Novartis Launches Initiative to Supply COVID-19 Therapies to Low and Middle-Income Countries – BioSpace

Switzerlands Novartis launched an initiative to assist patients in low-income and lower-middle-income countries in getting access to affordable drugs for COVID-19. Specifically, they are making 15 drugs from its Sandoz division available that can be used to treat symptoms related to COVID-19. They are drugs for gastrointestinal illness, acute respiratory symptoms, pneumonia and septic shock.

Novartis plans to make the drugs available to government, non-governmental organizations (NGOs) and other institutional customers in up to 79 countries at no profit. In addition, the initiative will allow the countries to pick therapies in the portfolio that they need. The primary eligibility requirement is that the countries are on the World Banks list of low-income and lower-middle-income (LIC; LMIC) countries.

Access to medicine can be a challenge for patients in low- and lower-middle-income countries and the situation has worsened during COVID-19, said Lutz Hegemann, chief operating officer for Global Health at Novartis. With our COVID-19 portfolio, we wish to help address the additional healthcare demands of the pandemic in the countries we are targeting.

The drugs included in the Novartis COVID-19 Response Portfolio are: Amoxicillin, Ceftriaxone, Clarithromycin, Colchicine, Dexamethasone, Dobutamine, Fluconazole, Heparin, Levofloxacin, Loperamide, Pantoprazole, Prednisone, Prednisolone, Salbutamol and Vancomycin.

The company indicates the portfolio is in addition to the Novartis Access portfolio, which are on- and off-patent medicines against key non-communicable diseases, by way of the local Novartis or Sandoz affiliate.

According to Reuters, Novartis initiative caused the NGO Doctors Without Borders to call for more drug pricing transparency and for the biopharmaceutical industry to follow no profiteering initiatives for new COVID-19 therapies.

Hegemann told Reuters that the company has not had supply chain problems for these drugs, but the focus was on ensuring that the vulnerable healthcare systems in Africa, Asia, South America and Eastern Europe didnt become overwhelmed.

We shouldnt underestimate the stress that COVID puts particularly on fragile health system, Hegemann told Reuters. He said the company planned to work with health authorities, faith-based organizations and NGOs to eliminate markups on drugs. We are not targeting classical commercial distribution channels, but very direct channels.

For the most part, Novartiss branded drugs arent commonly used to treat COVID-19, and its malaria drug hydroxychloroquine has had very mixed results in clinical trials for the disease. But Sandoz is the Swiss companys generics division and has many drugs that are used to treat symptoms of COVID-19 in patients in the hospital. They are typically steroids, antibiotics, an antifungal and lung medication. Most of them have been around for decades and are relatively inexpensive to manufacture.

Novartis should publish the actual at cost prices for these medicines, as well as any costs of R&D and costs of production for all of their medicines, a spokesperson for Doctors Without Borders said. Additionally, we hope that corporations like Novartis will follow similar no profiteering initiatives for any new COVID-19 products.

To date, Novartis has donated $40 million in support of communities globally impacted by the pandemic. They are also involved in two cross-industry research programs, the COVID-19 Therapeutics Accelerator, coordinated by the Bill & Melinda Gates Foundation, Wellcome Trust, and Mastercard, and a COVID-19 directed partnership organized by the Innovative Medicines Initiative (IMI).

It is still supplying hydroxychloroquine for investigator-initiated clinical trials and when governments require it, as appropriate. It is also supporting clinical trials of several of its drugs for COVID-19. In addition, AveXis, its gene therapy unit, inked a manufacturing agreement with Massachusetts Eye and Ear and Massachusetts General Hospital to manufacture its novel genetic COVID-19 vaccine candidate, AAVCOVID.

This initiative builds on our earlier global commitment to keep prices stable for a basket of essential drugs used to treating COVID-19 patients, said Richard Saynor, chief executive officer of Sandoz. The COVID-19 Response Portfolio for low-income and lower-middle-income countries is designed to support governments in treating COVID-19 symptoms before they lead to complications in patients.

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Novartis Launches Initiative to Supply COVID-19 Therapies to Low and Middle-Income Countries - BioSpace

Biotech Firms to Track and Monitor Patients Injected with Stem Cells or Gene Therapy Treatments – The Korea Bizwire

The new business entities should stop the supply and sale of human cells once they are verified to be harmful to human bodies. (Yonhap)

SEOUL, July 16 (Korea Bizwire) New government regulations will require South Korean biotech companies to track and monitor patients who are injected with stem cells or gene therapy treatments.

According to the Ministry of Food and Drug Safety on Wednesday, the Advanced Regenerative Bio Act, enacted to strengthen the safety management of advanced regenerative drugs as well as to facilitate their commercialization through rapid clinical studies, will take effect on Aug. 28.

Under this law, a new type of business called human cell management will be born, which will clear the way for professional management of human and animal cells and tissues in addition to animal organs.

During the process of operation, the new business entities should stop the supply and sale of human cells once they are verified to be harmful to human bodies.

In such cases, all of the human cells concerned should be withdrawn and disposed of.

The law will make it mandatory for biotech companies to register stem cell and gene therapy treatment injection data with the Korea Institute of Drug Safety & Risk Management.

They will also be obliged to report unusual cases and perform long-term tracking and monitoring of the patients.

Advanced regenerative medicine refers to the medical technology of curing, replacing or regenerating damaged cells or issues using human cells.

Kevin Lee (kevinlee@koreabizwire.com)

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Biotech Firms to Track and Monitor Patients Injected with Stem Cells or Gene Therapy Treatments - The Korea Bizwire

Eli Lilly’s mirikizumab joins the club beating up on Cosentyx as another head-to-head study leaves Novartis’ drug in the dust – Endpoints News

A day after Moderna and the NIH published much-anticipated data from their Phase I Covid-19 vaccine trial, attention is turning to AstraZeneca which, according to a UK report, is expected to publish its own early data tomorrow.

ITVs Robert Peston reported that AstraZeneca will publish the Phase I data inThe Lancet.

AstraZeneca and Moderna represent the two most ambitious Covid-19 vaccine efforts, having set the quickest timelines for approval (though they were recently joined in that regard by the Pfizer-BioNTech partnership) and some of the loftiest goals in total doses. Yet there is even less known about AstraZenecas vaccines effect on humans than there was about Modernas before yesterday. Although, in a controversial move, Moderna released some statistics from its Phase I in May, AstraZeneca has yet to say anything about what it saw in its Phase I trial a move consistent with the scientific convention to withhold data until it can be published in a peer-reviewed journal.

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Eli Lilly's mirikizumab joins the club beating up on Cosentyx as another head-to-head study leaves Novartis' drug in the dust - Endpoints News

Gene Therapy for Inherited Genetic Disorders Market : Report analyzes the segments and provides the relative contribution to the development – Jewish…

Global Gene Therapy for Inherited Genetic Disorders market- Report defines the vital growth factors, opportunities and market segment of top players during the forecast period from 2019 to 2025. The report Gene Therapy for Inherited Genetic Disorders offers a complete market outlook and development rate during the past, present, and the forecast period, with concise study, Gene Therapy for Inherited Genetic Disorders market effectively defines the market value, volume, price trend, and development opportunities. The comprehensive, versatile and up-to-date information on Gene Therapy for Inherited Genetic Disorders market is provided in this report.

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segment by Type, the product can be split intoEye DisordersHematological DisordersCentral Nervous System DisordersMuscular DisordersOthersMarket segment by Application, split intoHospitalClinicResearch InstituteOthers

Market segment by Regions/Countries, this report coversNorth AmericaEuropeChinaJapanSoutheast AsiaIndiaCentral & South America

The study objectives of this report are:To analyze global Gene Therapy for Inherited Genetic Disorders status, future forecast, growth opportunity, key market and key players.To present the Gene Therapy for Inherited Genetic Disorders development in North America, Europe, China, Japan, Southeast Asia, India and Central & South America.To strategically profile the key players and comprehensively analyze their development plan and strategies.To define, describe and forecast the market by type, market and key regions.

In this study, the years considered to estimate the market size of Gene Therapy for Inherited Genetic Disorders are as follows:History Year: 2015-2019Base Year: 2019Estimated Year: 2020Forecast Year 2020 to 2026For the data information by region, company, type and application, 2019 is considered as the base year. Whenever data information was unavailable for the base year, the prior year has been considered.

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Complete Analysis of the Gene Therapy for Inherited Genetic Disorders Market:

Comprehensive assessable analysis of the industry is provided for the period of 2019-2025 to help investors to capitalize on the essential market opportunities.

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Furthermore, Global Gene Therapy for Inherited Genetic Disorders Market following points are involved along with a detailed study of each point:

Generation of this Global Gene Therapy for Inherited Genetic Disorders Industry is tested about applications, types, and regions with price analysis of players that are covered.

Revenue, sales are planned for this Gene Therapy for Inherited Genetic Disorders market, including with various essentials along yet another facet is assessed in this section for foremost regions.

In continuation using earnings, this section studies consumption, and global Gene Therapy for Inherited Genetic Disorders market. This area also sheds light on the variance between ingestion and distribution. Export and Gene Therapy for Inherited Genetic Disorders significance data are provided in this part.

In this section, key players have been studied depending on product portfolio, their Gene Therapy for Inherited Genetic Disorders market company profile, volume, price, price, and earnings.

Gene Therapy for Inherited Genetic Disorders market analysis aside from business, the information, and supply, contact information from manufacturers, consumers and providers can also be presented. Additionally, a feasibility study to asset and SWOT analysis for endeavors have been contained.

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Gene Therapy for Inherited Genetic Disorders Market : Report analyzes the segments and provides the relative contribution to the development - Jewish...

Impact of COVID-19 on Cell and Gene Therapy Market In-depth Study And Forecast to 2027 | Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb…

Coherent Market Insights has recently published the Global research Report Titled Cell and Gene Therapy Market. The study provides an overview of current statistics and future predictions of the Cell and Gene Therapy Market. The study highlights a detailed assessment of the Market and displays market sizing trends by revenue & volume (if applicable), current growth factors, expert opinions, facts, and industry validated market development data.

The Global Cell and Gene Therapy Market research report assembles data collected from different regulatory organizations to assess the growth of the segments. In addition, the study also appraises the global market on the basis of topography. It reviews the macro- and microeconomic features influencing the growth of the in each region. Various methodological tools are used to analyze the growth of the worldwide Cell and Gene Therapy Market.

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Prominent Key Players Covered in the report:

Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST.

Detailed Segmentation:

By Therapy Type:Cell TherapyStem CellsT CellsDendritic CellsNK CellsTumor CellsGene TherapyGlobal Cell and Gene Therapy Market, By Indication:Cardiovascular DiseaseCancerGenetic DisorderInfectious DiseaseNeurological DisordersOthers

Major Regions as Follows:

North America (USA, Canada and Mexico)

Europe (Germany, France, the United Kingdom, Netherlands, Russia , Italy and Rest of Europe)

Asia-Pacific (China, Japan, Australia, New Zealand, South Korea, India and Southeast Asia)

South America (Brazil, Argentina, Colombia, rest of countries etc.)

Middle East and Africa (Saudi Arabia, United Arab Emirates, Israel, Egypt, Nigeria and South Africa)

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Cell and Gene Therapy Market share (regional, product, application, end-user) both in terms of volume and revenue along with CAGR

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TABLE OF CONTENTS

Part 01: Executive Summary

Part 02: Scope of the Report

Part 03: Research Methodology

Part 04: Market Landscape

Part 05: Pipeline Analysis

Part 06: Market Sizing

Part 07: Five Forces Analysis

Part 08: Market Segmentation

Part 10: Regional Landscape

Part 11: Decision Framework

Part 12: Drivers and Challenges

Part 13: Market Trends

Part 14: Vendor Landscape

Part 15: Vendor Analysis

Part 16: Appendix

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Impact of COVID-19 on Cell and Gene Therapy Market In-depth Study And Forecast to 2027 | Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb...

Personalized Gene Therapy Treatments For Cancer Consumption Market Size, Share & Trends Analysis Report with Top Players Adaptimmune Ltd.,…

The Global Personalized Gene Therapy Treatments For Cancer Consumption Market provides up-to-date information on current and future industry trends, enabling readers to identify products and services to increase revenue growth and profitability. This research report provides in-depth study of all key factors affecting global and regional markets, including drivers, imprisonment, threats, challenges, opportunities and industry-specific trends. This report cites worldwide trust and specimens with leading players downstream and upstream analysis.

Personalized cancer therapy is a treatment strategy centered on the ability to predict which patients are more likely to respond to specific cancer therapies. This approach is founded upon the idea that tumor biomarkers are associated with patient prognosis and tumor response to therapy.

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This market research report on analyzes the growth prospects for the key vendors operating in this market space including Adaptimmune Ltd., Advantagene, Inc., Amgen, Inc., Bellicum Pharmaceuticals, Inc., BioCancell Ltd., Blubird Bio, Celsion Corporation, Chengdu Shi Endor Biological, Engineering Technology Co., Ltd., Cold Genesys, Inc., Genprex, Inc., MaxiVax SA, Momotaro-Gene, Inc., MultiVir, Inc., OncoSec Medical, Inc., Sevion Therapeutics, Inc., SynerGene Therapeutics, Inc., Takara Bio, Inc., Tocagen, Inc., VBL Therapeutics Ltd.

The global Personalized Gene Therapy Treatments For Cancer Consumption Market is segmented on the basis of the type of product, application, and region. The analysts authoring the report provide an exact evaluation of all of the segments included in the report. The segments are studied keeping in view their market share, revenue, market growth rate, and other vital factors. The segmentation study equips interested parties to identify high-growth portions of the global market and understand how the leading segments could grow during the forecast period.

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Personalized Gene Therapy Treatments For Cancer Consumption Market Size, Share & Trends Analysis Report with Top Players Adaptimmune Ltd.,...

Tissue Engineering Gene Therapy Industry Market Overview with Detailed Analysis, Competitive landscape, Forecast to 2025 – 3rd Watch News

The Tissue Engineering Gene Therapy Industry market research report added by Market Study Report, LLC, is an in-depth analysis of the latest trends persuading the business outlook. The report also offers a concise summary of statistics, market valuation, and profit forecast, along with elucidating paradigms of the evolving competitive environment and business strategies enforced by the behemoths of this industry.

The Tissue Engineering Gene Therapy Industry market report is an exhaustive assessment of this business space and provides crucial insights with regards to development trends of the market during the estimated timeframe. Additional details including regional scope of the industry and various policies are outlined in the report. Moreover, the report encompasses numerous parameters such as the impact of current market trends on investors.

Details concerning the Tissue Engineering Gene Therapy Industry market scenario such as advantages and disadvantages of products launched by industry players are mentioned in the report. The study further offers a summary of the competitive scenario as well as a complete analysis of the raw material and downstream buyers matrix.

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COVID-19, the disease it causes, surfaced in late 2020, and now had become a full-blown crisis worldwide. Over fifty key countries had declared a national emergency to combat coronavirus. With cases spreading, and the epicentre of the outbreak shifting to Europe, North America, India and Latin America, life in these regions has been upended the way it had been in Asia earlier in the developing crisis. As the coronavirus pandemic has worsened, the entertainment industry has been upended along with most every other facet of life. As experts work toward a better understanding, the world shudders in fear of the unknown, a worry that has rocked global financial markets, leading to daily volatility in the U.S. stock markets.

Revealing a gist of the competitive landscape of Tissue Engineering Gene Therapy Industry market:

Explicating the geographical landscape of Tissue Engineering Gene Therapy Industry market:

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Additional takeaways from the Tissue Engineering Gene Therapy Industry market report:

Research Objective:

This report considers the below mentioned key questions:

Q.1. What are some of the most favorable, high-growth prospects for the global Tissue Engineering Gene Therapy Industry market?

Q.2. Which products segments will grow at a faster rate throughout the forecast period and why?

Q.3. Which geography will grow at a faster rate and why?

Q.4. What are the major factors impacting market prospects? What are the driving factors, restraints, and challenges in this Tissue Engineering Gene Therapy Industry market?

Q.5. What are the challenges and competitive threats to the market?

Q.6. What are the evolving trends in this Tissue Engineering Gene Therapy Industry market and reasons behind their emergence?

Q.7. What are some of the changing customer demands in the Tissue Engineering Gene Therapy Industry Industry market?

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Gene therapy or immunotherapy: which approach is more likely to deliver a cure for HIV? – aidsmap

Amidst speculation that a five-drug antiretroviral regimen and nicotinamide might have cured HIV in one man, researchers debated whether gene therapy or immunotherapy is more likely to lead to an HIV cure that can be delivered to millions during the AIDS 2020 Cure pre-conference last week.

A cure for HIV could take two forms, either a treatment or procedure that can eradicate the virus from the body or one which can keep the virus under control without the need for antiretroviral drugs remission, in the parlance of the field.

Eradication of HIV is challenging because the virus inserts its DNA into long-lived cells in the body where it may lie dormant for decades the so-called HIV reservoir. All this virus needs to be found, activated and purged, but presentations at AIDS 2020 show that the reservoir is more complex than previously assumed.

A unit of heredity, that determines a specific feature of the shape of a living organism. This genetic element is a sequence of DNA (or RNA, for viruses), located in a very specific place (locus) of a chromosome.

A type of experimental treatment in which foreign genetic material (DNA or RNA) is inserted into a person's cells to prevent or fight disease.

Use of immunologic agents such as antibodies, growth factors, and vaccines to modify (activate, enhance, or suppress) the immune system in order to treat disease. It is applied in the cancer field and in HIV research (attempts to eliminate the virus). Immunotherapy is also used to diminish adverse effects caused by some cancer treatments or to prevent rejection of a transplanted organ or tissue.

To eliminate a disease or a condition in an individual, or to fully restore health. A cure for HIV infection is one of the ultimate long-term goals of research today. It refers to a strategy or strategies that would eliminate HIV from a persons body, or permanently control the virus and render it unable to cause disease. A sterilising cure would completely eliminate the virus. A functional cure would suppress HIV viral load, keeping it below the level of detection without the use of ART. The virus would not be eliminated from the body but would be effectively controlled and prevented from causing any illness.

The HIV reservoir is a group of cells that are infected with HIV but have not produced new HIV (latent stage of infection) for many months or years. Latent HIV reservoirs are established during the earliest stage of HIV infection. Although antiretroviral therapycan reduce the level of HIV in the blood to an undetectable level, latent reservoirs of HIV continue to survive (a phenomenon called residual inflammation). Latently infected cells may be reawakened to begin actively reproducing HIV virions if antiretroviral therapy is stopped.

HIV is distributed across numerous tissues in the body, not just cells in the blood or lymph nodes, an autopsy study by the US National Institutes for Allergy and Infectious Disease shows. Predicting which tissues are the most important reservoirs is difficult, as the small study showed big variation between individuals.

Furthermore, the normal work of CD4 memory cells activation and proliferation in response to pathogens inevitably leads to cloning of cells containing HIV DNA and an increase in intact HIV DNA capable of producing new virus over time, Bethany Horsburgh of Australias Centre for Virus Research at Westmead Institute for Medical Research reported.

Even very early antiretroviral treatment appears unable to halt the development of a reservoir that can sustain SIV infection in the body, Dr Henintsoa Rabazantahary of Canadas Universit Laval told the conference. Her macaque study began treating some animals four days after infection, underscoring how quickly an intractable reservoir is established.

These findings emphasise the importance of approaches to curing HIV that go beyond the `shock and kill` regimens designed to activate HIV-infected cells, which have shown disappointing results in clearing the reservoir.

Gene therapy to eradicate HIV or immunotherapy to contain HIV are being explored as potential approaches but which is more likely to be successful? Two leading cure researchers debated the merits of the approaches at a pre-conference HIV cure workshop last week.

Professor Sharon Lewin, Director of the Doherty Institute of Infection and Immunity at the University of Melbourne argues that gene therapy is more likely to deliver an HIV cure than immunotherapeutic approaches aimed at long-term remission of HIV. Proof of concept for a gene therapy approach already exists, she said, in the form of the Berlin and London patients, Timothy Brown and Adam Castellijo, who were cured of HIV after stem cell transplants from donors with the CCR5 delta 32 mutation that confers resistance to HIV infection of cells.

Gene therapy can be used against multiple targets to engineer protection against HIV infection of cells, to purge the virus from infected cells and enhance immune defences that attack HIV.

But the big challenge for gene therapy is to develop an approach that doesnt require cells to be taken out of the body for gene editing in the laboratory. Almost all gene therapy studies underway are using this 'ex vivo' approach, which harvests cells, edits them in the laboratory and then returns them to the patients body. Although the ex vivo approach has already been proved to work, both for HIV and cancer immunotherapy using CAR T-cells, its expensive and requires state of the art laboratory equipment.

The alternative, in vivo gene therapy, would require nanoparticles or a vector such as adenovirus to deliver the edited gene to cells. One study has already shown that its possible to achieve sustained production of a broadly neutralising antibody against HIV, VRC07, using an adenovirus vector to deliver an antibody gene.

Elimination of host stem cells, achieved in the cases of the Berlin and London patients through gruelling chemotherapy prior to bone marrow transplants, might soon be achievable through antibodies-drug conjugates that would target stem cells, Lewin suggested.

Professor John Frater of the University of Oxford sees immunotherapy as more likely to deliver long-term remission. He argued that gene therapy is still largely unproven in any field and the long-term safety of gene therapy is still unclear. In contrast, immunotherapies are already being used to treat cancers such as melanoma and lymphoma, as well as rheumatoid arthritis. Elite controllers of HIV, or long-term non-progressors, also offer evidence that the immune system can control HIV in some circumstances.

Immunity is the best machine you could imagine its had millions of years of R & D so we should use it and make the most of it, he said. Do not confuse the failure of vaccines so far as a red flag for immunotherapy. A vaccine needs to target a rapidly mutating, fast-replicating virus, whereas an immunotherapy targets a stable antigen that is less prone to mutate the cells in the HIV reservoir. We need to think of it more like a strategy for cancer than infection, he said.

Broadly neutralising antibodies represent one promising avenue of immunological research, along with therapeutic vaccination or anti-PD1 to activate exhausted host defences, Professor Miles Davenport of the Kirby Institute of Immunity & Infection, Australia, told a symposium on emerging cure strategies.

But he warned that we still dont understand how immune control relates to viral rebound and how much the HIV reservoir might need to be reduced to make immunological control of HIV viable. What might overcome this challenge, he suggested, would be gene therapy approaches that could render 90% of cells resistant to infection. Modelling by his research group suggests that this level of transduction of cells would dramatically limit viral rebound, permitting immunological control of HIV.

In summary, it may not be a question of choosing between gene therapy or immunotherapy, but using both approaches to achieve HIV remission.

References

Rabezanahary H et al. Contribution of monocytes and CD4 T cell subsets in maintaining viral reservoirs in SIV-infected macaques treated early after infection with antiretroviral drugs. 23rd International AIDS Conference, abstract OA004, 2020.

Horsburgh H et al. Cell proliferation contributes to the increase of genetically intact HIV over time. 23rd International AIDS Conference, abstract OA005, 2020.

Imamichi H et al. Multiple sanctuary sites for intact and defective HIV-1 in post-mortem tissues in individuals with suppressed HIV-1 replication: Implications for HIV-1 cure strategies. 23rd International AIDS Conference, abstract 0A006, 2020.

S Lewin & J Frater. Gene therapy vs. immunotherapy: which is more likely to work? Debate. AIDS 2020: Virtual, HIV Cure pre-conference.

Davenport M. The promise of immunotherapy in HIV infection. AIDS 2020: Virtual symposium presentation, 'Pushing the boundaries: new approaches to a cure'.

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Gene therapy or immunotherapy: which approach is more likely to deliver a cure for HIV? - aidsmap

PreveCeutical advances Dual Gene Therapy Research Program combatting Type 2 diabetes and obesity – Proactive Investors USA & Canada

The company has identified a promising modified delivery vector based on stable small interfering RNA

PreveCeutical Medical Inc (CSE:PREC) () announced Wednesday that it has successfully demonstrated that the delivery efficiency of its diabetes-targeting Dual Gene Therapy Research Program could be tailored based on vector composition.

The company has identified a promising modified delivery vector based on stable small interfering RNA (siRNA) for possible preclinical experiments. The iRNA gets incorporated within the delivery vehicle, with strong cell entry and cargo release, and minimal toxicity.

These delivery systems have the potential to combine the benefits of silencing RNAi (ribonucleic acid interference) and modified delivery vectors, the company said.

The current results PreveCeutical has received is very exciting to us and shows a very high probability of success that will now allow us to move to the completion stage of the program, CEO Stephen Van Deventer said in a statement.

From here, PreveCeutical plans to proceed with the optimization of the delivery system that will be tested in animal models.

Ultimately, the idea is to down-regulate a particular gene of interest, which impacts Type 2 diabetes and obesity.

PreveCeutical is a health sciences company that develops innovative options for preventive and curative therapies utilizing organic and nature identical products.

The company's current research and development programs include dual gene curative and preventive therapies for diabetes and obesity; the cannabidiols sol-gel program aiming to provide relief across a range of indications from pain, inflammation, seizures, and neurological disorders; Nature Identical peptides for the treatment of various ailments; non-addictive analgesic peptides as a replacement to the highly addictive analgesics such as morphine, fentanyl and oxycodone; and a therapeutic product for treating athletes who suffer from concussions (mild traumatic brain injury).

Contact Andrew Kessel at [emailprotected]

Follow him on Twitter @andrew_kessel

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PreveCeutical advances Dual Gene Therapy Research Program combatting Type 2 diabetes and obesity - Proactive Investors USA & Canada

Research Report on Cell and Gene Therapy Market by Current Industry Status, Growth Opportunities, Top Key Players, and Forecast to 2025 – AlgosOnline

The Cell and Gene Therapy Market Report renders deep perception of the key regional market status of the Cell and Gene Therapy industry on a global level that primarily aims at core regions which comprises of continents like Europe, North America, and Asia and the key countries such as United States, Germany, China and Japan.

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The Cell and Gene Therapy market report is a trove of information pertaining to the various aspects of this industry space. Encompassing the ongoing as well as forecast trends likely to fuel the business graph of the Cell and Gene Therapy market across various geographies, the report also provides details about the driving factors that would help propel this industry to new heights during the projected period. Alongside a collection of the driving parameters, the Cell and Gene Therapy market reports also include a spate of other dynamics pertaining to the industry, such as the nominal risks prevailing in this marketplace as well as the growth prospects that this business sphere has in the future.

A short outline of the pointers encompassed in the Cell and Gene Therapy market scope:

The report provides substantial data pertaining to the market share that every company holds currently in the industry, as well as the market share that each of these firms is anticipated to accrue by the end of the forecast timeframe. As per the study, the Cell and Gene Therapy market, pertaining to the regional hierarchy, is segregated into Amgen Inc., Novartis AG, Fibrocell Science, Inc., bluebird bio, Inc., Kolon TissueGene, Inc., Dendreon Pharmaceuticals LLC., Organogenesis Holdings Inc., Kite Pharma, Inc., Human Stem Cells Institute, Orchard Therapeutics plc., Spark Therapeutics, Inc., Pfizer, Inc., Vericel Corporation, Sibiono GeneTech Co. Ltd.,, RENOVA THERAPEUTICS, ViroMed Co., Ltd., Shanghai Sunway Biotech Co. and Ltd. The report expounds on the information pertaining to the products manufactured by these companies, that would help new participants and other major stakeholders work on their product portfolio strategies.

Questions answered by the Cell and Gene Therapy market report with regards to the geographical spectrum of the business:

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What questions does the Cell and Gene Therapy market study answer with regards to the industry segmentation?

A few other pivotal pointers encompassed in the report include market competition trends, industry concentration rate, and details about the sales channels deployed by prominent sellers.

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Research Report on Cell and Gene Therapy Market by Current Industry Status, Growth Opportunities, Top Key Players, and Forecast to 2025 - AlgosOnline

Rocket Pharmaceuticals Announces First Patient Treated in Phase 1 Trial of RP-L301 Gene Therapy for Pyruvate Kinase Deficiency – Business Wire

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the first patient has received investigational therapy in the open-label, Phase 1 clinical trial of RP-L301, the Companys lentiviral vector (LVV)-based gene therapy for the treatment of Pyruvate Kinase Deficiency (PKD), a rare monogenic red blood cell disorder.

We are pleased to have treated the first patient in our Phase 1 trial of RP-L301, marking an important step forward in addressing a high unmet need for new therapies, said Jonathan D. Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket. PKD is a genetic disorder characterized by red cell destruction and anemia that can be severe or even life-threatening. Children are often most severely affected, and the current treatment optionschronic transfusions and splenectomyare associated with burdensome side effects including iron overload and end-organ damage. We believe gene therapy treatment with RP-L301 has the potential to be a safe and transformative approach to improve long-term patient outcomes.

The global Phase 1 open-label, single-arm, clinical trial is expected to enroll six adult and pediatric transfusion-dependent PKD patients in the U.S. and Europe. The trial will be comprised of three cohorts to assess RP-L301 in young pediatric (age 8-11), older pediatric (age 12-17) and adult populations. The trial is designed to assess the safety, tolerability and preliminary efficacy of RP-L301, and initial safety evaluation will occur in the adult cohort before evaluation in pediatric patients. Lucile Packard Childrens Hospital Stanford is the lead site in the U.S. for adult and pediatric patients. Hospital Infantil Universitario Nino Jesus is the lead site in Europe for pediatrics and Hospital Universitario Fundacion Jimenez Diaz is the lead site in Europe for adult patients. Further information about the clinical program is available here.

About Pyruvate Kinase DeficiencyPyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 3,000 to 8,000 patients in the United States and the European Union. Children are the most commonly and severely affected subgroup of patients. Currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.

RP-L301 was in-licensed from the Centro de Investigaciones Energeticas, Medioambientales y Tecnologicas (CIEMAT), Centro de Investigacion Biomedica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz (IIS-FJD).

About Rocket Pharmaceuticals, Inc.Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking StatementsVarious statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding when clinical trial sites will resume normal business operations, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-Q for the quarter ended March 31, 2020, filed May 8, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made , and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces First Patient Treated in Phase 1 Trial of RP-L301 Gene Therapy for Pyruvate Kinase Deficiency - Business Wire

Global Gene Therapies for Rare Diseases Market Assessment Revenue (US$ Mn) Forecast Till 2028 – Press Release – Digital Journal

This press release was orginally distributed by SBWire

Pune, Maharshtra -- (SBWIRE) -- 07/15/2020 -- InsightAce Analytic Pvt. Ltd. announces the release of market assessment report on "Global Gene Therapies for Rare Diseases Market Assessment Revenue (US$ Mn) Forecast Till 2028"

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According to the latest research by InsightAce Analytic, the global gene therapies for rare diseases market size is valued at US$ XX Million and it is expected to reach US$ 11,441.6 Million in 2028, recording a promising CAGR of 21.7% during the period of 2019-2028. By region, North America dominates the market with share of 85.3% of global Gene Therapies for Rare Diseases market. US is the key market holding majority of market for gene therapies for rare diseases in the region.

Gene therapy is defined as an experimental technique which utilizes genes in order to prevent or treat any disease condition. Different approaches for testing gene therapies includes, replacement of mutated gene causing disease with a healthy gene, inactivating the mutated gene with incorrect functioning. With advantage of single dose treatment against rare diseases, gene therapy confers effective lifelong improvement as compared to conventional treatment options. The market for Gene Therapies for Rare Diseases is anticipated to grow during the forecast period, owing to growing number of pipelines for gene therapy along with increasing drug approvals and introduction of novel gene therapies for treatment of various rare diseases. In addition, well established approval pathways and guidance by regulatory agencies on the development of gene therapy is further boosting the market growth.

According to report published by alliance for regenerative medicine 2019, the number of gene therapy for rare disease in Phase-1 is approximately 61, 141 in Phase-2 and 22 in Phase-3. Approximately 79 percent of clinical trials on gene therapy are progressing in rare disease for rare cancers such as haematological malignancies, ovarian cancers, pancreatic cancers, lung cancers, glioblastoma, etc., followed by Haematology disorders such as hemophilia, sickle cell disease, thalassemia, Fanconi's anemia, etc. with 6 percent.

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The Gene Therapies for Rare Diseases market is fragmented with presence of many players that operates in research and development, natural Gene Therapies for Rare Diseases providers in local as well as international market.

Global gene therapies for rare diseases market reports covers numerous prominent players like 4d Molecular Therapeutics, LLC, Abeona Therapeutics Inc., Advaxis, Adverum Biotech, Aevi Genomic, Applied Genetic Technologies Corporation (AGTC), Alcyone Lifesciences, Allife Medical Science and Technology, Amarna Therapeutics, American Gene Technologies, Amgen, Amicus Therapeutics, Anchiano Therapeutics, AnGes MG, Apic Bio, Armata Pharmaceuticals, Arrowhead, Arthrogen, Asklepios, Astellas, AVROBIO, BCM Families Foundation, Beijing Northland, Benitec, Biogen, BioMarin, Biosidus, bluebird bio, Boryung Group, Brain Neurotherapeuticsy Bio, Celsion Corporation, Chiesi, CRISPR Therapeutics, CSL Behring, Daewoong Pharma, Dicerna, Editas Medicine, Eiger BioPharmaceuticals, Enzo Therapeutics, Esteve, Evox Therapeutics, Expression Therapeutics, Fibrocell, Flexion Therapeutics, Fortress Biotech, Freeline Therapeutics, Gene Biotherapeutics, Genenta Science, GeneQuine, Genethon, GenSight, Gilead, Gradalis, GSK, Hanugen, Helixmith, Herantis, ID Pharma, Immusoft, Inovio, Intellia Therapeutics, Intrexon, J&J, Juventas, Kolon Life Science, Krystal Biotech, Locana, LogicBio Therapeutics, Lysogene, Medigene, MeiraGTx, Miltenyi Biotec, Molecular Templates, Momotaro-Gene, Neuralgene, Novartis, OncoSec, Orchard Therapeutics, Oxford Biomedica, Pfizer, Prevail Therapeutics, PTC Therapeutics, REGENXBIO, Renova Therapeutics, Reyon Pharmaceutical, Roche, Rocket Pharma, Roivant, Sangamo Therapeutics, Sarepta Therapeutics, Seelos Therapeutics, Solid BioSciences, Sterna Biologicals, Sutura Therapeutics, SynerGene, Takara, Takeda, Talee Bio, Theragene Pharma, Transgene, Ultragenyx, uniQure, VBL Therapeutics, Vertex, Voyager Therapeutics, Xalud, Xenon, and Ziopharm.

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Market SegmentsGlobal Gene Therapies for Rare Diseases Market Based on Product Revenue (US$ Mn) Forecast 2018-2028oZolgensma oInvossa K oStrimvelis oNeovasculgen oGlybera oLuxturna oZynteglooOthers

Global Gene Therapies for Rare Diseases Market Based on Disease Type (US$ Mn) Forecast 2018-2028oHemophiliaoDuchenne Muscular DystrophyoAchromatopsia oCystic FibrosisoInherited Retinal DystrophyoFragile X SyndromeoFriedreich ataxiaoAlpha-1 Antitrypsin DeficiencyoParoxysmal Nocturnal HemoglobinuriaoRetinitis PigmentosaoOthers

Global Gene Therapies for Rare Diseases Market Based on Region Revenue (US$ Mn) Forecast 2018-2028Europe Revenue (US$ Mn) Forecast 2018-2028oU.K.oGermanyoFranceoItalyoSpainoRussiaoRest of Europe

North America Revenue (US$ Mn) Forecast 2018-2028oU.S.oCanada

Asia Pacific Revenue (US$ Mn) Forecast 2018-2028

Latin America Revenue (US$ Mn) Forecast 2018-2028

Middle East & Africa Revenue (US$ Mn) Forecast 2018-2028

Why should buy this report:To receive a comprehensive analysis of the prospects for global gene therapies for rare diseases marketTo receive industry overview and future trends gene therapies for rare diseases marketTo analyse the gene therapies for rare diseases market drivers and challengesTo get information on gene therapies for rare diseases market size value/revenue (US$ Mn) forecast till 2028Major Investments, Mergers & Acquisition in gene therapies for rare diseases industry

For More Information @ https://www.insightaceanalytic.com/report-details/global-gene-therapies-for-rare-diseases-market-assessment/?About UsInsightAce Analytic is a market research and consulting firm that enables clients to make strategic decisions. Our qualitative and quantitative market intelligence solutions inform the need for market and competitive intelligence to expand businesses. We help clients gain competitive advantage by identifying untapped markets, exploring new and competing technologies, segmenting potential markets and repositioning products. Our expertise is in providing syndicated and custom market intelligence reports with an in-depth analysis with key market insights in a timely and cost-effective manner.

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Global Gene Therapies for Rare Diseases Market Assessment Revenue (US$ Mn) Forecast Till 2028 - Press Release - Digital Journal

Global Gene Therapy for Age related Macular Degeneration Market will Witness Steady Growth Till 2027 Post COVID 19 Pandemic, Top Manufactures…

Global Gene Therapy for Age related Macular Degeneration Market analysis 2015-2027, is a research report that has been compiled by studying and understanding all the factors that impact the market in a positive as well as negative manner. Some of the prime factors taken into consideration are: various rudiments driving the market, future opportunities, restraints, regional analysis, various types & applications, Covid-19 impact analysis and key market players of the Gene Therapy for Age related Macular Degeneration market. nicolas.shaw@cognitivemarketresearch.com or call us on +1-312-376-8303.

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Global Gene Therapy for Age related Macular Degeneration Market: Product analysis: Subretinal, Intravitreal, Unspecified

Global Gene Therapy for Age related Macular Degeneration Market: Application analysis: Monotherapy, Combination Therapy

Major Market Players with an in-depth analysis: RetroSense Therapeutics, REGENXBIO, AGTC

The research is presented in such a way that it consists of all the graphical representations, pie charts and various other diagrammatic representations of all the factors that are used for the research. Gene Therapy for Age related Macular Degeneration market research report also provides information on how the industry is anticipated to provide a highly competitive analysis globally, revenues generated by the industry and increased competitiveness and expansions among various market players/companies.

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The global estimations of the market value, market information/definition, classifications of all the types & applications, overall threats & dips that can be assumed and many other factors which consist the overall market scenario and its happening globally along with the forthcoming years are compiled in the Gene Therapy for Age related Macular Degeneration market research report. Hence this report can serve as a handbook/model for the enterprises/players interested in the Gene Therapy for Age related Macular Degeneration Market as it consists all the information regarding the Gene Therapy for Age related Macular Degeneration market.

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About Us: Cognitive Market Research is one of the finest and most efficient Market Research and Consulting firm. The company strives to provide research studies which include syndicate research, customized research, round the clock assistance service, monthly subscription services, and consulting services to our clients. We focus on making sure that based on our reports, our clients are enabled to make most vital business decisions in easiest and yet effective way. Hence, we are committed to delivering them outcomes from market intelligence studies which are based on relevant and fact-based research across the global market.Contact Us: +1-312-376-8303Email: nicolas.shaw@cognitivemarketresearch.comWeb: https://www.cognitivemarketresearch.com/

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Global Gene Therapy for Age related Macular Degeneration Market will Witness Steady Growth Till 2027 Post COVID 19 Pandemic, Top Manufactures...

Hemophilia Gene Therapy Market Value Share, Analysis and Segments 2018 to 2028 – Cole of Duty

A recent study published on the Global Hemophilia Gene Therapy market offers an in-depth understanding of the general prospects of this market. Further, the overview of the major findings of this study together with the megatrends affecting the increase of the Hemophilia Gene Therapy market is emphasized in the study. The market introduction and definition is included to help our readers understand the fundamental concepts of the analysis on the Hemophilia Gene Therapy industry.

According to the report, the Hemophilia Gene Therapy marketplace is set to increase In a CAGR of ~XX% over the forecast period (20XX-20XX) and reach a value of ~US$XX towards the end of 2029. The regional commerce analysis along with the leading importers and exporters is included in the study. Additionally, the supply-demand analysis and the key developments in the Hemophilia Gene Therapy market are highlighted in the report.

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Important Findings of this Report

Segmentation Of this Hemophilia Gene Therapy Market

Competitive landscape

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Important Queries pertaining to the keyword market catered to in the report:

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Hemophilia Gene Therapy Market Value Share, Analysis and Segments 2018 to 2028 - Cole of Duty

Gene Therapy for Rare Disease Market 2020 BY KEY BUSINESS OPPORTUNITIES, EMERGING TECHNOLOGIES, KEY COMPANIES WITH SIZE AND SHARE FORECAST TO 2027 -…

(July 2020)The Gene Therapy for Rare Disease Market Report presents a comprehensive analysis of the market scope, structure, potential, fluctuations, and financial impacts. The report also elaborates on the proper assessment of Industry Size, Share, product & sales volume, revenue, and growth rate. It also includes authentic and trustworthy estimations considering these terms.

This Gene Therapy for Rare Disease report go ahead with a vital overview of the Key Industry. The study features the opportunity and Gene Therapy for Rare Disease Market trends that are impacted on the global market. Key Players around various regions and analysis of each industry dimensions are mentioned in this report. The report also contains an essential Gene Therapy for Rare Disease insight regarding the things which are driving and affecting the earnings of the market. The Gene Therapy for Rare Disease Report contains sections together side landscape which clarifies actions such as venture and acquisitions and mergers.

To learn more about this report, request a sample copy*

[The sample copy includes: Report Summary, Table of Contents, Segmentation, Competitive Landscape, Report Structure, Methodology.]

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Market Overview:

The Gene Therapy for Rare Disease Market has witnessed significant growth rates with noticeable CAGR for the last couple of decades. According to the report, the market is expected to grow more energetically during the forecast period and it can also impact the global economic structure with a higher revenue share. The market also influence the potential to affect its peers and parent market as the growth rate of the market is being accelerated by rising disposable incomes, growing product demand, changing consumption technologies, innovative products, and raw material affluence.

The major manufacturers covered in this report:

(Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and BioMarin Pharmaceutical Inc.)

Market segmentation:

The Gene Therapy for Rare Disease market was divided into a variety of essential sectors, including applications, types and regions. Each market segment is intensively studied in the report, taking into account market acceptance, value, demand and growth prospects. Segmentation analysis allows customers to customize their marketing approach to perform better orders for each segment and identify the most prospective customer base.

Lucrative Opportunities:

This report investigate the challenges in front of the global metal complex as the study listed every one of them. This granted understanding of the market and benefits from any lucrative opportunities that are available. Researchers have provided an exhaustive study of the current market scenario while concentrating on the new business objectives. There is a comprehensive analysis of the change in customer requirements, customer preferences, and the vendor landscape of the overall market.

Regional Insights of Gene Therapy for Rare Disease Market

In terms of geography, this research report covers nearly all major regions of the world, such as North America, Europe, South America, the Middle East, and Africa and Asia Pacific. Europe and North America are expected to increase over the next few years. Gene Therapy for Rare Disease markets in the Asia Pacific region are expected to grow significantly during the forecast period. State-of-the-art technology and innovation are the most important characteristics of North America and the main reason the United States dominates the world market. The Gene Therapy for Rare Disease market in South America is also expected to grow in the near future.

The main questions answered in the report:

The Gene Therapy for Rare Disease Market Report provides future growth drivers and a competitive environment. This will help buyers of market reports clearly understand their significant growth and the resulting market strategy. The markets granular information helps monitor future profitability and make important decisions for growth.

The conclusion of this report provides an overview of the potential for new projects to succeed in the market in the near future, with a full range of global Gene Therapy for Rare Disease markets in terms of investment potential in various segments of the market.

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Gene Therapy for Rare Disease Market 2020 BY KEY BUSINESS OPPORTUNITIES, EMERGING TECHNOLOGIES, KEY COMPANIES WITH SIZE AND SHARE FORECAST TO 2027 -...

Gene Therapy Market Size By Product Analysis, Application, End-Users, Regional Outlook, Competitive Strategies And Forecast Up To 2026 – 3rd Watch…

New Jersey, United States,- Latest update on Gene Therapy Market Analysis report published with extensive market research, Gene Therapy Market growth analysis, and forecast by 2026. this report is highly predictive as it holds the overall market analysis of topmost companies into the Gene Therapy industry. With the classified Gene Therapy market research based on various growing regions, this report provides leading players portfolio along with sales, growth, market share, and so on.

The research report of the Gene Therapy market is predicted to accrue a significant remuneration portfolio by the end of the predicted time period. It includes parameters with respect to the Gene Therapy market dynamics incorporating varied driving forces affecting the commercialization graph of this business vertical and risks prevailing in the sphere. In addition, it also speaks about the Gene Therapy Market growth opportunities in the industry.

Gene Therapy Market Report covers the manufacturers data, including shipment, price, revenue, gross profit, interview record, business distribution etc., these data help the consumer know about the competitors better. This report also covers all the regions and countries of the world, which shows a regional development status, including Gene Therapy market size, volume and value, as well as price data.

Gene Therapy Market competition by top Manufacturers:

Gene Therapy Market Classification by Types:

Gene Therapy Market Size by End-user Application:

Listing a few pointers from the report:

The objective of the Gene Therapy Market Report:

Cataloging the competitive terrain of the Gene Therapy market:

Unveiling the geographical penetration of the Gene Therapy market:

The report of the Gene Therapy market is an in-depth analysis of the business vertical projected to record a commendable annual growth rate over the estimated time period. It also comprises of a precise evaluation of the dynamics related to this marketplace. The purpose of the Gene Therapy Market report is to provide important information related to the industry deliverables such as market size, valuation forecast, sales volume, etc.

Major Highlights from Table of contents are listed below for quick lookup into Gene Therapy Market report

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Gene Therapy Market Size By Product Analysis, Application, End-Users, Regional Outlook, Competitive Strategies And Forecast Up To 2026 - 3rd Watch...

Value of Cancer Gene Therapy Market Predicted to Surpass US$ by the of 2020 – Jewish Life News

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Some of the major companies operating in the global cancer gene therapy market are Cell Genesys, Advantagene, GenVec, BioCancell, Celgene and Epeius Biotechnologies. Other leading players in cancer gene therapy market include Introgen Therapeutics, ZIOPHARM Oncology, MultiVir and Shenzhen SiBiono GeneTech

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Value of Cancer Gene Therapy Market Predicted to Surpass US$ by the of 2020 - Jewish Life News

Gilead axes $445M Precision Biosciences gene therapy hep B pact – FierceBiotech

Gilead Sciences is ditching a new avenue of treating hepatitis B as it cuts ties with Precision Biosciences less than two years into their research pact.

Back in September 2018, Gilead laid up to $445 million on the table in biobucks that saw the pair collaborate on gene therapies aimed at eliminating viral infections in vivo by using Precisions genome editing platform.

Under the deal, Gilead was on tap to fully fund the effort and run clinical trials while Precision was on the hook for early development, formulation and preclinical work.

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Current HBV treatments may suppress viral replication, but they do not completely clear out the virus; the infections covalently closed circular DNA, or cccDNA, enables HBV replication to restart if treatment is stopped.

Precisions ARCUS editing platform is derived from a natural enzyme called a homing endonuclease that can target long sequences and is used to insert or delete DNA. The Durham, North Carolina-based companys fully synthetic version can be designed to locate and disrupt particular sequences and minimize off-target effects.

Gileads preliminary, in vitro studies using ARCUS nucleases had shown significant activity against cccDNA and integrated HBV DNA in human liver cells. But, two years down the line, Gilead no longer wants in.

Precision, which is predominately working on next-gen cancer therapies, will regain the license to its hep B program. Its not clear what prompted the decision, but the biotech could be on the lookout for a new partner.

This was a highly productive and well-aligned collaboration, and we deeply value the opportunity to advance our ARCUS genome editing technology and a potential cure for HBV alongside a global leader in infectious disease, said Derek Jantz, Ph.D., co-founder and chief scientific officer of Precision.

Key learnings from this program and how to develop liver-directed gene editing therapeutic candidates are directly applicable to our in vivo pipeline. While we consider new partnership opportunities for HBV, we are focused on progressing our internal lead proprietary gene correction program for primary hyperoxaluria type 1 for which we expect to nominate a clinical candidate later this year.

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Gilead axes $445M Precision Biosciences gene therapy hep B pact - FierceBiotech

Orchard Therapeutics and MolMed Announce Extension of Gene Therapy Manufacturing Collaboration – BioSpace

BOSTON and LONDON and MILAN, Italy, July 09, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and MolMed S.p.A (MLMD.MI), one of the companys principal contract development and manufacturing partners, today announced that they have extended their collaboration initiated in April 2018 for a period of five years through June 2025.

With the extension of the collaboration, MolMed will continue to support activities related to the development and manufacturing of vectors and drug products for several of Orchards investigational ex vivo hematopoietic stem cell (HSC) gene therapies in the upcoming years, including OTL-200 for metachromatic leukodystrophy (MLD) and OTL-103 for Wiskott Aldrich syndrome (WAS), as well as for additional pipeline programs including OTL-203 for mucopolysaccharidosis type I (MPS-I). MolMed is the first company to have obtained good manufacturing practice (GMP) authorization for the gene and cell therapy markets in Europe and is the manufacturer for Strimvelis, Orchards ex vivo HSC gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) and the first such treatment approved by the European Medicines Agency (EMA).

We are looking forward to continuing to build and expand upon our partnership with MolMed, who have supported the progression of many of our programs since their earliest clinical development stages, said Frank Thomas, president and chief operating officer of Orchard. Their expertise in gene therapy manufacturing, coupled with their deep knowledge of our programs, will be invaluable as our therapies for MLD and WAS approach anticipated approval and commercialization in Europe and across the globe.

Luca Alberici, MolMed's chief business officer, added, "We are pleased to have strengthened our collaboration with Orchard to support them in their mission of bringing potentially transformative therapies to those suffering from severe rare diseases. After being Orchards exclusive manufacturer for Strimvelis, we are looking forward to supporting their manufacturing needs for additional programs both in clinical trials and in potential commercial applications following the anticipated approval of OTL-200 for MLD in Europe later this year.

OTL-200 for MLD is currently under review by the EMA with a decision expected later this year.

About OrchardOrchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

About MolMedMolMed S.p.A. is a biotechnology company focused on research, development, manufacturing and clinical validation of novel cell and gene therapies. MolMed, established in 1996, has been listed since March 2008 on the Italian Stock Exchange managed by Borsa Italiana, and has its registered office in Milan, at the Biotechnology Department of Ospedale San Raffaele and an operating site at Bresso, at the OpenZone campus.

Availability of Other Information About OrchardInvestors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release, Orchards expectations regarding the timing of regulatory submissions for or marketing approval of its product candidates, and Orchards expectations concerning its partnership with MolMed. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development and commercial programs; the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the risk of delays in Orchards ability to commercialize its product candidates, if approved; and the risk that Orchard may not receive the expected benefits from its collaboration with MolMed or that Orchard or MolMed will not fully perform under the terms of their collaboration agreement. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter ended March 31, 2020, as filed with the U.S. Securities and Exchange Commission (SEC) on May 7, 2020, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Orchard Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

MolMed Contacts

Investor Relations & Communications DepartmentMolMed S.p.A.+39 02 21277.205investor.relations@molmed.com

Tommasina CazzatoPress OfficeCommunity Group+39 345 7357751tommasina.cazzato@communitygroup.it

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Orchard Therapeutics and MolMed Announce Extension of Gene Therapy Manufacturing Collaboration - BioSpace

Tissue Engineering Gene Therapy Market Consumption Status and Prospects Professional Market Research Report 2025 – CueReport

The latest report on Tissue Engineering Gene Therapy Industry market gives a broad evaluation of the global Tissue Engineering Gene Therapy Industry market by categorizing it in terms applications, types, and regions. The report gives a detailed analysis on competitive era and strategies adopted by the business to become the leader of the market in a positive way. Further, the report gives an overview of current market dynamics by studying various key segments based on the product, types, applications, end-to-end industries and market scenario. Under COVID-19 outbreak globally, this report provides 360 degrees of analysis from supply chain, import and export control to regional government policy and future influence on the industry.

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Tissue Engineering Gene Therapy Market Consumption Status and Prospects Professional Market Research Report 2025 - CueReport