Every year on Rare Disease Day, patients, families, and researchers join forces to draw attention to the unmet needs of those living with rare diseases. In honor of Rare Disease Day 2025, we shine a spotlight on Duchenne Muscular Dystrophy (DMD)—a severe muscle-wasting disorder—and highlight how next-generation therapies, namely PMO (phosphorodiamidate morpholino oligomers) and PPMO (peptide-conjugated PMOs), are creating new hope.
Understanding DMD: A Rare and Devastating Condition
DMD arises from mutations in the dystrophin gene, a protein crucial for maintaining muscle health. Without functional dystrophin, muscles weaken over time, leading to severe mobility challenges and a life expectancy often below 40. Affecting approximately 1 in every 3,500 to 5,000 male births, DMD underscores a critical need for more effective treatments.
Since 2016, several DMD therapies have received FDA approval, marking transformative steps for a disease once considered untreatable. Among the most promising approaches are antisense oligonucleotides (ASOs), specifically PMOs. These molecules are more stable than natural RNA, enabling them to better withstand degradation and potentially restore partial dystrophin production.
PMOs act by targeting faulty sections of mRNA, essentially “skipping” problematic exons so cells can produce a shortened but still functional form of dystrophin. While this approach shows promise, challenges remain, particularly in ensuring the drug effectively reaches muscle tissue. One promising strategy to enhance PMO therapy involves conjugating the PMO with a short cell-penetrating peptide (CPP), creating what’s known as a PPMO. These short peptides help transport the PMO across the cell membrane, potentially improving tissue uptake and reducing the required dose.
WuXi TIDES: Accelerating PMO/PPMO Therapies
Developing PMO and PPMO treatments requires specialized expertise in advanced chemistry and process optimization—from monomer synthesis to large-scale manufacturing and analytical validation. WuXi TIDES offers a fully integrated CRDMO platform that brings these capabilities under one roof, enabling customers to take a therapy from concept to clinical trials more swiftly and cost-effectively.
When the WuXi TIDES team was presented with a complex PPMO project, multiple teams worked in parallel to optimize synthesis, conjugation, formulation, and analytical methods. Within just 11 months, WuXi TIDES helped advance this candidate to IND (Investigational New Drug) filing.
Alongside faster development, our ongoing process improvements have reduced PMO manufacturing costs by 60% over the past three years. This cost efficiency not only accelerates clinical progress but also paves a clearer path for commercial viability, ultimately benefiting patients.
As we mark Rare Disease Day 2025, WuXi TIDES remain dedicated to delivering life-changing treatments—faster and more reliably—to those who need them most. Beyond DMD, we are actively supporting more than 20 innovative therapies across a range of complex modalities, expanding possibilities for those affected by rare diseases worldwide.
