Boosting R&D Productivity With Pooled CRISPR Screening

June 23, 2019 | 16 views

Pooled screening is a valuable problem-solving and cost-reduction asset at a time when pressure to conduct focused, productive pharmaceutical R&D is more intense than ever. With the emergence of CRISPR gene-editing technology, pooled screens are becoming an essential component of drug discovery and development in the age of functional genomics.

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MEDICAL

2022 U.S. Market Research Report with COVID-19 Forecasts2

Article | June 22, 2022

The global biotechnology market is expected to grow at a compound annual growth rate (CAGR) of 13.9 percent from 2022 to 2030, with a value estimated at USD 1,023.92 billion in 2021. The market is being propelled by strong government support in the form of initiatives aimed at modernizing the regulatory framework, improving approval processes and reimbursement policies, and standardizing clinical studies. The growing presence of personalized medicine and an increasing number of orphan drug formulations are opening up new avenues for biotechnology applications and driving the influx of emerging and innovative biotechnology companies, which is driving market revenue even further. The 2022 Biotech Research and Development Market Research Report is one of the most comprehensive and in-depth assessments of the industry in the United States, containing over 100 data sets spanning the years 2013 to 2026. This Kentley Insights report contains historical and forecasted market size, product lines, profitability, financial ratios, BCG matrix, state statistics, operating expense details, organizational breakdown, consolidation analysis, employee productivity, price inflation, pay bands for the top 20 industry jobs, trend analysis and forecasts on companies, locations, employees, payroll, and much more. Companies in the Biotech Research and Development industry are primarily engaged in biotechnology research and experimental development. Biotechnology research and development entails the investigation of the use of microorganisms and cellular and bimolecular processes to create or modify living or non-living materials. This biotechnology research and development may result in the development of new biotechnology processes or prototypes of new or genetically altered products that can be replicated, used, or implemented by various industries. This report was created using the findings of extensive business surveys and econometrics. The professionals follow reports with accurate and apt information on market sizing, benchmarking, strategic planning, due diligence, cost-cutting, planning, understanding industry dynamics, forecasting, streamlining, gap analysis, and other ana

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MEDTECH

Biotech in 2022

Article | July 20, 2022

The robust global channel of more than, 800 gene and cell curatives presently in trials will produce clinical readouts in 2022, revealing what lies ahead for advanced curatives. The impact will be felt in 2022, no matter how you slice it. Eventually, how well industry and non-supervisory bodies unite to produce new frameworks for advanced therapies will shape the year 2022 and further. Pacific Northwest talent will continue to contribute to the advancement of gene and cell curatives in both the short and long term, thanks to its deep pool of ground-breaking scientific developers, entrepreneurial directorial leadership, largely skilled translational scientists, and endured bio manufacturing technicians. We may see continued on-life science fund withdrawal from biotech in 2021, but this can be anticipated as a strong comeback in 2022 by biotech industry, backed by deep-pocketed life science investors who are committed to this sector. A similar investment, combined with pharma's cash-heavy coffers, can result in increased junction and acquisition activity, which will be a challenge for some but an occasion for others. Over the last five years, investment interest in Seattle and the Pacific Northwest has grown exponentially, from Vancouver, British Columbia, to Oregon. The region's explosive portfolio of new biotech companies, innovated out of academic centres, demonstrates the region's growing recognition of scientific invention. This created a belief that continued, especially because Seattle's start-ups and biotech enterprises are delivering on their pledge of clinical and patient impact. Talent and staffing will continue to be difficult to find. It's a CEO's market, but many of these funds' return, and are not rising in proportion to the exorbitant prices they're paying to enter deals. This schism has become particularly pronounced in 2021. Hence, everyone in biotech is concerned about reclamation and retention.

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MEDTECH

Next-Gen Genetics Cancer Therapies Creating Investment Prospects

Article | July 13, 2022

Genetic therapeutics such as genetic engineering and gene therapy are increasingly emerging as one of the most influential and transformed biotechnological solutions around the globe in recent times. These genetic solutions are being assessed across various medical domains, including cancer treatment, neurology, oncology, and ophthalmology. Citing the trend, the genetics industry is estimated to experience a tsunami of approvals, with over 1,000 cell and gene therapy clinical trials currently underway and over 900 companies worldwide focusing on these cutting-edge therapies. Growing Cancer Encourages Advancements in Genetic Technologies With the surging cases of cancers such as leukemias, carcinomas, lymphomas, and others, patients worldwide are increasing their spending on adopting novel therapeutic solutions for non-recurring treatment of the disease, such as gene therapy, genetic engineering, T-cell therapy, and gene editing. As per a study by the Fight Cancer Organization, spending on the treatment of cancer increased to $200.7 billion, and the amount is anticipated to exceed $245 billion by the end of 2030. Growing revenue prospects are encouraging biotechnology and biopharmaceutical companies to develop novel genetic solutions for cancer treatment. For instance, Bristol-Myers Squibb K.K., a Japanese pharmaceutical company, introduced a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, Abecma, for the treatment of relapsed or refractory (R/R) multiple myeloma in 2022. Amid a New Market: Genetics Will Attract Massive Investments Despite several developments and technological advancements, genetics is still considered to be in a nascent stage, providing significant prospects for growth to the companies that are already operating in the domain. Genetics solutions such as gene therapies, gene editing, and T-cell immunotherapy are emerging as highly active treatments across various medical fields, resulting in increasing research and development activities across the domain, drawing significant attention from investors. Given the potential of genetic treatments and the focus on finding new ways to treat cancer and other related diseases, it's easy to understand why companies are investing in the domain. For instance, Pfizer has recently announced an investment of around $800 million to construct development facilities supporting gene therapy manufacturing from initial preclinical research through final commercial-scale production. Due to these advancements, cell and gene therapies are forecast to grow from $4 billion annually to more than $45 billion, exhibiting growth at a 63% CAGR. The Future of Genetics Though there is a significant rise in advancement in genetic technologies and developments, the number of approved genetic treatments remains extremely small. However, with gene transfer and CRISPR solutions emerging as new modalities for cancer treatment, the start-up companies will attract a growing amount and proportion of private and public investments. This is expected present a tremendous opportunity for biopharma and biotechnology investors to help fund and benefit from the medical industry's shift from traditional treatments to cutting-edge genetic therapeutics in the coming years.

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MEDICAL

Advancement in Genomics Accelerating its Penetration into Precision Health

Article | June 22, 2022

Genomics is an interdisciplinary field of biology emphasizing the structure, editing, evolution, function, and mapping of genomes. It is creating deeper inroads across the precision health domain with the increasing introduction of advanced technologies such as quantum simulation, next-generation sequencing (NGS), and precise genome manipulation. As precision health focuses on providing the proper intervention to the right patient at the right time, genomics increasingly finds applications in human and pathogen genome sequencing in clinical and research spaces. Rising Hereditary Diseases Burden Paving the Way for Genomics in Precision Health In the last few years, a significant surge in the prevalence of diseases and ailments such as diabetes, obesity, baldness, and others has been witnessed across the globe. A history of family members with chronic diseases, such as cancer, diabetes, high blood pressure, hearing issues, and heart disease, can sometimes continue into the next generation. Hence, the study of genes is extensively being conducted for predicting health risks and early treatment of these diseases. It also finds use in CRISPR-based diagnostics and the preparation of precision medication for the individual. In addition, ongoing advancements in genomics are making it possible to identify different genetic traits that persuade people to more widespread diseases and health problems. The Emergence of Genomics Improves Disease Understanding Genomics refers to the study of the complete genetic makeup of a cell or organism. Increasing scientific research in the area substantially contributes to increasing knowledge about the human genome and assists in improving the ability to understand disease etiology, risk, diagnosis, treatment, and prevention. On account of these improvements, innovative genomic technologies and tools are being developed to enable better precision health not only for the individual but for various regional populations as well. The Way Forward With growing preference for personalized medicine and an increasing need for more accurate pathogen detection and diagnostics, genomics is gaining huge popularity across the precision health domain. Also, increasing research activities for developing novel high-precision therapeutics and rising importance of gene study in the prevention, diagnosis, and management of infectious and genetic diseases will further pave the way for genomics in the forthcoming years.

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Spotlight

Aprimo

Aprimo provides technology solutions for content, operations, and performance that enable enterprises to optimize their brand experiences and the resources they use to deliver them. Our platform gives enterprises the advantage by streamlining and governing all the behind-the-scenes activities – from ideation to distribution – involved in delivering exceptional brand experiences.

Related News

CRISPR Therapeutics, Vertex Report First Data from Trials of Gene-Editing Treatment CTX001

GEN | November 19, 2019

CRISPR Therapeutics and Vertex Pharmaceuticals today reported preliminary, mostly-positive safety and efficacy data from the first two patients enrolled in two Phase I/II trials assessing their CRISPR/Cas9 gene-edited therapy CTX001 for a pair of blood disorders—the first clinical trial of a gene-editing candidate sponsored by U.S. companies. “We are very encouraged by these preliminary data, the first such data to be reported for patients with beta thalassemia and sickle cell disease treated with our CRISPR/Cas9 edited autologous hematopoietic stem cell candidate CTX001,” CRISPR Therapeutics CEO Samarth Kulkarni, PhD, said in a statement. “These data support our belief in the potential of our therapies to have meaningful benefit for patients following a one-time intervention. We continue to enroll these studies as we drive forward to develop CRISPR/Cas9 therapies as a new class of transformative medicines to treat serious diseases.” Added Vertex Chairman, President and CEO Jeffrey Leiden, MD, PhD: “The data we announced today are remarkable and demonstrate that CTX001 has the potential to be a curative CRISPR/Cas9-based gene-editing therapy.”

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CRISPR Gene Editing Ability Improved by Specific Modifications of tracrRNA

GEN | November 11, 2019

Scientists at the City of Hope believe they may have found a way to sharpen the fastest, cheapest, and most accurate gene editing technique, CRISPR-Cas9, so that it can more successfully cut out undesirable genetic information. This improved cutting ability could one day fast-track potential therapies for HIV, sickle cell disease, and, potentially, other immune conditions. “Our CRISPR-Cas9 design may be the difference between trying to cut a ribeye steak with a butter knife versus slicing it with a steak knife,” said Tristan Scott, PhD, lead author of the study and a staff research scientist at City of Hope’s Center for Gene Therapy. “Other scientists have tried to improve CRISPR cutting through chemical modifications, but that’s an expensive process and is like diamond-coating a blade. Instead, we have designed a better pair of scissors you can buy at any convenience store.” The study, “Improved Cas9 activity by specific modifications of the tracrRNA,” published in Scientific Reports is the first time scientists have systematically gone through the guide RNA sequence to change it and improve CRISPR-Cas9 technology, Scott said. The Kevin Morris Lab at City of Hope has filed a patent application claiming this improved CRISPR-Cas9 design, which could result in a doubling of activity but the exact amount was dependent on the target site, Scott said.

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A Breath of Fresh CRISPR

GEN | November 04, 2019

Genome editing materials can’t just breeze into cells. Or can they? Even cells so well defended as lung and airway cells may admit wisps of genome editing proteins such as CRISPR-associated nucleases. All that’s needed is an inspired delivery method. One possibility is the aerosolization of amphiphilic peptides. Amphiphilic peptides combine hydrophilic and lipophilic properties and facilitate the translocation of proteins across membranes. These peptides are being evaluated for various applications, including genome editing. In fact, scientists from the University of Iowa, in collaboration with scientists from Feldan Therapeutics, recently used engineered amphiphilic peptides to deliver genome editing nucleases and ribonucleoproteins to cultured human airway epithelial cells and mouse lungs.

Read More

CRISPR Therapeutics, Vertex Report First Data from Trials of Gene-Editing Treatment CTX001

GEN | November 19, 2019

CRISPR Therapeutics and Vertex Pharmaceuticals today reported preliminary, mostly-positive safety and efficacy data from the first two patients enrolled in two Phase I/II trials assessing their CRISPR/Cas9 gene-edited therapy CTX001 for a pair of blood disorders—the first clinical trial of a gene-editing candidate sponsored by U.S. companies. “We are very encouraged by these preliminary data, the first such data to be reported for patients with beta thalassemia and sickle cell disease treated with our CRISPR/Cas9 edited autologous hematopoietic stem cell candidate CTX001,” CRISPR Therapeutics CEO Samarth Kulkarni, PhD, said in a statement. “These data support our belief in the potential of our therapies to have meaningful benefit for patients following a one-time intervention. We continue to enroll these studies as we drive forward to develop CRISPR/Cas9 therapies as a new class of transformative medicines to treat serious diseases.” Added Vertex Chairman, President and CEO Jeffrey Leiden, MD, PhD: “The data we announced today are remarkable and demonstrate that CTX001 has the potential to be a curative CRISPR/Cas9-based gene-editing therapy.”

Read More

CRISPR Gene Editing Ability Improved by Specific Modifications of tracrRNA

GEN | November 11, 2019

Scientists at the City of Hope believe they may have found a way to sharpen the fastest, cheapest, and most accurate gene editing technique, CRISPR-Cas9, so that it can more successfully cut out undesirable genetic information. This improved cutting ability could one day fast-track potential therapies for HIV, sickle cell disease, and, potentially, other immune conditions. “Our CRISPR-Cas9 design may be the difference between trying to cut a ribeye steak with a butter knife versus slicing it with a steak knife,” said Tristan Scott, PhD, lead author of the study and a staff research scientist at City of Hope’s Center for Gene Therapy. “Other scientists have tried to improve CRISPR cutting through chemical modifications, but that’s an expensive process and is like diamond-coating a blade. Instead, we have designed a better pair of scissors you can buy at any convenience store.” The study, “Improved Cas9 activity by specific modifications of the tracrRNA,” published in Scientific Reports is the first time scientists have systematically gone through the guide RNA sequence to change it and improve CRISPR-Cas9 technology, Scott said. The Kevin Morris Lab at City of Hope has filed a patent application claiming this improved CRISPR-Cas9 design, which could result in a doubling of activity but the exact amount was dependent on the target site, Scott said.

Read More

A Breath of Fresh CRISPR

GEN | November 04, 2019

Genome editing materials can’t just breeze into cells. Or can they? Even cells so well defended as lung and airway cells may admit wisps of genome editing proteins such as CRISPR-associated nucleases. All that’s needed is an inspired delivery method. One possibility is the aerosolization of amphiphilic peptides. Amphiphilic peptides combine hydrophilic and lipophilic properties and facilitate the translocation of proteins across membranes. These peptides are being evaluated for various applications, including genome editing. In fact, scientists from the University of Iowa, in collaboration with scientists from Feldan Therapeutics, recently used engineered amphiphilic peptides to deliver genome editing nucleases and ribonucleoproteins to cultured human airway epithelial cells and mouse lungs.

Read More

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