A CRISPR cure for Huntington’s?

The gene editing system CRISPR-Cas9 has generated excitement in scientific circles for its potential to cure diseases caused by a single defective gene, including the progressive neurological disorder Huntington’s. But editing genes with this technology is risky because cutting strands of DNA can lead to unintentional gene edits, causing dangerous off-target effects.

Spotlight

Epygen

EPYGEN is an Biotechnology organization, headquartered in Dubiotech Park, Dubai, UAE, with offices, labs and manufacturing facilities in Dubai, India and USA. Following the essence of it's corporate slogan “Building Future Strand by Strand”, Epygen conducts research and manufacturing of wide range of Enzymes Proteins for the Therapeutic as well as Industrial Enzyme space.

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MedTech

Nanostructures: Emerging as Effective Carriers for Drug Delivery

Article | September 22, 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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MedTech

Making Predictions by Digitizing Bioprocessing

Article | October 7, 2022

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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Research

AI and Biotechnology: The Future of Healthcare Industry

Article | July 11, 2022

Artificial intelligence has grasped the foundation in biotech. It can have the most innovative impact on biotechnology. AI has already established its presence in our day-to-day life. AI has made the existence of self-driving cars possible. Likewise, the benefits and quality that it can contribute to biotech can also be felt. With AI, bio technicians will be able to enhance virtual screening, overlook preliminary datasets from clinics, and decipher an enormous amount of information. It can also help in improving the medication process by gathering and analyzing every bit of information. The Significance of AI in Biotechnology In the past few years, the application of artificial intelligence in the biotechnology industry has shifted from being sci-fi to sci-fact. A vast number of biotech companies like Deep Genomics are adopting AI for making data-driven decisions and use analytics tools to work efficiently. Unlike the AI robots in sci-fi that are ready to take over the world. AI designed for biotech has been designed to solve certain problems or complete a bunch of tasks by using automated algorithms. The aim of AI technology for biotech is to collect insights along with hidden patterns from large amounts of data. All the different industries of biotech including agriculture, animal, medical, industrial, and bioinformatics are gradually being affected by artificial intelligence. Moreover, the biotech industry is realizing that AI enables them some of the important strength to their business, including: Expanding accessibility Cost-effectiveness Critical predictions Efficient decision-making Research centers like PwC have also estimated output of $15.7 trillion by 2030 solely with AI contribution in industries. A survey revealed that about 44% of life science experts are using AI for R&D activities, as well. Use of AI in Biotechnology Altering Biomedical and Clinical Data So far the most developed use of AI is its ability to read voluminous data records and interpret them. It can prove to be a life-save for bio technicians who would have to examine that much data from research publications by themselves for the validation of their hypothesis. With the help of AI, clinical studies of patients will also become easier as all the examination reports and prescriptions will be stored in one place for cross-reference. Furthermore, it will also help in blending and fetching data into usable formats for analysis. Test Result Prediction Through trial and error, AI along with machine learning can help in predicting the response of the patient to certain drugs to provide more effective outcomes. Drug Design & Discovery AI plays a vital role whether it’s designing a new molecule or identifying new biological targets. It helps in identifying and validating drugs. It reduces the cost and time spent on the entire drug trial process and reaches the market. Personalized Medications for Rare Diseases With the combination of body scan results, patients’ body and analytics, AI can also help in detecting dangerous diseases at an early stage. Improving Process of Manufacturing To improve the process of manufacturing in biotechnology, AI offers a wide range of opportunities. It controls quality, reduces wastage, improves useability, and minimizes the designing time. Moving Towards AI-Enhanced Biotech Future Ever since the concept of artificial intelligence has arrived, being curious by nature, humans have started working towards achieving this goal. It has been growing at a fast pace while showing unbelievable growth and achievements at times. In comparison to the traditional methods used in the biotechnology industry, AI-based methods seem more reliable and accurate. In the upcoming years, it will show its success by improving the quality of health people have. You can also develop your AI-based application or know more about it by taking IT consultations.

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Medical

Advancement in Genomics Accelerating its Penetration into Precision Health

Article | June 22, 2022

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

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Spotlight

Epygen

EPYGEN is an Biotechnology organization, headquartered in Dubiotech Park, Dubai, UAE, with offices, labs and manufacturing facilities in Dubai, India and USA. Following the essence of it's corporate slogan “Building Future Strand by Strand”, Epygen conducts research and manufacturing of wide range of Enzymes Proteins for the Therapeutic as well as Industrial Enzyme space.

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.

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