–Regulatory filing of Phase 1/2 clinical study for Alpha-1 Antitrypsin Deficiency (AATD) submitted to Australian Bellberry Human Research Ethics Committee (HREC)
-Clinical Advisory Board of lung and liver experts formed to support development of KRRO-110
-First participant dosing anticipated in first quarter of 2025
-Interim readout expected in second half of 2025 and completion of Phase 1/2 study anticipated in 2026
CAMBRIDGE, MA, USA I November 04, 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 a submission to the Bellberry HREC for a Phase 1/2 clinical study of KRRO-110 for AATD. A Clinical Advisory Board (CAB) comprised of distinguished lung and liver researchers and experts in AATD has also been assembled to help guide the clinical development strategy for KRRO-110.
“This regulatory filing reflects Korro’s transition to a clinical-stage company, and another important milestone in the development of KRRO-110,” said Kemi Olugemo, MD, Chief Medical Officer at Korro. “There remains significant unmet medical need for people living with AATD, and based on our preclinical data, KRRO-110 has the potential to be a best-in-class therapy given our differentiated approach for RNA editing. The breadth of pulmonary and hepatic expertise in our CAB will be invaluable to our clinical and regulatory strategy, ensuring we consider the holistic needs of patients with AATD. We are honored to work alongside the world’s leading experts in AATD who share our commitment to scientific excellence and improving patient outcomes.”
Subject to approval by the HREC and acceptance of the clinical trial notification (CTN) by Australia’s Therapeutic Goods Administration (TGA), Korro anticipates dosing the first participant in the Phase 1/2 study in the first quarter of 2025. An interim data readout for the Phase 1/2 study of KRRO-110 is anticipated in the second half of 2025, and completion of the study is expected in 2026.
“This is a major achievement for a company founded on RNA editing – to go from initial concepts and ideas to a first-in-human clinical study. I am immensely proud of the progress we have made since nomination of KRRO-110 for AATD in December 2023 and the contributions of the entire team at Korro,” added Ram Aiyar, PhD, CEO and President of Korro. “Our robust pipeline of fully owned and partnered programs showcases the transformative potential of our OPERA™ platform. With a strong balance sheet to support completion of the Phase 1/2 study and to advance the next set of programs, I am excited to see the potential of our platform realized.”
The newly appointed CAB members, listed below, include:
- Daniel Chambers, MBBS (Hons 1, University Medal), MRCP, FRACP, MD, FQA, Professor of Medicine, Thoracic & Transplant Physician, Interstitial Lung Disease Expert and Translational Clinician Researcher at The University of Queensland and The Prince Charles Hospital, Australia. Prof. Chambers is an internationally recognized authority in the fields of lung fibrosis, cell therapy for lung disease and lung transplantation.
- Monica Goldklang, MD, Assistant Professor of Medicine in Anesthesiology and Director, Alpha-1 Foundation Clinical Resource Center, Columbia University New York, USA. Dr. Goldklang’s main area of research interest is in translational studies, investigating the pathogenesis of smoking related lung disease and mechanisms of protease upregulation in lung injury.
- Noel G. McElvaney, MBBCh, FRCPI, DSc, Professor of Medicine at the Royal College of Surgeons in Ireland, Head of the Irish Centre for Genetic Lung Disease, Beaumont Hospital, Dublin, Ireland, and Founder of the Alpha-1 Foundation of Ireland. Prof. McElvaney’s main research areas are in AATD, chronic obstructive lung disease and cystic fibrosis.
- Pavel Strnad, MD, Professor of Translational Gastroenterology and Senior Physician at the University Hospital Aachen, Department of Medicine III, Aachen, Germany. Dr. Strnad currently leads a European initiative for the study of AATD-associated liver disease.
- Jeffrey Teckman, MD, Professor of Pediatrics, Biochemistry and Molecular Biology at Saint Louis University, USA. Dr. Teckman’s research with the Alpha-1 Foundation’s Liver Initiative focuses on adult Alphas, and his work with the Childhood Liver Disease Research and Education Network (ChiLDREN) focuses on rare pediatric liver disease.
- Alice M. Turner, MBChB (Hons), MRCP, PGCE (MedEd), PhD, Professor of Respiratory Medicine and Department Director of Research Knowledge and Transfer at the University of Birmingham Institute of Applied Health Research, UK. Dr. Turner’s research interests are predominantly in clinical aspects of AATD and chronic obstructive pulmonary disease, fields in which she has published widely.
For full CAB member bios, visit www.korrobio.com/our-team/.
About alpha-1 antitrypsin deficiency (AATD) and KRRO-110
AATD is a genetic disorder 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’s (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: 3,376
–Regulatory filing of Phase 1/2 clinical study for Alpha-1 Antitrypsin Deficiency (AATD) submitted to Australian Bellberry Human Research Ethics Committee (HREC)
-Clinical Advisory Board of lung and liver experts formed to support development of KRRO-110
-First participant dosing anticipated in first quarter of 2025
-Interim readout expected in second half of 2025 and completion of Phase 1/2 study anticipated in 2026
CAMBRIDGE, MA, USA I November 04, 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 a submission to the Bellberry HREC for a Phase 1/2 clinical study of KRRO-110 for AATD. A Clinical Advisory Board (CAB) comprised of distinguished lung and liver researchers and experts in AATD has also been assembled to help guide the clinical development strategy for KRRO-110.
“This regulatory filing reflects Korro’s transition to a clinical-stage company, and another important milestone in the development of KRRO-110,” said Kemi Olugemo, MD, Chief Medical Officer at Korro. “There remains significant unmet medical need for people living with AATD, and based on our preclinical data, KRRO-110 has the potential to be a best-in-class therapy given our differentiated approach for RNA editing. The breadth of pulmonary and hepatic expertise in our CAB will be invaluable to our clinical and regulatory strategy, ensuring we consider the holistic needs of patients with AATD. We are honored to work alongside the world’s leading experts in AATD who share our commitment to scientific excellence and improving patient outcomes.”
Subject to approval by the HREC and acceptance of the clinical trial notification (CTN) by Australia’s Therapeutic Goods Administration (TGA), Korro anticipates dosing the first participant in the Phase 1/2 study in the first quarter of 2025. An interim data readout for the Phase 1/2 study of KRRO-110 is anticipated in the second half of 2025, and completion of the study is expected in 2026.
“This is a major achievement for a company founded on RNA editing – to go from initial concepts and ideas to a first-in-human clinical study. I am immensely proud of the progress we have made since nomination of KRRO-110 for AATD in December 2023 and the contributions of the entire team at Korro,” added Ram Aiyar, PhD, CEO and President of Korro. “Our robust pipeline of fully owned and partnered programs showcases the transformative potential of our OPERA™ platform. With a strong balance sheet to support completion of the Phase 1/2 study and to advance the next set of programs, I am excited to see the potential of our platform realized.”
The newly appointed CAB members, listed below, include:
- Daniel Chambers, MBBS (Hons 1, University Medal), MRCP, FRACP, MD, FQA, Professor of Medicine, Thoracic & Transplant Physician, Interstitial Lung Disease Expert and Translational Clinician Researcher at The University of Queensland and The Prince Charles Hospital, Australia. Prof. Chambers is an internationally recognized authority in the fields of lung fibrosis, cell therapy for lung disease and lung transplantation.
- Monica Goldklang, MD, Assistant Professor of Medicine in Anesthesiology and Director, Alpha-1 Foundation Clinical Resource Center, Columbia University New York, USA. Dr. Goldklang’s main area of research interest is in translational studies, investigating the pathogenesis of smoking related lung disease and mechanisms of protease upregulation in lung injury.
- Noel G. McElvaney, MBBCh, FRCPI, DSc, Professor of Medicine at the Royal College of Surgeons in Ireland, Head of the Irish Centre for Genetic Lung Disease, Beaumont Hospital, Dublin, Ireland, and Founder of the Alpha-1 Foundation of Ireland. Prof. McElvaney’s main research areas are in AATD, chronic obstructive lung disease and cystic fibrosis.
- Pavel Strnad, MD, Professor of Translational Gastroenterology and Senior Physician at the University Hospital Aachen, Department of Medicine III, Aachen, Germany. Dr. Strnad currently leads a European initiative for the study of AATD-associated liver disease.
- Jeffrey Teckman, MD, Professor of Pediatrics, Biochemistry and Molecular Biology at Saint Louis University, USA. Dr. Teckman’s research with the Alpha-1 Foundation’s Liver Initiative focuses on adult Alphas, and his work with the Childhood Liver Disease Research and Education Network (ChiLDREN) focuses on rare pediatric liver disease.
- Alice M. Turner, MBChB (Hons), MRCP, PGCE (MedEd), PhD, Professor of Respiratory Medicine and Department Director of Research Knowledge and Transfer at the University of Birmingham Institute of Applied Health Research, UK. Dr. Turner’s research interests are predominantly in clinical aspects of AATD and chronic obstructive pulmonary disease, fields in which she has published widely.
For full CAB member bios, visit www.korrobio.com/our-team/.
About alpha-1 antitrypsin deficiency (AATD) and KRRO-110
AATD is a genetic disorder 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’s (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: 3,376
