Archive for the ‘Gene Therapy Research’ Category

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

By

By

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This is the one market research report to help you make the right strategic decisions.

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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.

Look into Table of Content of Cancer Gene Therapy Market Report at https://www.pioneerreports.com/TOC/411426

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

Report Price: USD 3500

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

CAMBRIDGE, Mass. Sanofi is accelerating nascent efforts in gene therapy, aiming to use its expertise in vaccines to catch up in a competitive field that's well ahead of the French pharma.

The company has prioritized gene therapy programs amid a broader effort to boost internal R&D speed and impact, said John Reed, Sanofi's head of research and development, in a Wednesday interview at Sanofi's Cambridge office.

"When I joined, I saw that we were dabbling in gene therapy and decided that we need to get more serious about gene therapy if we are going to continue to be impactful in that space," said Reed, who came over to Sanofi from Roche last July.

In particular, the company is retrofitting one of its vaccine facilities near Lyon, France, to produce GMP-grade adeno-associated viral vectors, or AAVs. Reed said he expects the plant to be operational in about a year.

The new R&D chief is steering the company away from areas for which it's historically been known, including, most notably, cardiovascular disease and diabetes. Sanofi is largely exiting cardiovascular R&D and is cutting spending in half on diabetes R&D, Reed said.

While vaccines make up a comparatively smaller portion of Sanofi's revenues, Reed noted the company's decades-long expertise in producing inactivated viruses could translate well to gene therapy. Reed was recently in Lyon to discuss the budget and headcount requirements for the change, he said.

"We have an opportunity to really leverage those competencies around vaccines for the gene therapy area," Reed said. "We are looking at how we can use that as a competitive advantage to be players in that space."

Several of Sanofi's pharma peers have bet heavily on gene therapy, investing in manufacturing and snapping up biotech leaders through multi-billion dollar acquisitions, such as by Novartis for AveXis and Roche for Spark Therapeutics.

Smaller companies like BioMarin Pharmaceutical, meanwhile, hold sizable leads in therapeutic areas that Sanofi hopes to play a larger role in, like hemophilia.

Reed acknowledged an acquisition "could be an accelerator" in establishing Sanofi's presence in cell and gene therapy.

"We flirt with those things all the time," he said, when asked about his openness to a deal like those for AveXis and Spark. "It's a bit challenging to point your finger at any one gene therapy company and say that solves all our problems."

"It's been really tough to pull the trigger on something like that," he added. "In the interim, we've been establishing the capabilities more internally."

How much it would be willing to pay, or afford, is another question. Under former CEO Olivier Brandicourt, the company last year targeted roughly 20 billion euros in acquisitions, a budget largely consumed by deals for Bioverativ and Ablynx in the blood disease space.

The company's first AAV-delivered gene therapy recently entered the clinic for a form of a rare eye disease called Leber congenital amaurosis, Reed added.

Two gene-edited cell therapies are in Phase 1/2 testing via a collaboration with Sangamo Therapeutics. Other programs remain preclinical as the group works on establishing GMP manufacturing capabilities.

All of this is taking place against a backdrop of change for Sanofi research and development teams.

Reed is working to narrow the company's focus to advance only first- or potentially best-in-class therapies, a bar that led Sanofi to cut several dozen programs from its pipeline earlier this year.

Reed has also restructured employee's incentives, taking away bonuses for starting projects and replacing them with an emphasis on starting first-in-human studies, a milestone Sanofi usually reaches slower than industry leaders.

"I don't want to reward people for starting projects, I want to reward them for finishing projects," he said. "We have too many projects."

Part of that's involved reducing bureaucracy and streamlining decision-making, moving from 33 committees that interact with R&D teams to three. Reed's given decision-making authority to team leaders for each molecule, calling them CEOs of their drug candidate.

Even before Reed came on board, productivity had begun to improve from a nadir in 2014, when Sanofi's entire organization produced only two clinical candidates that year. Now, Sanofi is delivering about six per year and, with the 2018 acquisitions of Bioverativ and Ablynx, should reach eight or nine per year.

Still, of the last 10 drugs Sanofi has won approvals for, only one was an internal project, Reed said. For the company's next 10 assets, Reed expects six or seven to have been internally developed.

As Reed re-focuses, Sanofi has exited or restructured partnerships this year with Regeneron, Alnylam Pharmaceuticals and Lexicon Pharmaceuticals.

Paring down the pipeline and restructuring deals also speaks to Sanofi's R&D budget, which the company expects to keep flat for the next few years. The pharma spends about half what companies with larger revenues like Pfizer, Novartis and Roche do.

Reed says the ultimate goal is to bring about 12 programs into clinical development each year, and growing internal R&D to the point where it's responsible for the majority of those candidates progressing.

"With the resources we have, that would be industry competitive," he added.

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Sanofi investing in gene therapy as R&D focus turns toward rare disease - BioPharma Dive

A more efficient approach to gene therapy that could lower costs and improve patient outcomes has recently been developed by a team from Scripps Research. This work, published on October 17 in the journal Blood, offers a potential alternative to the standard process of delivering gene therapy, which is expensive, time-consuming, and requires many steps to administer healthy genes to the patients stem cells.

If you can repair blood stem cells with a single gene delivery treatment, rather than multiple treatments over the course of many days, you can reduce the clinical time and expense, which removes some of the limitations of this type of approach, explained research leader Bruce Torbett, PhD, associate professor in the Department of Immunology and Microbiology.

The goal of gene therapy is to introduce a healthy version of a gene to a patients stem cells to replace a defective copy of this gene. This approach is designed to treat inherited conditions caused by genetic mutations, such as sickle cell anemia. Patients with sickle cell have a mutation in a gene that codes for a protein in blood cells, leading to misshaped cells that cause a myriad of clinical issues. The goal of gene therapy is to replace this mutated gene with a healthy copy to restore normal protein synthesis and eliminate the disease symptoms. This is often done by implanting the healthy gene into a modified virus, known as a viral vector, and having this virus use its innate ability to infiltrate host cells and inject this healthy gene into them.

Gene therapy treatments typically require the harvesting of a small population of hemopoietic stem cells, the cells that serve as precursors for all types of blood cells, from the patients blood. Viral vectors containing therapeutic genes are then introduced to these cells with the goal being for them to insert this genetic information into the stem cells.

The hemopoietic stem cells defend themselves from viral penetrance using interferon-induced transmembrane (IFITM) proteins that block the viral vectors. For this reason, many gene therapies require a large number of vectors and many attempts for success, which is an expensive process.

In their work, the Scripps team focused on caraphenol A, a molecular relative of resveratrol, a natural compound made by grapes and other plants present in wine. Resveratrol is known to have antioxidant and anti-inflammatory properties. Although caraphenol A shares these anti-inflammatory properties, it served a much different purpose in this work.

Observing the chemical properties of resveratrol and associated molecules such as caraphenol A, Torbett and colleagues wanted to investigate whether they could be used in gene therapy to improve the viral vectors ability to enter blood stem cells. Enhancing viral vector penetrance into host cells would be advantageous, being that the cells natural defense mechanisms against viral attacks present a challenge in gene therapy.

This is why gene therapy of hemopoietic stem cells has been hit-or-miss, explained Torbett. We saw a way to potentially make the treatment process significantly more efficient.

The researchers found that by adding caraphenol A to human hemopoietic stem cells with the viral vector present, the stem cells defense was weakened, and the viral penetrance increased. When these treated stem cells were implanted into mice in this study, they were observed to produce blood cells that contained the new genetic information.

In addition to saving costs, this approach also cuts down the time required for a patient to receive a gene therapy treatment. Reducing treatment time is not only convenient for the patient, but it lowers the chance that the stem cells lose their self-renewing properties as well. The more time the stem cells spend being outside of the body and being manipulated, the higher the likelihood of them losing their proliferative ability is.

Torbetts team is continuing to research how stem cells combat viral attacks, hoping to lower the cost of gene therapy while improving efficiency.

Excerpt from:
New Gene Therapy Approach Reduces Cost and Improves Efficiency - DocWire News

A made-in-Canada medical breakthrough that disappeared from the market because it wasn't profitable is being revived by the National Research Council of Canada (NRC).

It's the latest chapter in the saga of Glybera, the world's first approved gene therapy, which also became the world's most expensive drug after it was licensed toa Dutch company and priced at $1 million for a one-time dose.

Glybera treats arare and potentially deadly genetic disorder called lipoprotein lipase deficiency, or LPLD.Canada has the world's largest population of LPLD patients clustered in the Saguenay region of Quebec, where an ancestor with the genetic mutation settled several hundred years ago.

People with LPLD lack a critical enzyme that helps their bodies process the fat from food. There is currently no available treatment and no cure. Those with LPLDmust avoid most dietary fat to try to prevent painful and dangerous attacks of pancreatitis.

The decision to re-develop a Canadian version of Glybera is the result of a serendipitous series of events, beginning when the NRC'sdirector of research and development for translational bioscience happened to be watching CBC'sThe Nationallast November.

Dr. Danica Stanimirovic was in the process of selecting the first project for a new federally funded program aimed at bringing rare gene and cell therapies to Canadians at an affordable price. Thenshe sawCBC's feature report telling the story ofhow Glybera was pulled from the Europeanmarket after only one commercial sale. The drug was never offered for sale in Canada or the U.S.

"That really sparked some thinking," she said."We really have the abilityto advance that."

So she picked up the phone and called Dr. Michael Hayden in Vancouver.He's thescientist at the University of British Columbiaand the BC Children's Hospital whose team developed Glybera.Hayden said he was happy to get the call.

"I was thrilled because this represented a unique response to solve a big Canadian problem, particularly for families in Quebec.And I was just thrilled that we could do something as a national effort to achieve this."

The Glybera story started at UBC in the early 1990s, when Hayden and his teamdiscovered the first genetic mutations that caused LPLD. The researchers then developed a method to fix the malfunctioning gene and allow patients to live a nearly normal life.

After doing the preliminaryresearch, the Canadian discovery was licensed to a Dutch companycalled uniQure, which took Glybera through the rigorousclinical trialandapproval process.

When the treatment was approved by the European Medicines Agency in 2012, it made headlines as the world's firstgene therapy the first treatment that could repair a faulty gene.

When it went on sale in Europe in 2015,Glybera quickly made headlines again, this time as the "world's most expensive drug,"priced at $1 millionfor the one-time dose.

Dr. Sander van Deventer,uniQure's chief scientific officer, told CBC News last year that the price was a business calculation based on the price of other drugs that treat rare diseases. Many of those drugs cost more than $300,000 per patient per year.Because Glybera is a one-time treatment thatkeeps working for years, the $1-million price seemed reasonable, he said.

Less than twoyears later, the drug was pulled from the market after only one commercial sale. uniQure has no plans to revive the therapy.

Although Hayden discovered the gene mutation and developed the early phase of the treatment, he had no role in the commercialization of his discovery. And that meant he also had no control over the price.

"You don't determine the outcome, you don't determine its costs," he said."I'd say what went wrong is that it was very hard to be able to make sure that this got to patients at a reasonable cost."

Stanimirovic said the fact that Canada has such a large population of LPLD patients was an important factor in deciding to give Glybera a second chance.

"This gene mutation is very prevalent in Canada compared to other places in the world," she said. "For us, it was almost calling us to do something on the manufacturing side for this particular gene therapy."

LPLD is rare, affecting one or two out of every million people around the world. But inthe Saguenay region of Quebec, where the gene mutationhas been passed down through generations,the numbers are 30 times higher.Up to one in 50 people in some communities are carrying the gene mutation. Both parents must have the mutation for a child to inherit the disease.

The ultimate goal of gene therapy is to fix a genetic problem by giving the patient a new gene. Specially engineered viruses are used to deliver therepair gene to the patient's cells. The cost of manufacturing those virusesis often cited as one reason for the high price of therapies. The need to generate pharmaceutical shareholder profits is another factor.

"[Gene therapies] areusually targeted to very smallpatient populations," Stanimirovicsaid. "It's hard to make them in a typical pharma-driven model because it drives theprice of these therapies to astronomical levels."

At its facility in Montreal, theNRChas already developed expertise in producing viral vectors thatact as the delivery system for gene therapy. Because the scientistswill be re-engineeringGlyberausing new viral vectors,and improving the therapy, any remainingpatentswill not be an obstacle,Stanimirovicsaid.

The ultimate plan is to developpublic sector manufacturing capacity to create not just an affordable version of Glybera but other gene and cell therapies as well. The total federal funding for six projects including Glyberais estimated at about $80 million over seven years.

"Our goal is to create new partnership models that will create therapies that are more accessible and more affordable," said Stanimirovic. "We hope we can do that through public partnership or public/private partnerships. So the end goal is to really, through this project, develop Canadian capacity to take on subsequent gene therapies."

Hayden called the plan a "beautiful Canadian story."

"Now we have to translate this into something that will truly be effective forpatients in a limited time frame and I'm so excited to do this."

For patients suffering from LPLD, the wait is frustrating.

Felix Lapointe, a 10-year-old from Repentigny, Que.,was fiveweeks old when his mother learned the terrible news that her son had thepotentially deadly genetic disease.

Because there is no treatment available right now, he'smanaging the disease through a strict diet to reduce the risk of dangerous pancreatic attacks. He will have to wait another five years for the first clinical trials of the re-inventedGlybera.

"We'd like it to happen tomorrow morning," said Brenda Potter, Felix's mother. "Still, we're a little used to this. We'vebeen fighting for 10 years with doors closed. The possibility that something is comingis encouraging, but yes, it's long."

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Canadian breakthrough that became the world's most expensive drug, then vanished, gets second chance - CBC.ca

ROCKVILLE, Md., Oct. 18, 2019 /PRNewswire/ --American Gene Technologies (AGT) announced today the submission of an Investigational New Drug (IND)application to the U.S. Food and Drug Administration (FDA) for AGT's lead HIV program, AGT103-T, which is potentially a single-dose, lentiviral vector-based gene therapy developed for the purpose of eliminating HIV from people infected with the disease.

"Our aim is to treat HIV disease with an innovative cell and gene therapy that reconstitutes immunity to HIV and will control virus growth in the absence of antiretroviral drugs" said Chief Science Officer C. David Pauza, PhD. "Development of this complex product (AGT103-T) required our deep knowledge of both HIV disease and lentivirus vector technology; it is the first cell and gene immunotherapy addressing the most critical feature of HIV infection, which is the chronic absence of virus-specific CD4 T cells."

"We are excited to have reached this milestone of submitting our first IND application to the FDA for an HIV gene/cell therapy. This event brings us closer to reaching our mission to transform lives with genetic medicines. Based on our successful commercial-scale product manufacturing runs and features of the product observed in our laboratories, this therapy has a high potential to be effective. I feel confident that AGT103-Twill make an important difference in the lives of HIV infected persons," said Jeff Galvin, Founder and Chief Executive Officer of AGT."HIV is the first drug candidate to result from AGT's proprietary platform and model for creating gene and cell therapeutics more efficiently, predictably and reliably for clinical development. Our platform is also supporting robust efforts to cure the inherited disease phenylketonuria (PKU) and to introduce new therapies for cancer based on our proprietary methods for modifying tumor cells to activate the natural killing mechanisms of gamma delta T cells. Additionally, product development efforts behind this IND submission are supporting investigations into other chronic viral diseases that may be targeted in future human clinical trials.Success in HIV would allow AGT to accelerate these projects as well as quickly broaden our pipeline to dozens of infectious diseases, monogenic disorders, and cancers."

Upon acceptance by the FDA, this IND allows AGT to initiate a Phase 1 clinical trial that will investigate the safety of AGT103-T in humans, measure key biomarkers, and explore surrogate markers of efficacy. AGT expects to begin recruiting patients for the Phase 1 study in January.

About HIV

Today, approximately 37.9 million people worldwide and 1.1 million people in the United States are living with HIV/AIDS. The U.S. Government has estimated that 38,700 Americans were newly infected with HIV in 2016 and 1.7 million individuals globally were newly infected with HIV in 2018 (HIV.gov).

Since the late 1980s, antiretroviral drugs have restored quality of life to persons living with HIV and, in some cases, have even been used to prevent new infections. However, no approved treatments can cure HIV. This is an unmet medical need that AGT seeks to address.

About AGT 103-T

AGT103-Tis a genetically-modified cell product made from a person's own cells. AGT's approach is unique in that it focuses on repairing the key immune system damage caused by HIV. When HIV infection causes this specific damage, killing of T helper cells required for immunity to HIV, the infected person becomes unable to eliminate the virus and thus, becomes chronically infected. AGT's approach is designed to repair the T helper cell defect and provide durable virus control that is not compromised by HIV strains that vary in sequence or use alternate ways to enter and infect T cells. AGT's AGT103-T HIV therapeutic drug should work to remove infected cells from the body and decrease or eliminate the need for lifelong antiretroviral treatment.

"Previous cell and gene therapies for HIV provided very low doses of critical virus-specific CD4 T cells that are needed to repair the immune defect caused by HIV. AGT103-T becomes highly enriched in these specific cells during our proprietary 12-day manufacturing process. By providing high doses of virus-specific helper T cells, which are protected from HIV damage by a safe genetic modification, AGT's goal is to rebuild the capacity for normal, unhindered immune responses against HIV that may control the infection and protect against future virus exposures. We believe this product will be suitable for persons in different disease stages and with multiple types of HIV infection" explains Chief Science Officer C. David Pauza, PhD.

AGT has entered into a Research Collaboration Agreement with the National Institute of Allergy and Infectious Diseases (NIAID) and was able to demonstrate AGT103-T's mechanism of action. Read about AGT's NIAID Research Collaboration Agreement here.

About American Gene Technologies (AGT)

American Gene Technologies (AGT)is a gene and cell therapy company with a proprietary gene-delivery platform for rapid development of cell and gene therapies to cure infectious diseases, cancers, and inherited disorders. The Company's mission is to transform people's lives through genetic medicines that rid the body of disease. The Company expects to take its patented lead candidate for an HIV cure into the clinic in 2019. AGT has received seven patents for its unique immuno-oncology approach to stimulate gamma-delta () T cells to destroy a variety of solid tumors. The Company has developed a synthetic gene for treatingPhenylketonuria (PKU), a debilitating inherited disease. AGT's treatment for PKU has been granted Orphan Drug Designation by the Food and Drug Administration (FDA), and it is expected to reach the clinic in 2020.

AGT Contacts

C. Neil Lyons, Chief Financial Officer Phone: (301) 337-2269 Email: info@americangene.com

Sasha Whitaker, Digital Marketing and Communications Phone: (301) 337-2100 Email: swhitaker@americangene.com

SOURCE American Gene Technologies

http://www.americangene.com

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IND Application Submission To FDA For Phase 1 Trial Of Genetically Modified Autologous Cell Therapy For HIV Announced by American Gene Technologies -...

LONDON--(BUSINESS WIRE)--The report, interstitial cystitis drugs market 2019-2023, has been added to Technavio's 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.

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High prevalence of interstitial cystitis will drive the interstitial cystitis drugs market

Patients suffering from interstitial cystitis experience symptoms such as lower urinary tract issues and bladder pain. Women are highly affected by this condition. This condition is associated with various comorbidities such as endometriosis, fibromyalgia, and allergies. The increasing number of interstitial cystitis cases is driving the demand for interstitial cystitis drugs.

Advent of gene therapy An emerging trend in the interstitial cystitis drugs market

Most of the interstitial cystitis drugs and off-label drugs are small molecules and have failed to fully cure the disease. In addition, these drugs are expensive and cause many side-effects. This is encouraging companies to establish innovative treatment options such as regenerative therapy and gene therapy as these drugs cause lesser side-effects compared to the conventional off-label drugs already available in the market.

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Growth of Interstitial Cystitis Drugs Market to be impacted by the Advent of Gene Therapy | Technavio - Business Wire

Mount Sinai researchers have identified a targeted therapy for adolescent patients with neuroblastoma, a deadly pediatric nerve cancer, who would otherwise have no treatment options, according to a study published in October in Cancer Cell.

Neuroblastoma is one of the most common and aggressive pediatric nervous system tumors and generally has a poor prognosis, particularly when it advances in older children. Treatment success for the disease varies, but is exponentially less in adolescent patients, particularly because the disease lacks effective targeted therapies.

The Mount Sinai researchers found that neuroblastoma in older children and adolescents harboring deletions within a gene called ATRX may be responsive to a targeted therapy called tazemetostat. Tazemetostat disables an enzyme called EZH2 that inhibits genes that promote normal neuron development, in turn killing neuroblastoma cells. Neuroblastoma arises in immature nerve cells of the adrenal glands and portions of the spine during the development of the sympathetic nervous system, which controls the body's "flight or fight" response to stress. EZH2 inhibitors are already being tested in phase I and phase II clinical trials for other cancers, including lymphomas, sarcomas, and other solid tumors, with some favorable results.

"We hypothesized that mutant ATRX proteins contribute to aggressive neuroblastoma," said Emily Bernstein, PhD, Professor of Oncological Sciences at The Tisch Cancer Institute at the Icahn School of Medicine at Mount Sinai and senior corresponding author. "In this study, we aimed to decipher the underlying biology of these altered proteins in neuroblastoma, a tumor for which effective therapeutic strategies remain obscure, and to exploit identified dependencies."

Mount Sinai scientists continue to expand this research into the role of the mutant ATRX protein in the laboratory and hope to eventually open a clinical trial with collaborating institutions. Based on this research, they believe that EZH2 inhibitors could also be effective in other ATRX mutant cancers, such as pediatric glioblastoma multiforme and osteosarcoma.

Reference: Qadeer, et al. ATRX In-Frame Fusion Neuroblastoma Is Sensitive to EZH2 Inhibition via Modulation of Neuronal Gene Signatures. Cancer Cell,DOI: 10.1016/j.ccell.2019.09.002

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

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Targeted Therapy Could Help Children With Deadly Nerve Cancer - Technology Networks

TheGlobal Gene Therapy Marketresearch report is published to deliver a complete rundown of the global Gene Therapy industry structure, competition, segmentation, leading players, and industry environment. The report heavily emphasizes the evaluation of industry in terms of market size, share, demand, sales volume, and revenue reported by the industry. The report pursues historical and present market status to offer authentic estimations for the forecast period.

The global Gene Therapy market has been exhibiting robust performance over the last few years with a leaping CAGR. The market is expected to grow more intensely in the upcoming years as rising demand for the Gene Therapy, product awareness, technological advancements, rapid industrialization, raw material affluence, and increasing disposable incomes are boosting growth in the industry. The market is influencing its peers and parent markets and could impact on the international trading and economic structure.

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The report on the global Gene Therapy market additionally renders sequential elaboration on strategic moves performed by leading players which includes mergers, acquisitions, ventures, partnerships, amalgamations as well as product launches and brand promotions. Insightful analysis of strategies helps clients to gain a clear perception of their competitors potential moves and activities which make them alert while operating their business and building strategies.

The report digs deep into the operations of leading Gene Therapy manufacturers and companies. It analyzes various activities of players such as product innovation, research, recent developments, and technology adoptions, which aids them in delivering more effective product ranges in the industry. The report also sheds light on their financial assessment based on gross margin, sales volume, revenue, profitability, growth rate, and revenue share which helps to comprehend their positions, strengths, and weaknesses in the global Gene Therapy market.

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Study of crucial segments in the global Gene Therapy market:

The report also illuminates various segments of the global Gene Therapy market such as types, applications, regions, and end-users. Each segment is deeply evaluated in the report considering revenue, share, demand, production, and sales volume. The regional analysis of the market is also highlighted in the report which revolves around regions including North America, Europe, South America, Middle East & Africa, Asia Pacific, and vital regions from the rest of the world.

Furthermore, the report shifts its focus to current and forthcoming opportunities and challenges in the global Gene Therapy market. The report also helps clients convert opportunities into lucrative business gains and poses more critical challenges against competitors in the industry. It also hints at potential risks, obstacles, threats, and uncertainties that could harm the markets growth momentum in the near future.

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Gene Therapy Market Research Report by Downstream Applications, Competitive Analysis And Regional Forecast by 2024 - Online News Guru

The recent announcement that scientists discovered water on the planet K2-18b, 110 light years away, prompted a media swoon. News stories, including a piece written by me, billed it as the first detection of water on a potentially habitable planet outside our solar system.

The blowback from the astronomy community was swift. A chorus of critics stated on Twitter that, although K2-18b orbits its host star within a distance range astronomers call the habitable zone, the planet is most likely too hot and under too much pressure to support life.

The sentiments expressed in a Scientific American essay by Harvard University astronomer Laura Kreidberg were typical of many in the community. Kreidbergs piece suggested that news outlets were crying wolf and that scientists, press officers, and the press had all contributed to the misreporting of the story.

But in describing K2-18b as a potentially habitable planet, journalists were accurately reporting the views of the scientists who led one of the research studies. Those scientists repeatedly stated to reporters that the planet was potentially habitable and they continued to say so when the specific criticisms of their peers were put to them.

Also read:Science Needs More Space in Mainstream Media

The episode highlights a longstanding issue: How should we science journalists cover incremental research advances, especially when the underlying science is unsettled?

Recent history gives us numerous examples of how these so-called single-study stories can go wrong. Among the most notable was the coverage of a 1998 Lancet paper in which Andrew Wakefield and coauthors proposed that a combined measles, mumps, and rubella vaccine was linked to autism and bowel disease. Another high profile case involved papers published in Science in 2004 and 2005 in which a group led by Hwang Woo-Suk claimed to have cloned human embryonic stem cells for the first time.

Both research efforts were later discredited. In the case of the Wakefield paper, the selection of the sample group was biased, there were undisclosed vested interests, and the authors made several claims that did not stand up to scrutiny. The paper was retracted in 2010. Woo-Suks papers were simply fraudulent.

Id suggest that both of these cases are outliers; the media was duped as were the journals themselves by researchers who had an agenda or were simply dishonest about their results. Even when scientists act in good faith, however, things can go wrong. It is legitimate to ask those at the highest level of their profession to give their view on their own work, even if that view is speculative and at odds with what their rivals and colleagues have to say. But there are a few provisos.

First, a journalist should always reflect dissenting views. Sadly, much if not most coverage of the K2-18b story failed to do this, as is all too often the case with other single-study stories. (My own K2-18b coverage came under criticism for initially not including enough dissenting voices.) Many journalists believe that if research has been published in a peer-reviewed journal, it must be credible, and they make the mistake of reporting the research uncritically.

As I suggested in an article for BBC News, it is our job as science journalists to challenge what we are told. This is especially the case now that more of us report on controversial topics such as genetically modified crops, cloning, and climate change, which have complex political, as well as scientific, dimensions.

But even in the case of basic science discoveries in the realm, say, of anthropology or dark energy there is often plenty of debate. The standard narrative, people used to think x and now, because of this discovery they think y, is not the way science works, and it quite frankly makes for boring copy.

A second proviso is that all voices are not equal. The views of people who are not qualified in the particular area of research in question carry less weight than those of people who are. Like many serious journalism platforms, BBC News, where I work, has a strict policy of balance and impartiality. In the 1990s and 2000s, that policy led many of our programs to balance scientific voices warning of climate change or reassuring people about the safety of vaccines with the voices of people arguing the opposite.

The contrarian views would often come from pressure groups such as climate change deniers and anti-vaxxers or from scientists commenting on a specialty different than their own. The BBCs science and health correspondents argued strongly against the policy, which had been at the heart of the organisations journalism. In 2010, this led to a change in editorial guidelines, the new stance being that due weight should be given to the scientific consensus on any given subject before reflecting contrary views.

A third and final proviso is to beware of science journalisms oldest enemy: hype. Journalists should be wary both of researchers natural enthusiasm and of their sometimes-deliberate efforts to drum up publicity in order to secure research funding. In academia, cures for cancer, a new understanding of physics, and bottomless sources of clean energy are seemingly always between five and 10 years away.

The most recent example of a new technology being touted as an answer to all our problems is gene editing. In August 2017, Shoukhrat Mitalipov and colleagues reported in the journal Nature that they had successfully repaired a gene associated with a rare heart condition in a human embryo. The research has since been challenged, but in reporting the development, Britains bestselling daily newspaper, The Sun, loaded on the hyperbole, writing that the revolutionary work could help end 10,000 hereditary illnesses including cancer and that scientists say it could signal the end to inherited diseases.

Seventeen years earlier, British tabloids were saying the same thing about gene therapy. A story in The Daily Expresss Sunday Review in July 2000 asked: Could we be on the verge of the greatest medical advance ever seen, even greater than the defeat of smallpox or cholera the defeat of time itself? Similarly rosy predictions were made in 2007 about how microbes would provide an endless supply of biofuel.

Such credulous reporting may be becoming more prevalent, ironically, because of scientific institutions efforts to be helpful to science journalists. Organizations such as the UKs Science Media Center have sprouted up to coordinate the dissemination of press releases and other resources to journalists, often with the stated aim of countering misinformation. We science journalists get ideas, our editors get happy, uplifting stories, and the public gets a warm glow in its heart.

Also read:How Social Media Is Shaping Indian Science

But an information pipeline that runs uninterrupted from scientists to press officers to the news media puts us at risk of another kind of misinformation. A great science story counts for nothing if it gives readers a misleading impression or paints a cartoonish, one-dimensional picture of how science works. Such stories are their own brand of fake news. In writing them, we do neither the scientists, their press officers, nor our readers any favours.

When I started as a science journalist in the 1980s, single-study stories were the norm. Our job was to translate complex scientific information and artfully explain its significance to a non-scientific audience.

But many of us saw a responsibility to do more: to challenge, weigh, and assess the tablets of stone we were handed from omniscient researchers and to put them in a societal context. In other words, we became journalists, using our own skills and experience to add value and provide an important civic service.

My sense, though, is that because of staff and budget cuts, the extra time and effort needed to fulfil that role are seen by editors as luxuries, and so single-study stories are on the increase.

Perhaps in 20 years time scientists will have confirmed that K2-18b really is habitable. Until then, lets hope that science journalists will have the time and the self-confidence to listen to a range of views, and to give their own perspectives.

Pallab Ghosh is an award-winning science correspondent with BBC News. He works across television, radio, online, and digital platforms.

This article was originally published on Undark. Read the original article.

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How Should Science Journalists Cover Single-Study Stories? - The Wire

AMHERST, Mass. Peter Reinhart, director of the University of Massachusetts Amhersts Institute of Applied Life Sciences (IALS), has announced that six campus research teams have been named recipients of the first Manning/IALS Seed Grants. The awards will support next steps in their research such as proof-of-concept studies and business development, fundamental research into new products, technologies and services to benefit human health and wellbeing.

Earlier this year, alumnus Paul Manning and his wife, Diane, committed $1 million through their family foundation to establish theManning Innovation Program. It provides three years of support in advancing a robust and sustainable pipeline of applied and translational research projects from UMass Amherst.

The seed grants announced this week were awarded after a competitive process that narrowed 35 teams to six winners. Faculty researchers will not only receive seed funding of $100,000 each over three years, but also business training and mentorship from IALS, the College of Natural Sciences, the Berthiaume Center for Entrepreneurship and the Isenberg School of Management, among others.

The winning team leaders and their projects are:

The Manning Foundations gift provides an investment in UMass Amherst as a partner of choice in advancing and applying knowledge and innovation for the betterment of society.

Peter Reinhart, founding director of IALS, says, The Manning/IALS Innovation Program provides much-needed support allowing promising UMass Amherst research programs to move towards translational technology, prototypes, product candidates. This in turn will facilitate follow-on investments from venture organizations such as the Maroon Fund.

Paul Manning, a 1977 graduate of UMass Amherst, is an entrepreneur with 30 years of experience in the healthcare industry, who most recently founded PBM Capital Group in 2010. It is a healthcare-focused private investment group that looks for opportunities to use its entrepreneurial and operational experience to make high-growth pharmaceutical, molecular diagnostic, gene therapy, life science, health/wellness and consumer product investments.

Manning was also the anchor investor in Maroon Venture Partners, the first venture-capital fund at UMass Amherst. Created in 2017, the fund is a $6 million for-profit investment vehicle created to support alumni, faculty, and student businesses in their early stages.IALS was established in 2014, supported by a total investment of more than $150 million from the Massachusetts Life Science Center and the campus.

Originally posted here:
UMass Amherst Institute of Applied Life Sciences Announces Six Winners of the Inaugural Manning/IALS Prize - UMass News and Media Relations

The global gene therapy market rides on the back of technology. As consumer focus shifts from access to comfort, players in the market for gene therapy are looking at new opportunities to capitalize on the potential. This exclusive report from Transparency Market Research will take you through an extensive analysis of every aspect in the gene therapy market that is critical for defining your success strategy. It offers prudent information on markets under currents, trends that will open new doors, factors that will remain important, challenges that need to be overcome, prevailing competition in the market, and the geographical landscape.

Based on a tested and proven research methodology, our research analysts bring to you fact-checked information. Besides presenting the current market figures, our analysts provide you with accurate forecasts that can be the game-changer for your winning strategies for tomorrow. On the other hand, our reports also offer tailor-made insights. Further, our reports are packed with experts viewpoints which are transcribed from interviews conducted by our analysts.

For every market, information on leading players can be the difference between success and failure, be it a prominent brand or not. Our reports cover every significant players in the global gene therapy market providing information about the company profile, products, winning strategies and market revenues. Not just that, TMR also provides information on the competitive landscape, helping you understand what impacted in one company being the market leader and others not. It also explains on the companies imperatives that define their success in the global market for gene therapy.

To obtain all-inclusive information on forecast analysis of gene therapy Market, Request a PDF Brochure Here

Healthcare, unlike most industries, is typical of the region. Humans have multiple races and hence their genetic makeups are different. As a result, one condition has different impacts depending on the region. Therefore, information on how consumer requirements are different in regional landscape of the global gene therapy market is provided her in the report. Further, the economic capabilities of a country has a huge impact on healthcare infrastructure. TMRs report analysis the current economic scenario and also brings to you information on affordability during the coming years.

From market share to region-specific strategies, the report covers it all. At the same time, players in the gene therapy market who are looking to expand might want to assess the potential of a prospective region. Our reports can provide you with custom-made insights for specific regions in the global gene therapy market. The geographical analysis also covers regions-specific factors that could turn out to be hurdle for growth in the coming years.

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Gene Therapy Market Global Outlook and Overview 2018-2026 - Statsflash

The Gene synthesis market research report likewise conveys rundown of the main rivals and gives the crucial knowledge about the key elements affecting the In Vitro Diagnostic business. This market report additionally gives measurements on the present condition of the business as a profitable wellspring of direction and guidance for organizations and speculators keen on this market. It incorporates a far reaching examination of past, present just as well as forthcoming patterns in the market. The report uncovers significant elements of the market which can help the business experts in basic leadership.

Global Gene Synthesis Marketis set to rise from its initial estimated value of USD 3,542.49 million in 2018 to an estimated value of USD 19,295.56 million in 2026, registering a CAGR of 23.60% in the forecast period of 2019-2026. This rise in market value can be attributed to the rise in numbers of start-ups dealing with gene synthesis and growth in gene synthesis investments & funds.

Key data and information used while preparing this Gene synthesis report has been collected from the consistent sources that range from journals, websites, research papers, case studies, and magazines. Competitor strategies such as new product launches, expansions, agreements, joint ventures, partnerships, and acquisitions can be utilized well by the In Vitro Diagnostic industry to take better steps for selling goods and services. Gene synthesis market report is an analytical estimation of the key challenges in terms of sales, export/import, or revenue that an organization may have to face in the coming years.

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Global Gene Synthesis Market Report potential

Recent industry trends and developments

Market Penetration: Comprehensive information on the product portfolios of the top players in the Xyz market

Market share and size of all the foremost industry players

Complete report on Global Gene Synthesis Market Report 2019-2026..spread across 350 Pages, profiling Top companies and supports with tables and figures.

Few of the major competitors currently working in the gene synthesis market are Thermo Fisher Scientific, Inc., Genewiz, Eurofins Scientific, ATD Bio Ltd., OriGene Technologies, Inc., Bioneer Corporation, Atum, Integrated DNA Technologies, Inc., GenScript, Eurogentec, Twist Bioscience., BioCat GmbH, LGCBiosearch Technologies, Eton Bioscience, Inc., Quintara Biosciences, Bio Basic Inc., SBS Genetech Co., Ltd., Merck KGaA among others

WHY DATA BRIDGE MARKET RESEARCH?

Gene synthesis is the chemical in-vitro synthesis of double-stranded DNA molecules. It can develop mutated, recombinant, or entirely novel DNA sequences without any template DNA strand and synthesize oligos and RNA containing modified bases or chimeric DNA-RNA backbones along with DNA sequences. It has a crucial role in synthetic biology and biotechnology and also it is an important tool for various fields like vaccine development, molecular engineering, gene therapy, and heterologous gene expression in recombinant DNA technology. It can also be used in designing cancer enzymes and diagnosis of viral genomes for vaccine development.

Table Of Content:

Part 01: Executive Summary

Part 02: Scope Of The Report

Part 03: Global Market Landscape

Part 04: Global Market Sizing

Part 05: Global Market Segmentation By Product

Part 06: Five Forces Analysis

Part 07: Customer Landscape

Part 08: Geographic Landscape

Part 09: Decision Framework

Part 10: Drivers And Challenges

Part 11: Market Trends

Part 12: Vendor Landscape

Part 13: Vendor Analysis

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Market Segmentation:

By Products & Services (Consumables, Software, Services Market),Application(Diagnostics, Therapeutics, Research & Developmental Activities, Other Applications),Method(Solid Phase Synthesis, Chip based DNA synthesis, PCR based enzyme synthesis),End-User(Academic & Research Institutes Market, Diagnostic Laboratories Market, Biotech & Pharmaceutical Companies Market, Other Markets),Geography(North America, South America, Europe, Asia-Pacific, Middle East and Africa) Industry Trends & Forecast to 2026

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Competitive Analysis:Global Gene Synthesis Market

Global gene synthesis market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of gene synthesis market for global, Europe, North America, Asia Pacific, South America and Middle East & Africa.

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Gene Synthesis Market is Anticipated to Grow at a CAGR of 27.8% over the forecast period 2019 2026 With Major Key Players Like ATDBio Ltd, ATUM,...

Phenylketonuria (PKU) Market report covers the disease overview, definition, classification, symptoms, ethology, pathophysiology and diagnostic methods. The investigation depends on patient take-up by treatments, deals forecast of each medication by concentrate the explanations for the maximal utilization of new medicines. A relative investigation is likewise done based on part of the overall industry and size by evaluating the drugs take-up to extend the treatment situating in the market. It also covers the complete treatment methodologies and therapy areas under research and development

Phenylketonuria (PKU) Market Understanding and Treatment Algorithm

The report gives an exhaustive record of the all patient pool, diagnosed cases and potential patient pool qualified for the treatment. It additionally incorporates the clarification of changing patterns of the study of disease transmission in the wake of assessing various examinations, review reports and perspectives.

For producing such excellent Phenylketonuria (PKU) Treatment Market research report, principal attributes such as highest level of spirit, practical solutions, dedicated research and analysis, innovation, talent solutions, integrated approaches, most advanced technology and commitment plays a key role. Global Phenylketonuria (PKU) Treatment market report provides in-depth market data and forecast by analyzing key business trends and identifying potential growth avenues across the entire value chain. According to this Phenylketonuria (PKU) Treatment report, new highs will be made in the Phenylketonuria (PKU) Treatment market in 2018-2025. This report not only lends a hand for intelligent decision making but also better manages marketing of goods and services which leads to growth in the business.

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Phenylketonuria is a rare inherited genetic disorder that leads to the increase in levels of phenylalanine in blood. Phenylalanine is an amino acid which is comprised of proteins obtained from food and diet. Phenylalanine hydroxylase enzyme converts phenylalanine in to tyrosine amino acid in human body. The tyrosine is required to create neurotransmitters, such as epinephrine, norepinephrine and dopamine in human body. Phenylketonuria is caused due to a defect in the gene which helps to produce phenylalanine hydroxylase. Absence of this enzyme leads to a buildup of phenylalanine in the body and starts getting stored in the blood stream and consequently, damaging the brain. The patient of phenylketonuria can develop symptoms such as seizures, skin conditions, tremors or trembling and shaking, hyperactivity, stunted growth and more.

The incidence of phenylketonuria is approximately 1 in 10,000 in European populations, although it is less common in the African-American population, with an incidence of approximately 1 in 50,000. Phenyketonuria is rare in Finland and Japan, although its incidence may vary markedly between different regions.

Key Market Players:- DAIICHI SANKYO COMPANY, LIMITED, Ajinomoto Cambrooke, Inc., American Gene Technologies, Inc., Ultragenyx Pharmaceutical, Danone Nutricia , Reckitt Benckiser Group plc, Abbott, Promin Metabolics, Ajinomoto Cambrooke, Inc, Promin, Som innovation biotech, SL,Synthetic Biologics, Inc., Nestle, Codexis, BioMarin, Erytech Pharma are the key market players.

Market Drivers

Segmentation: Global Phenylketonuria (PKU) Treatment Market

By Type

By Drug Type

By Therapy Type

By Route of Administration

By End-Users

By Geography

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Key Developments in the Market:

Competitive Analysis: Global Phenylketonuria (PKU) Treatment Market

Global phenylketonuria (PKU) treatment market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of global phenylketonuria (PKU) treatment market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

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Phenylketonuria (PKU) Market 2019: Worldwide Industry Share, Size, Key Vendors, Growth Drivers, Regional, And Competitive Landscape Forecast To 2026 -...

Zacks Investment Research cut shares of Axovant Gene Therapies (NASDAQ:AXGT) from a buy rating to a hold rating in a research report sent to investors on Tuesday morning, Zacks.com reports.

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

A number of other equities research analysts also recently weighed in on AXGT. Svb Leerink assumed coverage on Axovant Gene Therapies in a research report on Friday, June 21st. They set an outperform rating and a $18.00 price target on the stock. Leerink Swann assumed coverage on Axovant Gene Therapies in a research report on Friday, June 21st. They set an outperform rating and a $5.79 price target on the stock. Cowen restated a hold rating on shares of Axovant Gene Therapies in a research report on Tuesday, July 9th. ValuEngine upgraded Axovant Gene Therapies from a sell rating to a hold rating in a research report on Thursday, August 1st. Finally, Robert W. Baird upgraded Axovant Gene Therapies from a neutral rating to an outperform rating and lowered their price target for the company from $16.00 to $13.00 in a research report on Monday, August 12th. Three research analysts have rated the stock with a hold rating and eight have issued a buy rating to the stock. The stock has a consensus rating of Buy and an average price target of $26.91.

Axovant Gene Therapies (NASDAQ:AXGT) last issued its quarterly earnings data on Friday, August 9th. The company reported ($1.23) earnings per share (EPS) for the quarter, topping the Zacks consensus estimate of ($1.34) by $0.11. Research analysts expect that Axovant Gene Therapies will post -4.25 EPS for the current year.

Several large investors have recently made changes to their positions in AXGT. Sphera Funds Management LTD. bought a new position in shares of Axovant Gene Therapies during the first quarter valued at about $6,794,000. BlackRock Inc. bought a new position in shares of Axovant Gene Therapies during the second quarter valued at about $1,482,000. Marshall Wace LLP bought a new position in shares of Axovant Gene Therapies during the first quarter valued at about $272,000. Jane Street Group LLC grew its stake in shares of Axovant Gene Therapies by 28.8% during the second quarter. Jane Street Group LLC now owns 46,455 shares of the companys stock valued at $289,000 after buying an additional 10,375 shares during the last quarter. Finally, Tower Research Capital LLC TRC grew its stake in shares of Axovant Gene Therapies by 955.3% during the second quarter. Tower Research Capital LLC TRC now owns 4,221 shares of the companys stock valued at $27,000 after buying an additional 3,821 shares during the last quarter. 13.48% of the stock is currently owned by hedge funds and other institutional investors.

About Axovant Gene Therapies

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

Further Reading: Diversification Important in Investing

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Axovant Gene Therapies (NASDAQ:AXGT) Cut to Hold at Zacks Investment Research - Mitchell Messenger

Eli Lilly is closing down its U.K.-based Erl Wood neuroscience center in Surrey, leading to cuts and relocations.

According to a local report from the Basingstoke Gazette, and confimed to FierceBiotech, the research house will be closed by the end of 2020, seeing around 80 redundancies from the neuroscience unit.

In total, around 270 employees will be hit, with two-thirds of staff moving over to a new location within the local area while neuroscience research will move over to its home in the U.S., according to the company, which spoke to the Gazette.

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The company added that this comes as part of a global review of research operations and off the back of years of on and off cuts from the Big Pharma.

Lilly told FierceBiotech in a statement that over time, Lilly has made a number of strategic choices to consolidate research operations around the world into global hubs. Overall, this has resulted in there being fewer than 45 neuroscience researchers, supported by approximately 35 chemistry researchers, working at the companys UK research site today.

The company added: Today, Lilly is announcing a proposal to consolidate laboratory-based UK research efforts to other global centres to further enable transformation which aims to deliver medicines from the lab to patients faster. This would include: Relocating neuroscience research to Lillys expanding site in Cambridge, Massachusetts, where Lilly maintains a hub specialising in the application of novel technologies including siRNA and gene therapy; Relocating a small number of roles to Lillys research centre in Indianapolis; Rebalancing and consolidating chemistry capabilities around the world."

Lilly said it would retain a small number of bioinformatics and neuroscience roles in the UK to focus on expanding external R&D collaboration with the UK and EU science community, and continue to undertake research collaborations and partnership activity in the U.K. with academia, the NHS and biotechs.

Under the plans, non-laboratory based employees would move to a new site within the local area. Lilly will now launch a formal consultation with employees and will be focused on ensuringevery employee is treated with dignity and respect throughout this period of change.

There are no changes being announced to Lillys commercial infrastructure, based in Basingstoke, and the company will retain core clinical and regulatory functions in the U.K.

It also pointed out that this was not down to Brexit.

Tim Garnett, chief medical officer at Lilly, said: I know that this proposal will be difficult news for many working for Lilly in the UK. Our dedicated teams have delivered world-class research from Erl Wood for the last 50 years. They are some of the most talented people in the industry and this proposal is not a reflection on their hard work or skill.

He added: Our focus now is on supporting our people and treating them with the dignity and respect they deserve. We will now enter a formal consultation with all the employees potentially affected.

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Eli Lilly to shutter neuroscience R&D center next year - FierceBiotech

CAMBRIDGE, Mass. and RESEARCH TRIANGLE PARK, N.C., Oct. 15, 2019 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, and Asklepios BioPharmaceutical, Inc.(AskBio), a fully integrated adeno-associated virus (AAV) gene therapy company, today announced the companies have entered a strategic research collaboration to explore in vivo delivery of genome editing medicines to treat neurological diseases. This collaboration brings together AskBios leading capsid development, clinical stage AAV vector delivery system, and manufacturing expertise with Editas Medicines leading genome editing technologies to potentially develop novel medicines for patients with high unmet need.

We are excited to collaborate with AskBio, a team with unparalleled experience in AAV technology and clinical-stage manufacturing. We believe that together we can deliver innovative genome editing medicines to the nervous system and rapidly advance medicines to treat neurological diseases and help patients in great need, said Charles Albright, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine.

The team at Editas Medicine has one of the most innovative technology platforms dedicated to finding solutions to severe diseases where there are few or no treatment options a mission consistent with AskBios, said Jude Samulski, Ph.D., Co-Founder, AskBio. With this shared goal in mind, we will combine our technologies to create an innovative approach to treating neurological diseases.

About Editas MedicineAs a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

About AskBioAsklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical stage gene therapy platform company dedicated to improving the lives of children and adults with rare genetic disorders. AskBios gene therapy platform includes an industry-leading proprietary cell line manufacturing process known as Pro10 and an extensive AAV capsid library. The company has generated hundreds of proprietary third generation gene vectors, several of which have entered clinical testing. AskBio maintains a portfolio of clinical programs across a range of indications, including Pompe, Limb Girdle Muscular Dystrophy, Cystic Fibrosis, Myotonic Muscular Dystrophy, Huntingtons, Hemophilia (Chatham Therapeutic/Takeda) and Duchenne Muscular Dystrophy (Bamboo Therapeutics/Pfizer). For more information, visit http://www.askbio.com.

Editas Medicine Forward-Looking Statements This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, target, should, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Editas Medicine may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of Editas Medicines product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for Editas Medicines foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption Risk Factors included in Editas Medicines most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission, and in other filings that Editas Medicine may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and Editas Medicine expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise.

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Editas Medicine and AskBio Enter Strategic Research Collaboration to Explore In Vivo Delivery of Genome Editing Medicines to Treat Neurological...

LONDON, Oct. 16, 2019 /PRNewswire/ -- The report "Protein Expression Global Market Report 2019" published By TheBusinessResearchCompany.com is about Protein Expression Market which is spread across 150+ pages and provides latest industry data, market trends, allowing you to identify the products and key players.

The protein expression market (https://www.thebusinessresearchcompany.com/report/protein-expression-global-market-report) in the U.S. is governed by the Food and Drug Administration (FDA) that lays down a series of guidelines for the manufacturers and retailers of this industry. Within the FDA, the Center for Drug Evaluation and Research (CDER) regulates biological products under FDA 101 which includes gene therapy products and vaccines. These regulations ensure quality, safety and efficacy of biological therapeutics products, and speed up innovations that make these products safer and effective. In May 2014, the US's FDA announced a fast-track initiative to review its drugs and biologics policy to speed the availability of therapies to patients with serious conditions, and orphan drugs for rare diseases, while preserving the safety and efficacy standards. In 2016, the FDA also removed a rule (Section 610.21 of the FDA code) which specified minimal potency limits for certain antibodies and antigens. The European Medicines Agency has also introduced policies which include a provision to waive the scientific advice fee, which encourage more academic groups and small companies to propose candidates for biologics.

Stringent Regulations Will Hinder The Global Protein Expressions Market Growth

Government regulations related to protein therapeutics and production of biologics may hinder the protein expression market growth. Government regulations on biologics to undergo rigorous preclinical and clinical trials prior to regulatory approval, and time-consuming process for approval of biologics with regards to health and the safety of any individual are restraining the market growth. Marketing and distribution of biologics including insulin, hormones, therapeutic antibodies, and vaccines depends upon the successful completion of clinical trials, which is a long, expensive, and uncertain process. According to the FDA, for an approval of a new biologic, under the regulations (21 CFR 314.81(b)(2)(vii) and 601.70, a clinical trial approval usually takes 10-12 months where firms are required to submit a report annually on the status of clinical safety, clinical efficiency, clinical pharmacology, and nonclinical toxicology study.

Protein expression is a process in which proteins are synthesized, modified, regulated and controlled in living organisms according to the host cell. Protein expression includes yeast expression, insect expression, and bacterial expression, algal expression and mammalian cell expression. The global protein expression market was valued at about $1.74 billion in 2018 and is expected to grow to $2.63 billion at an annual growth rate of nearly 11% through 2022. Request for Sample Copy @ https://www.thebusinessresearchcompany.com/sample.aspx?id=2493&type=smp

Growing Demand For Biologics Will Drive The Protein Expression Market

Increase in demand for biologics to counter various genetic disorders and chronic diseases is a major factor driving the research and sales of protein expression. Biologics is a medicine produced from living organisms or contains components of living organisms such as protein, tissue, genes, allergens, cells, blood components, blood, and vaccines. The increasing use of biologics (therapeutic protein and others) to cure chronic diseases such as cancer, cardiovascular conditions and genetic disorders, is increasing the demand for protein expression devices and equipment. According to the World Health Organization, chronic disease prevalence is expected to rise by 57% globally, by the year 2020. According to an article published by Chemistry World in the year 2018, analysts expect the biologics market to hold a market share of more than a quarter of the entire pharmaceutical industry by 2020. The global biologics market is expected to grow at 9.9% during 2018-2024.

Microfluidics Technology- A Trend In The Protein Expression Market

Companies in the protein expression industry are increasingly adopting Microfluidics technology to enhance protein expression tests in order to reduce the time, cost, labor, and increase the accuracy and performance. The microfluidics technology effectively analyzes biological samples than the traditional (macroscale) instruments. Microfluidics technology is used to measure the expression of proteins on cells and optimizes the output to generate results regarding protein expression. Therapeutics-on-a-chip (TOC) uses microfluidic platform and is able to synthesize proteins in a point-of-care setting to reduce cost associated with storage and transportation of therapeutic proteins. For instance, companies such as MissionBio, NanoCellect Biomedical, RainDance Technologies and Sphere fluidics have implemented this technology in protein expression test. Place a Direct Purchase Order of Complete Report @ https://www.thebusinessresearchcompany.com/purchaseoptions.aspx?id=2493

Browse Related Reports:

Therapeutic Proteins Market Report @ https://www.thebusinessresearchcompany.com/report/therapeutic-proteins-global-market-report

Biologics Market Report @ https://www.thebusinessresearchcompany.com/report/biologics-market

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Protein Expression Market Value is Expected to Reach $2.63 Billion by 2022 - PRNewswire

CAMBRIDGE, Mass., Oct. 16, 2019 (GLOBE NEWSWIRE) -- LogicBio Therapeutics Inc. (Nasdaq:LOGC), a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients, today announced upcoming presentations at the European Society of Gene and Cell Therapy (ESGCT) 27th Annual Congress, held in Barcelona, Spain, October 22-25, 2019.

We are thrilled to be presenting positive data on our Next Generation Capsid Development Program on the anniversary of our collaboration with Childrens Medical Research Institute of Australia, a leader in gene therapy, childhood cancer, embryology and neurological diseases. The goal of the collaboration is to develop novel, synthetic adeno-associated virus (AAV) capsids which are highly tropic for human tissues and optimized for manufacturing. These data give us further confidence that we can improve the performance of current AAV vectors, expanding our pipeline and strengthening our GeneRide platform, said Fred Chereau, CEO of LogicBio. Further, we are pleased to present additional preclinical data further supporting the durability of expression, compared to canonical gene therapy, in one of our GeneRide platform programs and to have been invited to speak on AAV manufacturing.

Panel PresentationTitle: AAV manufacturing: critical parameters influencing vector quality attributesPresenter: Matthias Hebben, Ph.D., VP, Technology Development, LogicBio Therapeutics (INV36)Session: 1d ATMP manufacturingSession date/time: October 23, 2019, 8:30-10:30 a.m. CEST

Poster PresentationsTitle: AAV development program: towards next generation of livertropic AAV variants (P025)Session date/time: October 23rd, 2019, 1:00-3:00 p.m. CEST

Title: Durability of factor IX expression in mice treated neonatally with a nuclease-free, promoterless, AAV-based gene therapy, GeneRide (P423)Session date/time: October 23rd, 2019, 1:00-3:00 p.m. CEST

Additional information on the meeting can be found on the ESGCT website: https://www.esgct.eu/home.aspx

About LogicBio TherapeuticsLogicBio Therapeutics is a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients with significant unmet medical needs using GeneRide, its proprietary technology platform. GeneRide enables the site-specific integration of a therapeutic transgene in a nuclease-free and promoterless approach by relying on the native process of homologous recombination to drive potential lifelong expression. Headquartered in Cambridge, Mass., LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families.

For more information, please visit http://www.logicbio.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the federal securities laws. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. These risks are discussed in the Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on April 1, 2019 with the SEC, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

Contacts

Brian LuqueAssociate Director, Investor Relationsbluque@logicbio.com951-206-1200

Stephanie SimonTen Bridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

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LogicBio Therapeutics to Present New Data on Next Generation Capsid Development Program and GeneRide Platform Program at the European Society of Gene...

The Company plans to use this funding to progress development of its SVac platform towards a first in man clinical study to commence in two to three years from now.

In addition to raising new funds, Amarna has recruited a new Supervisory Board to help underpin this new clinical phase of its growth and development. Thomas Eldered has been appointed as Supervisory Board Chairman. Bernhard Kirschbaum, Maarten de Chateau, Ted Fjllman, Pim Berger and Guillaume Jetten have also joined the Supervisory Board (see biographies at the end of the release).

Ben van Leent, CEO of Amarna said: We are very happy to have attracted such strong investors. This significant new funding allows Amarna to accelerate the development of SVac and our lead product AMA001, and to help us achieve our ultimate goal: to become a leading global gene therapy player.We would like to extend a warm welcome to all of the members of our new Supervisory Board, led by the outstanding healthcare and biotech pioneer Thomas Eldered. They bring many years of in-depth life science knowledge and entrepreneurship to Amarna.

Thomas Eldered, new Chairman of Amarnas Supervisory Board, commented: Im very much looking forward to working at Amarna. Its viral gene delivery vector platform has the potential to make major medical breakthroughs possible, so that patients can be actually cured of significant diseases for which, to date, effective treatment have not become available. Together with my highly qualified and experienced colleagues in our new Supervisory Board, Im fully committed to help progress Amarna into the next important clinical stages of development of its groundbreaking technology.

SV40 Vectors are non-immunogenic in humans Viral gene delivery vectors that are currently used for in vivo gene therapy are ineffective because the particles are instable upon injection (in the case of lentiviral vectors) or because the particles are immunogenic in humans (in the case of AAV vectors). Gene delivery vectors derived from the macaque polyomavirus Simian Virus 40 (SV40) are an attractive alternative to lentiviral and AAV vectors for clinical gene therapy. Humans can be considered nave to SV40 since the virus only replicates in macaques, where it causes symptomless infections. Replication-defective SV40 vectors are non-immunogenic in humans and moreover, have the capacity to induce immune tolerance to the transgene products. SV40 vectors therefore hold a great potential for clinical applications treating genetic disorders, cancer, allergies and degenerative/inflammatory conditions such as neurodegenerative and psychiatric diseases, atherosclerotic cardiovascular disease, diabetes mellitus, arthritis, chronic obstructive pulmonary disease and many more.

Amarnas SVac platform: the benefits Amarna has genetically engineered the SV40 genome used for the production of vector particles and in parallel generated a novel Vero-based packaging cell line named SuperVero that produces similar numbers of vector particles to the currently used packaging cell lines but without contaminating wild type SV40 particles. Since SVac is safe, highly efficient, non-immunogenic in humans and vector particles can be cost effectively produced in SuperVero cells, Amarnas vector platform paves the way to clinically evaluate a whole new generation of SVac-based therapeutics for todays major diseases.

Notes to Editors

About Amarna Therapeutics Amarna Therapeutics is a privately held Biotech company founded in 2008 with its head office in Leiden, The Netherlands, and with a research facility in Seville, Spain. The company has developed the SVac viral gene delivery vector platform, the key to the success of gene replacement therapy and therapeutic reverse vaccines. SVac is safe, highly efficient and non-immunogenic in humans, creating opportunities to treat diseases with high unmet clinical need including genetic disorders, degenerative/inflammatory/autoimmune diseases, infectious diseases and cancer.

About Flerie Invest AB Flerie Invest AB is a Venture Capital firm located in Stockholm Sweden focused on life science investments globally.

About the Netherlands Enterprise Agency (RVO.nl) The Netherlands Enterprise Agency operates under the auspices of the Dutch Ministry of Economic Affairs and Climate Policy and stimulates entrepreneurs, NGOs, knowledge institutions and organizations. The agency aims to improve opportunities, strengthen positions and help realize international ambitions with funding, networking, know-how and compliance with laws and regulations.

New Supervisory Board member biographies

Thomas Eldered received a graduate degree from Linkping Institute of Technology and founded Swedish listed pharmaceutical contract development and manufacturing company Recipharm AB. Presently, Thomas is Chief Executive Officer at Recipharm AB and at the Board of several companies including Kahr Medical Ltd, Provell Pharmaceuticals LLC, and Sixera Pharma AB.

Dr. Bernhard Kirschbaum studied biochemistry and physiological chemistry at the University of Konstanz and received his PhD in 1989. He moved on to work at the Rockefeller University and the Pasteur Institute before making a career in the pharmaceutical industry where he covered a broad range of technological and disease areas. In his most recent position, Bernhard served as head of Global Research and Early Development at Merck Serono where he was a member of the board. Currently he holds further Supervisory Board and SAB positions at Omeicos, Redx, FutuRx, Enlivex, KAHR and BioMedx.

Dr. Maarten de Chteau is Chief Executive Officer at Swedish biotech Sixera Pharma and Buzzard Pharmaceuticals, focusing on the development of drugs that restore the balance in diseased skin and cancer respectively. Maarten has extensive experience in pharmaceutical clinical development at Sanofi and Swedish Orphan Biovitrum (Sobi). Prior to that he worked as a financial analyst at two investment banks and conducted basic research at Lund University and Harvard Medical School. Maarten was the founder and CEO of Cormorant Pharmaceuticals, leading the development of an immune-oncology project. Cormorant was acquired by Bristol-Myers Squibb in 2016.

Dr. Ted Fjllman is the CEO of Prokarium, a London-based biotech, focusing on targeted oral vaccines and microbial immunotherapy. Ted has worked for clinical research and strategy consulting firms and built two early-stage companies. Ted holds a PhD from the University of Guelph, Canada, and has been recognized as a leader in biotechnology under the Sloan Foundation funded Synthetic Biology Leadership Accelerator Program (LEAP). He is a member of the international Bacterial Vaccines Network (BactiVac) management board, as well as an active member in the UK Bio-Industry Association (BIA).

Pim Berger is CEO and co-founder of Dutch IT company Schuberg Philis and one of Amarnas pioneering informal investors for over 10 years. He was managing director Netherlands of SiteSmith Inc., a US-based Internet infrastructure management services company. In 2000 SiteSmith Inc. was acquired by Metromedia Fiber Network Inc. (MFN) in a transaction valued at approximately $1.4 billion. Pim became Senior Vice President of MFNs European Region. In a management buyout by Pim and two partners in 2003 the Benelux subsidiary of MFN Inc. became Schuberg Philis.

Before this, Pim held several senior executive positions at Dutch IT companies Pink Elephant and PinkRoccade (now part of KPN). He holds a masters degree in computer engineering and economics from the Hogeschool IJsselland.

Guillaume Jetten is the CFO of Netherlands based MercachemSyncom, a leading European mid-sized contract research organization. Guillaume has been working in the healthcare industry for most of his career. He held positions at Merck, Sharpe & Dohme, and served as Chief Financial Officer (CFO) of Wolters Kluwer Health. He also worked as CFO for Galapagos between 2009 and 2014. Guillaume holds a masters degree in economics from the University of Maastricht and is a Certified Registered Accountant.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191016005612/en/

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Amarna Therapeutics Raises 10 million to Progress lead Development Candidate into Clinical Trials and Appoints a new Supervisory Board - BioSpace

DUBLIN--(BUSINESS WIRE)--The "Global Longevity and Anti-Senescence Therapy Market" report has been added to ResearchAndMarkets.com's offering.

Global longevity and anti-senescence market will witness rapid growth over the forecast period (2018-2023) owing to an increasing emphasis on Stem Cell Research and increasing demand for cell-based assays in research and development.

An increasing geriatric population across the globe and rising awareness of antiaging products among generation Y and later generations are the major factors expected to promote the growth of global longevity and anti-senescence market. Factors such as a surging level of disposable income and increasing advancements in anti-senescence technologies are also providing traction to the global longevity and anti-senescence market growth over the forecast period (2018-2023).

Senolytics, placenta stem cells and blood transfusions are some of the hot technologies picking up pace in the longevity and anti-anti-senescence market. Companies and start-ups across the globe such as Unity Biotechnology, Human Longevity Inc., Calico Life Sciences, Acorda Therapeutics, etc. are working extensively in this field for the extension of human longevity by focusing on the study of genomics, microbiome, bioinformatics, and stem cell therapies, etc. These factors are poised to drive market growth over the forecast period.

The report provides analysis based on each market segment including therapies and application. The therapies segment is further sub-segmented into Senolytic drug therapy, Gene therapy, Immunotherapy, and Others. Senolytic drug therapy held the largest market revenue share in 2017. The fastest growth of the gene therapy segment is due to the Large investments in genomics.

Report Scope

The scope of this report is broad and covers various therapies currently under trials in the global longevity and anti-senescence therapy market. The market estimation has been performed with consideration for revenue generation in the forecast years 2018-2023 after the expected availability of products in the market by 2023.

The global longevity and anti-senescence therapy market has been segmented by the following therapies: Senolytic drug therapy, Gene therapy, Immunotherapy and Other therapies which includes stem cell-based therapies, etc.

Revenue forecasts from 2028 to 2023 are given for each therapy and application, with estimated values derived from the expected revenue generation in the first year of launch.

The report also includes a discussion of the major players performing research or the potential players across each regional longevity and anti-senescence therapy market. Further, it explains the major drivers and regional dynamics of the global longevity and anti-senescence therapy market and current trends within the industry.

The report concludes with a special focus on the vendor landscape and includes detailed profiles of the major vendors and potential entrants in the global longevity and anti-senescence therapy market.

The report includes:

Key Topics Covered

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Market Overview

Chapter 4 Global Longevity and Anti-senescence Market by Therapy

Chapter 5 Global Longevity and Anti-senescence Market by Application

Chapter 6 Global Longevity and Anti-senescence Market by Region

Chapter 7 Industry Structure in Longevity and Anti-senescence Market

Chapter 8 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/zy7jt

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Global Longevity & Anti-Senescence Therapy Market Review 2017-2018 and Forecast to 2023 - ResearchAndMarkets.com - Business Wire

A special advisor to the Charcot-Marie-Tooth Associations (CMTA) board of directors says he will match donations dollar-for-dollar up to $500,000 to support the organizations research strategy to fight the disease he has CMT2.

Bruce Chizen was diagnosed in 2016, after decades of experiencing symptoms. However, his physician discouraged him from undergoing genetic testing because he couldnt do anything about the results.

Unsatisfied, Chizen forged ahead with his quest for more disease information. The first place he turned to was CMTAs website. Since then, hes been grateful for the organizations help and encouraged by its efforts to find a cure that hes vowed to support it however he can.

His initial CMTA donation led to breakfast with his Silicon Valley neighbor and CMTA board chair, Gilles Bouchard. The meeting resulted in him taking on his current role.

A Brooklyn, New York native, Chizen graduated from Brooklyn College where he studied health sciences and planned to become a teacher. However, he learned that he had a head for business, and started out as a Mattel Toys salesman, and then became a Microsoft sales manager in the mid-80s. That was followed by stints at Apple and then Adobe, where he worked for 14 years, seven as CEO.

With his extensive business and nonprofit experience, Chizen appreciates how CMTA is run, calling the organization extremely focused and effective.

Ive seen a lot of waste, the semi-retiree states.

Chizen considers education and community services an essential part of the groups mission, and wants to fortify them. Hes also encouraged by the success of the organizations Strategy to Accelerate Research (STAR), which was created in 2008 with five elements: Assays (tests), animal models, stem cells, partners, and clinical trials. The goal is to find treatments to slow, stop, or reverse CMT progression.

The path to a cure is possible and probable, Chizen said.

Visit this site to double your donations to the organization. Contributions will go to implementing CMTAs new Type 2 strategy, a multi-pronged approach that includes projects on gene therapy, small molecule therapies, axon degeneration pathways, mitofusin activity regulation, and human stem cell studies. The strategy is outlined in the fall issue of The CMTA Report, which focuses exclusively on CMT2 and includes patient profiles. Its available to download here.

CMT2 is caused by genetic defects that disrupt the structure and function of axons of the peripheral nerves. Its less common than CMT1 and accounts for about one-third of all dominant CMT cases.

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours dElegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled Belle of the Concours.

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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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Research Gets Boost With CMTA's Matching Donation Challenge - Charcot-Marie-Tooth News

ANI | Updated: Oct 14, 2019 14:31 IST

Washington D.C [USA], Oct 14 (ANI): Recently developed gene therapy seems to be a promising alternative for treating one of the most common causes of blindness, wet age-related macular degeneration (AMD), claims a study. Data presented at the 123rd Annual Meeting of the American Academy of Ophthalmology showed that six patients with wet age-related macular degeneration (AMD) went at least six months without the need for continued injections to control a disease that typically requires treatment every four to six weeks.Researchers said the hope is that gene therapy will free patients from nearly monthly eye injections by offering a potential "one-and-done" treatment. It's not just about convenience; a more consistent treatment may also help people keep more of their vision."This is potentially paradigm-shifting," said lead researcher, Szilard Kiss, M.D., director of Clinical Research and chief of the Retina Service in the Department of Ophthalmology at Weill Cornell Medical College in New York City."It's the next evolutionary leap in treating AMD. When you think about what science fiction is and what science reality is; gene therapy for AMD is becoming a clinical reality," added Dr Kiss.AMD is a degenerative eye disease that happens when part of the retina is damaged. The damage happens when new, weak blood vessels form behind the retina at the back of the eye. These abnormal vessels leak, causing scarring and killing off the cells that allow us to see.One main reason why is that patients are undertreated. This is because most people with AMD must go to the ophthalmologist's office every four to eight weeks for an injection directly into their eye (oftentimes in both eyes).This can be a difficult schedule to maintain for many elderly patients struggling with other maladies and reliant on others to get them to their ophthalmologist visits. It's also unsustainable for the health care system.Last year alone, ophthalmologists performed more than 8 million anti-VEGF injections in the United States.Researchers have been searching for a better alternative to monthly injections almost from the moment anti-VEGFs were introduced. Gene therapy is emerging as one of the more promising alternatives to long-term anti-VEGF treatment.The goal of Dr Kiss' research is to develop a gene therapy that allows the eye to make its own anti-VEGF medicine. The ideal gene therapy would be administered not through a surgical procedure in an operating room, but through an injection into the eye that can be done in the doctor's office, just like routine anti-VEGF treatment is done today.To do this, Dr Kiss and colleagues have developed a next-generation vector that can insert into the cells of the eye, the genetic material that makes a molecule similar to a widely used anti-VEGF medicine called aflibercept. Once inside the cells, the DNA sequence begins making the aflibercept protein. (ANI)

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Gene therapy effective for treating wet age-related macular degeneration: Study - ANI News