Gene Therapy

A few years ago, a clinical trial began in France in the hope of curing children with a type of genetic immune deficiency called SCID-X1. Children with this disease have a defective gene, called gamma-c, which prevents a subset of the cells of the immune system from forming, and predisposes the children to life-threatening infections. In an attempt to cure the childrenwho would otherwise die at a young agephysicians used gene therapy to provide them with normal gamma-c genes.

This particular trial has had striking success as well as tragedy. Eight of the eleven children are currently thriving. However, in two cases the therapy successfully introduced gamma-c genes, but these children have since developed leukemia. In both children, a gamma-c gene inserted next to another gene, called LMO2. The LMO2 gene has previously been linked to leukemia, and scientists speculate that the insertion of the gamma-c gene next to LMO2 may have overstimulated the gene, causing T cells to proliferate in excess. An LMO2 effect, in combination with the proliferation-inducing effects of the gamma-c gene itself, may be the cause of the leukemia in these two patients. Scientists are still investigating other possible causes.

From this single trial, it is clear that gene therapy holds significant promise, yet it is also clear that it poses significant risks. To learn more about the application of gene therapy in SCID, view the accompanying animation.

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Gene Therapy - Sumanas, Inc.

Overview

Gene therapy involves altering the genes inside your body's cells in an effort to treat or stop disease.

Genes contain your DNA the code that controls much of your body's form and function, from making you grow taller to regulating your body systems. Genes that don't work properly can cause disease.

Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body's ability to fight disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.

Researchers are still studying how and when to use gene therapy. Currently, in the United States, gene therapy is available only as part of a clinical trial.

Gene therapy is used to correct defective genes in order to cure a disease or help your body better fight disease.

Researchers are investigating several ways to do this, including:

Gene therapy has some potential risks. A gene can't easily be inserted directly into your cells. Rather, it usually has to be delivered using a carrier, called a vector.

The most common gene therapy vectors are viruses because they can recognize certain cells and carry genetic material into the cells' genes. Researchers remove the original disease-causing genes from the viruses, replacing them with the genes needed to stop disease.

This technique presents the following risks:

The gene therapy clinical trials underway in the U.S. are closely monitored by the Food and Drug Administration and the National Institutes of Health to ensure that patient safety issues are a top priority during research.

Currently, the only way for you to receive gene therapy is to participate in a clinical trial. Clinical trials are research studies that help doctors determine whether a gene therapy approach is safe for people. They also help doctors understand the effects of gene therapy on the body.

Your specific procedure will depend on the disease you have and the type of gene therapy being used.

For example, in one type of gene therapy:

Viruses aren't the only vectors that can be used to carry altered genes into your body's cells. Other vectors being studied in clinical trials include:

The possibilities of gene therapy hold much promise. Clinical trials of gene therapy in people have shown some success in treating certain diseases, such as:

But several significant barriers stand in the way of gene therapy becoming a reliable form of treatment, including:

Gene therapy continues to be a very important and active area of research aimed at developing new, effective treatments for a variety of diseases.

Explore Mayo Clinic studies testing new treatments, interventions and tests as a means to prevent, detect, treat or manage this disease.

Dec. 29, 2017

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Gene therapy - About - Mayo Clinic

Gene therapy is a revolutionary approach to treating genetic diseases. It involves a one-time delivery of a normal copy of the defective gene which ideally causes the body to naturally produce the missing enzyme. It is the most promising cure that these boys desperately need.

Gene therapy has shown promise as a cure for Hunter Syndrome.

A clinical trial is required to get FDA approval.

Funding is the final barrier to beginning the clinical trial.

In 2013, after it had been performed on other forms of MPS with positive results, a group of Hunter Syndrome families initiated a partnership with researchers at Nationwide Childrens Hospital in Columbus, Ohio to begin gene therapy research in Hunter Syndrome / MPS II. Now, the researchers are in the final stages of developing the first gene therapy clinical trial for boys with Hunter Syndrome. The only obstacle is the substantial funding required for such a trial. If the necessary funds are raised, the trial could begin as early as late 2017 or early 2018.

In the world of medicine, this treatment is considered an orphan drug because of the small patient population it seeks to treat. In the US, an orphan drug is a treatment for a disease that affects less than 200,000 people. Drug companies fund much of the research for more common diseases because in developing a potential blockbuster drug for a large patient population, they also are more likely to make a profit. Few companies are willing to invest in research for such a small population only an estimated 500 boys with Hunter Syndrome live in the United States. Without drug companies pursuing a cure for Hunter Syndrome, families tackled that lofty goal and have finally reached the point where a potential cure is ready and waiting with one obstacle: funding it.

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Project Alive | The hope for a cure: Gene therapy

Editor-in-Chief: Terence R. Flotte, MDDeputy Editors, Europe: Nathalie Cartier, MD and Thierry VandenDriessche, PhDDeputy Editors, U.S.: Barry J. Byrne, MD, PhD and Mark A. Kay, MD, PhDHuman Gene Therapy Editor: Guangping Gao, PhDMethods Editor: Hildegard Bning, PhDClinical Development Editor: James M. Wilson, MD, PhD

Latest Impact Factor* is 4.187 *2016 Journal Citation Reports (Clarivate Analytics, 2017)

Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases.

The Journal is divided into three parts. Human Gene Therapy, the flagship, is published 12 times per year. HGT Methods, a bimonthly journal, focuses on the applications of gene therapy to product testing and development. HGT Clinical Development, a quarterly journal, serves as a venue for publishing data relevant to the regulatory review and commercial development of cell and gene therapy products.

Human Gene Therapy was voted one of the most influential journals in Biology and Medicine over the last 100 years by the Biomedical & Life Sciences Division of the Special Libraries Association.

Human Gene Therapy, HGT Methods, and HGT Clinical Development are under the editorial leadership of Editor-in-Chief Terence R. Flotte, MD, University of Massachusetts Medical School; Deput Editors Europe Nathalie Cartier, MD, INSERM, andThierry VandenDriessche, PhD, Free University of Brussels (VUB); Deputy Editors U.S. Barry J. Byrne, MD, PhD,Powell Gene Therapy Center, University of Florida, College of Medicine and Mark A. Kay, MD, PhD, Stanford University School of Medicine; Human Gene Therapy Editor Guangping Gao, PhD, University of Massachusetts Medical School; Methods Editor Hildegard Bning, PhD, University of Cologne; Clinical Development Editor James M. Wilson, MD, PhD,University of Pennsylvania School of Medicine, Gene Therapy Program; and other leading investigators. View the entire editorial board.

Audience: Geneticists, medical geneticists, molecular biologists, virologists, experimental researchers, and experimental medicine specialists, among others.

Human Gene Therapy and HGT Methods provide Instant Online publication 72 hours after acceptance

Indexed/Abstracted in:MEDLINE;PubMed;PubMed Central;Current Contents/Life Sciences;Science Citation Index Expanded;Science Citation Index;Biotechnology Citation Index;Biological Abstracts;BIOSIS Previews;Journal Citation Reports/Science Edition;Derwent Drug File;EMBASE/Excerpta Medica;EMBiology;Scopus;Chemical Abstracts;ProQuest databases

The views, opinions, findings, conclusions and recommendations set forth in any Journal article are solely those of the authors of those articles and do not necessarily reflect the views, policy or position of the Journal, its Publisher, its editorial staff or any affiliated Societies and should not be attributed to any of them.

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Human Gene Therapy

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Video:ACGT fellow, Dr. Samuel Katz,Yale Medical Center, speak about cell and gene therapies for cancer treatment.

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The mission of ACGT is to seek out and support revolutionary scientific research into the causes, treatment and prevention of all types of cancer using cells and genes as medicine.

Cell and gene therapies are destroying tumors every day in the laboratory and in human trials, targeting only the cancer with few side effects to healthy tissue. ACGTs rigorous grant review process, under the guidance of a preeminent Scientific Advisory Council, have resulted in some of the most successful breakthroughs in the history of cancer treatments and led to many patients in complete remission.

100% of our funding supports innovation and translational science that is proving again and again to offer better treatment options than the traditional therapies of chemotherapy, surgery and radiation. Every donation brings us closer to saving lives and changing the way we treat cancer forever.

Video:ACGT fellow, Dr. Samuel Katz,Yale Medical Center, speak about cell and gene therapies for cancer treatment.

See all ACGT videos here

At the ACGT anniversary gala in April, Robert Vonderheide, MD, DPhil, the Hanna Wise Professor in Cancer Research and newly appointed Director of the Abramson Read More

This fourth in our blog series on the progress of cell and gene therapy for cancer focuses on a treatment that has made huge strides Read More

In our last blog, we described the progress and promise of CAR-T, an immunotherapy that has been particularly successful with leukemia and lymphoma. As a Read More

2017 All rights reserved by ACGT. ACGT is a 501(c)(3) organization and all contributions are tax-deductible under the law of your state.

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Alliance for Cancer Gene Therapy (ACGT) Foundation

Krystal Biotech has filed to raise about $35 million in a Nasdaq IPO. The money will equip Krystals ex-Intrexon executive team to file an IND for its rare skin disease gene therapy in the first quarter of next year.

That gene therapy, KB103, is designed to treat dystrophic epidermolysis bullosa, a rare disease that makes the skin fragile and susceptible to blisters and tears. Krystal thinks it can improve outcomes in these patients, who currently only have palliative options, by using a HSV-1-based vector to fix defects in the COL7A1 gene.

Pittsburgh, Pennsylvania-based Krystal has yet to back up the idea with clinical data. But having run preclinical tests of topical and intradermal formulations of KB103 and met with the FDA late last year, it plans to embark on a whistle-stop development program. The plan is to file an IND in the first quarter, have results by the middle of the year and use that readout to guide the design of a pivotal trial.

Krystal will need more than the forecast IPO haul to execute that strategy, although it does expect to have some money left over from the Nasdaq listing to fund work on its follow-up program, KB104. That gene therapy treatment for Netherton syndrome is due to reach the IND stage by the second half of next year.

The early-stage nature of Krystals pipeline is one of several characteristics that mark it out as a biotech trying to make the leap to public markets early in its development as a company. Notably, given the financial reporting requirements for public companies, Krystal has yet to hire a full-time CFO or in-house principal accounting officer. The biotechs seven-person team is heavily skewed towardR&D.

Krish Krishnan, CEO, and Suma Krishnan, COO, lead this small team. The pair have worked together for most of the past 13 years, starting at New River Pharmaceuticals in the run-up to its takeover by Shire. Both Krishnans popped up next at Pinnacle Pharmaceuticals, where Krish was CEO, before reuniting with their former New River boss Randal Kirk at Intrexon.

Krystal sees the pair strike out on their ownand stand to win big if their gene therapies succeed.A family trust owned by Krish and Suma owns more than two-thirds of Krystals stock. The only other organization to own more than 5% of the company is Sun Pharma, which bought its 16.5% stake in a $7 million series A round earlier this month. That round bolstered Krystals cash position, which stood at $3.5 million at the end of June.

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Krystal files for IPO to move gene therapies into clinic | FierceBiotech - FierceBiotech

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apic Bio, Inc., a pre-clinical stage gene therapy company leveraging its proprietary platform to advance therapies to treat rare diseases with complex mechanisms, in particular Alpha-1 Antitrypsin Deficiency (Alpha 1), launched today with an initial investment led by the venture philanthropy arm of the Alpha-1 Foundation and a private investor with the disease.

Its lead product, APB-101, targets the liver via an AAV delivered Dual Function Vector (df-AAV) whereby the Z-AAT protein is silenced and M-AAT protein is augmented. APB-101 has achieved a pre-clinical proof of concept with efficacy demonstrated in vitro and in vivo. It is currently undergoing pre-clinical GLP toxicology studies in non-human primates. Patients living with Alpha 1 lack sufficient levels of circulating AAT protein to protect lung tissue against damage from proteases, and experience the accumulation of mutant AAT polymers in the liver. Clinically, the deficiency is manifested by progressive emphysema and the accumulation presents a significant risk of liver cirrhosis.

John Reilly, Co-Founder & President said: We are grateful to TAP and A1AT Investors, LLC who have supported the successful start of Apic Bio by providing the first tranche of our seed financing round allowing us to secure key intellectual property rights and operational support. With such strong support from the advocacy and patient community, we are confident that we will identify the right corporate partners to help us achieve our business development goals and bring this exciting new therapy to patients.

The df-AAV platform allows treatment of other diseases with complex mechanisms where the mutant gene product must be reduced and the normal gene product must be augmented.

Dr. Chris Mueller, Co-founder and Chief Scientific Officer of Apic Bio said: We are encouraged by the feedback that we have received during our pre-IND meeting with the FDA that there is a clear path for us to conduct a first-in-human Phase 1/2 clinical study. Furthermore, we are very much looking forward to demonstrating the benefit of APB-101 to patients that have been living with alpha-1 and have had very little hope for a cure. Our data suggests this is a liver sparing approach for gene augmentation which may exceed the therapeutic and safety margins when compared to a strict gene augmentation without gene silencing that may exacerbate the underlying liver disease.

TAP is very pleased to provide this funding to Apic Bio. Their cutting-edge work on a therapy that addresses both the liver and lung disease brings us closer to finding a cure for Alpha-1 Antitrypsin Deficiency, thus fulfilling our mission, said Jean-Marc Quach, CEO for The Alpha-1 Project.

Todays launch of Apic Bio has been a long time coming for the hundreds of thousands of people who are challenged by Alpha 1, said Ed Krapels, who has been living with Alpha 1 and is the new companys first individual investor. Now that we are moving forward, we hope to work with patients, their advocates and researchers to make a cure readily available. Krapels added.

About Apic Bio: Apic Bio, Inc. is a spin-off from the University of Massachusetts Medical School (UMMS) and is based upon nearly 30 years of gene therapy research by its scientific founders Christian Mueller, PhD, Associate Professor of Pediatrics and a member of the Horae Gene Therapy Center at the University of Massachusetts Medical School, Terence R. Flotte, MD, the Celia and Isaac Haidak Professor in Medical Education, dean of the School of Medicine and provost and executive deputy chancellor of the University of Massachusetts Medical School; and colleagues at the Horae Gene Therapy Center. Their research is funded in part by an $11M grant from the National Heart, Lung, and Blood Institute (NHLBI).

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Apic Bio Launches to Advance First-in-Class Gene Therapy for ... - Business Wire (press release)

A darling British biotech that is working on the development of the worlds leading gene therapy for cancer has posted reduced losses and a leap in sales.

Oxford BioMedica signed a deal worth up to $100m (77m) with Swiss drugs giant Novartis last month to supply cell material for its potential blockbuster treatment, known as CTL019, for a type of leukaemia.

The living drug was recommended for approval by US regulators in June, with a final determination expected this autumn.

It would be the first gene therapy for cancer and if approved is expected to generate a spike in revenues for Oxford BioMedica, with the potential for further tie-ups on other cancer treatments.

Oxford BioMedica is also trialling its lentiviral vectors, which help manipulate genes, in potential treatments for Parkinsons Disease and an eye condition.

In half-year results today, Oxford BioMedica pared back losses to 2.2m, compared to 6.9m in the red the previous year. Revenues jumped 26pc to 15.7m.

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Gene therapy darling Oxford BioMedica pares losses - Telegraph.co.uk

The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has granted a Promising Innovative

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MHRA backs gene therapy in battle with 'bubble baby' disease - The Pharma Letter (registration)

Three patients who received SPK-8011 a gene therapy developed by Spark Therapeutics for hemophilia A show increased production of clotting factor VIII without any safety issues reported, says the company.

The patients are the first to receive the treatment as part of a Phase 1/2 clinical trial, with early data showing they have not yet experienced spontaneous bleeding episodes.

The encouraging start of our SPK-8011 clinical trial reinforces the strength of our gene therapy platform, delivers human proof-of-concept in a second liver-mediated disease a significant achievement in the gene therapy field and positions us well to potentially transform the current treatment approach for this life-altering disease with a one-time intervention, Katherine A. High, president and CEO of Spark Therapeutics, said inreleasingsecond-quarter 2017 financial results.

The study (NCT03003533) examines escalating doses of the treatment a one time infusion of a non-infectious virus that delivers a functional copy of the gene encoding clotting factor VIII.

The first two patients received the lowest dose and have now been followed for 23 weeks and 12 weeks. Measurements show their levels of the clotting factor steadily rising, reaching a stable level of 11 and 14 percent of normal values. Withthose results in hand, researchers decided to go for a higher dose in the third patient. So far, the increase of factor VIII in that third patient is higher than that observed in the other two, corresponding to the increased dose.

Researchers have not detected immune reactions to the therapy in any of the patients. This is important, as antibodies to SPK-8011 ould potentially render it ineffective.No serious adverse events have been reported and none of the patients required treatment with corticosteroids.

Spark, based in Philadelphia, has also reported on its second gene therapy, SPK-9001, now being developed to treat hemophilia B.

Researchers presented data from the Phase 1/2 study (NCT02484092) at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress, showing that the 10 participants had lowered their annual bleeding rate by 96 percent. They also lowered their annualized infusion rate by 99 percent.

In addition, their levels of clotting factor IX, 12 weeks after the treatment, had stabilized at 33 percent. Four of the patients have been followed for more than a year after the treatment, and one for as long as 18 months.

None have developed immune reactions to the treatment and no bleeding episodes have been recorded. One patient with severe joint disease had precautionary factor infusions for persistent knee pain.

We are excited about the progress we are making to achieve our goals of our investigational hemophilia A and B programs: to safely achieve predictable, consistent and sustained activity levels that prevent spontaneous bleeding, said High.

Read the rest here:

Spark's Gene Therapy SPK-8011 to Treat Hemophilia A and B Advance in Clinical Trials - Hemophilia News Today

Uniqure, the company best known for having the first approved gene therapy put on the market (for familial chylomicronemia syndrome), is expanding its pipeline to include a gene therapy for Huntingtons disease. While still in early development, the company stated they plan to file an Investigational New Drug (IND) application next year with the US Food and Drug Administration (FDA) in order to begin clinical studies in humans. Huntington's disease is a genetic neurodegenerative disorder that leads to loss of muscle coordination, behavioral abnormalities and cognitive decline when a person enters their 3rd or 4th decade. The disease is an autosomal dominant mutation, meaning that if a person has the condition, there is a 50% chance their offspring will have it as well, and is due to a mutation on the huntingin gene. Despite good understanding of the condition, current treatments can only alleviate the symptoms of it, but they do not delay the onset or slow the progression of the disease. It is hopeful that gene therapy can do that. In Huntingtons disease animal models, the gene therapy called AMT-130, an AAV5 vector carrying a DNA cassette encoding artificial micro-RNA, is effective. In April, data was presented at the 12th Annual CHDI HD Therapeutics Conference in Malta showing the drug was able to silence the human mutant huntingtin gene in pig model. In the animal study, AMT-130 was administered into the striatum and thalamus of minipigs that had the mutant Huntingtin gene. Three months after treatment, the vector was observed throughout the minipig brain and expression of mutant HTT mRNA was significantly reduced in the striatum and thalamus by 50% to 80% and reductions were also observed in the cortex (reduced up to 40% compared to controls). Lead author of that study, Prof. Jan Motlik, Director of the Institute of Animal Physiology and Genetics in the Czech Republic said, "This study demonstrated that a single administration of AAV5-miHTT resulted in significant reductions in HTT mRNA in all regions of the brain transduced by AMT-130, as well as in the cortex. Consistent with the reduction in HTT mRNA, we also observed a clear dose-dependent reduction in mutant huntingtin protein levels in the brain, with similar trends in the cerebral spinal fluid. Taking into account the similarities of CHDI's proprietary transgenic pig model to the human brain, these results provide additional data to support moving forward with clinical trials of uniQure's promising gene therapy for Huntington's disease." For more news and information about orphan drugs in development, followRare Disease ReportonFacebookandTwitter.

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Gene Therapy for Huntington's Disease in Development - Rare Disease Report

Investment Thesis

With an undervalued stock, a critically-acclaimed drug leading in its space and a barrage of financial and expertise-based backing, Sangamo Therapeutics (NASDAQ:SGMO) is the ideal vehicle for exposure to a market with high growth potential and a hedge against the antiquated methods (CRISPR/Cas9) of treating hemophilia. The same method has also been found to work to a high degree of accuracy on other genetic disorders.

Sangamo Therapeutics' accomplishments to date have helped to draw integrity to its operations and raise its profile against common competitors in the space (QURE, GSK, ONCE, BMRN, BLUE, BIVV (a pure-play hemophilia company). Principally, Sangamo has secured an exclusivity agreement (and subsequent vote of confidence) from Pfizer (NYSE:PFE) (also once a competitor) regarding the development and commercialization of gene therapy programs for hemophilia A (SB-525), which affects 85% of hemophilia sufferers (Nelson Pediatrics), bagging $70 million upfront and a potential $475 million in milestones and royalties (Reuters). (The Zinc Finger Protein Nuclease technology method adopted is targeted at diseases which are caused by genetic defects, as their removal is known to respond best to gene editing targeting, in particular, tauopathies, thalassemias, hemophilia, and HIV/AIDS).

The company's strong balance sheet was further bolstered by a raise of circa $78.1 million through public offering at a price of $7.25 per share, with a revised analyst share price target estimates from Jefferies LLC of $17 per share (jefferies.com), as a "financial investment hedge against emerging gene-editing technology of CRISPR," the CRISPR/Cas9 system, has seen less successful testing, coupled with scientific criticism for its reported high incidence of nonspecific DNA cleavage, mosaicism (failure to replicate mutant alleles in only some of the cells) and inversely, and overproduction of mutations in a given organism.

SGMO's latest 10-Q (SEC.gov):

In the context of gene therapy, Sangamo ($707.16 million market cap) (capitaliq.com) was the only firm with innovative gene therapy drugs to advance in two clinical trials (namely the SB-728-T program in the Sangamo ZFP Therapeutic Pipeline targeting HIV/AIDS), and the first to conduct genome editing studies in clinical trials, as covered in the Financial Times. The stock's performance has been volatile, and investors were disinclined to back the drug lottery in gene therapy as biotech became heavily saturated in players, regulations and setbacks, stunting the prospect of any tangible advancement.

Sangamo has jumped on the "radar" to become a buy. The FDA has authorized Fast Track designation, the scientific backing is legitimate (and has received industry-wide appraisal), the longer-term targets look plausible and Sangamo's management and consultants are capable of executing given their credentials and two decades of development.

By all measures of valuation, Sangamo is undervalued, and price-action enthusiasts will recognize the surge in trading volumes and the breaking of the 50-day moving average. Early riders would have capitalized on the short-term price depression triggered by the publicizing of the mucopolysaccharidosis Type I (MPS I) and MPS II delay, an overreaction from the market that posed a great value opportunity at the beginning of this month, whose benefits were felt just last week when the stock surged on the impressive FIH results.

Gene therapy (and emicizumab) renders recombinant factor VIII/IX proteins redundant. This a slice of the industry that is represented by $8-10 billion annually (with 90%+ profit margins) shared by Shire (NASDAQ:SHPG), Bayer (OTCPK:BAYRY), Novo Nordisk (NYSE:NVO), Pfizer and Bioverativ (NASDAQ:BIVV), an overvalued stock whose current operations consist entirely of the moribund method of treating hemophilia - a potential sell. It recently acquired True North and thus entering into cold agglutinin disease market, a result of severe hemolytic anemia.

Sangamo now has the backing and leeway to make its drugs commercially viable, magnetizing further interest - 48 institutions have increased their respective positions in SGMO, with Wasatch and BlackRock leading the pack. The smart money and medical experts are backing Sangamo.

Bold tickers indicate potential buys should this thesis play out as predicted.

This is not investment advice, you are advised to carry out your own due diligence.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Sangamo Therapeutics: The Breakthrough In Gene Therapy - Seeking Alpha

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GAINESVILLE, Florida Imagine only being able to see the things in front of you in soft focus, and just in black and white.

For people with the genetic eye condition achromatopsia, those are just some of the side effects.

Each time Tara Cataldo prepares to leave her house, she has to make sure her face is completely shielded from the sun.

"I need to have very dark, very tinted sunglasses to feel comfortable outside, to see really well," said Tara.

Tara has achromatopsia, a genetic condition that makes her eyes incredibly sensitive to light. She is also very nearsighted; even while wearing glasses or contacts, she can only see clearly at a very short distance.

"I cannot drive a car so I rely on public transportation and my bike to get around," she said.

"There are currently no approved or no effective treatments for achromatopsia," said Dr. Christine Kay, a surgical ophthalmologist at the University of Florida.

But she's working to change that.

She is one of a handful of experts testing a gene therapy.

"For achromatopsia the cells we have to target are cone cells responsible for decreased vision and color vision. and those are cells at the very bottom layer of the retina," explained Dr. Kay.

Using a tiny cannula, doctors deliver a normal copy of one of two mutated genes, the CNGA-3 or CNGB-3 gene, directly into the eye to restore vision.

Tara`s myopia is so severe that her risk of retinal detachment from any retinal surgery is high, which rules her out for the current trial.

In the meantime, Tara says she's learned to adapt to achromatopsia and live without limitations.

"And I hope, ya know, all young achromats, ya know, learn the same thing."

Dr Kay says if the gene could eventually be delivered to the surface of the retina, additional patients, like Tara, could be treated. AGTC, the biotech company that developed the therapies, and several U.S. universities have successfully tested this therapy in dogs and sheep.

NEW TECHNOLOGY: A new clinical trial is underway testing gene therapy for achromatopsia. The genes are responsible for releasing proteins essential for the function of all the cells. Researchers remove the virus from the host DNA so it does not have the capacity to make the patient sick, and then insert the gene of interest into the DNA. Surgically done by a vitro retinal surgeon, the gene is then directly delivered to the retinal tissue. Researchers try to avoid directly touching the retina, to avoid detaching it and keep the surgery less evasive. The CNGA3 or CNGB3 gene would help restore the patient`s vision. This can only be done on certain patients; those with extremely severe achromatopsia are not able to participate in this trial because of the risk of retinal rupturing.

If this story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Jim Mertens at jim.mertens@wqad.com or Marjorie Bekaert Thomas at mthomas@ivanhoe.com.

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YOUR HEALTH Gene therapy for better eyesight - WQAD.com

MOSCOW, August 17. /TASS/. A Russian gene therapy drug for individuals infected with HIV called Dinavir is undergoing pre-clinical trials, and the drug has already proved its efficiency on cells. The pre-clinical tests on animal models, clinical trials and the registration procedure may take up to 10 years, senior research fellow at the Epidemiology Central Research Institute of Rospotrebnadzor (the Federal Service on Surveillance for Customers Rights Protection and Human Well-Being) Dina Glazkova told TASS.

"This is not about the next year, but rather in five years, at the earliest. It takes up to 10 years on the average," she said.

Glazkova reiterated that the registration is made after the clinical trials. "Again, the clinical trials are costly, and the drug production is costly as well," the scientist added.

Dinavir proved to be safe while tested on cells, in vitro. A Phase II pre-clinical trial will utilize animal models to test the efficiency and safety of treatment. A Phase I clinical trial will be carried out on humans to test safety of the therapy and will take up not less than a year.

"Phase II takes up two to three years, and it is unclear how much will be required from us. Phase I is about safety, and it takes a few patients: five, maybe ten. Phase II is when we have to prove that the drug works in these five to ten [patients] and that it had a positive effect on them. Phase III is when we enroll a lot of patients [in the trial] to show that the five were cured not by accident and that it [the gene therapy] really works," Glazkova explained.

The gene therapy for HIV treatment is being developed by a group of researches at the Epidemiology Central Research Institute of Rospotrebnadzor.

In other media

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Scientists foresee Russian gene therapy for HIV cure may be registered in 5-10 years - TASS

Imagine only being able to see the things in front of you in soft focus, and just in black and white. For people with the genetic eye condition achromatopsia those are just some of the side effects. But, researchers are testing a new treatment designed to cure the condition by fixing the gene responsible.

Each time Tara Cataldo prepares to leave her house, she has to make sure her face is completely shielded from the sun.

I need to have very dark, very tinted sunglasses to feel comfortable outside and to see really well. Cataldo said.

Cataldo has achromatopsia, a genetic condition that makes her eyes incredibly sensitive to light. She is also very nearsighted; even while wearing glasses or contacts, she can only see clearly at a very short distance.

I cannot drive a car so I rely on public transportation and my bike to get around. Cataldo explained.

"There are currently no approved and no effective treatments for achromatopsia, said University of Florida surgical ophthalmologist Christine Kay.

Kay is working to change that. She is one of a handful of experts testing a gene therapy.

For achromatopsia the cells we have to target are cone cells responsible for decreased vision and color vision and those are cells at the very bottom layer of the retina, Kay explained.

Using a tiny cannula, surgeons deliver a normal copy of one of two mutated genes; the CNGA3 or CNGB3 gene, directly into the eye - restoring vision.

Cataldos myopia is so severe that her risk of retinal detachment from any retinal surgery is high, which rules her out for the current trial. In the meantime, Cataldo says shes learned to adapt to achromatopsia and live without limitations.

And I hope all young achromats learn the same thing, Cataldo said.

Kay says if the gene could eventually be delivered to the surface of the retina; additional patients, like Cataldo, could be treated. Applied Genetic Technologies Corporation, the biotech company that developed the therapies, and several U.S. universities have successfully tested this therapy in dogs and sheep.

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Achromatopsia: Gene Therapy Cures Eye Disease? - NBC 5 Dallas-Fort Worth

LONDON--(BUSINESS WIRE)--According to the latest market study released by Technavio, the global cancer gene therapy market is expected to grow at a CAGR of almost 21% during the forecast period.

This research report titled Global Cancer Gene Therapy Market 2017-2021 provides an in-depth analysis of the market in terms of revenue and emerging market trends. This market research report also includes up to date analysis and forecasts for various market segments and all geographical regions.

The rising prevalence rate of cancer has been a huge challenge for the global economies as the disease leads to high rate of mortality and economic losses. The current treatment options available come with many drawbacks such as severe side effects and relapse of cancer. These factors have led to high investment in the R&D for development of various novel therapies with cancer gene therapy being one of the major ones of them. The therapy mainly uses three types of treatment options namely oncolytic virotherapy, gene transfer therapy, and gene-induced immunotherapy.

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Technavios healthcare and life sciences research analysts categorize the global cancer gene therapy market into the following segments by therapy. They are:

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Technavios sample reports are free of charge and contain multiple sections of the report including the market size and forecast, drivers, challenges, trends, and more.

Oncolytic virotherapy

Oncolytic virotherapy is one of the fastest growing treatment modality. In this therapy, the anti-cancer cells specifically destroy the cancer cells without causing harm to the normal cells. Each virus has a specific cellular tropism that determines which tissue will be preferentially infected by the virus and thus will further lead to the disease.

According to Sapna Jha, a lead oncology research analyst from Technavio, The oncolytic virotherapy has shown encouraging results in the pre-clinical studies. The novel treatment option holds great opportunity to make a significant effect on quality and length of the life of the individual. Adenovirus is the most commonly used virus in oncolytic virotherapy.

Gene transfer

Gene transfer or gene insertion is one of the most exciting and emerging cancer treatment methods. The therapy is expected to be the fastest growing type of therapy in the cancer gene therapy market. This is a radical new treatment method that involves the introduction of a new gene into the cancer cell or the surrounding tissues.

Genes with different functions have been proposed for this therapy; some of them include antiangiogenesis genes, cellular stasis genes, and suicide genes. Many different viral vectors are used to deliver these genes, Adenovirus being most common of them. Other than viral vectors, certain non-viral methods are also studied in the various clinical trial, which includes oligodendromer DNA coatings and naked DNA transfer, adds Sapna.

Gene-induced immunotherapy

Immunotherapy works on the concept of boosting the immune system of the individual to target and destroy cancer cells. However, traditional immunotherapy has shown limited success rate in the field. Various gene therapy techniques are being used to overcome this limitation.

The next-generation gene-induced immunotherapy vaccines are already in clinical trial. Gene-induced immunotherapy is a type of gene therapy where genetically engineered genes are used to generate an immune response against cancer. Growing knowledge and understanding of mechanisms regulating the initiation and maintenance of cytotoxic immune response has led to the designing of several genetic immunization strategies.

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Technavio is a leading global technology research and advisory company. Their research and analysis focuses on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions.

With over 500 specialized analysts, Technavios report library consists of more than 10,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavios comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios.

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Cancer Gene Therapy Market - Forecasts and Opportunity Assessment by Technavio - Business Wire (press release)

August 16, 2017 09:25 ET | Source: Abeona Therapeutics Inc

NEW YORK andCLEVELAND, Aug. 16, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc.(Nasdaq:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, today announced the pivotal expansion of its gene therapy clinical trials for patients with MPS IIIA in the USA, Europe and Australia.

We have completed the necessary regulatory and ethical committee approvals and site initiations in Europe and Australia in order to accelerate enrollment, stated Juan Ruiz, M.D., Ph.D., Abeonas Chief Medical Officer. We remain very encouraged by the improvements observed in clinically relevant biomarkers post-dosing of ABO-102, including durable reductions in heparan sulfate measured in the CNS, reduction of organ disease pathology, and signals of CNS improvement or stabilization at one-year follow-up in Cohort 1 subjects, and look forward to providing a more fulsome clinical update at important clinical conferences, including ESGCT, this fall, he continued.

The planned expansion will enroll an additional eight to ten MPS IIIA subjects, with total enrollment of fourteen to sixteen subjects to be completed by 1Q2018. Per the design of the clinical trial, subjects will receive a single, intravenous injection of ABO-102 to deliver the AAV viral vector systemically throughout the body to introduce a corrective copy of the gene that underlies the cause of the MPS IIIA disease. Subjects are evaluated at multiple time points post-injection for safety assessments and initial signals of biopotency and clinical activity, which suggest that ABO-102 successfully reached target tissues throughout the body, including the central nervous system.

Sanfilippo syndromes (or mucopolysaccharidosis (MPS) type III): a group of four inherited genetic diseases each caused by a single gene defect, described as type A, B, C or D, which cause enzyme deficiencies that result in the abnormal accumulation of glycosaminoglycans (GAGs, or sugars) in body tissues. MPS III is a lysosomal storage disease, a group of rare inborn errors of metabolism resulting from deficiency in normal lysosomal function. The incidence of MPS III (all four types combined) is estimated to be 1 in 70,000 births. Mucopolysaccharides are long chains of sugar molecule used in the building of connective tissues in the body. There is a continuous process in the body of replacing used materials and breaking them down for disposal. Children with MPS III are missing an enzyme which is essential in breaking down the used mucopolysaccharides called heparan sulfate. The partially broken down mucopolysaccharides remain stored in cells in the body causing progressive damage. In MPS III, the predominant symptoms occur due to accumulation within the central nervous system (CNS), including the brain and spinal cord, resulting in cognitive decline, motor dysfunction, and eventual death. Importantly, there is no cure for MPS III and treatments are largely supportive care.

About Abeona: Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene therapies for life-threatening rare genetic diseases. Abeona's lead programs include ABO-102 (AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB). Abeona is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), EB-201 for epidermolysis bullosa (EB), ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona has a proprietary vector platform, AIM, for next generation product candidates. For more information, visit http://www.abeonatherapeutics.com.

Investor Contact: Christine Silverstein Vice President, Investor Relations Abeona Therapeutics Inc. +1 (212)786-6212 csilverstein@abeonatherapeutics.com

Media Contact: Andrea Lucca Vice President, Communications & Operations Abeona Therapeutics Inc. +1 (212)786-6208 alucca@abeonatherapeutics.com

This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and that involve risks and uncertainties. These statements include, without limitation, our plans for continued development and internationalization of our clinical programs, that patients will continue to be identified, enrolled, treated and monitored in the EB-101 clinical trial, and that studies will continue to indicate that EB-101 is well-tolerated and may offer significant improvements in wound healing; the addition of two additional global clinical sites will accelerate our ability to enroll and evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA. Such statements are subject to numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition; the ability to secure licenses for any technology that may be necessary to commercialize our products; the ability to achieve or obtain necessary regulatory approvals; the impact of changes in the financial markets and global economic conditions; our belief that initial signals of biopotency and clinical activity, which suggest that ABO-102 successfully reached target tissues throughout the body, including the central nervous system and the increased reductions in CNS GAG support our approach for intravenous delivery for subjects with Sanfilippo syndromes, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other reports filed by the Company with theSecurities and Exchange Commission. The Company undertakes no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

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Abeona Therapeutics Announces Pivotal Expansion of ABO-102 Gene Therapy Clinical Trials in Sanfilippo Syndrome ... - GlobeNewswire (press release)

Gene therapies for children with Batten disease are being evaluated in two clinical trials one in New York and the other in Ohio. The trials are testing ways to treat two types Batten disease by correcting the genetic defect that causes it.

One study is a Phase 1/2 clinical trial (NCT01414985) taking place at Weill Cornell Medical College in New York thats assessing the safety and effectiveness of using the AAVRh.10 virus to deliver a corrected CLN2 gene to children with late infantile Batten disease, also known as Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL).

This type of Batten disease, a lysosomal storage disorder, can be caused by a mutation in the CLN2 gene. The damage disrupts the ability of brain cells to recycle proteins, killing nerve cells within the brain and leading to progressive neurological and brain damage. The onset of symptoms, a mix of vision and motor problems, are usually evident in children between 2 and 4 years old.

The trial, which is recruiting eight patients ages 3 to 18 with a definite diagnosis of LINCL, will deliver a healthy CLN2 gene using the AAVRh.10 virus a primate-derived virus known to be safe in people as the gene transfer vector. Two study arms are planned: one, involving two patients, will receive a higher dose of gene copies, and the other, composed of six patients, will be given a lower dose.

According to a Weill Cornell document on file with the National Institutes of Health, studies in CLN2 knockout mice showed the virus to be an effective gene delivery system and the treatment to have the potential to slow down the progression of the disease.

Treatment effectiveness will be determined at months 1, 6, 12, and 18 using the Weill Cornell LINCL scale, a 12-point measure of central nervous system response through changes in feeding, gait, and motor and language skills. Secondary measures will be judged using MRI scans and questionnaires.

This study expects to conclude in December 2020, and will be led by Dr. Ronald Crystal of Weill Medical College. More information, including enrollment contacts, is available on its clinical trials.gov webpage.

In an earlier trial, the same Weill team used the AAV2 virus to deliver a corrected CLN2 gene, and reported that it showed both safety and small but significant benefit in treated patients. Researchers believe the AAVRh.10 virus is a more effective delivery system than AAV2, the NIH document states.

In the second trial, NCT02725580, the adeno-associated virus 9 (AAV9) will be used to carry a corrected CLN6 gene to patients with the variant late infantile Batten CLN6 disease. This open label and first-in-human trial, taking place at Nationwide Childrens Hospital in Columbus, Ohio, will enroll at least six people, ages 1 year or older, with a mutation in that gene. The treatment will be delivered once directly into the spinal cord through a lumbar puncture.

Safety and toxicity is this studys primary objective, but secondary measures of treatment efficacy will be made using MRI scans and cognitive, vision and language testing.

The Gray Foundation, established in 2015 by the parents of Charlotte and Gwenyth Gray, both of whom have this variant of Batten disease with a mutation in the CLN6 gene, is funding and collaborating in this study.

Its principal investigator is Dr. Jerry Mendell with the Center for Gene Therapy at Nationwide Childrens Hospital.

Trial enrollment and other information is available on the studys clinical trials.gov webpage.

Continued here:

Batten Disease Clinical Trials Enrolling Patients to Evaluate Gene Therapies - Batten Disease News

Friedreichs Ataxia (FA) is a genetic condition caused by mutations in theFXNgene. The mutation leads to a decrease in the production of frataxin protein causing progressive neuronal degeneration, loss of muscle control, fatigue, vision or hearing impairment, slurred speech, and heart problems. As FA is caused by mutations in a single gene, it might be treatable through agene therapyapproach.

Gene therapy consists of the intracellular delivery of genetic material to generate a therapeutic effect by correcting an existing abnormality or providing the cells with a new function.

Different types of gene delivery systems may be used in gene therapy to restore a specific gene function, such as viral and non-viral vectors.

Viral vectors are viruses that have been modified by deleting the harmful areas in their genomes, while non-viral vectors are physical and chemical systems, such as liposomes, nanoparticles, polymers, ultrasound, or laser-based or magnetic energy.

Non-viral vectors are advantageous in relation to viral vectors as delivery vehicles as they are simple to prepare and scale-up and usually have less pro-inflammatory effects than viral ones.

A gene therapy approach for the treatment of FA could be used either to correct the faulty frataxin geneor to correct any damage caused by the disease on other systems in the body such as the heart.

Voyager Therapeuticshas a pipeline of investigational molecules to target severe diseases of the central nervous system, such as FA. They are currently starting preclinical tests to assess the safety and effectiveness of VY-FXN01, a lead candidate for the treatment of theneurological symptoms of FA.

Earlier in 2014, mouse models of FA were used to demonstrate that gene therapy using a viral vector prevented and corrected cardiac damage.Researchers used an adeno-associated virus to introduce a normal gene into the heart tissue of mouse models of FA. This treatment restored heart function and reversed heart enlargement in mice that had already developed heart failure, a symptom seen in people with FA.

A project supported byGENEFA, a platform for an FA cure, is investigating ways to modify vectors to improve their delivery across the blood brain barrier(BBB). By generating modified viral vectors and virus-free synthetic nanoparticles the aim is to make the DNA delivery into the nervous system more efficient. It is hoped that these nanosystems will cross the BBB thanks to the peptides that are able to cross them (referred to as BBB-shuttles).

Note:Friedreichs Ataxia Newsis strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician orother qualified health providerwith any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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Gene Therapy for Friedreich's Ataxia - Friedreich's Ataxia News

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How this report will benefit you:

Read on to discover how you can exploit the future business opportunities emerging in this sector.

In this brand-new127 page reportyou will receive70 charts- all unavailable elsewhere.

The127-page report provides clear detailed insight into the gene therapy market. Discover the key drivers and challenges affecting the market.

By ordering and reading our brand-new report today you stay better informed and ready to act.

Report Scope:

Gene Therapy market forecastsfrom2017-2027

This reportassesses the approved gene therapy productsin the market and givesrevenue to 2027 for Neovasculgen

Providesqualitative analysis and forecastof thesubmarket by indicationfor the period 2017-2027: Cancer Cardiovascular disorders Rare diseases Ophthalmological diseases Other therapeutic uses

Profilesleading companiesthat will be important in the development of the gene therapy market. For each company, developments and outlooks are discussed and companies covered in this chapter include: UniQure Biogen Bluebird Bio Spark Therapeutics Applied Genetics Technologies Corporation Oxford Biomedica GenSight Biologics

Assesses the outlook for theleading gene treatment R&D pipelinefor 2016 and discusses technological progress and potential. Profiles appear for gene therapy drug candidates, withrevenue forecasts for six leading agents: SPK-RPE65 (Spark Therapeutics) Collategene (AMG0001, AnGes MG/Vical) Invossa (TissueGene-C, TissueGene Inc/Kolon Life Science) BC-819 (BioCancell) Lenti-D (Bluebird Bio) GSK2696273 (GlaxoSmithKline)

Provides qualitative analysis of trends that will affect the gene therapies market, from the perspective of pharmaceutical companies, during the period 2017 to 2027.SWOT analysisis provided andan overview of regulation of the gene therapy market by leading regiongiven.

Our study discussesfactors that influence the marketincluding these: Translation of research into marketable products modifying human DNA - gene transfer for therapeutic use, altering the nuclear genome Genomic editing technology and other supporting components Collaborations to develop and launch gene-based products - acquisitions and licensing deals Supporting technologies for human genetic modification, gene replacement and targeted drug delivery Gene therapies for ophthalmologic diseases - next-generation medicines Regulations in the United States, the European Union and Japan - overcoming technological and medical challenges to pass clinical trials.

Visiongain's study is intended for anyone requiring commercial analyses for the gene therapy market. You find data, trends and predictions.

Buy our report todayGene Therapy R&D and Revenue Forecasts 2017-2027: Cancer, Cardiovascular, Rare Diseases, Ophthalmologic, Other Diseases.

To request a report overview of this report please email Sara Peerun at sara.peerun@visiongain.com or call Tel: +44-(0)-20-7336-6100

Or click on https://www.visiongain.com/Report/1954/Gene-Therapy-R-D-and-Revenue-Forecasts-2017-2027

List of Companies and Organisations Mentioned in the Report:

Active Medical, Inc.

AngioDynamics, Inc.

Aspen Laboratories

AtriCure, Inc.

Barcapel Foundation

Biosense Webster, Inc.

Boston Scientific Corporation

British Association of Aesthetic Plastic Surgeons (BAAPS)

BSD Medical Corporation

C.R. Bard

Cosman medical, Inc.

Covidien

DFINE, Inc.

Endosense SA

Ethicon

Food and Drug Administration (FDA)

Galil medical, Inc.

Johnson & Johnson

Linvatec Canada ULC

Macmillan Cancer Support

Medtronic

Microsulis Medical Ltd.

Monteris Medical

National Institute of Health Research (NIHR)

nContact, Inc.

NeuroTherm, Inc.

NeuWave Medical, Inc.

NxThera, Inc.

Olympus Corporation

Perseon Corporation

Profound Medical Corp.

Royal Brompton & Harefield NHS Foundation Trust

Royal Philips

Shandong Provincial Hospital

Smith & Nephew

SonaCare Medical

St Jude Medical

Terumo Europe

The American Heart Association

Trod Medical N.V.

University College London

To see a report overview please email Sara Peerun on sara.peerun@visiongain.com

SOURCE Visiongain Ltd

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Gene Therapy R&D and Revenue Forecasts 2017-2027 - PR Newswire (press release)