4 ms·

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 sequ

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..

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..