{"event":"gene-editing-tracker","title":"Gene editing tracker: approvals, trials and setbacks","state":"developing","updated":"2026-10-10T00:00:00.000Z","entries":[{"id":"iec9ly","at":"2026-10-08T00:00:00.000Z","title":"CTX310 Phase 1b data set for AHA late-breaking session","status":"confirmed","cites":[{"id":"gene-editing-ctx310-aha-2026","n":25,"url":"https://crisprtx.com/about-us/press-releases-and-presentations/crispr-therapeutics-to-present-late-breaking-data-at-the-american-heart-association-aha-scientific-sessions-2026","publisher":"CRISPR Therapeutics","short":"CRISPR Therapeutics"}]},{"id":"1nntr5r","at":"2026-09-24T00:00:00.000Z","title":"FDA clears Prime Medicine's IND for PM647 in AATD","status":"confirmed","body":"The second in vivo prime editor to reach the clinic, after the Wilson disease program PM577a.","cites":[{"id":"gene-editing-pm647-ind-2026","n":11,"url":"https://www.nasdaq.com/press-release/prime-medicine-announces-us-fda-clearance-investigational-new-drug-application-pm647","publisher":"Prime Medicine (press release via Nasdaq)","short":"Prime Medicine"},{"id":"gene-editing-pm577a-program","n":24,"url":"https://www.sec.gov/Archives/edgar/data/1894562/000162828026053878/q220268-kex991xpressrelease.htm","publisher":"Prime Medicine (SEC filing)","short":"Prime Medicine"}]},{"id":"s7ggpb","at":"2026-09-08T00:00:00.000Z","title":"FDA accepts lonvo-z BLA with Priority Review; Beam reports longer base-editing follow-up","status":"confirmed","body":"Target action date 10 March 2027. The same day, Beam presented BEAM-302 data with up to 18 months of follow-up and an accelerated-approval plan.","cites":[{"id":"gene-editing-lonvo-z-bla-accepted","n":3,"url":"https://www.sec.gov/Archives/edgar/data/1652130/000119312526385194/ntla-ex99_1.htm","publisher":"Intellia Therapeutics (SEC filing)","short":"Intellia Therapeutics"},{"id":"gene-editing-beam302-ers-2026","n":34,"url":"https://www.nasdaq.com/press-release/beam-therapeutics-presents-updated-clinical-data-phase-1-2-trial-beam-302-alpha-1","publisher":"Beam Therapeutics (press release via Nasdaq)","short":"Beam Therapeutics"},{"id":"gene-editing-beam302-accelerated-path","n":9,"url":"https://www.nasdaq.com/press-release/beam-therapeutics-presents-updated-clinical-data-phase-1-2-trial-beam-302-alpha-1","publisher":"Beam Therapeutics (press release via Nasdaq)","short":"Beam Therapeutics"}]},{"id":"1qil1fy","at":"2026-08-28T00:00:00.000Z","title":"CTX310 holds lipid reductions at one year","status":"confirmed","body":"Mean reductions at the highest dose were 79% for ANGPTL3, 48% for triglycerides and 53% for LDL cholesterol, published in NEJM the same day.","cites":[{"id":"gene-editing-ctx310-durability-2026","n":58,"url":"https://crisprtx.com/about-us/press-releases-and-presentations/crispr-therapeutics-presents-phase-1a-data-for-ctx310-demonstrating-deep-and-durable-angptl3-editing-triglyceride-and-ldl-lowering-at-esc-congress-2026","publisher":"CRISPR Therapeutics","short":"CRISPR Therapeutics"}]},{"id":"12pim0k","at":"2026-08-06T00:00:00.000Z","title":"Intellia links nex-z liver signals to one HLA allele","status":"confirmed","body":"A genomic analysis of more than 600 patient samples found the highest transaminase elevations in carriers of one allele.","cites":[{"id":"gene-editing-nex-z-hla-finding","n":21,"url":"https://www.sec.gov/Archives/edgar/data/0001652130/000119312526336772/ntla-ex99_1.htm","publisher":"Intellia Therapeutics (SEC filing)","short":"Intellia Therapeutics"},{"id":"gene-editing-magnitude2-enrollment","n":20,"url":"https://www.sec.gov/Archives/edgar/data/0001652130/000119312526336772/ntla-ex99_1.htm","publisher":"Intellia Therapeutics (SEC filing)","short":"Intellia Therapeutics"}]},{"id":"cnj63h","at":"2026-08-04T00:00:00.000Z","title":"Beam doses first patient in BEAM-302 pivotal cohort","status":"confirmed","body":"Dosing took place in July 2026, in alpha-1 antitrypsin deficiency. Dosing is also complete in the risto-cel BEACON trial.","cites":[{"id":"gene-editing-beam302-pivotal","n":57,"url":"https://www.sec.gov/Archives/edgar/data/0001745999/000119312526331522/beam-ex99_1.htm","publisher":"Beam Therapeutics (SEC filing)","short":"Beam Therapeutics"},{"id":"gene-editing-beam-beacon-dosing","n":23,"url":"https://www.sec.gov/Archives/edgar/data/0001745999/000119312526331522/beam-ex99_1.htm","publisher":"Beam Therapeutics (SEC filing)","short":"Beam Therapeutics"}]},{"id":"xj4ldh","at":"2026-07-31T00:00:00.000Z","title":"Prime Medicine wins arbitration over its AATD prime editor","status":"confirmed","body":"The tribunal found PM647 within Prime Medicine's licensed field, with no breach and no damages. The company's filing dates the ruling to July 2026.","cites":[{"id":"gene-editing-prime-beam-arbitration","n":56,"url":"https://www.sec.gov/Archives/edgar/data/1894562/000162828026053878/q220268-kex991xpressrelease.htm","publisher":"Prime Medicine (SEC filing)","short":"Prime Medicine"}]},{"id":"tp21i5","at":"2026-07-01T00:00:00.000Z","title":"FDA extends Casgevy to children aged 2 and older","status":"confirmed","cites":[{"id":"gene-editing-casgevy-peds-2026","n":6,"url":"https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-young-children-sickle-cell-disease","publisher":"US Food and Drug Administration","short":"US Food and Drug…"},{"id":"gene-editing-casgevy-peds-data","n":55,"url":"https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-young-children-sickle-cell-disease","publisher":"US Food and Drug Administration","short":"US Food and Drug…"}]},{"id":"efywzk","at":"2026-06-30T00:00:00.000Z","title":"FDA clears Beam's IND for a base editor in phenylketonuria","status":"confirmed","body":"Beam's filing dates the BEAM-304 clearance to June 2026 without giving a day.","cites":[{"id":"gene-editing-beam304-ind-2026","n":54,"url":"https://www.sec.gov/Archives/edgar/data/0001745999/000119312526331522/beam-ex99_1.htm","publisher":"Beam Therapeutics (SEC filing)","short":"Beam Therapeutics"}]},{"id":"1op0a5n","at":"2026-06-13T00:00:00.000Z","title":"HAELO Phase 3 results for lonvo-z published in NEJM","status":"confirmed","body":"80 patients were randomized 2:1; no serious or Grade 3 or higher adverse events were reported in the lonvo-z group.","cites":[{"id":"gene-editing-haelo-nejm","n":52,"url":"https://www.sec.gov/Archives/edgar/data/0001652130/000119312526336772/ntla-ex99_1.htm","publisher":"Intellia Therapeutics (SEC filing)","short":"Intellia Therapeutics"},{"id":"gene-editing-haelo-nejm-details","n":53,"url":"https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=DOI:10.1056/NEJMoa2600931&resultType=core&format=json","publisher":"New England Journal of Medicine (via Europe PMC)","short":"New England Journal…"}]},{"id":"1f14mom","at":"2026-06-03T00:00:00.000Z","title":"FDA draft guidance on leveraging prior knowledge for editing products","status":"confirmed","cites":[{"id":"gene-editing-fda-prior-knowledge-2026","n":29,"url":"https://www.federalregister.gov/api/v1/documents/2026-11054.json","publisher":"Federal Register (Food and Drug Administration)","short":"Federal Register"}]},{"id":"16bix4x","at":"2026-04-30T00:00:00.000Z","title":"Intellia starts rolling BLA for lonvo-z","status":"confirmed","body":"The company's filings date the start to April 2026 without giving a day. Phase 3 HAELO showed an 87% attack reduction.","cites":[{"id":"gene-editing-lonvo-z-rolling-bla","n":51,"url":"https://www.sec.gov/Archives/edgar/data/0001652130/000119312526215721/ntla-ex99_1.htm","publisher":"Intellia Therapeutics (SEC filing)","short":"Intellia Therapeutics"},{"id":"gene-editing-haelo-results","n":15,"url":"https://www.sec.gov/Archives/edgar/data/0001652130/000119312526215721/ntla-ex99_1.htm","publisher":"Intellia Therapeutics (SEC filing)","short":"Intellia Therapeutics"}]},{"id":"t0bwpd","at":"2026-04-15T00:00:00.000Z","title":"FDA draft guidance on sequencing-based safety assessment of editing","status":"confirmed","cites":[{"id":"gene-editing-fda-ngs-guidance-2026","n":28,"url":"https://www.federalregister.gov/api/v1/documents/2026-07285.json","publisher":"Federal Register (Food and Drug Administration)","short":"Federal Register"}]},{"id":"1wjyibs","at":"2026-03-30T00:00:00.000Z","title":"Customizable prime-editing platform reported for urea cycle disorders","status":"confirmed","body":"An LNP-plus-AAV system corrected about 30 to 40% of variant copies in preclinical liver DNA, and the team is discussing an umbrella trial with the FDA.","cites":[{"id":"gene-editing-ucd-prime-platform-2026","n":50,"url":"https://www.chop.edu/news/new-fda-plausible-mechanism-framework-spurs-development-personalized-vivo-prime-editing","publisher":"Children's Hospital of Philadelphia","short":"Children's Hospital…"},{"id":"gene-editing-umbrella-trial-plan","n":30,"url":"https://www.chop.edu/news/new-fda-plausible-mechanism-framework-spurs-development-personalized-vivo-prime-editing","publisher":"Children's Hospital of Philadelphia","short":"Children's Hospital…"}]},{"id":"8h2xd7","at":"2026-02-25T00:00:00.000Z","title":"FDA draft guidance on plausible mechanism framework","status":"confirmed","cites":[{"id":"gene-editing-plausible-mechanism-2026","n":27,"url":"https://www.federalregister.gov/api/v1/documents/2026-03713.json","publisher":"Federal 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Therapeutics"}]},{"id":"4eldmm","at":"2025-12-07T00:00:00.000Z","title":"Prime editing results for chronic granulomatous disease published in NEJM","status":"confirmed","cites":[{"id":"gene-editing-pm359-nejm-2025","n":48,"url":"https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=DOI:10.1056/NEJMoa2509807&resultType=core&format=json","publisher":"New England Journal of Medicine (via Europe PMC)","short":"New England Journal…"}]},{"id":"c6i0ms","at":"2025-11-08T00:00:00.000Z","title":"CTX310 in vivo CRISPR Phase 1 results published in NEJM","status":"confirmed","body":"Serious adverse events occurred in two of 15 participants, including one sudden death 179 days after the lowest dose; the authors reported no dose-limiting toxic effects related to CTX310.","cites":[{"id":"gene-editing-ctx310-2025","n":46,"url":"https://crisprtx.com/about-us/press-releases-and-presentations/crispr-therapeutics-announces-positive-phase-1-clinical-data-for-ctx310-demonstrating-deep-and-durable-angptl3-editing-triglyceride-and-lipid-lowering","publisher":"CRISPR Therapeutics","short":"CRISPR Therapeutics"},{"id":"gene-editing-ctx310-serious-events","n":47,"url":"https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=DOI:10.1056/NEJMoa2511778&resultType=core&format=json","publisher":"New England Journal of Medicine (via Europe PMC)","short":"New England Journal…"}]},{"id":"hx651i","at":"2025-11-05T00:00:00.000Z","title":"Nex-z trial participant with severe liver injury dies","status":"confirmed","body":"Intellia's later filing says the investigator attributed the death to septic shock secondary to a perforated duodenal ulcer, in a course that also included acute liver injury.","cites":[{"id":"gene-editing-nex-z-death-cause","n":31,"url":"https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm","publisher":"Intellia Therapeutics (SEC filing)","short":"Intellia Therapeutics"}]},{"id":"1eh7g4m","at":"2025-10-31T00:00:00.000Z","title":"CHOP and Penn teams propose umbrella trials for bespoke editors","status":"confirmed","body":"A perspective in The American Journal of Human Genetics set out trial designs that treat many patient-specific editors as one drug.","cites":[{"id":"gene-editing-umbrella-trial-plan","n":30,"url":"https://www.chop.edu/news/new-fda-plausible-mechanism-framework-spurs-development-personalized-vivo-prime-editing","publisher":"Children's Hospital of Philadelphia","short":"Children's Hospital…"},{"id":"gene-editing-kj-expanded-access","n":40,"url":"https://www.chop.edu/news/researchers-behind-personalized-crispr-therapy-plan-launch-new-type-clinical-trial","publisher":"Children's Hospital of Philadelphia","short":"Children's Hospital…"}]},{"id":"11x6x4r","at":"2025-10-29T00:00:00.000Z","title":"FDA puts Intellia's nex-z Phase 3 trials on clinical hold","status":"confirmed","cites":[{"id":"gene-editing-nex-z-hold-2025","n":45,"url":"https://www.sec.gov/Archives/edgar/data/1652130/000119312525274596/d12611d8k.htm","publisher":"Intellia Therapeutics (SEC filing)","short":"Intellia Therapeutics"}]},{"id":"1ybigad","at":"2025-07-18T00:00:00.000Z","title":"FDA acts on liver deaths linked to AAV gene therapy Elevidys","status":"confirmed","body":"A gene therapy (not gene editing) safety event relevant to in vivo delivery.","cites":[{"id":"gene-editing-elevidys-2025","n":44,"url":"https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold","publisher":"US Food and Drug Administration","short":"US Food and Drug…"}]},{"id":"1tavcwx","at":"2025-06-17T00:00:00.000Z","title":"Eli Lilly agrees to acquire base-editing company Verve","status":"confirmed","body":"The deal closed in July 2025 for about $549.4 million net of cash acquired, plus a contingent value right of up to $3.00 a share.","cites":[{"id":"gene-editing-verve-lilly","n":42,"url":"https://www.sec.gov/Archives/edgar/data/1840574/000119312525141748/d30505dex991.htm","publisher":"Verve Therapeutics (SEC filing)","short":"Verve Therapeutics"},{"id":"gene-editing-verve-acquisition-closed","n":43,"url":"https://www.sec.gov/Archives/edgar/data/59478/000005947825000254/lly-20250930.htm","publisher":"Eli Lilly and Company (SEC filing)","short":"Eli Lilly and Company"}]},{"id":"18ui2h7","at":"2025-05-19T00:00:00.000Z","title":"First human prime-editing data (PM359)","status":"confirmed","cites":[{"id":"gene-editing-pm359-2025","n":41,"url":"https://www.sec.gov/Archives/edgar/data/1894562/000119312525122156/d863812dex991.htm","publisher":"Prime Medicine (SEC filing)","short":"Prime Medicine"}]},{"id":"1re5icw","at":"2025-05-15T00:00:00.000Z","title":"First personalized in vivo base-editing therapy reported","status":"confirmed","body":"An infant with CPS1 deficiency was treated under single-patient expanded access with a custom LNP-delivered base editor; reported in NEJM.","cites":[{"id":"gene-editing-kj-therapy","n":17,"url":"https://www.chop.edu/news/worlds-first-patient-treated-personalized-crispr-gene-editing-therapy-childrens-hospital","publisher":"Children's Hospital of Philadelphia","short":"Children's Hospital…"},{"id":"gene-editing-kj-nejm","n":39,"url":"https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=DOI:10.1056/NEJMoa2504747&resultType=core&format=json","publisher":"New England Journal of Medicine (via Europe PMC)","short":"New England Journal…"},{"id":"gene-editing-kj-expanded-access","n":40,"url":"https://www.chop.edu/news/researchers-behind-personalized-crispr-therapy-plan-launch-new-type-clinical-trial","publisher":"Children's Hospital of Philadelphia","short":"Children's Hospital…"}]},{"id":"wzdoyc","at":"2024-01-16T00:00:00.000Z","title":"FDA approves Casgevy for beta-thalassemia","status":"confirmed","cites":[{"id":"gene-editing-casgevy-tdt-2024","n":38,"url":"https://www.fda.gov/vaccines-blood-biologics/casgevy","publisher":"US Food and Drug Administration","short":"US Food and Drug…"}]},{"id":"1qb6h30","at":"2023-12-08T00:00:00.000Z","title":"FDA approves Casgevy and Lyfgenia for sickle cell disease","status":"confirmed","body":"Vertex set Casgevy's US list price at $2.2 million; Lyfgenia's wholesale cost was $3.1 million.","cites":[{"id":"gene-editing-casgevy-fda-2023","n":4,"url":"https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease","publisher":"US Food and Drug Administration","short":"US Food and Drug…"},{"id":"gene-editing-lyfgenia-2023","n":37,"url":"https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease","publisher":"US Food and Drug Administration","short":"US Food and Drug…"},{"id":"gene-editing-casgevy-list-price","n":7,"url":"https://www.biopharmadive.com/news/crispr-fda-approval-sickle-cell-gene-editing-vertex-casgevy/701522/","publisher":"BioPharma Dive","short":"BioPharma Dive"}]},{"id":"1qefgkq","at":"2023-11-16T00:00:00.000Z","title":"UK authorises Casgevy, the first CRISPR medicine","status":"confirmed","cites":[{"id":"gene-editing-casgevy-mhra-2023","n":2,"url":"https://www.gov.uk/government/news/mhra-authorises-world-first-gene-therapy-that-aims-to-cure-sickle-cell-disease-and-transfusion-dependent-thalassemia","publisher":"UK Medicines and Healthcare products Regulatory Agency","short":"UK Medicines…"}]},{"id":"xai6qa","at":"2021-06-26T00:00:00.000Z","title":"Early in vivo CRISPR results for NTLA-2001 published in NEJM","status":"confirmed","body":"An LNP-delivered Cas9 editor lowered serum TTR by a mean 87% at the higher dose.","cites":[{"id":"gene-editing-ntla2001-2021","n":36,"url":"https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=DOI:10.1056/NEJMoa2107454&resultType=core&format=json","publisher":"New England Journal of Medicine (via Europe PMC)","short":"New England Journal…"}]},{"id":"18j59bg","at":"2020-10-07T00:00:00.000Z","title":"CRISPR-Cas9 developers share Nobel Prize in Chemistry","status":"confirmed","cites":[{"id":"gene-editing-nobel-2020","n":35,"url":"https://news.berkeley.edu/2020/10/07/jennifer-doudna-wins-2020-nobel-prize-in-chemistry/","publisher":"UC Berkeley News","short":"UC Berkeley News"}]},{"id":"1f4xxjx","at":"2012-08-17T00:00:00.000Z","title":"Science paper shows programmable RNA-guided Cas9 cutting","status":"confirmed","body":"The work that launched CRISPR-Cas9 genome editing.","cites":[{"id":"gene-editing-jinek-2012","n":1,"url":"https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22745249%20AND%20SRC:MED&resultType=core&format=json","publisher":"Science (via Europe PMC)","short":"Science"}]}]}