Automated Extraction of High Molecular Weight DNA

SmartExtraction significantly simplifies the entire automated workflow of DNA extraction, setting new standards with regard to efficiency, yield and quality of the DNA.

Spotlight

BioMarin Pharmaceutical Inc.

BioMarin is a global pharmaceutical company focused on developing first-in-class and best-in-class therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases. We remain steadfast to our original mission—to bring new treatments to market that will make a big impact on small patient populations. These patient populations are mostly children, suffering from diseases so rare, that the entire patient population can number as few as 1,000 people worldwide. These conditions are often inherited, difficult to diagnose, progressively debilitating, have few, if any, treatment options and are usually ignored.

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MedTech

Expansion of BioPharma: Opportunities and Investments

Article | September 22, 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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Medical

Next-Gen Gene Therapy to Counter Complex Diseases

Article | August 16, 2022

Gene therapy has historically been used to treat disorders with in-depth knowledge caused by a single genetic mutation. Thanks to the introduction of new generation technologies, the potential of gene therapy is expanding tAo treat diseases that were previously untreatable. Evolution of Gene Therapy One of the major success stories of the twenty-first century has been gene therapy. However, it has not been the same in the past. The field's journey to this point has been long and mostly difficult, with both tragedy and triumph along the way. Initially, genetic disorders were thought to be untreatable and permanently carved into the genomes of individuals unfortunate enough to be born with them. But due to the constant technological advancement and research activities, gene therapy now has the potential to treat various genetic mutation-causing diseases with its ability to insert a new copy and replace faulty genes. Gene Therapy is Finding New Roads in the Medical Sector Gene therapy can help researchers treat a variety of conditions that fall under the general heading of epilepsy, instead of only focusing on a particular kind of disorder brought on by a genetic mutation. Following are some of the domains transformed by gene therapy. Neurology – Gene therapy can be used for the treatment of seizures by directly injecting it into the area causing an uncontrolled electrical disturbance in the brain. Furthermore, by using DNA sequences known as promoters, gene therapy can be restricted to specific neurons within that area. Ophthalmology – Genetic conditions such as blindness can be caused due to the mutation of any gene out of over 200 and resulting in progressive vision loss in children. With advanced gene therapies such as optogenetics, lost photoreceptor function can be transferred to the retinal cells, which are responsible for relaying visual information to the brain. This might give patients the ability to navigate in an unknown environment with a certain level of autonomy. The Future of Gene Therapy The news surrounding gene therapy has been largely favorable over the past few years, with treatment after treatment obtaining regulatory approvals, successful clinical trials, and garnering significant funds to begin development. With more than 1,000 clinical trials presently underway, the long-awaited gene therapy revolution might finally be here.

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MedTech

Better Purification and Recovery in Bioprocessing

Article | July 11, 2022

In the downstream portion of any bioprocess, one must pick through the dross before one can seize the gold the biotherapeutic that the bioprocess was always meant to generate. Unfortunately, the dross is both voluminous and various. And the biotherapeutic gold, unlike real gold, is corruptible. That is, it can suffer structural damage and activity loss. When discarding the dross and collecting the gold, bioprocessors must be efficient and gentle. They must, to the extent possible, eliminate contaminants and organic debris while ensuring that biotherapeutics avoid aggregation-inducing stresses and retain their integrity during purification and recovery. Anything less compromises purity and reduces yield. To purify and recover biotherapeutics efficiently and gently, bioprocessors must avail themselves of the most appropriate tools and techniques. Here, we talk with several experts about which tools and techniques can help bioprocessors overcome persistent challenges. Some of these experts also touch on new approaches that can help bioprocessors address emerging challenges.

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Medical

Nanostructures: Emerging as Effective Carriers for Drug Delivery

Article | July 14, 2022

Natural remedies have been employed in medicine since antiquity. However, a large number of them fail to go past the clinical trial stages. In vivo instability, poor solubility and bioavailability, a lack of target-specific delivery, poor absorption, and side effects of the medication are only a few of the problems caused by the use of large-sized materials in drug administration. Therefore, adopting novel drug delivery systems with targeted medications may be a solution to address these pressing problems. Nanotechnology has received tremendous attention in recent years and has been demonstrated to help blur the boundaries between the biological and physical sciences. With great success, it plays a vital part in enhanced medication formulations, targeted venues, and controlled drug release and delivery. Limitations of Traditional Delivery Trigger the Adoption of Nanoparticles The field of nanotechnology and the creation of drug formulations based on nanoparticles is one that is expanding and showcasing great potential. It has been thoroughly researched in an effort to develop new methods of diagnosis and treatment and to overcome the limitations of several diseases' current therapies. As a result, nanoparticles are being used to improve the therapeutic effectiveness and boost patient adherence to treatment by increasing medication bioavailability, drug accumulation at a particular spot, and reducing drug adverse effects. The nanoparticles could be transformed into intelligent systems housing therapeutic and imaging agents by manipulating their surface properties, size, correct drug load, and release with targeted drug delivery. Nanostructures facilitate the release of combination medications at the prescribed dose since they remain in the blood circulation system for a long time. Therefore, they result in fewer plasma fluctuations with decreased side effects. Due to their nanoscale, these structures can easily enter the tissue system, promote the absorption of drugs by cells, make medication administration more effective, and ensure that the medicine acts at the targeted location. The Way Ahead Nanomedicine and nano-delivery systems are a comparatively new but fast-evolving science in which nanoscale materials are used as diagnostic tools to deliver drug molecules at precisely targeted sites in a controlled manner. It is finding applications for the treatment of diseases such as cardiovascular, neurodegenerative, cancer, ocular, AIDS, and diabetes, among others. With more research and technological advancement, these drug delivery solutions will open up huge opportunities for companies that work with them.

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Spotlight

BioMarin Pharmaceutical Inc.

BioMarin is a global pharmaceutical company focused on developing first-in-class and best-in-class therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases. We remain steadfast to our original mission—to bring new treatments to market that will make a big impact on small patient populations. These patient populations are mostly children, suffering from diseases so rare, that the entire patient population can number as few as 1,000 people worldwide. These conditions are often inherited, difficult to diagnose, progressively debilitating, have few, if any, treatment options and are usually ignored.

Related News

AI, Diagnostics

Genomic Vision Announces launch of FiberSmart

Genomic Vision | March 08, 2023

On March 7, 2023, Genomic Vision, a leading biotech company that develops products and services for the analysis and control of genome changes, launched FiberSmart®, an AI-based technology for automating the quantification and detection of fluorescent signals on combed DNA molecules. Initially available for the analysis of Replication Combing Assays (RCA), Genomic Vision's proprietary method for directly visualizing DNA replication kinetics at the single molecule level. FiberSmart uses advanced AI methods to detect, visualize, and analyze DNA replication kinetics up to 3x more accurately and up to 10x faster than Genomic Vision's existing software solutions. The technology offers a simple and user-friendly interface, facilitating quick analysis of DNA replication signals to deduce essential parameters describing replication kinetics. It is compatible with Genomic Vision's FiberVision® and FiberVision-S® scanners for the RCA assay of the company's proprietary DNA combing technology with various potential applications, including in gene and cell therapy quality control. FiberSmart has been successfully tested and validated by AstraZeneca and the Fritz Lipmann Institute in Germany. Genomic Vision's CEO Aaron Bensimon said, "The launch of FiberSmart® is an important milestone for Genomic Vision as we bring the benefits of powerful AI technology to our users, who can now perform faster and more accurate genomic analysis seamlessly. Our proprietary DNA combing technique has multiple potential applications, particularly in the cell and gene therapy space, where highly accurate genomic analysis is paramount to ensure robust quality standards are met. With the launch of this software we are making it easier for users to exploit the full potential of our proprietary advanced genomic analysis technique." (Source – Business Wire) About Genomic Vision GENOMIC VISION is a leading biotechnology company specializing in the analysis of genome modifications, with a focus on ensuring their quality and safety in genome editing technologies and biomanufacturing processes. It utilizes advanced nanotechnology for DNA analysis to develop cutting-edge diagnostic and drug discovery solutions for cancer and acute diseases at the intersection of genome dynamics and human diseases. The company's approach employs Molecular Combing Technology, a powerful technique that directly visualizes single DNA molecules to detect and quantify changes in the genome landscape and their contribution to pathology.

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Medical

Spotlight Therapeutics Raises $36.5 Million Series B to Advance a Pipeline of Cell-Targeted In Vivo CRISPR Gene Editing Biologics

Spotlight Therapeutics | March 22, 2022

Spotlight Therapeutics, Inc. (“Spotlight”), a biotechnology company applying new insights to develop cell-targeted in vivo CRISPR gene editing biologics, today announced a $36.5M Series B financing to fuel a drive toward the clinic. The financing round was co-led by new investors GordonMD Global Investments and EPIQ Capital Group, with participation from Magnetic Ventures, as well as existing investors GV (formerly Google Ventures) and Emerson Collective and other investors. Craig Gordon, M.D., Founder, CEO and CIO of GordonMD Global Investments, joins the Company’s Board of Directors. Spotlight's proprietary technology platform, TAGE (Targeted Active Gene Editors), is a new class of biologics; highly engineered, modular programmable CRISPR effectors designed to target and edit selected cell types in vivo. This approach circumvents the complexity of packaged viral, viral-like, and nanoparticle delivery systems, opens the door to expanded applications, and holds the promise of increasing patient access. We are excited to help Spotlight advance its pioneering work, which shows promise for cell-targeted delivery of CRISPR effectors in vivo. Spotlight’s TAGE platform could enable significant expansion of CRISPR medicines to a wide range of diseases." Dr. Gordon. This Series B funding is a crucial milestone as we advance our lead first-in-class immuno-oncology (IO) program and progress our pipeline of programs in IO, ophthalmic diseases and hemoglobinopathies,It will enable us to execute our development plan, leveraging Spotlight’s unique cell-targeted in vivo delivery approach, as we aspire to unlock the full potential of gene editing and enable effective one-and-done medicines for patients.” Mary Haak-Frendscho, Ph.D., President and CEO of Spotlight Therapeutics. About Spotlight Therapeutics Established in mid-2018, Spotlight Therapeutics is a privately held biotechnology company advancing a pipeline of cell-targeted in vivo CRISPR gene editing therapies. Spotlight's proprietary technology platform TAGE (Targeted Active Gene Editors) is a new class of biologics, CRISPR effectors engineered for direct delivery in vivo, to achieve cell-selective therapeutic genome editing. Spotlight's pipeline is advancing its modular programmable CRISPR effectors towards clinical studies in immuno-oncology, ophthalmic diseases and hemoglobinopathies. The company is headquartered in Hayward, California.

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Medical

TGen Selects MemVerge to Accelerate Idiopathic Pulmonary Fibrosis Research Discovery with Big Memory Technology

MemVerge | March 21, 2022

MemVerge™, the pioneers of Big Memory software, today announced that TGen, the Translational Genomics Research Institute, an affiliate of City of Hope, has selected MemVerge Memory Machine Big Memory virtualization software to accelerate time to discovery for Idiopathic Pulmonary Fibrosis (IPF), a disease which affects 100,000 people annually in the U.S. Using MemVerge technology, TGen is able to dramatically speed analytical processing by nearly 36% for single-cell RNA sequencing. As a nonprofit medical research institute, TGen researchers process single-cell RNA sequences to characterize cell transcriptomic profiles. The process can take up to six and a half hours to analyze a matrix of 30,000 genes by 114,000 cells. With consistently growing datasets, this processing time was preventing a desired time to discovery. The data required for analysis was simply too large to retain in traditional memory, and scaling capacity with dynamic random-access memory (DRAM) was too costly. TGen has instead deployed memory virtualization technology from MemVerge which virtualizes both DRAM and PMem (persistent memory) memory technologies, to increase the memory pool available for processing without requiring more high-cost DRAM. The solution further speeds TGen's genomics sequencing analysis with Memory Machine ZeroIO in-memory snapshots which capture multi-terabyte data sets at any point for rapid reloads at each stage of processing. The ZeroIO snapshot service is 1,000 times faster than the fastest storage snapshot to SSD and enables TGen to run processing workflows in parallel. This ensures that in the event of a system crash, in-memory snapshots are available to instantly re-start long running jobs without lengthy reloading. By utilizing the snapshotting and cloning capabilities of Memory Machine, we were able to parallelize the processing workflow, As a result, we can now save nearly 36% of computational time while also taking advantage of the big memory nodes. This will save a lot of time in downstream analysis." Glen Otero, Ph.D., Vice President of Scientific Computing at TGen. MemVerge Memory Machine has quickly resulted in research value for TGen, We have removed performance barriers from their research process so that they are able to perform vital, life-saving, research faster than ever possible. Now TGen is expanding the use of Big Memory technology across other research use cases where results and discoveries can produce findings for a healthier tomorrow." Jonathan Jiang, COO of MemVerge. MemVerge Memory Machine makes 100% use of available memory capacity while providing new operational capabilities to memory-centric workloads. Memory Machine answers the need for a modern in-memory computing model to support emerging applications that require real-time analytics, true in-memory computing, and fault-tolerant memory persistence to speed massive processing workloads. About MemVerge MemVerge is pioneering Big Memory Computing and Big Memory Cloud technology for the memory-centric and multi-cloud future. MemVerge® Memory Machine™ is the industry's first software to virtualize memory hardware for fine-grained provisioning of capacity, performance, availability, and mobility. On top of the transparent memory service, Memory Machine provides another industry first, ZeroIO™ in-memory snapshots which can encapsulate terabytes of application state within seconds and enable data management at the speed of memory. The breakthrough capabilities of Big Memory Computing and Big Memory Cloud Technology are opening the door to cloud agility and flexibility for thousands of Big Memory applications.

Read More

AI, Diagnostics

Genomic Vision Announces launch of FiberSmart

Genomic Vision | March 08, 2023

On March 7, 2023, Genomic Vision, a leading biotech company that develops products and services for the analysis and control of genome changes, launched FiberSmart®, an AI-based technology for automating the quantification and detection of fluorescent signals on combed DNA molecules. Initially available for the analysis of Replication Combing Assays (RCA), Genomic Vision's proprietary method for directly visualizing DNA replication kinetics at the single molecule level. FiberSmart uses advanced AI methods to detect, visualize, and analyze DNA replication kinetics up to 3x more accurately and up to 10x faster than Genomic Vision's existing software solutions. The technology offers a simple and user-friendly interface, facilitating quick analysis of DNA replication signals to deduce essential parameters describing replication kinetics. It is compatible with Genomic Vision's FiberVision® and FiberVision-S® scanners for the RCA assay of the company's proprietary DNA combing technology with various potential applications, including in gene and cell therapy quality control. FiberSmart has been successfully tested and validated by AstraZeneca and the Fritz Lipmann Institute in Germany. Genomic Vision's CEO Aaron Bensimon said, "The launch of FiberSmart® is an important milestone for Genomic Vision as we bring the benefits of powerful AI technology to our users, who can now perform faster and more accurate genomic analysis seamlessly. Our proprietary DNA combing technique has multiple potential applications, particularly in the cell and gene therapy space, where highly accurate genomic analysis is paramount to ensure robust quality standards are met. With the launch of this software we are making it easier for users to exploit the full potential of our proprietary advanced genomic analysis technique." (Source – Business Wire) About Genomic Vision GENOMIC VISION is a leading biotechnology company specializing in the analysis of genome modifications, with a focus on ensuring their quality and safety in genome editing technologies and biomanufacturing processes. It utilizes advanced nanotechnology for DNA analysis to develop cutting-edge diagnostic and drug discovery solutions for cancer and acute diseases at the intersection of genome dynamics and human diseases. The company's approach employs Molecular Combing Technology, a powerful technique that directly visualizes single DNA molecules to detect and quantify changes in the genome landscape and their contribution to pathology.

Read More

Medical

Spotlight Therapeutics Raises $36.5 Million Series B to Advance a Pipeline of Cell-Targeted In Vivo CRISPR Gene Editing Biologics

Spotlight Therapeutics | March 22, 2022

Spotlight Therapeutics, Inc. (“Spotlight”), a biotechnology company applying new insights to develop cell-targeted in vivo CRISPR gene editing biologics, today announced a $36.5M Series B financing to fuel a drive toward the clinic. The financing round was co-led by new investors GordonMD Global Investments and EPIQ Capital Group, with participation from Magnetic Ventures, as well as existing investors GV (formerly Google Ventures) and Emerson Collective and other investors. Craig Gordon, M.D., Founder, CEO and CIO of GordonMD Global Investments, joins the Company’s Board of Directors. Spotlight's proprietary technology platform, TAGE (Targeted Active Gene Editors), is a new class of biologics; highly engineered, modular programmable CRISPR effectors designed to target and edit selected cell types in vivo. This approach circumvents the complexity of packaged viral, viral-like, and nanoparticle delivery systems, opens the door to expanded applications, and holds the promise of increasing patient access. We are excited to help Spotlight advance its pioneering work, which shows promise for cell-targeted delivery of CRISPR effectors in vivo. Spotlight’s TAGE platform could enable significant expansion of CRISPR medicines to a wide range of diseases." Dr. Gordon. This Series B funding is a crucial milestone as we advance our lead first-in-class immuno-oncology (IO) program and progress our pipeline of programs in IO, ophthalmic diseases and hemoglobinopathies,It will enable us to execute our development plan, leveraging Spotlight’s unique cell-targeted in vivo delivery approach, as we aspire to unlock the full potential of gene editing and enable effective one-and-done medicines for patients.” Mary Haak-Frendscho, Ph.D., President and CEO of Spotlight Therapeutics. About Spotlight Therapeutics Established in mid-2018, Spotlight Therapeutics is a privately held biotechnology company advancing a pipeline of cell-targeted in vivo CRISPR gene editing therapies. Spotlight's proprietary technology platform TAGE (Targeted Active Gene Editors) is a new class of biologics, CRISPR effectors engineered for direct delivery in vivo, to achieve cell-selective therapeutic genome editing. Spotlight's pipeline is advancing its modular programmable CRISPR effectors towards clinical studies in immuno-oncology, ophthalmic diseases and hemoglobinopathies. The company is headquartered in Hayward, California.

Read More

Medical

TGen Selects MemVerge to Accelerate Idiopathic Pulmonary Fibrosis Research Discovery with Big Memory Technology

MemVerge | March 21, 2022

MemVerge™, the pioneers of Big Memory software, today announced that TGen, the Translational Genomics Research Institute, an affiliate of City of Hope, has selected MemVerge Memory Machine Big Memory virtualization software to accelerate time to discovery for Idiopathic Pulmonary Fibrosis (IPF), a disease which affects 100,000 people annually in the U.S. Using MemVerge technology, TGen is able to dramatically speed analytical processing by nearly 36% for single-cell RNA sequencing. As a nonprofit medical research institute, TGen researchers process single-cell RNA sequences to characterize cell transcriptomic profiles. The process can take up to six and a half hours to analyze a matrix of 30,000 genes by 114,000 cells. With consistently growing datasets, this processing time was preventing a desired time to discovery. The data required for analysis was simply too large to retain in traditional memory, and scaling capacity with dynamic random-access memory (DRAM) was too costly. TGen has instead deployed memory virtualization technology from MemVerge which virtualizes both DRAM and PMem (persistent memory) memory technologies, to increase the memory pool available for processing without requiring more high-cost DRAM. The solution further speeds TGen's genomics sequencing analysis with Memory Machine ZeroIO in-memory snapshots which capture multi-terabyte data sets at any point for rapid reloads at each stage of processing. The ZeroIO snapshot service is 1,000 times faster than the fastest storage snapshot to SSD and enables TGen to run processing workflows in parallel. This ensures that in the event of a system crash, in-memory snapshots are available to instantly re-start long running jobs without lengthy reloading. By utilizing the snapshotting and cloning capabilities of Memory Machine, we were able to parallelize the processing workflow, As a result, we can now save nearly 36% of computational time while also taking advantage of the big memory nodes. This will save a lot of time in downstream analysis." Glen Otero, Ph.D., Vice President of Scientific Computing at TGen. MemVerge Memory Machine has quickly resulted in research value for TGen, We have removed performance barriers from their research process so that they are able to perform vital, life-saving, research faster than ever possible. Now TGen is expanding the use of Big Memory technology across other research use cases where results and discoveries can produce findings for a healthier tomorrow." Jonathan Jiang, COO of MemVerge. MemVerge Memory Machine makes 100% use of available memory capacity while providing new operational capabilities to memory-centric workloads. Memory Machine answers the need for a modern in-memory computing model to support emerging applications that require real-time analytics, true in-memory computing, and fault-tolerant memory persistence to speed massive processing workloads. About MemVerge MemVerge is pioneering Big Memory Computing and Big Memory Cloud technology for the memory-centric and multi-cloud future. MemVerge® Memory Machine™ is the industry's first software to virtualize memory hardware for fine-grained provisioning of capacity, performance, availability, and mobility. On top of the transparent memory service, Memory Machine provides another industry first, ZeroIO™ in-memory snapshots which can encapsulate terabytes of application state within seconds and enable data management at the speed of memory. The breakthrough capabilities of Big Memory Computing and Big Memory Cloud Technology are opening the door to cloud agility and flexibility for thousands of Big Memory applications.

Read More

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