ADA

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.

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. '

30 patients with Wiskott-Aldrich syndrome have been treated with etuvetidigene autotemcel. Credit: Telethon Foundation via LinkedIn. Italian research charity Telethon Foundation is one step closer to getting a gene therapy to treat rare disease Wiskott-Aldrich syndrome WAS approved in the European Union. The gene therapy, named etuvetidigene autotemcel, was developed by scientists of the San Raffaele-Telethon Institute for Gene Therapy in Milan. Before the Telethon Foundation assumed responsibility for the therapy, it was licensed to Orchard Therapeutics – a biotech specialising in gene therapies for rare diseases. However, in 2022, Orchard withdrew from the programme, citing strategic and commercial reasons. Telethon has submitted a marketing authorisation application (MAA) to the European Medicine’s Agency (EMA) for evaluating the gene therapy. Etuvetidigene autotemcel is designed as a one-time treatment for patients who lack a matched family donor for haematopoietic stem cell transplantation – the current standard of care for Wiskott-Aldrich syndrome. The therapy consists of autologous CD34+ stem and progenitor cells modified with a lentiviral vector encoding the WAS gene. The rare disorder affects platelet production and immune system function, often leading to severe infections, bleeding, and autoimmune complications. To date, 30 patients with Wiskott-Aldrich syndrome have been treated with the therapy – 27 within the clinical development programme, and three through an early access scheme. The therapy has been available in Italy since August 2023 under a determination by the Italian Medicines Agency (AIFA), which allows its use in patients over six months without a matched related donor. Telethon Foundation’s director general Ilaria Villa said that the foundation is also working on a submission to the US Food and Drug Administration (FDA), to make the therapy available in the US. This submission marks the second time the charity has taken over the development of a gene therapy after a commercial partner withdrew. It previously did so with simoladagene autotemcel, a therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCiD). That gene therapy also originated in the laboratories of the San Raffaele-Telethon Institute in Milan and was the subject of an industrial partnership with Orchard. According to Telethon, it is the first charity in the world to assume responsibility for the production and distribution of a drug. One of the key challenges associated with gene therapy development is funding. Due to small patient populations and uncertain commercial returns, venture capitalists and private investors are sometimes hesitant to support such therapies beyond early-stage clinical trials. While grants can help fund initial research, they rarely cover the full development and approval process, which requires significantly more investment. Another barrier is the high cost of commercial-grade manufacturing. The production of gene therapies requires advanced technology, stringent quality controls, and specialised facilities, making them expensive to scale. If a gene therapy is deemed commercially unviable, its development may be paused or abandoned. At the end of 2024, Pfizer terminated its partnership with Sangamo Therapeutics on a haemophilia A gene therapy. Pfizer originally licensed the therapy from Sangamo in 2017, and it successfully passed a Phase III clinical trial meeting its primary goal. However, Pfizer decided not to pursue approval due to questions about its potential commercial performance. To address these challenges, the EMA launched a pilot programme in 2022 to support academic and non-profit developers of advanced therapy medicinal products (ATMPs). While it does not introduce new regulatory mechanisms, the initiative aims to help non-profit organisations navigate existing regulatory pathways. Participants benefit from fee reductions and waivers for scientific advice, MAA submissions, and pre-authorisation inspections. The EMA expects to publish initial results from the pilot this year, outlining key findings and potential areas for regulatory improvement. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy