Archive for the ‘Crispr’ Category

John Leonard, CEO of Intellia Therapeutics, has just finished an hour-long meeting in the company's Cambridge headquarters, which is a five-minute jog from the MIT campus. He stretches his arms, interlocks his fingers and rests them at the back of his head. For a moment, the 64-year-old is relaxed. But he immediately reanimates when explaining the science of DNA editing, excitedly using markers, a duster and whatever else he can find to make his point. I've used my wife's necklaces on occasion, he says. Leonard suggests imagining human DNA as a necklace made of 3 billion beads and four different colors. The challenge is how do you find 20 beads to the exclusion of everything else, he adds. The beads hes referring to are genes, sections of DNA which give cells the instructions they need to do their work.

John Leonard

Many companies use Crispr, a revolutionary method of precisely editing DNA which was the basis for the Nobel Prize in Chemistry in 2020, to snip disease-causing genes in the lab and then inject the fixed cells back into patients. Intellia does that too. But it is its other gene editing platform which has grabbed eyeballs at Wall Street. The $3.6 billion market cap company has figured out how to use Cripsr outside of the lab, inside a living human. Their work could have major implications in developing new drugs for genetic diseases that currently only have limited or no treatments. Intellia is the first to do in vivo genome editing in a systemic manner. I think that's the real differentiating factor for me, says Jack Allen, senior analyst at Baird Equity Research.

Despite the novelty of its gene editing technology, the company is facing significant headwinds. In the last 12-months it lost $277 million on revenues of $33 million. Revenues have been declining on a quarterly basis since 2020 while losses have been widening. The company has raised a total of $1.8 billion, including $115 million when it when public in 2016, and they still have $1 billion in cash on hand. But at present burn rates, that will only last a couple of years. Intellia does has one promising drug in early-stage clinical trials, but around 90% of treatments at this stage fail to reach the market. And then there is the patent battle over their core technology.

It sounds pretty grim, and Intellias stock has taken a beating. Since the start of the year, Intellia shares are 62% down compared to 23% for the Nasdaq and 24% for Nasdaqs biotech index. Still Intellia has at least one ace in the hole: The man who runs the company is no stranger to these challenges. Leonard, a medical doctor by training, has a track record which few others can point to in this industry. In 1992, Leonard joined Abbott where his team's research earned an FDA approval for HIV drug, Norvir and Kaletra, which helped curtail the AIDS epidemic in the nineties. And in 2013 he joined Abbvie, Abbotts biopharmaceutical spin off, where he was instrumental in the development of Humira, which had sales of $21 billion last year, making it one of the worlds best-selling drugs.

I worked on Humira for 13 years, Leonard says. I learned a lot of principles about an organization. What makes it work? What makes it not work sometimes.

His experiences of getting these blockbuster drugs from lab bench to the market could help turn Intellias liver drug NTLA-2001, which it is co-developing along with Regeneron Pharmaceuticals, into a success. The drug is an injectable gene editing therapy for the treatment of ATTR amyloidosis a rare genetic condition of the liver which impacts 1 in 100,000 Americans and kills around 850 of them a year.

The market for treating this disease was $585 million in 2019. But more people probably have the disease and are not being properly diagnosed. Better diagnoses could lead this to become a $14.1 billion market within 7 years, according to a report by London-based consultancy GlobalData. There are currently three FDA approved drugs which slow the progress of the disease, but none of them are permanent cures and patients often end up still needing liver transplants. In February, Intellia released early data from its clinical trials showing a sustained, positive effect on participants, without worrying side effects.

Despite promising data, Intellias road ahead isnt bump free. The company licenses the Crispr technology which it uses to perform in vivo gene edits from the University of California, the University of Vienna, and pathogen researcher Emmanuel Charpentier (collectively known as the CVC group). University of California biochemist Jennifer Doudna who won the Nobel along with Charpentier for discovering the Crispr editing system is an Intellia cofounder, although she has limited day-to-day responsibilities.

Those patents held by the CVC group are where the legal trouble comes in. Patents obtained by the CVC group are at odds with others owned by the Broad Institute, the powerhouse medical research center started by the late billionaire Eli Broad and affiliated with both Harvard and MIT. That spawned a legal battle, starting in 2016, with tens of millions of dollars in royalties at stake, about who was first to invent the Crispr gene editing tool used in human and plant cells. In over 80 countries including China, Japan and the 27 nations in the European Union, it has been held that Doudnas CVC group invented it first. But in the U.S., a recent verdict by the Patent Trial and Appeal Board (PTAB) ruled in favor of the Broad Institute. The CVC group is appealing.

Fortunately, this ruling does not impact the development of CRISPR in anyway, says Doudna. Investors are continuing to put money into the space, she adds.

Of course, even if Intellia loses in court, they will still be able to license the technology. Anyone who does not have a license from the Broad Institute and that is conducting work with [Crispr] is probably going to have to get one at some point. I would imagine that that would include Intellia, says Jacob Sherkow, a professor of law at the University of Illinois.

"Fortunately, this (PTAB) ruling does not impact the development of CRISPR in anyway. Investors are continuing to put money into the space."

Leonard is looking beyond the intellectual property battle. His focus currently is on expanding the companys development pipeline to include treatments for many more diseases, such as hereditary angioedema, hemophilia, blood and ovarian cancers. But first, Leonard will have to solve the patent mess and raise more money. Given the promise of Intellias technology, he is optimistic.

I think when people make judgments about where to put their cash to work, they look at the real possibility of [drug development] programs making it to the marketplace. We're definitely in that category, Leonard says. So we think we're well positioned to continue the funding of the company as we go forward.

He is even more optimistic about the future of Crispr-based drugs, which have the potential to relegate a whole host deadly diseases to the history books. In the coming years we will be limited not by technology but by imagination.

Here is the original post:
Gene Genies: Inside The Revolutionary Biotech That Can Edit DNA Inside Living Humans - Forbes

A good bargain stock isn't just an inexpensive one. There are plenty of stocks out there that are $10 or less or have a low price-to-earnings ratio (P/E). Both of those metrics can be deceiving, however. A lot of low-priced stocks deserve to be where they are, and many stocks with low P/Es are downtrodden because their futures aren't that bright.

The best bargains are the ones that will be good stocks to have and hold for the long term, yet are priced to sell right now. CRISPR Therapeutics (CRSP 8.29%), Microsoft (MSFT 1.09%), and Garmin (GRMN 0.74%) all have great long-term prospects and, for the moment, all are priced at a discount, down more than 20% this year.

Following the rest of the market, CRISPR Therapeutics is down nearly 20% this year as it trades at around $60. That leaves CRISPR with a P/E ratio of 15.7, under the biotech industry average of 20.3. But it's not its low P/E that's important but the potential.

CRISPR is a clinical-stage biotech company that uses the CRISPR/Cas9 gene-editing platform to create therapies to treat cancer, blood diseases, diabetes, and other diseases.

The company has a potential blockbuster, Exacel, formerly known as CTX-001, that it is developing with Vertex Pharmaceuticals. The drug is in late-stage trials for the treatment of patients with transfusion-dependent beta-thalassemia (TDT) or severe sickle cell disease (SCD), two genetic blood disorders that can cause painful episodes because of malformed blood cells that cause blockages in the bloodstream.

The drug works by editing a patient's own stem cells. It is a single-dose therapy that could significantly help the 300,000 people who are born worldwide each year with SCD and the 80,000 with TDT. CRISPR and Vertex said they anticipate regulatory filings for Exa-cel by the end of this year.

Exa-cel is only the tip of a large pipeline for CRISPR Therapeutics, which has several therapies in early-stage trials. Those trials include VCTX210 to treat type 1 diabetes, as well as three immuno-oncology therapies: CTX-110 to treat certain B-cell cancers, CTX-120 to treat multiple myeloma, and CTX-130 to treat solid tumors and blood cancers.

The company reported $178,000 in collaboration revenue in the first quarter and a loss of $179.2 million in the quarter, which deepened from the loss of $113.2 million in the same period last year. However, it is in a strong cash position, with $2.4 billion, allowing it to finance the development of its growing pipeline.

Microsoft is down more than 27% this year, trading at a new 52-week low. That drop has a lot more to do with the market's current distaste for tech stocks and very little to do with Microsoft's fundamentals, which, if you look at its 2022fiscal third-quarter earnings, remain strong.

The company reported $49.4 billion in revenue, up 18% year over year; net income of $16.7 billion, up 8% over the same period in 2021; and diluted earnings per share (EPS) of $2.22. Microsoft's cloud-based services drove much of the growth, with Office commercial products and cloud services revenue up 12%, Office consumer products, and cloud services up 11%, and dynamics products and cloud services up 22%. The company's intelligent cloud revenue was up 26%, led by 29% growth in server products and cloud services and 46% by Azure and other cloud services revenue gains.

The only cloud on the horizon is that growing inflation and a rising dollar might cut into the company's future sales. It revised its 2022 fourth-quarter guidance downward, as noted in the chart below.

Source: Chart by author. Microsoft quarterly filing.

Microsoft also said it now expects EPS to be between $2.24 and $2.32, down from between $2.28 and $2.35. A lowered estimate doesn't thrill investors, but if you look carefully, all of those numbers would represent a stronger quarter sequentially and year over year.

The company also has a dividend, unusual for a fast-growing tech company. Microsoft raised its dividend in fiscal 2021 by 11% to $0.62 per quarterly share, and last year was the 12th consecutive year the company has boosted its dividend. That dividend represents a current yield of 1% and is considered safe with a 24.4% payout ratio.

Garmin sells navigation, communication, and information devices that use the Global Position System (GPS) technology, including everything from fitness trackers to marine, automotive, and aviation GPS systems. Its stock is down more than 28% this year, sending its P/E to 17.5.

The company did well during the pandemic, as its fitness trackers became big sellers. While sales of fitness trackers are down, the company is seeing growth in other areas as it operates in five segments: fitness, outdoor, aviation, marine, and auto. The company has grown annual revenue for six consecutive years, and over the past five years, the company has increased annual revenue by 59.6%.

In the first quarter, Garmin reported revenue of $1.2 billion, up 9% year over year, though EPS was $1.09, down 4% over the same period in 2021. Despite fitness sales being down 28%, outdoor sales were up 50%, marine was up 21%, and auto rose 11% compared with the first quarter of 2021. The company said the reason earnings dropped was due to the impact of high freight costs and currency shifts, two factors that will likely moderate. Garmin has said it anticipates annual revenue of $5.5 billion, representing a growth of 8% over 2021 annual revenue.

The Swiss company raised its dividend toward the end of last year by 9% to $0.73 per quarterly share, the fifth consecutive year it has increased its dividend. That equals a yield of roughly 3%.

See original here:
3 Bargain Stocks You Can Buy Today and Hold Forever - The Motley Fool

Item 5.02. Departure of Directors or Certain Officers; Election of Directors;

Approval of Amendment to CRISPR Therapeutics AG 2018 Stock Option and IncentivePlan

On June 9, 2022, at the 2022 Annual General Meeting of Shareholders (the "AnnualMeeting"), the shareholders of CRISPR Therapeutics AG (the "Company") approvedthe amendment (the "Plan Amendment") to the CRISPR Therapeutics AG 2018 StockOption and Incentive Plan (the "2018 Plan") to increase the number of commonshares, par value CHF 0.03 per share ("Common Shares") reserved for issuanceunder the 2018 Plan by 1,700,000 Common Shares and to increase the number ofshares that may be issued in the form of incentive stock options by 1,700,000Common Shares. The Plan Amendment previously had been approved, subject toshareholder approval, by the Company's Board of Directors (the "Board").

The Company's officers and directors are among the persons eligible to receiveawards under the 2018 Plan, as amended, in accordance with the terms andconditions thereunder. A detailed summary of the 2018 Plan and the PlanAmendment is set forth in the Company's proxy statement for the Annual Meetingfiled with the Securities and Exchange Commission on April 25, 2022 (the "ProxyStatement") under the caption "Proposal 13: Approval of Amendment to the CRISPRTherapeutics AG 2018 Stock Option and Incentive Plan", which summary isincorporated herein by reference. That detailed summary of the 2018 Plan andPlan Amendment, and the foregoing description of the Plan Amendment, arequalified in their entirety by reference to (i) the full text of the 2018 Plan,which is filed as Exhibit 99.1 to the Company's Registration Statement on FormS-8 filed on June 1, 2018, (ii) the full text of Amendment No. 1 to the 2018Stock Option and Incentive Plan, which is attached as Appendix A to theCompany's Definitive Proxy Statement on Schedule 14A filed on April 30, 2019,(iii) the full text of Amendment No. 2 to the 2018 Stock Option and IncentivePlan, which is attached as Appendix A to the Company's Definitive ProxyStatement on Schedule 14A filed on April 24, 2020, and (iv) the full text of thePlan Amendment, a copy of which is attached as Appendix A to the ProxyStatement, and in each case, incorporated herein by reference.

Item 5.03. Amendments to Articles of Incorporation or Bylaws; Change in Fiscal Year

At the Annual Meeting, the Company's shareholders approved amendments to itsArticles of Association as described in the Proxy Statement. The Company'samended and restated Articles of Association become effective upon registrationin the Commercial Register in the canton of Zug, Switzerland on or about June15, 2022, subject to the approval by the Swiss Federal Commercial Authority. Acopy of the amended and restated Articles of Association is attached hereto asExhibit 3.1 and incorporated herein by reference.

Item 5.07. Submission of Matters to a Vote of Security Holders

The Annual Meeting was held on June 9, 2022. Proxies were solicited pursuant tothe Proxy Statement.

At the Annual Meeting, the Company's shareholders were asked (i) to approve theSwiss statutory annual report, the consolidated financial statements and thestatutory financial statements of the Company for the year ended December 31,2021, (ii) to approve the appropriation of financial results, (iii) to dischargethe members of the Board of Directors and Executive Committee, (iv) to elect andre-elect nine members and the chairman to the Company's Board of Directors, (v)to re-elect three members of the Compensation Committee of the Board ofDirectors, (vi) to approve the compensation for the Board of Directors and theExecutive Committee, (vii) to approve the compensation paid to the Company'snamed executive officers under U.S. securities law requirements, (viii) toapprove the frequency of future shareholder advisory votes on the compensationpaid to the Company's named executive officers under U.S. securities lawrequirements, (ix) to approve an increase in the maximum size of the Board ofDirectors, (x) to approve an adjustment of the maximum number of authorizedshare capital and extend the date by which the Board of Directors may increasethe authorized share capital of the Company, (xi) to approve an adjustment ofthe conditional share capital for the conversion of bonds and similar debtinstruments, (xii) to approve an increase in the conditional share capital foremployee equity plans, (xiii) to approve the Plan Amendment, (xiv) to re-electthe independent voting rights representative, (xv) to re-elect Ernst & Young AGas the Company's statutory auditor and to re-elect Ernst & Young LLP as theCompany's independent registered public accounting

--------------------------------------------------------------------------------

firm for the year ending December 31, 2022, and (xvi) to approve the transactionof any other business that may properly come before the Annual Meeting.

The voting results reported below are final.

Proposal 1 - Approval of the Swiss Statutory Annual Report, the ConsolidatedFinancial Statements and the Statutory Financial Statements of the Company forthe Year Ended December 31, 2021

The Swiss statutory annual report, the consolidated financial statements and thestatutory financial statements of the Company for the year ended December 31,2021 were approved. The results of the vote were as follows:

Proposal 2 - Approval of the Appropriation of Financial Results

The proposal to carry forward the net income resulting from the appropriation offinancial results was approved. The results of the vote were as follows:

Proposal 3 - Discharge of the Members of the Board of Directors and ExecutiveCommittee

The discharge of the members of the Company's Board of Directors and theExecutive Committee from personal liability for their activities during the yearended December 31, 2021 was approved. The results of the vote were as follows:

--------------------------------------------------------------------------------

Proposal 4 - Election and Re-election of the Members and Chair of the Board ofDirectors

Rodger Novak, M.D., Samarth Kulkarni, Ph.D., Ali Behbahani, M.D., BradleyBolzon, Ph.D., H. Edward Fleming Jr., M.D., Simeon J. George, M.D., John T.Greene, Katherine A. High, M.D., and Douglas A. Treco, Ph.D. were each dulyre-elected as members of the Company's Board of Directors, Rodger Novak, M.D.was duly re-elected as the chairman of the Board of Directors, and Maria Fardis,Ph.D., was duly elected as a member of the Company's Board of Directors. Theresults of the election were as follows:

Samarth Kulkarni, Ph.D. 38,203,106 834,226 79,486 16,951,536Ali Behbahani, M.D. 27,384,666 11,649,601 82,551 16,951,536Bradley Bolzon, Ph.D. 38,760,145 275,056 81,617 16,951,536H Edward Fleming Jr., M.D. 38,817,215 217,290 82,313 16,951,536Simeon J. George, M.D. 38,646,772 386,485 83,561 16,951,536John T. Greene

Proposal 5 -Re-election of the Members of the Compensation Committee

Ali Behbahani, M.D., Simeon J. George, M.D., and John T. Greene, were each dulyre-elected as members of the Company's Compensation Committee of the Board ofDirectors. The results of the election were as follows:

Ali Behbahani, M.D. 28,225,426 10,801,138 90,254 16,951,536Simeon J. George, M.D. 37,702,802 1,324,072 89,944 16,951,536John T. Greene

Proposal 6 - Approval of the Compensation for the Board of Directors and theExecutive Committee

The total non-performance-related compensation for members of the Board ofDirectors from the Annual Meeting to the 2023 annual general meeting ofshareholders was approved on a binding basis. The results of the binding votewere as follows:

The grant of equity for members of the Board of Directors from the AnnualMeeting to the 2023 annual general meeting of shareholders was approved on abinding basis. The results of the binding vote were as follows:

--------------------------------------------------------------------------------

The total non-performance related compensation for members of the ExecutiveCommittee from July 1, 2022 to June 30, 2023 was approved on a binding basis.The results of the binding vote were as follows:

The total variable compensation for members of the Executive Committee for thecurrent year ending December 31, 2022 was approved on a binding basis. Theresults of the binding vote were as follows:

The grant of equity for members of the Executive Committee from the AnnualMeeting to the 2023 annual general meeting of shareholders was approved on abinding basis. The results of the binding vote were as follows:

Proposal 7 - Non-Binding Advisory Vote on the Compensation Paid to NamedExecutive Officers

The compensation for the named executive officers was approved on a non-bindingbasis. The results of the non-binding vote were as follows:

Proposal 8 - Non-Binding Vote on the Frequency of Advisory Votes on ExecutiveCompensation

The Company's shareholders approved, on a non-binding, advisory basis afrequency of One Year for the non-binding, advisory vote on the compensation ofthe Company's named executive officers. The Board considered these votingresults and other factors, and has determined that the Company will hold futureadvisory votes on its executive compensation on an annual basis. The results ofthe non-binding vote were as follows:

Proposal 9 - Approval of Increasing the Maximum Size of the Board of Directors

An increase in the maximum size of the Board of Directors was approved with atleast two thirds of the votes cast. The results of the vote were as follows:

--------------------------------------------------------------------------------

Proposal 10 - The Approval of an Adjustment of the Maximum Number of AuthorizedShare Capital and Extending the Date by Which the Board of Directors MayIncrease the Share Capital

The adjustment of the maximum number of authorized share capital and extensionof the date by which the Board of Directors may increase the share capital wasnot approved. The results of the election were as follows:

Proposal 11 - Approval of an Adjustment of the Conditional Share Capital for theConversion of Bonds and Similar Debt Instruments

An adjustment of the Company's conditional share capital for the conversion ofbonds and similar debt instruments was approved with at least two thirds of thevotes cast. The results of the vote were as follows:

Proposal 12 - Approval of an Adjustment of the Conditional Share Capital forEmployee Equity Plans

An adjustment of the Company's conditional share capital for employee equityplans was approved with at least two thirds of the votes cast. The results ofthe vote were as follows:

Proposal 13 - Approval of Amendment to the 2018 Stock Option and Incentive Plan

The Plan Amendment was approved. The results of the vote were as follows:

Proposal 14 - Re-election of the Independent Voting Rights Representative

Marius Meier, Attorney at Law, was duly re-elected as the independent votingrights representative. The results of the election were as follows:

--------------------------------------------------------------------------------

Proposal 15 - Re-election of the Auditors

Ernst & Young AG was duly elected as the Company's statutory auditor for theterm of office of one year, and Ernst & Young LLP was duly elected as theCompany's independent registered public accounting firm for the year endingDecember 31, 2022. The results of the election were as follows:

Proposal 16 - Transaction of Any Other Business

The transaction of any other business that properly came before the AnnualMeeting was approved. The results of the election were as follows:

--------------------------------------------------------------------------------

Item 9.01 Financial Statements and Exhibits

# A management contract or compensatory plan or arrangement required to be filedas an exhibit pursuant to Item 15(a)(3) of Form 10-K

--------------------------------------------------------------------------------

Edgar Online, source Glimpses

See more here:
CRISPR THERAPEUTICS AG : Change in Directors or Principal Officers, Amendments to Articles of Inc. or Bylaws; Change in Fiscal Year, Submission of...

TUEBINGEN, Germany and HOUSTON and CAMBRIDGE, Mass., June 07, 2022 (GLOBE NEWSWIRE) -- Immatics N.V. ( IMTX, Immatics), a clinical-stage biopharmaceutical company active in the discovery and development of T cell-redirecting cancer immunotherapies, and Editas Medicine, Inc. ( EDIT, Editas Medicine), a leading genome editing company, today announced that the two companies have entered into a strategic research collaboration and licensing agreement to combine gamma-delta T cell adoptive cell therapies and gene editing to develop medicines for the treatment of cancer. As part of the licensing agreement, Immatics gains non-exclusive rights to Editas Medicines CRISPR technology and intellectual property. Editas Medicine is the exclusive licensee of Harvard and Broad Institutes Cas9 patent estates and Broad Institutes Cas12a patent estate for human medicines.

By combining Editas Medicines gene editing technology with Immatics ACTallo allogeneic, off-the-shelf adoptive cell therapy platform based on gamma-delta T cells, gamma-delta T cells can be redirected to cancer cell targets with the goal of creating cells with enhanced tumor recognition and destruction.

Engineered cell therapies have the potential to significantly impact the treatment paradigm for cancer, and our partnership with the esteemed team at Editas Medicine will provide us with further versatility and flexibility in how we engineer our ACTallo cell therapies based on a specific tumor target, said Rainer Kramer, Ph.D., Chief Business Officer, Immatics. It has always been our focus to deliver innovative science to cancer patients and this collaboration with Editas Medicine will enable us to access CRISPR technologies and apply them to our off-the-shelf gamma-delta T cell platform.

We believe that our gene editing technology can modulate and enhance the potential of cell therapies to deliver transformative medicines for the treatment of cancer. We are excited to work with the team at Immatics to develop new experimental medicines with enhanced tumor fighting abilities to help patients with cancer, said Gilmore ONeill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine.

Under the terms of the agreement, Editas Medicine will be eligible to receive an undisclosed upfront cash payment as well as additional milestone payments based on development, regulatory, and commercial milestones. In addition, Immatics will pay royalties on future net sales on any products that may result from this collaboration.

About ImmaticsImmatics combines the discovery of true targets for cancer immunotherapies with the development of the right T cell receptors with the goal of enabling a robust and specific T cell response against these targets. This deep know-how is the foundation for our pipeline of Adoptive Cell Therapies and TCR Bispecifics as well as our partnerships with global leaders in the pharmaceutical industry. We are committed to delivering the power of T cells and to unlocking new avenues for patients in their fight against cancer.

For regular updates about Immatics, visit http://www.immatics.com. You can also follow us on Instagram, Twitter and LinkedIn.

About Editas MedicineAs a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Harvard and Broad Institutes Cas9 patent estates and Broad Institutes Cas12a patent estate for human medicines. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

Immatics Forward-Looking StatementsCertain statements in this press release may be considered forward-looking statements. Forward-looking statements generally relate to future events or Immatics future financial or operating performance. For example, statements concerning the timing of product candidates and Immatics focus on partnerships to advance its strategy are forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as may, should, expect, intend, will, estimate, anticipate, believe, predict, potential or continue, or the negatives of these terms or variations of them or similar terminology. Such forward-looking statements are subject to risks, uncertainties, and other factors which could cause actual results to differ materially from those expressed or implied by such forward looking statements. These forward-looking statements are based upon estimates and assumptions that, while considered reasonable by Immatics and its management, are inherently uncertain. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Factors that may cause actual results to differ materially from current expectations include, but are not limited to, various factors beyond management's control including general economic conditions and other risks, uncertainties and factors set forth in filings with the SEC. Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. You should not place undue reliance on forward-looking statements, which speak only as of the date they are made. Immatics undertakes no duty to update these forward-looking statements.

Editas Medicine Forward-Looking Statements This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, target, should, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the expected benefits of Editas Medicines collaboration with Immatics, including any future payments it may receive under the strategic research collaboration and licensing agreement and the potential to generate medicines from the collaboration. Editas Medicine may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of pre-clinical studies and clinical trials and clinical development of Editas Medicines product candidates; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for Editas Medicines foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption Risk Factors included in Editas Medicines most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, as updated by Editas Medicines subsequent filings with the Securities and Exchange Commission, and in other filings that Editas Medicine may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release represent Editas Medicines views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, Editas Medicine explicitly disclaims any obligation to update any forward-looking statements.

Read more:
Immatics and Editas Medicine Enter Strategic Research Collaboration and Licensing Agreement to Combine Gamma-Delta T Cell Adoptive Cell Therapies and...

Dive Brief:

Though small in scope, Immatics and Editas deal adds to a flurry of recent activity involving treatments that harness gamma delta T cells, rare white blood cells with unique tumor-fighting capabilities.

Unlike the T cells used in Novartis, Gilead and Bristol Myers Squibbs treatments, gamma delta cells have elements of both innate and adaptive immunity, which could enable them to generate a broader response against cancers. These cells also have key differences that make them less likely to trigger graft-versus-host disease, give them the potential to persist in the body for years, and to recognize a range of targets.

Those traits have already prompted drugmakers including Takeda, Johnson & Johnson, Bristol Myers and Regeneron to make investments. Clinical data presented at the American Society of Clinical Oncology has further elevated the profile of gamma delta cell therapy, as a treatment from Adicet Bio has shown early promise against non-Hodgkins lymphoma.

Immatics has already capitalized on the momentum, turning its alliance with Bristol Myers into a new, lucrative deal. Now Immatics is bringing gene editing tools in as well.

Genetic engineering is already part of many cell therapies, as CAR-T treatments involve modifications that help T cells recognize cancer. But CRISPR and other gene editing approaches could help do more. Allogene Therapeutics, for example, uses gene editing to make changes aimed at reducing the risk of graft-versus-host disease. Nkarta and CRISPR Therapeutics plan to give treatments involving natural killer cells, which share some similarities with gamma delta T cells, more tumor-killing punch.

Immatics and Editas appear to share a similar goal, saying in a statement that they want to make gamma delta cells with enhanced tumor recognition and destruction. Those potential benefits do come with added risk, however. U.S. regulators halted testing of Allogenes programs last year to investigate whether the gene editing involved in its treatment led to a chromosomal abnormality in a treated patient. Follow-up investigation exonerated Allogenes treatment, but the setback led to a lengthy delay.

For Editas, the deal adds to multiple other partnerships involving cell therapy. The company is already working with Bayer's Bluerock Therapeutics subsidiary on natural killer cell therapies for solid tumors, and with Bristol Myers on so-called alpha-beta T cell treatments.

See the original post here:
Immatics and Editas join up to bring CRISPR to 'gamma delta' cell therapy - BioPharma Dive

CRISPR Therapeutics (NASDAQ:CRSP Get Rating) and Inhibrx (NASDAQ:INBX Get Rating) are both medical companies, but which is the superior business? We will compare the two companies based on the strength of their valuation, earnings, dividends, institutional ownership, profitability, analyst recommendations and risk.

Earnings and Valuation

This table compares CRISPR Therapeutics and Inhibrxs revenue, earnings per share and valuation.

Profitability

This table compares CRISPR Therapeutics and Inhibrxs net margins, return on equity and return on assets.

Analyst Recommendations

This is a summary of recent ratings and recommmendations for CRISPR Therapeutics and Inhibrx, as reported by MarketBeat.

CRISPR Therapeutics presently has a consensus price target of $114.71, indicating a potential upside of 70.25%. Inhibrx has a consensus price target of $44.33, indicating a potential upside of 291.64%. Given Inhibrxs stronger consensus rating and higher possible upside, analysts plainly believe Inhibrx is more favorable than CRISPR Therapeutics.

Insider & Institutional Ownership

56.1% of CRISPR Therapeutics shares are owned by institutional investors. Comparatively, 59.6% of Inhibrx shares are owned by institutional investors. 10.7% of CRISPR Therapeutics shares are owned by company insiders. Comparatively, 25.8% of Inhibrx shares are owned by company insiders. Strong institutional ownership is an indication that large money managers, endowments and hedge funds believe a stock will outperform the market over the long term.

Volatility & Risk

CRISPR Therapeutics has a beta of 2.04, suggesting that its stock price is 104% more volatile than the S&P 500. Comparatively, Inhibrx has a beta of 3.1, suggesting that its stock price is 210% more volatile than the S&P 500.

Summary

CRISPR Therapeutics beats Inhibrx on 8 of the 14 factors compared between the two stocks.

About CRISPR Therapeutics (Get Rating)

CRISPR Therapeutics is a gene-editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene-editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

About Inhibrx (Get Rating)

Inhibrx, Inc., a clinical-stage biotechnology company, focuses on developing a pipeline of novel biologic therapeutic candidates. The company's therapeutic candidates include INBRX-109, a tetravalent agonist of death receptor 5, which is in Phase 2 clinical trials to treat cancers, such as chondrosarcoma, mesothelioma, and pancreatic adenocarcinoma; INBRX-105, a tetravalent conditional agonist of programmed death-ligand 1 and a conditional agonist of 4-1BB that is in Phase 1 clinical trials to treat patients with locally advanced or metastatic solid tumors; and INBRX-101, an alpha-1 antitrypsin (AAT)-Fc fusion protein therapeutic candidate, which is in Phase 1 clinical trials for use in the treatment of patients with AAT deficiency. It also provides INBRX-106, a hexavalent agonist of OX40 for a range of oncology indications. The company was founded in 2010 and is headquartered in La Jolla, California.

Receive News & Ratings for CRISPR Therapeutics Daily - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings for CRISPR Therapeutics and related companies with MarketBeat.com's FREE daily email newsletter.

More here:
Analyzing CRISPR Therapeutics (NASDAQ:CRSP) and Inhibrx (NASDAQ:INBX) - Defense World

- Vertex announces three additional abstracts on the burden of beta thalassemia and sickle cell disease accepted for poster presentation

BOSTON & ZUG, Switzerland & CAMBRIDGE, Mass., June 02, 2022--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (NASDAQ: CRSP) today announced new late-breaking clinical data accepted for oral presentation at the 2022 European Hematology Association (EHA) Congress. Vertex also announced three abstracts accepted for poster presentation at EHA.

Late-breaking abstract #LB2367 entitled "Efficacy and Safety of A Single Dose of CTX001 For Transfusion-Dependent eta-Thalassemia and Severe Sickle Cell Disease," will be an oral presentation on Sunday, June 12 at 09:4511:15 CEST. The abstract from Vertex and CRISPR Therapeutics includes data on patients treated in CLIMB111 and CLIMB121 and followed in CLIMB131 with CTX001, now known as exagamglogene autotemcel (exa-cel). This abstract has been selected for the media briefing program and is therefore embargoed until Saturday, June 11 at 09:00 am CEST.

In addition, three real-world evidence and health economics abstracts from Vertex have been accepted for poster presentation.

Abstract #P1704 entitled "Projected Lifetime Economic Burden of Severe Sickle Cell Disease in the United States," will be a poster presentation on Friday, June 10 at 16:3017:45 CEST. The abstract posted online projects the per-patient lifetime direct health care cost of severe sickle cell disease (SCD) from a U.S. health care payer perspective using an economic model developed based on published model frameworks.

Abstract #P1703 entitled "Economic Burden of TransfusionDependent BetaThalassemia in the United States," will be a poster presentation on Friday, June 10 at 16:3017:45 CEST. The abstract posted online estimates the economic burden of transfusion-dependent beta thalassemia (TDT) using administrative claims data to estimate the costs and health care utilization associated with disease management in the U.S.

Abstract #P1482 entitled "Patients With Severe Sickle Cell Disease on Standard-of-Care Treatment Are Very Unlikely to Become VOCFree for One Year: A Cohort Study of Medicaid Enrollees," will be a poster presentation on Friday, June 10 at 16:3017:45 CEST. The abstract posted online contextualizes the efficacy of exacel in eliminating vasoocclusive crises (VOCs) in patients with SCD using nationwide U.S. Medicaid claims data from 2000 to 2014 to assess the proportion of patients with recurrent VOCs who became VOCfree during a 1year follow up on standard of care.

Story continues

The accepted abstracts are now available online on the EHA website.

Exacel is being investigated in multiple ongoing clinical trials as a potential one-time therapy for patients with either TDT or SCD.

About exagamglogene autotemcel (exa-cel)

Exacel, formerly known as CTX001, is an investigational, autologous, ex vivo CRISPR/Cas9 geneedited therapy that is being evaluated for patients with TDT or SCD characterized by recurrent VOCs, in which a patients own hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygencarrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. The elevation of HbF by exacel has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published in The New England Journal of Medicine in January of 2021.

Based on progress in this program to date, exacel has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. Exa-cel has also been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), for both TDT and SCD.

Among geneediting approaches being evaluated for TDT and SCD, exacel is the furthest advanced in clinical development.

About CLIMB111 and CLIMB121

The ongoing Phase 1/2/3 openlabel trials, CLIMB111 and CLIMB121, are designed to assess the safety and efficacy of a single dose of exacel in patients ages 12 to 35 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now closed for enrollment. Patients will be followed for approximately two years after exacel infusion. Each patient will be asked to participate in CLIMB131, a longterm followup trial.

About CLIMB-131

This is a longterm, openlabel trial to evaluate the safety and efficacy of exacel in patients who received exacel in CLIMB111, CLIMB121, CLIMB141 or CLIMB151. The trial is designed to follow participants for up to 15 years after exacel infusion.

About CLIMB141 and CLIMB151

The ongoing Phase 3 open-label trials, CLIMB141 and CLIMB151, are designed to assess the safety and efficacy of a single dose of exacel in patients ages 2 to 11 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now open for enrollment and currently enrolling patients ages 5 to 11 years of age and will plan to extend to ages 2 to less than 5 years of age at a later date. Each trial will enroll up to 12 patients. Patients will be followed for approximately two years after infusion. Each patient will be asked to participate in CLIMB-131, a longterm followup trial.

About the GeneEditing Process in These Trials

Patients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patients cells will be edited using the CRISPR/Cas9 technology. The edited cells, exacel, will then be infused back into the patient as part of an autologous hematopoietic stem cell transplant (HSCT), a process which involves a patient being treated with myeloablative busulfan conditioning. Patients undergoing HSCT may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of exacel. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of exacel on multiple measures of disease and for safety.

About the VertexCRISPR Collaboration

Vertex and CRISPR Therapeutics entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. Exacel represents the first potential treatment to emerge from the joint research program. Under an amended collaboration agreement, Vertex now leads global development, manufacturing and commercialization of exacel and splits program costs and profits worldwide 60/40 with CRISPR Therapeutics.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1mediated kidney disease, pain, type 1 diabetes, alpha1 antitrypsin deficiency and Duchenne muscular dystrophy.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 12 consecutive years on Science magazine's Top Employers list and one of the 2021 Seramount (formerly Working Mother Media) 100 Best Companies. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

(VRTX-GEN)

Vertex Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, our plans and expectations to present clinical data from the ongoing exa-cel clinical trials during the EHA Congress, expectations regarding the abstracts that will be made available on the virtual platform and the clinical data that will be presented during the EHA Congress, including anticipated projections and estimates related to the various economic impacts of SCD and TDT, the potential benefits, efficacy, and safety of exa-cel, including the potentially transformative nature of the therapy and the potential of the treatment for patients, our plans and expectations for our clinical trials and pipeline products, the status of our clinical trials of our product candidates under development by us and our collaborators, including activities at the clinical trial sites, patient enrollment and expectations regarding clinical trial follow-up. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from a limited number of patients may not be indicative of final clinical trial results, that data from the company's development programs, including its programs with its collaborators, may not support registration or further development of its compounds due to safety and/or efficacy, or other reasons, that internal or external factors that could delay, divert, or change our plans and objectives with respect to our research and development programs, that future competitive or other market factors may adversely affect the commercial potential for exa-cel, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission (SEC) and available through the company's website at http://www.vrtx.com and on the SECs website at http://www.sec.gov. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(CRSP-GEN)

About CRISPR Therapeutics

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking Statement

This press release may contain a number of "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: i) the safety, efficacy and clinical progress of the ongoing exa-cel clinical trials, including expectations regarding the abstract that will be made available on the virtual platform and our plans to present and the clinical data that are being presented during the EHA Congress; and (ii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words "believes," "anticipates," "plans," "expects" and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, existing and prospective investors are cautioned that forward-looking statements are inherently uncertain, are neither promises nor guarantees and not to place undue reliance on such statements, which speak only as of the date they are made. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with exa-cel at this time) not to be indicative of final or future trial results; the potential that the exa-cel clinical trial results may not be favorable or may not support registration or further development; that future competitive or other market factors may adversely affect the commercial potential for exa-cel; CRISPR Therapeutics may not realize the potential benefits of its collaboration with Vertex; potential impacts due to the coronavirus pandemic, such as to the timing and progress of clinical trials; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220601006221/en/

Contacts

Vertex Pharmaceuticals Incorporated Investors: Michael Partridge, +1 617-341-6108OrManisha Pai, +1 617-961-1899OrMiroslava Minkova, +1 617-341-6135

Media: mediainfo@vrtx.com orU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275

CRISPR Therapeutics Investors: Susan Kim, +1 617-307-7503susan.kim@crisprtx.com

Media: Rachel Eides, +1-617-315-4493.rachel.eides@crisprtx.com

See the article here:
Vertex and CRISPR Therapeutics Announce Acceptance of Late-Breaking Abstract for CTX001 at the 2022 Annual European Hematology Association (EHA)...

The latest competent intelligence report published by WMR with the title An increase in demand and Opportunities for Global CRISPR-Cas9 Market 2022 provides a sorted image of the CRISPR-Cas9 industry by analysis of research and information collected from various sources that have the ability to help the decision-makers in the worldwide market to play a significant role in making a gradual impact on the global economy. The report presents and showcases a dynamic vision of the global scenario in terms of market size, market statistics, and competitive situation.

At present, the CRISPR-Cas9 market is possessing its presence over the globe. The Research report presents a complete judgment of the market which consists of future trends, growth factors, consumption, production volume, CAGR value, attentive opinions, profit margin, price, and industry-validated market data. This report helps the individuals and the market competitors to predict future profitability and to make critical decisions for business growth.

A sample report can be viewed by visiting (UseCorporate eMail ID to Get Higher Priority) at:https://www.worldwidemarketreports.com/sample/493454

Scope of CRISPR-Cas9 Market:

Emerging trends, The report on the CRISPR-Cas9 market givesa complete picture of demands and opportunities for the future that are beneficial for individuals and stakeholders in the market. This report determines the market value and the growth rate based on the key market dynamics as well as the growth improving factors. The entire study is based on the latest industry news, market trends, and growth probability. It also consists of a deep analysis of the market and competing scenario along with the SWOT analysis of the well-known competitors.

The Leading Players involved in the global CRISPR-Cas9 market are:

Editas Medicine CRISPR Therapeutics Horizon Discovery Sigma-Aldrich Genscript Sangamo Biosciences Lonza Group Integrated DNA Technologies New England Biolabs Origene Technologies Transposagen Biopharmaceuticals Thermo Fisher Scientific Caribou Biosciences Precision Biosciences Cellectis Intellia Therapeutics

Segmentation by Type:

Biotechnology Companies Pharmaceutical Companies Others

Segmentation by Applications:

Genetic Engineering Gene Library Human Stem Cells Others

Get PDF Brochure Here by Clicking:https://www.worldwidemarketreports.com/sample/493454

Global CRISPR-Cas9 Market Regional Analysis:

The research study has segregated the global CRISPR-Cas9 industry into segments, including product type, application, and vertical, to broaden the overall understanding of the industry. This assessment has been carried out on the basis of size, share, and CAGR. Additionally, regional analysis has been done by the experts stressing the growth potential of the key regions and countries. The report also encompasses accurate and reliable figures based on the CRISPR-Cas9 consumption and production in key regions.

North America: USA, Canada, Mexico, etc.Asia-Pacific: China, Japan, Korea, India, and Southeast AsiaThe Middle East and Africa: Saudi Arabia, the UAE, Egypt, Turkey, Nigeria, and South AfricaEurope: Germany, France, the UK, Russia, and ItalySouth America: Brazil, Argentina, Columbia, etc.

The report helps in providing a wider introduction to the market and also helps in dealing with the detailed methodology of research for the calculation of the size and forecasts of the market. The sources of secondary data are used and the primary inputs are taken for the validation of data. This section also helps in the outlines of the several segments that have also been covered as being a part of the report. Additionally, the Research Methodology reviews tend of providing the calculation for determining the inclinations of the global market.

Get customization &Inquire AboutDiscount:https://www.worldwidemarketreports.com/quiry/493454

Some of the Major Points of TOC cover:

Chapter 1: Techniques & Scope

1.1 Definition and forecast parameters1.2 Methodology and forecast parameters1.3 Information Sources

Chapter 2: Latest Trends Summary

2.1 Regional trends2.2 Product trends2.3 End-use trends2.4 Business trends

Chapter 3: CRISPR-Cas9 Industry Insights

3.1 Industry fragmentation3.2 Industry landscape3.3 Vendor matrix3.4 Technological and innovative landscape

Chapter 4: CRISPR-Cas9 Market, By Region

Chapter 5: Company Profiles

5.1 Company Overview5.2 Financial elements5.3 Product Landscape5.4 SWOT Analysis5.5 Systematic Outlook

Chapter 6: Assumptions and Acronyms

Chapter 7: Research Methodology

Chapter 8: Contact (Continue . . .)

Purchase of CRISPR-Cas9 Market Report at:https://www.worldwidemarketreports.com/buy/493454

Finally, the report CRISPR-Cas9 Market 2022 provides an industry development game plan, the industry information source, research findings, an appendix, and a conclusion. The report offers precise clarification of the market by highlighting the market manufacturing procedure, market competitors, sellers and merchants classification, the implementation of innovation, and business improvement designs. All these details will reassure clients of future plans and actions intended to compete with other players in the market. Further, the most recent improvements in the market are displayed.

Contact Us:

Worldwide Market Reports,Tel: U.S. +1-415-871-0703U.K. +44-203-289-4040Japan +81-50-5539-1737Email: [emailprotected]Website: https://www.worldwidemarketreports.com/

Follow this link:
CRISPR-Cas9 Market: Established Keyplayers Will Try to Generate New Growth Opportunities in the Upcoming Years: Editas Medicine, CRISPR Therapeutics,...

With a targeted disruptive innovation biotechnology-related exchange traded fund strategy, investors can focus on companies driving change in targeted therapeutics, bioinformatics, CRISPR technology, and more.

In the recent webcast, Change the DNA of Your Portfolio: Growth Opportunities Through Genomics, Nicholas Grous, associate portfolio manager at ARK Invest, highlighted five innovation platforms that will experience long-term growth, including artificial intelligence, energy storage, robotics, DNA sequencing, and blockchain technology. These five sub-categories are expected to enjoy long-term growth. For instance, gene sequencing is expected to expand to $3.6 trillion in 2030 from $125 billion in 2020. Overall, ARK Invest projects disruptive innovation technologies could grow to $210 trillion by 2030, compared to $14 trillion in 2020.

The increased adoption of disruptive innovation technologies will not just be a one-off event as these innovative ideas will cover a broad swathe of industries and touch upon many facets of the global economy. For example, Grous believed that the convergence of next-generation DNA sequencing, artificial intelligence, and gene therapies should boost returns on investment significantly, potentially creating a golden age of health care likely to rival that of the 1980s and 1990s.

Simon Barnett, research analyst at ARK Invest, explained that researchers had historically been forced to choose between accuracy with short-read sequencing or comprehensiveness with long-range sequencing to break the genome into smaller segments to analyze with high-resolution optics, which is then reassembled with computer algorithms. According to Wrights Law, for every cumulative doubling in data produced on its reinstalled base, the cost of synthesis-based LRS has declined and will continue to decline by 28%. Looking ahead, ARK projects the cost to sequence a whole human genome with long-read technology will drop to $100-$200 and its accuracy will be superior to SRS across all variant types by the end of 2025.

According to our research, gene editing breakthroughs are creating more effective therapies at a faster rate than historically has been the case, Barnett said.

For example, compared to zinc finger nucleases (ZFNs), which moved from discovery to the first human dose in roughly eight years, the relatively new CRISPR technology took less than half the time, three years, and can address 48% of known diseases, or almost twice ZFNs 28%. Prime and base editing CRISPR derivatives address even more diseases, 79% and 59%, respectively. Consequently, Barnett argued that CRISPR may be seen as a superior gene-editing method going forward, and it already shows as CRISPR has been dominating recent academic research and clinical trials.

Looking ahead, ARK expects gene editing and gene therapy companies to grow to $1.1 trillion in market capitalization by 2026. Given potential cures for diseases, the share of research and development funding for gene therapy innovations should continue to rise. By 2026, the share of total R&D spending devoted to gene editing and therapy companies could grow from 3% to 17%.

Additionally, Barnett noted that routine blood-based, multi-cancer screening combined with improvements in single-cancer screening could prevent 40% of metastatic diagnoses and increase loco-regional diagnoses by 10%. Even without improvements in cancer therapy, ARK estimates that earlier intervention could prevent 66,000 cancer deaths per year in the US alone.

The Central Dogma could help describe how biotechnology moves forward. The Central Dogma states that DNA (the genome) is transcribed into RNA (the transcriptome), which ultimately is translated into protein (the proteome). Proteins carry out virtually all critical-to-life functions but, when altered, can cause disease.

Understanding the interactions between and among the pillars of the Central Dogma, we will improve our ability to make predictions, diagnoses, and leaps of fundamental, biological insight, Barnett said.

We believe the future of molecular biology is based on multi-omics techniques that integrate pillars of the Central Dogma. Based on our research, multi-omics including life science tools, basic and translational research, population health efforts, and molecular diagnostics could impact oncology, organ health, and population health, scaling from $110 billion to roughly $300 billion during the next five years, he added.

As a way to capture this potential growth opportunity, investors can turn to theARK Genomic Revolution Multi-Sector Fund (NYSEArca: ARKG), an actively managed strategy that seeks long-term growth of capital by investing in domestic and foreign equity securities of companies across multiple sectors, including health care, information technology, materials, energy, and consumer discretionary, that are relevant to the funds investment theme of the genomics revolution.

ARKG aims for thematic multi-cap exposure to innovative elements including gene therapy bio-informatics, bio-inspired computing, molecular medicine, and pharmaceutical innovations. The ETF aims to capture long-term growth with a low correlation of relative returns to traditional growth strategies and a negative correlation to value strategies. Additionally, the fund offers a tool for diversification due to little overlap with traditional indices. It can be a complement to traditional value/growth strategies.

Financial advisors who are interested in learning more about growth opportunities in the biotech segment can watch the webcast here on demand

See the rest here:
An Innovative ETF Opportunity That Taps Into Our Increased Understanding of the Human Genome - ETF Trends

CAMBRIDGE, Mass., June 08, 2022 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, today announced that interim clinical data from the Phase 1 study of NTLA-2001, an investigational therapy in development for the treatment of transthyretin (ATTR) amyloidosis, will be shared in an oral presentation at the European Association for the Study of the Livers International Liver Congress 2022, taking place June 22 26 in London.

We are pleased to be presenting longer-term, follow-up data from the first-in-human study of NTLA-2001, which is designed to potentially halt progression and reverse ATTR amyloidosis, said Intellia President and Chief Executive Officer John Leonard, M.D. We look forward to sharing interim data we believe demonstrates the potential of our CRISPR-based investigational therapy to be a single-dose treatment that provides a deep and durable response.

The presentation will include new data from the ongoing Phase 1 study evaluating NTLA-2001 in people living with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). These include additional results from all four dose-escalation cohorts in Part 1, highlighting the durability of response after a single dose of NTLA-2001. Data supporting the fixed dose selection for Part 2, the single-dose expansion cohort of the polyneuropathy arm, will also be presented.

The presentation will build upon interim data presented in February, which showed significant reductions in serum TTR levels from baseline in a dose-dependent manner in 15 patients with ATTRv-PN. At the highest dose tested, 1.0 mg/kg, the mean and maximum serum TTR reductions were 93% and 98%, respectively, by day 28 across the six patients treated. The Company also previously announced it is now evaluating a fixed dose of 80 mg in Part 2 of the Phase 1 study, which is expected to deliver a similar exposure to the 1.0 mg/kg dose in the ATTRv-PN target population, and that the first patient has been dosed in the expansion cohort in the polyneuropathy arm. Intellia also continues to dose patients in the cardiomyopathy arm of the Phase 1 study, which is evaluating NTLA-2001 in dose-escalation cohorts of people with ATTR amyloidosis with cardiomyopathy (ATTR-CM).

Presentation Details

Title: In vivo CRISPR/Cas9 editing of the TTR gene with NTLA-2001 in patients with transthyretin amyloidosis dose selection considerations

Session: Rare liver diseases (including paediatric and genetic)

Date and Time: Friday, June 24, 2022 from 10:00-11:30 a.m. BST

Presenter: Dr. Edward J. Gane, MBChB, MD, FRACP, MNZM, Professor of Medicine at the University of Auckland, New Zealand and Chief Hepatologist, Transplant Physician and Deputy Director of the New Zealand Liver Transplant Unit at Auckland City Hospital, the trials national investigator in New Zealand

Intellia Therapeutics Investor Event and Webcast Information

Intellia will host a live webcast on Friday, June 24, 2022 at 8:00 a.m. ET to review the presented data. To join the webcast, please visit this link, or the Events and Presentations page of the Investors & Media section of the companys website at http://www.intelliatx.com. A replay of the webcast will be available on Intellias website for at least 30 days following the call.

About Transthyretin (ATTR) AmyloidosisTransthyretin amyloidosis, or ATTR amyloidosis, is a rare, progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis occurs when a person is born with mutations in theTTRgene, which causes the liver to produce structurally abnormal transthyretin (TTR) protein with a propensity to misfold. These damaged proteins build up as amyloid in the body, causing serious complications in multiple tissues, including the heart, nerves and digestive system. ATTRv amyloidosis predominantly manifests as polyneuropathy (ATTRv-PN), which can lead to nerve damage, or cardiomyopathy (ATTRv-CM), which can lead to heart failure. Some individuals without the genetic mutation produce non-mutated, or wild-type TTR proteins that become unstable over time, misfolding and aggregating in disease-causing amyloid deposits. This condition, called wild-type ATTR (ATTRwt) amyloidosis, primarily affects the heart. There are an estimated 50,000 people worldwide living with ATTRv amyloidosis and between 200,000 and 500,000 people with ATTRwt amyloidosis.

About NTLA-2001Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially be the first single-dose treatment for ATTR amyloidosis. NTLA-2001 is the first investigational CRISPR therapy candidate to be administered systemically, or through a vein, to edit genes inside the human body. Intellias proprietary non-viral platform deploys lipid nanoparticles to deliver to the liver a two-partgenome editingsystem: guide RNAspecific to the disease-causing gene and messenger RNAthat encodes the Cas9 enzyme, which carries out the precision editing. Robust preclinical data, showing deep and long-lasting transthyretin (TTR) reduction following in vivo inactivation of the target gene, supports NTLA-2001s potential as a single-administration therapeutic. Intellia leads development and commercialization of NTLA-2001 as part of a multi-target discovery, development and commercialization collaboration with Regeneron. The global Phase 1 trial is an open-label, multi-center, two-part study of NTLA-2001 in adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) or transthyretin amyloidosis with cardiomyopathy (ATTR-CM). Visitclinicaltrials.gov(NCT04601051) for more details.

About Intellia TherapeuticsIntellia Therapeutics, a leading clinical-stage genome editing company, is developing novel, potentially curative therapeutics leveraging CRISPR-based technologies. To fully realize the transformative potential of CRISPR-based technologies, Intellia is pursuing two primary approaches. The companys in vivo programs use intravenously administered CRISPR as the therapy, in which proprietary delivery technology enables highly precise editing of disease-causing genes directly within specific target tissues. Intellias ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases. Intellias deep scientific, technical and clinical development experience, along with its robust intellectual property portfolio, have enabled the company to take a leadership role in harnessing the full potential of genome editing to create new classes of genetic medicine. Learn more at intelliatx.com. Follow us on Twitter@intelliatx.

Forward-Looking Statements This press release contains forward-looking statements of Intellia Therapeutics, Inc. (Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations concerning: its ability to successfully extend its leadership position and harness the full potential of genomic medicines to bolster its genome editing capabilities and pipeline; the safety, efficacy, success and advancement of its clinical programs for NTLA-2001 for the treatment of transthyretin amyloidosis pursuant to its clinical trial applications (CTA), including the expected timing of data releases, regulatory filings, and the initiation, enrollment, and completion of clinical trials; the expansion of its CRISPR/Cas9 technology and related technologies to advance additional development candidates and timing expectations of advancing such development candidates; its ability to maintain and expand its related intellectual property portfolio; expectations of the potential impact of the coronavirus disease pandemic, including the impact of any variants, on strategy, future operations and timing of its clinical trials; its ability to optimize the impact of its collaborations on its development programs, including, but not limited to, its collaboration with Regeneron Pharmaceuticals, Inc.

Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain its intellectual property position; risks related to Intellias relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for its product candidates, including uncertainties related to regulatory approvals to conduct clinical trials; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; the risk that clinical study results will not be positive; and the risk that Intellias collaborations with Regeneron or its other collaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with the Securities and Exchange Commission (SEC). All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Investors:Ian KarpSenior Vice President, Investor Relations and Corporate Communications+1-857-449-4175ian.karp@intelliatx.com

Lina LiDirector, Investor Relations and Corporate Communications+1-857-706-1612lina.li@intelliatx.com

Media:Matt CrensonTen Bridge Communications+1-917-640-7930media@intelliatx.com mcrenson@tenbridgecommunications.com

Go here to see the original:
Intellia Therapeutics to Present Updated Interim Clinical Data from Ongoing Phase 1 Study of NTLA-2001 for the Treatment of Transthyretin (ATTR)...

ByKen Heydon*

Trade is a key multiplier in spreading the technology vital to the climate transition. But protectionist tendencies embedded in the implementation of the climate transition pose a major threat to the global trading system.

Technological innovation backed by a carbon tax to make it competitive is the essential requirement for transition to net-zero carbon emissions by 2050. And trade, by stimulating competition, is a catalyst of innovation.

Three areas of technological transformation stand out. Australia and its Asia Pacific neighbours have a stake in each of them.

The first is solar photovoltaic technology that uses solar panels to convert sunlight into electricity. Over the past decade, solar photovoltaic has become a pillar of the low-carbon sustainable energy system with installed capacity increasing 100-fold and costs declining by 77 per cent. Some 40 per cent of the decline in the cost of solar photovoltaic modules since 2001 isattributableto trade.

With the highest solar radiation per square meter of any continent, Australia is particularly well placed to profit from solar photovoltaic technology, as testified by the proposed DarwinSingapore undersea renewables-generated electricity cable. Malaysia, Vietnam, Thailand and the Philippines rank among the top 10 global exporters of selected solar photovoltaic goods.

The second area of trade-augmented innovation is the export of low-carbonhydrogen, important in hard-to-electrify sectors like steel production and facing a potential six-fold increase in demand by 2050.

Australia is at the forefront in using renewable energy to produce zero-emissions ammonia a low-cost carrier for hydrogen and no less than six Japanese corporations havehydrogen development projects in Australia.

The third area of innovation is CRISPR (clustered regularly interspaced short palindromic repeats). This gene-editing helps countries decarbonise their food systems by making export crops that are resistant to disease or bad weather, reducing the need for increased farmland via deforestation the source of 10 per cent of global carbon emissions.

For example, CRISPR-based genome editing of oil palm to eradicate Basal Stem Rot will significantly reduce plant loss and subsequent compensatory deforestation in Indonesia and Malaysia the leading exporters of palm oil. A switch to trade in gene-edited palm oil promises major environmental benefits.

Beyond trades multiplier role in spreading the benefits of specific technologies, it also has a more general function in stimulating the economic growth necessary to fund the energy transformation.

Yet despite trades role in the climate transition, the transition itself threatens trade. As countries environmental commitment varies, pressure mounts fortariff restrictionson imports of carbon-intensive products, like steel, from perceived environmental free riders.

The difficulty of identifying carbon intensity and free riding fuels the view that such penalties are simply disguised protectionism. The resulting tensions augmented by the climate transitions emotive power and by broader protectionist tendencies, including as countries favour their own climate technologies pose a major threat to the trading system.

In each of the three areas of technological promise there are strong contradictory forces of trade restriction.

Trade in solar photovoltaic technology is subject to both tariff and non-tariff barriers. For example, 31 WTO members apply tariffs over 10 per cent to certain essential solar photovoltaic materials such as polysilicon. Australia scores an own-goal by imposing duties (both dumping and countervailing) on solar equipment imports thus raising the costs of renewable electricity.

The threat to trade in hydrogen comes from a call by competition-fearing European Union electricity groups for a carbon tariff on EU hydrogen imports. Though the case for Australias blue hydrogen using brown coal and carbon capture and storage is controversial, a Carbon Border Adjustment Mechanism tax risks application to all hydrogen imports and a setback to carbon capture and storage, without which net zero will be impossible.

The threat to CRISPR gene-editing is a repeat of the type of regulatory barriers that invoke health and environmental concerns to hinder the development and trade of genetically modified organisms. Many countries farming regulations, including those of New Zealand, constrain both GMOs and CRISPR, even though CRISPR does not introduce DNA from other species.

The challenge is real. Protectionism risks hobbling trades role in fostering the technological promise of the climate transition raising the level of the carbon tax needed to effect that transition and weakening public support for it.

Resisting protectionist forces will require action at all levels. Domestically, by improving coherence in national policy, including in Australia in respect to solar equipment. Regionally, with a key role for APEC in respect to bothenvironmental goodsandservices. And multilaterally, where Australia is among 13 countries (including the United States and Canada) that have submitted to the WTO that gene-edited products should be regulated in the same way as conventional ones.

The stakes are particularly high for theWTO, which is about to face via the climate transition one of the most severe systemic challenges in its history.

*About the author: Ken Heydon is Visiting Fellow at the London School of Economics and Political Science. He is a former Australian trade official and senior member of the OECD secretariat.

Source: This article was published by East Asia Forum

Read more from the original source:
Protectionism Threatens The Climate Transition Analysis Eurasia Review - Eurasia Review

(March 2020)

It is predicted the development of a safe and effective vaccine to prevent COVID-19 will take 12 to 18 months, by which time hundreds of thousands to millions of people may have been infected. With a rapidly growing number of cases and deaths around the world, this emerging threat requires a nimble and targeted means of protection.

Could CRISPR be the next virus killer?

To address this global pandemic challenge, we are developing a genetic vaccine that can be used rapidly in healthy and patients to greatly reduce the coronavirus spreading. We developed a safe and effective CRISPR system to precisely target, cut and destroy COVID-19 virus and its genome, which stops coronavirus from infecting the human lung.

Weve shown that the CRISPR system can reduce 90% of coronavirus load in human cells. It can also protect humans against essentially 90% of all current and emerging coronaviruses. The project is ongoing, and we are working around the clock towards getting an actual product by combing our CRISPR method with an inhaler-based delivery device.

The project will likely to result in a potential therapeutics towards COVID-19, which can help slow down or eliminate the outbreak.

Relevant Publications or More Information

A preprint of the work (not yet peer reviewed)

Feature in WIRED magazine(March 18, 2020)

Collaborators:

Stanley Qi(Bioengineering)

David Lewis(Pediatrics-Immunology)

Jennifer Cochran(Bioengineering)

Drew Endy(Bioengineering)

Go here to see the original:
New genetic method of using CRISPR to eliminate COVID-19 virus genomes ...

Gado/Getty Images

CRISPR Therapeutics, which as its name suggests, is focused on using CRISPR-Cas9 gene editing to generate gene-based therapies for serious diseases, is expecting a busy year. And as reported in its first-quarter financials, as of March 31, it had $2.221 billion in cash, cash equivalents and marketable securities to drive its progress.

The company, along with collaboration partner Vertex Pharmaceuticals, expects to present updated data from its ongoing Phase III trials for CTX001 in transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD). They had announced that more than 75 patients in both trials had been dosed last quarter. They have also initiated two new Phase III trials of the therapy in pediatric patients with TDT and SCD. They plan to submit global regulatory filings for CTX001 for those two indications later this year.

I am pleased with the ongoing momentum across our broad portfolio of innovative gene therapy candidates and anticipate important company milestones in 2022, stated Samarth Kulkarni, Ph.D., chief executive officer of CRISPR.

On February 2, CRISPR and ViaCyte dosed the first patient in a Phase I trial of VCTX210 for treatment of type 1 diabetes (T1D). VCTX210 is an allogeneic, gene-edited, stem cell-derived therapy designed to generate pancreatic cells that can evade recognition by the immune system. In theory, these replacement cells would allow patients to produce their own insulin.

We are excited to work with CRISPR Therapeutics and ViaCyte to carry out this historic, first-in-human transplant of gene-edited, stem cell-derived pancreatic cells for the treatment of diabetes designed to eliminate the need for immune suppression, said Dr. James Shapiro, M.D., Ph.D., Canada Research Chair, Director of the Islet Transplant Program at the University of Alberta, and a clinical investigator of the trial. If this approach is successful, it will be a transformative treatment for patients with all insulin-requiring forms of diabetes.

In addition to that trial, the company indicated in its quarterly report that based on progress with its in vivo techniques for liver gene editing using both viral and non-viral vectors, is expected to move multiple programs leveraging in vivo approaches into the clinic in the next 18 to 24 months.

CRISPR has also made inroads with its immuno-oncology programs. It is continuing to enroll and treat patients in the Phase III trial of CTX110, an allogeneic chimeric antigen receptor T cell (CAR-T) therapy targeting CD19+ B-cell malignancies. It expects to report additional data this year. And it also has ongoing Phase I trials for CTX-120, its wholly-owned allogeneic CAR-T therapy targeting B-cell maturation antigen for relapsed or refractory multiple myeloma, and CTX130, a CAR-T therapy targeting CD70 for solid tumors and certain hematologic malignancies. CRISPR Therapeutics expects to offer updates for both trials in the first half of this year.

Total collaboration revenue was $0.2 million for the first quarter of 2022 and 2021. For this quarter, R&D expenses were $118.2 million, up from $70.6 million in the same period last year. This was driven largely by its immuno-oncology programs and expenses associated with its new U.S. R&D headquarters. General and administrative (G&A) expenses were $28.0 for the first quarter, up from $24.5 million in the first quarter of 2021, driven by headcount-related expenses. Overall, it reported a net loss of $179.2 million for the first quarter.

Simply Wall St. conducted an analysis of CRISPR Therapeutics investors. It noted the company has a market capitalization of $3.7 billion, and usually a company this size would have a high percentage of institutional investors, which does seem to be the case with CRISPR. This suggests some credibility amongst professional investors. But we cant rely on that fact alone since institutions make bad investments sometimes, just like everyone does, they write.

Institutional investors own more than half the issued stock that is not owned by hedge funds. The largest shareholder is ARK Investment Management, with 12%; the second largest, also with 12% of common stock, is Nikko Asset Management Col, and third, Capital Research and Management Company owns about 5.3% of the company stock.

In terms of the shareholders, 50% of ownership is controlled by the top 25 shareholders, meaning that no single shareholder has a majority interest in the ownership. Insider ownership makes up about $54 million in shares at current prices, while individual public investors hold a 35% stake. While this size of ownership may not be enough to sway a policy decision in their favor, they can still make a collective impact on company policies, writes Simply Wall St.

Kulkarni concluded, saying, We believe we are well-positioned and well-capitalized to advance our pipeline and platform to develop transformative medicines for patients suffering from serious disease.

Read more from the original source:
Cash-Rich CRISPR Therapeutics Expects Year Filled with Milestones - BioSpace

The CRISPR-Cas9 Market Report Forcast to 2022-2028 offers an in-depth analysis of driving factors, opportunities, restraints, and challenges for gaining key insight into the market. The study includes porters five forces model, attractiveness analysis, and competitor position grid analysis.The study also contains information about the CRISPR-Cas9 industrys regulatory environment, which will assist you in making an informed decision. The paper goes through the important regulatory agencies, as well as the major rules and regulations that have been put on this industry in different parts of the world.

Get Updated Sample Report (Get Higher Priority for Corporate Email ID): https://www.worldwidemarketreports.com/sample/493454

Segmentation based on Key players

Editas Medicine CRISPR Therapeutics Horizon Discovery Sigma-Aldrich Genscript Sangamo Biosciences Lonza Group Integrated DNA Technologies New England Biolabs Origene Technologies Transposagen Biopharmaceuticals Thermo Fisher Scientific Caribou Biosciences Precision Biosciences Cellectis Intellia Therapeutics

Segmentation based on Type

Biotechnology Companies Pharmaceutical Companies Others

Segmentation based on Application

Genetic Engineering Gene Library Human Stem Cells Others

Request a PDF Brochure here: https://www.worldwidemarketreports.com/sample/493454

The study also includes a regional analysis that covers various regions and contributes to the CRISPR-Cas9 markets growth. It includes the competitive landscape, which comprises the leading players, as well as strategies for announcing collaborations, introducing new products, and collaborating to help the CRISPR-Cas9market expand between 2022 and 2028. Furthermore, the research analyst has used a variety of research approaches to gather data on current trends and industry advancements that will drive CRISPR-Cas9market growth throughout the projection period.

Summary

Our research analysts will assist you in obtaining customized information for your report, which can be tailored to a particular region, application, or statistical detail. Furthermore, we are always happy to cooperate with a study that is triangulated with your own data in order to make the CRISPR-Cas9market research more thorough in your eyes.

An Overview of the Impact of COVID-19 on this Market:

COVID-19 appearance has brought the entire globe to a halt. We recognize that the current health crisis has had a significant impact on businesses in a variety of sectors. Governments and businesses are rallying to tackle this potentially dangerous disease. Some industries are doing well, while others are failing. During COVID-19 pandemics, we are working hard to keep your company afloat and growing. We will provide you with impact analysis of the coronavirus outbreak across industries, based on our knowledge and expertise, to assist you in planning for the future.

To learn more about the impact of Covid-19 in this report, go to: https://www.worldwidemarketreports.com/covidimpact/493454

Competitiveness Quadrants

A Competitive Quadrant, a patented methodology for analyzing and evaluating organizations positions based on their Industry Position and Market Performance scores, is also included in the report. The tool categorizes the participants into four groups depending on a number of factors. Financial performance from the last few years, growth strategies, innovation techniques, new product launches, investments, CRISPR-Cas9market share growth, and other factors are all taken into consideration.

What are the advantages of purchasing this report?

FAQ:

Buy this Research report Complete With Tables, Figures, Charts, and More:https://www.worldwidemarketreports.com/buy/493454

Contact Us :

Mr. ShahWorldwide Market Reports,Tel: U.S. +1-415-871-0703U.K. +44-203-289-4040Japan +81-50-5539-1737Email: [emailprotected]Website: https://www.worldwidemarketreports.com/

Read more:
COVID -19 Impact Study: CRISPR-Cas9 Market Is Expected to Witness an Incredible Growth by 2028: Editas Medicine, CRISPR Therapeutics, Horizon...

The following discussion and analysis of our financial condition and results ofoperations should be read in conjunction with (i) our unaudited condensedconsolidated financial statements and related notes appearing elsewhere in thisQuarterly Report on Form 10-Q and (ii) our audited consolidated financialstatements and related notes and management's discussion and analysis offinancial condition and results of operations included in our Annual Report onForm 10-K for the year ended December 31, 2021 filed with the Securities andExchange Commission, or the SEC, on February 15, 2022. Some of the informationcontained in this discussion and analysis or set forth elsewhere in thisQuarterly Report on Form 10-Q, including information with respect to our plansand strategy for our business and impact and potential impacts on our business,includes forward-looking statements that involve risks and uncertainties. As aresult of many factors, including, without limitation, those factors set forthin the "Risk Factors" section of our Annual Report on Form 10-K for the yearended December 31, 2021 and the "Risk Factors" section of subsequent QuarterlyReports on Form 10-Q, our actual results or timing of certain events coulddiffer materially from the results or timing described in, or implied by, theseforward-looking statements.

Special Note About Coronavirus (COVID-19)

Overview

We and Vertex have also initiated two additional Phase 3 clinical trials ofCTX001 in pediatric patients with TDT and SCD.

Immuno-Oncology

In addition, we are developing our own portfolio of CAR-T cell productcandidates based on our gene-editing technology.

In the fourth quarter of 2021, we released updated clinical data from theongoing CARBON trial for 26 patients treated with CTX110 who had reached atleast 28 days of follow-up.

Regenerative Medicine

In Vivo

Partnerships

Financial Overview

Revenue Recognition

Research and Development Expenses

employee-related expenses, including salaries, benefits and equity-basedcompensation expense;

costs of services performed by third parties that conduct research anddevelopment and preclinical and clinical activities on our behalf;

costs of purchasing lab supplies and non-capital equipment used in ourpreclinical activities and in manufacturing preclinical and clinical studymaterials;

facility costs, including rent, depreciation and maintenance expenses; and

fees and other payments related to acquiring and maintaining licenses under ourthird-party licensing agreements.

successful completion of preclinical studies and IND-enabling studies;

successful enrollment in, and completion of, clinical trials;

receipt of marketing approvals from applicable regulatory authorities;

establishing commercial manufacturing capabilities or making arrangements withthird-party manufacturers;

obtaining and maintaining patent and trade secret protection and non-patentexclusivity;

launching commercial sales of the product, if and when approved, whether aloneor in collaboration with others;

acceptance of the product, if and when approved, by patients, the medicalcommunity and third-party payors;

effectively competing with other therapies and treatment options;

a continued acceptable safety profile following approval;

enforcing and defending intellectual property and proprietary rights and claims;and

achieving desirable medicinal properties for the intended indications.

Except for activities we perform in connection with our collaborations withVertex and ViaCyte, as well as in connection with the Bayer Transaction, we donot track research and development costs on a program-by-program basis.

General and Administrative Expenses

Collaboration Expense, Net

Collaboration expense, net, consists of collaboration costs under ourcollaboration with Vertex.

Other Income (Expense), Net

Other income (expense), net consists primarily of interest income earned oninvestments.

Results of Operations

Comparison of three months ended March 31, 2022 and 2021 (in thousands):

3,504

Research and Development Expenses

$8.8 million of increased facility-related expenses, primarily related to ournew U.S. research and development headquarters;

$3.7 million of increased license fees; and

$2.9 million of increased consulting and professional services costs.

General and Administrative Expenses

$2.2 million of increased employee compensation, primarily due to increasedstock-based compensation expense of $1.9 million; and

$1.3 million of increased consulting and professional services costs.

Collaboration Expense, Net

$6.5 million of increased pre-commercial expenses associated with ourcollaboration with Vertex;

$5.3 million of increased manufacturing costs; offset by

$1.3 million of decreased other costs.

Other Income, Net

Liquidity and Capital Resources

Outlook

Cash Flows

The following table provides information regarding our cash flows for each ofthe periods below (in thousands):

Period to Period

Net cash used in operating activities $ (135,239 ) $ (100,663 ) $ (34,576 )Net cash used in investing activities

Investing Activities

Financing Activities

Critical Accounting Policies and Significant Judgments and Estimates

Recent Accounting Pronouncements

Edgar Online, source Glimpses

More here:
CRISPR THERAPEUTICS AG Management's Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) - Marketscreener.com

He Jiankui, known to his international colleagues as JK, is a scientist who was well-respected in the American gene editing community, but pushed the boundaries by gene-editing on an embryo.PHOTO ILLUSTRATION: BRYAN GEE/THE GLOBE AND MAIL. SOURCE: AP Photo/Kin Cheung

Samira Kiani is a professor of genetic engineering at the University of Pittsburgh. She is the co-producer of Make People Better, which premiered at this week at Hot Docs. The views presented are those of the author and do not necessarily represent the views of the University of Pittsburgh. This essay was co-written with journalist Brian Barth.

In October, 2018, I was invited to a secret meeting in Guangzhou, China. I was there because of my work as a genetic scientist who uses the CRISPR technology to cut and splice DNA, an approach to genetic engineering that has come to the forefront over the past decade. I dont think its an overstatement to say that CRISPR, a precise and efficient tool that allows us to edit genes, is on the verge of altering the course of human history to an extent far greater than the recent disruptions catalyzed by internet technology. If you think digital surveillance tools are frightening in the hands of autocracy, consider the power to bend the human genome to ones will. CRISPR provides that power. To use another analogy, the ability to edit genes with surgical precision is a scientific discovery on par with nuclear fission while there may be beneficial applications, it is by nature seductive to our darkest impulses.

Because of CRISPRs unknown risks, its use has been limited to certain applications by longstanding consensus within the scientific community, and to a lesser extent by regulatory agencies. Weve experimented extensively in petri dishes and increasingly on live animals. Theres been limited experimentation on human embryos in the lab, but a firm line has been drawn: Edited embryos are not to be implanted in womens bodies.

The man I met with in the lobby of the Westin hotel in Guangzhou had crossed that line. His name is He Jiankui, and his story has since been told. This is the first time Im telling my version.

I was in Guangzhou, a hotbed of biotech research, making a film that looks at all sides of this god-like power. Im as interested in the ethical implications of my work as I am in the technology itself, and have long worked at the intersections of science, art and society. Dr. He, known to his international colleagues as JK, was seeking a messenger, someone who could translate his work to the world in a way that would elicit sympathy, rather than horror. He seemed to think this was his chance.

Needless to say, our meeting changed the course of the film. It also changed the course of his life, and not for the better he spent the next three years in prison after being convicted of illegal medical practice. He was released in April.

For me, the meeting in Guangzhou was a turning point. It clarified something Id sensed internally for years: The dark sides of technological advancement have roots in the culture of science. Ive been part of that culture my entire life my childhood in Iran as the daughter of veterinary scientists; the medical school in Tehran my family pushed me to attend; my postdoctoral work at Massachusetts Institute of Technology with hard-charging CRISPR pioneers; my marriage to a stem cell researcher. If youre not at the top of your class, publishing in the most prestigious journals, getting the biggest grants and making the biggest breakthroughs, then youre a nobody. Its a culture of more, better, faster; of accomplishment at any cost, whether personal or societal. If someone gets hurt, if theres collateral damage so what? Its inevitable in the brutal contact sport of science.

Ive come to have a measure of empathy for JK because I understand that his choices are a product of the culture of the scientific community, a community that ultimately threw him under the bus rather than face its complicity. My meeting in Guangzhou put me on a path to change that culture. It hasnt been easy.

When JK invited our film crew to the off-the-record meeting, he did not give us the full story. He said he would soon implant edited embryos into human subjects and asked if this was something wed like to document on film. Turned out he had already done the deed twins given the pseudonyms Lula and Nana had been born just before we met (this was unearthed shortly after our meeting by the journalist Antonio Regalado, a subject in the documentary who wed invited to join us at the hotel). When the news broke, the Western scientific community feigned disgust JK was a rogue, conveniently located in a distant autocracy, who had carried out this horrible project in secret. That narrative could not have been further from the truth.

While I did not personally know him, plenty of scientists in the West did, and hed openly shared what he was up to with some of them. JK, was a young, highly ambitious scientist. Hed been educated at Stanford and was well-respected in the American gene editing community. He aimed high in his career, as in Nobel laureate-level. Nuclear fission-scale discoveries are what win Nobel Prizes never mind what happens with the technology later. He knew that the birth of gene edited human babies would shock the world; it was a calculated risk, in which he hoped the shock would subside into acceptance. He imagined that history would eventually celebrate him as the first.

JKs colleagues did not call him out before the news leaked because what he was doing, while considered taboo by society at large, is seen as an inevitability among CRISPR scientists. The fact that we edit human embryos at all should make it obvious that implanting those embryos is the anticipated next step. It is less scientific knowledge holding back the industry than social licence and regulatory environment.

The culture of science was pushing JK forward. Like me and many of my colleagues, he wanted to change the world for the better, and he understood that this did not happen by colouring within the lines. It requires pushing boundaries the question is which ones you push. The answers, I believe, should come not from a sense of competition with the guy next to you on the lab bench, but from the broadest possible cross-section of human communities. The culture of science is like a vacuum perhaps because we know the general public often does not understand what we do, we dont feel like were obligated to explain ourselves, much less to elicit input from beyond our silo walls. Integrating input from diverse perspectives is, for many scientists, a foreign concept, something that would divert from ones goals and slow the process of discovery. Theres truth in that, but I think it would redirect scientific goals to be more in alignment with human needs. Moving slower and not breaking things has its upsides.

Pushing boundaries is essential to human achievement, but the harder the push, the greater the responsibility. At a minimum, you need consent from those involved, which is why editing the DNA of a human embryo is an ethical abyss: Unborn children cannot give consent. In any matter related to public health, there must also be some form of collective consensus about the risks involved and when it comes to the integrity of the human gene pool, the bar couldnt be higher.

In JKs case, he deluded himself into thinking he had consensus to proceed with his experiment. During our meeting, he showed me the results of public opinion surveys indicating relatively strong support for gene editing in China. He had also obtained ethics approval from the medical institution involved in the project (Chinese authorities later claimed the approval was fraudulent). He was also banking on support for the specifics of the experiment to make Lulu and Nana genetically immune to HIV. While theres an obvious selling point for the public there, we have other tried and true methods at our disposal for controlling the spread of HIV. What the layperson would not realize, however, is that his choice in which genes to work with was also a matter of scientific expediency, as the mutation associated with HIV resistance, known as CCR5-delta 32, has been intensely studied and thus represents a low barrier to entry for someone wanting to feel reasonably confident that nothing will go awry when they start editing embryos.

Nonetheless, the experiment did not go as planned. We inherit a pair of each gene that comprises our genome (one from our mother and one from our father), but one of the twins embryos came out half-edited, meaning the intended change happened only on the copy from one parent. Second, he was unable to confirm that the genetic changes made in the embryos resulted in immunity to HIV. The fact that JK chose to implant the embryos anyway he claimed this was in part because the parents urged him to is a core ethical lapse largely overlooked in press reports.

JK had tacit support from the Chinese government for his work, but when the situation became suddenly scandalous in late 2018, they turned on him, just like his colleagues. Even if theyd stood by him, I dont believe the approval of a single jurisdiction has any relevance when it comes our shared genome, which transcends national borders. In my mind, to alter genes that will be passed down from generation to generation is a sacred act. Placing responsibility for it in the hands of regulatory agencies is to deny the full power and meaning of it. If we choose this path through a truly responsible process a big if there would be no need for the scientists involved to feel like they need to hide what theyre doing. They would be vaunted in the temples of the world.

Still from the film Make People Better, directed by Cody Sheehy and produced by author Samira Kiani shows the gene edited twin girls, nicknamed Lulu and Nana, who were born at an undisclosed hospital in China.COURTESY HOTDOCS AND Getty Images

I am by no means against gene editing it is my passion and livelihood. It has potential to cure our most intractable diseases and possibly address some of our most pressing environmental issues. Part of the promise of CRISPR is safety: Because it is so precise, the risk of unintended negative outcomes is much smaller and easier to mitigate than the previous generation of genetic engineering tools. We have conclusive evidence regarding safety for many applications of CRISPR technology. But this is not universally the case.

My own research involves somatic gene editing, a therapeutic application of CRISPR administered by injection to consenting subjects. JK, and a small number of other scientists, have experimented with germline editing, in which changes are made to the DNA of an embryo. Theres a huge difference: With somatic gene editing, the altered genetic traits cannot be passed to future generations, but with germline they can, permanently releasing those new traits into the gene pool. This makes germline far riskier.

We simply do not know the biological implications of human germline editing, and cant fully know them without waiting 80 years to see how an embryos altered genome plays out over the course of a lifetime. The culture of science is not equipped with that sort of patience. The social and political implications of gene editing are also huge unknowns, and unfortunately theres been scant debate among policy makers and the general public. Within the small, insular world of CRISPR researchers, it is assumed that work similar to JKs is under way, most likely in privately funded labs, or in countries where the idea of engineering humans for morally questionable reasons think designer babies and superhuman soldiers could be viewed not as taboo, but as politically and economically desirable.

At this point, we have little ability to anticipate the unintended negative consequences that might result from germline editing, but such uncertainties have not stopped the forces of hubris in the past. Nor have they stopped the forces of capital. I expect that human genetic engineering, both therapeutic and vanity applications, will become an unfathomably profitable industry in the coming decades. I am open to the possibility that germline editing could be ethically applied in narrowly defined arenas of public health. But the determination of where to draw the lines should not be left up to venture capitalists and self-centred founders, as it was to a large extent with the IT industry. They are already salivating.

In many ways, the present state of gene editing technologies resembles that of internet technologies 25 years ago on the cusp of changing the world, but still obscured behind closed doors. Many of us now wish that wed better understood the implications of those technologiesthen. If theres any hope of the biotech industry of the future having a moral, equitable and inclusive basis, the rules of engagement need to be determined now.

How do we catalyze a meaningful public debate before its too late? First, the public needs to better understand how theses technologies work and what the implications are. I believe communicating that should be part of the job description for all scientists. Second, scientists need to approach their work with a more holistic lens. The scientific method is by nature reductive, but we need not be reductive in deciding how to apply it and which paths of inquiry to pursue.

I have profound respect for the intellect and integrity of my colleagues. But I see a direct link between a difficulty with introspection or perhaps unwillingness, Im not sure which and the ethical shortcomings of my community. I often wonder how our discoveries, and the world that they foster, would look if EQ was valued as much as IQ. Im not saying individual scientists lack emotional intelligence in their personal lives, but on a collective level we do. Its evident in how we practise our work and in our relationship with society at large. Pushing ones way to the front of the line is not a sign of high EQ. But thats the culture of science. Ignoring what other people feel because it does not align with our needs or desires does not make for good relationships. But it makes for a successful scientist.

I dont have all the answers. But Im trying to transform the tiny domains where I have influence. Im developing a virtual museum to help educate the public about gene editing, highlighting both the benefits and the dangers. Im producing films and theatre that ask the hard questions about boundary-pushing innovations not just genetic engineering, but others on the verge of altering human history, such as AI and brain-machine interfaces in entertaining and unexpected ways. I am also one of the co-creators of Our Future Life, a global storytelling initiative aimed at breaking down barriers to dialogue around the biggest questions facing humanity. The common thread in all these is to engage the public imagination and encourage deep reflection. Perhaps most importantly, I work on a daily basis to instill these perspectives in my students. Believe me, it is an uphill battle.

When I was invited recently to give a guest lecture for a colleagues class on epigenetics, I decided to use the opportunity to facilitate a discussion about who the students are as scientists, rather than giving a dry talk on theory and technique. A month later, the professor whod invited me informed that Id received horrible reviews in the class evaluations. My presentation was not pertinent to the material, one student wrote. I couldnt disagree more.

Keep your Opinions sharp and informed. Get the Opinion newsletter. Sign up today.

See the rest here:
Opinion: Human genetic engineering is coming. We must discuss the social and political implications now - The Globe and Mail

This article was originally published here

Yonsei Med J. 2022 May;63(5):480-489. doi: 10.3349/ymj.2022.63.5.480.

ABSTRACT

PURPOSE: Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) is the pathogen of coronavirus disease 2019. Diagnostic methods based on the clustered regularly interspaced short palindromic repeats (CRISPR) have been developed to detect SARS-CoV-2 rapidly. Therefore, a systematic review and meta-analysis were performed to assess the diagnostic accuracy of CRISPR for detecting SARS-CoV-2 infection.

MATERIALS AND METHODS: Studies published before August 2021 were retrieved from four databases, using the keywords SARS-CoV-2 and CRISPR. Data were collected from these publications, and the sensitivity, specificity, negative likelihood ratio (NLR), positive likelihood ratio (PLR), and diagnostic odds ratio (DOR) were calculated. The summary receiver operating characteristic curve was plotted for analysis with MetaDiSc 1.4. The Stata 15.0 software was used to draw Deeks funnel plots to evaluate publication bias.

RESULTS: We performed a pooled analysis of 38 independent studies shown in 30 publications. The reference standard was reverse transcription-quantitative PCR. The results indicated that the sensitivity of CRISPR-based methods for diagnosis was 0.94 (95% CI 0.93-0.95), the specificity was 0.98 (95% CI 0.97-0.99), the PLR was 34.03 (95% CI 20.81-55.66), the NLR was 0.08 (95% CI 0.06-0.10), and the DOR was 575.74 (95% CI 382.36-866.95). The area under the curve was 0.9894.

CONCLUSION: Studies indicate that a diagnostic method based on CRISPR has high sensitivity and specificity. Therefore, this would be a potential diagnostic tool to improve the accuracy of SARS-CoV-2 detection.

PMID:35512751 | DOI:10.3349/ymj.2022.63.5.480

Link:
Evaluation of CRISPR-Based Assays for Rapid Detection of SARS-CoV-2: A Systematic Review and Meta-Analysis - DocWire News

CRISPR Therapeutics (NASDAQ:CRSP Get Rating) had its price objective decreased by investment analysts at Barclays from $107.00 to $99.00 in a report issued on Tuesday, The Fly reports. Barclayss price target would indicate a potential upside of 129.01% from the companys current price.

Other research analysts have also recently issued research reports about the stock. Credit Suisse Group initiated coverage on shares of CRISPR Therapeutics in a research report on Thursday, April 28th. They set a neutral rating and a $78.00 price target on the stock. Chardan Capital reduced their price objective on shares of CRISPR Therapeutics from $171.00 to $168.00 in a report on Tuesday. Truist Financial restated a buy rating and issued a $220.00 price objective on shares of CRISPR Therapeutics in a report on Wednesday, February 16th. The Goldman Sachs Group reduced their price objective on shares of CRISPR Therapeutics from $179.00 to $87.00 and set a neutral rating on the stock in a report on Thursday, February 17th. Finally, Stifel Nicolaus cut their price target on shares of CRISPR Therapeutics to $64.00 in a research note on Wednesday, February 16th. They noted that the move was a valuation call. One investment analyst has rated the stock with a sell rating, seven have issued a hold rating and ten have given a buy rating to the stock. According to MarketBeat, CRISPR Therapeutics presently has an average rating of Buy and a consensus target price of $124.64.

Shares of CRSP opened at $43.23 on Tuesday. CRISPR Therapeutics has a 52-week low of $43.05 and a 52-week high of $169.76. The firms fifty day moving average is $59.95 and its two-hundred day moving average is $69.03. The stock has a market cap of $3.35 billion, a PE ratio of 9.78 and a beta of 2.05.

A number of institutional investors and hedge funds have recently made changes to their positions in the stock. Sumitomo Mitsui Trust Holdings Inc. acquired a new position in CRISPR Therapeutics in the first quarter valued at approximately $289,982,000. Nikko Asset Management Americas Inc. boosted its holdings in shares of CRISPR Therapeutics by 7.0% in the first quarter. Nikko Asset Management Americas Inc. now owns 4,619,747 shares of the companys stock valued at $291,044,000 after acquiring an additional 300,276 shares during the period. Loomis Sayles & Co. L P boosted its holdings in shares of CRISPR Therapeutics by 42.1% in the fourth quarter. Loomis Sayles & Co. L P now owns 1,316,123 shares of the companys stock valued at $99,736,000 after acquiring an additional 390,209 shares during the period. BlackRock Inc. boosted its holdings in shares of CRISPR Therapeutics by 12.6% in the fourth quarter. BlackRock Inc. now owns 1,271,528 shares of the companys stock valued at $96,358,000 after acquiring an additional 142,244 shares during the period. Finally, Bellevue Group AG boosted its holdings in shares of CRISPR Therapeutics by 0.8% in the third quarter. Bellevue Group AG now owns 974,684 shares of the companys stock valued at $109,096,000 after acquiring an additional 7,800 shares during the period. 56.12% of the stock is owned by hedge funds and other institutional investors.

CRISPR Therapeutics Company Profile (Get Rating)

CRISPR Therapeutics is a gene-editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene-editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases.

Featured Stories

Receive News & Ratings for CRISPR Therapeutics Daily - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings for CRISPR Therapeutics and related companies with MarketBeat.com's FREE daily email newsletter.

Read more:
CRISPR Therapeutics (NASDAQ:CRSP) PT Lowered to $99.00 at Barclays - Defense World

Since the academic discovery of gene editing, farmers, researchers and all of agriculture have heard many promises about its potential. The trouble is, theres all this excitement without taking a realistic look at what is needed to apply the technology in a way that it is in line with commercial needs.

Were at a point that we need to figure out these challenges or CRISPR wont work in the real world.

Next to the obvious regulatory and licensing hurdles, these challenges include considerable technical obstacles which still need to be overcome.

At Hudson River Biotechnology, weve been working on a CRISPR workflow for four years. We gave our workflow the acronym TiGER. One of the biggest hurdles we worked through was finding a way to obtain a homogenous, edited plant within a commercially attractive timeline.

This differs per crop, but in all cases meant stepping away from the use of transgenes, which is the primary academic way of applying CRISPR. Two great bonuses here are that our method is more controllable and therefore much more efficient and that without transgenes it can be applied in a much wider range of crops.

Whatever editing approach you work with, it goes hand in hand with the challenge of regenerating edited cells into a plant. Besides stepping away from transgenes, weve learned that the key to considerably reduce the time to market lies in single-cell regeneration.

Single-cell regeneration means that after editing, individual protoplasts are regenerated into a whole plant. The benefit of this is that no additional measures have to be taken to guarantuee genetic stability. In other words, we can yield a transgene-free, genetically homogenous plant in one generation. Weve adopted an effective, standardized single-cell regeneration approach and are making great strides with recalcitrant species by using smart delivery molecules, regeneration boosters and advanced materials adopted from the pharma world.

Cas9, while it made many headlines, is associated with a very expensive license. We needed to find something with high efficacy but a more affordable cost structure, which eventually brought us to the MAD7 protein. While there was less technical know-how for this protein, such as guide-design rules, and it is not commercially available, we tackled this by developing our own prorietery software and producing high-grade MAD7 in house. Thanks to this, we have been able to obtain very high editing effiencies and have managed precise gene editing, while using a protein that is commercially attractive for our clients.

Still, strides need to be made to harnass CRISPRs full potential. We still need to continue advancing the science behind CRISPR technologies and need to work diligently to inform consumers and politicians about what gene editing is. Its not transgenic, and we need to be clear about that.

From a communications perspective, we need to be talking with people outside of agriculture more. I mentioned regulatory hurdles as one of the challenges to using CRISPR, and if we dont explain science to the public, it could become an insurmountable challenge. We cant let gene editing go down the same public perception route that GMOs did we need this technology too much.

From a technical perspective, we need to spend time working with more crops and more genes. As you can imagine, we know a lot more about some crops staples like corn, soybeans, etc. than others, and its the same for their DNA. If we can do more research, well better understand how genes work together and how to impact important traits such as disease and insect resistance.

We also need to enhance our CRISPR capabilities to do precise gene-editing and target complex traits. Theoretically, all positions in the genome can be customized. In practice however, some genomic sites are poorly edited and repair is rarely precise in plants.

We continuously innovate to make precise editing of any position in the genome a reality. We also want to do this simultaneously at many different sites (multiplexing). This, in combination with more insight in how regulatory elements control (tissue specific) gene expression and how multiple genes conver complex traits, will allow us to truly harvest the full potential of CRIPSR. Transcending it from the blunt on-off switch that it is now, to a volume regulator that allows us to tune to any trait required and meet future crop challenges in this changing world.

Read the original post here

See original here:
Viewpoint: What are the most serious hurdles blocking mass adoption of gene editing in agriculture? - Genetic Literacy Project

Enter genomic DNA sequence to find CRISPR target sites:(Omit header lines and special characters.)

Select genome:

---- Drosophila melanogaster ---- Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila melanogaster (reference genome, r_6) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila melanogaster (vas-cas9 III, BDSC 51324) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila melanogaster (nos-cas9 II, BDSC 78781) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila melanogaster (nos-cas9 III, BDSC 78782) ---- Other Drosophila Species ---- Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila ananassae (dana_r1.3_FB2011_07) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila erecta (dere_r1.3_FB2011_08) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila eugracilis (GCA_000236325.2) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila grimshawi (dgri_r1.3_FB2010_02) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila mauritiana (Dmau_MS17) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila mojavensis (dmoj_r1.3_FB2011_05) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila persimilis (dper_r1.3_FB2010_02) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila pseudoobscura (dpse_r3.1_FB2013_03) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila sechellia (droSec1) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila simulans (DsimV2) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila simulans (droSim1) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila virilis (droVir3) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila willistoni (dwil_r1.3_FB2010_02) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Drosophila yakuba (droYak2) ---- Other Organisms ---- Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Acyrthosiphon pisum (2) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Aedes aegypti (AaegL5.0 GCA_002204515.1) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Anopheles gambiae (strain M) (AgamM1) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Anopheles gambiae (strain S) (AgamS1) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Apis cerana cerana (GCA_002290385.1) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Apis mellifera (apiMel3) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Bombus impatiens (GCA_000188095.2) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Caenorhabditis elegans (ce10) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Ceratitis capitata (1.1) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Daphnia pulex (GCA_900092285.1_ASM90009228v1) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Daphnia pulex (GCA_000187875.1) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Lutzomyia longipalpis (GCA_000265325.1) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Manduca sexta (GCA_000262585.1) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Nasonia vitripennis (2.1) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Oncopeltus fasciatus (GCA_000696205.2) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Paracoccus denitrificans (GCA_000203895.1) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Rhodnius prolixus (GCA_000181055.3) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Schistosoma mansoni (GCA_000237925.2) Notice: Undefined index: genomeSelect in /var/www/html/genome-select.inc on line 56 Tribolium castaneum (Tcas 4.0 draft)

Read the original:
CRISPR Optimal Target Finder

Albany NY, United States: CRISPR cas systems are commonly used in microbial engineering that includes immunization of cultures, bacterial strain typing, and self-targeted cell killing. Further, CRISPR and cas genes market system is also applied to control metabolic pathways for an improved biochemical synthesis. This technology is also used for the improvement of crop production. These factors further drive growth in the CRISPR and cas genes market.

CRISPR and cas genes system has been a revolutionary initiative in the biomedical research field. The application of this technology in somatic cell genome editing events has targeted to its application. The technologies are commonly used for the treatment of different genetic disorders. But, the ethical issues while using the system from the CRISPR and cas genes market are somewhere curtailing the growth in the industry. Furthermore, the market is also witnessing a lack of proficient professionals, which restrains its growth opportunities.

Request a PDF Sample https://www.transparencymarketresearch.com/sample/sample.php?flag=S&rep_id=26417

The market forecast on CRISPR and cas genes market was estimated US$ 1,451.6 Mn. Now it is predicted to climb US$ 7,234.5 Mn during forecast period from 2018 to 2026. The market is estimated to reach a compound annual growth rate (CAGR) of 20.1% from 2018 to 2026.

Multiple Applications and Diverse Dominating Factors in CRISPR and Cas Genes Market

The report from market research on CRISPR and cas genes industry has marked its division on the basis of region, end-user, application, and product type. DNA-free cas and vector-based cas are the two types in which the CRISPR and cas genes market is bifurcated on the basis of product type. Between these two types, the vector-based cas section has dominated the market at international levelin 2017. This expression system is helpful for the researchers who are focusing to enrich Cas9-expressing cells and concentrate on the establishment of a stable cell line. The vector-based cas is available with an analytical that is used to support the creation of durable cell lines. These lines are designed with minimal possible background expression.

Request Brochure of Report https://www.transparencymarketresearch.com/sample/sample.php?flag=B&rep_id=26417

The major advantages of the DNA-free cas segment boost growth in the CRISPR and cas genes market. DNA-free cas components are used for the reduction of potential off-targets. They also find application to trace correlations with human illnesses.

Knockout/activation, functional genomics, disease models, and genome engineering are the classification types in the CRISPR and cas genes market on the basis of application in different verticals. Contract research organizations, government and academic research institutes, pharmaceutical and biotechnology companies are some of the key end-use industries in the market. Further, as per the market analysis report on CRISPR and cas genes market, the industry is spread in different regions that include Middle East & Africa, Latin America, Asia Pacific, Europe, and North America.

Request for Analysis of COVID-19 Impact on CRISPR and Cas Genes Market https://www.transparencymarketresearch.com/sample/sample.php?flag=covid19&rep_id=26417

The industry players from market have adopted inorganic and organic growth strategies for the expansion of product offerings, capturing market share, increasing consumer base, and strengthening geographical reach. Some of the key players in the CRISPR and Cas genes market include Dharmacon, Synthego, GenScript, OriGene Technologies, Inc., Applied StemCell, Inc., Addgene, and Cellecta, Inc.

Genome Engineering to Dominate CRISPR and Cas genes market

On the basis of application, the genome engineering section has dominated in the CRISPR and cas genes market. The genetic materials can be added, detached, and altered with the help of CRISPR technology at any specific location in the genome. Genomic engineering is related to the synthetic assembly of comprehensive chromosomal DNA, and it has been commonly taken from natural genomic sequences.

Enquiry Before Buying https://www.transparencymarketresearch.com/sample/sample.php?flag=EB&rep_id=26417

The CRISPR and Cas genes market has been dominated by pharmaceutical and biotechnology companies in terms of end-user. The strategic partnerships and innovations may boost growth in the market.

North America and Europe are the regions that account for the maximum share in the CRISPR and Cas genes market. Rising technological advancements and research activities are driving growth in the market.

Browse more Reports by Transparency Market Research:

High-flow Nasal Cannula Market: The global high-flow nasal cannula market is expected to be driven by the technological progress made by the prominent market players. In addition to that, clinical trials for the purpose of evaluation of new areas of application are likely to open up new avenues of growth for the global high-flow nasal cannula market in the years to come.

Blood Group Typing Market: The global blood group typing market is prophesied to gain impetus from the increasing number of applications in various types of tests. As noted by experienced analysts, antibody screening could draw a greater growth in the market due to the intensifying demand for the early diagnosis of diseases and swelling prevalence of chronic diseases.

About Us

Transparency Market Research is a next-generation market intelligence provider, offering fact-based solutions to business leaders, consultants, and strategy professionals.

Our reports are single-point solutions for businesses to grow, evolve, and mature. Our real-time data collection methods along with ability to track more than one million high growth niche products are aligned with your aims. The detailed and proprietary statistical models used by our analysts offer insights for making right decision in the shortest span of time. For organizations that require specific but comprehensive information we offer customized solutions through ad hoc reports. These requests are delivered with the perfect combination of right sense of fact-oriented problem solving methodologies and leveraging existing data repositories.

TMR believes that unison of solutions for clients-specific problems with right methodology of research is the key to help enterprises reach right decision.

Contact

Rohit BhiseyTransparency Market ResearchState Tower,90 State Street,Suite 700,Albany NY - 12207United StatesUSA - Canada Toll Free: 866-552-3453Email: sales@transparencymarketresearch.comWebsite: https://www.transparencymarketresearch.com/

More here:
CRISPR and Cas Genes Market is Anticipated to Reach US$ 7,234.5 Mn by 2026, Increase in Incidence of Genetic Disorders to Drive the Market - BioSpace

Ive combed the internet to find you todays most fun/important/scary/fascinating stories about technology.

1 Elon Musk is buying Twitter for $44 billionHes unlikely to enjoy owning it as much as he thinks he will.(The Atlantic $)+ Heres a recap of the past fortnight: a rollercoaster ride from beginning to end. (FT $)+ Musk claims that open sourcing Twitters algorithm will make it more transparent. (NYT $)+ Trump says he wouldnt rejoin, but his advisers arent convinced. (WP $)+ Musk thinks people might be willing to pay for Twitter. Good luck with that. (Protocol)+ Its prompting people to finally face up to their Twitter addictions. (Input)

2 How China wrote the playbook for censoring covid informationIts inspired many other countries to impose restrictions in the name of public health. (New Yorker $)+ A new covid outbreak in Beijing is heightening fears of a Shanghai-style lockdown. (NYT $)+ Fences have been erected in Shanghai to prevent residents from leaving their homes. (BBC)

3 Instagram hackers stole $3 million of Bored Ape Yacht Club NFTsThey managed to compromise the official Instagram account then use it to post a phishing link. (Bloomberg $)+ Of course theres a social network for NFTs in the works. (Quartz)+ Chinese NFT platforms dont want you to trade NFTs anymore. (TR)

4 Conspiracy theories are nothing to do with evidenceAnd everything to do with how they make us feel. (Wired $)+ We are living in an age of misinformation. (Vox)

5 The US is fighting back against Chinas chip dominanceBy keeping its most advanced chipmaking tools to itself. (The Economist $)+ The great chip crisis threatens the promise of Moores Law. (TR)

6 The crypto industry is on a lawyer hiring spreeTo go mainstream, you need legal smarts. (WSJ $)+ Dogecoin, Elon Musks favorite cryptocurrency, is surging after his Twitter purchase. (Bloomberg $)+ A deep dive into the mysterious subcultures of cryptocurrency obsessives. (SCMP)+ Its okay to opt out of the crypto revolution. (TR)

7 The metaverse is a vile quagmire of racism, rape jokes and assaultAnd theres next to no recourse for offenders, either. (The Guardian)+ It doesnt help that were struggling to define what the metaverse even is. (Wired $)+ Facebook is opening its first physical store to try to sell its metaverse-related gadgets. (WP $)

8 Europes wind energy companies are being targeted by cyberattacksRenewable energy is an increasingly attractive target for criminals as attention turns to oil and gas alternatives. (WSJ $)

9 Coders want to relieve low-income families of the burden of adminApplying for benefits is needlessly complicated. (The Atlantic $)

10 Adapting video games for TV is so difficult to get rightBut makes for great watching when it works. (The Guardian)+ Nintendos animated Super Mario Bros. movie has been delayed. (The Verge)

Link:
The Download: Fertility for trans men, and a Q&A with CRISPR's co-creator - MIT Technology Review

BERKELEY, CAAdmitting he felt self-conscious following a workplace accident involving the highly experimental gene-editing technology, a local half-lobster scientist told reporters Friday he would just have to hope his coworkers didnt notice his CRISPR mishap. The main thing I need to do is make sure I dont slip up and try to shake someones hand, because that would be a dead giveaway, said University of California, Berkeley, microbiologist Steven Lipstadt, using his gigantic claws to put on a novelty sombrero he had purchased on his lunch break in order to hide the long antennae projecting outward from his head. Luckily my lab coat covers up quite a bit of my tail, so as long as I stay behind my desk, no one should notice that. Maybe people will see my protruding crimson rostrum and think I just have a cold or something? Ugh. If anyone notices I look a little different today, Ill tell them I got a haircut. I just hope nobody here has a shellfish allergy. At press time, Lipstadt was reportedly worried the nighttime cleaning staff would discover the half-human lobster he had been forced to smother with a couch cushion and stuff into a trash can.

Link:
Half-Lobster Scientist Just Going To Hope Coworkers Dont Notice He Had Mishap With CRISPR - The Onion

This article was originally published here

Exp Biol Med (Maywood). 2022 Apr 27:15353702221090181. doi: 10.1177/15353702221090181. Online ahead of print.

ABSTRACT

COVID-19 is an infectious disease caused by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) virus affecting the world population. Early detection has become one of the most successful strategies to alleviate the epidemic and pandemic of this contagious coronavirus. Surveillance testing programs have been initiated in many countries worldwide to prevent the outbreak of COVID-19. In this study, we demonstrated that our previously established clustered regularly interspaced short palindromic repeats (CRISPR)-Cas12a-based assay could detect variants of concern during 2021 in Thailand, including Alpha, Beta, and Delta strains as well as Omicron strain in early 2022. In combination with the newly designed saliva collection funnel, we established a safe, simple, economical, and efficient self-collection protocol for the COVID-19 screening process. We successfully utilized the assay in an active case finding with a total number of 578 asymptomatic participants to detect the SARS-CoV-2 in saliva samples. We finally demonstrated that the validation and evaluation in a large-scale setting could provide valuable information and elaborate the practicality of the test in real-world settings. Our optimized protocol yielded effective results with high sensitivity, specificity, and diagnostic accuracy (96.86%). In addition, this study demonstrates COVID-19 active case findings in low-resource settings, which would be feasible and attractive for surveillance and outbreak prevention in the future.

PMID:35473361 | DOI:10.1177/15353702221090181

Here is the original post:
COVID-19 active case findings based on self-collected saliva samples with CRISPR-Cas12a detection - DocWire News

(Ryan Downie)

Stocks with huge growth potential usually come with additional risk. Stock valuations usually reflect potential gains, but companies with fantastic upside potential usually face tons of uncertainty. That's not necessarily a bad thing, but we all need to make informed decisions when building our portfolios. Every investor needs to figure out their ideal balance of risk and reward and allocate accordingly.

These two stocks might be too risky for some investors, but people who take the plunge could reap major rewards down the road.

Image source: Getty Images.

1. Zscaler

Zscaler (NASDAQ: ZS) is a cybersecurity stock that provides cloud-based software for large businesses. It focuses on edge security, ensuring that user devices are able to connect with an employer's network without compromising the data on that network. This is extremely important for any company with a distributed workforce. As working from home and remote collaboration become more common, these security services have become absolutely essential for any enterprise that aims to stay relevant in today's economy.

People are also reading

This makes Zscaler an exciting opportunity because it's occupying the right place at the right time. The story gets even more compelling when you consider Zscaler's leadership position and highly respected product portfolio. The company receives outstanding marks from Gartner, and it's commonly viewed as one of the leaders of its cybersecurity niche. This translates to high customer satisfaction, and its high net dollar retention rate above 125% is clear evidence that businesses are seeing lots of value in Zscaler products.

This has all translated to great financial results for Zscaler. The company reported 63% revenue growth last quarter. Its full-year forecasts call for an expansion of roughly 55% in sales. By the end of the year, Zscaler's annualized recurring revenue should be above $1.4 billion. The company isn't profitable on an earnings basis yet, but it produces positive free cash flow. That means that it can support its high growth without exhausting its financial resources, which is great for investors.

It's easy to see where Zscaler stock gets its upside. The risk comes entirely from valuation. The stock's price-to-sales ratio is over 33, and its PEG ratio is above 4. This is a fairly high level, even among promising growth stocks -- and its valuation ratios used to be nearly twice as high.

Zscaler's stock has experienced some wild valuation swings since its IPO in 2019, so investors should expect continued volatility. The current valuations already assume a ton of growth and strong cash flows, so the company needs to deliver phenomenal financial results just to meet expectations. That requires investors to take a leap of faith. If there's any indication that it could fall short of its aggressive goals, the stock is nearly certain to suffer big losses, at least in the short term. Even if the company continues to perform well, Zscaler stock can still take a beating if the market is moving downward due to macroeconomic issues.

2. CRISPR Therapeutics

CRISPR Therapeutics (NASDAQ: CRSP) is a gene editing technology company that could drastically disrupt the pharmaceutical and biotech markets. It's developing a number of medications for various serious illnesses, including various forms of cancer, sickle-cell disease, diabetes, and degenerative diseases. This could greatly improve patient outcomes while making treatment more efficient and affordable.

Gene editing is an emerging industry with no clear leader in the market. CRISPR has a number of medication candidates, and it's also engaged in joint ventures with high-profile partners such as Vertex Pharmaceuticals. There's a chance for CRISPR to secure early-mover status in a disruptive industry that's expected to grow nearly 20% annually over the next decade.

CRISPR's market cap is currently around $4.5 billion. If its treatments wind up being safe, effective, and affordable, its product portfolio should be worth much more than $4.5 billion, and investors could enjoy huge returns.

Like many biotech stocks, this great opportunity comes with plenty of risk. CRISPR doesn't have any revenue from commercial sales of its products. There are major regulatory hurdles to clear before it sells a single treatment. It also has to solve manufacturing, logistical, and marketing challenges once it receives approval for medications. CRISPR also faces competition from other gene-editing companies, as well as traditional drugmakers. There's a good chance that the company will need to raise more cash by selling shares or issuing debt before it becomes stable, which could impact its financial stability.

CRISPR is an exciting healthcare stock for sure, but it is also a high-profile boom-or-bust investment. It could play a role as a growth stock for many investors, but make sure that your portfolio is capable of handling the risk before you dive in.

10 stocks we like better than Zscaler

When our award-winning analyst team has a stock tip, it can pay to listen. After all, the newsletter they have run for over a decade, Motley Fool Stock Advisor, has tripled the market.*

They just revealed what they believe are the ten best stocks for investors to buy right now... and Zscaler wasn't one of them! That's right -- they think these 10 stocks are even better buys.

*Stock Advisor returns as of April 7, 2022

Ryan Downie has no position in any of the stocks mentioned. The Motley Fool owns and recommends CRISPR Therapeutics, Vertex Pharmaceuticals, and Zscaler. The Motley Fool recommends Gartner. The Motley Fool has a disclosure policy.

Get the latest local business news delivered FREE to your inbox weekly.

Read more:
These 2 Stocks Carry a Lot of Risk, but Their Upside Is Huge - Lincoln Journal Star