StemCell Therapy

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

- Competitors identified in this market include, among others,

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

I. INTRODUCTION, METHODOLOGY & REPORT SCOPE

II. EXECUTIVE SUMMARY

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

2. FOCUS ON SELECT PLAYERS

3. MARKET TRENDS & DRIVERS

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

III. MARKET ANALYSIS

GEOGRAPHIC MARKET ANALYSIS

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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Global Gene Therapy Industry - PRNewswire

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Written by AZoNanoJul 8 2020

Two researchers from Johns Hopkins Universityan ophthalmologist and an engineerhave successfully used nanoparticles to deliver gene therapy for blinding eye disease. They achieved this by performing experiments in mice and rats.

Image Credit: Johns Hopkins Medicine.

Using an exclusively designed large molecule, the researchers could compact huge bundles of therapeutic DNA to be delivered into the cells of the eye.

Reported in the Science Advances journal on July 3rd, 2020, the study offers evidence of the prospective value of nanoparticle-delivered gene therapy for the treatment of wet age-related macular degeneration.

Macular degeneration is an eye disease in which blood vessel growth is abnormal, causing damage to the light-sensitive tissue at the back of the eye, together with rarer, inherited blinding diseases of the retina.

Several gene therapy techniques rely on viral vectors, which tap the natural ability of a virus to carry genetic material into cells. But viruses tend to create an immune response that prevents repeat dosing, and the one most often used for ocular gene therapy is not capable of carrying large genes.

Some of the most prevalent inherited retinal degenerations are due to mutations in large genes that simply cannot fit into the most commonly used viral vector.

Peter Campochiaro, MD, Eccles Professor of Ophthalmology, Johns Hopkins University School of Medicine

Campochiaro is also a member of the Johns Hopkins Medicine Wilmer Eye Institute.

Campochiaro and Jordan Green, PhD, created a new technique to overcome such drawbacks, which involves using a biodegradable polymer that surrounds and compacts long DNA stretches. This helps create nanoparticles with the potential to enterthe cells. This technology enables the conversion of the cells of the eye into mini factories for a therapeutic protein.

The researchers first tested whether the nanoparticles enter their target cells by loading the nanoparticles with a gene for a fluorescent protein that makes cells glow similar to a glow stick.

Using the glowing molecule, the researchers were able to find the location, duration, and amount of gene expression that can be achieved using the nanoparticles.

Even eight months following treatment, it was found that most of the light-sensitive cells in the eyes of the rats glowed, demonstrating that the nanoparticles effectively loaded the fluorescent gene into the cells.

A similar experiment was also performed using the nanoparticles to shuttle a biologically relevant gene into the eye. A gene for vascular endothelial growth factor (VEGF) was loaded into the nanoparticles, where the gene takes part in the growth of abnormal blood vessels in people suffering from wet macular degeneration.

The eyes of 30 rats were injected with the nanoparticles that carried the VEGF gene, and the effects in the retina were determined one, two, and five months post-injection. One month post-injection, abnormal blood vessels developed in each tested rat under and inside the retina, similar to those seen in patients suffering from wet macular degeneration.

The abnormal blood vessels were found to be more widespread at two and five months post-injection, and there was related scarring under the retina such as those observed in chronic untreated wet macular degeneration.

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

Peter Campochiaro, MD, Eccles Professor of Ophthalmology, School of Medicine, Johns Hopkins University

Lastly, the researchers tested the ability of a nanoparticle to deliver a therapeutic gene for the disease by using mice that had been genetically engineered to develop a kind of wet macular degeneration such as those seen in humans. Nanoparticles were loaded with a gene that synthesizes a protein that neutralizes VEGF.

At present, such proteins that block VEGF proteins are injected by physicians into the eyes of people suffering from macular degeneration. This treatment helps control the overgrowth of abnormal, leaky blood vessels. However, this process must be repeated often and is cumbersome for patients and their caretakers.

Three weeks post-injection of nanoparticles with the gene for the anti-VEGF protein, a 60% decrease in abnormal blood vessels was observed in the mice.

The same effect was observed 35 days later.

These results are extremely promising. We have the ability to reach the cells most significantly affected by degenerative eye disease with nonviral treatments that can allow the eye to create its own sustained therapies.

Jordan Green, PhD, Professor of Biomedical Engineering, School of Medicine, Johns Hopkins University

In the United States, approximately 1.6 million people suffering from macular degeneration are administered injected drugs to the eye every four to six weeks. A gene therapy treatment could offer a means for the tissue of the eye to prevent further deterioration of vision with only a few initial treatments.

Genetic diseases that lead to blindness could be similarly treated by introducing functional versions of genes disabled by inherited mutations.

Jikui Shen, Jayoung Kim, Stephany Tzeng, Kun Ding, Zibran Hafiz, Da Long, and Jiangxia Wang from the Johns Hopkins University School of Medicine are the other researchers involved in this study.

This study was financially supported by the National Eye Institute (01EY031097, R21EY026148, R01EY028996, EY01765), the National Institute of Biomedical Imaging and Bioengineering (R01EB022148) Research to Prevent Blindness (the Dr H. James and Carole Free Catalyst Award and an unrestricted grant), the Louis B. Thalheimer Fund for Translational Research; the Barth Syndrome Foundation, Samsung, Conrad and Lois Aschenbach, Per Bang-Jensen, Andrew and Yvette Marriott, and Jean Lake.

Shen, J., et al. (2020) Suprachoroidal gene transfer with nonviral nanoparticles. Science Advances. doi.org/10.1126/sciadv.aba1606.

Source: https://www.hopkinsmedicine.org/

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Nanoparticles Used to Deliver Gene Therapy for Macular Degeneration - AZoNano

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

Pixabay/Arek Socha

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

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

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

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

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

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

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

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

NEW YORK and CLEVELAND, July 09, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced the appointment of Michael Amoroso as Senior Vice President and Chief Commercial Officer (CCO), effective immediately. Mr. Amoroso brings to Abeona over 20 years of product commercialization experience in the biotechnology and pharmaceutical industries, most recently as Senior Vice President and Head of Worldwide Commercial, Cell Therapy at Kite, a Gilead Company.

Mr. Amoroso will have overall responsibility for building the Companys commercial organization, developing the commercialization strategy for EB-101, its autologous, gene-corrected cell therapy for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) and its lead product candidate, as well as leading pre-commercial planning for its investigational adeno-associated virus vector (AAV)-based gene therapies.

Michael is a highly accomplished commercial leader with a focus on cell and gene therapies and a proven track record of launching innovative drugs for rare diseases, said Joo Siffert, M.D., Chief Executive Officer of Abeona. As we advance our pivotal Phase 3 VIITAL study of EB-101 in RDEB, Michaels proven track record in commercialization, supply chain management for personalized, autologous cell therapies, experience in developing novel launch plans, working closely with governments around the world to ensure patients have access, and ability for building commercial and organizational capabilities will lay the groundwork for our potential go-to-market strategy for EB-101. Furthermore, his history of integrating commercial perspective into pipeline programs will be instrumental in positioning our investigational AAV gene therapies to shape the treatment paradigm for patients with MPS IIIA, MPS IIIB, and other rare genetic diseases.

Prior to joining Abeona, Mr. Amoroso held various senior level commercial positions at leading biopharmaceutical companies, including Kite, Eisai Inc., Celgene Corporation (now a subsidiary of Bristol-Myers Squibb Company), and Sanofi. At Kite, he was responsible for the companys worldwide commercial organization leading the commercialization efforts for the autologous CAR T-cell therapy, YESCARTA, and the future cell therapy pipeline. Before Kite, Mr. Amoroso was Senior Vice President, Americas for Eisais Commercial Oncology Business Group, where he was accountable for teams charged with creating and driving commercial strategy and implementation for the companys approved products and earlier-stage assets. Previously, Mr. Amoroso worked at Celgene for six years in several commercial roles before serving as the organizations Commercial Lead for CAR T-cell therapy programs. In this capacity, he helped Celgene develop an organizational model to commercialize cell therapies including specialized manufacturing and customer services for patients with lymphoma and myeloma. Before joining Celgene, Mr. Amoroso held various marketing and sales leadership positions over his 10-plus year tenure at Sanofi. Mr. Amoroso earned his M.B.A. in Management from the Stern School of Business, New York University, and his B.A. in Biological Sciences, summa cum laude, from Rider University.

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

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

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

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

Continued here:
Abeona Therapeutics Announces Appointment of Michael Amoroso as Chief Commercial OfficerTo lead Abeona's commercial organization, EB-101...

Biogen has picked up a new, preclinical gene therapy program from Massachusetts Eye and Ear for inherited retinal degeneration due to mutations in the PRPF31 gene, among the most common causes for autosomal dominant retinitis pigmentosa. Theyre building on the work of Harvards Eric Pierce. The treatment of IRDs with highly effective AAV-based gene therapies is core to Biogens ophthalmology strategy, said Chris Henderson, the research head at Biogen. This agreement underscores our commitment to that strategy and builds off of our acquisition of Nightstar Therapeutics in 2019 and our active clinical trials of gene therapies for different genetic forms of IRD.

Sarepta has inked a collaboration with Hansa to develop their experimental drug imlifidase as a pre-treatment for their gene therapies. The drug is intended for use in patients who have neutralizing antibodies that would prevent gene therapies for Duchenne muscular dystrophy and Limb-girdle muscular dystrophy from working. Hansa gets a $10 million upfront and up to $397.5 million in milestones.

Its been raining money on Wall Street at least when it comes to drug developers. CRISPR Therapeutics $CRSP and Acceleron each raised a whopping $450 million this week after pricing follow-on offerings. CRISPR priced 6,428,572 common shares at a public offering price of $70.00 per share, while Acceleron $XLRN auctioned off 4,864,864 shares of common stock at a price to the public of $92.50 per share.

The transatlantic biotech player Immatics has completed its flip onto Nasdaq through the Arya Sciences Acquisition Corp. The cancer drug biotech will trade as $IMTX after it raised $253 million in the process. The SPAC was set up by Perceptive Advisors.

Seattle-based Neoleukin Therapeutics, meanwhile, raised $76.2 million $NLTX for its work on protein therapeutics.

Link:
Biogen bags an AAV gene therapy program from Massachusetts Eye and Ear; Biotechs raised $1B-plus in latest round of follow-ons - Endpoints News

Hemophilia Gene Therapy Market

IndustryGrowthInsights, 09-07-2020: The research report on the Hemophilia Gene Therapy Market is a deep analysis of the market. This is a latest report, covering the current COVID-19 impact on the market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions. The rapidly changing market scenario and initial and future assessment of the impact is covered in the report. Experts have studied the historical data and compared it with the changing market situations. The report covers all the necessary information required by new entrants as well as the existing players to gain deeper insight.

Furthermore, the statistical survey in the report focuses on product specifications, costs, production capacities, marketing channels, and market players. Upstream raw materials, downstream demand analysis, and a list of end-user industries have been studied systematically, along with the suppliers in this market. The product flow and distribution channel have also been presented in this research report.

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The Major Manufacturers Covered in this Report:Spark TherapeuticsUltragenyxShire PLCSangamo TherapeuticsBioverativBioMarinuniQureFreeline TherapeuticsHemophilia Gene Therap

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By Types:Hemophilia AHemophilia BHemophilia Gene Therap

By Applications:Hemophilia A Gene TherapyHemophilia B Gene Therapy

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In conclusion, the Hemophilia Gene Therapy Market report is a reliable source for accessing the research data that is projected to exponentially accelerate your business. The report provides information such as economic scenarios, benefits, limits, trends, market growth rate, and figures. SWOT analysis is also incorporated in the report along with speculation attainability investigation and venture return investigation.

About IndustryGrowthInsights:IndustryGrowthInsights has set its benchmark in the market research industry by providing syndicated and customized research report to the clients. The database of the company is updated on a daily basis to prompt the clients with the latest trends and in-depth analysis of the industry. Our pool of database contains various industry verticals that include: IT & Telecom, Food Beverage, Automotive, Healthcare, Chemicals and Energy, Consumer foods, Food and beverages, and many more. Each and every report goes through the proper research methodology, validated from the professionals and analysts to ensure the eminent quality reports.

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Hemophilia Gene Therapy Market 2020 Global analysis, opportunities and forecast to 2026 | By Top Leading Vendors like Spark Therapeutics, Ultragenyx,...

Gene Therapy Market Forecast 2020-2026

The Global Gene Therapy Market research report provides and in-depth analysis on industry- and economy-wide database for business management that could potentially offer development and profitability for players in this market. This is a latest report, covering the current COVID-19 impact on the market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions. The rapidly changing market scenario and initial and future assessment of the impact is covered in the report. It offers critical information pertaining to the current and future growth of the market. It focuses on technologies, volume, and materials in, and in-depth analysis of the market. The study has a section dedicated for profiling key companies in the market along with the market shares they hold.

The report consists of trends that are anticipated to impact the growth of the Gene Therapy Market during the forecast period between 2020 and 2026. Evaluation of these trends is included in the report, along with their product innovations.

Get a PDF Copy of the Sample Report for free @ https://industrygrowthinsights.com/request-sample/?reportId=168032

The Report Covers the Following Companies:Bluebird BioSangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVical Inc.AdvantageneGene Therap

By Types:Ex vivoIn VivoGene Therap

By Applications:CancerMonogenicInfectious diseaseCardiovascular diseaseOther

Furthermore, the report includes growth rate of the global market, consumption tables, facts, figures, and statistics of key segments.

By Regions:

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Years Considered to Estimate the Market Size:History Year: 2015-2019Base Year: 2019Estimated Year: 2020Forecast Year: 2020-2026

Important Facts about Gene Therapy Market Report:

What Our Report Offers:

Make an Inquiry of This Report @ https://industrygrowthinsights.com/enquiry-before-buying/?reportId=168032

About Industrygrowthinsights:Industrygrowthinsights has set its benchmark in the market research industry by providing syndicated and customized research report to the clients. The database of the company is updated on a daily basis to prompt the clients with the latest trends and in-depth analysis of the industry. Our pool of database contains various industry verticals that include: IT & Telecom, Food Beverage, Automotive, Healthcare, Chemicals and Energy, Consumer foods, Food and beverages, and many more. Each and every report goes through the proper research methodology, validated from the professionals and analysts to ensure the eminent quality reports.

Contact Info:Name: Alex MathewsAddress: 500 East E Street, Ontario,CA 91764, United States.Phone No: USA: +1 909 545 6473 | IND: +91-7000061386Email: [emailprotected]Website: https://Industrygrowthinsights.com

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How Gene Therapy Market Will Dominate In Coming Years? Report Covering Products, Financial Information, Developments, Swot Analysis And Strategies |...

Orphan Drug Designation granted to Neurogenes adeno-associated virus vector with engineered transgene encoding the human CLN5 gene

Neurogene Inc., a company founded with a mission to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to adeno-associated virus vector with engineered transgene encoding the human CLN5 gene for patients with CLN5, a form of Batten disease. Batten disease, a common name for a rare class of diseases called neuronal ceroid lipofuscinoses (NCL), affects an estimated 2-4 out of every 100,000 children in the United States.

"CLN5 is a devastating neurodegenerative disease with no FDA approved treatment options," said Rachel McMinn, Ph.D., Neurogenes Founder and Chief Executive Officer. "Receiving Orphan Drug Designation from the FDA is an important regulatory milestone, and we look forward to advancing our gene therapy program into the clinic."

The FDA grants Orphan Drug Designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides benefits to drug developers designed to support the development of drugs and biologics for small patient populations with unmet medical needs. These benefits include assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.

About CLN5Batten disease, also called neuronal ceroid lipofuscinoses (NCLs), is a family of rare and fatal neurodegenerative diseases caused by pathogenic changes in one of a series of genes that result in the accumulation of abnormal storage material across multiple organ systems, including the brain, eye, skin and other tissues. The most prominent effects occur in the brain, where the progressive and inevitable loss of neurons lead to devastating declines in cognitive and motor function in those with Batten disease. The subtype CLN5 is a rare, pediatric-onset and rapidly progressive disease caused by defects in the CLN5 gene. CLN5 disease is characterized by progressive deterioration in intellectual and motor capabilities and vision loss, as well as seizures and death in childhood or adolescence. Diagnosis of the disease is confirmed through genetic testing. Currently, there are no approved disease-modifying therapies available.

About Genetic TestingNeurogene is committed to lowering the barriers of obtaining a genetic diagnosis for patients and has partnered with Invitae to co-sponsor two genetic testing programs. Healthcare providers can order, at no charge, an Invitae Epilepsy panel for any child under the age of eight who has had an unprovoked seizure, or the Detect Lysosomal Storage Diseases panel for patients suspected of having a lysosomal storage disease. Visit https://www.invitae.com/en/sponsored-testing/ for more details.

About Neurogene Inc.Neurogene Inc. is focused on developing life-changing genetic medicines for patients and their families affected by rare, devastating neurological diseases. We partner with leading academic researchers, patient advocacy organizations and caregivers to bring therapies to patients that address the underlying genetic cause of a broad spectrum of neurological diseases where no effective treatment options exist today. Our lead programs are designed to use AAV-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene. Neurogene is also investing in novel technology to develop treatments for diseases not well served by gene therapy. For more information, visit http://www.neurogene.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200707005106/en/

Contacts

Nikki Kiddnikki.kidd@rturnmktg.com 773-257-7523

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FDA Grants Orphan Drug Designation to Neurogenes Gene Therapy for the Treatment of CLN5 Batten Disease - Yahoo Finance

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

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

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

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

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

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

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

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/

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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)

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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

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