Download the free whitepaper: Unravelling the complexities of genomics-driven drug discovery
Genomics-driven drug discovery holds immense promise in developing targeted therapies by leveraging genetic and biomarker information to understand the root causes of diseases. However, the field faces significant hurdles.
In this whitepaper, we examine the obstacles of limited recontactability, scarcity of multi-omics data sets, lack of diversity in data, and the challenge of scaling research for rare diseases. By addressing these challenges and proposing strategies to address them, this whitepaper aims to chart a path by which we can harness the full potential of genomics-driven drug discovery.
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Mapping new pathways
in orphan drug
development: Data,
design, and equity
Only 5% of rare diseases currently have approved treatments. While innovation in rare disease research is accelerating, orphan drug development remains hindered by data limitations, recruitment challenges, and outdated clinical models. In April 2025, alongside the World Orphan Drug Congress, Sano convened experts from biotech, pharma, and advocacy to explore how advances in genomics, data science, and patient engagement can transform this landscape.
This whitepaper captures key insights from that discussion, highlighting current challenges, potential strategies, and actionable recommendations to help sponsors, regulators, and advocates work together toward a more inclusive, data-driven, and patient-empowered rare disease ecosystem.
Download now to learn more.
