Wave Life Sciences Ltd.

AIRNA’s total capital raised so far is $245m. Credit: sesame via Getty Images. AIRNA has raised $155m in Series B financing to advance its RNA editing pipeline, and kick-start a clinical trial for its lead candidate, AIR-001. Venrock Healthcare Capital Partners and Forbion Growth co-led the new round, bringing AIRNA’s total raised capital to $245m. The US and Germany-based biotech plans to file a clinical trial application (CTA) for its lead candidate, AIR-001, for alpha-1 antitrypsin deficiency (AATD) by H2 2025. The company is developing AIR-001 as an investigational RNA editor – a therapy designed to modify RNA sequences to correct disease-causing mutations. This approach is different to DNA editing approaches such as clustered regularly interspaced short palindromic repeats (CRISPR), which make permanent changes to the genome. RNA editing is a reversible process that allows for targeted corrections without altering the underlying genetic code. The Series B funds will support the launch of a Phase I/II clinical trial for AIR-001 and the further development of other RNA-editing candidates, primarily targeting cardiometabolic diseases. AATD is a rare genetic disorder caused by mutations in the SERPINA1 gene, which encodes the AAT protein. Under normal circumstances, AAT is produced in the liver and transported to the lungs, where it regulates enzyme activity to prevent tissue damage. In patients with AATD, mutations can prevent AAT from exiting the liver, leading to toxic protein accumulation that damages the liver while also depriving the lungs of necessary protection. This can result in progressive lung disease, liver dysfunction, and symptoms such as shortness of breath, fatigue, and jaundice. The AATD treatment landscape is becoming increasingly competitive as multiple biotechs advance RNA-editing candidates. Korro Bio is investigating its candidate KRRO-110 in the Phase I/IIa REWRITE clinical trial (NCT06677307), with interim data expected in H2 2025. The company announced last month that the dosing of the first two cohorts had been completed, and the trial is expected to conclude next year. Wave Life Sciences is developing WVE-006, which demonstrated targeted RNA editing in the first two patients treated in the Phase Ib/IIa RestorAATion-2 clinical trial (NCT06405633). The growing interest in RNA editing has also drawn investment from major pharmaceutical companies. In November 2023, Roche expanded its partnership to develop a one-time RNA editing therapy using ShapeTX’s AI-powered platform for an undisclosed disease. In September 2024, Novo Nordisk and Korro Bio announced a collaboration leveraging Korro’s OPERA platform to enable oligonucleotide-directed RNA edits for two undisclosed targets, initially focused on cardiometabolic diseases. The deal included up to $530m in upfront, development, and commercial milestone payments, along with tiered royalties and R&D funding. Not all genetic medicine approaches for AATD have progressed as planned. In January 2025, Intellia Therapeutics discontinued the development of its CRISPR-based gene therapy NTLA-3001, which was intended to correct the SERPINA1 mutation at the DNA level. Preparations for the Phase I/II trial were halted, along with other early-stage programmes, signalling a shift in the company’s pipeline priorities. With fresh funding and a CTA planned for later this year, AIRNA is positioning itself within the growing RNA editing field. Forbion managing partner Dirk Kersten said: “AIRNA’s innovative approach to RNA editing has the distinctive potential to improve health across large populations by introducing healthy genetic variants for many conditions. We are excited to support the expansion of AIRNA’s pipeline of life-changing medicines and the advancement of AIR-001 into the clinic.”

When the transatlantic startup AIRNA launched in 2023, it was already years behind other companies that were trying to bring a new form of genetic medicine known as RNA editing into the clinic. But that competition hasn’t daunted AIRNA or its investors. On Tuesday, AIRNA announced that it has raised $155 million in Series B financing and plans to file a clinical trial application by the second half of this year. The new round, co-led by Venrock Healthcare Capital Partners and Forbion Growth, brings the Cambridge, MA and Tübingen, Germany-based startup’s total funding to $245 million. AIRNA’s lead program targets a liver and lung disease called alpha-1 antitrypsin deficiency, or AATD. The genetic condition may impact up to 100,000 people in the US, and it is becoming an increasingly competitive area of drug development. Two other biotechs, Korro Bio and Wave Life Sciences, have already begun clinical trials of their own RNA editing therapies for AATD. Wave disclosed preliminary data from the first two patients in the study in October, and said that its approach restored a vital protein that’s missing in the disease. And in March, the gene editing company Beam Therapeutics revealed the first data suggesting that its permanent fix for AATD may work, too. “It’s more important to be best for patients than to be first in patients. We spent quite a bit of time optimizing our therapy,” AIRNA president and CEO Kris Elverum told Endpoints News in an interview. “Too many people get caught up in the perception that one company is going to win. This is a large market.” AIRNA, Korro and Wave are three leaders in a race to develop therapies that alter the code of short-lived mRNA molecules. The approach has been pitched as a potentially safer and more versatile alternative to CRISPR gene editing — including the base editing technology employed by Beam — which makes permanent changes to DNA. Although the benefits of such transiency aren’t yet proven, AIRNA’s new financing comes at a time when many private and public investors are stepping back from companies developing genetic medicines. Questions about the long-term safety and efficacy of some of these treatments, combined with sluggish sales, have renewed questions about the business model of one-time cures. “Patients and physicians don’t want today’s one-and-supposedly-done therapies,” Elverum said. “Our RNA editing medicines are going to look and feel to patients and physicians like medicines that they’re all used to.” RNA editing relies on a natural enzyme, found in cells across the body, that changes adenosine bases to inosine bases in mRNA strands. AIRNA and its competitors have developed synthetic RNA molecules that essentially lasso that enzyme to precisely control that A-to-I editing — the inspiration for the company’s name — allowing them to fix the genetic mutation responsible for AATD. AIRNA plans to deliver the therapy, which from a chemical standpoint is similar to a gene-silencing siRNA, as an “infrequent” subcutaneous injection, Elverum said. “The key difference being that siRNA is a hammer that inhibits the entire RNA, whereas we have that genetic precision to just change one individual letter,” he said. The startup isn’t saying much more about its approach, or what differentiates it from Korro and Wave. Unlike its competitors, AIRNA hasn’t released preclinical data yet, although Elverum said that he plans to change that. AIRNA is also tight-lipped about what it plans to do with RNA editing next. The company mentioned an interest in cardiometabolic disease and in using the approach to introduce genetic mutations known to improve health, rather than just fix mutations linked to disease, but wouldn’t give specific examples. Elverum wouldn’t say what country he plans to submit its clinical trial application to, only that it will be outside the US. He also declined to list specific goals for the company’s new funding or how much runway it will provide the roughly 50-person company.

Dive Brief:Airna, a biotechnology startup developing RNA editing treatments, said Tuesday it raised $155 million in a Series B round that will help propel its lead program in alpha-1 antitrypsin deficiency into the clinic later this year.That medicine, codenamed AIR-001, repairs faulty RNA transcribed by the gene thats mutated in alpha-1 antitrypsin deficiency, or AATD. When the RNA is edited, cells are able to produce functional copies of AAT protein, lowering the risk of associated lung and liver disease.Airnas raise comes nearly six months after Wave Life Sciences unveiled promising Phase 1 data for an RNA editing drug also aimed at AATD. Similar to Wave, Airnas drug uses a sugar molecule to take its medicine into the liver.Dive Insight:No cure currently exists for AATD, which in some patients is managed with weekly injections of AAT protein. Airna estimates approximately 100,000 people in the U.S. have AATD.Co-founded by professors Thorsten Stafforst at the University of Tubingen and Jin Billy Li at Stanford University, Airna hopes to fill that niche with technology that can recruit an enzyme known as adenosine deaminase acting on RNA, or ADAR, to modify RNA.Kris Elverum, Airnas CEO, said the appeal of a drug like AIR-001 is its resemblance to biologic drugs, rather than experimental gene therapies that permanently alter DNA and can be costly to manufacture. AIR-001 is administered via a subcutaneous injection and its developers are experimenting with how to maintain its potency at lower dosages.Patients and physicians want to be able to have options that can give them a functional cure while maintaining flexibility and control over their healthcare decisions, Elverum said.The company has not disclosed specific conditions its targeting outside of AATD, but co-founder Stafforst has published research on the applicability of RNA editing in cardiometabolic and neurodegenerative diseases.RNA editing is not going to be the solution for every disease and every target, but what our data suggests is that the physiological effects of healthy variants that we identify can be reproduced through a single RNA edit, Elverum said.The Series B round was co-led by Venrock and Forbion, and included investors such as RTW Investments, Arch Venture Partners and Nextech Invest. Airna previously raised $90 million after emerging from stealth in 2023.Startups such as Shape Therapeutics, Ascidian Therapeutics and ADARx Pharmaceuticals, as well as established biotechs such as Wave and Korro Bio, have turned to RNA editing for tough-to-treat diseases. While the field has drawn hundreds of millions of venture capital dollars and the attention of pharma partners such as Roche and Novo Nordisk, its unclear whether any drugs resulting from that investment will succeed in finding a treatment niche.Airnas innovative approach to RNA editing has the distinctive potential to improve health across large populations by introducing healthy genetic variants for many conditions, Derk Kersten, a managing partner at Forbion, said in a statement. '