Metagenomi Presents Novel, Highly Specific and Efficient Adenine Base Editors for Broad Genome Editing at ESGCT
Metagenomi presented groundbreaking results for its Adenine Base Editors (ABEs) at the European Society of Gene and Cell Therapy Annual Congress in Rome. The company's ABE platform demonstrated exceptional performance across key metrics: over 95% genome targetability, achieving 95% protein knockdown in primary T-cells through triplex editing, and highly specific on-target deamination with no detectable translocations.
The platform showed excellent tolerability with no adverse effects on cell viability or expansion. Notable advantages include compatibility with various non-viral methods of site-specific template integration, enabling an all-in-one editing solution for next-generation cell therapies. This positions Metagenomi's ABE technology as a promising tool for both in vivo genome editing programs and ex vivo cell therapy applications.
Metagenomi ha presentato risultati rivoluzionari per i suoi Adenine Base Editors (ABE) durante il Congresso Annuale della Società Europea di Terapia Genica e Cellulare a Roma. La piattaforma ABE dell'azienda ha dimostrato prestazioni eccezionali in vari parametri chiave: oltre il 95% di targetabilità genomica, raggiungendo il 95% di abbattimento delle proteine nelle cellule T primarie tramite editing triplo, e deaminazione altamente specifica sul bersaglio senza traslocazioni rilevabili.
La piattaforma ha mostrato un'ottima tollerabilità senza effetti avversi sulla vitalità o sull'espansione cellulare. Tra i vantaggi notevoli c'è la compatibilità con vari metodi non virali di integrazione di modelli specifici, che consente una soluzione di editing tutto-in-uno per le terapie cellulari di nuova generazione. Questo posiziona la tecnologia ABE di Metagenomi come uno strumento promettente sia per i programmi di editing genico in vivo che per le applicazioni di terapia cellulare ex vivo.
Metagenomi presentó resultados innovadores para sus Editores de Bases de Adenina (ABE) en el Congreso Anual de la Sociedad Europea de Terapia Génica y Celular en Roma. La plataforma ABE de la compañía demostró un desempeño excepcional en métricas clave: más del 95% de capacidad de apuntar al genoma, logrando un 95% de reducción de proteínas en células T primarias a través de edición de triplos, y desaminación altamente específica en el objetivo sin translocaciones detectables.
La plataforma mostró una excelente tolerancia sin efectos adversos en la viabilidad o expansión celular. Entre las ventajas notables se encuentra la compatibilidad con varios métodos no virales de integración de plantillas específicas, lo que permite una solución de edición todo-en-uno para las terapias celulares de próxima generación. Esto posiciona la tecnología ABE de Metagenomi como una herramienta prometedora tanto para programas de edición genómica in vivo como para aplicaciones de terapia celular ex vivo.
메타제노미는 로마에서 열린 유럽 유전자 및 세포 치료학회 연례 회의에서 아데닌 염기 편집기(ADE)에 대한 혁신적인 결과를 발표했습니다. 회사의 ABE 플랫폼은 주요 지표에서 뛰어난 성능을 보여주었습니다: 95% 이상의 유전체 표적화, 삼중 편집을 통해 원형 T 세포에서 95%의 단백질 감소를 달성했으며, 검출 가능한 전위 이동 없이 높은 정확도의 표적 위에서 탈아미노화가 일어났습니다.
이 플랫폼은 세포 생존성이나 확장에 부정적인 영향을 주지 않는 우수한 내성을 보여주었습니다. 주목할 만한 장점은 특정 부위에서 비바이러스 방법과의 호환성으로, 차세대 세포 치료를 위한 올인원 편집 솔루션을 가능하게 합니다. 이는 메타제노미의 ABE 기술이 in vivo 유전체 편집 프로그램과 ex vivo 세포 치료 응용 프로그램 모두를 위한 유망한 도구로 자리 잡게 합니다.
Metagenomi a présenté des résultats révolutionnaires pour ses Éditeurs de Bases d'Adenine (ABE) lors du Congrès Annuel de la Société Européenne de Thérapie Génique et Cellulaire à Rome. La plateforme ABE de l'entreprise a démontré des performances exceptionnelles dans des indicateurs clés : plus de 95% de ciblage génomique, réalisant 95% de réduction des protéines dans les cellules T primaires grâce à l'édition triplex, et une désamination hautement spécifique sur cible sans translocations détectables.
La plateforme a montré une tolérance excellente sans effets indésirables sur la viabilité ou l'expansion cellulaire. Parmi les avantages notables, on trouve la compatibilité avec diverses méthodes non virales d'intégration de modèles spécifiques, permettant une solution d'édition tout-en-un pour les thérapies cellulaires de nouvelle génération. Cela positionne la technologie ABE de Metagenomi comme un outil prometteur tant pour les programmes d'édition du génome in vivo que pour les applications de thérapie cellulaire ex vivo.
Metagenomi präsentierte bahnbrechende Ergebnisse für seine Adenin-Baseneditoren (ABEs) auf dem Jahrestreffen der Europäischen Gesellschaft für Gen- und Zelltherapie in Rom. Die ABE-Plattform des Unternehmens zeigte außergewöhnliche Leistungen bei wichtigen Kennzahlen: über 95% Genomzielverfügbarkeit, erreicht 95% Proteinreduktion in primären T-Zellen durch Triplex-Bearbeitung, und hochspezifische On-Target-Deaminierung ohne nachweisbare Translokationen.
Die Plattform zeigte eine hervorragende Verträglichkeit ohne nachteilige Auswirkungen auf die Zellviabilität oder -expansion. Zu den bemerkenswerten Vorteilen gehört die Kompatibilität mit verschiedenen nicht-viralen Methoden zur standortspezifischen Template-Integration, die eine All-in-One-Bearbeitungslösung für die nächste Generation von Zelltherapien ermöglicht. Dies positioniert die ABE-Technologie von Metagenomi als vielversprechendes Werkzeug für sowohl in vivo Genom-Editing-Programme als auch ex vivo Zelltherapie-Anwendungen.
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Metagenomi Adenine Base Editors (ABEs) demonstrated over
Simultaneous ABE triplex editing resulted in over
ABE demonstrated highly specific on-target deamination with no detectable translocations and no significant genomic composition differences
ABE triplex editing had no adverse effects on cell viability, expansion, or other measures of cell health
EMERYVILLE, Calif., Oct. 24, 2024 (GLOBE NEWSWIRE) -- Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary gene editing toolbox, today presented a poster (P0533 Poster Session III) titled “Efficient and specific genome editing with metagenomics-derived base editor for human therapeutic applications” at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress in Rome, Italy.
“Base editors are powerful gene editing tools that are able to modify genes through single nucleotide changes, including multiplexed gene editing applications without risk of translocations. Our ABEs display critical attributes necessary for successful multiplexed gene editing with remarkable targetability, efficiency, precision and tolerability,” said Brian C. Thomas, PhD, CEO and founder of Metagenomi. “We remain focused on advancing our base editors for in vivo genome editing programs, while pursuing partnerships to advance ex vivo cell therapy applications.”
Today’s poster presentation described the following key advantages of the Metagenomi ABE platform:
Targetability: Metagenomi’s ABE is targetable to over
Efficiency: The ABE platform achieved over
Specificity: The ABE demonstrated highly specific on-target deamination with minimum-to-no indel formation. Genome-wide analyses confirmed no detectable translocations and no significant genomic composition differences when compared to unedited cells. This precision minimizes the risk of unintended genetic alterations, positioning the company’s ABE for long-term therapeutic applications.
Tolerability: Metagenomi’s ABE triplex protein knockdowns exhibited excellent tolerability in cells, with no adverse effects on cell viability, expansion, or changes in stress-related gene expression observed post-editing. Tolerability is especially critical in multiplex editing of primary T-cells, where maintaining cell health is vital for therapeutic efficacy.
About Metagenomi
Metagenomi is a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary, comprehensive metagenomics-derived toolbox. Metagenomi is harnessing the power of metagenomics, the study of genetic material recovered from the natural environment, to unlock four billion years of microbial evolution to discover and develop a suite of novel editing tools capable of correcting any type of genetic mutation found anywhere in the genome. Its comprehensive genome editing toolbox includes programmable nucleases, base editors, and RNA and DNA-mediated integration systems (including prime editing systems and clustered regularly interspaced short palindromic repeat associated transposases). Metagenomi believes its diverse and modular toolbox positions the company to access the entire genome and select the optimal tool to unlock the full potential of genome editing for patients. For more information, please visit https://metagenomi.co.
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This press release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions, include, but are not limited to, any statements relating to our growth strategy and product development programs, including the timing of and our ability to conduct IND-enabling studies, make regulatory filings such as INDs, statements concerning the potential of therapies and product candidates, statements concerning the timing of data presentations and publications, and any other statements that are not historical facts. Forward looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition, and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in “Risk Factors,” in our most recent Form 10-K and our most recent 10-Qs on file with the Securities and Exchange Commission. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.
Investor Contact:
Simon Harnest - CIO, SVP Investor Relations
simon@metagenomi.co
Media Contact:
Ashlye Hodge - Communications Manager
ashlye@metagenomi.co
FAQ
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