FDA Grants Fast Track to iECURE's ECUR-506 for Neonatal OTC Deficiency

PHILADELPHIA-- iECURE, Inc., a gene editing company dedicated to developing mutation-agnostic in vivo gene insertion therapies for liver disorders with substantial unmet needs, recently announced that its in vivo gene insertion program, ECUR-506, has received Fast Track designation from the U.S. Food and Drug Administration (FDA). The program is designed to treat neonatal onset Ornithine Transcarbamylase (OTC) deficiency, a severe metabolic disorder.

The Fast Track designation aims to facilitate the development and expedite the review of therapeutics intended to treat serious conditions with unmet medical needs. This designation allows for more frequent meetings and written communication from the FDA, ensuring that appropriate data is collected to support potential approval. Programs with Fast Track status may also qualify for accelerated approval and priority review if relevant criteria are met.

Joe Truitt, Chief Executive Officer of iECURE, stated, "Receiving Fast Track designation from the FDA validates the severe unmet need for patients with neonatal onset OTC deficiency and highlights the promising preclinical data we have gathered for ECUR-506. Fast Track designation could accelerate our efforts to make ECUR-506 available to physicians, which is crucial for these affected infants."

Earlier, ECUR-506 was awarded Rare Pediatric Disease and Orphan Drug designations by the FDA and Orphan designation by the European Commission for treating OTC deficiency.

iECURE's Phase 1/2 OTC-HOPE study, which is the first clinical trial for ECUR-506, is now enrolling newborn males with genetically confirmed neonatal onset OTC deficiency at the Great Ormond Street Hospital for Children in the United Kingdom. Additional trial sites in the United States and Australia are expected to begin enrollment later this year. The primary goal of the study is to evaluate the safety and tolerability of a single intravenous dose of ECUR-506. Secondary objectives include assessing the pharmacokinetics and efficacy of ECUR-506, as well as exploring disease-specific biological markers, developmental milestones, and quality of life.

ECUR-506 incorporates two adeno-associated virus (AAV) capsids, each carrying different genetic payloads. One vector contains an ARCUS® nuclease targeting the PCSK9 gene locus for gene editing, while the other contains a donor vector that inserts a healthy OTC gene. iECURE has licensed the ARCUS nuclease for ECUR-506 from Precision BioSciences. The engineered cut in the PCSK9 site serves as the insertion point for the functional OTC gene, potentially allowing for permanent expression of a healthy gene. This program marks the first clinical use of meganuclease-based in vivo gene insertion and is the first to be approved in the U.S. for study in infants using the ARCUS® nuclease technology.

The OTC-HOPE study focuses on newborn males up to seven months old diagnosed with severe neonatal onset OTC deficiency. The primary aim is to assess the safety and tolerability of a single intravenous dose of ECUR-506, with additional aims to evaluate its pharmacokinetics, efficacy, and effects on disease-specific markers, developmental milestones, and quality of life.

iECURE is a clinical-stage gene editing company focusing on therapies that utilize mutation-agnostic in vivo gene insertion to treat liver disorders with significant unmet needs. The company aims to replace and restore dysfunctional genes by inserting healthy copies, offering potentially curative benefits. iECURE's management team has extensive experience in global orphan drug and gene therapy clinical trials. They collaborate with the University of Pennsylvania's Gene Therapy Program (GTP), led by Dr. James M. Wilson, to leverage GTP's preclinical and translational expertise.

Precision BioSciences, the developer of the ARCUS® genome editing platform, focuses on creating lasting cures for genetic and infectious diseases. The ARCUS platform, known for its unique cutting mechanism, smaller size, and simpler structure, offers more defined therapeutic outcomes.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!