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Intellia Therapeutics | Company Profile | iPS Cells & Regenerative Medicine

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COMPANY PROFILE / iPS CELLS & REGENERATIVE MEDICINE

Intellia Therapeutics
A specialist in "in vivo genome editing": editing genes inside the body with a single infusion, without taking cells out

Intellia works with neither iPS cells nor cell transplantation. It earns a place in this field because it is the leading example of the approach at the opposite pole from therapies such as CASGEVY, which edit cells outside the body and put them back: editing directly inside the body. The company says it was the first in the world to take an in vivo genome-editing candidate into Phase 3. In April 2026 the HAELO Phase 3 trial met its primary endpoint and Intellia began a rolling BLA submission to the FDA. In October 2025, however, nex-z was placed on clinical hold. This profile concentrates on what the company invests in, who it works with and what it is aiming for.

Primary sources: Intellia's Form 10-K for the year ended December 2025 (filed with the U.S. SEC on 26 February 2026) and its Form 10-Q for the quarter ended June 2026 (filed 6 August 2026). Last updated October 2026.

What this article covers
  1. Intellia in 30 seconds, and where it stands in iPS cells and regenerative medicine
  2. The logo and how the name is written
  3. Where the company sits in iPS cells and regenerative medicine
  4. Company scale
  5. Resources committed to iPS cells and regenerative medicine
  6. Track record so far
  7. Plans ahead
  8. What the company is aiming for
  9. Partnership map for gene editing and cell therapy
  10. Technologies and products on offer
  11. The risks this company carries
  12. Glossary, references and claim-to-source audit

1. Intellia in 30 seconds, and where it stands in iPS cells and regenerative medicine

LEGAL NAMEIntellia Therapeutics, Inc.A Delaware corporation, founded in May 2014
HEADQUARTERSCambridge,
Massachusetts, U.S.
40 Erie Street (cover of the 10-K)
LISTINGNasdaq: NTLAA U.S.-listed company
CATEGORY IN THIS FIELDGenome editing & gene therapySpecialises in editing directly inside the body
HOW THE THERAPIES WORKIn vivo genome editing
(to the liver via LNP, single infusion)
No iPS cell or cell transplant product of its own
LEAD PRODUCTSlonvo-z (HAE)
nex-z (ATTR)
Both in Phase 3. Not yet approved
EMPLOYEES377As of 13 February 2026, of whom 263 are in R&D
CASH AND SECURITIESUS$628.4 millionAt 30 June 2026
The one thing to grasp first

Intellia is a specialist in "rewriting the genome directly inside the body" that handles neither iPS cells nor cell transplants. In its 10-K the company says it was the first in the world to advance an in vivo genome-editing candidate into Phase 3 Sourced.

We include it in this field because it is the leading example of the opposite approach to "ex vivo editing cell therapies such as CASGEVY", which are the main players in iPS cells and regenerative medicine: editing cells inside the body with a single infusion, without removing them. Note that Intellia itself has not stepped into the neighbouring territory of regenerative medicine, where cells are made and then transplanted Not yet confirmed.

2. The logo and how the name is written

A note on names: the legal entity is Intellia Therapeutics, Inc. We could not confirm an official Japanese-language rendering in the material we used, so this article uses the English name throughout. Regeneron Pharmaceuticals, its co-development partner, and Caribou Biosciences, from which it licensed the CRISPR/Cas9 patents, are both separate companies. It is also a separate legal entity from CRISPR Therapeutics, another "CRISPR company", which has its own profile in this series.

Official site: https://www.intelliatx.com/

3. Where the company sits in iPS cells and regenerative medicine

Three types of company involved in iPS cells and regenerative medicine All are "regenerative medicine companies", but they treat disease in completely different ways Large pharma & strategic partners Genome editing & gene therapy iPS cell & cell therapy Enters via deals; has sales network Rewrites genes directly to treat Makes and transplants cells Vertex / Novartis Bristol Myers Squibb and others CRISPR Therapeutics / Intellia Beam / Prime Medicine and others Fate / Sana Cuorips / Heartseed and others Late-stage and sales funding Editing tools, safety know-how Cell production and QC scale Thin in-house cell/gene base Swayed by partner strategy shifts Heavy cost and long-term safety Intellia is the middle box: a specialist in editing inside the body, without removing cells It widens cell therapy (allogeneic CAR-T) through licences to partners and handles no iPS cells itself
Fig. 1 Three types of company involved in iPS cells and regenerative medicine, and where Intellia sits. The classification is our own, and the example companies are drawn from those covered in this series Our calculation. Only the middle box is highlighted. In vivo editing differs from ex vivo editing cell therapies such as CASGEVY in both mechanism and manufacturing.
Therapeutic approachWhere Intellia fitsStatusCategory
Genome editing (directly inside the body)lonvo-z (KLKB1 gene) and nex-z (TTR gene). Delivered to the liver in lipid nanoparticles (LNPs)Phase 3. A rolling BLA for lonvo-z is under waySourced
Genome editing (cells edited outside the body)No product of its own is described. Licensed to AvenCell, Kyverna and others. Regeneron also holds non-exclusive rights to up to 10 ex vivo editing productsOne AvenCell programme is in Phase 1Sourced
Allogeneic cell therapy (CAR-T)Developed by AvenCell (a joint venture) and Kyverna (KYV-201). Intellia sits on the equity, milestone and royalty sideLed by the partnersSourced
iPS-cell-derived cellsNo mention of "iPS" in the 10-K or 10-QCould not be confirmedNot yet confirmed
Viral gene delivery such as AAVThe main products use LNPs. No AAV-based product is described-Sourced
Mesenchymal stem cells and tissue engineeringNot describedCould not be confirmedNot yet confirmed

Our explainers on genome editing, off-target effects and safety assessment, lipid nanoparticles, transthyretin amyloidosis and CAR-T cells give the background to each row of the table.

4. Company scale

Figures are in U.S. dollars and the fiscal year ends in December. All of Intellia's revenue is revenue from collaboration agreements; it has no product sales Sourced.

Measure (US$ millions)20242025First half of 2026
Collaboration revenue57.967.722.7
Research and development expense466.3388.9163.3
General and administrative expense125.8119.872.7
Net loss519.0412.7202.9
Cash and marketable securities (period end)Not confirmed in the sources used605.1628.4

Intellia had 377 employees at 13 February 2026 (263 mainly in R&D, and 106 with an M.D. or Ph.D.) Sourced. The 10-Q records that it cut its workforce in January 2025.

In April 2026 it raised equity, bringing in net proceeds of US$194.6 million (US$10.75 per share, 16,744,187 shares, including shares sold on exercise of the option to purchase additional shares) Sourced. General and administrative expense in the first half of 2026 was about US$16.5 million higher than a year earlier. The 10-Q attributes this to higher legal costs and to higher personnel and consulting costs as it builds up a commercial function.

A word of caution

The figures above are for the whole company. They do not measure the size of any iPS cell or regenerative medicine business. The company states plainly that all revenue to date has been collaboration revenue, and it has no product sales yet Sourced.

5. Resources committed to iPS cells and regenerative medicine

There are no figures for iPS cells and regenerative medicine alone. We show the company's resources as a genome-editing company, along four lines.

ResourceDetailsCategory
People (company-wide)377 (13 February 2026): 263 in R&D and 106 with an M.D. or Ph.D. Workforce reduction in January 2025Sourced
People (iPS cells and regenerative medicine)No dedicated team is describedCould not be confirmed
SitesHeadquarters in Cambridge (about 65,000 sq ft), with several research buildings in the same city. About 158,000 sq ft of office, laboratory and manufacturing space in Waltham (to be terminated by June 2028). About 125,000 sq ft at Technology Square in Cambridge, leased from September 2026Sourced
Money (R&D expense)US$388.9 million company-wide (2025); US$163.3 million in the first half of 2026Sourced
Money (cash)US$628.4 million (30 June 2026). Net proceeds of US$194.6 million from the April 2026 equity raiseSourced
Investments and acquisitionsAcquired Rewrite Therapeutics (2022; about US$45.0 million upfront plus up to US$155.0 million in milestones, with a US$25.0 million research milestone paid in 2023). Equity in AvenCell (initially 33.33%, below 10% at the end of December 2025) and in KyvernaSourced
In-licensingAn exclusive licence from Caribou Biosciences to CRISPR/Cas9-related patents (2014; for human therapeutic use). It includes an exclusive sublicence of rights held by the University of California, the University of Vienna and Dr. CharpentierSourced
Out-licensing and collaborationRegeneron (from April 2016): joint development of liver editing. Up to US$320.0 million in milestones per target and royalties up to the mid single-digit percent of sales. For nex-z in ATTR, development and promotion are shared, with Regeneron bearing about 25% of development costs and profits. Also agreements with AvenCell, Kyverna and ONKSourced
Universities, research institutes and public fundingThe University of California and the University of Vienna as patent rights holders. No joint research agreements or grants relating to iPS cells are describedSourced / could not be confirmed

6. Track record so far

WhenWhat happenedWhat it means
May 2014Incorporated in Delaware (originally named AZRN, Inc.; renamed in July)Started out as a CRISPR specialist
July 2014Obtains an exclusive licence to CRISPR/Cas9-related patents from Caribou BiosciencesSecured the rights to the editing tool
April 2016Licence and collaboration agreement with Regeneron (centred on liver editing)Gained access to a genetics database and mouse models
December 2016Agreement with the University of California, the University of Vienna, Dr. Charpentier, Caribou, CRISPR Therapeutics and others to coordinate management of the core patentsThe parties around the patents built a framework for cooperation
July 2021Co-founds AvenCell as a joint venture (allogeneic universal CAR-T)Chose to put cell therapy into a joint venture
December 2021Licenses its ex vivo editing platform for allogeneic CAR-T to Kyverna (KYV-201)Cell therapy led by the partner
2022Acquires Rewrite Therapeutics (merged in September)An acquisition combining upfront and contingent payments
2024Starts the Phase 3 trial of nex-z in ATTR cardiomyopathy (MAGNITUDE). In November, the IND for the Phase 3 trial in ATTRv-PN (MAGNITUDE-2) is acceptedIn vivo editing entered Phase 3
January 2025First patient dosed in the HAELO Phase 3 trial. Workforce reductionConcentrated resources on development
September 2025HAELO enrolment completed (80 people)Recruitment for the pivotal trial finished
29 October 2025The FDA places MAGNITUDE and MAGNITUDE-2 on clinical hold, after a patient showed Grade 4 liver enzyme and bilirubin elevations following dosing with nex-z. The patient died on 5 NovemberSafety of in vivo editing moved to the front
January and March 2026The FDA lifts the clinical holds on MAGNITUDE-2 (January) and MAGNITUDE (March)Development resumed
April 2026Announces that the HAELO Phase 3 trial met its primary endpoint. Begins a rolling BLA submission to the FDA. Raises net proceeds of US$194.6 million in an equity offeringMoved into filing for approval
June 2026HAELO results published in NEJMPeer-reviewed Phase 3 data
August 2026Reports that, in a genetic analysis of more than 600 samples, the highest liver enzyme values were concentrated in patients carrying particular HLA typesThe search for the cause of the safety signal advanced

7. Plans ahead

LONVO-Z BLAFDA acceptance expected
in the second half of 2026
Rolling submission in progress. Not a guarantee of approval
LONVO-Z LAUNCHPlanned for the
first half of 2027
If approved. The company's plan
MAGNITUDE-2 ENROLMENTExpected to complete in
the second half of 2026
Phase 3 in ATTRv-PN
NEX-Z AND HLAIn discussion with
the FDA
HLA typing results to be provided to participants
MAGNITUDEEnrolment continuesATTR cardiomyopathy. Estimated enrolment about 1,200
NEW SITELease starts in
September 2026
About 125,000 sq ft at Technology Square
REGENERONJoint development and promotionFor nex-z, Intellia leads clinical development and sales
iPS CELL-RELATEDNo plan describedNothing found in the 10-K or 10-Q

Of the items in the table, the company gives timing for the lonvo-z BLA and launch and the MAGNITUDE-2 enrolment Sourced. All are plans, not guarantees of approval or launch Not yet confirmed.

What could not be confirmed

None of the following appears in the material we used Not yet confirmed.

The price of lonvo-z / the timing of FDA approval / when the results of the nex-z Phase 3 trials will come / whether there is a causal link between HLA and liver enzyme elevation / any plan to enter iPS cells and regenerative medicine. Timings and prices in press reports cannot be verified against primary sources, so this article does not use them.

8. What the company is aiming for

This section includes things the company does not say directly. We set them out as inferences within what the primary sources support, with the evidence for each.

What the evidence suggestsPrimary information it rests onCategory
A policy of taking products all the way to launch itselfThe 10-K says it will build an integrated commercial organisation. A lonvo-z launch is planned for the first half of 2027Sourced
By not removing cells, it is trying to avoid the burdens carried by ex vivo editing therapies (collecting cells, conditioning, shipping to a manufacturing site)The 10-K says a single infusion, given in an outpatient setting. The primary sources contain no comparison with ex vivo editing therapiesInference
It does not own cell therapy itself but spreads it through licensingLicences to AvenCell, Kyverna and ONK, and to Regeneron for up to 10 ex vivo editing products. No cell therapy product of its own is describedInference
The safety of in vivo editing is the biggest hurdleThe clinical hold in October 2025 and the HLA analysis disclosed in August 2026. No serious adverse events were reported in HAELOInference
An intention to extend beyond the liver to other tissuesThe 10-K says it will advance gene editing in tissues other than the liverSourced
One piece of evidence in more detail

We read the August 2026 disclosure as important to readers interested in regenerative medicine too, because it hints that the immune type (HLA) may be linked to the safety of in vivo editing Inference. According to the 10-Q, in the analysis of more than 600 samples the largest liver enzyme elevations were seen in patients with particular HLA types Sourced. HLA is the immune type that is the key to "rejection" in cell transplantation, and it is also the subject of our explainer on MHC and universal donors. The company has not explained how this result arises, however, and is in discussion with the FDA. We cannot conclude that HLA is the cause Not yet confirmed.

9. Partnership map for gene editing and cell therapy

Gene-editing and cell therapy partnerships centred on Intellia Only relationships in the FY2025 10-K and June 2026 10-Q; press-only links are excluded Intellia Genome editing Regeneron Co-develops nex-z; bears ~25% Caribou Biosciences Exclusive patent licence (2014) AvenCell CAR-T joint venture (under 10%) Kyverna Allogeneic CD19 CAR-T licensed out Rewrite Therapeutics Acquired and merged in 2022 Co-development and licensing Equity, acquisition
Fig. 2 Intellia's partnerships in gene editing and cell therapy. Sources: Form 10-K for FY2025 and Form 10-Q for June 2026. Only relationships described in both filings are shown. Intellia also has an agreement with ONK Therapeutics, but its content could not be confirmed in the material we used, so it is left out.

The thing to notice in this map is that all of Intellia's involvement in cell therapy is on the "giving out" side. In AvenCell its stake, initially 33.33%, had fallen below 10% by the end of December 2025, and in Kyverna the partner bears development and costs Sourced.

10. Technologies and products on offer

ProductHow it worksTargetStatus
lonvo-z
(NTLA-2002)
Inactivates the KLKB1 gene in the liver inside the body. Delivered in LNPs as a single intravenous dose (50 mg)Hereditary angioedema (HAE)Phase 3 (HAELO) met its primary endpoint. Rolling BLA under way. RMAT, orphan and other designations
nex-z
(NTLA-2001)
Inactivates the TTR gene in the liver inside the body. Delivered in LNPs as a single dose (55 mg)ATTR amyloidosis (cardiomyopathy and polyneuropathy)Phase 3 (MAGNITUDE, MAGNITUDE-2). Clinical holds lifted. Developed with Regeneron
Research programmesEditing in tissues other than the liver, and new technologies such as DNA writing-Preclinical and research
Partners' cell therapiesAllogeneic CAR-T such as KYV-201 (Kyverna), using Intellia's ex vivo editing platformAutoimmune diseases and othersDeveloped by the partners

Trial results (figures from papers or the 10-Q, with comparator and time point)

TrialPopulation and designResultTime point and caveats
HAELO (Phase 3, NCT06634420)HAE, ages 16 and older. 80 people randomised 2:1 (52 lonvo-z, 28 placebo), double-blind, a single 50 mg doseMonthly attack rate from weeks 5 to 28 was 0.26 with lonvo-z and 2.10 with placebo (an 87% relative reduction). No serious adverse events in the lonvo-z groupMedian follow-up 7.5 months (as of 10 February 2026). Placebo recipients can cross over to lonvo-z at week 28
lonvo-z Phase 1/2 (10-K)32 people given 50 mg. Single-arm, no comparison groupMean reduction in plasma kallikrein of 89% at 24 months. 31 people (97%) were attack-free and off long-term prophylaxis (at the data cut)No comparison group. Data as of 29 August 2025
nex-z Phase 1, ATTR-CM (10-Q)36 people. Single-armMean reduction in serum TTR of 87% in the 9 people who reached 36 monthsAs of 23 August 2025. A non-randomised, after-the-fact comparison with a separate group of 1,792 people under the same conditions, including mortality, is also described (not randomised)
nex-z Phase 1, ATTRv-PN (10-Q)33 people at 0.3 mg/kg or higher. Single-armMean reduction in serum TTR of 92% at 24 months (90% in the 12 people who reached 36 months)Grade 3 or higher liver enzyme elevations in 3 people (not serious; resolved on their own)
A materials engineer's view: a therapy made by "chemical manufacturing" of nucleic acids and lipids

Intellia's therapy is not a cell but a drug in which Cas9 mRNA and guide RNA are packaged in lipid nanoparticles (LNPs) Sourced. There is no need to freeze living cells and ship them, and the manufacturing process comes closer to chemical manufacturing: synthesising nucleic acids, then mixing and purifying lipids Inference.

The 10-K says that drug substance, drug product, viral vectors and edited cells are supplied through outside manufacturers and its own capacity, and are made in GMP facilities Sourced. What determines quality is the composition and purity of the lipids, the uniformity of particle size, and the synthetic purity of the mRNA and guide RNA, which are different kinds of material from the culture media, scaffolds and cryopreservation media that matter in ex vivo editing cell therapies.

11. The risks this company carries

RiskDetailsCategory
Safety (nex-z)The FDA placed a clinical hold in October 2025. A patient who showed Grade 4 liver enzyme elevation after dosing died (according to the treating physician, the cause of death was septic shock following a perforated duodenal ulcer, and the course also included acute liver injury and its steroid treatment). The hold has been lifted, but the company is in discussion with the FDA about the link with HLASourced
No product yetCollaboration revenue only, and no approved product. Approval and launch of lonvo-z is the first hurdleSourced
Cash burnNet loss for the first half of 2026 was US$202.9 million. It has been covered by equity raises, and the company states that it may need to raise more fundsSourced
PatentsThe core patents obtained through Caribou are subject to interference proceedings with the Broad Institute, ToolGen and Sigma-Aldrich. The 10-K states that if any of them prevails, it could assert its rights against commercialisation of Intellia's productsSourced
Long-term safetyThe 10-K lists as risks unexpected adverse events and the potentially permanent nature of the effects of genome editingSourced
Partner's directionThe technology collaboration with Regeneron runs through April 2026, and we could not confirm an extension in the material we usedSourced / not yet confirmed

12. Glossary

In vivo editing
A method in which a drug is infused and edits cells directly inside the body. No cells are taken out.
LNP (lipid nanoparticle)
A tiny particle made of lipids that wraps nucleic acids and delivers them into cells.
KLKB1 / kallikrein
The gene that makes plasma kallikrein, which is involved in HAE attacks, and the enzyme itself.
HAE (hereditary angioedema)
An inherited disease with repeated attacks of swelling.
ATTR (transthyretin amyloidosis)
A disease in which a protein called TTR builds up in tissues. It has cardiomyopathy and polyneuropathy forms.
BLA
Biologics License Application, the U.S. application for approval of a biologic. A rolling submission sends in completed sections one by one.
Clinical hold
An order from the FDA to stop a clinical trial, for reasons such as safety.
HLA
The immune type, a set of markers by which the immune system tells "self" from "other". It is involved in transplant rejection.
Allogeneic CAR-T
A cell therapy in which a healthy donor's T cells are given a receptor that targets cancer or other cells, so that one batch can be used for many patients.
RMAT
Regenerative Medicine Advanced Therapy designation, an FDA programme that speeds up the development of regenerative medicine. Gene therapies can qualify too.
Fig. 3 Concept diagram of Intellia's in vivo editing (AI-generated concept; it does not depict actual equipment or products).

13. References

  1. Intellia Therapeutics Form 10-K for the fiscal year ended December 31, 2025 (filed with the U.S. SEC on 26 February 2026). Business overview, clinical work on lonvo-z and nex-z, agreements with Regeneron, AvenCell and Kyverna, patents, headcount, sites and financials. https://www.sec.gov/Archives/edgar/data/1652130/000119312526076550/ntla-20251231.htm
  2. Intellia Therapeutics Form 10-Q for the quarterly period ended June 30, 2026 (filed with the U.S. SEC on 6 August 2026). HAELO Phase 3 results, the rolling BLA, lifting of the nex-z clinical holds, the HLA analysis, and first-half 2026 financials. https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm
  3. ClinicalTrials.gov NCT06634420, HAELO: a Phase 3 study evaluating NTLA-2002 in hereditary angioedema (HAE). Start date 15 January 2025, 80 enrolled, primary endpoint the time-normalised number of confirmed HAE attacks. https://clinicaltrials.gov/study/NCT06634420
  4. ClinicalTrials.gov NCT06128629, MAGNITUDE: a Phase 3 study evaluating NTLA-2001 in ATTR cardiomyopathy. Estimated enrolment 1,200, primary endpoint a composite of cardiovascular death and cardiovascular events. https://clinicaltrials.gov/study/NCT06128629
  5. ClinicalTrials.gov NCT06672237, a Phase 3 study evaluating NTLA-2001 in ATTRv-PN (MAGNITUDE-2). Estimated enrolment 60, primary endpoints mNIS+7 and serum TTR. https://clinicaltrials.gov/study/NCT06672237
  6. N Engl J Med (indexed in PubMed) Cohn DM et al., "Lonvoguran Ziclumeran - In Vivo CRISPR Gene Editing in Hereditary Angioedema," N Engl J Med 2026 (published online 13 June 2026). Results of the HAELO Phase 3 trial. https://pubmed.ncbi.nlm.nih.gov/42294842/
  7. Intellia Therapeutics Official website (used to check the company overview and the logo). https://www.intelliatx.com/

14. Claim-to-source audit

Claim in the articleCategorySource
Legal name, headquarters location (Cambridge), Nasdaq: NTLA, founded in May 2014SourcedReferences 1 and 7 https://www.sec.gov/Archives/edgar/data/1652130/000119312526076550/ntla-20251231.htm https://www.intelliatx.com/
Collaboration revenue, R&D expense, G&A expense and net loss (2024 and 2025), cash (end of 2025)SourcedReference 1 https://www.sec.gov/Archives/edgar/data/1652130/000119312526076550/ntla-20251231.htm
First-half 2026 financials, cash of US$628.4 million, net proceeds of US$194.6 million from the equity raise, and the reason for higher G&A expenseSourcedReference 2 https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm
377 employees (13 February 2026), 263 in R&D, and the January 2025 workforce reductionSourcedReferences 1 and 2 https://www.sec.gov/Archives/edgar/data/1652130/000119312526076550/ntla-20251231.htm https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm
Sites (Cambridge, Waltham, Technology Square)SourcedReference 1 https://www.sec.gov/Archives/edgar/data/1652130/000119312526076550/ntla-20251231.htm
Exclusive licence from Caribou (2014) and the rights of the University of California, the University of Vienna and Dr. CharpentierSourcedReference 1 https://www.sec.gov/Archives/edgar/data/1652130/000119312526076550/ntla-20251231.htm
The Regeneron agreements (2016; up to US$320.0 million per target; about 25% share for nex-z)SourcedReferences 1 and 2 https://www.sec.gov/Archives/edgar/data/1652130/000119312526076550/ntla-20251231.htm https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm
AvenCell (initially 33.33%, below 10% at the end of December 2025), Kyverna (KYV-201), and the terms of the Rewrite acquisitionSourcedReference 1 https://www.sec.gov/Archives/edgar/data/1652130/000119312526076550/ntla-20251231.htm
The existence of an agreement with ONK Therapeutics (content could not be confirmed)SourcedReference 2 https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm
How lonvo-z and nex-z work (in vivo editing, LNP, single dose) and their Phase 3 statusSourcedReferences 1 and 2 https://www.sec.gov/Archives/edgar/data/1652130/000119312526076550/ntla-20251231.htm https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm
HAELO Phase 3 results (80 people, 0.26 versus 2.10, an 87% relative reduction, no serious adverse events)SourcedReferences 2, 6 and 3 https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm https://pubmed.ncbi.nlm.nih.gov/42294842/ https://clinicaltrials.gov/study/NCT06634420
lonvo-z Phase 1/2 (32 people, mean kallikrein reduction of 89%, 31 attack-free)SourcedReference 1 https://www.sec.gov/Archives/edgar/data/1652130/000119312526076550/ntla-20251231.htm
nex-z Phase 1 (ATTR-CM: 36 people, 87% reduction in the 9 people at 36 months; ATTRv-PN: 33 people, 92% reduction at 24 months)SourcedReference 2 https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm
Design and enrolment status of the MAGNITUDE and MAGNITUDE-2 Phase 3 trialsSourcedReferences 2, 4 and 5 https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm https://clinicaltrials.gov/study/NCT06128629 https://clinicaltrials.gov/study/NCT06672237
The FDA clinical hold of 29 October 2025, its lifting in January and March 2026, and the patient's death (5 November)SourcedReferences 1 and 2 https://www.sec.gov/Archives/edgar/data/1652130/000119312526076550/ntla-20251231.htm https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm
Disclosure of the August 2026 HLA analysis (more than 600 samples)SourcedReference 2 https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm
Start of the rolling BLA (April 2026), the launch plan (first half of 2027), and expected completion of MAGNITUDE-2 enrolment (second half of 2026)SourcedReference 2 https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm
Interference proceedings (Broad, ToolGen, Sigma-Aldrich) and the possibility that rights are assertedSourcedReference 1 https://www.sec.gov/Archives/edgar/data/1652130/000119312526076550/ntla-20251231.htm
The three types of company (large pharma and strategic partners / genome editing and gene therapy / iPS cell and cell therapy)Our calculationThis article's own classification. Example companies are drawn from those covered in this series
Use of iPS cells, mesenchymal stem cells, and any cell therapy product of its ownNot yet confirmedReferences 1 and 2 https://www.sec.gov/Archives/edgar/data/1652130/000119312526076550/ntla-20251231.htm https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm Not stated in the 10-K or 10-Q. This article does not conclude that they do not exist either
The price and approval timing of lonvo-z, timing of nex-z Phase 3 results, and a causal link between HLA and liver enzyme elevationNot yet confirmedReference 2 https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm Not stated in the 10-Q. Figures from press reports are not used
Extension of the technology collaboration with RegeneronNot yet confirmedReference 1 https://www.sec.gov/Archives/edgar/data/1652130/000119312526076550/ntla-20251231.htm The term runs to April 2026. An extension could not be confirmed in the material we used
The reading that it is trying to avoid the burdens of ex vivo editingInferenceReference 1 https://www.sec.gov/Archives/edgar/data/1652130/000119312526076550/ntla-20251231.htm This article's interpretation
The reading that it spreads cell therapy through licensing rather than owning itInferenceReference 1 https://www.sec.gov/Archives/edgar/data/1652130/000119312526076550/ntla-20251231.htm This article's interpretation
The reading that the safety of in vivo editing is the biggest hurdleInferenceReference 2 https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm This article's interpretation
The reading that the HLA result links immune type to the safety of in vivo editingInferenceReference 2 https://www.sec.gov/Archives/edgar/data/1652130/000119312526337952/ntla-20260630.htm This article's interpretation. The company has not explained the mechanism
The reading that manufacturing comes closer to chemical manufacturing: synthesising nucleic acids, then mixing and purifying lipidsInferenceThis article's interpretation (from a materials and process viewpoint)

Last updated 1 October 2026 / Troy Technical
Every figure in this article comes from the statutory filings Intellia has made with the U.S. Securities and Exchange Commission (SEC), ClinicalTrials.gov and peer-reviewed papers. No research-firm estimates or press-based figures have been used. Passages marked "Inference" are this article's interpretation of the primary sources, not statements made by the company. This article summarises a company's activities; it does not recommend any treatment and is not medical advice.

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