Transposable Elements Role in Genetic Expression Could Improve Study of Neurodegenerative Disease

GEN | April 15, 2020

Researchers in Michigan published a study “Transposable elements contribute to cell and species-specific chromatin looping and gene regulation in mammalian genomes” in Nature Communications that shows that transposable elements play an important role in regulating genetic expression with implications to advance the understanding of genetic evolution, with potential applications in neurodegenerative diseases. Transposable elements move around the cell, and, unlike previously thought, the authors of this paper found that when they go to different sites, transposable elements sometimes change the way DNA strands interact in 3D space, and therefore the structure of the 3D genome. It appears a third of the 3D contacts in the genome actually originate from transposable elements leading to an outsized contribution by these regions to looping variation and demonstrating their very significant role in genetic expression and evolution.

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We are living in a unique time in which the world is changing faster than ever. This largely is due to an influx and infusion of digital technologies that are reinventing and reimagining businesses across nearly every sector, and dramatically shifting societal and economic interactions. While the life science industry has, to some extent, adopted various digital technologies, it has not had a significant digital transformation in how it does business.


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CELL AND GENE THERAPY

Mission Bio Announces Pharma Assay Development Services for Solid Tumor Research

Mission Bio | July 18, 2022

Mission Bio, Inc., the pioneer in high-throughput single-cell DNA and multi-omics analysis, announced the launch of solid tumor assays through its Pharma Assay Development (PAD) services. The availability of this new service offering will help to accelerate the development of cancer therapeutics by reducing the time and cost associated with the characterization of solid tumor cells. Precision therapeutics for solid tumors have been historically bottlenecked due to technical challenges associated with the ability to deeply profile thousands of individual cells for a rich characterization of tumor evolution and heterogeneity. This must be done at a much higher resolution compared to bulk next-generation sequencing to enable detection of rare events, at a much earlier stage before progression. Utilizing Mission Bio's Tapestri™ platform, the company's PAD services for solid tumors partner with researchers to provide high-resolution data to unmask the underlying genetic diversity across cell populations. Insights into the clonal landscape and co-occurrence of mutations enable improved patient stratification for clinical trials and the identification of druggable targets for precision therapeutics. Researchers can also monitor treatment resistance by analyzing the acquisition of rare mutations driving tumor progression over the course of treatment. The expansion of Mission Bio's PAD services to solid tumor research comes just three months after the launch of the Solid Tumor Solution on the Tapestri™ Platform, demonstrating Mission Bio's commitment to continuously developing innovative single-cell technologies for its pharma customers. "We have seen tremendous uptake and interest from top-tier pharma companies for our blood cancer Pharma Assay Development program, and we expect the same momentum for our solid tumor services. With our solid tumor services, Mission Bio partners with pharma customers to elucidate the mechanisms of drug resistance through mutation acquisition, determine how cells transform from benign to malignant states, and reveal the genomic changes enabling cancer cells to metastasize – without having to bring single-cell technology or resources in-house. From a customer's perspective, it is as easy as shipping out samples and getting a fully analyzed report back." Todd Druley, MD, PhD, Chief Medical Officer of Mission Bio Mission Bio's PAD services deliver comprehensive support across the therapeutic development process. The company has a dedicated team that works with researchers to develop assays, identify high-impact samples, and analyze the data. As part of the service, pharma partners have access to Mission Bio's innovative technology, assay development team, R&D organization, and bioinformatics support. About Mission Bio Mission Bio is a life sciences company that accelerates discoveries and cures for a wide range of diseases by equipping researchers with the tools they need to better measure and predict our resistance and response to new therapies. Mission Bio's multi-omics approach improves time-to-market for new therapeutics, including innovative cell and gene therapies that provide new pathways to health. Founded in 2014, Mission Bio has secured investment from Novo Growth, Cota Capital, Agilent Technologies, Mayfield Fund, and others. The company's Tapestri™ platform gives researchers around the globe the power to interrogate every molecule in a cell together, providing a comprehensive understanding of activity from a single sample. Tapestri™ is the only commercialized multi-omics platform capable of analyzing DNA and protein simultaneously from the same sample at single-cell resolution. The Tapestri™ Platform is being utilized by customers at leading research centers, pharmaceutical, and diagnostics companies worldwide to develop treatments and eventually cures for cancer.

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MEDICAL

ABC Opens Commercial MEL BioSurfactant Production in Irvine, CA

ABC | May 19, 2022

Advanced BioCatalytics (ABC) is pleased to announce the opening of our new biosurfactant commercial pilot plant in Irvine California. The plant reflects the successful collaboration of the ABC R&D team and Dr. Wenjie Xia, professor of Microbiology at Nankai University in Tianjin China. The commercial pilot plant is the first step in commercial scale up of new glycolipid (biosurfactants) and is the first plant to produce commercial quantities of mannosylerythritol lipids (MEL) in North America. ABC pioneered the use of microbiology to produce metabolites and sustainable chemistry in 1996. Our products are used in water treatment, the energy sector, for agriculture uses, and in personal and home care applications. By using microbiology, we can create natural alternatives to petrochemicals. For our production we upcycle waste feed stocks such as used vegetable oils or other sources of bio-waste material. As a result, our chemistry also offers an alternative to plant based materials which have the unfortunate reliance on certain plant species which are contributing to global deforestation and destruction of biodiversity. The MEL material can be used in a variety of ways in the homecare and personal care sectors. MEL is free of 1,4 dioxane, mild on the skin, and produces minimal foam. This superfatting agent is ideal for delivering moisture in personal care products and can be used along with anionic and amphoteric surfactants for improved efficacy. The addition of MEL production compliments ABC's rhamnolipid and BioSS RL fermactant production. About Advanced BioCatalytics: Advanced BioCatalytics is an industrial biotechnology company that leverages its innovative Fermactant technology for applications in the Home and Personal Care, Energy, Agriculture, and Wastewater markets. These products have demonstrated the ability to be highly cost-effective, without depending on harmful chemistries such as caustics, acids or dangerous solvents, which can be harmful to the user and/or the environment. Advanced BioCatalytics was founded in 1996 and we are excited to be introducing a new technology that will change the future performance, and cost, of the world's cleaning products.

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INDUSTRIAL IMPACT

ThinkCyte's Shares Applications of Ghost Cytometry with Industrial and Academic Thought Leaders

ThinkCyte Inc. | July 12, 2022

ThinkCyte Inc., a biotechnology company pioneering novel cell analysis and sorting instruments, announced that applications of its Ghost Cytometry technology were presented at several leading industry meetings. In May, the company was selected to present applications of the platform to experts in drug discovery at the prestigious Keystone Symposia. The company was also selected to deliver a podium presentation at the 2022 International Association for the Advancement of Cytometry's annual meeting, sharing applications in clinical medicine and cellular therapy with a talk entitled 'Machine vision-based label-free cell analysis and sorting for clinical diagnosis and cell therapy.' The company and its Ghost Cytometry platform were also selected as one of three finalists for the Technology Showcase at CYTO, which highlights innovative and disruptive cytometric technologies. "We are incredibly pleased to be able to share our most recent work using Ghost Cytometry with the broader scientific community. It's an exciting time to be in the field of drug and cellular therapy with innovative approaches being developed to extract novel information from cells. We look forward to showcasing more of our internal work and collaborations with existing partners in the months ahead." ThinkCyte's CEO Waichiro Katsuda Ghost Cytometry is a novel flow-cytometry based platform for cell analysis and sorting. Using a combination of proprietary optical designs and artificial intelligence, Ghost Cytometry provides researchers with an entirely new data lens for capturing complex single-cell phenotypes into a "cellular fingerprint" and enables the isolation of live target cells, untouched by external labels, for downstream research and development applications. About ThinkCyte Inc. ThinkCyte, founded in 2016 with offices in Tokyo, Japan and San Carlos, California is a biotechnology company that develops innovative instruments based on integrated, multidisciplinary technologies to enable life science research, diagnostics, and therapeutic development. The company pioneered Ghost Cytometry, a proprietary AI-based, label-free cell sorting technology and works with major global biopharmaceutical companies and leading academic research institutes to further drive pioneering research.

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INDUSTRIAL IMPACT

Iveric Bio and DelSiTech Enter Exclusive Agreement for Development of Sustained Release Zimura®

IVERIC bio, Inc. | July 06, 2022

IVERIC bio, Inc. and DelSiTech Ltd, announced an exclusive global license agreement providing Iveric Bio with the right to develop and commercialize new formulations of Zimura® using DelSiTech’s silica-based sustained release technology. As part of Iveric Bio’s lifecycle expansion plan for Zimura, the Company is committed to developing sustained release technologies for the treatment of age-related macular degeneration (AMD). These technologies potentially could address patients being treated for geographic atrophy (GA) and intermediate AMD. “We are thrilled to collaborate with the DelSiTech team on investigating additional sustained release formulations for Zimura with their drug delivery expertise and advanced technology and look forward to evaluating a sustained release formulation in GA and potentially earlier stages of AMD. Previously reported post-hoc analyses from GATHER1 suggest that Zimura may have the potential to impact AMD in early stages before atrophy occurs in patients. We believe Zimura, which is a chemically synthesized RNA aptamer, is amenable to injectable sustained release formulations.” Pravin U. Dugel, MD, President of Iveric Bio “As a leading developer of long-acting controlled release formulations for small molecules and biological entities, we are excited about collaborating with Iveric Bio on bringing innovative solutions to patients living with AMD,” stated Lasse Leino, PhD, Chief Executive Officer of DelSiTech. “We are inspired by the opportunity to leverage our drug delivery technology to potentially help AMD patients early and improve their treatment experience.” “This agreement underscores our commitment to invest in lifecycle initiatives for Zimura,” said Glenn P. Sblendorio, Chief Executive Officer of Iveric Bio. “We are excited about the possibilities to expand Zimura into earlier stages of AMD and potentially allow for a next-generation treatment to help patients with GA.” “We are proud to bring our competencies into this promising alliance and contribute to Iveric Bio’s mission to address unmet needs for patients,” said Dr. Frederic Dargelas, Head of Business Development and Alliance Management for DelSiTech. Under the terms of the license agreement, Iveric Bio will pay DelSiTech an upfront payment of €1,250,000, as well as development and commercial milestones and royalties on net sales of licensed products. In addition to working with DelSiTech, Iveric Bio plans to explore the potential for Zimura in earlier stages of AMD by initiating a clinical trial studying the current formulation of Zimura in patients with intermediate AMD in the fourth quarter of 2022. The development strategy in this indication is subject to global regulatory feedback from the U.S. Food and Drug Administration (FDA) and other regulatory authorities, which Iveric Bio plans to obtain before initiating this trial. About Zimura GATHER1 and GATHER2 Clinical Trials Iveric Bio previously announced that GATHER1, the Company’s first Phase 3 clinical trial for Zimura (avacincaptad pegol) for GA, met its pre-specified primary efficacy endpoint with statistical significance. The most frequently reported ocular adverse events in this trial were related to the injection procedure. The Company expects topline data for GATHER2, the Company’s second Phase 3 clinical trial for Zimura for GA, to be available in the third quarter of 2022, approximately one year after the enrollment of the last patient in the trial plus the time needed for database lock and analysis. If 12-month results from GATHER2 are positive, the Company plans to submit applications with the FDA and the European Medicines Agency for marketing approval of Zimura for GA. There are no FDA or EMA approved treatments available for patients with GA. About Zimura Zimura is an investigational drug product and has not been approved for use anywhere globally. Zimura is designed to target and inhibit the cleavage of complement protein C5 and the formation of its downstream fragments, C5a and C5b. By inhibiting the formation of these fragments, Zimura is believed to decrease or slow the chronic inflammation and cell death associated with the retinal aging process by decreasing the formation of membrane attack complex (MAC) and inflammasome activity, thereby potentially avoiding or slowing the degeneration of retinal pigment epithelial cells. This potential mechanism is the rationale for Zimura as a potential therapy for GA and earlier stages of AMD. About Iveric Bio Iveric Bio is a science-driven biopharmaceutical company focused on the discovery and development of novel treatments for retinal diseases with significant unmet medical needs. The Company is committed to having a positive impact on patients’ lives by delivering high-quality, safe and effective treatments designed to address debilitating retinal diseases including earlier stages of age-related macular degeneration. About DelSiTech DelSiTech Ltd., located in Turku, Finland, is the leading technology specialist in biodegradable silica-based controlled release of small molecule drugs, biologics, and viral vectors. It develops and commercializes its proprietary, drug delivery technology in collaboration with a number of pharma and biotech companies to turn their ideas into novel drug products.

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Spotlight

We are living in a unique time in which the world is changing faster than ever. This largely is due to an influx and infusion of digital technologies that are reinventing and reimagining businesses across nearly every sector, and dramatically shifting societal and economic interactions. While the life science industry has, to some extent, adopted various digital technologies, it has not had a significant digital transformation in how it does business.

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