CRISPR is an extremely overhyped approach which found a marketing engine via popular science. There is 1 FDA approved CRISPR therapy as compared to 7 for AAV and 7 for Lentivirus.
Counting all viral vector therapies that have been approved, we’re sitting at 19 approved therapies versus 1 for CRISPR.
I think CRISPR ideas in a lab are just an easy way into the mainstream press, but viral vector delivery is the real future. It just didn’t get the same news cycle, for whatever reason.
You're correct about CRISPR Cas9. The off-target affects are difficult to manage.
The paper describes Cas12a2. This is a different mechanism with discovery origins in - of all things - agriculture. It does not attempt in any way to reprogram cells. It uses a guide protein to locate a specific mutation with exacting precision and, when it activates, unleashes total destruction of the cell.
The implications of Cas12a2 on undruggable conditions that exhibit known driver mutation profiles is profound.
Source: I have personally funded novel research based on Cas12a2 for an undruggable condition I have. I have personally seen my condition "cured" in vitro using this technology and it left all of my WT cells unharmed. Some of the researchers I've funded are co-authors in the paper linked. I am a layperson in this field (I'm a SWE, not in biotech), but I am happy to answer questions.
I hope this finally works out. I remember almost exactly ten years ago I got excited about one of these proposed cancer cures, tried to talk about it at lunch with my coworkers, and they laughed at me for believing.
I'm pretty optimistic. I think it's a threshold question where we need a number of basic technologies to all get over certain bars before the floodgates start to open.
Over the past 1-2 decades there has been unbelievable progress at the basic technology level but most people are unimpressed because they haven't translated yet due to not individually being sufficient to cause an explosion of progress. IMO, we're starting to see it finally as so many different technologies have gotten so cheap, fast, and good.
So we're waiting for the Apple of the medical world to take a bunch of preexisting things to be applied together in a way that makes the whole much more valuable than the pieces. Or we need all of the individual lions to come together to make the Voltron?
I'm not sure what this comment is trying to say. Theranos was a company build from the ground up on fraud. Apple, for all its faults, is provably at the forefront of technology used in personal computing devices.
The floodgates open = the market will see that at least some of that can actually work and make money => they will pour funding => new approaches built on that funding will start working, too?
Real in vivo genetic engineering isn't going away and will indeed be a powerful tool to face cancer. Any particular effort is doubtful because this is a journey measured in decades. It is not the same story as any one particular wonder drug fizzling out to nothing, it is a class of tools that is maturing into the realm of early therapeutic deployment.
> Much like other CRISPR therapies, delivery is a critical challenge, i.e., getting the large genome-cutting enzyme to all the targeted cells efficiently.
makes me think this is in vitro so far. So, years to decades away from being available for actual treatment in humans. Still good news.
Basically the issue is often that gene therapies end up in the liver since its the livers job to detoxify, but that may cause a dangerous immune response if the immune system notices it in the liver and attacks the organ, since the person could die from the damage.
I’m assuming this has been tried, but why doesn’t nano-encapsulated mRNA (that then makes the CRISPR sequences in cells) or whatever the peptide injectors do solve the problem?
You can target an individual by injecting that very individual with something lethal.
If that's not what you want, you'd need something like a virus to spread it. But then you have to ask yourself: what if that virus mutates? The specialization to certain gene markers is an evolutionary disadvantage, so evolution will tend to make it lose that restriction. Ooops.
Old concern, but it really doesn't work that way. Genetics don't respect human ideas like "nationalities" or "borders" - the targeting you can get by selecting on singular DNA variants is coarse enough to make ICBMs look like precision weapons.
Like many things of this nature, people keep bringing it up because it sounds Very Scary and Very Dystopian - not because it's worth giving an actual fuck about.
Same problem with chemo and radiation. A tumor may start off with a single cancerous mutation, but by the time it spreads there may be several. Once the cell repair machinery has been broken, the cancer cells are prone for more mutations.
Chemo, radiation, and CRISPR will kill everything it can reach that is susceptible. That leaves everything that was unreachable or resistant behind to start growing again.
Kill cancer cells is easy. Killing ONLY cancer cells is very hard.
What economic / political model would cause the society to prioritize this over adtech? It seems so unsettling that brilliant human minds are trying hard, every day, to figure out how to make it impossible to bypass watching ads on YouTube, instead of helping cure cancer.
The bargaining dynamics are stacked against biology researchers at every stage of their career, from needing years and years of unrelated performance to be admitted to terribly expensive programs before they can begin to do experiments, to requiring costly equipment and resources to work, to needing to work with a small number of very powerful companies.
As a result, life science researchers are more price-taking than proce-setting when it comes to their wages / salary. If money is the motivator, then the market as-is isn’t addressing this one.
> would cause the society to prioritize this over adtech?
Private pharmaceutical R&D spending in the U.S. is around $100bn per year [1]. NIH spends another $50bn a year on biomedical research [2].
That eclipses total investments into adtech per se, which generously counted shouldn’t exceed $50 to 60bn. (And that only by counting like a third to a half of Google, Amazon, et cetera R&D and capital spending as adtech.) More precisely counted, it probably doesn’t exceed $10bn.
And when you can measure how effective those ads are in changing human behavior; it's easier for businesses to spend there. As an American, I would love it if pharmaceutical companies couldn't market to consumers. It would free up money for research or lower prices.
I don't think an economic model would work. Only a political one would work where the government would redirect a lot of funds towards this, making it a lucrative profession.
Adtech works because there is a lot of money in it. There is a lot of money in it because people seek quick entertainment, and we have a LOT of people driving the demand.
Now compare that to cancer research. There's no short term gratification about it.
There's a fair bit of frequency illusion involved here. A lot of brilliant human minds aren't, in fact, working on ad tech, and a lot of the people working on ad tech aren't, in fact, that brilliant (as evidenced by them working adversarially against their own fellow humans, for one).
There's a wide world outside big tech, Silicon Valley, and software in general. It only tends to be a bit less visible online.
This is why I hate patents. If CRISPR were put behind a paywall, none of this would have happened. Everything having to be about profit is getting tiring.
> This is why I hate patents. If CRISPR were put behind a paywall, none of this would have happened. Everything having to be about profit is getting tiring.
CRISPR was the cause of a huge patent case and likely led to a change in US patent law because the impracticability of deciding who did something first in the laboratory.
It continues to influence research as some nations took a while to decide how they would resolve their own researchers' CRISPR claims with respect to MIT/UC Berkeley.
And yet... all the research has continued apace.
Edit: the CRISPR patent cases are continuing even today
Over on reddit people were debating whether cancer should be cured since it disproportionately affects rich people and it made me realise how far reddit has fallen. It's just a botnet now to manipulate elections.
I would imagine the charitable characterization of that discussion is much closer to “awesome, this will mean the Peter Thiels and Elon Musks of the world will live to 150 while both me and my children will be dead long before this trickles down to regular people” vs. “we shouldn’t cure cancer”.
After we launched our startup, we had all sorts of folks reach out to sell their GTM services. I went with one group from Vietnam that would make engagement bait Reddit questions with some accounts, and advertise our product in the comments section with others. It was expensive but it worked
Do you think (or care) about the ethics of this sort of behavior? Do you consider it unethical and if you do, under what conditions would you decide to do it anyway?
Reddit is a huge danger to society. There's no doubt that subs about specific non political (and non popular) topics are hugely beneficial, the overall damage the echo chambers do still outweigh these benefits.
The way the voting system works at Reddit encourages group think and bubbles. All it takes is five more down votes than up votes and a comment or post essentially disappears from view. It's a design that actively avoids debate.
I'm certain that is not a mainsteam opinion on reddit, but by its nature you will be able to find arbitrarily stupid opinions in individual echo chambers
Nature - https://www.nature.com/articles/s41586-026-10738-7
Counting all viral vector therapies that have been approved, we’re sitting at 19 approved therapies versus 1 for CRISPR.
I think CRISPR ideas in a lab are just an easy way into the mainstream press, but viral vector delivery is the real future. It just didn’t get the same news cycle, for whatever reason.
The paper describes Cas12a2. This is a different mechanism with discovery origins in - of all things - agriculture. It does not attempt in any way to reprogram cells. It uses a guide protein to locate a specific mutation with exacting precision and, when it activates, unleashes total destruction of the cell.
The implications of Cas12a2 on undruggable conditions that exhibit known driver mutation profiles is profound.
Source: I have personally funded novel research based on Cas12a2 for an undruggable condition I have. I have personally seen my condition "cured" in vitro using this technology and it left all of my WT cells unharmed. Some of the researchers I've funded are co-authors in the paper linked. I am a layperson in this field (I'm a SWE, not in biotech), but I am happy to answer questions.
I usually end Legend after the mannequin trap, and end Sunshine after the transit of mercury.
Over the past 1-2 decades there has been unbelievable progress at the basic technology level but most people are unimpressed because they haven't translated yet due to not individually being sufficient to cause an explosion of progress. IMO, we're starting to see it finally as so many different technologies have gotten so cheap, fast, and good.
> Much like other CRISPR therapies, delivery is a critical challenge, i.e., getting the large genome-cutting enzyme to all the targeted cells efficiently.
makes me think this is in vitro so far. So, years to decades away from being available for actual treatment in humans. Still good news.
If that's not what you want, you'd need something like a virus to spread it. But then you have to ask yourself: what if that virus mutates? The specialization to certain gene markers is an evolutionary disadvantage, so evolution will tend to make it lose that restriction. Ooops.
Like many things of this nature, people keep bringing it up because it sounds Very Scary and Very Dystopian - not because it's worth giving an actual fuck about.
Chemo, radiation, and CRISPR will kill everything it can reach that is susceptible. That leaves everything that was unreachable or resistant behind to start growing again.
Kill cancer cells is easy. Killing ONLY cancer cells is very hard.
As a result, life science researchers are more price-taking than proce-setting when it comes to their wages / salary. If money is the motivator, then the market as-is isn’t addressing this one.
Private pharmaceutical R&D spending in the U.S. is around $100bn per year [1]. NIH spends another $50bn a year on biomedical research [2].
That eclipses total investments into adtech per se, which generously counted shouldn’t exceed $50 to 60bn. (And that only by counting like a third to a half of Google, Amazon, et cetera R&D and capital spending as adtech.) More precisely counted, it probably doesn’t exceed $10bn.
[1] https://phrma.org/blog/phrma-member-companies-rd-investments...
[2] https://www.science.org/content/article/final-nih-budget-202...
Adtech works because there is a lot of money in it. There is a lot of money in it because people seek quick entertainment, and we have a LOT of people driving the demand.
Now compare that to cancer research. There's no short term gratification about it.
There's a wide world outside big tech, Silicon Valley, and software in general. It only tends to be a bit less visible online.
CRISPR was the cause of a huge patent case and likely led to a change in US patent law because the impracticability of deciding who did something first in the laboratory.
It continues to influence research as some nations took a while to decide how they would resolve their own researchers' CRISPR claims with respect to MIT/UC Berkeley.
And yet... all the research has continued apace.
Edit: the CRISPR patent cases are continuing even today
https://news.berkeley.edu/2025/05/12/federal-appeals-court-s...
https://www.broadinstitute.org/crispr/journalists-statement-...
>children
stepchildren, perhaps.