Archive for the ‘Gene Therapy Research’ Category

Data Bridge Market Research has recently added a concise research on the Clinical Laboratory Services Market to depict valuable insights related to significant market trends driving the industry. The report features analysis based on key opportunities and challenges confronted by market leaders while highlighting their competitive setting and corporate strategies for the estimated timeline. The development plans, market risks, opportunities and development threats are explained in detail. The CAGR value, technological development, new product launches and Industry competitive structure is elaborated.

Clinical laboratory services marketis expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses that the market is growing with a CAGR of 6.2% in the forecast period of 2020 to 2027 and expected to reach USD 217,941.33 million by 2027 from USD 134,692.52 million in 2019. Rising demand for early and accurate disease diagnosis is the factors for the market growth.

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Clinical laboratory services is playing significant role to enable doctors to make appropriate clinical and diagnostic decisions across various levels of health care services. Increasing demand for diagnostic tests is augmenting the market growth as they are used for clinical diagnoses tests.

The major players covered in the report are:-

Mayo Foundation for Medical Education and Research (MFMER), Laboratory Corporation of America Holding, Quest Diagnostics Incorporated, Spectra Laboratories (A Subsidiary of Fresenius Medical Care AG & Co. KGaA), DaVita Inc., Eurofins Scientific, UNILABS, SYNLAB International GmbH, MIRACA HOLDINGS Inc., Sonic Healthcare, ACM Global Laboratories, amedes Group, LifeLabs, Alere (A Subsidiary of Abbott), Charles River, Siemens Healthineers AG, BioReference Laboratories, Inc., NeoGenomics Laboratories, Inc., KingMed Diagnostics, Genomic Health, among other players domestic and global. Clinical laboratory services market share data is available for Global, North America, Europe, Asia-Pacific, Middle East and Africa and South America separately. DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.

Many business expansion and developments are also initiated by the companies worldwide which are also accelerating the growth of global clinical laboratory services market.

For instance,

Partnership, joint ventures and other strategies enhances the company market share with increased coverage and presence. It also provides the benefit for organisation to improve their offering for Clinical Laboratory Services through expanded model range.

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Global Clinical Laboratory Services Market By Specialty (Clinical Chemistry Testing, Hematology Testing, Microbiology Testing, Immunology Testing, Drugs of Abuse Testing, Cytology Testing, Genetic Testing), Provider (Hospital-Based Laboratories, Independent & Reference Laboratories, Nursing and Physician Office-Based Laboratories), Application (Bioanalytical & Lab Chemistry Services, Drug Development Related Services, Drug Discovery Related Services, Toxicology Testing Services, Cell & Gene Therapy Related Services, Preclinical & Clinical Trial Related Services, Other Clinical Laboratory Services), Geography (North America, Europe, Asia-Pacific, South America, Middle East and Africa) Market Trends and Forecast to 2027

Rising demand of clinical laboratory services across the world is one of the prominent factors for increasing demand of clinical chemistry testing. For instance, yearly in the U.K., the usually citizen has 14 tests completed by a laboratory specialist. However in the U.S., laboratory testing is the only highest-volume medical activity and approximately 13,000 million tests are performed per year. Hence, this factor has led the patients to shift towards clinical chemistry testing because it offers a wide range of tests which are performed in the clinical laboratory testing. In the current scenario the technological advancements boost the patients reach to laboratory testing, which is becoming gradually more precise and affordable. For instance, point-of-care testing (POCT) is regularly applied in hospitals and other healthcare practices, so the patients not need to travel to the clinical laboratories for testing. The increasing use of point-of-care testing has reduced demand tests which are conventionally executed at industry laboratories.

Rising demand of early and accurate disease diagnosis across the globe is one of the prominent factors for an upsurge demand of clinical laboratory services. For instance, in 2018, World Health Organization projected that projected that an estimated 1.8 million new cases and almost 861,000 deaths occur due to colorectal cancer (CRC). This factor has increased the number of clinical laboratories around the globe as Delays in accessing cancer care are very common in the late-stage presentation, especially in lower vulnerable populations and resource settings.

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Table of Contents-Snapshot Executive SummaryChapter 1 Industry OverviewChapter 2 Industry Competition by ManufacturersChapter 3 Industry Production Market Share by RegionsChapter 4 Industry Consumption by RegionsChapter 5 Industry Production, Revenue, Price Trend by TypeChapter 6 Industry Analysis by ApplicationsChapter 7 Company Profiles and Key Figures in Industry BusinessChapter 8 Industry Manufacturing Cost AnalysisChapter 9 Marketing Channel, Distributors and CustomersChapter 10 Market DynamicsChapter 11 Industry ForecastChapter 12 Research Findings and ConclusionChapter 13 Methodology and Data Source

Country Level Analysis, By Provider

North America dominates the clinical laboratory services market as the U.S. is leader in clinical laboratory services. In North America due to better advancement in products and services, this region is dominating the clinical laboratory services. North America accounts higher healthcare expenditure, especially in U.S. Asia-Pacific is growing with the highest CAGR due to increase in medical tourism as well as increase in population. Numbers of companies in emerging countries are increasing due to increase in demand for disease diagnosis in clinics, hospitals and other areas. Additionally, the increasing number of healthcare expenditure and increasing number of hospitals and clinical diagnostic laboratories in China and India upsurge demand of clinical laboratory services. The Asia-Pacific region is expected to grow with the highest growth rate in the forecast period of 2020 to 2027 because of increasing infectious diseases.

The country section of the report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as new sales, replacement sales, country demographics, regulatory acts and import-export tariffs are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of sales channels are considered while providing forecast analysis of the country data.

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Huge Investment by laboratory for Clinical Laboratory Services and New Technology Penetration

Global clinical laboratory services market also provides you with detailed market analysis for every country growth in life science industry with clinical laboratory services demand impact of technological development in laboratory services and changes in regulatory scenarios with their support for the clinical laboratory services market. The data is available for historic period 2010 to 2018.

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Clinical Laboratory Services Market Data Highlighting Major Vendors, Promising Regions, Anticipated Growth Forecast To 2026 - 3rd Watch News

The Global Biopreservation market report presents market dynamics focusing on all the important factors market movements depend on. It includes current market trends with a record from historic year and prediction of the forecast period. This report is a comprehensive market analysis of the Biopreservation market done on a basis of regional and global level. Important market analysis aspects covered in this report are market trends, revenue growth patterns market shares and demand and supply along with business distribution

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Bio-banking is further divided into veterinary IVF, human sperm and human eggs whereas regenerative medicine is sub-divided into gene therapy, cell therapy and more. On the basis of product outlook, market is divided into laboratory information management system, media and equipment.

Biopreservation is the increase in the shelf life and developed safety of foods, using the controlled or natural antimicrobial or microbiota compounds. It is the ecological and harmful method to deal with food preservation problem and is getting the attention in the current years. Growth in the demand for the developed methods of preservation and is continuously increasing the spending on healthcare is expected to boost the biopreservation market.

Pharmaceutical companies, biotechnology firms and hospitals are gradually accepting the systems for the in-house storage of the samples and is projected to offer a substantial growth to the global biopreservation market. Increasing the investments in the R&D activities by the research communities and companies are motivated to develop the capabilities of the product is increasing the biopreservation market with large potential.

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Developments in the technology like virtual bio-banks are expected to experience a substantial increase in the application, results in the biopreservation market growth. Moreover, rise in the preference of maintaining the repositories of biological specimen in the regenerative medicine and organ transplantation has promoted the biopreservation industry. With the help of initiatives by government like International Society of Biological and Environmental Repositories to augment the standards of preservation, practices and bio-banking is increasing the possibility for the growth of biopreservation market during the forecast period.

Rise in the expenditure for healthcare is expected to develop the healthcare quality and developed products involve the facilities of biopreservation, by widening the growth of biopreservation market. Moreover, gene banks, bio-banks and hospitals are the substantial consumers for biopreservation industry are influencing the manufacturers to produce the technologically developed products of biopreservation to increase the outcomes of patient and enhance the biopreservation market in the coming future.

With the help of several initiatives by government in healthcare are providing the grants, contracts and funds for research results in offering the growth to R&D activities set by several companies. This results in increasing the opportunity for implementing the developed services of bio-preservation, is projected to boost the growth of biopreservation market over the forecast period. Growth in the investments of R&D by research communities and companies to expand their effective products, services and capabilities is anticipated to increase the growth of biopreservation market. For example, formation of Cooperative Human Tissue Network are addressing the measurement of bio-banking, illustrates the continuous efforts by the community of research.

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Geographically, regions involved in the development of biopreservation market size are Europe, North America, Latin America, Asia Pacific and Middle East & Africa. U.S biopreservation market is dominating the North America and is propelled to increase the biopreservation industry in the coming years. Asia Pacific is estimated to hold the largest biopreservation market share over the forecast period.

Key players involved in the global biopreservation market are LabVantage Solutions, Thermo-Fischer Scientific, Custom Biogenic Systems, Biomatrica, Qiagen and more.

Key Segments in the Global Biopreservation Market are-

By Cell Provider Outlook, market is segmented into:

By Application, market is segmented into:

By Product Outlook, market is segmented into:

By Regions market is segmented into:

What to expect from the Global Biopreservation Market report?

Predictions of future made for this market during the forecast period.

Information on the current technologies, trends, devices, procedures, and products in the industry.

Detailed analysis of the market segmentation, depending on the types, devices, and products.

Government regulations and economic factors affecting the growth of the market.

An insight into the leading manufacturers.

Regional demographics of the market.

Who should buy this report?

Venture capitalists, Investors, financial institutions, Analysts, Government organizations, regulatory authorities, policymakers ,researchers, strategy managers, and academic institutions looking for insights into the market to determine future strategies

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Biopreservation Market Analysis 2019-2025 Emerging Trends, Growth Drivers, Challengers, Services, Competitive Landscape and Regional Outlook &...

Japanese pharma and biotech friend Takeda is set to spend nearly $1 billion in backloaded payments for a deal with early-stage startup Carmine Therapeutics.

The pair will target rare diseases using gene therapies out of the biotechs red blood cell extracellular vesicles (RBCEVs) platform, which it calls Regent and was first published in Nature back in 2018 (with the startup officially launching a year later).

An initial focus is non-viral gene therapies rather than the traditional path of adeno-associated virus (AAV)-based gene therapies that can lead to unwanted side effects.

De-risking the Development of Biotherapeutics Using Early Stage In Vitro Expression and Genetic Characterisation Tools

There is a high attrition rate during the development of biotherapeutics impacting the high cost of development. Early identification of the preferred expression host for manufacturing, along with lead candidate screening and material supply can help to reduce both attrition rates and cost.

The biotech said its RBCEV-based gene therapy has the potential for repeat dosing, a significantly larger transgene payload capacity in excess of 11KB, and enhanced bio-distribution in selected tissues through RBCEV surface modification.

The deal could be worth as much as $900 million, with Takeda leading Carmine's convertible note seed plus funding round by extending a $5 million convertible loan to the biotech. Theres an upfront payment and extra research funding cash, though details of dollar amounts here were not given.

Takeda nabs an option to license the programs following the completion of preclinical proof of concept studies; from here, it would be responsible for clinical development and sales.

Carmine Therapeutics was founded last year by Esco Ventures X, a life science fund, professor Harvey Lodish of the Whitehead Institute at MIT and Singaporean professors Minh Le and Jiahai Shi.

Lodish was a founding member of several big biotechs, including Genzyme (which was snapped up by Sanofi).

XQ Lin, founding CEO of Carmine Therapeutics and managing partner of Esco Ventures, said: We are pleased to enter this collaboration with Takeda, a recognized global leader in rare disease therapies, slightly more than a year since Carmine was created and incubated by Esco Ventures X. This provides Carmine with significant funding to further develop our REGENT(TM) platform, and advance our wholly-owned programs.

This also marks another, albeit very early venture into gene therapies for Takeda. In April, German CRO-biotech hybrid Evotec allied with Takeda to expand into gene therapy research. The move saw Evotec establish a 20-person team in Austria, the focal point of Takedas gene therapy operation, and sign up to work on programs for its Japanese partner.

Takeda acquired a gene therapy center in Orth an der Donau, Austria, through its purchase of Shire, which picked up the site two years earlier in its takeover of Baxalta. Throughout the series of changes in ownership, which began when Baxter spun out Baxalta in 2014, a team at the site has worked on gene therapies.

At Takeda, we're expanding our foundation in gene therapy by establishing a network of innovative partners like Carmine Therapeutics who are pursuing non-viral vector approaches that could leapfrog today's technology, added Takeda's rare disease drug discovery unit head Madhu Natarajan.

Developing alternative gene therapy delivery vehicles like the REGENT platform that could address the challenges of AAV gene therapy is critical to one day delivering next-generation cures for rare diseases.

Excerpt from:
Takeda pens $900M biobucks pact with Carmine for non-viral gene therapy work - FierceBiotech

PARIS--(BUSINESS WIRE)-- Regulatory News:

Lysogene (Paris:LYS)(FR0013233475 LYS), a Phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced that the company has entered into a collaborative research agreement with Yeda Research and Development Co Ltd, the commercial arm of the Weizmann Institute of Science.

The agreement involves collaboration between Lysogene and the lab of Prof. Anthony Futerman at the Weizmann Institute of Science, with the aim of developing a novel AAV gene therapy approach for neuronopathic Gaucher disease, Parkinson disease, and other diseases associated with mutations of the GBA1 gene. Under the terms of the agreement, Lysogene will provide expertise in AAV vector design and production, while the lab of Prof. Futerman will provide glucocerebrosidase variants with enhanced biological properties and conduct biological proof and concept studies. Lysogene has an exclusive option to license the program.

Ralph Laufer, Chief Scientific Officer at Lysogene said: We are thrilled to start this research collaboration with Prof. Futerman, a leading expert in the field of sphingolipid biology. The Weizmann Institute is one of the world's leading multidisciplinary research institutes, and the source of numerous groundbreaking medical discoveries and technological applications. Lysogene is developing gene therapy approaches for monogenic neurological disorders, including neuronopathic lysosomal storage diseases. This collaboration is a perfect fit with Lysogenes strategy, providing the opportunity to develop a novel therapy for a rare lysosomal disease, with the potential to expand into neurological diseases with much larger patient populations, such as Parkinson.

About Lysogene

Lysogene is a gene therapy company focused on the treatment of orphan diseases of the central nervous system (CNS). The company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing and a phase 1/3 clinical trial in GM1 gangliosidosis is in preparation. In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. will hold exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene will maintain commercial exclusivity of LYS-SAF302 in Europe. Lysogene is also collaborating with an academic partner on a gene therapy approach for the treatment of Fragile X syndrome, a genetic disease related to autism. http://www.lysogene.com.

Forward Looking Statement

This press release may contain certain forward-looking statements, forecasts and estimates with respect to Lysogenes clinical trials, clinical trial data releases, clinical development plans, anticipated future activities and cash runway of Lysogene. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice, (ii) factors beyond the Companys control, (iii) clinical trial results, (iv) increased manufacturing costs and (v) potential claims on its products. These statements may include, without limitation, any statements preceded by, followed by or including words such as target, believe, expect, aim, intend, may, anticipate, estimate, plan, objective, project, will, can have, likely, should, would, could and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Companys control that could cause the Companys actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. A further list and description of these risks, uncertainties and other risks can be found in the Companys regulatory filings with the French Autorit des Marchs Financiers, including in the 2019 universal registration document, registered with the French Markets Authorities on April 30, 2020, under number D.20-0427, and future filings and reports by the Company. Furthermore, these forward-looking statements are only as of the date of this press release. Readers are cautioned not to place undue reliance on these forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. If the Company updates one or more forward-looking statements, no inference should be drawn that it will or will not make additional updates with respect to those or other forward-looking statements.

This press release has been prepared in both French and English. In the event of any differences between the two texts, the French language version shall supersede.

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Lysogene Announces a Research Collaboration With the Weizmann Institute of Science - BioSpace

The Global Cell and Gene Therapy Market Market Research Report 2020 published by Prophecy Market Insights is an all-inclusive business research study on the current state of the industry which analyzes innovative strategies for business growth and describes significant factors such as top developers/manufacturers, production value, key regions, and growth rate. Impact of Covid-19 pandemic on the market will be completely analyzed in this report and it will also quantify the impact of this pandemic on the market.

The research study encompasses an evaluation of the market, including growth rate, current scenario, and volume inflation prospects, based on DROT and Porters Five Forces analyses. The market study pitches light on the various factors that are projected to impact the overall market dynamics of the Global Cell and Gene Therapy Market market over the forecast period (2019-2029).

Regional Overview:

The survey report includes a vast investigation of the geographical scene of the Global Cell and Gene Therapy Market market, which is manifestly arranged into the localities. The report provides an analysis of regional market players operating in the specific market and outcomes related to the target market for more than 20 countries.

Australia, New Zealand, Rest of Asia-Pacific

The facts and data are represented in the Global Cell and Gene Therapy Market report using graphs, pie charts, tables, figures and graphical representations helping analyze worldwide key trends & statistics on the state of the industry and is a valuable source of guidance and direction for companies and individuals interested in the market.

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The research report also focuses on global major leading industry players of Global Cell and Gene Therapy Market market report providing information such as company profiles, product picture and specification, R&D developments, distribution & production capacity, distribution channels, price, cost, revenue and contact information. The research report examines, legal policies, and competitive analysis between the leading and emerging and upcoming market trends.

Global Cell and Gene Therapy MarketMarket Key Companies:

The predictions mentioned in the Global Cell and Gene Therapy Market market report have been derived using proven research techniques, assumptions and methodologies. This market report states the overview, historical data along with size, share, growth, demand, and revenue of the global industry.

Segmentation Overview:

Global Cell and Gene Therapy Market, By-Products:

Global Cell and Gene Therapy Market, By Distribution Channel Type:

Global Cell and Gene Therapy Market, By End-Users:

The report provides an in-depth analysis of the Global Cell and Gene Therapy Market market segments and highlights the latest trending segment and major innovations in the market. In addition to this, it states the impact of these segments on the growth of the market. Apart from key players analysis provoking business-related decisions that are usually backed by prevalent market conditions, we also do substantial analysis of market based on COVID-19 impact, detailed analysis on economic, health and financial structure.

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Global Cell and Gene Therapy Market Market: Revenue Growth and Applications Insights - Cole of Duty

The Global Gene Therapy Technologies Market Research Report provides customers with a complete analytical study that provides all the details of key players such as company profile, product portfolio, capacity, price, cost, and revenue during the forecast period from 2020 to 2027. The report provides a full assessment. Gene Therapy Technologies market with future trends, current growth factors, meticulous opinions, facts, historical data and statistically supported and industry-validated market data.

This Gene Therapy Technologies market research provides a clear explanation of how this market will impress growth during the mentioned period. This study report scanned specific data for specific characteristics such as Type, Size, Application and End User. There are basic segments included in the segmentation analysis that are the result of SWOT analysis and PESTEL analysis.

To Learn More About This Report, Request a Sample Copy:https://www.worldwidemarketreports.com/sample/116778* The sample copy includes: Report Summary, Table of Contents, Segmentation, Competitive Landscape, Report Structure, Methodology.

Bluebird bio, Adaptimmune, GlaxoSmithKline, Merck, Celgene, Shanghai Sunway Biotech, BioCancell, Shenzhen SiBiono GeneTech, SynerGene Therapeutics, OncoGenex Pharmaceuticals, Genelux Corporation, Cell Genesys, Advantagene, GenVec, BioCancell, Celgene, Epeius Biotechnologies, Introgen Therapeutics, Ziopharm Oncology are some of the major organizations dominating the global market.(*Note: Other Players Can be Added per Request)

Key players in the Gene Therapy Technologies market were identified through a second survey, and their market share was determined through a primary and second survey. All measurement sharing, splitting, and analysis were solved using a secondary source and a validated primary source. The Gene Therapy Technologies market report starts with a basic overview of the Industry Life Cycle, Definitions, Classifications, Applications, and Industry Chain Structure, and when used together, how key players can meet market coverage, offered characteristics, and customer needs It helps to understand.

The report also makes some important suggestions for new Gene Therapy Technologies market projects before evaluating their feasibility. Overall, this report covers Gene Therapy Technologies market Sales, Price, Sales, Gross Profit, Historical Growth,and Future Prospects. It provides facts related to the widespread merger, acquisition, partnership, and joint venture activities on the market.

This report includes market size estimates of value (million US $) and trading volume (K MT). The top-down and bottom-up approaches are used to estimate and validate the market size of the Gene Therapy Technologies market, estimating the size of various other subordinate markets in the overall market. All ratio sharing, splitting, and analysis were determined using the secondary source and the identified primary source.

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Remarkable Attributes of Gene Therapy Technologies Market Report:

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Gene Therapy Technologies Market 2020 by Latest Innovations, Emerging Technology and Top Key Players: Bluebird bio, Adaptimmune, GlaxoSmithKline -...

5pm on June 26, 2020 - Kawasaki/Japan: The Innovation Center of NanoMedicine (iCONM), the National Institute for Quantum Science and Technology (QST), and the University of Tokyo jointly announced that a reagent for the selective and safe coating of the liver sinusoidal walls to control the clearance of gene therapy drugs was successfully developed. The contents of this research will be published in Science Advances by the American Association for the Advancement of Science (AAAS) at 2:00 pm on June 26, east coast of the United States (Japan standard time: 3:00 am on 27th): A. Dirisala, S. Uchida, K. Toh, J. Li, S. Osawa, T. A. Tockary, X. Liu, S. Abbasi, K. Hayashi, Y. Mochida, S. Fukushima, H. Kinoh, K. Osada, Kazunori Kataoka, "Transient stealth coating of liver sinusoidal wall by anchoring two-armed PEG for retargeting nanomedicines".

Recently, gene therapies have been successively approved in Europe, US, and Japan, and are expected to provide novel therapeutic options for cancer, chronic diseases, acquired and inherited genetic disorders. Whilst this is promising, in reality, when gene therapy drugs are systemically administered to living organisms, they are rapidly eliminated and metabolized in the liver, thus impeding the delivery of a sufficient amount to the target organs and raising the toxicity concerns. This elimination by the liver is caused by the adsorption of the gene therapy drugs to the vascular wall of the liver sinusoid, which is an intrahepatic capillary. To overcome this issue, we conceived to selectively coat the liver sinusoidal wall using polyethylene glycol (PEG). However, a long-term coating may impair the normal physiological functions of the liver, and therefore the coating should be transient. In addition, coating needs to be selective for liver sinusoids, as coating the blood vessels throughout the body would not only cause adverse effects but also decrease the delivery amount of gene therapy drugs to target organs. Towards this end, we have developed a coating agent with two-armed PEG conjugated to positively charged oligolysine, which demonstrated the selective coating on the liver sinusoidal wall, the first-of-its-kind strategy in the world. Interestingly, the coating with two-armed PEG was excreted into bile within 6 hours after binding to sinusoidal walls, while the coating with single chain of linear PEG bound to oligolysine persisted in the walls for a long time. In this way, the precise molecular design was necessary to achieve a transient coating.

This coating was subsequently applied to boost the delivery efficacy of gene therapy drugs. Adeno-associated virus (AAV) is widely used for viral gene therapy drugs, and its serotype 8 (AAV8) targets myocardium and skeletal muscles. When AAV8 was administered after prior coating of two-armed PEG to the liver sinusoidal wall, the transfer of AAV8 to the liver was suppressed, and as a result, the gene transfer efficiency into the myocardium and skeletal muscles was improved by 2 to 4 times. This approach is promising for the treatment of muscular dystrophy. In addition, we expanded the use of our strategy to virus-free gene delivery systems, which allows more economically attractive and safe gene therapy. We have been working on non-viral gene therapy for malignant tumors using plasmid DNA-equipped smart nanomachine for over 10 years. When the coating agent was used for this system, the adsorption of nanomachines to the sinusoidal wall was suppressed, resulting in an approximately 10-fold improvement in DNA transfer efficiency to colon cancer. As described above, we have succeeded in boosting the activity of gene therapy drugs while ensuring safety by using the coating agent developed this time.

The above findings are summarized as follows:

- The coating agent with two-armed PEG selectively coated the liver sinusoid wall for several hours and was then excreted in the bile.

- The coating agent with single chain of linear PEG is not excreted in bile and coated the liver sinusoidal wall for more than 9 hours, which raises a safety concern.

- The coating agent with two-armed PEG had selectivity for the liver sinusoid wall, without coating the blood vessels in the connective tissues.

- The coating agent improved the gene transfer efficacy to the myocardium and skeletal muscles using the AAV vector by 2 to 4 times, and the gene transfer efficiency to colorectal cancer using DNA-loaded smart nanomachines by 10 times.

- As a result, our approach is expected to allow for improving the effect of gene therapy drugs and reducing their dose needed to obtain therapeutic outcome, which will lead to the reduction of medical cost and adverse event opportunities.

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Development of safe liver sinusoid coating agents to increase the efficacy of gene therapy - Science Codex

In recent months, even as our attention has been focused on the coronavirus outbreak, there have been a slew of scientific breakthroughs in treating diseases that cause blindness.

Researchers at U.S.-based Editas Medicine EDIT, +3.85% and Ireland-based Allergan (now owned by AbbVie ABBV, +1.02% ) have administered CRISPR for the first time to a person with a genetic disease. This landmark treatment uses the CRISPR approach to a specific mutation in a gene linked to childhood blindness. The mutation affects the functioning of the light-sensing compartment of the eye, called the retina, and leads to loss of the light-sensing cells.

According to the World Health Organization, at least 2.2 billion people in the world have some form of visual impairment. In the United States, approximately 200,000 people suffer from inherited forms of retinal disease for which there is no cure. But things have started to change for good. We can now see light at the end of the tunnel.

I am an ophthalmology and visual sciences researcher, and am particularly interested in these advances because my laboratory is focusing on designing new and improved gene therapy approaches to treat inherited forms of blindness.

Gene therapy involves inserting the correct copy of a gene into cells that have a mistake in the genetic sequence of that gene, recovering the normal function of the protein in the cell. The eye is an ideal organ for testing new therapeutic approaches, including CRISPR. That is because the eye is the most exposed part of our brain and thus is easily accessible.

The second reason is that retinal tissue in the eye is shielded from the bodys defense mechanism, which would otherwise consider the injected material used in gene therapy as foreign and mount a defensive attack response. Such a response would destroy the benefits associated with the treatment.

In recent years, breakthrough gene therapy studies paved the way to the first-ever Food and Drug Administration-approved gene therapy drug, Luxturna TM, for a devastating childhood blindness disease, Leber congenital amaurosis Type 2. (Luxturna was developed by Spark Therapeutics and licensed to Novartis NVS, +0.84% NOVN, +0.47%. Spark Therapeutics has since been acquired by Roche ROG, +1.35% RHHBY, +1.70% .)

This form of Leber congenital amaurosis is caused by mutations in a gene that codes for a protein called RPE65. The protein participates in chemical reactions that are needed to detect light. The mutations lessen or eliminate the function of RPE65, which leads to our inability to detect light blindness.

The treatment method developed simultaneously by groups at University of Pennsylvania and at University College London and Moorefields Eye Hospital involved inserting a healthy copy of the mutated gene directly into the space between the retina and the retinal pigmented epithelium, the tissue located behind the retina where the chemical reactions takes place. This gene helped the retinal pigmented epithelium cell produce the missing protein that is dysfunctional in patients.

Although the treated eyes showed vision improvement, as measured by the patients ability to navigate an obstacle course at differing light levels, it is not a permanent fix. This is due to the lack of technologies that can fix the mutated genetic code in the DNA of the cells of the patient.

Lately, scientists have been developing a powerful new tool that is shifting biology and genetic engineering into the next phase. This breakthrough gene-editing technology, which is called CRISPR, enables researchers to directly edit the genetic code of cells in the eye and correct the mutation causing the disease.

Children suffering from the disease Leber congenital amaurosis Type 10 endure progressive vision loss beginning as early as one year old. This specific form of Leber congenital amaurosis is caused by a change to the DNA that affects the ability of the gene called CEP290 to make the complete protein. The loss of the CEP290 protein affects the survival and function of our light-sensing cells, called photoreceptors.

One treatment strategy is to deliver the full form of the CEP290 gene using a virus as the delivery vehicle. But the CEP290 gene is too big to be cargo for viruses. So another approach was needed. One strategy was to fix the mutation by using CRISPR.

The scientists at Editas Medicine first showed safety and proof of the concept of the CRISPR strategy in cells extracted from patient skin biopsy and in nonhuman primate animals.

These studies led to the formulation of the first-ever in human CRISPR gene therapeutic clinical trial. This Phase 1 and Phase 2 trial will eventually assess the safety and efficacy of the CRISPR therapy in 18 Leber congenital amaurosis Type 10 patients. The patients receive a dose of the therapy while under anesthesia when the retina surgeon uses a scope, needle and syringe to inject the CRISPR enzyme and nucleic acids into the back of the eye near the photoreceptors.

To make sure that the experiment is working and safe for the patients, the clinical trial has recruited people with late-stage disease and no hope of recovering their vision. The doctors are also injecting the CRISPR editing tools into only one eye.

An ongoing project in my laboratory focuses on designing a gene therapy approach for the same gene CEP290. Contrary to the CRISPR approach, which can target only a specific mutation at one time, my team is developing an approach that would work for all CEP290 mutations in Leber congenital amaurosis Type 10.

This approach involves using shorter yet functional forms of the CEP290 protein that can be delivered to the photoreceptors using the viruses approved for clinical use.

Gene therapy that involves CRISPR promises a permanent fix and a significantly reduced recovery period. A downside of the CRISPR approach is the possibility of an off-target effect in which another region of the cells DNA is edited, which could cause undesirable side effects, such as cancer. However, new and improved strategies have made such likelihood very low.

Although the CRISPR study is for a specific mutation in CEP290, I believe the use of CRISPR technology in the body to be exciting and a giant leap. I know this treatment is in an early phase, but it shows clear promise. In my mind, as well as the minds of many other scientists, CRISPR-mediated therapeutic innovation absolutely holds immense promise.

In another study just reported in the journal Science, German and Swiss scientists have developed a revolutionary technology, which enables mice and human retinas to detect infrared radiation. This ability could be useful for patients suffering from loss of photoreceptors and sight.

The researchers demonstrated this approach, inspired by the ability of snakes and bats to see heat, by endowing mice and postmortem human retinas with a protein that becomes active in response to heat. Infrared light is light emitted by warm objects that is beyond the visible spectrum.

The heat warms a specially engineered gold particle that the researchers introduced into the retina. This particle binds to the protein and helps it convert the heat signal into electrical signals that are then sent to the brain.

In the future, more research is needed to tweak the ability of the infrared sensitive proteins to different wave lengths of light that will also enhance the remaining vision.

This approach is still being tested in animals and in retinal tissue in the lab. But all approaches suggest that it might be possible to either restore, enhance or provide patients with forms of vision used by other species.

Hemant Khanna is an associate professor of ophthalmology at the University of Massachusetts Medical School. This was first published on The Conversation Gene therapy and CRISPR strategies for curing blindness (Yes, you read that right)

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The report is an analytical representation of the assessment of prime growth factors and key growth challenges facing participants in the Independent Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market. With a valuable overview of available areas of opportunity, this study presents detailed information about the most lucrative growth pockets that the companies in market are recommended to capitalize on. Potential market entrants can gain insights on the most profitable growth opportunities that already exist in Independent Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market and those that are most likely to be appearing in market over the course of near term.

The global Independent Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market report evaluates various factors associated with growth, including pricing structure, production capabilities, demand-supply scenarios and profit margins. The entire research intelligence is based on an exhaustive primary industry research and in-depth proactive secondary research that aim to extract valued data points about Independent Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market. The resultant data enables report readers to position themselves as potential market entrants and devise growth strategies to meet short- and long-term business goals.

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By Market Players:BioRelianceRichter-HelmUniQureCobra BiologicsMassBiologicsOxford BioMedicaLonzaMolMedFinVectorFUJIFILM Diosynth BiotechnologiesBrammer Biobluebird bioAldevronSpark TherapeuticsVGXIBiovianEurogentecNovasepPlasmidFactoryCell and Gene Therapy CatapultVigene Biosciences

By TypeAAVAdenoviralLentiviralRetroviralPlasmid DNAOther Vectors

By ApplicationCancersInherited DisordersViral InfectionsOthers

Market segment by Regions/Countries, this report covers

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Table of Contents

Chapter1IndustryOverviewChapter2GlobalViral Vectors, Non-Viral Vectors and Gene Therapy ManufacturingCompetitionbyTypes,Applications,andTopRegionsandCountriesChapter3ProductionMarketAnalysisChapter4GlobalViral Vectors, Non-Viral Vectors and Gene Therapy ManufacturingSales,Consumption,Export,ImportbyRegions(2015-2020)Chapter5NorthAmericaViral Vectors, Non-Viral Vectors and Gene Therapy ManufacturingMarketAnalysisChapter6EastAsiaViral Vectors, Non-Viral Vectors and Gene Therapy ManufacturingMarketAnalysisChapter7EuropeViral Vectors, Non-Viral Vectors and Gene Therapy ManufacturingMarketAnalysisChapter8SouthAsiaViral Vectors, Non-Viral Vectors and Gene Therapy ManufacturingMarketAnalysisChapter9SoutheastAsiaViral Vectors, Non-Viral Vectors and Gene Therapy ManufacturingMarketAnalysisChapter10MiddleEastViral Vectors, Non-Viral Vectors and Gene Therapy ManufacturingMarketAnalysisChapter11AfricaViral Vectors, Non-Viral Vectors and Gene Therapy ManufacturingMarketAnalysisChapter12OceaniaViral Vectors, Non-Viral Vectors and Gene Therapy ManufacturingMarketAnalysisChapter13SouthAmericaViral Vectors, Non-Viral Vectors and Gene Therapy ManufacturingMarketAnalysisChapter14CompanyProfilesandKeyFiguresinViral Vectors, Non-Viral Vectors and Gene Therapy ManufacturingBusinessChapter15GlobalViral Vectors, Non-Viral Vectors and Gene Therapy ManufacturingMarketForecast(2021-2026)Chapter16Conclusions

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Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Key Players, Industry Overview, Application and Analysis to 2020-2026 - 3rd...

Gene Therapy Market 2020: Latest Analysis

Chicago, United States:- The report titled Global Gene Therapy Market is one of the most comprehensive and important additions to Report Hive Research archive of market research studies. It offers detailed research and analysis of key aspects of the global Gene Therapy market. The market analysts authoring this report have provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global Gene Therapy market. Market participants can use the analysis on market dynamics to plan effective growth strategies and prepare for future challenges beforehand. Each trend of the global Gene Therapy market is carefully analyzed and researched about by the market analysts.

Top Players of Gene Therapy Market are Studied: Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical, Advantagene

Download Free Sample PDF (including full TOC, Tables, and Figures) of Gene Therapy Market Research 2020-2026:- @

Global Gene Therapy Market is estimated to reach xxx million USD in 2020 and projected to grow at the CAGR of xx% during 2020-2026. According to the latest report added to the online repository of Report Hive Research the Gene Therapy market has witnessed an unprecedented growth till 2020. The extrapolated future growth is expected to continue at higher rates by 2026.

Our exploration specialists acutely ascertain the significant aspects of the global Gene Therapy market report. It also provides an in-depth valuation in regards to the future advancements relying on the past data and present circumstance of Gene Therapy market situation. In this Gene Therapy report, we have investigated the principals, players in the market, geological regions, product type, and market end-client applications. The global Gene Therapy report comprises of primary and secondary data which is exemplified in the form of pie outlines, Gene Therapy tables, analytical figures, and reference diagrams. The Gene Therapy report is presented in an efficient way that involves basic dialect, basic Gene Therapy outline, agreements, and certain facts as per solace and comprehension.

Segmentation by Application: Cancer DiseasesMonogenic DiseasesInfectious DiseasesCardiovascular DiseasesOthers

Segmentation by Type: Ex vivoIn vivo

NOTE:Due to the pandemic, we have included a special section on the Impact of COVID 19 on the Gene Therapy Market which would mention How the Covid-19 is Affecting the Gene Therapy Industry, Market Trends and Potential Opportunities in the COVID-19 Landscape, Covid-19 Impact on Key Regions and Proposal for Gene Therapy Players to Combat Covid-19 Impact.

The Essential Content Covered in the GlobalGene Therapy Market Report:

* Top Key Company Profiles.* Main Business and Rival Information* SWOT Analysis and PESTEL Analysis* Production, Sales, Revenue, Price and Gross Margin* Market Share and Size

The report provides a 6-year forecast (2020-2026) assessed based on how the Gene Therapy market is predicted to grow in major regions like USA, Europe, Japan, China, India, Southeast Asia, South America, South Africa, Others.

Key Questions Answered In this Report:

What is the overall market size in 2019? What will be the market growth during the forecast period i.e. 2020-2026?

Which region would have high demand for product in the upcoming years?

What are the factors driving the growth of the market?

Which sub-market will make the most significant contribution to the market?

What are the market opportunities for existing and entry-level players?

What are various long-term and short-term strategies adopted by the market players?

What are the key business strategies being adopted by new entrants in the Gene Therapy Market?

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Table of Contents

Market Overview: This is the first section of the report that includes an overview of the scope of products offered in the global Gene Therapy market, segments by product and application, and market size.

Market Competition by Player: Here, the report shows how the competition in the global Gene Therapy market is growing or decreasing based on deep analysis of market concentrate rate, competitive situations and trends, expansions, merger and acquisition deals, and other subjects. It also shows how different companies are progressing in the global Gene Therapy market in terms of revenue, production, sales, and market share.

Company Profiles and Sales Data: This part of the report is very important as it gives statistical as well as other types of analysis of leading manufacturers in the global Gene Therapy market. It assesses each and every player studied in the report on the basis of main business, gross margin, revenue, sales, price, competitors, manufacturing base, product specification, product application, and product category.

Market Status and Outlook by Region: The report studies the status and outlook of different regional markets such as Europe, North America, the MEA, Asia Pacific, and South America. All of the regional markets researched about in the report are examined based on price, gross margin, revenue, production, and sales. Here, the size and CAGR of the regional markets are also provided.

Market by Product: This section carefully analyzes all product segments of the global Gene Therapy market.

Market by Application: Here, various application segments of the global Gene Therapy market are taken into account for research study.

Market Forecast: It starts with revenue forecast and then continues with sales, sales growth rate, and revenue growth rate forecasts of the global Gene Therapy market. The forecasts are also provided taking into consideration product, application, and regional segments of the global Gene Therapy market.

Upstream Raw Materials: This section includes industrial chain analysis, manufacturing cost structure analysis, and key raw materials analysis of the global Gene Therapy market.

Marketing Strategy Analysis, Distributors: Here, the research study digs deep into behavior and other factors of downstream customers, distributors, development trends of marketing channels, and marketing channels such as indirect marketing and direct marketing.

Research Findings and Conclusion: This section is solely dedicated to the conclusion and findings of the research study on the global Gene Therapy market.

Appendix: This is the last section of the report that focuses on data sources, viz. primary and secondary sources, market breakdown and data triangulation, market size estimation, research programs and design, research approach and methodology, and the publishers disclaimer.

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COVID-19 impact on Gene Therapy Market Share, Size, Revenue, Gross Margin and Growth Rate Analysis 2020-2026

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Impact of Corona on Gene Therapy Market 2020 Global Industry Size, Future Growth By 2026 | Sangamo, Spark Therapeutics, Dimension Therapeutics - 3rd...

Genespire and the San Raffaele Telethon Institute for Gene Therapy announce publication in Nature Biotechnology on enhanced gene editing technique in hematopoietic stem cells

Italy, Milan, 30 June 2020: The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and Genespire, a gene therapy company developing transformative therapies for genetic diseases, announce today the publication of data highlighting progress in the development of an improved targeted gene replacement technology in human hematopoietic stem cells (HSCs) in Nature Biotechnology.

The paper, entitled Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking, outlines technology developed by Pr. Luigi Naldini and his team at SR-Tiget, which is included in the strategic alliance with Genespire. It shows increased homology directed recombination (HDR) efficiency in HSCs by forcing cell-cycle progression and transiently upregulating components of the HDR machinery. The findings are validated by clonal tracking of the edited HSCs in experimental transplantation models, which shows improved polyclonal engraftment by long-term repopulating HSCs.

People with genetic diseases affecting the hematopoietic lineage may benefit from corrective targeted gene therapy in HSCs. These cells are self renewing and can differentiate into all the cell types of the hematopoietic lineage, therefore providing the potential for a one-time therapy. As compared to standard gene replacement approaches, gene editing corrects the disease-causing mutation in situ, restoring both function and physiological expression control of the affected gene. In principle, this targeted strategy may fulfill the goal of precision medicine at the most stringent genetic level. Its realization in HSCs, however, has been hampered until now by low efficiency of HDR-driven repair, likely because of the quiescent state of the more primitive progenitors. Use of the improved gene editing technology developed by SR-Tiget has been shown to yield a greater percentage of gene-edited HSCs and increased clonality, or the number of modified cells transplanted and engrafted in the recipient. In a clinical setting this should lead to increased hematopoietic cells chimerism in the patient receiving the corrective HSC therapy, and could accelerate the hematopoietic recovery after conditioning and increase the size, long-term stability, and safety of the engineered cell graft.

This approach can be applied to genetic diseases originating in the hematopoietic lineage, including primary immune deficiencies (PIDs), a key area of focus for Genespire. Genespire will continue to work with SR-Tiget and apply this technology to its future pipeline of gene therapies.

Julia Berretta, Chief Executive Officer of Genespire, commented: The focus of Genespires alliance with SR-Tiget is to research and develop novel gene therapies, addressing severe diseases with high unmet medical need. We are pleased with the publication of these data in Nature Biotechnology, which provide valuable insights into this pioneering technology developed by SR-Tiget, and we look forward to our future work with them to translate cutting edge science into transformational therapies.

Professor Luigi Naldini, Director of SR-Tiget and scientific co-founder of Genespire, said Our findings elucidate and overcome two main biological barriers to efficient HDR-mediated gene editing in HSCs, and show by clonal tracking that our enhanced editing protocol preserves their multilineage and self-renewal capacity long term after serial transplant. We look forward to our future work with Genespire to explore its potential in primary immunodeficiencies.

The full publication details are below and can be accesed online here.

Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking Samuele Ferrari, Aurelien Jacob, Stefano Beretta, Giulia Unali, Luisa Albano, Valentina Vavassori, Davide Cittaro, Dejan Lazarevic, Chiara Brombin, Federica Cugnata, Anna Kajaste-Rudnitski, Ivan Merelli, Pietro Genovese and Luigi Naldini

Enquiries:

About Genespire

Genespire is a biotechnology company focused on the development of transformative gene therapies for patients affected by genetic diseases, particularly primary immunodeficiencies and inherited metabolic diseases. Based in Milan, Italy, Genespire was founded in March 2020 by the gene therapy pioneer Prof. Luigi Naldini and Dr. Alessio Cantore, Fondazione Telethon and Ospedale San Raffaele. It is a spin-off of SR-Tiget, a world leading cell and gene therapy research institute and is backed by Sofinnova Partners. http://www.genespire.com

Story continues

About SR-Tiget

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele and Fondazione Telethon. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases. Over the years, the Institute has given a pioneering contribution to the field with relevant discoveries in vector design, gene transfer strategies, stem cell biology, identity and mechanism of action of innate immune cells. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and storage disorders, which have already treated >115 patients and have led through collaboration with industrial partners to the filing and approval of novel advanced gene therapy medicines.

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Genespire and the San Raffaele Telethon Institute for Gene Therapy announce publication in Nature Biotechnology on enhanced gene editing technique in...

XconomyNational

A new playbook is needed to ensure consistent chemistry, manufacturing, and controls (CMC) reviews for gene therapy products, the lack of which is hindering the development of these products, asserted a top official at the US Food and Drug Administration .

Now is the time to get things right asserted Peter Marks, director of the FDAs Center for Biologics Evaluation and Research, who spoke at a 15 June virtual Drug Information Association annual meeting session on how innovation can help overcome hurdles for these products.

The sessions moderator, Nancy Myers, president of Catalyst Healthcare Consultants, asked the panelists to describe some of their main CMC constriction points in developing gene therapy products, and to identify potential solutions. The other panelists were Karen Walker, the senior advisor for cell and gene therapy at Genentech, who formerly was at Novartis (NYSE: NVS) and worked on the development of Kymriah, and Michael Paglia, director of CMC for ElevateBio.

Myers said that there are two common types of roadblocks to getting gene therapy products through the development pipeline, and these are logistical and technical challenges. The logistical challenges are having a well-trained workforce, managing global distribution networks and ensuring products are transported in cold temperatures, while the technical challenges are ensuring the quality of the starting materials and scaling up production from the research site to commercial manufacturing.

Another roadblock is the lack of standards and lack of a regulatory framework for these products. Myers said that this is a new and growing field and companies are trying to lay the track as they are trying to drive the train down the track at the same time.

CONSISTENT CMC PLAYBOOK NEEDED

Myers first asked the panelists to discuss what they see as constriction points in manufacturing gene therapy products. In response, Marks said that a lack of consistent reviews is hindering their development.

It has become apparent over the last couple of months that, while we have excellent reviewers, it does happen that people can have differences of opinion. I think we will have to come around and have a clear playbook so that everyone gets the same advice especially as we have grown. I know that someone out there will say, we had two different CMC reviewers and two differences pieces of advice. I am not going to argue with that. That is an issue here. As we come to the post-COVID period we should to try to have more unity in what comes from our CMC reviews. I cannot say the problem is solved but the problem has been identified and is amenable to solutions.

He further noted that the lack of clear regulatory pathway for these products is a major roadblock in accelerating their development. We do not have the preclinical pathways set up and the clinical set up and the regulatory paradigm is yet to be fleshed out. Now is the ripe time to get things right.

Marks also noted some of the manufacturing challenges in the cell and gene therapy space: We are in a place where our current vectors are limiting what we can address in terms of our ability to product them on a very large scale, and what will probably take some years to get there. On the other hand, the piece that really interests me is how do we deal with hundreds and thousands of rare diseases that we cant address right now through the production of gene therapy products where we simply do not have the manufacturing capacity to be able to produce these products in a rapid manner because we just dont have the systems.

MORE ON WHY DEVICE-LIKE REVIEW COULD HELP

Marks expanded on an idea he had suggested in February, that reviews for gene therapies should be more aligned with the device model. (Also see Individualized Gene Therapy: US FDA Considering Device-Like Manufacturing Approval Process Pink Sheet, 28 Feb, 2020.)

It is becoming increasingly clear that for cell and gene therapies, the manufacturing is more like a device paradigm with continued innovations, he said. With a traditional drug you come up with a chemical process to make a small molecule and you are probably using the process similarly across the lifecycle, but you are not constantly finding ways to do things that fundamentally change the yield or quality of a product. Here we have issues that manufacturing changes can potentially change the product for the better.

He added that we have to find some balance here between the traditional drug manufacturing model of once and done to something that is asking you go through multiple cycles of a device every two to three years where you are changing the technology. With device cycles, you may have multiple generations of the device over years. With a device you can measure things nicely, with biologicals you cannot measure easily.

Walker concurred that these are not well-characterized products and so we need to invest heavily in Next Page

Joanne Serpick Eglovitch is a senior editor for Pink Sheet where she writes about manufacturing and quality issues.

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FDA Official: New "Playbook" Needed for CMC Reviews of Gene Therapy Products - Xconomy

The strategy analysis on Global Gene Therapy for Rare Disease Market gives insights of market size, trends, share, growth, development plans, Investment Plan, cost structure and drivers analysis. With precise data covering all key aspects of the existing market, this report offers existing data of leading manufacturers. The Gene Therapy for Rare Disease market report covers marketing channels and market positioning to potential growth strategies, providing in-depth analysis for new competitors or exists competitors in the Gene Therapy for Rare Disease industry. The Report Gives Detail Analysis on Market concern Like Gene Therapy for Rare Disease Market share, CAGR Status, Market demand and up to date Market Trends with key Market segments. The report provides key statistics on the market status of the Gene Therapy for Rare Disease manufacturers and is a valuable source of guidance and direction for companies and individuals interested in the industry. Overall, the report provides an in-depth insight of Gene Therapy for Rare Disease market covering all important parameters.

Note: *The Download PDF brochure only consist of Table of Content, Research Framework, and Research Methodology.

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Global Gene Therapy for Rare Disease market 2020-2026: Competitive AnalysisThe Gene Therapy for Rare Disease market report designed to provide entry support, customer profile and M&As as well as go-to-market strategy support. We provide a detailed analysis of key players operating in the global Gene Therapy for Rare Disease market, including key players such as Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and BioMarin Pharmaceutical Inc.

Scope of Gene Therapy for Rare Disease Market:

The Gene Therapy for Rare Disease market was valued at XX Million US$ in 2019 and is projected to reach XX Million US$ by 2024, at a CAGR of XX% during the forecast period. In this study, 2019 has been considered as the base year and 2020 to 2024 as the forecast period to estimate the market size for Gene Therapy for Rare Disease.

Due to the pandemic, we have included a special section on the Impact of COVID 19 on the Gene Therapy for Rare Disease Market which would mention How the Covid-19 is Affecting the Gene Therapy for Rare Disease Industry, Market Trends and Potential Opportunities in the COVID-19 Landscape, Covid-19 Impact on Key Regions and Proposal for Gene Therapy for Rare Disease Players to fight Covid-19 Impact.

The report also focuses on global major leading industry players of Global Gene Therapy for Rare Disease market providing information such as company profiles, product picture and specification, price, capacity, cost, production, revenue and contact information. Upstream raw materials and equipment and downstream demand analysis are also carried out. With tables and figures helping analyze worldwide Global Gene Therapy for Rare Disease market, this research provides key statistics on the state of the industry and is a valuable source of guidance and direction for companies and individuals interested in the market. In general, the research report is a compilation of key data with regards to the competitive landscape of this vertical and the multiple regions where the business has successfully established its position. The report provides detailed information regarding the major factors (drivers, restraints, opportunities, and challenges) influencing the growth of the Gene Therapy for Rare Disease market. The Gene Therapy for Rare Disease Market Report analyzes opportunities in the overall Gene Therapy for Rare Disease market for stakeholders by identifying the high growth segments.

The scope of the report is limited to the application of the type, and distribution channel. The regions considered in the scope of the report include North America Country (United States, Canada), South America, Asia Country (China, Japan, India, Korea), Europe Country (Germany, UK, France, Italy), Other Country (Middle East, Africa, GCC). This report presents the worldwide Gene Therapy for Rare Disease market size (value, production and consumption), splits the breakdown (data status 20152019 and forecast to 2024), by manufacturers, region, type and application.

Market segment by Type, the product can be split into:Product Type Segmentation: Epicel, IntegraIndustry Segmentation: Chemical, Cosmetic, Pharmaceutical

The Global Gene Therapy for Rare Disease Market report analyses the production of goods, supply, sales, and the current status of the market in a detailed manner. Furthermore, the report examines the production shares and market product sales, as well as the capacity, production capacity, trends in sales, cost analysis, and revenue generation. Several other factors such as import/export status, industrial statistics, demand and supply ratio, gross margin, and industry chain structure have also been studied in the Global Gene Therapy for Rare Disease Market report.

The report comprehends precise analytical information related to market forecasts for several upcoming years. The report also includes the particulars about the valuation of macro and microelements significant for the growth of already established Gene Therapy for Rare Disease Market contenders and emerging new companies. The report uses SWOT analysis for the growth assessment of the outstanding Gene Therapy for Rare Disease Market players. It also analyzes the most recent enhancements while estimating the expansion of the foremost Gene Therapy for Rare Disease Market players. Additionally, the key product category and segments along with sub-segments of the global Gene Therapy for Rare Disease Market are studied in the global Market research.

What Reports Provides

Full in-depth analysis of the parent market Important changes in market dynamics Segmentation details of the market Former, on-going, and projected market analysis in terms of volume and value Assessment of niche industry developments Market share analysis Key strategies of major players Emerging segments and regional markets Testimonials to companies in order to fortify their foothold in the market.

Further, in the research report, the following points are included along with an in-depth study of each point:

* Production Analysis Production is analyzed with respect to different regions, types, and applications. Here, the price analysis of various Market key players is also covered.* Sales and Revenue Analysis Both, sales and revenue are studied for the different regions of the global market. Another major aspect, price, which plays an important part in the revenue generation is also assessed in this section for the various regions.* Supply and Consumption In continuation of sales, this section studies the supply and consumption of the Market. This part also sheds light on the gap between supply and consumption. Import and export figures are also given in this part.* Other analyses Apart from the information, trade and distribution analysis for the Market, contact information of major manufacturers, suppliers and key consumers are also given. Also, SWOT analysis for new projects and feasibility analysis for new investment are included.

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Additionally, the report is joined by a vital examination of the Gene Therapy for Rare Disease marketplace considering progress, part commitments, and future market forecasts. Furthermore, it offers detailed data of vendors including the profile, specifications of a product, sales, applications, annual performance in the industry, investments, acquisitions and mergers, market size, revenue, market share, and more. The report also studies individual regional market size along with country-wise and region-wise market size during the forecast period. The report also understands the export and import, production, and consumption of every particular region holding the highest market share, market size, or CAGR.

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Gene Therapy for Rare Disease Market 2026: Global Demand, Key Players, Overview, Supply and Consumption Analysis - 3rd Watch News

The Global Gene Therapy Industry Market report by UpMarketResearch.com provides a detailed analysis of the area marketplace expanding; competitive landscape; global, regional, and country-level market size; impact market players; market growth analysis; market share; opportunities analysis; product launches; recent developments; sales analysis; segmentation growth; technological innovations; and value chain optimization. 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.

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Market Segmentation

The Global Gene Therapy Industry Market has been divided into product types, application, and regions. These segments provide accurate calculations and forecasts for sales in terms of volume and value. This analysis can help customers increase their business and take calculated decisions.

By Product Types,Type 1Type 2Type 3

By Applications,Application 1Application 2Application 3

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

The regional analysis segment is a highly comprehensive part of the report on the global Gene Therapy Industry market. This section offers information on the sales growth in these regions on a country-level Gene Therapy Industry market.

The historical and forecast information provided in the report span between 2018 and 2026. The report provides detailed volume analysis and region-wise market size analysis of the market.

Competitive Landscape of the Gene Therapy Industry Market

The chapter on competitive landscape provides information about key company overview, global presence, sales and revenue generated, market share, prices, and strategies used.

Major players in the global Gene Therapy Industry Market include company 1company 2company 3company 4company 5company 6company 7company 8company 9

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The Gene Therapy Industry Market Report Addresses:

The Report Provides:

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Gene Therapy Industry Market Industry Perspective, Comprehensive Analysis, Size, Share, Growth, Segment, Trends And Forecast, 2025 - Cole of Duty

You dont go more than 40 years in biotech without ever getting a product to market unless you can learn the art of writing a promotional press release. And Inovio captures the prize in baiting the hook.

Tuesday morning Inovio, which has been struggling to get its Covid-19 vaccine lined up for mass manufacturing, put out a release that touched on virtually every hot button in pandemic PR.

There was, first and foremost, an interim snapshot of efficacy from their Phase I program for INO-4800.

Analyses to date have shown that 94% (34 out of 36 total trial participants) demonstrated overall immunological response rates based on preliminary data assessing humoral (binding and neutralizing) and T cell immune responses.

Moderna was criticized for its decision to sketch initial antibody responses which it said were at or above the level needed to guard against the pandemic at 2 doses over various times. But it provided a treasure of information compared to Inovios barebones explanation. The full data set, Inovio says, will be published later in a peer-reviewed journal. In the meantime, theyre largely leaving the data readout to the reader to decipher.

Also dangling bait to day traders:

A Phase II/III study should be set to go this summer, if regulators sign off. Plus, Operation Warp Speed the presidents initiative at achieving a vaccine rollout, around the election is including 4800 in a non-human primate study. And then there was the trusty mouse study, in which their vaccine prevented viral replication in rodent lungs after the tiny creatures were challenged with SARS-CoV-2.

Usually, that sort of release delivers a powerful if temporary surge in the share price. But this morning, Wall Street proved skeptical.

Inovios stock price slid 11% as some experienced observers called them out.

Inovios attempt to break out of the pack seemed to largely backfire this morning. When youre in a race involving AstraZeneca, Pfizer, Moderna, a host of Chinese companies, and many, many more, it seems the usual bait doesnt work so well.

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Positive Covid-19 vaccine data? New mouse study? OWS inclusion? Yep, but somehow, the usual tidbits from Inovio backfire - Endpoints News

DUBLIN, July 1, 2020 /PRNewswire/ -- The "Rare Disease Diagnostics: Technologies and Global Markets" report has been added to ResearchAndMarkets.com's offering.

The global rare disease diagnostics market should reach $26.7 billion by 2024 from $17 billion in 2019, rising at a CAGR of 9.5% over the forecast period.

The scope of the report includes rare disease diagnostic technologies, applications, industries, initiatives, patents and companies. The market for rare disease diagnostic products and services is given for 2018 and 2019, and then forecast through 2024.

This report reviews the main diagnostic technologies and explains why genetic variation is important in clinical testing and disease. It then discusses significant large-scale research initiatives that impact rare disease diagnostic applications. Of particular interest is a discussion of global population-scale sequencing projects and their likely impact in linking genetic variation to rare disease diagnostics. The main market driving forces for rare disease diagnostic products and services are listed and discussed.

The report categorizes and quantifies the rare disease diagnostics market by the disease category, technology platform, test purpose, analysis target and geography segments.

More than 95 companies in the rare disease diagnostic industry are profiled in this report.

The research also provides a summary of more than 50 of the main industry acquisitions and strategic alliances that took place from April 2018 through April 2020, including key alliance trends.

The report includes:

Market Insights

Rare diseases comprise a growing public health priority, as they affect upward of 300 million people globally and they are difficult to diagnose and treat.

There is a pressing need for better ways to detect and diagnose rare diseases, as well as to provide companion diagnostics for therapy guidance, clinical trials enrollment and therapy monitoring applications.

Better diagnostic tests for rare diseases can make significant differences in the lives of those affected by these conditions. Many rare diseases go undiagnosed for long periods of time because patients, families and physicians may have limited awareness of certain diseases, and the symptoms may not be informative to healthcare workers who may not have encountered such diseases before.

Extended time to diagnosis of a rare disease, along with so-called diagnostic odysseys, can lead to negative outcomes, including misdiagnosis or disease progression. Rapid, accurate diagnostics can significantly shorten these diagnostic odysseys.

In addition to early detection and diagnostic potential, rare disease therapeutics will be important in orphan drug development and use. Orphan drugs address rare disease patient populations, and they are expected to have a high growth rate through 2024. By 2024, orphan drugs may make up as much as one-fifth of global prescription sales. Rare disease diagnostics can be used to help physicians make proper decisions regarding which therapies to use and ways to monitor the efficacy of those therapies during treatment courses. Rare disease diagnostics can also be used to help select patients for orphan drug clinical trials.

More than 70% of rare diseases are inherited conditions, and they thus have genetic components, so this industry relies heavily on genetic analysis methods, including polymerase chain reaction (PCR), next-generation sequencing (NGS) and Sanger sequencing.

Key Topics Covered

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Overview

Chapter 4 Technology Background

Chapter 5 Rare Disease Diagnostics Initiatives

Chapter 6 Rare Disease Diagnostic Industries

Chapter 7 Rare Disease Diagnostics Strategic Alliances and Acquisitions

Chapter 8 Rare Disease Diagnostics Markets

Chapter 9 Rare Disease Diagnostics Patents and Intellectual Property

Chapter 10 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/rp6ok6

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

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Research and Markets Laura Wood, Senior Manager [emailprotected]

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Rare Disease Diagnostics Industry Anticipated to Reach $26.7 Billion by 2024 - Market Shares by Disease Class, Indication, Analysis Platform, Analysis...

The Gene Therapy Market Research Report 2020 published by Prophecy Market Insights is an all-inclusive business research study on the current state of the industry which analyzes innovative strategies for business growth and describes significant factors such as top developers/manufacturers, production value, key regions, and growth rate. Impact of Covid-19 pandemic on the market will be completely analyzed in this report and it will also quantify the impact of this pandemic on the market.

The research study encompasses an evaluation of the market, including growth rate, current scenario, and volume inflation prospects, based on DROT and Porters Five Forces analyses. The market study pitches light on the various factors that are projected to impact the overall market dynamics of the Gene Therapy market over the forecast period (2019-2029).

Regional Overview:

The survey report includes a vast investigation of the geographical scene of the Gene Therapy market, which is manifestly arranged into the localities. The report provides an analysis of regional market players operating in the specific market and outcomes related to the target market for more than 20 countries.

Australia, New Zealand, Rest of Asia-Pacific

The facts and data are represented in the Gene Therapy report using graphs, pie charts, tables, figures and graphical representations helping analyze worldwide key trends & statistics on the state of the industry and is a valuable source of guidance and direction for companies and individuals interested in the market.

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The research report also focuses on global major leading industry players of Gene Therapy market report providing information such as company profiles, product picture and specification, R&D developments, distribution & production capacity, distribution channels, price, cost, revenue and contact information. The research report examines, legal policies, and competitive analysis between the leading and emerging and upcoming market trends.

Gene TherapyMarket Key Companies:

GlaxoSmithKline plc, Bluebird Bio, Inc., Adaptimmune Therapeutics plc, Celgene Corporation, Shanghai Sunway Biotech Co. Ltd., Merck KGaA, Transgene SA, and OncoGenex Pharmaceuticals, Inc.

The predictions mentioned in the Gene Therapy market report have been derived using proven research techniques, assumptions and methodologies. This market report states the overview, historical data along with size, share, growth, demand, and revenue of the global industry.

Segmentation Overview:

The report provides an in-depth analysis of the Gene Therapy market segments and highlights the latest trending segment and major innovations in the market. In addition to this, it states the impact of these segments on the growth of the market. Apart from key players analysis provoking business-related decisions that are usually backed by prevalent market conditions, we also do substantial analysis of market based on COVID-19 impact, detailed analysis on economic, health and financial structure.

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Gene Therapy Market: Predictable To Witness Sustainable Evolution over 2020-2030 - 3rd Watch News

Four more US biotechs filed to go public Friday as yet more companies clamber to get through a yawning IPO window and onto a market thats signaled its willingness to reward nearly any new drugmaker.

The new entrants are led by ALX Oncology and the biological analytics biotech Berkeley Lights, each of whom filed to raise $100 million. The autoimmune company Pandion Therapeutics also filed for $75 million, and Kiromic Biopharma, a tiny immuno-oncology startup based in San Antonio, filed for $25 million.

These companies will try to capitalize on a 2020 biotech IPO boom that the investment firm Renaissance Capital recently called historic. The spree began in January and, after a brief interlude when the pandemic first hit the US and Europe, has only picked up in the last two months. The 23 companies that have gone public averaged an 80% return on their offering price, according to Renaissance Capital numbers. Every single one priced above their midpoint or upsized their offering.

Unlike most of their fellow newly or would-be public biotechs, Berkeley Lights will enter the market with significant revenue on the books. The company doesnt make drugs but instead has built a digital cell biology platform that can analyze living cells from a variety of different dimensions and, in principal, accelerate drug development. Theyve partnered with Sanofi and Pfizer on antibody discovery and last year, signed a $150 million pact with Ginkgo Bioworks to help the synthetic biology unicorn advance its genetic engineering capabilities.

All told, the company earned $51 million in revenue last year. Unlike a drug developer, they have no cash earmarked for specific pipeline products, and said they will use proceeds for research, potential acquisitions and general corporate purposes.

For ALX Oncology, a successful offering would mean their second $100 million tranche of the year. In February, the California biotech raised $105 million to help advance its sole pipeline candidate: an antibody designed to target CD-47. Thats the same dont-eat-me signal targeted by Irv Weissmans Forty Seven Inc., the biotech Gilead paid $5 billion for in January. ALXs pitch is that their antibodys FC receptor is engineered to not attract macrophages, reducing toxicity. The biotech will use their proceeds to push the drug through its ongoinghead and neck squamous cell carcinomaand gastric cancer trial and begin new trials for it in acute myeloid leukemia and myelodysplastic syndrome. A portion is also earmarked for CMC work.

Founded out of Polaris in 2018, Pandion Therapeutics was tapped last year for an up-to $800 million partnership to help a reorganizing Astellas develop antibodies for auto-immune disorders. That deal included $45 million upfront and the company also earned $80 million from a Series B in April. The new funding will be used to push their lead molecule through Phase I/II trials in ulcerative colitis while also backing preclinical research, particularly on a pair of antibodies meant to turn on the PD-1 checkpoint and tamp down the immune system.

Kiromic, meanwhile, is in part just trying to stay alive. With less than $2 million 5 million when a subsequent $3 million Series B is included in the bank at years end, they acknowledged in their S-1 that theres substantial doubt regarding the Companys ability to continue as a going concern. In this climate, though, thats worked out just fine for other companies. Applied Molecular Transport went publicin May with the same concerns. They ultimately raised $177 million.

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The biotech IPO boom is becoming 'historic' as four more throw their hats in - Endpoints News

A cancer-fighting concept from the inventor of the first cancer vaccine is nearing prime time, and its biotech developer has received a significant new infusion of cash to get it there.

Bolt Biotherapeutics announced a $93.5 million Series C round led by Sofinnova Investments and joined by more than 9 others, including Pfizer Ventures and RA Capital Management. That money will go toward pushing the San Francisco biotechs platform of innate immune-boosting warheads through its first trial on metastatic solid tumors and into several more.

Its a fairly elegant but simple molecule, CEO Randy Schatzman told Endpoints News. Its basically an off-the-shelf therapeutic that physicians can call on to treat their patients without all the personalization and manufacturing difficulties that many of the I/O approaches have today.

The experimental drug is what Bolt calls an immune-stimulating antibody conjugate. Its based on a concept from Stanford immunologist Edgar Engleman, who invented the first cancer vaccine in Provenge, a treatment for prostate cancer. That vaccine involves a multi-step process where innate immune cells called dendrites are extracted, exposed to a protein from the patients own tumors (as you would expose the immune system to a viral or bacterial protein in a traditional vaccine) and then reinfused.

Bolts platform is designed to directly activate dendrites around tumors without the need for removal, treatment and re-infusion. The therapy is an antibody drug conjugate similar to those produced by Seattle Genetics, ADC Therapeutics, and Immunomedics, each of which contain an antibody to guide the therapy like a warhead to the tumor. The difference is that instead of strapping a chemical to the antibody that will kill cells in the area, Bolts drug has an agonist that will bind to receptors on the dendrites and activate them to attack the tumor.

If it works, the drug would turn the environment around the tumor from an immuno-suppressive to one that could not only kill the tumors once but prevent recurrence. Their first candidate usesa Herceptin biosimilar to target HER2, but in theory, Schatzman said, it should work with an antibody targeted at any oncogene and Bolt is exploring those options, both internally and with partnerships.

In February, five years after the companys foundation, they launched their first trial, putting the drug known as BDC-1001 into a Phase I/II trial for metastatic tumors that are HER2+, including gastric and breast cancer. That trial will test the drug both as a single-agent therapy and in combination with Mercks Keytruda. The idea is that activating the immune system in two different ways might make for a more effective treatment.

Despite some early concerns, Schatzman says, the company has not seen any delays in that trial from Covid-19.

After the first data emerge from the Phase I/II trial, the company will move into trials specifically for gastric and breast cancer, looking to see if it can not only clear cancers initially but also, by keeping the immune system on guard, ward off recurrence, an event that can come quickly for patients with these late-stage tumors.

Were going to see some quick answers in going past what is the current standard of care, Schatzman said. Id like to think it will take a long time to know where the end of this is, because in that sense, well have been very effective at treating these patients.

Read the original here:
Bolt Biotherapeutics nabs $93.5M to push Provenge inventor's new idea deeper in the clinic - Endpoints News

Editors note: Exposure to UV-C light poses known threats to human health. While the research discussed in this story examined ways that UV-C might be broadly deployed to stem the spread of COVID-19, it is presented only as information about new research. Readers are strongly cautionednot to experiment on their own with UV-C as an antimicrobial measure.

COVID-19 has upended society and brought chaos to generous swaths of the global economy, as governments have locked down to prevent the spread of the virus. The pandemic has also boosted interest in a variety of technologies that might help in the coronavirus fightincluding the use of ultraviolet light to sterilize scarce personal protective equipment (PPE) such as face masks, for reuse.

In recent papers, two research teams have now proposed approaches to take the use of UV-C much farther. The two groups lay out different schemes for using this high-energy light to scrub the pandemic virus from the air in occupied indoor spaceswhile still keeping the occupants safe from the usual harmful long-term effects of exposure to UV radiation. The teams argue that the approaches they suggest, if widely adopted, could sharply reduce viral transmission in public indoor spaces, and thereby help the world resume something closer to normal economic activity.

The antimicrobial effects of UV-C lightwhich occupies the wavelength band from 200 to 280 nmhave long been known, and 254-nm mercury-vapor germicidal lamps are routinely used to disinfect empty hospital rooms, clean up tainted water, and otherwise cleanse surfaces and areas where bacteria and viruses may lurk. The problem is that the same light that hammers pathogenic airborne bacteria also wreaks havoc on human cells, with long-term impacts such as skin cancer and eye damage.

As a result, much of the recent interest in leveraging UV-C light against the COVID-19 virus, SARS-CoV-2, has centered around possible uses in disinfecting PPE for doctors, nurses and first responders. (One such effort, focusing on a low-cost system that can be used to disinfect scarce N95 face masks for reuse in resource-limited areas, is being pursued by an interdisciplinary team including OSA Fellow and past president Thomas Baer.)

The teams behind the two recent papers favor substantially broadening the use of UV-C against SARS-CoV-2. They suggest that, with the right precautions and setups, the light might be used as a prophylactic in occupied indoor spaces such as workplaces, schools, hospitals and other public areas, silently scrubbing the air of the coronavirus even as the occupants of the buildings go about their daily lives.

One of the studies was led by OSA Fellow Javier Garca de Abajo of the Institute of Photonic SciencesICFO, Spain, and tapped a multinational, interdisciplinary team of researchers in virology, aerosols, immunology and other areas (ACS Nano, doi: 10.1021/acsnano.0c04596). In the study, the researchers looked at how the judicious, strategic installation of UV-C sources might attack the most common routes of indoor viral transmission.

The team began by inventorying the indoor places and systems most likely to spread the virus. These, the researchers argue, include interior ventilation systems in a wide range of settings, which can spread airborne viruses; infrastructure items touched by many persons, such as elevator buttons, stair rails and public-transit handles; and common public facilities with repeated, periodic high use, such as public toilets, storerooms and other areas.

The ICFO-led team argues that antimicrobial UV-C light sources could be placed in a variety of locations associated with viral spread, such as ventilation systems and other areas. The lamps could then be operated without a direct optical pathto humans, or while the roomsare not in use, to help reduce virus propagation without endangering human health. [Image: Sketches by Nacho Gaubert] [Enlarge image]

The team then analyzed how a range of UV-C sources, including LEDs and conventional mercury-vapor lamps, could be safely deployed to attack coronavirus in these individual spread scenarios. For example, the team argues that lamps could be deployed inside ventilation systems, with no direct optical path to occupants in the building, and run continuously, to disinfect ambient air. In other settings such as public restrooms, high-intensity UV-C light could be applied during the periods in which the rooms are unoccupied. And frequently touched surfaces, such as elevator buttons, might be subjected to continuous, weak UV-C illumination for ongoing disinfectionas human interactions with these surfaces tend to be brief, and thus would involve only a very low dose of radiation.

The team acknowledges that putting such an approach into effect would require a massive new deployment of UV lighting, with a correspondingly huge required uptick in its production. Yet the cost, they argue, may not be excessive, given the scale of the problem humanity now confrontsnot just in human mortality, but in the economic devastation that antiviral lockdowns have created.

The team estimates, in fact, that disinfection with fluorescence lamps could be implemented at a cost of a few dollars per person with minimum changes in infrastructure. Thus, they argue, a global capital investment of a few billion [U.S.] dollars could protect on the order of 109 indoor workers worldwide. The authors add, however, that current manufacturers of UV-C sources may have difficulty coping with the expected rise in demand originated by the SARS-CoV-2, pandemic, as the global market for UV-C light barely reaches one billion dollars a year at present.

Another team, based at Columbia Universitys Irving Medical Center, USA, took a different view of how UV-C might be used to fight coronavirus in occupied indoor settings (Sci. Reports, doi: 10.1038/s41598-020-67211-2). The team focused in particular on the specific wavelengths of UV-C light that might be used against the virusstressing that not all UV-C light is necessarily the same in terms of its hazard to human health.

Conventional germicidal lamps operate at a wavelength of 254 nm, as that is one of the sweet spots for UV-C absorption by DNA or RNA molecules; the light thus scrambles and inactivates the pathogens genetic machinery. It can also penetrate into the skin and eyes, causing similar genetic and other damage to living human cells.

The Columbia team points out, however, that light in the far UV-C, between 207 and 222 nm, is also effective at killing microorganismsyet studies to date suggest that these wavelengths do not cause the human health issues associated with the 254-nm radiation of germicidal lamps. The reason, according to the team, is that light in the far UV-C penetrates less than a few micrometers into biological materials. Thats too short a distance to pierce the nonliving protective layers of the skin and eye into the living cells beneath. But its more than sufficient to bore into tiny bacteria and viruses.

These considerations, the Columbia team argues, suggest that far-UV-C light should have about the same anti-microbial properties as conventional germicidal UV light, but without producing the corresponding health effects. Thus, they conclude, continuous, low-dose application of light in the wavelength area of 222 nm might be used in occupied public areas to stem the spread of SARS-CoV-2, without posing other hazards to the occupants themselves.

To test the idea out, the team used a misting device to aerosolize two common coronaviruses that were structurally similar to SARS-CoV-2, and then flowed the aerosols through the air in front of a 222-nm UV-C lamp. The researchers found that, at very low doses, the far UV-C light killed more than 99.9% of the viral load. The team is now at work on tests using the actual SARS-CoV-2 pathogenand says that preliminary data suggest that the far-UV-C light effectively kills that virus, too.

Putting these results together with the safety data, the team leader, David J. Brenner, asserted in a press release accompanying the work that far-UV-C at very low doses could be used in combination with other measures, like wearing face masks and washing hands, to limit the transmission of SARS-CoV-2 and other viruses.

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COVID-19: Putting UV-C to Work - Optics & Photonics News

Researchers who reported that delivering the human ACE2 protein into mouse airway cells creates COVID-19 models have released their findings to allow other scientists to make their own models.

Researchers have created a gene therapy approach that they say can convert any lab mouse into one that can be infected with SARS-CoV-2 and develop COVID-like lung disease. The team have made their gene therapy vector freely available to any researchers who wish to use it. The study was conducted by scientists at the University of Iowa (UI) Carver College of Medicine, US, and Medical University, Guangzhou, China.

There is a pressing need to understand this disease and to develop preventions and treatments, said Dr Paul McCray, UI professor of paediatrics and microbiology and immunology. We wanted to make it as easy as possible for other researchers to have access to this technology, which allows any lab to be able to immediately start working in this area by using this trick.

According to the researchers, the trick is the use of an adenovirus gene therapy vector that is inhaled by the mice to deliver the human angiotensin-converting enzyme 2 (ACE2) protein into mouse airway cells. This is the protein that SARS-CoV-2 uses to infect cells.Once the mouse airway cells express the hACE2 protein, the mice become susceptible to infection with the virus and they develop COVID-19-like lung symptoms.

Due to differences between the human and mouse ACE2 protein, wild-type mice are not susceptible to the SARS-Cov-2 virus, highlighting the need to develop models. Although the disease is not fatal in the models, the animals do get sick, losing weight and developing lung damage.

The vector is readily adaptable to any strain of mice (and other lab animals), which means research teams can rapidly convert mice with specific genetic traits into animals that are susceptible to SARS-Cov-2, allowing them to test whether those traits influence the disease.

The researchers showed that mice treated with this gene therapy could be used to evaluate a vaccine and several potential COVID-19 therapies, including a preventative strategy known as poly I:C, which boosts the innate immune response, convalescent plasma from recovered COVID-19 patients and the antiviral drug remdesivir. In each case, the therapies prevented weight loss, reduced lung disease and increased the speed of virus clearance in the mice. The team also showed that the mice are useful for studying important immune responses involved in clearing the SARS-CoV-2 virus.

The team say that their gene therapy vector is essentially an off-the-shelf tool that allows labs to create their own COVID-19 mouse model within a few days.

You can create these mice very quickly. You do not have to breed the strain, which is very time consuming and expensive, McCray explained. We think this technology will be useful for investigating COVID-19 lung disease and rapidly testing interventions that people think are promising for treating or preventing COVID-19.

The results were published in Cell.

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Method to convert mice into COVID-19 models available to researchers - Drug Target Review

Dive Brief:

Known for its muscular dystrophy drugs Exondys 51 and Vydonys 53, Sarepta has quickly become a leader in gene therapy as well.

The last couple of years have seen the Cambridge, Massachusetts-based biotech ink gene therapy manufacturing and development deals, including a partnership with Roche that could be worth more than $3 billion.

Sarepta now has six gene therapies in clinical testing and another half dozen in preclinical stages. Most recently, the company announced positive, albeit early, results from a study of its experimental treatment for limb-girdle muscular dystrophy, a potentially deadly genetic disease.

With gene therapy set to become a cornerstone of its business, Sarepta is trying to avoid some the challenges presented by current technologies.

For example, when the company teamed up with North Carolina-based StrideBio late last year, one of the main goals was to use the partner's technology to "address re-dosing challenges in patients who have received AAV-delivered gene therapy." The deal came just days after a study testing an experimental gene therapy from Solid Biosciences, one of Sarepta's rivals, was paused due to a patient experiencing an immune response and organ complications.

"If successful, the ability to re-dose will be an enormous leap forward in the science of gene therapy and provide invaluable benefits to patients beyond those we anticipate with one-time dosing," said Doug Ingram, Sarepta's CEO, in a June 18 statement announcing the Selecta deal.

Under terms of that deal, Sarepta will pay Selecta an initial, undisclosed amount. Selecta is eligible to receive pre-clinical milestone payments, and could take home additional development, regulatory and commercial milestones should Sarepta exercise its options to enter a licensing agreement.

Specifically, the deal with Selecta centers on gene therapies for Duchenne muscular dystrophy and certain limb-girdle muscular dystrophies.

With Codiac, Sarepta has offered up $72.5 million in upfront and near-term license payments plus research funding. In addition, Codiak is eligible for "significant" milestone payments, according to Sarepta.

The two-year deal gives Sarepta the option to license Codiak's technology for up to five neuromuscular targets. The companies said they will collaborate on the design of exosomes that can deliver and release gene therapy, gene editing or RNA payloads. If Sarepta exercises an option, Codiak will then be in charge of research until right before the candidate goes into in-human testing. Sarepta is responsible for clinical development and commercial activities.

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Sarepta tries to fine-tune its gene therapy approach - BioPharma Dive

Emergent Biosolutions has set out to capture a share of the expanding viral vector and gene therapy sector.

The US countermeasures developer said it will spend $75 million to add viral vector and gene therapy manufacturing capacity at a facility in Canton, Massachusetts.

At present the plant which was acquired from Sanofi in 2017 produces drug substance for live viral vaccines, including Emergents smallpox jab ACAM2000.

Image: iStock/marchmeena29

Emergent said the investment will establish a multi-suite production operation with 1,000 L of manufacturing capacity.

Syed T. Husain, SVP & head of CDMO Business Unit at Emergent BioSolutions, told us there are significant opportunities in the gene therapy sector.

There is an unmet need in this area. We are seeing long wait times for customers in this space. Emergent has foundational expertise in live viral development and manufacturing, among other competencies, and we are building upon this capability to help produce these therapies and get them to patients.

He added, We are in discussions with interested customers, we would be able to initiate development services in 2022, paving the way for manufacturing in 2023 as the new facility comes online.

The plan is that the Canton facility will make drug substance for viral vector gene therapies, working alongside Emergents development services site in Gaithersburg and its manufacturing plant in Rockville, Maryland.

Emergent spoke about the expansion of its CDMO business during its Q1 conference call in May, predicting that the division would make a greater revenue contribution than forecast.

CFO Rick Lindahl told analysts We now expect a greater revenue contribution from CDMO than previously thought we currently see 2020 CDMO full-year revenue in a range of $125 million to $145 million, representing 55% to 80% growth as compared to 2019.

He added We continue to make progress on our strategic and financial objectives, including the expansion of the CDMO services businesses and its contribution to the overall business.

The gene therapy sector is expected to grow significantly over the next few years.

According to Allied Market Research the gene therapy market, which was valued at $393 million in 2018, is estimated to reach $6.2 billion by 2026, a CAGR of 34.8%.

This take is shared by analysts at Capstoneheadwaters who wrote that pharma interest in gene therapies will increase demand for CDMOs with production capacity.

The authors said, The increasing volume of gene therapy drugs under development will require greater manufacturing capacity and, in turn, greater utilization of CDMO capacity.

CDMOs may be able to increase penetration of their outsourced manufacturing services by providing services at the commercial stage in addition to the earlier stages of preclinical development and clinical trials.

They added, We see the injection of new capital into both gene therapy drug developers and CDMOs focused on gene therapy manufacturing as a positive industry dynamic.

Excerpt from:
Emergent to add gene therapy capacity - Bioprocess Insider - BioProcess Insider

SOUTH SAN FRANCISCO, Calif., June 19, 2020 (GLOBE NEWSWIRE) -- Catalyst Biosciences, Inc. (NASDAQ: CBIO), today presented data from preclinical studies of its hemophilia B gene therapy CB 2679d-GT at the WorldFederation of Hemophilia Virtual Summit, taking place from June 14 -19, 2020.

The oral presentation, entitled: Combination of a Novel Chimeric AAV Capsid and Potency Enhanced FIX Variant for Hemophilia B Gene Therapy, given by Dr. Grant Blouse, senior vice president of translational research, provided preclinical results of CB 2679d-GT, the companys novel FIX gene therapy. CB 2679d-GT was designed to achieve clinically relevant FIX levels at a reduced viral load by combining engineered AAV capsids with Catalysts novel high potency FIX transgene.

The preclinical data from our constructs demonstrated a strong dose response and improved reduction in bleeding relative to the Padua variant, said Nassim Usman, Ph.D., president and chief executive officer of Catalyst. The enhanced FIX activity and reduced viral dose may offer advantages over current AAV-based gene therapies in clinical development.

Studies of CB 2679d-GT in hemophilia B mice have demonstrated a 4-fold reduction in blood loss and an 8-fold reduction in bleeding time when compared with the same dose of the Padua variant of FIX. Furthermore, when packaged in a proprietary chimeric AAV capsid, CB 2679d-GT demonstrated a clear dose response of high stable FIX levels across the three dose levels in hemophilia B mice.

A pilot non-human primate study compared the expression and tolerability of CB 2679d-GT in the novel chimeric capsid KP1 with the LK03 capsid. The study demonstrated that CB 2679d-GT was well tolerated with high FIX expression that stabilized to approximately 25% to 50% FIX above baseline levels at the 6-week interim data cutoff. The novel chimeric capsid had differentiated and superior response to anti-capsid neutralizing antibodies than that observed for the LK03 comparator during the screening of non-human primates for the study.

A copy of the presentation slides can be accessed on the Events and Presentations section of the Catalyst website.

About Catalyst BiosciencesCatalyst is a research and clinical development biopharmaceutical company focused on addressing unmet needs in rare hematologic and systemic complement-mediated disorders. Our protease engineering platform includes development programs in hemophilia, a research program on subcutaneous (SQ) systemic complement inhibitors and a partnered preclinical development program with Biogen for dry age-related macular degeneration (AMD). One of our key competitive advantages is that the product candidates generated by our protease engineering platform have improved functionality and potency. These characteristics allow for improved dosing of our candidates including SQ systemic administration of recombinant coagulation factors and complement inhibitors, low-dose, high activity gene therapy constructs, and less frequently dosed intravitreal therapeutics. Our most advanced asset, SQ MarzAA has successfully completed Phase 2 development in prophylaxis, significantly reducing the annualized bleed rate (ABR) in individuals with Hemophilia A or B with inhibitors. Following regulatory guidance from the U.S. Food and Drug Administration and European Medicines Agency, we recently announced the design of a Phase 3 registration study that is planned for late 2020. Subcutaneous dalcinonacog alfa (DalcA) is being developed for the treatment of Hemophilia B and has demonstrated efficacy and safety in a Phase 2b clinical trial. We have a discovery stage Factor IX gene therapy construct - CB 2679d-GT - for Hemophilia B, that has demonstrated superiority compared with the Padua variant in preclinical models. Finally, we have a global license and collaboration agreement with Biogen for the development and commercialization of anti-complement Factor 3 (C3) pegylated CB 2782 for the potential treatment of geographic atrophy-associated dry AMD.

Forward-Looking StatementsThis press release contains forward-looking statements that involve substantial risks and uncertainties. Forward-looking statements include statements about the superiority of CB 2679d-GT over the Padua variant, enhanced FIX activity of CB 2679d-GT, which may reduce viral dose and maintain high FIX activity levels while potentially decreasing liver toxicity, the chimeric capsid, which may have lower neutralization by pre-existing AAV antibodies, as well as plans for a Phase 3 trial of MarzAA in late 2020 and the Companys collaboration with Biogen for the development and commercialization of pegylated CB 2782 for the potential treatment of geographic atrophy-associated dry age-related macular degeneration. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially, including, but not limited to, the risk that trials and studies may be delayed as a result of the COVID-19 virus and other factors, that trials may not have satisfactory outcomes, that additional human trials will not replicate the results from animal trials or earlier human trials, that potential adverse effects may arise from the testing or use of DalcA or MarzAA, including the generation of neutralizing antibodies, which has been observed in patients treated with DalcA, the risk that costs required to develop or manufacture the Companys products will be higher than anticipated, including as a result of delays in development and manufacturing resulting from COVID-19 and other factors, the risk that Biogen will terminate Catalysts agreement, competition and other risks described in the Risk Factors section of the Companys quarterly report filed with the Securities and Exchange Commission on May 11, 2020, and in other filings with the Securities and Exchange Commission. The Company does not assume any obligation to update any forward-looking statements, except as required by law.

Contact:Ana KaporCatalyst Biosciences, Inc.investors@catbio.com

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Catalyst Biosciences Presents Preclinical FIX Gene Therapy Data in an Oral Presentation at the World Federation of Hemophilia Virtual Summit 2020 -...

The first time Lyell CEO Rick Klausner looked at what PACT Pharma was trying to accomplish with neoantigens, non-viral T cell engineering and cancer, he felt they couldnt get it done. But in the 3 years since theyve launched, Klausner has become a believer.

Now, hes a believer and a partner.

Early Thursday morning, Klausner and PACT CEO Alex Franzusoff announced a plan to jointly pursue one of the Holy Grails of oncology R&D. Blending their technologies and bringing a wide network of leading experts to the table, the two companies are working on a personalized T cell therapy for solid tumors. And an IND is in the offing.

The collaboration joins the Lyell team, which has been concentrating on overcoming the exhaustion that afflicts the first generation of cell therapies, with a PACT group that has developed tech to identify a patients unique signature of cancer mutations and use a non-viral method to engineer their T cells into cancer therapies.

I spent some time on Wednesday talking with Klausner and Franzusoff about the deal, which comes with an undisclosed set of financials as Lyell invests in the alliance.

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