On May 1, 2025 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, reported that five abstracts detailing Precision’s wholly owned or partnered programs have been accepted for presentation at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting being held May 13-17, 2025, in New Orleans, Louisiana (Press release, Precision Biosciences, MAY 1, 2025, View Source [SID1234652451]).

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"We are pleased to present new data at ASGCT (Free ASGCT Whitepaper) from multiple programs across our in vivo gene editing pipeline, highlighting the potentially unmatched breadth and therapeutic potential of our ARCUS gene editing technology," said Michael Amoroso, President and Chief Executive Officer at Precision BioSciences. "Clinically, we have made progress advancing our lead program, PBGENE-HBV, which is a novel gene editing approach being evaluated in a first-in-human study for chronic hepatitis B, and we look forward to sharing initial safety data from the first two dose cohorts at ASGCT (Free ASGCT Whitepaper). Additionally, we continue to be excited by the promising clinical results generated thus far by our partner iECURE for ECUR-506 in OTC deficiency, which utilizes an ARCUS nuclease and will also be presenting data at this conference."

"We are also excited to share promising preclinical results from two muscle programs, PBGENE-3243 program for m.3243-associated mitochondrial disease and PBGENE-DMD for the treatment of Duchenne Muscular Dystrophy (DMD)," Mr. Amoroso continued. "Collectively, these presentations highlight the progress we are making with ARCUS by deploying a variety of gene edit types, including gene elimination, gene insertion and gene excision across a range of complex genetic diseases as we seek to develop durable, curative treatments for patients."

Clinical Program Presentations:

Title: Initial Safety Data From ELIMINATE-B, the First Clinical Trial of a Gene Editing Treatment for Chronic Hepatitis B
Oral Presentation Session: Gene Therapy Trials – In-Vivo Gene Therapy Modification
Date and Time: Friday, May 16, 2025, 4:00 PM CT
Location: Room 393-396

Title: ARCUS in vivo OTC, Large Gene Insertion (clinical stage partnered program)
Oral Presentation Session: Advances in Genome Editing: Novel Large DNA Insertion Technologies and Their Potential Towards Curative Therapies
Date and Time: Wednesday, May 14, 2025, 8:00 AM CT
Location: NOLA Theater B

Preclinical Program Presentations:

Title: Excision of the C9orf72 Hexanucleotide Repeat Expansion Using a Dual-ARCUS Gene Editing Approach Reduces Neurotoxic RNA Foci and Dipeptides in an In Vivo Model of ALS
Poster Session: Poster Reception
Date and Time: Tuesday, May 13, 2025, 6:00 PM – 7:30 PM CT
Location: Poster Hall I2

Title: Systemic Delivery of a Mitochondria-Targeting ARCUS Gene Editing Nuclease by AAV Eliminates Mutant Mitochondrial DNA, Demonstrating Therapeutically Meaningful Heteroplasmy Shifts In Vivo
Oral Presentation Session: Gene Editing: New Tools and Technology Advances
Date and Time: Wednesday, May 14, 2025, 3:45 PM CT
Location: NOLA Theater A

Title: ARCUS-Mediated Gene Editing Excision of Exons 45-55 of the Human Dystrophin Gene using PBGENE-DMD Leads to Functional Dystrophin Protein and Durable Restoration of Skeletal Muscle-Function In Vivo for the Treatment of Duchenne Muscular Dystrophy
Poster Session: Poster Reception
Date and Time: Wednesday, May 14, 2025, 5:30 PM – 7:00 PM CT
Location: Poster Hall I2

The abstracts are now publicly accessible through the ASGCT (Free ASGCT Whitepaper) website here.