StemCell Therapy

Pfizer has tipped its pipeline to deliver new products that add more than $15 billion to revenues by 2025. The anticipated pipeline contributions led Pfizer to predict it will weather the next wave of patent expirations that is set to start in 2026.

At its virtual investor day Tuesday, Pfizer predicted (PDF) that JAK1 inhibitor abrocitinib, antisense therapy vupanorsen and a 20-valent pneumococcal conjugate vaccine can generate peak annual revenues of more than $3 billion each. Pfizer also tipped its Duchenne muscular dystrophy gene therapy to rake in an excess of $2 billion and identified six other potential blockbusters.

With Pfizer tipping another five pipeline prospects to generate revenues of $500 million to $1 billion, the Big Pharma said sales of as-yet-unapproved experimental products could come to $15 billion by 2025.

ESMO Post Show: Highlights From the Virtual Conference

Cancer experts and pharma execs will break down the headline-making data from ESMO, sharing their insights and analysis around the conferences most closely watched studies. This discussion will examine how groundbreaking research unveiled over the weekend will change clinical practice and prime drugs for key new indications, and panelists will fill you in on the need-to-know takeaways from oncologys hottest fields.

The peak years of the experimental medicines likely lie beyond 2025. The timing of the anticipated peaks of those products coincides with the years in which Pfizer expects to face another patent cliff.

In 2026 and 2027, the U.S. basic product patents on Prevnar 13, Eliquis and Xtandi are set to expire, potentially exposing products that generated sales of $6.3 billion in Pfizers home market last year to off-patent competition. All told, analysts expect Pfizer to lose up to $20 billion in sales due to patent expirations starting in 2026. Pfizers internal forecast is roughly in line with analyst estimates.

After Lyrica went off patent last year, Pfizer entered a window in which its portfolio is virtually free from the loss of exclusivity through to 2026. The lull gives Pfizer a period to get some of its pipeline prospects to market and start growing their sales, leading management to predict the current slate of experimental assets will at least replace the revenues lost to off-patent rivals starting in 2026.

That prediction rests on Pfizers ability to get key pipeline products to market. Pfizer used the virtual event to claim a growing effectiveness in that regard. As of 2015, 5% of the drugs Pfizer took into the clinic went on to win approval. By 2019, that figure had increased to 9%, causing Pfizers success rate to go from well below the industry average to slightly above the performance of its peers.

Pfizer also noted an improved phase 2 success rate. In 2017, 17% of Pfizers phase 2 trials succeeded. Since then, Pfizer has averaged 46%. The success rate so far this year stands at 53%. Pfizer claims to be moving faster, too, stating it is on course to reduce development timelines by 2.5 years between 2017 and 2021. Automation of processes accounts for the biggest anticipated time saving.

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Pfizer tips pipeline to add $15B to sales in coming years - FierceBiotech

TEL AVIV, Israel, Sept. 15, 2020 (GLOBE NEWSWIRE) -- VBL Therapeutics (Nasdaq: VBLT) will present today a corporate overview, including an update on the OVAL pivotal study, at the H. C. Wainwright 22nd Annual Global Investment Conference being held virtually on September 14-16, 2020.

"Our OVAL trial of VB-111 in ovarian cancer continues to progress well, with over a third of the study participants already enrolled," said Dror Harats, MD, Chief Executive Officer of VBL Therapeutics. "We are pleased that the high response rate seen in our interim analysis in March, continues to be high in the total patient population to date. With blinded data becoming more mature, we currently see a good correlation between the CA-125 and RECIST responses, as well as with preliminary PFS and OS data. So far, OVAL blinded data recapitulate what we have seen in our positive Phase 2 study, which is very encouraging."

Presentation Details:

About VBLVascular Biogenics Ltd., operating asVBL Therapeutics, is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class treatments for areas of unmet need in cancer and immune/inflammatory indications. VBL has developed three platform technologies: a gene-therapy based technology for targeting newly formed blood vessels with focus on cancer, an antibody-based technology targeting MOSPD2 for anti-inflammatory and immuno-oncology applications, and the Lecinoxoids, a family of small-molecules for immune-related indications. VBLs lead oncology product candidate, ofranergene obadenovec (VB-111), is a first-in-class, targeted anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. It is conveniently administered as an IV infusion once every 6-8 weeks. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in a VBL-sponsored all comers Phase 1 trial as well as in three VBL-sponsored tumor-specific Phase 2 studies. Ofranergene obadenovec is currently being studied in a VBL-sponsored Phase 3 potential registration trial for platinum-resistant ovarian cancer.

Forward Looking StatementsThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, look forward to, may, plan, potential, predict, project, should, will, would and similar expressions. These forward-looking statements may include, but are not limited to, statements regarding our programs, including VB-111, including their clinical development, therapeutic potential and clinical results. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. Among the factors that could cause actual results to differ materially from those described or projected herein include uncertainties associated generally with research and development, clinical trials and related regulatory reviews and approvals, the risk that historical clinical trial results may not be predictive of future trial results, the impact of the COVID-19 pandemic on our business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines and clinical results, that our financial resources do not last for as long as anticipated, and that we may not realize the expected benefits of our intellectual property protection. A further list and description of these risks, uncertainties and other risks can be found in our regulatory filings with theU.S. Securities and Exchange Commission, including in our annual report on Form 20-F for the year endedDecember 31, 2019, and subsequent filings with theSEC. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.VBL Therapeuticsundertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

INVESTOR CONTACT:Michael RiceLifeSci Advisors, LLC(646) 597-6979

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VBL Therapeutics to Provide an Update on the OVAL Study Today at the H. C. Wainwright 22nd Annual Global Investment Conference - GlobeNewswire

As the global COVID-19 situation is rapidly changing, staying abreast with the latest news can be challenging. In this article, Sheraz Gul provides an overview of the broad range of potential treatments in development and discusses how the regulatory landscape can shift at any time.

IN EARLY AUGUST 2020, the World Health Organization (WHO) reported the number of confirmed cases of COVID-19 has exceeded 20 million including over 700,000 deaths. These staggering figures have come about despite global lockdown measures being in place for the past few months. The resulting flattening of confirmed cases and deaths is now being jeopardised because, as the lockdown measures are being relaxed, we are seeing a resurgence of cases in some locations. In order to prevent a possible second wave of COVID-19 cases and deaths, the wearing of masks in public places is now mandatory in many countries. At the start of August 2020, New Zealand, which had gone 102 days without recording a locally transmitted case of COVID-19, saw new cases emerging. This highlights that we are still vulnerable to the spread of the virus.

Thus far, no US Food and Drug Administration (FDA)-approved therapy for use to treat COVID-19 is available. In order to provide some respite, on 1 May 2020 the FDA, as part of its Coronavirus Treatment Acceleration Program (CTAP), issued an Emergency Use Authorization (EUA) for the investigational antiviral drug remdesivir to treat suspected or laboratory-confirmed COVID-19 in adults and children hospitalised with severe disease. Although there is limited information known about the safety and effectiveness of using remdesivir to treat people in the hospital with COVID-19, this investigational drug has been shown in a clinical trial to shorten the time to recovery in some patients. It is noteworthy that prior to this, on 28 March 2020, the FDA also issued an EUA for chloroquine and hydroxychloroquine. This was subsequently revoked on 15 June 2020, as the FDA considered that chloroquine and hydroxychloroquine no longer met the statutory criteria for the EUA. In conclusion, the use and revocation of COVID-19 drugs can change at any time, so referring to official guidance is essential.

A recent update of the US ClinicalTrials. gov website listed around 2,000 COVID-19 interventional studies that are underway, around 90 of which are now completed. These cover a broad range of potential treatments including: 1) antiviral drugs to prevent viruses from multiplying; 2) immunomodulators aimed at reducing the bodys own immune reaction to the virus; 3) neutralising antibody therapies to fight the virus (manufactured, animalsourced and blood-derived from people who have previously had COVID-19); 4) cell therapy products (cellular immunotherapies and other types of both autologous and allogeneic cells, such as stem cells, and related products); and 5) gene therapy products that modify or manipulate the expression of a gene or alter the biological properties of living cells for therapeutic use. This diversity of therapeutic approaches is important as it allows opportunities for all types of treatments to be discovered, whilst expanding our understanding of the disease. Bearing in mind the timescale to discover a therapy for any given disease, the scientific community has made major advances towards developing a COVID-19 therapy. We eagerly await the output of various clinical trials over the next few months with the expectation that some of these yield sufficiently efficacious and safe therapies that gain approval by the regulators.

As the global COVID-19 situation is rapidly changing, it is advisable to stay abreast with the latest news only from reputable sources (see suggested reading below).

Sheraz Gul is an expert in drug discovery with experience gained in academia (University of London), industry (GlaxoSmithKline Pharmaceuticals) and the largest applied research organisation in Europe (Fraunhofer Institute). He is also an adjunct lecturer at NUI-Galway, Ireland and scientific co-founder of Transcriptogen Ltd. He has co-ordinated work packages in drug discovery projects, which have attracted more than 7 million funding and has organised 42 drug discovery workshops since 2011 across the globe and trained 780 scientists

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COVID-19 therapies in the pipeline - Drug Target Review

-Parkinsons Disease R&D Day to be hosted by Gavin Corcoran, MD, Chief R&D Officer, on October 30th, 2020, with leading movement disorder specialists

NEW YORK and BASEL, Switzerland, Sept. 08, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that the company will provide a corporate overview and participate in 1:1 meetings at upcoming virtual investor and patient conferences in September. Additionally, the company announced plans for their 1st Parkinsons Disease R&D Day with key opinion leaders.

Investor and Patient Conference Presentation details:

A live webcast of the presentations will be available in the Events section of Axovant's website at http://www.axovant.com. A replay will be available for approximately 30 days following the conference.

About Axovant Gene Therapies

Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit http://www.axovant.com.

Contacts:

Media & Investors

Parag MeswaniAxovant Gene Therapies Ltd.(212) 547-2523media@axovant.cominvestors@axovant.com

Source:Axovant Gene Therapies

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Axovant Announces First Parkinson's Disease Gene Therapy R&D Day and Participation in Upcoming September Conferences" - GlobeNewswire

One of the major challenges facing gene therapy a way to treat disease by replacing a patients defective genes with healthy ones is that it is difficult to safely deliver therapeutic genes to patients without the immune system destroying the gene, and the vehicle carrying it, which can trigger life-threatening widespread inflammation.

Three decades ago researchers thought that gene therapy would be the ultimate treatment for genetically inherited diseases like hemophilia, sickle cell anemia and genetic diseases of metabolism. But the technology couldnt dodge the immune response.

Since then, researchers have been looking for ways to perfect the technology and control immune responses to the gene or the vehicle. However, many of the strategies tested so far have not been completely successful in overcoming this hurdle.

Drugs that suppress the whole immune system, such as steroids, have been used to dampen the immune response when administering gene therapy. But its difficult to control when and where steroids work in the body, and they create unwanted side effects. My colleague Mo Ebrahimkhani and I wanted to tackle gene therapy with immune-suppressing tools that were easier to control.

I am a medical doctor and synthetic biologist interested in gene therapy because six years ago my father was diagnosed with pancreatic cancer. Pancreatic cancer is one of the deadliest forms of cancer, and the current available therapeutics usually fail to save patients. As a result, novel treatments such as gene therapy might be the only hope.

Yet, many gene therapies fail because patients either already have pre-existing immunity to the vehicle used to introduce the gene or develop one in the course of therapy. This problem has plagued the field for decades, preventing the widespread application of the technology.

Traditionally scientists use viruses from which dangerous disease-causing genes have been removed as vehicles to transport new genes to specific organs. These genes then produce a product that can compensate for the faulty genes that are inherited genetically. This is how gene therapy works.

Though there have been examples showing that gene therapy was helpful in some genetic diseases, they are still not perfect. Sometimes, a faulty gene is so big that you cant simply fit the healthy replacement in the viruses commonly used in gene therapy.

Another problem is that when the immune system sees a virus, it assumes that it is a disease-causing pathogen and launches an attack to fight it off by producing antibodies and immune response just as happens when people catch any other infectious viruses, like SARS-CoV-2 or the common cold.

Recently, though, with the rise of a gene editing technology called CRISPR, scientists can do gene therapy differently.

CRISPR can be used in many ways. In its primary role, it acts like a genetic surgeon with a sharp scalpel, enabling scientists to find a genetic defect and correct it within the native genome in desired cells of the organism. It can also repair more than one gene at a time.

Scientists can also use CRISPR to turn off a gene for a short period of time and then turn it back on, or vice versa, without permanently changing the letters of DNA that makes up or genome. This means that researchers like me can leverage CRISPR technology to revolutionise gene therapies in the coming decades.

But to use CRISPR for either of these functions, it still needs to be packaged into a virus to get it into the body. So some challenges, such as preventing the immune response to the gene therapy viruses, still need to be solved for CRISPR-based gene therapies.

Being trained as a synthetic biologist, I teamed up with Ebrahimkhani to use CRISPR to test whether we could shut down a gene that is responsible for immune response that destroys the gene therapy viruses. Then we investigated whether lowering the activity of the gene, and dulling the immune response, would allow the gene therapy viruses to be more effective.

CRISPR can precisely remove even single units of DNA. KEITH CHAMBERS/SCIENCE PHOTO LIBRARY/Getty Images

A gene called Myd88 is a key gene in the immune system and controls the response to bacteria and viruses, including the common gene therapy viruses. We decided to temporarily turn off this gene in the whole body of lab animals.

We injected animals with a collection of the CRISPR molecules that targeted the Myd88 gene and looked to see whether this reduced the quantity of antibodies that were produced to specifically fight our gene therapy viruses. We were excited to see that the animals that received our treatment using CRISPR produced less antibody against the virus.

This prompted us to ask what happens if we give the animal a second dose of the gene therapy virus. Usually the immune response against a gene therapy virus prevents the therapy from being administered multiple times. Thats because after the first dose, the immune system has seen the virus, and on the second dose, antibodies swiftly attack and destroy the virus before it can deliver its cargo.

We saw that animals receiving more than one dose did not show an increase in antibodies against the virus. And, in some cases, the effect of gene therapy improved compared with the animals in which we had not paused the Myd88 gene.

We also did a number of other experiments that proved that tweaking the Myd88 gene can be useful in fighting off other sources of inflammation. That could be useful in diseases like sepsis and even COVID-19.

While we are now beginning to improve this strategy in terms of controlling the activity of the Myd88 gene. Our results, now published in Nature Cell Biology,provide a path forward to program our immune system during gene therapies and other inflammatory responses using the CRISPR technology.

Samira Kiani, Associate Professor of Pathology, University of Pittsburgh

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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CRISPR Can Help Combat the Troubling Immune Response Against Gene Therapy - Gizmodo Australia

New Jersey, United States, The Gene Therapy Market report 2020 provides a detailed impression, describe the product industry scope and the market expanded insights and forecasts up to 2027. It shows market data according to industry drivers, restraints and opportunities, analyzes the market status, the industry share, size, future Trends and growth rate of the market. The Gene Therapy Market report is categorized by application, end user, technology, product / service types, and other, as well as by region. In addition, the report includes the calculated expected CAGR of chitosan acetate-market derivative from the earlier records of the Gene Therapy Market, and current market trends, which are organized with future developments.

Gene Therapy Market was valued at USD 3.69 Billion in 2019 and is projected to reach USD 24.78 Billion by 2027, growing at a CAGR of 26.9% from 2020 to 2027.

Gene Therapy Market, By Product

Viral Vectorso Adeno-associated virus vectorso Retroviral vectors Gammaretroviral vectors Lentiviral vectorso Other viral vectors (herpes simplex and adenoviral vectors) Non-viral Vectorso Oligonucleotideso Other non-viral vectors (plasmids and RNAi)

Gene Therapy Market, By Indication

Neurological Diseases Cancer Hepatological Diseases Duchenne Muscular Dystrophy Other Indications

Gene Therapy Market, By Delivery Method

Ex vivo In vivo

The report provides detailed coverage of the Gene Therapy Market, including structure, definitions, applications, and Industry Chain classifications. The Gene Therapy Market analysis is provided for the international markets including development trends, competitive landscape analysis, investment plan, business strategy, opportunities and development status of key regions. Development policies and plans are discussed and manufacturing processes and cost structures analyzed. This report also includes information on import / export consumption, supply and demand, costs, industry share, policy, Price, Sales and gross margins.

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Gene Therapy Market forecast up to 2027, with information such as company profiles, product picture and specification, capacity production, price, cost, revenue, and contact information. Upstream raw materials and equipment as well as downstream demand analyses are also carried out. The Gene Therapy Market size, development trends and marketing channels are analyzed. Finally, the feasibility of new investment projects is assessed and general research results are offered.

The Gene Therapy Market was created on the basis of an in-depth market analysis with contributions from industry experts. The report covers the growth prospects in the coming years and the discussion of the main providers.

To understand how the effects of COVID-19 are addressed in this report. A sample copy of the report is available at https://www.verifiedmarketresearch.com/product/gene-therapy-market/?utm_source=TDC&utm_medium=001

Verified Market Researchis a leading Global Research and Consulting firm servicing over 5000+ customers. Verified Market Research provides advanced analytical research solutions while offering information enriched research studies. We offer insight into strategic and growth analyses, Data necessary to achieve corporate goals, and critical revenue decisions.

Our 250 Analysts and SMEs offer a high level of expertise in data collection and governance use industrial techniques to collect and analyze data on more than 15,000 high impact and niche markets. Our analysts are trained to combine modern data collection techniques, superior research methodology, expertise, and years of collective experience to produce informative and accurate research.

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Gene Therapy Market is Thriving Worldwide 2020 | Trends, Growth and Profit Analysis, Forecast by 2027 - The Daily Chronicle

The report covers the forecast and analysis of the gene therapy market on a global and regional level. The study provides historical data from 2015 to 2018 along with a forecast from 2019 to 2027 based on revenue (USD Million). The study includes drivers and restraints of the gene therapy market along with the impact they have on the demand over the forecast period. Additionally, the report includes the study of opportunities available in the gene therapy market on a global level.

In order to give the users of this report a comprehensive view of the gene therapy market, we have included a competitive landscape and an analysis of Porters Five Forces model for the market. The study encompasses a market attractiveness analysis, wherein all the segments are bench marked based on their market size, growth rate, and general attractiveness.

Download Sample of This Strategic Report:https://www.kennethresearch.com/sample-request-10170423

The report provides company market share analysis to give a broader overview of the key players in the market. In addition, the report also covers key strategic developments of the market including acquisitions & mergers, new service launches, agreements, partnerships, collaborations & joint ventures, research & development, and regional expansion of major participants involved in the market on a global and regional basis.

The study provides a decisive view of the gene therapy market by segmenting the market based on the type, vector type, therapy area, and regions. All the segments have been analyzed based on present and future trends and the market is estimated from 2019 to 2027. The regional segmentation includes the current and forecast demand for North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.

Gene therapy is utilized for treating neurodegenerative disorders like Alzheimer, amyotrophic lateral sclerosis, and spinal muscular atrophy. Gene therapy is one of the key treatment kinds that will propel the market growth over the forecast period. Moreover, gene therapy also finds lucrative applications in precision medicine. In addition to this, a rise in the occurrence of cancer is prompting the demand to treat the disease through gene therapy.

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Based on the type, the market can be segregated into Germ Line Gene Therapy and Somatic Gene Therapy. In terms of vector type, the gene therapy industry can be divided into Viral Vectors, Non-Viral Vectors, and Human Artificial Chromosome. On the basis of therapy area, the market for gene therapy can be classified into Cancer, Neurological Diseases, Infectious Diseases, Genetic Disorders, Rheumatoid Arthritis, and Others.

The key players included in this market are Advanced Cell & Gene Therapy, Audentes Therapeutics, Benitec Biopharma, Biogen, Blubird Bio, Inc., Bristol-Myers Squibb Company, CHIESI Farmaceutici SPA, Eurofins Scientific, Geneta Science, Genzyme Corporation, Gilead, GlaxoSmithKline PLC, Human Stem Cells institute, Novartis AG, Orchard Therapeutics, Pfizer Inc., Sangamo therapeutics, Spark therapeutics, and Voyager Therapeutics.

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Gene Therapy Market By Industry Type, By Brand And Major Players 2020-2027 - The Daily Chronicle

TEL AVIV, Israel, Sept. 08, 2020 (GLOBE NEWSWIRE) -- VBL Therapeutics (Nasdaq: VBLT) announced today the successful completion of a Type B pre-IND meeting with the U.S. Food and Drug Administration (FDA) regarding the Company's development plan for VB-601. VB-601 is the Companys lead anti-MOSPD2 antibody for immune-inflammatory indications, for which a briefing package for the pre-IND meeting was submitted in June. Based on the FDA's feedback, VBL plans to advance its IND-enabling activities for VB-601 as planned.

We are very pleased with the outcome of the pre-IND meeting with the FDA, said Dror Harats, M.D., CEO of VBL Therapeutics. VB-601 is a first-in-class product candidate that has a completely novel mechanism of action. It can block the ability of monocyte to migrate to inflammatory sites, no matter which molecules try to attract them in. Therefore, it is important that we have reached alignment with the FDA on the path forward for VB-601. With this green light, we plan to continue to advance VB-601, aiming to start a first-in-human study in the second half of 2021.

About VB-601VBL is advancing a drug development program that is exploring the potential of MOSPD2 (motile sperm domain-containing protein 2), a protein that VBL has identified as a key regulator of cell motility, as a therapeutic target for inflammatory diseases. VB-601 is a proprietary anti-MOSPD2 monoclonal antibody which has potential for treatment of multiple chronic inflammatory diseases.

About VBLVascular Biogenics Ltd., operating asVBL Therapeutics, is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class treatments for cancer. VBLs lead oncology product candidate, ofranergene obadenovec (VB-111), is a first-in-class, targeted anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. It is conveniently administered as an IV infusion once every two months. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in a VBL-sponsored all comers phase 1 trial as well as in three VBL-sponsored tumor-specific phase 2 studies. Ofranergene obadenovec is currently being studied in a VBL-sponsored phase 3 potential registration trial for platinum-resistant ovarian cancer.

Forward Looking StatementsThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, look forward to, may, plan, potential, predict, project, should, will, would and similar expressions. These forward-looking statements may include, but are not limited to, statements regarding VBLs programs, including VB-601, including their pre-clinical and clinical development, therapeutic potential and clinical results. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. Among the factors that could cause actual results to differ materially from those described or projected herein include uncertainties associated generally with research and development, clinical trials and related regulatory reviews and approvals, the risk that historical clinical trial results may not be predictive of future trial results, that our financial resources do not last for as long as anticipated, and that we may not realize the expected benefits of our intellectual property protection. A further list and description of these risks, uncertainties and other risks can be found in our regulatory filings with theU.S. Securities and Exchange Commission, including in our annual report on Form 20-F for the year endedDecember 31, 2019, and subsequent filings with theSEC. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. VBL Therapeutics undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

INVESTOR CONTACT:

Michael RiceLifeSci Advisorsmrice@lifesciadvisors.com(646) 597-6979

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VBL Therapeutics Announces Successful Completion of Pre-IND Meeting with FDA Regarding Proposed Clinical Development of VB-601 - BioSpace

Seeing a child debilitated by illness is never easy.

When doctors tell youthere is nothing they nor you can do to help ease yourbaby's suffering, well, parents who know that type of helplessness often find it hard to describe.

We were told to take my son home and love him, becausehe probably wouldnt live past his second birthday, said Pace father Todd Hamrick. But, were way past that birthday now.

Hamricks son, Alek, was diagnosed at six months old with spinal muscular atrophy and not expected to live long enough to toddle. ButAlek, now 3, has beaten the odds and outlived that initial, bleak prognosis.

His parents attribute much of his success to his doctor, Richard Finkel, who entered Alek into a clinical trial for what they believe has been a wonder drug for their little boy, Evrysdi, which was recently approved by the Food and Drug Administration.

Whats hard to even get around is that people even bothered to research it, Todd Hamrick said. Its just a small amount of the population that has SMA. Its not like researching a blood pressure medication.

Spinal muscular atrophy, or SMA,is a genetic disorder caused by a loss of nerve cells that effect human motor function.

Essentially, those afflicted by SMA are made weak. Their muscleswaste away. In many cases, eventually, a person loses their ability to walk, to eat andevento breathe, and they die.

Aleks mother, Iwona Hamrick, is a nurse at a local hospital and wellremembers the moment she heardher sons diagnosis.

It was unimaginable. His pregnancy was normal, she said. We did genetic testing and it was negative. Unfortunately, at that time, they were not screening for his disease. So, from a healthy baby to a dying baby, you know?

The parents felt they had to travel, in more ways than one, to find the places and help that they could for their Alek.

Todd Hamrick said that he and wife decided to move from Gulf Breeze to Pace after Aleks diagnosis after feeling ostracized by many of their former acquaintances. The parents felt like some people who they used to know were made uncomfortable by their son's illness.

The area is very in-the-dark when it comes to children with issues, Todd Hamrick said. If your kids are healthy and you'rehealthy, its a great area. But, it doesnt attract the greatest talent or best and biggest facilities or endowments.

So, Alek traveled with his family to meet his future doctor, Finkel, at the Nemours Children's Hospital in Orlando.

Alekwas first treated with a gene therapy drug, and he made some response with that, Finkel told the News Journal. But more recently, he started on a second drug, which seems to be having an enhanced effect, I must say.

Finkel, an expert in the field of pediatric neurologic disorders, left Florida in March for a position leading the new Center for Experimental Neurotherapeutics at St. Jude Childrens Research Hospital in Memphis, Tennessee. Buthe has continued to monitor Aleks progress via video.

Both of the drugs he has received, the gene therapy and this new drug, Evrysdi, are designed to increase a certain protein in Aleks body that is deficient because of his genetic disorder, he explained. But they do it in different ways. The potential advantage of his new drug, Evrysdi because its an oral drug; you take it by mouth it goes into the stomach and into the bloodstream, and from there, it goes to all the tissues of the body.

And, we think that there is an enhanced effect, becauseit gets into the muscle tissue, Finkel continued. "These are very, very, early daysin trying to make assessments, soI dont want to say that we can come to any kind of conclusions yet."

However the cutting-edge drug works doesnt matter to a mother, whos just glad that it isworking.

It gave us hope. Thats for sure, Iwona Hamrick said. Becausewe felt helpless.

Since Alek started his new treatment last November, his strength has increased tremendously.

His muscle tone got better, Iwona Hamrick said. He is much stronger in the upper body, so much so, he is pushing his little wheelchair.

Alek can now cruise around his Pace home in an extraordinarily lite-weight wheelchair designed by a Swedish inventor who alsohas a child with SMA.

Alek had a lot of trouble before starting this medication even pushing it, Iwona Hamrick said. Sonowhe is just rolling around the house. Also, he is barring more weight on his legs.

Recently, Alek has started to be able to walk in a pool a huge milestone for the toddler.

But some worries remain the same.

Every day, Alek must use a type of breathing machine.

Its a cough assist machine, Todd Hamrick explained. We use it two times a day, when hes healthy. Becausewhere we can just clear our throats when we cough, he doesnt have that strength no lung strength.

Alek attends physical therapy, aqua-therapy,hippotherapy, occupational therapy and speech therapy sessions every week to try and ensure he remains healthy and continues to properly develop.

My worries have changed a lot, Todd Hamrick said. I used to worry my child was going to die. Now, Im worrying about if other kids will bully him at school.

"But that'sa great worry to have," he continued. "Compared to how it use to be, getting picked on is a great thing to worry about.

Colin Warren-Hicks can be reached at colinwarrenhicks@pnj.com or 850-435-8680.

Read or Share this story: https://www.pnj.com/story/news/2020/09/08/pace-florida-toddler-spinal-muscular-atrophy-helped-drug-evrysdi/5681766002/

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Pace toddler wasn't supposed to survive. Thanks to wonder drug, parents say he's thriving - Pensacola News Journal

NEW YORK and CLEVELAND, Sept. 08, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that the Company will participate in the following upcoming investor conferences.

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona’s 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 Company’s portfolio of AAV-based gene therapies also features ABO-201 for CLN3 disease. Abeona’s novel, next-generation AIM capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona’s fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit http://www.abeonatherapeutics.com.

Investor Contact: Greg Gin VP, Investor Relations Abeona Therapeutics +1 (646) 813-4709 ggin@abeonatherapeutics.com

Media Contact: Scott Santiamo Director, Corporate Communications Abeona Therapeutics +1 (718) 344-5843 ssantiamo@abeonatherapeutics.com

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Abeona Therapeutics to Participate in Upcoming Investor Conferences - Stockhouse

Roots Analysis has done a detailed study on Gene Therapy Market (3rd Edition), 2019-2030, covering key aspects of the industrys evolution and identifying potential future growth opportunities.

Key Market Insights

For more information, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

Table of Contents

4.2.1 Types of Viral Vectors

11.2 Analysis by Special Designations Awarded

11.3.1. Analysis by Target Indication

11.3.2. Analysis by Type of Vector Used

11.4.1. Analysis by Target Indication

11.4.2. Analysis by Type of Vector Used

11.5.1. Analysis by Target Indication

11.5.2. Analysis by Type of Vector Used

11.6.1. Analysis by Target Indication

11.6.2. Analysis by Type of Vector Used

11.7.1. Analysis by Target Indication

11.7.2. Analysis by Type of Vector Used

11.8.1. Analysis by Target Indication

11.8.2. Analysis by Type of Vector Used

11.9.1. Analysis by Target Indication

11.9.2. Analysis by Type of Vector Used

11.10.1. Analysis by Target Indication

11.10.2. Analysis by Type of Vector Used

11.11.1. Analysis by Target Indication

11.11.2. Analysis by Type of Vector Used

11.12.1. Analysis by Target Indication

11.12.2. Analysis by Type of Vector Used

11.13.1. Analysis by Target Indication

11.13.2. Analysis by Type of Vector Used

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Contact Information

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The Gene Therapy Market is projected to grow at an annualized rate of 45%, till 2030 - Scientect

[190+ Pages PDF Report] Facts & Factors (FnF) published a market research report onGene Therapy Market: By Size, Share, Growth Factors, Top Players, COVID-19 Analysis and Forecast, 2020-2026that includes a research report with TOC including a list of tables and figures in its research offerings.

Gene Therapy Market Report: Industry Insights, 2020-2026

This multi-client research study on theGene Therapy marketprovides in-depth research and analysis into Gene Therapy industry trends, market developments and technological insights. The report provides data and analysis of Gene Therapy penetration across application segments across countries and regions. The report presents a strategic analysis of the Gene Therapyy market through key drivers, challenges, opportunities, and growth contributors.

The global Gene Therapy market delivers value to customers through reliable market size for 2020 on the basis of demand and price analysis. The report presents near term and long term forecast of the addressable Gene Therapy market size to 2026.

The report presents an introduction to the Gene Therapy market in 2020, analyzing the COVID-19 impact both quantitatively and qualitatively.

Request an Exclusive FREE SAMPLE REPORT of Gene Therapy Market:https://www.fnfresearch.com/sample/gene-therapy-market-by-type-germ-line-gene

(The sample of this report is readily available on request).

The FREE PDF Sample Report Includes:

(Note: The sample of this report is updated with COVID-19 impact analysis before delivery)

Some of Following Top Market Players Profile Included in This Report:

2020 Gene Therapy Market: COVID-19 Impact Analysis

The global rise of COVID-19 has many businesses struggling and confused about what steps to take to minimize the economic impact. A simple look at the stock market will tell you that coronavirus has led to a volatile economy, but there are numerous other factors at play.

The long term COVID-19 impact on the business industry is largely still unknown; hence,Facts and Factors market researchanalysts have already covered the effects of COVID-19 on the business industry at a large level, as well as global and regional levels. Through our coverage below, get a better understanding of the business and economic implications coronavirus has on trends like remote working, consumer shopping behavior, global advertising spend, and essential industries like food, medical, travel, and transportation.

Download the Free Sample Report PDF Brochure Including COVID-19 Business Impact:https://www.fnfresearch.com/sample/gene-therapy-market-by-type-germ-line-gene

Gene Therapy Market 2020: Research Scope & Coverage

The report covers Gene Therapy market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the markets historic and forecast market growth by geography. The market size analysis gives the market size covering both the historic growth of the market, the impact of the COVID 19 virus, and forecasting its recovery. The report also provides a comprehensive analysis of current & future trends and emerging avenues for the growth of this market along with this research also offers an insight into the competitive landscape in terms of new technological developments, untapped segments, and value chain analysis.

The report, Gene Therapy Market, provides up-to-date information about market size, share shifts, and potential risks, as well as in-depth knowledge of products and services, which can help in planning and in executing category management activities. It focuses on the cost-saving aspects of procurement and on providing insights that can lead to the optimization of category spend.

Get Free Customized Copy of Gene Therapy Market Report @https://www.fnfresearch.com/customization/gene-therapy-market-by-type-germ-line-gene

(We customize your report according to your research need. Ask our sales team for report customization.)

Competitive Landscape

The market appears to be fragmented and with the presence of several. This Gene Therapy market research report will help clients identify new growth opportunities and design unique growth strategies by providing a comprehensive analysis of the markets competitive landscape and offering information on the products offered by companies.

Key Takeaways from this Gene Therapy Market Report

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Facts & Factorsis a leading market research company and offers customized research reports and consulting services. Facts & Factors aims at management consulting, industry chain research, and advanced research to assist our clients by providing planned revenue model for their business. Our report and services are used by prestigious academic institutions, start-ups, and companies globally to understand the international and regional business background.

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Know Top Players and Growing Trends in Gene Therapy Market 2020 Research Report and Industry Forecast till 2026 - Galus Australis

MarketsandResearch.biz has published the latest market research study on Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market 2020 by Company, Type and Application, Forecast to 2025 which investigates a few critical features of the market such as industry condition, division examination, market insights. The report studies the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market share, competition landscape, market share, growth rate, future trends, market drivers, opportunities and challenges, sales channels. The report has referenced down to earth ideas of the market in a straightforward and unassuming way in this report. The research contains the categorization of the market by top players/brands, region, type, and end-user. The report exhaustive essential investigation of current market trends, opportunities, challenges, and detailed competitive analysis of the industry players in the market.

The research report has comprehensively included numbers and figures with the help of graphical and pictorial representation which embodies more clarity on the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market. Then the report delivers key information about market players such as company overview, total revenue (financials), market potential, global presence, as well as market share, prices, production sites and facilities, products offered, and strategies adopted by them. Market status and outlook of global and major regions, from angles of players, countries, product types, and end industries have been analyzed.

NOTE: Our report highlights the major issues and hazards that companies might come across due to the unprecedented outbreak of COVID-19.

DOWNLOAD FREE SAMPLE REPORT: https://www.marketsandresearch.biz/sample-request/83910

Key strategic manufacturers included in this report: BioReliance, Richter-Helm, UniQure, Cobra Biologics, MassBiologics, Oxford BioMedica, Lonza, MolMed, FinVector, FUJIFILM Diosynth Biotechnologies, Brammer Bio, bluebird bio, Aldevron, Spark Therapeutics, VGXI, Biovian, Eurogentec, Novasep, PlasmidFactory, Cell and Gene Therapy Catapult, Vigene Biosciences

Market Potential:

Key market vendors have been predicted to obtain the latest opportunities as there has been an increased emphasis on spending more on the work of research and development by many of the manufacturing companies. Also, many of the market contenders are forecasted to make a foray into the emerging economies to find new opportunities. The global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market has gone through rapid business transformation by good customer relationships, drastic and competitive growth, significant changes within the market, and technological advancement in this market.

Geographically, this report is segmented into several key countries, with market size, growth rate, import and export of in these countries from 2015 to 2020, which covering: North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina), MENA (Saudi Arabia, UAE, Turkey and South Africa)

The market can be segmented into product types as: AAV, Adenoviral, Lentiviral, Retroviral, Plasmid DNA, Other Vectors

The market can be segmented into applications as: Cancers, Inherited Disorders, Viral Infections, Others

ACCESS FULL REPORT: https://www.marketsandresearch.biz/report/83910/global-viral-vectors-non-viral-vectors-and-gene-therapy-manufacturing-market-2020-by-company-type-and-application-forecast-to-2025

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Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market 2020 | Scope of Current and Future Industry 2026 - Good Night, Good...

Notice: This Content doesnt contain all the Information of the Report please fill the form (via link) and get all interesting information just one click in PDF with the latest update with chart and Table of Content

The report offers a systematic presentation of the existing trends, growth opportunities, market dynamics that are expected to shape the growth of the Cell Therapy Manufacturing Market. The various research methods and tools were involved in the market analysis, to uncover crucial information about the market such as current & future trends, opportunities, business strategies and more, which in turn will aid the business decision-makers to make the right decision in future.

This Report Covers Leading Companies Associated in Worldwide Cell Therapy Manufacturing Market: Pharmicell, Merck Group, Dickinson and Company, Thermo Fisher, Lonza Group, Miltenyi Biotec GmBH, Takara Bio Group, STEMCELL Technologies, Cellular Dynamics International, Becton, Osiris Therapeutics, Bio-Rad Laboratories, Inc., Anterogen, MEDIPOST, Holostem Terapie Avanazate, Pluristem Therapeutics, Brammer Bio, CELLforCURE, Gene Therapy Catapult EUFETS, MaSTherCell, PharmaCell, Cognate BioServices and WuXi AppTec.

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Brief Introduction to the research report. Table of Contents (Scope covered as a part of the study) Top players in the market Research framework (Structure Of The Report) Research methodology adopted by Coherent Market Insights

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The report begins with a brief introduction and market overview of the Cell Therapy Manufacturing industry followed by its market scope and size. Next, the report provides an overview of market segmentation such as type, application, and region. The drivers, limitations, and opportunities for the market are also listed along with current trends and policies in the industry.

The key players profiled in this report include:Pharmicell, Merck Group, Dickinson and Company, Thermo Fisher, Lonza Group, Miltenyi Biotec GmBH, Takara Bio Group, STEMCELL Technologies, Cellular Dynamics International, Becton, Osiris Therapeutics, Bio-Rad Laboratories, Inc., Anterogen, MEDIPOST, Holostem Terapie Avanazate, Pluristem Therapeutics, Brammer Bio, CELLforCURE, Gene Therapy Catapult EUFETS, MaSTherCell, PharmaCell, Cognate BioServices and WuXi AppTec.

Regions included:

o North America (United States, Canada, and Mexico)

o Europe (Germany, France, UK, Russia, and Italy)

o Asia-Pacific (China, Japan, Korea, India, and Southeast Asia)

o South America (Brazil, Argentina, Colombia)

o The Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

The PDF Research only provides a Table of Contents (ToC), the scope of the report and research framework of the report.

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Key Benefits:

o This study gives a detailed analysis of drivers and factors limiting the market expansion of Cell Therapy Manufacturing

o The micro-level analysis is conducted based on its product types, end-user applications, and geographies

o Porters five forces model gives an in-depth analysis of buyers and suppliers, threats of new entrants & substitutes and competition amongst the key market players

o By understanding the value chain analysis, the stakeholders can get a clear and detailed picture of this Cell Therapy Manufacturing market

The research study can answer the following Key questions:

What will be the progress rate of the Cell Therapy Manufacturing Market for the conjecture period, 2020 2027?What are the prominent factors driving the Cell Therapy Manufacturing Market across different regions?Who are the major vendors dominating the Cell Therapy Manufacturing industry and what are their winning strategies?What will be the market scope for the estimated period?What are the major trends shaping the expansion of the industry in the coming years?What are the challenges faced by the Cell Therapy Manufacturing Market?

Table of Contents

Report Overview: It includes the Cell Therapy Manufacturing market study scope, players covered, key market segments, market analysis by application, market analysis by type, and other chapters that give an overview of the research study.

Executive Summary: This section of the report gives information about Cell Therapy Manufacturing market trends and shares, market size analysis by region and analysis of global market size. Under market size analysis by region, analysis of market share and growth rate by region is provided.

Profiles of International Players: Here, key players of the Cell Therapy Manufacturing market are studied on the basis of gross margin, price, revenue, corporate sales, and production. This section gives a business overview of the players and shares their important company details.

Regional Study: All of the regions and countries analyzed in the Cell Therapy Manufacturing market report is studied on the basis of market size by application, the market size by product, key players, and market forecast.

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Cell Therapy Manufacturing Market Growth, Demand, And Value Is Increasing In The Upcoming Years - The Daily Chronicle

Chicago, United States:The global Cell and Gene Therapy Market report offers a complete research study that includes accurate estimations of market growth rate and size for the forecast period 2020-2025. It offers a broad analysis of market competition, regional expansion, and market segmentation by type, application, and geography supported by exact market figures. The all-inclusive market research report also offers Porters Five Forces Analysis and profiles some of the leading players of the global Cell and Gene Therapy Market. It sheds light on changing market dynamics and discusses about different growth drivers, market challenges and restraints, and trends and opportunities in detail. Interested parties are provided with market recommendations and business advice to ensure success in the global Cell and Gene Therapy Market.

Top Key players cited in the report:Amgen Inc., bluebird bio, Inc., Dendreon Pharmaceuticals LLC., Fibrocell Science, Inc., Human Stem Cells Institute, Kite Pharma, Inc., Kolon TissueGene, Inc., Novartis AG, Orchard Therapeutics plc., Organogenesis Holdings Inc., Pfizer, Inc., RENOVA THERAPEUTICS, .

Get Free PDF Sample Copy of this Report to understand the structure of the complete report: (Including Full TOC, List of Tables & Figures, Chart): https://www.reporthive.com/request_sample/2369809

The final report will add the analysis of the Impact of Covid-19 in this report Cell and Gene Therapy Market

Cell and Gene Therapy Marketreports offers important insights which help the industry experts, product managers, CEOs, and business executives to draft their policies on various parameters including expansion, acquisition, and new product launch as well as analyzing and understanding the market trends.

Each segment of the global Cell and Gene Therapy market is extensively evaluated in the research study. The segmental analysis offered in the report pinpoints key opportunities available in the global Cell and Gene Therapy market through leading segments. The regional study of the global Cell and Gene Therapy market included in the report helps readers to gain a sound understanding of the development of different geographical markets in recent years and also going forth. We have provided a detailed study on the critical dynamics of the global Cell and Gene Therapy market, which include the market influence and market effect factors, drivers, challenges, restraints, trends, and prospects. The research study also includes other types of analysis such as qualitative and quantitative.

Global Cell and Gene Therapy Market: Competitive Rivalry

The chapter on company profiles studies the various companies operating in the global Cell and Gene Therapy market. It evaluates the financial outlooks of these companies, their research and development statuses, and their expansion strategies for the coming years. Analysts have also provided a detailed list of the strategic initiatives taken by the Cell and Gene Therapy market participants in the past few years to remain ahead of the competition.

Global Cell and Gene Therapy Market: Regional Segments

The chapter on regional segmentation details the regional aspects of the global Cell and Gene Therapy market. This chapter explains the regulatory framework that is likely to impact the overall market. It highlights the political scenario in the market and the anticipates its influence on the global Cell and Gene Therapy market.

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Report Highlights

Table of Contents

Report Overview:It includes six chapters, viz. research scope, major manufacturers covered, market segments by type, Cell and Gene Therapy market segments by application, study objectives, and years considered.

Global Growth Trends:There are three chapters included in this section, i.e. industry trends, the growth rate of key producers, and production analysis.

Cell and Gene Therapy Market Share by Manufacturer:Here, production, revenue, and price analysis by the manufacturer are included along with other chapters such as expansion plans and merger and acquisition, products offered by key manufacturers, and areas served and headquarters distribution.

Market Size by Type:It includes analysis of price, production value market share, and production market share by type.

Market Size by Application:This section includes Cell and Gene Therapy market consumption analysis by application.

Profiles of Manufacturers:Here, leading players of the global Cell and Gene Therapy market are studied based on sales area, key products, gross margin, revenue, price, and production.

Cell and Gene Therapy Market Value Chain and Sales Channel Analysis:It includes customer, distributor, Cell and Gene Therapy market value chain, and sales channel analysis.

Market Forecast Production Side: In this part of the report, the authors have focused on production and production value forecast, key producers forecast, and production and production value forecast by type.

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About Us:Report Hive Research delivers strategic market research reports, statistical survey, and Industry analysis and forecast data on products and services, markets and companies. Our clientele ranges mix of United States Business Leaders, Government Organizations, SMEs, Individual and Start-ups, Management Consulting Firms, and Universities etc. Our library of 600,000+ market reports covers industries like Chemical, Healthcare, IT, Telecom, Semiconductor, etc. in the USA, Europe Middle East, Africa, Asia Pacific. We help in business decision-making on aspects such as market entry strategies, market sizing, market share analysis, sales and revenue, technology trends, competitive analysis, product portfolio and application analysis etc.

The rest is here:
COVID-19 Impact on Cell and Gene Therapy Market Research, Size, Growth And Trends 2020 to 2025: Keyplayer-Amgen Inc., bluebird bio, Inc. - Scientect

AVROBIO, a US-based company, developing lentiviral-based gene therapies, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for the Companys investigational gene therapy, AVR-RD-02, for the treatment of Gaucher disease.

Get a complete list of the presentations,here.

What is AVR-RD-02: AVR-RD-02 consists of the patients own hematopoietic stem cells, genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease. It is an ex vivo lentiviral-based investigational gene therapy. The stem cells are genetically modified to express functional glucocerebrosidase (GCase), which reduces levels of glucosylceramide and glucosylsphingosine, the accumulated substances which cause the symptoms of Gaucher disease.

How advanced is AVR-RD-02 development program: The Company is actively recruiting in Canada for its Phase 1/2 clinical trial of AVR-RD-02, which seeks to evaluate the safety and efficacy of the therapy in patients with Type 1 Gaucher disease.

What is an orphan drug designation: Orphan-drug designation provides special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor. For a drug to qualify for orphan designation both the drug and the disease or condition must meet certain criteria specified in the ODA and FDAs implementing regulations at 21 CFR Part 316. Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received orphan designation is not subject to a prescription drug user fee unless the application includes an indication for other than the rare disease or condition for which the drug was designated.

What is the gene therapy pipeline like for the treatment of Gaucher disease: There are only a few gene therapy candidates that are being developed for Gaucher disease.

For further information, check out the report here

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Roots Analysis is one of the fastest growing market research companies, sharing fresh and independent perspectives in the bio-pharmaceutical industry. The in-depth research, analysis and insights are driven by an experienced leadership team which has gained many years of significant experience in this sector. If youd like help with your growing business needs, get in touch at [emailprotected]

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Gaurav Chaudhary

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Gene Therapy Pipeline Update: AVROBIO Receives Orphan-Drug Designation | Roots Analysis - The News Brok

The report covers the forecast and analysis of the Cell and Gene Therapy Consumables market on a global and regional level. The study provides historical data from 2015 to 2018 along with a forecast from 2019 to 2027 based on revenue (USD Million). The study includes drivers and restraints of the Cell and Gene Therapy Consumables market along with the impact they have on the demand over the forecast period. Additionally, the report includes the study of opportunities available in the Cell and Gene Therapy Consumables market on a global level.

Download Sample of This Strategic Report:https://www.kennethresearch.com/sample-request-10172657

In order to give the users of this report a comprehensive view of the Cell and Gene Therapy Consumables market, we have included a competitive landscape and an analysis of Porters Five Forces model for the market. The study encompasses a market attractiveness analysis, wherein all the segments are bench marked based on their market size, growth rate, and general attractiveness.

The report provides company market share analysis to give a broader overview of the key players in the market. In addition, the report also covers key strategic developments of the market including acquisitions & mergers, new service & product launches, agreements, partnerships, collaborations & joint ventures, research & development, and regional expansion of major participants involved in the market on a global and regional basis.

The study provides a decisive view of the Cell and Gene Therapy Consumables market by segmenting the market based on product type, application/therapeutics, and regions. All the segments have been analyzed based on present and future trends and the market is estimated from 2019 to 2027. The regional segmentation includes the current and forecast demand for North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.

A rise in the awareness about the gene & cell therapies will propel the market growth during the period from 2019 to 2027. Nevertheless, conducting randomized control tests will inhibit the expansion of the market during the forecast timeline. However, the growing trend for treating neurodegenerative ailments through the use of gene treatment will proliferate the market growth over the forecast period.

The expansion of the market during the forecast timespan is owing to the high frequency of chronic ailments including cancer and heart disorders. Apart from this, inflation in the occurrence of these disorders produces lucrative demand for enhanced therapies and this will culminate in the market demand over the forecast timespan.

Request For Full Report:https://www.kennethresearch.com/sample-request-10172657

Based on the product type, the market is sectored into Kits & Buffers, Diagnostic Assay, Culture Medium, and Cryopreservation Media. Application/ Therapeutics- wise, the market for cell and gene therapy consumables are classified into Cardiovascular, Urology, Dermatology, Critical Care, Respiratory, Endocrine & Metabolic, Neuroscience, Hematology & Oncology, Obstetrics, Immunology, and Gastroenterology.

Some of the key players in the market include Amgen Inc., ATLANTA BIOLOGICALS, bluebird bio, Inc., Cook, Dendreon Pharmaceuticals, LLC, Fibrocell Science, Inc., General Electric, Kolon TissueGene, Inc., Orchard Therapeutics plc., Pfizer, Inc., PromoCell GmbH, RENOVA THERAPEUTICS, Sibiono GeneTech Co. Ltd., Spark Therapeutics, Inc., Vericel, Helixmith Co., Ltd., and Vitrolife.

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Kenneth Research is a reselling agency providing market research solutions in different verticals such as Automotive and Transportation, Chemicals and Materials, Healthcare, Food & Beverage and Consumer Packaged Goods, Semiconductors, Electronics & ICT, Packaging, and Others. Our portfolio includes set of market research insights such as market sizing and market forecasting, market share analysis and key positioning of the players (manufacturers, deals and distributors, etc), understanding the competitive landscape and their business at a ground level and many more. Our research experts deliver the offerings efficiently and effectively within a stipulated time. The market study provided by Kenneth Research helps the Industry veterans/investors to think and to act wisely in their overall strategy formulation

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Cell and Gene Therapy Consumables Market 2020-2027/ Size, Segments and Supply Demand Forecast - StartupNG

The Global Gene Therapy Market report focuses on market size, status, and forecast 2020-2024, along with this, the report also focuses on market opportunities and threats, tactical decision-making, and evaluating the market. The Gene Therapy market report delivers data and information on changing investment structure, technological advancements, market tendencies and developments, capacities, and detailed information about the key players of the global market. In addition to this, the report also involves the development of the Gene Therapy market in the major regions across the world.

Cutting-edge released the research study on Global Gene Therapy Market, which deals a exhaustive overview of the factors influencing the global business scope. Gene Therapy Market research report shows the latest market insights, current situation analysis with upcoming trends, and breakdown of the products and services. The Gene Therapy Industry Report delivers key statistics on the market status, size, share growth factors of the Gene Therapy .

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Top Leading players of Gene Therapy Market Covered in the Report:

Bluebird BioSangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVical Inc.Advantagene

The report has enclosed key geographic regions such as Europe, Japan, United States, India, Southeast Asia and Europe. As far as the sub-regions, North America, Canada, Medico, Australia, Asia-Pacific, India, South Korea, China, Singapore, Indonesia, Japan, Rest of Asia-Pacific, Germany, United Kingdom, France, Spain, Italy, Rest of Europe, Russia, Central & South America, Middle East & Africa are included.

Key Market Segmentation of Gene Therapy :

On the basis of types, the Gene Therapy Market from 2020 to 2024 is primarily split into:

Ex vivoIn Vivo

On the basis of applications, the Gene Therapy Market from 2020 to 2024 covers:

CancerMonogenicInfectious diseaseCardiovascular diseaseOther

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The Gene Therapy Market Research Report furthermore delivers a local examination of the market with a high focus on showcase development, development rate, and development potential. The research report calculates marketplace length estimate to analyze investment potentials and growth.

In this study, the years considered to estimation the market size of the Gene Therapy Industry Market: History Year: 2014-2018Base Year: 2018Estimated Year: 2019Forecast Year 2019 to 2024

The Gene Therapy market report provides answers to the following key questions:

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Global Gene Therapy Market 2020-2024 Expected To Witness The Highest Growth During Forecast period - Scientect

The global Gene Therapies for Cancer Treatment market study covers the projection size of the market both in terms of value (Mn/Bn US$) and volume (x units). The report estimates the lookup of different local distributors in the overall market and provides the market size of the Gene Therapies for Cancer Treatment market using both bottom-up and top-down approaches. To investigate the key players and their market contribution, primary and secondary research has been comprehensively performed. In addition, all the figures, subdivisions, and shares have been collected with the help of trustworthy sources.

In the Gene Therapies for Cancer Treatment market research study, 2018 is considered as the base year, and 2019-2029 is considered as the forecast period to predict the market size. The report identifies each Gene Therapies for Cancer Treatment market player on the basis of market share, production portfolio, and growth rate. In addition, the research study analyzes the strengths, weaknesses, opportunities and threats of the players.

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Global Gene Therapies for Cancer Treatment market report on the basis of market players

The following players are covered in this report:Takara BioTocagenVBL TherapeuticsCold GenesysGenprexMomotaro-GeneMultiVirSynerGene TherapeuticsZiopharm OncologyAnchiano TherapeuticsCelgeneCelsionBluebird BioGene Therapies for Cancer Treatment Breakdown Data by TypeSomatic Cell Gene Therapy (SCGT)Germline Gene Therapy (GGT)Gene Therapies for Cancer Treatment Breakdown Data by ApplicationCancer Research CentersDiagnostic LaboratoriesCancer HospitalsOthers

The report provides market share, consumption pattern, and influencing factors of each region. Prominent countries driving the regional growth are also covered in the report.

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The Gene Therapies for Cancer Treatment market report answers the following questions:

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Gene Therapies for Cancer Treatment Market Projected to Witness Vigorous Expansion by 2019-2025 - Scientect

Adenovirus vectors have become a popular delivery platform for gene therapy and vaccines. However, many people have preexisting immunity against adeno-associated viruses (AAVs), which could significantly reduce vector uptake and therefore render the therapeutic cargo they carry ineffective. Even in individuals that lack preexisting immunity, the first inoculation of a vector could induce immune responses that can thwart repeat administration.

To overcome that obstacle, scientists at the University of Pittsburgh created a CRISPR-based system that they say could effectively prevent immunity to the viral vector. The CRISPR tool briefly suppressedgenes that are related to anti-adenovirus antibody production and helped achieve better gene therapy uptake in mice. The results werepublished in Nature Cell Biology.

Samira Kiani, the studys co-senior author, has co-founded SafeGen Therapeutics with the goal to bring this platform to the clinic to enable safer gene therapy.

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Many clinical trials fail because of the immune response against AAV gene therapy, Kiani said in a statement. And then you cant re-administer the shot because people have developed immunity.

One recent case in point is CanSino Biologics adenovirus type 5 (Ad5)-vectored COVID-19 vaccine. In the shots phase 2 trial, about half of the participants had high levels of preexisting Ad5 neutralizing antibody. These individuals were found to produce significantly lower immune responses to the vaccine than did those with low preexisting anti-Ad5 immunity.

Kiani and colleagues set out to modify the gene expression that's associated with an immune response against AAV, but only temporarilyjust long enough for the therapeutic content to do its jobbecause they didnt want to tamper with normal immune functions.

RELATED:Hopkins team invents non-viral system for getting gene therapy into cells

CRISPR, which can edit out a defective gene or add in a functional one, is already being explored as a gene therapy strategy. So the University of Pittsburgh researchers figured they could hit two birds with one stone by utilizing CRISPR to also control the immune response.

They treated mice with the CRISPR-based system and then re-exposed them to AAV, and the animals didnt make more antibodies against the virus. The treated mice were more receptive to subsequent AAV-delivered gene therapy than were the control animals, the team reported.

Gene therapies hold promise as potential cures to many hard-to-treat diseases that would otherwise require chronic treatment. So scientists have been trying to perfect their transport vehicles for better safety and efficacy.

A team led by scientists at the San Raffaele Telethon Institute for Gene Therapy in Italy, for example,attached the CD47 protein to the surface of lentiviral vectors so they could escape detection and destruction by the immune system. And researchersfrom Johns Hopkins University developed a nonviral delivery system that uses a polymer nano-container, whichreleases the therapy once inside the cell.

Now, Kianihopes to validate the CRISPR-based technology at SafeGen. In addition to its potential as a gene therapy delivery platform, the researchers also found that their method can prevent or treat sepsis in mice, suggesting it might also be useful for treating inflammatory conditions.

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Using CRISPR to improve viral vectors for gene therapy - FierceBiotech