Metagenomi Presents Highly Specific and Efficient Genome Editing Tools at Nature Conference “RNA at the Bench and Bedside IV”
Metagenomi (Nasdaq: MGX) presented data demonstrating the precision and safety of its gene editing tools at the Nature Conference: RNA at the Bench and Bedside IV. The company highlighted two key developments: MGX-001, their hemophilia A candidate using the MG29-1 nuclease, showed no identifiable off-target editing and no evidence of translocations in primary human hepatocytes. Additionally, their Adenine Base Editor (ABE) for ex vivo cell therapy demonstrated no detectable translocations and no significant genomic base composition differences in primary T-cells compared to unedited cells.
Metagenomi (Nasdaq: MGX) ha presentato dati che dimostrano la precisione e la sicurezza dei suoi strumenti di editing genetico durante la Conferenza Nature: RNA at the Bench and Bedside IV. L'azienda ha evidenziato due sviluppi chiave: MGX-001, il loro candidato per l'emofilia A che utilizza il nuclease MG29-1, non ha mostrato editing indesiderato e non ci sono evidenze di traslocazioni in epatociti umani primari. Inoltre, il loro Adenine Base Editor (ABE) per la terapia cellulare ex vivo ha dimostrato l'assenza di traslocazioni rilevabili e nessuna differenza significativa nella composizione del materiale genetico nei linfociti T primari rispetto alle cellule non editate.
Metagenomi (Nasdaq: MGX) presentó datos que demuestran la precisión y la seguridad de sus herramientas de edición genética en la Conferencia Nature: RNA at the Bench and Bedside IV. La compañía destacó dos desarrollos clave: MGX-001, su candidato para hemofilia A que utiliza la nucleasa MG29-1, no mostró ediciones no deseadas y no hubo evidencia de translocaciones en hepatocitos humanos primarios. Además, su Adenine Base Editor (ABE) para terapia celular ex vivo demostró no tener translocaciones detectables y ninguna diferencia significativa en la composición genética de células T primarias en comparación con las células no editadas.
메타게노미 (Nasdaq: MGX)는 Nature Conference: RNA at the Bench and Bedside IV에서 자사의 유전자 편집 도구의 정확성과 안전성을 입증하는 데이터를 발표했습니다. 회사는 두 가지 주요 개발을 강조했습니다: MGX-001, MG29-1 핵산 효소를 사용하는 혈우병 A 치료 후보는 오프타겟 편집이 없고 주요 인간 간세포에서 전위증의 증거가 없었습니다. 추가로, 그들의 Adenine Base Editor (ABE)는 세포 외 치료를 위해 주요 T세포에서 비편집 세포와 비교할 때 감지 가능한 전위증이 없고, 유전적 기초 구성의 유의미한 차이가 없음을 보여주었습니다.
Metagenomi (Nasdaq: MGX) a présenté des données démontrant la précision et la sécurité de ses outils d'édition génétique lors de la conférence Nature : RNA at the Bench and Bedside IV. L'entreprise a souligné deux développements clés : MGX-001, leur candidat pour l'hémophilie A utilisant la nuclease MG29-1, n'a montré aucune édition hors cible identifiable et aucune preuve de translocations dans des hépatocytes humains primaires. De plus, leur Adenine Base Editor (ABE) pour la thérapie cellulaire ex vivo n'a montré aucune translocation détectable et aucune différence significative dans la composition génomique des lymphocytes T primaires par rapport aux cellules non éditées.
Metagenomi (Nasdaq: MGX) hat Daten präsentiert, die die Genauigkeit und Sicherheit seiner Werkzeuge zur Genbearbeitung auf der Nature Conference: RNA at the Bench and Bedside IV demonstrieren. Das Unternehmen hob zwei wichtige Entwicklungen hervor: MGX-001, ihr Kandidat für Hämophilie A unter Verwendung der MG29-1 Nuklease, zeigte keine identifizierbaren Off-Target-Bearbeitungen und keine Hinweise auf Translokationen in primären menschlichen Hepatozyten. Darüber hinaus zeigte ihr Adenine Base Editor (ABE) für ex vivo Zelltherapie keine nachweisbaren Translokationen und keine signifikanten Unterschiede in der genomischen Basenzusammensetzung in primären T-Zellen im Vergleich zu nicht bearbeiteten Zellen.
- MGX-001 demonstrated zero off-target editing in genomic assays
- MG29-1 nuclease showed no evidence of translocations in human hepatocytes
- Adenine Base Editor exhibited no detectable translocations in T-cells
- None.
Insights
The presentation showcases significant technical achievements in gene editing precision. The MG29-1 nuclease used in MGX-001 demonstrates remarkable specificity with no detectable off-target effects, a critical safety feature for gene therapy applications. This is particularly relevant for hemophilia A treatment, where precise editing is essential.
The absence of translocations in hepatocytes and the clean safety profile in T-cells are technically impressive. These characteristics could potentially reduce regulatory hurdles and accelerate the path to clinical trials. However, while the data is promising, it's important to note that this was presented at a conference and still requires validation through peer-reviewed publications and clinical trials.
This presentation reinforces Metagenomi's competitive position in the gene editing space. The company's proprietary metagenomics platform is yielding tools with potentially superior safety profiles compared to existing CRISPR technologies. The focus on both in vivo and ex vivo applications expands their market potential across multiple therapeutic areas.
The development of MGX-001 for hemophilia A targets a lucrative market, with current treatments generating billions in annual sales. The demonstration of high specificity could differentiate Metagenomi's approach from competitors. However, investors should note that the company is still in preclinical stages and successful conference data doesn't guarantee clinical or commercial success.
MGX-001, utilizing a highly specific and efficient MG29-1 nuclease, exhibits no identifiable off-target editing
MG29-1 nuclease targeting the albumin safe harbor locus showed no evidence of translocations in primary human hepatocytes
Metagenomi Adenine Base Editor (ABE) demonstrates no detectable translocations and no significant genomic base composition differences in primary T-cells
EMERYVILLE, Calif., Dec. 11, 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 talk titled “Specific and efficient genome editing with metagenomics-derived tools for in vivo and ex vivo therapeutic applications” at the Nature Conference: RNA at the Bench and Bedside IV.
“We believe the value proposition for single-dose gene editing therapies requires exquisite specificity characterization to ensure safety and efficacy. Today’s presentation highlights the precision of Metagenomi’s next-generation nucleases and ABEs, discovered through the company’s proprietary metagenomics platform and tailored for both in vivo and ex vivo therapeutic applications,” said Alan Brooks, SVP and Head of Preclinical. “MGX-001, Metagenomi’s development candidate for hemophilia A, which utilizes the novel nuclease MG29-1, exhibits no identifiable off-target editing using a series of orthogonal assays employed to evaluate potential off-target sites in the genome. The MG29-1 nuclease targeting the albumin safe harbor locus showed no evidence of translocations in primary human hepatocytes. For Metagenomi’s novel next-generation ABE for ex vivo cell therapy indications via multiplex editing, the data showed no detectable translocations and no significant genomic base composition differences in primary T-cells when compared to unedited cells. These examples demonstrate our strong capabilities in developing highly specific next-generation gene editing tools and support the company’s ability to potentially progress these systems toward the clinic for the benefit of patients.”
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 (CAST)). 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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Contact:
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IR@metagenomi.co
FAQ
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