Cambridge, Mass., November 21, 2024 — Korro Bio, Inc. (Korro) (NASDAQ: KRRO) has received approval from the Australian Bellberry Human Research Ethics Committee (HREC) and clearance from the Australian Therapeutic Goods Administration (TGA) to initiate a phase 1/2a clinical study of Krro-110 for Alpha-1 Antitrypsin Deficiency (AATD).
The rewrite study, which will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple escalating doses of Krro-110, is expected to include up to 64 participants, including healthy adults and clinically stable AATD patients with the PIZZ genotype.
The first participant dosing is anticipated in the first quarter of 2025, and completion of the study is expected in 2026. An interim readout in PIZZ participants is anticipated in the second half of 2025.
Korro's Chief Medical Officer, Kemi Olugemo, MD, stated that multiple dose treatment with Krro-110 in a human transgenic mouse model of PIZZ genotype achieved greater than 60% editing and resulted in secretion of functional M-AAT at therapeutically relevant levels.
Furthermore, Korro's Krro-110 is designed to co-opt an endogenous enzyme, adenosine deaminase acting on RNA (ADAR), to edit the "A" variant on serpina1 RNA, repair an amino acid codon, and restore secretion of normal AAT protein. The repair of the endogenous protein has the potential to clear protein aggregates from within liver cells to create a potentially clinically differentiated benefit for liver function and to preserve lung function by providing an adequate amount of normal AAT protein.
Today the company's shares have moved 3.2% to a price of $48.49. If you want to know more, read the company's complete 8-K report here.
