DelveInsight’s Gene Therapy Reports

What are your views on Gene Therapy?

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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 that Watson is talking about is contained in our genes, and deals with a new technique, GENE THERAPY.

gdfgGene therapy is an innovative way to fix genetic problem from the 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.

The treatment sounds magical, but the complexity of designing and delivering arises. The safety issues slowed the progress, following events like death of Jess Gelsinger, who died in 1999 just weeks after receiving an experimental gene therapy in a clinical trial being conducted by the University of Pennsylvania.

Although gene therapy is a promising treatment option for a number of diseases, the technique remains risky and is still under study to make sure that it will be safe and effective.

Also some genetic disorders are rare in nature affecting 1 in million. For such patients, Gene therapy can be a miracle but high cost of developing a treatment can make it unappealing for the pharmaceutical companies. With a limited number of patients to recover those expenses from, developers may never earn money from treating such rare genetic disorders. And some patients may never be able to afford them.

The first milestone in gene therapy, Gendicine, developed by Shenzhen SiBiono GeneTech was approved in China in 2003 for the treatment of head and neck cancers and the biggest watershed moment was Glybera, a gene therapy for lipoprotein lipase deficiency, launched in Germany at a price of more than 1 million euros ($1.1 million).

Meanwhile, companies like bluebird bio and Spark Therapeutics generated impressive clinical data with programmes of their own.

The success has generated renewed interest from both private and public equity investors as well as pharmaceutical companies, such as Bayer and Sanofi, which have willingly provided much-needed resources to companies working in the field in the form of financing and partnerships, helping to drive what could turn out to be a virtuous circle of success.

DelveInsight Gene Therapy Reports

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.

By Rashi Aggarwal, Associate Analyst, DelveInsight

Please give your views on Gene Therapy and lets discuss about its potential in the market.

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Gene Therapy to “Assassinate” HIV virus

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Transforming Cells into Molecular Assassins

The conventional approach to vaccination involves training the immune system to fight an infection. But the Researchers at Scripps Research Institute in California have derived a new technique that involves altering the DNA in order to give cells anti-HIV capabilities. A discovery that potentially could lead to a new therapy for patients as well as an alternative to a vaccine.

Problem with HIV

Usually when a cell has been infected by a virus, enzymes chop up the viral proteins into short peptides which combine with HLA proteins in Endoplasmic reticulum. These are then transferred to the surface of the infected cell, labelling the cell as threat but HIV being a highly variable virus has found a way to fool our Immune system by interfering with the binding process. The virus accumulates mutations in it viral proteins thus rendering them unable to bind to the HLA proteins or resulting in very weak binding. Hence effectively putting the T-cells out of action. Moreover HIV fuses with the cell and inserts its own genetic material i.e. a single-stranded RNA and transforms the host cell into a HIV manufacturing site.

Scripp’s Radical Approach

Researchers at Scripps Research Institute in California have successfully altered the DNA of monkeys to give their cells HIV-fighting properties. The technique involved using engineered Adeno-associated virus to introduce a new section of DNA inside healthy muscle cells. Once injected into muscle tissue, this strip of DNA codes for eCD4-IG protein that blocks the points where the HIV virus binds to both cellular receptors viz, CCR5 and CD4 leaving no point of entry. Thus enabling the cells to neutralize and provide sustained vaccine-like protection against HIV infection.

Pre-Clinical Study

The research team led by Dr. Farzan including scientists from more than a dozen research institutions and both campuses of The Scripps Research Institute (TSRI) conducted their pre-clinical studies on four rhesus monkeys. In their study, monkeys were infected with a hybrid version of HIV, administering up to four times the amount of virus it took to infect a control group. They found that the protein protected the monkeys from every strain of HIV-1, HIV-2 and SIV (simian immunodeficiency virus) that has been isolated from humans or rhesus macaques, including the hardest-to-stop variants and it does so for at least eight months after injection.

The study builds upon previous discoveries by the Farzan laboratory, showing that proteins based on HIV-binding region of CCR5 can be used to prevent infection. Keeping this in mind Farzan and his team developed the new drug candidate as a direct mimic of the receptors by fusing together elements of both CCR5 & CD4 receptors to which HIV binds. Thus preventing the infection by excluding the avenues through which the virus tends to escape.

Important Step

This therapy effectively eliminates the need for presentation of threat or viral peptides on the surface of cells by turning cells into factories that constantly spew out the artificial HIV-killers. Moreover it targets those areas of HIV which the virus struggles to change thus overcoming its rapidly mutating nature. Also the therapy in itself can prove as an alternative to antiretroviral drugs that are used for HIV treatment in terms of cost, side-effects as well as the difficult life-long regimen which the patients have to follow.

Although the long-term effects of this therapy are still unknown it is believed to have the potential to put HIV into sustained remission in chronically infected people. Nonetheless, the team plans to begin trials in patients who have HIV but are unable to take conventional drug therapies within the next year. The team also aims to test its efficacy as a vaccine, in people who don’t have the virus but are at high risk of infection, said Dr. Farzan.

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

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.

A CRISPR-Cas9 approach for Gene Therapy: The Hot New R&D Field!

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Only a hundred and fifty years have passed since Gregor Mendel’s discovery of simple Mendelian inheritance. In a remarkably short amount of time humans have achieved such impressive feats as sequencing the entire human genome and gaining understanding of the causes of most genetic disease. Now that researchers have all this information at hand, the focus has shifted to the design of reagents that can target specific genomic sequences. The rapid advancement of genome-editing techniques holds much promise for the field of human gene therapy. From bacteria to model organisms and human cells, genome editing tools such as zinc-finger nucleases (ZNFs), TALENs, and CRISPR/Cas9 have been successfully used to manipulate the respective genomes with unprecedented precision. With regard to human gene therapy, it is of great interest to test the feasibility of genome engineering because of their ease of customization and high-efficient site-specific cleavages that could potentially be used to treat a variety of human genetic disorders such as hemoglobinopathies, primary immunodeficiencies, and cancer.

Unraveling the potential of CRISPR-Cas9 for gene therapy

The molecular machinery from the prokaryotic clustered regularly interspaced short palindromic repeats (CRISPR)-Cas immune system has broadly been repurposed for genome editing in eukaryotes. In particular, the sequence-specific Cas9 endonuclease can be flexibly harnessed for the genesis of precise double-stranded DNA breaks, using single guide RNAs that are readily programmable. The endogenous DNA repair machinery subsequently generates genome modifications, either by random insertion or deletions using non-homologous end joining (NHEJ), or designed integration of mutations or genetic material using homology-directed repair (HDR) templates. This technology has opened new avenues for the investigation of genetic diseases in general, and for gene therapy applications in particular.

Patent Litigation over control of the revolutionary CRISPR-Cas9 tech

Despite the predicted utility of a successful gene editing technique, many current methods like Zinc Fingers Nucleases and TALENs have confounding issues like low efficiency, time-consuming procedures, and lack of specificity for both model organisms and humans. In the past several years, a new gene editing system viz, CRISPR-Cas9 derived from bacteria, has arisen as a frontrunner for efficient and successful gene editing.

Research in the area of CRISPR/Cas9 is gaining speed and this system could very well be the solution to many medical issues we face today. For evidence of CRISPR/Cas9’s promise, look no further than its attendant battle over intellectual property. Novartis and Atlas Venture joined together to form Editas Medicine, but a breakup of co-founders led Berkeley’s Jennifer Doudna to take her IP to the competing Intellia Therapeutics, while Swiss rival CRISPR Therapeutics has conflicting claims of its own backed by Versant. And now a team at Johns Hopkins has done some experiments to demonstrate its promise in engineering human stem cell therapies.

This proves that gene editing has staggering potential and that it can be developed as a naturalistic method of correcting defective genes by getting at the underlying causes of a broad range of diseases.

Gene Therapy’s fruition?

The world of gene therapy in which single-dose treatments correct debilitating defects enjoyed something of a renaissance in 2014. Strong clinical results from leaders in the once-maligned field spurred renewed optimism, helping a new generation of startups secure millions in venture financing to develop their next-generation approaches to the field. And that led to something of a trickle-up phenomenon in the industry, as the innovations of biotechs and academics convinced the world’s biggest players to give this field a second look. Now Bayer, Pfizer, Biogen Idec and Astellas are among the many companies toiling in gene therapy, joining high-profile biotechs like bluebird bio and uniQure.

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.

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.

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.

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.