Archive for the ‘Gene Therapy Research’ Category

Avantor executives joined other industry leaders at virtual Cell & Gene Therapy Bioprocessing & Commercialization Conference

Panels discussed critical topics including process efficiency and scaling solutions

RADNOR, Pa., Nov. 2, 2020 /PRNewswire/ --Executives from Avantor Inc. (NYSE: AVTR), a leading global provider of mission-critical products and services to customers in the life sciences, advanced technologies and applied materials industries, recently provided expert insight at the Cell & Gene Therapy Bioprocessing & Commercialization Virtual Conference. Cell and gene therapy (C>) are two of the most revolutionary applications driving the biopharmaceutical industry.

In separate panel discussions with other global industry leaders, Dr. Ger Brophy, EVP, Biopharma Production at Avantor and Claudia Berrn, SVP, Business Development and Commercial Operations at Avantor, each addressed novel considerations and challenges facing the biopharmaceutical industry, including the impact of COVID-19. They highlighted innovation to help drive the creation and commercialization of life-changing, personalized C> treatments.

In a panel on the evolution of C> and the path toward scalability and manufacturability of these treatments, Dr. Brophy gave his perspective on this growing need for the bioprocessing industry.

"Genuine progress is being made in the long-standing battle to effectively treat and control disease, and cell & gene therapies will only continue to unlock new frontiers in medicine," said Dr. Brophy. "We're beginning to see more clearly the issues that need to be addressed, and know that if products are to scale and become more accessible to patients worldwide, there needs to be efficiency in operations. There is an absolute requirement for automation, both to reduce variability and to generate process efficiencies. At Avantor, we are ready to make the essential materials and technologies available to companies leading the charge and treating patients. Innovation and agility are central to how we're partnering with the industry to resolve these issues."

On a separate panel, Ms. Berrn highlighted how the industry is navigating the challenges and impact of the global pandemic, from the fragmentation of supply chains to clinical trial disruption.

"In this critical moment for the industry and the world, it is more important than ever for leaders in the cell & gene therapy community to come together to strategize and share ideas," said Ms. Berrn. "Avantor is actively working with the world's leading pharmaceutical and biotechnology companies to accelerate the production of novel treatments. Collectively, our goal is to mitigate any challenges in the process of taking a groundbreaking treatment from its initial scientific discovery to delivery in treating patients."

Avantor provides products for biopharma production workflows, including cell and gene therapy offering. Search 'Avantor biopharma' in your browser.

About AvantorAvantor, a Fortune 500 company, is a leading global provider of mission-critical products and services to customers in the biopharma, healthcare, education & government, and advanced technologies & applied materials industries. Our portfolio is used in virtually every stage of the most important research, development and production activities in the industries we serve. One of our greatest strengths comes from having a global infrastructure that is strategically located to support the needs of our customers. Our global footprint enables us to serve more than 225,000 customer locations and gives us extensive access to research laboratories and scientists in more than 180 countries.We set science in motion to create a better world. For information, visit avantorsciences.com and find us on LinkedIn, Twitter and Facebook.

Robert DonohoeSenior Director, Corporate CommunicationsAvantorM: 484-688-4730[emailprotected]

Source: Avantor and Financial News

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http://avantorsciences.com

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Avantor Discusses Cell & Gene Therapy Production Trends Through the Lens of COVID-19 - PRNewswire

This report studies the Cell and Gene Therapy to get Covid-19 marketplace with Many details of the industry such as the market size, market standing, marketplace trends and prediction, the report also provides brief advice of their opponents as well as the particular growth opportunities with key market drivers. Locate the complete Cell and Gene Therapy to get Covid-19 market evaluation segmented by firms, area, type and software in the document.

New sellers from the marketplace are facing tough competition from Established foreign vendors as they fight with technological inventions, quality and reliability problems. The report will answer questions regarding the present market changes and the reach of competition, opportunity cost and much more.

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The report discusses the various Kinds of options for While the areas considered in the range of the report include North America, Europe, and assorted others. The analysis also highlights on how climbing digital security dangers is altering the industry situation.

Development policies and strategies are discussed along with Manufacturing processes and cost structures will also be examined. This report also claims import/export consumption, supply and demand Statistics, price, cost, earnings and gross earnings.

This report concentrates on the international Cell and Gene Therapy for Covid-19 Status, future prediction, growth opportunity, key marketplace and players.

The following players are covered in this report:

Amgen Inc.

bluebird bio, Inc.

Dendreon Pharmaceuticals LLC.

Fibrocell Science, Inc.

Human Stem Cells Institute

Kite Pharma, Inc.

Kolon TissueGene, Inc.

Novartis AG

Orchard Therapeutics plc.

Organogenesis Holdings Inc.

Pfizer, Inc.

RENOVA THERAPEUTICS

Shanghai Sunway Biotech Co., Ltd.,

Sibiono GeneTech Co. Ltd.,

Spark Therapeutics, Inc.

Vericel Corporation

ViroMed Co., Ltd.

The Cell and Gene Therapy for Covid-19 marketplace is a comprehensive record Which supplies a meticulous summary of the market share, size, trends, demand, product evaluation, program analysis, regional perspective, competitive strategies, predictions, and strategies affecting the Cell and Gene Therapy for Covid-19 Industry. The report contains a thorough analysis of this marketplace competitive landscape, with the assistance of comprehensive business profiles, SWOT analysis, project feasibility analysis, and a lot of other specifics about the essential companies working on the marketplace.

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The analysis aims Covid-19 in global sector.

Breakdown Data by Type

Rare Diseases

Oncology

Hematology

Cardiovascular

Ophthalmology

Neurology

Other Therapeutic Classes

Cell and Gene Therapy Breakdown Data by Application

Pharmaceutical and Biotechnology Companies

Research and Academic Institutions

Contract Research Organizations (CROs)

Hospital

Others

Based on regional and country-level analysis, the Cell and Gene Therapy market has been segmented as follows:

North America

United States

Canada

Europe

Germany

France

U.K.

Italy

Russia

Nordic

Rest of Europe

Asia-Pacific

China

Japan

South Korea

Southeast Asia

India

Australia

Rest of Asia-Pacific

Latin America

Mexico

Brazil

Middle East & Africa

Turkey

Saudi Arabia

UAE

Rest of Middle East & Africa

In the competitive analysis section of the report, leading as well as prominent players of the global Cell and Gene Therapy market are broadly studied on the basis of key factors. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on price and revenue (global level) by player for the period 2015-2020.

The following players are covered in this report:

Amgen Inc.

bluebird bio, Inc.

Dendreon Pharmaceuticals LLC.

Fibrocell Science, Inc.

Human Stem Cells Institute

Kite Pharma, Inc.

Kolon TissueGene, Inc.

Novartis AG

Orchard Therapeutics plc.

Organogenesis Holdings Inc.

Pfizer, Inc.

RENOVA THERAPEUTICS

Shanghai Sunway Biotech Co., Ltd.,

Sibiono GeneTech Co. Ltd.,

Spark Therapeutics, Inc.

Vericel Corporation

ViroMed Co., Ltd.

The Cell and Gene Therapy for Covid-19 market study report entirely Covers the very important data of their capacity, manufacturing, value, cost/profit, supply/demand import/export, further separated by country and company, and from application/type for the best possible upgraded information representation from the statistics, tables, pie graph, and charts. These data representations give predictive information concerning the potential estimations for persuasive market development. The detailed and in depth understanding concerning our publishers makes us from the box in the event of market evaluation.

Key questions Answered within this report

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Table of Contents

Chapter 1: Global Cell and Gene Therapy for Covid-19 Market Overview

Chapter 2: Cell and Gene Therapy for Covid-19 Market Data Analysis

Chapter 3: Cell and Gene Therapy for Covid-19 Technical Data Analysis

Chapter 4: Cell and Gene Therapy for Covid-19 Government Policy and News

Chapter 5: Global Cell and Gene Therapy for Covid-19 Market Manufacturing Process and Cost Structure

Chapter 6: Cell and Gene Therapy for Covid-19 Productions Supply Sales Demand Market Status and Forecast

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Cell and Gene Therapy Market Reviewed in a New Study Eurowire - Eurowire

DUBLIN, Nov. 4, 2020 /PRNewswire/ -- The "Global Non-Oncology Precision Medicine Market: Focus on Application Area, Ecosystem Type, Country Data (15 Countries) - Analysis and Forecast, 2020-2030" report has been added to ResearchAndMarkets.com's offering.

The global non-oncology precision medicine market was valued at $41.1 billion in 2019, and it is expected to grow at an impressive double-digit rate of 11.03% and reach a value of $129.96 billion in 2030.

The precision medicine approach is used for several disease management, such as oncology, immunology, neurology, and infectious diseases. In the non-oncology precision medicine market, the application of molecular biology is for studying the cause of a patient's disease at the molecular level for diseases other than cancer, so that target-based therapies or individualized therapies can be applied to cure the patient's health-related problems.

The existing non-oncology precision medicine market is favored by multiple factors, which include rising government initiatives, coupled up with the general population's growing awareness pertaining to molecular level diagnosis and treatment. Additionally, the increasing number of advancements in molecular diagnostics, more key players interested in investing for the development of novel personalized therapies, and increasing FDA approvals for such drugs among others.

Competitive Landscape

The non-oncology precision medicine market witnessed 59 collaborations and partnerships, four fundings and investments, 44 product launches and enhancements,26 regulatory and legal activities,18 mergers and acquisitions, and six business expansions during the period January 2016-August 2020.

Key Topics Covered:

1 Product Definition

2 Scope of the Research Study

3 Research Methodology3.1 Primary Data Sources3.2 Secondary Data Sources3.3 Market Estimation Model3.4 Selection Criteria for Company Profiles

4 Market Overview4.1 Introduction4.2 Enabling Technologies of Precision Medicine Emphasizing Focus on Prevention4.3 Promising Potential and Role of Precision Medicine

5 Impact of COVID-19 Pandemic on Global Non-Oncology Precision Medicine Market5.1 Unprecedented Demand for Non-Oncology Precision Medicine Due to COVID-195.2 COVID-19 Affecting Supply Chain of Non-Oncology Precision Medicine-Based Therapies5.3 Measures Taken to Address Current Problem

6 Global Non-Oncology Precision Medicine Market: Industry Analysis6.1 Legal and Regulatory Framework and Requirements6.1.1 North America6.1.1.1 Regulatory Requirements in U.S.6.1.2 Europe6.1.3 Asia-Pacific6.2 Patent Landscape

7 Global Non-Oncology Precision Medicine Market: Market Dynamics7.1 Overview7.2 Market Driving Factors7.2.1 Improved Availability of Targeted Therapies and Decreasing Trial and Error-Based Prescription7.2.2 Reduced Chances of Adverse Drug Reactions7.2.3 Decreasing Overall Cost of Genome Sequencing7.2.4 Rising Prevalence of Chronic Diseases7.3 Market Restraining Factors7.3.1 Unclear Regulatory Framework and Reimbursement Scenario for Personalized Medicine7.3.2 Limited Knowledge About Molecular Mechanism/Interaction7.3.3 Integrating and Securing Electronic Health Records7.4 Market Opportunities7.4.1 Research Alliance Across Industry and Academia Accelerating Market Entry7.4.2 Strong Pipeline and Increasing FDA Approvals

8 Global Non-Oncology Precision Medicine Market: Competitive Landscape8.1 Key Strategies and Developments8.1.1 Product Offerings8.1.2 Partnerships and Alliances8.1.3 Regulatory and Legal8.1.4 Mergers and Acquisitions8.1.5 Funding and Investments8.2 Market Share Analysis8.3 Growth-Share Analysis (by Company), 2019

9 Global Non-Oncology Precision Medicine Market (by Application), $Billion, 2019-20309.1 Overview9.2 Infectious Diseases9.2.1 Respiratory Infections9.2.2 Gastrointestinal Infections9.2.3 Sexually Transmitted Infections9.2.4 Others9.3 Neurology/Physiatry9.3.1 Neurodegenerative Disorders9.3.2 Neuropsychiatric Disorders9.3.3 Others9.4 Lifestyle and Endocrinology9.5 Cardiovascular9.5.1 Cardiac Myopathies and Arrhythmia9.5.2 Others9.6 Gastroenterology9.7 Other Applications

10 Global Non-Oncology Precision Medicine Market (by Ecosystem), $Billion, 2019-203010.1 Overview10.2 Applied Sciences10.2.1 Genomics10.2.2 Global Non-Oncology Precision Medicine Genomics Market (by Technology)10.2.2.1 Polymerase Chain Reaction (PCR)10.2.2.2 Precision Medicine Next-Generation Sequencing (PM NGS)10.2.2.3 Genome Editing10.2.2.4 Other Technologies10.2.3 Pharmacogenomics10.2.4 Other Applied Sciences10.3 Precision Diagnostics10.3.1 Molecular Diagnostics (MDx)10.3.2 Medical Imaging10.4 Digital Health and Information Technology10.4.1 Clinical Decision Support Systems (CDSS)10.4.2 Big Data Analytics10.4.3 IT Infrastructure10.4.4 Genomics Informatics10.4.5 In-Silico Informatics10.4.6 Mobile Health10.5 Precision Therapeutics10.5.1 Clinical Trials10.5.2 Cell Therapy10.5.3 Drug Discovery and Research10.5.4 Gene Therapy

11 Global Non-Oncology Precision Medicine Market (by Region), $Billion, 2019-2030

12 Company Profiles12.1 Company Overview12.2 Role of Abbott Laboratories in Global Non-Oncology Precision Medicine Market12.3 Financials12.4 SWOT Analysis

For more information about this report visit https://www.researchandmarkets.com/r/j2b11m

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$129.96 Billion Non-Oncology Precision Medicine Market, 2019-2020 & 2030 - Research Alliance Across Industry and Academia Accelerating Market...

The gene therapy market has undergone a series of transformation from past few years. Initially gene therapy of monogenetic diseases was popular; however now the gene therapy market is gaining popularity for treatment of cancer. Cancer gene therapy refers to a process of treatment of cancer by inserting therapeutic DNA into patient. Cancer gene therapy is gaining popularity as a result of its success rate in preclinical and clinical trial stages. The most common technique for cancer gene therapy involves replacing a mutated gene that is causing cancer with a healthy copy of gene. Other technique involves inactivation of a gene that is not working properly. A new technique that is being introduced to cancer gene therapy market involves insertion of new genes into the body that would help to fight against tumor cells.

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North America followed by Europe dominates the global market for cancer gene therapy market due to large number of aging population and technological advancement in the region. Asia is expected to witness high growth in cancer gene therapy market, due to increasing government initiatives, rising economy and improvement in healthcare infrastructure in the region. Some of the key driving forces for cancer gene therapy market in emerging countries are increasing R&D investment, large pool of patients and rising government funding.

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Several unmet medical needs for treatment of cancer have encouraged R&D of cancer gene therapy. Various factors, such as increasing prevalence of cancer, rising government initiatives, increasing funding from various government and non-government organizations, are driving the global cancer gene therapy market. In addition, ethical acceptance of gene therapy for treatment of diseases and growing popularity of DNA vaccines is driving the global cancer gene therapy market. However, less awareness and high cost involved in treatment are restraining the growth of global market for cancer gene therapy.

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Innovation of some innovative therapy with better success rate is expected to offer good opportunity for cancer gene therapy. In addition, despite of high cost involved in R&D, pharmaceutical companies are showing increased interest in this field. This is expected to offer good potential for cancer gene therapy market. Currently, most of the cancer gene therapy products are in clinical trial phases. The market is expected to rise after clearance or success of these products from clinical trials. Some of the major companies operating in the global cancer gene therapy market are Cell Genesys, Advantagene, GenVec, BioCancell, Celgene and Epeius Biotechnologies. Other leading players in cancer gene therapy market include Introgen Therapeutics, ZIOPHARM Oncology, MultiVir and Shenzhen SiBiono GeneTech

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The Cancer Gene Therapy Market to get on to the elliptical growth mode in the next decade - Eurowire

Fact.MR has recently announced the addition of a new study on the global Gene Therapy market to its document repository. This report is intended to provide a detailed analysis of all vital factors impacting negatively or positively on the development of the Gene Therapy market during the assessment period of 20AA to 20YY. Apart from this, the latest study highlights historical and current trends together with forecast on future trends shaping the growth of the global Gene Therapy market in the years ahead. This aside, the assessment of Gene Therapy Market presents analysis on challenges and opportunities during the assessment period.

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This growth is attributed to a plethora of factors. Increased research and development activities, growing investments in the Gene Therapy, and favorable regulatory policies by government bodies of several countries are some of the important factors driving the Gene Therapy market growth.

The recent COVID-19 (Coronavirus) pandemic has affected the revenues of companies engaged in almost every industrial sector. The companies operating in the global Gene Therapy market are also not an exception to this. The recently published Gene Therapy market research report sheds light on various strategies employed by industry leaders to sustain during this critical scenario of COVID-19 pandemic. In addition to this, the study includes various tactics used by market players to resolve many challenges related to the distribution of their products during the lockdown period.

The latest research report gives trustworthy data on different consumption patterns in various geographical regions.

Important regions covered in this report on the global Gene Therapy market include:

Based on product type, the global Gene Therapy market is classified into:

The research report profiles important players working in the Gene Therapy market. In addition to this, it covers data on the competitive landscape and various strategies utilized by these players to maintain their leading position in the market for Gene Therapy. Mergers, acquisitions, partnerships, collaborations, joint ventures, and new product launches are some of the key strategies utilized by vendors in the Gene Therapy market. Apart from this, the assessment gives important data on weaknesses, strengths, threats, and opportunities for all vendors working in the market for Gene Therapy.

The list of prominent players in the global Gene Therapy market includes the following names:

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Press Release-

Originally posted here:
Gene Therapy Size Inclined to Project Higher Revenue Share during the Stated Forecast Period 2020 2026 - The Cloud Tribune

Belgian companies Novasep and Handl Therapeutics have signed an agreement for the development and manufacturing of an adeno associated virus (AAV)-based gene therapy drug candidate for the treatment of neurodegenerative diseases.

Handl is developing multiple therapeutic AAV-based programs in collaboration with the Katholieke Universiteit Leuven, with the Center for Applied Medical Research of the University of Navarra, Spain, and with the Biomedical Neuroscience Institute of the University of Chile. The company is currently conducting invesrigational new drug (IND)-enabling pre-clinical studies.

Under the terms of the agreement, Novasep will develop and manufacture AAV vectors designed for these programs and will supply drug substance and drug product to support Handl Therapeutics preclinical and clinical studies.

Michael Linden, co-founder and head of research and development at Handl, said: We are excited to engage with Novasep to develop GMP manufacture capabilities for our novel gene therapies and are happy to have identified an outstanding partner right on our doorstep here in Belgium.

Cedric Volanti, Novaseps president of biopharma solutions, said: This new agreement recognizes Novaseps expertise in the field of viral vectors and will contribute to the important development of the cell and gene therapy market in Belgium.

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BRIEFNovasep and Handl sign gene therapy product development and manufacturing deal - The Pharma Letter

A new research study has been presented by dataintelo offering a comprehensive analysis on the Global Cancer Gene Therapy Market where user can benefit from the complete market research report with all the required useful information about this market. This is a latest report, covering the current COVID-19 impact on the market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions. The rapidly changing market scenario and initial and future assessment of the impact is covered in the report. The report discusses all major market aspects with expert opinion on current market status along with historic data. This market report is a detailed study on the growth, investment opportunities, market statistics, growing competition analysis, major key players, industry facts, important figures, sales, prices, revenues, gross margins, market shares, business strategies, top regions, demand, and developments.

The Cancer Gene Therapy Market report provides a detailed analysis of the global market size, regional and country-level market size, segment growth, market share, competitive landscape, sales analysis, impact of domestic and global market players, value chain optimization, trade regulations, recent developments, opportunity analysis, strategic market growth analysis, product launches, and technological innovations.

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Major Players Covered in this Report are: AdaptimmuneBluebird bioCelgeneShanghai Sunway BiotechShenzhen SiBiono GeneTechSynerGene TherapeuticsAltor BioScienceAmgenArgenxBioCancellGlaxoSmithKlineMerckOncoGenex PharmaceuticalsTransgene

Global Cancer Gene Therapy Market SegmentationThis market has been divided into Types, Applications, and Regions. The growth of each segment provides an accurate calculation and forecast of sales by Types and Applications, in terms of volume and value for the period between 2020 and 2026. This analysis can help you expand your business by targeting qualified niche markets. Market share data is available on the global and regional level. Regions covered in the report are North America, Europe, Asia Pacific, the Middle East & Africa, and Latin America. Research analysts understand the competitive strengths and provide competitive analysis for each competitor separately.

By Types:Oncolytic VirotherapyGene TransferGene-Induced Immunotherapy

By Applications:HospitalsDiagnostics CentersResearch Institutes

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Global Cancer Gene Therapy Market Regions and Countries Level AnalysisRegional analysis is a highly comprehensive part of this report. This segmentation sheds light on the sales of the Cancer Gene Therapy on regional- and country-level. This data provides a detailed and accurate country-wise volume analysis and region-wise market size analysis of the global market.

The report offers an in-depth assessment of the growth and other aspects of the market in key countries including the US, Canada, Mexico, Germany, France, the UK, Russia, Italy, China, Japan, South Korea, India, Australia, Brazil, and Saudi Arabia. The competitive landscape chapter of the global market report provides key information about market players such as company overview, total revenue (financials), market potential, global presence, Cancer Gene Therapy sales and revenue generated, market share, prices, production sites and facilities, products offered, and strategies adopted. This study provides Cancer Gene Therapy sales, revenue, and market share for each player covered in this report for a period between 2016 and 2020.

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Table of Contents1. Executive Summary2. Assumptions and Acronyms Used3. Research Methodology4. Market Overview5. Global Market Analysis and Forecast, by Types6. Global Market Analysis and Forecast, by Applications7. Global Market Analysis and Forecast, by Regions8. North America Market Analysis and Forecast9. Latin America Market Analysis and Forecast10. Europe Market Analysis and Forecast11. Asia Pacific Market Analysis and Forecast12. Middle East & Africa Market Analysis and Forecast13. Competition Landscape

About DataIntelo:DATAINTELO has set its benchmark in the market research industry by providing syndicated and customized research report to the clients. The database of the company is updated on a daily basis to prompt the clients with the latest trends and in-depth analysis of the industry. Our pool of database contains various industry verticals that include: IT & Telecom, Food Beverage, Automotive, Healthcare, Chemicals and Energy, Consumer foods, Food and beverages, and many more. Each and every report goes through the proper research methodology, validated from the professionals and analysts to ensure the eminent quality reports.

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Cancer Gene Therapy Market Report Delivering Growth Analysis With Key Trends Of Top Companies (2020-2026) - PRnews Leader

No boy should have a last stretch of days. But Bertrand Might lived his as well as any boy could: There was a Star Trek marathon with his brother and sister, sunrises on the lakeshore, and visits with family in parks, beaches, and backyards anywhere they could safely gather during the pandemic.

His father, Matt Might, said it ended up being an unplanned farewell for 12-year-old Bertrand, whose health had always been precarious. He was the first person in the world diagnosed with a particular neurodegenerative condition that causes developmental delays, seizure-like activity in the brain, and frequent infections.

One of those infections, unrelated to Covid-19, led to his death on Oct. 23 after he spiraled into septic shock. But if his passing came too soon, it did not come before his life led to crucial discoveries for dozens of children with his condition.

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What he did with NGLY1 alone was pretty powerful, said Matt Might, referring to the gene involved in his sons disease. After years of research, it was the discovery of a double mutation in Bertrands NGLY1 gene, and the constellation of symptoms linked to it, that explained the cause of the illness and built a worldwide community around it.

There are 70 families on the patient mailing list right now for a disease that eight years ago didnt exist, Might said.

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Bertrand also inspired a quest by his father, an artificial intelligence expert and computer programmer, to employ precision medicine on a wider scale, using genetic data to help tailor treatments to patients with rare and hard-to-treat diseases like his sons.

Might began that work initially to help Bertrand, but it led to a stint on President Obamas precision medicine initiative and the creation of a new precision medicine institute (PMI) he now leads at the University of Alabama, Birmingham.

PMI was founded on this algorithm that Bertrand taught me, Might said. How do you try to therapeutically modulate a specific genetic target? There is a central game plan we use every time somebody comes in.

Might and his team examine what gene is involved in a persons condition and whether it is under-reactive, over-reactive, toxic, or missing altogether. The answers to those questions form the basis for a scientific process that often gives patients hope when conventional medicine has failed to provide an accurate diagnosis or effective treatments. A permanent endowment has been established at UAB in Bertrands name to fund advanced diagnostics and research to identify novel therapies for patients with no other options.

In Bertrands case, the double mutation in NGLY1 left him without an enzyme that facilitates the recycling of cellular waste. It severely limited his mobility, requiring him to use a wheelchair, and also impaired his liver function and ability to communicate.

Still, Bertrand drove the science of his condition while enduring countless hospitalizations, often due to infections that made it difficult to breathe.

Throughout his life, he developed a love for dolphins and an aquarium his parents set up in his bedroom. He spent hours learning words and reading with his father and mother, Cristina, and he bonded with his younger brother and sister over movies and video games.

Im proud of Bertrand in multiple ways, Might said. I would often tell people to imagine a being created without the ability to even feel malice. He was just a pure being, and I loved that about him.

In recent years, the science that led to his diagnosis has also begun to unravel the biology of NGLY1 deficiency and its impact on patients. A project sponsored by the National Institutes of Health is underway to screen hundreds of thousands of molecules for therapeutic potential against the illness, while Might has used computational methods to identify treatments that showed efficacy in animal subjects.

On Bertrands last day in the hospital, as his condition continued to deteriorate, his father read him an email from the father of another patient with his illness. It said that the Food and Drug Administration seemed pleased with pre-clinical studies of a gene therapy for NGLY1 and outlined a series of steps toward a clinical trial.

It was so meaningful to know the community that Bertrand formed has spawned efforts well beyond my own, Might said. And in the end, he died in a world where the hope of a cure existed.

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The life and death of a boy whose diagnosis brought hope to other patients - STAT

This report studies the Gene Therapy For Heart Failure to get Covid-19 marketplace with Many details of the industry like the market size, market standing, market trends and forecast, the report also provides brief information of their opponents and the specific growth opportunities with key market drivers. Find the complete Gene Therapy For Heart Failure to get Covid-19 market evaluation segmented by firms, region, type and applications in the document.

New sellers in the market are facing tough competition from Established international vendors as they fight with technological innovations, quality and reliability problems. The report will answer questions regarding the current market changes and the reach of competition, opportunity cost and much more.

This Press Release will help you to understand the Volume, growth with COVID19 Impact Analysis. Click HERE To get SAMPLE PDF (Including TOC, Table & Figures) at: @ https://chronicalmarketresearch.com/request-for-sample-report/64529

The report discusses the various types of options for While the regions considered in the scope of the report include North America, Europe, and assorted others. The study also emphasizes on how climbing digital security dangers is changing the market scenario.

Development policies and strategies are discussed along with Manufacturing processes and cost structures are also analyzed. This report also claims import/export consumption, supply and demand Figures, price, cost, earnings and gross earnings.

This report focuses on the international Gene Therapy For Heart Failure for Covid-19 Status, future prediction, growth opportunity, key marketplace and key players.

The following players are covered in this report:

Askbio

RENOVACORINC

Uniqure

DINAQOR

PRECIGEN

Heartseed

Cardior

NOVARTIS

Blue Rock

The Gene Therapy For Heart Failure for Covid-19 market is a comprehensive record Which offers a meticulous overview of the market share, size, trends, demand, product evaluation, application analysis, regional perspective, competitive strategies, forecasts, and strategies impacting the Gene Therapy For Heart Failure for Covid-19 Industry. The report includes a thorough analysis of the market competitive landscape, with the assistance of comprehensive business profiles, SWOT analysis, project feasibility analysis, and several other specifics about the essential companies working on the marketplace.

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The study aims Of this report are:

To examine and forecast the market size of Gene Therapy For Heart Failure for Covid-19 in global sector.

To analyze the International Important players, SWOT analysis, value and International market share for best players.

To specify, describe and forecast the market by type, end use And area.

To analyze and compare the market standing and forecast among Global significant regions.

To analyze the International key regions market potential and Benefit, opportunity and challenge, restraints and risks.

To identify Substantial trends and factors driving or Inhibiting the market development.

To analyze the opportunities in the market for stakeholders

To strategically analyze each submarket with regard to Individual growth tendency and their contribution to the industry

To analyze competitive developments such as expansions,

To profile the key players and comprehensively Analyze their growth plans.

Breakdown Data by Type

Gene Modification

Gene Replacement

Others

Gene Therapy For Heart Failure Breakdown Data by Application

Hospital

Laboratory

Others

Based on regional and country-level analysis, the Gene Therapy For Heart Failure market has been segmented as follows:

North America

United States

Canada

Europe

Germany

France

U.K.

Italy

Russia

Nordic

Rest of Europe

Asia-Pacific

China

Japan

South Korea

Southeast Asia

India

Australia

Rest of Asia-Pacific

Latin America

Mexico

Brazil

Middle East & Africa

Turkey

Saudi Arabia

UAE

Rest of Middle East & Africa

The Gene Therapy For Heart Failure for Covid-19 market research report completely Covers the vital statistics of their capacity, production, value, cost/profit, supply/demand import/export, further divided by company and country, and by application/type for the best possible updated data representation from the figures, tables, pie chart, and graphs. These data representations give predictive data concerning the future estimations for persuasive market growth. The detailed and comprehensive understanding about our publishers makes us out of the box in case of market evaluation.

Key questions Answered in this report

What is going to the market size be in 2026 and what will the Growth rate be?

What will be the key market trends?

Whats driving this economy?

Which will be the challenges to promote growth?

Who will be the key vendors in this market space?

What are the market opportunities and threats faced by the Key vendors?

What are the strengths and weaknesses of the key vendors?

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Table of Contents

Chapter 1: Global Gene Therapy For Heart Failure for Covid-19 Market Overview

Chapter 2: Gene Therapy For Heart Failure for Covid-19 Market Data Analysis

Chapter 3: Gene Therapy For Heart Failure for Covid-19 Technical Data Analysis

Chapter 4: Gene Therapy For Heart Failure for Covid-19 Government Policy and News

Chapter 5: Global Gene Therapy For Heart Failure for Covid-19 Market Manufacturing Process and Cost Structure

Chapter 6: Gene Therapy For Heart Failure for Covid-19 Productions Supply Sales Demand Market Status and Forecast

Chapter 7: Gene Therapy For Heart Failure for Covid-19 Key Manufacturers

Chapter 8: Up and Down Stream Industry Analysis

Chapter 9: Marketing Strategy -Gene Therapy For Heart Failure for Covid-19 Analysis

Chapter 10: Gene Therapy For Heart Failure for Covid-19 Development Trend Analysis

Chapter 11: Global Gene Therapy For Heart Failure for Covid-19 Market New Project Investment Feasibility Analysis

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Originally posted here:
Gene Therapy For Heart Failure Market Report Provide the Development Strategies Adopted by Key Industry Players to Understand Competitive Scenario of...

LEXINGTON, Mass., Nov. 04, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, announced today the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its clinical candidate, LB-001 for the treatment methylmalonic acidemia (MMA). According to the FDA, the purpose of Fast Track designation is to get important new drugs to patients earlier by facilitating the development, and expediting the review, of drugs to treat serious conditions and fill an unmet medical need.

Commenting on the announcement, Daniel Gruskin, M.D., Senior Vice President, Head of Clinical Development of LogicBio, said, We are pleased the FDA has granted Fast Track designation to LB-001 in recognition of the importance of our efforts to bring a durable treatment to the children suffering from MMA. With Fast Track status, we plan to continue to work closely with the FDA to fully utilize the opportunities presented by this designation to make LB-001 available to patients as quickly as possible.

About Fast Track Designation

The FDAs Fast Track designation is a process designed to expedite or facilitate the review of product candidates to treat serious conditions and fill an unmet medical need. Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process. It may also allow for priority or rolling review of a companys Biologics License Application (BLA).

About LogicBio Therapeutics

LogicBio Therapeuticsis dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms. LogicBios proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is expected to begin in early 2021. In addition, LogicBio has a collaboration withTakedato research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.

LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples. Top-tier capsid candidates from this effort demonstrated significant improvements over benchmark AAVs currently in clinical development. LogicBio is developing these highly potent vectors for internal development candidates and potentially for business development collaborations.

LogicBio is headquartered inLexington, Mass. For more information, please visitwww.logicbio.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the federal securities laws, including those related to the timing, progress and results of the Companys research and development activities, including those related to LB-001, and the significance and benefits of receiving the FDAs Fast Track designation for LB-001 in MMA. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. In particular, the impact of the COVID-19 pandemic on the Companys ability to progress with its research, development, manufacturing and regulatory efforts, including the Companys plans to initiate, advance and complete its Phase 1/2 clinical trial for LB-001 in MMA, and the value of and market for the Companys common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements inthe United Statesand in other countries, and the effectiveness of actions taken globally to contain and treat the disease. These risks are discussed in the Companys filings with theU.S. Securities and Exchange Commission(SEC), including, without limitation, the Companys Annual Report on Form 10-K filed onMarch 16, 2020with theSEC, the Companys Quarterly Report on Form 10-Q filed onMay 11, 2020, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with theSEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

Contact:

Matthias JaffeChief Financial OfficerMjaffe@logicbio.com617-245-0399

Continue reading here:
LogicBio Therapeutics Receives FDA Fast Track Designation for LB-001 for the Treatment of Methylmalonic Acidemia (MMA) - GlobeNewswire

LEXINGTON, Mass., Nov. 03, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, today announced the appointment of Mariana Nacht, Ph.D., as chief scientific officer, effective Nov. 30, 2020, and the promotion of Kyle Chiang, Ph.D., to chief operating officer, effective Nov. 2, 2020.

Dr. Nacht brings more than 20 years of experience in both large and small biotech companies to her role at LogicBio. Most recently, she served as CSO and was a founding executive team member of Cereius, where she led a small internal research team and a group of collaborators to develop radiolabeled proteins for the treatment of brain metastases. Before that, she served as CSO of Vivid Biosciences, a functional precision medicine company, where she was also a founding executive team member. Dr. Nacht has also served in key scientific roles at Padlock Therapeutics (acquired by Bristol Myer Squibb in 2014) and Avila Therapeutics, a platform company that developed covalent irreversible inhibitors and was acquired by Celgene in 2012. Earlier in her career, she spent a decade working at Genzyme (now Sanofi Genzyme), where she led anti-angiogenesis and oncology target discovery efforts. Dr. Nacht received her B.S. in biology from Tufts University and her Ph.D. from the University of Pennsylvania.

We are proud to expand our leadership team as we prepare to launch our first clinical trial in pediatric patients with methylmalonic acidemia (MMA) and continue to advance both our GeneRide and Next Generation Capsid platforms, said Fred Chereau, CEO of LogicBio. Mariana brings terrific expertise in novel therapeutic platforms as well as deep experience in building and leading strong scientific teams to her position as CSO. Were thrilled to welcome her to LogicBio as we move into this exciting next phase of progress. Im also delighted to have Kyle promoted to our core leadership team. He has provided essential guidance on pipeline strategy and program development from the early days of LogicBio and he will continue to be an important voice in shaping our future growth.

I am very enthusiastic about the potential for the GeneRide platform to transform care for pediatric patients with rare genetic diseases, Dr. Nacht said. We all enter this field to make a difference for patients, and I am excited to be joining LogicBio just as LB-001, our lead program for children with MMA, is about to enter the clinic with the Phase 1/2 SUNRISE trial. Beyond LB-001, I look forward to further advancing LogicBios pipeline with the goal of bringing more durable and transformational therapies to people living with devastating genetic diseases.

Dr. Chiang was the second employee at LogicBio and has held positions of increasing responsibility since joining the team as director of translational science in 2016. Most recently, he served as vice president, product strategy, where he led LB-001 through early regulatory interactions and managed LogicBios collaboration with the Childrens Medical Research Institute to develop more potent and more easily manufacturable AAV capsids for gene therapy and genome editing applications. Before joining LogicBio, Dr. Chiang led aTyr Pharmas ATYR1940 program from discovery through early clinical development for patients with facioscapulohumeral muscular dystrophy. Dr. Chiang received his B.S. in biochemistry and cell biology from the University of California, San Diego and his Ph.D. in macromolecular cellular structure and chemistry from the Scripps Research Institute.

LogicBio also announced today that Bryan Yoon, Esq., the companys chief administrative officer, general counsel and corporate secretary, will be departing from the company effective Nov. 6, 2020. I want to thank Bryan for his contributions to LogicBio and we wish him the best in his next challenge, Mr. Chereau said.

AboutLogicBioTherapeuticsLogicBio Therapeuticsis dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms. LogicBios proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is expected to begin in early 2021. In addition, LogicBio has a collaboration withTakedato research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.

LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples. Top-tier capsid candidates from this effort demonstrated significant improvements over benchmark AAVs currently in clinical development. LogicBio is developing these highly potent vectors for internal development candidates and potentially for business development collaborations.

LogicBio is headquartered in Lexington, Mass. For more information, please visit http://www.logicbio.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the federal securities laws, including those related to the timing, progress and results of the Companys strategic directives and its research and development activities, including those related to LB-001 and its pipeline. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. In particular, the impact of the COVID-19 pandemic on the Companys ability to progress with its research, development, manufacturing and regulatory efforts, including the Companys plans to initiate, advance and complete its Phase 1/2 clinical trial for LB-001 in MMA, and the value of and market for the Companys common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the United States and in other countries, and the effectiveness of actions taken globally to contain and treat the disease. These risks are discussed in the Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on March 16, 2020 with the SEC, the Companys Quarterly Report on Form 10-Q filed on May 11, 2020, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

Contacts:

Investors:Matthew LaneGilmartin Investor Relationmatt@gilmartinir.com

Media:Stephanie SimonTenBridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

Here is the original post:
LogicBio Therapeutics Announces Appointment of Veteran Biotech Executive Mariana Nacht, Ph.D., as Chief Scientific Officer and Kyle Chiang, Ph.D.,...

WORCESTER, Mass., Nov. 04, 2020 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that interim Phase 1/2 data on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphoma (NHL), have been selected for a poster presentation at the 62nd American Society of Hematology (ASH) Annual Meeting, which is being held virtually from December 5 8, 2020. MB-106 is being developed in a collaboration between Mustang and Fred Hutchinson Cancer Research Center (Fred Hutch).

In the abstract posted today on the ASH website, Fred Hutch reported on four patients treated following a major revision in the cell manufacturing process. Complete remissions were observed in two follicular lymphoma patients (one each at dose levels 1 and 2), as well as a partial remission in a mantle cell lymphoma patient at dose level 2 and progressive disease in a follicular lymphoma patient at dose level 1. Dose level 1 was 3.3 x 105 CAR-T cells/kg and dose level 2 was 1 x 106 CAR-T cells/kg. As previously disclosed, no responses were seen in the 7 patients treated prior to cell process revision. Among the 11 total patients reported in the abstract, there was one occurrence of cytokine release syndrome (grade 3 unexplained alkaline phosphatase elevation in the setting of fever in a patient treated prior to cell process revision) and no occurrences of immune effector cell-associated neurotoxicity syndrome (any grade). No dose-limiting toxicity was observed.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, We are pleased that Fred Hutch will present interim data at ASH from the ongoing Phase 1/2 trial of MB-106, and at that time we expect to disclose data on at least eight total patients treated since the major cell process revision. In February 2020, we reported that the first patient treated in the trial with the revised MB-106 manufacturing process achieved a complete response at the lowest starting dose. The additional data disclosed today further indicate that MB-106 has an extremely favorable safety profile with evidence of promising clinical activity, even at low dose levels. We look forward to continuing progress on this CD20-targeted CAR T cell therapy program for patients with relapsed or refractory B-cell non-Hodgkin lymphomas.

Details of the presentation are as follows:

Title: Third Generation CD20 Targeted CAR T-Cell Therapy (MB-106) for Treatment of Patients with Relapsed/Refractory B-Cell Non-Hodgkin LymphomaSession: 704. Immunotherapies: Poster IAbstract: 1443Date and Time: Saturday, December 5, 2020, 10:00 a.m. - 6:30 p.m. ETPresenter: Mazyar Shadman, M.D., M.P.H., Associate Professor, Clinical Research Division, Fred Hutch, Seattle, WA

For more information, please visit the 62nd ASH Annual Meeting and Exposition website at https://www.hematology.org/meetings/annual-meeting/abstracts.

About B-cell Non-Hodgkin Lymphoma (NHL)There are several forms of NHL, including follicular lymphoma, mantle cell lymphoma, marginal zone lymphoma, lymphoplasmacytic lymphoma and small lymphocytic lymphoma, which account collectively for about 45% of all cases of NHL. Most types of NHL are incurable with available therapies, except for allogenic hematopoietic stem cell transplant (allo-SCT). More than 70,000 new cases of B-cell NHL are diagnosed each year in the United States, and more than 19,000 patients die annually due to this group of diseases.

About MB-106 (CD20-targeted CAR T Cell Therapy)CD20 is a membrane-embedded surface molecule which plays a role in the differentiation of B-cells into plasma cells. The CAR T was developed by Mustangs research partner, Fred Hutchinson Cancer Research Center (Fred Hutch), in the laboratory of Oliver Press, M.D., Ph.D., and Brian Till, M.D., in the Clinical Research Division and exclusively licensed to Mustang Bio in 2017. MB-106 has been optimized as a third-generation CAR derived from a fully human antibody and is currently in a Phase 1/2 open-label, dose-escalation trial at Fred Hutch in B-cell non-Hodgkin lymphoma patients. Additional information on the trial can be found at http://www.clinicaltrials.gov using the identifier NCT03277729.

About Mustang BioMustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission (SEC). Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking Statements

This press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

Here is the original post:
Mustang Bio Announces MB-106 Data Selected for Presentation at the 62nd American Society of Hematology (ASH) Annual Meeting - GlobeNewswire

The Global Viral Vector and Plasmid Manufacturing Market Report, published by Emergen Research, is an all-encompassing study of the Viral Vector and Plasmid Manufacturing market, which emphasizes the estimated market size, share, value, volume, and futuristic outlook. The report details the major products and application rangesavailable in this market, highlighting the revenue, price, sales, production, growth rate, and market share of each segment and sub-segment. The essential market presented in the report in a tabular format is aimed at helping readers interpret the global market dynamics. The market intelligence report delivers a profound study of the Viral Vector and Plasmid Manufacturing business domain, discussing its principal aspects, such as the import & export dynamics, production, consumption, sales channels, and consumer bases in the major regional segments. The global Viral Vector and Plasmid Manufacturing market, which held a significant value of USD 358.4 Million in 2019, is projected to reach a whopping market value of USD 1.12 billion by 2027 at a stunning CAGR of 14.3%.

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Brammer Bio, Cobra Biologics, Cell, and Gene Therapy Catapult, FinVector Vision Therapies, Fujifilm Diosynth Biotechnologies, MassBiologics, SIRION Biotech, Merck KGaA Inc., Thermo Fisher Scientific, and Unique NV,

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Regional Fragmentation:

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Global Viral Vector and Plasmid Manufacturing Market Insights and In-Depth Analysis 2020-2027 | Brammer Bio, Cobra Biologics, Cell, and Gene Therapy...

Hemophilia Gene Therapy Market Overview

The Hemophilia Gene Therapy Market is rapidly undergoing changes. These changes are indicators of market growth. This year-over-year uptrend of the market is pointing towards a steady yet elliptical rise for the next septennial 2020-2027.

The Hemophilia Gene Therapy Market report imparts a detailed insight into the forecast period (2018-2027). The assessment contains different sections that act as the pillars for the market. Factors such as market patterns help businesses in laying out a blueprint of actions to be taken over the course of the specified time frame. The report also brushes over the other components market drivers, limitations, growth opportunities, and hurdles to highlight the effect of these variables over the market. The report also delineates the key markets that can be targeted for starting a business venture. The market drivers and restrictions are intrinsic components while opportunities and hurdles are extrinsic factors of the market. The Hemophilia Gene Therapy Market Report gives a point-of-view of the cyclic development of the market, in the specified time frame, in the offing.

Hemophilia Gene Therapy Market: Competitive Landscape

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The Hemophilia Gene Therapy Market has been examined into different global market segments such as type, applications and global geographies. Each and every global market segment has been studied to get informative insights into various global regions.

Hemophilia Gene Therapy Market Segmentation:

Hemophilia Gene Therapy Market Segment by Type:

Hemophilia Gene Therapy Market Segment by Application:

Hemophilia Gene Therapy Market Segment by Global Presence:

North America Latin America Middle East Asia-Pacific Africa Europe

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Hemophilia Gene Therapy Market: Scope of the Report

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Market Research Intellect provides syndicated and customized research reports to clients from various industries and organizations with the aim of delivering functional expertise. We provide reports for all industries including Energy, Technology, Manufacturing and Construction, Chemicals and Materials, Food and Beverage, and more. These reports deliver an in-depth study of the market with industry analysis, the market value for regions and countries, and trends that are pertinent to the industry.

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Hemophilia Gene Therapy Market Analysis Of Growth, Size, Trends Progress And Challenges Till Upcoming Year 2020 2027 - Eurowire

Beathan Report has published the Global report on The Cancer Gene Therapy market, which is made up of advice about each of the important parameters of this market like consumption and the manufacturing patterns coupled with all the earnings patterns for the forecast period. Concerning creation aspect, the report provides complete detailed analysis about the manufacturing processes combined with the gross financials accumulated by the top most manufacturers working within this business. The primary facet of this Cancer Gene Therapy market thats covered in the report helps the clients and the associations to better understand the business profile concerning drivers, restraints, challenges, and opportunities pertaining and affecting the marketplace dynamics.

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Key market players

Major competitors identified in this market include Adaptimmune, Bluebird bio, Celgene, Shanghai Sunway Biotech, Shenzhen SiBiono GeneTech, SynerGene Therapeutics, Altor BioScience, Amgen, Argenx, BioCancell, GlaxoSmithKline, Merck, OncoGenex Pharmaceuticals, Transgene, etc.

Based on the Region:

Asia-Pacific (China, Japan, South Korea, India and ASEAN)

North America (US and Canada)

Europe (Germany, France, UK and Italy)

Rest of World (Latin America, Middle East & Africa)

According to the Cancer Gene Therapy report, the

COVID-19 has majorly affected the Overall worldwide companies and itll take a enormous time for the company recovery. Vast majority of the business sectors have realigned their company strategies, priorities, and have amended their economic planning in order to remain in the company and keep their standing on the global platform. The thorough evaluation of this Cancer Gene Therapy market will enable the new market entrants to obtain reliable market approaches and strategy strong action plans for the prediction period.

Based on the Type:

Oncolytic Virotherapy

Gene Transfer

Gene-Induced Immunotherapy

Based on the Application:

Hospitals

Diagnostics Centers

Research Institutes

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Major highlights of this Cancer Gene Therapy market report:

* COVID-19 effect on the earnings Streams of the Cancer Gene Therapy market players.

* Statistics of the overall sales quantity And general market revenue.

* Industry trends breakdowns.

* Estimated expansion rate of this Cancer Gene Therapy Market.

* Pros and cons of the direct and

* In-depth information about the important Distributors, traders, and dealers.

Table of Contents : Cancer Gene Therapy Market

Part 01: Executive Summary

Part 02: Scope Of The Report

Part 03: Research Methodology

Part 04: Market Landscape

Part 05: Pipeline Analysis

Part 06: Market Sizing

Part 07: Five Forces Analysis

Part 08: Market Segmentation

Part 09: Customer Landscape

Part 10: Regional Landscape

Part 11: Decision Framework

Part 12: Drivers And Challenges

Part 13: Market Trends

Part 14: Vendor Landscape

Part 15: Vendor Analysis

Part 16: Appendix

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Cancer Gene Therapy Market Business Opportunities, Segmentation Analysis, Top Companies and Forecast to 2028 - Eurowire

The Global Gene Therapies for Cancer Treatment Market report is deep study of the present market dynamics. It consists of the detailed study of current market trends along with the past statistics. The past years are considered as reference to get the predicted data for the forecasted period. Various important factors such as market trends, revenue growth patterns market shares and demand and supply are included in almost all the market research report for every industry. A significant development has been recorded by the market of Gene Therapies for Cancer Treatment, in past few years. It is also for it to grow further. Various important factors such as market trends, revenue growth patterns market shares and demand and supply are included in almost all the market research report for every industry. A systematized methodology is used to make a Report on the Global Gene Therapies for Cancer Treatment market. For the analysis of market on the terms of research strategies, these techniques are helpful. All the information about the Products, manufacturers, vendors, customers and much more is covered in research reports.

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There are different marketing strategies that every marketer looks up to in order to ace the competition in the Global Gene Therapies for Cancer Treatment market. Some of the primary marketing strategies that is needed for every business to be successful are Passion, Focus, Watching the Data, Communicating the value To Your Customers, Your Understanding of Your Target Market. There is a target set in market that every marketing strategy has to reach. The study is done with the help of analysis such as SWOT analysis and PESTEL analysis. SWOT analysis includes the study of Threats, weaknesses, strengths and opportunities that the Gene Therapies for Cancer Treatment market. Whereas PESTEL analysis is the study concerning Economic, Technological, legal political, social, environmental matters. For the analysis of market on the terms of research strategies, these techniques are helpful.

Major companies of this report:

Takara BioTocagenVBL TherapeuticsCold GenesysGenprexMomotaro-GeneMultiVirSynerGene TherapeuticsZiopharm OncologyAnchiano TherapeuticsCelgeneCelsionBluebird Bio

Browse the complete report @ https://www.orbispharmareports.com/global-gene-therapies-for-cancer-treatment-market-2019-present-status-and-future-opportunities-by-major-companies-typs-and-applications-2024/?utm_source=Nilam

Potential consumers, market values, and the future scope for the Gene Therapies for Cancer Treatment market are explained thoroughly to the users in this report. The key players of Gene Therapies for Cancer Treatment industry, their product portfolio, market share, industry profiles is studied in this report. It is very important for the vendors to provide customers with new and improved product/ services in order to gain their loyalty.

Segmentation by Type:

Somatic Cell Gene Therapy (SCGT)Germline Gene Therapy (GGT)

Segmentation by Application:

Cancer Research CentersDiagnostic LaboratoriesCancer HospitalsOthers

The study of various segments of the global Gene Therapies for Cancer Treatment market are also covered in the research report. In addition to that, for the forecast periods determination of factors like market size and the competitive landscape of the market is analyzed in the report. Due to the increasing globalization and digitization, there are new trends coming to the market every day. The research report provides the in-depth analysis of all these trends.

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About Us :

At Orbispharma we curate the most relevant news stories, features, analysis and research reports on the important challenges undertaken by the pharmaceutical and related sectors. Our editorial philosophy is to bring you sharp, focused and informed perspective of industries, the end users and application of all upcoming trends into the pharma sector. Orbispharma believes in conversations that can bring a change in one of the most crucial economic sectors in the world. With these conversations we wish our customers to make sound business decisions with right business intelligence.

Contact Us :

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Gene Therapies for Cancer Treatment Market 2020 Trends Analysis, Top Manufacturers 2024 : Mylan Pharmaceuticals, Pfizer, Roche, Almirall, Bausch...

Cancer Gene Therapy Market Forecast 2020-2026

The Global Cancer Gene Therapy Market research report provides and in-depth analysis on industry- and economy-wide database for business management that could potentially offer development and profitability for players in this market. This is a latest report, covering the current COVID-19 impact on the market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions. The rapidly changing market scenario and initial and future assessment of the impact is covered in the report. It offers critical information pertaining to the current and future growth of the market. It focuses on technologies, volume, and materials in, and in-depth analysis of the market. The study has a section dedicated for profiling key companies in the market along with the market shares they hold.

The report consists of trends that are anticipated to impact the growth of the Cancer Gene Therapy Market during the forecast period between 2020 and 2026. Evaluation of these trends is included in the report, along with their product innovations.

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The Report Covers the Following Companies:Cell GenesysAdvantageneGenVecBioCancellCelgene and Epeius BiotechnologiesIntrogen TherapeuticsZIOPHARM OncologyMultiVirShenzhen SiBiono GeneTech

By Types:Insertion of new genes into the bodyOther

By Applications:HospitalsDiagnostic CentresDrug ManufacturersResearch Institutes

Furthermore, the report includes growth rate of the global market, consumption tables, facts, figures, and statistics of key segments.

By Regions:

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Years Considered to Estimate the Market Size:History Year: 2015-2019Base Year: 2019Estimated Year: 2020Forecast Year: 2020-2026

Important Facts about Cancer Gene Therapy Market Report:

What Our Report Offers:

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About UpMarketResearch:Up Market Research (https://www.upmarketresearch.com) is a leading distributor of market research report with more than 800+ global clients. As a market research company, we take pride in equipping our clients with insights and data that holds the power to truly make a difference to their business. Our mission is singular and well-defined we want to help our clients envisage their business environment so that they are able to make informed, strategic and therefore successful decisions for themselves.

Contact Info UpMarketResearchName Alex MathewsEmail [emailprotected]Organization UpMarketResearchAddress 500 East E Street, Ontario, CA 91764, United States.

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Global Cancer Gene Therapy Market 2019 Trends, Market Share, Industry Size, Opportunities, Analysis and Forecast To 2025 - TechnoWeekly

Jay Bradner, president of the Novartis Institutes for BioMedical Research, pictured above. Photo courtesy of Novartis.

Novartis acquired Cambridge, Massachusetts-based Vedere Bio in a deal totaling up to $280 million. The takeout broadens Novartis footprint in gene and cell therapy, with Vedere Bios gene therapy platforms for ophthalmic disorders.

In April 2018, Novartis bought Illinois-based AveXis for $8.7 billion, largely to acquire the companys gene therapy candidate, AVXS-101, for spinal muscular atrophy, but also for the companys gene therapy platform. The drug was subsequently approved in May 2019 as Zolgensma.

The technology platforms Novartis picks up with Vedere Bio include light-sensing proteins that can be delivered to retinal cells as well as adeno-associated virus (AAV) delivery vectors for intravitreal injection. They believe the technologies can be used widely to treat vision loss caused by photoreceptor death, including a broad range of inherited retinal dystrophies.

Inherited retinal dystrophies (IRDs) are marked by loss of photoreceptor cells and progressive vision loss. More than 2 million people worldwide are affected, often with complete blindness. Existing therapies target only one of more than 250 genes associated with IRDs.

The next frontier in ophthalmology involves finding ways to bring potentially transformative gene therapies to a broader patient population, said Jay Bradner, president of the Novartis Institutes for BioMedical Research. The acquisition of Vedere Bio reflects our commitment to next-generation gene therapy and brings hope to patients with otherwise untreatable forms of vision loss.

Novartis reports it has three specific programs in gene therapyAAVs, chimeric antigen receptor T cells (CAR-Ts) and CRISPR. The Vedere Bio platform directly effects surviving cells in the retina, altering their behavior. It also is promising for other conditions associated with photoreceptor loss, including a dry form of age-related macular degeneration (AMD) known as geographic atrophy. More than 5 million people worldwide are affected by geographic atrophy.

Under the terms of the deal, Novartis is paying Vedere Bio $150 million upfront. Vedere will be eligible for up to $130 million in milestone payments for a total of $280 million.

The company was founded on technology from UC Berkeley researchers Ehud Isacoff and John G. Flanner. The technology arose jointly between UC Berkeley and the School of Veterinary Medicine at the University of Pennsylvania. It was formed in June 2019 in the Atlas Venture incubator, then launched with a Series A financing worth $21 million and began operations at LabCentral in Cambridge, Massachusetts.

Shortly before the acquisition, some of the companys earlier-stage restoration and vision preservation assets were spun out into a new entity, Vedere Bio II, Inc.

Vedere Bios photoreceptor-protein-based optogenetics program has important advantages over competing approaches and brings us one step closer to delivering functional vision to patients in need, said Cyrus Mozayeni, chief executive officer and president of Vedere Bio and Atlas Venture Entrepreneur in Residence. Our proprietary intravitreal capsids enable not only Vedere Bios optogenetics products but also other ocular gene therapies. Our sale to Novartis is an important milestone in advancing Vedere Bios most advance programs to patients around the world. At the same time, I look forward to working with our experienced team to advance our highly innovative, earlier stage assets as part of the newly established Vedere Bio II.

Vedere Bio II will run completely independently from Novartis and Vedere Bio. Its goal is to develop a pipeline of novel vision restoration and vision preservation drugs by focusing on underserved indications. It is supported by the full Vedere Bio investor syndicate, including Atlas Venture, Mission BioCapital and Foundation Fighting Blindness (RD Fund).

Kevin Bitterman, partner at Atlas Venture and chairman of the Vedere Bio board of directors, said, The acquisition of Vedere Bio by Novartis speaks to the strength of the underlying science from our founders and to the incredible job the team has done in advancing these programs over the past year.

Original post:
Novartis Expands Footprint in Gene and Cell Therapy with Vedere Bio Acquisition - BioSpace

According to Market Study Report, Cancer Gene Therapy Market provides a comprehensive analysis of the Cancer Gene Therapy Market segments, including their dynamics, size, growth, regulatory requirements, competitive landscape, and emerging opportunities of global industry.

The Cancer Gene Therapy Market is projected to reach USD XX Billion by 2023 from USD XX Billion in 2018, at a CAGR of XX%. This report spread across 122 Pages, Profiling 10 Companies and Supported with 114 Tables and 24 figures are now available in this research.

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The Cancer Gene Therapy Market report is a compilation of first-hand information, qualitative and competitive assessment industry analysts, inputs from industry experts, and industry participants across the value chain. The research report market provides an in-depth analysis of parent market trends, macro-economic indicators, and governing factors along with market attractiveness as per segments. The report also maps the qualitative impact of various market factors on market segments, trending Key Factors, and geographies.

The following players are covered in this report:

Adaptimmune

Bluebird bio

Celgene

Shanghai Sunway Biotech

Shenzhen SiBiono GeneTech

SynerGene Therapeutics

Altor BioScience

Amgen

Argenx

BioCancell

GlaxoSmithKline

Merck

OncoGenex Pharmaceuticals

Transgene

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Growing popularity of soft adventure sports and increasing focus towards fitness are significant factors driving the growth of the Cancer Gene Therapy market. However, high cost of Cancer Gene Therapy might hinder the growth of the Cancer Gene Therapy market. The demand for Cancer Gene Therapy is high in North America and Europe region and the APAC is expected to grow at high CAGR which is creating lucrative business opportunities for the companies operating in the market.

MARKET SEGMENTATION

Breakdown Data by Type

Oncolytic Virotherapy

Gene Transfer

Gene-Induced Immunotherapy

Cancer Gene Therapy Breakdown Data by Application

Hospitals

Diagnostics Centers

Research Institutes

Based on regional and country-level analysis, the Cancer Gene Therapy market has been segmented as follows:

North America

United States

Canada

Europe

Germany

France

U.K.

Italy

Russia

Nordic

Rest of Europe

Asia-Pacific

China

Japan

South Korea

Southeast Asia

India

Australia

Rest of Asia-Pacific

Latin America

Mexico

Brazil

Middle East & Africa

Turkey

Saudi Arabia

UAE

Rest of Middle East & Africa

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This report provides a thorough analysis of the Cancer Gene Therapy market through detailed research on a variety of topics to help players build strong growth strategies and strong presence in the industry. Readers will also be informed of the important sustainability strategies adopted by leading companies when operating in the Cancer Gene Therapy market. The analyst also thoroughly evaluated the impact of these strategies on market growth and competition.

Chapter Details of Cancer Gene Therapy Market:

Part 01: Executive Summary

Part 02: Scope of The Report

Part 03: Cancer Gene Therapy Market Landscape

Part 04: Cancer Gene Therapy Market Sizing

Part 05: Cancer Gene Therapy Market Segmentation by Type

Part 06: Five Forces Analysis

Part 07: Customer Landscape

Part 08: Geographic Landscape

Part 09: Decision Framework

Part 10: Drivers and Challenges

Part 11: Market Trends

Part 12: Vendor Landscape

Part 13: Vendor Analysis

Reason to access this report:

The report will help the market leaders/new entrants in this market with information on the closest approximations of the revenue numbers for the overall Cancer Gene Therapy market and its sub segments. This report will help the stakeholders to understand the competitive landscape, to gain more insights to better position their businesses, and to plan suitable go-to-market strategies.

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Web: http://www.beathanreports.com

About Us

At Beathan Report, we understand that the research we provide is only as good as the outcome it inspires. These reports are generated by well-renowned publishers on the basis of the data acquired from an extensive research and credible business statistics. Thats why we are proud to provide the widest range of research products, multilingual 24/7 customer support and dedicated custom research services to deliver the insights you need to achieve your goals. Take a look at few of our aspects that makes Beathan Report an asset to your business.

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Cancer Gene Therapy Market Growth and Future Prospects Analyzed - Aerospace Journal

Oct. 27, 2020 06:30 UTC

MONTPELLIER, France & LYON, France--(BUSINESS WIRE)-- Regulatory News:

Sensorion (Paris:ALSEN) (FR0012596468 ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, and Novasep, a leading supplier of services and technologies for the life sciences industry, announce the signature of an agreement for the manufacturing of adeno-associated virus (AAV) vectors. The agreement covers the gene therapy program targeting OTOF (OTOF-GT), the gene encoding the otoferlin protein. Mutations in OTOF lead to severe or profound congenital hearing loss.

Sensorion is currently advancing two preclinical gene therapy programs conducted under a broad multi-program research partnership with the Genetics and Physiology of Hearing Unit at Institut Pasteur (Paris). OTOF-GT, the most advanced program, aims to restore the expression of Otoferlin in the inner hair cells of the ear where it plays an essential role in hearing.

Under the terms of the agreement, Novasep will be in charge of developing and manufacturing (cell culture, AAV expression, purification, aseptic distribution and quality control) the two AAV vectors designed for the Sensorion OTOF-GT project and will supply Drug Product batches to support preclinical and clinical studies.

Securing state-of-the-art research and development capabilities and Good Manufacturing Practice (GMP) capacity, like those offered by Novasep, is a key step and underlines the progress of our Otoferlin gene therapy program, said Nawal Ouzren, CEO of Sensorion. Earlier this year, we announced the first positive preliminary preclinical OTOF-GT data in non-human primates.

We are delighted and proud to contribute to the progress of Sensorions innovative gene therapy programs targeting OTOF. Through this agreement, Novaseps expertise in the field of viral vector development and manufacturing is once again recognized and valued, said Cedric Volanti, Novaseps President of Biopharma Solutions.

About Sensorion

Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders. Its clinical-stage portfolio includes one Phase 2 product: SENS-401 (Arazasetron) for sudden sensorineural hearing loss (SSNHL). Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases enabling it to select the best targets and modalities for drug candidates. The Company is also working on the identification of biomarkers to improve diagnosis of these underserved illnesses. In the second half of 2019, Sensorion launched two preclinical gene therapy programs aimed at correcting hereditary monogenic forms of deafness including Usher Type 1 and deafness caused by a mutation of the gene encoding for Otoferlin. The Company is potentially uniquely placed, through its platforms and pipeline of potential therapeutics, to make a lasting positive impact on hundreds of thousands of people with inner ear related disorders, a significant global unmet medical need.www.sensorion-pharma.com

About Novasep

Novasep provides cost-effective and sustainable manufacturing solutions for the life sciences industries.

For 20 years, Novasep has acquired experience in developing and manufacturing a wide range of viral vectors through its sites in Belgium: AAV, Adenovirus, Lentivirus, HSV, VEEV, VSV, for cell & gene therapy, immunotherapy, vaccination and other therapies from process development to cGMP production.

Novasep also offers Fill & Finish services for viral vectors, attenuated and live viruses, mAbs, plasmids and other biologics, from formulation to packaging.

As part of its growth strategy Rise-2, Novasep recently unveiled a new facility, Senrise-IV, dedicated to the commercial production of viral vectors which has been completed last year by Senefill, a new Fill & Finish commercial facility for aseptic operations. Both facilities located in Seneffe, Belgium, will contribute to the success of biopharmaceuticals projects.www.novasep.com

Label: SENSORION ISIN: FR0012596468 Mnemonic: ALSEN

Disclaimer

This press release contains certain forward-looking statements concerning Sensorion and its business. Such forward looking statements are based on assumptions that Sensorion considers to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, which statements are subject to numerous risks, including the risks set forth in the 2019 Annual Report published on 29 April 2020 and available on our website and to the development of economic conditions, financial markets and the markets in which Sensorion operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Sensorion or not currently considered material by Sensorion. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Sensorion to be materially different from such forward-looking statements.

This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a solicitation of an offer to purchase or subscribe for, Sensorion shares in any country. The communication of this press release in certain countries may constitute a violation of local laws and regulations. Any recipient of this press release must inform him/herself of any such local restrictions and comply therewith.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201026005828/en/

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Sensorion and Novasep Announce Signature of a Gene Therapy Product Development and Manufacturing Agreement - BioSpace

Beathan Report recently released a research report on the Gene Therapy for Age-related Macular Degeneration market analysis, which studies the Gene Therapy for Age-related Macular Degeneration industry coverage, current market competitive status, and market outlook and forecast by 2025.

Gene Therapy for Age-related Macular Degeneration Market 2020-2025 Research Report categorizes the Gene Therapy for Age-related Macular Degeneration market by key players, product type, applications and regions, etc. The report also covers the latest industry data, key players analysis, market share, growth rate, opportunities and trends, investment strategy for your reference in analyzing the Gene Therapy for Age-related Macular Degeneration market.

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According to this latest study, the 2020 growth of Gene Therapy for Age-related Macular Degeneration will have significant change from the previous year. By the most conservative estimates of Gene Therapy for Age-related Macular Degeneration market size (most likely outcome) will be a year-over-year revenue growth rate of XX% in 2020, from US$ xx million in 2019. We give this scenario a XX% probability, where under the scenario the supply chain will start to recover and quarantines and travel bans will ease, over the Q2. Longer-term, the effect of COVID-19 will be felt throughout the year with some degree of harm done by the virus. Over the next five years the Gene Therapy for Age-related Macular Degeneration market will register a XX% CAGR in terms of revenue, the global market size will reach US$ xx million by 2025.

Many companies are operating in the market and conduct their businesses through joint ventures, which benefit the overall market. The Key Players Analysis for the industry is presented in this report.

Key market players

Major competitors identified in this market include RetroSense Therapeutics, REGENXBIO, AGTC, etc.

Based on the Region:

Asia-Pacific (China, Japan, South Korea, India and ASEAN)

North America (US and Canada)

Europe (Germany, France, UK and Italy)

Rest of World (Latin America, Middle East & Africa)

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This study especially analyses the impact of Covid-19 outbreak on the Gene Therapy for Age-related Macular Degeneration , covering the supply chain analysis, impact assessment to the Gene Therapy for Age-related Macular Degeneration market size growth rate in several scenarios, and the measures to be undertaken by Gene Therapy for Age-related Macular Degeneration companies in response to the COVID-19 epidemic.

In addition, this report discusses the key drivers influencing market growth, opportunities, the challenges, and the risks faced by key manufacturers and the market as a whole. It also analyzes key emerging trends and their impact on present and future development.

Based on the Type:

Subretinal

Intravitreal

Based on the Application:

Monotherapy

Combination Therapy

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Beathan Report,

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Web: http://www.beathanreports.com

About Us

At Beathan Report, we understand that the research we provide is only as good as the outcome it inspires. These reports are generated by well-renowned publishers on the basis of the data acquired from an extensive research and credible business statistics. Thats why we are proud to provide the widest range of research products, multilingual 24/7 customer support and dedicated custom research services to deliver the insights you need to achieve your goals. Take a look at few of our aspects that makes Beathan Report an asset to your business.

Read this article:
Gene Therapy for Age-related Macular Degeneration Market Analysis 2020 Growth by Top Companies and Trends by Types, Treatment, Diagnosis and...

Oct. 28, 2020 05:00 UTC

LONDON--(BUSINESS WIRE)-- Gyroscope Therapeutics Limited, a clinical-stage retinal gene therapy company, today announced that the first surgery using its proprietary Orbit Subretinal Delivery System (Orbit SDS) to deliver the companys investigational gene therapy, GT005, to a patient with geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD) has been conducted in the ongoing Phase I/II FOCUS trial. The surgery was conducted in the United States by Jeffrey Heier, M.D., at Ophthalmic Consultants of Boston.

Dry AMD is one of the leading causes of vision loss globally and gene therapies hold tremendous promise for this disease. Delivering gene therapy directly to the site of disease through a subretinal injection is a proven approach; however, the current method requires a vitrectomy. The Orbit SDS is an innovative delivery system that could address some of the challenges surgeons and patients may experience with a vitrectomy. I have been involved with the development of the Orbit SDS for several years, and I am excited to see its introduction into subretinal delivery of what we hope to be an important therapeutic intervention, said Dr. Heier, Co-President, Medical Director, Director of the Vitreoretinal Service, and Director of Retina Research at Ophthalmic Consultants of Boston.

In 2019, Gyroscope merged with Orbit Biomedical to create the only retinal gene therapy company that combines all elements of drug development, a manufacturing platform, and surgical delivery capabilities. Our vision is to develop gene therapy beyond rare disease and we believe our proprietary Orbit SDS has the potential to help us achieve that. We look forward to further evaluating the Orbit SDS in our GT005 clinical programme, said Khurem Farooq, Chief Executive Officer, Gyroscope.

About FOCUS

FOCUS [NCT03846193] is a Phase I/II open-label clinical trial that was initiated in January 2019. The trial is evaluating the safety and dose response of GT005 in people with GA secondary to dry AMD. Three doses of GT005 are being evaluated in FOCUS. In the initial dose-escalation phase of FOCUS, which is now complete, patients were treated using a subretinal injection involving vitrectomy and retinotomy. The trial has been expanded to include additional cohorts, including a dose-expansion cohort and two cohorts in which the Orbit SDS will be used to deliver GT005.

Gyroscope plans to enrol approximately 45 people who have a clinical diagnosis of GA secondary to dry AMD in FOCUS at sites in the United Kingdom and United States.

About Orbit SDS

The Orbit SDS delivers a controlled volume beneath the retina, with the aim of providing precise and consistent dosing. The subretinal space is accessed by advancing a flexible cannula through the suprachoroidal space (the space between the sclera and the choroid). A microneedle inside the cannula is then advanced to deliver a targeted dose beneath the retina. The microinjection procedure is designed to avoid damaging the structure of the eye by eliminating the need for both a vitrectomy (a procedure that involves removing the vitreous the gel-like substance that fills the eye) and a retinotomy (a hole in the retina).

The Orbit SDS is 510(k) cleared for microinjection into the subretinal space at the back of the eye using saline solution (BSS or BSS PLUS*).

About Dry AMD

Dry AMD is a leading cause of permanent vision loss in people over the age of 50, and is a devastating diagnosis. This gradual and permanent loss of central vision can severely impact a persons daily life as they lose the ability to drive, read and even see the faces of loved ones. There are currently no approved treatments for dry AMD.

Gyroscope estimates that nearly 3.5 million people in the United States and EU5 European countries have GA, and that more than 100,000 people with GA have certain Complement Factor I (CFI) mutations that correlate with low CFI levels in the blood and a higher risk of developing AMD.

About Gyroscope: Vision for Life

Gyroscope Therapeutics is a clinical-stage retinal gene therapy company developing gene therapy beyond rare disease to treat a leading cause of blindness, dry AMD. Our lead investigational gene therapy, GT005, is a one-time therapy delivered under the retina. GT005 is designed to restore balance to an overactive complement system by increasing production of the Complement Factor I protein. GT005 is currently being evaluated in a Phase I/II clinical trial called FOCUS and a Phase II clinical trial called EXPLORE.

Syncona Ltd, our lead investor, helped us create the only retinal gene therapy company to combine discovery, research, drug development, a manufacturing platform and surgical delivery capabilities. Headquartered in London with locations in Philadelphia and San Francisco, our mission is to preserve sight and fight the devastating impact of blindness. For more information visit: http://www.gyroscopetx.com and follow us on Twitter (@GyroscopeTx) and on LinkedIn.

*Balanced Salt Solution; BSS PLUS is a registered trademark of Alcon Laboratories, Inc.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201027006180/en/

The rest is here:
Gyroscope Therapeutics Announces First Patient Received Investigational Gene Therapy GT005 Via Orbit Subretinal Delivery System in Ongoing Phase I/II...

Global Cell Banking Outsourcing Market: Overview

The global cell banking outsourcing market is likely to be driven by the rising demand for biopharmaceutical production targeting novel active sites, stem cell therapy, and gene therapy. A cell bank is a facility storing cells extracted from various organ tissue and body fluids so as to cater to the needs of the future. The cell banks make storage of cells with an elaborate characterization of the entire cell line as it reduces the possibilities of cross contamination. These benefits are estimated to fuel expansion of the global cell banking outsourcing market over the timeframe of assessment, from 2020 to 2030.

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Cell banking outsourcing industries engage testing, characterization, storage, and collection of tissues, cell lines, and the cells. These activities are done to assist in the production of biopharmaceuticals and in the research and development activities so as to ensure minimum adverse effects and high effectiveness. The procedure of the cell storage involves first proliferation of cells, which then multiplies in a huge number of identical cells and is then put inside cryovials safety for use in future. Cells are primarily utilized in the production of regenerative medicine. A surge in the number of cell banks together with the high demand for stem cell therapies is likely to work in favor of the global cell banking outsourcing market over the tenure of analysis, from 2020 to2030.

The global cell banking outsourcing market has been segmented on the basis of four important parameters, which are bank type, phase, cell type, and region.

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Global Cell Banking Outsourcing Market: Notable Developments

The global cell banking outsourcing market is considered a fairly competitive market and is marked with the presence of many leading market players. The companies in this market are forging mergers, partnerships, and collaborations so as to gain larger revenue and market share. The following development is expected to play an important role in the market:

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Global Cell Banking Outsourcing Market: Key Trends

The global cell banking outsourcing market is characterized by the presence of the following restraints, drivers, and opportunities.

High Demand for Stem Cell Therapies to Trigger Growth of the Market

The rising number of stem cell therapies across the globe primarily influences the global cell banking outsourcing market. According to a survey conducted by World Network for Blood and Marrow Transplantation (WBMN), nearly 1 million hematopoietic stem cell transplantation processes were conducted in between 2006 to 2014. These figure comprised removal of stem cells procedures from peripheral blood or bone marrow, proliferating, and then finally storing them cell banks for future use by patients. Stem cell therapies are able to multiple disease, such as amyotrophic lateral sclerosis, type 1 diabetes, cancer, Alzheimer's disease, Parkinson's disease, and so on. Ability to cure such a wide variety of diseases is expected to propel growth of the global cell banking outsourcing market in the years to come.

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Global Cell Banking Outsourcing Market: Geographical Analysis

North America is expected to dominate the global cell banking outsourcing market throughout the timeframe of analysis, from 2020 to 2030. Such high growth of the North America market is ascribed to the increased production of antibiotics, therapeutics protein, and vaccines. In addition, presence of several biopharmaceutical companies in the region is anticipated to foster growth of the cell banking outsourcing market in North America in the near future.

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Cell Banking Outsourcing Market: High Demand for Stem Cell Therapies to Trigger Growth of the Market - BioSpace

NEW YORK--(BUSINESS WIRE)--Nov 2, 2020--

IVERIC bio, Inc. (Nasdaq: ISEE) today announced financial and operating results for the fiscal quarter ended September 30, 2020 and provided a general business update.

It has been a landmark year as we have achieved several major milestones for Zimura, stated Glenn P. Sblendorio, Chief Executive Officer and President of IVERIC bio. Over the past year, we announced positive 12-month and 18-month results from our GATHER1 Phase 3 clinical trial of Zimura for the treatment of geographic atrophy secondary to age-related macular degeneration. We initiated patient enrollment in GATHER2, our second Phase 3 clinical trial for the treatment of GA secondary to AMD. Additionally, we strengthened our balance sheet, advanced our two lead gene therapy product candidates to be on track to enter into the clinic next year and identified a lead compound for our HtrA1 inhibitor program.

Pravin U. Dugel, M.D., Chief Strategy and Business Officer of IVERIC bio added, We are excited about the momentum generated with Zimura as a potential treatment for GA and, based on scientific data, Zimuras potential impact in wet (neovascular) AMD and in earlier stages of dry AMD. We are also encouraged by our HtrA1 inhibitor, IC-500, which we believe could be another important target in the treatment of AMD. Looking forward, our goal is to expand our footprint in multiple stages and types of AMD, dry and wet. We intend to do this by potentially studying Zimura in additional indications and by advancing the development of IC-500. We expect to make great strides in 2021, as we continue to move our pipeline of therapeutics and gene therapy product candidates forward.

Therapeutics Programs Targeting Age-Related Macular Degeneration

The GATHER1 data were recently presented at the following virtual conferences:

IC-500: HtrA1 (high temperature requirement A serine peptidase 1 protein) Inhibitor Program

Gene Therapy Programs in Orphan Inherited Retinal Diseases (IRDs)

Corporate Update

In July 2020, Mark S. Blumenkranz, M.D., M.M.S., joined the Companys board of directors. Dr. Blumenkranz is a biotechnology industry leader and internationally known Vitreo-Retinal Specialist with notable expertise in pharmaceuticals for age-related macular degeneration and ocular gene therapy.

Third Quarter 2020 Operational Update and Cash Guidance

As of September 30, 2020, the Company had $231.1 million in cash, cash equivalents and available for sale securities. The Company has revised its estimated year-end 2020 cash, cash equivalents and available for sale securities to range between $210 million and $215 million, reflecting the impact of a fourth quarter $6.0 million milestone payment to Archemix Corp. The Company also estimates that its cash, cash equivalents and available for sale securities will be sufficient to fund its planned capital expenditure requirements and operating expenses, excluding any potential approval or sales milestones payable to Archemix Corp. or any commercialization expenses for Zimura, through at least mid-2024. These estimates are based on the Companys current business plan, including the continuation of its ongoing clinical development programs for Zimura, the progression of its IC-100 and IC-200 programs into the clinic, and the advancement of its IC-500 development program. These estimates also assume that the Company will enroll approximately 400 patients in the GATHER2 trial. These estimates do not reflect any additional expenditures related to potentially studying Zimura in other indications or resulting from the potential in-licensing or acquisition of additional product candidates or technologies or commencement of new sponsored research programs, and any associated development the Company may pursue.

2020 Q3 Financial Highlights

Conference Call/Web Cast Information

IVERIC bio will host a conference call/webcast to discuss the Companys financial and operating results and provide a business update. The call is scheduled for November 2, 2020 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 866-575-6539 (USA) or 323-794-2575 (International), passcode 6339331. A live, listen-only audio webcast of the conference call can be accessed on the Investors section of the IVERIC bio website at http://www.ivericbio.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 888-203-1112 (USA), passcode 6339331.

About IVERIC bio

IVERIC bio is a science-driven biopharmaceutical company focused on the discovery and development of novel treatment options for retinal diseases with significant unmet medical needs. The Company is currently developing both therapeutic product candidates for age-related retinal diseases and gene therapy product candidates for orphan inherited retinal diseases. Vision is Our Mission. For more information on the Company, please visit http://www.ivericbio.com.

Website Disclaimer

We have included website addresses in this press release solely as inactive references. The information contained on, or that can be accessed through, such websites is not a part of this press release.

Forward-looking Statements

Any statements in this press release about the Companys future expectations, plans and prospects constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Forward-looking statements include any statements about the Companys strategy, future operations and future expectations and plans and prospects for the Company, and any other statements containing the words anticipate, believe, estimate, expect, intend, goal, may, might, plan, predict, project, seek, target, potential, will, would, could, should, continue, and similar expressions. In this press release, the Companys forward looking statements include statements about its expectations regarding patient enrollment and patient retention in its second Phase 3 trial (GATHER2) of Zimura in geographic atrophy secondary to AMD and to use the results of its completed clinical trial of Zimura for the treatment of geographic atrophy secondary to AMD (GATHER1) as a Phase 3 trial, its development and regulatory strategy for Zimura and its other product candidates, including additional indications that the Company may pursue for the development of Zimura, the implementation of its business plan, its expectations regarding expected cash, cash equivalents and available for sale securities and the sufficiency of its cash resources, the timing, progress and results of clinical trials and other research and development activities and regulatory submissions, the potential utility of its product candidates, and the potential for its business development strategy. Such forward-looking statements involve substantial risks and uncertainties that could cause the Companys development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, those related to the progression and duration of the COVID-19 pandemic and responsive measures thereto and related effects on the Companys research and development programs, operations and financial position, the initiation and the progress of research and development programs and clinical trials, availability of data from these programs, reliance on contract development and manufacturing organizations, university collaborators and other third parties, establishment of manufacturing capabilities, expectations for regulatory matters, need for additional financing and negotiation and consummation of business development transactions and other factors discussed in the Risk Factors section contained in the quarterly and annual reports that the Company files with the Securities and Exchange Commission. Any forward-looking statements represent the Companys views only as of the date of this press release. The Company anticipates that subsequent events and developments will cause its views to change. While the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so except as required by law.

Three Months Ended September 30,

Nine Months Ended September 30,

2020

2019

2020

2019

$

18,841

$

10,383

$

45,311

$

28,077

6,643

4,674

17,930

15,353

25,484

15,057

63,241

43,430

(25,484

)

(15,057

)

(63,241

)

(43,430

)

33

495

437

1,782

Here is the original post:
IVERIC bio Reports Third Quarter 2020 Operational Highlights and Financial Results - The Baytown Sun

Newswise Mass Eye and Ear is proud to announce the successful conclusion of its historic campaign, Bold Science. Life-Changing Cures. which raised $252M from philanthropy to advance research to treat and cure diseases of vision, hearing, and the head and neck.

The campaign was led by co-chair Wyc Grousbeck, Boston Celtics CEO and Lead Owner and former Chairman of Mass Eye and Ear. Grousbeck was elected Chairman of the Foundation and Board of Directors of the Massachusetts Eye and Ear in 2010, and stepped down from the position earlier this year. He will continue to serve as a trustee and contribute to advancing Mass Eye and Ears mission, with a lasting legacy that includes two endowed chairs and a gene therapy research center he and his family established.

The campaigns finale culminated in a virtual celebration as part of the hospitals annual Sense-ation! Gala on Wednesday, October 28, 2020 at 7:30 PM EST. This years gala was held online due to the COVID-19 pandemic, and was hosted by longtime Mass Eye and Ear friends, Billy Costa, (Co-Host of KISS 108s Matty in the Morning and NESNs Dining Playbook) and founding gala chair Joey McIntrye (New Kids on the Block).

[Watch the entire 11th Annual Sense-ation Gala!]

Bold Science. Life-Changing Cures. Ends with Enduring Legacy

The Bold Science. Life-Changing Cures. campaign was launched with the goal of expediting cures for blindness, deafness and diseases of the head and neck by investing in three key areas: people, programs and places (our facilities). Through this campaign, the generosity of donors and organizations infused significant financial resources into Mass Eye and Ears cutting-edge research programs, and supported the facilities needed by the leading physicians and scientists in Ophthalmology and Otolaryngology-Head and Neck Surgery to conduct their world-class work.

The campaign resulted in a powerful and far-reaching impact on research at Mass Eye and Ear by faculty, including:

Additionally, new Research Centers were launched in gene therapy, tinnitus, and head and neck cancer and two world-class surgical training laboratories were built.

The campaign also will leave a lasting legacy of fourteen endowed Chairs for leading physicians and scientists that will remain for generations. The endowment of a Chair provides financial support for researchers, allowing them to focus on their efforts to develop treatments and cures.

Wyc Grousbeck Honored for Dedication to Mass Eye and Ear During 10 years as Chairman

Grousbeck, who served as Board Chairman at Mass Eye and Ear for the past decade, was honored at this years Sense-ation! Gala for his many contributions. During his tenure, he led the organization through remarkable growth, both clinically and as a world-renowned research center. Mass Eye and Ear surgical volume doubled and the number of clinical locations grew from 9 to 21 throughout the region. Grousbecks leadership played critical roles in two highly successful mergers: Schepens Eye Research Institute joining Mass Eye and Ear in 2012 and Mass Eye and Ear joining Mass General Brigham (formerly Partners HealthCare) in 2018. He also initiated and served as co-chair of the Bold Science. Life-Changing Cures. campaign.

Wycs leadership during the campaign has been nothing short of magnificent; he has showed us anything is possible, said John Fernandez, President of Mass Eye and Ear. When we first reached our $100M goal in the campaign, Wyc called on us to double that amount by 2020. With Wycs guidance, we were able to exceed that ambitious goal by more than $50M by the campaigns end.

Grousbeck first became involved with Mass Eye and Ear because his family has been touched by blindness, and since then, they have been devoted to research and educational initiatives relating to blindness and other vision-related conditions for many years.

Grousbeck and his familys philanthropic support established the Grousbeck Center for Gene Therapy at Mass Eye and Ear as well as the Grousbeck Family Chair in Gene Therapy, held by Luk H. Vandenberghe, PhD. Additionally, Wyc and his wife, Emilia Fazzalari, funded the Fazzalari-Grousbeck Chair in Otolaryngology-Head and Neck Surgery held by Tessa Hadlock, MD. Most recently, they seeded Dr. Vandenberghes work to develop an experimental and novel genetic vaccine for COVID-19.

Grousbeck was a driving force around establishing the first Sense-ation Gala 11 years ago, which brought celebrities, musicians, philanthropists, and scientists together to help raise funds for Mass Eye and Ear research and patient care. The gala to date has raised more than $12M for the Mass Eye and Ear Curing Kids Fund.

About Mass Eye and Ear

Massachusetts Eye and Ear, founded in 1824, is an international center for treatment and research and a teaching hospital of Harvard Medical School. A member ofMass General Brigham, Mass Eye and Ear specializes in ophthalmology (eye care) and otolaryngologyhead and neck surgery (ear, nose and throat care). Mass Eye and Ear clinicians provide care ranging from the routine to the very complex. Also home to the world's largest community of hearing and vision researchers, Mass Eye and Ear scientists are driven by a mission to discover the basic biology underlying conditions affecting the eyes, ears, nose, throat, head and neck and to develop new treatments and cures. In the 20202021 Best Hospitals Survey,U.S. News & World Reportranked Mass Eye and Ear #4 in the nation for eye care and #6 for ear, nose and throat care.For more information about life-changing care and research at Mass Eye and Ear, visit our blog,Focus, and follow us onInstagram,TwitterandFacebook.

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Mass Eye and Ear Capital Campaign Concludes with $252 Million Raised to Advance Research and Patient Care - Newswise