Precision BioSciences Presents Preclinical Data Highlighting the Capability of ARCUS for High-Efficiency Gene Editing Utilizing Homology-Directed Repair at the ESGCT 31st Annual Congress
Precision BioSciences (Nasdaq: DTIL) presented preclinical data at the ESGCT 31st Annual Congress showcasing their ARCUS platform's capability for high-efficiency gene editing through homology-directed repair (HDR). The research demonstrated gene insertion rates exceeding 85% in T cells and 39% in non-dividing primary human hepatocytes.
The ARCUS platform showed versatility in DNA editing, achieving all twelve possible base changes, specific base removal and insertions, and whole gene replacements. This capability extends beyond current limitations of base editors and prime editors, potentially offering broader therapeutic applications for diseases requiring function restoration.
Precision BioSciences (Nasdaq: DTIL) ha presentato dati preclinici al 31° Congresso Annuale ESGCT che dimostrano la capacità della loro piattaforma ARCUS per l'editing genico ad alta efficienza attraverso la riparazione mediata da omologia (HDR). La ricerca ha messo in evidenza tassi di inserimento genico superiori al 85% nelle cellule T e 39% negli epatociti umani primari non in divisione.
La piattaforma ARCUS ha dimostrato versatilità nell'editing del DNA, realizzando tutti e dodici i possibili cambiamenti di basi, rimozioni e inserimenti di basi specifiche, e sostituzioni di interi geni. Questa capacità va oltre le attuali limitazioni degli editor di basi e degli editor primari, offrendo potenzialmente applicazioni terapeutiche più ampie per le malattie che richiedono il ripristino della funzione.
Precision BioSciences (Nasdaq: DTIL) presentó datos preclínicos en el 31° Congreso Anual de ESGCT que destacan la capacidad de su plataforma ARCUS para la edición genética de alta eficiencia mediante reparación dirigida por homología (HDR). La investigación demostró tasas de inserción genética superiores al 85% en células T y 39% en hepatocitos humanos primarios no en división.
La plataforma ARCUS mostró versatilidad en la edición de ADN, logrando los doce cambios de bases posibles, eliminaciones e inserciones específicas de bases, y reemplazos de genes completos. Esta capacidad va más allá de las actuales limitaciones de los editores de bases y editores primarios, ofreciendo potencialmente aplicaciones terapéuticas más amplias para enfermedades que requieren la restauración de la función.
프레시전 바이오사이언스(주식 코드: DTIL)는 제31회 ESGCT 연례 Congress에서 동종 유전자 수정을 통해 높은 효율성을 보여주는 ARCUS 플랫폼의 전임상 데이터를 발표했습니다. 연구 결과는 T 세포에서 85% 이상 그리고 비분열 상태의 인체 간세포에서 39% 이상의 유전자 삽입률을 보여주었습니다.
ARCUS 플랫폼은 DNA 편집에서 다재다능성을 보여주었으며, 가능한 12가지 기본 변경, 특정 기본 제거 및 삽입, 전체 유전자 교체를 수행했습니다. 이러한 능력은 기존의 기본 편집기 및 프라임 편집기의 한계를 넘어, 기능 복원이 필요한 질환에 대해 더 넓은 치료적 적용을 제공할 수 있습니다.
Precision BioSciences (Nasdaq: DTIL) a présenté des données précliniques lors du 31e Congrès Annuel ESGCT mettant en avant la capacité de leur plateforme ARCUS pour l'édition génétique à haute efficacité à travers la réparation dirigée par homologie (HDR). La recherche a montré des taux d'insertion génique dépassant 85% dans les cellules T et 39% dans les hépatocytes humains primaires non divisants.
La plateforme ARCUS a montré une polyvalence dans l'édition de l'ADN, réalisant tous les douze changements de base possibles, ainsi que des suppressions et insertions de bases spécifiques, et des remplacements complets de gènes. Cette capacité va au-delà des limites actuelles des éditeurs de bases et des éditeurs primaires, offrant potentiellement des applications thérapeutiques plus larges pour les maladies nécessitant la restauration de la fonction.
Precision BioSciences (Nasdaq: DTIL) präsentierte präklinische Daten auf dem 31. Jahreskongress der ESGCT, die das Potenzial ihrer ARCUS-Plattform für effizientes Gen-Editing durch homologe Rekombination (HDR) demonstrieren. Die Forschung zeigte Einfügungsraten von über 85% in T-Zellen und 39% in nicht sich teilenden primären menschlichen Hepatozyten.
Die ARCUS-Plattform zeigte Vielseitigkeit bei der DNA-Bearbeitung und erreichte alle zwölf möglichen Basenänderungen, spezifische Basenentfernungen und -einfügungen sowie vollständige Genersetzungen. Diese Fähigkeit geht über die aktuellen Beschränkungen von Baseneditoren und Prime-Editoren hinaus und könnte potenziell breitere therapeutische Anwendungen für Krankheiten bieten, die eine Funktionswiederherstellung erfordern.
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- Stimulating gene editing by homology-directed repair (HDR) has the potential advantage of being able to achieve any type of edit, including gene insertion
- ARCUS approach potentially offers broader therapeutic than current gene editing modalities for diseases that require a gain in function
“Gene editing enzymes that support efficient HDR are relatively rare. However, we demonstrate here that ARCUS is capable of utilizing HDR with high efficiency to achieve a range of gene editing outcomes, including specific base changes, insertions, and the replacement of large segments of DNA within the genome," said Jeff Smith, PhD, Co-Founder and Chief Research Officer of Precision BioSciences. “These data continue to validate the ARCUS platform as an ideal modality for treating diseases that require gene insertion or repair to restore function whereas other gene editing modalities are largely focused on knocking-out function. In addition to the preclinical data presented today, we look forward to seeing supportive clinical validation of HDR-based ARCUS gene insertion through our partner iECURE, who is advancing their ECUR-506 clinical stage program utilizing an ARCUS nuclease for treating OTC deficiency,” added Jeff Smith.
Title: High-efficiency homology-directed insertion into the genome using ARCUS nucleases
Poster Number: #PO678
Presenter: Adam Mischler, PhD, Senior Scientist, Precision BioSciences Gene Discovery
Date and Time: Thursday, October 24, 2024, 6:00-7:30pm CEST
In preclinical work presented today, we show that targeted gene insertion can be achieved using ARCUS in greater than
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is an advanced gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics may enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, the Company’s pipeline is comprised of in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com.
The ARCUS platform is being used to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion (inserting DNA into a gene to cause expression/add function), elimination (removing a genome, e.g., viral DNA or mutant mitochondrial DNA), and excision (removing a large portion of a defective gene by delivering two ARCUS nucleases in a single AAV).
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the clinical development and expected safety, efficacy and benefit of our product candidates and gene editing approaches including editing efficiency, key capabilities and differentiating characteristics, the ability of ARCUS to achieve any type of edit including high-efficiency gene insertion, gene replacement, and base correction, the ability of ARCUS to stimulate gene editing by homology directed repair, the potentially broader therapeutic potential of ARCUS for diseases that require a gain in function than current gene editing modalities and the suitability of ARCUS nucleases for gene elimination, insertion and excision and differentiation from other gene editing approaches due to its small size, simplicity and distinctive cut. In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,” “designed,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “pursue,” “should,” “strive,” “target,” “will,” “would,” or the negative thereof and similar words and expressions.
Forward-looking statements are based on management’s current expectations, beliefs and assumptions and on information currently available to us. These statements are neither promises nor guarantees, and involve a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to, our ability to become profitable; our ability to procure sufficient funding to advance our programs; risks associated with our capital requirements, anticipated cash runway, requirements under our current debt instruments and effects of restrictions thereunder, including our ability to raise additional capital due to market conditions and/or our market capitalization; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the progression and success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities and preclinical and clinical studies, including clinical trial and investigational new drug applications; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators’ or other licensees’ ability to identify, develop and commercialize product candidates; pending and potential product liability lawsuits and penalties against us or our collaborators or other licensees related to our technology and our product candidates; the
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Investor and Media Contact:
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naresh.tanna@precisionbiosciences.com
Source: Precision BioSciences, Inc.
FAQ
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