iECURE

iECURE specializes in gene editing to develop novel in vivo (gene editing done inside the body) therapeutics for a range of therapeutic areas, starting off with rare pediatric liver diseases. It leverages ARCUS nucleases (discovered by Precision Biosciences) to insert mutation-agnostic, healthy copies of disease-causing genes to act independently unaffected by mutations, unlike approaches that directly target the disease-causing gene that is subject to multiple mutations. 

As of March 2024, the company had five wholly-owned programs. Its lead candidate for Ornithine Transcarbamylase (OTC) – neonatal onset was just entering phase 2 testing, while its other candidates were in discovery and preclinical stages.

Key customers and partnerships

The company has a close partnership with the University of Pennsylvania’s Gene Therapy Program (GTP) (December 2021) to leverage its expertise in vector engineering, gene therapy, and gene editing. The university also holds an equity share in the company. 

The company’s partnership with Precision Biosciences was initiated in September 2021 to advance Precision’s ARCUS-based gene editing therapy for FH and leverage ARCUS nucleases for iECURE’s programs. It also partnered with the Center for Breakthrough Medicines (CBM ) in May 2022 to source good manufacturing practices (GMP) grade adeno-associated virus (AAV) vectors for iECURE’s clinical studies. 

Funding and financials

In its Series A-1 funding round in November 2022, led by Novo Holdings A/S and LYFE Capital, iECURE raised USD 65.1 million to advance its clinical development pipeline and to accelerate the development of its gene editing products for rare liver diseases. As of January 2023, iECURE had raised USD 115 million.