Korro Bio receives EMA orphan drug designation for AATD treatment

CAMBRIDGE, Mass. - Korro Bio, Inc. (NASDAQ:KRRO), a clinical-stage biopharmaceutical company with a market capitalization of $128 million and strong liquidity position, announced Monday that the European Medicines Agency (EMA) Committee has granted orphan drug designation for KRRO-110, its investigational treatment for Alpha-1 Antitrypsin Deficiency (AATD). According to InvestingPro data, the company maintains a healthy current ratio of 9.41, with cash reserves exceeding its debt obligations.

This European designation follows the U.S. Food and Drug Administration’s orphan drug designation for KRRO-110 in March 2025. The therapy is currently being evaluated in the Phase 1/2a REWRITE clinical study, with an interim readout expected in the second half of 2025. While the company’s stock has experienced a significant decline of 70% over the past year, InvestingPro analysis suggests the stock is currently undervalued based on its Fair Value assessment.

"The EMA orphan drug designation for KRRO-110 is a significant milestone for Korro and highlights an urgent need to bring innovative solutions to people with AATD seeking new, disease-modifying therapies," said Kemi Olugemo, Chief Medical Officer at Korro, according to the company’s press release.

KRRO-110 is the first RNA editing oligonucleotide product candidate from Korro’s proprietary RNA editing platform. The therapy is designed to edit the SERPINA1 gene mutation that causes AATD, potentially restoring normal AAT protein production.

The REWRITE clinical study will evaluate the safety and tolerability of KRRO-110 in up to 64 participants, including healthy adults and AATD patients with the PiZZ genotype. Completion of the study is anticipated in 2026.

EMA orphan designation provides companies with development incentives, including protocol assistance, reduced regulatory fees, and market exclusivity upon approval. The designation is granted to medicines intended for treating diseases affecting fewer than 5 in 10,000 people in the European Union.

AATD is a genetic disorder that can lead to pulmonary emphysema and hepatic cirrhosis in affected adults. With earnings results expected on August 6, investors seeking deeper insights into Korro Bio’s financial health and growth prospects can access comprehensive analysis through InvestingPro’s detailed research reports, which include expert analysis of the company’s development pipeline and financial metrics.

In other recent news, Korro Bio has been the subject of notable analyst activity. H.C. Wainwright adjusted its 12-month price target for Korro Bio from $115 to $100, maintaining a Buy rating on the shares. This revision reflects market dynamics and anticipated regulatory challenges, particularly with the appointment of Vinay Prasad at the FDA’s Center for Biologics and Evaluations Research. Despite these concerns, Korro Bio’s key product, KRRO-110, will be reviewed by the Center for Drug Evaluations and Research, potentially mitigating some regulatory risks. Additionally, Korro Bio’s decision to reduce its workforce by 20% to extend its financial runway has been factored into H.C. Wainwright’s updated expectations.

Meanwhile, Cantor Fitzgerald initiated coverage of Korro Bio with an Overweight rating and set a 12-month price target of $74. The firm highlights Korro’s promising treatment, KRRO-110, which is being developed for Alpha-1 antitrypsin deficiency, as a significant potential player in the RNA editing oligonucleotide therapeutics field. Cantor Fitzgerald’s analysis suggests that KRRO-110 could become a leading treatment option in this competitive market. The firm’s confidence extends to Korro’s strategic focus and potential upcoming clinical programs, which could further enhance its position in the genetic medicine sector.

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