Archive for the ‘Gene Therapy Research’ Category

The Report published on DataIntelo.com about Cancer Gene Therapy Market is spread across several pages and provides newest industry data, market future trends, allowing you to identify the products and end users driving revenue growth and profitability. The industry report lists and studies the leading competitors, also provides the insights with strategic industry analysis of the key factors influencing the market dynamics.

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Cancer Gene Therapy Market Research Report is a professional and in-depth study on the current state which focuses on the major drivers and restraints for the key players. Cancer Gene Therapy Industry research report provides granular analysis of the market share, segmentation, revenue forecasts and geographic regions of the market.

Key manufacturers are included based on company profile, sales data and product specifications etc. AdaptimmuneBluebird bioCelgeneShanghai Sunway BiotechShenzhen SiBiono GeneTechSynerGene TherapeuticsAltor BioScienceAmgenArgenxBioCancellGlaxoSmithKlineMerckOncoGenex PharmaceuticalsTransgene

The report begins with the overview of the Cancer Gene Therapy Market and offers throughout development. It presents a comprehensive analysis of all the regional and major player segments that gives closer insights upon present market conditions and future market opportunities along with drivers, trending segments, consumer behaviour, pricing factors and market performance and estimation throughout the forecast period.

The report also covers geographical markets and key players that have adopted significant strategies for business developments. The data within the report is displayed in a statistical format to offer a better understanding upon the dynamics. The report compiles exhaustive information acquired through proven research methodologies and from dedicated sources across several industries.

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The report segments the Global Cancer Gene Therapy Market as In market segmentation by types of Cancer Gene Therapy, the report covers Oncolytic VirotherapyGene TransferGene-Induced Immunotherapy

In market segmentation by applications of the Cancer Gene Therapy, the report covers the following uses HospitalsDiagnostics CentersResearch Institutes

Geographically, this report studies the top producers and consumers in these key regions North America U.S., Canada, MexicoEurope U.K., France, Italy, Germany, Russia, Spain etc.Asia-Pacific China, Japan, India, Southeast Asia etc.South America Brazil, Argentina etc.Middle East & Africa Saudi Arabia, African countries etc.

Customization of the Report This report can be personalized to meet your requirements. Get in touch with our sales representative now with a guarantee to get access to a report that suits best to your business needs.

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Key Reasons to Purchase To gain insightful analyses of the market and have a comprehensive understanding of the Cancer Gene Therapy and its commercial landscape. Assess the Cancer Gene Therapy production processes, major issues, and solutions to mitigate the development risk. To understand the most affecting driving and restraining forces in the Cancer Gene Therapy Market and its impact on the global market. Learn about the market strategies that are being adopted by your competitors and leading organizations. To understand the future outlook and prospects for Cancer Gene Therapy Market.

Major Topics Covered in this Report Chapter 1 Study CoverageChapter 2 Executive SummaryChapter 3 Market Size by ManufacturersChapter 4 Production by RegionsChapter 5 Consumption by RegionsChapter 6 Market Size by TypeChapter 7 Market Size by ApplicationChapter 8 Manufacturers ProfilesChapter 9 Production ForecastsChapter 10 Consumption ForecastChapter 11 Upstream, Industry Chain and Downstream Customers AnalysisChapter 12 Opportunities & Challenges, Threat and Affecting FactorsChapter 13 Key FindingsChapter 14 Appendix

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Global Cancer Gene Therapy Market Insight Growth Analysis on Volume, Revenue and Forecast to 2019-2025 - Filmi Baba

Coherent Market Research adds Cell and Gene Therapy Market Report provides an analytical assessment of the prime challenges faced by this Market currently and in the coming years, which helps Market participants in understanding the problems.

The Cell and Gene Therapy Market report provides in-depth analysis and insights into developments impacting businesses and enterprises on a global and regional level. This report studies the global Cell and Gene Therapy Market size, industry status and forecast, competition landscape and growth opportunity. This research report categorizes the global Cell and Gene Therapy Market by companies, region, type, and end-use industry.

Cell and Gene Therapy Market Report covers the following major players: Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST.

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This Cell and Gene Therapy market research study is a collection of insights that translate into a gist of this industry. It is explained in terms of a plethora of factors, some of which include the present scenario of this marketplace in tandem with the industry scenario over the forecast timespan.

The report is also inclusive of some of the major development trends that characterize the Cell and Gene Therapy market. A comprehensive document in itself, the Cell and Gene Therapy market research study also contains numerous other pointers such as the current industry policies in conjunction with the topographical industry layout characteristics. Also, the Cell and Gene Therapy market study is comprised of parameters such as the impact of the current market scenario on investors.

Promising Regions & Countries Mentioned In The Cell and Gene Therapy Market Report:

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Regional landscape:

How will the details provided in the report help prominent stakeholders?

The information that this study delivers, pertaining to the geographical landscape, is indeed rather vital.

As per the study, the topographical spectrum of this industry covers the geographies of North America, Europe, Asia-Pacific & Middle East and Africa.

The study, in detail, enumerates the overview of the regional scope with respect to the growth rate that is likely to be recorded by each region over the projected duration.

Other important aspects pertaining to the topographical reach that may prove important for buyers include the remuneration and the production volume with regards to each region. The market share which every region holds in the industry has also been provided.

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Cell and Gene Therapy Market in-depth Analysis Including Key Players and Their Future Growth Rate Till 2027 - Market Research Sheets

WVE-120102 demonstrates statistically significant reduction in disease-causing mutant HTT protein in CSF vs. placebo

No difference in total HTT protein or neurofilament light chain in treated patients vs. placebo

Additional cohort expected to initiate in January 2020

Wave to host investor conference call and webcast at 8:00 a.m. ET today

CAMBRIDGE, Mass., Dec. 30, 2019 (GLOBE NEWSWIRE) -- WAVE Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced topline data from the ongoing Phase 1b/2a PRECISION-HD2 trial evaluating investigational therapy WVE-120102, designed to be the first allele-selective approach to treat Huntingtons disease (HD). In an analysis comparing all patients treated with multiple intrathecal doses of WVE-120102 to placebo, a statistically significant reduction of 12.4% (p<0.05) in mutant huntingtin (mHTT) protein was observed in cerebrospinal fluid (CSF). An analysis to assess a dose response across treatment groups (2, 4, 8 or 16 mg) suggested a statistically significant response in mHTT reduction at the highest doses tested (p=0.03). WVE-120102 was generally safe and well tolerated across all cohorts. These data support the addition of higher dose cohorts, and Wave expects to initiate a 32 mg cohort in January 2020.

This topline analysis has given us the opportunity to evaluate early data from our ongoing dose finding study. The data demonstrate a reduction in mutant HTT and a safety and tolerability profile that supports exploration of higher doses of WVE-120102, with the goal of maximizing mutant HTT reduction and avoiding a negative impact on the healthy huntingtin protein, said Michael Panzara, MD, MPH, Chief Medical Officer ofWave Life Sciences. We plan to initiate the 32 mg cohort imminently and look forward to sharing data in the second half of 2020.

WVE-120102 was developed as an allele-selective molecule, designed to preferentially lower mHTT protein by targeting single nucleotide polymorphism (SNP) rs362331 (SNP2) in order to keep the level of healthy or wild-type HTT (wtHTT) protein relatively intact. The wtHTT protein is important for neuronal function and may be neuroprotective in an adult brain. There is currently no assay available to directly measure wtHTT in the CSF; therefore, Wave is using an assay developed by CHDI Foundation, a not-for-profit biomedical research organization devoted to HD, to measure total HTT (tHTT) protein to indirectly assess effects on wtHTT. With this assay, a non-allele selective, pan-silencing approach would be expected to lead to a commensurate reduction in tHTT relative to mHTT.

While there was a statistically significant reduction in mHTT with WVE-120102 compared to placebo in the topline analysis, there was no difference in tHTT compared to placebo, suggesting WVE-120102 may have a potentially differential effect on huntingtin as measured by the mHTT and tHTT assays. Wave will continue to explore these potential effects with higher doses, where larger reductions of mHTT may be expected and where a more discernible impact on tHTT may be observed. Wave is continuing to work with CHDI Foundation and other research partners on methods to assess wtHTT protein preservation.

The topline analysis also assessed the presence of neurofilament light chain (NfL) in the CSF, and there was no difference in NfL between the WVE-120102 and placebo-treated groups. NfL is a protein component of the neuronal cytoskeleton. Its levels in the CSF are generally elevated when neurons are damaged in the setting of many neurological disorders, including HD.

WVE-120102 was generally safe and well tolerated among patients receiving doses up to 16 mg in both single and multidose portions of the study. A total of 72% of those who received WVE-120102 experienced an adverse event (AE) as compared with 83% on placebo, most of which were mild to moderate in intensity. The most common AEs (those occurring in at least 10% of patients on WVE-120102) were headache, procedural pain, falls and viral upper respiratory infection. There were no serious adverse events (SAEs) related to treatment with WVE-120102 and no study stopping rules were met, allowing dose escalation to continue. There were no notable changes in laboratory tests including liver or renal function tests, platelets or markers of immune activation.

PRECISION-HD2 is an ongoing, Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled trial, which is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple doses of WVE-120102 in adult patients with early manifest HD who carry SNP2. The trial included both single and multidose portions where patients were randomized to either WVE-120102 or placebo and received up to four intrathecal doses. The trial was designed for patients to receive a single dose and then undergo a washout period of at least eight weeks before entering the multidose portion which includes three monthly doses. Forty-four patients (31 WVE-120102 and 13 placebo) participated in the multidose portion and data from 39 patients (27 WVE-120102 and 12 placebo) were available for mHTT assessment as of data cut-off. In October 2019, Wave initiated an open-label extension (OLE) study open to patients outside of the U.S. who participated in the Phase 1b/2a PRECISION-HD2 trial and dosing of patients in the OLE is ongoing.

Wave is also conducting the PRECISION-HD1 Phase 1b/2a trial assessing WVE-120101 in early manifest HD patients who carry SNP rs362307 (SNP1). PRECISION-HD1 includes four cohorts (2, 4, 8 or 16 mg intrathecal doses). Given the PRECISION-HD2 results, PRECISION-HD1 will remain blinded and Wave plans to add a 32 mg cohort. Topline results from PRECISION-HD1, including those from the 32 mg cohort, are now expected in the second half of 2020.

Investor Conference Call and WebcastWave management will host an investor conference call today at 8:00 a.m. ET to discuss these topline results. The conference call may be accessed by dialing (866) 220-8068 for participants based in the United States or +1 (470) 495-9153 for participants based outside the United States and entering conference ID 9594572. The live webcast may be accessed by visiting the investor relations section of the Wave Life Sciences corporate website at http://www.ir.wavelifesciences.com. Following the webcast, a replay will be available on the website.

About Huntingtons Disease Huntingtons disease (HD) is a debilitating and ultimately fatal autosomal dominant neurological disorder, characterized by cognitive decline, psychiatric illness and chorea. HD causes nerve cells in the brain to deteriorate over time, affecting thinking ability, emotions and movement. HD is caused by an expanded cytosine-adenine-guanine (CAG) triplet repeat in the huntingtin (HTT) gene that results in production of mutant HTT (mHTT) protein. Accumulation of mutant HTT causes progressive loss of neurons in the brain. Wild-type, or healthy, HTT (wtHTT) protein is critical for neuronal function and suppression may have detrimental long-term consequences. Approximately 30,000 people in the United States have symptomatic HD and more than 200,000 others are at risk for inheriting the disease. There are currently no approved disease-modifying therapies available.

About WVE-120101 and WVE-120102WVE-120101 and WVE-120102 are investigational stereopure antisense oligonucleotides designed to selectively target the mutant huntingtin (mHTT) mRNA transcript of SNP rs362307 (SNP1) and SNP rs362331 (SNP2), respectively. SNPs, or single nucleotide polymorphisms, are naturally occurring variations within a given genetic sequence and in certain instances can be used to distinguish between two related copies of a gene where only one is associated with the expression of a disease-causing protein.

In vitro studies in patient-derived cell lines have shown that WVE-120101 and WVE-120102 selectively reduce levels of mHTT mRNA transcript and protein, while leaving wild-type, or healthy, HTT mRNA transcript and protein relatively intact. The healthy transcript is required to produce wild-type HTT (wtHTT) protein, which is important for neuronal function, as evidenced by multiple preclinical studies indicating that long-term suppression of wtHTT protein may have detrimental consequences. Waves allele-specific approach may also enable the company to address the pre-manifest, or asymptomatic, HD patient population in the future.

About Wave Life Sciences Wave Life Sciences (Nasdaq: WVE) is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases. Wave aspires to develop best-in-class medicines across multiple therapeutic modalities using PRISM, the companys proprietary discovery and drug development platform that enables the precise design, optimization and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future. To find out more, please visit http://www.wavelifesciences.comand follow Wave on Twitter @WaveLifeSci.

Forward-Looking StatementsThis press release contains forward-looking statements concerning our goals, beliefs, expectations, strategies, objectives and plans, and other statements that are not necessarily based on historical facts, including statements regarding the following, among others: our commitment to advancing genetic medicines; our intent to add higher dose cohorts to PRECISION-HD2, including our plans to initiate a 32 mg cohort in January 2020 and to report that data in the second half of 2020; our intent to add higher dose cohorts to the Precision-HD1 trial, including a 32 mg cohort, and to report topline results from PRECISION-HD1, including those from the 32 mg cohort, in the second half of 2020; our ability to deliver on the promise of our current and future pipeline; the future performance and results of our programs in clinical trials and in preclinical development; and the benefit of nucleic acid therapeutics generally. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including the following: our ability to finance our drug discovery and development efforts and to raise additional capital when needed; the ability of our preclinical programs to produce data sufficient to support our clinical trial applications and the timing thereof; our ability to continue to build and maintain the company infrastructure and personnel needed to achieve our goals; the clinical results of our programs, which may not support further development of product candidates; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials; our effectiveness in managing future clinical trials and regulatory processes; the effectiveness of PRISM; the continued development and acceptance of oligonucleotides as a class of medicines; our ability to demonstrate the therapeutic benefits of our candidates in clinical trials, including our ability to develop candidates across multiple therapeutic modalities; our dependence on third parties, including contract research organizations, contract manufacturing organizations, collaborators and partners; our ability to manufacture or contract with third parties to manufacture drug material to support our programs and growth; our ability to obtain, maintain and protect intellectual property; our ability to enforce our patents against infringers and defend our patent portfolio against challenges from third parties; and competition from others developing therapies for similar uses, as well as the information under the caption Risk Factors contained in our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) and in other filings we make with the SEC from time to time. We undertake no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstances.

Investor Contact:Kate Rausch617-949-4827krausch@wavelifesci.com

Media Contact:Alicia Suter617-949-4817asuter@wavelifesci.com

Patient Contact:Nikki Levy617-475-7236nlevy@wavelifesci.com

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Wave Life Sciences Announces Topline Data and Addition of Higher Dose Cohort in Ongoing Phase 1b/2a PRECISION-HD2 Trial in Huntington's Disease -...

The global CNS Gene Therapy Market reached ~US$ xx Mn in 2019 and is anticipated grow at a CAGR of xx% over the forecast period 2018 2028. The business intelligence study of the CNS Gene Therapy Market covers the estimation size of the market both in terms of value (Mn/Bn USD) and volume (x units).

In a bid to recognize the growth prospects in the CNS Gene Therapy Market, the market study has been geographically fragmented into important regions that are progressing faster than the overall market.

Each market player encompassed in the CNS Gene Therapy Market study is assessed according to its market share, production footprint, current launches, agreements, ongoing R&D projects, and business tactics. In addition, the CNS Gene Therapy Market study scrutinizes the strengths, weaknesses, opportunities and threats (SWOT) analysis.

ThisPress Release will help you to understand the Volume, growth with Impacting Trends. Click HERE To get SAMPLE PDF (Including Full TOC, Table & Figures) athttps://www.persistencemarketresearch.co/samples/27514

What insights readers can gather from the CNS Gene Therapy Market report?

The CNS Gene Therapy Market report answers the following queries:

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key players and product offerings

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Persistence Market Research (PMR) is a third-platform research firm. Our research model is a unique collaboration of data analytics and market research methodology to help businesses achieve optimal performance.

To support companies in overcoming complex business challenges, we follow a multi-disciplinary approach. At PMR, we unite various data streams from multi-dimensional sources. By deploying real-time data collection, big data, and customer experience analytics, we deliver business intelligence for organizations of all sizes.

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CNS Gene Therapy Market To Boost Demand And Forecast In Upcoming Year 2018 2028 - Market Reports Observer

Overview of Precision Cancer TherapiesMarket Report 2019

The report has been prepared based on the synthesis, analysis, and interpretation of information about the Precision Cancer Therapiesmarketcollected from specialized sources. The competitive landscape section of the report provides a clear insight into the market share analysis of key industry players. company overview, financial overview, product portfolio, new project launched, recent development analysis are the parameters included in the profile.

Precision cancer therapies is an evolving approach to cancer care that aims to leverage new knowledge regarding the pathogenesis of cancer to more precisely target therapy.Precision cancer medicine utilizes molecular diagnostic testing, including DNA sequencing, to identify cancer-driving abnormalities in the tumors genome.

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The key manufacturers in this market include :Abbott Laboratories, Bayer HealthCare, GlaxoSmithKline, OncoGenex Pharmaceuticals, Hospira, Boehringer Ingelheim, AstraZeneca, Aveo Pharmaceuticals,

By the product type, the market is primarily split into : Hormone Therapy, Immunotherapies, Targeted Therapy, Monoclonal Antibody Therapy, Gene Therapy,

By the end users/application, this report covers the following segments : Hospitals, Diagnostic Centers, Oncology Clinics, Research Institutes,

The Precision Cancer Therapies market report is a most important research for who looks for complete information on the Precision Cancer Therapiesmarket 2019. The report covers all information on the global and regional markets including old and future trends for market demand, size, trading, supply, competitors, and prices as well as global predominant vendors information. the report also provides a complete overview of Precision Cancer Therapiesmarket including Top Players or vendors, application, Type, Share, and latest market trends.

It drives closely through the prevalent regulatory landscape in various regions including, Europe, Asia-Pacific, North America, Latin America and the Middle East & Africa.

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The objective of the study is to define market sizes of different segments & countries in previous years and to forecast the values to the next eight years. The Precision Cancer Therapies Market report is designed to incorporate both qualitative and quantitative aspects of the industry with respect to each of the regions and countries involved in the study.

The study objectives of this report are:

At last, All the segments have been analyzed based on present and future trends and the market is estimated from 2019to 2025. Relevantly, the report and company profiles specify the key drivers that are impacting the demand in global Precision Cancer Therapiesmarkets.

Read Complete Report With TOC : @ https://www.garnerinsights.com/Global-Precision-Cancer-Therapies-Market-Size-Status-and-Forecast-2019-2025

Thus, Precision Cancer TherapiesMarket Report 2019 serves as a valuable material for all industry competitors and individuals having a keen interest in Precision Cancer TherapiesMarket study.

Contact UsKevin Thomas[emailprotected]Contact No:+1 513 549 5911 (US)+44 203 318 2846 (UK)

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Exclusive Research on Precision Cancer Therapies Market 2019-2025 Industry Growth Prospects by Key Companies Abbott Laboratories, Bayer HealthCare,...

Protein Associated with Leukemia May Lead to Targeted Therapy for Currently Incurable Acute Lymphoblastic Leukemia

ALL is a form of blood cancer that primarily affects children and young people and causes large quantities of malignant progenitor cells to build in a patients blood instead of healthy white blood cells. This is often caused by 2 chromosomes fusing together to create new abnormal genes that disrupt the system controlling normal blood development. Because of this process, certain types of leukemia are extremely resistant and unable to be cured with intensive chemotherapy or stem cell transplantation.

Researchers analyzed a protein called TCF3-HLF, which is typically associated with this type of leukemia and does not occur naturally. It is produced through the fusion of 2 chromosomes and contains elements of transcription factors, which activate the transcription of certain genes.

The analysis revealed that TCF3-HLF activates a whole range of genes, but it does so in the wrong contextat the wrong point in the blood development process. The formation of malignant white blood cells is then triggered, causing leukemia.

The study authors also discovered that the abnormal protein does not act alone, but instead gathers more than 100 other proteins around it, which helps to activate the genes. The researchers investigated the function of the individual proteins in the genetic machinery and used it to identify key elements that could be targeted through therapy.

Using the CRISPR/Cas9 method, researchers detached the specific parts they had identified from the machinery and found 11 critical factors that are crucial to the build-up of malignant abnormal blood cells in leukemia.

One of the essential components now identified is the protein EP300, a cofactor that boosts gene activation. The researchers used a new kind of substance called A-485, known to bind to EP300 and inhibit its activity. When A-485 was administered to human leukemia cells, the malignant cells died off.

The study authors noted that it is possible to stop the fundamental driving force behind the leukemia directly and thus develop a targeted type of therapy. Given that other forms of leukemia are caused by similar mechanisms, it may also be possible to identify a common denominator for developing new drugs to combat cancer.

REFERENCE

New approach to treating incurable leukemia in children discovered [press release]. University of Zurich. BioPortfolio website. Published November 24, 2019. https://www.bioportfolio.com/news/article/4148041/New-approach-to-treating-incurable-leukemia-in-children-discovered.html. Accessed December 4, 2019.

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Protein Associated with Leukemia May Lead to Targeted Therapy for Currently Incurable Acute Lymphoblastic Leukemia - Pharmacy Times

Throughout the year we have published the views of company executives, government regulators, industry analysts and scientists on a variety of topics and, in our popular annual feature, we include a selection of these that paints a picture of the significant events that shaped 2019. The major talking point was on the capital markets front where investors turned their backs on the biopharmaceutical sector for most of the year returned big time in the final quarter. M&As helped sparked life, with blockbuster deals taking place throughout the year, particularly in the red hot cell and gene therapy space, with an exclamation point added this week as Roche Holding AG followed up the completion of its $4.8 billion acquisition of Spark Therapeutics Inc. with the announcement of a blockbuster deal with Sarepta Therapeutics Inc., underscoring big pharmas appetite for gene therapy technologies.

Our favorite quote from the year

The Wright brothers showed that you could fly a plane, but it wasnt very far, and it wasnt very safe. Thats where cell therapy is now.At a scientific session at the American Society of Hematology meeting on gene editing, Wendell Lim, professor and chair of cellular and molecular pharmacology at the University of California, San Francisco, and the co-founder of Cell Design Labs, discussed his laboratorys work that uses gene editing to improve cell therapies, specifically CAR T cells

Capital markets

The biopharmaceutical sector headed into 2019 on a negative note, with most of the damage inflicted in December 2018 a month that will go down as one of the worst for equities, with the Dow Jones Industrial Average stumbling by almost 9% and the Nasdaq Composite performing just as poorly, dropping 9.5%.

Summing up the situation, the Cowen and Co. biotechnology analyst team wrote in its monthly biotech thermometer report to clients: "Investors are shell-shocked by the depreciation that was so quick, so dramatic, and so close to the end of the year. Sentiment is worse than we can remember over the last five years, and in fact many investors have developed a bear market mindset."

Fast forward 12 months and the curtain is now closing on a year where, for many months, the biopharmaceutical industry languished and company executives undoubtedly were wondering, during that difficult period, whether there would be a reversal of fortunes.

The answer came in the fourth quarter. To the surprise of industry pundits, public biopharmaceutical companies turned the corner and are on a major upswing, with the sector outperforming the general market in final frame with a 23% upswing year-to-date.

Reflecting on the change, Cowen and Co. analysts, writing in their monthly Biotech Thermometer noted, "Those who stuck with the sector through the famine of Q3 have feasted on a cornucopia of returns during the first two months of Q4."

Bear to bull

Most analysts speculated at the beginning of the year that the recipe to create an upswing in the sectors fortunes would be tied to an increase in M&A activity. SVB Leerink analyst Joseph Schwartz was certainly prescient in a note to investors earlier this year pointing to gene therapy companies as being prime targets. "With increasing pricing pressure and the emphasis on 'value' from payers, we believe curative gene therapy programs represent ideal acquisition targets for large pharma or biotech companies looking to diversify their product portfolios and reduce their reliance on price hikes."

RBC Capital Markets analyst Brian Abrahams, writing in the 2019 RBC Biotech Outlook Report, struck a similar note: "For 2019, we are more neutral vs. 2018, as growth and competitive headwinds, alongside more tangible manifestations of drug cost containment, potentially mitigate increasingly attractive valuations among larger-caps and what we expect will be increasing M&A activity and enthusiasm backstopping the smids."

Industry reels from FDA commissioner resignation

The announcement from Scott Gottlieb that he was resigning as FDA commissioner to return to the American Enterprise Institute in April took the industry by surprise, and the news weighed heavily on biopharmaceutical equities, with the BioWorld Biopharmaceutical index falling almost 4% on the news.

"Scott has helped us to lower drug prices, get a record number of generic drugs approved and onto the market, and so many other things. He and his talents will be greatly missed."President Donald Trump's tweet in response to Scott Gottlieb's announcement that he was resigning as FDA commissioner

The industry had to wait until December for a replacement when the U.S. Senate voted 72-18 to confirm Stephen Hahn as the commissioner of the FDA, providing the agency with another commissioner with a deep background in oncology. Secretary of Health and Human Services Alex Azar thanked the Senate for prioritizing the candidates nomination, adding that the development will be a major boost to the already rapid pace of the Presidents aggressive public health agenda.

Setting the agenda

Looking at the BioWorld Biopharmaceutical index it reveals that it did attain an almost 12% increase in value by February. Catalyzing the jump was the positive vibe that emanated from the January J.P. Morgan 37th annual health care conference, which every year sets the industrys agenda. From the market reaction to the upcoming plans laid bare by presentations from many of the leading biotechnology and big pharma companies in attendance, it appeared that the sector came out ahead.

Business development

"Once again we're seeing a lot of billion-dollar deals and they tend to be preclinical-stage deals that have multiple assets. It's a trend that started in 2016."David Thomas, vice president of industry research and analysis for the Biotechnology Innovation Organization, on its 2019 Emerging Therapeutic Company Trend Report, which showed that the number of global R&D-stage licensing deals surged by 107% in 2018 over the prior year

"The shift away from larger market drugs to more niche indications reflects the change in R&D focus of biopharma companies."Roger Humphrey, executive managing director and leader of JLL's Life Sciences group

Despite a decade of effort to streamline discovery and development and increase productivity, the projected return on investment in R&D at the worlds leading pharmaceutical companies has hit an all-time low, according to the 10th annual analysis by management consultancy Deloitte.

No other industry would operate on such low R&D returns.Substantive change is needed to shorten R&D cycle times, according to Karen Taylor, director of Deloittes health practice

Cash flows

Despite a rough ride on the capital markets for much of the year, particularly in the second and third quarters, the biopharmaceutical sector had no difficulty in attracting capital.

[This is] "the most exciting time to be investing in life sciences in a generation."Otello Stampacchia, managing director, Omega Funds, which raised $438 million to deploy into new and existing life sciences companies

"Capital is so available, it strikes me insane that almost half the industry needs to raise money this year."Dennis Purcell, founder and senior advisor at Aisling Capital LLC, during a session at the BIO CEO & Investor Conference

"It's very much a chess game in the current market; you have to think multiple steps out."Nol Brown, managing director, health care investment banking, at Cantor Fitzgerald, on planning whether to IPO during a session at the BIO CEO & Investor Conference

"It's a great time to be an entrepreneur. There are so many sources of capital. And there's a lot of it."Chau Quang Khuong, private equity partner at Orbimed Advisors, speaking at the Biocom Global Life Science Partnering Conference on the advantages of starting a company now

M&As

Bristol-Myers Squibb Co.'s announced $74 billion bid for Celgene Corp., the largest M&A in biopharma history, made for what J.P. Morgan analyst Cory Kasimov called "one of the most significant pre-conference announcements ever."

Although the deal did not spark a wave of M&A activity at the time, transactions remained sporadic for the rest of the year. According to the 2020 RBC Biotech Outlook report, Despite the unevenness throughout the course of 2019, the forces we believed would stimulate a meaningful pickup in acquisitions coalesced to generate new highs in biopharma acquisitions, with a record number of announced $1B+ public company deals.

"Two turkeys don't make an eagle. Both businesses have seen their share of challenges, with ABBV's Humira seeing competitive pressures both in the U.S. and EU and Allergan's rather well-documented business challenges. However, financial potential does have our attention, with the deal being immediately accretive to earnings."Christopher J. Raymond, Piper Jaffray analyst, in a research note on the first reaction to the announcement that Abbvie Inc. was proposing to acquire Allergan plc in a cash and stock deal worth about $63 billion

Gene therapy is hot

Barely a day went by this year without news relating to cell and gene therapy companies being announced. Given the ongoing developments, it was not surprising that the sector continued to attract financing and business development, with biopharma companies ensuring that they secured a position in the space for themselves through partnering and acquisitions.

"Gene therapy gets around many of the drug delivery challenges that have plagued other treatment modalities like small molecules. In part, that is because it's a one-time approach and also it really enables you to target the tissue bed of interest, in some cases direct to the CNS."Shankar Ramaswamy, chief business officer, Axovant Gene Therapies Ltd.

"Gene therapies are among the most exciting medical advancements of our time. However, they also pose a great technical challenge, since the AAV particles designed to deliver genes to tissues are subject to immediate and overwhelming immune attack, specifically by complement C3, which may result in significant safety and efficacy constraints. We believe that the targeted control of C3 may prevent the C3-mediated attack on AAV particles."Cedric Francois, CEO and co-founder of Apellis Pharmaceuticals Inc.

"[Many gene therapies] are introduced with much higher levels of uncertainty about their long-term safety and effectiveness than standard treatments, and patients and insurers are being asked to pay extremely high prices up front for the promise of long-term benefit."Steven Pearson, president, The Institute for Clinical and Economic Review, which is proposing adaptations to the framework it uses in assessing the value of potential single or short-term transformative therapies (SSTs). The proposals are intended to help decision-makers conduct a more reliable and transparent evaluation of the uncertainty, value and value-based pricing of SSTs

Some of the gene therapeutic products and candidates have truncated the size of the genes and have done some pretty clever maneuvers to try and make the gene inserts smaller in order to be incorporated by AAV.Daniel Dornbusch, head of business development at Dnarx Inc., explained, while noting that the full length might be better and there are many genes that cant be truncated

"Gene editing is a rapidly developing technology that represents one of the most exciting developments in medicine. These techniques will be integral to the next generation of advanced therapeutics and we welcome their potential to provide important, and potentially life-saving, treatments for patients. As with all breakthrough biotechnologies, we need to exercise caution and good stewardship in our research and development practices and ensure that work involving the genetic modification of cells takes place within the bioethical framework outlined in these principles."Janet Lambert, CEO, The Alliance for Regenerative Medicine commenting on the release of the Alliance's Therapeutic Developers' Statement of Principles, setting forth a bioethical framework for the use of gene editing in therapeutic applications

Artificial intelligence and machine learning

There is no doubt that the next wave of drug discovery will be enabled by powerful supercomputers dining on complex algorithms to uncover potential new scientific approaches for the development of innovative therapeutics, and industry executives certainly provided BioWorld with a wide range of opinions on its value.

"The idea behind machine learning is that it's a method that should generalize and be able to make decisions or make estimates or classifications based on data it hasn't seen before."John Irvine, chief scientist for data analytics at Draper Laboratories, Siemens Healthineers. The firm is working with Brisbane's Translational Research Institute and using artificial intelligence to diagnose medical conditions ranging from breast cancer to post-traumatic stress disorder

"I think the drive in this field is that we saw the success of Google and Facebook to basically take what seemed like white noise out in the ether and squeeze out this gold; it's like the new gold rush. And in Alzheimer's we've been desperate for success, and it seems like we have this messy, noisy data it almost feels like static sometimes and we want a machine learning algorithm to come in and wrangle that up and squeeze gold out of what looks like noise. Unfortunately, I don't think it's going to be that easy."Newman Knowlton, a statistician at Millcreek, Utah-based statistical consulting firm Pentara Corp.

"When I have casual conversation about this, I think of the first cell phone and what iPhone is like today. At first, we wanted only a few functions, but we eventually realized we needed more functionality to benefit from the technology. It's the same in biomedicine."Ken Drazan, CEO, Arsenal Biosciences Inc.

"Data-driven medical innovation is growing exponentially, and our partnership with Google will help us lead the digital transformation in health care. It will empower us to solve some of the most complex medical problems; better anticipate the needs of people we serve; and meet them when, where and how they need us. We will share our knowledge and expertise globally while caring for people locally and always do it with a human touch."Gianrico Farrugia, president and CEO of Mayo Clinic

"Across the globe, clinicians spend more time doing data entry in patients' records than they do treating patients, and I think AI has a real opportunity to change that."Chris Nave, Managing Director, Brandon Capital

Editors note: See Fridays issue for Part 2.

More:
Perspectives on the year: The industry has its say on the markets, M&A and gene therapy - BioWorld Online

Abstract / Synopsis:

RGX-314 gene therapy, a treatment for wet age-related macular degeneration delivered subretinally, showed dose-dependent sustained/improved vision and a good safety profile.

This article was reviewed by Jeffrey S. Heier, MD

RGX-314 (REGENXBIO) gene therapy, delivered subretinally to patients with neovascular age-related macular degeneration (AMD), who required a large number of prior injections of anti-vascular endothelial growth factor (VEGF) annually, provided a marked decrease in the number of injections needed and retained or improved the vision and anatomy in this challenging patient population.

RGX-314 uses a proprietary gene delivery platform that is hypothesized to deliver longer and higher protein expression with a lower immune response that earlier generation adeno associated viruses (AAVs) used for gene therapy.

The AAV8 vector is encoded to deliver a gene that leads to anti-VEGF antibody fragment protein production within the retina, said Jeffrey S. Heier, MD, co-president and director of Retinal Research, Ophthalmic Consultants of Boston.

Related: Anti-VEGF targeted as potential nAMD treatment

All 42 patients included in this study had undergone previous treatment for neovascular AMD and had a high need for anti-VEGF therapy, i.e., patients received more than 30 injections on average prior to coming in and an average of 9.6 annualized injections in the year prior to RGX-314.

In addition, the included patients had to have shown an anatomic response to anti-VEGF therapy during screening. After RGX-314 was delivered to the subretinal space, the patients were evaluated monthly to assess safety and the need for additional anti-VEGF therapy, Dr. Heier noted.

Re-treatment with an anti-VEGF drug could be provided beginning at four weeks after RGX-314 was delivered and then as needed every four weeks thereafter based on physician discretion, Dr. Heier explained. The criteria for retreatment was per investigator discretion which included choroidal neovascularization-related increased, new, or persistent fluid; vision loss of five or more letters as a result of fluid, or a new ocular hemorrhage.

Related: The ABCs of VEGF treatment for diabetic macular edema

The resultsFollowing delivery of RGX-314, Dr. Heier reported that the drug was well tolerated. No serious adverse events were reported; most adverse events were classified as mild, i.e., grade 1 in 79% of cases. In addition, no clinically determined immune responses occurred.

Two deaths that occurred were not related to RGX-314. Two serious adverse effects that were related to the delivery procedure were reported, specifically, a peripheral retinal detachment that was repaired successfully and a case of endophthalmitis that occurred after collection of an aqueous sample.

We observed a dose-dependent increase in RGX-314 protein across the five dose cohorts, he reported. The protein levels were measured in aqueous samples collected one month after delivery of RGX-314.

Cohort 3, which included six eyes treated with a dose of 6 x 1010 genome copies (GC)/eye, did well.

Cohort 3 had a gain in vision of +9 letters and stable anatomy of -40 m over 18 months despite relatively few injections, Dr. Heier reported.

Three of the six patients in this cohort were injection-free at 18 months. They had a mean increase of +11 letters of vision at 18 months and a concomitant mean decrease in the central retinal thickness of -21 m. The RGX-314 protein levels were sustained over one year.

Related: Decreasing burden of nAMD therapy

Originally posted here:
Study targets subretinal option for AMD treatment - Ophthalmology Times

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Cell and Gene Therapy Market Size Will Escalate Rapidly in the Near Future - Market Research Sheets

Beyond the bright colors and hallucinogenic imagery of psychedelic artthe visuals of Ram Dass's 1971 book Be Here Now has never left public consciousness there has long been a crusade to clinically research substances such as LSD, psilocybin, MDMA, DMT, and ibogaine. We've been informed, again and again and again, about the various ways that current pharmaceutical treatments in our for-profit mental health system is not only not working, but doing more damage than healing. Discussion over health care inevitably defaults to mechanisms for paying for a broken model, rarely touching upon the root causes of why so many people are depressed, sick, anxious, and suicidal in the first place.

We. Need. Better. Solutions.

In regard to psychedelics, an entire herd of elephants remain locked in a room. Thanks to the questionable (and admittedly racist) wars launched by the Nixon and Reagan administrations (first dreamed up during the Anslinger crusades), we've been denied access to these potentially therapeutic substances. Fortunately, a renaissance is occurring in psychedelics research, with ketamine being the first to be legally prescribed psychedelic for treatment-resistant depression and both psilocybin and MDMA being fast-tracked by the FDA after being labelled breakthrough therapies.

One challenge psychedelics advocates will have to face is how these drugs are treated moving through the current medical model. Regardless of personal feelings on the subject, these substances have to contend with a system that requires expensive clinical trials and will be sold in a capitalist marketplace. There will inevitably be patent issues and territorial fights. Unlike cannabis, which is a relatively mild substance with few documented consequences, psychedelics need to be rigorously evaluated and tested. While some label everyone working in medicine as minions of Big Pharma, we need to separate researchers and scientists from the shady dealings of shareholders and profiteers.

Michael Ehlers is an industry figure that has long taken an interest in psychedelics, predominantly from an outsider perspective. Now the former executive vice president for research and development at Biogen is accepting an advisory role with Field Trip Health, the psychedelics-focused organization that recently opened the world's first psilocybin research center. (You can listen to my talk with Field Trip co-founder, Ronan Levy, here.)

I chatted with Ehlers, he is also the former chief scientific officer for neuroscience at Pfizer, about his interest in psychedelics, their potential efficacy, their historical usage in ritual, and how the current model will deal with their vetting and potential applications. With every question, he was informed and honest, offering what he knows and being truthful about what he does not. There is a lot of work ahead in pharmaceuticals, yet it is undeniable the mental health industry needs a reboot, in the same way psychedelics are said to reboot the neural circuitry of the brain, making this class of substances an ideal medicine for study.

Part of my conversation with Ehlers is below; you can read the full transcript here.

Photo courtesy of Michael Ehlers

Derek: You have an accomplished career in the pharmaceutical industry. Now you've taken on an advisory role with a company specializing in psychedelics. I would love to know when you first became interested in psychedelics as a potential therapeutic tool.

Michael: I've followed this area for quite some time. I've been intensely involved in different aspects of drug discovery and development, particularly, although not exclusively, within CNS or neuroscience drug discovery, including neuropsychiatric disease. I've followed more peripherally some of the efforts both in standard pharmacology and then some of the emerging work, whether it was more acute, high-dose psychedelics or microdosing psychedelics in neuropsychiatric disease.

At the same time, I was following a lot of the work on some of the core receptor biology and neurobiology, which was really advancing in systems neuroscience. Following this field and some of the early indications of potential clinical efficacy were some of the things that really got me quite excited. I was particularly close with aspects of what's been done over the past 10 years with ketamine, which is a very different agent but also in the class, initially leading from small trials on ketamine for acute, anti-depressive actions, now to Janssen and J&J using a variation of this, esketamine, to get full-on FDA approval for the first new mechanism in depression in 20 years. The combination of these things indicated to me that there could be a new paradigm change or highly-active psychopharmacology to potentially treat some of these otherwise fairly intractable types of neuropsychiatric disorders.

There are some other things that were also on the horizon. The history of CNS drug development, particularly in neuropsychiatric disease, has been one where the empirical observations in human patients have really guided efficacious therapeutics by and large. Even though I know we like to talk a lot about rational drug discovery and development, at least in the field of neuropsychiatry, because there's still so much that is not known that we've had to rely a lot more on empirical observations in humans.

There's probably no more profound CNS pharmacology out there than that with psychedelics like psilocybin or LSD or ketamine. I've actually long thought it was just a matter of figuring out what a treatment paradigm could look likehow maybe when you dose it could you alter aspects of its dose exposure and distribution and then in what exact disease or syndrome.

Derek: You have a history of working with rare diseases. Field Trip is going to tackle a wide range of studies, but the ones that are really on everyone's mind (in terms of what psychedelics could potentially help) ranges from PTSD to treatment-resistant depression and anxiety. These are much more common diseases. Do you have any background in those diseases and, in the advisory role, what will you be doing for them?

Michael: I've got a lot of background in that. I worked for nine years in large biopharma, six years at Pfizer. I started in neuroscience and pain, but ultimately ran several divisions of Pfizer R & D, that did include rare disease, but included a bunch of other things. Then I ran R & D advising for three-and-a-half years. I've done clinical trials in depression, schizophrenia, PTSD, generalized anxiety disorder, Alzheimer's disease, and Parkinson's disease. I've done both rare diseases and a lot of common disorders: hemophilia, genetic disease, and some of the rare diseases as well. I've done stroke trials. I've had experience across a range.

One thing I like is about what Field Trip is doing and the prospect of these diseases is that they're incredibly common. Roughly 25 percent of people will have some experience with major depression in their lives. One percent of the world has schizophrenia. These are serious and significant disorders. I really love the fact that this fieldand Field Trip is really part of that in a leadership roleis looking to take some of these on.

Although the lore has been that there hasn't been that much innovation, I actually think that's not true. I think we're just at the beginning of a whole new era of advances in neuropsychiatric disease. I can point to several things that indicate that. I have a feeling that if we really understand that the best way to dose and conduct trials with psychedelics like psilocybin and be able to segment patients who are the most likely to benefit, this can become quite important.

Derek: You mentioned that pharma companies stepping away from neuropsychiatric disease. There is obviously a problem with SSRIs over the long-term. Efficacy rates tend to be high in the short-term, but over the long-term prove problematic. When you're stepping into substances that potentially could help treatment-resistant mental health diseases in one dose (or just a couple of doses), how do you think that companies are going to be able to monetize this, especially given the incredible amounts of money that have to go into R & D and clinical trials?

Michael: It's a very good question. I think we haven't solved that problem yet. There are a lot of open questions. Will some of these therapies really be single dose or short regiments and you're done? Will it have to be that there's some degree of maintenance where there's some regularity in the need for therapy? Will it really be like antibiotics or gene therapy? We don't know.

A lot of these neuropsychiatric diseases, although they're complex, have genetic features that are polygenetic but they're related. Whether you're talking about, schizophrenia, autism, bipolar disorder, ADHD, there's a complex genetic architecture that has shared features across all of those. The risk of relapse and occurrences will be there in a given population. I tend to think the likelihood of things like ketamine or psychedelic treatments for depression will be one of periodic needs.

The question you raised is an excellent one, which is what ultimately is the commercial model for that? Certainly, the hope is that it doesn't go down the road of antibiotics for which the commercial incentivization for real R & D and drug development has been catastrophic. I don't see that in this space. I just don't think it's going to be quite as simple as "one and done." The prevalence alone will be a strong incentive for investment when there's real efficacy potential.

Derek: Please correct me if I'm wrong; I'm fascinated by neuroscience, but not having an academic background my knowledge is limited. That's why I love talking to people about this. From my understanding, SSRIs work in a much different manner in terms of the serotonin release then psychedelics. Do you see any potential benefits or dangers in the ways that psychedelics deal with the serotonergic system?

Michael: It is quite different. From a simple pharmacology point of view, SSRIs are, as their name indicates, selective serotonin reuptake inhibitors: they block serotonin transporters that would normally release serotonin back up into nerve cells so that it increases serotonergic tone. Once released, it stays released in the extracellular space for longer, acting on all the different receptors in the places that it does.

The psychedelics typically act directly on serotonin receptors within serotonin transporters, but their action at different receptors has different potency. It's not a clean pharmacology. People will talk about 5-HT-2A receptors and they're clearly important, and there's been a lot of study on that, but we also know that if you just give a pure 5-HT-2A receptor an agonist you do not reproduce the effects of psilocybin or LSD.

The pharmacology is complex; it's clearly different than SSRIs. Obviously, the behavioral and therapeutic groups are very different. It just highlights that we really need to understand it better. It's going to reveal I think very important things about psychiatric disease and fundamental neuroscience.

A shaman gathers the raw materials to make ayahuasca in the jungle outside of Iquitos.

Photo by Andrew Lichtenstein/Corbis via Getty Images

Derek: One of the criticisms of the way that the industry is right now is that, why would a doctor spend an hour talking to a patient when you can see six patients in an hour and write a script? Efficacy rates are different for different people, dealing with the microbiome, for example, and the way that their gut processes drugs. It's a very complex issue. One thing I believe is going to be important is that psychotherapy is going to be tethered with psychedelics, especially if people have never done them before. Will that coupling provide a sustainable model?

Michael: Here's an aspect of what's important to understand: the field has understandably taken a cautious approach, which I think is warranted in this whole guided therapy concept and that will probably be required for certain dosing regimens. I would personally like to see this converted into what is a very standard thing in a lot of drug administration in practice or trials, which is more about medical monitoring. Change it from the notion of it's guided therapy to monitoring like you would for a lot of things. People go to IV infusion centers to get their IV drug. It's different, but there's nothing that unusual about the notion of having a monitored pharmaceutical or pharmacological drug intervention even in standard practice. This will likely be part of that.

If you're a neurologist treating MS and you've got MS patients on Alemtuzumab or Natalizumab as your IV drugs. They come in, you've got your IV clinic. They come in regularly, every month or every quarter depending on the drug, and they get their IV infusion. They get monitored while it happens because they can have an immune response. I see a future for some of these psychoactive therapeutics where you have something similar.

Now the question will be to what extent does the guided as opposed to monitoring aspect of that influence the degree of efficacy? That's something which really would need to be studied. To the extent it really requires some special type of guided activity that will be a little bit more of a limitation. To the extent that it can be ultimately the design in a more monitoring approach with education, the more widespread this can become.

Does that analogy make sense to you? There's a lot of precedence for this in other areas. The way this has gotten utilized now is still a remnant of causing people to have profound hallucinations and behavioral stuff and paranoia. Some people get afraid of that, so we need to have some monitoring.

We need to understand doses. We need to know the extent to which those experiences are part and parcel to a therapeutic response or not associated with a therapeutic response.

Derek: How much do you think anecdote is going to matter? One main issue I have with the whole cannabis legalization process is the extraction of CBD being sold for every possible ailment out there when the actual evidence is almost nothing at this point, besides epilepsy. At the same time, dealing with mental health disorders, how much are we going to rely on anecdote? If people think they're getting better, there's placebo, and it actually helps them get better.

Michael: I hope we moved beyond anecdotes, and I think that you're right about CBD, but it's interesting the way you put that because of the fact that rigorous trials have been done in rare epilepsies, like Dravet and Lennox-Gastaut syndrome, nobody disputes that. Patients in need can get insurance companies or health systems in other countries to reimburse for that. That's what I mean by saying real location impact is going to require that component of it too. You'd like to be able to generate the evidence because nothing comes without safety concerns. The nice thing about putting this all through the lens of drug discovery and development is that it allows the communityand here I mean the medical community, policymakers, others to have a much clearer view of the benefit-risk, and where the benefit-risk is positive, in which case that's usually a required element for real access for patients.

Of course, you could argue and say, "well, if it's just out there, people can try it, we'll see and that's fine," but this doesn't allow us from a clinical scientific vantage point to really know when and where we are going to provide benefits. That's what we really need to work toward. There's enough anecdotal evidence out there to justify rigorous evaluation.

--

Stay in touch with Derek on Twitter and Facebook. His next book is Hero's Dose: The Case For Psychedelics in Ritual and Therapy.

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The 2020s: The decade of psychedelic breakthroughs? - Big Think

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Gene Therapy Market gaining traction due to new market developments, says Polaris Analysts - News Cast Report

NEW YORK (Reuters) - Novartis is in discussion with patient groups over its lottery-style free drug program for its multi-million-dollar gene therapy for spinal muscular atrophy (SMA) after criticism that the process could be unfair to some babies with the deadly disease.

FILE PHOTO: The company's logo is seen at the new cell and gene therapy factory of Swiss drugmaker Novartis in Stein, Switzerland, November 28, 2019. REUTERS/Arnd Wiegmann

The company said on Friday that it will be open to refining the process in the future, but it is not making any changes at this time. The program is for patients in countries where the medicine, called Zolgensma, is not yet approved for the rare genetic disorder, which can lead to death and profound physical disabilities.

At $2.1 million per patient, Zolgensma is the worlds costliest single-dose treatment.

Novartis said the program will open for submission on Jan. 2 and the first allocation of drugs would begin in February. Novartiss AveXis unit, which developed the drug, will give out 50 doses of the treatment through June for babies under 2 years old, it said on Thursday, with up to 100 total doses to be distributed through 2020.

Patient advocacy group SMA Europe had a conference call with the company on Friday, according to Kacper Rucinski, a board member of the patient and research group who was on the call.

There are a lot of ethical questions, a lot of design questions that need to be addresses. We will be trying to address them in January, Rucinski said. He said the program has no method of prioritizing who needs the treatment most, calling it a Russian roulette.

The company said it developed the plan with the help of bioethicists with an eye toward fairness.

This may feel like youre blindly passing it out, but it may be the best we can do, said Alan Regenberg, who is on the faculty at Johns Hopkins Berman Institute of Bioethics and was not among the bioethicists Novartis consulted with on the decision. It may be impossible to separate people on the basis of prognosis out of the pool of kids under 2, he said.

According to Rucinski, the parties will continue their discussion in January to see what can be improved in the design of the program.

Novartis said on Thursday that because of manufacturing constraints it is focused on providing treatment to countries where the medicine is approved or pending approval. It has one licensed U.S. facility, with two plants due to come on line in 2020.

Zolgensma, hit by turmoil including data manipulation allegations and suspension of a trial over safety concerns, is the second SMA treatment, after Biogens Spinraza.

Not all of the SMA community are opposed to Novartis program.

Rajdeep Patgiri moved from the United Kingdom to the United States in April so his daughter could receive Zolgensma. She has responded well to the treatment, and Patgiri worries that negative attention to the program could keep patients from receiving the drug.

The best outcome for all patients would be if everybody could get the treatment. Given all the constraints, a lottery is probably the fairest way to determine who receives the treatment, he said.

Reporting by Michael Erman; Additional reporting by John Miller in Zurich; Editing by Leslie Adler

More:
Novartis in talks with patients upset about lottery-like gene therapy giveaway - Reuters

Roche obtains the exclusive right to launch and commercialize SRP-9001 outside the United States

At closing, Sarepta will receive an upfront payment of $1.15 billion, comprising $750 million in cash and $400 million in Sarepta stock, priced at $158.59 per share of common stock

Additionally, Sarepta is eligible to receive up to $1.7 billion in regulatory and sales milestones, plus royalties on net sales

Sarepta will continue to be responsible for clinical development and manufacturing of SRP-9001 with global clinical development costs shared equally with Roche

Sarepta will host a conference call on Monday, Dec. 23 at 08:30 a.m. ET

CAMBRIDGE, Mass., Dec. 23, 2019 (GLOBE NEWSWIRE) Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that Sarepta and Roche have entered into a licensing agreement providing Roche exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), Sareptas investigational gene therapy for Duchenne muscular dystrophy (DMD), outside the United States. Under the agreement, Sarepta will receive $1.15 billion in an upfront payment and an equity investment; up to $1.7 billion in regulatory and sales milestones; and royalties on net sales, anticipated to be in the mid-teens. In addition, Roche and Sarepta will equally share global development expenses. Sarepta retains all rights to SRP-9001 in the United States.

The collaboration combines Sareptas leading gene therapy candidate for DMD with Roches global reach, commercial presence and regulatory expertise to accelerate access to SRP-9001 for patients outside the United States. DMD is an X-linked rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. SRP-9001, currently in clinical development for DMD, is designed to deliver the micro-dystrophin-encoding gene directly to the muscle tissue for the targeted production of the micro-dystrophin protein.

As a mission-driven organization, we are inspired to partner with Roche with the goal of bringing SRP-9001 to patients outside the United States. This collaboration will not only increase the speed with which SRP-9001 could benefit DMD patients outside the United States, but will also greatly expand the scope of territories within which we could potentially launch SRP-9001 and improve and save lives, said Doug Ingram, president and chief executive officer, Sarepta. In addition to the validation that comes from joining forces with Roche, this licensing agreement one of the most significant ex-U.S. licensing transactions in biopharma will provide Sarepta with the resources and focus to accelerate our gene therapy engine and, if successful, bring SRP-9001 to patients as quickly as possible, potentially transforming the lives of countless DMD patients across the globe.

Said James Sabry, Head of Roche Pharma Partnering, We are excited to enter this licensing agreement with Sarepta. By working together to provide SRP-9001 to patients, we hope to fundamentally transform the lives of patients and families living with this devastating disorder for which there are currently only limited treatment options.

As part of the agreement, Sarepta will continue to be responsible for the global development plan and manufacturing build out for SRP-9001. Through its leading hybrid manufacturing platform, Sarepta will remain responsible for manufacturing of clinical and commercial supplies. Sarepta has also granted Roche an option to acquire ex-U.S. rights to certain future DMD-specific programs, in exchange for separate milestone and royalty considerations, and cost sharing.

The closing of the transaction is subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions. The parties anticipate that the agreement will close in the first quarter of 2020.

Goldman Sachs & Co. LLC is acting as the lead financial advisor to Sarepta. Morgan Stanley & Co. LLC is also serving as a financial advisor and Ropes & Gray LLP is serving as legal advisor to Sarepta.

Conference Call InformationThe conference call may be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for international callers. The passcode for the call is 2077714. Please specify to the operator that you would like to join the Sarepta Therapeutics Conference Call. The conference call will be webcast live under the investor relations section of Sareptas website at http://www.sarepta.com and will be archived there following the call for 90 days. Please connect to Sareptas website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

AboutSarepta TherapeuticsSarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for limb-girdle muscular dystrophy diseases (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For more information, please visit http://www.sarepta.com.

Sarepta Forward-Looking StatementThis press release contains forward-looking statements. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as believes, anticipates, plans, expects, will, intends, potential, possible and similar expressions are intended to identify forward-looking statements. These forward-looking statements include but are not limited to statements regarding the closing of the transaction; Sareptas right to receive any upfront payment or equity investment from Roche pursuant to the agreement; Sareptas right to receive regulatory and sales milestones, and royalty payments from Roche pursuant to the agreement; Roches obligation to share global development expenses pursuant to the agreement; the continued development and manufacturing of SRP-9001; SRP-9001 expected delivery of micro-dystrophin-encoding gene directly to the muscle tissue and the expected production of the micro-dystrophin protein; the expected increased speed with which SRP-9001 could benefit patients outside the United States and expansion of territories within which Sarepta could launch SRP-9001; the expectation that the licensing agreement will provide Sarepta with the resources and focus to accelerate its gene therapy engine and potentially bringing SRP-9001 to patients as quickly as possible and transforming the lives of countless DMD patients across the globe; potential regulatory approvals of SRP-9001; and the potential launch and commercialization of SRP-9001.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others, market conditions, the expected benefits and opportunities related to the licensing agreement may not be realized or may take longer to realize than expected due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement, challenges and uncertainties inherent in product research and development and manufacturing limitations; success in preclinical testing and early clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and early results from a clinical trial do not necessarily predict final results; our data for SRP-9001 may not be sufficient for obtaining regulatory approval; Sarepta may not be able to execute on its business plans, including meeting its expected or planned regulatory milestones and timelines, research and clinical development plans, and bringing SRP-9001 to market, for various reasons, some of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2018 and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Companys business, results of operations and the trading price of Sareptas common stock. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of Information

We routinely post information that may be important to investors in the For Investors section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics Announces Partnership with Roche in Territories Outside the United States for its Investigational Micro-dystrophin Gene Therapy...

MONTPELLIER, France--(BUSINESS WIRE)--Regulatory News:

Sensorion (Paris:ALSEN) (FR0012596468 ALSEN / PEA-PME eligible) a pioneering clinical-stage biotech company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, announces that the French government has awarded the PATRIOT consortium a Structuring Research and Development Project for Competitiveness (Projet de recherche et dveloppement Structurant pour la Comptitivit - PSPC) non-dilutive funding. This grant will be used to finance the development of SENS-401 in SSNHL, including work by Institut Pasteur to identify response biomarkers. This project fits with the spirit of the strategic collaboration established between Sensorion and Institut Pasteur and announced on the 27th of May 2019. The financing is conditioned upon the execution of a financing agreement with Bpifrance within three months.

The PATRIOT consortium consists of Sensorion, the French Army Biomedical Research Center (IRBA), Institut Pasteur and Electronique du Mazet1, a French MedTech company focusing on hearing assessment and diagnostics. This project structures collaborative research and development to respond to a high unmet medical need and contributes to the development of SENS-401 as a treatment option, up to regulatory approval.

The commitment and active involvement of the French ministry of Armed Forces to this project emphasizes on a clear unmet medical need and on the necessity to have efficient diagnostics and therapeutic solutions. Auditory deficit is an invisible injury causing disabilities and potential social isolation. An epidemiological surveillance report from the French armed forces epidemiology and public health center revealed that 62% of military personnel suffered from hearing loss following an acute noise trauma over the 2014-2016 period.2

The participation of the French Army in the Phase 2 study represents the largest military trial ever conducted in France. It will be carried out in multiple military sites where patients will be recruited according to the protocol approved by an ethics committee.

We are extremely pleased to have the support of the French ministry of Armed Forces and the involvement of military personnel in our ongoing Phase 2 study. We believe their involvement significantly de-risks the overall study and increases the quality of the data we can collect. The project will receive 10.8m non-dilutive funding, staged over five years. Sensorion will receive 5.6m over the duration of the project, says Nawal Ouzren, CEO of Sensorion.

About SENS-401

SENS-401 (Arazasetron), is a drug candidate that aims to protect and preserve inner ear tissue from damage that can cause progressive or sequelar hearing impairment. A small molecule that can be taken orally or via an injection, SENS-401 has received Orphan Drug Designation in Europe for the treatment of sudden sensorineural hearing loss, and Orphan Drug Designation from the US FDA for the prevention of platinum-induced ototoxicity in pediatric population. It has received Investigational New Drug (IND) clearance from the US Food and Drug Administration (FDA).

About SENS-401 Phase 2 trial

The AUDIBLE-S Phase 2 is a multi-center, randomized, double-blind, placebo-controlled study of SENS-401 in subjects with severe or profound sudden sensorineural hearing loss (SSNHL). Included patients will receive twice a day for 4 weeks one of the following: a 43,5mg dose of SENS-401, a 29mg dose of SENS-401 or a placebo. The primary endpoint is change in pure tone audiometry PTA (dB) in the affected ear from baseline to the end of treatment visit (day 28).

About Sensorion

Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders. Its clinical-stage portfolio includes one Phase 2 product: SENS-401 (Arazasetron) for sudden sensorineural hearing loss (SSNHL). Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases enabling it to select the best targets and modalities for drug candidates. The Company has also identified biomarkers to improve diagnosis and treatment of these underserved illnesses. Sensorion has launched in the second half of 2019 two preclinical gene therapy programs aiming at correcting hereditary monogenic forms of deafness including Usher Type 1 and deafness caused by a mutation of the gene encoding for Otoferlin. The Company is uniquely placed through its platforms and pipeline of potential therapeutics to make a lasting positive impact on hundreds of thousands of people with inner ear related disorders; a significant global unmet medical need.

http://www.sensorion-pharma.com

Label: SENSORION ISIN: FR0012596468 Ticker symbol: ALSEN

Disclaimer

This press release contains certain forward-looking statements concerning Sensorion and its business. Such forward looking statements are based on assumptions that Sensorion considers to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, which statements are subject to numerous risks, including the risks set forth in the "Document de reference" registration document filed with the "Autorit des Marchs Financiers" (AMF French Financial Market Authority) on September 7th, 2017 under nR.17-062 and to the development of economic conditions, financial markets and the markets in which Sensorion operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Sensorion or not currently considered material by Sensorion. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Sensorion to be materially different from such forward-looking statements.

This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a solicitation of an offer to purchase or subscribe for, Sensorion shares in any country. The communication of this press release in certain countries may constitute a violation of local laws and regulations. Any recipient of this press release must inform oneself of any such local restrictions and comply therewith.

1 Through its subsidiary named ECHODIA2 Traumatismes sonores aigus dans les armes - Rsultats de la surveillance pidmiologique 2014-2016 Centre dpidmiologie et de sant des armes July 12, 2018

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Sensorion Announces 5.6m Non-Dilutive Funding to Support SENS-401 Phase 2 Study in Sudden Sensorineural Hearing Loss; French Ministry of Armed Forces...

The world awoke to the need to combat global heating

Was 2019 the year people finally started to listen to climate scientists on global heating? The previous year, the Intergovernmental Panel on Climate Change (IPCC) had laid out the monumental challenge of limiting warming to 1.5C. Global CO2 emissions would need to halve within 12 years, and reach zero around 2050. But emissions are still rising, while UN summits make tiny steps towards agreeing how to reduce them. The emissions gap between target and reality grows ever wider and becomes ever harder to close.

This struck a chord with vast numbers of people, especially the young, who are thinking ahead to what their world could look like. Greta Thunberg kickstarted a global movement of school strikers, demanding that governments listen to the science and act as if the house is on fire. Although some countries have ramped up their own emissions cuts targets the UK now officially aims for net zero emissions by 2050 public concern and frustration has kept growing. More and more members of the public have taken extreme steps to call for urgent action on climate, with Extinction Rebellion protesters being arrested en masse and controversially keeping the issue in the news.

What if we dont turn things around quickly? Major impacts are already baked in, with glaciers melting worldwide, Greenland losing ice rapidly, and heatwaves and fires happening more often and more severely. Heavy rainfall is increasing, as is drought in some places. We already need to live with a different climate. Although some campaigners rhetoric is not scientific (we dont seriously expect 6 billion deaths), unchecked warming would still expose tens or even hundreds of millions to extreme heat stress conditions and flooding from the sea. The worst can still be avoided, but the longer we keep heating the planet, the harder it gets. Prof Richard Betts, climate scientist, University of Exeter and Met Office Hadley Centre

Right now, on top of the world, a ship is frozen into the Arctic sea ice at the north pole. The RV Polarstern is pirouetting on the spot in the dark, with no prospect of sunlight for months to come. This region of Earth, the Arctic Ocean, is still one of the most remote and inaccessible places on our planet. We know very little about what happens here during the polar night, when temperatures can easily drop to -30C, and thick sea ice crunches and bends in the darkness. But this region is critical to Earths climate, and its essential to understand more.

The Polarstern is here on a once-in-for-ever opportunity to fill that data gap. Its the centrepiece of the Mosaic expedition, probably the biggest polar expedition that will ever be launched. Twenty years from now, it may not be possible to do this: to freeze into the sea ice for an entire year and to drift inside this vast cold environment to watch and learn from the inside. Its taken 20 years to organise, and over the year of the expedition (September 2019 to September 2020), 600 scientists will rotate on and off the ship, supported by many more in research institutes around the world. There is no question that the data being gathered now will drive a revolution in our understanding of the north pole and our climate, and every one of us will be affected by what they find. Helen Czerski, physicist and oceanographer

The laws of physics can be expressed in a handful of compact equations, but their reach is simply breathtaking. A stunning demonstration of this came with our first picture of a black hole, captured by the Event Horizon Telescope.

The black hole is situated at the centre of the Messier 87 galaxy, 54m light years away. The glowing ring of radiation, emitted by tortured matter spinning through warped space-time, is already iconic. The matter is plunging towards an event horizon as big as the solar system, containing the mass of 6.5bn suns.

Remarkable aspects of the image abound. The theory of general relativity, published by Einstein a hundred years ago, predicted the existence and features of this beast remarkably well. The cataclysmic whirlpool may be beyond our imagination, but it is not beyond our mathematics.

The singularity predicted at the heart of a black hole is a different story though. That is where quantum mechanics and relativity conflict and break down. We would love to know what answers lie there, and we will be scouring the image for any clues.

Finally, the global collaboration necessary to capture the image shows we can work together on a worldwide scale to a common goal something that our science tells us we need to do more of, if we want to survive as a species and continue our exploration of the amazing universe in which we find ourselves. Jon Butterworth, professor of physics, University College London

Ebola in 2019 highlights both the achievements of scientific progress and the persistent deep-rooted challenges of improving health in the most difficult settings. The 2014 Ebola outbreak in west Africa killed more than 11,000 people and alerted the world to its dangers. It highlighted the rich worlds neglect of research and development for infectious diseases that only rarely present a global risk.

At the end of 2019 we have a licensed Ebola vaccine, another hopefully soon to be licensed, and two effective therapeutic drugs. We can now treat those infected as well as prevent spread of the disease, allowing us to move from reactive containment to primary prevention, as a result of large and innovative collaborations that demonstrate how to help develop new and effective technologies, now and in the future.

But the current outbreak in eastern Democratic Republic of Congo has lasted more than 15 months and killed more than 2,000 people. Treatment and control continues to be hugely difficult in a setting characterised by political, economic and social fragility. Effective vaccines and drugs are part of what is needed for epidemic prevention, but we must continue to strive to address the fundamental causes of ill health in these settings. Anne Mills, professor of health economics and policy, London School of Hygiene & Tropical Medicine

Remember that time when we were young, and we had those nice neat family trees about human evolution? Lucy, Homo erectus, skip a few, Neanderthals and finally us? That whole scheme has been thoroughly binned in the last decade, with more fossil discoveries and the addition of ancient DNA to the armoury. We now have less confidence in the relationships between many more members of the human family, apart from the ones whose legacy we can see in our own DNA our Neanderthal and Denisovan ancestors. Others will be found soon enough.

We remain an African species. Homo sapiens evolved in multiple places in Africa, and a few thousand left some 70,000 years ago to populate the rest of the world. But now we know that there were earlier diasporas from the motherland. This year, we discovered that we had made it all the way to Greece. Embedded in the roof of a cave in the southern Peloponnese, two crushed skulls were found by Katerina Harvati and her team, one a sprightly 170,000-year-old Neanderthal, but the other is us, Homo sapiens, and is more than 210,000 years old. This is far older and much farther afield than we had previously found. The revolution in the story of how we got here shows no signs of calming down. Dr Adam Rutherford is a geneticist and author. His book How to Argue With a Racist is out in February (W&N, 12.99)

Although announced in November 2018, the shock waves from the announcement by the Chinese scientist He Jiankui that two girls had been born from embryos that were genetically modified using genome editing have been reverberating throughout 2019, and will no doubt continue to do so for years to come. This was a misguided and badly conducted attempt to make children resistant to infection by HIV, the virus that causes Aids, by mutating the CCR5 gene, which encodes a protein expressed on the surface of white blood cells that the virus uses to gain entry. He showed disregard for normal scientific and clinical practice, ignored risks to the children born and potentially to subsequent generations.

But the work made the prospect of altering our genetic makeup more immediate rather than theoretical. It also raised concerns about where to draw the line with the possibility of not just avoiding genetic and perhaps infectious disease, but ultimately carrying out forms of enhancement. However, it had the positive benefit of stimulating debate worldwide and it has led to the launch of international efforts, notably a science academies panel to judge the science, clinical need, and the conditions that would have to be met for germline (potentially heritable) genome editing to be carried out; and a WHO-appointed committee to develop a framework of governance that can be adopted to control the use of the genome editing methods in treating or avoiding disease. Both of these efforts will report next year.

Meanwhile, the science of genome editing and its application in both the field and clinic are progressing rapidly. A novel and ingenious new method termed prime editing, published by David Liu and colleagues, can efficiently make precise, small changes in DNA, without the problems associated with earlier methods, such as those used by He Jiankui. Given that about 85% of disease-causing mutations in humans could, in theory, be corrected by prime editing, it clearly offers great promise. Ways to make animals and plants resistant to disease and to allow plants to cope with climate change have been developed using genome editing, and this year we have seen a huge jump in the number of clinical trials using the methods to treat patients with genetic diseases (somatic or non-heritable gene therapy), including cancers, blindness and sickle cell disease. Robin Lovell-Badge, group leader, the Francis Crick Institute, London

The rechargeable lithium-ion battery has helped power the global revolution in portable electronics, and, indeed, many of you will be reading this article on a mobile phone, laptop or tablet computer. In October this year, the three pioneers of the lithium-ion battery, John Goodenough from the University of Texas at Austin, Stan Whittingham from Binghamton University, New York, and Akira Yoshino from Japans Meijo University, were awarded the Nobel prize in chemistry.

For me, this award was long overdue and finally recognised an exciting area of materials chemistry. There are lots of reasons to welcome this news. For the sheer beauty of literally holding the result of their fundamental research in our hands. For the celebration of John Goodenough, who at 97 is the oldest person ever awarded any Nobel prize. For the fact that new materials lie at the heart of developing green technologies that can change the way we live and work. For spurring further development of better batteries for electric vehicles and for storing energy from wind and solar.

Perhaps most of all because it helps to shine a light on one of the most urgent challenges of our time: a low-carbon future to deal with climate change. Saiful Islam, professor of materials chemistry at the University of Bath

Menabe, a dry forest in western Madagascar, is on fire. The only habitat of the worlds smallest primate (Berthes mouse lemur) is going up in flames as hungry people, many escaping droughts in the south, clear land for agriculture (despite the area being officially protected). Worryingly, we learned in October that this is far from an isolated problem and protected areas are less effective than previously thought.

Using a global data set of population density, night-time light and agriculture, researchers compared the changes in human pressures over time in more than 12,000 protected areas with similar unprotected areas. On average, pressures have increased faster inside than outside protected areas and those in poorer countries are particularly likely to suffer higher pressures. A decade ago, governments agreed a target to increase the proportion of the globe under conservation by 2020. Next year they gather to review progress and, potentially, commit to new targets.

The evidence is clear; when it comes to protecting sites for conservation, quality matters. Designating protected areas without effective management (including support for local communities) wont stop the fires, hold back the expansions of farms, or, ultimately, protect species from extinction. Julia Jones, professor in conservation science, Bangor University

Games of hide-and-seek are among my favourite childhood memories, and I still case novel environments for good hiding places. Researchers in Germany studied how rats can learn to play hide-and-seek with humans. All the rats learned to look for the hiding experimenter and all but one learned to hide from her. The only reward the rats received was the experimenter tickling and playing with them.

The data suggests that the rats were enthusiastically engaging in the game, looking frantically for the experimenter, squeaking and executing Freudensprnge (joy jumps) when they found her. They seemed to understand what it means to hide, preferring opaque boxes rather than clear boxes, and remaining silent (no squeals) until found. Frequently when they were found, they would tease the experimenter by running away and hiding again.

Its worth bearing in mind how complex this hide-and-seek is involving changes in role (hider or seeker) and theory of mind, and its almost alarming how well these rats learned to do this, all in the absence of classic psychological rewards like food. The experimenters conclude that the rats learn to play this game for the sheer joy of playing the game, and this is disconcertingly similar to the way human children play. Sophie Scott, professor of cognitive neuroscience, University College London

Once, during an ill-judged holiday in Borneo, I tried to climb a small mountain. While I crawled, panted and coughed on the slopes, my Malaysian guide, Miki, shuffled around politely, hands in his pockets, playing football with small rocks to slow his pace enough to match mine. Unacclimatised to altitude, it took me a day and a half to get to the summit. When I asked Miki how long it would normally take him to do the same he told me that, unencumbered by tourists like me, he could run up and down the mountain in just over three hours.

The molecular mechanisms that underpin Mikis apparently superhuman adaptation to high-altitude life revolve around a family of proteins known as hypoxia-inducible factors (HIFs). These substances trigger alterations in a host of genes, which together help regulate oxygen levels in the human body. Their discovery helped explain how oxygen levels could be sensed and gave scientists insight into the mechanisms that allow the body to adapt and survive when demand for oxygen greatly outstrips supply.

This year Sir Peter Ratcliffe, Gregg Semenza and William Kaelin shared a Nobel prize for their part in unpicking that mystery.

Their work informs more than ill-advised summit attempts. HIFs and the regulation of oxygen levels are together central to almost all aspects of human life, whether in health or disease. The work has already been applied to develop drugs to treat anaemia and may one day lead to new treatments for stroke, spinal cord injury, chronic inflammation and even cancer. Prof Kevin Fong is a consultant anaesthetist at University College London Hospital

My choice is a venture that failed but was a heroic failure: the Israeli effort to land a small robotic vehicle on the moon. This project, named Beresheet (Hebrew for in the beginning), was supported by private and philanthropic funding. It attracted wide interest among the young, and showed what can be achieved with hi-tech ingenuity. To minimise the weight of fuel, it didnt follow a direct track but was boosted into successively higher orbits around the Earth until it was captured by the moons gravity. It was launched on 22 February and was planned to soft-land on 10 April. But a gyroscope malfunctioned; the retro-jets didnt ignite soon enough, and it crash-landed. I highlight Beresheet because its a precursor of a new style of space ventures small scale, privately funded, and genuinely involving the public. (Indeed, Beresheet carried, as a school project, hundreds of tardigrades, microscopic water bears, which may have survived the impact.) Were moving beyond an era when all space projects must involve national agencies or large commercial conglomerates.

Groups from many nations will be able to launch follow-ups similar in concept to Beresheet. Sophisticated, privately funded miniaturised probes will gather data about the moon as well as the Earth. Some may go deeper into space, using advanced robotics, and the sophisticated electronics developed for smartphones. There will still be scope for big projects maybe even some carrying humans. But space will become an arena for independent experimenters even hobbyists. Martin Rees, Astronomer Royal

There have been so many significant science stories in 2019 that I have been spoilt for choice. I could have gone with the climate crisis (David Attenboroughs speech at Davos or Greta Thunbergs at the UN); or maybe the announcement of the first image of a black hole by scientists on the Event Horizon Telescope project, which seems to have already achieved iconic status. Or I could have chosen Googles recent announcement that they had achieved quantum supremacy with their new quantum computer. But instead, Ive gone with quirky rather than significant. In May, it was announced that the international SI units of measurement had been redefined. For example, the kilogram will no longer be compared with a cylinder of metal sitting under a bell jar outside Paris. Instead check this out it can be fixed just by knowing the frequency of vibration of an atom of caesium. That frequency defines the length of a second, which together with the speed of light defines the length of a metre, which in turn, together with knowing Plancks constant of quantum theory, allows us to calculate what a kilogram is. It is so utterly cool, but probably only fascinating to geeky physicists like me. Still, I make no apology. Jim Al-Khalili, professor of physics and public engagement in science at the University of Surrey and presenter of The Life Scientific (BBC Radio 4)

See the article here:
The science stories that shaped 2019 - The Guardian

Charles River Labs is stumping up $380 million in cash to buy out cell therapy biomaterials producer HemaCare.

This will boost Charles Rivers work in cell therapy by adding HemaCares ability to produce human-derived cellular products for this growing market.

It supplies biomaterials, including human primary cell types, and cell processing services to support the discovery, development and manufacture of cell therapies, including allogeneic (donor-derived cells) and autologous (patient-derived cells) programs.

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This builds on the hope and hype around cell therapies like CAR-T and their capacity to hit back at certain oncology targets, namely blood cancers.

The CRO said that the deal will create a unique, comprehensive solution for cell therapy developers and manufacturers worldwide to help accelerate their critical programs from basic research and proof-of-concept to regulatory approval and commercialization.

James Foster, chairman, president and CEO Charles River, said: Cell and gene therapies are important new modalities, with an estimated 10 to 20 new product approvals per year within five years. In order to continue to enhance our ability to support our clients research efforts, particularly in biologics discovery and development, we are expanding our scientific capabilities in this emerging, high-growth market with the acquisition of HemaCare.

The addition of HemaCares innovative cell therapy products and services to our integrated, early-stage solutions will create a unique, go-to partner for clients to work with Charles River across a comprehensive cell therapy portfolio from idea to novel therapeutic.

RELATED: Charles River swoops on early-stage CRO Citoxlab

Pete van der Wal, president and CEO of HemaCare, added: We are very pleased to be joining the Charles River team, which is widely recognized as the industry-leading, early-stage contract research organization. Partnering with Charles River will strengthen the value proposition for our clients, enabling them to work seamlessly with one scientific partner to enhance the speed and efficiency with which they can advance their cell therapies. The transaction will offer compelling value to our shareholders. This is an exciting day that will usher in a new era for HemaCare and my talented colleagues.

Cell therapy is becoming a focus for biopharma, but the relatively new area requires cutting-edge tech to help nurture new research like CAR-T into an established market.

Charles River says its work in the area is currently making around $100 million a year, but it sees the addressable market for HemaCares products is expected to increase from approximately $200 million today to nearly $2 billion in 10 years and wants to be a part of that.

While spending nearly $400 million in cash (the company had a market cap of $257 million at the end of play last week, with Charles River paying a 33% premium), HemaCare is expected to immediately drive profitable revenue growth, with estimated revenue growth of at least 30% annually over the next five years, the CRO estimated.

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Charles River Labs snaps up HemaCare, eyeing the growing cell therapy market - FierceBiotech

Shares of Axovant Gene Therapies Ltd (NASDAQ:AXGT) have received a consensus rating of Buy from the eleven research firms that are currently covering the firm, Marketbeat Ratings reports. Two equities research analysts have rated the stock with a hold recommendation, eight have assigned a buy recommendation and one has issued a strong buy recommendation on the company. The average 1 year target price among analysts that have issued ratings on the stock in the last year is $24.72.

A number of equities analysts have issued reports on the company. Zacks Investment Research upgraded Axovant Gene Therapies from a hold rating to a strong-buy rating and set a $6.00 price target on the stock in a research report on Wednesday, November 13th. Chardan Capital raised their price objective on Axovant Gene Therapies from $10.00 to $15.00 and gave the company a buy rating in a report on Monday, October 28th.

Several hedge funds have recently added to or reduced their stakes in AXGT. BlackRock Inc. acquired a new stake in shares of Axovant Gene Therapies in the second quarter worth approximately $1,482,000. Tower Research Capital LLC TRC lifted its holdings in Axovant Gene Therapies by 955.3% in the 2nd quarter. Tower Research Capital LLC TRC now owns 4,221 shares of the companys stock valued at $27,000 after purchasing an additional 3,821 shares in the last quarter. Jane Street Group LLC lifted its holdings in Axovant Gene Therapies by 28.8% in the 2nd quarter. Jane Street Group LLC now owns 46,455 shares of the companys stock valued at $289,000 after purchasing an additional 10,375 shares in the last quarter. Finally, Barclays PLC acquired a new stake in Axovant Gene Therapies during the 3rd quarter worth $65,000. 14.80% of the stock is owned by institutional investors and hedge funds.

Shares of Axovant Gene Therapies stock traded up $0.05 during trading hours on Tuesday, reaching $5.26. The stock had a trading volume of 610,255 shares, compared to its average volume of 197,557. The stocks 50 day moving average is $5.42 and its 200 day moving average is $6.21. The company has a current ratio of 1.41, a quick ratio of 1.41 and a debt-to-equity ratio of 0.69. Axovant Gene Therapies has a twelve month low of $3.81 and a twelve month high of $19.60.

Axovant Gene Therapies (NASDAQ:AXGT) last posted its earnings results on Friday, November 8th. The company reported ($0.61) EPS for the quarter, beating the Zacks consensus estimate of ($1.15) by $0.54. As a group, equities analysts predict that Axovant Gene Therapies will post -3.56 earnings per share for the current fiscal year.

Axovant Gene Therapies Company Profile

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

Further Reading: How to build a Fibonacci channel

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Axovant Gene Therapies Ltd (NASDAQ:AXGT) Given Consensus Recommendation of Buy by Brokerages - Riverton Roll

Neveah Taouk, 4

At last, when Mary was seven and Neveah three, new developments in whole-genome sequencing enabled specialists to identify the disorder. The diagnosis gave the Taouks information but not hope. They knew what the problem was, but there was no treatment and no cure.

Desperate, Charlie contacted specialists around the world. I must have spoken to at least fifty people scientists, doctors, professors, he says. Most of them had never heard of the condition.

His search eventually led to Dr Wendy Gold, a specialist in rare genetic disorders in children, based at the University of Sydney and the Childrens Hospital at Westmead. We arranged to talk, says Charlie. To be honest, I wasnt expecting much. But then she said, Have you heard of gene therapy?

Gene therapy is a new and rapidly evolving field of research. One of the therapys forms involves adding new genes to a patients cells to replace missing or malfunctioning genes. The new genes are typically delivered to the appropriate cells in the body using a benign virus as a carrier. Gene therapy is already being used to treat diseases including spinal muscular atrophy. It could also be a promising treatment for Parkinsons disease. Dr Gold believed there was a chance it could help the Taouk girls.

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The gene therapy research that could save a family of four - News - The University of Sydney

A new method allows researchers to develop adeno-associated virus (AVV) commonly used as the vehicle for gene therapies that accurately target and deliver genes to specific cells in the body.

This new technology may be suitable to target dopaminergic neurons that are damaged in Parkinsons disease.

We believe that the new synthetic [lab-made] virus we succeeded in creating would be very well suited for gene therapy for Parkinsons disease, for example, and we have high hopes that these virus vectors will be able to be put into clinical use, Tomas Bjrklund, PhD,Lund University, Sweden, said in a press release.

Bjrklund is lead author of the studyA systematic capsid evolution approach performed in vivo for the design of AAV vectors with tailored properties and tropism, which was published in the journal Proceedings of the National Academy of Sciences.

The adeno-associated virus (AAV)is a common, naturally-occurring virus, which has been shown to work as an effective gene therapy delivery vehicle for genetic diseases, such asspinal muscular atrophy. In gene therapy, scientists deliver a working version of a faulty gene using a harmless AAV that was modified and inactivated in the lab. This way the virus functions only as a delivery vehicle and does not have the capacity to damage tissues and cause disease.

While AAVs have a natural ability to penetrate any cell of the body and infect as many cells as possible, their usefulness as a potential therapy requires the capacity to specifically deliver a working gene to a particular cell type, such as dopamine producing-nerve cells. Those are the ones hose responsible for releasing the neurotransmitter dopamine and that are gradually lost during Parkinsons disease.

A team of Swedish researchers have developed a new method called barcoded rational AAV vector evolution, or BRAVE that combines powerful computational analysis with the latest gene and sequencing technology to produce AAVs that can specifically target neurons.

To make AAVs neuron specific, the team selected 131 proteins known to specifically interact with synapses (the junctions between two nerve cells that allow them to communicate).

They then divided the proteins into small sequences, called peptides, and created a large library where each peptide could be identified by a specific pool of genetic barcodes (a short sequence of DNA that is unique and easily identified).

The peptide is then displayed on the surface of the AAV capsid, allowing researchers to test the simultaneous delivery of many cell-specific AAVs in a single experiment.

The team then injected these AAVs into the forebrain of adult rats and observed that around 13% of the peptides successfully homed to the brain. Moreover, 4% of the peptides were transported effectively through axons (long neuronal projections that conduct electrical impulses) toward the nerve cells body.

Researchers then selected 23 of these unique AAV capsids and injected them into rats striatum, a brain region involved in voluntary movement control and affected in Parkinsons disease. Twenty-one of the new AAV capsids had an improved transport capacity within nerve cells than in standard AAVs.

One particular capsid, called MNM008, showed a high affinity for rat dopaminergic neurons. Researchers then tested whether this viral vector also could target human dopaminergic neurons.

The team transplanted neurons generated from human embryonic stem cells into rats striatum. Six months later, they injected either MNM008 or a control AAV capsid and found that MNM008 was able to target these specific cells and be transported into dopaminergic neuronal cell bodies through axons.

Thanks to this technology, we can study millions of new virus variants in cell culture and animal models simultaneously. From this, we can subsequently create a computer simulation that constructs the most suitable virus shell for the chosen application in this case, the dopamine-producing nerve cells for the treatment of Parkinsons disease, Bjrklund said.

Overall, researchers believe the BRAVE method opens up the design and development of synthetic AAV vectors expressing capsid structures with unique properties and broad potential for clinical applications and brain connectivity studies.

The team has established a collaboration with a biotech company, Dyno Therapeutics, to use the BRAVE method in the design of new AAVs.

Together with researchers at Harvard University, we have established a new biotechnology company in Boston, Dyno Therapeutics, to further develop the virus engineering technology, using artificial intelligence, for future treatments, Bjrklund said.

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.

Total Posts: 208

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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New Gene Therapy Method May Open BRAVE New World in Parkinson's - Parkinson's News Today

In January 2019, then-FDA commissioner Scott Gottlieb ushered in the new year with a bold prediction: The agency, he said, would be approving between 10 and 20 gene and cell therapies per year by 2025. At the time, there were a whopping 800 such therapies in the biopharma pipeline and the FDA was aiming to hire 50 new clinical reviewers to handle the development of the products.

That momentum will no doubt start to pick up in 2020, as several companies in late-stage development of their gene and cell therapies achieve key milestones or FDA approval. Among the companies expected to make major strides in gene and cell therapies next year are Biomarin, with valoctocogene roxaparvovec to treat hemophilia A, Sarepta and its gene therapy for Duchenne muscular dystrophy, plus multiple players developing CAR-T treatments for cancer, including Bristol-Myers Squibb and Gilead.

But with such explosive growth comes challenges. Gene and cell therapies require enormous up-front investing in complex manufacturing processes, as well asinnovative approaches to securing insurance coverage for products that come with eye-popping price tagssuch as Novartis $2 million gene therapy Zolgensma to treat spinal muscular atrophy. Those are just a few of the obstacles that will be front-and-center in 2020 as more gene and cell therapies make their way towardthe finish line.

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Pharma companies will face challenges figuring out how to incorporate gene and cell therapies into their overall business, said Michael Choy, partner and managing director at Boston Consulting Group, in an interview with FiercePharma. They dont fit well into the normal paradigms of budgeting and decision-making. They require a different pace of evolution and specialized expertise. For now, companies are shoe-horning gene therapies into their current model, but over the long-term there will have to be changes.

That will become increasingly clear in 2020 as both Big Pharma and small up-and-comers move towardthe clinic with their gene and cell therapies. John Zaia, M.D., director of the Center for Gene Therapy at City of Hope, predicts there will be at least three gene and cell therapy FDA approvals in 2020. He also expects to see momentum among companies seeking to improve on the technology to address unmet needs in medicine.

For example, Zaia believes off-the-shelf CAR-T cancer treatments will show promise in early studiesand will be met with enthusiasm in the cancer community, he told FiercePharma in an email. The first generation of FDA-approved CAR-T treatments, Novartis Kymriah and Gileads Yescarta, take several weeks to make because they require removing T cells from patients and engineering them to recognize and attack the patients'cancers. Several companies are advancing off-the-shelf CAR-T treatments, including Precision BioSciences, which has been building out a manufacturing plant equipped to make 10,000 doses per year.

RELATED: Biotech building facility to make genome-edited, off-the-shelf CAR-T therapies

Gene therapies for inherited diseases will make strides in 2020, too, Zaia predicts. City of Hope is one of the participants in a phase 1 study of CSL Behrings gene therapy to treat adults with sickle cell disease. CSL will be racing against several companies working on the disease, including Bluebird Bio, which is testing its beta thalassemia gene therapy Zynteglo in sickle cell. There is a big push from many research centers to cure sickle cell diseaseand early results with the use of gene therapy look very promising, Zaia said. Years of research is finally coming to realization.

With such robust R&D underway in gene and cell therapies, its no surprise several players are stepping up their investments in manufacturing. In October, Sanofi said it would retrofit a vaccine plant in France so it couldbe used for gene therapy manufacturing. Pfizer shelled out $19 million for a North Carolina facility that will serve as its manufacturing hub for gene therapies. Even Harvard University is getting into the game, working with a consortium of contract manufacturers to build a $50 million facility dedicated to making cell therapies and viral vectors for gene therapies.

But how will the healthcare system pay for all of these complex therapies? Its a question that will continue to dog the industry, BCGs Choy said. Theres a lot of interest in outcomes-based payments and payments over time, but the issue is theyre very difficult to implementbecause the infrastructure to track outcomes over time doesnt really exist, he said.

Still, payers and pharma companies are hinting at their willingness to put that infrastructure in place. Pfizer, which is developing DMD and hemophilia gene therapies, said recently its brainstorming with payers on innovative strategies for reimbursement. Novartis and Spark have already pioneered payment strategies that deviate from the standard pay-everything-up-front system. Novartis has some pay-for-performance contracts in place for the $475,000 Kymriah. And in September, Cigna agreed to cover Novartis Zolgensma and Sparks Luxturna on a per-month, per-member schedule.

RELATED: Novartis, Spark gene therapies win a boost with soup-to-nuts Cigna coverage

Despite the many challenges in cell and gene therapy, some players are showing theres likely to be a robust market for these innovative treatments. In its first quarter on the market, Zolgensma brought in $160 million in salesfar surpassing analysts expectations.

The promise of huge returns on gene and cell therapies will likely drive acquisitions in 2020, Choy predicted. These treatments are so transformative for patients, and as the clinical proof of effectiveness continues to grow, youre going to see a lot more deal-making in this area, he said.

Buyers will likely show a willingness to invest in early-stage gene and cell therapies, especially if they come with technology platforms that allow for the development of many follow-up products, Choy added. For these types of therapies, the lifecycles will be much shorter than they are for traditional pharmaceuticals, particularly for rare diseases, he said. If you administer a one-time therapy, that revenue peaks quite quickly and then drops off. So to have a sustainable revenue from a gene therapy business, you need to replace that, which requires managing a pipeline.

Judging from recent events in the burgeoning gene and cell therapy industry, the news flow in 2020 will be generated not just by the industrys largest players, but also by its upstarts. In December, Ferring Pharmaceuticals spinout FerGene turned heads with data showing that its gene therapy to treat non-muscle invasive bladder cancer eliminated tumors in more than half of participants in a phase 3 trial. And Gileads Kite Pharma just applied for FDA approval for its mantle cell lymphoma CAR-T, KTE-X19, based on a 93% overall response rate in a phase 2 trial.

There were 75 gene therapy clinical trials initiated in 2018, nearly doubling the trial starts of 2016momentum thats likely to continue next year, BCG said in a recent report. The scientific foundation is in place, BCG analysts concluded, but there is still much to do to deliver the full benefit of gene therapy to patients."

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Pharma's gene and cell therapy ambitions will kick into high gear in 2020despite some major hurdles - FiercePharma

Drug Target Review lists its 10 most popular news stories from 2019, summarising the drug targets that you wanted to read about.

Drug Target Review has published a wide range of news stories this year, from the identification of novel drug targets to improvements in toxicology studies and developments in screening.

As the year draws to a close, we reflect on the biggest and most popular stories from 2019. To read the full pieces, click on the title of each news story.

A genetic analysis study revealed that variants of hundreds of genes work together in contributing to the development of Tourettes syndrome, in our tenth most popular story this year.

According to the researchers, from the Massachusetts General Hospital (MGH) and collaborators, their findings confirm that the underlying basis for Tourettes syndrome is polygenic, meaning that hundreds of small DNA changes cause the condition, rather than one inactive gene.

The scientists said their next step is to expand their sample size to around 12,000 patients, made possible with a potential international collaboration.

The study was published in the American Journal of Psychiatry.

A group of researchers identified new genetic targets on which BRCA2-driven cancer cells are dependent upon, providing a potential avenue for drug development.

The study, conducted at Brigham and Womens Hospital, used CRISPR and short-hairpin RNAs (shRNAs) to test 380 genes with a known or suspected role in DNA-damage response. This allowed the team to narrow in on the most promising genes: APEX2 and FEN1, two novel targets for breast cancer.

The results were published in Molecular Cell.

Immunotherapy treatment could reduce the persistence of HIV in patients receiving triple therapy, found a group of researchers.

The researchers, from the University of Montreal Hospital Research Centre, discovered that these therapies expose the virus to the immune system. Three proteins PD-1, LAG-3 and TIGIT were uncovered by the scientists as frequently expressed on the surface of HIV-hiding cells; these proteins are also cancer targets.

According to the team, their study could lead to the development of new HIV therapies based on cancer immunotherapies.

The study was published in Nature Communications.

Researchers at the Indiana University School of Medicine developed a blood test to measure pain and improve diagnosis. The team analysed hundreds of patient samples to reveal biomarkers in their blood, which could be used as a scale to determine pain.

According to the researchers, the biomarkers act like a signature that can be matched against a prescription database. This could allow medical professionals to select the appropriate compound and reduce pain for the patient.

The study was published in Molecular Psychiatry.

A team of scientists revealed that immune cells could be key in causing endometriosis, a pelvic pain experienced by women, through an investigation into macrophages. The study was led by researchers from Warwick Medical School and the University of Warwick.

Macrophages can adapt their function according to local signals from their surroundings and so become modified by disease. This led the researchers to add modified macrophages to a cell culture, which resulted in the production of higher levels of insulin-like growth factor-1 (IGF-1).

The team conclude that macrophages therefore present a drug target for endometriosis.

The results can be found in The FASEB Journal.

Scientists from the University of Pennsylvania imaged a molecule that induces inflammation and leads to lupus, in our fifth most popular story of 2019. The researchers discovered that the molecule is comprised of two sections: SHMT2 and BRISC, a cluster of proteins. When these two sections bind to each other, they cause inflammation.

When mice models lacking BRISC were tested, they were resistant to lupus. This led the team to conclude that a molecule which blocks BRISC and SHMT2 could be a drug target for lupus.

The findings were published in Nature.

A team of researchers reported that a CRISPR-Cas9 gene therapy which specifically reduces fat tissue and obesity-related metabolic disease was successful in mice.

The scientists, from Hanyang University, argue that their technique could be used as a way to combat type 2 diabetes and other obesity-related diseases.

Targeting Fabp4, a fatty acid metabolism gene, the researchers observed a 20 percent reduction of body weight in obese mice. It also resulted in improved insulin resistance after only six weeks of treatment.

The findings were published in Genome Research.

A compound that promotes the rebuilding of the protective sheath around nerve cells has been developed by researchers at the Oregon Health & Science University (OHSU).

The team found that the S3 compound reverses the effect of hyaluronic acid (HA) in mice. HA has been found to accumulate in the brain of patients with multiple sclerosis, and accumulation of HA

has also been linked to maturity failure of cells called oligodendrocytes, which generate myelin, the protective layer of axons.

The team therefore believe that the S3 compound could provide a therapeutic strategy for treating nervous system disorders.

The study can be found in Glia.

A group of researchers formed a complex view of the functional dysbiosis in the gut microbiome during inflammatory bowel disease (IBD), to reveal new targets for treatments.

The scientists, from theBroad InstituteofMITandHarvard University, observed microbial changes and human gene regulatory shifts from stool and blood samples of patients.

This multi-omic study enabled the team to discover that during periods of disease activity, IBD patients had higher levels of polyunsaturated fatty acids in both the blood and stool. They also identified other varying levels of nutrients and vitamins, presenting several potential drug targets.

The findings were published in Nature.

In our most popular news piece this year, researchers found that the small molecule PJ34 reduces the number of human pancreatic cancer cells in transplanted tumours by 90 percent.

The team, from Tel Aviv University, built on previous research to treat xenografts with their small molecule. It is permeable in the cell membrane, but affects human cancer cells exclusively, making it an attractive compound for development.

The scientists found that PJ34 causes a rapid cell death and in one mouse, the tumour completely disappeared. They concluded that the molecule could be a potent therapeutic against pancreatic cancer.

The results were published in Oncotarget.

Related organisationsBrigham and Women's Hospital, Hanyang University, Harvard University, Indiana University School of Medicine, Massachusetts General Hospital (MGH), MIT, Oregon Health & Science University (OHSU), Pennsylvania University, Tel Aviv University, University of Montreal Hospital Research Centre, Warwick Medical School, Warwick University

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DTR's news round-up 2019: the stories that defined the year - Drug Target Review

UMass Medical School and GE Healthcare Life Sciences have announced the companies plan to establish a new large-scale viral vector manufacturing facility that will be housed on the Worcester campus of the medical school.

The facility will be able to provide large quantities of high-quality recombinant adeno-associated virus vectors for preclinical research, according to a news release from the medical school.

The potential of gene therapy to treat human disease has finally become a reality, said Terence R. Flotte, the Celia and Isaac Haidak Professor of Medical Education, executive deputy chancellor, provost and dean of the School of Medicine and professor of pediatrics. However, the ability to move the field forward to treat additional serious diseases remains limited by the efficiency and flexibility of producing gene therapy vectors suitable for testing in new disease models."

A lack of large-scale vector manufacturing facilities has limited preclinical research capabilities, according to the news release.

Researchers often wait 12 to 24 months to secure enough vector for their research. With this facility, researchers will have access to GE Healthcares processing equipment, helping get research to the clinic faster, the medical school said.

Accelerating research that brings novel cell and gene therapies to patients is the mission of our business, said Catarina Flyborg, the general manager of cell and gene therapy at GE Healthcare Life Sciences. By partnering with UMass Medical School to create this large scale AAV manufacturing facility, we will provide researchers with the tools and AAV needed for pre-clinical research that will advance the cell and gene therapy industry and get therapies to patients faster.

The facility will be 3,220 square feet and will be fully operational in 2020. Four to six professional staff members will manage day-to-day operations, with Sylvain Cecchini, an associate professor of microbiology and physiological systems, as the core director, the statement said.

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UMass Medical School and GE Healthcare establishing manufacturing facility in Worcester - MassLive.com

WASHINGTON, Dec. 20, 2019 (GLOBE NEWSWIRE) -- via NEWMEDIAWIRE -- The Alliance for Regenerative Medicine (ARM), the international advocacy organization for the cell and gene therapy and broader regenerative medicine sector, today released the initial slate of presenting companies at the 2020 Cell & Gene Meeting on the Mediterranean. The event will be held April 15-17, 2020 in Barcelona, Spain.

The event, modeled after ARMs highly successful Cell & Gene Meeting on the Mesa, is expected to attract more than 500 attendees, including senior executives from leading cell therapy, gene therapy, and tissue engineering companies worldwide, large pharma and biotech representatives, institutional investors, academic research institutions, patient foundations, disease philanthropies, and members of the life science media community.

The second annual Cell & Gene Meeting on the Mediterranean will feature presentations by 50+ leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies.

The initial slate of 2020 presenting companies includes: Adaptimmune, AGTC, Ambys Medicines, American Gene Technologies, AskBio, Aspect Biosystems, Atara, Autolus Therapeutics, Avectas, AVROBIO, Axovant Gene Therapies, bluebird bio, Bone Therapeutics, Caribou Biosciences, Celavie Biosciences, Cellatoz Therapeutics, CEVEC, Cynata Therapeutics, Flexion Therapeutics, Fraunhofer IZI, GenSight Biologics, Healios, Iovance Biotherapeutics, Kiadis Pharma, Kytopen, LogicBio Therapeutics, MeiraGTx, Minerva Biotechnologies, MolMed, Novadip Biosciences, Orchard Therapeutics, Oxford Biomedica, PDC*line Pharma, Precision BioSciences, Promethera Biosciences, PTC Therapeutics, Recombinetics, REGENXBIO, ReNeuron, Rexgenero, Sangamo, SmartPharm Therapeutics, Standards Coordinating Body for Regenerative Medicine, Theradaptive, ThermoGenesis, Tmunity Therapeutics, Ultragenyx Pharmaceutical, VERIGRAFT, and Zelluna Immunotherapy.

Additional event details will be updated regularly on the conference website http://www.meetingonthemed.com.

Registration is complimentary for investors and credentialed members of the media. To learn more and to register, please visitwww.meetingonthemed.com. For members of the media interested in attending, please contact Kaitlyn Donaldson Dupont at kdonaldson@alliancerm.org.

For interested organizations looking to increase exposure to this fields top decision-makers via sponsorship, please contact Kelly McWhinney at kmcwhinney@alliancerm.org for additional information.

About The Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 350 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visithttp://www.alliancerm.org.

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The Alliance for Regenerative Medicine Releases Initial Slate of Presenting Companies at the 2020 Cell & Gene Meeting on the Mediterranean -...

In a recent study published by Prophecy Market Insights, titled, Global Personalized Gene Therapy Treatment Market Research Report, analysts offers an in-depth analysis of global Personalized Gene Therapy Treatment market. The study analyses the various aspect of the market by studying its historic and forecast data. The research report provides Porters five force model, SWOT analysis, and PESTEL analysis of the Personalized Gene Therapy Treatment market. The different areas covered in the report are Personalized Gene Therapy Treatment market size, drivers and restrains, segment analysis, geographic outlook, major manufacturers in the market, and competitive landscape.

Key Players of Personalized Gene Therapy Treatment Market:

Amgen, Inc., Chengdu Shi Endor Biological Engineering Technology Co., Ltd., SynerGene Therapeutics, Inc., Cold Genesys, Inc., Bellicum Pharmaceuticals, Inc., Takara Bio, Inc.,Ziopharm Oncology, Inc., , Sevion Therapeutics, Inc., OncoSec Medical, Inc., and Burzynski Clinic.

Download Sample Copy of This Report @ https://prophecymarketinsights.com/market_insight/Insight/request-sample/61

The research report, Personalized Gene Therapy Treatment Market presents an unbiased approach at understanding the market trends and dynamics. Analysts have studied the historical data pertaining to the market and compared it to the current market trends to paint an object picture of the markets trajectory. The report includes SWOT analysis and Porters five forces analysis to give the readers an in-depth assessment of the various factors likely to drive and restrain the overall market.

Market Segmentation:

Request PDF catalogue for this report @ https://prophecymarketinsights.com/market_insight/Insight/request-pdf/61

Table of Contents

Market Overview: The report begins with this section where product overview and highlights of product and application segments of the global Personalized Gene Therapy Treatment market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company: Here, the competition in the global Personalized Gene Therapy Treatment market is analyzed, taking into consideration price, revenue, sales, and market share by company, market concentration rate, competitive situations and trends, expansion, merger and acquisition, and market shares of top 5 and 10 companies.

Company Profiles and Sales Data: As the name suggests, this section gives the sales data of key players of the global Personalized Gene Therapy Treatment market as well as some useful information on their business. It talks about the gross margin, price, revenue, products and their specifications, applications, competitors, manufacturing base, and the main business of players operating in the global Personalized Gene Therapy Treatment market.

Market Status and Outlook by Region: In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the global Personalized Gene Therapy Treatment market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User: This part of the research study shows how different application segments contribute to the global Personalized Gene Therapy Treatment market.

Market Forecast: Here, the report offers complete forecast of the global Personalized Gene Therapy Treatment market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Upstream Raw Materials: The report provides analysis of key raw materials used in the global Personalized Gene Therapy Treatment market, manufacturing cost structure, and the industrial chain.

Marketing Strategy Analysis and Distributors: This section offers analysis of marketing channel development trends, indirect marketing, and direct marketing followed by a broad discussion on distributors and downstream customers in the global Personalized Gene Therapy Treatment market.

Research Findings and Conclusion: This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

Appendix: Here, we have provided a disclaimer, our data sources, data triangulation, market breakdown, research programs and design, and our research approach.

For More Info: https://prophecymarketinsights.com/market_insight/Global-Personalized-Gene-Therapy-Treatment-61

Contact Us:

Mr. Alex (Sales Manager)

Prophecy Market Insights

Phone: +1 860 531 2701

Email: [emailprotected]

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Personalized Gene Therapy Treatment Market To Boost CAGR Prospects (2020-2030) || PMI - Info Street Wire