Archive for the ‘Crispr’ Category

There is a new ongoing debate as to what plant breeding technologies constitute genetic modification. The transgenic GM crops introduced more than 20 years ago remain verboten for organic food production. If the pro-organic Cornucopia Institute and other organic food industry proponents have their way, all forms of gene editing and [New Breeding Techniques] would be classified as GM and join the list of practices prohibited for the production of food products eligible to be certified USDA Organic.

We strongly oppose any efforts to revisit the issue of any type of genetic engineering in organic certification, and we will work to ensure that all genetic engineering remains an excluded method, says Organic Farmers Association President David Colson. Any suggestion that we should explore gene-editing or any other type of genetic engineering, would distract from the core issues the organic market is facing right now.

On the other hand, some organic growers do see NBTs as a potential boon to their industry and are calling for revised rules that would allow growers to benefit from crop improvements created using gene editing. Klaas Martens, a prominent voice in the organic movement and a third-generation grain and livestock farmer, operates a 1,600-acre farm in New Yorks Finger Lakes region. He also owns a feed and seed business. Martens says he would be receptive to using CRISPR gene editing technology to grow versions of naturally occurring crops that restore soil health.

If it could be used in a way that enhances the natural system, and mimicked it, then I would want to use it, Martens says. But it would definitely have to be case by case.

The farmers who are opposed to an absolute ban of biotechnology for organic production underscore the belief held by many that USDA Certified Organic crops can help farming become more sustainable as a rising global population demands more food.

In my view, the use of genetic engineering technologies is the most powerful and honest organic tool we have, says Oliver Peoples, president and CEO of Yield10 Bioscience, an agricultural bioscience company focusing on the development of disruptive technologies to produce step-change improvements in crop yield for food and feed crops.

Read the original post

Read this article:
Some organic farming advocates poised to embrace CRISPR and other New Breeding Techniques because of their sustainability benefits - Genetic Literacy...

The research study on Modest recovery in Global CRISPR Technology Market is inclusive of a detailed summary of this industry. A highly focused approach to subjective research has been undertaken, with the description of product scope and elaborate industry insights and outlook until 2025. Introduced by Research Reports Inc., this report delivers information about the product pertaining to the parameters of cost, demand and supply graph, market trends, and the nature of the transaction.

Also, the report is liable to help shareholders and prominent investors understand the demands of customers for efficiently marketing the products and services.

A detailed analysis of the CRISPR Technology market has been provided in the report. The analysis is undertaken on the basis of the overall historical data, valid projections on the market size, qualitative insights, and more. The predictions of this report have been inferred based on conclusive analysis techniques and assumptions. In essence, this research report works like a repository of analysis as well as information for all the aspects of the industry including and not limited to:

A detailed evaluation of the popular trends prevalent in the CRISPR Technology market has been given in the report, in tandem with the microeconmic pointers and regulatory mandates. With this analysis, the report projects the lucrativeness of every market segment over the forecast period, 2020-2025.

Important factors analyzed in worldwide CRISPR Technology market report

Revenue and Sales Estimation: Historical remuneration, as well as sales volume, have been specified in the report this helps in preparing an accurate budget. The data is segmented with the help of bottom-up and top-down approaches to predict the overall market share as well as to calculate forecast numbers for the major geographies in the report in tandem with the key Types and Applications.

Manufacturing Analysis: The report is presently evaluated in terms of the numerous product types and applications. The global CRISPR Technology market study delivers essential highlights of the manufacturing process analysis that has been verified through primaries. These primaries have been collected via industry professionals and also major representatives of all the firms profiled in the report, in order to prepare courses of action to support the industry growth effectively.

Competition: Major contenders have been studied on the basis of their company profile, product/service price, sales, capacity, product portfolio, and cost to find out the present competitors strengths as well as weaknesses.

Demand & Supply and Effectiveness: CRISPR Technology report also delivers information about the production, distribution, consumption & export/import, and break-even point & marginal revenue). ** If applicable

Ask For Customized Report as per Your Business Requirement

Thermo Fisher Scientific, Merck KGaA, GenScript, Integrated DNA Technologies (IDT), Horizon Discovery Group, Agilent Technologies, Cellecta, GeneCopoeia, New England Biolabs, Origene Technologies, Synthego Corporation, Toolgen

Graphically, this report is split into numerous regions, with details on production, consumption, supply, and demand, growth rate, and market share of CRISPR Technology Market in these regions, between 2020 to 2025 (forecast), covering:- North America, Europe, Asia Pacific, Latin America, and Middle East & Africa

Brief introduction about CRISPR Technology Market:

Chapter 1. Global CRISPR TechnologyMarket Size (Sales) Market Share by Type (Product Category) [1,2,3,] in 2020

Chapter 2. CRISPR TechnologyMarket by Application/End Users [1,2,3]

Chapter 3. Global CRISPR TechnologySales (Volume) and Market Share Comparison by Applications

Chapter 4. Global CRISPR TechnologySales and Growth Rate (2020-2025)

Chapter 5. CRISPR TechnologyMarket Competition by Players/Suppliers, Region, Type, and Application

Chapter 6. CRISPR Technology(Volume, Value and Sales Price) structure specified for each geographic region included.

Chapter 7. Global CRISPR TechnologyPlayers/Suppliers Profiles and Sales Data

Chapter 8. Company primary Information and Top Competitors list are being provided for each vendor listed in the report.

Chapter 9. Market Sales, Revenue, Price and Gross Margin (2020-2025) table for each product type which includes Cost Structure Analysis, Key Raw Materials Analysis & Price Trends

Chapter 10. Supply Chain, Sourcing approach and Downstream Buyers, Industrialized Chain Analysis

Directly Buy This Report

Closure: A detailed point-by-point analysis, that contains information on the estimation of the parent market-relevant diversity in market segmentation and market dynamics until the second or third level. Historical, present, and projected market scope from the perspective of cost and capacity. The report also provides details on the reporting as well as interpretation of the latest industry progress, in tandem with market shares and strategies of major players, emerging niche segments as well as regional markets. An objective analysis of the growth curve of the market has been provided, that would guide stakeholders to increase their foothold in the market.

About Research Reports Inc:

Research Reports Inc. is one of the leading destinations for market research reports across all industries, companies, and technologies. Our repository features an exhaustive list of market research reports from thousands of publishers worldwide. We take pride in curating a database covering virtually every market category and an even more comprehensive collection of market research reports under these categories and sub-categories. We are one of the premier sources for such reports & report customization services.

Contact Us:

David ( Sales Manager )

US: +1-855-419-2424

UK : +440330807757

Email: ([emailprotected])

View original post here:
CRISPR Technology: Global Industry Share, Size, Trends, Growth, Investment Analysis, Development Factors, Future Scope, Challenges and Forecast to...

CAMBRIDGE, Mass., Feb. 20, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, will present fourth quarter and full-year 2019 financial results and operational highlights in a conference call on February 27, 2020 at 8 a.m. ET.

To join the call:

U.S. callers should dial 1-877-317-6789 and use conference ID# 10138773, approximately five minutes before the call.

International callers should dial 1-412-317-6789 and use conference ID# 10138773, approximately five minutes before the call.

A replay of the call will be available through the Events and Presentations page of the Investor Relations section of the companys website at http://www.intelliatx.com, beginning on February 27, 2020 at 12 p.m. ET.

About Intellia Therapeutics

Intellia Therapeutics is a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com and follow us on Twitter @intelliatweets.

Intellia Contacts:

Investor Contact: Lina LiAssociate Director, Investor Relations+1 857-706-1612lina.li@intelliatx.com

Story continues

Media Contact:Jennifer Mound SmoterSenior Vice President, External Affairs & Communications+1 857-706-1071jenn.smoter@intelliatx.com

Visit link:
Intellia Therapeutics to Hold Conference Call to Discuss Fourth Quarter and Full-Year 2019 Earnings and Company Update - Yahoo Finance

MORGAN STANLEY ASIA LTD. bought a fresh place in Mastercard Incorporated (NYSE:MA). The institutional investor bought 2.3 million shares of the stock in a transaction took place on 12/31/2019. In another most recent transaction, which held on 12/31/2019, SWEDBANK ROBUR FONDER AB bought approximately 1.6 million shares of Mastercard Incorporated. In a separate transaction which took place on 12/31/2019, the institutional investor, GOLDMAN SACHS & CO. LLC (PRIVATE bought 1.3 million shares of the companys stock. The total Institutional investors and hedge funds own 77.30% of the companys stock.

In the most recent purchasing and selling session, Mastercard Incorporated (MA)s share price increased by 0.97 percent to ratify at $344.56. A sum of 3010854 shares traded at recent session and its average exchanging volume remained at 3.31M shares. The 52-week price high and low points are important variables to concentrate on when assessing the current and prospective worth of a stock. Mastercard Incorporated (MA) shares are taking a pay cut of 0.43% from the high point of 52 weeks and flying high of 59.57% from the low figure of 52 weeks.

Mastercard Incorporated (MA) shares reached a high of $347.24 and dropped to a low of $342.6349 until finishing in the latest session at $343.99. Traders and investors may also choose to study the ATR or Average True Range when concentrating on technical inventory assessment. Currently at 5.68 is the 14-day ATR for Mastercard Incorporated (MA). The highest level of 52-weeks price has $343.10 and $215.93 for 52 weeks lowest level. After the recent changes in the price, the firm captured the enterprise value of $344.4B, with the price to earnings ratio of 43.37 and price to earnings growth ratio of 2.44. The liquidity ratios which the firm has won as a quick ratio of 1.40, a current ratio of 1.40 and a debt-to-equity ratio of 1.56.

Having a look at past record, were going to look at various forwards or backwards shifting developments regarding MA. The firms shares rose 4.12 percent in the past five business days and grew 6.46 percent in the past thirty business days. In the previous quarter, the stock rose 22.72 percent at some point. The output of the stock increased 23.91 percent within the six-month closing period, while general annual output gained 56.20 percent. The companys performance is now positive at 15.40% from the beginning of the calendar year.

According to WSJ, Mastercard Incorporated (MA) obtained an estimated Buy proposal from the 36 brokerage firms currently keeping a deep eye on the stock performance as compares to its rivals. 0 equity research analysts rated the shares with a selling strategy, 2 gave a hold approach, 29 gave a purchase tip, 4 gave the firm a overweight advice and 1 put the stock under the underweight category. The average price goal of one year between several banks and credit unions that last year discussed the stock is $359.44.

CRISPR Therapeutics AG (CRSP) shares on Wednesdays trading session, jumped 4.35 percent to see the stock exchange hands at $58.11 per unit. Lets a quick look at companys past reported and future predictions of growth using the EPS Growth. EPS growth is a percentage change in standardized earnings per share over the trailing-twelve-month period to the current year-end. The company posted a value of $0.97 as earning-per-share over the last full year, while a chance, will post -$5.03 for the coming year. The current EPS Growth rate for the company during the year is 134.10% and predicted to reach at -13.40% for the coming year. In-depth, if we analyze for the long-term EPS Growth, the out-come was 54.00% for the past five years.

The last trading period has seen CRISPR Therapeutics AG (CRSP) move -21.47% and 88.98% from the stocks 52-week high and 52-week low prices respectively. The daily trading volume for CRISPR Therapeutics AG (NASDAQ:CRSP) over the last session is 1.35 million shares. CRSP has attracted considerable attention from traders and investors, a scenario that has seen its volume jump 4.33% compared to the previous one.

Investors focus on the profitability proportions of the company that how the company performs at profitability side. Return on equity ratio or ROE is a significant indicator for prospective investors as they would like to see just how effectively a business is using their cash to produce net earnings. As a return on equity, CRISPR Therapeutics AG (NASDAQ:CRSP) produces 11.70%. Because it would be easy and highly flexible, ROI measurement is among the most popular investment ratios. Executives could use it to evaluate the levels of performance on acquisitions of capital equipment whereas investors can determine that how the stock investment is better. The ROI entry for CRSPs scenario is at 4.90%. Another main metric of a profitability ratio is the return on assets ratio or ROA that analyses how effectively a business can handle its assets to generate earnings over a duration of time. CRISPR Therapeutics AG (CRSP) generated 9.60% ROA for the trading twelve-month.

Volatility is just a proportion of the anticipated day by day value extendthe range where an informal investor works. Greater instability implies more noteworthy benefit or misfortune. After an ongoing check, CRISPR Therapeutics AG (CRSP) stock is found to be 5.65% volatile for the week, while 4.66% volatility is recorded for the month. The outstanding shares have been calculated 63.38M. Based on a recent bid, its distance from 20 days simple moving average is 5.27%, and its distance from 50 days simple moving average is -3.80% while it has a distance of 16.08% from the 200 days simple moving average.

The Williams Percent Range or Williams %R is a well-known specialized pointer made by Larry Williams to help recognize overbought and oversold circumstances. CRISPR Therapeutics AG (NASDAQ:CRSP)s Williams Percent Range or Williams %R at the time of writing to be seated at 32.64% for 9-Day. It is also calculated for different time spans. Currently for this organization, Williams %R is stood at 28.28% for 14-Day, 28.28% for 20-Day, 66.19% for 50-Day and to be seated 41.23% for 100-Day. Relative Strength Index, or RSI(14), which is a technical analysis gauge, also used to measure momentum on a scale of zero to 100 for overbought and oversold. In the case of CRISPR Therapeutics AG, the RSI reading has hit 52.87 for 14-Day.

See the rest here:
Stocks to Follow: Mastercard Incorporated (MA) and CRISPR Therapeutics AG (CRSP) - BOV News

A new Profession Intelligence Report released by Stats and Reports with the title Global Gene Editing Tools Market can grow into the most important market in the world that has played an important role in making progressive impacts on the global economy. Global Gene Editing Tools Market Report presents a dynamic vision to conclude and research market size, market hope and competitive environment. The study is derived from primary and secondary statistical data and consists of qualitative and numerical analysis. The main company in this survey is Thermofisher Scientific, CRISPR Therapeutics, Editas Medicine, NHGRI, Intellia Therapeutics, Merck KGaA, Horizon.

Gene editing or Genome editingis a way of making specific changes to the DNA of a cell or organism. An enzyme cuts the DNA at a specific sequence, and when this is repaired by the cell a change or edit is made to the sequence.

Free Sample Report @:www.statsandreports.com/request-sample/340551-global-gene-editing-tools-market-size-status-and-forecast-2019-2025

This report clearly shows that the Gene Editing Tools industry has achieved significant growth since 2018. It is based on an in-depth assessment of the industry. The analysis provided in this report shows the leading segments to gain a strong presence in the industry and the insights that help determine new strategies. In conclusion, analysts who value unbiased information about stakeholders, investors, product managers, marketing executives, and supply, demand, and future predictions value the report.

Preliminary Data:Get raw market data and contrast from wide front. Data is constantly filtered so that only validated and authenticated sources are considered. The data is also collected from many reputable paid databases and many reports in our repository. A comprehensive understanding of the market is essential to understanding and facilitating the complete value chain. We collect data from raw material suppliers, distributors, and buyers.

Furthermore, the years considered for the study are as follows:Historical year 2014-2018Base year 2019Forecast period** 2019 to 2025[** unless otherwise stated]

Research Methodology:The market engineering process uses a top-down and bottom-up approach and several data triangulation methods to evaluate and validate the size of the entire market and other dependent sub-markets listed in this report. Numerous qualitative and quantitative analyzes have been conducted in the market engineering process to list key information / insights. The major players in the market were identified through the second survey and the market rankings were determined through the first and second surveys.

Exclusive Discount Offer on Quick Purchase@www.statsandreports.com/check-discount/340551-global-gene-editing-tools-market-size-status-and-forecast-2019-2025

Crucial Research:During the first survey, we interviewed various key sources of supply and demand to obtain qualitative and quantitative information related to this report. Key supply sources include key industry participants, subject matter specialists from key companies, and consultants from several major companies and organizations active in the digital signage market.

Minor Research:The second study was conducted to obtain key information on the supply chain of the industry, the markets currency chain, pools of major companies, and market segmentation, with the lowest level, geographical market, and technology-oriented perspectives. Secondary data was collected and analyzed to reach the total market size, which was verified by the first survey.

This research many focuses on future market segments or regions or countries to channel efforts and investments to maximize growth and profitability. The report presents an in-depth analysis of key vendors or key players in the market competitive landscape and market.The research provides answers to the following key questions:

What are the Major applications of the Gene Editing Tools Market?Applications cover in these Reports Is:Sickle Cell Disease, Heart Disease, Diabetes, Alzheimers Disease, Obesity and Others

what are the Types of the Gene Editing Tools Market?Types Cover in this Research :Zinc finger nucleases (ZFNs), Transcription Activator-Like Effector-based Nucleases (TALENs), CRISPR-Cas system

Who are the main competitors in the market and what are their priorities, strategies, and developments?Lists of Competitors in Research Is:Thermofisher Scientific, CRISPR Therapeutics, Editas Medicine, NHGRI, Intellia Therapeutics, Merck KGaA, Horizo n

Read Full TOC of Gene Editing Tools Research Study at @www.statsandreports.com/report/340551-global-gene-editing-tools-market-size-status-and-forecast-2019-2025

All percent shares, breaks, and classifications were determined using the secondary sources and confirmed through the primary sources. All parameters that may affect the market covered in this study have been extensively reviewed, researched through basic investigations, and analyzed to obtain final quantitative and qualitative data. This has been the study of key quantitative and qualitative insights through interviews with industry experts, including CEOs, vice presidents, directors and marketing executives, as well as annual and financial reports from top market participants.

Years considered for the study are:Historical year 2014-2018Disreputable year 2019Estimate period** 2019 to 2025 [** unless otherwise stated]

Essentials of Table of Content:

1 Report Overview1.1 Research Scope1.2 Key Market Segments1.3 Target Player1.4 Market Analysis by Type1.5 Market by Application1.6 Learning Objectives1.7 years considered

Buy the Up-to-date Full Report @www.statsandreports.com/placeorder?report=340551-global-gene-editing-tools-market-size-status-and-forecast-2019-2025

2 Global Growth Trends2.1 Global Gene Editing Tools Market Size2.2 Trends of Gene Editing Tools Growth by Region2.3 Corporate trends

3 Gene Editing Tools Market shares by key players3.1 Global Gene Editing Tools Market Size by Manufacturer3.2 Global Gene Editing Tools Key players Provide headquarters and local3.3 Major Players Products / Solutions / Services3.4 Enter the Barriers in the Gene Editing Tools Market3.5 Mergers, acquisitions and expansion plans

4 Market By-products4.1 Global Gene Editing Tools Sales by Product4.2 Global Gene Editing Tools by Product Revenue4.3 Global Gene Editing Tools

Note: Regional Breakdown & Sectional purchase Available We provide Pie chats Best Customize Reports As per Requirements.

About Us

Stats and Reports is a global market research and consulting service provider specialized in offering wide range of business solutions to their clients including market research reports, primary and secondary research, demand forecasting services, focus group analysis and other services. We understand that how data is important in todays competitive environment and thus, we have collaborated with industrys leading research providers who works continuously to meet the ever-growing demand for market research reports throughout the year.

Contact:Stats and ReportsSatish K. (Global Sales Manager)Mangalam Chamber, Office No 16, Paud RoadSankalp Society, Kothrud, Pune, Maharashtra 411038Phone: +1 650-646-3808Email: [emailprotected]Web: https://www.statsandreports.comFollow Us on: LinkedIN| Twitter|

See the rest here:
Gene Editing Tools Market- increasing demand with Industry Professionals: Thermofisher Scientific, CRISPR Therapeutics - Instant Tech News

From the University of California, Berkeley:

The yellow monkeyflower's distinctive red spots serve as 'landing pads' for bees and other pollinators, helping them access the sweet nectar inside. A new study reveals the genetic programming that creates these attractive patterns.

The intricate spotted patterns dappling the bright blooms of the monkeyflower plant may be a delight to humans, but they also serve a key function for the plant. These patterns act as "bee landing pads," attracting nearby pollinators to the flower and signaling the best approach to access the sweet nectar inside.

"They are like runway landing lights, helping the bees orient so they come in right side up instead of upside down," said Benjamin Blackman, assistant professor of plant and molecular biology at the University of California, Berkeley.

In a new paper, Blackman and his group at UC Berkeley, in collaboration with Yaowu Yuan and his group at the University of Connecticut, reveal for the first time the genetic programming that helps the monkeyflower and likely other patterned flowers achieve their spotted glory. The study was published online today (Thursday, Feb. 20) in the journal Current Biology.

"While we know a good deal about how hue is specified in flower petals whether it is red or orange or blue, for instance we don't know a lot about how those pigments are then painted into patterns on petals during development to give rise to these spots and stripes that are often critical for interacting with pollinators," Blackman said. "Our lab, in collaboration with others, has developed the genetic tools to be able to identify the genes related to these patterns and perturb them so that we can confirm what's actually going on."

In the study, the research team used CRISPR-Cas9 gene editing to recreate the yellow monkeyflower patterns found in nature. On the left, a wild-type monkeyflower exhibits the typical spotted pattern. In the middle, a heterozygote with one normal RTO gene and one damaged RTO gene exhibits blotchier spots. And on the right, homozygote with two copies of the damaged RTO gene is all red, with no spots.

The positions of petals' spots aren't mapped out ahead of time, like submarines in a game of battleship, Blackman said. Instead, scientists have long theorized that they could come about through the workings of an activator-repressor system, following what is known as a reaction-diffusion model, in which an activator molecule stimulates a cell to produce the red-colored pigment that produces a spot. At the same time, a repressor molecule is expressed and sent to neighboring cells to instruct them not to produce the red pigment.

The results are small, dispersed bunches of red cells surrounded by cells that keep the background yellow color.

"By tweaking the parameters how strongly a cell turns on an inhibitor, how strongly the inhibitor can inhibit the activator, how quickly it moves between cells it can lead to big spots, small spots, striped patterns, really interesting periodic patterns," Blackman said.

In the study, UC Berkeley postdoctoral researcher Srinidhi Holalu and research associate Erin Patterson identified two natural varieties of the yellow monkeyflower one type with the typical red spots in the throat of the flower and a second type with an all-red throat appearing in multiple natural populations in California and Oregon, including at the UC Davis McLaughlin Reserve. In parallel, UConn postdoctoral researcher Baoqing Ding worked with a very similar plant with fully red-throated flowers found when surveying a population of Lewis's monkeyflower that had induced DNA mutations.

Monkeyflower plants with the RTO gene knocked out by CRISPR-Cas9 gene editing produce one big patch where all flowers exhibit a fully red throat, in contrast to wild fields where red-tongued flowers appear in small dispersed spots.

In a previous study, the Yuan lab had found that a gene called NEGAN (nectar guide anthocyanin) acts as an activator in the monkeyflower petals, signaling the cells to produce the red pigment. Through detailed genomic analysis in both monkeyflower species, the two groups were able to pinpoint that a gene called RTO, short for red tongue, acts as the inhibitor.

The red-throated forms of the monkeyflower have defective RTO inhibitor genes, resulting in a characteristic all-red throat, rather than red spots. To confirm their findings, Holalu used the CRISPR-Cas9 gene editing system to knock out the RTO gene in spotted variants of the flower. The result was flowers with a flashy red throat. Further experiments revealed how the functional form of the RTO protein moves to neighboring cells and represses NEGAN to prevent the spread of pigmentation beyond the local spots. This study is the first reported use of CRISPR-Cas9 editing to research the biology of monkeyflowers.

The team also collaborated with Michael Blinov at the UConn School of Medicine to develop a mathematical model to explain how different self-organized patterns might arise from this genetic system.

"This work is the simplest demonstration of the reaction-diffusion theory of how patterns arise in biological systems," said Yaowu Yuan, associate professor of ecology and evolutionary biology at UConn. "We are closer to understanding how these patterns arise throughout nature."

This press release was produced by the University of California, Berkeley. The views expressed here are the author's own.

Link:
How The Monkeyflower Gets Its Spots | Berkeley - Patch.com

If youve never heard of Crispr then get ready, because this is a technology youre likely to be hearing a lot more about in future. From genetically modified Chinese babies to foods that deliver man-made health benefits, the potential of Crispr is enormous but its implications have some deep thinkers concerned.

Announced in 2012, Crispr stand for Clustered Regularly Interspaced Short Palindromic Repeats a scientific name that doesnt do much to tell the average person what it does. However the technology represents an astonishing breakthrough, allowing scientists to essentially edit genes and change aspects of DNA in ways previously thought impossible.

Late last year, gene editing was voted to be the innovation of the last decade by readers of The Irish Times, beating out social media, the cloud and even smartphones.

So just what is it and how can something so low-key be so significant?

Crispr (pronounced crisper) allows users to edit genomes and alter DNA sequences to modify gene function. It can be used to correct genetic defects, treating and preventing the spread of diseases and improving crops. While gene editing was possible before Crispr, it cost an enormous amount of money and was relatively imprecise.

The comparison has been made that old-style gene editing could be likened to a blunt mallet while Crispr is more like a laser beam, capable of surgical accuracy at the level of DNA. The science behind this is predictably complicated, but, in essence, Crispr allows scientists to find a specific bit of DNA inside a cell and then alter that piece of DNA.

It can be used to turn genes on or off without altering their sequence and it means that scientists can alter the DNA of plants, animals and potentially human beings to do any number of things. The most significant include altering the DNA of living people to turn off genes that have resulted in them suffering from genetic disorders, or making changes to an individual persons genome so that they dont pass on any genetic defect.

In Ireland in 2018, scientists from Trinity College discovered a therapy for one of the most common soft tissue cancers using Crispr. Synovial sarcoma affects teenagers and young adults and has survival rates of less than 50 per cent.

Pre-clinical trials in mice have showed that drugs developed using information gained through Crispr were able to target cancerous cells, crucially leaving normal cells alone.

Crispr is a two or three component system that allows you to use specific targeting RNA molecules to direct an enzyme that will cut and edit DNA and in theory you can change the DNA any which way you might want to, said Dr Gerard Brien, senior research fellow researching childhood cancers in the genetics department at Trinity College.

In theory, if a disease is caused by a specific mutation, then you could fix that mutation, he said.

But its still early days. In theory, all sorts of things are possible but the practicalities of how to do these things in a way that is effective and safe were not anywhere near a point of understanding how to do that.

The problem, according to Dr Brien, is that of unwanted off target effects.

Were not yet at the point where we can make one clear and specific change and not unintentionally create other changes at the same time. Its these other unwanted changes that are the problem and that will be the stumbling block in using Crispr ethically in humans for the next twenty years, if not more, he said.

In 2020 though, Crispr has applications outside of medicine. Its already being used to alter the DNA of plants and animals to create new kinds of super-foods, genetically modified (GM) to be healthier and offer advantages to the consumer.

To date, some GM foods have been treated with suspicion by the public because their modifications have mostly been for the benefit of the farmers or retailers tomatoes that last longer on the shelf, for example but instead of this, think of coeliac-friendly GM wheat, rapeseed oil high in beneficial omega-3s and even GM potatoes that dont produce harmful cancer-causing acrylamides when fried.

Crispr is a complicated beast, however.

The problem with making changes to the DNA of a plant, animal or person is that its often quite hard to predict the full range of consequences. Tweak something here, and something over there can be affected without you realising.

Its for this reason that experiments on human beings are considered hugely unethical. But that hasnt stopped everyone from tinkering with the technology. In late 2019 Chinese biophysicist He Jiankui was jailed for three years and fined 3 million yuan (about 393,000) when he was convicted of violating a government ban on experimenting on human embryos.

He claimed to have edited the genes of a set of human twins, known by the pseudonyms Lulu and Nana, to give them protection against HIV, but was globally condemned when news of his actions broke. The Chinese court accused the man of having essentially gone on a glory run, saying the people involved in the experiment had acted in the pursuit of personal fame and gain. Theyve crossed the bottom line of ethics in scientific research and medical ethics.

The consequences of Hes actions are still unknown, but the effects will be permanent. If the twins he experimented on grow up and have children of their own, they will inherit his genetic modifications and potentially introduce a permanent change to the human genome.

One of the issues with the Chinese-born twins is that the germline was edited so they will pass their edits on to future generations and we dont know what the long-term effects of that will be, it could be that these changes to their genes could initiate cancer down the line. We just dont know, said Dr Oliver Feeney, a bioethicist and lecturer in University College Cork.

There is this thing called the precautionary principle which states that you shouldnt do something unless you have all the down-side risks guarded against and you know exactly whats going to happen. The problem is we dont do that in any other context. We generally just bulldoze through life and see what happens.

The question here is should we shape humans and the way they develop in future? Is that ethical? Most people would agree that Crispr-enabled treatments for diseases would be a good thing if theyre effective and safe, but what about enhancements? Were only at the start of the regulatory landscape that will be required to manage this.

Dr Feeney suggests that the concept of gene editing is inherently scary to some people and that can colour the way in which it is viewed.

There is a possibility that we could be too concerned about Crispr and any issues that might arise. Thats not to minimise the risks or anything, but there is a certain level of hype around this technology and perhaps also excessive fear about what it can do, he said.

Some people have gone as far as to suggest that engaging in gene editing opens the door to eugenics and a world of genetic haves and have-nots. Its ironic to consider that this is the same scenario presented in the movie Gattaca and here we are having the conversation for real 20 years later.

Read the original post:
Crispr gene-editing technology: what is it and why you need to know about it - The Irish Times

Do you want it locally grown, water-saving and pesticide-free? Urban agriculture might suit you, with a little help from gene editing. Zachary Lippmans team has already succeeded with Solanaceae fruit crops, optimizing tomatoes and ground-cherries for indoor production (see their paper in Nature Biotechnology).

By targeting three genes (SlER, SPG5, and SP), they made the plants display compact growth habit and early yield. The tomatoes produced were slightly smaller than the wild type, but each plant bore more fruit, and they tasted good.

Commenting the paper in the news and views section, Cathryn O Sullivan and colleagues foresee a whole CRISPR menu coming from urban agriculture in the future. It is unlikely that wheat or rice will ever be grown indoors, but urban farms will be interested in producing any plant that has high value and is eaten fresh.

First of all fruits and vegetables that grow on bushes or vines, such as tomato, strawberry, raspberry, blueberry, cucumber, capsicum, grapes, kiwifruit. Specialist crops such as hops, vanilla, saffron, coffee, and also medicinal or cosmetic crops may come next.

They think that one day even small trees (chocolate, mango, almonds) may be grown indoors. However, for indoor farming to be broadly adopted, the capital and operating costs of climate-controlled farms must be reduced, or they will benefit only the wealthiest communities.

Read the original article

Go here to read the rest:
CRISPR technology opens door to vertical farming of dozens of crops, from strawberries and cucumbers to mango and almond trees - Genetic Literacy...

DetailsCategory: More NewsPublished on Thursday, 20 February 2020 13:06Hits: 157

BERKELEY, CA & RICHMOND, CA, USA I February 19, 2020 I Caribou Biosciences, Inc., a leading CRISPR genome editing company, and ProMab Biotechnologies, Inc., a biotechnology CRO/CDMO specializing in antibody engineering and CAR-T development, today announced a sale and assignment agreement under which Caribou gains access to a ProMab humanized single-chain variable fragment (scFv) targeting the B Cell Maturation Antigen (BCMA) for use in allogeneic engineered cell therapies. Caribou intends to utilize this scFv in the development of its CB-011 program, an allogeneic CAR-T therapy targeting BCMA-positive tumors including multiple myeloma.

We are excited for the opportunity to have access to this highly advanced, humanized molecule and believe it will significantly advance our promising CB-011 CAR-T program, said Steven Kanner, PhD, Chief Scientific Officer of Caribou.

We anticipate that our humanized BCMA scFv will aid greatly in Caribous efforts to further its allogeneic CAR-T program, and hope our technology continues to improve the field of preclinical and clinical stage immunotherapy research by providing broad choices of validated antibodies, said John Wu, MD, Chief Executive Officer of ProMab.

Under the terms of the agreement, ProMab received an upfront payment and is eligible for royalties on net sales of licensed products containing the BCMA scFv.

About Caribou Biosciences, Inc. Caribou is a leading company in CRISPR genome editing founded by pioneers of CRISPR-Cas9 biology. The company is developing an internal pipeline of off-the-shelf CAR-T cell therapies, other gene-edited cell therapies, and engineered gut microbes. Additionally, Caribou offers licenses to its CRISPR-Cas9 foundational IP in multiple fields including research tools, internal research use, diagnostics, and industrial biotechnology. Interested companies may contact Caribou at This email address is being protected from spambots. You need JavaScript enabled to view it.. For more information about Caribou, visit http://www.cariboubio.com and follow the Company @CaribouBio. Caribou Biosciences and the Caribou logo are registered trademarks of Caribou Biosciences, Inc.

About ProMab Biotechnologies, Inc. ProMab Biotechnologies focuses on developing and commercializing mouse, rabbit, and human monoclonal antibodies as well as chimeric antigen receptor-T Cell (CAR-T) products. ProMabs CAR-T platform covers both hematological and solid cancers with intensive in vitro and in vivo pre-clinical validation designed for safer and better treatment. As a CRO in the immunology field for 19 years, ProMab offers standard laboratory procedures and animal studies for antibody discovery through the integration of the newest techniques in antibody library construction, next generation sequencing, unique humanization modeling, high-throughput screening, and artificial intelligence analysis systems. ProMab aims to out-license antibodies validated in CAR-T therapy in the preclinical stage or to bring CAR-T technologies to the early stage market of clinical study. ProMab has partnered with top biotechnology startups, medical institutions, and pharmaceutical companies to advance the development of cell therapies as well as bispecific antibodies targeting multiple cancers. For more information, visit http://www.promab.com.

SOURCE: Caribou Biosciences

Read the original:
Caribou Biosciences and ProMab Biotechnologies Announce Sale and Assignment Agreement for Humanized scFv Targeting BCMA | More News | News Channels -...

Though common, these big phages would have been completely missed by traditional lab techniques. It used to be that scientists could only discover viruses by first growing themand they often filtered out anything above a certain size. In science, you tend to find what you look for. The huge phages dont fit the standard conception of what a virus should be, so no one went looking for them. But Banfield used a different method, which she pioneered in the 1990s: Her team took environmental samplesscoops of soil or drops of waterand simply analyzed all the DNA within to see what popped out. And once Banfield realized that the huge phages existed, it wasnt hard to find more.

Read: Beware the Medusavirus

Her team, including researchers Basem Al-Shayeb and Rohan Sachdeva, identified huge phages in French lakes, in Tibetan springs, and on the Japanese seafloor. They found the viruses in geysers in Utah, salt from Chiles Atacama Desert, stomach samples from Alaskan moose, a neonatal intensive-care unit in Pittsburgh, and spit samples from Californian women. All of these phages have at least 200,000 DNA letters in their genome, and the largest of them has 735,000.

The team included researchers from nine countries, and so named the new viruses using words for huge in their respective languages. Hence: Mahaphage (Sanskrit), Kaempephage (Danish), Kyodaiphage (Japanese), and Jabbarphage (Arabic), but also Whopperphage (American English).

These huge phages have other strange characteristics. With so much DNA, the viruses are probably physically bigger than typical phages, which means that they likely reproduce in unusual ways. When phages infect bacteria, they normally make hundreds of copies of themselves before exploding outwards. But Banfield says that an average bacterium doesnt have enough room to host hundreds of huge phages. The giant viruses can probably only make a few copies of themselves at a timea strategy more akin to that of humans or elephants, which only raise a few young at a time, than to the reproduction of rodents or most insects, which produce large numbers of offspring.

Giant phages also seem to exert more control over their bacterial hosts than a typical virus. All viruses co-opt their hosts resources to build more copies of themselves, but the huge phages seem to carry out a much more thorough and directed takeover, Banfield says. Their target is the ribosomea manufacturing plant found in all living cells, which reads the information encoded in genes and uses that to build proteins. The huge phages seem equipped to fully commandeer the ribosome so that it ignores the hosts genes, and instead devotes itself to building viral proteins.

This takeover involves an unorthodox use of CRISPR. Long before humans discovered CRISPR and used it to edit DNA, bacteria invented it as a way of defending themselves against viruses. The bacteria store genetic snippets of phages that have previously attacked them, and use these to send destructive scissorlike enzymes after new waves of assailants. But Banfields team found that some huge phages have their own versions of CRISPR, which they use in two ways. First, they direct their own scissors at bacterial genes, which partly explains why they can so thoroughly take over the ribosomes of their hosts. Second, they seem to redirect the bacterial scissors into attacking other phages. They actually boost their hosts immune system to take out the competition.

See the rest here:
A Huge Discovery in the World of Viruses - The Atlantic

Given that just two genes are responsible for making cats a problem for many people, it seemed like a no-brainer to engineer cats that lacked those genes, or to simply breed cats with versions of the genes that made the animals less allergenic.

But so far, itchy-eyed cat lovers have been left disappointed.

But for all those who havent given up hope, there may be new options around the corner. An allergic owner might pop open a can of allergy-fighting food for the cat. Or maybe vaccinate the cat to produce fewer allergens. And allergy shots for owners might shift from burdensome weekly or monthly injections to a shot that offers immediate relief.

The new gene-editing technology called CRISPR/Cas9 might even come to the rescue, delivering the ultimate dream to those who can afford it: a cat that doesnt produce allergens at all. One company has made some progress applying CRISPR/Cas9 to cats.

Success in taming cat allergies could bring good news for people whose allergies have nothingto do with cats. If any of the cat allergyfighting measures prove safe and effective, they could be deployed against other allergens, especially airborne ones like pollen, dog dander or dust mites.

Read the original post

Visit link:
Dreaming of hypoallergenic cats and how CRISPR could 'come to the rescue' - Genetic Literacy Project

CRISPR is a powerful gene-editing technology that scientists use to change the genetic blueprint of plants and animals and even humans.

Since its development about 10 years ago, its been used to change the DNA of living things in beneficial ways creating pink tomatoes and mushrooms that dont go brown, for example, and crops that resist insect attacks.

This technology operates efficiently in virtually all cell types of organisms in which its been tested, CRISPR co-inventor Jennifer Doudna, a biochemist at the University of California, Berkeley, said in an interview last May. It was really quite amazing how quickly it was possible to harness this technology once it was clear how it operated.

CRISPR (also known as CRISPR/Cas9) could also be used to create human designer babies with specific traits for example, a specific eye color or possibly enhanced intelligence. Most scientists have scrupulously avoided pursuing this controversial line of research, although a Chinese scientist stoked controversy in 2018 when he claimed to have used CRISPR to edit the genes of twin girls before their birth in order to make them immune to HIV, the virus that causes AIDS.

The name CRISPR is an acronym for clustered regularly interspaced short palindromic repeats, but you dont need to understand that brain-boggling term in order to understand how CRISPR works.

In short, it works by identifying a specific strand of DNA for example, the genetic instructions that determine eye color and replacing it by cutting" the original DNA and pasting in replacement DNA.

There are other gene-editing techniques, but they are slow and expensive in comparison to CRISPR. What used to take weeks or months can now be done in days with CRISPR.

Some modern CRISPR gene-editing kits, consisting of a few petri dishes, pipettes and bottles of special proteins, are small enough to keep on a shelf in the fridge and it can take as little as two days to see results.

Beyond creating better crops and hardier farm animals, CRISPR offers the tantalizing prospect of revolutionizing human health by bringing cures for genetic diseases: We are really on the threshold of a technology that is going to enable that to treat it at its source, by correcting the code in the DNA, Doudna said in a recent video.

In a series of experiments conducted a few years ago at the Broad Institute, a biomedical institute of MIT and Harvard in Cambridge, Massachusetts, scientists used CRISPR to improve hearing in mice with a certain form of hereditary deafness.

And in experiments at several University of California campuses published in 2016, researchers fixed defective bone marrow cells in a way that could offer a cure for sickle-cell anemia, a potentially deadly condition that affects an estimated 250 million people around the world.

Scientists are also using CRISPR in an effort to wipe out malaria by creating malaria-resistant mosquitoes, which would replace the wild populations of mosquitoes that spread the disease.

Though scientists see huge potential in CRISPR technology for treating human genetic diseases, theyve generally avoided using CRISPR to edit the genes of human embryos, citing the potential dangers of the technology and the ethical issues that surround its use for that purpose.

The actions of the Chinese researcher, He Jiankui, have drawn stern criticism from scientists and bioethicists, who called the work dangerous, unethical and even amateur. They point out that scientific knowledge of CRISPR and human genetics is far from perfect and that the twin girls could suffer as they grow up from genetic problems created by the CRISPR editing of their genes.

FOLLOW NBC NEWS MACH ON TWITTER, FACEBOOK, AND INSTAGRAM.

Link:
What is CRISPR? - NBC News

In August of 2011, Carl June and his team published a landmark paper showing their CART treatment had cleared a patient of cancer. A year-to-the-month later, Jennifer Doudna made an even bigger splash when she published the first major CRISPR paper, setting off a decade of intense research and sometimes even more intense public debate over the ethics of what the gene-editing tool could do.

Last week, June, whose CART work was eventually developed by Novartis into Kymriah, published in Sciencethe first US paper showing how the two could be brought together. It was not only one of the first time scientists have combined the groundbreaking tools, but the first peer-reviewed American paper showing how CRISPR could be used in patients.

June used CRISPR to edit the cells of three patients with advanced blood cancer, deleting the traditional T cell receptor and then erasing the PD1 gene, a move designed to unleash the immune cells. The therapy didnt cure the patients, but the cells remained in the body for a median of 9 months, a major hurdle for the therapy.

Endpoints caught up with June about the long road both he and the field took to get here, if the treatment will ever scale up, and where CRISPR and other advancements can lead it.

The interview has been condensed and edited.

Youve spoken in the past about howyou started working in this field in the mid-90s after your wife passed away from cancer. What were some of those early efforts? How did you start?

Well, I graduated from high school and had a low draft number [for the Vietnam War] and was going to go to study engineering at Stanford, but I was drafted and went into the Naval Academy in 1971, and I did that so I wouldnt have to go to the rice fields.

The war ended in 73, 74, so when I graduated in 1975, I was allowed to go to medical school, and then I had a long term commitment to the Navy because they paid for the Acadamy and Medical school. And I was interested in research and at the time, what the Navy cared about was a small scale nuclear disaster like in a submarine, and like what happened at Chernobyl and Fukushima. So they sent me to the Fred Hutchinson Cancer Center where I got trained in cancer, as a medical oncologist. I was going to open a bone marrow transplant center in Bethesda because the Navy wanted one in the event of a nuclear catastrophe.

And then in 1989, the Berlin Wall came down and there was no more Cold War. I had gone back to the Navy in 86 for the transplant center, which never happened, so then I had to work in the lab full time. But in the Navy, all the research has to be about combat and casualty. They care about HIV, so my first papers were on malaria and infectious disease. And the first CAR-T trials were on HIV in the mid-90s.

In 96, my wife got diagnosed with ovarian cancer and she was in remission for 3-4 years. I moved to the University of Pennsylvania in 1999 and started working on cancer because I wasnt allowed to do that with the Navy. My wife was obviously a lot of motivation to do that. She passed away in 2001. Then I started working with David Porter on adoptive transfer T cells.

I got my first grant to do CAR-T cells on HIV in 2004, and I learned a whole lot. I was lucky to have worked on HIV because we did the first trials using lentiviruses, which is an engineered HIV virus.

I was trained in oncology, and then because of the Navy forced to work on HIV. It was actually a blessing in disguise.

So if you hadnt been drafted, you wouldve become an engineer?

Yes. Thats what I was fully intending. My dad was a chemical engineer, my brother is an engineer. Thats what I thought I was going to do. No one in my family was ever a physician. Its one of those many quirks of fate.

Back then, we didnt have these aptitude tests. It was just haphazard. I applied to three schools Berkeley, Stanford and Caltech and I got into all three. It was just luck, fate.

And it turned out when I went to the Naval Academy, they had added a pre-med thing onto the curriculum the year before, so thats what I did when I started, I did chemistry.

I wouldve [otherwise] been in nuclear submarines. The most interesting thing in the Navy then was the nuclear sub technology.

You talked about doing the first CAR-T trials on HIV patients because thats where the funding was. Was it always in your head that this was eventually going to be something for cancer?

So I got out of the Navy in 99 and moved to Penn. I started in 98 working on treating leukemia, and then once I got to Penn, I continued working one day a week on HIV.

Its kind of a Back-to-the-Future thing because now cancer has paved out a path to show that CART cells can work and put down the manufacturing and its going to be a lot cheaper making it for HIV. I still think thats going to happen.

Jim Riley, who used to be a postdoc in my lab, has some spectacular results in monkeys with HIV models. They have a large NIH and NIAID research program.

So were going to see more and more of that. The CAR technology is going to move outside of cancer, and into autoimmune and chronic infections.

I want to jump over to cytotoxic release syndrome (CRS)because a big part of the CRISPR study was that it didnt provoke this potentially deadly adverse effect. When did you first become aware that CRS was going to be a problem?

I mean we saw it in the very first patient we treated but in all honesty, we missed it. Im an MD, but I dont see the patient and David Porter tookcare of the first three patients and our first pediatric patient,Emily Whitehead.

In our first patients, 2 out of 3, had complete remission and there were fevers and it was CRS but we thought it was just an infection, and we treated with antibiotics for 3 weeks and[eventually] it went away. And sort of miraculously he was in remission and is still in remission, 9 years later.

And then when we treated Emily. She was at a 106-degree fever over three days, and there was no infection.

Ive told this story before. My daughter has rheumatoid arthritis, and I had been president of the Clinical Immunologists Society from 2009 to 2010, and the first good drug for juvenile rheumatoid arthritisthat came out. I was invited to give the Japanese scientist Tadamitsu Kishimoto the presidential award for inventing the drug.

Then in 2012, Emily Whitehead was literally dying from CRS, she had multiple organ failures. And her labs came back and IL-6 levels were 1000x normal. It turns out the drug I was looking at for my daughter, it blocks IL-6 levels. I called the physician and I said, listen theres something actionable here, since its in your formulary to give it to her off-label.

And she gave her the appropriate dose for rheumatoid arthritis. It was miraculous. She woke up very rapidly.

Now its co-labeled. When the FDA approvedKymriah, it was co-labeled. It kind of saved the field.

How were you feeling during this time? Did you have any idea what was happening to her?

No, not until we got the cytokine levels, and then it was really clear. The cytokine levels go up and it exactly coincided. Then we retroactively checked out adults and they had adverse reactions and it easy to see. We hadnt been on the lookout because it wasnt in our mouse models.

And it appeared with those who got cured. Its one of the first on-target toxicities seen in cancer, a toxicity that happens when you get better. All the toxicities from chemotherapy are off-target: like leukopenia or hair loss.

I had a physician who had a fever of 106, I saw him on a fever when he was starting to get CRS. When the nurse came in and it said 106, they thought the thermometer must be broken. On Monday, I saw him, and said how are you feeling and he said fine. And I looked at the thermometer and histemperature was still 102.

People will willingly tolerate on-target toxicity thats very different from chemotherapy if they know it helps get them better. Thats a new principle in cancer therapy.

You had these early CART results almost at the same time that Doudna publishes the first CRISPR papers, then still in bacteria. When did you first start thinking about combining the two?

Yeah, it was published inSciencein 2012 and thats when Emily Whitehead got treated. Its an amazing thing.

Thats something so orthogonal. You think how in the heck can that ever benefit CART cells? but my lab had done the first edited cells in patients, published in 2012. And we used zinc-fingered nucleases, which were the predecessors to CRISPR. It knocked out one gene at a time, but we showed it was safe.

I was already into gene editing because it could make T cells resistant to HIV. So it was pretty obvious that there were candidates in T cells that you can knock out. And almost every lab started working on some with CRISPR, cause it was much easier.

We were the first to get full approval by the FDA, so we worked on it from 2012, had all the preclinical data by 2016, and then it takes a while to develop a lot of new assays for this as we were very cautious to optimize safety and it took longer than we wanted, but in the end, we learned a tremendous amount.

So what did we learn?

First of all our patients had advanced metastatic cancer and had had a lot of chemotherapy. The first patient had had 3 bone marrow transplants.

One thing is feasibility: could you really do all the complex engineering? So we found out we could. feasibility was passed.

Another was the fact that cas9 came out of bacteria, forms of strep and staph. Everyone has pre-existing immunity to Cas9 and we had experience from the first trial with Sangamo[with zinc-finger nucleases] where some patients had a very high fever. In that case, we had used adenoviruses, and it turned out our patients had very high levels of baseline immune response to adenoviruses, so we were worried that would happen with CRISPR, and it did not happen.

It did not have any toxicity. If it had, it would have really set the field back. If there was animmune response to cas9 and CRISPR, there couldve been a real barrier to the field.

And then, the cells survived in the patients. The furthest on, it was 9 months. The cells had a very high level of survival. In the previous trials, the cells survived less than 7 days. In our case, the half-life was 85 days. We dont know the mechanism yet.

And we found very big precision in the molecular scissors, and that was a good thing for the field. You could cut 3 different genes on 3 different chromosomes and have such high fidelity.

It [CRISPR] is living up to the hype. Its going to fix all these diseases.

Whats the potential in CAR-T, specifically?

Well theres many many genes that you can add. There are many genes that knocking outwill make the cells work better. We started with the cell receptor. There are many, I think, academics and biotechs doing this now and it should make the cells more potent and less toxic.

And more broadly, what else are you looking at for the future of CART? The week before your paper, there were the results from MD Anderson on natural killer cells.

Different cell types, natural killer cells, stem cells putting CAR molecules into stem cells, macrophages. One of my graduate students started a company to do CAR macrophages and macrophages actually eat tumor cells, as opposed to T cells that punch holes in them.

There will be different cell types and there will be many more ways to edit cells. The prime editing and base editing. All different new variations.

Youve talked about how people used to think the immuno-oncology, if it ever worked, would nevertheless be a boutique treatment. Despite all the advancements, Novartis and Gilead still have not met the sales they once hoped to grab from their CART treatments. Are you confident CART will ever be widely accessible?

Oh yeah, Novartis sales are going up. They had a hiccup launching.

Back in 96 or 97, when Genentech launched Herceptin, their commercial antibody, they couldnt meet the demand either and then they scaled up and learned how to do better cultures. So right now Novartis is using tech invented in my lab in the 1990s culture tech thats complex and requires a lot of labor, so the most expensive part is human labor. A lot can be made robotic. The scale problem will be much easier.

Thats an engineering problem that will become a thing of the past. The manufacturing problem will get a lot cheaper. Here in the US, we have a huge problem with how drugs are priced. We have a problem with pricing. Thats a political issue.

But in cell therapy, its just kind of the growth things you see in a new industry. Itll get worked out.

This article has been updated to reflect that Jim Riley conducted work on CAR in HIV.

Originally posted here:
Carl June on CRISPR, CART and how the Vietnam War dropped him into medicine - Endpoints News

Key point: Humanity isn't ready for gene editing.

More than a year ago, the world was shocked by Chinese biophysicist He Jiankuis attempt to use CRISPR technology to modify human embryos and make them resistant to HIV, which led to the birth of twins Lulu and Nana.

Now, crucial details have been revealed in a recent release of excerpts from the study, which have triggered a series of concerns about how Lulu and Nanas genome was modified.

How CRISPR works

CRISPR is a technique that allows scientists to make precise edits to any DNA by altering its sequence.

When using CRISPR, you may be trying to knock out a gene by rendering it inactive, or trying to achieve specific modifications, such as introducing or removing a desired piece of DNA.

Read more: What is CRISPR gene editing, and how does it work?

Gene editing with the CRISPR system relies on an association of two molecules. One is a protein, called Cas9, that is responsible for cutting the DNA. The other molecule is a short RNA (ribonucleic acid) molecule which works as a guide that brings Cas9 to the position where it is supposed to cut.

The system also needs help from the cells being edited. DNA damage is frequent, so cells regularly have to repair the DNA lesions. The associated repair mechanisms are what introduce the deletions, insertions or modifications when performing gene editing.

How the genomes of Lulu and Nana were modified

He Jiankui and his colleagues were targeting a gene called CCR5, which is necessary for the HIV virus to enter into white blood cells (lymphocytes) and infect our body.

One variant of CCR5, called CCR5 32, is missing a particular string of 32 letters of DNA code. This variant naturally occurs in the human population, and results in a high level of resistance to the most common type of HIV virus.

The team wanted to recreate this mutation using CRISPR on human embryos, in a bid to render them resistant to HIV infection. But this did not go as planned, and there are several ways they may have failed.

First, despite claiming in the abstract of their unpublished article that they reproduced the human CCR5 mutation, in reality the team tried to modify CCR5 close to the 32 mutation.

As a result, they generated different mutations, of which the effects are unknown. It may or may not confer HIV resistance, and may or may not have other consequences.

Worryingly, they did not test any of this, and went ahead with implanting the embryos. This is unjustifiable.

A second source of errors could have been that the editing was not perfectly efficient. This means that not all cells in the embryos were necessarily edited.

When an organism has a mixture of edited and unedited cells, it is called a mosaic. While the available data are still limited, it seems that both Lulu and Nana are mosaic.

This makes it even less likely that the gene-edited babies would be resistant to HIV infection. The risk of mosaicism should have been another reason not to implant the embryos.

Read more: 'Designer' babies won't be common anytime soon despite recent CRISPR twins

Moreover, editing can have unintended impacts elsewhere in the genome.

When designing a CRISPR experiment, you choose the guide RNA so that its sequence is unique to the gene you are targeting. However, off-target cuts can still happen elsewhere in the genome, at places that have a similar sequence.

He Jiankui and his team tested cells from the edited embryos, and reported only one off-target modification. However, that testing required sampling the cells, which were therefore no longer part of the embryos - which continued developing.

Thus, the remaining cells in the embryos had not been tested, and may have had different off-target modifications.

This is not the teams fault, as there will always be limitations in detecting off-target and mosaicism, and we can only get a partial picture.

However, that partial picture should have made them pause.

A bad idea to begin

Above, we have described several risks associated with the modifications made on the embryos, which could be passed on to future generations.

Embryo editing is only ethically justifiable in cases where the benefits clearly outweigh the risks.

Technical issues aside, the researchers did not even address an unmet medical need.

While the twins father was HIV-positive, there is already a well-established way to prevent an HIV-positive father from infecting embryos. This sperm washing method was actually used by the team.

The only benefit of the attempted gene modification, if proven, would have been a reduced risk of HIV infection for the twins later in life.

But there are safer existing ways to control the risk of infection, such as condoms and mandatory testing of blood donations.

Implications for gene editing as a field

Gene editing has endless applications. It can be used to make plants such as the Cavendish banana more resistant to devastating diseases. It can play an important role in the adaptation to climate change.

In health, we are already seeing promising results with the editing of somatic cells (that is, non-heritable modifications of the patients own cells) in beta thalassemia and sickle cell disease.

However, we are just not ready for human embryo editing. Our techniques are not mature enough, and no case has been made for a widespread need that other techniques, such as preimplantation genetic testing, could not address.

Read more: Experts call for halt to CRISPR editing that allows gene changes to pass on to children

There is also much work still needed on governance. There have been individual calls for a moratorium on embryo editing, and expert panels from the World Health Organisation to UNESCO.

Yet, no consensus has emerged.

It is important these discussions move in unison to a second phase, where other stakeholders, such as patient groups, are more broadly consulted (and informed). Engagement with the public is also crucial.

Correction: this article originally described RNA (ribonucleic acid) as a protein, rather than a molecule.

Dimitri Perrin, Senior Lecturer, Queensland University of Technology and Gaetan Burgio, Geneticist and Group Leader, The John Curtin School of Medical Research, Australian National University

This article is republished from The Conversation under a Creative Commons license. Read the original article.

Image: Reuters

Link:
Gene Editing Might Let China Create The Perfect Human Being - The National Interest Online

A revolution is taking place in the knowledge base for life sciences and biotechnology, opening up new applications in healthcare, agriculture, and environmental protection. Political awareness of this potential dates back to 2001, when the European Commission recognized life sciences and biotech through the adoption of its life science and biotechnology strategy.

With the European Green Deal, the new European Commission has set out an ambitious roadmap towards a climate neutral continent in 2050. With that, Europe strives to become a global frontrunner and lead the way in tackling the climate crisis. Taking the potential of biotechnology and life sciences in benefiting people and planet, a renewed focus and impetus on life sciences and biotechnology are all the more necessary. Regaining leadership in the sector should be a fundamental priority for the EU.

In agriculture, biotechnology offers sustainable food solutions through applying newest technologies. Biotechnology, (including genetic modification of crops), has increased farmers yields and incomes while reducing CO2 emissions, and the need for farmer inputs. Meanwhile, a science-based, risk-proportionate and non-discriminatory regulatory framework that allows for gene editing in crops could pave the way for products which offer health and consumer benefits ..

Read the original post

Read the rest here:
Viewpoint: If Europe wants to be 'carbon neutral,' it needs to embrace biotechnologyGMO and CRISPR crops included - Genetic Literacy Project

Feb. 19, 2020 13:00 UTC

BERKELEY, Calif. & RICHMOND, Calif.--(BUSINESS WIRE)-- Caribou Biosciences, Inc., a leading CRISPR genome editing company, and ProMab Biotechnologies, Inc., a biotechnology CRO/CDMO specializing in antibody engineering and CAR-T development, today announced a sale and assignment agreement under which Caribou gains access to a ProMab humanized single-chain variable fragment (scFv) targeting the B Cell Maturation Antigen (BCMA) for use in allogeneic engineered cell therapies. Caribou intends to utilize this scFv in the development of its CB-011 program, an allogeneic CAR-T therapy targeting BCMA-positive tumors including multiple myeloma.

We are excited for the opportunity to have access to this highly advanced, humanized molecule and believe it will significantly advance our promising CB-011 CAR-T program, said Steven Kanner, PhD, Chief Scientific Officer of Caribou.

We anticipate that our humanized BCMA scFv will aid greatly in Caribous efforts to further its allogeneic CAR-T program, and hope our technology continues to improve the field of preclinical and clinical stage immunotherapy research by providing broad choices of validated antibodies, said John Wu, MD, Chief Executive Officer of ProMab.

Under the terms of the agreement, ProMab received an upfront payment and is eligible for royalties on net sales of licensed products containing the BCMA scFv.

About Caribou Biosciences, Inc. Caribou is a leading company in CRISPR genome editing founded by pioneers of CRISPR-Cas9 biology. The company is developing an internal pipeline of off-the-shelf CAR-T cell therapies, other gene-edited cell therapies, and engineered gut microbes. Additionally, Caribou offers licenses to its CRISPR-Cas9 foundational IP in multiple fields including research tools, internal research use, diagnostics, and industrial biotechnology. Interested companies may contact Caribou at licensing@cariboubio.com. For more information about Caribou, visit http://www.cariboubio.com and follow the Company @CaribouBio. Caribou Biosciences and the Caribou logo are registered trademarks of Caribou Biosciences, Inc.

About ProMab Biotechnologies, Inc. ProMab Biotechnologies focuses on developing and commercializing mouse, rabbit, and human monoclonal antibodies as well as chimeric antigen receptor-T Cell (CAR-T) products. ProMabs CAR-T platform covers both hematological and solid cancers with intensive in vitro and in vivo pre-clinical validation designed for safer and better treatment. As a CRO in the immunology field for 19 years, ProMab offers standard laboratory procedures and animal studies for antibody discovery through the integration of the newest techniques in antibody library construction, next generation sequencing, unique humanization modeling, high-throughput screening, and artificial intelligence analysis systems. ProMab aims to out-license antibodies validated in CAR-T therapy in the preclinical stage or to bring CAR-T technologies to the early stage market of clinical study. ProMab has partnered with top biotechnology startups, medical institutions, and pharmaceutical companies to advance the development of cell therapies as well as bispecific antibodies targeting multiple cancers. For more information, visit http://www.promab.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200219005112/en/

See the original post here:
Caribou Biosciences and ProMab Biotechnologies Announce Sale and Assignment Agreement for Humanized scFv Targeting BCMA - BioSpace

Zacks Investment Research upgraded shares of Crispr Therapeutics (NASDAQ:CRSP) from a sell rating to a hold rating in a research report sent to investors on Monday, Zacks.com reports.

According to Zacks, CRISPR Therapeutics AG is a gene-editing company. It focused on the development of transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR Therapeutics AG is headquartered in Basel, Switzerland.

Other research analysts have also recently issued research reports about the stock. Needham & Company LLC reiterated a buy rating and set a $84.00 price target on shares of Crispr Therapeutics in a research note on Monday, December 23rd. Chardan Capital reiterated a buy rating and set a $72.50 price target on shares of Crispr Therapeutics in a research note on Thursday, February 13th. Canaccord Genuity lifted their price target on shares of Crispr Therapeutics from $72.00 to $80.00 and gave the stock a positive rating in a research note on Wednesday, November 20th. TheStreet upgraded shares of Crispr Therapeutics from a d rating to a c rating in a research note on Monday, October 28th. Finally, William Blair reiterated a buy rating on shares of Crispr Therapeutics in a research note on Thursday, February 13th. Two equities research analysts have rated the stock with a sell rating, two have issued a hold rating and thirteen have assigned a buy rating to the companys stock. The stock presently has an average rating of Buy and a consensus price target of $78.29.

Crispr Therapeutics (NASDAQ:CRSP) last issued its earnings results on Wednesday, February 12th. The company reported $0.51 earnings per share (EPS) for the quarter, topping the Thomson Reuters consensus estimate of ($0.68) by $1.19. The company had revenue of $77.00 million for the quarter, compared to analysts expectations of $39.08 million. Crispr Therapeutics had a net margin of 23.09% and a return on equity of 11.74%. The firms quarterly revenue was up 76900.0% on a year-over-year basis. During the same quarter last year, the company posted ($0.92) earnings per share. On average, analysts predict that Crispr Therapeutics will post -4.61 EPS for the current year.

Several institutional investors and hedge funds have recently added to or reduced their stakes in CRSP. Nikko Asset Management Americas Inc. lifted its stake in shares of Crispr Therapeutics by 48.4% during the 3rd quarter. Nikko Asset Management Americas Inc. now owns 2,777,414 shares of the companys stock valued at $113,846,000 after buying an additional 906,006 shares in the last quarter. Orbimed Advisors LLC purchased a new position in shares of Crispr Therapeutics during the 3rd quarter valued at $21,167,000. FMR LLC lifted its stake in shares of Crispr Therapeutics by 71.8% during the 4th quarter. FMR LLC now owns 952,369 shares of the companys stock valued at $58,004,000 after buying an additional 398,012 shares in the last quarter. Renaissance Technologies LLC lifted its stake in shares of Crispr Therapeutics by 904.0% during the 4th quarter. Renaissance Technologies LLC now owns 394,564 shares of the companys stock valued at $24,031,000 after buying an additional 355,264 shares in the last quarter. Finally, ARK Investment Management LLC lifted its stake in shares of Crispr Therapeutics by 6.3% during the 4th quarter. ARK Investment Management LLC now owns 2,956,635 shares of the companys stock valued at $180,074,000 after buying an additional 174,495 shares in the last quarter. Institutional investors own 51.28% of the companys stock.

Crispr Therapeutics Company Profile

CRISPR Therapeutics AG, a gene editing company, focuses on developing transformative gene-based medicines for the treatment of serious human diseases using its regularly interspaced short palindromic repeats associated protein-9 (CRISPR/Cas9) gene-editing platform in Switzerland. Its lead product candidate is CTX001, an ex vivo CRISPR gene-edited therapy for treating patients suffering from dependent beta thalassemia or severe sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells.

Read More: What is a Call Option?

Get a free copy of the Zacks research report on Crispr Therapeutics (CRSP)

For more information about research offerings from Zacks Investment Research, visit Zacks.com

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.

View post:
Crispr Therapeutics (NASDAQ:CRSP) Upgraded to Hold by Zacks Investment Research - Enterprise Echo

Global CRISPR/Cas9 Market research report gives a comprehensive outlook of the markets 2019-2025 and offers an in-depth summary of the current market status, historic, and expected way forward for the CRISPR/Cas9 Market. Additionally, to this, the report provides data on the restraints negatively impacting the markets growth. The report includes valuable information to assist new entrants, as well as established players, to understand the prevailing trends in the Market.

Download Free Sample Copy of CRISPR/Cas9 Market Report: https://dataintelo.com/request-sample/?reportId=103652

Key Objectives of CRISPR/Cas9 Market Report: Study of the annual revenues and market developments of the major players that supply CRISPR/Cas9 Analysis of the demand for CRISPR/Cas9 by component Assessment of future trends and growth of architecture in the CRISPR/Cas9 Market Assessment of the CRISPR/Cas9 Market with respect to the type of application Study of the market trends in various regions and countries, by component, of the CRISPR/Cas9 Market Study of contracts and developments related to the CRISPR/Cas9 Market by key players across different regions Finalization of overall market sizes by triangulating the supply-side data, which includes product developments, supply chain, and annual revenues of companies supplying CRISPR/Cas9 across the globe

Major Players included in this report are as follows Caribou BiosciencesIntegrated DNA Technologies (IDT)CRISPR TherapeuticsMerckMirus BioEditas MedicineTakara BioThermo Fisher ScientificHorizon Discovery GroupIntellia TherapeuticsAgilent TechnologiesCellectaGenScriptGeneCopoeiaSynthego

CRISPR/Cas9 Market can be segmented into Product Types as Genome EditingGenetic engineeringgRNA Database/Gene LibrarCRISPR PlasmidHuman Stem CellsGenetically Modified Organisms/CropsCell Line Engineering

CRISPR/Cas9 Market can be segmented into Applications as Biotechnology CompaniesPharmaceutical CompaniesAcademic InstitutesResearch and Development Institutes

To Buy this report, Visit https://dataintelo.com/checkout/?reportId=103652

CRISPR/Cas9 Market: Regional analysis includes: Asia-Pacific (Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia) Europe (Turkey, Germany, Russia UK, Italy, France, etc.) North America (United States, Mexico, and Canada.) South America (Brazil etc.) The Middle East and Africa (GCC Countries and Egypt.)

Target Audience: CRISPR/Cas9 Equipment Manufacturers Traders, Importers, and Exporters Raw Material Suppliers and Distributors Research and Consulting Firms Government and Research Organizations Associations and Industry Bodies

Stakeholders, marketing executives and business owners planning to refer a market research report can use this study to design their offerings and understand how competitors attract their potential customers and manage their supply and distribution channels. When tracking the trends researchers have made a conscious effort to analyse and interpret the consumer behaviour. Besides, the research helps product owners to understand the changes in culture, target market as well as brands so they can draw the attention of the potential customers more effectively.

Customize Report and Inquiry for The CRISPR/Cas9 Market Report: https://dataintelo.com/enquiry-before-buying/?reportId=103652

Report structure: In the recently published report, DataIntelo.com has provided a unique insight into the CRISPR/Cas9 Industry over the forecasted period. The report has covered the significant aspects which are contributing to the growth of the global CRISPR/Cas9 Market. The primary objective of this report is to highlight the various key market dynamics listed as drivers, trends, and restraints.

These market dynamics have the potential to impact the global CRISPR/Cas9 Market. This report has provided the detailed information to the audience about the way CRISPR/Cas9 industry has been heading since past few months and how it is going to take a shape in the years to come.

DataIntelo has offered a comprehensive analysis of the CRISPR/Cas9 industry. The report has provided crucial information about the elements that are impacting and driving the sales of the CRISPR/Cas9 Market. The section of competitive landscape keeps utmost importance in the reports published by DataIntelo. Competitive landscape section consists of key market players functioning in the worldwide industry of CRISPR/Cas9.

The report has also analysed the changing trends in the industry. Several macroeconomic factors such as Gross domestic product (GDP) and the increasing inflation rate is expected to affect directly or indirectly in the development of the CRISPR/Cas9 Market.

Table of Contents 1 Industry Overview of CRISPR/Cas9 2 Manufacturing Cost Structure Analysis 3 Development and Manufacturing Plants Analysis of CRISPR/Cas9 4 Key Figures of Major Manufacturers 5 CRISPR/Cas9 Regional Market Analysis 6 CRISPR/Cas9 Segment Market Analysis (by Type) 7 CRISPR/Cas9 Segment Market Analysis (by Application) 8 CRISPR/Cas9 Major Manufacturers Analysis 9 Development Trend of Analysis of CRISPR/Cas9 Market 10 Marketing Channel 11 Market Dynamics 12 Conclusion 13 Appendix

Ask for Discount on CRISPR/Cas9 Market Report at: https://dataintelo.com/ask-for-discount/?reportId=103652

About DataIntelo: DATAINTELO has set its benchmark in the market research industry by providing syndicated and customized research report to the clients. The database of the company is updated on a daily basis to prompt the clients with the latest trends and in-depth analysis of the industry. Our pool of database contains various industry verticals that include: IT & Telecom, Food Beverage, Automotive, Healthcare, Chemicals and Energy, Consumer foods, Food and beverages, and many more. Each and every report goes through the proper research methodology, validated from the professionals and analysts to ensure the eminent quality reports.

Contact Info DataIntelo Name Alex Mathews Email [emailprotected] Website https://dataintelo.com Address 500 East E Street, Ontario, CA 91764, United States.

Read more here:
CRISPR/Cas9 Market Demand Analysis and Projected huge Growth by 2025 - News Parents

Reports Monitors report on the global CRISPR Technology market studies past as well as current growth trends and opportunities to gain valuable insights of the same indicators for the CRISPR Technology market during the forecast period from 2019 to 2024. The report provides the overall global market statistics of the global CRISPR Technology market for the period of 20192024, with 2018 as the base year and 2024 as the forecast year. The report also provides the compound annual growth rate (CAGR) for the global CRISPR Technology market during the forecast period.

SWOT Analysis of Leading Contenders covered in this report:- Thermo Fisher Scientific, Merck KGaA, GenScript, Integrated DNA Technologies (IDT), Horizon Discovery Group, Agilent Technologies, Cellecta, GeneCopoeia, New England Biolabs, Origene Technologies, Synthego Corporation, Toolgen and more.

Get access to sample report, Click here @https://www.reportsmonitor.com/request_sample/584056

The global CRISPR Technology market was xx million US$ in 2018 and is expected to xx million US$ by the end of 2024, growing at a CAGR of xx% between 2019 and 2024.

This report studies the CRISPR Technology market size (value and volume) by players, regions, product types and end industries, history data 2014-2018 and forecast data 2019-2024; This report also studies the global market competition landscape, market drivers and trends, opportunities and challenges, risks and entry barriers, sales channels, distributors and Porters Five Forces Analysis.

Product Type Segmentation:-

EnzymesKitsgRNALibrariesDesign Tools

Industry Segmentation:-

BiomedicalAgricultural

The CRISPR Technology market report includes an elaborate executive summary, along with a snapshot of the growth behavior of various segments included in the scope of the study. Furthermore, the report sheds light on changing competitive dynamics in the global CRISPR Technology market. These indices serve as valuable tools for existing market players as well as for entities interested in entering the global CRISPR Technology market.

Get a discount on this report@https://www.reportsmonitor.com/check_discount/584056

The report reaches inside into the competitive landscape of the global CRISPR Technology market. Key players operating in the global CRISPR Technology market have been identified, and each one of them has been profiled for their distinguishing business attributes. Company overview, financial standings, recent developments, and SWOTs are some of the attributes of players in the global CRISPR Technology market that have been profiled in this report.

Regional Coverage:-

The report has been prepared after extensive primary and secondary research. Primary research involves the bulk of research efforts wherein, analysts carry out interviews with industry leaders and opinion-makers. Extensive secondary research involves referring to key players product literature, annual reports, press releases, and relevant documents to understand the global CRISPR Technology market.

Secondary research also includes Internet sources, statistical data from government agencies, websites, and trade associations. Analysts have employed a combination of top-down and bottom-up approaches to study various phenomena in the global CRISPR Technology market.

Key Questions Answered in CRISPR Technology Market Report

View this report with a detailed description and TOC @ https://www.reportsmonitor.com/report/584056/CRISPR-Technology-Market

Contact UsJay MatthewsDirect: +1 513 549 5911 (U.S.)+44 203 318 2846 (U.K.)Email: [emailprotected]

Read the original post:
CRISPR Technology Market Analysis with Key Players, Applications, Trends and Forecasts to 2025 | Thermo Fisher Scientific, Merck KGaA, GenScript -...

European Pharmaceutical Review explores how plants can be used for large-scale, glycosylated protein bioproduction for the pharma industry.

Plants can be used to produce large quantities of complex proteins, particularly glycosylated proteins, which are becoming more widely used in a range of therapies. Monoclonal antibodies (mAbs) are among the types of glycosylated proteins that plants can produce, but while there are multiple benefits to their use as bioreactors, there are also some key considerations.

This article explores why and how plants can be used to produce proteins for use in therapies, but also the factors that show this method may not be applicable to all protein products.

For plants to produce synthetic proteins, they must first be expressed somewhere within their genome. This requires some form of recombinant protein expression or genetic engineering, and to achieve optimum yield just implanting the gene is insufficient. To achieve a high level of transcription, which allows for downstream translation and protein modification for stability, the regulatory gene elements including the promoter and polyadenylation site must also be expressed.1

Techniques for gene expression:

There are three commonly used types of expression mechanisms for plant bioproduction: nuclear, chloroplast and transient expression.

Nuclear expression involves genetically modifying the genome in the nuclei of plants cells to express a protein. This is the simplest and most widely used approach in the pharmaceutical industry, as it can be achieved with viral vectors, but a more modern technique is CRISPR-Cas9 technologies.1 A 2018 study showed that in cotton, CRISPR showed no offtarget editing and an editing efficiency of 66.7 to 100 percent at each of multiple sites.2 The nuclear expression techniques, although reliable, are becoming less popular as they typically require more time to develop.

The second method involves expression of a recombinant protein in the chloroplasts requires a particle gun to insert the transgene. There are several benefits to this technique, including the ease of manipulating the chloroplast genome compared with the nucleus and the number of chloroplasts per cell, which increases yield. Using a transgene cassette to precisely target and insert the foreign gene avoids placing it into a poorly transcribed part of the genome, ensuring a high level of expression and little chance of silencing. Transgenes are commonly integrated between the trnltrnA genes in the rrn operon, as this is a transcriptionally active region offering high levels of gene expression.1

The third mechanism, transient expression, is becoming more common as it allows the rapid insertion of proteins, with little time required for the production, modification and optimisation of the expression system. Some companies have begun marketing this kind of expression for the rapid, large-scale production of proteins for therapeutics. The Agrobacteriummediated transient expression technique is purported to have better efficiency than the integrated gene systems and the ability to reach a high percentage of cells in a treated tissue, resulting in higher yields.1

In prokaryotic cells, like Escherichia coli (E. coli), protein size is limited to less than 30 kilodaltons, mainly due to reliability of production and yield. However, in eukaryotic cells, eg, Chinese hamster ovary (CHO) andplant cells, it is easier to produce larger proteins with high yields.1

According to experts, when using cell line or bacterial production methods such as CHO cells and E. coli to produce proteins, once the initial cell line is created it is often difficult to scale up, as glycosylation profiles become variable.3 The inconsistencies in protein product both cost money and result in waste.

On the other hand, dependent on expression mechanisms, plants can reliably maintain the glycosylation profile required even as bioreactor volume increases.

As a result of consistent production capabilities, plants do not require scale-up protocols. This saves both time and money when setting up a bioreactor.

A further advantage is that, if the plant is made to generate the protein through a transient expression system, there is very little time required to set up a production system. One company claims their tobacco plant-based system can be tailored for large-scale fabrication of a protein product in under 12 months, compared to 20-22 months with CHO or E. coli, 3 and one study suggests this could be done in a matter of weeks.1

There are multiple options for plant expression systems, particularly with regards to species, and each is best suited to produce different proteins. Genetic engineering can also be employed to allow customised N-glycosylation to generate different target products.

The plant industry is well established, with conditions for growth often being less complex than that of cell lines or bacteria and, dependent on choice of plant species, cultivation costs can be further reduced.

A techno-economic analysis of the theoretical set-up of a new large-scale biomanufacturing facility, producing mAbs using tobacco plants, found that compared to CHO production platforms, the plant system resulted in significantly reduced capital investment. Moreover, the model calculated that there would be more than a 50 percent reduction in the cost of goods, compared with published values for similar products at this production scale.4

One company has paved the way for the creation of biobetters, using their FastGlycaneering Development Service. iBio has shown that certain methods of plant bioproduction can improve the potency and homogeneity of biological medicines and ensure fully humanised glycosylation patterns.

iBio have also stated that their system, due to its consistencies in upstream processing, is compatible with artificial intelligence (AI). The company aim to implement a new end-to-end manufacturing process using AI and blockchain to reduce costs through optimising both the process and workflows.3

Some of the major challenges include regulatory approval, environmental contamination, protein stability and the immunogenicity of non-human post-translational modifications.1

Environmental concerns are predominantly from the possibility of spreading genetic modifications to food crops through pollination. This is more of a concern with the nuclear expression systems than transient or chloroplast expression. However, this can be overcome with geographical or physical containment, using a less transferable genetic modification method or through using a self-pollenating species.1

A review suggested that companies are unlikely to go through the cost of a shift from an already approved production system to seek regulatory approval for a new one.1 While altering an approved process is often unfeasible, setting up systems for the production of new products in the pipeline could prove to be more cost effective in the long run. Another consideration is the rising need for quick, large-scale vaccine production in response to pandemics and epidemics such as the Covid-19 coronavirus and Ebola which, due to the speed at which a transient expression production system can be constructed, could encourage companies to branch into this type of production.

Protein stability is a concern, as plants have endogenous enzymes that can break down the protein products. Some methods to overcome this include changing plant species and co-expressing peptides to fuse and stabilise the produced proteins together.

Post-translational modifications such as Asparagine-linked glycosylation (N-glycosylation) are one of the key worries, as they can be immunogenic. Particularly likely to cause unfavourable side effects are N-glycan modifications, because they differ in plants and humans.

N-glycosylation is a post-translational modification conducted on many secreted or membrane proteins in plants and mammals. Endogenously, it enables protein folding, stabilisation and protein-protein interactions. It is similarly used in pharmaceutical bioproduction to stabilise products and provide antibodies and other proteins the correct pharmacokinetic properties and immunogenicity.5,6

The plant industry is well established, with conditions for growth often being less complex than that of cell lines or bacteria

While early N-glycosylation and N-glycan modifications are highly conserved between yeast, mammals and plants, later N-glycan modifications differ; they are more simplified in plants than mammals.5,6 So, to use plants as producers of fully humanised proteins, the plant glycosylation machinery is often removed and replaced with human machinery when the plant is modified to express the protein. Of note, chloroplasts have no glycosylation machinery, so cannot perform these modifications without the insertion of foreign DNA; although this can reduce immunogenicity of the products, it can limit which proteins can be produced by chloroplast expression.

Tobacco is the most widely used plant for production of recombinant proteins in the lab. High yield and rapid scale-up, due to large numbers of seeds produced, are the primary benefits. However, proteins stored in the leaves are vulnerable to degradation and must be stored or extracted appropriately, in a timely manner. Tobacco tissues can also contain phenols and toxic alkaloids that must be removed in downstream processing to make products safe.1

Cereals are primarily used due to their seed protein storage capabilities; cereal seeds have protein storage vesicles and a dry intracellular environment. Once dried, the seeds can be stored at room temperature with limited degradation to protein products or loss of activity. Use of food crops is particularly attractive as they offer the opportunity to administer oral vaccines produced in the crop by feeding them to patients with minimal processing. Some edible vaccines have reached Phase I trials.1

Peas are a particularly attractive option, as they have high protein content in their seeds similar to cereals and have lower nitrogen requirements, reducing cultivation costs. However, legumes usually have less leaf biomass than tobacco, meaning they require a larger area to produce the same quantity.1

Plants can be modified through several methods to express proteins and the requisite promoters and transcription controllers, for the production of therapeutic proteins. There are several important considerations, including protein expression methods and plant species; however, the many benefits, including reduced costs, adaptability and speed associated with plant bioproduction systems make them an attractive option.

A particular driver of this bioproduction process is the possibility of using transient expression to produce vast quantities of highly potent, fully humanised vaccines in response to pandemics and epidemics.

iBio

See the rest here:
Using plants as bioreactors to produce proteins for therapeutics - European Pharmaceutical Review

Sifting through a soup of genes sampled from many environments, including human saliva, animal poop, lakes, hospitals, soils and more, researchers have found hundreds of giant viruses - some with abilities only seen before in cellular life.

The international team, led by scientists from University of California, Berkeley, has discovered entire new groups of giant phages (viruses that infect bacteria) and pieced together 351 gene sequences.

Within these they found genes that code for unexpected things, including bits of the cellular machinery that reads and executes DNA instructions to build proteins, also known as translation.

"They have an unusual number of components of the translation machinery that you do not find on a typical virus," microbiologists Basem Al-Shayeb and Jill Banfield from UC Berkeley told ScienceAlert.

The translation process takes place in molecular structures known as ribosomes, and the researchers actually found genes that code for some of their components - ribosomal proteins.

"Typically, what separates life from non-life is to have ribosomes and the ability to do translation; that is one of the major defining features that separates viruses and bacteria, non-life and life," said microbial ecologist Rohan Sachdeva from UC Berkeley.

"Some large phages have a lot of this translational machinery, so they are blurring the line a bit."

The team also found sequences for CRISPR systems, which also happens to be the 'immune system' bacteria use against viruses, the very same system we humans have co-opted for our own gene manipulation purposes.

The newly discovered viruses all have genomes more than 200,000 base pairs long, whereas the average known phage size is more along the lines of 52,000 base pairs.

Some phage genomes identified by the team were true whoppers; the researchers have named one group Whopperphage, and designated the other nine new groups after the word "big" in the different languages of the contributing authors.

"The genomes of these phages are at least four times the size of a typical phage, and the largest is 15 times larger - 735,000 bases of DNA," Al-Shayeb and Banfield said.

These larger phages are thought to infect Bacteroidetes, a group of bacteria widely dispersed in our environment, from soil to our intestines.

The genomes of these hefty phages are large enough to rival those of small bacteria, but the amoeba-infecting pandoraviruses still hold the title of the largest viral genome at 2.5 million base pairs.

"Large phages have been found before, but they were spot findings," Sachdeva told the Innovative Genomics Institute. "What we found in this paper is they are essentially ubiquitous. We find them everywhere."

Like other phages, these chonkers inject their DNA into their bacterial host, hijacking the victim's gene replication equipment to make copies of themselves.

The researchers suspect that while this is happening, the giants also use some of their additional genes to derail early stages of translation inside the bacteria, and divert protein production to suit their own needs. Such control of protein creation has also been observed in animal viruses.

Al-Shayeb explained that giant phages use their CRISPR system for phage-on-phage warfare, by specifically targeting competing viruses that try to infect the same host bacterium. A study from last year shows how some phages use this system to thwart anti-phage measures their host bacteria may deploy.

A huge phage (Subject 26) infecting a bacterium and manipulating its response to other phages. (Jill Banfield Lab/UC Berkeley)

"The sense we have looking at these large genomes is that phages have acquired a lot of different genes and pathways - some of which we can predict, some of which we can't for really taking control of bacterial hosts' function during infection," Banfield told the Innovative Genomics Institute.

As we learn more about the links between our physical and mental health and the microbes we share our bodies and environments with, it is clear that what affects them can also profoundly impact us.

"Phages are also known to transfer genes for bacterial toxins and antibiotic resistance between bacteria, which contribute to disease," Al-Shayeb said.

"Since we have both harmful and useful bacteria living on us and within us, understanding what kinds of phages coexist with them in humans and animals and how they affect those environments is of great value."

The researchers suggest that the interesting CRISPR systems some of these phages possess may have the potential to help us control our own microbiomes, by altering the function of bacteria or eliminating the troublesome ones.

They now hope to grow some of these whopper phages in the lab, to learn more about these phage-associated CRISPR systems and "discover their roles and test for value in genome editing", according to Al-Shayeb and Banfield.

Biochemist Christoph Weigel, who was not associated with the study, suggested on Twitter that the paper provides "strong support" for considering viruses living "virocells".

"These huge phages bridge the gap between non-living bacteriophages, on the one hand, and bacteria and Archaea," explained Banfield.

"There definitely seem to be successful strategies of existence that are hybrids between what we think of as traditional viruses and traditional living organisms."

Whatever else this huge addition to our knowledge of viral biodiversity brings, it's already sparking further discussion on what it means to be alive.

This study was published in Nature.

Read more:
Scientists Discover Giant Viruses With Features Only Seen Before in Living Cells - ScienceAlert

According to researchers, a certain bacterial strain has acquired a new gene that makes it able to resist antibiotics via the DNA sequence known as CRISPR, clustered regularly interspaced short palindromic repeats (not to be confused with the CRISPR gene-editing technology).

Bacteria acquire CRISPR sequences from infecting viruses called bacteriophages, which insert fragments of DNA into bacterial genomes, the University of Washington School of Medicine, which was involved in the study, reported in a news release explaining how bacteria get these pieces of DNA via viral infection. (Yes, even bacteria can get a viral infection.) These bacteriophages hijack their host system to reproduce and can leave bits of DNA behind. In this case the CRISPR sequence appears to have included the drug-resistance gene.

The recent study, led by Dr. Alex Greninger, assistant professor of laboratory medicine at the UW School of Medicine, discovered nearly identical bacteria among these unrelated men in the two cities, and concluded it is likely being transmitted by men who have sex with men.

Campylobacter is one of the most common causes of diarrhea around the world. In fact, according to a Centers for Disease Control and Prevention estimate, it causes 1.5 million illnesses in the United States every year. People usually recover without treatment, but those with serious cases or compromised health require antibiotics. What makes this new strain particularly dangerous is that it is resistant to those antibiotics used for treatment.

Enteric infections can be sexually transmitted infections, Greninger said in the press release, about the intestinal infections that can be transmitted via anal intercourse, rimming, or other sexual practices. The international spread of related isolates among MSM populations has been shown before for Shigella, so it makes sense to see it in Campylobacter as well. The group of bacteria called Shigella cause about 500,000 cases of diarrhea in the United States annually, and outbreaks among gay and bisexual men have been noted for two decades.

Men who have sex with men are at higher risk of multidrug resistance because theyre more likely to have taken antibiotics to treat past STIs, the authors of the new study state. According to MedScape, Campylobacter infections may be more common and cause prolonged or recurrent diarrhea among those living with HIV.

While STI rates have increased significantly over the last few years, less is known about STIs related to intestinal bacteria. This outbreak among gay and bisexual men with a strain that is resistant to antibiotics raises the stakes.

The global emergence of multidrug-resistant enteric pathogens in MSM poses an urgent public health challenge that may require new approaches for surveillance and prevention, the authors concluded.

Read more:
Gay and Can't Stop Pooping? This Could Be Why - HivPlusMag.com

CRISPR Therapeutics (NASDAQ:CRSP) reported its fourth-quarter financial results on Wednesday afternoon, substantially beating revenue expectations and impressing both analysts and investors alike.

Fourth-quarter revenue grew to $77 million, a substantial increase from the mere $100,000 reported in Q4 2018. Total annual revenue came in at $289.6 million, while last year's income came in at a much smaller $3.1 million. While this surge is mainly due to collaboration agreements with Vertex Pharmaceuticals as opposed to product sales, it's still an impressive increase considering analysts had expected just $45.2 million for the quarter.

Image source: Getty Images.

Other financial metrics, such as CRISPR's cash position, have improved as well. By Dec. 31, cash and cash equivalents grew to $943.8 million, a 106.7% increase from the $456.6 million reported last year. The biotech company is also now reporting a net profit thanks to this revenue hike. Net income came in at $30.5 million, whereas last year CRISPR reported a net loss of $47.6 million in its 2018 fourth quarter.

CRISPR is developing four main treatments. CTX001 is a treatment for patients with sickle cell disease and transfusion-dependent beta thalassemia. Both of these are genetic blood disorders that impact the blood's ability to transport oxygen throughout the body.

The company's three other drugs, CTX110, CTX120, and CTX130, are types of cancer treatments known as CAR-T therapies. While other healthcare companies are developing their own CAR-T drugs, these types of therapies tend to be quite expensive. CRISPR's technology could make the development process for these types of drugs much cheaper than their competition.

See the original post here:
CRISPR Therapeutics Reports Q4 Earnings, Beats Revenue Expectations - The Motley Fool

CRISPR Therapeutics AG (CRSP) came out with quarterly earnings of $0.51 per share, beating the Zacks Consensus Estimate of $0.04 per share. This compares to loss of $0.92 per share a year ago. These figures are adjusted for non-recurring items.

This quarterly report represents an earnings surprise of 1,175%. A quarter ago, it was expected that this company would post a loss of $0.95 per share when it actually produced earnings of $2.40, delivering a surprise of 352.63%.

Over the last four quarters, the company has surpassed consensus EPS estimates two times.

CRISPR Therapeutics AG, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $77.02 million for the quarter ended December 2019, surpassing the Zacks Consensus Estimate by 3.42%. This compares to year-ago revenues of $0.12 million. The company has topped consensus revenue estimates two times over the last four quarters.

The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call.

CRISPR Therapeutics AG shares have lost about 6.4% since the beginning of the year versus the S&P 500's gain of 3.9%.

What's Next for CRISPR Therapeutics AG?

While CRISPR Therapeutics AG has underperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock?

There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately.

Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions.

Ahead of this earnings release, the estimate revisions trend for CRISPR Therapeutics AG was unfavorable. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #4 (Sell) for the stock. So, the shares are expected to underperform the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

It will be interesting to see how estimates for the coming quarters and current fiscal year change in the days ahead. The current consensus EPS estimate is -$1.08 on $7.50 million in revenues for the coming quarter and -$4.65 on $30.04 million in revenues for the current fiscal year.

Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the top 28% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1.

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportTo read this article on Zacks.com click here.Zacks Investment Research

Read this article:
CRISPR Therapeutics AG (CRSP) Beats Q4 Earnings and Revenue Estimates - Yahoo Finance

PHILADELPHIA - Three patients with advanced cancer suffered no serious side effects from being treated at the University of Pennsylvania in the first U.S. clinical study of cells edited with CRISPR, the gene editing technology.

But neither did they benefit, according to results published this month in Science. One patient with a bone marrow cancer called multiple myeloma has died and another has progressed. A patient with sarcoma, a soft tissue cancer, also progressed.

Pilot clinical trials are designed to assess safety, not effectiveness. And the inaugural U.S. test of CRISPR-edited cells in humans was so ethically and scientifically fraught that Penn spent more than two years getting necessary approvals for the January 2018 launch.

Still, the experiment was intended to combine and improve on two revolutionary immune-boosting approaches that showed startling effectiveness even in early trials. One approach, which cuts a natural brake on the immune system, has led to a class of cancer drugs called checkpoint inhibitors. The other approach genetically engineers immune soldiers called T cells to recognize and attack cancer cells; the first approved T-cell therapy, Novartis' Kymriah, was pioneered at Penn and Children's Hospital of Pennsylvania.

Although the CRISPR-edited cells did not melt away tumors or stop cancer progression, the cells did survive and grow in patients for up to nine months. In a previous Penn clinical trial that used engineered T cells to attack multiple myeloma, half the cells were dead in a week.

Penn T-cell researcher Carl June, principal leader of the new study and previous groundbreaking work, sees the CRISPR trial as another incremental step in a medical odyssey. CRISPR technology was invented just eight years ago, yet the version used by Penn is already so outmoded that the trial has been discontinued.

"We learned what we wanted to learn," June said of the study, on which Stanford University collaborated. "It opens up the door for a lot of new approaches."

What they learned was mostly reassuring.

The difficult, many-step manufacturing process was feasible. CRISPR was used on T cells to cut out two genes, one that codes for the immune system brake, and another that could hamper the T cells' ability to latch onto cancer cells. CRISPR also inserted a gene that enabled the T cells to recognize and target a protein found on the cancer cells but not healthy cells.

These edits accidentally caused some unusual rearrangements of DNA in a small fraction of T cells - one of the biggest worries with CRISPR. But as the T cells took hold and multiplied in patients, these rearrangements steadily decreased, "suggesting that they conferred no growth advantage," the researchers wrote.

The edited T cells did not trigger any of the dangerous toxicities related to revving up the immune system that are common with checkpoint inhibitors and engineered T cells.

However, the researchers noted that a longer trial with more patients and higher doses will be needed "to fully assess the safety of this approach."

A problem that has occurred with engineered T-cell therapies also showed up in the CRISPR trial: one patient's cancer cells stopped making the protein, called NY-ESO-1, that the T cells had been edited to target.

In future trials, "we would not just use NY-ESO-1 because we've learned the tumor can live without it," June said. "We'd want to use multiple targets."

Renier J. Brentjens, an oncologist and cell therapy researcher at Memorial Sloan-Kettering Cancer Center in New York City, called the paper an important "proof of principle."

"The T cells persisted and found the tumors. It would have been nice to see remissions or tumor regression, but it doesn't necessarily mean the approach is flawed," Brentjens said. "It may be that the target they used is not sufficient."

(c)2020 The Philadelphia Inquirer

Distributed by Tribune Content Agency, LLC.

Continued here:
Study suggests editing human genes to fight cancer is safe. But does it work? - PostBulletin.com