Archive for the ‘Gene Therapy Research’ Category

Sept. 19, 2019 06:38 UTC

MOUNTAIN VIEW, Calif.--(BUSINESS WIRE)-- The SanBio Group (SanBio Co., Ltd. and SanBio, Inc.)(TOKYO:4592), a scientific leader in regenerative medicine for neurological disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) Designation for SB623 cell therapy for the treatment of chronic neurological motor deficits secondary to traumatic brain injury (TBI). The designation is based on clinical results of SB623 including the Phase 2 Study of Modified Stem Cells in Traumatic Brain Injury (STEMTRA) trial.

Created under the 21st Century Cures Act, the RMAT designation is reserved for a regenerative medicine therapy intended to treat, modify, reverse, or cure a serious condition, and clinical evidence indicates that the therapy has the potential to address unmet medical needs for such disease or condition. Similar to the Breakthrough Therapy designation, the RMAT designation offers sponsors of cell and gene therapies eligibility for expedited development and regulatory review of their product candidate, including earlier and more frequent consultation with the FDA, and the potential for Priority Review and Accelerated Approval.

The RMAT designation for SB623 is an important regulatory milestone for SanBio as we investigate it as a treatment option for patients with chronic neurological motor deficits resulting from a traumatic brain injury, said Bijan Nejadnik, M.D., Chief Medical Officer and Head of Research. TBIs are one of the most common health conditions worldwide that often cause long-term complications or death. We look forward to working with the FDA on a potentially accelerated clinical development program to address this serious unmet medical need.

The RMAT designation augments the Sakigake Designation for innovative medical products from the Ministry of Health, Labour, and Welfare of Japan.

About SB623 SB623 is a proprietary, cell-based investigational product made from modified and cultured adult bone marrow-derived mesenchymal stem cells that undergo temporary genetic modification. Implantation of SB623 cells into injured nerve tissue in the brain is expected to trigger the brains natural regenerative ability to recover lost motor functions.

SanBio expects to initiate a Phase 3 trial for SB623 for the treatment of chronic neurological motor deficits secondary to TBI by the end of the fiscal year ending January 31, 2021. SB623 is also currently in a Phase 2b clinical trial for treatment of chronic motor deficit resulting from ischemic stroke.

About the Study of Modified Stem Cells in Traumatic Brain Injury (STEMTRA) Trial STEMTRA was a 12-month, Phase 2, randomized, double-blind, surgical sham-controlled, global trial evaluating the efficacy and safety of SB623 compared to sham surgery in patients with stable chronic neurological motor deficits secondary to TBI. In this study, SB623 cells were implanted directly around the site of brain injury.

To be eligible for this trial, patients (ages 18-75) must have been at least 12 months post-TBI and had a Glasgow Outcome Scale extended (GOS-E) score of 3-6 (e.g., moderate or severe disability). Patients must also have been able to undergo all planned neurological assessments and had no seizures in the prior three months. The primary endpoint was mean change from baseline in Fugl-Meyer Motor Scale (FMMS) score which measures changes in motor impairment at six months. The STEMTRA trial enrolled 61 patients from 13 surgical and 18 assessment sites in the U.S., Japan and Ukraine.

In this study, SB623 met its primary endpoint, with patients treated with SB623 achieving an average 8.3 point improvement from baseline in the FMMS, versus 2.3 in the control group, at 24 weeks (p=0.040). Of patients treated with SB623, 18 (39.1%) reached a 10 or more point improvement of FMMS compared to one control patient (6.7%; p=0.039). No new safety signals were identified. The most commonly reported adverse event were headaches.

About SanBio Group (SanBio Co., Ltd. and SanBio, Inc.) SanBio Group is a regenerative medicine company with cell-based products focused on neurological disorders in various stages of research, development and clinical trials. The Companys lead asset, SB623, is currently being investigated for the treatment of several conditions including chronic neurological motor deficit resulting from ischemic stroke and traumatic brain injury. SanBio has received a Japanese marketing license for regenerative medicine products from the Tokyo Metropolitan Government, and plans to begin marketing regenerative medicine products in Japan by the end of the fiscal year ending January 31, 2021. The Company is headquartered in Tokyo, Japan and Mountain View, California, and additional information about SanBio Group is available at https://sanbio.com.

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SanBio Granted Regenerative Medicine Advanced Therapy Designation from the US FDA for SB623 for the Treatment of Chronic Neurological Motor Deficits...

Study Touts Preventative Nerve Damage Imaging for 600,000 Annual Surgical Patients; Gene Therapy Research Looks to Increase 5-Year LSD Survival Rate

CULVER CITY, Calif., Sept. 13, 2019 /PRNewswire-PRWeb/ -- Two studies presented at the annual Congress of the World Molecular imaging Society (WMIS) focused on non-invasive real-time tracking of gene therapy and advances to decrease nerve damage using Fluorescence image-guided surgery.

With mean survival rate of five years (and most cases fatal) lysomal storage diseases (LSD) are among the most dismal of prognosis in all of medicine. In the study Real-Time Tracking of Gene Therapy by Bioactivated MR Probes, Thomas Meade, Ph.D., Professor, Northwestern University, stated that LSDs represent a large number of monogenetic diseases and, while rare, the prevalence is to hemophilia. The transformative results documented in an adeno-associated virus (AAV) gene therapy clinical trial in infants, affected by spinal muscular atrophy, demonstrated unequivocally the potential of in vivo gene transfer to treat monogenic neurological disorders. To date there is a lack of non-invasive ways to determine biodistribution or activity levels of these AAV therapies in patients.

"We pioneered the development of bioresponsive (bio-activated) MR contrast agents and since then the library of this class of probes has expanded from enzyme activated agents to include pH sensitive and redox activated," said Dr. Meade. "In our research, we want to determine if a new class of MR contrast agents could track the delivery of a gene by a noninvasive technique (MR imaging). The need for non-invasive, disease specific biomarkers that reflect treatment efficacy is paramount and not limited to gene therapy, but also applies to anything that augments the targeted enzyme activity."

According to Dr. Meade, his team is developing a new class of bioresponsive MR probes to track enzymatic activity in any organ, peripheral nervous system (PNS), or central nervous system (CNS) over time. As a result, gene therapy can be noninvasively monitored at both where and when the gene of interest is activated.

"The number of academic and biotech led programs has risen exponentially in the last few years," Dr. Meade concluded. "It is indeed an exciting and hopeful new era for the gene therapy field, but effective clinical trials with shortened paths to approval will require robust biomarkers to track therapeutic effect. It is our goal to support this important effort."

In the Nerve-Sparing Agents for IGS (Image Guided Surgery) presentation, Connor Barth, Ph.D., Senior Research Associate, Oregon Health & Science University, speaks about the widespread issue of resulting nerve damage from surgery.

"Nerve damage plagues surgical outcomes, significantly affecting post-surgical quality of life," said Dr. Barth. "Some 600,000 patients are affected by nerve damage during surgery annually in the United States. For some procedures, such as the radical prostatectomy, in which the prostate is removed as a prostate cancer cure, the rate of nerve damage can be as high as 60 percent."

During surgery, intraoperative nerve detection is completed using anatomical knowledge and conventional white light visualization when possible. However, neuroanatomy is variable between patients, especially in injured and diseased states that would be present during surgery, and nerves are typically protected deep within the tissue so that white light visualization is largely impossible. Fluorescence image-guided surgery offers a potential means for enhanced intraoperative nerve identification and preservation. To date, a variety of nerve-specific fluorophores have been tested in preclinical models; however, a clinically approved nerve-specific contrast agent does not yet exist.

"We have developed nerve-specific fluorophores that provide direct and buried nerve visualization during surgery with near-infrared fluorescence," Dr. Barth stated.

"The purpose of this study was to generate clinically relevant formulations and quantify the toxicity and pharmacology of our first-in-class, near-infrared nerve specific fluorophores. This work takes our technology one step closer to clinical translation for use during fluorescence-guided surgery."

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Dr. Barth believes this work will ultimately have a broad impact on surgical outcomes, significantly improving the rates of nerve damage during surgery and thus higher post-surgical quality of life for patients. This technology could also serve to make procedures available to surgeons where potential nerve damage has been prohibitive, broadening the clinical impact by enabling curative surgical treatments.

ABOUT WORLD MOLECULAR IMAGING SOCIETY

The WMIS is dedicated to developing and promoting translational research through multimodality molecular imaging. The education and abstract-driven WMIC is the annual meeting of the WMIS and provides a unique setting for scientists and clinicians with very diverse backgrounds to interact, present, and follow cutting-edge advances in the rapidly expanding field of molecular imaging that impacts nearly every biomedical discipline. Industry exhibits at the congress included corporations who have created the latest advances in preclinical and clinical imaging approaches and equipment, providing a complete molecular imaging educational technology showcase. For more information: http://www.wmis.org

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Nerve Damage Imaging and Single-Cell Tracking Gene Therapy are Highlights at World Molecular Imaging Society Annual Congress - Yahoo Finance

Researchers from the LSU School of medicine have recently found that metformin, a common diabetes treatment, may be effective in combatting cancer. The team found that the Type 2 diabetes drug could provide unique benefits to those with cancers that lack the Nischarin protein, often seen in breast cancer. This work was led by Suresh Alahari, PhD, Professor of Biochemistry and Genetics at LSU Health New Orleans School of Medicine and was published online in the International Journal of Cancer on September 16.

Nischarin, a protein molecule involved in tumor suppression and other biological processes, was discovered by Alahari. His research into this protein has been used largely in breast cancer, being that Nischarin expression is frequently reduced in this form of cancer. In this latest study, however, Alahari and colleagues showed that interfering with Nischarin expression can delay the development of the mammary gland, increase tumor growth and metastasis, and reduce the activation of AMPK.

The AMPK enzyme plays a pivotal role in metabolism and is thought to be therapeutic for metabolic diseases and potentially cancer. The full mechanism by which metformin acts is still unknown, but researchers have identified that the drug works in part by activating AMPK.

The clinical documentation that diabetic patients on a metformin regimen display reduced risks of developing cancer poses the tantalizing possibility that this approach to treating cancer might prove to be an effective and unrealized therapeutic opportunity, explained Alahari.

In their work, Alahari and colleagues showed that metformin successfully activates AMPK and inhibits tumor growth in malignancies that lack Nischarin. These findings indicate that metformin could serve as a powerful therapeutic agent in treating these unique tumors.

We found that Nischarin-deleted tumor cells had lower AMPK activity than Nischarin-positive cells, said Alahari. He continued to explain that metformin treatment activated AMPK more efficiently in Nischarin-deleted mice, and metformin suppressed tumor growth of Nischarin-deleted mice. Collectively, our data suggest that Nischarin disruption promotes breast tumor development, AMPK signaling is important for Nischarin-mediated suppression of breast tumors, and activation of AMPK by metformin suppresses breast tumor growth in Nischarin-lacking mice.

Breast cancer often displays reduced expression of Nischarin, particularly in triple-negative breast cancers. Impacting 2.1 million women every year, breast cancer is the most commonly occurring cancer among women as per the World Health Organization. Being that this disease accounts for roughly 15% of all cancer deaths among women, finding effective treatments is of paramount importance. With these new findings regarding metformins ability to combat Nischarin-lacking cancer, Alaharin and colleagues have made a breakthrough in the treatment of breast cancer and other tumors.

The discovery that the effectiveness of certain drugs, such as metformin, are influenced by the level of Nischarin expression could help identify specific patients in whom it is most likely to prove beneficial, added Alahari. In this way, Nischarin expression could serve as a biomarker to help inform decisions in management by identifying a subset of patients most likely to benefit from AMPK activator therapies.

In this work, Alahari was joined by LSU researchers Shengli Dong, Rajamani Rathinam, Steven Eastlack, Mazvita Maziveyi, and Bernardo Ruiz-Calderon. Their research was backed by funding from both LSU Health New Orleans and the Fred G. Brazda Foundation.

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Using Diabetes Medication to Treat Breast Cancer - DocWire News

SAN FRANCISCO, Sept. 19, 2019 (GLOBE NEWSWIRE) -- Cellworks Group, Inc., a leader in Precision Medicine and a global pioneer of Therapy Response Index (TRI) technology, today announced that Blood Advances, an American Society of Hematology Journal, published results from the iCare 1 Clinical Study, which used Cellworks Genomic Biosimulation platform to predict therapy responses for Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS) patients. The prospective study was designed to assess the biological and clinical predictive values of the genomics-informed computational biology modeling (CBM) of patients with AMLand MDS by comparing biosimulated predictions of treatment responses with actual clinical responses.

The iCare 1 prospective study demonstrated 90% accuracy for predicting response to Standard of Care (SOC) treatments in Acute Myeloid Leukemia (AML)and Myelodysplastic Syndromes (MDS) patients. Cellworks unique genomics-informed CBM technology was used in this study to understand the mechanisms of relapse after chemotherapy treatment and to propose new re-induction treatment options for the relapsed patients in this study.

Next-generation sequencing (NGS) has revealed a high number of genomic abnormalities in each MDS and AML patient, as well as a high level of diversity among patients, said Dr. Christopher R. Cogle, MD., Professor at the University of Florida College of Medicine. As clinicians, our greatest challenge is to interpret this high number of genomic abnormalities and genomic diversity into a personalized treatment for our patients. The limitations of time in clinic and the complexity of each patients disease demand that we use technologies like Cellworks biosimulation for the best care of our patients.

Cellworks computational biology modeling (CBM) and digital drug biosimulation platform relies on somatic gene mutations and gene copy number variations (CNVs) found in individual MDS or AML patients. Genomic abnormalities are converted by the computational system into patient-specific protein networks to generate a virtual disease model. The patient-specific disease models are then interrogated using a drug library via biosimulations based on mathematical modeling to predict phenotype response including inhibition of disease cell proliferation and survival.

For this prospective study, 120 patients with AML or MDS were recruited to assess the predictive accuracy of Cellworks CBM by comparing computer predictions of treatment response to actual clinical outcomes. Of the 120 patients, 96 patients had full genomic testing profiles and 50 were eligible for evaluation based on length of follow-up. For the 50 patients, 61 treatments were administered. Cellworks CBM maps of the 32% relapsed samples from iCare 1 patients accurately matched the patient's nonresponse of treatment at relapse and identified mechanisms for chemo-resistance in these patients.

NGS alone does not provide enough information to deliver personalized oncology, said Yatin Mundkur, CEO of Cellworks. By using NGS data as input to biosimulation modeling, we can accurately predict individual patient therapy responses and prevent patients from undergoing unnecessary therapy regimens, thereby avoiding toxic side effects. By improving the accuracy of the treatment selection, we can also reduce health care costs and help increase physician productivity.

Cellworks is transforming personalized cancer therapy through genomic biosimulation software models that represent biomolecular and physiological pathways using the genomic data of each patient. Cellworks personalized medicine predictions help transform lives through the early adoption of successful therapies, while saving time and cost across the healthcare ecosystem. Cellworks also benefits the biopharma industry through virtual clinical trials, improved target identification, lead validation and the ability to repurpose and rescue drugs.

About Cellworks Group, Inc.Cellworks Group, Inc. is a world leader in Precision Medicine in the key therapeutic areas of Oncology and Immunology. The Cellworks unique biosimulation platform is a unified representation of biological knowledge, curated from heterogeneous datasets, applied to finding cures. Backed by Sequoia Capital and Artiman Ventures, Cellworks has the worlds strongest transdisciplinary team of molecular biologists, cellular pathway modelers and machine learning software technologists working toward a common goal attacking serious diseases to improve the lives of patients. The company is based in San Francisco, California with a research and development facility in Bangalore, India. For more information, visit http://www.cellworks.life and follow us on Twitter @CellworksLife.

All trademarks and registered trademarks in this document are the properties of their respective owners.

Media Contacts:Barbara Reichert, Reichert Communications, LLCbarbara@reichertcom.com650-548-1002

Michele Macpherson, Chief Business OfficerCellworks Group, Inc.michele@cellworksgroup.com

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Blood Advances Publishes Cellworks Prospective Genomic Biosimulation Study that Predicts Cancer Therapy Response with 90% Accuracy - GlobeNewswire

Find a random person on the street and ask them what a cancer drug should do. What are the odds they dont say it should help patients live healthier and longer?

A confluence of forces have pushed clinical trials away from that seemingly central question, with developers and patient groups betting on the promise that aiming at more subtle measures can help bring needed therapies to market faster. But a new study from the British Medical Journal suggests the conventional wisdom may be right and that trend, among others, have led cancer drugs to hit the market based off studies that have a high risk of bias.

Researchers led by London School of Economics Professor Huseyin Naci tracked cancer drug approvals by the European Medical Association over three years from 2014 to 2016. Putting aside 13 trials not based on a randomized control model which have their own set of issues but can be useful for rare diseases and two trials without published data, they evaluated 39 trials that formed the basis of drug approvals in that period according to a standard criteria of bias and found: 19 (49%) were at high risk of bias and the ones that didnt look at improving survival were more likely to be at risk. Only 10 trials primarily looked at improving survival.

Among them, 20% of trials that looked at overall survival had a high risk of bias, compared to 55% of the rest.

Other factors besides endpoint were at play in studies at risk for bias, including missing data and red flags in how data were measured, such as the trial lacking a blinded assessment.

But in their discussion of the results, the authors focused in part on the paucity of trials that looked head-on at whether a drug will help patients survive, warning that trials that look at other surrogate endpoints speed up development but might mislead patients and bring drugs that dont work. Evaluating the study in a BMJ opinion column, Barbara Mintzes and Agnes Vitry took similar aim.

Uncertainty and exaggeration of the evidence that supports approval of cancer drugs causes direct harm if patients risk severe or fatal adverse effects without likely benefit, they wrote. Or forgo more effective and safer treatments.

The push toward these surrogate measures has come from both developers, eager to get their treatments to market, and patient groups hoping to secure access to new remedies as fast as possible. Targeting overall survival on average takes about an extra year, time many cancer patients dont have.

A federal law, the 21st Century Cures Act, that went into effect in 2017 codified that push. Yet data on trials that avoid the central question of elongating and improving quality of life have been mixed.

A 2018 JAMA Internal Medicine review of 52 articles covering 38 trials on a total of 14,000 patients with 12 different cancers from 2000 through 2016 found no significant association between progression-free survival and health-related quality of life.

The study also comes as one of many critically evaluating existing scientific literature in the wake of the replication crisis that broke out most publicly in psychology and has rippled across the sciences. In 2012 Amgens head of research, Glenn Begley,published a paper inNaturerevealing a 10-year company effort to reproduce 53 landmark studies in oncology. They got positive results in 6.

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Cancer trials aimed at 'surrogate' targets miss bigger mark study - Endpoints News

An early-stage update on Adverum Biotechnologies intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Over the six month period, patients did not require any anti-VEGF rescue injections and five of six patients saw a complete response with a total resolution of fluid following the Adverum injection. There were no serious adverse events, and the majority of side-effects were mild.

However, patients lost visual acuity by two letters on average, with a 90% confidence interval of -9.1 letters to +5.1 letters.

The range of individual patient data were not presented, though the wide confidence interval suggests that some patients may have experienced a loss of more than 10 letters during the course of the trial lack of rescue injections is difficult to square with declining vision. SVB Leerinks Mani Foroohar wrote in a note.

However, the study investigator insisted no loss in vision was due to wet AMD pathology and observed loss of visual acuity is due to normal variabilityin a small set of patients an assertion that, if proved out with additional follow-up, would very substantially improve the implied quality of this dataset.

Shares of the company which spectacularly failed years ago when it was christened Avalanche Biotechnologies $ADVM were down about 6.8% to $5.56 in Friday premarket trading. The stock sank on Thursday, evaporating millions from its market value.

This data suggest ADVM-022 is potentially active in delivering an expressible gene cassette in wet AMD, but mixed signals in this small dataset should lift some of the competitive overhang on RGNX shares, Foroohar added. RegenexBio experimental gene therapy for wet AMD, RGX-314, is currently in a Phase I/II trial.

Wet AMD, which is characterized by blurred vision or a blind spot in an individuals visual field, is typically caused by abnormal growth of blood vessels that leak fluid or blood into the macula. Macular degeneration is the leading cause of severe, irreversible vision loss in the elderly. Anti-VEGF injections such as Regenerons $REGN flagship Eylea, as well as Roches $RHBBY Lucentis and Avastin, are commonly used to treat wet AMD.

In April, the FDA imposed a clinical hold on an application to test ADVM-022 in humans, asking for additional data on Adverums chemistry, manufacturing and control process. In May, the hold was lifted. Late last year, the biotech abandoned its then lead experimental drug, ADVM-043, for the treatment of A1AT deficiency.

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Early snapshot of Adverum's eye gene therapy sparks concern about vision loss - Endpoints News

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionallyappear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diegos Paul Mischel published his first study in Sciencein 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors superhuman growth.

That insight and the ensuing five years of research will now get $46 million cash and company infrastructure to ramp into targeted therapies as Boundless Bio emerges from stealth mode with backing from ARCH Venture Partners and City Hill.Questions abound, from what precisely a drug would look like to what even gives rise to these wild DNA, but CEO Zachary Hornby isnt biting his tongue on the potential.

Were thinking about this as a fourth revolution in cancer therapies, Hornby, who was most recently COO of Ignyta, told Endpoints News. The first three revolutions, by Hornbys count, are chemotherapy in the 1940s, the first targeted therapies at the end of the 20th century, and the recent rise of immunotherapy.

The road to such a revolution would be long, but the embattled oncology field may be in need of new direction. A study released in April found 97% of cancer drugs tested in clinical trials failed to make it to market, and this month researchers found systemic targeting problems plagued two decades of cancer research.

The connection between this loose DNA, officially called extrachromosomal DNA or ecDNA, and cancer centers around a baffling and deadly fact about some tumors: While normal cells in a higher-order species like humans dont evolve within a generation, some cancer cells can evolve rapidly, ensuring their survival against attempted treatments. Why? How? Mischels mapping of cancer genome points to ecDNA.

Freed from a cells chromosomes, the DNA can replicate rapidly. That doesnt hurt if they code for nothing or something benign, but if they code for something that gives the cell an advantage, such as EGFR (a growth factor), the cells will grow rapidly as in any classical natural selection model. This, Hornby said, appears in over 25% of cancers, including notoriously hard to treat MET cancers.

EGFR inhibitors already exist to combat cancer cells that have already evolved (or been amplified), but Boundless Bio plans to use Mischels insights to destroy ecDNA in its early stages. Rather than attacking tumors after the cells have already amplified, the company would jam the process that gives rise to the evolution in the first place.

It opens a whole new avenue of cancer targets, including allowing us to pursue patient populations that to this point have been undruggable, Hornby said, pointing to MET and Myc. Thats just a really different approach than your typical targeted therapies.

But how they would do this is still cloudy.

Hornby said the most promising method was jamming the enzymatic machinery the molecular tools and parts that allow DNA to replicate and code proteins as their research has shown the machinery is slightly different in ecDNA than typical DNA. Another method theyre exploring is to inhibit the metabolic pathways ecDNA can use to fulfill the demands caused by its high replication rate; in other words, growing DNA that are hungry and depriving them of food could neutralize them.

Among the most notable things about Boundless potential therapies is that they may be approved for tumor type, rather than cancer type, i.e. like the new drug from Hornbys old company Ignyta, it could treat a wide range of cancers if the patient showed ecDNA was amplifying in the tumor.

The company will also invest in research to discover the underlying mechanism giving rise to ecDNA.

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'A fourth revolution in cancer therapies': ARCH-backed Boundless Bio flashes big check, makes bigger promises in debut - Endpoints News

A comprehensive analysis of the Gene Therapy Market including market sizing, market share by competitor, market share by distribution channel, drivers, restraints, product pricing trends, industry quotations, company profiles and market forecasts to 2024 for global market. Gene Therapy market report provides investors, analysts, researchers, business executives and others with analyses and forecasts for the Gene Therapy market, including Gene Therapy production, revenues, competitors marketing strategy and global product selling prices up to 2022.

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Gene Therapy Market Analysis by Major Companies, Segmentation, Market Dynamics & Trends - Real Viewpoint

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Hemophilia is a genetic bleeding disorder in which an individual lacks or has low levels of proteins called clotting factors. There are around 13 types of clotting factors that work with blood platelets, which are necessary for clotting process to initiate. There are three forms of hemophilia A, B, and C. Hemophilia A is the most common form and is caused due to deficiency in clotting factor VIII. Hemophilia B occurs due to deficiency of clotting factor IX and Hemophilia C is caused due to clotting factor XI deficiency. Hemophilia is incurable with current therapeutic options, which only reduces symptoms such as spontaneous bleeding in muscles and joints as well as increased risk for intracranial hemorrhage. These treatment options lasts only for a day and is costly. As against conventional clotting factor replacement therapy, gene therapy is expected to offer sustainable cure for hemophilia by correcting defective gene sequence (F8 or F9 gene) that codes for clotting factor VIII or IX in the patients body.

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Comprehensive Study on Global Hemophilia Gene Therapy Market 20182026 - Coherent Magazine

Zacks Investment Research upgraded shares of Tocagen (NASDAQ:TOCA) from a hold rating to a buy rating in a research note published on Wednesday morning, Zacks.com reports. Zacks Investment Research currently has $1.00 price objective on the stock.

According to Zacks, Tocagen, Inc. is a clinical-stage, cancer-selective gene therapy company which focuses on retroviral gene therapy platforms. The companys lead product candidate consists of Toca 511 & Toca FC, initially for the treatment of recurrent high grade glioma, a disease with a significant unmet medical need. Tocagen, Inc. is based in San Diego, United States.

TOCA has been the subject of a number of other reports. ValuEngine upgraded Tocagen from a hold rating to a buy rating in a research report on Thursday, August 1st. Leerink Swann set a $5.00 price target on Tocagen and gave the stock a hold rating in a research report on Wednesday, May 22nd. Cantor Fitzgerald downgraded Tocagen from an overweight rating to a neutral rating in a research report on Thursday, September 12th. Robert W. Baird downgraded Tocagen from an outperform rating to a neutral rating in a research report on Thursday, September 12th. Finally, LADENBURG THALM/SH SH downgraded Tocagen from a buy rating to a neutral rating in a research report on Thursday, September 12th. Nine research analysts have rated the stock with a hold rating and three have assigned a buy rating to the company. The stock presently has an average rating of Hold and an average price target of $10.26.

Tocagen (NASDAQ:TOCA) last posted its earnings results on Thursday, August 8th. The company reported ($0.72) earnings per share for the quarter, beating analysts consensus estimates of ($0.76) by $0.04. Tocagen had a negative net margin of 300.42% and a negative return on equity of 115.81%. The firm had revenue of $0.01 million during the quarter, compared to analyst estimates of $0.50 million. As a group, sell-side analysts expect that Tocagen will post -2.64 earnings per share for the current fiscal year.

Several large investors have recently added to or reduced their stakes in TOCA. JPMorgan Chase & Co. raised its holdings in shares of Tocagen by 5.1% in the second quarter. JPMorgan Chase & Co. now owns 476,684 shares of the companys stock valued at $3,074,000 after acquiring an additional 23,293 shares in the last quarter. Acadian Asset Management LLC grew its stake in shares of Tocagen by 151.0% during the second quarter. Acadian Asset Management LLC now owns 92,450 shares of the companys stock worth $618,000 after acquiring an additional 55,621 shares during the last quarter. Rhumbline Advisers grew its stake in shares of Tocagen by 9.4% during the first quarter. Rhumbline Advisers now owns 25,491 shares of the companys stock worth $277,000 after acquiring an additional 2,188 shares during the last quarter. GSA Capital Partners LLP purchased a new stake in shares of Tocagen during the second quarter worth about $551,000. Finally, Sphera Funds Management LTD. purchased a new stake in shares of Tocagen during the first quarter worth about $1,304,000. Institutional investors and hedge funds own 35.38% of the companys stock.

About Tocagen

Tocagen Inc, a clinical-stage cancer-selective gene therapy company, focuses on developing and commercializing product candidates designed to activate a patient's immune system against their cancer. Its cancer-selective gene therapy platform is built on retroviral replicating vectors (RRVs), which are designed to deliver therapeutic genes into the DNA of cancer cells.

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Tocagen (NASDAQ:TOCA) Upgraded to Buy by Zacks Investment Research - Mayfield Recorder

Global Gene Therapy Market 2019-2025 report is an in-depth study of Gene Therapy industry Size, Share, Trends and Analysis. It gets to the details of the competitive industry structure around the world. The worldwide Gene Therapy market report study, composed of competent standardized instruments such as S.W.O.T Analysis, offers a comprehensive assessment of the worldwide Gene Therapy market.

Gene Therapy business report offers a full percentage estimate of the CAGR of the period in question that will guide consumers to make choices based on the graph.

The study also covers the main players

Novartis, Kite Pharma, Inc., GlaxoSmithKline PLC, Spark Therapeutics Inc., Bluebird bio Inc.

who leads the worldwide Gene Therapy market.

The experts have calculated the size of the global Gene Therapy market on the basis of 2 major aspects: 1) Income and 2) Production Volume. The subtle analysis of the key chunks of the Gene Therapy market and their geographical diversification all the world has also been carried out. Numerous properties of global Gene Therapy market like upcoming aspects and growth factors related to every segment of the report have been put up thoroughly.

by Vector Type (Viral Vector and Non-viral Vector), Gene Type (Antigen, Cytokine, Tumor Suppressor, Suicide, Deficiency, Growth Factors, Receptors, and Others), and Applications (Oncological Disorders, Rare Diseases, Cardiovascular Diseases, Neurological Disorders, Infectious Disease, and Other Diseases) Analysis and Industry Forecast till 2025

Sample PDF Download Link: https://www.quintagoinsights.com/global-gene-therapy-market/request-sample/426

The worldwide Gene Therapy market study includes every feature of the worldwide Gene Therapy market right from basic market data to that of multiple significant criteria based on which the worldwide Gene Therapy market was diversified.

Gene Therapy industry research report includes a thorough assessment of present policies, rules, and rules along with the worldwide industry chain. Other than that, the worldwide Gene Therapy market report study also covers variables such as the manufacturing chain, top manufacturers, supply as well as demand for those products along with the price structure as well as income.

Also calculated in the global Gene Therapy market research report is the different properties of supply and demand, the chronological presentation, manufacturing capacity along with the detailed analysis of the global Gene Therapy market.

1. To evaluate the status of worldwide Gene Therapy, future forecasts, opportunities for development, key markets, and important players.

2. To present the growth of Gene Therapy in the U.S., Europe, and China.

3. To profile key players strategically and analyze their development plan and strategies comprehensively.

4. Defining, describing and forecasting the market by type of item, market and main areas.

Inquire about the report here: https://www.quintagoinsights.com/global-gene-therapy-market/enquiry-before-buy/426

In the end, as trading experts across the value chain, our panel of trading experts have made extensive attempts to do this group action and heavy-lifting add order to generate important players with helpful main & secondary information on the world electricity sub metering market for Gene Therapy. Furthermore, the study includes inputs from our trade advisors which can make it easier for important players to save half their time from the internal assessment. Companies Who receive and use this report will benefit fully from the inferences contained in it. With the exception of this, the study also offers an in-depth assessment of Gene Therapy.

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Global Gene Therapy Market Analysis Key Players Opportunities Segmentation and Forecast to 2025 - News Conduct - News Conduct

Axovant Gene Therapies Ltd (NASDAQ:AXGT) was the recipient of a significant drop in short interest during the month of August. As of August 15th, there was short interest totalling 1,061,600 shares, a drop of 17.1% from the July 15th total of 1,280,600 shares. Currently, 2.2% of the shares of the company are short sold. Based on an average trading volume of 662,600 shares, the short-interest ratio is presently 1.6 days.

Shares of Axovant Gene Therapies stock traded down $0.23 during trading on Wednesday, hitting $7.63. The companys stock had a trading volume of 78,183 shares, compared to its average volume of 386,879. The stock has a market cap of $178.68 million, a price-to-earnings ratio of -0.95 and a beta of 1.26. The company has a debt-to-equity ratio of 0.60, a current ratio of 1.70 and a quick ratio of 1.70. Axovant Gene Therapies has a 52 week low of $3.81 and a 52 week high of $20.80. The stock has a 50-day moving average price of $6.87 and a two-hundred day moving average price of $4.87.

Axovant Gene Therapies (NASDAQ:AXGT) last released its earnings results on Friday, August 9th. The company reported ($1.23) earnings per share (EPS) for the quarter, beating the consensus estimate of ($1.34) by $0.11. On average, equities analysts predict that Axovant Gene Therapies will post -4.25 EPS for the current fiscal year.

AXGT has been the subject of a number of analyst reports. Cowen reiterated a hold rating on shares of Axovant Gene Therapies in a research report on Tuesday, July 9th. JMP Securities boosted their price objective on shares of Axovant Gene Therapies from $8.00 to $28.00 and gave the company an outperform rating in a research report on Thursday, June 6th. Zacks Investment Research downgraded shares of Axovant Gene Therapies from a buy rating to a hold rating in a research report on Wednesday, August 14th. ValuEngine upgraded shares of Axovant Gene Therapies from a sell rating to a hold rating in a research report on Thursday, August 1st. Finally, Svb Leerink started coverage on shares of Axovant Gene Therapies in a research report on Friday, June 21st. They set an outperform rating and a $18.00 price objective for the company. Three research analysts have rated the stock with a hold rating and eight have assigned a buy rating to the companys stock. The company presently has an average rating of Buy and an average target price of $26.79.

Several hedge funds and other institutional investors have recently made changes to their positions in AXGT. Primecap Management Co. CA acquired a new position in shares of Axovant Gene Therapies during the first quarter valued at about $1,400,000. Marshall Wace LLP acquired a new position in shares of Axovant Gene Therapies during the first quarter valued at about $272,000. Sphera Funds Management LTD. acquired a new position in shares of Axovant Gene Therapies during the first quarter valued at about $6,794,000. BlackRock Inc. acquired a new position in shares of Axovant Gene Therapies during the second quarter valued at about $1,482,000. Finally, Tower Research Capital LLC TRC raised its holdings in shares of Axovant Gene Therapies by 955.3% in the 2nd quarter. Tower Research Capital LLC TRC now owns 4,221 shares of the companys stock worth $27,000 after acquiring an additional 3,821 shares during the last quarter. Hedge funds and other institutional investors own 13.48% of the companys stock.

Axovant Gene Therapies Company Profile

Axovant Gene Therapies Ltd., a clinical-stage gene therapy company, focuses on developing a pipeline of product candidates for debilitating neurological and neuromuscular diseases. The company's current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis, Parkinson's disease, oculopharyngeal muscular dystrophy, amyotrophic lateral sclerosis, and frontotemporal dementia.

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Axovant Gene Therapies Ltd (NASDAQ:AXGT) Sees Large Decrease in Short Interest - TechNewsObserver

Axovant Gene Therapies Ltd (NASDAQ:AXGT) was the recipient of a significant drop in short interest during the month of August. As of August 15th, there was short interest totalling 1,061,600 shares, a drop of 17.1% from the July 15th total of 1,280,600 shares. Currently, 2.2% of the shares of the company are short sold. Based on an average trading volume of 662,600 shares, the short-interest ratio is presently 1.6 days.

Shares of NASDAQ AXGT traded down $0.23 during trading on Wednesday, hitting $7.63. 78,183 shares of the company traded hands, compared to its average volume of 386,879. The businesss fifty day moving average is $6.87 and its 200 day moving average is $4.87. Axovant Gene Therapies has a fifty-two week low of $3.81 and a fifty-two week high of $20.80. The firm has a market capitalization of $178.68 million, a price-to-earnings ratio of -0.95 and a beta of 1.26. The company has a debt-to-equity ratio of 0.60, a quick ratio of 1.70 and a current ratio of 1.70.

Axovant Gene Therapies (NASDAQ:AXGT) last announced its quarterly earnings data on Friday, August 9th. The company reported ($1.23) earnings per share for the quarter, beating the consensus estimate of ($1.34) by $0.11. On average, analysts predict that Axovant Gene Therapies will post -4.25 EPS for the current year.

Several institutional investors and hedge funds have recently bought and sold shares of AXGT. Sphera Funds Management LTD. acquired a new stake in shares of Axovant Gene Therapies in the 1st quarter worth about $6,794,000. Primecap Management Co. CA acquired a new stake in shares of Axovant Gene Therapies in the 1st quarter worth about $1,400,000. BlackRock Inc. acquired a new stake in shares of Axovant Gene Therapies in the 2nd quarter worth about $1,482,000. Marshall Wace LLP acquired a new stake in shares of Axovant Gene Therapies in the 1st quarter worth about $272,000. Finally, Jane Street Group LLC raised its holdings in shares of Axovant Gene Therapies by 28.8% in the 2nd quarter. Jane Street Group LLC now owns 46,455 shares of the companys stock worth $289,000 after acquiring an additional 10,375 shares during the last quarter. Institutional investors own 13.48% of the companys stock.

Axovant Gene Therapies Company Profile

Axovant Gene Therapies Ltd., a clinical-stage gene therapy company, focuses on developing a pipeline of product candidates for debilitating neurological and neuromuscular diseases. The company's current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis, Parkinson's disease, oculopharyngeal muscular dystrophy, amyotrophic lateral sclerosis, and frontotemporal dementia.

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Axovant Gene Therapies Ltd (NASDAQ:AXGT) Short Interest Update - Tech Know Bits

Regenxbio (NASDAQ:RGNX) was downgraded by Zacks Investment Research from a hold rating to a sell rating in a research note issued on Thursday, Zacks.com reports.

According to Zacks, REGENXBIO Inc. is a biotechnology company. The Company focuses on the development, commercialization and licensing of recombinant adeno-associated virus gene therapy. Its products candidates include RGX-501, for the treatment of homozygous familial hypercholesterolemia which uses the AAV8 vector to deliver the human low-density lipoprotein receptor gene to liver cells; RGX-111, for the treatment of Mucopolysaccharidosis Type I which uses the AAV9 vector to deliver the human a-l-iduronidase gene to the central nervous system; RGX-121, for the treatment of Mucopolysaccharidosis Type II; RGX-314, for the treatment of wet age-related macular degeneration and RGX-321, for the treatment of X-linked retinitis pigmentosa. REGENXBIO Inc. is headquartered in Rockville, Maryland.

A number of other brokerages have also issued reports on RGNX. Svb Leerink upgraded shares of Regenxbio from an underperform rating to a market perform rating and decreased their target price for the stock from $38.00 to $37.00 in a research report on Tuesday, August 20th. ValuEngine upgraded shares of Regenxbio from a buy rating to a strong-buy rating in a research report on Thursday, August 1st. Leerink Swann upgraded shares of Regenxbio from an underperform rating to a market perform rating in a research report on Tuesday, August 20th. They noted that the move was a valuation call. BidaskClub upgraded shares of Regenxbio from a strong sell rating to a sell rating in a research report on Thursday, September 12th. Finally, Raymond James initiated coverage on shares of Regenxbio in a research report on Thursday, June 13th. They set an outperform rating on the stock. Two analysts have rated the stock with a sell rating, three have issued a hold rating, five have assigned a buy rating and one has given a strong buy rating to the company. Regenxbio currently has a consensus rating of Hold and a consensus target price of $79.56.

Regenxbio (NASDAQ:RGNX) last announced its earnings results on Wednesday, August 7th. The biotechnology company reported ($0.04) earnings per share (EPS) for the quarter, topping the Zacks consensus estimate of ($0.50) by $0.46. Regenxbio had a negative net margin of 88.57% and a negative return on equity of 15.39%. The firm had revenue of $7.88 million for the quarter, compared to analyst estimates of $5.45 million. During the same quarter last year, the business earned $0.30 earnings per share. Regenxbios revenue was down 80.3% compared to the same quarter last year. As a group, equities analysts predict that Regenxbio will post -2.85 EPS for the current year.

In other Regenxbio news, insider Kenneth T. Mills sold 15,000 shares of Regenxbio stock in a transaction on Friday, July 19th. The shares were sold at an average price of $49.49, for a total value of $742,350.00. Following the transaction, the insider now owns 261,000 shares in the company, valued at approximately $12,916,890. The sale was disclosed in a document filed with the SEC, which can be accessed through the SEC website. Also, SVP Patrick J. Christmas sold 5,486 shares of the firms stock in a transaction on Thursday, September 12th. The shares were sold at an average price of $42.00, for a total transaction of $230,412.00. Following the completion of the transaction, the senior vice president now directly owns 8,058 shares in the company, valued at approximately $338,436. The disclosure for this sale can be found here. In the last quarter, insiders sold 40,486 shares of company stock worth $1,786,162. 13.80% of the stock is owned by company insiders.

Several hedge funds have recently modified their holdings of the company. BlackRock Inc. raised its stake in shares of Regenxbio by 3.5% in the 2nd quarter. BlackRock Inc. now owns 5,047,103 shares of the biotechnology companys stock valued at $259,270,000 after acquiring an additional 169,888 shares during the period. Royal Bank of Canada raised its stake in shares of Regenxbio by 36.2% in the 2nd quarter. Royal Bank of Canada now owns 3,910 shares of the biotechnology companys stock valued at $201,000 after acquiring an additional 1,040 shares during the period. UBS Group AG raised its stake in shares of Regenxbio by 438.6% in the 2nd quarter. UBS Group AG now owns 263,094 shares of the biotechnology companys stock valued at $13,515,000 after acquiring an additional 214,244 shares during the period. Nuveen Asset Management LLC purchased a new position in shares of Regenxbio in the 2nd quarter valued at $10,206,000. Finally, UBS Asset Management Americas Inc. raised its stake in shares of Regenxbio by 0.6% in the 2nd quarter. UBS Asset Management Americas Inc. now owns 119,714 shares of the biotechnology companys stock valued at $6,150,000 after acquiring an additional 752 shares during the period. 79.40% of the stock is currently owned by institutional investors and hedge funds.

About Regenxbio

REGENXBIO Inc, a clinical-stage biotechnology company, provides gene therapy product candidates to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins or antibodies that are intended to impact disease. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform.

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Regenxbio (NASDAQ:RGNX) Downgraded by Zacks Investment Research to Sell - Mayfield Recorder

The report titled,Global Gene Therapy Market Professional Insight Survey and Forecast to 2028is recently generated by MarketResearch.biz. This research study includes the investigations done of the past progress, present ongoing market scenarios, and future prospects. The report analysis of Gene Therapy incorporates market size & share, upstream circumstance, in detail segmentation outlook, price & cost, and industry environment. It starts from the overview of industry chain structure and depicts the upstream. Besides, the report examinations market size and estimate in different geographies, type, and end-use segment, in addition, the report presents market competition overview among the major organizations and companies profiles, besides, market price and channel highlights are covered in the report.

This statistical surveying research report deals with the present scenario of the market. This investigation report summarizes the technologies, which can help to scale up the growth of the businesses in the near future and obtainable in a clear and professional manner with effective infographics. It clarifies a thorough synopsis of Gene Therapy market dependent on the central parameters. merchandises, End users, provinces and many other subdivisions are planned and elucidated. A transitory idea about the dynamic forces which help make the market more prosperous is deliberated in order to help customer appreciate the future market situation.

Prominent Top Key Vendors of Gene Therapy Report:-Novartis, Kite Pharma Inc, GlaxoSmithKline PLC, Spark Therapeutics Inc, Bluebird bio Inc, Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica PLC, NewLink Genetics Corp., Amgen Inc

Looking for more information on this Research Study? Click Here @https://marketresearch.biz/report/gene-therapy-market/request-sample

Objectives of theGene TherapyStudy:

Identification and measurement of global and enzymatic and reactive universal enzymes based on end-user, product, key application, and geometric region.

To identify the key micro-energy markets and their drivers in the Global Micro Enzymes market and their buyers and reagents, which are expected to provide growth opportunities in the coming years.

Identify market participants and opportunities for market participants and access payment details for major market leaders.

For detailed information about the key factors affecting market growth (drivers, restraints, issues, and opportunities).

For the four main areas, notably, North America (United States, Canada)), Europe (Germany, Spain, France, UK, Russia, and Italy), Asia-Pacific (China, Japan, India, Australia, and South Korea), and the rest (of Latin America and the Middle East & Africa).

To analyze the market structure and the key players involved in the Global Enzymes market and their buyers and reagents in the market and to evaluate in detail their key competencies.

To track and analyze the competitive landscape such as market sharing research and competitive leadership.

For the successful business viewpoint, the market study also examines different worldwide areas, for example, North America, Latin America, Asia-Pacific, Europe, The Middle East, and Africa by considering various segments for example vector type, gene type, application, and region. SWOT and Porters five analysis are also successfully discussed to analyze informative data such as cost, prices, revenue, and end-users. Gene Therapy research report has been evaluated base on various attributes such as manufacturing base, products or services and raw material to comprehend the necessities of the organizations. Apart from the regional outlook, the report also draws attention to leading industry key players to know the successful sales strategies of the businesses. Gene Therapy report can effectively help companies and decision-makers in addressing these challenges strategically to gain the maximum benefits in the highly competitive Gene Therapy Market.

Gene Therapy Market Segmentation byvector type, gene type, application, and region:-

By Vector:Viral vectorRetrovirusesLentivirusesAdenovirusesAdeno Associated VirusHerpes Simplex VirusPoxvirusVaccinia VirusNon-viral vectorNaked/Plasmid VectorsGene GunElectroporationLipofection

By Gene Therapy:AntigenCytokineTumor SuppressorSuicideDeficiencyGrowth factorsReceptorsOther

By Application:Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

Fill Free with your Queries to Get a Call From Our Industry Expert @https://marketresearch.biz/report/gene-therapy-market/#inquiry

Gene Therapy MarketTable Of Content Overview:

1 INTRODUCTION

1.1 Study Deliverables

1.2 Study Assumptions

1.3 Scope of the Study

2 RESEARCH METHODOLOGY

3 EXECUTIVE SUMMARY

4 MARKET DYNAMICS

4.1 Market Overview

4.2 Introduction to Market Drivers & Restraints

4.3 Market Drivers

4.3.1 Rising Adoption of Mega Data Centers

4.3.2 Rising Adoption of Cloud Computing

4.4 Market Restraints

4.4.1 High Initial Investment

4.5 Industry Attractiveness Porters Five Force Analysis

4.5.1 Threat of New Entrants

4.5.2 Bargaining Power of Buyers/Consumers

4.5.3 Bargaining Power of Suppliers

4.5.4 Threat of Substitute Products

4.5.5 Intensity of Competitive Rivalry

5 TECHNOLOGY SNAPSHOT

6 MARKET SEGMENTATION

6.1 By Type

6.1.1 Solutions

6.1.1.1 Power Distribution Unit

6.1.1.2 UPS

6.1.1.3 Busway

6.1.1.4 Other Solutions

6.1.2 Services

6.1.2.1 Consulting

6.1.2.2 System Integration

6.1.2.3 Professional Service

6.2 By End-user Application

6.2.1 Information Technology

6.2.2 Manufacturing

6.2.3 BFSI

6.2.4 Government

6.2.5 Energy

6.2.6 Other End-user Applications

6.3 Geography

6.3.1 North America

6.3.2 Europe

6.3.3 Asia-Pacific

6.3.4 Latin America

6.3.5 the Middle East & Africa

7 COMPETITIVE LANDSCAPE

7.1 Company Profiles

8 INVESTMENT ANALYSIS

9 MARKET OPPORTUNITIES AND FUTURE TRENDS

For Information of Multiple Category related Research Report, Visit @https://marketresearch.biz/categories/

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New Edition 2019-2028: Global Gene Therapy Market Insight Inflation & Massive Growth - Market News Live

Gene therapy is an experimental technique that aims to treat genetic diseases by altering a disease-causing gene or introducing a healthy copy of a mutated gene to the body. The U.S. Food and Drug Administrationapprovedthe first gene therapy for an inherited disease a genetic form of blindness in December 2017.

Sickle cell anemia is caused by a mutation in the HBB gene which provides the instructions to make part of hemoglobin, the protein in red blood cells that carries oxygen.

Researchers are working on two different strategies to treat sickle cell anemia with gene therapy. Both of these strategies involve genetically altering the patients own hematopoietic stem cells. These are cells in the bone marrow that divide and specialize to produce different types of blood cells, including the red blood cells.

One strategy is to remove some of the patients hematopoietic stem cells, replace the mutated HBB gene in these cells with a healthy copy of the gene, and then transplant those cells back into the patient. The healthy copy of the gene is delivered to the cells using a modified, harmless virus. These genetically corrected cells will then hopefully repopulate the bone marrow and produce healthy, rather than sickled, red blood cells.

The other strategy is to genetically alter another gene in the patients hematopoietic stem cells so they boost production of fetal hemoglobin a form of hemoglobin produced by babies from about seven months before birth to about six months after birth. This type of hemoglobin represses sickling of cells in patients with sickle cell anemia, but most people only produce a tiny amount of it after infancy. Researchers aim to increase production of fetal hemoglobin in stem cells by using a highly specific enzyme to cut the cells DNA in the section containing one of the genes that suppress production of fetal hemoglobin. When the cell repairs its DNA, the gene no longer works and more fetal hemoglobin is produced.

Gene therapy offers an advantage over bone marrow transplant, in that complications associated with a bone marrow donation now the only cure for the disease such as finding the right match are not a concern.

Twelve clinical trials studying gene therapy to treat sickle cell anemia are now ongoing. Nine of the 12 are currently recruiting participants.

Four trials (NCT02186418, NCT03282656, NCT02247843, NCT02140554) are testing the efficacy and safety of gene therapy to replace the mutated HBB gene with a healthy HBB gene. These Phase 2 trials are recruiting both children and adults in the United States and Jamaica.

Three trials (NCT02193191, NCT02989701, NCT03226691) are investigating the use ofMozobil (plerixafor) in patients with sickle cell anemia to increase the production of stem cells to be used for gene therapy. This medication is already approved to treat certain types of cancer. All three are recruiting U.S. participants.

One trial (NCT00669305) is recruiting sickle cell anemia patients in Tennessee to donate bone marrow to be used in laboratory research to develop gene therapy techniques.

The final study(NCT00012545) is examining the best way to collect, process and store umbilical cord blood from babies with and without sickle cell anemia. Cord blood contains abundant stem cells that could be used in developing gene therapy for sickle cell anemia. This trial is open to pregnant women in Maryland both those who risk having an infant with sickle cell anemia, and those who do not.

One clinical trial (NCT02151526) conducted in France is still active but no longer recruiting participants. It is investigating the efficacy of gene therapy in seven patients. For the trial, a gene producing a therapeutic hemoglobin that functions similarly to fetal hemoglobin is introduced into the patients stem cells. A case studyfrom one of the seven was published in March 2017; it showed that the approach was safe and could be an effective treatment option for sickle cell anemia.

***

Sickle Cell Anemia News is 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 or other qualified health provider with 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.

Original post:
Gene Therapy - Sickle Cell Anemia News

Because gene therapy involves making changes to the bodys set of basic instructions, it raises many unique ethical concerns. The ethical questions surrounding gene therapy include:

How can good and bad uses of gene therapy be distinguished?

Who decides which traits are normal and which constitute a disability or disorder?

Will the high costs of gene therapy make it available only to the wealthy?

Could the widespread use of gene therapy make society less accepting of people who are different?

Should people be allowed to use gene therapy to enhance basic human traits such as height, intelligence, or athletic ability?

Current gene therapy research has focused on treating individuals by targeting the therapy to body cells such as bone marrow or blood cells. This type of gene therapy cannot be passed to a persons children. Gene therapy could be targeted to egg and sperm cells (germ cells), however, which would allow the inserted gene to be passed to future generations. This approach is known as germline gene therapy.

The idea of germline gene therapy is controversial. While it could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known. Because people who would be affected by germline gene therapy are not yet born, they cant choose whether to have the treatment. Because of these ethical concerns, the U.S. Government does not allow federal funds to be used for research on germline gene therapy in people.

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What are the ethical issues surrounding gene therapy ...

April 30, 2019

Children born with a rare genetic disorder called X-linked severe combined immunodeficiency (X-SCID) dont have a functioning immune system. As a result, they cant fight off infections. Without treatment, an infant with X-SCID will usually die within the first year or two of life.

The best option for treatment of newly diagnosed infants with X-SCID has been stem-cell transplantation from a genetically matched sibling. But less than a quarter of children with X-SCID have a matched donor available. For those without a matched donor, standard treatment has been a half-matched bone marrow transplant from a parent. But most infants receiving this type of transplant only have part of their immune system, called T lymphocytes, restored. These infants will need lifelong injections of protective antibodies. In addition, as they grow into young adulthood, they may have chronic medical problems that affect growth, nutrition, and quality of life.

To develop a better approach to fix the immune systems of children with X-SCID, researchers have used gene therapy to alter patients own blood stem cells. An engineered virus brings a healthy copy of the gene into the stem cells to replace the mutated gene that causes the disease.

Early results from trials of gene therapy for X-SCID resulted in life-saving correction of T lymphocytes. But similar to bone marrow transplant from a parent, the immune restoration was incomplete. In addition, in those first gene therapy studies, almosta third of the children developed leukemia. The approach accidentally stimulated cells to grow uncontrollably. In later studies, improved design of the engineered virus didnt cause cancer, but also didnt fully restore a healthy immune system.

In 2010, Dr. Harry Malech of NIHs National Institute of Allergy and Infectious Diseases (NIAID) and Dr. Brian Sorrentino of St. Jude Childrens Research Hospital reported a new and safer version of gene therapy for X-SCID. They designed a harmless engineered virus (called a lentivector) that could deliver genes into cells without activating other genes that can cause cancer. Before the altered stem cells were returned to their bodies, patients were given low doses of the chemotherapy drug busulfan. This made it easier for the new stem cells to grow in the bone marrow. In young adults and children treated at the NIH Clinical Center, the new therapy proved to be both safe and effective at restoring the full range of immune functions.

Based on this work, a team led by Dr. Ewelina Mamcarz of St. Jude Childrens Research Hospital began treatment in 2015 of newly diagnosed infants with X-SCID using the lentivector and busulfan. The work was funded in part by NHLBI. The team described the treatment of eight infants with the disorder on April 18, 2019, in the New England Journal of Medicine.

By 3 to 4 months after infusion of the repaired stem cells, 7 of the 8 infants had normal levels of multiple types of immune cells in their blood. The last infant required a second stem-cell infusion, after which his immune-cell levels rose to a normal range.

The infants new immune systems were able to fight off infections that the researchers had detected before the gene therapy. Four of the eight discontinued immune-system boosting medications that theyd previously needed. Of those four, three developed antibodies in response to vaccination, indicating a fully functional immune system.

A year and a half after gene therapy, all children were healthy and growing normally.

The broad scope of immune function that our gene therapy approach has restored to infants with X-SCID as well as to older children and young adults in our continuing study at NIH is unprecedented, Malech says.

The researchers will continue to follow the participants over time. They plan to track how the childrens immune systems develop and look for any late side effects.

References:Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1. Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, Ma Z, Condori J, Dowdy J, Triplett B, Li C, Maron G, Aldave Becerra JC, Church JA, Dokmeci E, Love JT, da Matta Ain AC, van der Watt H, Tang X, Janssen W, Ryu BY, De Ravin SS, Weiss MJ, Youngblood B, Long-Boyle JR, Gottschalk S, Meagher MM, Malech HL, Puck JM, Cowan MJ, Sorrentino BP. N Engl J Med. 2019 Apr 18;380(16):1525-1534. doi: 10.1056/NEJMoa1815408. PMID: 30995372.

Funding:NIHs National Institute of Allergy and Infectious Diseases (NIAID); National Heart, Lung, and Blood Institute (NHLBI); and National Cancer Institute (NCI); American Lebanese Syrian Associated Charities; California Institute of Regenerative Medicine; and Assisi Foundation of Memphis.

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Gene Therapy Market Emerging Trends, Growth and New ...

About Conference

About Conference

In continuation to 1st successful past scientific meeting, 2nd EuroSciCon Conference on Cell and Gene Therapy will be held on April 08-09, 2019 on Paris, France.

EuroSciCon suggests every single person to attend "Genetherapy 2019 in the midst of April 08-09, 2019 at Paris, France which merges brief keynote introductions, speaker talks, Exhibitions, Symposia, Workshops.

Genetherapy 2019 will gather world-class educators, researchers, analysts, Molecular Biologist, Gene therapists , Young Researchers working in the related fields to consider, exchange views and their experiences before an extensive worldwide social occasion of individuals. The social gathering warmly welcomes Presidents, CEO's, Delegates and present day experts from the field of Gene therapy and Public wellbeing and other pertinent organization positions to take an interest in these sessions, B2B get together and board talks. The assembly of this event will be revolving around the topic Exploring the possibilities and breakthroughs in cell and gene therapy.

EuroSciCon is the longest running independent life science events company with a predominantly academic client base. Our multi professional and multi-specialty approach creates a unique experience that cannot be found with a specialist society or commercially. EuroSciCon are corporate members of the following organizations: Royal Society of Biology, IBMS Company.

This global meeting gives the chance to Molecular Biologist, Gene Therapists, young researchers, specialists and analysts throughout the world to assemble and take in the most recent advances in the field of Cell and Gene Therapy and to trade innovative thoughts and encounters.

2 days of scientific exchange

100+ abstracts submitted

20+ scientific sessions

50+ worldwide professionals

80+ healthcare experts

Genetherapy 2019 is the yearly gathering directed with the help of the Organizing Committee Members and individuals from the Editorial Board of the supporting cell and Gene therapy related journals.

Reason to attend?

Genetherapy 2019 is relied upon to give young researchers and scientists a platform to present their revolutions in the field of Cell and Gene Therapy. This conference invites Presidents, CEO's, Delegates and present day specialists from the field of Cell and Gene Therapy and Public wellbeing and other pertinent organization positions to take an interest in this sessions, B2B get together and board talks.

About City:

Paris, the world's most popular city destination, has plenty of must-see places but make sure you spend at least a day strolling off the beaten path, as this is the only way to discover the real Paris: a lively cosmopolitan but undeniably French city.

The city is known for its cafe culture and designer boutiques along the Rue du Faubourg Saint-Honor. Paris is the city of love, inspiration, art and fashion. It has a population of more than 2million people and is divided into 20 districts. Paris has a lot of interesting architecture and museums to offer; among them the famous tourist place to visit is the Eiffel Tower. A significant number of the acclaimed roads and city building areas structures where changed by Haussmann and Napoleon III (Charles Louis Napoleon Bonaparte). The lanes where made much wider, places and squares where fabricated and the structures totally modified. Paris has a nickname called La Ville-Lumiere. The famous places to visit in Paris are Notre Dame Cathedralwhich is Roman Catholic Cathedral situated in the eastern half of the city, Louvre Museum which is located at the heart of Paris , Champs Elysees which is a Arc of Triumph, Montmartre which is a hill located at the north of Paris and its height is 130 metre, it is best known White Domed Basilica of the sacred heart at the top, Quartier Latin which is called the famous private garden located on the left bank of the seine around the Sorbonne, Disneyland Paris which is located 32 km from central Paris , it has two theme parks Disneyland and Walt Disney studios.

Track 1: Cell Science Research

Cell Science Research examines cells their physiological properties, their structure, the organelles they contain, interchanges with their condition, their life cycle, division, end and cell work.

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Track 2: Cell & Gene Therapy

Quality treatment is described as a plan of approaches that modify the announcement of a man's characteristics or repair bizarre characteristics. Each system incorporates the association of a specific nucleic destructive (DNA or RNA). Nucleic acids are frequently not taken up by cells, henceforth exceptional transporters; implied 'vectors' are required. Vectors can be of either mainstream or non-viral nature however Cell treatment is portrayed as the association of living whole cells into the patient for the treatment of a disease. The start of the cells can be from a comparable individual (autologous source) or from another individual (allogeneic source). Cells can be gotten from undifferentiated life forms, for instance, bone marrow or induced pluripotent central microorganisms (iPSCs), rethought from skin fibroblasts or adipocytes. Youthful microorganisms are associated with respect to bone marrow transplantation particularly. Distinctive methods incorporate the utilization of basically create cells, isolated in vitro (in a dish) from essential microorganisms.

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Cell and Gene Therapy Conferences|Molecular Biology Conferences|Top Molecular Biology Conferences

Track 3: Regenerative Medicine

Regenerative Medicine implies a social affair of biomedical approaches to manage investigate and clinical applications which are away to supplant or "recouping" human cells, tissues or organs to restore or set up conventional limits which were vexed on account of afflictions. The field of Regenerative medication has pulled in much thought as it holds the assurance of recuperating hurt tissues and organs in the body by supplanting hurt tissue or by strengthening the body's own repair segments to patch hurt tissues or organs. It in like manner may enable analysts to create tissues and organs in the lab and safely install them inside the body. Regenerative courses of action subsequently can be a dynamic progress in the field of therapeutic administrations.

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Track 4: Immunotherapy

Due to rapidly pushing field of tumor immunology as of late, there has been age of a couple of new procedures for treating development called Immunotherapies. Immunotherapy is a sort of treatment that extends the nature of safe response against tumors either by enabling the activities of specific sections of safe structure or by checking signals conveyed by illness cells that cover safe responses. A couple of sorts of immunotherapy are also called as biologic treatment or biotherapy. Late movements in development immunotherapies have given new supportive systems. These consolidate tumor-related macrophages as treatment centers in oncology, in-situ commencement of platelets with checkpoint inhibitors for post-careful development immunotherapy, safe checkpoint blockade and related endocrinopathies and some more.

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Track 5 : Genetics and stem cell biology

An undifferentiated mass of cell in a multicellular animal which is prepared for offering rise to uncertain number of cells of a comparable sort, and from which certain diverse sorts of cell rise by detachment. Undifferentiated life forms can isolate into specific cell creates. The two describing characteristics of an undifferentiated cell are endless self-restoration and the ability to isolate into a specific adult. There are two critical classes of youthful microorganisms: pluripotent that can end up being any cell in the adult body, and multipotent that is kept to transforming into a more limited masses of cells.

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Track 6: Epigenetics

The examination of changes in living creatures caused by alteration of quality verbalization instead of adjustment of the inherited code itself. Epigenetics are unfaltering heritable characteristics that can't be cleared up by changes in DNA progression.

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Track 7: Human Genomics

The human genome is the total arrangement of nucleic corrosive groupings for people, encoded as DNA inside the 23 chromosome combines in cell cores and in a little DNA particle found inside individual mitochondria. Human genomes incorporate both protein-coding DNA genes and noncoding DNA

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Track 8: Next Generation Sequencing

Deoxyribonucleic destructive, for the most part known as DNA, contains the outlines of life. Inside its structures are the codes required for the party of proteins and non-coding RNA these sub-nuclear mechanical assemblies impact all the natural systems that make and care forever. By understanding the game plan of DNA, examiners have had the ability to outline the structure and limit of proteins and what's more RNA and have gotten a cognizance of the essential purposes behind ailment. Front line Sequencing (NGS) is an able stage that has enabled the sequencing of thousands to countless iotas in the meantime. This compelling device is evolving fields, for instance, redid medicine, inherited infections, and clinical diagnostics by offering a high throughput elective with the capacity to progression various individuals meanwhile.

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Track 9: Gene Editing and CRISPR Based Technologies

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) Technology is a champion among the most fit yet clear mechanical assembly for genome changing. It urges and empowers investigators to easily change DNA groupings and modify quality limits. It has various potential applications that join helping innate disseminates, treating and keeping the spread of diseases and improving yields. CRISPR broadly used as CRISPR-Cas9 where CRISPRs are particular stretches out of DNA and Cas9 is the protein which is an aggravate that exhibitions like a few nuclear scissors, fit for cutting DNA strands. The assurance of CRISPR advancement anyway raises moral stresses as it isn't 100% compelling. Regardless, the progression of CRISPR-Cas9 has disturbed the designed science industry these days, being a clear and great quality changing device.

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Track 10: Proteomics

Proteomics is the broad scale examination of proteomes. A proteome is a course of arrangement of proteins made in a living being, structure, or regular setting. We may imply, for instance, the proteome of a creature composes (for example, Homo sapiens) or an organ (for example, the liver). The proteome isn't relentless; it fluctuates from cell to cell and changes after some time. To some degree, the proteome reflects the key transcriptome. Regardless, protein activity (regularly reviewed by the reaction rate of the systems in which the protein is incorporated) is similarly changed by various components despite the verbalization level of the appropriate quality.

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International Proteomics Conferences|Top Microbiology Conferences|Cell Microbiology Conferences

Track 11: Viral Gene Therapy

Customary strategies for quality treatment fuse transfection. It twisted up clearly inefficient and confined fundamentally in view of movement of value into right now duplicating cells invitro. Quality treatment utilizes the transport of DNA into cells by techniques for vectors, for instance, natural nanoparticles or viral vectors and non-viral systems. The Several sorts of contaminations vectors used as a piece of value treatment are retrovirus, adenovirus, disease adeno-related and herpes simplex contamination. While other recombinant viral vector structures have been delivered, retroviral vectors remain the most surely understood vector system for quality treatment traditions and most prominent application on account of their certain significance as the essential vectors made for compelling quality treatment application and the soonest phases of the field of value treatment.

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Track 12: Cell Therapy of Cardiovascular Disorders

Cardiovascular contaminations have transformed into a growing clinical issue all around. the other test in the treatment of the cardiovascular disease is cell transplantation or cell cardiomyoplasty. Exceptional ischaemic harm and relentless cardiomyopathies incite unending loss of cardiovascular tissue and in the end heart disillusionment. Force medications wide mean to tighten the over the top changes that happen when harm and to cut back shot segments of vas diseases. Regardless, they don't improve the patient's close to home fulfillment or the figure more than coordinate. Unmistakable sorts of undifferentiated living beings have been used for primary microorganism treatment.

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Track 13: Regulatory and Safety Aspects of Cell and Gene Therapy

Cell treatment things require a combination of prosperity examinations. Comparable living being and quality things are heterogeneous substances. There are a few zones that particularly ought to be tended to as it is extremely not the same as that of pharmaceuticals. These range from making group consistency, thing soundness to thing prosperity, quality and sufficiency through pre-clinical, clinical examinations and displaying endorsement. This review plots the present headings/administers in US, EU, India, and the related challenges in making SCBP with highlight on clinical point.

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Track 14: Markets & Future Prospects for Cell & Gene Therapy

The immense number of associations related with cell treatment has extended development incredibly in the midst of the past couple of years. More than 500 associations have been recognized to be locked in with cell treatment and 305 of these are profiled 291 co-tasks. Of these associations, 170 are related with fundamental microorganisms. The Profiles of 72 academic establishments in the US related with cell treatment close by their business facilitated efforts. Allogeneic development with in excess of 350 clinical preliminaries is prepared to order the commercialization of cell medicines in publicize. Advance R&D in cell and quality treatment is depended upon to bloom given the normally based purposes of intrigue.

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Track 15: Gene therapy for Diseases

Gene therapy is the addition of particular genes at some particular locales into a person's cells or tissues to treat an illness, in which the inadequate or non-working quality is then supplanted with the working quality.

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Gene therpy Conferences|Cell and Gene Therapy Conferences|International cell and Gene therapy Conferences|Top Gene Therapy research conferences

Track 16: Stem Cell therapy

Stem Cell therapyis the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use. Recent studies are going on for the treatment ofSpinal cordinjury as well. Thus,Stem cell therapyhas a great scope in future as well.

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Stem Cell Biology Conferences|Genetic Cell Biology Conferences|Top Stem Cell Biology Conferences|International Stem Cell Therapy Conferences

Track 17: Gene Editing

Gene Editing is where the defective gene is being expelled or supplanted from the genome, in order to change the imperfect type of quality to a working structure. Different methods, for example, gene substitution, gene knock out, gene knock down are utilized for this reason. Additionally, site coordinated mutagenesis has been broadly utilized for gene altering purposes.

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International Cell and Gene therapy Conferences | Gene ...

Michael Passineau, PhD, is a man who speaks in metaphors. For good reason he works within a realm of medicine not many people understand. Director of the Gene Therapy Program at Allegheny Health Network (AHN) and a leading force behind AHNs scientific research to address clinical needs, Passineau leans into the way a good metaphor can bring clarity to challenging conceptsincluding the nature of his work.

I think of clinicians as chefs, he says. At the end of each day, theyve done something tangible. Theyve made a meal. Researchers, on the other hand, are a bit like sculptors. We can work for years on something, but once its complete, its permanent.

For over a decade, his research has revolved around gene therapy more specifically, the use of ultrasound technology, instead of viral administration, to deliver therapeutic DNA into the cells of salivary glands. The goal: restore saliva flow to patients who suffer from radiation-induced dry mouth, or xerostomia.

Supported by grants from the National Institutes of Health (NIH), Passineau, radiation oncologist Dr. Mark Trombetta, and their research team are on track to petition the U.S Food and Drug Administration for Investigational New Drug (IND) status, which would allow their work to move out of the lab and into Phase 1 clinical trials with humans.

Xerostomia is an iatrogenic complication, meaning it is caused by treatment in this case, head and neck radiation to treat cancer. When the beam of radiation passes through the head, it damages the salivary glands, resulting in chronic dry mouth. This can lead to permanent loss of function in the salivary glands, difficulty eating, and loss of teeth.

Its anything but trivial, says Passineau. To illustrate how this condition impacts a persons quality of life, I often have donors and executives take a piece of surgical gauze and chew on it while I describe my research. After about five minutes, they understand how difficult it really is.

While there are a few existing medications used to treat xerostomia, they are difficult to administer, and their effects are not long lasting. Most people deal with the condition by carrying a water bottle at all times or by taking saliva substitutes. Unfortunately, these options dont work particularly well.

Were all made of trillions of cells, says Passineau, beginning an attempt to explain gene therapy in a nutshell.

Each cell has a role to play, whether its beating heart muscle, growing hair and nails, or perceiving light signals in your retina, he continues. The way those biochemical machines are engineered is dictated by your genomic DNA, which is DNA you get from your parents. Those genes code for proteins, which are the gears and springs that make cells function as biochemical machines.

Gene therapy means reprogramming the cell changing the machine code telling the cell how to work. That requires getting new DNA to travel inside the cell. Passineau says this is one of the most difficult tasks in the world since our cells are designed to repel foreign DNA.

In nature, foreign DNA gets into human cells only through viral infection or during conception. Virally administered gene therapy approaches have been developed, but one drawback is that after a viral vector is introduced into the body, the immune system fights back and will also react to the vector on subsequent treatments, making them ineffective.

Thats where Passineaus research comes in. Weve developed a method of delivering genes that doesnt require viral administration, he explains. Instead, we use soundwaves.

To understand how ultrasonic administration, or sonoporation, works, Passineau turns again to metaphor.

Picture an agricultural pond, with a thick layer of algae on it. If you throw a ping-pong ball into the middle, it will just sit on top, he says. That is very much what a cell membrane is like the outer covering is rather rigid. So, to deliver the genes, we have to get through the cell membrane. It is only seven nanometers thick but its the longest seven nanometers in nature for someone like me.

Passineaus ground-breaking research uses soundwaves to temporarily alter the permeability of the cell membrane, allowing for the transfer of therapeutic DNA into the cell.

Lets understand how this works in our pond metaphor before getting into what that means for gene therapy.

Imagine we explode a grenade above the pond, Passineau says. For a moment, the layer of slime would open up, and youd see down to the bottom of the pond. Then, it would close again.

In Passineaus lab, the grenade is a mix of a gene drug for xerostomia known as Aquaporin-1 and a solution of microbubbles. Used routinely in cardiac imaging and other medical applications, microbubbles have a resonant frequency that can be used to create the desired explosion.

The classic example is a crystal glass if an opera singer hits the right frequency for that glass, it will vibrate. If she really turns up the volume, the glass will shatter, because it cant absorb the energy, Passineau says. That same thing happens with the microbubbles.

So after administering the microbubble and Aquaporin-1 solution to the treatment site, a low-frequency ultrasound beam is used to create an ultrasonic acoustic field in which the bubbles vibrate. Turn up the power, and the bubbles implode. That opens up the cell membrane long enough for the gene drug to get in, before it closes back up.

For gene therapy researchers like Passineau, the membrane around our cells is the longest seven nanometers in nature.

Sonoporation works well for what were doing in the salivary glands, but not so well for the heart, and certainly not for the brain, Passineau points out. However, we have other applications we intend to use this research for.

He explains that one promising use involves Sjogrens syndrome, an autoimmune disease affecting nearly 4 million Americans (90 percent of whom are women). The diseases debilitating symptoms include severe dry mouth, which may be treatable with Passineaus gene therapy technique.

Another research area he says he is excited about is the use of gene therapy to combat obesity and overeating. Do you remember when you were a kid and youd eat too fast and your mom would tell you to slow down because your brain didnt know whether or not your stomach was full yet? he asks. Well, that was absolutely true.

He explains that, when we eat, our intestines stretch and release a protein called peptide YY (PYY), which circulates through the blood, eventually entering the saliva and interacting with receptors on your tongue.

Think of your appetite as a glass of water, he says. To feel full, you have to fill the glass with PYY. Some people have bigger glasses than others, but if we can use gene therapy to modify saliva and make the glass start half full, then a person would feel full without needing to eat as much.

Passineau adds that poor health outcomes and high costs associated with obesity make this an attractive target for research investment. Obesity adds billions of dollars to the cost of medical care in the U.S. each year, and some studies estimate the cost as high as $190 billion per year.

If gene therapy was this easy, everyone would be doing it. Instead, as Passineau points out, it is one of the most difficult tasks in the world.

At AHN, research is a small but important piece of the operation, Passineau says. Its important to note that everything we do in research is driven by physicians who have recognized clinical needs, and who have partnered with us to develop novel solutions.

Similarly, looking at the value that research can deliver, and its potential impact on both health and overall health care costs, Passineau says that federal funding has an essential role in advancing further discoveries in areas like gene therapy and sonoporation. Government investment really is the lifeblood of what drives research, he says.

Another impact on the success and pace of advances in medical research is whether talented, driven young people decide to take this path. Passineau admits that, like the process of research itself, the path to becoming a successful researcher can be long and sometimes feels like three steps forward, two steps back. But if the work feels meaningful, its all worth it.

I landed where I am today because I figured out what I was good at, he says. Inventing, solving big picture problems and helping people.

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Highmark Health Blog | Gene Therapy Research: Dr. Passineau

News Release

Wednesday, April 17, 2019

NIH scientists and funding contributed to development of experimental treatment

A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening inherited disorder in which infection-fighting immune cells do not develop or function normally. Eight infants with the disorder, called X-linked severe combined immunodeficiency (X-SCID), received an experimental gene therapy co-developed by National Institutes of Health scientists. They experienced substantial improvements in immune system function and were growing normally up to two years after treatment. The new approach appears safer and more effective than previously tested gene-therapy strategies for X-SCID.

These interim results from the clinical trial, supported in part by NIH, were published today in The New England Journal of Medicine.

Infants with X-SCID, caused by mutations in the IL2RG gene, are highly susceptible to severe infections. If untreated, the disease is fatal, usually within the first year or two of life. Infants with X-SCID typically are treated with transplants of blood-forming stem cells, ideally from a genetically matched sibling. However, less than 20% of infants with the disease have such a donor. Those without a matched sibling typically receive transplants from a parent or other donor, which are lifesaving, but often only partially restore immunity. These patients require lifelong treatment and may continue to experience complex medical problems, including chronic infections.

"A diagnosis of X-linked severe combined immunodeficiency can be traumatic for families," said Anthony S. Fauci, M.D., director of NIHs National Institute of Allergy and Infectious Diseases (NIAID). These exciting new results suggest that gene therapy may be an effective treatment option for infants with this extremely serious condition, particularly those who lack an optimal donor for stem cell transplant. This advance offers them the hope of developing a wholly functional immune system and the chance to live a full, healthy life.

To restore immune function to those with X-SCID, scientists at NIAID and St. Jude Childrens Research Hospital in Memphis, Tennessee, developed an experimental gene therapy that involves inserting a normal copy of the IL2RG gene into the patients own blood-forming stem cells. The Phase 1/2 trial reported today enrolled eight infants aged 2 to 14 months who were newly diagnosed with X-SCID and lacked a genetically matched sibling donor. The study was conducted at St. Jude and the Benioff Childrens Hospital of the University of California, San Francisco. Encouraging early results from a separate NIAID-led study at the NIH Clinical Center informed the design of the study in infants. The NIH study is evaluating the gene therapy in older children and young adults with X-SCID who previously had received stem cell transplants.

The gene therapy approach involves first obtaining blood-forming stem cells from a patients bone marrow. Then, an engineered lentivirus that cannot cause illness is used as a carrier, or vector, to deliver the normal IL2RGgene to the cells. Finally, the stem cells are infused back into the patient, who has received a low dose of the chemotherapy medication busulfan to help the genetically corrected stem cells establish themselves in the bone marrow and begin producing new blood cells.

Normal numbers of multiple types of immune cells, including T cells, B cells and natural killer (NK) cells, developed within three to four months after gene therapy in seven of the eight infants. While the eighth participant initially had low numbers of T cells, the numbers greatly increased following a second infusion of the genetically modified stem cells. Viral and bacterial infections that participants had prior to treatment resolved afterwards. The experimental gene therapy was safe overall, according to the researchers, although some participants experienced expected side effects such as a low platelet count following chemotherapy.

"The broad scope of immune function that our gene therapy approach has restored to infants with X-SCID as well as to older children and young adults in our study at NIH is unprecedented," said Harry Malech, M.D., chief of the Genetic Immunotherapy Section in NIAIDs Laboratory of Clinical Immunology and Microbiology. Dr. Malech co-led the development of the lentiviral gene therapy approach with St. Judes Brian Sorrentino, M.D., who died in late 2018. These encouraging results would not have been possible without the efforts of my good friend and collaborator, the late Brian Sorrentino, who was instrumental in developing this treatment and bringing it into clinical trials, said Dr. Malech.

Compared with previously tested gene-therapy strategies for X-SCID, which used other vectors and chemotherapy regimens, the current approach appears safer and more effective. In these earlier studies, gene therapy restored T cell function but did not fully restore the function of other key immune cells, including B cells and NK cells. In the current study, not only did participants develop NK cells and B cells, but four infants were able to discontinue treatment with intravenous immunoglobulins infusions of antibodies to boost immunity. Three of the four developed antibody responses to childhood vaccinations an indication of robust B-cell function.

Moreover, some participants in certain early gene therapy studies later developed leukemia, which scientists suspect was because the vector activated genes that control cell growth. The lentiviral vector used in the study reported today is designed to avoid this outcome.

Researchers are continuing to monitor the infants who received the lentiviral gene therapy to evaluate the durability of immune reconstitution and assess potential long-term side effects of the treatment. They also are enrolling additional infants into the trial. The companion NIH trial evaluating the gene therapy in older children and young adults also is continuing to enroll participants.

The gene therapy trial in infants is funded by the American Lebanese Syrian Associated Charities (ALSAC), and by grants from the California Institute of Regenerative Medicine and the National Heart, Lung, and Blood Institute, part of NIH, under award number HL053749. The work also is supported by NIAID under award numbers AI00988 and AI082973, and by the Assisi Foundation of Memphis. More information about the trial in infants is available on ClinicalTrials.gov using identifier NCT01512888. More information about the companion trial evaluating the treatment in older children and young adults is available using ClinicalTrials.gov identifier NCT01306019.

NIAID conducts and supports research at NIH, throughout the United States, and worldwide to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses. News releases, fact sheets and other NIAID-related materials are available on the NIAID website.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

E Mamcarz et al. Lentiviral gene therapy with low dose busulfan for infants with X-SCID. The New England Journal of Medicine DOI: 10.1056/NEJMoa1815408 (2019).

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Gene therapy restores immunity in infants with rare ...

Researchers from St. Jude Childrens Research Hospital have cured babies with bubble boy disease through gene therapy. Angela Gosnell, Knoxville News Sentinel

MEMPHIS, Tenn. Researchers from St. Jude Childrens Research Hospital have cured babies with bubble boy disease through gene therapy involving a re-engineeredvirus, according to a newly published study.

St. Jude performed the therapy oninfants newly diagnosed withX-linked severe combined immunodeficiency (SCID-X1) a genetic condition also known as "bubble boy" disease according to a study published in the New England Journal of Medicine's April 18 issue.

The diseaseprevents babies from developing an immune system to fight even routine infections.In January 2018, St. Jude researchers reported that babies in the trial developed fully functioning immune systems but would be monitored further to confirm its long-term benefits.

Corresponding authors Dr. Ewelina Mamcarz and Dr. Stephen Gottschalk from St. Jude Children's Research Hospital. St. Jude performed a new therapy oninfants newly diagnosed withX-linked severe combined immunodeficiency (SCID-X1), a genetic condition called "bubble boy" disease, according to a study published in the New England Journal of Medicine's April 18 issue.(Photo: Peter Barta / St. Jude Childrens Research Hospital)

Previous infections cleared in all infants, and all continued to grow normally, the study said of the results.

St. Jude and UCSF Benioff Childrens Hospital San Francisco treated the children enrolled in the clinical trial with gene therapy developed by St. Judes Brian Sorrentino, the studys senior author,who led groundbreaking gene therapy research before his death in November at 60 years old.

Brian Sorrentino(Photo: Courtesy of Memorial Park Funeral Home)

James Downing, CEO of St. Jude Children's Research Hospital, said it was the lifelong ambition of Sorrentino, a survivor of pediatric cancer, to develop a cure.

Were comfortable, I think, at this point stating this is a cure, Downing said. Only time will say this will be a durable, lifelong cure.

After the therapy, the babies received their standard vaccinations and are now living a normal life with fully functioning immune systems, St. Jude says. Ten infants have received the therapy so far.

Study co-author Stephen Gottschalk, chair of the St. Jude Department of Bone Marrow Transplantation and Cellular Therapy, said the researchers hope the therapy will be a template for treating other blood disorders.

Newborns with bubble boy disease, caused by a mutation inside a specific gene,must be placed inprotective isolation because they lack a proper immune system. Contact with the outside world is a major infection risk.

Perhaps the most well-known person with the disease was David Vetter, who died in 1984 at 12 years old. He helped inspire the 1976 movie "The Boy in the Plastic Bubble."

David Vetter had to stay inside a bubble in Houston on Dec. 17, 1976. Vetter was born with a genetic disorder leaving him no natural immunity against disease. Vetter died in 1984.(Photo: AP)

Most with the disease die by age 2 without treatment.

This disease is called bubble boy disease because babies had to be kept in special plastic chambers to protect them from infections, said first and corresponding author Ewelina Mamcarzof the St. Jude Department of Bone Marrow Transplantation and Cellular Therapy. We dont have these chambers now, we are more advanced, but we need to protect them from infections as simple as a common cold virus (that) can kill them.

The patients came to researchers between 2 and 14 months of age, Mamcarz said, with severe life-threatening infections.

The gene therapy works like this: A deactivated virus is inserted into the patients bone marrow, which deliversthe correct gene copy into blood stem cells, replacing the defective one. These cells are then frozen and undergo testing.

This virus is able to effectively deliver a healthy copy of the gene into a stem cell in a way that was not possible before, Mamcarz said.

The patient then receives two days of low-dose busulfan, a chemotherapy drug that makes space in the marrow for the stem cells to grow, and the cells are then infused back into the patient.

Dr. Ewelina Mamcarz, first and corresponding author of a study published in the New England Journal of Medicine about a therapy performed at St. Jude Children's Research Hospital oninfants newly diagnosed withX-linked severe combined immunodeficiency (SCID-X1), a genetic condition called "bubble boy" disease.(Photo: Peter Barta / St. Jude Childrens Research Hospital)

It takes about 10 days from the time the cells are taken outto when they are infused into the patient, Mamcarz said.

The proper immune cells were found within three months of the treatment in all but one patient, who needed a second dose of gene therapy, St. Jude says.

This novel approach has shown really outstanding results for the infants, Downing said. The treatment has fully restored the immune system in these patients, which wasnt possible before, and has no immediate side effects.

The gene therapy developed and produced at St. Jude differs from previous gene replacement efforts in part by not activating adjacent genes that could cause leukemia. The viruses are equipped with insulators to block that accidental activation.

Past gene therapy did not have insulators, which inadvertently caused leukemia, Gottschalk said.

Gael Jesus Pino Alva, 2, and his mother, Giannina Alva. Gael was treated with a new therapy designed to fight X-linked severe combined immunodeficiency (SCID-X1), a genetic condition known as "bubble boy" disease, at St. Jude Children's Research Hospital.(Photo: Peter Barta / St. Jude Childrens Research Hospital)

Current treatments for bubble boydisease are limited. Bone marrow transplants from compatible sibling donors are the best bet, but most patientslack a properdonor.

Mamcarz said researchers would like to treat more patients and follow them for longer periods of time to see if the gene therapy performed in the clinicaltrial can truly be used as an upfront treatment, and it's still too early to determine costs.

But the results from the research are a first, and their approach could be used to eventually treatother disorders like sickle cell disease, she said.

The kids are cured because for the first time, we are able to restore all three types of cells that constitute a full immune system: T cells, B cells and NK cells, Mamcarz said. Our patients are able to generate a healthy, fully functioning immune system. That is the first for gene therapy.

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Downingsaid the search for a cure has been a journey spanning more than a decade. Early gene therapy studies with the viral vectorsled to leukemia, he said, causing the work to stall. But Sorrentino pushed on.

Brian Sorrentino decided we really needed to produce vectors we could trust in not inducing leukemia, Downingsaid.

The patients' quality of life following the treatmentshows theyindeed found a cure, Downing said.

The question will become, Will it be a durable cure? Will it last 10, 20, 50 years for these children? And only time will tell," he said.

Follow Max Garland on Twitter:@MaxGarlandTypes.

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WiseGuyReports.Com Publish a New Market Research Report On Gene Therapy 2019 Global Market Outlook,Research,Trends and Forecast to 2026.

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Gene Therapy Industry 2019

Description:-

The global gene therapy market is anticipated to reach USD 4,300 million by 2021. The demand for gene therapy is primarily driven by continuous technological advancements and successful progression of several clinical trials targeting treatments with strong unmet need. Moreover, rising R&D spend on platform technologies by large and emerging biopharmaceutical companies and favorable regulatory environment will accelerate the clinical development and the commercial approval of gene therapies in the foreseeable future. Despite promise, the high cost of gene therapy represents a significant challenge for commercial adoption in the forecast period.

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Gene therapy involves inactivating a mutated gene that is not functioning properly and introducing a new gene to assist in fighting a disease. Overall, the field of gene therapy continues to mature and advance with many products in development and nearing commercialization. For instance, Spark Therapeutics received approval of Luxturna, a rare form inherited blindness in December 2017. Gene therapy market in late 2017 also witnessed the approvals of Gilead/Kite Pharmas Yescarta and Novartis Kymriah in the cancer therapeutic area.

Gene therapy offers promise in the treatment of range of indications in cancer and genetic disorders. Large Pharmaceuticals and Biotechnology companies exhibit strong interest in this field and key among them include Allergan, Shire, Biomarin, Pfizer and GSK. The gene therapy space is witnessing a wave of partnerships and alliances. Pfizer has recently expanded its presence in gene therapy with the acquisition of Bamboo Therapeutics and Allergan entered the field, with the acquisition of RetroSense and its Phase I/II optogenetic program.

North America holds a dominating position in the global gene therapy market which is followed by Europe and the Asia Pacific. The U.S. has maximum number of clinical trials ongoing followed by Europe. Moreover, the field of gene therapy in the U.S. and Europe continues to gain investor attention driven by success of high visible clinical programs and the potential of gene therapy to address strong unmet need with meaningful commercial opportunity. Moreover, the increasing partnerships and alliances and the disruptive potential of gene therapy bodes well for the sector through the forecast period.Key Findings from the study suggest products accessible in the market are much competitive and manufacturers are progressively concentrating on advancements to pick up an aggressive edge. Companies are in a stage of development of new items in order to guarantee simple implementation and connection with the current gene. The hospatility segment is anticipated to grow at a high growth rate over the forecast period with the expanding utilization of smart locks inferable from expanding security-related worries among clients amid their stay at the hotels. North America is presumed to dominate the global smart locks market over the forecast years and Asia Pacific region shows signs of high growth owing to the booming economies of India, and China.

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Table Of Contents Major Key Points

1. Gene Therapy Overview1.1. History and Evolution of Gene Therapies1.2. What is Gene Therapy1.3. Types of Gene Therapy1.4. Ex vivo and in vivo Approaches of Gene Therapy1.5. RNAi Therapeutics1.6. CAR-T Technology based Gene Therapy1.7. Types of Vectors used for Gene Therapy1.7.1. Viral1.7.2. Non-Viral

2. Historical Marketed Gene Therapies [2003-2012]2.1. Rexin-G (Epeius Biotechnologies Corporation)2.2. Gendicine (SiBiono GeneTech Co., Ltd)2.3. Neovasculgen [Human Stem Cells Institute (HSCI))2.4. Glybera (UniQure Biopharma B.V.)

3. First Countries to get an access to Gene Therapies3.1. Philippines for Rexin-G [2003]3.2. China for Gendicine [2003]3.3. Russia for Neovasculgen [2011]3.4. Selected European Countries for Glybera [2012]

4. Marketed Gene Therapies [Approved in Recent Years]4.1. KYMRIAH (tisagenlecleucel)4.1.1. Therapy Description4.1.2. Therapy Profile4.1.2.1. Company4.1.2.2. Approval Date4.1.2.3. Mechanism of Action4.1.2.4. Researched Indication4.1.2.5. Vector Used4.1.2.6. Vector Type4.1.2.7. Technology4.1.2.8. Others Development Activities4.1.3. KYMRIAH Revenue Forecasted till 20214.2. YESCARTA (axicabtagene ciloleucel)4.2.1. Therapy Description4.2.2. Therapy Profile4.2.2.1. Company4.2.2.2. Approval Date4.2.2.3. Mechanism of Action4.2.2.4. Researched Indication4.2.2.5. Vector Used4.2.2.6. Vector Type4.2.2.7. Technology4.2.2.8. Others Development Activities4.2.3. YESCARTA Revenue Forecasted till 20214.3. LUXTURNA (voretigene neparvovec-rzyl)4.3.1. Therapy Description4.3.2. Therapy Profile4.3.2.1. Company4.3.2.2. Approval Date4.3.2.3. Mechanism of Action4.3.2.4. Researched Indication4.3.2.5. Vector Used4.3.2.6. Vector Type4.3.2.7. Technology4.3.2.8. Others Development Activities4.3.3. LUXTURNA Revenue Forecasted till 20214.4. STRIMVELIS4.4.1. Therapy Description4.4.2. Therapy Profile4.4.2.1. Company4.4.2.2. Approval Date4.4.2.3. Mechanism of Action4.4.2.4. Researched Indication4.4.2.5. Vector Used4.4.2.6. Vector Type4.4.2.7. Technology4.4.2.8. Others Development Activities4.4.3. STRIMVELIS Revenue Forecasted till 2021

5. Comparison of current Regulatory Status for Gene Therapy Products5.1. U.S5.2. Europe5.3. Japan

6. Emerging Gene Therapies [Phase III]6.1. Gene Based Therapeutics under Development6.2. Therapy Description

7. Indication of Focus in Gene Therapy7.1. Cancer7.2. Neurodegenerative Disorders7.3. Lysosomal Storage Disorders (LSDs)7.4. Ocular Diseases7.5. Muscle Disorders7.6. Anemia7.7. Hemophilia7.8. Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency

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Gene Therapy 2019 Global Market Outlook,Research,Trends ...

Apr 12, 2019 (The Expresswire via COMTEX) -- Hemophilia Gene Therapy Market report is a complete study of current trends in the Market, industry growth drivers, and restraints. It provides Market forecasts for the coming years

Global Hemophilia Gene Therapy Market report observes different predilections, obstructions, and difficulties looked by the best makers/Economy by Business Leaders contenders of Complete Reports Hemophilia Gene Therapy market. Our specialists' group has considered every single angle directly from the piece of the pie, size, status, and development.

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Synopsis of the Hemophilia Gene Therapy Market: -

Hemophilia Gene Therapymarket competition by top manufacturers/ Key player/ Economy by Business Leaders: Spark Therapeutics, Ultragenyx, Shire PLC, Sangamo Therapeutics, Bioverativ, BioMarin, uniQure, Freeline Therapeutics,. And More

Hemophilia is a rare bleeding disorder in which the blood does not clot normally. Hemophilia is a monogenic disease (a disease that is caused by a genetic defect in a single gene). There are two types of hemophilia caused by mutations in genes that encode protein factors which help the blood clot and stop bleeding when blood vessels are injured. Individuals with hemophilia experience bleeding episodes after injuries and spontaneous bleeding episodes that often lead to joint disease such as arthritis. The most frequent forms of hemophilia affect males.

Hemophilia Gene Therapy Market Segment by Type covers:

Hemophilia Gene Therapy Market Segment by Applications can be divided into:

Scope of theHemophilia Gene Therapy MarketReport:

Hemophilia Gene Therapy Market Segment by Regions, regional analysis covers

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The next part also sheds light on the gap between supply and consumption. Apart from the mentioned information,growth rateof Hemophilia Gene Therapy market in 2024is also explained.Additionally, type wise and application wise consumptiontables andfiguresof Hemophilia Gene Therapy marketare also given.

Table of Contents

Market Overview 1.1 Hemophilia Gene Therapy Introduction 1.2 Market Analysis by Type 1.3 Market Analysis by Applications 1.4 Market Analysis by Regions 1.4.1 North America (United States, Canada and Mexico) 1.4.1.1 United States Market States and Outlook (2013-2023) 1.4.1.2 Canada Market States and Outlook (2013-2023) 1.4.1.3 Mexico Market States and Outlook (2013-2023) 1.4.2 Europe (Germany, France, UK, Russia and Italy) 1.4.2.1 Germany Market States and Outlook (2013-2023) 1.4.2.2 France Market States and Outlook (2013-2023) 1.4.2.3 UK Market States and Outlook (2013-2023) 1.4.2.4 Russia Market States and Outlook (2013-2023) 1.4.2.5 Italy Market States and Outlook (2013-2023) 1.4.3 Asia-Pacific (China, Japan, Korea, India and Southeast Asia) 1.4.3.1 China Market States and Outlook (2013-2023) 1.4.3.2 Japan Market States and Outlook (2013-2023) 1.4.3.3 Korea Market States and Outlook (2013-2023) 1.4.3.4 India Market States and Outlook (2013-2023) 1.4.3.5 Southeast Asia Market States and Outlook (2013-2023) 1.4.4 South America, Middle East and Africa 1.4.4.1 Brazil Market States and Outlook (2013-2023) 1.4.4.2 Egypt Market States and Outlook (2013-2023) 1.4.4.3 Saudi Arabia Market States and Outlook (2013-2023) 1.4.4.4 South Africa Market States and Outlook (2013-2023) 1.4.4.5 Nigeria Market States and Outlook (2013-2023) 1.5 Market Dynamics 1.5.1 Market Opportunities 1.5.2 Market Risk 1.5.3 Market Driving Force 2 Manufacturers Profiles

3 Global Hemophilia Gene Therapy Market Analysis by Regions

4 Global Hemophilia Gene Therapy Market Competition, by Manufacturer

5 Sales Channel, Distributors, Traders and Dealers

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