Single CRISPR treatment provides long-term benefits in mice

Medical Xpress | February 18, 2019

Researchers at Duke University have shown that a single systemic treatment using CRISPR genome editing technology can safely and stably correct a genetic disease Duchenne muscular dystrophy (DMD) for more than a year in mice, despite observed immune responses and alternative gene editing outcomes. The study appears online on February 18 in the journal Nature Medicine. In 2016, Charles Gersbach, the Rooney Family Associate Professor of Biomedical Engineering at Duke, published one of the first successful uses of CRISPR to treat an animal model of genetic disease with a strategy that has the potential to be translated to human therapy. Many additional examples have since been published, and several genome editing therapies targeting human diseases are currently in clinical trials, with more on the way. Gersbach's latest research focuses on a mouse model of DMD, which is caused by the body's inability to produce dystrophin, a long protein chain that binds the interior of a muscle fiber to its surrounding support structure.

Spotlight

As we grow and develop, very specific chemical reactions activate and deactivate parts of our genome at strategic times and in specific locations, essentially turning our genes on and off. Epigenetics is the study of these processes and the factors that influence them.So why care about epigenetics if it is only in your cells? What could you possibly do to affect your DNA? It turns out that what you eat, where you live, how you react, when you sleep, if you exercise, and even how you age can affect epigenetics.

Spotlight

As we grow and develop, very specific chemical reactions activate and deactivate parts of our genome at strategic times and in specific locations, essentially turning our genes on and off. Epigenetics is the study of these processes and the factors that influence them.So why care about epigenetics if it is only in your cells? What could you possibly do to affect your DNA? It turns out that what you eat, where you live, how you react, when you sleep, if you exercise, and even how you age can affect epigenetics.

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INDUSTRIAL IMPACT

Sartorius Stedim Biotech completes acquisition of Novasep's chromatography division

Sartorius Stedim Biotech S.A. | February 08, 2022

Sartorius Stedim Biotech, a leading partner of the biopharma industry, announced today that it closed the acquisition of the Novasep chromatography division as of February 7, 2022, following approval by the U.S. Federal Trade Commission. The transaction had already been agreed upon at the beginning of 2021. The business acquired generated sales of around 40 million euros in 2020 at a double-digit profit margin; final figures for 2021 are not yet available. The majority of the approximately 100 employees work at the Pompey site in eastern France and some in the USA, China, and India. The portfolio acquired comprises chromatography systems primarily suited for smaller biomolecules, such as oligonucleotides, peptides, and insulin, as well as innovative systems for continuous manufacturing of biologics. Since 2018, Novasep and Sartorius Stedim Biotech have also been jointly developing optimized systems for a membrane-based chromatography technology. As the acquisition is expected to generate additional non-organic sales revenue growth of around 1 percentage point in 2022, Sartorius Stedim Biotech has updated its sales revenue forecast for the current year as follows: Consolidated sales revenue is now expected to increase by about 15 percent to 19 percent with non-organic growth from acquisitions projected to contribute about 2 percentage points. The company's underlying EBITDA margin remains forecasted at more than 35 percent this year. All forecasts are based on constant currencies. This press release contains forward-looking statements about the future development of the Sartorius Stedim Biotech Group. Forward-looking statements are subject to known and unknown risks, uncertainties and other factors that could cause actual results to differ materially from those expressed or implied by such statements. Sartorius Stedim Biotech assumes no liability for updating such statements in light of new information or future events. Sartorius Stedim Biotech shall not assume any liability for the correctness of this English text. The original French press release is the legally binding version. A profile of Sartorius Stedim Biotech Sartorius Stedim Biotech is a leading international partner of the biopharmaceutical industry. As a total solutions provider, the company helps its customers to manufacture biotech medications safely, rapidly and economically. Headquartered in Aubagne, France, Sartorius Stedim Biotech is quoted on the Eurolist of Euronext Paris. With its own manufacturing and R&D sites in Europe, North America and Asia and an international network of sales companies, Sartorius Stedim Biotech has a global reach. The Group has been annually growing by double digits on average and has been regularly expanding its portfolio by acquisitions of complementary technologies. In 2021, the company employed more than 10,400 people, and earned sales revenue of around 2.89 billion euros according to preliminary figures.

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MEDICAL

Zynex Recognized as Fourth Largest Biotechnology/Bioscience Company in Colorado

Zynex, Inc. | March 24, 2022

Zynex, Inc. (Nasdaq: ZYXI) an innovative medical technology company specializing in the manufacture and sale of non-invasive medical devices for pain management, rehabilitation, and patient monitoring, today announced the Denver Business Journal has recognized Zynex as the fourth largest Biotechnology/Bioscience public company in Colorado based on number of companywide employees as of November 1, 2021. 2021 was a strong year for us with growth on the top and bottom lines, In 2022, we expect sales rep growth and productivity to improve and are seeing momentum in order growth as the economy normalizes post COVID-19. We are positioned for another solid year of revenue growth." Thomas Sangaard, CEO and founder of Zynex Inc. About Denver Business Journal American City Business Journals (ACBJ) is the largest publisher of metropolitan business newsweeklies in the United States, with 44 business publications across the country reaching more than 3.6 million readers each week. About Zynex, Inc. Zynex, founded in 1996, develops, manufactures, markets and sells medical devices used for pain management and rehabilitation as well as non-invasive fluid, sepsis and laser-based pulse oximetry monitoring systems for use in hospitals.

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MEDICAL

Slate Bio Announces the Launch with Seed Financing to Develop Next Generation IL-2 Therapies

Slate Bio | January 20, 2021

Slate Bio, Inc., reports the closing of a $1.75 million venture financing. Epidarex Capital drove the speculation round with cooperation from the UVA Licensing and Ventures Group Seed Fund, Center for Innovative Technology's GAP BioLife Fund, VTC Seed Fund, PharmaDirections, Inc., the board and others. Record is a pre-clinical biotechnology organization creating outlook changing IL-2 mixes dependent on examination directed at the University of Virginia School of Medicine. IL233, Slate's lead candidate, is a first in class drug that initiates constant abatement in animal models of autoimmune and inflammatory diseases. Slate's IL233 is a bifunctional IL-2 combination cytokine that increases Regulatory T cells (Tregs) and tissue resident Tregs for autoimmune and provocative infections. IL233 joins the de-gambling certainty of IL-2 and the collaboration and toughness of IL-33. IL233 goes about as a pharmacological kick off to revive the body's inherent pathways to stop inflammation, end mal-adaptive repair and promote regeneration of damaged tissues to restore homeostasis.

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