oligonucleotides
(3)
AIRNA
AIRNA is a clinical-stage biotechnology company building RNA-editing medicines that harness ADAR, a naturally occurring human enzyme, to make precise, programmable and non-permanent edits to RNA. Its RESTORE+ platform uses short oligonucleotides to recruit ADAR to a target transcript and swap a single letter, correcting disease-causing variants without altering DNA. AIRNA's lead candidate, AIR-001, targets alpha-1 antitrypsin deficiency (AATD) and has advanced into a Phase 1/2 clinical trial, with a broader pipeline aimed at cardiometabolic and other diseases. The company is headquartered in Cambridge, Massachusetts, with research operations in Tübingen, Germany, and has raised roughly $245 million from investors including ARCH, Forbion and Venrock.
DNA Script
DNA Script is a French life-sciences technology company that pioneered Enzymatic DNA Synthesis (EDS) and built the SYNTAX System, the first benchtop DNA printer that lets researchers make custom DNA oligonucleotides on-site in hours instead of waiting days for outsourced orders. Founded in 2014 in Le Kremlin-Bicetre near Paris, the company aims to make biology programmable by putting rapid, template-free DNA production directly on the lab bench.
Creyon Bio
Creyon Bio is a San Diego biotech reinventing how RNA-targeted medicines are designed. Founded in 2019 by former Ionis Pharmaceuticals scientists, the company pairs machine learning with quantum-level molecular modeling in its NucleIQ platform - an AI-Powered Oligo Engineering Engine - to predict the safety, efficacy, and delivery of oligonucleotide-based medicines before they ever reach a lab bench. The goal: replace slow, expensive trial-and-error with computational design rules that make precision therapeutics engineerable 'on demand,' for both rare and common diseases across neuromuscular, CNS, and immunologic areas.