Precision BioSciences Gets FDA Fast Track for ECUR-506 in Neonatal OTC Deficiency

Precision BioSciences, Inc., a leading gene editing company based in Durham, N.C., has announced that its partner iECURE has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for its gene insertion program, ECUR-506. This program aims to treat neonatal onset Ornithine Transcarbamylase (OTC) deficiency through the use of a PCSK9-specific ARCUS nuclease, licensed from Precision BioSciences.

The Fast Track designation is designed to accelerate the development and review process for drugs that address severe conditions with unmet medical needs. Therapies granted this designation may benefit from more frequent meetings and written communication with the FDA. Additionally, they become eligible for expedited approval processes, including accelerated approval and priority review, as long as they meet the relevant criteria. ECUR-506 had already received Rare Pediatric Disease and Orphan Drug designations from the FDA, as well as Orphan designation from the European Commission for treating OTC deficiency.

Michael Amoroso, Chief Executive Officer of Precision BioSciences, acknowledged the significance of this regulatory milestone. He highlighted that the Fast Track designation underscores both the urgent need for treatments targeting neonatal onset OTC deficiency and the therapeutic promise of iECURE's novel gene insertion method. Amoroso further noted that this development validates Precision’s ARCUS platform, which is also being used to advance a hepatitis B program expected to reach IND and/or CTA stage in 2024.

The OTC-HOPE study, a Phase 1/2 clinical trial, is the first human study aimed at investigating ECUR-506 in newborn males diagnosed with neonatal onset OTC deficiency. The primary objective of the study is to assess the safety and tolerability of a single intravenous dose of ECUR-506. The study will also evaluate the pharmacokinetics and efficacy of the treatment. Enrollment for the OTC-HOPE study is currently open in the United Kingdom, with U.S. and Australian sites expected to begin enrolling later in the year.

Precision BioSciences’ ARCUS platform represents a significant advancement in the field of gene editing. The platform’s unique approach includes using nucleases for gene insertion, elimination, and excision. Unlike other gene editing technologies, ARCUS nucleases are characterized by their smaller size, simpler structure, and precise cutting capabilities, which contribute to their potential for generating defined therapeutic outcomes.

ECUR-506 employs a dual-vector system developed by iECURE. One vector delivers an ARCUS nuclease that targets the PCSK9 gene locus for gene editing, while the other vector inserts a functional OTC gene. The cut at the PCSK9 site serves as an insertion point for the OTC gene, potentially leading to permanent expression of a healthy gene. The OTC-HOPE study is the first clinical program to utilize this approach for in vivo gene insertion.

OTC deficiency, the most prevalent urea cycle disorder, is an inherited condition marked by a genetic defect in a liver enzyme critical for ammonia detoxification. Elevated ammonia levels in affected individuals can result in severe, sometimes irreversible, neurological damage, coma, or even death. The severe form of this disorder typically manifests shortly after birth and predominantly affects males. Currently, the only corrective treatment for severe neonatal onset OTC deficiency is a liver transplant, as existing medical therapies do not adequately mitigate life-threatening symptoms.

iECURE, a clinical-stage gene editing company, focuses on therapies that use in vivo gene insertion to address liver disorders with significant unmet needs. Their strategy involves replacing and restoring dysfunctional genes by inserting healthy copies, aiming for long-term therapeutic benefits. The company collaborates with the University of Pennsylvania’s Gene Therapy Program, led by James M. Wilson, M.D., Ph.D., leveraging its expertise to develop their treatment pipeline.

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