Monthly Archives: May 2020

A new treatment that helps to relieve coronavirus symptoms could be brought to market in three months' time if further trials go well, according to a researcher involved in the project.

"It's very early to say at this stage," said Dr.Fatima al-Kaabi, head of hematology and oncology at the Sheikh Khalifa Medical City in the United Arab Emirates.

"We've been happy that our initial safety results are promising, that's why we're heading into the next phase, of effectiveness of this treatment," she told CNBC's Hadley Gamble on Monday.

"If all ... went well and it worked well, then I would propose ... three months' time," she said, when asked how quickly the treatment, which was developed by doctors and researchersat the Abu Dhabi Stem Cell Center, could reach the market.

To date, there are no known vaccines or specific antiviral medicines against Covid-19.U.S. health officials say developing a vaccine will take at least 12 to 18 months.

The UAE has 14,163 cases and126 deaths due to the coronavirus, based on data from Johns Hopkins University.

The remedy uses a "minimally invasive" method where a Covid-19 patient's stem cells are extracted, activated and turned into a fine mist to be inhaled. This alleviates symptoms such as shortness of breath and possibly coughing, said Dr. al-Kaabi.

"It is hypothesized to have its therapeutic effect by regenerating lung cells and modulating the immune response to keep it from overreacting to the COVID-19 infection and causing further damage to healthy cells,"the UAE's ministry of health and preventionsaid a statement.

Some 73 patients with moderate to severe symptoms received this treatment, and all were "successfully treated and cured," the statement said, adding that none reported "immediate adverse effects." Around a quarter of these patients were intubated and in the intensive care unit.

The treatment was given along with "conventional medical intervention" and will not replace established protocols, according to the statement.

"We're hopeful," said Dr. al-Kaabi, noting that the results of further trials on the efficacy of the treatment will only be out a couple of weeks' time. "We've seen (a) favorable outcome."

Another treatment for the coronavirus, an antiviral drug from Gilead Sciences, has been in the spotlight following positive preliminary results from trials. America's Food and DrugAdministration granted the medicine emergency use authorization last week. That means doctors can administerremdesivir to patients hospitalized with Covid-19, even though the drug has not undergone the same FDA review as other treatments.

Separately, researchers cut short a study testing anti-malaria drug chloroquine as a potential Covid-19 treatment last month. The drug gained widespread international attention after two small studies published in France found the coronavirus infection cleared a lot faster for patients taking it when compared to a control group.

However, citing a high risk of death, scientists have now scrapped the trials, warning it should prompt some degree of skepticism from the public toward enthusiastic claims of the drug. President Donald Trump had touted chloroquine as a potential "game changer" in the fight against the virus.

CNBC'sBerkeley Lovelace Jr. andWilliam Feuer contributed to this report.

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Stem cell treatment in the UAE sees 'favorable' outcomes for coronavirus patients - CNBC

CARLSBAD, Calif.--(BUSINESS WIRE)--Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, today announced that updated results from a Phase I/IIa study of its lead product candidate, OpRegen, a retinal pigment epithelium (RPE) cell transplant therapy currently in development for the treatment of dry age-related macular degeneration (AMD), were published online via the ARVOLearn platform as part of the 2020 Association for Research in Vision and Ophthalmology (ARVO) Meeting. The presentation entitled, Phase I/IIa Clinical Trial of Human Embryonic Stem Cell (hESC)-Derived Retinal Pigmented Epithelium (RPE, OpRegen) Transplantation in Advanced Dry Form Age-Related Macular Degeneration (AMD): Interim Results (Abstract # 3363764), was presented by Christopher D. Riemann, M.D., Vitreoretinal Surgeon and Fellowship Director, Cincinnati Eye Institute (CEI) and University of Cincinnati School of Medicine. Dr. Riemanns presentation is available on the Media page of the Lineage website. Lineage will also host a live call with Dr. Riemann, on Monday, May 11, 2020 at 5:00 p.m. ET/2:00 p.m. PT to further discuss the results of treatment with OpRegen. Interested parties can access the call on the Events and Presentations section of Lineages website.

This update is significant as it builds on our earlier reports of gains in visual acuity and provides a more comprehensive picture of treatment with OpRegen for dry AMD, with meaningful improvements in the progression of geographic atrophy, visual acuity, and reading speed observed in our first Cohort 4 patient and first Orbit SDS with thaw-and-inject formulation dosed patient, stated Brian M. Culley, Lineage CEO. As dry AMD is a slow and progressive disease, it takes many months to observe changes to retinal anatomy or visual acuity. With the benefit of longer follow-up, we now can report that some OpRegen treated patients are able to see better, have less growth in their area of GA, and are able to read faster, all of which represent significant enhancements to vision and quality of life metrics. In addition to these individual results, the pooled data continues to suggest a treatment effect in both visual acuity and GA progression. Notably, we also are reporting additional evidence that OpRegen cells remain present for at least 4 years and hope that longer follow-up periods will reinforce a growing body of evidence that OpRegen is well-tolerated and can provide sustained and clinically meaningful benefits with a single dose of RPE cells. Our near-term objective is to treat and monitor the final four patients in Cohort 4 of the current study and utilize these data to direct our clinical, regulatory, and partnership discussions. Our goal is to combine the best cell line, the best production process, and the best delivery system, to position OpRegen as the front-runner in the race to address the unmet need in the potential billion-dollar dry AMD market.

As a principal investigator on the OpRegen clinical study, I am excited to present this most recent update, where all Cohort 4 patients treated with OpRegen had improved Best Corrected Visual Acuity up to one year or at their last visit, demonstrating a substantial treatment response, stated Christopher D. Riemann, M.D. The pooled Cohort 4 data demonstrate a significant, greater than 10-letter sustained visual acuity improvement over the entire followup period. Reading center assessments of GA also suggest a reduction in GA progression in the OpRegen treated eye when compared to fellow eye in Cohort 4. I am encouraged by the results observed in patients treated to date with OpRegen and I look forward to dosing patients in this study at CEI.

KOL Call Information and Webcast

Lineage will host a conference call with Dr. Riemann, on Monday, May 11, 2020 at 5:00 p.m. ET/2:00 p.m. PT to further discuss the results following treatment with OpRegen. A live webcast of the conference call will be available online in the Events and Presentations section of Lineages website. Interested parties may also access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the Lineage Cell Therapeutics Call. A replay of the webcast will be available on Lineages website for 30 days and a telephone replay will be available through May 19, 2020, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 6597936.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical programs are in markets with billion dollar opportunities and include three allogeneic (off-the-shelf) product candidates: (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; and (iii) VAC2, a cancer immunotherapy of antigen-presenting dendritic cells in Phase 1 development for the treatment of non-small cell lung cancer. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as believe, may, will, estimate, continue, anticipate, design, intend, expect, could, plan, potential, predict, seek, should, would, contemplate, project, target, tend to, or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to Lineages objectives with respect to OpRegen. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks in Lineages filings with the Securities and Exchange Commission (the SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading Risk Factors in Lineages periodic reports with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 12, 2020 and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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Lineage Cell Therapeutics Reports New Data With OpRegen for the Treatment of Dry AMD With Geographic Atrophy - Business Wire

CRANBURY, N.J., May 06, 2020 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel medicines for rare diseases today announced the acceptance of several abstracts for presentation at the American Society of Gene & Cell Therapy 23rd Annual Meeting being held virtually on May 12 15. Preclinical data from its Pompe gene therapy program, which Amicus is developing with the Gene Therapy Program of the Perelman School of Medicine at the University of Pennsylvania, will be presented as an oral presentation. Preclinical data related to the CLN6 and CLN8 Batten disease programs, with our partners at Sanford Research and Nationwide Childrens Hospital, will be presented in respective posters.

Oral Platform Presentation: Thursday, May 14, 2020,4:45-5:00p.m. ET

Pompe Disease:

Poster Session: Tuesday, May 12, 2020, 5:30-6:30 p.m. ET

CLN6 Batten Disease:

Poster Session: Wednesday, May 13, 2020, 5:30-6:30 p.m. ET

CLN8 Batten Disease:

All abstracts for the American Society of Gene & Cell Therapy 23rd Annual Meeting are now available online.

About Pompe DiseasePompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA leads to accumulation of glycogen in cells, which results in the clinical manifestations of Pompe disease. The disease can be debilitating and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.

About Batten DiseaseBatten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs. In these diseases, a defect in a specific gene triggers a cascade of problems that interferes with a cells ability to recycle certain molecules. Each gene is called CLN (ceroid lipofuscinosis, neuronal) and given a different number designation as its subtype. There are 13 known forms of Batten disease often referred to as CLN1-8; 10-14. The various types of Batten disease have similar features and symptoms but vary in severity and age of onset.

Most forms of Batten disease/NCLs usually begin during childhood. The clinical course often involves progressive loss of independent adaptive skills such as mobility, feeding, and communication. Patients may also experience vision loss, personality changes, behavioral problems, learning impairment, and seizures. Patients typically experience progressive loss of motor function and eventually become wheelchair-bound, are then bedridden, and die prematurely.

About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the companys website at http://www.amicusrx.com, and follow us on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans or projections will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we may not be able to manufacture or supply sufficient clinical products; and the potential that we will need additional funding to complete all of our studies and manufacturing. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2019. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this press release to reflect events or circumstances after the date hereof.

CONTACTS:

Investors/Media:Amicus TherapeuticsAndrew FaughnanDirector, Investor Relationsafaughnan@amicusrx.com(609) 662-3809

FOLDG

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UPDATE Amicus Therapeutics Announces Upcoming Presentations at the American Society of Gene & Cell Therapy 23rd Annual Meeting - GlobeNewswire

Dublin, May 05, 2020 (GLOBE NEWSWIRE) -- The "Cell Expansion Market to 2027 - Global Analysis and Forecasts By Product; Cell Type; Application; End User, and Geography" report has been added to ResearchAndMarkets.com's offering.

The global cell expansion market is projected to reach US$ 42,837.11 Mn in 2027 from US$ 11,929.43 Mn in 2018. The cell expansion market is expected to grow with a CAGR of 15.6% from 2019-2027.

Driving factors include increasing adoption of regenerative medicines, rising prevalence of cancer. However, the risk contamination during cell expansion is expected to hamper the market during the forecast period.

Cancer is one of the major cause of human death worldwide. In recent years, the cases of cancer have been increasing tremendously and the trend is anticipated to remain the same in the upcoming years. According to the World Health Organization in 2018, approximately 9.6 million deaths across the globe were due to cancer. Furthermore, the National Cancer Institute predicted that in 2018, approximately 1,735,350 new cancer cases would be diagnosed in the US.

Changes in lifestyle have resulted in more exposure to oncogenic factors. Cancer can be cured if diagnosed and treated at an initial stage. Cancer sequencing using next-generation sequencing (NGS) methods provides more information. Additionally, cell expansion related procedures also aids in research, diagnostics and treatment of cancer.

Furthermore, Asia Pacific region is also facing the problem of the growing prevalence of cancer. The top 15 countries with Cancer prevalence are Japan, Taiwan, Singapore, South Korea, Malaysia, Thailand, China, Philippines, Sri Lanka, Vietnam, Indonesia, Mongolia, India, Laos, and Cambodia. According to the National Institute of Cancer Prevention and Research (NICPR), in 2018, in India, total deaths due to cancer were 784,821.

The global Cell Expansion market is segmented by product, cell type, application, end user. Based on product, the cell expansion market is segmented into consumables and instruments. In 2018, the consumables accounted for the largest market share in the global cell expansion market by product. These consumables are essential components of any laboratory experiment hence they are expected to witness significant growth during the forecast period. Based on cell type, the cell expansion market has been segmented into human cell and animal cell. Furthermore based on application the cell expansion market has been segmented into Regenerative Medicine And Stem Cell Research, Cancer And Cell-Based Research and Other Applications. Based in end user market is segmented into Biopharmaceutical And Biotechnology Companies, Research Institutes, cell banks and others.

Some of the essential primary and secondary sources included in the report are the National Institute of Cancer Prevention and Research (NICPR), Association for Management Education and Development, Center for Cancer Research, International Society for Stem Cell Research (ISSCR), American Association of Blood Banks (AABB), National Institute of Cancer Prevention and Research and others.

Reasons to Buy

Key Topics Covered:

1. Introduction

2. Cell Expansion Market - Key Takeaways

3. Research Methodology

4. Cell Expansion- Market Landscape4.1 Overview4.2 PEST Analysis4.3 Expert Opinions

5. Global Cell Expansion Market - Key Market Dynamics5.1 Key Market Drivers5.1.1 Increasing Adoption of Regenerative Medicines5.1.2 Rising Prevalence of Cancer5.2 Key Restraints5.2.1 Risk Contamination During Cell Expansion5.3 Key Opportunity5.3.1 Middle Income Countries Creating Development Opportunities5.4 Future Trend5.4.1 Consistent Research in Drug Discovery Activities5.5 Impact Analysis

6. Cell Expansion Market - Global Analysis6.1 Global Cell Expansion Market Revenue Forecasts And Analysis6.2 Global Cell Expansion Market, By Geography - Forecasts And Analysis6.3 Market Positioning Of Key Players

7. Cell Expansion Market - Revenue And Forecasts To 2027 - Product7.1 Overview7.2 Global Cell Expansion Market, by Product , 2018 & 2027 (% Share)7.3 Consumables7.3.1 Overview7.3.2 Global Consumables Market Revenue and Forecast to 2027 (US$ Mn)7.3.3 Reagents, Media & Serum7.3.3.1 Overview7.3.3.2 Global Reagents, Media & Serum Market Revenue and Forecast to 2027 (US$ Mn)7.3.4 Disposables7.3.4.1 Overview7.3.4.2 Global Disposables Market Revenue and Forecast to 2027 (US$ Mn)7.3.4.3 Culture Tissue Flasks7.3.4.3.1 Overview7.3.4.3.2 Global Culture Tissue Flasks Market Revenue and Forecast to 2027 (US$ Mn)7.3.4.4 Bioreactor Accessories7.3.4.4.1 Overview7.3.4.4.2 Global Bioreactor Accessories Market Revenue and Forecast to 2027 (US$ Mn)7.3.4.5 Other Disposables7.3.4.5.1 Overview7.3.4.5.2 Global Other Disposables Market Revenue and Forecast to 2027 (US$ Mn)7.4 Instruments7.4.1 Overview7.4.2 Global Instruments Market Revenue and Forecast to 2027 (US$ Mn)7.4.3 Cell Expansion Supporting Equipment7.4.3.1 Overview7.4.3.2 Global Cell Expansion Supporting Equipment Market Revenue and Forecast to 2027 (US$ Mn)7.4.4 Bioreactors7.4.4.1 Overview7.4.4.2 Global Bioreactors Market Revenue and Forecast to 2027 (US$ Mn)7.4.5 Automated Cell Expansion Systems7.4.5.1 Overview7.4.5.2 Global Automated Cell Expansion Systems Market Revenue and Forecast to 2027 (US$ Mn)

8. Cell Expansion Market Analysis and Forecasts to 2027 - Cell Type8.1 Overview8.2 Global Cell Expansion Market, by Cell Type, 2018 & 2027 (% Share)8.3 Human Cells8.3.1 Overview8.3.2 Global Human Cell Market Revenue and Forecast to 2027 (US$ Mn)8.3.3 Adult Stem Cells8.3.3.1 Overview8.3.3.2 Global Adult Stem Cells Market Revenue and Forecast to 2027 (US$ Mn)8.3.4 Induced Pluripotent Stem Cells8.3.4.1 Overview8.3.4.2 Global Induced Pluripotent Stem Cells Market Revenue and Forecast to 2027 (US$ Mn)8.3.5 Embryonic Stem Cells8.3.5.1 Overview8.3.5.2 Global Embryonic Stem Cells Market Revenue and Forecast to 2027 (US$ Mn)8.3.6 Differentiated Cells8.3.6.1 Overview8.3.6.2 Global Differentiated Cells Market Revenue and Forecast to 2027 (US$ Mn)8.4 Animal Cells8.4.1 Overview8.4.2 Global Animal Cell Market Revenue and Forecast to 2027 (US$ Mn)

9. Cell Expansion Market Analysis And Forecasts To 2027 - Application9.1 Overview9.2 Global Cell Expansion Market Share by Application 2018 & 2027 (%)9.3 Regenerative Medicine And Stem Cell Research9.3.1 Overview9.3.2 Global Regenerative Medicine And Stem Cell Research Market Revenue and Forecast to 2027 (US$ Mn)9.4 Cancer And Cell-Based Research9.4.1 Overview9.4.2 Global Cancer And Cell-Based research Market Revenue and Forecast to 2027 (US$ Mn)9.5 Other Applications9.5.1 Overview9.5.2 Global Other Applications Market Revenue and Forecast to 2027 (US$ Mn)

10. Cell Expansion Market Analysis And Forecasts To 2027 - End User10.1 Overview10.2 Global Cell Expansion Market Share by End User 2018 & 2027 (%)10.3 Biopharmaceutical And Biotechnology Companies10.3.1 Overview10.3.2 Global Biopharmaceutical And Biotechnology Companies Market Revenue and Forecast to 2027 (US$ Mn)10.4 Research Institutes10.4.1 Overview10.4.2 Global Research Institutes Market Revenue and Forecast to 2027 (US$ Mn)10.5 Cell Banks10.5.1 Overview10.5.2 Global Cell Banks Market Revenue and Forecast to 2027 (US$ Mn)10.6 Other End Users10.6.1 Overview10.6.2 Global Other End Users Market Revenue and Forecast to 2027 (US$ Mn)

11. Cell Expansion Market - Geographic Analysis11.1 North America Cell Expansion Market, Revenue and Forecast to 202711.2 Europe Cell Expansion Market, Revenue and Forecast to 202711.3 APAC Cell Expansion Market, Revenue and Forecast to 202711.4 MEA Cell Expansion Market, Revenue and Forecast to 202711.5 South and Central America Cell Expansion Market, Revenue and Forecast to 2027

12. Cell Expansion Market - Industry Landscape12.1 Overview12.2 Growth Strategies In The Cell Expansion Market, 2017-201912.3 Organic Growth Strategies12.3.1 Overview12.3.1.1 Recent Organic Developments By Players In The Cell Expansion Market12.4 Inorganic Growth Strategies12.4.1 Overview12.4.2 Recent Developments By Players In The Cell Expansion Market

13. Global Cell Expansion Market-Key Company Profiles13.1 BD13.1.1 Key Facts13.1.2 Business Description13.1.3 Financial Overview13.1.4 Product Portfolio13.1.5 SWOT Analysis13.1.6 Key Developments13.2 Merck KGaA13.3 Thermo Fisher Scientific, Inc.13.4 Terumo Corporation13.5 General Electric Company13.6 Corning Incorporated13.7 Miltenyi Biotec13.8 Danaher13.9 Lonza13.10 STEMCELL Technologies, Inc.

14. Appendix14.1 About the Publisher14.2 Glossary Of Terms

For more information about this report visit https://www.researchandmarkets.com/r/hjxwqh

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Insights on the Worldwide Cell Expansion Industry to 2027 - Analysis and Forecasts - GlobeNewswire

Press Release

6 May 2020

Immunicum AB (publ) Receives Regenerative Medicine Advanced Therapy Designation from FDA for Ilixadencel in Kidney Cancer

Immunicum AB (publ; IMMU.ST) announced today that it has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for the Companys lead candidate, ilixadencel, a cell-based, off-the-shelf immune primer for the treatment of metastatic Renal Cell Carcinoma (mRCC). The FDAs decision was made based on the previously communicated results from the Phase II MERECA clinical trial that evaluated the safety and efficacy of ilixadencel in combination with Sutent (sunitinib) in patients with newly diagnosed mRCC. Advantages of the RMAT designation include all the benefits of the Fast Track and Breakthrough Therapy Designation programs including guidance and early interactions with the FDA to discuss potential surrogate or intermediate endpoints to support accelerated approval as well as potential ways to satisfy post-approval requirements.

We are very excited to have received the RMAT designation for ilixadencel in kidney cancer as it recognizes both the potential of our novel therapeutic approach as well as the clear need for viable therapies to address this difficult-to treat disease. As a designation similar to the FDAs Breakthrough Therapy Designation, we will now also have the opportunity to receive direct guidance from the FDA which will inform key development decisions and ultimately bring us closer to delivering ilixadencel to patients in need, commented Alex Karlsson-Parra, CEO of Immunicum.

Established in 2017 under the 21st Century Cures Act in the United States, RMAT designation is an expedited program designed to facilitate the development and review of regenerative medicine therapies intended to address an unmet medical need in patients with serious conditions. An investigational regenerative medicine therapy (e.g. cell or gene therapy) is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious condition and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such a disease or condition. As a cell therapy medicinal product, ilixadencel falls within the definition of a regenerative medicine therapy.

The latest results of the Phase II MERECA trial were presented in February at the ASCO-SITC Clinical Immuno-Oncology Symposium 2020 in Orlando, Florida. As of December 2019, the patient follow up data indicates a separation in Kaplan-Meier survival curves in favor of the ilixadencel treatment group in line with the projected separation based on the data from July 2019. The median OS value could not be calculated yet in either group as the data is not mature. The confirmed ORR for the ilixadencel treatment group was 42.2% (19/45) versus 24.0% (6/25) for the sunitinib control group.

Refer to the following link for more information on Regenerative Medicine Advanced Therapy Designation: https://bit.ly/3c7bFah

About MERECAMERECA is an exploratory, international, randomized, controlled and open-label Phase II clinical trial in which a total of 88 newly diagnosed, intermediate and poor-prognosis metastatic renal cancer patients were enrolled. Based on a 2-to-1 randomization, patients received either two intratumoral doses of ilixadencel before nephrectomy (surgical removal of the tumor-affected kidney) and subsequent treatment with sunitinib or sunitinib therapy alone post-nephrectomy. The primary objectives of the study are to evaluate median OS and 18-month survival rates. Secondary objectives include evaluation of safety and tolerability, tumor response and immunological profiling including T cell infiltration.

About renal cell cancer / carcinomaThere are approximately 273,000 new cases of Renal Cell Cancer diagnosed worldwide each year, representing approximately two percent of all cancers. The therapeutic effect of existing treatments, called targeted therapies, is often of short duration, with limited survival gain. With no alternatives to these therapies, there exists a relatively large unsatisfied medical need for new treatments that are effective, more cost-efficient and have less unwanted side effects.

About ilixadencelIlixadencel is an off-the-shelf cell-based cancer immunotherapy developed for the treatment of solid tumors. Its active ingredient is activated allogeneic dendritic cells, derived from healthy blood donors. Injection of these cells in the patients tumor generates an inflammatory response which in turns leads to tumor-specific activation of the patients cytotoxic T cells. To-date ilixadencel has been tested in a range of clinical trials in various solid tumor indications including metastatic Renal Cell Carcinoma (mRCC), hepatocellular carcinoma (HCC) and gastrointestinal stromal tumors (GIST) and in combination with several standard-of-care cancer therapies such as the tyrosine kinase inhibitors Sutent (sunitinib) and Stivarga (regorafenib), and the checkpoint inhibitor Keytruda (pembrolizumab). Ilixadencel has consistently maintained a positive safety and tolerability profile and demonstrated initial signs of efficacy as seen in the randomized Phase II MERECA trial. Ilixadencel is currently moving towards late-stage clinical development.

The information is such information that Immunicum is obliged to make public pursuant to EU Market Abuse Regulation. The information was released for public disclosure through the contact persons detailed below on 6 May 2020 at 8:00 am CET.

For more information, please contact:

Alex Karlsson-Parra, CSO and Interim CEO, ImmunicumTelephone: +46 8 732 8400E-mail: info@immunicum.com

Sijme Zeilemaker, COO, ImmunicumTelephone: +46 8 732 8400E-mail: info@immunicum.com

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Immunicum AB (publ) Receives Regenerative Medicine Advanced Therapy Designation from FDA for Ilixadencel in Kidney Cancer - GlobeNewswire

The novel coronavirus causing COVID-19 seems to hit some people harder than others, with some people experiencing only mild symptoms and others being hospitalized and requiring ventilation. Though scientists at first thought age was the dominant factor, with young people avoiding the worst outcomes, new research has revealed a suite of features impacting disease severity. These influences could explain why some perfectly healthy 20-year-old with the disease is in dire straits, while an older 70-year-old dodges the need for critical interventions.

These risk factors include:AgeDiabetes (type 1 and type 2)Heart disease and hypertensionSmokingBlood typeObesityGenetic factors

About 8 out of 10 deaths associated with COVID-19 in the U.S. have occurred in adults ages 65 and older, according to the U.S. Centers for Disease Control and Prevention (CDC). The risk of dying from the infection, and the likelihood of requiring hospitalization or intensive medical care, increases significantly with age. For instance, adults ages 65-84 make up an estimated 4-11% of COVID-19 deaths in the U.S, while adults ages 85 and above make up 10-27%.

The trend may be due, in part, to the fact that many elderly people have chronic medical conditions, such as heart disease and diabetes, that can exacerbate the symptoms of COVID-19, according to the CDC. The ability of the immune system to fight off pathogens also declines with age, leaving elderly people vulnerable to severe viral infections, Stat News reported.

Related: Coronavirus in the US: Latest COVID-19 news and case counts

Diabetes mellitus a group of diseases that result in harmful high blood sugar levels also seems to be linked to risk of more severe COVID-19 infections.

The most common form in the U.S. is type 2 diabetes, which occurs when the body's cells don't respond to the hormone insulin. As a result, the sugar that would otherwise move from the bloodstream into cells to be used as energy just builds up in the bloodstream. (When the pancreas makes little to no insulin in the first place, the condition is called type 1 diabetes.)

In a review of 13 relevant studies, scientists found that people with diabetes were nearly 3.7 times more likely to have a critical case of COVID-19 or to die from the disease compared with COVID-19 patients without any underlying health conditions (including diabetes, hypertension, heart disease or respiratory disease), they reported online April 23 in the Journal of Infection.

Even so, scientists don't know whether diabetes is directly increasing severity or whether other health conditions that seem to tag along with diabetes, including cardiovascular and kidney conditions, are to blame.

That fits with what researchers have seen with other infections and diabetes. For instance, flu and pneumonia are more common and more serious in older individuals with type 2 diabetes, scientists reported online April 9 in the journal Diabetes Research and Clinical Practice. In a literature search of relevant studies looking at the link between COVID-19 and diabetes, the authors of that paper found a few possible mechanisms to explain why a person with diabetes might fare worse when infected with COVID-19. These mechanisms include: "Chronic inflammation, increased coagulation activity, immune response impairment and potential direct pancreatic damage by SARS-CoV-2."

Related: 13 coronavirus myths busted by science

Mounting research has shown the progression of type 2 diabetes is tied to changes in the body's immune system. This link could also play a role in poorer outcomes in a person with diabetes exposed to SARS-CoV-2, the virus that causes COVID-19.

No research has looked at this particular virus and immune response in patients with diabetes; however, in a study published in 2018 in the Journal of Diabetes Research, scientists found through a review of past research that patients with obesity or diabetes showed immune systems that were out of whack, with an impairment of white blood cells called Natural Killer (NK) cells and B cells, both of which help the body fight off infections. The research also showed that these patients had an increase in the production of inflammatory molecules called cytokines. When the immune system secretes too many cytokines,a so-called "cytokine storm" can erupt and damage the body's organs. Some research has suggested that cytokine storms may be responsible for causing serious complications in people with COVID-19, Live Science previously reported. Overall, type 2 diabetes has been linked with impairment of the very system in the body that helps to fight off infections like COVID-19 and could explain why a person with diabetes is at high risk for a severe infection.

Not all people with type 2 diabetes are at the same risk, though: A study published May 1 in the journal Cell Metabolism found that people with diabetes who keep their blood sugar levels in a tighter range were much less likely to have a severe disease course than those with more fluctuations in their blood sugar levels.

Scientists aren't sure whether this elevated risk of a severe COVID-19 infection also applies to people with type 1 diabetes (T1D). A study coordinated by T1D Exchange a nonprofit research organization focused on therapies for those with type 1 diabetes launched in April to study the outcomes of T1D patients infected with COVID-19. When a person with T1D gets an infection, their blood sugar levels tend to spike to dangerous levels and they can have a buildup of acid in the blood, something called diabetic ketoacidosis. As such, any infection can be dangerous for someone with type 1 diabetes.

People with conditions that affect the cardiovascular system, such as heart disease and hypertension, generally suffer worse complications from COVID-19 than those with no preexisting conditions, according to the American Heart Association. That said, historically healthy people can also suffer heart damage from the viral infection.

The first reported coronavirus death in the U.S., for instance, occurred when the virus somehow damaged a woman's heart muscle, eventually causing it to burst, Live Science reported. The 57-year-old maintained good health and exercised regularly before becoming infected, and she reportedly had a healthy heart of "normal size and weight." A study of COVID-19 patients in Wuhan, China, found that more than 1 in 5 patients developed heart damage some of the sampled patients had existing heart conditions, and some did not.

In seeing these patterns emerge, scientists developed several theories as to why COVID-19 might hurt both damaged hearts and healthy ones, according to a Live Science report.

In one scenario, by attacking the lungs directly, the virus might deplete the body's supply of oxygen to the point that the heart must work harder to pump oxygenated blood through the body. The virus might also attack the heart directly, as cardiac tissue contains angiotensin-converting enzyme 2 (ACE2) a molecule that the virus plugs into to infect cells. In some individuals, COVID-19 can also kickstart an overblown immune response known as a cytokine storm, wherein the body becomes severely inflamed and the heart could suffer damage as a result.

People who smoke cigarettes may be prone to severe COVID-19 infections, meaning they face a heightened risk of developing pneumonia, suffering organ damage and requiring breathing support. A study of more than 1,000 patients in China, published in the New England Journal of Medicine, illustrates this trend: 12.3% of current smokers included in the study were admitted to an ICU, were placed on a ventilator or died, as compared with 4.7% of nonsmokers.

Cigarette smoke might render the body vulnerable to the coronavirus in several ways, according to a recent Live Science report. At baseline, smokers may be vulnerable to catching viral infections because smoke exposure dampens the immune system over time, damages tissues of the respiratory tract and triggers chronic inflammation. Smoking is also associated with a multitude of medical conditions, such as emphysema and atherosclerosis, which could exacerbate the symptoms of COVID-19.

A recent study, posted March 31 to the preprint database bioRxiv, proposed a more speculative explanation as to why COVID-19 hits smokers harder. The preliminary research has not yet been peer-reviewed, but early interpretations of the data suggest that smoke exposure increases the number of ACE2 receptors in the lungs the receptor that SARS-CoV-2 plugs into to infect cells.

Many of the receptors appear on so-called goblet and club cells, which secrete a mucus-like fluid to protect respiratory tissues from pathogens, debris and toxins. It's well-established that these cells grow in number the longer a person smokes, but scientists don't know whether the subsequent boost in ACE2 receptors directly translates to worse COVID-19 symptoms. What's more, it's unknown whether high ACE2 levels are relatively unique to smokers, or common among people with chronic lung conditions.

Several early studies have suggested a link between obesity and more severe COVID-19 disease in people. One study, which analyzed a group of COVID-19 patients who were younger than the age of 60 in New York City, found that those who were obese were twice as likely as non-obese individuals to be hospitalized and were 1.8 times as likely to be admitted into critical care.

"This has important and practical implications" in a country like the U.S. where nearly 40% of adults are obese, the authors wrote in the study, which was accepted into the journal Clinical Infectious Diseases but not yet peer-reviewed or published. Similarly, another preliminary study that hasn't yet been peer-reviewed found that the two biggest risk factors for being hospitalized from the coronavirus are age and obesity. This study, published in medRxiv looked at data from thousands of COVID-19 patients in New York City, but studies from other cities around the world found similar results, as reported by The New York Times.

A preliminary study from Shenzhen, China, which also hasn't been peer-reviewed, found that obese COVID-19 patients were more than twice as likely to develop severe pneumonia as compared with patients who were normal weight, according to the report published as a preprint online in the journal The Lancet Infectious Diseases. Those who were overweight, but not obese, had an 86% higher risk of developing severe pneumonia than did people of "normal" weight, the authors reported. Another study, accepted into the journal Obesity and peer-reviewed, found that nearly half of 124 COVID-19 patients admitted to an intensive care unit in Lille, France, were obese.

It's not clear why obesity is linked to more hospitalizations and more severe COVID-19 disease, but there are several possibilities, the authors wrote in the study. Obesity is generally thought of as a risk factor for severe infection. For example, those who are obese had longer and more severe disease during the swine flu epidemic, the authors wrote. Obese patients might also have reduced lung capacity or increased inflammation in the body. A greater number of inflammatory molecules circulating in the body might cause harmful immune responses and lead to severe disease.

Blood type seems to be a predictor of how susceptible a person is to contracting SARS-CoV-2, though scientists haven't found a link between blood type per se and severity of disease.

Jiao Zhao, of The Southern University of Science and Technology, Shenzhen, and colleagues looked at blood types of 2,173 patients with COVID-19 in three hospitals in Wuhan, China, as well as blood types of more than 23,000 non-COVID-19 individuals in Wuhan and Shenzhen. They found that individuals with blood types in the A group (A-positive, A-negative and AB-positive, AB-negative) were at a higher risk of contracting the disease compared with non-A-group types. People with O blood types (O-negative and O-positive) had a lower risk of getting the infection compared with non-O blood types, the scientists wrote in the preprint database medRxiv on March 27; the study has yet to be reviewed by peers in the field.

In a more recent study of blood type and COVID-19, published online April 11 to medRxiv, scientists looked at 1,559 people tested for SARS-CoV-2 at New York Presbyterian hospital; of those, 682 tested positive. Individuals with A blood types (A-positive and A-negative) were 33% more likely to test positive than other blood types and both O-negative and O-positive blood types were less likely to test positive than other blood groups. (There's a 95% chance that the increase in risk ranges from 7% to 67% more likely.) Though only 68 individuals with an AB blood type were included, the results showed this group was also less likely than others to test positive for COVID-19.

The researchers considered associations between blood type and risk factors for COVID-19, including age, sex, whether a person was overweight, other underlying health conditions such as diabetes mellitus, hypertension, pulmonary diseases and cardiovascular diseases. Some of these factors are linked to blood type, they found, with a link between diabetes and B and A-negative blood types, between overweight status and O-positive blood groups, for instance, among others. When they accounted for these links, the researchers still found an association between blood type and COVID-19 susceptibility. When the researchers pooled their data with the research by Zhao and colleagues out of China, they found similar results as well as a significant drop in positive COVID-19 cases among blood type B individuals.

Why blood type might increase or decrease a person's risk of getting SARS-CoV-2 is not known. A person's blood type indicates what kind of certain antigens cover the surfaces of their blood cells; These antigens produce certain antibodies to help fight off a pathogen. Past research has suggested that at least in the SARS coronavirus (SARS-CoV), anti-A antibodies helped to inhibit the virus; that could be the same mechanism with SARS-CoV-2, helping blood group O individuals to keep out the virus, according to Zhao's team.

Many medical conditions can worsen the symptoms of COVID-19, but why do historically healthy people sometimes fall dangerously ill or die from the virus? Scientists suspect that certain genetic factors may leave some people especially susceptible to the disease, and many research groups aim to pinpoint exactly where those vulnerabilities lie in our genetic code.

In one scenario, the genes that instruct cells to build ACE2 receptors may differ between people who contract severe infections and those who hardly develop any symptoms at all, Science magazine reported. Alternatively, differences may lie in genes that help rally the immune system against invasive pathogens, according to a recent Live Science report.

For instance, a study published April 17 in the Journal of Virology suggests that specific combinations of human leukocyte antigen (HLA) genes, which train immune cells to recognize germs, may be protective against SARS-CoV-2, while other combinations leave the body open to attack. HLAs represent just one cog in our immune system machinery, though, so their relative influence over COVID-19 infection remains unclear. Additionally, the Journal of Virology study only used computer models to simulate HLA activity against the coronavirus; clinical and genetic data from COVID-19 patients would be needed to flesh out the role of HLAs in real-life immune responses.

Originally published on Live Science.

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Why COVID-19 kills some people and spares others. Here's what scientists are finding. - Livescience.com

As the rumblings of a pandemic began to be felt at the beginning of the year, scientists at Penn started work to develop a vaccine and assess possible treatments. But the scope of COVID-19 studies at the University goes much broader. Scientists whose typical work finds them investigating autoimmune disease, influenza, HIV/AIDS, Ebola, cancer, hemophilia, and more, are now applying their deep understanding of biology to confront a novel threat.

The more scientists and clinicians observe about the virus, the more avenues of investigation emerge, aiming to shed light on questions such as what happens once the virus enters the body, what treatments might be of benefit, and how society should take action to keep transmission low.

To dig into what scientists around campus are asking and learning, Penn Today spoke with several who have pivoted their research to focus on COVID-19. Their work, while in its early days, is in many cases already finding applications in the fight against this ferocious virus, and may well shape the next steps to defeat it.

Another respiratory infection, influenza, has been a focus of research led by Andrew Vaughan of the School of Veterinary Medicine. But Vaughan didnt hesitate to begin studies of the novel coronavirus once its eventual impact became apparent.

Its not a stretch for our lab, he says. All the projects in our lab focus on repair and regeneration of the lungs after injury. The majority of my studies are to some degree agnostic about what is causing the injury.

Earlier work by his group, for example, showed that a lung cell transplant could boost healing in mice affected by a severe bout with flu. Now, graduate students and research specialists in his labworking no more than two together at a time to maximize social distancingare conducting new experiments focused more specifically on the biology of SARS-CoV-2, alongside parallel efforts by Edward Morrissey from the Perelman School of Medicine (PSOM). Knowing that the Ace2 receptor on lung cells is the gateway for the virus into the human body, theyre genetically manipulating alveolar type-two lung cells, those that are particularly essential for continuing oxygen exchange deep in the lungs, to alter or block ACE2 gene expression to try to prevent viral entry.

These alveolar type-two cells seem to be particularly susceptible to injury in both influenza and perhaps even more so in COVID-19, says Vaughan. In a perfect world, you might be able to take these genetically edited type-two cells and use them as a cellular therapy. I dont know that this is going to happen in time to impact this pandemic, but even if the pathogen the next time around is slightly different, we may still be able to employ these types of regenerative responses to help the lung recover better from injury.

In a separate project, Vaughan is partnering with Penn Vets Montserrat Anguera to explore a curious feature of COVID-19 disease: the fact that more men than women become severely ill and die. A number of hypotheses have been put forward to explain the disparity, but the two labs are investigating one particular possibility.

Dr. Anguera had posted something on Twitter saying that the ACE2 gene happens to be on the X chromosome, meaning that women have two copies of it, says Vaughan. I immediately texted her and said, I think theres something to that.

Normally women inactivate one of their X chromosomes, but some genes can escape this inactivation. This means its possible women may have higher ACE2 expression than men. Somewhat counterintuitively, scientists have actually found that higher ACE2 levels actually reduce lung injury, even though ACE2 is also what the virus depends on to enter cells.

Hormone expression levels are, of course, another factor that may influence sex differences in disease. Together, Anguera and Vaughans groups are both studying ACE2 expression and exposing alveolar type-two cells to various hormones to see how expression of viral receptors, Ace2 and others, changes. Ultimately wed like to see if this changes susceptibility to infection, working with Susan Weiss and others, says Vaughan.

Individual differences in how people respond to infection may be influenced by their unique genomic sequences. Penn Integrates Knowledge Professor Sarah Tishkoff of PSOM and the School of Arts & Sciences, is probing the rich sources of genomic data her group already had in hand to look for patterns that could explain differences in disease susceptibility. As in Vaughan and Angueras work, ACE2 is a focus.

This gene is very important for general health, Tishkoff says. Women have two copies, men have one; it plays a role in regulating blood pressure; its in the kidneys; its in the gut. We want to understand the role that variation at this gene may play in risk for COVID-19, severity of disease in people with underlying health conditions, and differences in the prevalence of disease in men and women.

Using genomic data from 2,500 Africans collected for another project, Tishkoffs team is looking for patterns of genetic diversity. Early findings suggest that natural selection may have acted upon on version of the ACE2 gene, making it more common in some African populations with with high exposure to animal viruses.

Shes also collaborating with Anurag Vermaand Giorgio Sirugo of Penn Medicine to analyze genetic variation in samples from the Penn Medicine Biobank, looking in particular at people of African descent. Were seeing disturbing health disparities with COVID, with African Americans at higher risk for serious illness, says Tishkoff. This disparity mostlikelyhas to do with inequities in access to health care and socioeconomic factors, but were also looking to see if genomic variation may be playing a role.

Looking ahead, Tishkoff hopes to partner with Daniel Rader and others through the Center for Global Genomics and Health Equity to work with the West Philadelphia community. Wed like to do testing to understand the prevalence of infection and identify environmental and genetic risk factors for disease, she says.

The immune reaction to SARS-CoV-2 is a double-edged sword. The immune system is what eliminates the virus, says E. John Wherry of PSOM. The immune system is what we need to activate with a good vaccine. But also, especially in many respiratory infections, the immune system is what also causes damage. A healthy outcome means your immune system is striking a balance between killing off the virus and not doing so much damage that it kills you.

Wherry and PSOMs Michael Betts have embarked on a study to discern both the magnitude of patients immune responses as well as their flavor, that is, what components in the immune system are being activated by the coronavirus. Theyre doing so by working with clinicians at the Hospital of the University of Pennsylvania (HUP) and, soon, at Penn Presbyterian Medical Center, to collect blood samples from patients with severe and more mild infections, as well as patients who have recovered from illness, to profile their immune reactions.

Its one of the beautiful things about Penn. Everyone is working as a team, being selfless, being present, and bringing all their expertise to bear on this crisis. E. John Wherry, Perelman School of Medicine

We are observing a huge amount of heterogeneity across these patient samples, says Betts. But were also identifying some relatively unifying characteristics, indicating there are mechanisms that everyone uniformly uses to fight off this infection.

This variety across patients strongly suggests that the treatments that work for one patient may not for another, Wherry and Betts note. For that reason, they are speaking daily with their colleagues on the front lines of COVID-19 care, relaying what theyre finding out in the lab to adjust and personalize care in the clinic.

Its one of the beautiful things about Penn, says Wherry. Everyone is working as a team, being selfless, being present, and bringing all their expertise to bear on this crisis.

Plenty of recent scientific attention has been paid to the role of the gut microbiome in health. But the medical schools Ronald Collman and Frederic Bushman have been devoting attention to how the community of bacteria, viruses, fungi, and parasites that dwell in the respiratory tract affect health and disease risk. They are now addressing that question in the context of COVID-19.

There are two reasons were interested in studying this, Collman says. First is that the microbiome can help set the tone for the immune response to infections, influencing whether a patient ends up with mild or severe disease. And second, the microbiome is where infectious agents that can cause infection can arise from. So if a patient dies of an eventual pneumonia, the pathogen that caused that pneumonia may have been part of that individuals respiratory tract microbiome.

Working with nurses at HUP to collect samples, Collman and Bushman are analyzing the microbiome of both the upper (nose and throat) and lower (lung) portions of the respiratory tract of COVID-19 patients. These samples are being used by other groups, such as those developing diagnostic tests, while Collman and Bushmans labs work to identify the types and quantities of organisms that compose the microbiome to find patterns in how they correlate with disease.

Were hoping that if we can find that the response to the virus is different in people with different upper respiratory tract microbiomes, then we could manipulate the microbiome, using particular antibiotics, for example, to make it more likely that patients would have a mild form of the disease.

Absent a vaccine, researchers are looking to existing drugssome already approved by the U.S. Food and Drug Administration for other maladiesto help patients recover once infected. Throughout his career, Ronald Harty of Penn Vet has worked to develop antivirals for other infections, such as Ebola, Marburg, and Lassa Fever.

Our antivirals are sometime referred to as host-oriented inhibitors because theyre designed to target the interaction between host and viral proteins, says Harty. Though many of the biological details of how SARS-CoV-2 interacts with the human body are distinct from the other diseases Harty has studied, his group noticed a similarity: A sequence hes targeted in other virusesa motif called PPxYis also present in the spike protein of SARS-CoV-2, which the coronavirus uses to enter cells.

This caught our eye, says Harty, and piqued our interest in the very intriguing possibility that this PPxY motif could play a role in the severity of this particular virus.

Harty is testing antivirals he has helped identify that block the replication of Ebola, Marburg, and other viruses to see if they make a dent on the activity of SARS-CoV-2. Those experiments will be done in collaboration with colleagues whose labs can work in BSL-III or -IV laboratories, such as Penns Weiss.

Also of interest is the speculation that the coronavirus might disrupt cell-cell junctions in the human body, making them more permeable for virus spread. Hartys lab will be examining the potential interactions between the viral structural proteins and human proteins responsible for maintaining these cellular barriers.

Another faculty member is assessing whether a drug developed for a very different conditionin this case, pulmonary arterial hypertension (PAH)could serve coronavirus patients. Henry Daniell of the School of Dental Medicine recently shared news that a drug grown in a plant-based platform to boost levels of ACE2 and its protein product, angiotensin (1-7), was progressing to the clinic to treat PAH. Daniell is now working with Kenneth Margulies from Penn Medicine to explore whether this novel oral therapy can improve the clinical course of patients with symptomatic COVID-19 infection.

Reduced ACE2 expression has been linked to acute respiratory distress, severe lung injury, multi-organ failure and death, especially in older patients. The earlier preclinical studies in PAH animal models showed that orally delivered ACE2 made in plant cells accumulated ten times higher in the lungs than in the blood and safely treated PAH. Now, new clinical studies have been developed to explore whether oral supplementation of ACE2 and angiotensin-1-7 can help mitigate complications of COVID-19 disease. The fact that freeze-dried plant cells can be stored at room temperature for as long as a year and can be taken at home by COVID-19 patients make this novel approach an attractive potential option.

This trial has been given a high priority by the Penn Clinical Trial Working Group, says Daniell. Im pleased that this looks to be on the cusp of moving forward to help the growing number of COVID-19 patients.

As the coronavirus began to spread in the United States, biologist Joshua Plotkin of the School of Arts & Sciences began to raise alarms about Philadelphias St. Patricks Day parade, which had been scheduled to be held March 15, potentially drawing thousands to downtown streets. He had good reason to be concerned: His studies of the 1918 flu pandemic had explored disease incidence and spread, and it was hard to avoid noticing the role of the Liberty Loan parade down Broad Street in triggering a rampant spread of flu a century ago.

Now, with work conducted with two graduate students from Princeton University, Dylan Morris and Fernando Rossine, along with Princeton faculty member Simon Levin, Plotkin has mathematically sound advice for policymakers hoping to effectively stem the spread of a pandemic. In a preprint on arXiv.org, they share optimal, near-optimal, and robust strategies for how to time interventions such as social distancing.

This boils down to knowing what is the best way, of all the infinite possibilities, to intervene using public health measure, says Plotkin. Thats a problem we can solve with math, my colleagues Dylan and Fernando realized.

Their analysis makes the realistic assumption that policymakers can only enforce social distancing for a limited amount of time, and aims to minimize the peak incidence of disease. The optimal strategy, they found, is to start by introducing moderate social distancing measures to keep the incidence rate the same for a period of time. This would mean that every person with COVID-19 would infect one additional person. Then the intervention should switch over to a full suppressionthe strongest possible quarantinefor the rest of the period. At the end of that period, all restrictions would be lifted.

This works because you dont want to fully suppress disease spread right off the bat, says Plotkin, because then at the end, after you remove restrictions, there will be a second peak that is just as large as the first. By employing a moderate suppression at the beginning, youre building up a population of people who are going to recover and become immune, without letting the epidemic get out of control.

Unsurprisingly, timing is key. Attempting the optimal intervention would be disastrous, in practice, because of inevitable errors in timing. Intervening too early is pretty bad, because you get a bigger second peak, he says. But intervening too late is even worse. The key lesson is that a robust intervention is more important than an optimal one.

Plotkin and his colleagues are hoping to share the findings widely, including with local decision makers, to help them navigate a likely second wave of COVID-19.

Montserrat Anguera is an associate professor of biomedical sciences at the University of Pennsylvania School of Veterinary Medicine.

Michael Betts is a professor of microbiology at the University of Pennsylvania Perelman School of Medicine.

Frederic Bushman is the William Maul Measey Professor in Microbiology at the University of Pennsylvania Perelman School of Medicine.

Ronald Collman is a professor of Medicine at the University of Pennsylvania Perelman School of Medicine.

Henry Daniell is vice-chair and W.D. Miller Professor in the Department of Basic and Translational Sciences in the University of Pennsylvania School of Dental Medicine.

Ronald Harty is a professor of pathobiology and microbiology at the University of Pennsylvania School of Veterinary Medicine.

Kenneth Margulies is a professor of medicine and physiology and research and fellowship director of the Heart Failure and Transplant Program at the University of Pennsylvania Perelman School of Medicine.

Joshua Plotkin is the Walter H. and Leonore C. Annenberg Professor of the Natural Sciences in the Department of Biology at the University of Pennsylvania School of Arts & Sciences. He has secondary appointments in the Department of Mathematics and in the School of Engineering and Applied Sciences Department of Computer and Information Science.

Sarah Tishkoff is the David and Lyn Silfen University Professor with appointments in the Perelman School of Medicines Department of Genetics and the School of Arts and Sciences Department of Biology. A Penn Integrates Knowledge Professor, she is also director of the Penn Center for Global Genomics and Health Equity.

Andrew Vaughan is an assistant professor of biomedical sciences at the University of Pennsylvania School of Veterinary Medicine.

E. John Wherry is chair of the Department of Systems Pharmacology and Translational Therapeutics, director of the Institute for Immunology, and the Richard and Barbara Schiffrin Presidents Distinguished Professor at the University of Pennsylvania Perelman School of Medicine.

Homepage image: Researchers around the University are taking a variety of approaches to study the novel coronavirus (particles of which are shown in purple), informed by past expertise and newly formed collaborations. (Image: National Institutes of Health)

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Coming together to solve the many scientific mysteries of COVID-19 - Penn: Office of University Communications