MHRA Approves Phase 1/2 Expansion for iECURE's ARCUS Gene Editing in OTC Deficiency

Innovative gene editing therapies are at the forefront of medical advancements, aiming to provide curative solutions for patients suffering from genetic disorders. Precision BioSciences, Inc., a company at the cutting edge of gene editing technology, has developed the ARCUS® platform to facilitate sophisticated gene edits, including gene insertion, excision, and elimination. This technology is designed to offer long-lasting clinical benefits, particularly for children with severe genetic conditions that lack effective treatments.

Recently, Precision BioSciences' partner, iECURE, has achieved a significant milestone with the approval from the U.K. Medicines & Healthcare products Regulatory Agency (MHRA) for a Clinical Trial Authorization (CTA) application. This approval allows for the expansion of the Phase 1/2 OTC-HOPE study into the United Kingdom, which is evaluating ECUR-506 for the treatment of Ornithine Transcarbamylase (OTC) deficiency in infants. The study is a testament to the potential of ARCUS nucleases to efficiently insert genes and restore function, marking a step forward in the quest to revolutionize treatment for OTC deficiency.

OTC deficiency is a common urea cycle disorder, an inherited metabolic disorder caused by a defect in a liver enzyme that detoxifies ammonia. This condition can lead to life-threatening symptoms such as neurological damage, coma, and even death. The current standard of care, a liver transplant, is not a cure but a corrective measure. Medical therapies available do not address the root cause of the disease.

Precision BioSciences' ARCUS® platform stands out due to its unique approach to genome editing, which includes a smaller size and simpler structure compared to other technologies. The platform's capabilities aim to drive more precise and defined therapeutic outcomes. Precision BioSciences is leveraging this technology to develop a pipeline of in vivo gene editing candidates designed to cure a broad range of genetic and infectious diseases where current treatments are inadequate.

iECURE's gene editing strategy for conditions like OTC deficiency involves the use of two adeno-associated virus (AAV) capsids, each with different payloads. ECUR-506, the therapy under investigation, consists of two vectors: one carrying an ARCUS® nuclease targeting gene editing at the PCSK9 gene locus and another that inserts a functional OTC gene. The goal is to achieve permanent expression of a healthy gene, offering a potential cure.

The OTC-HOPE study is a Phase 1/2 clinical trial for baby boys with genetically confirmed OTC deficiency. It will test the safety, tolerability, pharmacokinetics, and efficacy of ECUR-506, as well as its impact on disease-specific biologic markers, developmental milestones, and quality of life.

iECURE is a clinical-stage company focused on developing therapies using in vivo gene insertion to treat liver disorders with significant unmet needs. The company's approach aims to replace dysfunctional genes with healthy copies, offering durable gene expression and potentially curative therapeutic benefits. iECURE collaborates with the University of Pennsylvania’s Gene Therapy Program, utilizing its expertise and infrastructure to advance its pipeline of potential product candidates.

This collaboration and the advancements in gene editing technology exemplify the commitment to finding cures for devastating genetic disorders. The progress made by Precision BioSciences and iECURE signifies a new era in medicine where the promise of gene editing is being realized, offering hope to patients and their families.