Exosome Technologies Market Report- Capricor Therapeutics Inc , Evox Therapeutics Ltd , ReNeuron Group Plc , Stem Cell Medicine Ltd and more – Hitz…

Latest report, Exosome Technologies Market: Recent Surge in R&D with Capricor, Codiak and Evox Therapeutics among Key Players, explores the application of exosome technologies within the pharmaceutical and healthcare industries. Exosomes are small cell-derived vesicles that are abundant in bodily fluids, including blood, urine and cerebrospinal fluid as well as in in vitro cell culture.

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Top Company Analysis in this Report:Capricor Therapeutics Inc , Evox Therapeutics Ltd , ReNeuron Group Plc , Stem Cell Medicine Ltd , Tavec Inc. , Codiak Biosciences Inc., Therapeutic Solutions International Inc., ArunA Biomedical Inc., Ciloa and more

These vesicles are being used in a variety of therapeutic applications, including as therapeutic biomarkers, drug delivery systems and therapies in their own right. Research within this area remains in the nascent stages, although a number of clinical trials have been registered within the field. Exosomes have several diverse therapeutic applications, largely centering on stem cell and gene therapy. Exosomes have been identified as endogenous carriers of RNA within the body, allowing for the intercellular transportation of genetic material to target cells.

As such, developers have worked to engineer exosomes for the delivery of therapeutic miRNA and siRNA-based gene therapies. As RNA is highly unstable within the body, a number of different biologic vector systems have been developed to enhance their transport within the circulation, including viruses and liposomes. Similarly, exosomes derived from stem cells have also been identified for their therapeutic applications, particularly in the treatment of cancer and cardiovascular disease. Exosome technologies offer several advantages over existing biologic-based drug delivery systems.

They have a long circulatory half-life as a result of their high stability and ability to avoid breakdown by the mononuclear phagocyte system and reticuloendothelial systems. Moreover, exosomes have several functional properties that favor their use in therapeutic delivery. Exosomes can be engineered to incorporate targeting ligands, allowing them to deliver cargo selectively to cells. Their small size allows them to penetrate the blood-brain barrier for the delivery of central nervous system therapies, whereas in cancer they can accumulate within the tumor via enhanced permeability and retention effects.

Finally, clinical trials have shown relatively large-scale production to be possible and indicate that exosome therapies can be safely administered to humans. Additionally, exosomes are being investigated for their potential as prognostic and diagnostic biomarkers for several different disease indications. Exosomes make good candidates for biomarker research because of two unique characteristics: their presence in various accessible bodily fluids, and their resemblance to their parent cells of origin. R&D in exosome technologies has increased markedly in recent years. This report provides detailed information on the various healthcare applications of exosomes, and assesses the pipeline, clinical trial and company landscapes.

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Table of Contents in this Report:

1 Table of Contents 41.1 List of Tables 61.2 List of Figures 7

2 Exosomes in Healthcare 82.1 Overview of Exosomes 82.2 Drug Delivery Systems 92.2.1 Modified Release Drug Delivery Systems 92.2.2 Targeted Drug Delivery Systems 102.2.3 Liposomes 122.2.4 Viruses 142.2.5 Exosomes 172.3 The Exosome Lifecycle 182.4 Exosomes in Biology 182.5 Exosomes in Medicine 192.5.1 Biomarkers 192.5.2 Vaccines 202.6 Exosomes as a Therapeutic Target 202.7 Exosomes as Drug Delivery Vehicles 212.8 Therapeutic Preparation of Exosomes 212.8.1 Isolation and Purification 222.8.2 Drug Loading 222.8.3 Characterization 232.8.4 Bioengineering 232.8.5 Biodistribution and In Vivo Studies 232.8.6 Advantages of Exosome Therapies 242.8.7 Disadvantages of Exosome Therapies 242.9 Exosomes in Therapeutic Research 252.9.1 Exosome Gene Therapies 252.9.2 Exosome in Stem Cell Therapy 262.10 Exosomes in Oncology 272.10.1 Immunotherapy 272.10.2 Gene Therapy 282.10.3 Drug Delivery 292.10.4 Biomarkers 302.11 Exosomes in CNS Disease 302.11.1 Tackling the Blood-Brain Barrier 302.11.2 Exosomes in CNS Drug Delivery 312.11.3 Gene Therapy 322.12 Exosomes in Other Diseases 332.12.1 Cardiovascular Disease 332.12.2 Metabolic Disease 33

3 Assessment of Pipeline Product Innovation 363.1 Overview 363.2 Exosome Pipeline by Stage of Development and Molecule Type 363.3 Pipeline by Molecular Target 373.4 Pipeline by Therapy Area and Indication 383.5 Pipeline Product Profiles 383.5.1 AB-126 ArunA Biomedical Inc. 383.5.2 ALX-029 and ALX-102 Alxerion Biotech 393.5.3 Biologics for Autism Stem Cell Medicine Ltd 393.5.4 Biologic for Breast Cancer Exovita Biosciences Inc. 393.5.5 Biologics for Idiopathic Pulmonary Fibrosis and Non-alcoholic Steatohepatitis Regenasome Pty 393.5.6 Biologic for Lysosomal Storage Disorder Exerkine 393.5.7 Biologics for Prostate Cancer Cells for Cells 403.5.8 CAP-2003 Capricor Therapeutics Inc. 403.5.9 CAP-1002 Capricor Therapeutics Inc. 413.5.10 CIL-15001 and CIL-15002 Ciloa 423.5.11 ExoPr0 ReNeuron Group Plc 423.5.12 MVAX-001 MolecuVax Inc. 433.5.13 Oligonucleotides to Activate miR124 for Acute Ischemic Stroke Isfahan University of Medical Sciences 443.5.14 Oligonucleotides to Inhibit KRAS for Pancreatic Cancer Codiak BioSciences Inc. 443.5.15 Proteins for Neurology and Proteins for CNS Disorders and Oligonucleotides for Neurology Evox Therapeutics Ltd 443.5.16 TVC-201 and TVC-300 Tavec Inc. 45

4 Assessment of Clinical Trial Landscape 484.1 Interventional Clinical Trials 484.1.1 Clinical Trials by Therapy Type 484.1.2 Clinical Trials by Therapy Area 494.1.3 Clinical Trials by Stage of Development 504.1.4 Clinical Trials by Start Date and Status 504.2 Observational Clinical Trials 514.2.1 Clinical Trials by Therapy Type 514.2.2 Clinical Trials by Therapy Area 514.2.3 Clinical Trials by Stage of Development 524.2.4 Clinical Trials by Start Date and Status 534.2.5 List of All Clinical Trials 54

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Exosome Technologies Market Report- Capricor Therapeutics Inc , Evox Therapeutics Ltd , ReNeuron Group Plc , Stem Cell Medicine Ltd and more - Hitz...

Tulane team researching ways to end opioid addiction – News from Tulane

Michael J. Moore, center, a professor of biomedical engineering in the Tulane School of Science and Engineering, is leading a team of researchers on a project called the HEAL Initiative, or Helping to End Addiction Long-term Initiative. Researchers include Jeffrey Tasker, the Catherine and Hunter Pierson Chair in Neuroscience, left, and James Zadina, director of the Neuroscience Laboratory at the Veterans Administration Medical Center and an adjunct professor of medicine at the Tulane School of Medicine. (Photo by Matthew Hinton)

A Tulane University researcher is joining more than 40 universities from across the United States in looking for ways to improve treatment of chronic pain and ultimately achieve long-term recovery from opioid addiction.

Michael J. Moore, professor of biomedical engineering in the Tulane School of Science and Engineering, is part of a $945 million National Institutes of Health project called the HEAL Initiative, or Helping to End Addiction Long-term Initiative.

In 2016, an estimated 50 million U.S. adults suffered from chronic pain and in 2018, an estimated 10.3 million people 12 years and older misused opioids, including heroin.

This is indeed an exciting opportunity to work on a problem of great public health significance to our nation.

Tulane biomedical engineering professor Michael J. Moore

Its clear that a multi-pronged scientific approach is needed to reduce the risks of opioids, accelerate development of effective non-opioid therapies for pain and provide more flexible and effective options for treating addiction to opioids, NIH Director Francis S. Collins said in a statement. This unprecedented investment in the NIH HEAL Initiative demonstrates the commitment to reversing this devastating crisis.

Moores share of the project is $1.2 million. He will be teaming up with Jeffrey Tasker, the Catherine and Hunter Pierson Chair in Neuroscience, and James Zadina, director of the Neuroscience Laboratory at the Veterans Administration Medical Center and an adjunct professor of medicine at the Tulane School of Medicine.

This is indeed an exciting opportunity to work on a problem of great public health significance to our nation, Moore said.

The management of pain both acute and chronic can be a frustratingly futile endeavor for both patients and clinicians, Moore said. Desperate attempts at treatment with opioids and other narcotics has led to a heartbreaking and calamitous epidemic of addiction to prescription painkillers.

The epidemic has prompted federal agencies and the pharmaceutical industry to work toward identifying the next generation of painkillers. Unfortunately, Moore said, there are few adequate model systems currently in use to enable rapid screening of the analgesic properties of drug candidates.

Moores proposal seeks to develop the first model of pain that utilizes living human cells on a computer chip, mimicking the transmission of pain and enabling the evaluation of the cellular basis of tolerance to certain drugs. Moore said the model will eventually enable experimental drugs to be screened in a way that is faster, less expensive and more effective.

He and his team are collaborating with Randolph Ashton, an associate professor of biomedical engineering at the University of Wisconsin, and Swaminathan Rajaraman, an assistant professor of electrical and computer engineering at the University of Central Florida. Ashton is developing human stem-cell derived spinal neurons, and Rajaraman is developing specially-made microelectrodes for taking electrical measurements from the cells.

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Tulane team researching ways to end opioid addiction - News from Tulane

The most amazing medical breakthroughs of 2019 – New York Post

Richard Berry had tried nearly everything to fight his prostate cancer: surgery, radiation and chemotherapy. But after two years of treatment, his cancer had metastasized. The opportunity to participate in a clinical trial was his last hope.

I wasnt ready to die, Berry, 75, of Keene, NH, tells The Post. I didnt want to mess around.

In 2015, he connected with Dr. Christopher Sweeney, an oncologist at Bostons Dana-Farber Cancer Institute, who was leading a trial in which the drug enzalutamide an androgen-receptor inhibitor that stops the growth of cancer cells was being used in conjunction with testosterone suppression to treat metastasized prostate cancer.

Berry became one of 1,125 patients involved in the trial. Remarkably, hes now cancer-free.

Hes one of the most dramatic cases to date, Sweeney tells The Post of Berrys progress. Hes in a deep remission. This is what I had hoped to see. However, the magnitude of the effect and the early results were a surprise and delight.

So far, Sweeney says, hes seen a handful of deep remissions like Berrys and that the results were positive sooner than we anticipated. The study was published in July in the New England Journal of Medicine.

The drug, which is already FDA-approved for other uses, is currently under FDA review to make it more widely available for metastasized prostate cancer patients and not just for use in clinical trials.

Approval is anticipated any day now, according to Dana-Farber Cancer Institute reps.

Meanwhile, Berrys scans continue to be clear a year after he stopped the treatment.

I dont even think about [the cancer] anymore, Berry says. I feel good. I have energy again, and, overall, Im just happy.

Here, six more amazing medical breakthroughs that could be a reality and soon.

A blood test for breast cancer

Researchers at the University of Nottingham in England have created a blood test that could potentially detect breast cancer up to five years before a lump or other symptoms appear. The test, which would be much cheaper and easier to conduct than a mammogram, looks for autoantibodies (produced by the body in reaction to cancer cells) in the blood.

The researchers said in a statement that another larger study is planned, and, if all goes well, the test could be on the market within the next five years.

The discovery was presented at the UKs National Cancer Research Institutes annual conference in early November, which showcases the worlds biggest cancer advances. A similar test for lung cancer is currently being studied in Scotland.

A new cystic fibrosis find

After two clinical trials, a new drug, Trikafta, a triple combination therapy, was found to cause significant improvements in cystic fibrosis patients respiratory health and lung function, leading the FDA to fast-track its approval in October.

The trials results were published in the Lancet and the New England Journal of Medicine.

Trikafta is now available to patients 12 and older who have the most common cystic fibrosis mutation, which could affect almost 90% of people with the disease, or about 27,000 people in the US.

A promising Alzheimers drug

Early disappointing results halted studies, but after a comprehensive review of the data, researchers are excited once again about an Alzheimers drug called aducanumab.

After 18 months of taking the drug, participants showed lowered cognitive decline (about 15% to 27%) on memory and cognitive ability tests compared to a placebo. The results were published in late October in the journal Nature.

Due to these results, the drug manufacturer is currently seeking FDA approval for aducanumab to treat Alzheimers disease soon after diagnosis.

Crispr for everything

Crispr is a gene-editing tool that allows scientists to modify DNA and genes.

The technique is being used in numerous ways, from medical research to growing crops. Some of its proposed innovations destroying superbugs, eradicating malaria and curing HIV are quite far off.

Still, the progress is exciting. A patient has been successfully treated for sickle cell disease using the technology, and clinical trials using Crispr to treat cancers such as non-Hodgkins lymphoma are currently recruiting. Next month, at the American Society of Hematology meeting in Orlando, Fla., scientists will present the results of a three-person cancer study where Crispr was used to treat two people with multiple myeloma and one with sarcoma.

Another sickle cell breakthrough

The first patient in an ongoing clinical trial for sickle cell disease, Manny Johnson, 22, who used to require monthly blood transfusions, has been symptom-free for more than a year after undergoing treatment in Boston. Another sufferer, Jennelle Stephenson, 28, reported showing no signs of sickle cell anemia in March after undergoing a yearlong treatment in DC.

The treatment, which is based on 70 years of research, uses a novel gene therapy treatment an infusion of the patients own reworked bone marrow to help the body produce normal red blood cells consistently.

Clinical trials are ongoing around the country. The results of the first adolescent participant, a 16-year-old girl, are expected next month from Dana-Farber.

Peanut allergy promise

A new study out of Stanford University showed that after one injection of the antibody, etokimab, which is already being studied to treat other immune issues such as asthma and eczema, people with severe peanut allergies were able to eat peanut protein just two weeks later.

The study was published Nov. 14 online in the journal JCI Insight.

Currently, the only available treatment for the potentially deadly allergy is oral immunotherapy, where patients eat small doses in graduating scales under medical care. The process can take up to a year and can cause allergic reactions.

A larger study is planned to shore up dosing, timing and hopefully be used to treat other food allergies.

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The most amazing medical breakthroughs of 2019 - New York Post

Precision Medicine and Personalized Treatments Shifting the Tide in Mantle Cell Lymphoma – Curetoday.com

While resistance can often make it a challenge to treat mantle cell lymphoma, therapies have come a long way in recent years thanks to the emergence of personalized treatment and the use of BTK inhibitors, according to one expert.

Ruan, a hematologist and oncologist from Weil Cornell Medicine in New York City, recently sat down with OncLive, CUREs sister publication, to discuss the treatment trends in this disease space.

We're moving toward precision medicine and personalized treatment (in MCL), said Ruan. The objective is to overcome resistance and come up with treatment plans that address the underlying disease biology and take personal factors about the patients into consideration.

When asked about the novel agents that are currently showing promise in MCL, Ruan explained that BTK inhibitors have resulted in highoverall response rates (ORRs), more complete remissions (CRs) and a lower number of side effects in this patient population.

Since the approval of Revlimid (lenalidomide) in 2013, a number of newer BTK inhibitors have been approved, with first-generation Imbruvica (ibrutinib) standing out as the top choice for many. So far, this class of agents has the best single-agent activity in MCL to date, explained Ruan. For example, ibrutinib would give about a 65% to 67% ORR, and about 20% of that would be CRs. In contrast, lenalidomide would give you about a 30% ORR and up to about 8% CR.

A newer BTK inhibitor, Calquence (acalabrutinib), is considered just as effective as Imbruvica with an overall response rate of 81% and complete response in 40% of patients who took it during the phase 2 study that led to its approval. That's quite remarkable for a single agent for patients who have relapsed/refractory MCL, Ruan said.

The field continues to grow with the testing of new agents, including BCL-2 inhibitor, Venclexta (venetoclax), which is currently approved in chronic lymphocytic leukemia and other indolent B-cell non-Hodgkin lymphomas. In MCL, we have data to suggest that it has promising activity as a single agent, she said. Currently, it's being studied in combination with a variety of other agents, including BTK inhibitors. They seem to work very well and are helping to move treatment away from chemotherapy.

However, treating MCL can be challenging because of treatment resistance, explained Ruan. All of the agents are being introduced, tested, and approved in the setting of relapsed disease, said Ruan. That is a big challenge for a sizable portion of patients who do not respond maybe they have a genetic mutation that is not responsive to targeted therapy or they develop a secondary mutation in the process of being treated and then they become resistant.

The issue of resistance is important to consider when treating MCL, according to Ruan. We have to provide novel agents that are sensitive to the disease at every stage of the evolution and come up with additional therapies that can overcome the resistance, she said.

In the future, Ruan predicts the shift toward precision medicine and personalized treatment will continue. The objective is to overcome resistance and come up with treatment plans that address the underlying disease biology and take personal factors about the patients into consideration, she said. This is important for us to understand MCL individually.

This precise approach would mean doing more genetic analysis to determine what type of disease is being treated in order to create a treatment plan that would provide the most benefit for the patient.

If we could know (about mutations) ahead of time, we could have the treatment designed and tailored to maximize treatment effectiveness and minimize adverse events, said Ruan.

It doesn't make sense to subject patients to all of the cytotoxicity associated with conventional intensive chemotherapy while other options, such as participation in clinical trials with novel agents, could be very effective.

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Low-Concentration PTX And RSL3 Inhibits Tumor Cell Growth Synergistica | CMAR – Dove Medical Press

Jing Ye, Xiaohua Jiang, Zhihuai Dong, Sunhong Hu, Mang Xiao

Department of Otolaryngology Head and Neck Surgery, Sir Run Run Shaw Hospital, College of Medicine, Zhejiang University, Hangzhou, Zhejiang, Peoples Republic of China

Correspondence: Mang XiaoDepartment of Otolaryngology Head and Neck Surgery, Sir Run Run Shaw Hospital, College of Medicine, Zhejiang University, East Qingchun Road Nr.3, Hangzhou, Zhejiang 310016, Peoples Republic of ChinaEmail joelxm@zju.edu.cn

Introduction: RSL3-induced ferroptosis is a cell death pathway dependent upon intracellular iron and is characterized by accumulation of lipid hydroperoxides. Glutaminolysis, a glutamine-fueled intracellular metabolic pathway, is an essential pathway of ferroptosis in cancer cells. Recent findings showed low-concentration paclitaxel (PTX) could inhibit cell death by upregulating p53 expression; downregulating glutaminolysis-related genes.Methods: The therapeutic effect of RSL3 plus low-concentration PTX combination therapy was investigated in HPSCC cells harboring mutant p53 (mtp53). Relative cell viability, ferroptosis-specific lipid peroxidation and relevant protein expression were evaluated.Results: We demonstrated that neither PTX nor RSL3 in low concentration caused significant cell death; however, the combination therapy is shown to induce ferroptosis and significant cell death in mtp53 HPSCC. We discovered that low-concentration PTX enhanced the RSL3-induced ferroptosis by upregulating mtp53 expression. Furthermore, mtp53-mediated transcriptional regulation of SLC7A11 could be the key determinant.Discussion: Although gain-of-function of p53 variants remains to be characterized, our findings provide new insight into the synergistical cell death by regulating ferroptosis and p53.

Keywords: HPSCC, ferroptosis, low-concentration paclitaxel, RSL3, synthetic cell death, mtp53, SLC7A11, GOF p53 variants

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Low-Concentration PTX And RSL3 Inhibits Tumor Cell Growth Synergistica | CMAR - Dove Medical Press

THUMPD3-AS1 Is Correlated With Non-Small Cell Lung Cancer And Regulate | OTT – Dove Medical Press

Jia Hu, Youfang Chen, Xiaodong Li, Huikai Miao, Rongzhen Li, Dongni Chen, Zhesheng Wen

Department of Thoracic Oncology, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-Sen University Cancer Center, Guangzhou 510060, Peoples Republic of China

Correspondence: Zhesheng Wen Email wenzhsh@sysucc.org.cn

Background: Of all malignancies, lung cancer is the leading cause of death, and non-small cell lung cancer (NSCLC) accounts for 8085% of all lung cancers. In this study, the long non-coding RNA (lncRNA) THUMPD3-AS1 was observed to be highly expressed in NSCLC and correlated with TNM stages and relapse, suggesting that THUMPD3-AS1 is involved in the regulation of NSCLC.Methods: The aim of this study was to investigate the regulatory function and mechanism of THUMPD3-AS1 in NSCLC cells by cellular function and molecular biology experiments.Results: Overexpression and knockdown analysis revealed that THUMPD3-AS1 promoted tumor progression by increasing cell proliferation and self-renewal of NSCLC cells. Moreover, THUMPD3-AS1 may act as an endogenous sponge of microRNA-543 (miR-543) which can regulate the target gene ONECUT2 in NSCLC cells.Conclusion: Our study indicated that THUMPD3-AS1 regulated NSCLC cell self-renewal by regulating the expression of miR-543 and ONECUT2, and THUMPD3-AS1 can potentially act as a biomarker or therapeutic target in NSCLC.

Keywords: non-small cell lung cancer, self-renewal, THUMPD3-AS1, miR-543, ONECUT2

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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THUMPD3-AS1 Is Correlated With Non-Small Cell Lung Cancer And Regulate | OTT - Dove Medical Press