iECURE Selected for U.S. FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot Program to Support Manufacturing Readiness of ECUR-506 Ahead of Planned BLA Submission
FDA Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) program supports manufacturing readiness for therapies with expedited clinical development timelines
ECUR-506 selected as one of up to nine programs annually
Participation enables additional FDA engagement on CMC strategy ahead of a potential BLA submission
iECURE, Inc., a clinical-stage genome editing company developing variant-agnostic in vivo targeted gene insertion therapies for the treatment of severe, inherited, neurometabolic disorders, today announced that the U.S. Food and Drug Administration (FDA) has selected ECUR-506, the company's investigational in vivo targeted gene insertion therapy for neonatal-onset ornithine transcarbamylase (OTC) deficiency, to participate in the Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) Program. iECURE was invited by the FDA to participate in the CDRP program, which selects up to nine development programs annually across the FDA's two product review centers for biologics and drugs.
The FDA established the CDRP program to support therapies with accelerated clinical development timelines by facilitating earlier and increased engagement between sponsors and the FDA on CMC development strategies and goals. The program is designed to help ensure that CMC development and readiness keeps pace with clinical development so that promising therapies addressing serious unmet medical needs may reach patients sooner.
Participation in the CDRP program is expected to enable iECURE to have more frequent discussions with the FDA on CMC strategy and activities as the company advances ECUR-506 toward a potential BLA application and if approved, commercialization, and continues to build manufacturing capabilities supporting its broader gene insertion platform.
"Selection for the FDA's CDRP program underscores the importance of aligning clinical progress with manufacturing readiness when developing complex genetic medicines for serious rare diseases," said Joe Truitt, Chief Executive Officer of iECURE. "Following our recent RMAT designation, we appreciate the opportunity to work closely with the FDA through this highly selective program as we advance ECUR-506 and continue building our manufacturing and regulatory capabilities. These additional CMC interactions will help support development activities aligned with the accelerated clinical timeline, with the goal of bringing ECUR-506 to infants affected by neonatal-onset OTC deficiency as quickly as possible."
ECUR-506 is currently being evaluated in the ongoing OTC-HOPE clinical trial in male infants with neonatal-onset OTC deficiency and has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA. These regulatory designations are supported by encouraging clinical data to date from the study, including a complete clinical response observed in the first infant treated with ECUR-506. The company expects to release additional data from the OTC-HOPE clinical trial in the first half of 2026.
About the OTC-HOPE Study
The OTC-HOPE study is a first-in-human clinical trial evaluating ECUR-506 in male infants with genetically confirmed neonatal-onset OTC deficiency. The trial is enrolling eligible male infants up to seven months of age at screening who are diagnosed with severe neonatal-onset OTC deficiency and meet study entry criteria. The primary objective is to assess the safety, tolerability and efficacy of intravenous administration of a single dose of ECUR-506. The study will also assess the pharmacokinetics of ECUR-506 administration and the potential effects of ECUR-506 on clinical outcome measures, disease-specific biologic markers, developmental milestones and quality of life. The main study will occur in a series of stages over an up to 10-month period, including screening, stabilization, dosing eligibility, study drug administration, and six-month follow-up. Upon completion, participants transition to the 14.5 year long term follow up study (ECUR-LTFU). For more information, visit https://OTC-HOPE.com.
About ECUR-506
ECUR-506 is an investigational in vivo targeted gene insertion therapy designed to restore OTC enzyme activity by inserting a functional copy of the OTC gene into the well-characterized PCSK9 gene locus in liver cells. The therapy utilizes two adeno-associated virus (AAV) vectors using the same capsid, each carrying a distinct payload. One vector contains an ARCUS® nuclease designed to create an insertion site within the PCSK9 locus, while the second vector delivers a functional OTC gene for targeted insertion. iECURE has licensed the ARCUS® nuclease for ECUR-506 from Precision BioSciences (NASDAQ:DTIL).1
About iECURE
iECURE is a clinical-stage genome editing company focused on developing therapies that utilize variant-agnostic in vivo targeted gene insertion to address severe genetic diseases with significant unmet need. The company's approach is designed to restore the function of a dysfunctional gene by inserting a functional copy into a patient's genome, enabling durable gene expression and the potential for long-term therapeutic benefit. iECURE is advancing a pipeline of investigational therapies targeting inherited neurometabolic disorders, a group of rare genetic diseases that can lead to severe metabolic and neurological complications, including ornithine transcarbamylase (OTC) deficiency, citrullinemia type 1 (CTLN1), and phenylketonuria (PKU). For more information, visit https://iecure.com and follow on LinkedIn.
About Precision BioSciences & ARCUS®
Precision BioSciences, Inc. is a clinical stage gene editing company dedicated to improving life (NASDAQ:DTIL) with its novel and proprietary ARCUS® genome editing platform that is designed to differ from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics may enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, Precision's pipeline is comprised of in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases, such as chronic hepatitis B where no adequate treatments exist. For more information about Precision BioSciences, visit www.precisionbiosciences.com.
[1] iECURE has licensed the ARCUS® nuclease from Precision BioSciences for four gene insertion programs including OTC, CTLN1 and PKU.
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