StemCell Therapy

SunTrust Robinson just initiated coverage on SGMO with a "buy" rating

The shares of Sangamo Therapeutics Inc (NASDAQ:SGMO) are up 1.7% at $10 at last check, after moving higher on the charts in their last five-consecutive closes. Additionally, SunTrust Robinson just initiated coverage on the gene therapy specialist with a "buy" rating and a price target of $22 -- a 120% premium to current levels.

Coming into today, the majority of analysts covering SGMO shared this bullish sentiment. Of the six in coverage, four brokerages rated it a "strong buy," while just two called it a "hold." Meanwhile, the consensus 12-month target price of $20.17 is more than double the equity's current perch and sits at a level the stock hasn't touched since April 2018.

The options pits have taken a bullish approach to SGMO, too. On the International Securities Exchange (ISE), Cboe Options Exchange (CBOE), and NASDAQ OMX PHLX (PHLX)the security sports a 50-day call/put volume ratio of 22.21, which sits in the highest percentile of its annual range, indicating an unusually heavy appetite for calls of late.

Echoing this, Sangamo stock's Schaeffer's put/call open interest ratio (SOIR) of 0.10 sits in just the 2nd percentile of its annual range, suggesting short-term option players have rarely been more call-biased during the past 12 months.

SGMO saw a notable rally off its mid-March, three-year low of $4.81, surging toward an annual high of annual high of $12 on June 1. The shares eventually gapped lower after weeks of consolidation just below the area and now both the $10 mark and the security's 20-day moving average are keeping the security from closing this bear gap, though it still boasts an 18% lead for the year.

Read more:
Gene Therapy Stock Moves Higher on Analyst Upgrade - Schaeffers Research

The global 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 .

For Better Understanding, Download FREE Sample Copy of Cancer Gene Therapy Report in Just One Single Step @ https://www.researchmoz.us/enquiry.php?type=S&repid2271992

Key Questions Answered in This Report:

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 Insights that Study is going to provide: The 360-degree Cancer Gene Therapy market overview based on a global and regional level Market Share & Sales Revenue by Key Players & Emerging Regional Players Competitors In this section, various Cancer Gene Therapy industry leading players are studied with respect to their company profile, product portfolio, capacity, price, cost, and revenue. A separate chapter on Cancer Gene Therapy market Entropy to gain insights on Leaders aggressiveness towards market [Merger & Acquisition / Recent Investment and Key Developments] Patent Analysis** No of patents / Trademark filed in recent years.

Grab Maximum Discount on Cancer Gene Therapy Market Research Report [Single User | Multi User | Corporate Users] @https://www.researchmoz.us/enquiry.php?type=E&repid2271992

Table of Content:Global Cancer Gene Therapy Market Size, Status and Forecast 20261. Report Overview2. Market Analysis by Types3. Product Application Market4. Manufacturers Profiles/Analysis5. Market Performance for Manufacturers6. Regions Market Performance for Manufacturers7. Global Cancer Gene Therapy Market Performance (Sales Point)8. Development Trend for Regions (Sales Point)9. Upstream Source, Technology and Cost10. Channel Analysis11. Consumer Analysis12. Market Forecast 2020-202613. Conclusion

For More Information Kindly Contact: ResearchMozMr. Rohit Bhisey,90 State Street,Albany NY,United States 12207Tel: +1-518-621-2074USA-Canada Toll Free: 866-997-4948Email: [emailprotected]Media Release @ https://www.researchmoz.us/pressreleaseFollow me on Blogger: https://trendingrelease.blogspot.com/

Excerpt from:
Cancer Gene Therapy Market Is Set to Boom in 2020, Coming Years - Jewish Life News

The Illinois Department of Public Health (IDPH) now includes spinal muscular atrophy (SMA) as part of its newborn screening effort.

The addition of SMA the 49th disorder in the states screening program means that every baby born in Illinois will be tested for the autosomal recessive neurodegenerative disease that affectsone in every 8,000 to 10,000 people worldwide.

Spinal Muscular Atrophy is a disease that robs people of physical strength, including the ability to walk, eat, or breathe, Ngozi Ezike, MD, director of the IDPH, said in a press release. It is the number one genetic cause of death for infants. Early diagnosis of babies with SMA can lead to potentially life-saving interventions. By screening every baby born in Illinois, we hope to identify cases early so therapy can begin as soon as possible, she said.

SMA is characterized by progressive muscle weakness caused by the loss of specialized nerve cells motor neurons in the spinal cord and the part of the brain connected to the spinal cord. Because motor neurons control voluntary muscle movements, their loss leads to muscular weakness and atrophy. Movement becomes increasingly slower, and the ability to control voluntary movement ultimately may be totally lost.

Starting treatment early is the only way to prevent motor neuron loss. In fact, infants identified as having SMA should begin therapy before SMA symptoms appear. Currently, Biogens Spinraza and the gene therapy Zolgensma, developed byNovartissubsidiaryAveXis,are the only disease-modifying SMA treatments available. Other medications aim to manage SMA symptoms or prevent complications. Several experimental therapiesalso are being developed.

In 2018, SMA was added to the federal Recommended Uniform Screening Panel for newborn testing (RUSP). The RUSP is a list of disorders the U.S. Department of Health and Human Services recommends for states universal newborn screening programs. Such disorders are chosen based on evidence that supports the potential net benefit of screening, the ability of states to screen for them, and the availability of effective therapies.

In preparation for adding SMA to its screening program, the IDPH bought new equipment, developed new test methods, and modified computer systems to provide lab results and facilitate follow-up tracking.

The agency also obtained test validation from the federalClinical Lab Improvement Amendments (CLIA). The CLIA regulate laboratory testing and require clinical labs to be certified by the Centers for Medicare and Medicaid Services before they can accept human samples for diagnostic testing.

Newborn screening for SMA tests for the presence of the survival motor neuron 1 (SMN1) gene. If testing results reveal that the gene is absent or markedly reduced in signal, immediate referral will be made to a pediatric multidisciplinary neuromuscular center for diagnostic testing and evaluation. This sitehas for more information.

Still, screening newborns for genetic diseases that have therapies that can prevent disease progression has a long way to go in the U.S. As it is, no state currently tests for all 35 disorders federally recommended, and even those that come close can be hamstrung by competing interests and obligations. Each state decides the scope of its newborn screenings.

The 2019 approval of Zolgensma Spinraza has been on the market since 2016 sparked a pushamong some scientists, physicians, and patient advocates to have all babies around the world tested for the disease.

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours dElegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled Belle of the Concours.

Total Posts: 85

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

Read more from the original source:
Illinois Department of Public Health Expands Newborn Screening to SMA - SMA News Today

By Jocelyn KaiserJun. 29, 2020 , 5:20 PM

Two boys have died after receiving high doses of a gene therapy treatment for their rare muscle disease, Biopharma Dive reports. The patients, born with x-linked myotubular myopathy, developed liver problems that apparently led to sepsis, according to a 23 June letter to patient groups from trial sponsor Audentes Therapeutics. They were older patients and had existing liver disease; several younger patients who got lower doses of the treatment have done well and now breathe on their own without a ventilator. The U.S. Food and Drug Administration (FDA) has put the trial on hold. Audentes, which had stopped enrollment before the deaths, has postponed plans to seek FDA approval for the drug this year. Since the 1999 death of gene therapy patient Jesse Gelsinger slowed research, the field has rebounded and FDA has approved two rare disease treatments. But recently, animal studies have suggested high doses of gene therapy can cause dangerous liver toxicity.

Read more:
Two deaths in gene therapy trial for rare muscle disease - Science Magazine

In recent months, even as our attention has been focused on the coronavirus outbreak, there have been a slew of scientific breakthroughs in treating diseases that cause blindness.

Researchers at U.S.-based Editas Medicine EDIT, +1.45% and Ireland-based Allergan (now owned by AbbVie ABBV, +0.32% ) have administered CRISPR for the first time to a person with a genetic disease. This landmark treatment uses the CRISPR approach to a specific mutation in a gene linked to childhood blindness. The mutation affects the functioning of the light-sensing compartment of the eye, called the retina, and leads to loss of the light-sensing cells.

According to the World Health Organization, at least 2.2 billion people in the world have some form of visual impairment. In the United States, approximately 200,000 people suffer from inherited forms of retinal disease for which there is no cure. But things have started to change for good. We can now see light at the end of the tunnel.

I am an ophthalmology and visual sciences researcher, and am particularly interested in these advances because my laboratory is focusing on designing new and improved gene therapy approaches to treat inherited forms of blindness.

Gene therapy involves inserting the correct copy of a gene into cells that have a mistake in the genetic sequence of that gene, recovering the normal function of the protein in the cell. The eye is an ideal organ for testing new therapeutic approaches, including CRISPR. That is because the eye is the most exposed part of our brain and thus is easily accessible.

The second reason is that retinal tissue in the eye is shielded from the bodys defense mechanism, which would otherwise consider the injected material used in gene therapy as foreign and mount a defensive attack response. Such a response would destroy the benefits associated with the treatment.

In recent years, breakthrough gene therapy studies paved the way to the first-ever Food and Drug Administration-approved gene therapy drug, Luxturna TM, for a devastating childhood blindness disease, Leber congenital amaurosis Type 2. (Luxturna was developed by Spark Therapeutics and licensed to Novartis NVS, +0.26% NOVN, -0.93%. Spark Therapeutics has since been acquired by Roche ROG, -0.55% RHHBY, -0.45% .)

This form of Leber congenital amaurosis is caused by mutations in a gene that codes for a protein called RPE65. The protein participates in chemical reactions that are needed to detect light. The mutations lessen or eliminate the function of RPE65, which leads to our inability to detect light blindness.

The treatment method developed simultaneously by groups at University of Pennsylvania and at University College London and Moorefields Eye Hospital involved inserting a healthy copy of the mutated gene directly into the space between the retina and the retinal pigmented epithelium, the tissue located behind the retina where the chemical reactions takes place. This gene helped the retinal pigmented epithelium cell produce the missing protein that is dysfunctional in patients.

Although the treated eyes showed vision improvement, as measured by the patients ability to navigate an obstacle course at differing light levels, it is not a permanent fix. This is due to the lack of technologies that can fix the mutated genetic code in the DNA of the cells of the patient.

Lately, scientists have been developing a powerful new tool that is shifting biology and genetic engineering into the next phase. This breakthrough gene-editing technology, which is called CRISPR, enables researchers to directly edit the genetic code of cells in the eye and correct the mutation causing the disease.

Children suffering from the disease Leber congenital amaurosis Type 10 endure progressive vision loss beginning as early as one year old. This specific form of Leber congenital amaurosis is caused by a change to the DNA that affects the ability of the gene called CEP290 to make the complete protein. The loss of the CEP290 protein affects the survival and function of our light-sensing cells, called photoreceptors.

One treatment strategy is to deliver the full form of the CEP290 gene using a virus as the delivery vehicle. But the CEP290 gene is too big to be cargo for viruses. So another approach was needed. One strategy was to fix the mutation by using CRISPR.

The scientists at Editas Medicine first showed safety and proof of the concept of the CRISPR strategy in cells extracted from patient skin biopsy and in nonhuman primate animals.

These studies led to the formulation of the first-ever in human CRISPR gene therapeutic clinical trial. This Phase 1 and Phase 2 trial will eventually assess the safety and efficacy of the CRISPR therapy in 18 Leber congenital amaurosis Type 10 patients. The patients receive a dose of the therapy while under anesthesia when the retina surgeon uses a scope, needle and syringe to inject the CRISPR enzyme and nucleic acids into the back of the eye near the photoreceptors.

To make sure that the experiment is working and safe for the patients, the clinical trial has recruited people with late-stage disease and no hope of recovering their vision. The doctors are also injecting the CRISPR editing tools into only one eye.

An ongoing project in my laboratory focuses on designing a gene therapy approach for the same gene CEP290. Contrary to the CRISPR approach, which can target only a specific mutation at one time, my team is developing an approach that would work for all CEP290 mutations in Leber congenital amaurosis Type 10.

This approach involves using shorter yet functional forms of the CEP290 protein that can be delivered to the photoreceptors using the viruses approved for clinical use.

Gene therapy that involves CRISPR promises a permanent fix and a significantly reduced recovery period. A downside of the CRISPR approach is the possibility of an off-target effect in which another region of the cells DNA is edited, which could cause undesirable side effects, such as cancer. However, new and improved strategies have made such likelihood very low.

Although the CRISPR study is for a specific mutation in CEP290, I believe the use of CRISPR technology in the body to be exciting and a giant leap. I know this treatment is in an early phase, but it shows clear promise. In my mind, as well as the minds of many other scientists, CRISPR-mediated therapeutic innovation absolutely holds immense promise.

In another study just reported in the journal Science, German and Swiss scientists have developed a revolutionary technology, which enables mice and human retinas to detect infrared radiation. This ability could be useful for patients suffering from loss of photoreceptors and sight.

The researchers demonstrated this approach, inspired by the ability of snakes and bats to see heat, by endowing mice and postmortem human retinas with a protein that becomes active in response to heat. Infrared light is light emitted by warm objects that is beyond the visible spectrum.

The heat warms a specially engineered gold particle that the researchers introduced into the retina. This particle binds to the protein and helps it convert the heat signal into electrical signals that are then sent to the brain.

In the future, more research is needed to tweak the ability of the infrared sensitive proteins to different wave lengths of light that will also enhance the remaining vision.

This approach is still being tested in animals and in retinal tissue in the lab. But all approaches suggest that it might be possible to either restore, enhance or provide patients with forms of vision used by other species.

Hemant Khanna is an associate professor of ophthalmology at the University of Massachusetts Medical School. This was first published on The Conversation Gene therapy and CRISPR strategies for curing blindness (Yes, you read that right)

More:
How gene therapy and CRISPR are helping to cure blindness - MarketWatch

- Updated data will be presented in an oral presentation on the efficacy and safety of AAV gene therapy FL180a in the B-AMAZE study in severe haemophilia B patients -

- Additional data will be presented in 5 poster presentations -

LONDON, June 29, 2020 (GLOBE NEWSWIRE) -- Freeline, a biotechnology company focused on developing curative gene therapies for chronic systemic diseases, today announces that Professor Pratima Chowdary of the Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital UK and UCL Cancer Institute, and Chief investigator for the study, will present data in a late-breaking abstract session at the International Society on Thrombosis and Haemostasis (ISTH) 2020 Congress on behalf of co-authors, which will be held virtually 12-14 July 2020.In addition, Freeline has five posters at the conference and will be presenting data on the Factor IX Padua variant and health economics from its Adeno-Associated Virus (AAV)-based haemophilia gene therapy platform.

We are pleased to have an abstract on our novel investigational gene therapy treatment for haemophilia B accepted as a late-breaking abstract at the upcoming ISTH conference, said Theresa Heggie, CEO. Results we have presented previously from our lead program suggest that FLT180a has the potential to create sustained FIX activity levels in the normal range in patients with severe haemophilia B and has the potential to address a significant unmet medical need and advance the standard of care.

Late breaking abstract:

Additional five abstracts/poster presentations:

Founded in 1969, the ISTH is the leading worldwide not-for-profit organization dedicated to advancing the understanding, prevention, diagnosis and treatment of thrombotic and bleeding disorders. ISTH is an international professional membership organisation with more than 5,000 clinicians, researchers and educators working together to improve the lives of patients in more than 100 countries around the world.

Further informationJW CommunicationsJulia Wilson+44 (0) 7818 430877juliawilsonuk@gmail.com

About FreelineFreeline is a clinical-stage biotechnology company focused on AAV-based gene therapy targeting the liver. Its vision is to create better lives for people suffering from chronic, systemic diseases using the potential of gene therapy as a one-time treatment to provide a potential functional cure. Freeline is headquartered in the UK and has operations in Germany and the US.

About HaemophiliaHaemophilia is a genetic bleeding disorder where a protein made by the body to help make blood clot is either partly or completely missing. This protein is called a clotting factor. In Haemophilia A, there is a deficiency of the clotting factor VIII (eight) protein and in Haemophilia B, there is a deficiency of the clotting factor IX (nine) protein. Haemophilia mainly affects boys and men; however, women can be carriers of the affected gene and may experience symptoms. Haemophilia A is the most common type of Haemophilia affecting about one in every 5,000 males, while Haemophilia B affects about one in every 30,000 males. Haemophilia is classified as mild, moderate or severe, depending on the level of clotting factor VIII or IX in the blood and is diagnosed through blood tests.

About FLT180aThe Freeline Haemophilia B programme, FLT180a, uses a synthetic AAVS3 capsid and a gain of function variant of human factor IX (FIX). The therapy is currently being studied in a Phase 1/2 trial, B-AMAZE, with the goal of normalising FIX activity in patients with moderate and severe Haemophilia.

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Freeline announces acceptance of Late Breaking Abstract at the International Society on Thrombosis and Haemostasis (ISTH) 2020 Meeting - GlobeNewswire

North America is the largest regional market for gene therapies

This press release was orginally distributed by SBWire

Northrook, IL -- (SBWIRE) -- 06/29/2020 -- [135 Pages Report] The Global Gene Therapy Market is estimated to grow from USD 3.8 billion in 2019 to USD 13.0 billion by 2024, at a CAGR of 27.8%

The growth of this market is majorly driven by the high incidence of cancer and other target diseases, availability of reimbursements, and the increasing funding for gene therapy research. However, the high cost of gene therapies is expected to hamper market growth to a certain extent during the forecast period.

Recent Developments:

- In May 2019, Novartis AG (Switzerland) received FDA approval for ZOLGENSMA.

- In June 2019, Biogen (US) acquired Nightstar Therapeutics (UK) to enhance its presence in the market.

- In June 2018, Amgen (US) collaborated with the University of Texas MD Anderson Cancer Center (US) to develop a variety of Amgen's early-stage oncology therapies.

Browse 127 Market Data Tables and 25 Figures spread through 135 Pages and in-depth TOC - Request Research Sample Pages: https://www.marketsandmarkets.com/requestsampleNew.asp?id=122857962

Market Segmentation in Detailed:

The non-viral vectors segment accounted for the largest share of the gene therapy market, by vector, in 2018

The gene therapy market, by vector, has been segmented into viral and non-viral vectors. Non-viral vectors accounted for the largest share of the market in 2018. This is mainly attributed to the high market penetration of oligonucleotide-based non-viral vector gene therapies.

The demand for gene therapies for the treatment of cancer is expected to grow at a high rate

Based on indication, the gene therapy market is segmented into neurological diseases, cancer, hepatological diseases, Duchenne muscular dystrophy, and other indications. The neurological diseases segment accounted for the largest share of the market in 2018. However, the cancer segment is estimated to grow at the highest CAGR during the forecast period owing to the increasing incidence of cancer and the rising demand for CAR T-cell therapies.

Download PDF Brochure: https://www.marketsandmarkets.com/pdfdownloadNew.asp?id=122857962

Geographical Regions Mapped in Report:

The global gene therapy market is segmented into North America, Europe, the Asia Pacific, and the Rest of the World. In 2018, North America accounted for the largest share of the market, followed by Europe. Moreover, the North American market is estimated to register the highest growth rate during the forecast period. Factors such as the rising prevalence of chronic diseases, high healthcare expenditure, presence of advanced healthcare infrastructure, favorable reimbursement scenario, and the presence of major market players in the region are driving market growth in North America.

Maior Key Players Mapped in Research Report:

The prominent players operating in the gene therapy market include Biogen (US), Sarepta Therapeutics, Inc. (US), Gilead Sciences, Inc. (US), Novartis AG (Switzerland), Amgen, Inc. (US), Spark Therapeutics, Inc. (US), MolMed S.p.A. (Itlay), Orchard Therapeutics plc. (UK), Sibiono GeneTech Co. Ltd. (China), Alnylam Pharmaceuticals, Inc. (US), Human Stem Cells Institute (Russia), AnGes, Inc. (Japan), Dynavax Technologies (US), Jazz Pharmaceuticals, Inc. (Ireland), and Akcea Therapeutics (US).

For more information on this press release visit: http://www.sbwire.com/press-releases/release-1295441.htm

The rest is here:
Gene Therapy Market Worth $13.0 Billion : Anticipated to Witness High Growth in the Near Future - Press Release - Digital Journal

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced the appointment of Kim Raineri, as chief manufacturing and technology officer. He brings deep global experience in the cell and gene therapy industry, with a distinguished track record of innovation and implementation of Good Manufacturing Practices (GMP).

AVROBIOs founding chief manufacturing and technology officer, Kim Warren, Ph.D., will be retiring at the end of July. Among many other accomplishments, she led the development of plato, the companys proprietary gene therapy platform designed to bring gene therapy to patients worldwide through an efficient, automated, closed manufacturing system developed to be rapidly deployed to contracted manufacturing sites.

We have made it a priority to build leadership in gene therapy manufacturing by integrating and optimizing pioneering technologies as part of our plato platform. As we continue to implement our global clinical trials and prepare for eventual commercial scale, we are delighted to bring Kim Raineri on board. His expertise in global GMP for cell and gene therapy will be highly valuable in keeping AVROBIO at the forefront of this industry, said Geoff MacKay, AVROBIOs president and CEO. I also would like to thank Kim Warren for her integral contributions to AVROBIO these past five years. As one of my co-founders, Kim has played a key role in building the company and establishing plato as an unrivaled lentiviral gene therapy platform.

I am thrilled to join AVROBIO, a leader in lentiviral gene therapy and a true pioneer in driving manufacturing advances that address the gene therapy fields need for faster, more scalable and more automated production, said Raineri. The AVROBIO team has created a state-of-the-art gene therapy platform and is clearly committed to continuous innovation on behalf of the patient communities they strive to serve. I am excited to contribute to that work.

Raineri has broad, global experience in GMP operations, including in the cell and gene therapy, biologics and medical device spaces. Prior to joining AVROBIO, he served as the vice president of operations for Nikon CeLL innovation Co., Ltd, a Japanese contract development and manufacturing organization. During his tenure, he established the company as the preferred provider of custom process development and manufacturing services for cell and gene therapy products in the Japanese market. Previously, Raineri held management positions at Lonza, serving as the business director for the cell therapy contract manufacturing operations in Singapore for five years, and prior to that as director of operations at Lonzas Maryland facilities. Raineri was also previously the senior manager of the Tissue Processing Lab at CryoLife Inc. He holds a B.S. from the University of Miami and an MBA from Kennesaw State University.

About AVROBIO

Our vision is to bring personalized gene therapy to the world. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a preclinical program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking Statement

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding the expected benefits from the appointment of Mr. Raineri to the position of chief manufacturing and technology officer, the expected benefits, timing and results of our implementation of the plato platform in our clinical trials and gene therapy programs, as well as future innovations to our plato platform and other gene therapy manufacturing technologies.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned preclinical or clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

Continued here:
AVROBIO Announces the Appointment of Kim Raineri as Chief Manufacturing and Technology Officer - Business Wire

5pm on June 26, 2020 - Kawasaki/Japan: The Innovation Center of NanoMedicine (iCONM), the National Institute for Quantum Science and Technology (QST), and the University of Tokyo jointly announced that a reagent for the selective and safe coating of the liver sinusoidal walls to control the clearance of gene therapy drugs was successfully developed. The contents of this research will be published in Science Advances by the American Association for the Advancement of Science (AAAS) at 2:00 pm on June 26, east coast of the United States (Japan standard time: 3:00 am on 27th): A. Dirisala, S. Uchida, K. Toh, J. Li, S. Osawa, T. A. Tockary, X. Liu, S. Abbasi, K. Hayashi, Y. Mochida, S. Fukushima, H. Kinoh, K. Osada, Kazunori Kataoka, "Transient stealth coating of liver sinusoidal wall by anchoring two-armed PEG for retargeting nanomedicines".

Recently, gene therapies have been successively approved in Europe, US, and Japan, and are expected to provide novel therapeutic options for cancer, chronic diseases, acquired and inherited genetic disorders. Whilst this is promising, in reality, when gene therapy drugs are systemically administered to living organisms, they are rapidly eliminated and metabolized in the liver, thus impeding the delivery of a sufficient amount to the target organs and raising the toxicity concerns. This elimination by the liver is caused by the adsorption of the gene therapy drugs to the vascular wall of the liver sinusoid, which is an intrahepatic capillary. To overcome this issue, we conceived to selectively coat the liver sinusoidal wall using polyethylene glycol (PEG). However, a long-term coating may impair the normal physiological functions of the liver, and therefore the coating should be transient. In addition, coating needs to be selective for liver sinusoids, as coating the blood vessels throughout the body would not only cause adverse effects but also decrease the delivery amount of gene therapy drugs to target organs. Towards this end, we have developed a coating agent with two-armed PEG conjugated to positively charged oligolysine, which demonstrated the selective coating on the liver sinusoidal wall, the first-of-its-kind strategy in the world. Interestingly, the coating with two-armed PEG was excreted into bile within 6 hours after binding to sinusoidal walls, while the coating with single chain of linear PEG bound to oligolysine persisted in the walls for a long time. In this way, the precise molecular design was necessary to achieve a transient coating.

This coating was subsequently applied to boost the delivery efficacy of gene therapy drugs. Adeno-associated virus (AAV) is widely used for viral gene therapy drugs, and its serotype 8 (AAV8) targets myocardium and skeletal muscles. When AAV8 was administered after prior coating of two-armed PEG to the liver sinusoidal wall, the transfer of AAV8 to the liver was suppressed, and as a result, the gene transfer efficiency into the myocardium and skeletal muscles was improved by 2 to 4 times. This approach is promising for the treatment of muscular dystrophy. In addition, we expanded the use of our strategy to virus-free gene delivery systems, which allows more economically attractive and safe gene therapy. We have been working on non-viral gene therapy for malignant tumors using plasmid DNA-equipped smart nanomachine for over 10 years. When the coating agent was used for this system, the adsorption of nanomachines to the sinusoidal wall was suppressed, resulting in an approximately 10-fold improvement in DNA transfer efficiency to colon cancer. As described above, we have succeeded in boosting the activity of gene therapy drugs while ensuring safety by using the coating agent developed this time.

The above findings are summarized as follows:

- The coating agent with two-armed PEG selectively coated the liver sinusoid wall for several hours and was then excreted in the bile.

- The coating agent with single chain of linear PEG is not excreted in bile and coated the liver sinusoidal wall for more than 9 hours, which raises a safety concern.

- The coating agent with two-armed PEG had selectivity for the liver sinusoid wall, without coating the blood vessels in the connective tissues.

- The coating agent improved the gene transfer efficacy to the myocardium and skeletal muscles using the AAV vector by 2 to 4 times, and the gene transfer efficiency to colorectal cancer using DNA-loaded smart nanomachines by 10 times.

- As a result, our approach is expected to allow for improving the effect of gene therapy drugs and reducing their dose needed to obtain therapeutic outcome, which will lead to the reduction of medical cost and adverse event opportunities.

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Development of safe liver sinusoid coating agents to increase the efficacy of gene therapy - Science Codex

Fior Markets has announced a new report titledGlobal Gene Therapy Marketbegins with the market definition and the market scope. The report provides a market overview, regional market opportunity, sales, and revenue by region, manufacturing cost analysis, industrial chain, market effect factors analysis. The report has mentioned the target audience of the globalGene Therapymarket. The research explains what type of users or what kinds of consumers deal are there. It reports studies of various key segments based on the product, types, applications, end-to-end industries. The document covers the market size forecast (2020-2025), and analysis of industry drivers, challenges, and opportunities.

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

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The industry seems to be fairly competitive. To understand any market with ease the market is segregated into segments, such as its product type, application, technology, end-use industry. By dividing the market into small components helps in understanding the market with more clarity. Data is represented with the help of tables and figures that include a graphical representation of the numbers in the form of histograms, bar graphs, pie charts, etc. Another major segment of the globalGene Therapymarket is its geographical presence on the global platform.

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A Competitive Perspective:

Competition is a major subject in any market research analysis. The competitive analysis provided in the report will help players to easily study key strategies adopted by leading players of the globalGene Therapymarket. They will also be able to plan counterstrategies to gain a competitive advantage in the market. Major as well as emerging players of the market are closely studied taking into consideration their market share, production, revenue, sales growth, gross margin, product portfolio, and other significant factors. Information about company profiles, product offerings, and key financials of important players operating at the market as well as revenue, gross margins, product sales, price patterns has been further given in the report.

This report focused and concentrates on these companies including:Spark Therapeutics LLC, Bluebird Bio, UniQure N.V., Juno Therapeutics, GlaxoSmithKline, Chiesi Farmaceutici S.p.A., Bristol Myers Squibb, Celgene Corporation, Human Stem Cell Institute, Voyager Therapeutics, Shire Plc, Sangamo Biosciences, Dimension Therapeutics and others.

This report focuses on the globalGene Therapymarket status, future forecast, growth opportunity, key market, and key players in major regions includingNorth America, Europe, Asia Pacific, South America, and the Middle East and Africa.

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Moreover, to summarize, the overall globalGene Therapymarket report investigates the contemporary market to forecast the growth prospects, challenges, opportunities, risks, threats, and trends in the market. Marketing channel development, marketing approach, and market positioning are further discussed in the report. The all-inclusive market feasibility is examined to figure out the profit-making trends. Constraints and openings with extensive impact analysis are additionally revealed in the report.

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Global Gene Therapy Market 2020 Trends, lucrative Opportunities, Analysis to 2025 - The Collegian

Germantown, MD, US, June 29, 2020 Orgenesis Inc. (NASDAQ: ORGS)(Orgenesis or the Company), a pioneering, global biotech company committed to accelerating commercialization and transforming the delivery of cell and gene therapies (CGTs), today announced that it has been added to the Russell 3000(R) Index, following the annual Russell indexes reconstitution, effective after the market open on June 29, according to a final list of additions posted on June 26.

Annual Russell indexes reconstitution captures the 4,000 largest US stocks, ranking them by total market capitalization. Membership in the US all-cap Russell 3000(R) Index, which remains in place for one year, means automatic inclusion in the large-cap Russell 1000(R) Index or small-cap Russell 2000(R) Index as well as the appropriate growth and value style indexes.

Vered Caplan, CEO of Orgenesis, commented, Were pleased to be included in the Russell 3000 Index. We believe this inclusion will help improve the Companys exposure within the global investment community. The addition to the Russell 3000(R) Index reflects our commitment to building shareholder value through growth of our global CGT Biotech Platform and building our pipeline of POCare Therapeutics and Technologies, with an ultimate goal of providing life-changing treatments at reduced costs within the point-of-care setting.

Russell indexes are widely used by investment managers and institutional investors for index funds and as benchmarks for active investment strategies. Approximately 9 trillion dollars in assets are benchmarked against Russells US indexes. Russell indexes are part of FTSE Russell, a leading global index provider. For more information on the Russell 3000(R) Index and the Russell indexes reconstitution, go to the Russell Reconstitution section on theFTSE Russell website.

About Orgenesis

Orgenesis is a pioneering global biotech company which is unlocking the full potential of personalized therapies and closed processing systems through its Cell & Gene Therapy Biotech Platform, with the ultimate aim of providing life changing treatments at the Point of Care to large numbers of patients at low cost. The Platform consists of: (a)POCare Therapeutics, a pipeline of licensed cell and gene therapies (CGTs), and proprietary scientific knowhow; (b)POCare Technologies, a suite of proprietary and in-licensed technologies which are engineered to create customized processing systems for affordable point of care therapies; and (c)POCare Network, a collaborative, international ecosystem of leading research institutes and hospitals committed to clinical development and supply of CGTs at the point of care. By combining science, technologies and a collaborative network, Orgenesis is able to identify the most promising new therapies and provide a pathway for them to reach patients more quickly, more efficiently and at scale, thereby unlocking the power of cell and gene therapy for all. Additional information is available at:www.orgenesis.com.

About FTSE Russell

FTSE Russell is a leading global index provider creating and managing a wide range of indexes, data and analytic solutions to meet client needs across asset classes, style and strategies. Covering 98 percent of the investable market, FTSE Russell indexes offer a true picture of global markets, combined with the specialist knowledge gained from developing local benchmarks around the world.

FTSE Russell index expertise and products are used extensively by institutional and retail investors globally. Approximately 16 trillion dollars is currently benchmarked to FTSE Russell indexes. For over 30 years, leading asset owners, asset managers, ETF providers and investment banks have chosen FTSE Russell indexes to benchmark their investment performance and create investment funds, ETFs, structured products and index-based derivatives. FTSE Russell indexes also provide clients with tools for asset allocation, investment strategy analysis and risk management.

A core set of universal principles guides FTSE Russell index design and management: a transparent rules-based methodology is informed by independent committees of leading market participants. FTSE Russell is focused on index innovation and customer partnership applying the highest industry standards and embracing the IOSCO Principles. FTSE Russell is wholly owned by London Stock Exchange Group.

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Orgenesis Added to Russell 3000(R) Index - PharmiWeb.com

The Global Cancer Gene Therapy Market Research Report provides customers with a complete analytical study that provides all the details of key players such as company profile, product portfolio, capacity, price, cost, and revenue during the forecast period from 2020 to 2027. The report provides a full assessment. Cancer Gene Therapy market with future trends, current growth factors, meticulous opinions, facts, historical data and statistically supported and industry-validated market data.

This Cancer Gene Therapy market research provides a clear explanation of how this market will impress growth during the mentioned period. This study report scanned specific data for specific characteristics such as Type, Size, Application and End User. There are basic segments included in the segmentation analysis that are the result of SWOT analysis and PESTEL analysis.

To Learn More About This Report, Request a Sample Copy:https://www.worldwidemarketreports.com/sample/106386* The sample copy includes: Report Summary, Table of Contents, Segmentation, Competitive Landscape, Report Structure, Methodology.

Adaptimmune, GlaxoSmithKline, Bluebird bio, Merck, Celgene, Shanghai Sunway Biotech, BioCancell, Shenzhen SiBiono GeneTech, SynerGene Therapeutics, OncoGenex Pharmaceuticals, Genelux Corporation, Cell Genesys, Advantagene, GenVec are some of the major organizations dominating the global market.(*Note: Other Players Can be Added per Request)

Key players in the Cancer Gene Therapy market were identified through a second survey, and their market share was determined through a primary and second survey. All measurement sharing, splitting, and analysis were solved using a secondary source and a validated primary source. The Cancer Gene Therapy market report starts with a basic overview of the Industry Life Cycle, Definitions, Classifications, Applications, and Industry Chain Structure, and when used together, how key players can meet market coverage, offered characteristics, and customer needs It helps to understand.

The report also makes some important suggestions for new Cancer Gene Therapy market projects before evaluating their feasibility. Overall, this report covers Cancer Gene Therapy market Sales, Price, Sales, Gross Profit, Historical Growth,and Future Prospects. It provides facts related to the widespread merger, acquisition, partnership, and joint venture activities on the market.

This report includes market size estimates of value (million US $) and trading volume (K MT). The top-down and bottom-up approaches are used to estimate and validate the market size of the Cancer Gene Therapy market, estimating the size of various other subordinate markets in the overall market. All ratio sharing, splitting, and analysis were determined using the secondary source and the identified primary source.

What Cancer Gene Therapy Market report offers:

Remarkable Attributes of Cancer Gene Therapy Market Report:

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Worldwide Market Reports is your one-stop repository of detailed and in-depth market research reports compiled by an extensive list of publishers from across the globe. We offer reports across virtually all domains and an exhaustive list of sub-domains under the sun. The in-depth market analysis by some of the most vastly experienced analysts provide our diverse range of clients from across all industries with vital decision making insights to plan and align their market strategies in line with current market trends.

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Cancer Gene Therapy Market Report by Manufacturers, Regions, Type and Application Forecast 2020 2027: Adaptimmune, GlaxoSmithKline, Bluebird bio -...

A report on Gene Therapy for Age-related Macular Degeneration market compiled by Brand Essence Market Research provides a succinct analysis regarding the values and trends existing in the current business scenario. The study also offers a brief summary of market valuation, market size, regional outlook and profit estimations of the industry. Furthermore, the report examines the competitive sphere and growth strategies of leading players in the Gene Therapy for Age-related Macular Degeneration market.

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

Download Premium Sample of the Report:https://industrystatsreport.com/Request/Sample?ResearchPostId=1&RequestType=Sample

Key playersof the Gene Therapy for Age-related Macular Degeneration market are RetroSense Therapeutics, REGENXBIO, AGTC

Gene Therapy for Age-related Macular Degeneration Market Segmentation:

Product Type: Subretinal,Intravitreal,Unspecified

Application: Monotherapy,Combination Therapy

Region Coverage (Regional Production, Demand & Forecast by Countries etc.):North America (U.S., Canada, Mexico)Europe (Germany, U.K., France, Italy, Russia, Spain etc.)Asia-Pacific (China, India, Japan, Southeast Asia etc.)South America (Brazil, Argentina etc.)Middle East & Africa (Saudi Arabia, South Africa etc.)

Table of Contents

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

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

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

Read More:https://industrystatsreport.com/ICT-and-Media/Global-Low-Density-SLC-NAND-Flash-Memory-Market/Summary

About us: Brandessence Market Research and Consulting Pvt. ltd.

Brandessence market research publishes market research reports & business insights produced by highly qualified and experienced industry analysts. Our research reports are available in a wide range of industry verticals including aviation, food & beverage, healthcare, ICT, Construction, Chemicals and lot more. Brand Essence Market Research report will be best fit for senior executives, business development managers, marketing managers, consultants, CEOs, CIOs, COOs, and Directors, governments, agencies, organizations and Ph.D. Students. We have a delivery center in Pune, India and our sales office is in London.

Contact us at: +44-2038074155 or mail us at[emailprotected]

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What is Trending in Gene Therapy for Age-related Macular Degeneration Market? What are the Strategies to Boost Business in Near Years? - Owned

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

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

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

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

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

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

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

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

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

The key players covered in this study RetroSense Therapeutics REGENXBIO AGTC

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

Market segment by Application, split into Monotherapy Combination Therapy

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

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

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

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

Table of Contents

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

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

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

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

About us: Brandessence Market Research and Consulting Pvt. ltd.

Brandessence market research publishes market research reports & business insights produced by highly qualified and experienced industry analysts. Our research reports are available in a wide range of industry verticals including aviation, food & beverage, healthcare, ICT, Construction, Chemicals and lot more. Brand Essence Market Research report will be best fit for senior executives, business development managers, marketing managers, consultants, CEOs, CIOs, COOs, and Directors, governments, agencies, organizations and Ph.D. Students. We have a delivery center in Pune, India and our sales office is in London.

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2025 Growth: Gene Therapy for Age-related Macular Degeneration Market Insights and Forecast 2020 to 2025 - 3rd Watch News

The report examines the Gene Therapy market with respect to the industry trends, growth rate, prospects, drivers, restraints, threats, and lucrative opportunities, by means of distinguishing the high-growth segments of the market through the various stakeholders. The statistical surveying study also elucidates the different strategies, collaborations, mergers and acquisitions, product launches, innovations, and activities in the R&D sector in the Gene Therapy Market.

The Gene Therapy Market research report covers the present scenario and the growth prospects of the global Gene Therapy industry for 2020-2027. The report enlists several important factors, starting from the basics to advanced market intelligence which plays a crucial part in strategizing.

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

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Key Vendors of Gene Therapy Market:GlaxoSmithKline plc, Bluebird Bio, Inc., Adaptimmune Therapeutics plc, Celgene Corporation, Shanghai Sunway Biotech Co. Ltd., Merck KGaA, Transgene SA, and OncoGenex Pharmaceuticals, Inc.

An exhaustive study has been carried out on the key players operating in the Gene Therapy Market. The report covers the revenue share, cost, product offering, recent developments, gross profit, business overview, and mergers & acquisitions, which helps the customers to understand the key players in a more profound manner.

The Gene Therapy Market report analyses the market potential for each geographical region based on the growth rate, macroeconomic parameters, consumer buying patterns, and market demand and supply scenarios.

Regions of Gene Therapy market: Americas APAC EMEA

Key questions answered in Gene Therapy market report: What will the market size be in 2027 and what will the growth rate be? What are the key market trends? What is driving this market? What are the challenges to market growth? Who are the key vendors in this market space? What are the market opportunities and threats faced by the key vendors? What are the strengths and weaknesses of the key vendors?

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Why this is a useful Report to you.-This report provides pin-point analysis for changing competitive dynamics-It provides a forward-looking perspective on different factors driving or restraining market growth-It provides a six-year forecast assessed on the basis of how the market is predicted to grow-It helps in understanding the key product segments and their future-It provides pin point analysis of changing competition dynamics and keeps you ahead of competitors-It helps in making informed business decisions by having complete insights of market and by making in-depth analysis of market segments.

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Gene Therapy Market 2020 Global Trends, Sales Revenue, Development Strategy, Key Vendors, Competitive Landscape, Opportunity Assessment and Potential...

The same day Oxford Biomedica signed a 5-year deal to make AstraZenecas Covid-19 vaccine, the UK gene therapy company announced they received word Sanofi was sending a couple of decade-old experimental drugs back to them.

In 2009, Sanofi and Biomedica slashed a co-development deal on TroVax, a cancer vaccine that had been one of the centerpieces of the then 14-year-old company. But the pair immediately replaced it with a deal, 8 years before the approval of Spark Therapeutics Luxturna, to develop gene therapies for two eye-related disorders.

Now, with little to show for it but a pair of very early studies, Sanofi is kicking the therapies back to Biomedica. The French drugmaker had already announced last year, amid an internal shakeup, that they were not interested in developing the drugs further, but what would happen to the therapies hung in the air.

In some ways, it still does. Biomedica wouldnt commit to progressing or retiring them. It wasnt even clear when Biomedica would regain the rights.

The timing of return of these programs and operational details are yet to be determined, the biotech said in a statement. However, when the rights to the two programs are returned, the group will undertake its own internal evaluation to determine the potential future for these programs and decide whether to commit further resources to them.

Biomedicas $100 million partnership with Sanofis rare blood disorder subsidiary Bioverativremains intact.

The two gene therapies are SAR422459, for treating a progressive macular disease called Stargardt disease, and SAR421869, for treating Ushers Syndrome, a disorder that can cause both vision and hearing loss. The Stargardt therapy began a Phase I/II study in France in 2011, but the study was ended prematurely last August after Sanofi reprioritized its pipeline. Although the study was designed to measure biological markers of efficacy, Sanofi included no such data when it posted results on clinicaltrials.gov earlier this year.

The therapy for Ushers Syndrome entered its Phase I/II study in 2012. That study was suspended in 2017 while Sanofi reviewed its pipeline and then also terminated last year.

If Biomedica considers the therapies to still have potential, its likely they will try to out-license them again. The company is built around partnerships, licensing out a few in-house candidates and helping manufacture Novartiss CAR-T therapy Kymriah and, as of yesterday, AstraZenecas Covid-19 vaccine. They areexplicitly not a commercial-stage biotech.All of the preclinical candidates on its website are listed as to be spun out, or out-licensed, and they run no clinical trials.

So the best question may be not whether Biomedica sees potential, but if anyone else does.

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Sanofi sends a pair of gene therapies back to Oxford Biomedica. Are they salvageable? - Endpoints News

The Personalized Gene Therapy Treatment Market Research Report 2020 published by Prophecy Market Insights is an all-inclusive business research study on the current state of the industry which analyzes innovative strategies for business growth and describes significant factors such as top developers/manufacturers, production value, key regions, and growth rate. Impact of Covid-19 pandemic on the market will be completely analyzed in this report and it will also quantify the impact of this pandemic on the market.

The research study encompasses an evaluation of the market, including growth rate, current scenario, and volume inflation prospects, based on DROT and Porters Five Forces analyses. The market study pitches light on the various factors that are projected to impact the overall market dynamics of the Personalized Gene Therapy Treatment market over the forecast period (2019-2029).

Regional Overview:

The survey report includes a vast investigation of the geographical scene of the Personalized Gene Therapy Treatment market, which is manifestly arranged into the localities. The report provides an analysis of regional market players operating in the specific market and outcomes related to the target market for more than 20 countries.

Australia, New Zealand, Rest of Asia-Pacific

The facts and data are represented in the Personalized Gene Therapy Treatment report using graphs, pie charts, tables, figures and graphical representations helping analyze worldwide key trends & statistics on the state of the industry and is a valuable source of guidance and direction for companies and individuals interested in the market.

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The research report also focuses on global major leading industry players of Personalized Gene Therapy Treatment market report providing information such as company profiles, product picture and specification, R&D developments, distribution & production capacity, distribution channels, price, cost, revenue and contact information. The research report examines, legal policies, and competitive analysis between the leading and emerging and upcoming market trends.

Personalized Gene Therapy TreatmentMarket Key Companies:

Amgen, Inc., Chengdu Shi Endor Biological Engineering Technology Co., Ltd., SynerGene Therapeutics, Inc., Cold Genesys, Inc., Bellicum Pharmaceuticals, Inc., Takara Bio, Inc.,Ziopharm Oncology, Inc., , Sevion Therapeutics, Inc., OncoSec Medical, Inc., and Burzynski Clinic.

The predictions mentioned in the Personalized Gene Therapy Treatment market report have been derived using proven research techniques, assumptions and methodologies. This market report states the overview, historical data along with size, share, growth, demand, and revenue of the global industry.

Segmentation Overview:

The report provides an in-depth analysis of the Personalized Gene Therapy Treatment market segments and highlights the latest trending segment and major innovations in the market. In addition to this, it states the impact of these segments on the growth of the market. Apart from key players analysis provoking business-related decisions that are usually backed by prevalent market conditions, we also do substantial analysis of market based on COVID-19 impact, detailed analysis on economic, health and financial structure.

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2020 Prediction for Personalized Gene Therapy Treatment market: Key Players and Driving Factors Analysis - 3rd Watch News

DUBLIN, June 26, 2020 /PRNewswire/ -- The "Non-Viral Transfection Reagents and Systems Market, 2020-2030" report has been added to ResearchAndMarkets.com's offering.

Over the last few years, the exponential growth in the pipeline of nucleic acid based therapies has led to the escalating interest of pharmaceutical industry in this domain. Presently, more than 2,000 trials evaluating different types of gene therapies are underway. Moreover, according to experts at the US FDA, around 40 new gene therapies are likely to be approved by 2022. In this context, it is worth highlighting that viral vectors are a crucial element in gene therapy development and manufacturing. Although, viral vectors have shown significant success in R&D, their applications are limited due to immunogenicity and toxicity related concerns, high development costs and the limitation on amount of genomic material that they can carry. Excessively high price tags associated with viral-based therapies, such as Zolgensma (USD 2.1 million) and Luxtruna (USD 850,000), have led to several reimbursement challenges, thereby decreasing patient access. Owing to the aforementioned concerns related to viral vectors, therapy developers are evaluating a variety of non-viral methods of gene delivery.

In the present scenario, non-viral transfection systems are not yet widely used in therapy development and clinical studies, primarily due to their relatively low efficiency compared to viruses. The applications of these methods are largely restricted to fundamental research, including protein and gene expression, and cell line development. However, there are a number of companies that have developed proprietary technologies and products to facilitate physical (electroporation, gene gun, microinjection and sonoporation), chemical (transfection reagents) and other non-viral methods of transfection (transposon based systems, piggyBac and magnetofection). We believe that, as the demand for advanced therapy medicinal products, which require genetic engineering, the opportunity for non-viral transfection system developers is also likely to grow.

The Non-Viral Transfection Reagents and Systems Market, 2020-2030' report features an extensive study of the various systems and technologies available for non-viral transfection, in addition to the current market landscape and future potential of product developers.

Amongst other elements, the report features:

In order to account for future uncertainties and to add robustness to the model, three forecast scenarios have been provided, portraying the conservative, base and optimistic tracks of the market's evolution. The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders in this domain.

In addition, the report features detailed transcripts of interviews held with the following individuals:

A selection of companies mentioned includes:

For more information about this report visit https://www.researchandmarkets.com/r/e2ipme

About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Non-Viral Transfection Reagents and Systems Market Analysis 2020-2030, Featuring Key Player Profiles for Altogen Biosystems, MilliporeSigma, OZ...

DetailsCategory: AntibodiesPublished on Sunday, 28 June 2020 14:40Hits: 307

Reblozyl regulates late-stage red blood cell (RBC) maturation to potentially reduce or eliminate the need for regular RBC transfusions

Reblozyl is the first and only erythroid maturation agent to be approved in the European Union, representing a new class of therapy

PRINCETON, NJ & CAMBRIDGE, MA, USA I June 26, 2020 IBristol Myers Squibb (NYSE: BMY) and Acceleron Pharma Inc. (NASDAQ: XLRN) today announced that the European Commission (EC) has approved Reblozyl (luspatercept) for the treatment of:

"Dependence on blood transfusions caused by anemia in hematologic malignancies like MDS can often mean frequent and lengthy hospital visits, which can pose additional health risks and affect patients quality of life, said Uwe Platzbecker, M.D., lead investigator of the MEDALIST study, Head of Clinic and Policlinic for Hematology and Cell Therapy, Leipzig University Hospital. Todays approval of Reblozyl provides healthcare professionals with a new therapy that has been shown to significantly reduce the number of red blood cell transfusions needed by MDS patients and, in some cases, helped them to achieve transfusion independence.

While beta thalassemia remains an orphan disease, the lifelong blood transfusions often needed by patients can have a significant impact on the limited blood supply in their communities, and there are few treatment alternatives, said Maria Domenica Cappellini, M.D., lead investigator of the BELIEVE study, Professor of Medicine, University of Milan, Fondazione IRCCS Ca Granda. The European Commissions approval of Reblozyl provides eligible adult patients with beta thalassemia a new, much needed treatment option for their anemia, and with it, the possibility of becoming less dependent on red blood cell transfusions.

Reblozyl is the first and only erythroid maturation agent approved in the European Union, representing a new class of therapy for eligible patients. This approval is based on data from the pivotal Phase 3 MEDALIST and BELIEVE studies, evaluating the ability of Reblozyl to effectively address anemia associated with MDS and beta thalassemia, respectively.

Across the EU, 25 million blood transfusions occur every year, some of which are needed by patients with anemia due to hematologic diseases like MDS and beta thalassemia, said Diane McDowell, M.D., vice president, Hematology Global Medical Affairs, Bristol Myers Squibb. Reblozyl has the potential to address the ineffective erythropoiesis associated with MDS and beta thalassemia, decrease patients dependence on red blood cell transfusions and impact the underlying consequences of the high burden of anemia for these patients. Alongside our partners at Acceleron, we recognize the continuing need in disease-related anemias and are committed to working collaboratively with European health authorities to make Reblozyl available to these patients as quickly as possible.

About MEDALIST

MEDALIST is a Phase 3, randomized, double-blind, placebo-controlled, multi-center study evaluating the safety and efficacy of Reblozyl plus best supportive care (BSC) versus placebo plus BSC in adults with IPSS-R-defined very low-, low- or intermediate-risk non-del(5q) MDS. All patients were RBC transfusion-dependent and were either refractory or intolerant to prior erythropoiesis stimulating agent (ESA) therapy, or were ESA nave and unlikely to respond due to endogenous serum erythropoietin levels of 200 U/L, and had no prior treatment with disease modifying agents.

The trial showed a statistically significant improvement in RBC transfusion burden with Reblozyl, the studys primary endpoint, with 37.9% of patients treated with Reblozyl achieving independence from RBC transfusions for at least eight weeks during the first 24 weeks of the trial compared to 13.2% of patients on placebo. The trial also met the secondary endpoint of transfusion independence for at least 12 weeks within the first 24 and 48 weeks of the study, which was achieved in a significantly greater proportion of patients receiving Reblozyl versus placebo.

The majority of treatment-emergent adverse events (TEAEs) were Grade 1-2. Grade 3 or 4 TEAEs were reported in 42.5% of patients who received Reblozyl and 44.7% of patients who received placebo. Discontinuation due to an adverse reaction (Grades 1-4) occurred in 4.5% of patients who received Reblozyl. The most common (>10%) all-grade adverse reactions included fatigue, musculoskeletal pain, dizziness, diarrhea, nausea, hypersensitivity reactions, hypertension, headache, upper respiratory tract infection, bronchitis and urinary tract infection.

Results of the MEDALIST trial were first presented during the Plenary Session of the American Society of Hematology (ASH) Annual Meeting in December 2018 (ASH Abstract #001) and were selected for the Best of ASH. The New England Journal of Medicine published the MEDALIST trial results in January 2020.

About MDS

MDS are a group of hematologic malignancies characterized by ineffective production of healthy red blood cells, white blood cells and platelets, which can lead to anemia and frequent or severe infections, and can progress to Acute Myeloid Leukemia (AML). People with MDS who develop anemia often require regular blood transfusions to increase the number of healthy red blood cells in circulation. Frequent transfusions are associated with an increased risk of transfusion reactions, infections and iron overload. There are approximately 50,000 patients with MDS in the EU5 countries (France, Germany, Italy, Spain and the United Kingdom).

About BELIEVE

BELIEVE is a Phase 3, randomized, double-blind, placebo-controlled multi-center study comparing Reblozyl plus BSC versus placebo plus BSC in adults who require regular RBC transfusions (6-20 RBC units per 24 weeks with no transfusion-free period greater than 35 days during that period) due to beta thalassemia.

The trial showed a statistically significant improvement in RBC transfusion burden during weeks 13 to 24 compared to the baseline 12-week interval prior to randomization (21.4% Reblozyl versus 4.5% placebo), meeting the studys primary endpoint. The trial also met the secondary endpoint of transfusion burden reduction of at least 33% (with a reduction of at least two units) during weeks 37 to 48, which was achieved in a significantly greater proportion of patients receiving Reblozyl versus placebo. The trial also met an exploratory endpoint, with 70.5% of patients treated with Reblozyl achieving at least a 33% reduction in RBC transfusion burden of at least two units for any 12 consecutive weeks compared to the 12-week interval prior to treatment, compared to 29.5% of patients on placebo.

The majority of TEAEs were Grade 1-2. Discontinuation due to an adverse reaction (Grades 1-4) occurred in 5.4% of patients who received Reblozyl. The most common adverse reactions (>10%) were headache, bone pain, arthralgia, fatigue, cough, abdominal pain, diarrhea and dizziness.

Results of the BELIEVE trial were first presented at the ASH Annual Meeting in December 2018 and selected for the Best of ASH. The New England Journal of Medicine published the BELIEVE trial results in March 2020.

About Beta Thalassemia

Beta thalassemia is an inherited blood disorder caused by a genetic defect in hemoglobin. The disease is associated with ineffective erythropoiesis, which results in the production of fewer and less healthy RBCs, often leading to severe anemiaa condition that can be debilitating and can lead to other complications for patientsas well as other serious health issues. Treatment options for anemia associated with beta thalassemia are limited, consisting mainly of frequent RBC transfusions that have the potential to contribute to iron overload, which can cause serious complications such as organ damage. Across the United States, Germany, France, Italy, Spain and the United Kingdom, there are approximately 17,000 patients with beta thalassemia.

About Reblozyl

Reblozyl (luspatercept-aamt), a first-in-class erythroid maturation agent, promotes late-stage red blood cell maturation in animal models. Bristol Myers Squibb and Acceleron are jointly developing Reblozyl as part of a global collaboration. Reblozyl is currently approved in the U.S. for the treatment of:

Reblozyl is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia.

Bristol Myers Squibb: Advancing Cancer Research

At Bristol Myers Squibb, patients are at the center of everything we do. The goal of our cancer research is to increase patients quality of life, long-term survival and make cure a possibility. We harness our deep scientific experience, cutting-edge technologies and discovery platforms to discover, develop and deliver novel treatments for patients.

Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational chimeric antigen receptor (CAR) T-cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering potential new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.

About Bristol Myers Squibb

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.

Celgene and Juno Therapeutics are wholly owned subsidiaries of Bristol-Myers Squibb Company. In certain countries outside the U.S., due to local laws, Celgene and Juno Therapeutics are referred to as, Celgene, a Bristol Myers Squibb company and Juno Therapeutics, a Bristol Myers Squibb company.

About Acceleron

Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. Acceleron's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.

Acceleron focuses its commercialization, research, and development efforts in hematologic and pulmonary diseases. In hematology, Acceleron and its global collaboration partner, Bristol Myers Squibb, are co-promoting REBLOZYL (luspatercept-aamt), the first and only approved erythroid maturation agent, in the United States for the treatment of anemia in certain blood disorders. The Companies are also developing luspatercept for the treatment of chronic anemia in patient populations of MDS, beta-thalassemia, and myelofibrosis. In pulmonary, Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension, having recently reported positive topline results of the Phase 2 PULSAR trial.

For more information, please visit http://www.acceleronpharma.com. Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn.

SOURCE: Bristol-Myers Squibb

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European Commission Approves Reblozyl (luspatercept) for the Treatment of Transfusion-Dependent Anemia in Adult Patients with Myelodysplastic...

In the race to produce a coronavirus vaccine, some companies are experimenting with a novel approach using gene therapy. The faster, cheaper method could trigger peoples bodies to produce their own antibodies against the virus more quickly than a traditional vaccine would.

The new techniquestill in the early stages of researchinvolves taking antibodies from the blood of someone who has survived COVID-19 and isolating their genetic material. Doctors would then inject the DNA into the arm or leg of a patient, where it would provide instructions for the muscle to produce more antibodies to fight the virus. The gene therapy technique could provide immunity or lessen the severity of the disease in those already infected.

Doctors could enhance the treatment with a gene gun. The device delivers an electric shock that causes the muscle cells to pop open for an instant, increasing absorption of the injected DNA. The procedure could, within just hours, turn the muscle into an antibody-producing factory, according to SmartPharm, one of the companies working on the treatment.

You can make DNA very readily, its dirt cheap, and you let the muscle make the antibody, Sorrento Therapeutics CEO Henry Ji, who is working with SmartPharm, told MIT Technology Review.

Traditional vaccines depend on the recipients immune system to mount a protective response against the virus. But it takes several weeks for a body to generate an immune response strong enough to provide protection. Some peoples bodies cant do it at all, particularly the elderly and others at high risk for COVID-19. With the new treatment, the muscles could produce protective levels of the antibody within a day or two. The antibody production could last weeks, or even months, regardless of the strength of the patients immune system.

Vaccines usually only work if given before someone contracts the disease. But the gene therapy can neutralize the virus even in an active infection.

Scientists have not yet tested this method of preventing and treating COVID-19 in humans. But David Weiner, director of the vaccine and immunotherapy center at the Wistar Institute in Philadelphia, told MIT Technology Review his center has tested anti-COVID-19 gene injections on animals. Last year, researchers at AbCellera in Vancouver, Canada, developed a gene shot that allowed mice to survive a dose of avian influenza 20 times the lethal amount.

DNA injections might also prove useful as an inexpensive way to treat cancer and arthritis, Weiner said. Even if scientists cannot develop the technique in time to prevent or treat COVID-19, the research could enable a quick solution to the next pandemic.

In the future, we will have gone through the drill, Ji said. Youll just snap your gene in.

Link:
A different kind of shot in the arm against COVID-19 - WORLD News Group