rna-editing
(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.
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.
Amber Bio
Amber Bio is a biotechnology startup pioneering a new RNA writing platform that performs multi-kilobase edits, aiming to treat thousands of disease-causing mutations with a single product. Founded in 2021 by Jacob Borrajo and Basem Al-Shayeb out of the Broad Institute and UC Berkeley, the company launched in August 2023 with a $26M seed round co-led by Playground Global and Andreessen Horowitz Bio + Health.