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Company Type: Therapeutic development

Main focus: Developing in vivo gene insertion therapies to treat liver diseases

Company stage: Clinical

Diseases: “Loss of function” liver disorders

Genome-editing tool: ARCUS® nuclease

Funding stage: Series A

Location: Blue Bell, Philadelphia, USA



Partners: University of Pennsylvania

iECURE’s approach to gene editing for its initial programmes, including a rare liver disorder known as OTC deficiency, relies on the delivery of twin adeno-associated virus (AAV) capsids carrying different payloads. iECURE has licensed the ARCUS nuclease for GTP-506 from Precision BioSciences. GTP-506 comprises two vectors, an ARCUS® nuclease vector (GTP-506A) targeting gene editing in the well-characterised PCSK9 gene locus and a therapeutic donor vector (GTP-506D) that inserts the OTC gene to provide the desired genetic correction. The cut in the PCSK9 site serves as the insertion site for the therapeutic gene, providing a potential path to permanent expression of a healthy gene.



Company: iECURE
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