Arrakis Therapeutics

What Arrakis Therapeutics does

Arrakis Therapeutics is a private biopharmaceutical company founded in 2015 and headquartered in Waltham, Massachusetts, that pioneers the discovery of RNA-targeted small molecule (rSM) medicines — a new therapeutic class designed to directly bind and modulate disease-causing RNA structures with orally bioavailable small molecules. The company has built a proprietary end-to-end discovery platform integrating RNA bioinformatics (TRYST), photoaffinity-based binding site mapping (PEARL-seq), open-source structure analysis tools (SHAPEware), curated chemical libraries, RNA-specific assays, and AI/ML-driven chemotype analysis. Its lead wholly-owned candidate is an oral rSM for myotonic dystrophy type 1 (DM1), with IND filing planned for 2026 supported by preclinical data demonstrating splicing correction and myotonia reversal in vivo with broad biodistribution to skeletal muscle and heart.

The company operates a hybrid business model in which it generates revenue through strategic partnerships and licensing agreements with major pharmaceutical companies — notably a $190M upfront, multi-billion-dollar-potential collaboration with Roche (April 2020) and a $75M upfront, several-billion-dollar-potential collaboration with Amgen for RNA degrader therapeutics (January 2022) — while independently advancing its own pipeline in DM1, oncology, and genetically validated disease targets. Arrakis's customers are enterprise pharmaceutical partners rather than end patients or prescribers; Arrakis leads discovery to a defined point, then partners assume preclinical and clinical development. The company has raised approximately $113M in equity (Series A 2017, Series B 2019) and has not yet commercialized any product.

The company is led by CEO Michael Gilman (former founding CEO of Padlock Therapeutics and Stromedix; executive roles at Biogen and ARIAD), with Founder and Chief Innovation Officer Jennifer Petter (ex-Avila, Celgene, Mersana) and Board Chair Katrine Bosley (former CEO of Editas Medicine and Avila Therapeutics). Its Scientific Advisory Board is chaired by Melissa J. Moore, PhD, Chief Scientific Officer of Moderna's mRNA research platform and a National Academy of Sciences member. Headcount is 51-100, consistent with a mid-stage platform biotech scaling toward clinical development.

Name

Arrakis Therapeutics

Legal name

Arrakis Therapeutics

Website

https://arrakistx.com

Company type

Private

Founded year

2015

Operating status

Operating

Headcount range

51–100 employees

Short description

Arrakis Therapeutics is a private biopharmaceutical company developing orally bioavailable small molecules that directly bind disease-causing RNA, using a proprietary rSM drug discovery platform. It serves enterprise pharmaceutical partners (Roche, Amgen) via licensing collaborations and advances a wholly-owned DM1 candidate toward IND filing in 2026.

Ownership category

akta.pro rank

Product category

RNA-targeted small molecule drug discovery

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

RNA Therapeutics Platforms & Design (sequence design, target ID/validation, AI/bioinformatics) (HLAAADAG)

akta.pro secondary industries

RNA Chemistry & Oligonucleotide Engineering (backbones, modifications, stereochemistry) (HLAAADAE), In Vivo Discovery & Translational Models Platforms (humanized models, PK/PD, efficacy) (HLAAAIAK), Target Identification & Validation Platforms (omics-driven, chemoproteomics, genetic validation) (HLAAAIAI), RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH), RNA Therapeutics Regulatory, Quality & Pharmacovigilance (GxP, comparability, post-market) (HLAAADAJ), Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

Keywords

• RNA-targeted drug discovery
• Small molecule therapeutics
• Biopharmaceutical platform
• Neuromuscular disease drugs
• RNA bioinformatics

Headquarters

HQ city

Waltham

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Partnership and Collaboration Revenue: Arrakis generates revenue through strategic partnerships with pharmaceutical companies. The company receives upfront payments, preclinical, clinical, and commercial milestone payments, and royalties on products developed through partnerships. Notable deals include Roche ($190M upfront, multi-billion dollar potential) and Amgen ($75M upfront, potential royalties up to low double digits per program)
2. Series Financing: Pre-revenue biotech company funded through venture capital. Raised $38M Series A (2017) and $75M Series B (2019) from life sciences investors to advance platform development and pipeline programs

Go-to-market motion2 records

Distribution channels2 records

Marketing channels5 records

Core offering

Arrakis Therapeutics develops RNA-targeted small molecule (rSM) drug candidates through a proprietary end-to-end discovery platform that integrates RNA bioinformatics, chemical biology, RNA-specific assays, and RNA-directed medicinal chemistry. Its lead internal asset is an orally bioavailable rSM drug candidate for myotonic dystrophy type 1 (DM1), with additional programs in oncology and genetically validated targets, and it monetizes the platform through major pharmaceutical collaborations with Roche and Amgen.

Product overview

Arrakis Therapeutics is a biopharmaceutical company pioneering RNA-targeted small molecule (rSM) medicines — a new class of genetic medicines that directly target RNA with small molecules for oral delivery. The company's offering centers on its proprietary rSM Platform, which integrates bioinformatics tools (TRYST), chemical biology techniques (PEARL-seq), and RNA-guided medicinal chemistry to systematically discover and design RNA-targeted small molecules. The rSM Toolkit provides industrial-scale discovery capabilities including SHAPEware (open-source bioinformatics software) for RNA structure analysis. The company's lead product is an oral rSM drug candidate for Myotonic Dystrophy Type 1 (DM1), with additional programs in oncology and genetically validated targets. The platform enables targeting of previously 'undruggable' disease drivers by binding directly to pathogenic RNA sequences.

Differentiator

Problem solved

Functional benefit

Products and services

• DM1 Drug Candidate (oral RNA-targeted small molecule for myotonic dystrophy type 1) Lead first-in-class, orally administered RNA-targeted small molecule in development for myotonic dystrophy type 1 (DM1). The candidate selectively binds the pathogenic CUG repeat RNA, disrupts toxic nuclear aggregates, releases sequestered MBNL1 splicing factor, and corrects splicing defects that drive myotonia. Indication: patients with myotonic dystrophy type 1 (DM1), a genetic neuromuscular disease affecting skeletal muscle, heart, central nervous system, and gastrointestinal tract.
• rSM Drug Discovery Platform Proprietary end-to-end drug discovery platform integrating RNA bioinformatics, structural tools, curated RNA-focused chemical libraries, RNA-specific assays, and RNA-directed medicinal chemistry to systematically discover and design RNA-targeted small molecules. Sold/offered to large pharmaceutical partners through strategic collaboration and licensing agreements; also used for internal pipeline development.
• SHAPEware open-source bioinformatics software Standalone open-source bioinformatics software tool for analyzing SHAPE-based experimental data to explore and predict RNA secondary structure and potential ligand binding sites. Made freely available to academic and industry RNA researchers.

Quantifiable outcome

• Oral administration achieved broad biodistribution including to skeletal muscle and heart in preclinical studies
• +3 more outcomes

Segments3 records

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

AI capability2 records

Feature5 records

Partnerships

Three partnerships are on record, tiered flagship and core.

• AmgenflagshipStrategic or Co-development Partner · 11 January 2022Multi-target collaboration focused on discovery and development of RNA degrader therapeutics creating targeted RNA degraders that selectively destroy RNAs encoding disease-causing proteins by inducing their proximity to nucleases. Arrakis receives $75M upfront for five initial programs with option to nominate additional programs. Eligible for preclinical, clinical, regulatory and sales milestones, and royalties up to low double digits per program. Potential for several billion dollars if all milestones met and program options exercised.
• RocheflagshipStrategic or Co-development Partner · 8 April 2020Strategic collaboration and license agreement for discovery of RNA-targeted small molecule drugs against broad set of targets across Roche's R&D areas. Arrakis leads discovery and research activities to a defined point, then Roche pursues preclinical and clinical development. Arrakis received $190M upfront payment with potential for multi-billion dollar future payments including preclinical, clinical, commercial and sales milestones and royalties.
• Katrine Bosley (Board Member)coreStrategic or Co-development Partner · 21 November 2019Appointed as Chairman of the Board of Directors. Katrine Bosley is a proven biotech entrepreneur with nearly 30 years experience, former CEO of Editas Medicine and Avila Therapeutics. Brings expertise growing biotech platform companies.

Scale indicators5 records

Recent moves5 records

Expansion highlights5 records

Broad incumbents

• Ionis Pharmaceuticals: Ionis is the established leader in antisense oligonucleotide (ASO) therapeutics with multiple approved drugs (Spinraza, Tegsedi, Waylivra, Wainua) and a broad pipeline across neurological, cardiovascular, and rare diseases. As the incumbent in RNA-targeted therapeutics, Ionis is broadly comparable to Arrakis in disease focus (neurology, rare diseases) and represents the alternative modality (ASOs vs. rSMs) for many of the same targets Arrakis is pursuing.
• Alnylam Pharmaceuticals: Alnylam is the commercial leader in RNA interference (siRNA) therapeutics with multiple approved drugs (Onpattro, Givlaari, Oxlumo, Amvuttra) and a deep pipeline in rare genetic and cardiometabolic diseases. Broadly comparable to Arrakis in therapeutic focus on genetic and rare diseases and as the dominant incumbent in the RNA-targeting therapeutic space, though using a fundamentally different modality (siRNA vs. rSMs).

Emerging players

• Wave Life Sciences: Wave Life Sciences is developing stereopure oligonucleotide therapeutics (PRISM platform) for genetic diseases including DM1, Huntington's disease, and ALS. Comparable to Arrakis in target overlap (DM1, repeat expansion disorders, CNS diseases) and as a competitor pursuing RNA-modulating therapies for the same indications, though via oligonucleotide chemistry rather than small molecules.
• PepGen: PepGen is developing conjugated peptide-enhanced oligonucleotide therapeutics for DM1 and Duchenne muscular dystrophy, with a proprietary delivery platform (Enhanced Delivery Oligonucleotide). Directly comparable to Arrakis in DM1 focus and in pursuing RNA-targeting therapeutics for neuromuscular diseases, though using a distinct modality (conjugated oligonucleotides vs. rSMs).

Direct peers

• Skyhawk Therapeutics: Skyhawk Therapeutics develops small molecules that modify RNA splicing using a proprietary platform (SkySTAR) — directly comparable to Arrakis's rSM platform in both modality (RNA-targeting small molecules) and therapeutic focus on neurological and rare diseases. Skyhawk has secured major partnerships with Biogen, Vertex, and Merck, mirroring Arrakis's partnership-driven business model.
• Expansion Therapeutics: Expansion Therapeutics is a repeat-expansion-focused company developing small molecules for diseases caused by trinucleotide and other repeat expansions, including myotonic dystrophy type 1 (DM1) — directly competing with Arrakis's lead DM1 program. Expansion's lead program EX-5050 for DM1 makes it the most direct indication-level competitor to Arrakis.
• Remix Therapeutics: Remix Therapeutics is developing small molecule therapeutics that modulate RNA processing, with a platform (REMaster) targeting RNA processing machinery to address disease-driving transcripts. Directly comparable to Arrakis in approach (RNA-targeted small molecules) and focus on previously undruggable disease targets across oncology and neurological disease.
• Ribometrix: Ribometrix is a privately held biotech developing small molecule drugs that target RNA structures, with a proprietary platform combining structural biology, chemistry, and computational methods to identify and optimize RNA-binding small molecules. Directly comparable to Arrakis in technology approach (RNA-targeted small molecules) and platform-driven discovery model.
• Dyne Therapeutics: Dyne Therapeutics is developing oligonucleotide-based therapies for muscle diseases including DM1, Duchenne muscular dystrophy, and facioscapulohumeral muscular dystrophy, with a proprietary platform for muscle-targeted delivery. Directly comparable to Arrakis in DM1 focus and in targeting muscle diseases via RNA-modulating approaches, though using a different modality (antisense oligonucleotides).
• PTC Therapeutics: PTC Therapeutics is advancing RGLS8429, an oligonucleotide-based therapy for DM1, into clinical development — making it a direct indication-level competitor to Arrakis's lead DM1 program. As an established commercial-stage rare disease biotech, PTC represents the most credible near-term competitive threat in the DM1 indication Arrakis is targeting with its lead rSM.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks7 records

Key highlights7 records

Customer concentration

• Xx.com/arrakistx
• LinkedInlinkedin.com/company/arrakis-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles15 records

Funding overview

Funding rounds2 records

Investors13 records

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M&A

Investments

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Frequently asked questions about Arrakis Therapeutics