KING OF PRUSSIA, Pa., Jan. 22, 2020 /PRNewswire/--The Discovery Labs and Deerfield Management Company have formed The Center for Breakthrough Medicines, a Contract Development and Manufacturing Organization (CDMO) and specialty investment company, to alleviate the critical lack of capacity that is preventing patients from accessing critically needed cell and gene therapies. The CDMO is occupying over 40 percent of The Discovery Labs' 1.6 million square foot biotech, healthcare and life sciences campus in King of Prussia, PA.

The CDMO provides preclinical through commercial manufacturing of cell and gene therapies and component raw materials. It offers process development, plasmid DNA,viral vectors, cell banking, cell processing, and support testing capabilities all under one roof. The immense $1.1 billion facility will provide instant capacity as the largest known single source for accelerating the delivery and affordability of lifesaving and life-changing therapies from the bench to the patient's bedside.

The Company has initiated a substantial hiring effort targeting the best and brightest of the life sciences community including, experts in CGMP manufacturing. The Company expects to hire over 2,000 team members within the next 30 months.

The CDMO has retained Nucleus Careers, a cloud-based specialty life sciences human capital recruiting and retention management expert, to buildout the entire team. Nucleus has proprietary recruiting and retention software designed for large scale human capital buildouts of high growth companies.

In addition to developing the world's largest single-point cell and gene therapy manufacturing facility, The Discovery Labs is establishing THE COLONY which will provide custom built discovery labs, breakthrough funding, sponsored research agreements, housing and relocation for the world's leading iconic experts in cell and gene therapy.

THE COLONY will seek to work hand in hand with scientists from both academic and pharmaceutical institutions to unlock and expedite groundbreaking therapies.

Marco A. Chacn, Ph.D., Founder of Paragon Bioservices and Chairman of The Discovery Labs states, "musicians, artists, members of religious communities and great thinkers throughout time have formed colonies where freedom of thought and expression combined with unlimited dreams and potential have resulted in the world's greatest accomplishments." Dr. Chacn went on to say, "the goal of THE COLONY is to unshackle the potential of the world's greatest scientific minds."

The ability for the industry's greatest scientists to cohabitate, collaborate, cooperate, and communicate via technology and in person will create an exponential therapeutic "X FACTOR." THE COLONY seeks to unlock institutional barriers prohibiting the world's greatest scientists from moving at a pace necessary in today's ever-changing therapeutic revolution. THE COLONY will partner with the institutions where the scientists currently work by providing equity, license fees, and revenue sharing.

"The Center for Breakthrough Medicines will be serving companies from the earliest stages through commercialization. Its exceptional scale and offering will quickly relieve the production bottleneck for advanced therapies by reducing the time, complexity, and cost of commercializing vitally needed gene and cell therapies," noted Audrey Greenberg, Board Member and Executive Managing Director for The Discovery Labs.

The addition of this end-to-end manufacturing capability is expected to significantly enhance the offerings of The Discovery Labs in an area that has become one of the largest life sciences hubs in the world. Renovations are underway to construct a total of 86 plasmid, viral vector production, universal cell processing, CGMP testing, process development and cell banking suites. The viral vector and cell processing suites will be fully compliant with both U.S. Food and Drug Administration and European Medicines Agency standards. All suites will offer the flexibility to meet client-specific workflows and will be able to adapt quickly to meet demand. The Company is in the process of reserving capacity now for late 2020.

"Today brilliant scientists are advancing an unprecedented number of gene and cell therapy drug candidates. The real tragedy, however, is a scarcity of manufacturing know-how, which is complex and expensive," said Alex Karnal, Partner and Managing Director of Deerfield Management and a Board Member of the Discovery Labs. "With its visionary business model, it is hoped that The Center for Breakthrough Medicines will help realize the promise of cell and gene therapies in time to treat the many patients who need them."

The Discovery Labs provides a central campus where the world's greatest scientists can collaborate on new therapeutic discoveries to eradicate diseases affecting small and large segments of the global population. The Center for Breakthrough Medicines will work with these leaders, life sciences companies, large pharmaceutical companies, and academic and government institutions.

This new manufacturing capability is a transformational addition to The Discovery Labs market offering and dovetails with The Discovery Labs biotech incubator, Unite IQ. Unite IQ offers immediate space to emerging life sciences companies and scientists giving them the ability to grow from startup to enterprise company on one campus. The incubator and accelerator space at Unite IQ provides a comprehensive home for startups with every resource needed to initiate business operations. Unite IQ tenants are expected to utilize the discovery, development, testing, and manufacturing capabilities of the Center for Breakthrough Medicines with seamless forward integration of processes and analytics, and seamless tech transfer from research lab to large scale production

The Emerging Field of Cell and Gene Therapy in Pennsylvania

The demand for clinical and commercial manufacturing capacity is acute and expected to remain that way. The current shortfall in manufacturing for cell and gene therapies is severely underserved with few approved products. There are currently approximately 1,100 advanced therapies in the pipeline pending FDA approval. This will greatly increase highly skilled manufacturing demand. Dr. Peter Marks, Director of the FDA Center for Biologics Evaluation and Research, states, "what keeps me up at night is will we be able to manufacture these on a scale that will allow us to bring the benefit of these therapies to patients?"He further added that "if we can help see cost of goods and ability to manufacture reproducibly improve, I think that'll be a big thing."All of this adds up to a supply constrained market that The Center for Breakthrough Medicines aims to help address.

With the potential to treat and even cure disabling, and deadly diseases, gene and cell therapies are ushering in a new era of medicine. These therapies may eventually be able to cure genetic conditions, such as cystic fibrosis, hemophilia A, and a range of cancers. The Philadelphia area has become the epicenter for the flourishing field of gene and cell therapy. Research from CBRE currently ranks the market among the top biotech clusters for medical research and health services. The cluster has become known worldwide as "Cellicon Valley"for its leadership in research and development of this rapidly evolving field. The Discovery Lab's suburban Philadelphia location offers a talent rich environment due to the area's preponderance of large pharmaceutical companies and the Philadelphia region's position boasting the top 10 universities and primary school systems in nation.

Over the past three years, multiple Philadelphia companies have received approvals for major breakthroughs in cell and gene therapy. In 2017, the U.S. FDA approved the first-ever CAR-T cell therapy, Novartis's Kymriah, which originated at the University of Pennsylvania. Shortly thereafter, the FDA gave landmark approval for the first-ever gene therapy to treat a genetic blindness condition to Spark Therapeutics, a start-up founded by researchers at Children's Hospital of Philadelphia. These discoveries and others in the pipeline are attracting billions of dollars of venture capital. The Greater Philadelphia Region set a recent record in venture capital financing.

The Discovery Labs Center for Breakthrough Medicines joins more than 25 healthcare, life sciences and tech-enabled companies that already call The Discovery Labs King of Prussia home.

Brian O'Neill, Founder of The Discovery Labs Center for Breakthrough Medicines, and Tony Khoury, Board Member of The Discovery Labs and Engineer at Project Pharma, will be speaking at the 2020 PhacilitateWorld Stem Cell Summit discussing The Future of Gene Therapy Manufacturing at 4 p.m. today at the Hyatt Regency in Miami, Florida.

Contact Audrey Greenberg at agreenberg@thediscoverylabs.com for more information about development services, manufacturing capacity, incubator space or leasing information at the property.

About The Discovery LabsPart of MLP Ventures, The Discovery Labs is a global provider of world-class cGMP manufacturing, turnkey laboratory solutions, critical materials and office space that support therapeutic products and services to the biotechnology and pharmaceutical industry so that groundbreaking medicines get to the patients that need them. The location in eastern King of Prussia is a prototype for a global rollout of The Discovery Labs, providing Big Pharma, emerging life sciences, consumer and technology companies flexible, end-to-end technical real estate and business infrastructure for the customer's entire lifecycle from discovery to delivery, including manufacturing capacity. It is the first fully integrated environment that merges technology and life sciences under one roof to drive innovation.

About Deerfield Management

Deerfield is a healthcare investment management firm committed to advancing healthcare through investment, information and philanthropy.

Media Contact:Tony DeFazio, DeFazio Communications(o) 484-534-3306 (c) 484-410-1354tony@defaziocommunications.com

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CHICAGO, Jan. 20, 2020 /PRNewswire/ -- According to the new market research report "Gene Therapy Marketby Vectors [Non-viral (Oligonucleotides), Viral (Retroviral (Gammaretroviral, Lentiviral), Adeno-associated], Indication (Cancer, Neurological Diseases), Delivery Method (In Vivo, Ex Vivo), Region - Global Forecast to 2024", published by MarketsandMarkets, is projected to reach USD 13.0 billion by 2024 from USD 3.8 billion in 2019, at a CAGR of 27.8% from 2019 to 2024.

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The high incidence of cancer and other target diseases, availability of reimbursement, and the launch of new products are the major factors driving the growth of the market.

The non-viral vectors segment accounted for the largest share of the 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.

Browsein-depth TOC on "Gene Therapy Market" 127 - Tables25 - Figures 129 - Pages

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

Based on indication, the 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.

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North America is the largest regional market for gene therapy products

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.

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).

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Regenerative Medicine Marketby Type [Cell-Based Immunotherapy & Cell Therapy (Allogeneic & Autologous Products), Tissue Engineering, Gene Therapy], Applications (Wounds & Dermal, Musculoskeletal, Oncology), Region - Global Forecast to 2024

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Early research in animal models and human samples reveals how loss of communication between nerve cells contributes to the symptoms of multiple sclerosis (MS), and shows how gene therapy could be used to preserve such connections and protect againstvision loss.

Researchers say their work identifies a new approach for developing MS therapies that target nerve cell communication, rather than myelin loss, and could be applicable to other neurodegenerative disorders.

The study, Targeted Complement Inhibition at Synapses Prevents Microglial Synaptic Engulfment and Synapse Loss in Demyelinating Disease, was published in the journal Immunity.

MS is a neurological disease marked by inflammation and a self-attack of the immune system against a persons brain, spinal cord, and optic nerves.

This attack damages the protective fatty substance covering nerve fibers (axons), which are necessary for proper transmission of nerve signals called myelin. As the myelin sheath is lost (demyelination), the communication between nerve cells is damaged or even interrupted, and nerve cell death occurs, leading to a range of disease symptoms.

Some MS patients experience a version of the disease called progressive MS, in which symptoms continuously worsen over time while their central nervous system (brain and spinal cord) shrinks (atrophies), and the junctions at which nerve cell terminals meet to communicate with each other, called synapses, are lost.

The majority of MS medications work to inhibit the self-attacking immune responses and inflammatory demyelination, but the neurodegenerative aspects of the disease have been more difficult to stop, particularly for patients with progressive MS.

Most MS research and FDA-approved treatments focus on demyelination and axon death, Dorothy P. Schafer, PhD, professor at the University of Massachusetts Medical School, said in a press release.

Far less is known about what happens to the synaptic connections between neurons, which has proven to be a key aspect of neurodegeneration likely leading to cognitive decline in other diseases such as Alzheimers disease, Schafer said.

Using tissue samples from deceased MS patients, a primate model of MS, and mice models of demyelinating disease, Schafer and colleagues investigated how synapses change during MS.They specifically looked at synapses involved in transmitting visual information from the eye to the brain via the optic nerve.

According to the studys first author, Sebastian Werneburg, PhD, a postdoctoral researcher at Schafers lab, the visual system is an ideal model for investigating MS because its easy to access for therapeutic intervention, subtle changes can be readily detected, and the visional pathway is affected in almost half of all patients with the disease.

Most MS patients experience vision problems at some point, which result from damage to the optic nerve or from lack of coordination in the eye muscle. These problems can be the first indication of the disease.

Similar to other neurodegenerative diseases, researchers found a profound synaptic loss in patient samples as a consequence of immune cells called microgliaeating nerve cell connections.

Microglia are cells that serve as one of the first and main forms of immune defense in the central nervous system, acting to clear cellular debris and dead neurons via phagocytosis a process by which some cells engulf other cells or particles.

In mice, synapse loss occurred independently of local demyelination and neuronal degeneration, but coincided with a rise in a specific immune factor called C3. C3 is part of the complement system, and is normally not present in the brains of adults. It is produced and activated during demyelinating diseases, but it is not clear why.

As C3 was seen to bind to synapses in models of MS, researchers reasoned this complement protein might be involved with the ongoing destruction of synapses in mice with MS-like disease.

To test this hypothesis, they specifically neutralized C3 at synapses of the visual pathway using gene therapy in mice. The strategy basically worked by delivering genetic material to synapses that provided instructions for the production of a C3 inhibitor.

After injection of the therapy, the inhibitor successfully blocked C3, reduced microglia engulfment, and preserved nerve cell connections, which improved eyesight in mice.

As a result of this inhibition, we saw improved visional function in mice, Werneburg said.

Overall, based on the results, the team believes that C3 probably is sending a signal to microglia telling them to eliminate synapses.

The next step will be to determine how C3 turns active during MS and other neurodegenerative diseases.

Its possible that therapies targeting different circuits of the brain can be used to protect against synaptic damage in other neurodegenerative diseases such as Alzheimers, Schafer said.

Ana is a molecular biologist with a passion for discovery and communication. As a science writer she looks for connecting the public, in particular patient and healthcare communities, with clear and quality information about the latest medical advances. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in genetics, molecular biology, and infectious diseases

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Patrcia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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Gene Therapy Recovers Vision in Mice Models of MS, Uncovers How... - Multiple Sclerosis News Today

Recommendation and review posted by Bethany Smith

New research by Dorothy P. Schafer, PhD, at the University of Massachusetts Medical School, reveals the molecular process in which synaptic connections in the brain are damaged in multiple sclerosis and how this contributes to neurodegenerative symptoms. The paper, published in Immunity, also shows how gene therapy may be used to preserve neural circuits and protect against vision loss in the disease.

These findings suggest a path for developing therapies that may protect synapses from the damaging effects of MS and could be broadly applicable to other neurodegenerative disorders, according to Dr. Schafer, assistant professor of neurobiology, and Sebastian Werneburg, PhD, a postdoctoral fellow in the Schafer lab.

"Most MS research and FDA-approved treatments focus on demyelination and axon death," said Schafer. "Far less is known about what happens to the synaptic connections between neurons, which has proven to be a key aspect of neurodegeneration likely leading to cognitive decline in other diseases such as Alzheimer's disease."

Multiple sclerosis is a neurological disease of the central nervous system affecting more than 2 million people worldwide. The disease involves an abnormal response of the body's peripheral immune system against the brain, spinal cord and optic nerves, which damages the fatty substance surrounding nerve fibers called myelin. Recurrent episodes of inflammation result in demyelination. As the myelin is stripped away, the nerve fibers are exposed to inflammatory attacks from the immune system and the transmission of nerve signals within the central nervous system are altered or stopped completely. A small subset of MS patients experience chronic progressive neurodegenerative symptoms accompanied by significant synaptic loss and central nervous system atrophy. This version of the disease is called progressive MS.

FDA-approved medications for treating MS have been developed to limit and reduce the number of relapses, which delay progression of the disease and minimize demyelination, but there is no cure for the disease and patients are still left with disability. Current therapies work to inhibit peripheral immune attack of the central nervous system and inflammatory demyelination, but the neurodegenerative aspects of the disease have proven harder to decelerate, particularly for patients with progressive MS.

Vison loss is one of the most common symptoms of MS and is often one of the first that patients notice. Problems with vision result from damage to the optic nerve that connects the eye to the brain or from lack of coordination in the eye muscle.

"The retinogeniculate system, which comprises neurons that extend their axons via the optic nerve to the thalamus in the brain, is an ideal model for investigating MS because it's easy to access for therapeutic intervention, subtle changes can be readily detected and the visional pathway is affected in almost half of all patients with the disease," said Dr. Werneburg.

Profound synaptic loss was observed in animal models as microglia engulfed and eliminated presynaptic connections. Microglia are the immune cells of the central nervous system and are emerging as key players in regulating neural circuit structure in health and disease. One of the vast number of functions microglia perform in the brain is similar to the role macrophages perform in the immune system: clearing cellular decay and dead neurons from tissue.

"We found the protein C3 in abundance at synapses," said Werneburg.

C3 is not normally found in adult brain tissue. C3 protein usually only shows up in neural tissue during the developmental stages of the brain when synapses are being pruned. Synaptic pruning eliminates weak or unused synapsis as the brain matures to help efficiency and conserve energy.

In the case of demyelinating disease, it is not known why C3 is being produced and activated. This complement protein binds to synapses, sending the signal to microglia that the otherwise healthy-seeming synapse should be eliminated. This leads microglia to attack synapses.

Schafer, in collaboration with Guangping Gao, PhD, the Penelope Booth Rockwell Professor in Biomedical Research, professor of microbiology & physiological systems, director of the UMMS Horae Gene Therapy Center and Viral Vector Core, and co-director of the Li Weibo Institute of Rare Disease, used a gene therapy approach and adeno-associated virus to deliver Crry, an inhibitor of C3, specifically to synapses in the visual system while leaving the rest of the brain untouched, to see if synapses could be spared and vision preserved. Crry is a natural inhibitor of complement proteins such as C3. These regulators help protect cells or tissue from unwanted attack by the immune system.

After injection of the AAV into the circuit, Crry localized to synapses and successfully preserved them by binding to C3 so microglia couldn't damage them.

"As a result of this inhibition, we saw improved visional function in mice," said Werneburg.

Schafer said the protective effects of the AAV-delivered inhibitor were specific to the visual circuit. "It's possible that therapies targeting different circuits of the brain can be used to protect against synaptic damage in other neurodegenerative diseases such as Alzheimer's."

The next step for Schafer and colleagues will be to determine how the C3 protein is being activated and produced during MS and other neurodegenerative diseases.

Reference: Werneburg, S., Jung, J., Kunjamma, R. B., Ha, S.-K., Luciano, N. J., Willis, C. M., Gao, G., Biscola, N. P., Havton, L. A., Crocker, S. J., Popko, B., Reich, D. S., & Schafer, D. P. (2020). Targeted Complement Inhibition at Synapses Prevents Microglial Synaptic Engulfment and Synapse Loss in Demyelinating Disease. Immunity, 52(1), 167-182.e7. https://doi.org/10.1016/j.immuni.2019.12.004

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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Gene Therapy Protects Eyesight in Models of Multiple Sclerosis - Technology Networks

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Gene therapy is a very hot area. Scientists can now fix defective genetic mutations that cause diseases. An initial focus is in rare diseases with urgent clinical needs, but the future holds the possibility for significant expansion.

Another promising area is the broadening of the scope of cell therapy, which is currently being used for blood cancers, into solid tumors. If this is successful, it could transform the field of personalized cancer care.

Finally, Biogen has a novel antibody drug for the treatment of Alzheimers. If the FDA approves this therapy, it could lead to the resurgence of Alzheimers drug development, which represents a huge unmet medical need.

How about at OMRF? What did 2019 deliver, and what has you excited for 2020?

The FDA approved Adakveo in November, a new drug for sickle cell disease based on discoveries made by OMRFs Dr. Rod McEver. In 2020, Im excited about Progentec, an OMRF spin-off company thats developing novel diagnostic and disease management solutions for autoimmune patients.

Also, we have a new startup called Ambocept thats developing novel cancer therapeutics. Finally, two drugs that began in OMRF labs are progressing in clinical trials: one to treat hearing loss, the other for people suffering from deadly brain cancers.

Paula Burkes, Business writer

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Meet Helen Obando, a shy 16-year-old who likes to dance when her body isnt ravaged by the debilitating symptoms of sickle cell disease. The genetic blood disorder can cause strokes, organ damage and intense pain.

After a lifetime of pain and potential permanent damage to her body, Helen had the opportunity to receive a breakthrough experimental treatment at Boston Childrens Hospital that would make her the youngest person in the U.S. to have her DNA reset in an attempt to cure her sickle cell disease.

The outcome of her gene therapy could help determine how an estimated 100,000 people in the U.S. and millions more around the world are treated. Sickle cell disease most commonly affects people from sub-Saharan Africa, and about 1 in 500 African-Americans have the blood disorder, the most commonly inherited blood disorder in the U.S. But some people with the disease have southern European, Middle Eastern or Asian backgrounds or, like Helen, are Hispanic. For decades, attention and money for research have not matched the scale of the problem.

Why has it taken so long for the scientific research community to push ahead with promising therapies for sickle cell?

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The first six months of his young life have been a roller-coaster but Mighty Hudson Cowie is back home in Alberta after an experimental gene therapy procedure in Tennessee was successful.

Its been so long since weve got to see our family, our friends, Hudsons dad Ian said. Even just to be able to show him off to everybody. Weve spent the last six months seeing how amazing of a little guy he is.

Now we finally get to share that with everybody.

Hudson was diagnosed with Severe Combined Immunodeficiency (SCID) within days of his birth on June 23. The condition, known to many as Bubble Boy Disease, prevents his body from fighting illnesses. Essentially, he didnt have an immune system.

READ MORE:Hundreds attend donor drive for Mighty Hudson, Alberta baby with rare immune disease

In August, Hudson was accepted into a gene therapy program at St. Jude Childrens Research Hospital in Memphis. A medical team removed some of his bone marrow cells and replaced the faulty gene with a corrected one. The cells were placed back into his body through an IV.

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They took his own bone marrow out in his bone marrow, hes got hematopoietic stem cells. They were able to take those cells and modify them to create a product, a repaired version of those cells, and then give them back to him, Ian told Global News on Monday.

Medicine is amazing. We took a chance on science and were so glad that we did. Its amazing what theyre able to do.

Ian and his wife Hayley were weighing two options for their little boy: using donor cells or a revamped version of Hudsons own cells. They decided on the second, which meant a trial at St. Judes.

We weighed the pros and cons of both options and for us, [and] personally, we decided that gene therapy seemed like the safer way to go, Ian said.

It didnt rely on a donor, it was the new up-and-coming medicine, the existing results for gene therapy were already incredibly promising and then, as an added bonus, it had substantially less chemotherapy requirements.

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WATCH: (Sept. 9, 2019) A Morinville baby with a rare disorder received a life-changing procedure in the U.S. Hudson Cowies parents give Su-Ling Goh an update on therapy for his immune system.

It honestly sounded too good to be true, Hayley added.

With bone marrow transplant, we heard of graft versus host disease, where its somebody elses cells and they can reject them. With gene therapy, having his own cells, he wasnt going to reject them because his body already knows them.

A few months after the transplant, they started to see results: Hudsons first T-cells, a type of white blood cell thats a key component of the immune system.

From three months, it exploded. At four months, it was even more T-cells. The growth was exponential, Ian said.

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I think thats the thing every doctor hopes to get to say to their patients one day: Hes cured. And the day that we left Memphis, she said that. She said: Hes cured.

We squealed like little schoolgirls. It was very exciting.

Hudsons immune system right now can be compared to that of a newborn baby.

As hes introduced to small bugs, itll just keep growing and growing and be normal, Hayley explained.

There are still unknowns, but the family is very hopeful.

He was Patient 12 on the trial and from what we were informed, everyone on the trial has done tremendously well, Ian said.

I just think its amazing, Hayley added. Its amazing that we were the first ones who got on newborn screening and that this trial was even available for us. It just seems that everything completely lined up.

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I feel like everything has just fallen into place to get him cured.

The treatment is just amazing. Our doctors are brilliant, its just all incredible and hes doing great.

Hudson was one of the first babies to be screened for SCID through a new program. Since SCID was added in May 2019, the Alberta Health Services Newborn Metabolic Screening Program has screened over 34,000 newborns and has diagnosed four cases of SCID.

While very much welcome, being home is still a big change for the Cowies.

Im still processing, even now, Hayley said. Its nice to go see people but we were in isolation for so long that it doesnt just flip all of a sudden. I know were still really protective and we can hear a cough from miles away.

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It was surreal to leave but I feel like Im still dreaming a little bit.

They havent attempted any big outings yet especially given its flu season but they have been able to go to some family dinners.

Its an adjustment for sure, but a good adjustment, Hayley said. He loves people Hes always just smiling and playful hes just happy.

The Cowies will be back in Tennessee for one day for a checkup this weekend. Hudson will have another checkup with the St. Judes team next month. Hell have followups at least once a year until hes 10 years old.

2020 Global News, a division of Corus Entertainment Inc.

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Alberta baby Mighty Hudson home after gene therapy: hes cured - Global News

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Building on an R&D tie-up with an Indian stem cell institution last year, UK biotech PhoreMost has begun a collaboration with Japans Otsuka working on several gene therapy projects.

Cambridge-based PhoreMost said it will use its next-generation phenotypic screening platform Siteseeker to identify novel targets for Otsukas therapeutics discovery programmes.

Novel targets identified will be further validated and characterised by Otsuka as part of its internal development pipeline, with an initial focus on gene therapy applications of identified targets.

Siteseeker looks at different protein shapes to find functionally active peptides that can be targeted by new therapies.

The technology looks at the entire proteome all of the proteins expressed in a live cell environment looking for druggable targets for a chosen disease.

Financial details of the agreement were not disclosed.

Dr Chris Torrance, CEO of PhoreMost, said: This collaboration with Otsuka is further recognition of the power of the Siteseeker approach to drive the identification of novel, druggable targets.

We are particularly excited to be exploring not only small molecule therapeutics but also gene therapy applications of our platform.

PhoreMost was one of two UK-based companies to receive funding from the government-backed agency Innovate UK to receive funding for small molecule research.

The 1 million funding was announced in 2018, and supported PhoreMost and the immune-oncology firm NeoPhore.

The companies won the funding as part of a competition organised by Innovate UK and funded by the UKs Biomedical Catalyst.

PhoreMost is also working with Indias Centre for Chemical Biology and Therapeutics, part of the Institute for Stem Cell Science and Regenerative Medicine (inStem).

The project began in July last year and, with funding from the Indian government, aims to create chemical tools that modulate novel classes of drug targets.

InStem is researching the genetic mechanisms of potency, differentiation and proliferation in human pluripotent cells.

It aims to examine diseases that can potentially be treated by stem cells.

Follow this link:
UK biotech PhoreMost to work with Otsuka on gene therapy projects - - pharmaphorum

Recommendation and review posted by Bethany Smith

PALM BEACH, Florida, Jan. 22, 2020 /PRNewswire/ -- Pancreatic cancer is a notoriously aggressive and hard-to-treat malignancy; the five-year survival rate is less than 10%. Treatment options for pancreatic cancer are limited to surgery, radiation, and chemotherapies; thus, demand is high for safer, more-efficacious drugs, which will serve to drive the market. ResearchAndMarkets projects that the global pancreatic cancer therapy market is expected to reach US$ 4,056.4 Mn in 2025 from US$ 2,011.2 Mn in 2017. The market is estimated to grow with a CAGR of 8.1% from 2018-2025. The growth of the pancreatic cancer therapy market is primarily attributed to the increase in number of therapies launched in the market. The development of new drugs is thus expected to drive the growth of pancreatic cancer therapy market. The report continued by saying: "However, these drugs travel throughout the body and thus can affect normal and healthy cells adversely. Blood-forming cells in the bone marrow, hair follicles and cell in the mouth, digestive tract, and reproductive system are likely to be affected by chemotherapeutic drugs. The current treatment options available for pancreatic cancer patients only help to extend the patients' lives by a few months. Active biotech and pharma companies in the markets this week include Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), Mallinckrodt plc (NYSE: MNK), Genprex, Inc. (NASDAQ: GNPX), Soligenix, Inc. (NASDAQ: SNGX), BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX).

"This demands potential drugs that are able to enhance the effectiveness of chemotherapy drugs. Combination of two or more chemotherapy drugs are a suitable way out to increase the efficacy of the drug as well as proves advantageous in the treatment the biologic therapy segment is expected to grow at the fastest rate during the coming years owing to increasing number of novel upcoming immunotherapies as well as targeted cell therapy that treats the cancer without any severe side effects and has number of therapeutic benefits over the conventional chemotherapies This demands potential drugs that are able to enhance the effectiveness of chemotherapy drugs. Combination of two or more chemotherapy drugs are a suitable way out to increase the efficacy of the drug as well as proves advantageous in the treatment."

Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC) BREAKING NEWS: Oncolytics Biotech Announces Publication of an Abstract for the 2020Gastrointestinal Cancers Symposium Highlighting CEACAM6 as a Potential Prognostic Biomarker Candidate for Pancreatic Cancer- Oncolytics Biotech currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, today stated that the previously announced abstract for a poster to be presented at the 2020Gastrointestinal Cancers Symposium sponsored by ASCO in San Francisco, has been published. The abstract highlights new biomarker data from the randomized study NCI 8601: Carboplatin and Paclitaxel With or Without Viral Therapy in Treating Patients With Recurrent or Metastatic Pancreatic Cancer.

The abstract, CEACAM6 is a candidate biomarker for Reolysin (pelareorep) sensitivity in pancreatic adenocarcinoma (PDAC), was co-authored by Dr. Anne Noonan, Department of Medical Oncology, Ohio State University Wexner Medical Center, Richard Solove Research Institute and James Cancer Hospital, and Dr. Tanios Bekaii-Saab Senior Associate Consultant, Division of Hematology/Oncology, Department of Internal Medicine, Mayo Clinic, Phoenix, Arizona.

Data in the abstract associate low levels of the gene CEACAM6 with prolonged progression free survival (PFS) in pelareorep-treated patients with pancreatic cancer, with PFS improving from 5.72 months to 10.32 months (p=0.05). This effect was not seen in non-pelareorep treated patients. Consequently, CEACAM6 may serve as a prognostic biomarker for sensitivity of pancreatic tumors to pelareorep treatment. Additional data will be announced following the poster presentation.

Read this full press release and more news for ONCY at: https://www.financialnewsmedia.com/news-oncy/

Other recent developments in the biotech industry include:Mallinckrodt plc (NYSE: MNK) a global biopharmaceutical company, recently confirmed enrollment of the first patient in the company's Phase 4, multi-center, multiple-dose, open-label study to assess the effects of Acthar Gel as a therapy option in patients with severe keratitis.

"In my experience, a considerable number of severe keratitis patients can have persistent disease that may not be resolved by first-line treatment," saidEugene McLaurin, MD and Fellow, American Academy of Ophthalmology and American College of Surgeons."I am pleased that the first patient has been enrolled in this important Phase 4 study, the results of which may potentially provide data to further support Acthar Gel as a treatment option in appropriate keratitis patients."

Genprex, Inc. (NASDAQ: GNPX) a clinical-stage gene therapy company utilizing a unique, non-viral proprietary platform designed to deliver tumor suppressor genes to cancer cells, recently announced that the U.S Food and Drug Administration (FDA) has granted Fast Track Designation for Genprex's Oncoprex immunogene therapy in combination with EGFR inhibitor osimertinib (AstraZeneca's Tagrisso, which had worldwide sales in 2018 of $1.86 billion, $2.31 billion in the first 9 months of 2019 and is currently AstraZeneca's highest grossing product) for the treatment of non-small cell lung cancer (NSCLC) patients with EFGR mutations that progressed after treatment with osimertinib alone. Oncoprex is comprised of the TUSC2 (Tumor Suppressor Candidate 2) gene complexed with a lipid nanoparticle. TUSC2 is the active agent in Oncoprex.

Genprex has treated more than 50 lung cancer patients with Oncoprex in Phase I and II clinical trials. The company believes the data from these trials are encouraging as to both safety and efficacy.

Soligenix, Inc. (NASDAQ: SNGX) a late-stage pharmaceutical company working to improve the current standard of care for numerous rare diseases through the development and commercialization of novel treatments, is nearing the release of topline data after completing final enrollment for SGX301, a pivotal Phase 3 trial targeting the treatment of cutaneous T-cell lymphoma. A successful report may position the companyfor significant increases to shareholder value before the end of Q1 2020, and ultimately position the company to commercialize its first drug in a market that is estimated at a more than $200 million revenue opportunity.

The Phase 3clinical trial is focused on the potential benefits ofSoligenix's topical drug ointment SGX301, or synthetic hypericin, in the treatment of cutaneous T-cell lymphoma (CTCL). CTCL is a rare type of Non-Hodgkin's Lymphoma that sits high on the list of conditions that has no current or effective drug treatment, pushing Soligenix to fill the demand to serve this unmet medical need.

BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX) recently announced the appointments of Charles Gayer as chief commercial officer and Allen Hodge as vice president and general manager for the United States.

Mr. Gayer joined BioCryst in 2015 as vice president of global strategic marketing. Since July 2019 he has served as interim chief commercial officer, playing a key role in defining the strategy, and building the commercial operations, to support the launch of berotralstat, BioCryst's oral kallikrein inhibitorfor hereditary angioedema (HAE).

DISCLAIMER: FN Media Group LLC (FNM), which owns and operates FinancialNewsMedia.com and MarketNewsUpdates.com, is a third party publisher and news dissemination service provider, which disseminates electronic information through multiple online media channels.FNM is NOT affiliated in any manner with any company mentioned herein. FNM and its affiliated companies are a news dissemination solutions provider and are NOT a registered broker/dealer/analyst/adviser, holds no investment licenses and may NOT sell, offer to sell or offer to buy any security.FNM's market updates, news alerts and corporate profiles are NOT a solicitation or recommendation to buy, sell or hold securities. The material in this release is intended to be strictly informational and is NEVER to be construed or interpreted as research material.All readers are strongly urged to perform research and due diligence on their own and consult a licensed financial professional before considering any level of investing in stocks. All material included herein is republished content and details which were previously disseminated by the companies mentioned in this release.FNM is not liable for any investment decisions by its readers or subscribers.Investors are cautioned that they may lose all or a portion of their investment when investing in stocks. For current services performed FNM expects to be compensated forty nine hundred dollars for news coverage of the current press releases issued by Oncolytics Biotech Inc. by a non-affiliated third party. FNM HOLDS NO SHARES OF ANY COMPANY NAMED IN THIS RELEASE.

This release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E the Securities Exchange Act of 1934, as amended and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. "Forward-looking statements" describe future expectations, plans, results, or strategies and are generally preceded by words such as "may", "future", "plan" or "planned", "will" or "should", "expected," "anticipates", "draft", "eventually" or "projected". You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events, or results to differ materially from those projected in the forward-looking statements, including the risks that actual results may differ materially from those projected in the forward-looking statements as a result of various factors, and other risks identified in a company's annual report on Form 10-K or 10-KSB and other filings made by such company with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and FNM undertakes no obligation to update such statements.

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Hopeful New Therapies For Pancreatic Cancer Expected to be a Big Boon for Biotechs - P&T Community

Recommendation and review posted by Bethany Smith

The latest release from CMIwith title Personalized Gene Therapy Treatments for Cancer Market Research Report 2020-2026 (by Product Type, End-User / Application and Regions / Countries) provides an in-depth assessment of the Personalized Gene Therapy Treatments for Cancer including key market trends, upcoming technologies, industry drivers, challenges, regulatory policies, key players company profiles and strategies. Global Personalized Gene Therapy Treatments for Cancer Market study with 100+ market data Tables, Pie Chat, Graphs & Figures is now released BY Coherent Market Insights. The report presents a complete assessment of the Market covering future trends, current growth factors, attentive opinions, facts, and industry-validated market data forecast until 2026.

Global Personalized Gene Therapy Treatments for Cancer Market Segmentations

The segmentation chapter allows readers to understand aspects of the Global Personalized Gene Therapy Treatments for Cancer Market such as products/services, available technologies, and applications. These chapters are written in a way that describes years of development and the process that will take place in the next few years. The research report also provides insightful information on new trends that are likely to define the progress of these segments over the next few years.

Free Download PDF Research Report Brochure @ https://www.coherentmarketinsights.com/insight/request-pdf/60(**Note: Free Sample with TOC, Graph, Charts)

Key companies covered as a part of this study include (Amgen, Inc., SynerGene Therapeutics, Inc., Chengdu Shi Endor Biological Engineering Technology Co., Ltd., Cold Genesys, Inc., Takara Bio, Inc., Bellicum Pharmaceuticals, Inc., Ziopharm Oncology, Inc., OncoSec Medical, Inc., Sevion Therapeutics, Inc., and Burzynski Clinic.)

Global Personalized Gene Therapy Treatments for Cancer Market and Competitive Analysis

Know your current market situation! Not only an important element for new products but also for current products given the ever-changing market dynamics. The study allows marketers to stay in touch with current consumer trends and segments where they can face a rapid market share drop. Discover who you really compete against in the marketplace, with Market Share Analysis know market position, % market Share and Segmented Revenue of Personalized Gene Therapy Treatments for Cancer Market

Segmentation and Targeting

Essential demographic, geographic, psychographic and behavioral information about business segments in the Personalized Gene Therapy Treatments for Cancer market is targeted to aid in determining the features company should encompass in order to fit into the business requirements. For the Consumer-based market the study is also classified with Market Maker information in order to better understand who the clients are, their buying behavior and patterns.

*** For the global version, a list of below countries by region can be added as part of customization at minimum cost.North America (United States, Canada & Mexico).Asia-Pacific (Japan, China, India, Australia, etc).Europe (Germany, UK, France, etc).Central & South America (Brazil, Argentina, etc).Middle East & Africa (United Arab Emirates, Saudi Arabia, South Africa, etc).

Personalized Gene Therapy Treatments for Cancer Product/Service Development

Knowing how the product/services fit the needs of clients and what changes would require to make the product more attractive is the need of an hour. Useful approaches to focus group by utilizing User Testing and User Experience Research. Demand-side analysis always helps to correlate consumer preferences with innovation.

Marketing Communication and Sales Channel

Understanding marketing effectiveness on a continual basis help determine the potential of advertising and marketing communications and allow us to use best practices to utilize an untapped audience. In order to make marketers make effective strategies and identify why the target market is not giving attention, we ensure the Study is Segmented with appropriate marketing & sales channels to identify potential market size by Revenue and Volume* (if Applicable).

Pricing and Forecast

Pricing/subscription always plays an important role in buying decisions; so we have analyzed pricing to determine how customers or businesses evaluate it not just in relation to other product offerings by competitors but also with immediate substitute products. In addition to future sales Separate Chapters on Cost Analysis, Labor*, production* and Capacity are Covered.

How geography and sales fit together

This study is helpful to all operators who want to identify the exact size of their target audience at a specific geographic location. The keywordMarket allows entrepreneurs to determine local markets for business expansion. This study answers the questions below:

1. Where do the requirements come from?2. Where do non-potential customers reside?3. What is the buying behavior of customers in a specific region?4. What is the spending power of the customers in a particular region?

** Have Any Query? Ask Our Industry Expert @ https://www.coherentmarketinsights.com/insight/talk-to-analyst/60

Having our reviews and subscribing our report will help you solve the subsequent issues:

*Uncertainty about the future: Our research and insights help our customers predict the upcoming revenue pockets and growth areas. This will guide customers to invest their resources.

*Understanding market sentiments: It is very important to have a fair understanding of market sentiment for your strategy. Our insights will help you see every single eye on market sentiment. We maintain this analysis by working with key opinion leaders on the value chain of each industry we track.

*Understanding the most reliable investment center: Our research evaluates investment centers in the market, taking into account future demand, profits, and returns. Clients can focus on the most prestigious investment centers through market research.

*Evaluating potential business partners: Our research and insights help our clients in identifying compatible business partners.

Furthermore, the years considered for the study are as follows:

Historical year 2013-2018Base year 2019Forecast period** 2020 to 2026 [** unless otherwise stated]

About Coherent Market Insights:

Coherent Market Insights is a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity.

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Personalized Gene Therapy Treatments for Cancer Market 2020-2026: Deep Analysis of Current Trends and Future Demand by Top Key Players - Vital News 24

Recommendation and review posted by Bethany Smith

In 2017 we reported on an experimental immunotherapy cancer treatment which showed extraordinary results in clinical trials. This type of treatment is known as T-cell cancer therapy.

How exactly does T-cell cancer therapy work?

The field of immunotherapy aims to "supercharge" the body's own immune system, better enabling it to fight cancer.

T-cells are white blood cells that form part of the immune system, detecting and attacking abnormal or foreign cells in the body.

In cancer patients, T-cells normally aren't able to completely rid the body of cancer.

In T-cell therapy, immune cells are engineered to better recognise and fight cancer cells. Researchers extract these cells from blood and edit them with gene transfers to produce a potent receptor that can more effectively fight cancer. The cells are then placed back into the patients body

While this type of treatment was initially used in patients with an advanced form of leukaemia, a new breakthrough meant that T-cell therapy could be used for many other types of cancer.

What does the new research entail?

According to a news report, in 2020, researchers discovered a new immune cell receptor. This means that T-cell therapy as we know it can work much, much better and fight more cancers as it was only able to recognise a handful of cancers in the past.

This meant that, because of a T-cell receptor called human leukocyte antigen (HLA), the treatment had to be personalised for every single patient. This is the receptor that enables the cells to detect and fight the cancer.

Usually HLA varies from person to person, but that is where the new discovery could be a breakthrough.

The new study was led by scientists from Cardiff University in the UK who used the CRISPR-Cas9 screening to discover a new type of receptor called MR1. The full study can be found in the journal Nature Immunology.

These receptors do exactly what HLA does, but there is one big difference the treatment doesnt differ from person to person, resulting in a much better basis for T-cell therapy.

Too soon to tell, but prospect is exciting

There is, however, one caveat. The research is still very new and was conducted in a lab, which means that positive results first need to be achieved in clinical trials.

Experts remain optimistic and call this discovery an exciting new frontier.

"This research represents a new way of targeting cancer cells, which is really quite exciting, although much more research is needed to understand precisely how it works,"says research and policy director Alasdair Rankin from the blood cancer charity Bloodwise, who was not involved in the research

Image credit: iStock

Compiled by Marelize Wilke

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New breakthrough in cancer research promises better immune therapy - Health24

Recommendation and review posted by Bethany Smith

Dublin, Jan. 23, 2020 (GLOBE NEWSWIRE) -- The "Global Healthcare Market Outlook, 2020" report has been added to ResearchAndMarkets.com's offering.

Amid rising global trade tensions and sluggish global economic outlook for 2020, the global healthcare market is expected to cross the $2 trillion mark in 2020.

Healthcare will be among the top two priorities for voters in the 2020 presidential election in the US. In the European region, looming BREXIT indecision is likely to have a strong impact on Europe's biggest digital health market (UK). Globally, 2020 will be a reality check for long-pending national healthcare policies and regulatory reforms that must re-invigorate future strategies.

The new vision for healthcare for 2020 and beyond will not just focus on access, quality, and affordability but also on predictive, preventive, and outcome-based care models promoting social and financial inclusion. Social Determinants of Health (SDOH) will emerge has a big theme across progressive health systems to proactively engage the right patients and improve health outcomes to help healthcare organizations meet quality standards. In 2020, consumer-driven models of healthcare will gain more market traction, as they stand to better bridge the gap of what consumers want and what healthcare can deliver.

Continued steps will be taken by retail (Walmart, Costco, Amazon, Ali Health), and consumer tech (Google, Apple, Microsoft, and so on) companies globally; to make further headway (intrude) into vetted healthcare space. In 2020, the convergence of Artificial Intelligence (AI), Blockchain, and the Internet of Things (IoT) will further catalyze the space of innovation adoption and related applications in the healthcare realm. For example, while Blockchain will improve data liquidity to empower AI and analytics vendors/applications to digest a large amount of data, AI can manage Blockchain systems more efficiently than humans.

Research Scope

Every year, the team of futurists, analysts, and consultants at the publisher's Transformational Healthcare Group come together to render a comprehensive analysis to predict the themes, technologies, and global forces that will define the next 12 to 18 months (future) for the healthcare industry.

As a part of this research deliverable, the publisher provides bold perspectives and predictions for the global healthcare market in 2020. The sectors covered include pharmaceuticals and biotech, in-vitro diagnostics, medical technologies, medical imaging, and healthcare IT. The analysis captures sectoral and regional trends and provides predictions for the upcoming year. The study provides guidance on where to find the greatest opportunities for expansion.

Predictions for the global healthcare market in 2020 include:

Key Issues Addressed

Key Topics Covered

1. Executive Summary

2. Revisiting 2019 Predictions

3. Global Healthcare Market Outlook for 2020

4. Key 2020 Healthcare Market Predictions

5. Regional Predictions 2020

6. Sector Outlook 2020

7. Key Conclusions

For more information about this report visit https://www.researchandmarkets.com/r/79rxhs

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

CONTACT: ResearchAndMarkets.comLaura Wood, Senior Press Managerpress@researchandmarkets.comFor E.S.T Office Hours Call 1-917-300-0470For U.S./CAN Toll Free Call 1-800-526-8630For GMT Office Hours Call +353-1-416-8900

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Global Healthcare Market Outlook, 2020: Key Predictions & Growth Opportunities for the $2+ Trillion Industry - Yahoo Finance

Recommendation and review posted by Bethany Smith

The gene therapy company sold 7.6 million shares of stock at a price of $1.05 per share

Genprex Inc () announced an $8 million offering of stock through securities purchase agreements made with influential investors.

The gene therapy company sold 7.6 million shares at a price of $1.05 per share, in accordance with Nasdaq rules governing registered direct offerings.

Genprex, based in Austin, Texas and Cambridge, Massachusetts, plans to use the proceeds to advance its clinical programs in non-small cell lung cancer, as well as for working capital and general corporate purposes.

The company is developing a new approach to treating cancer, including its initial product candidateOncoprex, which is an immunogene therapy for non-small cell lung cancer.

Contact Andrew Kessel at [emailprotected]

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Genprex rakes in $8M in at-the-market stock offering - Proactive Investors USA & Canada

Recommendation and review posted by Bethany Smith

The Gene Therapy and Antisense Drugs Market research report added by Market Study Report, LLC, provides a succinct analysis on the recent market trends. In addition, the report offers a thorough abstract on the statistics, market estimates and revenue forecasts, which further highlights its position in the industry, in tandem with the growth strategies adopted by leading industry players.

The Gene Therapy and Antisense Drugs market study is a well-researched report encompassing a detailed analysis of this industry with respect to certain parameters such as the product capacity as well as the overall market remuneration. The report enumerates details about production and consumption patterns in the business as well, in addition to the current scenario of the Gene Therapy and Antisense Drugs market and the trends that will prevail in this industry.

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What pointers are covered in the Gene Therapy and Antisense Drugs market research study?

The Gene Therapy and Antisense Drugs market report Elucidated with regards to the regional landscape of the industry:

The geographical reach of the Gene Therapy and Antisense Drugs market has been meticulously segmented into United States, China, Europe, Japan, Southeast Asia & India, according to the report.

The research enumerates the consumption market share of every region in minute detail, in conjunction with the production market share and revenue.

Also, the report is inclusive of the growth rate that each region is projected to register over the estimated period.

The Gene Therapy and Antisense Drugs market report Elucidated with regards to the competitive landscape of the industry:

The competitive expanse of this business has been flawlessly categorized into companies such as

Segmentation

On the basis of therapeutic area, the gene therapy and antisense drugs market is segmented into cancer, anemia, rheumatoid arthritis, cardiovascular diseases, HIV/AIDS, cystic fibrosis, diabetes mellitus and obesity, and renal diseases.

By gene transfer method, ex vivo gene transfer and in vivo gene transfer are the segments of the market. The former involves the transfer of cloned genes into cells, i.e., cells are altered outside the body before being implanted into the patient, whereas the latter involves the transfer of cloned genes directly into the patients tissues. The outcome of in vivo gene transfer technology mainly depends on the general efficacy of gene transfer and expression.

Global Gene Therapy and Antisense Drugs Market: Regional Outlook

The global gene therapy and antisense drugs market is segmented into North America, Asia Pacific, Europe, and Rest of the World. Amongst these, North America holds the leading position in the market followed by Europe. The increasing incidence of cancer and other fatal diseases, unhealthy lifestyle practices such as excessive smoking and excessive consumption of high fat content food, and increasing research efforts for treatment against cancer are the major factors driving the gene therapy and antisense drugs market in these regions.

Asia Pacific is expected to emerge as a significant market for gene therapy and antisense drugs. The high population density including a large geriatric population, expeditiously increasing demand for technologically advanced therapeutics, and increasing government support for improved healthcare infrastructure in the region is driving the growth of this regional market. Furthermore, favorable reimbursement policies and tax benefits on newer therapies will further fuel the growth of the Asia Pacific gene therapy and antisense drugs market.

Major Companies Mentioned in Report

Some of the leading companies operating in the global gene therapy and antisense drugs market are GenVec Inc., Avigen Inc., Genome Therapeutics Corp., Tekmira Pharmaceuticals Corporation, Isis Pharmaceuticals, Cell Genesys Inc., and others. These companies are profiled for their key business attributes in the report.

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Exclusive details pertaining to the contribution that every firm has made to the industry have been outlined in the study. Not to mention, a brief gist of the company description has been provided as well.

Substantial information subject to the production patterns of each firm and the area that is catered to, has been elucidated.

The valuation that each company holds, in tandem with the description as well as substantial specifications of the manufactured products have been enumerated in the study as well.

The Gene Therapy and Antisense Drugs market research study conscientiously mentions a separate section that enumerates details with regards to major parameters like the price fads of key raw material and industrial chain analysis, not to mention, details about the suppliers of the raw material. That said, it is pivotal to mention that the Gene Therapy and Antisense Drugs market report also expounds an analysis of the industry distribution chain, further advancing on aspects such as important distributors and the customer pool.

The Gene Therapy and Antisense Drugs market report enumerates information about the industry in terms of market share, market size, revenue forecasts, and regional outlook. The report further illustrates competitive insights of key players in the business vertical followed by an overview of their diverse portfolios and growth strategies.

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Some of the Major Highlights of TOC covers:

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R&D Activities to Fast-track the Growth of the Gene Therapy and Antisense Drugs Market Between 2017 2025 - Fusion Science Academy

Recommendation and review posted by Bethany Smith

This report presents the worldwide In Situ Hybridization market size (value, production and consumption), splits the breakdown (data status 2018 and forecast to 2025), by manufacturers, region, type and application.

This study also analyzes the market status, market share, growth rate, future trends, market drivers, opportunities and challenges, risks and entry barriers, sales channels, distributors and Porters Five Forces Analysis.

The report presents the market competitive landscape and a corresponding detailed analysis of the major vendor/key players in the market.

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Top Companies in the Global In Situ Hybridization Market:

key drivers, restraints, recent trends, and growth opportunities in the global market for in situ hybridization. The market shares, product portfolios, technological developments, and business strategies of the major players are discussed in detail. The report also sheds light on factors such as market size, supply and demand ratio, market attractiveness, and key segments of the in situ hybridization market.

Global In Situ Hybridization Market: Drivers and Restraints

Technological advancements in the field of in situ hybridization, such as development of the cytogenetic technique, is one of the key factors driving the market. The rising prevalence of cancer has necessitated clinical research, which is another major market driver. The introduction of new therapeutic agents has led to a high rate of adoption of companion diagnostics, boosting the overall market expansion. Besides these factors, the growth of the pharmaceutical and biotechnology industry has improved the prospects of the in situ hybridization market worldwide.

Florescence in situ hybridization (FISH) can be used in the detection of genetic abnormalities such as aneuploidy, characteristic gene fusion, or loss of a chromosomal region. It is also useful for research in the fields of gene mapping and identification of genetic aberrations, which are responsible for cancer. As this technique is simple yet effective, FISH will ensure the growth of the global in situ hybridization market.

On the contrary, strict regulatory policies will obstruct the growth of the in situ hybridization market. However, the emergence of molecular cytogenetics will present significant opportunities due to unmet needs in accurate disease diagnosis, rising number of chromosomal disorders, and surge in population.

Global In Situ Hybridization Market: Regional Outlook

On the basis of geography, the global market for in situ hybridization can be segmented into Europe, Asia Pacific, Latin America, North America, and the Middle East and Africa. North America holds a large share in the overall market, with Europe and Asia Pacific also exhibiting promising growth. Extensive research activities in countries such as Canada and the U.S., increased adoption of companion diagnostics, and beneficial government policies have been aiding the in situ hybridization market in North America.

Over the forecast period 2017-2025, Asia Pacific will undergo tremendous growth on account of growing incidence of cancer and its diagnosis, increased healthcare expenditure, and increasing health awareness among people. The presence of international companies in countries such as India and China will further provide an impetus to the market.

Companies Mentioned in the Report

The major companies operating in the market for in situ hybridization include Bio Sb, Inc., Advanced Cell Diagnostics, Inc., Merck KGaA, Abbott Laboratories, Inc., Leica Biosystems Nussloch GmbH, Danaher Corporation, and Agilent Technologies. Several companies are using business strategies such as product enhancement, collaborations, acquisitions, and partnerships with a view to increasing profit.

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The report provides a valuable source of insightful data for business strategists and competitive analysis of In Situ Hybridization Market. It provides the In Situ Hybridization industry overview with growth analysis and futuristic cost, revenue and many other aspects. The research analysts provide an elaborate description of the value chain and its distributor analysis. This Tire In Situ Hybridization study provides comprehensive data which enhances the understanding, scope and application of this report.

Influence of the In Situ Hybridization market report:

-Comprehensive assessment of all opportunities and risk in the In Situ Hybridization market.

In Situ Hybridization market recent innovations and major events.

-Detailed study of business strategies for growth of the In Situ Hybridization market-leading players.

-Conclusive study about the growth plot of In Situ Hybridization market for forthcoming years.

-In-depth understanding of In Situ Hybridization market-particular drivers, constraints and major micro markets.

-Favorable impression inside vital technological and market latest trends striking the In Situ Hybridization market.

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The report has 150 tables and figures browse the report description and TOC:

Table of Contents

1 Study Coverage

1.1 In Situ Hybridization Product

1.2 Key Market Segments in This Study

1.3 Key Manufacturers Covered

1.4 Market by Type

1.4.1 Global In Situ Hybridization Market Size Growth Rate by Type

1.4.2 Hydraulic Dredges

1.4.3 Hopper Dredges

1.4.4 Mechanical Dredges

1.5 Market by Application

1.5.1 Global In Situ Hybridization Market Size Growth Rate by Application

2 Executive Summary

2.1 Global In Situ Hybridization Market Size

2.1.1 Global In Situ Hybridization Revenue 2014-2025

2.1.2 Global In Situ Hybridization Production 2014-2025

2.2 In Situ Hybridization Growth Rate (CAGR) 2019-2025

2.3 Analysis of Competitive Landscape

2.3.1 Manufacturers Market Concentration Ratio (CR5 and HHI)

2.3.2 Key In Situ Hybridization Manufacturers

2.3.2.1 In Situ Hybridization Manufacturing Base Distribution, Headquarters

2.3.2.2 Manufacturers In Situ Hybridization Product Offered

2.3.2.3 Date of Manufacturers Enter into In Situ Hybridization Market

2.4 Key Trends for In Situ Hybridization Markets & Products

3 Market Size by Manufacturers

3.1 In Situ Hybridization Production by Manufacturers

3.1.1 In Situ Hybridization Production by Manufacturers

3.1.2 In Situ Hybridization Production Market Share by Manufacturers

3.2 In Situ Hybridization Revenue by Manufacturers

3.2.1 In Situ Hybridization Revenue by Manufacturers (2019-2025)

3.2.2 In Situ Hybridization Revenue Share by Manufacturers (2019-2025)

3.3 In Situ Hybridization Price by Manufacturers

3.4 Mergers & Acquisitions, Expansion Plans

More Information.

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Global Cake Mix Market Industry: A Latest Research Report to Share Market Insights and Dynamics - Fusion Science Academy

Recommendation and review posted by Bethany Smith

The Global Gene Therapy Market is growing at an exciting pace driven by changing dynamics and risk ecosystem, a study of which forms the crux of the report. The study on the global Gene Therapy market takes a closer look at several regional trends and the emerging regulatory landscape to assess its prospects. The critical assessment of the numerous growth factors and breaks in the global Gene Therapy market offered in the analyses helps in assessing the lucrativeness of its key segments.

Download Free PDF Brochure for Latest Research Study of Gene Therapy Market:: https://www.globalmarketers.biz/report/life-sciences/global-gene-therapy-industry-market-research-report/7517 #request_sample

This Report Covers Leading Companies Associated in Worldwide Market:

UniQure NVBluebird Bio,Celladon,Avalanche Bio,Sangamo,Shanghai Sunway Biotech Co. LtdAdvantagene,Sibiono GeneTech,Spark Therapeutics,Dimension Therapeutics,

Summary of Market: The global Gene Therapy market is valued at xx million US$ in 2019 is expected to touch xx million US$ by the close of 2025, growing at a CAGR of xx% during 2020-2025.

The report emphases on Gene Therapy Market volume and value at Global Level, Regional Level And Company Level. From a global standpoint, this report embodies overall market size by studying historical data and future outlook.

The report is bifurcated into product type, applications, and regions worldwide. The above areas are further bifurcated into country-level data statistics for the below countries.

The key regions and countries covered in this report are:

Please note, the regional and country level data can be altered and provided as per clients custom requirements.

Global Gene Therapy Market Segmentation, By Product Type:

Viral vectorNon-viral vector

Global Gene Therapy Market Segmentation, By Application:Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

Inquire Before Buying: https://www.globalmarketers.biz/report/life-sciences/global-gene-therapy-industry-market-research-report/7517 #inquiry_before_buying

Research objectives:

To study and estimate the market size of Gene Therapy , in terms of value.

To find development and challenges for the global market.

To observe worthwhile expansions including expansions, new services presents in worldwide industry.

To classify and assess the side view of important companies of Global Gene Therapy industry.

Key Questions Answered in the Report:

How is the Gene Therapy market expected to Grow In Terms Of Value during the study period?

What are the Competition Developments and Trends in the Gene Therapy market?

What are the core Macro-Economic and Industry Factors impacting the growth of the Gene Therapy market?

What are the Key Challenges, Opportunities, and Improvements faced by market players in the global Gene Therapy market?

Table of Content

1 Report Outline

1.1 Research Opportunity

1.2 Major Industrialists

1.3 Market Segment by Type

1.4 Market Segment by Application

1.5 Study Objectives

1.6 Years Considered

2 Global Evolution Trends

2.1 Production and Volume Analysis

2.1.1 Global Gene Therapy Production Value 2015-825

2.1.2 Global Gene Therapy Production 2015-2025.

2.1.3 Global Gene Therapy Capacity 2015-2025.

2.1.4 Global Gene Therapy Marketing Pricing and Trends

2.2 Major Producers Growth Rate (CAGR) 2020-2025.

2.2.1 Global Gene Therapy Market Size CAGR of Major Regions

2.2.2 Global Gene Therapy Market Share of Major Regions

2.3 Industry Trends

2.3.1 Market Top Trends

2.3.2 Market Operators

3 Market Share by Industrialists

3.1 Capacity and Production by Industrialists

3.1.1 Global,Gene Therapy Capacity by Industrialists

3.1.2 Global Gene Therapy Production by Industrialists

3.2 Revenue by Industrialists

3.2.1. Gene Therapy Revenue by Industrialists (2015-2020)

3.2.2. Gene Therapy Revenue Share by Industrialists (2015-2020)

3.2.3 Global Gene Therapy Market Concentration Ratio (CR5 and HHI)

3.3. Gene Therapy Price by Industrialists

3.4 Major Industrialists of Gene Therapy Plants/Factories Distribution and Area Served

3.5 Date of Major Industrialists Enter into Gene Therapy Market

3.6 Major Industrialists Gene Therapy Product Offered

3.7 Mergers & Acquisitions, Expansion Plans

4 Market Dimensions by Type

4.1 Production and Production Rate for Each Type

4.2 Global Gene Therapy Production Market Share by Type

4.3 Global Gene Therapy Production Value Market Share by Type

4.4. Gene Therapy Ex-factory Price by Type

5 Market Size by Application

5.1 Overview

5.2 Global Gene Therapy Consumption by Application

6 Production by Regions

6.1 Global Gene Therapy Production (History Data) by Regions 2015-2020.

6.2 Global Gene Therapy Production Value (History Data) by Regions

6.3 North America

6.3.1 North America Production Growth Rate 2015-2020.

6.3.2 North America Production Value Growth Rate 2015-2020.

6.3.3 Major Players in North America

6.3.4 North America Import & Export

6.4 Europe

6.4.1 Europe Production Growth Rate 2015-2020.

6.4.2 Europe Production Value Growth Rate 2015-2020.

6.4.3 Major Players in Europe

6.4.4 Europe Import & Export

6.5 China

6.5.1 China Production Growth Rate 2015-2020.

6.5.2 China Production Value Growth Rate 2015-2020.

6.5.3 Major Players in China

6.5.4 China Import & Export

6.6 Japan

6.6.1 Japan Production Growth Rate 2015-2020.

6.6.2 Japan Production Value Growth Rate 2015-2020.

6.6.3 Major Players in Japan

6.6.4 Japan Import & Export

7. Gene Therapy Consumption by Regions

7.1 Global Gene Therapy Consumption (History Data) by Regions

7.2 North America

7.2.1 North America Consumption by Type

7.2.2 North America Consumption by Application

7.2.3 North America Consumption by Countries

7.2.4 United States

7.2.5 Canada

7.2.6 Mexico

7.3 Europe

7.3.1 Europe Consumption by Type

7.3.2 Europe Consumption by Application

7.3.3 Europe Consumption by Countries

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Gene Therapy Market Is Thriving Worldwide with major players Like: UniQure NV Bluebird Bio, Celladon, Avalanche Bio - Expedition 99

Recommendation and review posted by Bethany Smith

A recent market study published by Marketresearch.biz in its upcoming report outlook titled, Gene Therapy Market: Global Industry Analysis and Astonishing Growth [2020-2029] offers key market insights and emerging trends on the global Gene Therapy market.

The report has offered an exhaustive analysis of the Gene Therapy Market taking into consideration all the crucial aspects like growth factors, constraints, market developments, and future prospects. Market researchers and industry experts have pointed out the key market trends and prospects that may impact the overall Gene Therapy Market growth. This will help players to leverage the opportunities to strengthen their position. Also, the report throws light on the important factors that are contributing to the Gene Therapy Market growth. Additionally, challenges and impeding factors that could hamper the growth of the Gene Therapy Market in the years to come are mentioned in the report.

The Gene Therapy market report includes comprehensive information about the markets major competitors, including various organizations, companies, associations, suppliers and manufacturers competing for production, supply, sales, revenue generation, and after-sales performance expectations. The bargaining power of numerous vendors and buyers have also been included in the research report.

Download Free PDF Brochure with Full Analysis of Key Players:https://marketresearch.biz/report/gene-therapy-market/request-sample

Following Key Players are Analysed in this Report: Novartis, Kite Pharma Inc, GlaxoSmithKline PLC, Spark Therapeutics Inc, Bluebird bio Inc, Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica PLC, NewLink Genetics Corp., Amgen Inc

Market Dynamics:

Set of qualitative informative data that incorporates PORTER Five Forces Model, Macro-Economic factors, PESTEL Analysis, SWOT Analysis, Value Chain Analysis, Regulatory Framework along with Industry Background and Overview.

Worldwide Gene Therapy Research Methodology

Marketresearch.biz presents a detailed picture of the market by way of study, synthesis, and summation of information from various sources. The information thus presented is reliable, comprehensive, and the result of extensive research, both primary and secondary. The analysts have presented the different features of the market with a particular focus on identifying the key business influencers.

Gene Therapy Market Data Break Down is illuminated below by vector type, gene type, application, and region:

By Vector:Viral vectorRetrovirusesLentivirusesAdenovirusesAdeno Associated VirusHerpes Simplex VirusPoxvirusVaccinia VirusNon-viral vectorNaked/Plasmid VectorsGene GunElectroporationLipofection

By Gene Therapy:AntigenCytokineTumor SuppressorSuicideDeficiencyGrowth factorsReceptorsOther

By Application:Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

The report cast light on an extensive analysis based on the most prominent Gene Therapy manufacturers operating in the industry. Activities performed by robust Gene Therapy manufacturers/companies are product development, research, and innovation as well as technology adoptions that are intensifying the competitive intensity and companys ability to offer better product lineup. The companies are also performing strategic acquisitions, ventures, mergers, and partnerships to enlarge their serving area and strengthen their existence worldwide.

The study delivers an exact evaluation of the financial operations of companies covering Gene Therapy sales volume, capital investment, gross margin, profitability, revenue, cash flow, and growth rate. Their manufacturing capacity, production volume, product specifications, import-export activities, production processes, raw material sourcing, and distribution networks are also elaborated in this Gene Therapy report. Companies are also engaged in product launches, promotional activities, and brand developments as part of strategic planning.

How will the report help new companies to plan their investments in the Gene Therapy market?

The Gene Therapy market research report classifies the competitive spectrum of this industry in elaborate detail. The study claims that the competitive reach spans the companies.

The report also mentions about the details such as the overall remuneration, pricing trends, product sales figures, gross margins, etc.

Information about the sales & distribution area alongside the details of the company, such as company overview, product specifications, buyer portfolio, etc., are provided in the Gene Therapy market study.

Inquire/Speak To Expert for Further Detailed Information About Gene Therapy Report :https://marketresearch.biz/report/gene-therapy-market/#inquiry

Regional Outlook: Regional analysis is another important part of the report which is segregated into different sections. One section of the report is entirely dedicated to regional consumption analysis whereas another for regional production analysis. It includes North America, Europe, China, Japan, Southeast Asia, India.

Some of the Major Highlights of TOC covers in Gene Therapy Market Report:

Chapter 1: Methodology & Scope of Gene Therapy Market

Definition and forecast parameters

Methodology and forecast parameters

Data Sources

Chapter 2: Executive Summary of Gene Therapy Market

Business trends

Regional trends

Product trends

End-use trends

Chapter 3: Gene Therapy Industry Insights

Industry segmentation

Industry landscape

Vendor matrix

Technological and innovation landscape

Chapter 4: Gene Therapy Market, By Region

Chapter 5: Company Profile

Business Overview

Financial Data

Product Landscape

Strategic Outlook

SWOT Analysis

And CONTINUE

Analysts with high skill in information gathering and governance use industry strategies to collate and examine data at all stages. Our analysts are trained to combine superior research methodology, modern data collection techniques, subject expertise and years of collective experience to deliver useful and accurate research reports.

We will be happy to attend to your queries regarding the above-market study. Kindly contact us atinquiry@marketresearch.bizto learn more about the market report.

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Gene Therapy Market Segmentation 2020 | Analyzing the Impact Followed by Restraints till 2029 - News Monitoring

Recommendation and review posted by Bethany Smith

Coherent Market Insights has recently announced the addition of a new research report to its repository named, Global Cell and Gene Therapy Market Status and Forecast 20192026, covering top-line subjective and quantitative synopsis data. The market review provides an eccentric tool for analyzing the market in terms of strengths, and weakness, marking opportunities, and supporting strategic and proficient decision-making. The key drivers and restraints affecting the growth of the Cell and Gene Therapy are stated.

A comprehensive analysis of the Cell and Gene Therapy market is presented in this document, along with a brief overview of the segments in the industry. The study presents a feasible estimate of the current market scenario, including the Cell and Gene Therapy market size with regards to the volume and renumeration. The report is a collection of significant data related to the competitive landscape of the industry. It also contains data with regards to several regions that have successfully established its position in the Cell and Gene Therapy market.

Get Free Sample Copy of this Report: https://www.coherentmarketinsights.com/insight/request-sample/2475

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

The report also points out the latest trends in the market and the various opportunities for the market to grow in the near future. Insightful information about the key players such as business market overview, product offerings, and industry revenue segmentation has been provided in the report. It also serves an extensive analysis of different sections and sub-segments which offers market insights toward the historic market scenarios along with future growth and prospect.

Our competitor profiling includes evaluation of distribution channels and products and services offered by and financial performance of companies operating in the global Cell and Gene Therapy market. We also provide Porters Five Forces, PESTLE, and SWOT analysis to assess competitive threat and examine other aspects of the global Cell and Gene Therapy market. The report offers strategic recommendations, competitor benchmarking for performance measurement, and analysis of partnership, merger, and acquisition targets and industry best practices. It also provides analysis of profitability and cost across the industry value chain.

Competitive Rivalry: The Cell and Gene Therapy report incorporates the detailed analysis of the leading organizations and their thought process and what are the methodologies they are adopting to maintain their brand image in this market. The report aides the new bees to understand the level of competition that they need to fight for to strengthen their roots in this competitive market.

Principal Research:

The research team works with industry experts from the Global Cell and Gene Therapy industry including the management organizations, processing organizations, value chain analytics by service providers of the Cell and Gene Therapy market.

Subordinate Research:

In the Secondary research vital information about the Cell and Gene Therapy industries value chain, total pool of key players, and application areas. Market separation is done as per the industrial drifts to the deepest level, terrestrial markets and key developments from both market place and technology-oriented viewpoints.

Get Free PDF Brochure of this Report: https://www.coherentmarketinsights.com/insight/request-pdf/2475

This report forecasts revenue growth at a global, regional & country level, and provides an analysis of the market trends in each of the sub-segments from 2019 to 2026.

North America (USA, Canada, Mexico, etc.)

Asia-Pacific (China, Japan, India, Korea, Australia, Indonesia, Taiwan, Thailand, etc.)

Europe (Germany, UK, France, Italy, Russia, Spain, etc.)

Middle East & Africa (Turkey, Saudi Arabia, Iran, Egypt, Nigeria, UAE, Israel, South Africa, etc.)

South America (Brazil, Argentina, Colombia, Chile, Venezuela, Peru, etc.)

In addition, the Cell and Gene Therapy market research will help solve the following problems:

Ultimately, Cell and Gene Therapy reports provide detailed information and expert analysis on key consumer trends and behaviours in the market, as well as an overview of market data and key brands. Cell and Gene Therapy Market Reports provide all data with easy-to-understand information

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About us:

Coherent Market Insights is a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity.

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Continued here:
Cell and Gene Therapy Market to Exceed Revenues Worth US$ By the End of 2020 2027 - Melanian News

Recommendation and review posted by Bethany Smith

About a decade ago, the ill-fated therapeutic cancer vaccine Provenge was approved, eventually bankrupting its developer Dendreon. Since then, a number of drugmakers have seen similar efforts splutter and fizzle, although the emergence of immunotherapies checkpoint inhibitors and CAR-T drugs offered a glimmer of hope in resuscitating the field. Banking on that promise is PDC*line Pharma, which secured a 20 million injection on Wednesday.

Spun out of the French Blood Bank, the Belgian-French biotech is developing off-the-shelf cancer vaccines that are based on a therapeutic cell line of plasmacytoid dendritic cells, which are sometimes referred to as the Swiss army knife of immune cells due to their diverse range of function. PDC*line Pharmas technology is being developed to be synergistic with checkpoint inhibitors.

Scientists initially hoped that turbocharging the immune system to battle cancer cells would be enough to shrink tumors. But that didnt quite occur since cancer cells possess the ability to put brakes on that immune assault.

In the case of Provenge, data showed the product helped prostate cancer patients live longer but there was no evidence of tumor shrinkage or cancer cell death. Still,Wall Street tagged it with blockbuster expectations. But its adoption was restricted by its complex autologous therapeutic administration, high price tag and changing treatment landscape. Since then, a number of other therapeutic cancer vaccine makers have crashed and burned including Argos Therapeutics and Bavarian Nordic.

A few years ago, the emergence of checkpoint inhibitors which are engineered to unleash the immune system emerged as the perfect partner in crime for the therapeutic cancer vaccine. That is what PDC*line Pharma hopes its vaccine will accomplish.

Its lead product is currently in a Phase I/II study in patients with the most common form of lung cancer. Enrollment is expected to be completed by 2022, chief Eric Halioua told Endpoints News.

Provenges complicated autologous administration, cost of goods, batch-to-batch variability and underwhelming efficacy all contributed to its tepid sales, Halioua suggested, indicating that PDC*line Pharmas plan to incorporate a checkpoint inhibitor into the equation would likely be beneficial.

You need a good vaccine to activate (the) immune system, and you need something to break the defense mechanisms of the tumor. And all the first generations of vaccines were not in these situations.

Although the company is initially testing its off-the-shelf vaccine as a monotherapy, eventually a checkpoint inhibitor will be added to the mix.

But caution is warranted: Efforts to combine therapeutic cancer vaccines and checkpoint inhibitors have also met with failure. Last month, French biotech Transgene abandoned its therapeutic lung cancer vaccine after mid-stage data showed that when tested alongside Bristol-Myers Opdivo, the combination did not significantly shrink tumors.

Altogether PDC*line Pharma which recently signed a 108 million licensing deal (plus royalties) for its lung cancer vaccine in South Korea and other Asian regions has raised more than 30 million since its inception in 2014.

In this latest round of Series B funding, PDC*line Pharma lured five new investors: Korean Investment Partners, as well as two South Korean funds Shinhan-Cognitive Start-up Fund and UTC 2019 BIOVENTUREFUND in addition to two Belgian funds, SRIW (The Regional Investment Company of Wallonia) and Sambrinvest (the investment fund of Charleroi).

The new investors joined existing investors SFPI-FPIM, the Belgian Federal Holding and Investment Company, Noshaq Group, the Financire Spin-off Luxembourgeoise/INVESTSUD Group, among other undisclosed names.

Read more:
French-Belgian biotech banks 20M to break ground in blood-splattered field of therapeutic cancer vaccines - Endpoints News

Recommendation and review posted by Bethany Smith

Experts at Newcastle University have shown that the rate of kidney disease in people with Joubert syndrome is determined by the genetic makeup of the individual and each patient may respond differently to the treatment.

Joubert syndrome is a complex disorder, affecting approximately one in 80,000 newborns, causing varying degrees of physical, mental and sometimes visual impairments. It is often associated with severe kidney disease that requires dialysis and ultimately transplantation.

The study, published online in the Proceedings of the National Academy of Sciences, is the first time that an explanation has been given for the difference of disease progression in Joubert syndrome patients.

Significant breakthrough

The Newcastle research has identified a second gene called BSND a 'modifier gene' which determines the severity of kidney disease in patients with CEP290 mutations of Joubert syndrome.

It has been assumed that these modifier genes exist, but they have never been found before in rare genetic conditions until now.

Professor John Sayer and Dr Colin Miles, from the Translational and Clinical Research Institute, Newcastle University, led the Medical Research Council-funded research.

Professor Sayer said: "We have shown, using mouse and human DNA samples, that BSND is a modifier gene for the severity of kidney disease in Joubert syndrome.

"This is the first time that a modifier gene for inherited kidney disease has been identified, and this information will improve diagnoses and will be used to develop therapies to reduce the severity of kidney disease in affected patients.

"Our research is a major step forward and, in the future, we may be able to offer a therapy that switches on the protective modifier gene and reduces the development of genetic kidney disease.

"This work paves the way towards personalised therapies in patients with inherited kidney disease."

The international study used mouse models and DNA samples from patients with Joubert syndrome to progress the research.

Scientists used mouse models of disease and genetic manipulation to see how the kidney disease responded to modifier gene manipulation, cross-referenced with DNA sequencing data from patients around the world to prove the modifier gene was relevant in humans.

Challenging disease

Professor Sayer, a Consultant Nephrologist at Newcastle Hospitals NHS Foundation Trust, said: "The treatment of genetic kidney disease is challenging, as this requires both the correction of the underlying gene defect and the delivery of the treatment.

"We have shown that the kidney disease in a mouse can be dramatically changed by switching on or off a modifier gene.

"This will mean that we can use this information to carry out treatments, including genetic therapies, to lessen the effects of inherited kidney diseases, such as Joubert syndrome.

"We are testing these treatments further in our model systems before we move into patient studies."

Within the next three years, research will start to test the treatment of patients with modifier genes in the hope of developing personalized treatment plans.

Patient story

Siblings Emma, 11, and Ben Buckley, eight, have Joubert syndrome and both developed kidney failure before the age of eight.

They were diagnosed with Joubert syndrome from a few months of age and both have required dialysis and a kidney transplant.

They suffer from a range of medical issues due to Joubert syndrome, including visual impairment, communication problems, and developmental delay.

The two children, of Whitley Bay, North Tyneside, have been instrumental in helping further the research over the years, allowing the Newcastle scientists to study the mutation in detail.

Parents Leanne and Michael say they welcome the findings of the Newcastle University-led study as it will help to give patients a chance of preventing kidney failure in the future.

Leanne said: "It is very important that research is done into Joubert syndrome and the linked kidney damage, as this will hopefully prevent patients in the future needing a kidney transplant.

"All throughout Ben and Emma's lives, they have lived with the effects of Joubert syndrome and scientists found they had a problem with the CEP290 gene.

"Both Ben and Emma have needed dialysis and kidney transplants because of their kidney problems and I would like to hope this research will help prevent kidney failure for other affected children.

"We were happy for Ben and Emma to provide samples for the study as anything that helps further understanding into the condition is well worth doing, so it's great to see the study's positive results."

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Discovery of gene that modifies severity of inherited kidney disease - Devdiscourse

Recommendation and review posted by Bethany Smith

Researchers at theUniversity of Maryland School of Medicine (UMSOM) developed a new nanoparticle drug formulation that targets a specific receptor on cancer cells and appears to be more effective than a standard nanoparticle therapy currently on the market to treat metastatic breast cancer, according to a study published today in the journal Science Advances. The new DART (decreased, non-specific adhesivity, receptor-targeted) nanoparticles bypass healthy cells and tissues and bind to tumor cells, dispersing evenly throughout the tumor while releasing the chemotherapy drug paclitaxel.

The marketed drug Abraxane, a nanoformulation containing paclitaxel that is currently used to treat women with aggressive breast cancer, is an effective agent, but it was not designed to selectively deliver paclitaxel to only the cancerous cells within the body, said study corresponding co-author Jeffrey Winkles, PhD, a professor of surgery at UMSOM. Our DART nanoparticle specifically targets the Fn14 receptor found abundantly on breast cancer cells; it uses this receptor to gain entry through the plasma membrane and deliver the drug to destroy the cancer. Winkles group discovered the Fn14 receptor and described its potential as a target for new therapeutics more than a decade ago.

For this study, UMSOM researchers engineered and tested a new therapeutic nanoparticle platform to deliver the drug paclitaxel to treat triple-negative breast cancer. About one in five women with breast cancer have this type of aggressive tumor, which is particularly difficult to treat; these cancers lack receptors commonly expressed by most breast cancer cells, like hormone receptors, for which effective drugs have been designed. But many triple-negative breast cancers express high levels of Fn14; indeed, most solid tumor types, including lung, prostate, and colorectal cancer, overexpress this cell surface receptor.

After much initial hype and some disappointments in the field, nanoparticle delivery systems for cancer treatment are starting to show real promise for patients, said study co-author Graeme Woodworth, MD, professor and interim chair in the Department of Neurosurgery at UMSOM. We established a proof-of-concept with this study, outlining the design of an optimized nanoparticle delivery system that balances the specific binding to cancer cells while minimizing non-specific, off-target binding to other cells in a very fine-tuned way.

To accomplish this, the research team attached a monoclonal antibody called ITEM 4 to the surface of the nanoparticle because it specifically binds to Fn14, providing a key to unlock entry into the cancer cell. The surface of the nanoparticles also was coated with polyethylene glycol to keep them circulating through the bloodstream and lymph system until they reached the tumor and to prevent them from being quickly flushed out of the body.

Many drug delivery carriers exhibit non-specific binding to healthy cells and tissues in addition to the diseased cells they are targeting, which often leads to unintended side effects or toxicities, said study corresponding co-author Anthony Kim, PhD, associate professor of neurosurgery and pharmacology at UMSOM. This DART nanoparticle platform has unique capabilities to improve therapeutic delivery to difficult-to-treat locations within the body while also allowing us to potentially increase the maximum tolerated dose of the encapsulated drug without increasing side effects to patients.

The researchers filled their optimized DART nanoparticle formulation with paclitaxel and tested it against Abraxane (the marketed nanoparticle that also contains paclitaxel) in animals with triple-negative breast cancer tumors. In one set of experiments, the nanoparticles were delivered to mice harboring breast tumors grown above the natural breast region. They found the DART formulation led to a significantly increased median overall survival (68 days) compared to Abraxane treatment (45 days). They also saw a clear benefit to using the DART nanoparticles when they compared the treatments again in animals that harbored breast tumors implanted in the brain (akin to a metastatic brain tumor).

This is a compelling finding and significant step forward in the use of nanoparticles to treat cancer, said UMSOM Dean E. Albert Reece, MD, PhD, MBA, the John Z. and Akiko K. Bowers Distinguished Professor and University of Maryland, Baltimore executive vice president for medical affairs. It fits squarely with our School of Medicines mission to advance the field of potentially lifesaving therapies for patients with the most difficult to treat cancers.

The UMSOM researchers are members of the University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer Center. A former PhD student, Jimena Dancy, PhD, and a former postdoctoral fellow, Aniket Wadajkar, PhD, are listed as co-first authors on the publication. Researchers from the Translational Genomics Research Institute in Phoenix, Ariz., and the Mayo Clinic Arizona in Scottsdale, Ariz., also contributed to the work.

This study was primarily funded by the National Institutes of Health.

Future research includes testing the DART therapy in other cancer types, including an aggressive form of brain cancer called glioblastoma and developing a similar version of the nanoparticle designed to work specifically in humans. This would involve using a humanized antibody on the surface of the nanoparticle and scaling up the formulation. The researchers recently received a grant from the TEDCO Maryland Innovation Initiative Commercialization Program to move forward with efforts to adapt their nanoparticle system and eventually test the treatment in cancer patients.

Read this article:
Nanoparticle Therapy Shows Promise With Breast Cancer - UMB News

Recommendation and review posted by Bethany Smith

Endometriosis is a painful condition where the lining of the uterus grows on other parts of the organ such as the fallopian tubes. The new study, published in eLife, shows initial results from treating endometriosis in mice with cannabinoids suggesting they can alleviate symptoms of the disease.

The researchers say this new finding will pave the way for further clinical research.

The findings have led to the start of a clinical trial in collaboration with the Gynecology Service of the Hospital Clinic of Barcelona, Spain. The trial will evaluate the possible benefits of the naturally occurring cannabinoid 9-tetrahydrocannabinol (THC; the main psychoactive constituent of the cannabis plant) in women with endometriosis.

Endometriosis is a common, chronic and painful disease caused when the lining of the womb the endometrium grows outside of the womb cavity. These growths affect reproductive organs and can cause pain, infertility, anxiety, depression and result in a considerable impact on quality of life. Treatment options include surgery or hormone therapy, but these are not always effective and often have significant side-effects.

Rafael Maldonado, Professor at the University Pompeu Fabra of Barcelona, Spain, said: With a lack of effective treatments, women with endometriosis usually rely on self-management strategies like dietary changes or exercise. Although cannabis comes with a large number of potential side effects, its medicinal properties could provide pain relief in endometriosis and other conditions.

Since medical THC is available in some countries, the findings of our study may be of interest for gynecologists and pain specialists who manage the treatment of women with endometrial pain.

The team studied mice with endometrial implants in their pelvis to mimic endometriosis in humans. Those with the implants were more sensitive to pain in their pelvis that can also be associated with emotional and cognitive alterations similar to symptoms seen in some women with endometriosis.

The team next found that mice with endometriosis had similar anxiety-like symptoms experienced by some women with the condition. This was measured by the amount of time the animals spent in open areas of a maze, as those with higher anxiety levels tend not to explore too far. However, their experiments could not reveal whether THC had any significant effects in treating this anxiety.

As endometriosis can be known to impair cognitive function in some women, the team also studied memory performance in the mice. They provided the animals with two identical objects and allowed them to become familiar with them. They then replaced one of the objects and timed how long the mice spent exploring the new versus familiar object, to give an indication of what the animals remembered.

The team found that memory was impaired in the mice with endometriosis compared with those that did not have the condition. However, mice treated with THC did not show this impairment, suggesting that THC may have a protective effect.

Finally, the team studied the effects of THC on the endometrium inside and outside of the womb, and found that mice with endometriosis treated with THC for 32 days had smaller endometrial growths.

First author Alejandra Escudero-Lara, a PhD student at the University Pompeu Fabra of Barcelona, said: Together, our findings show that THC limits the development and symptoms of endometriosis in an experimental model, and highlight the interest of conducting further research to ensure the safety and beneficial effects of this treatment in women with endometriosis.

Continued here:
Endometriosis and cannabis: THC improves symptoms - Health Europa

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Stubborn belly fat is more than just an annoyanceit can be dangerous. That's because stomach fat is visceral and surrounds vital organs such as the pancreas and intestines, says Rachel Stahl, R.D. at Weill Cornell Medicine.

It's also biologically active, meaning this type of fat changes the normal balance of hormones and can cause metabolic problems.

"They are not just standard cells storing fat," Stahl tells Men's Health.

Excess belly fat could also be a sign that you have too much fat surrounding your heart, liver, and kidneys, says endocrinologist Dr. Rasa Kazlauskaite, M.D. at Rush University Medical Center in Chicago, IL. When this happens "You are that much closer to developing diabetes," Kazlauskaite tells Men's Health.

So how much belly fat is too much?

Guys who have a waist circumference greater than 40 inches are at a higher risk of diabetes and heart disease, according to the National Institute of Health.

Measure around the middle of your waist, just above the hipbones, using a tape measure, advises the NIH.

Although losing belly fat isn't easy, there are ways to reduce your waistline:

Unfortunately, you can't force fat reduction in one area of your body, says Dr. W. Scott Butsch, M.D., and Director of Obesity Medicine at the Cleveland Clinic.

Instead, you'll want to focus on reducing overall calories, Butsch tells Men's Health.

"Weight loss in general should lead to a decrease in belly fat," he says.

Men's Health

Brain scans show that protein lowers activity in regions of the brain that stimulate food cravings. Protein also increases hormones that make you feel satiated, so you feel fuller for longer periods of time.

They're nutrient dense and provide plenty of fiber, meaning you'll get full on fewer calories. Load up half of your plate with vegetables, or begin every meal with a salad.

Kazlauskaite says guys who want to reduce belly fat should watch how much alcohol they consume. Aside from the obviousempty caloriesalcohol needs to be metabolized by the body, which briefly stops fat burning, she says.

Losing weight shouldn't be your only motivation to hit the gym. And exercise doesn't burn as many calories as you may believe. However, paired with a healthy overall diet, a regular workout routine can help you maintain a calorie deficit.

Clinically, Butsch says many of his patients notice a decrease in belly fat when they incorporate strength training. In fact, obese adolescents who incorporated both aerobic and strength training into their workouts lost the highest amounts of visceral fat, according to a 2014 study published in the Journal of Sports Sciences.

Sugar from juice and sodas are rapidly absorbed into the bloodstream, which spikes insulin, says Kazlauskaite. There's no evidence that these beverages actually cause obesity. However, studies show that frequently drinking sugar-sweetened beverages is linked to weight gain, obesity, and type 2 diabetes, according to the Centers for Disease Control & Prevention.

Eating an apple is different from a drinking a glass of apple juice, explains Kazlauskaite.

That's because you consume more nutrients like fiber when you consume the food in its natural form, she says. Plus, the process of chewing and consuming the food more slowly can help keep you full. "When you eat an apple, you eat slower," she says. "Your body has more time to digest it."

Cortisol, known as the stress hormone, is linked to a higher percent of belly fat and weight gain. In fact, people who have high levels of cortisol for long periods of time are more likely to develop abdominal obesity, according to a 2018 review of studies published in Current Obesity Reports.

Butsch says simple activities like yoga, meditation, or simply avoiding conflict can help keep your waistline trim.

"I think even just taking the higher road in an argument [helps]," he tells Men's Health.

Read the original:
Get Rid of Stubborn Belly Fat With 9 Expert Backed Tips - Men's Health

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BY Mary E. Burman

The table is loaded with food and the conversation is freewheeling with lots of laughter. Suddenly I remember that I have metastatic breast cancer. I'm feeling generally good with few side effects from my current treatment that hopefully will keep the rogue cells in my bones. At times like this, I can forget that I have metastatic breast cancer which will most likely cause my death. When that will happen is unknowable, and uncertainty is now a constant part of my life. Of course, all of us face uncertainty.

As the saying goes, anybody can walk out the door, get hit by a bus and be dead in seconds. However, as someone with a metastatic disease said, "The only thing I can think of is that I'm going to die. And that's the one certainty that I have, and I know people say () you could cross a street and be run over by a bus. But you live your life not thinking about it. I have to deal with it. My bus is here. All the time. And my bus is outside the door every morning and uncertainty is always with me.

For me, uncertainty manifests itself in several ways. There is a lot of ambiguity about my breast cancer which is "atypical" starting out as hormone positive but is now probably triple negative. It is not as aggressive as I was initially diagnosed with breast cancer 10 years ago. That makes the future unpredictable since most studies don't capture the outcomes for someone like me who is a healthy 60-year-old with an atypical cancer. While metastatic breast cancer is unfamiliar to me, my family has experienced a rare form of ataxia that has resulted in the death of several siblings for most of my adult life I've wondered whether I'd get it. Returning to the bus metaphor, while I was thinking Bus #9 (the ataxia bus) would hit me, it turned out to be Bus #3 (the breast cancer bus) resulting in a new and different uncertainty.

The feeling of uncertainty waxes and wanes and is stressful, fatiguing, intrusive, unending and isolating. Ultimately, uncertainty in metastatic breast cancer is not something you get past. It's always there waiting for the results of tests, figuring out the best treatment, wondering about prognosis and feeling anxious about costs of care. However, paradoxically, uncertainty leaves the door open for hope. Persons with metastatic breast cancer live in two worlds that of uncertainty and that of hope. My future is not written in stone because there is no way of knowing if I will live 2 years or 15 years. Consequently, that leaves space for envisioning the future, e.g., hope for time with my family, hope for being able to accomplish some things, and hope for new treatments.

Simultaneously living with uncertainty while embracing hope is not easy but it can be done. Arthur Brooks explores career transitions in the Atlantic magazine in an article entitled "Your professional decline is coming (much) sooner than you think: Here's how to make the most of it". While not about living with metastatic breast cancer, it a useful framework for living with uncertainty.

He promotes four different strategies for living well. First, jump which means focusing on what is most important in our personal and professional lives and walking away what isn't important. For me that meant stepping out of my administrative roles at work and downsizing to a much smaller and more manageable house and yard. Second, we should find ways to serve and help others. For me, I have renewed my volunteer work for a nonprofit clinic in my community, which provides me much satisfaction. Third, worship by exploring our own spiritual self and I've found great joy in reading, thinking and exploring new realms of spirituality. Finally, Brooks advocates that we connect dedicating time to meaningful and purposeful relationships.

Connecting and reconnecting with others who are comfortable with metastatic breast cancer has been extremely beneficial to me. Ultimately, the bus doesn't leave my street, but it doesn't have to impact my daily life which can be filled with hope, meaningful activities, and relationships despite the uncertainty.

Read the rest here:
Buses and Uncertainty - Curetoday.com

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Some people watch what they eat. Paige and Tanner Szczesekhave to watch when they eat.

The two children, ages 6 and 2, from Cheektowaga, have Type 1 diabetes. Like others with their condition, they must take extra insulin before meal and snacks. It takes time to work. Eat too soon, and blood sugar can climb dangerously high a constant worry for their mother, Ashley.

My biggest goal is that my children can just feel like children, she said.

Thanks to a new drug developed with an assist in Western New York, they have more of a chance.

The Food and Drug Administration last month approved of a drug that brings researchers a step closer to developing an artificial pancreas that will provide fast-acting insulin in proper amounts at just the right time for those with diabetes.

Fiasp, made by Novo Nordisk, starts working in 2 minutes, hits full force within 10 minutes and even can be taken shortly after someone withdiabetesstarts eating.

Previous fast-acting insulins took at least two or three times as long to do that job.

The newest drug has been available to adults since 2017, but the Food and Drug Administration wanted testing on children before making it available to them. More than 700 children in 17 countries participated in the clinical trial, including five children ages 13 to 17 who have been patients in the diabetes centers at UBMD Pediatrics and next door at Oishei Childrens Hospital on the Buffalo Niagara Medical Campus.

This was a big commitment for the families, said Dr. Kathleen E. Bethin, a clinical professor in the Department of Pediatrics in the Jacobs School of Medicine and Biomedical Sciences at the University at Buffalo, and a physician at both diabetes centers. The kids were in the study for almost a year. There were a lot of extra blood draws and more blood sugar checks than are typically required.

Fiasp brings diabetes researchers closer to mimicking a closed-loop system that uses a glucose monitor, continuous insulin infusion pump and other technologies to allow people whose bodies dont make insulin to live like those whose do.

The new fast-acting insulin drug Fiasp boosts the prospects for a closed-loop insulin system often referred to as "an artificial pancreas," diabetes researchers in Buffalo and elsewhere say. (John Hickey/Buffalo News)

The goal in the diabetes field is to develop insulin analogs that behave more like natural insulin, which is rapid on, rapid off, meaning its quickly released, then quickly dissipates, said Dr. Lucy D. Mastrandrea, chief of the Division of Endocrinology/Diabetes at UBMD Pediatrics and medical director for the Oishei Childrens Hospital Diabetes Center. Part of the reason this drug was developed was to have a better timeline of action thats closer to natural insulin.

The body breaks down carbohydrates into blood sugar to use for energy. Insulin is a hormone needed to bring glucose from the bloodstream into human cells. When blood sugar gets too low, the process breaks down and can lead to learning challenges, seizures, loss of consciousness and death. When it spikes, especially often and over time, complications include limb amputation, heart and kidney disease, and stroke.

For those without diabetes, the pancreas, liver and other organs work together to automatically and seamlessly produce insulin and adjust levels as needed.

Type 2 diabetes is diagnosed when the body doesnt use insulin properly. It often can be managed through a combination of healthy eating, regular exercise and oral medications. Sometimes, insulin also is needed.

Those with Type 1 diabetes produce no insulin. They need to inject manufactured basal insulin to maintain levels throughout each day, load carbs or take medication when blood sugar levels get too low, and add fast-acting insulin to lower them when blood sugar levels climb.

Nearly 18,000 new cases of Type 1 diabetes are diagnosed each year. The majority of children have Type 1, while the majority of those diagnosed in adulthood have Type 2.

Our goal is to keep them in as best control as possible during their childhood years, so that they're not running the risk of dealing with complications when they're in their 20s and 30s, Mastrandrea said.

Drug-maker Eli Lilly engineered the first human-derived insulin, Humulin, in 1981. The company in 1996 developed a faster-acting insulin, Humalog, which is still routinely used. NovoLog and Apidra are among other brands that can lower blood sugar within 20 to 30 minutes after they are injected through a needle or an insulin pump, said Dr. Paresh Dandona, a leading international diabetes researcher, head of the Western New York Center of Diabetes-Endocrinology in Amherst and distinguished professor and chief of endocrinology, diabetes and metabolism in the UB medical school.

Administering the drugs takes planning and guesswork because eating, exercise, stress, illness and other factors affect blood sugar levels. That means those with diabetes need to predict related dips and spikes well in advance. Mealtimes generally are the most challenging because they can spark pronounced spikes.

The food hits you a lot faster than the insulin does, said Szczesek.

Fiasp changes the equation. The newer formulation of NovoLog includes niacinamide (vitamin B3) to boost the speed of absorption.

There still is a lag, but it's the best thing we have, said Dandona, whose clinic helped with adult trials several years ago and who has prescribed the drug to some of his patients during the last two years, with good results.

Paige Szczesek, 6, of Cheektowaga, looks at her personal diabetes manager, which helps her and others more closely track her blood glucose levels and insulin use. (John Hickey/Buffalo News)

Paige and Tanner Szczesek are on the front end of the learning curve when it comes to keeping a proper balance. Their father, Shane, also was diagnosed as a child with Type 1 diabetes.

Greater speed is a godsend for the children, each of whom has a continuous glucose monitor and insulin pump to help control their blood sugar. Paige also has a personal diabetes manager, as part of her pump, that helps her family determine when she needs more insulin and how much. Sweet treats are always on hand for times when their blood sugar drops.

When she needs more fast-acting insulin, someone needs to decide when to administer it, then see how it's working. Ashley Szczesek uses a smartphone to keep tabs on blood glucose levels for both children. She teams up with Paiges school nurse and teachers to address shortfalls and spikes. There are phone calls or text messages every time blood sugar readings warrant, as well as reports about when and what the first-grader has eaten.

I can't just send my kid into school and say, I'll see you at the end of the day,' Ashley Szczesek said. I have an alarm set on my phone for when they're high or they're low. On top of that, especially during the night, I'll normally set several alarms to get up and check their blood sugars. If they're low, I go wake them up and give them something to bring up their blood sugar. If they're high enough, I give them some extra insulin. Between us as parents and the children, there's a lot of sleep loss."

Paige started using Fiasp last year after the clinical trial ended. Mastrandrea prescribed it off-label. Her brother started taking it a few weeks ago.

Paiges A1c level has dropped by 1 percent, to about 7 percent, higher than those without diabetes but in a good range for someone Paiges age with the condition.

Fiasp can ease the diabetes burden, but not erase it.

Those without adequate health insurance may be unable to cover higher co-pays or other out-of-pocket costs. Some people have not wanted to switch for that reason, Mastrandrea said.

Meanwhile, researchers continue to pursue a biologic cure, as well as an insulin pump that works with a continuous glucose monitor to deliver insulin on a minute-by-minute basis as needed.

In order to do that really well, Mastrandrea said, you want to have insulins that are faster-acting, absorb better and behave the way my pancreas does. Fiasp is in that category.

Some of Dandonas patients already have the most advanced insulin pump, the Medtronic 670G, though the device can be complicated for most adults to use, let alone children, he said, and still needs agents to more quickly bring blood sugars into the balanced range.

Still, for those in the field and for families like the Szczeseks, recent progress has been nothing short of remarkable.

Early prototypes of closed-looped models like the 670G once weighed two to three times that of adult patients, Dandona said, and now we have a tiny device doing the same thing.

Read more here:
Breakthrough diabetes insulin drug developed with help from Buffalo - Buffalo News

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