 24-month path towards regulatory filing readiness. Each workstream is designed to answer a development question.
Months01–03
Planned workstream
Manufacturing
Set up GMP manufacturing for DAWN-01, including technology transfer and pilot batch production.
Formal Go/No-Go gates support disciplined development. Timing is subject to funding, study outcomes and regulatory review. Filing readiness does not mean regulatory approval.
05 / A considered conversation
See the detail.
Meet the people.
We welcome investors and strategic partners who want to understand the science, execution plan and delivery-platform opportunity.
Scientific evidence
Review the lead programme and original preclinical findings.
Intellectual property
Discuss portfolio detail and controlled access for diligence.
Scientific leadership
Meet the clinicians and researchers behind Dawn.
Begin an investor discussion.
Request the investor materials and discuss the next development stage.
Dawn Therapeutics is a preclinical company. This information supports investor and strategic discussions and is not an offer to sell securities.
Start a conversation
Progress begins
with connection.
Tell us what you have in mind. Prepare an email to the right team at Dawn.
info@dawntherapeutics.comClear answers
Questions, answered.
What is Dawn’s lead programme?
DAWN-01 is an investigational preclinical programme for Hurler syndrome (MPS I-H). It uses a stealth lentiviral delivery platform designed to carry a functional IDUA gene.
Has DAWN-01 been approved for patients?
No. DAWN-01 is in preclinical development and is not an approved treatment. Further development depends on funding, study outcomes and regulatory review.
What does the 24-month plan cover?
The planned work includes manufacturing transfer, dose-finding, toxicology, regulatory advice and preparation of a CTA/IND submission package. It is a development framework, not a guaranteed approval timeline.
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