Proprietary lipid nanoparticle for in vivo kidney delivery.
Leveraging the potential of nonviral delivery for greater safety and scalability of genetic medicines
Therapeutic payloads are directed to specific organs and cells, ensuring targeted delivery beyond the liver.
Minimize immune responses and eliminate the risk of insertional mutagenesis linked with viral vectors.
Greater scalability than viral vectors due to their simpler production processes and the ability to be synthesized in larger quantities more efficiently.
Ionizable Lipid
Cholestrol
Neutral Phospholipid
PEG Lipid
Ionizable lipids with defined ionization economy, high stability, and lower toxicity
LNP designed for reabsorption route, escaping glomerular filtration to be taken up by target cell types
Size, charge, and PEGylation of the LNPs designed toachieve target tissue and cell tropism
We depart from the current linear (sequence only) approach of designing gene editing medicines to a comprehensive 3-D genome approach for higher precision, safety, reliability
Every cell has the same DNA. However, different cells behave differently due to their epigenetics and the 3-D spatial structure of the genome. These parameters give us a unique opportunity to build greater precision in gene editing. Moreover, these epigenetic marks and 3Dspatial organization dictate the impact of genome editing in the short and long term.
Helex’s EPIC-Cure™ technology is built on proprietary cell and disease-specific 3D genome data, which improves the specificity of editing and minimizes off-target effects. The platform’s targeted guide-RNA design coupled with comprehensive guide-RNA safety analysis makes the approach to drug design highly efficient and safer for clinical applications. EPIC-Cure™ can be used to design short guide-RNAs for various genome editors.
Helex platform is built on proprietary bioinformatics and intelligent AI powered systems to identify unique sequences on the gene, or in the gene context to edit or modulate genes cell specifically. The platform enables the design of appropriate gene editing apparatus considering efficiency, safety including long term impact of the editing event.
EPIC-Cure™ platform’s integrative analysis looks at impact on changes in structure, chromatin accessibility, chromosome neighborhoods, transcription factories to estimate long-term safety.
Current industry measures off-target across the genome based on sequence homology; in exonic, intronic regions, cancer causing hotspots.
Autosomal Dominant Polycystic Kidney Disease
(due to mutations in PKD1 gene)
Autosomal Dominant Polycystic Kidney Disease
(due to mutations in PKD2 gene)
Autosomal Recessive Polycystic Kidney Disease
Tubulointerstitial Kidney Disease
