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    Gene editing tracker: approvals, trials and setbacks

    Gene editing moved from a 2012 lab discovery to an approved medicine in 2023, and as of October 2026 the first in vivo CRISPR therapy is under FDA Priority Review with a decision due by 10 March 2027.[1][2][3] This tracker lists confirmed milestones, safety events and what to watch next.

    Editor reviewedStrict sourcingUpdated Gene editing and gene therapyHealth and medicineLife sciences

    What we know

    • Casgevy is approved in 39 countries as of August 2026, at a US list price of $2.2 million.[7]
    • The FDA extended Casgevy to children aged 2 and older on 1 July 2026.[6]
    • Lonvo-z cut hereditary angioedema attacks by 87% versus placebo in Phase 3.[15]
    • The FDA's target action date for lonvo-z is 10 March 2027, with no advisory committee planned.[8]
    • Nex-z Phase 3 holds imposed in October 2025 were lifted in January and March 2026 with enhanced liver monitoring.[33]
    • The nex-z trial death was attributed by the investigator to septic shock from a perforated duodenal ulcer, after acute liver injury.[31]
    • Beam had dosed 38 patients with the in vivo base editor BEAM-302 as of 17 August 2026.[34]
    • Prime editing now has two cleared in vivo INDs, for Wilson disease and alpha-1 antitrypsin deficiency.[11]
    • The FDA issued three gene-editing-related draft guidances between February and June 2026.[29]

    What we don't know yet

    • Whether the FDA will approve lonvo-z by its 10 March 2027 target date, and with what post-marketing conditions.
    • Which HLA allele Intellia's genomic analysis implicated in nex-z liver enzyme elevations, and whether genotyping will be used to screen patients.
    • When the FDA will finalize its plausible mechanism, prior-knowledge and sequencing guidances, and how they will apply to gene editors.
    • Whether Beam's risto-cel application is filed by year-end 2026 as planned, and whether the CHOP-Penn umbrella trial for urea cycle disorders opens.
    • How many patients have received Casgevy worldwide; the sources reviewed report revenue, not patient counts.
    Story status

    Story status

    State
    developing
    Started
    2012-08-17
    Timeline entries
    32
    Last entry

    Timeline data (JSON)

    This tracker follows gene editing from discovery to the clinic.[1][4] The latest verified entry is 8 October 2026. It describes evidence and regulatory status, not medical advice. For background, start with the crash course hub and the crispr-cas9 page.

    Where things stand (October 2026)

    One CRISPR medicine, casgevy, is approved. It is an ex vivo therapy for sickle cell disease and beta-thalassemia, available in 39 countries, in the US from age 2, at a $2.2 million US list price.[5][6][7] No in vivo CRISPR therapy is approved yet. Intellia’s lonvo-z is under FDA Priority Review, and the agency has said it is not currently planning an advisory committee meeting on the application.[3][8] base-editing and prime-editing programs are in pivotal, registration-directed or newly cleared first-in-human stages.[9][10][11]

    Key numbers

    • $2.2 million, Casgevy’s US list price at approval; Lyfgenia was priced at $3.1 million.[7]
    • 39 countries where Casgevy is approved as of August 2026, and $76 million in second-quarter 2026 revenue.[5][12]
    • 5,500 more patients Vertex estimates became eligible with the 2026 pediatric approval.[13]
    • 7.74 million people living with sickle cell disease worldwide in 2021, nearly 80% of them in sub-Saharan Africa.[14]
    • 87% fewer hereditary angioedema attacks with a single lonvo-z infusion than with placebo in Phase 3.[15]
    • 89% of known disease-associated variants that prime editing could in principle correct, as estimated in the 2019 paper.[16]
    • About six months to design and manufacture the first personalized base editor for one infant.[17]

    What to watch next

    • 10 March 2027: FDA action date for lonvo-z. An approval would make it the first in vivo CRISPR therapy.[3] Intellia plans a US launch in the first half of 2027 if it is approved, and announced a debt facility of up to $400 million in September 2026.[18][19]
    • Nex-z enrollment and safety. Intellia expected to finish MAGNITUDE-2 enrollment in the second half of 2026, and is sharing HLA genotyping results with investigators after its genomic analysis.[20][21]
    • Base-editing filings. Beam plans to file for approval of risto-cel for sickle cell disease as early as year-end 2026, with updated BEACON data expected by then, and BEAM-302’s pivotal cohort is enrolling toward an accelerated-approval package.[22][23][9]
    • Prime-editing milestones. Prime Medicine targets a PM359 filing in the first half of 2027, with first data from the Wilson disease and AATD programs expected in 2027.[10][24][11]
    • CRISPR Therapeutics’ in vivo programs. Phase 1b CTX310 data in severe hypertriglyceridemia go to the AHA meeting on 9 November 2026, and the company says it is working on in vivo editing of blood stem cells.[25][26]
    • Regulation of editing and bespoke editors. The FDA issued draft guidances on a plausible mechanism framework, on sequencing-based safety assessment and on the use of prior knowledge during 2026.[27][28][29] A CHOP-Penn umbrella trial covering seven urea cycle disorders would be an early test of the framework in practice.[30] See personalized-gene-editing.

    Open questions

    • Whether the FDA approves lonvo-z by its 10 March 2027 action date, and on what safety conditions.[3]
    • Whether the HLA association found in the nex-z trials explains the severe liver events, and how screening will be used.[21][31]
    • Whether biomarker-based accelerated approval is accepted for BEAM-302 in AATD.[9]
    • Whether in vivo editing of blood stem cells can replace the collection and chemotherapy conditioning that ex vivo therapy needs.[26][32]
    • Whether gene editing reaches the countries carrying most of the sickle cell burden.[14]

    How this tracker is maintained

    • Entries come from FDA, Federal Register, SEC and journal feeds.
    • An entry is marked confirmed only with a tier-1 source or two independent publishers.
    • Where a company filing gives only a month, the entry is dated to a representative day in that month and the body says so.

    Timeline

    32 confirmed

    1. confirmed

      CTX310 Phase 1b data set for AHA late-breaking session[25]

    2. confirmed

      FDA clears Prime Medicine's IND for PM647 in AATD[11][24]

      The second in vivo prime editor to reach the clinic, after the Wilson disease program PM577a.

    3. confirmed

      FDA accepts lonvo-z BLA with Priority Review; Beam reports longer base-editing follow-up[3][34][9]

      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.

    4. confirmed

      CTX310 holds lipid reductions at one year[58]

      Mean reductions at the highest dose were 79% for ANGPTL3, 48% for triglycerides and 53% for LDL cholesterol, published in NEJM the same day.

    5. confirmed

      Intellia links nex-z liver signals to one HLA allele[21][20]

      A genomic analysis of more than 600 patient samples found the highest transaminase elevations in carriers of one allele.

    6. confirmed

      Beam doses first patient in BEAM-302 pivotal cohort[57][23]

      Dosing took place in July 2026, in alpha-1 antitrypsin deficiency. Dosing is also complete in the risto-cel BEACON trial.

    7. confirmed

      Prime Medicine wins arbitration over its AATD prime editor[56]

      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.

    8. confirmed

      FDA extends Casgevy to children aged 2 and older[6][55]

    9. confirmed

      FDA clears Beam's IND for a base editor in phenylketonuria[54]

      Beam's filing dates the BEAM-304 clearance to June 2026 without giving a day.

    10. confirmed

      HAELO Phase 3 results for lonvo-z published in NEJM[52][53]

      80 patients were randomized 2:1; no serious or Grade 3 or higher adverse events were reported in the lonvo-z group.

    11. confirmed

      FDA draft guidance on leveraging prior knowledge for editing products[29]

    12. confirmed

      Intellia starts rolling BLA for lonvo-z[51][15]

      The company's filings date the start to April 2026 without giving a day. Phase 3 HAELO showed an 87% attack reduction.

    13. confirmed

      FDA draft guidance on sequencing-based safety assessment of editing[28]

    14. confirmed

      Customizable prime-editing platform reported for urea cycle disorders[50][30]

      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.

    15. confirmed

      FDA draft guidance on plausible mechanism framework[27]

    16. confirmed

      One year on, CHOP reports continued benefit in the first bespoke-editing patient[49]

      The hospital says the child tolerated three infusions with no serious side effects, but that the treatment is not a cure.

    17. confirmed

      FDA lifts hold on MAGNITUDE-2; MAGNITUDE follows in March[33]

    18. confirmed

      Prime editing results for chronic granulomatous disease published in NEJM[48]

    19. confirmed

      CTX310 in vivo CRISPR Phase 1 results published in NEJM[46][47]

      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.

    20. confirmed

      Nex-z trial participant with severe liver injury dies[31]

      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.

    21. confirmed

      CHOP and Penn teams propose umbrella trials for bespoke editors[30][40]

      A perspective in The American Journal of Human Genetics set out trial designs that treat many patient-specific editors as one drug.

    22. confirmed

      FDA puts Intellia's nex-z Phase 3 trials on clinical hold[45]

    23. confirmed

      FDA acts on liver deaths linked to AAV gene therapy Elevidys[44]

      A gene therapy (not gene editing) safety event relevant to in vivo delivery.

    24. confirmed

      Eli Lilly agrees to acquire base-editing company Verve[42][43]

      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.

    25. confirmed

      First human prime-editing data (PM359)[41]

    26. confirmed

      First personalized in vivo base-editing therapy reported[17][39][40]

      An infant with CPS1 deficiency was treated under single-patient expanded access with a custom LNP-delivered base editor; reported in NEJM.

    27. confirmed

      FDA approves Casgevy for beta-thalassemia[38]

    28. confirmed

      FDA approves Casgevy and Lyfgenia for sickle cell disease[4][37][7]

      Vertex set Casgevy's US list price at $2.2 million; Lyfgenia's wholesale cost was $3.1 million.

    29. confirmed

      UK authorises Casgevy, the first CRISPR medicine[2]

    30. confirmed

      Early in vivo CRISPR results for NTLA-2001 published in NEJM[36]

      An LNP-delivered Cas9 editor lowered serum TTR by a mean 87% at the higher dose.

    31. confirmed

      CRISPR-Cas9 developers share Nobel Prize in Chemistry[35]

    32. confirmed

      Science paper shows programmable RNA-guided Cas9 cutting[1]

      The work that launched CRISPR-Cas9 genome editing.

    Sources

    Each numbered claim is a statement we checked against the sources listed with it. Status shows how well established it is.

    1. [1]

      A 2012 Science paper by Jinek, Doudna, Charpentier and colleagues showed that a two-RNA structure directs the Cas9 protein to make double-stranded breaks in target DNA, and highlighted its potential for RNA-programmable genome editing. confirmedas of 2026-10-10

    2. [2]

      On 16 November 2023 the UK medicines regulator, the MHRA, authorised Casgevy for patients aged 12 and over with sickle cell disease or transfusion-dependent beta-thalassemia, the first authorisation of a CRISPR-based medicine. confirmedas of 2026-10-10

    3. [3]

      On 8 September 2026 Intellia said the FDA had accepted its lonvo-z application with Priority Review and a target action date of 10 March 2027; the company says lonvo-z would be the world's first in vivo CRISPR-based therapy if approved. confirmedas of 2026-10-10

    4. [4]

      On 8 December 2023 the FDA approved Casgevy, the first FDA-approved therapy using CRISPR/Cas9, for people aged 12 and older with sickle cell disease and recurrent vaso-occlusive crises. confirmedas of 2026-10-10

    5. [5]

      As of August 2026, Casgevy was approved in 39 countries across North America, Europe and the Middle East. confirmedas of 2026-08-03

    6. [6]

      On 1 July 2026 the FDA expanded Casgevy's approval to patients aged 2 years and older with sickle cell disease or transfusion-dependent beta-thalassemia, 53 days after filing, under the Commissioner's National Priority Voucher pilot program. confirmedas of 2026-10-10

    7. [7]

      Vertex set Casgevy's US list price at $2.2 million per patient when it was approved in December 2023; Lyfgenia's wholesale acquisition cost was set at $3.1 million. confirmedas of 2026-10-10

    8. [8]

      Lonvo-z holds five regulatory designations - FDA Orphan Drug and Regenerative Medicine Advanced Therapy, the UK MHRA Innovation Passport, EMA PRIME and European Commission Orphan Drug - and the FDA told Intellia in September 2026 that it was not currently planning an advisory committee meeting on the application. confirmedas of 2026-09-08

    9. [9]

      Beam says that after FDA feedback it is pursuing an accelerated approval pathway for BEAM-302 based on a primary endpoint of AAT biomarkers measured over 12 months at a 60 mg dose, and expects to enroll about 50 additional patients with AATD-associated lung disease in a pivotal expansion of its Phase 1/2 trial. confirmedas of 2026-09-08

    10. [10]

      The FDA granted PM359 Regenerative Medicine Advanced Therapy designation in June 2026, and Prime Medicine is working toward a BLA submission in the first half of 2027. confirmedas of 2026-08-06

    11. [11]

      On 24 September 2026 Prime Medicine said the FDA had cleared its investigational new drug application for PM647, an in vivo prime editor designed to correct the E342K (Pi*Z) mutation in SERPINA1; the global single-arm Phase 1/2 study will start with adults who have lung-only AATD and then add a cohort with significant liver disease, with initial clinical data expected in 2027. confirmedas of 2026-09-24

    12. [12]

      Casgevy generated $76 million in revenue in the second quarter of 2026, up 78% from the prior quarter. confirmedas of 2026-10-10

    13. [13]

      Vertex estimates that about 5,500 more patients with sickle cell disease or beta-thalassemia became eligible for Casgevy with the 2026 pediatric approval. confirmedas of 2026-10-10

    14. [14]

      WHO estimates that 7.74 million people were living with sickle cell disease in 2021, with 515,000 new births with the disease, and that sub-Saharan Africa accounts for nearly 80% of global cases. confirmedas of 2026-10-10

    15. [15]

      In the Phase 3 HAELO trial, a single infusion of lonvo-z reduced hereditary angioedema attacks by 87% versus placebo over weeks 5 to 28 (mean monthly attack rate 0.26 versus 2.10), and 62% of treated patients were attack-free and therapy-free versus 11% on placebo. confirmedas of 2026-10-10

    16. [16]

      The 2019 prime editing paper estimated that the method could in principle correct up to 89% of known disease-associated genetic variants. confirmedas of 2026-10-10

    17. [17]

      In 2025 a team at Children's Hospital of Philadelphia and Penn Medicine designed and manufactured, within about six months, a personalized base-editing therapy delivered by lipid nanoparticles to the liver for an infant with severe CPS1 deficiency, a rare urea-cycle disorder. confirmedas of 2026-10-10

    18. [18]

      Intellia plans a US launch of lonvo-z in the first half of 2027 if it is approved. reportedas of 2026-10-10· forecast

    19. [19]

      Intellia reported $628.4 million in cash, cash equivalents and marketable securities as of 30 June 2026, which it expects to fund operations at least into 2028, and in September 2026 it announced a non-dilutive debt facility with OrbiMed of up to $400 million. confirmedas of 2026-09-04

    20. [20]

      As of August 2026 Intellia expected to complete enrollment in MAGNITUDE-2 in the second half of 2026. reportedas of 2026-08-06· forecast

    21. [21]

      In August 2026 Intellia said that a genomic analysis of more than 600 patient samples from nex-z trials, run with Regeneron and outside experts, found the highest observed liver transaminase elevations in patients carrying one specific HLA allele, and that it would give HLA genotyping results to investigators and to patients enrolled or entering screening in the ongoing Phase 3 trials. confirmedas of 2026-08-06

    22. [22]

      Beam plans to submit a Biologics License Application for risto-cel (formerly BEAM-101), a base-edited cell therapy for sickle cell disease, as early as year-end 2026. reportedas of 2026-08-04· forecast

    23. [23]

      Beam reported in August 2026 that dosing was complete for all adult and adolescent patients in the Phase 1/2 BEACON trial of risto-cel in sickle cell disease, with updated data expected by year-end 2026. confirmedas of 2026-08-04

    24. [24]

      New Zealand's Medsafe cleared Prime Medicine's trial application for the Wilson disease prime editor PM577a in June 2026, the company's first clinical authorization for an in vivo prime-editing therapy, and the FDA cleared its US investigational new drug application in July 2026, establishing a global open-label Phase 1/2 study with initial clinical data anticipated in 2027. confirmedas of 2026-08-06

    25. [25]

      On 8 October 2026 CRISPR Therapeutics said Phase 1b data for CTX310 in severe hypertriglyceridemia would be presented in a late-breaking oral session at the American Heart Association Scientific Sessions on 9 November 2026. confirmedas of 2026-10-08

    26. [26]

      CRISPR Therapeutics says it is advancing in vivo editing of blood-forming stem cells using lipid nanoparticle delivery, an approach it says could expand the treatable population for sickle cell disease and beta-thalassemia beyond ex vivo therapy. confirmedas of 2026-08-03

    27. [27]

      On 25 February 2026 the FDA published draft guidance on a "plausible mechanism" framework for generating evidence of effectiveness and safety for individualized therapies that target specific genetic conditions with a known biological cause; comments closed on 27 April 2026. confirmedas of 2026-10-10

    28. [28]

      On 15 April 2026 the FDA published draft guidance recommending next-generation sequencing methods for the nonclinical safety assessment of genome editing in human gene therapy products, with comments due by 14 July 2026. confirmedas of 2026-10-10

    29. [29]

      On 3 June 2026 the FDA published draft guidance on the prior knowledge that sponsors of ex vivo and in vivo genome-editing products may scientifically leverage to advance product development, with comments due by 1 September 2026. confirmedas of 2026-10-10

    30. [30]

      The CHOP and Penn team behind the personalized CPS1 therapy is planning an umbrella Phase 1/2 trial under the FDA's plausible mechanism framework that would enroll patients with any of seven urea cycle disorders receiving customized prime editors, treating all versions as a single drug; the researchers say successful treatment of as few as 5 to 10 participants might be enough to support approval of the platform. confirmedas of 2026-03-30

    31. [31]

      Intellia's August 2026 quarterly report states that the MAGNITUDE participant who had Grade 4 liver transaminase elevations and increased bilirubin after a nex-z dose died on 5 November 2025, and that the principal investigator reported the cause as septic shock secondary to a perforated duodenal ulcer, with a clinical course that also included acute liver injury treated with corticosteroids and an autopsy report supporting the clinical diagnoses. confirmedas of 2026-08-06

    32. [32]

      Treatment with Casgevy involves collecting the patient's own stem cells, editing them, giving myeloablative conditioning (high-dose chemotherapy) to clear the bone marrow, and infusing the edited cells back as a one-time treatment. confirmedas of 2026-10-10

    33. [33]

      The FDA lifted the clinical hold on Intellia's MAGNITUDE-2 trial in January 2026 and the hold on MAGNITUDE in March 2026, after the company agreed to study modifications including enhanced monitoring of liver laboratory tests; enrollment then advanced in both Phase 3 trials. confirmedas of 2026-08-06

    34. [34]

      At the European Respiratory Society Congress in September 2026 Beam reported that 38 patients had been dosed with BEAM-302 as of 17 August 2026, and that in the 60 mg lung-disease cohort steady-state total AAT averaged 14.4 micromolar against 5.0 micromolar at baseline, above the 11 micromolar protective threshold, with an 84% reduction in circulating mutant Z-AAT. confirmedas of 2026-09-08

    35. [35]

      Jennifer Doudna and Emmanuelle Charpentier shared the 2020 Nobel Prize in Chemistry for developing CRISPR-Cas9 genome editing. confirmedas of 2026-10-10

    36. [36]

      In a 2021 NEJM study, NTLA-2001, a lipid nanoparticle carrying Cas9 mRNA and a guide RNA targeting the TTR gene, given by infusion, lowered blood TTR protein by a mean 87% at the 0.3 mg/kg dose, with mainly mild adverse events. confirmedas of 2026-10-10

    37. [37]

      On the same day as Casgevy, the FDA approved Lyfgenia, a sickle cell gene therapy that uses a lentiviral vector rather than gene editing; 28 of 32 patients (88%) had complete resolution of vaso-occlusive events in its trial. confirmedas of 2026-10-10

    38. [38]

      The FDA approved Casgevy for transfusion-dependent beta-thalassemia on 16 January 2024. confirmedas of 2026-10-10

    39. [39]

      The case was reported in NEJM in May 2025; CPS1 deficiency has an estimated 50% mortality in early infancy, and in the seven weeks after the first infusion the infant tolerated more dietary protein and half the starting dose of a nitrogen-scavenger medicine, with no serious adverse events. confirmedas of 2026-10-10

    40. [40]

      The infant treated in 2025 received his therapy through the single-patient expanded-access (compassionate use) pathway, and his team says that version of the therapy will not work for any other patient, although swapping the part that targets the genome could address other liver-centred genetic disorders. confirmedas of 2025-10-31

    41. [41]

      In May 2025 Prime Medicine reported the first clinical data for prime editing in humans, from PM359, an ex vivo prime-edited stem cell therapy for chronic granulomatous disease (CGD); the first patient reached 58% DHR-positive neutrophils by day 15 and 66% by day 30, with no serious adverse events related to PM359. confirmedas of 2026-10-10

    42. [42]

      On 17 June 2025 Eli Lilly agreed to acquire Verve Therapeutics for $10.50 per share in cash plus a contingent value right of up to $3.00 per share; Verve's lead program VERVE-102 is designed to permanently turn off the PCSK9 gene in the liver to lower cholesterol. confirmedas of 2026-10-10

    43. [43]

      Eli Lilly completed its acquisition of Verve Therapeutics in July 2025 for $10.50 per share in cash, about $549.4 million net of cash acquired, plus a contingent value right of up to $3.00 per share worth up to roughly $300 million; the acquired in-process research and development related primarily to VERVE-102. confirmedas of 2025-10-30

    44. [44]

      In July 2025 the FDA said three deaths appeared to result from acute liver failure in people treated with AAV-vector gene therapies from Sarepta, requested that Sarepta suspend Elevidys distribution, and placed some of its trials on clinical hold. confirmedas of 2026-10-10

    45. [45]

      On 29 October 2025 the FDA placed clinical holds on Intellia's Phase 3 MAGNITUDE and MAGNITUDE-2 trials of nex-z after a patient dosed in MAGNITUDE had Grade 4 liver transaminase elevations and increased bilirubin. confirmedas of 2026-10-10

    46. [46]

      In November 2025 CRISPR Therapeutics reported Phase 1 data, published in NEJM, for CTX310, an LNP-delivered CRISPR therapy that edits the ANGPTL3 gene in liver cells; at the highest dose mean ANGPTL3 fell 73%, triglycerides 55% and LDL cholesterol 49%, with no treatment-related serious adverse events reported. confirmedas of 2026-10-10

    47. [47]

      In the published CTX310 Phase 1 trial, serious adverse events occurred in two of 15 participants (13%): one had a spinal disk herniation and the other died suddenly 179 days after treatment with the lowest, 0.1 mg per kilogram dose; the authors reported no dose-limiting toxic effects related to CTX310. confirmedas of 2025-11-08

    48. [48]

      The PM359 results were published in NEJM in December 2025: two participants with p47phox-deficient chronic granulomatous disease received the prime-edited stem cell therapy after busulfan conditioning, neutrophils and platelets engrafted promptly in both, and NADPH oxidase activity appeared within one month and was maintained for six and four months respectively at last follow-up. confirmedas of 2025-12-07

    49. [49]

      Children's Hospital of Philadelphia said in February 2026, one year after the first infusion, that the infant treated with the personalized CPS1 base editor had tolerated three infusions given from February through April 2025 with no serious side effects, handled more dietary protein, needed less nitrogen-scavenging medication and showed better ammonia control during childhood illnesses, and that the treatment is not a cure. confirmedas of 2026-02-24

    50. [50]

      In March 2026 researchers at Children's Hospital of Philadelphia, Penn Medicine and the Broad Institute reported in The American Journal of Human Genetics a customizable two-part in vivo prime-editing platform for urea cycle disorders - a lipid nanoparticle carrying editor mRNA to the liver plus an AAV supplying guide RNAs - that corrected about 30 to 40% of copies of a disease-causing variant in preclinical liver DNA, above the roughly 10% the researchers consider necessary for clinical benefit. confirmedas of 2026-03-30

    51. [51]

      Intellia began a rolling Biologics License Application to the FDA for lonvo-z in April 2026. confirmedas of 2026-10-10

    52. [52]

      The HAELO Phase 3 results were published in the New England Journal of Medicine in June 2026. confirmedas of 2026-10-10

    53. [53]

      The HAELO Phase 3 trial randomized 80 patients 2:1 to a single 50 mg infusion of lonvo-z or placebo; median follow-up was 7.5 months as of 10 February 2026, and no serious or Grade 3 or higher adverse events were reported in the lonvo-z group. confirmedas of 2026-06-13

    54. [54]

      In June 2026 the FDA cleared Beam's investigational new drug application for BEAM-304, a base editor for phenylketonuria that initially targets the R408W mutation, and the company began clinical start-up activities for a Phase 1/2 trial. confirmedas of 2026-08-04

    55. [55]

      The pediatric approval rested on trials in which all 8 evaluable children with sickle cell disease (ages 5 to 12) had no severe crises for 12 months and 8 of 9 with beta-thalassemia became transfusion-independent; use in children aged 2 to 5 relied on extrapolation. confirmedas of 2026-10-10

    56. [56]

      In July 2026 Prime Medicine announced a binding arbitration ruling under its 2019 collaboration agreement with Beam Therapeutics: the tribunal declared that PM647 is within Prime Medicine's defined field, that Prime Medicine did not breach the agreement and that it owes no monetary damages. confirmedas of 2026-08-06

    57. [57]

      In July 2026 Beam dosed the first patient in the global pivotal cohort of its BEAM-302 trial in AATD-associated lung disease. confirmedas of 2026-10-10

    58. [58]

      At the European Society of Cardiology Congress on 28 August 2026, CRISPR Therapeutics reported one-year Phase 1a follow-up for CTX310, with mean reductions from baseline at the highest dose of 79% for ANGPTL3, 48% for triglycerides and 53% for LDL cholesterol, and said no additional treatment-related adverse events had occurred since the previous update; the data were published in NEJM the same day. confirmedas of 2026-08-28

    Revision history (3)
    1. Page created.
    2. Refresh: timeline extended to 8 October 2026 with FDA guidances, the nex-z HLA finding, CTX310 durability and serious adverse events, Beam's ERS data, the PM647 and PM577a INDs, Casgevy's list price and the Verve close; the nex-z death cause and hold-lift dates now cite Intellia's quarterly report.
    3. Added key numbers and open questions sections.

    Created Oct 10, 2026. Last reviewed by an editor on Oct 10, 2026. Next scheduled review: Jan 10, 2027.

    Cite this page

    "Gene editing tracker: approvals, trials and setbacks." ContentLora, updated Oct 10, 2026. https://contentlora.com/events/gene-editing-tracker

    Spotted an error? Suggest a correction or emailcorrections@contentlora.com.

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