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Good Craziness and Bad Craziness

It’s fair to say that there’s a high level of excitement these days in biopharma (and the associated academic disciplines) due to the tools we have at our disposal these days. CRISPR and other gene-editing technologies, new looks at RNA, chromatin, and epigenetics, all sorts of unusual modes for altering cells and whole-animal phenotypes – there’s a lot going on, and there are a lot of frontiers that have opened up.

Frontiers are pretty wild places, though, and there are some pretty wild claims. How about editing genes without a double-strand break (as used in CRISPR)? What if it were a sort of super-selective viral vector, the sort of thing that gene-therapy folks have been searched for? That would be pretty interesting, and there’s a company (Homology Medicine) that says that they have just that, or something very close. But as this article at Technology Review shows, not everyone is buying into it. Well, some people are, to the tune of $127 million so far, but there are skeptics who think that the underlying science just doesn’t work.

This is a testable hypothesis, of course. The company will be working very hard to produce convincing results, and if they do, they can expect to see a lot more than $127 million. I’d be a little jumpy about investing at this point, though, because it’s certainly true that a lot of neat stuff doesn’t quite pan out. (The current research boom, looked at from that perspective, is due to a slightly higher-than-normal run of neat stuff actually working).

Homology Medicine, then, probably has enough money to demonstrate that they’re worth more money, which is about right. I would assume that they’re frantically engaged in just that process, trying to figure out what would be the most inarguable results that have the highest chance of being achieved in the shortest period of time without quite using up all the cash. That’s not an easy problem, but there are a lot of worse problems – for example, not being able to generate any of those results. As the article shows, there’s a not-dismissable chance that that could happen, too. But one way or another, we’ll find out. The people who have put up the $127 million will find out before the rest of us, but eventually, everyone who cares will know.

So this, to me, is still normal and acceptable – that’s how science works and how startups work. As you slide along the same scale, though, you find the pitches getting wilder and the investors getting more clueless. There’s a real dividing line between companies who are dealing with intelligent backers who are willing to take risks, and the ones who are actively trying to avoid people who will ask too many questions. In the first category, you have Homology – and most every other successful company in this business, if you trace them back far enough. You also have a huge pile of failures, too, the good ideas that didn’t pan out.

But the second category are the shady stem-cell clinics and the like, a vast range of scammers who promise everything and don’t want to deal with people who know anything about the details. If a company is upfront about what they’re trying to do, has data that they can present about how it’s working, and a clear plan for where to go next, then good luck to them. If it’s on a “trust us!” basis, though, or if the science behind it turns out to be a lot of gibberish, then you’re looking at trouble. You know what company straddled these two domains, ruinously? Theranos.

15 comments on “Good Craziness and Bad Craziness”

  1. Anon says:

    Crisp gene editing without CRISPR?

  2. Anon says:

    Theranos straddled no lines. They were always clearly in the second category from start to end.

    1. David Antonini says:

      Nah, they had a plan, and data. Not necessarily good/real data, or a ethical, long term viable plan…

      1. Isidore says:

        Even mobsters have a plan, to make/steal/extort money, and also data, the latter in the form of reputation and/or weaponry.

  3. emjeff says:

    I’m willing to bet that Patrick Vallence at GSK would throw tons of money at this. Look at what he spent on Sirtris (~700 million) , and what he got for it (0).

  4. Duncan Bayne says:

    Rand was on the ball with this one.

    “I don’t like people who speak or think in terms of gaining anybody’s confidence. If one’s actions are honest, one does not need the predated confidence of others, only their rational perception. The person who craves a moral blank check of that kind, has dishonest intentions, whether he admits it to himself or not.”

    1. milkshaken says:

      Rand was getting doped up to her gills on amphetamines. All she really knew since her traumatized childhood was that commies and big government were evil. And she never had any realistic appreciation how free market and capital operated, only romantic cartoonish notions of it. Her description of titans of industry as lonely and struggling geniuses is very flattering and her elitism is ironclad, thats why many privileged people like it despite its total lack of merit. It is a catnip for adolescent sociopaths dreaming to make it big

      1. Duncan Bayne says:

        Come on milkshaken, show us on the doll where Miss Rand touched you 😉

        1. milkshaken says:

          she hurt my elbow: I hurled the Atlas Shrugged across the room in a fit of rage, it was too shitty and too heavy

          1. Mad Chemist says:

            Good man, Atlas Shrugged is only good as a paperweight.

          2. Gordonjcp says:

            “Atlas Shrugged” is the only book I have ever destroyed, simply because I couldn’t stand the thought of anyone else reading that onanistic drivel.

          3. peptoidchemist says:

            bravo!

  5. luysii says:

    Here is some craziness, not from a company but from academics in Florida, California (UC San Diego) and Singapore [ Cell vol. 170 pp. 899 – 912 ’17]. If correct, they have found a way to treat MicroSatellite Repeat Expansion diseases (all 18) listed. These include horrors like ALS and Huntington’s chorea. It involves CRISPR, Cas9, AAV and guide RNAs. Cells from patients with 4 of the disease were partially normalized by the treatment. For more detail read the paper or have a look at https://luysii.wordpress.com/2017/08/31/18-at-one-blow-said-the-molecular-biologist/

  6. Bryan Lanning says:

    Yeah, it does not work in small scale of staff. Ive done it in an academic lab with only one CRISPR scientist and its bad ( Sabatini Alumni, but only one in a lab of 25 or so people) and it is BS. However, with a larger Crispr focused lab, you can get good quality controls working so the results mean something. See non-top ten biology departments but with people that think, instead of do whatever Harvard says.

  7. Passerby says:

    With the guy running the U.S. government doing it on a “Trust me” basis, can you be surprised to see companies going down the same route?

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