StemCell Therapy

Since 1996, Carla Hutton has monitored, researched, and written about regulatory and legislative issues that may potentially affect Bergeson & Campbell, P.C. (B&C) clients. She is responsible for creating a number of monthly and quarterly regulatory updates for B&C's clients, as well as other documents, such as chemical-specific global assessments of regulatory developments and trends. She authors memoranda for B&C clients on regulatory and legislative developments, providing information that is focused, timely and applicable to client initiatives. These tasks have proven invaluable to many clients, keeping them aware and abreast of developing issues so that they can respond in kind and prepare for the future of their business.

Ms. Hutton brings a wealth of experience and judgment to her work in federal, state, and international chemical regulatory and legislative issues, including green chemistry, nanotechnology, the Federal Insecticide, Fungicide, and Rodenticide Act (FIFRA), the Toxic Substances Control Act (TSCA), Proposition 65, and the Registration, Evaluation, Authorization and Restriction of Chemicals (REACH) program.

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Nanotoxicity Studies May Be Affected by Nanoparticles in Syringes - The National Law Review

A recent market study published by QY Research consists of a detailed assessment of the key market dynamics. The report provides the historical as well as present growth parameters of the global Nanobots market. The report features salient and unique factors, which are expected to significantly impact the growth of the global Nanobots market throughout the forecast period.

Scope of the report:

The report commences with a scope of the global Nanobots market that includes the key findings and vital statistics of the market. This market research report also consists of the market value of the major segments of the global Nanobots market. QY Research has found a detailed taxonomy and the definition of the global market that helps the readers to better understand the basic information of the Nanobots market. It also highlights the exclusions and inclusions that help the client to understand the scope of the Nanobots market.

The report consists of key market trends, which are likely to impact the growth of the market over the forecast period. Evaluation of in-depth industry trends is included in the report, along with their product innovations and key market growth.

QY Research report also includes the macro-economic factors, which are likely to influence the growth of the global Nanobots market during the forecast period. Along with the factors, the report also analyzes the growth opportunities of the global Nanobots market. It sheds light on the trends, restraints, and drivers to understand the growth prospects followed by the key players in the global Nanobots market.

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Competitive Landscape:

The report provides a list of all the key players in the Nanobots market along with a detailed analysis of the strategies, which the companies are adopting. The strategies mainly include new product development, research, and development, and also provides revenue shares, company overview, and recent company developments to remain competitive in the market.

The Nanobots key manufacturers in this market include:

Xidex CorpZymergen IncSynthace LimitedGinkgo BioworksAdvanced Diamond TechnologiesAdvanced Nano Products Co Limited

Segment Analysis:

The report provides detailed segments based on product type and applications so that the readers can better understand each segment that influences the Nanobots market growth.

By the product type, the market is primarily split into

Microbivore NanorobotsRespirocyte NanorobotsClottocyte NanorobotsCellular Repair Nanorobots

By the end users/application, this report covers the following segments

Nano MedicineBiomedicalMechanicalOther applications

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Geographical Outlook:

In 2018, the global Nanobots market size was increased to xx million US$ from xx million US$ in 2014, and it will reach xx million US$ in 2025, growing at CAGR of xx%% between 2019 and 2025.

QY Research report provides a detailed information to the clients about the various factors that are impacting on the growth of the regions across North America (United States, Canada and Mexico), Asia Pacific (China, Japan, South Korea, India, Australia, Indonesia, Thailand, Malaysia, Philippines and Vietnam), Middle East and Africa (Turkey, GCC Countries, Egypt and South Africa), and South America (Brazil and others).

About Us:

QYResearch always pursuits high product quality with the belief that quality is the soul of business. Through years of effort and supports from huge number of customer supports, QYResearch consulting group has accumulated creative design methods on many high-quality markets investigation and research team with rich experience. Today, QYResearch has become the brand of quality assurance in consulting industry.

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“Global Non-woven Table Linen Market Size Growth, Development and Opportunities in 2019-2025: Intexcomfort , Mungo , Jomar, Inc. , Linomeda etc.”

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Global Nanobots Market Size by forecasting the high Growth Segments 2019-2025: Xidex Corp, Zymergen Inc, Synthace Limited, Ginkgo Bioworks, Advanced...

DUBLIN--(BUSINESS WIRE)--The "Nanobiotechnologies - Applications, Markets & Companies" report from Jain PharmaBiotech has been added to ResearchAndMarkets.com's offering.

Nanotechnology is the creation and utilization of materials, devices, and systems through the control of matter on the nanometer-length scale (a nanometer is one billionth of a meter. Nanobiotechnology, an integration of physical sciences, molecular engineering, biology, chemistry and biotechnology holds considerable promise of advances in pharmaceuticals and healthcare.

The report starts with an introduction to various techniques and materials that are relevant to nanobiotechnology. It includes some of the physical forms of energy such as nanolasers. Some of the technologies are scaling down such as microfluidics to nanofluidic biochips and others are constructions from bottom up. Application in life sciences research, particularly at the cell level sets the stage for role of nanobiotechnology in healthcare in subsequent chapters.

Some of the earliest applications are in molecular diagnostics. Nanoparticles, particularly quantum dots, are playing important roles. In-vitro diagnostics, does not have any of the safety concerns associated with the fate of nanoparticles introduced into the human body. Numerous nanodevices and nanosystems for sequencing single molecules of DNA are feasible. Various nanodiagnostics that has been reviewed will improve the sensitivity and extend the present limits of molecular diagnostics.

An increase in the use of nanobiotechnology by the pharmaceutical and biotechnology industries is anticipated. Nanotechnology will be applied at all stages of drug development - from formulations for optimal delivery to diagnostic applications in clinical trials. Many of the assays based on nanobiotechnology will enable high-throughput screening.

Some of the nanostructures such as fullerenes are themselves drug candidates as they allow precise grafting of active chemical groups in three-dimensional orientations. The most important pharmaceutical applications are in drug delivery. Apart from offering a solution to solubility problems, nanobiotechnology provides and intracellular delivery possibilities. Skin penetration is improved in transdermal drug delivery. A particularly effective application is as nonviral gene therapy vectors. Nanotechnology has the potential to provide controlled release devices with autonomous operation guided by the needs.

Nanomedicine is now within the realm of reality starting with nanodiagnostics and drug delivery facilitated by nanobiotechnology. Miniature devices such as nanorobots could carry out integrated diagnosis and therapy by refined and minimally invasive procedures, nanosurgery, as an alternative to crude surgery. Applications of nanobiotechnology are described according to various therapeutic systems.

Nanotechnology will markedly improve the implants and tissue engineering approaches as well. Of the over 1,000 clinical trials of nanomedicines, approximately 100 are selected and tabulated in major therapeutic areas. Other applications such as for management of biological warfare injuries and poisoning are included. Contribution of nanobiotechnology to nutrition and public health such as supply of purified water are also included.

There is some concern about the safety of nanoparticles introduced in the human body and released into the environment. Research is underway to address these issues. As yet there are no FDA directives to regulate nanobiotechnology but as products are ready to enter market, these are expected to be in place.

Future nanobiotechnology markets are calculated on the basis of the background markets in the areas of application and the share of this market by new technologies and state of development at any given year in the future. This is based on a comprehensive and thorough review of the current status of nanobiotechnology, research work in progress and anticipated progress.

There is definite indication of large growth of the market but it will be uneven and cannot be plotted as a steady growth curve. Marketing estimates are given according to areas of application, technologies and geographical distribution starting with 2018. The largest expansion is expected between the years 2023 and 2028.

Profiles of 252 companies, out of over 500 involved in this area, are included in the last chapter along with their 185 collaborations. The report is supplemented with 51 Tables, 32 figures and 800 references to the literature.

For more information about this report visit https://www.researchandmarkets.com/r/lrcr1q

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Nanobiotechnologies Applications, Markets & Companies Analysis 2019 - Updated Forecasts to 2023 & 2028 - ResearchAndMarkets.com - Business...

According to Stratistics MRC, the GlobalNanorobotics Marketis accounted for $4.10 Billion in 2017 and is expected to reach $11.88 Billion by 2026 growing at a CAGR of 12.5% during the forecast period. Growing application of nanotechnology and regenerative medicine, rising acceptance and preferment of entrepreneurship and increasing investments by government and universities are the key factors fuelling the market growth. However, high manufacturing cost may hinder the growth of the market.

Nanorobotics is an evolving technology arena that creates robots or machines which have machinery near to the scale of a nanometre (109 meters). It denotes the nanotechnology engineering regulation of planning, designing, and building nanorobots, primarily from molecular components. Nanorobotics is an attractive new field, especially in medicine, which focus on directed drug delivery using nanoscale molecular machines.

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By Type, Nanomanipulator is expected to hold considerable market growth during the forecast period. Nanomanipulator is a specialized nanorobot and microscopic viewing system for working with objects on an extremely small scale. Nanomanipulators are mainly used to influence the atoms and molecules and were among the first nanorobotic systems to be commercially accessible. By geography, Europe dominated the highest market share due to rising aging population and rising governmental healthcare expenditure.

Some of the key players in Nanorobotics include Bruker, JEOL, Thermo Fisher Scientific, Ginkgo Bioworks, Oxford Instruments, EV Group, Imina Technologies, Toronto Nano Instrumentation, Klocke Nanotechnik, Kleindiek Nanotechnik, Xidex, Synthace, Park Systems, Smaract and Nanonics Imaging

Types Covered: Nanomanipulator Magnetically Guided Bacteria-Based Bio-Nanorobotics

Applications Covered: Biomedical Nanomedicine Mechanical Other Applications

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Regions Covered: North Americao USo Canadao Mexico Europeo Germanyo UKo Italyo Franceo Spaino Rest of Europe Asia Pacifico Japano Chinao Indiao Australiao New Zealando South Koreao Rest of Asia Pacific South Americao Argentinao Brazilo Chileo Rest of South America Middle East & Africao Saudi Arabiao UAEo Qataro South Africao Rest of Middle East & Africa

What our report offers: Market share assessments for the regional and country level segments Market share analysis of the top industry players Strategic recommendations for the new entrants Market forecasts for a minimum of 9 years of all the mentioned segments, sub segments and the regional markets Market Trends (Drivers, Constraints, Opportunities, Threats, Challenges, Investment Opportunities, and recommendations) Strategic recommendations in key business segments based on the market estimations Competitive landscaping mapping the key common trends Company profiling with detailed strategies, financials, and recent developments Supply chain trends mapping the latest technological advancements

Free Customization Offerings:All the customers of this report will be entitled to receive one of the following free customization options: Company Profilingo Comprehensive profiling of additional market players (up to 3)o SWOT Analysis of key players (up to 3) Regional Segmentationo Market estimations, Forecasts and CAGR of any prominent country as per the clients interest (Note: Depends of feasibility check) Competitive Benchmarkingo Benchmarking of key players based on product portfolio, geographical presence, and strategic alliances

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Nanorobotics Market to receive overwhelming hike in Revenues by 2026 - Guru Online News

SEATTLE--(BUSINESS WIRE)--Omeros Corporation (Nasdaq: OMER) today announced positive data from its pivotal clinical trial of the companys novel investigational complement inhibitor narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), a frequently lethal complication of HSCT. These preliminary data were recently provided to FDA as part of the companys ongoing interactions with the Agency on the narsoplimab Biologics License Application (BLA). All safety and efficacy endpoints including the composite primary endpoint and the secondary endpoints are agreed with FDA. The reported data support a strongly positive benefit-risk balance.

The primary efficacy endpoint in this single-arm open-label trial of HSCT-TMA patients is the proportion of patients who achieve a highly rigorous set of response criteria that requires both improvement in HSCT-TMA laboratory markers and improvement in clinical status (organ function and transfusions). Patients who did not fully meet these criteria were considered non-responders. The secondary endpoints include survival rates and change from baseline in HSCT-TMA laboratory markers. Consistent with the pre-specified statistical analysis plan for the trial, the primary and secondary endpoints are assessed for (1) all patients who received at least one dose of narsoplimab and (2) patients who received at least 4 weeks of narsoplimab dosing. Patients enrolled in this trial had a high expected mortality rate. In severe cases of HSCT-TMA, mortality can exceed 90 percent.

Primary Efficacy Endpoint:

Secondary Endpoints:

Safety:

The HSCT-TMA patient population enrolled in this trial had multiple high-risk features that portend a poor outcome. These include persistence of HSCT-TMA despite modification of immunosuppression (which was a criterion for entry into the trial), graft-versus-host disease, significant infections, non-infectious pulmonary complications and neurological findings. Patients in the trial had a high expected death rate, with 93 percent of them having multiple risk factors.

Patient enrollment in the pivotal trial has been completed. The details of the endpoints, including the response criteria agreed with FDA, and the number of patients in the trial remain confidential for competitive business reasons.

Last year the company reported data on 19 HSCT-TMA patients treated with narsoplimab on which FDA granted breakthrough therapy designation. The results reported today are even stronger. The response rate remains equally high at 56 percent, while the 100-day survival has improved from 53 percent to 65 percent.

The response rate in this high-risk population would be expected to be 10 to 15 percent with a 100-day survival rate of less than 20 percent. The response rate and 100-day survival achieved with narsoplimab in this trial demonstrate an unprecedented effect in this condition, said Rafael Duarte M.D., Ph.D., F.R.C.P., Associate Professor, Head of Hematology Department and Hematopoietic Transplantation Program, University Hospital Puerta de Hierro Majadahonda, Madrid, Spain, and Secretary of the European Society for Blood and Marrow Transplantation. The other secondary endpoints are equally impressive. The data are consistent with my personal experience with narsoplimab. Patients with severe forms of HSCT-TMA have a dismal prognosis with no treatment currently available. I expect a treatment with this profile would be widely adopted for use in these patients and even lead to increased physician recognition of the disorder.

Omeros reported the initiation of its rolling BLA in October. Narsoplimab, also referred to as OMS721, is Omeros lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2) and has breakthrough therapy designation from FDA for this indication.

The striking results seen in our pivotal trial are tremendously gratifying, said Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. Our rolling BLA is underway the nonclinical sections have been submitted and the data from this trial form the efficacy basis of the application. We continue to compile the remaining sections of the BLA and look forward to continued partnership with regulators to make narsoplimab widely available for the treatment of this devastating condition.

Data from this pivotal trial will also support the narsoplimab marketing authorization application for HSCT-TMA in Europe. The data are planned for publication and for presentation at international congresses in the first part of 2020.

In addition to breakthrough therapy designation from FDA, narsoplimab has orphan drug designation in both the U.S. and Europe for HSCT-TMA. Narsoplimab also has been awarded breakthrough therapy designation for immunoglobulin A nephropathy (IgAN), and Omeros has Phase 3 programs for narsoplimab ongoing in IgAN and in atypical hemolytic uremic syndrome (aHUS).

Conference Call and Webcast Details

Omeros management will host a webcast and conference call to present data from its pivotal trial of narsoplimab in HSCT-TMA. The call will be held today at 8:30 a.m. Eastern Time; 5:30 a.m. Pacific Time. To access the live conference call via phone, please dial (844) 831-4029 from the United States and Canada or (920) 663-6278 internationally. The participant passcode is 3774209. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available for one week following the call and may be accessed by dialing (855) 859-2056 from the United States and Canada or (404) 537-3406 internationally. The replay passcode is 3774209.

To access the live or subsequently archived webcast and presentation materials on the internet, go to the companys website at http://www.omeros.com and select Events under the Investors section of the website. To access the live webcast, please connect to the website at least 15 minutes prior to the call to allow for any software download that may be necessary.

About Omeros Corporation

Omeros is an innovative biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers. In addition to its commercial product OMIDRIA (phenylephrine and ketorolac intraocular solution) 1%/0.3%, Omeros has multiple Phase 3 and Phase 2 clinical-stage development programs focused on complement-mediated disorders and substance abuse. In addition, the company has a diverse group of preclinical programs including GPR174, a novel target in immuno-oncology that modulates a new cancer immunity axis recently discovered by Omeros. Small-molecule inhibitors of GPR174 are part of Omeros proprietary G protein-coupled receptor (GPCR) platform through which it controls 54 new GPCR drug targets and their corresponding compounds. The company also exclusively possesses a novel antibody-generating platform.

About HSCT-TMA

Hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) is a significant and often lethal complication of stem cell transplants. This condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by conditioning regimens, immunosuppressant therapies, infection, GvHD, and other factors associated with stem cell transplantation. Endothelial damage, which activates the lectin pathway of complement, plays a central role in the development of HSCT-TMA. The condition occurs in both autologous and allogeneic transplants but is more common in the allogeneic population. In the United States and Europe, approximately 25,000 to 30,000 allogeneic transplants are performed annually. Recent reports in both adult and pediatric allogeneic stem cell transplant populations have found an HSCT-TMA incidence of approximately 40 percent, and high-risk features may be present in up to 80 percent of these patients. In severe cases of HSCT-TMA, mortality can exceed 90 percent and, even in those who survive, long-term renal sequalae are common. There is no approved therapy or standard of care for HSCT-TMA.

About Narsoplimab

Narsoplimab, also known as OMS721, is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), a novel pro-inflammatory protein target and the effector enzyme of the lectin pathway of complement. Importantly, inhibition of MASP-2 does not appear to interfere with the antibody-dependent classical complement activation pathway, which is a critical component of the acquired immune response to infection. Omeros controls the worldwide rights to MASP-2 and all therapeutics targeting MASP-2.

Phase 3 clinical programs are in progress for narsoplimab in hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), in immunoglobulin A (IgA) nephropathy, and in atypical hemolytic uremic syndrome (aHUS). The FDA has granted narsoplimab breakthrough therapy designations for HSCT-TMA and for IgA nephropathy; orphan drug status for the prevention (inhibition) of complement-mediated thrombotic microangiopathies, for the treatment of HSCT-TMA and for the treatment of IgA nephropathy; and fast track designation for the treatment of patients with aHUS. The European Medicines Agency has granted orphan drug designation to narsoplimab for treatment in HSCT and for treatment of primary IgA nephropathy.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, which are subject to the safe harbor created by those sections for such statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, likely, look forward to, may, on track, plan, potential, predict, project, prospects, scheduled, should, slated, targeting, will, would and similar expressions and variations thereof. Forward-looking statements, including statements regarding anticipated regulatory submissions, expectations regarding regulatory exclusivities, the timing and results of ongoing or anticipated clinical trials, and the therapeutic application of Omeros investigational product, are based on managements beliefs and assumptions and on information available to management only as of the date of this press release. Omeros actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, availability and timing of data from clinical trials and the results of such trials, unproven preclinical and clinical development activities, regulatory oversight, intellectual property claims, competitive developments, litigation, and the risks, uncertainties and other factors described under the heading Risk Factors in the companys Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 1, 2019. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the company assumes no obligation to update these forward-looking statements, whether as a result of any new information, future events or otherwise, except as required by applicable law.

Source: Omeros Corporation

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Omeros Reports Positive Data Across Primary and Secondary Endpoints in Pivotal Trial of Hematopoietic Stem Cell Transplant-Associated Thrombotic...

NEW YORK, Dec. 4, 2019 /PRNewswire/ --

Hunter Syndrome Treatment Market Size, Share & Trend Analysis By Treatment (Enzyme Replacement Therapy, Hematopoietic Stem Cell Transplant), By Region, And Segment Forecasts, 2019 - 2026

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The global hunter syndrome treatment market size is expected to reach a value of USD 1.52 billion by 2026, expanding at a CAGR of 7.1%. High unmet needs, robust pipeline, increasing awareness about this rare disease and growing R&D activities for the development of novel therapies are expected to drive market growth over the forecast period.

Hunter syndrome, also referred as mucopolysaccharidosis type II (MPS II), is a rare genetic disorder caused by the missing or malfunctioning iduronate-2-sulfatase enzyme. According to the data published by the National Institute of Neurological Disorders and Stroke, MPS II syndrome occurs in around 1 in every 100,000 to 150,000 male births.

Presently, there are no approved curative therapies for the treatment of Hunter syndrome.The available treatment options such as enzyme replacement therapy (ERT) and hematopoietic stem cell transplant (HSCT) are focused on providing symptomatic relief and management of complications associated with disease progression.

Shire plc's Elaprase (idursulfase) is the only key drug available for the treatment of Hunter syndrome worldwide, with GC Pharma's Hunterase (idursulfase beta) being approved only in South Korea.

Key players are focused on extensive R&D activities for product development and gaining approval as novel therapies.Launch of such novel therapies in the near future is expected to significantly fuel the Hunter syndrome treatment market growth.

For instance, in May 2018, REGENXBIO Inc. received the U.S. FDA's Fast Track designation for its novel drug candidate RGX-121, indicated for the disease treatment.

Further Key Findings from the Study Suggest: The enzyme replacement therapy segment has acquired the largest share in 2018, owing to the increased adoption of Elaprase and the potential approval of Hunterase worldwide The absence of curative therapies for MPS II creates lucrative opportunities for the key players in the market for developing new therapies The launch of late-stage pipeline therapies is expected to drive the market growth during the forecast period Currently, Shire Plc. is one of the leading players, supported by strong sales of their marketed drug ELAPRASE for the disease treatment Major players in the market are adopting inorganic growth strategies such as partnerships and collaborations for the development and commercialization of novel therapies In 2018, North America held a dominant position in the global market, owing to favorable reimbursement scenario, high awareness regarding rare disorders, and presence of major players Some of the key companies in hunter syndrome treatment market include GC Pharma, Sangamo Therapeutics, Inc.; JCR Pharmaceuticals Co Ltd.; RegenxBio Inc.; and Shire Plc. (Takeda Pharmaceutical Company)

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Researchers at Karolinska Institutet have developed a new method to separate different types of a common herpes virus (HHV-6) that has been linked to multiple sclerosis (MS).

By analyzing antibodies in the blood against the most divergent proteins of herpesvirus 6A and 6B, these researchers were able to show that MS-patients carry the herpesvirus 6A to a greater extent than healthy individuals.

This is relevant news since as many as 80 percent of all children are infected with the HHV-6 virus before 2 years of age.

But since it hasnt been possible to tell the variants apart post-infection, it has been difficult to say whether HHV-6A or B is a risk factor for MS, said these researchers in a related press release.

The findings published on November 26, 2019, in Frontiers in Immunology, indicate a potential role for HHV-6A in MS vaccine development.

Multiple sclerosis is an autoimmune disease that affects the central nervous system (CNS). The cause of the disease is unclear, but one plausible explanation is a virus tricks the CNS to attack the bodys own tissue.

MS-related news article

HHV-6 has previously been associated with MS, but in those studies, it wasnt possible to distinguish between 6A and 6B.

HHV-6 infection has been associated with complications of varying severity in hematopoietic stem cell transplant recipients, to a lesser degree in solid organ transplant recipients, and in those who are otherwise immunosuppressed, says the HHV-6Foundation.org.

These researchers have been able to show that HHV-6B can cause mild conditions such as roseola in children, but it has been unclear if HHV-6A is the cause of any disease.

In this study, however, the researchers were able to distinguish between the A and B virus by analyzing antibodies in the blood against the proteinsimmediate-early protein 1A and 1B (IE1A and IE1B)that diverge the most between the 2 viruses.

This is a big breakthrough for both the MS and herpes virus research, says Anna Fogdell-Hahn, associate professor at the Department of Clinical Neuroscience at Karolinska Institutet and one of the studys senior authors, in this press release.

Recent herpes vaccine news

For one, it supports the theory that HHV-6A could be a contributing factor to the development of MS.

On top of that, we are now able, with this new method, to find out how common these different types of HHV-6 are, something we havent been able to do previously.

The researchers compared antibody levels in blood samples of some 8,700 MS-patients against more than 7,200 healthy people whose gender, date of birth, date of the blood sample and other factors matched those with MS.

They concluded that people with MS had a 55 percent higher risk of carrying antibodies against the HHV-6A protein than the control group.

In a sub-group of almost 500 people, whose blood samples were drawn before the onset of the disease, the risk of developing MS in the future was more than doubled if they had a 6A viral infection.

The younger the people were when the virus was first discovered in the blood, the higher the risk was of developing MS in the future.

HHV-6B, on the other hand, was not positively associated with MS.

Instead, MS-patients had lower levels of antibodies toward IE1B than those without MS.

Antibodies toward Epstein-Barr virus (EBV), another herpes virus that is also associated with MS, were analyzed with the same method and the researchers were able to show that individuals affected with both viruses had an even greater risk of MS.

This indicates that several virus infections could be acting jointly to increase the risk of MS.

Both HHV-6A and 6B can infect our brain cells, but they do it in slightly different ways.

Therefore, it is now interesting to go forward and attempt to map out exactly how the viruses could affect the onset of MS, says Anna Fogdell-Hahn.

The research has been financed by grants from the Swedish Research Council, Stockholm County Council, Swedish Brain Foundation, KAW Foundation, Margareta af Ugglas Foundation, MultipleMS Horizon 2020, Multiple Sclerosis Society of Canada and the Swedish Society of Medical Research. Some of the researchers have previously received grants/fees by pharmaceutical companies in various contexts.

Multiple Sclerosis news published by Precision Vaccinations

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Herpes Virus Linked to Multiple Sclerosis - PrecisionVaccinations

Newest Learn about at the International Nanomedicine Market

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As in keeping with the record, the worldwide Nanomedicine marketplace is projected to develop at a CAGR of ~XX% and exceed the worth of ~US$ against the tip of 2029. Additionally, an in-intensity research of the micro and macro-financial elements which are expected to steer the trajectory of the Nanomedicine marketplace all through the forecast length (2019-2029) is integrated within the record.

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A radical analysis of the long run possibilities of the Nanomedicine marketplace throughout more than a few areas is tracked within the record.

segmentation, and demanding situations out there were mentioned at duration within the analysis record.

Review of the Cloud Computing Market in Healthcare Trade

As in keeping with the analysis record, the worldwide marketplace for cloud computing within the healthcare business used to be valued at US$1.82 bn in 2011 and is projected to succeed in a worth of US$6.79 bn via the tip of 2018. The marketplace is projected to sign up a exceptional 21.30% CAGR between 2012 and 2018.

The safety of get entry to and knowledge integrity and several other different advantages presented via cloud computing are estimated to inspire the expansion of the marketplace in the following couple of years. Alternatively, information leakage considerations, information integration and consistency, and knowledge relocation are one of the key elements estimated to restrain the expansion of the worldwide cloud computing marketplace within the healthcare business in the following couple of years.

At the moment, the instrument-as-a-provider phase leads the worldwide cloud computing marketplace within the healthcare business. However, the platform-as-a-provider phase is estimated to witness considerable enlargement within the coming years. Moreover, at the foundation of finish use, the non-scientific knowledge techniques marketplace is projected to guide the worldwide marketplace within the close to long run.

A number of the key geographical segments, North The usa is estimated develop at a swift tempo and account for an enormous proportion within the world marketplace for cloud computing within the healthcare business in the following couple of years. The top enlargement of this area may also be attributed to the presence of numerous biopharmaceutical gamers. As well as, the emerging focal point on analysis and construction actions is anticipated to give a contribution widely against the expansion of the marketplace.

Firms Discussed within the Analysis Document

The world marketplace for cloud computing within the healthcare business is very fragmented in nature as no key gamers grasp greater than a proportion of 10% out there at this time. CareCloud, ClearDATA Networks, Oracle Company, Microsoft, Agfa Healthcare, IBM Company, and Merge Healthcare are one of the key gamers running within the world cloud computing marketplace within the healthcare business.

Key Segments of the International Cloud Computing marketplace in Healthcare Trade

International Cloud Computing marketplace in Healthcare Trade, via provider fashions

International Cloud Computing marketplace in Healthcare Trade, via pricing fashions

International Cloud Computing marketplace in Healthcare Trade, via finish customers

International Cloud Computing marketplace in Healthcare Trade, via Geography

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Robust Growth Of The Nanomedicine Market Predicted Over The Forecast Period 2013 - 2019 - Hitz Dairies

Yuehe Lin

Yuehe Lin, professor in the School of Mechanical and Materials Engineering, has been named a fellow of the National Academy of Inventors (NAI).

He was cited for his highly prolific spirit of innovation in creating or facilitating inventions that have made a tangible impact on the quality of life, economic development, and welfare of society, according to the selection committee. Lin is one of 168 NAI Fellows for 2019, which is the highest professional distinction accorded solely to academic inventors. The program includes more than 1,000 fellows from 250 universities around the world that hold more than 41,500 U.S. patents.

Dr Lin has made significant research contributions with real-world impact in the fields of energy and health, said Mary Rezac, dean of WSUs Voiland College of Engineering and Architecture. His hugely varied work from finding possible new ways to treat cancer to innovations in water splitting for a future hydrogen economy and development of a better catalyst for fuel cells often comes down to simple solutions that can provide real change and improvements in peoples lives.

With WSU since 2013, Lin, who also holds a joint appointment at Pacific Northwest National Laboratory, conducts research in nanotechnology, particularly development of small-scale devices, materials and analytical systems for biomedical diagnosis, drug delivery and energy and environmental applications.

He has more than 500 peer-reviewed publications, which have been cited more than 50,500 times, according to Google Scholar. He has an h-index, a measure of a scientists productivity and impact, of 112. He has been named among the worlds most highly cited researchers every year from 2014 to 2019 by the Web of Science Group.

He has received funding from the National Institutes of Health, U.S. Centers for Disease Control and Prevention and U.S. Departments of Energy and Defense. He holds more than 20 patents, some of which have been licensed to industrial partners for commercialization.

Lin is a fellow of the American Association for the Advancement of Science, Royal Society of Chemistry and American Institute of Medical and Biological Engineering as well as a member of the Washington State Academy of Sciences. He serves as editor or editorial board member for approximately 20 international journals, including Advance Materials Technologies; Analytica Chimica Acta; Biosensors and Bioelectronics; Electroanalysis; International Journal of Nanomedicine; Research; Journal of Nanoscience and Nanotechnology; and Sensors and Actuators B.

Lin will join the induction ceremony for fellows on April 10 as part of the annual Conference of the National Academy of Inventors in Phoenix, AZ.

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Yuehe Lin named to National Academy of Inventors - WSU News

The Healthcare Nanotechnology Market report gives a purposeful depiction of the area by the practice for research, amalgamation, and review of data taken from various sources. The market analysts have displayed the different sidelines of the area with a point on recognizing the top players (Amgen, Teva Pharmaceuticals, Abbott, UCB, Roche, Celgene, Sanofi, Merck & Co, Biogen, Stryker, Gilead Sciences, Pfizer, 3M Company, Johnson & Johnson, Smith & Nephew, Leadiant Biosciences, Kyowa Hakko Kirin, Shire, Ipsen, Endo International) of the industry. The Healthcare Nanotechnology market report correspondingly joins a predefined business market from a SWOT investigation of the real players. Thus, the data summarized out is, no matter how you look at it is, reliable and the result of expansive research.

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The global version of this report with a geographical classification such as

North America (the United States, Canada, and Mexico)Europe (Germany, UK, France, Italy, Russia, Spain, and Benelux)Asia Pacific (China, Japan, India, Southeast Asia, and Australia)Latin America (Brazil, Argentina, and Colombia)The Middle East and Africa

Reason to buy Healthcare Nanotechnology Market Report : 1) Breakdown of the sales data at the country level, with sales, revenue and market share for key countries in the world, from 2014 to 2019.2) The Healthcare Nanotechnology competitive situation, sales, revenue and global market share of top manufacturers are analyzed emphatically by landscape contrast.3) Describe Healthcare Nanotechnology sales channel, distributors, customers, research findings and conclusion, appendix and data source.4) The details of the competitive landscape outlined in this report are likely to provide an analysis of the prominent industry vendors, their growth profiles, strategies, and tactics, etc., that would help investors in decision-making.5) To project the size of Healthcare Nanotechnology submarkets, with respect to key regions (along with their respective key countries).6) To strategically profile the key players and comprehensively analyze their growth strategies.7) Focuses on the key global Healthcare Nanotechnology players, to define, describe and analyze the value, market share, market competition landscape, SWOT analysis and development plans in the next few years.

This report contributes an overall summary of the global Healthcare Nanotechnology market, including business perspectives, market strategies, assembles data related to various business firms, its year of establishment, contact information, market outline, sales revenue, industry segments, the business most prestigious location, and regional presence. The report includes several plans and policies related to the Healthcare Nanotechnology industry, moreover, it describes the management process, product appearance, manufacturing cost, and market volume. In addition, the global Healthcare Nanotechnology market report implicates financial usage, the quantity of product, chain format, demand and supply ratio. This report justifies the various business trends followed by the marketing sectors as well as the distributors of the Healthcare Nanotechnology industry.

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The next part also sheds light on the gap between supply and consumption. Apart from the mentioned information, the growth rate of the Healthcare Nanotechnology market in 2023 is also explained. Finally, the possibility analysis of new project investment is done in the report, which contains a comprehensive SWOT analysis of the Healthcare Nanotechnology market.

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Global Healthcare Nanotechnology Market 2019 by Manufacturers, Countries, Type and Application, Forecast to 2025 - Global Market News 24

Dublin, Dec. 04, 2019 (GLOBE NEWSWIRE) -- The "Biomarkers: Discovery Techniques and Applications - A Global Market Overview 2019" report has been added to ResearchAndMarkets.com's offering.

The report reviews analyzes and projects the global Biomarkers market for the period 2016-2025 in terms of market value in US$ and the compound annual growth rates (CAGRs) projected from 2018 through 2025.

The global market for Biomarkers is dominated by Oncology, in terms of application, estimated at a market share of 41.2% in 2018 and forecast to touch US$15.6 billion in 2019. The overall market for Biomarkers is projected to reach approximately US$50 billion by 2021.

The factors driving the Biomarkers market growth includes elevated demand for personalized medicine, technological advancement, immense utilization of biomarkers in drug discovery and development procedures, massive research on biomarkers, increasing awareness about the role of biomarkers in early disease diagnosis, increase research funding, and many more

Research Findings & Coverage

Key Market Trends

For more information about this report visit https://www.researchandmarkets.com/r/9jqzri

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

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

Excerpt from:
Analysis on the World Market for Biomarkers 2016-2019 & 2025: Key Trends, Discovery Techniques & Applications - Yahoo Finance

December 2019

Cambridge, UK: Global courier for cell and gene therapy, Biocair, will exhibit at Cell Therapy Manufacturing and Gene Therapy Congress between 3-6 December 2019. Taking place in Amsterdam, Cell Therapy Manufacturing and Gene Therapy Congress brings together key players from the industry to discuss the critical issues facing the development, scale-up and manufacture of cell-based and gene therapies.

Speaking about the logistics surrounding cell and gene therapy, Gert de Gier, Cell and Gene Business Development Director at Biocair, comments that with cell and gene therapies moving into the clinical trial phase for a wide variety of diseases, the results of these studies and their potential impact to patients are invaluable. Therefore the transportation of the materials and need for a secure supply chain is absolutely critical.

Patient samples can be bio-hazardous, time critical and have an extremely short shelf-life. Biocair has developed a highly customised project-based approach to the challenging requirements of the clinical and commercial cell and gene markets, providing chain of custody provision across every touchpoint, validated and pre-conditioned temperature-controlled packaging for all temperature ranges, total transparency in supply chain design and unrivalled short transit times (12 36hrs).

Biocairs specialist services are precisely documented in standard operating procedures and are regularly audited by its 12, 500 life science customers across the world. For more information, contact your local Biocair office or contact enquiries@biocair.com.

Notes to editors

Biocair is a specialist courier with over 30 years of dedicated experience in the pharmaceutical, biotechnology and life science sectors. The company has built up a unique, client-centric approach by employing scientists in front-line logistics positions and assembling a team of best-in-class industry experts in quality, cold chain and regulatory compliance amongst others. Biocair focuses on providing the most comprehensive service options available whilst delivering flexible, tailored, cost effective logistics solutions to all clients.

Biocair operates across Europe, Africa, Asia and the Americas.

Continued here:
Biocair Exhibits at Cell Therapy Manufacturing and Gene Therapy Congress 2019 - BioSpace

Dive Brief:

Astellas is paying up to join the gene therapy race.

At $60 per share, the Japanese pharma's takeover offer is more than double the $28.61 at which shares in Audentes traded at Monday's market close a multiple on par with Roche's bid for Spark but higher than the premium Novartis offered for AveXis, and Biogen for Nightstar Therapeutics.

Impressive clinical results, paired with the prospect of speedy development, have made gene therapies an attractive investment for larger drugmakers. And approvals for AveXis' Zolgensma (onasemonogene abeparvovec) and Spark's Luxturna (voretigene neparvovec) on data from only a few dozen patients indicates a FDA willing to clear new therapies on less information than usual, providing the benefit is clear enough.

*Over closing price the day preceding deal announcement SOURCE: Companies

For Astellas, its interest in Audentes appears linked as much to the options buying the biotech opens up as it is to the company's lead drug candidate.

Importantly, Audentes owns a sizable manufacturing plant capable of supporting clinical and commercial gene therapy production. Manufacturing the inactivated viruses used to deliver the one-time treatments has proved a bottleneck for the field, so acquiring capabilities could give Astellas a leg up in the future.

And Audentes this year secured a collaboration with Nationwide Children's Hospital in Ohio a hotbed for gene therapy science that gave the biotech several programs aimed at Duchenne muscular dystrophy and myotonic dystrophy type 1.

That work remains preclinical, however. Nearer term, Audentes offers Astellas a gene therapy candidate that could be submitted to regulators for approval as early as the middle of next year. Called AT132, the therapy is designed to treat x-linked myotubular myopathy, a rare and usually fatal neuromuscular disorder affecting infants and young boys.

Mutations in the MTM1 gene result in missing or dysfunctional myotubularin, a protein essential to skeletal muscle cell development. Without it, affected infants are extremely weak and often require ventilator assistance to breathe. Half of those diagnosed die in the first 18 months of life, according to estimates cited by Audentes.

AT132 proposes a genetic fix for the disease, delivering a functional copy of the MTM1 gene via a type of viral vector known as AAV8. Results from a Phase 1/2 study showed infants treated with the therapy were able to sit, stand and even walk. Importantly, the ten treated patients were able to significantly reduce their dependence on ventilatory support compared to two study participants who were used as a control.

Neuromuscular conditions like XLMTM and DMD require much higher doses of gene therapy than diseases of the eye, brain or liver, making Audentes manufacturing capacity a vital asset.

Audentes is also developing a therapy for Pompe disease, which, like DMD, is targeted by several other companies. A clinical-stage program in the ultra rare condition called Crigler-Najjar syndrome, however, appears to have been put on hold this year by Audentes.

Astellas said it will house Audentes in an independent subsidiary, and plans for the acquisition to form a foundation for a new research focus centered on genetic regulation. A statement on the deal seemed to hint more gene therapy partnerships could be forthcoming, or at least made possible by buying Audentes.

At $3 billion, Astellas' acquisition is the largest buyout of a gene therapy developer since Roche-Spark, as well as the largest in a run of deals brokered by the pharma, which is perhaps best known for its development of the prostate cancer drug Xtandi (enzalutamide).

Astellas' interest in Audenetes could ease some worries that would-be acquirers might put off dealmaking in gene therapy until the Federal Trade Commission reaches a decision in its review of Roche-Spark. Repeated delays have raised eyebrows among investors and analysts, who largely expected the deal to close rapidly.

"In light of the pending [Spark-Roche] transaction, some investors may find Astellas-[Audentes] as another potentially drawn-out and uncertain tie-up,' wrote Joseph Schwartz, an analyst at SVB Leerink, in a note to clients.

"However, our review of Astellas' product portfolio and R&D pipeline suggests minimal overlap with [Audentes]'s focus on neuromuscular diseases."

Schwartz and colleagues were able to identify only two drug candidates that could share a target with Audentes', potentially making a takeover from the much larger Astellas easier to clear.

Excerpt from:
Astellas joins gene therapy race with $3B Audentes buy - BioPharma Dive

LONDON, Dec. 3, 2019 /PRNewswire/ -- Alacrita, a leading life science consulting firm, with offices in the United States and the United Kingdom, announces the departure of Rob Johnson who leaves the company to take on a new role at a gene therapy startup. Anthony Walker, co-founder of Alacrita and managing partner, will take over leadership activities of the U.S. office.

Rob is a co-founder of Alacrita and was instrumental in establishing and growing the firm's U.S. presence. He guided it over the past seven years, leading the expansion of its consulting team there as well as of its portfolio of U.S. clients in the biotech and pharmaceutical sectors.

This continues a long history of Alacrita involvement in start-up companies. Alacrita consultants have played leading roles in a number of startup enterprises including Onyvax, Biotica Technology, Leucid Bio and other companies that have been spun out of university clients. Alacrita has worked with over 35 academic institutions on commercialization of early stage technology, including writing multiple business plans for successful spin-outs.

"Rob has been a driving force at Alacrita, leading our U.S. team and playing a major role in the progression of our firm in the United States. As we have grown there, his leadership has been effective and steady. Beyond his deep life-science expertise and leadership skill, he is a wonderful colleague who I have worked with for over 20 years. We could not be more excited for him in his new role," said Anthony Walker, Alacrita managing partner and co-founder.

"We are grateful for Rob's leadership of our U.S. operations. Rob worked tirelessly to help us build Alacrita into the transatlantic firm that it is today. We truly wish him well in his new endeavor," said Simon Turner, Alacrita managing partner.

"My time at Alacrita will always be meaningful. I am grateful for the incredible team that I have worked with all these years, for all that we have achieved together, as well as for the chance to help so many of our clients succeed with their projects. As I move on to a new challenge and chapter, I know I leave my post in capable hands that will continue the firm's strong trajectory upward," said Rob Johnson.

Anthony Walker, who takes over direction of the firm's U.S. office, leverages more than 30 years of experience, including over a decade spent buildingand managing a biotechnology company and nearly 20 years as a management consultant to the pharmaceutical and biotech industries.

Alacrita conducts more than 250 successful client assignments every year.The firm's team combines extensive industry experience (strategic, technical and commercial), advanced functional capabilities and a track record of success across the domain. Learn more by visiting http://www.alacrita.com.

For further information, please contact:

Anthony Walker, Managing Partner, U.S. Email: usa@alacrita.com Telephone: +1-617-714-9696 Address: 303 Wyman Street, Waltham, MA 02451

Simon Turner, Managing Partner, Europe Email: europe@alacrita.com Telephone: +44-(0)207-691-4915 Address: London BioScience Innovation Centre, 2 Royal College St, London, United Kingdom

Original post:
Alacrita's Rob Johnson Departs for Gene Therapy Space - P&T Community

The optimistic outlook is seen in an adorable 10-year-old girl named Jojo, who became the first patient to receive a gene therapy treatment, called AXO-AAV-GM1, during a human clinical trial this summer at the National Institutes of Health in Maryland. Auburn's College of Veterinary Medicine and the University of Massachusetts Medical School developed the treatment that has moved from helping cats with GM1 to hopefully helping children.

"Jojo is doing well and has experienced no major complications," said Dr. Doug Martin, professor in the Department of Anatomy, Physiology and Pharmacology in Auburn's veterinary college and the Scott-Ritchey Research Center. "Seeing all of the effort come together to help patientswho haveno treatment options today gives us great hope."

Auburn scientists for several decades have researched treatments to improve and extend the lives of cats affected by GM1. Martin is leading Auburn's effort, which was started by his mentor, Professor Emeritus Henry Baker.

To move the treatment toward human medicine, Martin developed a partnership with UMass Medical School researchers Drs. Miguel Sena-Esteves and Heather Gray-Edwards, an Auburn alumnaand they have worked collaboratively for 15 years, combining animal and human medicine studies to cure rare diseases that affect both animals and humans. In December 2018, the gene therapy was licensed to Axovant Gene Therapies Ltd., a clinical-stage company developing innovative gene therapies.

"This treatment is extremely promising because it has worked well in GM1mice and cats, and it is delivered by a single IV injection that takes less than an hour," Martin said. "We're hopeful that the treatment makes a real difference for patients and their families.

"The NIH is hoping to begin treating three or four more children in the next few months. As the trial progresses and more patients are treated, we'll have a good idea of whether the gene therapy helps children as much as it has helped the animals."

The NIH clinical trial is led by Dr. Cynthia Tifft, deputy clinical director at the National Human Genome Research Institute. "GM1 gangliosidosis is a devastating disease in young children, for which there are no currently approved treatment options. The development of a safe and effective gene therapy for these patients would be a welcome advancement in the field of pediatric lysosomal storage disorders affecting the brain," Tifft said.

For Auburn graduates Sara and Michael Heatherly of Opelika, whose son Porter was the first known case of GM1 in Alabama and died in 2016, the knowledge of a treatment is one of mixed emotions.

"We are excited to know there is hope for the future of children diagnosed with GM1," Michael Heatherly said. "We are thankful for everyone who has dedicated their time, resources and careers to move this treatment forward and to Axovant for bringing all of their work to life and making it a reality for GM1 patients.

"We understood early on the research would not help Porter, but we wanted to help spread the word of the research and the progress that was being made."

The Heatherlys gave Auburn researchers a reason to hope, and work harder for a cure. To honor the family, which held fundraisers for several years to support the research, the College of Veterinary Medicine's Scott-Ritchey Research Center incorporated Porter's likeness in a creative identity for the center.

SOURCE Auburn University

http://www.auburn.edu

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Auburn University research leads to gene therapy that provides hope for children with deadly disease - PRNewswire

DAYTON, Ohio, Dec. 3, 2019 /PRNewswire/ -- CSafe Global understands temperature control, gained from 40 years of experience in developing innovative temperature-controlled packaging - from solutions designed to transport vital pediatric vaccines to crucial life-saving cancer drugs. CSafe is known for protecting what matters most to pharmaceutical companies, so that patients receive what matters most to them.

CSafe recognizes the importance of thermally protecting shipments of life-enhancing medications as they are shipped to patients in need around the world. The criticality of temperature protection within the emerging Cell and Gene Therapy (CGT) space is further amplified due to the value and uniqueness of CGT products and the complicated supply chain. The new AcuTemp Plus Series has been developed with this in mind, and will confidently meet the evolving and increasingly demanding customer expectations for temperature performance, solution quality, and system robustness required of packaging solutions to protect shipments of novel cell and gene therapies.

"We are proud to expand our cold chain packaging offerings for the Cell and Gene Therapy market, and have focused and invested significantly in R&D to develop best-in-class solutions to meet the demanding product requirements for this emerging class of medication," stated Patrick Schafer, CEO of CSafe Global. "Test results of the new AcuTemp Plus Series have been very impressive with temperature performances outlasting other market available solutions by a large margin."

The AcuTemp Plus Series of packaging solutions has been designed with proprietary, high-performance ThermoCorVacuum Insulated Panels (VIP) to guarantee precise end-to-end control of internal payload temperatures. In addition to industry-leading performance, the innovative range has been developed with simplicity in mind, benefiting from optimized system components and solution design. The new series is available in multiple size configurations and temperature profiles, along with different integrated track and trace options to meet customer needs. Furthermore, the AcuTemp Plus Series is supported with a fully managed, end-to-end service infrastructure leveraging CSafe's industry-unique retest and reuse program - REPAQ.

With a market-leading portfolio of both active and passive temperature-management solutions, it is CSafe's mission to bring peace of mind to every life-science customer.

CONTACT: Josh Angliss, jangliss@csafeglobal.com

View original content:http://www.prnewswire.com/news-releases/csafe-expands-cold-chain-offerings-for-the-cell-and-gene-therapy-market-with-launch-of-high-performing-acutemp-plus-series-of-temperature-controlled-packaging-300968270.html

SOURCE CSafe Global

Original post:
CSafe expands cold chain offerings for the Cell and Gene Therapy market with launch of high-performing AcuTemp Plus Series of temperature-controlled...

Shares of Regenxbio (RGNX) have risen by 190% since I initially uncovered and recommended a position in this gene therapy pioneer in 2016. The stock has appreciated by just 52% since my February 2018 update piece.

With the recently announced acquisition of collaboration partner Audentes Therapeutics (BOLD) by Astellas Pharma for $2.7 billion (also a prior recommendation), I thought it's time to revisit this gene therapy "picks and shovels" play.

Chart

Figure 1: RGNX daily advanced chart (Source: Finviz)

When looking at charts, clarity often comes from taking a look at distinct time frames in order to determine important technical levels to get a feel for what's going on. In the above chart (daily advanced), we can see a downtrend taking place from April into Q3, with share price bottoming in the low 30s in September. Recently, the stock received a boost after news of Audentes Therapeutics being acquired (had licensed NAV technology for various indications including XLMTM, Crigler-Najjar and CPVT). Readers might recall that when collaboration partner AveXis was acquired for $8.7 billion in 2018, its suitor Novartis (NVS) was required to pay them $100 million upfront as an accelerated licensee payment (while remaining on the hook for $80 million in commercial milestones plus royalties on Zolgensma). A move to highs last witnessed in 2018 would equate to a bit less than a double from today's levels.

In my last update piece, I touched on the following keys to our bullish thesis:

Figure 3: Internally developed pipeline (Source: corporate presentation)

Figure 4: Collaboration partner pipeline (Source: corporate presentation)

Let's take a look at recent events and how they've affected our thesis here.

In late July Regenxbio and Swiss biotech firm Neurimmune announced an exclusive license to develop novel AAV gene therapies using NAV vectors to deliver human antibodies against targets implicated in chronic neurodegenerative diseases, including tauopathies. Both companies will be jointly responsible and share development costs equally, with each having co-development and co-commercialization options (or can elect to receive a phase-based worldwide royalty instead of continued development investment).

On July 31st Regenxbio announced a license agreement with Pfizer (PFE), specifically non-exclusive global license to NAV AAV9 vector for commercialization of gene therapies for the treatment of Friedreich's ataxia. In return for these rights, Regenxbio received an upfront payment, and has the potential to receive ongoing fees, development and commercial milestone payments, and royalties on net sales of products incorporating their IP.

On October 30th, it was disclosed that the company exercised its option under the previously announced option and license agreement to Clearside Biomedical's (CLSD) in-office SCS Microinjector for the delivery of adeno-associated virus-based therapeutics, including RGX-314 delivery to the suprachoroidal space to potentially treat wet age-related macular degeneration, diabetic retinopathy, and related conditions where chronic anti-vascular endothelial growth factor (VEGF) treatment is currently the standard of care. Deal terms were modest, involving a small upfront payment, up to $34 million in development milestones, up to $102 million in sales milestones and mid-single digit royalties on net sales of products incorporating SCS Microinjector.

Figure 5: Widespread retinal transduction observed in both subretinal and suprachoroidal delivery of AAV8 in non-human primates (Source: Corporate Presentation)

Keep in mind that Regenxbio is currently suing the FDA, as they disclosed a clinical hold for RGX-314 suggesting the agency opted for this action "without notice or explanation". The clinical hold is related to issues associated with delivery systems currently being used (seems agency is not a fan of subretinal injections, which allows for a more direct approach but can have a complicated adverse event profile (involves potential events such as intraocular inammation, retinal detachment, ocular hemorrhage, etc). A more convenient intravitreal approach is being utilized by Adverum Biotechnologies (ADVM). While such differences may not be that big of a deal in clinical trials, you can bet that when and if such gene therapies make it to the market, those that offer the ideal mix of high convenience and best safety profile will be the most likely to win this high stakes race.

Figure 6: Subretinal injection versus intravitreal injection (Source: Adverum corporate presentation)

For the third quarter of 2019, the company reported cash and equivalents of $417.1 million as compared to net loss of $34.6 million. Research and development costs nearly doubled to $35.7 million, while SG&A increased to $12.4 million

Regarding RGX-314 in treating wet AMD and diabetic retinopathy, we are informed of the clinical hold related to use of third-party commercially available devices used to deliver the gene therapy candidate. Specifically, the company states that all 42 patients have been dosed in the phase 1/2a study and that the partial clinical hold on IND is not related to the gene therapy candidate itself. Management guides for phase 2b study in wet AMD to get underway in Q1 2020, around which time IND will be filed for the study in diabetic retinopathy. We are reminded that data here continues to be encouraging, with no drug-related serious adverse events and subjects in Cohort 5 showing reduction of over 80% from the mean annualized injection rate during the 12 months prior to receiving RGX-314. Additionally, durable effects on vision and retinal thickness had been demonstrated over 1.5 years in the third cohort, with 50% of subjects remaining free of anti-VEGF injections more than 1.5 years after RGX-314 administration.

Figure 7: Cohort 5 injections pretreatment and after being treated with RGX-314 (Source: Corporate Presentation)

Management continues to state that they are evaluating in-office delivery of RGX-314 to the suprachoroidal space and will unveil plans for this route of administration next year.

As for RGX-501 for the treatment of Homozygous Familial Hypercholesterolemia (HoFH), interim data from Cohort 2 is expected at the end of the year (keep in mind this time they are using corticosteroid prophylaxis). Also at the end of the year, we'll get an interim data update on the phase 1/2 study of RGX-121 for the treatment of MPS II.

As these gene therapy companies are increasingly valued on the basis of their manufacturing capabilities as reflected in recent buyouts (including Audentes Therapeutics), it's worth noting that Regenxbio's new cGMP production facility (allows for production of NAV Technology-based vectors at scales up to 2,000 Liters) is expected to be operational in 2021.

As for partnered efforts, Novartis revealed Q3 Zolgensma sales of $160 million resulting in royalty revenue to Regenxbio of $9.2 million. Audentes Therapeutics' AT132 in XLMTM is well on its way to potential regulatory approval with BLA to be filed mid-2020.

To conclude, even after the recent bounce this gene therapy "picks and shovels" has continued upside ahead. With 20+ partnered product candidates, one can almost think of it as a mutual fund of sorts as investors gain exposure to various rare disease programs as well as programs targeting more prevalent conditions. On the other hand, the company has much more to prove with wholly-owned programs, as the anti-VEGF market is known for being intensely competitive with multiple "next-generation" approaches being tried out by larger companies aiming to garner a slice of this lucrative pie. Consider Baker Brothers' recent royalty deal, purchasing a 4.5% rate for $225 million (assumes KSI-301 worth over $5 billion using back of the envelope math). Another overlooked catalyst is phase 1 data for Danon Disease program with collaboration partner Rocket Pharmaceuticals (RCKT) in 2020, a lucrative indication of high unmet medical need considering estimated US+ EU prevalence of 15,000 to 30,000 patients.

For readers who are interested in the story and have done their due diligence, Regenxbio remains a Buy and I suggest patiently accumulating dips over the next couple of quarters.

Additional dilution in the near term does not look likely considering the current cash position and operational runway. Disappointing data for wet AMD and other programs not to mention setbacks in the clinic (especially delays or safety/tolerability concerns) would weigh on the stock. Failure to get the recent clinical hold lifted would weigh on the stock and the company's competitive position in the wet AMD market (and related indications). Competition for certain indications should not be ignored, especially by peers with significantly greater resources.

Author's Note: I greatly appreciate you taking the time to read my work and hope you found it useful. Consider clicking "Follow" next to my name to receive future updates and look forward to your thoughts in the Comments section below.

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Regenxbio: Revisiting This Gene Therapy 'Picks And Shovels' Play - Seeking Alpha

New GMP conditioning of PEIpro-GMP designed to support clinical phase and commercialization stages of production

Strasbourg, France December 3, 2019 Polyplus-transfection(R) SA, the leading biotechnology company that supports gene and cell therapy by supplying innovative transfection solutions, today announces the availability of the first GMP-grade transfection reagent PEIpro(R)-GMP in additional conditioning that will ensure additional flexibility and cost-efficiency.

Polyplus-transfection commercialized PEIpro-GMP at the end of 2018. This was the first transfection solution for the gene and cell therapy industry that is compliant with global cGMP viral vector manufacturing requirements. Polyplus-transfection is now expanding the PEIpro-GMP packaging options from a 1 liter single-use bag to several conditioning options from 10 milliliters up to 1 liter in both single-use bags and bottles.

The use of closed-systems and single-use technologies for clinical trials and commercial manufacturing of viral vectors continues to increase. Polyplus-transfection is now providing two sizes of PEIpro-GMP in bags to ensure aseptic connections to successful perform large-scale transfection in closed sterile systems. Polyplus-transfection now additionally supplies PEIpro-GMP in bottle conditioning that will allow the adaption to different manufacturing scale-up and scale-out strategies. These increasing scale-out strategies require the performance of multiple smaller-scale transfections in multiple cell culture units at the same time. Demand for transfection reagents has thus increased for the smaller as well as for the larger conditioning options. The increases in options will therefore result in additional flexibility for therapeutic developers in the setup of cost-efficient viral vector manufacturing processes.

Polyplus-transfection has been developing strong supplier-manufacturer relationships over eighteen years, and certainly since the conception of the modern gene and cell therapy industries. As a result, we have unrivalled first hand visibility on the ever-changing needs of the gene and cell therapy markets, said Claire Wartel, PhD, director of quality and compliance, Polyplus-transfection. The focus of the market has evolved to commercialization and late-stage clinical stages. This means the therapeutic production scale and strategy needs to be adaptable to the production of each viral vector, whilst complying with cGMP manufacturing requirements. To provide this critical function in making available therapies to patients, Polyplus-technology is now able to offer a sustainable solution for viral vector platforms. This will benefit gene and cell therapy developers, the wider industry, and most importantly, the patient community reliant on the ongoing development of these therapies.

Claire Wartel recently gave more information on Identifying and mitigating risks in the viral vector supply chain in a podcast interview that can be accessed here.

About Polyplus-transfection SA

Polyplus-transfection SA is a biotechnology company specializing in nucleic acid delivery solutions located close to the University of Strasbourg in Eastern France and a market leader for transfection reagents for cell & gene therapy. Polyplus-transfections vast portfolio of delivery solutions can be used for all types of applications, from research and process development, all the way to clinical trials. For more information, please visit the Polyplus-transfection web site at: http://www.polyplus-transfection.com.

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Polyplus-transfection expands GMP portfolio to deliver flexibility for gene and cell therapy manufacturing - BioSpace

By Murielle Gonzalez 2-Dec-2019

Pharmaceuticals

New building in Stein also hosts the production of difficult-to-manufacture solid dosage forms

A cell processing specialist at work in new cell and gene facility in Stein. Photo as seen on Novartis website

Novartis has announced the opening of a new manufacturing facility for cell and gene therapies in Stein, Switzerland.

Our site in Stein is vital for new launches of solid and liquid drugs, explained Steffen Lang, Global Head of Novartis Technical Operations and member of the Novartis Executive Committee.

Lang continued: The construction of the new manufacturing facility is another investment in the production of breakthrough cell-based therapies that can potentially change the lives of patients.

In addition to manufacturing areas for novel CAR-T cell therapies, the new building also hosts the production of innovative, difficult-to-manufacture solid dosage forms, such as tablets and capsules.

Last September, the first clinical production of a cell and gene therapy badge was successfully completed, Novartis said.

Unlike conventional drug production, cell and gene therapy asks for the manufacture of a personal dose for each patient.

For this purpose, patients who have already undergone various therapies have a small amount of their own blood cells taken, which are then sent to Stein.

Here we enrich part of the white blood cells, the T cells, and genetically modify them so that they can recognize and fight the cancer cells in the patients blood, said Dorothea Ledergerber, project manager of the Stein plant for cell and gene therapies.

The altered cells are then sent back to a hospital and administered to the patient by infusion.

For Ledergerber, Novartis is doing pioneering work here. She explained: We have the unique opportunity to offer patients for whom there have been no other therapeutic options a totally new perspective by using these novel CAR-T cell therapies.

Novartis celebrated the opening in the presence of Federal Councillor Alain Berset and other guests on 28th November 2019.

Read more:
Novartis opens facility for innovative cell and gene therapies in Switzerland - Cleanroom Technology

Eleven drugmakers led by Pfizer and Novartis have set aside a combined $2 billion to invest in gene therapy manufacturing since 2018, according to a Reuters analysis, in a drive to better control production of the world's priciest medicines.

The full scope of Novartis' $500 million plan, revealed to Reuters in an interview with the company's gene therapy chief, has not been previously disclosed. It is second only to Pfizer, which has allocated $600 million to build its own gene therapy manufacturing plants, according to filings and interviews with industry executives.

Gene therapies aim to correct certain diseases by replacing the missing or mutated version of a gene found in a patient's cells with healthy copies. With the potential to cure devastating illnesses in a single dose, drugmakers say they justify prices well above $1 million per patient.

But the treatments are also extremely complex to make, involving the cultivation of living material, and still pose a risk of serious side effects.

Drugmakers say building their own manufacturing plants is a response to rising costs and delays associated with relying on third-party contract manufacturers, which are also expanding to capitalize on demand.

They say owning their own facilities helps safeguard proprietary production methods and more effectively address any concerns raised by the U.S. Food and Drug Administration (FDA), which is keeping a close eye on manufacturing standards.

"There's so little capacity and capability at contract manufacturers for the novel gene therapy processes being developed by companies," said David Lennon, president of AveXis, Novartis's gene therapy division. "We need internal manufacturing capabilities in the long term."

The approach is not without risks.

Bob Smith, senior vice president of Pfizer's global gene therapy business, acknowledged drugmakers take a "leap of faith" when they make big capital investment outlays for treatments before they have been approved or, in some cases, even produced data demonstrating a benefit.

PUSHING THE LIMITS

The rewards are potentially great, however.

Gene therapy is one of the hottest areas of drug research and, given the life-changing possibilities, the FDA is helping to speed treatments to market.

It has approved two so far, including Novartis's Zolgensma treatment for a rare muscular disorder priced at $2 million, and expects 40 new gene therapies to reach the U.S. market by 2022.

There are currently several hundred under development by around 30 drugmakers for conditions from hemophilia to Duchenne muscular dystrophy and sickle cell anemia.

The proliferation of these treatments is pushing the limits of the industry's existing manufacturing capacity.

Developers of gene therapies that need to outsource manufacturing face wait times of about 18 months to get a production slot, company executives told Reuters.

They are also charged fees to reserve space that run into millions of dollars, more than double the cost of a few years ago, according to gene therapy developer RegenxBio.

As a result, companies including bluebird bio, PTC Therapeutics and Krystal Biotech are also investing in gene therapy manufacturing, according to a Reuters analysis of public filings and executive interviews.

They follow Biomarin Pharmaceutical Inc, developer of a gene therapy for hemophilia, which constructed one of the industry's largest manufacturing facilities in 2017.

REGULATORY SCRUTINY

The FDA is keeping a close eye on standards.

This comes amid the agency's disclosure in August that it is investigating alleged data manipulation by former executives at Novartis' AveXis unit.

AveXis had switched its method for measuring Zolgensma's potency in animal studies. When results using the new method didn't meet expectations, the executives allegedly altered the data to cover it up, the FDA and Novartis have said.

One of the former executives, Brian Kaspar, denied wrongdoing in a statement to Reuters. Another, his brother Allan Kaspar, could not be reached for comment.

Novartis and the FDA say human clinical trials, which found Zolgensma effective in treating the most severe form of spinal muscular atrophy in infants, were not affected. Novartis also says its investments in gene therapy production started long before it became aware of the data manipulation allegations.

But the scandal has highlighted the importance of having a consistent manufacturing process for gene therapies, industry executives say.

According to four of them, the FDA has stressed in recent meetings the need for continuity in production processes all the way from the development of a drug to its commercialization.

By bringing production in-house, drugmakers may avoid pitfalls such as the need to switch to a larger facility if contract manufacturers' capacity proves limited, executives say.

The FDA is finalizing new guidelines for gene therapy manufacturing, expected at the end of the year.

"Manufacturing consistency is always a major concern for the agency," FDA spokeswoman Stephanie Caccomo told Reuters.

Highlighting the pressures on the industry, Sarepta Therapeutics, which largely outsources manufacturing, delayed a clinical trial of its Duchenne treatment in August, telling investors it wanted to avoid any questions from regulators about consistency in producing its therapy at commercial scale.

ENOUGH GROWTH FOR ALL?

"Between the trade secrets, the cost schedules and the time lag, it makes a whole lot of sense, if you can do it, to build out your own facilities and more and more gene therapy companies have started to do that," said Krish Krishnan, chief executive of Krystal Biotech Inc.

Krystal, which is developing therapies for rare skin diseases, has built one manufacturing facility and plans to invest more than $50 million in a new one it will start constructing in December.

MeiraGTx, which focuses on gene therapies for eye conditions, estimates it is currently spending roughly $25 million a year on manufacturing, including process development.

Despite such moves, however, contract manufacturers like Lonza and Thermo Fisher are confident their businesses will continue to grow due to the strength of demand.

Thermo Fisher has told investors its Brammer gene therapy manufacturing division, acquired in May, could soon earn $500 million in revenue a year, double its projected 2019 earnings.

Lonza CEO Marc Funk is also optimistic.

"Demand in gene therapy has increased," he said in an interview. "We believe this is going to continue in the coming years."

Continued here:
Pfizer, Novartis lead pharma spending spree on gene therapy production - Japan Today