StemCell Therapy

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.

The report also maps the qualitative impact of various market factors on market segments and geographies.

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

President Trump's Operation Warp Speed, with its promise of a coronavirus vaccine by the end of the year, is a lofty goal. To me, it is cruel. Only a vaccine can put an end to the virus' grip on our world. To make this promise on that timeline is obviously just a political move. For those of us who are older than 60 years, have children we haven't seen in two months and are afraid to leave our houses, this may really be the ultimate hoax.

Responding to the letter about Democrats in cities doing nothing for minorities for decades, it is by design. The Democrats do not want a vibrant, well-educated middle class. They want dependency on government that breeds a steady stream of voters. If someone becomes dependent on no one, they become mobile and can vote with their feet, and leave the Democrat control of their lives. Democrats want you dependent on them, thats the bottom line.

American democracy has been chained by the right and hijacked by the left. Middle America has been hoodwinked by upper-class elitists on both sides. Right-wing conservatives led by the top 2% super-rich have snookered the working class into believing that there is no difference between economic, religious or cultural conservative idealism. Left-wing academic, artsy and so-called progressive elitists have teamed with the media creating a massively profitable industrial complex feeding off the latest righteous crusades. The elites continue enriching themselves at the middles expense. Dont let the elites on either side crush our backbone. Stand up and fight for Middle America.

Independent Patriot

A Black national anthem and a white national anthem to be played at the start of the National Football Leagues first game? Really? This will do nothing but emphasize our differences rather than promote togetherness. I want an anthem that points out our weaknesses but yet strives for greatness. I want an anthem that explains fighting for the right to make all men equal not just in an hour of need but always. I want an anthem I can appreciate, that speaks for everyone, that all people would be proud to stand for.

After cancellation by the American Legion, it is inspiring to see the teams find a way to still play ball. They applied good old American ingenuity have a problem, find a way to fix it. They have banded together to play 33 games. There will be those who disapprove but what could be safer than healthy young men playing ball in the fresh air. Good luck to the Berks Independent Baseball League.

Proud to be an American

Seniors don't forget what you were promised from those you elected. They lied, you are still paying property taxes and you will lose your home. Make your vote count.

Night light

President Obama acknowledged on multiple occasions that he did not have the authority to unilaterally alter immigration law, yet its exactly what he did when he implemented DACA. At some point the courts will rightfully rule DACA unconstitutional as it did with DAPA. Its heartbreaking that the Democrats in Congress refused a very fair deal from President Trump in 2018 to resolve the fate of the Dreamers in exchange for increased border security and real immigration reform. President Trump has been trying to solve the problem, but the Democrats chose to leave the Dreamers in legal limbo just to have a campaign issue.

Robert Minninger

Thank you, Pottstown for having peaceful protests. Some questions for the white folks participating. Do you live in Pottstown? If not, why not? If you do live in Pottstown, what part of town do you live in and why did you choose it? Is it racially diverse? If you're protesting for equality yet going back to your all-white, no-crime neighborhood, you're a hypocrite.

Truth hurts

The inadequate response to the coronavirus in the Oval Office as well as similar actions by governors in states such as Alabama, Arizona, Arkansas and South Carolina (states with the highest rates in the nation when adjusted for population) have increased the coronavirus spread. Wear a mask. Stay safe!

M. Furlong needs to stop playing the name game in order to divert attention from the real issue of racial inequality in this country. The poor use of funds to help people of color can be blamed on both Democrats and Republicans since forever. And until he takes one small step toward impartiality, hell never understand the meaning of Black Lives Matter.

If you havent heard already, Tower Health is closing the Maternity Unit at Pottstown Hospital. The Maternity Unit has provided this community with outstanding care for mothers and their newborns and has provided a multitude of services like birthing classes, baby care classes, breastfeeding classes, car seat checks. Where will these girls go to deliver? Most of them dont have transportation to Reading or Phoenixville. This community is losing a great asset. We all need to contact the hospital, state and local reps and demand they keep this unit open. We need to keep our moms to be and babies safe!

This week's Stay-At-Home readings are: How To Avoid Traffic Tickets by Constance Lee Parks; How To Find Love by Mary Mee; Housecleaning Tips by Mary Maids; Positive Thinking by Will Power

Funnyman

The potential for "One-shot cures" of gene therapy is valid, however, such treatments do not offer recurring revenue like chronic therapies do. While gene therapy is very attractive to patients, it becomes a challenge to doctors of genome medicine looking for sustained cash flow. This should tell you what is most important and which path they'll follow since prescription drugs have made Big Pharma and docs quite wealthy.

Med Tech

Why are school police out and about in district cars when school isnt in session?

Ano Nymous

Should Rep. Houlahan be judged by the company she keeps? Disdain for the military is not a recent Democrat position. To mask this reality in historically Republican districts, Nancy Pelosi recruited former officers for 2018 congressional campaigns. One such candidate was Chrissy Houlahan who became a reliable ally of Rep. Adam Schiff (D-CA) in a seditious, fact-bereft attempt to unseat a president. Last week, Houlahan featured Rep. Karen Bass (D-CA) in a virtual town hall meeting. Who is Bass? An unapologetic radical who lamented the death of "Commandante El Jefe" Fidel Castro.

M. Furlong

Schools do not teach manners, that is the parents' job. What we see today are children with knowledge, sports achievements, different dress codes, anger over everything but no manners or common sense. You, as parents, have failed the youth of today and tomorrow. When an officer of the law says stop, why do children keep running?

Star Light

I'm so glad I got to see Mount Rushmore on television this past weekend because the way things are going, that will be the next thing the protesters want torn down.

I watched the amateur looters on the news dressed in T-shirts and shorts, but the professional looters who steal from us daily dress in suits and ties. They're known as "Congress!"

Jim Fitch

Sound Off is a forum to spur dialogue from residents of the communities The Mercury serves on topics of general interest in those communities and beyond. We will not publish comments that are potentially libelous, slanderous, mean-spirited, vulgar or inappropriate. Publication of Sound Off comments is at the sole discretion of the editor. Email your brief comment to letters@pottsmerc.com. Please use "Sound Off" in the subject line of the email.

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The Mercury's Sound Off for Sunday, July 12 | Opinion - The Mercury

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.

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

It might be the next best thing to a coronavirus vaccine.

Scientists have devised a way to use the antibody-rich blood plasma of COVID-19 survivors for an upper-arm injection that they say could inoculate people against the virus for months.

Using technology thats been proven effective in preventing other diseases such as hepatitis A, the injections would be administered to high-risk healthcare workers, nursing home patients, or even at public drive-through sites potentially protecting millions of lives, the doctors and other experts say.

The two scientists who spearheaded the proposal an 83-year-old shingles researcher and his counterpart, an HIV gene therapy expert have garnered widespread support from leading blood and immunology specialists, including those at the center of the nations COVID-19 plasma research.

But the idea exists only on paper. Federal officials have twice rejected requests to discuss the proposal, and pharmaceutical companies even acknowledging the likely efficacy of the plan have declined to design or manufacture the shots, according to a Times investigation. The lack of interest in launching development of immunity shots comes amid heightened scrutiny of the federal governments sluggish pandemic response.

There is little disagreement that the idea holds promise; the dispute is over the timing. Federal health officials and industry groups say the development of plasma-based therapies should focus on treating people who are already sick, not on preventing infections in those who are still healthy.

Dr. Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases at the National Institutes of Health, said an upper-arm injection that would function like a vaccine is a very attractive concept.

However, he said, scientists should first demonstrate that the coronavirus antibodies that are currently delivered to patients intravenously in hospital wards across the country actually work. Once you show the efficacy, then the obvious next step is to convert it into an intramuscular shot.

But scientists who question the delay argue that the immunity shots are easy to scale up and should enter clinical trials immediately. They say that until theres a vaccine, the shots offer the only plausible method for preventing potentially millions of infections at a critical moment in the pandemic.

Beyond being a lost opportunity, this is a real head-scratcher, said Dr. Michael Joyner, a Mayo Clinic researcher who leads a program sponsored by the Food and Drug Administration to capitalize on coronavirus antibodies from COVID-19 survivors. It seems obvious.

The use of so-called convalescent plasma has already become widespread. More than 28,000 patients have already received the IV treatment, and preliminary data suggest that the method is safe. Researchers are also looking at whether the IV drip products would prevent new infections from taking root.

The antibodies in plasma can be concentrated and delivered to patients through a type of drug called immune globulin, or IG, which can be given through either an IV drip or a shot. IG shots have for decades been used to prevent an array of diseases; the IG shot that prevents hepatitis A was first licensed in 1944. They are available to treat patients who have recently been exposed to hepatitis B, tetanus, varicella and rabies.

Yet for the coronavirus, manufacturers are only developing an intravenous solution of IG.

Joyner told The Times that 600 COVID-19 survivors donating their plasma each day could, depending on donation volumes and concentrations, generate up to 5,000 IG shots. With millions of probable survivors in the United States, he said, capacity isnt a problem.

Plasma companies said theyve focused their efforts on an intervention for the sickest patients. Grifols, for example, said it has not developed a shot because it is pursuing a federally supported IV formula to treat patients already infected with a serious case of COVID-19, but the company acknowledged that an antibody injection would be a good choice for prevention.

Advocates for the immunity shots say businesses are reluctant to invest in a product that could soon be replaced by a vaccine, so the government should offer financial incentives to offset that risk. Billions of federal dollars are already being spent on vaccine research through Operation Warp Speed, and funding for an IG shot that could serve as a bridge to a vaccine would come with a relatively modest price tag, they say.

Antibodies are the most precious resource on the planet right now, next to air. We have the industry, the technology, and the know-how to produce a proven product, said Patrick Schmidt, the chief executive of FFF Enterprises, a major distributor of IG products in the United States.

The amount of money and resources going into a vaccine, with no guarantee it will work this could have saved lives by now.

::

The proposal for an injection approach to coronavirus prevention came from an immunization researcher who drew his inspiration from history.

Dr. Michael Oxman knew that, even during the 1918 flu pandemic, the blood of recovered patients appeared to help treat others. Since then, convalescent plasma has been used to fight measles and severe acute respiratory syndrome, or SARS, among other diseases.

Like other doctors, Oxman surmised that, for a limited time, the blood coursing through the veins of coronavirus survivors probably contains immune-rich antibodies that could prevent or help treat an infection.

On March 27, he and Dr. John Zaia, the director of City of Hopes Center for Gene Therapy, submitted a proposal to the federal Biomedical Advanced Research and Development Authority, or BARDA, urging the rollout of IG shots for first responders and members of other high-risk groups.

The agency granted $12.5 million to Grifols and $14.5 million to Emergent BioSolutions to produce plasma-based COVID-19 medicines in IV form drips, among more than 50 different biomedical partnerships to fight the pandemic. But the immunity shot proposal was rejected.

The pair followed up with a detailed proposal to conduct a clinical trial at UC San Diego. They believed injectable 5-milliliter vials of IG could be given quickly by minimally trained healthcare workers, offering at least two months of immunity to doctors and nurses, as well as residents of nursing homes, college dormitories and military submarines.

The submission was backed by four other infectious disease researchers and statisticians, but it was also rejected, records show.

A spokeswoman for BARDA told The Times that the agency had received thousands of submissions, and that while we are interested in the potential of [IG] for treatment and prevention, we are focused intently on treatments for hospitalized patients to save lives.

The strategy baffled Oxman and Zaia, who said the IG shots are a far more efficient delivery system that can potentially reach many more people.

Whats more, prophylactic shots would probably require far fewer antibodies than IV treatments, Joyner said. With IG shots, plasma donations could possibly go twice or even five times as far, he said.

If a second wave of the virus were to arrive before an effective vaccine, that stockpile would be all the more essential.

Oxman started focusing his attention on the key players in the industry the manufacturers who dominate the development of plasma drugs. He held weekly phone calls with Schmidt, the distributor; together, the two tried to persuade seven companies to produce the shots themselves and bring them to health agencies for testing. They were unsuccessful.

Takeda and CSL Behring, two large companies who co-lead the new CoVIg-19 Plasma Alliance to develop an IG product for IV drips, said their efforts are trained on the sickest. The IV formula represents the fastest path to reach patients, assuming the trial is successful, said Julie Kim, the head of the plasma-derived therapies business unit at Takeda.

Financial calculations may be another factor for companies. Intravenous plasma products are traditionally the main economic driver for the industry, supply experts said, in part because vaccines have replaced many short-term immunity shots over the years. The money-making antibodies are also far more diluted in intravenous drugs than in injectable ones, which boosts profit margins.

They charge a fortune off of intravenous drugs in the hospital. They dont want to devote the manufacturing plant to something that wont make oodles of money, said one infectious disease expert, who has advocated for coronavirus IG shots but asked not to be publicly identified.

Researchers also said industry executives have little incentive to produce the immunity shots for the coronavirus, given the possibility that a longer-lasting vaccine could replace it within a year.

Representatives for CSL, Takeda and Grifols all challenged that assertion.

The choice of one delivery method or another has no connection with the potential financial or pricing implications, a Grifols spokesman told The Times.

Throughout May, researchers and doctors at Yale, Harvard, Johns Hopkins, Duke and four University of California schools sent a barrage of letters to dozens of lawmakers. They held virtual meetings with health policy directors on Capitol Hill, but say they have heard no follow-up to date.

Dr. Arturo Casadevall, the chair of the National COVID-19 Convalescent Plasma Project, said he spoke to FDA officials who told him they do not instruct companies on what to produce. Casadevall told The Times that the leaders of the national project were very supportive of the need to develop an IG shot rapidly and that he believed it would be very helpful in stemming the epidemic.

Joyner, of the Mayo Clinic, said there are probably 10 million to 20 million people in the U.S. carrying coronavirus antibodies and the number keeps climbing. If just 2% of them were to donate a standard 800 milliliters of plasma on three separate occasions, their plasma alone could generate millions of IG shots for high-risk Americans.

At a hot-spot meatpacking plant, or at a mobile unit in the parking lot outside a mall trust me, you can get the plasma, Joyner said. This is not a biological problem nor a technology problem. Its a back-of-the-envelope intelligence problem.

The antibody injections, for now, do not appear to be a high priority for the government or the industry.

Grifols, on April 28 the same day that the U.S. topped 1 million confirmed coronavirus cases made a major product announcement that would expand its leadership in disease treatment with immunoglobulins.

The product was a new vial for IG shots to treat rabies.

More here:
An injection may block COVID-19, but feds have failed to act - Los Angeles Times

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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 suggest 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

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

Global Cancer Gene Therapy Market: Overview

Cancer could be defined as uncontrolled cell growth in the body leading to organ malfunction. If untreated, it can lead to death. Uncontrolled growth of cell is managed by the body in several ways, one of them is by deploying white blood cells to detect and eradicate these cancerous cells. It has been discovered that the immune system could be manipulated to influence cancerous cells to destroy itself.

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Radiation and chemotherapy therapy have consistent and reliable effects to decrease cancerous cells in the body. Recently, immunotherapy for hematological cancers has experienced a recognition and is of interest for many researchers Scientists have developed methods to isolate, replicate, and develop cancer-destroying cells from the patients blood cancer and injecting those cells back for the destruction of their cancers, with durable remissions.

New options for the treatment is needed to be developed if order to achieve elimination of cancer suffering and death by 2020. According to NCI, 5-year survival rate for cancers such as lung (15%), glioblastoma (5%), pancreatic (4%), and liver (7%) remains very low. Current available treatments have several side effects, the systemic toxicity due to chemotherapy results in nausea, mild cognitive impairments, and mouth ulcerations, in addition to long-term side effects such as increasing risk of developing other types of cancers. Therefore, new and innovative treatment methods are required to reduce the suffering of cancer patients.

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GlobalCancer Gene Therapy Market: Drivers and Restraints

The emerging field of cancer Gene Therapy offers varied potential treatments. Gene therapy involves a range of treatment types, which use genetic material to alter cells (either in vivo or in vitro) to help cure the disease. Cancer Gene Therapy shown efficacy in various in vitro and preclinical testing. Preclinical testing for cancer gene therapy has been performed on glioma, pancreatic cancer, liver cancer, and many other cancers.

Increase in prevalence of cancer, rise in government funding and initiatives, growth in pipeline of cancer gene therapy products, and collaborations to develop and launch gene-therapy products are some factors driving the market. According to NCBI researchers, development of genetically-modified T-cell therapies for treatment of cancer has had maximum clinical impact among other gene therapies. However, high treatment cost is a major limitation in the cancer gene therapy market. The reason behind the huge cost for cancer gene therapy is the necessity of rigorous, exhaustive clinical trials; also treatment by cancer gene therapy differs from person to person depending upon the genetic acceptance of every patient, unlike other drugs thereby limiting the market growth.

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GlobalCancer Gene Therapy Market: Key Segments

Based on type, the cancer gene therapy market is segmented into gene transfer immunotherapy and oncolytic virotherapy. Immunotherapy uses genetically modified cells and viral particles to stimulate the immune system to destroy cancer cells. Immunotherapy include treatment with either cytokine gene delivery or tumor antigen gene delivery. Oncolytic virotherapy uses viral particles, which replicate within the cancer cell causing the death of the cell. It is an emerging treatment modality that is expected to shows great promise, particularly in metastatic cancer treatment.

It includes treatment with adenovirus, retrovirus, lentivirus, herpes simplex virus, adeno-associated virus, simian virus, alphavirus, and vaccinia virus. Gene transfer is the newest treatment modality that is expected to introduce new modified genes into cancerous cell or associated tissue for destruction of cell or to slow down cancer growth. This technique is flexible as a wide variety of vectors and genes are used for clinical trials with positive outcomes. As gene therapy advance, they could be used alone or in combination with other treatments to control the disease. Gene transfer or gene replacement is performed using naked/plasmid vectors, electroporation, sonoporation, magnetofection, and gene gun.

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Based on region, the global cancer gene therapy market is segmented into North America, Europe, Asia Pacific, Latin America and Middle East & Africa. North America is anticipated to hold the largest market share. The U.S. dominates the cancer gene therapy market owing to its increase in funding for research & development and other government initiatives. Key players in the biotech industry are engaging in research & development of gene therapy products. Moreover, rising demand for DNA vaccines and growing interest of venture capitalists to investment in commercialization of gene-based cancer therapies are likely to propel the market. The cancer gene therapy market in Asia Pacific is anticipated to expand at a rapid pace as in China cancer gene therapy is anticipated to attribute for largest revenue, due to the recent launch of Gendicine and rising healthcare expenditure with strong R&D facilities.

GlobalCancer Gene Therapy Market: Key Players

Key players operating in the global cancer gene therapy market are Adaptimmune, ZioPharm Oncology Altor Bioscience, MolMed, bluebird bio, Shanghai Sunway Biotech company limited , MultiVir, Shenzhen SiBiono GeneTech, Corporation.

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Cancer Gene Therapy Market Outlook for Major Applications/end Users, Consumption, Share and Growth Rate 2025 - Cole of Duty

A group of patient advocacy groups has sent a letter to the Centers for Medicare and Medicaid Services urging the agency to establish the new Medicare Severity-Diagnosis Related Group for Chimeric Antigen Receptor T-cell Immunotherapy.

In the letter, the stakeholders argued that CAR-T therapies offer improved outcomes for patients with relapsed/refractory diffuse large B-cell lymphoma and B-cell acute lymphoblastic leukemia and provides hope for many more with other hard-to-treat cancers

The new MS-DRG would also give hospitals and providers a new course of treatment for those cancers.

The letter backed up its praise with a study in which lymphoma patients had significant improvements in their physical, social and emotional wellbeing following CAR-T therapy.

The organizations signing the letter include the American Cancer Society Cancer Action Network, BMT Infonet, Cancer Support Community, CLL Society, CrowdCare Foundation/Myeloma Crowd, International Myeloma Foundation, Leukemia & Lymphoma Society, Lymphoma Research Foundation, Society for Immunotherapy of Cancer and The Pink Fund.

WHY THIS MATTERS

This letter is a comment to CMS' proposed inpatient prospective payment system rule issued in May.

Under the current rule, CAR-T hospital cases are paid at the same rate as bone marrow transplants and qualify for additional payments through the temporary new technology add-on payment for high-cost cases that are set to expire this year.

The rule would create a separate hospital payment category for CAR-T therapy.

The new MS-DRG for CAR-T would provide predictable payment rates for hospitals administering the therapy.

CAR-T is a gene therapy that uses a patient's own genetically modified immune cells as a treatment for certain types of cancer. This is instead of additional chemotherapy or other types of treatment paid for under the inpatient prospective payment system.

The open comment period for the proposed rule ends today, Friday, July 10.

THE LARGER TREND

For years, organizations such as the American Society for Blood and Marrow Transplantation have been asking for an MS-DRG to be created for CAR-T therapies.

ON THE RECORD

"Our organizations commend CMS for its action and appreciate its forward-leaning policy proposal that will optimize patient access for CAR-T therapy," the stakeholders wrote. "We support finalization of the proposed rule and look forward to working with CMS to further support novel treatments for unmet medical needs among immunotherapy patients."

Twitter:@HackettMalloryEmail the writer:mhackett@himss.org

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Stakeholder urge CMS to finalize new CAR-T therapy payment rule - Healthcare Finance News

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimers drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agencys decision-making time to a mere 6 months.

The news drove a 10% spike in Biogens stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Flagship execs take a lesson from nature to master 'gene writing,' launching a star-studded biotech with big ambitions to cure disease - Endpoints...

NEW YORK and CLEVELAND, July 10, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that two poster presentations related to its clinical program for recessive dystrophic epidermolysis bullosa (RDEB) were featured at the Society for Pediatric Dermatology (SPD) 45th Annual Meeting. The first poster includes a detailed analysis of patients with RDEB in the EB-101 Phase 1/2a trial showing that wound healing following EB-101 treatment was associated with improved long-term pain relief. A separate poster provides insights on the significant disease burden associated with RDEB, highlighting data from a literature review on the clinical characteristics, humanistic consequences and economic impact of living with RDEB on patients and their families.

The large wounds of RDEB cause substantial pain, and only palliative treatments are currently available, said Joo Siffert, M.D., Chief Executive Officer of Abeona. The data presented at SPD showed that EB-101 treatment of large, chronic wounds resulted in considerable and durable reduction in wound burden, which was associated with long-term pain relief for up to five years. The second poster at SPD helps to characterize the disease burden and management of RDEB, providing an important reminder of the extraordinary toll RDEB takes on quality of life, and underscores the need for therapies that reduce wound burden and the associated humanistic and economic impact.

EB-101 Treatment of Large, Chronic Wounds Is Associated with Durable Healing and Pain Reduction in Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Jean Tang, M.D., Ph.D., Professor of Dermatology, Stanford University Medical Center and Principal Investigator of the EB-101 pivotal Phase 3 VIITALTM study, presented long-term outcomes following EB-101 treatment for large, chronic wounds in patients with RDEB. EB-101 treatment resulted in considerable and durable reduction in wound burden in the range of three to five years in a Phase 1/2a study. Wound healing of 50% or greater following EB-101 treatment was associated with no pain at treated sites at three years, four years and five years post-treatment, compared with presence of pain in 53% of wound sites at baseline. The ongoing VIITALTM study will further characterize the relationship between reduction of wound burden and pain relief following EB-101 treatment.

The Full Burden of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

M. Peter Marinkovich, M.D., Bullous Disease Clinic Director, Stanford University Medical Center, and Investigator in the VIITALTM study, presented findings from a literature review of 65 studies that provide new insights on the disease burden from the perspective of patients with RDEB and their families. Key observations of the clinical, humanistic and economic burden of RDEB include:

Abeonas posters from the SPD 45th Annual Meeting are available on the News/Events page under the Investors & Media section of Abeonas website at http://www.abeonatherapeutics.com.

About Recessive Dystrophic Epidermolysis BullosaRecessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning type VII collagen, which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

About EB-101EB-101 is an autologous, gene-corrected cell therapy currently being investigated in the pivotal Phase 3 VIITALTM study for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. The EB-101 VIITALTM study is a multi-center, randomized clinical trial enrolling 10 to 15 RDEB patients with approximately 30 large, chronic wound sites treated in total. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into a patients own skin cells (keratinocytes and their progenitors) and transplanting them back to the patient to enable normal Type VII collagen expression and facilitate wound healing. Abeona produces EB-101 for the VIITALTM study at its fully-functional gene and cell therapy manufacturing facility in Cleveland, OH. In a Phase 1/2a clinical trial, EB-101 provided durable wound healing for RDEB patients lasting 2+ to 5+ years, including for the largest, most challenging wounds that affect the majority of the RDEB population. More information on the clinical trials of EB-101 can be found at https://www.abeonatherapeutics.com/clinical-trials/rdeb and ClinicalTrials.gov (Identifier: NCT04227106).

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeonas clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Companys portfolio of AAV-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeonas novel, next-generation AIM capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeonas fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITALTM study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit http://www.abeonatherapeutics.com.

Forward-Looking StatementsThis press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Companys clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Companys goals and objectives. We have attempted to identify forward looking statements by such terminology as may, will, believe, estimate, expect, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Companys Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

Investor Contact:Greg GinVP, Investor RelationsAbeona Therapeutics+1 (646) 813-4709ggin@abeonatherapeutics.com

Media Contact:Scott SantiamoDirector, Corporate CommunicationsAbeona Therapeutics+1 (718) 344-5843ssantiamo@abeonatherapeutics.com

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Abeona Therapeutics Announces Two Presentations Related to Its RDEB Clinical Program at the Society for Pediatric Dermatology 45th Annual Meeting -...

Global Cancer Gene Therapy Market Report 2019-Market Size, Share, Price, Trend and Forecast report is based on comprehensive analysis conducted by experienced and professional experts. The report mentions, factors that are influencing growth such as drivers, restrains of the market. The report offers in-depth analysis of trends and opportunities in the Cancer Gene Therapy Market. The report offers figurative estimations and predicts future for upcoming years on the basis of the recent developments and historic data. For the gathering information and estimating revenue for all segments, researchers have used top-down and bottom-up approach. On the basis of data collected from primary and secondary research and trusted data sources the report offers future predictions of revenue and market share.

The Leading Market Players Covered in this Report are : Adaptimmune,GlaxoSmithKline,Bluebird bio,Merck,Celgene,Shanghai Sunway Biotech .

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Impact of Covid-19 in Cancer Gene Therapy Market:The utility-owned segment is mainly being driven by increasing financial incentives and regulatory supports from the governments globally. The current utility-owned Cancer Gene Therapy are affected primarily by the COVID-19 pandemic. Most of the projects in China, the US, Germany, and South Korea are delayed, and the companies are facing short-term operational issues due to supply chain constraints and lack of site access due to the COVID-19 outbreak. Asia-Pacific is anticipated to get highly affected by the spread of the COVID-19 due to the effect of the pandemic in China, Japan, and India. China is the epic center of this lethal disease. China is a major country in terms of the chemical industry.

Key Businesses Segmentation of Cancer Gene Therapy MarketOn the basis on the end users/applications,this report focuses on the status and outlook for major applications/end users, sales volume, Cancer Gene Therapy market share and growth rate of Cancer Gene Therapy foreach application, including-

On the basis of product,this report displays the sales volume, revenue (Million USD), product price, Cancer Gene Therapy market share and growth rate ofeach type, primarily split into-

Cancer Gene Therapy Market Regional Analysis Includes: Asia-Pacific(Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia) Europe(Turkey, Germany, Russia UK, Italy, France, etc.) North America(the United States, Mexico, and Canada.) South America(Brazil etc.) The Middle East and Africa(GCC Countries and Egypt.)

Key Highlights of the Table of Contents:Cancer Gene Therapy Market Study Coverage:It includes key market segments, key manufacturers covered, the scope of products offered in the years considered, global Cancer Gene Therapy market and study objectives. Additionally, it touches the segmentation study provided in the report on the basis of the type of product and applications.Cancer Gene Therapy Market Executive summary:This section emphasizes the key studies, market growth rate, competitive landscape, market drivers, trends, and issues in addition to the macroscopic indicators.Cancer Gene Therapy Market Production by Region:The report delivers data related to import and export, revenue, production, and key players of all regional markets studied are covered in this section.Cancer Gene Therapy Market Profile of Manufacturers:Analysis of each market player profiled is detailed in this section. This segment also provides SWOT analysis, products, production, value, capacity, and other vital factors of the individual player.

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Key Questions Answered: To gain insightful analyses of the Cancer Gene Therapy market and have a comprehensive understanding of the global market and its commercial landscape. Assess the production processes, major issues, and solutions to mitigate the development risk. To understand the most affecting driving and restraining forces in the market and its impact on the global Cancer Gene Therapy market. Learn about the Cancer Gene Therapy market strategies that are being adopted by leading respective organizations. To understand the future outlook and prospects for the market. Besides the standard structure reports, we also provide custom research according to specific requirements.

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Cancer Gene Therapy Market Present Scenario, the Growth Prospects with Forecast 2024 - 3rd Watch News

The global Hemophilia Gene Therapy Market analysis provides pioneering landscape of market along with market augmentation history and key development involved in the industry. The report also features comprehensive research study for high growth potential industries professional survey with market analysis. Hemophilia Gene Therapy Market report helps the companies to understand the market trends and future market prospective,opportunities and articulate the critical business strategies.

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The report incorporates an estimated impact of strict standards and regulations set by the government over the market in the upcoming years. The market report also comprises exhaustive research done using several analytical tools such as SWOT analysis to identify the market growth pattern.

Major Players Are:BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC, Sangamo Therapeutics, Inc., and Freeline Therapeutics

Regions & Countries Mentioned In The Hemophilia Gene Therapy Market Report:

Key Highlights of the Table of Contents:

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Role of Hemophilia Gene Therapy Market Report:

The global Hemophilia Gene Therapy Market has been appearing as one of the most profit-making businesses in the globe. The market has been exhibiting considerable growth figures led by raw material affluence, increasing population, expanding regions, rapid elevating demand, and advanced technologies. The report is likely to be performed vigorously in the upcoming phase, analysts predicted after studying the market at a minute level.

Various analytical tools such as SWOT, Feasibility analysis, Porters Five Forces analysis, Value Chain analysis, and Capacity utilization analysis are implemented while evaluating the Hemophilia Gene Therapy Market which certainly helps a reader to get a deeper perception of the market and its participants. Additionally, it covers a cardinal evaluation of market history, patterns, changing dynamics, market and manufacturing trends, demand and supply activities, and technological development.

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Hemophilia Gene Therapy Market to Etch New Growth Ratios with Adoption in Developing Economies - 3rd Watch News

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

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

NEW YORK, July 9, 2020 /PRNewswire/ --

Global Gene Therapy Market to Reach US$4.2 Billion by the Year 2027 Amid the COVID-19 crisis, the global market for Gene Therapy estimated at US$701.2 Million in the year 2020, is projected to reach a revised size of US$4.2 Billion by 2027, growing at a CAGR of 29.3% over the analysis period 2020-2027.Lentivirus, one of the segments analyzed in the report, is projected to grow at a 21.7% CAGR to reach US$130.1 Million by the end of the analysis period.After an early analysis of the business implications of the pandemic and its induced economic crisis, growth in the AAV segment is readjusted to a revised 24.9% CAGR for the next 7-year period. This segment currently accounts for a 13.5% share of the global Gene Therapy market.

Read the full report: https://www.reportlinker.com/p05817594/?utm_source=PRN

The U.S. Accounts for Over 26.8% of Global Market Size in 2020, While China is Forecast to Grow at a 36.3% CAGR for the Period of 2020-2027 The Gene Therapy market in the U.S. is estimated at US$188.2 Million in the year 2020. The country currently accounts for a 26.84% share in the global market. China, the world second largest economy, is forecast to reach an estimated market size of US$1.1 Billion in the year 2027 trailing a CAGR of 36.3% through 2027. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at 23.2% and 26.7% respectively over the 2020-2027 period. Within Europe, Germany is forecast to grow at approximately 25.1% CAGR while Rest of European market (as defined in the study) will reach US$1.1 Billion by the year 2027.

RetroVirus & Gamma RetroVirus Segment Corners a 55.5% Share in 2020 In the global RetroVirus & Gamma RetroVirus segment, USA, Canada, Japan, China and Europe will drive the 28.6% CAGR estimated for this segment. These regional markets accounting for a combined market size of US$293.4 Million in the year 2020 will reach a projected size of US$1.7 Billion by the close of the analysis period. China will remain among the fastest growing in this cluster of regional markets. Led by countries such as Australia, India, and South Korea, the market in Asia-Pacific is forecast to reach US$662.2 Million by the year 2027, while Latin America will expand at a 30.1% CAGR through the analysis period. We bring years of research experience to this 16th edition of our report. The 248-page report presents concise insights into how the pandemic has impacted production and the buy side for 2020 and 2021. A short-term phased recovery by key geography is also addressed.

- Competitors identified in this market include, among others,

Read the full report: https://www.reportlinker.com/p05817594/?utm_source=PRN

I. INTRODUCTION, METHODOLOGY & REPORT SCOPE

II. EXECUTIVE SUMMARY

1. MARKET OVERVIEW Global Competitor Market Shares Gene Therapy Competitor Market Share Scenario Worldwide (in %): 2019 & 2028 Impact of Covid-19 and a Looming Global Recession

2. FOCUS ON SELECT PLAYERS

3. MARKET TRENDS & DRIVERS

4. GLOBAL MARKET PERSPECTIVE Table 1: Gene Therapy Global Market Estimates and Forecasts in US$ Thousand by Region/Country: 2020-2027

Table 2: Gene Therapy Global Retrospective Market Scenario in US$ Thousand by Region/Country: 2012-2019

Table 3: Gene Therapy Market Share Shift across Key Geographies Worldwide: 2012 VS 2020 VS 2027

Table 4: Lentivirus (Vector) World Market by Region/Country in US$ Thousand: 2020 to 2027

Table 5: Lentivirus (Vector) Historic Market Analysis by Region/Country in US$ Thousand: 2012 to 2019

Table 6: Lentivirus (Vector) Market Share Breakdown of Worldwide Sales by Region/Country: 2012 VS 2020 VS 2027

Table 7: AAV (Vector) Potential Growth Markets Worldwide in US$ Thousand: 2020 to 2027

Table 8: AAV (Vector) Historic Market Perspective by Region/Country in US$ Thousand: 2012 to 2019

Table 9: AAV (Vector) Market Sales Breakdown by Region/Countryin Percentage: 2012 VS 2020 VS 2027

Table 10: RetroVirus & Gamma RetroVirus (Vector) Geographic Market Spread Worldwide in US$ Thousand: 2020 to 2027

Table 11: RetroVirus & Gamma RetroVirus (Vector) Region Wise Breakdown of Global Historic Demand in US$ Thousand: 2012 to 2019

Table 12: RetroVirus & Gamma RetroVirus (Vector) Market Share Distribution in Percentage by Region/Country: 2012 VS 2020 VS 2027

Table 13: Modified Herpes Simplex Virus (Vector) World Market Estimates and Forecasts by Region/Country in US$ Thousand: 2020to 2027

Table 14: Modified Herpes Simplex Virus (Vector) Market Historic Review by Region/Country in US$ Thousand: 2012 to 2019

Table 15: Modified Herpes Simplex Virus (Vector) Market Share Breakdown by Region/Country: 2012 VS 2020 VS 2027

Table 16: Adenovirus (Vector) World Market by Region/Country in US$ Thousand: 2020 to 2027

Table 17: Adenovirus (Vector) Historic Market Analysis byRegion/Country in US$ Thousand: 2012 to 2019

Table 18: Adenovirus (Vector) Market Share Distribution in Percentage by Region/Country: 2012 VS 2020 VS 2027

Table 19: Other Applications (Vector) World Market Estimates and Forecasts in US$ Thousand by Region/Country: 2020 to 2027

Table 20: Other Applications (Vector) Market Worldwide Historic Review by Region/Country in US$ Thousand: 2012 to 2019

Table 21: Other Applications (Vector) Market Percentage Share Distribution by Region/Country: 2012 VS 2020 VS 2027

III. MARKET ANALYSIS

GEOGRAPHIC MARKET ANALYSIS

UNITED STATES Market Facts & Figures US Gene Therapy Market Share (in %) by Company: 2019 & 2025 Market Analytics Table 22: United States Gene Therapy Market Estimates and Projections in US$ Thousand by Vector: 2020 to 2027

Table 23: Gene Therapy Market in the United States by Vector: A Historic Review in US$ Thousand for 2012-2019

Table 24: United States Gene Therapy Market Share Breakdown by Vector: 2012 VS 2020 VS 2027

CANADA Table 25: Canadian Gene Therapy Market Estimates and Forecasts in US$ Thousand by Vector: 2020 to 2027

Table 26: Canadian Gene Therapy Historic Market Review by Vector in US$ Thousand: 2012-2019

Table 27: Gene Therapy Market in Canada: Percentage Share Breakdown of Sales by Vector for 2012, 2020, and 2027

JAPAN Table 28: Japanese Market for Gene Therapy: Annual Sales Estimates and Projections in US$ Thousand by Vector for the Period 2020-2027

Table 29: Gene Therapy Market in Japan: Historic Sales Analysisin US$ Thousand by Vector for the Period 2012-2019

Table 30: Japanese Gene Therapy Market Share Analysis by Vector: 2012 VS 2020 VS 2027

CHINA Table 31: Chinese Gene Therapy Market Growth Prospects in US$Thousand by Vector for the Period 2020-2027

Table 32: Gene Therapy Historic Market Analysis in China in US$ Thousand by Vector: 2012-2019

Table 33: Chinese Gene Therapy Market by Vector: Percentage Breakdown of Sales for 2012, 2020, and 2027

EUROPE Market Facts & Figures European Gene Therapy Market: Competitor Market Share Scenario (in %) for 2019 & 2025 Market Analytics Table 34: European Gene Therapy Market Demand Scenario in US$ Thousand by Region/Country: 2020-2027

Table 35: Gene Therapy Market in Europe: A Historic Market Perspective in US$ Thousand by Region/Country for the Period2012-2019

Table 36: European Gene Therapy Market Share Shift by Region/Country: 2012 VS 2020 VS 2027

Table 37: European Gene Therapy Market Estimates and Forecasts in US$ Thousand by Vector: 2020-2027

Table 38: Gene Therapy Market in Europe in US$ Thousand by Vector: A Historic Review for the Period 2012-2019

Table 39: European Gene Therapy Market Share Breakdown byVector: 2012 VS 2020 VS 2027

FRANCE Table 40: Gene Therapy Market in France by Vector: Estimates and Projections in US$ Thousand for the Period 2020-2027

Table 41: French Gene Therapy Historic Market Scenario in US$ Thousand by Vector: 2012-2019

Table 42: French Gene Therapy Market Share Analysis by Vector: 2012 VS 2020 VS 2027

GERMANYTable 43: Gene Therapy Market in Germany: Recent Past, Current and Future Analysis in US$ Thousand by Vector for the Period2020-2027

Table 44: German Gene Therapy Historic Market Analysis in US$ Thousand by Vector: 2012-2019

Table 45: German Gene Therapy Market Share Breakdown by Vector: 2012 VS 2020 VS 2027

ITALY Table 46: Italian Gene Therapy Market Growth Prospects in US$ Thousand by Vector for the Period 2020-2027

Table 47: Gene Therapy Historic Market Analysis in Italy in US$ Thousand by Vector: 2012-2019

Table 48: Italian Gene Therapy Market by Vector: Percentage Breakdown of Sales for 2012, 2020, and 2027

UNITED KINGDOM Table 49: United Kingdom Market for Gene Therapy: Annual Sales Estimates and Projections in US$ Thousand by Vector for thePeriod 2020-2027

Table 50: Gene Therapy Market in the United Kingdom: Historic Sales Analysis in US$ Thousand by Vector for the Period 2012-2019

Table 51: United Kingdom Gene Therapy Market Share Analysis byVector: 2012 VS 2020 VS 2027

SPAIN Table 52: Spanish Gene Therapy Market Estimates and Forecasts in US$ Thousand by Vector: 2020 to 2027

Table 53: Spanish Gene Therapy Historic Market Review by Vector in US$ Thousand: 2012-2019

Table 54: Gene Therapy Market in Spain: Percentage Share Breakdown of Sales by Vector for 2012, 2020, and 2027

RUSSIATable 55: Russian Gene Therapy Market Estimates and Projections in US$ Thousand by Vector: 2020 to 2027

Table 56: Gene Therapy Market in Russia by Vector: A Historic Review in US$ Thousand for 2012-2019

Table 57: Russian Gene Therapy Market Share Breakdown byVector: 2012 VS 2020 VS 2027

REST OF EUROPE Table 58: Rest of Europe Gene Therapy Market Estimates and Forecasts in US$ Thousand by Vector: 2020-2027

Table 59: Gene Therapy Market in Rest of Europe in US$ Thousand by Vector: A Historic Review for the Period 2012-2019

Table 60: Rest of Europe Gene Therapy Market Share Breakdown by Vector: 2012 VS 2020 VS 2027

ASIA-PACIFIC Table 61: Asia-Pacific Gene Therapy Market Estimates and Forecasts in US$ Thousand by Region/Country: 2020-2027

Table 62: Gene Therapy Market in Asia-Pacific: Historic Market Analysis in US$ Thousand by Region/Country for the Period 2012-2019

Table 63: Asia-Pacific Gene Therapy Market Share Analysis by Region/Country: 2012 VS 2020 VS 2027

Table 64: Gene Therapy Market in Asia-Pacific by Vector: Estimates and Projections in US$ Thousand for the Period 2020-2027

Table 65: Asia-Pacific Gene Therapy Historic Market Scenario in US$ Thousand by Vector: 2012-2019

Table 66: Asia-Pacific Gene Therapy Market Share Analysis by Vector: 2012 VS 2020 VS 2027

AUSTRALIA Table 67: Gene Therapy Market in Australia: Recent Past, Current and Future Analysis in US$ Thousand by Vector for the Period 2020-2027

Table 68: Australian Gene Therapy Historic Market Analysis in US$ Thousand by Vector: 2012-2019

Table 69: Australian Gene Therapy Market Share Breakdown byVector: 2012 VS 2020 VS 2027

INDIA Table 70: Indian Gene Therapy Market Estimates and Forecasts in US$ Thousand by Vector: 2020 to 2027

Table 71: Indian Gene Therapy Historic Market Review by Vectorin US$ Thousand: 2012-2019

Table 72: Gene Therapy Market in India: Percentage Share Breakdown of Sales by Vector for 2012, 2020, and 2027

SOUTH KOREA Table 73: Gene Therapy Market in South Korea: Recent Past, Current and Future Analysis in US$ Thousand by Vector for thePeriod 2020-2027

Table 74: South Korean Gene Therapy Historic Market Analysis in US$ Thousand by Vector: 2012-2019

Table 75: Gene Therapy Market Share Distribution in South Korea by Vector: 2012 VS 2020 VS 2027

REST OF ASIA-PACIFIC Table 76: Rest of Asia-Pacific Market for Gene Therapy: Annual Sales Estimates and Projections in US$ Thousand by Vector for the Period 2020-2027

Table 77: Gene Therapy Market in Rest of Asia-Pacific: Historic Sales Analysis in US$ Thousand by Vector for the Period2012-2019

Table 78: Rest of Asia-Pacific Gene Therapy Market Share Analysis by Vector: 2012 VS 2020 VS 2027

LATIN AMERICA Table 79: Latin American Gene Therapy Market Trends by Region/Country in US$ Thousand: 2020-2027

Table 80: Gene Therapy Market in Latin America in US$ Thousand by Region/Country: A Historic Perspective for the Period 2012-2019

Table 81: Latin American Gene Therapy Market PercentageBreakdown of Sales by Region/Country: 2012, 2020, and 2027

Table 82: Latin American Gene Therapy Market Growth Prospects in US$ Thousand by Vector for the Period 2020-2027

Table 83: Gene Therapy Historic Market Analysis in Latin America in US$ Thousand by Vector: 2012-2019

Table 84: Latin American Gene Therapy Market by Vector: Percentage Breakdown of Sales for 2012, 2020, and 2027

ARGENTINA Table 85: Argentinean Gene Therapy Market Estimates andForecasts in US$ Thousand by Vector: 2020-2027

Table 86: Gene Therapy Market in Argentina in US$ Thousand by Vector: A Historic Review for the Period 2012-2019

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Global Gene Therapy Industry - PRNewswire

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

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

Madison researchers have developed a safer and more efficient way to deliver a promising new method for treating cancer and liver disorders and for vaccination including a COVID-19 vaccine from Moderna Therapeutics that has advanced to clinical trials with humans.

The technology relies on inserting into cells pieces of carefully designed messenger RNA (mRNA), a strip of genetic material that human cells typically transcribe from a persons DNA in order to make useful proteins and go about their business. Problems delivering mRNA safely and intact without running afoul of the immune system have held back mRNA-based therapy, but UWMadison researchers are making tiny balls of minerals that appear to do the trick in mice.

These microparticles have pores on their surface that are on the nanometer scale that allow them to pick up and carry molecules like proteins or messenger RNA, saysWilliam Murphy, a UWMadison professor of biomedical engineering and orthopedics. They mimic something commonly seen in archaeology, when we find intact protein or DNA on a bone sample or an eggshell from thousands of years ago. The mineral components helped to stabilize those molecules for all that time.

Murphy and UWMadison collaborators used the mineral-coated microparticles (MCMs) which are 5 to 10 micrometers in diameter, about the size of a human cell in a series of experiments to deliver mRNA to cells surrounding wounds in diabetic mice. Wounds healed faster in MCM-treated mice, and cells in related experiments showed much more efficient pickup of the mRNA molecules than other delivery methods.

The researchers described their findings today in the journal Science Advances.In a healthy cell, DNA is transcribed into mRNA, and mRNA serves as the instructions the cells machinery uses to make proteins. A strip of mRNA created in a lab can be substituted into the process to tell a cell to make something new. If that something is a certain kind of antigen, a molecule that alerts the immune system to the presence of a potentially harmful virus, the mRNA has done the job of a vaccine.

The UWMadison researchers coded mRNA with instructions directing cell ribosomes to pump out a growth factor, a protein that prompts healing processes that are otherwise slow to unfold or nonexistent in the diabetic mice (and many severely diabetic people).

mRNA is short-lived in the body, though, so to deliver enough to cells typically means administering large and frequent doses in which the mRNA strands are carried by containers made of molecules called cationic polymers.

Oftentimes the cationic component is toxic. The more mRNA you deliver, the more therapeutic effect you get, but the more likely it is that youre going to see toxic effect, too. So, its a trade-off, Murphy says. What we found is when we deliver from the MCMs, we dont see that toxicity. And because MCM delivery protects the mRNA from degrading, you can get more mRNA where you want it while mitigating the toxic effects.

The new study also paired mRNA with an immune-system-inhibiting protein, to make sure the target cells didnt pick the mRNA out as a foreign object and destroy or eject it.

Successful mRNA delivery usually keeps a cell working on new instructions for about 24 hours, and the molecules they produce disperse throughout the body. Thats enough for vaccines and the antigens they produce. To keep lengthy processes like growing replacement tissue to heal skin or organs, the proteins or growth factors produced by the cells need to hang around for much longer.

What weve seen with the MCMs is, once the cells take up the mRNA and start making protein, that protein will bind right back within the MCM particle, Murphy says. Then it gets released over the course of weeks. Were basically taking something that would normally last maybe hours or even a day, and were making it last for a long time.

And because the MCMs are large enough that they dont enter the bloodstream and float away, they stay right where they are needed to keep releasing helpful therapy. In the mice, that therapeutic activity kept going for more than 20 days.

They are made of minerals similar to tooth enamel and bone, but designed to be reabsorbed by the body when theyre not useful anymore, says Murphy, whose work is supported by the Environmental Protection Agency, the National Institutes of Health and the National Science Foundation and a donation from UWMadison alums Michael and Mary Sue Shannon.

We can control their lifespan by adjusting the way theyre made, so they dissolve harmlessly when we want.

The technology behind the microparticles was patented with the help of the Wisconsin Alumni Research Foundation and is licensed to Dianomi Therapeutics, a company Murphy co-founded.

The researchers are now working on growing bone and cartilage and repairing spinal cord injuries with mRNA delivered by MCMs.

Reference: Khalil et al. (2020).Single-dose mRNA therapy via biomaterial-mediated sequestration of overexpressed proteins. Science Advances.DOI: 10.1126/sciadv.aba2422.

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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Safer and More Efficient Method To Deliver Gene Therapy - Technology Networks

Scientists report promising results from a study that aims to develop a new approach to treating the most common inherited eye condition

Pixabay/Arek Socha

The study, which was published in Stem Cell Reports, involved using a viral vector to carry a replacement gene to malfunctioning cells in retinal organoids.

Following treatment, analysis revealed that the mini retinas had begun to produce a protein that is essential for vision.

Professor Mike Cheetham, from UCL, helped to develop the mini retinas that were used to test the gene therapy.

He highlighted that the mini retinas allow researchers to reproduce many different elements of inherited disease.

It makes it possible for us to study in detail why people go blind and try to find ways to prevent blindness. Its exciting that the gene therapy seems to be so effective for this form of retinitis pigmentosa, Professor Cheetham said.

The research was part-funded by Fight for Sight. The charitys chief executive, Sherine Krause, described the findings as incredibly promising.

Professor Cheetham and his teams work in collaboration with Trinity College Dublin represents a significant breakthrough for eye research and shows the importance of science to find new treatments for the prevailing causes of sight loss, she said.

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UK researchers use mini retinas to test gene therapy for RP - AOP