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FDA approves a CRISPR-based medicine for treatment of sickle cell disease

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Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#41
post #21

> Vertex set the price of Casgevy at $2.2 million > Patients must spend weeks, even months, in the hospital before and after the therapy is administered. Yoiks. So how many actual people are going to be able to get this treatment?

Collectively paying for rare but expensive treatments is literally the problem that insurance solves. This isn't wildly out of the expected range for this sort of thing. And it will surely get cheaper as it evolves.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#42
post #30

Earlier quoted context omitted.

Why anyone would be interested by a charlatan?

He primarily focuses on sleep, nutrition, and exercise as measurable methods to reduce the speed of aging. Not certain how that makes him a charlatan.

Yeah, sleep, food and exercise make you healthier, who would have thought.

You don't need to create a company claiming to "biohack" and sell products for that, blood transfer ( ! ), or 30 health specialists to monitor how many erection you have over night https://www.insider.com/bryan-johnson-sleeps-with-small-devi... , I just can't laughing.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#43

[flagged]

Given that NIH doesn’t want to focus on “lengthening life” or indeed reducing disability anymore it seems that only wealthy people will get to live longer.

https://directorsblog.nih.gov/2023/09/28/words-matter-action...

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#45
post #29

Earlier quoted context omitted.

This is Day 1 so the price and how well it works today is almost certainly the worst it will ever be. Insurance will likely cover the cost. It's a very bad, painful, and outright deadly genetic mutation and $2.2 million is practically nothing compared to doubling someone's lifespan or giving them an extra 10 years. More info I found relevant regarding cost for typical treatment and out of pocket estimated costs: http…

I don't agree that it is nothing. 2 millions, if applied properly, could do good for many people. Take ten children from poverty, give ten children chance to get a good education, etc. There is always a some kind of moral dilemma: should you spent millions to try to extend extremely I'll person or help with that money to some healthy poor children?

It's possible to do both :)

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#46
Here's basically how the process works:

* Harvest stem cells from the patient.

* Prepare a DNA plasmid with the Cas9 gene, guide RNA for the desired genetic modification, and an antibiotic resistance gene.

* Electroporate the plasmid into the harvested stem cells. Grow the electroporated stem cells in antibiotic-containing nutrient media. Only cells with the plasmid (and thus antibiotic resistance) survive.

* Expand and freeze the genetically modified cells.

* Administer chemotherapy to the patient to eliminate defective bone marrow stem cells.

* Inject the modified stem cells back into the patient, where they repopulate the bone marrow with the CRISPR edits, aiming to correct the genetic mutation.

This process isn't new but one of the biggest challenges is propagating genetic modifications to all effected cells in the body. This is why it's much easier to GMO an egg / sperm because once the change is made there, it's replicated in every new cell thereafter.

Other techniques utilize harmless viruses to transfect genetic modifications to the body, but this has other trade-offs. mRNA vaccines don't propagate to every cell, but the cells which do successfully transcribe the mRNA are able to generate enough of the target protein that the body can recognize it and develop an immunity to it. Eventually, the modified cells will die and no cells will be left to produce the mRNA vaccine protein.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#50

Earlier quoted context omitted.

I don't agree that it is nothing. 2 millions, if applied properly, could do good for many people. Take ten children from poverty, give ten children chance to get a good education, etc. There is always a some kind of moral dilemma: should you spent millions to try to extend extremely I'll person or help with that money to some healthy poor children?

It's possible to do both :)

How? Society does not have unlimited resources.

Especially the one that extremely ill people need, humans.

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