Spinal Muscular Atrophy Phase 3 Candidate ISIS-SMNRx Expected to Reach USD 0.47 Billion in Sales in the U.S

Spinal muscular atrophy (SMA) is a hereditary autosomal recessive disease affecting areas of the nervous system that control voluntary muscle movement. SMA is identified as a leading genetic disorder causing mortality among infants. According to Orphanet, one infant in 6,000 births is estimated to suffer from SMA. Furthermore, over 10 million people in the U.S. are carriers of SMA gene. Depending on the age of onset and symptoms, spinal muscular atrophy is classified in four main types. Type 1 SMA is the most severe form, and affects infants aged between zero and six months. Over 60% of annual SMA cases are of type 1 SMA, wherein infants usually do not survive for more than 2 years. Type 2 SMA is a moderate form affecting children aged between six months and eighteen months. Patients with Type 3 SMA and Type 4 SMA usually have a normal lifespan.

Currently, no cure exists for spinal muscular atrophy, and treatment is directed towards managing common symptoms of SMA. Physiotherapy is strongly recommended in early stages of the disease to extend range of motion and improve muscle flexibility. Other treatment methods are targeted to treat gastrointestinal and respiratory disorders, which are common in SMA patients. The SMA pipeline is dry, with single drug in phase 3, three in phase 2, and one in phase 3 clinical trials. ISIS-SMNRx by Isis Pharmaceutical is an antisense drug that has shown promising results in phase 2 clinical trials. Phase 3 study of ISIS-SMNRx for treatment of Type 1 SMA and Type 2 SMA is expected to complete in 2016. In the United States, the drug is expected to reach annual sales of USD 0.47 billion by 2023. Olesoxime (TRO19622) has completed its phase2/3 trials, and has shown promising results, with potential to effectively treat type 2 and type 3 SMA. Other potential candidates include Olesoxime (TRO19622), LMI070, RO6885247, CK-2127107, scAAV9.CB.SMN and RG3039.

The United States and Europe have higher percentage of diagnosed cases of SMA. Government and private organization in these regions are also involved in creating awareness about the disease. Higher confirmed carrier population would also help in faster market growth in these regions. Furthermore, government incentives for development of therapeutic alternatives for rare diseases also boosts research and development in the SMA landscape.Companies with potential candidates in the spinal muscular atrophy pipeline include AveXis, Inc., Cytokinetics, Inc., F. Hoffmann-La Roche Ltd., Novartis AG, and Isis Pharmaceuticals, Inc.   

Spinal Muscular Atrophy Pipeline is segmented as below:
Late Stage Candidates (Phase 3)
ISIS-SMN Rx

Early Stage Candidates (Phase 1 and Phase 2)
Olesoxime (TRO19622)
LMI070
RO6885247
CK-2127107
CB.SMN
RG3039

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Nanocapsules Market Segment Forecasts up to 2023

Nanocapsules are drug delivery agents within the size range of 10nm to 1000 nm that are normally composed of a capsule shell and a hollow space in which desired substance or drug molecules are embedded. The ultrafine size of nanocapsules match the biological composition of human body such as RBCs which permits enhanced permeation and retention (EPR) through narrow channels during diffusion. Due to this EPR effect, nanocapsules are used as drug carriers. Nanocapsules can be formulation using various methods such as loading of drug molecules into hollow polyelectrolyte shell and another method being electrostatic layer by layer (LbL) technique. These latest LbL self-assembled polyelectrolyte capsules provide an efficient way to perform multiple drug delivery tasks simultaneously. Some nanocapsules are used for targeted drug delivery in anti-tumor and anti-tuberculosis therapies wherein the outer sphere of nanocapsules is fabricated by the lithography technique. Lipid nanocapsules (DNA LNCs) and multimodular systems (DNA MMS) are used in gene therapy.

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Global investment in nanotechnology research has witnessed a six fold increase during the last decade. Consequently, nanotechnology will completely revolutionize the industrial processes in the near future due to its remarkable economic potential. Some of the key drivers for the market growth include investments and breakthroughs in biomedical, pharmaceutical, electronics, molecular and diagnostic fields. Besides, these nanocapsules are being increasingly used in agrochemicals, environment and wastewater treatments, genetic engineering, consumer goods and cosmetic procedures. Some of the most advanced future applications would be nanocapsule dressings to deliver antibiotics aiding faster wound healing without invasion to the skin. These nanocapsule bandages would be consistently demanded by defence and protection forces as they need not be changed periodically. According to the experts, electronics and magnetic sector will be the largest potential market for nanocapsules, followed by biopharmaceutical industry, catalysis, energy and engineering. However, growth in the nanocapsules market will also pose new challenges with respect to the toxicity and environmental impact of nanocapsules. Recently efforts have been made to overcome these drawbacks by developing eco-friendly versatile nanocapsules. For instance, nanocapsules introduced by the Center for Self-assembly and Complexity, Institute for Basic Science (IBS). Other factors such as cost escalation and consequences on the global economy especially in cases of under-developed economies with poor R&D investment would exhibit low social and ethical acceptance of nanocapsules.

Funding for nanotechnology is soaring worldwide at a competitive level, with each major economic power and many developed economies making a substantial investment to revamp their industrial and postindustrial supply chains. Technological inventions and discoveries in nanotechnology would cause tremendous growth in the global nanocapsule market. Due to a variety of potential applications, nanotechnology is expected to become the key technology in the near future. U.S. has invested around USD 3.7 billion through its National Nanotechnology Initiative for research activities. The European Union had spent approximately USD 1.2 billion and Japan USD 750 million by 2012. Moreover, there is a steady demand for medically advanced procedures in developed as well as developing regions having a well-established healthcare infrastructure. In the Asia Pacific region, the demand for nanocapsules is projected to rise owing to the high disease burden of tuberculosis and cancer. Some of the key factors that are likely to contribute toward the market growth are growing awareness about genetic counselling, genetic disorders, high prevalence of tuberculosis and multi-drug resistant tuberculosis, increasing trend in cancer and tumor ailments and surging demand for oncological treatments.

Presently, nanocapsules are under different stages of clinical trial phases facing several hurdles to gain market entry. However, few commercially available nanocapsules which are now commercially used as several pigments in sunscreen, scratch resistance coatings, catalysts, and biomarkers. Some of the key market players for these commercially available nanocapsules are Abraxis, Artificial Cell Technologies, Inc., BioDelivery Sciences International, Inc., Biophan Technologies, Inc., Cornerstone Pharmaceuticals, Inc., Genetic Immunity, Inc., Keystone Nano, Inc., Makefield Therapeutics, Inc., Nanospectra Biosciences, Inc., and Mersana Therapeutics, Inc.

This research report analyzes this market on the basis of its market segments, major geographies, and current market trends. This report provides comprehensive analysis of
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Current market trends
Market structure
Market projections for upcoming years

This report is a complete study of current trends in the market, industry growth drivers, and restraints. It provides market projections for the coming years. It includes analysis of recent developments in technology, Porter’s five force model analysis and detailed profiles of top industry players. The report also includes a review of micro and macro factors essential for the existing market players and new entrants along with detailed value chain analysis.

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Neuromodulation Devices Market - Global Industry Size, Market Share, Trends, Analysis, And Forecast 2012 - 2018

According to a new market report published by Transparency Market Research  "Neuromodulation Devices Market - Global Industry Analysis, Size, Share and Forecast, 2012 - 2018 " the global neuromodulation devices market was valued at USD 2,758 million in 2011 and is expected to grow at a CAGR of 14.4% from 2012 to 2018, to reach an estimated value of USD 7,072.6 million by 2018.

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The market is driven by factors such as the aging world population leading to chronic diseases such as Parkinson´s and Alzheimer´s disorders and technological advancements in the field of neuromodulation devices including the invention of transversal neuromodulation technology by NMT (Neurowave Medical Technologies). Moreover, the rising prevalence of chronic diseases, increasing demand for minimally invasive surgeries, the availability of external funding to conduct clinical studies to help come up with new and technologically advanced products and the rising healthcare related consumer expenditure have also driven the growth in this market. The fact that a neuromodulation device could be used as an add-on treatment and so can be used simultaneously with other medications also drives the market forward.

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The North American region holds majority of the market share and accounted for a share of 65% in 2011. The North American neuromodulation device market was valued at USD 1,792.7 million in 2011. The economic developments in the Asia Pacific region make it the fastest growing region (CAGR of more than 15% from 2012 to 2018) in the neuromodulation devices market.

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The future growth of the market will be ensured by capitalizing on market opportunities such as increasing healthcare related consumer expenditure in emerging economies in the Asia Pacific, Latin American and Eastern European regions. The availability of medical device manufacturing outsourcing options helps in curbing costs and thereby enhances profit margins. Also, the presence of consolidation options in the healthcare industry helps in strengthening the revenue base to conduct R&D exercises for new product development and thus helps in strengthening market share.

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Liver Diseases Therapeutics Market - Global Industry Analysis, Size, Share, Trends And Forecast 2012 – 2018

According to the latest market report published by Transparency Market Research "Liver Diseases Therapeutics Market (Chemotherapy, Anti-Virals, Vaccines, Targeted Therapy and Immunosuppressants) - Global Industry Analysis, Size, Share, Trends and Forecast, 2012 - 2018", the global liver diseases therapeutics market was worth USD 6.5 billion in 2011 and is expected to reach USD 10.9 billion in 2018, growing at a CAGR of 8.6% from 2012 to 2018. In the overall global market, anti-viral drugs are expected to create its lead position in terms of revenue generation by 2018. Anti-viral drugs will have a share of 32.7% of the global market followed by vaccines at 19.1% in 2018.

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The major drivers of the global liver disease therapeutics market are increasing aging population inducing chronic diseases such as hepatitis and liver cancer, increasing global prevalence of liver disorder, high unmet needs existing in liver cancer and increased vaccination in emerging economies. However side-effects and risks associated with the medication, strict FDA approval norms and other government regulations and availability of alternate treatment procedures may act as the barrier for this market. Strong pipeline drugs and consolidation opportunities in the healthcare industry will pose future growth prospect for this market.

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Anti-viral drugs represent the largest market in the liver disease therapeutics, which is justified by the fact that many oral, direct-acting antiviral medications are in development phase for treating hepatitis C. Currently, anti-virals are only available for hepatitis B and C. However, many anti-viral hepatitis C pipeline drugs namely GS-7977 (Nucleoside/Nucleotide polymerase inhibitor), Daclatasvir (NS5a Inhibitor), Asunaprevir (Protease Inhibitor), Simeprevie (Protease Inhibitor), and Vaniprevir (Protease Inhibitor), are expected to accelerate this market in near future.

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This research is specially designed to estimate and analyze the demand for liver diseases therapeutics in global market. The research provides an in-depth analysis of the product sales, drugs class wise trend analysis and the major player pertaining to this market. High level analysis of the mentioned drug class of global liver diseases therapeutics market is performed and the report provides detailed analysis, comprehensive historical data and statistically tested forecast of the drug classes thus covered. Detailed qualitative information is given for different liver diseases and the application of different drugs as the treatment and therapy option.

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Narcolepsy Market - Global Industry Analysis, Size, Trends And Forecast, 2013 – 2019

Transparency Market Research Reports incorporated a definite business overview and investigation inclines on "Narcolepsy Marke ". This report likewise incorporates more illumination about fundamental review of the business including definitions, requisitions and worldwide business sector industry structure. Narcolepsy is a chronic neurological disorder, which is characterized by brain’s inability to control the sleep-wake cycle of an individual. This disorder is not caused due to any mental illness, but is mainly caused due to genetic abnormalities occurring from environmental factors or biologic factors.

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Narcoleptic individuals exhibita typical characteristic symptom, i.e. excessive daytime sleepiness (EDS), where individuals experience disturbed night sleep and abnormal daytime sleep pattern. Narcolepsy disorder is often confused with insomnia. Other major symptoms of this disorder include, cataplexy, sleep paralysis, hypnagogic hallucinations, automatic behavior and night weakness.

Some of the major factors driving the growth of this market include increasing prevalence of various sleep disorders including narcolepsy, rising awareness about the disorder and increasing demand for advanced therapeutics and diagnostics.Narcoleptic disorder is equally prevalent to Parkinson’s disease or multiple sclerosis and is more prevalent than cystic fibrosis. There are more than 3 million people suffering from this disorder worldwide.Rising awareness about the disorder, and the resultant increase in demand for new and innovative drugs and other treatment optionswill lead to the growth of this market in future.

Geographically, North America dominates the global narcolepsy market followed by Europe. Major factors responsible for their dominance are high prevalence rate of narcolepsy in these regions. U.S. alone accounts for more than 200,000 narcoleptic individuals and is also characterized by high awareness levels among the general population. Asia-Pacific region exhibited fastest growth in this market due to increasing awareness levels about the disease and its treatment options and increasing spending power of the population. 

Some of the key players operating in this market are Addrenex Pharmaceuticals, Inc., Arena Pharmaceuticals, Inc., BIOPROJET, Cephalon, Inc., Evotec AG, Graymark Healthcare, Inc., Hypnion, Inc., Jazz Pharmaceuticals, Inc., Ligand Pharmaceuticals, Inc., and Teva Pharmaceutical Industries Ltd.

This research report analyzes this market depending on its market segments, major geographies, and current market trends. Geographies analyzed under this research report include 

• North America
• Asia Pacific
• Europe
• Rest of the World 

This report provides comprehensive analysis of 

• Market growth drivers
• Factors limiting market growth
• Current market trends
• Market structure

This report is a complete study of current trends in the market, industry growth drivers, and restraints. It provides market projections for the coming years. It includes analysis of recent developments in technology, Porter’s five force model analysis and detailed profiles of top industry players. The report also includes a review of micro and macro factors essential for the existing market players and new entrants along with detailed value chain analysis. 

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Advances In Protein/Antibody Engineering Market - Global Industry Analysis, Size, Share, Growth, Trends, And Forecast, 2012 - 2018

Antibodies and proteins developed with new and advantageous functions and properties are known as engineering of proteins and antibodies. Protein engineering can be defined as a process of developing valuable proteins and modification of antibodies to increase similarity and is called antibody engineering. Antibody and protein engineering is used in curing inflammatory disorders, as a new treatment of diseases, for protein purification, and in diagnostic procedures. There are two approaches used for designing new antibodies and proteins - directed evolution and rational designing. 

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The market for antibody engineering is changing quickly due to novel technologies. Moreover, protein engineering offers flexibility in engineering, and designing new and desirable antibodies is expected to be the major factor driving the growth of the market. The future holds huge opportunities for this market due to the rising prevalence of diseases such as cancer and increasing health care concerns. 

Emerald Bio, ProSci Incorporated, Precision Antibody, Fusion Antibodies, BioXcell, GenTarget Inc, ImaginAb, Covalab, and IBL International are some of the major players in this market.

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This research report analyzes this market depending on its market segments, major geographies, and current market trends. Geographies analyzed under this research report include

North America 

Asia Pacific 

Europe

Rest of the World  

This report provides comprehensive analysis of 

Market growth drivers 

Factors limiting market growth

Current market trends 

Market structure

Market projections for upcoming years

This report is a complete study of current trends in the market, industry growth drivers, and restraints. It provides market projections for the coming years. It includes analysis of recent developments in technology, Porter’s five force model analysis and detailed profiles of top industry players. The report also includes a review of micro and macro factors essential for the existing market players and new entrants along with detailed value chain analysis. 

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Global Therapeutic Vaccines Market - Industry Analysis, Market Share, Size, Growth, Trends, And Forecast, 2012 - 2018

Therapeutic vaccines function by triggering a body immune response against a disease. The global therapeutic vaccines market was valued at USD 292 million in 2011. The introduction of therapeutic vaccines has opened up new avenues in the field of chronic disease treatment and prophylactic immunization. Provenge was the first U.S. FDA approved immunotherapy drug to treat prostate cancer in men in 2010, which has encouraged rigorous research and development initiatives by pharmaceutical companies to develop novel vaccines for untapped disease segments.

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As a result of these initiatives, many more therapeutic vaccines are currently in the pipeline such as breast cancer vaccine, lung cancer vaccine; Alzheimer’s vaccine, malaria vaccine, and diabetes vaccine are expected to be introduced in the market during the period from 2012 to 2018. 

The market is currently facing growth hindrances as R&D approaches, and regulatory and reimbursement policies are inconspicuous owing to the nascent nature of the market. However, regulatory bodies are working enthusiastically to lay out policies and growth strategies, due to the fact that it holds the potential to solve or curb chronic diseases, with no currently available treatments, such as HIV AIDS.

our components hold imperative importance in designing a therapeutic vaccine:

Antigens: These are substances that evoke an immune response from the body and are responsible for the production of antibodies

Adjuvants: These are immunological agents that help in modifying the effects of a drug or vaccine. Companies are now engaged in designing new adjuvants that will help in enhancing the immunogenicity of an antigen

Cost effective production systems for vaccines

Designing innovative drug delivery devices to enhance vaccine efficacy

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The North American therapeutic vaccine market is expected to generate the maximum revenue in the global market during the forecasted period due to the presence of efficient R&D facilities and sophisticated medical infrastructures. The market is segmented into three major application segments namely oncology, infectious diseases and other chronic disorders.

The therapeutic vaccine market with oncology applications market held the majority of the market share in 2011, as most of the research and development exercises are expected to be directed towards this segment during 2012 to 2018.

Merck and GSK have strong therapeutic vaccine product pipelines and are expected to perform better during 2012 to 2018 than their competitors who have fewer products in the pipeline. Some of the other key players of the market include Sanofi-Pasteur, Cytos Biotechnology AG, and Intellect Neurosciences.

This research report analyzes this market depending on its market segments, major geographies, and current market trends. Geographies analyzed under this research report include :-

North America ,Asia Pacific ,Europe ,Rest of the World 

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Global Multiple Sclerosis Drugs Market - Industry Analysis, Size, Share, Growth, Trends, And Forecast, 2012 - 2018

Multiple sclerosis (MS) is an inflammation of fatty myelin sheaths around the axons of the brain and spinal cord. MS tends to affect the ability of nerve cells to communicate with each other in the brain and spinal cord. MS affects people with random symptoms based on the location and severity of attack. One event can last for days, weeks or months and can affect any part of the body. Symptoms include imbalance, loss of motor function, spasms, numbness, incontinence, and difficulty in swallowing.

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There is no known cure for the disease currently and the only drugs available in the market are for delaying the progression of the disease. MS is estimated to affect 2.5 million people worldwide with around 5000 new cases developing each year. The National Multiple Sclerosis Society, Multiple Sclerosis Foundation, Multiple Sclerosis Association of America and several others are organizations which are determined to ensure improvement in the lives of affected people to make them more educated and spread awareness of this disease.

Until the last year, the MS drugs market was led primarily by injectables, yet this trend is changing very quickly to give way to oral MS drugs which is expected to grow strongly beginning from 2013. Gilenya and Aubagio are the top current oral drugs in this segment and Biogen’s oral BG-12 and Teva Laquinimod are two other drugs in the pipeline for approval with a significantly higher efficacy to be launched in the next five years. Although oral MS drugs seem to be growing quickly, it is not expected that they would unsettle injectables anytime soon. Several fatal side effects have been linked to use of Gilenya among those who have a heart disease and thus shows that there is still a long way to go for oral MS drugs.

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Top contenders in this segment till now are Copaxone, Avonex, Rebif, Betaseron, and Tysabri. Most of these drugs work actively to control MS relapses. Other treatments such as stem-cell transplantation are currently under investigation. The process includes the injection of stem cells which will replace the affected inflamed and diseased cells, greatly improving patient’s quality of life.

This research report analyzes this market depending on its market segments, major geographies, and current market trends. Geographies analyzed under this research report include : -

North America, Asia Pacific, Europe, Rest of the World 

This report is a complete study of current trends in the market, industry growth drivers, and restraints. It provides market projections for the coming years. It includes analysis of recent developments in technology, Porter’s five force model analysis and detailed profiles of top industry players. The report also includes a review of micro and macro factors essential for the existing market players and new entrants along with detailed value chain analysis.

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