StemCell Therapy

The Food and Drug Administration has halted a clinical trial involving a Duchenne muscular dystrophy gene therapy from Solid Biosciences (SLDB) after a patient suffered serious kidney and blood-related injuries, the company said Tuesday.

This is the third time that the Cambridge, Mass.-based Solid has run into a serious safety problem with its gene therapy, called SGT-001. The FDA placed similar clinical holds on the same clinical trial after each prior incident, but later allowed the company to proceed with patient dosing.

SGT-001 uses an inactivated virus to deliver a miniaturized but functional version of the dystrophin gene to muscle cells. The gene therapy is designed to be a one-time and potentially curative treatment for all Duchenne patients, regardless of the mutation that causes their disease.

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Sarepta Therapeutics (SRPT) and Pfizer (PFE) are also developing their own gene therapies targeted at Duchenne.

Six patients have been dosed with SGT-001, starting with three at a lower dose; interim results in those patients were previously reported and found to be disappointing. Three more patients were then treated at a higher dose of SGT-001.

The sixth patient became ill soon after being treated in October, experiencing an over-activation of the immune system, an acute kidney injury, reductions in platelets and red blood cells, and cardio-pulmonary insufficiency, Solid said.

All of the toxicities were deemed related to SGT-001 by the patients treating doctor. The patient is being treated and is recovering, Solid said.

Solid reported the patients status to the FDA, which then placed the clinical trial on hold. In a statement, the company said it will work with the FDA in an effort to resolve the hold and determine next steps for the clinical trial.

Pfizers Duchenne gene therapy has also been tied to similar immune system over-activation and related kidney toxicity, although its clinical trial remains active.

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Solid's Duchenne gene therapy trial halted after patient suffers toxicity - STAT

18 November 2019

A gene therapytrial for Duchenne muscular dystrophy (DMD) has been halted after a patient experienced serious side effects.

The clinical trial, run by the US life science company, Solid Biosciences, was put on hold by the FDA (Food and Drug Administration)after the patient had an adverse reaction in response to the experimental gene therapy, called SGT-001. The symptoms included a decrease in red blood cell count, acute kidney injury, over-activation of the immune system and reduced heart and lung function.

'We are encouraged that this patient is recovering,' said Ilan Ganot, chief executive officer and co-founder of Solid Biosciences in Cambridge, Massachusetts. 'In the coming weeks, we anticipate that we will have a better understanding of the biological activity and potential benefit of SGT-001.'

DMD is a severemuscle-wasting disease that affects around one in every 3500 newborn boys. The disease is caused by a mutation in the gene responsible for making an essential muscle protein, called dystrophin. The resulting deficiency or absence of dystrophin leads to a progressive decline in muscle strength. There is currently no cure for the disease.

To date, six patients in the IGNITE DMD clinical trial, taking place at the University of Florida, have received a one-off intravenous infusion of SGT-001. The treatment is an adeno-associated virus (AAV)-based gene therapy, in which an inactivated virus is designed to deliver a synthetic, functional form of dystrophin, called microdystrophin, to muscles.

The first three patients, aged between fourand 17 years, were given the lowest dose outlined in the study protocol. A second cohort of three patients subsequently received a higher dose, which was believed to have led to the adverse reaction reported to the FDA.

The trial waspreviously halted in March 2018, after a patient receiving the low dose of the therapy experienced a similar reaction, from which he later recovered. The trial was resumed in June of the same year after the study design was amended.

Solid Biosciences have reported that all five other patients dosed in the trial are doing well and continue to be examined. They added that they will work with the FDA to determine the next steps for the trial, including how best to manage administration of the therapy.

'We remain committed to bringing meaningful new therapies to the Duchenne community and continue to believe in the differentiated construct of SGT-001 and the potential benefits it may offer to patients,' Ganot said.

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Duchenne gene therapy trial halted after serious reaction - BioNews

Tokyo-based FUJIFILM will invest 13 billion yen (about $120 million) to expand the companys gene therapy business and establish anew Gene Therapy Innovation Center adjacent to its current facility in College Station, Texas and add about 100 jobs.

The investment will include the addition of dedicated gene therapy laboratories and will be part of the existing FUJIFILM Diosynth Biotechnologies (FDB) in Texas, which opened last year. That site has been the companys center for excellence in gene therapy since 2014. The company is expanding its contract and development services for gene therapies as the market for CDMOs in gene therapy is expected to increase to $1.7 billion by 2025, the company said in its announcement.

The Gene Therapy Innovation Center, supported by a $55 million investment, will be approximately 60,000 square feet and will house state-of-the-art upstream, downstream and analytical development technologies. The facility is expected to be operational in the fall of 2021. Gene Therapy remains a strategic investment area for FUJIFILM.

Gerry Farrell, COO at FUJIFILM Diosynth Biotechnologies in Texas, said they anticipate breaking ground on the new facility in the first quarter of 2020. The new Texas sit will triple the companys gene therapy development capabilities and will add approximately 100 jobs to its Texas campus, Farrell said.

Martin Meeson, president and chief operating officer of the U.S. division of FUJIFILM Diosynth Biotechnologies, said the investment will allow FUJIFILM to support the incredible growth that the gene therapy sector has experienced over the past few years.

We know that we need to invest now, in technology, assets and people in order to achieve a market leadership position. The expansion through the construction of the Gene Therapy Innovation Center demonstrates our ongoing commitment for growth, Meeson said in a statement.

FUJIFILMs main goal behind its new strategy is to position itself as a key provider of leading, future-proofed end-to-end gene therapy solutions, from pre-clinical to commercial launch. For the company, this investment builds on earlier plans to introduce its gene therapy fill finish services.

For FUJIFILM, this investment in Texas comes several months after it snapped up Biogens biologics manufacturing operations in Denmark. FUJIFILM paid the Cambridge, Mass.-based company $890 million for the site that had about 800 employees. The acquisition is part of the companys expanded manufacturing strategy. Last year, the company acquired two biotechnology units from JXTG Holdings Inc. for about $800 million.

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FUJIFILM Expands Texas Holdings with New Gene Therapy Center - BioSpace

Dive Brief:

The companies identified Rett, Angelman and Dravet syndromes and Niemann-Pick disorder as the first four conditions that the new collaboration will try to treat. Four more could emerge, which would trigger an additional $42.5 million in additional payments.

Sarepta is paying the initial $48 million upfront fee in the form of combined cash and shares. StrideBio will be eligible for additional undisclosed development, regulatory and sales milestones, as well as royalties. The privately-held partner also will have an option to co-commercialization rights to one of the gene therapies, if successful.

The four StrideBio agents will join a Sarepta pipeline that already has 23 identified projects in clinical or pre-clinical development.

Cambridge, Massaschusetts-based Sarepta stated the collaboration will utilize StrideBio's "unique approach" to engineering capsids, the shells surrounding the adeno-associated virus (AAV) used by many researchers to deliver genes to cells.

StrideBio's technology tries to better target which cells their AAV-based therapies reach, as well as avoid triggering neutralizing antibodies, which can reduce the effectiveness of a gene therapy.

Immune responses to some AAV-based therapies have raised safety concerns. On Tuesday, Solid Biosciences announced the Food and Drug Administration had put a hold on its AAV9-based gene therapy for Duchenne muscular dystrophy (DMD) because of immune responses, although Sarepta's own DMD gene therapy has not seen anything similar.

As part of the agreement, Sarepta and StrideBio "plan to focus on strategies intended to address re-dosing challenges in patients who have received AAV-delivered gene therapy."

Re-treatment of patients who don't respond or have unlimited response to gene therapies is an unanswered question in this quickly evolving field, and drug developers and payers alike will closely watch any developments that emerge from the Sarepta-StrideBio re-dosing work.

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Sarepta brings in more gene therapies with StrideBio deal - BioPharma Dive

GERMANTOWN, Md., Nov. 18, 2019 /PRNewswire/ --Triple-Gene LLC, a clinical stage cardiovascular gene therapy company and majority owned subsidiary of Intrexon Corporation (NASDAQ: XON), yesterday presented preliminary data from the Phase I trial (clinical trial identifier: NCT03409627) of INXN-4001, a multigenic investigational therapeutic candidate under evaluation for the treatment of heart failure, in a poster at the American Heart Association (AHA) Annual Meeting.1 On November 7, 2019, Triple-Gene announcedthat enrollment in this Phase I study has been completed.

"We are pleased to have had the opportunity to share our early clinical data from this first-in-human study with the cardiology community at this prestigious meeting," stated Thomas D. Reed, PhD, Co-founder and Managing Director of Triple-Gene. "The data presented yesterday suggest that the combination of our transiently expressed, non-integrating naked plasmid DNA with the focused cardiac delivery enabled by Retrograde Coronary Sinus Infusion (RCSI) has the potential to open a new biologics treatment paradigm for treating cardiovascular diseases."

Triple-Gene's investigational therapy uses non-viral delivery of a constitutively controlled multigenic plasmid designed to express human S100A1, SDF-1, and VEGF165 gene products, which affect progenitor cell recruitment, angiogenesis, and calcium handling, respectively, and target the underlying molecular mechanisms of pathological myocardial remodeling. The plasmid therapy is delivered via RCSI, which allows for cardiac-specific delivery to the ventricle.

Dr. David Bull, who was the first investigator to dose a patient with INXN-4001, stated, "Having carefully reviewed the science behind Triple-Gene's product candidate, I was very excited to initiate the INXN4001 clinical trial at the University of Arizona. Heart failure is a devastating disease, and my patients on Left Ventricular Assist Devices (LVAD) have very few therapeutic treatment options. Triple-Gene's novel triple-effector plasmid, as delivered by the minimally invasive RCSI procedure, represents a potential game-changing approach for addressing disease pathology in this high-risk patient population."

Amit N. Patel, MD, MS, Co-founder and Clinical Director of Triple-Gene added, "We are pleased to have completed dosing of the twelve patients in this clinical trial between our two clinical sites at the University of Arizona, Tucson, AZ and The Christ Hospital, Cincinnati, OH, and look forward to sharing additional data for this investigational therapy once the trial is complete. Based upon the promising early results to date, we are now exploring clinical trial designs that contemplate repeat dosing as well as additional orphan-like heart failure subtypes."

1Jaruga-Killeen E, Bull DA, Lotun K, Henry T, Egnaczyk G, Reed TD and Patel AN. Safety of first-in-human triple gene therapy for heart failure patients. Presented at the American Heart Association Annual Meeting, November 17, 2019.

About Triple-GeneTriple-Gene LLC is a clinical stage gene therapy company focused on advancing targeted, controllable, and multigenic gene therapies for the treatment of complex cardiovascular diseases. The Company's lead product is a non-viral investigational gene therapy candidate that drives expression of three candidate effector genes involved in heart failure. Triple-Gene is a majority owned subsidiary of Intrexon Corporation(NASDAQ: XON) co-founded by Amit Patel, MD, MS, and Thomas D. Reed, PhD, Founder and Chief Science Officer of Intrexon. Learn more about Triple-Gene at http://www.3GTx.com.

About Intrexon CorporationIntrexon Corporation (NASDAQ: XON) is Powering the Bioindustrial Revolution with Better DNAto create biologically-based products that improve the quality of life and the health of the planet through two operating units Intrexon Health and Intrexon Bioengineering. Intrexon Health is focused on addressing unmet medical needs through a diverse spectrum of therapeutic modalities, including gene and cell therapies, microbial bioproduction, and regenerative medicine. Intrexon Bioengineering seeks to address global challenges across food, agriculture, environmental, energy, and industrial fields by advancing biologically engineered solutions to improve sustainability and efficiency. Our integrated technology suite provides industrial-scale design and development of complex biological systems delivering unprecedented control, quality, function, and performance of living cells. We call our synthetic biology approach Better DNA, and we invite you to discover more at http://www.dna.comor follow us on Twitter at @Intrexon, on Facebook, and LinkedIn.

TrademarksIntrexon, Powering the Bioindustrial Revolution with Better DNA,and Better DNA are trademarks of Intrexon and/or its affiliates. Other names may be trademarks of their respective owners.

Safe Harbor Statement Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon our current expectations and projections about future events and generally relate to our plans, objectives and expectations for the development of our business. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release.

For more information contact:

Investor Contact:

Steven Harasym

Vice President, Investor Relations

Intrexon Corporation

Tel: +1 (301) 556-9850

investors@dna.com

Corporate Contact:

Marie Rossi, PhD

Vice President, Communications

Intrexon Corporation

Tel: +1 (301) 556-9850

publicrelations@dna.com

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Triple-Gene Presented Preliminary Phase 1 Trial Data of Investigational Multigenic Therapeutic Candidate INXN-4001 for Treatment of Heart Failure at...

NEW YORK and BASEL, Switzerland, Nov. 18, 2019 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (NASDAQ: AXGT), a clinical-stage company developing innovative gene therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for the Companys investigational gene therapy, AXO-AAV-GM1, for the treatment of GM1 gangliosidosis. GM1 gangliosidosis is a progressive and fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene leading to impaired production of the beta-galactosidase enzyme. The Company is planning to complete enrollment of patients in Part A of the AXO-AAV-GM1 registrational program evaluating safety and efficacy and expects to announce 6-month data from Part A in mid-2020.

We are pleased to receive orphan drug designation for our GM1 gangliosidosis program as we advance the development of this potentially transformative, one-time gene therapy, said Dr. Gavin Corcoran, chief R&D officer at Axovant. There are currently no FDA approved therapies for patients with GM1 gangliosidosis, and the orphan drug designation underscores the significant unmet medical need. We look forward to increased interaction with the FDA to facilitate the development of AXO-AAV-GM1 and address this unmet need.

FDA orphan drug designation is granted to investigational therapies which are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug designation provides several benefits to drug developers, which in addition to others, include increased FDA interaction, eligibility for 7-year market exclusivity, potential for tax credits towards the cost of clinical trials, and prescription drug user fee waivers at the time of filing a biologics license application (BLA). For more information about orphan drug designation, please visit the FDA website at http://www.fda.gov.

About AXO-AAV-GM1

AXO-AAV-GM1 is an investigational gene therapy that delivers a functional copy of theGLB1gene via an adeno-associated viral (AAV) vector, with the goal of restoring -galactosidase enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1s ability to improve -galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.

About Axovant Gene Therapies

Axovant Gene Therapies, part of the Roivant family of companies, is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visitwww.axovant.com.

In 2018, Axovant licensed exclusive worldwide rights from the University of Massachusetts Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

About Roivant

Roivantaims to improve health by rapidly delivering innovative medicines and technologies to patients.Roivantdoes this by buildingVants nimble, entrepreneurial biotech and healthcare companies with a unique approach to sourcing talent, aligning incentives, and deploying technology to drive greater efficiency in R&D and commercialization. Roivant today is comprised of a central technology-enabled platform and 20 Vants with over 45 investigational medicines in clinical and preclinical development and multiple healthcare technologies. For more information, please visitwww.roivant.com.

Forward Looking Statements and Information

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, might, will, would, should, expect, believe, estimate, and other similar expressions are intended to identify forward-looking statements. For example, all statements Axovant makes regarding the initiation, timing, progress, and reporting of results of its preclinical programs, clinical trials, and research and development programs; cash to be used in operating activities; its ability to advance its gene therapy product candidates into and successfully initiate, enroll, and complete clinical trials; the potential clinical utility of its product candidates; its ability to continue to develop its gene therapy platforms; its ability to develop and manufacture its products and successfully transition manufacturing processes; its ability to perform under existing collaborations with, among others, Oxford BioMedica and theUniversity of Massachusetts Medical School, and to add new programs to its pipeline; its ability to enter into new partnerships or collaborations; its ability to retain and successfully integrate its leadership and personnel; and the timing or likelihood of its regulatory filings and approvals are forward-looking. All forward-looking statements are based on estimates and assumptions by Axovants management that, although Axovant believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Axovant expected.Such risks and uncertainties include, among others, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the expectations for regulatory submissions and approvals; the continued development of its small molecule and gene therapy product candidates and platforms; Axovants scientific approach and general development progress; and the availability or commercial potential of Axovants product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Axovants most recent Quarterly Report on Form 10-Q filed with theSecurities and Exchange CommissiononNovember 8, 2019, as updated by its subsequent filings with theSecurities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made.Axovant undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts:

Media and Investors

Parag MeswaniAxovant Gene Therapies(212) 547-2523investors@axovant.commedia@axovant.com

Originally posted here:
Axovant Gene Therapies Receives Orphan Drug Designation from FDA for AXO-AAV-GM1 for the Treatment of GM1 Gangliosidosis - BioSpace

CHICAGO, Nov. 13, 2019 /PRNewswire/ -- According to Arizton's recent research report, Cell and Gene Therapy Market - Global Outlook and Forecast 2019-2024 is expected to grow at a CAGR of more than 24% during the forecast period.

Key Highlights Offered in the Report:

Key Offerings:

Get your free sample today! https://www.arizton.com/market-reports/cell-and-gene-therapy-market

Cell and Gene Therapy Market Segmentation

Market Segmentation by Products

Market Segmentation by Distribution Channel Type

Market Segmentation by End-users

Cell and Gene Therapy Market Dynamics

CAR T-cell therapy has gained significant traction in recent years. It is the single most rapidly growing type of product in the market that generates revenue at a phenomenal rate. At present, it is the fastest advancing technology in cancer treatment and has the capability to replace many existing therapies. CAR T-cell therapy addresses current challenges in cancer care through superior efficacy, safety, and delivery mechanisms. CAR T-cell therapy has brought itself into focus due to the personalized nature of this therapy and the utilization of advanced genetic engineering technology. The wide acceptance and use of CAR T-cell therapy is fueling the growth of the global cell and gene therapy market.

Key Drivers and Trends fueling Market Growth:

Cell and Gene Therapy MarketGeography

The US dominates the cell and gene therapy market in North America due to the high prevalence of chronic diseases and other conditions. There is also comparably high utilization and wide accessibility of these therapies. In Europe, cell and gene therapy products are considered to be part of the Advanced Therapy Medicinal Products (ATMPs), which are commonly known as regenerative medicine globally. The major factors leading to the growth in APAC region are the growing prevalence of cancers, osteoarthritis, burns, and other chronic wounds, the introduction of advanced products in Japan, advanced R&D activities in countries such as South Korea, India.

Get your free sample today! https://www.arizton.com/market-reports/cell-and-gene-therapy-market

Market Segmentation by Geography

Major Vendors

Other vendors include - Anterogen, Tego Sciences, Japan Tissue Engineering, JCR Pharmaceuticals, Medipost, MolMed, AVITA Medical, CollPlant, Corestem, Biosolution, Stempeutics Research, Orchard Therapeutics, Takeda Pharmaceutical Company, CHIESI Farmaceutici, CO.DON, AnGes, GC Pharma, JW CreaGene, APAC Biotech, Nipro Corp., Terumo, Orthocell, and bluebird bio.

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About Arizton:

Arizton Advisory and Intelligence is an innovation and quality-driven firm, which offers cutting-edge research solutions to clients across the world. We excel in providing comprehensive market intelligence reports and advisory and consulting services.

We offer comprehensive market research reports on industries such as consumer goods & retail technology, automotive and mobility, smart tech, healthcare, and life sciences, industrial machinery, chemicals and materials, IT and media, logistics and packaging. These reports contain detailed industry analysis, market size, share, growth drivers, and trend forecasts.

Arizton comprises a team of exuberant and well-experienced analysts who have mastered in generating incisive reports. Our specialist analysts possess exemplary skills in market research. We train our team in advanced research practices, techniques, and ethics to outperform in fabricating impregnable research reports.

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The Cell and Gene Therapy Market to Reach Revenues of Over $6.6 billion by 2024 - Market Research by Arizton - PRNewswire

COLLEGE STATION, Texas, Nov. 14, 2019 /PRNewswire/ -- FUJIFILM Diosynth Biotechnologies (FDB), a leading global biologics Contract Development and Manufacturing Organization (CDMO) has announced the expansion of its gene therapy services with the addition of dedicated process and analytical development laboratories. As a part of a capital investment of approximately 13 billion yen (approx. $120 million USD) in the gene therapy fieldby FUJIFILM Corporation,aninvestment of approximately $55 million USD will be made to establish anew Gene Therapy Innovation Center adjacent to FDB's existing state-of-the-art cGMP gene therapy manufacturing facility in College Station, Texas and forms part of the company's strategy to meet the growing demands in the Viral Gene Therapy Market. The gene therapy market forecast for CDMOs is expected to grow to $1.7Bn by 2025.1

The Gene Therapy Innovation Center will be approximately 60,000 square feet and will house state-of-the-art upstream, downstream and analytical development technologies. The facility will be operational in the fall of 2021.

"We are very much aware of the incredible growth in such an important therapeutic space," said Martin Meeson, President and COO of FUJIFILM Diosynth Biotechnologies, US. "We know that we need to invest now, in technology, assets and people in order to achieve a market leadership position. The expansion through the construction of the Gene Therapy Innovation Center demonstrates our ongoing commitment for growth."

FDB's main goals behind this new strategy are to provide leading, future proofed end-to-end gene therapy solutions, from pre-clinical to commercial launch. This follows an earlier announcement made by FDB to introduce its gene therapy fill finish services. "We expect to break ground in the first quarter of 2020," said Gerry Farrell, COO at FUJIFILM Diosynth Biotechnologies, Texas, "this new facility will triple our gene therapy development capabilities and will add approximately 100 jobs to our Texas Campus."

Gene Therapy remains a strategic investment area for Fujifilm.

About Fujifilm FUJIFILM Diosynth Biotechnologies an industry-leading Biologics Contract Development and Manufacturing Organization (CDMO) with locations in Teesside, UK, RTP, North Carolina, College Station, Texas and Hillerod, Denmark. FUJIFILM Diosynth Biotechnologies has over thirty years of experience in the development and manufacturing of recombinant proteins, vaccines, monoclonal antibodies, among other large molecules, viral products and medical countermeasures expressed in a wide array of microbial, mammalian, and host/virus systems. The company offers a comprehensive list of services from cell line development using its proprietary pAVEway microbial and Apollo cell line systems to process development, analytical development, clinical and FDA-approved commercial manufacturing. FUJIFILM Diosynth Biotechnologies is a partnership between FUJIFILM Corporation and Mitsubishi Corporation. For more information, go to: http://www.fujifilmdiosynth.com

FUJIFILM Holdings Corporation, Tokyo, Japan, brings cutting edge solutions to a broad range of global industries by leveraging its depth of knowledge and fundamental technologies developed in its relentless pursuit of innovation. Its proprietary core technologies contribute to the various fields including healthcare, graphic systems, highly functional materials, optical devices, digital imaging and document products. These products and services are based on its extensive portfolio of chemical, mechanical, optical, electronic and imaging technologies. For the year ended March 31, 2019, the company had global revenues of $22 billion, at an exchange rate of 111 yen to the dollar. Fujifilm is committed to responsible environmental stewardship and good corporate citizenship. For more information, please visit: http://www.fujifilmholdings.com.

All product and company names herein may be trademarks of their registered owners.

1 Market research conducted by FUJIFILM Diosynth Biotechnologies strategic business development group.

SOURCE FUJIFILM Diosynth Biotechnologies

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FUJIFILM Diosynth Biotechnologies Announces $55 Million USD Investment To Expand Gene Therapy Development Capabilities - PRNewswire

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy in Neurology" report has been added to ResearchAndMarkets.com's offering.

Gene therapy is an evolving area in healthcare that promises to revolutionize the treatment landscapes across various therapy areas.

In this report, the focus will be on neurology indications. The report provides an analysis of the overall gene therapy pipeline that is being developed for various neurology indications with an emphasis on late-stage pipeline products. In addition to pipeline analysis, the report also focuses on trends observed in clinical trials in this area, unmet needs and challenges, as well as partnership strategies adopted by pharmaceutical companies to keep up with developments in the field of gene therapies.

Recently approved gene therapies for spinal muscular atrophy have reinvigorated the potential of such therapies to transform patient care. While various methodologies can be adopted in order to deliver therapeutic benefits of gene therapy including gene augmentation, gene suppression, and gene editing, an important component of gene therapy is whether to use viral or non-viral vectors in order to deliver such therapies to the point of care.

Ongoing collaborations between different industry players and a buildup of real-world evidence establishing safety and efficacy are expected to drive the growth of gene therapies for neurology indications. Of the 38 pipeline products that are currently in development, 45% are adeno-associated virus (AAV) based delivery platforms. Other types include Lentiviral, which accounts for 13%.

A majority of the current pipeline products are in Phase II development and the most common neurology indications - for which gene therapies are currently being evaluated - include Parkinson's disease, pain and amyotrophic lateral sclerosis. The dominance of viral vectors is expected to continue as such platforms account for the bulk of these pipeline products, with adeno-associated virus being the most common among the viral vectors.

In terms of completed, ongoing and planned clinical trials, academic institutes account for 21% of these trials, despite industry sponsors being most dominant. A deeper analysis of these clinical trials also suggest that across most indications, the average trial duration for a viral based product is longer compared to a non-viral based product such as oligonucleotides or plasmid DNA.

There are also challenges associated with the development of gene therapies, most prominent being their high price points. Key opinion leaders (KOLs) interviewed highlighted the need to create sustainable funding solutions so that such therapies become accessible to patients everywhere irrespective of where patients are located. In terms of unmet needs, KOLs highlighted the need for a favorable route of administration that is both sustainable in terms of usage of healthcare resources and favorable from a patient perspective.

While development of gene therapies are expected to pick up pace, the next wave of such therapies are expected to be ones that target diseases that are more frequent. While monogenic rare diseases are the obvious first-to-go choice for which gene therapies can be developed, targeting more frequent diseases will need a holistic approach in order to address a wider mechanism of action. If gene therapies for frequent diseases do become available, then that will result in a more pronounced effect on healthcare not only in terms of providing better treatment options for patients but also test the ability of healthcare organizations to adapt with high price points of these therapies.

Scope

Reasons to Buy

Key Topics Covered

1 Preface

2 Executive Summary

2.1 Key Findings

2.2 KOL Insights on Competitive Landscape for Gene Therapy for Neurology Indications

3 Overview - Gene Therapy in Neurology

3.1 Objectives of Gene Therapy

3.2 Gene Therapy Versus Conventional Therapies

3.3 Optimization of Gene Expression

3.4 Gene Transfer Methods and Vectors Used for Gene Therapy

3.5 Classifications of Gene Therapy

3.6 Sources

4 Gene Therapy in the 8MM

4.1 Global Regulatory Agencies' Definitions of Gene Therapy

4.2 Gene Therapy in the US

4.3 Gene Therapy in the EU

4.4 Gene Therapy in Japan

4.5 Gene Therapy in China

4.6 Currently Marketed Gene Therapies in Neurology

4.7 Sources

5 Pipeline Assessment in the 8MM

5.1 Pipeline Overview

5.2 Pipeline Products - Phase III

5.3 Pipeline Products - Phase II

5.4 Orchard Therapeutics' OTL-200

5.5 Biogen's Tofersen sodium

5.6 Roche's RG-

5.7 Sylentis' Tivanisiran

5.8 ViroMed's Donaperminogene Seltoplasmid

5.9 Sources

6 Clinical Trials Mapping and Design

6.1 Clinical Trial Mapping for all Pipeline Products by Phase, by Sponsor, and by Location

6.2 Clinical Trial Mapping for all Pipeline Products by Status and by Indication

6.3 Clinical Trial Mapping by Phase and Indication for Phase III Therapies

6.4 Clinical Trial Mapping by Phase for Phase II Therapies

6.5 Clinical Trial Duration by Indication for Phase III Therapies (By Types of Molecules)

6.6 Clinical Trial Duration by Indication for Phase II Therapies (By Types of Molecules)

6.7 Ongoing Clinical Development of Phase III Gene Therapies

7 Unmet Needs, Barriers, and Key Company Strategies

7.1 Unmet Needs Within Gene Therapy for CNS Indications

7.2 Challenges and Other Factors to Consider During Different Stages of Product Development

7.3 Key Company Strategies: Acquisitions

7.4 Key Company Strategies: Strategic Partnerships

7.5 Sources

8 Payer Perspective on Gene Therapies in Neurology

8.1 Current Neurology Space

8.2 Challenges Associated with Reimbursement of Novel Gene Therapies

8.3 Cost of Gene Therapies

8.4 Strategies to Tackle the Cost of Gene Therapies

8.5 Innovative Reimbursement Models and Clinical Comparators

9 Market Outlook

9.1 Phase III Gene Therapy Pipeline for Neurology

9.2 Key Launch Dates for Phase III Gene Therapy Pipeline Products

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/5pahu7

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Gene Therapy in Neurology, 2019: Market Outlook, Pipeline Assessment, Unmet Needs, Barriers, Key Company Strategies & Payer Perspective -...

According to a latest report published by Global Marketers Biz named as Mesenchymal Stem Cells Market offers data for the forecast period 2019-2024. A comprehensive research updates and data which includes following key aspects for the global Mesenchymal Stem Cells Market in terms of volume and revenue Visitor Demographics, Facility Size, Demand & Growth Opportunities, Global Industry Forecast Analysis and Revenue Source.

The Mesenchymal Stem Cells Market report offers in-depth analysis and insights into developments impacting businesses and enterprises on global & regional level. A detailed breakdown of key trends, drivers, restraints, and opportunities influencing revenue growth is presented in this research report. This study focuses on the global Mesenchymal Stem Cells market by share, volume, value, and regional appearance along with the types and applications.

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Key Players of Mesenchymal Stem Cells Report are:

LonzaThermo FisherBio-TechneATCCMilliporeSigmaPromoCell GmbHGenlantisCelprogenCell ApplicationsCyagen Biosciences

Market is Segmented into below points:

Market by Type/Products:

Human MSCMouse MSCRat MSCOther

Market by Application/End-Use:

Research InstituteHospitalOthers

Market by Region:

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Global Mesenchymal Stem Cells Market Forecast, by regions, type and application, with sales and revenue, from 2019 to 2024. - Montana Ledger

While resistance can often make it a challenge to treat mantle cell lymphoma, therapies have come a long way in recent years thanks to the emergence of personalized treatment and the use of BTK inhibitors, according to one expert.

Ruan, a hematologist and oncologist from Weil Cornell Medicine in New York City, recently sat down with OncLive, CUREs sister publication, to discuss the treatment trends in this disease space.

We're moving toward precision medicine and personalized treatment (in MCL), said Ruan. The objective is to overcome resistance and come up with treatment plans that address the underlying disease biology and take personal factors about the patients into consideration.

When asked about the novel agents that are currently showing promise in MCL, Ruan explained that BTK inhibitors have resulted in highoverall response rates (ORRs), more complete remissions (CRs) and a lower number of side effects in this patient population.

Since the approval of Revlimid (lenalidomide) in 2013, a number of newer BTK inhibitors have been approved, with first-generation Imbruvica (ibrutinib) standing out as the top choice for many. So far, this class of agents has the best single-agent activity in MCL to date, explained Ruan. For example, ibrutinib would give about a 65% to 67% ORR, and about 20% of that would be CRs. In contrast, lenalidomide would give you about a 30% ORR and up to about 8% CR.

A newer BTK inhibitor, Calquence (acalabrutinib), is considered just as effective as Imbruvica with an overall response rate of 81% and complete response in 40% of patients who took it during the phase 2 study that led to its approval. That's quite remarkable for a single agent for patients who have relapsed/refractory MCL, Ruan said.

The field continues to grow with the testing of new agents, including BCL-2 inhibitor, Venclexta (venetoclax), which is currently approved in chronic lymphocytic leukemia and other indolent B-cell non-Hodgkin lymphomas. In MCL, we have data to suggest that it has promising activity as a single agent, she said. Currently, it's being studied in combination with a variety of other agents, including BTK inhibitors. They seem to work very well and are helping to move treatment away from chemotherapy.

However, treating MCL can be challenging because of treatment resistance, explained Ruan. All of the agents are being introduced, tested, and approved in the setting of relapsed disease, said Ruan. That is a big challenge for a sizable portion of patients who do not respond maybe they have a genetic mutation that is not responsive to targeted therapy or they develop a secondary mutation in the process of being treated and then they become resistant.

The issue of resistance is important to consider when treating MCL, according to Ruan. We have to provide novel agents that are sensitive to the disease at every stage of the evolution and come up with additional therapies that can overcome the resistance, she said.

In the future, Ruan predicts the shift toward precision medicine and personalized treatment will continue. The objective is to overcome resistance and come up with treatment plans that address the underlying disease biology and take personal factors about the patients into consideration, she said. This is important for us to understand MCL individually.

This precise approach would mean doing more genetic analysis to determine what type of disease is being treated in order to create a treatment plan that would provide the most benefit for the patient.

If we could know (about mutations) ahead of time, we could have the treatment designed and tailored to maximize treatment effectiveness and minimize adverse events, said Ruan.

It doesn't make sense to subject patients to all of the cytotoxicity associated with conventional intensive chemotherapy while other options, such as participation in clinical trials with novel agents, could be very effective.

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Precision Medicine and Personalized Treatments Shifting the Tide in Mantle Cell Lymphoma - Curetoday.com

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Relevant clinical advances that improve outcomes for patients with glioblastoma have been few and far between over the last two decades, said Frederic Sottilini, CEO of CarThera. Despite multimodal therapy, median survival remains around 15 months for these patients, virtually all of whom recur. Our goal is to optimize the selection and delivery of drug therapies to extend the lives of patients with glioblastoma.

The two companies were brought together by one of the worlds leading neuro-oncology and glioblastoma experts, John de Groot, M.D., professor and chairman ad interim, The University of Texas MD Anderson Cancer Center, who recognized the synergistic nature of their respective clinical initiatives. CarThera is currently conducting a multi-center clinical study of its novel ultrasound technology, SonoCloud-9, designed to increase the permeability of the blood brain barrier to improve the delivery of chemotherapeutic agents to the brains of patients with recurrent glioblastoma. KIYATEC is conducting a multi-center clinical study of its ex vivo 3D cell culture technology to accurately predict pre-treatment, patient-specific response to recommended standard of care cancer drugs for newly diagnosed and recurrent glioblastoma.

As someone who cares for patients with glioblastoma, I applaud the efforts of CarThera and KIYATEC to bring evidence-based advances to the clinic for the purpose of improving outcomes for patients with glioblastoma, said Dr. de Groot. I envision these two technologies as being complementary with the potential to transform the way in which neuro-oncologists manage glioblastoma patients.

Under the terms of the clinical collaboration, KIYATEC will conduct ex vivo drug response profiling on glioblastoma tissue samples from patients enrolled in CarTheras clinical study. CarThera will benefit from having ex vivo drug response profiling for patients enrolled in its study, while KIYATEC will correlate its patient-specific, pre-treatment drug response predictions with actual clinical outcomes of patients in CarTheras study. For both companies, this collaboration represents an opportunity to enrich their portfolios of clinical evidence with the goal of helping clinicians improve outcomes for their patients with glioblastoma.

Both of our companies are dedicated to ensuring that glioblastoma patients receive the most appropriate drug therapy at the right time, and that the efficacy of that therapy is maximized to its fullest therapeutic potential, said Matthew Gevaert, CEO and co-founder of KIYATEC. We believe that this clinical collaboration has the potential to help us accelerate and deliver on the long-awaited promise of personalized medicine for these deserving patients.

Both companies will be sending delegates to the 24th Annual Meeting of the Society for Neuro-Oncology, November 20-24 in Phoenix, Arizona.

About KIYATEC, Inc. KIYATEC leverages its proprietary ex vivo 3D cell culture technology platforms to accurately model and predict response to approved and investigational cancer drugs targeting a spectrum of solid tumors. The companys Drug Development Services business works in partnership with leading biopharmaceutical companies to unlock response dynamics for its investigational drug candidates across the majority of solid tumor types. The companys Clinical Services business is currently engaged in the validation of clinical assays as well as investigator-initiated studies in ovarian cancer, breast cancer, glioblastoma and rare tumors, in its CLIA-certified laboratory. To learn more about KIYATEC, visit http://www.kiyatec.com.

About CarThera CarThera designs and develops innovative therapeutic ultrasound-based medical devices for treating brain disorders. The company is a spin-off from AP-HP, Greater Paris University Hospitals, the largest hospital group in Europe, and Sorbonne University. Since 2010, CarThera has been leveraging the inventions of Professor Alexandre Carpentier, a neurosurgeon at AP-HP who has achieved worldwide recognition for his innovative developments in treating brain disorders. CarThera developed SonoCloud, an intracranial ultrasound implant that temporarily opens the blood-brain barrier (BBB). CarThera is based at the Brain and Spine Institute (Institut du Cerveau et de la Moelle pinire, ICM) in Paris, France, and has laboratories at the Bioparc Lannec business incubator in Lyon, France. The company, led by Frederic Sottilini (CEO), works closely with the Laboratory of Therapeutic Applications of Ultrasound (Laboratoire Thrapie et Applications Ultrasonores, LabTAU, INSERM) in Lyon.www.carthera.eu

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CarThera and KIYATEC Collaborate in Effort to Bring Personalized Medicine to Patients Stricken by Brain Cancer - BioSpace

This report provides data on patterns, improvements, target business sectors, materials, limits, and advancements. By thinking from the customers perspective, a team of researchers, forecasters, analysts, and industry experts work carefully to formulate this Global Precision Medicine Market report. Furthermore, it works to determine the impact of buyers, substitutes, new entrants, competitors, and suppliers on the market. This gives more accurate understanding of the market landscape, issues that may affect the industry in the future, and how to best position specific brands. The Global Precision Medicine Market report helps to develop a successful marketing strategy for your business and acts as a backbone to the business.

Global Precision Medicine Marketto grow with a substantial CAGR in the forecast period of 2019-2026. Growing prevalence of cancer worldwide and accelerating demand of novel therapies to prevent of cancer related disorders are the key factors for lucrative growth of market

Key Market Players:

Few of the major competitors currently working in the global precision medicine market areNeon Therapeutics, Moderna, Inc, Merck & Co., Inc, Bayer AG, PERSONALIS INC, GENOCEA BIOSCIENCES, INC., F. Hoffmann-La Roche Ltd, CureVac AG, CELLDEX THERAPEUTICS, BIONTECH SE, Advaxis, Inc, GlaxoSmithKline plc, Bioven International Sdn Bhd, Agenus Inc., Immatics Biotechnologies GmbH, Immunovative Therapies, Bristol-Myers Squibb Company, Gritstone Oncology, NantKwest, Inc among others.

Global Precision Medicine Market By Application (Diagnostics, Therapeutics and Others), Technologies (Pharmacogenomics, Point-of-Care Testing, Stem Cell Therapy, Pharmacoproteomics and Others), Indication (Oncology, Central Nervous System (CNS) Disorders, Immunology Disorders, Respiratory Disorders, Others), Drugs (Alectinib, Osimertinib, Mepolizumab,Aripiprazole lauroxil and Others), Route of Administration (Oral,Injectable), End- Users (Hospitals, Homecare, Specialty Clinics, Others), Geography (North America, South America, Europe, Asia-Pacific, Middle East and Africa) Industry Trends and Forecast to 2026

Competitive Analysis:

The precision medicine market is highly fragmented and is based on new product launches and clinical results of products. Hence the major players have used various strategies such as new product launches, clinical trials, market initiatives, high expense on research and development, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of mass spectrometry market for global, Europe, North America, Asia Pacific and South America.

Market Definition:

Precision medicines is also known as personalized medicines is an innovative approach to the patient care for disease treatment, diagnosis and prevention base on the persons individual genes. It allows doctors or physicians to select treatment option based on the patients genetic understanding of their disease.

According to the data published in PerMedCoalition, it was estimated that the USFDA has approved 25 novels personalized medicines in the year of 2018. These growing approvals annually by the regulatory authorities and rise in oncology and CNS disorders worldwide are the key factors for market growth.

Market Drivers

Market Restraints

Key Developments in the Market:

Competitive Analysis:

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

Market Segmentation:

By technology:-big data analytics, bioinformatics, gene sequencing, drug discovery, companion diagnostics, and others.

By application:- oncology, hematology, infectious diseases, cardiology, neurology, endocrinology, pulmonary diseases, ophthalmology, metabolic diseases, pharmagenomics, and others.

On the basis of end-users:- pharmaceuticals, biotechnology, diagnostic companies, laboratories, and healthcare it specialist.

On the basis of geography:- North America & South America, Europe, Asia-Pacific, and Middle East & Africa. U.S., Canada, Germany, France, U.K., Netherlands, Switzerland, Turkey, Russia, China, India, South Korea, Japan, Australia, Singapore, Saudi Arabia, South Africa, and Brazil among others.

In 2017, North America is expected to dominate the market.

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Global Precision Medicine Market Share 2019 Analysis With Observational Studies of Neon Therapeutics, Moderna, Inc, Merck & Co., Inc, Bayer AG,...

For centuries, medicine has largely relied on a single primary source of information for the diagnosis and treatment of disease: individual patients. This data stream has become increasingly complex over time due to advances in imaging, biochemistry and genetics, but it is still patient-based.

Now, a second stream is emerging that I believe is likely to impact the entire healthcare delivery system, both in terms of outcomes and economics: population-based data. The sources of this data could include census records, various government agencies and community health records. The information it provides could span neighborhood and housing conditions, economic stability, education levels, social and community conditions and the availability of healthcare. All of these could contribute to a three-dimensional view of the patient and provide insights into treatment options and the management of chronic conditions. In fact, research (via the National Academy of Medicine) suggests that social determinants account for 80% to 90% of "modifiable contributors to healthy outcomes."

This figure is more than an interesting statistic. It has demonstrable real-world consequences. For example, a Maricopa County, Arizona, program that incorporates social determinants played a significant role in a 67% decrease in suicides in its first three months. The data collected related to poverty, food and housing insecurity, and environmental exposures such as homes with lead paint. Efforts to reduce some of the problems that were discovered, such as delivering meals or installing air conditioners, were likely central to the programs success.

The amount of healthcare data is huge and growing. According to Research and Markets, it more than quadrupled to 700 exabytes from 2013 to 2017 and is projected to triple again to 2,314 exabytes by 2020. Its potential value to businesses is even more impressive. For example, EY estimates that the value of the U.K. National Health Service's datasets could be 5 billion (approximately $6.4 billion) per year and deliver around 4.6 billion (approximately $5.9 billion) of benefit to patients per year in potential operational savings for the NHS, enhanced patient outcomes and generation of wider economic benefits to the U.K.."

New Sources Of Patient Data

The amount of patient-based data is also growing due to new technology developments. Wearables are one prominent source of this new data. The Apple Watch, for example, can monitor and track heart rhythm with a level of sophistication that would have been unimaginable only a few years ago. Glucose monitors can be connected to smartphones to support behavioral changes in patients who need help managing their diabetes.

One of the most exciting developments to me is affordable genome sequencing. It was once a process that cost a thousand dollars or more, but one company recently offered it for $199. Access to this data is a very important development for personalized medicine, especially for cancer patients for whom targeted therapies based on genome analysis could mean the difference between life and death.

Data-Centric Business Models

The explosion in healthcare data has led to a number of data-driven business partnerships. Pfizer collaborated with Flatiron Health to use real-world data in an application for FDA drug approval. In another example, a large insurance provider created an app for smartwatches to help members track their actions so they can receive rewards for activities that improve their well-being. There is also a marketplace for anonymous data. Cloud-based HealthVerity Marketplace, for example, lets customers browse and license data from over 50 billion anonymized transactions.

More partnerships and vertical marketplaces for data will surely emerge over time.

Challenges Ahead

While the idea of data-driven healthcare is widely embraced, putting it into practice is not so simple. There are several barriers to widespread use of statistical data as a new weapon in the ongoing battle against disease.

The technical challenges of collecting and analyzing health data are not trivial. One 2010 survey (viaPR Newswire) conducted for a web-based electronic health record (EHR) company found that the average American patient accumulates records from 18 different providers over a lifetime. The data from many of those visits may be in incompatible formats. In my experience, data collected by governments tends to be in silos as well. As a result, I expect that a considerable amount of data integration work will be required before the data is even ready for analysis.

The twin concerns of privacy and security are also major barriers to data sharing. Generally speaking, patients may be willing to share their health data when it is for a good cause -- such as better outcomes for others. However, there is always the risk that such data will be used to discriminate against people with health conditions. In addition, there is the issue of security -- over 13 million healthcare records were reportedly exposed in breaches in 2018 alone. Another issue is that providers may fear losing a competitive advantage by sharing data.

However, none of these are insurmountable barriers. I believe digital data has enormous potential to influence outcomes for the better and to deliver business value. That potential will not go untapped. Patient data will still be extremely important, but I'm sure it will be enhanced by data from external sources in a win-win model.

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How We Can Improve The Healthcare Industry With A Healthy Dose Of Data - Forbes

Todays clinical labs are experiencing a shortage of personnel, but the labs themselves cant slow down.

Value-based healthcare puts a premium on delivering patient outcomes, Timea Zsiray, director of strategy and marketing for Beckman Coulters Workflow and Informational Technology Solutions Business, explained during a press conference at AACC 2019. Clinical labs are an essential part of healthcare, as 65 to 70% of [healthcare] decisions are made based on lab data, she explained.

Consequently, labs have to improve turnaround time while delivering accurate results, she added.

Automation is a promising solution. She pointed out that 32 main steps are typically performed in the lab, most of which are performed manually, and addedthat manual processes are "time consuming and error prone. There is "a need to make lab personnels jobs easier.

However, automation is not just the movement of tubesbut also the digital movement of data, Zsiray.

Healthcare professionals who spoke during the press conference all agreed that healthcare systems are experiencing a shortage of well-trained medical professionals. These speakers included Dr. David Koch, associate professor of pathology at Emory University School of Medicine and director of clinical chemistry, special chemistry, toxicology and point-of-care testing at Grady Memorial Hospital in Atlanta; Dr. Steven H. Wong, professor of pathology and director of the clinical chemistry and toxicology core laboratory, and co-director of the clinical and translational mass spectrometry center at Wake Forest School of Medicine; and Robin Ades, senior regional director of operations for Alverno Laboratories.

Said Koch: We dont have enough people going into the clinical lab profession.

Added Ades: Small- and mid-sized labs have the same staffing issues, especially in rural areas where we dont have the pool of applicants as in the past. And efficiency is expected of all labs, regardless the size. Turnaround time expectations are the same despite the size of the healthcare system, she added.

These professionals are hopeful that automation can help fill the gap. Automation makes it possible to deliver high throughput, Wong said.

Automation has been critical to our success at Grady Hospital in Atlanta, said Koch, adding later that automation helps all staff, even part-time and weekend staff. They have to produce the same high-quality result as the Monday-Friday staff.

Automation may also enable labs to maintain staff engagement. Weve found that we needed our techs doing clinical scientific lab work, not mundane tasks such as specimen processing and loading them into instruments, said Ades. Koch agreed: Graduates want to be involved in meaningful work. Getting the result to the doctor that will benefit the patient is the most important, rather than fussing over minutia that could be handled by automation.

If staff is satisfied and happy, they are more likely to stay, said Wong.

However, todays emerging automation solutions mean more than just moving samples around in a lab. Automation can also contribute to efficiency in processing and results delivery. AutomatedIT allows labs"to be more fail safe in terms of labeling and specimen errors, said Ades. AddedKoch: We can rely on IT to auto-validate much of the data.

To support such automation needs, Beckman Coulters newest solution automates sample handling and more. At AACC Beckman Coulter highlighted WorkflowConnect, WorkflowAdvanced, and WorkflowCommand solutions that are designed to meet the needs of small-, mid-, and high-volume laboratories. It also demonstrated its newest total laboratory automation solution, the DxA 5000, which is intended to improve efficiency through several onboard funtions that help identify and address pre-analytical errors, automate labor-intensive and error-prone laboratory tasks, and auto-verify and release normal results.

The DxA 5000 combines sample-processing hardware with informatics from WorkflowConnect, WorkflowAdvanced, and WorkflowCommand middleware. Tubes are bar coded and after being loaded into the system move through centrifugation and other processes. Data is captured on the status of each sample as it moves through processing, and results are captured and transmitted as soon as available.

Automation used to be moving a tube from A to B, explained Dr. Christoph Moellers, vice president and general manager of Beckman Coulters Workflow and Informational Technology Solutions Business. Now, we receive a tube and deliver a resulteverything in between is automated. We used to focus on push mechanismsa tube comes in and we push it down the line. Now we use a pull mechanismwe pull requests to analyzers to maximize operational throughput.

"We didnt have the software in place before to perform that pull, " he added. Now, "we have that connection in the middleware.

Ades appreciates such capability. Its the scalability of the data flow, she said. The middleware piece is important for us to be standardized across our systems.

In addition, the automated system features a minimum amount of failure points in the system to maintain uptime, Moellers said. He said that Beckman Coulter had interviewed 180 customers globally and found that labs are challenged to maintain short turnaround time with consistent results.

Beckman Coulters solutions are also intended to meet the needs of labs of varying size. We believe in automation for all, so we designed the system to be modular, so we can scale it, he said. Mid-sized hospitals dont always have the buying power or the space in terms of footprint.

Beckman Coulters focus on data could also help it meet the needs of both centralized and decentralized laboratories. I am sure whatever direction [trends go], you need a tool to connect the dots and the data. Thats why informatics is so critical, Moellers said.

Added Koch: The key is that the lab houses the data and provides the data to the clinician. Automated solutions help small and large labs and all those in between.

Wong believes that automation could support emerging diagnostics testing such as multiple-panel biomarker testing, personalized medicine, and genomics and enhance the delivery of that information.

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Supporting the Digital Movement of Diagnostics Data - Medical Device and Diagnostics Industry

BANGALORE, India, Nov. 18, 2019 /PRNewswire/ -- Molecular diagnostic screening is used to identify different DNA or RNA sequences, including single nucleotide polymorphism (SNP), deletions, rearrangements, insertions, and others for further detection of the disease. These combined methods, such as genome and proteome, helps examine biological markers at the molecular level. These methods are used to treat various infectious diseases

It is expected that technological advances in molecular diagnostics will drive the market dramatically as they allow for greater precision, portability and cost-effectiveness. The launch of MinION, a compact and inexpensive sequencer for users in point-of-care facilities and small peripheral laboratories by Nanopore Technologies, is expected to fuel market growth.

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Trends Influencing The Molecular Diagnostics Market Share

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Region Wise Analysis Of Molecular Diagnostics Market Share

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Top Investment Pockets

The global molecular diagnostics market is segmented by application into infectious diseases, oncology, genetic testing, blood screening, and others (microbiology, neurological diseases, and cardiovascular diseases).

Infectious diseases projected the highest revenue in 2016, with a CAGR of 9.3% for the forecast period of 2017-2023, owing to an increase in the number of patients suffering from different viral and bacterial infections, such as Human Immunodeficiency Virus (HIV), Hepatitis C virus(HCV), Human Papillomavirus(HPV), Chlamydia trachomatis/ Neisseria gonorrhoeae (CT/NG), and others. Along with the improving healthcare facilities treating these diseases in the emerging nations.

Molecular Diagnostics Market Key Segments

By Product

By Technology

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Global Molecular Diagnostics Market Size was Valued at 5,962 Million US$ in 2016 and is Expected to Reach 10,557 Million US$ with a CAGR of 9.1% by...

Here are the top stories covered by DocWire Newsthis week in the Hematology & Oncology section. This week, a report indicated that where you live impacts your chances of surviving lung cancer, ruxolitinib is associated with worsening cardiometabolic health, and more.

Men with theBRCA2mutation have an increased risk ofprostate cancercompared with non-BRCA2carriers and should undergo regular prostate-specific antigen testing, according to interim research from the IMPACT study, presented at the National Cancer Research Institute 2019 Cancer Conference. The results were also published inEuropean Urology.

In the past decade, newlung cancercases have decreased by 19% and five-year survival has increased by 26%, according to a promising report from theAmerican Lung Association. Despite this,lung cancerremains the leading cause of death in the United States, and the chance of surviving the disease is largely dictated by where a person lives.

Secondary surgical cytoreduction followed by chemotherapy does not extend overall survival (primary endpoint) compared with chemotherapy alone in patients with platinum-sensitive, recurrent ovarian cancer, according to a study published inThe New England Journal of Medicine.

Melanoma of the eye may soon be detected using a simple test, according to a study thatappeared inTranslational Vision Science & Technology.

Ruxolitinib, an oral JAK1/2 inhibitor approved by the U.S. Food and Drug Administration for the treatment of myeloproliferative neoplasms, is associated with worseningcardiometabolic health, according to an article published inScientific Reports.

In case you missed it, more hem/onc headlines are featured below:

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Hem/Onc Roundup: BRCA2 Mutation and Prostate Cancer, Cancer Drug Affects Cardio Health, and more - DocWire News

The stock of Seneca Biopharma, Inc. (NASDAQ:SNCA) hit a new 52-week low and has $0.79 target or 4.00 % below todays $0.83 share price. The 7 months bearish chart indicates high risk for the $3.19 million company. The 1-year low was reported on Nov, 18 by Barchart.com. If the $0.79 price target is reached, the company will be worth $127,600 less.The 52-week low event is an important milestone for every stock because it shows very negative momentum and is time when sellers come in. During such technical setups, fundamental investors usually stay away and are careful buying the stock.

The stock decreased 10.91% or $0.101 during the last trading session, reaching $0.825. About 143,863 shares traded. Seneca Biopharma, Inc. (NASDAQ:SNCA) has 0.00% since November 18, 2018 and is . It has by 0.00% the S&P500.

More notable recent Seneca Biopharma, Inc. (NASDAQ:SNCA) news were published by: Seekingalpha.com which released: 32 Stem Cell Companies to Watch Seeking Alpha on February 04, 2009, also Seekingalpha.com with their article: An Early Valuation Of Neuralstems NSI-189 For MDD Seeking Alpha published on June 25, 2014, Seekingalpha.com published: 15 Biotech Names For 2015 Seeking Alpha on January 05, 2015. More interesting news about Seneca Biopharma, Inc. (NASDAQ:SNCA) were released by: Finance.Yahoo.com and their article: The Daily Biotech Pulse: Therapix Calls Off Merger, Alimeras Reverse Split, 2 Biotechs To Debut Yahoo Finance published on November 08, 2019 as well as Seekingalpha.coms news article titled: Is It Time To Invest In Stem Cell Biotechs? Seeking Alpha with publication date: June 03, 2016.

Seneca Biopharma, Inc., a clinical stage biopharmaceutical company, focuses on the research and development of nervous system therapies based on its proprietary human neuronal stem cells and small molecule compounds. The company has market cap of $3.19 million. The company's stem cell based technology enables the isolation and expansion of human neural stem cells from various areas of the developing human brain and spinal cord enabling the generation of physiologically relevant human neurons of various types. It currently has negative earnings. The Companys lead product candidate is NSI-189, a chemical entity, which has been completed Phase II clinical trial for the treatment of major depressive disorder, as well as is in preclinical study for the treatment-refractory depression, Angelman Syndrome, Alzheimer's disease, ischemic stroke, diabetic neuropathy, irradiation-induced cognitive deficit, and long-term potentiation enhancement.

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After Touching 52-Week Low, Is Seneca Biopharma, Inc. (SNCA)'s Near-Term Analysis Negative? - FinanceRecorder

The circulating tumor cell (CTC) market report analytically evaluates the most important issues that may appear on the market in relation to sales, exports / imports or imports. The circulating tumor cell (CTC) market analysis described in this report provides a mixed test of segments to witness the fastest development of predicted prediction frames. This report not only displays market segmentation in the most granular pattern, but also thoroughly analyzes patents and key market players to provide a competitive advantage. In this circulating tumor cell (CTC) market report, we collected data using a large sample size data collection module and ran a baseline year analysis.

The major players in the global circulating tumor cells (CTC) market are Thermo Fisher Scientific Inc., STEMCELL Technologies Inc, SRI International, QIAGEN, NanoString Technologies, Inc., Miltenyi Biotec Gmbh, Menarini Silicon Biosystems., Ikonisys Inc., Greiner Bio One International GmbH, General Electric, F. Hoffmann-La Roche Ltd, BioCep Ltd., Precision Medicine Group, Creatv MicroTech, Inc., Aviva Biosciences and Advanced Cell Diagnostics, Inc.

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Circulating Tumor Cells (CTC) Market is expected to grow globally with an estimated CAGR of 15.45% to reach USD 27.80 billion by 2028.Market growth is growing due to the expansion of cluster chip technology. Another major factor driving the market is the increasing need for cancer diagnostic treatment.

This circulating tumor cell (CTC) market report is used as an excellent market report because it is generated by several important factors. The circulating tumor cell (CTC) market research report also provides company profile, product specifications, production value, manufacturer contact information and company market share. The circulating tumor cell (CTC) market report conducts analysis and discussion of important market trends, market size, sales volume and market share in the semiconductor and electronics industry. The Circulating Tumor Cell (CTC) Market Report provides a description of market definition, market segmentation, industry-specific research on key developments and competitive analysis of the circulating tumor cell (CTC) market. Research methodology with exceptional tools and skills.

The Circulating Tumor Cells (CTC) Market is segmented based on Technology

o Immunocytochemical Technologies

o Molecular (RNA)-Based Technologies

o Functional In vitro Cell Invasion Assay

o Xenotransplantation Models

o Ex Vivo Positive Selection

o In Vivo Positive Selection

o Negative Selection

o Microchips

o Single Spiral Microchannel

The Circulating Tumor Cells (CTC) Market is segmented based on Application

The Circulating Tumor Cells (CTC) Market is segmented based on End User

Geologically, this report is divided into a few key regions:

Chapter 1: Circulating Tumor Cells (CTC) Market Methodology & Scope

Definition and forecast parameters

Methodology and forecast parameters

Data Sources

Chapter 2: Circulating Tumor Cells (CTC) Market Executive Summary

Business trends

Regional trends

Product trends

End-use trends

Chapter 3: Circulating Tumor Cells (CTC) Market Industry Insights

Industry segmentation

Industry landscape

Vendor matrix

Technological and innovation landscape

Chapter 4: Circulating Tumor Cells (CTC) Market, By Region

Chapter 5: Circulating Tumor Cells (CTC) Market Company Profile

Business Overview

Financial Data

Product Landscape

Strategic Outlook

SWOT Analysis

Key advantage of Circulating Tumor Cells (CTC) Market -:

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Circulating Tumor Cells (CTC) Market To Reach USD 27.80 billion by 2028 : Thermo Fisher Scientific Inc., STEMCELL Technologies Inc, SRI International,...

Inadequate prediction of drug metabolism or toxicity is the Achilles heel of the pharmaceutical industry, leading to high drug attrition rates. This can have significant implications for companies, with late-stage failure typically resulting in the loss of substantial financial investments, time and resources. By defining and predicting the Absorption, Distribution, Metabolism, Excretion and Toxicity (ADME-Tox) profile of compounds as early as possible, companies can focus their resources on the most relevant candidates to increase the likelihood of successfully bringing new safe and efficient therapies to the market.To implement early ADME-Tox testing, its crucial to have access to valid in vitro models of the key sites of drug metabolism the liver, kidneys and intestines. Preclinical models that reliably replicate the in vivo cellular environment of these organs enable scientists to study and accurately predict drug metabolism, transport and toxicity prior to targets entering the clinical trial phase, ultimately helping to lower attrition rates.

For many years, biologists have relied on in vitro 2-dimensional (2D) cell culture models to perform preclinical ADME-Tox assays. While these models are useful for informing aspects of drug discovery, such as cytotoxicity, they are limited in their translatability to drug metabolism and toxicity in humans. Recently, in vitro 3-dimensional (3D) organoid models that more closely mimic human biological systems show promise in supporting ADME-Tox studies in the early stages of the drug development process, compared to their 2D counterparts.

For modeling kidney cell function in vitro and to evaluate general nephrotoxicity, researchers use various types of human proximal tubule cell lines. These cells demonstrate specific properties of the kidney epithelium, such as the transport of solutes, which plays an important role in drug excretion,. Limitations of these in vitro models are due to the different cell types not expressing all necessary transporters, metabolizing enzymes or biomarkers at physiological levels.3

Given these challenges, there is a clear need for advanced in vitro 3D models that more accurately emulate drug permeability, metabolism, transport and toxicity in humans.

Due to a greater understanding of the cell microenvironment, organoid technology has advanced over the last few years and has the potential to streamline the drug development process. Using organoids to support in vitro ADME-Tox studies can help to predict metabolic mechanisms and ascertain key safety and efficacy measures before commencing human clinical trials. Evidence suggests that kidney and intestine organoid models could be more valuable to investigate the metabolism, transport and toxicity of drugs.3,4 Indeed, a kidney organoid model has recently been cultured using human induced pluripotent stem cells. Composed of both glomerular tissue, as well as proximal and distal tubule cells, this model offers a more accurate representation of the kidney. As such, it has the potential to inform and refine preclinical toxicity screening studies.3

In another recent study, human primary cells from intestinal epithelium was engineered into a 3D intestinal organoid using a scaffold system [4]. This model showed complex tissue properties and characteristics of mature epithelium, including the four main types of differentiated epithelial cells (enterocytes, goblet cells, paneth cells and enteroendocrine cells). The tight junction formation, microvilli polarization, digestive enzyme secretion and low oxygen tension in the lumen were also representative of mature epithelium. In addition to these physical properties, the organoids also exhibited complex behavior, such as innate antibacterial responses to E. coli similar to those observed in patients with inflammatory bowel disease (IBD). This suggests that the model could be used in in vitro studies investigating host-microbe-pathogen interplay and IBD pathogenesis.

These findings highlight the future potential of in vitro 3D kidney and intestine organoids for drug development. Because these systems closer resemble in vivo tissues, they could help predict drug responses early in development and offer vast possibilities for modeling many diseases in the future.

1. Van Breemen R.B and Li Y. Caco-2 cell permeability assays to measure drug absorption. Expert Opin Drug Metab Toxicol. 2005 Aug;1(2):17585

2. Yamaura Y, et al. Functional Comparison of Human Colonic Carcinoma Cell Lines and Primary Small Intestinal Epithelial Cells for Investigations of Intestinal Drug Permeability and First-Pass Metabolism. Drug Metabolism and Disposition. March 2016;44:329335

3. Bajaj P, et al. Emerging Kidney Models to Investigate Metabolism, Transport, and Toxicity of Drugs and Xenobiotics. Drug Metabolism and Disposition. November 2018;46(11):16921702

4. Chen Y, et al. In vitro enteroid-derived three-dimensional tissue model of human small intestinal epithelium with innate immune responses. PLoS One. 2017;12(11): e0187880

Read more:
The Promise of in vitro 3D Organoid Models: Meeting the ADME-Tox Testing Needs of the Pharmaceutical Industry - Technology Networks