PHILADELPHIA, PA — A rare-disease gene therapy developed in Philadelphia has cleared a major regulatory hurdle, positioning it on an accelerated path toward approval in the United States and the United Kingdom.
iECURE, Inc. announced that the U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ECUR-506, its investigational in vivo targeted gene insertion therapy for neonatal onset ornithine transcarbamylase deficiency, one of the most severe and life-threatening pediatric metabolic disorders.
The RMAT designation provides iECURE with intensive FDA guidance and access to expedited development and review pathways, reflecting early clinical evidence that ECUR-506 could address a profound unmet medical need. The designation was established under the 21st Century Cures Act to speed therapies for serious or life-threatening diseases toward patients.
In a parallel regulatory milestone, iECURE said it has reached alignment with the FDA on the primary and key secondary endpoints, comparators, and sample size for its ongoing OTC-HOPE study—an agreement that could allow the trial to support a future Biologics License Application for ECUR-506.
The momentum is not limited to the U.S. ECUR-506 has also been awarded an Innovation Passport under the United Kingdom’s relaunched Innovative Licensing and Access Pathway, or ILAP. Only three investigational medicines were selected under the new ILAP framework, all focused on rare diseases, placing ECUR-506 among a small group of therapies prioritized for accelerated development and patient access in the UK.
“RMAT designation represents an important validation of ECUR-506 and the clinical data generated to date,” said Joe Truitt, chief executive officer of iECURE. He said the combination of FDA alignment on the OTC-HOPE study and the UK Innovation Passport provides a clear regulatory roadmap toward delivering a potentially durable treatment for affected infants and families.
Neonatal onset OTC deficiency is a rare genetic disorder that can cause rapid ammonia accumulation in the blood, leading to severe neurological damage or death if untreated. ECUR-506 is designed to address the disease at its biological root through targeted gene insertion. The therapy uses two adeno-associated virus vectors—one carrying an ARCUS nuclease to create a precise insertion site and another delivering a functional copy of the OTC gene.
The ARCUS nuclease technology is licensed from Precision BioSciences (Nasdaq: DTIL).
Encouraging data from the ongoing OTC-HOPE trial underpin the regulatory progress, including a complete clinical response observed in the first infant treated with ECUR-506, according to the company.
The OTC-HOPE study is a first-in-human trial enrolling male infants up to seven months old with genetically confirmed neonatal onset OTC deficiency. The trial is evaluating safety, tolerability, pharmacokinetics, and early signs of efficacy following a single intravenous dose, with participants transitioning into a long-term follow-up study lasting more than 14 years.
Details about the study are available at https://OTC-HOPE.com.
Beyond OTC deficiency, iECURE said insights from the program may inform broader applications of its targeted gene insertion platform across other indications, potentially expanding the impact of the technology well beyond a single rare disease.
For families facing a diagnosis that has long carried devastating odds, the regulatory breakthroughs signal that a fundamentally new treatment approach may be moving closer to reality.
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