Month: December 2014

DelveInsight’s Ophthalmologic disorders based Gene Therapy Reports

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Gene Therapy: Eye for the cure!

Recent technical advances have led to the demonstration of the molecular basis of various ocular diseases. Gene transfer into ocular tissues has been demonstrated with growing functional success and may develop into a new therapeutic tool for clinical ophthalmology.  There are prospects for commercially available gene therapies for retinal disease in the near future and one thing is for certain that the future is brighter for thousands of patients with inherited retinal degenerations potentially amenable to treatment with this technology.

About Gene Therapy

Gene therapy is the addition of new genes to a patient’s cells to replace missing or defective copies, to restore or impart a new function to overcome a disease usually of genetic origin. Over the past several years, the unlocking of the human genome and the discovery that certain genes, or lack thereof or genetic defects therein, can be the cause of certain diseases has led to the ability to identify genes associated with retinal and other ocular diseases. According to the eyeGene National Ophthalmic Disease Genotyping Network, more than 100 ocular gene types have been identified, and the number increases yearly. To date, the genes for some 35 ocular disorders have been identified. Ophthalmologic disorders are responsible for 48% of the population becoming totally blind. In addition, more than 60 million people suffer from glaucoma and an increasing aging population is also resulting in more people suffering from refractive errors. It is estimated that in the U.S. and Europe, refractive errors affect more than 30% of the population aged 40 or older. Ocular gene therapy research has made rapid progress in the past few years. Although laboratory and animal experiments started were successful many years ago, the application in human beings took very long due to several biological and regulatory hurdles. However, the recent successful gene therapy clinical trials are promising and encouraging.

Gene Therapy: Role in Ophthalmological disorders

The eye is an attractive target for gene therapy because of its accessibility and its immune privilege. Significant advancements have been made in understanding the genetic pathogenesis of ocular diseases, and gene replacement and gene silencing have been implicated as potentially efficacious therapies. Recent improvements have been made in the safety and specificity of vector-based ocular gene transfer methods. Proof-of-concept for vector-based gene therapies has also been established in several experimental models of human ocular diseases. Novel methods are being developed to enhance the performance and regulation of recombinant adeno-associated virus and lentivirus-mediated ocular gene transfer. Gene therapy prospects have advanced for a variety of retinal disorders, including retinitis pigmentosa, retinoschisis, Stargardt disease and age-related macular degeneration. Advances have also been made using experimental models for non-retinal diseases, such as uveitis and glaucoma.

Current and possible candidates for gene therapy in the field of Ophthalmological disorders include Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy), Juvenile Macular Degeneration (Stargardt Disease) and Ocular Pain etc. The three main types of gene therapies used in the field of ophthalmological disorders are gene replacement for loss-of-function mutations, gene knockdown for gain-of-function mutations, and gene enhancement/knockdown for non-monogenic diseases. However, all of these approaches have historically been subject to the same limitations: 1) how to deliver the vector into the affected cells 2) how to achieve broad distribution throughout the tissue of interest 3) how to maintain persistent transgene expression and functional rescue and 4) how to avoid both local and systemic toxic responses. Inspite of this gene therapy holds the promise of curing ocular diseases, and improving the quality of life for millions who suffer from visual impairments.

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 Applied Genetic Technologies Corporation (AGTC), Oxford Biomedica, and Pfizer etc., are operating in the field of ophthalmologic disorders in the gene therapy domain. A growing number of partnership between companies in drug development for example between Sanofi and Oxford Biomedica etc., are driving the new gene therapy research. The industry’s collective pipeline is brimming with 300+ therapies for various therapeutic areas. The recent success of gene replacement therapy for ophthalmological disorders is a big step forward in the field of genomic medicine. These results have enthused the medical community and basic scientists equally and have unveiled the potentials that is in store for the future of medicine. Once these experiments are refined and tailored to the needs of these patients with unambiguous success, nearly 500 eye genetic diseases and 1500 genetic diseases in other parts of the body could be potentially cured.

DelveInsight’s Gene Therapy Reports

DelveInsight’s Gene Therapy Reports cover the entire gene therapy market insights for ophthalmologic 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.

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DelveInsight’s Hematological disorders based Gene Therapy Reports

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Gene Therapy to “target” Hematological disorders!

About Hematological disorders

Hematology is the study of blood in health and disease. It includes problems with the red blood cells, white blood cells, platelets, blood vessels, bone marrow, lymph nodes, spleen, and the proteins involved in bleeding and clotting (hemostasis and thrombosis).

Haemoglobin comprises of four globin chains: fetal haemoglobin (Hb F) which has two α and two gamma chains (α2γ2) and adult haemoglobin (Hb A) which has two α and two β chains (α2β2). Genes in the α-globin and β-globin gene clusters (on chromosomes 16 and 11) control globin-chain production. However, due to spontaneous mutation, haemoglobin gene variants are present at low prevalence (carriers 1–1.5/1000) in all sizeable populations. They fall into two broad groups – structural variants that change the amino acid sequence and produce an unusual haemoglobin, and thalassaemias that lower or abolish production of globin chains. Prevention of hematological disorders is making only a small impression as affected birth prevalence is estimated at 2.55 per 1000. The yardstick of under-5 mortality depicts the broad effect of hematological disorders on health, because most affected children die in early childhood and most survivors have chronic disease. It has long been the hope that one day it would be possible to cure these diseases by fixing the mutation that causes the disease and thus affect a cure. As a result of the various technological advancements over the past decade, we now know the identities of the specific genes involved in vast majority of these hematological diseases. Gene therapy aims to correct this disease process by restoring, modifying or enhancing cellular functions through the introduction of a functional gene into a target cell.

Gene Therapy: Role in Hematological disorders

The compelling desire to target therapy at a molecular level has resulted in the emergence of new direction in medicine, called gene therapy. The simplicity of the basic concepts of gene therapy together with exciting early results in animal models have fuelled great enthusiasm for this field. Gene therapy has emerged as an avenue of hope for many hematological disorders that cannot be effectively treated with drugs, recombinant therapeutic proteins or transplantation. In addition, gene therapy has the potential to transform the lives of those patients who are dependent on life-long parenteral therapy with recombinant proteins or blood and its products. A number of preclinical and clinical studies have been conducted with the aim of developing gene therapy for hemophilia, Fanconi anemia, sickle cell disease, beta-thalassemia, chronic granulomatous disease, and other hematological disorders. Results from these studies and from preclinical studies preceding the trial demonstrate that it is possible to safely administer high doses of a viral vector in vivo. Thus, making gene therapy a promising candidate for treating Hematological disorders.

Gene Therapy: The Market Scenario

Gene therapy is an emerging field which has the potential of altering the world of diagnosis, treatment and drug discovery. Gene therapy treatments have shown promise in the field of haematology as a result the market opportunities for gene therapy in the field of hematological disorders have been on a rise with large biopharmaceutical companies such as Genethon, uniQure biopharma. etc. operating in this domain. A growing number of partnership between companies in drug development for example between Anges and Daiichi Sankyo etc., are driving the new gene therapy research. The industry’s collective pipeline is brimming with 300+ therapies for various therapeutic areas. However, the immediate challenges of the field as it moves toward clinical trials are to optimize gene transfer and to minimize the adverse consequences that can result from random integration of vectors into the genome by improving current vector design or developing novel vectors.

DelveInsight’s Gene Therapy Reports

DelveInsight’s Gene Therapy Reports cover the entire gene therapy market insights for hematological 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.

DelveInsight’s Respiratory disorders based Gene Therapy Reports

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Gene Therapy’s “expanding” role in Respiratory disorders!

About Respiratory disorders

Respiratory disorders encompasses pathological conditions affecting the organs and tissues that make gas exchange possible in higher organisms, and includes conditions of the upper respiratory tract, trachea, bronchi, bronchioles, alveoli, pleura and pleural cavity, and the nerves and muscles of breathing.

Respiratory disorders affect both children and adults. Using a conservative definition, it is estimated that millions of people of all ages and all ethnic backgrounds suffer from respiratory disorders. These disorders are the leading cause of death in the United States. According to WHO respiratory disorders lead to million deaths worldwide, representing 1% of the total global disease burden. It appears that the global prevalence of respiratory disorders ranges 1–18% of the population in different countries.

Gene Therapy: Role in Respiratory disorders

Until the late 1990s, aerosol therapy consisting of beta2-adrenergic agonists, anti-cholinergics, steroidal and non-steroidal agents, mucolytics and antibiotics were used to treat patients with asthma, COPD and cystic fibrosis. But with the recent technological advancements the use of gene therapy as a treatment modality for pulmonary disorders has attracted significant interest with the lung becoming an important target organ for gene therapy over the past decade. This is because there are a number of lung diseases that could benefit from this type of treatment. These include: Emphysema, alpha-1-antitrypsin deficiency and cystic fibrosis etc. Since the initiation of the first clinical trials for cystic fibrosis lung disease using recombinant adenovirus in the early 1990s, the field has encountered numerous obstacles. Despite these obstacles, enthusiasm for lung gene therapy remains high. A broad range of respiratory diseases have been identified as potential targets for gene therapy. However, the biggest hurdles for establishing gene therapy in clinical practice are efficient in vivo gene transfer and the molecular knowledge of which genes are most usefully targeted in a given acquired disease. Many laboratories are trying to improve the former, and rapid progress is being made in the development of new gene transfer agents. The aim for respiratory diseases is to produce efficient targeted transfer of the gene or antisense oligonucleotide of choice into either the pulmonary epithelium or endothelium. If this can be achieved gene therapy for both inherited and acquired respiratory diseases is likely to be a realistic therapeutic option in the future.

Gene Therapy: The Market Scenario

The market for respiratory disorder treatments is continuing to experience rapid expansion as healthcare payers increasingly acknowledge the high treatment costs associated with chronic indications. A shift in developmental focus has become the driving force for market transformation, with research efforts now targeting the causative disease gene in an effort to prevent symptom presentation and pathological decline. In the short term, a number of impending product launches will satisfy unmet demand within high-value market niches by addressing issues of patient compliance and targeting patients at the more severe end of the disease spectrum. Many large biopharmaceutical companies such as Alnylam Pharmaceuticals, Applied Genetic Technologies Corporation (AGTC). etc. are operating in this domain. Challenges nevertheless abound in the respiratory disease market—not the least of which is the loss of patent protection and/or market exclusivity for many top-selling respiratory products. The constraining effects of these factors will be balanced against several companies’ robust respiratory drug-development pipelines, as well as licensing and acquisition activity that has been critical to the evolution of the respiratory drug market.

DelveInsight’s Gene Therapy Reports

DelveInsight’s Gene Therapy Reports cover the entire gene therapy market insights for respiratory 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.

DelveInsight’s Immunology based Gene Therapy Reports

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Gene Therapy: The “Hope” for Immunologic disorders!

About Immunologic disorders

Immunological disorders are a dysfunction of the immune system. These disorders cause abnormally low activity or overactivity of the immune system. In cases of immune system overactivity, the body attacks and damages its own tissues. These diseases decrease the body’s ability to fight invaders, causing vulnerability to infections. Most immunological diseases are caused by errors (mutations) in specific genes. It has long been the hope that one day it would be possible to cure these diseases by fixing the mutation that causes the disease and thus affect a cure. As a result of the human genome project and similar efforts to map all of the genes present in human beings, we now know the identities of the specific genes involved in many diseases, including the vast majority of immunological diseases. We have finally reached the stage where that long held hope is becoming a reality.

Gene Therapy: Role in Immunological disorders

In its broadest sense, gene therapy is the transfer of a gene, or genes to patients, for therapeutic purposes by using vectors as agents of gene transfer and gene expression. Traditional pharmacological approaches to treatment entail the synthesis of small, diffusable compounds given orally or by injection. These approaches have yet to provide ideal agents for use in immunological diseases. Recent research, however, has identified a number of proteins with the potential to improve treatment, but these are difficult to administer long term. Gene therapy provides the opportunity to deliver protein products, as well as therapeutic species of nucleic acids, such as antisense RNA, much more efficiently than traditional methods of drug therapy. Gene therapy’s greatest strength is its ability to produce high, sustained concentrations of therapeutic macromolecules within a defined anatomical location. Thus, making gene therapy an ideal candidate for treating immunological disorders.

Gene Therapy: The Market Scenario

Collectively, immunological disorders constitute a major, unmet, clinical challenge. Although no single disorder is highly prevalent, there are over 150 of them. Because these diseases are generally incurable and difficult to manage, there is a pressing need for novel approaches to their treatment. As a result the market opportunities for gene therapy in the field of immunological disorders have been reinvigorated by the entry of large biopharmaceutical companies such as Glaxosmithkline, Novartis. etc. which are operating in this domain. Many companies have started investing in and researching on gene therapy as it has the potential to transform many areas of immunological disorders, leading to more rapid development timelines and access to better treatment options within a shorter time frame. However, there are some risks that need to be overcome and safer vectors need to be developed. Various laboratories around the world are working at modifications of the viral vectors in order to improve their safety. Nevertheless, gene therapy must still be regarded as an experimental therapy. It is likely that the inherent problems will be worked out in the coming years and that a larger number of immunological diseases will be cured by gene therapy.

DelveInsight’s Gene Therapy Reports

DelveInsight’s Gene Therapy Reports cover the entire gene therapy market insights for immunological 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.

DelveInsight’s Cardiovascular disorders based Gene Therapy Reports

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Gene Therapy to “heal” Cardiovascular disorders!

Coronary artery disease, heart failure, and cardiac arrhythmias are major causes of morbidity and mortality in the developed countries. Pharmacologic drugs and device therapies have multiple limitations, and there exists an unmet need for improved clinical outcomes without side effects. These limitation in currently available therapies have prompted extensive investigation into new treatment modalities and gene therapy has emerged as a viable alternative. The goal of gene therapy is to modify a gene or genetic pathway to provide therapeutic value and prevent or reduce disease.

About Gene Therapy

Gene therapy involves the transfer of genetic information into a patient to correct a congenital or acquired disorder. Despite the advances in conventional pharmacology, implantable devices and surgery, cardiovascular disorders remain one of the leading causes of mortality in contemporary societies accounting for half a million deaths per year in the United States. The overall deaths due to cardiovascular diseases accounts for 1 of every 3 deaths in America. Cardiac diseases can affect every age group, but prevalence is seen in patients 65 or older. There is an ongoing need to explore novel and more promising approaches targeting their pathobiology. Over the past decades, advancements in somatic gene transfer delivery have transformed cardiovascular gene therapy from a futuristic fantasy to a scientific reality.

Gene Therapy: Role in Cardiovascular disorders

With the rapid development of economy, urbanization and changing lifestyles, the number of people with cardiovascular diseases have increased globally. Therefore, there is a strong impetus for more effective treatment and prevention. Gene therapy, which was initially envisioned as a treatment strategy for inherited monogenic disorders, has emerged with broad therapeutic potential, including treatment of acquired polygenic diseases such as peripheral vascular disease, ischemic heart disease, arrhythmias, and congestive heart failure. Sequencing information from the human genome and the development of gene transfer vectors and delivery systems have given researchers the tools to target specific genes and pathways that play a role in cardiovascular diseases. Thus, placing cardiovascular disorders within the reach of gene-based therapies. Current and possible candidates for gene therapy in the field of cardiovascular disorders include Critical Limb Ischemia, Coronary artery disease, Hypercholesterolemia and Congestive Heart Failure etc. However, efficient delivery to the target cells and sustained expression of transgenes are acting as limitations in this field. Inspite of this there is much optimism and confidence in the use gene therapy for treatment of cardiovascular 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 AnGes, uniQure biopharma, and Bio Sidus etc., are operating in the field of cardiovascular disorders in the gene therapy domain. A growing number of partnership between companies in drug development for example between The Medicines Company and Alnylam Pharmaceuticals etc., are driving the new gene therapy research. The industry’s collective pipeline is brimming with 300+ therapies for various therapeutic areas. The global market opportunities for gene therapy in the field of cardiovascular disorders have risen drastically due to improvement in our understanding of the molecular mechanisms associated with heart failure and the development of vectors with cardiotropic properties. Gene therapy is now being considered as a viable adjunctive treatment to mechanical and pharmacological therapies for various cardiovascular disorders.

DelveInsight’s Gene Therapy Reports

DelveInsight’s Gene Therapy Reports cover the entire gene therapy market insights for cardiovascular 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.

DelveInsight’s Musculoskeletal disorders based Gene Therapy Reports

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Gene Therapy at the “Forefront” for Musculoskeletal disorders!

About Musculoskeletal disorders

Musculoskeletal disorders (MSDs) are injuries or pain in the body’s joints, ligaments, muscles, nerves, tendons, and structures that support limbs, neck and back. MSDs are degenerative diseases and inflammatory conditions that cause pain and impair normal activities. They can affect many different parts of the body including upper and lower back, neck, shoulders and extremities (arms, legs, feet, and hands).

Musculoskeletal disorders are the leading cause of disability in the United States and account for more than one-half of all chronic conditions in people over 50 years of age in developed countries with one in every two adults reporting a musculoskeletal condition requiring medical attention. The economic impact of these conditions is also staggering costing billion dollars annually comprising of direct expenditures in health care costs and the indirect expenditures in lost wages. However, the rapid progress in our understanding of some of the molecular mechanisms involved in the bone regeneration has revolutionized the clinical management of many bone and musculoskeletal disorders.

Gene Therapy: Role in Musculoskeletal disorders

Gene therapy has made remarkable progress in the field of musculoskeletal system. Since the introduction of this concept much of the preclinical and clinical data have emerged. The most promising area of research in this field is the tissue repair, because it doesn’t require prolonged period of gene expression, local delivery is reasonably simple and it avoids substantial risk associated with systemic delivery, and levels of gene expression don’t need to be so finely regulated. Gene transfer is successfully being used to aid the repair and regeneration of bone, cartilage, ligament tendon, meniscus and intervertebral disc. Other potential applications of gene therapy in musculoskeletal system include osteoporosis, aseptic loosening, genetic diseases and tumors. Highly encouraging data gained from these studies have confirmed that gene therapy is a promising therapeutic solution to treat various musculoskeletal system disorders.

Gene Therapy: The Market Scenario

A new biologic era of orthopaedic surgery has been initiated by basic scientific advances that have resulted in the development of gene therapy approaches for treating musculoskeletal disorders. This is evident from the large no. of companies such as Avexis, Sylentis S.A. etc. which are operating in the field of musculoskeletal disorders in the gene therapy domain. Many companies have started investing in and researching on gene therapy as it has the potential to transform many areas of musculoskeletal care, leading to treatments that are less invasive, more effective, and less expensive than existing modalities. Moreover, extensive laboratory investigations and preliminary clinical investigations have established the feasibility and promise of gene therapy.

DelveInsight’s Gene Therapy Reports

DelveInsight’s Gene Therapy Reports cover the entire gene therapy market insights for musculoskeletal 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.

DelveInsight’s Metabolic disorders based Gene Therapy Reports

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Gene Therapy to “reverse” Metabolic disorders!

Metabolic disorders are collectively common, frequently severe and in many instances difficult or impossible to treat. Accordingly, there is a compelling need to explore novel therapeutic modalities, including gene therapy, and examine multiple phenotypes where the risks of experimental therapy are outweighed by potential benefits to trial participants.

About Gene Therapy

Gene therapy is defined as the treatment or cure of human diseases by transfer of nucleic acids (DNA or RNA) to affected cells and it covers a broad range of therapeutic applications from Mendelian disorders to complex metabolic diseases. Most diseases caused by genetic deficiencies could in theory, be treated by the introduction and expression of a normal gene into an appropriate target tissue. It seems like that gene therapy strategies for most metabolic disorders will not require strict gene regulation, as a fraction of the normal levels of gene activity could result in amelioration or significant improvement in the clinical outcome.

Metabolic disorders refer to different types of medical conditions caused by genetic defects — most commonly inherited from both parents — that interfere with the body’s metabolism. Metabolism is the complex set of chemical reactions that your body uses to maintain life, including energy production. When these chemical processes don’t work properly due to a hormone or enzyme deficiency, a metabolic disorder occurs. Inherited metabolic disorders fall into different categories, depending on the specific substance and whether it builds up in harmful amounts (because it can’t be broken down), it’s too low, or it’s missing. Metabolic disorders affects millions of people world-wide and is the fourth or fifth leading cause of death in the developed world. Gene therapy is an experimental form of treatment that aims to use the transcriptional machinery of the patient to produce the active factor that exerts the intended therapeutic effect, ideally in a permanent, tissue-specific and manageable way.

Gene Therapy: Role in Metabolic disorders

Genetic manipulations for altering gene expression pattern in vivo with therapeutic outcome, known as gene therapy, offers a fundamentally different approach to pharmacotherapy for inherited as well as acquired metabolic disorders. Current and possible candidates for gene therapy in the field of metabolic disorders include Sanfilippo type A syndrome, Metachromatic Leukodystrophy (MLD), Crigler-Najjar syndrome, Urea cycle disorders and Diabetic Macular Edema etc. because of their poor prognosis.

Significant progress has been made in the pre-clinical arena and achievement of efficacy in different animal models has been reported using multiple gene transfer technologies. However, interspecies differences in disease biology, vector performance, host–vector interactions and increased size with the associated necessity to functionally correct a greater number of cells to reach the threshold required for therapeutic benefit are acting as biological limitations in this field. Despite these challenges, it is predictable that the gene therapy based strategy in modulating metabolism and treating metabolic disorders will surely impact how we live a healthy life 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, Pfizer, and Alnylam etc., are operating in the field of metabolic disorders in the gene therapy domain. A growing number of partnership between companies in drug development for example between Lysogene and RegenX Biosciences etc., are driving the new gene therapy research. The industry’s collective pipeline is brimming with 300+ therapies for various therapeutic areas. Gene therapy has the potential of providing a definitive cure for patients with these diseases. The global market opportunities for gene therapy have risen drastically indicating that there’s big money in gene therapy.

DelveInsight’s Gene Therapy Reports

DelveInsight’s Gene Therapy Reports cover the entire gene therapy market insights for metabolic 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.