Clinical Trials
Pioneering Tomorrow’s Treatments Through Rigorous Clinical Research
At Dawn Therapeutics, we are committed to advancing our investigational therapies toward human studies, guided by ethical principles, scientific rigor, and patient-focused design. To date, no clinical trials have yet begun — our programmes remain in preclinical development as we prepare for future clinical initiation.
Our Clinical Focus Areas
We plan to pursue clinical evaluation in three core domains, using our precision delivery platforms:
Neurological Diseases
- with high unmet medical need
Rare Genetic
Disorders
- especially in brain and bone tissues, where current treatment options are extremely limited
Musculoskeletal and Joint Disorders
- including conditions with minimal or no effective treatment
Precision Delivery
- CNS Barrier Crossing
- Targeted Cell Delivery
- Tissue-Specific Vectors
- Biomarker Monitoring
Why Clinical Trials Matter
Clinical trials are the bridge between scientific discovery and real-world medical impact. They enable us to transform innovative research into life-changing treatments.
Translating Discovery to Care:
They serve as the critical bridge from laboratory innovation to patient care, enabling assessment of safety, efficacy, and dosing in humans.
Building the Evidence Base:
They provide the rigorous data required for regulatory approval and medical validation.
Learning from Diversity:
They allow us to study diverse patient populations and refine therapeutic approaches accordingly.
Pathway to Participation:
They offer a route for patients and clinicians to engage in cutting-edge research—once trials begin.
Our Approach to Clinical Trials
We combine patient-centered design with cutting-edge methodologies and global regulatory compliance to deliver world-class clinical research.
Patient-Centered Design
We engage patients, families, and advocacy groups early to shape trial protocols that minimize burden (e.g. flexible visits, remote monitoring, multilingual materials).
- Flexible visit schedules
- Remote monitoring capabilities
- Multilingual communication
- Cultural sensitivity protocols
Adaptive & Efficient Methodologies
Future trials will apply designs such as seamless phase progression and response-adaptive allocation as appropriate, informed by biomarker date.
- Adaptive trial designs
- Biomarker-driven recruitment
- Digital health tools
- Real-time data collection
Regulatory Partnership
We intend to collaborate proactively with regulatory bodies (FDA, EMA, MHRA, and others) to align trial design with global compliance expectations.
- FDA (United States)
- EMA (European Union)
- MHRA (United Kingdom)
- Regional authorities worldwide
Our Approach to Clinical Trials
We are advancing multiple candidates through Phase I to Phase III trials, targeting devastating diseases with unmet medical needs.
Neurological Disorders
- Huntington's Disease
- Parkinson's Disease
- ALS/Motor Neuron Disease
- 12 sites globally
Rare Genetic Disorders
- Hurler Syndrome (MPS I)
- Hunter Syndrome (MPS II)
- Lysosomal Storage Disorders
- 8 specialized centers
Developmental Disorders
- Autism Spectrum Disorder
- Intellectual Disabilities
- Neurodevelopmental Delays
- 6 pediatric centers
Metabolic Disorders
- Metabolic Syndromes
- Enzyme Deficiencies
- Mitochondrial Disorders
- 4 metabolic centers
Precision Delivery
- CNS Targeting Vectors
- Tissue-Specific Delivery
- Biomarker Systems
- Platform technology
For Patients & Families
While there are no active clinical trials currently open, we are planning for future human studies.
If you or a loved one are interested in staying informed:
Join our interest registry:
we will notify you when trials begin recruitment
Contact us:
contact our team for updates or to learn more about our programmes (e.g. trials@dawntherapeutics.com)
Explore More:
Explore resources from trusted patient organizations for guidance in the meantime
- trials@dawntherapeutics.com
- +44 (0)XXX XXX XXXX
For Healthcare Professionals & Investigators
We welcome collaboration with physicians, clinics, and research institutions worldwide to advance breakthrough treatments for patients in need.
Collaborative Partnerships:
We welcome engagement with clinics, physicians, and research institutions ahead of trial initiation.
Investigator Network:
Clinical investigators may express interest in collaborating through our Global Investigator Network.
Training & Support:
Comprehensive materials, trial protocols, and ongoing assistance will be provided to ensure high-quality execution.
Collaboration Benefits
- Access to cutting-edge therapies for your patients
- Comprehensive protocol training and ongoing support
- Contribution to advancing medical science
- Publication opportunities and research collaboration
- clinicalops@dawntherapeutics.com
Transparency, Ethics & Scientific Integrity
We are committed to the highest standards of scientific integrity, ethical conduct, and transparent communication throughout our clinical research program.
Public Registration:
All future trials will be listed on ClinicalTrials.gov and relevant registries once recruitment begins.
View Registrations
Global Compliance:
We adhere to ICH-GCP principles with independent safety monitoring and institutional ethics oversight.
Ethics Framework
Responsible Data Sharing:
Results—positive or negative—will be published and communicated transparently to advance scientific progress.
Publications
Ethical Accountability:
Every programme is guided by scientific integrity, respect for participants, and continuous dialogue with regulators, clinicians, and patient communities to uphold the highest ethical standards.
Safety Protocols
Our Commitment to Scientific Integrity
Open Science
- Pre-registration of study protocols
- Data sharing with research community
- Collaborative research partnerships
Patient Rights
- Comprehensive informed consent
- Right to withdraw at any time
- Privacy and data protection
Quality Assurance
- Independent monitoring and auditing
- Regulatory compliance verification
- Continuous improvement processes
Global Clinical Network
Our international network of clinical sites and research partners enables us to conduct trials across diverse populations and healthcare systems.
45+
Clinical Sites
12
Countries
850+
Patients Enrolled
25+
Investigator Partners
- North America
18 sites
- Europe
15 sites
- Asia-Pacific
8 sites
- Other Regions
4 sites
Transforming Research Into Real-World Cures
Dawn Therapeutics – Through precision and compassion, we’re advancing breakthrough treatments that offer hope to patients and families worldwide.
For Patients
Access potentially life-changing treatments through our clinical trials.
- trials@dawntherapeutics.com
- +44 (0)XXX XXX XXXX
For Healthcare Professionals
Join our global network of clinical investigators and research partners.
- clinicalops@dawntherapeutics.com
- Global Investigator Network
For Patients
Access potentially life-changing treatments through our clinical trials.
- partnerships@dawntherapeutics.com
- Strategic Partnerships
