ElevateBio to Highlight Growth of its Genetic Medicine Business Driven By Gene Editing and Manufacturing Partnerships at the 42nd Annual J.P. Morgan Healthcare Conference

- Driving the future of genetic medicine through 20+ partners across ElevateBio’s integrated ecosystem of technology platforms and manufacturing capabilities

- Accelerating gene editing therapeutic development through Life Edit’s base editing partnerships with Novo Nordisk and Moderna, expansion into reverse transcriptase (RT) editing, and the addition of a novel lipid nanoparticle platform for non-viral delivery capabilities

- Expanding ElevateBio BaseCamp’s cGMP manufacturing capabilities to messenger RNA (mRNA) in addition to a wide range of existing cell therapy and viral vector manufacturing collaborations

- Expanding ElevateBio cGMP manufacturing footprint through partnership with the University of Pittsburgh with a groundbreaking at BaseCamp Pittsburgh planned for Q1 2024

WALTHAM, Mass.--(BUSINESS WIRE)--ElevateBio, LLC (ElevateBio), a technology-driven company focused on powering the creation of life-transforming genetic medicines, today announced advancements across its ecosystem of technology platforms and manufacturing capabilities that enable the biopharmaceutical industry to discover, develop, manufacture, and commercialize a broad range of advanced therapeutics. ElevateBio is accelerating the genetic medicine space through 20+ partnerships for a range of disease areas – including autoimmune, cardiometabolic, monogenic blood disorders, and hematologic and solid cancers – while expanding its offerings to partners ranging from large pharmaceutical companies to emerging biotechnology firms. Chairman and Chief Executive Officer, David Hallal, will provide a business update at the 42nd Annual J.P. Morgan Healthcare Conference on January 8, 2024 at 9:30 am PT.

ElevateBio is a biotechnology company commercializing its enabling technologies, manufacturing capabilities, and industry-leading expertise through partnerships to accelerate development across a breadth of therapeutic approaches and modalities including gene edited therapies, lentiviral gene therapies, T and HSC cell therapies, and mRNA vaccines. The Company’s integrated ecosystem combines its BaseCamp^® process development and current Good Manufacturing Practice (cGMP) manufacturing business with its growing technology stack that includes a full-spectrum gene editing platform, comprehensive cell engineering technologies, and an expanded viral and non-viral therapeutic delivery platform.

“2023 was a transformational year at ElevateBio as we continued to expand our leadership position in driving genetic medicines and scale our operations to serve an increasing number of corporate partners through all stages of development and commercialization,” said David Hallal, Chairman & CEO of ElevateBio. “With our expanding technology stack, capabilities, and the ability to streamline them with our manufacturing expertise, we are confident that we will continue to rapidly grow our business in 2024 by accelerating the development of genetic medicines for our partners and helping to bring these life-transforming therapies to patients.”

Key 2023 Highlights Across the ElevateBio Ecosystem

Partnerships:

ElevateBio speeds up therapeutic development of genetic medicines for biopharmaceutical partners through its integrated technology platforms and end-to-end manufacturing capabilities model.

• Grew to more than 20 partners, accelerating the development of investigational therapies for the treatment of blood cancers, solid tumors, genetic diseases, autoimmune disorders, viral infections, and more.
• Announced two transformative R&D collaborations with Moderna and Novo Nordisk to discover and develop gene editing therapeutics.
• Advanced partnership with the University of Pittsburgh with expectation to break ground in Q1 2024 for a new BaseCamp facility at Hazelwood Green.

BaseCamp:

BaseCamp is ElevateBio’s genetic medicine process development and cGMP business enabling biopharmaceutical partners with its end-to-end capabilities.

• Completed its highest annual number of technology transfers to date including process scale up, optimization, analytical development, and assay validation.
• Continued rapid translation of programs with GMP readiness for multiple partners.
• Expanding mRNA manufacturing capabilities to support leading global biopharmaceutical partners.
• Enabled partners with both viral (LentiPeak^TM lentiviral vector platform) and non-viral (Life Edit C-base editors) T cell engineering processes.
• Supported partners with BaseCamp’s LentiPeak^TM lentiviral vector platform, including final fill/finish.
• Completed process characterization for partners as part of BLA readiness.
• Supported partners with supply chain management, from cell procurement through final drug product shipment and receipt of clinical administration.
• Conducted development and generated data in collaboration with multiple cell and viral vector technology companies developing novel manufacturing devices, reagents, analytics, and automation to reduce cost of goods (COGs) of genetic medicines.

Life Edit:

Life Edit Therapeutics is ElevateBio’s next-generation gene editing business, offering 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.

• Developed additional C-base editors for gene knock-outs in novel cell therapies and A-base editors for in vivo gene editing therapeutics.
• Advanced the development of RT editors to rewrite DNA with precise insertions, deletions, and substitutions.
• Demonstrated multiplex base editing, including editing of up to five target genes using C-base editors.
• Demonstrated simultaneous knock-in/knock-out in primary T-cells.
• Demonstrated in vivo gene editing in the liver and brain in animal studies.
• Identified over 790,000 CRISPR-Cas systems and leveraging artificial intelligence (AI) to rapidly progress protein discovery and engineering efforts to discover and develop promising therapeutic options for genetic disorders and diseases previously considered untreatable.

Technologies:

ElevateBio’s technology-driven hybrid platforms model adds even more value with the addition of new technologies to enable partners across the genetic medicine industry.

• Acquired a novel lipid nanoparticles (LNP) platform, with both liver targeting and de-targeting potential, adding non-viral delivery to the Company’s existing viral delivery systems.
• Invested in the development of proprietary circRNA technology, potentially paving the way for groundbreaking treatments that harness the potential of RNA technology.
• Completed GMP capable novel reprogrammed induced pluripotent stent stems (iPSCs) suitable for the development of iPSC-derived cell 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 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 Twitter.