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ProQR Therapeutics 2019

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Tom.. I wrote few things that are so important to why the price of Proqr went up and may go up even much more and much faster. but needs one to "hear"
Tom3
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quote:

joel12 schreef op 13 juli 2019 05:22:

Tom.. I wrote few things that are so important to why the price of Proqr went up and may go up even much more and much faster. but needs one to "hear"
Thanks for the reminder. That is why I have invested decent amounts of money in ProQR, Arrowhead and AFMD. Their science could be game changers. Strong management is a bonus. In the end Big Pharma must be willing to buy these companies. Only very few will escape and emerge as runner-up big pharma. Arrowhead fits in this picture. ProQR too if they show improvement is some areas. AFMD is my wild card.
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you take a stab at Proqr almost at every corner Tom.
Proqr has done in the shortest time what Arwr and definetly AFMD have not in their wildest dreams.

Afmd is a 20+ years long company with currently 1 clinical candidate. during the 20 years has scraped out at least 5 candidates.

Arwr also has been around for a long time and may have a chance ,, though who knows? its technology is not highly differentiated from alny, drna, abus
but in the past months Gilead said it will buy out an RNAi company and its valuation went up . has also a multi million dollar partnership so its more "safe"

anyway. the idea is that there is only one Proqr. and it does majic. dont mix it with substitutes.
Tom3
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www.pharmasalmanac.com/articles/what-...

De Boer lijkt het Axiomer platform naar voren te gaan schuiven:

Daniel A. de Boer,
Chief Executive Officer, ProQR Therapeutics N.V.
A: One truly novel technology that could have significant impact on the lives of patients in the future is the use of RNA editing. Targeted RNA editing has the potential to directly correct the causes of genetic conditions. While therapies are still in the early stages of development, RNA editing potentially combines the benefits of both gene therapy and small molecule therapies. Like gene therapy and gene editing, RNA editing can specifically repair a disease-causing gene mutation but can do so without permanently changing a person's genetic code, reducing the chance of permanent off-target effects. Additionally, like small molecule drugs and unlike gene-editing therapies, small molecule RNA-editing therapies can often be administered in simple fashion and delivered without viral vectors. There are thousands of diseases caused by genetic mutations that can be corrected with RNA editing, and we have only just begun to witness the impact these types of therapies can have on the lives of people suffering from devastating genetic conditions.
Tom3
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quote:

joel12 schreef op 13 juli 2019 11:38:

you take a stab at Proqr almost at every corner Tom.
Proqr has done in the shortest time what Arwr and definetly AFMD have not in their wildest dreams.

Afmd is a 20+ years long company with currently 1 clinical candidate. during the 20 years has scraped out at least 5 candidates.

Arwr also has been around for a long time and may have a chance ,, though who knows? its technology is not highly differentiated from alny, drna, abus
but in the past months Gilead said it will buy out an RNAi company and its valuation went up . has also a multi million dollar partnership so its more "safe"

anyway. the idea is that there is only one Proqr. and it does majic. dont mix it with substitutes.
You forgot this one:
ir.arrowheadpharma.com/static-files/5...

AFMD has closed a significant partnership with Genentech/Roche.

ProQR still has no partner. Do you think this will change soon?
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i dont forget anything.
ARWR first of all failed its first main trial and now hopes to do better with a combination.. y know that?
arwr has a similar maybe identical technology as DRNA and ABUS which last has 1/10 or less valuation.

yes ARWR and AFMD did partnerships so what? AGTC had a 1 bill dollar partnership that felt apart ,,, the examples of companies to had done billion dollars partnerships and ultimately go down in flames is loooooooong.
OMED .. oncomed.. read about it.. billion dollars partnerships with Celgene.. done. zero.

the AFMD partnership may or may not seek value for the company. dont know .. if it will it will take years to see that. i think best for t company to get bought out.

in any case in most cases the upside of the companies is before the partnerships are done not after. case in point AFMD went up from 1.5 to 7. in the past FATE went p from 1 to 8 or so when Juno did partnership w it.. and went down and then took a long time to go back up again.

if you ask weather proqr will do a partnership? hhhhhh no it will never do ... :)
Tom3
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ABUS went from $11 to $ 2,40 in one year so I doubt they have identical technology compared to ARWR (that went from $ 16.40 to $ 27.15 in one year). ProQR went from $ 23 (March 2015) to $ 3 (March 2018) due to successes? But may be it is now time to invest in ABUS.
4finance
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quote:

Tom3 schreef op 13 juli 2019 18:42:

ABUS went from $11 to $ 2,40 in one year so I doubt they have identical technology compared to ARWR (that went from $ 16.40 to $ 27.15 in one year). ProQR went from $ 23 (March 2015) to $ 3 (March 2018) due to successes? But may be it is now time to invest in ABUS.
Dat ABUS heb ik ook al eens naar gekeken. Is dat een gokje waard?

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ARWR also had the same descent what in the world are you talking about? finviz.com/quote.ashx?t=ARWR&ty=c...

and stop taking stabs at Proqr because that doesnt make you any smarter.

as to that one company went up while the other went down .. that doesnt immediately say about their respective technologies being the same or not. biotech investing is more complicated than going to the supermarket and judging based on price which product is better.
Tom3
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4finance schreef op 13 juli 2019 19:03:

[...]

Dat ABUS heb ik ook al eens naar gekeken. Is dat een gokje waard?

AB 729 is hun nieuwe troefkaart na teleurstellende resultaten van ARB 1467. Ik heb geen idee of dat gaat werken:

www.evaluate.com/vantage/articles/new...

Blackrock zit er nog in met een flinke portie aandelen, wellicht is het toch niet zo slecht als de meesten op de beurs nu denken.
Tom3
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De Troon Jan schreef op 13 juli 2019 23:30:

En nu maandag zakken?
Hoezo? Nabeurs was er nog een levendige handel waar tot $ 9,50 per aandeel werd betaald. Ik heb echter geen hoera verhalen op social media etc gezien tot nu toe. We moeten het maar afwachten.
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There is a big chance that the biotech index XBI will start the week down. thats because technically, altough ready to breakout, its against resistance and looks a bit weak at least for the next day or so.

If the buys in Proqr where in expectation of some meaningful news from the weekends conference .. and nothing , which most likely thats the case, will come out,, Monday the stock will go down as those that bought, maybe day traders will sell .

however , there are many possible other scenarios .. and could be that Proqr not only will not go down, but rather explode on news that should be in the area of business development.

so
1) if the jump was due to day traders or swing traders expecting news from the weekend .. it will go down Monday.
2) but if the buys where insiders knowing what will happen outside of the weekends presentation.. it will go up and continue based on the magnitude of the upcoming news.

i am ready for both scenarios. hope however the 2) will happen.
Hunter300
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July 12, 2019 08:35 AM EDT
R&D
Broad star Feng Zhang un­veils a new CRISPR plat­form, edit­ing RNA and elim­i­nat­ing Alzheimer's threat — in cells
Broad In­sti­tute star sci­en­tist Feng Zhang is back in the spot­light, adapt­ing CRISPR tech­nol­o­gy in a shift from per­ma­nent­ly edit­ing DNA to re­vis­ing RNA — tem­porar­i­ly if need­ed. And he il­lus­trat­ed the promise of this ap­proach by de­ac­ti­vat­ing APOE4, which may be a tick­ing time bomb for peo­ple at risk of de­vel­op­ing Alzheimer’s.

CRISPR/Cas9 gene edit­ing tech has tak­en the lab by storm, in part be­cause of the work Zhang and his one-time col­leagues Jen­nifer Doud­na and Em­manuelle Char­p­en­tier ac­com­plished. They’re still scrap­ping over the patents to the orig­i­nal Cas9 work. But Zhang, who found­ed Beam Ther­a­peu­tics with David Liu and Kei­th Joung, has moved on in search of bet­ter tech, and in a pa­per pub­lished in Sci­ence, says they have made re­al progress in switch­ing from DNA to RNA edit­ing.

They call this new ad­vance RES­CUE: RNA Edit­ing for Spe­cif­ic C to U Ex­change. And it builds on RE­PAIR: RNA Edit­ing for Pro­gram­ma­ble A to I.

Us­ing Cas13, Zhang’s team was able to take the APOE4 gene — be­lieved to car­ry the added risk of spurring Alzheimer’s — and changed it to a be­nign APOE2. The RNA ed­i­tors con­vert­ed “the nu­cleotide base ade­nine to in­o­sine, or let­ters A to I. Zhang and col­leagues took the RE­PAIR fu­sion and evolved it in the lab un­til it could change cy­to­sine to uri­dine, or C to U.”

But there are al­so ways to achieve a tem­po­rary change that could ben­e­fit pa­tients with­out cre­at­ing po­ten­tial risks.

In a sep­a­rate cell ex­per­i­ment, Zhang and his group were able to or­ches­trate a tran­si­to­ry spike in ß-catenin ac­ti­va­tion and cell growth. That kind of tem­po­rary im­pact could erase threats of can­cer, as­so­ci­at­ed with un­con­trolled cell growth while treat­ing wounds.

“To treat the di­ver­si­ty of ge­net­ic changes that cause dis­ease, we need an ar­ray of pre­cise tech­nolo­gies to choose from. By de­vel­op­ing this new en­zyme and com­bin­ing it with the pro­gram­ma­bil­i­ty and pre­ci­sion of CRISPR, we were able to fill a crit­i­cal gap in the tool­box,” says Zhang, the James and Pa­tri­cia Poitras Pro­fes­sor of Neu­ro­science at MIT.

It’s an in­trigu­ing ex­per­i­ment, but don’t look for the ex­per­i­ment in cells to make the leap in­to prac­tice any­time soon. MIT’s Jonathan Gooten­berg summed it up for WBUR:

“It’s a first step in a very large jour­ney. We’re still at the base of the moun­tain, you might say.”

AUTHOR

This article was posted on Twitter a few days ago by John Carroll (Endpoints news). I think it shows that even the DNA editing guru’s see a huge potential in RNA editing.
holenbeer
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Tja, dan kan je voor de zekerheid maar beter wat spreiden in recent afgestrafte RNA-clubs, lijken mensen te hebben gedacht.
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right on! it also says clearly that in the brain better to modify the RNA and not the DNA

to add to that the words of Proqr CEO here www.pharmasalmanac.com/articles/what-...

are relevant and important.

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big congrats to probably the worlds best biotech company GLPG for the todays gilead upsized partnership. i just wonder if todays proqr buys where not due to that.. and how it will work out between the 2 neighbors
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