iECURE Secures FDA RMAT Designation for ECUR-506, Advancing OTC Deficiency Treatment
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Jan 07 2026
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Source: Newsfilter
- Regulatory Milestone: iECURE's ECUR-506 has received FDA's Regenerative Medicine Advanced Therapy (RMAT) designation, which will provide accelerated review and intensive guidance for its development in treating neonatal onset ornithine transcarbamylase deficiency, thereby expediting the clinical development process.
- Clinical Study Alignment: The company has aligned with the FDA on primary and secondary efficacy endpoints, comparators, and sample size for the ongoing OTC-HOPE study, laying the groundwork for a future Biologics License Application (BLA) and showcasing its potential in the rare disease space.
- Innovation Passport: ECUR-506 has also been awarded an Innovation Passport under the UK's Innovative Licensing and Access Pathway (ILAP), making it one of only three selected investigational medicinal products, underscoring its potential to address significant unmet medical needs.
- Clinical Data Support: Encouraging clinical data from the OTC-HOPE study, including a complete clinical response observed in the first treated infant, further validates the efficacy of ECUR-506 and may inform the development of other indications in the pipeline.
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Analyst Views on DTIL
Wall Street analysts forecast DTIL stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for DTIL is 45.00 USD with a low forecast of 30.00 USD and a high forecast of 60.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
2 Analyst Rating
2 Buy
0 Hold
0 Sell
Moderate Buy
Current: 3.960
Low
30.00
Averages
45.00
High
60.00
Current: 3.960
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.
Precision BioSciences Advances PBGENE-HBV and PBGENE-DMD Trials, Data Updates Expected in 2026
- Clinical Trial Progress: Precision BioSciences' PBGENE-HBV is currently undergoing the Phase 1/2a ELIMINATE-B trial, which has shown dose-dependent antiviral activity, with data updates expected at medical conferences in 2026, potentially offering a cure for chronic hepatitis B patients.
- Strong Financial Position: As of December 31, 2025, Precision anticipates having approximately $137 million in cash and cash equivalents, which is expected to fund key data milestones for PBGENE-HBV and PBGENE-DMD, ensuring operational stability through 2028.
- New DMD Treatment Approach: The PBGENE-DMD Phase 1/2 FUNCTION-DMD trial is expected to receive IND clearance in Q1 2026, aiming to provide durable functional muscle improvement for 60% of DMD patients, with initial data anticipated by the end of 2026.
- Collaborative Project Advancements: The ECUR-506 project with iECURE has reached key alignment with the FDA, supporting its potential as a curative treatment for neonatal OTC deficiency, with clinical trial data expected to be released in the first half of 2026, further enhancing Precision's market position.
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iECURE Secures FDA RMAT Designation for ECUR-506, Advancing OTC Deficiency Treatment
- Regulatory Milestone: iECURE's ECUR-506 has received FDA's Regenerative Medicine Advanced Therapy (RMAT) designation, which will provide accelerated review and intensive guidance for its development in treating neonatal onset ornithine transcarbamylase deficiency, thereby expediting the clinical development process.
- Clinical Study Alignment: The company has aligned with the FDA on primary and secondary efficacy endpoints, comparators, and sample size for the ongoing OTC-HOPE study, laying the groundwork for a future Biologics License Application (BLA) and showcasing its potential in the rare disease space.
- Innovation Passport: ECUR-506 has also been awarded an Innovation Passport under the UK's Innovative Licensing and Access Pathway (ILAP), making it one of only three selected investigational medicinal products, underscoring its potential to address significant unmet medical needs.
- Clinical Data Support: Encouraging clinical data from the OTC-HOPE study, including a complete clinical response observed in the first treated infant, further validates the efficacy of ECUR-506 and may inform the development of other indications in the pipeline.
Continue Reading