Category Archives: Gene Medicine

Pune, India, April 08, 2021 (GLOBE NEWSWIRE) -- According to our new research study on Microplate Reader Market Forecast to 2027 COVID-19 Impact and Global Analysis by Type, Application, and End User the Microplate Reader Market was valued at US$ 534.5 million in 2019 and is projected to reach US$ 704.4 million by 2027; it is expected to grow at a CAGR of 3.6% during 20202027. The high cost of advanced microplate readers is the major factor hindering the market growth.

Microplate Reader Market: Key InsightsIn 2019, North America dominated microplate reader market with more than one-third revenue share. The increase in the demand for advanced and quick processing technologies by biopharmaceutical and biotechnology companies; presence of key market players and their increasing number of product lunches; extensively expanding research activities in various fields of life sciences, such as genomic and proteomics research and drug discovery; and rise in clinical diagnostics conducted by various academic and research institutes are the prime factors contributing to the market growth in this region.

Pharmaceuticals R&D is gaining traction worldwide owing to the rising focus on drug discovery. The US biopharmaceutical industry has been the forefront of the development of new therapeutics worldwide. As per the Pharmaceutical Research and Manufacturers of America (PhRMA), the biopharmaceutical industry invested US$ 83 billion in R&D in 2019. The increasing demand for new and advanced approaches in drug discovery is bolstering the adoption of microplate readers.

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Various companies are developing microplate readers for the drug discovery process to make the research easier and faster. For instance, BioTek Instruments, Inc. offers Synergy HT, a unique multi-detection microplate reader, for high-throughput screening (HTS) and drug discovery. Moreover, pharmaceutical companies are investing heavily in the drug discovery, gene sequencing, and targeted protein development to meet the growing demands of the healthcare sector. Understanding the proteindrug interactions is a crucial and vital parameter in drug discovery.

Further, the rise in personalized medicine development activities is propelling the demand for genetic studies, which, in turn is likely to contribute to the growth of the microplate reader market. The field of precision medicine requires genomic information of every individual to offer targeted treatment for a particular indication. The increasing prevalence of chronic diseases has increased the demand for biologics, orphan disease drugs, and personalized medicines. For instance, in 2019, the FDA approved 12 personalized medications to address the root cause of disease and integrate precision medicines with clinical care. Thus, increasing government efforts are strengthening personalized medicine research activities.

Microplate Reader Market: Segmental OverviewOn the basis of type, the microplate reader market is segmented into single-mode microplate readers and multi-mode microplate readers. The single-mode microplate readers segment held a larger market share in 2019, while the multi-mode microplate readers segment is anticipated to register a higher CAGR in the market during the forecast period. The market share of multi-mode microplate readers segment is estimated to increase exponentially in the coming years owing to their ability to detect two or more applications. Most of the microplate readers can be upgraded to powerful multi-mode microplate readers, on demand. Moreover, the multi-mode microplate readers allow supplementary detection modes, reagent injectors, and other features to be upgraded at any time.

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On the basis of application, the microplate reader market is segmented into genomics and proteomics research, drug discovery, clinical diagnostics, and other applications. The drug discovery segment held the largest market share in 2019; however, the clinical diagnostics segment is anticipated to register the highest CAGR in the market during the forecast period. The projected growth of the market for clinical diagnostics segment is mainly attributed to the fact that microplate readers offers a broad portfolio of genetic tests with well-known applications such as non-invasive prenatal testing (NIPT), hereditary cancer screening, cardiovascular disease predisposition testing, rare diseases diagnosis, and personalized medicine applications, further allowing patients and their care providers to select individualized treatment based on their genetic and metabolic profiles.

On the basis of end user, the microplate reader market is segmented into hospital and diagnostic laboratories, biotechnology and pharmaceutical companies, and research and academic institutes. The biotechnology and pharmaceutical companies segment held the largest share of the market in 2019, and it is further expected to register fastest CAGR during the forecast period. The future growth of the market for this segment is ascribed to the ability of microplate readers to improve the laboratory processes in pharmaceutical industries by utilizing applications that detect samples in seconds. Moreover, along with the increased detection speeds, the upgradable choices are automated for more effortless operation and workflow.

Microplate Reader Market: Competition Landscape & Key DevelopmentsBiotek Instruments, Inc.; Hercuvan Lab Systems; Molecular Devices, Llc; Thermo Fisher Scientific; Bmg Labtech; Promega Corporation; Perkinelmer Inc; Bio-Rad Laboratories, Inc.; Tecan Trading Ag; and Lonza are key companies operating in the microplate reader market. The market leaders are continuously looking forward to expanding and diversifying their market presence and reaching out to new customers, thus tapping prevailing business opportunities.

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In January 2019, BioTek Instruments introduced next-generation Epoch 2 Microplate Spectrophotometer, with added features that allow convenient absorbance measurements.

In June 2018, Molecular Devices announced the launch of SpectraMax ABS and ABS Plus Microplate Readers, encompassing a wide range of absorbance-based applications such as ELISAs, microbial growth, and protein quantitation. These products can be easily integrated into full robotic systems.

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Microplate Reader Market Value Worth $704.4 Million by 2027, Says The Insight Partners - Impact of COVID-19 Pandemic and Global Analysis -...

STRASBOURG, France--(BUSINESS WIRE)--Regulatory News:

Transgene (Paris:TNG) (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapeutics against cancer, today announces initial promising results from a Phase I study combining intravenous (IV) oncolytic virus TG6002 and oral 5-FC in patients with advanced gastrointestinal carcinomas. These data provide a clinical proof of concept for Transgenes double deleted VVcopTK-RR- patented virus backbone: after IV administration, TG6002 reached the tumor, multiplied within tumor cells, and induced the local expression of its payload (the FCU1 gene).

These results will be presented at the American Association for Cancer Research (AACR) virtual meeting taking place from April 10-15, 2021.

DATA CONFIRM THAT THE CHEMOTHERAPY AGENT 5-FU IS PRODUCED IN PATIENTS TUMORS AFTER INTRAVENOUS ADMINISTRATION

TG6002 is a novel oncolytic virus that has been engineered to combine multiple mechanisms of action. It has been designed to:

The data demonstrate that high concentration and continuous production of 5-FU chemotherapy can be obtained within the tumors through the local conversion of the pro-drug 5-FC (administered orally). This mechanism of action is based on the in-tumor expression of the proprietary FCU1 gene that has been integrated within the genome of TG6002.

In this study, extensive analyses are being performed including metastasis biopsy with synchronous blood sampling, assessment of virus presence, quantification of 5-FC and 5-FU and assessment of neutralizing antibody titers.

These analyses have allowed Transgene to document TG6002s pharmacokinetics (PK) and biodistribution, and the functioning of the FCU1 gene when given by IV administration.

Detailed results:

TG6002 infects tumors after intravenous administration, remains active and effectively express FCU1 gene selectively in tumor tissue; Absence of widespread virus distribution in the body and association of FCU1 activity with high virus concentration in tumor tissue suggest that the replication of TG6002 is concentrated in tumor cells; None of the patients presented clinical signs of extra-tumoral dissemination of the virus suggesting a high tumor specificity of the viral replication; The study is continuing with escalating dosing of TG6002.

CLINICAL PROOF OF CONCEPT OF THE FEASIBILITY OF THE IV ADMINISTRATION OF TRANSGENES PROPRIETARY ONCOLYTIC VIRUS

To-date, the only oncolytic virus that has received regulatory approval is only approved for intra-tumoral administration, restricting its use to superficial lesions.

Transgene aims to enlarge the number of solid tumors, such as gastro-intestinal tumors, that could be addressed by an oncolytic virus, by developing oncolytics that can be administered intravenously.

The findings that will be presented at AACR demonstrate the relevance of intravenous administration of Transgenes next generation oncolytic viruses including TG6002.

These data also suggest that candidates derived from Transgenes unique Invir.IO platform could also be given intravenously, extending the use of these therapies to a broad range of solid tumors.

The e-poster presentation will be available on the AACR website beginning at 8:30 am US EDT on Saturday, April 10, until Monday, June 21. The text of this abstract will be posted at 12:01 am US EDT on Friday, April 9 on the AACR website.

About the trial (NCT03724071)This trial is a single-arm open-label Phase I/II trial evaluating the safety and tolerability of multiple ascending doses of TG6002 administered intravenously in combination with oral 5-FC, a non-cytotoxic pro-drug that can be converted in 5-FU, its active metabolite. Based on the safety profile of TG6002, several dose levels have been added to the initial Phase I clinical protocol. At the end of this Phase I part, Phase II patients will receive the recommended dose of TG6002. The trial has safety as primary endpoint for the Phase I part and efficacy for the Phase II part. The trial also evaluates pharmacokinetic properties and biodistribution of TG6002, along with immune modulation of the tumor micro-environment. This European study will enroll up to 40 patients suffering from advanced gastrointestinal carcinomas who have failed and/or are intolerant to standard therapeutic options in the Phase I part. Patients with colon cancer and liver metastases will be enrolled in the Phase II part.

Dr. Philippe Cassier, M.D., PhD, head of the early-phase trials unit at Centre Lon Brard (Lyon, France) is the principal investigator of the trial.

About TG6002TG6002 has been engineered to directly kill cancer cells (oncolysis), to enable the production of a chemotherapy agent (5-FU) within the tumor, and to elicit an immune response by the body against the tumor cells. In preclinical experiments, TG6002 has been shown to induce the shrinkage of the primary tumor as well as the regression of distant metastases (Foloppe, et al., Molecular Therapy Oncolytics, https://doi.org/10.1016/j.omto.2019.03.005).

The production of 5-FU directly in the tumor aims to achieve a better anti-tumoral effect with limited chemotherapy-induced side effects.

TG6002 induces the production of 5-FU in the cancer cells it has infected, by enabling the local conversion of the pro-drug 5-FC (administered orally) into 5-FU. 5-FU is a common chemotherapy agent for patients with gastro-intestinal cancers. This mechanism of action is based on the in-tumor expression of the proprietary FCU1 gene that has been encoded in the genome of TG6002, taking advantage of the virus selective replication in the tumor cells.

When administered systemically, 5-FU is associated with side effects that can lead to treatment discontinuation. With TG6002, 5-FU is produced within the tumor where it is expected to be present at a high concentration level in contrast to the very low levels anticipated in the rest of the patients body.

About TransgeneTransgene (Euronext: TNG) is a biotechnology company focused on designing and developing targeted immunotherapies for the treatment of cancer. Transgenes programs utilize viral vector technology with the goal of indirectly or directly killing cancer cells.The Companys clinical-stage programs consist of two therapeutic vaccines (TG4001 for the treatment of HPV-positive cancers, and TG4050, the first individualized therapeutic vaccine based on the myvac platform) as well as two oncolytic viruses (TG6002 for the treatment of solid tumors, and BT-001, the first oncolytic virus based on the Invir.IO platform).With Transgenes myvac platform, therapeutic vaccination enters the field of precision medicine with a novel immunotherapy that is fully tailored to each individual. The myvac approach allows the generation of a virus-based immunotherapy that encodes patient-specific mutations identified and selected by Artificial Intelligence capabilities provided by its partner NEC.With its proprietary platform Invir.IO, Transgene is building on its viral vector engineering expertise to design a new generation of multifunctional oncolytic viruses. Transgene has an ongoing Invir.IO collaboration with AstraZeneca.Additional information about Transgene is available at: http://www.transgene.fr Follow us on Twitter: @TransgeneSA

DisclaimerThis press release contains forward-looking statements, which are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those anticipated. The occurrence of any of these risks could have a significant negative outcome for the Companys activities, perspectives, financial situation, results, regulatory authorities agreement with development phases, and development. The Companys ability to commercialize its products depends on but is not limited to the following factors: positive pre-clinical data may not be predictive of human clinical results, the success of clinical studies, the ability to obtain financing and/or partnerships for product manufacturing, development and commercialization, and marketing approval by government regulatory authorities. For a discussion of risks and uncertainties which could cause the Companys actual results, financial condition, performance or achievements to differ from those contained in the forward-looking statements, please refer to the Risk Factors (Facteurs de Risque) section of the Universal Registration Document, available on the AMF website (http://www.amf-france.org) or on Transgenes website (www.transgene.fr). Forward-looking statements speak only as of the date on which they are made, and Transgene undertakes no obligation to update these forward-looking statements, even if new information becomes available in the future.

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Transgene Presents Initial Phase I Data of TG6002, Highlighting the Potential of the Intravenous Administration of Its Oncolytic Viruses - Business...

For many people, the word biostatistics doesnt evoke much. But according to three biostatisticians at Tufts, its biologys special sauce, operating behind the scenes to make breakthroughs possible.

These include Kathryn Barger, associate director in the Biostatistics and Data Management Unit at the Jean Mayer USDA Human Nutrition Research Center on Aging (HNRCA) at Tufts; Paola Sebastiani, who works at Tufts Medical Center and is the director of the Biostatistics, Epidemiology, Research, and Design Center at Tufts (BERD); and Misha Eliasziw, a researcher and associate professor at Tufts University School of Medicine in the Department of Public Health and Community Medicine, and an associate professor at the Friedman School of Nutrition Science and Policy.

Defined as the application of statistics to biological topics, this field has flourished at Tufts. The universitys biostatisticians have contributed to a number of methodological advances that have had a significant impact on the field, according to Sebastiani, who recently established the Center for Quantitative Methods and Data Science, which is supported by Tufts Medical Center and the Data Intensive Study Center.

For proof of the impact this work can have, one need look no further than the COVID-19 pandemic, says Eliasziw. It was the biostatisticians who analyzed the data from the vaccine clinical trials and came up with the findings for physicians to interpret, she says. One of the reasons people are being vaccinated is because of biostatistics. It can actually make a difference in peoples lives.

Biostatisticians are different from statisticians, working primarily with medical and health-related investigators to design and contextualize ethically and scientifically sound studies. The translators and interpreters of the scientific world, they analyze data and translate complex science into actionable evidence.

Biostatistics is the method that transforms research data into scientific evidenceand is incredibly rewarding, says Barger.

Barger works at the intersection of medicine, nutrition, and aginga crucial field as more than 71 million Baby Boomers live longer, and hopefully better.

One of the perks of the job is working across a wide range of fields. Biostatistics is great for anyone who wants to collaborate with researchers in different scientific fields because statistical skills can be applied everywhere, Barger says. Even within the HNRCA, my role is unique in that across different research areas, I have been able to work with scientists in all of them.

Right now, Barger and her colleagues are studying how vitamin B12 status could influence risk of cognitive decline. Cognitive health and age-related disease such as Alzheimers are major problems that urgently need solutions, and its exciting to be part of the research in finding answers to prevent or ameliorate these health issues, she says.

Barger also finds it rewarding that at the end of the day, her work helps provide scientific evidence for establishing recommendations for healthy eating and other interventions. There are so many benefits to nutritional interventions, Barger says. To contribute to that evidence, to be able to ultimately inform people on things they can do to improve their disease risk later in life, is incredibly important.

Sebastiani also focuses on human aging and longevity at Tufts Medical Center. Her research focuses on one of sciences fundamental mysteries: why some people age healthfully and well, and some do not. Im thinking about intervention: to help people live longer and in a healthy way; to maintain good cognitive function; good physical function; and how we relate that to the genetic background of individuals, Sebastiani says.

The secret? People dont like it, but its nutrition, she says, laughing. The only intervention that has been shown to work in humans is to really restrict the number of calories that you consume every day, drastically.

Sebastiani works primarily with medical clinical investigators to support and collaborate on research projects, deciding how to analyze data, establishing best practices, and justifying each experiments design. For example, a scientist might test a drug on mice. A biostatistician will help to decide when to test, which dosages, and how many mice to safely enroll. Sebastiani does the same thing for humanshelping to select just the right number of participants and presenting the work at conferences and before funding agencies.

Sebastiani is now studying how a drug called Metformin might be used along with diet and exercise to change a persons profile of biomarkerssuch as specific proteinsand help extend life. Additionally, shes examining whether the Mediterranean diet so popular in southern Europebuilt mainly around plant-based foodscould potentially affect the APOE gene, whose variants are associated with a high risk for Alzheimers.

Sebastiani is also helping further the field of biostatistics itself via the Center for Quantitative Methods and Science, where she hopes to collaborate with computer scientists, epidemiologists, and statisticians to harness big data and machine learning in a multidisciplinary setting. The landscape of biomedical science is changing, and biostatisticians need to team up with other data scientists to solve big data problems, she says.

Tufts biostatisticians have already helped create models used around the world to predict health outcomes for patients with myocardial infarction and kidney diseases; machine learning methods for analyzing genetic and genomic data; and techniques for clinical trial design and evaluation of treatment effects, Sebastiani says. More recently, BERD biostatistician Norma Terrin contributed to the development and validation of a new tool to analyze negative reactions in newborns. This scale will now be adopted for use in clinical trials by the Food and Drug Administration and other regulators around the world, Sebastiani says.

If youre looking for certainty, biostatistics is the wrong place to find it, says Eliasziw, who teaches introductory and advanced courses in biostatistics at the School of Medicine.

Thats what differentiates biostatistics from mathematicsthe uncertainty, Eliasziw says. Sometimes frustrated new students want to know what the right solution is, and my answer is not satisfying: It depends on what the question is, and who you are studying.

What biostatistics does offer is a more fluid way to approach information. One of the things I find so enjoyable about biostatistics, is that its an art form. Theres creativity involvedusing graphical displays to tell a story, bringing unrelated ideas together, Eliasziw says. Human creativity and ingenuity is something that cannot be replaced by artificial intelligence.

Like Barger and Sebastiani, Eliasziw does research in nutrition, studying how to reduce childhood obesity primarily among children with intellectual disabilities and autism spectrum disorder. But she is also working on how to reduce other public health problems, including traffic-related air pollution; sexual assault among women on university campuses; and substance use among adolescents.

She uses similar methodologies and techniques across all her work. The basic skills can be applied in different areas, says Eliasziw, who worked on the prevention of stroke and cancers before she came to Tufts ten years ago. It makes life interesting, not doing the same old thing. And you can bring along ideas from one area to another.

For example, Eliasziw brought a technique called survival analysisdetermining the expected duration of time until a given eventfrom oncology to sexual assault prevention, and a method called propensity score analysis from cardiovascular literature to childhood obesity prevention. This cross-application of methods makes biostatisticians uniquely effective researchers who are in high demand across many fields, Eliasziw said. People call me Ghostbuster, she joked. If you have a problem, who are you gonna call?

Playing this role makes for a rewarding career, she says. The focus of biostatistics is very much on solving real life problems, she says. Its really about the application. Thats whats enjoyable.

Monica Jimenez can be reached atmonica.jimenez@tufts.edu.

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The Art and Science of Biostatistics | Tufts Now - Tufts Now

A team of researchers in Indiana has found that the best way to find new treatments for autoimmune diseases, including type 1 diabetes (T1D), is to study the immune system and targeted tissues together, based on an article published earlier this year.

Looking at the immune system in isolation is akin to attempting to fly a plane with only one wing, said Decio L. Eizirik, MD, PhD, the scientific director of the Indiana Biosciences Research Institute Diabetes Center, who is the senior author of the paper that appeared in Science Advances.1

The research, funded in part by the JDRF, springs from the fact that autoimmune diseases are increasing worldwide, and the prevalence of T1D, systemic lupus erythematosus (SLE), multiple sclerosis, and rheumatoid arthritis (RA) has reached 0.5% to 5% depending on the region. JDRFs clinical research strategy has focused in recent years not only on developing treatments and ultimately cures for T1D, but also on preventing it by screening those at greatest risk of developing the disease, and pursuing interventions to halt its onset.

As the authors wrote, While the immune targets of T1D, SLE, MS, and RA are distinct, they share several similar elements, including common variants that pattern disease risk, local inflammation with contribution by innate immunity, and downstream mechanisms mediating target tissue damage.

They focused on the increasing evidence that target tissues are not innocent bystanders of the autoimmune attack, but participate in a deleterious dialog with the immune system that contributes to their own demise. In their work, the researchers mined RNA sequencing datasets from relevant organ and tissue cells in the different diseases, and identified similar and dissimilar gene signatures. In doing so, they identified both candidate genes for the 4 major diseases as well as major common gene expression changes in tissues among them.

One common gene is TYK2, a protein that regulates interferon signaling. The team showed in its research that use of TYK2 inhibitors - already in use for other autoimmune diseases - protect -cells against immune-mediated damage.

"This research is significant in reaching the JDRF's mission to cure, treat and prevent T1D," Frank Martin, PhD, JDRF director of research, said in a statement. "Discovering the common pathways of tissue destruction across multiple autoimmune diseases will dramatically accelerate our path to a cure for T1D. Drugs that are effective in one autoimmune disease could be equally beneficial for another and quickly repurposed to make a big impact for people living with that disease. Characterizing the similarities and differences between multiple autoimmune diseases has the potential to transform the way we treat and cure these diseases in the future.

JDRF has undertaken a large-scale screening project, called T1Detect, that offers participants a blood test to find antibodies, which tell whether a person is at an early stage of T1D and likely to become insulin dependent. The project comes not only as rates of T1D are rising overall, but as they are rising faster among Black and Hispanic youth. The difference now is that there may soon be a treatment at hand.

Another JDRF-funded study, reported last month in Science Translational Medicine, showed that the monoclonal antibody teplizumab delayed the onset of T1D.2 FDA has scheduled an advisory panel on teplizumab for May 27 and a targeted action date for the drug is set for July 2, 2021.

References

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Understanding the Connected Nature of Type 1 Diabetes, Other Autoimmune Diseases - AJMC.com Managed Markets Network

SEATTLE--(BUSINESS WIRE)-- NanoString Technologies, Inc. (NASDAQ: NSTG), a leading provider of life science tools for discovery and translational research, today announced the highlights of spatial biology abstracts that will be presented at the 2021 meeting of the American Association of Cancer Research (AACR), which will be held virtually from April 10 - 15, 2021.

The GeoMx Digital Spatial Profiler (DSP) enables researchers to characterize tissue morphology to rapidly and quantitatively profile RNA and proteins. To date, NanoString and its collaborators have presented DSP data in dozens of abstracts at major scientific meetings and more than 45 peer-reviewed publications, demonstrating DSPs utility to address a wide range of biological questions in formalin-fixed paraffin-embedded (FFPE) and frozen tissues. At AACR 2021, eight abstracts that used GeoMx DSP will be presented during the poster session on Saturday, April 10.

Four of the eight abstracts will be presented by investigators from the GeoMx Breast Cancer Consortium (GBCC), an international network of breast cancer researchers. Their goal is to apply innovative approaches and decipher the spatial context of breast cancer to develop a comprehensive atlas and database of novel biomarkers for the disease.

GBCC Abstracts

Poster 2718: Digital spatial profiling in HER2 positive breast cancer: The road to precision medicine

In this work, the GeoMx DSP was used to profile 71 protein targets and gene expression profiling was done using NanoString's nCounter PanCancer IO360 assay for primary and metastatic tissues from human epidermal growth factor 2 positive (HER2+) breast cancer (BC) patients. A detailed characterization of carefully chosen immune cold, warm and hot regions of interest (ROI) in the tumor and tumor immune microenvironment of (HER2+) of these samples established that primary tumors had a higher number of immune cells than the metastatic sites. These findings, therefore, suggest that immunotherapy in early-stage BC could be more effective than in advanced BC.

Poster 2701: Molecular profiling to assess the immune response to neoadjuvant SABR in early breast cancer

NanoString's Human PanCancer immune profiling panel was used to assess the impact of localized radiotherapy to elicit an immune response in primary breast carcinomas before lumpectomy. They analyzed 25 patient samples for low-risk primary breast carcinomas from the SIGNAL 2.0 clinical trial using the GeoMx DSP platform, pre, and post stereotactic body radiation therapy (SBRT). Significant differences were found in the gene expression patterns in the immune microenvironment gene expression patterns and cellular composition after radiotherapy, demonstrating that SBRT treatment indeed evokes an immune response, increasing the innate immune response.

Poster 2698: Spatial gene expression profiling in breast cancer

Transcriptome profiling was performed for a cohort of breast cancer lumpectomies using the Cancer Transcriptomic Atlas (CTA) assay on the GeoMx DSP platform. Analysis of 60 patient samples revealed region-specific heterogeneity in unifocal and multifocal cancer tumors. This study demonstrates and establishes the importance of interactions between immune and tumor cells in the tumor microenvironment and the need to develop a strategy to stratify patients to available targeted therapies.

Poster 2726: Characterization of immune microenvironment and heterogeneity in breast cancer subtypes

In this work, the immune microenvironment of Luminal A, Luminal B, Basal, and HER2 tumor subtypes in a cohort of early breast cancer patients was studied using protein biomarkers. The markers were delineated in a spatial context using the GeoMx DSP. Characterization of the immune microenvironment subtypes provided evidence for potential clinical use for GeoMx DSP in diagnosing and better stratifying breast cancer patients based on spatial heterogeneity in tumor and tumor microenvironment.

Other spatial abstracts

Poster 339: Resistance to trastuzumab is associated with alpha-smooth muscle actin expression in the stroma of patients with HER2+ breast cancer

GeoMx DSP was used to identify biomarkers for resistance to trastuzumab in HER2+ breast cancer. Fifty-eight protein targets were analyzed in three different regions of interest (tumor [PanCK+], leukocyte [CD45+/CD68-], and macrophage [CD68+]) in a cohort of 151 breast cancer patients that received trastuzumab. The study uncovered a-SMA as a potential biomarker to augment the predictive value of the current standard of care HER2 assay and justifies its further validation in the light of the many new HER2 targeted therapies.

Poster 705: SARS-CoV-2 infection of the human heart governs intracardiac innate immune response

Spatial profiling of human post-mortem cardiac samples of SARS-CoV-2 infected myocardium was carried out using NanoString's Whole Transcriptome Analysis (1,864 genes) panel, along with a matching proteome panel on the GeoMx digital spatial profiler. The purpose of their investigation was to elucidate the molecular mechanisms underlying cardiac toxicity, a severe cause of morbidity and mortality in patients on DOX therapy. The study showed interesting gender-specific differential gene expression patterns in the myocardium between SARS-CoV-2 infected and control regions of interest. Signatures of enhanced innate and acquired immune signaling, apoptosis and autophagy, chromatin remodeling, reduced DNA repair, and reduced oxidoreductase activity were all observed in regions of infection. Additionally, DOX-induced increase in the expression of TMPSS2 and cathepsins A, B, and F, clearly indicated enhanced SARS-CoV-2 susceptibility in the myocardium, thus placing cancer patients on DOX therapy at increased risk of cardiac damage.

Poster 2731: Cell-type deconvolution of African American breast tumors reveals spatial heterogeneity of the immune microenvironment

Researchers at the University of Chicago carried out spatial gene expression analysis within localized segments of TNBC tumors from a cohort of self-reported African American patients in the Chicago Multi-Ethnic Breast Cancer Study (ChiMEC). Regions of interest for spatial characterization of tumor and tumor microenvironment using the GeoMx DSP Cancer Transcriptome Atlas assay were manually selected based on the specific morphologies. The 1,825 genes interrogated in the CTA assay provided a granular understanding of the immune landscape's heterogeneity within tumors.

Poster 2771: Comprehensive analysis of immuno oncology markers in the tumor microenvironment of solid tumor samples using GeoMxTM digital spatial profiler (DSP) and MultiOmyxTM hyperplexed immunofluorescence (IF)

This study describes a multi-faceted highly multiplexed tissue analysis of critical Immuno oncology (IO) protein markers in a pan-cancer cohort of up to 35 FFPE samples originating from breast, head, and neck, prostate, non-small cell lung cancer (NSCLC), endometrial and colorectal indications using NanoString human IO panel on GeoMx DSP in combination with a complementary MultiOmyx Hyperplexed Immunofluorescence (IF) assay. The spatial and quantitative data outputs from DSP nCounter system and cell classification information from the MultiOmyx assay provided the researchers an ability not only to characterize the immunophenotypes but also to visualize the spatial distribution of tumor-infiltrating immune cells at a single-cell resolution within the TME.

Spotlight Theaters at AACR

NanoString will be hosting two spotlight theaters during AACR 2021. The first spotlight theater presentation is April 11 from 1:00-2:00 pm EDT, featuring Joseph Beechem, Ph.D., senior vice president of R&D and chief scientific officer for NanoString, with an overview of the latest developments in spatial biology, True spatial genomics: Measuring the transcriptome in regions, cell and sub-cellular compartments. Dr. Beechem will explain spatial technologies' evolution and their applications from multi-cell to single-cell and subcellular resolution, using the GeoMx DSP and the companys Spatial Molecular Imager.

The second NanoString spotlight theater is Tuesday, April 13, from 11:00-12:00 pm EDT, and is entitled: New Approaches for Cellular Therapies: Technology Symposium Featuring the GeoMx DSP and nCounter CAR-T Characterization. This panel will include three speakers, Dr. Ryan Golden, Resident Physician in Clinical Pathology, Carl June Lab, University of Pennsylvania; Dr. Marco Ruella, Assistant Professor of Medicine, University of Pennsylvania; and Ghamdan Al-Eryani, Ph.D. Student, Tumor Progression Group from the Garvan Institute. Each speaker will discuss new approaches to CAR-T characterization using the spatially-resolved and bulk RNA analysis, from understanding resistance in CART immunotherapy in lymphoma to TCR diversity in melanoma.

NanoString has launched a Technology Access Program (TAP) for the recently announced single and subcellular Spatial Molecular Imager to complement the existing TAP program for GeoMx. Under the program, customers can submit tissue samples to NanoString for analysis using the spatial profiling platforms and receive a complete data package. Researchers interested in participating in NanoString's Technology Access Program should contact the company at TAP@nanostring.com.

About NanoString Technologies, Inc.

NanoString Technologies is a leading provider of life science tools for discovery and translational research. The companys nCounter Analysis System is used in life sciences research and has been cited in more than 4,000 peer-reviewed publications. The nCounter Analysis System offers a cost-effective way to easily profile the expression of hundreds of genes, proteins, miRNAs, or copy number variations, simultaneously with high sensitivity and precision, facilitating a wide variety of basic research and translational medicine applications, including biomarker discovery and validation. The companys GeoMx Digital Spatial Profiler enables highly-multiplexed spatial profiling of RNA and protein targets in a variety of sample types, including FFPE tissue sections.

For more information, please visit http://www.nanostring.com.

NanoString, NanoString Technologies, the NanoString logo, GeoMx, and nCounter are trademarks or registered trademarks of NanoString Technologies, Inc. in various jurisdictions.

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NanoString Highlights Spatial Biology Research from the 2021 American Association of Cancer Research (AACR) Conference - BioSpace

White Paper: Enabling the Future of Cell and Gene Therapies through Non-Proprietary Patient-Owned Data Collection

ALISO VIEJO, Calif. (PRWEB) March 30, 2021

RARE-X, a collaborative platform for patient-controlled data collection and sharing, has published a new white paper exploring the primary issues and requirements for data gathering faced by cell and gene therapy developers. Enabling the Future of Cell and Gene Therapies through Non-Proprietary Patient-Owned Data Collection considers solutions and the role a standardized non-proprietary data collection platform, such as RARE-X, can play in addressing research challenges. RARE-X published the white paper in partnership with The ARM Foundation for Cell and Gene Medicine.

A significant finding of the white paper is that patient organizations are best situated to gather the data needed by stakeholders and ensure that it is appropriately shared with others, Morrie Ruffin, co-founder and board member, ARM Foundation for Cell and Gene Medicine.

The paper draws from the insights of top cell and gene therapy researchers and data scientists from biotech, government, and universities. An accompanying RARE-Xtra audio interview is also available, including discussions with a panel of researchers, biotech executives, and policy influencers.

Visit the RARE-X website to download a complimentary copy of Enabling the Future of Cell and Gene Therapies through Non-Proprietary Patient-Owned Data Collection or listen to the RARE-Xtra recorded panel at RARE-X.org.

ABOUT RARE-XRARE-X is a 501(c)(3) patient advocacy organization focused on supporting the acceleration and development of life-altering treatments and future cures for patients impacted by rare disease. Enabled by best-in-class technology, patients, researchers, and other technology vendors, RARE-X will gather structured, fit-for-purpose data to share broadly, benefitting from 21st-century governance, consent, and federated data sharing technology. RARE-X is building the largest collaborative patient-driven, open-data access project for rare diseases globally. For more information, visit http://www.rare-x.org.

About the ARM Foundation for Cell and Gene MedicineThe ARM Foundation for Cell and Gene Medicine serves as the education and information catalyst on issues fundamental to making gene and cell therapies, tissue-engineered products and other regenerative medicine treatments available to patients. It is an independent, 501(c)(3) non-profit organization established in 2018 by the Alliance for Regenerative Medicine (ARM), the preeminent international advocacy organization for regenerative medicine and advanced therapies. For more information, please visit http://www.thearmfoundation.org.

Media Contact:Tom Hume, Marketing Communications RARE-Xtomh@rare-x.org

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RARE-X Explores Patient-Owned Data Gathering for Cell and Gene Therapy Developers - PR Web

LEXINGTON, Mass., March 30, 2021 /PRNewswire/ --LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood, today announced that chief executive officer Frederic Chereau will be participating on a panel at the 2021 Redburn Gene Therapy Summit. During the panel, entitled "Gene therapy: a changing regulatory landscape," Mr. Chereau will offer a brief overview of LogicBio and discuss the design of the SUNRISE clinical trial of LB-001 for the treatment of methylmalonic acidemia (MMA). The panel will take place at 9:15 a.m. EDT on Wednesday March 31st.

About LogicBio Therapeutics, Inc.

LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood. The company's proprietary GeneRide platform is a new approach to precise gene insertion that harnesses a cell's natural DNA repair process leading to durable therapeutic protein expression levels. LogicBio's cutting-edge sAAVy capsid development platform is designed to support development of treatments in a broad range of indications and tissues. The company is based in Lexington, MA. For more information, visit https://www.logicbio.com/.

Media Contacts:

Adam DaleyBerry & Company Public RelationsW: 212-253-8881C: 614-580-2048[emailprotected]

Jenna UrbanBerry & Company Public RelationsW: 212-253-8881C: 203-218-9180[emailprotected]

Investor Contacts:

Matt Lane Gilmartin Group 617-901-7698[emailprotected]

SOURCE LogicBio Therapeutics, Inc.

https://www.logicbio.com

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LogicBio Therapeutics to Present at 2021 Redburn Gene Therapy Summit - PRNewswire

- In 2020, continued to advance genetic medicine pipeline with execution of IND-enabling studies for AK-OTOF and general alignment with FDA on the path to a 2022 IND submission for AK-antiVEGF

- Raised approximately $349 million in gross proceeds, which is expected to fund operations for at least two years

- Due to recent third-party manufacturing delays, IND submission for AK-OTOF program now expected in the first half of 2022; all other IND-enabling activities remain on track

- Establishing internal cGMP manufacturing infrastructure and capabilities in 2021

BOSTON, March 29, 2021 (GLOBE NEWSWIRE) -- Akouos,Inc. (Nasdaq: AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today reported financial results for the fourth quarter and full year ended December 31, 2020 and provided business highlights.

2020 was a year of tremendous progress for Akouos, marked by continued advancement of our pipeline, expansion of our team, and strengthening of our capital position to further our leadership in the development of precision genetic medicines for inner ear conditions, said Manny Simons, Ph.D., founder, president, and CEO of Akouos. We continue to be excited by the nonclinical data reported to date, which demonstrate the durable recovery of function of AK-OTOF. Due to third-party manufacturing delays, including impacts from the ongoing COVID-19 pandemic, we now expect to submit the IND for AK-OTOF in the first half of 2022. All other IND-enabling activities remain on track. We continue to work with multiple third-party manufacturers to advance cGMP campaigns, for both the AK-OTOF and AK-antiVEGF IND submissions planned for 2022, and we continue to build our internal cGMP manufacturing infrastructure and capabilities.

Business and Pipeline Highlights for 2020, Recent Developments, and Anticipated Milestones

Fourth Quarter and Full Year 2020 Financial Results

About Akouos

Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

Cautionary Note Regarding Forward-Looking Statements

Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, plans, and timing of our future clinical trials and our research and development programs, the timing of our IND submissions for AK-OTOF and AK-antiVEGF, our expectations regarding our manufacturing capabilitiesand timelines, and the period over which we believe that our existing cash,cash equivalents and marketable securities will be sufficient to fund our operating expenses. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, will, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: our limited operating history; uncertainties inherent in the development of product candidates, including the initiation and completion of nonclinical studies and clinical trials; whether results from nonclinical studies will be predictive of results or success of clinical trials; the timing of and our ability to submit applications for, and obtain and maintain regulatory approvals for, our product candidates; our expectations regarding our regulatory strategy; our ability to fund our operating expenses and capital expenditure requirements with our cash, cash equivalents, and marketable securities; the potential advantages of our product candidates; the rate and degree of market acceptance and clinical utility of our product candidates; our estimates regarding the potential addressable patient population for our product candidates; our commercialization, marketing, and manufacturing capabilities and strategy; our ability to obtain and maintain intellectual property protection for our product candidates; our ability to identify additional products, product candidates, or technologies with significant commercial potential that are consistent with our commercial objectives; the impact of government laws and regulations; risks related to competitive programs; the potential that our internal manufacturing capabilities and/or external manufacturing supply may experience delays; the impact of the COVID-19 pandemic on our business, results of operations, and financial condition; our ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in the Risk Factors included in the Companys Quarterly Report on Form 10-Q for the three months ended September 30, 2020 filed with the Securities and Exchange Commission, and in other filings that the Company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Condensed Consolidated Balance Sheet Data(Unaudited)

(in thousands)

Condensed Consolidated Statements of Operations and Comprehensive Loss(Unaudited)

(in thousands, except share and per share data)

Contacts

Media:

Katie Engleman, 1ABkatie@1abmedia.com

Investors:

Courtney Turiano, Stern Investor RelationsCourtney.Turiano@sternir.com

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Akouos Reports Fourth Quarter and Full Year 2020 Financial Results and Provides Business Highlights - BioSpace

MADISON, Wis., March 29, 2021 /PRNewswire/ --A pair of top C-level Executives from blockbuster gene therapy company AveXis have joined ENDSULIN, a new venture that could reach millions more than any gene therapy to date. Spearheaded by one of the disease's most accomplished pioneers, the company aims to upend a multi-billion dollar treatment industry.

Both Brian Kaspar and Thomas Dee, the founding Chief Science and Chief Finance Officers, respectively, of AveXis (developer of Zolgensma, bought by Novartis for $8.7 Billionin May of 2018) have accepted board roles at ENDSULIN. While AveXis sought to "transform rare diseases," ENDSULIN is solely targeting one of the most prolific Type 1 diabetes (T1D) with what they hope will be a one-time treatment to free patients from daily insulin injections by more precisely regulating their glycemic control.

"The science itself is remarkable, but the potential to change millions of lives is unprecedented," Kaspar said. "We've proven gene therapy can revolutionize medicine with lasting, durable treatments. But now we need players like ENDSULIN with the right vision and focus who can harness that momentum to give patients the relief they deserve."

The former AveXis leaders round out a team of pioneering experts at the forefront of diabetes, gene therapy and drug development, strategically brought together to accelerate ENDSULIN's undivided focus: FDA authorization to begin human clinical trials.

Founder Hans Sollinger's previous discoveries have shaped modern pancreatic transplantation treatment for T1D. But because of the complexity of the disease, while the science of management has advanced, insulin shots have been the enduring standard of care since the 1920s almost a full century.

"25 years ago, I had performed more kidney-pancreas transplants than anyone in the world, but I'd only helped 500 people," Sollinger said. "The most important aspect of my search for a cure became its reach, in terms of both scale and accessibility. As I advanced, it became more and more obvious that gene therapy was the way to achieve it all, with no compromises."

Through decades of research at the UW Hospitals and Clinics, Sollinger's team developed a highly unique and targeted expression system that addresses one of the greatest challenges in treating T1D: precise regulation of insulin in response to rapidly fluctuating glucose levels. Several publications over the last 10 years have demonstratedlong-lasting euglycemia control in hundreds of mice and rats, indicating a durable cure may be within reach.

"We've focused everything we are doing around our mission to get this treatment to patients," said Eric Spyra, Interim CEO and Board Member. "Using existing commercial infrastructure and following the path of other liver-directed AAV gene therapies, we're putting all the pieces in place now that will accelerate this therapy through every stage."

ENDSULIN has already initiated a pilot study in autoimmune-modeled naturally diabetic companion dogs, while executing their IND-enabling plan and preclinical studies. The company is preparing for a Series A funding round this year.

ABOUT ENDSULINENDSULIN is reshaping the way we approach a cure for diabetes. They are working to free patients from daily injections and 24/7 management using the most cutting-edge gene therapy technology, developed from decades of research by noted diabetes leader Hans Sollinger, MD, PhD, Dr hc, at the University of Wisconsin Hospitals and Clinics. Their sole focus is to get a durable, one-time treatment to the millions of people who need it.

SOURCE ENDSULIN

http://www.endsulin.com

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Top Executives from AveXis Join New Gene Therapy Venture -- This Time Targeting Tens of Millions More Patients - PRNewswire

Gustavo Aguirre and William Beltran, veterinary ophthalmologists and vision scientists at the University of Pennsylvania School of Veterinary Medicine, have studied a wide range of different retinal blinding disorders. But the one caused by mutations in the NPHP5 gene, leading to a form of Leber congenital amaurosis (LCA), is one of the most severe.

Children with this disorder are not visual, says Aguirre. They have a wandering, searching look on their faces and are usually diagnosed at a young age.

A nearly identical disease naturally occurs in dogs. In a new paper in the journal Molecular Therapy, Aguirre, Beltran, and colleagues at Penn and other institutions have demonstrated that a canine gene therapy can restore both normal structure and function to the retinas cone photoreceptor cells, which, in LCA patients, otherwise fail to develop normally. Delivering a normal copy of either the canine or human version of the NPHP5 gene restored vision in treated dogs.

Whats amazing is that you can take this disease in which cone cells have incompletely formed, and the therapy restores their functionthey had no function whatsoever beforeand recover their structure, says Aguirre.

That plasticity is incredible and gives us a lot of hope, Beltran says.

LCA includes a wide range of inherited vision disorders characterized by blindness that strike in early childhood. The form of LCA associated with NPHP5 mutations is rare, affecting about 5,000 people worldwide. Known as a ciliopathy, it affects the cilia of cells of the retina. The cilia cells are antennalike structures on photoreceptor cells that translate the energy from light into visual signals.

In the NPHP5 disease, rod photoreceptor cellsthose responsible for vision in low lightdegenerate and progressively die early in the disease. Yet the cone photoreceptors, which enable color vision and, in the central retina, the perception of fine detail, while abnormal structurally, survive, albeit without function.

Aguirre and Beltran, together with colleagues and coauthors on the current work, Artur Cideciyan and Samuel Jacobson in Penns Perelman School of Medicine, have found success with gene therapy approaches to treating a variety of inherited vision disorders. Often, they have aimed to treat early in the course of a retinal disease, before photoreceptor cells have died or entirely degenerated. But the fact that cone cells persisted in this form of LCA led the researchers to consider whether a therapy that targeted cones could not just stop but reverse the course of the disease.

Testing this approach, the team delivered retinal injections of adeno-associated viral vectors, a platform for ferrying the normal version of the NPHP5 gene, into one eye of each of nine five-week-old dogs with the vision disorder. Known as gene augmentation therapy, the injection is used to supply a healthy gene in disorders where the causative mutation leads to a defective or absent protein.

To determine the effectiveness of the treatment, the researchers used a technique called electroretinography, which measures the electrical response of photoreceptor cells to a light stimulus, as well as optical coherence tomography, which allows for the noninvasive imaging of fine cross sections of the retina. Both means of evaluating the experimental therapy rendered encouraging results. In the dogs treated eyes, the outer segment of the cones regrew.

In addition, when the treated dogs were about six months old, their vision was tested using an obstacle-avoidance course. When their treated eye was blindfolded, they had difficulty at navigating; however, when that eye was uncovered, their ability to avoid obstacles was notably improved.

Whats so appealing and so exciting here is that were not just stopping a disease process, were actually reverting a photoreceptor cell that is abnormal to become normal and function, says Beltran. This disease in dogs very closely parallels the disease in humans, in quite specific terms, so theres a lot of support for the thought that a similar treatment approach could also help children.

Ongoing studies suggest that the treatment may be effective even when delivered at later stages of disease. With further support, the researchers hope to move the research along the path to a clinical trial in people.

Gustavo D. Aguirre is professor of medical genetics and ophthalmology in the Department of Clinical Sciences and Advanced Medicine at the University of Pennsylvania School of Veterinary Medicine.

William Beltran is professor of ophthalmology in the Department of Clinical Sciences and Advanced Medicine and director of the Division of Experimental Retinal Therapies at Penns School of Veterinary Medicine.

Artur V. Cideciyan is a research professor of ophthalmology at the Scheie Eye Institute in the University of Pennsylvania's Perelman School of Medicine.

Samuel G. Jacobson is a professor of ophthalmology at the Scheie Eye Institute in Penns Perelman School of Medicine.

In addition to Aguirre, Beltran, Cideciyan, and Jacobson, coauthors on the study were Penn Vets Valrie L. Dufour, Ana Ripolles-Garca, Raghavi Sudharsan, Roman Nikonov, and Simone Iwabe; Penn Medicines Malgorzata Swider; and the University of Floridas Sanford L. Boye and William W. Hauswirth.

The study was supported in part by the National Eye Institute (grants EY006855, EY017549, and EY001583) with additional support from the Foundation Fighting Blindness, the Van Sloun Fund for Canine Genetic Research, Hope for Vision, the Research to Prevent Blindness Foundation, and the Sanford and Susan Greenberg End Blindness Outstanding Achievement Prize.

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Turning back the clock on a severe vision disorder | Penn Today - Penn Today