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Sanofi quits Voyager.
https://www.fiercebiotech.com/biotech/sanofi-quits-voyager-g...

Uniqure looking for a buyout.
https://www.fiercebiotech.com/biotech/uniqure-weighs-sale-af...

EDITAS still cant get out of their own way.

I was hoping SPARK would have a plug and play platform for eyeballs (genetic eye disease) - it wasnt so simple as their platform didnt work for a second genetic disease as well.

I question whether the market is saturated with gene therapy for rare genetic disease, and even at $1m / treatment with a baseline incidence of 1,000 / year for treatment, that's $1b / treatment... but there still does not seem to be a reliable "plug and play" platform yet, these trials take an amazing amount of time (not to mention single arm trials are often garbage!) and there is going to be competition amongst each other, likely leading to decreased profit margins and forcing companies to do more and more expensive trials to prove safety & efficacy +/- monitoring. It seems like the market & pharma are beginning to see that "it may not be so easy" and that a "let's see how this all plays out & pay more later" strategy is probably better than "spend less now and ask questions later & hope we win the lottery."

by the way, my calls on gene therapy?
CRSPR: down 9% since thumbs down March 2018
NTLA: down 31% since thumbs down April 2018
EDIT: down 48% since thumbs down March 2018


https://boards.fool.com/crsp-technicals-33018459.aspx
https://boards.fool.com/crsp-and-ntla-seem-like-all-hype-edi...
https://boards.fool.com/this-is-exactly-why-im-so-confused-b...

Have we just surpassed peak gene therapy? Or was that when there was a Freakonomics podcast on it? :P
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Fuma, how about Sangamo / SGMO?

https://www.fool.com/investing/2019/04/03/does-sangamo-have-...

SGMO's zinc finger nuclease gene editing sounds like it is be way more precise (safe/effective/less off-target changes) than what CSPR and EDIT are using.

At least 2 trials in progress in humans. SB-525 and ST-400.

Partnerships with Pfizer, Sanofi, and others.

Stock has been a dog lately, but I'm hanging in hoping for positive results from the ongoing trials.
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Sangamo has always been a dog. It's been around TWO DECADES and has changed targets a bunch. Sangamo is one of these companies that continues to miss deadlines, fail in trials, yet somehow doesnt go to bagel-town, and 5-10 years later reinvents itself, only to fail again. Geron is another. Novavax another. NKTR is likely going to be on that list. Sangamo was big on peripheral neuropathy for awhile. then onto HIV. then regenerative medicine (I think?) ... poor focus, poor execution, long history of failures.

There are some companies that fade into the background for long enough that the last wave of biotech newbies who got burned have abandoned biotech, only to feast on the next round of newbies.

It's an art, really.

Rulebook #10. If a company has changed therapeutic focus more than twice and doesnt have a marketed drug and has been public more than 5 years, run.
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Point taken on poor focus, execution.

I get the sense that if CEO Sandy Mcrae was replaced stock would jump for joy.

His word choice during presentations has not been comforting to investors.

Ever the optimist myself, I try to take that as ultra-cautious approach and making sure not to let stock expectations get ahead of the science.
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Rulebook #10. If a company has changed therapeutic focus more than twice and doesnt have a marketed drug and has been public more than 5 years, run.

Fair rule -- but in defense of the timelines, I would just note that with gene therapy we are in new world and approval times may stretch. We are talking about in-vivo gene editing of humans, a genetic change that could be with them for life, not a drug that will clear their system in a short time. FDA allowing testing of 1,2, 5 patients at a time. 5 year rule may not work in this space.

Another risks with the gene editing from investment standpoint, is it seems like many companies going after similar well-understood single gene issues, e.g. hemophilia, sickle cell, beta thalassemia. They may be on track to validate their platform but may not be first to market and if not best in class, may not get to market at all.
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How about Bluebird / BLUE?

Going to market in Europe with treatment for beta-thalassemia

https://www.ashclinicalnews.org/online-exclusives/bluebird-b...

But delayed until 2020:
https://www.biopharmadive.com/news/bluebird-delays-gene-ther...

Also working on sickle cell:
http://investor.bluebirdbio.com/news-releases/news-release-d...
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