Theme: Genomic therapies from base pairs to bedside
OMICS International welcomes you to attend the 4th International Conference and Exhibition on Cell & Gene Therapy during August 10-12, 2015 at London, United Kingdom. We cordially invite all the participants who are interested in sharing their knowledge and research in the arena of Cell & Gene Therapy.
Cell and Gene Therapy Conference is to ameliorate the knowledge, awareness, and education on cell and gene therapy leading to the discovery of genetic and cellular therapies which aid to alleviate the human disease as it is the most significant emerging technology in the eyes of Medical, Biotechnology, Pharmaceuticals and Academia. Cell and Gene Therapy Conference 2015 is an excellent opportunity for the delegates from Universities and Institutes to interact with the world class Scientists.
Cell Therapy Conferences will provide a perfect platform to all the Doctors, Researchers Business Delegates and Scientists to approach and deliver all the attendees about the latest scientific advancements on the respective sphere.
Gene Therapy Conferences strategic astuteness is to be an event for bringing together Scientists, Physicians, International mix of leading Universities, Cell Gene Therapy Institutions to transform the practice of medicine by incorporating the use of genetic and cellular therapies to control and cure human disease.
This three-day Gene Therapy Event will address key issues concerning cell and gene therapy in the broader context of cellular and genetic disorder. Organized around daily themes, the Conference focuses on moving from present knowledge to future solutions
London is the capital city of England and the United Kingdom. It is the most populous city in the United Kingdom. Standing on the River Thames, London has been a major settlement for two millennia, its history going back to its founding by the Romans, who named it Londinium. The global market for products to treat genetic disorders was worth $12.8 billion in 2009 and is estimated to reach $17.3 billion by 2014, a compound annual growth rate (CAGR) of more than 6%. The chromosomal disorders segment was valued at $1.5 billion in 2009. By 2014, this market is expected to be worth $1.8 billion, a compound annual growth rate (CAGR) of 3.7%. In the UK limbal stem cell therapy costs an average of £3,000/subject whereas a conventional corneal transplant is billed at £6,000.
OMICS International Organises 300+ International Conferences Every Year across USA, Europe & Asia with support from 1000 more scientific societies and Publishes 400+ Open access journals which contains over 30000 eminent personalities, reputed scientists as editorial board members.
The previous conferences on Cell & Gene Therapy which were held at Chicago, USA and San Antonio, USA have explained Research & Advancements in Cell and Gene Therapy, gained so much of interest for eminent scientists all over the world in the field of Cell & Gene Therapy. Now it is the time for the 4th International Conference and Exhibition on Cell & Gene Therapy to address Genomic therapies from base pairs to bedside which will be held from August 10-12, 2015 at London, UK.
For more Details Please Visit: http://www.conferenceseries.com/cell-gene-therapy-stem-cell-clinical-trails.php
Dendritic cells (DCs) represent unique antigen-producing cells capable of sensitizing T cells to both new and recall antigens. In fact, these cells are the most potent antigen-producing cells. The goal of DC based cancer immunotherapy is to use the cells to prime specific antitumor immunity through the generation of effector cells that attack and lyse tumors. Dendritic cell vaccines are increasingly being used to treat cancer in mainstream hospitals from The Preston Robert Tisch Cancer Center at Duke Medical School in Carolina to Cedars Sinai Medical Center in LA. The treatment, which is defined under the banner of Immunotherapy, aims to increase the body´s immune response against the cancer.
Probably the first use of Dendritic cell vaccines was at Stanford Cancer Institute where Dendritic cells (DCs) were isolated from patients with non-Hodgkin´s lymphoma. The DCs were loaded with immunoglobulins from the patients´ cancer cells. After re-injection a significant response was obtained from the T-cells and out of 6 patients, two had a complete response, with complete cancer regression. Most patients who are treated with dendritic cell therapy participate to clinical trials because for most cancers (except for prostate cancer) this cancer therapy is still in an experimental phase. Currently there is only one official approval granted by the American Food and Drug Administration in 2010, namely for the use of dendritic cell therapy in prostate cancer. This DC vaccine is marketed as Provenge® and costs approximately $93000 for a full course.
Fresh cell therapy also known as cell therapy, cellular therapy, live cell therapy and glandular therapy is a procedure that involves injecting cells or cell components of animals, usually sheep, into humans. Practitioners of fresh cell therapy believe that the procedure can have a healing and rejuvenating effect on the human body. T cells are white blood cells in the immune system that fight infection. The goal with PLAT is to reprogram a patient's own T cells so they can seek out and destroy cancer cells wherever they are hiding in the body. CTL019 is a clinical trial of T cell therapy for patients with B cell cancers such as acute lymphoblastic leukemia (ALL), B cell non-Hodgkin lymphoma (NHL), and the adult disease chronic lymphocytic leukemia (CLL). At this time, The Children's Hospital of Philadelphia is the only hospital enrolling pediatric patients on this trial. In July 2014, CTL019 was awarded Breakthrough Therapy designation by the U.S. Food and Drug Administration for the treatment of relapsed and refractory adult and pediatric acute lymphoblastic leukemia (ALL). The investigational therapy is the first personalized cellular therapy for the treatment of cancer to receive this important classification. As per the ASH meeting, Juno went public for a whopping $264.6 million, the largest biotech initial public offering (IPO) of 2014. Within a month, the company’s valuation rose from $2 billion to $4.7 billion, the largest among biotechs in a decade. By the end of 2016, the company plans to have 10 drug trials for six diseases up and running using CAR T cells produced in a brand-new manufacturing facility.
Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:
- Replacing a mutated gene that causes disease with a healthy copy of the gene.
- Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
- Introducing a new gene into the body to help fight a disease.
The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2014-2024. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright. The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets. Profiles of 183 companies involved in developing gene therapy are presented along with 225 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade.
Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included. Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2014, over 2050 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the geographical areas and applications. Since the death of Jesse Gelsinger in the US following a gene therapy treatment, the FDA has further tightened the regulatory control on gene therapy. A further setback was the reports of leukemia following use of retroviral vectors in successful gene therapy for adenosine deaminase deficiency. Several clinical trials were put on hold and many have resumed now. The report also discusses the adverse effects of various vectors, safety regulations and ethical aspects of gene therapy including germline gene therapy.
The U.S. National Institutes of Health, through its National Library of Medicine, has developed ClinicalTrials.gov to provide patients, family members and members of the public current information about clinical research studies. The database is a registry of federally and privately supported clinical trials conducted in the United States and around the world. ClinicalTrials.gov gives you information about a trial's purpose, who may participate, locations, and phone numbers for more details. Clinical gene therapy trials recently received at ClinicalTrials.gov database. Some of the clinical trials for gene therapy are mention below:
Gene Therapy for Transfusion Dependent Beta-thalassemia
Intervention: Genetic: Autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene
Sponsors: IRCCS San Raffaele; Fondazione Telethon
Recruiting - verified May 2015
Obesity is one of the most pressing problems in the U.S. Family studies have shown that genetic factors play a significant role in the pathogenesis of obesity. Rare mutations in humans and model organisms have provided insights into multiple pathways that may lead to obesity. Studies of candidate genes indicate that some genes involved in pathways regulating energy expenditure and food intake play a critical role in the predisposition of obesity. In obesity cases where the genetic defect is clearly identified, transfer of copies of a functional gene to diseased cells will provide a cure similar to monogenic diseases applicable in gene therapy. Although using gene therapy to treat obesity has been the major focus in the past, efforts have also been made in applying the gene therapy approach to obesity prevention. Progress has been made in recent years, demonstrating that gene transfer of exendin-4 (Di Pasquale et al., 2012; Samson et al., 2008), adiptonectin (Kandasamy et al., 2012; Ma and Liu, 2013), an anti-inflammatory cytokine such as IL-10 (Gao et al., 2013c), and the leptin gene (Kalra and Kalra, 2002) are highly effective in animal models in blocking diet-induced obesity. Our recent studies demonstrate that hydrodynamic delivery of the FGF21 gene enhances thermogenesis in adipose tissue and suppresses de novo lipogenesis in the liver, consequently leading to multiple beneficial metabolic effects in mice fed an HFD, including reduction in adiposity, alleviation of fatty liver, and improvement in glucose homeostasis (Gao et al., 2014).
Gene therapy is the therapeutic delivery of nucleic acid polymers into a patient's cells as a drug to treat disease. The polymers are either expressed as proteins, interfere with protein expression, or possibly correct genetic mutations.The most common form uses DNA that encodes a functional, therapeutic gene to replace a mutated gene. The polymer molecule is packaged within a "vector", which carries the molecule inside cells. Gene therapy was conceptualized in 1972, by authors who urged caution before commencing human gene therapy studies. The first gene therapy experiment approved by the US Food and Drug Administration (FDA) occurred in 1990, when Ashanti DeSilva was treated for ADA-SCID. By January 2014, some 2,000 clinical trials had been conducted or approved. Early clinical failures led to dismissals of gene therapy. Clinical successes since 2006 regained researchers' attention, although as of 2014, it was still largely an experimental technique. These include treatment of retinal disease Leber's congenital amaurosis, X-linked SCID, ADA-SCID, adrenoleukodystrophy, chronic lymphocytic leukemia (CLL), acute lymphocytic leukemia (ALL), multiple myeloma, haemophilia and Parkinson's disease. Between 2013 and April 2014, US companies invested over $600 million in the field. The first commercial gene therapy, Gendicine, was approved in China in 2003 for the treatment of certain cancers. In 2012 Glybera, a treatment for a rare inherited disorder, became the treatment to be approved for clinical use in either Europe or the United States after its endorsement by the European Commission.
Gene therapy in Parkinson disease consists in the creation of new cell that produces a specific neurotransmitter (dopamine), protect the neural system, or the modification of gene’s that are related to the disease. Then these cells are transplanted to a patient with the disease. There are different kinds of treatments that focus on reducing the symptoms of the disease but currently there is no cure. Parkinson's disease (PD) is a progressive neurological condition result of the death of the cell that contains and produces dopamine in substantia nigra. People with PD may develop disturbance in their motor activities. Some activities can be tremor or shaking, rigidity and slow movements (bradykinesia). Patients may eventually present certain psychiatric problems like depression and dementia. There are many new PD treatments in clinical trials and several of those are focusing on gene therapeutic approaches that compensate the loss of dopamine or protect the nervous system dopamine neurons from degeneration. There are some important reasons for focusing on gene therapy as a treatment for PD. The main idea of the gene therapy is to create new generations of cells that produce particular neurotransmitter (dopamine) and then transplant these cells to the patients with PD This is because the neurons cannot proliferate nor be renewed; and replacing lost neurons it is a process that is currently going under investigation. Also, the use of embryonic dopaminergic cells cannot be used because these cells are difficult to obtain and modifications of cell can only be made on somatic cells not germline. Startups like Voyager, Spark Therapeutics, Audentes Therapeutics, and others have emerged. Large companies like Pfizer, Bayer, Celgene, and Biogen Idec are placing bets with startups and research institutions in the space.
Progress in developing effective gene transfer approaches to treat HIV-1 infection has been steady. Many different transgenes have been reported to inhibit HIV-1 in vitro. However, effective translation of such results to clinical practice, or even to animal models of AIDS, has been challenging. Among the reasons for this failure are uncertainty as to the most effective cell population(s) to target, the diffuseness of these target cells in the body, and ineffective or insufficiently durable gene delivery. Better understanding of the HIV-1 replicative cycle, host factors involved in HIV-1 infection, vector biology and application, transgene technology, animal models, and clinical study design have all contributed vastly to planning current and future strategies for application of gene therapeutic approaches to the treatment of AIDS. This review focuses on the newest developments in these areas and provides a strong basis for renewed optimism that gene therapy will have an important role to play in treating people infected with HIV-1. University of Pennsylvania researchers have successfully genetically engineered the immune cells of 12 HIV positive patients to resist infection, and decreased the viral loads of some patients taken off antiretroviral drug therapy (ADT) entirely—including one patient whose levels became undetectable.
Gene Therapy has made important medical advances in less than two decades. Within this short time span, it has moved from the conceptual stage to technology development and laboratory research to clinical translational trials for a variety of deadly diseases. Among the most notable advancements are the following:
- Severe Combined Immune Deficiency (ADA-SCID): DA-SCID is also known as the bubble boy disease. Affected children are born without an effective immune system and will succumb to infections outside of the bubble without bone marrow transplantation from matched donors.
- Chronic Granulomatus Disorder (CGD): CGD is a genetic disease in the immune system that leads to the patients' inability to fight off bacterial and fungal infections that can be fatal.
- Hemophilia: Patients born with Hemophilia are not able to induce blood clots and suffer from external and internal bleeding that can be life threatening. In a clinical trial conducted in the United States, the therapeutic gene was introduced into the liver of patients, who then acquired the ability to have normal blood clotting time.
- Other genetic disorders: After many years of laboratory and preclinical research in appropriate animal models of disease, a number of clinical trials will soon be launched for various genetic disorders that include congenital blindness, lysosomal storage disease and muscular dystrophy, among others.
The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2014-2024. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright. The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets. Profiles of 181 companies involved in developing gene therapy are presented along with 223 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade
Regenerative stem cell therapy requires the liposuction of a smaller quantity of fat. The stem cells are then isolated and injected after suitable preparation. Due to the large number of stem cells in fat tissue, it is not necessary to expand the stem cells artificially in the laboratory. After the stem cells have been harvested and isolated, they can either be injected locally in a given organ or tissue or injected systemically, depending on the indication. On the basis of technology used, this market is segmented into small molecules & biologics, gene therapy and cell therapy. The small molecules and biologics segment have the largest contribution among all other technologies used for creating regenerative products. This is due to their efficiency in penetrating to internal membranes of the internal organs. However, gene therapies are expected to be the fastest emerging technology, growing at a CAGR of 28.6% during 2014-2020. The key reason for the growth of this technology can be attributed to the tremendous potential of gene therapy in minimizing immune rejections, which commonly occur after transplantations. On the basis of geography, this market can be classified into North America, Europe, Asia-Pacific and LAMEA. Currently, North America dominates the global market due to heavy investment in development of regenerative products as well as more number of commercialized products. However, the growing focus on research and development in Japan and South Korea makes Asia-Pacific the fastest growing region at a CAGR of 30.9% during 2014-2020.
Cell therapy is therapy in which cellular material is injected into a patient. Nowadays, various cell therapies are approved and used clinically. And many products are currently under active investigation worldwide and their market size is expected to grow rapidly in the near future. Ministry of Food and Drug Safety mentioned the global trend of stem cell therapy product in August 28, 2013 and said that market size will grow to 6.6 billion dollars in 2016 from 3.5 billion dollars last year. Global markets for stem cells by BCC Research (2012), cited by Ministry of Food and Drug Safety, predicted the global market size of stem cell therapy product would be 6.6 billion dollars in 2016 and grow 11.7% on average every year. Currently, US's stem cell therapy product occupying the biggest market share amounts to 1.3 billion dollars and Europe's is 872 million dollars. US's stem cell therapy product market is predicted to have an average annual growth rate of 11.5% (2.3 billion dollars in 2016), and Europe's is 10.9% (1.5 billion dollars in 2016). Until August 1, 2013, three stem cell therapies has acquired a sale permit for domestic use in Korea: Hearticellgram-AMI by FCB-Phamicell (the world's first stem cell therapy product), Cartistem by Medipost (the world's first allogenous stem cell therapy product) and Cupistem by Anterogen. Besides, 24 medicines are undergoing clinical trial till January 1, 2013. Companies profiled in this stem cell therapy industry research report include Aastrom Biosciences Inc., Advanced Cell Technologies, Inc., Athersys Inc., Celgene Corporation, Cellular Dynamics International, Cytori Therapeutics, Inc., Fibrocell Science, Inc., Gamida Cell Ltd., Mesoblast Ltd., Pluristem Therapeutics Inc. and Stemcells Inc
Some of the Research Programs at Universities and Institutions for Cell Therapy: Georgetown University Medical Center's Center for Cell Reprogramming Focuses on research in conditional reprogramming and its application to cancer biology and regenerative medicine. Harvard Stem Cell Institute Supports research into all aspects of stem cell biology, with special emphasis on those areas with the greatest potential for improving human health. Johns Hopkins Institute for Cell Engineering (ICE) Represents the stem cell research effort at the Johns Hopkins University School of Medicine. McGowan Institute for Regenerative Medicine Established for University of Pittsburgh School of Medicine and University of Pittsburgh Medical Center scientists and clinical faculty working to develop tissue engineering, cellular therapies, biosurgery, and artificial and biohybrid organ devices. Midwest Stem Cell Therapy Center (MSCTC) The center is housed within the University of Kansas Medical Center (KUMC) campus in Kansas City, Kansas. National Human Neural Stem Cell Resource Provides neural stem cells harvested from the post-natal, post-mortem, human brain to the research community for stem cell research. New York Stem Cell Science (NYSTEM) Supports basic, applied, translational or other research and development activities that will advance scientific discoveries in fields related to stem cell biology. Companies profiled in this stem cell therapy industry research report include Aastrom Biosciences Inc., Advanced Cell Technologies, Inc., Athersys Inc., Celgene Corporation, Cellular Dynamics International, Cytori Therapeutics, Inc., Fibrocell Science, Inc., Gamida Cell Ltd., Mesoblast Ltd., Pluristem Therapeutics Inc. and Stemcells Inc.
Cytokine therapy has been taken as a natural alternative for disease control. Disease control in food production animals is normally mediated through the use of vaccines, chemicals and antibiotics. Cytokine therapy has proven to be a novel therapeutic approach in treating patients with advanced malignancies. The purpose of this type of therapy is to manipulate the immune response in such a way as to generate the appropriate immune effector cells to eradicate solid tumors. Cytokine therapy is administrated only after the conventional form of therapies have been performed such as chemotherapy, radiotherapy, and surgery. Advances in the understanding of the role of cytokines in immune and inflammatory disorders have led to the development of cytokine-based therapies. Therapies have been developed with the express aim to block/inhibit or restore the activity of specific cytokines. Cytokines delivered by gene therapy and antisense oligonucleotide treatment are also being assessed. Some of the products that has been used are as follows: Bone morphogenetic protein (BMP), used to treat bone-related conditions; Erythropoietin (EPO), used to treat anemia; Granulocyte colony-stimulating factor (G-CSF), used to treat neutropenia in cancer patients; Granulocyte macrophage colony-stimulating factor (GM-CSF), used to treat neutropenia and fungal infections in cancer patient; Interferon alfa, used to treat hepatitis C and multiple scelerosis; Interferon beta, used to treat multiple scelerosis; In the past 25 years, numerous cytokine-based therapies and inhibitors of cytokines and their receptors have reached the market, with several of these products attaining over $1 billion in annual sales. Several such therapies represent established markets, but there are many new opportunities being pursued by pharmaceutical companies.
Genomic information has already helped to shape the development and use of some of the newest cancer treatments. For example, the drug imatinib (Gleevec) was designed to inhibit an altered enzyme produced by a fused version of two genes found in chronic myelogenous leukemia. Another example is the breast cancer drug trastuzumab (Herceptin), which works only for women whose tumors have a particular genetic profile called HER-2 positive. Studies have also found lung cancer patients whose tumors are positive for EGFR mutations respond to the drugs gefitinib (Iressa) and erlotinib (Tarceva) which target this mutation. The Global Genomics Market study is part of the Biotechnology subscription title, which also includes research in the following markets: Next Generation Sequencing (NGS) Market [Platforms (Illumina HiSeq, MiSeq, Life Technologies Ion Proton/PGM, 454 Roche), Bioinformatics (RNA-Seq, ChIP-Seq), (Pyrosequencing, SBS, SMRT), (Diagnostics, Personalized Medicine)] – Global Forecast to 2017, and Proteomics Market (Protein Microarray, Mass Spectrometry, NMR Spectroscopy, Chromatography, Electrophoresis, Surface Plasmon Resonance, X-ray Crystallography) - Instruments, Reagents & Services - Trends & Global Forecasts to 2017. Leading market players in this market include Roche Diagnostic (Switzerland), Life Technologies, Corp. (U.S.), QIAGEN (Netherlands), Illumina, Inc. (U.S.), and Bio-Rad Laboratories, Inc. (U.S.). New analysis from MarketsandMarkets "Genomics Market by Products - [Instruments (NGS platform, Microarray, RT-PCR), Consumables (Genechips, Reagents for DNA Extraction & Purification, Sequencing)], Services (Sequencing & Microarray Services, and Software) - Global Forecast to 2018" finds that the Global Genomics Market earned revenues of $11.11 billion in 2013 and estimates this market to reach over $19.0 billion by 2018.
Cancer is an abnormal, uncontrolled growth of cells due to gene mutations and can arise in most cells. No single mutation is found in all cancers. In healthy adults, the immune system may recognize and kill the cancer cells; unfortunately, cancer cells can sometimes evade the immune system resulting in expansion and spread of these cancer cells leading to serious life threatening disease. North America followed by Europe dominates the global market for cancer gene therapy market due to large number of aging population and technological advancement in the region. Asia is expected to witness high growth in cancer gene therapy market, due to increasing government initiatives, rising economy and improvement in healthcare infrastructure in the region. Some of the key driving forces for cancer gene therapy market in emerging countries are increasing R&D investment, large pool of patients and rising government funding. Innovation of some innovative therapy with better success rate is expected to offer good opportunity for cancer gene therapy. In addition, despite of high cost involved in R&D, pharmaceutical companies are showing increased interest in this field. This is expected to offer good potential for cancer gene therapy market. Currently, most of the cancer gene therapy products are in clinical trial phases. The market is expected to rise after clearance or success of these products from clinical trials. Some of the major companies operating in the global cancer gene therapy market are Cell Genesys, Advantagene, GenVec, BioCancell, Celgene and Epeius Biotechnologies. Other leading players in cancer gene therapy market include Introgen Therapeutics, ZIOPHARM Oncology, MultiVir and Shenzhen SiBiono GeneTech.
Cystic fibrosis is an inherited disease characterized by the buildup of thick, sticky mucus that can damage many of the body's organs. The disorder's most common signs and symptoms include progressive damage to the respiratory system and chronic digestive system problems. The features of the disorder and their severity varies among affected individuals. e US market for Cystic Fibrosis in Millions of US$. Annual estimates and forecasts are provided for the period of 2007 through 2015. Also, a six-year historic analysis is provided for this market. The report profiles 56 companies including many key and niche players such as Abbott Laboratories, Axcan Pharma, Inc., Digestive Care, Inc., Gilead Sciences, Inc., Eurand N.V., Inspire Pharmaceuticals, Inc., KaloBios Pharmaceuticals, Inc., Nektar Therapeutics, PTC Therapeutics, Inc., Transave, Inc., Vertex Pharmaceuticals, Inc., Actelion Ltd., F. HoffmannLa Roche Ltd., Novartis AG, and Pharmaxis Ltd. Market data and analytics are derived from primary and secondary research. The value of the Cystic Fibrosis (CF) market is expected to increase significantly in value over the forecast period across the leading eight developed nations, from $695.6 million in 2012 to almost $4.5 billion in 2019. This equates to a Compound Annual Growth Rate (CAGR) of 30.4%. Novel treatments with disease-modifying mechanisms of action are the primary factor driving the growth of the value of the market. The positive impact of new market entrants will offset the effects of key patent losses during the forecast period. In 2015 – 2016, Cystic Fibrosis Canada is funding more than $5.2 million in leading-edge research, supporting 46 research projects, 24 fellowship and studentship training awards, two national core facilities, and targeted research programs and partnerships aimed at advancing CF knowledge and achieving excellence in the quality of care for CF patients.
Gene transfer can be targeted to somatic (body) or germ (egg and sperm) cells. In somatic gene transfer the recipient's genome is changed, but the change is not passed on to the next generation. In germline gene transfer, the parents' egg and sperm cells are changed with the goal of passing on the changes to their offspring. Germline gene transfer is not being actively investigated, at least in larger animals and humans, although a great deal of discussion is being conducted about its value and desirability. Thus, both kinds of studies - germline gene transfer at the gamete and zygote stages - have significant risks. In cases in which the gene has failed to be introduced or fails to be activated, the resulting child would likely be no worse off than he or she would have been without the attempted gene transfer. However, those with partial or multiple copies of a gene could be in significantly worse condition. The problems resulting from errors caused by the gene insertion could be severe - even lethal - or they might not be evident until well after the child has been born, perhaps even well into adulthood, when the errors could be passed on to future generations. Gene transfer clinical trials have a unique oversight process that is conducted by the National Institutes of Health (NIH) through the Recombinant DNA Advisory Committee (RAC) and the NIH Guidelines for Research Involving Recombinant DNA Molecules and by the Food and Drug Administration (FDA) through regulation (including scientific review, regulatory research, testing, and compliance activities, including inspection and education). Of note, FDA regulations apply to all clinical gene transfer research, while NIH governs gene transfer research that is supported with NIH funds or that is conducted at or sponsored by institutions that receive funding for recombinant DNA research.
All viruses attack their hosts and introduce their genetic material into the host cell as part of their replication cycle. This genetic material contains basic 'instructions' of how to produce more copies of these viruses, hijacking the body's normal production machinery to serve the needs of the virus. The host cell will carry out these instructions and produce additional copies of the virus, leading to more and more cells becoming infected. Some types of viruses actually physically insert their genes into the host's genome. This incorporates the genes of that virus among the genes of the host cell for the life span of that cell. Houston's MultiVir, a developer of viral vectors to deliver anticancer gene therapy, just filed for a $70 million IPO with the Securities and Exchange Commission as it seeks funding for clinical trials of its Phase I/II lead candidates for colorectal cancer and head and neck cancer, and to take the first FDA-approved gene therapy to the market. MultiVir reported a loss of $2.9 million in 2014, up from $1 million in 2013. R&D expenditure more than doubled during that time period to $1.67 million in 2014. A $70 million IPO would allow it to keep investing in drug delivery and biological research at a significantly high rate, in the hopes of a payoff down the road for patients and Wall Street investors.
- Cell and Gene Therapy: Potential Applications
- Plant Stem Cell Rejuvenation
- Plant Stem Cells: Human Therapeutics
- Stem Cell Therapies
- Cellular Therapies
- Advanced Gene Therapeutics
- Molecular basis of epigenetics
- Cancer Therapies
- Bioengineering Therapeutics
- Clinical Trials and Research in Cell and Gene Therapies
- Regulatory and Ethical Issues of Therapies
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