{"success":true,"data":{"pressRelease":{"id":"8703","rtpr_id":"nGNX9kxW0R","ticker":"NTLA","exchange":"NASDAQ","all_tickers":["NTLA"],"title":"Intellia Therapeutics Initiates Rolling Submission of Biologics License Application to FDA for Lonvoguran Ziclumeran (lonvo-z) as a One-Time Treatment for Hereditary Angioedema","author":"Globe Newswire","published_at":"2026-04-27T11:00:00.370Z","article_body":"CAMBRIDGE, Mass., April 27, 2026 (GLOBE NEWSWIRE) -- Intellia Therapeutics,\nInc.\n(https://www.globenewswire.com/Tracker?data=XSbLXuLM_FJPa5n4RY-9U0ayTGTtFrUuqcdVUivSkYZ09lePzGxCBH591z_fX3RkbTYZMthG784urNz0kYsjReyhOe5s2i6NK9c-r9szGZuj9p3WElIVfhl_4dJw8rGW)\n(Nasdaq: NTLA), a leading biopharmaceutical company focused on revolutionizing\nmedicine leveraging CRISPR gene editing and other core technologies, today\nannounced it has initiated a rolling submission of a biologics license\napplication (BLA) to the U.S. Food and Drug Administration (FDA) seeking\napproval of lonvo-z (formerly known as NTLA-2002) for hereditary angioedema\n(HAE). Designed as a one-time treatment that is administered in an outpatient\nsetting, lonvo-z is an in vivo CRISPR gene editing candidate that is intended\nto inactivate the kallikrein B1 (KLKB1) gene to permanently lower kallikrein\nand bradykinin levels.\n\nIntellia also separately announced positive topline data today from the Phase\n3 HAELO clinical trial of lonvo-z in HAE. The trial met its primary and all\nkey secondary endpoints, demonstrating that a one-time dose of lonvo-z led to\nfreedom from both HAE attacks and the use of ongoing therapy for most patients\nduring the six-month primary observation period.\n\n“If approved, lonvo-z will become the world’s first in vivo CRISPR-based\ngene editing therapy,” said John Leonard, M.D., Intellia President and Chief\nExecutive Officer. “The promising results from HAELO reinforce our\nconviction that lonvo-z could revolutionize how HAE is treated for many\npatients, with the potential to free most of them from both attacks and the\nneed for ongoing therapy with just one dose. We look forward to our continued\nengagement with the FDA as we seek to ease many of the burdens for people\nliving with HAE.”\n\nPursuant to the Regenerative Medicine Advanced Therapy (RMAT) designation that\nthe FDA granted to lonvo-z for the treatment of HAE, a rolling BLA allows\nIntellia to submit portions of the BLA on an ongoing basis and provides the\nFDA with an opportunity to expedite its review. In addition to the RMAT\nprogram, Intellia participated in the FDA’s Chemistry, Manufacturing, and\nControls (CMC) Development and Readiness Pilot. This program allows sponsors\nto discuss their CMC product development strategies and goals with FDA review\nstaff and address their questions. The increased communication is intended to\nhelp sponsors complete CMC activities to expedite their drug development\nprograms to support application submission and earlier patient access.\n\nIntellia anticipates completing its BLA submission in the second half of 2026.\nIf the filing is accepted by the FDA, the agency is expected to determine if\nit will grant a priority review and provide a target action date to complete\nits evaluation. If approved, Intellia plans to launch lonvo-z commercially in\nthe first half of 2027.\n\nAbout Lonvo-z\nBased on Nobel Prize-winning CRISPR/Cas9 technology, lonvo-z has the potential\nto become the first one-time treatment for hereditary angioedema (HAE).\nLonvo-z is an in vivo CRISPR gene editing candidate that is intended to\npermanently lower kallikrein by inactivating the kallikrein B1 (KLKB1) gene\nwith a single dose. Lonvo-z has received five notable regulatory designations:\nOrphan Drug and RMAT Designation by the U.S. Food and Drug Administration\n(FDA), the Innovation Passport by the U.K. Medicines and Healthcare products\nRegulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the\nEuropean Medicines Agency, as well as Orphan Drug Designation (ODD) by the\nEuropean Commission.\n\nAbout Hereditary Angioedema\nHereditary angioedema (HAE) is a rare, genetic disease characterized by\nsevere, recurring and unpredictable inflammatory attacks in various organs and\ntissues of the body, which can be painful, debilitating and life-threatening.\nIt is estimated that one in 50,000 people are affected by HAE. There are\npreventative and on-demand treatment options to help manage the condition,\nincluding long- and short-term prophylaxis used to prevent swelling attacks.\nCurrent treatment options often include lifelong therapies, which may require\nchronic intravenous (IV) or subcutaneous (SC) administration as often as twice\nper week or daily oral administration to ensure constant pathway suppression\nfor disease control. Despite chronic administration, breakthrough attacks\nstill occur. Kallikrein inhibition is a clinically validated strategy for the\npreventive treatment of HAE attacks.\n\nAbout Intellia Therapeutics\nIntellia Therapeutics, Inc. (Nasdaq: NTLA) is a leading clinical-stage\nbiopharmaceutical company focused on revolutionizing medicine leveraging\nCRISPR gene editing and other core technologies. The company’s mission is to\ntransform the lives of people with severe diseases by developing and\ncommercializing potentially curative treatments. With deep scientific,\ntechnical and clinical development experience, Intellia aims to reset the\nstandard for medicine by durably treating the root causes of disease. Learn\nmore at intelliatx.com\n(https://www.globenewswire.com/Tracker?data=kaMSRLJE3Jt4qVEaeL4NvN3lgPDpWrfjFJWVB-u3gofwkoA5Er2ECRzNwIDjLgj-lOlt7AYrOB-JrvlXv6yj5yDCef09x5O85Ddfwo04fjU=)\nand follow us @intelliatx.\n\nForward-Looking Statements\nThis press release contains “forward-looking statements” of Intellia\nTherapeutics, Inc. (“Intellia” or the “Company”) within the meaning of\nthe Private Securities Litigation Reform Act of 1995. These forward-looking\nstatements include, but are not limited to, express or implied statements\nregarding Intellia’s beliefs and expectations concerning: the success and\nadvancement of its program for lonvoguran ziclumeran or “lonvo-z” (also\nknown as NTLA-2002) for the treatment of hereditary angioedema (“HAE”),\nincluding its plan to complete the submission of a biologics license\napplication (“BLA”) for lonvo-z in the second half of 2026, its\nexpectations regarding review and approval of that BLA, including the\npotential for expedited review, its expectations regarding a planned\nU.S. launch of lonvo-z in first half of 2027, and the possibility that\nlonvo-z to revolutionize how HAE is treated for many patients with the\npotential to free most from both attacks and the need for ongoing therapy with\njust one dose.\n\nAny forward-looking statements in this press release are based on\nmanagement’s current expectations and beliefs of future events and are\nsubject to a number of risks and uncertainties that could cause actual results\nto differ materially and adversely from those set forth in or implied by such\nforward-looking statements. These risks and uncertainties include, but are not\nlimited to: uncertainties related to the conduct of clinical studies and other\ndevelopment and commercialization requirements for its product candidates,\nincluding lonvo-z, including risks related to the ability to develop and\nsuccessfully commercialize lonvo-z or any of Intellia’s product candidates;\nrisks related to Intellia’s ability to protect and maintain its intellectual\nproperty position; risks related to Intellia’s relationship with third\nparties, including its contract manufacturers, collaborators, licensors and\nlicensees; risks related to the ability of its licensors to protect and\nmaintain their intellectual property position; risks related to the results of\npreclinical studies or clinical studies not being predictive of future results\nin connection with future studies; the risk that clinical study results will\nnot be positive; and risks related to the potential delay of planned clinical\ntrials due to regulatory feedback or other developments. For a discussion of\nthese and other risks and uncertainties, and other important factors, any of\nwhich could cause Intellia’s actual results to differ from those contained\nin the forward-looking statements, see the section entitled “Risk Factors”\nin Intellia’s most recent annual report on Form 10-K, as well as discussions\nof potential risks, uncertainties, and other important factors in Intellia’s\nother filings with the Securities and Exchange Commission, including its\nquarterly report on Form 10-Q. All information in this press release is as of\nthe date of the release, and Intellia undertakes no duty to update this\ninformation unless required by law.\n\nInvestor Contact:\nJason Fredette\nVice President, Investor Relations and Corporate Communications\nIntellia Therapeutics, Inc.\njason.fredette@intelliatx.com\n\nMedia Contact:\nMike Tattory\nVice President\nLifeSci Communications\nmtattory@lifescicommunications.com \n\n(https://www.globenewswire.com/NewsRoom/AttachmentNg/9b6b17bb-c8a4-4009-b5f8-8cadb4d2296a)\n\n\n\nGlobeNewswire, Inc. 2026","article_body_html":"","raw_payload":{"data":{"id":"nGNX9kxW0R","title":"Intellia Therapeutics Initiates Rolling Submission of Biologics License Application to FDA for Lonvoguran Ziclumeran (lonvo-z) as a One-Time Treatment for Hereditary Angioedema","author":"Globe Newswire","ticker":"NTLA","created":"2026-04-27T11:00:00.370Z","tickers":["NTLA"],"exchange":"NASDAQ","article_body":"CAMBRIDGE, Mass., April 27, 2026 (GLOBE NEWSWIRE) -- Intellia Therapeutics,\nInc.\n(https://www.globenewswire.com/Tracker?data=XSbLXuLM_FJPa5n4RY-9U0ayTGTtFrUuqcdVUivSkYZ09lePzGxCBH591z_fX3RkbTYZMthG784urNz0kYsjReyhOe5s2i6NK9c-r9szGZuj9p3WElIVfhl_4dJw8rGW)\n(Nasdaq: NTLA), a leading biopharmaceutical company focused on revolutionizing\nmedicine leveraging CRISPR gene editing and other core technologies, today\nannounced it has initiated a rolling submission of a biologics license\napplication (BLA) to the U.S. Food and Drug Administration (FDA) seeking\napproval of lonvo-z (formerly known as NTLA-2002) for hereditary angioedema\n(HAE). Designed as a one-time treatment that is administered in an outpatient\nsetting, lonvo-z is an in vivo CRISPR gene editing candidate that is intended\nto inactivate the kallikrein B1 (KLKB1) gene to permanently lower kallikrein\nand bradykinin levels.\n\nIntellia also separately announced positive topline data today from the Phase\n3 HAELO clinical trial of lonvo-z in HAE. The trial met its primary and all\nkey secondary endpoints, demonstrating that a one-time dose of lonvo-z led to\nfreedom from both HAE attacks and the use of ongoing therapy for most patients\nduring the six-month primary observation period.\n\n“If approved, lonvo-z will become the world’s first in vivo CRISPR-based\ngene editing therapy,” said John Leonard, M.D., Intellia President and Chief\nExecutive Officer. “The promising results from HAELO reinforce our\nconviction that lonvo-z could revolutionize how HAE is treated for many\npatients, with the potential to free most of them from both attacks and the\nneed for ongoing therapy with just one dose. We look forward to our continued\nengagement with the FDA as we seek to ease many of the burdens for people\nliving with HAE.”\n\nPursuant to the Regenerative Medicine Advanced Therapy (RMAT) designation that\nthe FDA granted to lonvo-z for the treatment of HAE, a rolling BLA allows\nIntellia to submit portions of the BLA on an ongoing basis and provides the\nFDA with an opportunity to expedite its review. In addition to the RMAT\nprogram, Intellia participated in the FDA’s Chemistry, Manufacturing, and\nControls (CMC) Development and Readiness Pilot. This program allows sponsors\nto discuss their CMC product development strategies and goals with FDA review\nstaff and address their questions. The increased communication is intended to\nhelp sponsors complete CMC activities to expedite their drug development\nprograms to support application submission and earlier patient access.\n\nIntellia anticipates completing its BLA submission in the second half of 2026.\nIf the filing is accepted by the FDA, the agency is expected to determine if\nit will grant a priority review and provide a target action date to complete\nits evaluation. If approved, Intellia plans to launch lonvo-z commercially in\nthe first half of 2027.\n\nAbout Lonvo-z\nBased on Nobel Prize-winning CRISPR/Cas9 technology, lonvo-z has the potential\nto become the first one-time treatment for hereditary angioedema (HAE).\nLonvo-z is an in vivo CRISPR gene editing candidate that is intended to\npermanently lower kallikrein by inactivating the kallikrein B1 (KLKB1) gene\nwith a single dose. Lonvo-z has received five notable regulatory designations:\nOrphan Drug and RMAT Designation by the U.S. Food and Drug Administration\n(FDA), the Innovation Passport by the U.K. Medicines and Healthcare products\nRegulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the\nEuropean Medicines Agency, as well as Orphan Drug Designation (ODD) by the\nEuropean Commission.\n\nAbout Hereditary Angioedema\nHereditary angioedema (HAE) is a rare, genetic disease characterized by\nsevere, recurring and unpredictable inflammatory attacks in various organs and\ntissues of the body, which can be painful, debilitating and life-threatening.\nIt is estimated that one in 50,000 people are affected by HAE. There are\npreventative and on-demand treatment options to help manage the condition,\nincluding long- and short-term prophylaxis used to prevent swelling attacks.\nCurrent treatment options often include lifelong therapies, which may require\nchronic intravenous (IV) or subcutaneous (SC) administration as often as twice\nper week or daily oral administration to ensure constant pathway suppression\nfor disease control. Despite chronic administration, breakthrough attacks\nstill occur. Kallikrein inhibition is a clinically validated strategy for the\npreventive treatment of HAE attacks.\n\nAbout Intellia Therapeutics\nIntellia Therapeutics, Inc. (Nasdaq: NTLA) is a leading clinical-stage\nbiopharmaceutical company focused on revolutionizing medicine leveraging\nCRISPR gene editing and other core technologies. The company’s mission is to\ntransform the lives of people with severe diseases by developing and\ncommercializing potentially curative treatments. With deep scientific,\ntechnical and clinical development experience, Intellia aims to reset the\nstandard for medicine by durably treating the root causes of disease. Learn\nmore at intelliatx.com\n(https://www.globenewswire.com/Tracker?data=kaMSRLJE3Jt4qVEaeL4NvN3lgPDpWrfjFJWVB-u3gofwkoA5Er2ECRzNwIDjLgj-lOlt7AYrOB-JrvlXv6yj5yDCef09x5O85Ddfwo04fjU=)\nand follow us @intelliatx.\n\nForward-Looking Statements\nThis press release contains “forward-looking statements” of Intellia\nTherapeutics, Inc. (“Intellia” or the “Company”) within the meaning of\nthe Private Securities Litigation Reform Act of 1995. These forward-looking\nstatements include, but are not limited to, express or implied statements\nregarding Intellia’s beliefs and expectations concerning: the success and\nadvancement of its program for lonvoguran ziclumeran or “lonvo-z” (also\nknown as NTLA-2002) for the treatment of hereditary angioedema (“HAE”),\nincluding its plan to complete the submission of a biologics license\napplication (“BLA”) for lonvo-z in the second half of 2026, its\nexpectations regarding review and approval of that BLA, including the\npotential for expedited review, its expectations regarding a planned\nU.S. launch of lonvo-z in first half of 2027, and the possibility that\nlonvo-z to revolutionize how HAE is treated for many patients with the\npotential to free most from both attacks and the need for ongoing therapy with\njust one dose.\n\nAny forward-looking statements in this press release are based on\nmanagement’s current expectations and beliefs of future events and are\nsubject to a number of risks and uncertainties that could cause actual results\nto differ materially and adversely from those set forth in or implied by such\nforward-looking statements. These risks and uncertainties include, but are not\nlimited to: uncertainties related to the conduct of clinical studies and other\ndevelopment and commercialization requirements for its product candidates,\nincluding lonvo-z, including risks related to the ability to develop and\nsuccessfully commercialize lonvo-z or any of Intellia’s product candidates;\nrisks related to Intellia’s ability to protect and maintain its intellectual\nproperty position; risks related to Intellia’s relationship with third\nparties, including its contract manufacturers, collaborators, licensors and\nlicensees; risks related to the ability of its licensors to protect and\nmaintain their intellectual property position; risks related to the results of\npreclinical studies or clinical studies not being predictive of future results\nin connection with future studies; the risk that clinical study results will\nnot be positive; and risks related to the potential delay of planned clinical\ntrials due to regulatory feedback or other developments. For a discussion of\nthese and other risks and uncertainties, and other important factors, any of\nwhich could cause Intellia’s actual results to differ from those contained\nin the forward-looking statements, see the section entitled “Risk Factors”\nin Intellia’s most recent annual report on Form 10-K, as well as discussions\nof potential risks, uncertainties, and other important factors in Intellia’s\nother filings with the Securities and Exchange Commission, including its\nquarterly report on Form 10-Q. All information in this press release is as of\nthe date of the release, and Intellia undertakes no duty to update this\ninformation unless required by law.\n\nInvestor Contact:\nJason Fredette\nVice President, Investor Relations and Corporate Communications\nIntellia Therapeutics, Inc.\njason.fredette@intelliatx.com\n\nMedia Contact:\nMike Tattory\nVice President\nLifeSci Communications\nmtattory@lifescicommunications.com \n\n(https://www.globenewswire.com/NewsRoom/AttachmentNg/9b6b17bb-c8a4-4009-b5f8-8cadb4d2296a)\n\n\n\nGlobeNewswire, Inc. 2026"},"type":"article","timestamp":"2026-04-27T11:00:00.424526694Z","server_sent_at_ms":1777287600424},"received_at":"2026-04-27T11:00:00.477Z","source_url":null},"analysis":{"id":"6536","press_release_id":"8703","analysis_json":{"industry":{"label":"Pharmaceuticals, Biotechnology & Life Sciences","sector":"Health Care"},"redFlags":[],"eventType":"regulatory","narrative":"Intellia initiated a rolling BLA submission for lonvo-z, seeking FDA approval for the world's first in vivo CRISPR-based gene editing therapy for hereditary angioedema.\n\nThe submission is backed by positive Phase 3 HAELO data where a one-time dose met primary and key secondary endpoints, freeing most patients from attacks and ongoing therapy.\n\nThe company anticipates completing the submission in the second half of 2026 and plans a commercial launch in the first half of 2027.","sentiment":"bullish","agentHooks":{"shouldPost":true,"suggestedAngle":"Historic first in vivo CRISPR therapy moves to FDA review with strong Phase 3 data."},"keyFigures":{"drugName":"Lonvoguran Ziclumeran (lonvo-z)","phaseOfTrial":"Phase 3"},"quotedText":"If approved, lonvo-z will become the world’s first in vivo CRISPR-based gene editing therapy,","namedEntities":{"people":[{"name":"John Leonard","role":"President and Chief Executive Officer"},{"name":"Jason Fredette","role":"Vice President, Investor Relations and Corporate Communications"},{"name":"Mike Tattory","role":"Vice President, LifeSci Communications"}],"products":["Lonvoguran Ziclumeran","lonvo-z","NTLA-2002"],"companies":[{"name":"Intellia Therapeutics, Inc.","ticker":"NTLA"},{"name":"U.S. Food and Drug Administration","relationship":"regulator"}],"dollarAmounts":[]},"materialImpact":{"score":5,"reasoning":"Initiation of a rolling BLA submission for a potential first-in-class in vivo CRISPR therapy, supported by positive Phase 3 data showing clinical benefit. This represents a major de-risking event and a significant milestone toward commercialization."},"tickerRelevance":{"others":[],"primary":"NTLA"},"globalImportance":65,"audienceRelevance":75,"eventTypeSecondary":["clinical_trial"],"importanceComponents":{"novelty":"First in vivo CRISPR therapy","tickerTier":"Mid-cap Biotech","eventGravity":"BLA submission + Phase 3 success","sectorWeight":"Biotech/FDA Binary"}},"event_type":"regulatory","event_type_secondary":["clinical_trial"],"sentiment":"bullish","material_impact_score":5,"narrative":"Intellia initiated a rolling BLA submission for lonvo-z, seeking FDA approval for the world's first in vivo CRISPR-based gene editing therapy for hereditary angioedema.\n\nThe submission is backed by positive Phase 3 HAELO data where a one-time dose met primary and key secondary endpoints, freeing most patients from attacks and ongoing therapy.\n\nThe company anticipates completing the submission in the second half of 2026 and plans a commercial launch in the first half of 2027.","key_figures":{"drugName":"Lonvoguran Ziclumeran (lonvo-z)","phaseOfTrial":"Phase 3"},"named_entities":{"people":[{"name":"John Leonard","role":"President and Chief Executive Officer"},{"name":"Jason Fredette","role":"Vice President, Investor Relations and Corporate Communications"},{"name":"Mike Tattory","role":"Vice President, LifeSci Communications"}],"products":["Lonvoguran Ziclumeran","lonvo-z","NTLA-2002"],"companies":[{"name":"Intellia Therapeutics, Inc.","ticker":"NTLA"},{"name":"U.S. Food and Drug Administration","relationship":"regulator"}],"dollarAmounts":[]},"model_name":"glm-4.7","prompt_hash":"sha256:1fc968c3ac7f7ebb","schema_hash":"sha256:1f8af1196c7db48c","created_at":"2026-04-27T11:40:22.093Z","global_importance":65,"audience_relevance":75,"importance_components":{"novelty":"First in vivo CRISPR therapy","tickerTier":"Mid-cap Biotech","eventGravity":"BLA submission + Phase 3 success","sectorWeight":"Biotech/FDA Binary"}},"durationMs":57035,"modelName":"glm-4.7"}}