Archive for October, 2019

10/22/2019

ABERDEEN - In a first for the sector, industry bodies Subsea UK and the Umbilical Manufacturers Federation have joined forces to deliver a conference focusing on the new developments and opportunities which exist in the subsea umbilicals industry.

The global subsea umbilicals, risers and flowline market is forecast to be worth more than 5billion by 2021 and the half-day conference, which will take place on Thursday, 21 November at the Chester Hotel in Aberdeen, will focus on the opportunities which exist in the growing sector. Ahead of the conference, a networking reception will take place at the hotel the night before (Wednesday 20 November).

A number of experts, including representatives from oil and gas super-majors and service companies will explore a range of topics relating to subsea umbilicals including project achievements to date, life extension and safety. Shell, Equinor, Subsea 7, Aker Solutions, Oceaneering, JDR Cable Systems, Fibron, Nexans, MAATS Tech and Trelleborg will all deliver presentations at the event.

Neil Gordon, chief executive at Subsea UK, said: The subsea umbilical market has seen a massive growth in recent years, and it is predicted to expand and be part of a multi-billion-pound market. We have developed this conference in direct response to the number of opportunities which exist in subsea umbilicals and created a program which showcases the latest technical advances and knowledge which exists in the sector.

The Subsea UK and UMF conference will take place on Thursday, 21 November at the Chester Hotel, Aberdeen from 9am 7:30pm. A drinks reception is happening the evening before from 6.30pm at the hotel. For more information and to purchase tickets visit: https://www.subseauk.com/1479/subsea-uk-events.

Originally posted here:
Conference to examine opportunities in the subsea umbilical industry - WorldOil

TheCorrosion Monitoring Instrument marketreport [6 Year Forecast 2019-2025] focuses on Major Leading Industry Players, providing info likecompany profiles, product type, application and regions, production capacity, ex-factory price, gross margin, revenue, market share and speak to info.Upstream raw materials and instrumentation and downstream demand analysis is additionally administrated. The Corrosion Monitoring Instrument market business development trends and selling channels square measure analyzed. From a global perspective, It also represents overall Corrosion Monitoring Instrument industry size by analyzingqualitative insights and historical data.

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The study encompasses profiles of major companies operating in the global Corrosion Monitoring Instrument market.Key players profiled in the report on the global Corrosion Monitoring Instrument market includeHoneywell, ClampOn, Emerson, Intertek, Buckleys (UVRAL) Ltd, PT Korosi Specindo, Cosasco, Alabama Specialty Products and so on. Market leaders engage in strategic pricing and product improvement to increase their profit margins.

Scope of Corrosion Monitoring Instrument Market

A corrosion monitoring program provides comprehensive monitoring of all critical components of industrial objects, assets, facilities and plants for signs of corrosion. For reliable operation it is important to identify the location, rate, and underlying causes of corrosion. A corrosion monitoring program identifies any non-conforming alloy components, as these are generally susceptible to accelerated corrosion and can give relatively frequent cause for catastrophic failure.

Corrosion Monitoring can provide significant advantages when integrated into both preventative maintenance and the processes inherent to safety management programs. Based on the results of the Corrosion Monitoring program, informed decisions can be made, not only regarding the remaining life of the object affected by corrosion but also regarding life extension strategies, prospective material selection, and cost-effective methods for remedy of corrosion issues and problems.

This reprot focsues on the instrument market.

In 2019, the market size of Corrosion Monitoring Instrument is xx million US$ and it will reach xx million US$ in 2025, growing at a CAGR of xx% from 2019; while in China, the market size is valued at xx million US$ and will increase to xx million US$ in 2025, with a CAGR of xx% during forecast period.

In this report, 2018 has been considered as the base year and 2019 to 2025 as the forecast period to estimate the market size for Corrosion Monitoring Instrument.

This report studies the global market size of Corrosion Monitoring Instrument, especially focuses on the key regions likeUnited States, European Union, China, and other regions (Japan, Korea, India and Southeast Asia).

This study presents the Corrosion Monitoring Instrument production, revenue, market share and growth rate for each key company, and also covers the breakdown data (production, consumption, revenue and market share) by regions, type and applications. history breakdown data from 2014 to 2019, and forecast to 2025.

For top companies in United States, European Union and China, this report investigates and analyzes the production, value, price, market share and growth rate for the top manufacturers, key data from 2014 to 2019.

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On the basis of product,this report displays the sales volume, revenue (Million USD), product price, market share and growth rate of Corrosion Monitoring Instrument for each type, primarily split into-

On the basis on the end users/applications,this report focuses on the status and outlook for major applications/end users, sales volume, market share and growth rate of Corrosion Monitoring Instrument for each application, including-

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Turmeric has numerous uses when it comes to health benefits. They are being used in Indian households for a long time.These include several health benefits and medicinal uses. Turmeric is one of the most powerful spices. It has a unique taste with a mix of citrusy bitterness. It is also associated with Ayurvedic practices.

Also read:Indian Food: What Are The Uses Of Turmeric In Indian Dishes?

Turmeric also has some benefits to enhance your beauty. Its anti-inflammatory properties help in removing dead skin cells. It can also be used to wash your fash or apply once in a while. There are several benefits you can receive from turmeric. These are some of the imperative ones.

Also read:Basil Benefits: Top Benefits Of Basil For Your Skin

The anti-inflammatory properties that are found in turmeric are used to soothe osteoarthritis and rheumatoid arthritis. These collectively work in your favour. The antioxidant destroys the free radicals in the body that damage the cells. These can help alleviate and relax your mild joint pains. It cannot be used as a substitute for medication.

There is a compound in turmeric that has not been studied as much as the other compounds like curcumin - aromatic turmerone or ar-turmerone. This compound has reportedly been repairing brain stem cells. It also helps in the recovery from neurodegenerative diseases like stroke and Alzheimer's.

A substance in turmeric Lipopolysaccharide has anti-bacterial, anti-fungal, and anti-viral agents. This also helps to stimulate the immune system. Make sure you consume only a teaspoon in warm water.

Also read:Jackfruit: Delicious Recipes To Make With The Diabetic Friendly Fruit

The anti-inflammatory and antioxidant properties of curcumin help to reduce the onset of Type 2 diabetes. It helps to moderate insulin levels and boosts the effect of medications that treat diabetes. But always remember not to use it as a source of medication.

Turmeric increases the production of vital enzymes that detoxify our blood in the liver by breaking down and reducing the toxins. It also helps with the circulation of blood. Overall, it is known to improve liver health.

Original post:
Turmeric: Uses and benefits of the spice that you must know - Republic World - Republic World

Humans diverged from chimpanzees and other great apes roughly 6m years ago. But despite us being closely related, human brains are vastly different enabling us to engage in complex language, science, art, morality and much more. But what exactly was it that enabled our brains to reach such mindboggling heights?

We know that the human brain has dramatically expanded in size over the past 6m years. Humans are in fact the mammals with the largest brain relative to body size. But which specific evolutionary genetic changes enabled larger and more complex brains has long remained a bit of a mystery. Now a new study, published in Nature, offers clues.

One important reason why it has been so hard to study primate brain development is that, until relatively recently, scientists did not have access to living, developing brain tissue. This is what can ultimately allow us to functionally test theories of brain evolution as we can essentially watch how a brain develops over time in a dish and manipulate biological pathways to see what role they play in brain development.

But in the last few years, scientists have worked out how to make lab-grown models of developing brain tissue so-called brain organoids to begin to address these questions.

Organoids are clusters of cells that organise themselves into mini versions of our organs, such as the brain or the liver. Thats because they are made by culturing stem cells, which have the potential to develop into any tissue of the body. These stem cells can be generated directly from cells of adult origin, such as skin or blood cells. They are then grown in a gel that allows them to develop three dimensionally. And thats exactly what the researchers behind the new study did.

So what kind of genetic changes do we think contributed to human brain evolution? Only about 1.5% of our DNA actually consists of genes with instructions for making proteins. Proteins are the molecules that do most of the work in cells and determine the cells structure and function. It was once thought that the remaining 98.5% of DNA was junk with no clear purpose. However, it is now known that some of this DNA may play an important role in controlling which genes are expressed meaning determining how they are turned on and off.

The number of changes in protein-coding regions of DNA are far too few to explain the striking differences observed between humans and other primates. In fact, of the genetic regions that have changed the most since our divergence from chimpanzees, 92% do not overlap with protein-coding DNA.

It is predicted that at least a third of these regions play a role in controlling the expression of genes. It has long been hypothesised that the majority of differences observed between the brains of great apes and us are due to changes in the timing and expression of genes, rather than changes to the gene itself. The vast majority of our genes are therefore identical.

The main focus of the new study was to identify how genes are regulated differently in humans compared to other primates. The authors did this by generating brain organoids from human, chimpanzee and macaque stem cells and compared these at various points over the course of four months. This mimics how a brain forms in the womb, with organoids consisting of multiple growing buds of brain tissue that first consist largely of neural progenitor cells that in later stages begin to make neurons.

From the outside, brain organoids look more like small popcorn than a mini brain and do not reach sizes larger than around five or six millimetres due to a lack of blood supply.

The authors observed that human brain development occurs at a slower pace than the other two primates. This delayed maturation of the human brain makes sense as, given more time, the cells that generate neurons will have a longer period to expand their population, giving rise to more neurons and a bigger brain later on.

The researchers were also able to look at the expression of genes in individual cell types of the brain organoids. They measured the expression of a gene by looking at the levels of a messenger molecule that is made from reading DNA and is necessary to direct the formation of proteins. By comparing gene expression in cells that were developing to become the cerebral cortex which plays an important role in advanced cognitive processes such as awareness, thought, memory, language and consciousness they detected 98 genes that were differently expressed in humans.

Gene expression doesnt tell the whole story though. Its rate is ultimately controlled by a process called gene regulation. In order to identify potential regulatory mechanisms, the authors pinpointed regions of DNA that are accessible or open at the various stages in particular cells. These accessible regions of DNA have the potential to interact with proteins and can regulate gene expression.

By comparing organoids between human and chimpanzee, the researchers were able to identify regions of DNA that were differently accessible in humans potentially playing a regulatory role. Regulatory regions of DNA are more likely to be found in close proximity to the genes they are regulating the expression of. More than 60% of the genes that were expressed differently in humans were also in close proximity to differently accessible regions. This suggests that human-specific development and gene expression is a result of evolutionary changes in regions of DNA that are capable of regulating gene expression.

A significant proportion of the regions of DNA that we already know have changed the most since our divergence from chimps were found to overlap with those being different in terms of accessibility suggesting the team has indeed highlighted key regulatory processes responsible for making us humans.

This study takes the first steps in pinpointing interesting candidate genetic regions responsible for human brain complexity. The authors do not dive deeper into the mechanisms of what the altered expression of a specific gene actually means in terms of how the brain grows and functions. It does, however, provide an excellent resource and starting point to direct future research in this direction.

This research is not only important in understanding what makes us human, but also in working out how certain human disorders may arise. Several studies have found that mutations in regions of DNA with human-specific changes are associated with neurodevelopmental disorders.

Excerpt from:
Lab-grown mini brains shed light on how humans split from great apes - The Conversation UK

Some gene therapies, such as bluebird bios Zynteglo, work by modifying a patients own blood stem cells to deliver a functioning gene. While they hold great potential as life-saving treatments for many immune system disorders and blood-based conditions, the process for administration can be costly and time-consuming.

In a study published in the journal Blood, scientists at Scripps Researchdescribed a possible way to more efficiently deliver genes to improve gene therapy treatment outcomesand it involves a close relative to a natural compound found in grapes.

Take Zynteglo, formerly known as LentiGlobin. Approved in the EU to treat beta thalassemia, an inherited blood disorder, it uses a lentiviral vector to insert a functioning version of the beta-globin gene into a patients blood-producing hematopoietic stem cells (HSCs) outside of the body. When the cells are given back to the patient, the gene can multiply and start to make healthy red blood cells.

Industry Insight Survey: Direct-to-Patient Distribution of Clinical Supplies

This industry survey seeks to gain insight on trial sponsors' perspective on offering a DTP option and their current level of awareness and understanding of any factors that may influence their ability to do so. The first 50 qualified respondents will receive a $5 Amazon gift card.

However, HSCs protect themselves with structures known as interferon-induced transmembrane (IFITM) proteins against viral attacks. Therefore, in preparation for a gene therapy, the lentiviral vectors could be intercepted several timeswasting a large amount of expensive materialsbefore a successful delivery.

This is why gene therapy of hemopoietic stem cells has been hit-or-miss, Bruce Torbett, the studys senior author, said in a statement. We saw a way to potentially make the treatment process significantly more efficient.

Torbett and colleagues at Scripps Research focused on caraphenol A, a small molecule related to resveratrol, which is a natural compound produced by grapes widely known for its antioxidant and anti-inflammatory effects.

The team found that adding caraphenol A to human HSCs, along with lentiviral vectors, could reduce the cells natural defenses by lowering the levels of IFITM2 and IFITM3, allowing the vectors to pass more easily. The effect was even more pronounced once the HSCs were transplanted into mice, as the cells divided and produced blood cells with the corrected gene, according to the team.

RELATED:Improving viral vectors for hemophilia gene therapies by tricking the immune system

Because gene therapy holds the promise of a potential cure for debilitating diseases that are deadly or require long-term care, scientists have been exploring new ways to improve it. A research team at the San Raffaele Telethon Institute for Gene Therapy in Italy recently found integrating the protein CD47 in the surface of lentiviral vectors could help them escape detection and destruction by the immune system once inside the body.

Torbett and his team believe caraphenol A could reduce costs of HSC-based gene therapy, which are high: Bluebird has priced Zynteglo at 1.6 million ($1.8 million) in Europe. It could also save time by helping patients get the therapy sooner. And because the longer stem cells stay outside of the body, the more likely they are to lose their ability to self-generate, more efficient gene delivery could also preserve their self-renewing properties, Tobett said.

Read more:
Could a grape-based compound improve gene therapy efficiency? - FierceBiotech

Injecting the gene-editing tool CRISPR into the brains of adolescent mice counteracts the effects of a mutation in a top autism gene. The finding suggests that mutations in this gene, SCN2A, may be treatable at any age.

This gives us hope that this is something thats not just a developmental deficit, and that once all the changes are there that youre stuck with them, says Perry Spratt, a graduate student in Kevin Benders lab at the University of California, San Francisco. Spratt presented the unpublished findings today at the 2019 Society for Neuroscience annual meeting in Chicago, Illinois.

SCN2A encodes a sodium channel that helps neurons propagate electrical signals. The mice have a harmful mutation in one copy of the gene, causing their neurons to fire sluggishly and make immature connections. The treatment reverses the problem by ramping up expression of the intact copy.

The first inkling that the reversal might work even after infancy came in June: The team reported then that introducing an SCN2A mutation into adolescent mice has many of the same effects on neurons as a mutation present from conception.

At the cellular level we see a lot of the same deficits as we see when the genes been gone throughout life, Spratt says.

The team engineered mice in which the mutation in SCN2A can be corrected by injecting an enzyme called Cre recombinase.

Running short: Neurons from mice missing SCN2A (right) have signal-receiving branches that are less mature than those in controls (left).

Injecting the Cre enzyme into the brains of 4- to 5-week-old mutant mice restored normal expression of SCN2A. It also reversed the problems in the mices neurons.

But this strategy cannot be applied to people because it would require genetic engineering of embryos. So the researchers created a virus that ferries a modified version of CRISPR into cells. This version of the editing tool does not snip DNA but can instead activate any gene.

The researchers injected the virus into the brains of 4-week-old mutant mice, along with synthetic RNA snippets designed to guide CRISPR to SCN2A. The treatment boosted SCN2A expression and reversed the problems in the mices neurons.

The team also tested one important control: They found that the treatment does not cause neurons to become hyperactive and so does not run the risk of triggering seizures.

The remarkable thing about this is that it can go gangbusters without making the cells hyperexcitable, says Bender.

The team is testing ways to deliver the virus to the whole brain. They also plan to test whether CRISPR reverses learning and memory problems in the mutant mice.

For more reports from the 2019 Society for Neuroscience annual meeting, please click here.

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CRISPR therapy may reverse autism mutation's effects well past infancy - Spectrum

Fargo, N.D., Oct. 22, 2019 (GLOBE NEWSWIRE) -- Aldevron has announced the immediate availability of Nanoplasmids and antibiotic-free RNA-OUT technology for use in gene therapy manufacturing. Through a license agreement with Nature Technology Corporation (NTC), Aldevron can now provide its clients the option of using NTCs plasmid technology to provide a more efficient system for manufacturing viral vectors used in gene and cell therapy. Gene and cell therapy companies interested in using NTCs technology can now go directly to Aldevron for their manufacturing needs without first obtaining a license from NTC. For applications where the NTC plasmid is not the active pharmaceutical ingredient or in the final product clients do not need to pay any royalties or license fees beyond the purchase price of the product.

Gene and cell therapy researchers continue to make incredible discoveries that will improve lives for millions of people, said Michael Chambers, Aldevron CEO. Aldevron is committed to providing the best manufacturing technologies to support their breakthrough treatments. Our partnership with NTC enables us to provide plasmid technology that significantly improves the manufacturing process and reduces regulatory risk, resulting in reduced costs and faster time to patients.

The fields of gene and cell therapy are surging with the approvals of Kymriah and Yescarta CAR-T treatments for cancer and Luxturna for the treatment of Leber congenital amaurosis, a rare genetic ocular disease. With nearly 3,000 clinical trials underway, improvements in manufacturing are required to provide the capacity to accommodate the growth in research and anticipated commercial products. The NTC technology, combined with Aldevrons unequaled scale and throughput, assure clients will have the products they need.

Our Nanoplasmid and RNA-OUT technology has been under development for 12 years and can provide demonstrable advantages for viral vector manufacturing, said Clague Hodgson, CEO of NTC. We are pleased to collaborate with Aldevron to make this technology immediately available to its clients so they can avoid the delay and expense associated with the licensing process thereby reducing the barriers to delivering transformative treatments to patients.

Providing these novel plasmids furthers our strategy to go beyond the traditional role of a contract development and manufacturing organization, added Chambers. Aldevron provides a variety of options for clients, including off-the-shelf, royalty-free helper plasmids for AAV production and packaging plasmids for lentiviral production, in pursuit of our goal to enable our clients to make a difference in the lives of the patients they serve. For more information on Nanoplasmids, please visitaldevron.com/nanoplasmid.

About Aldevron

Aldevron serves the biotechnology industry with custom production of nucleic acids, proteins, and antibodies. Thousands of clients use Aldevron-produced plasmids, RNA and gene editing enzymes for projects ranging from discovery research to clinical trials to commercial applications. These products are critical raw materials and key components in commercially available drugs and medical devices. Aldevron specializes in GMP manufacturing and is known for inventing the GMP-Source quality system. Company headquarters are in Fargo, North Dakota, with additional facilities in Madison, Wisconsin, and Freiburg, Germany. To learn more, visitwww.aldevron.com.

About Nature Technology Corporation

Nature Technology Corporation is a developer of safe and effective nucleic acid-based gene therapeutics, DNA vaccines, and associated technologies, including antibiotic-free selection (RNA-OUT), regulatory compliance, viral vector retrofitting, and best in class NanoplasmidsTM. NTC proprietary vector and manufacturing platforms offer wide applications to improve the safety and performance of plasmids and biologics. NTC also provides cost effective outsourcing of product development services including custom gene design, vector construction, plasmid DNA manufacturing and recombinant proteins. For more information about NTC products and business related inquiries visitwww.natx.comor contact Deborah Moorad-Watts, email: dmoorad@natx.com.

Original post:
Aldevron, Nature Technology Corporation team up to offer Nanoplasmids for gene therapy manufacturing - GlobeNewswire

In order to help individuals suffering from emotion dysregulation, Stanford Psychophysiology Laboratorys Wehab Lab has developed a haptic breathing pacer (HBP) prototype. The technology also helps those diagnosed with Autism Spectrum Disorder better regulate anxiety levels. (Photo : Pardis Miri)

The Wehab Lab of the Stanford Psychophysiology Laboratory has been working on developing a haptic breathing pacer (HBP) to address issues surrounding mental health and emotion dysregulation, an individuals inability to change an undesired emotional experience. The device applies vibrations to a users body, with which they can synchronize their breathing to reduce arousal in high-stress situations.

Post-graduate researcher Pardis Miri described it as important for helping individuals cope with a stressor.

There is significant recent interest in developing technology that can help people regulate high-arousal negative affect in the course of their everyday lives, she wrote. Negative affect is a personality trait that includes emotions such as anger, anxiety and stress. These technologies have all been designed for a neurotypical population and, for the most part, [are] not well-evaluated for efficacy. The term neurotypical refers to individuals without a developmental disorder.

Technologies being developed by other researchers are not evaluated with consideration for those with individual differences or the neurodiverse population, according to Miri. But Miris team found that their inconspicuous and personalizable breathing pacer prototype could be able to help individuals outside of the neurotypical population, she said.

The prototype proved to be especially promising, Miri wrote, for individuals who score low in terms of two traits: openness, as measured by the Big Five scale, and usage of reappraisal, a psychological strategy that lessens the emotional impact of a stressful event.

These are characteristics commonly observed in Autism Spectrum Disorder (ASD) population, and so our findings suggest potential interventional success for individuals diagnosed with ASD, Miri said.

Helping these children successfully regulate their affect could have a major impact on their development and could prevent them from serious health issues, Miri wrote.

Horia Margarit M.S. 17, a member of the research team, used machine-learning techniques to analyze data collected from the prototypes.

He used a method published in 2017, he said. Very few people even know about it, and thats one of the major triumphs of our research because we were able to show this level of success using that technique.

The team is focusing on improving the efficacy of their pacer for individuals with ASD.

We believe our approach is promising for individuals diagnosed with ASD, and we also understand the risks that are involved, Miri wrote. Our goal is to create a personalizable vibrotactile system that utilizes the strengths of the autistic population to respond to their affect regulatory needs.

Miri said her aim is to demonstrate the efficacy of her teams approach in regulating the affect that individuals diagnosed with ASD experience in their daily lives.

This is tremendously promising research because the entire concept behind this is similar to that of CRISPR and gene therapy: personalized medicine, Margarit said. What Pardis and I did was understanding people on a personal level in terms of mental health.

Contact Camryn Pak at cpak23 at stanford.edu.

We're a student-run organization committed to providing hands-on experience in journalism, digital media and business for the next generation of reporters.Your support makes a difference in helping give staff members from all backgrounds the opportunity to develop important professional skills and conduct meaningful reporting. All contributions are tax-deductible.

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Researchers work on device to help individuals cope with autism - The Stanford Daily

NEW YORK, Oct. 21, 2019 /PRNewswire/ --

Personalized Medicine Market Size, Share & Trends Analysis By Product (Personalized Medical Care, Therapeutics, Personalized Nutrition & Wellness), By Region (Asia Pacific, Europe, North America), And Segment Forecasts, 2019 - 2025

Read the full report: https://www.reportlinker.com/p05807253/?utm_source=PRN

The global personalized medicine (PM) market size is expected to reach USD 3.18 trillion by 2025 registering a CAGR of 10.6% over the forecast period. Growing number of approved companion diagnostics and biomarker products have been proved promising avenues for the development of advanced precision diagnostic tests. Several personalized diagnostics and therapeutic products based on companion diagnostics are under clinical trials and development pipelines, which also add an incremental opportunity to accelerate this market.

Gene therapies are widely utilized to diagnose both chronic and genetic disorders according to individual's genetic makeup.Growing adoption of gene sequencing and data analytics technologies is anticipated to escalate precision medicine market growth.

Decreasing cost of whole genome sequencing, advancement in cell biology sector, and the development of "Human Genome Project" are several other factors influencing the demand for gene therapy hence, boosting the overall market. Moreover, key leaders are undergoing numerous strategic developments to launch novel personalized products, which will also contribute to the market expansion.

For instance, in September 2018, Vertex Pharmaceuticals collaborated with Genomics for the delivery of precision medicine based on individual's genetic differences and lifestyle. Similarly, in January 2018, Foundation Medicine collaborated with European Organization for Research and Treatment of Cancer (EORTC) to facilitate Foundation's comprehensive genomic profiling to across personalized medicine and clinical trial enrolment.

Further key findings from the study suggest: PM therapeutics is projected to register the fastest CAGR over the forecast period owing to the development of high-capacity rapid sequencing platforms for medical therapies Reduction in sequencing cost leads to an expected growth of genomic sequencing and genetic research, thus contributing to lucrative opportunities for genomic medicine Asia Pacific is projected to be the fastest-growing regional market due to rising number of collaborative initiatives between eastern and western market leaders Chinese personalized medicine market is benefitted due to initiatives implemented by the Chinese government and reimbursements for disease treatment Prominent companies include GE Healthcare; Illumina, Inc.; Asuragen, Inc.; Abbott Laboratories; Dako A/S; Exact Science Corp.; and Biogen, Inc.

Read the full report: https://www.reportlinker.com/p05807253/?utm_source=PRN

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The global personalized medicine (PM) market size is expected to reach USD 3.18 trillion by 2025 registering a CAGR of 10.6% over the forecast period...

LONDON--(BUSINESS WIRE)--

The market will accelerate at a CAGR of over 8% between 2019-2023

The report, global age-related macular degeneration (AMD) therapeutics market has been added to Technavios catalog. It provides a comprehensive analysis of the market, including its global and regional market share as well as market segmentation based on type and geography for the forecast period 2019-2023.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20191021005715/en/

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This report on the age-related macular degeneration (AMD) therapeutics market includes:

Age-related macular degeneration (AMD) therapeutics market analysis and forecast 2019-2023: Features

Competitive landscape

Market segmentation

Type

Geography

Market drivers

Market trends

Market challenges

Five forces analysis

Market landscape

Market sizing & forecast

Age-Related Macular Degeneration (AMD) Therapeutics Market 2019-2023: Competitive Landscape

Vendors covered

Vendor classification

Amgen Inc.

Bausch Health Companies, Inc.

F. Hoffmann-La Roche Ltd.

Novartis AG

Regeneron Pharmaceuticals Inc.

Age-Related Macular Degeneration (AMD) Therapeutics Market 2019-2023: Geographic Landscape

North America - Market size and forecast 2018-2023

Europe - Market size and forecast 2018-2023

Asia - Market size and forecast 2018-2023

ROW - Market size and forecast 2018-2023

Key leading countries

Age-Related Macular Degeneration (AMD) Therapeutics Market 2019-2023: Type Landscape

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High prevalence of AMD will drive the age-related macular degeneration (AMD) therapeutics market

The prevalence of AMD is increasing significantly, owing to the presence of high-risk factors such as aging, smoking, high cholesterol, and high blood pressure. Moreover, despite the high prevalence of AMD, no approved therapies are available in the market for the treatment of the condition. Thus, the high prevalence coupled with the huge unmet medical need of dry AMD are expected to drive market growth during the forecast period.

Story continues

Development of gene therapy for AMD An emerging trend in the osteoporosis market

At present, only a few drugs are approved for the treatment of AMD. However, the side effects associated with them are very severe, and the majority of them act against VEGF only. Hence, there is a huge unmet need for safe and novel drugs to treat AMD. The currently available anti-VEGF therapies require repetitive and inconvenient intraocular injections. Hence, several companies are working on novel drugs to combat AMD, among which gene therapy is expected to cure the disease effectively. Hence, the development of gene therapy is expected to be a positive trend for the global AMD therapeutics market.

Other Key Topics Covered in the Report are:

MARKET LANDSCAPE

MARKET SIZING

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Growth of Age-Related Macular Degeneration (AMD) Therapeutics Market to be Impacted by the Development of Gene Therapy for AMD | Technavio - Yahoo...

ImmuneCyte to Begin Operations in Fourth Quarter of 2019

RANCHO CORDOVA, Calif., Oct. 22, 2019 /PRNewswire/ -- Cesca Therapeutics Inc. (Nasdaq: KOOL), a market leader in automated cell processing and autologous cell therapies for regenerative medicine, and ThermoGenesis, its wholly owned device subsidiary, today announced that the company has entered into a definitive joint venture agreement with HealthBanks Biotech (USA) Inc., one of the world's leading stem cell bank networks, to commercialize its proprietary cell processing platform, CAR-TXpress, for use in immune cell banking as well as for cell-based contract development and manufacturing services (CMO/CDMO). The joint venture will be named ImmuneCyte Life Sciences Inc. ("ImmuneCyte") and is expected to officially launch during the fourth quarter of 2019.

Under terms of the agreement, ImmuneCyte will initially be owned 80% by HealthBanks Biotech and 20% by Cesca. Cesca will contribute to ImmuneCyte exclusive rights to use ThermoGenesis' proprietary cell processing technology for the immune cell banking business and non-exclusive rights for other cell-based contract development and manufacturing services. Cesca will also contribute its clinical development assets to the joint venture, as the company has decided to discontinue these activities in order to focus exclusively on the device business.

Once operational, ImmuneCyte will be among the first immune cell banks in the U.S. to provide clients with the opportunity to bank their own healthy immune cells for future use as a resource for cell-based immunotherapies, such as dendritic cell and chimeric antigen receptor (CAR) T-cell therapies. ImmuneCyte will utilize ThermoGenesis' proprietary CAR-TXpress platform which allows for the isolation of different components from 200 ml of blood in cGMP compliant, closed system. Given that the CAR-TXpress platform can increase cell processing efficiency by up to 16-fold as compared with the traditional, labor-intensive ficoll gradient centrifugation-based cell processing method, ImmuneCyte is expected to offer customers an unparalleled competitive advantage, including an ability to store their own immune cells at a tangibly lower cost.

"The ImmuneCyte joint venture will be paramount to the execution of our strategy to become a preferred cell processing and manufacturing solution provider in the cell and gene therapy field," said Dr. Chris Xu, Chairman and Chief Executive Officer of Cesca Therapeutics. "CAR-T therapeutic research is advancing rapidly. Partnering with HealthBanks Biotech, one of the foremost stem cell bank networks, with an experienced team and an established global infrastructure, will offer customers the ability to preserve younger, healthier and uncontaminated immune cells for potential future use. By applying our proprietary CAR-TXpress technology to immune cell banking and other CDMO cellular manufacturing services, we will allow for the manufacture and production of more effective and less costly immunotherapies."

In 2017, the U.S. Food and Drug Administration (FDA) approved two CAR-T cell therapies, under breakthrough designation, for the treatment of advanced B cell leukemia and lymphomas. Both use autologous (a patient's own) immune T cells to fight cancer and have reported an over 80% response rate in the "no-option" patient group, for those who have failed both chemo- and radiation therapies. This has helped to spur massive global interest for the development of additional CAR-T immunotherapies1. By the end of September 2019, there were over 800 CAR-T cell clinical trials registered on the http://www.clinicaltrials.gov website, targeting a wide variety of blood cancers and solid tumors.

Although highly effective, several recent studies on the eligibility of patients to enroll in CAR-T clinical trials showed that as many as 30-50% of cancer patients may not be eligible to enroll or to get sufficient CAR-T cells manufactured for the therapy. Reasons may include: (1) the function of the immune system declines with age and can be negatively affected by other medical conditions, (2) most standard cancer therapies, such as chemotherapy and radiation, destroy the immune system, and (3) in many cases of advanced cancer, cancer cells will enter circulation, invade and interfere with the body's natural production of immune cells. According to a recently reported JULIE trial, a CAR-T clinical trial in relapsed or refractory diffuse large B-cell lymphoma (DLBCL), one-third of the 238 screened patients failed to be enrolled, and more than half of the 238 failed to receive the intended CAR-T therapy2,3. ImmuneCyte will offer customers the ability to preserve younger, healthier and uncontaminated immune cells, for potential future use in advanced cancer immunotherapy.

About HealthBanks Biotech (USA) Inc.HealthBanks Biotech, headquartered in Irvine, CA, is one of the leading stem cell bank networks in the world and offers services globally through its sister companies located in the United States and other regions and nations. HealthBanks Biotech is accredited by the FDA, AABB, and CAP. The HealthBanks Biotech group was originally founded in 2001 with a vision that stem cells and cell and gene therapies could transform modern medicine. HealthBanks Biotech is a subsidiary of Boyalife Group, Inc. (USA), an affiliate of Boyalife (Hong Kong) Limited, the largest stockholder of Cesca. For more information about HealthBanks Biotech (USA) Inc., please visit: http://www.healthbanks.us.

About ImmuneCyte Life Sciences Inc.ImmuneCyte will provide clients with the opportunity to bank their own immune cells when the cells are "healthy and unaffected" as a future resource for cellular immunotherapies, such as CAR-T. ImmuneCyte utilizes a proprietary CAR-TXpress platform, a GMP compliant close-system capable of automated separating and cryopreserving different components from blood. For more information about ImmuneCyte Life Sciences Inc., please visit: http://www.immunecyte.com.

About Cesca Therapeutics Inc.Cesca Therapeutics develops, commercializes and markets a range of automated technologies for CAR-T and other cell-based therapies. Its device division, ThermoGenesis develops, commercializes and markets a full suite of solutions for automated clinical biobanking, point-of-care applications, and automation for immuno-oncology. The Company has developed a semi- automated, functionally closed CAR-TXpress platform to streamline the manufacturing process for the emerging CAR-T immunotherapy market. For more information about Cesca and ThermoGenesis, please visit: http://www.cescatherapeutics.com.

Company Contact:Wendy Samford916-858-5191ir@thermogenesis.com

Investor Contact:Paula Schwartz, Rx Communications917-322-2216pschwartz@rxir.com

References:

1. Facts About Chimeric Antigen Receptor (CAR) T-Cell Therapy, Leukemia and Lymphoma Society (2018). https://www.lls.org

2. Updated Analysis of JULIET Trial: Tisagenlecleucel in Relapsed or Refractory DLBCL (2018).

3. Eligibility Criteria for CAR-T Trials and Survival Rates in Chemorefractory DLBCL. Journal of Clinical Pathways (2018).

View original content:http://www.prnewswire.com/news-releases/cesca-therapeutics-forms-joint-venture-with-healthbanks-biotech-usa-to-provide-immune-cell-banking-and-cell-processing-services-300942618.html

SOURCE Cesca Therapeutics Inc.

Company Codes: NASDAQ-SMALL:KOOL

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Cesca Therapeutics Forms Joint Venture with Healthbanks Biotech (USA) to Provide Immune Cell Banking and Cell Processing Services - BioSpace

NEW YORK and CLEVELAND, Oct. 21, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced the presentations of data from the Transpher A Study, the Companys ongoing Phase 1/2 clinical trial evaluating ABO-102 for the treatment of Sanfilippo syndrome type A (MPS IIIA), and research updates from its library of novel AIMTM adeno-associated virus (AAV) capsids at the 27th European Society of Gene and Cell Therapy (ESGCT) Congress, to be held October 22-25, 2019 in Barcelona, Spain.

The data will be presented as follows:

Safety, Tolerability, Biopotency and Neurocognitive Data of ABO-102 in Transpher A, an Open-Label, Multicenter, Single-Dose, Dose-Escalation, Phase 1/2 Clinical Trial in Sanfilippo Syndrome type A (Mucopolysaccharidosis IIIA)Oral Presentation #039Presenter: Maria Jose de Castro, M.D., Hospital Universitario Santiago de CompostelaSession 4c: Metabolic and Genetic DiseasesDate/Time: Friday, October 25, 2019, 9:00 a.m. to 11:00 a.m. CESTLocation: Room 112

Novel AAV Capsids Show Increased Evasion to Neutralizing Antibodies Against Natural SerotypesPoster #P347Session Title: Poster Session IDate/Time: Wednesday, October 23, 2019, 1:00 p.m. to 3:00 p.m. CESTLocation: Multipurpose Hall

Development of an Improved Novel AAV Capsids for Intramuscular DeliveryPoster #P027Session Title: Poster Session IDate/Time: Wednesday, October 23, 2019, 1:00 p.m. to 3:00 p.m. CESTLocation: Multipurpose Hall

Novel AAV Capsids for Delivery to the Retina by Intravitreal AdministrationPoster #P009Session Title: Poster Session IDate/Time: Wednesday, October 23, 2019, 1:00 p.m. to 3:00 p.m. CESTLocation: Multipurpose Hall

Development of a Novel AAV Capsid with Improved PNS Tropism for Treating Pompe Disease by Intravenous AdministrationPoster #P007Session Title: Poster Session IDate/Time: Wednesday, October 23, 2019, 1:00 p.m. to 3:00 p.m. CESTLocation: Multipurpose Hall

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Companys clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Companys portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Its preclinical assets include ABO-401, which uses the novel AIM AAV vector platform to address all mutations of cystic fibrosis. Abeona has received twenty regulatory designations from the FDA and EMA for its pipeline candidates. For more information, visit http://www.abeonatherapeutics.com.

Forward-Looking StatementsThis press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Companys clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Companys goals and objectives. We have attempted to identify forward looking statements by such terminology as may, will, believe, estimate, expect, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Companys Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

Investor Contact:Sofia WarnerSenior Director, Investor RelationsAbeona Therapeutics+1 (646) 813-4710swarner@abeonatherapeutics.com

Media Contact:Scott SantiamoDirector, Corporate CommunicationsAbeona Therapeutics+1 (718) 344-5843ssantiamo@abeonatherapeutics.com

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Abeona Therapeutics Announces Presentations at the 27th European Society of Gene and Cell Therapy (ESGCT) Congress - BioSpace

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

GenSight Biologics (Paris:SIGHT)(Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported its cash position as of September 30, 2019, and provided recent operational updates.

Cash and cash equivalents

GenSight Biologics cash and cash equivalents totaled 5.1 million as of September 30, 2019, compared to 14.3 million as of June 30, 2019. This amount does not include a 4.3 million reimbursement of the 2018 Research Tax Credit, expected in the coming days. Including this impending reimbursement, cash and cash equivalents would be 9.4 million as of September 30, 2019.

The cash burn in the first three quarters of 2019 principally reflects the final steps of pharmaceutical development for GS010 in preparation for a marketing authorization filing in Europe. These are mainly preparatory activities to ensure manufacturing readiness to commercialize under Good Manufacturing Practices (GMP).

In line with our expectations and including the impending reimbursement of the 2018 Research Tax Credit, we are fully financed until the end of the first quarter of 2020, commented Thomas Gidoin, Chief Financial Officer of GenSight Biologics. We have been actively working on a financing transaction in the past weeks, favoring less dilutive options, and expect to be able to announce a closing shortly.

Clinical Development of GS010

Results at 96 weeks of the RESCUE Phase III clinical trial evaluating the efficacy and safety of a single intravitreal injection of GS010 in subjects with LHON due to the ND4 mutation with an onset of vision loss up to 6 months were reported in September 2019. This last set of results showed a durable bilateral improvement in visual acuity of 25 ETDRS letters equivalent versus nadir in GS010-treated eyes. These results from RESCUE show a remarkable correspondence with those from the REVERSE trial, which studied the treatment of subjects at 6 to 12 months after onset of vision loss.

In a natural history study conducted by Santhera1, 28% of subjects with the G11778A-ND4 mutation achieved a spontaneous clinically relevant recovery (CRR) from nadir in at least one eye. By comparison, 63% of RESCUE subjects achieved this definition of CRR in at least one eye at Week 96, showing a clear superiority to natural history. A similar comparison for REVERSE subjects reveals a much higher rate of CRR (78%) from nadir compared to natural history.

GenSight also reported results from a non-clinical study to investigate the local biodistribution of GS010. In this study, tissue samples from the non-injected eye of monkeys that had been unilaterally injected with GS010 were found to contain GS010 DNA three months after injection, indicating the expression of the therapeutic gene in the uninjected contralateral eye. These findings support the mechanism for bilateral visual improvement with unilateral GS010 gene therapy, which was consistently observed in LHON subjects in the REVERSE and RESCUE Phase III trials.

Regulatory Pathway of GS010

GenSight is planning to schedule a pre-submission meeting with the EMA in early 2020 and expects to submit an application for marketing approval in Europe in the third quarter of 2020.

An End of Phase II meeting with the U.S. Food and Drug Administration (FDA) was requested as planned, which the agency has now confirmed for December 19, 2019.

We strongly believe that the remarkable results from REVERSE and RESCUE, showing a clinically significant and durable bilateral improvement of vision, and demonstrating clear superiority to natural history, are a compelling core for our marketing authorization application in Europe, commented Bernard Gilly, co-founder and Chief Executive Officer of GenSight Biologics. The demonstration of GS010 DNA presence in the uninjected contralateral eye now provides a powerful scientific rationale to support bilateral improvement. We are eager to meet with regulatory agencies in the next few weeks to discuss these major findings.

Number of outstanding shares

As of September 30, 2019, GenSight Biologics number of outstanding shares was 29,028,291 ordinary shares.

GenSight Biologics will report its cash position as of December 31, 2019 on January 21, 2020.

GenSight will host a conference call today, October 22, 2019, at 9:30am CEST in French, and at 2.30pm CEST (8.30am EST) in English, to further discuss this operational update.

Conference call in French

Dial-in numbers:

France: +33 (0) 1 7037 7166Password: GenSight Franais

Conference call in English

Dial-in numbers:

United States: +1 212 999 6659France: +33 (0) 1 7037 7166United Kingdom: +44 (0) 20 3003 2666Password: GenSight English

A replay of the calls will be available using the following dial-in numbers:

France: +33 (0) 1 7037 7166United Kingdom (standard international access): +44 (0) 20 3003 2666Code: 5645038#

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, GS010, is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

1 Magda et al (2019), Natural History of Lebers Hereditary Optic Neuropathy (LHON): Findings from a Large Patient Cohort, Poster presented at NANOS March 16-21, 2019; Poster Session II: Scientific Advancements; Poster: 163

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GenSight Biologics Reports its Cash Position as of September 30, 2019 and Provides Operational Update - Business Wire

CAMBRIDGE, Mass., Oct. 21, 2019 /PRNewswire/ -- SmartPharm Therapeutics, a preclinical-stage pharmaceutical company focused on developing next-generation, non-viral gene therapies for the treatment of rare diseases, today announced the appointment of John T. Potts, MD, DSc, to its Board of Directors. Dr. Potts' distinguished, five-decade career in healthcare and the life sciences spans medical disciplines and encompasses ground-breaking preclinical and clinical research.

"We are honored to welcome John to the SmartPharm Board. His acumen and experience will be invaluable to the Company during its journey into the clinic," said Steve Gorlin, Chairman of the Board for SmartPharm.

"I am excited to be joining SmartPharm Therapeutics and believe the Company's approach to next-generation, non-viral, gene-encoded protein therapies holds significant potential to impact medicine in a similar manner to biologics, while eliminating their onerous manufacturing and delivery systems," commented Dr. Potts.

Dr. Potts is the Jackson Distinguished Professor of Clinical Medicine at the Massachusetts General Hospital (MGH) and Harvard Medical School. After completing his MD at the University of Pennsylvania and internship and residency at MGH, he worked at the National Institutes of Health for almost a decade, studying protein chemistry with Nobel laureate Christian Anfinsen. He returned to MGH as Chief of Endocrinology in 1968 where he subsequently served as Director of Research, Chairman of the Department of Medicine and Physician-in-Chief. An internationally-recognized authority on calcium metabolism and the hormonal mechanisms that govern it, he has been a pioneer in identifying the chemistry and biology of clinical disorders of bone and mineral ion metabolism.

Dr. Potts has played an active role in the life sciences industry. He is a founder of Radius Health, Inc., a biopharmaceutical company focused on developing and commercializing innovative endocrine therapeutics in the areas of osteoporosis and oncology, where he currently serves as a Scientific Advisory Board Member. He has also served on advisory boards with MPM Capital and HealthCare Ventures. He has previously held board of directors positions with Cell Genesys, Zeltiq Aesthetics, Genentech and NantKwest.

An author on over 500 publications, his honors and awards include the Association of Professors of Medicine's Robert H. Williams, MD, Distinguished Chair of Medicine Award; the Biomedical Science Career Program Hope Award; the Endocrine Society's Fred Konrad Koch Award; and the American Society for Bone and Mineral Research's William F. Neumann Award. He holds active and honorary memberships in a number of scientific and professional organizations including the National Academy of Medicine, American Academy of Arts and Sciences, and the National Academy of Sciences.

About SmartPharm Therapeutics

SmartPharm Therapeutics Inc. is a privately-held, development-stage pharmaceutical company focused on developing next-generation, non-viral gene therapies for the treatment of serious or rare diseases with the vision of creating "biologics from within." The Company is currently developing a novel pipeline of non-viral, gene-encoded proteins for the treatment of conditions that require enzyme replacement therapy and tissue restoration. SmartPharm commenced operations in 2018 and is headquartered in Cambridge, MA, USA. For more information, please visit http://www.smartpharmtx.com.

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SmartPharm Therapeutics Appoints John T. Potts, MD, DSc, to Board of Directors - BioSpace

Vancouver, Canada, October 21, 2019 ISCT, the International Society for Cell and Gene Therapy, the global professional society of clinicians, researchers, regulatory specialists, technologists and industry partners in the cell and gene therapy sector, today announces it has formed a global consortium of a wide range of leading professional and education societies to combat the rise in the number of unproven commercial cell banking services. Full details of the statement can be foundhere.

The consortium partners include the International Society for Stem Cell Research (ISSCR), Society for Immunotherapy of Cancer (SITC), American Society for Transplantation and Cellular Therapy (ASTCT),American Society of Gene & Cell Therapy (ASGCT), European Society for Blood and Marrow Transplantation (EBMT), Foundation for the Accreditation of Cellular Therapy (FACT), Joint Accreditation Committee ISCT-EBMT (JACIE) and the Forum for Innovative Regenerative Medicine (FIRM).

The consortium has been formed following ISCT issuingpatient advice and concern on unproven T-cell preservation services on August 7, 2019. These services include the banking of T-cells, dental cells and cells for the derivation of induced pluripotent stem cells for potential therapeutic uses.

The joint statement from ISCT and the consortium partners includes an agreement on a number of key points. Commercial cell banking services are not supported by current scientific evidence, as opposed to the range of cell therapies such as CAR-T therapies, that follow established approval processes. Additionally, cell banking services cannot claim to know that the cells they preserve today could ever be appropriate for clinical use, could be used by manufacturers, or meet the requirements of many national and international regulatory agencies. As a result, there is no clear pathway to legitimate clinical use. All parties agree offering these services commercially to patients is thus premature, misleading, and drives false hope.

In addition, the ISCT joint statement makes clear that patients, being misled by these services, are thus prevented from giving a full and valid informed consent. Cell banking companies mislead patients in a number of ways, including using tokens of scientific legitimacy that suggest a stronger scientific basis than currently exists. These tokens include endorsements from individuals or scientific advisory boards that might not fully endorse the specific products, links to scientific articles, and references to ongoing clinical trials.

ISCTs raison detre is to lead the industry in supporting scientifically validated cell and gene therapies. As a result, ISCT will continue to welcome all innovations, including cell banking approaches, that increase the number of patients who can benefit from these therapies, said Bruce Levine,President-Elect, ISCT and one of the inventors of CAR-T therapies.However, ISCT also leads industry action on unproven cell therapies and services in the cell and gene sector. This is why ISCT has forged a consortium throughout the industry against the marketing of speculative cell banking services that do not have appropriate pre-clinical, and clinical evidence and a plausible pathway to the clinical use of banked cells. We collectively believe these banks have the potential to be detrimental to the future development of cell and gene therapies.

About ISCT

Established in 1992, ISCT, the International Society for Cell and Gene Therapy is a global society of clinicians, regulators, researchers, technologists and industry partners with a shared vision to translate cellular therapy into safe and effective therapies to improve patients lives worldwide.

ISCT is the global leader focused on pre-clinical and translational aspects of developing cell-based therapeutics, thereby advancing scientific research into innovative treatments for patients. ISCT offers a unique collaborative environment that addresses three key areas of translation: Academia, Regulatory and Commercialization. Through strong relationships with global regulatory agencies, academic institutions and industry partners, ISCT drives the advancement of research into standard of care.

Comprised of over 1,500 cell therapy experts across five geographic regions and representation from over 50 countries, ISCT members are part of a global community of peers, thought leaders and organizations invested in cell therapy translation. For more information about the society, key initiatives and upcoming meetings, please visit:

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ISCT forms cell and gene therapy sector-wide coalition to combat the rise of unproven commercial cell banking services - PharmiWeb.com

Tay-Sachs Disease Pipeline Insight Market Research 2019 report by Mart Research outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Tay-Sachs Disease pipeline landscape is provided which includes the disease overview and Tay-Sachs Disease treatment guidelines. The assessment part of the report embraces, in depth Tay-Sachs Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Tay-Sachs Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Tay-Sachs Disease Understanding

According to National Organization for Rare Disorders (NORD), Tay-Sachs disease is a rare, neurodegenerative disorder in which deficiency of an enzyme hexosaminidase A results in excessive accumulation of certain fats (lipids) known as gangliosides in the brain and nerve cells. This abnormal accumulation of gangliosides leads to progressive dysfunction of the central nervous system. This disorder is categorized as a lysosomal storage disease. Tay-Sachs disease is broken down into the classic or infantile form, the juvenile form, and the adult or late-onset form. In individuals with infantile Tay-Sachs disease, symptoms typically first appear between three and five months of age. In individuals with the late-onset form, symptoms may become apparent anytime from adolescence through the mid-30s.

Browse Full Tay-Sachs Disease Pipeline Insight Market Research Report @ https://martresearch.com/market-analysis/tay-sachs-disease-pipeline-insight/2/42159

Companies Covered in Tay-Sachs Disease Pipeline Insight Mart ResearchAxovant Gene TherapiesIntraBioActelion PharmaceuticalsCastle Creek PharmaceuticalsAvigenGene Therapy Research InstitutionOxford Glycobiology InstituteAmicus TherapeuticsAldagenRecursion Pharmaceuticals

Drugs Covered in Tay-Sachs Disease Pipeline Insight Market Research Report:AXO AAV GM2N-Acetyl-L-LeucineMiglustatCCP 010Lysosomal storage disease gene therapyResearch programme: Tay-Sachs disease gene therapyAcetylleucineResearch programme: calcium channel modulatorsCDP 923Research programme: Lysosomal storage disorder therapeuticsALD 601Research programme: rare genetic disorder therapeutics

r is categorized as a lysosomal storage disease. Tay-Sachs disease is broken down into the classic or infantile form, the juvenile form, and the adult or late-onset form. In individuals with infantile Tay-Sachs disease, symptoms typically first appear between three and five months of age. In individuals with the late-onset form, symptoms may become apparent anytime from adolescence through the mid-30s.

Companies Covered in Tay-Sachs Disease Pipeline Insight Market Research Report:

Axovant Gene Therapies

IntraBio

Actelion Pharmaceuticals

Castle Creek Pharmaceuticals

Avigen

Gene Therapy Research Institution

Oxford Glycobiology Institute

Amicus Therapeutics

Aldagen

Recursion Pharmaceuticals

Drugs Covered in Tay-Sachs Disease Pipeline Insight Market Research Report:

AXO AAV GM2

N-Acetyl-L-Leucine

Miglustat

CCP 010

Lysosomal storage disease gene therapy

Research programme: Tay-Sachs disease gene therapy

Acetylleucine

Research programme: calcium channel modulators

CDP 923

Research programme: Lysosomal storage disorder therapeutics

ALD 601

Research programme: rare genetic disorder therapeutics

Tay Sachs disease Pipeline Development Activities

The report provides insights into different therapeutic candidates in discovery and preclinical, phase 1, phase 2, and phase 3 stage. Drugs under development as a monotherapy or combination therapy are also included. It also analyses key players involved in Tay Sachs disease targeted therapeutics development with respective active and inactive (dormant or discontinued) projects with the appropriate reasons if available. Tay Sachs disease pipeline report covers 6+ companies. Some of the key players include IntraBio Inc (IB1001-201), Castle Creek Pharmaceuticals (CCP 010) etc.

The report is built using data and information traced from the researchers proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university web sites and industry-specific third party sources, etc.

Tay Sachs disease Analytical Perspective by Mart Research

In-depth Tay-Sachs Disease Commercial Assessment of products

This report provides an in-depth Commercial Assessment of therapeutic drugs have been included which comprises of collaborations, Licensing, Acquisition Deal Value Trends. The sub-segmentation is described in the report which includes Company-Company Collaborations (Licensing / Partnering), Company-Academia Collaborations, and Acquisition analysis in both Graphical and tabulated form.

Tay-Sachs Disease Clinical Assessment of products

The report comprises of comparative clinical assessment of products by development stage, product type, route of administration, molecule type, and MOA type across this indication.

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Scope of the report

The Tay Sachs disease report provides an overview of therapeutic pipeline activity for Tay Sachs disease across the complete product development cycle including all clinical and non-clinical stages

It comprises of detailed profiles of Tay Sachs disease therapeutic products with key coverage of developmental activities including technology, collaborations, licensing, mergers and acquisition, funding, designations and other product related details

Detailed Tay Sachs disease Research and Development progress and trial details, results wherever available, are also included in the pipeline study

Therapeutic assessment of the active pipeline products by development stage, product type, route of administration, molecule type, and MOA type

Coverage of dormant and discontinued pipeline projects along with the reasons if available across Tay Sachs disease

Reasons to Buy

Establish a comprehensive understanding of the current pipeline scenario across Tay Sachs disease to formulate effective R&D strategies

Assess challenges and opportunities that influence Tay Sachs disease R&D

Develop strategic initiatives by understanding the focus areas of leading companies.

Gather impartial perspective of strategies of the emerging competitors having potentially lucrative portfolio in this space and create effective counter strategies to gain competitive advantage. Get in detail information of each product with updated information on each project along with key milestones. Devise Tay Sachs disease in licensing and out licensing strategies by identifying prospective partners with progressing projects for Tay Sachs disease to enhance and expand business potential and scope. Our extensive domain knowledge on therapy areas support the clients in decision-making process regarding their therapeutic portfolio by identifying the reason behind the inactive or discontinued drugs.

Table of Content for Tay-Sachs Disease Pipeline Insight Market Research Report:Chapter One: Report IntroductionChapter Two: Tay-Sachs Disease (GM2 gangliosidosis, type 1, HexA deficiency, Hexosaminidase A deficiency, Hexosaminidase alpha-subunit deficiency (variant B))Chapter Three: Tay-Sachs Disease Current Treatment PatternsChapter Four: Tay-Sachs Disease Mart Researchs Analytical PerspectiveChapter Five: Tay-Sachs Disease Pipeline TherapeuticsChapter Six: Tay-Sachs Disease -Products AnalysisChapter Seven: Recent TechnologiesChapter Eight: Tay-Sachs Disease Key CompaniesChapter Nine: Tay-Sachs Disease Key ProductsChapter Ten: Dormant and Discontinued ProductsChapter Eleven: Tay-Sachs Disease Unmet NeedsChapter Twelve: Tay-Sachs Disease Future Perspectives

List of Tables for Tay-Sachs Disease Pipeline Insight Market Research Report:Table 1. Diagnostic GuidelinesTable 2. Treatment GuidelinesTable 3. Assessment SummaryTable 4. Company-Company Collaborations (Licensing / Partnering) AnalysisTable 5. Tay-Sachs Disease Acquisition AnalysisTable 6. Assessment by Phase of DevelopmentTable 7. Assessment by Product Type (Mono / Combination)Table 8. Assessment by Stage and Product TypeTable 9. Assessment by Route of AdministrationTable 10. Assessment by Stage and Route of AdministrationTable 11. Assessment by Molecule TypeTable 12. Assessment by Stage and Molecule TypeTable 13. Assessment by MOATable 14. Assessment by Stage and MOATable 15. Late Stage Products (Phase-III)Table 16. Mid Stage Products (Phase-II)Table 17. Early Stage Products (Phase-I)Table 18. Pre-clinical and Discovery Stage ProductsTable 19. Inactive ProductsTable 20. Dormant ProductsTable 21. Discontinued Products

To Check Discount on Tay-Sachs Disease Pipeline Insight Market Research Report @ https://martresearch.com/contact/discount/2/42159

About us: Research is and will always be the key to success and growth for any industry. Most organizations invest a major chunk of their resources viz. time, money and manpower in research to achieve new breakthroughs in their businesses. The outcome might not always be as expected thereby arising the need for precise, factual and high-quality data backing your research. This is where MART RESEARCH steps in and caters its expertise in the domain of market research reports to industries across varied sectors.

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Tay-Sachs Disease Pipeline Insight Market Research Report 2019: by Trends, Development, Types, Therapeutics, Drugs, Clinical Trials, Competitive...

CRISPR Therapeutics (CRSP) has been on my watchlist for a couple of years but the stars never aligned for me to pull the trigger on a buy. Due to some recent updates, I am moving CRSP up to the top of my year-end shopping list and will be stalking an entry point in the coming weeks or months.

I intend to discuss the primary reasons why I have waited for an entry, as well as what has tempted me to start a position. In addition, I discuss some of my leading downside risks associated with this ticker and how I plan to manage my potential position over the next year.

(Image Source: CRISPR)

CRISPR Therapeutics is a leader in gene editing, specifically in the advancement of Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated protein 9 "CRISPR-Cas9". CRISPR-Cas9 is a gene therapy, however, it is a revolutionary technology that intends to edit the deleterious gene. This is executed by altering explicit genomic sequences that should correct the bad gene or to alleviate the symptoms of the disease. Essentially, CRISPR is attempting to create curative gene therapies for both rare and common diseases.

CRISPR's programs can be divided up into ex vivo, where the company edits cells outside of the body and in vivo, the CRISPR-Cas9-based therapeutic targeted directly into the cells inside the body. CRISPR's leading pipeline programs take aim at hemoglobin related genetic disorders such as transfusion-dependent beta-thalassemia "TDT" and severe sickle cell disease "SCD". Both conditions desperately need a curative therapy because the current standard-of-care is insufficient. In addition to gene editing, the company has several gene-edited allogeneic cell therapy programs, including a few CAR-T candidates for hematological and solid tumor cancers. Furthermore, the company is taking gene-editing into regenerative medicines to address Type 1 diabetes, as well as taking on DMD and few other indications (Figure 1).

Figure 1: CRISPR Pipeline (Source: CRSP)

CRISPR-Cas9 for gene editing was co-invented by Dr. Emmanuelle Charpentier, who is one of CRISPR's founding members. The company now has the rights to Dr. Charpentier's CRISPR-Cas9 technologies, as well as some supplementary technology from other in-licensing efforts. What is more, CRISPR has a strong list of partnerships that will maximize CRISPR-Cas9's ability both clinically and commercially.

CRISPR has always been on my radar and I have admittedly traded the ticker a few times. However, I have been very cautious about investing in the company for a few reasons. One of which, is the fact that it is named after the technology associated with the gene therapies. Although there are other types of gene therapies, CRISPR appears to be the "brand" or term the market associates with all things gene therapy. Consequently, any bad news, setback, or regulatory failure in gene therapy has had a negative impact on the ticker. Essentially, CRISPR becomes a punching bag for anything that goes wrong in the industry. CRSP experienced strong selling pressure following Sarepta Therapeutics (SRPT) receiving a CRL for their DMD gene therapy. As a result, the stock fell from just under $50 a share to ~$43 per share in a few days. Of course, the rest of the industry experienced some selling pressure, but it appears CRSP gets the worst of it. As a result, I have been reluctant to commit to CRSP knowing that it will always be a target for shorts.

Another reason why I have passed on CRSP was due to its early pipeline. The company's pipeline is still mostly pre-clinical, so I would have been investing in science and not a mature product portfolio. Although I am happy to invest in an early-stage biotech/pharma company, I wanted to see if the FDA was willing to allow gene-editing therapies to be put into humans considering the controversy associated with the technology.

My last reason for holding off an investment was the valuation. The company's market valuation appeared to have a premium price on it. Admittedly, the company's high-priced valuation was due to its name and the hype around the gene therapy and not from a fundamental perspective. Although I am sold on the technology, I wasn't going to commit to a long-term investment to an early-stage company that is years away from a pivotal trial, regardless of the technology was from one of the co-founders of the CRISPR-Cas9.

In general, I knew the company was too early in the regulatory process and I expected the ticker to be toyed around with by traders and analysts. Therefore, I wanted to save myself from an unwanted rollercoaster ride of an investment.

The company is now a clinical-stage gene-editing company, which eliminates one of the reasons why I decided to hold off on starting a long-term investment. CRISPR's lead product candidate, CTX001, is ex-vivo CRISPR therapy that is being developed in collaboration with Vertex Pharmaceuticals (VRTX) for TDT or severe SCD. CTX001 is essentially a process that requires the company to engineer a patient's hematopoietic stem cells to increase the levels of fetal hemoglobin "HbF" in red blood cells.

Figure 2: CTX001 SCD (Source: CRSP)

The increase of HbF has the potential to assuage the need for transfusions for TDT and SCD patients.

Figure 3: CTX001 HbF (Source: CRSP)

CRISPR Therapeutics is enrolling in both Phase I/II studies of CTX001 in TDT and severe SCD. The company expects preliminary safety and efficacy data before the year-end. So far, the first TDT patient treated with CTX001 remains "transfusion independent, greater than four months following engraftment."

Now that CXT001 is in the clinic, we can expect several catalysts to come from these programs. Again, I did believe in the technology but the market needs to see that the pre-clinical data will transfer into humans. If the data is promising, we can expect an increase in attention from the market and from Street analysts. The company is expecting to report preliminary results for CTX001 by year-end, so I wouldn't mind having some skin-in-the-game ahead of that readout.

Another reason for a buy is the expanding use of the company's CRISPR-Cas9 technology. In addition to gene editing, the CRISPR-Cas9 has the potential to generate next-gen CAR-T therapies that may be superior to the contemporary autologous CAR-T (Figure 4).

Figure 4: CAR-T Highlights (Source: CRSP)

The company has programs including CTX110, a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting CD19. In pre-clinical mouse studies, the company detected that CTX110 extended the survival in CD19-positive xenograft tumors. In addition to CTX110, the company has CTX120 that is targeting BCMA in multiple myeloma and CTX130 that could target both solid tumors and hematologic malignancies.

Figure 3: CTX110 & CTX120 (Source: CRSP)

In preclinical studies both CTX120 and CTX130 were able to record "complete tumor elimination" in their respective targets.

Figure 4: CTX130 (Source: CRSP)

In addition to cell-therapies, CRISPR is using their ex vivo gene-editing in regenerative medicine by using stem cells to restore or exchange damaged or malfunctioning tissue. Their leading regenerative program is diabetes with their partner, ViaCyte Inc (Figure 5).

Figure 5: In Diabetes (Source: CRSP)

CRISPR has other in vivo disease targets that impact the liver, lung and muscle. These will use the company's lipid nanoparticle-based delivery vehicles "LNPs" and AAV vectors to help deliver the genetic material.

Figure 6: Neuro & Liver (Source: CRSP)

Other notable programs include Glycogen Storage Disease Type Ia "GSDIa", DMD and cystic fibrosis.

Admittedly, I have always seen CRISPR as a pure gene-editing company, however, they have a diverse pipeline and the technology to expand it into numerous other indications and areas of therapy. I expect the company will expand on their research efforts, which should increase the pending value of the company and add some catalysts to the calendar.

The company intends to advance their CRISPR-Cas9 therapeutics both autonomously and in strategic partnerships (Figure 7). The company has closed three major partnerships with Vertex, Casebia/Bayer (OTCPK:BAYRY) and ViaCyte. These partnerships not only provide CRISPR with some support, but they confirm that other reputable companies see CRISPR as a leader in this field.

Figure 7: CRSP Partnerships (Source: CRSP)

As the company expands its pipeline, I anticipate an increase in the number of strategic partnerships and deals. In fact, the company recently announced a partnership with KSQ Therapeutics to in-license IP to advance their cell therapy programs.

My final reason for stalking an entry is the improved risk/reward scenario. The stock appears to have lost some momentum and has had a relatively flat 2019. The stock has recently experienced a strong pull-back, yet, the company has made substantial progress in both the pipeline and strategic collaborations.

Figure 8: CRSP Daily (Source: Trendspider)

This recent pull-back has cut the market cap down to ~$2B which I believe is fair considering the long-term outlook for the company.

Figure 9: CRSP Valuation (Source: Seeking Alpha)

Looking at the CRSP's annual revenue estimates (Figure 10), we can see the Street expects the company to have relatively flat revenues for the next couple of years and will start to experience strong revenue growth in the second half of the next decade. This rapid growth is expected to drop the forward price-to-sales ratio below 1x at some point in 2027. The sector's average price-to-sales is about 5x, so that would equal ~$13B market cap in 2027.

Figure 10: CRSP Annual Revenue Estimates (Source: Seeking Alpha)

I understand this is years away, but I expect these estimates to only improve as the company continues to add pipeline programs and other strategic partnerships. Indeed, I could wait another year or two to further reduce my risk and cost for time. However, I don't want to be left out of this ticker if the company starts reporting curative results in multiple programs. What would happen if the company demonstrates the potential to cure Type 1 diabetes? Do you think it will be trading at a $2B market cap? What about if they cure cystic fibrosis? Sickle Cell? Eliminate renal cancer with an autologous CAR-T therapy? I believe the company would be undervalued at $2B even if they only cured one of these indications. Assuming the company has at least one winner in their current pipeline, I have to say the current risk/reward is enticing enough for me to start a speculative position.

I believe the biggest threat to CRISPR comes from the impressive competition from other gene therapy companies and institutions. These include companies that are working on CRISPR-Cas9 technology such as Intellia Therapeutics (NTLA) and most notably Editas Medicine (EDIT). Other gene-editing companies such as bluebird bio (BLUE), Sangamo Therapeutics (SGMO), Cellectis (CLLS), Precision BioSciences (DTIL), and Allogene Therapeutics (ALLO) use other gene-editing platforms. There is a multitude of gene therapy companies that I didn't mention, but I think you get my point. Some of these companies are not in the same league as CRISPR, however, I believe CRISPR's biggest threat is something that is currently in a theoretical stage or perhaps hasn't been thought of yet. As gene therapy products begin to hit the market and start dethroning current standard-of-care therapies, I expect big pharma to start devoting a larger percentage of their R&D to gene therapies. As a result, more universities and institutions will start pioneering new CRISPR tech and perhaps another level of gene editing. If they are successful, they will most likely dethrone CRISPR-Cas9 as cutting-edge technology.

CRISPR is a developmental biotech company, so of course, the financials will be an issue until the company has a product on the market. At the end of Q2, the company had $427.9M in the bank. The company's R&D expenses were $39.5M for Q2, which led to a net loss of $53.7M. Usually, I would consider $428M to be a strong cash position for pre-revenue biotech, but I don't think we are seeing peak cash-burn. The company has several programs that haven't even hit the clinic yet, so we can expect that cash position to melt rapidly in the coming years. In fact, the company recently secured a $200M at-the-market sale of common stock, so we should expect the company to tap the market when they need to. Even then, investors need to accept the strong possibility the company will execute secondary offerings in the future.

CRISPR Therapeutics has evolved over the past couple of years and so has my view of a potential investment. The company's premium name brought a premium price, which generated a substantial amount of attention from traders who have created detached or arbitrary valuations for the ticker. Now, the company has moved into the clinic and will be generating data that could reveal curative level results for dreadful diseases and conditions. If CRISPR is able to gain regulatory approval, the product could replace the current standard-of-care for the indication and eventually dominate as patients demand a cure, rather than dealing with a lifetime of medications or procedures.

Due to this potential, I am moving CRSP up to the top of my shopping list as we close out 2019. I expect many of the speculative biotech names to experience end-of-the-year tax selling, so I am going to keep a close eye on the charts to see if I can get a nice discount to establish a large position. In the meantime, I rely on the chart's technicals to find an entry. Returning to figure 8, we can see the stock has returned to its previous trading range after a month of selling pressure. If the share price is able to hold this area over the next couple of weeks, I will look to click the buy button on a low volume day. If the stock breaks below $35, I will most likely hold off on a buy until the end of the year. Once I have established a position, I will look to add to my position following data readouts with a goal to have a full-sized position by the end of 2021.

Disclosure: I am/we are long SRPT. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: I am also long BLUE.

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CRISPR Therapeutics: At The Top Of My Shopping List - Seeking Alpha

SAN DIEGO, Oct. 22, 2019 (GLOBE NEWSWIRE) -- CV Sciences, Inc. (OTCQB:CVSI) (the Company, CV Sciences, our, us or we), a preeminent supplier and manufacturer of hemp cannabidiol (CBD) products, today announced the appointment of Dr. Paul Blake to its Board of Directors effective October 21, 2019.

We are very pleased to have Dr. Blake join our Board of Directors, said Joseph Dowling, Chief Executive Officer of CV Sciences. His decades of experience leading pharmaceutical companies will be invaluable to us as we continue to strengthen our industry leadership position in CBD consumer products and drug development.

Dr. Blake brings with him deep expertise in clinical research, development, and medical affairs within the pharmaceutical industry. He currently serves as Chief Medical Officer at Heron Therapeutics Inc., a commercial-stage biotechnology company focused on improving the lives of patients suffering from pain or cancer by developing best-in-class treatments. At Heron, Dr. Blake is responsible for clinical research, medical affairs and drug safety.

Prior to joining Heron, he served as Chief Medical Officer of BioDelivery Sciences International, and before that he was the Chief Development Officer at Oxford BioMedica, a gene therapy company. His prior positions include Chief Medical Officer and Senior Vice President of Clinical Research and Development of Aeterna Zentaris, Inc., and Senior Vice President and then Executive Vice President of Worldwide Medical and Regulatory Operations at Cephalon, Inc. From 1992 to 1998, he held the position of Senior Vice President and Medical Director, Clinical Research and Development at SmithKline Beecham Pharmaceuticals (now GSK). Prior to that, he worked for ICI Pharmaceuticals (now Astra Zeneca) and G.D. Searle.

Dr. Blake is qualified in medicine from the Royal Free Hospital School of Medicine, London University. He is a Fellow of the Royal College of Physicians and a Fellow of the Faculty of Pharmaceutical Medicine of the Royal College of Physicians. He is also a Fellow of the American College of Clinical Pharmacology.

Dr. Blake has been involved in the development of several pharmaceutical drugs including: Tenormin, Zestril, Kytril, Relafen, Paxil, Coreg, Havrix, Hycamtin, Famvir, Requip, Avandia, Provigil and Fentora.

Dr. Blake served on the board of Memory Pharmaceutical, Inc., until it was purchased by Roche in 2009 and was a founding board member of Protez Pharmaceuticals, Inc., until it was purchased by Novartis in 2008. He was also a Director of ViaCell, Inc., until its purchase by Perkin Elmer in 2007 and of Oxford BioMedica from 2008 until 2016.

He has also served as a member of the Inter-Company Collaboration for AIDS Drug Development and as a trustee of the Childrens Health Fund.

I am excited to join CV Sciences Board of Directors and to contribute to the Companys mission of providing the highest-quality CBD products to consumers and patients, said Dr. Blake. CV Sciences is a leading pioneer in the CBD space and is uniquely positioned to continue to bring innovative products and novel therapeutics utilizing CBD to market.

About CV Sciences, Inc.

CV Sciences, Inc. (OTCQB:CVSI) operates two distinct business segments: a consumer product division focused on manufacturing, marketing and selling plant-based CBD products to a range of market sectors; and a drug development division focused on developing and commercializing CBD-based novel therapeutics utilizing CBD. The Companys PlusCBD Oil products are sold at more than 5,500 retail locations through the U.S. and it is the top-selling brand of hemp-derived CBD on the market, according to SPINS, the leading provider of syndicated data and insights for the natural, organic and specialty products industry. CV Sciences state-of-the-art facility follows all guidelines for Good Manufacturing Practices (GMP) and the Companys full spectrum hemp extracts are processed, produced, and tested throughout the manufacturing process to confirm the cannabinoid content meets strict company standards. With a commitment to science, PlusCBD Oils benefits in healthy people are supported by human clinical research data, in addition to three published clinical case studies available on PubMed.gov. PlusCBD Oil was the first hemp CBD supplement brand to invest in the scientific evidence necessary to receive self-affirmed Generally Recognized as Safe (GRAS) status. CV Sciences, Inc. has primary offices and facilities in San Diego, California. Additional information is available from OTCMarkets.com or by visiting http://www.cvsciences.com.

FORWARD-LOOKING DISCLAIMER

This press release may contain certain forward-looking statements and information, as defined within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, and is subject to the Safe Harbor created by those sections. This material contains statements about expected future events and/or financial results that are forward-looking in nature and subject to risks and uncertainties. Such forward-looking statements by definition involve risks, uncertainties.

CONTACT INFORMATION:Investor Contact:ICRScott Van Winkle617-956-6736scott.vanwinkle@icrinc.com

Media Contact:ICRCory Ziskind646-277-1232cory.ziskind@icrinc.com

Originally posted here:
CV Sciences, Inc. Appoints Dr. Paul Blake to its Board of Directors - GlobeNewswire

GlobalGene TherapyMarket,added byFior Marketsin its huge repository, offers a brilliant, complete research study of the globalGene Therapymarket. The report contains a detailed study of crucial market dynamics, including growth drivers, restraints, and opportunities. It chiefly focuses on current and historical market scenarios. It takes into account market competition, segmentation, geographical expansion, regional growth, market size, and other factors. The research study will surely benefit stakeholders, market players, investors, and other market participants. They will get a sound understanding of the global market and the industry.

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Taylor is the senior news reporter at Honest Newsly. He covers business and commodities news. Formerly, he worked with Western Mass News and Meredith COrporation as the senior news reporter.

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Gene Therapy Market 2019 Global Opportunity Analysis and Industry Forecast to 2025 - Honest Newsly

Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Pipeline Insight Market Research 2019 report by Mart Research outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Fanconi Anemia (FA) Fanconi Hypoplastic Anemia pipeline landscape is provided which includes the disease overview and Fanconi Anemia (FA) Fanconi Hypoplastic Anemia treatment guidelines. The assessment part of the report embraces, in depth Fanconi Anemia (FA) Fanconi Hypoplastic Anemia commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Fanconi Anemia (FA) Fanconi Hypoplastic Anemia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Fanconi Anemia Understanding

According to the Cancer and Blood Disorders Center, Fanconi Anemia (Fanconi hypoplastic anemia, Fanconi pancytopenia, Fanconi panmyelopathy) is a rare inherited disease characterized by multiple physical abnormalities, bone marrow failure, and a higher than normal risk of cancer. Researchers have shown that mutations in one of at least 15 different genes can cause FA. The proteins normally produced by these genes form a kind of cellular machine that helps detect and repair damaged DNA in blood stem cells and other cells in the body, in FA this damaged DNA repair is slowed. Therefore, blood stem cells (in the bone marrow) accumulate damaged DNA and do not survive. FA is usually discovered between birth and age 10-15 years; however, there also have been cases identified in adulthood. FA occurs equally in males and females. It has been identified in all ethnic groups. Researchers continue to clone and characterize the genes responsible for FA, which is bringing considerable progress in the diagnosis and understanding of this disease. It is more common in male as compared to female.

Fanconi Anemia Pipeline Development Activities

The report provides insights into different therapeutic candidates in discovery and preclinical, phase 1, phase 2, and phase 3 stage. Drugs under development as a monotherapy or combination therapy are also included. It also analyses key players involved in FA targeted therapeutics development with respective active and dormant or discontinued projects. FA pipeline report covers 7+ companies. Some of the key players include Rocket pharma (RP-L102), Bellicum Pharmaceuticals (BPX-501), etc.

The report is built using data and information traced from the researchers proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university web sites and industry-specific third party sources, etc.

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Companies Covered in Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Pipeline Insight Mart Research:Abeona TherapeuticsRocket PharmaceuticalsCIEMATCellenkosPluristem TherapeuticsBioLineRxForesee PharmaceuticalsGamida CellAmgenNovartis

Drugs Covered in Fanconi Anemia (FA) Fanconi Hypoplastic AnemiaPipeline Insight Market Research:Research programme: rare haematological disorder gene therapiesResearch programme: gene therapiesRP L101RP L102CK 0801PLX R18MotixafortideFP 045OmidubicelRomiplostimEltrombopag

Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Analytical Perspective by Mart Research

In-depth Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Commercial Assessment of products

This report provides an in-depth Commercial Assessment of therapeutic drugs have been included which comprises of collaborations, Licensing, Acquisition Deal Value Trends. The sub-segmentation is described in the report which includes Company-Company Collaborations (Licensing / Partnering), Company-Academia Collaborations, and Acquisition analysis in both Graphical and tabulated form.

Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Clinical Assessment of products

The report comprises of comparative clinical assessment of products by development stage, product type, route of administration, molecule type, and MOA type across this indication.

Scope of the report

The Fanconi Anemia (FA) Fanconi Hypoplastic Anemia report provides an overview of therapeutic pipeline activity for Fanconi Anemia (FA) Fanconi Hypoplastic Anemia across the complete product development cycle including all clinical and non-clinical stages

It comprises of detailed profiles of Fanconi Anemia (FA) Fanconi Hypoplastic Anemia therapeutic products with key coverage of developmental activities including technology, collaborations, licensing, mergers and acquisition, funding, designations and other product related details

Detailed Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Research and Development progress and trial details, results wherever available, are also included in the pipeline study

Therapeutic assessment of the active pipeline products by development stage, product type, route of administration, molecule type, and MOA type

Coverage of dormant and discontinued pipeline projects along with the reasons if available across Fanconi Anemia (FA) Fanconi Hypoplastic Anemia.

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Table of Content for Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Pipeline Insight Market Research Report:Chapter One: Report IntroductionChapter Two: Fanconi Anemia (Fanconi hypoplastic anemia, Fanconi pancytopenia, Fanconi panmyelopathy)Chapter Three: Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Current Treatment PatternsChapter Four: Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Mart Researchs Analytical PerspectiveChapter Five: Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Pipeline TherapeuticsChapter Six: Fanconi Anemia (FA) Fanconi Hypoplastic Anemia -Products AnalysisChapter Seven: Recent TechnologiesChapter Eight: Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Key CompaniesChapter Nine: Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Key ProductsChapter Ten: Dormant and Discontinued ProductsChapter Eleven: Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Unmet NeedsChapter Twelve: Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Future Perspectives

List of Tables for Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Pipeline Insight Market Research Report:Table 1. Diagnostic GuidelinesTable 2. Treatment GuidelinesTable 3. Assessment SummaryTable 4. Company-Company Collaborations (Licensing / Partnering) AnalysisTable 5. Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Acquisition AnalysisTable 6. Assessment by Phase of DevelopmentTable 7. Assessment by Product Type (Mono / Combination)Table 8. Assessment by Stage and Product TypeTable 9. Assessment by Route of AdministrationTable 10. Assessment by Stage and Route of AdministrationTable 11. Assessment by Molecule TypeTable 12. Assessment by Stage and Molecule TypeTable 13. Assessment by MOATable 14. Assessment by Stage and MOATable 15. Late Stage Products (Phase-III)Table 16. Mid Stage Products (Phase-II)Table 17. Early Stage Products (Phase-I)Table 18. Pre-clinical and Discovery Stage ProductsTable 19. Inactive ProductsTable 20. Dormant ProductsTable 21. Discontinued Products

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Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Fanconi Hypoplastic Anemia Pipeline Insight Market Research Report 2019: by Trends, Development, Types,...

Ichthyosis Pipeline Insight Market Research 2019 report by Mart Research outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Ichthyosis pipeline landscape is provided which includes the disease overview and Ichthyosis treatment guidelines. The assessment part of the report embraces, in depth Ichthyosis commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Ichthyosis collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Intrahepatic Cholangiocarcinoma Understanding

Ichthyosis, also known as disorders of keratinization (DOK), encompass a heterogeneous group of skin diseases linked by the common finding of abnormal barrier function, which initiates a default compensatory pathway of hyperproliferation. The unifying phenotypic feature of the ichthyosis is the clinical manifestation of localized and/or generalized scaling. Other include erythroderma, palmoplantar keratoderma, hypohidrosis, and recurrent infections. Most known forms of ichthyosis are a rare genetic disorder, which are hereditary in nature (some forms are dominant and some are recessive). Although ichthyoses are primarily inherited disorders with onset at or shortly after birth, rare acquired forms have been reported in the setting of malignancy, nutritional deficiency, and autoimmune or infectious disease. Mutations in over 50 genes have been reported to cause ichthyoses and these affect a host of cellular functions including DNA repair, lipid biosynthesis, adhesion, and desquamation as well as other pathways. Ichthyosis vulgaris (IV) and X-linked recessive ichthyosis (XLRI) are classified as the common ichthyoses, given their high prevalence.

Ichthyosis Pipeline Development Activities

The report provides insights into different therapeutic candidates in discovery and preclinical, phase 1, phase 2, and phase 3 stage. Drugs under development as a monotherapy or combination therapy are also included. It also analyses key players involved in Ichthyosis targeted therapeutics development with respective active and dormant or discontinued projects Ichthyosis pipeline report covers 11+ companies. Some of the key players include Mayne Pharma/Galderma (Trifarotene), Patagonia Pharmaceuticals (PAT-001), Exicure (XCUR 17), etc.

The report is built using data and information traced from the researchers proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university web sites and industry-specific third party sources, etc.

Ichthyosis Analytical Perspective by Mart Research

In-depth Ichthyosis Commercial Assessment of products

This report provides an in-depth Commercial Assessment of therapeutic drugs have been included which comprises of collaborations, Licensing, Acquisition Deal Value Trends. The sub-segmentation is described in the report which includes Company-Company Collaborations (Licensing / Partnering), Company-Academia Collaborations, and Acquisition analysis in both Graphical and tabulated form.

Ichthyosis Clinical Assessment of products

The report comprises of comparative clinical assessment of products by development stage, product type, route of administration, molecule type, and MOA type across this indication.

Browse Full Ichthyosis Pipeline Insight Market Research Report @ https://martresearch.com/market-analysis/ichthyosis-pipeline-insight/2/42147

Companies Covered in Ichthyosis Pipeline Insight Mart Research:Patagonia PharmaceuticalsCellegy PharmaceuticalsMayne Pharma/GaldermaJanssen PharmaceuticaFoamixDermaXonAldeyra TherapeuticsAzitraBristol-Myers SquibbAllerganChugai PharmaceuticalKrystal BiotechGlaxoSmithKlineNovartisTeijin PharmaExicure

Drugs Covered in IchthyosisPipeline Insight Market Research:IsotretinoinMonolaurinTrifaroteneLiarozoleAmmonium lactateDX 0386ReproxalapDX 0308Research programme: skin disorder therapeuticsAcitretinAmmonium lactate; BMS-186091MaxacalcitolBercolagene telserpavecResearch programme: gene therapies; KB 105BMS 181163SecukinumabTacalcitolResearch programme: SNA based therapeuticsXCUR 17DX 0332

Ichthyosis Analytical Perspective by Mart Research

In-depth Ichthyosis Commercial Assessment of products

This report provides an in-depth Commercial Assessment of therapeutic drugs have been included which comprises of collaborations, Licensing, Acquisition Deal Value Trends. The sub-segmentation is described in the report which includes Company-Company Collaborations (Licensing / Partnering), Company-Academia Collaborations, and Acquisition analysis in both Graphical and tabulated form.

Ichthyosis Clinical Assessment of products

The report comprises of comparative clinical assessment of products by development stage, product type, route of administration, molecule type, and MOA type across this indication.

Scope of the report

The Ichthyosis report provides an overview of therapeutic pipeline activity for Ichthyosis across the complete product development cycle including all clinical and non-clinical stages

It comprises of detailed profiles of Ichthyosis therapeutic products with key coverage of developmental activities including technology, collaborations, licensing, mergers and acquisition, funding, designations and other product related details

Detailed Ichthyosis Research and Development progress and trial details, results wherever available, are also included in the pipeline study

Therapeutic assessment of the active pipeline products by development stage, product type, route of administration, molecule type, and MOA type

Coverage of dormant and discontinued pipeline projects along with the reasons if available across Ichthyosis.

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Reasons to Buy

Establish a comprehensive understanding of the current pipeline scenario across Ichthyosis to formulate effective R&D strategies

Assess challenges and opportunities that influence Ichthyosis R&D

Develop strategic initiatives by understanding the focus areas of leading companies.

Gather impartial perspective of strategies of the emerging competitors having potentially lucrative portfolio in this space and create effective counter strategies to gain competitive advantage

Get in detail information of each product with updated information on each project along with key milestones

Devise Ichthyosis in licensing and out licensing strategies by identifying prospective partners with progressing projects for Ichthyosis to enhance and expand business potential and scope

Our extensive domain knowledge on therapy areas support the clients in decision-making process regarding their therapeutic portfolio by identifying the reason behind the inactive or discontinued drugs

Table of Content for Ichthyosis Pipeline Insight Market Research Report:Chapter One: Report IntroductionChapter Two: IchthyosisChapter Three: Ichthyosis Current Treatment PatternsChapter Four: Ichthyosis Mart Researchs Analytical PerspectiveChapter Five: Ichthyosis Pipeline TherapeuticsChapter Six: Ichthyosis -Products AnalysisChapter Seven: Recent TechnologiesChapter Eight: Ichthyosis Key CompaniesChapter Nine: Ichthyosis Key ProductsChapter Ten: Dormant and Discontinued ProductsChapter Eleven: Ichthyosis Unmet NeedsChapter Twelve: Ichthyosis Future Perspectives

List of Tables for Ichthyosis Pipeline Insight Market Research Report:Table 1. Diagnostic GuidelinesTable 2. Treatment GuidelinesTable 3. Assessment SummaryTable 4. Company-Company Collaborations (Licensing / Partnering) AnalysisTable 5. Ichthyosis Acquisition AnalysisTable 6. Assessment by Phase of DevelopmentTable 7. Assessment by Product Type (Mono / Combination)Table 8. Assessment by Stage and Product TypeTable 9. Assessment by Route of AdministrationTable 10. Assessment by Stage and Route of AdministrationTable 11. Assessment by Molecule TypeTable 12. Assessment by Stage and Molecule TypeTable 13. Assessment by MOATable 14. Assessment by Stage and MOATable 15. Late Stage Products (Phase-III)Table 16. Mid Stage Products (Phase-II)Table 17. Early Stage Products (Phase-I)Table 18. Pre-clinical and Discovery Stage ProductsTable 19. Inactive ProductsTable 20. Dormant ProductsTable 21. Discontinued Products

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Ichthyosis Pipeline Insight Market Research Report 2019: by Trends, Development, Types, Therapeutics, Drugs, Clinical Trials, Competitive Analysis...

Pratteln, Switzerland, October 21, 2019 Santhera Pharmaceuticals (SIX: SANN) announces that the UKs Medicines and Healthcare Products Regulatory Agency (MHRA) has informed ReveraGen BioPharma about having designated vamorolone as Promising Innovative Medicine (PIM) for the treatment of Duchenne muscular dystrophy (DMD).

We congratulate ReveraGen on this success and are excited about the PIM designation as it further validates the potential of vamorolone as an innovative treatment approach addressing the high unmet medical need in young patients with DMD, said Thomas Meier, PhD, CEO of Santhera.

The PIM designation indicates that the UK MHRA considers vamorolone a promising candidate for the Early Access to Medicines Scheme (EAMS). In the UK, the EAMS, of which PIM is the first step, aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need.

Vamorolone is a first-in-class steroidal anti-inflammatory investigational drug in development as treatment for DMD. Data from non-clinical and clinical studies indicated that vamorolone treatment results in a persistent improvement of muscle function with less adverse effects typically reported for traditional corticosteroids [1-6].

Vamorolone has been granted Orphan Drug status in the US and in Europe and has received Fast Track and Rare Pediatric Disease designations by the US FDA.

About Vamorolone first-in-class dissociative steroid

Vamorolone is a first-in-class drug candidate that binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors. This has the potential to dissociate efficacy from typical steroid safety concerns and therefore could replace existing corticosteroids, the current standard of care in children and adolescent patients with DMD. There is significant unmet medical need in this patient group as high dose corticosteroids have severe systemic side effects that detract from treatment compliance and patient quality of life.

The currently ongoing 48-week Phase IIb VISION-DMD study (VBP15-004; NCT03439670) is designed as a pivotal trial to demonstrate efficacy and safety of vamorolone compared with prednisone and placebo in 120 boys aged 4 to <7 with DMD that have not yet been treated with corticosteroids. For more information: https://vision-dmd.info/2b-trial-information.

Vamorolone is being developed by US-based ReveraGen BioPharma Inc. with participation in funding and design of studies by several international non-profit foundations, the US National Institutes of Health, the US Department of Defense and the European Commissions Horizon 2020 program. In November 2018, Santhera acquired from Idorsia the option to an exclusive sub-license to vamorolone in all indications and all countries worldwide (except Japan and South Korea).

About Santhera

Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular and pulmonary diseases with high unmet medical need. Santhera is building a Duchenne muscular dystrophy (DMD) product portfolio to treat patients irrespective of causative mutations, disease stage or age. A marketing authorization application for Puldysa (idebenone) is currently under review by the European Medicines Agency. Santhera has an option to license vamorolone, a first-in-class dissociative steroid currently investigated in a pivotal study in patients with DMD to replace standard corticosteroids. The clinical stage pipeline also includes POL6014 to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases, as well as omigapil and an exploratory gene therapy approach targeting congenital muscular dystrophies. Santhera out-licensed ex-North American rights to its first approved product, Raxone (idebenone), for the treatment of Leber's hereditary optic neuropathy (LHON) to Chiesi Group. For further information, please visit http://www.santhera.com.

Raxone and Puldysa are trademarks of Santhera Pharmaceuticals.

About ReveraGen BioPharma

ReveraGen was founded in 2008 to develop first-in-class dissociative steroidal drugs for Duchenne muscular dystrophy and other chronic inflammatory disorders. The development of ReveraGens lead compound, vamorolone, has been supported through partnerships with foundations worldwide, including Muscular Dystrophy Association USA, Parent Project Muscular Dystrophy, Foundation to Eradicate Duchenne, Save Our Sons, JoiningJack, Action Duchenne, CureDuchenne, Ryans Quest, Alexs Wish, DuchenneUK, Pietros Fight, Michaels Cause, and Duchenne Research Fund. ReveraGen has also received generous support from the US Department of Defense CDMRP, National Institutes of Health (NCATS, NINDS, NIAMS), and European Commission (Horizons 2020). http://www.reveragen.com

References:

[1] Hoffman EP et al. (2019). Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. Neurology 93(13):e1312-e1323. doi:10.1212/WNL.0000000000008168

[2] Hoffman EP et al. (2018). Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes. Steroids 134: 43-52.

[3] Conklin LS et al. (2018). Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug. Pharmacol Res. 136:140-150. doi: 10.1016/j.phrs.2018.09.007.

[4] Mavroudis PD et al. (2019). Population pharmacokinetics of vamorolone (VBP15) in healthy men and boys with Duchenne muscular dystrophy. J Clin Pharmacol. 59(7):979-988. doi: 10.1002/jcph.1388.

[5] Heier CR at al. (2013). VBP15, a novel antiinflammatory and membranestabilizer, improves muscular dystrophy without side effects. EMBO Mol Med 5: 15691585

[6] Heier CR et al. (2019). Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy. Life Science Alliance DOI 10.26508/lsa.201800186

For further information please contact:

Santhera

public-relations@santhera.com or

Eva Kalias, Head External Communications

Phone: +41 79 875 27 80

eva.kalias@santhera.com

ReveraGen

Eric Hoffman, PhD, CEO

Phone: +1 240-672-0295

eric.hoffman@reveragen.com

Disclaimer / Forward-looking statements

This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.

# # #

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Vamorolone Designated Promising Innovative Medicine (PIM) for Treatment of Duchenne Muscular Dystrophy by the UK MHRA - BioSpace

Cancer Gene Therapy Market Report 2019 to 2024 is the definitive study of the global Cancer Gene Therapy market. The content includes orientation technology, industry drivers, geographic trends, market statistics, market forecasts, producers, and equipment suppliers.

The report firstly introduced the Cancer Gene Therapy basics: definitions, classifications, applications and market overview; product specifications; manufacturing processes; cost structures, raw materials and so on. Then it analyzed the worlds main region market conditions, including the product price, profit, capacity, production, supply, demand and market growth rate and forecast etc. In the end, the report introduced new project SWOT analysis, investment feasibility analysis, and investment return analysis.

About Cancer Gene Therapy Industry

Analyst projects that the Cancer Gene Therapy market size will grow from XX Million USD in 2018 to XX Million USD by 2024, at an estimated CAGR of XX%. The base year considered for the study is 2018, and the market size is projected from 2019 to 2024.

Look insights of Global Cancer Gene Therapyindustry market research report athttps://www.pioneerreports.com/report/411426

The overviews, SWOT analysis and strategies of each vendor in the Cancer Gene Therapy market provide understanding about the market forces and how those can be exploited to create future opportunities.

Key Players in this Cancer Gene Therapy market are:

By TypeGene Induced Immunotherapy, Oncolytic Virotherapy, Gene Transfer

By ApplicationHospitals, Diagnostics Centers, Research Institutes

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This research report consists of the worlds crucial region market share, size (volume), trends including the product profit, price, Value, production, capacity, capability utilization, supply, and demand and industry growth rate.

Important application areas of Cancer Gene Therapy are also assessed on the basis of their performance. Market predictions along with the statistical nuances presented in the report render an insightful view of the Cancer Gene Therapy market. The market study on Global Cancer Gene Therapy Market 2018 report studies present as well as future aspects of the Cancer Gene Therapy Market primarily based upon factors on which the companies participate in the market growth, key trends and segmentation analysis.

Geographically this report covers all the major manufacturers from India, China, USA, UK, and Japan. The present, past and forecast overview of Cancer Gene Therapy market is represented in this report.

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The Cancer Gene Therapy Market Report is Prepared with the Main Agenda to Cover the following points:

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No of Pages in Cancer Gene Therapy Market:NOP

Analysis & Forecast Time Period: 2015-2024

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Cancer Gene Therapy Market Size, Market Opportunities, SWOT Analysis, Key Players and Forecast to 2024 - The Charterian

I burned my finger the other day. I didnt feel it right away, which is not that odd of an occurrence for me.

Often, I dont feel the heat and dont realize I have burned myself while cooking. I have Charcot-Marie-Tooth (CMT) type 1A, which may affect the feeling in my extremities.

CMT is also known as hereditary motor sensory neuropathy or peroneal muscular atrophy. Faulty genes may damage nerve cells or interfere with the formation of myelin, an insulating material that protects electrical signals as they travel through the bodys nervous system, including to the arms and legs.

When the myelin sheath is damaged, nerve impulses can slow or stop. That may explain why it can take 30 seconds for me to feel pain after stubbing my toe or burning myself. It would be nice if there was a way to stop that effect of CMT.

There is ongoing research on ways to treat CMT. One recent study claimed that varying amounts of lecithin seemed to ease the course of the disease, but this has not been tested on humans.

Pharnext is sponsoring clinical trials for PXT3003, a combination of baclofen, naltrexone, and sorbitol. According to the manufacturer, PXT3003 has shown positive effects on muscle cells, neuromuscular junctions, and immune cells. In current studies, the investigational medications long-term safety is being researched. Meanwhile, earlier this year, the U.S. Food and Drug Administration granted fast track designation for PXT3003s development.

This is an exciting time for CMT1A patients. The research is promising. In preclinical trials, PXT3003 inhibited the overexpression of the PMP22 gene and eased neuromuscular symptoms. In clinical trials, patients showed improvements. The amount of research and the positive results give us hope that a treatment may be on the horizon.

For now, only mobility aids, physical therapy, and medication can help ease the pain. These help us manage everyday living, but dont target the cause of the disease.

I am excited to see where the research leads. My hope is that these treatments are found to be effective and can help those of us with CMT1A. I would be happy to have medication available that stops the progression of my symptoms. It would be even better if the effects could be reversed, but I know that is asking a lot.

I would settle for not burning my fingertips when cooking.

***

Note: Charcot-Marie-Tooth News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Charcot-Marie-Tooth News or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to Charcot-Marie-Tooth.

Jill Price is a fourth grade teacher and a mom to a teenage son. She was diagnosed with CMT 1a at the age of 2. Jill loves to travel and enjoys spending time with her family and friends.

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Is a Treatment for CMT in the Works? - Charcot-Marie-Tooth News

NEW YORK, Oct. 21, 2019 /PRNewswire/ --

Predictive Genetic Testing & Consumer/Wellness Genomics Market Size, Share & Trends Analysis Report By Test Type (Population Screening, Susceptibility), By Application, By Setting Type, And Segment Forecasts, 2019 - 2025

Read the full report: https://www.reportlinker.com/p05807251/?utm_source=PRN

The global predictive genetic testing & consumer/wellness genomics market size is expected to reach USD 8.5 billion by 2025, registering a CAGR of 13.9% over the forecast period. Factors influencing the market progression include growing awareness about utilization of genetic tests that aid in prediction of gene susceptibility to disease development prior to symptoms. Moreover, rise in sales of these products owing to growing inclination of physicians is driving the market.

Introduction of novel platforms in next-generation sequencing technology aids in enhancing the accuracy of predictive genetic and consumer genomics kits. Market participants are engaged in implementing novel protocols to launch products that require minimal technical assistance and provide optimal customer satisfaction.

Pharmaceutical firms are engaged in several agreement models with genomic vendors for the release of novel therapeutics based on patient's phenotypic and genotypic information. For instance, in July 2018, GlaxoSmithKline plc purchased 23andMe's customer data to develop a new drug, thus promoting patient-centered healthcare.

Further key findings from the study suggest: High adoption rate of these products in clinical practice for detection of disease susceptibility resulted in the largest share of this segment in 2018 Growing awareness related to direct to consumer (DTC) genetic tests and entry of new players is attributive to the fastest growth of the consumer genomics segment Genetic susceptibility tests are of great interest in gynecology and endocrinology-related research and diagnosis, thus allowing it to capture the maximum revenue share. These tests enable the identification of susceptible genes or mutations in adenomyosis, endometriosis, and postmenopausal osteoporosis Application of predictive genetic and consumer genomics is highest in the detection of breast and ovarian cancer as these tests hold the potential to identify BRCA mutations in patients Advent of predictive tests for identification of MLH1, MSH2 genes for colorectal cancer diagnosis is expected to fuel market growth in the coming years Colorectal screening initiatives such as mass screening and population-based screening are expected to boost segment growth North America dominated the market with the largest revenue share in 2018 owing to the presence of major players and high adoption rate Asia Pacific is expected to witness substantial growth in the coming years owing to increasing adoption of these products in developing economies Manufacturers contributing significantly to market growth include Pathway Genomics; ARUP Laboratories; BGI; Illumina, Inc.; 23andMe, Inc.; Color Genomics Inc.; and Myriad Genetics, Inc. Established as well as emerging companies are involved in adopting strategic moves for the launch of novel products.

Read the full report: https://www.reportlinker.com/p05807251/?utm_source=PRN

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The global predictive genetic testing & consumer/wellness genomics market size is expected to reach USD 8.5 billion by 2025, registering a CAGR of...