Advancing Targeted Genetic Medicines for Neurological, Musculoskeletal, and Rare Genetic Disorders
At Dawn Therapeutics, our focus lies in understanding and addressing rare genetic diseases, neurological disorders, and musculoskeletal conditions through targeted delivery of gene and RNA therapeutics.
All programmes described below are investigational and preclinical. They represent areas of ongoing or exploratory research aimed at future translational development.
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 Tiles
Hurler Syndrome (MPS I H)
Lead preclinical programme delivering the IDUA gene to both brain and skeletal tissues using an in vivo lentiviral platform.
Autism Spectrum Disorder (ASD)
Preclinical interest in molecular and synaptic modulation strategies leveraging our brain-penetrant delivery platform.
Alzheimer’s Disease
Preclinical studies exploring RNA-based modulation of neuroinflammatory and amyloid pathways using our CNS-targeted delivery platform.
Parkinson’s Disease
Exploratory research assessing delivery of neuroprotective genes and RNA constructs to dopaminergic regions of the brain.
Amyotrophic Lateral Sclerosis (ALS)
Early exploratory work focused on motor-neuron targeting and neuroinflammation using CNS-directed gene-delivery systems.
Friedreich Ataxia
Exploration of vector delivery strategies for mitochondrial and iron-binding protein regulation, currently at the conceptual stage.
Huntington’s Disease
Platform feasibility studies for RNA interference and gene-silencing approaches to reduce toxic protein accumulation in neural tissue.
Spinal Cord Injury
Preclinical evaluation of regenerative-gene and RNA payload delivery to support tissue recovery and axonal repair.
Osteoarthritis
Experimental delivery of RNA and gene constructs to cartilage and bone to promote repair and reduce inflammatory signalling.
Musculoskeletal Disorders (General)
Investigation of platform-specific targeting to skeletal tissue for inherited and degenerative musculoskeletal diseases.
Duchenne Muscular Dystrophy (DMD)
Feasibility studies for targeted gene delivery to muscle tissue; early research phase only.
Other Rare CNS / Musculoskeletal Conditions
Exploratory stage research evaluating cross-applicability of our tissue-targeted delivery technologies to additional rare disorders.
Hunter Syndrome (MPS II)
Exploratory research evaluating potential platform adaptation for enzyme-deficiency disorders affecting CNS and bone.
Sanfilippo Syndrome (MPS III)
Early discovery efforts investigating CNS-directed delivery for neurological forms of mucopolysaccharidosis.
Morquio Syndrome (MPS IV)
Concept-stage development focused on skeletal manifestations of lysosomal enzyme deficiency.
Maroteaux–Lamy Syndrome (MPS VI)
Evaluation of targeted vector strategies for connective-tissue and skeletal involvement.
Sly Syndrome (MPS VII)
Exploratory work assessing feasibility of cross-corrective enzyme expression via targeted gene delivery.
Natowicz Syndrome (MPS IX)
Research interest in ultra-rare forms of MPS to assess platform adaptability for multi-systemic disease models.
Our platforms are designed for precise, tissue-specific delivery of therapeutic payloads — with the brain and musculoskeletal system as our primary focus.
Dawn Therapeutics’ approach enables modular adaptation across multiple disease areas, while maintaining scientific rigour, ethical responsibility, and regulatory readiness.
All programmes listed are investigational and currently in preclinical development or research and development. Inclusion of a disease area does not imply therapeutic availability or active clinical trials.
