Transforming Treatment Across Neurological, Musculoskeletal, and Rare Genetic Diseases
At Dawn Therapeutics, we are dedicated to developing RNA and gene therapies that deliver life-changing impact for patients living with rare, complex, and currently untreatable conditions.
Our proprietary tissue-targeted delivery system enables precise therapeutic delivery to the brain, cartilage, and bone — unlocking new possibilities in neurological disorders, musculoskeletal diseases, and rare genetic syndromes. We focus on diseases where current treatments are ineffective, symptomatic only, or entirely unavailable, driving innovation from preclinical discovery through to clinical development.
Disease Area Grid
Explore the therapeutic areas we are actively researching and developing. Click each condition to learn more about our approach, clinical programmes, and partnerships.
Central Nervous System (CNS) Disorders
Hurler Syndrome
MPS I – Lysosomal storage disorder
Targeted vectors delivering functional gene copies to brain and connective tissues.
Autism Spectrum Disorder
Neurodevelopmental condition
Gene modulation therapies targeting molecular pathways in ASD.
Alzheimer's Disease
Progressive neurodegenerative disease
RNA therapeutics reducing protein accumulation and neuroinflammation.
Addiction Disorders
Chronic relapsing brain conditions
RNA modulation of neural pathways and reward responses.
Multiple Sclerosis
Autoimmune CNS disease
Gene therapies targeting myelin repair and immune regulation.
Parkinson's Disease
Neurodegenerative movement disorder
Gene therapy restoring dopamine production and neuron protection.
Musculoskeletal Disorders
Osteoarthritis
Degenerative joint condition
Localized gene therapy promoting cartilage regeneration.
Rare Genetic Disorders
Hunter Syndrome
MPS II – X-linked lysosomal disorder
Tissue-targeted delivery to CNS and connective tissues.
Other Rare Disorders
Expanding research portfolio
Platform adaptability for rapid application to new indications.
Our Approach
At the heart of our disease area strategy is our next-generation targeted delivery platform
Precision Tissue Targeting
Maximise efficacy and minimise off-target effects through precise delivery systems.
Payload Flexibility
From messenger RNA to CRISPR-based gene editing tools, adaptable to diverse therapeutic needs.
Scalable Manufacturing
Seamless transition from laboratory research to clinical application and commercial scale.
Ready to Transform Treatment Together?
Interested in collaborating or learning more about our work in these disease areas? Explore opportunities to partner with us in advancing breakthrough therapies.