Gene Therapy Products

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.

Suicide Gene Therapy: Where do we stand?

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Suicide Gene Therapy: The Genetic “Kill Switch”

What it is?

Suicide gene therapy is based on the introduction of a viral or a bacterial gene into tumor cells, which allows the conversion of a non-toxic compound into a lethal drug.

Basically suicide gene therapy also known as Gene Directed Enzyme/Prodrug therapy (GDEPT) or as Gene Prodrug Activation Therapy (GPAT) uses viral vectors to deliver suicide genes into tumor cells which possess the enzyme that converts prodrug to active metabolites, it increases the toxicity level several fold inside the tumor whereas the vast majority of the host cells are unaffected. Thus it accomplishes the same end goal of chemotherapy treatments, but by different means. While chemotherapy targets all of the body’s rapidly dividing cells with the intention of killing cancer cells, suicide gene therapy delivers a cancer-killing drug solely to tumors. This bypasses chemotherapy side effects like hair loss and nausea, which are caused by collateral damage to non-cancerous cells.

How it works?

There are several suicide gene therapies. Among them Herpes Simplex Virus thymidine kinase (HSV-tk) and Cytodine Deaminase (CD) are important.  The Herpes Simplex Virus deposits a gene into the cancer cells that causes them to produce a special enzyme. Once the virus triggers production of the enzyme, doctors initiate step two by injecting the patient with a unique type of chemotherapy drug called a prodrug. Suicide gene therapy typically uses ganciclovir (GCV) and its nucleoside analogs (acyclovir etc). When administered, GCV and other prodrugs are nontoxic, and thus cause no harm to healthy cells.

But when GCV comes in contact with the special enzyme, the prodrug turns highly toxic. This starts a natural biological process called programmed cell death, which causes cells to commit suicide. Because HSV-tk and CD are not present in any of the body’s healthy cells, the prodrug only destroys cancer cells that were genetically altered by the virus.

Although only a limited number of tumor cells will take in HSV-tk or CD from the virus, the activated prodrug is passed on to neighboring cells through what doctors call the bystander effect. Further, cells that destroy themselves as a response to treatment attract immune cells that clear the tumor site of dead and dying cancer cells.

Limitations of (HSV-tk)-GCV system

This HSV-tk system suffers from limitations which include (i) the potential for production of inactive catalytic molecules due to the utilization of alternative splicing sites, (ii) the potential immunogenicity of the viral enzyme, (iii) the potential need to administer GCV to control cytomegalovirus infections (a complication often encountered in allo-HSCT) and thus cause unintended elimination of HSV/Tk-engineered cells, and (iv) the requirement for active cell division in order to mediate cell death, which takes time and renders the system less effective for use in post mitotic cells. Moreover, major improvements are needed in vector design to enhance targeting and delivery of suicide genes.

Live and Let Die: A New Suicide Gene Therapy

Despite of the limitations associated with HSV/tk-GCV system suicide gene therapy holds enormous potential in the eye of the researchers which is evident from the research done on combining suicide gene therapy with other treatments, improving the bystander effect and finding the optimal method for delivering GCV and the HSV-tk gene. One such example is iCasp9 (a late executor of the intrinsic pathway of apoptosis, leading to DNA fragmentation and rapid cell death) which represents more than a simple upgrade of the HSV/Tk-GCV system as it is not dependent on DNA synthesis as is HSV/Tk-GCV, allowing application in non-replicating cells. It involves dimerization of the subunits which is induced by addition of a biologically inert small molecule (AP1903) that has been shown in clinical studies to be well tolerated. Dimerization of iCasp9 activates one of the last steps in the apoptotic cascade, resulting in rapid cell death—as soon as 30 minutes after administration of the activator.

The field of suicide gene therapy is rapidly maturing and will no doubt be part of the future of cancer therapeutics. In addition, combination of Gene & Cell therapy approaches like “chimeric antigen receptors” or CARs for short also hold great promise for increasing the effectiveness of current chemotherapeutic treatment regimens.

For more information 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.

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 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.