StemCell Therapy

Notice: This Content doesnt contain all the Information of the Report please fill the form (via link) and get all interesting information just one click in PDF with the latest update with chart and Table of Content

The report offers a systematic presentation of the existing trends, growth opportunities, market dynamics that are expected to shape the growth of the Cell Therapy Manufacturing Market. The various research methods and tools were involved in the market analysis, to uncover crucial information about the market such as current & future trends, opportunities, business strategies and more, which in turn will aid the business decision-makers to make the right decision in future.

This Report Covers Leading Companies Associated in Worldwide Cell Therapy Manufacturing Market: Pharmicell, Merck Group, Dickinson and Company, Thermo Fisher, Lonza Group, Miltenyi Biotec GmBH, Takara Bio Group, STEMCELL Technologies, Cellular Dynamics International, Becton, Osiris Therapeutics, Bio-Rad Laboratories, Inc., Anterogen, MEDIPOST, Holostem Terapie Avanazate, Pluristem Therapeutics, Brammer Bio, CELLforCURE, Gene Therapy Catapult EUFETS, MaSTherCell, PharmaCell, Cognate BioServices and WuXi AppTec.

We Do Offer Sample of this report. Kindly go through the following information in order to access the report.

Note- This report sample includes:

Brief Introduction to the research report. Table of Contents (Scope covered as a part of the study) Top players in the market Research framework (Structure Of The Report) Research methodology adopted by Coherent Market Insights

Get Sample Copy Of this Research Report @ https://www.coherentmarketinsights.com/insight/request-sample/1728

The report begins with a brief introduction and market overview of the Cell Therapy Manufacturing industry followed by its market scope and size. Next, the report provides an overview of market segmentation such as type, application, and region. The drivers, limitations, and opportunities for the market are also listed along with current trends and policies in the industry.

The key players profiled in this report include:Pharmicell, Merck Group, Dickinson and Company, Thermo Fisher, Lonza Group, Miltenyi Biotec GmBH, Takara Bio Group, STEMCELL Technologies, Cellular Dynamics International, Becton, Osiris Therapeutics, Bio-Rad Laboratories, Inc., Anterogen, MEDIPOST, Holostem Terapie Avanazate, Pluristem Therapeutics, Brammer Bio, CELLforCURE, Gene Therapy Catapult EUFETS, MaSTherCell, PharmaCell, Cognate BioServices and WuXi AppTec.

Regions included:

o North America (United States, Canada, and Mexico)

o Europe (Germany, France, UK, Russia, and Italy)

o Asia-Pacific (China, Japan, Korea, India, and Southeast Asia)

o South America (Brazil, Argentina, Colombia)

o The Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

The PDF Research only provides a Table of Contents (ToC), the scope of the report and research framework of the report.

Get Free PDF Brochure Of this Research Report @ https://www.coherentmarketinsights.com/insight/request-pdf/1728

Key Benefits:

o This study gives a detailed analysis of drivers and factors limiting the market expansion of Cell Therapy Manufacturing

o The micro-level analysis is conducted based on its product types, end-user applications, and geographies

o Porters five forces model gives an in-depth analysis of buyers and suppliers, threats of new entrants & substitutes and competition amongst the key market players

o By understanding the value chain analysis, the stakeholders can get a clear and detailed picture of this Cell Therapy Manufacturing market

The research study can answer the following Key questions:

What will be the progress rate of the Cell Therapy Manufacturing Market for the conjecture period, 2020 2027?What are the prominent factors driving the Cell Therapy Manufacturing Market across different regions?Who are the major vendors dominating the Cell Therapy Manufacturing industry and what are their winning strategies?What will be the market scope for the estimated period?What are the major trends shaping the expansion of the industry in the coming years?What are the challenges faced by the Cell Therapy Manufacturing Market?

Table of Contents

Report Overview: It includes the Cell Therapy Manufacturing market study scope, players covered, key market segments, market analysis by application, market analysis by type, and other chapters that give an overview of the research study.

Executive Summary: This section of the report gives information about Cell Therapy Manufacturing market trends and shares, market size analysis by region and analysis of global market size. Under market size analysis by region, analysis of market share and growth rate by region is provided.

Profiles of International Players: Here, key players of the Cell Therapy Manufacturing market are studied on the basis of gross margin, price, revenue, corporate sales, and production. This section gives a business overview of the players and shares their important company details.

Regional Study: All of the regions and countries analyzed in the Cell Therapy Manufacturing market report is studied on the basis of market size by application, the market size by product, key players, and market forecast.

Apply Promo Code CMIFIRST1000 And Get Instant Discount Of USD 1000

Ask For Discount Before Purchasing This Business Report : https://www.coherentmarketinsights.com/insight/request-discount/1728

Thanks for reading this article, you can also get individual chapter wise section or region wise report version like North America, LATAM, West Europe, MENA Countries, Southeast Asia or Asia Pacific.

**BE SAFE AND STAY HOME**

About Us:

Coherent Market Insights is a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity.

Contact Us:Coherent Market Insights,1001 4th Ave,#3200 Seattle, WA 98154, U.S.Email: [emailprotected]Phone: US +1-206-701-6702/UK +44-020 8133 4027

See original here:
Cell Therapy Manufacturing Market Growth, Demand, And Value Is Increasing In The Upcoming Years - The Daily Chronicle

Chicago, United States:The global Cell and Gene Therapy Market report offers a complete research study that includes accurate estimations of market growth rate and size for the forecast period 2020-2025. It offers a broad analysis of market competition, regional expansion, and market segmentation by type, application, and geography supported by exact market figures. The all-inclusive market research report also offers Porters Five Forces Analysis and profiles some of the leading players of the global Cell and Gene Therapy Market. It sheds light on changing market dynamics and discusses about different growth drivers, market challenges and restraints, and trends and opportunities in detail. Interested parties are provided with market recommendations and business advice to ensure success in the global Cell and Gene Therapy Market.

Top Key players cited in the report:Amgen Inc., bluebird bio, Inc., Dendreon Pharmaceuticals LLC., Fibrocell Science, Inc., Human Stem Cells Institute, Kite Pharma, Inc., Kolon TissueGene, Inc., Novartis AG, Orchard Therapeutics plc., Organogenesis Holdings Inc., Pfizer, Inc., RENOVA THERAPEUTICS, .

Get Free PDF Sample Copy of this Report to understand the structure of the complete report: (Including Full TOC, List of Tables & Figures, Chart): https://www.reporthive.com/request_sample/2369809

The final report will add the analysis of the Impact of Covid-19 in this report Cell and Gene Therapy Market

Cell and Gene Therapy Marketreports offers important insights which help the industry experts, product managers, CEOs, and business executives to draft their policies on various parameters including expansion, acquisition, and new product launch as well as analyzing and understanding the market trends.

Each segment of the global Cell and Gene Therapy market is extensively evaluated in the research study. The segmental analysis offered in the report pinpoints key opportunities available in the global Cell and Gene Therapy market through leading segments. The regional study of the global Cell and Gene Therapy market included in the report helps readers to gain a sound understanding of the development of different geographical markets in recent years and also going forth. We have provided a detailed study on the critical dynamics of the global Cell and Gene Therapy market, which include the market influence and market effect factors, drivers, challenges, restraints, trends, and prospects. The research study also includes other types of analysis such as qualitative and quantitative.

Global Cell and Gene Therapy Market: Competitive Rivalry

The chapter on company profiles studies the various companies operating in the global Cell and Gene Therapy market. It evaluates the financial outlooks of these companies, their research and development statuses, and their expansion strategies for the coming years. Analysts have also provided a detailed list of the strategic initiatives taken by the Cell and Gene Therapy market participants in the past few years to remain ahead of the competition.

Global Cell and Gene Therapy Market: Regional Segments

The chapter on regional segmentation details the regional aspects of the global Cell and Gene Therapy market. This chapter explains the regulatory framework that is likely to impact the overall market. It highlights the political scenario in the market and the anticipates its influence on the global Cell and Gene Therapy market.

Request For Customization:https://www.reporthive.com/request_customization/2369809

Report Highlights

Table of Contents

Report Overview:It includes six chapters, viz. research scope, major manufacturers covered, market segments by type, Cell and Gene Therapy market segments by application, study objectives, and years considered.

Global Growth Trends:There are three chapters included in this section, i.e. industry trends, the growth rate of key producers, and production analysis.

Cell and Gene Therapy Market Share by Manufacturer:Here, production, revenue, and price analysis by the manufacturer are included along with other chapters such as expansion plans and merger and acquisition, products offered by key manufacturers, and areas served and headquarters distribution.

Market Size by Type:It includes analysis of price, production value market share, and production market share by type.

Market Size by Application:This section includes Cell and Gene Therapy market consumption analysis by application.

Profiles of Manufacturers:Here, leading players of the global Cell and Gene Therapy market are studied based on sales area, key products, gross margin, revenue, price, and production.

Cell and Gene Therapy Market Value Chain and Sales Channel Analysis:It includes customer, distributor, Cell and Gene Therapy market value chain, and sales channel analysis.

Market Forecast Production Side: In this part of the report, the authors have focused on production and production value forecast, key producers forecast, and production and production value forecast by type.

Get Free Sample Copy of this report:https://www.reporthive.com/request_sample/2369809

About Us:Report Hive Research delivers strategic market research reports, statistical survey, and Industry analysis and forecast data on products and services, markets and companies. Our clientele ranges mix of United States Business Leaders, Government Organizations, SMEs, Individual and Start-ups, Management Consulting Firms, and Universities etc. Our library of 600,000+ market reports covers industries like Chemical, Healthcare, IT, Telecom, Semiconductor, etc. in the USA, Europe Middle East, Africa, Asia Pacific. We help in business decision-making on aspects such as market entry strategies, market sizing, market share analysis, sales and revenue, technology trends, competitive analysis, product portfolio and application analysis etc.

The rest is here:
COVID-19 Impact on Cell and Gene Therapy Market Research, Size, Growth And Trends 2020 to 2025: Keyplayer-Amgen Inc., bluebird bio, Inc. - Scientect

AVROBIO, a US-based company, developing lentiviral-based gene therapies, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for the Companys investigational gene therapy, AVR-RD-02, for the treatment of Gaucher disease.

Get a complete list of the presentations,here.

What is AVR-RD-02: AVR-RD-02 consists of the patients own hematopoietic stem cells, genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease. It is an ex vivo lentiviral-based investigational gene therapy. The stem cells are genetically modified to express functional glucocerebrosidase (GCase), which reduces levels of glucosylceramide and glucosylsphingosine, the accumulated substances which cause the symptoms of Gaucher disease.

How advanced is AVR-RD-02 development program: The Company is actively recruiting in Canada for its Phase 1/2 clinical trial of AVR-RD-02, which seeks to evaluate the safety and efficacy of the therapy in patients with Type 1 Gaucher disease.

What is an orphan drug designation: Orphan-drug designation provides special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor. For a drug to qualify for orphan designation both the drug and the disease or condition must meet certain criteria specified in the ODA and FDAs implementing regulations at 21 CFR Part 316. Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received orphan designation is not subject to a prescription drug user fee unless the application includes an indication for other than the rare disease or condition for which the drug was designated.

What is the gene therapy pipeline like for the treatment of Gaucher disease: There are only a few gene therapy candidates that are being developed for Gaucher disease.

For further information, check out the report here

Read more insights at

Roots Analysis Leaders in Pharmaceutical & Biotechnology Market Research

You may also be interested in the following titles:

About Roots Analysis

Roots Analysis is one of the fastest growing market research companies, sharing fresh and independent perspectives in the bio-pharmaceutical industry. The in-depth research, analysis and insights are driven by an experienced leadership team which has gained many years of significant experience in this sector. If youd like help with your growing business needs, get in touch at [emailprotected]

Contact Information

Roots Analysis Private Limited

Gaurav Chaudhary

+1 (415) 800 3415

[emailprotected]

Continue reading here:
Gene Therapy Pipeline Update: AVROBIO Receives Orphan-Drug Designation | Roots Analysis - The News Brok

The report covers the forecast and analysis of the Cell and Gene Therapy Consumables market on a global and regional level. The study provides historical data from 2015 to 2018 along with a forecast from 2019 to 2027 based on revenue (USD Million). The study includes drivers and restraints of the Cell and Gene Therapy Consumables market along with the impact they have on the demand over the forecast period. Additionally, the report includes the study of opportunities available in the Cell and Gene Therapy Consumables market on a global level.

Download Sample of This Strategic Report:https://www.kennethresearch.com/sample-request-10172657

In order to give the users of this report a comprehensive view of the Cell and Gene Therapy Consumables market, we have included a competitive landscape and an analysis of Porters Five Forces model for the market. The study encompasses a market attractiveness analysis, wherein all the segments are bench marked based on their market size, growth rate, and general attractiveness.

The report provides company market share analysis to give a broader overview of the key players in the market. In addition, the report also covers key strategic developments of the market including acquisitions & mergers, new service & product launches, agreements, partnerships, collaborations & joint ventures, research & development, and regional expansion of major participants involved in the market on a global and regional basis.

The study provides a decisive view of the Cell and Gene Therapy Consumables market by segmenting the market based on product type, application/therapeutics, and regions. All the segments have been analyzed based on present and future trends and the market is estimated from 2019 to 2027. The regional segmentation includes the current and forecast demand for North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.

A rise in the awareness about the gene & cell therapies will propel the market growth during the period from 2019 to 2027. Nevertheless, conducting randomized control tests will inhibit the expansion of the market during the forecast timeline. However, the growing trend for treating neurodegenerative ailments through the use of gene treatment will proliferate the market growth over the forecast period.

The expansion of the market during the forecast timespan is owing to the high frequency of chronic ailments including cancer and heart disorders. Apart from this, inflation in the occurrence of these disorders produces lucrative demand for enhanced therapies and this will culminate in the market demand over the forecast timespan.

Request For Full Report:https://www.kennethresearch.com/sample-request-10172657

Based on the product type, the market is sectored into Kits & Buffers, Diagnostic Assay, Culture Medium, and Cryopreservation Media. Application/ Therapeutics- wise, the market for cell and gene therapy consumables are classified into Cardiovascular, Urology, Dermatology, Critical Care, Respiratory, Endocrine & Metabolic, Neuroscience, Hematology & Oncology, Obstetrics, Immunology, and Gastroenterology.

Some of the key players in the market include Amgen Inc., ATLANTA BIOLOGICALS, bluebird bio, Inc., Cook, Dendreon Pharmaceuticals, LLC, Fibrocell Science, Inc., General Electric, Kolon TissueGene, Inc., Orchard Therapeutics plc., Pfizer, Inc., PromoCell GmbH, RENOVA THERAPEUTICS, Sibiono GeneTech Co. Ltd., Spark Therapeutics, Inc., Vericel, Helixmith Co., Ltd., and Vitrolife.

About Kenneth Research

Kenneth Research is a reselling agency providing market research solutions in different verticals such as Automotive and Transportation, Chemicals and Materials, Healthcare, Food & Beverage and Consumer Packaged Goods, Semiconductors, Electronics & ICT, Packaging, and Others. Our portfolio includes set of market research insights such as market sizing and market forecasting, market share analysis and key positioning of the players (manufacturers, deals and distributors, etc), understanding the competitive landscape and their business at a ground level and many more. Our research experts deliver the offerings efficiently and effectively within a stipulated time. The market study provided by Kenneth Research helps the Industry veterans/investors to think and to act wisely in their overall strategy formulation

Contact Us

Kenneth Research

Email : [emailprotected]

Phone: +1 313 462 0609

Read more:
Cell and Gene Therapy Consumables Market 2020-2027/ Size, Segments and Supply Demand Forecast - StartupNG

The Global Gene Therapy Market report focuses on market size, status, and forecast 2020-2024, along with this, the report also focuses on market opportunities and threats, tactical decision-making, and evaluating the market. The Gene Therapy market report delivers data and information on changing investment structure, technological advancements, market tendencies and developments, capacities, and detailed information about the key players of the global market. In addition to this, the report also involves the development of the Gene Therapy market in the major regions across the world.

Cutting-edge released the research study on Global Gene Therapy Market, which deals a exhaustive overview of the factors influencing the global business scope. Gene Therapy Market research report shows the latest market insights, current situation analysis with upcoming trends, and breakdown of the products and services. The Gene Therapy Industry Report delivers key statistics on the market status, size, share growth factors of the Gene Therapy .

Get a PDF Sample Copy of this Gene Therapy Market Report:

https://www.globalmarketers.biz/report/automotive-and-transportation/global-automotive-gasoline-engine-turbocharger-market-2019-by-manufacturers,-regions,-type-and-application,-forecast-to-2024/130925#request_sample

Top Leading players of Gene Therapy Market Covered in the Report:

Bluebird BioSangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVical Inc.Advantagene

The report has enclosed key geographic regions such as Europe, Japan, United States, India, Southeast Asia and Europe. As far as the sub-regions, North America, Canada, Medico, Australia, Asia-Pacific, India, South Korea, China, Singapore, Indonesia, Japan, Rest of Asia-Pacific, Germany, United Kingdom, France, Spain, Italy, Rest of Europe, Russia, Central & South America, Middle East & Africa are included.

Key Market Segmentation of Gene Therapy :

On the basis of types, the Gene Therapy Market from 2020 to 2024 is primarily split into:

Ex vivoIn Vivo

On the basis of applications, the Gene Therapy Market from 2020 to 2024 covers:

CancerMonogenicInfectious diseaseCardiovascular diseaseOther

Ask For Discount:

https://www.globalmarketers.biz/discount_inquiry/discount/130925

The Gene Therapy Market Research Report furthermore delivers a local examination of the market with a high focus on showcase development, development rate, and development potential. The research report calculates marketplace length estimate to analyze investment potentials and growth.

In this study, the years considered to estimation the market size of the Gene Therapy Industry Market: History Year: 2014-2018Base Year: 2018Estimated Year: 2019Forecast Year 2019 to 2024

The Gene Therapy market report provides answers to the following key questions:

Major Points Covered in Table of Contents:

For More Information and get a complete Table of content of Gene Therapy Market report:

https://www.globalmarketers.biz/report/automotive-and-transportation/global-automotive-gasoline-engine-turbocharger-market-2019-by-manufacturers,-regions,-type-and-application,-forecast-to-2024/130925#table_of_contents

Read more:
Global Gene Therapy Market 2020-2024 Expected To Witness The Highest Growth During Forecast period - Scientect

The global Gene Therapies for Cancer Treatment market study covers the projection size of the market both in terms of value (Mn/Bn US$) and volume (x units). The report estimates the lookup of different local distributors in the overall market and provides the market size of the Gene Therapies for Cancer Treatment market using both bottom-up and top-down approaches. To investigate the key players and their market contribution, primary and secondary research has been comprehensively performed. In addition, all the figures, subdivisions, and shares have been collected with the help of trustworthy sources.

In the Gene Therapies for Cancer Treatment market research study, 2018 is considered as the base year, and 2019-2029 is considered as the forecast period to predict the market size. The report identifies each Gene Therapies for Cancer Treatment market player on the basis of market share, production portfolio, and growth rate. In addition, the research study analyzes the strengths, weaknesses, opportunities and threats of the players.

Make An EnquiryAbout This Report @ https://www.marketresearchhub.com/enquiry.php?type=E&repid=2692433&source=atm

Global Gene Therapies for Cancer Treatment market report on the basis of market players

The following players are covered in this report:Takara BioTocagenVBL TherapeuticsCold GenesysGenprexMomotaro-GeneMultiVirSynerGene TherapeuticsZiopharm OncologyAnchiano TherapeuticsCelgeneCelsionBluebird BioGene Therapies for Cancer Treatment Breakdown Data by TypeSomatic Cell Gene Therapy (SCGT)Germline Gene Therapy (GGT)Gene Therapies for Cancer Treatment Breakdown Data by ApplicationCancer Research CentersDiagnostic LaboratoriesCancer HospitalsOthers

The report provides market share, consumption pattern, and influencing factors of each region. Prominent countries driving the regional growth are also covered in the report.

Request Sample Report @ https://www.marketresearchhub.com/enquiry.php?type=S&repid=2692433&source=atm

Highlights of the report:

The Gene Therapies for Cancer Treatment market report answers the following questions:

You can Buy This Report from Here @ https://www.marketresearchhub.com/checkout?rep_id=2692433&licType=S&source=atm

See the original post here:
Gene Therapies for Cancer Treatment Market Projected to Witness Vigorous Expansion by 2019-2025 - Scientect

Adenovirus vectors have become a popular delivery platform for gene therapy and vaccines. However, many people have preexisting immunity against adeno-associated viruses (AAVs), which could significantly reduce vector uptake and therefore render the therapeutic cargo they carry ineffective. Even in individuals that lack preexisting immunity, the first inoculation of a vector could induce immune responses that can thwart repeat administration.

To overcome that obstacle, scientists at the University of Pittsburgh created a CRISPR-based system that they say could effectively prevent immunity to the viral vector. The CRISPR tool briefly suppressedgenes that are related to anti-adenovirus antibody production and helped achieve better gene therapy uptake in mice. The results werepublished in Nature Cell Biology.

Samira Kiani, the studys co-senior author, has co-founded SafeGen Therapeutics with the goal to bring this platform to the clinic to enable safer gene therapy.

Accelerate Clinical Operations Across Sponsors, CROs, and Partners

The most advanced life sciences organizations know that digital innovation and multi-platform integrations are essential for enabling product development. New platforms are providing the life sciences industry with an opportunity to improve the efficiency of clinical trials and reduce costs while remaining compliant and reducing risk.

Many clinical trials fail because of the immune response against AAV gene therapy, Kiani said in a statement. And then you cant re-administer the shot because people have developed immunity.

One recent case in point is CanSino Biologics adenovirus type 5 (Ad5)-vectored COVID-19 vaccine. In the shots phase 2 trial, about half of the participants had high levels of preexisting Ad5 neutralizing antibody. These individuals were found to produce significantly lower immune responses to the vaccine than did those with low preexisting anti-Ad5 immunity.

Kiani and colleagues set out to modify the gene expression that's associated with an immune response against AAV, but only temporarilyjust long enough for the therapeutic content to do its jobbecause they didnt want to tamper with normal immune functions.

RELATED:Hopkins team invents non-viral system for getting gene therapy into cells

CRISPR, which can edit out a defective gene or add in a functional one, is already being explored as a gene therapy strategy. So the University of Pittsburgh researchers figured they could hit two birds with one stone by utilizing CRISPR to also control the immune response.

They treated mice with the CRISPR-based system and then re-exposed them to AAV, and the animals didnt make more antibodies against the virus. The treated mice were more receptive to subsequent AAV-delivered gene therapy than were the control animals, the team reported.

Gene therapies hold promise as potential cures to many hard-to-treat diseases that would otherwise require chronic treatment. So scientists have been trying to perfect their transport vehicles for better safety and efficacy.

A team led by scientists at the San Raffaele Telethon Institute for Gene Therapy in Italy, for example,attached the CD47 protein to the surface of lentiviral vectors so they could escape detection and destruction by the immune system. And researchersfrom Johns Hopkins University developed a nonviral delivery system that uses a polymer nano-container, whichreleases the therapy once inside the cell.

Now, Kianihopes to validate the CRISPR-based technology at SafeGen. In addition to its potential as a gene therapy delivery platform, the researchers also found that their method can prevent or treat sepsis in mice, suggesting it might also be useful for treating inflammatory conditions.

Read the original here:
Using CRISPR to improve viral vectors for gene therapy - FierceBiotech

Gene therapy generally relies on viruses, such as adeno-associated virus (AAV), to deliver genes into a cell. In case of CRISPR-based gene therapies, molecular scissors can then snip out a defective gene, add in a missing sequence or enact a temporary change in its expression, but the bodys immune response to AAV can thwart the whole endeavor.

To overcome that obstacle, researchers at the University of Pittsburgh created a system that uses CRISPR in a different way. Their system briefly suppresses genes that are related to AAV antibody production so the virus can deliver its cargo unimpeded. These results published today inNature Cell Biology.Many clinical trials fail because of the immune response against AAV gene therapy, said study co-senior author Samira Kiani, associate professor of pathology in Pitt'sSchool of Medicineand member of thePittsburgh Liver Research Center(PLRC) andMcGowan Institute for Regenerative Medicine. And then you cant readminister the shot because people have developed immunity.

So Kiani and her long-time collaborator Mo Ebrahimkhani, associate professor of pathology at Pitt, member of PLRC and the McGowan Institute, set out to modify gene expression related to the bodys immune response to AAV. But this gene is important for normal immune function, so the researchers didnt want to shut it down forever, just tamp it down momentarily.

Since CRISPR is such a convenient system for editing the genome, the pair figured they would put it to use for altering the master switches that orchestrate genes involved in immune response.

Were hitting two birds with one stone, said Ebrahimkhani. You can use CRISPR to do your gene therapy, and you can also use CRISPR to control the immune response.

When the researchers treated mice with their CRISPR-controlled immune suppression system and then exposed them to AAV again, the animals didnt make more antibodies against the virus. These animals were more receptive to subsequent AAV-delivered gene therapy compared to controls.

Beyond gene therapy, the study also shows that CRISPR-based immune suppression can prevent or treat sepsis in mice, highlighting the potential for this tool to be broadly useful for a range of inflammatory conditions, including cytokine storm andacute respiratory distress syndrome, both of which can crop up with COVID-19, though more studies are needed to engineer safety features.

The main goal of this study was to develop CRISPR-based tools for inflammatory conditions, said study lead author Farzaneh Moghadam, a PhDstudent in Kianis lab. But when we looked at bone marrow samples, we saw that the group treated with our tool showed a lower immune response to AAV compared to the control group. That was very interesting, so we started exploring how this tool contributes to antibody formation against AAV and could potentially address safety and efficacy concerns with gene therapy trials.

Kiani co-founded SafeGen Therapeutics with the goal of bringing this technology to the clinic.

This study was supported by National Institute of Biomedical Imaging and Bioengineering, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, and a DARPA Young Faculty Award.

Reference: Moghadam F, LeGraw R, Velazquez JJ, et al. Synthetic immunomodulation with a CRISPR super-repressor in vivo. Nature Cell Biology. 2020;22(9):1143-1154. doi:10.1038/s41556-020-0563-3.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

Continued here:
Editing the Immune Response To Aid the Delivery of Gene Therapy Cargo - Technology Networks

TipRanks

As fears of a tech bubble and stretched valuations become the talk of the town, investors are turning to Wall Street titans for guidance, namely Ken Griffin. Founding hedge fund Citadel in 1990, the firm now boasts over $35 billion worth of assets under management.As a 19-year-old sophomore at Harvard University, Griffin began trading from his dorm room with a fax machine, computer and phone. Now, the CEO of Citadel, whose net worth stands at $15.5 billion, is known as one of the Wall Street greats. Looking at the funds performance during the COVID crisis, its even more clear why Griffin has legendary status.Unlike the average hedge fund, which had a negative return of between 3-4% in the first half of 2020, Citadels flagship Wellington fund saw its returns land between 13-14% for the same period.Bearing this in mind, we wanted to take a closer look at three stocks Citadel snapped up recently. Using TipRanks database, we found out that each ticker has earned a Strong Buy consensus rating from the analyst community. Not to mention all three of them boast massive upside potential.AVEO Pharmaceuticals (AVEO)Hoping to provide better outcomes for patients, AVEO Pharmaceuticals advances targeted medicines for oncology and other unmet medical needs. Following an important regulatory milestone, its no wonder all eyes are on this healthcare name.Griffin is among those singing AVEOs praises. Increasing its holding by a whopping 2,357%, Citadel bought up 383,720 shares in Q2. With the total position now landing at 400,003 shares, it is valued at $1,824,013.H.C. Wainwright analyst Swayampakula Ramakanth reminds investors that on June 1, the FDA accepted the NDA for tivozanib, the company's lead candidate, for review, based on the fact that the TIVO-3 study reported positive final overall survival (OS) data. In the study, AVEOs therapy was compared to sorafenib, marketed as Nexavar by Bayer, for the treatment of advanced renal cell carcinoma (RCC) in the third and fourth-line settings.Looking more closely at the data, which was presented at the ASCO 2020 virtual meeting, the final OS analysis resulted in an overall hazard ratio (HR) of 0.97, which favored tivozanib. Ramakanth was encouraged by the OS results as they suggest tivozanib at least has a similar overall relative risk of deaths compared to sorafenib.Considering that TIVO-3 study met both the primary endpoint of progression free survival (PFS) and the secondary endpoint of overall response rate (ORR), with comparable OS to the active comparator, we believe tivozanib would likely get a green light for the U.S. approval, which could be a major catalyst in the next 12 months, Ramakanth opined.Adding to the good news, the dose escalation for the Phase 1b/2 DEDUCTIVE study, evaluating tivozanib in combination with durvalumab, a monoclonal antibody against PD-L1 marketed as Imfinzi by AstraZeneca in hepatocellular carcinoma (HCC), has been wrapped up, with it progressing to Phase 2. As the CDC estimates about 33,000 patients suffer from liver cancer every year in the U.S., Ramakanth sees an additional opportunity.To this end, Ramakanth rates AVEO a Buy rating along with a $12 price target. Should his thesis play out, a potential twelve-month gain of 163% could be in the cards. (To watch Ramakanths track record, click here)Other analysts dont beg to differ. 3 Buy ratings and no Holds or Sells have been assigned in the last three months. So, the word on the Street is that AVEO is a Strong Buy. The $15 average price target is more aggressive than Ramakanths and implies 229% upside potential. (See AVEO stock analysis on TipRanks)IDEAYA Biosciences (IDYA)Next up we have IDEAYA Biosciences, an oncology-focused precision medicine company that develops targeted therapeutics by using molecular diagnostics. Based on the strength of its technology, this name has scored several fans.Reflecting a new position for Griffin's Citadel, the fund pulled the trigger on 248,005 shares in Q2. As for the value of this holding, it comes in at $2,881,818. Writing for Northland Capital, analyst Tim Chiang believes shares are undervalued based on the future potential of its precision medicine oncology pipeline, which targets specific biomarkers. Expounding on this, he stated, IDEAYA is applying its capabilities across multiple classes of precision medicine, including direct targeting of oncogenic pathways and synthetic lethality which represents an emerging class of precision medicine targets.Part of what makes IDYA a stand-out, in Chiangs opinion, is the fact that its preclinical programs use its synthetic lethality (SL) platform, which targets tumors with MTAP gene deletion and homologous recombination deficiency (HRD) including BRCA mutations.We believe the longer-term upside potential with IDYA shares is significant given the potential utility of SL. The first clinically validated SL gene pair was PARP-BRCA1/2, and based on the efficacy of PARP inhibitors, the SL approach to treating cancer has achieved substantial commercial validation, the analyst explained.To back this up, Chiang points out that several PARP inhibitors have already been approved for the treatment of tumors with BRCA and other DNA damage repair alterations, including ovarian, breast and pancreatic cancers. These inhibitors include AstraZeneca's olaparib, GlaxoSmithKline's niraparib, Pfizer's talazoparib and Clovis rucaparib. He added, We estimate these four drugs generated over $1.6 billion in worldwide sales in 2019 and are expected to reach over $6 billion in sales by 2024.It should be noted that multiple IND filings are set to come within the next 4-12 months, with IDYAs lead SL candidate, IDE397, which was designed to inhibit MTAP and MAT2A and thus cause the death of cancerous tumor cells, entering the clinic in 2021.It should come as no surprise, then, that Chiang joined the bulls. To start off his IDYA coverage, he puts an Outperform rating and $28 price target on the stock. This target implies a possible twelve-month rise of 141% could be on the horizon. (To watch Chiangs track record, click here)Similarly, the rest of the Street is getting onboard. 5 Buy ratings assigned in the last three months add up to a Strong Buy analyst consensus. In addition, the $25.20 average price target puts the potential twelve-month gain at 116%. (See IDYA stock analysis on TipRanks)Ocular Therapeutix (OCUL)Using its patented bioresorbable hydrogel-based formulation technology, Ocular Therapeutix develops cutting-edge therapies for diseases and conditions of the eye. The progress of its clinical programs has caught Wall Streets attention, with some arguing that now is the time to get in on the action.Griffin and Citadel didnt want to miss out on an opportunity. Snapping up 161,032 shares during Q2, the hedge fund gave the holding a 272% boost. The total position is now comprised of 220,269 shares and is valued at $1,718,098.Representing Raymond James, 5-star analyst Dane Leone cites the potential to address the unmet needs in the dry eye disease indication as a key component of his bullish thesis. The company boasts two assets targeting the condition, OTX-CSI (chronic) and OTX-DED (acute). OTX-CSI incorporates the FDA-approved immunomodulator cyclosporine as the active drug in the intracanalicular insert, which is released for an estimated three months to increase tear production.When it comes to OTX-DED, Leone argues OCUL was clever in pursuing the development of OTX-DED (a low dose form of DEXTENZA, a corticosteroid intracanalicular insert placed in the punctum, a natural opening in the eye lid, and into the canaliculus to deliver dexamethasone to the ocular surface for up to 30 days without preservatives) for the treatment of episodic dry eye, as the prior safety data from the DEXTENZA approval enabled management to file a Phase 2-enabling IND by YE20.Speaking to the possible opportunity here, both products could provide revenue generation for physicians in the treatment of dry eyes using procedure CPT code 0356T, which could provide incentive for rapid adoption in the dry eye space that is currently valued at $5.1 billion, in Leones opinion.As for the clinical and regulatory pathway, the Phase 2 trial for OTX-DED is set to kick off in 2H21, slightly after OTX-CSI, which will see a Phase 2 clinical trial evaluating two different formulations initiated by 4Q20. Based on the short treatment duration of OTX-DED, management thinks it will still be brought to market first.To this end, Leone remains optimistic about the companys long-term growth narrative. As a result, he rates OCUL a Strong Buy along with a $15 price target. This figure implies shares could rise 89% in the year ahead. (To watch Leones track record, click here) Turning now to the rest of the Street, other analysts are on the same page. With 100% Street support, or 3 Buy ratings to be exact, the consensus is unanimous: OCUL is a Strong Buy. The $13.50 average price target brings the upside potential to 70%. (See OCUL stock analysis on TipRanks)To find good ideas for stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights.Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

Link:
Global Gene Therapy Market is Forecast to Reach US$ 6.42 billion in 2024, Witnessing Growth at a CAGR of 19.29% Over 2020-2024 - Yahoo Finance

Gyros Protein Technologies AB has announced the introduction of Gyrolab AAVX Titer Kit for adeno-associated virus (AAV) vector titer determination in AAV vector manufacturing. The new Gyrolab AAVX Titer Kit for physical titer determination, one of the critical quality attributes (CQAs) of the FDA process validation guidelines, has a broad applicability across numerous AAV serotypes and adds to the Companys wide range of ready-to-use kits that are already used by scientists and bioengineers in the rapidly growing cell and gene therapy market, where AAV vectors are commonly used.

Gyrolab AAVX Titer Kit supports scientists in the development and production of cell and gene therapies to maximize performance and productivity, and reduces the time needed to produce patient treatments. With compressed development timelines for these therapies, the new kit generates results 4 times faster and requires 10 times less sample compared to traditional ELISA methods, accelerating AAV vector workflows and enabling high quality data to be produced from small sample volumes. Featuring an analytical range of >3 logs, Gyrolab AAVX Titer Kit also minimizes repeat testing compared to ELISA.

Gyrolab AAVX Titer Kit is based on a highly selective AAVX affinity ligand developed with the Thermo Scientific CaptureSelect technology by Thermo Fisher Scientific. These ligands are also the basis of POROS CaptureSelect AAVX Affinity Resin, which is frequently used to purify AAV viral vectors. The AAVX ligand binds with high affinity and selectivity to native and recombinant AAV particles of various serotypes (AAV1 to AAV8, AAVrh10).

Read more:
Gyrolab AAVX Titer Kit Introduced to Support Cell and Gene Therapy Market - Technology Networks

The development prediction report titled Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market 2020 by Company, Regions, Type and Application, Forecast to 2025 focuses on thoughtful insights and facts relating to the market. The report investigates the most recent market patterns such as market development openings, size, share, and drivers. The report throws light on the markets historical data, key vendors, region-wise market, and projections for 2020 to 2025 time-period. Report authors have categorized global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market segments, regions, and product types and distribution channels to provide market analysis and information. All the relevant points of interest market product type, producing price, scope, applications are covered in the report. In the later section, market dynamics are covered including market growth factors, limitations, market opportunities, and challenges are mentioned.

NOTE: Our analysts monitoring the situation across the globe explains that the market will generate remunerative prospects for producers post COVID-19 crisis. The report aims to provide an additional illustration of the latest scenario, economic slowdown, and COVID-19 impact on the overall industry.

Competitive Rivalry:

The report offers a comprehensive study of prime players operating in this global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market by highlighting their product description, business outline, and business strategy. The report conjointly encompasses the amount of production, future demand, and also the health of the organization. Later, the report highlights all the recent developments, product launches, joint ventures, merges, and accusations by the top players. The leading players are also covered with product description, business outline, and production, company profile, product portfolio, product/service price, capacity, sales, and cost.

DOWNLOAD FREE SAMPLE REPORT: https://www.marketsandresearch.biz/sample-request/83503

The report analyses every sub-segment regarding the individual growth trends, contribution to the total market, and the upcoming forecasts. For the forecast period, the report offers estimates of global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market value and volume. The report discusses key drivers, market challenges, cost structure, barriers, technology, regions, and applications. It spotlights on effective manufacturing methods, production volume, manufacturing facilities, capacities, pricing analysis, product description.

Top key players involved in the industry are: BioReliance, Richter-Helm, UniQure, Cobra Biologics, MassBiologics, Oxford BioMedica, Lonza, MolMed, FinVector, FUJIFILM Diosynth Biotechnologies, Brammer Bio, bluebird bio, Aldevron, Spark Therapeutics, VGXI, Biovian, Eurogentec, Novasep, PlasmidFactory, Cell and Gene Therapy Catapult, Vigene Biosciences

Segmentation by type and analysis of the market: AAV, Adenoviral, Lentiviral, Retroviral, Plasmid DNA, Other Vectors

Segmentation by application and analysis of the market: Cancers, Inherited Disorders, Viral Infections, Others

The report offers an exhaustive geographical analysis of the global market, covering important regions such as: North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina), MENA (Saudi Arabia, UAE, Turkey and South Africa)

Moreover, the report analyzes the worlds main regions contributing to the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market development. These regions are assessed the product price, profit, capacity, production, supply, demand, and market growth rate and forecast, etc. Additionally, manufacturing processes, cost structures, import/export consumption, supply and demand figures, cost, price, revenue, and gross margins are analyzed.

ACCESS FULL REPORT: https://www.marketsandresearch.biz/report/83503/global-viral-vectors-non-viral-vectors-and-gene-therapy-manufacturing-market-2020-by-company-regions-type-and-application-forecast-to-2025

Key Attributes of Market Report:

Customization of the Report:

This report can be customized to meet the clients requirements. Please connect with our sales team ([emailprotected]), who will ensure that you get a report that suits your needs. You can also get in touch with our executives on +1-201-465-4211 to share your research requirements.

About Us

Marketsandresearch.biz is a leading global Market Research agency providing expert research solutions, trusted by the best. We understand the importance of knowing what global consumers watch and buy, further using the same to document our distinguished research reports. Marketsandresearch.biz has worldwide presence to facilitate real market intelligence using latest methodology, best-in-class research techniques and cost-effective measures for worlds leading research professionals and agencies. We study consumers in more than 100 countries to give you the most complete view of trends and habits worldwide. Marketsandresearch.biz is a leading provider of Full-Service Research, Global Project Management, Market Research Operations and Online Panel Services.

Contact UsMark StoneHead of Business DevelopmentPhone: +1-201-465-4211Email: [emailprotected]Web: http://www.marketsandresearch.biz

You May Check Also Other Reports

Global Dehydrated Alfalfa Market Research Report With COVID-19 Update

Global Frozen Meat Processing Equipment Market Research Report With COVID-19 Update

Global External Cardiotocography Market Research Report With COVID-19 Update

Global Glutaric Anhydride Market Research Report With COVID-19 Update

Global Infrared Laser Thermometers Market Research Report With COVID-19 Update

Read more:
Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market 2020 Analysis, Types, Applications, Forecast and COVID-19 Impact...

The success of the approved gene therapies has led to an upward surge in the interest of biopharmaceutical developers in this field, resulting in a significant boost in clinical research initiatives and several high value acquisitions

Roots Analysis has announced the addition of Gene Therapy Market (3rd Edition), 2019-2030 report to its list of offerings.

Encouraging clinical results across various metabolic, hematological and ophthalmic disorders have inspired research groups across the world to focus their efforts on the development of novel gene editing therapies. In fact, the gene therapy pipeline has evolved significantly over the past few years, with three products being approved in 2019 alone; namely Beperminogene perplasmid (AnGes), ZOLGENSMA (AveXis) and ZYNTEGLO (bluebird bio). Further, there are multiple pipeline candidates in mid to late-stage (phase II and above) trials that are anticipated to enter the market over the next 5-10 years.

To order this 550+ page report, which features 190+ figures and 355+ tables, please visit this link

Around 470 gene therapies are currently under developmentNearly 45% of pipeline drugs are in the clinical phase, while rest are in the preclinical / discovery stage. Gene augmented therapies presently represent 66% of the total number of such interventions that are in the pipeline. It is worth mentioning that majority of such product candidates are being developed as in vivo gene therapies.

More than 30% of clinical stage pipeline therapies are being designed for treating oncological disordersConsidering the overall pipeline, over 20% of product candidates are being developed to treat various types of cancers, followed by those intended for the treatment of metabolic (15%) and ophthalmic disorders (12%). It is also worth highlighting that adenovirus vectors are presently the preferred vehicles used for the delivery of anticancer gene therapies.

Over 60% of gene therapy developers are based in North AmericaOf the 110 companies developing gene therapies in the abovementioned region, 64 are start-ups, 26 are mid-sized players, while 18 are large and very large companies. Further, within this region, most of the developers are based in the US, which has emerged as a key R&D hub for advanced therapeutic products.

More than 31,000 patents have been filed / published related to gene therapies, since 2016Of these, 17% of patent applications / patents were related to gene editing therapies, while the remaining were associated with gene therapies. Leading assignees, in terms of the size of intellectual property portfolio, include (industry players) Genentech, GSK, Sangamo Therapeutics, Bayer and Novartis, (non-industry players) University of California, Massachusetts Institute of Technology, Harvard College, Stanford University and University of Pennsylvania.

USD 16.5 billion has been invested by both private and public investors, since 2014Around USD 3.3 billion was raised through venture capital financing, representing 20% of the total capital raised by industry players till June 2019. Further, there have been 28 IPOs, accounting for more than USD 2.2 billion in financing of gene therapy related initiatives. These companies have also raised significant capital in secondary offerings.

30+ mergers / acquisitions have been established between 2014 and 2019Examples of high value acquisitions reported in recent past include the acquisition of AveXis by Novartis (2018, USD 8,700 million) and Bioverativ by Sanofi (2018, USD 11,600 million).

North America and Europe are anticipated to capture over 85% of market share by 2030With a promising development pipeline and encouraging clinical results, the market is anticipated to witness an annualized growth rate of over 40% during the next decade. In addition to North America and Europe, the market in China / broader Asia Pacific region is also anticipated to grow at a relatively faster rate.

To request a sample copy / brochure of this report, please visit this link

The USD 10 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom gene therapies are likely to be the most promising treatment options for genetic disorders. The report includes detailed transcripts of discussions held with the following experts:

The research covers brief profiles, featuring an overview of the therapy, current development status and clinical results. Each profile includes information on therapeutic indication, targeted gene, route of administration, special designations, mechanism of action, dosage, patent portfolio, technology portfolio, clinical trials and recent developments (if available).

For additional details, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

or email [emailprotected]

Contact:Gaurav Chaudhary+1 (415) 800 3415+44 (122) 391 1091[emailprotected]

Originally posted here:
Gene Therapy Market is projected to be worth USD 10 Billion by 2030, growing at an annualized rate of over 40% - Scientect

LONDON Gyroscope Therapeutics Ltd. is poised to move the field of ocular gene therapy on from the treatment of inherited rare diseases to address more common eye conditions, after receiving FDA 510(k) clearance for its Orbit SDS subretinal delivery device.

Orbit SDS uses a cannula to deliver a therapeutic payload to the subretinal space, which it accesses via a suprachoroidal approach route. A microneedle inside the cannula enables targeted dose delivery.

That eliminates the need for a vitrectomy, and is both faster, easier and safer than the surgical procedure, removing concerns about gene therapy vectors leaking from the subretinal space.

Although vitrectomy has been used to deliver several gene therapies, it is not practical for treating hundreds of thousands of patients with dry age-related macular degeneration (AMD) and other common eye diseases. FDA 510(k) clearance of Orbit SDS now opens the way to scale up administration of ocular gene therapies to much larger indications.

The FDA clearance creates the potential to speed up development and widen patient access. In addition to a replacing a defective gene with a functional one, it will be possible to expand into the routine delivery of therapeutic proteins.

The company says it will use the device in its own clinical trials and make it available to other companies developing cell and gene therapies for eye diseases.

Charlotte Arnold, vie president of corporate affairs at Stevenage, U.K.-based Gyroscope told BioWorld the company now plans to integrate Orbit SDS into its GT005 program in dry AMD. We anticipate starting to generate data delivering GT005 using the Orbit SDS in a planned future cohort in the ongoing phase I/II Focus trial, she said.

Unlike the wet form of AMD that can be treated with anti-VEGF antibodies to prevent neovascularization, there is no therapy approved for dry AMD, which is both a cause of significant visual impairment and a precursor to wet AMD.

GT005 uses an adeno-associated virus to deliver the gene encoding an endogenous anti-inflammatory factor, to restore the balance of a complement system known to be hyperactivated in AMD. The aim is that a one-off treatment will prevent further degeneration.

The FDA nod is an important component of developing gene therapies to help preserve sight, said Khurem Farooq, CEO of Gyroscope. The Orbit [device] is exquisitely designed to target the subretinal space, with the aim of providing precise and consistent dosing, Farooq said.

The clearance for Orbit SDS rests on a phase IIb study of palucorcel (CNTO-2476), in the treatment of geographic atrophy of age-related AMD, an advanced stage of AMD, characterized by progressive loss of retinal pigment epithelium. That leads to loss of photoreceptors, resulting in visual disturbance and difficulty adjusting to low light conditions. Central vision is lost progressively.

The Orbit device was used in the phase I/II trial after initial studies in which palucorel was administered by retinotomy showed there was a risk of retinal detachment. A further study used subretinal delivery, but the technique was judged too technically difficult to take forward.

That led on to the development of Orbit SDS, which was purpose designed for subretinal delivery.

In the 21-patient phase I/II palucorel study the investigators reported that administration to the subretinal site was achieved successfully in most patients and [was] not associated with any moderate or severe, or serious ocular adverse events. The investigators were unable to deliver cells in 3 of 21 participants. Most adverse events were mild and resolved within one month.

Vitrectomy can be associated with an increase in intraocular pressure and accelerated formation of cataracts. No such effects were seen in the Orbit SDS study.

Palucorel is a cell therapy derived from human umbilical tissue that expresses anti-inflammatory factors including interleukin-6, brain-derived growth factor, fibroblast growth factors, and anti-angiogenic thrombospondins. While it showed positive effects in two earlier trials, there were no significant improvements in vision in the Orbit SDS-administered trial.

However, the investigators say, This first in human trial of a suprachoroidal approach for delivery of subretinal liquids was successful from a surgical perspective and clearly demonstrates that subretinal delivery via the suprachoroidal space is feasible.

Gyroscope acquired the Orbit SDA device when it merged with its developer, Orbit Biomedical of Philadelphia in April 2019, to become a fully integrated retinal gene therapy company, with clinical, manufacturing and delivery capabilities.

Orbit Biomedical, now renamed Gyroscope USA Inc., is an ISO 13485 medical device company certified to design, manufacture and distribute ophthalmic surgical instruments.

Following on from the FDA clearance, Arnold said Gyroscope is pursuing a CE mark in Europe for Orbit SDS as well. We also plan to enter into licensing and collaboration arrangements for use of the Orbit SDS by other companies for delivery of gene and cell therapies being developed to treat eye diseases, she said.

Read the original:
Gyroscope Therapeutics FDA clearance puts subretinal delivery device in Orbit - BioWorld Online

Non-Hodgkin lymphoma (NHL) is among the most common cancers in the United States. In 2020, an estimated 77,240 Americans will be diagnosed with the disease and about 20,000 people will die from it.1 Seventy-two percent of patients live for at least 5 years after diagnosis.2 However, the success of treatment varies widely across the many subtypes of NHL. Patients diagnosed with diffuse large B-cell lymphoma (DLBCL), the most common subtype, have a 5-year survival rate of 63% for all disease stages combined. By contrast, follicular lymphoma (FL) has a 5-year survival rate of 88% for all stages combined. Patients with mantle cell lymphoma, one of the more difficult-to-treat subtypes, have an average survival time of less than 5 years.3

But the treatment landscape is transforming across the entire spectrum of NHL. Novel immunotherapies and small molecule inhibitors are offering both previously treated and untreated patients entirely new options and new combinations. At the American Society of Clinical Oncology (ASCO) 2020 Virtual Scientific Program, researchers led by Jeremy Abramson, of Massachusetts General Hospital in Boston, provided a compelling overview of these new and upcoming treatments.4 Their overview, published in the ASCO Educational Book, offered a detailed and vital look at the present and future of NHL treatment.

CAR T-cell immunotherapy has been hailed as a major game-changer for some blood cancers.5 For aggressive B-cell lymphomas, such as DLBCL, anti-CD19 chimeric antigen receptor T cells (CAR-T) are a new option for patients who have relapsed following chemoimmunotherapy or autologous stem cell transplant (ASCT). For this patient group, which has a median overall survival of about 4 months, anti-CD19 CAR-T agents have elicited durable remissions in about 40% of patients.5-7 Clinical trial data has led to the approval of axicabtagene, ciloleucel, and tisagenlecleucel by both the US Food and Drug Administration and the European Medicine Agency. CAR T-cells offer curative intent therapy to patients with relapsed DLBCL who are not eligible for stem cell transplant, Dr Abramson told Cancer Therapy Advisor. Previously these patients only had palliative options available.

Some clinicians envision CAR-T as a potential first-line therapy for patients with DLBCL who relapsed after first-line chemoimmunotherapy. These patients, noted Anton Hagenbeek, MD, PhD, professor of Hematology at Amsterdam University Medical Centers, who was not involved with the review paper, represent one of the highest unmet needs in the treatment of lymphoma to date. And Helen Heslop, MD, who directs the Center for Cell and Gene Therapy at Baylor College of Medicine in Houston, Texas, noted that trials for first-line CAR-T therapy are already underway in acute lymphoblastic leukemia. Not everyone sees this potential. First-line treatment in NHL is generally very effective and much less expensive than CAR T-cells, explained Edward Copelan, MD, who chairs the Department of Hematologic Oncology and Blood Disorders at Carolinas Healthcare System in Charlotte, North Carolina, and who was not an author of the review. Though Dr Copelan emphasized that patients at extremely high risk of relapse following standard immunochemotherapy may respond well to CAR-T therapy.

Dr Abramson and co-authors highlighted a difficult conundrum with regard to DLBCL. Although the addition of rituximab to cyclophosphamide, doxorubicin, hydrochloride, and vincristine sulfate (CHOP) chemotherapy is curative for most patients, the number of patients who may be cured after relapse has declined. Thus improvements in second-line therapy are desperately needed. Several clinical trials are currently evaluating anti CD19 CAR-T for primary refractory or early relapsed aggressive B-cell lymphoma compared with traditional salvage therapy or ASCT. The same approach is being studied for relapsed DLBCL patients who are not transplant candidates.

Whether CAR-T therapy will find a place on the first line of care is another current question. The cost and logistics of CAR-T therapy make it unlikely that this approach will replace R-CHOP as the standard initial treatment, Dr Abramson and colleagues noted. I do not think CAR T-cells will replace frontline chemotherapy, Dr Abramson said. However, the authors note that for patients who do not respond well to initial treatment, such an approach may make sense a possibility that is now being examined in the clinical trial setting. Dr Hagenbeek is more certain about the first-line role for CAR-T therapy, based on the dismal prognosis of DLBCL that is refractory to R-CHOP.

On the subject of CAR-T therapy as first-line therapy, the authors pay special attention to so-called double-hit lymphomas (DHLs), also known as double expressor lymphomas. According to a multicenter study published in 2017, R-CHOP is curative for more patients than conventional wisdom has held.9 Clinical trials are currently investigating the use of anti-CD19 CAR-T early for patients who consistently test positive for disease on PET scans during initial therapy, though Dr Abramson and colleagues call for caution with this approach because the prognostic value of PET/CT scans is controversial. Dr Hagenbeek is optimistic about the potential for CAR-T therapy to improve the prognosis for patients with double- and even triple-hit lymphomas, if applied in the first complete, PET-negative metabolic remission. Because these patients have a relatively small tumor load, this approach could, said Dr. Hagenbeek, completely eradicate minimal residual disease. Its the double-hit patients who Dr Copelan sees as the likeliest candidates for first-line CAR T-cell treatment.

Read more here:
Targeted Therapy for Non-Hodgkin Lymphoma: Current Progress and Future Plans - Cancer Therapy Advisor

Data on all eight patients demonstrate sustained engraftment and supranormal IDUA enzyme expression

Translation of metabolic correction to clinical outcomes in first two patients continues to support potential of hematopoietic stem cell gene therapy in a second neurometabolic disorder

Data support planned initiation of registrational trial in 2021

BOSTON and LONDON, Sept. 01, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics(Nasdaq: ORTX), a global gene therapy leader, today announced additional interim data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203, an investigationalex vivoautologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at theSan Raffaele Telethon Institute for Gene Therapy(SR-Tiget) inMilan, Italy. The readout from the primary endpoint at one year of follow-up is expected in 2021. Today's results are being shared virtually in an invited oral presentation at the 46th Annual Meeting of the European Society for Blood and Bone Marrow Transplantation (EBMT).

We continue to see encouraging data from the ongoing clinical trial in MPS-I, including promising preliminary clinical effects on motor development, acquisition of cognitive skilIs and growth in the first two patients that were treated now 1.5 and 2 years ago, respectively. Additionally, new preliminary analyses of radiological outcome measures suggest that treatment with OTL-203 leads to stabilization or improvement in disease-related neurological abnormalities, as measured by brain and spine MRI, which we look to confirm with longer follow-up, saidMaria Ester Bernardo, M.D., Ph.D., principal investigator at SR-Tiget. "These data, taken together with those from clinical studies of HSC gene therapy for other metabolic disorders and leukodystrophies, support the potential for this therapeutic approach to correct a wide spectrum of multisystemic manifestations of the disease, bringing clinically meaningful benefits for patients.

Interim Study Results

Eight patients with the severe Hurler subtype of MPS-I had been treated with OTL-203 in the ongoing proof-of-concept study, which completed enrollment in December 2019. As of July 2020, all patients had been followed for a minimum of six months, with the longest follow-up extending out to 24 months. Treatment with OTL-203 was generally well-tolerated with a safety profile consistent with the selected conditioning regimen. Consistent with previous analyses, treatment across all eight patients continued to demonstrate:

We continue to see positive trends in all biomarker and clinical measures as we follow patients in the OTL-203 proof of concept study for longer periods of time, saidBobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With a growing amount of data to support advancing this program, we have recently convened a panel of disease experts to develop a design for a registrational trial that we intend to take to the regulators in advance of initiating the study in 2021 and ultimately progressing towards commercialization.

About OTL-203 and MPS-I

Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (also known as GAGs). The accumulation of GAGs across multiple organ systems results in symptoms including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births. There are three subtypes of MPS-I; approximately 60 percent of children born with MPS-I have the most severe subtype, called Hurler syndrome, and rarely live past the age of 10 when untreated.

Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an investigationalex vivoautologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by theSan Raffaele Telethon Institute for Gene TherapyinMilan, Italy.

About Orchard

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

Orchard has its global headquarters inLondonandU.S.headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us onTwitterandLinkedIn.

Availability of Other Information About Orchard

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

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, the therapeutic potential of Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding the timing of clinical trials for its product candidates, including the product candidates referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs; the risk that Orchard will not realize the anticipated benefits of its new strategic plan or the expected cash savings associated with such plan; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be successfully developed, approved or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates or that long-term adverse safety findings may be discovered; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedJune 30, 2020, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

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

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

See the rest here:
Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I - GlobeNewswire

The company is to invest 3.4m (around US$4.5m) alongside the grant.

The funds will support research into the manufacturing challenges associated with scaling gene therapies for widespread patient access, to further develop technologies to improve the safety and efficacy of current therapies, and to enable the treatment of genetic diseases with more complicated disease pathways the industry is not yet able to address.

Along with the creation of 11 new jobs in Edinburgh, the developer said it will further enhance its Pro10 platform, an AAV manufacturing process that can be scaled and applied throughout the group.

The grant will also advance development of the tool kit of inducible, repressible, tunable and responsive expression cassettes to be adopted in the current clinical pipeline and new disease targets.

Gene therapy has the potential to treat a wide range of diseases including certain forms of muscular dystrophy, congestive heart failure and some diseases of the central nervous system but, at present, only two market-approved therapies are available.

David Venables, president, AskBio Europe, commented: The grant awarded by Scottish Enterprise supports AskBio in working towards developing even safer and more effective gene therapies through improved development and manufacturing techniques. Science and innovation keep progressing, and that makes this an exciting time to develop this type of therapeutic agent.

AskBios technology is inside both currently approved AAV gene therapies, which include Luxturna, developed by Spark Therapeutics, for the treatment of patients with inherited retinal disease, and Zolgensma, developed by AveXis, for the treatment of patients with spinal muscular atrophy (SMA).

AveXis licenses AskBios self-complementary DNA technology for Zolgensma.

While the promise of such therapies is being shown, significant barriers remain before gene therapies can become more broadly impactful, according to AskBio.

With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, AskBio has generated hundreds of proprietary third generation AAV capsids and promoters, several of which have entered clinical testing.

BioPharma-Reporter (BPR) spoke to Ken Macnamara, (KM), PhD, chief operating officer, AskBio Europe,to get the AAV developers take on the factors preventing gene therapy going mainstream.

BPR: What criteria did AskBio have to fulfill to be awarded this grant?

KM: The research must be highly novel with significant risk from which a successful outcome will accelerate business growth within Scotland and globally.

BPR: What are the current manufacturing challenges associated with scaling gene therapies for widespread patient access?

KM: As we see growing evidence that gene therapy is a viable, transformational medicine, along with an acceleration in the number of AAV therapeutics moving towards regulatory approval, the ability to manufacture these therapies for diseases with large patient populations does not exist today and costs are extremely high.

Many companies can manufacture small batches of therapeutics for clinical applications, but as they approach commercialization, the challenges of production costs and timelines remain an issue. We recognized this more than a decade ago and focused on creating robust, scalable manufacturing capabilities.

Today, the challenges for manufacturing gene therapy are being met by simply adding large amounts of capacity, which is not the long-term answer. There is a significant amount of innovation taking place that will no doubt shape the future of manufacturing AAV gene therapeutics. This work continues today in our Edinburgh and US facilities to further improve the technology.

BPR: What are some of the typical safety and efficacy issues linked to current therapies?

KM: Currently approved gene therapies have provided effective therapy by targeting tissues in the body with an administered gene that produces a new, effective protein. This new gene replaces the defective or missing gene causing the patients underlying disease.

Because the techniques are relatively new, some of the risks may be unpredictable; however, medical researchers, institutions, and regulatory agencies are working to ensure that gene therapy research is as safe as possible.

AAV is not known to cause human disease, and it cannot make more of itself without outside help, so it will not replicate in the body like normal viruses do. AAV is engineered to carry therapeutic genes by removing some of its genetic cargo and replacing it with human gene sequences. This results in an AAV vector, a therapeutic genetic medicine.

Risks associated with AAV gene therapy vector administration include unwanted immune system reactions. The body's immune system may see the newly introduced AAV vectors as intruders and attack them, which may cause inflammation and, in severe cases could be local and mild or throughout a greater area of the body and be more serious. AAV vectors can also target tissues other than the intended tissue. Thus, it's possible that AAV vectors may affect additional cells, not just the targeted cells containing mutated genes. These are called off-target effects. If this happens, healthy cells may be damaged.

BPR: Can you indicate the other significant barriers that remain before gene therapies can become more broadly impactful?

KM: Therapies need to express the gene in the right tissue, at the right level, for the right amount of time. There is a great deal of research happening throughout the gene therapy field to identify the best means of delivering and controlling activation of the genetic material. Furthermore, the response of the patients immune system also needs to be considered based on the therapy. Additional funding, like that from Scottish Enterprise, can help speed up the development process of promising therapies.

BPR: How does AskBio envisage exploring the treatment of genetic diseases with more complicated disease pathways that the industry is not yet able to address?

KM: One of the most exciting advances in modern medicine has been the discovery of how AAV vectors can be used as an effective delivery system for therapeutic genetic material into living tissue. AAV gene therapy has broad therapeutic implications for a vast array of diseases.

Some genetic diseases are caused by mutations in a single gene, while others are a result of mutations in multiple genes, for example, cancer. Additionally, environmental factors, such as smoking and diet, can play a role in diseases. The complexity of these disease characteristics creates variables in developing and testing potential treatments. Currently the gene and cell therapy options that exist today are limited to treating diseases caused by a single gene mutation.

AskBios Edinburgh team leads the gene therapy field in the design and development of synthetic gene expression cassettes. The technology is essential for controlling the expression of AAV therapeutics, thereby improving their safety and efficacy. This R&D project will enable AAV therapeutics to be turned on and off and dialed up or down depending on the amount of drug needed at any given time. This technology provides a desired safety switch and level of variable dosing that previously did not exist. Before this breakthrough, AAV therapeutics could only express at one constant level and could not be turned off, which limited the type of therapeutics for which AAV could be used and may hold the key to treating pathway diseases where multiple genes are affected.

BPR: On the job creation front, is the talent already hired or are you starting a recruitment drive?

KM: The grant allows us to make some positions permanent and bring in new talent.

Ken Macnamara joined AskBio in 2019 with a wealth of R&D, business operations, financial planning, intellectual property and quality/compliance experience gained from start-up to multinational firms. He most recently was COO at Synpromics.

Dr Macnamara began his career at the University of Edinburgh, where he earned a PhD in chemistry before helping to start Lab901 (Scottish SME). There, he was a product development manager responsible for developing the TapeStation and ScreenTape technologies from concept to market success. Lab901 was acquired by Agilent Technologies in 2011. Dr Macnamara then served as R&D director for the Microfluidics business at Agilent.

Read more:
Reducing barriers to mainstream gene therapy - BioPharma-Reporter.com

Paula Cannon. (USC Photo/Richard Carrasco)

An HIV research program led by scientists at USC and the Fred Hutchinson Cancer Research Center in Seattle has received a five-year, $14.6million grant from the National Institutes of Health. The team is advancing a gene therapy approach to control the virus without the need for daily medicines.

The programs co-directors are Paula Cannon, PhD, Distinguished Professor of Molecular Microbiology and Immunology at the Keck School of Medicine of USC, and Hans-Peter Kiem, MD, PhD, the Stephanus Family Endowed Chair for Cell and Gene Therapy at Fred Hutch. Other key partners are David Scadden, MD, a professor at Harvard University, and the biotechnology company Magenta Therapeutics.

The NIH award will support preclinical studies that combine gene editing against HIV with technologies for safer and more effective hematopoietic stem cell transplants. Such transplants, also known as bone marrow transplants, are currently used for severe blood cancers. They renew a patients immune system, which can be damaged by cancer therapies, by infusing healthy donor blood stem cells that can grow into any type of blood or immune cell.

The researchers goal is to build a therapy that prepares patients for a stem cell transplantation using their own cells with little to no toxicity, engineers their own stem cells to fight HIV and stimulates those cells to quickly produce new and engineered immune cells once theyre reintroduced into the patient.

This grant funds a team with an overarching goal of developing what our perfect HIV gene therapy would look like, Cannon said. All of these pieces could happen separately, but the fact that the NIH has funded us as a team means that the sum will be so much bigger than the parts.

Halting HIV without daily drugs

About 38million people worldwide are living with HIV, the virus that causes AIDS. HIV is manageable with daily antiretroviral drugs, but the research team seeks a more durable solution.

Their strategy is inspired by the three cases where patients seem to have been cured of HIV. All had aggressive leukemia and received blood stem cell transplants from donors who also carried a mutation that confers immunity to HIV. The mutation was in the CCR5 gene, which encodes a receptor that HIV uses to infect immune cells and is present in about 1 percent of the population.

Timothy Ray Brown, famously nicknamed the Berlin patient, received such a transplant in 2007; he has been off antiretroviral drugs since then, and the virus remains undetectable in his system. In recent years, patients in London and Dusseldorf have shown similar results.

I think of the Berlin patient as proof of principle that replacing the immune system with one thats HIV-resistant by removing CCR5 is a possible way to treat somebody, Cannon said.

However, the rigors of the blood stem cell transplant process, combined with the difficulty in finding tissue-matched CCR5-negative donors, make it highly unlikely that this will provide more than a very rare treatment.

Three for one gene therapy

The research team will tackle these two major problems. First, to get around the lack of CCR5-negative donors, Cannon has already helped pioneer the use of gene editing to remove CCR5 from a patients own stem cells. This is now an investigational treatment for HIV in a clinical trial at City of Hope in Duarte, California.

She will now combine CCR5 disruption with additional genetic changes, so that the progeny of engineered stem cells will release antibodies and antibody-like molecules that block HIV.

Our engineered cells will be good neighbors, Cannon said. They secrete these protective molecules so that other cells, even if they arent engineered to be CCR5-negative, have some chance of being protected.

Meanwhile, Kiems group is providing a third approach by adapting an emerging cancer treatment called CAR T cell therapy. This re-engineers T cells of the immune system with chimeric antigen receptors (CARs), which are customized to recognize cancer cells.

In this project, Kiem and colleagues will create stem cells whose T cell descendants can instead hunt down HIV-infected cells.

A gentler blood stem cell transplant

The grant also supports two other components that relate to the blood stem cell transplant.

Magenta Therapeutics is developing less-toxic protocols for conditioningpreparing a patients bone marrow to receive a transplant. Traditionally, mild chemotherapy or radiotherapy is needed to make room for newly infused stem cells and to help them re-engraft.

The company is instead using antibody-drug conjugates to deliver this conditioning much more narrowly and to reduce the side effects that occur with systemic chemo or radiation.

Meanwhile, Scadden and his team are addressing another drawback of stem cell transplants and conditioning, the delay before infused stem cells generate new immune cells in sufficient numbers. In cancer patients, this delay leaves them highly susceptible to infection.

Scadden is approaching this using an injectable gel that biochemically resembles the bone marrow environment, to quickly repopulate the immune system with HIV-fighting cells.

With success, the teams research may free HIV patients from the need for daily medication and the expense and potential side effects that come with it. Their work may also improve other therapies based on blood stem cells, for conditions such as cancer, sickle cell disease and autoimmune disorders.

A home run would be that we completely cure people of HIV, Cannon said. What Id be fine with is the idea that somebody no longer needs to take anti-HIV drugs every day because their immune system is keeping the virus under control, so that it no longer causes health problems and, importantly, they cant transmit it to anybody else.

By Wayne Lewis

The rest is here:
Gene therapy research for HIV awarded $14.6 million NIH grant - USC News

Could gene therapy provide a solution to HIV? A new research project aims to find out.

The National Institutes of Health(NIH) has backed researchers at the University of Southern California and the Fred Hutchison Cancer Center with a five-year, $14.6 million grant to develop a gene therapy that could potentially control HIV without the need for daily medications. Most HIV patients take a well-regimented cocktail of medications each day to control the virus. This therapy could change that. According to an announcement from the Keck School of Medicine at USC, the goal will be to develop a therapy that prepares patients for a stem cell transplantation using their own cells with little to no toxicity, engineers their own stem cells to fight HIV and stimulates those cells to quickly produce new and engineered immune cells once they're reintroduced into the patient. The hematopoietic stem cell transplants, also known as bone marrow transplants, have been used to treat some blood cancers. The idea is to infuse an HIV patient withhealthy donor blood stem cells that can grow into any type of blood or immune cell.

The gene therapy strategy has been inspired by three cases where leukemia patients who also had HIV received blood stem cell transplants from donors who also carried a mutation that confers immunity to HIV. The mutation was in the CCR5 gene, which encodes a receptor that HIV uses to infect immune cells and is present in about 1 percent of the population, USC said.

The program will engineer blood cells to remove CCR5 from a patient's own stem cells.That will be combined with other genetic changes so that the progeny of engineered stem cells will release antibodies and antibody-like molecules that block HIV.

In addition to the potential gene therapy treatment, researchers are also assessing whether or not CAR-T treatments will benefit HIV patients. Researchers from Harvard University developed a Dual CAR T-cell immunotherapy that can potentially help fight HIV infection. First reported by Drug Target Review, the HIV-specific CAR-T cell is being developed to not only target and eliminated HIV-infected cells, but also reproduce in vivo to enable the patients to fight off the infection. HIVs primary target it T cells, which are part of the bodys natural immune response.

Todd Allen, a professor of Medicine at Harvard Medical School, said the Dual CAR-T cell immunotherapy has so far provided a strong, long-lasting response against HIV-infection while being resistant to the virus itself.

According to the report, theDual CAR T cell was developed through the engineering of two CARs into a single T cell. Each of the CARs contained a CD4 protein that allowed it to target HIV-infected cells and a costimulatory domain, which signaled the CAR T cell to increase its immune functions. As DTR reported, the first CAR contained the 4-1BB co-stimulatory domain, which stimulates cell proliferation and persistence, while the second has the CD28 co-stimulatory domain, which increases its ability to kill infected cells.

To protect the CAR-T cells from HIV, the team added the protein C34-CXCR4, which prevents HIV from attaching to and infecting cells. When that was added, the researchers found in animal models that the treatment was long-lived, replicated in response to HIV infection, killed infected cells effectively and was partially resistant to HIV infection.

Still, other researchers are looking to those rare individuals who are infected with HIV but somehow on their own are able to suppress the virus without the need for any treatment. Researchers have sought to replicate what this small percentage of patients can naturally do in other patients who require those daily regimens of medications. Through the sequencing of the genetic material of those rare individuals, researchers made an interesting discovery.

The team discovered large numbers of intact viral sequences in the elite controllers chromosomes. But in this group, the genetic material was restricted to inactive regions, where DNA is not transcribed into RNA to make proteins, MedNewsToday reported.

Now the race is on to determine how this can be replicated and used to treat the nearly 38 million people across the globe who have been diagnosed with HIV.

The rest is here:
New HIV Gene Therapy, CAR-T Treatments Could be on the Horizon for Patients - BioSpace

As many as 40,000 people in Germany suffer from retinitis pigmentosa. This hereditary disorder is characterized by loss of photoreceptors in the retina, and can be caused by mutations in many different genes. Depending on the nature of the underlying genetic defect, the severity of the condition can vary between night blindness and progressive visual field loss that can ultimately result in total blindness. The first gene therapies for the disease have recently been approved. However, these approaches have certain disadvantages, which limit their range of application.

A research team led by PD Dr.Elvir Becirovicat the Department of Pharmacology of Natural Sciences (Head: Prof. Dr.Martin Biel) has developed a new strategy in collaboration with Prof. Dr.Stylianos Michalakisof the Opthalmology Clinic in the LMU Medical Center. This approach is designed to compensate for the causative hereditary defect by activating genes with similar functions that are normally repressed in the affected tissues, and utilizes a variant of the CRISPR/Cas9 technology that was first described in 2015. In the online journalScience Advances, the team describes the first successful application of this method in the context of gene therapy.

Currently, two strategies are being used in the development of gene therapies: In the context of gene supplementation, an attempt is made to replace the defective gene with an intact version. However, this is currently only possible for relatively small genes. The second strategy aims to correct disease-causing mutations, but this usually has to be tailored to each individual mutation. In view of the high effort and the associated development costs, a broad application of this strategy is therefore not possible. "To overcome these limitations, we have developed a new strategy," says Becirovic.

Many genes in the human genome fall into families, whose members fulfill similar functions in different cell types, or are activated at different stages during the differentiation of a particular cell type. Our idea was to compensate for the mutant genes loss of function by specifically activating genes that have a similar function but are normally not expressed in retinal cells, says Becirovic. To do so, we delivered a system called Cas9-VPR into the affected retinal cells. The Cas9-VPR system is a derivative of the CRISPR/Cas9 technology that is widely used for the targeted modification of genes. Akin to the classical CRISPR/Cas9 system, Cas9-VPR utilizes the same targeting principle to guide an activating protein to the particular gene of interest.

Becirovic and colleagues made use of a mouse model for retinitis pigmentosa to test the activation approach. These mice lack the light-sensitive rhodopsin protein that is normally expressed exclusively in the rod cells of the retina, which are required for dim light and night vision. The researchers delivered the Cas9-VPR system into the rod cells with the aid of a harmless virus. By introducing Cas9-VPR into the rods of the mice, the scientists switched on genes closely related to the rhodopsin gene, which are normally active in the cones responsible for color and daylight vision. In this way, we were able to compensate for the lack of rhodopsin function in the rod cells, to attenuate the rate of retinal degeneration and improve retinal function without detectable side-effects, says Becirovic.

The authors believe that a similar strategy can be applied to a wide range of genes and genetic diseases, and offers a number of significant advantages over existing strategies. Given the growing importance of gene therapy and its potential benefits for patients, we are convinced that our approach could soon be used in initial clinical feasibility studies, says Becirovic.

Reference: Bhm S, Splith V, Riedmayr LM, et al. A gene therapy for inherited blindness using dCas9-VPRmediated transcriptional activation. Sci Adv. 2020;6(34):eaba5614. doi:10.1126/sciadv.aba5614.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

See more here:
A New Target for Congenital Blindness Gene Therapy Is in Sight - Technology Networks

For the first time ever, a team of neurologists and neurosurgeons atThe Ohio State University Wexner Medical CenterandThe Ohio State College of Medicinehas performed a novel gene therapy brain infusion to treat a patient with Parkinsons disease.

This multicenter, Phase 1bclinical safety studyis sponsored byBrain Neurotherapy Bio, Inc.and funded by theCalifornia Institute for Regenerative Medicineto test GDNF gene therapy in patients with early to moderate stages of Parkinsons disease. The one-time treatment involves infusion of a gene therapy solution into deep structures of the brain that are affected by the disease.

Parkinsons disease is a neurodegenerative movement disorder that affects one million people in the United States. Degeneration of neural pathways deep in the brain causes symptoms such as tremor, slow movement and behavioral abnormalities, said Ohio State neurosurgeon Dr. James Brad Elder who performed the gene therapy surgery on Aug. 25.

The overall goal of this gene therapy treatment strategy is to slow the neurologic deterioration associated with Parkinsons disease by enhancing levels of a naturally occurring growth factor called GDNF. Targeting gene therapy delivery to the putamen, a deep brain structure affected by Parkinsons disease, will hopefully improve overall quality of life, Elder said.

The patient, a 55-year-old Ohio man first diagnosed with Parkinsons disease in 2008, takes medicine to help control his progressively worsening disease. He said the gene therapy surgery gives him hope that his disease wont get any worse, and that he may even feel better without medications. But he added that it could take up to six months before he notices any improvements.

There has long been evidence in animal and cell culture models of Parkinson's disease suggesting that glia cell derived neurotrophic factor (GDNF) has promise as a therapy for the disease, said Dr. Sandra Kostyk, director of theMovement Disorders Divisionat Ohio State Wexner Medical Center. Patients with Parkinsons disease and related disorders are diagnosed and treated in the Movement Disorders clinics and neurosurgery programs at Ohio State.

One of the biggest clinical hurdles has been getting the molecule to the regions in the brain that would benefit these patients the most. GDNF is a relatively large molecule that cant be administered as a pill, nor intravenously, since it cant penetrate the blood brain barrier.

This new targeted gene delivery approach overcomes many of the obstacles that have slowed GDNF clinical trial research and is expected to facilitate the production of a continuous supply of GDNF to a critical region of the brain affected by Parkinsons disease.

This is a onetime treatment strategy that could have ongoing lifelong benefits. Though its hoped that this treatment will slow disease progression, we dont expect this strategy to completely stop or cure all aspects of the disease.Were cautiously optimistic as this research effort moves forward, Kostyk said.

Brain Neurotherapy Bio is a biotechnology startup company founded in 2018 byDr. Krystof Bankiewiczto develop gene therapies for neurological disorders. Bankiewicz is also a member of Ohio State Wexner Medical CentersNeurological Institute.

Ive been investigating therapeutic gene therapy approaches for Parkinsons disease for nearly 30 years, and this marks a significant milestone that may lead to major therapeutic opportunities for those suffering with this devastating condition, said Bankiewicz, who is CEO and chairman of the board of Brain Neurotherapy Bio.

Additional sites for this clinical trial include the University of California San Francisco and the University of California Irvine medical centers. For more information, emailOSUgenetherapyresearch@osumc.edu.

Read the original post:
Wexner Medical Center performs gene therapy brain infusion for Parkinson's disease - The Highland County Press