Genetic Disorders

DelveInsight’s Gene Therapy Reports: Launched

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Immense Growth Potential in the Gene Therapy Market!

DelveInsight has launched its Gene Therapy Report: “Gene Therapy Insight: Pipeline Assessment, Market Trend, Technology and Competitive Landscape”. These Reports are the outcome of very best analytical abilities and diligent market research amalgamated with opinions of gene therapy industry experts. The report uncovers the potential of global Gene Therapy Market with insights into 15 Therapy Areas with collective pipeline of more than 300 gene therapies and the companies at their forefront.

Report Summary

These reports provide information across the gene therapy value chain covering gene therapy profiles core insights, pre-clinical data, clinical data, technology details, funding and licensing opportunities. The Report provides the gene therapy targets which are close to 190 with the target gene name, localization of gene, molecular function of target with descriptive mechanism of action. Using the propriety DelveInsight Competitive Matrix models, the report also provides the first in class market analytics providing predictive analysis of early market winners of the clinical therapies and pre-clinical therapies in a demographic presentation view.

Report Highlights

  • Global Gene Therapy Market & Pipeline Insight.
  • Trends in gene therapy partnering deals.
  • Current Prominent Research Areas and Key Players.
  • Companies Targeting Prominent Therapeutic Areas.
  • Number of Gene Therapies in Clinical Trials.
  • Number of Gene Therapies by Vectors used.
  • Technology and their innovative Companies.
  • Early Market Winners for Gene Therapy.

Scope

Reasons to buy

The Report insight covers therapy areas like; Oncology, Central nervous system, Genetic Disorders, Hematological disorders, Metabolic disorders, Ophthalmology, Cardiovascular disease, Respiratory, Immunology and other therapy areas.

For more information on Gene Therapy reports for various therapeutic areas contact us at info@delveinsight.com.

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Gene Therapy’s Emergence: The “New” approach for Huntington’s disease

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Many companies have tried over the years to find a way to reverse the course of crippling neurodegenerative disorders like Huntington’s but have failed. Fortunately, Gene therapy has proven to be an answer to a wide range of serious neurodegenerative disorders like Huntington’s, and Parkinson’s etc.

The HD gene vs Gene Therapy

Huntington’s disease (HD) is an incurable, inherited disease entailing progressive loss of brain cells and motor function due to a defective gene (HD gene) which produces repeated copies of a defective protein called huntingtin, or mHTT which particularly damages a brain region called the striatum. About 30,000 Americans have Huntington’s disease (HD). Fortunately, Gene therapy which is a novel therapeutic branch of modern medicine has shown the potential for curing this disease by allowing the researchers to transfer genetic information into patient tissues and organs in order to eliminate or restore the normal functions of the diseased genes. A variety of gene therapy approaches have been tested in mouse models of HD, ranging from those aimed at ameliorating downstream pathology or replacing lost neuronal populations to more upstream strategies like gene silencing to reduce mHtt levels.

What’s already happening?

Companies like Genethon are working to provide effective treatment for curing Huntington’s disease through intrastriatal administration of a lentiviral vector carrying the gene hCNTF (Ciliary Neurotrophic Factor Human), a neuroprotective agent which protects striatal cells and maintains basal ganglia connectivity. This project is based upon ectopic expression of neurotrophic factors mediated by lentiviral vectors and is being conducted in partnership with Reference Centre for Huntington Disease as well as MIRCen (Molecular Imaging Research Center).

Another institute viz, The Children’s Hospital of Philadelphia’s (CHOP) gene therapy is also working on this disorder and recently experts have found a way to fine tune protein signals in order to provide significant relief for patients suffering from this disorder. This study is based upon adjusting the levels of a key signaling protein, in order to improve motor function and brain abnormalities in experimental animals with a form of Huntington’s disease. Neuroscientists already know that a signaling protein called mTORC1 regulates cell growth and metabolism and that it plays a key role in Huntington’s disease (HD). But the current study at The Children’s Hospital of Philadelphia’s (CHOP) Center for Cellular and Molecular Therapeutics contradicts the assumptions that inhibiting or shutting off the mTORC1 pathway, which interacts with the deleterious mHTT proteins, could help treat HD. Their study has shown that the mTORC1 pathway is already impaired in Huntington’s disease, and that improving this pathway’s functions can actually have a protective effect. However, restoring that pathway must be done very carefully as either too much or too little is detrimental. This proves that brain cells are capable of responding even after disease onset, and hints at the potential for reversing Huntington’s disease. (For details on this study, refer to http://www.ncbi.nlm.nih.gov/pubmed/25556834)

What’s hope and what’s hype?

Inspite of its checkered past gene therapy investigators in the 21st century agree that the field is enjoying a renaissance and that it has emerged with enormous potential in the field of Neurodegenerative disorders. Many companies like UniQure Biopharma, Sanofi, Oxford BioMedica etc., are operating in the field of Neurodegenerative disorders in the gene therapy domain. A growing number of partnership between companies in drug development for example between Audentes and Genethon etc., are driving the new gene therapy research. Thus leading to an increase in the global market opportunities for gene therapy with more companies focusing in this field.

DelveInsight’s Reports have already established a reputation of offering the affordable and comprehensive industry coverage and “on-the-ground” analysis in virtually every region of the world. These reports provide complete information for over 300 gene therapies which are in the pipeline for various therapy areas like; Oncology, Genitourinary, Dermatology, Central nervous system, Genetic Disorders, Hematological disorders, Metabolic disorders, Ophthalmology, Cardiovascular disease, Respiratory , Immunology, and many more…

DelveInsight’s Gene Therapy Reports cover the entire gene therapy market scenario including technology assessments, licensing opportunities, collaborations, market trends, pipeline coverage and competitive landscape. The report essentially provides DelveInsight’s proprietary market and pipeline analytics which identifies the front runners of all therapeutic areas. It also identifies the potential market movers and future regulatory landscape. These reports are designed to provide the clients with the means to out produce their competitors by developing a product that makes history.

For more info on Gene Therapy Reports for various Therapeutic areas contact us at: info@delveinsight.com.

Are you ready to embrace Gene Therapy? DelveInsight’s Gene Therapy Reports

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Gene Therapy: The “Big Value” Market!

Gene therapy has been under scientific research for over 2 decades, but viable therapies have yet to gain commercial acceptance due to safety and delivery-related issues. However, Pfizer’s move and some other recent developments in the industry suggest that the therapy may be coming off age. With the superlatively well-funded biotech Moderna Therapeutics raising another $450 million in venture cash, for its mRNA based proprietary approach to treat previously undruggable targets in a wide range of disease areas (with 45 preclinical programs in its pipeline), it has become clear that the Gene therapy market is going to be of great interest for many companies globally due to the possibility of a permanent cure that it offers for any of the more than 10,000 human diseases caused by a defect in a single gene.

About Gene Therapy

Gene therapy aims to fix a genetic problem at its source. By adding a corrected copy of a defective gene, gene therapy promises to help diseased tissues and organs work properly. This approach is different from traditional drug-based approaches, which may treat symptoms but not the underlying genetic problems. Put simply, it introduces a “good” gene into a person who has a disease caused by a “faulty” gene.

Gene Therapy Reports: Understanding the potential of Gene Therapy Market

During the past two decades gene therapy has made important medical advances. 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. The researchers have learned from their mistakes and developed much more realistic approach and solutions to many of the problems which led to major clinical and commercial successes. It has become an upcoming research area in 21st century with the industry’s collective pipeline brimming with therapies close to 300.

There is a significant increase in the companies coming up with gene therapies for various therapeutic areas such as Novartis, GlaxoSmithKline, Sanofi, UniQure Biopharma, Oxford BioMedica etc., which are operating in the gene therapy domain. Moreover recent academic and industry partnerships for example between Celgene Corporation and Baylor College of Medicine, GSK and the Roswell Park Cancer Institute etc., are driving new gene therapy research.

DelveInsight’s Gene Therapy Reports

DelveInsight’s Gene Therapy Reports cover the entire gene therapy market insights including technology assessments, licensing opportunities, collaborations, market trends, pipeline coverage and competitive landscape. The report essentially provides DelveInsight’s proprietary market and pipeline analytics which identifies the front runners of all therapeutic areas. It also identifies the potential market movers and future regulatory landscape. These reports provide complete insight over all the therapy areas associated with gene therapy like; Oncology, Genitourinary, Dermatology, Central nervous system, Genetic Disorders, Hematological disorders, Metabolic disorders, Ophthalmology, Cardiovascular disease, Respiratory , Immunology, and many more…

DelveInsight’s Gene Therapy Reports: Reasons to buy

  • Complete Market and Pipeline intelligence and complete understanding over therapeutics development for gene therapy.
  • Devise corrective measures for pipeline projects by understanding gene therapies for specific therapy areas.
  • Developing strategic initiatives to support your gene therapy development activities.
  • Optimize your portfolio and keep you in touch with the rapidly changing pharmaceutical markets, and make the best decisions for your business.
  • Develop and design in licensing and out licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope
  • Evaluate the marketing and pipeline strategy for gene therapies and their Funding availabilities.
  • Identifying the upcoming leaders in the gene therapy market in the coming years.
  • Getting a first mover advantage by identifying the early market winners for clinical and preclinical gene therapies.

For more info on Gene Therapy Reports for various Therapeutic areas refer to the following links:

DelveInsight’s Oncology based Gene Therapy Reports

DelveInsight’s Otolaryngology based Gene Therapy Reports

DelveInsight’s Central Nervous System based Gene Therapy Reports

DelveInsight’s Genitourinary & Sex hormones based Gene Therapy Reports

DelveInsight’s Dermatology based Gene Therapy Reports

DelveInsight’s Genetic disorders based Gene Therapy Reports

DelveInsight’s Gastrointestinal based Gene Therapy Reports

DelveInsight’s Infectious disorders based Gene Therapy Reports

DelveInsight’s Metabolic disorders based Gene Therapy Reports

DelveInsight’s Musculoskeletal disorders based Gene Therapy Reports

DelveInsight’s Cardiovascular disorders based Gene Therapy Reports

DelveInsight’s Immunology based Gene Therapy Reports

DelveInsight’s Respiratory disorders based Gene Therapy Reports

DelveInsight’s Hematological disorders based Gene Therapy Reports

DelveInsight’s Ophthalmologic disorders based Gene Therapy Reports

DelveInsight’s Genetic disorders based Gene Therapy Reports

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Gene Therapy to “fix” Genetic disorders!

“We used to think that our fate was in our stars, but now we know that, in large measure, our fate is in our genes, “quotes James Watson. This fate and our future is locked in our genes, and Gene therapy is unlocking these doors. Many physicians are predicting that in twenty years gene therapy may change the practice of medicine from a treatment-based to a prevention-based practice.

About Gene Therapy

Gene therapy is a novel therapeutic branch of modern medicine. Its emergence is a direct consequence of the revolution heralded by the introduction of recombinant DNA methodology in the 1970s. Gene therapy is still highly experimental, but has the potential to become an important treatment regimen. Basically, it allows the transfer of genetic information into patient tissues and organs in order to eliminate or restore the normal functions of the diseased genes.

Genetic disorders affect millions of people world-wide. Scientists have currently identified more than 4000 different genetic disorders. These are the harmful effects on an individual caused by inherited genetic diseases or mutations. Usually genetic disorders are recessive, so they are only expressed in a small percentage of the population, but a much larger percentage are carriers. When expressed in the homozygous recessive individual, they often code for the wrong protein or amino acid sequence. Gene therapy aims to get rid of these genetic conditions at their source.

Gene Therapy: Mending Genetic disorders

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. During this period molecular genetic technologies have been spectacularly successful in identifying and characterizing novel disease genes, and in devising novel diagnostic tests for inherited disorders. Current and possible candidates for gene therapy in the field of genetic disorders include Severe Combined Immune Deficiency (SCID), Adenosine deaminase (ADA) deficiency and Duchenne muscular dystrophy (DMD) etc.

Gene therapy is likely to be most successful with diseases caused by single gene defects which is due to the absence of complex interplay between different genetic loci and/or environmental factors. However, some genetic disorders may not be so easy to treat as others and within the single gene disorder category differing pathogeneses means that certain single gene disorders will be more amenable to gene therapy approaches than others. However, low efficiency of gene transfer & expression and low longevity of gene expression are the acting limitations in this field. Inspite of this, there is much optimism and confidence in the potential of Gene therapy to develop an effective treatment for genetic disorders in the near future.

Gene Therapy: The Market Scenario

Many companies are investing in and researching on this field using gene therapy due to its promising effects. Large Pharmaceutical and Biotech giants, such as GlaxoSmithKline, Sanofi, and Bluebird Bio etc., are operating in the field of Genetic disorders in the gene therapy domain. A growing number of partnership between companies in drug development for example between Oxford BioMedica and Sanofi etc., are driving the new gene therapy research. The collective pipeline of industry is brimming with 300+ therapies of which genetic disorders collectively accounts for 11 % of gene therapies in pipeline. The global market opportunities for gene therapy have risen drastically and continue to be of great interest for many companies globally.

DelveInsight’s Gene Therapy Reports

DelveInsight’s Gene Therapy Reports cover the entire gene therapy market insights for genetic disorders including technology assessments, licensing opportunities, collaborations, market trends, pipeline coverage and competitive landscape. The report essentially provides DelveInsight’s proprietary market and pipeline analytics which identifies the front runners in this therapeutic area. It also identifies the potential market movers and future regulatory landscape.

Break the Gene Therapy Barrier with DelveInsight’s Gene Therapy Reports

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Gene Therapy: The Future is here!

Scientific research and clinical trials aren’t glamorous, but they are essential for effective treatments and perhaps ultimately a cure for various debilitating diseases like cancer etc. Gene therapy’s potential to revolutionize medicine in the future is exciting, and one day it may be possible to treat an unborn child for a genetic disease even before symptoms appear.

About Gene Therapy

Gene Therapy involves using genes as medicines by transferring a therapeutic gene through a genetically engineered vector into the targeted cells of a patient to repair an unhealthy gene thus help in curing disease by synthesizing correct proteins. Put simply, it introduces a “good” gene into a person who has a disease caused by a “bad” gene.

Gene Therapy: The Past

Gene therapy has had rather a bad decade as it got off with two unapproved trials in the early 70s and early 80s in which first one was an attempt made to treat two young girls with arginase deficiency syndrome using in vivo gene therapy with wild-type Shope papilloma virus in order to replace the missing arginase enzyme in the patients and the second one was ex vivo gene therapy for ß-thalassemia, a bone marrow transplant using marrow cells treated with a ß-globin-containing plasmid, again in two patients. Unfortunately in neither case was any real follow-up reported, since both trials were stopped. The final blow to Gene therapy field came in September 1999 with the widely publicized death of an eighteen-year-old man undergoing gene therapy for a liver disease at the University of Pennsylvania, an event that brought the scrutiny of the press onto the world of gene therapy.

But at the turn of the millennium, through the year 2000, reports of the first successful trials began to be heard indicating that real success may be just around the corner: factor IX for hemophilia, transduced stem cells for X-linked SCID, and oncolytic vectors etc. In the ten year cycle of failed clinical trials the researchers learned from their mistakes and developed much more realistic approach and solutions to many of the problems which led to major clinical and commercial successes.

Gene Therapy: The Present Scenario

The past two decades have seen significant increase in the companies coming up with gene therapies for various therapeutic areas. The human clinical trials are majorly performed for the oncology indications. However, the market for gene therapy is currently in the experimental stages due the risk involved with the use of genes designed in laboratory growth as well as the regulatory approval of products which are currently in clinical trials resulting in only three drugs hitting the market in past decade. Inspite of this, Gene therapy has proved to be a promising approach for many incurable diseases with many promising therapies in phase III clinical trials.

Gene Therapy: The Market Scenario

Many companies have started investing in and researching on gene therapy due to its promising effects. A growing number of Big Pharma companies are partnering in drug development with innovation-rich biotech firms. Large Pharmaceutical and Biotechnology giants, such as Novartis, GlaxoSmithKline, Sanofi etc., are operating in the gene therapy domain. Moreover recent academic and industry partnerships for example between Celgene Corporation and Baylor College of Medicine, GSK and the Roswell Park Cancer Institute etc., are driving new gene therapy research. The industry’s collective pipeline is brimming with 300+ drugs of which oncology collectively accounts for 29 % of gene therapies in pipeline. Despite of high cost involved recent years have seen many companies coming up for the treatment of deadly diseases. The global market opportunities for gene therapy have risen drastically and continue to be great area of interest for many companies globally.

DelveInsight’s Gene Therapy Reports

DelveInsight’s Gene Therapy Reports cover the entire gene therapy market insights including technology assessments, licensing opportunities, collaborations, market trends, pipeline coverage and competitive landscape. The report essentially provides DelveInsight’s proprietary market and pipeline analytics which identifies the front runners of all therapeutic areas. It also identifies the potential market movers and future regulatory landscape.

The Report insight covers therapy areas like; Oncology, Central nervous system, Genetic Disorders, Hematological disorders, Metabolic disorders, Ophthalmology, Cardiovascular disease, Respiratory and Immunology and many more…

Get the First Mover Advantage in “The Big Value” Gene Therapy Market

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Drive your Research towards a Brighter Future with DelveInsight!

What do you feel when you see, cancer is getting cured by replacing the faulty or missing gene with the functional gene so that they can produce a protein to repair the anomaly responsible for the disease.

Awesome Innovation Right!Pre_Launch

DelveInsight is proud to announce the Pre-Launch of its Gene Therapy reports. These Reports are the outcome of very best analytical abilities and diligent market research amalgamated with opinions of gene therapy industry experts.

Are you an active Player in Gene Therapy! Get the first mover Advantage. Out produce everyone around you and develop a product that will make history!

Emerging Role of Gene Therapy

Every day we see the impact of innovation. Of most of the innovation delivered, Gene therapy is the biggest innovation. The scientific society has seen many transformations in past two decades with respect to gene therapy market. It has become an upcoming research area in 21st century with the industry’s collective pipeline brimming with therapies close to 300. Gene Therapy is preferred over drugs and surgery as it’s a onetime treatment which restores natural body function and did not just offer a short-term fix.

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Gene Therapy Research Companies and their Roles

Many companies have started investing in and researching on gene therapy due to its promising effects. A growing number of Big Pharma companies are partnering in drug development with innovation-rich biotech firms. Large Pharmaceutical and Biotechnology giants, such as Novartis, GlaxoSmithKline, Sanofi etc., are operating in the gene therapy domain. Moreover recent academic and industry partnerships for example between Celgene Corporation and Baylor College of Medicine, GSK and the Roswell Park Cancer Institute etc., are driving new gene therapy research.

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Gene Therapy Market Outlook

Gene Therapy involves using genes as medicines by transferring a therapeutic gene through a genetically engineered vector into the targeted cells of a patient to repair an unhealthy gene thus help in curing disease by synthesizing correct proteins. It involves the administration of DNA or RNA to modify the expression of genes or correct abnormal genes and is widely used in curing several disorders related to Oncology, Metabolic, Central Nervous system and Genetic etc., with oncology collectively accounting for 29 % of gene therapies in pipeline. However, the market for gene therapy is currently in the experimental stages due the risk involved with the use of genes designed in laboratory growth as well as the regulatory approval of products which are currently in clinical trials resulting in just three drugs hitting the market in past decade. Inspite of this, Gene Therapy has proved to be a promising approach for many incurable diseases.

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What DelveInsight Have for You!

DelveInsight is launching its Gene Therapy Reports in coming week. DelveInsight Gene Therapy Reports cover the entire gene therapy market insights including technology assessments, licensing opportunities, collaborations, market trends, pipeline coverage and competitive landscape. The report essentially provides DelveInsight’s proprietary market and pipeline analytics which identifies the front runners of all therapeutic areas. It also identifies the potential market movers and future regulatory landscape.

The Report insight covers therapy areas like; Oncology, Central nervous system, Genetic Disorders, Hematological disorders, Metabolic disorders, Ophthalmology, Cardiovascular disease, Respiratory and Immunology and many more…

DelveInsight’s Gene Therapy Reports are available for booking immediately after pre-launch so hurry up book your report and get 1 year subscription for the quarterly updated Gene therapy Report for free by paying additional amount of USD 500. Claim The Offer

For more information contact Website: www.delveinsight.com

Email: info@delveinsight.com

Phone Number: +91-11-4568 9769, +91 9650213330