Category Archives: Cell Medicine

A regenerative medicine startup led by a Mayo Clinic cardiologist is setting up shop in a downtown Rochesters Minnesota BioBusiness Center, according to newly filed city documents. The filing indicated Rion LLC, a Minnesota company registered to Dr. Atta Behfar of the Mayo Clinic Center for Regenerative Medicine, has signed a three-year lease for just over 2,000 square feet at the city-owned BioBusiness Center. The lease begins July 1. The nine-story BioBusiness Center opened in downtown Rochester in 2007 as a center for innovation in biotechnology, promoting the linkages between the researchers and practitioners at Mayo Clinic; instructors and students at the University of Minnesota Rochester, and the biotechnology business community. It houses the Mayo Clinic Business Accelerator among other tenants. Behfar is an assistant medical professor and leads a laboratory at Mayo concentrating on applying regenerative medicine the practice of using stem cells to regenerate damaged or missing tissue to prevent and cure chronic heart conditions. Specifically, his group focuses on development and use of both stem cells and protein-based therapies to reverse injury caused by lack of blood flow to the heart. The business direction of Rion, meanwhile, appears to be specifically geared toward a cutting-edge development in the field of regenerative medicine the use of extracellular vesicles (EVs) in speeding and directing the growth of regenerating tissues in the heart and elsewhere in the body. EVs, long brushed off by researchers as mere debris in the bloodstream, are membrane-enclosed spheres that break off from the surfaces of nearly all living cells when disturbed. They transport lipids, proteins and nucleic acids, and have now been found to be important players in cell-to-cell communication, influencing the behavior and even the identity of cells. Their emerging role in regenerative medicine could potentially be huge. For instance, by bioengineering them to transport protein payloads from stem cells, they can be used to signal the bodys own cells to regenerate tissue instead of transplanting the stem cells themselves, thus eliminating the chance of host immune system rejection. A patent application filed last year by Rion, Behfar, Mayo Center for Regenerative Medicine Director Dr. Andre Terzic and two other local inventors is aimed at adapting the healing properties of a specific type of EV into a unique kind of product that could have wide applications. It focuses on EVs derived from blood platelets, which are well known to stop bleeding, promote the growth of new tissues and blood vessels, relieve inflammation and provide a host of other benefits. The patent describes a system of encapsulating platelet EVs derived from human or animal blood into a platelet honey and delivering it to target areas of the body, such as damaged tissues or organs. Its purported effect is to regenerate, repair and restore damaged tissue, with possible uses including treating heart disease; healing damaged bones or joints; wound treatment; and cosmetic skin applications. A brief business description provided by Rion to Rochester city officials stated the company is focused on the delivery of cutting edge regenerative technologies to patients at low cost and in off-the-shelf fashion. Building on initial research at Mayo Clinic, Rion LLC aims to develop and bring to practice products in the space of wound healing, orthopedics and cardiac disease. The statement also added the company is an enthusiastic backer of Rochesters efforts to develop a local biotech business cluster, and is seeking to participate in the realization of the Destination Medical Center initiative.

See the article here:
Mayo-Connected Regenerative Medicine Startup Inks Downtown Rochester Lease - Twin Cities Business Magazine

Posted 28 June 2017 By Zachary Brennan

With a new Texas law now in the books to allow companies to sell unproven stem cell treatments without US Food and Drug Administration (FDA) approval, some experts wonder when FDA will step in to shut down companies and clinics exposing people to unapproved medical products.

Similar to the Right to Try laws spreading across the US and attempting to undercut FDAs regulation of investigational products, the Texas law, which had been brewing in some form since 2012, applies to certain investigational stem cell treatments for patients with certain severe chronic diseases or terminal illnesses.

And though the Texas law says that it applies to stem cell treatments currently under investigation in clinical trials, it also blocks the Texas Medical Board from revoking, failing to renew or suspending a physicians license based solely on the physicians recommendations to an eligible patient regarding access to or use of an investigational stem cell treatment.

An investigation by Nature in 2012 uncovered unproven and costly stem cell treatments being sold in Texas.

Leigh Turner, anassociate professorat the University of Minnesotas Center for Bioethics and School of Public Health and co-author of a paper in Cell on the selling of stem cell therapies directly to US consumers, explained to Focus the unpredictable nature of Texas law, as the state already has one of the highest concentrations of unregulated stem cell clinics (California and Florida also have high concentrations of such clinics, he said, noting his investigation found more than 500 such clinics in the US).

There are lots of credible stem cell researchers in Texas, but theyre not the ones pushing for this bill, Turner said, noting that the greatest concern should be focused on clinics offering unproven stem cell treatments for a range of diseases with few or no treatment options, like ALS, autism, spinal cord injuries and others.

And though there are provisions in the bill that, according to Turner, could knock some businesses out of the marketplace in Texas, he said its hard to know which way itll break, though it seems clearly based on the fantasy that Texans have to go elsewhere to receive these investigational stem cell therapies.

FDA has so far let these direct-to-consumer stem cell clinics flourish and has only issued a limited number of warning letters to companies like Cell Vitals in 2014, Irvine Stem Cell Treatment Center in 2015 and Lavian in 2016.

Former FDA Commissioner Robert Califf, Center for Biologics Evaluation and Research Director Peter Marks and CBER Deputy Director Celia Witten in December 2016 made the case in the New England Journal of Medicine that the hype over such treatments outpaces the evidence that they are safe and effective, though the agency has not cracked down on the direct-to-consumer stem cell market.

FDA couldve done something since 2009, this is a marketplace thats been around a while, Turner said.

And because many of the clinics engage in interstate commerce, Turner said they do clearly fall within FDAs jurisdiction. But its not just FDA, he added, the Federal Trade Commission could also act, as could state consumer protection agencies or state medical boards.

So why isnt FDA taking action? The agency did not respond to a request for comment and Turner said he has not received a straight answer from the agency.

Will they knock out these businesses? Talking with FDA, I didnt hear that, he said, adding that he did not get the impression that theres a comprehensive, organized plan to deal with the emergence of such a large and growing marketplace.

Read more from the original source:
New Texas Law on Stem Cell Treatments: Showdown With FDA Coming? - Regulatory Focus

June 28, 2017 Disruption of a region in chromosome 6 or epigenetic modifications of the DNA block Sestrin1 expression and these contribute to the development of follicular lymphoma. Credit: Elisa Oricchio/Natalya Katanayeva/EPFL

Follicular lymphoma is an incurable cancer that affects over 200,000 people worldwide every year. A form of non-Hodgkin lymphoma, follicular lymphoma develops when the body starts making abnormal B-cells, which are white blood cells that in normal conditions fight infections. This cancer is associated with several alterations of the cell's DNA, but it has been unclear which gene or genes are involved in its development. EPFL scientists have now analyzed the genomes of more than 200 patients with follicular lymphoma, and they discover that a gene, Sestrin1, is frequently missing or malfunctioning in FL patients. The discovery opens to new treatment options and it is now published in Science Translational Medicine.

One of the common features of follicular lymphoma is a genetic abnormality between two chromosomes (14 and 18). In an event known as "chromosomal translocation" the two chromosomes "swap" certain parts with each other. This triggers the activation of a gene that protects cells from dying, making cells virtually immortalthe hallmark of a tumor.

Moreover, approximately 30% of follicular lymphoma patients lose also a portion of chromosome 6, affecting multiple genes involved in suppressing the emergence of a tumor. These patients typically have poor prognosis. Another 20 % of patients have alterations causing chromosomal disorganization and the consequent malfunctioning of several genes and proteins. The bottom line is that for both group of patients it is very difficult to pinpoint which of all the affected genes are actually causing the disease.

The lab of Elisa Oricchio at EPFL, with colleagues from the US and Canada, analyzed the genomes of over 200 follicular lymphoma patients. Their analyses revealed that a specific gene, Sestrin1, can be harmed by both loss of chromosome 6 and silenced in patients.

Sestrin1 helps the cell defending itself against DNA damagefor example after exposure to radiationand oxidative stress. In fact, Sestrin1 is part of the cell's anti-tumor mechanism that stops potentially cancerous cells from growing.

Disruption of a region in chromosome 6 or epigenetic modifications of the DNA block Sestrin1 expression and these contribute to the development of Follicular Lymphoma.

Beyond identifying the Sestrin1 gene as frequently altered in FL patients, the scientists demonstrated that Sestrin1 is able to suppress tumors in vivo. They showed that Sestrin1 exerts its anti-tumor effects by blocking the activity of a protein complex called mTORC1, which is well known for controlling protein synthesis as well as acting as a sensor for nutrient or energy changes in the cell.

Finally, the identification of loss of Sestrin1 as a key event behind the development of follicular lymphoma is particular important because it helps identifying patients that will benefit from new therapies. Indeed, this study shows that the therapeutic efficacy of a new drug that is currently in clinical trial depends on Sestrin1. Importantly, this dependency can be extended beyond follicular lymphoma to other tumor types.

Explore further: Combination therapy may help patients with follicular lymphoma

More information: E. Oricchio el al., "Genetic and epigenetic inactivation of SESTRIN1 controls mTORC1 and response to EZH2 inhibition in follicular lymphoma," Science Translational Medicine (2017). stm.sciencemag.org/lookup/doi/10.1126/scitranslmed.aak9969

A new study in The Journal of Experimental Medicine reveals that a high-risk group of patients with follicular lymphoma could benefit from a novel drug combination.

Mutations present in a blood cancer known as follicular lymphoma have revealed new molecular targets for potential treatments, according to researchers at Queen Mary University of London (QMUL) together with collaborators ...

Immune cellular therapy is a promising new area of cancer treatment. Anti-cancer therapeutics, such as chimeric antigen receptor (CAR) modified T cells, can be engineered to target tumor-associated antigens to attack and ...

Follicular lymphoma (FL), the second most common form of non-Hodgkin lymphoma, is a largely incurable disease of B cells, yet in many cases, because of its indolent nature, survival can extend to well beyond 10 years following ...

The goal for many cancer patients is to reach the five-year, disease-free mark, but new research from UR Medicine's Wilmot Cancer Institute suggests that two years might be a more practical survival goal for people with follicular ...

(HealthDay)An initial watch-and-wait strategy does not have a detrimental effect on the freedom from treatment failure (FFTF) or overall survival rate in selected patients with low-tumor burden follicular lymphoma compared ...

Cancerous tumors are formidable enemies, recruiting blood vessels to aid their voracious growth, damaging nearby tissues, and deploying numerous strategies to evade the body's defense systems. But even more malicious are ...

Follicular lymphoma is an incurable cancer that affects over 200,000 people worldwide every year. A form of non-Hodgkin lymphoma, follicular lymphoma develops when the body starts making abnormal B-cells, which are white ...

Leukemia researchers led by Dr. John Dick have traced the origins of relapse in acute myeloid leukemia (AML) to rare therapy-resistant leukemia stem cells that are already present at diagnosis and before chemotherapy begins.

Adding an investigational antibody to the chemotherapy rituximab appears to restore its cancer-killing properties in certain leukemia patients with a natural resistance to the drug, according to a small, proof-of-concept ...

Early detection is particularly important in cutaneous melanoma, the most aggressive type of skin cancer. With a thickness of little more than one millimetre, the tumour may begin to spread, sending its cells to colonise ...

A study by the University of Aberdeen has found that a higher concentration of the molecules that breakdown omega-3 fatty acids is associated with a higher chance of survival from bowel cancer.

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Go here to read the rest:
The gene behind follicular lymphoma - Medical Xpress

This press release was orginally distributed by SBWire

New York, NY -- (SBWIRE) -- 06/27/2017 -- Stem cell therapy, a way to replace damaged tissues through regenerative medicine, holds hope for patients everywhere. However, aside from very few applications, cell therapy has not made the leap from laboratory to market. One of the major hurdles have been a cost-effective way of achieving purified stem cells. A big challenge in cell therapy has been separating mature donor cells, which can cause severe damage to the patient, from the donor's stem cells which heal through regenerative medicine. Cellect Biotechnology Ltd., developer of a novel stem cell collection technique, has an effective and elegant technology for harvesting stem cells.

A key component of Cellect's platform technology, ApoGraft, is the ApoTainer which uses cell suicide inducing proteins to kill older cells through apoptosis, or programmed cell death. Cellect has discovered that in an apoptotic environment, mature cells die, while stem cells survive and thrive. The cell selection process is quick, just hours, and the result is a rich, non-toxic batch of stem cells ready to be implanted.

In its first indication, Cellect is evaluating its technology in allogenic (donor) bone marrow transplant (BMT) where up to 50% of patients suffer graft-versus-host disease (GvHD) under current methods, often leading to sickness and death. With the right stem cell procedure, GvHD can be reduced and potentially eliminated.

BMT, a procedure fraught with the chance for GvHD because of its mix of old cells that trigger rejection of tissue implants, is Cellect's initial indication. Cellect demonstrated success in its first stem cell transplant in March using ApoGraft in BMT in its Phase I/II trial, leading to the independent Data and Safety Monitoring Board's (DSMB) approval to continue patient enrollment. A total of 12 patients suffering from blood cancer will be treated, with an eye on efficacy and safety in preventing GvHD.

75,000 BMTs are performed in the US annually at an average cost of $800,000 per transplant, resulting a $60 billion market that is growing due to an aging population and is mainly limited by the GvHD.

Currently, stem cells are either sourced from the patient (autologous) prior to undergoing high-dose chemo or radiation therapy, or they are harvested from donor (allogeneic) stem cells. Chances for perfect donor match for blood cancer patients undergoing BMT are only 25% not good odds. Allogenic donors are subject to an intensive and long procedure including rigorous physical exams, blood samples taken in up to four separate appointments, time commitment of 30 hours spread over four to six weeks, and travel expense.

Both autologous and allogeneic methods can be hazardous because of the combined mix of old cells and vital stem cells that have a better chance to avoid GvHD. This is standard medicine, and not an optimal solution. Cellect's technology eliminate the non-matched immune response carrying cells . Time and money can be saved.

Operating under a well-thought out business model, Cellect plans to out-license its ApoGraft platform to pharma, biotech, research centers and hospitals, furnishing them with an unprecedented tool. Non-exclusive licensing gives Cellect a broad array of potential partners.

Cellect's robust intellectual property protection includes patents that cover all aspects of selecting only the cells needed for the transplantation, with the promise of avoiding GvHD. Yet Cellect's platform is not only for blood cancers. Other future applications can be directed to autoimmune disease such as Juvenile diabetes, all significant markets.

Cellect has drawn leaders in key areas important to further regenerative medicine into mainstream treatment options. Scientific and medical advisors include researchers and practitioners from Dana Farber, Harvard, King's College London, Pfizer Inc., the FDA, and Stanford. All have a deep interest in making stem cell therapy a reality. All will be critical to advising the company on current and future regulatory pathways. All strongly believe in Cellect's technology.

At the end of the first quarter of 2017, Cellect had cash of approximately $7 million. With the stock trading at about $8.50, the market cap is $45 million.

If the Phase I/II study proves successful, Cellect will be well positioned to partner with pharma and biotech firms who will use the ApoGraft platform in whichever applications they wish, giving Cellect an opportunity for ongoing revenue from licensing and royalties. This could boost its cash levels, stock price and market cap.

Cellect leads the world in providing a pioneering method to harvest stem cells that seek to cure many diseases, starting with proof in BMT, without adverse side effects. Its goal is to make ApoGraft available for clinics to use easily and effectively, much like any modern-day medical tool. Its technology is both simple and revolutionary, making stem cell medicine a nearer-term reality than ever before.

RAY DIRKS Research suggests that Readers/Investors place no more than 1% of the funds they devote to common stocks in any one issue. It's best to diversify.

About Ray Dirks Ray Dirks came to Wall Street with Goldman, Sachs & Co. in 1963 where he was established as the leading insurance stock analyst dealing with institutional investors and high -net worth investors both in the U.S. and internationally.

In 1973 Ray uncovered the biggest Ponzi scheme of the 20th century, the Equity Funding fraud. Over the years Ray has expanded his stock market research to include Healthcare Stocks and Special Situations. Ray has written two books, "The Great Wall Street Scandal" and "Heads You Win, Tails You Win," published by McGraw-Hill and Bantam Books respectively. He continues to provide research to institutions and individuals, and he manages money for some individual investors.

For more information, log on to http://www.raydirks.com.

For more information on this press release visit: http://www.sbwire.com/press-releases/cellects-novel-technology-isolates-therapeutic-stem-cells-for-regenerative-medicine-825784.htm

Read this article:
Cellect's Novel Technology Isolates Therapeutic Stem Cells for Regenerative Medicine - Digital Journal (press release)

~By Dr. Udaya Kumar Maiya, MBBS, MD, DNB, DCCF-Paris

In the entire field of oncology, perhaps the most disruptive ongoing revolution is the application of Chimeric Antigen Receptor - T Cells or (CAR-T) to cancer immunotherapy. In this therapy, engineered T-cells that express a specific tumor antigen binding domain called Chimeric Antigen Receptor is infused into the patient. As the construct binds to its target domain, the T-cell is activated and attacks the cancer cell with great specificity. Remission rates as high as 94% in late-stage acute lymphoblastic leukemia (ALL) have been observed. While ALL itself is a very curable cancer with high CRs (Complete Remission) seen with HSCT therapy (hematopoietic stem cell transplant), subdued whispers are being heard in laboratory corridors and newsrooms whether cancer has finally found its nemesis.

CAR-T therapy has three specific problems: one, off-target toxicity; two, lack of an allogenic version of the therapy; and three, difficulty in applying CAR-T therapy to solid tumors. While the first issue is being widely tackled with various forms of biologic switches, and there is considerable ongoing research to develop allogenic off-the-shelf models of CAR-T, CAR-T's application to solid tumors has been restricted by the inherently immuno-suppressive solid tumor microenvironment which limits T-cell infiltration, and the original focus on the B-cell surface specific CD19 receptor which has also somewhat restricted research to the hematologic space.

However, while liquid or blood cancers account for only about 9% of cancer deaths in the US, solid tumors like carcinomas, sarcomas, and lymphomas account for over 43% cancer deaths in the US. If there is a small company developing CAR-T for solid tumors, that should be a valuable speculative investment.

Celyad (NASDAQ:CYAD), a small Belgium-based drug developing company, is working to develop a CAR-T therapy for solid tumors. Its approach is to move away from the CD19 receptor and work with a construct which expresses Natural Killer Receptors (NKRs), binding to eight different ligands that are expressed in over 80% of solid tumors. With a market cap of just $444mn, very little debt, low cash burn rate, and a cash runway to see it through the next couple years, Celyad is solidly positioned to appreciate many times per my valuation sheet below.

About the ongoing revolution in CAR-T

The chimeric antigen receptor T (CAR-T) is a construct of two elements, the CAR and the T cell. CARs are synthetic receptors consisting of an antigen-binding domain called scFv or single-chain variable fragment. The scFv component identifies and then binds to one of the numerous tumor-associated antigens (TAAs) expressed on the tumor cell surface. T-cell is then activated and kills the targeted tumor cell. Third generation CARs have multiple co-stimulatory domains besides the primary activation domain and provide for greater expansion, prolonged anti-tumor activity, and cytokine secretion.

Here's an excellent selection from a CAR-T primer presented at ASCO:

"CAR T cells can recognize a wide range of molecules or antigens - proteins or pieces of proteins, sugars, and fat molecules (each specific CAR is programmed to recognize one specific antigen). When the receptor attaches to an antigen molecule on a cancer cell, it sends a signal to turn on the T cell's destruction mode. Unlike traditional cancer treatments, this "living therapy" needs to be given to the patient only once because CAR T cells continue to multiply in the patient's body. This means the anticancer effects of CAR T cells persist and can even increase as the cells multiply. The persistence of CAR T cells in the body differs between CAR T-cell products, with some persisting one or two months and others for years."

CAR-T has been spectacularly successful. Early success came with CD19-targeting CAR-Ts in acute lymphoblastic leukemia or ALL, with up to 90% remission of up to two years durability in a poor-prognosis disease with expected survival duration of no more than six months. Recently, a BCMA-targeting CAR-T was responsible for 94% remission in high-mortality refractory multiple myeloma patients, as shown by results presented at ASCO 2017.

However, CAR-T in solid tumors has not been very successful. A 2016 article reports that "To date, the two most positive trials reported have used GD2 CARs to target neuroblastoma (3 of 11 patients with complete remissions), and HER2 CARs for sarcoma (4 of 17 patients showing stable disease)." Both were academy-sponsored trials; neither was as spectacularly successful as the hematologic cancer trials. This article also lists a number of barriers to tumor invasion by CAR-T cells, which is shown in the graphic below:

(Source)

Clearly, new research is needed to develop CAR-Ts for this very large market.

The Solid Tumor market

The solid tumor (malignant) market is much larger than the blood cancer market.

(Source)

According to some research, the global solid tumors drugs market is expected to reach more than $43 billion by 2020.

On the other hand, according to this market research, the leukemia therapeutics market was valued at $6.3 billion in 2010 and is expected to reach $11.3 billion by 2020 at a CAGR of 3.84% between 2015 and 2020. The market was dominated by chronic myeloid leukemia in 2010. However, in 2020, the market is expected to be equally dominated by acute lymphocytic leukemia and chronic myeloid leukemia, with sales amounting to $3.91 billion and $3.58 billion, respectively.

So, what we see here is that CAR-T is only being investigated in a market that is almost a fourth of the much larger solid tumor market. There is, thus, a huge opportunity here.

According to the WHO, in 2015, the most common causes of cancer death are cancers of: Lung (1.69 million), Liver (788,000), Colorectal (774,000), Stomach (754,000), and Breast (571,000). As you can see, these are all solid tumor cancers. Leukemia comprises only about 2.5% of all new cancer incidents annually.

Celyad's approach to using CAR-T in solid tumors

Celyad is developing CAR-Ts using T-cells designed to express the Natural Killer Receptor Group 2D (NKG2D) receptor. This approach is based on preclinical work done by Professor Charles Sentman at Dartmouth College (USA) who showed that these receptors fused with CD3 (CD3-zeta) chain of the T-cell receptor complex drive impressive anti-tumor activity against established tumors in mouse models. The good thing about NKG2D CARs is that they not only target tumor cells but also target immuno-suppressive cells (MDSCs, Tregs) within the tumor microenvironment (TME) which also express NKG2D ligands. Immuno-suppression in the TME is a major issue that has hindered CAR-T development in solid tumors.

Celyad scientists have a number of important publications in the application of NKR-based CAR-T immunotherapy, one of which is presented below in abstract simply in order to demonstrate research pedigree.

(Source)

Professor Sentman lists a number of human tumor cells that express NKG2D ligands: carcinomas (ovarian, bladder, breast, lung, liver, colon, kidney, and prostate), melanoma, Ewing's sarcoma, glioma, neuroblastoma, various leukemias (acute myelogenous leukemia, chronic myelogenous leukemia, and chronic lymphocytic leukemia), lymphomas, and multiple myeloma. Together, these constitute about 80% of all solid tumors as well as some leukemias.

Celyad's intellectual property portfolio includes four patent families exclusively licensed to Celyad by Dartmouth College. This includes four issued U.S. patents; six pending U.S. patent applications; and 13 foreign patent applications pending in various jurisdictions.

It also has an allogenic program in preclinical stage. Allogenic, off-the-shelf CAR-T therapy is an important critical development need for the future because it will drastically reduce cost of therapy and make it more efficient and less time-consuming to administer.

In other areas of development, Celyad has a cardiology candidate named C-Cure, which is being evaluated in an European Phase III clinical trial. This trial is now complete, although with mixed results, as the primary endpoint was met (p=0.015) for only a subset of patients, although representing 60% of the population of the study. Celyad is now looking for a partner to further develop and commercialize the product.

Preclinical and clinical studies

The company has completed a Phase I study at the Dana-Farber Cancer Institute in the US in patients with AML and MM without prior lymphodepletive preconditioning chemotherapy. Preconditioning chemotherapy and subsequent lymphodepletion is a major drawback of existing CAR-T. No major safety issues were reported - "There were no cases of cytokine release syndrome, cell-related neurotoxicity, auto-immunity, or CAR T-related death." This, although premature and needs following up, is promising because CAR-T does have a number of safety issues associated with it. The trial data was presented at 2016 ASH Annual Meeting and demonstrated the drug to be safe and well tolerated in the highest dose level tested (3x107) as well as showing early clinical activity signals, including prolonged survival in both Acute Myeloid Leukemia (AML) and Multiple Myeloma (MM) patients:

"However, cases of unexpected survival without further therapy and responses to subsequent treatments were noted. For example, a patient with p53-mutated AML survived 4 months despite 50% blasts at infusion, and another entered PR at 6+months after cells on an IDH-1 inhibitor with <5% IDH and 54% p53 mutation burden at initiation. RCR testing at 3 (n=6) and 6 months (n=1) was negative."

Such signs of clinical activity were unexpected based on the single-dose schedule and dose-level being 100 times below the estimated pharmacological effective dose, according to the report.

In murine models in in vivo studies, CAR-T NKR-2 demonstrated the ability to recognize and eliminate "most tumor cell types over the natural life of the animal." Even a single injection of murine CAR-T NKR-2 showed some anti-tumor activity, and three sequential doses produced complete recovery and overall survival without adverse events. Note that NKR ligands in mice are quite different from those in humans.

As can be seen from the following graphics, the NKR platform showed significant survival benefits in early preclinical murine models:

Source: Company Presentation

Celyad is running seven Phase I trials with autologous CAR-T using NKR targeting two heme cancers and five solid tumor malignancies. THINK (THerapeutic Immunotherapy with NKR-2) is a multinational open-label Phase I study to assess the safety and clinical activity of multiple administrations of autologous NKR-2 T-cells in seven refractory cancers including five solid tumors (colorectal, ovarian, bladder, triple-negative breast, and pancreatic cancers) and two hematological tumors (acute myeloid leukemia and multiple myeloma).

Source - Investor presentation

Differences with standard CD19-based CAR-T therapy

A number of critical, even-game changing differences exist between standard CD19 based CAR-T therapy and Celyad's NKR-2 approach.

Financials

Celyad is a $450mn market cap company with about $80mn in cash balance as of the March quarter. This, the company says, is enough to last it for two entire years - which, given it has pretty early stage trials as of now, sounds about right. However, as it goes towards later stage trials, hopefully no later than 2019, it will need much more money.

If its cardiology product begins generating revenue, it can make some money out of it. Also, it has signed couple decent sized deals with a Japanese company named ONO and CAR-T giant Novartis. The ONO deal is worth $311mn plus double-digit royalties and is for the development and commercialization of Celyad's allogeneic CAR-T NKR-2 immunotherapy in Japan, Korea, and Taiwan. The other deal with Novartis (NYSE:NVS) is worth about $96mn, including upfront and milestone payments and single-digit royalties for a non-exclusive license for its allogeneic TCR-deficient CAR-T cells patents. I don't have separate figures for the milestones, but I assume it has been planned keeping future developments in mind; so that assures me it will have some cash flow for the Phase III trials. The company is, however, trading very near its 52-week highs, so, at these levels, dilution is something of a long-term worry.

Market Opportunity and Valuation

We have done a valuation of CYAD's autologous CAR-T platform here. The valuation note and the complete spreadsheet are provided below.

Source: Author

The valuation, admittedly, is a little simplistic because we have taken the broad solid tumor market as our target indication. We have adjusted for that by taking a very conservative market penetration rate. The recent approval of Keytruda based on genetic markers and not on cancer type gives us hope that this may become the norm for drugs that are based on markers. Given that Keytruda already makes almost $2 billion per year, our revenue figures do not look unrealistic to me. Moreover, autologous immune therapies, unlike checkpoint inhibitors, may charge a premium because this is not just a drug but a drug and a service facility.

Another issue is year of approval. We have taken this to be 2020, and that, I am afraid, is very optimistic. To mitigate that, we have taken the terminal year a little conservatively as 2028. The company has a set of major patent families from Dartmouth, and just last year, it acquired its own patent covering allogenic modules. There are a number of pending patents as well. It can also be argued that a drug+facility setup needs more than patent expiry to be challenged, because even if patent expiry leads to a copycat drug, the technical knowhow behind the facility needed for the autologous CAR-T production may be a trade secret. Given that, I believe 2028 is overly conservative and rightly compensates our early approval date. Now, the assumptions below.

This valuation is based on the expected solid tumor market size of $43 billion by 2020. A large bulk of this global market consists of the US market, where CYAD will launch its product. Keeping this in view, we have assumed the initial market penetration to be 3% of the global market size. The company is expected to grow its market share by 2 percent per annum, while the overall global market is expected to grow by 8% annually. We have assumed the operating margin at 25% which is in line with industry standards and provides for slightly higher expenses due to the additional costs surrounding autologous therapy. Further, free cash flow has been assumed to be 85% of the operating income.

The cash flows have been discounted at 10%. Also, the cash flows are risk adjusted to account for the fact that the drug is still under testing phase. The probability of the drug approval has been set at 30%, which is purely based on our study of the trial results so far. The double discounted cash flow is used for arriving at the present value of the funds. The per share valuation is thus found by dividing the present value by the total number of shares currently outstanding. It has been assumed that the company will not be issuing any new equity in the near future but can continue with Phase III based on collaborations and other revenue channels besides dilution. Based on these assumptions, the fair value of the stock has been arrived at $108.25 which shows over 125% upside from its current value.

Investment Strategy and catalysts

The stock is trading quite near its 52-week high after two key positive news came out in March, one about a patent being upheld by the USPTO, and the other the FDA's nod to begin the THINK clinical trial. While I see considerable upside to the stock, the window is quite large; meaning, we have a lot of time. So, while we could take a small position right now, we should wait for any dip in price to accumulate. I don't see any dilution risk in the next couple years either; however, this is early stage technology, so while investing at this stage has its rewards, there's always a risk. To mitigate that risk, follow the basic rule of healthcare retail investing - whenever the stock presents a profit-taking opportunity, take profit. And, an associated personal rule that I always follow, sell out before a catalyst. My rationale is: a catalyst's result, such as a PDUFA, is never certain. But bullish expectations before a major catalyst often buoy the stock. So, to reduce risks but still make a decent profit, pre-catalyst sell-out of most of one's holdings is important. Also, consider that stocks often go down sometime after approval and remain depressed until very good market uptake figures come through - and that takes a quarter or two; for example, consider Acadia (NASDAQ:ACAD) and its nuplazid. Given all that, Celyad should be mostly a catalyst play with only a small tranche held out for beyond approval scenarios.

Competition and risks

The NKR-2 ligands are sometimes, although very rarely, seen in healthy cells. So, there is a chance of off-target toxicity. This can be enhanced by certain drugs that help overexpress NKR-2. While these drugs may aid in the efficacy of Celyad's therapy, they may also add to the off-target toxicity. That is something we haven't seen in these early trials, but we will have to see how the larger and better powered trials go.

Another issue is competition. Bellicum (NASDAQ:BLCM) has its BPX-601 program in early stages, and this also comes with its proprietary rimiducid-controlled switch. The approach here is via the prostate stem cell antigen, or PSCA route; and CYAD's NKG2D route has its advantages, having broader expression. Juno (NASDAQ:JUNO) also has a modest program in a few solid tumor cancers. While Juno is much higher valued than CYAD, BLCM is close. Here, the slight edge that CYAD has, besides its NKR-2 angle, is its cardio program which can generate revenue for the company.

Dilution is the third risk here. The company says it has cash until 2019. A large scale Phase III trial will cost a few hundred millions for a CAR-T trial, if I am not mistaken. The two deals it has right now together do not make up that amount before Phase III as far as I understand. However, if Phase II is successful, I expect more intense deal-making.

Bottom line

As with all early stage biotech, everything is speculative here. However, the idea of using CAR-T constructs in solid tumor is itself revolutionary. Moreover, the Dartmouth College association, the IP portfolio, the solid results with low toxicity in early trials in vivo and in the clinic, and lastly the technology surrounding NKR-2 all point to a promising future for the company. I would buy this stock for the long term, reduce my acquisition cost as opportunities present themselves, and play the catalysts as they come around the PDUFA.

Disclosure: I am/we are long CYAD, BLCM.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Continue reading here:
Buy Celyad And Get In On The CAR-T Space In Solid Tumors: Medicine's Next Frontier - Seeking Alpha

June 26, 2017 In this image of neurons in the cerebellum of the brain, the yellow cells are Purkinje cells in which the channelrhodopsin-2 gene is being produced. Credit: Horwitz Lab/UW Medicine Seattle

UW Medicine researchers have developed a technique for inserting a gene into specific cell types in the adult brain in an animal model.

Recent work shows that the approach can be used to alter the function of brain circuits and change behavior. The study appears in the journal Neuron in the NeuroResources section.

Gregory Horwitz, associate professor of physiology and biophysics at the University of Washington School of Medicine in Seattle, led the research team. He said that the approach will allow scientists to better understand what roles select cell types play in the brain's complex circuitry.

Researchers hope that the approach might someday lead to developing treatments for conditions, such as epilepsy, that might be curable by activating a small group of cells.

"The brain is made up of a mix of many cell types performing different functions. One of the big challenges for neuroscience is finding ways to study the function of specific cell types selectively without affecting the function of other cell types nearby," Horwitz said. "Our study shows it is possible to selectively target a specific cell type in an adult brain using this technique and affect behavior nearly instantly."

In their study, Horowitz and his colleagues at the Washington National Primate Research Center in Seattle inserted a gene into cells in the cerebellum, a small structure located at the back of the brain and tucked under the brain's larger cerebrum.

The cerebellum's primary function is controlling motor movements. Disorders of the cerebellum generally lead to often disabling loss of coordination. Recent research suggests the cerebellum may also be important in learning and may be involved in such conditions as autism and schizophrenia.

The cells the scientists selected to study are called Purkinje cells. These cells, named after their discoverer, Czech anatomist Jan Evangelista Purkinje, are some of the largest in the human brain. They typically make connections with hundreds of other brain cells.

"The Purkinje cell is a mysterious cell," said Horwitz. "It's one of the biggest and most elaborate neurons and it processes signals from hundreds of thousands of other brain cells. We know it plays a critical role in movement and coordination. We just don't know how."

The gene they inserted, called channelrhodopsin-2, encodes for a light-sensitive protein that inserts itself into the brain cell's membrane. When exposed to light, it allows ions - tiny charged particles - to pass through the membrane. This triggers the brain cell to fire.

The technique, called optogenetics, is commonly used to study brain function in mice. But in these studies, the gene must be introduced into the embryonic mouse cell.

"This 'transgenic' approach has proved invaluable in the study of the brain," Horwitz said. "But if we are someday going to use it to treat disease, we need to find a way to introduce the gene later in life, when most neurological disorders appear."

The challenge for his research team was how to introduce channelrhodopsin-2 into a specific cell type in an adult animal. To achieve this, they used a modified virus that carried the gene for channelrhodopsin-2 along with segment of DNA called a promoter. The promoter stimulates the cell to start expressing the gene and make the channelrhodopsin-2 membrane protein. To make sure the gene was expressed only by Purkinje cells, the researchers used a promoter that is strongly active in Purkinje cells, called L7/Pcp2."

In their paper, the researchers reported that by painlessly injecting the modified virus into a small area of the cerebellum of rhesus macaque monkeys, the channelrhodopsin-2 was taken up exclusively by the targeted Purkinje cells. The researchers then showed that when they exposed the treated cells to light through a fine optical fiber, they were able stimulate the cells to fire at different rates and affect the animals' motor control.

Horwitz said that it was the fact that Purkinje cells express L7/Pcp2 promoter at a higher rate than other cells that made them more likely to produce the channelrhodopsin-2 membrane protein.

"This experiment demonstrates that you can engineer a viral vector with this specific promoter sequence and target a specific cell type," he said. "The promoter is the magic. Next, we want to use other promoters to target other cell types involved in other types of behaviors."

Explore further: New insights into control of neuronal circuitry could lead to treatments for an inherited motor disorder

More information: Yasmine El-Shamayleh et al, Selective Optogenetic Control of Purkinje Cells in Monkey Cerebellum, Neuron (2017). DOI: 10.1016/j.neuron.2017.06.002

Journal reference: Neuron

Provided by: University of Washington

While the definitive causes remain unclear, several genetic and environmental factors increase the likelihood of autism spectrum disorder, or ASD, a group of conditions covering a "spectrum" of symptoms, skills and levels ...

Many cognitive processes, such as decision-making, take place within seconds or minutes. Neuroscientists have longed to capture neuron activity during such tasks, but that dream has remained elusiveuntil now.

Many genes linked to late-onset Alzheimer's disease (AD) are expressed in myeloid cells and regulated by a single protein, according to research conducted at the Icahn School of Medicine at Mount Sinai and published June ...

Neuroscientists from the University of Chicago have developed a computer model that can simulate the response of nerves in the hand to any pattern of touch stimulation on the skin. The tool reconstructs the response of more ...

Viruses have evolved to be highly effective vehicles for delivering genes into cells. Seeking to take advantage of these traits, scientists can reprogram viruses to function as vectors, capable of carrying their genetic cargo ...

Since scientists began studying the brain, they have asked whether the biology they observed can really be tied to external behaviors. Researchers are building a substantial understanding of the biophysical, molecular, and ...

Adjust slider to filter visible comments by rank

Display comments: newest first

Epilepsy's cause is doubtless pheromonal.

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

More here:
Study shines light on brain cells that coordinate movement - Medical Xpress

Dr. Purita has been a pioneer in the Regenerative Medicine space for over a decade.

Boca Raton, Florida (PRWEB) June 27, 2017

Robert J. Colucci CEO of PuRxCell, LLC announced today that PuRxCell, a Florida based company, has been established to train physicians at all levels of experience and their staffs in Dr. Puritas unique Stem Cell treatment and processing protocols. PuRxCell also provides a comprehensive line of products to support these cutting edge protocols.

Dr. Purita has been a pioneer in the Regenerative Medicine space for over a decade, said CEO R.J. Colucci. He continued, He has performed more than 8500 treatment procedures without serious adverse effect. These unique protocols not only improve treatment outcomes but also significantly reduce the cost of the treatment and increase margins, which is important in the world of increasing competition and reduced insurance reimbursements. Colucci said, Our mission at Purxcell is simple: Deliver individualized training to physicians - offer cutting edge treatment and processing protocols and products - lower the cost of treatment and provide physicians and their staffs a continuum of training and support."

In my 35 years as an Orthopedic Surgeon, I have never been as excited as I am over the advancements in Regenerative Medicine. Over the last 10 years I have focused my efforts on developing and refining cutting edge PRP and Stem Cell procedures and adjunct therapies, said Joseph Purita, PuRxCells Chief Medical Officer.

Purita continued, I have been hesitant to pass on specific details about my experiences and unique protocols. However, I have come to the conclusion that I want some of my colleagues and ultimately their patients to benefit from what I have learned. As such, PuRxCell is offering direct physician training as well as access to my proprietary treatment protocols and products. Purita noted, I was also unhappy with the current costs of disposable products for Stem Cell treatment protocols. I have expended considerable effort to finding ways to lower procedure and disposable costs while at the same time improving the quality of patient outcomes. All this eventually led me to form a dedicated, full service Regenerative Medicine company, PuRxCell. We have developed blood, adipose and bone marrow processing products designed to help customers dramatically reduce cost without sacrificing treatment outcomes.

Founded in 2016, Purxcell has multiple level of training programs tailored to individual physicians needs and experience levels. Physician training ranges from 2-5 days, while training for lab staff typically ranges from 1-2 days. PuRxCell also offers Regenerative Medicine practice and marketing support in line with the mission of providing full-service Regenerative Medicine products and services.

PuRxCell has its corporate headquarters office in Boca Raton, Florida, with satellite offices in Colorado and Coconut Creek, FL. More information about PuRxCell can be found at http://www.purxcell.com.

For additional information contact: Robert Colucci at r.colucci(at)purxcell(dot)com or at 877-498-5500 ext. 1

Share article on social media or email:

Go here to see the original:
Regenerative Medicine Pioneer Offers Comprehensive Stem Cell Training - PR Web (press release)

June 26, 2017 Credit: CC0 Public Domain

Cancer tumours manipulate a natural cell process to promote their survival suggesting that controlling this mechanism could stop progress of the disease, according to new research led by the University of Oxford.

Non-sense mediated decay (NMD) is a natural physiological process that provides cells with the ability to detect DNA errors called nonsense mutations. It also enables these cells to eliminate the mutated message (decay) that comes from these faulty genes, before they can be translated into proteins that can cause disease formation. NMD is known among the medical community for the role it plays in the development of genetic diseases such as Cystic Fibrosis and some hereditary forms of cancers. But not all nonsense mutations can elicit NMD, so until now, it's wider impact on cancer was largely unknown.

Biomedical researchers and computer scientists from the University of Oxford Medical Sciences Division and the University of Birmingham developed a computer algorithm to mine DNA sequences from cancer to accurately predict whether or not an NMD would eliminate genes that had nonsense mutations. The work originally focused on ovarian cancers, and found that about a fifth of these cancers use NMD, to become stronger. This is because NMD ensures that the message from a gene called TP53, which ordinarily protects cells from developing cancer is almost completely eliminated. In the absence of NMD, a mutated TP53 might still retain some activity but NMD ensures that this is not the case.

Based on this research, the team predicts that because cancers essentially feed on NMD, they become dependent on it in some cases. If scientists were therefore able to inhibit or control the process, it is possible that they could also control cancer and prevent the progression of the disease.

Dr Ahmed Ahmed, Co-author and Professor of Gynaecology Oncology at the Nuffield Department of Obstetrics & Gynaecology and the head of the Ovarian Cancer Cell Laboratory, at the Weatherall Institute of Molecular Medicine at the University of Oxford, said: "Our first observations of evidence of the role of NMD in ovarian cancer were tantalizing. We found that NMD precisely explained why there was almost no expression of TP53 in certain ovarian cancers. We went on to test the role of NMD in other cancer types and the evidence of the role of NMD was compelling. This opens the door for exciting possibilities for customised treatments including individualized immunotherapies for patients in the future."

Following the ovarian cancer analysis, the team expanded the study to include other cancer types. They analysed about a million different cell mutations in more than 7,000 tumours from the Cancer Genome Atlas covering 24 types of cancer. The team was able to map how each cancer type used NMD revealing the remarkable extent to which NMD helps cancer to survive.

Katherine Taylor, CEO of Ovarian Cancer Action, who part-funded the research, said: "This is very exciting news. Professor Ahmed and his team have identified how cancer cells rely on a process called NMD for their survival. This discovery could help clinicians identify and inhibit the process, giving them much better control of a person's cancer.

"Ovarian cancer is a very complicated disease and survival rates are low, with only 46% of women living beyond five years after diagnosis. So understanding how we can prevent the disease from thriving is imperative if we are to improve the outcome for more women.

"It's fantastic to see how our funding is helping make real progress and we couldn't do this without the generosity of our supporters. We look forward to seeing where Professor Ahmed takes his research next."

Moving forward the team will focus on testing their theory and understanding to what degree stopping the NMD process allows them to control tumours.

Co-author, Dr Christopher Yau, a computational scientist at the Institute of Cancer and Genomic Sciences, University of Birmingham said: "As a result of these findings, we now plan to apply the same computer algorithm to determine if NMD affects cancer patients in The 100,000 Genomes Project. These investigations may pave the way to new treatment possibilities for NHS patients in the future."

Explore further: Two Oxford research discoveries offer hope for managing ovarian cancer

More information: The full paper citation is 'A pan-cancer genome-wide analysis reveals tumour dependencies by induction of nonsense-mediated decay,' and it will be published in Nature Communications on Monday 26 June 2017.

Oxford University researchers have found a way to detect ovarian cancer early and identified an enzyme that is key in making ovarian cancer more deadly. Their results, published in two journals, provide new research routes ...

Levels of circulating tumor DNA (ctDNA) detected in a blood test are correlated with the size of ovarian cancers and can predict a patient's response to treatment or time to disease progression, according to a retrospective ...

Testing for a gene commonly mutated in ovarian cancers could pick out patients who will respond well to a promising new class of cancer drugs, a major new study reveals.

A recent discovery by researchers from the Cancer Science Institute of Singapore (CSI Singapore) at the National University of Singapore (NUS) may lead to a new treatment strategy for an aggressive ovarian cancer subtype.

A new discovery that sheds light on the genetic make up of ovarian cancer cells could explain why some women survive longer than others with this deadly disease. A multi-disciplinary team led by the Research Institute of ...

Mutations in the BRCA1 gene are one of the most common risk factors for breast and ovarian cancers. Although tumors that harbor BRCA1 mutations initially respond well to cancer treatments, many tumors eventually become less ...

With 20,000 diagnoses each year, ovarian cancer is the ninth most common cancer and fifth leading cause of cancer death among women in the United States. So many women die from ovarian cancer because it often goes undetected ...

Two challenges in treating patients with estrogen-positive breast cancer (ER+) have been an inability to predict who will respond to standard therapies and adverse events leading to therapy discontinuation. A study at The ...

Cancer tumours manipulate a natural cell process to promote their survival suggesting that controlling this mechanism could stop progress of the disease, according to new research led by the University of Oxford.

An international research team has found a way to improve the anti-cancer effect of a new medicine class called 'Smac mimetics'.

A subgroup of patients with osteosarcoma - a form of bone cancer - could be helped by an existing drug, suggest scientists from the Wellcome Trust Sanger Institute and their collaborators at University College London Cancer ...

Some patients with a form of advanced kidney cancer that carries a poor prognosis benefited from an experimental drug targeted to an abnormal genetic pathway causing cancerous growth, according to research led by a Dana-Farber ...

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

See the original post here:
Cancer hijacks natural cell process to survive - Medical Xpress - Medical Xpress

June 26, 2017 PET scan of a human brain with Alzheimer's disease. Credit: public domain

Many genes linked to late-onset Alzheimer's disease (AD) are expressed in myeloid cells and regulated by a single protein, according to research conducted at the Icahn School of Medicine at Mount Sinai and published June 19 in the journal Nature Neuroscience.

Mount Sinai researchers led an international, genome-wide study of more than 40,000 people with and without the disease and found that innate immune cells of the myeloid lineage play an even more central role in Alzheimer's disease pathogenesis than previously thought.

Specifically, the research team identified a network of genes that are implicated in AD and expressed by myeloid cells, innate immune cells that include microglia and macrophages. Furthermore, researchers identified the transcription factor PU.1, a protein that regulates gene expression and, thus, cell identity and function, as a master regulator of this gene network.

"Our findings show that a large proportion of the genetic risk for late-onset AD is explained by genes that are expressed in myeloid cells, and not other cell types," says Alison Goate, DPhil, Professor of Neuroscience and Director of The Ronald M. Loeb Center for Alzheimer's Disease at the Icahn School of Medicine at Mount Sinai and principal author of the study. "Dysregulation of this network is certainly a cause of Alzheimer's, but we have more work to do to better understand this network and regulation by PU.1, to reveal promising therapeutic targets."

Using a combination of genetic approaches to analyze the genomes of 14,406 AD patients, and 25,849 control patients who do not have the disease, researchers found that many genes which are known to influence the age at which AD sets in, are expressed in myeloid cells. This work pinpointed SPI1, a gene that encodes the transcription factor PU.1, as a major regulator of this network of AD risk genes and demonstrated that lower levels of SPI1/PU.1 are associated with later age at onset of AD.

To test the hypothesis that SPI1 expression levels influence expression of other AD risk genes and microglial function, the researchers used a mouse microglial cell line, BV2 cells that can be cultured in a dish. When researchers knocked down expression of SPI1, the gene that produces PU.1 in cells, they found that the cells showed lower phagocytic activity (engulfment of particles), while overexpression of SPI1 led to increased phagocytic activity. Many other AD genes expressed in microglia also showed altered expression in response to this manipulation of SPI1 expression.

"Experimentally altering PU.1 levels correlated with phagocytic activity of mouse microglial cells and the expression of multiple AD genes involved in diverse biological processes of myeloid cells," says Dr. Goate. "SPI1/PU.1 expression may be a master regulator capable of tipping the balance toward a neuroprotective or a neurotoxic microglial function."

The researchers stress that because the PU.1 transcription factor regulates many genes in myeloid cells, the protein itself may not be a good therapeutic target. Instead, further studies of PU.1's role in microglia and AD pathogenesis are necessary, as they may reveal promising downstream targets that may be more effective in modulating AD risk without broad effects on microglial function. Increased understanding is crucial to facilitating the development of novel therapeutic targets for a disease that currently has no cure.

Explore further: Phagocytes in the braingood or bad?

More information: Kuan-lin Huang et al. A common haplotype lowers PU.1 expression in myeloid cells and delays onset of Alzheimer's disease, Nature Neuroscience (2017). DOI: 10.1038/nn.4587

The role of microglial cells in neurodegenerative disease is not fully understood. But new results from researchers in Munich and Basel suggest that stimulation of this arm of the immune system might well delay the onset ...

Scientists have, for the first time, characterized the molecular markers that make the brain's front lines of immune defensecells called microgliaunique. In the process, they discovered further evidence that microglia ...

Using human skin cells, University of California, Irvine neurobiologists and their colleagues have created a method to generate one of the principle cell types of the brain called microglia, which play a key role in preserving ...

On diagnosis of acute myeloid leukemia, the mesenchymal stem cells (MSCs) in bone marrow often show alterations in gene and protein expression, proliferation capacity, and function, but whether these are a cause or result ...

Glioblastoma is an extremely aggressive brain tumor with limited treatment options. Recent progress in using immunotherapy-based treatment options in other tumor types has spurred interest in developing approaches that might ...

While the definitive causes remain unclear, several genetic and environmental factors increase the likelihood of autism spectrum disorder, or ASD, a group of conditions covering a "spectrum" of symptoms, skills and levels ...

Many cognitive processes, such as decision-making, take place within seconds or minutes. Neuroscientists have longed to capture neuron activity during such tasks, but that dream has remained elusiveuntil now.

Many genes linked to late-onset Alzheimer's disease (AD) are expressed in myeloid cells and regulated by a single protein, according to research conducted at the Icahn School of Medicine at Mount Sinai and published June ...

Neuroscientists from the University of Chicago have developed a computer model that can simulate the response of nerves in the hand to any pattern of touch stimulation on the skin. The tool reconstructs the response of more ...

Viruses have evolved to be highly effective vehicles for delivering genes into cells. Seeking to take advantage of these traits, scientists can reprogram viruses to function as vectors, capable of carrying their genetic cargo ...

Since scientists began studying the brain, they have asked whether the biology they observed can really be tied to external behaviors. Researchers are building a substantial understanding of the biophysical, molecular, and ...

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Here is the original post:
Alzheimer's disease risk linked to a network of genes associated with myeloid cells - Medical Xpress

The single-cell analysis market is expected to reach USD 3.59 billion by 2022 from USD 1.67 billion in 2017, at a CAGR of 16.5%.

Technological advancements in single-cell analysis products, increasing government funding for cell-based research, growing biotechnology and biopharmaceutical industries, wide applications of single-cell analysis in cancer research, growing focus on personalized medicine, and increasing incidence and prevalence of chronic and infectious diseases are driving the growth of the single-cell analysis market.

On the other hand, the high cost of single-cell analysis instruments may hinder the growth of the market in the coming years.

Consumables segment are expected to witness high growth during forecast period

Based on product, the single-cell analysis market is segmented into consumables and instruments.

Consumables are expected to register the highest CAGR during the forecast period.

Consumables are further segmented into beads, microplates, reagents, assay kits, and other consumables, whereas, instruments are further segmented into flow cytometers, NGS systems, PCR instruments, HCS systems, microscopes, cell counters, spectrophotometers, cell microarrays, and other instruments.

Players from the market are increasingly focusing on technological advancements and product development to launch efficient cytometry instruments which is driving the growth of the segment.

Human cells to dominate the single-cell analysis market

Based on the type of cells, the single-cell analysis market is segmented into human, animal, and microbial cells.

The human cells segment is expected to dominate this market with the largest share in 2017.

The large share of this segment can be attributed to the growing application areas of human stem cells and rising incidence of diseases such as cancer.

"Single-cell analysis market projected to register a CAGR of 16.5%" The single-cell analysis market is expected to reach USD 3.59 billion by 2022 from USD 1.67 billion in 2017, at a CAGR of 16.5%. Technological advancements in single-cell analysis products, increasing government funding for cell-based research, growing biotechnology and biopharmaceutical industries, wide applications of single-cell analysis in cancer research, growing focus on personalized medicine, and increasing incidence and prevalence of chronic and infectious diseases are driving the growth of the single-cell analysis market. On the other hand, the high cost of single-cell analysis instruments may hinder the growth of the market in the coming years.

"Consumables segment are expected to witness high growth during forecast period" Based on product, the single-cell analysis market is segmented into consumables and instruments. Consumables are expected to register the highest CAGR during the forecast period. Consumables are further segmented into beads, microplates, reagents, assay kits, and other consumables, whereas, instruments are further segmented into flow cytometers, NGS systems, PCR instruments, HCS systems, microscopes, cell counters, spectrophotometers, cell microarrays, and other instruments. Players from the market are increasingly focusing on technological advancements and product development to launch efficient cytometry instruments which is driving the growth of the segment.

"Human cells to dominate the single-cell analysis market" Based on the type of cells, the single-cell analysis market is segmented into human, animal, and microbial cells. The human cells segment is expected to dominate this market with the largest share in 2017. The large share of this segment can be attributed to the growing application areas of human stem cells and rising incidence of diseases such as cancer.

"NGS to account with the highest growth rate in the single-cell analysis market" Based on technique, the single-cell analysis market is segmented into flow cytometry, NGS, PCR, microscopy, mass spectrometry, and other techniques (including single-molecule fluorescence in situ hybridization, micromanipulation, and automated capillary electrophoresis). NGS segment is projected to grow at the highest rate during the forecast period.

"North America to dominate the market during forecast period" In 2017, North America is expected to account for the largest share of the single-cell analysis market. Factors such as increasing collaborations among prominent players, technological advancements and expanding biotechnology and pharmaceutical industries are supporting the growth of the single-cell analysis market in this region. Asian region is expected to register the highest CAGR during the forecast period. The large population in China and India, rising geriatric population, and increasing incidence of chronic diseases are the major factors driving the growth of the Asian market.

The primary interviews conducted for this report can be categorized as follows:

By Company Type: Tier 1 35%; Tier 2, 40%; Tier 3, 25%. By Designation: C- level- 35%; D-level- 25%; others- 40%. By Region: North America 43%; Europe- 19%; Asia- 29%; and Rest of the World (RoW) - 9%

*Note: Others include sales managers, marketing managers, and product managers Tiers of the companies are defined by their total revenue. As of 2016: Tier 1 = > USD 1 billion, Tier 2 = USD 100 million to USD 1 billion, and Tier 3 = < USD 100 million

List of Companies Benchmarked in the report

Merck KGaA (Germany) Becton, Dickinson and Company (U.S.) Promega Corporation (U.S.) Danaher Corporation (U.S.) General Electric Company (U.K.) Thermo Fisher Scientific Inc. (U.S.) Miltenyi Biotec (Germany) Illumina, Inc. (U.S.) Bio-Rad Laboratories, Inc.(U.S.) Fluidigm Corporation (U.S.) NanoString Technologies, Inc. (U.S.) Agilent Technologies (U.S.) Abcam Plc (U.S.) NuGEN Technologies Inc.(U.S.) LumaCyte (U.S.) PluriSelect Life Science UG & Co. KG (Germany) Sysmex Partec (U.S.) Bio-Techne Corporation (U.S.) Promega Corporation (U.S.) 10x Genomics (U.S.) WaferGen Bio-systems, Inc. (U.S.) Bruker (U.S.) Fluxion Biosciences (U.S.).

Research Coverage: The report provides a picture on global single-cell analysis across various medical devices. It aims at estimating the market size and future growth potential of this market across different segments such as products, cell type, technique, application, end user, and regions. Furthermore, the report also includes an in-depth competitive analysis of the key players in the market along with their company profiles, recent developments, and key market strategies.

Key Benefits of Buying the Report: The report will help the market leaders/new entrants in this market by providing them the closest approximations of the revenue numbers for the overall single-cell analysis market and the subsegments. This report will help stakeholders to better understand the competitor landscape and gain more insights to better position their businesses and make suitable go-to-market strategies. The report also helps the stakeholders to understand the pulse of the market and provides them information on key market drivers, restraints, challenges, and opportunities. Read the full report: http://www.reportlinker.com/p04579530/Single-Cell-Analysis-Market-by-Product-Cell-Type-Technique-Application-End-User-Global-Forecasts-to.html

About Reportlinker ReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

http://www.reportlinker.com

__________________________ Contact Clare: clare@reportlinker.com US: (339)-368-6001 Intl: +1 339-368-6001

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/single-cell-analysis-market-is-expected-to-reach-usd-359-billion-by-2022-300479702.html

SOURCE Reportlinker

http://www.reportlinker.com

Read the original post:
Single-cell Analysis Market is expected to reach USD 3.59 billion by 2022 - PR Newswire (press release)