Iecure Raises $65 Million to Advance In Vivo Gene Editing Programs for Rare Pediatric Liver Diseases

In-vivo gene editing biotech Iecure completed a $65 million series A-1 financing to get its lead gene replacement therapy for a rare inherited liver disease known as ornithine transcarbamylase deficiency into the clinic.

Photo: Joseph Truitt, CEO of Iecure

Ornithine transcarbamylase (OTC) deficiency is the most common urea cycle disorder, an inherited metabolic condition caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia. Individuals with OTC deficiency can build-up excessive levels of ammonia in their blood, potentially resulting in devastating consequences, including cumulative and irreversible neurological damage, coma, and death. The severe form of the condition emerges shortly after birth and is more common in boys than girls. The only treatment for early onset severe OTC deficiency is a liver transplant. Currently available medical therapies do not correct the disease and do not eliminate the risk of life-threatening symptoms or crises.

Iecure is developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need. The company, co-founded by Jim Wilson, head of the University of Pennsylvania’s Gene Therapy Program, believes its approach has the potential to replace and restore the function of a dysfunctional gene by knocking-in a healthy copy, regardless of mutation, to offer durable gene expression and long-term, potentially curative, therapeutic benefit. The approach relies on the delivery of twin adeno-associated virus capsids carrying different payloads.

Lead candidate GTP-506 for OTC deficiency comprises two vectors, an ARCUS nuclease vector (GTP-506A) targeting gene editing in the well-characterized PCSK9 gene locus and a therapeutic donor vector (GTP-506D) that inserts the OTC gene to provide the desired genetic correction. Iecure licensed the ARCUS nuclease for GTP-506 from Precision BioSciences. 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. The FDA has granted both Orphan Drug and Rare Pediatric designations to GTP-506 for the treatment of OTC deficiency.

Novo Holdings and LYFE Capital co-led the financing with significant participation from existing investors Versant Ventures and OrbiMed Advisors. This financing, coupled with the $50 million raised in the prior Series A financing announced in September 2021, will bring the company’s total funds raised to $115 million.

“In the last year, we have made significant progress in both advancing development of our lead program for neonatal onset OTC and building a world-class team with an extensive track record in developing and commercializing novel therapies,” said Joseph Truitt, CEO of Iecure. “We believe that this funding will enable us to execute all the tasks necessary to begin clinical development of what could be the first mutation-agnostic in vivo gene insertion therapeutic program.”

Proceeds from the Series A-1 financing will enable the advancement of GTP-506, including funding IND-enabling studies, starting clinical trials (subject to regulatory approval), and achieving early human data readouts. In addition, the Series A-1 financing is expected to fuel further progress on Iecure’s portfolio of gene editing products for the treatment of patients with rare liver diseases, including citrullinemia type 1 (CTLN1) and phenylketonuria (PKU).

Author: Rare Daily Staff