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Your daily 6: Third vaccine looks effective, no single ‘word of the year’ and Trump team called ‘a national embarrassment’ – Ravalli Republic

November 24th, 2020 9:57 am

HARTFORD, Conn. (AP) Jury duty notices have set Nicholas Philbrook's home on edge with worries about him contracting the coronavirus and passing it on to his father-in-law, a cancer survivor with diabetes in his mid-70s who is at higher risk of developing serious complications from COVID-19.

Philbrook and his wife, Heather Schmidt, of Camarillo, California, have been trying to convince court officials that he should be excused from jury duty because her father lives with them. But court officials told him that is not a valid reason and he must appear in court early next month.

"My main concern is you still have to go into a building, you still have to be around a set number of people," said Philbrook, 39, a marketing company editor. "In an enclosed space, how safe are you? It just doesn't feel like a right time still to be doing that kind of stuff on a normal basis."

People across the country have similar concerns amid resurgences of the coronavirus, a fact that has derailed plans to resume jury trials in many courthouses for the first time since the pandemic started.

Within the past month, courts in Hartford, Connecticut, San Diego and Norfolk, Virginia, have had to delay jury selection for trials because too few people responded to jury duty summonses. The non-response rates are much higher now than they were before the pandemic, court officials say.

Judges in New York City, Indiana, Colorado and Missouri declared mistrials recently because people connected to the trials either tested positive for the virus or had symptoms.

"What the real question boils down to are people willing to show up to that court and sit in a jury trial? said Bill Raftery, a senior analyst with the National Center for State Courts. "Many courts have been responsive to jurors who have said that they're not comfortable with coming to court and doing jury duty and therefore offering deferrals simply because of concerns over COVID."

Also this month, state court systems in Connecticut, New York and New Jersey and courts in Denver, Colorado, were among those to suspend all jury trials because of rising virus rates. On Friday, federal officials announced that about two dozen U.S. district courts across the county have suspended jury trials and grand jury proceedings because of virus outbreaks and too few people showing up for jury duty.

Courts are under pressure to resume trials because of the case backlogs piling up during the pandemic.

A few courts have held trials in person and by video conference. Although video conferences may appear to be the best bet, many criminal defense lawyers oppose them because it's harder to determine witness credibility and to see if jurors are paying attention, said Christopher Adams, a lawyer in Charleston, South Carolina, and president of the National Association of Criminal Defense Lawyers.

"For almost everybody, there is no compelling need for trials to go forward during the pandemic," he said, adding that most courts are not holding jury trials at the moment.

Adams said another concern is how representative juries would be if trials went ahead the virus' impact and the level of concern about it across different demographics, such as Black, Latino and elderly populations that are dying at higher rates, could affect who feels safe to serve jury duty.

"What we can't allow is to have trials where there's not a fair cross section of the community represented," he said.

But many criminal defense lawyers are pointing to a major issue with not holding trials defendants who are detained while awaiting trial. Although jails and prisons across the country have released thousands of low-risk inmates because of concerns about the virus, many people remain locked up in pretrial detention.

A case in federal court in Hartford, Connecticut, offers a glimpse of how the virus can upend proceedings.

In October, 150 people were summoned for jury duty for the trial of Amber Foley, who is fighting child pornography charges and demanding her constitutional right to a speedy trial. It would be the first criminal trial in Connecticut, in state or federal court, since the pandemic began.

Only about half the potential jurors showed up and many others were excused for various reasons including concerns about COVID-19. Only 19 people were left, short of the 31 people estimated to be needed to pick a jury of 12 and one alternate juror.

And then, two court security officers tested positive for the virus, forcing the temporary closure of the courthouse for cleaning and prompting Judge Vanessa Bryant's law clerk to go into isolation and get tested because of contact with the officers.

Bryant decided last week to postpone Foley's jury selection until mid-January. Like judges in other parts of the country, she ruled the interests of public health outweigh those of a speedy trial.

"Despite every effort being made by the Court, the Court must reluctantly conclude that it is unable to empanel a representative jury from the 200 prospective jurors summoned without jeopardizing the safety of all trial participants," Bryant wrote in a ruling.

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Your daily 6: Third vaccine looks effective, no single 'word of the year' and Trump team called 'a national embarrassment' - Ravalli Republic

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Editorial Article: Increased NGS throughput for personalized genomics with automated cell counting – SelectScience

November 24th, 2020 9:55 am

Sangmi Han discusses how cell counters have become an integral part of Macrogens single-cell RNA sequencing workflow

Macrogen, Inc. is a leading expert in genomic analysis, proactively seeking to improve the fields of genetic and genomic analyses through research and development. To further this mission, Macrogen is working to identify the causal genes for rare diseases with a view to building a big data system that brings together patient genomic and medical data. Using this integrated database, the team aims to help predict disease onset and prognosis, and innovate personalized medicines at an individual level.

Here, we speak with Sangmi Han, Macrogens Head of the Next-Generation Sequencing (NGS) Technical Services Department, about how the LUNA-FX7 Automated Cell Counter from Logos Biosystems has increased the throughput, reliability, and accuracy of her labs single-cell RNA sequencing and precision medicine workflows. Han explains how these state-of-the-art cell counters are helping Macrogen to predict, prevent, and treat diseases on a personal level.

SH: Macrogen is a global leader in precision medicine and biotechnology with the aim of medical innovation for the future by integrating genomic, medical, and life sciences data. We possess the worlds fifth-class genome analysis platform, sequencing and data analysis capacity, and support systematic genome research through precise genome analysis services and personalized medicine that diagnoses, treats, monitors, predicts, and prevents diseases based on the captured genomic information.

SH: I look after whole genome sequencing and metagenome sequencing for microbial identification. When accurate genomic information is obtained from customers and patients, the genes that cause a specific disease can be found based on this sequencing data. Through this genetic analysis, diagnostic kits and panels can be developed with our technology and diseases can be more accurately diagnosed, predicted, prevented, and treated, leading to a much higher quality of patient life.

SH: A current trend in the NGS market is the single-cell library where genes can be analyzed through the unique barcoding of each cell. To construct single-cell sequencing libraries, we count the cells that have been isolated from specific tissues and provided to us by customers. Since we need to perform single-cell RNA sequencing for a certain number of cells in order to enter a sufficient number of barcodes, cell counting is an essential process and must be accurate. In addition, as the demand for single-cell library construction increases, the capacity of cell counting and quality control equipment is becoming ever more important.

To ensure this high accuracy in our cell counts, we use the LUNA-FX7 Automated Cell Counter from Logos Biosystems, the latest and most powerful in the LUNA Family. This instrument provides not only high accuracy but also high throughput, with the LUNA 8-channel slides enabling the counting of eight separate samples at once, fully assisting us in overcoming any potential difficulties we may face during our experiments. We also cross-check these results using manual counting via a microscope. As experimental analysis of single-cell libraries is conducted according to the number of target cells, cell counting is highly important. The better the input number of cells matches the output result, the more reliable the data.

SH: In the NGS market, advanced and precision kits are becoming increasingly popular and being released at a rapid rate. Comprehensive services, from companies like Macrogen, can be provided through the performance of various sequencing analyses, from human to non-human. As a result, we are sure that the use of big data will drastically increase and, due to the vast amount of data available to us, the market size of personal genomics and disease prediction and prevention services will only continue to grow.

Do you use Logos Biosystems products in your lab? Write a review today for your chance to win>>

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Determining the Biomarkers of COVID-19 Outcome and Severity – Contagionlive.com

November 24th, 2020 9:55 am

Metabolon a biotechnology company based in Durham, North Carolina, has been selected by the NIAID to participate in a study on the coronavirus disease 2019 (COVID-19). The study is a part of the NIAIDs Immunophenotyping Assessment in a COVID-19 Cohort (IMPACC) research.

The program is being led by researchers at the Precision Vaccines Program (PVP) at Boston Childrens Hospital. The PVP is a collaboration of academics, government, and industry leaders who are working on advancing the next generation of vaccines for at-risk populations.

It features roughly 400 scientists and administrators around the world through the Precision Vaccines Network (PVN). The PVN employs systems vaccinology, humanin vitromodeling, adjuvant discovery and development through human clinical trials.

The study is focused on researching how a SARs-CoV-2 infection effects the human immune systems response network, focusing mainly on determining the biomarkers of COVID-19 outcome and severity.

The study is supporting the PVPs Clinical Data Collection Center (CDCC), assisting them in their efforts on integrating clinical data like sex, age, and the diseases severity and outcomes with biological data, including metabolomics.

A fundamental aspect to unlocking the genome using multi-omics research, a type of biological analysis, is Metabolomics. This approach aids in population health initiatives and helps in reaching the global goal of personalized medicine.

Metabolon will conduct research on global metabolomics using biological samples of serum from a population of approximately 1000 study participants. The company will collect samples from the participants and examine them across different time points, observing metabolic changes in the immune response to a SARS-CoV-2 infection over time. They will then correlate the data with the severity and outcome of a COVID-19 infection.

"It's an honor to collaborate with NIAID and participating researchers across the U.S. on the IMPACC study to assess and precisely characterize the impact of SARS-CoV-2 on health," Greg Michelotti, PhD, the scientific director of Metabolon, said. "Metabolomics provides a detailed snapshot of the phenotype, enabling deeper understanding of health and disease. The actionable metabolomics insights will help inform development of diagnostics, prognostics, therapeutics and vaccines to address the COVID-19 pandemic."

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Determining the Biomarkers of COVID-19 Outcome and Severity - Contagionlive.com

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Outlook on the Companion Diagnostic Global Market to 2025 with COVID-19 Updates – PRNewswire

November 24th, 2020 9:55 am

DUBLIN, Nov. 24, 2020 /PRNewswire/ -- The "Companion Diagnostic Markets - the Future of Diagnostics, by Funding Source and Application with Customized Forecasting/Analysis, COVID-19 Updates, and Executive and Consultant Guides 2021-2025" report has been added to ResearchAndMarkets.com's offering.

Companion Diagnostics are poised to revolutionize the diagnostics industry. The market is finally moving out of the lab and into the clinic. Oncology, especially immune-oncology is leading the way. And the FDA is holding the door open for this diagnostic technology of the future. But COVID-19 is impacting healthcare treatment everywhere and lowering demand for specialized cancer testing. Find out the latest outlook for this important market.

Will Personalized Companion Diagnostics become the norm for diagnostics?

Learn all about how diagnostic players are jockeying for position with their pharmaceutical counterparts and creating new and significant business opportunities. And some players are already taking the lead. It is a dynamic market situation with enormous opportunity. Diagnostic companies are trying to back the right horse. The science is racing forward. And the cost of molecular diagnostics continues to fall.

This report provides data that analysts and planners can use. Hundreds of pages of information including a complete list of Current 2020 United States Medicare Fee Payment Schedules to help understand test pricing in detail. Forecast demand for new testing regimes or technologies. Make research investment decisions. Existing laboratories and hospitals can use the information directly to forecast and plan for clinical facilities growth.

The report includes detailed breakouts for 18 Countries and 4 Regions.

Companion Diagnostic Market - Strategic Situation Analysis

Key Topics Covered:

1. Introduction and Market Definition

2. Market Overview 2.1 Players in a Dynamic Market 2.1.1 Academic Research Lab 2.1.2 Diagnostic Test Developer2.1.3 Instrumentation Supplier 2.1.4 Distributor and Reagent Supplier 2.1.5 Independent Testing Lab2.1.6 Public National/regional lab 2.1.7 Hospital lab 2.1.8 Physician Office Labs 2.1.9 Audit Body 2.1.10 Certification Body2.2 Personalized Medicine and Companion Diagnostics 2.2.1 Basics 2.2.2 Method 2.2.3 Disease risk assessment 2.2.4 Applications 2.2.5 Diagnosis and intervention 2.2.5.1 Companion Diagnostics 2.2.6 Drug development and usage 2.2.7 Respiratory proteomics 2.2.8 Cancer genomics 2.2.9 Population screening 2.2.10 Challenges 2.2.11 Regulatory oversight 2.2.12 Intellectual property rights 2.2.13 Reimbursement policies2.2.14 Patient privacy and confidentiality 2.3 Chromosomes, Genes and Epigenetics 2.3.1 Chromosomes2.3.2 Genes 2.3.3 Epigenetics 2.4 Cancer Genes 2.4.1 Germline vs Somatic 2.4.2 Changing Clinical Role 2.5 Structure of Industry Plays a Part 2.5.1 New Pharmaceutical Funding Market 2.5.2 Economies of Scale2.5.2.1 Hospital vs. Central Lab 2.5.3 Physician Office Labs 2.5.4 Physicians and POCT

3. Market Trends3.1 Factors Driving Growth3.1.1 Level of Care 3.1.2 Immuno-oncology 3.1.3 Liability3.1.4 Aging Population3.2 Factors Limiting Growth3.2.1 State of knowledge3.2.2 Genetic Blizzard. 3.2.3 Protocol Resistance3.2.4 Regulation and coverage 3.3 Instrumentation and Automation 3.3.1 Instruments Key to Market Share 3.3.2 Bioinformatics Plays a Role 3.4 Diagnostic Technology Development3.4.1 Next Generation Sequencing Fuels a Revolution. 3.4.2 Single Cell Genomics Changes the Picture 3.4.3 Pharmacogenomics Blurs Diagnosis and Treatment3.4.4 CGES Testing, A Brave New World 3.4.5 Biochips/Giant magneto resistance based assay

4. Companion Diagnostics Recent Developments 4.1 Recent Developments - Importance and How to Use This Section 4.1.1 Importance of These Developments 4.1.2 How to Use This Section

5. Profiles of Key Players

6. The Global Market for Companion Diagnostics6.1 Global Market Overview by Country6.1.1 Table - Global Market by Country6.1.2 Chart - Global Market by Country 6.2 Global Market by Application - Overview 6.2.1 Table - Global Market by Application 6.2.2 Chart - Global Market by Application - Base/Final Year Comparison 6.2.3 Chart - Global Market by Application - Base Year 6.2.4 Chart - Global Market by Application - Final Year 6.2.5 Chart - Global Market by Application - Share by Year6.3 Global Market Funding Source - Overview 6.3.1 Table - Global Market by Funding Source 6.3.2 Chart - Global Market Funding Source - Base/Final Year Comparison 6.3.3 Chart - Global Market Funding Source - Base Year 6.3.4 Chart - Global Market Funding Source - Final Year6.3.5 Chart - Global Market Funding Source - Share by Year

7. Global Companion Diagnostic Markets - By Application 7.1 Oncology 7.1.1 Table Oncology - by Country 7.1.2 Chart - Oncology Growth7.2 Neurology 7.2.1 Table Neurology - by Country 7.2.2 Chart - Neurology Growth 7.3 Cardiology 7.3.1 Table Cardiology - by Country 7.3.2 Chart - Cardiology Growth7.4 Other Application7.4.1 Table Other Application - by Country7.4.2 Chart - Other Application Growth

8. Global Companion Diagnostic Markets - Funding Source8.1 Global Market Pharmaceutical 8.1.1 Table Pharmaceutical - by Country8.1.2 Chart - Pharmaceutical Growth 8.2 Global Market Venture 8.2.1 Table Venture - by Country8.2.2 Chart - Venture Growth 8.3 Global Market Clinical 8.3.1 Table Clinical - by Country8.3.2 Chart - Clinical Growth 8.4 Global Market Other Funding 8.4.1 Table Other Funding - by Country 8.4.2 Chart - Other Funding Growth

For more information about this report visit https://www.researchandmarkets.com/r/wci97x

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

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

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Outlook on the Companion Diagnostic Global Market to 2025 with COVID-19 Updates - PRNewswire

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Oculis to Present Progress in its Portfolio of Novel Topical Treatments for Retinal and Front-of-the-Eye Diseases at Upcoming Investor Conferences -…

November 24th, 2020 9:55 am

LAUSANNE, Switzerland, Nov. 24, 2020 (GLOBE NEWSWIRE) -- Oculis S.A., a biopharmaceutical company focused on developing transformative topical ophthalmic treatments to improve the sight and lives of patients, announced that Riad Sherif M.D., CEO, will present an overview of the company at the following investor conferences:

Date/Time: The company presentation has been made available on demand as of Monday, November 23 at 10AM ET to registered attendees on the conference website. The presentation will be available for playback throughout the duration of the event.

Piper Sandler conferences and events are by invitation only. If you wish to attend, please contact yourPiper Sandlerrepresentative. http://www.pipersandler.com

Date/Time: The company presentation will be made available online as part of a virtual library the week of the event to registered attendees on the conference website.

To access the presentation, please register HERE: https://hcwevents.com/bioconnect/

Dr. Sherif will provide an overview of Oculis portfolio and the significant progress it has made during 2020 with the advancement of its lead candidates OCS-01 and OCS-02.

A key focus of the presentations will be on the development of OCS-01 as a potentially transformative and first topical treatment for Diabetic Macular Edema (DME) and as the first once a day steroid for the treatment of inflammation and pain following ocular surgery. Positive clinical results from Phase 2 trials of OCS-01 in both indications were announced this year in February and May, respectively. The Company is now moving to Phase 3 pivotal trials following a positive End of Phase 2 meeting with FDA in both indications. These trials are expected to start in the first half of 2021.

Dr. Sherif will also discuss progress with OCS-02, a novel and a potentially first-in-class topical anti-TNF alpha antibody. OCS-02 has successfully completed two proof-of-concept (POC) clinical trials in Dry Eye Disease (DED) and Acute Anterior Uveitis and aims to initiate Phase 2b trials in 2021. In DED, Oculis will continue to validate a novel biomarker identified in patients who showed a highly positive response to treatment. If confirmed in the next trial, this finding could accelerate and de-risk the pivotal trials program and potentially lead to a novel, personalized medicine approach to the treatment of DED, with an associated companion diagnostic.

About Oculis

Oculis S.A. is a biopharmaceutical company focused on developing transformative topical ophthalmic treatments using its innovative formulation technologies to improve the sight and lives of patients. Oculis novel topical (eye drop) treatments are non-invasive and represent an unprecedented technical advance for patients with retinal and front-of-the-eye diseases.

The Companys leading clinical candidates include OCS-01 and OCS-02:

In addition, Oculis formulation discovery focus and capabilities are enabling the development of a pipeline of topical drugs targeting sight-threatening eye diseases that affect both the anterior and posterior segments of the eye.

Oculis has an experienced management team from global ophthalmic companies and is supported by leading international life science investors. Oculis is headquartered in Lausanne, Switzerland, with research operations in Iceland, and U.S. operations in Boston, MA.

To learn more, visit http://www.oculis.com

Contacts

OculisDr. Riad Sherif, CEOriad.sherif@oculis.comSylvia Cheung, CFOsylvia.cheung@oculis.com Louie-Anne Gauthier, VP, Strategic Marketing and BD&Llouie-anne.gauthier@oculis.com

Media Relations

Citigate Dewe RogersonMark Swallow / Sylvie Berrebi / Marine Perrieroculis@citigatedewerogerson.com+44 (0)20 7638 9571

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Oculis to Present Progress in its Portfolio of Novel Topical Treatments for Retinal and Front-of-the-Eye Diseases at Upcoming Investor Conferences -...

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Wearables Driving the Future of Precision Medicine – Healthcare Tech Outlook

November 24th, 2020 9:55 am

With their potential to track health continuously outside care facilities, wearables hold a significant potential to improve current precision medicine initiatives.

FREMONT, CA: Wearable devices have come a long way from being just entertaining to bringing in tangible health benefits. Previously, wearables with basic functionality were appreciated by fitness enthusiasts and professional athletes. But today, the capabilities of wearables emerged way beyond counting steps are taken and calories burnt. Next generations of smart wearables expanded their data collection and analytical abilities toward overall health awareness. They start identifying patterns related to the users mood, sleep quality and other vitals. Today, wearables are instead marketed as personal devices for people who is interested in making more informed choices about their health.

This trend of personalized health tracking fell in line with the value-based care paradigm and its stress on precision medicine. The present healthcare industry aspires to leverage wearables as a primary data source to develop highly personal and flexible patient treatment plans, enabling the introduction of timely therapy modifications based on slight alterations in health patterns. Additionally, wearables can also help to promote patients responsibility toward their care routines and encourage informed patient-physician communication. Smart wearable manufacturers and medical-grade software vendors also depend on custom software solutions suitable for clinical use. While researchers and decision-makers can benefit from wearables to support their efforts, it all comes down to two major groups of sensors collecting particular biological signs.

Physiological sensors measure the biological signs with electrical, thermal, acoustic and optical components. It monitors bodily functions like the gut and respiratory activity, vital signs like blood pressure, heart rate, temperature, blood oxygen saturation levels and more. Both consumer-facing and ambulatory-use wearables allow numerous biological tracking options. Some include, smartwatches with the ability to generate ECG, identify falls and abnormal heart rhythms, patch sensors for prenatal contraction monitoring, abdominal patch analyzing gastrointestinal motility and facilitating diagnostics of digestive disorders, and fertility tracker with heart rate, temperature and stress level monitoring capabilities.

Being more aware of their wellness and biological signals, patients can start actively engaging in the dialogue about their therapy. They can consider their possible health outcomes in the long run, and realize that both their opinion and actions matter.

Check This Out:Top Medical Device Consulting Companies

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Tissue Sectioning Devices Market: Growth of Personalized Therapeutics and Diagnostics to Boost Market – BioSpace

November 24th, 2020 9:55 am

Tissue Sectioning Devices: Introduction

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Key Drivers and Restraints of Global Tissue Sectioning Devices Market

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Growth of personalized therapeutics and diagnostics to boost market

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Hydrogen Peroxide Tissue Sectioning Devices Segment to Dominate Global Market

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North America to Dominate Global Tissue Sectioning Devices Market

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Leading Players Operating in Global Tissue Sectioning Devices Market

The global tissue sectioning devices market was highly fragmented in 2019. Key players operating in the global tissue sectioning devices market are:

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Persephone Biosciences Initiates the ARGONAUT Study of Gut Microbiome-Linked Immune Modulation in Cancer Treatment Response – BioSpace

November 24th, 2020 9:55 am

SAN DIEGO, Nov. 23, 2020 /PRNewswire/ --Persephone Biosciences Inc., a biotechnology company that leverages real world data and artificial intelligence to design microbiome therapeutics and diagnostics to promote normal immune function and fight disease, today announced the initiation of the ARGONAUT clinical study to determine the impact of gut microbiome composition and function on the immune system and efficacy of cancer treatment.

ARGONAUT is a longitudinal, prospective, observational study that will enroll up to 4,000 advanced-stage cancer patients of diverse racial backgrounds to collect data for the development of precision microbiome medicines and for the identification of companion diagnostics to guide therapeutic decisions. Four types of cancers will be profiled: non-small cell lung cancer (NSCLC), triple negative breast cancer (TNBC), colorectal cancer (CRC) and pancreatic cancer.

"I believe this study will provide important new insights into how the microbiome can inform the treatment of patients with cancer. This work could help improve patients' response to existing and future treatments through the use of more personalized treatment regimens," said Dr. Sandip Patel, MD, Associate Professor, UC San Diego Moore's Cancer Center. "Significant cancer health disparities currently exist, but with this nationwide study that include tumor types, like TNBC, that are more prevalent in at-risk demographics, we believe we can address these disparities and develop equitable precision medicine, thereby potentially increasing survival rates and quality of life."

Chief Executive Officer and co-founder of Persephone Biosciences, Stephanie Culler, added, "The ARGONAUT study, the largest study ever of its kind in the U.S., will provide us with more data than ever before on the importance of gut microbiome in patients' response to oncology treatment. With this data, Persephone Biosciences will be the partner of choice for oncology companies wishing to improve the therapeutic benefit of existing drugs, as well as develop effective new therapies targeting microbiome optimization".

Eligible subjects will provide 2 samples each of blood and stool over a 6-month period and will be followed for up to two years.

About ARGONAUT

The official title of the ARGONAUT study is: "Development and Analysis of a Blood and Stool Sample Bank for Cancer Patients, Enabling the Systematic Study of the Effect of Blood and Gut Microbiomes on Response to Treatment." More information can be found at http://www.clinicaltrials.gov using the identifier NCT04638751.

About Persephone Biosciences:

Persephone Biosciences, a Y Combinator company, takes a novel approach to improving the efficacy of existing therapies, leveraging the systemic impact of the gut microbiome on the human immune system. Persephone's technology platform is based on population-scale observational clinical trials, advanced genomics and immunology techniques, and machine learning to understand the impact of gut microbes on patient prognosis and immune system function. The results are used to design precision immunotherapies and companion diagnostics for unmet needs. For more information, visit http://www.persephonebiosciences.com.

CONTACTS:Stephanie Culler, Ph.D.CEO and Co-founderPersephone Biosciences, Inc.investors@persephonebiosciences.com

Laurence WattsManaging DirectorGilmartin Group, LLC.laurence@gilmartinir.com

Media Contacts:Evan Wicker, Ph.D.Russo Partners, LLCevan.wicker@russopartnersllc.com212-845-4235

Olipriya Das, Ph.D.Russo Partners, LLCOlipriya.Das@russopartnersllc.com646-942-5588

View original content:http://www.prnewswire.com/news-releases/persephone-biosciences-initiates-the-argonaut-study-of-gut-microbiome-linked-immune-modulation-in-cancer-treatment-response-301178470.html

SOURCE Persephone Biosciences

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Persephone Biosciences Initiates the ARGONAUT Study of Gut Microbiome-Linked Immune Modulation in Cancer Treatment Response - BioSpace

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History of the Partnership between Tesla & Curevac Its Older than Covid-19 – CleanTechnica

November 24th, 2020 9:55 am

November 22nd, 2020 by Johnna Crider

Tesla has partnered with Curevac to help fight the coronavirus, but what is the extent of this partnership? Ive seen claims all over Twitter from a few of Elon Musks critics saying that he should stick to making rockets. Honestly, telling someone with Elons net worth to not help is kind of rude, if you ask me. Opinions and thoughts about that matter aside, NZZ gives a detailed breakdown of Teslas partnership with Curevac, and no, Elon is not in some lab with a syringe filled with God only knows what while standing over a poor hapless soul strapped down to a gurney (visions that may come to mind if you look at some of the tweets out there).

This partnership is actually older than the coronavirus itself, and shows that many armchair critics and click chasers havent really done their research. The first takeaway from the German article is that Curevac and Tesla are working together on an RNA printer. Tesla is helping by building the parts of an RNA printer that will produce not only this vaccine, but also others and other medicines. That said, we should all recognize that this is no normal printer. Anyone who imagines an RNA printer as a handy printer that will shortly spit out vaccines against Sars-CoV-2, for example, is not entirely correct, the author of the article noted before explaining the story of this partnership.

Curevac, founded in 2000, was the first biotech company to work with the messenger molecule mRNA (messenger ribonucleic acid) for the fields of oncology, protein therapy, and vaccines. It came to the conclusion years ago that it needed an industrial plan in order to conduct clinical studies at all and then produce billions of doses annually, according to its CEO, Franz-Werner Haas. After that, the company wanted to eventually produce smaller quantities at a faster pace something geared toward personalized medicine. For example, its a matter of producing a personalized active ingredient for an oncology patient. This would take too much time on a large system, and the quantities would also be too large. Curevac needed additional support to implement its idea of mobile production units, stating, We are biologists, we are doctors, we are technicians, but we are not mechanical engineers. With this in mind, the company brought in Grohmann Engineering as a technical partner, which Tesla bought and is now Tesla Grohmann Automation.

An RNA printer, the article points out, is a small mobile production facility (not a printer you plug in, press a button, and print from) that is made for a certain type of pharmaceutical. It uses a specific RNA sequence as an active ingredient. This field of application is not limited to vaccines it is also being tested for antibody therapies for various diseases. These diseases range from personalized cancer medicine to gene therapy using the Crispr/Cas technique.

A Curevac spokesman described the RNA printer, which is still a prototype, as a mini-factory that is 34 meters long, 2 meters wide, and 2 meters high. Its slightly larger than an SUV and can be shipped in a container. Additionally, work is being done to make it smaller and give it more functions.

The article noted that the focus for right now will most likely be on vaccines, and that extensive immunization against certain pathogens is pretty much a logistical problem. When a vaccine is distributed, it requires a functioning cold chain from the manufacturer to the most remote village. Also, RNA is often an unstable molecule that decomposes quickly even at room temperature. Often when transporting it, the temperature has to be at least -20 to sometimes -80 degrees Celsius.

Curevac is actually working on an idea of making any mRNA vaccine directly where it is needed. This, the author pointed out, is what the RNA printer could do. The printer consists of a bioreactor in which the DNA template is overwritten in RNA. Next, the finished messenger molecules are freed from the process chemicals and then embedded in fat droplets, which prevent them from decomposing too quickly.

Grohmann Engineering GmbH, which is an engineering automation company, was acquired by Tesla in 2016. It was officially taken over by Tesla on January 3, 2017. Back then, the partnership was uncertain, and Curevac actually sent representatives to California to encourage Tesla to continue the collaboration. Tesla, as we all see clearly, did so, in addition to Tesla Grohmanns is focused on automation processes for the production of EVs and power storage systems.

With this bit of history in mind, its easy to see how it was natural for Elon Musk and Tesla to get involved with creating a vaccine for the coronavirus. Many dont take the time to fully research this partnership and are quick to loathe Elon Musk for being Elon Musk someone who wants to help. Whether calling it a publicity stunt, stock manipulation, or whatever the critics say these days, its clear that those who seek to criticize are not doing their research.

If one was doing actual research, they would have not only realized that Tesla is working with Curevac to make molecule printers, but that the company has been working with Curevac behind the scenes since Tesla acquired Grohmann Engineering GmbH 4 years ago. And the research would have you understand that Teslas engineers are working in partnership with Curevac in a meaningful way. Just note that they are making the mechanical parts of the printer, not the actual vaccine itself.

Speaking of the vaccine, in a video call with members of the press, Haas explained that Curevac had good feedback on its Phase 1 trials and is moving into Phases 2 and 3. Curevac is also recruiting 36,000 participants. A virus doesnt differentiate between nationality, sex, skin color, religion it is an international problem, that must be tackled internationally. He added that, I will say it in the words of Bill Gates he said it four years ago such outbreaks will happen again and again. The thing is we dont know what they will be and when they will come.

I think I learned more about RNA and mRNA writing this article than I did in school. And what Ive gathered from this is that the folks at Curevac the actual scientists making the vaccine know what they are doing and know what they need from Tesla. Tesla is helping and meeting that need, and has years of experience working with Curevac.

Tesla helping the vaccine would be the equivalent of me giving some copper wire to a friend who works in electrical engineering. I know nothing about the engineering work they do, but I use copper in my art and can get them some.

We dont know the type of benefit Tesla is receiving from Curevac, but we do know that Teslas contribution is something that will help save lives and create a valuable asset to the medical field. Actually, Elon Musk has stated before that Tesla isnt going to financially benefit from this. Emotionally, Teslas staff may benefit, especially if Curevacs prototype that Tesla is helping to create becomes a world-changing success in the medical industry what they are hoping it to be. And this, in my opinion, is why I believe Elon and Tesla are working with Curevac to help.

Im interested in things that change the world or that affect the future and wondrous, new technology where you see it, and youre like, Wow, how did that even happen? How is that possible? Elon Musk

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Tags: CureVac, Grohmann Engineering, Tesla, Tesla Grohmann, Tesla Grohmann Automation

Johnna Crider is a Baton Rouge artist, gem, and mineral collector, member of the International Gem Society, and a Tesla shareholder who believes in Elon Musk and Tesla. Elon Musk advised her in 2018 to Believe in Good.Tesla is one of many good things to believe in. You can find Johnna on Twitter

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History of the Partnership between Tesla & Curevac Its Older than Covid-19 - CleanTechnica

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AMRA Medical Research Reveals New MRI-based Data Connected to Risk for Coronary Heart Disease at The Liver Meeting Digital Experience – PRNewswire

November 24th, 2020 9:55 am

The researchers measured the visceral adipose tissue (VAT) and liver fat (LF) from magnetic resonance images of 12,276 participants from the UK Biobank, the researchers assessed and divided individuals into four groups defined by sex-specific median values of VAT and LF: low VAT-low LF, low VAT-high LF, high VAT-low LF, and high VAT-high LF. Participants were followed for 1.3 years (on average) to detect VAT-LF groups' associations with CHD incidence.

In total, 176 CHD events were recorded, revealing that high visceral adiposity increased risk for CHD; this effect was exacerbated in those who also had low liver fat elevating the risk for CHD greater than two-fold compared with low VAT-low LF. High liver fat in combination with low visceral adiposity (low VAT-high LF), did not increase the risk for CHD. After adjusting for age and body mass index, high VAT-high LF association with CHD diminished, but the increased risk of CHD among those with high VAT-low LF persisted.

"We believe knowledge of patient risk for disease is limited when assessment is restricted to single, isolated fat depots. When developing NAFLD treatments, a decrease in liver fat alone may not be sufficient to lower patients' cardiometabolic risk. In fact, the research shows that decreasing liver fat without resolving visceral obesity may put the patient at greater risk of heart disease. This is what we want to investigate further,"stated Jennifer Linge, Lead Scientist, Personalized Medicine at AMRA Medical.

The results suggest that heterogeneity of body fat distribution affects CHD risk. Specifically, risk for CHD increased among those with high visceral adiposity, which intensifies in the presence of low liver fat indicating that liver triglyceride regulation plays a vital role in cardiovascular health in the context of visceral obesity.

Learn more about these findings by viewing the recorded presentation. The corresponding abstract "Can Low Liver Fat Be Bad for Your Heart? The High Visceral Fat, Low Liver Fat Phenotype: A Risk Factor for Coronary Heart Disease" (number 89) can be found in the journal HEPATOLOGY.

About UK Biobank

UK Biobankis large-scalebiomedical database and research resource, containing in-depth genetic andhealth information from halfa million UK participants. The database, which isregularly augmented with additional data, is globally accessible to approvedresearchersand scientists undertaking vital research into the most common andlife-threatening diseases. UK Biobank's researchresource is a majorcontributor to the advancement of modern medicine and treatment and has enabledseveral scientific discoveriesthat improve human health.

About AMRA Medical

AMRAis a ground-breaking international digital health company at the forefront of medicalimaging and precision medicine. The company has developed a new global standard in bodycomposition assessment, the ability to automatically produce multiple fat and musclebiomarkers with unrivaled precision and accuracy, as well as contextual disease insights allfrom a single, rapid, whole-body MRI.

SOURCE AMRA Medical

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CHOP Researchers Reverse Severe Lymphatic Disorder in Patient with Noonan Syndrome by Targeting Genetic Pathway – BioSpace

November 24th, 2020 9:55 am

Precise treatment leads to resolution of patient's debilitating symptoms and complete remodeling of her lymphatic system

PHILADELPHIA, Nov. 20, 2020 /PRNewswire/ -- Researchers at Children's Hospital of Philadelphia (CHOP) have resolved a severe lymphatic disorder in a girl with Noonan Syndrome that had led to upper gastrointestinal bleeding, fluid collection around the lungs, and numerous surgeries that had been unable to resolve her symptoms. By identifying a genetic mutation along a pathway related to lymphatic vessel development and function, the research team was able to target the pathway using an existing drug they had used in a previous case to remodel a patient's lymphatic system.

The case study, which was published today in Pediatrics, describes a resolution of the patient's symptoms within three months while on the medication.

"This study is quite significant," said first author Yoav Dori, MD, PhD, Director of the Jill and Mark Fishman Center for Lymphatic Disorders at CHOP. "Inhibiting this pathway seems to have sweeping, widespread effects on the lymphatic system. How this process occurs is not fully understood, but is remarkable in its speed and breadth. This gives us a lot of hope for treating other patients with genetic mutations along this same pathway in the future."

The patient described in the paper, Maria, first came to CHOP when she was 14, after experiencing severe anemia due to upper gastrointestinal bleeding, as well as other symptoms including fluid build-up in the cavity around her lungs, chronic fatigue, delayed puberty, and difficulty gaining weight. Maria had been born with Noonan Syndrome, a genetic disorder that prevents normal development in various parts of the body and often results in short stature, heart defects and other physical problems, including an abnormal lymphatic system. Despite aggressive medical therapy elsewhere, Maria continued to bleed internally, and she underwent multiple blood transfusions to try to stabilize her health.

Within two days of transferring to CHOP, the lymphatics team, led by Dori, determined Maria had many lymphatic irregularities, which were leading to internal bleeding and lung problems, so they scheduled Maria's first intervention, a lymphatic embolization procedure that would seal the leaky vessels in her gut.

However, within two months of the procedure, Maria's gastrointestinal bleeding recurred. Over the following 8 months, she underwent two additional procedures, as well as a cauterization procedure to close off some of the blood vessels in her gut, but the benefits of each procedure lasted only about three months before the bleeding and her symptoms returned.

Based on whole exome sequencing done at CHOP's Center for Applied Genomics, the research team learned that Maria had a genetic mutation in the SOS1 gene, which operates along the RAS-MAPK pathway. This pathway involves mitogen-activated protein kinase (MEK), and Maria's mutation caused an overproduction of MEK, which resulted in the uncontrolled proliferation of her lymphatic vessels.

The research team had previously used a MEK inhibitor in another patient with a severe lymphatic disorder with great success. That patient had a mutation in the ARAF gene, which is also on the RAS-MAPK pathway. Within months of beginning treatment with trametinib, a MEK inhibitor, the patient saw a resolution of his symptoms and a complete remodeling of his lymphatic system.

Given that SOS1 operates on the same pathway as ARAF, Jean Belasco, MD, an oncologist in CHOP's Cancer Center who co-led the study, applied for compassionate use of the drug in Maria's case, given the lack of other treatment options.

"The success of trametinib in another patient with a mutation on the RAS-MAPK pathway encouraged us to try this approach, since other procedures and therapies continued to be unsuccessful," Belasco said. "Although we are in the early days of this type of personalized medicine, the hope is that by looking at patients' mutations, we can find more drugs and better care for patients with genetic diseases."

Within three months of starting the drug, Maria's vital signs stabilized. The bleeding stopped, her electrolyte, hemoglobin, and albumin levels returned to normal, and she began to gain weight. Maria's mother noticed that Maria wasn't going through periods of exhaustion anymore, and her pallor improved.

"She looks better than she's ever looked," her mother said. "She looks like a normal teenager. It's like night and day. She's also a lot happier. I think she knew deep down she was dying. The medicine gave her hope."

Hakon Hakonarson, Director of the Center for Applied Genomics and co-author of the paper, said that although Maria's SOS1 mutation is distinctly different than the ARAF mutation seen in the other patient, the drug was equally effective because it targets and blocks the function of MEK. He likened the scenario to a pathway where 15 events need to occur for a cell to function. Maria's SOS1 mutation might occur at step nine, whereas the ARAF mutation might occur at step three, but both genes are on a chain that ultimately passes through a tunnel that leads to phosphorylation and overactivity of MEK. Since both mutations were so-called gain of function mutations, MEK and thus lymphatic activity was overexpressed in both patients. The MEK inhibitor put the brakes on a system in overdrive.

"Remarkable advances in genetics have allowed us to uncover these mutations and cluster them into selective pathways and determine effective therapies based on genetic mutations with very high precision," said Hakonarson. "No one could have guessed that this drug would have worked for Maria without knowing the underlying genetics. This discovery is extremely important because Noonan Syndrome has the biggest patient population with alterations in MEK signaling. Not all Noonan patients will have mutations that respond to this therapy, but a very good number of them will."

He added that the treatment could also benefit patients with other genetic defects, though he noted the ongoing use of the drug treats the symptoms caused by these mutations, but does not fix the gene or cure the underlying condition.

"MEK inhibition has the potential to have significant effects on other organ systems affected by RAS-MAPK gene defects, such as the heart, eyes, skin and the coagulation system," Hakonarson said.

Hakonarson is also part of CHOP's Comprehensive Vascular Anomalies Program (CVAP), a CHOP Frontier Program that uses state-of-the-art genomics and personalized research strategies to determine the causes of complex vascular conditions and identify targeted therapies. The program works closely with the Lymphatic Imaging and Interventions Frontier Program, which is led by Dori. CHOP's Frontier Programs conduct cutting-edge research that translates into advanced clinical care. The CVAP, in particular, draws on the extensive clinical and genomic research capacity within the Cancer Center and Center for Applied Genomics.

Even with the success of the breakthrough treatment pioneered by these programs, it is not entirely clear why MEK inhibitors not only resolve patients' symptoms but also completely remodel their lymphatic systems. Hakonarson said one possibility is that when mutated genes cause uncontrolled growth of the lymphatic system, the body's vessels leak fluid everywhere in the body. When you shut down the unregulated growth, other homeostatic mechanisms that are balancing the system come into effect, so the overreactive cells that were growing out of control die and are replaced by normal cells that gradually build up the lymphatic system.

Whatever the mechanism, Maria's mother said her daughter had no hesitation at being the first patient with Noonan Syndrome to try this treatment to resolve a lymphatic issue.

"Maria saw the value from the beginning," she said. "She saw the value for herself, but she was also thinking of other Noonan kids, some of whom have passed away from lymphatic issues. She was willing and eager."

Dori et al. "Severe Lymphatic Disorder Resolved with MEK Inhibition in a Noonan Patient with SOS1 Mutation," Pediatrics, published online November 20, 2020, doi: 10.1542/2020-000123

About Children's Hospital of Philadelphia: Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, Children's Hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. In addition, its unique family-centered care and public service programs have brought the 564-bed hospital recognition as a leading advocate for children and adolescents. For more information, visit http://www.chop.edu

Contact: Natalie SolimeoChildren's Hospital of Philadelphia267-426-6246solimeon@chop.edu

View original content:http://www.prnewswire.com/news-releases/chop-researchers-reverse-severe-lymphatic-disorder-in-patient-with-noonan-syndrome-by-targeting-genetic-pathway-301177697.html

SOURCE Children's Hospital of Philadelphia

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Infections of the lung: a predictive, preventive and personalized perspective through the lens of evolution, the emergence of SARS-CoV-2 and its…

November 24th, 2020 9:55 am

This article was originally published here

EPMA J. 2020 Nov 13:1-21. doi: 10.1007/s13167-020-00230-1. Online ahead of print.

ABSTRACT

The long evolutionary battle between humans and pathogens has played an important role in shaping the current network of host-pathogen interactions. Each organ brings new challenges from the perspective of a pathogen to establish a suitable niche for survival while subverting the protective mechanisms of the host. Lungs, the organ for oxygen exchange, have been an easy target for pathogens due to its accessibility. The organ has evolved diverse capabilities to provide the flexibility required for an organisms health and at the same time maintain protective functionality to prevent and resolve assault by pathogens. The pathogenic invasions are strongly challenged by healthy lung architecture which includes the presence and activity of the epithelium, mucous, antimicrobial proteins, surfactants, and immune cells. Competitively, the pathogens in the form of viruses, bacteria, and fungi have evolved an arsenal of strategies that can over-ride the hosts protective mechanisms. While bacteria such as Mycobacterium tuberculosis (M. tuberculosis) can survive in dormant form for years before getting active in humans, novel pathogens can wreak havoc as they pose a high risk of morbidity and mortality in a very short duration of time. Recently, a coronavirus strain SARS-CoV-2 has caused a pandemic which provides us an opportunity to look at the host manipulative strategies used by respiratory pathogens. Their ability to hide, modify, evade, and exploit cells processes are key to their survival. While pathogens like M. tuberculosis have been infecting humans for thousands of years, SARS-CoV-2 has been the cause of the recent pandemic. Molecular understanding of the strategies used by these pathogens could greatly serve in design of predictive, preventive, personalized medicine (PPPM). In this article, we have emphasized on the clinically relevant evasive strategies of the pathogens in the lungs with emphasis on M. tuberculosis and SARS-CoV-2. The molecular basis of these evasive strategies illuminated through advances in genomics, cell, and structural biology can assist in the mapping of vulnerable molecular networks which can be exploited translationally. These evolutionary approaches can further assist in generating screening and therapeutic options for susceptible populations and could be a promising approach for the prediction, prevention of disease, and the development of personalized medicines. Further, tailoring the clinical data of COVID-19 patients with their physiological responses in light of known host-respiratory pathogen interactions can provide opportunities to improve patient profiling and stratification according to identified therapeutic targets.

PMID:33204369 | PMC:PMC7661834 | DOI:10.1007/s13167-020-00230-1

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Q&A: The Magic of University Entrepreneurship – UConn Today

November 24th, 2020 9:55 am

As one of the nations top 25 public research universities, UConn is home to many faculty and student inventors. The discoveries they make in the lab could some day become products and services that help society. They could also launch Connecticut companies that provide high paying jobs in the state. But its not always easy to think like an entrepreneur when youve been trained as an academic researcher.

Abhijit (Jit) Banerjee, Ph.D., MBA, is leading UConns mission to help more faculty and students participate in commercialization activities. Banerjee, who has extensive experience facilitating public-private partnerships, joined UConns Office of the Vice President for Research as Associate Vice President, Innovation and Entrepreneurship in May 2020. He leads Technology Commercialization Services, a group that specializes in transforming UConn inventions into products, services, and companies that help society.

Our efforts to bring the fruits of our research to the world are tremendously strengthened by Jits arrival at UConn, says President Thomas Katsouleas. His background, skills, and passion for the work are going to help us become even more prolific and successful in demonstrating the value of the world-class research being done at UConn.

Banerjee recently answered a few questions about becoming a Husky during a pandemic and the importance of innovation to tackle the worlds great challenges.

Connecticut has a rich history of innovation and invention. How does UConn contribute to this legacy? What do you think the University can do to help the states economic health now and in the future?

The innovation engine runs 24/7, 365 days at UConn. The Universitys 14 schools and colleges are filled with world-class faculty and students who are building on UConns strong legacy of innovation. Our footprint isnt limited to the local geography either. UConn has a statewide impact, with programs like the UConn Technology Incubation Program (TIP), a flagship program that supports UConn companies as well as external ventures. TIPs three locations in Storrs, Farmington, and the soon-to-be-launched Stamford site support the states economy because it grows and attracts companies that want to locate in Connecticut and will create jobs here. Another example is the Innovation Partnership Building at UConn Tech Park, which is a world-class engineering living lab that is an asset to the states engineering and manufacturing industry.

With all of these hubs of innovation, I am convinced that the University still has too many well kept secrets for businesses and citizens around our state. We have expert faculty in every field, talented students to bolster the states innovation workforce, and unparalleled facilities to help solve problems and make breakthroughs for any industry sector, from manufacturing to personalized medicine.

In a fiercely competitive national environment where every state is aggressively looking to expand their funding and improve their economic health, success comes when the state, its agencies, and major innovation engines like UConn work hand-in-hand. I plan to do everything I can to help UConn continue our collaborative approach to support state economic development goals.

In your newly created role within the Office of the Vice President for Research, you are responsible for leading UConns efforts to enhance entrepreneurship, innovation, and technology commercialization for UConn companies and external ventures. How do you and your team support these efforts?

The Technology Commercialization Service (TCS) team is responsible for protecting intellectual property that is generated at UConn and bringing inventions from bench to marketplace or bedside through commercialization. A major focus for TCS is fostering an entrepreneurial ecosystem that enables faculty and students to pursue commercialization of their discoveries whether it be through licensing to existing companies or forming startups. The TCS team has dedicated and passionate individuals who are deeply committed to the University and all faculty, regardless of the discipline.

As Ive met with leaders at each school and college, it has become clear that there is an opportunity for TCS to take much bigger and strategic roles across the University, and we are excited to take that on.

How has the pandemic affected innovation, in your opinion? What challenges and opportunities does it provide for faculty and student innovators?

The impact of COVID-19 on global well-being is now being felt in every sphere, from mental health to the economy to education. This pandemic has taught us to quickly adapt, pivot, and rely on both instincts and innovation to get through this and survive the new normal.

As the world collectively works to find a vaccine for this pathogen, we shouldnt stop innovating and seeking solutions for other major challenges we still face. Climate change, wild fires, tsunami, and hurricanes are impacting both human lives and our ecosystem. As we know, any slight imbalance in this ecosystem can cause disasters which are often irreversible. There are thousands of other conditions that still need effective treatments. We can still do more to develop innovative technologies that support the defense sector. For all of these problems, science, innovation, and commercialization can help find solutions. I am confident UConn inventors will be part of the solution and TCS is here to support them on that path.

You joined UConn while the University was still in the middle of the COVID-19 ramp down. How do you like being a Husky so far during these unprecedented times?

COVID-19 has changed the way we conduct business and interact, and for the unforeseen future, we need to accept this as the new normal. Our individual responsibility to stay healthy and follow guidance from scientific experts to reduce risk of COVID-19 exposure and infection adds another layer of complexity. From what Ive seen so far, at the individual and organizational level, UConn Nation is pivoting and adapting to continue working towards our educational, research, and service goals.

But I have to admit that even before I came to UConn, I felt an affinity for Husky Nation, since our family dog was a Siberian Husky named Mishi. We recently lost him to cancer, but he brought out the best in us as humans and I can see that same core value in everyone Ive met so far at UConn.

To learn more about UConn technologies and startups, visit http://www.innovation.uconn.edu

Follow UConn Research on Twitter & LinkedIn.

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CDMOs Are the Unsung Heroes of the Gene and Cell Therapy… – Labiotech.eu

November 24th, 2020 9:53 am

Cell and gene therapy is booming amid the ongoing pandemic, and this is driving up demand for synthetic DNA products, often produced and delivered by CDMOs.

Karen Fallen started her career at Celltech Biologics in the UK, which was acquired in 1996 by Swiss giant Lonza, a world leader in the pharmaceutical manufacturing space. She stayed there for 31 years before moving on to the role of CEO at Touchlight DNA Services in October.

The market in DNA and nucleic acid medicine is just exploding. Cell and gene therapies have the biggest growth of all the biologics out there and DNA is the essential starting material, Fallen told me.

For several years, UK-based company Touchlight Genetics has been producing a form of synthetic DNA, called doggyboneDNA, that is designed to simplify the manufacturing of DNA vaccines and gene therapy.

To meet the ever-increasing demand for its products, Touchlight Genetics created a contract development and manufacturing organization (CDMO) arm of the business called Touchlight DNA Services, which Fallen now leads.

Traditionally, the DNA necessary for these treatments has been produced by bacteria. Touchlight is developing a method to create this DNA enzymatically, which removes the need to include certain genes required by the bacteria and thus makes the final product simpler and safer.

Its really quick to actually produce the DNA, much faster than the bacterial methodology of doing it, Fallen noted. Its also very scalable.

The increasing need for fast, cheap and accurate DNA synthesis in recent years has led a number of biotechs in Europe to try and meet the demand, such as DNAScript, Evonetix, Nuclera, and Camena Bioscience, among others.

The demand has further increased with the Covid-19 pandemic, since many of the companies developing vaccines or treatments for Covid-19 require DNA as a starting material. So do many tests to diagnose Covid-19 infections.

Theres a lot of potential RNA and [DNA] vaccines out there, Fallen said. The demand for DNA was already outstripping supply, and that was just with cell and gene therapy demands. The Covid-19 impact has just accentuated that.

While CDMOs are crucial to the drug development industry, they often dont receive the same level of public and media attention as biotechs and big pharma. But Fallen thinks their importance is only increasing as cutting-edge treatments become commonplace, as many biotechs working on advanced therapies do not have the capacity for advanced manufacturing.

I think that the CDMO industry over the last few years has really come into its own At least 70% of the innovator molecules out there reside with biotechs that dont always have the capability to develop or manufacture them, Fallen told me.

The supply chain is imperative. Its all very well developing a molecule, but if you cant manufacture it or deliver it, then youre going to go nowhere.

Starting a new job as a CEO is a challenge for most people, but particularly so at the moment with the pandemic hanging over everyone and most people working completely remotely.

I met all the people by Zoom, which isnt often the easiest thing to do. But that gives you an idea of the culture of the company.

Thanks to her experience running a business unit at Lonza, Fallen has learned a lot about how to be a good leader. Ive always found that people follow you because they want to, not because youre in a position of power You need to listen to people, see what motivates them, and then you can get people on board.

Youve also got to make tough decisions quickly as well. You cant have an endless amount of data to make decisions on, youve just got to go with it and have the confidence to do that.

Fallen is a strong believer in supporting others. I have become, surprisingly, even to myself, a role model. I spent a fair amount of time talking with some of the more junior people [at Lonza] mentoring them and giving them my perspective on the world. I have shared what Ive learned as Ive been going along and given some advice on how to handle certain situations.

Although she has been lucky in that respect, Fallen recognizes that diversity can be a problem in CDMOs, biotechs, and pharma companies, particularly at higher management levels.

Youve got to try and get rid of unconscious biases. I know a lot of companies are putting people into positions just to get the numbers up, but Im not convinced thats the right way of doing it. I think you just have to have an open mind and give people the opportunity. From my experience, that is mostly successful.

Youve got to take a risk with people sometimes. Ive been in that position of people taking risks with me. Youve got to then support them, youve got to develop them in the right way, dont just talk them into something. But if you do that, I think you get what you want out of people.

Cover illustration by Elena Resko, picture provided by Touchlight DNA Services.

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Health Beat: Boy saved by groundbreaking gene replacement therapy – WFMZ Allentown

November 24th, 2020 9:53 am

SALT LAKE CITY - No doubt about it, Cinch Wight is going to be a cowboy just like his dad.

"He loves the dog and the horses and the cows," shared Cinch's dad, Alex Wight.

But it has been a wild ride for this young bronco. A mandatory newborn screening test at birth revealed Cinch had spinal muscular atrophy, or SMA.

"That was the first time I'd ever even heard the term and what it was, and so it was very scary," recalledCinch's mom, Amber Wight.

A neuromuscular disorder can paralyze a baby in the first few weeks of life.

"My first thought was he's never going to be able to ride broncs or anything like that," expressed Alex.

But just one day after Cinch was born, the FDA approved a new gene therapy.

"We were pretty excited to get a phone call from the department of health, you know, and have this baby here who we can use this treatment on after its approval," explained Dr. Russell Butterfield, a pediatric neurologist at University of Utah Health and Intermountain Primary Children's Hospital.

A critical gene in little Cinch was missing. Butterfield used an infusion to deliver a virus carrying a new copy of the gene into Cinch's nerve cells.

"It's like a delivery truck to deliver genes to where you want them to go. What that does do is it stops the disease right where it is," Butterfield explained.

Just a few years ago, most children born with SMA didn't make it to their second birthday. Now?

"The hardest is holding a baby in one hand and holding that drug in the other and really feeling the weight of that, and understanding that how different this child's life will be with this new medicine," Butterfield said.

It took a lot of courage for this family to get this far. That's why Alex wrote a book for his son, a true story about how real cowboys never give up.

"I wanted to let him know that no matter how hard it gets, as long as he keeps going, he'll be all right," shared Alex.

Doctors don't know if the one-time infusion will last a lifetime or will have to be repeated, and there could be a possible risk of inflammation to the liver that doctors will closely monitor.

The gene replacement therapy costs $2.1 million. Insurance paid for most of it, but Alex hopes sales from his children's book will help pay the rest. You can find the book, "A Cowboy and His Horse," on Amazon.

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Singapore: Public consultation on Proposed Regulations of Cell, Tissue and Gene Therapy Products – Lexology

November 24th, 2020 9:53 am

In brief

From 6 to 27 November 2020, the Health Sciences Authority (HSA) will be carrying out a public consultation seeking feedback on the proposed Health Products Act ("Amendment of First Schedule") Order 2020 ("HPA Amendment Order"), and the proposed Health Products (Cell Tissue and Gene Therapy Products) Regulations 2020 ("CTGTP Regulations") (collectively, the "Proposed Regulations"), which seek to regulate cell, tissue and gene therapy products (CTGTP). The Proposed Regulations seek to flesh out (i) a definition of CTGTP, (ii) a risk-based regulatory approach for CTGTPs, and (iii) introduce requirements unique to CTGTP.

Comments

CTGTP are a notably emerging and cutting edge field of medicine, providing great potential for the development of new treatment opportunities. Apart from being able to engineer the growth of healthy and functional tissues to reconstruct, regenerate or repairdamaged tissues or organs, CTGTP may also be used in therapies to treat or cure disease through the modification of individual genes.

The HSA has noted that the CTGTP regulations are reportedly intended to facilitate patient access to therapies that meet appropriate safety, efficacy and quality standards, and to provide a fit-for-purpose regulatory framework to support product development and facilitate product commercialisation. Given the novelty of this legislation, it may be beneficial for businesses to participate in the public consultation given their expertise and experience in this area of medicine. This could also assist toensure that any eventual laws appropriately account for the unique nature of CTGTP while not being excessively onerous on suppliers, importers, and manufacturers.

In more detail

From 6 to 27 November 2020, the Health Sciences Authority (HSA) will be carrying out a public consultation seeking feedback on the proposed Health Products Act ("Amendment of First Schedule") Order 2020 ("HPA Amendment Order"), and the proposed Health Products (Cell Tissue and Gene Therapy Products) Regulations 2020 ("CTGTP Regulations") (collectively, the "Proposed Regulations"), which seek to regulate cell, tissue and gene therapy products (CTGTP) as a new category of health products. The Proposed Regulations are intended to provide (i) a definition of CTGTP, (ii) a risk-based regulatory approach for CTGTPs, and (iii) introduce requirements unique to CTGTP.

Part (i) will be addressed via the HPA Amendment Order, which seeks to insert the following definition of CTGTP under the Health Products Act: "a substance consisting of human cells or tissues that are either autologous obtained from the same individual) or allogeneic (obtained from another individual), viable animal cells or tissues, or recombinant nucleic acids (i.e. modified DNA or RNA as carriers of a therapeutic gene); that is intended for diagnosis, treatment or prevention of any human disease or medical condition".

The CTGTP Regulations in turn, addressparts (ii) and (iii). The proposed regulatory framework is generally split into the following areas:

The draft HPA Amendment Order and CTGTP Regulations can be foundhereandhere. Feedback on the regulations may be providedhere.

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Singapore: Public consultation on Proposed Regulations of Cell, Tissue and Gene Therapy Products - Lexology

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Genenta to Appoint Stephen Squinto, Experienced Biotech Executive and Investor, as Chairman – BioSpace

November 24th, 2020 9:53 am

MILAN, Italy and NEW YORK, Nov. 23, 2020 (GLOBE NEWSWIRE) -- Genenta Science, a clinical-stage biotechnology company pioneering the development of a hematopoietic stem progenitor cell gene therapy for cancer (Temferon), announced that highly experienced biotech executive and investor Stephen Squinto, PhD joint Genenta and will be appointed as Chairman of its Board of Directors.

The appointment will be effective with the approval of the Genenta Shareholders Meeting. Pierluigi Paracchi, currently Chairman and Chief Executive Officer of Genenta, will continue to serve as CEO.

Dr Squinto is currently Executive Partner of the healthcare investment company OrbiMed Advisors and has more than 25 years experience in the biotech industry, including as Chief Executive Officer of the gene therapy company Passage Bio. He was a co-founder of Alexion Pharmaceuticals, where he served as Chief Global Operations Officer and Global Head of Research, and previously held several senior leadership positions at Regeneron Pharmaceuticals.

Dr Squinto currently serves on the Board of Directors of a several biotech and healthcare companies and has received numerous honors and awards from academic and professional organizations for his scientific work. He received his PhD in Biochemistry and Biophysics from Loyola University of Chicago.

Pierluigi Paracchi, CEO of Genenta, said: I am delighted to welcome Steve Squinto to Genenta as our new Chairman. Steve will provide valuable input and guidance to the development on Genenta, based on his outstanding and extensive time in biotech industry. In particular, his time as CEO of the gene therapy company Passage Bio provides a clear parallel to Genenta, as we continue to develop the stem cell gene therapy Temferon.

Stephen Squinto said: It as honor to be invited to be Chairman of Genenta, a truly exciting company which has the potential to revolutionize the way we treat cancer through its novel immuno-gene therapy Temferon. I am looking forward to working with the outstanding team already in place to progress this treatment, which has potential against a broad range of tumors both as first line and as combination therapy, further through clinical trials and towards market.

About Genenta Science

Genenta (www.genenta.com) is a clinical-stage biotechnology company pioneering the development of a proprietary hematopoietic stem cell gene therapy for cancer. Temferon is based on ex-vivo gene transfer into autologous hematopoietic stem/progenitor cells (HSPCs) to deliver immunomodulatory molecules directly via tumor-infiltrating monocytes/macrophages (Tie2 Expressing Monocytes - TEMs). Temferon, which is under investigation in a Phase I/II clinical trial in newly diagnosed Glioblastoma Multiforme patients, is not restricted to pre-selected tumor antigens nor type and may reach solid tumors, one of the main unresolved challenge in immuno oncology. Based in Milan, Italy, and New York, USA, Genenta has raised 33.6 million (~$40 million ) in three separate rounds of financing.

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Genenta to Appoint Stephen Squinto, Experienced Biotech Executive and Investor, as Chairman - BioSpace

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Orchard Therapeutics to Present at Piper Sandler 32nd Annual Virtual Healthcare Conference – GlobeNewswire

November 24th, 2020 9:53 am

BOSTON and LONDON, Nov. 24, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that its chief executive officer, Bobby Gaspar, M.D., Ph.D. will participate in a pre-recorded fireside chat as part of the Piper Sandler 32nd Annual Virtual Healthcare Conference. Management will also be available for one-on-one meetings on Tuesday, December 1, 2020.

The fireside chat is available for on-demand viewing under "Events" in the Investors & Media section of the company's website at http://www.orchard-tx.com and will remain available for approximately 90 days.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (TwitterandLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Contacts

Investors

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

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Orchard Therapeutics to Present at Piper Sandler 32nd Annual Virtual Healthcare Conference - GlobeNewswire

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Reactions from the 2020 SYNGAP1 Scientific Conference – Spectrum

November 24th, 2020 9:53 am

Bridging the gap: SYNGAP1 protein is located mostly at synapses, the junctions between neurons (green).

Spectrum is covering the 2020 International SYNGAP1 Scientific Conference, which took place virtually because of the coronavirus pandemic. Here were highlighting researchers reactions to noteworthy presentations.

Drug test: A new assay allows researchers to test thousands of candidate drugs for their ability to boost expression of the autism gene SYNGAP1. The tool may help researchers identify and screen potential treatments for people with mutations that silence the gene. Gavin Rumbaugh, professor of neuroscience at Scripps Research in Jupiter, Florida, presented the unpublished results on 18 November.

The assay uses neurons from mice with one intact and one mutated copy of SYNGAP1. The researchers genetically engineer the mice so that SYNGAP1 protein made from the intact copy is tagged with luciferase the enzyme that gives fireflies their glow.

They then grow these neurons in tiny wells and add a different candidate drug to each well. The amount of SYNGAP1 protein in the dish gives a proportionate amount of light in your well, Rumbaugh says.

Rumbaugh and his team plan to use the platform to run through more than 100,000 different experimental compounds in 2021, he says.

Thats going to be really exciting for drug discovery efforts for SYNGAP1. I think thats going to be a game changer, says Karun Singh, senior scientist at the University Health Network in Toronto, Canada, who was not involved in the work.

It will be very exciting to see if they are able to uncover any useful hits with their novel approach, says Helen Bateup, associate professor of neurobiology at the University of California, Berkeley, who was not involved in the work.

Treatment across ages: A leading theory of autism is that the condition is characterized by a signaling imbalance: too much excitation or too little inhibition in the brain. One of the key players in creating this imbalance is thought to be inhibitory interneurons, which employ the neurotransmitter gamma-aminobutyric acid (GABA). And mutations to SYNGAP1 may disrupt GABAs function, said James Clement, assistant professor of neuroscience at the Jawaharlal Nehru Centre for Advanced Scientific Research in Bangalore, India, in a presentation on 18 November.

GABA is excitatory early on in brain development and inhibitory later on a switch that seems to be impaired in mice with SYNGAP1 mutations, he says. He and his team have tested a new compound that restores the GABA switch in mice and eases almost all SYNGAP1-related traits including seizures, learning issues and motor impairment in the mice. It works in newborn and adolescent mice. Due to a pending patent application, Clement and his lab are not revealing the compounds name.

I think its important to test efficacy at multiple ages, as they have done, to understand which phenotypes or problems can be improved with early treatment and which might still be responsive to treatment even if its administered later in life, says Bateup, who was not involved in the work. The idea that GABA may remain depolarizing for longer in SYNGAP1 mutant mice is quite interesting.

Clements lab was the only other lab that was presenting at this meeting that presented data from a very early age, says Shilpa Kadam, associate professor of neurology at the Kennedy Krieger Institute in Baltimore, Maryland, who was not involved in the work.

Motor coordination: For mice, the loss of SYNGAP1 function in the striatum impairs their goal-directed learning and seems to lead to inflexible behavior, Bateup said in a presentation on 18 November.

Helen Bateups work looking at striatal function as it relates to motor coordination and motor learning is also pretty exciting and may shed light not only on the motor-coordination difficulties but also the repetitive or habitual motor behaviors, says Constance Smith-Hicks, child neurologist and research scientist at the Kennedy Krieger Institute, who was not involved in the work.

Bateups presentation also demonstrated that SYNGAP1 deletion seems to affect neurons differently depending on which type of dopamine receptor they express.

We know SYNGAP1 is at most excitatory synapses, so why shes seeing some functional effects in one type of cell and not the other, I find that interesting, says Richard Huganir, professor of neuroscience and psychological and brain sciences at Johns Hopkins University in Baltimore, Maryland, who was not involved in the work.

Its exciting to be able to kind of pinpoint which pathway might be involved and get a better understanding of the circuits that are disrupted, says Singh, who was not involved in the work.

Protein levels: People with a nonfunctional copy of SYNGAP1 have about half the typical amount of SYNGAP1 protein. Increasing the activity of the intact copy of the gene could help restore typical functioning, Huganir said in a presentation on 18 November.

He and his team tested this idea on two unique mouse models in unpublished work. Instead ofhaving one intact and one missing copyof the SYNGAP1 gene, as is typical for SYNGAP1 mouse models, each mouse model carriesone intact copy of the gene and one with a mutation seen in people. Both mice produce about half the typical amount of SYNGAP1 protein and show the same behaviors as the classical knockout mouse, despite having different types of mutations.

These new mouse models are crucial because they can directly correlate to what is happening in the humans, says Clement, who was not involved in the work.

Huganir and his team are testing different types of gene therapies to increase SYNGAP1 protein up to the typical levels, and have found that there are two SYNGAP1 protein isoforms, or slight variations of the protein.

One of the isoforms can restore synaptic plasticity in the animal model for SYNGAP1, so I think thats really exciting because even though theres multiple isoforms, it seems that one might be more important from a gene therapy point of view, says Singh, who was not involved in the work. Its pretty exciting to have a specific target now.

Read more reports from the2020 International SYNGAP1 Scientific Conference.

Link:
Reactions from the 2020 SYNGAP1 Scientific Conference - Spectrum

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Exploring First-line Therapies in Peripheral T-Cell Lymphomas – Targeted Oncology

November 24th, 2020 9:52 am

During the Targeted Oncology Case Based Peer Perspectives event, Javier L. Munoz, MD, Hematologist/Oncologist Director, Lymphoma Program Mayo Clinic, discussed the case of 60-year-old patient with peripheral T-cell lymphoma (PTCL).

Targeted Oncology: What are the standard regimens in treating T-cell lymphoma?

MUNOZ: Historically, we have been prescribing CHOP [cyclophosphamide, doxorubicin hydrochloride, vincristine sulfate, and prednisone] or CHOP-like regimens. There are retrospective German data showing the addition of etoposides, so CHOEP instead of CHOP, may be beneficial, particularly for patients who were younger than 65 years.1 That is not a randomized trial, it was a retrospective study, so the strength of the data was relatively weak.1 The data of brentuximab vedotin [Adcetris] plus CHP [cyclophosphamide, doxorubicin, prednisone] versus CHOP [was from] a randomized trial, so its a stronger level of evidence compared with that etoposide data.

What do the National Comprehensive Cancer Network (NCCN) guidelines recommend?

The category 1 recommendation for ALCL [anaplastic large cell lymphoma] is brentuximab vedotin plus CHP, according to the NCCN.2 For that particular indication, I think most people are not going to argue that brentuximab plus CHP is the way to go.

For the other subtypesand numbers for the other subtypes were smaller compared with ALCLits a bit more blurry. You still have brentuximab vedotin plus CHP, but you also have the options of CHOP, CHOEP.

What was the rationale for ECHELON-2 [NCT01777152] in this population? This was a randomized study, brentuximab vedotin plus CHP versus CHOP.3 The bottom line is that this trial was a replacement strategy. Vincristine, or Oncovin, is a vinca alkaloid, and it causes neuropathy. For brentuximab, an antibody-drug conjugate, its payload is also a vinca drug. Vinca alkaloids cause neuropathy, so the trial replaced those agents, resulting in this trial. It was a double-dummy, [double-blind] study, so patients received placebobrentuximab and CHP or they received CHOP. The CD30 expression needed to be 10% or higher for the patient to be able to enroll. The investigators allowed multiple subtypes of T-cell lymphomas: PTCL not otherwise specified, angioimmunoblastic T-cell lymphoma, hepatosplenic T-cell lymphoma, enteropathy-associated T-cell lymphoma.

The reality is that most of you are going see PTCL, not otherwise specified, because its the most common subtype I see; maybe the runner-up is angioimmunoblastic T-cell lymphoma, and then the next one is going to be ALCL. And again, there are a couple of flavors: ALK-positive and ALK-negative. ALK-negative is, prognostically, closer to PTCL. The primary end point was progression-free survival [PFS] but, of course, they looked at other things like overall survival.3

What were the outcomes of this trial for patients? PFS was a primary end point, and this was the most important factor or variable that they looked at, and with CHOP you get 20.8 months of PFS; with brentuximab plus CHP you got 48.2 months. So, more than double the primary end point, and that is why I like to start with PTCL, because ECHELON-2 definitely looked more impressive than the ECHELON-1 trial [NCT01712490]. Theyre both important papers; ECHELON-1 was published in New England Journal of Medicine,4 ECHELON-2 was only published in Lancet. The difference, the numbers that you see, are more dramatic in ECHELON-2 in PTCL compared with Hodgkin lymphoma, but both are relevant papers. More than double the results, and you could see that that separation is maintained over time.

They looked at overall survival, and there was also a difference. Even though the primary end point was PFS, your patients are also going to live longer if you go with brentuximab plus CHP.

The complete response rate was 68% for [brentuximab vedotin plus CHP] versus 56% for CHOP. Of course, the objective response rate is also higher for brentuximab plus CHP, as well [TABLE5 ].

As I said, both drugs can potentially cause neuropathy. Lets take a look at the safety profile. Peripheral neuropathy for brentuximab plus CHP, grade 3 or higher, was 4%, for CHOP, it was 3%. Remember, vincristine in the CHOP regimen can also cause neuropathy. Not a big difference there. For any grade of neuropathy, the numbers are 45% for brentuximab plus CHP and 41% for CHOP.

Now, could you die from CHOP, or brentuximab plus CHP? Of course. Luckily for us, its single digits. Its rareIm sure that we have all had patients that we have lost during CHOP or R-CHOP [rituximab (Rituxan) plus CHOP] for B-cell lymphomas, but 3% for brentuximab plus CHP and 4% for CHOP.

If this patient achieved a complete response to frontline therapy, would you consider transplant?

Before the ECHELON trials, most of us knew that CHOP was suboptimal; we just did not know what was better than CHOP. Thats why we were playing with etoposide, based on that retrospective German data and doing CHOEP, sometimes even EPOCH [etoposide phosphate, prednisone, vincristine sulfate, cyclophosphamide, and doxorubicin hydrochloride] for these patients. Because CHOP was suboptimal, we wanted to do something additional, and that is why many patients have received consolidation with transplants.

We consider so many factors for transplant-eligible patientsthe performance status, comorbidities, age; but in general, Im prone to offer [transplant] to patients who have these T-cell lymphomas because, again, its a bad disease, and its not a curative intent. You are not giving the treatment with a curative intent. You want to keep the disease at bay as much as possible.

What is the impact of consolidative stem cell transplant after frontline brentuximab plus CHP for patients?

In a post hoc analysis, for the patients who achieved complete remission [CR], 38 patients in CR, received the transplant, and 76 patients in CR did not; it was certainly not the primary end point.5 It was presented at the American Society of Hematology Annual Meeting in 2019. Again, we are tempted to prescribe the transplant because, historically, these patients have done so poorly. The majority of these patients in CR after brentuximab plus CHP did not receive a transplant, 38 patients did receive a transplant, and it seems that the patients that received the consolidation with the transplant did better than the patients who did not receive the transplant. You have the 3-year progression-free survival numbers: 76% and 53%, though my bias at this point would be young, relatively young, or with few comorbidities, good performance statusI would certainly send the patient to get a second opinion.

What would be your approach toward second-line therapy at progression?

The number 1 option is a clinical trial. Id also consider pralatrexate [Folotyn]. Belinostat [Beleodaq], pralatrexate, and romidepsin [Istodax] are going to give an overall response somewhere between 20% to 30%, and complete remissions that are approximately 10%, give or take. Duration of response is usually going to be less than a year. Bottom line, we need to do better for PTCL when its relapsed/refractory. We need to do good when it comes to our frontline therapies, so, hopefully, we can provide a long period of time of remission for our patients.

References:

1. Schmitz N, Trmper L, Ziepert M, et al. Treatment and prognosis of mature T-cell and NK-cell lymphoma: an analysis of patients with T-cell lymphoma treated in studies of the German high-grade non-Hodgkin lymphoma study group. Blood. 2010;116(18):3418- 3425. doi:10.1182/blood-2010-02-270785

2. NCCN. Clinical practice guidelines in oncology. T-cell lymphomas, version 1.2021. Accessed October 14, 2020. https://bit.ly/3kcUHLL

3. Horwitz S, OConnor OA, Pro B, et al; ECHELON-2 Study Group. Brentuximab vedotin with chemotherapy for CD30-positive peripheral T-cell lymphoma (ECHELON-2): a global, double-blind, randomised, phase 3 trial. Lancet. 2019;393(10168):229-240. doi:10.1016/S0140-6736(18)32984-2

4. Connors JM, Jurczak W, Straus DJ; ECHELON-1 Study Group. Brentuximab vedotin with chemotherapy for stage III or IV hodgkins lymphoma. N Engl J Med. 2018;378(4):331-344. doi: 10.1056/NEJMoa1708984

5. Savage KJ, Horwitz SM, Advani RH. An exploratory analysis of brentuximab vedotin plus chp (A+CHP) in the frontline treatment of patients with CD30+ peripheral T-cell lymphomas (ECHELON-2): impact of consolidative stem cell transplant. Blood. 2019; 134(suppl 1):464. doi:10.1182/blood-2019-122781

Link:
Exploring First-line Therapies in Peripheral T-Cell Lymphomas - Targeted Oncology

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