Bioprocessing Technology Trends of RNA-Based Therapeutics and Vaccines

RNA-based therapeutics are an emerging class of therapies that has bright prospects in the treatment and prevention of difficult-to-treat diseases. In 2014, DNA and RNA therapeutics saw 12% year-over-year growth and industry analytics data suggest that the RNA-based therapeutics market will reach $1.2 billion by 2020.  In a recently published article, an overview of the types of RNA-based therapeutics in existence and their optimal methods of manufacture, purification, formulation and delivery are presented. The article reviews the current dynamics in the RNA therapeutics/vaccines market as well as differences between small-interfering RNA (siRNA), RNA interference (RNAi), microRNA (miRNA), and messenger RNA (mRNA).

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Genetic Intelligence, Inc

We build machines that make transcendent medicine by finding and targeting truly causal genetic features in the Whole Genome, even from small sample sizes.

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MedTech

Better Purification and Recovery in Bioprocessing

Article | October 7, 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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MedTech

Next-Gen Gene Therapy to Counter Complex Diseases

Article | September 22, 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

Advancement in Genomics Accelerating its Penetration into Precision Health

Article | July 13, 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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Diagnostics

Making Predictions by Digitizing Bioprocessing

Article | April 20, 2021

With advances in data analytics and machine learning, the move from descriptive and diagnostic analytics to predictive and prescriptive analytics and controls—allowing us to better forecast and understand what will happen and thus optimize process outcomes—is not only feasible but inevitable, according to Bonnie Shum, principal engineer, pharma technical innovation, technology & manufacturing sciences and technology at Genentech. “Well-trained artificial intelligence systems can help drive better decision making and how data is analyzed from drug discovery to process development and to manufacturing processes,” she says. Those advances, though, only really matter when they improve the lives of patients. That’s exactly what Shum expects. “The convergence of digital transformation and operational/processing changes will be critical for the facilities of the future and meeting the needs of our patients,” she continues. “Digital solutions may one day provide fully automated bioprocessing, eliminating manual intervention and enabling us to anticipate potential process deviations to prevent process failures, leading to real-time release and thus faster access for patients.” To turn Bioprocessing 4.0 into a production line for precision healthcare, real-time release and quickly manufacturing personalized medicines will be critical. Adding digitization and advanced analytics wherever possible will drive those improvements. In fact, many of these improvements, especially moving from descriptive to predictive bioprocessing, depend on more digitization.

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Genetic Intelligence, Inc

We build machines that make transcendent medicine by finding and targeting truly causal genetic features in the Whole Genome, even from small sample sizes.

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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.

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