The startup is a clinical-stage gene editing company focused on treating rare liver disorders in children through mutation-agnostic in vivo gene insertion. By knocking in a functional copy of a dysfunctional gene, the platform aims to restore gene function and provide long-term therapeutic benefits.
Funding
$115M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Many rare, monogenic liver diseases, particularly in children, lack effective and durable treatments, leading to significant morbidity and mortality. Traditional gene therapy approaches often fail to produce lasting results due to rapid cell division in children's livers, and mutation-specific therapies are resource-intensive given the numerous potential disease-causing mutations.
Solution
iECURE is developing mutation-agnostic _in vivo_ gene insertion therapies to treat rare pediatric liver diseases. Their approach involves inserting a full, healthy copy of the dysfunctional gene into the patient's chromosomes, enabling long-term, stable expression of the gene and potentially eliminating disease symptoms. The company's lead program, ECUR-506, uses an ARCUS nuclease to cut the genome at the PCSK9 site, allowing for insertion of a functional OTC gene. This method aims to provide a curative therapeutic benefit by replacing the function of the dysfunctional gene, regardless of the specific mutation causing the disease.
Target Audience
The primary target audience includes patients, particularly newborn males, with severe neonatal onset urea cycle disorders and other rare pediatric liver diseases, as well as the physicians and medical centers specializing in the treatment of these conditions.
Features
- _In vivo_ gene insertion approach that is independent of specific disease-causing mutations
- Utilizes an ARCUS nuclease to target the PCSK9 site for gene insertion
- Employs adeno-associated virus (AAV) vectors to deliver the nuclease and healthy gene copy
- Focuses on liver disorders, particularly in children, with programs targeting ornithine transcarbamylase (OTC) deficiency, citrullinemia type 1 (CTLN1), and phenylketonuria (PKU)
- ECUR-506, the lead candidate, is undergoing Phase 1/2 clinical trials for neonatal onset OTC deficiency
- Collaboration with the University of Pennsylvania’s Gene Therapy Program (GTP) for preclinical development
- ECUR-506 has received FDA Fast Track designation, Rare Pediatric Disease designation, and Orphan Drug designation, as well as Orphan designation from the European Commission