back to article $2.8m gene therapy treatment is America's most expensive drug ever

A one-time gene therapy treatment recently approved by America's FDA is gaining attention not just for its clinical value, but for its price: at $2.8 million, Bluebird Bio's Zynteglo is the most expensive drug in US history. Zynteglo is a treatment designed to tackle the rare condition known as transfusion-dependent beta- …

  1. Brian Miller

    The ODIN, anyone?

    So if this doesn't work then the insurance will only be paying $560,000 for it, and I'll bet that Bluebird Bio will still be making a profit off the failed treatment.

    I really hope that things like this in the future can be handled with something like The ODIN kit and public AI.

    1. Dave314159ggggdffsdds Silver badge

      Re: The ODIN, anyone?

      "I'll bet that Bluebird Bio will still be making a profit off the failed treatment."

      That says something about you and your fact-free prejudices. Obviously it's completely untrue. If this drug doesn't work as advertised, Bluebird Bio will lose all the money they've invested in it.

      The simple reality for those without your mindset is simply that drugs cost an absolute fortune to develop, and those with few patients to be treated have to have a high price per treatment. The US system of funding such treatments is as barmy as ever.

      1. Anonymous Coward
        Anonymous Coward

        Re: The ODIN, anyone?

        > If this drug doesn't work as advertised, Bluebird Bio will lose all the money they've invested in it.

        You seem to be confusing "a treatment didn't work" with "the drug doesn't work".

        No drug or treatment has a 100% efficacy rate. What the commenter seems to have been talking about was the individual cases where a treatment of an otherwise effective drug fails to have the expected effect.

        It does feel rather skeevy to profit from a failed treatment, as it produces perverse incentives (eg. one can get peopel to buy even high-priced placebos if you spend enough on marketing), however such a profit in this case seems to be speculation on the part of the commenter.

  2. An_Old_Dog Silver badge
    Holmes

    Non-affordable for the non-rich

    No insurance affordable by someone not independently-rich will pay out for this. As to being cheaper in the long run than paying for transfusions, insurance companies' actuaries may conclude it's cheaper to pay for some tranfusions, knowing some (or many?) transfusion-receiving patients will die sooner than will die later.

    Further, most "major medical" health insurance policies pay only 80% of treatment cost. The remaining 20% of that $2.8M, which the patient will pay, is $560,000.00.*

    As to "discounts" ... we'll see.

    *People living in countries with socialized medicine may possibly be in a better situation.

    1. Snowy Silver badge
      Holmes

      Re: Non-affordable for the non-rich

      N.I.C.E is never going to say yes to this.

      1. gandalfcn Silver badge

        Re: Non-affordable for the non-rich

        Not being Septic they could probably get it far cheaper.

      2. Brewster's Angle Grinder Silver badge

        Re: Non-affordable for the non-rich

        Why not? Here's a gene therapy they approved in Feb. The list price there was £2.6m, although there seems to have been some behind-the-scenes haggling about the price before it got approved. But then their job is to ensure we are getting good value for money and are not being ripped off.

    2. DS999 Silver badge

      Re: Non-affordable for the non-rich

      Health insurance plans have a copay like 20% or whatever, but only up to a certain yearly maximum which is usually in the $5000 to $10,000 range. If the therapy was covered by insurance that's what the patient would pay, the insurance would pay the rest. Plans used to have a lifetime maximum benefit limit in the $1 to $2 million range, but the ACA banned those.

      I imagine very few insurance companies will approve this treatment. While theoretically paying $2.8 million is better than paying $6.4 million for lifetime treatment there are two issues with that math. One is that the present value of $6.4 million in treatments over someone's entire life is probably much less than $2.8 million, the other is that few people will remain on the same insurance policy their whole life (and they'll definitely be off it when they're eligible for Medicare at age 67 or whatever)

      So from the perspective of an insurance company, they would much rather keep paying for transfusions since they will spend less that way unless you get on their insurance with a new job at age 18 and keep working there until you retire.

      Countries on socialized medicine may not fare any better. When there are only one or two treatments that cost $2 million and only a few hundred or few thousand people need it, that's one thing. What happens when they have a $1 million treatment to cure diabetes, or that is guaranteed to prevent Alzheimer's so long as you are treated before any symptoms show up and you don't know if you will ever even get it? The sheer number of people who would need/want such treatment will break the bank.

      1. jmch Silver badge

        Re: Non-affordable for the non-rich

        "What happens when they have a $1 million treatment to cure diabetes, or that is guaranteed to prevent Alzheimer's so long as you are treated before any symptoms show up and you don't know if you will ever even get it? The sheer number of people who would need/want such treatment will break the bank."

        The prohibitive cost is, I guess, dependent on having both a very large research expense combines with a very small pool of potential patients. I very much doubt the raw materials + labour going into creating and administering an individual dose or course of treatment would be much higher than a few ks.

        Any treatment that has to be administered to millions of patients will see it's unit cost drop dramatically.

        Incidentally, while not exactly a 'cure' for diabetes, it's extremely easy to prevent - just cut out the huge amounts of processed carbohydrates from the diet and replace most with fresh vegetables. Of course there is also a cost to that, since fresh veg costs much more than bread, pasta etc, which is why obesity and diabetes correlate strongly with lower incomes.

        Maybe if governments are really interested in their population's health they would start subsidising vegetable production instead of subsidising wheat, corn etc

        1. DS999 Silver badge

          Re: Non-affordable for the non-rich

          Any treatment that has to be administered to millions of patients will see it's unit cost drop dramatically.

          Eventually, but not at first. They'll price it high to capture rich people and those with gold plated health insurance, then slowly lower it over time. It'll be many years before it becomes even remotely reasonable.

          That's how you maximize income, so that's what they'll do. They don't care about improving people's health, they care about making as much money as possible.

          1. Dave314159ggggdffsdds Silver badge

            Re: Non-affordable for the non-rich

            More fact free ranting from the funny mindset mob.

            1. Swarthy
              Facepalm

              Re: Non-affordable for the non-rich

              And yet another content-free ad hominem.

        2. Twanky

          Re: Non-affordable for the non-rich

          while not exactly a 'cure' for diabetes, it's extremely easy to prevent

          Partly agree: Type 2 should certainly be managed this way while there is some insulin still being produced by the patient. Type 1 is different - especially in the young. Fixing Type 1 would be amazing, but as I say below, I don't think the industry would be interested in a cure. They'd prefer a new expensive treatment for the rest of the patient's (long) life.

      2. Anonymous Coward
        Anonymous Coward

        Re: Non-affordable for the non-rich

        I would argue that a $1M cure for diabetes or Alzheimers would have massive demand among countries with socialised medicine.

        The UK's NHS spends an estimated £10bn a year on diabetes, but the overall cost to the UK economy is estimated at £23bn a year.

        The difference with a socialised medicine system is the latter figure is the important one, not the former, the government cares about the overall cost, not just medical.

        Certainly the business case would probably be positive for young children with diabetes, the overall cost to the country for a 12 year old with accompanying complications would be way more than $1M over their lifetime.

      3. Twanky

        Re: Non-affordable for the non-rich

        What happens when they have a $1 million treatment to cure diabetes

        It depends who you mean by 'they'. Academics would love to research it and get their research published. They might even want to benefit humanity by defeating a medical scourge. However, the big pharmaceutical companies would have little to no interest in pursuing any breakthrough; they don't want to cure people. They want to keep treating them year after year. Given that many academics are funded by big industry they won't get funding to research something their paymasters are not interested in.

      4. Dave314159ggggdffsdds Silver badge

        Re: Non-affordable for the non-rich

        "the present value of $6.4 million in treatments over someone's entire life is probably much less than $2.8 million"

        Whut? No, obviously not even close. It's far higher than $2.8m at NPV.

        "the other is that few people will remain on the same insurance policy their whole life"

        You can't switch an ongoing claim!

        "What happens when they have a $1 million treatment to cure diabetes, or that is guaranteed to prevent Alzheimer's so long as you are treated before any symptoms show up and you don't know if you will ever even get it? The sheer number of people who would need/want such treatment will break the bank."

        That can't happen. The 'sheer number of people' means that the cost per dose will be low instead of high. The cost of developing drugs tends to be fairly similar, whether the drug turns out to have 5 applications or 5 billion. Obviously the drugs that are used by many, many patients are far cheaper per dose.

        1. DS999 Silver badge

          Re: Non-affordable for the non-rich

          Whut? No, obviously not even close. It's far higher than $2.8m at NPV

          How do you figure that? I'm assuming they calculated $6.4 million as "$100K a year for 64 years" or similar. Go check out the NPV of that and you'll see it is less than $2.8 million.

          You can't switch an ongoing claim!

          You think anyone who has this condition and requires transfusions every couple weeks is stuck with the same insurance company forever? That may have been true prior to the ACA, when providers were allowed to deny coverage for pre-existing conditions, but that's no longer the case. Someone with this condition might be on their parent's insurance, then get new insurance when they get a job after college, then switch jobs multiple times during their career and have different insurance each time.

          So why would any of those insurance providers want to pay $2.8 million, when they could get off much cheaper paying the $100K a year or whatever for transfusions knowing that he probably will be off their insurance in a few years or a few decades at worst will add up to less?

          1. Dave314159ggggdffsdds Silver badge

            Re: Non-affordable for the non-rich

            "I'm assuming they calculated $6.4 million as "$100K a year for 64 years" or similar."

            They explicitly stated otherwise. Life expectancy here is <30 years. In fact, now I think about it properly, the $6.4m figure is probably at NPV to start with, so we don't need any adjustments.

            "You think anyone who has this condition and requires transfusions every couple weeks is stuck with the same insurance company forever? "

            No, that _claim_ is. Why do you think other providers are willing to take you on, in that situation? The original insurer continues to pay existing claims.

            "So why would any of those insurance providers want to pay $2.8 million, when they could get off much cheaper paying the $100K a year or whatever for transfusions knowing that he probably will be off their insurance in a few years or a few decades at worst will add up to less?"

            Because you're inventing numbers and getting the maths wrong. They have told you it is cheaper the other way around and that's what they're going to do, but you prefer this weird conspiracy theory of yours even when it conflicts with basic arithmetic, the law on corporate manslaughter, and so-on.

      5. Anonymous Coward
        Anonymous Coward

        Re: Non-affordable for the non-rich

        The thing is, you can't really use US prices to understand what such a treatment would cost in any other country, because the US health insurance system is so bonkers. Prices quoted by and to insurance companies can to be 10-100 times the price an individual might shell out if they paid out of pocket.

    3. tmTM

      Re: Non-affordable for the non-rich

      With such a small number of patients who exactly is going to buy it if the insurance company won't? Maybe a handful of rich folk, probably not even 10.

      Cost of bringing a drug to market can be easily $1bn, making a paltry $20m in sales is a pathetic return on that investment.

      1. Anonymous Coward
        Anonymous Coward

        @tmTM - Re: Non-affordable for the non-rich

        Yep, they have a lot of people to bribe.

    4. Anonymous Coward
      Anonymous Coward

      Re: Non-affordable for the non-rich

      Would health insurers even touch your case if they knew you had this disease anyway? As it's a genetic disorder, I'd expect it to be a known pre-existing condition at least. Do they pay out for those at all?

      1. Dave314159ggggdffsdds Silver badge

        Re: Non-affordable for the non-rich

        The way the US health insurance system is set up is bizarre. The answer isn't simple. The simplest way someone could be covered, though, is to be born to a parent who already has insurance that covers their kids.

    5. martinusher Silver badge

      Re: Non-affordable for the non-rich

      Insurance has a couple of tricks up its collective sleeve. One is that you have to get most treatments 'pre-approved' before insurance companies will pay for it. The other is that the insurance companies will simply deem the treatment 'experimental' and decline to cover it. (This little trick is what upends a lot of chemotherapy patients financially.) US insurance companies have their own versions of NICE; our version is proprietary and confidential so you don't get to understand what will get covered and why -- if coverage is declined you can appeal and so on but the chances are you'll die before the insurance company does.

      The goal with these therapies is to try to find a treatment that's covered under Medicare. The government not only has deep pockets but is also 'flexible' -- there's an art and a science to making it bend to your will and its remarkably sophisticated. Medicare also has the twist that it is forbidden by law for bargaining for discounts on drug pricing (thank you, Congress -- "your flexible friends"), its obliged to pay 'list' price, which as you can see is just about anything you can think of.

    6. codejunky Silver badge

      Re: Non-affordable for the non-rich

      @An_Old_Dog

      "*People living in countries with socialized medicine may possibly be in a better situation."

      People with socialized medicine will probably not have the option as the socialised medicine provider will consider it too expensive and so nobody will be able to access it without travelling to the US.

  3. Jellied Eel Silver badge

    Ethical minefield, and maybe eugenics wasn't so last century?

    So never really having heard much about this disease, other than vaguely recollecting thalassemia, I hit da Wiki for a quick overview-

    https://en.wikipedia.org/wiki/Beta_thalassemia

    Beta thalassemia is a hereditary disease allowing for a preventative treatment by carrier screening and prenatal diagnosis.

    So it's avoidable. Assuming you can get that screening. If both genetic donors are screened prior to conception. If screening's done after conception, then there might be an option to avoid future suffering via a termination. If that's available. I think there's been something in the news about that recently.

    Or, perhaps we could screen everyone and give them a social credit score based on whether they carry a risk. We could combine that with Covid-style proximity warnings that flash red, if there's a risk of passing that on. Or, because Big Tech has been hoovering people's medical data, they may know you're a risk and decline cover.

    Or, perhaps we could use the same techniques to fix the genes of carriers, preventing the problem arising. But if that'd cost $2.8m a shot, it would be a hard sell as preventative medicine. I suspect ethics and politics wrt stem cell research & treatment might also come up. Plus if you can fix 'bad' genes, why not improve 'good' ones?

    Or we could look at how much these treatments actually cost, and regulate on a cost+ basis. There are obvious humane considerations around avoiding painful or distressing treatments that may or may not prevent early deaths though.

    1. jmch Silver badge

      Re: Ethical minefield, and maybe eugenics wasn't so last century?

      Seen Gattaca, have you?

      1. Jellied Eel Silver badge

        Re: Ethical minefield, and maybe eugenics wasn't so last century?

        Seen Gattaca, have you?

        Yup, also read Plato. Or it's just been a social/medical issue for a very long time, especially now genetic modification is getting ever easier. But like I said in the original post, it touches on a whole range of issues.

        Also curious why the Neros with their opposing thumbs. I guess they think that eradicating diseases is a bad idea? Especially after we've just persuaded a fair chunk of humanity to allow experimentation on their mitochondria. Consequences of that little experiment are slowly coming to light. And meanwhile, in other news, there's another disease that's highlighting simple messaging about safe sex and avoiding STDs isn't working.

        1. jmch Silver badge

          Re: Ethical minefield, and maybe eugenics wasn't so last century?

          "Neros with their opposing thumbs"

          not me, but I guess maybe related to the social credit score thingy?

          Otherwise you raise some very relevant points - being able to screen for genetic hereditary diseases by parents pre-conception is a very good idea. The problem is when there are a host of other characteristics that can be chosen and filtered on, without a full understanding of the complexity behind it all.

          The reason I referenced Gattaca is that it celebrates the human spirit, that determination and drive can overcome genetic disabilities, and that a future where DNA screening is routine is a very bleak one indeed. Hence while some screening could be a good idea, it's a slippery slope.

          1. Jellied Eel Silver badge

            Re: Ethical minefield, and maybe eugenics wasn't so last century?

            ..not me, but I guess maybe related to the social credit score thingy?

            Well, that's coming. Or here. Got vaxxed? You can join in with society! Unvaxxed? Sorry, remain in lockdown, citizen. Plus it's also riffing on the general accumulation and trade of people's personal information, especially medical information. Once that's out there, the Internet never forgets, and there are many potential uses for genetic information for both good, or bad. Sorry, citizen, you are defective. Report for sterilisation immediately. Or picking up on another commentor, we could speed up the elimination of undesirable traits by termination, or sterilisation at birth.

            After all, we've done this kind of thing in the past.

            Or there's fluffy stuff like this-

            https://www.weforum.org/agenda/2019/05/this-credit-card-has-a-carbon-emission-spending-limit/

            Tapping into that problem, Swedish fintech company Doconomy has launched a new credit card that monitors the carbon footprint of its customers - and cuts off their spending when they hit their carbon max.

            Everyone gets a 'free' monthly carbon credit. Buy stuff, it's CO2 cost is deducted. Exceed your limit and you can no longer buy stuff. If you're someone like Al Gore or Bill Gates, you can easily afford to buy more credit off the little people to pay for jet fuel. And like any other 'social credit' system, you can use it to enforce social policy. Who needs to use gentle 'nudges' when you can shove hard?

            And it'd be trivial to apply to eugenics policies as well. Want more credits so you can buy insect protein? 10kg credit for every pack of condoms. According to our elite, we've got an overpopulation crisis, so let's nudge a bit to manage that and encourage poor people to not breed. Once you've eliminated alternative payment mechanisms, you can use the card for enforcement as well, as we've done with contact tracking & tracing. This person is not authorised to breed, so why are they buying maternity products?

            The reason I referenced Gattaca is that it celebrates the human spirit, that determination and drive can overcome genetic disabilities, and that a future where DNA screening is routine is a very bleak one indeed.

            Indeed. It's a great movie, and a concept used in much SF, mostly dystopian. Yet a depressing number of people seem to think enabling these outcomes is a brilliant idea... And maybe it is, especially if carbon credits are transferable. Get a basic 250kg for every person eliminated. Think how much you could buy with the carbon footprint of one of the 0.1% who seem really keen on controlling the majority!

      2. An_Old_Dog Silver badge

        Re: Ethical minefield, and maybe eugenics wasn't so last century?

        Gattaca was a fine and relevant movie.

    2. Filippo Silver badge

      Re: Ethical minefield, and maybe eugenics wasn't so last century?

      Eugenics is stupid.

      Let's pretend (though it takes some effort) that we don't care about the ethical concerns.

      Getting rid of a trait, say a disease, through eugenics, takes several generations at best - long enough for all individuals that carry the trait to die without reproducing. You're unlikely to have a perfect worldwide program, so make that many generations. But let's be cautiosly optimistic and call it a century. Oh, and you can never really get rid of a problem this way, because random mutation, but nevermind.

      Now, considering that gene therapy is in its infancy, and we have only just started on practical applications of computational biology, how long do you think it will take before the cost of developing and deploying cures such as the one described in the article falls by several orders of magnitude? Ten years, twenty, maybe thirty? Let's be pessimistic, fifty?

      The objective will be achieved, much faster than with eugenics, and without having to get all nazi in the process.

      1. Jellied Eel Silver badge

        Re: Ethical minefield, and maybe eugenics wasn't so last century?

        Eugenics is stupid.

        Maybe. But the idea of curing, rather than treating inherited diseases isn't. The article demonstrates this with the pricing justification. If it works for thalassemia, why not cure sickle cell anaemia? But it's a very, very slippery slope.

        Getting rid of a trait, say a disease, through eugenics, takes several generations at best - long enough for all individuals that carry the trait to die without reproducing.

        You can accelerate that by simply making sure people who carry an undesirable trait, can't reproduce. We've been there, done that, and now have more ways to enforce policy.

        Now, considering that gene therapy is in its infancy, and we have only just started on practical applications of computational biology, how long do you think it will take before the cost of developing and deploying cures such as the one described in the article falls by several orders of magnitude?

        We don't know the cost, we've only seen the price. But technology advances rapidly. So take a handy tool like an mRNA vaccine. Concept published in 1989, first human trials in 2001. Then there was a thing discovered in 2020 and after a thorough, comprehensive 8 week trial, millions of people dutifully rolled up their sleeves to enrol in a grand experiment involving critical cellular functions.

        So now we know people are willing to participate in mass trials, it's 'just' some refinement. Mitochondria are critical for DNA repair, so tweak mtDNA or use mRNA to identify, repair or replace defective or undesirable sequences. And mitochondria play a big role in cellular differentiation, you could potentially make the fix permanent and inheritable. Maybe. Just convince most of the world's women that it's a GoodThing(tm) and just as safe as a vaccine!

        So potentially a simple, permanent and final solution. Also gender neutral because any woman can get the jab, although for some, it'd be pretty pointless. And it's also something SF writers have warned about..

        1. Ken Hagan Gold badge

          Re: Ethical minefield, and maybe eugenics wasn't so last century?

          If you are talking about the Covid vaccines, the m in mRNA does not stand for mitochondrial.

          1. Jellied Eel Silver badge

            Re: Ethical minefield, and maybe eugenics wasn't so last century?

            If you are talking about the Covid vaccines, the m in mRNA does not stand for mitochondrial.

            Hey, don't shoot the messenger. mRNA vaccines are just one of our first attempts at hacking our core OS. So in theory, safe-ish given the mRNA should be short lived, and ribosomes won't keep churning out the proteins. Or if they do, it should only be for the lifespan of those cells.

            But for gene therapies and more permanent cures, you're using some of the same principles. Correct an existing point mutation that has a negative outcome, eg sickle cell or thalassemia with a fresh, clean copy. So hacking our DNA or RNA to update our OS. Or induce more mutations, or add/remove/replace other OS features. That seems a tad risky, even though if we could do it reliably, could be used for a lot of good. Or inevitably lead to dystopian* 'designer babies' for the rich and shameless. Sure, we could regulate that, but we've already seen how easy it is to bypass. Research banned in your own country? Just shift the work to somewhere where it isn't.

            Or we get simpler, dystopian consequences, like the cost of treatments meaning cures are only available to the elite.

            *Having been a long-time SF fan, I remember watching a movie with a relative who was a geneticist. Dr Tyrell was explaining to a patient the challenges of gene therapy, and the patient wasn't entirely happy with the answer. The explanation sounded like mostly word-salad, with a few recognisable bits implying bad things, but relative said it was actually a pretty good explanation of the challenges & was quite impressed.

        2. Filippo Silver badge

          Re: Ethical minefield, and maybe eugenics wasn't so last century?

          I'm not quite sure why you're bringing up mRNA vaccines, which have nothing to do with inherited disease or DNA editing.

          However, if I understand correctly, your point appears to be that you consider eugenics to be ethically superior to DNA editing?

          I'm afraid I can't fathom the kind of thought that goes on behind that position, so I can only wish you well.

          I take solace in the knowledge that I don't really have to convince anyone, because eradicating disease through eugenics just won't happen, on account of being entirely pointless.

          1. Jellied Eel Silver badge

            Re: Ethical minefield, and maybe eugenics wasn't so last century?

            However, if I understand correctly, your point appears to be that you consider eugenics to be ethically superior to DNA editing?

            Obviously you don't understand correctly, but playing with straw is a depressingly common form of argumentation amongst people who lack critical thinking skills.

            If you try again, you'll see my point is the exact opposite to the one you're implying. DNA editing enables eugenics, and that's an issue that's sadly never gone away. Paul Erlich popularised it again with his infamous 'Population Bomb' book, which gained a lot of traction amongst our elite, spawning groups like the 'Optimum Population Trust'. Pretty much every prediction made by Erlich has been proved wrong, yet the ideas remain depressingly popular.

            I'm not quite sure why you're bringing up mRNA vaccines, which have nothing to do with inherited disease or DNA editing.

            Mostly as an example of how easily the public can be manipulated into doing something with very high potential risks. Otherwise, have a read about the roll of mitochondria, tRNA (transfer RNA), mRNA and ribosomes. The vaccine spoofs the tRNA part to get the organelle producing proteins. Hack a mitochondrion's RNA and you could get it to do that continuously, but then you'd make a lot less profit from flogging booster vaccine shots.

            ps.. I suppose you could be confused by eugenics in general, so here's a handy wiki definition-

            Eugenics.. from Ancient Greek εύ̃ (eû) 'good, well', and -γενής (genḗs) 'come into being, growing') is a set of beliefs and practices that aim to improve the genetic quality of a human population.

            So gene therapy to cure a disease would be a good use of eugenics. Problem is people's perception of, and ability to improve human's genetic quality. Plus the way popular perception of eugenics tends to focus on the potential abuses.

  4. Anonymous Coward
    Anonymous Coward

    Beta thalassemia is a hereditary disease allowing for a preventative treatment by carrier screening and prenatal diagnosis.

    Apparently, by abortion of that fetus, Some people are not going to do that.

    1. DS999 Silver badge

      Or use IVF

      Screen the fertilized eggs and only implant ones that don't have the disease. If I knew I had that disease in my family history, or had it myself, that's definitely how I would go. I'd bank a bunch of sperm to use for IVF then have a vasectomy to prevent accidental pregnancy.

      1. Jellied Eel Silver badge

        Re: Or use IVF

        If I knew I had that disease in my family history, or had it myself, that's definitely how I would go.

        Yup, same. I've also volunteered for a bunch of genetic studies. But it's not without risk of abuse, ie companies selling screening tests, or being exclusionary if you can't get free/cheap screening. Technology does seem to be making it simpler, faster and cheaper to do that though.

    2. midgepad

      New mutations may occur

      For various conditions.

  5. JDPower666

    Greed, plain and simple.

  6. StrangerHereMyself Silver badge

    Worth nothing

    Gene therapy isn't patentable since you're merely repairing a faulty gene. This means other companies may come up with a similar "cure" and sell it for less. Competition will eventually drive the price to zero.

    Gene therapy is a dead end, in that sense.

    1. Dave314159ggggdffsdds Silver badge

      Re: Worth nothing

      "Gene therapy isn't patentable since you're merely repairing a faulty gene."

      LOL. Why do people make these things up? Obviously it ain't true.

      1. StrangerHereMyself Silver badge

        Re: Worth nothing

        It is true. You can't patent repairing something.

        Their patent will not hold up in court.

        1. Dave314159ggggdffsdds Silver badge

          Re: Worth nothing

          That's nonsense. Why wouldn't you be able to patent a repair process?

          On top of the process patents, it has been made explicit that gene therapies can be patented, anyway.

          https://www.taylorwessing.com/synapse/ti-patenting-gene-sequences.html

    2. Filippo Silver badge

      Re: Worth nothing

      Not a big deal. You can't patent the correct DNA sequence, of course, but you definitely can patent a procedure or method to carry out the repairing process. That's good enough.

      What you describe would only be a problem if we were at a stage where merely identifying the broken DNA sequence and the right DNA sequence would be enough to trivially produce a medication that fixes the problem. We are nowhere near that, and until we get there, there's plenty of money to be made.

      1. StrangerHereMyself Silver badge

        Re: Worth nothing

        So you can patent say repair the faulty gene with CRISPR and patent that? I doubt that. At the very least it will be contested in court, since the procedure has to be unique and non-trivial.

        That would be like patenting the procedure to fix a broken part by replacing the part.

        1. Filippo Silver badge

          Re: Worth nothing

          Sure you can. Producing a gene therapy is not a standardized process where you take the right sequence, stick it to a CRISPR block, and jab away. That might work in a Petri dish, but it's not a therapy. You have to figure out how to minimize off-site interactions. How to elude the immune system in just the right way. How to stabilize the whole thing. How to make sure the compound is delivered to the right site. How to reliably synthesize the lot. The end result is a set of compounds and protocols, which is unique, and definitely non-trivial.

          We might eventually get to a point where there's a reliable standard way of producing an effective gene therapy directly from a DNA sequence, but it's a long way off.

          If that happens, then I agree that the results wouldn't be patentable - however, in the same scenario, creating a new therapy would also be extremely cheap, so that's fine.

        2. Dave314159ggggdffsdds Silver badge

          Re: Worth nothing

          Why have you invented this bizarre notion? Have you misunderstood something real? I can't think what, but you seem very sure of your imaginary ground.

          Yes, one could patent a 'procedure to fix a broken part by replacing the part', where that is non-trivial.

  7. Anonymous Coward
    Anonymous Coward

    Covid "vaccine" is gene therapy

    Look up Moderna's IPO report on the Securities Exchange Commission. Lots of money to be made in "gene therapy". Way more money was procured than the paltry $2.8M in this article. Just because each dose was 'free' to the masses doesn't mean the government didn't pay big on the back end.

    Of course, "gene therapy" has an negative connotation because the term is commonly associated with things like gender changes. Hence the recent re-branding to "gene editing".

    This is like one of us techies here pulling up a binary editor and changing bits, and hoping to modify the program's operation. Yeah, we might get the changes we want, but there is also a huge possibility of getting changes we don't want. And don't forget to factor in rushing a solution to general availability with minimal production testing.

    Go ahead and downvote me if you got the covid shot and don't like the after action truth. It is your genetics that got haphazardly 'edited'.

    1. Jamesit

      Re: Covid "vaccine" is gene therapy

      "Canadian researchers discover 'weak spot' in COVID-19 virus key to treatments

      Staff The Canadian Press

      Researchers at the University of British Columbia have discovered what they are calling a “ weak spot” in the virus that causes COVID-19.

      A study published in the peer-reviewed scientific journal Nature Communications says the “key vulnerability” is found in all major variants of the SARS-CoV-2 virus."

      https://rock101.com/news/9069304/british-columbia-research-covid-treatment/

    2. dl1jph

      Re: Covid "vaccine" is gene therapy

      Please learn the differences between gene editing and abusing the cell's protein synthesis machinery - the latter is what the COVID vaccines do and there's no way for that to permanently change anything, since our cells have no way of turning RNA to DNA. In your code analogy, what actually happens with the vaccines is closer to someone getting creative with an API that can't affect global state than messing with the binary itself. It also affects only a small number of muscle cells - those get killed off quickly by the immune system (since they appear to be infected by a virus as far as that's concerned) and get replaced within a few days.

      What the article discusses is something completely different, because it actually does alter the DNA in such a way that it gets copied along during cell division and they're doing it across most of the cell population responsible for a life-sustaining functionality. This is obviously far more risky - if it goes badly wrong, it's going to create a situation in which survival is unlikely. This is potentially a risk worth taking in a situation that's life-limiting anyway, but there's no way anything close to that level of risk is ever going to get regulatory approval for preventative purposes.

      1. Oglethorpe

        Re: Covid "vaccine" is gene therapy

        To be fair, some chromosomal changes do happen as a result of vaccination but that's just standard V(D)J recombination in some immune cells (not throughout the body), which happens every day, vaccine or not. The only difference the vaccine made was in promoting the division of certain cells which achieved complimentary recombination.

        Not that I'm agreeing with the paranoid ramblings you replied to but it's easier to combat misinformation if you acknowledge the kernel of truth that the deceptive turd is wrapped around.

    3. Spazturtle Silver badge

      Re: Covid "vaccine" is gene therapy

      mRNA is not gene therapy, it is a simple instruction to tell your cells to produce a certain protein and it is consumed in the process.

    4. Filippo Silver badge

      Re: Covid "vaccine" is gene therapy

      > "gene therapy" has an negative connotation

      It does? I don't think I've ever heard "gene therapy" used with a negative connotation in common usage. Maybe it's a thing in your social bubble?

      > Go ahead and downvote me if you got the covid shot and don't like the after action truth.

      It's not "truth", so there's nothing for me to like or dislike. You get downvotes for a poorly constructed argument, e.g. implying that because Moderna is working on gene therapy and also COVID vaccines, then COVID vaccines are gene therapy, or that because Moderna is a for-profit organization, then their products are harmful. Faulty logic gets downvotes.

  8. Filippo Silver badge

    It's good to hear that gene therapy is finally starting to deliver on its promises: permanent fixes to nasty life-long diseases.

    Since it's difficult to develop and then it has to be tailored to the individual, it can't help being very costly... for now. There's lots of room for automation in there. Very difficult, but not unfeasible. Eventually, though it may take decades, prices will come down.

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