rna-editing
(6)
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.
Todd Bazemore
Todd Bazemore is the President and CEO of AIRNA, a Cambridge, Massachusetts biotech developing RNA-editing medicines that rewrite disease-causing genetic variants without permanently altering DNA. Appointed in June 2026, he arrived as the company's lead program, AIR-001 for alpha-1 antitrypsin deficiency, moved into the clinic on the back of an oversubscribed $155 million Series B. A University of Massachusetts Lowell graduate with more than 30 years in rare-disease drug development, he previously ran Kala Bio, led U.S. operations at Santhera, and was chief commercial officer at Dyax during its $6.5 billion acquisition by Shire.
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.
Jacob Borrajo
Jacob Borrajo is the Founder and CEO of Amber Bio, a Cambridge-based biotech company pioneering a first-of-its-kind RNA writing platform capable of multi-kilobase edits. With a PhD in Biological Engineering from MIT and research training at the Broad Institute, Borrajo has spent his career building at the intersection of CRISPR, machine learning, and synthetic biology. Amber Bio launched in August 2023 with a $26 million seed round co-led by Andreessen Horowitz and Playground Global, with strategic participation from Eli Lilly and the Retinal Degeneration Fund. His platform's key innovation: treating diseases with high allelic diversity using a single therapeutic product, by editing RNA rather than DNA - making gene therapy safer, reversible, and dramatically more scalable.
Nessan Bermingham
Nessan Bermingham, Ph.D., is a serial biotech entrepreneur and Operating Partner at Khosla Ventures who has co-founded eight biotechnology companies including Intellia Therapeutics - one of the first public CRISPR gene editing companies - which he built from concept to IPO in under two years. A child of an Irish Army officer raised on a military base in Co. Kildare, he traded academia for Wall Street, Wall Street for venture capital, and venture capital for founding the companies reshaping genetic medicine. Today he shepherds a portfolio of cutting-edge biotech companies at Khosla Ventures while pursuing 155-mile ultra-marathons and aspiring to race at Dakar.