Supporting Patients and Families Every Step of the Way
Our Commitment to You
At Dawn Therapeutics, patients are at the heart of everything we do. We understand the challenges of living with rare and complex diseases — and we are dedicated to:
- Providing accurate, understandable information about each condition we work on.
- Connecting families to clinical trials and support networks.
- Advocating for patient needs in research, healthcare policy, and global access.
Find Your Condition
We are developing treatments for over 20 rare and complex diseases. Click below to learn more about your condition:
- Central Nervous System Disorders (e.g., Alzheimer’s, Parkinson’s, Huntington’s, Autism Spectrum Disorder, Addiction Disorders).
- Lysosomal Storage Disorders (MPS I, II, III, IV, VI, VII, IX, and others).
- Musculoskeletal Disorders (Osteoarthritis, Duchenne Muscular Dystrophy, Friedreich’s Ataxia, ALS).
Understanding Gene and RNA Therapies
We know medical terminology can be confusing. Our goal is to make it simple:
Gene Therapy
Repairs or replaces faulty genes to treat or prevent disease
RNA Therapy
Uses messenger RNA (mRNA), antisense oligonucleotides (ASOs), or RNA interference (RNAi) to target disease at the molecular level.
Precision Delivery
Our technology sends the medicine directly to the affected tissues — brain, cartilage, muscle, or liver — for the greatest impact and fewer side effects.
Clinical Trials for Patients
Clinical trials are how new treatments become available.
- Offer screening to see if you qualify for one of our trials.
- Work with leading hospitals and specialists worldwide.
- Provide travel and accommodation support for eligible participants.
Support & Resources
Patient Advocacy Partners
Connecting you with charities and support groups.
Educational Materials
Condition-specific guides, videos, and FAQs
Counselling & Family Support
Access to genetic counsellors and therapists.
Our Promise
Transparency
We will always keep you informed about progress, risks, and benefits.
Compassion
Your voice matters in our decision-making.
Access
We aim to make our therapies available globally, not just in select markets.