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Precision BioSciences Announces Oral Presentation at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

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Precision BioSciences (NASDAQ: DTIL) has announced an upcoming oral presentation of preclinical data from their PBGENE-DMD program at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference. The presentation, scheduled for March 19, 2025, at 8:00 AM CT, will focus on the treatment of Duchenne muscular dystrophy (DMD) using their proprietary ARCUS® platform.

The presentation will detail how ARCUS-mediated excision of exons 45-55 leads to functional Del45-55 dystrophin and restoration of skeletal muscle function in DMD treatment. The company's ARCUS platform is being developed for various in vivo gene editing therapies, including gene elimination, insertion, and excision programs.

Precision BioSciences (NASDAQ: DTIL) ha annunciato una prossima presentazione orale dei dati preclinici del loro programma PBGENE-DMD alla Conferenza Clinica e Scientifica dell'Associazione Distrofia Muscolare 2025. La presentazione, programmata per il 19 marzo 2025, alle 8:00 AM CT, si concentrerà sul trattamento della distrofia muscolare di Duchenne (DMD) utilizzando la loro piattaforma proprietaria ARCUS®.

La presentazione illustrerà come l'escissione mediata da ARCUS degli esoni 45-55 porti alla produzione di distrofina Del45-55 funzionale e al ripristino della funzione muscolare scheletrica nel trattamento della DMD. La piattaforma ARCUS dell'azienda è in fase di sviluppo per varie terapie di editing genetico in vivo, comprese le strategie di eliminazione, inserimento ed escissione genica.

Precision BioSciences (NASDAQ: DTIL) ha anunciado una próxima presentación oral de datos preclínicos de su programa PBGENE-DMD en la Conferencia Clínica y Científica de la Asociación de Distrofia Muscular 2025. La presentación, programada para el 19 de marzo de 2025, a las 8:00 AM CT, se centrará en el tratamiento de la distrofia muscular de Duchenne (DMD) utilizando su plataforma propietaria ARCUS®.

La presentación detallará cómo la excisión mediada por ARCUS de los exones 45-55 conduce a la producción de distrofina Del45-55 funcional y a la restauración de la función muscular esquelética en el tratamiento de la DMD. La plataforma ARCUS de la empresa se está desarrollando para diversas terapias de edición genética in vivo, incluyendo programas de eliminación, inserción y excisión de genes.

Precision BioSciences (NASDAQ: DTIL)는 2025년 근육병 협회 임상 및 과학 회의에서 PBGENE-DMD 프로그램의 전임상 데이터에 대한 구두 발표를 예정하고 있다고 발표했습니다. 발표는 2025년 3월 19일 오전 8시 CT에 예정되어 있으며, 뒤셴 근육 이영양증(DMD) 치료에 있어 그들의 독점적인 ARCUS® 플랫폼을 사용할 것입니다.

발표에서는 ARCUS 매개 방식으로 엑손 45-55를 절제함으로써 기능적인 Del45-55 디스트로핀 생성과 DMD 치료에서 골격 근육 기능 회복을 어떻게 이끌어내는지를 상세히 설명할 것입니다. 회사의 ARCUS 플랫폼은 유전자 제거, 삽입 및 절제 프로그램을 포함하여 다양한 생체 내 유전자 편집 치료를 위해 개발되고 있습니다.

Precision BioSciences (NASDAQ: DTIL) a annoncé une prochaine présentation orale de données précliniques de son programme PBGENE-DMD lors de la Conférence Clinique et Scientifique de l'Association de Dystrophie Musculaire 2025. La présentation, prévue pour le 19 mars 2025 à 8h00 CT, se concentrera sur le traitement de la dystrophie musculaire de Duchenne (DMD) en utilisant leur plateforme propriétaire ARCUS®.

La présentation détaillera comment l'excision médiée par ARCUS des exons 45-55 conduit à la production de dystrophine Del45-55 fonctionnelle et à la restauration de la fonction musculaire squelettique dans le traitement de la DMD. La plateforme ARCUS de l'entreprise est en cours de développement pour diverses thérapies d'édition génique in vivo, y compris des programmes d'élimination, d'insertion et d'excision de gènes.

Precision BioSciences (NASDAQ: DTIL) hat eine bevorstehende mündliche Präsentation präklinischer Daten aus ihrem PBGENE-DMD-Programm auf der 2025 Muscular Dystrophy Association Clinical & Scientific Conference angekündigt. Die Präsentation, die für den 19. März 2025 um 8:00 Uhr CT geplant ist, wird sich auf die Behandlung der Duchenne-Muskeldystrophie (DMD) mit ihrer proprietären ARCUS®-Plattform konzentrieren.

In der Präsentation wird erläutert, wie die von ARCUS vermittelte Exzision der Exons 45-55 zu funktioneller Del45-55 Dystrophin und zur Wiederherstellung der Skelettmuskel-Funktion bei der DMD-Behandlung führt. Die ARCUS-Plattform des Unternehmens wird für verschiedene in vivo-Gentransfertherapien entwickelt, einschließlich Gene-Löschungs-, Einfügungs- und Exzisionsprogrammen.

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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, including gene elimination, gene insertion, and gene excision programs, today announced that the company will present preclinical data from the PBGENE-DMD program for the treatment of Duchenne muscular dystrophy (DMD) during an oral presentation at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 16-19, 2025.

Presentation Details:

Title: ARCUS-Mediated Excision of Exons 45-55 Leads to Functional Del45-55 Dystrophin and Restoration of Skeletal Muscle-Function for the Treatment of DMD
Date and Time: Wednesday, March 19, 2025, 8:00 AM CT

The abstract is now publicly accessible through the MDA website here.

About Precision BioSciences, Inc.

Precision BioSciences, Inc. is a clinical stage 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 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 such as in the DMD program for oral presentation at the MDA Clinical & Scientific Conference).

Investor and Media Contact:

Naresh Tanna

Vice President of Investor Relations

naresh.tanna@precisionbiosciences.com

Source: Precision BioSciences, Inc.

FAQ

What will Precision BioSciences (DTIL) present at the 2025 MDA Conference?

Precision BioSciences will present preclinical data from their PBGENE-DMD program, specifically focusing on ARCUS-mediated excision of exons 45-55 for DMD treatment, on March 19, 2025.

How does DTIL's ARCUS platform approach DMD treatment?

DTIL's ARCUS platform targets DMD treatment through gene editing, specifically using ARCUS-mediated excision of exons 45-55 to achieve functional Del45-55 dystrophin and restore skeletal muscle function.

When and where will DTIL present their DMD treatment data?

The presentation will take place on Wednesday, March 19, 2025, at 8:00 AM CT during the 2025 Muscular Dystrophy Association Clinical & Scientific Conference.

What gene editing capabilities does DTIL's ARCUS platform offer?

DTIL's ARCUS platform is designed for in vivo gene editing therapies, including gene elimination, gene insertion, and gene excision programs.

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