AIRNA
- Company typePrivate
- Founded2021
- HeadquartersCambridge, United States
- Headcount51–100
- GTM typeB2B
- OfferingServices
AIRNA firmographics
Firmographics- Name
- AIRNA
- Legal name
- AIRNA Corporation
- Website
- https://airna.com
- Company type
- Private
- Founded year
- 2021
- Operating status
- Operating
- Headcount range
- 51–100 employees
- Ownership category
- akta.pro rank
AIRNA industry classification
Industry- Product category
- RNA Editing Therapeutics
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714)
- 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), Antisense Oligonucleotides (ASO) Therapeutics (splice modulation/knockdown) (HLAAADAC), RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH), RNA Delivery Technologies (LNPs, polymers, conjugates incl. GalNAc, peptides, exosomes) (HLAAADAD), RNA Therapeutics Regulatory, Quality & Pharmacovigilance (GxP, comparability, post-market) (HLAAADAJ)
Keywords
Where AIRNA is headquartered
LocationHeadquarters
- HQ city
- Cambridge
- HQ country
- United States
- HQ region
- North America
Offices2 records
Markets served
AIRNA business model
Business model- GTM type
- B2B
- Offering type
- Services
- Cost components
- Personnel, Technology or R&D, Operations, Marketing or Sales, Others
Revenue model
- RNA Editing Therapeutic Development and Commercialization: As a clinical-stage biotech, AIRNA generates value through the development and potential commercialization of RNA editing therapeutics. The company is advancing AIR-001 through Phase 1/2 clinical trials for AATD, with a broader pipeline of cardiometabolic and rare disease programs in development. Revenue will derive from product sales upon regulatory approval, with potential for co-development, licensing, or partnership deals for specific programs or territories. The company also seeks strategic collaborations to extend its platform capabilities into therapeutic areas of mutual interest.
Go-to-market motion1 record
Distribution channels1 record
Marketing channels6 records
AIRNA product offering
Product offeringCore offering
AIRNA is a clinical-stage biotech developing RNA editing therapeutics through its proprietary RESTORE+ platform, which uses engineered antisense oligonucleotides to recruit endogenous ADAR proteins for precise A-to-I (read as G) RNA edits without altering DNA. The lead candidate, AIR-001, is in Phase 1 trials for alpha-1 antitrypsin deficiency (AATD), with a pipeline targeting cardiometabolic diseases such as cardiovascular disease and LDL cholesterol reduction.
Product overview
AIRNA is a clinical-stage biotech company developing RNA-editing therapeutics through its proprietary RESTORE+ platform. The company's lead product candidate is AIR-001, an RNA editing therapy for alpha-1 antitrypsin deficiency (AATD) currently in Phase 1 clinical trials. AIRNA is also advancing a pipeline of cardiometabolic programs targeting cardiovascular disease through protective variant introduction, as well as additional rare genetic disorder programs. The platform leverages adenosine deaminase acting on RNA (ADAR) to make precise edits to RNA without altering DNA.
Differentiator
Problem solved
Functional benefit
Products and services
- AIR-001 AIR-001 is AIRNA's lead GalNAc-conjugated RNA editing oligonucleotide therapeutic candidate for AATD, designed to repair the disease-causing PiZ mutation (G-to-A) at the RNA level and restore functional M-AAT protein production. Administered subcutaneously and supported by up to 59% in vivo RNA editing in preclinical models, with no observed off-target edits.
- RESTORE+ Platform RESTORE+ is AIRNA's proprietary ADAR-mediated RNA editing platform. It uses engineered antisense oligonucleotides to recruit naturally occurring human ADAR proteins to make precise single-letter A-to-I (read as G) corrections in RNA, without permanently altering DNA. The platform supports best-in-class editing efficiency, optimal safety, patient-friendly subcutaneous administration, and reversible, repeatable dosing.
- Cardiometabolic RNA-Editing Programs (LDLR, ApoB) AIRNA's preclinical pipeline programs for cardiometabolic disease that introduce protective cardiovascular variants identified through human genetics. The LDLR program targets LDL receptor editing for LDL cholesterol reduction, and the ApoB program targets apolipoprotein B to lower LDL cholesterol without hepatotoxicity.
Quantifiable outcome
- Up to 59% precise RNA editing in vivo with AIR-001 in a mouse model of AATD, corresponding to >40 µM corrected M-AAT protein in serum and 70 µM total AAT
- +5 more outcomes
Companies that use AIRNA
Customer profileNamed customers1 record
Segments3 records
Ideal customer profiles3 records
AIRNA technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature4 records
AIRNA partnerships and signals
Strategic signalPartnerships
Three partnerships are on record, tiered foundational co-founder institution and exploratory/partnerships sought.
- University of Tübingenfoundational co-founder institutionCo-founded by Thorsten Stafforst, PhD from the University of Tübingen, who pioneered the ADAR RNA editing approach. The university's research underpins the RESTORE+ platform technology. Deep ongoing research collaboration through Tübingen-based R&D operations.
- Stanford Universityfoundational co-founder institutionCo-founded by Jin Billy Li, PhD from Stanford University, who made key contributions to ADAR RNA editing science. Represents an academic collaboration providing foundational IP and continued scientific advisory through co-founder relationship.
- Strategic Collaborators (unspecified)exploratory/partnerships soughtAIRNA states it has deep expertise in precision RNA editing therapeutics across a range of genetically tractable disease targets and is actively seeking strategic collaborations to extend these capabilities to therapeutic areas of mutual interest. Contact: [email protected]
Scale indicators6 records
Recent moves6 records
Expansion highlights5 records
AIRNA competitors and assessment
Company assessmentEmerging players
- Verve Therapeutics: Verve Therapeutics develops in vivo gene editing therapies for cardiovascular disease, including LDL cholesterol reduction. Its cardiometabolic gene editing programs directly overlap with AIRNA's LDLR and ApoB pipeline programs, though using a different editing modality.
- Beam Therapeutics: Beam Therapeutics is a clinical-stage company developing base editing therapies, which like ADAR-mediated RNA editing enable precise single-letter genetic corrections without double-strand breaks. It represents an adjacent editing modality pursuing overlapping genetic disease indications.
- Tenaya Therapeutics: Tenaya Therapeutics develops genetic medicines for heart disease using gene therapy, gene editing, and cellular approaches. Its cardiometabolic focus overlaps with AIRNA's LDLR and ApoB programs, though using different modalities and delivery approaches.
Direct peers
- Arrowhead Pharmaceuticals: Arrowhead Pharmaceuticals develops RNAi-based therapeutics using TRiM platform with multiple clinical-stage programs targeting liver and other tissues. It is a direct peer in oligonucleotide-based genetic medicines with cardiometabolic and rare disease programs.
- Korro Bio: Korro Bio is a clinical-stage biotech developing ADAR-mediated RNA editing therapeutics for genetic diseases, including alpha-1 antitrypsin deficiency. It is the most directly comparable company to AIRNA, pursuing the same modality, similar target tissues, and overlapping indications.
- Wave Life Sciences: Wave Life Sciences develops RNA-based therapeutics including RNA editing programs (AIMer platform) using ADAR-mediated editing, as well as siRNA and other oligonucleotide modalities. It is a direct competitor in the RNA editing space targeting similar rare and neurological diseases.
- Ascidian Therapeutics: Ascidian Therapeutics (acquired by Roche in 2024) developed RNA editing therapeutics for neurological and other genetic diseases using an exon editing approach. It represents a direct competitor in the RNA editing modality space targeting rare diseases.
- ProQR Therapeutics: ProQR Therapeutics develops RNA-editing oligonucleotide therapies (Axiomer platform) that leverage ADAR for A-to-I editing. It targets rare genetic diseases and shares AIRNA's RNA editing mechanism and rare-disease commercial focus.
Broad incumbents
- Alnylam Pharmaceuticals: Alnylam is the commercial leader in RNAi therapeutics with multiple approved products and a deep pipeline. As a broader incumbent in RNA-targeted modalities, it shares AIRNA's liver-targeted oligonucleotide delivery approach (GalNAc) and rare-disease commercial infrastructure.
- Ionis Pharmaceuticals: Ionis is the leading antisense oligonucleotide (ASO) therapeutics company with multiple approved products and a broad pipeline. AIRNA's guide oligonucleotides are ASOs, making Ionis a relevant broad incumbent in the underlying chemistry and delivery platform.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat5 records
Key risks6 records
Key highlights6 records
Customer concentration
AIRNA social profiles
Digital presenceAIRNA compliance and trust
Trust signalCompliance2 records
AIRNA financial estimates
Financial estimateRevenue estimate
Valuation estimate
AIRNA leadership team
Management profileNumber of profiles
Profiles11 records
AIRNA subsidiaries and ownership
Company hierarchySubsidiaries1 record
AIRNA funding detail
Funding detailFunding overview
Funding rounds3 records
Investors11 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
AIRNA M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about AIRNA
What does AIRNA do?
AIRNA is a clinical-stage biotech developing RNA editing therapeutics through its proprietary RESTORE+ platform, which uses engineered antisense oligonucleotides to recruit endogenous ADAR proteins for precise A-to-I (read as G) RNA edits without altering DNA. The lead candidate, AIR-001, is in Phase 1 trials for alpha-1 antitrypsin deficiency (AATD), with a pipeline targeting cardiometabolic diseases such as cardiovascular disease and LDL cholesterol reduction.
Is AIRNA a public or private company?
AIRNA is a private company. It is classified as venture growth investor backed and is currently operating.
When was AIRNA founded?
AIRNA was founded in 2021. It employs 51 to 100 people.
Where is AIRNA based?
AIRNA is headquartered in Cambridge, United States, in the North America region.
How does AIRNA make money?
One revenue line is on record: RNA Editing Therapeutic Development and Commercialization.
Who are AIRNA's main competitors?
Emerging players on record are Verve Therapeutics, Beam Therapeutics and Tenaya Therapeutics. Direct peers are Arrowhead Pharmaceuticals, Korro Bio, Wave Life Sciences, Ascidian Therapeutics and ProQR Therapeutics. Broad incumbents are Alnylam Pharmaceuticals and Ionis Pharmaceuticals.
Does AIRNA have an API?
No public API is recorded for AIRNA.
What industry is AIRNA in?
AIRNA's product category is RNA Editing Therapeutics. Its primary akta.pro industry code is HLAAADAG, RNA Therapeutics Platforms & Design (sequence design, target ID/validation, AI/bioinformatics), with a secondary code of HLAAADAE, RNA Chemistry & Oligonucleotide Engineering (backbones, modifications, stereochemistry). Its NAICS code is 541714.