Lab-Grown Engineered Human Mini Livers Offer New Insights into NAFLD Drug Failure

GEN | August 07, 2019

Scientists at the University of Pittsburgh School of Medicine claim to be the first to have successfully grown genetically modified miniature 3D human livers in the laboratory, which mimic the progression of non-alcoholic fatty liver disease NAFLD and can be used to test candidate drugs. The teams proof-of-concept studies using the mini livers are published in Cell Metabolism, and indicate how a human gene that is involved in key metabolic processes regulates fat accumulation in the liver. The studies may also explain why one drug candidate against fatty liver disease that seemed promising in mouse models, wasn’t effective in clinical trials. “This is the first time we can create genetically engineered human mini livers with a disease using stem cells in the lab,” said senior author Alejandro Soto-Gutierrez, MD, PhD, associate professor of pathology at the University of Pittsburgh School of Medicine, and faculty member of the McGowan Institute for Regenerative Medicine and the Pittsburgh Liver Research Center. Soto-Gutierrez and colleagues reported on their achievements in a paper titled, “Generation of Human Fatty Livers Using Custom-Engineered Induced Pluripotent Stem Cells with Modifiable SIRT1 Metabolism.” NAFLD is a complex disorder that can increase the risk of diabetes mellitus, cardiovascular and kidney diseases, and potentially lead to liver failure, the authors explained. Already affecting more than 100 million people in the United States, the incidence of NAFLD is rapidly on the rise in parallel with the global epidemic of obesity and diabetes. Liver failure resulting from progressive NAFLD-related damage and liver cirrhosis is now the second leading indication for liver transplantation. “However, a shortage of donor livers, the high cost of the procedure, and the requirement for patients to remain on lifelong immunosuppression limit the use of transplantation.”

Spotlight

A team of researchers with members from several institutions in France, Brazil, and Sweden has discovered two new strains of giant viruses, which they note have "the most complete translational apparatus of the known virosphere." In their paper published in the journal Nature Communications, the group describes characteristics of the viruses including details about their genomes.

Spotlight

A team of researchers with members from several institutions in France, Brazil, and Sweden has discovered two new strains of giant viruses, which they note have "the most complete translational apparatus of the known virosphere." In their paper published in the journal Nature Communications, the group describes characteristics of the viruses including details about their genomes.

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MEDTECH

Carl Zeiss Meditec and Precise Bio Announce Partnership in the Development and Commercialization of Tissue-Based Implants for Ophthalmology

Precise Bio | July 19, 2022

Carl Zeiss Meditec and Precise Bio, a regenerative medicine company advancing the use of bio-printed tissues and organs, announced today that the companies have entered into a partnership to develop and commercialize fabricated corneal tissue for transplants in patients that require endothelial keratoplasty and natural lenticule transplants for treating keratoconus and vision correction. Under the terms of the agreement, Carl Zeiss Meditec will invest in Precise Bio and fund the further development of Precise Bio's two cornea transplant products and has exclusive worldwide commercialization rights for these products. Financial terms were not disclosed. "This investment in Precise Bio is expected to complement our leading portfolio of cataract and corneal refractive workflow solutions. The technology has the potential to advance treatment options for corneal disease, as well as for elective procedures – furthering enabling optimization of patient care." Euan S. Thomson, PhD, President of Ophthalmic Devices and Head of the Digital Business Unit for Carl Zeiss Meditec "We are very excited with this partnership that builds on our successful, ongoing collaboration with ZEISS for the development of corneal tissues to address unmet needs in the field of ophthalmology," stated Aryeh Batt, Co- Founder and CEO of Precise Bio. "This strategic agreement leverages ZEISS' global leadership in ophthalmology and Precise' innovative and unique 4D bio-fabrication platform technology. We are confident that the synergy between the two companies will allow us to develop breakthrough solutions for recovering patients' eyesight, bringing hope to hundreds of millions of patients worldwide." Precise Bio combines engineering, biomaterials, cell technology, bioengineering, and 3D printing into an unparalleled 4D bio-fabrication platform for transplantable organs and tissues. The Company's proprietary platform technology allows to fabricate tissues by 'printing' cells in a single-cell resolution and spatial accuracy, enabling complex organoid constructs with both structural integrity and long-term cell viability. Overcoming multiple technology challenges, Precise Bio's 4D bio-fabrication technology allows to reliably scale up tissue engineering for clinical use, and produce large quantities in a reproducible, quality controlled and cost-effective process. Precise Bio's ophthalmological pipeline consists of three products, the two cornea products that are the subject of the collaboration with Carl Zeiss Meditec and a retinal implant for age-related macular degeneration, all of which demonstrated successful results in animal models. The Company's ophthalmological products address global markets exceeding $10 billion annually. Bio-fabricated tissues comprised of human cells and natural materials, mimicking the anatomical structure and natural tissue components, can replace damaged or diseased tissue, substitute for donor tissue in cases where there is lack of tissue or solve unmet therapeutic needs. About ZEISS Carl Zeiss Meditec AG AX and TecDAX of the German stock exchange, is one of the world's leading medical technology companies. The Company supplies innovative technologies and application-oriented solutions designed to help doctors improve the quality of life of their patients. The Company offers complete solutions, including implants and consumables, to diagnose and treat eye diseases. The Company creates innovative visualization solutions in the field of microsurgery. With approximately 3,531 employees worldwide, the Group generated revenue of €1,646.8m in fiscal year 2020/2. The Group's head office is located in Jena, Germany, and it has subsidiaries in Germany and abroad; more than 50 percent of its employees are based in the USA, Japan, Spain and France. The Center for Application and Research ore, India and the Carl Zeiss Innovations Center for Research and Development in Shanghai, China, strengthen the Company's presence in these rapidly developing economies. Around 41 percent of Carl Zeiss Meditec AG's shares are in free float. The remaining approx. 59 percent are held by Carl Zeiss AG, one of the world's leading groups in the optical and optoelectronic industries. About Precise Bio Precise Bio develops regenerative medicine therapies that can transform patient care across a wide range of medical indications. Precise Bio's proprietary, break-through 4D bio-fabricating platform overcomes limitations of existing extrusion and ink-jet printers and paves the way for fabricating clinically viable tissues and organs. The Company's initial product pipeline is focused on ophthalmology and includes fabricated functional corneal grafts, intrastromal lenticules for treating keratoconus and vision correction, and a retinal implant for age-related macular degeneration. The Company also has earlier R&D programs to address unmet needs in additional medical indications, which are limited by the number of available donor tissues and organs, or where today's critical health problems can be solved using Precise Bio's naturally-based, hyper-accurate technology.

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DIAGNOSTICS

Meridian Launches New qPCR Master Mixes for Stool Samples to Accelerate Development of Molecular Assays

Meridian | June 21, 2022

Meridian Bioscience, Inc. (NASDAQ: VIVO), a leading global provider of diagnostic testing solutions and life science raw materials, announced today two new sample-specific master mixes, Lyo-Ready™ Direct DNA qPCR Stool Mix, and Lyo-Ready™ Direct RNA/DNA qPCR Stool Mix. These innovative master mixes improve the molecular detection of DNA and RNA from crude fecal specimens while allowing room temperature stabilization of diagnostic assays. Stool samples are used in many gastrointestinal diagnostic tests to screen for harmful bacteria, fungi, viruses, autoimmune conditions, and colorectal cancer (CRC). The fecal occult blood test (FOBT) has been the most widely used gastrointestinal immunoassay to screen CRC for over three decades. However, recent large-scale studies have shown that the FOBT lacks the sensitivity to detect early stages of CRC. Better diagnostic tools are needed to improve assay accuracy, sensitivity, and affordability. Molecular testing is the newest alternative and offers significant improvement in sensitivity and accuracy, especially when multiple targets are detected in the results of a multiplex test. Detection of DNA or RNA from stool is challenging because it contains an array of PCR inhibitors including bile salts, polysaccharides, and catabolic substances. To overcome these challenges, assays typically require expensive and time-consuming sample processing and extraction steps which increase the time to results and cost – ultimately delaying physician diagnosis and patient treatment. Meridian's new Lyo-Ready™ Direct DNA qPCR Stool and Lyo-Ready™ Direct RNA/DNA qPCR Stool Mixes remove the need for time-consuming nucleic acid extraction or lengthy component optimization. This enables test developers to quickly develop faster and more sensitive assays for stool sample analysis. In addition, the mixes can be used in liquid or lyophilized format when ambient temperature assay stabilization is needed, removing the need for cold storage. The use of Lyo-Ready™ Direct qPCR Stool mixes for DNA and RNA is ideal for cancer biomarker detection, early cancer detection, and cancer treatment monitoring where assay sensitivity can be lifesaving. The specific adaptation of our chemistries to stool specimens makes our master mixes the perfect tool for any developer seeking faster new product introduction, shorter time to results, or the ability to transform a wet assay to an ambient temperature-stable assay with minimum developmental time." Florent Chang-Pi-Hin, Ph.D., Vice President of Research & Development - Life Science. About Meridian Bioscience, Inc. Meridian is a fully integrated life science company that develops, manufactures, markets, and distributes a broad range of innovative diagnostic products. We are dedicated to developing and delivering better solutions that give answers with speed, accuracy, and simplicity that are redefining the possibilities of life from discovery to diagnosis. Through discovery and development, we provide critical life science raw materials used in immunological and molecular tests for human, animal, plant, and environmental applications. Through diagnosis, we provide diagnostic solutions in areas including gastrointestinal and upper respiratory infections and blood lead level testing. We build relationships and provide solutions to hospitals, reference laboratories, research centers, veterinary testing centers, physician offices, diagnostics manufacturers, and biotech companies in more than 70 countries around the world.

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CELL AND GENE THERAPY

CSL Plasma Reimagines Donor Experience With First Donations Completed on Innovative Plasma Collection Technology

CSL Plasma | August 08, 2022

CSL Plasma, the plasma collection business of global biotherapeutics leader CSL Behring, began a new era in plasma donations, collecting its first plasma donations using leading-edge plasma collection technology that is expected to significantly reduce average collection time – by approximately one-third – and improve the donor experience. The Rika Plasma Donation System, only available at CSL Plasma and developed by Terumo Blood and Cell Technologies, is designed to reduce collection time, and improve the donor and operator experience. When considering prior average CSL Plasma donation procedure times, this could represent about a 30% reduction in average donation time for donors. The new plasmapheresis system first launched today in the Aurora, Colorado, CSL Plasma location, as part of a phased rollout to CSL Plasma U.S. collection centers. Plasma collected at CSL Plasma facilities is used in the manufacturing of lifesaving plasma-derived therapies. "Today marks a threshold moment for plasma donors following years of dedication, innovation and collaboration – demonstrating how we are driven by our promise in delivering lifesaving and life-extending therapies to patients around the world. We look forward to the positive impact this new technology brings and are thankful for plasma donors who continue to do the amazing every day when they donate at CSL Plasma." CSL Chief Operating Officer Paul McKenzie CSL Plasma believes features of the new Rika system can enable the collection of more plasma, in shorter periods of time, supporting quality and safety, and, ultimately, better serving patients who rely on plasma-based therapies. One of the first plasma donors to experience the Rika system, Kelly Geiser is a resident of the Denver area. She has donated plasma since 2017, and is a Platinum level donor as part of CSL Plasma's donor loyalty program, iGive Rewards®. Geiser appreciated that she was able to complete the donation procedure with the new technology in under 35 minutes and in the fastest donation time she has experienced, while every plasma donor's experience will vary. Donating plasma is also personal for Geiser, who says that therapies made from plasma have helped her and her family, including a parent who recently underwent emergency surgery. "It feels good knowing I'm helping people and, who knows, I might've helped somebody I know," said Geiser. According to Terumo BCT, benefits of the Rika Plasma Donation System include Rika completes one plasma collection in 35 minutes or less on average. The proprietary design of the system's centrifuge maximizes the plasma yield per cycle. The Rika Plasma Donation System was designed to allow no more than 200 milliliters of blood cells outside the donor's body, resulting in a low extracorporeal volume, which may contribute to a more comfortable donor experience. With less than 200 milliliters of blood cells outside the donor's body at any given time, should the initial procedure not be completed, the donor can return and donate without being deferred. The device is designed with an advanced user interface to guide CSL Plasma front-line employees. Operators can quickly and confidently resolve alarms, thanks to guidance provided on the Rika system's touchscreen, which also alerts operators about situations that require attention and provides suggested actions to resolve the situation. The system allows for real-time data analytics to drive device maintenance and repairs. CSL Plasma is implementing the new devices at Colorado centers and then expanding to other U.S. locations, providing donors more opportunities to "do the amazing" and be rewarded for their generosity. "As a long-time leader in our industry, CSL Plasma looks forward to this new plasmapheresis technology to delight our plasma donors and attract new donors while supporting a better operating experience for our employees," said Walter Charles, CSL Plasma Senior Vice President and General Manager. "As we complete the implementation of new technology at U.S. centers, we continue to address the critical and ongoing need for human plasma to produce life-saving medicines for people with serious and rare diseases." About CSL Plasma CSL Plasma operates one of the world's largest and most sophisticated plasma collection networks, with more than 300 plasma collection centers in the U.S., Europe and China. Headquartered in Boca Raton, Florida, CSL Plasma is a subsidiary of CSL Behring, a global biotherapeutics company and a member of the CSL Group of companies. Plasma collected at CSL Plasma facilities is used by CSL Behring for the sole purpose of manufacturing lifesaving plasma-derived therapies for people in more than 100 countries. The parent company, CSL Limited headquartered in Melbourne, Australia, employs more than 25,000 people. About CSL Behring CSL Behring is a global biotherapeutics leader driven by our promise to save lives. Focused on serving patients' needs by using the latest technologies, we discover, develop and deliver innovative therapies for people living with conditions in the immunology, hematology, cardiovascular and metabolic, respiratory, and transplant therapeutic areas. We use three strategic scientific platforms of plasma fractionation, recombinant protein technology, and cell and gene therapy to support continued innovation and continually refine ways in which products can address unmet medical needs and help patients lead full lives.

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