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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

#101

Post by confused_boner »

KyleSanderson wrote: post #19

Lyfgenia’s approval came with a black box warning about the possibility that patients who receive the therapy might later develop blood cancer and should be monitored for that risk. Two patients in trials of the drug died of blood cancers, and studies concluded that the cancers were caused by the chemotherapy conditioning regimen for the treatment, not Lyfgenia itself.

>the cancers were caused by the chemotherapy conditioning regimen for the treatment, not Lyfgenia itself.

I am certain some media group is going to conveniently leave this part out of the title of their article, and surely no one is gonna waste time reading the actual article and the rumors will take off.

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

#102

Post by ls612 »

lostlogin wrote: post #69
w0mbat wrote: post #44

The gene that causes sickle cell anaemia actually provides partial immunity to malaria, which is why this gene has not been bred out of the population over time.

> which is why this gene has not been bred out of the population over time. Is that why? Or is it just the people with it aren’t sick enough to die before procreating?

No the important part is that the mutation is recessive, but being heterozygous for it is enough to confer malaria resistance.

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

#103

Post by soulbadguy »

lotsofpulp wrote: post #98
soulbadguy wrote: post #89

Earlier quoted context omitted.

> If it was just that (in the US), then there would be non need to prevent insurers from pricing based on health of the insured. Or legislating a 3x cap on premiums between highest and lowest premium. Or legislating out of pocket maximums. I do not follow the point you are making here. The regulation and legislation around health insurance do not change the nature of it. I think you are assuming that insurers have pe…

Sorry, I don’t really know how else I can explain it. The age rating factor itself is pretty self explanatory. Instead of charging a sicker or older person $10,000 per month and healthier or younger people $100 per month because that is close to the expected loss in the calendar year for the insurer, they are mandated to charge younger/healthier people $1,000 per month so the older person can only be charged $3,000 p…

Here what i am understanding of the point you are making :

When we offer a service to a group of person, and somehow mandate a flat price for that service. The people using the service less are subsidizing the cost for the people who use the service less.?

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

#104

Post by skullone »

itishappy wrote: post #27
skullone wrote: post #9

Earlier quoted context omitted.

We voluntarily did DNA tests on both of us to rule out any parental defects, as well as tests in utero to see if there were issues. Luckily there were none, but we had some discussions on what we might do. It seems reasonable and responsible to perform these tests to be informed on your major life choices.

It seems reasonable from an informed person choice perspective, but (imho) rather uncomfortable as a government mandated breeding incentives program. If you don't mind sharing, what was the (rough) cost to you for the testing?

I think insurance paid for the tests in fact. We had the doctors collect stem cells from the umbilical cord and they're stored at some place. That was like $1000 each kid, and $100 a year to keep. But after like 10 years you get some money back if you don't use them and they get "donated" to someone or to research.

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

#105

Post by Modified3019 »

biomcgary wrote: post #64
freedomben wrote: post #35

Earlier quoted context omitted.

thanks that's neat, although I wish it wasn't with a Solanaceae member. Do you know if they are working on other types of produce or are they just working on potatoes?

Are you concerned about off-target edits activating toxin producing pathways?

Maybe they are referring to the small percentage of people are sensitive to the whole family (potatoes, tomatoes, peppers, tobacco, etc) and find any exposure produces inflammation/digestion issues.

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

#106

Post by lotsofpulp »

soulbadguy wrote: post #103
lotsofpulp wrote: post #98

Earlier quoted context omitted.

Sorry, I don’t really know how else I can explain it. The age rating factor itself is pretty self explanatory. Instead of charging a sicker or older person $10,000 per month and healthier or younger people $100 per month because that is close to the expected loss in the calendar year for the insurer, they are mandated to charge younger/healthier people $1,000 per month so the older person can only be charged $3,000 p…

Here what i am understanding of the point you are making : When we offer a service to a group of person, and somehow mandate a flat price for that service. The people using the service less are subsidizing the cost for the people who use the service less.?

Yes, although I think you meant to type

> The people using the service less are subsidizing the cost for the people who use the service more.?

Also, it is not a flat price, it is a capped price.

As an aside, think about how the optics would have been if the politicians were transparent that a significant portion of the tax liability to pay for the healthcare would be levied based on age.

Then think about older, rich people taking advantage of this and retiring early (between age 50 to 65), and because they can afford to have very low income (but a lot of assets), they qualify for even more subsidies during their most expensive years to insure, without negatively affecting their lifestyle.

https://www.healthcare.gov/glossary/premium-tax-credit

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

#107

Post by refactor_master »

monero-xmr wrote: post #75
ponector wrote: post #37

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?

This is the effective altruism / utilitarianism insanity. If we only thought about "what the best use of $2 million is" we would still be living in huts.

Actually, utilitarianism and capitalism have been the strongest growth factors for human prosperity and welfare since forever.

Many societies with excessively strong opinions on morals however are literally living in huts.

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

#108

Post by seydor »

mritchie712 wrote: post #25

Ohalo (the company Dave Friedberg is now CEO of) recently got approval for a potato edited by CRISPR: > Ohalo had two RSRs under consideration this year for its potato, one which focuses on higher concentrations of beta carotene – enhancing the overall health and nutrition value of the potato – and another which results in reduced glucose and fructose content in the potato, which, according to Ohalo, will reduce the…

monster-potato?

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

#109

Post by elektor »

huytersd wrote: post #65
elektor wrote: post #3

Now comes the hard question, how will the US payer system afford it? "An August report from the nonprofit Institute for Clinical and Economic Review found that the treatment and similar gene-editing therapies for sickle cell disease would be cost-effective if priced between $1.35 million and $2.05 million. In the U.S., patients with the condition and their insurers pay on average between $1.6 million and $1.7 million…

You answered your own question. The taxpayer already pays the same amount for lifetime treatment. This is just going to be the same except the person can lead a completely normal life after this. Also over time this will probably be much, much cheaper than the current lifetime treatment. There’s no reason a lot of the process can’t be easily automated.

No, I did not "answer my own question". These therapies are priced at $2.2 and $3.1 million, much greater than their calculated cost-effective price. And these are not small molecules that can be easily made generic, so not likely to get that much cheaper over time.

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

#110

Post by krapp »

refactor_master wrote: post #107
monero-xmr wrote: post #75

Earlier quoted context omitted.

This is the effective altruism / utilitarianism insanity. If we only thought about "what the best use of $2 million is" we would still be living in huts.

Actually, utilitarianism and capitalism have been the strongest growth factors for human prosperity and welfare since forever. Many societies with excessively strong opinions on morals however are literally living in huts.

>Many societies with excessively strong opinions on morals however are literally living in huts.

Setting aside that the US government is deeply influenced by Christian conservatism and the culture by Puritan ideals, to the point that no American President can be elected without vocally professing faith in God, Christ, or being seen with a Bible in hand, and thus is the most moralizing culture within Western civilization by far (particularly where sex and gender are concerned,) which hut-dwelling societies are you talking about, specifically?

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