Therapeutic Applications
Enabling the discovery and development of genomic medicines for the biopharmaceutical industry.
// Gene Editing and
R&D Technologies
From Platform to Patients
The Life Edit platform, combined with ElevateBio’s industry-leading manufacturing and development expertise, enables us and our partners to bring novel treatments to patients across previously intractable diseases. Our approach matches the optimal editing modality and delivery method to each therapeutic challenge, expanding the range of addressable genetic disorders.
Flexible Therapeutic Approaches
Technology Platforms
We have built one of the largest proprietary toolboxes of genome editing technologies that provides flexible editing and unprecedented access to the genome.
Therapeutic Applications
Our technology can be applied in an array of therapeutic designs and applications, unlocking new possibilities for addressing a wide range of diseases and disorders.
LETI-101: A Novel Approach to Treat Huntington’s Disease
We are developing an investigational gene editing therapy for Huntington’s disease (HD), a rare inherited neurodegenerative disorder caused by CAG trinucleotide expansion in the huntingtin gene. This mutation produces a toxic protein (mutHTT) that aggregates in the brain, causing progressive neurodegeneration in young adults.
Our approach precisely targets the mutHTT protein while preserving essential wild-type huntingtin protein. Using our collection of RNA-guided nucleases, we can target both T and C alleles of a PAM-altering SNP for allele-specific editing of the mutHTT gene—a level of precision that could transform treatment for this devastating disease.
We are advancing this program through internal development while seeking partnership opportunities to accelerate clinical development and deliver a life-transforming therapy to HD patients.
Read Amy Pooler’s, SVP of Research & Development, byline article on LETI-101
Partnership-Powered Innovation
We work with biopharmaceutical partners to tackle their most challenging genetic targets. Our platform capabilities are validated through strategic collaborations with Novo Nordisk, advancing base editing for rare genetic disorders and cardiometabolic diseases, and Moderna, combining their mRNA platform with our gene editing technologies for in vivo therapies. These partnerships demonstrate our ability to accelerate breakthrough therapies across diverse therapeutic areas while expanding access to our gene editing innovations.
The Breadth of our Platform and Technology Offers Unmatched Editing Capabilities
Gene Editing Toolbox
We have a powerful suite of proprietary gene editing technologies to accelerate the development of life-changing and curative therapies for patients.
AI & Protein Engineering
We harness artificial intelligence to accelerate CRISPR discovery, combining our protein engineering expertise with a vast library of over 20 billion proteins to design optimal editing systems for thousands of previously untreatable genetic disorders.
Partner with ElevateBio®
Wherever you are in your cell and gene therapy product lifecycle, we can strengthen and accelerate the development of your transformative therapies with our enabling technologies unmatched manufacturing capabilities.
// Work with us
