{"success":true,"data":{"pressRelease":{"id":"129248","rtpr_id":"nBw2G3VGka","ticker":"DTIL","exchange":"NASDAQ","all_tickers":["DTIL"],"title":"iECURE Presents Updated Clinical Data from OTC-HOPE Trial of ECUR-506 in Neonatal-Onset Ornithine Transcarbamylase Deficiency at SSIEM 2026","author":"Business Wire","published_at":"2026-08-27T12:00:00.778Z","article_body":"iECURE Presents Updated Clinical Data from OTC-HOPE Trial of ECUR-506 in\nNeonatal-Onset Ornithine Transcarbamylase Deficiency at SSIEM 2026\n\n\n * Phase 1/2 dosing completed across all dose cohorts; participant evaluations\nongoing\n\n * Preliminary safety observations show ECUR-506 was generally well tolerated,\nwith no unexpected treatment-related safety events observed\n\n * Updated low-dose cohort data demonstrate a 74% reduction in annualized\nhyperammonemic crises (HACs), the most serious and life-threatening\nmanifestation of the condition, following ECUR-506 administration\n\n * Low-dose cohort data provide evidence of targeted hepatic editing and improved\nmetabolic control\n\niECURE, Inc., a clinical-stage genome editing company developing\nvariant-agnostic, in vivo targeted gene insertion therapies for severe\ninherited neurometabolic disorders, today announced encouraging updated\nclinical data from the ongoing OTC-HOPE clinical trial evaluating ECUR-506 for\nneonatal-onset ornithine transcarbamylase (OTC) deficiency. The data are being\npresented today in an oral platform presentation at the Annual Symposium of\nthe Society for the Study of Inborn Errors of Metabolism (SSIEM) in Helsinki,\nFinland.\n\nUpdated data from the low-dose cohort demonstrated a 57% reduction in the\nannualized rate of hyperammonemic events (HAEs; p=0.02) and a 74% reduction in\nthe annualized rate of hyperammonemic crises (HACs; p=0.01) following ECUR-506\nadministration. HAEs are episodes of elevated ammonia, with more severe\nepisodes known as HACs. HACs are acute medical emergencies characterized by\nelevated ammonia levels with neurologic symptoms and can lead to\nhospitalization, irreversible neurological injury, or death.\n\n“The clinical activity observed in the low-dose cohort is encouraging,\nparticularly given that OTC-HOPE is evaluating some of the youngest and most\nseverely affected patients with OTC deficiency,” said Julien Baruteau, M.D.,\nPh.D., University College London Great Ormond Street Institute of Child Health\nand an investigator in the OTC-HOPE study. “The safety, editing and clinical\ndata generated to date are helping us better understand the relationship\nbetween underlying disease severity and the level of enzyme restoration that\nmay be needed to achieve metabolic correction. These findings support the\ncontinued evaluation of higher ECUR-506 dose levels.”\n\nThe presentation also includes preliminary safety observations from all nine\nparticipants treated across the low-, intermediate- and high-dose cohorts, as\nwell as analyses of editing efficiency and metabolic control from the low-dose\ncohort.\n\n“Completing Phase 1/2 dosing across all three dose cohorts represents an\nimportant milestone for the OTC-HOPE program and provides us with a growing\nbody of clinical experience with ECUR-506,” said Gabe Cohn, M.D., Chief\nMedical Officer of iECURE. “With nine participants now treated, we are\nencouraged by the safety profile observed to date and the clinical and editing\ndata emerging from the study. We look forward to continuing to build on these\nfindings as we advance the clinical development of ECUR-506.”\n\nUpdated OTC-HOPE Data Presented at SSIEM\n\nSafety\n\n\n * Nine participants have received ECUR-506 across three dose cohorts, with three\nparticipants each receiving the low dose (1.3 x 10¹³ GC/kg), intermediate\ndose (2.4 x 10¹³ GC/kg) or high dose (4.0 x 10¹³ GC/kg).\n\n * As of July 28, 2026, ECUR-506 was generally well tolerated across all three\ndose cohorts, with no unexpected treatment-related safety events reported.\n\n * There were no cases of thrombotic microangiopathy or infusion reactions.\n\n * Asymptomatic, non-dose-dependent, transient Grade 2–3 transaminitis resolved\nwith reactive immunosuppression in seven of eight participants; one\nparticipant received prophylactic corticosteroids following ECUR-506\nadministration per protocol.\n\n * One participant experienced hypoxemic respiratory failure resulting in death,\nwhich was determined to be unrelated to ECUR-506 and attributable to\nunderlying OTC deficiency disease progression and resulting complications.\n\nTargeted Hepatic Editing\n\n\n * Twenty-four-week liver biopsies were performed in two of the three\nparticipants in the low-dose cohort; the family of the third participant did\nnot consent to the biopsy.\n\n * Analyses demonstrated evidence of targeted hepatic editing following ECUR-506\nadministration.\n\n * Reductions in serum PCSK9 levels were observed across the low-dose cohort,\nproviding additional evidence consistent with editing at the targeted PCSK9\nlocus.\n\n * Liver biopsy analyses from the intermediate- and high-dose cohorts are\npending.\n\nClinical Response\n\n\n * In the low-dose cohort, annualized rates of HAE and HAC (the more severe\nsubset of HAEs) were reduced by 57% (p=0.02) and 74% (p=0.01), respectively,\ncompared with pre-treatment rates following ECUR-506 administration. The\nupdated 74% HAC reduction, compared with the previously reported 65%\nreduction, reflects the identification of an additional pre-treatment HAC\nsince the prior data cutoff, included following updated review of the\npre-treatment medical history.\n\n * The three participants demonstrated heterogeneous clinical responses despite\nreceiving the same dose. One participant, whose peak ammonia level at initial\npresentation was 840 µmol/L, achieved a complete clinical response and\ndiscontinued ammonia scavenger therapy and protein restriction. The other two\nparticipants had peak ammonia levels of 1,600 µmol/L or greater at\npresentation and demonstrated improvements in clinical measures consistent\nwith reduced disease burden, with one participant successfully weaned from one\nof two ammonia scavenger medications.\n\n * These observations, together with published natural history data demonstrating\na relationship between OTC enzyme activity, initial ammonia levels and disease\nseverity, support the hypothesis that baseline disease severity may influence\ntreatment response. Patients with more profound underlying OTC deficiency may\nrequire greater restoration of OTC enzyme activity to achieve metabolic\nstability.\n\n“We are pleased with the progress of the OTC-HOPE program and what we have\nlearned through the completion of Phase 1/2 dosing,” said Joe Truitt, Chief\nExecutive Officer of iECURE. “This milestone reflects the tremendous\ncommitment of participating families, investigators and clinical teams, and\nbrings us another step forward in our goal of developing a meaningful new\ntreatment option for infants with neonatal-onset OTC deficiency.”\n\nAbout OTC Deficiency\n\nOrnithine transcarbamylase (OTC) deficiency is a rare, serious genetic\ndisorder caused by a defect in a liver enzyme responsible for removing ammonia\nfrom the bloodstream. As a result, ammonia, a waste product generated when the\nbody breaks down protein, accumulates in the blood (hyperammonemia) to levels\nthat are toxic to the brain.\n\nThe disease is characterized by recurrent and often unpredictable\nhyperammonemic crises (HACs), which can lead to hospitalization, irreversible\nneurological injury, and death. Newborns with neonatal-onset OTC deficiency\ntypically present shortly after birth with symptoms such as lethargy, poor\nfeeding, and vomiting, which can rapidly progress to seizures, coma, and\nlife-threatening complications if not promptly treated.\n\nCurrent management includes a strict protein-restricted diet and ammonia\nscavenger medications, which must be taken multiple times daily and often\nthroughout a patient’s life. While these approaches may help manage ammonia\nlevels, they do not eliminate the risk of metabolic crises or ongoing disease\nburden, and patients remain vulnerable to acute decompensation and long-term\ncomplications.\n\nAbout the OTC-HOPE Study\n\nThe OTC-HOPE study is a first-in-human clinical trial evaluating ECUR-506 in\nmale infants with genetically confirmed neonatal-onset OTC deficiency. The\ntrial is enrolling eligible male infants up to seven months of age at\nscreening who are diagnosed with severe neonatal-onset OTC deficiency and meet\nstudy entry criteria. The primary objective is to assess the safety,\ntolerability and efficacy of intravenous administration of a single dose of\nECUR-506. The study will also assess the pharmacokinetics of ECUR-506\nadministration and the potential effects of ECUR-506 on clinical outcome\nmeasures, disease-specific biologic markers, developmental milestones and\nquality of life. The main study includes screening, stabilization, dosing\neligibility, study drug administration, and six-month follow-up, after which\nparticipants transition to a 14.5 year long term follow-up study (ECUR-LTFU).\nFor more information, visit https://OTC-HOPE.com\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2FOTC-HOPE.com&esheet=54595379&newsitemid=20260827549114&lan=en-US&anchor=https%3A%2F%2FOTC-HOPE.com&index=1&md5=0d9396879ec17826de69a3c936c5e036)\n.\n\nAbout ECUR-506\n\nECUR-506 is an investigational in vivo targeted gene insertion therapy\ndesigned to restore OTC enzyme activity by inserting a functional copy of the\nOTC gene into the well-characterized PCSK9 gene locus in liver cells. The\ntherapy utilizes two adeno-associated virus (AAV) vectors using the same\ncapsid, each carrying a distinct payload. One vector contains an ARCUS®\nnuclease designed to create an insertion site within the PCSK9 locus, while\nthe second vector delivers a functional OTC gene for targeted insertion.\niECURE has licensed the ARCUS® nuclease for ECUR-506 from Precision\nBioSciences (Nasdaq: DTIL).(1)\n\nAbout iECURE\n\niECURE is a clinical-stage genome editing company focused on developing\ntherapies that utilize variant-agnostic in vivo targeted gene insertion to\naddress severe genetic diseases with significant unmet need. The company’s\napproach is designed to restore the function of a missing or dysfunctional\ngene by inserting a functional copy into a patient’s genome, enabling\ndurable gene expression and the potential for long-term therapeutic benefit.\niECURE is advancing a pipeline of investigational therapies targeting\ninherited neurometabolic disorders, a group of rare genetic diseases that can\nlead to severe metabolic and neurological complications, including ornithine\ntranscarbamylase (OTC) deficiency, citrullinemia type 1 (CTLN1), and\nphenylketonuria (PKU). For more information, visit https://iecure.com\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fiecure.com&esheet=54595379&newsitemid=20260827549114&lan=en-US&anchor=https%3A%2F%2Fiecure.com&index=2&md5=2383826e595c021284a4ccb3d765f365)\nand follow on LinkedIn.\n\nAbout Precision BioSciences & ARCUS®\n\nPrecision BioSciences, Inc. is a clinical stage gene editing company dedicated\nto improving life (Nasdaq: DTIL) with its novel and proprietary ARCUS® genome\nediting platform that is designed to differ from other technologies in the way\nit cuts, its smaller size, and its simpler structure. Key capabilities and\ndifferentiating characteristics may enable ARCUS nucleases to drive more\nintended, defined therapeutic outcomes. Using ARCUS, Precision’s pipeline is\ncomprised of in vivo gene editing candidates designed to deliver lasting cures\nfor the broadest range of genetic and infectious diseases, such as chronic\nhepatitis B where no adequate treatments exist. For more information about\nPrecision BioSciences, visit www.precisionbiosciences.com\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=http%3A%2F%2Fwww.precisionbiosciences.com&esheet=54595379&newsitemid=20260827549114&lan=en-US&anchor=www.precisionbiosciences.com&index=3&md5=7c69b519919633993a98291fb02fc538)\n.\n\n[1] iECURE has licensed the ARCUS® nuclease from Precision BioSciences for\nfour gene insertion programs including OTC, CTLN1 and PKU.\n\n\n\nView source version on businesswire.com:\nhttps://www.businesswire.com/news/home/20260827549114/en/\n(https://www.businesswire.com/news/home/20260827549114/en/)\n\nInvestors: \n\nDavid Garrett\n\ndgarrett@iecure.com (mailto:dgarrett@iecure.com)\n\nMedia: \n\nJanine Bogris\n\njanine.bogris@inizioevoke.com (mailto:janine.bogris@inizioevoke.com)\n\n\nCopyright Business Wire 2026","article_body_html":"","raw_payload":{"data":{"id":"nBw2G3VGka","title":"iECURE Presents Updated Clinical Data from OTC-HOPE Trial of ECUR-506 in Neonatal-Onset Ornithine Transcarbamylase Deficiency at SSIEM 2026","author":"Business Wire","ticker":"DTIL","created":"2026-08-27T12:00:00.778Z","tickers":["DTIL"],"exchange":"NASDAQ","article_body":"iECURE Presents Updated Clinical Data from OTC-HOPE Trial of ECUR-506 in\nNeonatal-Onset Ornithine Transcarbamylase Deficiency at SSIEM 2026\n\n\n * Phase 1/2 dosing completed across all dose cohorts; participant evaluations\nongoing\n\n * Preliminary safety observations show ECUR-506 was generally well tolerated,\nwith no unexpected treatment-related safety events observed\n\n * Updated low-dose cohort data demonstrate a 74% reduction in annualized\nhyperammonemic crises (HACs), the most serious and life-threatening\nmanifestation of the condition, following ECUR-506 administration\n\n * Low-dose cohort data provide evidence of targeted hepatic editing and improved\nmetabolic control\n\niECURE, Inc., a clinical-stage genome editing company developing\nvariant-agnostic, in vivo targeted gene insertion therapies for severe\ninherited neurometabolic disorders, today announced encouraging updated\nclinical data from the ongoing OTC-HOPE clinical trial evaluating ECUR-506 for\nneonatal-onset ornithine transcarbamylase (OTC) deficiency. The data are being\npresented today in an oral platform presentation at the Annual Symposium of\nthe Society for the Study of Inborn Errors of Metabolism (SSIEM) in Helsinki,\nFinland.\n\nUpdated data from the low-dose cohort demonstrated a 57% reduction in the\nannualized rate of hyperammonemic events (HAEs; p=0.02) and a 74% reduction in\nthe annualized rate of hyperammonemic crises (HACs; p=0.01) following ECUR-506\nadministration. HAEs are episodes of elevated ammonia, with more severe\nepisodes known as HACs. HACs are acute medical emergencies characterized by\nelevated ammonia levels with neurologic symptoms and can lead to\nhospitalization, irreversible neurological injury, or death.\n\n“The clinical activity observed in the low-dose cohort is encouraging,\nparticularly given that OTC-HOPE is evaluating some of the youngest and most\nseverely affected patients with OTC deficiency,” said Julien Baruteau, M.D.,\nPh.D., University College London Great Ormond Street Institute of Child Health\nand an investigator in the OTC-HOPE study. “The safety, editing and clinical\ndata generated to date are helping us better understand the relationship\nbetween underlying disease severity and the level of enzyme restoration that\nmay be needed to achieve metabolic correction. These findings support the\ncontinued evaluation of higher ECUR-506 dose levels.”\n\nThe presentation also includes preliminary safety observations from all nine\nparticipants treated across the low-, intermediate- and high-dose cohorts, as\nwell as analyses of editing efficiency and metabolic control from the low-dose\ncohort.\n\n“Completing Phase 1/2 dosing across all three dose cohorts represents an\nimportant milestone for the OTC-HOPE program and provides us with a growing\nbody of clinical experience with ECUR-506,” said Gabe Cohn, M.D., Chief\nMedical Officer of iECURE. “With nine participants now treated, we are\nencouraged by the safety profile observed to date and the clinical and editing\ndata emerging from the study. We look forward to continuing to build on these\nfindings as we advance the clinical development of ECUR-506.”\n\nUpdated OTC-HOPE Data Presented at SSIEM\n\nSafety\n\n\n * Nine participants have received ECUR-506 across three dose cohorts, with three\nparticipants each receiving the low dose (1.3 x 10¹³ GC/kg), intermediate\ndose (2.4 x 10¹³ GC/kg) or high dose (4.0 x 10¹³ GC/kg).\n\n * As of July 28, 2026, ECUR-506 was generally well tolerated across all three\ndose cohorts, with no unexpected treatment-related safety events reported.\n\n * There were no cases of thrombotic microangiopathy or infusion reactions.\n\n * Asymptomatic, non-dose-dependent, transient Grade 2–3 transaminitis resolved\nwith reactive immunosuppression in seven of eight participants; one\nparticipant received prophylactic corticosteroids following ECUR-506\nadministration per protocol.\n\n * One participant experienced hypoxemic respiratory failure resulting in death,\nwhich was determined to be unrelated to ECUR-506 and attributable to\nunderlying OTC deficiency disease progression and resulting complications.\n\nTargeted Hepatic Editing\n\n\n * Twenty-four-week liver biopsies were performed in two of the three\nparticipants in the low-dose cohort; the family of the third participant did\nnot consent to the biopsy.\n\n * Analyses demonstrated evidence of targeted hepatic editing following ECUR-506\nadministration.\n\n * Reductions in serum PCSK9 levels were observed across the low-dose cohort,\nproviding additional evidence consistent with editing at the targeted PCSK9\nlocus.\n\n * Liver biopsy analyses from the intermediate- and high-dose cohorts are\npending.\n\nClinical Response\n\n\n * In the low-dose cohort, annualized rates of HAE and HAC (the more severe\nsubset of HAEs) were reduced by 57% (p=0.02) and 74% (p=0.01), respectively,\ncompared with pre-treatment rates following ECUR-506 administration. The\nupdated 74% HAC reduction, compared with the previously reported 65%\nreduction, reflects the identification of an additional pre-treatment HAC\nsince the prior data cutoff, included following updated review of the\npre-treatment medical history.\n\n * The three participants demonstrated heterogeneous clinical responses despite\nreceiving the same dose. One participant, whose peak ammonia level at initial\npresentation was 840 µmol/L, achieved a complete clinical response and\ndiscontinued ammonia scavenger therapy and protein restriction. The other two\nparticipants had peak ammonia levels of 1,600 µmol/L or greater at\npresentation and demonstrated improvements in clinical measures consistent\nwith reduced disease burden, with one participant successfully weaned from one\nof two ammonia scavenger medications.\n\n * These observations, together with published natural history data demonstrating\na relationship between OTC enzyme activity, initial ammonia levels and disease\nseverity, support the hypothesis that baseline disease severity may influence\ntreatment response. Patients with more profound underlying OTC deficiency may\nrequire greater restoration of OTC enzyme activity to achieve metabolic\nstability.\n\n“We are pleased with the progress of the OTC-HOPE program and what we have\nlearned through the completion of Phase 1/2 dosing,” said Joe Truitt, Chief\nExecutive Officer of iECURE. “This milestone reflects the tremendous\ncommitment of participating families, investigators and clinical teams, and\nbrings us another step forward in our goal of developing a meaningful new\ntreatment option for infants with neonatal-onset OTC deficiency.”\n\nAbout OTC Deficiency\n\nOrnithine transcarbamylase (OTC) deficiency is a rare, serious genetic\ndisorder caused by a defect in a liver enzyme responsible for removing ammonia\nfrom the bloodstream. As a result, ammonia, a waste product generated when the\nbody breaks down protein, accumulates in the blood (hyperammonemia) to levels\nthat are toxic to the brain.\n\nThe disease is characterized by recurrent and often unpredictable\nhyperammonemic crises (HACs), which can lead to hospitalization, irreversible\nneurological injury, and death. Newborns with neonatal-onset OTC deficiency\ntypically present shortly after birth with symptoms such as lethargy, poor\nfeeding, and vomiting, which can rapidly progress to seizures, coma, and\nlife-threatening complications if not promptly treated.\n\nCurrent management includes a strict protein-restricted diet and ammonia\nscavenger medications, which must be taken multiple times daily and often\nthroughout a patient’s life. While these approaches may help manage ammonia\nlevels, they do not eliminate the risk of metabolic crises or ongoing disease\nburden, and patients remain vulnerable to acute decompensation and long-term\ncomplications.\n\nAbout the OTC-HOPE Study\n\nThe OTC-HOPE study is a first-in-human clinical trial evaluating ECUR-506 in\nmale infants with genetically confirmed neonatal-onset OTC deficiency. The\ntrial is enrolling eligible male infants up to seven months of age at\nscreening who are diagnosed with severe neonatal-onset OTC deficiency and meet\nstudy entry criteria. The primary objective is to assess the safety,\ntolerability and efficacy of intravenous administration of a single dose of\nECUR-506. The study will also assess the pharmacokinetics of ECUR-506\nadministration and the potential effects of ECUR-506 on clinical outcome\nmeasures, disease-specific biologic markers, developmental milestones and\nquality of life. The main study includes screening, stabilization, dosing\neligibility, study drug administration, and six-month follow-up, after which\nparticipants transition to a 14.5 year long term follow-up study (ECUR-LTFU).\nFor more information, visit https://OTC-HOPE.com\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2FOTC-HOPE.com&esheet=54595379&newsitemid=20260827549114&lan=en-US&anchor=https%3A%2F%2FOTC-HOPE.com&index=1&md5=0d9396879ec17826de69a3c936c5e036)\n.\n\nAbout ECUR-506\n\nECUR-506 is an investigational in vivo targeted gene insertion therapy\ndesigned to restore OTC enzyme activity by inserting a functional copy of the\nOTC gene into the well-characterized PCSK9 gene locus in liver cells. The\ntherapy utilizes two adeno-associated virus (AAV) vectors using the same\ncapsid, each carrying a distinct payload. One vector contains an ARCUS®\nnuclease designed to create an insertion site within the PCSK9 locus, while\nthe second vector delivers a functional OTC gene for targeted insertion.\niECURE has licensed the ARCUS® nuclease for ECUR-506 from Precision\nBioSciences (Nasdaq: DTIL).(1)\n\nAbout iECURE\n\niECURE is a clinical-stage genome editing company focused on developing\ntherapies that utilize variant-agnostic in vivo targeted gene insertion to\naddress severe genetic diseases with significant unmet need. The company’s\napproach is designed to restore the function of a missing or dysfunctional\ngene by inserting a functional copy into a patient’s genome, enabling\ndurable gene expression and the potential for long-term therapeutic benefit.\niECURE is advancing a pipeline of investigational therapies targeting\ninherited neurometabolic disorders, a group of rare genetic diseases that can\nlead to severe metabolic and neurological complications, including ornithine\ntranscarbamylase (OTC) deficiency, citrullinemia type 1 (CTLN1), and\nphenylketonuria (PKU). For more information, visit https://iecure.com\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fiecure.com&esheet=54595379&newsitemid=20260827549114&lan=en-US&anchor=https%3A%2F%2Fiecure.com&index=2&md5=2383826e595c021284a4ccb3d765f365)\nand follow on LinkedIn.\n\nAbout Precision BioSciences & ARCUS®\n\nPrecision BioSciences, Inc. is a clinical stage gene editing company dedicated\nto improving life (Nasdaq: DTIL) with its novel and proprietary ARCUS® genome\nediting platform that is designed to differ from other technologies in the way\nit cuts, its smaller size, and its simpler structure. Key capabilities and\ndifferentiating characteristics may enable ARCUS nucleases to drive more\nintended, defined therapeutic outcomes. Using ARCUS, Precision’s pipeline is\ncomprised of in vivo gene editing candidates designed to deliver lasting cures\nfor the broadest range of genetic and infectious diseases, such as chronic\nhepatitis B where no adequate treatments exist. For more information about\nPrecision BioSciences, visit www.precisionbiosciences.com\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=http%3A%2F%2Fwww.precisionbiosciences.com&esheet=54595379&newsitemid=20260827549114&lan=en-US&anchor=www.precisionbiosciences.com&index=3&md5=7c69b519919633993a98291fb02fc538)\n.\n\n[1] iECURE has licensed the ARCUS® nuclease from Precision BioSciences for\nfour gene insertion programs including OTC, CTLN1 and PKU.\n\n\n\nView source version on businesswire.com:\nhttps://www.businesswire.com/news/home/20260827549114/en/\n(https://www.businesswire.com/news/home/20260827549114/en/)\n\nInvestors: \n\nDavid Garrett\n\ndgarrett@iecure.com (mailto:dgarrett@iecure.com)\n\nMedia: \n\nJanine Bogris\n\njanine.bogris@inizioevoke.com (mailto:janine.bogris@inizioevoke.com)\n\n\nCopyright Business Wire 2026"},"type":"article","timestamp":"2026-08-27T12:00:01.11720776Z","server_sent_at_ms":1787832001117},"received_at":"2026-08-27T12:00:01.168Z","source_url":"https://www.businesswire.com/news/home/20260827549114/en/"},"analysis":{"id":"118161","press_release_id":"129248","analysis_json":{"industry":{"label":"Biotechnology","sector":"Health Care"},"redFlags":["One participant death determined to be unrelated to ECUR-506 but attributed to underlying disease progression","Transient Grade 2-3 transaminitis observed in seven of eight participants"],"eventType":"clinical_trial","narrative":"Partner iECURE presented updated Phase 1/2 data for ECUR-506, a therapy utilizing Precision BioSciences' ARCUS nuclease, demonstrating a 74% reduction in life-threatening hyperammonemic crises in neonatal-onset OTC deficiency.\n\nThe drug was generally well tolerated across nine participants in low, intermediate, and high dose cohorts, with no unexpected treatment-related safety events reported.\n\nThese results represent a significant validation milestone for the ARCUS platform, which iECURE has licensed for use in ECUR-506 and other gene insertion programs.","sentiment":"bullish","agentHooks":{"shouldPost":true,"suggestedAngle":"Platform validation: iECURE reports strong ECUR-506 data using Precision BioSciences' ARCUS tech."},"keyFigures":{"drugName":"ECUR-506","phaseOfTrial":"Phase 1/2","customDimensions":{"hac_reduction":"74%","hae_reduction":"57%","low_dose_gc_kg":"1.3 x 10^13","high_dose_gc_kg":"4.0 x 10^13","patients_treated":9,"intermediate_dose_gc_kg":"2.4 x 10^13"}},"quotedText":"Completing Phase 1/2 dosing across all three dose cohorts represents an important milestone for the OTC-HOPE program and provides us with a growing body of clinical experience with ECUR-506","namedEntities":{"people":[{"name":"Julien Baruteau","role":"Investigator, University College London"},{"name":"Gabe Cohn","role":"Chief Medical Officer of iECURE"},{"name":"Joe Truitt","role":"Chief Executive Officer of iECURE"}],"products":["ECUR-506","ARCUS"],"companies":[{"name":"iECURE, Inc.","relationship":"partner"},{"name":"Precision BioSciences","ticker":"DTIL","relationship":"licensor"}],"dollarAmounts":[]},"materialImpact":{"score":3,"reasoning":"Updated data from partner iECURE shows a statistically significant 74% reduction in hyperammonemic crises for ECUR-506, validating the licensed ARCUS nuclease platform. While a partner program, the strong safety and efficacy profile de-risks the underlying technology."},"tickerRelevance":{"others":[],"primary":"DTIL"},"globalImportance":30,"audienceRelevance":25,"eventTypeSecondary":[],"importanceComponents":{"tickerTier":"small-cap","eventGravity":"partner-platform-validation","sectorWeight":"biotech"}},"event_type":"clinical_trial","event_type_secondary":null,"sentiment":"bullish","material_impact_score":3,"narrative":"Partner iECURE presented updated Phase 1/2 data for ECUR-506, a therapy utilizing Precision BioSciences' ARCUS nuclease, demonstrating a 74% reduction in life-threatening hyperammonemic crises in neonatal-onset OTC deficiency.\n\nThe drug was generally well tolerated across nine participants in low, intermediate, and high dose cohorts, with no unexpected treatment-related safety events reported.\n\nThese results represent a significant validation milestone for the ARCUS platform, which iECURE has licensed for use in ECUR-506 and other gene insertion programs.","key_figures":{"drugName":"ECUR-506","phaseOfTrial":"Phase 1/2","customDimensions":{"hac_reduction":"74%","hae_reduction":"57%","low_dose_gc_kg":"1.3 x 10^13","high_dose_gc_kg":"4.0 x 10^13","patients_treated":9,"intermediate_dose_gc_kg":"2.4 x 10^13"}},"named_entities":{"people":[{"name":"Julien Baruteau","role":"Investigator, University College London"},{"name":"Gabe Cohn","role":"Chief Medical Officer of iECURE"},{"name":"Joe Truitt","role":"Chief Executive Officer of iECURE"}],"products":["ECUR-506","ARCUS"],"companies":[{"name":"iECURE, Inc.","relationship":"partner"},{"name":"Precision BioSciences","ticker":"DTIL","relationship":"licensor"}],"dollarAmounts":[]},"model_name":"glm-4.7","prompt_hash":"sha256:727b4b9429a443af","schema_hash":"sha256:05005c02d9cffac9","created_at":"2026-08-27T12:35:44.976Z","global_importance":30,"audience_relevance":25,"importance_components":{"tickerTier":"small-cap","eventGravity":"partner-platform-validation","sectorWeight":"biotech"}},"durationMs":116770,"modelName":"glm-4.7"}}