Korro Bio Announces Dosing of First Participants in REWRITE Phase 1/2a Study of KRRO-110 for Alpha-1 Antitrypsin Deficiency and Provides Pipeline Update
Korro Bio has initiated dosing in the REWRITE Phase 1/2a study for KRRO-110, targeting Alpha-1 Antitrypsin Deficiency (AATD). KRRO-110 aims to restore therapeutic M-AAT protein levels using the body's ADAR enzyme. The study will evaluate safety and tolerability in up to 64 participants, including healthy adults and AATD patients.
Interim data from Part 1 is expected in the second half of 2025, with study completion anticipated in 2026. The company is also advancing its pipeline, including a partnered program with Novo Nordisk in a cardiometabolic indication.
Korro's CEO, Ram Aiyar, highlighted the company's evolution to a clinical-stage organization and its strategy to take three product candidates into the clinic over the next three years. For more details, visit ClinicalTrials.gov (NCT06677307).
Korro Bio ha avviato la somministrazione nel studio REWRITE di fase 1/2a per KRRO-110, mirato a trattare la Deficienza di Alfa-1 Antitripsina (AATD). KRRO-110 ha l'obiettivo di ripristinare i livelli terapeutici della proteina M-AAT utilizzando l'enzima ADAR del corpo. Lo studio valuterà la sicurezza e la tollerabilità in un massimo di 64 partecipanti, tra cui adulti sani e pazienti con AATD.
I dati intermedi dalla Parte 1 sono attesi nella seconda metà del 2025, con la conclusione dello studio prevista per il 2026. L'azienda sta anche facendo progressi nel suo pipeline, inclusa un programma in collaborazione con Novo Nordisk per un'indicazione cardiometabolica.
Il CEO di Korro, Ram Aiyar, ha sottolineato l'evoluzione dell'azienda verso un'organizzazione in fase clinica e la sua strategia di portare tre candidati alla sperimentazione clinica nei prossimi tre anni. Per ulteriori dettagli, visitare ClinicalTrials.gov (NCT06677307).
Korro Bio ha iniciado la dosificación en el estudio REWRITE de fase 1/2a para KRRO-110, dirigido a la deficiencia de alfa-1 antitripsina (AATD). KRRO-110 tiene como objetivo restaurar los niveles terapéuticos de la proteína M-AAT utilizando la enzima ADAR del cuerpo. El estudio evaluará la seguridad y la tolerabilidad en hasta 64 participantes, incluidos adultos sanos y pacientes con AATD.
Se esperan datos interinos de la Parte 1 en la segunda mitad de 2025, con la finalización del estudio anticipada para 2026. La empresa también está avanzando en su pipeline, incluyendo un programa asociado con Novo Nordisk en una indicación cardiometabólica.
El CEO de Korro, Ram Aiyar, destacó la evolución de la empresa hacia una organización en etapa clínica y su estrategia de llevar tres candidatos de producto a la clínica en los próximos tres años. Para más detalles, visita ClinicalTrials.gov (NCT06677307).
Korro Bio는 KRRO-110에 대한 REWRITE 1/2a 단계 연구에서 투여를 시작했습니다. 이 연구는 알파-1 항트립신 결핍증 (AATD)을 겨냥하고 있습니다. KRRO-110은 신체의 ADAR 효소를 사용하여 치료적 M-AAT 단백질 수준을 복원하는 것을 목표로 합니다. 이 연구는 건강한 성인 및 AATD 환자를 포함한 최대 64명의 참가자를 대상으로 안전성과 내약성을 평가할 것입니다.
1부의 중간 데이터는 2025년 하반기에 예상되며, 연구 완료는 2026년으로 예정되어 있습니다. 회사는 또한 Novo Nordisk와의 협력 프로그램을 포함하여 파이프라인을 발전시키고 있습니다.
Korro의 CEO인 Ram Aiyar는 회사가 임상 단계 조직으로 발전했음을 강조했으며, 향후 3년 동안 세 가지 제품 후보를 임상으로 가져갈 전략을 가지고 있다고 밝혔습니다. 자세한 내용은 ClinicalTrials.gov (NCT06677307)를 방문하세요.
Korro Bio a lancé le dosage dans l'étude REWRITE de phase 1/2a pour KRRO-110, ciblant la déficience en alpha-1 antitrypsine (AATD). KRRO-110 vise à Restaurer les niveaux thérapeutiques de la protéine M-AAT en utilisant l'enzyme ADAR du corps. L'étude évaluera la sécurité et la tolérance auprès de jusqu'à 64 participants, y compris des adultes en bonne santé et des patients atteints d'AATD.
Les données intermédiaires de la Partie 1 sont attendues dans la seconde moitié de 2025, avec l'achèvement de l'étude prévu pour 2026. L'entreprise fait également des progrès dans son pipeline, y compris un programme partenaire avec Novo Nordisk dans une indication cardiométabolique.
Le PDG de Korro, Ram Aiyar, a souligné l'évolution de l'entreprise vers une organisation en phase clinique et sa stratégie visant à amener trois candidats produits à la clinique au cours des trois prochaines années. Pour plus de détails, visitez ClinicalTrials.gov (NCT06677307).
Korro Bio hat mit der Dosierung in der REWRITE Phase 1/2a-Studie für KRRO-110 begonnen, das auf Alpha-1-Antitrypsin-Mangel (AATD) abzielt. KRRO-110 hat zum Ziel, therapeutische M-AAT-Proteinspiegel unter Verwendung des ADAR-Enzyms des Körpers wiederherzustellen. Die Studie wird die Sicherheit und Verträglichkeit bei bis zu 64 Teilnehmern bewerten, darunter gesunde Erwachsene und AATD-Patienten.
Die Zwischenanalysen aus Teil 1 werden in der zweiten Hälfte von 2025 erwartet, während der Abschluss der Studie für 2026 vorgesehen ist. Das Unternehmen entwickelt auch sein Portfolio weiter, einschließlich eines Partnerschaftsprogramms mit Novo Nordisk in einer kardiometabolischen Indikation.
Korros CEO, Ram Aiyar, hob die Entwicklung des Unternehmens zu einer klinischen Organisation sowie die Strategie hervor, in den nächsten drei Jahren drei Produktkandidaten in die Klinik zu bringen. Weitere Details finden Sie auf ClinicalTrials.gov (NCT06677307).
- Initiation of dosing in REWRITE study for KRRO-110.
- KRRO-110 aims to restore therapeutic M-AAT protein levels in AATD patients.
- Interim data expected in the second half of 2025.
- Korro advancing additional pipeline programs, including a partnered program with Novo Nordisk.
- Company aims to bring three product candidates to the clinic over the next three years.
- Study completion anticipated only in 2026, indicating a lengthy timeline.
Insights
The initiation of the REWRITE Phase 1/2a study marks a critical milestone in KRRO-110's development pathway. The trial's design is robust, targeting up to 64 participants across both healthy volunteers and AATD patients with the PiZZ genotype. The dual-part structure (single and multiple ascending doses) is strategically crafted to establish both safety parameters and early efficacy signals.
The focus on pharmacokinetic and pharmacodynamic endpoints is particularly noteworthy for potential investors, as these will provide important data points for dose optimization in later-stage trials. The planned interim readout in H2 2025 could serve as a significant catalyst for the stock, especially if the data demonstrates successful RNA editing and protein level restoration.
In simpler terms: Think of this like test-driving a new car model - first with professional drivers (healthy volunteers) to ensure basic safety, then with actual customers (AATD patients) to confirm it meets their needs. The interim results will tell us if the "car" is working as designed.
The advancement of KRRO-110 represents a strategic positioning in the RNA editing space, with Korro's OPERA platform potentially offering advantages over traditional gene therapy approaches. The company's "3-2-1 strategy" through 2027, aiming to bring three products to clinic across two tissue types using one platform, demonstrates ambitious but focused pipeline development.
The partnership with Novo Nordisk in cardiometabolic indications adds significant credibility and potential future revenue streams. The platform's versatility, spanning both rare and prevalent diseases, suggests broader commercial opportunities than typical rare disease-focused biotechs.
For the average investor: Imagine Korro as a company that's built a new type of Swiss Army knife for genetic diseases - they're now testing the first blade (KRRO-110), but the same tool could potentially fix many different genetic "problems." The Novo Nordisk partnership is like having a major car manufacturer validate your new engine design.
- KRRO-110 is the first product candidate from Korro’s proprietary RNA editing OPERATM platform
- Interim readout from REWRITE expected in the second half of 2025
- Korro to advance additional pipeline programs towards clinical development
CAMBRIDGE, Mass., Jan. 13, 2025 (GLOBE NEWSWIRE) -- Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent diseases, today announced the initiation of dosing in its REWRITE study investigating KRRO-110 as a potential treatment for individuals with Alpha-1 Antitrypsin Deficiency (AATD).
“KRRO-110 is designed to restore therapeutic M-AAT protein levels in individuals with at least one Z allele by leveraging the body’s endogenous ADAR enzyme. The swift initiation of the REWRITE study for AATD is a testament to our collective strength and focused execution at Korro,” said Kemi Olugemo, MD, Chief Medical Officer at Korro. “We are encouraged by our preclinical data, which demonstrates rapid restoration of functional AAT protein levels and durable editing. Our goal is to create a clinically differentiated, disease-modifying treatment to address both lung and liver manifestations of AATD. We look forward to evaluating the therapeutic potential of KRRO-110 and sharing interim data in the second half of this year.”
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. Interim data from Part 1 (single ascending doses in healthy volunteers and individuals with AATD) is expected in the second half of 2025, and completion of the study is anticipated in 2026. For additional information about the REWRITE study, visit ClinicalTrials.gov (NCT06677307).
In addition to advancing KRRO-110, Korro is progressing a pipeline of wholly owned programs and a Novo Nordisk partnered program in a cardiometabolic indication towards the clinic.
“We have evolved from a research organization to a clinical-stage drug development organization with our first participant in the REWRITE study recently dosed with KRRO-110. We have taken time to build our pipeline to demonstrate the versatility of RNA editing, building on data from other oligonucleotide programs,” said Ram Aiyar, PhD, President and CEO. “Over the next three years, we expect to take three product candidates into the clinic, in two tissue types with a single modular platform. This will be the crux of our 3-2-1 strategy through 2027. I am excited about the prospect that RNA editing holds beyond the treatment of rare genetic disease and applying our platform to highly prevalent diseases.”
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 adults 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 clinical-stage 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, enabling 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.
Korro intends to use its Investor Relations website as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor Korro’s Investor Relations website, in addition to following Korro’s press releases, SEC filings, public conference calls, presentations, and webcasts.
Forward-Looking Statements
Certain statements in this press release may constitute “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements include, but are not limited to, express or implied statements regarding expectations, hopes, beliefs, intentions or strategies of Korro regarding the future including, without limitation, express or implied statements regarding: the timing of the interim data readout and completion of the Phase 1/2a clinical study of KRRO-110 for AATD (REWRITE), including Korro’s ability to complete the Phase 1/2a clinical study; KRRO-110’s potential to treat individuals with AATD; KRRO-110’s ability to restore therapeutic M-AAT protein levels; KRRO’s potential as a clinically differentiated, disease-modifying treatment to address both lung and liver manifestations of AATD; Korro’s ability to progress a pipeline of programs towards the clinic; the timing of, and Korro’s ability to, deliver three product candidates in two tissue types with a single modular platform by the end of 2027; among others. In addition, any statements that refer to projections, forecasts, or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “strive,” “would,” “aim,” “target,” “commit,” and similar expressions may identify forward-looking statements, but the absence of these words does not mean that statement is not forward looking. Forward-looking statements are based on current expectations and assumptions that, while considered reasonable are inherently uncertain. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Factors that may cause actual results to differ materially from current expectations include, but are not limited to, various factors beyond management’s control including risks of conducting a first-in-human clinical study; challenges with addressing any regulatory concerns necessary to proceed with enrollment and dosing; risks associated with enrolling sufficient participants and other risks inherent in biopharmaceutical development; risks associated with conducting pre-clinical studies and clinical studies and risks of replicating results from pre-clinical studies in clinical studies; and other risks associated with obtaining regulatory approvals and protecting intellectual property; as well as risks associated with general economic conditions; and other risks and uncertainties indicated from time to time in Korro’s filings with the SEC, including Part II Item 1A. “Risk Factors” in Korro’s most recent Quarterly Report on Form 10-Q filed with the SEC, as such may be amended or supplemented by its other filings with the SEC. Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. You should not place undue reliance on forward-looking statements in this press release, which speak only as of the date they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by law, Korro does not undertake or accept any duty to release publicly any updates or revisions to any forward-looking statements to reflect any change in its expectations or in the events, conditions or circumstances on which any such statement is based. This press release does not purport to summarize all of the conditions, risks and other attributes of an investment in Korro.
Korro Bio Contact Information
Investor & Media Contact
Tim Palmer
IR@korrobio.com
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