Archive for the ‘Crispr’ Category

Editors note: WRAL TechWire today launches a new package of features focusing on innovation. This package will be part of Innovation Thursday.

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INNOVATION ROAD TRIP: Part One

Earlier this year, WRAL published a series titled Innovation Road Trip that featured six video and story packages from across North Carolina. Check out part one:

How CRISPR is solving problems through DNA editing

Written by Abbey Slattery, WRAL Digital Solutions

Eradication of certain diseases, increasing crop sizes, reducing pest populations the current and future applications of CRISPR have the potential to change ways of life around the world.

A tool for editing genomes including altering DNA and modifying gene functions CRISPR is short for CRISPR-Cas9 and refers to a string of DNA and the Cas9 enzyme. CRISPR works by finding a specific section of DNA, cutting it, then inserting a mutation or replacing a faulty gene.

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Innovation Thursday: How CRISPR is solving problems through DNA editing - WRAL TechWire

Cell and gene therapies are taking the world by storm. With CRISPR gene-editing technology, the genome is no longer an untouchable sacred text. Instead, genomic DNA is actively manipulated in the laboratory and the clinic. This revolution is changing the bench, the bedside, and the boardroom. As a result, the cell and gene therapy market is valued at USD 4.99 billion and is expected to grow to over USD 36 billionby 2027.

The potential of personalized medicinewhere treatments are specific to an individual's geneticsexplains this exponential growth. Cell therapies, such as CAR-Ts, modify cells outside the patient and reintroduce them as a targeted treatment. In comparison, gene therapies alter the DNA within cells. The final genetic dosage from a cell or gene therapy walks a razor's edge between therapeutic and toxic. This makes the quality control testing of cell and gene therapies absolutely vital. Many turn to qRT-PCR for this task, but this method cannot deliver absolute quantification as each run requires a standard curve, creating inconsistencies.

In this article, we explore how Droplet Digital PCR (ddPCR) technology is revolutionizing quality control for cell and gene therapies, enabling scientists to save time, money, and resources through confident quantification.

Before we dive into how ddPCR assays reimaginethe limits of quality control in cell and gene therapies, lets explore ddPCR technology. Like qRT-PCR, ddPCR technology requires a nucleic acid sample. However, in ddPCR assays, samples are partitioned into 20,000 discrete droplets. Individual PCR reactions occur within each droplet, which on average contains just a single copy of the donor DNA. During PCR cycling, positive droplets that contain the target DNA will produce a fluorescent signal. This digital system results in absolute quantification without the need for standard curves, leading to unparalleled precision, simplified workflows, and removal of PCR bias.

Generating cell and gene therapies is complex, requiring precise quality control. CRISPR, while powerful, does not have a 100% success rate. However, you need 100% certainty in your product. Thats where the absolute quantification from ddPCR technology comes in. Here we explore several instances where ddPCR assays allow scientists to expand cell and gene therapy boundaries.

The ideal quality control assay for gene therapy would simultaneously quantify the transfer of genetic material and changes in expression. In their 2021 publication, Clarner et al. did just that using a one-step RT-ddPCR method. Their method quantified transgene expression and potency with both RNAi and augmentation vectors in vitroand in vivo,using non-human primate models. They noted that the absolute quantification from ddPCR reduced variability and provided a more streamlined workflow.

In a second example, CRISPR can generate knock-ins to increase gene dosage, which is technically challenging because integration efficiency is low. The process of developing knock-ins becomes time and resource-intensive and requires processing many failures.CRISPR SNIPERuses ddPCR technology to quantify knock-ins with precision and efficiency. Because ddPCR assays partition donor DNA into 20,000 droplets, CRISPR SNIPER can accurately measure low-frequency integration events that qRT-PCR simply cannot detect.

Finally, ddPCR methods are excellent for cell therapy transgene quantification. CAR-T cells are generated using viral vectors that insert transgenes into T-cell DNA. However, the number of integrated transgene copies can be variable.Lu et al. used ddPCR technology to accurately measure transgene copy insertion in CAR-T cells with high levels of reproducibility.

Across the board, ddPCR assays are uniquely positioned to catapult cell and gene therapies to the next level, standardizing and safeguarding the production process worldwide. ddPCR methods possess the accuracy, precision, and reproducibility required for cell and gene therapy quality control. Across applications from knock-in generation to CAR-T cell transgene quantification, this technology allows scientists to focus on what they do best: science. Meanwhile, ddPCR technology can take care of quality control.

Learn more about ddPCR applications for cell and gene therapy manufacturing.

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Next-level quality control in cell and gene therapy - BioPharma Dive

1. CRISPR 'cousin' put to the test in landmark heart-disease trial  Nature.com
2. CRISPR cure for high cholesterol enters first human trial  Freethink
3. Boston biotech Verve tests 'CRISPR 2.0 in a patient for the first time  The Boston Globe
4. Edits to a cholesterol gene could stop the biggest killer on earth  MIT Technology Review
5. Verve starts first human test of gene editing treatment for heart disease  BioPharma Dive
6. View Full Coverage on Google News

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CRISPR 'cousin' put to the test in landmark heart-disease trial - Nature.com

Can CRISPR edit out a heart attack? What happens on #GutTok? And is health care recession-proof?

We cover all that and more this week on The Readout LOUD, STATs biotech podcast. Sek Kathiresan, cardiologist and CEO of Verve Therapeutics, joins us to explain the companys work on preventing heart disease with genome editing. Then, STATs Isabella Cueto joins us to discuss Hot girls have IBS, an internet in-joke that evolved into a movement for people with chronic illness. We also break down the latest news in the life sciences, including a long-awaited victory for Novavax and ostensible good news for biotech.

For more on what we cover, heres the latest on Verve; heres the story on hot girls have IBS; heres the news on Novavax;heres where you can find episodes of Color Code; heres where you can subscribe to the First Opinion Podcast;and heres our complete coverage of the Covid-19 pandemic.

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Be sure to sign up on Apple Podcasts, Spotify, Stitcher,Google Play, or wherever you get your podcasts.

And if you have any feedback for us topics to cover, guests to invite, vocal tics to cease you can email [emailprotected].

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CRISPR for the heart, biotech's recovery, & what it means to be a 'hot girl' - STAT

BOSTON Researchers from the Center for Regenerative Medicine at Boston Medical Center and Boston University School of Medicine used variants of CRISPR to understand the functions of the genes that cause emphysema and chronic obstructive pulmonary disease (COPD). Published in Science Advances, researchers discovered functional consequences by turning off the expression of the gene that contributes to the pathogenesis of these diseases.

This is the first time that CRISPRi and CRISPRa have been applied in human induced pluripotent stem cells to understand the functional role of these genes, says Andrew Wilson, MD, a pulmonologist at Boston Medical Center and associate professor of medicine at Boston University School of Medicine. It gets us closer to understanding how inherited factors help contribute to susceptibility to emphysema.

COPD and emphysema is the third leading cause of death worldwide, creating a significant burden of disease. Emphysema is a complex genetic disease caused by a mutation or variant in a number of genes that contribute to making some individuals more susceptible to disease than others. Genome-wide association studies (GWAS) have implicated variants in or near a growing number of genes, but understanding their functions and how they potentially contribute to the development of COPD and emphysema is quite limited.

There have been no new significant pharmacological agents developed to help treat the large number of patients afflicted with COPD or emphysema worldwide, says Rhiannon Werder, MD, a postdoctoral fellow at the Center for Regenerative Medicine at Boston Medical Center and Boston University School of Medicine. Our hope is that this study will help in the understanding of the genetics of the disease, improve our understanding of how the disease occurs at a cellular level, and support the development of new therapies to treat these conditions.

Researchers devised a system using variants of CRISPR to either turn off expression of a gene of interest using CRISPR interference (CRISPRi) or overexpress a gene of interest using CRISPR activation (CRISPRa) in induced pluripotent stem cells (iPSCs). Researchers grew these cells in a dish and differentiated them to generate cells that reside in the lung. The cell type studied is called the type 2 alveolar epithelial cell, a progenitor cell for the alveolus the alveolus is the part of the lung where gas exchange occurs and is the structure that is damaged in emphysema. So by understanding how GWAS genes affect type 2 cells, researchers can start to understand how these might contribute to diseases that affect these cells, like emphysema.

Once type 2 cells were generated, researchers then used CRISPRi to turn off expression of nine different GWAS genes and analyzed them to see how the cells were affected, especially their ability to proliferate, something that they need to be able to do in response to injury like that which occurs in emphysema. Researchers noticed that turning off one particular gene, desmoplakin (DSP), caused the cells to increase their proliferation and increased their expression of genes associated with cellular maturation. Researchers found that cells in which DSP expression was turned off before smoke exposure turned off expression of cell junction genes to a greater degree than in controls. These were also better at forming new colonies, a measure of progenitor function, than controls. Researchers then looked in mice that had DSP deleted from their lung epithelial cells, compared to control mice with normal DSP. Researchers found that the type 2 cells in the DSP deletion mice were more proliferative following injury, consistent with findings in human iPSC-derived type 2 cells.

DSP appears to modulate the proliferative capacity of type 2 cells at baseline and following injuries that are relevant to human disease, such as smoke exposure. Lower levels of DSP expression increase the proliferative capacity of type 2 cells in the system, potentially making them better able to respond to an injury. In contrast, higher levels of expression as found in cells containing the variant associated with COPD risk by GWAS appear to make the cells less proliferative after smoke exposure, potentially explaining how this gene contributes to disease.

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CRISPR Technology Highlights Genes That Contribute to the Development of Emphysema and COPD - Boston Medical Center

Kenneth Research

Key Companies Covered in the Global Gene Therapy Market by Kenneth Research are Kineta, Inc., Novartis AG, Amgen Inc., bluebird bio, Inc., Gilead Sciences, Inc., Orchard Therapeutics plc, SIBIONO, Questex, CRISPR Therapeutics, Editas Medicine, and others.

New York, July 13, 2022 (GLOBE NEWSWIRE) -- According to the World Health Organization (WHO), around 10 million deaths, or nearly 1 in 6 deaths, were caused by cancer in 2020, making it the top cause of death globally. Breast, lung, colon, rectum, and prostate cancers are the most prevalent types of cancer. If found early and appropriately treated, many tumors (30% to 50%) are curable. According to the American Cancer Society (ACS), 1,918,030 new cancer cases and 609,360 cancer deaths are expected in 2022, with lung cancer as the primary cause of death accounting for about 350 of those fatalities daily in the United States.

In recent research titled Global Gene Therapy Market, Kenneth Research provided a brief overview of market elements including growth drivers, restraint factors, current market trends, and potential for future growth. The influence of COVID-19 and its effects on end-users are both thoroughly examined in the market research report, which covers the forecast period, i.e., 2022-2031. In addition, the research study examines the product portfolios and market expansion plans of the principal competitors.In 2020, according to the World Cancer Research Fund (WCRF), there were 18 million new cases of cancer worldwide. 9.3 million of these instances involved men, while 8.8 million involved women. The growth of the global gene therapy market can be attributed on account of the rising prevalence of cancer cases. Also, the adoption of gene therapies for the treatment of cancer is predicted to grow the market further. For instance, at the University of Pennsylvania, the first trial for testing a CRISPR-created cancer medicine was launched in the United States in 2019.CRISPR is a gene-editing-tool, that can modify any DNA segment within the 3 billion letters of the human genome. The global gene therapy market is expected to gather around USD 6 billion in revenue by 2031 and grow with a CAGR of ~34% over the forecast period. Get A Sample Copy of This Report @ https://www.kennethresearch.com/sample-request-10070542

The global gene therapy market is segmented on the basis of region into North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa. On the back of rapid rising cancer incidence rates, and the availability of high disposable income, the market in North America is predicted to experience significant expansion over the course of the forecast period. For instance, the Cancer Facts & Figures 2021 by the American Cancer Society, the study estimates that 1.9 million new instances of cancer were diagnosed and 608,570 cancer deaths in the United States in 2021. Also, an increase in the demand for gene-therapy-related R&D activities further helps the growth of the market. According to the World Bank Data, the domestic general government healthcare expenditure in the U.S. was 5,552.60 IN 2019 whereas in Canada the domestic general health care expenditure was 3,873.70 in 2019. Thus, a rise in government health care support is expected to expand the gene therapy-related R&D activities and further aid on to improve the market in the region.On the other hand, the global gene therapy market in the Asia Pacific region is anticipated to experience the greatest CAGR throughout the forecast period owing to the growing population in the region and increased approval and availability of gene therapy products. According to the World Bank data, the total population of China was 1.41 billion in 2020 whereas, India had 1.38 billion people in 2020. As the population grows, the likelihood of contracting a disease increases. Additionally, it is anticipated that increased government efforts to upgrade the health care infrastructure and rising healthcare costs in that region are expected to expand the industry. Also, the health care expenditure in Japan in 2019 was 10.74% whereas, in China, the GDP was 5.35%. In addition to that, the domestic general government health expenditure per capita for Japan was 3,846.54 in 2019 and China was 492.72 in 2019. Thus, growing health care expenditure and government support in health care expansion are further expected for the growth of the market in the region.

Browse to access In-depth research report on Gene Therapy Market with detailed charts and figures: https://www.kennethresearch.com/report-details/gene-therapy-market/10070542

The study further incorporates Y-O-Y Growth, demand & supply and forecasts future opportunities in North America (U.S., Canada), Europe (U.K., Germany, France, Italy, Spain, Hungary, Belgium, Netherlands & Luxembourg, NORDIC[Finland, Sweden, Norway, Denmark], Poland, Turkey, Russia, Rest of Europe), Latin America (Brazil, Mexico, Argentina, Rest of Latin America), Asia Pacific(China, India, Japan, South Korea, Indonesia, Singapore, Malaysia, Australia, New Zealand, Rest of Asia Pacific), Middle East and Africa(Israel, GCC[Saudi Arabia, UAE, Bahrain, Kuwait, Qatar, Oman], North Africa, South Africa, Rest of the Middle East and Africa).The global gene therapy market is segmented by indication into cancer, metabolic disorders, eye disorders, cardiovascular diseases, and others. Among that the cancer segment is predicted to hold the largest share over the forecast period. On account of the growing widespread presence of cancer cases, the growth of the market can be accredited. The estimated number of new cases of cancer patients in India was around 11,57,294 cases which had risen to 13,24,413 total cases in 2020. In addition to that, the total number of cancer patients was 1,708,921 in 2018 in the U.S., according to the Centers for Disease Control and Prevention (CDC) which got increased to an estimated rate of 1.8 million new cases in 2020. The statistical studies exhibit an increasingly widespread of the disease worldwide which is expected to drive the growth of the segment. Gene therapies are used to treat a variety of malignancies, including those of the brain, lung, breast, pancreatic, liver, prostate, bladder, head & neck, skin, and ovary. For instance, according to the World Cancer Research Fund (WCRF), the most common cancers around the world were breast and lung cancers, accounting to 12.5% and 12.2% respectively of all new cases that were expected to be diagnosed in 2020. Also, there were 1.9 million new instances of colorectal cancer, accounting for 10.7% of all cancer cases in 2020.

Get a Sample PDF of Global Gene Therapy Market @ https://www.kennethresearch.com/sample-request-10070542

The global gene therapy market is segmented by end-user into pharma & biotech, and academia. Numerous ongoing researches and studies have been conducted in the pharma and biotech sector which is anticipated to account for the growth of the segment. For instance, based on a study by PhRMA, there were 289 gene therapies done in clinical development by biopharmaceutical companies in 2018 which had increased to 362 gene therapies in 2020. Also, 6 diseases were already being treated using gene therapy, whereas 362 cell and gene therapies were in the development stage in 2020. In addition to that, 9 cell or gene therapy products have been approved by U.S. Food and Drug Administration (FDA) as of February 2020; they are used to treat cancer, eye conditions, and uncommon inherited diseases.

The global gene therapy market is also segmented on the basis of technology and application.

Global Gene Therapy Market, Segmentation by Technology:

Global Gene Therapy Market, Segmentation by Application:

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Some of the well-known leaders in the global gene therapy market that are included in our report are Kineta, Inc., Orchard Therapeutics plc, SIBIONO, Questex, CRISPR Therapeutics, Editas Medicine, and others.

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About Kenneth Research

Kenneth Research is a leading service provider for strategic market research and consulting. We aim to provide unbiased, unparalleled market insights and industry analysis to help industries, conglomerates and executives to take wise decisions for their future marketing strategy, expansion and investment, etc. We believe every business can expand to its new horizon, provided a right guidance at a right time is available through strategic minds. Our out of box thinking helps our clients to take wise decision so as to avoid future uncertainties.

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Global Gene Therapy Market to Garner a Revenue of About USD 6 Billion by 2031 by Growing with a CAGR of ~34% During 2022-2031; Market to Grow on...

What happened

The downtrodden biotech space has kicked off the second half of 2022 with a boom. Hard-hit gene-editing and gene therapy companies in particular have started the back half of the year on the right foot. Underscoring this point, Bluebird Bio (BLUE 6.97%) stock has already risen by 17% over the holiday-shortened week as of Thursday's closing bell, according to data provided by S&P Global Market Intelligence.

What's more, shares of CRISPR Therapeutics (CRSP 0.21%) have gained 22.6% over the same period, and fellow gene editor Editas Medicine (EDIT -0.20%) also saw its equity rise in price by a healthy 20.7% this week. By contrast, Bluebird and Editas shares both fell by over 50% over the first six months of 2022, while CRISPR's stock price stumbled by a noteworthy 20% during the first half of the year.

Image Source: Getty Images.

What's behind this sudden trend reversal? The most likely explanation is simply short-sellers covering their positions (buying back their borrowed shares). In keeping with this theme, Bluebird, Editas, and CRISPR all saw a sharp rise in their short interest during the first six months of 2022. Short-sellers piled into these three names earlier this year due to the fact that they are all cash flow negative, which is a tough spot to be in during a persistent bear market and an era of rising interest rates. Bluebird, in fact, is staring down a serious cash crunch at the moment.

Short-sellers, for their part, are probably backing away at this stage for no other reason than to play it safe in the event that big pharma starts to go bargain shopping.

Why might big pharma target beaten-down gene-editing and gene therapy companies in the second half of the year? The key reason is that these high-value fields are starting to move beyond the research stage of their life cycle and into the realm of commercially available therapies.

Speaking to this point, Bluebird's gene therapies for beta thalassemia and cerebral adrenoleukodystrophy appear to be on their way toward a formal approval from the Food and Drug Administration (FDA) following a pair of positive advisory committee votes last month. What's more, CRISPR is also expected to file for regulatory approval for its Vertex Pharmaceuticalspartnered blood disorder candidate, exa-cel, later this year.

Are any of these three biotech stocks still worth buying? CRISPR is arguably the most attractive bargain among the three. The company's ex-vivo gene-editing platform has posted stellar trial results so far, and Vertex could very well decide to buy its partner as a result.

Bluebird, on the other hand, is a tough call. The company ought to have a compelling buyout case if the FDA does grant it a pair of approvals soon. The bad news is that the biotech's balance sheet may force a sale at a heavily discounted price (relative to the commercial potential of its lead assets).

Finally, Editas might simply get lost in the mix when everything is said and done. There are several gene-editing companies vying for the spot of top dog, and Editas' clinical pipeline lags in several key areas at the moment. Time will tell.

George Budwell has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends CRISPR Therapeutics, Editas Medicine, and Vertex Pharmaceuticals. The Motley Fool recommends Bluebird Bio. The Motley Fool has a disclosure policy.

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Why Shares of Bluebird Bio, CRISPR Therapeutics, and Editas Medicine Soared This Week - The Motley Fool

Can gene-editing technology CRISPR create new crops that help fight climate change as they grow? Thats what a group of researchers hopes to do with $11 million in funding from the Chan Zuckerberg Initiative. The funding will go toward efforts to enhance plants starting with rice and soil so that theyre better at trapping carbon dioxide. The effort, which was announcedlast week, is being led by the Innovative Genomics Institute, which was founded byNobel laureateand co-inventor of CRISPR Jennifer Doudna.

[Jennifer] and I saw eye to eye on climate and how big of a problem it is in the world. And we just didnt want to sit on the sidelines anymore, says Innovative Genomics Institute (IGI) executive director Brad Ringeisen.

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The rice genome is easier to manipulate than other crops, according to Ringeisen, in part because its already been studied a lot and iswell understood. One of the scientists involved in IGIs initiative is Pamela Ronald, whose research is widely known for leading to thedevelopment of rice varietiesthat tolerate flooding for much longer than other types using a different type of genetic engineering thats more likeprecision breeding. That rice is now grown by more than 6 million farmers across India and Bangladesh,according toRonalds laboratory at the University of California, Davis.

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Climate change-fighting rice? Plants trap carbon dioxide as they grow and CRISPR gene editing can optimize this process - Genetic Literacy Project

In a trio of studies published on June 27 in the journalNature Microbiology, researchers at The University of Texas at Austin have discovered "fingerprints" of mysterious viruses hidden in an ancient group of microbes that may include the ancestors of all complex life on Earth: from fungi to plants to humans.

Ths discovery is significant; it explores the hypothesis that viruses were imperative to the evolution of humans and other complex life forms.

These microbes known as Asgard archaea after the abode of the gods in Norse mythology are usually found in the frigid sediments deep in the ocean and in boiling springs, and existed on Earth before the firsteukaryoticcells, which carry theirDNAinside a nucleus.

Some scientists have hypothesized that viruses may have played in role in how life forms first came to be by infecting the Asgard archaea. They may have even given rise to some of the first precursors to the nucleus. But, no Asgard-infecting viruses had been discovered hitherto. The latest research by Ian Rambo (a former doctoral student at UT Austin) and other members of Brett Baker's lab sheds light on how viruses might have played a role in this billions-year-old history.

"These are the first studies investigating Asgard archaeal viruses; there was nothing known before," Susanne Erdmann, group leader of the archaeal virology research group at the Max Planck Institute for Marine Microbiology in Bremen, Germany, who was not involved in the studies, toldLive Science. In the future, this line of research may reveal if and how viruses were involved in the emergence of eukaryotic cells on Earth, she said.

In the new research, scientists searched for evidence of viral infection embedded in the DNA of Asgard archaea - which comes in the form of viral DNA called "CRISPR spacers."

According to Rambo, most people who think of CRISPR relate it to the famous gene-editing tool that allows scientists to easily manipulate genetic sequences. This tool was originally adapted from the natural defense mechanisms of bacteria and archaea.

CRISPR refers to a region of DNA made up of short, repeated sequences with "spacers" sandwiched between each repeat. Interestingly, bacteria and archaea swipe these spacers from viruses that infect them, and the cells maintain a memory bank of viral DNA that helps them recognize the viruses should they attack again.

"It's an adaptive immune system that remembers these previous infections," said Rambo, who is now a postdoctoral scholar with the USDA's Agricultural Research Service.

Rambo and his colleagues had hunted in the Guaymas Basin in the Gulf of California the body of water between Baja California and mainland Mexico for such DNA spacers in Asgard archaea specimens collected from sediments near hydrothermal vents, roughly 1.25 miles (2 kilometers) beneath the water's surface.

The team matched the spacers they found to longer stretches of viral DNA gathered from the deep-sea environment.

The researchers could infer the kinds of proteins the various genes code for and how the viruses might function, working with viral DNA.

But, eventually, they could only figure out the functions of some of the viruses' genes; the functions of the vast majority of the genes are still unknown, Erdmann said. Also, because CRISPR doesn't work against all viruses, many more Asgard-infecting viruses are yet to be discovered, she said.

These hidden viruses could be found by growing Asgard archaea in the lab. "However, culturing Asgard archaea has been proven very difficult," Erdmann said. Until now, only one research group has managed to culture Asgard archaea, and it took them 12 long years to do it as archaeal cells take weeks to replicate.

But until more Asgards can be grown in the lab, CRISPR spacer matching is probably the most efficient way to find more viruses, Krupovic said. As more viruses are found, their role in the emergence of eukaryotes, including humans, may gain more clarity, added Rambo.

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Newly discovered viruses can offer clues about the rise of complex life on Earth - Interesting Engineering

Smartphones, superglue, electric cars, video chat. When does the wonder of a new technology wear off? When you get so used to its presence that you dont think of it anymore? When something newer and better comes along? When you forget how things were before?

Whatever the answer, the gene-editing technology CRISPR has not reached that point yet. Ten years after Jennifer Doudna and Emmanuelle Charpentier first introduced their discovery of CRISPR, it has remained at the center of ambitious scientific projects and complicated ethical discussions. It continues to create new avenues for exploration and reinvigorate old studies. Biochemists use it, and so do other scientists: entomologists, cardiologists, oncologists, zoologists, botanists.

Cathie Martin, a botanist at the John Innes Centre in Norwich, England, and Charles Xavier, founder of the X-Men superhero team: They both love mutants.

But while Professor X has an affinity for superpowered human mutants, Dr. Martin is partial to the red and juicy type. We always craved mutants, because that allowed us to understand functionality, Dr. Martin said of her research, which focuses on plant genomes in the hopes of finding ways to make foods especially tomatoes in her case healthier, more robust and longer lasting.

When CRISPR-Cas9 came along, one of Dr. Martins colleagues offered to make her a mutant tomato as a gift. She was somewhat skeptical, but, she told him, I would quite like a tomato that produces no chlorogenic acid, a substance thought to have health benefits; tomatoes without it had not been found before. Dr. Martin wanted to remove what she believed was the key gene sequence and see what happened. Soon a tomato without chlorogenic acid was in her lab.

Instead of looking for mutants, it was now possible to create them. Getting those mutants, it was so efficient, and it was so wonderful, because it gave us confirmation of all these hypotheses we had, Dr. Martin said.

Most recently, researchers at Dr. Martins lab used CRISPR to create a tomato plant that can accumulate vitamin D when exposed to sunlight. Just one gram of the leaves contained 60 times the recommended daily value for adults.

Dr. Martin explained that CRISPR could be used across a broad spectrum of food modifications. It could potentially remove allergens from nuts and create plants that use water more efficiently.

I dont claim that what we did with vitamin D will solve any food insecurity problems, Dr. Martin said, but its just a good example. People like to have something that they can hang on to, and this is there. Its not a promise.

Infectious Disease

Christian Happi, a biologist who directs the African Centre of Excellence for Genomics of Infectious Diseases in Nigeria, has spent his career developing methods to detect and contain the spread of infectious diseases that spread to humans from animals. Many of the existing ways to do so are costly and inaccurate.

For instance, in order to perform a polymerase chain reaction, or PCR, test, you need to go extract RNA, have a machine thats $60,000 and hire someone who is specially trained, Dr. Happi said. Its both costly and logistically implausible to take this kind of testing to most remote villages.

Recently, Dr. Happi and his collaborators used CRISPR-Cas13a technology (a close relative of CRISPR-Cas9) to detect diseases in the body by targeting genetic sequences associated with pathogens. They were able to sequence the SARS-CoV-2 virus within a couple of weeks of the pandemic arriving in Nigeria and develop a test that required no on-site equipment or trained technicians just a tube for spit.

If youre talking about the future of pandemic preparedness, thats what youre talking about, Dr. Happi said. Id want my grandmother to use this in her village.

The CRISPR-based diagnostic test functions well in the heat, is quite easy to use and costs one-tenth of a standard PCR test. Still, Dr. Happis lab is continually assessing the accuracy of the technology and trying to persuade leaders in the African public health systems to embrace it.

He called their proposal one that is cheaper, faster, that doesnt require equipment and can be pushed into the remotest corners of the continent. This would allow Africa to occupy what I call its natural space.

Hereditary Illness

In the beginning there was zinc finger nuclease.

That was the gene-editing tool that Gang Bao, a biochemical engineer at Rice University, first used to try to treat sickle cell disease, an inherited disorder marked by misshapen red blood cells. It took Dr. Baos lab more than two years of development, and then zinc finger nuclease would successfully cut the sickle cell sequence only around 10 percent of the time.

Another technique took another two years and was only slightly more effective. And then, in 2013, soon after CRISPR was used to successfully edit genes in living cells, Dr. Baos team changed tack again.

From the beginning to having some initial results, CRISPR took us like a month, Dr. Bao said. The method successfully cut the target sequence around 60 percent of the time. It was easier to make and more effective. It was just amazing, he said.

The next challenge was to determine the side effects of the process. That is, how did CRISPR affect genes that werent being purposefully targeted? After a series of experiments in animals, Dr. Bao was convinced that the method would work for humans. In 2020 the Food and Drug Administration approved a clinical trial, led by Dr. Matthew Porteus and his lab at Stanford University, that is ongoing. And there is also hope that with CRISPRs versatility, it might be used to treat other hereditary diseases. At the same time, other treatments that have not relied on gene editing have had success for sickle cell.

Dr. Bao and his lab are still trying to determine all the secondary and tertiary effects of using CRISPR. But Dr. Bao is optimistic that a safe and effective gene-editing treatment for sickle cell will be available soon. How soon? I think another three to five years, he said, smiling.

Cardiology

It is hard to change someones heart. And thats not just because we are often stubborn and stuck in our ways. The heart generates new cells at a much slower rate than many other organs. Treatments that are effective in other parts of the human anatomy are much more challenging with the heart.

It is also hard to know what is in someones heart. Even when you sequence an entire genome, there are often a number of segments that remain mysterious to scientists and doctors (called variants of uncertain significance). A patient might have a heart condition, but theres no way to tie it definitively back to their genes. You are stuck, said Dr. Joseph Wu, director of the Stanford Cardiovascular Institute. So traditionally we would just wait and tell the patient we dont know whats going on.

But over the past couple of years, Dr. Wu has been using CRISPR to see what kind of effects the presence and absence of these befuddling sequences have on heart cells, simulated in his lab with induced pluripotent stem cells generated from the blood. By cutting out particular genes and observing the effects, Dr. Wu and his collaborators have been able to draw links between the DNA of individual patients and heart disease.

It will be a long time before these diseases can be treated with CRISPR, but diagnosis is a first step. I think this is going to have a big impact in terms of personalized medicine, said Dr. Wu, who mentioned that he found at least three variants of uncertain significance when he got his own genome sequenced. What do these variants mean for me?

Sorghum is used in bread, alcohol and cereal all over the world. But it hasnt been commercially engineered to the same degree as wheat or corn, and, when processed, it often isnt as tasty.

Karen Massel, a biotechnologist at the University of Queensland in Australia, saw quite a bit of room for improvement when she first started studying the plant in 2015. And because millions of people eat sorghum worldwide, if you make a small change you can have a huge impact, she said.

She and her colleagues have used CRISPR to try to make sorghum frost tolerant, to make it heat tolerant, to lengthen its growth period, to change its root structure we use gene editing across the board, she said.

Not only could this lead to more delicious and healthier cereal, but it could also make the plants more resistant to the changing climate, she said. But it is still no small task to accurately edit the genomes of crops with CRISPR.

Half the genes that we knock out, we just have no idea what they do, Dr. Massel said. The second we try to get in there and play God, we realize were a bit out of our depth. But, using CRISPR combined with more traditional breeding techniques, Dr. Massel is optimistic, despite being a self-described pessimist. And she hopes that further advances will lead to commercializing gene-edited foods, making them more accessible and more acceptable.

In 2012, a 6-year-old girl was suffering from acute lymphoblastic leukemia. Chemotherapy had been unsuccessful, and the case was too advanced for a bone-marrow transplant. There didnt seem to be any other options, and the girls physicians told her parents to go back home.

Instead, they went to the Childrens Hospital of Philadelphia, where doctors used an experimental treatment called chimeric antigen receptor (CAR) T-cell therapy to turn the girls white blood cells against the cancer. Ten years later, the girl is cancer free.

Since then, Dr. Carl June, a medical professor at the University of Pennsylvania who helped develop CAR T-cell therapy, and his collaborators, including Dr. Ed Stadtmauer, a hematologist-oncologist at Penn Medicine, have been working to improve it. That includes using CRISPR, which is the simplest and most accurate tool to edit T-cells outside the body. Dr. Stadtmauer, who specializes in dealing with various types of blood and lymph system cancers, said that the last decade or so has just seen a revolution of treatment of these diseases; its been rewarding and exciting.

Over the past couple of years, Dr. Stadtmauer helped run a clinical trial in which T-cells that underwent significant CRISPR editing were inserted into patients with treatment-resistant cancers. The results were promising.

Nine months into the trial the edited T-cells had not been rejected by the patients immune systems and were still present in the blood. The real benefit is that scientists now know that CRISPR-aided treatments are possible.

Even though its really sort of science fiction-y biochemistry and science, the reality is that the field has moved tremendously, Dr. Stadtmauer said. He added that he was less excited by the science than how useful CRISPR had become. Every day I see maybe 15 patients who need me, he said. Thats what motivates me.

More:
The Many Uses of CRISPR: Scientists Tell All - The New York Times

Over the last several decades, genetic research has seen incredible advances in gene sequencing technologies. In 2004, scientists completed the Human Genome Project, an ambitious project to sequence the human genome, which cost $3 billion and took 10 years. Now, a person can get their genome sequenced for less than $1,000 and within about 24 hours.

Scientists capitalized on these advances by sequencing everything from the elusive giant squid to the Ethiopian eggplant. With this technology came promises of miraculous breakthroughs: all diseases would be cured and world hunger would be a thing of the past.

So, where are these miracles?

We need about 60 to 70% more food production by 2050.

In 2015, a group of researchers founded Yield10 Bioscience, an agriculture biotech company that aimed to use artificial intelligence to start making those promises into reality.

Two things drove the development of Yield10 Bioscience.

One, obviously, [the need for] global food security: we need about 60 to 70% more food production by 2050, explained Dr. Oliver Peoples, CEO of Yield10 Bioscience, in an interview with Freethink. And then, of course, CRISPR.

It turns out that having the tools to sequence DNA is only step one of manufacturing the miracles we were promised.

The second step is figuring out what a sequence of DNA actually does. In other words, its one thing to discover a gene, and it is another thing entirely to discover a genes role in a specific organism.

In order to do this, scientists manipulate the gene: delete it from an organism and see what functions are lost, or add it to an organism and see what is gained. During the early genetics revolution, although scientists had tools to easily and accurately sequence DNA, their tools to manipulate DNA were labor-intensive and cumbersome.

Its one thing to discover a gene, and it is another thing entirely to discover a genes role in a specific organism.

Around 2012, CRISPR technology burst onto the scene, and it changed everything. Scientists had been investigating CRISPR a system that evolved in bacteria to fight off viruses since the 80s, but it took 30 years for them to finally understand how they could use it to edit genes in any organism.

Suddenly, scientists had a powerful tool that could easily manipulate genomes. Equipped with DNA sequencing and editing tools, scientists could complete studies that once took years or even decades in mere months.

Promises of miracles poured back in, with renewed vigor: CRISPR would eliminate genetic disorders and feed the world! But of course, there is yet another step: figuring out which genes to edit.

Over the last couple of decades, researchers have compiled databases of millions of genes. For example, GenBank, the National Institute of Healths (NIH) genetic sequence database, contains 38,086,233 genes, of which only tens of thousands have some functional information.

For example, ARGOS is a gene involved in plant growth. Consequently, it is a very well-studied gene. Scientists found that genetically engineering Arabidopsis, a fast-growing plant commonly used to study plant biology, to express lots of ARGOS made the plant grow faster.

Dozens of other plants have ARGOS (or at least genes very similar to it), such as pineapple, radish, and winter squash. Those plants, however, are hard to genetically manipulate compared to Arabidopsis. Thus, ARGOSs function in crops in general hasnt been as well studied.

The big crop companies are struggling to figure out what to do with CRISPR.

CRISPR suddenly changed the landscape for small groups of researchers hoping to innovate in agriculture. It was an affordable technology that anyone could use but no one knew what to do with it. Even the largest research corporations in the world dont have the resources to test all the genes that have been identified.

I think if you talk to all the big crop companies, theyve all got big investments in CRISPR. And I think theyre all struggling with the same question, which is, This is a great tool. What do I do with it? said Dr. Peoples.

The algorithm can identify genes that act at a fundamental level in crop metabolism.

The holy grail of crop science, according to Dr. Peoples, would be a tool that could identify three or four genetic changes that would double crop production for whatever youre growing.

With CRISPR, those changes could be made right now. However, there needs to be a way to identify those changes, and that information is buried in the massive databases.

To develop the tool that can dig them out, Dr. Peoples team merged artificial intelligence with synthetic biology, a field of science that involves redesigning organisms to have useful new abilities, such as increasing crop yield or bioplastic production.

This union created Gene Ranking Artificial Intelligence Network (GRAIN), an algorithm that evaluates scientific databases like GenBank and identifies genes that act at a fundamental level in crop metabolism.

That fundamental level aspect is one of the keys to GRAINs long-term success. It identifies genes that are common across multiple crop types, so when a powerful gene is identified, it can be used across multiple crop types.

For example, using the GRAIN platform, Dr. Peoples and his team identified four genes that may significantly impact seed oil content in Camelina, a plant similar to rapeseed (true canola oil). When the researchers increased the activity of just one of those genes via CRISPR, the plants had a 10% increase in seed oil content.

Its not quite a miracle yet, but with more advances in gene editing and AI happening all the time, the promises of the genetic revolution are finally starting to pay off.

Wed love to hear from you! If you have a comment about this article or if you have a tip for a future Freethink story, please email us attips@freethink.com.

Continued here:
How artificial intelligence is boosting crop yield to feed the world - Freethink

Data Bridge Market Research analyses a growth rate in the global clustered regularly interspersed short palindromic repeats (CRISPR) market in the forecast period 2022-2029. The expected CAGR of global bone marrow biopsy market is tend to be around 10.7% in the mentioned forecast period. The market was valued at USD 762.39 million in 2021, and it would grow upto USD 1719.33 million by 2029. In addition to the market insights such as market value, growth rate, market segments, geographical coverage, market players, and market scenario, the market report curated by the Data Bridge Market Research team also includes in-depth expert analysis, patient epidemiology, pipeline analysis, pricing analysis, and regulatory framework.

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Applied StemCell (US), Agilant Technologies Inc. (US), Synthego (US), Thermo Fisher Scientific Inc (US), GenScript (US), Addgene (US), Merck KGaA (Germany), Intellia Therapeutics, Inc (US), Cellectis (France), Precision Biosciences (US), Caribou Biosciences, Inc (US), Transposagen Biopharmaceuticals, Inc (US), OriGene Technologies, Inc (US), Novartis AG (Switzerland), New England Biolabs Ltd. (UK)

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U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

The persuasive Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market marketing report provides top to bottom examination of the market as far as income and developing business sector is concerned. This business report displays systemic company profiles which illustrate how the moves of several key players and brands are driving the market. It also covers predictions regarding reasonable arrangement of uncertainties and latest techniques. The report also performs study on the market drivers and market restraints which are derived from SWOT analysis. The large scaleGlobal Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Marketmarket report considers wide scope that takes into account market scenarios, comparative pricing between major players, expenditure and profit of the specified market regions.

Under the topic of market segmentation, research and analysis is carried out based on application, vertical, deployment model, end user, and geography. Besides, competitive analysis assists to get ideas about the strategies of key players in the market via theGlobal Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Marketmarket document. Few of these strategies can be listed as; new product launches, expansions, agreements, partnerships, joint ventures, acquisitions, and others that help to broaden their footprints in the HEALTHCARE industry. The market share of key competitors on worldwide level is studied where main regions such as Europe, North America, Asia Pacific and South America are tackled in the universalGlobal Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Marketmarket survey report.

Highlights of TOC: Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Market

1 Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Market Overview

2 Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Market Competitions by Manufacturers

3 Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Capacity, Production, Revenue (Value) by Region (2022-2029

4 Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Supply (Production), Consumption, Export, Import by Region (2022-2029)

5 Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Production, Revenue (Value), Price Trend by Type

6 Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Market Analysis by Application

7 Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Manufacturers Profiles/Analysis

8 Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Manufacturing Cost Analysis

9 Industrial Chain, Sourcing Strategy and Downstream Buyers

10 Marketing Strategy Analysis, Distributors/Traders

11 Market Effect Factors Analysis

12 Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Market Forecast (2022-2029)

13 Research Findings and Conclusion

14 Appendix

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Goals and objectives of the Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Market Study

This Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Market Research/analysis Report Focus on following important aspects:

Key questions answered in the report:

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Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Size, Top Leading Countries, Companies, Consumption, Drivers, Trends,...

Blaupunkt, a German audio electronics brand has launched a new set of TWS earbuds in India called theBTW100TWS. They come withENC CRISPR Technologythat filters ambient noises while on a call and only picks up human voices. The earbuds are budget-friendly and have great German quality and technology. These TWS earbuds come with a stem design and charge inside an oval shaped charging case. Moreover, they have a battery life of up to 40 hours playtime.

The earbuds are powered by a powerful 10mm driver that produces punchy bass along with crystal clear mids and highs. The speakers produce Stereo high definition sound. For connectivity, the earbuds use Bluetooth 5.1 which enables a maximum range of 30ft without signal loss or mic dropout. These earbuds also feature an 80ms low latency mode for gaming and are enabled with Intuitive Touch Controls.

The Blaupunkt BTW100 TWS have a straight stem with chrome edges while the charging case has a clamshell-like design. The charging case packs a large 400mAh battery that is backed by TurboVolt Charging feature providing 1 hour of playback time with 15 minutes. Additional features include sweat, water & dust resistance.

The Blaupunkt BTW100 TWS earbuds are priced atRs 2,999and are available in two colour options Black and White. The product is available onAmazon.

Link:
Blaupunkt Launched Its BTW100 Truly Wireless Bluetooth Earbuds with ENC CRISPR Technology - Digit

Larger, deeper root systems can help store more carbon in the soil, because if a plant dies and parts of it are deep underground, the carbon in those pieces is less likely to make its way back into the air quickly. Roots arent the only possible storage option, Ringeisen says. Modified plants could also be used to make bio-oil or biochar, which can be pumped deep underground for storage.

Optimizing plants for carbon removal will be challenging, says Daniel Voytas, a genetic engineer at the University of Minnesota and a member of IGIs scientific advisory board.

Many of the traits that researchers want to alter in plants are influenced by multiple genes, which can make precise editing difficult, he says. And while some plants, like tobacco and rice, have been so extensively studied that researchers broadly understand how to tweak them, the genetics of others are less well understood.

Most of the IGIs initial research on photosynthesis and root systems will focus on rice, Ringeisen says. At the same time, the institute will also work on developing better gene-editing techniques for sorghum, a staple crop that has been particularly tough for researchers to crack. The team hopes to eventually understand and potentially alter soil microbes as well.

This is not easy, but were embracing the complexity, Ringeisen says. Ultimately, he hopes that when it comes to climate change, plants and microbes and agriculture can actually be part of the solution, rather than part of the problem.

View original post here:
This CRISPR pioneer wants to capture more carbon with crops - MIT Technology Review

Shares of CRISPR Therapeutics fell more than 11% on Monday as investors reacted negatively to the endorsement of a rival beta-thalassemia gene therapy developed by bluebird bio.

Last week, the U.S. Food and Drug Administrations Cell, Tissue and Gene Therapies Advisory Committee unanimously supported bluebirds beti-cel, a one-time gene therapy for patients with transfusion-dependent beta-thalassemia, a rare, inherited blood disorder caused by a genetic defect in hemoglobin.

Beti-cel, also known as betibeglogene autotemcel, is marketed in Europe as Zynteglo. Late-stage clinical data showed that 89% of patients who could be evaluated achieved transfusion independence following treatment with beti-cel, and safety data has been positive. The FDA is expected to give its final verdict on beti-cel by Aug. 19.

One day after the advisory committee endorsed beti-cel for beta-thalassemia, CRISPR Therapeutics and its partner Vertex Pharmaceuticalsreleasedpositive data for their gene therapy candidate, exa-cel. Exa-cel is a CRISPR-Cas9-based gene editing therapy for both transfusion-dependent beta-thalassemia (TDT) and severe sickle cell disease (SCD).

Data shared by the companies showed that 42 of 44 patients with TDT who received exa-cel have remained transfusion free for up to 37.2 months. The two patients who were not transfusion free had 75% and 89% reductions in transfusion volume, the companies said.

In SCD, the data was also positive. All 31 patients with sickle cell disease that is characterized by recurrent vaso-occlusive crises (VOCs) were free of the events following treatment with exa-cel. Data showed the patients had a duration of up to 32.3 months, CRISPR and Vertex reported, which expanded their partnership in this space last year.

Carmen Bozie, head of global medicines development and medical affairs at Vertex, touted the data. Bozie noted that of the 75 patients treated with exa-cel, 33 have one year or more of follow-up after infusion with the gene therapy. The data demonstrate the potential of exa-cel as a one-time functional cure for patients with transfusion-dependent beta-thalassemia or severe sickle cell disease, she said in a statement.

While bluebirds beti-cel was largely free of serious adverse events, Vertex and CRISPR reported that two of the 44 TDT patients experienced an SAE. One of the patients experienced three serious events that were connected to exa-cel, as well as busulfan, which was administered along with the gene therapy. That patient experienced hemophagocytic lymphohistiocytosis (HLH), a life-threatening condition related to excessive immune response, as well as acute respiratory distress syndrome and headache. The other patient experienced idiopathic pneumonia syndrome that was considered related to both exa-cel and busulfan, the companies said.

Among the 31 patients with SCD, there were no SAEs considered related to exa-cel.

CRISPR and Vertex are not alone in chasing bluebird bio to market with a gene therapy for beta-thalassemia. Editas Medicine is also developing its own gene therapy for the debilitating disease.

Earlier this year, Editas wonRare Pediatric Disease designationfor its experimental beta-thalassemia gene therapy, EDIT-301. The therapeutic is designed to edit the HBG1/2 promoter to disrupt the binding site of BCL11a and ameliorate disease symptoms.

In May, EDIT-301 won Orphan Drug designation for the treatment of beta-thalassemia and SCD. Editas expects to initiate a Phase I/II study of EDIT-301 in patients with transfusion-dependent beta-thalassemia later this year.

Here is the original post:
CRISPR Tx Shares Fall as bluebird's Gene Therapy Soars - BioSpace

The Global CRISPR/Cas9 MarketReport provides in-depth analysis of emerging trends, market drivers, development opportunities, and market constraints that may have an impact on the market dynamics of the industry. Each market sector is examined in depth in the Market Research Intellect, including goods, applications, and a competitive analysis.

The report was created using three different reconnaissance systems. The first step requires conducting extensive primary and secondary research on a wide range of topics. Approvals, evaluations, and discoveries based on accurate data obtained by industry specialists are the next steps. The research derives an overall estimate of the market size using top-down methodologies. Finally, the research evaluates the market for a number of sections and subparts using information triangulation and market separation techniques.

The primary objective of the report is to educate business owners and assist them in making an astute investment in the market. The study highlights regional and sub-regional insights with corresponding factual and statistical analysis. The report includes first-hand, the latest data, which is obtained from the company website, annual reports, industry-recommended journals, and paid resources. The CRISPR/Cas9 report will facilitate business owners to comprehend the current trend of the market and make profitable decisions.

Market Leaders Profiled:

Report Analysis & Segments:

The CRISPR/Cas9 is segmented as per the type of product, application, and geography. All of the segments of the CRISPR/Cas9 are carefully analyzed based on their market share, CAGR, value and volume growth, and other important factors. We have also provided Porters Five Forces and PESTLE analysis for a deeper study of the CRISPR/Cas9.The report also constitutes recent development undertaken by key players in the market which includes new product launches, partnerships, mergers, acquisitions, and other latest developments.

Based on Product Type CRISPR/Cas9 is segmented into

Based on the Application CRISPR/Cas9 is segmented into

The report provides insights on the following pointers:

1 Market Penetration: Comprehensive information on the product portfolios of the top players in the CRISPR/Cas9.

2 Product Development/Innovation: Detailed insights on the upcoming technologies, R&D activities, and product launches in the market.

3 Competitive Assessment: In-depth assessment of the market strategies, and geographic and business segments of the leading players in the market.

4 Market Development: Comprehensive information about emerging markets. This report analyzes the market for various segments across geographies.

5 Market Diversification: Exhaustive information about new products, untapped geographies, recent developments, and investments in the CRISPR/Cas9.

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Various Analyses Covered:

Regional assessment of the CRISPR/Cas9 has been carried out over six key regions which include North America, Asia-pacific, Europe, Latin America, Middle East, and Africa. Moreover, the report also delivers deep insights on the ongoing research & development activities, revenue, innovative services, the actual status of demand and supply, and pricing strategy. In addition to this, this report also delivers details on consumption figures, export/import supply, and gross margin by region. In short, this report provides a valuable source of guidance and clear direction for the marketer and the part interested in the market.

North America(United States, Canada)Asia Pacific(China, Japan, India, South Korea, Australia, Indonesia, Others)Europe(Germany, France, United Kingdom, Italy, Spain, Russia, Others)Latin America(Brazil, Mexico, Others) The Middle East and Africa

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CRISPR/Cas9 Market Size And Forecast to 2028 |Caribou Biosciences, Integrated DNA Technologies (IDT), CRISPR Therapeutics, Merck, Mirus Bio Indian...

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

John Leonard

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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Gene Genies: Inside The Revolutionary Biotech That Can Edit DNA Inside Living Humans - Forbes

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

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

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

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

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

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

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

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

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

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

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

Source: Chart by author. Microsoft quarterly filing.

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

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

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

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

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

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

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3 Bargain Stocks You Can Buy Today and Hold Forever - The Motley Fool

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

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

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

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

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

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

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

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

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

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

The voting results reported below are final.

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

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

Proposal 2 - Approval of the Appropriation of Financial Results

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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Proposal 15 - Re-election of the Auditors

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

Proposal 16 - Transaction of Any Other Business

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

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Item 9.01 Financial Statements and Exhibits

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

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Edgar Online, source Glimpses

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

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

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

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

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

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

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

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

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

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

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

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

Dive Brief:

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

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

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

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

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

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

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

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Immatics and Editas join up to bring CRISPR to 'gamma delta' cell therapy - BioPharma Dive

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

Earnings and Valuation

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

Profitability

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

Analyst Recommendations

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

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

Insider & Institutional Ownership

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

Volatility & Risk

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

Summary

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

About CRISPR Therapeutics (Get Rating)

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

About Inhibrx (Get Rating)

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

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

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

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

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

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

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

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

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

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

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

About exagamglogene autotemcel (exa-cel)

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

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

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

About CLIMB111 and CLIMB121

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

About CLIMB-131

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

About CLIMB141 and CLIMB151

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

About the GeneEditing Process in These Trials

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

About the VertexCRISPR Collaboration

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

About Vertex

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

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

(VRTX-GEN)

Vertex Special Note Regarding Forward-Looking Statements

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

(CRSP-GEN)

About CRISPR Therapeutics

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

CRISPR Therapeutics Forward-Looking Statement

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

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Contacts

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

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

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

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

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

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

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

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