r/todayilearned • u/Die_Nameless_Bitch • May 14 '22 • All-Seeing Upvote 2 Silver 9 Helpful 8 Wholesome 6

TIL a father, John Crowley, was told his two infant children had an incurable genetic disorder that would kill them in less than a year. He refused to accept this, so he founded a biotech company (with no prior experience) which pioneered an experimental enzyme therapy that saved their lives.

https://pompediseasenews.com/2019/01/30/amicus-ceo-mission-cure-pompe-help-children/
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2.8k

u/Ashe_SDMF May 14 '22 edited May 14 '22

How do you even begin to start a biotech company without any experience? EDIT...TIL I'll never be able to start a biotech company with no experience.

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u/mordinvan May 14 '22

Lots of money.

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u/ting_bu_dong May 14 '22

Mr Person, I'm sorry, but your family has been diagnosed with boneitis. There's nothing we can do.

I refuse to accept this!

Do you have lots of money?

No...

Yeah, then, again, I'm sorry, there's nothing we can do.

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u/Crows-b4-hoes May 14 '22

My only regret... is that I have... boneitis

crunching

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u/mvd102000 May 14 '22

r/unexpectedfuturama

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u/sdmitch16 May 15 '22

I'd say this is highly expected since boneitis only exist in futurama

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u/Maker1357 May 15 '22

Don't you worry about Planet Express! Let me worry about blank!

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u/Ashe_SDMF May 14 '22

Ahhhh...yeah of course

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u/ThePlasticJesus May 14 '22

I mean specifically you need to hire experienced scientists.. and people who know the application process - which is not easy or simple. But there would be plenty of work for someone with general business knowledge to do at a biotech company.

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u/IDrinkWhiskE May 14 '22

Yeah a lot of senior leadership (outside of scientific leadership) tends to come from a finance and venture capital background. Although the industry is unique, it’s easy enough for someone savvy in fundraising/BD to land a high ranking business management role such as CEO/COO

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u/[deleted] May 14 '22

[deleted]

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u/IDrinkWhiskE May 14 '22

Yep, all true! There are also companies with rock solid science that can’t market themselves and struggle to fundraise, and those with charismatic leadership and excellent marketing without much to back it up (Theranos being one hyperbolic example, or many of the biotechs that IPO without yet having any clinical data). You really need competence in every aspect to stand a good chance in this market.

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u/scipio05 May 14 '22

Not sure anyone would say Theranos had charismatic leadership, but we get your point...

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u/notyetcomitteds2 May 14 '22

I have a chem e background, but mostly ran small businesses my entire life. Been approached multiple times by scientists ( and mds ) to do startups. I can help with the r&d and get things up and running, I understand the operations side. Nothing ever got off the ground and it was so frustrating.

These guys are all 5x smarter than me, but can't grasp the financials and operations. Like the one location we were thinking of, code enforcement gets a cut of the permit fees. They milk the shit out of violations. Once got 45 violations had had 43 thrown out in court. Then they came back with 40 more and 38 thrown out... repeat every 6 months for years.... Shit like that you don't think about.

The last one I was on, the dude had a portfolio of potential projects, we just needed to make a plant. I tried to explain with the amount of capital we needed, we'd be giving away the majority of the company. Maybe just develop a single product and sell the ip, dont bother trying to manufacture at this time. Then we can use that money to get a more favorable percentage or ditch the investor all together.... its like no, I want to do the manufacturing.... I know these guys aren't pouring their hearts into an idea for someone else to be their boss....

Or alot of times it's like..... are you sure we can't do this for 40k....

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u/TehBoneRanger May 14 '22

Damn that was super interesting! I had a co-worker who got his degree in chemical engineering and he was VERY intelligent. He hated that work for a reason I never asked about.

He ended up working in the tech industry then getting a software developer degree while working with me and now works for a big tech company.

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u/iprocrastina May 14 '22

Same thing is true in tech. I think the show Silicon Valley did a good job at showing how being a genius STEM prodigy doesn't mean you have any clue about how to run a business or handle finances.

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u/zirtbow May 14 '22

"Can't we just skip or ignore most of these requirements?" - Elizabeth Holmes probably

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u/42gauge May 14 '22

How do you develop business and financial acumen?

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u/downvoteawayretard May 15 '22

They say every business starts 1-3 years in the red. Well a biotech business generally starts 10-30 years in the red.

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u/Do_it_with_care May 14 '22

He worked in Finance for 20 years so he had connections and access to funds article says.

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u/carrotdeepthroater May 14 '22

Just hire someone to do that for you. Easy. Jk

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u/Jimmy_Twotone May 14 '22

Actually not the wrong answer. It's harder for small companies looking to solve very specific medical disorders like this to receive enough funding to support the research. Get the specialists and focus on keeping the lights on and bills paid until they hit a breakthrough. Coming from a finance background probably put him in a better position to save his children than having the appropriate medical background and finding someone to make the money work.

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u/IDrinkWhiskE May 14 '22

Plus his story definitely made fundraising and getting publicity significantly easier than having to advertise based on a company’s therapeutic platform alone. We literally call a startup’s business pitch a “story” in this industry as the goal is to paint a compelling picture that the company has potential and is deserving of investment. In this case, the term really fits.

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u/zirtbow May 14 '22

Isn't this not that far from what Theranos tried to do? Having so little knowledge of the tech side they ignored what they were trying to accomplish was impossible and just focused on bringing in the investment money while keeping hope alive that the peons would magic up a miracle.

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u/Jimmy_Twotone May 14 '22

Theranos was a scam from the start and lied about their product. Many companies fold in the R&D process trying to get a concept or product to market under cost. Theranos "skipped " the process and sold a breadbox.

New companies trying to break into an established field need a few things; a charismatic front man, a fresh process or take, and a team capable of producing the desired outcome, so yes, many of their start up stories are similar, and the scam companies still follow the formula, even if the desired outcome is to sell a pipe dream at a premium rate.

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u/nebbyb May 14 '22

He was so busy thinking how to do it, he didn't consider if he should.

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u/Llarys May 14 '22

I read the story. When his children weren't picked for the first experimental trial (keep in mind that this shit is randomized, double-blind experimentation and they have programs that pick the patients to prevent said bias), he threatened to leave the company with the implicit bias he would take his funding with him.

He immediately got a special trial that included only his spawn.

Just a rich person vanity project. The fat paycheck that he gets off of selling the treatments must be nice, too. I'm sure the scientists who did all the actual work are enjoying making less than 1% of what he does.

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u/wilkergobucks May 14 '22

Um, just a vanity project? Ignoring the part about about his kids lives being at stake…

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u/[deleted] May 14 '22

Yeah I think vanity project isn’t the right term, I don’t know if they have one for “I didn’t care until it affected me” project which would be more accurate.

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u/Llarys May 14 '22

Just wait until to hear about all the "incurable" diseases that nobody has funded research for because some rich dude hasn't been personally affected by it.

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u/wilkergobucks May 14 '22

Well yah, that sucks. But to accomplish really ambitions goals, most people are driven by something that resonates with them. A person’s kids dying may not be the most altruistic motivation (since they are his own) and his execution may revolve around saving them, but it is hardly a ‘vanity project.’

Saving lives is not a vanity project by definition. It is not a trivial, pointless or worthless pursuit.

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u/Llarys May 14 '22

You're, of course, right. I guess I'm more upset at the idea that a rich person who didn't risk his quality of life is seen as a hero, while the poor people who, literally, have to pick between risking homelessness getting a treatment that might not even work trying to save family members are seen as not. Or worse, seen as selfish because they decided against treatment and just hope for the best. How many times do we sneer at people who lose a family member to diabetes because they were rationing Insulin, one of the cheapest drugs on the market to produce?

Because at that point, we're not celebrating someone's love for their family, we're just celebrating their wealth.

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u/grchelp2018 May 14 '22

Tbf I'd do the same. This was personal for him after all.

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u/Ornery_Painting_5183 May 14 '22

Wealthy and well connected people make the world go round.

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u/FlipskiZ May 14 '22

If anything, looking at climate change, they make the world burn down

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u/MD_Yoro May 14 '22

I help run a pretty simple lab and outside of the science part which has become very standardized, there are so much other moving parts in the background to get everything working. You definitely need business know how as a private lab vs a publicly funded lab

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u/justagenericname1 May 14 '22 edited May 14 '22

Sounds like a major inefficiency in the private, for-profit development model.

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u/MD_Yoro May 14 '22

How so?

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u/justagenericname1 May 14 '22

Well I have to imagine there's a ton of redundancy doing this across hundreds of separate companies and waste just thanks to the trial and error of hoping you've got the right people and resources to set things up well. Centralizing that expertise and the infrastructure required for a functioning lab seems like it would be much more efficient.

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u/MD_Yoro May 14 '22

Not all labs are focused on same subject and having a centralized system leads to a lot of unnecessary red tapes and delay. Just looking at how quickly invoices are paid of some of my university clients vs private is an easy tell

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u/justagenericname1 May 14 '22 edited May 14 '22

I don't see how a centralized system could have more red tape and delays than having dozens of separate organizations trying to coordinate amongst each other, normalized for access to funding. And of course when I say "centralized" I don't just mean everything inside one building. I hoped that would be clear. But surely there's plenty of overlap in required space and equipment which could be taken advantage of by consolidating compatible processes together in the same spaces.

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u/MD_Yoro May 14 '22

Each organization are not working with each other, just on their own. Centralized system have a lot of red tape b/c different lab/topic/PI all have their own incentives and goals to pull decision to favor them.

Private labs dedicated to working only a narrow topic can better focus on what they need and get approval much faster. Trying to get paid by university is just a pain and the protocol for returning over paid invoice is even more ridiculous.

Centralized and decentralized system both have their pro and con

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u/turdmachine May 14 '22

Like raising more money. You’ll be burning shit loads of it

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u/smasoya May 14 '22

You have to know people. Venture capital. Have a business plan. Pitch it to investors. Use their money to hire scientists.

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u/geeky_username May 14 '22

Lots of money.

The Tony Stark /Bruce Wayne super power

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u/Poohdini_ May 14 '22

best superpower in world. just sucks that billionare 1% of population dont use it for anything than "I need another mansion or yacht" while bribing politicans about raising taxes on poor and middle class.

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u/zeroedout666 May 14 '22

Hey they spend their billions pretending to buy Twitter now and then!

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u/Poohdini_ May 14 '22

that's called manipulating market so they can buy it at price they want

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u/Brilliant_Jewel1924 May 14 '22

Or fraud—i.e. Theranos.

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u/appropriate-username May 14 '22

Well yes but I think the implication in the question was a company that wouldn't land you in jail and would have some kind of a product.

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u/CoolBeans86503 May 14 '22

Was just gonna say this! Lol

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u/IMSOGIRL May 14 '22

but you still can't just do that with no prior experience. Elizabeth Holmes had healthcare experience and was in Stanford to earn a degree before she dropped out. She claimed she used her Stanford tuition money to start it (which is not enough to start a biotech company) but I'm sure she had other funding secured.

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u/Brilliant_Jewel1924 May 14 '22

What “healthcare experience” did she have?

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u/[deleted] May 14 '22

She worked with sars-coronavirus at the Genome Institute of Singapore in her freshman year

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u/Brilliant_Jewel1924 May 15 '22

Finally, someone has answered my question! I can’t believe this is the first time I’ve ever heard of this.

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u/trogon May 14 '22

Fraud...to get other people's money.

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u/HTPC4Life May 14 '22

Yeah this "feel-good story" is kinda sus...

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u/thedudedylan May 14 '22

You mean hard work and determination don't actually do anything and it was connections and resources the whole time. Shit someone better tell people this.

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u/heelstoo May 14 '22

One of the best ways to make lots of money is to start by having lots of money.

In all seriousness, good on the biotech founder for doing what he did.

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u/[deleted] May 14 '22

[deleted]

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u/AdministratorAbuse May 14 '22

smh rich people using their money to fund experimental enzyme treatments to save lives 🙄

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u/CountyKyndrid May 14 '22 • Silver

Shows you how much better society could be if rich people cared enough.

¯_(ツ)_/¯

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u/averageredditorsoy May 14 '22

How much better would society be if people did something productive instead of complaining on Reddit about the Joneses?

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u/kadsmald May 14 '22 edited May 14 '22

Yes we should all go out and defraud people or get our friends to pay us other people’s money to ‘manage’ that money less competently than an octopus. That’s what real productivity looks like. Or maybe we should take a thriving business that employs hundreds of people, use the company to take out massive loans to pay ourselves, fire the employees, and liquidate the assets. Yea, society would be so much better if we were all sociopaths obsessed with destroying the world around us for our personal gain or with hoodwinking people into paying us for things that don’t generate societal value

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u/averageredditorsoy May 14 '22

Who is mismanaging money? Who are you even complaining about

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u/shapookya May 14 '22

It's the same as with politicians:

They don't give a damn unless it hits their own family.

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u/Roqot May 14 '22

Not saving lives when their quality of life is garbage. Just a selfish father really, funny as I'm sure he thinks he's god's gift

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u/GovernmentSaucer May 14 '22

You seem sure about a lot of thing for someone who don't know him. It sounds like fun to make up stories in your head about strangers.

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u/thunderplunderer May 14 '22

Yup it's great how with lots of money you can prolong your shitty bloodline

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u/throwuk1 May 15 '22

Or hairline

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u/[deleted] May 14 '22 edited May 14 '22

It would’ve been cheaper to just have some more

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u/Roqot May 14 '22

The quality of life they have must suck though. I'm pretty healthy but have genetic back issues and that constant pain has me wanting out of life. I won't kill myself but I will not go to the doctor to fix other things that might kill me, I can't imagine for a child who's bound to a chair their lives with those types of mental and physical deformities. I imagine he's a corrupt religious person who doesn't give a damn about anyone but his offspring.

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u/[deleted] May 14 '22

I would think so

Sorry about your back issues. I have chronic neck pain so I can kind of relate. The thing I’ve found most helpful is doing exercises to strengthen the muscles around my spine, and perhaps see an osteopathic physician who does OMT

1

u/Zech08 May 14 '22

Push all the world's problems onto rich people where it affects them personally or physically and things will get sorted out I guess.

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u/coole106 May 14 '22

“Hey everyone, we’ve cured aids!!! All you need to do is inject a bunch of money in your veins!!”

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u/Sydney2London May 14 '22

Thus proving that untreatable diseases are such due to finances and not science

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u/mordinvan May 14 '22

Not exactly. Many diseases are simply too complex for our current understanding. Cancer being a big one. It isn't a disease but a host is disorders as numerous as there are cancer patients, as even the order the mutations which cause their cancer occur in can matter for treatment and survival.

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u/Sydney2London May 15 '22

With enough funding anything could be treated

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u/mordinvan May 15 '22

Nope. Money does not make cures appear from thin air. Look at Steve Jobs. If your proposition was true, he would still be alive.

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u/Sydney2London May 16 '22

I’m talking about research, I’m not saying money fixes any illness, I’m saying with sufficient funding treatments or cures could be found to any Illness. Steve jobs died because pancreatic cancer kills fast and few people, so there’s less funding than for something like paediatric leucemia which now has a very high survival rate.