Clinical Trials
Pioneering Tomorrow's Treatments Through Rigorous Clinical Research
At Dawn Therapeutics, our mission to deliver life-changing gene and RNA-based therapies is grounded in robust, ethical, and scientifically rigorous clinical trials. We conduct and support studies that bring innovative treatments from the laboratory into the hands of patients who need them most.
Our Clinical Focus Areas
Our clinical programmes focus on rare genetic disorders, neurological diseases, musculoskeletal conditions, and metabolic syndromes, with precision delivery systems.
Neurological
Diseases
- ALS/Motor Neuron Disease
- Huntington's Disease
- Parkinson's Disease
- Autism Spectrum Disorder
Rare Genetic
Disorders
- Hurler Syndrome (MPS I)
- Hunter Syndrome (MPS II)
- Lysosomal Storage Disorders
- Metabolic Syndromes
Musculoskeletal
- Osteoarthritis
- Rheumatoid Arthritis
- Bone Disorders
- Metabolic Syndromes
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.
Diverse Patient Populations
Evaluate safety and efficacy across different demographics, ensuring treatments work for everyone.
Optimized Therapeutics
Fine-tune dosing, delivery, and formulation for maximum therapeutic benefit and minimal side effects.
Regulatory Evidence
Generate regulatory-grade evidence to secure approvals from global health authorities.
Expanded Access
Provide potentially life-saving treatments for patients with few or no alternatives.
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-Centred Design
We work closely with patients, families, and advocacy groups to design trials that minimize burden and maximize access.
- Flexible visit schedules
- Remote monitoring capabilities
- Multilingual communication
- Cultural sensitivity protocols
Cutting-Edge
Methodologies
Advanced trial designs and digital health tools accelerate decision-making and enhance patient engagement.
- Adaptive trial designs
- Biomarker-driven recruitment
- Digital health tools
- Real-time data collection
Global Regulatory
Compliance
Full compliance with ICH-GCP guidelines and proactive collaboration with international health authorities.
- 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
If you or your loved one is interested in participating in a clinical trial, we’re here to help you navigate the process with compassion and clarity.
Find Eligible Trials
Search our patient portal for trials matching your condition and location.
Personalized Support
Our clinical trials support team provides one-on-one assistance.
Safety First
Comprehensive informed consent and ongoing safety monitoring.
- trials@dawntherapeutics.com
- +44 (0)XXX XXX XXXX
For Patients & Families
We welcome collaboration with physicians, clinics, and research institutions worldwide to advance breakthrough treatments for patients in need.
Refer Patients
Connect eligible patients with potentially life-changing treatments.
Join Our Network
Become part of our global investigator network.
Training Materials
Access protocols and comprehensive training resources.
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
We are committed to the highest standards of scientific integrity, ethical conduct, and transparent communication throughout our clinical research program.
Trial Registration
All trials registered on ClinicalTrials.gov and regional registries
View Registrations
Ethical Standards
Full compliance with ICH-GCP and institutional review boards
Ethics Framework
Results Publication
Commitment to publishing all results, positive and negative
Publications
Patient Safety
Independent safety monitoring and adverse event reporting
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