Act now on CRISPR babies

A researcher steps forward and says he has plans to edit the genes of babies. He wants to alter a gene called CCR5 to protect children from HIV. He seems to have the skills, tools and position to do so — and he starts to tell other scientists about his plans.

Spotlight

Access Genetics

Access Genetics, LLC is a privately held Minnesota-based company founded in 2001 by Dr. Ronald McGlennen, an internationally recognized molecular diagnostics expert with over a decade of experience in implementing and developing molecular genetic laboratories. As a medical technology service company, we offer turnkey solutions to assist laboratories in the implementation of molecular genetic testing by recommending a tailored, cost-effective and state-of-the-art suite of hardware, software, prepackaged materials and supplies, process guides, custom tools, staff training, technical support, telemedicine interpretations, and other optional consultative services. This comprehensive service package makes Access Genetics unlike other companies in the genetic testing market that may only provide a piece of the testing puzzle.

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Medical

Expansion of BioPharma: Opportunities and Investments

Article | July 14, 2022

Biopharmaceutical innovations are among the most ingenious and refined achievements of modern medical science. New concepts, techniques, and therapies are emerging, such as the cell therapy Provenge, which can be used to treat cancer, and gene therapies, which provide even more amazing promises of disease remission and regenerative medicine. In addition, the COVID-19 pandemic has caused a huge boom in the pharmaceutical industry. This is because more and more attention is being paid to increasing manufacturing capacity and starting new research on drug development. Biopharma: Leading the Way in the Pharma Sector In the past couple of years, the biopharmaceutical sector has deepened its roots across the medical and pharmaceutical industries, on account of the transformation of pharmaceutical companies towards biotechnology, creating opportunities for growth. Also, growing advancements in technologies such as 3D bioprinting, biosensors, and gene editing, along with the integration of advanced artificial intelligence and virtual and augmented reality are estimated to further create prospects for growth. According to a study, the biopharmaceutical sector makes nearly $163 billion around the world and grows by more than 8% each year, which is twice as fast as the traditional pharma sector. Massive Investments Directed Towards Biopharma Investing in biotech research and development (R&D) has yielded better returns than the pharma industry average. Hence, a number of pharmaceutical companies are shifting their presence toward biopharma to capitalize on the upcoming opportunities by investing in and expanding their biotechnology infrastructure. For instance, Thermo Fisher Scientific Inc., an American manufacturer of scientific instrumentation, reagents and consumables, and software services, announced an investment of $97 million to expand its bioanalytical laboratory operations into three new locations in the U.S. With this investment, the company will add 150,000 square feet of scientific workspace and install the most advanced drug development technologies to produce life-changing medicines for patients in need.

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MedTech

Immunology: A New Frontier in Medical Science

Article | July 13, 2022

Introduction Recent developments in the bioengineering of monoclonal antibodies (mAbs) have revolutionized the treatment of numerous rheumatic and immunological disorders. Currently, several immunological disorders are successfully being targeted and treated using innovative medical techniques such as immunotherapy. Leading companies are increasingly investing in research activities to expand the usage and application of immunology for the treatment of various infectious diseases, including multiple sclerosis, inflammatory bowel disorders, lupus, and psoriasis, leading companies are increasingly investing in research activities. Today, the efforts of researchers in immunology, with a long history of study and research, have borne fruit, as bioengineered mAbs are now being employed in clinical practices. Accelerating Investments: Paving the Way for Immunology The increasing prevalence of infectious diseases, cancer, and immune-mediated inflammatory disorders (IMIDs) is raising the need for more precise classification and an in-depth understanding of the pathology underlying these ailments. Numerous leaders in the biotechnology domain are thus focusing on undertaking numerous strategies, such as new facility launches and collaborations, to address the need by finding deeper inroads into immunology and its use in disease treatments. For instance, in 2022, the University of Texas MD Anderson Cancer Center announced the launch of a visionary research and innovation hub, the James P. Allison Institute, to find new roads in immunotherapy, develop new treatments, and foster groundbreaking science. These developments will result in better diagnosis through the use of selective biomarkers, and early detection of fatal diseases and their treatment, which will prevent complications from happening. Also, the identification of high-risk populations through a deeper understanding of genetic and environmental factors can assist in the prevention of disease through immunotherapy. The Way Forward Immunology has led to the development of biotechnology, making it possible to develop novel drugs and vaccines, as well as diagnostic tests, that can be used to prevent, diagnose, and treat a wide range of autoimmune, infectious, and cancerous diseases. With the rapid advancement in technology and the integration of artificial intelligence, immunology is finding its way into an array of domains and industries, encompassing several research areas including medicine, pharmaceuticals, agriculture, and space. Today, not only researchers but also leading biotech and pharmaceutical companies have recognized that conventional therapies with pharmaceutical and chemical products are being replaced by products derived from immunology. This is because they work well for health problems, are environmentally friendly, and are also emerging as a wealth-generating business in the medical field.

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MedTech

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

Article | July 11, 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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Wisconsin biotech companies could play key roles in long-term economic recovery from COVID-19 pandemic

Article | April 19, 2020

Whether it’s called a modern “Manhattan Project” or a medical moon shot, the concept of long-term economic recovery rests on how confident people are they won’t risk serious illness by venturing forth in public again. Wisconsin stands to be a significant part of such an undertaking, whatever it’s called. The shorter-term debate is well under way over the gradual lifting of COVID-19 emergency rules, such as the now-extended “safer-at-home” order in Wisconsin. At least a dozen states, including regional coalitions on the East and West coasts, are exploring next steps as they seek to balance responses to the virus with calls for reopening the economy, at least, in part. Wisconsin’s ability to shape longer-term responses will come from private and public resources, which range from companies engaged in production of diagnostics.

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Spotlight

Access Genetics

Access Genetics, LLC is a privately held Minnesota-based company founded in 2001 by Dr. Ronald McGlennen, an internationally recognized molecular diagnostics expert with over a decade of experience in implementing and developing molecular genetic laboratories. As a medical technology service company, we offer turnkey solutions to assist laboratories in the implementation of molecular genetic testing by recommending a tailored, cost-effective and state-of-the-art suite of hardware, software, prepackaged materials and supplies, process guides, custom tools, staff training, technical support, telemedicine interpretations, and other optional consultative services. This comprehensive service package makes Access Genetics unlike other companies in the genetic testing market that may only provide a piece of the testing puzzle.

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.

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

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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