Investigating the Human Intestinal Mucus Barrier Up-close and Personal – Technology Networks

We have a mutualistic but complicated relationship with the collection of microbes in our gut, known as the intestinal microbiome. This complex community of bacteria breaks down different food components, and releases nutrients such as vitamins and a plethora of other factors that control functions in tissues way beyond the intestinal tract. However, the sheer numbers of microbes also present a threat as they can trigger inflammation, which is thought to be at the root of many intestinal diseases, including inflammatory bowel disease, radiation-induced intestinal injury, and some cancers.

To allow the uptake of beneficial substances from the gut lumen, and at the same time prevent gut microbes from contacting the intestinal epithelial tissue surface, specialized cells called goblet cells continuously produce mucus, the slimy goo-like substance that coats the entire intestinal surface. Mucus thus far has been notoriously difficult to study: its structure quickly disintegrates in surgically removed sections of the gut, the system most often used to study mucus, and no in vitro culture system has been able to reconstitute an in vivo-like mucus layer with the natural structure seen in living intestine outside the human body. Adding to these difficulties, mucus also differs between humans and other species, different sections of the intestinal tract, and even different individuals.

Now, focusing on the large intestine or colon which houses the greatest number of commensal microbes and has the thickest mucus layer, a team of tissue engineers at Harvards Wyss Institute for Biologically Inspired Engineering has developed a colon-on-a-chip (Colon Chip) microfluidic culture device lined by patient-derived colon cells that spontaneously accumulates a mucus layer with the thickness, bi-layered structure, and barrier functions typically found in normal human colon. The mucosal surface in their model also responds to the inflammatory mediator prostaglandin E2 (PGE2) by mounting a rapid swelling response. Their findings are published in Cellular and Molecular Gastroenterology and Hepatology.

Our approach provides researchers with the opportunity to find answers to questions about normal and disease-associated mucus biology, such as its contributions to intestinal inflammatory diseases and cancers, and complex host-microbiome interactions, said Founding Director Donald Ingber, M.D., Ph.D., who is the senior investigator on the study. Importantly, we use patient-derived cells to line these devices and so this represents an entirely new approach for personalized medicine where it can be possible to study how mucus functions or dysfunctions in a particular patient, and to tailor therapy accordingly.

Ingber is also the Judah Folkman Professor of Vascular Biology at Harvard Medical School and the Vascular Biology Program at Boston Childrens Hospital, as well as Professor of Bioengineering at Harvards John A. Paulson School of Engineering and Applied Sciences. His team is part of a multi-institutional collaboration supported by a Cancer Research UK Grand Challenge grant in which his Wyss team investigates how inflammation-related changes contribute to formation of cancers, including colon cancers. The Grand Challenge is an ambitious international cancer research initiative, supporting world-leading teams of scientists to take on some of the toughest challenges in cancer, and giving them the freedom to try novel approaches at scale.

The teams approach starts out with patient-derived colon cells from colon resections and endoscopic biopsies that are first grown as organoids, tiny organized balls of colon tissue that contain mainly epithelial stem cells. After fragmenting the organoids, their cells are used to populate the upper of a two parallel channels of a microfluidic chip that are separated by a porous membrane. Simply by perfusing the channels continuously with nutrient medium, the colon stem cells grow into a continuous sheet and form highly functional goblet cells that secrete mucus.

Growing the cells on-chip under flow results in about 15% of epithelial cells spontaneously differentiating into goblet cells. Distributed throughout the epithelium, these produce an in vivo-like mucus layer, said first-author Alexandra Sontheimer-Phelps, a graduate student from the University of Freiburg, Germany, working in Ingbers group. At the same time, other epithelial cells that keep dividing also replenish the goblet cell population just like in living colon, which means that the chip can be maintained in steady-state conditions for more than two weeks, which makes it highly useful for longer-term studies.

The Wyss team showed that the colon epithelium in the chip is fully polarized with distinct markers restricted to its lumen-exposed, mucus-secreting side and its opposite membrane-binding side. Its goblet cells secrete the major mucus protein mucin 2 (MUC2), which when linked to complex chains of sugar molecules, assembles into multi-molecular network or gel that takes up water. Our approach actually produces the bi-layered structure of normal colon mucus with an inner dense layer that we show is impenetrable to bacteria-mimicking particles flowed through the intestinal channel, and a more loose outer layer that allows particles to enter. This has never been accomplished before in vitro, said Sontheimer-Phelps.

To investigate the functionality of the mucus, she and her co-workers exposed the chip to the inflammatory mediator PGE2. The mucus underwent rapid swelling within minutes and independent of any new mucus secretion, and this process of mucus accumulation can be visualized in living cultures by viewing the chips from the side with dark field illumination. This dynamic response could be blocked by inhibiting one particular ion channel, which pumps ions into the colon epithelium and passively allow water molecules to follow and apparently, this drives mucus swelling when stimulated by signals such as PGE2.

Mucus has long been thought to be a passive, host barrier, but it is becoming increasingly clear that microbial species affect its structure and function in addition to feeding on its carbohydrates as an energy source. Our in vitro system brings us one step closer to figuring out how individual bacterial species and more complex microbial communities can affect mucus and vice versa, as well as how this complex interplay impacts development of intestinal diseases. We also now have a testbed to discover new therapeutic drug and probiotic strategies that might prevent or reverse these diseases said Ingber.

Reference:Sontheimer-Phelps, A., Chou, D. B., Tovaglieri, A., Ferrante, T. C., Duckworth, T., Fadel, C., Ingber, D. E. (2019). Human colon-on-a-chip enables continuous in vitro analysis of colon mucus layer accumulation and physiology. Cellular and Molecular Gastroenterology and Hepatology. https://doi.org/10.1016/j.jcmgh.2019.11.008

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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Investigating the Human Intestinal Mucus Barrier Up-close and Personal - Technology Networks

Medicine of the future: cell technologies in Ukraine – Interfax-Ukraine

How to score a decisive penalty to the disease? A football-player Andrii Shevchenko tells own experience of stem cell treatment

December 3rd, the Institute of Cell Therapy will present in Kiev the first results of the placental stem cells clinical trials in Ukraine in a partnership with the Institute of Traumatology and Orthopedics of NAMS of Ukraine and Kiev Clinical Hospital 6. This time the specialists will focus attention on such prevalent diseases as knee joints arthrosis and joints injuries, in particular in professional athletes. Andrii Shevchenko, a legendary football-player and trainer of the national team of Ukraine will participate in the press-conference.

Cell therapy is an innovative, extremely promising method of diseases and injuries treatment, that allows to restore damaged tissues of the body with the help of the new cells, the stem cells. Stem cells have a unique capability to transform into all types of tissues and cells of the body, in particular the cells of blood, liver, myocardium, cartilaginous or nervous tissue. Courtesy of this they restore damaged organs and their functions.

Knee arthrosis is called the disease of the piano players and athletes. The most frequently this disease affects people, whose body is exposed to the extreme loadings, in particular athletes. The females and males, aged over 50, are also in the risk group, from which almost one third is diagnosed the arthrosis of knee joints. This disease is accompanied by the severe pain, limitations at walking and sometimes even by the total inability to move.

The result of the clinical trials, conducted by the Institute of Cell Therapy in the partnership with the Institute of Traumatology and Orthopedics of NAMS of Ukraine and Kiev Clinical Hospital 6 should become the opportunity to use cell therapy for knee arthrosis treatment, improve of the life quality of the patients, suffering from this pathology as well as to avoid or postpone the surgery on knee joint replacement for the artificial one. Stem cells are used worldwide for the treatment of approximately 100 severe diseases, in some disorders this is the only effective method of therapy.

Institute of Cell Therapy is a hightechnological medical institution, specializing on research, medical services and has the own Centre of Science with a Laboratory for Placenta Stem Cells. The Institute already has the experience of clinical trials performance on cell and tissue drugs, after completion of which the Ministry of Health of Ukraine issued a permission for the use of the tested technology of manufacturing and application of cell/tissue preparations, in particular for the treatment of pancreonecrosis and disorders of lower limbs peripheral arteries with the umbilical cord blood stem cells and autologous adipose tissue.

Today 4 clinical trials on the use of cell and tissue therapy methods are on the final stage and other 4 trials are going on.

The press-conference, devoted to the innovative approach in the therapy of knee joint using placenta stem cells will take place on December 3rd at 11 am at the address: Medical campus, Liubomyr Husar (Kosmonaut Komarov avenue, 3, Institute of Cell Therapy.

Participants of the press-conference are:

1. Mykola Sokolov, MD, PhD, Chief Doctor of the Institute of Cell Therapy, leading specialist in cell technologies.

2. Volodymyr Shablii, PhD, Deputy CEO of the Stem Cells Bank, Chief of the Laboratory for Placenta Stem Cells of the Institute of Cell Therapy. Member of the International Placenta Stem Cell Society.

3. Ievgen Golyuk, MD, PhD, Chief of the Scientific and Practical Centre of Tissue and Cell Therapy of the state institution Institute of Traumatology and Orthopedics of the National Academy of Medical Sciences of Ukraine.

4. Roman Birsa, MD, a physician of the highest qualification category, orthopedist-traumatologist of the Department of Traumatology and Orthopedics, Kiev City Clinical Hospital 6.5. Petro Nemtinov, MD, PhD, a senior researcher of the Coordination Centre of Transplantation of Organs, Tissues and Cells of the Ministry of Health of Ukraine.

Special guest: Andrii Shevchenko, a legendary football player, the chief trainer of the national football team of Ukraine.

Accreditation of journalists is mandatory: office@med-info.com.ua; tel. 098 20 47 59

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Medicine of the future: cell technologies in Ukraine - Interfax-Ukraine

Stem Cell Therapy May Improve Heart Health In New Ways – TheHealthMania

Recently, a new study that appears in the journal Nature, focuses on stem cell therapy and shows unexpected ways in which it may be helpful in recovering the health of the heart. Stem cell therapy has become popular in the past few years due to its benefits for a big number of health conditions.

Currently, there is major ongoing research on stem cells since they are responsible for the regeneration of new cells and may play a fundamental role in understanding the development of a variety of different diseases as well as their potential treatments.

Some of the recent discoveries of medical science include using stem cells as regenerative medicine as they can be turned into particular types of cells that may be able to replace tissues damaged as a result of health issues and thereby control the disease.

Read also:New Study Reveals Hydromethylthionine Slows Cognitive Decline and Brain Atrophy

The therapy can be specifically useful for people with conditions such as type 1 diabetes, spinal cord injuries, Alzheimers disease, Parkinsons disease, stroke, cancer, burns, amyotrophic lateral sclerosis, heart disease, and osteoarthritis.

At the moment, the most successful procedure that involves stem cell therapy is performing a bone marrow transplant. This surgical operation replaces the cells which have been damaged during chemotherapy by programmed stem cells. People are usually able to maintain and live a normal life after recovery from the surgery.

Furthermore, stem cell usage in clinical trials designed for testing the effectiveness, safety, and potential negative impact of new drugs. To do so, the stem cells can be programmed into becoming the type of cells that the drug aims to target.

The new study, which was led by Jeffery Molkentin who is a professor of the Howard Hughes Medical Institute (HHMI) and the director of Molecular Cardiovascular Microbiology a Cincinnati Childrens Hospital Medical Center, takes data from a study from the same journal, Nature, from the years 2014 which was conducted by the same medical team.

In the new paper, the team with Molkentin as the principal investigator found some unexpected results. There were two types of stem cells in the clinical trial cardiac progenitor cells and bone marrow mononuclear cells.

The main objective of the new trial was to re-evaluate the results of the 2014 study, which showed that injecting c-kit positive heart stem in the heart does not help in the regeneration of cardiomyocytes, to see how the cell therapy can be made to be effective.

It was instead discovered that injecting an inert chemical called zymosan, which is designed particularly for inducing an innate immune response, or dead stem cells can also be beneficial for the recovery of heart as they may speed up the healing procedure.

Injecting either dead stem cells or zymosan led to a reduction in the development of cellular matrix connective tissue in the areas which had been damaged in the heart. In addition, the mechanical properties of the targeted scar also improved.

Another important finding was that chemical substances such as zymosan are required to be injected directly into the heart for optimum results. In previous clinical trials, direct injections were avoided for safety reasons.

Molkentin and the team state that follow-up studies and trials on this new discovery are imminent as they may be important for developing therapies in the future.

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Stem Cell Therapy May Improve Heart Health In New Ways - TheHealthMania

Intermountain to open new center for pediatric precision medicine – Healthcare IT News

Intermountain Primary Children's Hospital, along with University of Utah Health, and Intermountain Precision Genomics are teaming up to launch a pediatric center for personalized medicine that will serve the Intermountain West.

WHY IT MATTERS The center will use precision genomics to discover, address and treat genetic diseases, many of which affect infants and children and can cause life-long disability.

The Center will focus on precision diagnosis, gene therapies and novel therapeutics, and stem cell, immunologic and regenerative medicine.

Precision medicine includes applications across diagnostics, prevention and screening that takes into account individual variabilities in genes, environment, and lifestyle for every individual.

Through its work on precision diagnosis, the Center hopes to provide more targeted care to critically-ill children based on their genetic make-up, where rapid whole genome sequencing can quickly identify genetic causes of hard-to-diagnose diseases.

The initial efforts will be focused on providing answers to critically ill infants in the newborn intensive care unit, and children with severe seizures and heart conditions.

The research into gene therapies and novel therapeutics will help enable children with previously debilitating and fatal genetic diseases, with clinical trials testing gene therapy treatments for Duchenne's Muscular Dystrophy, Adrenoleukodystrophy, and other serious diseases.

The Center is also developing novel therapeutics that target specific diseases and improve health, with a release noting the Center is one of only six hospitals nationwide to provide gene therapy for the common childhood genetic condition spinal muscular atrophy.

Stem cell research uses a child's own cells, or genetically modifies a child's cells and immune system, to fight disease and promote healing, with additional research aimed at developing immunotherapy as a tool to fight pediatric brain tumors.

The organization also noted clinical trials are testing the use of stem cells in repairing diseased hearts and other tissues.

THE LARGER TRENDIntermountain has been busy on this front recently. In June, the health system announced that it is performing a massive clinical DNA study, pairing 500,000 samples drawn from Intermountain Healths patient population and analyzing them with help from deCODE, a subsidiary of Reykjavik-based Amgen.

"Better health and being able to cure common diseases is the promise of precision medicine, but its not happening fast enough," said Dr. Marc Harrison, president and CEO at Intermountain Healthcare, announcing that initiative. "For too long, the genetic code to better health has been locked. This collaboration with deCODE unlocks that insight so we can rapidly advance well-being not only for ourselves and our families, but for generations to come.

Intermountain's new pediatrics personalized medicine announcement also follows Mount Sinai's just-announced plans to build new precision medicine supercomputer, which will have 15 terabytes of memory, 14 petabytes of raw storage and a peak speed of 220 teraflops per second, to manage massive amounts of genomic data.

ON THE RECORD"Our mission is to leverage the expertise of our scientists, the clinical care of our physicians and care-givers, and the dedication of our community, to discover and develop new cures for children," said Dr. Josh Bonkowsky, Intermountain's medical director of the Primary Children's Center for Personalized Medicine, in a statement. "The work we are doing here and now is transforming pediatric medicine. We will not be done until we have put these diseases out of business."

Nathan Eddy is a healthcare and technology freelancer based in Berlin.Email the writer:nathaneddy@gmail.comTwitter:@dropdeaded209

Healthcare IT News is a publication of HIMSS Media.

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Intermountain to open new center for pediatric precision medicine - Healthcare IT News

Homology Medicines Announces Peer-Reviewed Publication Demonstrating its AAVHSC Vectors Crossed the Blood-Brain-Barrier and Blood-Nerve-Barrier in…

BEDFORD, Mass., Nov. 26, 2019 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today a peer-reviewed publication demonstrating its proprietary adeno-associated viral vectors (AAVHSCs) crossed the blood-brain-barrier and blood-nerve-barrier in non-human primates (NHPs), highlighting their potential to deliver gene therapy for central and peripheral nervous system disorders.

The publication includes the initial characterization of biodistribution with three of Homologys 15 AAVHSCs, including their ability to transduce, or target, key cells following a single intravenous (I.V.) administration in NHPs. AAVHSCs are naturally occurring vectors originally isolated from human hematopoietic stem cells.

Many neurological diseases, including lysosomal storage and neuromuscular disorders, have cognitive and systemic components requiring a genetic medicine to reach multiple tissues to target the disease-relevant cell types, said Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. Here we evaluated the ability of three of our novel AAVHSCs to cross the blood-brain-barrier and the blood-nerve barrier after I.V. administration in NHPs in addition to other key tissues, which allows us to choose the vectors best suited for particular diseases. We have observed that small sequence changes among our family of AAVHSCs are associated with differences in their ability to target disease-relevant tissues. We continue to characterize these properties and the potential of AAVHSCs as vehicles for therapeutic delivery.

Following I.V. administration of AAVHSC -7, -15 and -17 in NHPs, analyses showed transduction and transgene expression:

The publication, Clade F AAVHSCs Cross the Blood Brain Barrier and Transduce the Central Nervous System in Addition to Peripheral Tissues Following Intravenous Administration in Nonhuman Primates, was peer-reviewed and published in the journal PLOS ONE. For more information, please visithttps://journals.plos.org/plosone/article?id=10.1371/journal.pone.0225582or http://www.homologymedicines.com/publications.

About Homology Medicines, Inc. Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homologys proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicinesin vivoeither through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visitwww.homologymedicines.com.

Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; beliefs about preclinical data and the properties and potential of our AAVHSCs; and our position as a leader in the development of genetic medicines. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities and potential expansion of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarter endedSeptember 30, 2019and our other filings with theSECcould cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

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Homology Medicines Announces Peer-Reviewed Publication Demonstrating its AAVHSC Vectors Crossed the Blood-Brain-Barrier and Blood-Nerve-Barrier in...

Discover The Latest Frontier in Anti-aging Medicine and 3 Regenerative Therapies You Should Know… – YourObserver.com

Regenerative Medicine and Stem Cell technologies... All Hype? Or The Future of Anti-Aging?

We have entered into the rapidly evolving age of Regenerative Medicine, using breakthroughs in cell to cell communication to reset your body's natural ability to heal and repair itself. Advances in Stem Cell technology race forward at an ever increasing rate as people just like you are demanding non-surgical alternatives to the multiple degenerative conditions of aging and inflammation.

If you are asking What is a Stem Cell?

Think of them as simply the master cells of rebuilding the body. They are the building blocks of our genetic code and cellular programming that coordinate healing through bio-signaling to restore our innate capacity of repair & regeneration.

Regenerative Medicine is the vanguard of 21st century health According to Journal: American College of Cardiology

Regenerative Aesthetics is a new field of regenerative medicine that aims to restore and renew the body at the cellular level, dramatically reversing the sands of time and maintaining an aesthetically desirable youthful appearance.

What we really now know is that men and women all over the world want their hair back, they want their sexual organs to work and they want to look and feel their best into their later years.

This brings us to the 3 Regenerative Therapies you should know about.

The first and most widely known of these regenerative therapies is Platelet Rich Plasma or PRP which utilizes your own Blood Plasma and Platelets in a concentrated form to activate the healing cascade. Recruiting your own innate Stem Cells for accelerated wound healing and tissue regeneration.

Known as Liquid Gold, your platelets and plasma have been shown to rejuvenate the

Unfortunately, we are finding clinically that not all PRP is created equally. In fact, some people have a very low concentration of these regenerative growth factors or a high amount of inflammatory cytokines resulting in inconsistencies from person to person, session to session. As PRP continues to get more popular in the mainstream, we find it important to share some of the newer, more optimal Regenerative Technologies that have been emerging.

Which leads us to... The second regenerative therapy you should know about.

Stem Cell Growth factors & Cytokines. Sourced from Bone Marrow Mesenchymal Stem Cells (MSC's) however, they do not contain any actual stem cells or DNA. Growth factors are naturally occurring proteins in your body that regulate cellular growth, healing, proliferation and differentiation under controlled conditions and play a role in cellular communication. They are master bio-signals acting as command and control over your body's natural healing processes and modulation of inflammation.

It has been shown that cells in aging skin generate less growth factors than cells in youthful skin. For example, by the time you are 50, on average 4% of these regenerative bone marrow MSCs are in circulation compared to what you had when you were in your teens. Hence, we age because we damage faster than we repair in our later years.

By simply adding concentrated MSC Growth factors & Cytokines to our Regenerative Therapies we can consistently improve hair loss, skin rejuvenation, collagen growth, sexual organ function, and more. We know for a fact that daily use of skin care products containing stable growth factors and cytokines help reduce the appearance of fine lines & wrinkles and improve skin tone & texture.

Lastly, and most importantly is the latest frontier and the 3rd regenerative therapy you should know about.

The Future of Regenerative Medicine... Known as Stem Cell Exosomes. Science is showing us that the optimal way to provide true stem cell therapy is to directly provide the cell bio-signals in high concentrations. After all, the signaling is what we really require to regenerate a normal healthy physiology.

Exosomes are regarded as the purest form of cellular therapy available today, providing a safe and anti-inflammatory environment for healing and repairing.

The AABB recently reported that up to one in three people in the U.S. could benefit from regenerative medicine.

At Rejuvenate 528 Regenerative Aesthetics Medical Spa, we can include exosomes for enhanced wellness to the majority of our Regenerative Aesthetic Services. This can benefit your overall health and vitality as this is reversing challenges of Inflammation!

Beauty radiates and vibrates at different frequencies in everyone I see! We love to uplift and Rejuvenate both the inner vitality and the outer Radiance of all of our patients and clients. They come for the personal attention and integrative approach using regenerative medicine modalities with ancient technologies. PA Sheri Suiter

Tap into your own healing potential with these types of bio-hacking technologies to enhance your regenerative potential and get the results you truly desire. Live longer, stronger and younger.

Book a consultation for the following Regenerative procedures:

*Medical Microneedling*Vampire Facial*Liquid Facelift*Breastlift*O-Shot*P-Shot*Penile Enhancement*Hair Restoration*Stretch mark & scar repair*Hand Rejuvenation*Joint Inflammation*Overall Vitality & Wellness

Sheri Suiter CLT, MS, PA-C, Founder of Rejuvenate 528 Regenerative Aesthetics Medical Spa in Sarasota, FLRejuvenate528.com

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Discover The Latest Frontier in Anti-aging Medicine and 3 Regenerative Therapies You Should Know... - YourObserver.com

Worldwide Markets for Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing, Forecast to 2030 – Robust Pipeline of Therapy Candidates and…

Dublin, Nov. 27, 2019 (GLOBE NEWSWIRE) -- The "Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market (3rd Edition), 2019-2030 (Focus on AAV, Adenoviral, Lentiviral, Retroviral, Plasmid DNA and Other Vectors)" report has been added to ResearchAndMarkets.com's offering.

This report features an extensive study of the rapidly growing market of viral and non-viral vector and gene therapy manufacturing, focusing on contract manufacturers, as well as companies with in-house manufacturing facilities. The study presents an in-depth analysis of the various firms / organizations that are engaged in this domain, across different regions of the globe.

At present, 10+ genetically modified therapies have received approval / conditional approval in various regions of the world; these include (in the reverse chronological order of year of approval) Zynteglo (2019), Zolgensma (2019), Collategene (2019), LUXTURNA (2017), YESCARTA (2017), Kymriah (2017), INVOSSA (2017), Zalmoxis (2016), Strimvelis (2016), Imlygic (2015), Neovasculagen (2011), Rexin-G (2007), Oncorine (2005) and Gendicine (2003). In addition, over 500 therapy candidates are being investigated across different stages of development. The growing number of gene-based therapies, coupled to their rapid progression through the drug development process, has created significant opportunities for companies with expertise in vector manufacturing.

Presently, a number of industry (including both well-established companies and smaller R&D-focused initiatives), and non-industry players (academic institutes) claim to be capable of manufacturing different types of viral and non-viral vectors. In addition, there are several players offering novel technology solutions, which are capable of improving existing genetically modified therapy products and upgrading their affiliated manufacturing processes.

Considering prevalent and anticipated future trends, we believe that the vector and gene therapy manufacturing market is poised to grow steadily, driven by a robust pipeline of therapy candidates and technical advances aimed at mitigating existing challenges related to gene delivery vector and advanced therapy medicinal products.

Chapter Outlines

Chapter 2 is an executive summary of the insights captured in our research. The summary offers a high-level view on the likely evolution of the vector and gene therapy manufacturing market in the short to mid-term, and long term.

Chapter 3 is a general introduction to the various types of viral and non-viral vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages, limitations and applications of currently available gene delivery vehicles. The chapter also provides a brief description of the clinical and approved pipeline of genetically modified therapies. Further, it includes a review of the latest trends and innovations in the contemporary vector manufacturing market.

Chapter 4 provides a detailed overview of around 80 companies, featuring both contract service providers and in-house manufacturers that are actively involved in the production of viral vectors and / or gene therapies utilizing viral vectors. The chapter provides details on the year of establishment, scale of production, type of viral vectors manufactured (AAV, adenoviral, lentiviral, retroviral and others), location of manufacturing facilities, applications of vectors (gene therapies, cell therapies, vaccines and others) and purpose of production (fulfilling in-house requirements / for contract services).

Chapter 5 provides an overview of around 30 industry players that are actively involved in the production of plasmid DNA and other non-viral vectors and / or gene therapies utilizing non-viral vectors. The chapter provides details on the year of establishment, scale of production, location of manufacturing facilities, applications of vectors (gene therapies, cell therapies, vaccines and others) and purpose of vector production (fulfilling in-house requirements / for contract services).

Chapter 6 provides an overview of around 80 non-industry players (academia and research institutes) that are actively involved in the production of vectors (both viral and non-viral) and / or gene therapies. The chapter provides details on the year of establishment, scale of production, location of manufacturing facilities, type of vectors manufactured (AAV, adenoviral, lentiviral, retroviral, plasmid DNA and others), applications of vectors (gene therapies, cell therapies, vaccines and others) and purpose of vector production (fulfilling in-house requirements / for contract services).

Chapter 7 features detailed profiles of the US-based contract service providers / in-house manufacturers that possess commercial-scale capacities for the production of viral vectors/plasmid DNA. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience and an informed future outlook.

Chapter 8 features detailed profiles of EU based contract service providers / in-house manufacturers that possess commercial-scale capacities for the production of viral vectors/plasmid DNA. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience, and an informed future outlook.

Chapter 9 features detailed profiles of Asia-Pacific based contract service provider(s) / in-house manufacturer(s) that possess commercial scale capacities for production of viral vectors/plasmid DNA. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience, and an informed future outlook.

Chapter 10 provides detailed information on other viral / non-viral vectors (including alphavirus vectors, Bifidobacterium longum vectors, Listeria monocytogenes vectors, myxoma virus-based vectors, Sendai virus-based vectors, self-complementary vectors (improved versions of AAV), and minicircle DNA and Sleeping Beauty transposon vectors (non-viral gene delivery approach)) that are currently being utilized by pharmaceutical players to develop gene therapies, T-cell therapies and certain vaccines, as well. This chapter presents overview on all the aforementioned types of vectors, along with examples of companies that use them in their proprietary products. It also includes examples of companies that are utilizing specific technology platforms for the development/manufacturing of some of these novel vectors.

Chapter 11 features an elaborate analysis and discussion of the various collaborations and partnerships related to the manufacturing of vectors or gene therapies, which have been inked amongst players. It includes a brief description of the purpose of the partnership models (including licensing agreements, mergers/acquisitions, product development, service alliances, manufacturing, and others) that have been adopted by the stakeholders in this domain, since 2015. It consists of a schematic representation showcasing the players that have forged the maximum number of alliances. Furthermore, we have provided a world map representation of the deals inked in this field, highlighting those that have been established within and across different continents.

Chapter 12 presents a collection of key insights derived from the study. It includes a grid analysis, highlighting the distribution of viral vectors and plasmid DNA manufacturers on the basis of their scale of production and purpose of manufacturing (fulfilling in-house requirement/contract service provider). In addition, it consists of a logo landscape, representing the distribution of viral vector and plasmid DNA manufacturers based on the type of organization (industry / non-industry) and size of employee base. The chapter also consists of six world map representations of manufacturers of viral / non-viral vectors (lentiviral, adenoviral, AAV and retroviral vectors, and plasmid DNA), depicting the most active geographies in terms of the presence of the organizations. Furthermore, we have provided a schematic world map representation to highlight the locations of global vector manufacturing hubs across different continents.

Chapter 13 highlights our views on the various factors that may be taken into consideration while pricing viral vectors/plasmid DNA. It features discussions on different pricing models/approaches that manufacturers may choose to adopt to decide the prices of their proprietary products.

Chapter 14 features an informed estimate of the annual demand for viral and non-viral vectors, taking into account the marketed gene-based therapies and clinical studies evaluating vector-based therapies. This section offers an opinion on the required scale of supply (in terms of vector manufacturing services) in this market. For the purpose of estimating the current clinical demand, we considered the active clinical studies of different types of vector-based therapies that have been registered till date. The data was analysed on the basis of various parameters, such as number of annual clinical doses, trial location, and the enrolled patient population across different geographies. Further, in order to estimate the commercial demand, we considered the marketed vector-based therapies, based on various parameters, such as target patient population, dosing frequency and dose strength.

Chapter 15 features an informed analysis of the overall installed capacity of the vectors and gene therapy manufacturers. The analysis is based on meticulously collected data (via both secondary and primary research) on reported capacities of various small-sized, mid-sized and large companies, distributed across their respective facilities. The results of this analysis were used to establish an informed opinion on the vector production capabilities of the organizations across different types of vectors (viral vectors, plasmid DNA, and both), scale of operation (clinical and commercial) and geographies (North America, EU, Asia-Pacific and the rest of the world).

Chapter 16 presents a comprehensive market forecast analysis, highlighting the likely growth of vector and gene therapy manufacturing market till the year 2030. We have segmented the financial opportunity on the basis of [A] type of vectors (AAV vector, adenoviral vector, lentiviral vector, retroviral vector, plasmid DNA and others), [B] applications (gene therapy, cell therapy and vaccines), [C] therapeutic area (oncological disorders, inflammation & immunological diseases, neurological disorders, ophthalmic disorders, muscle disorders, metabolic disorders, cardiovascular disorders and others), [D] scale of operation (preclinical, clinical and commercial) and [E] geography (North America, Europe, Asia Pacific and rest of the world). Due to the uncertain nature of the market, we have presented three different growth tracks outlined as the conservative, base and optimistic scenarios.

Chapter 17 provides details on the various factors associated with popular viral vectors and plasmid DNA that act as market drivers and the various challenges associated with the production process. This information has been validated by soliciting the opinions of several industry stakeholders active in this domain.

Chapter 18 presents insights from the survey conducted on over 160 stakeholders involved in the development of different types of gene therapy vectors. The participants, who were primarily Director / CXO level representatives of their respective companies, helped us develop a deeper understanding on the nature of their services and the associated commercial potential.

Chapter 19 summarizes the entire report. The chapter presents a list of key takeaways and offers our independent opinion on the current market scenario and evolutionary trends that are likely to determine the future of this segment of the industry.

Chapter 20 is a collection of transcripts of the interviews conducted with representatives from renowned organizations that are engaged in the vector and gene therapy manufacturing domain. In this study, we spoke to Menzo Havenga (Chief Executive Officer and President, Batavia Biosciences), Nicole Faust (Chief Executive Officer & Chief Scientific Officer, CEVEC Pharmaceuticals), Jeffrey Hung (Chief Commercial Officer, Vigene Biosciences), Olivier Boisteau, (Co-Founder / President, Clean Cells) and Xavier Leclerc (Head of Gene Therapy, Clean Cells), Laurent Ciavatti (Business Development Manager, Clean Cells), Joost van den Berg (Director, Amsterdam BioTherapeutics Unit), Bakhos A Tannous (Director, MGH Viral Vector Development Facility, Massachusetts General Hospital), Colin Lee Novick (Managing Director, CJ Partners), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Semyon Rubinchik (Scientific Director, ACGT), Alain Lamproye (President of Biopharma Business Unit, Novasep), Astrid Brammer (Senior Manager Business Development, Richter-Helm), Brain M Dattilo (Business Development Manager, Waisman Biomanufacturing), Marco Schmeer (Project Manager, Plasmid Factory) and Tatjana Buchholz (Marketing Manager, Plasmid Factory), and Nicolas Grandchamp (R&D Leader, GEG Tech).

Chapter 21 is an appendix, which provides tabulated data and numbers for all the figures in the report.

Chapter 22 is an appendix that provides the list of companies and organizations that have been mentioned in the report.

Key Topics Covered

1. Preface2. Executive Summary3. Introduction4. Viral Vector and Gene Therapy Manufacturers (Industry Players): Competitive Landscape5. Plasmid DNA and Gene Therapy Manufacturers (Industry Players): Competitive Landscape6. Vector and Gene Therapy Manufacturers (Non-Industry Players): Competitive Landscape7. Vector and Gene Therapy Manufacturers in North America8. Vector and Gene Therapy Manufacturers in Europe9. Vector and Gene Therapy Manufacturers in Asia-Pacific10. Emerging Vectors11. Recent Collaborations and Partnerships12. Key Insights13. Viral Vector and Plasmid DNA Cost Price Analysis14. Capacity Analysis15. Demand Analysis16. Market Sizing and Opportunity Analysis17. Key Drivers and Challenges18. Survey Analysis19. Concluding Remarks20. Executive Insights21. Appendix I: Tabulated Data22. Appendix II: List of Companies and Organizations

Companies Mentioned

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Worldwide Markets for Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing, Forecast to 2030 - Robust Pipeline of Therapy Candidates and...

Health & Biotech: Mesoblast still not in the black, but theyre less in the red than last year – Stockhead

Stem cell biotech Mesoblast (ASX:MSB) has slashed its quarterly losses as it cut deals in Japan and China, and stepped up from R&D to partnering on its current treatments.

The September quarter loss was more than three times lower than in the same period last year, hitting $US5.5m ($8.1m) from $US19.5m ($28.8m). Revenue was up 46 per cent to $17m.

Mesoblast, which makes cellular medicines for remestemcel-L for conditions such as acute graft versus host disease (GVHD), advanced heart failure and chronic low back pain due to degenerative disc disease, recognised the change came from two areas.

The company struck a $US150m deal with German pain management company Grnenthal in September which paid out $US15m that month, for a partnership to develop and commercialise an allogeneic stem cell therapy used to treat chronic low back caused by degenerative disc disease.

Royalty revenue on sales of TEMCELL in Japan, for GVHD rose to $US1.9m.

The other side of the story is that R&D costs have plunged by 33 per cent to $12.2m as the costs for phase three clinical trials for advanced heart failure drug Revascor and low back pain drug MPC-06-ID begin to wind down.

The last patient visit for a 24-month follow up in the MPC-06-ID trial will be before June next year, and full primary endpoints or the key outcomes a trial wants to deliver are expected by the end of 2019.

Mesoblast is showing the way for small caps like Cynata (ASX:CYN) which are tackling similar diseases using cellular treatments.

Proteomics (ASX:PIQ) is spending $1.25m to help build its Western Australian Proteomics Facility to do research on biological markers affecting medicine, agriculture, the environment and marine world. Bioplatforms Australia, and the University of Western Australia are co-investors in the $4.4m site.

Bio-Gene (ASX:BGT) has won a fight with AusIndustry over a tax refund, getting $350,000 the organisation had deemed out of the refund scope. Its also pleased with the $465,293 refund it got for fiscal 2019 wouldnt we all be?

Memphasys (ASX:MEM) is now selling its Felix sperm separator to investors, after it launched earlier this month. The device, initially tested on thoroughbred horses, separates bad (human) sperm from good. Memphasys is playing up the human fertility rates in decline card, and putting fear into men that their lack of babies is, in fact, their fault.

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Health & Biotech: Mesoblast still not in the black, but theyre less in the red than last year - Stockhead

Regenerative Medicine Market: Rising Government Investments in Regenerative Medicine Research – The Chicago Sentinel

Biologics Market growth is largely driven by the rising prevalence of chronic diseases and genetic disorders, growing government investments in regenerative medicine research, and the increasing number of regenerative medicine companies globally.

What the Market Looks Like?

Predicted to grow at a CAGR of 23.8% during the forecast period, the global Regenerative Medicine market is estimated to reach USD 38.7 Billion by the end of 2024. North America to grow at the highest rate during the forecast period. North America has both public and private banks that store a limited number of cells and tissues.

Rising government investments in regenerative medicine research

The need for newer, better therapies for diseases/conditions such as cancer, diabetes, dermal diseases, musculoskeletal diseases, burns, and CVD has resulted in an overall increase in the number of research activities as well as the availability of funding for regenerative medicine research. Currently, several government organizations are investing in research institutes to develop advanced technologies in cell therapy. For instance, in January 2016, the government of Canada granted USD 20 million to the Centre for Commercialization of Regenerative Medicine. This grant will be utilized for establishing a stem-cell therapy development facility in Toronto, Canada. In addition, the NIH funding for the development of cell therapies is increasing significantly, annually.

The US is the major participant in the global regenerative medicine research sector, with various organizations in the country supporting regenerative medicine research. Since 2004, the California Institute of Regenerative Medicine (CIRM) has sponsored over 750 projects, with over USD 2.98 billion in funding. The NIH also funded USD 4.8 billion for research and development under the 21st Century Cures Act, including funding for regenerative medicine, for a ten-year period starting in 2017. Furthermore, under the 21st Century Cures Act, the FDA launched novel procedures in 2017, such as the regenerative medicine advanced therapy (RMAT) designation, to accelerate the access to the most promising regenerative medicine therapies.

Owing to the importance of regenerative medicine, the number of government and private bodies supporting R&D activities related to stem cell therapeutics and regenerative medicine is expected to increase further in the coming years.

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What Drives the Market?

The growth of the global market for Regenerative Medicine is primarily influenced by the following factors:

The other factors supporting market growth include the growing pipeline of regenerative medicine products and the rising demand for organ transplantation.

Geographical growth scenario of Regenerative Medicine Market

Geographically, the Regenerative Medicine market has been studied for North America, Europe, Asia Pacific, and the Rest of the World. North America accounted for the largest market share in 2018 and is also projected to witness the highest growth in the forecast period.

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Leading market players and strategies adopted

The prominent players in the Regenerative Medicine market include Kite Pharma (US), Novartis (Switzerland), Vericel Corporation (US), Integra LifeSciences (US), Wright Medical (US), MiMedx (US), Osiris Therapeutics (US), Stryker Corporation (US), and Spark Therapeutics (US).

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Regenerative Medicine Market: Rising Government Investments in Regenerative Medicine Research - The Chicago Sentinel

Research Antibodies and Reagents Market Worth $14.56 Billion by 2025 – Exclusive Report by Meticulous Research – GlobeNewswire

London, Nov. 25, 2019 (GLOBE NEWSWIRE) -- According to a new market research report Research Antibodies and Reagents Market by Product (Reagent [Sample Preparation (Media, Probe, Buffer), Antibody Production Reagent], Antibody [Type, Source, Research Area]), Technology (Western Blot, ELISA), Application, End-User - Global Forecast to 2025, published by Meticulous Research, the research antibodies and reagents market is expected to grow at a CAGR of 6% from 2019 to 2025 to reach $14.56 billion by 2025.

Research antibodies act as a vital tool in laboratory techniques that are conducted for research purpose. Due to their high specificity and sensitivity, antibodies and associated reagents make an exquisite tool aiding in the detection of molecules and its analysis, which enables the researchers to draw conclusions related to drug target, disease occurrence, and immune system pathways. The rising proteomics and genomic research studies, increase in the funding for research activities, and growing industry-academia collaborations are the key factors driving steady growth in the global research antibodies and reagents market.

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In recent years, the funding for healthcare research projects has increased significantly. According to the Research America, investment in medical and health research and development by the U.S. has increased by 18.1% between 2014 and 2017. In 2017, total expenditure on medical and health R&D in the U.S. was $182.3 billion. Industry, including pharmaceutical, medical technology, biotechnology and health IT companies, accounted for the largest share of the U.S. investment in medical and health R&D over the reporting period. Similarly, in Europe, according to European Union (EU), Horizon 2020 program was launched in 2016, with an investment of $88.5 billion (80 billion); of which around 10% (~$9 billion) was utilized for healthcare sector. The initiative was aimed at developing and supporting personalized medicine projects. Also, during the period of 2014-2016, the European Commission invested more than $2.7 billion (2.4 billion) in projects that supported innovation in personalized medicine. Therefore, such increase in funding has led to rise in research activities in pharmaceutical and biotechnology industries, thereby boosting the demand for research antibodies and reagents.

The research antibodies and reagents market study presents historical market data in terms of value (2017 and 2018), estimated current data (2019), and forecasts for 2025 by product, technology, application, end user, and geography. The study also evaluates industry competitors and analyzes their market share at the global and regional levels.

Based on product, reagents segment is estimated to account for the largest share of the overall research antibodies and reagents market in 2019 and is slated to grow at the fastest CAGR during the forecast period. The large share of this segment is attributed to recurrent use of reagents in many research assays and techniques and increasing developments in research reagents.

Based on technology, western blotting segment is estimated to account for the largest share of the overall research antibodies and reagents market in 2019. This is mainly attributed to increasing focus on initiating developments in diagnosis and therapy procedures and rising research funding for biomarker discovery and antibody & cell-based products. On the other hand, Enzyme-linked Immunosorbent Assay (ELISA) is slated to grow at the fastest CAGR during the forecast period due to increasing use of ELISA technology for screening of active ingredients and validation of obtained hits in drug discovery.

Based on application, proteomics segment is estimated to hold the largest share of the overall research antibodies and reagents market in 2019. This is mainly attributed to the increase in search for ideal candidates for use as anti-cancer drugs, need for designing more effective drugs, assessment of drug efficacy and patient response, and development of antibodies especially monoclonal antibodies. Growing use of proteomic technologies in development of target-based therapies also supports the large share and growth in this market.

Based on end user, pharmaceutical and biotechnology companies segment is estimated to hold the largest share of the overall research antibodies and reagents market in 2019, due to high adoption of research antibodies & reagents in proteomics research and drug discovery and development programs. Rising focus of industry players on developing innovative therapeutics for chronic diseases is contributing to the growth of this market. On the other hand, the demand for research antibodies from contract research organizations (CROs) is slated to grow fastest during the forecast period, mainly due to increase in outsourcing activities by pharmaceutical industries to CROs.

The report also includes extensive assessment of the key strategic developments adopted by leading market participants in the industry over the past 4 years (2016-2019). The research antibodies and reagents market has witnessed number of partnerships & agreements in the recent years. For instance, in January 2018, Abcam plc (U.K.) entered into an agreement with Roche to facilitate licensing of product portfolio of Spring Bioscience Corporation to Abcam for research use only (RUO) applications, which includes recombinant antibodies. Similarly, in October 2017, Bio-Techne Corporation (U.S.) entered into an agreement with Joint Consortia Framework for the development of research antibodies & antibody-based reagents. Similarly, in January 2018, Illumina, Inc. (U.S.) launched iSeq 100 sequencing system, which offers exceptional data accuracy, at a low capital cost.

The global research antibodies and reagents market is highly fragmented with the presence of key players, such as, Abcam plc (U.K.), Merck KGaA (Germany), Cell Signaling Technology, Inc. (U.S.),GE Healthcare (U.S.), Thermo Fisher Scientific Inc. (U.S.), F. Hoffmann La-Roche Ltd (Switzerland), Rockland Immunochemicals Inc. (U.S.), Johnson & Johnson (U.S.), Agilent Technologies, Inc. (U.S.), Eli Lily and Company (U.S.), Becton Dickinson and Company (U.S.), Danaher Corporation (U.S.), PerkinElmer, Inc. (U.S.), GenScript Biotech Corporation (U.S.), Lonza (Switzerland), Bio-Techne Corporation (U.S.), Bio-Rad Laboratories, Inc. (U.S.), Teva Pharmaceutical Industries Limited (Israel), Santa Cruz Biotechnology, Inc. (U.S.), and BioLegend, Inc.(U.S.) among others.

Browse key industry insights spread across 280 pages with 256 market data tables & 61 figures & charts from the report:https://www.meticulousresearch.com/product/research-antibodies-reagents-market-5055/

Scope of the Report:

Research Antibodies and Reagents Market, by Product

Research Antibodies and Reagents Market, by Technology

Research Antibodies and Reagents Market, by Application

Research Antibodies and Reagents Market, by End User

Research Antibodies and Reagents Market, by Geography

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Research Antibodies and Reagents Market Worth $14.56 Billion by 2025 - Exclusive Report by Meticulous Research - GlobeNewswire