ElevateBio Data at ASGCT 29th Annual Meeting Showcase Expanded Gene Editing Platform and Services Spanning New Modalities, AI Discovery, and LNP Delivery

• Data highlight advancements across ElevateBio's targeted gene insertion platform, powered by retrotransposon and machine learning-optimized recombinase approaches, and its new epigenetic editing platform
  
• Adenine base editors incorporating AI-designed, functional deaminases enable targeting of previously intractable sites, underscoring ElevateBio’s AI-powered discovery capabilities
  
• Proprietary LNP platform achieved therapeutically relevant in vivo editing across multiple modalities in mice and non-human primates, with favorable tolerability and demonstrated re-dosing
  
• New capabilities expand ElevateBio’s portfolio of service offerings from gene editing discovery through commercial manufacturing for advanced therapies
  

WALTHAM, Mass., May 14, 2026 (GLOBE NEWSWIRE) -- ElevateBio, a technology-driven advanced therapy CDMO and gene editing services partner, today announced data from presentations at the American Society of Gene and Cell Therapy (ASGCT) 29th Annual Meeting, held May 11-15 in Boston, MA. The data span multiple gene editing modalities, AI-powered protein discovery and optimization, and proprietary lipid nanoparticle (LNP) delivery, collectively demonstrating the strength of ElevateBio’s technologies and gene editing discovery services.

“The data we’ve presented at ASGCT demonstrate the strength of the technologies and capabilities we've built at ElevateBio, where AI-powered discovery, multiple editing modalities, and optimized delivery reinforce one another," said Amy Pooler, Ph.D., Chief Scientific Officer at ElevateBio. "We're seeing increasing interest from potential partners in targeted gene insertion, and our work in both recombinases and retrotransposons positions us to meet that demand. When we combine multiple editing modalities with our ability to discover and optimize proteins using generative AI, we can match the right editing approach to each disease target, giving our partners the best chance of bringing transformative therapies to patients."

ElevateBio’s Gene Editing Tools and Discovery Services
ElevateBio is expanding its gene editing platform to provide biopharmaceutical partners with the technologies and capabilities needed to advance therapeutic programs and increase the probability of success. The Company’s tools span multiple modalities – including nucleases, base editors, reverse transcriptase (RT) editors, large gene insertion, and epigenetic editors – ensuring the optimal technology can be applied to a specific target. These capabilities are backed by ElevateBio's cGMP manufacturing infrastructure, providing partners with an integrated path from discovery through commercialization.

"Adding these new gene editing capabilities enables ElevateBio to meet the evolving needs of advanced therapy developers," said Chris Murphy, Chief Executive Officer at ElevateBio. "Whether a partner needs a proof-of-concept in months or a fully built product pipeline, we provide the end-to-end expertise and services to realize the promise of these new modalities, from gene editing discovery through cGMP commercial manufacturing."

Targeted Gene Insertion: Proprietary Recombinases and Retrotransposons
Targeted gene insertion addresses diseases that require the insertion of full-length genes rather than single-base corrections, representing a significant expansion of the therapeutic reach of gene editing. ElevateBio’s gene insertion technology spans two enzyme classes, large serine recombinases (LSRs) and retrotransposons, mined from the Company’s curated bioinformatic database through active learning-guided protein engineering.

Data from oral presentation #294, "Identification of novel, high-efficiency large serine recombinases and retrotransposases to enable targeted gene insertion," and poster #1331, "Active learning-guided optimization of large gene insertion effectors in mammalian cells,” include:

• Mined 164 proprietary LSRs from a catalog of 10+ billion proteins using a custom-built bioinformatic discovery platform, with ~80% of the mined LSRs demonstrating activity in mammalian cells, many with greater activity than Bxb1 (the field's benchmark)
• A CRISPR-recombinase approach achieved ~90% TCR knockout and 67% targeted CAR insertion at the TRAC locus, confirming high specificity of integration
• Active learning-guided machine learning optimization of recombinase proteins improved insertion potency by up to 3.4-fold versus wildtype proteins, demonstrating that AI can rapidly optimize proteins even without known structures
  

These results demonstrate a targeted insertion platform with multiple integration technologies applicable to in vivo and ex vivo therapeutic development, supported by generative AI for optimized programmability.

AI-Powered Protein Discovery: Generative AI-Designed Deaminases
ElevateBio's generative AI platform enables the design of proprietary editing proteins unconstrained by natural evolution, expanding the sequence space beyond what traditional protein engineering approaches can access. The Company is leveraging its generative AI capabilities to design novel recombinases and retrotransposons that transcend natural editing systems.

Data from poster #2303, "Leveraging generative AI to design novel, functional deaminases for adenine base editing,” include:

• Designed de novo adenine deaminases that break free from evolutionary constraints. These artificial sequences were generated from an AI model trained on 370,000 curated sequences from ElevateBio's 10+ billion protein catalog
• Achieved greater than 20% on-target A-to-G editing with exceptional specificity, including less than 1% bystander effects at the critical +2 position just two nucleotides from the on-target site, enabling targeting of previously intractable sites

These results represent a shift from nature-constrained to AI-powered protein engineering for next-generation therapeutics, establishing an end-to-end workflow for designing purpose-built therapeutic proteins.

LNP-Mediated Gene Editing: Optimized In Vivo Delivery to Liver and T Cells
ElevateBio's proprietary LNP platform enables in vivo delivery of gene editing systems, with iterative optimization of RNA chemistry, guide design, and editor proteins that translates across modalities and compounds across partner programs.

Data from oral presentation #295, "Iterative engineering and optimization strategies for maximizing LNP-mediated gene editing in the liver," and poster #3090, “Engineering hepatocyte-targeted LNPs for RNA and DNA delivery to primary human T cells”, include:

• Proprietary LNP formulation with iterative mRNA and guide RNA modifications increased nuclease editing potency in mice by approximately 3-fold against Hao1, a gene associated with primary hyperoxaluria, outperforming industry-leading vendors when combined with optimized guides
• In non-human primate (NHP) studies, LNP-delivered adenine base editing (ABE) system achieved therapeutically relevant protein reduction (protein <50% vs baseline) with minimal transient liver enzyme elevation, no immunosuppression required, and successful re-dosing at 10 weeks
• LNPs outperformed electroporation in delivering large gene insertion payloads to primary human T cells, showing dose-dependent improvements in insertion efficiency of up to 88% CD19-CAR+ vs <20% via electroporation
  

These results demonstrate that ElevateBio's optimization approach delivers pharmaceutically relevant editors via LNP, with learnings translating across editing modalities.

Epigenetic Editing: Programmable Gene Control Without Altering DNA
Epigenetic editing modulates gene expression without altering the underlying DNA sequence and is particularly suited for diseases driven by abnormal gene silencing or activation. This flexibility makes epigenetic editing well-suited for indications requiring correction of gene dosage and modulation of gene networks or pathways, where permanent DNA alterations may be unnecessary or undesirable.

Data from poster #1313 "Versatile and robust epigenetic modulation platform for programmable gene activation and repression," include:

• Compact RNA-guided epigenetic activators demonstrated therapeutically relevant 2-fold gene activation in a neuro-developmental disease model, enabling the precise increase needed to address haploinsufficiency
• Durable and tunable gene repression was demonstrated across multiple human cell types, including primary T cells. Long-term evaluation showed durable B2M repression in Jurkat cells persisted for at least four weeks following transient mRNA transfection.
• Multiplexed gene modulation with simultaneous repression of B2M and activation of CD25 in the same cell using separate RNA-guided epigenetic editors
  

These results establish epigenetic editing as a versatile new modality with precise tuning of gene expression using ElevateBio’s modular platform to target metabolic, immunogenic, and neuro-developmental pathways.

Further details on all of ElevateBio's presentations at ASGCT 2026 are available on the company's website at www.elevate.bio/ASGCT-2026.

About ElevateBio
ElevateBio is a technology-driven advanced therapy contract development and manufacturing organization (CDMO) powering the creation of life-transforming therapies. The company helps biopharmaceutical partners design, develop, and manufacture therapies from early discovery through commercialization, combining proprietary gene editing technologies and discovery services, cGMP manufacturing capabilities, and industry-leading expertise to accelerate development across a breadth of therapeutic approaches and modalities. Through continuous investment in automation, AI, and next-generation technologies, ElevateBio delivers the quality, speed, and scale partners need to bring advanced therapies to more patients. For more information, visit www.elevate.bio.

Investor contact:
Catherine Hu
chu@elevate.bio

Media contact:
DJ Webster
dwebster@elevate.bio‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬‬ 

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