Editas Medicine (Seite 8)

[Schaeffi]

Editas: The Most Comprehensive CRISPR Platform - But At What Cost?

https://seekingalpha.com/article/4183403-editas-comprehensiv…

Auszüge daraus übersetzt:

positiv:
Editas is the only preclinical CRISPR editing platform that incorporates multiple cutting enzymes: Cas9 and Cpf1. Delivery is the key that unlocks the tissue of interest to be edited. The Editas platform has a much broader delivery toolkit than CRISPR or Intellia, incorporating adeno-associated viruses or AAVs, lipid nanoparticles or LNPs and electroporation for delivery.

They can deliver DNA, RNA or RNP to effect genome editing. In short, this delivery toolkit is more comprehensive than either CRISPR or Intellia and is gearing up to deliver not only to the eye for their near-term pipeline, but also the liver, lung, bone marrow and muscle. Their lead program is expected to file for an IND soon which could result in the first CRISPR clinical trial.

Editas ist die einzige präklinische CRISPR-Editing-Plattform, die mehrere Schneideenzyme enthält: Cas9 und Cpf1. Die Lieferung ist der Schlüssel, der das zu bearbeitende Gewebe freigibt. Die Editas-Plattform verfügt über ein viel breiteres Delivery-Toolkit als CRISPR oder Intellia, das adeno-assoziierte Viren oder AAVs, Lipid-Nanopartikel oder LNPs und Elektroporation zur Bereitstellung enthält.

Sie können DNA, RNA oder RNP liefern, um das Genom zu bearbeiten. Kurz gesagt, dieses Toolkit ist umfassender als CRISPR oder Intellia und bereitet sich darauf vor, nicht nur das Auge für ihre kurzfristige Pipeline, sondern auch die Leber, die Lunge, das Knochenmark und die Muskeln zu versorgen. Es wird erwartet, dass ihr Leitprogramm demnächst eine IND beantragen wird, was zu der ersten klinischen Studie mit CRISPR führen könnte.

Übersetzt mit www.DeepL.com/Translator




negativ:
A bigger issue is the success payments which are tied to the company market capitalization. In effect, these success payouts are triggered by market cap increases representing a tax on shareholders. Worse yet is the over $50 million spent over the past three years defending IP that has already been licensed.

Ein größeres Thema sind die Erfolgszahlungen, die an die Marktkapitalisierung des Unternehmens gebunden sind. Tatsächlich werden diese Erfolgsauszahlungen durch die Erhöhung der Marktkapitalisierung ausgelöst, die eine Steuer auf die Aktionäre darstellt. Schlimmer noch sind die über 50 Millionen Dollar, die in den letzten drei Jahren für die Verteidigung von IP ausgegeben wurden, die bereits lizenziert wurden.

Übersetzt mit www.DeepL.com/Translator



und noch ein weiterführender link draus
Genomic Medicine: Catch The Gene Therapy Wave
https://seekingalpha.com/article/4173868-genomic-medicine-ca…


S.

[Schaeffi]

Gene-Editing Selloff Is Overdone

https://www.barrons.com/articles/gene-editing-selloff-is-ove…


bleibe dabei, CRISPR wird mächtig.

S.

[belliora]

http://www.scinexx.de/wissen-aktuell-22826-2018-06-12.html

[Schaeffi]

A serious new hurdle for CRISPR: Edited cells might cause cancer, find two studies by Statnews .com

hier ein Auszug aus dem "Bösen Artikel"

Hmm wollten hier welchen den Shorts in die Hände spielen oder einfach den
Ausbruch der Aktien noch stoppen??? Fragen über Fragen !!!




Editing cells’ genomes with CRISPR-Cas9 might increase the risk that the altered cells, intended to treat disease, will trigger cancer, two studies published on Monday warn — a potential game-changer for the companies developing CRISPR-based therapies.

In the studies, published in Nature Medicine, scientists found that cells whose genomes are successfully edited by CRISPR-Cas9 have the potential to seed tumors inside a patient. That could make some CRISPR’d cells ticking time bombs, according to researchers from Sweden’s Karolinska Institute and, separately, Novartis.

CRISPR has already dodged two potentially fatal bullets — a 2017 claim that it causes sky-high numbers of off-target effects was retracted in March, and a report of human immunity to Cas9 was largely shrugged off as solvable. But experts are taking the cancer-risk finding seriously.

The CEO of CRISPR Therapeutics, Sam Kulkarni, told STAT the results are “plausible.” Although they likely apply to only one of the ways that CRISPR edits genomes (replacing disease-causing DNA with healthy versions) and not the other (just excising DNA), he said, “it’s something we need to pay attention to, especially as CRISPR expands to more diseases. We need to do the work and make sure edited cells returned to patients don’t become cancerous.”

Another leading CRISPR scientist, who asked not to be named because of involvement with genome-editing companies, called the new data “pretty striking,” and raised concerns that a potential fatal flaw in some uses of CRISPR had “been missed.”

Related: CRISPR advances are coming fast. Here’s your guide
On the other hand, the Novartis paper has been available in preliminary form since last summer, and CRISPR experts “haven’t freaked out,” said Erik Sontheimer of the University of Massachusetts Medical School, whose CRISPR research centers on novel enzymes and off-target effects. “This is something that bears paying attention to, but I don’t think it’s a deal-breaker” for CRISPR therapies.

The Karolinska and Novartis groups tested CRISPR on different kinds of human cells — retinal cells and pluripotent stem cells, respectively. But they found essentially the same phenomenon. Standard CRISPR-Cas9 works by cutting both strands of the DNA double helix. That injury causes a cell to activate a biochemical first-aid kit orchestrated by a gene called p53, which either mends the DNA break or makes the cell self-destruct.

Whichever action p53 takes, the consequence is the same: CRISPR doesn’t work, either because the genome edit is stitched up or the cell is dead. (The Novartis team calculated that p53 reduces CRISPR efficiency in pluripotent stem cells seventeenfold.) That might explain something found over and over: CRISPR is woefully inefficient, with only a small minority of cells into which CRISPR is introduced, usually by a virus, actually having their genomes edited as intended.

“We found that cutting the genome with CRISPR-Cas9 induced the activation of … p53,” said Emma Haapaniemi, the lead author of the Karolinska study. That “makes editing much more difficult.”

The flip side of p53 repairing CRISPR edits, or killing cells that accept the edits, is that cells that survive with the edits do so precisely because they have a dysfunctional p53 and therefore lack this fix-it-or-kill-it mechanism.

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The reason why that could be a problem is that p53 dysfunction can cause cancer. And not just occasionally. P53 mutations are responsible for nearly half of ovarian cancers; 43 percent of colorectal cancers; 38 percent of lung cancers; nearly one-third of pancreatic, stomach, and liver cancers; and one-quarter of breast cancers, among others.

The Novartis team was trying to see how it could increase the efficiency of CRISPR editing of pluripotent stem cells. Because this kind of stem cell can morph into virtually any kind of cell, it might be able to treat a variety of diseases. Neuroscientist Ajamete Kaykas of the company’s Institutes for BioMedical Research in Cambridge, Mass., got CRISPR’s efficiency at inserting or deleting chunks of DNA up to 80 percent. Unfortunately, when CRISPR worked, it was because p53 didn’t, which raises cancer concerns.

As a result, the Novartis paper concludes that “it will be critical to ensure that [genome-edited cells] have a functional p53 before and after [genome] engineering.” The Karolinska team warns that p53 and related genes “should be monitored when developing cell-based therapies utilizing CRISPR-Cas9.”

The p53 finding doesn’t mean CRISPR is toast. For one thing, “the two papers present preliminary results,” biochemist Bernhard Schmierer of the Karolinska, co-leader of its study, told STAT. “It is unclear if the findings translate into cells actually used in current clinical studies.”

For another, the p53 problem might be worse with Cas9 than with other DNA-cutting enzymes used in CRISPR. And, crucially, it probably affects only one avenue of genome-editing.


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CRISPR edits genomes in either of two ways. It slices out a chunk of disease-causing DNA, in a process called non-homologous end joining (NHEJ), or gene disruption. That’s how CRISPR Therapeutics is going after sickle cell disease. Alternatively, CRISPR both cuts out a disease-causing stretch of DNA and replaces it with healthy nucleotides, in homology-directed repair (HDR), or gene correction. Several university labs are investigating HDR to treat Duchenne muscular dystrophy, among many other diseases.

In the normal, mature cells she and her team studied, Haapaniemi said, gene disruption “can happen even when p53 is activated.”

That’s good news for CRISPR Therapeutics’ sickle-cell and thalassemia programs as well as for Editas Medicine’s lead product, targeting a form of blindness, and others in its pipeline, all of which use NHEJ gene disruption. It also should not affect the gene-disruption approach that Intellia Therapeutics and Regeneron are taking to transthyretin amyloidosis.

CRISPR-based editing of T cells to treat cancer, as scientists at the University of Pennsylvania are studying in a clinical trial, should also not have a p53 problem. Nor should any therapy developed with CRISPR base editing, which does not make the double-stranded breaks that trigger p53. Developed by Harvard’s David Liu, base editing replaces a wrong DNA “letter” with the right one, without cutting, and is the basis for startup Beam Therapeutics.

The p53 problem, however, might affect other products that companies hope to develop via gene correction, including glycogen storage disease, cystic fibrosis, and severe combined immunodeficiency.

It’s also a potential problem for stem cells. There, the Novartis team showed, p53 inactivation seems to be necessary for both NHEJ disruption and HDR correction. (Novartis’ Kaykas said he could not speak to a reporter without clearance from the company’s communications office.) That could be an issue for therapies using CRISPR’d stem cells: The same dysfunctional p53 that allows CRISPR to work its magic also makes cells likely to become cancerous.


Quelle Statnews.com


Gruss S.

[belliora]

Jetzt habe ich die Schnauze voll vom Thema: weg damit...
https://www.statnews.com/2018/06/11/crispr-hurdle-edited-cel…

[belliora]

Wobei die Bude nicht mit hoch lief, aber dafür im Schlepptau mit runter knallt, starke Aktie.....lol

[belliora]

https://www.technologyreview.com/the-download/611271/fda-hal…

alles nur reine Zockerbuden...absolut abhängig von externen Faktoren.....

[belliora]

Antwort auf Beitrag Nr.: 57.828.671 von Schaeffi am 25.05.18 06:32:42

Für 6 Millionen Dollar, wow, da liege ich ja nur knapp drunter....

[Schaeffi]

Top Performing Investor Adds Editas Medicine (EDIT), Sienna Biopharmaceuticals (SNNA) to Its Portfolio

https://www.insidermonkey.com/blog/top-performing-investor-a…

[belliora]

hab ich selber auch, mit Crispr hätte ich 200%.....man muss ja ziele haben.....