Gene Therapy Reduces Obesity & Reverses Type 2 Diabetes in Mice

BioTecNika | August 30, 2019

To overcome the side effects of current anti-obesity drugs, scientists have devised a specific gene silencing therapy against Fabp4- a fatty acid metabolism gene. Obesity affects nearly half a billion people worldwide, including children. According to WHO reports obesity-related diseases, including heart disease, stroke, type 2 diabetes, and cancer, are a leading cause of death. Both genetic and environmental factors cause obesity. The development of effective anti-obesity drugs is required. Presently these drugs exhibit severe off-target effects. In a study published in Genome Research, Jee Young Chung and colleagues developed a gene therapy technique that specifically reduces fat tissue and reverses obesity-related metabolic disease in obese mice. Scientists used a CRISPR interference system wherein catalytically dead Cas9 protein, and single-guide RNA was targeted to white adipocytes with a tissue-specific fusion peptide attached. The complex was internalized with limited toxicity to the cells, and upon internalization, the molecule complex decreased the expression of Fabp4 and reduced lipid storage in adipocytes.

Spotlight

Meet the NeatCell C-Pro application. It automates the mononuclear cell fraction enrichment from diverse cellular products, and is designed for use with a density-gradient medium like Ficoll-Paque media with the Sepax C-Pro instrument and CT-90.1 single-use kit.

Spotlight

Meet the NeatCell C-Pro application. It automates the mononuclear cell fraction enrichment from diverse cellular products, and is designed for use with a density-gradient medium like Ficoll-Paque media with the Sepax C-Pro instrument and CT-90.1 single-use kit.

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INDUSTRIAL IMPACT, MEDICAL

Bicycle Therapeutics Announces a Strategic Collaboration with Novartis to Discover, Develop and Commercialize Bicycle® Radio-Conjugates

Businesswire | March 29, 2023

Bicycle Therapeutics plc a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle®) technology, today announced that it has entered into a strategic collaboration agreement with Novartis to develop, manufacture and commercialize Bicycle® radio-conjugates (BRCs) for multiple agreed upon oncology targets. “This collaboration builds on the groundbreaking clinical work we have been doing in the toxin conjugate field and provides new and additional validation for this unique technology,” said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. “We look forward to working closely with Novartis to pioneer the discovery and development of potential new cutting-edge radiopharmaceutical cancer treatments based on Bicycles. We believe the properties of Bicycles make them well suited for the development of precision guided radiopharmaceuticals and represents the next leg in the application of our proprietary discovery platform in oncology.” Under the terms of the agreement, Bicycle will utilize its proprietary phage platform to discover Bicycles to be developed into BRCs. Novartis will be responsible for further development, manufacture and commercialization of the BRCs. Novartis will fund all pre-clinical and clinical development and commercialization activities. Bicycle will receive a $50 million upfront payment and is eligible for development and commercial-based milestone payments totaling up to $1.7 billion. Bicycle will also be eligible to receive tiered royalties on Bicycle-based medicines commercialized by Novartis. About Bicycle Therapeutics Bicycle Therapeutics is a clinical-stage biopharmaceutical company developing a novel class of medicines, referred to as Bicycles, for diseases that are underserved by existing therapeutics. Bicycles are fully synthetic short peptides constrained with small molecule scaffolds to form two loops that stabilize their structural geometry. This constraint facilitates target binding with high affinity and selectivity, making Bicycles attractive candidates for drug development. Bicycle is evaluating BT5528, a second-generation Bicycle Toxin Conjugate targeting EphA2; BT8009, a second-generation BTC targeting Nectin-4, a well-validated tumor antigen; and BT7480, a Bicycle TICA™ targeting Nectin-4 and agonizing CD137, in company-sponsored Phase I/II trials. In addition, BT1718, a BTC that targets MT1-MMP, is being investigated in an ongoing Phase I/IIa clinical trial sponsored by the Cancer Research UK Centre for Drug Development. Bicycle is headquartered in Cambridge, UK, with many key functions and members of its leadership team located in Cambridge, MA.

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DIAGNOSTICS

PhenomeX Launches Beacon Quest™ Optofluidic System with Opto® T Cell Profiling Workflows

PRNewswire | June 06, 2023

PhenomeX Inc. the functional cell biology company, announced the launch of the Beacon Quest™ optofluidic system, with Opto® T Cell Profiling workflows for immunotherapy translational research and a platform price under $1 million USD, less than half of the higher-throughput Beacon system, enabling broader accessibility to the cutting-edge technology critical for next-generation precision medicine. The Opto T Cell Profiling workflows have enabled scientists to comprehensively profile single T cells to correlate polyfunctionality with cytotoxicity and recover those same cells for downstream analysis such as transcriptome and genome sequencing, revealing functional connections of phenotype with molecular mechanism. This depth of understanding of biology and response to therapeutics is not available on any other platform. Combined with the value-matched Beacon Quest system, more academic, non-profit, and government customers can now access the power of optofluidic and NanoPen® chamber technology and propriety single T Cell Profiling applications along with the optimum level of features to meet their research and budgetary needs. "At PhenomeX, we are excited to launch the Beacon Quest, as it supports our objective of offering scientists and researchers globally the ability to affordably access the main applications of the Beacon system, including antibody discovery, cell line development and single-cell functional multiomic T-cell profiling," said Siddhartha Kadia, Ph.D., chief executive officer of PhenomeX. "We look forward to partnering with and providing significant value in the academic research segments, particularly in immuno-oncology translational cancer centers and innovative cell and gene therapy development centers." Beacon Quest for T Cell Profiling Workflow Features Opto T Cell Profiling workflows on the Beacon Quest system enable multi-functional characterization of single T cells, including for development of efficacious immunotherapies and cell-based cancer therapeutics, that are ideally suited for academic, non-profit, and government customers. The Beacon Quest system enables a variety of assays to profile single T cells including detection of cytokine secretion, cytotoxicity, cell surface markers, transcriptome profiling and TCRSeq, with the potential to study growth characteristics and cell motility. The resulting analysis is used to identify cells with phenotypes of interest and guide recovery of the same cells for downstream assays, enabling researchers to gain a deeper understanding of the underlying biology by Investigating molecular mechanisms associated with polyfunctional T cells; Identifying desirable phenomes by correlating polyfunctionality with cytotoxicity; and Linking desirable phenotypes to T cell receptor sequences of the same cells. In addition to supporting the Opto T Cell Profiling workflows, Beacon Quest will enable customers to access the Opto B Discovery workflows and Opto Cell Line Development workflows. To further increase customer accessibility and affordability, PhenomeX will also offer reduced priced OptoSelect® chips (OptoSelect® Research chips) for Beacon Quest, helping academic customers accelerate the pace of scientific discovery for clinical research use applications. About PhenomeX Inc. PhenomeX is empowering scientists to leverage the full potential of each cell and drive the next era of functional cell biology that will advance human health. We enable scientists to reveal the most complete insights on cell function and obtain a full view of the behavior of each cell. Our unique suite of proven high-throughput tools and services offer unparalleled resolution and speed, accelerating the insights that are key to advancing discoveries that can profoundly improve the prevention and treatment of disease. Our award-winning platforms are used by researchers across the globe, including those at the top 15 global pharmaceutical companies and approximately 85% of leading U.S. comprehensive cancer centers.

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MEDTECH, MEDICAL

Twist Bioscience Launches Portfolio of RNA Sequencing Tools

Businesswire | May 23, 2023

Twist Bioscience Corporation a company enabling customers to succeed through its offering of high-quality synthetic DNA using its silicon platform, today announced the launch of a portfolio of RNA sequencing tools, which includes the Twist RNA Exome, Twist RNA Library Prep Kit and the Twist Ribosomal RNA (rRNA) & Hemoglobin (Globin) Depletion Kit, as well as custom target enrichment capabilities for RNA and whole transcriptome sequencing. This comprehensive suite of RNA tools can enable targeted or whole transcriptome research across fields including precision medicine, biomarker discovery and immuno-oncology research. “Twist’s RNA sequencing portfolio builds on our established NGS capabilities in genomic sequencing and methylation detection and expands into gene expression analysis, opening a new and significant market for Twist,” said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. “This offering allows our customers to leverage Twist’s leading target enrichment platform to discover and resolve low abundance RNA transcripts in the human transcriptome. Our RNA sequencing workflows are designed specifically for challenging samples. By enriching relevant transcripts, we can enable researchers to get high-quality data from a range of sample types, including damaged RNA.” Unlike the genome, which is relatively static, the transcriptome changes over time in response to varying disease states and drug therapies. Gene expression levels also differ across tissues and cell types. To fully understand this dynamic quality of RNA, researchers often sequence RNA samples from the same source at multiple time points, incurring additional costs and resource burdens. Each component of the Twist RNA sequencing portfolio is purpose-built to maximize data quality, minimize workflow inefficiency, decrease wasted sequencing reads, and support comprehensive transcriptomic profiling across even low-quality and low-input samples common in oncology. The Twist RNA sequencing portfolio provides end-to-end workflows that can be used to study total RNA and measure the whole transcriptome, protein coding sequences of messenger RNA (mRNA) or custom targets. These products can also be integrated into customers’ existing workflows. Twist RNA sequencing tools enable minimal hands-on time and increase the scale of samples sequenced through utilizing automation. They are compatible with a wide range of sample inputs, including difficult or low-quality samples. RNA panel designs can also now incorporate Twist’s new exon-aware design algorithm, which reduces bias in detection and can provide an important tool for researchers studying rare gene transcripts that drive tumor biology. Twist Targeted RNA Sequencing Workflow Twist target enrichment for RNA leverages the performance, efficiency and sensitivity of Twist target enrichment for sequencing of RNA transcripts of interest across samples and species. It can be used to enrich relevant transcripts with the RNA Exome or in custom RNA panels. Twist RNA Exome The Twist RNA Exome with Twist’s exon-aware design algorithm enables targeted sequencing of the protein coding regions of the human transcriptome without bias toward specific combinations of exons. The Twist RNA Exome covers up to 99.9% of protein coding sequencing in Gencode and RefSeq databases. This coverage, along with the exon-aware design approach, could enable researchers to capture important transcripts including those in low input and degraded samples from formalin fixed paraffin embedded (FFPE) sections. Twist Whole Transcriptome Sequencing Workflow Twist Whole Transcriptome Sequencing can be used to discover novel transcripts, including low expressing genes and novel isoforms. It can be used with a variety of samples including whole blood, fresh and frozen tissue samples and low quality samples, such as FFPE samples. The Twist rRNA & Globin Depletion Kit can be used with this workflow to deplete rRNA and hemoglobin targets so that researchers can study the rest of the transcriptome. The full workflow can be completed in less than five hours with minimal hands-on time. About Twist Bioscience Corporation Twist Bioscience is a leading and rapidly growing synthetic biology and genomics company that has developed a disruptive DNA synthesis platform to industrialize the engineering of biology. The core of the platform is a proprietary technology that pioneers a new method of manufacturing synthetic DNA by “writing” DNA on a silicon chip. Twist is leveraging its unique technology to manufacture a broad range of synthetic DNA-based products, including synthetic genes, tools for next-generation sequencing (NGS) preparation, and antibody libraries for drug discovery and development. Twist is also pursuing longer-term opportunities in digital data storage in DNA and biologics drug discovery. Twist makes products for use across many industries including healthcare, industrial chemicals, agriculture and academic research.

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