Archive for the ‘Gene Therapy Research’ Category

The first six months of his young life have been a roller-coaster but Mighty Hudson Cowie is back home in Alberta after an experimental gene therapy procedure in Tennessee was successful.

Its been so long since weve got to see our family, our friends, Hudsons dad Ian said. Even just to be able to show him off to everybody. Weve spent the last six months seeing how amazing of a little guy he is.

Now we finally get to share that with everybody.

Hudson was diagnosed with Severe Combined Immunodeficiency (SCID) within days of his birth on June 23. The condition, known to many as Bubble Boy Disease, prevents his body from fighting illnesses. Essentially, he didnt have an immune system.

READ MORE:Hundreds attend donor drive for Mighty Hudson, Alberta baby with rare immune disease

In August, Hudson was accepted into a gene therapy program at St. Jude Childrens Research Hospital in Memphis. A medical team removed some of his bone marrow cells and replaced the faulty gene with a corrected one. The cells were placed back into his body through an IV.

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They took his own bone marrow out in his bone marrow, hes got hematopoietic stem cells. They were able to take those cells and modify them to create a product, a repaired version of those cells, and then give them back to him, Ian told Global News on Monday.

Medicine is amazing. We took a chance on science and were so glad that we did. Its amazing what theyre able to do.

Ian and his wife Hayley were weighing two options for their little boy: using donor cells or a revamped version of Hudsons own cells. They decided on the second, which meant a trial at St. Judes.

We weighed the pros and cons of both options and for us, [and] personally, we decided that gene therapy seemed like the safer way to go, Ian said.

It didnt rely on a donor, it was the new up-and-coming medicine, the existing results for gene therapy were already incredibly promising and then, as an added bonus, it had substantially less chemotherapy requirements.

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WATCH: (Sept. 9, 2019) A Morinville baby with a rare disorder received a life-changing procedure in the U.S. Hudson Cowies parents give Su-Ling Goh an update on therapy for his immune system.

It honestly sounded too good to be true, Hayley added.

With bone marrow transplant, we heard of graft versus host disease, where its somebody elses cells and they can reject them. With gene therapy, having his own cells, he wasnt going to reject them because his body already knows them.

A few months after the transplant, they started to see results: Hudsons first T-cells, a type of white blood cell thats a key component of the immune system.

From three months, it exploded. At four months, it was even more T-cells. The growth was exponential, Ian said.

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I think thats the thing every doctor hopes to get to say to their patients one day: Hes cured. And the day that we left Memphis, she said that. She said: Hes cured.

We squealed like little schoolgirls. It was very exciting.

Hudsons immune system right now can be compared to that of a newborn baby.

As hes introduced to small bugs, itll just keep growing and growing and be normal, Hayley explained.

There are still unknowns, but the family is very hopeful.

He was Patient 12 on the trial and from what we were informed, everyone on the trial has done tremendously well, Ian said.

I just think its amazing, Hayley added. Its amazing that we were the first ones who got on newborn screening and that this trial was even available for us. It just seems that everything completely lined up.

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I feel like everything has just fallen into place to get him cured.

The treatment is just amazing. Our doctors are brilliant, its just all incredible and hes doing great.

Hudson was one of the first babies to be screened for SCID through a new program. Since SCID was added in May 2019, the Alberta Health Services Newborn Metabolic Screening Program has screened over 34,000 newborns and has diagnosed four cases of SCID.

While very much welcome, being home is still a big change for the Cowies.

Im still processing, even now, Hayley said. Its nice to go see people but we were in isolation for so long that it doesnt just flip all of a sudden. I know were still really protective and we can hear a cough from miles away.

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It was surreal to leave but I feel like Im still dreaming a little bit.

They havent attempted any big outings yet especially given its flu season but they have been able to go to some family dinners.

Its an adjustment for sure, but a good adjustment, Hayley said. He loves people Hes always just smiling and playful hes just happy.

The Cowies will be back in Tennessee for one day for a checkup this weekend. Hudson will have another checkup with the St. Judes team next month. Hell have followups at least once a year until hes 10 years old.

2020 Global News, a division of Corus Entertainment Inc.

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Alberta baby Mighty Hudson home after gene therapy: hes cured - Global News

Meet Helen Obando, a shy 16-year-old who likes to dance when her body isnt ravaged by the debilitating symptoms of sickle cell disease. The genetic blood disorder can cause strokes, organ damage and intense pain.

After a lifetime of pain and potential permanent damage to her body, Helen had the opportunity to receive a breakthrough experimental treatment at Boston Childrens Hospital that would make her the youngest person in the U.S. to have her DNA reset in an attempt to cure her sickle cell disease.

The outcome of her gene therapy could help determine how an estimated 100,000 people in the U.S. and millions more around the world are treated. Sickle cell disease most commonly affects people from sub-Saharan Africa, and about 1 in 500 African-Americans have the blood disorder, the most commonly inherited blood disorder in the U.S. But some people with the disease have southern European, Middle Eastern or Asian backgrounds or, like Helen, are Hispanic. For decades, attention and money for research have not matched the scale of the problem.

Why has it taken so long for the scientific research community to push ahead with promising therapies for sickle cell?

Read full, original post: A Teenagers Breakthrough Gene Therapy for Sickle Cell Disease

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Teenager's experimental gene therapy treatment could change the lives of millions of sickle cell patients worldwide - Genetic Literacy Project

Building on an R&D tie-up with an Indian stem cell institution last year, UK biotech PhoreMost has begun a collaboration with Japans Otsuka working on several gene therapy projects.

Cambridge-based PhoreMost said it will use its next-generation phenotypic screening platform Siteseeker to identify novel targets for Otsukas therapeutics discovery programmes.

Novel targets identified will be further validated and characterised by Otsuka as part of its internal development pipeline, with an initial focus on gene therapy applications of identified targets.

Siteseeker looks at different protein shapes to find functionally active peptides that can be targeted by new therapies.

The technology looks at the entire proteome all of the proteins expressed in a live cell environment looking for druggable targets for a chosen disease.

Financial details of the agreement were not disclosed.

Dr Chris Torrance, CEO of PhoreMost, said: This collaboration with Otsuka is further recognition of the power of the Siteseeker approach to drive the identification of novel, druggable targets.

We are particularly excited to be exploring not only small molecule therapeutics but also gene therapy applications of our platform.

PhoreMost was one of two UK-based companies to receive funding from the government-backed agency Innovate UK to receive funding for small molecule research.

The 1 million funding was announced in 2018, and supported PhoreMost and the immune-oncology firm NeoPhore.

The companies won the funding as part of a competition organised by Innovate UK and funded by the UKs Biomedical Catalyst.

PhoreMost is also working with Indias Centre for Chemical Biology and Therapeutics, part of the Institute for Stem Cell Science and Regenerative Medicine (inStem).

The project began in July last year and, with funding from the Indian government, aims to create chemical tools that modulate novel classes of drug targets.

InStem is researching the genetic mechanisms of potency, differentiation and proliferation in human pluripotent cells.

It aims to examine diseases that can potentially be treated by stem cells.

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UK biotech PhoreMost to work with Otsuka on gene therapy projects - - pharmaphorum

The latest release from CMIwith title Personalized Gene Therapy Treatments for Cancer Market Research Report 2020-2026 (by Product Type, End-User / Application and Regions / Countries) provides an in-depth assessment of the Personalized Gene Therapy Treatments for Cancer including key market trends, upcoming technologies, industry drivers, challenges, regulatory policies, key players company profiles and strategies. Global Personalized Gene Therapy Treatments for Cancer Market study with 100+ market data Tables, Pie Chat, Graphs & Figures is now released BY Coherent Market Insights. The report presents a complete assessment of the Market covering future trends, current growth factors, attentive opinions, facts, and industry-validated market data forecast until 2026.

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The segmentation chapter allows readers to understand aspects of the Global Personalized Gene Therapy Treatments for Cancer Market such as products/services, available technologies, and applications. These chapters are written in a way that describes years of development and the process that will take place in the next few years. The research report also provides insightful information on new trends that are likely to define the progress of these segments over the next few years.

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Key companies covered as a part of this study include (Amgen, Inc., SynerGene Therapeutics, Inc., Chengdu Shi Endor Biological Engineering Technology Co., Ltd., Cold Genesys, Inc., Takara Bio, Inc., Bellicum Pharmaceuticals, Inc., Ziopharm Oncology, Inc., OncoSec Medical, Inc., Sevion Therapeutics, Inc., and Burzynski Clinic.)

Global Personalized Gene Therapy Treatments for Cancer Market and Competitive Analysis

Know your current market situation! Not only an important element for new products but also for current products given the ever-changing market dynamics. The study allows marketers to stay in touch with current consumer trends and segments where they can face a rapid market share drop. Discover who you really compete against in the marketplace, with Market Share Analysis know market position, % market Share and Segmented Revenue of Personalized Gene Therapy Treatments for Cancer Market

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Essential demographic, geographic, psychographic and behavioral information about business segments in the Personalized Gene Therapy Treatments for Cancer market is targeted to aid in determining the features company should encompass in order to fit into the business requirements. For the Consumer-based market the study is also classified with Market Maker information in order to better understand who the clients are, their buying behavior and patterns.

*** For the global version, a list of below countries by region can be added as part of customization at minimum cost.North America (United States, Canada & Mexico).Asia-Pacific (Japan, China, India, Australia, etc).Europe (Germany, UK, France, etc).Central & South America (Brazil, Argentina, etc).Middle East & Africa (United Arab Emirates, Saudi Arabia, South Africa, etc).

Personalized Gene Therapy Treatments for Cancer Product/Service Development

Knowing how the product/services fit the needs of clients and what changes would require to make the product more attractive is the need of an hour. Useful approaches to focus group by utilizing User Testing and User Experience Research. Demand-side analysis always helps to correlate consumer preferences with innovation.

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Pricing/subscription always plays an important role in buying decisions; so we have analyzed pricing to determine how customers or businesses evaluate it not just in relation to other product offerings by competitors but also with immediate substitute products. In addition to future sales Separate Chapters on Cost Analysis, Labor*, production* and Capacity are Covered.

How geography and sales fit together

This study is helpful to all operators who want to identify the exact size of their target audience at a specific geographic location. The keywordMarket allows entrepreneurs to determine local markets for business expansion. This study answers the questions below:

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Historical year 2013-2018Base year 2019Forecast period** 2020 to 2026 [** unless otherwise stated]

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Personalized Gene Therapy Treatments for Cancer Market 2020-2026: Deep Analysis of Current Trends and Future Demand by Top Key Players - Vital News 24

PALM BEACH, Florida, Jan. 22, 2020 /PRNewswire/ -- Pancreatic cancer is a notoriously aggressive and hard-to-treat malignancy; the five-year survival rate is less than 10%. Treatment options for pancreatic cancer are limited to surgery, radiation, and chemotherapies; thus, demand is high for safer, more-efficacious drugs, which will serve to drive the market. ResearchAndMarkets projects that the global pancreatic cancer therapy market is expected to reach US$ 4,056.4 Mn in 2025 from US$ 2,011.2 Mn in 2017. The market is estimated to grow with a CAGR of 8.1% from 2018-2025. The growth of the pancreatic cancer therapy market is primarily attributed to the increase in number of therapies launched in the market. The development of new drugs is thus expected to drive the growth of pancreatic cancer therapy market. The report continued by saying: "However, these drugs travel throughout the body and thus can affect normal and healthy cells adversely. Blood-forming cells in the bone marrow, hair follicles and cell in the mouth, digestive tract, and reproductive system are likely to be affected by chemotherapeutic drugs. The current treatment options available for pancreatic cancer patients only help to extend the patients' lives by a few months. Active biotech and pharma companies in the markets this week include Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), Mallinckrodt plc (NYSE: MNK), Genprex, Inc. (NASDAQ: GNPX), Soligenix, Inc. (NASDAQ: SNGX), BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX).

"This demands potential drugs that are able to enhance the effectiveness of chemotherapy drugs. Combination of two or more chemotherapy drugs are a suitable way out to increase the efficacy of the drug as well as proves advantageous in the treatment the biologic therapy segment is expected to grow at the fastest rate during the coming years owing to increasing number of novel upcoming immunotherapies as well as targeted cell therapy that treats the cancer without any severe side effects and has number of therapeutic benefits over the conventional chemotherapies This demands potential drugs that are able to enhance the effectiveness of chemotherapy drugs. Combination of two or more chemotherapy drugs are a suitable way out to increase the efficacy of the drug as well as proves advantageous in the treatment."

Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC) BREAKING NEWS: Oncolytics Biotech Announces Publication of an Abstract for the 2020Gastrointestinal Cancers Symposium Highlighting CEACAM6 as a Potential Prognostic Biomarker Candidate for Pancreatic Cancer- Oncolytics Biotech currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, today stated that the previously announced abstract for a poster to be presented at the 2020Gastrointestinal Cancers Symposium sponsored by ASCO in San Francisco, has been published. The abstract highlights new biomarker data from the randomized study NCI 8601: Carboplatin and Paclitaxel With or Without Viral Therapy in Treating Patients With Recurrent or Metastatic Pancreatic Cancer.

The abstract, CEACAM6 is a candidate biomarker for Reolysin (pelareorep) sensitivity in pancreatic adenocarcinoma (PDAC), was co-authored by Dr. Anne Noonan, Department of Medical Oncology, Ohio State University Wexner Medical Center, Richard Solove Research Institute and James Cancer Hospital, and Dr. Tanios Bekaii-Saab Senior Associate Consultant, Division of Hematology/Oncology, Department of Internal Medicine, Mayo Clinic, Phoenix, Arizona.

Data in the abstract associate low levels of the gene CEACAM6 with prolonged progression free survival (PFS) in pelareorep-treated patients with pancreatic cancer, with PFS improving from 5.72 months to 10.32 months (p=0.05). This effect was not seen in non-pelareorep treated patients. Consequently, CEACAM6 may serve as a prognostic biomarker for sensitivity of pancreatic tumors to pelareorep treatment. Additional data will be announced following the poster presentation.

Read this full press release and more news for ONCY at: https://www.financialnewsmedia.com/news-oncy/

Other recent developments in the biotech industry include:Mallinckrodt plc (NYSE: MNK) a global biopharmaceutical company, recently confirmed enrollment of the first patient in the company's Phase 4, multi-center, multiple-dose, open-label study to assess the effects of Acthar Gel as a therapy option in patients with severe keratitis.

"In my experience, a considerable number of severe keratitis patients can have persistent disease that may not be resolved by first-line treatment," saidEugene McLaurin, MD and Fellow, American Academy of Ophthalmology and American College of Surgeons."I am pleased that the first patient has been enrolled in this important Phase 4 study, the results of which may potentially provide data to further support Acthar Gel as a treatment option in appropriate keratitis patients."

Genprex, Inc. (NASDAQ: GNPX) a clinical-stage gene therapy company utilizing a unique, non-viral proprietary platform designed to deliver tumor suppressor genes to cancer cells, recently announced that the U.S Food and Drug Administration (FDA) has granted Fast Track Designation for Genprex's Oncoprex immunogene therapy in combination with EGFR inhibitor osimertinib (AstraZeneca's Tagrisso, which had worldwide sales in 2018 of $1.86 billion, $2.31 billion in the first 9 months of 2019 and is currently AstraZeneca's highest grossing product) for the treatment of non-small cell lung cancer (NSCLC) patients with EFGR mutations that progressed after treatment with osimertinib alone. Oncoprex is comprised of the TUSC2 (Tumor Suppressor Candidate 2) gene complexed with a lipid nanoparticle. TUSC2 is the active agent in Oncoprex.

Genprex has treated more than 50 lung cancer patients with Oncoprex in Phase I and II clinical trials. The company believes the data from these trials are encouraging as to both safety and efficacy.

Soligenix, Inc. (NASDAQ: SNGX) a late-stage pharmaceutical company working to improve the current standard of care for numerous rare diseases through the development and commercialization of novel treatments, is nearing the release of topline data after completing final enrollment for SGX301, a pivotal Phase 3 trial targeting the treatment of cutaneous T-cell lymphoma. A successful report may position the companyfor significant increases to shareholder value before the end of Q1 2020, and ultimately position the company to commercialize its first drug in a market that is estimated at a more than $200 million revenue opportunity.

The Phase 3clinical trial is focused on the potential benefits ofSoligenix's topical drug ointment SGX301, or synthetic hypericin, in the treatment of cutaneous T-cell lymphoma (CTCL). CTCL is a rare type of Non-Hodgkin's Lymphoma that sits high on the list of conditions that has no current or effective drug treatment, pushing Soligenix to fill the demand to serve this unmet medical need.

BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX) recently announced the appointments of Charles Gayer as chief commercial officer and Allen Hodge as vice president and general manager for the United States.

Mr. Gayer joined BioCryst in 2015 as vice president of global strategic marketing. Since July 2019 he has served as interim chief commercial officer, playing a key role in defining the strategy, and building the commercial operations, to support the launch of berotralstat, BioCryst's oral kallikrein inhibitorfor hereditary angioedema (HAE).

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Hopeful New Therapies For Pancreatic Cancer Expected to be a Big Boon for Biotechs - P&T Community

In 2017 we reported on an experimental immunotherapy cancer treatment which showed extraordinary results in clinical trials. This type of treatment is known as T-cell cancer therapy.

How exactly does T-cell cancer therapy work?

The field of immunotherapy aims to "supercharge" the body's own immune system, better enabling it to fight cancer.

T-cells are white blood cells that form part of the immune system, detecting and attacking abnormal or foreign cells in the body.

In cancer patients, T-cells normally aren't able to completely rid the body of cancer.

In T-cell therapy, immune cells are engineered to better recognise and fight cancer cells. Researchers extract these cells from blood and edit them with gene transfers to produce a potent receptor that can more effectively fight cancer. The cells are then placed back into the patients body

While this type of treatment was initially used in patients with an advanced form of leukaemia, a new breakthrough meant that T-cell therapy could be used for many other types of cancer.

What does the new research entail?

According to a news report, in 2020, researchers discovered a new immune cell receptor. This means that T-cell therapy as we know it can work much, much better and fight more cancers as it was only able to recognise a handful of cancers in the past.

This meant that, because of a T-cell receptor called human leukocyte antigen (HLA), the treatment had to be personalised for every single patient. This is the receptor that enables the cells to detect and fight the cancer.

Usually HLA varies from person to person, but that is where the new discovery could be a breakthrough.

The new study was led by scientists from Cardiff University in the UK who used the CRISPR-Cas9 screening to discover a new type of receptor called MR1. The full study can be found in the journal Nature Immunology.

These receptors do exactly what HLA does, but there is one big difference the treatment doesnt differ from person to person, resulting in a much better basis for T-cell therapy.

Too soon to tell, but prospect is exciting

There is, however, one caveat. The research is still very new and was conducted in a lab, which means that positive results first need to be achieved in clinical trials.

Experts remain optimistic and call this discovery an exciting new frontier.

"This research represents a new way of targeting cancer cells, which is really quite exciting, although much more research is needed to understand precisely how it works,"says research and policy director Alasdair Rankin from the blood cancer charity Bloodwise, who was not involved in the research

Image credit: iStock

Compiled by Marelize Wilke

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New breakthrough in cancer research promises better immune therapy - Health24

Dublin, Jan. 23, 2020 (GLOBE NEWSWIRE) -- The "Global Healthcare Market Outlook, 2020" report has been added to ResearchAndMarkets.com's offering.

Amid rising global trade tensions and sluggish global economic outlook for 2020, the global healthcare market is expected to cross the $2 trillion mark in 2020.

Healthcare will be among the top two priorities for voters in the 2020 presidential election in the US. In the European region, looming BREXIT indecision is likely to have a strong impact on Europe's biggest digital health market (UK). Globally, 2020 will be a reality check for long-pending national healthcare policies and regulatory reforms that must re-invigorate future strategies.

The new vision for healthcare for 2020 and beyond will not just focus on access, quality, and affordability but also on predictive, preventive, and outcome-based care models promoting social and financial inclusion. Social Determinants of Health (SDOH) will emerge has a big theme across progressive health systems to proactively engage the right patients and improve health outcomes to help healthcare organizations meet quality standards. In 2020, consumer-driven models of healthcare will gain more market traction, as they stand to better bridge the gap of what consumers want and what healthcare can deliver.

Continued steps will be taken by retail (Walmart, Costco, Amazon, Ali Health), and consumer tech (Google, Apple, Microsoft, and so on) companies globally; to make further headway (intrude) into vetted healthcare space. In 2020, the convergence of Artificial Intelligence (AI), Blockchain, and the Internet of Things (IoT) will further catalyze the space of innovation adoption and related applications in the healthcare realm. For example, while Blockchain will improve data liquidity to empower AI and analytics vendors/applications to digest a large amount of data, AI can manage Blockchain systems more efficiently than humans.

Research Scope

Every year, the team of futurists, analysts, and consultants at the publisher's Transformational Healthcare Group come together to render a comprehensive analysis to predict the themes, technologies, and global forces that will define the next 12 to 18 months (future) for the healthcare industry.

As a part of this research deliverable, the publisher provides bold perspectives and predictions for the global healthcare market in 2020. The sectors covered include pharmaceuticals and biotech, in-vitro diagnostics, medical technologies, medical imaging, and healthcare IT. The analysis captures sectoral and regional trends and provides predictions for the upcoming year. The study provides guidance on where to find the greatest opportunities for expansion.

Predictions for the global healthcare market in 2020 include:

Key Issues Addressed

Key Topics Covered

1. Executive Summary

2. Revisiting 2019 Predictions

3. Global Healthcare Market Outlook for 2020

4. Key 2020 Healthcare Market Predictions

5. Regional Predictions 2020

6. Sector Outlook 2020

7. Key Conclusions

For more information about this report visit https://www.researchandmarkets.com/r/79rxhs

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

CONTACT: ResearchAndMarkets.comLaura Wood, Senior Press Managerpress@researchandmarkets.comFor E.S.T Office Hours Call 1-917-300-0470For U.S./CAN Toll Free Call 1-800-526-8630For GMT Office Hours Call +353-1-416-8900

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Global Healthcare Market Outlook, 2020: Key Predictions & Growth Opportunities for the $2+ Trillion Industry - Yahoo Finance

The gene therapy company sold 7.6 million shares of stock at a price of $1.05 per share

Genprex Inc () announced an $8 million offering of stock through securities purchase agreements made with influential investors.

The gene therapy company sold 7.6 million shares at a price of $1.05 per share, in accordance with Nasdaq rules governing registered direct offerings.

Genprex, based in Austin, Texas and Cambridge, Massachusetts, plans to use the proceeds to advance its clinical programs in non-small cell lung cancer, as well as for working capital and general corporate purposes.

The company is developing a new approach to treating cancer, including its initial product candidateOncoprex, which is an immunogene therapy for non-small cell lung cancer.

Contact Andrew Kessel at [emailprotected]

Follow him on Twitter @andrew_kessel

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The Gene Therapy and Antisense Drugs Market research report added by Market Study Report, LLC, provides a succinct analysis on the recent market trends. In addition, the report offers a thorough abstract on the statistics, market estimates and revenue forecasts, which further highlights its position in the industry, in tandem with the growth strategies adopted by leading industry players.

The Gene Therapy and Antisense Drugs market study is a well-researched report encompassing a detailed analysis of this industry with respect to certain parameters such as the product capacity as well as the overall market remuneration. The report enumerates details about production and consumption patterns in the business as well, in addition to the current scenario of the Gene Therapy and Antisense Drugs market and the trends that will prevail in this industry.

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What pointers are covered in the Gene Therapy and Antisense Drugs market research study?

The Gene Therapy and Antisense Drugs market report Elucidated with regards to the regional landscape of the industry:

The geographical reach of the Gene Therapy and Antisense Drugs market has been meticulously segmented into United States, China, Europe, Japan, Southeast Asia & India, according to the report.

The research enumerates the consumption market share of every region in minute detail, in conjunction with the production market share and revenue.

Also, the report is inclusive of the growth rate that each region is projected to register over the estimated period.

The Gene Therapy and Antisense Drugs market report Elucidated with regards to the competitive landscape of the industry:

The competitive expanse of this business has been flawlessly categorized into companies such as

Segmentation

On the basis of therapeutic area, the gene therapy and antisense drugs market is segmented into cancer, anemia, rheumatoid arthritis, cardiovascular diseases, HIV/AIDS, cystic fibrosis, diabetes mellitus and obesity, and renal diseases.

By gene transfer method, ex vivo gene transfer and in vivo gene transfer are the segments of the market. The former involves the transfer of cloned genes into cells, i.e., cells are altered outside the body before being implanted into the patient, whereas the latter involves the transfer of cloned genes directly into the patients tissues. The outcome of in vivo gene transfer technology mainly depends on the general efficacy of gene transfer and expression.

Global Gene Therapy and Antisense Drugs Market: Regional Outlook

The global gene therapy and antisense drugs market is segmented into North America, Asia Pacific, Europe, and Rest of the World. Amongst these, North America holds the leading position in the market followed by Europe. The increasing incidence of cancer and other fatal diseases, unhealthy lifestyle practices such as excessive smoking and excessive consumption of high fat content food, and increasing research efforts for treatment against cancer are the major factors driving the gene therapy and antisense drugs market in these regions.

Asia Pacific is expected to emerge as a significant market for gene therapy and antisense drugs. The high population density including a large geriatric population, expeditiously increasing demand for technologically advanced therapeutics, and increasing government support for improved healthcare infrastructure in the region is driving the growth of this regional market. Furthermore, favorable reimbursement policies and tax benefits on newer therapies will further fuel the growth of the Asia Pacific gene therapy and antisense drugs market.

Major Companies Mentioned in Report

Some of the leading companies operating in the global gene therapy and antisense drugs market are GenVec Inc., Avigen Inc., Genome Therapeutics Corp., Tekmira Pharmaceuticals Corporation, Isis Pharmaceuticals, Cell Genesys Inc., and others. These companies are profiled for their key business attributes in the report.

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Exclusive details pertaining to the contribution that every firm has made to the industry have been outlined in the study. Not to mention, a brief gist of the company description has been provided as well.

Substantial information subject to the production patterns of each firm and the area that is catered to, has been elucidated.

The valuation that each company holds, in tandem with the description as well as substantial specifications of the manufactured products have been enumerated in the study as well.

The Gene Therapy and Antisense Drugs market research study conscientiously mentions a separate section that enumerates details with regards to major parameters like the price fads of key raw material and industrial chain analysis, not to mention, details about the suppliers of the raw material. That said, it is pivotal to mention that the Gene Therapy and Antisense Drugs market report also expounds an analysis of the industry distribution chain, further advancing on aspects such as important distributors and the customer pool.

The Gene Therapy and Antisense Drugs market report enumerates information about the industry in terms of market share, market size, revenue forecasts, and regional outlook. The report further illustrates competitive insights of key players in the business vertical followed by an overview of their diverse portfolios and growth strategies.

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Some of the Major Highlights of TOC covers:

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R&D Activities to Fast-track the Growth of the Gene Therapy and Antisense Drugs Market Between 2017 2025 - Fusion Science Academy

This report presents the worldwide In Situ Hybridization market size (value, production and consumption), splits the breakdown (data status 2018 and forecast to 2025), by manufacturers, region, type and application.

This study also analyzes the market status, market share, growth rate, future trends, market drivers, opportunities and challenges, risks and entry barriers, sales channels, distributors and Porters Five Forces Analysis.

The report presents the market competitive landscape and a corresponding detailed analysis of the major vendor/key players in the market.

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Top Companies in the Global In Situ Hybridization Market:

key drivers, restraints, recent trends, and growth opportunities in the global market for in situ hybridization. The market shares, product portfolios, technological developments, and business strategies of the major players are discussed in detail. The report also sheds light on factors such as market size, supply and demand ratio, market attractiveness, and key segments of the in situ hybridization market.

Global In Situ Hybridization Market: Drivers and Restraints

Technological advancements in the field of in situ hybridization, such as development of the cytogenetic technique, is one of the key factors driving the market. The rising prevalence of cancer has necessitated clinical research, which is another major market driver. The introduction of new therapeutic agents has led to a high rate of adoption of companion diagnostics, boosting the overall market expansion. Besides these factors, the growth of the pharmaceutical and biotechnology industry has improved the prospects of the in situ hybridization market worldwide.

Florescence in situ hybridization (FISH) can be used in the detection of genetic abnormalities such as aneuploidy, characteristic gene fusion, or loss of a chromosomal region. It is also useful for research in the fields of gene mapping and identification of genetic aberrations, which are responsible for cancer. As this technique is simple yet effective, FISH will ensure the growth of the global in situ hybridization market.

On the contrary, strict regulatory policies will obstruct the growth of the in situ hybridization market. However, the emergence of molecular cytogenetics will present significant opportunities due to unmet needs in accurate disease diagnosis, rising number of chromosomal disorders, and surge in population.

Global In Situ Hybridization Market: Regional Outlook

On the basis of geography, the global market for in situ hybridization can be segmented into Europe, Asia Pacific, Latin America, North America, and the Middle East and Africa. North America holds a large share in the overall market, with Europe and Asia Pacific also exhibiting promising growth. Extensive research activities in countries such as Canada and the U.S., increased adoption of companion diagnostics, and beneficial government policies have been aiding the in situ hybridization market in North America.

Over the forecast period 2017-2025, Asia Pacific will undergo tremendous growth on account of growing incidence of cancer and its diagnosis, increased healthcare expenditure, and increasing health awareness among people. The presence of international companies in countries such as India and China will further provide an impetus to the market.

Companies Mentioned in the Report

The major companies operating in the market for in situ hybridization include Bio Sb, Inc., Advanced Cell Diagnostics, Inc., Merck KGaA, Abbott Laboratories, Inc., Leica Biosystems Nussloch GmbH, Danaher Corporation, and Agilent Technologies. Several companies are using business strategies such as product enhancement, collaborations, acquisitions, and partnerships with a view to increasing profit.

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The report provides a valuable source of insightful data for business strategists and competitive analysis of In Situ Hybridization Market. It provides the In Situ Hybridization industry overview with growth analysis and futuristic cost, revenue and many other aspects. The research analysts provide an elaborate description of the value chain and its distributor analysis. This Tire In Situ Hybridization study provides comprehensive data which enhances the understanding, scope and application of this report.

Influence of the In Situ Hybridization market report:

-Comprehensive assessment of all opportunities and risk in the In Situ Hybridization market.

In Situ Hybridization market recent innovations and major events.

-Detailed study of business strategies for growth of the In Situ Hybridization market-leading players.

-Conclusive study about the growth plot of In Situ Hybridization market for forthcoming years.

-In-depth understanding of In Situ Hybridization market-particular drivers, constraints and major micro markets.

-Favorable impression inside vital technological and market latest trends striking the In Situ Hybridization market.

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The report has 150 tables and figures browse the report description and TOC:

Table of Contents

1 Study Coverage

1.1 In Situ Hybridization Product

1.2 Key Market Segments in This Study

1.3 Key Manufacturers Covered

1.4 Market by Type

1.4.1 Global In Situ Hybridization Market Size Growth Rate by Type

1.4.2 Hydraulic Dredges

1.4.3 Hopper Dredges

1.4.4 Mechanical Dredges

1.5 Market by Application

1.5.1 Global In Situ Hybridization Market Size Growth Rate by Application

2 Executive Summary

2.1 Global In Situ Hybridization Market Size

2.1.1 Global In Situ Hybridization Revenue 2014-2025

2.1.2 Global In Situ Hybridization Production 2014-2025

2.2 In Situ Hybridization Growth Rate (CAGR) 2019-2025

2.3 Analysis of Competitive Landscape

2.3.1 Manufacturers Market Concentration Ratio (CR5 and HHI)

2.3.2 Key In Situ Hybridization Manufacturers

2.3.2.1 In Situ Hybridization Manufacturing Base Distribution, Headquarters

2.3.2.2 Manufacturers In Situ Hybridization Product Offered

2.3.2.3 Date of Manufacturers Enter into In Situ Hybridization Market

2.4 Key Trends for In Situ Hybridization Markets & Products

3 Market Size by Manufacturers

3.1 In Situ Hybridization Production by Manufacturers

3.1.1 In Situ Hybridization Production by Manufacturers

3.1.2 In Situ Hybridization Production Market Share by Manufacturers

3.2 In Situ Hybridization Revenue by Manufacturers

3.2.1 In Situ Hybridization Revenue by Manufacturers (2019-2025)

3.2.2 In Situ Hybridization Revenue Share by Manufacturers (2019-2025)

3.3 In Situ Hybridization Price by Manufacturers

3.4 Mergers & Acquisitions, Expansion Plans

More Information.

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Global Cake Mix Market Industry: A Latest Research Report to Share Market Insights and Dynamics - Fusion Science Academy

The Global Gene Therapy Market is growing at an exciting pace driven by changing dynamics and risk ecosystem, a study of which forms the crux of the report. The study on the global Gene Therapy market takes a closer look at several regional trends and the emerging regulatory landscape to assess its prospects. The critical assessment of the numerous growth factors and breaks in the global Gene Therapy market offered in the analyses helps in assessing the lucrativeness of its key segments.

Download Free PDF Brochure for Latest Research Study of Gene Therapy Market:: https://www.globalmarketers.biz/report/life-sciences/global-gene-therapy-industry-market-research-report/7517 #request_sample

This Report Covers Leading Companies Associated in Worldwide Market:

UniQure NVBluebird Bio,Celladon,Avalanche Bio,Sangamo,Shanghai Sunway Biotech Co. LtdAdvantagene,Sibiono GeneTech,Spark Therapeutics,Dimension Therapeutics,

Summary of Market: The global Gene Therapy market is valued at xx million US$ in 2019 is expected to touch xx million US$ by the close of 2025, growing at a CAGR of xx% during 2020-2025.

The report emphases on Gene Therapy Market volume and value at Global Level, Regional Level And Company Level. From a global standpoint, this report embodies overall market size by studying historical data and future outlook.

The report is bifurcated into product type, applications, and regions worldwide. The above areas are further bifurcated into country-level data statistics for the below countries.

The key regions and countries covered in this report are:

Please note, the regional and country level data can be altered and provided as per clients custom requirements.

Global Gene Therapy Market Segmentation, By Product Type:

Viral vectorNon-viral vector

Global Gene Therapy Market Segmentation, By Application:Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

Inquire Before Buying: https://www.globalmarketers.biz/report/life-sciences/global-gene-therapy-industry-market-research-report/7517 #inquiry_before_buying

Research objectives:

To study and estimate the market size of Gene Therapy , in terms of value.

To find development and challenges for the global market.

To observe worthwhile expansions including expansions, new services presents in worldwide industry.

To classify and assess the side view of important companies of Global Gene Therapy industry.

Key Questions Answered in the Report:

How is the Gene Therapy market expected to Grow In Terms Of Value during the study period?

What are the Competition Developments and Trends in the Gene Therapy market?

What are the core Macro-Economic and Industry Factors impacting the growth of the Gene Therapy market?

What are the Key Challenges, Opportunities, and Improvements faced by market players in the global Gene Therapy market?

Table of Content

1 Report Outline

1.1 Research Opportunity

1.2 Major Industrialists

1.3 Market Segment by Type

1.4 Market Segment by Application

1.5 Study Objectives

1.6 Years Considered

2 Global Evolution Trends

2.1 Production and Volume Analysis

2.1.1 Global Gene Therapy Production Value 2015-825

2.1.2 Global Gene Therapy Production 2015-2025.

2.1.3 Global Gene Therapy Capacity 2015-2025.

2.1.4 Global Gene Therapy Marketing Pricing and Trends

2.2 Major Producers Growth Rate (CAGR) 2020-2025.

2.2.1 Global Gene Therapy Market Size CAGR of Major Regions

2.2.2 Global Gene Therapy Market Share of Major Regions

2.3 Industry Trends

2.3.1 Market Top Trends

2.3.2 Market Operators

3 Market Share by Industrialists

3.1 Capacity and Production by Industrialists

3.1.1 Global,Gene Therapy Capacity by Industrialists

3.1.2 Global Gene Therapy Production by Industrialists

3.2 Revenue by Industrialists

3.2.1. Gene Therapy Revenue by Industrialists (2015-2020)

3.2.2. Gene Therapy Revenue Share by Industrialists (2015-2020)

3.2.3 Global Gene Therapy Market Concentration Ratio (CR5 and HHI)

3.3. Gene Therapy Price by Industrialists

3.4 Major Industrialists of Gene Therapy Plants/Factories Distribution and Area Served

3.5 Date of Major Industrialists Enter into Gene Therapy Market

3.6 Major Industrialists Gene Therapy Product Offered

3.7 Mergers & Acquisitions, Expansion Plans

4 Market Dimensions by Type

4.1 Production and Production Rate for Each Type

4.2 Global Gene Therapy Production Market Share by Type

4.3 Global Gene Therapy Production Value Market Share by Type

4.4. Gene Therapy Ex-factory Price by Type

5 Market Size by Application

5.1 Overview

5.2 Global Gene Therapy Consumption by Application

6 Production by Regions

6.1 Global Gene Therapy Production (History Data) by Regions 2015-2020.

6.2 Global Gene Therapy Production Value (History Data) by Regions

6.3 North America

6.3.1 North America Production Growth Rate 2015-2020.

6.3.2 North America Production Value Growth Rate 2015-2020.

6.3.3 Major Players in North America

6.3.4 North America Import & Export

6.4 Europe

6.4.1 Europe Production Growth Rate 2015-2020.

6.4.2 Europe Production Value Growth Rate 2015-2020.

6.4.3 Major Players in Europe

6.4.4 Europe Import & Export

6.5 China

6.5.1 China Production Growth Rate 2015-2020.

6.5.2 China Production Value Growth Rate 2015-2020.

6.5.3 Major Players in China

6.5.4 China Import & Export

6.6 Japan

6.6.1 Japan Production Growth Rate 2015-2020.

6.6.2 Japan Production Value Growth Rate 2015-2020.

6.6.3 Major Players in Japan

6.6.4 Japan Import & Export

7. Gene Therapy Consumption by Regions

7.1 Global Gene Therapy Consumption (History Data) by Regions

7.2 North America

7.2.1 North America Consumption by Type

7.2.2 North America Consumption by Application

7.2.3 North America Consumption by Countries

7.2.4 United States

7.2.5 Canada

7.2.6 Mexico

7.3 Europe

7.3.1 Europe Consumption by Type

7.3.2 Europe Consumption by Application

7.3.3 Europe Consumption by Countries

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Gene Therapy Market Is Thriving Worldwide with major players Like: UniQure NV Bluebird Bio, Celladon, Avalanche Bio - Expedition 99

A recent market study published by Marketresearch.biz in its upcoming report outlook titled, Gene Therapy Market: Global Industry Analysis and Astonishing Growth [2020-2029] offers key market insights and emerging trends on the global Gene Therapy market.

The report has offered an exhaustive analysis of the Gene Therapy Market taking into consideration all the crucial aspects like growth factors, constraints, market developments, and future prospects. Market researchers and industry experts have pointed out the key market trends and prospects that may impact the overall Gene Therapy Market growth. This will help players to leverage the opportunities to strengthen their position. Also, the report throws light on the important factors that are contributing to the Gene Therapy Market growth. Additionally, challenges and impeding factors that could hamper the growth of the Gene Therapy Market in the years to come are mentioned in the report.

The Gene Therapy market report includes comprehensive information about the markets major competitors, including various organizations, companies, associations, suppliers and manufacturers competing for production, supply, sales, revenue generation, and after-sales performance expectations. The bargaining power of numerous vendors and buyers have also been included in the research report.

Download Free PDF Brochure with Full Analysis of Key Players:https://marketresearch.biz/report/gene-therapy-market/request-sample

Following Key Players are Analysed in this Report: Novartis, Kite Pharma Inc, GlaxoSmithKline PLC, Spark Therapeutics Inc, Bluebird bio Inc, Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica PLC, NewLink Genetics Corp., Amgen Inc

Market Dynamics:

Set of qualitative informative data that incorporates PORTER Five Forces Model, Macro-Economic factors, PESTEL Analysis, SWOT Analysis, Value Chain Analysis, Regulatory Framework along with Industry Background and Overview.

Worldwide Gene Therapy Research Methodology

Marketresearch.biz presents a detailed picture of the market by way of study, synthesis, and summation of information from various sources. The information thus presented is reliable, comprehensive, and the result of extensive research, both primary and secondary. The analysts have presented the different features of the market with a particular focus on identifying the key business influencers.

Gene Therapy Market Data Break Down is illuminated below by vector type, gene type, application, and region:

By Vector:Viral vectorRetrovirusesLentivirusesAdenovirusesAdeno Associated VirusHerpes Simplex VirusPoxvirusVaccinia VirusNon-viral vectorNaked/Plasmid VectorsGene GunElectroporationLipofection

By Gene Therapy:AntigenCytokineTumor SuppressorSuicideDeficiencyGrowth factorsReceptorsOther

By Application:Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

The report cast light on an extensive analysis based on the most prominent Gene Therapy manufacturers operating in the industry. Activities performed by robust Gene Therapy manufacturers/companies are product development, research, and innovation as well as technology adoptions that are intensifying the competitive intensity and companys ability to offer better product lineup. The companies are also performing strategic acquisitions, ventures, mergers, and partnerships to enlarge their serving area and strengthen their existence worldwide.

The study delivers an exact evaluation of the financial operations of companies covering Gene Therapy sales volume, capital investment, gross margin, profitability, revenue, cash flow, and growth rate. Their manufacturing capacity, production volume, product specifications, import-export activities, production processes, raw material sourcing, and distribution networks are also elaborated in this Gene Therapy report. Companies are also engaged in product launches, promotional activities, and brand developments as part of strategic planning.

How will the report help new companies to plan their investments in the Gene Therapy market?

The Gene Therapy market research report classifies the competitive spectrum of this industry in elaborate detail. The study claims that the competitive reach spans the companies.

The report also mentions about the details such as the overall remuneration, pricing trends, product sales figures, gross margins, etc.

Information about the sales & distribution area alongside the details of the company, such as company overview, product specifications, buyer portfolio, etc., are provided in the Gene Therapy market study.

Inquire/Speak To Expert for Further Detailed Information About Gene Therapy Report :https://marketresearch.biz/report/gene-therapy-market/#inquiry

Regional Outlook: Regional analysis is another important part of the report which is segregated into different sections. One section of the report is entirely dedicated to regional consumption analysis whereas another for regional production analysis. It includes North America, Europe, China, Japan, Southeast Asia, India.

Some of the Major Highlights of TOC covers in Gene Therapy Market Report:

Chapter 1: Methodology & Scope of Gene Therapy Market

Definition and forecast parameters

Methodology and forecast parameters

Data Sources

Chapter 2: Executive Summary of Gene Therapy Market

Business trends

Regional trends

Product trends

End-use trends

Chapter 3: Gene Therapy Industry Insights

Industry segmentation

Industry landscape

Vendor matrix

Technological and innovation landscape

Chapter 4: Gene Therapy Market, By Region

Chapter 5: Company Profile

Business Overview

Financial Data

Product Landscape

Strategic Outlook

SWOT Analysis

And CONTINUE

Analysts with high skill in information gathering and governance use industry strategies to collate and examine data at all stages. Our analysts are trained to combine superior research methodology, modern data collection techniques, subject expertise and years of collective experience to deliver useful and accurate research reports.

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Gene Therapy Market Segmentation 2020 | Analyzing the Impact Followed by Restraints till 2029 - News Monitoring

Coherent Market Insights has recently announced the addition of a new research report to its repository named, Global Cell and Gene Therapy Market Status and Forecast 20192026, covering top-line subjective and quantitative synopsis data. The market review provides an eccentric tool for analyzing the market in terms of strengths, and weakness, marking opportunities, and supporting strategic and proficient decision-making. The key drivers and restraints affecting the growth of the Cell and Gene Therapy are stated.

A comprehensive analysis of the Cell and Gene Therapy market is presented in this document, along with a brief overview of the segments in the industry. The study presents a feasible estimate of the current market scenario, including the Cell and Gene Therapy market size with regards to the volume and renumeration. The report is a collection of significant data related to the competitive landscape of the industry. It also contains data with regards to several regions that have successfully established its position in the Cell and Gene Therapy market.

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The Top players including:Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST.

The report also points out the latest trends in the market and the various opportunities for the market to grow in the near future. Insightful information about the key players such as business market overview, product offerings, and industry revenue segmentation has been provided in the report. It also serves an extensive analysis of different sections and sub-segments which offers market insights toward the historic market scenarios along with future growth and prospect.

Our competitor profiling includes evaluation of distribution channels and products and services offered by and financial performance of companies operating in the global Cell and Gene Therapy market. We also provide Porters Five Forces, PESTLE, and SWOT analysis to assess competitive threat and examine other aspects of the global Cell and Gene Therapy market. The report offers strategic recommendations, competitor benchmarking for performance measurement, and analysis of partnership, merger, and acquisition targets and industry best practices. It also provides analysis of profitability and cost across the industry value chain.

Competitive Rivalry: The Cell and Gene Therapy report incorporates the detailed analysis of the leading organizations and their thought process and what are the methodologies they are adopting to maintain their brand image in this market. The report aides the new bees to understand the level of competition that they need to fight for to strengthen their roots in this competitive market.

Principal Research:

The research team works with industry experts from the Global Cell and Gene Therapy industry including the management organizations, processing organizations, value chain analytics by service providers of the Cell and Gene Therapy market.

Subordinate Research:

In the Secondary research vital information about the Cell and Gene Therapy industries value chain, total pool of key players, and application areas. Market separation is done as per the industrial drifts to the deepest level, terrestrial markets and key developments from both market place and technology-oriented viewpoints.

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This report forecasts revenue growth at a global, regional & country level, and provides an analysis of the market trends in each of the sub-segments from 2019 to 2026.

North America (USA, Canada, Mexico, etc.)

Asia-Pacific (China, Japan, India, Korea, Australia, Indonesia, Taiwan, Thailand, etc.)

Europe (Germany, UK, France, Italy, Russia, Spain, etc.)

Middle East & Africa (Turkey, Saudi Arabia, Iran, Egypt, Nigeria, UAE, Israel, South Africa, etc.)

South America (Brazil, Argentina, Colombia, Chile, Venezuela, Peru, etc.)

In addition, the Cell and Gene Therapy market research will help solve the following problems:

Ultimately, Cell and Gene Therapy reports provide detailed information and expert analysis on key consumer trends and behaviours in the market, as well as an overview of market data and key brands. Cell and Gene Therapy Market Reports provide all data with easy-to-understand information

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Cell and Gene Therapy Market to Exceed Revenues Worth US$ By the End of 2020 2027 - Melanian News

Experts at Newcastle University have shown that the rate of kidney disease in people with Joubert syndrome is determined by the genetic makeup of the individual and each patient may respond differently to the treatment.

Joubert syndrome is a complex disorder, affecting approximately one in 80,000 newborns, causing varying degrees of physical, mental and sometimes visual impairments. It is often associated with severe kidney disease that requires dialysis and ultimately transplantation.

The study, published online in the Proceedings of the National Academy of Sciences, is the first time that an explanation has been given for the difference of disease progression in Joubert syndrome patients.

Significant breakthrough

The Newcastle research has identified a second gene called BSND a 'modifier gene' which determines the severity of kidney disease in patients with CEP290 mutations of Joubert syndrome.

It has been assumed that these modifier genes exist, but they have never been found before in rare genetic conditions until now.

Professor John Sayer and Dr Colin Miles, from the Translational and Clinical Research Institute, Newcastle University, led the Medical Research Council-funded research.

Professor Sayer said: "We have shown, using mouse and human DNA samples, that BSND is a modifier gene for the severity of kidney disease in Joubert syndrome.

"This is the first time that a modifier gene for inherited kidney disease has been identified, and this information will improve diagnoses and will be used to develop therapies to reduce the severity of kidney disease in affected patients.

"Our research is a major step forward and, in the future, we may be able to offer a therapy that switches on the protective modifier gene and reduces the development of genetic kidney disease.

"This work paves the way towards personalised therapies in patients with inherited kidney disease."

The international study used mouse models and DNA samples from patients with Joubert syndrome to progress the research.

Scientists used mouse models of disease and genetic manipulation to see how the kidney disease responded to modifier gene manipulation, cross-referenced with DNA sequencing data from patients around the world to prove the modifier gene was relevant in humans.

Challenging disease

Professor Sayer, a Consultant Nephrologist at Newcastle Hospitals NHS Foundation Trust, said: "The treatment of genetic kidney disease is challenging, as this requires both the correction of the underlying gene defect and the delivery of the treatment.

"We have shown that the kidney disease in a mouse can be dramatically changed by switching on or off a modifier gene.

"This will mean that we can use this information to carry out treatments, including genetic therapies, to lessen the effects of inherited kidney diseases, such as Joubert syndrome.

"We are testing these treatments further in our model systems before we move into patient studies."

Within the next three years, research will start to test the treatment of patients with modifier genes in the hope of developing personalized treatment plans.

Patient story

Siblings Emma, 11, and Ben Buckley, eight, have Joubert syndrome and both developed kidney failure before the age of eight.

They were diagnosed with Joubert syndrome from a few months of age and both have required dialysis and a kidney transplant.

They suffer from a range of medical issues due to Joubert syndrome, including visual impairment, communication problems, and developmental delay.

The two children, of Whitley Bay, North Tyneside, have been instrumental in helping further the research over the years, allowing the Newcastle scientists to study the mutation in detail.

Parents Leanne and Michael say they welcome the findings of the Newcastle University-led study as it will help to give patients a chance of preventing kidney failure in the future.

Leanne said: "It is very important that research is done into Joubert syndrome and the linked kidney damage, as this will hopefully prevent patients in the future needing a kidney transplant.

"All throughout Ben and Emma's lives, they have lived with the effects of Joubert syndrome and scientists found they had a problem with the CEP290 gene.

"Both Ben and Emma have needed dialysis and kidney transplants because of their kidney problems and I would like to hope this research will help prevent kidney failure for other affected children.

"We were happy for Ben and Emma to provide samples for the study as anything that helps further understanding into the condition is well worth doing, so it's great to see the study's positive results."

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Discovery of gene that modifies severity of inherited kidney disease - Devdiscourse

About a decade ago, the ill-fated therapeutic cancer vaccine Provenge was approved, eventually bankrupting its developer Dendreon. Since then, a number of drugmakers have seen similar efforts splutter and fizzle, although the emergence of immunotherapies checkpoint inhibitors and CAR-T drugs offered a glimmer of hope in resuscitating the field. Banking on that promise is PDC*line Pharma, which secured a 20 million injection on Wednesday.

Spun out of the French Blood Bank, the Belgian-French biotech is developing off-the-shelf cancer vaccines that are based on a therapeutic cell line of plasmacytoid dendritic cells, which are sometimes referred to as the Swiss army knife of immune cells due to their diverse range of function. PDC*line Pharmas technology is being developed to be synergistic with checkpoint inhibitors.

Scientists initially hoped that turbocharging the immune system to battle cancer cells would be enough to shrink tumors. But that didnt quite occur since cancer cells possess the ability to put brakes on that immune assault.

In the case of Provenge, data showed the product helped prostate cancer patients live longer but there was no evidence of tumor shrinkage or cancer cell death. Still,Wall Street tagged it with blockbuster expectations. But its adoption was restricted by its complex autologous therapeutic administration, high price tag and changing treatment landscape. Since then, a number of other therapeutic cancer vaccine makers have crashed and burned including Argos Therapeutics and Bavarian Nordic.

A few years ago, the emergence of checkpoint inhibitors which are engineered to unleash the immune system emerged as the perfect partner in crime for the therapeutic cancer vaccine. That is what PDC*line Pharma hopes its vaccine will accomplish.

Its lead product is currently in a Phase I/II study in patients with the most common form of lung cancer. Enrollment is expected to be completed by 2022, chief Eric Halioua told Endpoints News.

Provenges complicated autologous administration, cost of goods, batch-to-batch variability and underwhelming efficacy all contributed to its tepid sales, Halioua suggested, indicating that PDC*line Pharmas plan to incorporate a checkpoint inhibitor into the equation would likely be beneficial.

You need a good vaccine to activate (the) immune system, and you need something to break the defense mechanisms of the tumor. And all the first generations of vaccines were not in these situations.

Although the company is initially testing its off-the-shelf vaccine as a monotherapy, eventually a checkpoint inhibitor will be added to the mix.

But caution is warranted: Efforts to combine therapeutic cancer vaccines and checkpoint inhibitors have also met with failure. Last month, French biotech Transgene abandoned its therapeutic lung cancer vaccine after mid-stage data showed that when tested alongside Bristol-Myers Opdivo, the combination did not significantly shrink tumors.

Altogether PDC*line Pharma which recently signed a 108 million licensing deal (plus royalties) for its lung cancer vaccine in South Korea and other Asian regions has raised more than 30 million since its inception in 2014.

In this latest round of Series B funding, PDC*line Pharma lured five new investors: Korean Investment Partners, as well as two South Korean funds Shinhan-Cognitive Start-up Fund and UTC 2019 BIOVENTUREFUND in addition to two Belgian funds, SRIW (The Regional Investment Company of Wallonia) and Sambrinvest (the investment fund of Charleroi).

The new investors joined existing investors SFPI-FPIM, the Belgian Federal Holding and Investment Company, Noshaq Group, the Financire Spin-off Luxembourgeoise/INVESTSUD Group, among other undisclosed names.

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French-Belgian biotech banks 20M to break ground in blood-splattered field of therapeutic cancer vaccines - Endpoints News

New research has found that the cell and gene therapy clinical environment in the UK has encouraged commercial sponsorship from around the world.

Researchers have found that the UK accounts for over 12 percent of global cell and gene therapy clinical trials. Another finding revealed that these trials have increased by approximately 45 percent in the UK compared to 2018.

According to a report published by Cell and Gene Therapy Catapult (CGT Catapult), international companies are also recognising the appeal of the UK cell and gene therapy environment. The findings show they are sponsoring the majority of UK commercial clinical trials, which account for 77 percent of the total 127 ongoing trials. This is an increase from the 25 percent of commercially sponsored trials in 2013.

The researchers suggest that the National Health Service (NHS) and UK ecosystem are providing the right platforms to allow innovative therapies to progress through to the clinic in ever increasing numbers.

Keith Thompson, CEO of CGT Catapult said: The total number of cell and gene therapy clinical trials in the UK has been increasing consistently by an average of 25 percent year-on-year since 2013. This has been enabled by the development of the UKs fantastic ecosystem to support the development and clinical adoption of cell and gene therapies. The infrastructureand initiatives that have been put in place, with strong backing by the government, including the Advanced Therapy Treatment Centrenetwork, are giving companies the confidence to setup and run their innovative clinical studies here. The result is that we are now seeing therapies moving from academic projects towards becoming commercial products that can be delivered at scale by the NHS.

The report highlights that the main indication for cell and gene therapy clinical trials remains oncology at 39 percent, followed by 13 percent ophthalmology and 12 percent haematology.

Health Minister Baroness Nicola Blackwood said: These extraordinary figures show the UKs life sciences sector is leading the world in getting cutting-edge treatments to NHS patients as quickly as possible.

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UK accounts for 12 percent of global cell and gene therapy clinical trials - European Pharmaceutical Review

GenScript Biotech Global Forum Highlights Advances in Cell and Gene Therapy and Opportunities in China

GenScript Biotech Corp., a leading global biotechnology group and a pioneer in the field of gene synthesis, held its inaugural "Global Forum on Cell & Gene Therapy and the Booming China Market," during the JP Morgan Healthcare Conference week, attracting hundreds of industry leaders, investors and others to address the challenges and opportunities in this innovative field.

"As an industry, we are on the brink of achieving some extraordinary breakthroughs in cell and gene therapy for cancer and other diseases," said GenScript Biotech CEOFrank Zhang, PhD. "Four gene and cell therapies have recently been approved by the FDA, bringing new hope to patients, and this is only the beginning. Our vision is to make cancer a chronic or curable disease rather than a deadly one, and to transform the treatment of cancer, autoimmune and other diseases by leveraging the advantages of cell and gene therapy."

While significant advances are being made, the Forum also tackled some of the more pressing challenges, such as mitigating treatment side effects, improving treatment efficacy in solid tumors and scaling up manufacturing. Panelists from Kite Pharma, GE Healthcare Life Sciences, Ziopharm Oncology, Oxford Biomedica, Genethon, CARsgen Therapeutics, J&J Innovation Asia Pacific, the American Society of Gene & Cell Therapy, Loncar Investment, Lilly Asia Ventures, and many others participated in the event.

In the U.S. alone, the U.S. Food and Drug Administration is expected to approve 40-60 cell and gene therapies by 2030. During a panel discussion focused on regulatory issues, experts considered what regulators will need to do to keep up with the rapid pace of innovation, the new hospital-based regulatory pathway inChina, how to ensure quality through the manufacturing process, and the challenges and opportunities that come with regulatory harmonization among different countries.

Chinacontinues to attract significant attention from industry and investors and is poised to grow even more. During his welcoming remarks, Zhang notedChina'semergence as a global economic leader, with a projected$1.1 trillionspend on healthcare this year, as well as the growing disease burden inChina. By 2030, an estimated 4.3 million Chinese will be diagnosed with one of the 14 major cancers, according to research from IMS Health. Panelists addressed issues such as the amount of capital required to achieve scale inChina, and advantages of the market inChina.

"The drug development business is changing rapidly andChinais at the fore in a number of ways," Zhang said. "Biotech and pharma companies do not need or desire to have the infrastructure to scale their drugs through commercialization. With lower costs,Chinais a natural place for companies to contract out costly development and manufacturing to organizations that have the expertise and experience to collaborate with them through the entire discovery to development lifecycle."

For its part, GenScript has put significant resources into its Contract Development and Manufacturing Organization (CDMO) business to meet the increasing demand. In 2018, the company officially launched its biologics CDMO segment, and last year opened a new GMP compliant biologics research center. GenScript is also leading the way in cell therapy through its antibody discovery service and plasmid and virus production capabilities.

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Advances in Cell and Gene Therapy and Opportunities in China - BSA bureau

A new team at Medical College of Georgia is taking aim at defective gene sequences that cause disease using the revolutionary CRISPR method that allows precise editing of specific sequences to create animal models of those diseases.

Drs. Lin Gan and Joseph Miano sit at a conference table outside their new lab at Augusta University, and in the blink of an eye the discussion goes from how they began using a certain technology in its early days of 2013 to how they are applying the latest iteration that came out only last month.

Thats how fast they, and their field of genome editing and manipulation, are moving.

Gan and Miano and colleague Xiaochun Long were recently recruited as a team to the Medical College of Georgia at AU, where Gan became the founding director of the Transgenic and Genome Editing Core. He is a Georgia Research Alliance Eminent Scholar in Neuroscience, and Miano is a J. Harold Harrison Distinguished University Chair in Vascular Biology.

The floor of their lab shows they are only a few months into the new stint in Augusta.

Were still living out of boxes, Miano said as he stepped around cardboard boxes.

The two have been together since the early 1990s in Houston and spent the past 20 years working together at the University of Rochester, though they had no idea they were both headed there initially.

Unbeknownst to me, he was being recruited at the same time, Miano said.

Were meant to be together and we didnt even coordinate our move, Gan said jokingly.

The move to Augusta was deliberate. Miano had interviewed at MCG in 1995 and wanted no part of it then and was not interested again after recently giving a talk at the school and receiving some initial offers.

But then I came down for a second visit and I thought, Hmm, this is interesting, Miano said. They have really built it up well. Theres an opportunity here to make a difference.

But the three were a package deal Long and Miano are now married, and Gan would not have come without them.

I also see this as an opportunity, Gan said. I can definitely have more support here for the Core.

He was quite familiar with MCG and in particular the eye researchers there, which is also an interest for him.

There is a very strong eye group here, Gan said, and he has been supplying them with animal models of disease for more than a decade. Creating those animals models, where a gene may be knocked out or silenced, got a lot easier for him with the advent of CRISPR, or clustered regularly interspaced short palindromic repeats.

It is the ability to take a short length of genetic material known as a guide RNA, which will match up with a highly specific target in DNA and in many instances attach an enzyme that then precisely cuts the DNA at that spot, allowing researchers to target very specific sections of that DNA and either silence genes or in some cases begin the replacement of defective sections.

Gan and Miano use CRISPR to create animal models in mice of human disease that other researchers can use to study those diseases. They often involve a small mutation in the genetic material that gives rise to the condition, known as a single nucleotide polymorphism or SNP.

CRISPR is very effective for SNPs, for mutations, Gan said. It is a little quicker.

There are a lot of them, and more are being added all of the time, Miano said.

The latest database of SNPs, that last time I looked was over 600 million, he said.

Miano was at the conference in 2012 when Dr. Jennifer Doudna of the University of California, Berkley made what he called a jaw-dropping revelation that a bacterial defense system against viruses allows for the precise targeting and cutting of DNA. CRISPR soon became a hot new technique that he and Gan adopted the following year.

Though they are primarily working in mice, CRISPR can be used in other animal models and outside the field as well, Miano said.

I think what is exciting, as Lin says, anything with a genome can be edited, he said. The agricultural field is blowing up. There are all kinds of stuff going on in agriculture with editing.

It has already reached the human level. The National Library of Medicines database of clinical trials involving CRISPR-aided therapy or testing lists 20 active studies, although only a handful are in the U.S. and most are in China. Therapies are most likely to focus first on what Miano called the low-hanging fruit, the 7,000 or so diseases that arise from a single gene defect, such as cystic fibrosis.

More complex conditions, such as heart disease, are going to be tough, he said.

There is no silver bullet for those, but CRISPR will be at the center of that work, he said.

For his part, Gan would like to focus on the eye. The tissue there is thinner and more concentrated, so correcting a defect in vision in, for instance, the macular area of the retina at the back of the eye involves manipulating much fewer cells than trying to correct something in the brain or elsewhere.

This is great for manipulation because a lot of our vision comes from the macular region, Gan said. Its a very small region. That is why he believes in terms of therapies, the eye will be one of the first.

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Genome editing heralds new era of disease research, therapy - The Augusta Chronicle

SAN FRANCISCO Novartis sparked a new round of drug pricing criticism last May when the Swiss pharma revealed its new gene therapy Zolgensma would cost $2.1 million per patient.

In the eight months since then, however, insurers have reimbursed treatment for all but one eligible infant who have received the one-time therapy, according to company CEO Vas Narasimhan, speaking Monday at the J.P. Morgan Healthcare Conference.

Commercially, it seems, Novartis' launch has gone according to plan.

But the drugmaker is still awaiting the Food and Drug Administration's judgment on a scandal that erupted last summer following revelations preclinical testing data for Zolgensma had been inappropriately altered. And a clinical hold from the regulator on another study of the gene therapy has slowed plans for expanding treatment into older children.

Resolving both will be key tests in 2020, as will be expanding newborn screening for spinal muscular atrophy, the genetic disease that Zolgensma treats.

David Lennon

Novartis

This year could also feature the start of human testing for two new gene therapies from AveXis, the biotech developer of Zolgensma that Novartis bought in 2018 to gain access to its drug pipeline.

AveXis President David Lennon spoke with BioPharma Dive about the challenges the business is facing, along with how Novartis' ownership has accelerated its ambitions.

This interview has been condensed and edited for clarity.

What's the latest progress on newborn screening for spinal muscular atrophy?

LENNON: It's still a key priority with the states, as it was approved last July. There's been a lot of progress. Sixteen states have implemented newborn screening that represents about 32% of new births in the U.S. that are screened. That's our internal estimate of what we see. We estimate that number will get to 70% over the course of this year as more states implement it.

There's an amazing impact when you have newborn screening. In states where we have newborn screening, [there is] a two- to three-fold higher utilization of gene therapy for newborns than in states where we don't. A lot of that has to do with, when newborn screening gets set up, a dedicated referral pathway to the best institutions also gets created. The parents are informed of the diagnosis and get referred to a geneticist to get counsel.

Do you think the newborn screening process should be reformed, given the state-to-state disparities?

LENNON: It's a travesty. You're just waiting for these kids to present clinically. The bureaucracy that limits the adoption of these kinds of policies is silly in many cases.

RUSP [The Recommended Uniform Screening Panel] recommends and has a two-year implementation requirement that's soft, there's no enforcement around it. It's up to the states to put it in place. Implementation often takes active patient groups and an active physician to advocate for it getting into the state.

How do you grade Novartis' job on communicating Zolgensma's price to payers and the public?

LENNON: With payers, which were the folks we had to really convince, we did an outstanding job. Payers understand the value it provides, they readily wrote policies to cover the product even with a broad label at launch, very positive discussions and good coverage in general very early. I'd give us strong marks on the payer side.

In the public domain, I think it's more problematic. Healthcare cost transparency and understanding of healthcare costs in the general public is not great. It's very easy to just go to the extreme and say, '$2.1 million, I don't understand; that must be ridiculous.' If I was to say it cost $2 million to do a heart transplant, people might say 'I totally get that.'

We anticipate about 300 or 400 [will be treated with Zolgensma], basically the same number of pediatric heart transplants that get done every year. When you start to try to draw some comparisons, it quickly gets lost in the 'but it's $2.1 million dollars.' We have to do a better job of helping people generate relevant transparency that allows us to put these things in context.

How did you choose the pipeline products to prioritize?

LENNON: Our next two programs take on different challenges and opportunities. One is Rett syndrome, where organic [gene] expression needs to be much more targeted. If we overexpress this protein, you actually create a similar syndrome to the one we are trying to address. There's a kind of 'Goldilocks' area of expression. That program is using a promoter that is more controlled and has a natural feedback mechanism that limits the amount of expression.

The third program, called AVXS-301, is for a genetic form of Lou Gehrig's disease amyotrophic lateral sclerosis or ALS. That is actually an inhibitory construct. The first two programs we've had are gene replacement the gene's defective, we're adding back protein. In this case, the defect leads to overexpression or active expression of a mutated form of SOD1 that drives inflammation and degradation of the neurons of these patients. We are inhibiting that by introducing a short hairpin RNA that actually inhibits the expression of the defective gene.

Those programs existed as part of the acquisition of AveXis. Since then, we started partnering with [the Novartis Institutes of Biomedical Research] to apply this platform to different diseases. The first one we announced is a project for Friedreich's ataxia, a muscle-wasting disease.

You took over AveXis in 2018, coming from Novartis. What was AveXis particularly skilled at doing, and what's an area that Novartis' ownership could help in?

LENNON: Technically, this organization was very skilled at taking a problem, breaking it down, developing options to address it and agreeing on the best solution forward. It was an organization that was optimistic it could continually find that. Whether you believe in optimism or luck, the organization was very effective at finding that.

Where the organization really benefits from the Novartis backbone is then: How do you take the process that you developed and make that an industry-leading, highly reproducible and globally scalable system? That's where Novartis is brilliant. That experience has allowed us to bring in quality systems, management talent that knows how to scale, and financial wherewithal that allows us to scale to now multiple sites around the country.

When you look at AveXis, we could do one thing at a time. Now we can start to do seven or eight things at a time, because Novartis has the backbone and scale because they're used to managing 143 projects at once.

Ned Pagliarulo contributed to this article.

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Novartis' David Lennon on next steps for AveXis, selling Zolgensma's price to the public - BioPharma Dive

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January 13, 2020

We expect that a satellite cell with the corrected DMD gene would quite quickly and continuously propagate the edited gene throughout the muscle tissue, said Prof. Amy Wagers, who leads the research. (Photo credit: Jon Chase/Harvard Staff Photographer.)

Cambridge, Mass. January 13, 2020 Harvard University stem cell researchers led by Amy Wagers, PhD, are embarking on a major study of Duchenne muscular dystrophy (DMD). Supported by research funding from Sarepta Therapeutics, under a multi-year collaboration agreement coordinated by Harvards Office of Technology Development (OTD), the project aims to use in-vivo genome editing, in mouse models of DMD, to fully and precisely restore the function of the dystrophin protein, which is crucial for proper muscular growth and development. Approaches validated by this work may point the way to an eventual therapeutic strategy to reverse DMD in humans.

Duchenne muscular dystrophy is a genetic disease caused by the lack of a protein called dystrophin that normally helps to support the structural integrity of muscle fibers, including those in the heart. Without the dystrophin protein, cells are weaker and degenerate more quickly. Over time, affected individuals boys, typically, as it is a recessive X-linked disorder lose their capacity to move independently.

Its really a devastating disease; it robs young boys of their capacity to be young boys, said Wagers, who is the Forst Family Professor of Stem Cell and Regenerative Biology, Co-Chair of the Department of Stem Cell & Regenerative Biology, and an Executive Committee Member of Harvard Stem Cell Institute. Though it is early days, Im hopeful that through this work we may identify and validate new avenues for therapy to completely rescue the proper expression and function of the dystrophin protein and regenerate healthy muscle tissue.

Researchers worldwide have pursued a variety of promising approaches such as cell and gene therapies, small-molecule therapies, and others to lessen or prevent the disease and improve patients quality of life.

The strategy pursued by the Wagers Lab at Harvard aims to fully correct the genetic template for dystrophin at its source, in the DNA of stem cells (satellite cells) that create and regenerate muscle cells. Combining cutting-edge CRISPR/Cas9 genome editing technologies with a deep knowledge of stem cell science and regenerative biology, this approach if successful might offer a permanent restoration of muscular function.

In skeletal muscle, muscle fibers are terminally post-mitotic, meaning they cannot divide and they cannot reproduce themselves, Wagers explains. If you lose muscle fibers, the only way to produce new muscle is from stem cells, specifically the satellite cells. The satellite cells are self-renewing, self-repairing, and ready to spring into action to create new muscle fibers. So we expect that a satellite cell with the corrected DMD gene would quite quickly and continuously propagate the edited gene throughout the muscle tissue.

At present, research conducted in mice has shown promising results. In June, the Wagers Lab published the results of editing stem cells in vivo, demonstrating that stem cell genes can be edited in living systems, not only in a dish. In that work, Wagers and her team delivered genome editing molecules to the cells using adeno-associated viruses (AAVs). Her lab has also successfully used gene editing in heart, muscle, and satellite cells to partially restore the function of the DMD gene that encodes dystrophin, by chopping out faulty sequences of code that are disrupting the proper reading frame.

The new stem-cell approach pursued in collaboration with Sarepta would build on these achievements and use more precise genome editing approaches, in animal models of DMD, to entirely replace genetic mutations in the DMD gene with correctly encoded sequences. The project will also explore alternate delivery methods and strategies to mitigate immune effects of in vivo genome editing.

This ambitious project will benefit greatly from the resources and insights of a company with deep clinical experience in the development of therapeutics for muscular dystrophy, said Vivian Berlin, Managing Director of Strategic Partnerships at Harvard OTD. Preclinical discoveries by Harvard researchers may open entirely new possibilities for lifesaving treatments in the long run, offering much-needed hope to patients and families in the future. Were grateful to be able to sustain the important momentum already established in Prof. Wagers lab, through this collaboration.

As we work to bring forward new treatments for patients with DMD, Sarepta is excited to support Prof. Wagers and her lab to accelerate the development of a gene editing approach, which has shown significant potential in early studies, said Louise Rodino-Klapac, Sareptas Senior Vice President of Gene Therapy. This multi-year collaboration is part of Sareptas broader commitment to pursuing all therapeutic modalities and advancing our scientific understanding of gene editing in order to maximize the potential of this approach to help patients.

Under the terms of the agreement between Harvard and Sarepta, the company will have the exclusive option to license any arising intellectual property for the purpose of developing products to prevent and treat human disease. As with any research agreement facilitated by OTD, the right of academic and other not-for-profit researchers to use the technology in further scholarly work is preserved.

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Muscular dystrophy collaboration aims to correct muscle stem cells' DNA - Harvard Office of Technology Development

19 January 2020 11min read

But ethical and economic challenges could limit the benefits medtech brings to healthcare.

The Nobel Prize in Medicine 2019 was awarded to three men for their discoveries of how cells sense and adapt to oxygen availability. The Nobel Prize in Medicine 2018 was given to two men for their discovery of cancer therapy by inhibition of negative immune regulation. The Nobel Prize in Chemistry 2017 went to a trio for developing cool microscope technology that revolutionises biochemistry by allowing researchers to study three-dimensional structures of biomolecules in their search for a cure for the Zika and other viruses. One of the three chemistry winners of 2017, Joachim Frank of the US, said at the time that he thought the chance of winning a Nobel Prize was minuscule because there are so many innovations and discoveries happening.

Frank is still right about that. Technological leaps in medicine dubbed medtech are accelerating as researchers find better ways to treat more diseases, in more ways, for more people. Advances are occurring in biotechnology, immunotherapy, surgery, and foetal and neonatal care to name just some areas. Artificial-intelligence software trained on data from digitalised health records and devices can spot problems faster and more reliably than can humans. HCA Healthcare, the largest for-profit hospital operator in the US, for instance, now uses algorithms trained on 31 million cases to detect the sepsis infection that kills about 270,000 people a year in the US.

More medtech advances are certain. Money is pouring into research and development overseen by regulators and doctors to ensure benefits outweigh risks. Common sights soon might be robot physicians, remote surgery and mini 3D-printed organs. Bacteria genetically reprogrammed to destroy tumours in mice could one day work on humans. Genome scans and gene therapies could become routine.

For all this promise, however, medtech comes with two certain and one likely drawback. The first definite disadvantage is that medtech is raising ethical issues that could stop the deployment of key advances. The two most sensitive are the gene-editing of foetuses (superbabies) that could alter human experience and protecting the privacy of patient data, an issue highlighted in November when it emerged that US healthcare provider Ascension had secretly handed over the records of tens of millions of patients to data crunchers at Google. Medtechs other certain shortcoming is the cost. Many advancements might never become mainstream because they could prove too expensive for governments burdened with budget deficits and heavy debt loads that are already facing rising healthcare costs as their populations age.

Medtechs contentious disadvantage is doctors are finding that self-monitoring via devices, which often detects harmless abnormalities and fuels hypochondria, is leading to unwarranted anxiety, incorrect diagnoses and unneeded treatments. All up, medtechs value to society will be tied to the extent to which these disadvantages limit the spread of its unquestionable benefits.

Many of medtechs ethical issues could be resolved, to be sure, but that wont be easy. Some medtech advancements, especially those based on AI, are economical. Medtech needs to be assessed with the perspective that there is much it is not solving. Medtech advances, for example, arent enough to avert the recent decline in life expectancy in western countries due to heart attacks tied to obesity. Medtech pharmaceutically does little for autoimmune diseases such as arthritis that afflict one in four US adults though it is improving joint-replacement surgery. Researchers are yet to find a cure for infections made drug-resistant due to the overuse of antimicrobial drugs that the World Health Organisation says could kill 10 million people a year by 2050. Nothing medtech has come up with is usurping MRI scans and X-rays.

Be these as they may, medtech advancements are ushering in treatments that produce better outcomes for patients. Only time will tell how much ethical, economic and other possible drawbacks limit mainstream access to medtechs benefits.

The biotech era

Eras become known for their medical advancements. From the 1920s to the 1950s, for example, the key medical leaps were vaccines and antibiotics. Later epochs might regard todays advances to be centred on cell and gene therapy, robotic surgery and perhaps AI.

Hope for cures from gene therapy, an area of research that emerged from the late 1980s, accelerated in the early 2000s when the human genome was sequenced. And treatments are underway now and more are likely. Biopolymers (nucleic acid) are injected into cells to treat inherited eye diseases and immune deficiencies while researchers are studying how gene therapy could treat cancer, heart disease and diabetes. A stellar example of gene therapy improving lives is that a Novartis subsidiary has developed a one-time gene-based treatment (Zolgensma) that is a curing treatment for children born with spinal muscular atrophy (who without this advance constantly need treatment over their short lives). The problem is one dose costs US$2.1 million.

Aside from the costs, gene therapy comes with other challenges too. The finicky nature of genes has made progress slow. Other hindrances are rejection, side effects such as cancer, and the risk that other genes might be delivered to a cell. Some treatments are so risky authorities have halted them. Some breakthroughs have proved false a recent study debunks that a certain gene causes depression. That the ethical issues surrounding gene therapy are unresolved became an urgent issue in 2018 when two Chinese babies were born with modified genes.

Inventions to assist surgeons have proved faster to everyday use (and less problematic). Robots have aided orthopaedic surgeons since the mid-1980s and now help with general, transplant, urological and other procedures. One measure of their widespread use is that Intuitive, the US-based maker of the 1999-launched da Vinci surgical system, counts that tens of thousands of surgeons have conducted more than six million procedures in at least 66 countries using its equipment. The benefits of robotic-assisted surgery are less invasive, more precise and safer procedures due to fewer and tinier incisions (microsurgery) and reduced human error.

While less-invasive surgery shortens hospital stays and robotic surgerys lower margins of error reduce the need and costs of further treatment, robotic-assisted procedures are expensive. Assuming cost issues can be overcome, technology will expand its role in surgery and robots could use AI more extensively to help surgeons make more decisions.

AIs use in healthcare goes well beyond surgery too. AI programs including chatboxes are diagnosing heart disease and cancer, identifying retinal damage, analysing suicide risk, streamlining drug-development processes, proposing remedies for multiple sclerosis, even helping the dumb speak. AI s promise is more timely, economical, convenient and streamlined treatments.

AIs usual drawbacks apply, however. Personal data needs privacy protection, which can impede research. Data can be dodgy and data-training algorithms can be flawed and biased, which could lead to misdiagnosis. AI is vulnerable to hacking, whereby malicious tweaks lead to errors. AIs deployment often runs ahead of peer review and ethical considerations.

A neurotic world?

One medtech achievement is to elevate the practitioner Doctor Me. The term (sometimes stated as Doctor You) is for when people use devices and self-testing to monitor their health or genetic risks.

Self-monitoring comes with many advantages. It can save lives. The unwell can gain comfort if their vital signs are normal. The data collected can help everyones health and allow people to find others with similar issues, which could provide clues for treatments and moral support.

The problem, however, is that Doctor Me has ushered in the nocebo effect, essentially a form of hypochondria. The nocebo effect occurs when patients think they are experiencing a side effect to a greater degree than possible or when people fret they are suffering from an ailment that a test showed they are at risk of say people self-tested as prone to Alzheimers imagine they have the affliction when they forget something.

A Stanford study of 2018 found the nocebo effect is ripe in self-testing genetics, a flagship area of medtech that is not foolproof. The expression could become ubiquitous soon because more people are testing their disposition to Alzheimers, cancer and obesity by 2017, already one in 25 in the US knew their genetic data. If the nocebo effect becomes widespread, authorities may need to limit self-testing.

While future Nobel Prizes await those making medtech advances, perhaps others lie ahead for those who find ways to resolve medtechs ethical, economic and hypochondriac challenges.

Published by Michael Collins, Investment Specialist, Magellan Group

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Technology is reshaping modern medicine - TheBull.com.au

As the annual JP Morgan Healthcare Conference draws to a close, lets celebrate the remarkable innovation that drives the biopharma industry. Heres a look at the top 10 companies noted in the BioSpace Ideal Employer 2019 survey for being the most innovative and what theyve been up to recently.

Regeneron Pharmaceuticals. Ranked as the top innovator in the survey, Regeneron has long had a reputation for innovation, and is often cited as Sanofis innovation engine because of its numerous collaborations with the French-based company. Regeneron has seven marketed products, including Arcalyst for rare autoinflammatory disease, Eylea for a common cause of blindness, Praluent for high cholesterol, and Dupixent for atopic dermatitis. It also has REGN-EB3, a three-antibody therapy used to treat Ebola.

On January 9, Regeneron announced results from LUMINA-1, a Phase II trial of garetosmab in patients with fibrodysplasia ossificans progressive (FOP), an ultra-rare genetic disorder that leads to abnormal bone formation. After 28 weeks, the drug decreased total lesion activity compared to placebo by 25%.

Verily Life Sciences. Formerly known as Google Life Sciences, Verily is Alphabets life science research company. It was originally a division of Google X. At this point, the company doesnt have any marketed products and its not completely clear on the companys overall focus. It has numerous partnerships, such as one with Sanofi to develop products for managing diabetes, a disease-detecting nanoparticle platform called project Tricorder, and a partnership with Johnson & Johnson on surgical robotics. It also has partnerships with Alon, 3M, Allergan, Biogen, Dexcon, GlaxoSmithKline, Mayo Clinic, Brigham and Womens Hospital and many others.

On December 23, 2019, Verily partnered with Emory Healthcare to deploy new solutions to help improve cost-effectiveness, operational efficiency and quality. This deal with utilize Emorys academic medical center and partner with Verilys expertise in data science, analytics, user experience and product development. The initial focus is a deep analysis of existing drugs and lab-ordering patterns at Emory.

Illumina. Illumina develops, manufactures, and markets laboratory devices, with particular emphasis on DNA sequencing, genotyping, gene expression and proteomics. On January 4, 2020, Illumina announced a 15-year, non-exclusive deal with Roche. Not only will that increase the availability of next-generation sequencing-based in vitro diagnostic (IVD) tests on Illuminas systems, but the two companies will collaborate to complement Illuminas pan-cancer assay TruSight Oncology 500 (TSO 500) with new companion diagnostic (CDx) claims.

That deal came only a short time after Illumina canceled a $1.2 billion merger with another next-generation sequencing company, Pacific Biosciences (PacBio). Illumina decided the deal was not likely to be approved by antitrust regulators in the U.S. and UK. Illumina holds about 80% of the global DNA sequencing market.

bluebird bio. Bluebird bio focuses on the nascent field of gene therapy. Currently its sole approved product is Zynteglo. It was approved by the European Commission (EC) on June 14, 2019 for patients 12 years or older with transfusion-dependent beta-thalassemia who did not have a 0/0 genotype and for patients where hematopoietic stem cell (HSC) transplantation wasnt appropriate, but a human leukocyte antigen (HLA-matched related HSC donor isnt available. Its a little difficult to mention bluebird without mentioning the price of Zynteglo, which is $1.8 million in Europe. In addition to its scientific innovation, bluebird bio is innovative in terms of pricing structure. Zynteglos price is spread out over five years, with an initial upfront price of 315,000 euros with the four additional yearly payments due only if the treatment continues to work.

The product launched in Germany on Jan. 13, 2020. Bluebird initiated the rolling Biologics Licensing Application for Zynteglo in the U.S. and is currently in discussions with the U.S. Food and Drug Administration (FDA) on the timing and various components of the submission. They hope to complete the BLA submission in the first half of this year.

Biogen. Biogen specializes in therapies for central nervous system disorders. Some of its most well-known products are Alprolix for hemophilia B, Avonex, Fampyra, Tecfidera and Tysabri for multiple sclerosis, and Spinraza for spinal muscular atrophy (SMA). However, most recently, the company has been in the headlines for its aducanumab for Alzheimers disease. The drug was declared a failure in March 2019, but was resurrected this year after some of the later trial data showed effectiveness at the highest dose. Still, its not a slam dunk to be approved by the FDA and there are plenty of skeptics.

Analysts expect Biogen to submit aducanumab to the FDA in a matter of weeks, although the company is being tight-lipped about the timetable.

Bayer. Based in Germany, Bayer is one of the largest pharma companies in the world. In 2018, Bayer acquired U.S.-based Monsanto, which no longer exists under the Monsanto name. The companys business units include Bayer Crop Science, Consumer Health, Pharmaceuticals, Animal Health and Business Services.

On Jan. 16, 2020, Bayer sold one of its last Germany-based manufacturing facilities to Shanghai, China-based WuXi Biologics. The plant will be run by WuXi Biologics and act as a backup site for the manufacture of Bayers Kovaltry (antihemophilic factor). The primary site for Kovaltry product is Bayers facility in Berkeley, California.

Novartis. Based in Switzerland, Novartis has a well-known portfolio of drugs, including Clozaril, Voltaren, Tegretol, Diovan, Gleevec, and Ritalin. Its Sandoz Division is a global leader in generic drugs and biosimilars.

Last year, the FDA approved Zolgensma, a gene therapy for SMA, which was developed by its subsidiary, AveXis. Although there was some controversy over data manipulation in preclinical studies, it was determined not to affect the safety or efficacy of the therapy.

In late November 2019, Novartis acquired The Medicines Company for $9.7 billion, only a week after The Medicines Company announced positive data from its ORION-10 Phase III trial for inclisiran for lowering cholesterol.

GlaxoSmithKline. Headquartered in London, GSK markets drugs for numerous major diseases, such as asthma, cancer, infections, diabetes and mental health. Its best-known drugs include Advair, Augmentin, Flovent, Lamictal and others.

At the recent JP Morgan Healthcare Conference, Emma Walmsley, GSKs chief executive officer, predicted it will have six regulatory approvals in the U.S. this year.

I am pleased with the progress and the momentum that weve been able to make over the past couple years, she told CNBCs Jim Cramer.

Walmsley noted positive data for a number of programs, including ones gained from its $5.1 billion acquisition of Tesaro Oncology in 2018. She also noted a two-drug regimen for HIV that the companys subsidiary ViiV Healthcare has been developing.

Genentech. Generally viewed as the first modern biotechnology company, Genentech is a subsidiary of Swiss-based Roche, although Genentech is based in South San Francisco. It has a laundry list of successful drugs, often in the oncology market, such as Avastin, Tarceva, Zelboraf, Kadcyla, Alecansa, Venclexta and Tecentriq. It also has antivirals, such as Xofluza, Hemlibra for hemophilia A, and Esbriet for idiopathic pulmonary fibrosis.

On Dec. 12, 2019, Genentech announced that its Phase III IMspire150 trial in patients with previously untreated BRAF V600 mutation-positive advanced melanoma, hit its primary endpoint of progression-free survival (PFS). It showed adding Tecentriq to Cotellic and Zeleboraf decreased the risk of the disease getting worse or death, compared to placebo plus Cotellic and Zelboraf.

Amgen. Based in Thousand Oaks, California, Amgens best-selling products are Neulasta, an immunostimulatory for patients undergoing chemotherapy, and Enbrel, used to treat rheumatoid arthritis and other autoimmune diseases. Other products include Epogen, Aranesp, Prolia and XGeva.

On Nov. 1, 2019, the company expanded its presence in China by taking a 20.5% stake in China-based BeiGene Co. Amgen paid $2.7 billion in cash for the stake. As a result of the deal, BeiGene will commercialize Xgeva, Kyprolis and Blincyto in China.

On Jan. 13, 2020, the company inked strategic collaborations with Guardant Health and QIAGEN to develop blood- and tissue-based companion diagnostics for investigational cancer treatment AMG 510. AMG 510 is the first KRASG12C inhibitor to advance to the clinic for multiple cancer types.

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The report presents an in-depth assessment of the Global Red Biotechnology including enabling technologies, key trends, market drivers, challenges, standardization, regulatory landscape, deployment models, operator case studies, opportunities, future roadmap, value chain, ecosystem player profiles and strategies. The report also presents forecasts for Global Red Biotechnology investments from 2020 till 2025.

Industry Overview-

The Red Biotechnology Market is expected to register a CAGR of 5.7% during the forecast period. Red biotechnology is a process that utilizes organisms to improve health and helps the body to fight against diseases. Red biotechnology has become a very important part of the field of diagnostics, gene therapy, and clinical research and trials. Genetic engineering and the development and production of various new medicinal products to treat life-threatening diseases are also part of the benefits of red biotechnology. Severe Combined Immune Deficiency (SCID) and Adenosine deaminase (ADA) deficiency are genetic disorders that were successfully treated with gene therapy. Several promising gene therapies are under development for the treatment of cancer and genetic disorders. According to the World Health Organization (WHO), approximately 6,000 to 8,000 rare diseases found and out of them, nearly 80% are genetic disorders. Rising incidence and prevalence of chronic and rare diseases and increased funding in the healthcare industry are the key driving factors in the red biotechnology market.

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Top Leading Manufactures-

Pfizer Inc, AstraZeneca PLC, F. Hoffmann-La Roche Ltd, Celgene Corporation, Takeda Pharmaceutical Company Limited, Biogen Inc, Amgen Inc, Gilead Sciences Inc, Merck KGaA, CSL Limited

Biopharmaceutical Industry Segment is Expected to Hold a Major Market Share in the Red biotechnology Market

- Biopharmaceuticals are medical drugs that are produced by using biotechnology. Biopharmaceuticals are proteins, antibodies, DNA, RNA or antisense oligonucleotides used for therapeutic or diagnostic purposes, and these products are produced by means other than direct extraction from a native (non-engineered) biological source.- The first biopharmaceutical product approved for therapeutic use was recombinant human insulin (Humulin), which was developed by Genentech and marketed by Eli Lily in the year 1982 and in the year 2019, Novartis received FDA approval for gene therapy product in the treatment of spinal muscular atrophy (SMA) condition. Using an AAV9 viral vector, called Zolgensma, which delivers SMN protein into the motor neurons of afflicted patients.- According to the World Health Organization (WHO), globally Cancer is the second leading cause of death and an estimated 9.6 million deaths in the year 2018.- Increasing incidence and prevalence of chronic and rare diseases and rapid expansion of the biopharmaceutical industries are the key driving factors in the biopharmaceutical industry segment.

North America is Expected to Hold a Significant Share in the Market and Expected to do Same in the Forecast Period

North America expected to hold a major market share in the global red biotechnology market due to the rising prevalence of chronic and rare diseases, increased expenditure in the healthcare industry in this region. According to the National Institutes of Health (NIH), in the year 2019, approximately 1.8 million people will be diagnosed with cancer in the United States and estimated 268,600 women and 2,670 men will be diagnosed with breast cancer. Moreover, the rise in the adoption of advanced technologies in gene therapy and increasing investments in research and development is fueling the growth of the overall regional market to a large extent.

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Key Strategic Developments : The study also includes the key strategic developments of the market, comprising R & D, new product launch, M & A, agreements, collaborations, partnerships, joint ventures, and regional growth of the leading competitors operating in the market on a Global and regional scale.

Key Market Features: The report evaluated key market features, including revenue, price, capacity, capacity utilization rate, gross, production, production rate, consumption, import/export, supply/demand, cost, market share, CAGR, and gross margin. In addition, the study offers a comprehensive study of the key market dynamics and their latest trends, along with pertinent market segments and sub-segments.

Analytical Tools: Global Red Biotechnology Market report includes the accurately studied and assessed data of the key industry players and their scope in the market by means of a number of analytical tools. The analytical tools such as Porter's five forces analysis, feasibility study, and investment return analysis have been used to analyzed the growth of the key players operating in the market.

The research includes historic data from 2014 to 2020 and forecasts until 2025 which makes the reports an invaluable resource for industry executives, marketing, sales and product managers, consultants, analysts, and other people looking for key industry data in readily accessible documents with clearly presented tables and graphs.

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Red Biotechnology Market Size, Status and Recent Advancements, Forecast 2020 to 2025 - MENAFN.COM

Axovant Gene Therapies (NASDAQ:AXGT) was upgraded by stock analysts at ValuEngine from a hold rating to a buy rating in a report released on Friday, January 3rd, ValuEngine reports.

A number of other equities research analysts have also commented on the company. Chardan Capital increased their price target on Axovant Gene Therapies from $10.00 to $15.00 and gave the company a buy rating in a research report on Monday, October 28th. Zacks Investment Research raised Axovant Gene Therapies from a hold rating to a strong-buy rating and set a $6.00 price target for the company in a research report on Wednesday, November 13th. One equities research analyst has rated the stock with a sell rating, one has issued a hold rating and nine have assigned a buy rating to the companys stock. The company currently has a consensus rating of Buy and a consensus target price of $24.66.

Shares of NASDAQ:AXGT opened at $4.43 on Friday. The company has a quick ratio of 1.41, a current ratio of 1.41 and a debt-to-equity ratio of 0.69. Axovant Gene Therapies has a twelve month low of $3.81 and a twelve month high of $19.60. The stocks 50 day moving average is $5.14 and its 200-day moving average is $6.11. The firm has a market capitalization of $105.98 million, a price-to-earnings ratio of -1.00 and a beta of 1.18.

Large investors have recently made changes to their positions in the business. Tower Research Capital LLC TRC grew its stake in shares of Axovant Gene Therapies by 955.3% during the 2nd quarter. Tower Research Capital LLC TRC now owns 4,221 shares of the companys stock worth $27,000 after acquiring an additional 3,821 shares during the period. Barclays PLC bought a new position in shares of Axovant Gene Therapies during the 3rd quarter worth $65,000. Jane Street Group LLC grew its stake in shares of Axovant Gene Therapies by 28.8% during the 2nd quarter. Jane Street Group LLC now owns 46,455 shares of the companys stock worth $289,000 after acquiring an additional 10,375 shares during the period. Woodstock Corp bought a new position in shares of Axovant Gene Therapies during the 4th quarter worth $83,000. Finally, BlackRock Inc. bought a new position in shares of Axovant Gene Therapies during the 2nd quarter worth $1,482,000. Hedge funds and other institutional investors own 14.80% of the companys stock.

Axovant Gene Therapies Company Profile

Axovant Gene Therapies Ltd., a clinical-stage gene therapy company, focuses on developing a pipeline of product candidates for debilitating neurological and neuromuscular diseases. The company's current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis, Parkinson's disease, oculopharyngeal muscular dystrophy, amyotrophic lateral sclerosis, and frontotemporal dementia.

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Axovant Gene Therapies (NASDAQ:AXGT) Upgraded to Buy by ValuEngine - Slater Sentinel

Zacks Investment Research downgraded shares of Axovant Gene Therapies (NASDAQ:AXGT) from a hold rating to a sell rating in a report released on Wednesday morning, Zacks.com reports.

According to Zacks, Axovant Sciences Ltd. is a biopharmaceutical company which focuses on the acquisition, development and commercialization of therapeutics for the treatment of neurodegenerative disorders. Its product candidate includes RVT-101 which is in different clinical trial for the treatment of Alzheimers disease and other forms of dementia. Axovant Sciences Ltd. is based in Hamilton, Bermuda.

Other equities research analysts also recently issued reports about the stock. ValuEngine raised shares of Axovant Gene Therapies from a hold rating to a buy rating in a report on Friday, January 3rd. Chardan Capital upped their price objective on shares of Axovant Gene Therapies from $10.00 to $15.00 and gave the company a buy rating in a report on Monday, October 28th. One investment analyst has rated the stock with a sell rating, one has issued a hold rating and nine have issued a buy rating to the company. The stock currently has a consensus rating of Buy and an average price target of $24.66.

NASDAQ AXGT traded down $0.22 during trading on Wednesday, hitting $4.43. 227,466 shares of the companys stock traded hands, compared to its average volume of 146,417. Axovant Gene Therapies has a 12-month low of $3.81 and a 12-month high of $19.60. The company has a debt-to-equity ratio of 0.69, a current ratio of 1.41 and a quick ratio of 1.41. The business has a 50 day moving average of $5.14 and a 200-day moving average of $6.11.

Axovant Gene Therapies (NASDAQ:AXGT) last released its quarterly earnings data on Friday, November 8th. The company reported ($0.61) EPS for the quarter, beating the Thomson Reuters consensus estimate of ($1.15) by $0.54. Research analysts predict that Axovant Gene Therapies will post -3.58 earnings per share for the current year.

A number of hedge funds and other institutional investors have recently added to or reduced their stakes in the business. BlackRock Inc. purchased a new stake in shares of Axovant Gene Therapies in the second quarter worth about $1,482,000. Tower Research Capital LLC TRC lifted its holdings in shares of Axovant Gene Therapies by 955.3% in the second quarter. Tower Research Capital LLC TRC now owns 4,221 shares of the companys stock worth $27,000 after buying an additional 3,821 shares in the last quarter. Jane Street Group LLC lifted its holdings in shares of Axovant Gene Therapies by 28.8% in the second quarter. Jane Street Group LLC now owns 46,455 shares of the companys stock worth $289,000 after buying an additional 10,375 shares in the last quarter. Barclays PLC purchased a new stake in shares of Axovant Gene Therapies in the third quarter worth about $65,000. Finally, Woodstock Corp purchased a new stake in shares of Axovant Gene Therapies in the fourth quarter worth about $83,000. 14.80% of the stock is currently owned by institutional investors and hedge funds.

Axovant Gene Therapies Company Profile

Axovant Gene Therapies Ltd., a clinical-stage gene therapy company, focuses on developing a pipeline of product candidates for debilitating neurological and neuromuscular diseases. The company's current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis, Parkinson's disease, oculopharyngeal muscular dystrophy, amyotrophic lateral sclerosis, and frontotemporal dementia.

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Axovant Gene Therapies (NASDAQ:AXGT) Downgraded to Sell at Zacks Investment Research - Riverton Roll