We are building a pipeline to maximize the number of rSM medicines for patients.

Based on the productivity of our platform, Arrakis has a pipeline of RNA-targeted drug programs that show promise for addressing a range of diseases, including cancer, cardiovascular conditions, neurodegeneration and rare diseases. In addition to the wholly-owned programs for rSM medicines in our internal pipeline, we are establishing collaborations with large biopharmaceutical partners to expand the R&D efforts from our platform.

Collaborations to expand value:

As an example of our partnership model, learn about our strategic collaboration with Roche

We are expanding the universe of drug discovery and new treatments for patients

Arrakis is increasing the opportunities for small molecule medicines beyond the domain of well-studied protein targets for today's treatments. Specifically, this new universe has orders of magnitude more potential targets than those currently accessible to the pharmaceutical industry.

Our Pipeline of rSM Medicines

We have taken a multi-pronged approach to building our pipeline of RNA-targeted small molecule (rSM) medicines by pursuing deeply researched, valuable protein drug targets, including:

  • known targets implicated in important disease biology that have historically proved "undruggable" with today's conventional medicines, and
  • other genetically and pharmacologically validated targets in cancer, rare disease, and cardiovascular disease.

Multiple mechanisms can include translation inhibition, splicing inhibition and degradation promotion

We pursue multiple sub-targets for each gene, exploring intervention by at multiple distinct mechanisms to discover which step in the RNA lifecycle can be modulated to have the most significant therapeutic impact. We have built a portfolio of rSMs that demonstrate both in vitro binding to RNA and in-cell modulation of target proteins, and are aggressively moving our programs towards lead optimization.

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