News Release

Wednesday, April 15, 2020

NIH-funded study offers new path to modeling eye disease, advancing therapies

Researchers have discovered a technique for directly reprogramming skin cells into light-sensing rod photoreceptors used for vision. The lab-made rods enabled blind mice to detect light after the cells were transplanted into the animals eyes. The work, funded by the National Eye Institute (NEI), published April 15 in Nature. The NEI is part of the National Institutes of Health.

Up until now, researchers have replaced dying photoreceptors in animal models by creating stem cells from skin or blood cells, programming those stem cells to become photoreceptors, which are then transplanted into the back of the eye. In the new study, scientists show that it is possible to skip the stem-cell intermediary step and directly reprogram skins cells into photoreceptors for transplantation into the retina.

This is the first study to show that direct, chemical reprogramming can produce retinal-like cells, which gives us a new and faster strategy for developing therapies for age-related macular degeneration and other retinal disorders caused by the loss of photoreceptors, said Anand Swaroop, Ph.D., senior investigator in the NEI Neurobiology, Neurodegeneration, and Repair Laboratory, which characterized the reprogrammed rod photoreceptor cells by gene expression analysis.

Of immediate benefit will be the ability to quickly develop disease models so we can study mechanisms of disease. The new strategy will also help us design better cell replacement approaches, he said.

Scientists have studied induced pluripotent stem (iPS) cells with intense interest over the past decade. IPSCs are developed in a lab from adult cells rather than fetal tissue and can be used to make nearly any type of replacement cell or tissue. But iPS cell reprogramming protocols can take six months before cells or tissues are ready for transplantation. By contrast, the direct reprogramming described in the current study coaxed skin cells into functional photoreceptors ready for transplantation in only 10 days. The researchers demonstrated their technique in mouse eyes, using both mouse- and human-derived skin cells.

Our technique goes directly from skin cell to photoreceptor without the need for stem cells in between, said the studys lead investigator, Sai Chavala, M.D., CEO and president of CIRC Therapeutics and the Center for Retina Innovation. Chavala is also director of retina services at KE Eye Centers of Texas and a professor of surgery at Texas Christian University and University of North Texas Health Science Center (UNTHSC) School of Medicine, Fort Worth.

Direct reprogramming involves bathing the skin cells in a cocktail of five small molecule compounds that together chemically mediate the molecular pathways relevant for rod photoreceptor cell fate. The result are rod photoreceptors that mimic native rods in appearance and function.

The researchers performed gene expression profiling, which showed that the genes expressed by the new cells were similar to those expressed by real rod photoreceptors. At the same time, genes relevant to skin cell function had been downregulated.

The researchers transplanted the cells into mice with retinal degeneration and then tested their pupillary reflexes, which is a measure of photoreceptor function after transplantation. Under low-light conditions, constriction of the pupil is dependent on rod photoreceptor function. Within a month of transplantation, six of 14 (43%) animals showed robust pupil constriction under low light compared to none of the untreated controls.

Moreover, treated mice with pupil constriction were significantly more likely to seek out and spend time in dark spaces compared with treated mice with no pupil response and untreated controls. Preference for dark spaces is a behavior that requires vision and reflects the mouses natural tendency to seek out safe, dark locations as opposed to light ones.

Even mice with severely advanced retinal degeneration, with little chance of having living photoreceptors remaining, responded to transplantation. Such findings suggest that the observed improvements were due to the lab-made photoreceptors rather than to an ancillary effect that supported the health of the hosts existing photoreceptors, said the studys first author Biraj Mahato, Ph.D., research scientist, UNTHSC.

Three months after transplantation, immunofluorescence studies confirmed the survival of the lab-made photoreceptors, as well as their synaptic connections to neurons in the inner retina.

Further research is needed to optimize the protocol to increase the number of functional transplanted photoreceptors.

Importantly, the researchers worked out how this direct reprogramming is mediated at the cellular level. These insights will help researchers apply the technique not only to the retina, but to many other cell types, Swaroop said.

If efficiency of this direct conversion can be improved, this may significantly reduce the time it takes to develop a potential cell therapy product or disease model, said Kapil Bharti, Ph.D., senior investigator and head of the Ocular and Stem Cell Translational Research Section at NEI.

Chavala and his colleagues are planning a clinical trial to test the therapy in humans for degenerative retinal diseases, such as retinitis pigmentosa.

The work was supported by grants EY021171, EY025667, EY025905, and EY025717 and NEI Intramural Research Program grants ZIAEY000450, ZIAEY000474 and ZIAEY000546.

The University of North Texas has a patent pending on the chemical reprogramming method reported in this paper. CIRC Therapeutics is a start-up company that plans to commercialize treatments using the technology.

This press release describes a basic research finding. Basic research increases our understanding of human behavior and biology, which is foundational to advancing new and better ways to prevent, diagnose, and treat disease. Science is an unpredictable and incremental process each research advance builds on past discoveries, often in unexpected ways. Most clinical advances would not be possible without the knowledge of fundamental basic research.

NEI leads the federal governments research on the visual system and eye diseases. NEI supports basic and clinical science programs to develop sight-saving treatments and address special needs of people with vision loss. For more information, visit https://www.nei.nih.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

Mahato B, Kaya KD , Fan Y, Sumien N, Shetty RA, Zhang W, Davis D, Mock T , Batabyal S, Ni A, Mohanty S, Han Z, Farjo R, Forster M, Swaroop A and Chavala SH. Pharmacologic fibroblast reprogramming into photoreceptors restores vision. Published online April 15, 2020 in Nature.http://dx.doi.org/10.1038/s41586-020-2201-4

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Researchers restore sight in mice by turning skin cells into light-sensing eye cells - National Institutes of Health

NEW YORK, April 15, 2020 /PRNewswire/ -- The stem cell therapy market was valued at US$ 1,534.55 million in 2019 and is estimated to reach US$ 5,129.66 million by 2027; it is expected to grow at a CAGR of 16.7% from 2020 to 2027.

Read the full report: https://www.reportlinker.com/p05882135/?utm_source=PRN

The increasing awareness related to the stem cells therapy in effective disease management and growing demand for regenerative medicines are the key factor driving the stem cell therapy market. However, high cost related of the stem cell therapy limits the growth of the market.Stem cell research has been widely investigated globally for various medical applications, especially for the treatment of humans.This raises the importance of creating public awareness about stem cell research and its clinical potential.

The main role of stem cells is in the replacement of dying cells and reconstruction of damaged tissues. Based on the extensive stem cell research, many scientists have claimed that these cells could probably be used in the treatment of various diseases, including cancer and cardiovascular disease.There is a large number of potential treatment procedures that are undergoing clinical trials, and a notably few stem cell therapies have won FDA (i.e., US Food and Drug Administration) approval for clinical usage. For instance, in 2019, the FDA approved Fedratinib for the first-line treatment for myelofibrosis. Moreover, stem cell therapies are widely used in bone marrow transplantation, and these therapies have benefited thousands of people suffering from leukemia. Hematopoietic stem cells are used for treating more than 80 medical diseases, including immune system disorders, blood disorders, neurological disorders, metabolic disorders, genetic disorders, and several types of cancers, such as leukemia and lymphoma; this is also likely to boost the demand for this treatment procedure during the forecast period. Researchers are further investigating the use of stem cell therapies in the treatment of autoimmune disorders.

The global stem cell therapy market has been segmented on the basis of type, treatment, application type, and end user.Based on type, the market has been segmented into adult stem cell therapy, induced pluripotent stem cell therapy, embryonic stem cell therapy, and others.

The adult stem cell therapy held the largest share of the market in 2019; however, induced pluripotent stem cell therapy is estimated to register the highest CAGR in the market during the forecast period.Based on treatment, the stem cell therapy market has been segmented into allogeneic and autologous.

The allogeneic segment held a larger share of the market in 2019; however, the market for the autologous segment is expected to grow at a higher CAGR during the forecast period.Based on application type, the stem cell therapy market has been segmented into musculoskeletal, dermatology, cardiology, drug discovery and development, and other applications.

The musculoskeletal segment held the largest share of the stem cell therapy market in 2019, whereas the drug discovery and development segment is expected to report the highest CAGR during 20202027. Based on end user, the market has been segmented into academic and research institutes, and hospitals and specialty clinics. The academic & research institutes held the largest share of the market in 2019, and it is also expected to report the highest CAGR during the forecast period.Several essential secondary sources referred to for preparing this report are the FDA, World Health Organization (WHO), Organisation for Economic Co-operation and Development, National Institutes of Health, Spanish Agency for Medicines (AEMPS), Japanese Society for Regenerative Medicine, and Indian Council of Medical Research, among others.

Read the full report: https://www.reportlinker.com/p05882135/?utm_source=PRN

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Stem Cell Therapy Market to 2027 - Global Analysis and Forecasts by Type; Treatment; Application; End User, and Geography - P&T Community

Sleep, diet, exercise, and stress: these are factors known to change a persons risk of developing numerous non-communicable diseases. Such lifestyle impacts on healthbeneficial or harmfulexert much of their influence via inflammation. About 10 years ago, Matthias Nahrendorf began wondering just how inflammation and lifestyle might be linked biologically, and started thinking about how to pinpoint the mechanism in the cardinal case of cardiovascular disease.

A persons level of inflammation can easily be measured with a simple white blood cell test. White blood cells fight off bacterial invasions and repair damaged tissues, but they can also damage healthy tissue when they become too abundant. You can find them in atherosclerotic plaques, and you can find them in acute infarcts, says Nahrendorf, a professor of radiology who conducts high-resolution imaging research at Massachusetts General Hospital. You can find them in failing hearts and the brain, where they increase the risk of stroke.

By linking exercise to reduced white blood cell production, Nahrendorf shows how a lifestyle factor can modulate cardiovascular risk.

When Nahrendorf learned that the most potent, toxic, and pro-inflammatory white blood cells live only a few hours, or at most a day, he immediately realized that the paramount questionsgiven that they die off quickly yet remain abundant in the bloodare, where and why are they produced? What is their source? Perhaps, he hypothesized, lifestyle factors regulate hematopoiesis (blood production).

To test this idea, he decided to study the effects of exercise on the production of these leukocytes in healthy mice. First, though, he consulted the scientific literature on exercise in mice. Previous researchers, he learned, had found that exercise increases production of inflammatory immune cellswhich I thought was counterintuitive, Nahrendorf recalls. When he looked more carefully, he discovered that the type of exercise used in the studies was forced and thus possibly stressful because it was induced by electric shocks. He therefore decided to test only voluntary exercise. He and his colleagues put a wheel in each mouses cage, so the animals could choose to run if they were interested.

The mice never ran during the day. That is when they rest, Nahrendorf explains. But in the dark, they ran a lot, averaging six to seven miles every night. After three weeks, the exercising mice had measurably lower levels of circulating white blood cells. Exercise, he found, had pushed their blood stem cells (cells that can produce all the different types of blood cells) into a state of quiescence: a kind of dormancy in which they generate fewer pro-inflammatory white blood cells and platelets, without decreasing the number of oxygen-carrying red blood cells. Soon the exercising mice had fewer circulating white blood cells than their sedentary counterparts, dampening inflammationan effect that persisted for weeks.

The local signals within bone marrow that induce quiescence in blood stem cells were already well known, but the fact that exercise could trigger them was not. Nahrendorf next wanted to learn the identity of the trigger linking exercise to blood stem cell quiescence. Further investigation revealed that the only receptors with enhanced activity in the bone marrow niche where most blood stem cells exist were binding to a well-known hormone called leptin; it is produced by fat cells and regulates hunger.

Leptin is like the fuel gauge in a car. When the tank is fullmeaning energy (and food) are abundantleptin levels run high. As exercise uses up the gas in the tank, this lowers leptin levels, which signal that reserves are running low, thereby inducing hunger and the urge to eat in order to replenish depleted energy stores. Nahrendorf and his co-authors speculate in their 2019 Nature Medicine paper that leptins role in regulating energetically costly hematopoiesis may have evolved to produce blood cells only when whole body energy was abundantnot when people are exerting themselves. Contemporary sedentary behavior, they continue, which increases leptin and consequently hematopoiesis, may have rendered this adaptation a risk factor for cardiovascular disease (CVD) and perhaps also for other diseases with inflammatory components.

But with fewer circulating immune cells, would exercising mice be more vulnerable to infection? Nahrendorf challenged them with a protocol designed to induce infection in the blood, and found just the opposite: exercising mice had a more robust immune response, as semi-dormant blood stem cells swiftly sprang into activity and produced infection-fighting leukocytes, improving survival of the active mice as compared to those with no running wheels in their cages. Next, they investigated whether exercise would help mice with established atherosclerosis, and found that exercise was not only protective, it also reduced the size of existing plaques in the aorta.

Whether these associations would hold up in humans remained an open question. For answers, Nahrendorf turned to a study known as CANTOS, which had measured levels of inflammation in 4,892 patients who suffered heart attacks (see Raw and Red Hot, May-June 2019, page 46). When he approached the studys co-authors, Mallinckrodt professor of medicine Peter Libby and Braunwald professor of medicine Paul Ridker, he learned, serendipitously, not only that they possessed self-reported exercise levels for the participants, but also that they had tested leptin levels as well. They analyzed their raw data and found the same relationship among exercise, leptin, and leukocytes as in the mice. Data from a second human study cemented the result.

By identifying a previously unknown molecular mechanism linking voluntary exercise to reduced white blood cell production, Nahrendorf and his colleagues have highlighted how a lifestyle factor can modulate cardiovascular risk. Their discovery, the researchers hope, will point the way to wider adoption of healthy exercise regimens, and health-enhancing anti-inflammatory drugs.

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Stem cell activity linked to lifestyle - Harvard Magazine

Northwestern University scientists received top honors from the Clinical Research Forum as part of its 2020 Top Ten Clinical Research Achievement Awards program, taking home the associations highest honor and capturing more finalist nominations than any other institution.

The remarkable success of these brilliant and dedicated investigators shows the strength and breadth of Northwesterns clinical research program and demonstrates our shared commitment as an institution to groundbreaking science that transforms human health, said Eric G. Neilson, MD, vice president for medical affairs and Lewis Landsberg Dean, Northwestern University Feinberg School of Medicine.

John Rogers, PhD, the Louis Simpson and Kimberly Querrey Professor of Materials Science and Engineering, Biomedical Engineering and Neurological Surgery, was awarded the prestigious Herbert Pardes Clinical Research Excellence Award for the research study that best shows a high degree of innovation and creativity, advances science and has an impact upon human disease.

Richard Burt, MD, chief of Immunotherapy and Autoimmune Diseases in the Department of Medicine, was chosen to receive the Distinguished Clinical Research Award. Norrina Allen, PhD, associate professor of Preventive Medicine in the Division of Epidemiology, and Daniela Matei, MD, the Diana, Princess of Wales Professor of Cancer Research and a professor of Medicine in the Division of Hematology and Oncology, were also named to the list of top 20 finalists.

Rogers and Burt are members of the Northwestern University Clinical and Translational Sciences (NUCATS) Institute. The Clinical Research Forum, an organization dedicated to supporting the clinical translational research enterprise and promoting understanding and support for clinical research and its impact on health and healthcare, celebrates outstanding research accomplishments that exemplify innovation and impact on human disease.

Northwestern studies honored by the Clinical Research Forum are:

Skin-like Devices for Wireless Monitoring of Vital Signs in Neonatal Intensive Care (John Rogers, PhD), published in Science. Reporting on the development and validation of a pair of soft, flexible wireless sensors that replace the tangle of wire-based sensors that currently monitor babies in hospitals neonatal intensive care units. The study concluded that that the wireless sensors provided data as precise and accurate as that from traditional monitoring systems, and were gentler on a newborns fragile skin and allow for more skin-to-skin contact with the parent, which has been shown to improve the health of infants and promote emotional bonding.

Hematopoietic Stem Cell Transplantation for Frequently Relapsing Multiple Sclerosis (Richard Burt, MD), published in JAMA. Reporting the success of a process called hematopoietic stem cell transplantation, which temporarily shuts down and reboots patients immune systems with the application of a patients own stem cells, this study demonstrated significant improvement over the current therapies. The study found benefits for patients which no drug had been able to accomplish before.

Associations of Dietary Cholesterol or Egg Consumption with Incident Cardiovascular Disease and Mortality (Norrina Allen, PhD), published in JAMA.

The results of this large study found that adults who ate more eggs and dietary cholesterol had a significantly higher risk of cardiovascular disease and death from any cause.

The study suggested that current U.S. dietary guideline recommendations for dietary cholesterol and eggs, one of the richest sources of dietary cholesterol among all commonly consumed foods, may need to be re-evaluated.

Adjuvant Chemotherapy plus Radiation for Locally Advanced Endometrial Cancer (Daniela Matei, MD), published in New England Journal of Medicine.

This study found that radiation combined with chemotherapy did not increase recurrence-free survival in women with stage III/IVA endometrial cancer, normally the standard of care in these cases.

Endometrial cancer, which begins in the uterus, is the most common gynecologic cancer with most cases occurring in women after age 55, and both occurrence of and mortality from the disease are rising.

Nominees and Top Ten Awardees were announced at the end of January, and the Herbert Pardes Clinical Research Excellence Award and the Distinguished Clinical Research Achievement Awards were announced virtually on April 15.

Learn more about Northwestern University Feinberg School of Medicine at https://www.feinberg.northwestern.edu/.

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Northwestern Scientists Awarded Top Honors for Achievement in Clinical Research - Northwestern University NewsCenter

Widespread testing, even of those not showing symptoms, is one of the most effective ways to track the prevalence of coronavirus infections in communities.

The broad-based assays currently being ramped up by many hospitals and health systems around the country require a steady stream of single-use supplies. In addition to the familiar nasal/laryngeal swabs needed to collect samples, the much-sought-after test kits include vials of a liquid called viral transport medium that fixes and preserves those samples for later analysis in a lab.

UC Irvine Health has recently experienced increased demand for VTM, as medical staff recently have been conducting about 300 COVID-19 tests per day. Each test kit vial contains 2 milliliters of the fluid, so the systems current daily requirement is approximately 600 milliliters.

Logistics officials at UC Irvine Medical Center in Orange realized that they had only two weeks before exhausting their inventory, so they asked UC Irvines Emergency Operations Center which has been integrally involved in many aspects of the campuss response to the coronavirus crisis about a solution.

Randall Styner, emergency management director and EOC coordinator, shared the request recently on one of his groups regularly scheduled video conferences, and participants sprang into action. A follow-up call was scheduled between the UC Irvine Office of Research and medical center staff to ascertain whether VTM could be synthesized in campus labs and, if so, which labs could be utilized; if the necessary chemicals and reagents were on hand; and how much of the liquid was required.

Right then and there, we determined that it would be possible to do this, Styner says. VTM is basically a preservative made of materials that are common in lab settings, and its used all over campus.

Our people are wildly enthusiastic about doing something to help those in need.Aileen Anderson

He reached out to Bruce Morgan, associate vice chancellor for research administration, to see if his organization would approve of making VTM in UC Irvine labs and for help in soliciting faculty expertise. The request went up to Pramod Khargonekar, vice chancellor for research, who quickly gave the green light.

All indicators were pointing to this thing shooting down the rails like a rocket, Styner says. As soon as the word got out, people started getting a plan together.

Among the first to be contacted was the Department of Chemistry, since its faculty have unrivaled experience in mixing chemicals. Gregory Weiss, a professor in the department, sent a message asking colleagues in the School of Physical Sciences, the School of Biological Sciences and the School of Medicine to locate materials. They did so within hours.

The request from the vice chancellor for research was pretty extraordinary, and everybody dropped everything to respond, Weiss says.

The VTM synthesis project ultimately moved to the School of Medicine because its faculty have extensive expertise in required cell culture procedures.

We set up a mini-task force of related medical investigators and faculty from various schools, says Aileen Anderson, professor of physical medicine & rehabilitation and director of the Sue & Bill Gross Stem Cell Research Center. We sourced all the reagents needed for the media and set up a system to manage production in Gross Hall with social distancing measures in place to protect our staff.

Styner says that many of the components of VTM are readily available at UC Irvine and that teams here will create it under Centers for Disease Control and Prevention guidelines. The fluid is a mixture of saline, disinfectant, and such exotic ingredients as fetal bovine serum and sheep blood agar. Other necessary reagents were obtained from a supplier in Orange County.

Labs on campus should be able to generate more than enough VTM for COVID-19 testing through UC Irvine Health, Styner says, and UC Irvine will make the solution available to other healthcare providers in the region who need it. Anderson says UC Irvine will produce enough VTM for 16,000 test kits over the next four to six weeks, with the first of it reaching the medical center by April 10.

Were happy to lend our support in any way we can, she says. Our people are wildly enthusiastic about doing something to help those in need.

Khargonekar says the project is a great example of what the campus research community can do to help the medical center in this crisis, adding, Im very inspired by the tremendous energy and speed with which people are rising to the challenges.

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Campus labs produce sample-preserving fluid for COVID-19 test kits - University of California

MELBOURNE, Australia, April 17, 2020 (GLOBE NEWSWIRE) --Cynata Therapeutics Limited (ASX: CYP), a clinical-stage biotechnology company specialising in cell therapeutics, is pleased to announce that a scientific paper describing the use of Cymerus mesenchymal stem cells (MSCs) in a model of Acute Respiratory Distress Syndrome (ARDS) has been accepted for publication in the American Journal of Respiratory and Critical Care Medicine (AJRCCM).1The AJRCCM, commonly known as The Blue Journal, is widely regarded as the foremost peer-reviewed journal in the field of respiratory and critical care medicine.

Background

The study was conducted in 14 sheep with severe ARDS supported by extracorporeal membrane oxygenation (ECMO), which were given an endobronchial infusion of either Cymerus MSCs (n=7) or placebo (n=7). Animals were monitored and supported for 24 hours, at which time the study concluded.

ARDS is an inflammatory process leading to build-up of fluid in the lungs and respiratory failure. It can occur due to a range of insults, including infection, trauma and inhalation of noxious substances. It has received significant global attention in recent times, as it is one of the most serious complications experienced by patients suffering from COVID-19. ARDS accounts for approximately 10% of all ICU admissions and almost 25% of patients requiring mechanical ventilation, and results in hospital mortality ofup to 46%.2 In addition, survivors of ARDS are often left with severe long-term illness and disability.3

ECMO is a last-line intervention used in patients whose lungs are unable to provide an adequate amount of oxygen to the blood, despite the use of ventilators and other interventions. ECMO circulates blood through an artificial lung, oxygenating the blood before returning it to the patients circulation. ECMO can help support the vital organs in patients with severe ARDS, but it is not in itself a treatment for ARDS and the mortality among patients supported by it remains high.

This study was conducted independently of Cynata by a group of leading academics known as the Combining Extracorporeal Life Support and Cell Therapy in Critical Illness (CELTIC)Investigators, led by Professor John Fraser of the Critical Care Research Group, The Prince Charles Hospital, Brisbane. The study was funded by the Queensland Government, the National Health and Medical Research Council (NHMRC), the Intensive Care Society UK, and the Prince Charles Hospital Foundation.

Key Results

Cymerus MSC treatment was shown to exert a number of important beneficial effects in this study:

There were no statistically significant differences in oxygenation index between groups. The authors of the paper suggested that this may have been due to the severity of the lung injury induced; the fact that the observation period may have been too short to observe all beneficial effects of the treatment; and practical challenges performing these assessments during ECMO.

The authors also observed that a different dose regimen and/or route of administration could lead to further improved outcomes.

The study also found that MSCs adhere to the membranes in the ECMO device, resulting in a significant increase in pressure, and there was a higher incidence of thrombosis in the lungs observed post-mortem. While this did not lead to failure of the ECMO device or other observed adverse events, the study team considered that it could potentially do so, and therefore concluded that they cannot currently recommend the use of MSCs in combination with ECMO. It is important to note that this finding is relevant to MSCs in general (regardless of source), as it is related to the propensity of MSCs to adhere to plastic, but it does not have implications for the treatment of patients with ARDS who are NOT receiving ECMO.

Dr Kilian Kelly, Cynatas Chief Operating Officer, commented:

We are very encouraged by the beneficial effects of Cymerus MSCs on a number of important, clinically-relevant endpoints in this model of ARDS. These results provide valuable guidance on the potential clinical utility of Cymerus MSCs in the treatment of ARDS. It is also very useful to learn more about the practical mechanical challenges associated with administering MSCs at the same time as ECMO, but it is important to note that most patients with ARDS do not receive ECMO. Furthermore, in humans with ARDS who are not receiving ECMO, we expect to be able to administer repeated intravenous infusions of MSCs, which may have advantages compared to the approach that was taken in this preclinical study. We are currently in discussions with leading key opinion leaders about a possible clinical trial in human patients with ARDS, including those who have developed ARDS as a result of the devastating COVID19 pandemic.

Authorised for release by Dr Ross Macdonald, Managing Director & CEO

About Cynata Therapeutics (ASX: CYP)

Cynata Therapeutics Limited (ASX: CYP) is an Australian clinical-stage stem cell and regenerative medicine company focused on the development of therapies based on Cymerus, a proprietary therapeutic stem cell platform technology. Cymerus overcomes the challenges of other production methods by using induced pluripotent stem cells (iPSCs) and a precursor cell known as mesenchymoangioblast (MCA) to achieve economic manufacture of cell therapy products, including mesenchymal stem cells (MSCs), at commercial scale without the limitation of multiple donors.

Cynatas lead product candidate CYP-001 met all clinical endpoints and demonstrated positive safety and efficacy data for the treatment of steroid-resistant acute graft-versus-host disease (GvHD) in a Phase 1 trial. Cynata plans to advance its Cymerus MSCs into Phase 2 trials for GvHD, critical limb ischemia and osteoarthritis. In addition, Cynata has demonstrated utility of its Cymerus MSC technology in preclinical models of asthma, diabetic wounds, sepsis, heart attack and cytokine release syndrome, a life-threatening condition stemming from cancer immunotherapy.

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1 Millar JE, Bartnikowski N, Passmore MR, et al. Combined Mesenchymal Stromal Cell Therapy and ECMO in ARDS: A Controlled Experimental Study in Sheep. Am J Crit Care Med, 2020.2 Bellani G, Laffey JG, Pham T, et al. Epidemiology, Patterns of Care, and Mortality for Patients With Acute Respiratory Distress Syndrome in Intensive Care Units in 50 Countries. Jama. 2016;315(8):788.3 Herridge MS, Tansey CM, Matte A, et al. Functional disability 5 years after acute respiratory distress syndrome. N Engl J Med. 2011;364(14):1293-304.

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Preclinical Study Showing Beneficial Effects of Cymerus MSCs in Acute Respiratory Distress Syndrome Accepted for Publication in Leading Peer-Reviewed...

In the on-line debate, Francesco Curcio, a clinical pathology professor at the University of Udine, said that the coronavirus would not disappear, and that new viruses could also appear, which is why it was important to be ready and find a good solution immediately.

Curcio, who heads the Laboratory Medicine Department at the University Hospital of Udine, noted that currently used for treatment of Covid-19 were drugs for other viral diseases, including HIV and malaria, "which sometimes give good results, and sometimes not."

In his opinion, currently the best solution are mesenchymal stem cells, a sub-group of stem cells, because they have the ability to balance out the functioning of the immune system and regeneration of tissue.

Tamara Lah Turnek, the former director of the Slovenian National Institute of Biology, added that mesenchymal stem cell therapy could be considered in the second phase of Covid-19, when it was possible to contract an acute respiratory distress syndrome (ARDS).

ARDS is a consequence of a strong inflammatory response, called cytokine storm, and there is no cure for it for the time being, but the application of mesenchymal stem cells eases its consequences and improves the changes of recovery.

In this process, according to Lah Turnek, mesenchymal stem cells play two important roles at the same time - taking care of a balanced response of the immune system, and encouraging other cells to regenerate tissue.

In order to thoroughly research mesenchymal stem cell therapy and put it on the market, a consortium of 13 partners from eight countries has been formed, with the Slovenian biotech company Educell being the lead partner.

Educell director Miomir Kneevi said that while not much could be done for a major part of the population during this pandemic, "we can learn and prepare tools for the next outbreak, which will certainly happen."

He said that the goal was not to raise false hopes, but to show how mesenchymal stem cells could help, adding that it needed to be established what was the right quantity of cells and who will respond to therapy and who will not.

Sufficient production of stem cells also needs to be ensured, Kneevi said, noting that this was "a different type of medical intervention, which we must develop together with hospitals, producers and scientists."

B. N. Manohar, the director general of the late stage life science company Stempeutics from India's Bangalore, a partner in the consortium, said that mesenchymal stem cells had proven to be safe, and that they should thus be placed on the market.

If the results of clinical studies are good and positive, he believes that it will be possible to convince regulators to approve the therapy.

A study on seven patients in China has shown that it has some positive effects, and positive results were also shown on a patient in Udine, who was given mesenchymal stem cells prepared in Slovenia after all other therapies had failed.

The patient who had previously been mechanically ventilated for 20 days was not saved, but according to Curcio, results were promising, as the cells had done their job in terms of inflammation control.

Kneevi said that it depended on the European Medicines Agency when the first clinical studies would be available to researchers, but he announced that some results and solutions would certainly be available in the autumn.

Patrick Soon-Shiong, the director general of the partner company NantWorks, a network of health and technology startups, expressed the hope that mesenchymal stem cells would also help in other diseases caused by irregular functioning of the immune system.

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Stem cell therapy debated as option to fight coronavirus - STA - Slovenska Tiskovna Agencija

Herzeliya, Israel and Calgary, Alberta--(Newsfile Corp. - April 17, 2020) - InnoCan Pharma Corporation (CSE: INNO) ("InnoCan" or the "Company") announced that its wholly-owned subsidiary, InnoCan Pharma Ltd. of Herzliya Israel, has entered into a sponsored research agreement dated April 17, 2020 (the "Research Agreement") with Ramot at Tel Aviv University ("Ramot") to collaborate with Tel Aviv university to develop a novel, revolutionary approach to treat COVID-19 by using Cannabidiol (CBD) loaded Exosomes ("ICLX").

Under the terms of the Research Agreement, InnoCan and a team led by Prof. Daniel Offen, a leading researcher specializing in Neuroscience and Exosome technology at Tel Aviv University, will collaborate to develop the cell therapy product, based on Prof. Offen's work in the field. Innocan has agreed to fund the research based on agreed milestones, in the aggregate amount of approximately US $450,000 for the first stage.

InnoCan and Ramot are collaborating on a new, revolutionary exosome-based technology that targets both central nervous system (CNS) indications and the Covid-19 Corona Virus. CBD-Loaded Exosomes hold the potential to provide a highly synergistic effect of anti-inflammatory properties and help in the recovery of infected lung cells. This product, which is expected to be administrated by inhalation, will be tested against a variety of lung infections.

Exosomes are small particles created when stem cells are multiplied. Exosomes can act as "homing missiles", targeting specific damaged organs and have an important role in cell-to-cell communication. When the cell healing properties of the exosomes are combined with the anti-inflammatory properties of CBD, it is expected to reach high synergetic effect. The research results may be beneficial to additional treatments for Central Nerve System ( CNS ) indications such as epilepsy and Alzheimer's Disease.

The Research Agreement also gives InnoCan Pharma the exclusive option to receive an exclusive worldwide royalty-bearing license to Ramot's background technology and the research results to allow InnoCan to develop and manufacture ICLX for the treatment of COVID-19 and potentially for other types of lung inflammations and additional respiratory related illnesses. Once InnoCan Pharma provides notice of intent to exercise the option, InnoCan Pharma and Ramot have agreed to negotiate the license agreement in good faith according to predefined commercial terms annexed to the Research Agreement.

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Background

The world is suffering from a rapid rise in illness due to the fast growing spread of the COVID-19 pandemic. The lungs are the organ most affected by COVID-19, causing pneumonia that rapidly progresses to acute respiratory distress syndrome and can further result in respiratory failure, septic shock, or multi-organ failure, and in the most severe cases death.

Recent in-vivo and in-vitro studies have demonstrated that exosomes derived from mesenchymal stem cells (MSC) can promote regeneration and improve immune reaction processes in damaged tissues. Exosomes contain anti-inflammatory agents that can target inflamed organs. Prof. Offen and his team have already successfully loaded exosomes with various molecules. They have also succeeded in treating different tissue injuries in animal models, while significantly reducing inflammation and pathological impairment. To date, there have been hundreds of publications and several clinical studies using exosomes globally, demonstrating their therapeutic potential at different applications.

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Animal studies have also demonstrated CBD as effective in reducing lung inflammation. Based on these findings and the capability of attaching molecules to Exosomes as was shown at Prof. Offen studies, InnoCan believes that its ICLX therapy has the potential to treat the COVID-19 virus by combining CBD with exosomes, thereby creating therapeutic synergies. The suggested combination may have strong synergetic effects, thereby increasing the potential efficacy of planned treatments.

Prof. Dani Offen, of the Sackler School of Medicine, Sagol School of Neuroscience at Tel Aviv University, said, "I am pleased to work with the InnoCan team on this exciting ICLX development project. We are facing a challenging time, and I believe our unique approach holds a promise to offer a treatment for COVID-19, pneumonia and perhaps for other lung inflammations as well."

Iris Bincovich, CEO of InnoCan's CEO, also commented, "COVID-19 has quickly become one of the largest challenges in healthcare today. With the development of ICLX, we are creating a new treatment, to join the global mission to combat the effects of COVID-19. We are determined to make a difference as quickly as possible to assist patients worldwide."

Keren Primor Cohen, CEO of Ramot at Tel Aviv University, summarized, "We hope that this collaboration with Innocan Pharma will assist in transforming Prof. Offen's promising technology into a real treatment for the global threat of COVID-19."

The Company is not making any express or implied claims that the research to be conducted under the Research Agreement will be successful or that any products developed from the research will have the ability to eliminate, cure, treat or contain the Covid-19 Coronavirus or other lung conditions.

Innocan Pharma has also been approved for listing on the Frankfurt Stock Exchange (FSE) under the trading symbol: FRA: IP4.

About InnoCan Pharma Corporation

The Company, through its wholly-owned subsidiary, Innocan Pharma Ltd. ("Innocan Pharma Israel"), is a pharmaceutical tech company that focuses on the development of several drug delivery platforms combining cannabidiol ("CBD"). The Company signed on a worldwide exclusive license agreement with Yissum, the commercial arm of the Hebrew University of Jerusalem to develop CBD drug delivery platform based on a unique-controlled release Liposome to be administrated by Injection. The company planes, together with Prof. Berenholtz, Head of the Laboratory of Membrane and Liposome Research of the Hebrew University to test the Liposome platform on several potential indications. -The company is also working on dermal product integrating CBD with other pharmaceutical ingredients as well as the development and sale of CBD-integrated pharmaceuticals. include, but are not limited to, topical treatments for relief of psoriasis symptoms as well as the treatment of muscle pain and rheumatic pain.The founders and officers of InnoCan have commercially successful track records in the pharmaceutical and technology sectors in Israel and globally.

About Ramot

Ramot is the technology transfer company of Tel Aviv University, one of Israel's foremost research and teaching universities. It is one of the major hubs that has contributed to Israel's global reputation as the "Startup Nation". Founded in 1956, Tel Aviv University is located in Israel's cultural, financial and industrial center. Rooted in both academic and corporate arenas, Ramot is uniquely positioned to cultivate the special relationships between these two compelling worlds, creating win-win connections that support fertile, groundbreaking research while providing companies with discoveries that give them a crucial competitive edge.

So far, these collaborations have produced more than 60 active start-up companies and the registration of over 70 patents a year. Around 300 additional patents are currently commercially available while awaiting the finalization of the patenting process. Dry eye therapy with Tubilux Pharma, Circadin, a treatment for sleeping disorders with Neurim Pharmaceuticals Blistex Inc., improved flash memory with SanDisk and others have proven that the industry can benefit greatly from the talent pool Tel Aviv University has to offer, and that rigorous research and out-of-the-box thinking can create a product that answers a consumer's everyday needs.

For further information, please contact:For InnoCan Pharma Corporation:Iris Bincovich, CEO+972-54-3012842info@innocanpharma.com

NEITHER THE CANADIAN SECURITIES EXCHANGE NOR ITS REGULATION SERVICES PROVIDER HAVE REVIEWED OR ACCEPT RESPONSIBILITY FOR THE ADEQUACY OR ACCURACY OF THIS RELEASE.

Caution regarding forward-looking information

Certain information set forth in this news release, including, without limitation, information regarding the markets, requisite regulatory approvals and the anticipated timing for market entry, is forward-looking information within the meaning of applicable securities laws. By its nature, forward-looking information is subject to numerous risks and uncertainties, some of which are beyond InnoCan's control. The forward-looking information contained in this news release is based on certain key expectations and assumptions made by InnoCan, including expectations and assumptions concerning the anticipated benefits of the product markets, satisfaction of regulatory requirements in various jurisdictions and satisfactory completion of requisite production and distribution arrangements.

Forward-looking information is subject to various risks and uncertainties which could cause actual results and experience to differ materially from the anticipated results or expectations expressed in this news release. The key risks and uncertainties include but are not limited to: general global and local (national) economic, market and business conditions; governmental and regulatory requirements and actions by governmental authorities; and relationships with suppliers, manufacturers, customers, business partners and competitors. There are also risks that are inherent in the nature of product distribution, including failure to obtain any required regulatory and other approvals (or to do so in a timely manner) and availability in each market of product inputs and finished products. The anticipated timeline for entry to markets may change for a number of reasons, including the inability to secure necessary regulatory requirements, or the need for additional time to conclude and/or satisfy the manufacturing and distribution arrangements. As a result of the foregoing, readers should not place undue reliance on the forward-looking information contained in this news release concerning the timing of launch of product distribution. A comprehensive discussion of other risks that impact InnoCan can also be found in InnoCan's public reports and filings which are available under InnoCan's profile at http://www.sedar.com.

Readers are cautioned that undue reliance should not be placed on forward-looking information as actual results may vary materially from the forward-looking information. InnoCan Pharma does not undertake to update, correct or revise any forward-looking information as a result of any new information, future events or otherwise, except as may be required by applicable law.

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InnoCan Pharma Collaborates with Tel Aviv University to Develop a New Revolutionary Approach to Treat the COVID-19 Corona Virus with Exosomes-Loaded...