Key Data
| Total Companies | 252 |
| Therapeutics | 213 |
| Platforms & Tools | 17 |
| Diagnostics | 10 |
| Top Modality | mRNA (76) |
| Top Indication | Oncology (96) |
Data sourced from HOUSTON, FLYTE’s proprietary biotech intelligence platform tracking 12,000+ early-stage companies worldwide.
About This Report
RNA therapeutics has crossed the hype boundary. The field that spent 2020–2023 riding the mRNA vaccine wave is now producing clinical data across seven distinct modalities — from siRNA and ASO (the quiet workhorses generating actual revenue) to tRNA, circular RNA, and saRNA platforms that are just entering human testing for the first time.
This report catalogs 252 early-stage companies developing RNA-based therapeutics or enabling RNA drug discovery and delivery. The landscape spans mRNA (76 companies), ASO (42), siRNA/RNAi (38), saRNA and emerging platforms (66), microRNA (13), circular RNA (11), and tRNA (6). Therapeutic focus clusters in oncology (96 companies), rare disease (89), and neurology (80) — with the delivery problem remaining the single biggest barrier between preclinical promise and clinical proof.
Capital is flowing to companies that solve delivery. Atrium Therapeutics launched with $270M for cardiac-targeted siRNA. aerska raised $39M to cross the blood-brain barrier with RNAi. The $2.25 billion Arbutus/Genevant settlement with Moderna over LNP patents underscores just how valuable the delivery infrastructure has become. The modality is mature. The delivery layer is where the next wave of value creation lives.
What’s Inside
- Executive Summary
- Modality Breakdown
- Stage Breakdown
- Indication Breakdown
- Recent Investments
- Recent Signals & Clinical Milestones
- Prominent Early-Stage Players
- Full Company Directory (252 Companies)
Sample Companies
8 of 252 companies featured in the full report
| Company | Focus | Stage | Modality |
|---|---|---|---|
| Alltrna | Rare Disease | Phase 1 | tRNA |
| Arrowhead Pharmaceuticals | Cardiometabolic | Commercial | siRNA / RNAi |
| Korro Bio | Liver / Genetic | Preclinical | RNA Editing |
| ARTHEx Biotech | Neuromuscular | Phase 2 | microRNA |
| Ascidian Therapeutics | Ophthalmology / Neuro | Phase 2 | RNA Exon Editing |
| aerska | Neurology | Preclinical | siRNA / RNAi |
| Tangram Therapeutics | Metabolic (MASH) | Phase 1/2 | siRNA / RNAi |
| Tacit Therapeutics | Neuro (HD, Dravet) | Preclinical | RNA Splicing |
Recent Signals
6 of 30+ signals tracked in the full report
Mar 2026
Arbutus/Genevant reach $2.25B LNP patent settlement with Moderna — $950M upfront, validating delivery IP as the most valuable layer in RNA.
Mar 2026
Alltrna’s AP003 becomes the first tRNA-based therapeutic to enter human clinical trials — a new modality unlocked.
Feb 2026
Atrium Therapeutics launches with ~$270M to develop cardiac-targeted siRNA therapeutics for rare genetic cardiomyopathies.
Dec 2025
CAMP4 Therapeutics enters $440M+ collaboration with GSK to develop regulatory RNA-targeting ASO therapeutics for neurodegenerative and kidney diseases.
Mar 2026
Korro Bio closes oversubscribed $85M PIPE to advance RNA editing pipeline through upcoming clinical milestones.
Mar 2026
Arrowhead presents Phase 2b plozasiran data showing 83% triglyceride reduction sustained over two years — and doses first dual-RNAi candidate ARO-DIMER-PA.
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