The challenge

There is a critical need for disease-modifying therapies that can slow, stop or prevent the progression of neurodegenerative diseases, chronic conditions that destroy parts of the nervous system over time, especially the brain. More than 50 million people worldwide are currently affected, and that number continues to grow as populations age yet despite decades of research, only a few therapeutic targets have been prosecuted and no disease-modifying therapies exist to stop or slow disease progression for most neurodegenerative disorders.

Historically, progress has been constrained by an incomplete understanding of the complex biology of these diseases and by the limitations of available drug platforms, which drive extreme upregulation or downregulation of target activity. However, recent advances on both fronts have brought the industry to what we believe is an exciting tipping point.

Unlocking new targets

In recent years the investment in genetic studies and long-term natural history studies has yielded important new insights into disease biology and identified a new generation of promising disease-modifying targets across multiple neurodegenerative diseases.

Given the complexity of the biological pathways involved, many of these targets require highly controlled approaches with optimal safety profiles to enable early intervention with treatments that can halt disease progression. Existing drug modalities, including gene therapy and gene editing approaches, cannot deliver this level of fine-tuned, safe intervention, and as a result, these high-potential targets have remained out of reach.

Harness’s MISBA^® platform has been developed specifically to deliver controlled changes to individual targets, with ASOs modulating the translation of target proteins from the mRNA already present, providing intrinsically cell specific, self-limiting upregulation.

Learn more here.