Core Insights - Korro Bio, Inc. has received orphan drug designation from the FDA for its investigational medicine KRRO-110, aimed at treating Alpha-1 Antitrypsin Deficiency (AATD) [1][2] - KRRO-110 is the first RNA editing candidate from Korro's OPERA platform and is currently in Phase 1/2a clinical trials [2][4] - AATD is a genetic disorder that can lead to serious health issues, including pulmonary emphysema and hepatic cirrhosis [5] Company Overview - Korro Bio is a clinical-stage biopharmaceutical company focused on developing genetic medicines through RNA editing for both rare and prevalent diseases [6] - The company aims to leverage its proprietary RNA editing platform to create precise and transient genetic modifications, enhancing the specificity and tolerability of treatments [6] Clinical Development - The REWRITE study is a two-part clinical trial evaluating the safety and tolerability of KRRO-110 in up to 64 participants, with interim data expected in the second half of 2025 [4] - The study includes healthy adults and clinically stable AATD patients, focusing on pharmacokinetic and pharmacodynamic parameters to guide future dosing [4] Regulatory Insights - The FDA's orphan drug designation provides various incentives, including tax credits for clinical testing and potential market exclusivity post-approval [3] - This designation highlights the urgent need for new treatments for AATD patients, emphasizing the potential impact of KRRO-110 [2][3]
KRRO-110 Receives Orphan Drug Designation from U.S. FDA for Alpha-1 Antitrypsin Deficiency