What's going on with gene therapies? • Hacker News Board

Re: What's going on with gene therapies?

#31

Post by mandevil » Thu, Jul 17, 2025, 9:34 PM UTC

michaelbrave wrote: post #30

mandevil wrote: post #19

Earlier quoted context omitted.

This is actually a lot harder than you think because we still don't understand gene therapy well enough to be able to be sure that any given treatment works without the really expensive part, the Randomized Control Trial. In 2017 a gene therapy was approved in the US for RPE65-associated Retinitis Pigmentosa, Luxterna, the first in-vivo gene therapy approved by the FDA (yay! List price was a mere 425,000/eye). Just a…

I think a lot of our diseases if looked at genetically instead of symptoms-wise that we will probably find out that it's actually multiple conditions that we just group together for manifesting in similar ways. I've felt this in my own life with ADHD things where it seems to me that there are at least 3-4 different types of ADHD and that they respond to treatments/medications differently, and this makes me think that…

This is such a deep hole of complexity. (My wife is a pharmacist, I make computers do what I tell them to, and she has convinced me that her field is far more complex than us software people can imagine.)

To pick one story my wife has told me, take the example of Multiple Sclerosis. In the 1970's and 1980's, thanks to the MRI machine, there was finally a good diagnosis tool for MS: can you see the lesions in the scan? If you can congrats, you have MS. If you can't, it might be early MS where the lesions are small enough that we can't see them (generally they are visible several years after initial symptoms). But there were a lot of people who had MS-like symptoms and no lesions, so diagnosis of Chronic Fatigue Syndrome (named just in 1970, in contrast to MS which was identified in the 19th Century) started to rise. Most of those people would have been diagnosed with MS in 1950, but now we can rule that out and so they go into the new bucket instead. What is going on with patients in that CFS bucket? It's a mystery. Is it one thing or many? Who knows! Is it genetic or environmental? Who knows!

And how does one get original flavor MS? It's not fully genetic- comparing identical and fraternal twins we can see that it's not purely genetic but there must be a genetic influence. The current most accepted theory, according to my wife, is basically Long COVID- but for the Epstein-Barr virus (what causes Mononucleosis) instead of COVID-19. So if your immune system is somehow susceptible to this (the genetic component, which we apparently do not understand), and it encounters EBV (and there is a bit of bad luck? Who knows!) then somehow the immune system gets confused and starts attacking your own nervous system.

We computer people are simply attacking much more tractable problems.

Re: What's going on with gene therapies?

#32

Post by marbro » Thu, Jul 17, 2025, 9:47 PM UTC

l0new0lf-G wrote: post #27

Could it be just that gene therapies should be publicly funded? Taxpayer money has paid for a substantial percentage of research around the world, and it doesn't seem necessary that we rely on profit-driven private companies for the actual therapies when they could be done at publicly funded hospitals too.

[dead]

Re: What's going on with gene therapies?

#33

Post by anon84873628 » Thu, Jul 17, 2025, 9:51 PM UTC

toast0 wrote: post #20

itake wrote: post #14

Earlier quoted context omitted.

I think we are saying the same thing. Companies chase high risk high reward over low risk low reward. Presumably, curing a genetic disorder for a very small number of people using existing technology is low risk and low reward. > Why wouldn't it be sold if it were available? Just because something works in a small lab doesn’t mean it’s ready to sell. To launch a product, companies need to do a lot more: testing, cert…

> Presumably, curing a genetic disorder for a very small number of people using existing technology is low risk and low reward. I don't know if that presumption is reasonable. Yes, low reward; but I suspect the risk/cost for a gene therapy is about the same, regardless of the number of people affected. You still need to do all the pre-human trials, which are still expensive; then you've got to do human trials, which…

Perhaps in the future we will have regulation that allows, let's call it, "experimental" genetic therapy for diseases with a very small patient population, similar to what is already allowed for certain terminally ill patients. That could reduce the economic barriers to treating these groups.

Re: What's going on with gene therapies?

#34

Post by steeleyespan » Thu, Jul 17, 2025, 9:54 PM UTC

I have Alpha 1 Antitrypsin Deficiency which often leads to lung and liver transplants (if available) late stage, blood product early stage.

It’s pretty rare, but there is gene therapy in the works promising 2027 timeline or so for availability.

Re: What's going on with gene therapies?

#35

Post by steeleyespan » Thu, Jul 17, 2025, 10:01 PM UTC

steeleyespan wrote: post #34

I have Alpha 1 Antitrypsin Deficiency which often leads to lung and liver transplants (if available) late stage, blood product early stage. It’s pretty rare, but there is gene therapy in the works promising 2027 timeline or so for availability.

I was wrong looks like 2030!

Re: What's going on with gene therapies?

#36

Post by Fomite » Thu, Jul 17, 2025, 10:04 PM UTC

pedro_caetano wrote: post #12

From what I understand the situation is quite the opposite, there is a huge pipeline of investment both in startups as well as very large corps on the development and application of gene therapy. As anecdata, Luxturna costs north of a quarter of a million per vial, it treats a fairly rare disease (Leber congenital amaurosis). Roche still poured millions into development. Bio-tech, specially in a Medical field, is a v…

It's also just hard. Biology is hard. Being a startup in a heavily regulated field, rather than the "What if $Industry but with no regulations?" of the Uber/AirBnB model, and where iteration doesn't necessarily show returns makes life for biotech startups difficult.

Re: What's going on with gene therapies?

#37

Post by NooneAtAll3 » Thu, Jul 17, 2025, 10:04 PM UTC

pedro_caetano wrote: post #12

From what I understand the situation is quite the opposite, there is a huge pipeline of investment both in startups as well as very large corps on the development and application of gene therapy. As anecdata, Luxturna costs north of a quarter of a million per vial, it treats a fairly rare disease (Leber congenital amaurosis). Roche still poured millions into development. Bio-tech, specially in a Medical field, is a v…

isn't that exactly what article is saying?

> Bluebird Bio, once a leader with three FDA-approved gene therapies, sold itself to private equity for around $30 million earlier this year, a 99.7% decline from their all-time-high $10b valuation

*investment* is there, market isn't

Re: What's going on with gene therapies?

#38

Post by Fomite » Thu, Jul 17, 2025, 10:06 PM UTC

l0new0lf-G wrote: post #27

Could it be just that gene therapies should be publicly funded? Taxpayer money has paid for a substantial percentage of research around the world, and it doesn't seem necessary that we rely on profit-driven private companies for the actual therapies when they could be done at publicly funded hospitals too.

We really don't have a great pipeline for publicly funded developments going all the way to the treatment stage.

Re: What's going on with gene therapies?

#39

Post by folli » Thu, Jul 17, 2025, 10:10 PM UTC

l0new0lf-G wrote: post #27

Could it be just that gene therapies should be publicly funded? Taxpayer money has paid for a substantial percentage of research around the world, and it doesn't seem necessary that we rely on profit-driven private companies for the actual therapies when they could be done at publicly funded hospitals too.

This argument is not limited to gene therapies, but would apply to pretty much every pharmaceutical product.

One issue is however that the actual costs are not so much in early R&D (what the publicly funded universities and hospitals are doing), but in the later stage (clinical trials) which needs deep pockets and appetite for risk, which only big pharma has, because they see a potential big payout.

Re: What's going on with gene therapies?

#40

Post by anon84873628 » Thu, Jul 17, 2025, 10:10 PM UTC

southernplaces7 wrote: post #6

JohnMakin wrote: post #2

I have an extremely rare autosomal dominant subtype of a certain genetic disorder that affects maybe less than a few thousand individuals at any one time in the USA. Much common versions of the disorder that affect children have been successfully treated/cured with gene therapy. However gene treatment unlikely to ever reach people like me because so few are affected by it, it's not viable commercially. It's extremely…

But in all seriousness, who would you direct your anger at in this case? For one thing, being angry in a general sense implies that a fortuitous blend of circumstances is owed to you by the world in a broad sense so that your specific, completely accidental (of nature) misfortune can be remedied. Secondly, many people suffer from much more common conditions that also have no remedy so far (i'm an example of such), an…

I find this comment to be downright insulting to the parent. You manage to:

1) Call them entitled for feeling angry 2) Remind them that other people have it worse 3) Imply they're stupid for imaging things could work any other way

And then try to defend your obviously immature, unproductive, and unsolicited criticism in the "I'm just being logical!" excuse.

The parent didn't go on a screed and rage at the world. They didn't try to claim their anger is righteous. And they certainly didn't ask you to play two bit therapist.

Rather, they shared a totally reasonable and relatable emotional response to the situation they are in. I personally found it relevant and insightful to the discussion.

I highly encourage you to reevaluate to how you respond to these sorts of comments in the future.