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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

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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

DURHAM, N.C.--(BUSINESS WIRE)-- Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for sophisticated gene edits, today announced a poster presentation at the European Society of Gene & Cell Therapy (ESGCT) 31st Annual Congress being held October 22-25, 2024, in Rome, Italy. The poster highlighted preclinical data demonstrating the ability of ARCUS to achieve high-efficiency gene insertion, gene replacement and base correction via homology-directed repair (HDR).

“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 85% of T cells and 39% of non-dividing primary human hepatocytes. These high rates of gene insertion were accomplished primarily through HDR as evidenced by the dependency on homology arms in the repair template and on the unique ARCUS 3’ 4 base pair overhang cut that resembles a processed end for stimulating HDR. Using HDR, ARCUS successfully demonstrates the ability to support a wide range of DNA editing approaches using a template to define the specific edit. Unlike base editors’ limitation to two possible base changes, ARCUS was able to produce all twelve possible base changes, in addition to producing very specific base removal and insertions. Unlike prime editors, ARCUS was also able to generate whole gene insertions and even replace large segments within a gene to restore function. The characteristic enzymology of ARCUS nucleases drives high rates of HDR, which can potentially be leveraged to achieve a multitude of highly precise gene editing functions for therapeutic benefit.

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 U.S. and foreign regulatory landscape applicable to our and our collaborators’ or other licensees’ development of product candidates; our or our collaborators’ or other licensees’ ability to advance product candidates into, and successfully design, implement and complete, clinical trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; delays or difficulties in our and our collaborators’ and other licensees’ ability to enroll patients; changes in interim “top-line” and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; our or our licensees’ ability to obtain orphan drug designation or fast track designation for our product candidates or to realize the expected benefits of these designations; our or our collaborators’ or other licensees’ ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; the rate and degree of market acceptance of any of our product candidates; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate executives and personnel; effects of system failures and security breaches; insurance expenses and exposure to uninsured liabilities; effects of tax rules; effects of any pandemic, epidemic, or outbreak of an infectious disease; the success of our existing collaboration and other license agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events; effects of sustained inflation, supply chain disruptions and major central bank policy actions; market and economic conditions; risks related to ownership of our common stock, including fluctuations in our stock price; our ability to meet the requirements of and maintain listing of our common stock on Nasdaq or other public stock exchanges; and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2024, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC’s website at www.sec.gov and the Investors page of our website under SEC Filings at investor.precisionbiosciences.com. All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Investor and Media Contact:

Naresh Tanna

Vice President of Investor Relations

naresh.tanna@precisionbiosciences.com

Source: Precision BioSciences, Inc.

FAQ

What gene insertion rates did DTIL's ARCUS platform achieve in T cells during the 2024 ESGCT presentation?

The ARCUS platform achieved gene insertion rates greater than 85% in T cells, as presented at the ESGCT 31st Annual Congress in October 2024.

How does DTIL's ARCUS platform perform HDR gene editing in hepatocytes?

ARCUS achieved 39% gene insertion rates in non-dividing primary human hepatocytes through homology-directed repair (HDR).

What advantages does DTIL's ARCUS platform offer over base editors?

Unlike base editors to two possible base changes, ARCUS can produce all twelve possible base changes, specific base removal and insertions, and whole gene insertions.

When and where did Precision BioSciences present their ARCUS HDR data?

The data was presented at the European Society of Gene & Cell Therapy (ESGCT) 31st Annual Congress in Rome, Italy, October 22-25, 2024.

Precision BioSciences, Inc.

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