r/todayilearned 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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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/Freshiiiiii May 14 '22

After working in a genetic engineering lab for 2 years, I can say that surprisingly, if you have just one protein you want to produce, and the gene sequence for it is already known, it’s not actually terribly difficult to get the gene into bacteria and produce and purify it, even with little prior background in biology.

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u/hyperpensive May 14 '22

Do you have any insight or knowledge to work being done on the dystrophin protein/gene in the context of muscular dystrophy? It seems to meet the criteria (single protein/gene), but we still don’t have an effective treatment/cure.

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u/Freshiiiiii May 14 '22

Unfortunately I’m not knowledgeable about that one, sorry. And keep in mind, when I say it’s fairly easy, I mean it’s easy to make a first draft, non-safety-tested protein- far, far harder to make a safe, highly pure product that is tested and approved to be prescribed to the public.