Gene Therapy Market Highlights

Global Gene Therapy Market is expected to register a CAGR of 40.7% during the forecast period of 2019 to 2025 and was valued at USD 524 million in 2018. Gene therapy is a technique in which a piece of DNA is incorporated into the cell via a vector. The key players are investing in the manufacturing of cell and gene therapy treatments due to its target specificity. The gene therapy is mainly of two types, somatic gene therapy, and germline gene therapy, depending on the type of cells its targets.

The growth of the global gene therapy market is attributed to various factors such as robust product pipeline, increasing investment by key players, and high prevalence of target diseases and demand for innovative medication. However, the high cost of product development and stringent regulatory policies are expected to curb the growth of the global gene therapy market. The global gene therapy market is currently dominated by several market players. The key players are involved in product launches and strategic collaborations to strengthen their market positions.

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Gene Therapy Market Regional Analysis

The market has been divided, by region, into the Americas, Europe, Asia-Pacific, and the Middle East & Africa. The Americas is expected to be the largest market owing to the increasing prevalence of gene therapy and rising per capita healthcare expenditure. The gene therapy market in the Americas has further been branched into North America and Latin America, with the North American market divided into the US and Canada. The European gene therapy market has been categorized as Western Europe and Eastern Europe. The Western European market has further been classified as Germany, France, the UK, Italy, Spain, and the rest of Western Europe. The gene therapy market in Asia-Pacific has been segmented into Japan, China, India, South Korea, Australia, and the rest of Asia-Pacific. Due to the increasing number of patients and raising awareness about the treatment methods, the market in Asia-Pacific is expected to be the fastest-growing. The gene therapy market in the Middle East & Africa has been divided into the Middle East and Africa.

Gene Therapy Market Segmentation

The global gene therapy market has been segmented based on product, indication, and end user.

The market, based on product, has been divided into kymriah, luxturna, yescarta, zolgensma, strimvelis, zynteglo, imlygic, and others.

The global gene therapy market has on the basis of indication is segmented, into oncology, genetic diseases/ disorders, and others. The oncology segment is expected to hold the largest market share and to be the fastest-growing segment due to high prevalence of cancer and robust pipeline for cancer gene therapy.

The end-user segments of the market are hospitals & clinics, specialty treatment centers, and others. The hospitals & clinics segment is expected to hold the largest market share due to increasing number of hospitals. Also, hospitals are generally the first treatment choice for diseases.

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Gene Therapy Market Key Players

Some of the key players in the global gene therapy market are Amgen, Inc. (US), Novartis AG (Switzerland), Spark Therapeutics, Inc. (US), Gilead Sciences, Inc. (US), uniQure N.V. (Netherlands), Orchard Therapeutics plc (UK), Celgene Corporation (US), Bristol-Myers Squibb Company (US), Thermo Fisher Scientific Inc. (US), CEVEC (Germany), and Lonza (Switzerland).

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Gene Therapy Market Size Is Projected to Register a CAGR of 40.7% By 2025 - Jewish Life News

ReportsandMarkets newly added a research report on the Hemophilia Gene Therapy market, which represents a study for the period from 2020 to 2026. The research study provides a near look at the market scenario and dynamics impacting its growth. This report highlights the crucial developments along with other events happening in the market which are marking on the growth and opening doors for future growth in the coming years. Additionally, the report is built on the basis of the macro- and micro-economic factors and historical data that can influence the growth.

Furthermore, this report on the Hemophilia Gene Therapy market put foremost descriptive viewpoint over the market, its peer market, as well as its parent market. In order to achieve this, bottom-up and top-down approaches are adopted by the analysts and researchers for the estimation of segmental, global, and regional revenue along with its consumption volume. Further, these findings are validated from the primary research and by discussing with seniors working in the market.

The final report will add the analysis of the Impact of Covid-19 in this report Hemophilia Gene Therapyindustry.

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Key Players

The global Hemophilia Gene Therapymarket has been comprehensively analyzed and the different companies that occupy a large percentage of the market share in the regions mentioned have been listed out in the report. Industry trends that are popular and are causing a resurgence in the market growth are identified. A strategic profile of the companies is also carried out to identify the various subsidiaries that they own in the different regions and who are responsible for daily operations in these regions. Business data for each of the companies mentioned are covered in the report published on the Hemophilia Gene Therapymarket.

Major Companies Included in Report are Ultragenyx, BioMarin, Shire PLC, uniQure, Sangamo Therapeutics, Bioverativ, Freeline Therapeutics, and Spark Therapeutics

Research objectivesTo study and analyze the global Hemophilia Gene Therapyconsumption (value & volume) by key regions/countries, product type and application, history data from 2013to 2019, and forecast to 2026.To understand the structure of Hemophilia Gene Therapymarket by identifying its various subsegments.Focuses on the key global Hemophilia Gene Therapymanufacturers, to define, describe and analyze the sales volume, value, market share, market competition landscape, SWOT analysis and development plans in next few years.To analyze the Hemophilia Gene Therapywith respect to individual growth trends, future prospects, and their contribution to the total market.To share detailed information about the key factors influencing the growth of the market (growth potential, opportunities, drivers, industry-specific challenges and risks).To project the consumption of Hemophilia Gene Therapysubmarkets, with respect to key regions (along with their respective key countries).To analyze competitive developments such as expansions, agreements, new product launches, and acquisitions in the market.To strategically profile the key players and comprehensively analyze their growth strategies.

Market segmentationHemophilia Gene Therapymarket is split by Type and by Application. For the period 2015-2026, the growth among segments provide accurate calculations and forecasts for sales by Type and by Application in terms of volume and value. This analysis can help you expand your business by targeting qualified niche markets.

Research Methodology:Our market forecasting is based on a market model derived from market connectivity, dynamics, and identified influential factors around which assumptions about the market are made. These assumptions are enlightened by fact-bases, put by primary and secondary research instruments, regressive analysis and an extensive connect with industry people. Market forecasting derived from in-depth understanding attained from future market spending patterns provides quantified insight to support your decision-making process. The interview is recorded, and the information gathered in put on the drawing board with the information collected through secondary research.

The report provides insights on the following pointers:1. Market Penetration: Provides comprehensive information on Hemophilia Gene Therapyoffered by the key players in the Global Hemophilia Gene TherapyMarket2. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and new product developments in the Global Hemophilia Gene TherapyMarket3. Market Development: Provides in-depth information about lucrative emerging markets and analyzes the markets for the Global Hemophilia Gene TherapyMarket4. Market Diversification: Provides detailed information about new products launches, untapped geographies, recent developments, and investments in the Global Hemophilia Gene TherapyMarket5. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, and manufacturing capabilities of the leading players in the Global Hemophilia Gene TherapyMarket

The report answers questions such as:1. What is the market size of Hemophilia Gene Therapymarket in the Global?2. What are the factors that affect the growth in the Global Hemophilia Gene TherapyMarket over the forecast period?3. What is the competitive position in the Global Hemophilia Gene TherapyMarket?4. Which are the best product areas to be invested in over the forecast period in the Global Hemophilia Gene TherapyMarket?5. What are the opportunities in the Global Hemophilia Gene TherapyMarket?6. What are the modes of entering the Global Hemophilia Gene TherapyMarket?

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TABLE OF CONTENT:

Chapter 1:Hemophilia Gene TherapyMarket Overview

Chapter 2: Global Economic Impact on Industry

Chapter 3:Hemophilia Gene TherapyMarket Competition by Manufacturers

Chapter 4: Global Production, Revenue (Value) by Region

Chapter 5: Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6: Global Production, Revenue (Value), Price Trend by Type

Chapter 7: Global Market Analysis by Application

Chapter 8: Manufacturing Cost Analysis

Chapter 9: Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10: Marketing Strategy Analysis, Distributors/Traders

Chapter 11: Hemophilia Gene TherapyMarket Effect Factors Analysis

Chapter 12: GlobalHemophilia Gene TherapyMarket Forecast to 2026

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Latest Trends in Hemophilia Gene Therapy Market 2020, Research Report Covers Updated Data Considering Impact of Covid-19 on Share, Size and Future...

The next few months could bring the first gene therapy for hemophilia, a new type of multiple myeloma drug and an additional treatment option for a devastating muscular condition.

The Food and Drug Administration is set to make decisions on approval of all three, as well as on a closely watched arthritis pill and the first oral drug for spinal muscular atrophy, between July and September.

A regulatory OK for Roctavian, BioMarin Pharmaceutical's experimental hemophilia A treatment, would be a particularly significant milestone the first gene therapy for one of the world's most well-known inherited diseases. Approvals for Roche's risdiplam, NS Pharma's viltolarsen, Gilead's filgotinib and GlaxoSmithKline's belantamab mafodotin would also be notable.

The list of experimental drugs slated for the FDA's review during the third quarter was originally longer, but the agency cleared three targeted cancer therapies months ahead of schedule. (Seattle Genetics' Tukysa, Novartis' Tabrecta and Eli Lilly's Retevmo were all approved in April and May.)

Even as it's moved quickly to approve new drugs, the FDA has acknowledged the strain resulting from the work the agency is doing to oversee COVID-19 vaccine and drug development. So far, new drug reviews haven't been heavily affected, but the FDA has warned they could be slowed.

The five experimental drugs are listed in order of the currently scheduled date by which the FDA has agreed to make a decision on approval.

Around a dozen drugmakers are developing multiple myeloma treatments that target a protein known as BCMA, which is found on nearly all malignant cells. GlaxoSmithKline looks to be in a position to get there first.

GSK's drug, called belantamab mafodotin, is an antibody-drug conjugate, designed to bind to the BCMA target and deliver a small chemotherapy payload to attack cancerous cells. The conjugate approach is employed by Seattle Genetics' Adcetris and Roche's Kadcyla for other types of blood cancer.

Behind belantamab mafodotin, however, several other BCMA-targeting therapies are nearing key milestones, including a CAR-T cell therapy from Bristol-Myers Squibb and Bluebird bio, as well as a bispecific antibody from Regeneron.

They would trail belantamab mafadotin if the GSK drug is approved by the FDA's target decision date sometime this month. But the rival therapies could potentially offer advantages in greater effectiveness or more manageable side effects. Notably, balantamab mafadotin is associated with an eye-related side effect called keratopathy.

The FDA's decision to approve Sarepta Therapeutics' Exondys 51 for Duchenne muscular dystrophy in 2016 was one of the most controversial in the agency's recent history. The drug, a type of genetic treatment known as "exon-skipping," was the first specifically cleared for patients with DMD.

Approval late last year of Sarepta's second drug Vyondys 53 for a different segment of DMD patients was nearly as dramatic, with the FDA reversing an initial rejection after the company appealed to more senior agency officials.

The FDA's review of a DMD treatment developed by NS Pharma, a little-known subsidiary of Japan's Nippon Shinyaku, promises to be less eventful.

NS' drug, called viltolarsen and designed to treat the same group of DMD patients as Vyondys 53, works similarly to both of Sarepta's drugs. Study results published in JAMA Neurology this past May suggest it might even work better than Vyondys 53, although comparing drugs across trials can be misleading.

Like Sarepta, however, NS is offering the FDA limited evidence to go on. The study supporting the drugmaker's application primarily tested whether viltolarsen increased the production of a key protein that's largely missing in children with DMD and, notably, lacked a placebo comparison.

Having cleared Exondys 51 and then Vyondys 53, however, the FDA seems to have signaled it will accept early data as compelling enough to merit approval. A decision is expected sometime in August.

For Gilead and partner drugmaker Galapagos, much is riding on filgotinib. The drug is at the center of a research collaboration between the two companies, first inked in 2015 and then expanded with a $5.1 billion pact last year.

Part of a class of drugs called JAK inhibitors, filgotinib is aimed first at rheumatoid arthritis, a chronic condition for which it will have much competition. Three other JAK inhibitors, Pfizer's Xeljanz, Eli Lilly's Olumiant and AbbVie's Rinvoq, are already approved for rheumatoid arthritis, which is also commonly treated with biologic drugs like Humira.

Some analysts on Wall Street see filgotinib as safer than its JAK-blocking rivals, but the FDA appears to be viewing the risk of blood clots as characteristic of the drug class.

Gilead and Galapagos still think filgotinib can stand out and are counting on an August approval from the FDA to prove it. Clearance for rheumatoid arthritis would be the first step in proving the drug's future for other inflammatory conditions, like Crohn's disease, uveitis and psoriatic arthritis.

An approval for BioMarin's Roctavian would be a significant moment for the still-emerging gene therapy field. The experimental hemophilia A treatment wouldn't be the first gene therapy to gain FDA clearance that milestone went to Luxturna for an inherited form of blindness but it would offer thousands of patients long-lasting control of a disruptive and damaging bleeding disorder.

Roctavian also typifies the substantial benefits gene therapy can offer. Studies showed the treatment largely eliminated bleeding episodes, enabling patients to drastically reduce how much they rely on expensive factor replacement therapy that aids blood clotting. Years later, patients who received the treatment are still largely free of both.

BioMarin has suggested a price for Roctavian as high as $3 million, arguing the drug's value is clear when factor replacement therapy can sometimes cost as much as $1 million per year for severe hemophilia patients.

The company recently detailed follow-up data up to four years post-treatment in patients who were enrolled in early clinical trials. While their levels of blood clotting activity appear to decline over time, they are all still producing the Factor VIII protein that's reduced or missing in patients with hemophilia.

Spinal muscular atrophy, or SMA, is an often-fatal neuromuscular condition that primarily affects infants and children. Until three and a half years ago, there was no treatment.

An approval for Roche and PTC Therapeutics' risdiplam, expected by August 24, would bring to market the third treatment for the disease and the first one taken orally, which may be more convenient for some patients.

The companies have played up risdiplam's advantages compared to Spinraza, the first SMA drug, as well as versus Novartis' gene therapy Zolgensma. Spinraza is administered via a spinal injection, while Zolgensma is infused intravenously for treating infants.

Roche and PTC sought to submit to the FDA study data covering a broad range of patients, from infants to adults and across various levels of disease severity. In doing so, however, they caused the agency to extend its approval review by three months in order to take into account results from a study in less severe adolescents and adults.

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5 FDA approval decisions to watch in the 3rd quarter - BioPharma Dive

Cell and Gene Therapy Market 2020: Latest Analysis

Chicago, United States:-TheCell and Gene Therapy market report5 Years Forecast [2020-2025]focuses on theCOVID19 Outbreak Impact analysis of key points influencing the growth of the market. The research report on the Cell and Gene Therapy Market is a deep analysis of the market. This is a latest report, covering the current COVID-19 impact on the Cell and Gene Therapy market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions. The rapidly changing market scenario and initial and future assessment of the impact is covered in the report. Experts have studied the historical data and compared it with the changing market situations. The report covers all the necessary information required by new entrants as well as the existing players to gain deeper insight.

Furthermore, the statistical survey in the report focuses on product specifications, costs, production capacities, marketing channels, and market players. Upstream raw materials, downstream demand analysis, and a list of end-user industries have been studied systematically, along with the suppliers in this market. The product flow and distribution channel have also been presented in this research report.The report assesses the key opportunities in the market and outlines the factors that are and will be driving the growth of the Cell and Gene Therapyindustry. Growth of the overall Cell and Gene Therapymarket has also been forecasted for the period 2020-2025, taking into consideration the previous growth patterns, the growth drivers and the current and future trends.

Top Players of Cell and Gene Therapy Market are studied:[ Amgen Inc., bluebird bio, Inc., Dendreon Pharmaceuticals LLC., Fibrocell Science, Inc., Human Stem Cells Institute, Kite Pharma, Inc., Kolon TissueGene, Inc., Novartis AG, Orchard Therapeutics plc., Organogenesis Holdings Inc., Pfizer, Inc., RENOVA THERAPEUTICS ]

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Note: Covid-19 pandemic affects most industries in the globe. Here at acquire market research we offer you comprehensive data of related industry which will help and support your business in all possible ways.Due to the pandemic of COVID-19 businesses have seen a decrease in their profits. While our intention is to help businesses regain their profits we also provide information regarding the COVID-19 virus to help our customers stay safe during the pandemic

Cell and Gene TherapySegmentation by Product

General Type

Cell and Gene TherapySegmentation by Application

Pharmaceutical and Biotechnology CompaniesResearch and Academic Institutions

Benefits of Buying this Market Research Report:

Our exploration specialists acutely ascertain the significant aspects of the global Cell and Gene Therapy market report. It also provides an in-depth valuation in regards to the future advancements relying on the past data and present circumstance of Cell and Gene Therapy market situation. In this Cell and Gene Therapy report, we have investigated the principals, players in the market, geological regions, product type, and market end-client applications. The global Cell and Gene Therapy report comprises of primary and secondary data which is exemplified in the form of pie outlines, Cell and Gene Therapy tables, analytical figures, and reference diagrams. The Cell and Gene Therapy report is presented in an efficient way that involves basic dialect, basic Cell and Gene Therapy outline, agreements, and certain facts as per solace and comprehension.

Table of Contents.

Report Overview:It includes major players of the globalkeywordmarket covered in the research study, research scope, and market segments by type, market segments by application, years considered for the research study, and objectives of the report.

Global Growth Trends:This section focuses on industry trends where market drivers and top market trends are shed light upon. It also provides growth rates of key producers operating in the globalkeywordmarket. Furthermore, it offers production and capacity analysis where marketing pricing trends, capacity, production, and production value of the globalkeywordmarket are discussed.

Market Share by Manufacturers:Here, the report provides details about revenue by manufacturers, production and capacity by manufacturers, price by manufacturers, expansion plans, mergers and acquisitions, and products, market entry dates, distribution, and market areas of key manufacturers.

Market Size by Type:This section concentrates on product type segments where production value market share, price, and production market share by product type are discussed.

Market Size by Application:Besides an overview of the globalkeywordmarket by application, it gives a study on the consumption in the globalkeywordmarket by application.

Production by Region:Here, the production value growth rate, production growth rate, import and export, and key players of each regional market are provided.

Consumption by Region:This section provides information on the consumption in each regional market studied in the report. The consumption is discussed on the basis of country, application, and product type.

Company Profiles:Almost all leading players of the globalkeywordmarket are profiled in this section. The analysts have provided information about their recent developments in the globalkeywordmarket, products, revenue, production, business, and company.

Market Forecast by Production:The production and production value forecasts included in this section are for the globalkeywordmarket as well as for key regional markets.

Market Forecast by Consumption:The consumption and consumption value forecasts included in this section are for the globalkeywordmarket as well as for key regional markets.

Value Chain and Sales Analysis:It deeply analyzes customers, distributors, sales channels, and value chain of the globalkeywordmarket.

Key Findings:This section gives a quick look at the important findings of the research study.

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Covid-19 Impact On Cell and Gene Therapy Market 2020: Global Industry Overview By Top Key Players Analysis And Growth Factors Up To 2025 | Amgen Inc.,...

The study on the Gene Therapy for Age-related Macular Degeneration Marketby Brand Essence Market Research is a compilation of systematic details in terms of market valuation, market size, revenue estimation, and geographical spectrum of the business vertical. The study also offers a precise analysis of the key challenges and growth prospects awaiting key players of the Gene Therapy for Age-related Macular Degeneration market, including a concise summary of their corporate strategies and competitive setting.

In 2018, the Global Gene Therapy for Age-related Macular Degeneration Market size was xx million US$ and it is expected to reach xx million US$ by the end of 2025, with a CAGR of xx% during 2019-2025.

Download Premium Sample of the Report: https://industrystatsreport.com/Request/Sample?ResearchPostId=11998&RequestType=Sample

Latest research report on Gene Therapy for Age-related Macular Degeneration Market delivers a comprehensive study on current market trends. The outcome also includes revenue forecasts, statistics, market valuations which illustrates its growth trends and competitive landscape as well as the key players in the business.

Macular degeneration is a condition in which, macula, a part of the retina, gets damaged or deteriorated. This condition usually affects individuals who are aged 50 years and above and therefore, it is called age-related macular degeneration (AMD). AMD is the leading cause of vision loss and is directly related to the advancement of age. But smoking also plays a vital role in causing AMD. AMD is characterized by the presence of a blurred area near the center of vision that leads to distorted vision. There are two different types of AMD, including dry (atrophic) AMD (dAMD) and wet (neovascular/exudative) AMD (wAMD). The dAMD is the most common type of AMD and accounts for almost 80%-90% of the overall AMD cases.

It has been observed that age-related macular degeneration (AMD) is one of the major causes for vision loss and is characterized by the formation of a blurred area near the center of vision, a condition that mostly affects the geriatric population. According to the CDC, almost 2 million individuals in the US suffer from AMD and by 2050, this number will reach more than 5 million. This will subsequently demand the need for the development of innovative treatments for AMD, driving the markets growth.

The market research analysts have predicted that with the introduction of techniques such as fluorescein angiography, the global age-related macular degeneration market will register a CAGR of more than 7% by 2020. With the unavailability of FDA-approved treatment for dry AMD (dAMD) and the treatment of wet AMD (wAMD) involving the need of intravitreal injections for an indefinite period, gene therapy is emerging as the most-efficient approach for the treatment of age-related macular degeneration (AMD).

According to this pipeline analysis report, most of the gene therapy molecules in the pipeline are being developed for wet AMD (wAMD). Our market research analysts have also identified that most of these molecules are in the pre-clinical development stage and a considerable number of molecules have been discontinued from development.

This report focuses on the global Gene Therapy for Age-related Macular Degeneration status, future forecast, growth opportunity, key market and key players. The study objectives are to present the Gene Therapy for Age-related Macular Degeneration development in United States, Europe and China.

The key players covered in this study RetroSense Therapeutics REGENXBIO AGTC

Market segment by Type, the product can be split into Subretinal Intravitreal Unspecified

Market segment by Application, split into Monotherapy Combination Therapy

In this study, the years considered to estimate the market size of Gene Therapy for Age-related Macular Degeneration are as follows: History Year: 2014-2018 Base Year: 2018 Estimated Year: 2019 Forecast Year 2019 to 2025

Market segment by Regions/Countries, this report covers United States Europe China Japan Southeast Asia India Central & South America

The study objectives of this report are: To analyze global Gene Therapy for Age-related Macular Degeneration status, future forecast, growth opportunity, key market and key players. To present the Gene Therapy for Age-related Macular Degeneration development in United States, Europe and China. To strategically profile the key players and comprehensively analyze their development plan and strategies. To define, describe and forecast the market by product type, market and key regions.

For the data information by region, company, type and application, 2018 is considered as the base year. Whenever data information was unavailable for the base year, the prior year has been considered.

Table of Contents

1 Report Overview1.1 Study Scope1.2 Key Market Segments1.3 Players Covered1.4 Market Analysis by Type1.4.1 Global Gene Therapy for Age-related Macular Degeneration Market Size Growth Rate by Type (2014-2025)1.4.2 Topical Products1.4.3 Botulinum1.4.4 Dermal Fillers1.4.5 Chemical Peels1.4.6 Microabrasion Equipment1.4.7 Laser Surfacing Treatments1.5 Market by Application1.5.1 Global Gene Therapy for Age-related Macular Degeneration Market Share by Application (2014-2025)1.5.2 Hospitals1.5.3 Dermatology Clinics1.6 Study Objectives1.7 Years Considered

2 Global Growth Trends2.1 Gene Therapy for Age-related Macular Degeneration Market Size2.2 Gene Therapy for Age-related Macular Degeneration Growth Trends by Regions2.2.1 Gene Therapy for Age-related Macular Degeneration Market Size by Regions (2014-2025)2.2.2 Gene Therapy for Age-related Macular Degeneration Market Share by Regions (2014-2019)2.3 Industry Trends2.3.1 Market Top Trends2.3.2 Market Drivers2.3.3 Market Opportunities

3 Market Share by Key Players3.1 Gene Therapy for Age-related Macular Degeneration Market Size by Manufacturers3.1.1 Global Gene Therapy for Age-related Macular Degeneration Revenue by Manufacturers (2014-2019)3.1.2 Global Gene Therapy for Age-related Macular Degeneration Revenue Market Share by Manufacturers (2014-2019)3.1.3 Global Gene Therapy for Age-related Macular Degeneration Market Concentration Ratio (CR5 and HHI)3.2 Gene Therapy for Age-related Macular Degeneration Key Players Head office and Area Served3.3 Key Players Gene Therapy for Age-related Macular Degeneration Product/Solution/Service3.4 Date of Enter into Gene Therapy for Age-related Macular Degeneration Market3.5 Mergers & Acquisitions, Expansion Plans

Read More: https://industrystatsreport.com/Lifesciences-and-Healthcare/Dynamic-Growth-On-Gene-Therapy-for-Age-related-Macular-Degeneration-Market-Size-and-Share/Summary

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Covid-19 Outbreak: Gene Therapy for Age-related Macular Degeneration Market Insights and Forecast 2020 to 2025 - Owned

Mutations in more than 270 genes have been implicated in inherited eye diseases like retinitis pigmentosa. Now, Biogen has formed a research pact with Harvards Massachusetts Eye and Ear thats aimed at developing a gene therapy to help some patients with these blinding diseases.

The gene at the center of the new agreement, PRPF31, has been linked to autosomal dominant retinitis pigmentosa. PRPF31 mutations are believed to cause an estimated 25% of all retinitis pigmentosa cases. The partners did not disclose the financial terms of the deal.

The tie-up comes eight months after a Mass Eye and Ear team published preclinical research demonstrating a gene therapy technique for repairing cells withmutated PRPF31 genes. The technique partially restored the structure and function of retinal pigment epithelium cells, the team reported in the journal Molecular Therapy Methods & Clinical Development. The research was led by Eric Pierce, M.D., Ph.D., professor at Harvard Medical School and director of the inherited retinal disorders service at Mass Eye and Ear.

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Pierces team developed the technique, called adeno-associated virus (AAV)-mediated gene augmentation therapy, with the goal of preserving and possibly bringing back some vision in patients with PRPF31 mutations, he said in a statement. Biogen now has an exclusive license to develop the therapy worldwide and will fund the studies necessary to seek FDA approval.

Biogen has been working to build its expertise in gene therapy. In March 2019, it acquired Nightstar Therapeutics, which is in early development of a treatment for X-linked retinitis pigmentosa, for $877 million. Biogen fought off three other bidders to consummate that deala clear sign of the growing interest in gene therapy.

RELATED: Biogen-Nightstar deal sheds light on gene therapy feeding frenzy

Not all of Biogens forays into gene therapy for ocular diseases have succeeded, though. In 2018, the company pulled out of a research collaboration with Applied Genetic Technologies to develop several gene therapies, including one to treat the inherited retinal disorder X-linked retinoschisis. That therapy was shelved after it was ineffective in a phase 1/2 trial.

Several other gene therapies are being developed to treat retinitis pigmentosa. They include Allergans RST-001, which the company picked up when it acquired RetroSense Therapeutics for $60 million in 2016. RST-001 targets channelrhodopsin, a photosensitivity gene, and is designed to restore light sensitivity to retinal cells. It is currently enrolling patients for a phase 2a trial.

Mass Eye and Ear was the first center to administer Luxturna, Spark Therapeutics gene therapy for retinal degeneration caused by mutations in the gene RPE65, after the product was approved in 2017. One of the exciting aspects of our collaboration with Biogen is that mutations in the PRPF31 gene affect approximately 10 to 20 times more people than mutations in the RPE65 gene, Pierce said in the statement. Success with PRPF31 gene therapy could provide visual benefit to more patients, which is our ultimate goal.

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Biogen boosts gene therapy strategy with Harvard pact focused on inherited eye disease - FierceBiotech

In experiments in rats and mice, two Johns Hopkins scientists an engineer and an ophthalmologist report the successful use of nanoparticles to deliver gene therapy for blinding eye disease. A uniquely engineered large molecule allows researchers to compact large bundles of therapeutic DNA to be delivered into the cells of the eye.The research, described in Science Advances, provides evidence of the potential value of nanoparticle-delivered gene therapy to treat wet age-related macular degeneration an eye disease characterized by abnormal blood vessel growth that damages the light-sensitive tissue in the back of the eye as well as more rare, inherited blinding diseases of the retina.

Many gene therapy approaches depend on viral vectors, which use a viruss natural ability to carry genetic material into cells. However, viruses create an immune response, which prevents repeat dosing, and the most commonly used one for ocular gene therapy cannot carry large genes.

Some of the most prevalent inherited retinal degenerations are due to mutations in large genes that simply cannot fit into the most commonly used viral vector, says Peter Campochiaro, M.D., the Eccles Professor of Ophthalmology at the Johns Hopkins University School of Medicine, and a member of the Johns Hopkins Medicine Wilmer Eye Institute.

To overcome such limitations, Campochiaro and Jordan Green, Ph.D., developed a new approach involving a biodegradable polymer that surrounds and compacts long stretches of DNA, creating nanoparticles that can enter the cells. This technology allows the researchers to convert the cells of the eye into minifactories for a therapeutic protein.

To first test whether the nanoparticles could reach their target cells, the researchers loaded the nanoparticles with a gene for a florescent protein that causes cells to light up like a glow stick.

This glowing molecule allowed the researchers to determine the location, amount and duration of gene expression achievable with the nanoparticles.

They found that even eight months after treatment, the majority of the light-sensitive cells in the rats eyes glowed, showing that the nanoparticles effectively deposited the florescent gene into the cells.

Next, the researchers set up a similar experiment, this time using the nanoparticles to shuttle a biologically relevant gene into the eye. They loaded the nanoparticles with a gene for vascular endothelial growth factor (VEGF), which is responsible for the growth of abnormal blood vessels in people with wet macular degeneration.

The researchers injected the eyes of 30 rats with the nanoparticles carrying the VEGF gene and determined the effects in the retina one, two and five months after injection. One month after injection, each rat tested had developed abnormal blood vessels under and within the retina, like those seen in patients with wet macular degeneration. The abnormal blood vessels were more extensive at two and five months after injection, and there was associated scarring under the retina similar to that seen in chronic untreated wet macular degeneration.

These results show that the genes delivered by nanoparticles stayed active within the cells for several months, says Campochiaro.

Finally, to test a nanoparticles ability to deliver a therapeutic gene for the disease, the researchers used mice genetically engineered to develop a form of wet macular degeneration similar to that in humans. The researchers loaded nanoparticles with a gene that produces a protein that neutralizes VEGF.

Currently, physicians inject such proteins that block VEGF proteins into the eyes of people with macular degeneration, a treatment that helps control the overgrowth of abnormal, leaky blood vessels. But this procedure must be repeated frequently and is burdensome for patients and their caretakers.

Three weeks after injecting nanoparticles containing the gene for the anti-VEGF protein, the mice had a 60% reduction in abnormal blood vessels when compared to control mice. The same effect was seen 35 days later.

These results are extremely promising, says Jordan Green, Ph.D., professor of biomedical engineering at the Johns Hopkins University School of Medicine. We have the ability to reach the cells most significantly affected by degenerative eye disease with nonviral treatments that can allow the eye to create its own sustained therapies.

An estimated 1.6 million people in the U.S. with macular degeneration receive injected drugs to the eye every four to six weeks. A gene therapy treatment could provide a way for the eyes tissue to prevent further vision deterioration with as little as a few initial treatments. Genetic diseases that cause blindness could be treated in a similar way, by introducing functional versions of genes that inherited mutations have disabled.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Success in Use of Nanoparticles To Deliver Gene Therapy for Eye Disease in Rodents - Technology Networks

Hansa Biopharma has granted an exclusive license to Sarepta Therapeutics for developing and promoting a pretreatment aimed to make a gene therapy to treat muscular dystrophy available to people that are naturally resistant to the treatment.

Gene therapy can be used to treat genetic conditions by delivering a healthy copy of the faulty gene using harmless recombinant viruses. The adeno-associated virus (AAV) is one of the most commonly used in gene therapy, and the basis of Sarepta Therapeutics gene therapy technology.

However, up to 70% of people naturally carry neutralizing antibodies against AAV. This prevents the transfer of the healthy gene sequence and raises safety concerns for the patient. Swedish company Hansa Biopharma is developing a pretreatment called imlifidase that works to eliminate these neutralizing antibodies prior to gene therapy treatments.

Imlifidase is an enzyme from the bacterium Streptococcus pyogenes that breaks down the antibodies that are involved in generating an immune response against external pathogens, inhibiting their activity within hours after administration.

Imlifidase is completely unique in development, said Emanuel Bjorne, VP Business Development at Hansa Biopharma. There is no product in the market that has this mode of action.

Sarepta Therapeutics aims to use imlifidase to extend its gene therapies for Duchenne muscular dystrophy and Limb-girdle muscular dystrophy to those patients that are naturally resistant to the AAV vectors used to deliver these treatments. According to the agreement, Hansa will get 8.8M ($10M) upfront and is eligible for a total of 350M ($397M) in development, regulatory, and sales milestone payments, in addition to royalties to future sales.

Sarepta Therapeutics will conduct preclinical evaluation of imlifidase as a pretreatment for gene therapy with Hansa providing our imlifidase expertise to the collaboration, said Bjorne. If everything goes according to plan, the company will start a clinical study of imlifidase in combination with Sareptas gene therapy in the second half of next year.

In preclinical models, Hansas technology has been successful in clearing the antibodies that prevent the success of gene therapies. If successful, this could offer the potential of extending existing gene therapy treatments to patients who would otherwise not be able to benefit from them. Hansa Biopharma is also investigating the use of this drug as a way to prevent the rejection of transplanted organs, and to treat cancer and rare autoimmune conditions.

Antara Mazumdar is a computational biologist based in Groningen, The Netherlands. She is also a freelance science writer who writes about various areas of biological research. Prior to that, she studied biomedical science and bioinformatics in New Delhi, India. Outside of work, she enjoys organizing scientific and cultural events, singing and is a traveling enthusiast.

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Hansa Biopharma Gets up to 350M to Make Gene Therapy Work in Resistant... - Labiotech.eu

All five participants in the 5x1011 vg/kg and 11012 vg/kg dose cohorts and seven participants in the 21012 vg/kg dose show an acceptable safety profile, stable and durable factor VIII expression and substantial improvement in annualized bleed rate (ABR) after between two and 3.3 years follow-up

Represents the longest stable expression of FVIII following investigational gene therapy and reinforces the ability of AAV gene therapy targeting hepatocytes to achieve stable and durable FVIII expression

Optimizing the dose and immunomodulatory regimen; Phase 3 dosing expected in 2021

PHILADELPHIA, July 12, 2020 (GLOBE NEWSWIRE) -- Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced updated data from three dose cohorts of the ongoing Phase 1/2 clinical trial of investigational SPK-8011 in hemophilia A. These data were presented at the International Society of Thrombosis and Hemostasis (ISTH) 2020 Virtual Congress by Principal Investigator Lindsey A. George, M.D., The Perelman School of Medicine, University of Pennsylvania and Childrens Hospital of Philadelphia.

Fourteen participants in the Phase 1/2 trial received a single administration of investigational SPK-8011, two at a dose of 5x1011 vg/kg, three at a dose of 11012 vg/kg and nine at a dose of 21012 vg/kg. As of the June 3, 2020 data cutoff, results from the five total participants in the 5x1011 vg/kg and 11012 dose cohorts and seven participants in the 21012 vg/kg dose cohort show an acceptable safety profile, 91-percent reduction in annualized bleed rate (ABR), 96-percent reduction in FVIII infusions and stable and durable factor FVIII expression after between two and 3.3 years of follow-up.

As previously disclosed, two of nine participants in the 21012 dose cohort lost FVIII expression likely due to a capsid-based immune response. The seven other participants in the 21012 dose cohort and the five total participants in the 51011 vg/kg and 11012 vg/kg dose cohorts continue to show stable and durable factor FVIII expression. These data represent the longest stable expression of FVIII following investigational gene therapy and reinforce the ability of AAV gene therapy targeting hepatocytes to achieve stable and durable FVIII expression.

We are very encouraged by these interim data that continue to show an acceptable safety profile and a substantial reduction in bleeds for more than two years of observation on average, with one participant being observed for more than three years, said Federico Mingozzi, Ph.D., chief scientific officer, Spark Therapeutics. Our focus is on optimizing the dose and immunomodulatory regimen before moving to a Phase 3 clinical study that falls in line with our strategy to progress a hemophilia A gene therapy that, at thelowest effective doseand the optimal immunomodulatory regimen, demonstrates safety, predictability, efficacy, and durability.

Since previous disclosure of these data, two participants experienced mild and non-serious steroid-associated adverse events (e.g. weight gain, insomnia, adrenal insufficiency and worsening gastroesophageal reflux that resolved with medical intervention). Previously disclosed adverse events include one participant experiencing an acute reaction to the infusion that resolved. Three participants reported liver function test (LFT) elevations. One of the events was grade two transaminitis, and two were grade one. All three events resolved. One participant was electively admitted to the hospital to receive intravenous steroids. The event subsequently resolved. The admission to the hospital for these infusions met the criteria for a serious adverse event.

Across the Phase 1/2 study and through the data cutoff, all 14 participants demonstrated rapid clearance of vector from semen, serum, saliva and urine within two weeks post-vector administration. The vector was not detectable in peripheral blood mononuclear cells (PBMCs), semen, serum, saliva and urine by six weeks post-vector administration in all participants.

Spark Therapeutics continues optimizing the dose and immunomodulatory regimen for investigational SPK-8011 and SPK-8016, for hemophilia A patients with inhibitors. The Phase 3 run-in study is ongoing, and dosing participants in Phase 3 is expected to occur in 2021.

Additionally, Spark has scaled-up its state-of-the-art gene therapy manufacturing capabilitiesutilizing suspension cell culture at a scale of nearly 500-litersatcell separationwith a corresponding chromatography purification process, which is the scale required to meet clinical and potential commercial demand for hemophilia A.

About SPK-8011 for hemophilia AInvestigationalSPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the AAV-LK03 capsid, also referred to as Spark200, contains a codon-optimized human factor VIII gene under the control of a liver-specific promoter. The Food and Drug Administration (FDA) granted orphan-disease designation and breakthrough therapy designation in the U.S., while the European Commission has granted orphan designation toSPK-8011.

About Roche and Spark Therapeutics gene therapy research in hemophilia AWe believe gene therapy has the potential to revolutionize medicine and improve the lives of patients with genetic and other serious diseases. Pairing Roches long-standing commitment to developing medicines in hemophilia with Spark Therapeutics proven gene therapy expertise brings together the best team of collaborators researching gene therapies in hemophilia A.

It is our aligned objective to develop gene therapies for hemophilia A that, with the lowest effective dose and the optimal immunomodulatory regimen, demonstrate safety, predictability, efficacy, and durability for patients.

About Hemophilia AHemophilia is a rare genetic bleeding disorder that causes the blood to take a long time to clot because of a deficiency in one of several blood clotting factors. People living with hemophilia are at risk of excessive and recurrent bleeding spontaneously and from modest injuries, which have the potential to be life threatening. There are approximately 15,000 people with hemophilia A in the U.S. and 19,000 in the five major European countries. Hemophilia A is about four times as common as hemophilia B. Hemophilia A is characterized by mutations in the factor VIII gene (F8), which lead to deficient blood coagulation and an increased risk of bleeding or hemorrhaging. The current standard of care for hemophilia A requires recurrent intravenous infusions of either plasma-derived or recombinant factor VIII to control and prevent bleeding episodes. There exists a significant need for novel therapeutics to treat people living with hemophilia.

About Spark Therapeutics AtSpark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challengethe inevitability of genetic diseases,includingblindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.We currently have four programs in clinical trials.At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. For more information, visit http://www.sparktx.com, and follow us on Twitter and LinkedIn.

Media Contact:Kevin Giordanokevin.giordano@sparktx.com(215) 294-9942

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Spark Therapeutics Announces Updated Data on SPK-8011 from Phase 1/2 Clinical Trial in Hemophilia A at ISTH 2020 Virtual Congress - GlobeNewswire

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CNS Gene Therapy Market is Anticipated to Attain a Market Value of US$XX by the End of 2018 2028 - 3rd Watch News

Market Research Exploreoffers an exhaustive analysis on the Global Gene Therapy Market covering diverse significant factors that pose an impact on the current and forthcoming market conditions. The report elaborates on how the Gene Therapy market has been performing over the last five years at the global and regional levels. It also analyzes expected ups and downs in the market over the next five years. The report assists market players to operate their business wisely. Market scope, establishment, profitability, maturity, and growth prospects are also highlighted in the global Gene Therapy market report.

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The global Gene Therapy market has been segmented into a number of various vital segments such as types, applications, and regions. The report evaluates each segment at a minute level in view of its growth prospects, global demand, and current revenue. It also focuses on the segments that are exhibiting exponential growth during the year and help market players in selecting more profitable segments for their Gene Therapy businesses and precisely determine the actual needs and wants of their customer base.

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Expansive survey of Global Gene Therapy Market 2020

According to the report findings, the global Gene Therapy market report is extremely competitive and encouraging leading manufacturers and companies to execute various business and marketing strategies such as M&A activities, brand promotions, product launches, partnerships, and other expansions to perform comfortably in the relentless competition. The report further examines highlights new product developments, innovations, and technology adoptions done by the competitors in order to offer upgraded products and services in the global Gene Therapy market.

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Gene Therapy- Global Market Set to Register the Highest Economic Growth by 2025 - 3rd Watch News

Carmine Therapeutics and Takeda Collaborate to Discover and Develop Rare Disease Gene Therapies Using Novel Red Blood Cell Extracellular Vesicles Platform

Singapore based startup Carmine Therapeutics has announced that it has signed a research collaboration agreement with Japanese firm Takeda Pharmaceutical Company Limited o discover, develop and commercialize transformative non-viral gene therapies for two rare disease targets using Carmines REGENT(TM) technology, based on red blood cell extracellular vesicles. In addition, Takeda has committed a $5M convertible loan in support of the development of Carmines novel REGENT platform.

Under the terms of the agreement, Carmine will receive an upfront payment, research funding support, and is eligible for over $900M in total milestone payments plus tiered royalties.

Takeda has an option to license the programs following the completion of pre-clinical proof of concept studies and would be responsible for clinical development and commercialization.

Carmine Therapeutics is pioneering a novel class of therapeutics based on its REGENT(TM) technology which leverages red blood cell extracellular vesicles (RBCEVs), first published in Nature Communications in 2018.

An initial focus is non-viral gene therapies. Compared to adeno-associated virus (AAV)-based gene therapy, RBCEV-based gene therapy has the potential for repeat dosing, a significantly larger transgene payload capacity in excess of 11KB, and enhanced bio-distribution in selected tissues through RBCEV surface modification.

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Carmine Therapeutics teams up with Takeda for non-viral gene therapies - BSA bureau

UK-based Freeline, a biotechnology startup focused on developing curative gene therapies for chronic systemic diseases, has announced the closing ofan around 106 million extended Series C financing. Thisround includes a previously announced 35.4 million from Syncona, in addition to a more recentadditional amount of around 70.8 million led by Novo Holdings A/S, Eventide Asset Management and Wellington Management Company, with additional participation from Cowen Healthcare Investments, Acorn Bioventures and Ample Plus Fund.

Freeline, founded in 2015, aims to create better lives for people suffering from chronic, debilitating diseases. Combining innovation with an entrepreneurial spirit, they aim for life-changing impact by developing and commercialising innovative gene therapies. At the centre of their work is the goal to create functional cures for a wide range of liver-based diseases, which have previously been underserved and incurable.

The Stevenage-based biotech startup will use the funds to bring its lead programme in Haemophilia B into a pivotal trial, as well as to continue its Phase 1/2 clinical programme for Fabry Disease and further progress its pipeline programmes for Gaucher Disease and Haemophilia A. Additionally, Freeline expects to use the proceeds from the financing to further develop its proprietary gene therapy platform using next-generation AAV technology and expand its manufacturing capabilities.

The potential of gene therapy to change patients lives has never been greater and we are delighted to have leading US and European biotechnology investors join us in this extended Series C financing, said Theresa Heggie, CEO of Freeline. It comes at an exciting time for Freeline with our lead programme in Haemophilia B progressing through clinical development, and with promising programmes behind that, including a gene therapy treatment for Fabry Disease in the clinic and for Gaucher Disease and Haemophilia A in late preclinical development.

Our investment strategy is to identify and invest in US and European life science companies which are true leaders in their area of expertise, developing innovative product candidates that significantly advance patient care, said Thomas Dyrberg, Managing Partner, Novo Ventures, Novo Holdings A/S. We have been impressed by the Freeline platform and its scientific co-founders, as well as their experienced management team, and we are delighted to support Freeline as they continue to build momentum.

In connection with the financing, Freeline will appoint Thomas Dyrberg of Novo Holdings A/S to its Board of Directors.

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British biotech Freeline secures 106 million for its curative liver gene therapies - EU-Startups

New Jersey, United States,- The Gene Therapy Market report upholds the future market predictions related to Gene Therapy market size, revenue, production, Consumption, gross margin, and other substantial factors. It also examines the role of the prominent Gene Therapy market players involved in the industry including their corporate overview. While emphasizing the key driving factors for the Gene Therapy market, the report also offers a full study of the future trends and developments of the market.

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Gene Therapy Market Size By Types, By Application, By Region, And Forecast 20202027 - Owned

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Gene Therapy For CNS Disorders Market Size By Product Analysis, Application, End-Users, Regional Outlook, Competitive Strategies And Forecast Up To...

The research study presented here is a brilliant compilation of different types of analysis of critical aspects of the global Retinal Gene Therapy market. It sheds light on how the global Retinal Gene Therapy market is expected to grow during the course of the forecast period. With SWOT analysis and Porters Five Forces analysis, it gives a deep explanation of the strengths and weaknesses of the global Retinal Gene Therapy market and different players operating therein. The authors of the report have also provided qualitative and quantitative analyses of several microeconomic and macroeconomic factors impacting the global Retinal Gene Therapy market. In addition, the research study helps to understand the changes in the industry supply chain, manufacturing process and cost, sales scenarios, and dynamics of the global Retinal Gene Therapy market.

Each player studied in the report is profiled while taking into account its production, market value, sales, gross margin, market share, recent developments, and marketing and business strategies. Besides giving a broad study of the drivers, restraints, trends, and opportunities of the global Retinal Gene Therapy market, the report offers an individual, detailed analysis of important regions such as North America, Europe, and Asia Pacific. Furthermore, important segments of the global Retinal Gene Therapy market are studied in great detail with key focus on their market share, CAGR, and other vital factors.

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Regional Wise Outlook

Geographically, the retinal gene therapy market is segmented into seven regions viz. South Asia, East Asia, North America, Latin America, Oceania, Europe, and Middle East and Africa. The North America retinal gene therapy market is the leader in the concerned global retinal gene therapy market. The only product approved is from a US based manufacturer, which reflects to the current market share for North America. The retinal gene therapy is also available in Europe from Spark Therapeutics. The deal is such that Novartis has exclusive rights to commercialize and distribute Luxturna in Europe and in all other countries outside the US, when regularized for use. So, clearly the current market for retinal gene therapy is consolidated in these two region.

The only player involved in this market of retinal gene therapy is Spark Therapeutics, Inc. The present market structure of retinal gene therapy is expected to change with efforts and research present in clinical phase.

The report is a compilation of first-hand information, qualitative and quantitative assessment by industry analysts, inputs from industry experts and industry participants across the value chain. The report provides in-depth analysis of parent market trends, macro-economic indicators and governing factors along with market attractiveness as per segments.

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Regional analysis of the Retinal Gene Therapy market report includes

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Table of Contents Covered In Retinal Gene Therapy Market Are:

Industry Overview: The first section of the research study touches on an overview of the global Retinal Gene Therapy market, market status and outlook, and product scope. Additionally, it provides highlights of key segments of the global Retinal Gene Therapy market, i.e. regional, type, and application segments.

Competition Analysis: Here, the report brings to light important mergers and acquisitions, business expansions, product or service differences, market concentration rate, the competitive status of the global Retinal Gene Therapy market, and market size by player.

Company Profiles and Key Data: This section deals with the company profiling of leading players of the global Retinal Gene Therapy market on the basis of revenue, products, business, and other factors mentioned earlier.

Market Size by Type and Application: Besides offering a deep analysis of the size of the global Retinal Gene Therapy market by type and application, this section provides a study on top end users or consumers and potential applications.

North America Market: Here, the report explains the changes in the market size of North America by application and player.

Europe Market: This section of the report shows how the size of the Europe market will change in the next few years.

China Market: It gives analysis of the China market and its size for all the years of the forecast period.

Rest of Asia Pacific Market: The Rest of Asia Pacific market is analyzed in quite some detail here on the basis of application and player.

Central and South America Market: The report explains the changes in the size of the Central and South America market by player and application.

MEA Market: This section shows how the size of the MEA market will change during the course of the forecast period.

Market Dynamics: Here, the report deals with the drivers, restraints, challenges, trends, and opportunities of the global Retinal Gene Therapy market. This section also includes the Porters Five Forces analysis.

Research Findings and Conclusion: It gives powerful recommendations for new as well as established players for securing a position of strength in the global Retinal Gene Therapy market.

Methodology and Data Source: This section includes the authors list, a disclaimer, research approach, and data sources.

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Key Questions Answered in Retinal Gene Therapy Market Report are:

Research Methodology of Retinal Gene Therapy

Retinal Gene Therapy Market Report uses trustworthy primary and secondary research sources to compile its reports. It also relies on latest research techniques to prepare highly detailed and accurate research studies such as this one here. It uses data triangulation, top down and bottom up approaches, and advanced research processes to come out with comprehensive and industry-best market research reports.

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Retinal Gene Therapy Market Set to Surpass US$XX Million by the end of 2019 2029 - 3rd Watch News

A confirmed diagnosis may take time.

Diagnosis represents the first step on this rare disease journey. Sometimes doctors will notice something off about the child during a newborn screening, and a genetic test will identify a known mutation in the DNA. But not all conditions are so quickly detected, and it can take several years for parents to get a confirmed diagnosis.

About half of all children never get that far, according to Marshall Summar, M.D., the director of the Rare Disease Institute at Childrens National Hospital in Washington, D.C. When you sequence someones DNA, you are going to find a lot of changes, Dr. Summar said. Figuring out which change might be the one that is causing it is a tremendous challenge.

Genetic counselors warn parents beforehand that they may not get a definitive answer as to what condition their child could have. They may have to check back each year. Dr. Summar estimates that between five and 10 new rare diseases are described in the scientific literature every week, making it challenging for the medical field to keep up.

Meanwhile, the realization that a child may have a debilitating, lifelong condition weighs heavily. Some parents, particularly mothers, blame themselves, said Lemuel Pelentsov, Ph.D., a nurse who studies the needs of rare disease families at the University of South Australia, in Adelaide. In a 2016 study by Dr. Pelentsov and his colleagues, about 40 percent of the 300 rare parents surveyed reported being treated for depression and an equal number for anxiety. One of the things they do to combat that, he said, is get very invested in the childs disease.

When parents reach out to other parents, they are not simply looking for emotional support or advice. They are rebuilding a social life, one that will revolve around their childs disease. Many rare diseases have their own support groups. Global Genes is an umbrella group that supports 600 disease-specific foundations, as well as parents of children whose diseases are so rare they have no foundation.

We encourage folks to work together, said Kimberly Haugstad, the organizations executive director whose son has a rare form of hemophilia, a condition in which the blood doesnt clot normally. The parent is going to come from such different places in their own walk of life.

Each year, Global Genes hosts a Rare Boot Camp to mentor and teach parents how to set up a nonprofit, create patient registries and fund research. After attending the boot camp, the Van Wyks and other parents founded GACI Global, an organization that connects families affected by GACI, along with medical professionals.

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The Wilderness of Rare Genetic Diseases and the Parents Navigating It - The New York Times

The research study on Global Gene Therapy market 2019 presents an extensive analysis of current Gene Therapy market size, drivers, trends, opportunities, challenges, as well as key Gene Therapy market segments. Further, it explains various definitions and classification of the Gene Therapy industry, applications, and chain structure.In continuation of this data, the Gene Therapy report covers various marketing strategies followed by key players and distributors. Also explains Gene Therapy marketing channels, potential buyers and development history. The intent of global Gene Therapy research report is to depict the information to the user regarding Gene Therapy market forecast and dynamics for the upcoming years. The Gene Therapy study lists the essential elements which influence the growth of Gene Therapy industry. Long-term evaluation of the worldwide Gene Therapy market share from diverse countries and regions is roofed within the Gene Therapy report. Additionally, includes Gene Therapy type wise and application wise consumption figures.

The Final Report will cover the impact analysis of COVID-19 on this industry.

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After the basic information, the global Gene Therapy Market study sheds light on the Gene Therapy technological evolution, tie-ups, acquisition, innovative Gene Therapy business approach, new launches and Gene Therapy revenue. In addition, the Gene Therapy industry growth in distinct regions and Gene Therapy R;D status are enclosed within the report.The Gene Therapy study also incorporates new investment feasibility analysis of Gene Therapy. Together with strategically analyzing the key micro markets, the report also focuses on industry-specific drivers, restraints, opportunities, and challenges in the Gene Therapy market.

Global Gene Therapy Market Segmentation 2019: Gene TherapyThe study also classifies the entire Gene Therapy market on basis of leading manufacturers, different types, various applications and diverse geographical regions. Overall Gene Therapy market is characterized by the existence of well-known global and regional Gene Therapy vendors. These established Gene Therapy players have huge essential resources and funds for Gene Therapy research as well as developmental activities. Also, the Gene Therapy manufacturers focusing on the development of new Gene Therapy technologies and feedstock. In fact, this will enhance the competitive scenario of the Gene Therapy industry.

The Leading Players involved in global Gene Therapy market are:

By Gene Therapy Type (Germline Gene Therapy and Somatic Gene Therapy)

By Type of Vector (Viral Vector and Non-viral Vector)

By Disease Indication (Cardio Vascular Diseases, Cancer, Genetic Disorders, Neuro Disorders, Infectious Diseases, and Others)

By Region (North America, Europe, Asia Pacific, Latin America, Middle East, and Africa)

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Worldwide Gene Therapy Market Different Analysis:Competitors Review of Gene Therapy Market: Report presents the competitive landscape scenario seen among top Gene Therapy players, their company profile, revenue, sales, business tactics and forecast Gene Therapy industry situations. Production Review of Gene Therapy Market: It illustrates the production volume, capacity with respect to major Gene Therapy regions, application, type, and the price. Sales Margin and Revenue Accumulation Review of Gene Therapy Market: Eventually explains sales margin and revenue accumulation based on key regions, price, revenue, and Gene Therapy target consumer. Supply and Demand Review of Gene Therapy Market: Coupled with sales margin, the report depicts the supply and demand seen in major regions, among key players and for every Gene Therapy product type. Also interprets the Gene Therapy import/export scenario. Other key reviews of Gene Therapy Market: Apart from the above information, correspondingly covers the company website, number of employees, contact details of major Gene Therapy players, potential consumers and suppliers. Also, the strengths, opportunities, Gene Therapy market driving forces and market restraints are studied in this report.

Highlights of Global Gene Therapy Market Report:* This report provides in detail analysis of the Gene Therapy and provides market size (US$ Million) and Cumulative Annual Growth Rate (CAGR (%)) for the forecast period: 2019 ; 2029. * It also elucidates potential revenue opportunity across different segments and explains attractive investment proposition matrix for world Gene Therapy market. * This study also provides key insights about Gene Therapy market drivers, restraints, opportunities, new product launches, approvals, regional outlook, and competitive strategies adopted by the leading Gene Therapy players. * It profiles leading players in the worldwide Gene Therapy market based on the following parameters ; company overview, financial performance, product portfolio, geographical presence, distribution strategies, key developments and strategies and future plans. * Insights from Gene Therapy report would allow marketers and management authorities of companies to make an informed decision with respect to their future product launches, market expansion, and Gene Therapy marketing tactics. * The world Gene Therapy industry report caters to various stakeholders in Gene Therapy market. That includes investors, device manufacturers, distributors and suppliers for Gene Therapy equipment. Especially incorporates government organizations, Gene Therapy research and consulting firms, new entrants, and financial analysts. *Various strategy matrices used in analyzing the Gene Therapy market would provide stakeholders vital inputs to make strategic decisions accordingly.

Global Gene Therapy Market Report Provides Comprehensive Analysis of Following: ; Gene Therapy Market segments and sub-segments ; Industry size ; Gene Therapy shares ; Gene Therapy Market trends and dynamics ; Market Drivers and Gene Therapy Opportunities ; Supply and demand of world Gene Therapy industry ; Technological inventions in Gene Therapy trade ; Gene Therapy Marketing Channel Development Trend ; Global Gene Therapy Industry Positioning ; Pricing and Brand Strategy ; Distributors/Traders List enclosed in Positioning Gene Therapy Market.

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Moreover, the report organizes to provide essential information on current and future Gene Therapy market movements, organizational needs and Gene Therapy industrial innovations. Additionally, the complete Gene Therapy report helps the new aspirants to inspect the forthcoming opportunities in the Gene Therapy industry. Investors will get a clear idea of the dominant Gene Therapy players and their future forecasts.

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Gene Therapy Market : Facts, Figures and Analytical Insights 2020 2029 - 3rd Watch News

The global analysis of Gene Therapy for Age-related Macular Degeneration Market and its upcoming prospects have recently added by HealthCare Intelligence Markets to its extensive repository. It has been employed through the primary and secondary research methodologies. This market is expected to become competitive in the upcoming years due to the new entry of a number of startups in the market. Additionally, it offers effective approaches for building business plans strategically which helps to promote control over the businesses.

This report highlights the business cost structure that includes cost of raw material as well as manpower. It throws light on various internal and external factors that are driving or restraining the Gene Therapy for Age-related Macular Degeneration Market. To enlarge the businesses rapidly, it offers some significant ways to discover the wide-ranging global opportunities.

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Reportedly, by anlayzing the increasing demand and desirable resources estimated by the remarkable vendors, the worldwide Gene Therapy for Age-related Macular Degeneration industry has been assessed and predicts the upcoming industrial growth rates of the market. Besides this, the report on the Gene Therapy for Age-related Macular Degeneration Market segments the global market into distinct categories like product types, applications, topological regions, well-established industry players.

Major Key Vendors: RetroSense Therapeutics, REGENXBIO, AGTC

Competitive landscape of global Gene Therapy for Age-related Macular Degeneration Market has been studied to understand the competitive products and services across the globe. For effective global regional outlook analysts of the report examines global regions such as, North America, Latin America, Japan, Asia-Pacific, and India on the basis of productivity.

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Finally, all aspects of the Global Gene Therapy for Age-related Macular Degeneration Market are quantitatively as well qualitatively assessed to study the Global as well as regional market comparatively. This market study presents critical information and factual data about the market providing an overall statistical study of this market on the basis of market drivers, limitations and its future prospects. The report supplies the international economic competition with the assistance of Porters Five Forces Analysis and SWOT Analysis.

Prime Objectives that are Covered In this Report are as follows:

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Explore Trending Report On Gene Therapy for Age-related Macular Degeneration Market Along With COVID-19 Updates, Trend, Scope, CAGR, Forecast Till...

The market report examined the current COVID-19 situation in the Cancer Gene Therapy Industry and the segments future prospects thoroughly. Furthermore, key market strategies are discussed, including product developments, partnerships, mergers and acquisitions, etc. Research study helps to perceive the real condition, major players and drivers in the market. It also analyzes demand from up-stream raw materials and equipment. This report includes a thorough study of the Cancer Gene Therapy Market using SWOT analysis, The Cancer Gene Therapy Market Report also provides a comprehensive analysis of key market players based on the organizations goals such as production quantity, profiling, product outline, the organizations required raw materials, and others growth factors .

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Covid-19 Impact on the Global Cancer Gene Therapy Market:

Cancer Gene Therapy Market Report provides an overview of the market based on key parameters such as market size, sales, sales analysis and key drivers. The market size of the market is expected to grow on a large scale during the forecast period (2019-2026). This report covers the impact of the latest COVID-19 on the market. The coronavirus epidemic (COVID-19) has affected all aspects of life around the world. This has changed some of the market situation. The main purpose of the research report is to provide users with a broad view of the market. Initial and future assessments of rapidly.

Cancer Gene Therapy MarketCompetitive Analysis:

Leading market players Genelux Corporation, Cell Genesys, Advantagene, GenVec, BioCancell, Celgene, Epeius Biotechnologies, Introgen Therapeutics, Ziopharm Oncology, Shenzhen SiBiono GeneTech, and Altor Bioscience., Provided in this report. These players have adopted various strategies including expansions, mergers & acquisitions, joint ventures, new product launches, and collaborations to gain a strong position in the industry.

The Covid-19 (corona virus) pandemic is impacting society and the overall economy across the world. The impact of this pandemic is growing day by day as well as affecting the supply chain. The COVID-19 crisis is creating uncertainty in the stock market, massive slowing of supply chain, falling business confidence, and increasing panic among the customer segments. The overall effect of the pandemic is impacting the production process of several industries, and many more. Trade barriers are further restraining the demand- supply outlook. As government of different regions have already announced total lockdown and temporarily shutdown of industries, the overall production process being adversely affected; thus, hinder the overall Cancer Gene Therapy market globally.

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COVID-19 Scenario:

Cancer Gene Therapy MarketSegmentation:

The research offers a detailed segmentation of the global Cancer Gene Therapy market. Key segments analyzed in the research by Product, by Type, by End User and geography. Extensive analysis of sales, revenue, growth rate, and market share of each for the historic period and the forecast period is offered with the help of tables.

Cancer Gene Therapy MarketRegional Analysis:

The market is analyzed based on regions and competitive landscape in each region is mentioned. Regions discussed in the study include North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India and Southeast Asia), South America (Brazil, Argentina, and Colombia), Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa). These insights help to devise strategies and create new opportunities to achieve exceptional results.

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Cancer Gene Therapy Market The New Ways to Win in Emerging Markets In COVID-19 Crisis Forecast to 2020-2027 with GSK, Bluebird bio, Merck, Celgene,...