iECURE Reports Positive Results from OTC-HOPE Trial
Written by Emily J. Thompson, Senior Investment Analyst
Updated: May 18 2026
0mins
iECURE announced positive results from a preliminary analysis of the completed low-dose cohort of ECUR-506 from the ongoing OTC-HOPE clinical trial in neonatal-onset ornithine transcarbamylase, OTC, deficiency. Data were presented at the Society for Inherited Metabolic Disorders, SIMD, 2026 Annual Meeting. The first infant treated with ECUR-506 demonstrated a durable clinical response, with sustained discontinuation of standard-of-care therapies and no hyperammonemic events through 18 months post dose, as of February 11, 2026. In addition, participants treated with the low dose of ECUR-506 experienced statistically significant reductions in the annualized rates of HAEs and hyperammonemic crises, with 57% and 65% reductions, respectively, in pre-dose to post-dose rates of events per person-year, as of April 20, 2026.
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Analyst Views on DTIL
Wall Street analysts forecast DTIL stock price to rise
2 Analyst Rating
2 Buy
0 Hold
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Moderate Buy
Current: 7.150
Low
30.00
Averages
45.00
High
60.00
Current: 7.150
Low
30.00
Averages
45.00
High
60.00
About DTIL
Precision BioSciences, Inc. is a clinical-stage gene editing company dedicated to improving life (DTIL) with its proprietary ARCUS genome editing platform. Using ARCUS, the Company’s pipeline is comprised of in vivo gene editing candidates designed to deliver cures for a range of genetic and infectious diseases. The ARCUS platform is used to develop in vivo gene editing therapies for gene edits, including gene insertion, elimination, and excision. The Company's in vivo gene editing pipeline programs include PBGENE-HBV, PBGENE-3243, PBGENE-DMD, PBGENE-LIVER, PBGENE-CNS, PBGENE-NVS and iECURE-OTC. PBGENE-HBV is its lead in vivo gene editing program designed to eliminate cccDNA, the key source of replicating hepatitis B virus (HBV) and inactivating integrated HBV DNA in hepatocytes. Its PBGENE-3243 program is a first of its kind potential treatment for m.3243 associated mitochondrial disease. The Company's iECURE-OTC program is developed in partnership with iECURE, Inc.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
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See More
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See More
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- Platform Advantages: PBGENE-HBV is the world's first gene editing program targeting the root cause of chronic Hepatitis B, cccDNA, aiming for a viral cure; if successful, it will establish Precision BioSciences as a leader in gene therapy, driving long-term growth.
See More
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- Crisis Rate Reduction: Following ECUR-506 treatment, the annualized rate of HACs decreased by approximately 52%, from about 3.12 to 1.49 events per year, providing better survival and developmental opportunities for patients and underscoring the clinical significance of the new therapy.
- Good Safety Profile: ECUR-506 was generally well tolerated across all dose cohorts, with no unexpected safety events observed, aligning with expected safety characteristics, suggesting optimistic prospects for its application in high-risk populations.
- Clinical Trial Progress: Preliminary results from the OTC-HOPE trial support continued evaluation of ECUR-506's efficacy, with iECURE planning to further validate its effectiveness in upcoming pivotal trials, offering hope for infants affected by OTC deficiency.
See More
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- Revenue Growth: The company achieved Q1 revenue of $10.84M, a significant increase from $29K year-over-year, beating market expectations by $6.79M, demonstrating substantial progress in the commercialization of its gene editing technologies.
- Cash Reserves: As of March 31, 2026, Precision BioSciences holds a cash balance of $125.8M, including cash, cash equivalents, and restricted cash, providing financial stability for future clinical stage gene editing projects.
- Project Milestones: The company anticipates utilizing its cash reserves to support data milestones for its two wholly owned clinical stage gene editing programs, PBGENE-HBV and PBGENE-DMD, through 2028, reflecting the sustainability of its long-term strategic vision.
See More
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- Historical Performance Review: Over the past year, Precision BioSciences has exceeded EPS estimates 75% of the time, demonstrating relative stability in earnings forecasts, which may bolster investor confidence ahead of the upcoming report.
- Market Reaction Anticipation: As the earnings report approaches, investors will closely monitor the company's performance, particularly in the current economic climate, where any results exceeding expectations could positively impact the stock price.
See More
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- Trial Design and Objectives: The FUNCTION-DMD study aims to enroll ambulatory patients aged 2-7 with mutations between exons 45 and 55, representing approximately 60% of boys with DMD, to evaluate safety, tolerability, and efficacy, potentially providing durable muscle function improvements.
- Multidisciplinary Care Center: Arkansas Children's Hospital, recognized as a PPMD-certified Duchenne Care Center, delivers specialized multidisciplinary care, ensuring high standards in clinical services while providing comprehensive support for patients participating in the FUNCTION-DMD study.
- FDA Special Designations: PBGENE-DMD has received Orphan Drug Designation and Fast Track designation from the FDA, highlighting its potential in treating DMD and likely leading to significant functional improvements for patients, thereby enhancing Precision BioSciences' leadership in the gene editing sector.
See More
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- ARCUS Platform Advantages: Precision BioSciences' ARCUS gene editing platform, with its unique cutting mechanism and smaller structure, is dedicated to developing gene editing therapies for high unmet need diseases, showcasing significant market potential.
- Diverse Treatment Approaches: The platform supports various gene editing strategies, including gene elimination, defective gene excision, and gene insertion, aimed at providing lasting cures for a broad range of genetic and infectious diseases, further solidifying the company's position in the industry.
See More
