Pipeline

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... Read More that directly addresses the pathological RNA underlying myotonic dystrophy type 1 (DM1.)

DM1 is a genetic neuromuscular disease affecting at least 1 in 8,000 people worldwide or ~45,000 people in the United States. It is a multi-systemic disease, affecting the heart, skeletal muscle, the central nervous system, and the gastro-intestinal tract.

DM1 is caused by a trinucleotide repeat expansion in the 3`UTR of the RNA encoding DMPK (myotonic dystrophy protein kinase) resulting in the binding and sequestration of splicing factors important for cellular function.

There are currently no approved drugs to treat DM1, and while there are promising oligonucleotide therapies in clinical development, these may not address the full systemic manifestations of DM1 because of their limited biodistribution.

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.

Expanding the universe of new treatments for patients

Our pipeline of rSM medicines

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