Stem Cell Clinic

ONLINE: The Future of Medicine – Isthmus

Watch here: https://www.youtube.com/watch?feature=youtu.be&v=VVkQU91KbEs

press release: The UW has a long history of pioneering medical advancements that have transformed the world. From performing the first bone marrow transplant in the United States to cultivating the first laboratory-derived human embryonic stem cells. Now, where will UW medical research go next?

On the next Wisconsin Medicine Livestream, meet trailblazing doctors, researchers, and medical leaders who are charting a bold course to completely alter the health care landscape. During this insightful panel discussion, well explore how gene therapy and cell replacements could hold the keys to treating inherited and acquired blindness. Youll also discover the remarkable potential in xenotransplantation where nonhuman animal source organs are transplanted into human recipients. In addition, you will learn about UW Healths journey to build a multidisciplinary program to serve the community. These, and other, fascinating developments in treatment and care are happening right now at the UW and are the future of medicine. The presentation will be moderated by Robert Golden, the dean of the University of WisconsinMadisons School of Medicine and Public Health.

Our Guests:

David Gamm, professor, Department of Ophthalmology and Visual Sciences; Emmett A. Humble Distinguished Director, McPherson Eye Research Institute; Sandra Lemke Trout Chair in Eye Research

Dr. Gamms lab is at the forefront in developing cell-based therapies to combat retinal degenerative diseases (RDDs). As the director of the McPherson Eye Research Institute and a member of the Waisman Center Stem Cell Research Program, the UW Stem Cell and Regenerative Medicine Center, and the American Society for Clinical Investigation, his efforts are directed toward basic and translational retinal stem cell research. The Gamm Lab uses induced pluripotent stem cells to create retinal tissues composed of authentic human photoreceptor cells rods and cones that can detect light and initiate visual signals in a dish. The aims of his laboratory are to investigate the cellular and molecular events that occur during human retinal development and to generate cells for use in retinal disease modeling and cell replacement therapies. In collaboration with other researchers at UWMadison and around the world, the lab is developing methods to produce and transplant photoreceptors and/or retinal pigment epithelium (RPE) in preparation for future clinical trials. At the same time, the Gamm Lab uses lab-grown photoreceptor and RPE cells to test and advance a host of other experimental treatments, including gene therapies. In so doing, the lab seeks to delay or reverse the effects of blinding disorders, such as retinitis pigmentosa and age-related macular degeneration, and to develop or codevelop effective interventions for these RDDs at all stages of disease.

Dhanansayan Shanmuganayagam, assistant professor, Department of Surgery, School of Medicine and Public Health; Department of Animal and Dairy Sciences, UWMadison; director, Biomedical, and Genomic Research Group

Dr. Shanmuganayagams research focuses on the development and utilization of pigs as homologous models to close the translational gap in human disease research, taking advantage of the overwhelming similarities between pigs and humans in terms of genetics, anatomy, physiology, and immunology. He and his colleagues created the human-sized Wisconsin Miniature Swine breed that is unique to the university. The breed exhibits greater physiological similarity to humans, particularly in vascular biology and in modeling metabolic disorders and obesity. He currently leads genetic engineering of swine at the UW. His team has created more than 15 genetic porcine models including several of pediatric genetic cancer-predisposition disorders such as neurofibromatosis type 1 (NF1). In the context of NF1, his lab is studying the role of alternative splicing of the nf1 gene on the tissue-specific function of neurofibromin and whether gene therapy to modulate the regulation of this splicing can be used as a viable treatment strategy for children with the disorder.

Dr. Shanmuganayagam is also currently leading the efforts to establish the University of Wisconsin Center for Biomedical Swine Research and Innovation (CBSRI) that will leverage the translatability of research in pig models and UWMadisons unique swine and biomedical research infrastructure, resources, and expertise to conduct innovative basic and translational research on human diseases. The central mission of CBSRI is to innovate and accelerate the discovery and development of clinically relevant therapies and technologies. The center will also serve to innovate graduate and medical training. As the only center of its kind in the United States, CBSRI will make UWMadison a hub of translational research and industry-partnered biomedical innovation.

Petros Anagnostopoulos, surgeon in chief, American Family Childrens Hospital; chief, Section of Pediatric Cardiothoracic Surgery; professor, Department of Surgery, Division of Cardiothoracic Surgery

Dr. Anagnostopoulos is certified by the American Board of Thoracic Surgery and the American Board of Surgery. He completed two fellowships, one in cardiothoracic surgery at the University of Pittsburgh School of Medicine and a second in pediatric cardiac surgery at the University of California, San Francisco School of Medicine. He completed his general surgery residency at Henry Ford Hospital in Detroit. Dr. Anagnostopoulos received his MD from the University of Athens Medical School, Greece. His clinical interests include pediatric congenital heart surgery and minimally invasive heart surgery.

Dr. Anagnostopoulos specializes in complex neonatal and infant cardiac reconstructive surgery, pediatric heart surgery, adult congenital cardiac surgery, single ventricle palliation, extracorporeal life support, extracorporeal membrane oxygenation, ventricular assist devices, minimally invasive cardiac surgery, hybrid surgical-catheterization cardiac surgery, off-pump cardiac surgery, complex mitral and tricuspid valve repair, aortic root surgery, tetralogy of Fallot, coronary artery anomalies, Ross operations, obstructive cardiomyopathy, and heart transplantation.

When: Tuesday, Sept. 29, at 7 p.m. CDT

Where: Wisconsin Medicine Livestream: wiscmedicine.org/programs/ending-alzheimers

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ONLINE: The Future of Medicine - Isthmus

funded study sheds light on abnormal neural function in rare genetic disorder – National Institutes of Health

News Release

Monday, September 28, 2020

Findings show deficits in the electrical activity of cortical cells; possible targets for treatment for 22q11.2 deletion syndrome.

A genetic study has identified neuronal abnormalities in the electrical activity of cortical cells derived from people with a rare genetic disorder called 22q11.2 deletion syndrome. The overexpression of a specific gene and exposure to several antipsychotic drugs helped restore normal cellular functioning. The study, funded by the National Institutes of Health (NIH) and published in Nature Medicine, sheds light on factors that may contribute to the development of mental illnesses in 22q11.2 deletion syndrome and may help identify possible targets for treatment development.

22q11.2 deletion syndrome is a genetic disorder caused by the deletion of a piece of genetic material at location q11.2 on chromosome 22. People with 22q11.2 deletion syndrome can experience heart abnormalities, poor immune functioning, abnormal palate development, skeletal differences, and developmental delays. In addition, this deletion confers a 20-30% risk for autism spectrum disorder (ASD) and an up to 30-fold increase in risk for psychosis. 22q11.2 deletion syndrome is the most common genetic copy number variant found in those with ASD, and up to a quarter of people with this genetic syndrome develop a schizophrenia spectrum disorder.

This is the largest study of its type in terms of the number of patients who donated cells, and it is significant for its focus on a key genetic risk factor for mental illnesses, said David Panchision, Ph.D., chief of the Developmental Neurobiology Program at the NIHs National Institute of Mental Health. Importantly, this study shows consistent, specific patient-control differences in neuronal function and a potential mechanistic target for developing new therapies for treating this disorder.

While some effects of this genetic syndrome, such as cardiovascular and immune concerns, can be successfully managed, the associated psychiatric effects have been more challenging to address. This is partly because the underlying cellular deficits in the central nervous system that contribute to mental illnesses in this syndrome are not well understood. While recent studies of 22q11.2 deletion syndrome in rodent models have provided some important insights into possible brain circuit-level abnormalities associated with the syndrome, more needs to be understood about the neuronal pathways in humans.

To investigate the neural pathways associated with mental illnesses in those with 22q11.2 deletion syndrome, Sergiu Pasca, M.D., associate professor of psychiatry and behavioral sciences at Stanford University, Stanford, California, along with a team of researchers from several other universities and institutes, created induced pluripotent stems cells cells derived from adult skin cells reprogramed into an immature stem-cell-like state from 15 people with 22q11.2 deletion and 15 people without the syndrome. The researchers used these cells to create, in a dish, three-dimensional brain organoids that recapitulate key features of the developing human cerebral cortex.

What is exciting is that these 3D cellular models of the brain self-organize and, if guided to resemble the cerebral cortex, for instance, contain functional glutamatergic neurons of deep and superficial layers and non-reactive astrocytes and can be maintained for years in culture. So, there is a lot of excitement about the potential of these patient-derived models to study neuropsychiatric disease, said Dr. Pasca.

The researchers analyzed gene expression in the organoids across 100 days of development. They found changes in the expression of genes linked to neuronal excitability in the organoids that were created using cells from individuals with 22q11.2 deletion syndrome. These changes prompted the researchers to take a closer look at the properties associated with electrical signaling and communication in these neurons. One way neurons communicate is electrically, through controlled changes in the positive or negative charge of the cell membrane. This electrical charge is created when ions, such as calcium, move into or out of the cell through small channels in the cells membrane. The researchers imaged thousands of cells and recorded the electrical activity of hundreds of neurons derived from individuals with 22q11.2 deletion syndrome and found abnormalities in the way calcium was moved into and out of the cells that were related to a defect in the resting electrical potential of the cell membrane.

A gene called DGCR8 is part of the genetic material deleted in 22q11.2 deletion syndrome, and it has been previously associated with neuronal abnormalities in rodent models of this syndrome. The researchers found that heterozygous loss of this gene was sufficient to induce the changes in excitability they had observed in 22q11.2-derived neurons and that overexpression of DGCR8 led to partial restoration of normal cellular functioning. In addition, treating 22q11.2 deletion syndrome neurons with one of three antipsychotic drugs (raclopride, sulpiride, or olanzapine) restored the observed deficits in resting membrane potential of the neurons within minutes.

We were surprised to see that loss in control neurons and restoration in patient neurons of the DGCR8 gene can induce and, respectively, restore the excitability, membrane potential, and calcium defects, said Pasca. Moving forward, this gene or the downstream microRNA(s) or the ion channel/transporter they regulate may represent novel therapeutic avenues in 22q11.2 deletion syndrome.

Grants:MH107800; MH100900; MH085953; MH060233; MH094714

About the National Institute of Mental Health (NIMH):The mission of theNIMHis to transform the understanding and treatment of mental illnesses through basic and clinical research, paving the way for prevention, recovery and cure. For more information, visit theNIMH website.

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

Khan, T. A., Revah, O., Gordon, A., Yoon, S., Krawisz, A. K., Goold, C., Sun, Y., Kim, C., Tian, Y., Li, M., Schaepe, J. M., Ikeda, K., Amin, N. D., Sakai, N., Yazawa, M., Kushan, L., Nishino, S., Porteus, M. H., Rapoport, J. L. Paca, S. (2020). Neuronal defects in a human cellular model of 22q11.2 deletion syndrome. Nature Medicine. doi: 10.1038/s41591-020-1043-9

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funded study sheds light on abnormal neural function in rare genetic disorder - National Institutes of Health

Stem Cell Market Potential Growth, Size, Share, Demand and Analysis of Key Players Research Forecasts to 2027 – The Daily Chronicle

Fort Collins, Colorado The Stem Cell Market is growing at a rapid pace and contributes significantly to the global economy in terms of turnover, growth rate, sales, market share and size. The Stem Cell Market Report is a comprehensive research paper that provides readers with valuable information to understand the basics of the Stem Cell Report. The report describes business strategies, market needs, dominant market players and a futuristic view of the market.

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Stem cell market garnered a revenue of USD 9.45 billion in the year 2019 globally and has been foreseen to yield USD 15.55 billion by the year 2027 at a compound annual growth (CAGR) of 8.0% over the forecast period.

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Stem Cell Market, By Therapy (2016-2027)

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The market is geographically spread across several key geographic regions and the report includes regional analysis as well as production, consumption, revenue and market share in these regions for the 2020-2027 forecast period. Regions include North America, Latin America, Europe, Asia Pacific, the Middle East, and Africa.

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Stem Cell Market Potential Growth, Size, Share, Demand and Analysis of Key Players Research Forecasts to 2027 - The Daily Chronicle

The new pharma collaborations driving transformative research in oncology – – pharmaphorum

The pharmaceutical industry is one of the most scientifically innovative and competitive industries, particularly in oncology. As of 2018, there were over 1,100 cancer therapies in development, and as of 2020, 362 of them were cell and gene therapies. As a result, there is a need for continued innovation and increased efficiency in terms of drug development to manage cost, complexity and speed to provide potentially transformative therapies for cancer patients.

Within the last two decades, large pharmaceutical corporations have established themselves firmly in oncology by prioritising internal R&D efforts, as well as developing and accessing novel science and technology through collaborations and alliances with biotech companies and academic institutions.

Dramatic advances in the understanding of basic molecular mechanisms of underlying disease has continued to shift R&D focus toward precision medicine choosing the right therapy for a patient based on molecular understanding of their disease and less on traditional cancer therapies such as cytotoxic chemotherapies and broad-cell cycle inhibitors.

As a result of this shift in drug development, a highly concentrated overlay in product modalities and mechanisms of action has crowded the oncology pipeline across a very broad range of hematological and solid tumour indications.

The industry is asking itself how to stay innovative, how to develop and bring to market higher quality therapies to patients and how to do this faster and more efficiently.

A diversity of collaboration types

There is broad recognition that given the breadth and complexity of emerging science driving innovation in oncology, collaborations are essential in order that relevant expertise, know-how and capabilities can be combined in the right way to address patient needs.

Such collaborations take on many forms, ranging from early, multi-party alliances and consortia which are often pre-competitive in nature driving the development and shared learnings from technologies that may be enabling the field as a whole, through to more bespoke collaborations between entities.

Cell therapy research has been built on collaborations amongst scientists and entrepreneurs, providing early proof of concept for modalities thought to be too difficult to commercialise but with a strong potential for patient benefit

These may be more focused on collaborative research and development of novel products, to secure the necessary data for regulatory approvals to make such products available widely to the patients who can benefit from them.

Pre-competitive collaborations, often in basic and preclinical research, can reduce the barrier of competition and drive benefits for all stakeholders, most notably, the patient. As summarised by The National Institutes of Health, this includes reducing the number of redundant clinical trials, enhancing the statistical strength of studies, reducing overall costs and risks, and improving study participant recruitment, all while triggering creativity and innovation between collaborators.

These benefits strengthen capabilities and accelerate product development, ultimately producing higher quality and more effective therapies.

One powerful example is The National Institutes of Healths Partnership for Accelerating Cancer Therapies (PACT), which brought together 11 pharmaceutical companies to accelerate the development of new cancer immunotherapies.

Aligning with the focus of the Cancer Moonshot Research Initiative, PACT aimed to retrospectively analyse patient data from past clinical trials with the goal of predicting future patient outcomes.

This type of approach supports the ability to compare data across all trials and facilitates information sharing between partners, undoubtedly accelerating the pathway to effective therapies.

A second example is the establishment of The Parker Institute for Cancer Immunotherapy, to enable leading academic researchers and companies to come together in a pre-competitive setting, to enable rapid shared understanding and development of immunotherapeutic approaches, including the study of combination regimens.

Such combination trials, particularly those encompassing investigational products, have historically been challenging to undertake given the need for bespoke company-to-company and other 1:1 collaborative agreements. Bringing together multiple academic and industry participants under an open innovation model provides a basis to significantly accelerate the generation of scientific and clinical data that may substantially inform the field of cancer immunotherapy as a whole.

Oncology cell therapy research has been built on foundational academic collaborations amongst scientists and entrepreneurs, providing early proof of concept for modalities thought to be too difficult to commercialise but with a strong potential for patient benefit.

Examples include Kite Pharma, formed from the foundational work at the National Cancer Institute, Juno from the collaboration between the Fred Hutchinson Cancer Center and Memorial Sloan Kettering Cancer Center (all working on the first CAR T-cell candidates), or Adaptimmune working with University of Penn to first show efficacy of optimised TCR T-cells.

For collaborations that are more geared to development of novel therapies, aiming for regulatory approval and commercial availability, bespoke collaborations between biotech and pharma companies are commonplace, whereby the respective expertise and capabilities of each partner are combined in order to optimise and accelerate development, and to enable subsequent, larger scale manufacture and distribution. There are many examples of such collaborations, for which the structure can vary widely depending on the expertise of each partner, and the collaborative ways of working.

For example, under a traditional pharma/biotech collaboration and licensing model, a biotech partner may have primary responsibility for significant elements of research and early product development, and the pharma partner may lead the majority of later stage development, as well as post-approval commercial manufacture and supply. This logically aligns with organisational expertise and scale, and this type of collaboration structure has historically proven to work well. Many novel therapies have been successfully developed through such partnerships.

The rapid emergence of cell and gene therapy has required the industry to establish new and distinct capabilities, such as optimal process development and manufacture of autologous, patient specific cell therapies, whilst minimising the vein-to-vein time (the elapsed time between apheresis treatment for a patient, and reinfusing the final autologous manufactured product).

There are a growing number of biotech and pharma companies that have established or are establishing such end-to-end cell therapy capabilities, which can also play into how collaborations are structured in the field.

Case Study: From Technology Agreement to co-development and co-commercialisation partnership

In 2015, Adaptimmune and Universal Cells signed an agreement to drive the development of technologies leveraging gene-edited Induced Pluripotent Stem Cell (iPSC) lines, towards the development of allogeneic, or off-the-shelf, T-cell therapies. Universal Cells brought leading gene editing capability to make targeted gene edits to modify the characteristics of selected iPSC cell lines, and Adaptimmune the technology to differentiate iPSCs into T-cells.

Back then the science for this collaboration was early and under-developed with both parties embarking on a long-term effort and making significant at-risk investments to determine if edited, functional T-cells could be produced.

Today, Universal Cells (now an Astellas company) and Adaptimmune have established capabilities and expertise to progress novel cell therapies into clinical development, as well as with manufacturing and supply chain.

Based on this progress, in January 2020, Adaptimmune and Astellas signed a product-focused agreement to co-develop and co-commercialise up to three new stem-cell derived allogeneic T-cell therapies for people with cancer.

Given the scientific synergy between Universal Cells and Adaptimmune, and that each company is developing capabilities that may effectively address later stage product development and post-approval commercial supply, the 2020 partnership was structured as a co-development and co-commercialisation agreement. It enables the companies to work closely together, throughout the continuum of research, development and commercialisation.

Astellas and Adaptimmune will collaborate through to the end of phase 1, with Universal Cells leading gene editing activities and Adaptimmune leading iPSC to T-cell differentiation, early product characterisation and development. Beyond that, Astellas and Adaptimmune will decide whether to develop and commercialise a product candidate together under a co-development and co-commercialisation cost and profit-sharing arrangement, or for one company to take it forward alone.

This partnership is an example of how companies can harness their individual science and bring together highly complementary skills and expertise. It will enable the development of new, off-the-shelf T-cell therapies for people with cancer, which could potentially offer significant advantages such as broader access, reduced vein-to-vein time, and lower cost. The co-development and co-commercialisation nature of the agreement allows both companies to collaborate closely and on a long term basis, whilst leveraging end-to-end capabilities established by each company, maximising the velocity of product development, and ultimately delivering novel therapies to patients.

This type of agreement exemplifies how early speculative scientific collaboration can benefit all parties, most importantly the patient. It is one example from many in oncology, that underlines the value of long-term partnership within a field that is evolving rapidly across many scientific, operational and commercial frontiers.

Bringing together both teams of passionate and forward-thinking scientists may contribute to unlocking the current opportunities and challenges of off-the-shelf T-cell therapy development more effectively and efficiently for patients.

Similarly to what we are seeing as the world comes together to fight COVID-19, we as leaders in oncology owe it to patients to constantly look for ways to bring our innovative ideas as quickly as possible to the market. Working together might make that happen faster.

About the author

Helen Tayton-Martin is chief business officer at Adaptimmune.

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The new pharma collaborations driving transformative research in oncology - - pharmaphorum