Category Archives: Cell Medicine

News Release

Tuesday, October 27, 2020

Researchers from the National Institutes of Health (NIH) have discovered a new inflammatory disorder called vacuoles, E1 enzyme, X-linked, autoinflammatory and somatic syndrome (VEXAS), which is caused by mutations in the UBA1 gene. VEXAS causes symptoms that included blood clots in veins, recurrent fevers, pulmonary abnormalities and vacuoles (unusual cavity-like structures) in myeloid cells. The scientists reported their findings in the New England Journal of Medicine.

Nearly 125 million people in the U.S. live with some form of a chronic inflammatory disease. Many of these diseases have overlapping symptoms, which often make it difficult for researchers to diagnose the specific inflammatory disease in a given patient.

Researchers at the National Human Genome Research Institute (NHGRI), part of the NIH, and collaborators from other NIH Institutes took a unique approach to address this challenge. They studied the genome sequences from more than 2,500 individuals with undiagnosed inflammatory diseases, paying particular attention to a set of over 800 genes related to the process of ubiquitylation, which helps regulate both various protein functions inside a cell and the immune system overall. By doing so, they found a gene that is intricately linked to VEXAS, a disease which can be life-threatening. So far, 40% of VEXAS patients who the team studied have died, revealing the devastating consequences of the severe condition.

Usually, researchers discover a previously unknown disease by studying several patients with similar symptoms, then searching for a gene or multiple genes that may play a role in causing the disease. However, this was not a viable option for the NIH research team.

We had many patients with undiagnosed inflammatory conditions who were coming to the NIH Clinical Center, and we were just unable to diagnose them, said David B. Beck, M.D., Ph.D., clinical fellow at NHGRI and lead author of the paper. Thats when we had the idea of doing it the opposite way. Instead of starting with symptoms, start with a list of genes. Then, study the genomes of undiagnosed individuals and see where it takes us.

Out of the genome sequences of 2,560 patients with undiagnosed inflammatory conditions, over 1,000 patients had undiagnosed recurrent fevers and body-wide inflammation. The rest, part of the NIH Undiagnosed Diseases Network, had unusual and unclassified disorders.

Our objective was to see if any of the 2,560 patients shared variations in the same gene, said Daniel Kastner, M.D., Ph.D., scientific director of the Intramural Research Program at NHGRI and a senior author of the paper. Instead of looking at clinical similarities, we were instead taking advantage of shared genomic similarities that could help us discover a completely new disease.

Out of the 800 genes, one stood out. Three middle-aged males had rare and potentially damaging genomic variants in the UBA1 gene, but each of the three males appeared to have two copies of the UBA1 gene with one copy harboring the mutation, which was not unexpected because humans usually have two copies of every gene. However, the UBA1 gene resides in the X chromosome, and males have only one X chromosome (and one Y chromosome).

We were amazed to see this and wondered what it could mean. And thats when it clickedthis was only possible if there was mosaicism in these men, said Dr. Beck.

Mosaicism occurs when some people have groups of cells with mutations that are different from the rest of the body. The team predicted that there were specific cells in the patients bodies that carried the UBA1 gene in its normal form while other cells carried the gene in its mutated form.

Using DNA-sequencing methodologies, the researchers found that the mosaicism was indeed present in the patients myeloid cells, which are responsible for systemic inflammation and act as the first line of defense against infections.

The researchers then analyzed the genome sequences of additional individuals from various NIH cohorts and databases, which led to the discovery of an additional 22 adult males with the UBA1 gene mutations. Most of the individuals had symptoms that included blood clots in veins, recurrent fevers, pulmonary abnormalities and vacuoles (unusual cavity-like structures) in the myeloid cells.

Out of the combined 25 individuals, researchers were able to find a link between the various clinical rheumatologic and blood-related diagnoses made for the patients. Because these conditions exist in people with UBA1 mutations, the team grouped the various conditions into a new disease: VEXAS.

By using this genome-first approach, we have managed to find a thread that ties together patients carrying all of these seemingly unrelated, disparate diagnoses, Dr. Kastner said.

The researchers hope that this new genome-first strategy will help healthcare professionals improve disease assessments and provide appropriate treatments for thousands of patients who have various inflammation-related conditions. The study may also pave the way for a new and more appropriate classification of inflammatory diseases.

Additional research support for this study was provided by the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute of Dental and Craniofacial Research, the National Heart, Lung, and Blood Institute, National Institute of Allergy and Infectious Diseases, the National Cancer Institute and the NIH Clinical Center.

NHGRI is one of the 27 institutes and centers at the National Institutes of Health. The NHGRI Extramural Research Program supports grants for research, and training and career development at sites nationwide. Additional information about NHGRI can be found athttps://www.genome.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

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Scientists use clues in the human genome to discover new inflammatory syndrome - National Institutes of Health

Find out which biopharma companies are raking in the cash this week, as companies from around the globe provide updates on their financing rounds and IPOs.

Foghorn Therapeutics

Foghorn boomed onto the NASDAQ with a $120 million IPO, surpassing its original expectations by $20 million. The Massachusetts-based biotech is investing in their proprietary Gene Traffic Control system, which tells the cells when to express which genes where and in what order within the chromatin regulatory system. The company currently has over 10 programs in its pipeline. Foghorn is anticipating filing an IND later this year for its lead candidate to begin a Phase I trial for the treatment of uveal melanoma, a cancer of the eye.

Scorpion Therapeutics

Scorpion Therapeutics aims to put the sting in cancer with a $108 million launch to pursue what they call precision oncology 2.0. The company plans to tackle the existing challenges of precision medicine with a three-prong approach developing better medicines for known cancer-driving mutations, developing drugs for targets previously thought to be undruggable, and finding new, untapped targets that could lead to even better drugs. Scorpion is focused on advancing several pipeline programs toward the clinic, while working on identifying new targets. We envision this being a robust pipelineits not about one or two programs, said Lina Gugucheva, Scorpion's chief business officer. Once were in the clinic, we will continue to add multiple programs into the clinic with no rate abatement, or speed abatement, in the next five- to 10-year horizon.

AavantiBio

Former Sarepta Therapeutics executive Bo Cumbo left to launch his new gene therapy company, AavantiBio, with a $107 million Series A. AavantiBios strategic partnership with University of Floridas Powell Gene Therapy Center provide their foundational research in rare genetic disorders. The company's lead program is aimed at Friedrichs Ataxia, a rare inherited genetic disease that causes cardiac and central nervous system dysfunction. AavantiBio has a unique opportunity to change the lives of those living with FA and other rare diseases, Cumbo said. Central to our mission is building on the advancements in gene transfer therapies and harnessing these revolutionary technologies to realize their potential as life-altering medicines.

Sirnaomics

An oversubscribed Series D will help biopharma company Sirnaomics' continued development of novel RNAi therapeutics to treat a range of disorders including cancers, fibrosis, metabolic diseases and viral infections. The $105 million round was co-led by existing investor Rotating Boulder Fund, and new investors Walvax Biotechnology and Sunshine Riverhead Capital. Sirnaomics is the only biopharma conducting R&D and clinical development in the field of RNAi therapeutics in both the U.S. and China. In April they saw positive results for their Phase IIa test of candidate STP705 in squamous cell carcinoma. They are preparing for an IPO in the near future.

Be Biopharma

Be Bio is a leader in developing B cells as medicines. B cells are prolific protein producers that can be collected from peripheral blood, have a programmable lifetime that could last decades, can target specific tissues, and have customizable functionality. Be Bio plans to use their $52 million Series A to precisely engineer B cells to treat a range of diseases. Our mission is to develop what we see as a new class of cell medicines that have a broad new pharmacology, Aleks Radovic-Moreno, Ph.D., said of B cells potential. We think it's a big new white space that's enabled by the rich biology of these cells.

Orbus Therapeutics

An expanded Series A will support Orbus ongoing STELLAR study, a Phase III clinical trial studying eflornithine in patients with anaplastic astrocytoma, a type of brain tumor, whose cancer has recurred following radiation and adjuvant chemotherapy. The $71 million funding includes an initial financing of $32.5 million in 2015. In the U.S., more than 3,600 new cases of anaplastic glioma are diagnosed each year with a median survival of just over three years despite treatment. The STELLAR studys primary endpoint is duration of overall survival in the 340 patients they plan to enroll.

Primmune Therapeutics

Primmune aims to advance the development of their novel orally-administered, small molecule toll-like receptor 7 agonists as therapeutic-adjuvants for acute viral diseases and cancer. The company's $27.4 million Series A gives it the capital to take their PRTX007 candidate into a short-term pharmacokinetic, pharmacodynamic and tolerability study, followed by a clinical proof-of-concept in an acute viral disease setting. Primmune's early data shows the potential for PRTX007 to drive the natural innate immune response to combat systemic diseases.

Prokarium

Privately-held Prokarium closed a $21 million Series B led by Korea Investment Partners with participation from the UK governments Future Fund, Flerie Invest and Riyadh Valley Company. The funds will be utilized to support the clinical development of Prokariums microbial immunotherapy for non-muscle invasive bladder cancer as well as expansion of its pipeline across multiple solid tumors. The bladder cancer therapy is expected to be in the clinic by 2022. Prokarium added Hyam Levitsky, MD, to its Board of Directors and strengthened the executive team with the following promotions: Kristen Albright, PharmD to COO, Livija Deban, Ph.D., to VP of Research and Peter McGowan, FCCA, as CFO.

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Biopharma Money on the Move: October 21-27 - BioSpace

Harvard Business School (HBS) and Harvards Department of Stem Cell and Regenerative Biology (HSCRB) have received a $25 million gift from Chris and Carrie Shumway (MBA 1993) to fund programs promoting leadership in life sciences, including the MS/MBA Biotechnology: Life Sciences, a joint degree offered by HBS and the Graduate School of Arts and Sciences and Harvard Medical School through HSCRB. The gift will help bridge the worlds of business and science by fostering an environment of collaboration and innovation at HBS and across Harvard, supporting work on pathbreaking research and educating a new generation of leaders in the life sciences. The gift will provide support for curriculum development and programming, faculty research, and student financial aid.

Every day we are contending with the challenges posed by a worldwide pandemic and seeing firsthand the importance of strong connections between the lab bench and the private sector, said Harvard President Larry Bacow. The Shumways have given us the opportunity to think deeply about how we train leaders at this intersection that will only grow in complexity and importance. We are grateful for their foresight and their exceptional generosity.

This gift will provide the University with the resources to support future leaders in life sciences, dramatically increase innovation and the commercialization of new discoveries, and efficiently and quickly take transformative discoveries from the laboratory to patients and the marketplace, said HBS Dean Nitin Nohria. The world needs more business leaders working at the intersection of science and society, with deep understanding of not only biotechnology and life sciences, but the management skills needed to help these important companies thrive.

To meet this need, the MS/MBA Biotechnology: Life Sciences Program, welcomed its first cohort of students this past August. In supporting financial aid that will cover the incremental costs of the MS/MBA program for these students, this gift will enable Harvard to attract and support outstanding students who already have experience and knowledge in life sciences. To recognize this support, the students in this program will be known as Shumway Fellows and this program will prepare them to take on leadership roles in biotechnology and life sciences organizations.

The program builds upon students existing biotech and life sciences knowledge and equips them with the latest business and scientific insights. This empowers them to launch, grow, and lead transformative organizations that will advance new drug discoveries or therapeutics. Students in the program study general management at HBS and life-sciences at HSCRB, which is a joint department of Harvards Faculty of Arts and Sciences (FAS) and Harvard Medical School (HMS). They benefit from the leadership of program co-chairs Amitabh Chandra (Henry and Allison McCance Professor of Business Administration at HBS and the Ethel Zimmerman Winer Professor of Public Policy and Director of Health Policy Research at the Harvard Kennedy School of Government) Mark Fishman (Professor of Stem Cell and Regenerative Biology at Harvard University and Chief of Pathways Consult Service at Massachusetts General Hospital), and Douglas Melton (Xander University Professor at Harvard University and Co-Director of the Harvard Stem Cell Institute).

The MS/MBA program is a collaborative effort from several schools across Harvard to fill a unique need we see in the industry, said Emma Dench, dean of GSAS. The program provides students with the opportunity to become conversant in both biomedical science and business, and this generous gift will help to ensure that we can prepare them to lead in the rapidly growing life sciences fields.

The future of life sciences innovation will increasingly require deep scientific knowledge coupled with targeted business acumen, said George Q. Daley, dean of HMS. With this joint degree, Harvard will be uniquely positioned to educate students to lead at this nexus.

Sometimes it is at the intersection of disciplines where we find opportunities to ask new questions that have the potential to completely change the way we think about a problem, said Edgerley Family Dean of the Faculty of Arts and Sciences Claudine Gay. This gift will enable Harvard to attract and support talented scholars in the life sciences and equip them with the tools they need to work at the interface of business and life sciences, asking the new questions that promote discovery and innovation in this important area.

Harvard has created an exceptional ecosystem that can develop and cultivate talented professionals across medicine, business and life sciences who are best positioned to tackle the worlds biggest global health challenges today and in the future, said Chris Shumway, managing partner and founder of Shumway Capital. As Harvard is geographically situated at the epicenter of the life sciences community with access to some of the worlds leading hospitals, pharma and biotechnology companies, we believe a tremendous opportunity exists to foster cross-pollination of ideas by marshalling the resources of Harvard and connecting with industry leaders to quickly understand and solve problems.

The gift will also support HBS as it engages and works closely with entrepreneurs and practitioners in the field on the creation of new research and the development of executive education training to serve their unique needs.

The acceleration of global health issues, including the current pandemic, demonstrates the need for entrepreneurial thinking, Chris Shumway added. Leaders born out of these programs will be equipped to drive organizations at the forefront of groundbreaking discoveries with the mindset needed to solve complex problems worldwide.

As an entrepreneur and Managing Partner of Shumway Capital, Chris Shumway has invested in, advised, and built growth businesses for over 25 years, including in biotechnology and life sciences. The Shumways are long-time supporters and advocates for philanthropic initiatives. As strong proponents of education reform, they established the Shumway Foundation with the primary goal of helping to break the cycle of poverty through better opportunities in education. The Shumway Foundation also actively supports other results-based non-profit organizations.

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Harvard Business School and Harvard's Department of Stem Cell and Regenerative Biology Receive $25 Million Gift from Chris and Carrie Shumway to...

MIAMI, Oct. 20, 2020 (GLOBE NEWSWIRE) -- Organicell Regenerative Medicine, Inc. (OTCBB: BPSR), a clinical-stage biopharmaceutical company dedicated to the development of regenerative therapies, announced that it has recently entered into an agreement with Regenerative Care Network (RCN). Organicell and RCN plan to collaborate on clinical research projects investigating use of Organicells lead therapeutic, Zofin, as a novel therapeutic for heart failure with preserved ejection fraction, known as HFpEF. This collaboration will focus on the conduct of clinical trials to take place at various medical institutions in Dallas and Houston, as soon as possible after an Investigational New Drug Application (IND) is filed with and approved by the FDA and Institutional Review Board (IRB) approval of the planned study is received.

HFpEF, ordiastolic heart failure, is a complex inflammatory disease in which the heart is unable to receive and pump adequate blood to meet the bodys needs. Among the approximately 6 million people in the USA with heart failure, about 50% have HFpEF, for which there is no effective treatment other than relief of congestion with diuretics.

RCN, under the leadership of its Medical Director, Dr. Vincent Friedewald, has extensive experience in managing patients with all forms of heart failure, including the large and growing population of patients with HFpEF. Dr. Friedewald is expected to be the principal clinical trial investigator for the planned study.

From reviewing our clinical data, we have good reason to believe that Zofin may potentially be an effective treatment for inflammatory heart conditions, such as HFpEF. This collaboration comes at timely importance due to mounting research that shows that COVID-19 patients are developing this disease as an indirect effect of the infection, said Dr. George Shapiro, the Chief Medical Officer at Organicell, who is himself a cardiologist.

About Regenerative Care Network: Regenerative Care Network is physician-owned andwas founded on the principal of providing regenerative cell therapyfor conditions that medical evidence supports as effective. Patients with conditions that have been shown to improve with regenerative cell therapyare given access to trainedphysicians who administer the appropriate cell therapy in compliance withthe State of Texas Laws.

About Organicell Regenerative Medicine, Inc.:

Organicell Regenerative Medicine, Inc. is a clinical-stage biopharmaceutical company that harnesses the power of nanoparticles to develop innovative biological therapeutics for the treatment of degenerative diseases. The companys proprietary products are derived from perinatal sources and manufactured to retain the naturally occurring microRNAs, without the addition or combination of any other substance or diluent. Based in South Florida, the company was founded in 2008 by Albert Mitrani, Chief Executive Officer and Dr. Maria Ines Mitrani, Chief Science Officer. To learn more, please visit https://organicell.com/.

About Zofin:

Zofin is an acellular biologic therapeutic derived from perinatal sources and is manufactured to retain naturally occurring microRNAs, without the addition or combination of any other substance or diluent. This product contains over 300 growth factors, cytokines, and chemokines as well as other extracellular vesicles/nanoparticles derived from perinatal tissues. Zofin is currently being tested in a phase I/II randomized, double blinded, placebo trial to evaluate the safety and potential efficacy of intravenous infusion of Zofin for the treatment of moderate to SARS related to COVID-19 infection vs placebo.

Forward-Looking Statements

Certain of the statements contained in this press release should be considered forward-looking statements within the meaning of the Securities Act of 1933, as amended (the Securities Act), the Securities Exchange Act of 1934, as amended (the Exchange Act), and the Private Securities Litigation Reform Act of 1995. These forward-looking statements are often identified by the use of forward-looking terminology such as will, believes, expects, potential or similar expressions, involving known and unknown risks and uncertainties. Although the Company believes that the expectations reflected in these forward-looking statements are reasonable, they do involve assumptions, risks and uncertainties, and these expectations may prove to be incorrect. We remind you that actual results could vary dramatically as a result of known and unknown risks and uncertainties, including but not limited to: potential issues related to our financial condition, competition, the ability to retain key personnel, product safety, efficacy and acceptance, the commercial success of any new products or technologies, success of clinical programs, ability to retain key customers, our inability to expand sales and distribution channels, legislation or regulations affecting our operations including product pricing, reimbursement or access, the ability to protect our patents and other intellectual property both domestically and internationally and other known and unknown risks and uncertainties, including the risk factors discussed in the Companys periodic reports that are filed with the SEC and available on the SECs website (http://www.sec.gov). You are cautioned not to place undue reliance on these forward-looking statements All forward-looking statements attributable to the Company or persons acting on its behalf are expressly qualified in their entirety by these risk factors. Specific information included in this press release may change over time and may or may not be accurate after the date of the release. Organicell has no intention and specifically disclaims any duty to update the information in this press release.

Media Contact:Rosh LoweMicDrop786-290-4400rosh@micdrop.one

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Organicell Partners with Regenerative Care Network to Study Potential Therapeutic Benefits of Zofin for Patients with Heart Failure - GlobeNewswire

Oct. 20, 2020 12:00 UTC

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of cancer, today announced the appointment of Matthew R. Patterson, a biotechnology executive with nearly 30 years of experience in research, development, and commercialization of innovative treatments, to its Board of Directors.

Matt is a highly respected leader in biotech, and for good reason, said Robert Ang, MBBS, MBA, Vors President and Chief Executive Officer. His expertise, guidance, and insights will be critical as we continue to advance our science towards the goal of developing potentially transformative therapies for patients with blood cancers.

Mr. Patterson has held senior leadership positions in both private and publicly-traded biotechnology companies. He is the co-founder of Audentes Therapeutics and was its Chief Executive Officer for eight years until its acquisition by Astellas Pharma in 2020; he also chaired the companys Board of Directors and continues to serve as a strategic advisor to the company. Additionally, he is a member of the Board of Directors of Homology Medicines, Inc., and the Board of Directors of 5:01 Acquisition Corp. Mr. Patterson also currently serves as the Chairman of the Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the gene and cell therapy and broader regenerative medicine sector.

Prior to Audentes, Mr. Patterson was an entrepreneur-in-residence with OrbiMed. Earlier in his career, he worked for Genzyme Corporation, BioMarin Pharmaceutical, and Amicus Therapeutics. Mr. Patterson received his bachelors degree in biochemistry from Bowdoin College.

Vors innovative approach to cell therapy and passionate team have the potential to transform the lives of cancer patients, Mr. Patterson said. I am excited to provide guidance and mentorship to Robert and the team as they continue to build a world class cell therapy company.

About Vor Biopharma

Vor Biopharma aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies. By removing biologically redundant proteins from eHSCs, these cells become inherently invulnerable to complementary targeted therapies while tumor cells are left susceptible, thereby unleashing the potential of targeted therapies to benefit cancer patients in need.

Vors platform could be used to potentially change the treatment paradigm of both hematopoietic stem cell transplants and targeted therapies, such as antibody drug conjugates, bispecific antibodies, and CAR-T cell treatments.

Vor is based in Cambridge, Mass. and has a broad intellectual property base, including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil.

About VOR33

Vors lead product candidate, VOR33, consists of engineered hematopoietic stem cells (eHSCs) that lack the protein CD33. Once these cells are transplanted into a cancer patient, we believe that CD33 will become a far more cancer-specific target, potentially avoiding toxicity to the normal blood and bone marrow associated with CD33-targeted therapies. Vor aims to improve the therapeutic window and effectiveness of CD33-targeted therapies, thereby potentially broadening the clinical benefit to patients suffering from acute myeloid leukemia.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201020005188/en/

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Vor Biopharma Appoints Matthew R. Patterson to its Board of Directors - BioSpace

Researchers use an enhanced technique to pattern unaltered cells within a 3D hydrogel, allowing them to recreate complex biological tissue for regenerative medicine.

Image credit: Dr. Manuel Gonzlez Reyes on Pixabay

The dream for regenerative medicine is to one day repair broken bones and tissue by simply growing a replacement using a patients own cells. To date, significant strides have been made in this field, but what a research team from the University of Pennsylvania have pointed out is that current replacement tissues are simplified models at best. In some cases, this is sufficient, but regenerated tissues or grafts generally fair better and last longer when their ability to mimic actual tissue is enhanced.

In a study recently published in Advanced Materials, the team, led by Professor Robert Mauck, director of the McKay Lab and a professor of orthopaedic surgery and bioengineering, developed a new way to rebuild complex biological tissues using a modified technique that allows them to deliberately place unaltered cells within a 3D hydrogel scaffold.

We found that we were able to arrange objects, such as cells, in ways that could generate new, complex tissues without having to alter the cells themselves, said the studys first author, Hannah Zlotnick, a graduate student in bioengineering who works in the McKay Orthopaedic Research Laboratory at Penn Medicine. Others have had to add magnetic particles to the cells so that they respond to a magnetic field, but that approach can have unwanted long-term effects on cell health. Instead, we manipulated the magnetic character of the environment surrounding the cells, allowing us to arrange the objects with magnets.

As proof-of-concept, the team looked at cartilage regeneration as a viable application of this new strategy as cartilage degeneration is a common occurrence that can lead to joint instability and chronic pain. Given the complexity of cartilage tissue, few treatments exist that have lasting effects. Current fixes are to fill those holes in with synthetic or biologic materials, which can work but often wear away because they are not the same exact material as what was there before, stated the authors in a statement. Its similar to fixing a pothole in a road by filling it with gravel and making a tar patch: the hole will be smoothed out but eventually wear away with use because its not the same material and cant bond the same way.

The complexity of cartilage tissue is what makes repairing it so challenging. There is a natural gradient from the top of cartilage to the bottom, where it contacts the bone, Zlotnick explained. Superficially, or at the surface, cartilage has a high cellularity, meaning there is a higher number of cells. But where cartilage attaches to the bone, deeper inside, its cellularity is low.

Zlotnick and her collaborators used a modified technique called magnetic patterning to create their cartilage mimics. While cells are already weakly diamagnetic (i.e., will respond to an applied magnetic field), usually these approaches require them to be magnetically tagged so that their position within the gel can be better manipulated. However, this can alter their properties and the treatments longevity. Ideally, objects should be magnetically manipulated without altering their intrinsic magnetic character, according to the authors.

Rather than magnetizing the cells, the team instead enhanced the magnetic susceptibility of the cellcontaining hydrogel and their position fixed by solidifying the surrounding solution afterward. The team showed successfully that the addition of a gadoliniumbased magnetic contrast agent to a hydrogel precursor allowed a variety of diamagnetic objects, such as cells, drug delivery agents, and polystyrene beads, to be patterned in response to a magnetic field. The objects can then be easily locked in place by exposing the hydrogel to UV light, which triggers a polymerization reaction called photo crosslinking, and then washing away the magnetic solution.

These magneto-patterned engineered tissues better resemble the native tissue, in terms of their cell disposition and mechanical properties, compared to standard uniform synthetic materials or biologics that have been produced, said Mauck. By locking cells and other drug delivering agents in place via magneto-patterning, we are able to start tissues on the appropriate trajectory to produce better implants for cartilage repair.

While there are still some hurdles to overcome before these materials can be used in a clinical setting, its an important step forward for the field of regenerative medicine.

This new approach can be used to generate living tissues for implantation to fix localized cartilage defects, and may one day be extended to generate living joint surfaces, Mauck explained.

Reference: Hannah M. Zlotnick, et al. MagnetoDriven Gradients of Diamagnetic Objects for Engineering Complex Tissues, Advanced Materials (2020). DOI: 10.1002/adma.202005030

Quotes adapted from University of Pennsylvania press release

Excerpt from:
Growing new cartilage with magnetic fields and hydrogels - Advanced Science News

Our world is suffering from metastatic cancer. Stage 4.

Ibram X. Kendi 2016 National Book Award winner, founder of the Boston University Center for Antiracist Research, and one of Time magazines 100 most influential people of 2020 boldly proclaims his diagnosis in his bestselling book How to Be an Antiracist.

The cancer he refers to is a metaphor for racism and the racist policies he says have spread to almost every part of the body politic.

But for Kendi, who has become a leading voice on systemic racism in America, cancer is more than a metaphor; its a personal reality. In 2013, his wife, a pediatrician, was diagnosed with stage 2 breast cancer at age 34. And less than five years later, Kendi himself was diagnosed with stage 4 metastatic colon cancer at age 35.

The link between racism and health has been laid bare this year as Kendi and his team at Boston University have tracked the racial breakdown behind COVID-19 data, revealing that Black people and people of color are infected and die at disproportionately high rates.

But is there a cure for racism?

Kendi will have a candid discussion with AAMC President and CEO David J. Skorton, MD, about how entrenched programs and policies have stood in the way of justice and equity at the AAMCs (Association of American Medical Colleges) annual meeting, Learn Serve Lead 2020: The Virtual Experience, on Monday, Nov. 16.

Kendi spoke with AAMCNews about his thoughts on how systemic racism and health care are connected and what academic medical institutions can do to change the prognosis.

The parallels include, first, that when I was diagnosed with cancer when someone came into a room and told me that I had cancer it was devastating. It hurt me. And I didnt want to believe it. In many ways, when individuals are diagnosed as being racist, it hurts them. They dont want to believe it. But I think whats different with the diagnosis of cancer, an individual does not view the doctor as seeking to hurt them, even though they feel hurt. If anything, they see the doctor as seeking to treat them, heal them. And what if, when we were diagnosed as being racist, we saw those individuals as trying to treat us, rather than attack us? And then, finally, I think when it comes to how we can treat metastatic racism that has literally spread to every part of the body politic and we know that because we can see the tumor cells of racial inequity everywhere its the same way we treat metastatic cancer. So, we can go in and surgically remove the racist policies that are, in effect, leading to those inequities.

We can also flood the body with the chemotherapy of anti-racist policies, which do two things: Either they reduce the size of racial inequities or they can prevent the reoccurrence of racial inequities.

According to the COVID Racial Data Tracker, which is a collaboration between our Center for Antiracist Research and the COVID Tracking Project by the Atlantic,nationwide, Black people are dying at 2.4 times the rate of White people, and Latinx and Native American people are also dying at much higher rates than White people. It just goes to show that we have a serious problem here of racial disparities, whether youre talking about COVID or cancer or heart disease, asthma, respiratory disease, and on down the line. That is not because there is something wrong with the behaviors of Black and Brown and Indigenous people. Its because theres something wrong with our society, our health care system who has access to health insurance, who has access to quality health care, whos more likely to be discriminated against by medical professionals, who has better access to preventative care, who is more likely to live in polluted neighborhoods. We have a serious problem and we need anti-racist policies to solve it.

Whats fascinating is, among many though certainly not all medical researchers and even those who are teaching medical students, there are still widespread beliefs in biological racial distinctions, and that is a basic assumption of too much medical research. If your basic assumption is that there is such thing as a Black disease or that White people have a particular biological makeup thats distinct from Native people, then youre going to ask research questions based on that. You have medical researchers right now asking questions based on these assumptions. For example, they say, You have these COVID racial disparities, so let me figure out whats distinct about the biological makeup of Black people thats causing them to die at higher rates. Is it because they have bigger noses? And what happens is that racist questions lead to racist answers.

I think Americans including, certainly, medical researchers should read books like Fatal Invention: How Science, Politics, and Big Business Re-create Race in the Twenty-First Century by Dorothy E. Roberts, which completely debunks the notion of biological races. For example, take a disease like sickle cell anemia, which is considered to be a Black disease as opposed to a malarial disease. In other words, it is a disease where people in the United States who originate from areas with higher concentrations of malaria are more likely to have the sickle cell: whether thats sub-Saharan Africa, whether thats Southern Europe, whether thats parts of Latin America. So, what happens is, if you believe this is a Black disease, then youre going to miss that Portuguese American who could have sickle cell; youre going to miss that Latin American who could have sickle cell too. I just dont think people realize how much of their medical research and practice is based in racist ideas, and that is leading to malpractice and pseudoscience.

I am as many people are advocating for our medical professionals to racially reflect the people who they are serving, and so anything that the leader can do to diversify the staff if need be of their institution is helpful. But I think its also critically important that institutions are really taking stock of how medical professionals are treating different population groups. One of the things that medical leaders can do is they can collect racial data. For example, to take this off of medicine, lets say were collecting a tremendous amount of data on a police officer: the racial makeup of every single person he stopped, the racial makeup of every single person who he stopped and frisked, the racial makeup of every single person he had a violent altercation with. Then what happens is you can begin to see, as a leader, patterns patterns of racism, patterns of racist practices that then allow you to be like, OK, this cop should probably not be policing Black and Brown people because theyre policing in a neighborhood thats majority White, but the majority of the people theyre pulling over and having violent altercations with are Black. Its the same thing for a medical professional. Is that medical professional less likely to provide pain medicine to Black patients? Is it more likely for, lets say, Latinx patients to complain about that medical professional? And also, this isnt necessarily just for the leader. Its for the medical professional herself himself because people have racist ideas and it may be coming out in the way that they provide care and they may not even realize it. I think this will allow them to realize it and potentially change.

I define racism with an m as a powerful collection of racist policies that are leading to racial inequity that are substantiated by racist ideas of racial hierarchy. Is there a cure for racism? There is. Its anti-racism. So, if you have an institution that is governed by policies that are leading to racial inequities and then the people in the institution believe that, for instance, White people are smarter, then theyre not going to see it as a problem when White people are in the most senior positions. But that same institution can eliminate those policies, can replace them with anti-racist policies that are leading to racial equity. There could be White people in senior positions and people of color in senior positions and theres equity there, and people would view that as normal.

So anti-racism is the cure for racism.

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Is there a "cure" for racism? Celebrated anti-racist activist and author Ibram X. Kendi says yes, there is - AAMC

Thermo Fisher Scientific has launched a modular, closed cell therapy processing system, Gibco CTS Rotea Counterflow Centrifugation System, which aids in scalable, cost-effective cell therapy development and manufacturing.

The modular system is the first Gibco instrument that enables workflows from research to GMP clinical development and commercial manufacturing.

According to the data from the Alliance for Regenerative Medicine, 675 clinical trials are progressing for cell therapy and cell-based immune-oncology around the world.

However, few of these developing cell therapies reach the commercial stage due to many factors.

These factors include safety and efficacy requirements, difficulties in transferring research protocols to manufacturing processes, lack of scalability, high cost, labour and equipment, as well as the complexity of the processes.

A modular, closed system for cell processing enables decoupling of time-consuming processes from rapid processes, improving facility and equipment usage and cutting costs.

Using CTS Rotea system from research through process development and commercial manufacturing reduces process delays associated with changing systems.

The use of sterile, closed, single-use kits aid cell processing in grade C clean rooms, enabling cost-effective transfer and scale-out of processes.

Thermo Fisher Scientific biosciences business president Amy Butler said: Our goal is to help advance the development of cell therapies, including exciting new CAR T cell therapies and even potential cell therapies to repair lung damage caused by Covid-19.

The CTS Rotea system will help researchers overcome manufacturing hurdles and bring the vast potential of cell therapies to more patients.

Designed to integrate effortlessly into existing workflows, the multifunctional system processes low to mid-range input volumes and deliver low output volumes.

The system has an instrument, closed sterile single-use kit and user-programmable software.

It can support a broad range of protocols for cell separation, washing and concentration with a cell recovery yield of over 95%.

Medical Device Contract Product Development and Manufacturing Services

Pressure Sensors and Technologically Advanced Microelectronics

Custom Single Screw Extruders, Extrusion Systems and Screws

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Thermo Fisher Scientific introduces cell therapy processing system - Medical Device Network

ROSEMONT, Pa., Oct. 19, 2020 /PRNewswire/ - Medunik USA is proud to announce that Siklos (hydroxyurea), indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in children, 2 years of age and older, with sickle cell anemia with recurrent moderate to severe painful crises1, is now available in a 100 mg scored tablet in addition to the 1,000 mg triple-scored tablets.

Siklos has a Boxed Warning regarding low blood cell counts and cancer; please read Important Safety Information below.

"Medunik USA is proud to offer pediatric SCA patients a new option that helps optimize daily dosing. With the newly available Siklos 100 mg scored tablets, dose adjustments can now be made in 50 mg increments. Coupled with Siklos 1,000 mg triple-scored tablets (4 x 250 mg), this will offer more accurate dose adjustmentsand may make it more convenient than compounded hydroxyurea," affirmed Tanya Carro, General Manager, at Medunik USA.

The importance of optimal dosing of hydroxyurea in sickle cell anemia (SCA) patients, based on patient body weight and biological and clinical response, has been well established.2,3 This is particularly relevant in pediatric populations, where patient weight is constantly changing.

In addition to its flexible dosing, Siklos may help increase patient compliance, as it is dissolvable in water for patients who are unable to swallow tablets whole.4 Siklos tablets should be taken once daily, at the same time every day, with a glass of water. For patients who are not able to swallow the tablets, they can be dispersed immediately before use in a small quantity of water in a teaspoon.

"I can't stress enough the importance of treating pediatric patients as early as possible. Why wait for painful episodes to occur and risk complications when you can give a child Siklos and reduce the frequency of recurrent painful crises once they turn 2? There's no reason to wait. Physicians and parents need to know about Siklos!," said Dr. Corey Hebert, MD, Chief Medical Officer at Dillard University and well-known medical broadcast journalist.

Medunik is committed to providing Siklos at the lowest possible cost to all patients. That is why the company continues to offer cost savings and free home delivery through the Siklos At Home program.

For more information about prescribing Siklos tablets, please visit siklosusa.com.

SIKLOS (hydroxyurea) tablets, for oral use

WHAT IS SIKLOS?

SIKLOS is a prescription medicine that is used to reduce the frequency of painful crises and reduce the need for blood transfusions in children, 2 years of age and older, with sickle cell anemia with recurrent moderate to severe painful crises.

It is not known if SIKLOS is safe and effective in children less than 2 years of age.

IMPORTANT SAFETY INFORMATION

WARNING: LOW BLOOD CELL COUNT and CANCERSee full prescribing information for complete Boxed Warning.

WHAT IS THE MOST IMPORTANT INFORMATION YOU SHOULD KNOW ABOUT SIKLOS?

WHO SHOULD NOT TAKE SIKLOSDo not take SIKLOS if you are allergic to hydroxyurea or any of the ingredients inSIKLOS.See the Medication Guide for a list of the ingredients inSIKLOS.

WHAT SHOULD YOU TELL YOUR HEALTH CARE PROVIDER BEFORE TAKING SIKLOS?Tell your healthcare provider about all of your medical conditions, including if you:

Tell your healthcare provider about all the medicines you take,including prescription and over-the-counter medicines, vitamins, and herbal supplements.

WHAT ARE THE POSSIBLE SIDE EFFECTS OF SIKLOS?

SIKLOS may cause serious side effects, including:

See "What is the most important information I should know about SIKLOS?"

The most common side effects of SIKLOS include:

These are not all the possible side effects of SIKLOS.

You are encouraged to report negative side effects of prescription drugs to the FDA at http://www.fda.gov/medwatch, or 1-800-FDA-1088.

Please read the Full Prescribing Information, including Boxed Warning, Medication Guide and Instructions for Use, at http://www.SIKLOSusa.com.

For more information about SIKLOS, we invite you to contact our Medical Information Service at 1 844-884-5520 or https://www.medunikusa.com/en/medical-information-service.

About Medunik USABased in Pennsylvania, Medunik USA works to improve the health and quality of life of Americans living with rare diseases by making orphan drug therapies available in the United States. With strategic partnerships at the global level, the company has critical experience in approval and market access processes as well as the marketing of orphan drug therapies. Medunik USA makes critical medications to treat rare diseases available to American patients who might not otherwise have access to these medications. For more information, visit: http://www.medunikusa.com.

References1. Siklos (hydroxyurea) tablets, for oral use [Prescribing Information]. Addmedica, May 20182. Optimizing hydroxyurea therapy for sickle cell anemia, Ware et al. Hematology Am Soc Hematol Educ Program. 2015;2015:436-432.3. Hydroxyurea for the Treatment of Sickle Cell Disease: Efficacy, Barriers, Toxicity, and Management in Children, Strouse et al. Pediatric Blood Cancer. 2012 August; 59(2): 365371.4. Parental and Other Factors Associated with Hydroxyurea Use for Pediatric Sickle Cell Disease, Oyeku et al. Pediatr Blood Cancer. 2013 April ; 60(4): 653658.

SOURCE Medunik USA

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Siklos, the first and only hydroxyurea-based treatment for pediatric patients with sickle cell anemia, now available in 100 mg scored tablets to help...

When first- and second-line treatments have been exhausted, few options remain for patients with advanced head and neck cancer.

A new phase 2 clinical trial by researchers at the University of Michigan Rogel Cancer Center found the drug axitinib was able to extend the lives of these patients by several months, and also identified a subset of patients with a specific mutation for whom the drug is likely to work best.

Survival increased from less than 6 months with the current standard treatments to nearly 10 months in the 28 patients enrolled in the trial, the research team reported in the journal Cancer. Additionally, 75% of patients with alterations in the PI3K signaling pathway, which is involved in cell cycle regulation, had a good response to the therapy, versus 17% of those without the alterations.

These are patients with metastatic cancer for whom there are no good options outside of clinical trials, says study first author Paul Swiecicki, M.D., an oncologist at Michigan Medicine, U-Ms academic medical center. And its a very timely study because tyrosine kinase inhibitors like axitinib, which target tumors blood supply, have shown considerable synergy when combined with immunotherapy.

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One of the stumbling blocks for this type of combination therapy has been the significant side effects that occur when the two approaches are used at the same time, he says. The current study, however, showed that axitinib may actually prime the body in a way that makes subsequent immunotherapy more effective.

Although the number of patients was small, our study was able to look at what happened to those who received immunotherapy after axitinib and found these patients cancers responded extremely well similar to what weve seen from other studies where people received the treatments simultaneously, Swiecicki says. This supports the idea that we may be able to combine the two approaches in a new way by giving them sequentially rather than at the same time, which should cut down on the severity of the side effects.

Axitinib is currently approved for the treatment of renal cell carcinoma.

Based on the mechanism of action of the drug, and what we know about how head and neck cancer grows, we were optimistic it could make a difference for head and neck cancer patients, Swiecicki says.

The study is innovative in another way: it applies new criteria for measuring the effectiveness of axitinib.

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Axitinib Improves Survival for Patients with Incurable Head and Neck Cancer - Michigan Medicine