Trinds Logo


Approach: Neuromuscular focused Contract Research Organization
Funding Impact: Clinical trial support for Biotech/Pharma from a patient-centric perspective

summit plc logo


Approach: Utrophin upregulation
Funding Impact: Enabled initiation of Phase 2 clinical trial

PTC logo

PTC Therapeutics

Approach: Stop codon read-through
Funding Impact: Conditional approval of Translanrna in EU

Biomarin Logo


Approach: Exon skipping
Funding Impact: Helped support clinical development of exon skipping drugs
Successful Exit: Prosensa was acquired by BioMarin in 2015


Approach: Statistical analysis of natural history studies
Funding Impact: Collaboration amongst biotech/pharma to share data and enrich clinical trial design

Nationwide Children’s Hospital

Approach: Exon skipping (Duplication exon 2 mutations)
Funding Impact: Advanced pre-clinical IND package enabling clinical trial for Duchenne boys