Advancing the Frontier of Genetic Medicine (Preclinical Stage)
At Dawn Therapeutics, our R&D efforts center on development of gene- and RNA-based delivery systems with precision targeting to brain and bone/cartilage — aimed at rare genetic disorders and difficult-to-treat diseases with unmet medical needs. All programmes are currently in preclinical development.
Our Ethical & Scientific Commitment
We uphold rigorous standards of transparency, data integrity, and patient-centered design. Our R&D roadmap is steered by scientific validation, regulatory alignment, and long-term societal value.
Our Core Expertise
Precision RNA Therapeutics
- Early-stage development of siRNA, saRNA, mRNA, and antisense modalities for central nervous system and musculoskeletal targets
- Exploration of aptamer- and ligand-mediated targeting to improve tissue selectivity and delivery efficiency
Gene Therapy & Editing
- Lentiviral, AAV, and genome-editing approaches under evaluation for optimized safety and efficacy
- Development of scalable vector manufacturing compatible with future regulatory standards
Targeted Delivery Innovation
- Proprietary tissue-targeting platforms for the brain and cartilage/bone compartments
- Multi-modal delivery strategies and investigational imaging guidance to maximize localization and minimize off-target exposure
Clinical Development
- Design of translational pathways and anticipated protocols for first-in-human studies
- Regulatory planning and CRO/academic partnerships to support IND-enabling work and future clinical programs
Why Our R&D Is Distinct
Focused Mission:
We prioritize rare genetic brain and skeletal disorders and diseases with unmet therapeutic needs.
Technological Leverage
Our delivery systems are designed to enhance existing therapies by improving concentration at the target tissue and reducing systemic toxicity.
Rigorous Validation Process
Each step is governed by Go/No-Go milestones, biomarker-driven decisions, and best-in-class scientific standards.
Collaborative Ecosystem:
We partner with academic institutions, biotech companies, regulatory experts, and patient advocates to accelerate development.
Therapeutic Areas of Focus
Our R&D pipeline spans 21 disease areas with transformative potential
Rare CNS Disorders
- • Huntington's disease
- • Parkinson's disease
- • Sanfilippo syndrome
Musculoskeletal
- • Huntington's disease
- • Parkinson's disease
- • Sanfilippo syndrome
Genetic Syndromes
- • Hurler syndrome (MPS I)
- • Hunter syndrome (MPS II)
- • Sly syndrome (MPS VII)
Global Access
- • Autism spectrum disorder
- • Developmental delays
- • Cognitive disorders
Highest Standards
- • NAFLD & NASH
- • Type 2 diabetes
- • Metabolic syndromes
Additional Areas
- • Oncology
- • Immunology
- • Ophthalmology
Collaborate With Us
We welcome collaborations, licensing opportunities, and joint research ventures with partners who share our vision of delivering life-changing therapies to patients worldwide.
