Proprietary lipid nanoparticle for in vivo kidney delivery.
Targeted Delivery
Therapeutic payloads are directed to specific organs and cells, ensuring targeted delivery beyond the liver.
Lower Immunogenicity
Minimize immune responses and eliminate the risk of insertional mutagenesis linked with viral vectors.
Scalability
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
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 3D spatial organisation dictate the global impact of edit in the short and long term.
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.
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.
Preventing editing in unwanted tissues in cases of vector biodistribution through our Double Lock Safety system. Tissue specific vector modifications (first lock) combined with Hele-GUIDE™ tissue specific gRNAs (Second lock) for greater safety.
As we unlock the tremendous potential for in vivo gene editing, one of the biggest challenges is editing in unwanted tissues due to vector biodistribution. Hele-GUIDE™ is our proprietary technology that allows us to make the guide RNAs tissue specific to prevent editing irrespective of transduction into unwanted tissues.
Stargardt Disease
Undisclosed
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
