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DURHAM, N.C. – Precision BioSciences, Inc. (NASDAQ:), a gene editing company, announced today that its partner iECURE has received approval from the U.K. Medicines & Healthcare products Regulatory Agency (MHRA) to expand the Phase 1/2 OTC-HOPE study into the United Kingdom. This study evaluates ECUR-506, a treatment for Ornithine Transcarbamylase (OTC) deficiency in infants, which incorporates the company’s proprietary ARCUS nuclease for gene editing.
The OTC-HOPE study aims to explore the potential of ECUR-506 in treating OTC deficiency, a genetic disorder that leads to excessive ammonia levels in the blood, causing severe neurological damage or even death. The current standard treatment for severe early-onset OTC deficiency is a liver transplant, with no existing medical therapies able to correct the disease.
ECUR-506 consists of two adeno-associated virus (AAV) vectors, one carrying the ARCUS nuclease and the other inserting a functional OTC gene. The MHRA’s approval to conduct trials in the UK follows a previous nod from the Australian Therapeutic Goods Administration (TGA).
The Phase 1/2 clinical trial, named OTC-HOPE, is a first-in-human study targeting baby boys with genetically confirmed severe neonatal onset OTC deficiency. The trial will test up to two dose levels of ECUR-506, focusing on the safety, tolerability, and efficacy of the treatment, as well as its impact on disease-specific biological markers and patient quality of life.
Michael Amoroso, CEO of Precision BioSciences, remarked on the milestone, highlighting it as a validation of ARCUS’s gene-editing capabilities. He also pointed out the importance of regulatory progress as a reflection of the company’s broader strategy to partner with development partners while advancing its own hepatitis B program.
iECURE, the clinical-stage company conducting the OTC-HOPE study, is collaborating with the University of Pennsylvania’s Gene Therapy Program to bring forward potential gene editing therapies for liver disorders.
The information for this article is based on a press release statement from Precision BioSciences, Inc.
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