ElevateBio Announces Advancements of its Proprietary Lipid Nanoparticle (LNP) Delivery Platform with Liver Targeting and De-Targeting Potential

• Proprietary LNP platform delivered Life Edit gene editing system in murine studies, leading to high in vivo editing with low immunogenicity profile.
• Non-viral delivery expands the Company’s capabilities, enhancing flexibility in therapeutic design and applications for partners and internal programs

Waltham, Mass., October 16, 2024 – ElevateBio, LLC (ElevateBio), a technology-driven company focused on powering the creation of life-transforming genetic medicines, today announced the successful in vivodelivery of Life Edit gene editing systems with its proprietary LNP platform to enable nuclease editing in the liver in murine models. Data on the LNP platform, including additional data demonstrating durable activity in non-human primates (NHPs), will be presented by Life Edit, ElevateBio’s gene editing and R&D technology business, at the 6th Annual Formulation & Delivery US Congress in San Diego, California at 5:15 p.m. PST today.

The proprietary LNP delivery system provides a non-viral delivery option designed to optimize the precision and activity of gene editing therapies. In murine models, the platform demonstrated the ability to deliver gene editing systems precisely to the liver while maintaining a low immunogenicity profile. It also has the potential for repeat dosing with minimal immune response for other therapeutic applications. Importantly, the platform demonstrates potential for liver de-targeting, allowing it to bypass the liver and selectively target other tissues, expanding its flexibility to address a range of genetic conditions.

“This is an important milestone for Life Edit as we advance our proprietary delivery platforms alongside our base and reverse transcriptase editing capabilities to develop and deliver safe and efficacious gene editing therapeutics for a broad range of genetic diseases,” said Tedd Elich, Chief Scientific Officer at Life Edit. “As part of ElevateBio’s broad collection of cutting-edge technologies spanning cell, RNA, and gene editing, this platform reinforces our mission to offer comprehensive solutions to innovators across the industry that are developing genetic medicines.”

ElevateBio’s Life Edit gene editing platform is available to make a broad spectrum of edits – including base editing and reverse transcriptase editing – for the development of ex vivo and in vivo therapies to treat and potentially cure disease. The non-viral LNP platform complements the Company’s viral delivery systems for precise DNA targeting in cells and tissues. The flexibility of viral and non-viral delivery systems allows tailored solutions for a broad spectrum of genetic diseases, from single-gene disorders to complex multi-gene conditions.

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 multiple R&D technology platforms – including Life Edit, a next-generation, full-spectrum gene editing platform; comprehensive cell engineering technologies; and an expanded viral and non-viral therapeutic delivery platform – 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.

About Life Edit Therapeutics Inc.

Life Edit, ElevateBio’s next-generation gene editing business, offers one of the world’s largest and most diverse libraries of RNA-guided nucleases (RGNs), base editors, and reverse transcriptase (RT) editors that provide flexible editing and unprecedented access to the genome. The platform allows Life Edit to target any genomic sequence and potentially develop novel human therapeutics for the most challenging genetic diseases by enabling ex vivo engineering for cell therapies and regenerative medicines and in vivo delivery of gene therapies. In addition to developing its own pipeline of cell and gene therapies, Life Edit will continue to strengthen its platform of genome-editing enzymes, provide gene-editing expertise to strategic partners, and form other third-party partnerships to discover and develop new therapies.

For more, visit www.lifeeditinc.com or follow Life Edit on LinkedIn or X.

Investor contact:
Catherine Hu
[email protected]

‪Media contact:
DJ Webster
[email protected]