3 ms·

This is awesome. I have Crohn's and am looking forward to an eventual cure. One thing I don't understand is why big pharma and VCs seem to focus on rare diseas

This is awesome. I have Crohn's and am looking forward to an eventual cure. One thing I don't understand is why big pharma and VCs seem to focus on rare diseases instead of a gene therapy for changing eye color or hair color. With eye/hair color gene treatments, patients could undergo the therapy multiple times (if desired) and the pool of patients is all humans (not just rare diseases sufferers). This allows for amortization of the R&D expense over substantially more treatments. Plus the advances in gene therapy technology could then be applied to therapies for rare diseases

Kalium 9y ago
I can think of a couple of reasons. First, they think of themselves as companies that address diseases. They are concerned with the ill, rather than people seeking cosmetic changes. Second, I can see some potential for major backlash. Literally going into the business of designer babies? Ethically tricky. Doing it only for the rich, because it'll be incredibly expensive at first? "Tricky" doesn't begin to cover it. Public policy in first-world countries is not always noted for being far-sighted and even-handed, and a public backlash could easily shut down all gene therapies.
- king07828 9y ago
  >they think of themselves as companies that address diseases. They are concerned with the ill, rather than people seeking cosmetic changes Agreed. The joke being that black hair (or blonde hair or red hair) is a disease that can be "cured" by a different hair color :) >backlash ... [against] designer babies Agreed that this is an issue, especially if the treatment were marketed at unborn children. It may be better to focus on cosmetic treatments for adults to try and avoid this. I expect this technology will eventually become ubiquitous in our lifetimes, with the only question being the time frame for which it will come to pass
pishpash 9y ago
How much can you charge for cosmetic changes that can be easily done by less expensive means? I'm sure they've done the math.
- king07828 9y ago
  >cosmetic changes that can be easily done by less expensive means The less expensive means (contacts, hair dyes) are temporary and require maintenance. The benefits of a genetic treatment is that it is permanent (no cleaning of contacts, no subsequent salon visits), but should also be reversible
frgtpsswrdlame 9y ago
>I have Crohn's and am looking forward to an eventual cure. Check this out, it has a lot of interesting information: http://www.crohnsmapvaccine.com http://www.crohnsmapvaccine.com Could be interesting although obviously still in phase 1.
- king07828 9y ago
  This is awesome! from the link: >Historically, MAP in humans has been difficult to study as it cannot be seen under an ordinary microscope and is very difficult to grow. Testing for MAP by the presence of its DNA (using PCR) has found MAP in up to 92% of Crohn’s patients but until now no-one has developed a test to show MAP in-situ in the tissues of people with Crohn’s disease. It is fascinating that there is a virus that evades regular diagnostic techniques (i.e., using a microscope). That the virus can be found using genetic testing speaks to the power of genetic technology and how revolutionary the true understanding of genetics will be
frozenport 9y ago
Sounds hard because you need to first disable existing machinery that makes the natural color Then what if you want to change?
patall 9y ago
I am sorry for that but to be honest your comment suggests that you do not have tiniest idea of how hard medical research is. Not only are the topics you suggest much more complicated than rare diseases (i.e. we do not know the exact molecular process but they are certainly much more complex and in the case of eye color, entirely developmental, so changing the genetic background would not switch your eye color), but also much more expensive (for these are nonmedical applications and therefore side affects would kill your drug). So we are talking in the order of 10 billion in R&D expenses. And then, unlike in case of orphan diseases, your patents will be weak and the margin will be low (compared to cost).
- king07828 9y ago
  I am sorry, but to be honest your comment suggests that you do not have tiniest idea of how insulting the first sentence of your comment is. Instead of simply conveying the idea that things are expensive, the first sentence of your comment is styled as a personal attack, which does not add to the discussion and should not have been included. For example, an alternative first sentence, which conveys the point being made without being a personal attack would be "Medical research is hard." Regarding the substance of your remarks, if 10,000 people have a rare disease then the 100 million dollars it takes to develop that therapy can only be amortized over that small pool, which translates to $10,000 per person, assuming that every person pays for the therapy. If 3 billion people (a rough estimate of everyone connected to the internet) is the market size for hair/eye color treatment and the 10 billion dollars of R&D gets amortized over that full-size, then the cost is $3.33 per person. If only 10% of people want to change colors, and only 10% of those people are willing to partake in the therapy, then the cost becomes $333.33 per person to amortize the R&D expense, which is in the same order of magnitude as contacts or a salon hair treatment.
aaavl2821 9y ago
the cost of manufacturing a gene therapy would probably exceed what most people are willing to pay for changing eye or hair color. why use gene therapy when colored contacts or hair dye work, and much more easily reversible in case you dont like it? also, it is incredibly expensive to get a gene therapy through fda trials. there hasnt really been a "true" gene therapy approved in the us yet (excepting car-t), and the first two approved in europe were pulled from the market because they werent profitable even at a price of $1M per patient. thats why these companies go after severe rare disease -- you can charge more for saving a kids life than you could for changing hair color, and you have to charge a lot to justify the expense and incredibly high risk of developing a gene therapy. and with a rare disease, you can go to market with a 20-30 person sales force to cover all patients, whereas to market a hair or eye color thearpy youd need thousands of people calling on every hair salon and eye doctor in the country. as of now, the success rate in terms of fda approval for gene therapy is 0% -- thats an important input in financial calculations. further complicating things is that you generally cant dose these drugs more than once bc your immune system rejects the vector after the first dose, so you have to get all your economics upfront
- king07828 9y ago
  >the cost of manufacturing ... >it is incredibly expensive ... These may be outweighed by the potential market size of 3 billion people (a rough estimate of everyone connected to the internet). If R&D and manufacturing have a total cost of 10 billion dollars, then this reduces to $3.33 per person of the potential market size. >cant dose these drugs more than once This may be a showstopper for now. At a certain point, the technology should improve to allow multiple treatments so that a single individual could have red hair for a year, blonde hair for a year, black hair for a year, ... The cool thing about growing blonde hair instead of dying hair blonde, is that nothing more needs to be done and no further treatments need to be had (unlike dyes). This does assume that the individual has enough patience for the growth process

Kalium 9y ago

I can think of a couple of reasons. First, they think of themselves as companies that address diseases. They are concerned with the ill, rather than people seeking cosmetic changes. Second, I can see some potential for major backlash. Literally going into the business of designer babies? Ethically tricky. Doing it only for the rich, because it'll be incredibly expensive at first? "Tricky" doesn't begin to cover it. Public policy in first-world countries is not always noted for being far-sighted and even-handed, and a public backlash could easily shut down all gene therapies.

king07828 9y ago
>they think of themselves as companies that address diseases. They are concerned with the ill, rather than people seeking cosmetic changes Agreed. The joke being that black hair (or blonde hair or red hair) is a disease that can be "cured" by a different hair color :) >backlash ... [against] designer babies Agreed that this is an issue, especially if the treatment were marketed at unborn children. It may be better to focus on cosmetic treatments for adults to try and avoid this. I expect this technology will eventually become ubiquitous in our lifetimes, with the only question being the time frame for which it will come to pass

pishpash 9y ago

How much can you charge for cosmetic changes that can be easily done by less expensive means? I'm sure they've done the math.

king07828 9y ago
>cosmetic changes that can be easily done by less expensive means The less expensive means (contacts, hair dyes) are temporary and require maintenance. The benefits of a genetic treatment is that it is permanent (no cleaning of contacts, no subsequent salon visits), but should also be reversible

frgtpsswrdlame 9y ago

>I have Crohn's and am looking forward to an eventual cure. Check this out, it has a lot of interesting information: http://www.crohnsmapvaccine.com http://www.crohnsmapvaccine.com Could be interesting although obviously still in phase 1.

king07828 9y ago
This is awesome! from the link: >Historically, MAP in humans has been difficult to study as it cannot be seen under an ordinary microscope and is very difficult to grow. Testing for MAP by the presence of its DNA (using PCR) has found MAP in up to 92% of Crohn’s patients but until now no-one has developed a test to show MAP in-situ in the tissues of people with Crohn’s disease. It is fascinating that there is a virus that evades regular diagnostic techniques (i.e., using a microscope). That the virus can be found using genetic testing speaks to the power of genetic technology and how revolutionary the true understanding of genetics will be

frozenport 9y ago

Sounds hard because you need to first disable existing machinery that makes the natural color Then what if you want to change?

patall 9y ago

I am sorry for that but to be honest your comment suggests that you do not have tiniest idea of how hard medical research is. Not only are the topics you suggest much more complicated than rare diseases (i.e. we do not know the exact molecular process but they are certainly much more complex and in the case of eye color, entirely developmental, so changing the genetic background would not switch your eye color), but also much more expensive (for these are nonmedical applications and therefore side affects would kill your drug). So we are talking in the order of 10 billion in R&D expenses. And then, unlike in case of orphan diseases, your patents will be weak and the margin will be low (compared to cost).

king07828 9y ago
I am sorry, but to be honest your comment suggests that you do not have tiniest idea of how insulting the first sentence of your comment is. Instead of simply conveying the idea that things are expensive, the first sentence of your comment is styled as a personal attack, which does not add to the discussion and should not have been included. For example, an alternative first sentence, which conveys the point being made without being a personal attack would be "Medical research is hard." Regarding the substance of your remarks, if 10,000 people have a rare disease then the 100 million dollars it takes to develop that therapy can only be amortized over that small pool, which translates to $10,000 per person, assuming that every person pays for the therapy. If 3 billion people (a rough estimate of everyone connected to the internet) is the market size for hair/eye color treatment and the 10 billion dollars of R&D gets amortized over that full-size, then the cost is $3.33 per person. If only 10% of people want to change colors, and only 10% of those people are willing to partake in the therapy, then the cost becomes $333.33 per person to amortize the R&D expense, which is in the same order of magnitude as contacts or a salon hair treatment.

aaavl2821 9y ago

the cost of manufacturing a gene therapy would probably exceed what most people are willing to pay for changing eye or hair color. why use gene therapy when colored contacts or hair dye work, and much more easily reversible in case you dont like it? also, it is incredibly expensive to get a gene therapy through fda trials. there hasnt really been a "true" gene therapy approved in the us yet (excepting car-t), and the first two approved in europe were pulled from the market because they werent profitable even at a price of $1M per patient. thats why these companies go after severe rare disease -- you can charge more for saving a kids life than you could for changing hair color, and you have to charge a lot to justify the expense and incredibly high risk of developing a gene therapy. and with a rare disease, you can go to market with a 20-30 person sales force to cover all patients, whereas to market a hair or eye color thearpy youd need thousands of people calling on every hair salon and eye doctor in the country. as of now, the success rate in terms of fda approval for gene therapy is 0% -- thats an important input in financial calculations. further complicating things is that you generally cant dose these drugs more than once bc your immune system rejects the vector after the first dose, so you have to get all your economics upfront

king07828 9y ago
>the cost of manufacturing ... >it is incredibly expensive ... These may be outweighed by the potential market size of 3 billion people (a rough estimate of everyone connected to the internet). If R&D and manufacturing have a total cost of 10 billion dollars, then this reduces to $3.33 per person of the potential market size. >cant dose these drugs more than once This may be a showstopper for now. At a certain point, the technology should improve to allow multiple treatments so that a single individual could have red hair for a year, blonde hair for a year, black hair for a year, ... The cool thing about growing blonde hair instead of dying hair blonde, is that nothing more needs to be done and no further treatments need to be had (unlike dyes). This does assume that the individual has enough patience for the growth process