Advancing Breakthrough Therapies Through Strategic Partnerships
From Dawn of Dreams to Dawn of Discoveries.
Collaborating with innovators, healthcare providers, and advocacy groups to accelerate the delivery of transformative RNA and gene therapies
Who We Are
Dawn Therapeutics is a UK-based biotechnology company founded in November 2019, born from scientific and clinical leadership committed to solving some of the most challenging neurological and skeletal diseases. (UK company registration: November 2019) We are dedicated to creating precision delivery platforms for genetic medicines to reach the brain and bone, and translating them toward clinical readiness.
Our Mission
To enable safe, effective, and accessible genetic therapies by building targeted delivery systems that overcome biological barriers. We aim to address rare genetic disorders, neurological conditions with high unmet need, and opportunities to enhance existing treatments through precise targeting.
Our Vision
To transform the lives of patients and families affected by CNS disorders, lysosomal storage diseases, and musculoskeletal conditions through scientific innovation, ethical responsibility, and global collaboration.
Our Core Focuses
- Lead Indication — MPS-I H (Hurler syndrome): Our flagship programme, DAWN-01, is an in vivo lentiviral vector designed to deliver the IDUA gene to both central nervous system and skeletal tissues. This is our most advanced asset and serves as the proof concept for our dual-delivery strategy.
- Deliberate Strategic Expansion: After validating our core concept in MPS-I H, we will extend our delivery platforms to additional rare genetic, neurological, and musculoskeletal diseases—especially those lacking effective therapies.
- Platform-enabled Therapeutic Improvement: Beyond de novo therapy development, we aim to harness our precise delivery technology to improve existing therapeutics—making them safer, more effective, and less toxic by confining action to the target tissue.
Our Expertise
Dawn Therapeutics brings together leaders from:
- Gene Therapy & RNA Therapeutics Development
- Neurology, Genetics, and Musculoskeletal Research
- Regulatory Affairs & Clinical Trial Management
- Manufacturing & Commercialisation
Our proprietary delivery technologies are designed for brain, cartilage, muscle, and liver targeting, unlocking treatment potential in diseases previously considered untreatable.
Our Story
Founded with the vision to bridge the gap between cutting-edge science and patient impact, Dawn Therapeutics emerged from a team of scientists, clinicians, and entrepreneurs united by a shared goal — making targeted genetic therapies a reality for the people who need them most.
Our Technology Platforms
Advanced delivery systems engineered to overcome biological barriers and deliver therapeutic payloads with precision.
Brain-Targeting Platform
Capable of penetrating the blood–brain barrier and transporting DNA, RNA, or molecular cargos into neuronal and glial tissues.
Cartilage & Bone Targeting Platform
Engineered to deliver payloads into skeletal and joint tissues with precision, overlaid on musculoskeletal disease biology.
Payload Modality Agnosticism
Accommodates multiple therapeutic modalities (mRNA, RNAi, ASOs, gene transfer) enabling flexibility by disease.
Scalable Manufacturing & Regulatory Foresight
Strategies built for GMP adoption, quality systems, and regulatory alignment necessary for eventual clinical translation.
Guiding Principles & Philosophy
Our commitment to safety, transparency, and global impact guides every decision we make.
Safety by Design
We integrate safety and risk profiling at every step—biodistribution, immunogenicity, and toxicology planning.
Translational Rigor
Our pathway follows Go/No-Go decision gates, biomarker endpoints, and early regulatory engagement.
Ethics, Transparency & Accountability
We commit to scientific integrity, publish data transparently, and involve patient voices responsibly.
Collaborative Mindset
We partner with academia, biotech firms, and global stakeholders for innovation and progress.
Equitable Vision
Designing programs with affordability, localization, and inclusion across geographies.
Our Journey
From foundation to future — our roadmap to transforming genetic medicine.
2019 — Foundation
Dawn Therapeutics founded in the UK by its scientific and clinical founders.
Discovery — Platform Engineering
Platform engineering, payload concept validation, and delivery proof-of-concept.
Preclinical Development
Optimization, toxicology, biodistribution, and manufacturing planning.
Regulatory & IND Enabling
CMC development, GLP studies, and regulatory consultation.
Future Goals — Clinical Trials & Global Scale
Initiate first-in-human clinical trials, expand pipeline, and scale global access.
Our Team & Network
Composed of scientists, translational biologists, and regulatory strategists with deep experience in gene therapy, neurobiology, and musculoskeletal disease.
Commitment to Partners & Patients
We build trustworthy, transparent relationships with collaborators and patient communities. Even before clinical stages, we ensure forward progress under rigorous standards of safety and ethics.
We welcome inquiries from those who share our mission to translate precision genetic medicines into tangible global impact.
Get in Touch