ElevateBio Announces Presentations at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting

– Oral and poster presentations will highlight advancements from ElevateBio’s two business units: Life Edit, its gene editing and R&D technology business, and BaseCamp^®, its process development and cGMP manufacturing business

– ElevateBio's combined focus on cutting-edge editing technologies and optimized manufacturing solutions directly addresses the industry's most critical challenges

– Life Edit is expanding its proprietary gene editing toolbox with novel CRISPR proteins to advance genomic medicines with optimized on-target efficacy while minimizing off-target editing

/EIN News/ -- WALTHAM, Mass., April 28, 2025 (GLOBE NEWSWIRE) -- ElevateBio, a technology-driven company focused on powering the creation of life-transforming genetic medicines, today announced presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting taking place May 13-17, in New Orleans, LA.

ElevateBio's presentations demonstrate how the Company's integrated approach combines CRISPR gene editing technologies with cGMP manufacturing solutions to drive the cell and gene therapy industry forward. By enhancing the potential safety, efficacy, and manufacturability of these transformative medicines, ElevateBio enables its biopharma partners to accelerate advanced therapies from concept to clinic more efficiently. These advancements showcase how ElevateBio's dual focus on advanced editing technologies with expanded targeting capabilities and optimized manufacturing solutions directly addresses the industry's most critical challenges.

Below is a summary of ElevateBio's oral and poster presentations organized by business unit. All poster presentations will take place in Poster Hall I2. ElevateBio’s abstracts are available on ASGCT’s website here. ElevateBio will be exhibiting at booth #509.

Life Edit, ElevateBio’s Gene Editing and R&D Technology Business

"Life Edit continues to expand its proprietary gene editing toolbox with novel CRISPR proteins that increase our ability to target a wider range of genomic sites with high precision," said Amy Pooler, Ph.D., Senior Vice President, Research & Development at ElevateBio Life Edit. "Our presentations showcase not only our growing collection of editors with diverse genome recognition sites, but also critical advancements in understanding and reducing off-target RNA editing effects. These capabilities, combined with our expertise in optimizing all components of CRISPR-based medicines, position us to develop highly specific genetic therapies, as demonstrated by our promising LETI-101 program for Huntington's disease."

Oral Presentation:

Title: Intrastriatal AAV5.SGN.LEGB (LETI-101) Administration Selectively Targeting Mutant Allele of the HTT Gene Resulted in Broad CNS Distribution and Transgene Expression in Critical NHP Brain Regions Associated with Huntington’s Disease Pathology
Session: Pharmacology/Toxicology Studies and Analytics/Assay Development Session II
Date/time: May 17, 2025, at 9:15-9:30 a.m. ET
Location: Room 288-290
Presentation Overview: The presentation includes preclinical data from a non-human primate study evaluating LETI-101, Life Edit’s partnership-ready development candidate for Huntington's disease (HD). These data cover safety observations, biodistribution findings, transgene expression analysis across key brain regions, and immunogenicity assessments following intrastriatal administration, providing important insights supporting the advancement of LETI-101 to clinical trials. These findings follow previously demonstrated reduction of mHTT protein in HD patient-derived fibroblasts and clinically relevant BACHD murine model following intrastriatal injections with LETI-101.

Poster Presentations:

Title: Signature of Base Editor RNA Edits in the Transcriptome and Reduction of RNA Editing (# AMA1610)
Date/time: May 14, 2025, at 5:30-7 p.m. ET
Poster Overview: Life Edit scientists have developed a method to distinguish between RNA edits made by base editors and naturally occurring RNA edits, addressing a key challenge in evaluating the safety of these therapeutic tools. The method revealed that RNA editing from base editors is dose-dependent and temporary and supports the advancement of improved genomic medicines with higher on-target efficacy while minimizing off-target RNA editing.

Title: Deep CRISPR Nuclease Portfolio and Multiple Editing Modalities Accelerates Identification of Viable Clinical Candidates (# AMA1674)
Date/time: May 15, 2025, at 5:30-7 p.m. ET
Poster Overview: Life Edit has developed the largest portfolio of compact, mammalian-active nucleases with diverse protospacer adjacent motifs (PAMs), enabling full access to human disease mutations. These RNA-guided nucleases, base editors, and reverse transcriptase editors have been validated in vitro and in vivo with flexible delivery via proprietary LNP, AAV, and ex vivo methods. The deep portfolio of editing technologies along with artificial intelligence (AI), structure-guided design, and directed evolution allow Life Edit to rapidly develop potent, specific, and clinically viable genome editing candidates.

BaseCamp, ElevateBio’s Process Development and cGMP Manufacturing Business:

“BaseCamp's end-to-end capabilities are designed to overcome the manufacturing challenges that often hinder the potential of many cell and gene therapies," said Mike Paglia, Chief Technology Officer at ElevateBio BaseCamp. "Our presentations demonstrate how we're innovating across the manufacturing process—from improved cell isolation methods, to optimized non-viral delivery approaches for gene editing, to scalable lentiviral vector production capable of supporting commercial demands. These advancements collectively reduce complexity and accelerate timelines while improving access to potentially life-saving therapies.”

Oral Presentation:

Title: Advancing Lentiviral Vector Manufacturing: A Platform for Commercial Cell Therapy Success
Session: Vector Product Engineering, Development and Manufacturing (excluding AAV)
Date/time: May 17, 2025, at 10:30-10:45 a.m. ET
Location: Room 383-385
Presentation Overview: The presentation outlines BaseCamp’s scalable lentiviral vector manufacturing platform process, LentiPeak, developed for ex vivo cell therapy applications. Key findings include the GMP manufacturing performance, scale-down model validation, and clinical capacity per batch production. BaseCamp is also establishing a comprehensive approach integrating robust process design, clinical insights, and risk-based analysis, providing a clear pathway for commercializing LV-based therapies.

Poster Presentations:

Title: Non-Viral Delivery of DNA Template and Gene Editing Components for Targeted Gene Insertion in Human Primary T-Cells: Electroporation vs. Lipid Nanoparticles (# AMA1080)
Date/Time: May 13, 2025, at 6-7:30 p.m. ET
Poster Overview: BaseCamp scientists compared electroporation and lipid nanoparticles (LNPs) for delivering gene editing components in human primary T-cells, revealing distinct advantages: electroporation achieved higher editing efficiencies while LNP-treated cells maintained superior viability and expansion potential. These findings provide critical insights for optimizing non-viral CAR-T cell manufacturing, potentially reducing costs and improving accessibility of these transformative therapies through more efficient, scalable production methods.

Title: Peripheral Blood Mononuclear Cell (PBMC) Isolation Using Cell Fractionation Filters (# AMA872)
Date/Time: May 15, 2025, at 5:30-7 p.m. ET
Poster Overview: BaseCamp scientists evaluated high-precision cell separation filters and cell fractionation filters with pore sizes between 5-9 µm as a novel method for isolating peripheral blood mononuclear cells (PBMCs) from whole blood, achieving up to 80% CD3+ T cell recovery—a 2-fold improvement over traditional Ficoll gradient separation—while maintaining high cell viability and effective RBC/platelet depletion. This improved PBMC isolation method enables a closed-system T cell enrichment workflow directly from whole blood instead of leukapheresis collections, potentially transforming CAR-T manufacturing by reducing costs, shortening timelines, and improving accessibility for patients who would benefit from these potentially life-saving therapies.

About ElevateBio
ElevateBio is a technology-driven company built to power transformative genetic medicines today and for many decades to come. The Company commercializes its enabling technologies, manufacturing capabilities, and industry-leading expertise through partnerships to accelerate development across a breadth of therapeutic approaches and modalities. The ElevateBio ecosystem combines Life Edit, its gene editing and R&D technology business, with BaseCamp^®, its end-to-end genetic medicine cGMP manufacturing and process development business, to accelerate the discovery and development of advanced therapeutics.

ElevateBio aims to be the dominant engine inside the world’s greatest scientific advancements harnessing human cells and genes to alter disease. For more, visit www.elevate.bio or follow ElevateBio on LinkedIn or X.

Investor contact:
Catherine Hu
chu@elevate.bio

Media contact:
DJ Webster
dwebster@elevate.bio