Korro Bio’s KRRO-110 Receives Orphan Drug Designation from FDA for Alpha-1 Antitrypsin Deficiency
Cambridge, MA, March 14, 2025 – Korro Bio, Inc. (Nasdaq: KRRO), a pioneering clinical-stage biopharmaceutical company specializing in the development of RNA editing medicines for both rare and prevalent diseases, recently announced significant progress in its research and development efforts. The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the investigational medicine KRRO-110 for the treatment of Alpha-1 Antitrypsin Deficiency (AATD).
What is Alpha-1 Antitrypsin Deficiency (AATD)?
Alpha-1 Antitrypsin Deficiency is a rare, genetic disorder that affects approximately 1 in 1,500 to 1 in 5,000 individuals of European descent. It is characterized by a deficiency in the production or function of Alpha-1 Antitrypsin (AAT), a protein produced in the liver that helps protect the lungs from damage. As a result, individuals with AATD are susceptible to developing lung diseases such as emphysema and chronic obstructive pulmonary disease (COPD) at a much younger age than the general population.
About KRRO-110
KRRO-110 is an investigational RNA editing medicine designed to address the underlying cause of AATD by increasing the production of functional AAT protein in the liver. This is achieved by editing the patient’s own cells to produce a correctly folded AAT protein. By doing so, KRRO-110 has the potential to treat the root cause of the disease, offering a more effective and sustainable approach compared to current treatments, which typically involve regular infusions of exogenous AAT protein.
Impact on Individuals with AATD
For individuals with AATD, the granting of orphan drug designation to KRRO-110 comes as welcome news. Orphan drug designation provides several benefits, including tax incentives, extended market exclusivity, and waiver of certain regulatory fees. These incentives encourage pharmaceutical companies to invest in the development of treatments for rare diseases, where the market size and financial returns may not be as attractive as more common conditions. In this case, the designation signals that the FDA recognizes the potential of KRRO-110 to address an unmet medical need and improve the lives of those affected by AATD.
Global Implications
Beyond the immediate impact on individuals with AATD, the granting of orphan drug designation to KRRO-110 also has broader implications. It represents a significant step forward in the development of RNA editing medicines as a new class of therapeutics. By targeting the underlying genetic causes of diseases, RNA editing medicines have the potential to treat a wide range of genetic disorders, both rare and prevalent. This advancement could lead to a paradigm shift in the way we approach the treatment of genetic diseases, offering hope to millions of people worldwide.
Conclusion
In conclusion, the granting of orphan drug designation to Korro Bio’s KRRO-110 for the treatment of Alpha-1 Antitrypsin Deficiency marks an important milestone in the development of RNA editing medicines as a new class of therapeutics. This designation not only offers hope to the individuals with AATD but also signals the potential for these innovative treatments to address a wide range of genetic disorders, ultimately improving the lives of millions of people around the world.
- Korro Bio’s KRRO-110 receives orphan drug designation from the FDA for Alpha-1 Antitrypsin Deficiency
- AATD is a rare genetic disorder affecting approximately 1 in 1,500 to 1 in 5,000 individuals of European descent
- KRRO-110 is an investigational RNA editing medicine designed to increase the production of functional AAT protein in the liver
- Orphan drug designation provides incentives for pharmaceutical companies to invest in the development of treatments for rare diseases
- RNA editing medicines have the potential to treat a wide range of genetic disorders, both rare and prevalent
