Category Archives: Genetic Engineering

In an effort to improve treatment for obsessive compulsive disorder (OCD), researchers headed by teams at Brown University, and Baylor College of Medicine, have for the first time recorded electrical signals in the human brain that are associated with ebbs and flows in OCD symptoms, over an extended period, while individuals went about daily living in their homes. The research could be an important step in making an emerging therapy called deep brain stimulation (DBS) responsive to everyday changes in OCD symptoms.

In addition to advancing DBS therapy for cases of severe and treatment resistant OCD, this study has the potential for improving our understanding of the underlying neurocircuitry of the disorder, said Wayne Goodman, PhD, at Baylor College of Medicine. This deepened understanding may allow us to identify new anatomic targets for treatment that may be amenable to novel interventions that are less invasive than DBS. Goodman is co-author of the researchers published paper in Nature Medicine, which is titled, Long-term ecological assessment of intracranial electrophysiology synchronized to behavioural markers in obsessive-compulsive disorder.

OCD causes recurring unwanted thoughts and repetitive behaviors, and is a leading cause of disability. The condition, which is often debilitating, may affect perhaps 2-3% of the worlds population, the authors noted. Up to 20-40% of cases dont respond to traditional drug or behavioral treatments. Approximately 10% of individuals fail to achieve benefit from any intervention.

Deep brain stimulation, a technique that involves delivering mild electrical pulses via small electrodes precisely placed in the brain, can be effective in treating more than 50% of patients for whom other therapies failed. Over half of patients with treatment-resistant OCD are responders to DBS targeted to the ventral capsule/ventral striatum (VC/VS) region, the researchers further noted. To date, however, the number of patients who have received DBS for OCD is still in the hundreds.

One limitation of DBS is that it is unable to adjust to moment-to-moment changes in OCD symptoms, which are impacted by the physical and social environment. But adaptive DBS which can adjust the intensity of stimulation in response to real-time signals recorded in the braincould be more effective than traditional DBS and reduce unwanted side effects.

OCD is a disorder in which symptom severity is highly variable over time and can be elicited by triggers in the environment, said David Borton, PhD, an associate professor of biomedical engineering at Brown University, a biomedical engineer at the US Department of Veterans Affairs Center for Neurorestoration and Neurotechnology and a senior author of the new research. A DBS system that can adjust stimulation intensity in response to symptoms may provide more relief and fewer side effects for patients. But in order to enable that technology, we must first identify the biomarkers in the brain associated with OCD symptoms, and that is what we are working to do in this study. As the authors noted, An electrophysiological biomarker of symptom state would enable aDBS for OCD and other psychiatric disorders, which may provide a better approach for treating fluctuations in symptom intensity.

The research, led by Nicole Provenza, a recent Brown biomedical engineering PhD graduate from Bortons laboratory, was a collaboration between Bortons research group, affiliated with Browns Carney Institute for Brain Science and School of Engineering; the research groups of Wayne Goodman PhD, and Sameer Sheth MD, PhD, at Baylor College of Medicine; and Jeff Cohn, PhD, from the University of Pittsburghs Department of Psychology and Intelligent Systems Program and Carnegie Mellon University.

For their study, Goodmans team recruited five participants with severe OCD who were eligible for DBS treatment. Sheth, lead neurosurgeon, implanted in each participant an investigational DBS device from Medtronic, which is capable of both delivering stimulation and recording native electrical brain signals. Using the sensing capabilities of the hardware, the team gathered brain-signal data from participants in both clinical settings and at home as they went about daily activities. The DBS implants used in our study allow for real-time frequency-domain analysis of electrophysiological activity recorded simultaneously during stimulation delivery from the implanted electrodes, they wrote.

Along with the brain signal data, the team also collected a suite of behavioral biomarkers. In the clinical setting, these included facial expression (automatic facial affect recognition; AFAR) and body movement. Using computer vision and machine learning, they discovered that the behavioral features were associated with changes in internal brain states. At the participants homes, the team measured self-reports of OCD symptom intensity as well as biometric dataheart rate and general activity levelsrecorded by a smart watch and paired smartphone application, provided by Rune Labs. All of those behavioral measures were then time-synched to the brain-sensing data, enabling the researchers to look for correlations between the two.

Here, we acquired electrophysiological data with behavioral readouts over both short and long timescales, the team commented. In the clinic, we examined changes in affect (AFAR) during DBS parameter changes over short timescales (seconds to minutes). At home during participant-controlled recordings, we captured behavioral changes (self-reported OCD symptoms) over longer timescales (days to weeks to months) in natural settings, collected continuous data during natural and planned exposures, and developed methods to synchronize behavioral metrics to intracranial electrophysiology.

This is the first time brain signals from participants with neuropsychiatric illness have been recorded chronically at home alongside relevant behavioral measures, Provenza said. Using these brain signals, we may be able to differentiate between when someone is experiencing OCD symptoms, and when they are not, and this technique made it possible to record this diversity of behavior and brain activity.

Provenzas analysis of the data showed that the strategy did pick out brain-signal patterns potentially linked to OCD symptom fluctuation. While more work needs to be done across a larger cohort, this initial study shows that this technique is a promising way forward in confirming candidate biomarkers of OCD. we demonstrated the utility of at-home data collection for biomarker identification by observing correlations between spectral power and self-reported OCD symptom intensity.

We were able to collect a far richer dataset than has been collected before, and we found some tantalizing trends that wed like to explore in a larger cohort of patients, Borton said. Now we know that we have the toolset to nail down control signals that could be used to adjust stimulation level according to peoples symptoms.

Once those biomarkers are positively identified, they could then be used in an adaptive DBS system. Currently, DBS systems employ a constant level of stimulation, which can be adjusted by a clinician at clinical visits. Adaptive DBS systems, in contrast, would stimulate and record brain activity and behavior continuously without the need to attend clinic. When the system detects signals associated with an increase in symptom severity, it could ramp up stimulation to potentially provide additional relief. Likewise, stimulation could be toned down when symptoms abate. Such a system could potentially improve DBS therapy while reducing side effects.

Work on this line of research is ongoing. Because OCD is a complex disorder than manifests itself in highly variable ways across patients, the team hopes to expand the number of participants to capture more of that variability. They seek to identify a fuller set of OCD biomarkers that could be used to guide adaptive DBS systems. Once those biomarkers are in place, the team hopes to work with device makers to implement their DBS devices.

Our goal is to understand what those brain recordings are telling us and to train the device to recognize certain patterns associated with specific symptoms, Sheth said. The better we understand the neural signatures of health and disease, the greater our chances of using DBS to successfully treat challenging brain disorders like OCD. As the authors concluded, This work demonstrates the feasibility and utility of capturing chronic intracranial electrophysiology during daily symptom fluctuations to enable neural biomarker identification, a prerequisite for future development of adaptive DBS for OCD and other psychiatric disorders, the author concluded. The platform presented here lays the groundwork for future transformational studies reliant on ecological neural and behavioral monitoring and assessment of neuropsychiatric illness.

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Electrical and Behavioral Signals in OCD Could Guide Adaptive Therapy - Genetic Engineering & Biotechnology News

Gene therapy is still in its infancy and has yet to achieve its full potential. First-generation gene therapies have been challenged by safety issues due to their inability to target cells and organs without also penetrating non-targeted cells and organs, especially the liver. Capsida's proprietary, targeted, non-invasive gene therapy technology allows more selective targeting of specific tissues and cells, overcoming many of the problems associated with first-generation gene therapies, specifically off-target cell and organ activity. In addition, it allows the gene therapy to be delivered non-invasively through intravenous (IV) administration. The company's already strong leadership team is poised to actualize the promise of gene therapy with the addition of Mr. Anastasiou and the promotions of Drs. Flytzanis and Goeden.

"I can't imagine a more exciting time to join this organization," said Mr. Anastasiou. "Capsida is enabling gene therapy to become what the industry, physicians, and patients have been dreaming it will be. Our patent-protected technology allows the targeting of cells and organs while limiting the negative impact on non-targeted areas, and can be applied across multiple therapeutic areas. Another important benefit of our technology is that we are able to deliver the gene therapy non-invasively through IV administration. I'm honored to lead this talented team to achieve Capsida's potential and to improve and even save patients' lives."

Mr. Anastasiou joins Capsida from Lundbeck, where he was an executive vice president and a member of the executive committee, reporting to the CEO. As the president of Lundbeck's U.S. and Canadian business operations, Mr. Anastasiou has built organizations from the ground up. He brings significant leadership experience managing diverse organizations and bringing them together to achieve common goals. He led as many as 1,200 employees and achieved net revenues of $1.5 billion. During his 12-year tenure at Lundbeck, Mr. Anastasiou held several progressive leadership positions, playing a pivotal role in developing and launching multiple products and building the company's cross-functional capabilities. Mr. Anastasiou serves on the Board of PhRMA and the global advisory board for the Healthcare Businesswomen's Association. Mr. Anastasiou begins his new role with Capsida on January 3, 2022.

"We're thrilled to welcome Peter as Capsida's new CEO," said Beth Seidenberg, M.D., founding managing director at Westlake Village BioPartners, one of the company's lead investors, and Capsida board member. "Peter has deep industry expertise, a broad network, and significant public company experience, which will be valuable as Capsida grows. In addition, his strong track record of success demonstrates he is a visionary leader who will be able to deliver on the promise of targeted non-invasive gene therapy to help underserved patients and achieve business success."

"During his tenure at Lundbeck, Peter has created significant shareholder value, creating and leading organizations and successful blockbuster product launches," said Clare Ozawa, Ph.D., managing director at Versant Ventures, one of Capsida's lead investors, and Capsida board member."Under Peter's leadership, we will continue to build Capsida as the industry's leading targeted, non-invasive gene therapy company with the ability to transform the lives of patients with life-threatening genetic disorders."

Prior to Lundbeck, Mr. Anastasiou held management roles at Neuronetics, Inc., Bristol-Meyers Squibb Company, and Eli Lilly and Company. He holds an MBA from the Kelley School of Business at Indiana University, and a B.A. in economics and management from Albion College.

Capsida co-founders Nicholas Flytzanis, Ph.D., promoted to CSO and Nick Goeden, Ph.D., promoted to CTO

In addition to Mr. Anastasiou's appointment, Capsida announced that Dr. Flytzanis has been promoted toCSO and Dr. Goeden has been promoted to CTO.

"The promotions of Drs. Flytzanis and Goeden are in recognition of the significant contributions they have made since co-foundingCapsida in 2019," said Mr. Anastasiou. "Their steadfast commitment to delivering on the promise of Capsida's differentiated, non-invasive gene therapy platform has been a key driver behind many of the company's early achievements."

"Drs. Flytzanis' and Goeden's strong scientific and technical expertise and know-how have already delivered results in the startup of Capsida based on Caltech'sbasic research on targeted non-invasive gene delivery to the brain," said Capsida co-founder Viviana Gradinaru, Ph.D. "Their promotions are timely as Capsida enters the phase of delivering from the lab and for the patients."

Prior to co-founding Capsida, Dr. Flytzanis served as scientific director of the CLOVER research center at the California Institute of Technology (Caltech), leading an interdisciplinary team to develop and disseminate emerging technologies focused on the cross-section of neurological research and gene therapy. His research spans the fields of tissue clearing and imaging, optogenetics and rodent behavior, and adeno-associated virus (AAV) engineering and gene therapy, with collaborations across multiple institutions. During his Ph.D., Dr. Flytzanis applied protein engineering and directed evolution across biological modalities, with a focus on developing AAVs as therapeutic tools for neurological disease.

Dr. Flytzanis holds a Ph.D. in biology from Caltech and a B.S. in biology from the Massachusetts Institute of Technology.

Prior to co-founding Capsida, Dr. Goeden led a team developing the novel adeno-associated virus (AAV) engineering technology underlying Capsida's biologically driven gene therapy platform. During his tenure as a postdoctoral fellow in Dr. Gradinaru's lab at Caltech, he developed high-throughput methods for screening combinatorial libraries to explore the AAV fitness landscape and engineered novel AAVs with high efficiency and specificity for the rodent and primate brain. During his Ph.D., Dr. Goeden developed a novel organ bioreactor to study real-time metabolomics in diseased states, exploring the relationship between gene expression and the pathophysiology of neurodevelopmental disorders.

Dr. Goeden holds a Ph.D. in neuroscience from The University of Southern California and a B.S. in biology from Caltech.

About Capsida Biotherapeutics

Capsida Biotherapeutics Inc. is an industry-leading gene therapy platform company creating a new class of targeted, non-invasive gene therapies for patients with debilitating and life-threatening genetic disorders. Capsida's technology allows for the targeted penetration of cells and organs, while limiting collateral impact on non-targeted cells and organs, especially the liver. This technology allows for the delivery of the gene therapy in a non-invasive way through intravenous administration. Capsida's technology is protected by a growing intellectual property portfolio which includes more than 30 patent applications and one issued U.S. patent 11,149,256. The company is exploring using the technology across a broad range of life-threatening genetic disorders. Its initial pipeline consists of multiple neurologic disease programs. The company has strategic collaborations with AbbVie and CRISPR, which provide independent validation of Capsida's technology and capabilities. Capsida is a multi-functional and fully integrated biotechnology company with proprietary adeno-associated virus (AAV) engineering, multi-modality cargo development and optimization, translational biology, process development and state-of-the-art manufacturing, and broad clinical development experience. Capsida's biologically driven, high-throughput AAV engineering and cargo optimization platform originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at the California Institute of Technology. Visit us at http://www.capsida.com to learn more.

SOURCE Capsida Biotherapeutics

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Capsida Biotherapeutics Poised to Capitalize on Industry-leading Gene Therapy Technology With New CEO, CSO, and CTO - PRNewswire

EMERYVILLE, Calif.--(BUSINESS WIRE)--Metagenomi, a genetic medicines company with a versatile portfolio of next-generation gene editing tools, today announced that the company will share data related to their novel, compact, and hypo-immune gene editing systems at the 63rd Annual Meeting and Exposition of the American Society of Hematology (ASH), which is taking place in Atlanta, GA and virtually, December 1114.

The development of CAR T therapies and other genetically engineered cell therapies in recent years has resulted in significant benefits for patients, yet there remains a large unmet need for gene editing systems that can be used to develop novel immunotherapy approaches to treat blood cancers, said Brian C. Thomas, PhD, CEO and Co-Founder of Metagenomi. At ASH, we are presenting data on our novel nucleases that display highly efficient and specific gene editing both in vivo and ex vivo and hold significant potential to drive the development of new and efficacious therapies for patients.

In a poster titled A Novel Type V CRISPR System with Potential for Genome Editing in the Liver, it is shown that Metagenomis novel Type V CRISPR-associated nuclease was highly active in the liver of mice when systemically administered via lipid nanoparticles (LNP). The nuclease was derived from a unique natural environment and is phylogenetically distinct from previously identified Type V systems. Moreover, no antibodies to the nuclease were detected in serum from 50 healthy human donors, while between one third and half of the same serum samples contained antibodies that bind to spCas9, which is derived from a Streptococcus bacteria that commonly infects humans. In summary, this novel Type V CRISPR-associated nuclease is a promising new gene editing system for in vivo editing of the liver.

In a separate poster titled Novel CRISPR-Associated Gene Editing Systems Discovered in Metagenomic Samples Enable Efficient and Specific Genomic Engineering for Cell Therapy Development, three novel gene editing systems were used to make reproducible and efficient edits to human immune cells, demonstrating utility for the next generation of cell therapy development for blood cancers. Metagenomis novel gene editing systems were used to disrupt the T cell receptor alpha-chain constant region and the T cell receptor beta-chain constant region in approximately 90 percent of cells. Beta-2 microglobulin was edited in 95 percent of T cells. A chimeric antigen receptor (CAR) construct was also shown to be integrated in up to 60 percent of T cells. Novel gene editing systems were deployed in NK cells to disrupt CD38 a cell surface immune modulator that can be targeted in the development of cancer immunotherapy and to integrate a CAR construct that led to robust CAR-directed cellular cytotoxicity. B cell editing occurred in approximately 80% of target cells with successful transgene integration. Whats more, as these gene editing systems are taken from environmental samples as opposed to human pathogens, pre-existing immunity is expected to be rare. In summary, these novel systems were shown to result in highly efficient and specific gene edits in human immune cells and display the potential for use in cell therapy development.

Details of the presentations are below:

Presentation Title: A Novel Type V CRISPR System with Potential for Genome Editing in the LiverSession Title: 801. Gene Therapies: Poster IPresenting Author: Morayma Temoche-Diaz, PhDPublication Number: 1862 Session Time: Saturday, December 11, 5:30 p.m. ET

Presentation Title: Novel CRISPR-Associated Gene-Editing Systems Discovered in Metagenomic Samples Enable Efficient and Specific Genome Engineering for Cell Therapy DevelopmentSession Title: 801. Gene Therapies: Poster IIIPresenting Author: Gregory Cost, PhD, Vice President of Biology, MetagenomiPublication Number: 3984 Session Time: Monday, December 13, 6:00 8:00 p.m. ET

About Metagenomi

Metagenomi is a gene editing company committed to developing potentially curative therapeutics by leveraging a proprietary toolbox of next-generation gene editing systems to accurately edit DNA where current technologies cannot. Our metagenomics-powered discovery platform and analytical expertise reveal novel cellular machinery sourced from otherwise unknown organisms. We adapt and forge these naturally evolved systems into powerful gene editing systems that are ultra-small, extremely efficient, highly specific and have a decreased risk of immune response. These systems fuel our pipeline of novel medicines and can be leveraged by partners. Our goal is to revolutionize gene editing for the benefit of patients around the world. For more information, please visit https://metagenomi.co/.

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Metagenomi to Present Preclinical In Vivo and Ex Vivo Gene-Editing Data at the 63rd American Society of Hematology (ASH) Annual Meeting - Business...

Erika Moore studies lupus using a unique biomaterial that she developed.

Erika Moore, PhD

Long before she took the helm of her engineering lab as an assistant professor of materials science and engineering at the University of Florida, Erika Moore was fascinated by the careful dance between immune cells and biomaterials engineered to interact with living systems. Now, her curiosity has led her to design biomaterials to help diverse lupus patients receive personalized care.

How did you first realize that biomaterials could be useful for studying the immune system?

I had to start wearing contacts when I was 11 years old, and as I put something into my eye every day that was foreign to my body, it really piqued my interest in understanding biomaterials. While doing my undergraduate degree at Johns Hopkins University, I started to realize that immune cells are probably going to be an important part of how our bodies respond to biomaterials.

My area of expertise is at the meeting point of immunology, biomaterials science, and translational medicine. I was classically trained as a biomedical engineer, so the combination of engineering and medicine is just ingrained into me. Then I was drawn to creating new materials and using those materials to study immunology, but I'm not a classically trained immunologist. Biomedical engineers have been designing materials and putting them into rodents to validate them, but we haven't always considered how the immune system responds to these materials. During my PhD studies at Duke University, I focused on macrophages and understanding their contribution to biomaterials.

How did you connect this to studying autoimmunity and lupus specifically?

My personal connection to systemic lupus erythematosus (SLE), the autoimmune disease that I study, is that I identify as a woman of color, as a Black woman. SLE is a hugely sex-differential disease. About 90% of the people with SLE are women. If you break down that 90%, about 70% of that 90% are women of color. I actually have many friends with lupus; there are women in my life who have died from lupus, and I never realized how much of a health inequity and health disparity it was. When I realized that there was this autoimmune disease that disproportionately affects communities that I identify with, I wanted to use my privilege and education to try to improve outcomes for that disease.

Erika Moore developed a jello-like tissue system to study how immune cells stimulate tissues and blood vessel growth.

Erika Moore, PhD

That brings us back to the science. If we want to study something in the clinic, we have to enroll patients. But that takes a lot of time, effort, and energy. So, during graduate school, I wanted to create a tissue model outside the body that would recreate or mimic some of the interactions that occur in the body. The 3-D model system that I created then is a little bit squishylike jellomimicking some soft tissues in the body. You can take images of it, destroy it, and create it again. I used this system in graduate school to understand how macrophages help us build new tissues and blood vessels.

If we introduce other variables, we can study tissue responses in specific human contexts. For example, women with lupus usually have super inflammatory immune cells, including a lot of macrophage activation. If we take immune cells from healthy controls or patients with lupus and study them in our biomaterial models, what differences do we observe in those cells? And what does that tell us about what might be happening clinically?

The promise of our model system is that we can have personalized medicine approaches in our mini-jello tissues that we create in the lab. Maybe in the future, we can take some patient cells, put them in our system, and then introduce a drug to see how they respond. Does it get better? Does it get worse? That's better than just putting volunteers on a drug without any prior knowledge or background.

How do you consider the variation in lupus between populations?

I want to understand the contribution of ancestry to disease pathology and disease progression. We use markers of genetic ancestry to understand how different patients respond. We also use socio-cultural, educational, and financial markers because we understand that ancestry doesn't always capture lived experience. We know that environment and geneticsnature and nurturecombine to inform biology and cell function. We can declare those variables and use them to determine which groups we encapsulate into our jello biomaterial tissue models, and then understand how their cells respond differently. For example, does the number of discrimination events you have seen inform how your cells respond?

What are the challenges for this kind of interdisciplinary work?

The hardest part so far has been knowledge. I am personally driven to study this autoimmune disease because of its disproportionate impact, so I really want to learn, and I'm still learning. Because my work is interdisciplinary, I have to master multiple fields. I'm really grateful for my collaborators who have been helpful in educating me to ask the right questions, because not all questions are worth answering immediately. You have to prioritize. I know a lot of people with lupus, and I ask, what's helpful? What's not working? It's a shared community effort. I am really sharing the voices of many people, not just myself.

This interview has been edited and condensed for clarity.

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An Engineers Perspective on Autoimmunity - The Scientist

DUBLIN--(BUSINESS WIRE)--The "CRISPR Technology Global Market Report 2021: COVID-19 Growth and Change to 2030" report has been added to ResearchAndMarkets.com's offering.

The global CRISPR technology market is expected to grow from $0.8 billion in 2020 to $0.95 billion in 2021 at a compound annual growth rate (CAGR) of 18.8%. The market is expected to reach $2.22 billion in 2025 at a CAGR of 24%.

The CRISPR technology market consists of sales of CRISPR technology products and services which is a gene-editing technology that allows researchers to alter DNA sequences and modify gene function. The revenue generated by the market includes the sales of products such as design tools, plasmid & vector, Cas9 & gRNA, libraries & delivery system products and services that include design & vector construction, screening and cell line engineering.

These products and services are used in genome editing/genetic engineering, genetically modifying organisms, agricultural biotechnology and others which include gRNA database/gene library, CRISPR plasmid, human stem cell & cell line engineering by end-users. The end-users include pharmaceutical & biopharmaceutical companies, biotechnology companies, academic & research institutes and contract research organizations.

The CRISPR technology market covered in this report is segmented by product type into design tools, plasmid and vector, CAS9 and G-RNA, delivery system products. It is also segmented by application into genome editing/ genetic engineering, genetically modified organisms, agricultural biotechnology, others and by end-user into industrial biotech, biological research, agricultural research, therapeutics and drug discovery.

Stringent government regulations are expected to retard the growth of the CRISPR technology market during the period. There is no existence of internationally agreed regulatory framework for gene editing products and countries are in the process of evaluating whether and to what extent current regulations are adequate for research conducted with gene editing and applications and products related to gene editing.

The Court of Justice of the European Union ruled that it would treat gene-edited crops as genetically modified organisms, subject to stringent regulation. In April 2019, the Australian government stated that the Office of the Gene Technology Regulator (OGTR) will regulate only the gene-editing technologies that use a template, or that insert other genetic material into the cell.

According to an article of 2020, in India, as per the National Guidelines for Stem Cell Research, genome modification including gene-editing by CRISPR-Cas9 technology of stem cells, germ-line stem cells or gamete and human embryos is restricted only to in-vitro studies. Thus, strict regulations by the government present a threat to the growth of the market.

The application of CRISPR technology as a diagnostic tool is expected to boost the market during the period. The Sherlock CRISPR SARS-CoV-2 kit is the first diagnostic kit based on CRISPR technology for infectious diseases caused due to COVID-19.

In May 2020, FDA announced the emergency use authorization to the Sherlock BioSciences Inc's Sherlock CRISPR SARS-CoV-2 kit which is a CRISPR-based SHERLOCK (Specific High-sensitivity Enzymatic Reporter unLOCKing) diagnostic test. This test helps in specifically targeting RNA or DNA sequences of the SARS-CoV-2 virus from specimens or samples such as nasal swabs from the upper respiratory tract and fluid in the lungs from bronchoalveolar lavage specimens.

This diagnostic kit has high specificity and sensitivity and does not provide false negative or positive results. Widening the application of CRISPR technology for the diagnosis of infectious diseases will increase the demand for CRISPR technology products and services.

Several advancements in CRISPR technology are trending in the market during the period. Advancements in technology will help in reducing errors, limiting unintended effects, improving the accuracy of the tool, widening its applications, developing gene therapies and more.

In 2019, a study published in Springer Nature stated the development of an advanced super-precise new CRISPR tool that allows researchers more control over DNA changes. This tool seems to have the capability of providing a wider variety of gene edits which might potentially open up conditions that have challenged gene-editors.

Also, in 2020, another study in Springer Nature stated that researchers have used enzyme engineering to boost the accuracy of the technique of error-prone CRISPR-Cas9 system to precisely target DNA without introducing as many unwanted mutations. The advancements in CRISPR technology will result in better tools that are capable of providing better outcomes.

Major players in the CRISPR technology market are

Key Topics Covered:

1. Executive Summary

2. CRISPR Technology Market Characteristics

3. CRISPR Technology Market Trends and Strategies

4. Impact Of COVID-19 On CRISPR Technology

5. CRISPR Technology Market Size and Growth

5.1. Global CRISPR Technology Historic Market, 2015-2020, $ Billion

5.1.1. Drivers Of the Market

5.1.2. Restraints On the Market

5.2. Global CRISPR Technology Forecast Market, 2020-2025F, 2030F, $ Billion

5.2.1. Drivers Of the Market

5.2.2. Restraints On the Market

6. CRISPR Technology Market Segmentation

6.1. Global CRISPR Technology Market, Segmentation by Product Type, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

6.2. Global CRISPR Technology Market, Segmentation by Application, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

6.3. Global CRISPR Technology Market, Segmentation by End-User, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

7. CRISPR Technology Market Regional and Country Analysis

7.1. Global CRISPR Technology Market, Split by Region, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

7.2. Global CRISPR Technology Market, Split by Country, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

For more information about this report visit https://www.researchandmarkets.com/r/gvqw3u

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Global CRISPR Technology Market Research Report 2021: Focus on Design Tools, Plasmid and Vector, Cas9 and G-RNA, & Delivery System Products -...

Gene therapy is a powerful developing technology that has the potential to address myriad diseases. For example, Huntington's disease, a neurodegenerative disorder, is caused by a mutation in a single gene, and if researchers could go into specific cells and correct that defect, theoretically those cells could regain normal function.

A major challenge, however, has been creating the right "delivery vehicles" that can carry genes and molecules into the cells that need treatment, while avoiding the cells that do not.

Now, a team led by Caltech researchers has developed a gene-delivery system that can specifically target brain cells while avoiding the liver. This is important because a gene therapy intended to treat a disorder in the brain, for example, could also have the side effect of creating a toxic immune response in the liver, hence the desire to find delivery vehicles that only go to their intended target. The findings were shown in both mouse and marmoset models, an important step towards translating the technology into humans.

A paper describing the new findings appears in the journalNature Neuroscience. The research was led byViviana Gradinaru(BS '05), professor of neuroscience and biological engineering, and director of theCenter for Molecular and Cellular Neuroscience.

The key to this technology is the use of adeno-associated viruses, or AAVs, which have long been considered promising candidates for use as delivery vehicles. Over millions of years of evolution, viruses have evolved efficient ways to gain access into human cells, and for decades researchers have been developing methods to harness viruses' Trojan-Horse-like abilities for human benefit.

AAVs are made up of two major components: an outer shell, called a capsid, that is built from proteins; and the genetic material encased inside the capsid. To use recombinant AAVs for gene therapy, researchers remove the virus's genetic material from the capsid and replace it with the desired cargo, such as a particular gene or coding information for small therapeutic molecules.

"Recombinant AAVs are stripped of the ability to replicate, which leaves a powerful tool that is biologically designed to gain entrance into cells," says graduate student David Goertsen, a co-first author on the paper. "We can harness that natural biology to derive specialized tools for neuroscience research and gene therapy."

The shape and composition of the capsid is a critical part of how the AAV enters into a cell. Researchers in the Gradinaru lab have been working for almost a decade onengineering AAV capsids that cross the blood-brain barrier (BBB)and todevelop methods to select for and against certain traits, resulting in viral vectors more specific to certain cell types within the brain.

In the new study, the team developed BBB-crossing capsids, with one in particular AAV.CAP-B10that is efficient at getting into brain cells, specifically neurons, while avoiding many systemic targets, including liver cells. Importantly, both neuronal specificity and decreased liver targeting was shown to occur not just in mice, a common research animal, but also in laboratory marmosets.

"With these new capsids, the research community can now test multiple gene therapy strategies in rodents and marmosets and build up evidence necessary to take such strategies to the clinic," says Gradinaru. "The neuronal tropism and decreased liver targeting we were able to engineer AAV capsids for are important features that could lead to safer and more effective treatment options for brain disorders."

The development of an AAV capsid variant that works well in non-human primates is a major step towards the translation of the technology for use in humans, as previous variants of AAV capsids have been unsuccessful in non-human primates. The Gradinaru lab's systematicin vivoapproach, which uses a process called directed evolution to modify AAV capsids at multiple sites has been successful in producing variants that can cross the BBBs of different strains of mice and, as shown in this study, in marmosets.

"Results from this research show that introducing diversity at multiple locations on the AAV capsid surface can increase transgene expression efficiency and neuronal specificity," says Gradinaru. "The power of AAV engineering to confer novel tropisms and tissue specificity, as we show for the brain versus the liver, has broadened potential research and pre-clinical applications that could enable new therapeutic approaches for diseases of the brain."

Reference: Goertsen D, Flytzanis NC, Goeden N, et al. AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset. Nat Neuro. 2021. doi: 10.1038/s41593-021-00969-4

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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One Step Closer to Targeted Gene Therapy - Technology Networks

Like many of the technologies that are driving innovation in the alternative protein space, plant molecular farming has traditionally been used in the pharmaceutical industry. The practice which involves genetically editing a crop so that its cells produce a desired protein is being discussed as a way to rapidly produce proteins for COVID-19 vaccines.

In the food industry, molecular farming is one route to producing the animal proteins that give egg, dairy, and meat products their visual, taste, and functional properties. Molecular farming allows you to use the exact same protein that would normally be produced by a chicken or cow, without the need for any actual animals.

Moolec Science, a spinoff of Argentina-based agtech company Bioceres Crop Solutions, is probably the most prominent name in molecular farming for the food industry. Moolec already sells chymosin, a cheesemaking enzyme, which the company grows in safflower plants. Theyve also successfully grown meat proteins in soybean and pea plants.

The Moolec team believes that molecular farming can help to bring down the end costs of alternative meat products. (Theres nothing better than low-tech farming to produce at an enhanced scale and low cost, company CEO and co-founder Gastn Paladini told The Spoon back in October.) And they may be right.

Molecular farming can help producers to avoid some of the costly and tricky problems of growing proteins in traditional bioreactors. When you use a plant as your bioreactor, as food scientist and thought leader Tony Hunter pointed out in an article this year, you dont need to worry about maintaining sterile conditions: Plants have built-in immune systems.

Moolec plans to launch its first animal-free meat protein in late 2022 or early 2023. The company is currently working toward regulatory approval for its products and its progress will be an interesting test of regulatory tolerance of Moolecs brand of genetic engineering.

One potential concern for regulators as they scrutinize molecular farming processes will be the possibility of gene flow from modified crops to related plants. Tiamat Sciences, a Belgium-based molecular farming startup, is limiting that possibility by growing its crops in a contained vertical farming system.

Tiamat has plans to expand alongside the cell-based meat industry. By targeting nascent markets on the verge of scale-up, weve already demonstrated significant traction for our solutions and an early revenue potential that is outstanding for a biotech startup, said Tiamats founder and CEO France-Emmanuelle Adil in a recent press release. The company currently produces GRAS-certified, animal-free growth factors for cultivated meat, and also manufactures proteins for the pharmaceutical industry.

Last month, Tiamat announced that it had raised a $3 million seed funding round led by Silicon Valley venture capital firm True Ventures. The company is using those funds to construct a pilot facility in Durham, N.C. so we may see them boost their capacity in the year to come.

Molecular farming startups still have some issues to work out. As Tony Hunter noted in his piece on molecular farming, plant tissue has larger and fewer protein-producing cells compared to the same volume of mammal tissue, making plants less productive as protein factories. And there are costs associated with extracting protein molecules from plants at the cellular level.

Still, the same upsides of molecular farming that make it attractive to the pharmaceutical industry will likely continue to spark interest from alternative protein producers especially as those producers seek ways to bring down the retail prices of their products.

Related

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In 2022, Molecular Farming Startups Will Move Toward Commercialization of Animal-Free Proteins - The Spoon

Dublin, Dec. 06, 2021 (GLOBE NEWSWIRE) -- The "Rat And Mouse Model Market Size, Share & Trends Analysis Report By Type (Knockout, Knock-in), By Technology, By Service (Breeding, Rederivation), By Application (COVID-19, CVS), By End-use, And Segment Forecasts, 2021 - 2028" report has been added to ResearchAndMarkets.com's offering.

The global rat and mouse model market size is anticipated to reach USD 2.9 billion by 2028, according to a new report by the publisher. The market is expected to expand at a CAGR of 5.5% from 2021 to 2028.

Recent advancements in the field of genetic engineering including piggyBac technologies, Cas-CLOVER, and CRISPR/Cas9 facilitate researchers to develop immunodeficient rat models which can be used for human cell/tissue regeneration and transplants.

Key companies have leveraged advanced technologies such as machine learning, big data analytics, genome engineering tools, and next-generation sequencing technology to develop mouse and rat models with more human-like microbiomes, immune systems, and genetic backgrounds. For instance, models from the Jackson Laboratory reflect the genetic diversity showcased by the human population.

Market participants are focused to develop novel methods to certify that in vivo and in vitro testing complement each other. In this regard, an increase in the adoption rate of organoid systems has been observed. These systems guide engineering as well as enhance the use and selection of animal models. Moreover, the rise in utilization of mouse model in the analysis of SARS-CoV-2 infection in upper and lower respiratory tract infections drives the global market.

Rat And Mouse Model Market Report Highlights

The outbred model system generated the largest revenue amongst model type as outbred strains are genetically diverse and feature well-characterized, stable genetic variation which accurately reflects the genetic structure of human populations

Therefore, outbred strains such as the Diversity Outbred (DO) mice are used to analyze the genetic complexity behind cancer, autoimmune disorders, addiction, obesity, heart disease, diabetes, and Alzheimer's disease

The CRISPR/Cas9 segment is anticipated to witness the fastest CAGR from 2021 to 2028 as CRISPR/Cas9 can target any definite exons for exclusion or inclusion within an mRNA

Additionally, the use of the CRISPR/Cas9 system to generate the knockout mouse models simplifies the whole process and reduces the timeframe from 1-2 years to 6 months

The cryopreservation segment is expected to gain significant traction during the forecast period

The technique provides a fast and reliable procedure for archiving sperm and embryos of valuable mice strains

Cancer is the largest revenue-generating application segment as the mouse and human genomes are homologous. Thus, it provides a good tool for cancer research as well as for drug discovery

Pharmaceutical and biotechnology companies dominated the end-use segment in terms of revenue share in 2020. Rat and mice models are some of the most used animal models in drug discovery

Techniques such as CRISPR/Cas9, homologous recombination, and random transgenesis help the biotech and pharma companies to analyze hypotheses or to generate rat models for human diseases

Genetically engineered models play a vital role to assess and characterize target identification, disease pathology, and in vivo assessment of new molecular entities

Profitable opportunities offered by the developing nations and have attracted investments from global companies in the Asia Pacific region

For instance, in July 2021, ERS Genomics Limited signed a non-exclusive license agreement with Japan SLC. This provided the latter access to CRISPR/Cas9 patent portfolio offered by ERS Genomics

As Japan SLC offers transgenic, hybrid, congenic, immunodeficient, inbred, and outbred animal models to research organizations; an addition of CRISPR/Cas9 technology will enhance its portfolio for rat and mouse models in Japan

Key Topics Covered:

Chapter 1 Methodology and Scope

Story continues

Chapter 2 Executive Summary

Chapter 3 Market Variables, Trends & Scope3.1 Market Segmentation3.2 Rat & Mouse Model Market Lineage Outlook3.2.1 Parent Market Outlook3.3 Penetration and Growth Prospect Mapping

Chapter 4 Industry Outlook4.1 Market Variable Analysis4.1.1 Rise in adoption of personalized medicine and subsequent demand of rat & mouse models for precision medicine approaches4.1.2 Application of genome editing tools for generation of rodent models4.1.3 Increase in the number of research activities involving the use of rat and mouse models4.1.4 Grants & investments4.2 Market Restraint Analysis4.2.1 Ethical issues pertaining to the use of animals for research4.2.2 Rise in adoption of Zebrafish models for biomedical research4.3 Market Opportunity Analysis4.3.1 Rise in demand for humanized rat & mouse models4.3.2 Utility of rat/mouse models to examine the pathogenesis of SARS-CoV-24.4 Market Challenge Analysis4.4.1 Alternatives to Animal Testing4.4.1.1 In Vitro Testing4.4.1.2 Computer (in Silico) Modeling4.4.2 Speculations regarding ban on animal testing

Chapter 5 Business Environment Analysis5.1 SWOT Analysis; By factor (Political & Legal, Economic and Technological)5.2 Porter's Five Forces Analysis

Chapter 6 Competitive Analysis6.1 Recent Developments & Impact Analysis, by Key Market Participants6.1.1 Strategy Framework6.1.2 Market Participant Categorization6.1.3 Major Deals & Strategic Alliances Analysis6.2 Mergers and acquisitions6.3 Partnerships6.4 Agreements6.5 Synergies, Collaborations, and other initiatives6.5.1 Market Entry Strategies

Chapter 7 Type Business Analysis7.1 Rat & Mouse Model Market: Type Movement Analysis7.2 Knockout7.2.1 Global Rat and mouse model Market for Knockout estimates And Forecast, 2018 - 2028 (USD Million)7.3 Knock-in7.3.1 Global Rat and mouse model Market for Knock-in estimates And Forecast, 2018 - 2028 (USD Million)7.4 Outbred7.4.1 Global Rat and mouse model Market for Outbred estimates And Forecast, 2018 - 2028 (USD Million)7.5 Inbred7.5.1 Global Rat and mouse model Market for Inbred estimates And Forecast, 2018 - 2028 (USD Million)7.6 Others7.6.1 Global Rat and mouse model Market for Others estimates And Forecast, 2018 - 2028 (USD Million)

Chapter 8 Technology Business Analysis8.1 Rat & Mouse Model Market: Technology Movement Analysis8.2 Nuclear transferase8.2.1 Global Rat & Mouse Model Market FOR Nuclear transferase estimates And Forecast, 2018 - 2028 (USD Million)8.3 Microinjection8.3.1 Global Rat & Mouse Model Market FOR Microinjection estimates And Forecast, 2018 - 2028 (USD Million)8.4 Embryonic stem cell8.4.1 Global Rat & Mouse Model Market FOR Embryonic stem cell estimates And Forecast,2018 - 2028 (USD Million)8.5 CRISPR/Cas98.5.1 Global Rat & Mouse Model Market FOR CRISPR/Cas9 estimates And Forecast, 2018 - 2028 (USD Million)8.6 Other Technologies8.6.1 Global Rat & Mouse Model Market FOR Others estimates And Forecast, 2018 - 2028 (USD Million)

Chapter 9 Service Business Analysis9.1 Rat & Mouse Model Market: Service Movement Analysis9.2 Cryopreservation9.2.1 Global Rat & Mouse Model Market FOR Cryopreservation estimates And Forecast, 2018 - 20289.3 Breeding9.3.1 Global Rat & Mouse Model Market for Breeding estimates And Forecast, 2018 - 20289.4 Rederivation9.4.1 Global Rat & Mouse Model Market for Rederivation estimates And Forecast, 2018 - 20289.5 Genetic testing9.5.1 Global Rat & Mouse Model Market for Genetic testing estimates And Forecast, 2018 - 20289.6 Quarantine depending9.6.1 Global Rat & Mouse Model Market for Quarantine depending estimates And Forecast, 2018 - 20289.7 Others9.7.1 Global Rat & Mouse Model Market for Others estimates And Forecast, 2018 - 2028

Chapter 10 Application Business Analysis10.1 Rat & Mouse Model Market: Application Movement Analysis10.2 Cardiovascular diseases10.2.1 Global Rat & Mouse Model Market FOR Cardiovascular diseases Market estimates And Forecast, 2018 - 202810.3 Genetic diseases10.3.1 Global Rat & Mouse Model Market FOR Genetic diseases Market estimates And Forecast, 2018 - 202810.4 Cancer10.4.1 Global Rat & Mouse Model Market FOR Cancer estimates And Forecast, 2018 - 2028 (USD Million)10.5 Infectious diseases10.5.1 Global Rat & Mouse Model Market FOR Infectious diseases estimates And Forecast, 2018 - 2028 (USD Million)10.5.2 COVID-1910.5.2.1 Global Rat & Mouse Model Market FOR COVID-19 estimates And Forecast, 2018 - 2028 (USD Million)10.5.3 Others10.5.3.1 Global Rat & Mouse Model Market FOR Others estimates And Forecast, 2018 - 2028 (USD Million)10.6 Transplantation10.6.1 Global Rat & Mouse Model Market FOR Transplantation estimates And Forecast, 2018 - 2028 (USD Million)10.7 Toxicology studies10.7.1 Global Rat & Mouse Model Market FOR Toxicology studies estimates And Forecast, 2018 - 2028 (USD Million)10.8 Others10.8.1 Global Rat & Mouse Model Market FOR Others estimates And Forecast, 2018 - 2028 (USD Million)

Chapter 11 End-use Business Analysis11.1 Rat & Mouse Model Market: End Use Movement Analysis11.2 Pharmaceutical & Biotechnology Companies11.2.1 Global Rat & Mouse Model Market for Pharmaceutical & Biotechnology companies estimates And Forecast, 2018 - 2028 (USD Million)11.3 Academic and research facilities11.3.1 Global Rat & Mouse Model Market for Academic and research facilities estimates And Forecast, 2018 - 2028 (USD Million)11.4 Contract Research & Manufacturing Organizations11.4.1 Global Rat & Mouse Model Market for Contract research & manufacturing organizations estimates And Forecast, 2018 - 2028 (USD Million)

Chapter 12 Regional Business Analysis

Chapter 13 Company Profile13.1 Charles River Laboratories, Inc.13.1.1 Company overview13.1.2 Financial Performance13.1.3 Product benchmarking13.1.4 Strategic initiatives13.2 The Jackson Laboratory.13.2.1 Company overview13.2.2 Financial Performance13.2.3 Product benchmarking13.2.4 Strategic initiatives13.3 Laboratory Corporation of America13.3.1 Company overview13.3.2 Financial Performance13.3.3 Product benchmarking13.3.4 Strategic initiatives13.4 Perkin Elmer (Horizon Discovery Group plc)13.4.1 Company overview13.4.2 Financial Performance13.4.3 Product benchmarking13.4.4 Strategic initiatives13.5 genOway13.5.1 Company overview13.5.2 Financial Performance13.5.3 Product benchmarking13.5.4 Strategic initiatives13.6 Envigo13.6.1 Company overview13.6.2 Financial Performance13.6.3 Product benchmarking13.6.4 Strategic initiatives13.7 Janvier Labs13.7.1 Company overview13.7.2 Financial Performance13.7.3 Product benchmarking13.7.4 Strategic initiatives13.8 Taconic Biosciences, Inc.13.8.1 Company overview13.8.2 Financial Performance13.8.3 Product benchmarking13.8.4 Strategic initiatives13.9 Biomere (Biomedical Research Models, Inc.)13.9.1 Company overview13.9.2 Financial Performance13.9.3 Product benchmarking13.9.4 Strategic initiatives13.10 Transposagen Biopharmaceuticals, Inc.13.10.1 Company overview13.10.2 Financial Performance13.10.3 Product benchmarking13.10.4 Strategic initiatives

For more information about this report visit https://www.researchandmarkets.com/r/720b37

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Worldwide Rat and Mouse Model Industry to 2028 - Utility of Rat/Mouse Models to Examine the Pathogenesis of SARS-CoV-2 Presents Opportunities - Yahoo...

The global genome editing market is expected to reach US$ 10,691.0 million by 2025 from US$ 3,210.1 million in 2017; it is estimated to grow at a CAGR of 17.0% from 2018 to 2027.

According The Insight Partners study on Genome Editing Market Forecast to 2027 COVID-19 Impact and Global Analysis by Technology, Application, End User, The report highlights trends existing in the market, and drivers and hindrances pertaining to the market growth. Factors such as Increase in funding for the genome editing, rising prevalence of the genetic disorders, rise in the advancements for genome editing technology and rise in the production of genetically modified crops are the driving factors for the growth of the market.

Genome editing is a technique that is utilized for the changes that are to be done in the DNA of a cell or an organism. The technique involves cutting DNA sequences for the addition or removing the DNA in the genome. The changes in the genome are done for the required characteristics of the cell. Genome editing is done for the research purpose, the treatment of the diseases, and the biotechnological purpose.

Get Sample PDF Copy of Genome Editing Market at: https://www.theinsightpartners.com/sample/TIPHE100000853/

Market Insights

Increase in Funding for the Genome Editing

The market for genome editing is expected to grow in the coming near future due to the growth factor that is driving the market is the increase in the funding. The different government in the different regions are increasing their funds and grants to develop genome editing research. Owing to genome editings advantages, the various government is supporting their public and private research and academic institutes for increasing the research activities for the genome editing and genetic engineering.

Across the world, funding is being provided by every nation. However, the more funds, for instance, in January 2018 US government announced donating US$ 190 million for research for the next six years. Also, the government is hoping to develop therapies to treat cancer and other diseases using gene editing. In addition, the National Institutes of Health (NIH) has kept approximately US$ 45.5 million aside for the next four fiscal years for the Somatic Cell Genome Editing program. Moreover, in the Asia Pacific region, the countries are also investing more in the development of genome editing technology for two-three years back. For instance, in April 2016, Japan invested approximately US$76million for the five years for the creation of Japanese owned genome editing technologies.

Furthermore, the investments are made for private companies operating for genome editing. For instance, in August 2015, Editas Medicine is a company at the forefront of developing the gene-editing technology CRISPR has received US$ 120 million to create a new treatment for the conditions which include cancer, retinal diseases, and sickle cell anemia. Therefore, the rise in the funding for genome editing is likely to drive the market for genome editing in the forecast period. The rise in the funding will enhance the research and development of the gene-editing technologies and products for the researchers for efficient and effective genome editing. The funding will also enable the biopharmaceutical and pharmaceutical companies to develop technologies for the therapies using gene editing to treat and diagnose chronic diseases.

It also includes the impact of the COVID-19 pandemic on the market across all the regions. The Genome Editing Market , by region, is segmented into North America, Europe, Asia Pacific (APAC), Middle East and Africa (MEA), and South and Central America (SAM).

COVID-19 first began in Wuhan (China) during December 2019 and since then it has spread at a fast pace across the globe. The US, India, Brazil, Russia, France, the UK, Turkey, Italy, and Spain are some of the worst affected countries in terms confirmed cases and reported deaths. The COVID-19 has been affecting economies and industries in various countries due to lockdowns, travel bans, and business shutdowns.

Download the Latest COVID-19 Analysis on Genome Editing Market Growth Research Report at: https://www.theinsightpartners.com/covid-analysis-sample/TIPHE100000853

Based on technology, the genome editing market is segmented into transcription activator-like effector nucleases (TALENS), clustered regularly interspaced short palindromic repeats (CRISPR), zinc finger nucleases (ZFNs), antisense RNA and others. In 2017, the CRISPR segment held the largest share of the market, by technology owing to the applications and its benefits offered. The TALENs segment is expected to grow at the fastest rate during the coming years.

Based on application, the genome editing market is segmented into genetic engineering, cell line engineering and others. In 2017, cell line segment held the largest share of the market, by application. Moreover, the genetic engineering segment is expected to grow at the fastest rate during the coming years owing to its sub segments such as animal genetic engineering and plant genetic engineering that are being carried out extensively.

Based on end user, the genome editing market is segmented into biotechnology & pharmaceutical companies, contract research organizations, academic & government research organization and other end users. The market is dominated by the biotechnology & pharmaceutical companies and is expected to surge significantly during the forecast period from 2017 to 2025. The biotechnology & pharmaceutical companies segment is expected gain its market share during the forecast period. Also, biotech & pharmaceutical companies is expected to show a prime CAGR owing to the increasing government funding and partnerships between the various organizations in all the regions.

Genome Editing Market : Competitive Landscape and Key Developments

Transposagen Biopharmaceuticals, Inc.,Integrated DNA Technologies, Inc.,Thermo Fisher Scientific Inc.,GenScript,Lonza,Horizon Discovery Group plc,Sangamo Therapeutics, Inc.,New England Biolabs,Editas Medicine,Merck KGaA.

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The Insight Partners is a one stop industry research provider of actionable intelligence. We help our clients in getting solutions to their research requirements through our syndicated and consulting research services. We specialize in industries such as Semiconductor and Electronics, Aerospace and Defense, Automotive and Transportation, Biotechnology, Healthcare IT, Manufacturing and Construction, Medical Device, Technology, Media and Telecommunications, Chemicals and Materials.

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Genome Editing Market to hit US$ 10691.0 Million, Globally, by 2025 at 17.0% CAGR: The Insight Partners - Digital Journal

Epicentre

IT HAS now been nearly two years since the human species was presented with the conundrum of the origins of a pandemic disease that eventually came to kill more than five million people worldwide, with the numbers growing every day. There are always controversies about epidemics, but unlike with AIDS in its early days, few have claimed that a virus was not the precipitating cause of Covid-19 although there were many like former US president Donald Trump and his followers who downplayed its seriousness.

Instead, the main controversy has been how the pandemic virus originated. While this debate has been exceptionally rancorous, what is not generally recognised is how much agreement there is among the adversaries. Nearly everyone agrees that the virus, SARS-CoV2, is derived from a type of coronavirus that is endemic to, and tolerated by, bats, and that it emerged after a few genetic changes in the city of Wuhan, in the Huwei province of China.

Those changes made the virus particularly well suited to attaching to human cells that line the respiratory tract and blood vessels, and particularly pathogenic in some vulnerable subpopulations the old, the obese, and the diabetic. It is also unpredictably fatal in some individuals with no obvious predispositions. But these random strikes are rare, leaving ample opportunity for people to live in fear, or alternatively, to disdain those who do, depending on temperamental proclivities that under the current situation inevitably align with political allegiance.

So where and how did the last few steps occur that turned a virus which was innocuous in animals to one that is devastating in humans? Infectious diseases have frequently emerged from spillovers from wild or domesticated animals that have come into contact with humans in unnatural settings like farms and food markets, or degraded habitats. This has been the preferred explanation for emergent diseases by the scientific community, and since there is a food market in Wuhan that sells live animals, including some exotic species, it was readily taken up in an environment predisposed to negativity concerning China. But the Wuhan live food market was not selling bats. Moreover, there are few wild bats in Wuhan, and those harbouring viruses related to SARS-Cov2 live in Chinese caves hundreds of miles from Wuhan or in other regions of Southeast Asia, such as Laos. Further, how the virus evolved to be so well adapted to humans in bats or other intermediate species and wind up at the Wuhan market was a mystery considering that no SARS-Cov2 or related viruses have been detected in any wild animals in or around the market or elsewhere in Wuhan.

There are also several laboratories in Wuhan which work on bat viruses, including SARS-type coronaviruses. One of these is the only bio-safety level 4 lab in China, operating under internationally agreed standards for the most hazardous kinds of microbiological and virus research. Leaks of experimental viruses have often occurred from research labs throughout the world, and a few have caused infectious outbreaks. But the scientific establishment resists lab-leak scenarios since they raise questions about their capacity to conduct their activities safely and threaten to bring scientists under increased scrutiny and to impose additional controls on their work.

Conventional opinion in the United States was happy to go along with the claims of prominent scientists and scientific administrators such as the infectious disease specialist Anthony Fauci and National Institutes of Health director Francis Collins that the allegation of the release of an experimental virus from a Wuhan lab accidental, in all reasonable versions was a conspiracy theory. A Wuhan lab leak, like the Wuhan market origin, would by itself have been compatible with mainstream Sinophobia. But the eagerness of Trump and his cohorts to play the China virus card, along with the recognition that coronavirus research in Wuhan was conducted in close collaboration with the University of North Carolina research group directed by Ralph Baric and the EcoHealth Alliance, a multidisciplinary New York-based organisation headed by Peter Daszac, which would have implicated the US in the laboratory scenario, drove many away from this plausible position. The fact that this work had been conducted with the approval of Fauci and Collins themselves made the mobilisation by these figures of strenuous rejection of the Wuhan lab-leak conjecture, as noted by the journalist Sam Husseini, an actual conspiracy.

The circumstantial case for the human adaptation of SARS-CoV2 during transit in a Wuhan laboratory is made persuasively by two excellent books, The Origin of the Deadliest Pandemic in 100 Years: An Investigation by Elaine Dewar, a Canadian science journalist, and Viral: The Search for the Origin of Covid-19 by Matt Ridley and Alina Chan, respectively a science populariser and a molecular biologist. The two books come to essentially identical conclusions by entirely different methods. Dewar explicitly followed the model of the legendary US political journalist IF Stone, scrutinising the public record, doggedly pursuing inconsistencies, and taking note of abrupt termination of phone conversations. Her book is a saga of the concomitant growth of expertise and rage. Chan began as a trained expert, a genetics researcher at the MIT-affiliated Broad Institute, who put her career on hold to track the SARS-CoV2 origin story as it emerged, often against the desires of Chinese researchers in Wuhan and their US collaborators, in the scientific literature, as well as by the freelance efforts of the Decentralised Radical Autonomous Search Team Investigating COVID-19, or DRASTIC, activist group and the Ithaca, New York-based Bioscience Resource Project.

Similar views on the laboratory origin of SARS-Cov2 have been put forward by others with more questionable intellectual pedigrees. For instance, Ridley, a member of the British House of Lords, is a sceptic on anthropogenic climate change, and the science journalist Nicholas Wade, who wrote a long piece on the subject in the Bulletin of the Atomic Scientists, is an advocate of genetic race science specifics that feature in lazy dismissals of the lab-leak hypothesis in the absence of any evidence at all of natural emergence. Another advocate of the lab-leak scenario, think-tanker Jamie Metzl, has recently written a book advocating human germline genetic engineering. But Dewar and Chan show their work you can virtually watch how their ideas take form and for this reason can serve as honest guides in the face of incomplete, and, perhaps, withheld, evidence.

Since these books have been written, new information has emerged in the form of leaked and FOIA-obtained grant proposals, one funded by the NIH and one turned down by the US military research agency DARPA, that document collaborative work and planned experiments by scientists at the Wuhan Institute of Virology and the US EcoHealth Alliance. As described in articles in The Intercept by Sharon Lerner, Maia Hibbett, and Mara Hvistendahl, this work included culturing bat coronaviruses isolated from the wild with human lung cells, and infection with such viruses of humanised mice mice genetically engineered to have human virus-binding receptors to develop variants that were more infectious for humans, the objective presumably being eventual vaccine design. The investigators also proposed inserting a furin cleavage site into some of the bat coronaviruses, an infection-enabling feature of the virus spike protein not found in bat viruses most closely related to SARS-CoV2. It has also come to light that among the bat virus isolates brought to and studied in the Wuhan lab were ones from Laos. The fact that the identified bat virus with the best genetic match to SARS-CoV2 was found at a site 1,000 miles away from Wuhan was previously used to discredit the lab-leak hypothesis.

For those following the score of this danse macabre, this is where we stand right now.

CounterPunch.org, December 8. Stuart A Newman is professor of cell biology and anatomy at New York Medical College and co-author of Biotech Juggernaut: Hope, Hype and Hidden Agendas of Entrepreneurial Bioscience.

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