– Dr. Sorensen to become CEO of a start-up hemostasis and thrombosis company – – Dr. Sorensen to become CEO of a start-up hemostasis and thrombosis company –
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Codiak BioSciences Announces the Transition of Benny Sorensen, M.D., Ph.D. to Scientific Advisory Board Member and Clinical Consultant Roles
AUSTIN, Texas, July 06, 2021 (GLOBE NEWSWIRE) -- XBiotech Inc.’s (NASDAQ: XBIT) (“XBiotech”) Board of Directors has declared an extraordinary cash dividend of approximately $2.50 per share, or up to an aggregate of $75 million, to holders of its common stock. This one-time, special dividend will be payable on July 23, 2021 to stockholders of record at the close of business on July 16, 2021.
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XBiotech Announces Dividend to Holders of Common Stock
Geneva, Switzerland, July 7, 2021 - Addex Therapeutics (SIX:ADXN), a clinical-stage pharmaceutical company pioneering allosteric modulation-based drug discovery and development, announced today that Chief Executive Officer, Tim Dyer, will present at Access to Giving Virtual Conference at 9 AM ET on July 14, 2021. Mr. Dyer will give a corporate update, including an overview of recent advances in Addex’s clinical trial program. The conference is free to all registrants.
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Addex Therapeutics to Present at Access to Giving Virtual Conference on July 14, 2021
Our cells have abilities that go far beyond the fastest, smartest computer. They generate mechanical forces to propel themselves around the body and sense their local surroundings through a myriad of channels, constantly recalibrating their actions.
The idea of using cells as medicine emerged with bone marrow transplants, and then CAR-T therapy for blood cancers. Now, scientists are beginning to engineer much more complex living therapeutics by tapping into the innate capabilities of living cells to treat a growing list of diseases.
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That includes solid tumors like cancers of the brain, breast, lung, or prostate, and also inflammatory diseases like diabetes, Crohns, and multiple sclerosis. One day, this work may extend to regenerating tissues outside or even inside the body.
Taking a page from computer engineers, biologists are trying their hands at programming cells by building DNA circuits to guide their protein-making machinery and behavior.
We need cells with GPS that never make mistakes in where they need to go, and with sensors that give them real-time information before they deliver their payload, said Hana El-Samad, PhD, a professor of biochemistry and biophysics. Maybe they kill a little bit and then deliver a therapeutic payload that cleans up. And the next program over encourages the rejuvenation of healthy cells.
These engineered cell therapies would be a huge leap from traditional therapies, like small molecules and biologics, which can only be controlled through dose, or combination, or by knowing the time it takes for the body to get rid of it.
If you put in drugs, you can block things and push things one way or the other, but you can't read and monitor whats going on, said Wendell Lim, PhD, a professor of cellular and molecular pharmacology who directs the Cell Design Institute at UCSF. A living cell can get into the disease ecosystem and sense what's going on, and then actually try to restore that ecosystem.
Like people, cells live in communities and share duties. They even take on new identities when the need arises, operating through unseen forces that biologists term, self-organizing.
We need cells with GPS that never make mistakes in where they need to go, and with sensors that give them real-time information before they deliver their payload.
Hana El-Samad, PhD
Some living cell therapies could be controlled even after they enter the body.
Lim and others say it is possible to begin adapting cells into therapy, even when so much has yet to be learned about human biology, because cells already know so much.
Their built-in power includes dormant embryonic abilities, so a genetic nudge in the right place could enable a cell to assume a new function, even something it has never done before.
When a cell, a building block thats 10 microns in diameter can do that, and you have 10 trillion of them in your body, its a whole new ballgame, said Zev Gartner, PhD, a professor of pharmaceutical chemistry who studies how tissues form. Were not talking about engineering in the same way that somebody working at Ford or Intel or Apple or anywhere else thinks about engineering. Its a whole new way of thinking about engineering and construction.
For several years now, synthetic biologists have been building rudimentary feedback circuits in model organisms like yeast by inserting engineered DNA programs. Recently, Lim and El-Samad put these circuits into mice to see if they could tamp down the excess inflammation from traumatic brain injury.
They demonstrated that engineered T-cells could get into the sites of injury in the brain and perform an immune-modulating function. But its just a prototype of what synthetic circuits could do.
You can imagine all kinds of scenarios of therapies that dont cause any side effects, and do not have any collateral damage, said El-Samad.
UCSF researchers are building ever more complex circuits to move cells around the body and sense their surroundings. They hope to load them with DNA programs that trigger the cells protein-making machinery to do things like remove cancerous cells, then repair the damage caused by the tumors haphazard growth.
Or they could make cells that send signals to finetune the immune system when it overreacts to a threat or mistakenly attacks healthy cells. Or build new tissue and organs from our bodys own cells to repair damage associated with trauma, disease, or aging.
The fact that biological systems and cellular systems can self-organize is a huge part of biology, and thats something were starting to program, Lim said. Then we can make cells that do the functions that we want. We aspire to not only have immune cells be better at killing and detecting cancer but also to suppress the immune system for autoimmunity and inflammation or go to the brain to fight degeneration.
These UCSF scientists are on their way to engineering cell-based solutions to different diseases.
Tejal Desai, PhD, a professor and chair of the Department of Bioengineering and Therapeutic Sciences, is employing nanotechnology to create tiny depots where cells that have been engineered to treat Type 1 diabetes or cancer can refuel with oxygen and nutrients.
Having growth factors or other factors that keep them chugging along is very helpful, she said. Certain cytokines help specific immune cells proliferate in the body. We can design synthetic particles that present cytokines and have a signal that says, Come over to me. Basically, a homing signal.
Ophir Klein, MD, PhD, a professor of orofacial sciences and pediatrics, employs stem cell biology to research treatments for birth defects and conditions like inflammatory bowel disease. He is working with Lim and Gartner to create circuits that induce cells to grow in new ways, for example to repair the damage to intestines in Crohns disease.
Cells and tissues are able to do things that historically we thought they were incapable of doing, Klein said. We dont assume that the way things happen or dont happen is the best way that they can happen, and were trying to figure out if there are even better ways.
Faranak Fattahi, PhD, a Sandler Faculty Fellow, is developing cell replacement therapy for damaged or missing enteric neurons, which regulate the muscles that move food through the GI tract. She generated these gut neurons using iPS cell technology.
What we want to do in the lab is see if we can figure out how these nerves are misbehaving and reverse it before transplanting them inside the tissue, she said. Now, she is working with Lim to refine the cells, so they integrate into tissues more efficiently without being killed off by the immune system and work better in reversing the disease.
Matthias Hebrok, PhD, a professor in the Diabetes Center, has created pancreatic islets, a complex cellular ecosystem containing insulin-producing beta cells, glucagon-producing alpha cells and delta cells.
Now, he is working on how to make islet transplants that dont trigger the immune system, so diabetes patients can receive them without immune-suppressing drugs.
We might be able to generate stem-cell derived organs that the recipients immune system will either recognize as self or not react to in a way that would disrupt their function.
In health, the community of cells in these islets perform the everyday miracle of keeping your blood sugar on an even keel, regardless of what you ate or drank, or how little or how much you exercised or slept.
To me, at least, thats the most remarkable thing about our cells, Gartner said. All of this stuff just happens on its own.
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Beyond CAR-T: New Frontiers in Living Cell Therapies - UCSF News Services
Firstly, two news items on glioblastoma that will be of particular interest to scientists at our Research Centre at Queen Mary, University of London. This brain tumour type is the most aggressive and most common primary high-grade tumour diagnosed in adults.
We begin with some fascinating research into a new stage of the stem cell life cycle could be the key to unlocking new methods of brain cancer treatment. Following brain stem cell analysis, through single-cell RNA sequencing, data mapped out a circular pattern that has been identified as all of the different phases of the cell cycle. A new cell cycle classifier tool then took a closer, high-resolution look at what's happening within the growth cycles of stem cells and identified genes that can be used to track progress through this cell cycle. When the research team analysed cell data for Gliomas, they found the tumour cells were often either in the Neural G0 or G1 growth state and that as the tumours became more aggressive, fewer and fewer cells remained in the resting Neural G0 state. They correlated this data with the prognosis for patients with Glioblastoma and found those with higher Neural G0 levels in tumour cells had less aggressive tumours. So, if more cells could be pushed into this quiescent, or sleepy, state tumours would become less aggressive. Current cancer drug treatments focus on killing cancer cells. However, when the cancer cells are killed, they release cell debris into the surrounding area of the tumour, which can cause the remaining cells to become more resistant to drugs. If, instead of killing cells, we put them to sleep could that potentially be a better way forward?
For the first time, scientists have discovered stem cells of the hematopoietic system in glioblastomas. These hematopoietic stem cells promote division of the cancer cells and at the same time suppress the immune response against the tumour so Glioblastomas. In tissue samples of 217 Glioblastomas, 86 WHO grade II and III Astrocytomas, and 17 samples from healthy brain tissue, researchers used computer-assisted transcription analysis to draw up profiles of the cellular composition. The tissue samples were taken directly from the post-surgery, resection margins - where remaining tumour cells and immune cells meet. The team were able to distinguish between signals from 43 cell types, including 26 different types of immune cells. To their great surprise, the researchers discovered hematopoietic stem and precursor cells in all the malignant tumour samples, while this cell type was not found in healthy tissue samples. An even more surprising observation was that these blood stem cells seem to have fatal characteristics: They suppress the immune system and at the same time stimulate tumour growth. When the researchers cultured the tumour-associated blood stem cells in the same petri dish as Glioblastoma cells, cancer cell division increased. At the same time, the cells produced large amounts of the PD-L1 molecule, known as an "immune brake", on their surface.
On diagnosis of an Ependymoma an adult is often treated with surgery followed by radiation. When a tumour comes back, there had been no standard treatment options. Recently, thats changed, thanks to results from the first prospective clinical trial for adults with Ependymoma, which showed the benefits of a combination regimen including a targeted drug and chemotherapy.
Also of relevance to our Research Centre at QMUL, a study may have identified the cell of origin of Medulloblastoma. Using organoids to simulate tumour tissue in 3D an approach also used by researchers at QMUL - this organoid model has enabled researchers to identify the type of cell that can develop into Medulloblastoma. These cells express Notch1/S100b, and play a key role in onset, progression and prognosis.
Research has been looking at how Medulloblastoma travels to other sites within the central nervous system and has shown that an enzyme called GABA transaminase, abbreviated as ABAT, aids metastases in surviving the hostile environment around the brain and spinal cord and in resisting treatment. These findings may provide clues to new strategies for targeting lethal Medulloblastoma metastases.
You can register to join an online lecture on the molecular analysis of paediatric Medulloblastoma and vulnerabilities, the development of models that recapitulate the patients diseases and how models allow to identify new therapies using a pre-clinical pipeline. It is on July 13th.
From the 12 15 of August you can watch The Masters Live World Course in Brain and Spine Tumour Surgery this event wont be streamed or saved on social media and registration is free.
Still focussing on neuro surgery this link takes you to a Neurosurgeon's guide to Cognitive Dysfunction in Adult Glioma
Grounds for optimism to end with as a prominent clinician/scientist believes Glioblastoma outcomes could change for the better soon. Frederick F. Lang Jr, MD, chair of neurosurgery at The University of Texas MD Anderson Cancer Centre, and a co-leader of the institutions Glioblastoma Moon Shot programme says I am optimistic that we are going to see changes in the survival as we start to [better] understand the groups of people we're treating, and as we separate out the tumours more precisely and classify them better. Then, as we understand the biology of [the disease] better and better, we're going to see changes in the near future terms of survival. The University of Texas MD Anderson Cancer Centre is pursuing several novel approaches, including viro-immunotherapy and genetically engineered natural killer cells to treat patients with GBM, while also conducting tumour analysis to better comprehend the disease.
Whether to find out more about the Glioblastoma tumour microenvironment work or research into Medulloblastoma carried out at our Queen Mary University of London (QMUL) centre, the techniques at the forefront of tumour neurosurgery being employed by Consultant Neurosurgeon Kevin ONeill at our Imperial College, London Centre or the work into Meningioma and Acoustic Neuroma ( Thursday was Acoustic Neuroma Awareness Day) that Professor Oliver Hanemann focuses on at our University of Plymouth Centre, it is always worth checking our Research News pages and for an overview of our research strategy check out Brain Tumour Research our research strategy.
Finally, a request for you all to support our #StopTheDevastation campaign click through, find out more, get involved and say #NoMore to brain tumours.
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Sleeper cells, cells of origin and hematopoietic stem cells - Brain Tumour Research
HOUSTON, July 6, 2021 /PRNewswire/ --Marker Therapeutics, Inc.(Nasdaq:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, today announced completion of the six-patient safety lead-in portion of the Company's Phase 2 trial of MT-401, its lead MultiTAA-specific T cell product candidate, for the treatment of post-transplant acute myeloid leukemia (AML).
"We are pleased with the results of the safety lead-in portion of the trial, in which all six patients met the safety endpoints following infusion of our MultiTAA-specific T cell therapy," said Mythili Koneru, M.D., Ph.D., Chief Medical Officer ofMarker Therapeutics. "We are currently enrolling patients in the main portion of our first Company-sponsored trial and continue to activate clinical sites across the U.S. We are looking forward to further advancing MT-401 in this disease setting. Despite recent advances in how hematological malignancies are treated, patients remain in urgent need of new therapeutic options."
About Marker's Phase 2 AML Post-Transplant Study
The multicenter Phase 2 AML study is evaluating the clinical efficacy of MT-401 in patients with AML following an allogeneic stem-cell transplant in both the adjuvant and active disease setting. In the adjuvant setting, approximately 120 patients will be randomized 1:1 to either MT-401 at 90 days post-transplant versus standard-of-care observation, while approximately 40 patients with active disease will receive MT-401 as part of the single-arm group.
The primary objectives of the trial are to evaluate relapse-free survival in the adjuvant group and determine the complete remission rate and duration of complete remission in active disease patients. Additional objectives include, for the adjuvant group, overall survival and graft-versus-host disease relapse-free survival while additional objectives for the active disease group include overall response rate, duration of response, progression-free survival and overall survival.
InApril 2020, the FDA granted Orphan Drug designation to MT-401 for the treatment of patients with AML following allogeneic stem cell transplant.
About Marker Therapeutics, Inc.Marker Therapeutics, Inc. is a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications. Marker's cell therapy technology is based on the selective expansion of non-engineered, tumor-specific T cells that recognize tumor associated antigens (i.e. tumor targets) and kill tumor cells expressing those targets. This population of T cells is designed to attack multiple tumor targets following infusion into patients and to activate the patient's immune system to produce broad spectrum anti-tumor activity. Because Marker does not genetically engineer its T cell therapies, we believe that our product candidates will be easier and less expensive to manufacture, with reduced toxicities, compared to current engineered CAR-T and TCR-based approaches, and may provide patients with meaningful clinical benefit. As a result, Marker believes its portfolio of T cell therapies has a compelling product profile, as compared to current gene-modified CAR-T and TCR-based therapies.
To receive future press releases via email, please visit: https://www.markertherapeutics.com/email-alerts.
Forward-Looking StatementsThis release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Statements in this news release concerning the Company's expectations, plans, business outlook or future performance, and any other statements concerning assumptions made or expectations as to any future events, conditions, performance or other matters, are "forward-looking statements." Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: our research, development and regulatory activities and expectations relating to our non-engineered multi-tumor antigen specific T cell therapies; the effectiveness of these programs or the possible range of application and potential curative effects and safety in the treatment of diseases; the timing, conduct and success of our clinical trials, including the Phase 2 trial of MT-401; and the overall market opportunity for our product candidates. Forward-looking statements are by their nature subject to risks, uncertainties and other factors which could cause actual results to differ materially from those stated in such statements. Such risks, uncertainties and factors include, but are not limited to the risks set forth in the Company's most recent Form 10-K, 10-Q and other SEC filings which are available through EDGAR at http://www.sec.gov. Such risks and uncertainties may be amplified by the COVID-19 pandemic and its impact on our business and the global economy. The Company assumes no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.
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Marker Therapeutics Announces Completion of Safety Lead-In Portion of Phase 2 Study in Post-Transplant AML - PRNewswire
Law360 (July 1, 2021, 5:54 PM EDT) -- The May 31 expiration of the U.S. Food and Drug Administration's enforcement discretion period for the regulatory oversight of cell and tissue products, coupled with a June 2 decision in the U.S. Court of Appeals for the Eleventh Circuit, pave the way for the FDA to take more aggressive action against companies, clinics and individuals using cells and tissues to create FDA-regulated products.
The regulation of human cells, tissues, and cellular and tissue-based products, which fall within the larger category of products known as regenerative medicine, is a legally complex area fraught with both misperceptions and misunderstandings. It is therefore critical...
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A New Era For FDA Regulation Of Cell And Tissue Products - Law360
DUBLIN--(BUSINESS WIRE)--The "Cell Therapy Global Market Report 2021: COVID-19 Growth and Change to 2030" report has been added to ResearchAndMarkets.com's offering.
The global cell therapy market is expected to grow from $7.2 billion in 2020 to $7.82 billion in 2021 at a compound annual growth rate (CAGR) of 8.6%.
Major players in the cell therapy market are Fibrocell Science Inc., JCR Pharmaceuticals Co. Ltd., PHARMICELL Co. Ltd., Osiris Therapeutics Inc., MEDIPOST, Vericel Corporation, Anterogen Co. Ltd., Kolon TissueGene Inc., Stemedica Cell Technologies Inc., and AlloCure.
The growth is mainly due to the companies resuming their operations and adapting to the new normal while recovering from the COVID-19 impact, which had earlier led to restrictive containment measures involving social distancing, remote working, and the closure of commercial activities that resulted in operational challenges. The market is expected to reach $12.06 billion in 2025 at a CAGR of 11%.
The cell therapy market consists of sales of cell therapy and related services. Cell therapy (CT) helps repair or replace damaged tissues and cells. A variety of cells are used for the treatment of diseases includes skeletal muscle stem cells, hematopoietic (blood-forming) stem cells (HSC), lymphocytes, mesenchymal stem cells, pancreatic islet cells, and dendritic cells.
The cell therapy market covered in this report is segmented by technique into stem cell therapy; cell vaccine; adoptive cell transfer (ACT); fibroblast cell therapy; chondrocyte cell therapy. It is also segmented by therapy type into allogeneic therapies; autologous therapies, by application into oncology; cardiovascular disease (CVD); orthopedic; wound healing; others.
The high cost of cell therapy hindered the growth of the cell therapy market. Cell therapies have become a common choice of treatment in recent years as people are looking for the newest treatment options. Although there is a huge increase in demand for cell therapies, they are still very costly to try. Basic joint injections can cost about $1,000 and, based on the condition, more specialized procedures can cost up to $ 100,000. In 2020, the average cost of stem cell therapy can range from $4000 - $8,000 in the USA. Therefore, the high cost of cell therapy restraints the growth of the cell therapy market.
Key players in the market are strategically partnering and collaborating to expand the product portfolio and geographical presence of the company. For instance, in April 2018, Eli Lilly, an American pharmaceutical company entered into a collaboration agreement with Sigilon Therapeutics, a biopharmaceutical company that focused on the discovery and development of living therapeutics to develop cell therapies for type 1 diabetes treatment by using the Afibromer technology platform.
Similarly, in September 2018, CRISPR Therapeutics, a biotechnological company that develops transformative medicine using a gene-editing platform for serious diseases, and ViaCyte, a California-based regenerative medicine company, collaborated on the discovery, development, and commercialization of allogeneic stem cell therapy for diabetes treatment.
In August 2019, Bayer AG, a Germany-based pharmaceutical and life sciences company, acquired BlueRock Therapeutics, an engineered cell therapy company, for $1 billion. Through this transaction, Bayer AG will acquire complete BlueRock Therapeutics' CELL+GENE platform, including a broad intellectual property portfolio and associated technology platform including proprietary iPSC technology, gene engineering, and cell differentiation capabilities. BlueRock Therapeutics is a US-based biotechnology company focused on developing engineered cell therapies in the fields of neurology, cardiology, and immunology, using a proprietary induced pluripotent stem cell (iPSC) platform.
The rising prevalence of chronic diseases contributed to the growth of the cell therapy market. According to the US Centers for Disease Control and Prevention (CDC), chronic disease is a condition that lasts for one year or more and requires medical attention or limits daily activities or both and includes heart disease, cancer, diabetes, and Parkinson's disease.
Stem cells can benefit the patients suffering from spinal cord injuries, type 1 diabetes, Parkinson's disease (PD), heart disease, cancer, and osteoarthritis. According to Cancer Research UK, in 2018, 17 million cancer cases were added to the existing list, and according to the International Diabetes Federation, in 2019, 463 million were living with diabetes.
According to the Parkinson's Foundation, every year, 60,000 Americans are diagnosed with PD, and more than 10 million people are living with PD worldwide. The growing prevalence of chronic diseases increased the demand for cell therapies and contributed to the growth of the market.
Key Topics Covered:
1. Executive Summary
2. Cell Therapy Market Characteristics
3. Cell Therapy Market Trends And Strategies
4. Impact Of COVID-19 On Cell Therapy
5. Cell Therapy Market Size And Growth
5.1. Global Cell Therapy Historic Market, 2015-2020, $ Billion
5.2. Global Cell Therapy Forecast Market, 2020-2025F, 2030F, $ Billion
6. Cell Therapy Market Segmentation
6.1. Global Cell Therapy Market, Segmentation By Technique, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion
6.2. Global Cell Therapy Market, Segmentation By Therapy Type, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion
6.3. Global Cell Therapy Market, Segmentation By Application, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion
7. Cell Therapy Market Regional And Country Analysis
7.1. Global Cell Therapy Market, Split By Region, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion
7.2. Global Cell Therapy Market, Split By Country, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion
Companies Mentioned
For more information about this report visit https://www.researchandmarkets.com/r/719lux
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Cell Therapy Global Market Report 2021: COVID-19 Growth and Change to 2030 - ResearchAndMarkets.com - Business Wire
Newswise University of Maryland School of Medicine (UMSOM) Professor of Diagnostic Radiology & Nuclear Medicine, Linda Chang, MD, MS, received the National Institute on Drug Abuse (NIDA) 2021 Avant Garde Award (DP1) for HIV/AIDS and Substance Use Disorder Research a National Institutes of Health (NIH) Directors Pioneer Award. This prestigious award supports researchers with exceptional creativity, who propose high-impact research with the potential to be transformative to the field. Her proposed project will involve a team of experts in brain imaging, infectious diseases, addiction, animal research, and gene-editing technology with the goal to essentially eradicate all traces of HIV from the body, and treat commonly co-existing substance use disorders. 2021 Avant Garde Awardees are expected to receive more than $5 million over five years.
I am extremely pleased, and feel very fortunate to have received this award, says Dr. Chang, who has a secondary appointment in the Department of Neurology at UMSOM. This project takes my work in a new direction. I believe my track record of being able to work across multiple disciplines with various researchers to initiate new areas of research and getting good results, along with the outstanding collaborators and resources at UMB, gave the proposal reviewers confidence that my team and I can significantly advance this new project.
About 38 million people around the world live with HIV, according to the Centers for Disease Control and Prevention. Although antiretroviral therapies can treat HIV to the point of undetectable viral levels and lead to long, healthy lifespans, these medications must be taken for life to prevent a resurgence, as HIV can hide from these drugs by integrating copies of itself into a persons genome. Once the drugs are stopped, the virus can reemerge.
From start to finish, Dr. Changs plan is to remove HIV from the genome, even in tough to reach spots like the brain, get more of the antiretroviral therapies into the brain, and stimulate the reward system in the brain to reduce drug cravings. The work will start out in mice before it can be tested in people.
Dr. Chang plans to use the gene-editing technology known as CRISPR to cut out copies of the hidden HIV genes in the genomes of mice, so they can be eradicated by antiretroviral drugs.
However, getting the CRISPR therapy into the brain can be difficult because of the blood-brain barrier, which protects the brain from infectious bacteria and foreign substances. The blood-brain barrier also prevents antiretroviral drugs from reaching high enough concentrations in the brain and central nervous system to effectively destroy HIV.
To seek out HIV in the brain, Dr. Chang and her team will temporarily disrupt the blood-brain barrier to allow more of the antiretroviral drugs or the CRISPR compounds to cross over the blood-brain barrier using an unique resource at the University of Marylandthe MRI-guided focused ultrasound system. This technique uses the MRI scan to help guide 2,000 pinpointed beams of high energy sound waves, along with microscopic bubbles, to non-invasively and temporarily open an area of the brain with the goal of eliminating the hidden reservoirs of virus in the brains immune cells.
About half of the people with HIV use substances, like drugs or alcohol, or have substance use disorders. Even tobacco or cannabis use in people with HIV is at 2-3 times that of the general population. Together with Victor Frenkel, PhD, an Associate Professor in the Department of Radiology and the Director of Translational Focused Ultrasound, and Donna Calu, PhD, Assistant Professor in the Department of Anatomy and Neurobiology, Dr. Chang will use low energy MR-guided focused ultrasound to suppress brain activity in the reward center of the brain, the nucleus accumbens. They hope this approach will suppress drug cravings in people with HIV who have substance use disorders.
The different components of this project will first be tested in mouse or rat models before moving onto clinical studies. As HIV does not normally infect mice, researchers use humanized mice that have weak immune systems, which are replaced with human blood stem cells that become human immune cells that can be infected with HIV. Although these humanized mice make lots of T cells a main cell for HIV infectionthey dont make the immune cells that HIV uses to hide in the brain, known as microglia. Recently, Dr. Changs collaborator Howard E. Gendelman, MD, Margaret R. Larson Professor of Internal Medicine and Infectious Diseases Chair at University of Nebraska Medical Center, and his lab created a modified humanized mouse that has an extra human gene that allows the human blood stem cells to now make microglia.
These new mice mean that these experiments can be done in a fraction of the time and cost and without the other hurdles that come along with using non-human primates, which are the only other animal that a special strain of HIV can infect, says collaborator Alonso Heredia, PhD, Associate Professor of Medicine and scientist at UMSOMs Institute of Human Virology.
He adds, There have been many attempts to eradicate HIV in the body, and it is thought they have not been successful, in part because we cannot get to the HIV reservoirs in the brain. If this works, we will be much closer to a practical cure for HIV. Dr. Heredia will be collaborating with Dr. Chang on this project using HIV-infected humanized mice that he has developed for his other ongoing projects.
For the addiction studies, Dr. Changs team will use the expertise and rodent models of addiction developed and optimized by Mary Kay Lobo, PhD, Professor of Anatomy and Neurobiology, and Dr. Calu. The mice will self-administer fentanyl, a powerful, synthetic opioid.
Dr. Frenkel and Dheeraj Gandhi, MBBS, Professor of Diagnostic Radiology and Nuclear Medicine and Clinical Director of Center of Metabolic Imaging and Therapeutics at UMSOM, are the teams MRI-guided focused ultrasound and clinical research experts.
My hearty congratulations to Dr. Chang and her colleagues and collaborators. If anything is called cutting edge this work surely qualifies for that praise. We wish this group all the success possible, said Robert C. Gallo, MD, The Homer & Martha Gudelsky Distinguished Professor in Medicine, Co-Founder and Director, Institute of Human Virology (IHV), University of Maryland School of Medicine, a Global Virus Network (GVN) Center of Excellence, and GVN Co-Founder and International Scientific Advisor.
Dr. Chang is an expert in using brain imaging to study how HIV or drug use affect the brain in adults and during adolescence, and how exposure to drugs in the womb affects childhood development. She has also conducted clinical trials for treating HIV-associated cognitive disorders and substance use disorders.
Dr. Chang joined UMSOM in 2017 through the Deans initiative Special Trans-Disciplinary Recruitment Award Program (STRAP). The STRAP Initiative was part of UMSOM's multi-year research strategy ACCEL-Med (Accelerating Innovation and Discovery in Medicine) to increase the quality and reputation of clinical and basic science research bringing UMSOM among other top-tier medical research schools.
Dr. Changs arrival to UMSOM spurred the exact kind of collaborative efforts we had hoped to foster through our recruitment program in order to accelerate discoveries, treatments and cures for the worlds most pressing diseases, says E. Albert Reece, MD, PhD, MBA, Executive Vice President for Medical Affairs, UM Baltimore, and the John Z. and Akiko K. Bowers Distinguished Professor and Dean, UMSOM.I look forward to following her teams progress on this ambitious project in the hope that one day we can eradicate HIV.
Dr. Chang served on the National Advisory Council on Drug Abuse for NIDA and is a current member on the Council of Councils at the NIH.
Now in its third century, the University of Maryland School of Medicine was chartered in 1807 as the first public medical school in the United States. It continues today as one of the fastest-growing, top-tier biomedical research enterprises in the world -- with 45 academic departments, centers, institutes, and programs; and a faculty of more than 3,000 physicians, scientists, and allied health professionals, including members of the National Academy of Medicine and the National Academy of Sciences, and a distinguished two-time winner of the Albert E. Lasker Award in Medical Research.
With an operating budget of more than $1.2 billion, the School of Medicine works closely in partnership with the University of Maryland Medical Center and Medical System to provide research-intensive, academic and clinically-based care for nearly 2 million patients each year. The School of Medicine has more than $563 million in extramural funding, with most of its academic departments highly ranked among all medical schools in the nation in research funding. As one of the seven professional schools that make up the University of Maryland, Baltimore campus, the School of Medicine has a total population of nearly 9,000 faculty and staff, including 2,500 student trainees, residents, and fellows.
The combined School of Medicine and Medical System (University of Maryland Medicine) has an annual budget of nearly $6 billion and an economic impact of more than $15 billion on the state and local community. The School of Medicine, which ranks as the 8th highest among public medical schools in research productivity, is an innovator in translational medicine, with 600 active patents and 24 start-up companies. The School of Medicine works locally, nationally, and globally, with research and treatment facilities in 36 countries around the world. Visitmedschool.umaryland.edu
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SHANGHAI and HONG KONG, July 5, 2021 /PRNewswire/ -- Antengene Corporation Limited ("Antengene", SEHK: 6996.HK), a leading innovative biopharmaceutical company dedicated to discovering, developing and commercializing global first-in-class and/or best-in-class therapeutics in hematology and oncology, recently announced that China's National Medical Products Administration (NMPA) has accepted the Investigational New Drug (IND) application for single agent selinexor, a first-in-class orally available Exportin 1 (XPO1) inhibitor, for the treatment of patients with myelofibrosis (MF) in China.
MF is a clonal hematologic neoplasm which can emerge either as primary MF, polycythemia vera (PV) or essential thrombocythemia (ET)[1]. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is currently the only curative treatment for MF. However, such treatment is difficult to carry out and has a low rate of success. According to the National Comprehensive Cancer Network (NCCN) Guidelines for the Treatment of MF, patients with intermediate-2 or high-risk MF ineligible for allo-HSCT and with a platelet count of 50109/L should be treated with ruxolitinib or fedratinib, while there are few follow-on treatment alternatives for patients failed or resistant to ruxolitinib. At present, only ruxolitinib has been approved for clinical treatment in China, and as a result, MF remains a disease with limited treatment options, representing an urgent unmet medical need.
This randomized, open-label, multicenter Phase II study is designed to evaluate the safety and efficacy of selinexor versus physician's choice (PC) in patients with MF who had at least six months of treatment with a JAK1/2 inhibitor. Approximately 112 patients with MF from 75 trial centers across the world will be randomized in a 1:1 ratio into one of the two treatment arms.
"This acceptance by the NMPA of the IND application for the China study of selinexor in patients with MF marks another major step forward in our effort to develop selinexor into a novel cancer drug. It also paves the way for our on-going exploration of additional indications for Antengene's novel assets," said Dr. Jay Mei, Founder, Chairman and CEO of Antengene. "We are hopeful that, through this novel drug candidate with strong potential in this disease, coupled with our deep expertise in the field of hematologic malignancies, we will be able to bring renewed hope to patients with MF in China. Moving forward, we will work closely with the NMPA to advance this trial in China, and strive to bring this innovative therapeutic to patients in the region and beyond."
About Selinexor (XPOVIO)
Selinexor is a first-in-class oral selective inhibitor of nuclear export (SINE) compound discovered and developed by Karyopharm Therapeutics Inc. (NASDAQ: KPTI), Selinexor is currently being developed by Antengene, which has the exclusive development and commercial rights in certain Asia-Pacific markets, including Greater China, South Korea, Australia, New Zealand and the ASEAN countries.
In July 2019, the US Food and Drug Administration (FDA) approved selinexor in combination with low-dose dexamethasone for the treatment of relapsed/refractory multiple myeloma (RRMM) and in June 2020 approved selinexor as a single-agent for the treatment of relapsed/refractory diffuse large B-cell lymphoma (RR DLBCL). In December 2020, selinexor also received FDA approval as a combination treatment for multiple myeloma after at least one prior therapy. In February 2021, selinexor was approved by the Israeli Ministry of Health for the treatment of patients with RRMM or RR DLBCL and in March 2021, the European Commission (EC) has granted conditional marketing authorization for selinexor (NEXPOVIO) for the treatment of adult patients with RRMM.
Selinexor is so far the first and only oral SINE compound approved by the FDA and is the first drug approved for the treatment of both MM and DLBCL. Selinexor is also being evaluated in several other mid-and later-phase clinical trials across multiple solid tumor indications, including liposarcoma and endometrial cancer. In November 2020, at the Connective Tissue Oncology Society 2020 Annual Meeting (CTOS 2020), Antengene's partner, Karyopharm, presented positive results from the Phase III randomized, double blind, placebo controlled, cross-over SEAL trial evaluating single agent, oral selinexor versus matching placebo in patients with liposarcoma. Karyopharm also announced that the ongoing Phase III SIENDO trial of selinexor in patients with endometrial cancer passed the planned interim futility analysis and the Data and Safety Monitoring Board (DSMB) recommended the trial should proceed as planned without any modifications. Top-line SIENDO trial results are expected in the second half of 2021.
Antengene is currently conducting five late-stage clinical trials of selinexor for the treatment of MM, DLBCL, non-small cell lung cancer, and peripheral T and NK/T-cell lymphoma. Furthermore, Antengene has submitted New Drug Applications (NDAs) for selinexor in multiple Asia-Pacific markets including China, Australia, South Korea, and Singapore, and was granted the Priority Review status by China's NMPA and an Orphan Drug Designation by the Ministry of Food and Drug Safety of South Korea (MFDS).
About Antengene
Antengene Corporation Limited ("Antengene", SEHK: 6996.HK) is a leading clinical-stage R&D driven biopharmaceutical company focused on innovative medicines for oncology and other life threatening diseases. Antengene aims to provide the most advanced anti-cancer drugs to patients in the Asia-Pacific Region and around the world. Since its establishment in 2017, Antengene has built a broad and expanding pipeline of clinical and pre-clinical stage assets through partnerships as well as in-house drug discovery, and obtained 15 investigational new drug (IND) approvals and submitted 5 new drug applications (NDA) in multiple markets in Asia Pacific. Antengene's vision is to "Treat Patients Beyond Borders". Antengene is focused on and committed to addressing significant unmet medical needs by discovering, developing and commercializing first-in-class/best-in-class therapeutics.
Forward-looking statements
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[1] J. Mascarenhas, B.K. Marcellino, M. Lu, M. Kremyanskaya, F. Fabris, L. Sandy, M. Mehrotra, J. Houldsworth, V. Najfeld, S. El Jamal, B. Petersen, E. Moshier, R. Hoffman, A phase I study of panobinostat and ruxolitinib in patients with primary myelofibrosis (PMF) and post-polycythemia vera/essential thrombocythemia myelofibrosis (post-PV/ET MF).
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SOURCE Antengene Corporation Limited
Company Codes: HongKong:6996
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Antengene Announces Acceptance of IND Application in China for the Phase II Clinical Trial of Single-Agent Selinexor for the Treatment of...