How to back up your Android phone and keep all of your important information safe – CNET

Keep a current back up of your Android phone.

Keeping a current backup of your Android phone and all of the data it holds is an important task that's part of owning a phone. Let's be honest, phones break, get misplaced or -- even worse -- get stolen. And when that happens, the financial burden of replacing a phone is stressful enough; don't add to the stress by not having a back up of your calendar or photos.

Here's the thing about backing up an Android phone: It can be confusing. The backup process and even the service used is slightly different if you own a Pixel 4 or a Galaxy S10.

It's a good idea to sit down with your phone in hand and take a few minutes to make sure it's being regularly backed up.

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Built in to Android is a backup service, similar to Apple's iCloud, that automatically backs up things like your device settings, Wi-Fi networks and app data to Google Drive. The service is free and doesn't count against storage in your Google Drive account.

Google's backup service is built in to every Android phone, but some device makers like Samsung provide their own solutions as well. If you own a Galaxy phone, you can use one or both services -- it doesn't hurt to have a backup of a backup.

Google's backup service is free and should be turned on automatically.

Google's backup service should be turned on by default after you set up your Android device, but you should double-check that's indeed the case. If you have trouble finding the backup settings by following the steps outlined below, use the search bar in the Settings app to find "backup."

To view your backup settings, open theSettingsapp on your Android device and tap onSystem>Backup. There should be a switch labeled "Back up to Google Drive." If it's turned off, turn it on. You can select which Google account you want to use to store your backups by tapping on theAccountsoption if you are signed into more than one Google account on your phone.

With backup turned on, your phone will automatically back upthe following information:

You'll see a list of data categories under theActive Backupssection, along with the last time that information was backed up. Tap a section, such as App Data, to view more information or fine-tune which apps are backed up. For example, if you tap Photos & Videos, the backup settings page for Google Photos app is automatically opened.

If you're switching from one Pixel phone to another, your home screen layout is backed up and restored. However, I've had mixed luck when restoring a back up from a Pixel to, say, a Motorola phone. Your mileage will undoubtedly vary.

At any time, you can visit this section and tapBack Up Nowto manually start a backup. Make sure your phone is connected to a strong Wi-Fi network, and plug it into a charger to speed up the process. If you're upgrading phones, you should manually start a backup before setting up your new phone.

Google Photos provides free unlimited photo and video backup, as long as you're all right with Google changing them to "high-quality." That just means that Google caps the photo size to 16 megapixels and videos at 1080p.

You can opt to have Google Photos back up your photos and videos in their original quality, but anything backed up will count against your Google Drive storage limit.

Make sure you have Google Photos installed on your Android phone, turn on backup, and pick the quality you'd like to use.

The app will automatically back up your photos and videos whenever you're connected to Wi-Fi.

Samsung Cloud gives you 15GB of free backup storage.

Samsung offers its own backup and restore service through Samsung Cloud, and in my experience, it's been slightly more reliable than Google's backup service.

Check to make sure Samsung's backup service is turned on by opening the Settings app and selectingAccounts and backup>Backup and restore. There you'll find backup settings for your Samsung account, as well as Google account.

Use both services. Using both not only creates two backups of your phone but also gives you the flexibility to switch to a non-Samsung phone a year from now, if you decide to, because you can use Google's backup service to restore your phone.

Samsung provides multiple options for device backup.

Under the Samsung account section, tapBack up dataand make sure all of the data categories you want to be backed up to Samsung are checked. You can manually start a backup by selecting theBack upbutton at the bottom of the screen.

Backups to Samsung Cloud will use the 15GB of free storage space your Samsung Account has, with options to upgrade to 50GB for $0.99 or 200GB for $2.99 per month.

My favorite aspect of Samsung's backup service is that you can restore a single piece of data -- home screen layout, for example -- at any time. For instance, if you decide to rearrange your home screen and move a bunch of apps around, but later change your mind, you can go into Samsung's back up settings and restore your home screen from the last backup with just a couple of taps.

Under the Google Account section, make sureBack up my datais turned on. Selecting Google Account will open the same Google backup settings screen outlined in the section above, where you can view the current status of backups, as well as begin a manual backup.

In 2019, there's no reason anyone should lose their contacts list if they break their phone.

When it comes to backing up files you've downloaded or stored on your phone's storage, you have a couple of options. You can either connect your phone to your computer and move the files over manually, or you can use cloud storage services like Google Drive or Dropbox.

Set up either app on your Android device, then select the files or folders you want to upload and let the app do its thing.

Going forward, if you get in the habit of saving any files in the cloud, you'll always have a copy backed up, and you shouldn't have to worry about routinely manually uploading any updates to either service.

If you do lose your Android phone, we have a guide to help you track it down and get it back as soon as possible. We also have a handful of tips and tricks for mastering Android 10, the latest and greatest version of Android.

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How to back up your Android phone and keep all of your important information safe - CNET

Global Stem Cell and Regenerative Therapy Market – Yahoo Finance

Report Scope: The scope of this report is broad and covers various type of product available in the stem cell and regenerative medicines market and potential application sectors across various industries.

New York, Oct. 24, 2019 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Stem Cell and Regenerative Therapy Market" - https://www.reportlinker.com/p05791357/?utm_source=GNW The current report offers a detailed analysis of the stem cell and regenerative medicines market.

The report highlights the current and future market potential of stem cell and regenerative medicines and provides a detailed analysis of the competitive environment, recent development, merger and acquisition, drivers, restraints, and technology background in the market. The report also covers market projections through 2024.

The report details market shares of stem cell and regenerative medicines based on products, application, and geography.Based on product the market is segmented into therapeutic products, cell banking, tools and reagents.

The therapeutics products segments include cell therapy, tissue engineering and gene therapy. By application, the market is segmented into oncology, cardiovascular disorders, dermatology, orthopedic applications, central nervous system disorders, diabetes, others

The market is segmented by geography into the following regions: North America, Europe, Asia-Pacific, South America, and the Middle East and Africa. The report presents detailed analyses of major countries such as the U.S., Canada, Mexico, Germany, the U.K. France, Japan, China and India. For market estimates, data is provided for 2018 as the base year, with forecasts for 2019 through 2024. Estimated values are based on product manufacturers total revenues. Projected and forecasted revenue values are in constant U.S. dollars, unadjusted for inflation.

Report Includes: - 28 data tables - An overview of global markets for stem cell and regenerative medicines - Analyses of global market trends, with data from 2018, estimates for 2019, and projections of compound annual growth rates (CAGRs) through 2024 - Details of historic background and description of embryonic and adult stem cells - Information on stem cell banking and stem cell research - A look at the growing research & development activities in regenerative medicine - Coverage of ethical issues in stem cell research & regulatory constraints on biopharmaceuticals - Comprehensive company profiles of key players in the market, including Aldagen Inc., Caladrius Biosciences Inc., Daiichi Sankyo Co. Ltd., Gamida Cell Ltd. and Novartis AG

Summary The global market for stem cell and regenerative medicines was valued at REDACTED billion in 2018.The market is expected to grow at a compound annual growth rate (CAGR) of REDACTED to reach approximately REDACTED billion by 2024.

Growth of the global market is attributed to the factors such as growingprevalence of cancer, technological advancement in product, growing adoption of novel therapeuticssuch as cell therapy, gene therapy in treatment of chronic diseases and increasing investment fromprivate players in cell-based therapies.

In the global market, North America held the highest market share in 2018.The Asia-Pacific region is anticipated to grow at the highest CAGR during the forecast period.

The growing government funding for regenerative medicines in research institutes along with the growing number of clinical trials based on cell-based therapy and investment in R&D activities is expected to supplement the growth of the stem cell and regenerative market in Asia-Pacific region during the forecast period.

Reasons for Doing This Study Global stem cell and regenerative medicines market comprises of various products for novel therapeutics that are adopted across various applications.New advancement and product launches have influenced the stem cell and regenerative medicines market and it is expected to grow in the near future.

The biopharmaceutical companies are investing significantly in cell-based therapeutics.The government organizations are funding research and development activities related to stem cell research.

These factors are impacting the stem cell and regenerative medicines market positively and augmenting the demand of stem cell and regenerative therapy among different application segments.The market is impacted through adoption of stem cell therapy.

The key players in the market are investing in development of innovative products. The stem cell therapy market is likely to grow during the forecast period owing to growing investment from private companies, increasing in regulatory approval of stem cell-based therapeutics for treatment of chronic diseases and growth in commercial applications of regenerative medicine.

Products based on stem cells do not yet form an established market, but unlike some other potential applications of bioscience, stem cell technology has already produced many significant products in important therapeutic areas. The potential scope of the stem cell market is now becoming clear, and it is appropriate to review the technology, see its current practical applications, evaluate the participating companies and look to its future.

The report provides the reader with a background on stem cell and regenerative therapy, analyzes the current factors influencing the market, provides decision-makers the tools that inform decisions about expansion and penetration in this market.Read the full report: https://www.reportlinker.com/p05791357/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Global Stem Cell and Regenerative Therapy Market - Yahoo Finance

Stem cell therapy is for animals too – SciTech Europa

Stem cell therapy for animals has seen breakthroughs

Stem cell therapy is increasingly becoming a more mainstream form of medicine. Usually applied to humans, the use of this regenerative treatment is now also being extended to animals including cats and dogs. Regenerative medicine, particularly stem cell treatment has seen many advancements in recent years with some groundbreaking studies coming to light.

Taking the cells from bone marrow, umbilical cords, blood or fat, stem cells can grow to become any kind of cell and the treatment has seen many successes in animals. The regenerative therapy has been useful particularly for treatment of spinal cord and bone injuries as well as problems with tendons, ligaments and joints.

Expanded Potential Stem Cells (EPSCs) have been obtained from pig embryos for the first time. The cells offer groundbreaking potential for studying embryonic development and producing transnational research in genomics and regenerative medicine, biotechnology and agriculture.

The cells have been efficiently derived from pig preimplantation embryos and a new culture medium developed in Hong Kong and Cambridge enabled researchers from the FLI to establish permanent embryonic stem cell lines. The cells have been discovered in a collaboration between research groups from the Institute of Farm Animal Genetics at the Friedrich-Loeffler-Institut (FLI) in Mariensee, Germany, the Wellcome Trust Sanger Institute in Cambridge, UK and the University of Hong Kong, Li Ka Shing Faculty of Medicine, School of Biomedical Sciences.

Embryonic stem cells (ESC) are derived from the inner cells of very early embryos, the so-called blastocysts. Embryonic stem cells are all-rounders and can develop into various cell types of the body in the culture dish. This characteristic is called pluripotency. Previous attempts to establish pluripotent embryonic stem cell lines from farm animals such as pigs or cattle have resulted in cell lines that have not really fulfilled all properties of pluripotency and were therefore called ES-like.

Dr Monika Nowak-Imialek of the FLI said: Our porcine EPSCs isolated from pig embryos are the first well-characterized cell lines worldwide. EPSCs great potential to develop into any type of cell provides important implications for developmental biology, regenerative medicine, organ transplantation, disease modelling and screening for drugs.

The stem cells can renew themselves meaning they can be kept in culture indefinitely, and also show the typical morphology and gene expression patterns of embryonic stem cells. Somatic cells have a limited lifespan, so these new stem cells are much better suited for long selection processes. It has been shown that these porcine stem cell lines can easily be modified with new genome editing techniques such as CRISPR/Cas, which is particularly interesting for the generation of porcine disease models.

The EPSCs have a high capacity to develop not only into numerous cell types of the organism, but also into extraembryonic tissue, the trophoblasts, making them very unique and lending them their name. This capacity could prove valuable for the future promising organoid technology, where organ-like small cell aggregations are grown in 3D aggregates that can be used for research into early embryo development, various disease models and testing of new drugs in petri dishes. In addition, the authors were able to show that trophoblast stem cells can be generated from their porcine stem cells, offering a unique possibility to investigate functions or diseases of the placenta in vitro.

A major hurdle to using neural stem cells derived from genetically different donors to replace damaged or destroyed tissues, such as in a spinal cord injury, has been the persistent rejection of the introduced material (cells), necessitating the use of complex drugs and techniques to suppress the hosts immune response.

Earlier this year, an international team led by scientists at University of California San Diego School of Medicine successfully grafted induced pluripotent stem cell (iPSC)-derived neural precursor cells back into the spinal cords of genetically identical adult pigs with no immunosuppression efforts. The grafted cells survived long-term, displayed differentiated functionality and caused no tumours.

The researchers also demonstrated that the same cells showed similar long-term survival in adult pigs with different genetic backgrounds after only short course use of immunosuppressive treatment once injected into injured spinal cord.

Senior author of the paper Martin Marsala, MD, professor in the Department of Anesthesiology at UC San Diego School of Medicine said: The promise of iPSCs is huge, but so too have been the challenges. In this study, weve demonstrated an alternate approach.

We took skin cells from an adult pig, an animal species with strong similarities to humans in spinal cord and central nervous system anatomy and function, reprogrammed them back to stem cells, then induced them to become neural precursor cells (NPCs), destined to become nerve cells. Because they are syngeneic genetically identical with the cell-graft recipient pig they are immunologically compatible. They grow and differentiate with no immunosuppression required.

Co-author Samuel Pfaff, PhD, professor and Howard Hughes Medical Institute Investigator at Salk Institute for Biological Studies, said: Using RNA sequencing and innovative bioinformatic methods to deconvolute the RNAs species-of-origin, the research team demonstrated that pig iPSC-derived neural precursors safely acquire the genetic characteristics of mature CNS tissue even after transplantation into rat brains.

NPCs were grafted into the spinal cords of syngeneic non-injured pigs with no immunosuppression finding that the cells survived and differentiated into neurons and supporting glial cells at all observed time points. The grafted neurons were detected functioning seven months after transplantation.

Then researchers grafted NPCs into genetically dissimilar pigs with chronic spinal cord injuries, followed by a transient four-week regimen of immunosuppression drugs again finding long-term cell survival and maturation.

Marsala continued: Our current experiments are focusing on generation and testing of clinical grade human iPSCs, which is the ultimate source of cells to be used in future clinical trials for treatment of spinal cord and central nervous system injuries in a syngeneic or allogeneic setting.

Because long-term post-grafting periods between one and two years are required to achieve a full grafted cells-induced treatment effect, the elimination of immunosuppressive treatment will substantially increase our chances in achieving more robust functional improvement in spinal trauma patients receiving iPSC-derived NPCs.

In our current clinical cell-replacement trials, immunosuppression is required to achieve the survival of allogeneic cell grafts. The elimination of immunosuppression requirement by using syngeneic cell grafts would represent a major step forward said co-author Joseph Ciacci, MD, a neurosurgeon at UC San Diego Health and professor of surgery at UC San Diego School of Medicine.

Other recent advancements include the advancement toward having a long-lasting repair caulk for blood vessels. A new method has been for generating endothelial cells, which make up the lining of blood vessels, from human induced pluripotent stem cells. When endothelial cells are surrounded by a supportive gel and implanted into mice with damaged blood vessels, they become part of the animals blood vessels, surviving for more than 10 months.

The research was carried out by stem cell researchers at Emory University School of Medicine and could form the basis of a treatment for peripheral artery disease, derived from a patients own cells.

Young-sup Yoon, MD, PhD, who led the team, said: We tried several different gels before finding the best one. This is the part that is my dream come true: the endothelial cells are really contributing to endogenous vessels.

When cells are implanted on their own, many of them die quickly, and the main therapeutic benefits are from growth factors they secrete. When these endothelial cells are delivered in a gel, they are protected. It takes several weeks for most of them to migrate to vessels and incorporate into them.

Other groups had done this type of thing before, but the main point is that all of the culture components we used would be compatible with clinical applications.

This research is particularly successful as previous attempts to achieve the same effect elsewhere had implanted cells lasting only a few days to weeks, using mostly adult stem cells, such as mesenchymal stem cells or endothelial progenitor cells. The scientists also designed a gel to mimic the supportive effects of the extracellular matrix. When encapsulated by the gel, cells could survive oxidative stress inflicted by hydrogen peroxide that killed unprotected cells. The gel is biodegradable, disappearing over the course of several weeks.

The scientists tested the effects of the encapsulated cells by injecting them into mice with hindlimb ischemia (restricted blood flow in the leg), a model of peripheral artery disease.

After 4 weeks, the density of blood vessels was highest in mice implanted with gel-encapsulated endothelial cells. The mice were nude, meaning genetically immunodeficient, facilitating acceptance of human cells.

The scientists found that implanted cells produce pro-angiogenic and vasculogenic growth factors. In addition, protection by the gel augmented and prolonged the cells ability to contribute directly to blood vessels. To visualise the implanted cells, they were labelled beforehand with a red dye, while functioning blood vessels were labelled by infusing a green dye into living animals. Implanted cells incorporated into vessels, with the highest degree of incorporation occurring at 10 months.

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Stem cell therapy is for animals too - SciTech Europa

Stem Cell Therapy Market Will Achieve 10.2% CAGR to Cross $15bn by 2025: Global Market Insights, Inc. – Post Register

SELBYVILLE, Del. - October 23, 2019 - ( Newswire.com )

Global Stem Cell Therapy Market value will achieve over 10% CAGR to surpass USD 15 billion by 2025; according to a new research report by Global Market Insights, Inc. Rapid advancements in stem cell therapies in developed region such as North America and Europe will boost industry growth. Key industry players are indulged in introducing novel stem cell therapies that address limitations of existing therapies. Significant efficiency possessed by stem cell therapy has increased its preference in treatment of cancer that should positively impact the industry growth. However, high cost associated with stem cell therapies may hinder stem cell therapy industry growth to certain extent.

Increasing prevalence of chronic disease such as cancer, cardiovascular diseases will surge the demand for stem cell therapy business. Stem cell therapies have unique properties, such as immunosuppression, secretion of bioactive factors that fosters the development of tumor targeting technologies. Introduction of innovations in the regenerative medicine will help in conquering challenges in combating numerous diseases that should foster the industry growth.

Request for a sample of this research report @ https://www.gminsights.com/request-sample/detail/3331

Allogenic segment of stem cell therapy market accounted for over 39.5% revenue in 2018. Significant growth is attributed to the advantages associated with allogenic stem cell therapies. Allogenic stem cell involves transfer of stem cell from donor to patients. It overrules the limitations of autologous stem cell therapy such as difficulty in obtaining healthy and sufficient amount of stem cells from patients of diabetes, rheumatoid arthritis and other chronic diseases. Therefore, people have started relying more on the allogenic regenerative therapies that should augment the segmental growth.

Cardiovascular segment of stem cell therapy market is anticipated to witness around 9.5% CAGR throughout the forecast time frame. Increasing incidence of cardiovascular disease and growing healthcare concerns will surge the demand for stem cells employed in cardiovascular diseases. According to WHO, every year 17.9 million people die of cardiovascular disease. Stem cell therapies possess enormous potential to replace the conventional cardiovascular treatments that should increase its adoption over the forecast period.

Stem Cell Therapy Market Statistics, By Application

1.1. Key segment trends

1.2. Oncology 1.2.1. Market size, by region, 2014-2025 (USD Million)

1.3. Orthopedic 1.3.1. Market size, by region, 2014-2025 (USD Million)

1.4. Cardiovascular 1.4.1. Market size, by region, 2014-2025 (USD Million)

1.5. Neurology 1.5.1. Market size, by region, 2014-2025 (USD Million)

1.6. Others 1.6.1. Market size, by region, 2014-2025 (USD Million)

Browse key industry insights spread across 130 pages with 91 market data tables & 8 figures & charts from the report, Stem Cell Therapy Market Forecast 2019 - 2025 in detail along with the table of contents:

https://www.gminsights.com/industry-analysis/stem-cell-therapy-market

Clinics segment of stem cell therapy industry was valued at USD 2.5 billion in 2018. Considerable revenue size is attributed to superior treatment provided by clinics to cure life threating disease. Clinics are equipped with advance equipment and skilled workforce that ultimately enhance treatment outcomes. Moreover, clinics are believed to provide sophisticated stem cell therapies that will stimulate the segmental growth.

North America stem cell therapy market held over 9.5% CAGR in 2018 owing to favorable regulatory scenario and demographic trends. Improving regulatory scenario for stem cell therapies in the U.S. and Canada should positively impact the market growth. For instance, FDA has framed pre-marketing authorization rules for the commercialization of stem cell therapies. This helps to minimize adverse events caused due to defective regenerative therapies. Moreover, several companies focus on R&D activities to develop innovative stem cell therapies that should surge the regional growth.

Notable industry players operational in industry are Astellas Pharma, Cellectis, Celyad, DiscGenics, Gamida Cell, Capricor Therapeutics, Novadip Biosciences, Cellular Dynamics, CESCA Therapeutics, OxStem, ReNeuron Group, Mesoblast, and Takeda Pharmaceuticals. Industry players are adopting strategic initiatives such as collaborations product launches, geographic expansions, mergers and acquisitions in order to sustain industry competition and acquire prominent market. For instance, in 2019 Gamida cell formed agreement with Lonza to commercialize omidubicel after its FDA approval. Omiducel is potential life-saving stem cell treatment for treating hematologic malignancies. Thus, such collaborations will boost the companys growth considerably.

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Bioreactors Market Growth Report 2025: Biopharmaceutical products are individualized products with highly specific manufacturing requirements. Advanced biopharmaceutical manufacturing technologies have enabled development of effective drug delivery systems and drug device combination products. Some of the key industry players operating in the market include Eppendorf, GE Healthcare, Merck Millipore, Sartorius, and Thermo Fisher Scientific.

https://www.gminsights.com/industry-analysis/bioreactor-market

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Original Source: Stem Cell Therapy Market Will Achieve 10.2% CAGR to Cross $15bn by 2025: Global Market Insights, Inc.

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Stem Cell Therapy Market Will Achieve 10.2% CAGR to Cross $15bn by 2025: Global Market Insights, Inc. - Post Register

How Young India is fuelling the future of stem cell therapy and signing up to save lives – YourStory

Eighteen-year-old Aisha Choudhary was just like any other adolescent eyes filled with dreams and a heart brimming with energy. The only difference was she was battling a rare genetic disease, Severe Combined Immune Deficiency (SCID). Diagnosed when she was six months old and undergoing medical treatment for years, she was iron-willed in playing the cards she was dealt.

Since one of the most effective cures for SCID is a stem cell transplant (grafting of the parent cells from which all blood cells develop), Aishas parents, Niren and Aditi, decided to opt for that treatment mode. But their cells were not a complete match with their daughters, and they had to look at external donors. However, due to a low number of voluntary, registered stem cell donors, Aisha could not get a compatible donor whose genetic markers were a close enough match to hers. With no other alternative treatment available, Aisha had a bone marrow transplant. But, it came with a side-effect that cost her life Pulmonary Fibrosis, a disease known to damage the lung tissues.

Aishas Choudhary's role has been played by Zaira Wasim in The Sky is Pink.

Aishas journey has been captured in The Sky is Pink, a recent Bollywood movie starring Priyanka Chopra, Farhan Akhtar, Zaira Wasim, and Rohit Saraf.

The 18-year-olds life story is mirrored in the experiences of many who await stem cell donation as treatment for blood-related illnesses likeleukemia, lymphoma, and sickle cell anemia every year. With very few individuals signing up as donors and the probability of finding a match being a dismal 0.0008 percent in India (against a lean 16 percent abroad), fatalities are mounting year on year.

However, in recent times, there has been one small break in the clouds a number of youngsters, non-governmental organisations, and medical professionals have come forward and are working to spread awareness about stem cell donation and motivate a larger number of people to register as donors.

The stem cells in a human body mainly comprise red blood cells, platelets, and white blood cells. These are found in the umbilical cord of newborns and in the peripheral or circulating blood and bone marrow.

A stem cell donation is as simple and painless as a blood donation.

Certain diseases like blood cancer and leukemia tend to destroy the bone marrow or affect its functioning.For these, treatments like chemotherapy and radiotherapy are tried initially. However, in some cases, they do not prove effective for a cure. The only recourse then is replacing the patients stem cells with those of a healthy person.

One of the main criteria for a successful transplant is a good match between the stem cells of the donor and those of the patient. Therefore, a donor registry will administer a cheek swab test (tissue samples extracted from the cheek) on all potential donors to match cell characteristics. This procedure of pairing generic markers is called Human Leukocyte Antigen (HLA) in medical terms.

A cheek swab test in progress.

Each potential donors tissue is entered in the registry and given an identification number after the test is done. If the registry finds a match at any point in time, the donor is contacted to initiate the transplant.

There are many organisations today that are leading the charge in saving the lives of people suffering from serious blood disorders like cancer, thalassemia, and anaemia.

For instance, Datri, an Ahmedabad-based NGO, is working to create a wide and diverse database of potential stem cell donors by organising donation drives. Founded in 2009 by two doctors and an engineer, the organisation focuses on conducting awareness campaigns and helping individuals sign up on its registry as a committed and voluntary benefactor.

The team of the NGO Datri.

The idea for Datri was initially born in the minds of doctors Nezih Cereb and Soo Young Yang, who run a laboratory, Histogenetics, for determining tissue matches between patients and donors. Since pairing tissue types is imperative for any stem cell transplant, and confronting a severe shortage of donors, the doctor duo would run from pillar to post to meet hospitals requirements. Working with a number of the hospitals in India, they realised just how acute the shortfall was in people willing to donate stem cells. They recognised the immediate need to create a donor registry here.

Soon after, Raghu Rajagopal, an engineer from BITS Pilani and Director of ready-to-eat venture Millets and More, connected with them and they decided to start Datri.

Today, the functioning of the registry, its maintenance, and even the substantial costs involved in conducting the HLA matching are taken care of by the lab. In the last 10 years, Datri has gotten over four lakh people to register as donors and has saved around 600 lives through successful transplantation.

Every day, about 40 people are diagnosed with blood disorders in India. Though these can be cured through a stem cell transplant from a genetically matched donor, there is only a 25 percent chance of finding a match from within the family. Others have no option but to rely on unrelated donors. But the chances of getting a match is anywhere between one in 10,000 and one in two million. There is an urgent need to rope in as many potential donors as possible, which is precisely what Datri is trying to do, Raghu explains.

Another organisation that is dedicated to fighting blood disorders with stem cell treatment is DKMS-BMST. It was formed through a joint venture between two renowned NGOs DKMS, which is one of the largest international blood stem cell donor centres globally, and the Bangalore Medical Services Trust (BMST).

The team of DKMS-BMST.

DKMS was founded in Germany in 1991 by businessman Dr Peter Harf, after he lost his wife to leukemia. BMST was born in 1984 from the vision of Dr Latha Jagannathan, a medical director and managing trustee. Since both organisations had a common goal to find a matching donor for every patient with a blood disorder, they decided to come together to achieve it.

A group of youngsters registering to be stem cell donors.

So far, more than 37,000 people in India have registered as potential donors after attending DKMS-BMSTs donor drives.

In highly populous countries like India, thousands of people are in need of stem cell transplants every year to survive. Though donating stem cells is a painless and non-invasive process, it remains a lesser-known medical concept in India, with only 3.6 lakh people willing to play a part in it. Besides, the chances of stem cells of people of the same ethnicity matching are higher than those of individuals from different ethnic backgrounds. But, it is due to sheer lack of awareness that India lags severely in stem cell donations, say experts.

Students taking a cheek swab test at one of the colleges in Bengaluru.

Dr Govind Eriat, a reputed hematologist and bone marrow transplant specialist, says,

With a major hurdle to stem cell donation in India proving to be the myths surrounding the subject, the youth are coming forward to deconstruct common misconceptions.

For instance, 21-year-old Tejaswini Patel, a student of Information Science at New Horizon College of Engineering, Bengaluru, has been busting the false ideas on stem cell donation, starting among her family and friends. She says,

She adds, with a notable sense of pride, In the last two years alone, around 400 students from my college have registered themselves as donors.

(Edited by Athirupa Geetha Manichandar)

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How Young India is fuelling the future of stem cell therapy and signing up to save lives - YourStory

ReNeuron Presents Positive Data at the 27th Annual Congress of the European Society of Gene and Cell Therapy on Lead Cell Line – PRNewswire

PENCOED, Wales, Oct. 23, 2019 /PRNewswire/ --ReNeuron Group plc (AIM: RENE), a UK-based global leader in the development of cell-based therapeutics, is pleased to announce that new data relating to its CTX stem cell platform will be presented today at the 27th Annual Congress of the European Society of Gene and Cell Therapy(ESGCT), a leading scientific conference taking place this week in Barcelona, Spain.

Dr. Steve Pells, Principal Investigator at ReNeuron, will present new data showing the phenotypic stability and scalability of a mesenchymal stem cell line derived from the Company's proprietary, conditionally immortalized, human neural stem cell line (CTX) following re-programming to a pluripotent state.

The Company has previously presented data demonstrating that its CTX stem cell line, currently undergoing clinical evaluation for the treatment of stroke disability, can be successfully and rapidly re-programmed to an embryonic stem cell-like state enabling differentiation into any cell type. In essence, this means that the Company is able to take its neural stem cells back to being stem cells that can be made to develop into any other type of stem cell including bone, nerve, muscle and skin.

The new data being presented today show for the first time that these CTX-iPSCs (induced pluripotent stem cells) can indeed be differentiated along different cell lineages to generate, for example, mesenchymal stem cell lines. Further, the mesenchymal stem cell lines generated can be grown at scale by virtue of the Company's conditional immortalization technology, enabling the efficient production of clinical-grade cell therapy candidates.

These results are particularly encouraging as they demonstrate that CTX, a well-characterized, clinical-grade neural stem cell line, could be used to produce new conditionally immortalized allogeneic (i.e. non-donor-specific) cell lines from any of the three primary germ cell layers which form during embryonic development. ReNeuron is currently exploring the potential to develop further new allogeneic cell lines as potential therapeutic agents in diseases of unmet medical need for subsequent licensing to third parties.

Further information about the conference may be found at:

https://www.esgct.eu/congress/barcelona-2019.aspx

"The data we are presenting at the ESGCT Annual Congress represent a significant advance in the use of cell re-programming to generate new allogeneic cell lines as potential therapeutic candidates," commented Dr. Randolph Corteling, Head of Research at ReNeuron. "Importantly, the maintenance of the immortalization technology within these new cell lines may allow for the scaled production of 'off the shelf' allogeneic stem cells, such as haematopoietic stem cells as a potential alternative approach to those cancer immunotherapies currently in development that rely on the use of the patient's own T-cells."

About ReNeuronReNeuron is a global leader in cell-based therapeutics, harnessing its unique stem cell technologies to develop 'off the shelf' stem cell treatments, without the need for immunosuppressive drugs. The Company's lead clinical-stage candidates are in development for the blindness-causing disease, retinitis pigmentosa, and for disability as a result of stroke. ReNeuron is also advancing its proprietary exosome technology platform as a potential delivery system for drugs that would otherwise be unable to reach their site of action. ReNeuron's shares are traded on the London AIM market under the symbol RENE.L. For further information visit http://www.reneuron.com.

ENQUIRIES:

ReNeuron

+44 (0)20 3819 8400

Olav Helleb, Chief Executive Officer

Michael Hunt, Chief Financial Officer

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+44 (0) 20 7466 5000

Mark Court, Tilly Abraham

Argot Partners (US)

Stephanie Marks, Claudia Styslinger

Stifel Nicolaus Europe Limited

+1 212 600 1902

+44 (0) 20 7710 7600

Jonathan Senior, Stewart Wallace, Ben Maddison (NOMAD and Joint Broker)

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+44 (0) 20 7496 3000

Aubrey Powell, James Moat, Mia Gardner

(Joint Broker)

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ReNeuron Presents Positive Data at the 27th Annual Congress of the European Society of Gene and Cell Therapy on Lead Cell Line - PRNewswire

NIH and Gates Foundation lay out ambitious plan to bring gene-based treatments for HIV and sickle cell disease to Africa – Science Magazine

A new $200 million collaboration aims to speed development of genetic cures for people in Africa with sickle cell disease (above) and, separately, HIV infection.

By Jon Cohen, Jocelyn KaiserOct. 23, 2019 , 5:00 PM

Two major U.S. biomedical research funders plan to each put at least $100 million over 4 years toward bringing cutting-edge, gene-based treatments to a part of the world that often struggles to provide access to even basic medicines: sub-Saharan Africa. The National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation today announced the unusual collaboration to launch clinical trials for gene-based cures for HIV and sickle cell disease within the region in the coming decade.

The ambitious goal is to steer clear of expensive, logistically impractical strategies that require stem cell transplantation, and instead develop simpler, affordable ways of delivering genes or gene-editing drugs that can cure these diseases. Yes, this is audacious, NIH Director Francis Collins said during a press teleconference this morning on the project. But if we dont put our best minds, resources, and visions together right now, we would not live up to our mandate to bring the best science to those who are suffering.

After decades of work and setbacks, the traditional gene therapy approach of delivering DNA into the body to replace a defective gene or boost a proteins production is now reaching the clinic for several diseases, including inherited blindness, neuromuscular disease, and leukemia. Animal studies and some clinical trials have suggested that two diseases prevalent in Africa, HIV and sickle cell disease, can be treated by gene therapies or newer genome-editing tools such as CRISPR.

But in most cases, introducing those therapeutic genes or the components of a genome editor involves removing stem cells from the body, adding or modifying genes, then reinfusing the cells back into the body. That is essentially a stem cell transplant with ones own cells, an expensive procedure that is also typically risky because physicians wipe out most of a patients existing stem cells with chemotherapy so the corrected cells can engraft and grow. It remains out of reach for most people in sub-Saharan Africa, where few places have the medical infrastructure to support such intensive interventions.

Yet sub-Saharan Africa is home to about two-thirds of the 20 million people with sickle cell disease and the 38 million living with HIV. The NIH-Gates partnership is an incredible opportunity to find new therapies and possible cures for two diseases that affect millions of Africans and to make them available at affordable costs, said Matshidiso Moeti, who heads the Regional Office for Africa at the World Health Organization.

Anthony Fauci, director of NIHs National Institute of Allergy and Infectious Diseases, noted that if this collaboration pans out, it could also lead to enormous cost savings. If we do successfully achieve an HIV cure, it will ultimately be important not only for millions of individuals with HIV, but also will save hundreds of billions of dollars in health care costs, said Fauci, whose institute already funds a major HIV cure initiative.

In sickle cell disease, which involves a defect in the oxygen-carrying hemoglobin in red blood cells, several ongoing gene therapy and gene-editing clinical trials in the United States and Europe are either adding a new hemoglobin gene to cells or turning on the gene for a fetal form of the protein. Other clinical trials for HIV have used CRISPR or other genome editors in stem cells to cripple a receptor, CCR5, that the virus depends on to establish infection.

Instead of modifying a persons stem cells and transplanting them back, the new collaboration will seek to ferry a therapeutic gene or gene-editing tools directly into the body (in vivo) with vectorssuch as harmless viruses or nanoparticles, Collins said. The treatment itself would be similar to a simple blood transfusion. Although studies are already underway with viral vectors that deliver new genes to certain tissues in people, in vivo gene therapy has only been used to modify blood stem cells in animal models of certain diseases. Figuring out how to home in on and modify those cells in people is a big part of the collaborations plan, Collins said.

Hematologist Alexis Thompson of the Northwestern University Feinberg School of Medicine in Chicago, Illinois, who is involved with some sickle cell gene therapy trials, calls the NIH-Gates collaboration phenomenal.But, she says, a more urgent need is to expand efforts to screen newborns in Africa for sickle cell disease and treat them with antibiotics; at the moment, the majority die before age 5 from bacterial infections because the sickled cells impair the spleens ability to filter bacteria and make antibodies. Unless more children with sickle cell disease mutations survive longer, there will be few to be cured with the new gene-based treatments, Thompson says. Its almost being able to crawl or walk before you sprint.(Gates and NIH say they plan to support screening efforts outside of the new collaboration.)

For HIV, a big impetus for the cure push builds on two people infected with the AIDS virus who were cured with stem cell transplants. These two men each had blood cancers that required the transplants, which intentionally used blood from donors who had white blood cells with crippled CCR5 receptors. After the transplants, whatever HIV remained in these men could not enter new host cells, and their infections petered out. This new initiative hopes to speed development of direct injections of gene editor components that can target theCCR5gene in blood cells and cripple it. The potential beauty of in vivo gene editing is that it might be given ultimately as a single shot, curing everyone in a scalable manner, says Steven Deeks, a leading HIV cure researcher at the University of California, San Francisco.

The collaboration will also try to speed development of more experimental interventions that directly excise HIVs genetic material from a patients cells or allow people to artificially make superpotent antibodies against the virus. This might be science fiction now, but one day may be a real possibility, Deeks says.

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NIH and Gates Foundation lay out ambitious plan to bring gene-based treatments for HIV and sickle cell disease to Africa - Science Magazine

Green Tea Acts as a "Remote Control" To Switch on Cell Therapy – Technology Networks

Let's play a game of word association. I'll go first.

Cell Therapy

What words spring to mind? CRISPR? Medicine? Genetic disorders? Cancer? Gene therapy?

What about green tea? Unlikely, I imagine.

But in a new study published today in Science Advances, researchers from East China Normal University have created an elegant system for activating genetically edited cells using green tea.1Realizing the promise of cell therapiesEngineered cell therapies, deemed the "next frontier" in modern medicine, contain specific cellular material that triggers a desired effect in vitro or in vivo. Such therapies are in development in laboratories across the globe for an array of different conditions, including acute myocardial infarction (heart attack), brain cancer, breast cancer, diabetes and liver diseases. They offer a novel avenue of therapeutics for patients suffering from diseases for which treatment options are limited.For their efficacious and safe use in the clinic, scientists need to be able to regulate the activity of these cells in vivo. Essentially, they require a "remote control". This has proven a major barrier for the delivery of cell therapies to patients. Initial work in this field has adopted antibiotics such as doxycycline or tetracycline as remote-control triggers for gene expression in the cells. However, regular use of antibiotics may result in antibiotic resistance and other adverse side effects.So, what alternatives exist?Haifeng Ye, Professor at East China Normal University, says "Ideal trigger molecules for clinical biomedical applications would be natural, non-toxic, highly soluble, inexpensive, and perhaps even beneficial to health."Previous studies have reported that remote control switches can be activated through the use of food or cosmetic preservatives, vanillic acid, benzoate and phloretin for example. These molecules do not naturally occur in food however, and the safety implications of their long-term use is not well known.A green solution?Nothing beats a good cup of tea. It is the second most popular beverage on the planet (following water) and can be found in the household cupboards of 80% of Americans. Tea is available in a variety of forms, including but not limited to black tea, oolong tea, white tea and green tea. A plethora of research studies have documented the numerous health benefits of green tea consumption, including anticarcinogenic, anti-inflammatory, antimicrobial, and antioxidant effects.The components of green tea most heavily researched with regards to health are the polyphenols, of which the most pertinent are flavonoids, and the most pertinent flavonoids are the catechins.2Post green-tea consumption, the tea catechins and phenolic acids undergo metabolic processing to form the antioxidant protocatechuic acid (PCA). In their latest study, Ye and team have utilized this antioxidant as a "remote control" for activating gene switches in cells. "PCA is a major tea catechin compound produced by humans following green tea consumption that has powerful antioxidant activity. Therefore, in this study, we showed the use of protocatechuic acid (we call it PCA), a metabolite after tea drinking, as a trigger molecule," Ye told Technology Networks.PCA-inducible gene switchesIn the study, the scientists engineered PCA-inducible gene switches in mammalian cells. Initially, they explored the potential for using PCA to monitor cell-based long-term therapies in vivo by integrating the genetic switch into HEK-293 cells and found that the cell line demonstrated reversible and tunable induction kinetics, which the authors regard as "excellent switching performance". This was characterized by negligible basal expression and nonsaturating increases in the transgene output over the course of a 15-day trial.Next, they microencapsulated and implanted the HEK cells into mice. Ye tells us, "The alginate-poly (L-lysine)-alginate-based encapsulation technology was used in our study for cell therapy. This clinically validated implant technology enables the free diffusion of metabolites, nutrients and proteins of lower molecular weights (<72 kDa) across the biocompatible capsule membrane while shielding their cellular content from physical contact with the hosts immune system. The implant technology has been successfully validated in human clinical trials and the performance of the material is continuously improved for clinical applications."The researchers found that, regardless of delivery method (intraperitoneal, oral intake from water, or oral intake from concentrated green tea), PCA could control the secretion of a reporter protein, SEAP, in a dose-dependent manner.Making CRISPR more crisp?CRISPR gene-editing shows promise in revolutionizing personalized medicine. A notable key issue with CRISPR, however, is the "off target" effects that limit its specificity. In this study, the scientists used the PCA-responsive cells to perform more targeted CRISPR gene editing: "By applying newly-designed fusion-protein-based PCA-controlled gene switches to Pol III promoters, we created trigger-inducible expression systems for gRNAs to program PCA-mediated CRISPR/Cas9-activity," says Ye.Exploring diabetes treatment with PCA-induced cell therapyYe and colleagues next tested the potential of the PCA remote control system for treating experimental diabetes using a mouse model. Using the switch, they engineered two different cell lines: one that enabled PCA-inducible expression of the reporter protein SEAP and insulin, and the other producing a short variant of human glucagon-like peptide 1 and SEAP. Implantation of these cells into mouse models of type 1 diabetes and type 2 diabetes mellitus resulted in restored homeostatic fasting blood glucose concentrations and glucose tolerance upon PCA injection.Recognizing that the translation of research findings from mouse models to humans in the clinic can be problematic, the scientists then decided to explore the PCA remote control switch efficacy in non-human primate models of diabetes. In parallel to the treatment efficacy observed in the type 2 diabetic mice, daily oral administration of PCA rapidly increased the expression of glucagon-like peptide 1 and restored glucose homeostasis in diabetic monkeys.In terms of safety, blood biochemical analyses related to inflammatory responses found that white blood cell count, lymphocytes, monocytes, eosinophils, and basophils, did not increase at any point during the treatment when compared with pre-treatment.The study findings certainly excite the authors, "Although there have not yet been preclinical studies for the application of engineered cellbased therapies in humans, this first-in-monkey study demonstrates the feasibility of safely and successfully scaling up a treatment strategy by controlling microencapsulated engineered cells to release therapeutic outputs from animals such as mice to larger NHPs. Therefore, this study substantiates the medical utility of concepts developed in synthetic biology," they note in the discussion of the paper.How much tea is too much tea?Hypothetically, if this therapy was to reach the clinic, I ponder over the possibility of an individual consuming "too much" green tea, and how this might impact the therapy. Ye is quick to inform me that this would not be an issue, "Only custom prepared concentrated green tea can activated the implanted designer cells. The normal green tea drinks cannot activate the implanted cells because of low concentration," he says.The future looks greenThe study is comprehensive, assessing the PCA "switch" in a variety of cell lines and mammalian models with a variety of control measures in place.Thus, in which direction will this research go next? I ask Ye, who tells me, " We will next focus on solving the following limitations:(1) The PCAON-switch was stably integrated into [the] genome by a "Sleeping Beauty" transposon system. Due to a random integration, unwanted insertional mutagenesis might occur. We will next consider using gene editing tools, such as CRISPR, to enable facile and permanent integration of the switch into the targeted genomic sequences in human cells without insertional mutagenesis;(2) The chassis of the HEK-293 cells are easily handled, transfected, and compatible to the PCAON-switch. For translational applications, they must also be safe (no side effects) in humans. Hence, we will test the therapeutic efficiency of the PCAON-switch in autologous parental cells from patients own mesenchymal stem cells, which may provide immunocompatible and noncarcinogenic autologous or allogeneic cell sources;(3) The lifespan of the designer cells inside the alginate microcapsules is an imperative issue. To realize long-term cell therapy, we will further improve the encapsulation technology."Haifeng Ye, Professor at East China Normal University, was speaking with Molly Campbell, Science Writer, Technology Networks.References:1. A green teatriggered genetic control system for treating diabetes in mice and monkeys," by J. Yin; L. Yang; K. Dong; J. Jiang; S. Xue; Y. Xu; X. Wang; H. Ye at East China Normal University in Shanghai, China; L. Mou; Y. Lu at First Affiliated Hospital of Shenzhen University in Shenzhen, China.2. Reygaert. 2018. Green Tea Catechins: Their Use in Treating and Preventing Infectious Diseases. Biomed Research International. doi: 10.1155/2018/9105261.

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Green Tea Acts as a "Remote Control" To Switch on Cell Therapy - Technology Networks

United Therapeutics receives permit for cell therapy facility build-out at Mayo – Jacksonville Daily Record

United Therapeutics received a building permit Tuesday for a $9.5 million build-out of its cell therapy facility on the second floor of Mayo Clinics Discovery and Innovation Building.

The 21,843-square-foot space will house an automated stem cell manufacturing site, which is one of the first of its kind in the country. The Whiting-Turner Contracting Co. is the project contractor.

The technology, approved by the FDA in 2018, allows the Mayo Clinic Center for Regenerative Medicine to produce cells from the bone marrow of a stem cell donor in large enough quantities to be used as treatments in clinical trials. It allows for the treatment of multiple patients at the same time.

Construction began in 2017 on the $32.4 million building at 14221 Kendall Hench Drive. It held a grand opening in August.

The first floor houses three ex-vivo lung perfusion surgical suites used for lung restoration, another form of regenerative medicine. It turns donor lungs, which previously would have previously been unusable, into viable transplant organs. United Therapeutics also collaborates with Mayo Clinic on lung restoration.

The third floor houses the Life Sciences Incubator for biotech entrepreneurs, which offers coworking space, wet labs, business resources, networking and entrepreneurial training.

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United Therapeutics receives permit for cell therapy facility build-out at Mayo - Jacksonville Daily Record

BrainStorm Cell Therapeutics President and CEO to be Featured as Keynote Speaker at Cell Series UK 2019 – Yahoo Finance

NEW YORK, Oct. 24, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, today announced, Chaim Lebovits, President and CEO, will serve as a Keynote Speaker at Cell Series UK.Cell Series UK, will be held October 29-30, 2019, at London Novotel West, London, UK. The Conference, organized by Oxford Global, is one of the foremost events in Europe focused on regenerative medicine and cellular innovation.

Ralph Kern MD, MHSc, Chief Operating and Chief Medical Officer of Brainstorm, who will also participate at Cell Series UK stated, We are very pleased to have Chaim Lebovits presenting at this prestigious conference where global leaders in stem cell and regenerative medicine will have the opportunity to learn more about NurOwn and the critical research being conducted by the Company. Mr. Lebovits Keynote Address, Stem Cell Therapeutic Approaches For ALS, will be presented to leading members of the scientific and business community including potential partners and investors.

About NurOwnNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

AboutBrainStorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn Cellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm received U.S. FDA clearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) in December 2018 and has been enrolling clinical trial participants since March 2019. For more information, visit the company's website.

Safe-Harbor Statements Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

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BrainStorm Cell Therapeutics President and CEO to be Featured as Keynote Speaker at Cell Series UK 2019 - Yahoo Finance

Cesca Therapeutics Forms Joint Venture With HealthBanks Biotech (USA) To Provide Immune Cell Banking And Cell Processing Service – Clinical Leader

ImmuneCyte to Begin Operations in Fourth Quarter of 2019

Rancho Cordova, CA /PRNewswire/ - Cesca Therapeutics Inc. (Nasdaq: KOOL), a market leader in automated cell processing and autologous cell therapies for regenerative medicine, and ThermoGenesis, its wholly owned device subsidiary, today announced that the company has entered into a definitive joint venture agreement with HealthBanks Biotech (USA) Inc., one of the world's leading stem cell bank networks, to commercialize its proprietary cell processing platform, CAR-TXpress, for use in immune cell banking as well as for cell-based contract development and manufacturing services (CMO/CDMO). The joint venture will be named ImmuneCyte Life Sciences Inc. ("ImmuneCyte") and is expected to officially launch during the fourth quarter of 2019.

Under terms of the agreement, ImmuneCyte will initially be owned 80% by HealthBanks Biotech and 20% by Cesca. Cesca will contribute to ImmuneCyte exclusive rights to use ThermoGenesis' proprietary cell processing technology for the immune cell banking business and non-exclusive rights for other cell-based contract development and manufacturing services. Cesca will also contribute its clinical development assets to the joint venture, as the company has decided to discontinue these activities in order to focus exclusively on the device business.

Once operational, ImmuneCyte will be among the first immune cell banks in the U.S. to provide clients with the opportunity to bank their own healthy immune cells for future use as a resource for cell-based immunotherapies, such as dendritic cell and chimeric antigen receptor (CAR) T-cell therapies. ImmuneCyte will utilize ThermoGenesis' proprietary CAR-TXpress platform which allows for the isolation of different components from 200 ml of blood in cGMP compliant, closed system. Given that the CAR-TXpress platform can increase cell processing efficiency by up to 16-fold as compared with the traditional, labor-intensive ficoll gradient centrifugation-based cell processing method, ImmuneCyte is expected to offer customers an unparalleled competitive advantage, including an ability to store their own immune cells at a tangibly lower cost.

"The ImmuneCyte joint venture will be paramount to the execution of our strategy to become a preferred cell processing and manufacturing solution provider in the cell and gene therapy field," said Dr. Chris Xu, Chairman and Chief Executive Officer of Cesca Therapeutics. "CAR-T therapeutic research is advancing rapidly. Partnering with HealthBanks Biotech, one of the foremost stem cell bank networks, with an experienced team and an established global infrastructure, will offer customers the ability to preserve younger, healthier and uncontaminated immune cells for potential future use. By applying our proprietary CAR-TXpress technology to immune cell banking and other CDMO cellular manufacturing services, we will allow for the manufacture and production of more effective and less costly immunotherapies."

In 2017, the U.S. Food and Drug Administration (FDA) approved two CAR-T cell therapies, under breakthrough designation, for the treatment of advanced B cell leukemia and lymphomas. Both use autologous (a patient's own) immune T cells to fight cancer and have reported an over 80% response rate in the "no-option" patient group, for those who have failed both chemo- and radiation therapies. This has helped to spur massive global interest for the development of additional CAR-T immunotherapies1. By the end of September 2019, there were over 800 CAR-T cell clinical trials registered on the http://www.clinicaltrials.gov website, targeting a wide variety of blood cancers and solid tumors.

Although highly effective, several recent studies on the eligibility of patients to enroll in CAR-T clinical trials showed that as many as 30-50% of cancer patients may not be eligible to enroll or to get sufficient CAR-T cells manufactured for the therapy. Reasons may include: (1) the function of the immune system declines with age and can be negatively affected by other medical conditions, (2) most standard cancer therapies, such as chemotherapy and radiation, destroy the immune system, and (3) in many cases of advanced cancer, cancer cells will enter circulation, invade and interfere with the body's natural production of immune cells. According to a recently reported JULIE trial, a CAR-T clinical trial in relapsed or refractory diffuse large B-cell lymphoma (DLBCL), one-third of the 238 screened patients failed to be enrolled, and more than half of the 238 failed to receive the intended CAR-T therapy2,3. ImmuneCyte will offer customers the ability to preserve younger, healthier and uncontaminated immune cells, for potential future use in advanced cancer immunotherapy.

About HealthBanks Biotech (USA) Inc.HealthBanks Biotech, headquartered in Irvine, CA, is one of the leading stem cell bank networks in the world and offers services globally through its sister companies located in the United States and other regions and nations. HealthBanks Biotech is accredited by the FDA, AABB, and CAP. The HealthBanks Biotech group was originally founded in 2001 with a vision that stem cells and cell and gene therapies could transform modern medicine. HealthBanks Biotech is a subsidiary of Boyalife Group, Inc. (USA), an affiliate of Boyalife (Hong Kong) Limited, the largest stockholder of Cesca. For more information about HealthBanks Biotech (USA) Inc., please visit: http://www.healthbanks.us.

About ImmuneCyte Life Sciences Inc.ImmuneCyte will provide clients with the opportunity to bank their own immune cells when the cells are "healthy and unaffected" as a future resource for cellular immunotherapies, such as CAR-T. ImmuneCyte utilizes a proprietary CAR-TXpress platform, a GMP compliant close-system capable of automated separating and cryopreserving different components from blood. For more information about ImmuneCyte Life Sciences Inc., please visit: http://www.immunecyte.com.

About Cesca Therapeutics Inc.Cesca Therapeutics develops, commercializes and markets a range of automated technologies for CAR-T and other cell-based therapies. Its device division, ThermoGenesis develops, commercializes and markets a full suite of solutions for automated clinical biobanking, point-of-care applications, and automation for immuno-oncology. The Company has developed a semi- automated, functionally closed CAR-TXpress platform to streamline the manufacturing process for the emerging CAR-T immunotherapy market. For more information about Cesca and ThermoGenesis, please visit: http://www.cescatherapeutics.com.

References:1. Facts About Chimeric Antigen Receptor (CAR) T-Cell Therapy, Leukemia and Lymphoma Society (2018). https://www.lls.org

2. Updated Analysis of JULIET Trial: Tisagenlecleucel in Relapsed or Refractory DLBCL (2018).

3. Eligibility Criteria for CAR-T Trials and Survival Rates in Chemorefractory DLBCL. Journal of Clinical Pathways (2018).

SOURCE: Cesca Therapeutics Inc.

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Cesca Therapeutics Forms Joint Venture With HealthBanks Biotech (USA) To Provide Immune Cell Banking And Cell Processing Service - Clinical Leader

Rocket Pharmaceuticals Presents First Evidence of Long-Term Improvement and Stabilization in Blood Counts and Durable Mosaicism in RP-L102 Process A…

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a leading U.S.-based multi-platform clinical-stage gene therapy company, today presents updated long-term follow-up from the Phase 1/2 clinical trial of RP-L102 at the European Society of Cell and Gene Therapy (ESGCT) 27th Annual Congress in Barcelona, Spain. RP-L102 is the Companys lentiviral vector (LVV)-based gene therapy for the treatment of Fanconi Anemia (FA). The data are included in an oral presentation by Dr. Juan Bueren, Scientific Director of the FA gene therapy program and Head of the Hematopoietic Innovative Therapies Division at CIEMAT in Spain / CIBERER / IIS-FJD, entitled, Gene Therapy for Patients with Fanconi Anaemia.

Two critical validations for an FA gene therapy product are: 1) stem cell engraftment in the absence of cytotoxic conditioning and 2) evidence of sustained clinical improvement. We are proud to report that the maturing long-term data from the patients treated with RP-L102 meet both of these requirements, said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. In all four patients, bone marrow MMC-resistance, a key measure of phenotypic reversal and engraftment, meets or exceeds the 10% threshold agreed to by the FDA and EMA for the upcoming registration-enabling Phase 2 trial, and all four patients now resemble FA mosaic patients as evaluated by peripheral T-cell chromosomal fragility assay. Remarkably, patients 02002 and 02006, who received what we consider adequate drug product similar to the upcoming Phase 2 trial, now demonstrate durable robust bone marrow MMC-resistance levels of approximately 60% and 32%, respectively, confirming phenotypic correction in long-term bone marrow stem and progenitor cells. Of note, each of the four initial patients continue to show evidence of a proliferative advantage, with ongoing increases in peripheral mononuclear cell VCNs. In addition, improvement or stabilization of peripheral blood counts, which had declined substantially prior to gene therapy, suggests a halt in bone marrow failure progression. In patient 02002, hemoglobin levels are now similar to those in the first year after birth, and all lineages in patients 02002 and 02006 are now stable or improving.

Dr. Shah continued, Preliminary VCN data from three additional patients who were treated with a viable drug product also show engraftment in a dose-dependent manner, consistent with the first four patients. With this progress to date, we look forward to the upcoming results from the first two patients receiving Process B of RP-L102, designed to enable consistent results with commercial-grade product.

The presentation described nine pediatric patients (ages 3-7 years) who received RP-L102 utilizing fresh or cryopreserved mobilized peripheral blood CD34+ cells that were transduced with the therapeutic vector. Four of these patients have been followed for more than 2 years (24-39 months for patients 02002, 02004, 02005, and 02006). The Phase 1/2 study of RP-L102 is an ongoing, open-label, single-center study designed to evaluate the safety and efficacy of Process A RP-L102 without the use of any conditioning regimen conventionally used in allogenic transplant.

Dr. Bueren noted, These results indicate the feasibility of engraftment in FA patients using autologous, gene corrected HSCs in the absence of any conditioning regimen. This indicates the potential of this therapeutic approach as a definitive hematologic treatment, while avoiding the burdensome side effects associated with allogeneic transplant, including the risk of post-transplant mortality and a substantially higher risk of head and neck cancer. The ability to treat patients without the use of genotoxic conditioning and to restore blood cell counts is a life-altering advancement for patients and their families, as well as the scientific community which has dedicated over two decades to finding a minimally toxic alternative for FA patients.

Rocket expects initial data from the Phase 1 Process B trial of RP-L102 by year-end. The registration-enabling Phase 2 study in Spain is now enrolling, and additional global sites will follow.

Full results from the ESGCT presentation will be available online at the conclusion of the oral presentation: https://www.rocketpharma.com/esgct-presentations/.

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a FANC-A gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANC-A gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natures gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells1.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rockets multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket's clinical programs using LVV-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using AAV-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rockets pre-clinical pipeline program is for Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the pre-clinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2018. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Presents First Evidence of Long-Term Improvement and Stabilization in Blood Counts and Durable Mosaicism in RP-L102 Process A...

Global Stem Cell Therapy Market Global and Regional Analysis by Top Key Market Players, Key Regions, Product Segments, and Applications 2024 – Globe…

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Orchard Therapeutics Presents Data from OTL-200 in Patients with Metachromatic Leukodystrophy Using Cryopreservation – BioSpace

BOSTON and LONDON, Oct. 22, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced initial results from a clinical trial with a cryopreserved formulation of OTL-200, a gene therapy in development for the treatment of metachromatic leukodystrophy (MLD) at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. The initial data show that cellular engraftment with OTL-200 using a cryopreserved formulation is similar to that observed using a fresh formulation with the longest patient having 12 months of follow-up since treatment. The data are being featured this week in a poster session at the European Society of Gene & Cell Therapy (ESGCT) Annual Congress in Barcelona, Spain.

MLD is a devastating and rapidly progressing disease with no standard treatment options. In its most severe forms, patients will not survive beyond their first decade of life.

These data compare the initial results of OTL-200 in the first four MLD patients treated using a cryopreserved formulation to a previously presented integrated analysis of 29 patients treated with a fresh formulation that demonstrated meaningful clinical outcomes. Hematopoietic stem cells are collected, purified and transduced in the same way for both formulations. For the cryopreserved formulation, following transduction, the gene-corrected cells are placed in a specific medium that allows them to be stably frozen. After successful testing and release, the cryopreserved cells are shipped to the site of care where they are thawed and administered to patients who have received conditioning.

Presenting the first supportive data on OTL-200 using a cryopreserved formulation represents a cross-functional effort involving our clinical, CMC and regulatory teams as we prepare for the upcoming European regulatory submission for MLD followed by a BLA in the U.S., said Mark Rothera, president and chief executive officer of Orchard. If approved, a cryopreserved formulation of OTL-200 would more readily facilitate global commercialization and patient access efforts, which are key elements in our mission to deliver potentially curative therapies to patients suffering from often-deadly rare diseases.

Mr. Rothera continued, With over 40 patients now treated using a cryopreserved formulation across our pipeline of six clinical-stage programs, we are confident our approach is supported by a robust set of evidence.

Study Results At the time of the analysis, four early-onset MLD patients (two late infantile and two early juvenile) have been treated with the cryopreserved formulation of OTL-200. All patients are alive and were followed for a minimum of one month, with the longest follow-up out to 12 months in the first patient treated (median follow-up of 0.38 years). The age at the time of treatment ranged from seven months to 42 months.

The initial results in patients receiving the cryopreserved formulation (n=4) demonstrated the following:

Figure 1. Profiles of VCN in bone marrow CD34+ cells: OTL-200 cryopreserved vs. OTL-200 fresh

https://www.globenewswire.com/NewsRoom/AttachmentNg/83f41457-927b-4b1b-9ac2-9d48ac10353a

Figure 2. ARSA activity profile in peripheral blood: OTL-200 cryopreserved vs. OTL-200 fresh

https://www.globenewswire.com/NewsRoom/AttachmentNg/393ca5f0-98ad-47f8-b723-35c5c6c08d8f

c = cryopreserved; f = fresh; Sbj. = subject

We are pleased that these initial data suggest that using gene-corrected cells that have been cryopreserved has a similar impact on clinical biomarkers for early-onset MLD patients as the OTL-200 fresh formulation, said Dr. Valeria Calbi, a hematologist at San Raffaele Scientific Institute and SR-Tiget and an investigator of the study. The four treated patients showed good levels of engraftment of gene-corrected cells and reconstitution of ARSA activity at multiple time points, as well as encouraging early trends in GMFM scores that we look forward to evaluating with additional follow-up. We believe that these data further support the positive benefit / risk profile of OTL-200 as a therapy with potential lifelong benefit for patients with MLD.

Next Steps for OTL-200 Orchard remains on track to submit a marketing authorization application, or MAA, in Europe for MLD in the first half of 2020, as well as a biologics licensing application, or BLA, in the U.S. approximately one year later.

About MLD and OTL-200Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, the gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged and patients with MLD will experience neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures, finding it more and more difficult to move, talk, swallow, eat and see. Currently, there are no effective treatments for MLD. In its late infantile form, mortality at 5 years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1 OTL-200 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MLD. OTL-200 was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

About OrchardOrchard Therapeutics is a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.

Orchards portfolio of ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis, a gammaretroviral vector-based gene therapy and the first such treatment approved by the European Medicines Agency for severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. Strimvelis, as well as the programs in MLD, WAS and TDT were acquired by Orchard from GSK in April 2018 and originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy initiated in 2010.

Orchard currently has offices in the U.K. and the U.S., including London, San Francisco and Boston.

Forward-Looking StatementsThis press release contains certain forward-looking statements which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including OTL-200 for the treatment of metachromatic leukodystrophy, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, including OTL-200, and the likelihood that such data will be positive and support further clinical development and regulatory approval of its product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including OTL-200, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading Risk Factors in Orchards annual report on Form 20-F for the year ended December 31, 2018 as filed with the U.S. Securities and Exchange Commission (SEC) on March 22, 2019, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

1Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature. Journal of Child Neurology 2010, DOI: http://doi.org/10.1177/0883073809341669

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

Figure 1

Profiles of VCN in bone marrow CD34+ cells: OTL-200 cryopreserved vs. OTL-200 fresh

Figure 2

ARSA activity profile in peripheral blood: OTL-200 cryopreserved vs. OTL-200 fresh; c = cryopreserved; f = fresh; Sbj. = subject

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Orchard Therapeutics Presents Data from OTL-200 in Patients with Metachromatic Leukodystrophy Using Cryopreservation - BioSpace

Progress toward improving detection, monitoring and treatment of metastatic cancers – Brain Tumour Research

24 October 2019

Most cancers kill because tumour cells spread, or metastasise beyond the primary site, for example breast, to invade other organs, brain being one. Now, a University of Southern California (USC), study has found that circulating tumour cells can actually target specific distant organs.

Their study of brain-invading breast cancer reveals that circulating tumour cells have a molecular signature indicating specific organ preferences.

The findings, which appear in Cancer Discovery, explain how tumour cells in the blood target a particular organ and may enable the development of treatments to prevent the spread of these metastatic cancers.

In this study breast cancer cells from the blood of breast cancer patients with metastatic tumours were isolated, expanded and grown in the lab.

Analysis of these cells identified regulator genes and proteins within the cells that apparently directed the cancers spread to the brain. The team were therefore able to predict that a patients breast cancer cells would eventually migrate to the brain.

Assistant professor of stem cell and regenerative medicine at the Keck School of Medicine at USC, Min Yu, also discovered that a protein on the surface of these brain-targeting tumour cells helps them to breech the blood brain barrier and lodge in brain tissue, while another protein inside the cells shield them from the brains immune response, enabling them to grow there.

We can imagine someday using the information carried by circulating tumour cells to improve the detection, monitoring and treatment of the spreading cancers, Yu said.

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Even the Trump Organization Doesnt Want to Be Associated With Trump – Vanity Fair

As anyone with even a cursory knowledge of Donald Trumps business history knows, the real estate developer made the bulk of his money licensing his name to businesses he does not own, slapping it on everything from hotels to condos to cologne in the biggest, most garish letters possible. Truly no product was too small or ridiculous for Trump to emblazon, whether it was a fake university, a line of vodka, or steaks sold through the Sharper Image catalog. And while his name never connoted the luxury and class he thought it did, after he kicked off his bid for the presidency in 2015, it suddenly evoked racism, debatable mental stability, and questionable moral conduct, leading a number of hotels and condos around the world to try to remove it from their buildings, both out of disgust and a genuine belief that it was costing them money. Where licensing deals had lapsed, there was nothing the Trump Organization could do about such decisions. But where they remained in place, the presidents family business, run by his two adult sons, fought the tenants and investors tooth and nail. Which makes the news that the Trump Organization has chosen to hide the Trump name from one of its marquee properties all the more humiliating.

The Washington Post reports that at the Wollman Rink and Laser Rink in Central Park, which Trump has run since the 1980s, Trump Organization employees have been removing the five-letter name from sight, taking it off the boards around each rink where giant red TRUMP signs once surrounded customers and off of the desk where visitors rent skates, now covered in a large white tarp until it can be permanently taken away. Its a complete rebranding, Geoffrey Croft, of NYC Park Advocates, told the Post. Theyve taken [the name] off everything. Off the uniforms, everything. (The Trump Organization did not respond to the Posts request for comment.)

And while some might assume that the citywhere the president is deeply unpopularwas behind the move, a spokeswoman said it was the companys decision. The Trump Organization notified us in late August that they planned to change the on-rink branding, said Crystal Howard, a spokeswoman for the city parks department. While Howard said the Trump Organization did not explain its decision, an employee who spoke to the Post hazarded a guess: Even the presidents business knows hes bad for business. I do believe thats the answer. It was hurting business, the employee said. A lot of the schools, you know, liberal private schools up here, come to parties up here. That was a big income earner up here Monday and Tuesday night. (In early 2017, a skating party for students from the Dalton School was canceled after some parents refused to let their children go to a rink with Trumps name on it.)

And in other not-great news for noted narcissists:

Support for the impeachment inquiry into President Trump has reached a new high, a Quinnipiac University poll released Wednesday found. Pollsters discovered that 55% of respondents approved of the inquiry, while 43% opposed it. Last week 51% supported it while 45% did not.... Almost half of all respondents48%backed impeachment and removal of Trump, up 2 percentage points from last week. Forty-six percent said they are now against impeaching and removing him.

Earlier this month the president bragged that only 25% of Americans wanted him impeached, so hell have to update his models.

How one industry is bleeding Wall Street dry of talent Ronan Farrows producer reveals how NBC killed its Weinstein story Ivankas $360 million deal is raising eyebrows at the FBI The big turn for Elizabeth Warrens campaign Why a leading neurocriminologist left Joker completely stunned The Fox News movies uncanny depictions of the networks drama From the Archive: The real-life story of the security guard turned bombing suspect at the heart of Clint Eastwoods latest movie

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Even the Trump Organization Doesnt Want to Be Associated With Trump - Vanity Fair

Pastor Warns If Trump Is Removed From Office, ‘Guys That Know How to Fight’ Will Hunt Down Democrats – Newsweek

Christian church leader Rick Wiles threatened that "There's gonna be violence in America" if President Donald Trump is removed from office.

Wiles, the senior pastor at Flowing Streams Church in Florida, made the remarks on his right-wing TruNews program Tuesday evening. He claimed that cowboys, mountain men and "guys that know how to do violence" would start attacking and "hunting down" Democrats.

"If they take him [Trump] out, there's gonna be violence in America," the religious leader said. "That's all there is to it," he asserted.

"However he leaves, there's gonna be violence in America," Wiles went on. "I believe there are people in this country, veterans, there are cowboys, mountain men, I mean guys that know how to fight," he said, "and they're going to make a decision that people who did this to Donald Trump are not gonna get away with it."

"And they're gonna hunt them down," the pastor said.

"The Trump supporters are going to hunt them down," he added. "It's going to happen and this country is going to be plunged into darkness and they brought it upon themselves because they won't back off."

Wiles' threat came as it has appeared increasingly likely that Trump will be impeached by the Democrat-controlled House of Representatives. The fast-moving impeachment inquiry, which was launched at the end of September, has already revealed that the president pressured Ukrainian leaders to investigate his political rivals and allegedly withheld bipartisan approved military aid to the country as a "quid pro quo" to open the probes.

Although it appears likely that Trump will be impeached in the House, most analysts do not believe he will be removed from office by the Republican-controlled Senate. Although a few GOP senators have expressed serious concerns about Trump's actions towards Ukraine, a two-thirds majority of the legislative body is required to remove the president from office. That would mean all the 45 Democrats, the body's two independents and 19 Republicans would need to vote for Trump's ouster.

Despite it remaining unlikely that 19 or more Republican senators will turn on Trump, a survey by the conservative Daily Caller website suggested this week that it's not outside the realm of possibility. Of the 53 Republicans in the upper house of Congress, only seven confirmed definitively that they do not support Trump's impeachment and removal from office. Additionally, 22 of the lawmakers declined to comment altogether.

Wiles and his TruNews program have a history of promoting highly controversial, anti-Semitic and anti-LGBTQ ideas and conspiracy theories. In 2017, the conservative religious figure argued that Hurricane Harvey's devastation in Texas resulted from Houston supporting the LGBTQ community. "Here's a city that has boasted of its LGBT devotion, its affinity for the sexual perversion movement in America. They're under water," he said at the time.

TruNews has also described former President Barack Obama as a "demon from hell."

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Pastor Warns If Trump Is Removed From Office, 'Guys That Know How to Fight' Will Hunt Down Democrats - Newsweek

Corden: Trump may have just alienated his three black voters – USA TODAY

The comics take a look at the latest in impeachment proceedings, including outrageous tweets, in Best of Late Night Eileen Rivers, USA TODAY Opinion

Impeachment proceedings appear to bereaching a critical point: Summariesof Tuesday's testimony from Bill Taylor, the country'ssenior diplomat in Ukraine, revealed cover ups, quid pro quoand bullying not just from the president, but throughout the administration. If ever testimony solidified the need for an impeachment vote, Taylor delivered it. Hard to imagine things getting much worse, right?

Enter President Donald Trump's Twitter account.

His go-to platform during times of stressincluded a new way of describing the impeachment proceedings. He called the thoroughly constitutional look intohis unseemly (and potentially illegal) activity, a "lynching."

Comedian and writer for "Late Night with Seth Meyers" Amber Ruffin shows why impeachmentproceedings are the furthest thing from a lynching and why calling Trump out on his racism is the furthest thing froma distraction. Take a look at today's Best of Late Night, above, for the deets.

James Corden warned Trump that he was on his way to losingwhat little black support he has.

James Corden(Photo: Robert Hanashiro, USA TODAY)

And the comics fill you in on the other outrageous tweetattached to the president's account.

After you watch our favorite jokes from last night's late-night lineup, vote for yours in the poll below.

Follow Eileen Rivers on Twitter @msdc14.

Read or Share this story: https://www.usatoday.com/story/opinion/best-of-late-night/2019/10/23/corden-meyers-ruffin-fallon-on-trump-and-impeachment-lynching/4072144002/

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Corden: Trump may have just alienated his three black voters - USA TODAY

The Fantasy of Republicans Ditching Trump – POLITICO

Getty Images

Opinion

By RICH LOWRY

October 24, 2019

Rich Lowry is editor of National Review and a contributing editor with Politico Magazine.

Republican senators will soon be receiving an invitation to tear apart the Republican Party ahead of the 2020 elections, and they are going to decline to accept it.

Its a trope of pro-impeachment commentary that it should be simple for Republican senators to swap out President Donald Trump, who puts them in an awkward position every day, for Vice President Mike Pence, an upstanding Reagan conservative who could start with a fresh slate in the runup to the 2020 election.

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This ideas only flaw is that is entirely removed from reality.

If Senate Republicans vote to remove Trump on anything like the current facts, even the worst possible interpretation of them, it would leave the GOP a smoldering ruin. It wouldnt matter who the Democrats nominated for 2020. They could run Bernie Sanders on a ticket balanced by Elizabeth Warren and promise to make Alexandria Ocasio-Cortez secretary of the Treasury and Ilhan Omar secretary of Defense, and theyd still win.

A significant portion of the Republican Party would consider a Senate conviction of Trump a dastardly betrayal. Perhaps most would get over it, as partisan feelings kicked in around a national election, but not all. And so a party that has managed to win the popular vote in a presidential election only once since 1988 would hurtle toward November 2020 divided.

How does anyone think that would turn out?

A lot of Trump supporters are going to want to blame the Republican establishment even if Trump loses in 2020 fair and square, with the backing of the united party apparatus. Imagine what they will think if a couple of dozen Republican senators decide to deny him the opportunity to run for reelection, without a single Republican voter having a say on his ultimate fate. Its hard to come up with any scenario better designed to stoke the populist furies of Trumps most devoted voters.

Trump himself isnt going to get convicted by the Senate and say, Well, Im a little disappointed in your judgment to be honest. But it was a close call, and Mike Pence is a great guy, and Im just grateful I had the opportunity to serve this country in the White House for more than three years.

He wont go away quietly to lick his wounds. He wont delete his Twitter account. He wont make it easy on anyone. He will vent his anger and resentment at every opportunity. It will be human scum every single day.

And its not as though the media is going to lose its interest in the most luridly telegenic politician that weve ever seen. The mainstream press would be delighted to see Trump destroyed, yet sad to bid him farewell. The obvious way to square the circle would be to continue to give Trump lavish coverage in his post-presidency. Hed be out of the White House but still driving screaming CNN chyrons every other hour.

In other words, Trumps removal wouldnt be a fresh start for Pence and the GOP in an accelerated post-Trump era; it would be more like getting stuck in the poisonous epilogue of the Trump era, awaiting the inevitable advent of the Elizabeth Warren, Joe Biden or Pete Buttigieg era.

All of this is why the cracks in the Republican Senate coverage is so ridiculous and overwrought. It depends on the idea that GOP senators who, it is true, are continually frustrated by Trumps controversies are on the verge of engineering their partys own destruction. There was even some cracks showing analysis around Mitch McConnell saying the other day that he didnt remember a conversation with Trump, as recounted by the president, praising his call with the Ukrainian president. The obvious explanation is that McConnell really didnt recall such a conversation, not that the shrewdest, most realistic politician in Washington was getting ready to immolate himself and his party as soon as the articles of impeachment arrive from the House.

Mitt Romney has gotten a lot of coverage for his excoriating comments about the Ukraine mess and the Syria pullout. He really might vote to convict when it comes to it, but hes not a broad indicator of the direction of the party. As goes Romney on impeachment ... so goes Romney on impeachment.

Its possible to come up with a scenario in which Ukraine developments are much worse than its possible to imagine right now, and Trumps support craters, even among Republicans. Then, you might have GOP senators voting to convict. This is just another path to the destruction of the partys hopes in 2020, though, because theres no way it would snap back from a Nixonian meltdown at the top in less than a year.

In short, Mike Pence might be elected president one day, but its not going to be while presiding over a party that has just jettisoned Donald Trump.

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The Fantasy of Republicans Ditching Trump - POLITICO

A lynching is something Donald Trump will never know – The Boston Globe

A lynching isnt something you can win. Emmett Till was lynched. He was 14 years old.

In 1955, Till was a Chicago teen visiting family in Mississippi. A white woman claimed he grabbed her, whistled at her, and made crude comments. She lied. Her husband and his friend kidnapped him, tortured him beyond recognition, and shot him in the head.

They used barbed wire to tie his body to a cotton gin fan and threw him into the Tallahatchie River. His killers were acquitted by an all-white jury.

What happened to Till? That is a lynching. Last Saturday, the Emmett Till Memorial Commission unveiled the fourth marker dedicated to him since 2008, when the original sign was stolen. The signs have been taken, vandalized, and shot at over and over.

The latest memorial is bulletproof. Sixty-four years after his murder, racists dont want us to remember Till and the racist violence that eats at the soul of America.

Not enough of us learn about the true history of the country in school. When Watchmen debuted on HBO Sunday night, it opened with the Greenwood massacre, the terrorist attack of a Tulsa neighborhood known as Black Wall Street, where there were 300 black-owned businesses.

The scenes show black people murdered in the street, businesses on fire, and a plane dropping a bomb. This is not TV exaggeration. This happened in America. And it was clear from social media Sunday night and all day Monday that thousands of viewers had never heard of what is referred to as the Tulsa Race Riot.

Its been almost 100 years, and this month, archeologists are searching Tulsa for evidence of mass graves. At least 300 black men, women, and children were murdered; 10,000 were displaced. And it took a brilliant television show to get the country talking about it.

Be clear. There was no riot. It was terrorism inflicted on black people by white supremacists, the kind of people Trump likes to believe are very fine. Trump is the same man who called Black Lives Matter activist Mercutio Southall Jr. disgusting and said he deserved to be roughed up by Trump supporters at a rally in November 2015.

The delight he takes in the pain of black people, the way he encouraged the country to turn against football players protesting police brutality and oppression this is the way of supremacy. Racists used to smile and pose for pictures during lynchings.

Trump believes in the lynching of black people. In 1989, he spent some $85,000 on full-page ads to bring back the death penalty to execute Korey Wise, Antron McCray, Yusef Salaam, Raymond Santana, and Kevin Richardson. Teenagers. Five black and brown boys wrongfully accused of raping a white woman. He wanted The Central Park Five, now known as The Exonerated Five, dead. He didnt care about proof. He just wanted murder.

Last year, the National Memorial for Peace and Justice opened in Alabama. The site includes 800 6-foot monuments to symbolize thousands of racial terror lynching victims the Equal Justice Initiative researchers documented 4,075 victims between 1877 and 1950.

It is the nations first memorial dedicated to the legacy of enslaved black people, people terrorized by lynching, racial segregation, and Jim Crow. People who even in 2019 are considered a threat upon sight, their skin criminalized.

Earlier this month, Atatiana Jefferson was killed by a police officer who was supposed to be performing a safety check. Black people are not safe in this country. But Trump considers his impeachment inquiry a lynching.

People say its a distraction and we shouldnt pay attention. Except we must. This is a man who encourages hate. His own supporters happily wear shirts that read, Rope. Tree. Journalist. Some Assembly Required.

Two years ago, when I was a Kansas City Star columnist, I was swarmed with e-mails, phone calls, and letters laced with racist rhetoric and Trump support. The n-word was the norm in my inbox. The words tar and feather were a cheer.

You are exactly why tall trees and ropes were made, a reader named Jeff from Montana wrote to me.

Any time I wrote about Trump or the NFL, or protested police brutality, I would get this kind of violent response. My life was threatened twice. One reader thought I should be tied to the back of a truck and dragged out of town.

This is not shocking. I am not an isolated incident. What Ive experienced is subtle compared with what those whove come before me endured.

Ida B. Wells risked her life as an investigative journalist a black woman, traveling the country and exposing the horrors of racism and lynching.

Our countrys national crime is lynching. It is not the creature of an hour, the sudden outburst of uncontrolled fury, or the unspeakable brutality of an insane mob, she said in a speech in 1900.

It represents the cool, calculating deliberation of intelligent people who openly avow that there is an unwritten law that justifies them in putting human beings to death without complaint under oath, without trial by jury, without opportunity to make defense, and without right of appeal.

Black men were murdered for looking white men in the eye. Black women were raped by white men as punishment. We like to think of these things as Americas tragic history.

Except black people are still dying. They are still being tortured. They are still being convicted upon sight.

We dont want to remember our racist truth. We want to shoot holes through Americas reality the way supremacists tried to destroy the memory of Emmett Till.

Its easier to be retroactively heartbroken while learning of American tragedy and being entertained on HBO than it is to recognize the truth of right now.

Lynching was real. It happened. And the effects of lynch culture live on today.

Police brutality, the school-to-prison pipeline, segregated schools, excessive sentencing, and the continued racial inequity are all rooted in racism and the supremacist mentality that believed lynching was some sort of national justice.

Trump was right. Were witnessing a lynching. But it is not his.

Jene Osterheldt can be reached at jenee.osterheldt@globe.com. Follow her on Twitter @sincerelyjenee.

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A lynching is something Donald Trump will never know - The Boston Globe