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Gene Therapy Was Hailed as a Revolution – Then Came the Bill

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Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#51

Post by folli »

codeulike wrote: post #36
refurb wrote: post #4

Earlier quoted context omitted.

Unlike small molecules, gene therapy isn't something that somebody can just whip up in a lab. Similar to biologics, there are often some trade secrets involved in the manufacturing that require a lot of time and money to figure out.

CRISPR's not that hard, this guy did it in his garage. Delivering it to cells is a bit harder, but not that hard unless you're talking brain cells, then the blood-brain barrier causes trouble. https://www.theguardian.com/science/2017/dec/24/josiah-zayne...

CRISPR is unrelated to Zolgensma, which uses an Adeno-associated virus to deliver a functioning version of the SMN1 gene.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#54

Post by fapjacks »

nec4b wrote: post #43
deadbunny wrote: post #37

Earlier quoted context omitted.

I work for a government driven genome research company in the UK, there are many others like this across Europe. Gov funding isn't such a problem in Europe where healthcare is a government issue.

Could you link to drugs that you have developed and how much they cost?

Can you name a single drug that was developed without public assistance from taxpayers?

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#55

Post by dekhn »

anfilt wrote: post #23
refurb wrote: post #4

Earlier quoted context omitted.

Unlike small molecules, gene therapy isn't something that somebody can just whip up in a lab. Similar to biologics, there are often some trade secrets involved in the manufacturing that require a lot of time and money to figure out.

Still does not stop someone from trying since they now know what to look for. Also genetic synthesis has gotten cheaper and so has sequencing. Moreover a lot techniques for gene therapy are already in the literature. Moreover you have to document what it's doing or what your doing for approval (in-case of a procedure).

gene therapy isn't something you can just whip up in a random lab and apply to people without there being dire consequences (heck, that happens even in top research institutions).

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#56

Post by dekhn »

codeulike wrote: post #36
refurb wrote: post #4

Earlier quoted context omitted.

Unlike small molecules, gene therapy isn't something that somebody can just whip up in a lab. Similar to biologics, there are often some trade secrets involved in the manufacturing that require a lot of time and money to figure out.

CRISPR's not that hard, this guy did it in his garage. Delivering it to cells is a bit harder, but not that hard unless you're talking brain cells, then the blood-brain barrier causes trouble. https://www.theguardian.com/science/2017/dec/24/josiah-zayne...

anybody with a decent level of biochemical knowledge can do these things in a garage (I built a biolab in my garage and work at a lab that does CRISPR). However, the person you cited is clueless and is just kind of injecting things into his body without any really thought to efficacy or safety.

There is a big difference between injecting reagents into a single human, and doing actual medicine.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#57

Post by Palptine »

codeulike wrote: post #36
refurb wrote: post #4

Earlier quoted context omitted.

Unlike small molecules, gene therapy isn't something that somebody can just whip up in a lab. Similar to biologics, there are often some trade secrets involved in the manufacturing that require a lot of time and money to figure out.

CRISPR's not that hard, this guy did it in his garage. Delivering it to cells is a bit harder, but not that hard unless you're talking brain cells, then the blood-brain barrier causes trouble. https://www.theguardian.com/science/2017/dec/24/josiah-zayne...

Tell that to the guy that tried to create HIV resistance babies in China. He had a decent lab yet both babies he created didn't have ccr5 hit: the crispr missed target.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#58

Post by mruts »

awongh wrote: post #35

One thing that I'd like to know is if the profit margin is the same for one of these one time treatments as a repeat treatment. If we assume that these things must be produced within a market system, it seems like the real moral question isn't what the dollar amount is, (since it logically follows that the cost to produce it must increase with the total revenue it's going to produce) but it would seem troublesome to…

I’m not that well versed in gene therapy, but for pharmaceuticals the calculus is as follows: It takes billions of dollars and decades for a new drug to be put on the market. In addition to the cost, there’s the risk of the FDA not approving it. Once approved, the drug can essentially be produced for free. The government gives the company the right to charge whatever they want for a set number of years until the patent expires (including much of the research time). After that period, competition is allowed in.

It seems clear to me that costs and profits need to come from somewhere. But I wonder if there is a better system than just making each pill super expensive for a long period of time.

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