Airna Biotech secures $60M for RNA editing drugs

Airna, an RNA editing startup, has secured an additional $60 million in funding to advance its Series A round. The Boston and Germany-based biotech firm focuses on developing genetic medicines targeting lung, cardiovascular, metabolic, and blood diseases. Airna launched in September last year with an initial funding of $30 million, aiming to create a treatment for alpha-1 antitrypsin deficiency (AATD), a rare inherited disorder. According to CEO Kris Elverum, this new funding will help the company bring its AATD drug candidate to clinical trials as soon as next year.

Airna promotes its RNA-based drug development approach as a safer alternative to direct DNA editing methods like CRISPR. CEO Elverum likens DNA to the "hardware" of a cell, providing the foundational structure, whereas RNA is the "software" that drives cellular operations. The company's scientific foundation is built around enzymes called adenosine deaminases acting on RNA (ADAR), which play a role in gene modification and regulation.

The lead project for Airna aims to correct the genetic mutation causing AATD, a condition impacting the liver or lungs and leading to ailments such as chronic obstructive pulmonary disease (COPD). It is estimated that up to 100,000 people in the United States suffer from AATD. Current treatment options depend on symptoms and may include bronchodilators, antibiotics, and sometimes injections of replacement AAT protein. However, Elverum points out that an infrequent subcutaneous injection capable of addressing the underlying disease would be a more favorable option.

Elverum emphasizes the advantages of targeting RNA, stating that it allows the company to address root causes of diseases while offering a cost-effective treatment solution suitable for large-scale diseases and early-stage intervention. The additional Series A funding was spearheaded by European life sciences investor Forbion and included contributions from Arch Venture Partners, Ono Venture Investment, Alexandria Venture Investments, and ND Capital. Rodger Novak, co-founder of CRISPR Therapeutics, chairs the company’s board of directors.

Several companies are exploring RNA-modulating therapies for AATD. Wave Life Sciences has initiated a clinical trial for its treatment, while Sanofi's INBRX-101, which was acquired in January and is in Phase 2 trials, is among the most advanced. Arrowhead Pharmaceuticals, in collaboration with Takeda, is developing an experimental drug targeting liver disease.

Airna's innovative approach to RNA editing and strong backing from investors position it as a promising player in the rapidly evolving field of genetic medicine. By focusing on safer RNA-based treatments, the company aims to offer more effective and accessible therapies for a range of serious health conditions.

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