StemCell Therapy

NEW YORK, Dec. 02, 2020 (GLOBE NEWSWIRE) -- Predictive Oncology (NASDAQ: POAI), a knowledge-driven company focused on applying artificial intelligence (AI) to personalized medicine and drug discovery, today announced that its virtual Special Meeting of Stockholders, scheduled for December 1, 2020, was convened and adjourned without any business being conducted due to the fact that quorum was not achieved. The Special Meeting will be reconvened December 30, 2020 at 3 p.m. CST and will be conducted via live audio webcast at http://www.virtualshareholdermeeting.com/POAI2020SM, so as to allow more opportunity for stockholders to vote on all proposals described in the Companys definitive proxy statement filed with the Securities and Exchange Commission (SEC) on November 3, 2020.

The record date for the Special Meeting remains October 28, 2020. Stockholders of record may vote at the reconvened Special Meeting, vote by proxy using the proxy card enclosed with the Companys definitive proxy statement, vote by proxy via facsimile, email or on the Internet. Whether or not stockholders of record plan to attend the reconvened Special Meeting, the Companys Board of Directors and management urge them to vote by proxy to ensure their vote is counted. Stockholders who have previously submitted their proxy or otherwise voted and who do not want to change their vote need not take any action.

A copy of the Companys proxy statement as previously filed with the SEC is available at no charge on the SEC website at http://www.sec.gov. In addition, copies of the proxy statement and other documents may be obtained free of charge by accessing the Companys website at https://investors.predictive-oncology.com/ or by contacting the Companys Corporate Secretary at (651) 389-4806 or by mail to Corporate Secretary, Predictive Oncology Inc., 2915 Commers Drive, Suite 900, Eagan, MN 55121.

YOUR PARTICIPATION IS IMPORTANT - PLEASE VOTE TODAY!

About Predictive Oncology Inc.

Predictive Oncology (NASDAQ: POAI) operates through three segments (Skyline, Helomics and Soluble Biotech), which contain four subsidiaries: Helomics, TumorGenesis, Skyline Medical and Soluble Biotech.

Helomics applies artificial intelligence to its rich data gathered from patient tumors to both personalize cancer therapies for patients and drive the development of new targeted therapies in collaborations with pharmaceutical companies. TumorGenesis Inc. specializes in media that help cancer cells grow and retain their DNA/RNA and proteomic signatures, providing researchers with a tool to expand and study cancer cell types found in tumors of the blood and organ systems of all mammals, including humans. Skyline Medical markets its patented and FDA cleared STREAMWAY System, which automates the collection, measurement and disposal of waste fluid, including blood, irrigation fluid and others, within a medical facility, through both domestic and international divisions. Soluble Biotech is a provider of soluble and stable formulations for proteins including vaccines, antibodies, large and small proteins and protein complexes.

Forward-Looking Statements

Certain matters discussed in this release contain forward-looking statements. These forward-looking statements reflect our current expectations and projections about future events and are subject to substantial risks, uncertainties and assumptions about our operations and the investments we make. All statements, other than statements of historical facts, included in this press release regarding our strategy, future operations, future financial position, future revenue and financial performance, projected costs, prospects, plans and objectives of management are forward-looking statements. The words anticipate, believe, estimate, expect, intend, may, plan, would, target and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Our actual future performance may materially differ from that contemplated by the forward-looking statements as a result of a variety of factors including, among other things, factors discussed under the heading Risk Factors in our filings with the SEC. Except as expressly required by law, the Company disclaims any intent or obligation to update these forward-looking statements.

Investor Relations Contact:

Hayden IRJames Carbonara(646)-755-7412

Excerpt from:
Predictive Oncology Announces Adjournment of Special MeetingScheduled to Reconvene on December 30, 2020 - BioSpace

The Scientist's annual award recognizes exciting biomedical technologies that have the potential to generate the biggest impact on scientific research. Seven Bridges GRAF was selected by The Scientist's independent panel of expert judges for its potential to improve methods for analyzing next-generation sequencing data, helping researchers overcome the inherent flaws in the human genome linear reference sequence, and improving understandings of genetic diversity in understudied populations such as people of African, Asian, Hispanic and Middle Eastern descent.

"Seven Bridges is the first to offer this type of graph-based bioinformatics solution," said William Moss, CEO of Seven Bridges. "This recognition underscores the greater importance Seven Bridges GRAFTMbrings to the research community and its value in helping scientists develop new understandings of global population diversity. We hope that this research ultimately improves precision medicine across global populations."

The company's comprehensive graph-based bioinformatics solution is designed to greatly improve the analysis of non-European ethnicities; support large, more diverse population studies; enable research on underrepresented populations; and aid in the development of personalized references for more precise analysis. Seven Bridges GRAF enables:

"The COVID-19 pandemic birthed a shared scientific purpose and accelerated innovation in 2020," said The Scientist Editor-in-Chief Bob Grant. "The winners of this year's competition embody the spirit of togetherness and ingenuity marshaled by the research community in the face of this unprecedented challenge."

In addition to the honor from The Scientist, this year, Seven Bridges GRAF was selected as the top performer in the precisionFDA Truth Challenge for the major histocompatibility complex (MHC) region. The MHC region contains highly polymorphic genes that play a vital role in the immune system, making it difficult to accurately process NGS data in this region.

"The MHC region contains highly polymorphic genes that play a vital role in the immune system, making it difficult to accurately process NGS data in this region," said Dr. H. Serhat Tetikol, the GRAF Product Director at Seven Bridges. "From a computational perspective, the complexity of such genomic loci is only exacerbated when one takes into account the variation between different ancestries. This is where the current linear human genome reference clearly falls short, and where our GRAF technology can provide significant utility over traditional methods."

For more information about Seven Bridges GRAF, visit http://www.sevenbridges.com/graf/.

About Seven BridgesSeven Bridges enables researchers to extract meaningful insights from genomic and phenotypic data in order to advance precision medicine. The Seven Bridges Ecosystem consists of a compliant analytic platform, intelligently curated content, transformative algorithms, unprecedented access to federated data sets, and expert on-demand professional services. This holistic approach to bioinformatics is enabling researchers at the world's leading academic, biotechnology, clinical diagnostic, government, medical centers, and pharmaceutical entities to increase R&D efficiency, enhance the hypothesis resolution process, isolate critical biomarkers, and even turn a failing clinical trial around while also reducing computational workflow times and data storage costs. To learn more, visitsevenbridges.comor follow us onLinkedInandTwitter.

Contact: Eric Schubert +1 415 692 6799 [emailprotected]

SOURCE Seven Bridges

Home

Read the rest here:
Seven Bridges GRAF Named #3 on The Scientist's Top 10 Innovations List for 2020 - PRNewswire

Optimal patient stratification is critical in the era of personalized medicine. Germline polymorphisms play an important role in the treatment response of various human diseases, including bladder cancer. Intravesical BCG therapy is widely-used for bladder cancer. However, tumor recurrence and progression are very common. Stratification based on germline polymorphisms may contribute to circumvent this clinical challenge. Autophagy pathway plays an important role in the nonspecific protective effects of BCG. Patients that carry C allele of rs3759601 in autophagy gene ATG2B showed increased risk of recurrence and progression in European population. We thus sought to analyze rs3759601 and its relevance in BCG response in Asian NMIBC patients.

Functional impact of rs3759601 ATG2B (p.Gln1383Glu) was analyzed by bioinformatics programs including NCBI Conserved Domain Search, Clustal Omega, Polyphen and SIFT. NMIBC patients who received intravesical BCG at multiple hospitals in Singapore from 1995 to 2016 were included. These patients were genotyped for rs3759601 using high resolution melt analysis. The rs3759601 polymorphism was studied in correlation with the bladder cancer recurrence rate and disease progression rate in our cohort. Statistical analysis was conducted using Kaplan-Meier plots and the Chi-squared analysis.

In total, 307 individules were included in the study including 161 NMIBC patients and 146 healthy controls, predominately Chinese. The rs3759601 genotype distributions in our NMIBC patients were (GG 72.1%; GC 27.9%; CC 0%), which were distinct from the Dutch report (GG 32.8%; GC 47.4% and CC 19.8%, Buffen K et al, 2014). Consistently, the C allele frequencies of rs3759601 are 0.171 in our controls and 0.177 in East Asians from 1,000 Genome, but 0.406 in Europeans from 1,000 Genome. In silico analysis suggested rs3759601 ATG2B (p.Gln1383Glu) alteration is unlikely to be functionally deleterious. Statistical analysis revealed no significant association between ATG2B rs3759601 C allele and risk of bladder cancer recurrence (P= 0.353, GC vs. GG: hazard ratio [HR]= 1.324), or cancer progression (P= 0.454, GC vs. GG: HR=0.658).

In contrast to European NMIBC patients, ATG2B rs3759601 C allele is much less common in Asians and it not associated with BCG response in Asian NMIBC patients.

Urologic oncology. 2020 Nov 26 [Epub ahead of print]

Zhijiang Zang, Yew Koon Lim, Yiong Huak Chan, Lata Raman, Kesavan Esuvaranathan, Edmund Chiong, Ratha Mahendran

Department of Urology, National University Hospital, National University Health System, Singapore. Electronic address: ., Department of Surgery, Yong Loo Lin School of Medicine, National University of Singapore, Singapore., Biostatistics Unit, Yong Loo Lin School of Medicine, National University of Singapore, Singapore., Department of Urology, National University Hospital, National University Health System, Singapore; Department of Surgery, Yong Loo Lin School of Medicine, National University of Singapore, Singapore.

PubMed http://www.ncbi.nlm.nih.gov/pubmed/33250346

Read the original:
Polymorphism in autophagy gene ATG2B is not associated with bladder cancer recurrence after intravesical Ba... - UroToday

Hologics Jennifer Meade, division president of breast and skeletal care, discusses the focus for breast imaging in the near future and some latest technologies.

Hologic received 510(k) clearance from the U.S. Food & Drug Administration for its Genius AI Detection software this week during the Radiology Society of North America (RSNA) annual meeting. This move is one more development that is feeding growing trends in breast imaging, said Jennifer Meade, division president of breast and skeletal care for womens imaging vendor Hologic.

Meade spoke with Diagnostic Imaging not only about some of Hologics more recent product offerings, but she also discussed feedback from the radiology community and some growing trends in breast imaging. In the coming years, she said, the industry can expect to see a greater focus on improved patient outcomes, workflow efficiency, and patient experience. In addition, she said developments and investments will continue around artificial intelligence, as well as personalized medicine.

With this latest FDA clearance, the Genius AI Detection software is set to follow that path, according to company information. It is designed to optimize workflow in four specific ways by providing a case score that reflects confidence of malignancy, categorizing cases based on the number of lesions present, flagging the most concerning cases in less than a minute, and providing an estimate of how much time a radiologist can expect the image review to take.

For more RSNA coverage, click here.

Read more:
Trends in Breast Imaging Care: Improving Outcomes, Efficiency, and Experience - Diagnostic Imaging

DataIntelo, a prominent market research firm, has published a detailed report on Global Personalized Medicines Market. This market research report provides comprehensive and in-depth analysis on the market which can possibly help an enterprise to identify lucrative opportunities and assist them with fabricating creative business strategies. The market report provides information about the current market scenario regarding the global supply and demand, key market trends and opportunities in the market, and challenges and threats faced by the industry players.

The Personalized Medicines market report talks about the competitive scenario among the industry players and imparts aspiring and emerging industry players with the future market insights in a detailed manner. This market report includes crucial data and figures which are structured out in a concise yet understandable manner. The research report covers the updates on the government regulations and policies which illustrates key opportunities and challenges of the market. DataIntelo has been monitoring the market since few years and collaborated with eminent players of the industry to give better insights on the market. It has conducted vigorous research and implied robust methodology to provide accurate predictions about the market.

You can buy the complete report on @ https://dataintelo.com/checkout/?reportId=98645

Impacts of Advancements and COVID-19 on the market.

Amidst the COVID-19, few segments of the market have witnessed a disruption due to the gap in supply and demand which has impacted the growth of the Personalized Medicines market. Along with this, the latest advancements have changed the market dynamics of the market. This research report covers the wide-range analysis of the COVID-19 impact to the industry and gives out insights on the change in the market scenario due to the advancements.

Request A Free Sample Report @ https://dataintelo.com/request-sample/?reportId=98645

Market Segmentation

Some of the major companies that are covered in the report.

3G BiotechQuest DiagnosticsLaboratory Corporation of AmericaAbbottAgendia NVAsuragen IncBecton DickinsonCardioDx Inc.Foundation MedicineQiagenSiemens HealthcareAmgenBristol-Myers SquibbGE HealthcarePfizerSanofi23andMeIllumina

Note: Additional companies

Based on the type, the market is segmented into

PM DiagnosticsPM TherapeuticsPersonalized Medical CarePersonalized Nutrition & Wellness

Based on the application, the market is segregated into

Hospitals PharmaciesRetail PharmaciesDietary Care CentersOthers

Based on the geographical location, the market is segregated into

Asia Pacific: China, Japan, India, and Rest of Asia PacificEurope: Germany, the UK, France, and Rest of EuropeNorth America: The US, Mexico, and CanadaLatin America: Brazil and Rest of Latin AmericaMiddle East & Africa: GCC Countries and Rest of Middle East & Africa

DataIntelo provides yearly updates on the Personalized Medicines market that assist the clients to stay ahead in the competitive space.

Why one should buy this Personalized Medicines Report?

The market research report provides all valuable constituents of the market such as revenue growth, product pricing & analysis, growth potential, and guidelines to tackle the challenges in the market. The report covers all the crucial mergers & acquisitions, partnerships, and collaborations that created further created opportunities or in some cases, challenges for the industry players.

This report includes latest product news, advancements, and updates from the prominent player of the industry that has leveraged their position in the market. It also provides business strategies implemented by the key players and yardstick to arrive on informed business decisions. Moreover, it gives insights on the consumer behavior patterns that can help the enterprise to curate the business strategies accordingly.

DataIntelo bestows the clients with the specialized customized options related to the regional analysis, company analysis, and product analysis, among others.

Complete Table Content of the Market

Executive Summary

Assumptions and Acronyms Used

Research Methodology

Personalized Medicines Market Overview

Personalized Medicines Supply Chain Analysis

Personalized Medicines Pricing Analysis

Global Personalized Medicines Market Analysis and Forecast by Type

Global Personalized Medicines Market Analysis and Forecast by Application

Global Personalized Medicines Market Analysis and Forecast by Sales Channel

Global Personalized Medicines Market Analysis and Forecast by Region

North America Personalized Medicines Market Analysis and Forecast

Latin America Personalized Medicines Market Analysis and Forecast

Europe Personalized Medicines Market Analysis and Forecast

Asia Pacific Personalized Medicines Market Analysis and Forecast

Asia Pacific Personalized Medicines Market Size and Volume Forecast by Application

Middle East & Africa Personalized Medicines Market Analysis and Forecast

Competition Landscape

If you have any questions on this report, please reach out to us @ https://dataintelo.com/enquiry-before-buying/?reportId=98645

About the Company

DataIntelo is the largest aggregator of the market research report in the industry with more than 800 global clients. The company has extensively invested in the research analysts training and programs to keep the analyst tapped with the best industry standards and provide the clients with the&utmost experience. Our dedicated team has been collaborating with the industry experts to give out the precise data and figures related to the industry. It conducts primary research, secondary research, and consumer surveys to provide an in-depth analysis of the market. The market research firm has worked in several business verticals and has been successful to earn high credentials over the time.

Contact Info:

Name: Alex Mathews

Address: 500 East E Street, Ontario,

CA 91764, United States.

Phone No: USA: +1 909 545 6473

Email:[emailprotected]

Website:https://dataintelo.com

Continued here:
Personalized Medicines Market 2020 |Global Industry Analysis By Trends, Size, Share, Company Overview, Growth And Forecast By 2026| Latest Research...

CHICAGO, Nov. 26, 2020 /PRNewswire/ -- BASE10 Genetics and DNA Link today announced their collaboration on a research project to evaluate the usability of DNA Link's AccuFind COVID-19 IgG antibody test in a healthcare setting.

Study will expand access to antibody testing outside of laboratory settings

Although Covid-19 deaths and new confirmed cases continue to rise across nursing homes in the US, many of those infected do recover. This has led to growing interest in the relationship between antibodies and their protective value whether generated by previous Covid-19 exposure or by a vaccine, once available.

At present, most antibody tests on the market require samples to be sent to a lab for analysis. The extra cost and time associated has constrained population wide studies of SARS-CoV-2 antibodies.

"An antibody test that can be done at the point of care without the need for a lab can more quickly provide information clinicians and administrators need to make decisions for their nursing home patients and staff," says Dr. Michael Fang, BASE10 CEO.

"This research is an important milestone in our efforts to expand access to antibody testing," says Elizabeth Freund, VP of Innovation at BASE10. "DNA Link's AccuFind performs very well in laboratory settings. The next logical step is to test its performance when used by healthcare professionals outside the lab. Many factors have the potential to affect test performance in lesser controlled settings so it's an ambitious goal to be sure. However, if FDA believes the data demonstrates its safety and effectiveness under these conditions, we'll be one step closer to FDA emergency use authorization for point of care use."

The study is set to launch in December.

About BASE10 Genetics, Inc.BASE10 is a precision medicine company based in Chicago. BASE10's proprietary platform identifies precision diagnostics technologies with high potential for improving health outcomes, and creates turnkey, disease management programs that can be deployed at scale for insurance payors.

For more information about BASE10 Genetics and its solutions, contact media@base10genetics.com.

About DNA Link, Inc.DNA Link, Inc. is a leading genomic service corporation based in Korea with a focus on personalized medicine and new drug development. The company combines biotechnology with information & communications technology to support total analysis of genomic and clinical information to accelerate research and development in the field of bio-industry and genomic analysis industry.

For more information about DNA Link and its solutions, contact ikjun.choi@dnalink.com

View original content:http://www.prnewswire.com/news-releases/base10-and-dna-link-partners-on-research-to-support-authorization-of-antibody-test-at-the-point-of-care-301181072.html

SOURCE BASE10 Genetics, Inc.

Read the rest here:
BASE10 and DNA Link Partners on Research to Support Authorization of Antibody Test at the Point of Care - BioSpace

The Personalized Medicines Market grew in 2019, as compared to 2018, according to our report, Personalized Medicines Market is likely to have subdued growth in 2020 due to weak demand on account of reduced industry spending post Covid-19 outbreak. Further, Personalized Medicines Market will begin picking up momentum gradually from 2021 onwards and grow at a healthy CAGR between 2021-2025

Deep analysis about market status (2016-2019), competition pattern, advantages and disadvantages of products, industry development trends (2019-2025), regional industrial layout characteristics and macroeconomic policies, industrial policy has also been included. From raw materials to downstream buyers of this industry have been analysed scientifically. This report will help you to establish comprehensive overview of the Personalized Medicines Market

Get a Sample Copy of the Report at: https://i2iresearch.com/report/global-personalized-medicines-market-2020-market-size-share-growth-trends-forecast-2025/

The Personalized Medicines Market is analysed based on product types, major applications and key players

Key product type:PM DiagnosticsPM TherapeuticsPersonalized Medical CarePersonalized Nutrition & Wellness

Key applications:Hospitals PharmaciesRetail PharmaciesDietary Care CentersOthers

Key players or companies covered are:3G BiotechQuest DiagnosticsLaboratory Corporation of AmericaAbbottAgendia NVAsuragen IncBecton DickinsonCardioDx Inc.Foundation MedicineQiagenSiemens HealthcareAmgenBristol-Myers SquibbGE HealthcarePfizerSanofi23andMeIllumina

The report provides analysis & data at a regional level (North America, Europe, Asia Pacific, Middle East & Africa , Rest of the world) & Country level (13 key countries The U.S, Canada, Germany, France, UK, Italy, China, Japan, India, Middle East, Africa, South America)

Inquire or share your questions, if any: https://i2iresearch.com/report/global-personalized-medicines-market-2020-market-size-share-growth-trends-forecast-2025/

Key questions answered in the report:1. What is the current size of the Personalized Medicines Market, at a global, regional & country level?2. How is the market segmented, who are the key end user segments?3. What are the key drivers, challenges & trends that is likely to impact businesses in the Personalized Medicines Market?4. What is the likely market forecast & how will be Personalized Medicines Market impacted?5. What is the competitive landscape, who are the key players?6. What are some of the recent M&A, PE / VC deals that have happened in the Personalized Medicines Market?

The report also analysis the impact of COVID 19 based on a scenario-based modelling. This provides a clear view of how has COVID impacted the growth cycle & when is the likely recovery of the industry is expected to pre-covid levels.

Contact us:i2iResearch info to intelligenceLocational Office: *India, *United States, *GermanyEmail: [emailprotected]Toll-free: +1-800-419-8865 | Phone: +91 98801 53667

Continued here:
Personalized Medicines Market Segmentation by Application, Market landscape, Market size and forecast by 2026 - The Market Feed

Latest added DNA Sequencing Market research study by MarketDigits offers detailed product outlook and elaborates market review till 2026. The market Study is segmented by key regions that is accelerating the marketization. At present, the market is sharping its presence and some of the key players in the study are GE Healthcare Life Sciences, Johnson & Johnson, Commonwealth Biotechnologies, ZS Genetics, Agilent Technologies, Siemens AG. The study is a perfect mix of qualitative and quantitative Market data collected and validated majorly through primary data and secondary sources.

This report studies the DNA Sequencing Market size, industry status and forecast, competition landscape and growth opportunity. This research report categorizes the DNA Sequencing Market by companies, region, type and end-use industry.

Request for Free Sample Copy of This Report (with COVID-19 Impact ) @ https://marketdigits.com/dna-sequencing-market/sample

Scroll down 100s of data Tables, charts and graphs spread through Pages and in-depth Table of Content on DNA Sequencing Market By Product Type (Instruments, Consumables, and Others), Platform (Sanger, Next Generation Sequencing, qPCR, and Others), Application (Diagnostics, Personalized Medicine, and Others), End User (Hospitals & Healthcare Organizations, Academics & Research Institutions, Pharmaceutical & Biotechnology Companies, and Others) and Geography Global Forecast To 2026. Early buyers will get 10% customization on study.

To Avail deep insights of DNA Sequencing Market Size, competition landscape is provided i.e. Revenue Analysis (M $US) by Company (2018-2020), Segment Revenue Market Share (%) by Players (2018-2020) and further a qualitative analysis is made towards market concentration rate, product/service differences, new entrants and the technological trends in future.

Unlock new opportunities in DNA Sequencing Market; the latest release from MarketDigits highlights the key market trends significant to the growth prospects, Let us know if any specific players or list of players needs to consider to gain better insights.

Grab Complete Details with TOC For Free @ https://marketdigits.com/dna-sequencing-market/toc

Global DNA Sequencing Market: Synopsis

The global DNA sequencing market is projected to achieve an impressive growth rate on the back of its extensive usage in a number of applications and ease of adoption with the emergence of innovative technologies in the field. A DNA sequencing Android app named GelApp 2.0 has been recently introduced with a 56.0% greater band detection accuracy for proteins and 36.0% for DNA than its original version. The interesting, rather most innovative, part of the apps development procedure was the implementation of Monte Carlo Tree Search, a gaming algorithm, for cutting-edge image processing.

With a healthy CAGR of 17.5%, the worldwide DNA sequencing market is foretold to expand substantially from an estimated US$6.6 billion in 2016.

The segmentation followed in this global DNA sequencing report touches four important parameters, including technology. With the help of such critical market segmentation, the analysts have provided a comprehensive study of the global market and significant segments that are envisaged to make a statement in the near future. An illustrative portion on SWOT analysis of the chief segments of the DNA sequencing sector, including characteristic graphics, helps to easily analyze the future outlook of the market.

The report on the international DNA sequencing market takes into account the industry growth drivers, challenges for market players, and current trends impacting the industry. With such conclusive data and accurate statistics, report buyers can devise result-oriented strategies to secure a competitive place in the global market. An in-depth evaluation of the key factors of the market illustrated in the report is a crucial stepping stone to make important business decisions.

Global DNA Sequencing Market : Trends and Opportunities

New market players are envisioned to get attracted to the global DNA sequencing market as technological innovations keep taking precedence. Besides this, established players can expect to leverage new opportunities in the market with sophisticated technologies such as chain terminator DNA sequencing and next generation DNA sequencing. Vendors can also find lucrative opportunities in the clinical field as DNA sequencing is highly demanded in numerous clinical applications. Next generation DNA sequencing, particularly, is receiving high attention with the providence of novel platforms from the ongoing quantum leaps in microfluidics, bioinformatics, nanotechnology, and imaging. Such breakthroughs are also predicted to revolutionize a number of life science fields, thus providing an easy path of growth for the global market.

The services segment of the international DNA sequencing market had secured a colossal share in 2016 while instruments and consumables was one of the most rapidly growing segments in the same year.

Global DNA Sequencing Market: Geography

Considering the most critical geographical regions as a base, the analysts have offered an all-encompassing interpretation of the global DNA sequencing market. North America, Europe, Asia Pacific, and Rest of the World are the major geographies studied in the report. North America could hold a momentous share in the global market while Asia Pacific is anticipated to take advantage of the demand for personalized medicine and higher awareness.

Global DNA Sequencing Market: Company Profiling

Among other prominent players in the global DNA sequencing market, Microchip Biotechnologies, Beckman Coulter (Fullerton, CA), Abbott Laboratories, GE Healthcare Life Sciences, Johnson & Johnson, Commonwealth Biotechnologies, ZS Genetics, Agilent Technologies, Siemens AG, Pacific Biosciences, Myriad Genetics, Bayer Corporation, Integrated DNA Technologies, 454 Life Sciences, Hamilton Thorne Biosciences, and LI-COR Biosciences (Lincoln, NE) are expected to make their presence known.

Market segmentation based on type:

Market segmentation based on technology:

This study by MarketDigits is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers or customers journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.

Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:

The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.

Below is a snapshot of these quadrants.

1. Customer Experience Map

The study offers an in-depth assessment of various customers journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.

2. Actionable Results

The findings presented in this study by MarketDigits are an indispensable guide for meeting all business priorities, including mission-critical ones. The results when implemented have shown tangible benefits to business stakeholders and industry entities to boost their performance. The results are tailored to fit the individual strategic framework. The study also illustrates some of the recent case studies on solving various problems by companies they faced in their consolidation journey.

3. Strategic Frameworks

The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at MarketDigits helps you break down the complex scenario and bring resiliency in uncertain times.

The DNA Sequencing market research report makes an organization armed with data and information generated by sound research methods. This market analysis helps to get up to date about various segments that are relied upon to observe the rapid business development amid the estimate forecast frame. This market research report offers an in-depth overview of product specification, technology, product type and production analysis considering major factors such as revenue, cost & gross margin. DNA Sequencing market report plays very essential role when it is about achieving an incredible growth in the business.

The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:

1. What can be the best investment choices for venturing into new product and service lines?

2. What value propositions should businesses aim at while making new research and development funding?

3. Which regulations will be most helpful for stakeholders to boost their supply chain network?

4. Which regions might see the demand maturing in certain segments in near future?

5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?

6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?

7. Which government regulations might challenge the status of key regional markets?

8. How will the emerging political and economic scenario affect opportunities in key growth areas?

9. What are some of the value-grab opportunities in various segments?

10. What will be the barrier to entry for new players in the market?

DNA Sequencing Market Reports Table of Contents

1.1. Market Definition and Scope

1.2. Market Segmentation

1.3. Key Research Objectives

1.4. Research Highlights

4.1. Introduction

4.2. Overview

4.3. Market Dynamics

4.4. Porters Five Force Analysis

5.1. Technological Advancements

5.2. Pricing Analysis

5.3. Recent Developments

Any Questions? Inquire Here Before Buying @ https://marketdigits.com/dna-sequencing-market/analyst

About Market Digits :

MarketDigits is one of the leading business research and consulting companies that helps clients to tap new and emerging opportunities and revenue areas, thereby assisting them in operational and strategic decision-making. We at MarketDigits believe that market is a small place and an interface between the supplier and the consumer, thus our focus remains mainly on business research that includes the entire value chain and not only the markets.

We offer services that are most relevant and beneficial to the users, which help businesses to sustain in this competitive market. Our detailed and in-depth analysis of the markets catering to strategic, tactical, and operational data analysis & reporting needs of various industries utilize advanced technology so that our clients get better insights into the markets and identify lucrative opportunities and areas of incremental revenues.

Contact Us :

Market Digits

Phone : +91-9822485644

Email : sales@marketdigits.com

Read the original post:
DNA Sequencing Market : Overview Report by 2020, Covid-19 Analysis, Future Plans and Industry Growth with High CAGR by Forecast 2026 - The Courier

Janssen is tapping little-known and privately held Hemera Biosciences for a new gene therapy aimed at reversing a severe disease.

The deal, financials of which are not being shared, is for the investigational phase 1 gene therapy HMR59, which is given as a one-and-done shot into the eye with the aim of helping preserve vision in patients with geographic atrophy, a late-stage and severe form of age-related macular degeneration (AMD).

Accelerate Biologics, Gene and Cell Therapy Product Development partnering with GenScript ProBio

GenScript ProBio is the bio-pharmaceutical CDMO segment of the worlds leading biotech company GenScript, proactively providing end-to-end service from drug discovery to commercialization with professional solutions and efficient processes to accelerate drug development for customers.

Patients with AMD often have low levels of CD59, a protein that protects the retina from damage caused by an essential part of the body's natural immune response called complement. In geographic atrophy, which affects around 5 million people globally, an overactivity of complement destroys cells in the macula, the central part of the retina.

HMR59 is designed to increase the ability of retina cells to make a soluble form of CD59, with the theory being it can help stop further damage and retain a patients vision. It has already passed a phase 1 test, with a second in patients with wet AMD currently conducting follow-up visits to evaluate long-term safety, according to a statement.

There are currently no medical treatments that can regenerate retinal cells that have atrophied, though gene therapies and stem cell therapies are the leading R&D hopes in this field.

Geographic atrophy is a devastating form of AMD that impacts the ability to accomplish everyday tasks, such as reading, driving, cooking, or even seeing faces, said James List, M.D., Ph.D., global therapeutic area head, cardiovascular and metabolism, Janssen R&D.

Our aim with this novel, single-administration gene therapy is to use our development expertise and deep heritage in vision care to help improve patient outcomes by intervening early, halting the progression to blindness, and preserving more years of sight.

RELATED: Biogen boosts gene therapy strategy with Harvard pact focused on inherited eye disease

Janssen will hope to have the success seen from gene therapy Luxturna, a drug from Novartis and Spark Therapeutics for retinal degeneration caused by mutations in the gene RPE65, after the product was approved in 2017, and helped Spark into a Roche buyout in 2019.

Other companies are also on the hunt, such as the recent tie-up between Biogen and Harvard University's Massachusetts Eye and Ear thats aimed at developing a gene therapy to help some patients with these blinding diseases.

The gene at the center of the new agreement, PRPF31, has been linked to autosomal dominant retinitis pigmentosa. Several other gene therapies are being developed to treat retinitis pigmentosa. They include Allergans RST-001, which the company picked up when it acquired RetroSense Therapeutics for $60 million in 2016.

Iveric Bio is also working on a geographic atrophy therapy, known as Zimura (avacincaptad pegol), with it posting clinically meaningful late-stage follow-up data this summer for the complement C5 inhibitor.

Read more from the original source:
Janssen buys up eye disorder gene therapy asset from Hemera Biosciences - FierceBiotech

Bayer has created a cell and gene therapy platform to support its growing pipeline of advanced therapy medicinal products. The platform is intended to enable Bayer to make its expertise and resources available to its partners while preserving their autonomy and culture.

Germanys Bayer has moved into cell and gene therapies on multiple fronts in recent years, buying up induced pluripotent stem cell specialist BlueRock Therapeutics and adeno-associated virus (AAV) gene therapy player Asklepios BioPharmaceutical while investing in a clutch of other biotechs. The deals have given Bayer a pipeline of five advanced assets and more than 15 preclinical prospects.

Accelerate Biologics, Gene and Cell Therapy Product Development partnering with GenScript ProBio

GenScript ProBio is the bio-pharmaceutical CDMO segment of the worlds leading biotech company GenScript, proactively providing end-to-end service from drug discovery to commercialization with professional solutions and efficient processes to accelerate drug development for customers.

Rather than subsume BlueRock and AskBio, Bayer opted to allow the businesses to operate as independent companies in an attempt to preserve their cultures. Yet, Bayer also wants to enable the companies to realize the benefits that can come from being part of a larger organization.

The cell and gene therapy platform is the result of that effort to get the best of both worlds. Bayer will use the platform to provide support to its cell and gene therapy businesses and orchestrate its operations in the area across the product life cycle. Specific areas of support offered by the platform span preclinical through to commercial, strategy implementation and project management.

Bayer is investing in its internal capabilities to strengthen the platform as well as looking to enter into strategic collaborations, acquire technologies and strike licensing deals. The deals entered into so far have given Bayer infrastructure as well as product candidates.

Notably, AskBio has a CDMO unit, Viralgen, specializing in AAV gene therapy production. As limited access to manufacturing capacity has been a barrier to speedy gene therapy development, buying the CDMO could help Bayer remove a constraint on the progress of its programs and become a more attractive partner for startups. Bayer thinks allowing acquired startups autonomy makes it attractive, too.

Wolfram Carius, who joined Bayer from Sanofi in 2016, is heading up the new cell and gene therapy platform. Carius said the platform is vital to accelerate innovation at its source, and to ensure its translation into tangible therapies for patients who have no time to wait in a statement.

Bayers platform is a twist on strategies being pursued by many of its peers, which have identified cell and gene therapies as growth areas and bought in assets but sought to avoid smothering the startups. Kite, for example, operates as its own business unit within Gilead Sciences, and Spark Therapeutics is an independent company within the Roche group.

More:
Bayer creates cell and gene therapy platform to support partners - FierceBiotech

Gene therapy pioneer Jim Wilson and the University of Pennsylvania are teaming up with Regeneron to help deliver its COVID-19 antibody cocktail using adeno-associated virus (AAV) tech in the hope of curbing infection via a nasal spray.

The antibody cocktail, made up of casirivimab and imdevimab, was given a speedy authorization by the FDA less than two weeks ago as a treatment for certain COVID-19 patients. But, keeping up with the fast pace of SARS-CoV-02 R&D, Regeneron is not resting on its laurels and now wants to find a quicker way of delivering its therapy while also working on it as a prophylactic.

Accelerate Biologics, Gene and Cell Therapy Product Development partnering with GenScript ProBio

GenScript ProBio is the bio-pharmaceutical CDMO segment of the worlds leading biotech company GenScript, proactively providing end-to-end service from drug discovery to commercialization with professional solutions and efficient processes to accelerate drug development for customers.

RELATED: Regeneron, following in Lilly's footsteps, wins FDA emergency nod for COVID-19 antibody cocktail

These antibodies are currently injected into patients, but Regeneron and Penn will use Wilsons gene therapy know-how to attempt a nasal spray formulation using AAV vectors. The belief is that this could prevent infection with the virus using a technology typically used in high-tech gene therapies.

The group plans to study the safety and effectiveness of using AAV vectors to introduce the sequence of the cocktails virus-neutralizing antibodies directly to nasal epithelial cells and see whether it can help protect against the disease.

The first step is to finish preclinical trials; if successful, an IND will be sent off to the FDA for human trials.

Wilsons team said it was hopeful that introducing the therapy via single dose of AAV will be able to produce similar protection Regeneron has seen for its cocktail, but for potentially a longer duration.

Regeneron scientists specifically selected casirivimab and imdevimab to block infectivity of SARS-CoV-2, the virus that causes COVID-19, and we have been encouraged by the promising clinical data thus far, said Christos Kyratsous, Ph.D., vice president of research, infectious diseases and viral vector technologies at Regeneron.

In the quest to use cutting-edge science to help end this disruptive and often very devastating disease, we are excited to explore alternate delivery mechanisms such as AAV that may extend the potential benefits of this investigational therapy to even more people around the world.

Read this article:
Wilson, Penn ink Regeneron pact to use gene therapy tech to deliver COVID-19 antibodies - FierceBiotech

Nadofaragene firadenovec, a novel intravesical therapy for nonmuscle-invasive bladder cancer (NMIBC) that does not respond to bacillus Calmette- Gurin (BCG) therapy shows efficacy across important patient subgroups, investigators reported at SUO 2020, the virtual annual meeting of the Society of Urologic Oncology.

In the original phase 3 trial of 157 patients with high-grade BCG-unresponsive NMIBC, clinicians delivered nadofaragene firadenovec (also known as rAd-IFNa/Syn3), a non-replicating adenovirus vector-based gene therapy containing the gene interferon alfa-2b, by catheter into the bladder epithelium once every 3 months. The 3-month complete response rate was 59.6% overall and 53.4% in patients with carcinoma in situ (CIS) with or without Ta or T1 tumors. In addition, 72.9% of patients with high-grade Ta or T1 tumors and no CIS had freedom from high-grade recurrence. At 12 months, 30.5% overall, 24.3% of patients with CIS (with or without Ta or T1 tumors), and 43.8% of patients with high-grade Ta or T1 tumors (and no CIS) were free from high-grade recurrence.

For both the CIS and high-grade Ta/T1 only cohorts, there were no significant differences in response rates at 3 and 15 months between male and female patients, patients younger and older than 70 years, BCG-refractory vs BCG-relapsed disease, patients with more or less than 3 prior lines of therapy, number of prior non-BCG regimens, and patients with more or less than 3 prior courses of BCG, Vikram Narayan, MD, of Emory University in Atlanta, Georgia, reported.

Duration of response also did not differ significantly among groups, Dr Narayan reported. Only in the CIS cohort, patients who received 3 or fewer prior courses of BCG had significantly longer duration of response compared with patients who received more than 3 courses: 12.68 vs 4.96 months. A multivariable analysis confirmed that none of these baseline characteristics or prior therapy significantly influenced response rates at 3 and 15 months or duration of response.

These results demonstrate the efficacy of nadofaragene firadenovec regardless of patient characteristics or prior treatment history, Dr Narayan stated. Nadofaragene firadenovec represents a potential novel treatment option for patients with high-grade BCG-unresponsive NMIBC that advances the current treatment paradigm.

The FDA is currently reviewing a biologics license application (BLA) for nadofaragene firadenovec.

Disclosure: This clinical trial was supported by FKD Therapies in Oy, Finland. Please see the original reference for a full list of authors disclosures.

References

Narayan V, Boorjian S, Alemozaer M, et al. Subgroup analyses of the phase 3 study of intravesical nadofaragene firadenovec in patients with high-grade, BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). Presented at: SUO 2020, December 3-5, 2020. Poster 23.

Boorjian SA, Alemozaffar M, Bad Konety BR, et al. Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial. Published online November 27, 2020. Lancet Oncol. doi:10.1016/S1470-2045(20)30540-4

Kulkarni GS. Nadofaragene firadenovec: a new gold standard for BCG-unresponsive bladder cancer? Published online November 27, 2020. Lancet Oncol. doi:10.1016/S1470-2045(20)30586-6

TechBear. FerGene announces Landmark Phase 3 study published in Lancet Oncology [news release]. FerGene; November 30, 2020.

See the rest here:
Gene Therapy for NMIBC Effective Regardless of Patient Characteristics - Renal and Urology News

Six years after Merck and Bristol Myers Squibb captured the attention of the oncology world with the first approval of their PD-1 drugs Keytruda and Opdivo, sales revenue has started to level off after a host of rivals joined the hunt for new OKs for metastatic conditions, where the FDA has proven quick to act.

But a key analyst covering biopharma believes that theres a vast, still largely untapped frontier for new approvals to come in the adjuvant setting that could once again ignite the growth of these leading cancer franchises. And once again, hes pointing to the 2 leaders in the field as the most likely players to come out ahead way, way ahead.

Unlock this story instantly and join 94,600+ biopharma pros reading Endpoints daily and it's free.

SUBSCRIBE SIGN IN

See more here:
UPDATED: Bayer continues its cell and gene therapy push, enveloping different projects under one strategic roof while hunting new deals - Endpoints...

"The need for reproducible, scalable, and economical production of cell and gene therapies is creating a demand for digital bioprocessing technologies," said Nitin Naik, Global Life Sciences Vice President at Frost & Sullivan. "These technologies are critical to realize the true commercial potential of cell and gene therapies in the next two to three years and serve as a conduit to improve market access and control the total cost of therapy."

Naik added: "From a market segment perspective, while the stem cell market is lucrative, the highest growth is expected to be in gene-modified cell therapies, with a pipeline of 269 products,* followed by gene therapies, which account for 182 assets in the pipeline.* Further, although allogeneic stem cell therapies dominate the marketed product catalogs, interest in disease-modifying CAR-T therapies, which are largely autologous, is driving demand for the evolution of manufacturing technologies, models, and capacity expansion investment by CDMOs." (*as of August 2020)

To tap into the growth prospects exposed by the CGT market, companies must focus on:

Supply Chain Optimization and Decentralized Manufacturing to Expand the Contract Cell and Gene Therapy Manufacturing Market, 20202026 is the latest addition to Frost & Sullivan's Healthcare research and analyses available through the Frost & Sullivan Leadership Council, which helps organizations identify a continuous flow of growth opportunities to succeed in an unpredictable future.

About Frost & Sullivan

For six decades, Frost & Sullivan has been world-renowned for its role in helping investors, corporate leaders and governments navigate economic changes and identify disruptive technologies, Mega Trends, new business models and companies to action, resulting in a continuous flow of growth opportunities to drive future success.Contact us: Start the discussion.

Supply Chain Optimization and Decentralized Manufacturing to Expand the Contract Cell and Gene Therapy Manufacturing Market, 20202026

MF98

Contact:Mariana Fernandez Corporate Communications P: +1 210 348 10 12 E: [emailprotected] http://ww2.frost.com

SOURCE Frost & Sullivan

Excerpt from:
Technological Advancements in Manufacturing Boost the Cell and Gene Therapy Market, Says Frost & Sullivan - PRNewswire

Bayer has launched a Cell and Gene Therapy (C>) Platform, aiming to accelerate its presence in this area.

Bayer is planning to strengthen its capabilities for its internal C> activities, while also pursuing external strategic collaborations, technology acquisitions and licensing.

The overall aim is to establish robust platforms with broad application against a range of therapeutic areas.

The G> Platform will combine multiple functions to provide support across the entire value chain for the research and development of cell and gene therapies.

Bayer has made a series of acquisitions over the last few years to bolster its presence in C>, including its recent acquisition of Asklepios Biopharmaceutical which closed on 1 December, and its acquisition of BlueRock Therapeutics in 2019.

These two companies will be the first to be integrated into the new C> Platform, Bayer has announced in a statement.

The German pharma company added that it has also established a C> pipeline consisting of five advanced assets and over 15 preclinical candidates.

This is a defining moment for Bayer. Cell and gene therapies are leading innovation in healthcare, and it is our goal to be at the forefront of this revolution in science, said Stefan Oelrich, member of the board of management, Bayer AG and president of its pharmaceuticals division.

The C> field is growing at an unprecedented pace. With the establishment of Bayers own C> Platform our company will propel its presence in this area. This will complement our existing C> pipeline which already includes five advanced assets with at least three investigational new drugs annually for the next years, he added.

Go here to see the original:
Bayer establishes new cell and gene therapy platform - PharmaTimes

SALT LAKE CITY (Ivanhoe Newswire)

Gene replacement therapy: Its a game-changer when it comes to treating life-threatening illnesses. It can replace disease-causing genes with healthy genes, knock out a gene thats not working right, or add a new gene to the body to help fight disease. To date, the FDA has approved four types of gene therapy including one that was given the OK just in time to save one little boys life.

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 Cinchs 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.

Cinchs mom, Amber Wight recalled, That was the first time Id ever even heard the term and what it was. And so, it was very scary.

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

My first thought was, hes 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 Russell Butterfield, MD,pediatric neurologist at University of Utah Health and Intermountain Primary Childrens Hospital.

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

Its 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, elaborated Dr. Butterfield.

Just a few years ago, most children born with SMA didnt 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 childs life will be with his new medicine, expressed Dr. Butterfield.

It took a lot of courage for this family to get this far. Thats 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, hell be all right, shared Alex.

Doctors dont 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 dollars. Insurance paid for most of it, but Alex hopes sales from his childrens book will help pay the rest. You can find the book, A Cowboy and His Horse, at https://www.amazon.com/COWBOY-HIS-HORSE-ALEX-WIGHT/dp/B08CWG46ZX.

Contributors to this news report include Cyndy McGrath, Executive Producer; Marsha Lewis, Field Producer; Rusty Reed, Videographer; Roque Correa, Editor.

Read the original:
A little cowboy saved by groundbreaking gene replacement therapy - Wink News

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Dec. 02, 2020 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced that the first patient has been dosed in the high-dose cohort of the Phase 1/2 (Stage 1) study for Type I (infantile) and Type II (late infantile and juvenile onset) GM1 gangliosidosis.

AXO-AAV-GM1 is the only gene therapy in the clinic targeting patients with Type I and Type II GM1 gangliosidosis, a devastating and fatal pediatric disease, said Gavin Corcoran, M.D., Chief R&D Officer of Sio. The initiation of the high-dose cohort builds on evidence of extension of survival in naturally-occurring GM1 disease animal models and encouraging clinical data from an expanded access study conducted by a National Human Genome Research Institute (NHGRI) team led by our principal investigator, Dr. Cynthia Tifft at the National Institutes of Healths (NIH) Clinical Center. Our team and academic partners are dedicated to improving the lives of children affected by this devastating disease, and we look forward to reporting topline data from the low-dose cohort before year end.

The Phase 1/2 study (NCT03952637) is designed to evaluate the safety, tolerability, and potential efficacy of AXO-AAV-GM1 delivered intravenously in patients with Type I and Type II GM1 gangliosidosis.

AXO-AAV-GM1 has received both Orphan Drug Designation and Rare Pediatric Disease Designation and is the only gene therapy in clinical development for both Type I and Type II GM1 gangliosidosis.

GM1 gangliosidosis is a progressive and fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene that cause impaired production of the -galactosidase enzyme. Currently, there are no approved treatment options for GM1 gangliosidosis. In 2019, Sio reported clinically meaningful improvements from baseline to six-month follow-up for the first GM1 Type II child dosed with low-dose AXO-AAV-GM1 gene therapy under an expanded access protocol.

About AXO-AAV-GM1

AXO-AAV-GM1 delivers a functional copy of the GLB1 gene via an adeno-associated viral (AAV) vector, with the goal of restoring -galactosidase enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1s ability to improve -galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.

About Sio Gene Therapies

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visit http://www.siogtx.com.

In 2018, Sio licensed exclusive worldwide rights from the University of Massachusetts Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as will, expect, believe, estimate, and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding costs associated with its operating activities are forward-looking. All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the development of a suspension-based manufacturing process for AXO-Lenti-PD; the scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Sios scientific approach and general development progress; and the availability or commercial potential of Sios product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Sios most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 13, 2020, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts:

Media

Josephine Belluardo, Ph.D.LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

Investors and Analysts

David NassifSio Gene Therapies Inc.Chief Financial Officer and General Counsel(646) 677-6770investors@siogtx.com

See original here:
Sio Gene Therapies Announces First Patient Dosed in High-Dose Cohort of AXO-AAV-GM1 Clinical Trial in Patients with GM1 Gangliosidosis - BioSpace

When I first started at CHEO as a neuromuscular expert in 2010, there were absolutely no treatments available, he said, noting that children diagnosed then with SMA were given the necessary nutritional and respiratory supports, and assisted devices such as wheelchairs, to help them live the best life possible.

But research at CHEO, led by Dr. Alex MacKenzie, who discovered a gene NAIP which may contribute to the severity of the disease, helped develop treatment therapies.

One of them, which Aidan was first given when he was about three weeks old, is known as Spinraza. Approved in Canada, it corrects how the faulty gene produces certain proteins, but requires repeated treatments via spinal taps throughout the persons life.

A second treatment, a gene-replacement therapy called Zolgensma, only needs to be administered once, but is not yet approved in Canada and is prohibitively expensive: more than $2 million in the U.S.

Our immediate families volunteered to remortgage their houses and help fundraise, recalls Adam, while the couple planned to sell their house and move in with family.

Fortunately, they didnt have to. Through the manufacturers managed access program, the drug was made available on a compassionate basis to Aidan, who received it two weeks after his first treatment.

Theres no definitive test to say how well Aidans treatment has worked, but, according to Dr. McMillan, many of the newborns who initially received Zolgensma are now five and six years old, and they continue to show robust strong effects to the gene-replacement therapy, and theyve had no symptoms of clinical deterioration and no signs of any wearing-off effects.

For Sully and Deschamps, the proof so far is in seeing their son approach his first birthday with no signs of the disease. We see him as a physically active child who climbs two sets of stairs with ease and cruises around the house like he owns it. He dances to music, he throws and chases balls, and he chases our cats. We are so thankful.

bdeachman@postmedia.com

More:
Ottawa baby a pioneer of gene therapy for rare disease - Windsor Star

Professor Laurence D Hurst explains why understanding the nucleotide mutations in viruses, including SARS-CoV-2, can have significant implications for vaccine design.

With 61 codons specifying 20 amino acids, some can be encoded by more than one codon and it is often presumed that it does not matter which one a gene uses. When I first studied genetics, some books I read taught that mutations between such alternative codons (eg, GGA->GGC, both giving glycine) were called synonymous mutations, while others referred to them as silent mutations. However, are synonymous mutations really silent meaning they are identical in terms of fitness and function? Although they may specify the same amino acid, does that mean they are all the same?

Figure 1: Intronless GFP transgene expression is higher for variants of GFP with higher GC content at synonymous sites5

Perhaps one of the biggest surprises over recent years has been the discovery that versions of the same gene, differing only at synonymous sites, can not only have different properties, but effects that are not modest.1-5 For example, two versions of green fluorescent protein (GFP) differing only at synonymous sites can have orders of magnitude differences in their expression level.4 We similarly recently discovered that for an intronless transgene to express in human cell lines it needs to be GC rich, which can be achieved by altering the synonymous sites,5 as seen in Figure 1. It is no accident, we suggest, that the well-expressed endogenous intronless genes in humans (such as histones) are all GC rich and that our functional retrogenes tend to be richer in GC content than their parental genes.

The realisation that synonymous sites matter has clear relevance to the design of transgenes or other artificial genes, be these for experiments, gene therapy, protein production (eg, in bacteria) or for vaccine design. In the case of vaccines, we might wish to modulate a viral protein to be effectively expressed in human cells to illicit a strong and robust immune response.6 Conversely to the design of attenuated vaccines, we seek to produce a tuned down version of the virus that can function but is weak.7

The challenge is knowing not just which synonymous sites can be altered but knowing how they should be altered. One approach is mass randomisation try many alternatives and see what works.4,8,9 In principle this is fine, but this approach requires many randomisations, which is still technically difficult for long attenuated viruses. An alternative strategy that we have been exploring is to let nature tell us; we can apply tools and ideas from population genetics to better understand what natural selection favours and disfavours and in turn to estimate the strength of selection.

it will be interesting to see if we can learn a lesson from nature as to how to weaken a virus

Estimation of the strength of selection is possible from knowledge of the site frequency spectrum, (ie, how common variants are) from which we can infer the distribution of fitness effects (DFE). If a site is under strong purifying selection, then mutations may occur in the population but these are rapidly eliminated, so variants are always rare. By contrast, if they are selectively neutral, we expect some variants to be quite common. We recently applied this methodology to show that synonymous mutations in human genes that disrupt exonic splice enhancer motifs are often under strong selection and affect many synonymous sites in our genes.10 This has implications for both diagnostics and for transgene design for gene therapy, as we often remove introns in heterologous genes, so freeing up these residues from their role in specifying exons ceases.11

The same DFE methodology cannot easily be applied to viruses, as the methods assume free recombination (ie, we assume one mutation does not impact the fate of others in the same genome). However, other population genetical tools can still be applied. Recently, we examined SARS-CoV-2 and identified the profile of mutations that we see at four-fold degenerate sites.12 From this profile we could estimate what the synonymous site composition would be, assuming that the only forces are mutational biases and neutral evolution (ie, no selection). We observed that in this genome there is a strikingly strong C->U mutation bias and a G->U one. In the raw data this is not so obvious as G and C are quite rare. However, the mutability of the sites per occurrence of the site reveals the underlying patterns.

Figure 2: The rate of mutational flux from one dinucleotide to another in the coding sequence of SARS-CoV-2. The direction of flux is indicated by the indentation of the connecting links: the inner layer represents flux out while the outermost layer represents flux into the node. The frequency of the flux exchange is represented by the width of any given link where it meets the outer axis. Dinucleotide nodes are coloured according to their GC-content. Hence, it is evident that there is high flux away from GC-rich dinucleotides whereas AU-rich dinucleotides are largely conserved.12

With knowledge of the mutational bias we then asked what the equilibrium frequency of the four nucleotides would be using four simultaneous equations. This is the nucleotide content at which for every mutation changing a particular base there is an equal and opposite one creating the same base somewhere else in the genome, ensuring overall unchanged nucleotide content. Given the strong C->U and G->U mutational biases, it is no surprise that the equilibrium content is very U rich (we estimate equilibrium U content should be about 65 percent). However, while the four-fold sites are indeed U rich, they are not that U rich, being closer to 50 percent. A clue as to why the mutation bias is so skewed to generating U comes from analysis of equilibrium UU content: UU residues are predicted to be very common, with CU residues being particularly mutable generating UU (Figure 2) this is expected due to human APOBEC proteins attacking and mutating/editing the virus.13

One probable explanation for this difference between predicted and observed nucleotide content is selection against U content. There may be many U residues appearing in the population, but many are pushed out of the population owing to purification selection, ie, because of the deleterious effects of the mutations. That such selection is happening in the SARS-CoV-2 genome is also clear from the sequence data. We estimate that for every 10 mutations that appear in the sequence databases, another six are lost because of selection prior to genome sequencing. Indeed, UU content is about a quarter of that predicted (Figure 3).

Figure 3: The predicted (under neutral mutational equilibrium) and observed dinucleotide content of SARS-CoV-2. Note the very high predicted levels of UU given the strong mutational flux to UU residues (see Figure 2) and the net underrepresentation in actual sequence.9

This leaves two problems: why is selection operating on SARS-CoV-2 and what can we do with this information? In some cases, we have a good idea as to why: many mutations to U at codon sites generate stop codons. However, we have observed that U destabilises the transcripts and is associated with lower-reported transcript levels;12 a full explanation of the causes of selection on nucleotide content therefore requires manipulation of the sequences.

The second question, what to do with this information, is perhaps more urgent. It has previously been noted that nucleotide content manipulation is a viable means to attenuate viruses.7 Currently there are three groups investigating this route to make a vaccine for SARS-CoV-2: Indian Immunologicals Ltd/Griffith University, Codagenix/Serum Institute of India and Acbadem Labmed Health Services/Mehmet Ali Aydinlar University. In prior attempts, attention has been paid to CpG levels and UpA levels (which we find to be correlated between SARS genes and between different viruses).12 CpGs attract the attention of zinc antiviral protein (ZAP) and UpA attracts an RNAase L. Not surprisingly, some viruses, including SARS-CoV-2, therefore have low levels of both dinucleotide pairs given the levels of the underlying nucleotides.

The challenge is knowing not just which synonymous sites can be altered but knowing how they should be altered

In the past, attenuation strategies have focused on modulating synonymous sites to increase CpG and UpA, making the virus more visible to antiviral proteins.14 We in turn suggest a general strategy to utilise this method and to increase U content as well.12 Given the evidence that selection on the virus is to reduce U content, while our antiviral proteins are mutating it to increase U content, it will be interesting to see if we can learn a lesson from nature as to how to weaken a virus. This is an unusual circumstance in which we predict that we should build in more of the already most common synonymous site nucleotides (U in this case) to degrade the virus. More generally, it is assumed that the most used codons are those that tend to increase the fitness of the organism. In the face of such a severe mutation bias, however, this simpler logic no longer holds.

Laurence D Hurst is Professor of Evolutionary Genetics and Director of the Milner Centre for Evolution at the University of Bath. He is currently also the President of the Genetics Society. He completed his D.Phil in Oxford, after which he won a research fellowship and then moved to Cambridge University as a Royal Society Research Fellow. While on the fellowship he assumed his current Chair at Bath University. In 2015 he was elected a Fellow of the Academy of Medical Sciences and a Fellow of the Royal Society. He is a recipient of the Genetics Society Medal and the Scientific Medal of the Zoological Society of London.

Related topicsDisease research, DNA, Gene Therapy, Genetic analysis, Genomics, Protein, Proteogenomics, Proteomics, Research & Development, RNAs, Vaccine

See the article here:
Tweaking synonymous sites for gene therapy and vaccines - Drug Target Review

SEATTLE--(BUSINESS WIRE)--Shape Therapeutics, Inc. (ShapeTX), a next-generation gene therapy company with an industry-leading RNA targeting technology platform, announces today the unveiling of the AAVidTM capsid discovery platform and results from its first AAV5 variant library in a non-human primate selection campaign.

The AAVidTM capsid discovery platform uses non-random mutational fitness to create massive capsid libraries of billions of unique AAV variants for direct-to-NHP in vivo biological selection. By combining cutting-edge DNA synthesis, advanced synthetic biology, next-generation sequence barcoding and machine learning algorithms, ShapeTX generates industry-leading library size and diversity to enable the development of best-in-class human therapeutics.

Wildtype first-generation AAVs are enabling the recent advances in gene therapy, but they have been plagued by toxicities in the clinic due in part to a lack of tissue specificity, resulting in the need for high doses. Our AAVidTM platform solves the issue by creating novel capsid variants with specific tissue-tropism, said Francois Vigneault Ph.D., President and CEO at ShapeTX. Weve stayed quiet for the past three years while developing a superior AAV platform technology and are excited to announce that we have best-in-class AAV variants in hand. Today, we are announcing our novel liver-tropic AAV5 variants stay tuned for more to come.

David J. Huss, Ph.D., Vice President and Head of Research added, The vast structural space for exploration at the AAV capsid/target cell interface necessitates enormous library size and diversity, which until now, has only been probed with capsid library sizes in the tens of thousands to millions. At ShapeTX, we set out to create a superior AAV capsid discovery platform with library sizes in the billions of unique variants, thereby maximizing the opportunity for novel virus/target cell interactions. Dr. Huss presented the details of the platform at the 2nd RNA Editing Summit on Dec. 2, 2020.

About Shape Therapeutics, Inc

Shape Therapeutics is a biotechnology company developing next-generation RNA-targeted therapies to treat the worlds most challenging diseases. The ShapeTX technology platform includes RNAskip, a proprietary suppressor tRNA technology that enables premature stop codon readthrough; RNAfixTM, a precision RNA editing technology using endogenous Adenosine Deaminase Acting on RNA (ADAR); and AAVidTM, a next-generation engineered adeno-associated virus (AAV) platform producing highly specific, tissue-tropic AAVs. The power of the ShapeTX platforms resides in redirecting the cellular machinery already present in our cells, thereby bypassing the risks of immunogenicity and DNA damage seen with other contemporary editing technologies. ShapeTX is committed to data-driven scientific advancement, passionate people and a mission of providing life-long cures to patients. Shape Life!

Excerpt from:
Shape Therapeutics Unveils AAVid Capsid Discovery Platform and Identification of Novel Tissue-Specific AAV Variants, Solving a Fundamental Delivery...