Our pipeline of rSM medicines

We have taken a matrixed approach to building our pipeline of RNA-targeted small molecule (rSM) medicines by pursuing different rSM modalities across deeply researched, valuable 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.

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

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 wholly-owned programs for rSM medicines in our internal pipeline, we are establishing collaborations with large biopharmaceutical partners to expand the productivity of our platform.

Collaborations to expand value:
As an example of our partnership model, learn about our strategic collaborations with Roche and Amgen.

INTRINSIC
CUG repeat-targeted rSM

Our lead program is an rSM with intrinsic function that directly addresses the pathological RNA underlying myotonic dystrophy type 1 (DM1.)   Learn more >

MYC-targeted rSM

rSMs with intrinsic function to inhibit translation of a tumor-specific MYC mRNA

PROXIMITY
Degraders

rSM-ligand conjugates for targeted RNA degradation

COVALENT
Translation inhibitors

Small molecules that inhibit translation by blocking ribosomal progression on a specific RNA, e.g., STAT3, YAP1, antivirals, etc.

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