Our Mission
Advancing Science, Changing Lives through collaborative research and innovative treatments
Accelerate Research
Fast-track innovative therapies from lab to clinic through cutting-edge research and development.
Empower Patients
Advocate and create global access to treatments for those affected by rare genetic disorders.
Collaborate Globally
Partner with researchers, biotech companies, and governments to drive medical breakthroughs.
Our Focus Areas
Targeting the most challenging unmet needs in medicine
Liver Cancer & Liver Failure
Including hepatocellular carcinoma and advanced cirrhosis
Metabolic Liver Diseases
NAFLD, NASH, and metabolic syndrome research
Rare Genetic Disorders
MPS, lysosomal storage diseases, and genetic syndromes
Neurological Disorders
Parkinson's, Alzheimer's, and Huntington's disease
First Gene Therapy Trials
Habib 4X Device
RNA Aptamer Systems
Global Partnerships
Our Approach
From Lab to Life – A comprehensive pipeline ensuring breakthrough treatments reach patients
Precision-Targeted Therapeutics
Developing treatments that target specific molecular pathways
Global Collaborations
Partnerships with leading institutions worldwide
Rigorous Clinical Standards
Maintaining the highest safety and efficacy standards
Equitable Access
Ensuring treatments reach patients regardless of location
We believe in the power of partnerships to deliver medical breakthroughs faster
Collaborating with academic institutions, biotechnology companies, and healthcare systems worldwide
Patient Stories & Advocacy
Real stories of hope, progress, and resilience
Accelerate Research
Living with MPS I, Emma's family found hope through our research programs and patient advocacy initiatives.
Michael's Recovery
After participating in our liver cancer gene therapy trial, Michael's life was transformed.
The Rodriguez Family
Finding community and support through our rare disease advocacy programs and research initiatives.
How You Can Help
Join us in bringing hope to those living with life-limiting conditions
Donate
Support research and clinical trials that can change lives
Partner
Collaborate with us to develop new therapies
Volunteer
Contribute your expertise to our advocacy programmes
News & Updates
Press Release
Dawn Therapeutics Announces New Preclinical Data for DTX-101
Publication
Targeted Lentiviral Vectors for CNS and Musculoskeletal Disorders
