PHILADELPHIA, PA — iECURE said the U.S. Food and Drug Administration has selected ECUR-506, the company’s investigational gene insertion therapy for neonatal-onset ornithine transcarbamylase deficiency, for the agency’s Chemistry, Manufacturing, and Controls Development and Readiness Pilot Program as the company advances the treatment through clinical development.
The Philadelphia-based company said the FDA invited ECUR-506 into the program, which selects up to nine drug and biologic development programs each year for increased engagement on manufacturing and product readiness.
According to iECURE, the pilot program is intended to help companies align manufacturing development with accelerated clinical timelines for therapies aimed at serious unmet medical needs.
The company said participation is expected to provide more frequent discussions with the FDA on chemistry, manufacturing, and controls strategy as it works toward a potential biologics license application for ECUR-506 and expands manufacturing capabilities for its broader gene insertion platform.
Chief Executive Officer Joe Truitt said the program is intended to help align clinical development with manufacturing readiness for complex genetic medicines for rare diseases.
ECUR-506 is being evaluated in the ongoing OTC-HOPE clinical trial in male infants with neonatal-onset ornithine transcarbamylase deficiency, according to the company.
iECURE said the therapy has also received Regenerative Medicine Advanced Therapy and Fast Track designations from the FDA.
The company said it expects to release additional data from the OTC-HOPE trial in the first half of 2026.
According to iECURE, the first-in-human study is enrolling eligible male infants up to seven months old with genetically confirmed neonatal-onset ornithine transcarbamylase deficiency. The trial is designed to assess the safety, tolerability and efficacy of a single intravenous dose of ECUR-506, as well as pharmacokinetics, clinical outcomes, disease-specific biologic markers, developmental milestones and quality of life.
The main study runs for up to 10 months, including screening, stabilization, dosing eligibility, treatment administration and six months of follow-up, the company said. Participants then transition to a 14.5-year long-term follow-up study.
iECURE said ECUR-506 is designed to restore enzyme activity by inserting a functional copy of the OTC gene into the PCSK9 gene locus in liver cells. The therapy uses two adeno-associated virus vectors carrying separate payloads, including an ARCUS nuclease and a functional OTC gene for targeted insertion.
The company said it licensed the ARCUS nuclease for ECUR-506 from Precision BioSciences (Nasdaq: DTIL) as part of a broader agreement covering four gene insertion programs, including OTC, CTLN1 and PKU.
More information about the trial is available at https://OTC-HOPE.com. More information about the company is available at https://iecure.com.
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