LETI-101: A Novel Precision Editing Approach as a Potential One-Time Treatment for Huntington’s Disease
A promising development-ready candidate seeking partnership
Program Overview & Partnership Opportunity
ElevateBio Life Edit is seeking a strategic partner to advance LETI-101 through clinical development. LETI-101, an investigational gene editing therapy for the treatment of Huntington’s disease, has demonstrated compelling preclinical data and received favorable regulatory feedback, positioning it as a partnership-ready asset for clinical development.
Compelling Preclinical Data
>80% reduction of mutant huntingtin protein in vivo while preserving wild-type protein
Regulatory Alignment
Gained UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) alignment on CMC and development strategy
Development Ready Program
Partnership-ready development candidate poised to begin IND-enabling studies
Technology Advantage Behind LETI-101
LETI-101 combines Life Edit’s proprietary CRISPR nuclease with AAV5 delivery to selectively target the disease-causing gene in Huntington’s disease.
Life Edit’s allele-selective approach recognizes the difference between wild-type HTT and mutant HTT by targeting a specific T-SNP recognition site in Exon 50, allowing selective editing of only the disease-causing gene.
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Allele-Selective Approach
Targets the T allele of an exonic SNP in the HTT gene to treat HD -
Potential One-Time Treatment
Potential for durable benefit without chronic administration -
Preservation of Wild-Type HTT
Maintains essential wild-type huntingtin protein needed for cellular function -
Demonstrated Safety in NHP studies
Well-tolerated delivery and robust expression across critical HD-affected brain regions in NHP studies
Learn More About the Science Behind LETI-101
Life Edit’s Gene Editing Platform
Life Edit is the gene editing and R&D technology business of ElevateBio, a technology-driven company built to power the development of transformative genetic medicines. Life Edit has one of the world’s largest and most diverse libraries of RNA-guided nucleases (RGNs), base editors, and reverse transcriptase editors, that provide flexible editing and unprecedented access to the genome.
LETI-101 showcases the power of Life Edit’s expansive gene editing capabilities and ElevateBio’s integrated approach to genetic medicine development.
Extensive Nuclease Collection
Our diverse library of nucleases with varied Protospacer Adjacent Motifs (PAM) recognition sequences enables targeting of previously inaccessible genetic sites
Compact Size
Proprietary nucleases are smaller than standard CRISPR tools, enabling packaging in a single viral vector
Versatile Applications
The platform enables allele-selective editing, base editing, and reverse transcriptase editing – also known as prime editing – providing flexibility to make any edit, anywhere
Scalable Manufacturing
Seamless transition from research to cGMP manufacturing, supported by ElevateBio BaseCamp
LETI-101 represents just one application of our gene editing platform. Powered by artificial intelligence, we are rapidly progressing CRISPR discovery and engineering efforts to enable the development of gene editing therapies for a broad range of challenging disorders, including monogenic and polygenic diseases.
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