iECURE focuses on mutation-agnostic in vivo gene insertion (knock-in) editing to replace or restore dysfunctional genes in the liver, aiming for durable gene expression and potential curative benefit. Its lead program, GTP-506, uses twin AAV capsids: an ARCUS® nuclease vector targeting the PCSK9 locus and a donor vector that inserts the OTC gene; ARCUS was licensed from Precision BioSciences. The company has collaboration ties with the University of Pennsylvania Gene Therapy Program, which helped generate its initial pipeline, and Penn holds equity and receives sponsored research support. FDA has granted Orphan Drug and Rare Pediatric Designations to GTP-506 for OTC deficiency. iECURE says its management team has experience running global orphan drug and gene therapy trials and commercializing products. The company has raised capital to advance IND-enabling studies, begin clinical trials subject to regulatory approval, and seek early human data readouts across its liver-focused pipeline, including programs for CTLN1 and PKU. iECURE develops mutation-agnostic in vivo gene insertion therapies that insert full, functioning genes into patients' chromosomes to provide durable, long-term expression. The company’s initial focus is on liver disorders and it is advancing a pipeline of up to 13 programs developed in the University of Pennsylvania Gene Therapy Program (GTP), with exclusive rights to three liver programs and options on more than 10 additional assets. In primate models, iECURE’s programs have demonstrated integration of wild-type genes into the genome and long-term expression. Its initial programs use an endonuclease-based editing technology delivered via AAV, and subsequent programs may incorporate other delivery technologies or in-license additional capabilities. Under a joint R&D initiative, GTP will lead preclinical research until IND submissions, after which iECURE will execute clinical studies and prepare for potential commercialization. Leadership and the board combine industry veterans and investors, and the company partners closely with Penn and Dr. James Wilson on translational research.

Total raised

$115M

Funding rounds

2

Latest round

Series A

Latest activity

Nov 2022