Intellia's gene edit inside the body still works 4 years on, as the FDA weighs the first approval of its kind by 10 March

The stories

11 on this beat today.

Every story told the same way: what happened, who is involved, why it matters, how it works, the truth and the good news.

1. 01

   Intellia's gene edit inside the body still works 4 years on, as the FDA weighs the first approval of its kind by 10 March

   4 years

   that lonvo-z has kept working since treatment

   the data come from its maker, Intellia Therapeutics [1]

   10 March

   when the FDA is expected to decide on lonvo-z

   a yes would make it the first CRISPR medicine given inside the body [1]

   64

   people given Casgevy, the only approved CRISPR treatment, in 2025

   up from 5 in the second half of 2024 [2]

   What happened

   • Intellia Therapeutics says its gene-editing treatment lonvo-z still works 4 years after patients received it. [1]
   • Lonvo-z is for one form of hereditary angioedema, an inherited illness that causes painful, sometimes dangerous swelling. [1]
   • Intellia has asked the FDA, the US drug regulator, to approve it, and a decision is expected by 10 March. [1]
   • If approved, it would be the first CRISPR medicine given directly inside the body. [1] Chief executive John Leonard told investors so on 12 November. [1]
   • Lonvo-z uses CRISPR, a gene-editing tool, to switch off one gene in the liver. [1]
   • That gene makes prekallikrein, a protein made in the liver that helps blood vessels leak. [1]
   • Fatty particles carry the editing kit through the blood to the liver, in a procedure that takes one day. [1]

   Who is involved

   Jonathan Bernstein, a University of Cincinnati specialist in the illness, works on a lonvo-z trial. [1]

   Of the four-year data, he said: "It's very reassuring." [1]

   • The FDA the US drug regulator; it is expected to decide on lonvo-z by 10 March
   • Jonathan Bernstein a swelling-illness specialist at the University of Cincinnati who works on a lonvo-z trial; he called the data reassuring
   • Intellia Therapeutics a US gene-editing company in Cambridge, Massachusetts; it made lonvo-z and asked the FDA to approve it

   How it unfolded

   Last year the FDA paused 2 other Intellia trials of editing inside the body. [1]

   A man in one of them, older and with other illnesses, had liver damage and died. [1]

   The FDA lifted the pause this year, after requiring added safety steps. [1]

   1. 12 Nov Intellia's chief tells investors lonvo-z could be the first in-body CRISPR medicine [1]
   2. 10 Mar FDA decision due
   3. Last year 2 trials paused after a death
   4. This year pause lifted, with safety steps
   5. Last year the FDA pauses 2 other Intellia trials after a patient dies [1]
   6. This year the FDA lifts the pause, with added safety steps [1]
   7. By 10 Mar the FDA's decision is expected [1]

   Why it matters

   People with the illness juggle medicines for life to prevent and treat swelling attacks, Bernstein says. [1]

   A gene edit done in one day could offer them relief from that, he says. [1]

   Think it through

   1. 1

      Why does editing inside the body matter?

      The only CRISPR treatment the FDA has approved, Casgevy, edits blood cells outside the body and puts them back. [1] Patients first need harsh chemotherapy. [1]

      Casgevycells edited outside

      vsvs

      Lonvo-zedited inside
   2. 2

      Has the outside method held Casgevy back?

      Each Casgevy dose is made anew for each patient, which adds time and money to every treatment. [1] Only 5 people got it in the second half of 2024. [2]

      Cells taken out→Edited for one person→Put back after chemotherapy
   3. 3

      Has it picked up since?

      In 2025, 64 people got Casgevy, bringing in $116 million. [2]

      5 peopleSecond half of 2024

      ↓→

      64 people2025
   4. 4

      How many gene treatments are there for rare diseases?

      Of 30 US-approved gene therapies, 24 treat at least one rare disease, covering 16 conditions, a review found. [3]

      24 of 30

      approved gene therapies that treat a rare disease

   5. 5

      So what is the catch with editing inside?

      Inside the body, doctors cannot screen out cells edited in the wrong place. [1] The body may also react badly to the treatment. [1]

      Edit inside the body→No check on each cell→Wrong edits cannot be removed
   6. 6

      So what is really going on?

      The FDA's answer by 10 March decides if lonvo-z becomes the first approved in-body CRISPR medicine. [1] Intellia brings 4 years of data, and a death in another trial. [1]

      4 years of dataIntellia

      ↓→

      decides by 10 MarchThe FDA

   How it works

   Geneticists call switching off a gene a knockout: the cell can no longer make what that gene codes for.

   With its prekallikrein gene switched off, the liver stops making the protein that helps vessels leak. [1]

   Seen it before

   Turning off the main water tap stops every leak in a house, instead of mopping each puddle.

   The truth

   • The four-year data were released by Intellia itself, the company asking to sell the treatment. [1]
   • Nobody knows yet

     How many swelling attacks lonvo-z prevents after four years, and in how many people.

     The report we could read says only that it continues to be effective; the counts are in Intellia's own release and the paywalled part of the article. [1]

   • Nobody knows yet

     What price Intellia will set for lonvo-z.

     No price appears in the reporting we read, and the FDA decision is not due until 10 March. [1]

   Good news

   • A gene edit given in a single day for an inherited swelling illness was still working four years later.

1. 02

   Brain implants give ALS patients a voice

   What happened

   • Brain implants let a 46-year-old man who lost his voice to ALS speak clearly, the New York Times reports. [4]
   • ALS is a disease that can take away control of the muscles used to speak. [4]
   • Researchers at the University of California, Davis decoded his brain signals and turned them straight into speech. [4]
   • A 58-year-old trained singer whose voice ALS had silenced was able to sing a musical scale. [4]

   Who is involved

   • A man, 46 spoke again
   • A woman, 58 sang a scale

   How it unfolded

   Older systems turned brain signals into text, then into speech, so people spoke after a delay. [4]

   Why it matters

   The work shows these implants moving from one-off demonstrations toward everyday use, the New York Times reports. [4]

   Cindy Chestek, a University of Michigan engineer not involved in the work, called it "good enough for real communication." [4]

   Think it through

   1. 1

      Why did the old delay matter?

      Users watched words appear one by one, then clicked to send, which made real conversation hard. [4]

   2. 2

      So is it ready for patients?

      So far the report describes 2 people with experimental implants. [4]

      2 of 2

      people in the report so far

   How it works

   Engineers call this a brain-computer interface: a link that turns brain signals into actions. [4]

   Electrodes in the brain pick up signals as the person tries to speak, and software turns them into speech sounds. [4]

   Seen it before

   A live interpreter speaks while you talk, instead of waiting for you to write it all down.

   Good news

   • After singing her scale, the former singer cried tears of joy. [4]

   What comes next

   The team will present the work next week at the Society for Neuroscience meeting in Washington. [4]

1. 03

   First NHS patient gets a drug that delays type 1 diabetes

   What happened

   • Annette Quilter, a GP from Shropshire, became the first UK patient to get teplizumab on the NHS. [5]
   • Teplizumab is a drug that can delay type 1 diabetes while it is still at an early stage. [5]
   • Type 1 diabetes starts when the body's own immune system attacks the cells that make insulin. [5]
   • Trials show the drug can halve the rate at which people come to need insulin. [5]
   • Treatment is one 30-minute drip a day for 14 days in a row. [5]

   Who is involved

   Quilter, who is 42, was found to have early type 1 diabetes this summer, and screening showed she was eligible. [5]

   • Annette Quilter GP, 42; first NHS patient

   How it unfolded

   The NHS has offered it since June 2026 to eligible adults and children over 8 with stage 2 disease. [5]

   1. June 2026 the NHS starts offering it
   2. This week the first patient is treated

   Why it matters

   She said having time to prepare before starting insulin "means a great deal". [5]

   Think it through

   1. 1

      Why must it be caught so early?

      It slows the attack on insulin-making cells, and the NHS gives it at stage 2, before insulin is needed. [5]

      Screening finds stage 2→The drug slows the attack→Insulin needed later
   2. 2

      So does it cure type 1 diabetes?

      No: it delays the need for insulin, giving people more time before injections or a pump. [5]

      A cureno

      vsvs

      A delayyes

   How it works

   Immunologists call this autoimmunity: the body's defences attack its own healthy cells.

   In type 1 diabetes the attack falls on insulin-making cells in the pancreas, and teplizumab slows it. [5]

   Seen it before

   Slowing a leak in a bucket does not stop it emptying, but it buys time before it runs dry.

   Why some medicines change the course of an illness and others only ease it

   Pharmacologists sort medicines by whether they act on what causes an illness or only on the harm it does, and the two are proved differently.

   The twist

   A medicine that acts on the cause can only save what is still there, which is why teplizumab is given at stage 2, before a person needs insulin.

   The picture

   Insulinreplaces the hormone

   vsvs

   Teplizumabslows the attack

   Step by step

   1. 1 An illness has a cause, and the harm the cause does
   2. 2 Easing medicines block the harm or replace what is lost
   3. 3 So they are taken again and again, often for life
   4. 4 Course-changing medicines act on the cause itself
   5. 5 So they must come early, while there is still something to save
   6. 6 Proof takes years of watching the illness, not one blood number

   The same force, elsewhere today

   Where this chain is also running, in today's other stories.

   • The first NHS patient on teplizumab

     Insulin replaces the hormone the body has lost, while teplizumab slows the immune attack on the cells that make it.

   • Betaine for a rare Finnish brain disease

     Betaine aims at the cause by helping the faulty enzyme fold, but the trial could only show a lower waste marker, not a slower disease.

   • The new advice on blood fats

     Drugs can cut the triglyceride number, but no trial has yet shown fewer heart attacks, so a lower reading is not yet a changed course.

   • The review of gene therapy studies

     Most rare diseases still have no medicine that changes the illness, so families rely on treatments that only ease it.

   Where you've seen this

   A leaking roof

   buckets catch each drip, and only fixing the tile stops new ones

   Pests on a farm

   spraying kills this year's bugs, and changing where they breed stops next year's

   A school before exams

   practice tests lift this term's scores, and better teaching changes what pupils know

   The catch

   Easing medicines are not second best: for most rare diseases nothing yet reaches the cause, and insulin keeps people with type 1 diabetes alive.

   And the whole of it

   A family with a rare illness, a GP waiting to start insulin and a regulator reading four years of data each see one part of this. Whether any of us gets a medicine that changes an illness depends on how early we were tested and how far the science has come.

   Good news

   • A University of Birmingham researcher called it the first treatment that can delay type 1 diabetes needing insulin. [5]

1. 04

   A new drug beats chemotherapy for food-pipe cancer

   What happened

   • A drug called iza-bren beat chemotherapy against advanced food-pipe cancer in a 497-patient trial, the journal JAMA reports. [6]
   • Iza-bren is an antibody joined to a cancer-killing chemical, built to grip 2 proteins on cancer cells. [6]
   • Every patient had cancer that kept growing after the standard first treatment. [6]
   • The middle patient on iza-bren lived 9.79 months, against 7.2 months on chemotherapy. [6]
   • The middle patient went 4.17 months before the cancer grew or they died, against 1.97 on chemotherapy. [6]
   • The trial ran in 80 hospitals in China. [6]

   Iza-bren9.79 months

   Chemotherapy7.2 months

   Why it matters

   Patients whose food-pipe cancer grows after first treatment have lacked a treatment that works well, the authors write. [6]

   Think it through

   1. 1

      What did the extra months cost patients?

      Severe side effects from treatment hit 85.1% of patients on iza-bren and 59.3% on chemotherapy, mostly in the blood. [6]

      Iza-bren85.1%

      Chemotherapy59.3%
   2. 2

      Does the result reach patients elsewhere?

      The authors back iza-bren as a new second treatment for patients in China, where the trial ran. [6]

   How it works

   Cancer doctors call this an antibody-drug conjugate: an antibody that carries a drug to cells it recognises. [6]

   Iza-bren's antibody grips 2 growth-signal proteins on the cancer cells and brings its chemical with it. [6]

   Seen it before

   A courier with the right address brings a parcel to one house, not to the whole street.

   Good news

   • In a trial across 80 hospitals in China, people with advanced food-pipe cancer lived longer on a new drug than on chemotherapy.
2. 05

   Oura buys an AI doctor service for $625 million

   What happened

   • Oura, the maker of a wearable ring, will pay $625 million in cash for Counsel Health. [10]
   • Counsel makes a chatbot people can ask about health worries before a clinician treats them online. [8]
   • Counsel had disclosed $36 million in funding before the deal. [8]
   • Oura's sales rose 153% to $533 million in the three months to 30 September. [7]

   How it unfolded

   Oura delayed plans to sell its shares to the public just over a month ago, citing market uncertainty. [8]

   Why it matters

   Counsel's clinicians will get the data Oura's ring collects, to help diagnose and treat customers. [9]

   Think it through

   1. 1

      Why is Eli Lilly in this story?

      Lilly, which makes weight-loss drugs, lists Counsel as a provider for people seeking a prescription for them. [9]

   2. 2

      Why does that tie matter?

      Drugmakers increasingly promote online prescribers of these drugs, and STAT says this deal is expected to complicate one such tie. [9]

      Lilly lists Counsel→Counsel prescribes→Oura now owns Counsel

   How it works

   Economists call it vertical integration: one company owning several steps from product to customer.

   Oura already sells the ring that collects health data. [9] Now it will own the service whose clinicians act on it. [9]

   Seen it before

   A gym that buys a physio clinic can send its own members straight to its own physios.

   The truth

   • Nobody knows yet

     What Eli Lilly's listing of Counsel Health involves, now that Oura is buying Counsel.

     STAT reports the tie is growing more complicated, but the terms are behind its paywall and in nothing else we could read. [9]

3. 06

   An old medicine tested on a rare Finnish brain disease

   What happened

   • Betaine, a medicine, cut a waste marker by 68.6% in children with a rare brain disease in a 48-month trial. [11]
   • The disease, aspartylglucosaminuria or AGU, slowly harms thinking from early childhood. [11]
   • Most known patients are Finnish, and most of them carry the same gene fault. [11]
   • In AGU a faulty enzyme fails, and waste called glycoasparagines builds up. [11]
   • Doctors gave 25 children aged 3 to 17 betaine by mouth, and analysed 21 of them. [11]
   • Betaine is already approved, as Cystadane, for another inherited illness called homocystinuria. [11]

   Why it matters

   If betaine slows AGU, children could keep more of their thinking skills, which barely grow after age 7. [11]

   Think it through

   1. 1

      Why test a drug made for another illness?

      Many homocystinuria patients have taken betaine over the years, and this trial found it safe in AGU too. [11]

      Homocystinurialong used

      ↓→

      AGUsafe in this trial
   2. 2

      So did the children's thinking improve?

      Unknown: the trial measured waste in urine, and the authors call for bigger, longer studies of the disease's course. [11]

      Measuredurine waste

      vsvs

      Not yet shownslower disease

   How it works

   Biochemists call this a pharmacological chaperone: a small drug that helps a faulty enzyme fold so it can work. [11]

   Betaine binds the faulty Finnish enzyme and helps it fold, raising its activity in patients' cells in the lab. [11]

   Seen it before

   A splint holds a weak finger straight so it can still do its job.

   Betaine aims at the cause by helping the faulty enzyme fold, but the trial could only show a lower waste marker, not a slower disease.

   The idea, in story 03: Why some medicines change the course of an illness and others only ease it →

   The truth

   • The trial was open-label, so doctors and families knew the children were taking betaine. [11]
4. 07

   Nearly half of gene therapy studies aim at rare diseases

   What happened

   • A review of 1,464 registered gene therapy studies found 646, or 44%, aimed at rare diseases. [3]
   • Those rare-disease studies covered 175 different rare diseases. [3]
   • New rare-disease studies rose from 21 a year in 2015 to 73 in 2024. [3]
   • Haemophilia, an inherited eye disease, Duchenne muscular dystrophy and sickle cell were studied most. [3]
   • About 63% of the rare-disease studies were at the earliest stages of testing. [3]

   Why it matters

   Most rare diseases still lack a treatment that changes the illness itself, the authors write. [3]

   Think it through

   1. 1

      Why so many studies and so few medicines?

      Most studies are early-stage, and the authors say later-stage trials are now the priority. [3]

      Early studies→Later-stage trials→Approval
   2. 2

      Who runs most of the trials?

      The US is a site in 410 of the 646 rare-disease studies, and the authors want more countries taking part. [3]

      410of 646 rare-disease studies with a US site

   How it works

   Researchers call this a cross-sectional study: one snapshot of every record on one date.

   This snapshot froze the trial records in October 2025, so it counts studies started, not their results. [3]

   Seen it before

   A class photo shows who was in the class that day, not how anyone's year went.

   Most rare diseases still have no medicine that changes the illness, so families rely on treatments that only ease it.

   The idea, in story 03: Why some medicines change the course of an illness and others only ease it →

   What comes next

   The review, in the journal Gene Therapy, was published on 13 November. [3]

5. 08

   Experts: high blood fats flag heart risk

   What happened

   • Heart experts from the International Atherosclerosis Society issued new advice on high triglycerides, a kind of fat in the blood. [12]
   • About 29% of adults worldwide have high triglycerides, a 2025 review of over 200 studies estimated. [12]
   • The rate is 34% in men and 25% in women, and rising as obesity and diabetes spread. [12]
   • The harm to the heart comes from cholesterol inside leftover fat particles, not from triglycerides themselves, the group writes. [12]
   • So far, no trial has shown that drugs aimed at triglycerides cut heart attacks or strokes, it says. [12]
   • High triglycerides are often missed and left untreated, though a simple blood test finds them. [12]

   Why it matters

   A high reading should push doctors to tackle a patient's other heart risks, the experts say. [12]

   Think it through

   1. 1

      Why lower triglycerides at all, then?

      High levels can also cause fatty liver and sudden pancreatitis, an inflamed pancreas, the experts note. [12]

      High triglycerides→Fatty liver→Pancreatitis
   2. 2

      So which other risks should doctors check?

      The four standard ones are diabetes, high cholesterol, high blood pressure and smoking, the statement lists. [12]

      Diabetessugar

      ++

      Cholesterolfats

      ++

      Blood pressurepressure

      ++

      Smokingsmoke

   How it works

   Doctors call a reading like this a marker: it flags danger without causing the harm itself. [12]

   Triglycerides travel in the same leftover particles whose cholesterol does the damage, so a high reading flags it. [12]

   Seen it before

   Smoke warns of a fire, but fanning the smoke away does not put out the pan.

   Drugs can cut the triglyceride number, but no trial has yet shown fewer heart attacks, so a lower reading is not yet a changed course.

   The idea, in story 03: Why some medicines change the course of an illness and others only ease it →
6. 09

   Marea's antibody cuts blood fats by 72%

   What happened

   • An antibody from Marea Therapeutics cut triglycerides by 72.2% in patients with very high levels, in a mid-stage trial. [13]
   • Patients who started at a lower level, still high, saw a 57.2% fall. [13]
   • More than half of 235 treated adults reached normal levels, against 3.4% on a dummy drug. [13]
   • Side effects were slightly less common on the drug than on the dummy, 69.5% against 77.6%. [13]

   How it unfolded

   Marea became part of Lisata Therapeutics, a cancer drugmaker, in a September deal. [13]

   Why it matters

   Statins and weight-loss drugs lower triglycerides only modestly, leaving patients at risk of pancreatitis, chief executive Josh Lehrer says. [13]

   Think it through

   1. 1

      What do the heart experts say about such drugs?

      A new expert statement calls some new triglyceride drugs promising, but says no trial yet shows fewer heart attacks. [12]

      Promiselower blood fats

      vsvs

      Proofnot yet on hearts
   2. 2

      So who is Marea aiming at first?

      People at risk of pancreatitis, Lehrer says, while the company weighs obesity later. [13]

      Very high triglycerides→Pancreatitis risk→Marea's first patients

   How it works

   Drug developers call this a phase 2b trial: a mid-sized test of dose and effect before the final, biggest trial. [13]

   Marea plans that final, phase 3 trial for a longer-lasting version, MAR005, in people with severe levels. [13]

   Seen it before

   A cook tries a new recipe on a few friends before making it for the whole party.

   The truth

   • What is really going on Two other results this week moved a number before showing a changed illness: betaine cut a waste marker in urine, and Marea's antibody cut a blood fat. [11][13]

   Good news

   • More than half of 235 adults with high blood fats reached normal levels on a new antibody, against 3.4% on a dummy drug.

   What comes next

   A second part of the trial now compares the drug with a dummy in 100 patients. [13]

7. 10

   Vor pays a Chinese drugmaker for a long-lasting drug

   What happened

   • Vor Bio, a US biotech, will pay RemeGen, a Chinese drugmaker, $10 million in cash for rights to a drug. [14]
   • The rights cover the drug, RC298, everywhere outside Greater China. [14]
   • Vor also gives RemeGen share warrants worth about $46 million, and up to $300 million if set goals are met. [14]
   • RC298 is designed to block 2 signals, BAFF and APRIL, that help immune cells which attack the body's own tissue. [14]

   Cash now$10 million

   Share warrants$46 million

   Milestones, at most$300 million

   How it unfolded

   Last year Vor licensed a similar drug, telitacicept, from RemeGen for $45 million up front. [14]

   That deal came a month after Vor laid off 95% of its staff. [14]

   Why it matters

   Telitacicept is injected weekly, and a longer-lasting drug could reach more autoimmune diseases, Fierce Biotech notes. [14]

   Think it through

   1. 1

      Why so little cash up front?

      Most of the value is in shares, and in payments due only if RC298 clears set steps. [14]

      $10 million now→Shares→Payments if goals are met
   2. 2

      What does Vor need from this?

      Vor rebuilt itself around telitacicept, now in final-stage trials for 2 autoimmune diseases. [14]

      2 of 2

      diseases in final-stage trials

   How it works

   Dealmakers call this licensing out: a company sells rights to its drug abroad but keeps its home market.

   RemeGen keeps Greater China and runs the first human tests, and Vor takes later trials and sales elsewhere. [14]

   Seen it before

   An author can sell a book's foreign rights and keep selling it at home.

   The truth

   • Nobody knows yet

     How long RC298 lasts between doses.

     Vor has not yet reported a dosing interval, only that the drug is designed to last longer than the weekly telitacicept. [14]

   What comes next

   Vor expects RemeGen to apply in China to start human tests in the first half of 2027. [14]

8. 11

   Europe clears a swab test for 13 germs

   What happened

   • Cepheid, a Danaher company, won European approval for a test that checks one swab for 13 germs. [15]
   • The germs cause breathing infections, and they include both viruses and bacteria. [15]
   • Many of these infections look alike, with fever, cough and sore throat. [15]
   • In a typical season flu causes up to 50 million cases with symptoms in Europe, the company says. [15]
   • Flu also kills 15,000 to 70,000 people in Europe a year, it says. [15]

   Why it matters

   Knowing the germ can support treatment choices and responsible antibiotic use, Cepheid says. [15]

   Think it through

   1. 1

      Who gains most from knowing the germ?

      Children, older adults and people with weak immune systems face more risk of complications, the company says. [15]

   2. 2

      Is this independent evidence?

      No: these figures come from Cepheid's own announcement, which cites outside studies. [15]

   How it works

   Lab scientists call this multiplex PCR: copying the genes of many possible germs from one sample at once. [15]

   Cepheid's panel looks for 13 germs in one swab, because symptoms alone do not show which germ it is. [15]

   Seen it before

   Checking the whole class register at once is quicker than calling one name at a time.