"Founded in 2012, Moderna reached unicorn status — a $1 billion valuation — in just two years, faster than Uber, Dropbox, and Lyft, according to CB Insights. The company’s premise: Using custom-built strands of messenger RNA, known as mRNA, it aims to turn the body’s cells into ad hoc drug factories, compelling them to produce the proteins needed to treat a wide variety of diseases. But mRNA is a tricky technology. Several major pharmaceutical companies have tried and abandoned the idea, struggling to get mRNA into cells without triggering nasty side effects."
They kept trying, like the little engine that could, focusing on one disease, but...
" It never proved safe enough to test in humans, according to several former Moderna employees and collaborators who worked closely on the project. Unable to press forward with that technology, Moderna has had to focus instead on developing a handful of vaccines, turning to a less lucrative field that might not justify the company’s nearly $5 billion valuation."
The reason they turned to vaccines is that vaccines are only given once, so the toxic effects don't add up the way that meds for chronic illnesses do. But vaccines aren't as profitable.
"The indefinite delay on the Crigler-Najjar project signals persistent and troubling safety concerns for any mRNA treatment that needs to be delivered in multiple doses, covering almost everything that isn’t a vaccine, former employees and collaborators said."
Oh dear, what was going to happen to poor Moderna!
"“[The technology] would have to be a miraculous, Hail Mary sort of save for them to get to where they need to be on their timelines,” one former employee said. “Either [Bancel] is extremely confident that it’s going to work, or he’s getting kind of jittery that with a lack of progress he needs to put something out there.....
In order to protect mRNA molecules from the body’s natural defenses, drug developers must wrap them in a protective casing. For Moderna, that meant putting its Crigler-Najjar therapy in nanoparticles made of lipids. And for its chemists, those nanoparticles created a daunting challenge: Dose too little, and you don’t get enough enzyme to affect the disease; dose too much, and the drug is too toxic for patients."
The Hail Mary wasn't coming through research and development. They needed something else, something hyped and mandated. Whatever could that be?
The CEO, Stéphane Bancel, thought it would come by 2017, but he had to wait another 4 years for the company-saving miracle to manifest.
“I’m sure that five years from now we’ll look at 2017 as the inflection point that Moderna went for a liftoff,"Bancel said. “We have a chance to transform medicine, and we won’t quit until we are done and we have impacted patients.”
And they did. They have impacted patients in ways the patients never expected, and no doubt would not have wanted, but the important thing is that those pesky safety signals are no longer a problem for profits.
Oh. They weren't. But now a new sheriff is in town. Uh, oh!
https://www.statnews.com/2017/01/10/moderna-trouble-mrna/
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