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Based on CTX110's data. I view it as positive, and based on the profile, the company plans to expand into a potential registrational trial that incorporates consolidation dosing in Q1 2022.
I bought the AH dip without knowing what caused it. Anyone know what's up?
CTX-110 clinical data event (Oct 12) https://www.veracast.com/webcasts/clients/webcasts/1Ava18.cfm
SITC:
CRSP presentation:
Title: CRISPR/Cas9 gene-edited allogeneic CAR-T cells targeting CD33 show high preclinical efficacy against AML without long-term hematopoietic toxicity
Abstract Number and Type: 133, poster
Date and Time: Friday, November 12, 2021, 7:00 a.m. – 8:30 p.m. ET
Presented jointly with NKTX:
Title: A combined strategy of CD70 CAR co-expression with membrane-bound IL-15 and CISH knockout results in enhanced NK cytotoxicity and persistence
Abstract Number and Type: 16439, oral
Date and Time: Wednesday, November 10, 2021, 2:40 p.m. ET
Title: CISH gene-knockout anti-CD70-CAR NK cells demonstrate potent anti-tumor activity against solid tumor cell lines and provide partial resistance to tumor microenvironment inhibition
Abstract Number and Type: 113, poster
Date and Time: Friday, November 12, 2021, 7:00 a.m. – 8:30 p.m. ET
CRISPR Therapeutics to Participate in the Chardan's 5th Annual Genetic Medicines Conference
BusinessWire, GlobeNewswire and PR Newswire News
8:00 AM ET
CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that members of its senior management team are scheduled to participate in the Chardan's 5th Annual Genetic Medicines Conference on Monday, October 4, 2021 at 2:00 p.m. ET.
A live webcast of the event will be available on the "Events & Presentations" page in the Investors section of the Company's website at https://crisprtx.gcs-web.com/events. A replay of the webcast will be archived on the Company's website for 14 days following each presentation.
The company anticipates filing of lead candidate CTX-001 in 18 to 24 months. More than 45 patients have been dosed across two trials and enrollment should be completed by the end of the year (we might get more data at ASH).
In addition, the company plans to report additional PhI data this year on CTX-110. Also, top-line PhI data from three trials by the end of the year on CTX-120 and CTX-130 (being tested in two trials).
Head n shoulders pattern back then
You were compelling. You influenced me to sell! DAMN
They also have a number of collaboration, one of which is with Stride Bio, whose platform allows them to modify AAV surfaces which are no longer recognized by pre-existing neutralising antibodies, modify binding sites to alter the tissue specificity, and engineer capsids to enhance potency as well as transduction efficiency.
In addition, they have in-licensed novel (AAV) vectors from Duke Uni and an IgG-degrading enzyme to clear neutralising antibodies https://insight.jci.org/articles/view/139881
This publication includes information on the company's development of proprietary small Cas9 variants which may allow for more efficient delivery in vivo using viral delivery vehicles. They expect to move multiple programs (a number of which are partnered) utilising in vivo approaches (both viral and non-viral) into the clinic in the next 18 to 24 months https://www.nature.com/articles/s41467-021-24454-5
Covered at $121 from $155.
For full discloser, are you short CRSP?
And at what price will you cover.
Bail while you have a prayer.
JMO
Crispr Therapeutics: Get Ready To Bail Out
Jul. 05, 2021
Summary
The company is lagging behind established pharmaceutical companies.
Other cheaper alternatives are on the way.
The company’s valuation is excessive.
Preamble
CRISPR Therapeutics (NASDAQ:CRSP) is a company that has the kind of valuation that investors in pets.com could only have dreamed about, even at the peak of the “dot.com” hysteria. For many owners of CRSP stock, the rich valuation for the company is more than justified since they believe that CRISPR has the “silver bullet” for malignant tumors and will therefore turn stock holders into gazillionaires in due course. I’m afraid, I’m not so sure. My primary reason for being doubtful is that there are already established companies providing CAR-T treatments and there is a plethora of equally effective and relatively low-cost remedies on the horizon. Whilst it is true that CRISPR is developing treatments for a number of disease areas, my focus in this article is their anti-cancer product line.
An overview of current cancer research
To fully explain my perspective, I believe it would be helpful to give a brief synopsis of the state-of-the-art advances. Broadly speaking, investigators in cancer research are developing therapies that can be categorized as small molecules, large molecules or CAR-T treatments.
Over decades of research, many of the cellular mechanisms that lead to cancer have become understood and drugs that target these processes have been made available. For instance, it has been known for quite some time that tyrosine kinase, an enzyme necessary for cell division, cannot switch off in some cancers due to a faulty structure. In recent years, a number of novel compounds have been approved that can inhibit these defective enzymes. One such compound is gilteritinib, which sells under the trade name Xospata and is produced by the Japanese biotechnology company Astellas Pharma (OTCPK:ALPMF). This product would be called a small molecule since it can enter a cancerous cell to perform its action. As you may imagine, the problem with small molecules is that they can enter healthy as well as abnormal cells, and this is the primary reason for the side effects often associated with cancer treatments. Additionally, these small molecules cannot cure the underlying problem, but merely slow its progress, and so must be taken for an extended period of time; often for life. For those unfortunates that have to be prescribed this medication, the cost is quite steep at around $25,000 a month.
The mode of action of large molecules is quite different, they attempt to activate a person’s own immune system to destroy the cancer. These large molecules consist of two parts, a component that locks onto the malignant cell and another that activates the immune response.
The majority of readers may be familiar with the idea that cancer is caused by flawed genes that then leads to uncontrolled reproduction of cells. Coincidentally, each flaw is often represented by a unique lipid or protein or polysaccharide on the surface of a cancerous cell known as a tumor-specific antigen. In fact, researchers can typically determine which gene is defective according to the antigen on the outside of a cell.
So, scientists are trying to produce large molecules (antibodies) that can bind to these specific antigens and then cause the immune system to destroy those cells. There are several methods being explored to introduce these tumor-specific antibodies into patients.
One such method is by the use of mRNA that is coded to enable cells within the patient to manufacture the antibody. I guess by now many readers have heard of mRNA given the extensive coverage that has been given to mRNA-based COVID 19 vaccines. For those who are unsure of the mechanism of action, put simply, mRNA vaccines induce cells to produce the “spike” protein of the virus and so coax an effective immune response to the actual virus. One company investigating the use of mRNA to fight cancer is BioNTech (NASDAQ:BNTX), the COVID 19 vaccine partner of Pfizer (NYSE:PFE). Initial results are more than encouraging; fewer adverse reactions and impressive improvements in patient outcomes. According to the company, it is envisaged that the costs associated with the treatment are expected to be relatively low, although this is difficult to tell given the company do not, as yet, have a product on the market.
Other biotech companies are synthesizing specific antibodies directly and then administering them to patients. There is a “penny stock” British biotechnology company called Scancell (OTC:SCNLF) that has demonstrated extremely encouraging conclusions following their phase 1/2 study for their lead melanoma product, SCIB1. Using their proprietary technology, “T cell responses were induced in nearly 90% of patients with no serious adverse events or dose limiting toxicities." Even though patients had stage III and IV cancer, around half were still breathing 5 years after the study had started.
Finally, there is CAR-T research that involves editing the genes of cells within the immune system so that these cells can recognize cancer cells and then attack them. You might be forgiven for thinking that this research is so sophisticated that very few companies have the capability to employ such technology to develop this kind of treatment. However, in actual fact there are around a hundred such companies around the world doing similar work to CRISPR.
On a recent conference call, CEO Samarth Kulkarni described progress thus far with their lead immuno oncology products. At the time of writing, the company’s principal oncology products are only in phase 1 testing. Needless to say, it will be several years before these products will be generating any income for the company.
Competitive CAR-T products
Let us consider one of CRISPR's blockbuster hopes, CTX110, which, as I mentioned, is several years away from being available for doctors to recommend to their patients. This is assuming that efficacy and safety can be confirmed, which is not a forgone conclusion. For those who are unfamiliar with CTX110, it is envisaged that it will be a one-treatment-only fix for B-cell blood plasma cell cancer.
Sounds great, right? A once-only treatment for blood cancer a few short years away. The only problem is that there are already two enormous companies manufacturing approved CAR-T therapies for B-cell blood cancer; Novartis (NYSE:NVS) and Gilead (NASDAQ:GILD). Furthermore, way back in 2018, it was reported that there were more than 200 ongoing clinical trials on CAR-T therapies, chiefly to tackle hematological cancers (lymphoma, leukemia, and myeloma).
It was announced in June 2020 that Gilead had received the European Medicines Agency approval for their CAR-T product, Yescarta. Since then, they have built treatment and production centers all around the world. On the market for only a short period of time, growth for the Gilead therapy has been an impressive 36% Y-O-Y. Originally brought to market by Novartis in 2018, Kymriah is now manufactured in across 5 sites globally, including Japan.
Compared to mRNA cancer vaccines and directly administered antibodies, the cost of manufacturing CAR-T cells is huge. For instance, a single treatment with Kymriah can cost around half a million dollars, then there may be further costs associated with continuing care.
To sum up
CRISPR Therapeutics is a company researching in the field of CAR-T medication with around $2 billion in the bank and almost zero revenue. On top of that, the company is burning through approximately $400 million a year in research costs. Despite this, its market cap is at $12.5 bn.
Competition for the company’s lead oncology target is already established globally in the shape of recognized pharmaceutical giants. If this were not bad enough, alternatives to the pricey CAR-T treatments are under development.
Yeah, I checked the price this morning and then checked a short while ago and there was a huge runup without any news that I could find.
Some big buys coming in today. $CRSP
This is from today's ARK Investments weekly newsletter:
At the Peripheral Nerve Society Conference this weekend, Intellia Therapeutics made history by presenting the first data of a CRISPR Cas9 based in vivo gene editing therapy in patients with Hereditary Transthyretin Amyloidosis (hATTR). Please find the results of this milestone trial in this New England Journal of Medicine publication.
hATTR is a disease in which amyloid proteins build up and, if untreated, can cause multiple organ failures. Typically, the primary impact is on the heart and nerves.
Until now, patients with hATTR faced limited curative treatment options. Alnylam’s Patisiran knocks down serum TTR by 80%, but greater knock down rates could enhance the clinical benefits.
Intellia Therapeutic’s presentation highlighted that editing the TTR gene could result in a one-time treatment, eliminating the need for chronic therapy. In a small six-person dose escalation study, the company showed that, in the second cohort at 0.3 mg/kg, three patients achieved an 87% average mean reduction in TTR and a max reduction of 96%, surpassing that of Patrisiran and other therapies. In our view, as Intellia escalates the therapeutic dose, higher reductions in TTR will lead to better patient outcomes.
We believe this data will enter the history books, signifying that in vivo gene editing and one-time cures are possible. The only question remaining is will it be durable?
It looks like it is going to be a very good day for CRSP.
Intellia Shares Climb on Positive Gene-Editing Data
By Matt Grossman
Intellia Therapeutics Inc. shares soared Monday morning after the company reported positive interim data from a Phase 1 clinical study of a gene-editing candidate treatment.
Shares rose 43% in pre-market trading after closing last week at $88.83.
The candidate treatment, NTLA-2001, is being developed for transthyretin amyloidosis. It is the first CRISPR/Ca9-based therapy candidate to be administered via intravenous infusion for precision editing of a human gene, Intellia said.
The treatment is meant to deactivate a gene in liver cells to prevent production of a protein that causes the sometimes-fatal complications of amyloidal.
Dr. John Leonard, Intellia's chief executive, said that the company's data also serve as a promising foundation for treating a variety of other genetic diseases with Intellia's platform.
Intellia is working with Regeneron Pharmaceuticals Inc. to develop NTLA-2001.
Breaking through that 136.00 resistance level. $CRSP
ASCO abstract (TIP) https://meetinglibrary.asco.org/record/201308/abstract
Some preclinical data from NKTX last year https://www.nkartatx.com/file.cfm/75/docs/Nkarta_AACR_2020_gene_editing_891_20.pdf https://www.nkartatx.com/file.cfm/75/docs/Nkarta_AACR_2020_NK_plus_T_4235_9.pdf
And here I thought I got a great deal! Nice entry.
Started a position here at $0.94 USD pre-market this morning. GLTA.
Crisper looking like a steal at these levels imo
Lic. deal! So smart. Less $ out of pocket and I'm sure they get lots of money in profit sharing.
JMO
Blew through that 50 DMA this morning $CRSP
CRISPR Therapeutics Is Upgraded After $900 Million Deal -- Barrons.com
8:10 am ET April 21, 2021 (Dow Jones) Print
By Josh Nathan-Kazis
A day after CRISPR Therapeutics said that Vertex Pharmaceuticals would pay it $900 million for an extra 10% of the profits from sales of a gene-editing therapy called CTX001, a Jefferies analyst upgraded the stock to Buy, from Hold.
CRISPR (ticker: CRSP) and Vertex (VRTX) have jointly developed CTX001, which is being tested to treat patients with sickle cell disease and beta thalassemia, under a deal first signed in 2015. Under the new agreement, the 50/50 split under the old deal will now be a 60/40 split in favor of Vertex. In addition to the $900 million up front, Vertex will pay an additional $200 million when the therapy receives regulatory approval.
In a research note published early Wednesday, Jefferies analyst Maury Raycroft wrote that the deal validated the large market opportunity for the therapy, which he said is underappreciated by analysts. What's more, he said that the CTX001 program "has turned more into execution more for registration + launch [preparation]."
Raycroft upgraded CRISPR to Buy, from Hold, and set a price target of $172, up from $165. The stock closed Tuesday at $121.67.
Shares of CRISPR climbed 5.8% on Tuesday in response to the news, and were up another 1.8% in premarket trading on Wednesday. The stock is down 20.5% so far this year, and up 132.2% over the past 12 months.
In his note, Raycroft wrote that the recent pullback in shares had created a buying opportunity for investors. The stock is down 34.3% since Jan. 20, a period in which the S&P 500 has climbed 7.4%, and the SPDR S&P Biotech ETF (XBI) is down 14.9%.
Raycroft also said that the large amount of capital that CRISPR now has in the bank could help the company accelerate its other programs.
On CTX001, Raycroft wrote that the company will present more data on the therapy in 2021, and will fully enroll both its continuing studies in sickle cell disease and beta thalassemia this year. He also said that the company told him that they had made progress on ongoing discussions with regulators about what data they will need to file for approval for the therapy.
Of the 21 analysts who cover CRISPR tracked by FactSet, 13 rate it a Buy, while six rate it a Hold and two rate it a Sell. Their average target price of $153.44 implies a return of 26.1%.
Write to editors@barrons.com
(END) Dow Jones Newswires
These edits could be added to not only the anti-CD70 product, but also others (anti-CD19, anti-BCMA, anti-CD33 and anti-PTK7).
Other potential improvements being explored are TET2 disruption and ways to stop activation-induced cell death https://www.nature.com/articles/s41586-018-0178-z https://www.jci.org/articles/view/121491
Bounce off of the 200dma?
I think a sub-$100 entry looks much better than $116
From AACR:
CD70 and its ligand, CD27, have been described as both activating and suppressing for different cell types including B and T cells. There has been speculation that the CD70/CD27 axis can act in a checkpoint or co-stimulatory manner in certain immuno-oncology settings. Knockout (KO) of CD70 function scored highly in a CRISPR/Cas9 screen of candidate genes for enhanced T cell activity. Deletion of other checkpoint candidates such as PD1, TIM3, LAG3 and TIGIT scored much lower than CD70, both alone and in combination with each other. T cells with CD70 KO showed resistance to exhaustion upon repeated stimulation in culture. CAR-T cells with CD70 KO similarly showed exhaustion resistance, as well as a reduction in apoptosis, increased proliferation, and improved target cell lysis upon sequential rechallenges. CTX130 is an investigational allogeneic CAR-T therapy currently being studied in patients with CD70-expressing tumors, including clear cell renal cell carcinoma and B and T cell malignancies. CTX130 contains KOs of TRAC to avoid GvHD, B2M to protect the product from patient T cells, and CD70 for enhanced CAR-T performance. Comparing CTX130 with and without CD70 KOs shows that CAR-T cells with CD70 KO have increased potency, enhanced ability to withstand multiple tumor challenges in vivo, and increased resistance to overexpression of PDL1 on target cells. Interestingly, while CD70 surface expression may be extremely low or undetectable after manufacturing of CD70-targeted CAR-T cells without CD70 KO, the beneficial properties outlined here are only achievable when the CD70 gene is genetically knocked out. In summary, KO of CD70 confers benefit to CAR-T cells that far exceeds KO of other checkpoint related genes, and this benefit was present regardless of the antigen being targeted. CD70 KO is included in the CTX130 investigational allogeneic CAR-T therapy currently in clinical trials.
https://www.abstractsonline.com/pp8/#!/9325/presentation/2640
I think it is likely that 100, today’s low, was the bottom. It closed up more than 14 points from today’s low.
...and the bottom is ??? The last time the market had a big correction a year ago, wasn't this one of the first stocks to go back up ?
It looks close to hitting bottom IMO. Look for rapid rebound on the way back to $190-200.
This is the least talkative IHUB board out there. So, when is this going to go back up ? Its down almost 50% from the high a few months ago ! How long until it reaches $200 again ? Are they working on a way to alter our genes to make our cells more effective in fighting Covid and other serious viruses ? Are they in the market to buy any of the other smaller genetic engineering companies ?
A New Gene-Editing Technique May Be Able to Help Children Who Prematurely Age!
Base-editing is a variant of the gene-manipulation breakthrough called Crispr-Cas editing whose discovery won the chemistry Nobel Prize in October for the scientists Jennifer Doudna and Emmanuelle Charpentier. The ability of Crispr-Cas molecules to target and disrupt troublesome genes is being developed by companies like Crispr Therapeutics (CRSP), Editas Medicine (EDIT), and Intellia Therapeutics (NTLA).
https://www.barrons.com/articles/a-new-gene-editing-technique-may-be-able-to-help-children-who-prematurely-age-51609948802?siteid=yhoof2
I just added this as 25% of my portfolio. Great fundamentals and potential. This is easily a 10x bagger in 5 years.
Its also held in the ARKG fund @ 8.44% of the portfolio which is the second highest in the fund. That means CRSP was held in very high regards even before the recent news on sickle cell. If that success keeps improving this will be one Amazing stock. Plus an Incredible help to Humanity!
CRISPR Therapeutics and Vertex Present New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at American Society of Hematology Annual Meeting and Exposition, Together With Publication in the New England Journal of Medicine
https://finance.yahoo.com/news/crispr-therapeutics-vertex-present-data-173000391.html
Happen to agree with you. Held a minimal amount of shares at $30 until recently. Hearing of the Nobel prize was what caused me to look much deeper into this stock. Needless to say it has become a substantial investment for the long term. I think your future pps prediction could actually reasonable at some point. VERY exciting stuff! In my opinion of course.
I like this ticker looks like good news for the future might make it move on up Monday
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