6 ms·

Oh man. I agree with your sentiment, but this is a remarkable over simplification! The sequence may cost 700$ to synthesise. But it's delivery vector is a viru

Oh man. I agree with your sentiment, but this is a remarkable over simplification! The sequence may cost 700$ to synthesise. But it's delivery vector is a virus. Synthesizing the DNA, encapsulating the DNA in the virus.. not trivial stuff. While I agree that drug prices in the US are absurd beyond comprehension or justification, and I've had a ring side view of life sci research, drug development as well as the drug pricing; to posit that, hah, a path breaking gene therapy product just costs a few hundreds or a couple of thousands to produce is misrepresenting the complexity as well as the significance of the achievement. The amount of research (largely public funded but for the clinical trials, which may also have been public funded: no idea) to get to this point is staggering. the company also likely invested large amounts to Shepard a lab possibility into a viable treatment. FDA approval is a big deal because they are, justifiably, exceedingly strict. It is an arduous process. There has to be a better solution, which would start with allowing the US CMS / Medicare & Medicaid to negotiate drug prices with the manufacturers. The problem is policy.

jfarlow 9y ago
It is a bit of a simplification - but not by that many orders of magnitude ;). The really hard part (like small molecules) is the R&D to come up with that sequence. Unlike small molecules though, most people have the technological prowess to brew beer, make yoghurt, or buy an espresso. I've encapsidated plasmids into viruses for viral delivery in the lab and it took a day or two of work in a BSL2 hood to produce thousands of units of the virus - delivery in that case was relatively trivial; though you are right, I've never delivered the virus to humans - and that is not (yet) trivial. But neither is it as complicated as even the most basic of surgeries - literally. As far as I've heard, the 'treatment' to cure blindness with the above (and similar therapies) is a puff of air (containing aerosolized AAV) into a patient's eye, once - while alternative treatments are things like a syringe inserted behind the eyeball every few weeks for the rest of one's life. I'd argue that the significance of the achievement of these modern gene therapies is in just how simple the technology is relative to other medical procedures. In this case 'simple' is very much a result of an astounding amount of knowledge and work. It's so powerful precisely because it is simple. And to be clear - I don't actually think anyone should be trying to cure their own cancers with garage-built immunotherapies today. Home-grown insulin-kombucha, maybe... But the technologies of today realistically enable that possibility in the nearish future precisely because they are so elegantly built. Recouping the cost of that R&D to produce something so simple & powerful is always a challenge - and that challenge is far more social in nature than scientific.
- moh_maya 9y ago
  Again, I don't disagree with you. But shepherding a drug or treatment though the FDA is a mission in itself. It is expensive. The cost of goods may be relatively low once the process of production at scale is worked through, but it is hardly trivial. Notwithstanding what I've said, I genuinely find it appalling that these drugs are priced at five figures. That's just criminal. In the range of thousands (or even 10s of thousands, if it's a single shot treatment), I'd think the pricing is justified. Maybe if it's a rare disorder, charge a little more. But the travesty in the US system is that the govt., Via Medicare & Medicaid, is just not allowed to negotiate on costs. That's what the UK NICE does. The American system is insane!
- moh_maya 9y ago
  // offtopic BTW, big fan of what you and serotiny are trying to do. I've just moved back to my home country.. am dabbling in other stuff at the moment, but the dream is to join with friends and start an automated high throughput industrial biotech company. PhD work used a lot of robotics and high throughput screening & mutagenesis and what have you.. and services like serotiny give me the comfort that my pie in the sky idea has a chance..

jfarlow 9y ago

It is a bit of a simplification - but not by that many orders of magnitude ;). The really hard part (like small molecules) is the R&D to come up with that sequence. Unlike small molecules though, most people have the technological prowess to brew beer, make yoghurt, or buy an espresso. I've encapsidated plasmids into viruses for viral delivery in the lab and it took a day or two of work in a BSL2 hood to produce thousands of units of the virus - delivery in that case was relatively trivial; though you are right, I've never delivered the virus to humans - and that is not (yet) trivial. But neither is it as complicated as even the most basic of surgeries - literally. As far as I've heard, the 'treatment' to cure blindness with the above (and similar therapies) is a puff of air (containing aerosolized AAV) into a patient's eye, once - while alternative treatments are things like a syringe inserted behind the eyeball every few weeks for the rest of one's life. I'd argue that the significance of the achievement of these modern gene therapies is in just how simple the technology is relative to other medical procedures. In this case 'simple' is very much a result of an astounding amount of knowledge and work. It's so powerful precisely because it is simple. And to be clear - I don't actually think anyone should be trying to cure their own cancers with garage-built immunotherapies today. Home-grown insulin-kombucha, maybe... But the technologies of today realistically enable that possibility in the nearish future precisely because they are so elegantly built. Recouping the cost of that R&D to produce something so simple & powerful is always a challenge - and that challenge is far more social in nature than scientific.

moh_maya 9y ago
Again, I don't disagree with you. But shepherding a drug or treatment though the FDA is a mission in itself. It is expensive. The cost of goods may be relatively low once the process of production at scale is worked through, but it is hardly trivial. Notwithstanding what I've said, I genuinely find it appalling that these drugs are priced at five figures. That's just criminal. In the range of thousands (or even 10s of thousands, if it's a single shot treatment), I'd think the pricing is justified. Maybe if it's a rare disorder, charge a little more. But the travesty in the US system is that the govt., Via Medicare & Medicaid, is just not allowed to negotiate on costs. That's what the UK NICE does. The American system is insane!
moh_maya 9y ago
// offtopic BTW, big fan of what you and serotiny are trying to do. I've just moved back to my home country.. am dabbling in other stuff at the moment, but the dream is to join with friends and start an automated high throughput industrial biotech company. PhD work used a lot of robotics and high throughput screening & mutagenesis and what have you.. and services like serotiny give me the comfort that my pie in the sky idea has a chance..