Interstitial Lung and Liver Disease

Financing led by Venrock Healthcare Capital Partners with co-lead Forbion Growth, and participation from RTW Investments, Nextech Invest, and other new investors Current investors ARCH Venture Partners, Forbion Ventures, ND Capital, and others also participated AIRNA expected to file clinical trial application for Alpha-1 antitrypsin deficiency (AATD) RNA editing product candidate (AIR-001) with potential best-in-class profile in 2H 2025 CAMBRIDGE, MA, USA & TÜBINGEN, Germany I April 01, 2025 I AIRNA, a biotech company pioneering RNA editing therapeutics to transform the lives of patients with rare and common conditions, today announced the closing of an oversubscribed $155 million Series B financing. The financing was led by Venrock Healthcare Capital Partners, co-led by Forbion Growth, and included participation from RTW Investments, Nextech Invest, ARCH Venture Partners, Forbion Ventures, ND Capital, and other new and existing investors. AIRNA will use the proceeds to advance its lead RNA editing product candidate (AIR-001) for AATD into a Phase 1/2 clinical trial, and to develop a pipeline of novel RNA-editing medicines for cardiometabolic and other diseases. “AIRNA is developing a new class of genetic medicines that could provide functional cures for a wide range of diseases,” said Kris Elverum, President and Chief Executive Officer of AIRNA. “This partnership of new and existing investors allows us to rapidly deliver AIR-001 to patients with AATD, as well as progress a pipeline of medicines to realize the full potential of RNA editing.” AATD is caused by mutations in the SERPINA1 gene that lead to insufficient levels of functional alpha-1 antitrypsin (M-AAT) protein, which results in lung and liver disease. AIR-001 precisely repairs the most common, harmful SERPINA1 mutation (PiZ) to address the underlying cause of both lung and liver disease and restore functional M-AAT production. AIR-001 was designed for potent and durable M-AAT production, convenient, subcutaneous dosing, and well-tolerated safety to give patients a preferred therapeutic option. “Patients with AATD have limited therapeutic options, and AIRNA is developing a potentially best-in-class medicine that has been optimized for potency, durability, and safety,” said Ken Greenberg, M.D., partner at Venrock Healthcare Capital Partners. “Venrock is thrilled to collaborate with the tremendous team at AIRNA, and a world-class investor syndicate, to bring novel RNA editing medicines to patients.” AIRNA’s proprietary platform harnesses natural mechanisms to potently edit a target RNA with a safe and flexible medicine. Precise RNA editing has unique potential to safely introduce beneficial genetic variants that promote optimal health, in addition to repairing disease-driving mutations, such as those causing AATD. AIRNA identifies variants that drive health through human genetics, and is developing new medicines to transform the lives of patients with cardiometabolic and other diseases. “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 AIR-001 into the clinic,” said Dirk Kersten, Managing Partner at Forbion. Alongside this financing, AIRNA announced the appointment of Melissa McCracken, Ph.D., a partner at Nextech Invest, to the Board of Directors. Dr. McCracken has a track record of growing the next generation of innovative biopharma companies, with a focus on precision approaches. About AIRNA AIRNA is harnessing advances in genetics to develop transformative RNA-editing medicines that improve human health across both rare and broad populations. RNA editing offers the precision of genetic medicine approaches while maintaining treatment convenience, flexibility, and reversibility. Our proprietary platform is based on groundbreaking research by pioneers of the field and company co-founders Thorsten Stafforst (University of Tübingen) and Jin Billy Li (Stanford University) and enables optimal potency, safety, and delivery. AIRNA is advancing a robust pipeline of therapeutic candidates that are designed to provide functional cures for severe or chronic diseases by repairing harmful genetic variants or introducing beneficial variants promote optimal health. AIRNA’s lead program has the potential to be a best-in-class therapeutic for alpha-1 antitrypsin deficiency (AATD). AIRNA has headquarters in Cambridge, MA, with research operations in Tübingen, Germany. Learn more at https://airna.com/ . SOURCE: AIRNA

AIRNA is focused on genetic variations that cause disease, plus using precision editing tech to tweak RNA into more healthy variations.\n AIRNA, a transatlantic RNA editing biotech, is prepping to launch its lead asset into clinical trials with fuel from a $155 million series B fundraise.The round was led by Venrock Healthcare Capital Partners and Forbion Growth, AIRNA announced in an April 1 release. Forbion had previously led AIRNA’s $60 million financing in July 2024. Other investors in the most recent round include RTW Investments, Arch Venture Partners, Nextech Invest, Forbion Ventures, ND Capital and other unnamed investors.  The series B cash will go toward launching a phase 1/2 trial for AIR-001, AIRNA’s drug candidate for alpha-1 antitrypsin deficiency (AATD), and will also help bolster the company’s early pipeline of RNA editing therapeutics.AIRNA plans to ask for approval to enter AIR-001 into clinical trials in the second half of this year, the company’s CEO Kris Elverum told Fierce Biotech in an interview. Elverum declined to disclose more information about the trial’s design and timeline.The biotech is also working to advance other preclinical programs that stem from AIRNA’s proprietary platform, Elverum added, including one in cardiometabolic disease.The company, headquartered in Cambridge, Massachusetts, with a subsidiary in Tübingen, Germany, was founded in 2021 and is open to partnerships with larger companies, according to the CEO.“There\'s only so much we\'re able to do as a small company,” Elverum said. “There are other companies that have capabilities in certain areas that would be better suited for bringing novel medicines to patients.”That being said, AIRNA remains committed to “building a significant and distinctive biopharma company for the long term,” Elverum added. AIRNA\'s lead asset is designed to treat AATD, a lung and liver disease that is caused by a mutated copy of the SERPINA1 gene, which leads to low levels of the protective alpha-1 antitrypsin (AAT) protein in the blood. AAT is made in the liver but ultimately migrates to the lungs; without enough of the protein, patients with AATD experience lung symptoms like coughing and shortness of breath. Malfunctioning AAT can also build up in the liver and cause cirrhosis, and patients with AATD may ultimately need a lung or liver transplant.The most common causative mutation of AATD is called PiZ, which is when an adenosine occurs at a site in the gene’s DNA instead of a guanosine. AIR-001 aims to fix this glitch by swapping the errant adenosine for a nucleotide called inosine, which the body treats the same as a guanosine.“We\'re able to repair the mutation for alpha-1 antitrypsin deficiency through this approach to give patients what we believe will be a functional cure,” Elverum said.AIRNA’s platform takes advantage of an enzyme that can be found in all of our cells, called adenosine deaminase acting on RNA (ADAR). AIRNA cofounder Thorsten Stafforst, Ph.D., a biochemist at the University of Tübingen in Germany, published a paper in 2019 demonstrating that oligonucleotides can be used to recruit ADAR to make targeted adenosine-to-inosine edits in RNA.Though AIRNA’s founders have also studied other RNA editing mechanisms, AIRNA is focused solely on harnessing ADAR.“Our data would indicate that there\'s a wide swath of opportunities for making multiple medicines with that one A to I letter change,” Elverum said.With AATD and other genetic conditions, AIRNA is focused on genetic variations that cause disease. But the company is also interested in using its precision editing tech to tweak RNA into more healthy variations.“It\'s the 90-year-old smoker who doesn\'t develop heart disease. Why is that?” Elverum said. “As a team, we\'ve spent a lot of time identifying these healthy variants and our data would indicate that we can recapitulate the positive physiological effects of these healthy variants through a single RNA edit.” AIR-001 requires regular subcutaneous doses, because editing RNA is not permanent the way editing DNA is. Elverum sees the temporary nature of RNA editing as a distinct advantage to the approach over one-and-done DNA editing gene therapies. “People want to maintain control over their health,” Elverum said. With RNA editing therapies, patients can work with their physicians to adjust dosing and find what works best for them, he added, or stop taking the medicine entirely if they want.Elsewhere in the industry, a one-and-done gene therapy for AATD from Beam Therapeutics recently saw early success in a phase 1/2 trial, appearing to correct the PiZ mutation in nine patients, with no serious adverse events reported. Beam\'s trial is expected to enroll about 106 adult patients with AATD and is taking place in two parts. The first part includes patients with AATD and lung disease, while the second part will include patients with AATD, plus patients with AATD that also have mild to moderate liver disease.

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.