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Archive - Apr 18, 2020

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ReNeuron Announces Exosome Research Collaboration; Agrees to Produce Exosomes for the Delivery of Gene-Silencing Sequences

On April 7, 2020, ReNeuron Group plc (AIM: RENE), a UK-based global leader in the development of cell-based therapeutics, announced that it has signed a research agreement with an unnamed major pharmaceutical company to explore the potential use of the Company's proprietary exosomes to deliver novel therapeutics. The agreement follows the Company's strategy of collaborating with pharmaceutical and biotechnology companies to use its exosome technology as a novel delivery vehicle. ReNeuron's exosomes are derived from its CTX neural stem cell line and have the ability to cross the blood brain barrier and to be manufactured at scale. The research collaboration will focus on the use of the Company's exosomes for the delivery of gene silencing sequences created by the pharmaceutical company. ReNeuron will be responsible for manufacturing exosomes and then loading them with the gene silencing sequences after which the pharmaceutical company will evaluate the loaded exosomes. ReNeuron will be paid by the pharmaceutical company for manufacturing and loading the exosomes in the initial phase of the collaboration. This is ReNeuron's second research collaboration following the signing of an ongoing agreement with a U.S. pharmaceutical company in January 2019. "We are delighted to have signed this latest exosome research collaboration agreement with an experienced leader in the discovery and development of novel gene silencing-based therapeutics," commented Olav Hellebø, MBA, Chief Executive Officer of ReNeuron.

Capricor Initiates Compassionate Use Program for Severe COVID-19 Patients Using CAP-1002, Its Novel Cell Therapy Based on Cardiosphere-Derived Cells; CDCs Release Exosomes That May Mitigate Severe Inflammatory Response Associated with COVID-19

On April 3, 2020, Capricor Therapeutics (NASDAQ: CAPR) a clinical-stage biotechnology company focused on the development of first-in-class biological therapeutics for the treatment or prevention of serious diseases, announced it is providing CAP-1002, its novel cell therapy to patients with advanced COVID-19 under the compassionate use pathway. Two patients were treated in the previous week at a leading healthcare center in Los Angeles, California with additional patients planned in the coming weeks. Infusions of CAP-1002 were administered safely and patients are currently clinically stable. “Physicians leading the fight against COVID-19 patients approached Capricor to discuss the use of CAP-1002 due to its strong immunomodulatory capabilities. They believe that the use of CAP-1002 for the treatment or attenuation of ARDS (acute respiratory distress syndrome) pneumonia in COVID-19 patients is based on solid scientific rationale and pre-clinical data. We know from previously published pre-clinical data that CAP-1002 mitigates the release of anti-inflammatory cytokines, as well as macrophage activation in a number of models of inflammation, including sepsis and autoimmune diseases. It is believed that COVID-19-induced ARDS pneumonia is a response to an exaggerated and sustained cytokine storm. As such, we are hopeful that CAP-1002 will be of value to patients with respect to the treatment of COVID-19,” said Linda Marbán, PhD, Capricor’s President and Chief Executive Officer. The compassionate use act allows the FDA to immediately collect information on experimental treatments and then make the appropriate decisions about the safety and efficacy of those treatments.

Orgenesis & Excella-Bio Announce Breakthrough Manufacturing Process for Bioxomes™ (Proprietary Synthetic Exosomes/Extracellular Vesicles); Patented Process Results in Uniform, Scalable Production & Ability to Deliver Specific Cargo to Targeted Cells

On March 31, 2020, Orgenesis Inc. (NASDAQ: ORGS) (“Orgenesis” or the “Company”), a pioneering, global biotech company committed to lowering costs, accelerating commercialization, and transforming the delivery of cell and gene therapies (CGTs), announced that it has developed a breakthrough and patented manufacturing process for Bioxomes™ (synthetic exosomes/extracellular vesicles), through its collaboration and licensing agreement with Excella-Bio Ltd. Exosomes, or extracellular vesicles (EVs), are small vesicles that can transfer DNA, RNA, and proteins from the cell of origin to other, often targeted cells, thereby enabling alteration of the function of the targeted cells. It is belieed that exosomes can provide the same therapeutic benefit of whole cells, without the risks and difficulties of administering entire cells to patients. Together, Orgenesis and Excella-Bio have developed Bioxomes, which are synthetic exosomes/EVs. Until now, exosome/EV production has been based on conventional ultracentrifugation or ultrafiltration. These are both complex and costly techniques. Bioxomes are engineered and produced through a patented method as membrane nanoparticles isolated from cell cultures of various sources. Orgenesis and ExcellaBio have now demonstrated the optimization and scale-up of Bioxomes™, while generating consistent and repeatable results, including uniform particles sizes. These Bioxomes have demonstrated the ability to fuse with cell membranes and deliver an intracellular cargo, in a similar manner to that of natural exosomes. Bioxomes can be sourced effectively from various cell cultures. These include mesenchymal stem cells, immortalized cells, immune cells, and epithelial cells.

Mysterious Tuft Cells Found to Play Role in Pancreatitis

Persistent inflammation of the pancreas (chronic pancreatitis) is a known risk factor for developing pancreatic cancer, the third-deadliest cancer in the United States. Tuft cells (cells sensitive to chemical (chemosensory) changes typically found in the intestines and respiratory tract) had previously been discovered in the pancreas, but their function has largely remained a mystery. Now, a team of Salk Institute scientists, led by Professor Geoffrey Wahl, PhD, and Staff Scientist Kathleen DelGiorno, PhD, has uncovered the formation of tuft cells during pancreatitis and the surprising role of tuft cells in immunity, using mouse models of pancreatitis. The findings, published online in Frontiers in Physiology on February 14, 2020, could lead to the development of new biomarkers to test for pancreatitis and pancreatic cancer. The open-access article is titled “Tuft Cell Formation Reflects Epithelial Plasticity in Pancreatic Injury: Implications for Modeling Human Pancreatitis.” "By understanding these early stages of pancreas disease, we hope our work will lead to the development of new strategies to diagnose and treat pancreatitis and pancreatic cancer early on," says Dr. Wahl, co-corresponding author and holder of the Daniel and Martina Lewis Chair in Salk's Gene Expression Laboratory. The pancreas is an abdominal organ that plays a role in digestion and blood sugar regulation. Yet, scientists know little about how the pancreas recovers from injuries, such as pancreatitis, or fights off pancreatic cancer. Most of the pancreas is composed of acinar cells, which produce and secrete digestive enzymes. Acinar cells also have the ability to transform into another type of cell called a tuft cell.

Male Ring-Tailed Lemurs Exude Fruity-Smelling Odorants from Their Wrists to Attract Mates

In an article published online in Current Biology on April 16, 2020, scientists reported that male ring-tailed lemurs (Lemur catta) become more attractive to females by secreting a fruity and floral aroma from their wrists. Using detailed chemical analysis, the researchers identified three compounds responsible for this sweet scent, marking the first time that pheromones have been identified in a primate. The open-access article is titled “Key Male Glandular Odorants Attracting Female Ring-Tailed Lemurs.” "During the yearly breeding season, male lemurs rub the glands on their wrists against their fluffy tails and then wave them at females in a behavior called 'stink flirting,'" says senior author Kazushige Touhara, PhD, Professor and biochemist at the University of Tokyo. Ring-tailed lemurs have well-developed scent glands on their shoulders and wrists. These glands are typically used to designate social rank, territory, and reproductive status. However, behavioral observations show they also use their scent glands to catch the attention of females. "Because only ring-tailed lemurs have these wrist glands and exhibit 'stink flirting' behavior, we reasoned that specific odorants for sexual communication must be involved," Dr. Touhara says. At the Japanese Monkey Center (JMC) in Aichi and The Research Institute of Evolutionary Biology in Tokyo, Japan, Dr. Touhara and his team tracked the behavior of a conspiracy of ring-tailed lemurs. They observed that female lemurs sniffed the scent markings left by males more often and for longer periods of time during the breeding season--when females are sexually receptive.