-REWRITE study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple escalating doses of KRRO-110
-First participant dosing anticipated in the first quarter of 2025
-Interim readout expected in second half of 2025 and completion of Phase 1/2a study anticipated in 2026
CAMBRIDGE, MA, USA I November 21, 2024 I Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent diseases, today announced that it 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).
“We are thrilled to receive approval to proceed with our clinical study in Australia,” said Kemi Olugemo, MD, Chief Medical Officer at Korro. “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. This approval allows us to demonstrate the potential of KRRO-110 in patients with the PiZZ genotype who remain at risk for serious lung and liver complications despite receiving standard-of-care treatment.”
About REWRITE
REWRITE is a two-part single and multiple dose-escalating study that will evaluate the safety and tolerability of KRRO-110 in up to 64 participants, including healthy adults and clinically stable AATD patients with the PiZZ genotype. Secondary and exploratory endpoints include pharmacokinetic and pharmacodynamic parameters that will guide optimal dose selection for later stage studies.
Korro expects to dose the first participant 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.
For additional information about the REWRITE study, visit ClinicalTrials.gov (NCT06677307).
About Alpha-1 Antitrypsin Deficiency (AATD) and KRRO-110
AATD is a genetic disorder most commonly caused by a single missense mutation (G-to-A) in the SERPINA1 gene. Affected adult individuals experience pulmonary emphysema and/or hepatic cirrhosis, as well as end organ manifestations. KRRO-110 is the first RNA editing oligonucleotide product candidate from Korro’s proprietary RNA editing platform, Oligonucleotide Promoted Editing of RNA (OPERA™). 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. This 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.
About Korro
Korro is a biopharmaceutical company focused on developing a new class of genetic medicines for both rare and highly prevalent diseases using its proprietary RNA editing platform. Korro is generating a portfolio of differentiated programs that are designed to harness the body’s natural RNA editing process to affect a precise yet transient single base edit. By editing RNA instead of DNA, Korro is expanding the reach of genetic medicines by delivering additional precision and tunability, which has the potential for increased specificity and improved long-term tolerability. Using an oligonucleotide-based approach, Korro expects to bring its medicines to patients by leveraging its proprietary platform with precedented delivery modalities, manufacturing know-how, and established regulatory pathways of approved oligonucleotide drugs. Korro is based in Cambridge, Massachusetts. For more information, visit korrobio.com.
SOURCE: Korro
Post Views: 1,242
-REWRITE study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple escalating doses of KRRO-110
-First participant dosing anticipated in the first quarter of 2025
-Interim readout expected in second half of 2025 and completion of Phase 1/2a study anticipated in 2026
CAMBRIDGE, MA, USA I November 21, 2024 I Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent diseases, today announced that it 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).
“We are thrilled to receive approval to proceed with our clinical study in Australia,” said Kemi Olugemo, MD, Chief Medical Officer at Korro. “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. This approval allows us to demonstrate the potential of KRRO-110 in patients with the PiZZ genotype who remain at risk for serious lung and liver complications despite receiving standard-of-care treatment.”
About REWRITE
REWRITE is a two-part single and multiple dose-escalating study that will evaluate the safety and tolerability of KRRO-110 in up to 64 participants, including healthy adults and clinically stable AATD patients with the PiZZ genotype. Secondary and exploratory endpoints include pharmacokinetic and pharmacodynamic parameters that will guide optimal dose selection for later stage studies.
Korro expects to dose the first participant 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.
For additional information about the REWRITE study, visit ClinicalTrials.gov (NCT06677307).
About Alpha-1 Antitrypsin Deficiency (AATD) and KRRO-110
AATD is a genetic disorder most commonly caused by a single missense mutation (G-to-A) in the SERPINA1 gene. Affected adult individuals experience pulmonary emphysema and/or hepatic cirrhosis, as well as end organ manifestations. KRRO-110 is the first RNA editing oligonucleotide product candidate from Korro’s proprietary RNA editing platform, Oligonucleotide Promoted Editing of RNA (OPERA™). 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. This 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.
About Korro
Korro is a biopharmaceutical company focused on developing a new class of genetic medicines for both rare and highly prevalent diseases using its proprietary RNA editing platform. Korro is generating a portfolio of differentiated programs that are designed to harness the body’s natural RNA editing process to affect a precise yet transient single base edit. By editing RNA instead of DNA, Korro is expanding the reach of genetic medicines by delivering additional precision and tunability, which has the potential for increased specificity and improved long-term tolerability. Using an oligonucleotide-based approach, Korro expects to bring its medicines to patients by leveraging its proprietary platform with precedented delivery modalities, manufacturing know-how, and established regulatory pathways of approved oligonucleotide drugs. Korro is based in Cambridge, Massachusetts. For more information, visit korrobio.com.
SOURCE: Korro
Post Views: 1,242