Arrakis Therapeutics is developing RNA-targeted small molecule therapeutics to address neurological disorders and other diseases by utilizing a novel RNA degradation approach. Their focus on rSM medicines aims to improve treatment efficacy and patient outcomes in complex disease areas.
Funding
$115.5M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
VPFounders
Product
Problem
Many disease-causing proteins are considered "undruggable" by traditional small molecule therapies due to their structure or function. Current genetic medicines, such as oligonucleotides, may have limited biodistribution, failing to address systemic manifestations of disease.
Solution
Arrakis Therapeutics is pioneering RNA-targeted small molecule (rSM) therapeutics, a new class of genetic medicines designed to address a wide range of diseases. Their platform focuses on discovering small molecule drug compounds that directly bind to RNA, modulating its function and unlocking previously inaccessible biological targets. By targeting RNA, Arrakis aims to create orally available medicines with improved biodistribution and the potential to treat diseases currently unaddressed by existing therapies. The company is developing a pipeline of rSM medicines across various modalities, including intrinsic function inhibitors, proximity-induced degraders, and covalent translation inhibitors.
Target Audience
Arrakis Therapeutics focuses on patients with diseases that are currently untreatable or poorly managed by existing therapies, including those with cancer, rare diseases, and cardiovascular conditions.
Features
- Discovery platform for identifying small molecules that selectively bind to RNA targets
- Capability to drug targets previously considered undruggable by traditional methods
- Development of rSMs with intrinsic function to directly modulate RNA activity
- rSM-ligand conjugates for targeted RNA degradation
- Small molecules that inhibit translation by blocking ribosomal progression on specific RNAs
- Lead program targeting the CUG repeat expansion in myotonic dystrophy type 1 (DM1)
- Programs targeting MYC mRNA to inhibit translation in tumor-specific contexts
- Strategic collaborations with pharmaceutical partners to expand the reach of the rSM platform