What iECURE does

iECURE is a clinical-stage biotechnology company developing mutation-agnostic in vivo gene insertion therapies for rare neurometabolic disorders. The company is headquartered in the Philadelphia area (Blue Bell, Pennsylvania) and was founded in 2021 on research from the University of Pennsylvania's Gene Therapy Program led by James M. Wilson, M.D., Ph.D. Its lead candidate, ECUR-506, is an adeno-associated virus-delivered gene insertion therapy that uses the ARCUS nuclease (licensed from Precision BioSciences) to insert a functional OTC gene into the PCSK9 locus in liver cells, targeting neonatal-onset ornithine transcarbamylase (OTC) deficiency. The pipeline also includes earlier-stage programs in citrullinemia type 1 (CTLN1) and phenylketonuria (PKU), with a non-viral lipid nanoparticle delivery platform under development with the University of Pennsylvania to extend applicability to adult indications.

The core technology stack is a dual-modality gene-insertion platform: ECUR-506 uses two AAV vectors with the same capsid, one carrying the ARCUS nuclease and the other carrying the therapeutic gene payload. iECURE has secured four FDA designations for ECUR-506 (Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy) plus European Commission Orphan Designation, and was selected for the FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program ahead of a planned BLA. The ongoing OTC-HOPE Phase 1/2 trial, conducted under INDs/clearances in the United States, United Kingdom, and Australia, has reported a 57% reduction in annualized hyperammonemic events (p=0.018) and a 65% reduction in crisis rate (p=0.011) in the completed low-dose cohort, with 18-month durability and standard-of-care discontinuation demonstrated in the first treated infant.

iECURE is a private, pre-revenue company. It has raised approximately $115 million in equity capital across a $50 million Series A in September 2021 and a $65 million Series A-1 in November 2022, with backers including Novo Holdings A/S, LYFE Capital, Versant Ventures, OrbiMed Advisors, Qatar Investment Authority, and Double Point Ventures. Revenue mechanics upon commercialization would follow ultra-rare disease gene-therapy pricing and reimbursement models, including specialty pharmacy distribution and likely outcomes-based arrangements, but the company has no commercialized product today. Its customer base, as a clinical-stage biotech, consists of clinical-trial sites and patients enrolled in the OTC-HOPE study, with University of Pennsylvania serving as both a research partner and equity holder.

Name

iECURE

Legal name

iECURE, Inc.

Website

https://iecure.com

Company type

Private

Founded year

2021

Operating status

Operating

Headcount range

11–50 employees

Short description

iECURE is a clinical-stage biotechnology company developing variant-agnostic in vivo gene insertion therapies, using ARCUS nuclease technology delivered via AAV, to treat rare neurometabolic disorders such as neonatal-onset OTC deficiency.

Ownership category

akta.pro rank

Product category

Gene Therapy Pharmaceuticals

NAICS

Other Scientific and Technical Consulting Services (54169)

SIC

In Vitro & In Vivo Diagnostic Substances (2835)

akta.pro primary industry

Neurology/CNS Gene Therapies (HLAAACAH)

Keywords

• Gene editing therapies
• Rare disease therapeutics
• Neurometabolic disorder treatments
• In vivo gene insertion
• Clinical-stage biotechnology

Headquarters

HQ city

Blue Bell

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Supply Chain, Infrastructure

Revenue model

1. Gene Therapy Commercialization: iECURE is a clinical-stage company with no current revenue. Upon regulatory approval, the company plans to commercialize its gene editing therapies for rare neurometabolic disorders. As a company developing treatments for ultra-rare diseases, pricing and reimbursement will likely follow models typical for cell and gene therapies, potentially including outcomes-based arrangements.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels5 records

Core offering

iECURE is a clinical-stage biotechnology company that develops investigational in vivo gene insertion therapies to permanently correct the underlying genetic cause of rare neurometabolic disorders. Its lead candidate, ECUR-506, uses ARCUS nuclease technology delivered via AAV to insert a functional OTC gene into the PCSK9 locus of liver cells, restoring enzyme activity in patients with neonatal-onset ornithine transcarbamylase (OTC) deficiency. The pipeline additionally targets citrullinemia type 1 (CTLN1) and phenylketonuria (PKU).

Product overview

iECURE is a clinical-stage genome editing company developing variant-agnostic in vivo targeted gene insertion therapies for severe inherited neurometabolic disorders. The company's core technology platform utilizes ARCUS nuclease technology (licensed from Precision BioSciences) delivered via AAV vectors to enable permanent insertion of functional genes into specific genomic loci in liver cells. The lead product candidate is ECUR-506 (also referred to as GTP-506), an investigational therapy for neonatal-onset ornithine transcarbamylase (OTC) deficiency, currently in Phase 1/2 clinical trials. The pipeline also includes programs for citrullinemia type 1 (CTLN1) and phenylketonuria (PKU). The company was founded based on research from the University of Pennsylvania's Gene Therapy Program under James M. Wilson, M.D., Ph.D.

Differentiator

Problem solved

Functional benefit

Products and services

• ECUR-506 (formerly GTP-506) Investigational gene editing therapy that uses ARCUS nuclease technology delivered via dual AAV vectors to insert a functional copy of the OTC gene into the PCSK9 locus of liver cells, intended for the treatment of neonatal-onset ornithine transcarbamylase (OTC) deficiency, an ultra-rare urea cycle disorder.
• CTLN1 Gene Editing Program

Quantifiable outcome

• 57% reduction in annualized hyperammonemic event rate (p=0.018) and 65% reduction in annualized hyperammonemic crisis rate (p=0.011) in low-dose cohort (n=3)
• +3 more outcomes

Named customers2 records

Segments2 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Four partnerships are on record, tiered minor and core.

• Center for Breakthrough Medicines (CBM)minorImplementation/ SI/ Consulting Partner · 25 May 2022iECURE entered an agreement with Center for Breakthrough Medicines (CBM) to supply materials for future clinical programs, supporting manufacturing and supply chain needs for clinical development.
• University of Pennsylvania (Dr. Michael Mitchell's Lab)coreStrategic or Co-development Partner · 16 December 2021iECURE entered an exclusive agreement with the University of Pennsylvania to develop next-generation lipid nanoparticles (LNPs) for liver gene editing applications from Dr. Michael Mitchell's laboratory. This collaboration enables non-viral delivery of gene editing payloads beyond AAV for adult indications or alternative gene editing technologies. The agreement expanded pipeline programs from 13 to 15.
• Precision BioSciencescoreOEM/ Whitelabel/ Licensing PartneriECURE has licensed the ARCUS® nuclease technology from Precision BioSciences for four gene insertion programs including OTC deficiency (ECUR-506), citrullinemia type 1 (CTLN1), and phenylketonuria (PKU). The ARCUS platform is a novel genome editing technology designed to differ from other technologies in the way it cuts, its smaller size, and simpler structure.
• University of Pennsylvania Gene Therapy ProgramcoreStrategic or Co-development PartneriECURE advances its pipeline in close partnership with the University of Pennsylvania's Gene Therapy Program (GTP), directed by James M. Wilson, M.D., Ph.D. The company was founded on pioneering academic research in genetic medicine led by Dr. Wilson, including early work in rare liver disorders such as OTC deficiency. Preclinical efforts for early R&D are led by the GTP team.

Scale indicators6 records

Recent moves7 records

Expansion highlights5 records

Direct peers

• CRISPR Therapeutics: Clinical-stage gene editing company developing CRISPR/Cas9-based in vivo and ex vivo therapies. Directly comparable to iECURE as a platform-level gene editing competitor pursuing liver and other indications with validated editing technologies.
• Intellia Therapeutics: In vivo and ex vivo CRISPR/Cas9 gene editing company with active programs in liver-directed indications. Closely comparable to iECURE in modality (in vivo gene editing), target organ (liver), and clinical-stage gene editing focus.
• Beam Therapeutics: Clinical-stage base editing company developing in vivo liver-directed therapies including for rare metabolic disorders. Directly comparable to iECURE as an in vivo gene editing competitor targeting liver indications with next-generation editing chemistries.
• Verve Therapeutics: In vivo base editing company focused on cardiovascular disease, acquired by Eli Lilly. Highly relevant comparable — iECURE board member Burt Adelman co-founded Verve — illustrating the strategic value and exit pathway for in vivo editing platforms.
• Editas Medicine: Clinical-stage CRISPR-based gene editing company with in vivo and ex vivo programs. Comparable to iECURE as a direct gene editing modality competitor advancing multiple programs through clinical development.
• Homology Medicines: AAV-based gene therapy company that developed a Phase 1/2 PKU program led by iECURE's current CMO Gabriel Cohn. Direct comparable on indication overlap (PKU), modality (AAV gene therapy), and clinical-stage status; relevant precedent for competitive positioning.
• Passage Bio: Clinical-stage AAV gene therapy company for rare CNS disorders, founded with Versant Ventures involvement (same lead investor as iECURE). Comparable as a clinical-stage rare disease AAV gene therapy company with shared investor base and similar development-stage risks.

Emerging players

• Precision BioSciences: Developer of ARCUS nuclease gene editing platform and iECURE's core technology licensor. Comparable as a peer editing platform company, with a strategic supply/licensing relationship that ties the two companies' scientific and commercial trajectories together.

Broad incumbents

• uniQure: Established commercial-stage AAV gene therapy company with approved products. Comparable as a liver-directed AAV gene therapy player; iECURE COO Paul Firuta previously held senior commercial leadership there, providing relevant operational and commercial benchmarking.
• Sangamo Therapeutics: Pioneer in genome editing (zinc finger nucleases) with clinical programs across multiple indications. Comparable as a broader incumbent in the gene editing space pursuing in vivo editing for rare metabolic diseases, though using a different nuclease platform than iECURE.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/iecure

Compliance5 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles12 records

Funding overview

Funding rounds2 records

Investors4 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about iECURE