Category Archives: Cell Biology

Cytovia Therapeutics and Cellectis Partner to Develop TALEN Gene-Edited iPSC-Derived Natural Killer Cells – BioSpace

CAMBRIDGE, Mass. and NEW YORK, Feb. 16, 2021 (GLOBE NEWSWIRE) -- Cytovia Therapeutics, Inc., a biopharmaceutical company developing allogeneic off-the-shelf gene-edited Natural Killer (NK) and Chimeric Antigen Receptor (CAR)-NK cells derived from induced pluripotent stem cells (iPSCs), and Cellectis (Euronext Growth: ALCLS - Nasdaq: CLLS) a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), announced today that they have entered into a strategic research and development collaboration to develop TALEN gene-edited iPSC NK and CAR-NK cells.

The financial terms of the partnership include up to $760 million of development, regulatory, and sales milestones from Cytovia to Cellectis for the first 5 TALEN gene-edited iPSC-derived NK products (partnership products). Cellectis will also receive single-digit royalty payments on the net sales of all partnered products commercialized by Cytovia. Cellectis will receive an equity stake of $15 million in Cytovia stock or an upfront cash payment of $15 million if certain conditions are not met by December 31, 2021, as well as an option to invest in future financing rounds.

We are excited to collaborate with Cellectis, a gene-editing pioneer and leader in the development of gene-edited allogeneic cancer therapies, to further accelerate Cytovias NK cell programs, said Dr. Daniel Teper, Chairman & CEO of Cytovia Therapeutics. Cellectis has a deep understanding and proven expertise in gene-edited cell therapies, and their gene editing technology, TALEN, will yield NK and CAR-NK treatments with improved potency, persistence, and safety for a variety of cancers, including solid tumors. We look forward to leveraging Cellectis insights and experience to help move Cytovias CAR-NKs into clinical trials by 2022.

Cellectis will develop custom TALEN, which Cytovia will use to edit iPSCs. Cytovia will be responsible for the differentiation and expansion of the gene-edited iPSC master cell bank into NK cells and will conduct the pre-clinical evaluation, clinical development, and commercialization of the mutually-agreed-upon selected therapeutic candidates. Cellectis is granting Cytovia a worldwide license to its TALEN gene-editing technology, enabling Cytovia to modify NK cells addressing multiple gene targets for therapeutic use in several cancer indications.

We are thrilled to partner with Cytovia, a pioneer in the development of NK cells derived from iPSCs, said Dr. Andr Choulika, CEO of Cellectis. We are looking forward to this collaboration and the opportunity to further expand the potency of our proprietary TALEN gene-editing technology to iPSCs and CAR-NKs. Down the road, this collaboration should allow for NK cell therapies to be made available to cancer patients, which is very much in line with Cellectis mission to provide life-saving product candidates to address unmet patient needs in this field.

About CellectisCellectis is developing the first of its kind allogeneic approach for CAR-T immunotherapies in oncology, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients. As a clinical-stage biopharmaceutical company with over 20 years of expertise in gene editing, Cellectis is developing life-changing product candidates utilizing TALEN, its gene editing technology, and PulseAgile, its pioneering electroporation system to harness the power of the immune system in order to target and eradicate cancer cells.

As part of its commitment to a cure, Cellectis remains dedicated to its goal of providing lifesaving UCART product candidates to address unmet needs for multiple cancers including acute myeloid leukemia (AML), B-cell acute lymphoblastic leukemia (B-ALL) and multiple myeloma (MM).

Cellectis headquarters are in Paris, France, with additional locations in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). For more information, visit http://www.cellectis.com.

Follow Cellectis on social media: @cellectis, LinkedIn and YouTube.

TALEN is a registered trademark owned by Cellectis.

About Cytovia TherapeuticsCytovia Therapeutics Inc. is a biotechnology company that aims to accelerate patient access to transformational immunotherapies, addressing several of the most challenging unmet medical needs in cancer. Cytovia focuses on Natural Killer (NK) cell biology and is leveraging multiple advanced patented technologies, including an induced pluripotent stem cell (iPSC) platform for CAR (Chimeric Antigen Receptors) NK cell therapy, next-generation precision gene-editing to enhance targeting of NK cells, and NK engager multi-functional antibodies. Our initial product portfolio focuses on both hematological malignancies such as multiple myeloma and solid tumors including hepatocellular carcinoma and glioblastoma. The company is establishing R&D and GMP manufacturing operations in the greater Boston area and partners with the University of California San Francisco (UCSF), the New York Stem Cell Foundation (NYSCF), the Hebrew University of Jerusalem, INSERM, and CytoImmune Therapeutics.Learn more at http://www.cytoviatx.com and follow Cytovia Therapeutics on Social Media (Facebook, LinkedIn, Twitter, and Youtube).

About Gene-Edited, iPSC-derived NK CellsChimeric Antigen Receptors (CAR) are fusion proteins that combine an extracellular antigen recognition domain with an intracellular co-stimulatory signaling domain. Natural Killer (NK) cells are modified genetically to allow insertion of a CAR. CAR-NK cell therapy has demonstrated initial clinical relevance without the limitations of CAR-T, such as Cytokine Release Syndrome, neurotoxicity or Graft vs Host Disease (GVHD). In addition, CAR-NKs are naturally allogeneic, available off-the-shelf and may be able to be administered on an outpatient basis. Recent innovative developments with the induced pluripotent stem cell (iPSC)-derived CAR-NKs, an innovative technology, allow large quantities of true off-the-shelf, homogeneous genetically modified CAR NK cells to be produced from a gene-edited iPSC master cell bank, and thus hold promise to expand access to cell therapy for many patients.

For further information, please contact:

Cellectis Media contacts:Margaret Gandolfo, Communications Manager, 646-628-0300, margaret.gandolfo@cellectis.comConor McGoldrick, Zeno Group, Assistant Account Executive, 914-355-0927, Conor.Mcgoldrick@zenogroup.com

Cellectis IR contact:Simon Harnest, SVP, Corporate Strategy and Finance, 646-385-9008, simon.harnest@cellectis.com

Cytovia Investor Relations contact: Anna Baran-DjokovicVP of Investor Relations646-355-1787anna@cytoviatx.com

Cytovia Media contact: Chris MaggosLifeSci Advisors+41 79 367 6254chris@lifesciadvisors.com

Disclaimer

This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by words such as at this time, believe, expected, forward looking, promising and will, or the negative of these and similar expressions. These forward-looking statements, are based on our managements current expectations and assumptions and on information currently available to management. These forward-looking statements are made in light of information currently available to us and are subject to numerous risks and uncertainties, including with respect to the duration and severity of the COVID-19 pandemic and governmental and regulatory measures implemented in response to the evolving situation. Furthermore, many other important factors, including those described in our Annual Report on Form 20-F and the financial report (including the management report) for the year ended December 31, 2019 and subsequent filings Cellectis makes with the Securities Exchange Commission from time to time, as well as other known and unknown risks and uncertainties may adversely affect such forward-looking statements and cause our actual results, performance or achievements to be materially different from those expressed or implied by the forward-looking statements. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.

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Cytovia Therapeutics and Cellectis Partner to Develop TALEN Gene-Edited iPSC-Derived Natural Killer Cells - BioSpace

Research Associate in Stem Cells and Regenerative Medicine job with KINGS COLLEGE LONDON | 246711 – Times Higher Education (THE)

Job descriptionThe Centre for Stem Cells & Regenerative Medicine is located in Guys Hospital.It is internationally recognized for research on adult and pluripotent stem cells and is a focus for cutting-edge stem cell research currently taking place across the College and its partner NHS trusts, as part of Kings Health Partners. Through the Centre, Kings aims to drive collaboration between scientists and clinicians to translate the potential of stem cells into clinical reality for patients.Applications are invited for a postdoctoral researcher funded as part of the PIs Wellcome Clinical Fellowship, and will work with a dynamic group of scientists focussed on reproductive biology, early embryonic development and the causes of infertility. The post holder will contribute to the regenerative medicine theme and will be involved in the generation and processing of single cell experiments using a variety of techniques.This is an exciting opportunity following our recent work (Sangrithi et al. 2017, Dev Cell & Lau et al. 2020, Dev Cell). The project aims to discover the function of genes on the X-chromosome in male germline stem cells (spermatogonia) and their role in idiopathic and sex chromosome aneuploidy associated infertility. We aim to understand physiological gene regulatory networks functional in spermatogonial stem cells using a combination of single-cell methods, to explain how perturbation in X-gene dosage in SSCs may cause infertility. The postholder will also identify and validate candidate disease bio-markers.This post will be offered on an a fixed-term contract until 05/04/2026This is a full-time post - 100% full time equivalent

Key responsibilities Carry out world class research. Are adept at working in a wet lab setting with experience in designing and executing experiments. Familiarity in single cell work nucleic acid manipulation is desirable Communicate results effectively in writing and orally Contribute to publications arising from the research projects Keep clear and up-to-date records of work Attend and present at seminars, journal clubs and conferences Contribute to collaborative atmosphere of the department Share skills by training others Comply with all relevant safety legislation to ensure a safe working environment Take part in public engagement activities To support grant writing, for maintaining the continual research in this domain, e.g. Fellowships Post holder will be expected to plan and prioritise their own workload, with competing and shifting priorities under pressure of deadlinesThe above list of responsibilities may not be exhaustive, and the post holder will be required to undertake such tasks and responsibilities as may reasonably be expected within the scope and grading of the post.

Skills, knowledge, and experience

Essential criteria PhD awarded in the biological sciences Excellent general knowledge of molecular biology Knowledge of cell biology Knowledge of flow cytometry Relevant postdoctoral experience Experience in a molecular biology research lab Excellent record keeping / attention to detail Organized and systematic approach to research Pro-active, enthusiastic, positive attitude Self-motivated, with the ability to work under pressure & to meet deadlines Keen interest in infertility and regenerative medicine Ability to think strategically

Desirable criteria Understanding of the biology of germ cells and embryo development Previous experience in working with the laboratory mouse ES cell culture experience General knowledge of computational tools for single cell RNAseq Ability to make collaborative and independent decisions*Please note that this is a PhD level role but candidates who have submitted their thesis and are awaiting award of their PhDs will be considered. In these circumstances the appointment will be made at Grade 5, spine point 30 with the title of Research Assistant. Upon confirmation of the award of the PhD, the job title will become Research Associate and the salary will increase to Grade 6.Further informationABOUT THE SCHOOLThe School of Basic & Medical Biosciences is led by Professor Mathias Gautel and comprises five departments with a wide range of expertise and interests. Using a bench to bedside approach, the School aims to answer fundamental questions about biology in health and disease and apply this to the development of new and innovative clinical practise, alongside providing a rigorous academic programme for students.DepartmentsThe Centre for Human & Applied Physiological Sciences (CHAPS) uses an integrative and translational research approach focusing on fundamental questions about human physiological function in health and disease to explore 3 research themes: skeletal muscle & aging, sensory-motor control & pain and aerospace & extreme environment adaptation.The Centre for Stem Cells & Regenerative Medicine focuses on cutting-edge stem cell research, how stem cells interact with their local environment and how these interactions are important for developing effective cell therapies in the clinic.The Department of Medical & Molecular Genetics uses cutting-edge technologies and analysis techniques to explore the mechanistic basis of disease, improve diagnostics and understand the epigenetic mechanisms of gene regulation and RNA processing, working from whole population level to complex and rare disease genomesThe Randall Centre of Cell & Molecular Biophysics takes a multi-disciplinary approach at the interface of Biological and Physical Sciences to explore the underlying mechanisms behind common diseases.St Johns Institute of Dermatology seeks to improve the diagnosis and management of severe skin diseases, through a better understanding of the basic pathogenetic mechanisms that cause and sustain these conditions focussing on cutaneous oncology, genetic skin disorders, inflammatory & autoimmune skin disorders, and photomedicine.About the Department of Centre for Stem Cells & Regenerative MedicineThe Centre for Stem Cells & Regenerative Medicine is led by Professor Fiona Watt, whos laboratory comprises approximately 30 research staff and visiting scientists and is internationally recognised for research on adult and pluripotent stem cells. Along with Professor Watts group there are nine other research groups operating at the Centre, bringing the total number of staff to approximately 80 people.Research at the Centre is focused on how stem cells interact with their local environment, or niche. We believe that an understanding of these interactions is important for developing effective cell therapies in the clinic. Located on the Guys Hospital campus, the Centre acts as a focus for cutting-edge stem cell research taking place across the College and its partner NHS Trusts, as part of Kings Health Partners. To facilitate collaborations within Kings and with external partners, we have opened a Stem Cell Hotel where researchers can access specialist equipment and technical support to study stem cell behaviour at single cell resolution. We also host an international seminar series and run the Stem Cells @ Lunch seminar series to share ideas and unpublished data. Our researchers are committed to public engagement and take part in diverse outreach events.Detailed information about the Centre for Stem Cells & Regenerative medicine can be found in the link below:http://www.kcl.ac.uk/lsm/research/divisions/gmm/departments/stemcells/index.aspx

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Research Associate in Stem Cells and Regenerative Medicine job with KINGS COLLEGE LONDON | 246711 - Times Higher Education (THE)

Study seeks to identify biological markers that predict mesothelioma response to treatment – Baylor College of Medicine News

The National Cancer Institute (NCI) has granted a $2.5 million, five-year R37 MERIT Award to Dr. Bryan Burt, associate professor of surgery and chief of the Division of General Thoracic Surgery, for his research project titled, Proteomic Determinants of Response to Checkpoint Blockade in Malignant Pleural Mesothelioma.

Malignant pleural mesothelioma (MPM) is a fatal cancer of the lining of the lungs that has defeated standard therapies for decades. In recent years, emerging clinical data has shown that treatment with a form of immunotherapy called immune checkpoint inhibitors (ICIs) results in meaningful extension of life in half of patients with MPM, but is associated with immune-related side effects, Burt said.

The goal of this study is to develop a clinically relevant test that would enable physicians to determine whether a patient would be most likely or less likely to respond to ICIs before the patient gets treatment, saving those less likely to respond from immune-related adverse events.

We want to be able to predict not only who is going to respond, but also the strength of the response.

In other words, whether the tumor will completely or partially shrink or just remain stable for long periods of time, which is important too, Burt said. We hope to design a test that would allow us to predict those possible outcomes.

To develop the test, Burt is taking a closer look inside MPM tissues.

Preliminary data collected retrospectively showed that the tumors of patients who respond to ICIs tend to have a certain immune cell composition, which is quite complex, Burt said.

We developed a technique to analyze the presence of about 30 different cell types in a very small bit of a tumor sample.

Burt also is looking at the architecture of the tissue samples. In addition to determining how many cells there are of each type, we also study tissue architecture to see how these cells are organized in the tumor. Are they close to blood vessels? Are they close to each other? Our preliminary data showed that tissue immune cell architecture in the tumor also predicts response to treatment, Burt said.

The study also seeks to better understand the biological mechanism supporting the responders.

Our preliminary data suggested that MPMs with high levels of neoantigens, new tumor surface molecules that can warn the immune system of the presence of the tumor, is not the only requirement for responding to ICIs, Burt said. Its also important to take into consideration molecules called MHC, which present neoantigens to the immune system and facilitate the stimulation of the soldier immune cells. We have found that when both neoantigens and certain MHC molecules are there, the patient responds well to the therapy.

Burt hopes that the analyses of tumor neoantigens and HLA molecules can be developed into a test to predict response to ICIs, but also that it will help understand neoantigen biology that could be applicable to other tumors as well.

We anticipate that after this study supported by the R37 MERIT Award we will be able to use both cellular organization and neoantigen:MHC concordance to predict response to ICIs, Burt said.

It will then be time for a clinical trial to conduct a rigorous prospective evaluation in which treatment depends on the results of the test.

Burts research submission to the NCI as an R01 application received a score within the NCI pay line for experienced investigators and was thus converted to an R37 MERIT Award. This award enables National Institutes of Health institutes, such as the NCI, to give investigators with stellar records of research accomplishment a five-year award with the possibility of extension for additional years without undergoing another Integrated Review Groups (IRG) peer review. Burts five-year award has an opportunity for an extension of up to two additional years. The plan is to conduct the study between 2021 and 2028.

By Ana Mara Rodrguez, Ph.D.

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Study seeks to identify biological markers that predict mesothelioma response to treatment - Baylor College of Medicine News

Nuclear TEAD4 with SIX1 Overexpression is an Independent Prognostic Ma | CMAR – Dove Medical Press

Tong Yu,1,2,* Jinglue Song,1,2,* Hui Zhou,1,2 Tingyu Wu,1,2 Zhonglin Liang,1,2 Peng Du,1,2 Chen-Ying Liu,1,2 Guanghui Wang,3 Long Cui,1,2 Yun Liu1,2

1Department of Colorectal Surgery, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, Peoples Republic of China; 2Shanghai Colorectal Cancer Research Center, Shanghai, Peoples Republic of China; 3Guizhou Provincial Peoples Hospital, Guizhou, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Yun Liu Tel + 86-021-25078825Email liuyun@xinhuamed.com.cnGuanghui Wang Email wangguanghui0625@163.com

Introduction: Stage IIII colorectal cancer patients are under risk of tumor recurrence and metachronous metastasis after radical surgery. An increased expression of transcription factor TEAD4 is associated with epithelial-mesenchymal transition, metastasis and poor prognosis in colorectal cancer. However, the mechanistic role of TEAD4 in driving colon cancer progression and its prognostic value in early stage of CRC remains unclear.Methods: In this study, the regulation, function and prognostic significance of TEAD4 and its new direct target gene SIX1 in CRC progression were evaluated using human tissues, molecular and cell biology.Results: We show that TEAD4 directly upregulates the expression of SIX1 at transcriptional level in CRC cells, establishing that SIX1 is a new direct target gene of TEAD4. TEAD4 promotes EMT and cell migration of CRC cells, while SIX1 knockdown attenuates this effect and SIX1 overexpression enhances this effect, indicating that SIX1 mediates the function of TEAD4 in promoting cell migration in CRC cells. Clinically, nuclear TEAD4, overexpression of SIX1 and nuclear TEAD4 with SIX1 overexpression predict poor prognosis in CRC patients.Discussion: Our study identifies TEAD4-SIX1-CDH1 form a novel signaling axis, which contributes to CRC progression, and its aberrant expression and activation predicts poor prognostic for CRC patients in stage IIII.

Keywords: colorectal cancer, TEAD4, SIX1, hippo pathway

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

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Cell Biology Cloud Computing Market 2026 | Future Growth and Opportunities with Dazzling Key Players Accenture, Amazon Web Services, Benchling, Cisco…

The Cell Biology Cloud Computing Market study added by ResearchMoz, exhibits a comprehensive analysis of the growth trends present in the global business scenario. The study further presents conclusive data referring to the commercialization aspects, industry size and profit estimation of the market.

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Accenture, Amazon Web Services, Benchling, Cisco Systems, Dell Emc, IBM, DXC Technology, Oracle, ScaleMatrix, IPERION, NovelBio

Cell Biology Cloud Computing Breakdown Data by TypePublic Cloud ComputingPrivate Cloud ComputingHybrid Cloud ComputingCell Biology Cloud Computing Breakdown Data by ApplicationGenomicsDiagnosticsClinical TrialsPharma ManufacturingOthers

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The Cell Biology Cloud Computing Market report focuses on the requirements of the clients from several global Market regions such as North America, Latin America, Asia-Pacific, Europe, and India.

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Table of Content:

Cell Biology Cloud Computing Market Research Report 2020-2026

Chapter 1: Industry Overview

Chapter 2: Cell Biology Cloud Computing Market International and Market Analysis

Chapter 3: Environment Analysis of Cell Biology Cloud Computing

Chapter 4: Analysis of Revenue by Classifications

Chapter 5: Analysis of Market Revenue Status

Chapter 6: Analysis of Revenue by Regions and Applications

Chapter 7: Analysis of Cell Biology Cloud Computing Market Key Manufacturers

Chapter 8: Sales Price and Gross Margin Analysis

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2020 Outlook on the Global Singlecell Technology Industry – Market Size was Over $800 Million in 2019 – ResearchAndMarkets.com – Business Wire

DUBLIN--(BUSINESS WIRE)--The "Singlecell Technology Market Landscape 2020" report has been added to ResearchAndMarkets.com's offering.

The global single cell technology (SCT) market has emerged since 2014. There are over 43 companies with various 49 SCT products commercialized worldwide, and over 15 pre-commerce-stage startups are lined up and keep increasing. The market size is over $800 M in 2019, at a CAGR of ~24%.

The market is expected to grow significantly by the growing awareness of the single cell projects among biology communities, the growth of precision medicine and diagnostic industry, the antibody therapeutic drug development, the entry of new players, etc.

However, the high cost of the equipment, the requirement of in-person demo, various remaining technical challenges & defaults of the products that rushed into the market too early, and lack of interdisciplinary expertise on the customer sides could limit the market growth rates. Acquisitions and partnerships are actively seen as for most of the competitive technology industry.

The majority (~60%) of the market players are small-sized startup companies with under 100 employees, however, the market share is highly concentrated on four major companies (BD, 10x genomics, Fluidigm, and Berkeley light), yet this market share can change as the market is expanding and more new players are entering, more differentiation among players is expected.

Primarily, the SCT market has a complex market structure characterized by its wide range of technology mixtures and various types of users across various life science research fields. The general functionalities of the SCT are: single cell (SC) isolation, SC-sequencing, SC-protein analysis, or SC-focused manipulation. Among those, the first two category products (SC-isolation and SC-sequencing) account for ~72% of the market.

SCT can be applied in most of the life science application as it is defined by any biological science experimentation that handles and generate single cell resolution data. Stem cell research and cancer research are the fields of studies that have adapted SCT the most, and immunology and neurobiology also have a growing need in SCT. The market is categorized by the core technologies (11 categories) and by the applications (5 categories).

The core technology categories include conventional cell picking, limited dilution, FACS, and laser capture microdissection (LCM) technologies, but also novel technologies for automatic single cell picking, automatic single cell dispensing, microwell-based single cell isolation/analysis, microfluidic chip based-single cell analysis, droplet microfluidics-based, novel cytometry, and cell manipulation. The five market application categories include SC-isolation, SC-DNA/RNA sequencing, cell line development, protein or functional analysis, and drug discovery & diagnostics.

Companies Mentioned

Key Topics Covered:

Preface

Executive Summary

Part One: the Market and Strategy Perspectives

Chapter 1 Market Structure and Market Size

1.1 Market Structure

1.2 Market Size

1.3 Market's Dynamic Growth & Restraint Factors

Chapter 2 Projecting Strategic Plans

2.1 Strategic Moves by Current SCT Companies

2.2 Market Innovating Strategy

Part Two: Biology and Its Evolution- Market from the Customers Perspective

Chapter 3 Single Cell Biology by -Omics and Field of Study

3.1 Single Cell Biology: Physics and Engineering Meet Biology

3.2 Single Cell Biology by -Omics Fields

3.3 Single Cell Biology by Field of Studies

Chapter 4 Understanding the Customer and Their Workflow

4.1 Understand the Customer's Workflow

4.2 Understand the Customer Experience Cycle

Part Three: Market from the Technology Providers Perspective

Chapter 5 Market Segments by Technology

5.1 Overview

5.2 Manual Picking

5.3 Limited Dilution

5.4 FACS: Fluorescence Activated Cell Sorter

5.5 Laser Capture Microdissection (LCM)

5.6 Automatic Single Cell Pickers

5.7 Single Cell Dispensing

5.8 Microwell Based Single Cell Isolation

5.9 Microfluidics Chip-Based Single Cell Analysis

5.10 Droplet Microfluidics

5.11 New Type of Cytometry and Spectroscopy

5.12 Fluidics and Cell Manipulation

Chapter 6 Market Segments by Applications

6.1 Overview

6.2 SC-Dna and Rna Sequencing

6.3 Cell Line Development

6.4 Protein or Functional Analysis

6.5 Drug Discovery and Diagnostics

Chapter 7 Emerging Early Stage SCT Startup Companies

7.1 Emerging Early Stage SCT Startup Companies

or more information about this report visit https://www.researchandmarkets.com/r/6q7yf9

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2020 Outlook on the Global Singlecell Technology Industry - Market Size was Over $800 Million in 2019 - ResearchAndMarkets.com - Business Wire

Taysha Gene Therapies Announces Formation of Independent Scientific Advisory Board – Business Wire

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the CNS in both rare and large patient populations, today announced the formation of an independent Scientific Advisory Board (SAB) that will work closely with senior management to advance the companys clinical development and commercialization efforts.

We are excited and privileged to have the opportunity to work with this cross-functional group of esteemed scientific and clinical thought leaders on initiatives from discovery, through pre-clinical and clinical development and commercialization, said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. They bring a wealth of knowledge in the development of gene therapy products and diseases of the CNS that will be invaluable as we advance our extensive pipeline of AAV-based gene therapies for the treatment of monogenic diseases of the CNS. Formalizing the SAB is an important accomplishment that will help position Taysha for sustained success as we further our R&D initiatives.

The SAB brings together the expertise of esteemed independent scientists and clinicians covering Tayshas key areas of research in monogenic diseases and gene therapy products. Members of the SAB will provide scientific review and guidance to the company around its R&D and related business activities.

Members of Tayshas SAB include:

Deborah Bilder, M.D., is an Associate Professor at the University of Utah in Educational Psychology, General Pediatrics, and Child Psychiatry. Her research interests include clinical trials, medications, and biologics that target rare genetic conditions and has authored over 45 peer-reviewed articles. She is the Principal Investigator for the Utah Registry of Autism and Developmental Disabilities and Co-Principal Investigator for the Utah site of the Centers for Disease Control and Preventions Autism and Developmental Disabilities Monitoring Network. Dr. Bilder is Co-Chair of the DAC Committee in psychiatry at the University of Utah and a consultant for the Utah Regional Education in Neurodevelopmental and Related Disabilities program. She has been awarded the Triple Board Program Teaching Award from the University of Utah Division of Child and Adolescent Psychiatry. She is a steering committee member for BioMarin Pharmaceutical Phase 3 Clinical Trial and also serves as a medical advisor for the Utah chapter of Make-a-Wish Foundation. Dr. Bilder earned her medical degree from Vanderbilt University.

Alan Boyd, B.Sc., M.B., Ch.B., FRSB, FFLM, FRCP, FFPM, is the CEO and Founder of Boyd Consultants and a fellow and Immediate Past-President of the Faculty of Pharmaceutical Medicine, Royal Colleges of Physicians, UK. Professor Boyd is also a Council Member and the Independent Clinician Trustee on the Board of the Academy of Medical Royal Colleges, UK. He is also an honorary professor at the University of Birmingham Medical School, in recognition of his expertise in medicine development. He has significant pharmaceutical industry experience and was the Head of Medical Research at AstraZeneca and the Research and Development Director at Ark Therapeutics Ltd, specializing in the development of gene therapy products. He is a graduate in biochemistry and medicine from the University of Birmingham, UK.

Wendy K. Chung, M.D., Ph.D., is a Kennedy Family Professor of Pediatrics in Medicine, Attending Physician in the Division of Molecular Genetics, Department of Pediatrics and Medicine, and the Director of Clinical Genetics, Clinical Cancer Genetics, and Precision Medicine Resource at the Irving Institute for Translational Research, all at Columbia University. Her research interests include spinal muscular atrophy, autism, and neurogenetics. Dr Chung has authored over 500 peer-reviewed articles and 75 textbook chapters and serves on the Editorial Board of Molecular Case Studies and The American Journal of Human Genetics. Dr Chung is the Director of Clinical Research at the Simons Foundation Autism Research Initiative (SFARI) and a member of the National Academy of Medicine. Dr. Chung earned her medical degree from Cornell University Medical College and her doctorate from Rockefeller University.

David P. Dimmock, M.D., is the Senior Medical Director of Rady Childrens Institute for Genomic Medicine. Dr. Dimmock is an expert in the field of clinical genomic medicine, the Principal Investigator on multiple clinical trials of novel therapeutics in rare metabolic diseases and an author of over 100 peer-reviewed articles, publications, chapters, books and reviews. He has been an invited advisor to the U.S. Food and Drug Administration in the Office of Orphan Diseases and has overseen regulatory submissions for whole genome sequencing devices. At the Center for Disease Control, he was a member of the Planning and Organizing Committee of NeXT-StoC to develop guidance to ensure analytic quality of next-generation sequencing tests. In addition, he was a member of the National Genomics Board UK and CLIAC NGS Guidelines Forum. He is a Scientific Advisory Board member for BioMarin Pharmaceuticals. Dr. Dimmock is a graduate from St. Georges, University of London.

Michael W. Lawlor, M.D., Ph.D., is a Professor of Pathology, Biomedical Engineering, Physiology, Cell Biology, Neurobiology, and Anatomy and the Associate Director of the Neuroscience Research Center at the Medical College of Wisconsin. He is a Board-Certified Anatomic Pathologist and Neuropathologist, and his research interests include pediatric muscle disease and gene therapy. Dr. Lawlor is an Editorial Board member of Muscle and Nerve and Journal of Neuropathology and Experimental Neurology. He is currently serving as an SAB member for Solid Biosciences in support of its gene therapy programs. Dr. Lawlor earned his medical degree and doctorate from Loyola University School of Medicine and his residency, fellowship, and postdoctoral training was completed at Massachusetts General Hospital and Boston Childrens Hospital in association with Harvard Medical School.

Gerald S. Lipshutz, M.D., M.S., is a Professor-in-Residence in the Departments of Surgery and Molecular and Medical Pharmacology, Surgical Director of the Pancreas/Auto-islet Transplant Program and Chairman of the Academic Medicine College at the David Geffen School of Medicine at University of California, Los Angeles. His clinical specialties and interests include liver and pancreas transplantation and gene and cell therapies for single-gene metabolic disorders of the liver. Dr. Lipshutz is a grant reviewer for the Wellcome Trust and the US National Institutes of Health where he is a standing member of the Gene and Drug Delivery (GDD) study section. He is a Principal Investigator at the UCLA Lipschutz Hepatic Regenerative Medical Laboratory and for several NIH-funded and industry-sponsored studies for gene therapies. He is author of over 70 peer-reviewed articles and is an Editorial Board member of Molecular Therapy - Methods and Clinical Development and Gene Therapy. Dr. Lipshutz earned his medical degree from the University of California, Los Angeles.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

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Taysha Gene Therapies Announces Formation of Independent Scientific Advisory Board - Business Wire

Anticancer drug may reduce mortality and speed up recovery for severe COVID-19 patients – News-Medical.Net

Treating severe COVID-19 patients with the anticancer drug bevacizumab may reduce mortality and speed up recovery, according to a small clinical study in Italy and China that was led by researchers at Karolinska Institutet in Sweden between February and April 2020. On average, blood oxygen levels, body temperature and inflammatory markers significantly improved in patients treated with a single dose of bevacizumab in addition to standard care. The research is published in Nature Communications.

To reduce COVID-19 mortality, we aim to develop an effective therapeutic paradigm for treating patients with severe COVID-19. Our findings suggest that bevacizumab plus standard care is highly beneficial for patients with severe COVID-19 and should be considered as a potential first-line therapeutic regimen for this group."

Yihai Cao, Corresponding Author, Professor of Vascular Biology, Department of Microbiology, Tumor and Cell Biology, Karolinska Institutet

Bevacizumab is a medication that has been used to treat various types of cancer since 2004. It works by slowing the formation of new blood vessels by inhibiting a growth factor known as VEGF. Many patients with severe COVID-19 have elevated levels of VEGF as well as symptoms associated with this marker, including excess fluid and disorganized blood vessels in the lungs. Against this background, the researchers designed a clinical trial to investigate the effect of combining bevacizumab with standard care for treating patients with severe COVID-19.

Twenty-six patients were recruited from two hospitals in China and Italy between mid-February and early April in 2020. The patients had confirmed COVID-19 and symptoms such as difficulty breathing, low blood oxygen levels and pneumonia. They were retrospectively matched with 26 patients of similar characteristics who received standard care at the same hospitals in roughly the same time period and thus served as the control group.

The recruits received standard care plus a single low dose of about 7.5 mg/kg bevacizumab, which markedly improved blood oxygen levels within 24 hours compared to the control group. By the end of the 28-day follow-up period, 92 percent of the bevacizumab-treated patients no longer needed the same level of oxygen support as before the trial began, compared with an improvement rate of 62 percent for the controls.

None of the bevacizumab-treated patients died and 17 (65 percent) improved so much that they were able to leave the hospital within the follow-up period. In the control group, three died and only 46 percent were discharged within 28 days. Bevacizumab also shortened the duration of oxygen-support to a median of nine days compared with 20 for the standard care group.

Other interesting findings include reduction in fever, an increase in white blood cells and a sharp decrease of c-reactive protein (CRP) levels, an inflammatory marker. No severe safety concerns were detected.

"Many patients with severe COVID-19 require significant oxygen support during long hospital stays, which pose global challenges to medical supplies," Yihai Cao says. "Our study shows that bevacizumab could help reduce the need for oxygen support and reduce days in hospital, thus improving the outcome for the individual patient while easing pressure on medical resources."

The limitations of the study include the non-randomized nature of the trial, the short-term follow-up and the small size of the cohort.

The next step will be to design randomized and placebo-controlled trials by recruiting a large number of patients, allowing further assessment of the potential benefits of bevacizumab both in and of itself and in combination with other therapies such as antivirals and anti-inflammatory drugs.

Source:

Journal reference:

Pang, J., et al. (2021) Efficacy and tolerability of bevacizumab in patients with severe Covid-19. Nature Communications. doi.org/10.1038/s41467-021-21085-8.

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Anticancer drug may reduce mortality and speed up recovery for severe COVID-19 patients - News-Medical.Net

USC scientist Ya-Wen Chen receives American Lung Association grant to advance stem cell-based lung therapies – India Education Diary

USC Stem Cell scientist Ya-Wen Chen hopes to pioneer a new approach to regenerating damaged lung tissue, with support from a Catalyst Grant from the American Lung Association (ALA). The award provides $50,000 year for up to two years.

For many patients with chronic lung diseases, the only available treatment is transplantationa difficult, dangerous surgery that involves challenges ranging from the severe shortage of donor organs to immune rejection, said Chen, who is an assistant professor of medicine, and stem cell biology and regenerative medicine at USC. Even patients who are lucky enough to receive donor organs only have a 10 to 20 percent survival rate at 10 years. If we can encourage these patients own cells to repair damage and heal their lungs, we could dramatically improve this prognosis.

With this goal in mind, Chen is using human stem cells to generate rudimentary lung-like structures known as lung bud organoids. Within these organoids, Chens group will probe how a specific population of cells repairs the tiny gas-exchange interfaces called alveoli in damaged lungs.

Specifically, Chen is interested in a population of cells known as distal small airway epithelial progenitors or SAEPs, which could have the potential to improve lung function in patients with idiopathic pulmonary fibrosis (IPF) or chronic obstructive pulmonary disease (COPD). A group of lung diseases that includes chronic bronchitis and emphysema, COPD affects at least 16 million Americans and is the third leading cause of death in the U.S. An additional 200,000 Americans are living with IPF, a progressive, incurable and often deadly disease that, for unknown reasons, causes scar tissue to form in the lungs, impeding breathing.

Our ultimate goal is to leverage patients existing stem and progenitor cells to promote healing through a non-surgical, regenerative approach, said Chen, a member of the USC Hastings Center for Pulmonary Research, as well as USCs stem cell research center.

Chen is one of 98 scientists to receive research support from the ALA, which has committed $11.55 million total to support scientific investigations aimed at reducing the burden of lung disease.

Despite the fact that the pandemic poses significant economic challenges, said ALA President and CEO Harold Wimmer, the American Lung Association is prioritizing research and significantly increasing award funding to help improve the lung health of all Americans.

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USC scientist Ya-Wen Chen receives American Lung Association grant to advance stem cell-based lung therapies - India Education Diary

Gene Linked Between Alzheimer’s Disease and COVID-19 – Contagionlive.com

A study conducted by City of Hope, an independent biomedical research and treatment center, has discovered the gene ApoE4, which increases the risk for Alzheimers disease, can also increase the susceptibility to and severity of an infection with the coronavirus disease 2019 (COVID-19). Results from the research were published in the journal Cell Stem Cell.

The study was initially started due to an interest in assessing how a COVID-19 infection impacts the brain. Because the disease presents symptoms like a loss of smell and taste, it was believed that the virus had underlying neurological effects.

The investigators employed pluripotent stem cells (iPSCs), a type that easily becomes any kind of cell, to create neurons and helper cells called astrocytes. They infected both cells with the SARS-CoV-2 virus and found that they were susceptible to the disease. They then created 3D brain tissue models called organoids, one with and one without the astrocytes, also infecting them with COVID-19 and discovered that the astrocytes actually amplified the infection.

Additionally, the team used reprogrammed iPSCs to generate neurons from the cells of an Alzheimers patient containing ApoE4. Modifying the iPSCs with a gene editing tool so that they contained ApoE3, a neutral gene type, they created more astrocytes and neurons.

Findings showed that in comparison to the ApoE3 cells, the ApoE4 cells showed a significantly higher susceptibility to COVID-19 and had more damage inflicted on their neurons and astrocytes.

"Our study provides a causal link between the Alzheimer's disease risk factor ApoE4 and COVID-19 and explains why some (e.g., ApoE4 carriers) but not all COVID-19 patients exhibit neurological manifestations," Yanhong Shi, director of the Division of Stem Cell Biology at City of Hope and co-corresponding author said. "Understanding how risk factors for neurodegenerative diseases impact COVID-19 susceptibility and severity will help us to better cope with COVID-19 and its potential long-term effects in different patient populations."

The next step in the process will be to continue studying the impact that COVID-19 has on the brain to further understand the potential long-term neurological impacts like severe headaches experienced by some months after the initial infection.

"COVID-19 is a complex disease, and we are beginning to understand the risk factors involved in the manifestation of the severe form of the disease" said Vaithilingaraja Arumugaswami, a co-corresponding author said. "Our cell-based study provides a possible explanation as to why individuals with Alzheimer's' disease are at increased risk of developing more severe COVID-19 symptoms."

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Gene Linked Between Alzheimer's Disease and COVID-19 - Contagionlive.com