24 March 2023 - - The European Commission granted orphan designation for US-based gene editing company iECURE's lead product candidate GTP-506 for the treatment of Ornithine Transcarbamylase deficiency, the company said.

GTP-506 previously received Orphan Drug and Rare Pediatric Disease designations from the US Food and Drug Administration for the treatment of OTC deficiency.

Orphan designation in the European Union is granted by the European Commission based on a positive opinion issued by the European Medicines Agency Committee for Orphan Medicinal Products.

The European Commission grants orphan status for products intended for the treatment, prevention, or diagnosis of life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the EU, and where there is no satisfactory method of treating, preventing, or diagnosing such condition authorized for marketing in the EU or if such a method exists, the product represents a significant benefit to those affected by the condition.

Orphan designation provides companies with certain benefits and incentives in the EU, including clinical protocol assistance, possible waivers or reductions in regulatory fees and ten years of market exclusivity after approval.

OTC deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for the detoxification of ammonia. Individuals with OTC deficiency can build-up excessive levels of ammonia in their blood potentially resulting in devastating consequences, including 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's approach to gene editing for its initial programs, including OTC deficiency, relies on the delivery of twin adeno-associated virus capsids carrying different payloads.

GTP-506 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 has 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.

iECURE is a gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need.

Precision BioSciences, Inc. is a clinical stage biotechnology (NASDAQ: DTIL) offering a novel and proprietary ARCUS genome editing platform.

The University of Pennsylvania and Dr. Wilson each hold equity interests in iECURE. Penn also receives significant sponsored research support from the company, and both Penn and Dr. Wilson stand to benefit from licensing revenues received from iECURE based on successful technology development and commercialization of the technologies licensed from Penn. Dr. Wilson serves as chief scientific Advisor for iECURE.