At Boston Children’s Hospital, we’re redefining what’s possible for children with rare genetic diseases. The TREATMENT Program integrates world-class research and clinical excellence to move discoveries from the lab to life-saving therapies.
Our focus areas include advanced genomic testing, disease mechanism discovery, and therapy development using gene editing, ASOs, and RNAi.
Through collaborative clinical trials, we bring hope and healing to children around the world.
The TREATMENT Program: A new model for rare disease research
The Targeted Research and Exploration Advancing Trial Models, Editing, and Next-generation Therapies (TREATMENT) Program is our answer to a critical challenge: accelerating the pace of discovery for rare and complex genetic conditions.
This collaborative program brings together:
- Experts in genomics, gene therapy, RNA-based treatments, and gene editing
- Data scientists, model developers, and clinical trial specialists
- Families who generously share insights, data, and lived experience
Together, we’re reshaping the path from diagnosis to treatment — not just for one condition, but for many.
Research that drives real change
Boston Children’s leads more pediatric clinical trials than any other hospital. Through the TREATMENT Program, we’re focused on:
Diagnosing rare conditions with speed and accuracy
We use advanced genomic testing and data science to pinpoint the causes of complex disorders and identify patients who may benefit from emerging therapies.
Building models to study disease and test treatments
We create cell lines, stem cells, and mouse models that mimic human disease — giving researchers the tools they need to understand disease mechanisms and test new therapies in a controlled, safe environment.
Developing and testing new therapies
We explore a range of therapeutic strategies, including:
- Drug repurposing (testing FDA-approved drugs for new uses)
- Antisense oligonucleotides (ASOs)
- Gene knockdown (siRNA, RNAi)
- Gene addition and gene editing (base editing, prime editing)
Each approach is tailored to the disease’s genetic cause and the age window when treatment is most likely to be effective.
Leading clinical trials that put families first
When treatments show promise, we lead clinical trials that prioritize family-centered care, safety, and meaningful outcomes. We work closely with caregivers to ensure measures reflect what truly matters in a child’s daily life.
Powered by collaboration
No single institution can solve rare disease alone. That’s why Boston Children’s is building a connected network of scientists, families, industry partners, and clinical collaborators all working toward a shared goal: making tomorrow’s cures a reality today.
Why it matters
More than 100 million children worldwide are affected by rare genetic diseases. Many go undiagnosed. Most have no treatments. That’s why our work in research and discovery is urgent — and why families around the globe turn to Boston Children’s for leadership, partnership, and hope.