When genome editing goes off-target

HANNAH R. KEMPTON | April 19, 2019 | 49 views

Editing DNA in eukaryotic cells with CRISPR-based systems has revolutionized the genome engineering field. Cas (CRISPR-associated) endonucleases are directed to a particular location in the genome by a short guide RNA, providing an easily programmable strategy to target any section of DNA. As of now, two CRISPR-based approaches can introduce targeted, permanent edits. DNA cleavage with the Cas endonuclease facilitates small insertions or deletions of nucleotides that can disable the targeted gene (1). A second modified “base editor” system can generate precise single-base mutations in the targeted DNA (2). For both approaches, it is imperative that DNA modifications are made in the intended region (“on-target”) and not elsewhere in the genome (“off-target”). On pages 286, 289, and 292 of this issue, Wienert et al. (3), Zuo et al. (4), and Jin et al. (5), respectively, describe methods that identify off-target activities, which will be invaluable in therapeutic contexts as well as for stringent evaluation of future iterations of gene-editing tools.

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How to Choose a Reliable Biotech Clinical Trial Management System?

Article | July 12, 2022

Introduction The medical and life-science industries are experiencing a robust transformation with the increasing prevalence of various types of diseases, including infectious diseases, chronic disorders, and acute conditions around the world. As a result, a significant rise in demand for more effective therapeutic drugs and bionics is being witnessed, leading to a swift increase in the number of clinical trials. For a successful trial, it is important for biotech companies to ensure the data submitted to regulatory bodies regarding clinical trials is accurate, reliable, and definitive from an ethical point of view. A reliable clinical trial management system plays a vital role in collecting, monitoring, and managing clinical data. The availability of high-quality clinical data also helps clinical research institutions make efficient treatment decisions and provide proper patient care. Hence, a number of biotech companies and research organizations are focusing on leveraging innovative clinical trial management solutions to handle a large amount of data, particularly in multi-center trials, and generate reliable, high-quality, and statistically sound data from clinical trials. However, selecting the most appropriate and reliable clinical trial management system is vital for the clinical trial's success. Let's see some of the steps that will assist these firms in choosing the right CTMS. Key Steps for Selecting Right Biotech Clinical Trial Management System Prioritize Study Needs Considering and prioritizing study needs is a crucial step in choosing the most reliable clinical trial management system for biotech companies. Prioritizing helps them to identify a solution that improves the study's quality and removes uncertainty for researchers when faced with difficult choices. Hence, biotech and life-science organizations should choose a clinical trial system that is simple to use, well-organized, and suitably designed to minimize the number of clicks required to complete a task. Select CTMS with Multiple Integrations Integrated clinical trial management systems provide the best value for the companies’ funds as they guarantee the smooth functioning of research protocols. In addition, integrations are necessary to fully understand the importance and advantages of clinical trial management software for ensuring smooth transitions between site management and data collection. Biotech and clinical research should look for CTMS platforms that can integrate with electronic medical record (EMR) platforms and clinical research process content (CRPC) billing grids. This will allow them to use the same billing designations and ensure compliance while minimizing the need for duplicate processes. Ensure System Compliance and Security Clinical research organizations need to adhere to a plethora of complex regulations in order to ensure compliance with one of the most challenging environments of principles, which is information security and privacy. Security and system compliance are vital aspects of choosing the right CTMS solutions for biotech firms as they assist in building trust and form a part of the system’s duties. While selecting CTMS systems, it is essential for companies engaged in clinical research to ensure that these platforms are able to configure both, group and individual permissions, along with having a data backup and recovery plan for hosted systems. This will allow companies to assess the privacy and security implications of research and anticipate complications that may arise in each phase of the project. Assess the Scalability Choosing a scalable CTMS that can accommodate various types of fluctuations and expansions enables biotech and clinical firms to quickly adapt to fast-changing trends and demand spikes while reducing maintenance costs and enhancing user agility. As scalability also means secure and expanded data storage, these businesses should instead use SaaS solutions than manually manage an ever-growing collection of hard drives. The right CTMS ensures accommodating the firm’s availability requirements without incurring the capital costs associated with expanding a physical infrastructure. The Closing Thought A well-executed and successful clinical trial involves multiple stages and processes. Several quality controls and stringent adherence to regulations are essential for the steps, along with efficient cross-departmental processes and procedures. Incorporating the right CTMS paves the way for paperless data collection, regulatory filing, and fiscal management tools for biotech researchers and administrative personnel.

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MEDICAL

Top 3 Biotech Clinical Data Management Trends to Watch in 2022

Article | July 14, 2022

Introduction The administration of medical records and data has advanced significantly during the past few decades. Clinical data management, which was once only a small subset of biotech research organizations, has now developed into a mission-critical, specialized unit. In the late 1990s, electronic data capture (EDC) began to alter the traditional function of clinical data management. After that, the data configuration and management of data queries for the EDC system fell under the purview of clinical data management services. Today, clinical data management is not only responsible for managing the clinical data configuration and data queries but also developing and implementing data administration plans, ensuring data accuracy and completeness, and maintaining optimum data security. In recent years, as digital technologies have gained acceptance around the globe, data has become a vital aspect in decision-making across numerous industries, and the life sciences and biotechnology sectors are no exception. Using data has provided granular insights to biotech organizations, assisting them in creating breakthroughs in drug development and medical research and signifying the importance of clinical trial management systems in these medical verticals. The Biggest Biotech Clinical Data Management Trends to Know About Today The future of clinical data management is contingent upon the implementation of systems and regulations. It is imperative for all organizations participating in a medical or life science trial to have transparent rules in place for sharing and retaining patient data. Also, there is a need to have a standardized format for maintaining these records and documents related to trials. This assists biotech organizations in reducing the chances of ambiguity regarding who owns what kind of data or paperwork at any given time. Over the past couple of years, the focus of the life science and biotechnology industries has shifted towards developing more effective medications and therapies, implementing personalized treatment, and finding cures for diseases such as cancer and AIDS. In response to this, a substantial rise in the number of clinical trials is being witnessed globally. As the number of clinical trials continues to accelerate, the spending on these trials rises as well. In response to this, the worldwide cost of conducting clinical trials is anticipated to reach US$ 49.80 billion in 2022. With the transition of the world from traditional to digital, medical professionals and biotech businesses are increasingly shifting towards adopting high-tech and reliable clinical trial management systems for various applications, starting from diagnosis and clinical trials to patient data documentation. But, what are the future trends in biotechnology clinical data management? Let’s discuss. Cloud-Based Clinical Metadata Repositories Automation is emerging as a new frontier in the biotech clinical data management domain, along with other innovative technologies such as artificial intelligence and machine learning. Because of this, life science establishments are witnessing a huge shift from paper-based documentation toward data-based documentation, which is creating mountains of research, compliance, and clinical data. The growing demand for new and more effective medications and drugs is augmenting the need to expedite clinical trials. This is resulting in an increased number of initiatives aimed at optimizing clinical trial processes to prepare and launch successful trials. However, pharmaceutical and biotechnology laboratories are encountering several challenges in collecting, managing, and analyzing metadata due to its complexities. So, what is the best solution to this problem? The answer to this is cloud-based clinical metadata repositories. Clinical research facilities are leveraging advanced, all-in-one, cloud-based clinical metadata repositories to assist them in centralizing and managing metadata; increasing metadata quality, consistency, and accuracy; and speeding up clinical trial management, documentation, and compliance processes. Shift Towards Digital Solutions Electronic Case Report Form Adequate research and accurate data are crucial for a clinical trial to succeed. Whether developing new drugs, medication, or therapies; conducting life science research; or studying the latest clinical trial systems, it is best to use electronic solutions as it reduces the room for mistakes during the transition of clinical data from paper-based format. Realizing this, biotech organizations are shifting towards using electronic case report forms to speed up record retrieval, improve record security, and cut down on operational costs associated with running clinical trials. The electronic case report form assists in lowering the failure rate of the clinical trial, enhancing efficiency, and optimizing security along with improving clinical trial documentation and productivity, further driving its adoption in the medical space. Electronic Clinical Outcome Assessment Electronic clinical outcome assessment is surfacing as one of the fast-growing future trends in biotechnology. It allows clinical trial facilities to automate data entry and improve the reliability of the collected information. The technology enables clinical trial institutions to automatically record patient-provided information about side effects, symptoms, drug timing, and other aspects during the clinical trial for increased precision. It also helps these institutions analyze the results of medication or therapy in clinical trials and lets clinical researchers use medical technologies like biosensor-enabled devices, self-service applications, and medical wearables for evaluation. Hence, biotech clinical facilities are increasingly deploying advanced electronic clinical outcome assessment systems to ensure adherence to protocols and regulations. Clinical Trial Customization The success of a new drug is determined by numerous factors other than its effectiveness, safety, and creativity of its developers, such as a successful clinical trial. Each clinical trial involves a number of decision-making points, and one wrong choice in any of these aspects can jeopardize the success of the entire endeavor. A crucial component of making well-informed decisions is data management, which is a part of clinical study as a whole. Clinical trial customization is emerging as one of the most prominent biotech clinical trial management trends. Every clinical trial is unique and needs a tailored approach to be successful. With the emergence of the trend of personalized treatment around the globe, biotech and pharmaceutical organizations are adopting innovative customized clinical trial management solutions to accelerate the pace of clinical trials and approvals. This is giving clinical researchers innovative ways to come up with new medicines for patients and streamline the clinical data as per the requirements for faster approvals. What Are the Key Clinical Data Management Challenges Faced by Biotech Companies? Groundbreaking medical interventions are of no use without reliable, accurate, and extensive clinical trial data. Without the data, biotech and pharmaceutical companies will not be able to provide the assurance of safety and efficacy required to bring the medication to market. Regulatory bodies such as the Food and Drug Administration (FDA), the Medicines and Healthcare Products Regulatory Agency (MHRA), and others are putting stricter rules in place to ensure the quality of clinical data. In addition, the fast-changing clinical development environment is creating more obstacles for biotech and medical spaces to ensure the accuracy, standard, and completeness of the clinical trial data. Hence, clinical teams are spending valuable time cleaning up data instead of analyzing it. Time spent trying to figure out issues with clinical trial data is detrimental and expensive but also mission-critical. This is because a small issue in the data can lead to numerous consequences, from small delays to calamitous setbacks, making it necessary to rerun clinical trials. This problem will only get more challenging to address as the volume of data and the types of data sources continue to grow. Here are some of the major clinical data management challenges that biotech firms encounter Standardization of Clinical Metadata Stringent Regulatory Compliance Increased Clinical Trial Complexity Mid-Study Changes Why Are Clinical Data Management Systems Garnering Popularity in the Biotech Industry? With the changing regulatory and clinical landscape, biotech and pharmaceutical companies are facing several obstacles in the management of clinical data and clinical trials. In addition, regulatory agencies are moving toward integrated electronic systems, which is making it more and more important for clinical laboratories to change the format of their submissions. Because of this, several biotech clinical labs are focusing on adopting innovative laboratory solutions, such as biotech clinical data management systems, to meet the need for standardized data inputs and replace all manual ways of working with electronic systems. A clinical data management system establishes the framework for error-free data collection and high-quality data submission, resulting in speedier drug discovery and shorter time-to-market. These solutions are gaining huge traction among biotech and pharmaceutical companies, owing to their ability to effectively manage clinical data, accelerate clinical trials, and ensure compliance. Let’s see some of the features of biotech clinical data management software that are most sought after by life-science companies Controlled, standardized data repository. Centralized data analysis and administration. Reduced operational expenditures for clinical data processes. Enhanced process effectiveness. Superior submission quality Compliance with predefined standards. Clinical Data Management Systems: The Future The role of clinical data management systems is evolving at a rapid pace as the life science and medical industries continue to incorporate digital solutions for diverse operations. These systems are being used in a variety of biotech clinical settings, ranging from clinical data compliance to data science and analytics, to help them analyze large and growing volumes of clinical data. Hence, a number of high-tech medical companies are aiming at integrating innovative technologies, such as artificial intelligence and machine learning, into clinical data management software to automate clinical data management tasks, improve clinical data submission, and enhance data quality. These new biotech clinical management technologies are anticipated to help life science laboratories gain a better understanding of diseases and speed up clinical trials in the coming years. FAQ What is a clinical data management system? A clinical data management system (CDMS) is a tool used in clinical research to track, record, and manage clinical trial data across medical establishments such as biotech laboratories. What are the key functions of the biotech clinical data management system? Some of the key functions of biotech clinical data management system are Documentation of Protocols and Regulations Patient Recruitment Real-time Clinical Study Analytics Reporting Investigator Relationship Management Electronic Visit Report Why is a clinical data management system needed for clinical trials today? A clinical data management system helps shorten the time from drug development to marketing by assisting in the collection of high-quality, statistically sound, and accurate data from clinical trials.

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MEDTECH

Top 10 biotech IPOs in 2019

Article | July 11, 2022

The big question at the start of 2019 was whether the IPO window would stay open for biotech companies, particularly those seeking to pull off ever-larger IPOs at increasingly earlier stages of development. The short answer is yes—kind of. Here’s the long answer: In the words of Renaissance Capital, the IPO market had “a mostly good year.” The total number of deals fell to 159 from 192 the year before, but technology and healthcare companies were standout performers. The latter—which include biotech, medtech and diagnostics companies—led the pack, making up 43% of all IPOs in 2019. By Renaissance’s count, seven companies went public at valuations exceeding $1 billion, up from five the year before

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Cell Out? Lysate-Based Expression an Option for Personalized Meds

Article | February 18, 2020

Cell-free expression (CFE) is the practice of making a protein without using a living cell. In contrast with cell line-based methods, production is achieved using a fluid containing biological components extracted from a cell, i.e., a lysate. CFE offers potential advantages for biopharma according to Philip Probert, PhD, a senior scientist at the Centre for Process Innovation in the U.K.

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Verantos Named "BioInformatics Company of the Year" by BioTech Breakthrough

BioTech Breakthrough, Verantos | October 28, 2021

Verantos, the market leader in high-validity real-world evidence (RWE) generation, today announced it has been named "BioInformatics Company of the Year" in the inaugural BioTech Breakthrough Awards program conducted by BioTech Breakthrough. This independent market intelligence organization evaluates and recognizes standout life sciences and biotechnology companies, products, and services around the globe. The Verantos real-world evidence (RWE) platform leverages data science, artificial intelligence, and advanced data sources to generate evidence that enables precision medicine. It also provides a more inclusive approach to a learning healthcare system, in which underrepresented populations are better represented than in other evidence generation approaches. The platform is able to de-identify, enrich, and integrate data from electronic health records, medical claims, and registries to produce exacting and exceptionally valid RWE. Bryan Vaughn, Managing Director of BioTech Breakthrough Awards said, "Verantos continues to develop high-validity RWE in collaboration with pharmaceutical, regulatory and scientific institutions, enabling personalized medicine, and improved, more efficient care. Congratulations on being our choice for 'BioInformatics Company of the Year." Verantos has generated advanced RWE for top-tier biotechnology and pharmaceutical firms in addition to leading academic medical centers and health systems. It has also developed advanced science funded by the Food and Drug Administration, the National Institutes of Health, and the National Science Foundation. Additionally, Verantos CEO Dan Riskin testified before Congress on the 21st Century Cures Initiative and has helped shape policy in the use of advanced clinical data and technologies. These partnerships and initiatives have allowed Verantos to trailblaze in their industry and benefit patients living with common conditions across the globe. "We are thrilled to receive this award from BioTech Breakthrough. It supports our mission to advance real-world evidence to improve the standard of care, and furthers our vision to pioneer personalized medicine by setting the quality standard for real-world evidence. said Dan Riskin, CEO of Verantos. The annual BioTech Breakthrough Awards program conducts the industry's most comprehensive analysis and evaluation of the top technology companies, solutions and products in the life sciences and biotechnology industry today. This year's program attracted more than 1,200 nominations from over 12 different countries throughout the world. About Verantos Verantos is the market leader in high-accuracy, real-world evidence generation. The Verantos RWE platform integrates heterogeneous real-world data sources and generates evidence with the accuracy necessary for regulatory and reimbursement use. The Verantos RWE platform leverages data science and artificial intelligence along with advanced data sources such as electronic health records to generate RWE capable of supporting clinical assertions. About BioTech Breakthrough Part of Tech Breakthrough, a leading market intelligence and recognition platform for global technology innovation and leadership, the BioTech Breakthrough Awards program is devoted to honoring excellence in life science and biotechnology solutions, services and companies. The BioTech Breakthrough Awards provide public recognition for the achievements of biotechnology companies and products in categories including BioPharma, Genomics, Therapeutics, Food Science and BioAgriculture, and more.

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INDUSTRIAL IMPACT

Omega Therapeutics Announces Strategic Research Collaboration with Stanford University School of Medicine

Omega Therapeutics, Inc | October 14, 2021

Omega Therapeutics, Inc. ("Omega"), a development-stage biotechnology company pioneering the first systematic approach to use mRNA therapeutics as programmable epigenetic medicines by leveraging its OMEGA Epigenomic Programing™ platform, today announced a strategic research collaboration with researchers at the Stanford University School of Medicine to explore the therapeutic potential of Omega Epigenomic Controllers (OECs) to control ocular disease genes associated with inflammation or regeneration of ocular tissues. Under the terms of the collaboration, Omega and members of the Ophthalmology Department of Stanford University School of Medicine will use the OMEGA Epigenomic Programming platform to discover and research novel ocular targets for potential future OEC development candidates. Albert Wu, M.D., Ph.D., FACS, Associate Professor of Ophthalmology, will serve as principal investigator. Other contributors will include Jeffrey Goldberg, M.D., Ph.D., Professor and Chair of Ophthalmology, and Michael Kapiloff, M.D., Ph.D., Associate Professor (Research) of Ophthalmology. "Through this research collaboration, we aim to expand the reach of our OMEGA platform within regenerative medicine, immunology, and inflammation with ocular disease targets. We will continue exploration of the broad potential of our disruptive platform and OECs, our new class of mRNA therapeutics as programmable epigenetic medicines." Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics About Omega Therapeutics Omega Therapeutics is a development-stage biotechnology company pioneering the first systematic approach to use mRNA therapeutics as programmable epigenetic medicines by leveraging its OMEGA Epigenomic Programming™ platform. The OMEGA platform harnesses the power of epigenetics, the mechanism that controls gene expression and every aspect of an organism's life from cell genesis, growth and differentiation to cell death. The OMEGA platform enables control of fundamental epigenetic processes to correct the root cause of disease by returning aberrant gene expression to a normal range without altering native nucleic acid sequences. Omega's engineered, modular, and programmable mRNA-encoded epigenetic medicines, Omega Epigenomic Controllers™, target specific intervention points amongst the thousands of mapped and validated novel DNA-sequence-based epigenomic loci to durably tune single or multiple genes to treat and cure disease through Precision Genomic Control™. Omega is currently advancing a broad pipeline of development candidates spanning a range of disease areas, including oncology, regenerative medicine, multigenic diseases including immunology, and select monogenic diseases.

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MEDTECH

Outcomes4Me Partners with Invitae to Offer Genetic Testing to Breast Cancer Patients

Outcomes4Me | October 12, 2021

Outcomes4Me Inc., developer of a leading free mobile app and platform to navigate cancer treatment and care, today announced that it has partnered with Invitae Corporation (NYSE: NVTA), a leading medical genetics company, to expand education and access to genetic testing to breast cancer patients and survivors. The collaboration leverages the strengths of Invitae, which supplies clinical grade genetic testing, and Outcomes4Me’s 360-degree, validated and evidence-based cancer support and treatment options via its free and easy-to-use app. Initially and currently available in the United States, patients can now receive genetic counseling through Invitae’s partnership with Genome Medical, get testing, and upload their results within the Outcomes4Me app.There is a misconception that genetic testing is only useful as a preventative tool prior to a cancer diagnosis. According to Outcomes4Me patient data, almost half of users (46 percent) who qualified for testing (based on NCCN Guidelines®) did not receive testing or did not know if they had received testing. However, genetic testing can provide insights that can help inform and refine precision therapy use and clinical treatment trial enrollment. In addition, genetic testing results can be used to help prevent recurrence and reduce incidence of other inherited cancers. A cancer diagnosis is often overwhelming for patients and their families. Outcomes4Me demystifies cancer by providing the most up-to-date and validated research, support, and treatment options, all grounded in science and data and curated according to the patient’s specific diagnosis. Outcomes4Me partners with the researchers, doctors, and academics that set the rigorous standards of cancer care for all treatment providers, including the National Comprehensive Care Network® (NCCN®), Vanderbilt-Ingram Cancer Center (VICC) and Massachusetts General Hospital (MGH). The collaboration with Invitae expands access to genetic testing, a vitally important tool in the patient’s cancer care arsenal. “Outcomes4Me is an indispensable platform for patients with breast cancer, giving them the personalized knowledge and access to timely new trials and targeted therapies that could lead to better health outcomes. By providing access to our comprehensive genetic testing and counseling services, Outcomes4Me is adding a valuable resource that will empower patients to advance their knowledge, understanding, and therefore, self-advocacy during treatment and survivorship.” Ed Esplin, M.D., Ph.D., FACMG, FACP, Clinical Geneticist at Invitae Unlike popular direct-to-consumer genetic testing services, which test for a few specific genetic variants for certain genes, Invitae provides state-of-the-art clinical grade next-generation sequencing-based (NGS) genetic testing that comprehensively analyses more than 80 genes, including all known mutations of the important BRCA1/BRCA2 genes. This comprehensive approach, combined with associated genetic counseling, not only provides insights for cancer patients, but also for family members who may be at risk. “Our collaboration with Invitae reinforces Outcomes4Me’s mission to give patients back control. Because of this work with Invitae, our valued community now has rare direct access to a much-needed testing service. Outcomes4Me will proudly continue to democratize the best in cancer treatment, research, and support by removing barriers and bias in information flow.” Maya R. Said, Sc. D., Founder and CEO of Outcomes4Me About Invitae Invitae Corporation (NYSE: NVTA) is a leading medical genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. About Outcomes4Me Outcomes4Me is on a mission to improve health outcomes by empowering patients with understandable, relevant and evidence-based information. Outcomes4Me has developed a platform for shared decision-making between patients and providers. The platform harnesses regulatory-grade, real-world data and patient experiences generating deeper insights and better outcomes to improve care and accelerate research. The Outcomes4Me mobile app enables cancer patients to make decisions and take control of their care based on information that is personalized to their specific condition, including finding treatment options, matching to clinical trials, and tracking and managing symptoms. Based in Boston, Massachusetts, Outcomes4Me, a woman-led company, comprises seasoned healthcare, oncology, pharmaceutical, consumer and technology veterans.

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INDUSTRIAL IMPACT

Verantos Named "BioInformatics Company of the Year" by BioTech Breakthrough

BioTech Breakthrough, Verantos | October 28, 2021

Verantos, the market leader in high-validity real-world evidence (RWE) generation, today announced it has been named "BioInformatics Company of the Year" in the inaugural BioTech Breakthrough Awards program conducted by BioTech Breakthrough. This independent market intelligence organization evaluates and recognizes standout life sciences and biotechnology companies, products, and services around the globe. The Verantos real-world evidence (RWE) platform leverages data science, artificial intelligence, and advanced data sources to generate evidence that enables precision medicine. It also provides a more inclusive approach to a learning healthcare system, in which underrepresented populations are better represented than in other evidence generation approaches. The platform is able to de-identify, enrich, and integrate data from electronic health records, medical claims, and registries to produce exacting and exceptionally valid RWE. Bryan Vaughn, Managing Director of BioTech Breakthrough Awards said, "Verantos continues to develop high-validity RWE in collaboration with pharmaceutical, regulatory and scientific institutions, enabling personalized medicine, and improved, more efficient care. Congratulations on being our choice for 'BioInformatics Company of the Year." Verantos has generated advanced RWE for top-tier biotechnology and pharmaceutical firms in addition to leading academic medical centers and health systems. It has also developed advanced science funded by the Food and Drug Administration, the National Institutes of Health, and the National Science Foundation. Additionally, Verantos CEO Dan Riskin testified before Congress on the 21st Century Cures Initiative and has helped shape policy in the use of advanced clinical data and technologies. These partnerships and initiatives have allowed Verantos to trailblaze in their industry and benefit patients living with common conditions across the globe. "We are thrilled to receive this award from BioTech Breakthrough. It supports our mission to advance real-world evidence to improve the standard of care, and furthers our vision to pioneer personalized medicine by setting the quality standard for real-world evidence. said Dan Riskin, CEO of Verantos. The annual BioTech Breakthrough Awards program conducts the industry's most comprehensive analysis and evaluation of the top technology companies, solutions and products in the life sciences and biotechnology industry today. This year's program attracted more than 1,200 nominations from over 12 different countries throughout the world. About Verantos Verantos is the market leader in high-accuracy, real-world evidence generation. The Verantos RWE platform integrates heterogeneous real-world data sources and generates evidence with the accuracy necessary for regulatory and reimbursement use. The Verantos RWE platform leverages data science and artificial intelligence along with advanced data sources such as electronic health records to generate RWE capable of supporting clinical assertions. About BioTech Breakthrough Part of Tech Breakthrough, a leading market intelligence and recognition platform for global technology innovation and leadership, the BioTech Breakthrough Awards program is devoted to honoring excellence in life science and biotechnology solutions, services and companies. The BioTech Breakthrough Awards provide public recognition for the achievements of biotechnology companies and products in categories including BioPharma, Genomics, Therapeutics, Food Science and BioAgriculture, and more.

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INDUSTRIAL IMPACT

Omega Therapeutics Announces Strategic Research Collaboration with Stanford University School of Medicine

Omega Therapeutics, Inc | October 14, 2021

Omega Therapeutics, Inc. ("Omega"), a development-stage biotechnology company pioneering the first systematic approach to use mRNA therapeutics as programmable epigenetic medicines by leveraging its OMEGA Epigenomic Programing™ platform, today announced a strategic research collaboration with researchers at the Stanford University School of Medicine to explore the therapeutic potential of Omega Epigenomic Controllers (OECs) to control ocular disease genes associated with inflammation or regeneration of ocular tissues. Under the terms of the collaboration, Omega and members of the Ophthalmology Department of Stanford University School of Medicine will use the OMEGA Epigenomic Programming platform to discover and research novel ocular targets for potential future OEC development candidates. Albert Wu, M.D., Ph.D., FACS, Associate Professor of Ophthalmology, will serve as principal investigator. Other contributors will include Jeffrey Goldberg, M.D., Ph.D., Professor and Chair of Ophthalmology, and Michael Kapiloff, M.D., Ph.D., Associate Professor (Research) of Ophthalmology. "Through this research collaboration, we aim to expand the reach of our OMEGA platform within regenerative medicine, immunology, and inflammation with ocular disease targets. We will continue exploration of the broad potential of our disruptive platform and OECs, our new class of mRNA therapeutics as programmable epigenetic medicines." Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics About Omega Therapeutics Omega Therapeutics is a development-stage biotechnology company pioneering the first systematic approach to use mRNA therapeutics as programmable epigenetic medicines by leveraging its OMEGA Epigenomic Programming™ platform. The OMEGA platform harnesses the power of epigenetics, the mechanism that controls gene expression and every aspect of an organism's life from cell genesis, growth and differentiation to cell death. The OMEGA platform enables control of fundamental epigenetic processes to correct the root cause of disease by returning aberrant gene expression to a normal range without altering native nucleic acid sequences. Omega's engineered, modular, and programmable mRNA-encoded epigenetic medicines, Omega Epigenomic Controllers™, target specific intervention points amongst the thousands of mapped and validated novel DNA-sequence-based epigenomic loci to durably tune single or multiple genes to treat and cure disease through Precision Genomic Control™. Omega is currently advancing a broad pipeline of development candidates spanning a range of disease areas, including oncology, regenerative medicine, multigenic diseases including immunology, and select monogenic diseases.

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MEDTECH

Outcomes4Me Partners with Invitae to Offer Genetic Testing to Breast Cancer Patients

Outcomes4Me | October 12, 2021

Outcomes4Me Inc., developer of a leading free mobile app and platform to navigate cancer treatment and care, today announced that it has partnered with Invitae Corporation (NYSE: NVTA), a leading medical genetics company, to expand education and access to genetic testing to breast cancer patients and survivors. The collaboration leverages the strengths of Invitae, which supplies clinical grade genetic testing, and Outcomes4Me’s 360-degree, validated and evidence-based cancer support and treatment options via its free and easy-to-use app. Initially and currently available in the United States, patients can now receive genetic counseling through Invitae’s partnership with Genome Medical, get testing, and upload their results within the Outcomes4Me app.There is a misconception that genetic testing is only useful as a preventative tool prior to a cancer diagnosis. According to Outcomes4Me patient data, almost half of users (46 percent) who qualified for testing (based on NCCN Guidelines®) did not receive testing or did not know if they had received testing. However, genetic testing can provide insights that can help inform and refine precision therapy use and clinical treatment trial enrollment. In addition, genetic testing results can be used to help prevent recurrence and reduce incidence of other inherited cancers. A cancer diagnosis is often overwhelming for patients and their families. Outcomes4Me demystifies cancer by providing the most up-to-date and validated research, support, and treatment options, all grounded in science and data and curated according to the patient’s specific diagnosis. Outcomes4Me partners with the researchers, doctors, and academics that set the rigorous standards of cancer care for all treatment providers, including the National Comprehensive Care Network® (NCCN®), Vanderbilt-Ingram Cancer Center (VICC) and Massachusetts General Hospital (MGH). The collaboration with Invitae expands access to genetic testing, a vitally important tool in the patient’s cancer care arsenal. “Outcomes4Me is an indispensable platform for patients with breast cancer, giving them the personalized knowledge and access to timely new trials and targeted therapies that could lead to better health outcomes. By providing access to our comprehensive genetic testing and counseling services, Outcomes4Me is adding a valuable resource that will empower patients to advance their knowledge, understanding, and therefore, self-advocacy during treatment and survivorship.” Ed Esplin, M.D., Ph.D., FACMG, FACP, Clinical Geneticist at Invitae Unlike popular direct-to-consumer genetic testing services, which test for a few specific genetic variants for certain genes, Invitae provides state-of-the-art clinical grade next-generation sequencing-based (NGS) genetic testing that comprehensively analyses more than 80 genes, including all known mutations of the important BRCA1/BRCA2 genes. This comprehensive approach, combined with associated genetic counseling, not only provides insights for cancer patients, but also for family members who may be at risk. “Our collaboration with Invitae reinforces Outcomes4Me’s mission to give patients back control. Because of this work with Invitae, our valued community now has rare direct access to a much-needed testing service. Outcomes4Me will proudly continue to democratize the best in cancer treatment, research, and support by removing barriers and bias in information flow.” Maya R. Said, Sc. D., Founder and CEO of Outcomes4Me About Invitae Invitae Corporation (NYSE: NVTA) is a leading medical genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. About Outcomes4Me Outcomes4Me is on a mission to improve health outcomes by empowering patients with understandable, relevant and evidence-based information. Outcomes4Me has developed a platform for shared decision-making between patients and providers. The platform harnesses regulatory-grade, real-world data and patient experiences generating deeper insights and better outcomes to improve care and accelerate research. The Outcomes4Me mobile app enables cancer patients to make decisions and take control of their care based on information that is personalized to their specific condition, including finding treatment options, matching to clinical trials, and tracking and managing symptoms. Based in Boston, Massachusetts, Outcomes4Me, a woman-led company, comprises seasoned healthcare, oncology, pharmaceutical, consumer and technology veterans.

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