Nippon Shinyaku and Capricor Therapeutics Enter into Exclusive Partnership for Commercialization and Distribution of Exosome-Secreting Cardiosphere-Derived Cells (CAP-1002) for the Treatment of Duchenne Muscular Dystrophy in US

On January 25, 2022, Nippon Shinyaku Co., Ltd. (Headquarters: Kyoto, Japan, President: Toru Nakai) announced that Nippon Shinyaku and Capricor Therapeutics (Headquarters: California, USA, CEO: Linda Marbán, NASDAQ: CAPR) have entered into an exclusive distribution agreement for CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD) in the territory of the US.

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Gene Therapy for Treatment of Huntington’s Disease (HD) Shows Signs of Benefit in Pre-Clinical Studies

Gene therapy targeting the messenger RNA (mRNA) of the mutated huntingtin gene (HTT) can provide long-lasting therapeutic benefit in Huntington’s disease after a single administration. An adeno-associated virus (AAV) gene therapy containing a primary artificial microRNA (pri-amiRNA) targeting HTT mRNA is described in the peer-reviewed journal Human Gene Therapy. The January 17, 2022 open-access article is titled “Efficient and Precise Processing of the Optimized Primary Artificial MicroRNA in a Huntingtin-Lowering Adeno-Associated Viral Gene Therapy In Vitro and in Mice and Nonhuman Primates.”

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Hippos Recognize Each Other’s Voices, Respond Differently to Calls of Strangers

Hippopotamuses are rather vocal animals. Their “wheeze honk” calls can be heard over long distances, leading researchers to suspect the calls play an important role in maintaining social groups. Now, a study in published in Current Biology on January 24, 2022 shows that hippos recognize each other’s voices. They also respond less aggressively to the calls of a neighbor compared to those of a stranger. The open-access article is titled “Voice-Mediated Interactions in a Megaherbivore.”

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Univercells Technologies Partners with RoosterBio to Advance Scalable and Continuous Manufacture of Exosomes

On January 20, 2022, Univercells Technologies, a leading provider of novel biomanufacturing technologies for flexible and scalable viral production, announced a strategic partnership with RoosterBio Inc., a leading supplier of human mesenchymal stem/stromal cells (hMSCs), highly engineered media, and hMSC bioprocess systems. This partnership will optimize manufacturing of extracellular vesicles (EVs) using scalable and continuous bioprocessing technologies to propel the commercialization of regenerative therapies at affordable costs. The goal is to deliver complete solutions for exosome manufacture, leveraging Univercells Technologies’ intensified and integrated platforms and RoosterBio’s hMSC bioprocess and highly engineered media systems. As part of the partnership, RoosterBio will establish an EV production process using their hMSCs on microcarriers in the scale-X™ hydro fixed-bed bioreactor which features 2.4 m² surface for cell growth.

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First Clinical-Grade Transplant of Gene-Edited Pig Kidneys into Brain-Dead Human Announced; Achievement Termed “Game-Changing Moment in the History of Medicine”

On January 20, 2022, the University of Alabama at Birmingham Marnix E. Heersink School of Medicine announced the first peer-reviewed research outlining the successful transplant of genetically modified, clinical-grade pig kidneys into a brain-dead human individual, replacing the recipient’s native kidneys. These positive results demonstrate how xenotransplantation could address the worldwide organ shortage crisis. In the study published on January 20, 2022 in the American Journal of Transplantation, UAB researchers tested the first human preclinical model for transplanting genetically modified pig kidneys into humans. The study recipient had two genetically modified pig kidneys transplanted in his abdomen after his native kidneys were removed. The organs were procured from a genetically modified pig at a pathogen-free facility. The open-access article is titled “First Clinical-Grade Porcine Kidney Xenotransplant Using a Human Decedent Model.”

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Research Illuminates How Melanoma Readies Lymph Nodes for Tumor Spread; Tumor-Secreted Exosomes Carry Nerve Growth Factor Receptor (NGFR) to Lymph Nodes, Priming the Nodes for Tumor Metastasiss

Melanoma cells release small extracellular packages (exosomes) containing the protein nerve growth factor receptor (NGFR), which primes nearby lymph nodes for tumor metastases, according to a new study by Weill Cornell Medicine investigators. The study results, published on November 25, 2022 in Nature Cancer, may one day help doctors determine which patients need more aggressive treatment and could help with the development of new therapies, said senior author, David Lyden, MD, PhD, the Stavros S. Niarchos Professor in Pediatric Cardiology and a Professor of Pediatrics and of Cell and Developmental Biology at Weill Cornell Medicine. The article is titled “Melanoma-Derived Small Extracellular Vesicles Induce Lymphangiogenesis and Metastasis Through an NGFR-Dependent Mechanism.”

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CNIO Researchers Identify a Mechanism That Causes Liver Cirrhosis; Breakthrough Achieved Thanks to Development of First Animal Model–A Genetically Modified Mouse–with Liver Cirrhosis Similar to That Observed in Humans

Liver cirrhosis is a deadly disease that is still poorly understood, in large part due to the lack of animal models that would allow it to be studied. The Centro Nacional de Investigaciones Oncológicas (CNIO) group led by Nabil Djouder, PhD, has created the first genetically modified mouse that develops liver cirrhosis comparable to human cirrhosis and has thus managed to identify the molecular mechanisms underlying this disease. “Determining the molecular mechanisms causing cirrhosis will help us to understand how it progresses to liver cancer,” Dr. Djouder says. The paper was published on December 24, 2022 in the Journal of Hepatology, a high-impact journal in the field. The article is titled “Histone Acetylation of Bile Acid Transporter Genes Plays a Critical Role in Cirrhosis.”

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Nanotherapy Offers New Hope for Treatment of Type 1 Diabetes; Approach Might Have Far-Reaching Implications

Individuals living with Type 1 diabetes must carefully follow prescribed insulin regimens every day, receiving injections of the hormone via syringe, insulin pump or some other device. And without viable long-term treatments, this course of treatment is a lifelong sentence. Pancreatic islets control insulin production when blood sugar levels change, and in Type 1 diabetes, the body’s immune system attacks and destroys such insulin-producing cells. Islet transplantation has emerged over the past few decades as a potential cure for Type 1 diabetes. With healthy transplanted islets, Type 1 diabetes patients may no longer need insulin injections, but transplantation efforts have faced setbacks as the immune system continues to eventually reject new islets. Current immunosuppressive drugs offer inadequate protection for transplanted cells and tissues and are plagued by undesirable side effects. Now a team of researchers at Northwestern University has discovered a technique to help make immunomodulation more effective. The method uses nanocarriers to re-engineer the commonly used immunosuppressant rapamycin. Using these rapamycin-loaded nanocarriers, the researchers generated a new form of immunosuppression capable of targeting specific cells related to the transplant without suppressing wider immune responses.

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Epilepsy Research Strengthens Case for New Gene Therapy for Dravet Syndrome

Research from the University of Virginia (UVA) School of Medicine suggests how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, and potentially prolong survival for people with the condition. The gene therapy, developed by Stoke Therapeutics, is now in clinical trials. Because most Dravet syndrome cases are caused by a mutation in the SCN1A gene, resulting in a reduction in SCN1A protein production, the novel approach is designed to boost production of SCN1A to normal levels. If successful, the approach, called Targeted Augmentation of Nuclear Gene Output (TANGO),  would be the first treatment for the fundamental cause of the disease, a lack of this particular protein in specialized brain cells. The new research–from UVA’s Manoj K. Patel, PhD, and Eric R. Wengert, PhD, and their collaborators–demonstrates how the experimental therapy restores the cells’ proper function and reduces seizures in lab mice. 

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Respiratory Viruses That Hijack Immune Mechanisms May Have Achilles’ Heel

One viral protein could provide information to deter pneumonia causing the body’s exaggerated inflammatory response to respiratory viruses, including the virus that causes COVID-19. That viral protein is NS2 of respiratory syncytial virus (RSV), and a study has found that if the virus lacks this protein, the human body’s immune response can destroy the virus before exaggerated inflammation begins. The research, conducted at Washington State University’s College of Veterinary Medicine, was published on January 18, 2022 in the journal MBio. The open-access article is titled “Human Respiratory Syncytial Virus NS2 Protein Induces Autophagy by Modulating Beclin1 Protein Stabilization and ISGylation.”

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