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.
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.
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.
As we unlock the tremendous potential for in vivo gene editing, one of the biggest challenges is editing of unwanted tissues due to vector biodistribution. Helex is 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. 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
