iECURE Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ECUR-506 for Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency

Key Terms

Regenerative Medicine Advanced Therapy (RMAT) regulatory

A Regenerative Medicine Advanced Therapy (RMAT) is a U.S. regulatory designation for cell, gene and tissue-based treatments addressing serious or life-threatening conditions that shows early evidence of potential benefit. Think of it as a VIP lane with extra access to the regulator — more interaction, guidance and faster review — which can shorten development time and lower costs, making a program more valuable to investors, though it does not guarantee approval.

Biologics License Application (BLA) regulatory

A biologics license application (BLA) is a formal request to a government agency seeking approval to sell a biological medicine, such as vaccines or gene therapies, in the market. It is similar to a detailed report that proves the product is safe, effective, and manufactured properly. For investors, a BLA signifies a critical step toward commercial availability, often impacting a company's valuation and market prospects.

Innovation Passport regulatory

An innovation passport is a regulatory designation that identifies a promising new medical product and sets out a tailored, accelerated pathway for its development and review. Like a VIP pass that lets you skip slow lines, it gives developers earlier regulatory feedback and more flexible testing options, which can cut time and cost and reduce risk — factors investors watch because they can speed revenue and raise a product’s commercial value.

Innovative Licensing and Access Pathway (ILAP) regulatory

An innovative licensing and access pathway is a regulatory route that lets promising new medicines or treatments reach patients faster by offering earlier market authorization or special access conditions while additional data or pricing agreements are completed. For investors, it matters because the pathway can accelerate a product’s revenue start and reduce launch uncertainty—like giving a company a fast lane to sell—but it may also carry extra obligations, milestone risks, and negotiation over price and coverage.

orphan drug regulatory

A drug designated for an orphan disease is a medicine developed to treat a rare condition that affects only a small number of people. Regulators often give these drugs special incentives—such as reduced costs, faster review, and temporary exclusive selling rights—to encourage development, which matters to investors because those incentives can make a small market financially viable and reduce competition, much like a temporary patent on a niche product.

Rare Pediatric Disease regulatory

A rare pediatric disease is a serious medical condition that primarily affects children and occurs so infrequently that only a small number of patients exist. Investors care because treatments for such conditions often get special regulatory incentives—think of government fast lanes and rewards for developers—making smaller markets potentially profitable due to pricing power, shorter development timelines, and reduced competition, much like a niche product that receives government-backed advantages.

rolling review regulatory

A rolling review is a regulatory process where health authorities examine data on a drug or vaccine as it becomes available instead of waiting for a complete file at the end. For investors, this can speed up the timeline to approval and reduce uncertainty because regulators assess progress in real time—think of reading and approving chapters of a book as they’re finished rather than waiting for the whole manuscript, which can bring forward potential market access and revenue.

priority review regulatory

Priority review is a regulatory fast-track that shortens the time an agency spends evaluating a drug, vaccine or medical device application so a decision comes sooner than normal. For investors, it matters because a faster review is like an express lane to market: it can speed revenue potential and reduce regulatory uncertainty, but it does not guarantee approval and still requires the product to meet safety and effectiveness standards.

Reached alignment with FDA on endpoints, comparators and sample size for the ongoing OTC-HOPE study which could support Biologics License Application (BLA)

Awarded Innovation Passport under the United Kingdom’s relaunched Innovative Licensing and Access Pathway (ILAP)

PHILADELPHIA--(BUSINESS WIRE)-- iECURE, Inc., a genome editing company focused on the development of variant-agnostic in vivo targeted gene insertion therapies for the treatment of liver disorders with significant unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ECUR-506, the company’s investigational in vivo targeted gene insertion therapy for the treatment of neonatal onset ornithine transcarbamylase (OTC) deficiency. RMAT designation will provide the added benefit of intensive FDA guidance and expedited review throughout the program’s development.

Separately, the company met and aligned with the FDA on the primary and key secondary efficacy endpoints, comparators, and study size for the ongoing OTC-HOPE study that could support a Biologics License Application (BLA) for ECUR-506 in the treatment of neonatal onset OTC deficiency.

In addition, ECUR-506 has been awarded an Innovation Passport under the United Kingdom’s Innovative Licensing and Access Pathway (ILAP). ECUR-506 is one of only three investigational medicinal products selected under the UK’s relaunched ILAP, all of which are focused on rare diseases, underscoring the potential of ECUR-506 to address significant unmet medical need.

“RMAT designation represents an important validation of ECUR-506 and the clinical data generated to date in neonatal onset OTC deficiency,” said Joe Truitt, CEO of iECURE. “Building on our alignment with the FDA on the OTC-HOPE study design, this milestone provides a clear and actionable regulatory path forward. Together with the Innovation Passport granted under the UK’s ILAP, these advancements reinforce our commitment to moving as efficiently as possible toward delivering a potentially durable therapeutic option for infants and families affected by this devastating disease.”

“Importantly, data generated in the neonatal onset OTC deficiency program may also inform the broader development of our targeted gene insertion platform as a plausible mechanism for other indications in our pipeline,” Truitt added.

The RMAT program was established as part of the 21st Century Cures Act to expedite the development and review of regenerative medicine therapies for serious or life-threatening diseases where early clinical evidence indicates the potential to address an unmet medical need. RMAT designation offers benefits similar to the FDA's Breakthrough Therapy and the Fast Track programs, including enhanced and frequent interactions with the FDA throughout development, and eligibility for expedited review mechanisms such as rolling and priority review. In addition to RMAT designation, ECUR-506 has previously received Rare Pediatric Disease and Orphan Drug designations from the FDA for the treatment of neonatal onset OTC deficiency.

The ILAP was launched by the Medicines & Healthcare products Regulatory Agency (MHRA) in 2021 and subsequently relaunched to further accelerate development and access to promising medicines. The relaunched ILAP is the only end-to-end access pathway globally to integrate early, coordinated engagement across regulatory authorities, health technology assessment bodies, and healthcare delivery partners. ILAP brings together the MHRA, the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC), the All Wales Therapeutics and Toxicology Centre (AWTTC), and the NHS, with the shared goal of reducing development timelines and enabling earlier patient access to innovative medicines in the UK.

iECURE’s receipt of these regulatory designations is supported by encouraging clinical data from the ongoing OTC-HOPE clinical study, including a complete clinical response observed in the first infant treated with ECUR-506.

About the OTC-HOPE Study

The OTC-HOPE study is a first-in-human clinical trial of ECUR-506 in male infants with genetically confirmed neonatal onset OTC deficiency and has been cleared to evaluate ascending dose levels of ECUR-506, if necessary. The study is enrolling eligible males 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 and tolerability of intravenous administration of a single dose of ECUR-506. The study will also assess the pharmacokinetics and efficacy 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 a 10-month period, including screening, stabilization, dosing eligibility, study drug administration, and six-month follow-up. Upon completion of the OTC-HOPE study, 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

iECURE’s targeted gene insertion approach for its initial programs, including OTC deficiency, employs the delivery of two adeno-associated virus (AAV) vectors using the same capsid, each carrying a distinct payload. ECUR-506 consists of an ARCUS^® nuclease vector designed to create an insertion site within the well-characterized PCSK9 gene locus, and a donor vector carrying a functional copy of the 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 for the treatment of liver disorders with significant unmet need. We believe our approach has the potential to restore the function of a dysfunctional gene, regardless of variant, by knocking-in a functional copy of that gene to offer durable gene expression and long-term, potentially curative, therapeutic benefit. Our management team has extensive experience in executing global orphan drug and gene therapy clinical trials and successfully commercializing multiple products. We intend to leverage our team’s core strength in research and development strategy to identify what we believe to be the most suitable target and modality for our product candidates to address particular liver diseases. 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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Investors:

David Garrett

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Media:

Donna Lutz

dlutz@iecure.com

Source: iECURE, Inc.