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Novartis to Invest $35M in Gamida Cell for 15% Equity – Analyst Blog

Posted: August 21, 2014 at 1:40 am

Novartis ( NVS ) recently entered into an investment and option agreement with Israel-based Gamida Cell, a company which focuses on stem cell expansion technologies and therapeutic products.

As per the terms of the agreement, Novartis will invest $35 million in Gamida Cell. In exchange, Novartis will receive a 15% stake in Gamida Cell and an option to fully acquire the company.

The option for full acquisition is exercisable for a limited period of time following achievement of certain milestones in connection with the development of pipeline candidate, NiCord. These milestones are expected to be achieved during 2015. Novartis will also be required to pay the other shareholders in Gamida Cell approximately $165 million upon exercising the option along with potential milestone payments of $435 million.

We note that Gamida Cell is developing stem cell therapy for the potential treatment of blood cancers, solid tumors, non-malignant hematological diseases such as sickle cell disease and thalassemia, neutropenia and acute radiation syndrome, autoimmune diseases and genetic metabolic diseases as well as conditions that can be helped by regenerative medicine.

The company is currently evaluating NiCord for the potential treatment of hematological malignancies such as leukemia and lymphoma in a phase I/II study using its proprietary NAM technology.

Meanwhile, enrolment is on for the company's phase I/II study on NiCord for pediatric sickle cell disease.

We remind investors that Novartis has been taking strategic steps to realign its portfolio in order to focus on its core portfolio of pharmaceuticals, eye care and generics. Novartis' recent deal to acquire oncology products from GlaxoSmithKline ( GSK ) and the divestiture of the Vaccines business is a step in the right direction.

Novartis, a large-cap pharma, currently carries a Zacks Rank #3 (Hold). Right now, Allergan ( AGN ) and AbbVie ( ABBV ) look well positioned among the large-cap pharmas. While Allergan carries a Zacks Rank #1 (Strong Buy), AbbVie is a Zacks Rank #2 (Buy) stock.

NOVARTIS AG-ADR (NVS): Free Stock Analysis Report

ABBVIE INC (ABBV): Free Stock Analysis Report

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Novartis to Invest $35M in Gamida Cell for 15% Equity - Analyst Blog

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In search of a 'virtual cure' for diabetes

Posted: August 20, 2014 at 12:48 pm

Paul Laikind, CEO of ViaCyte, which is making a treatment for diabetes from human embryonic stem cells.

San Diego-based ViaCyte has received FDA permission to try its stem cell-based diabetes therapy. The Phase 1/2 combination trial will look for both safety and early signs of efficacy.

ViaCyte grows replacement insulin-producing cells from human embryonic stem cells, which are placed in a semipermeable pouch. The pouch will be implanted into patients, allowing insulin and other hormones to enter the patient's bloodstream.

The combination product of pouch and cells is called VC-01. ViaCyte says the product has the potential to provide a "virtual cure" for Type 1 diabetes. The company announced last month it had applied to begin the clinical trial.

Animal trials have shown that the replacement cells successfully duplicate the function of the insulin-producing beta cells. These cells secrete not only insulin, which lowers blood sugar, but hormones such as glucagon, which raises it. Providing a range of hormones as in the natural pancreas is expected to provide better control of blood sugar than with insulin alone.

The green light is not only good news for privately held ViaCyte, but for California's stem cell agency, the California Institute for Regenerative Medicine. The agency has granted ViaCyte more than $38 million to research and develop the treatment. CIRM has been under pressure in recent years to show that its $3 billion in state bond funding is leading to therapies.

CIRM was created to help develop stem cell treatments for diseases that are currently incurable with traditional approaches, C. Randal Mills president and CEO of the stem cell agency, said in an agency statement. Working in collaboration with CIRM for the past 6 years, ViaCyte has developed a particularly novel approach to overcome the challenges of treating type 1 diabetes. Anytime a product, particularly one as innovative as this one, progresses from the lab and into clinical trials its very encouraging news, particularly for the patients suffering from the disease.

The ViaCyte team is very pleased to have received FDA acceptance for our clinical trial protocol and look forward to initiating this study shortly, ViaCyte president and CEO Paul Laikind said in the statement. The commencement of this trial marks a significant milestone that could not have been achieved without the support we have received and continue to receive from CIRM.

Inadequate control of blood sugar increases the risk of heart disease, stroke, kidney failure and other complications of diabetes.

The ViaCyte product contains immature beta cells, grown from the embryonic stems cells. After implantation, the cells mature and begin to release the appropriate hormones in response to blood sugar levels.

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Zebrafish help to unravel Alzheimer's disease

Posted: August 19, 2014 at 9:48 pm

PUBLIC RELEASE DATE:

19-Aug-2014

Contact: Evgenia Salta Evgenia.Salta@cme.vib-kuleuven.be 32-163-77957 VIB (the Flanders Institute for Biotechnology)

New fundamental knowledge about the regulation of stem cells in the nerve tissue of zebrafish embryos results in surprising insights into neurodegenerative disease processes in the human brain. A new study by scientists at VIB and KU Leuven identifies the molecules responsible for this process.

Zebrafish as a model

The zebrafish is a small fish measuring 3 to 5 cm in length, with dark stripes along the length of its body. They are originally from India, but also a popular aquarium fish. Zebrafish have several unusual characteristics that make them popular for scientific research. Zebrafish eggs are fertilized outside the body, where they develop into embryos. This process occurs very quickly: the most important organs have formed after 24 hours and the young fish have hatched after 3 days. These fish are initially transparent, making them easy to study under the microscope. Zebrafish start reproducing after only 3 months. The genetic code of humans and zebrafish is more than 90 % identical. In addition, the genetic material of these fish is easy to manipulate, meaning that they are often used as a model in the study of all sorts of diseases.

Stem cells in the brain

Evgenia Salta, scientist in the team of Bart De Strooper (VIB KU Leuven), used zebrafish as a model in molecular brain research and discovered a previously unknown regulatory process for the development of nerve cells. Evgenia Salta explains: "The human brain contains stem cells, which are cells that have not matured into nerve cells yet, but do have the potential to do this." Stem cells are of course crucial in the development of the brain. Similar stem cells also exist in zebrafish. Therefore, these fish form an ideal model to study the behavior of these cells. A so-called Notch signaling pathway regulates the further ripening of these cells during early embryonic development. Scientists are still largely in the dark about Notch processes in the brains of Alzheimer patients, but the research by Evgenia Salta is changing this situation.

MicroRNA

The expression of genes, which form the basis of the Notch signaling pathway, is regulated in part by microRNAs (miRNAs), which are short molecules that can inhibit or activate genes. Evgenia Salta: "We specifically studied how miRNA-132 regulates the Notch signaling pathway in stem cells."

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Stem Cell Research Holy Grail' Uncovered, Thanks to Zebrafish

Posted: August 19, 2014 at 9:48 pm

By Estel Grace Masangkay

With help from the zebrafish, a team of Australian researchers has uncovered how hematopoietic stem cells (HSC) renew themselves, considered by many to be the holy grail of stem cell research.

HSCs are a significant type of stem cell present in the blood and bone marrow. These are needed for the replenishment of the bodys supply of blood and immune cells. HSCs already play a part in transplants in patients with blood cancers such as leukemia and myeloma. The stem cells are also studied for their potential to transform into vital cells including muscle, bone, and blood vessels.

Understanding how HSCs form and renew themselves has potential application in the treatment of spinal cord injuries, degenerative disorders, even diabetes. Professor Peter Currie, of the Australian Regenerative Medicine Institute at Victorias Monash University, led a research team to discover a crucial part of HSCs development. Using a high-resolution microscopy, Prof. Curies team caught HSCs on film as they formed inside zebrafish embryos. The discovery was made while the researchers were studying muscle mutations in the aquatic animal.

Zebrafish make HSCs in exactly the same way as humans do, but whats special about these guys is that their embryos and larvae develop free living and not in utero as they do in humans. So not only are these larvae free-swimming, but they are also transparent, so we could see every cell in the body forming, including HSCs, explained Prof. Currie.

While playing the film back, the researchers noticed that a buddy cell came along to help the HSCs form. Called endotome cells, they aided pre-HSCs to turn into HSCs. Prof. Currie said, Endotome cells act like a comfy sofa for pre-HSCs to snuggle into, helping them progress to become fully fledged stem cells. Not only did we identify some of the cells and signals required for HSC formation, we also pinpointed the genes required for endotome formation in the first place.

The next step for the researchers is to locate the signals present in the endotome cells that trigger HSC formation in the embryo. This can help scientists make different blood cells on demand for blood-related disorders. Professor Currie also pointed out the discoverys potential for correcting genetic defects in the cell and transplanting them back in the body to treat disorders.

The teams work was published in the international journal Nature.

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Blood Cells Generate Neurons in Crayfish; Could Have Implications for Treatment of Neurodegenerative Disorders

Posted: August 18, 2014 at 11:54 pm

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Newswise A new study by Barbara Beltz, the Allene Lummis Russell Professor of Neuroscience at Wellesley College, and Irene Sderhll of Uppsala University, Sweden, published in the August 11 issue of the journal Developmental Cell, demonstrates that the immune system can produce cells with stem cell properties, using crayfish as a model system. These cells can, in turn, create neurons in the adult animal. The flexibility of immune cells in producing neurons in adult animals raises the possibility of the presence of similar types of plasticity in other animals.

We have been suspicious for some time that the neuronal precursor cells (stem cells) in crayfish were coming from the immune system, Beltz wrote. The paper contains multiple lines of evidence that support this conclusion, in addition to the experiments showing that blood cells transferred from a donor to a recipient animal generate neurons.

Beltz, whose research focuses on the production of new neurons in the adult nervous system, uses the crustacean brain as the model system because the generations of precursor cells are spatially segregated from one another. According to Beltz, this separation is crucial because it allowed the researchers to determine that the first generation precursors do not self-renew. For the Developmental Cell study, the cells of one crayfish were labeled and this animals blood was used for transfusions into another crayfish. They found that the donor blood cells could generate neurons in the recipient.

In many adult organisms, including humans, neurons in some parts of the brain are continually replenished. While this process is critical for ongoing health, dysfunctions in the production of new neurons may also contribute to several neurological diseases, including clinical depression and some neurodegenerative disorders.

Beltz notes, of course, that it is difficult to extrapolate from crayfish to human disease. However, because of existing research suggesting that stem cells harvested from bone marrow also can become neural precursors and generate neurons, she says it is tempting to suggest that the mechanism proposed in crayfish may also be applicable in evolutionarily higher organisms, perhaps even in humans.

Prior studies conducted in both humans and mice and published about a decade ago, showed that bone marrow recipients who had received a transplant from the opposite gender had neurons with the genetic signature of the opposite sex. The implication was that cells from the bone marrow generated those neurons. However, it is currently thought that neuronal stem cells in mammals, including humans, are self-renewing and therefore do not need to be replenished. Thus, these findings have not been interpreted as contributing to a natural physiological mechanism.

Every experiment we did confirmed the close relationship between the immune system and adult neurogenesis, Beltz said. Often when one is doing research, experiments can be fussy or give variable results. But for this work, once we started asking the right questions, the experiments worked first time and every time. The consistency and strength of the data are remarkable.

Our findings in crayfish indicate that the immune system is intimately tied to mechanisms of adult neurogenesis, suggesting a much closer relationship between the immune system and nervous system than has been previously appreciated, said Sderhll. If further studies demonstrate a similar relationship between the immune system and brain in mammals, these findings would stimulate a new area of research into immune therapies to target neurological diseases.

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Kansas Governor: Stem cells could guarantee 20 more years of Bill Snyder

Posted: August 18, 2014 at 11:54 pm

Politicians are known to make promisesthey cannot keep,this is no surprise.Kansas State head coach Bill Snyder has also become a bit of an ageless wonder in college football, but even he would tell you his time will come to step away from the sport of college football. Just do not tell that to Kansas governor Sam Brownback, who jokingly promised another 20 years of Snyder leading Kansas State on the sidelines.

Were going to be able to keep Bill Snyder for another 20 years, Gov. Brownbacktold an audience of oil producers at the Kansas Independent Oil and Gas Association convention, per The Wichita Eagle. We got it figured out.

Brownbacks comment came during a discussion on stem cell research. The comment drew a good amount of laughter, reportedly. this time, at least, there were no suggestions Snyder was into anyacts of sorcery, as Washington State head coach Mike Leach once suggested in a Reddit Q&A (or AMA, for the Reddit-literate). Maybe Snyder would not need any help from stem cells if he were indeed a sorcerer.

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Adipose-derived stem cells and nerve regeneration

Posted: August 16, 2014 at 11:45 am

PUBLIC RELEASE DATE:

14-Aug-2014

Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research

Stem cell researchers at the Blond McIndoe Laboratory, University of Manchester, UK, led by Dr Adam Reid, present a review of the current literature on the suitability of adipose-derived stem cells in peripheral nerve repair.

Injuries to peripheral nerves are common and cause life-changing problems for patients alongside high social and health care costs for society. Current clinical treatment relies on sacrificing a nerve from elsewhere in the body to provide a nerve graft at the injury site, but much work has been done to develop a bioengineered nerve graft that would not require this sacrifice. Stem cells are prime candidates as accelerators of regeneration in these nerve grafts.

This prospect, reported in Neural Regeneration Research (Vol. 9, No.14, 2014), presents the current literature on the potential of adipose-derived stem cells as tools to improve nerve regeneration through bioengineered nerve grafts. "Adipose-derived stem cells have the potential to stimulate improved nerve regeneration", stated the authors. "Their incorporation into bioengineered nerve graft treatments could revolutionize the current clinical approach to peripheral nerve repair".

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Article: "Adipose derived stem cells and peripheral nerve regeneration" by Alessandro Faroni, Richard JP Smith, Adam J Reid (1 Blond McIndoe Laboratories, Institute of Inflammation and Repair, University of Manchester, Manchester, UK; 2 Department of Plastic Surgery & Burns, University Hospital of South Manchester, Manchester, UK)

Faroni A, Smith RJP, Reid AJ. Adipose derived stem cells and peripheral nerve regeneration. Neural Regen Res. 2014;9(14):1341-1346.

Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research http://www.nrronline.org/

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Stem cells in the skeletal muscle promote the regeneration of severe nerve peripheral injury

Posted: August 15, 2014 at 7:47 am

PUBLIC RELEASE DATE:

13-Aug-2014

Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research

A research group at the muscle physiology and cell biology unit, the Tokai University School of Medicine, Japan, led by Dr. Tetsuro Tamaki, have developed the stem cell isolation method from the skeletal muscle, termed skeletal muscle-derived multipotent stem cells (Sk-MSCs), which can differentiate into Schwann and perineurial/endoneurial cells, and vascular relating pericytes, endothelial and smooth muscle cells in the damaged peripheral nerve niche. Application of the Sk-MSCs in the bridging conduit of the long nerve gap injury resulted favorable axonal regeneration showing superior effects than healthy nerve autograft, which have been considered gold standard therapy. This also means that the sacrifice of healthy nerves, and the loss of related functions does not need.

Accidental loss of main peripheral nerve route, resulted severe loss of related motor and sensory functions, and if this is the case in arms or legs, largely affects the quality of life. Therefore, application of this method to the human therapy is likely to have a great significance.

The study, reported on Neural Regeneration Research (Vo. 9, No.14, 2014), also introduced that the Sk-MDSCs naturally express multiple neurotrophic and nerve/vascular growth factors. This ability facilitates growth of responsible nerve and vascular cells both in donor and recipient. This ability also suggested that the Sk-MSCs may be the useful tool as an adjuvant for tissue repair after the large resection surgery. In future research, the potential of human Sk-MSCs needs to be clarified. Dr. Tamaki stressed that mild cell isolation, and appropriate shorter term expansion culture may be a key factor to obtain better results, in particular, the human Sk-MSCs, and his group is currently investigating these issues.

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Article: "Bridging long gap peripheral nerve injury using skeletal muscle-derived multipotent stem cells" by Tetsuro Tamaki (Muscle Physiology & Cell Biology Unit, Department of Regenerative Medicine, Division of Basic Clinical Science, Tokai University School of Medicine, Isehara, Japan)

Tamaki T. Bridging long gap peripheral nerve injury using skeletal muscle-derived multipotent stem cells. Neural Regen Res. 2014;9(14):1333-1336.

Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research http://www.nrronline.org/

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Tissue development 'roadmap' created to guide stem cell medicine

Posted: August 15, 2014 at 7:47 am

In a boon to stem cell research and regenerative medicine, scientists at Boston Children's Hospital, the Wyss Institute for Biologically Inspired Engineering at Harvard University and Boston University have created a computer algorithm called CellNet as a "roadmap" for cell and tissue engineering, to ensure that cells engineered in the lab have the same favorable properties as cells in our own bodies. CellNet and its application to stem cell engineering are described in two back-to-back papers in the August 14 issue of the journal Cell.

Scientists around the world are engaged in culturing pluripotent stem cells (capable of forming all the body's tissues) and transforming them into specialized cell types for use in research and regenerative medicine. Available as an Internet resource for any scientist to use, CellNet provides a much needed "quality assurance" measure for this work.

The two papers also clarify uncertainty around which methods are best for stem cell engineering, and should advance the use of cells derived from patient tissues to model disease, test potential drugs and use as treatments. For example, using CellNet, one of the studies unexpectedly found that skin cells can be converted into intestinal cells that were able to reverse colitis in a mouse model.

"To date, there has been no systematic means of assessing the fidelity of cellular engineering -- to determine how closely cells made in a petri dish approximate natural tissues in the body," says George Q. Daley, MD, PhD, Director of the Stem Cell Transplantation Program at Boston Children's and senior investigator on both studies. "CellNet was developed to assess the quality of engineered cells and to identify ways to improve their performance."

Gene Signatures

CellNet applies network biology to discover the complex network of genes that are turned on or off in an engineered cell, known as the cell's Gene Regulatory Network or GRN. It then compares that network to the cell's real-life counterpart in the body, as determined from public genome databases. Through this comparison, researchers can rigorously and reliably assess:

"CellNet will also be a powerful tool to advance synthetic biology -- to engineer cells for specific medical applications," says James Collins, PhD, Core Faculty member at the Wyss Institute and the William F. Warren Distinguished Professor at Boston University, co-senior investigator on one of the studies.

Putting CellNet to the Test

The researchers -- including co-first authors Patrick Cahan, PhD and Samantha Morris, PhD, of Boston Children's, and Hu Li, PhD, of the Mayo Clinic, first used CellNet to assess the quality of eight kinds of cells created in 56 published studies.

In a second study, they applied CellNet's teachings to a recurring question in stem cell biology: Is it feasible to directly convert one specialized cell type to another, bypassing the laborious process of first creating an iPS cell? This study looked at two kinds of directly converted cells: liver cells made from skin cells, and macrophages made from B cells.

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Stem cell discovery: Australian scientists make significant find while studying zebrafish

Posted: August 14, 2014 at 3:44 pm

Australian scientists studying zebrafish have stumbled upon what they say is one of the most significant discoveries in stem cell research.

In research published on Thursday in the journal Nature, the Monash University scientists revealed that they uncovered how one of the most important stem cells in blood and bone marrow, the haematopoietic stem cell (HSC), is formed.

Professor Peter Currie, from Monash University's Australian Regenerative Medicine Institute, said the discovery brought researchers closer to growing HSCs in a lab.

"HSCs are the basis of bone marrow transplantations as a therapy, so when a leukaemia patient receives bone marrow, it's really these HSCs that do the heavy lifting," Professor Currie said.

"So when clinicians do bone marrow transplants, they need to find a matching donor recipients and we know that's a hit-or-miss procedure.

"So for many years people have been trying to make HSCs in the dish, and they've had very little success in doing this."

Professor Currie, who led the study, said the discovery brought scientists much closer to achieving that aim.

"It's the discovery of a completely new cell type that basically is required to give instructions to the HSC to make it become what it needs to become," he said.

"It means we now understand how HSC form in the body better, we can use that information to try to grow these cells in the dish and we hope that will lead to better treatment for people with leukaemia and blood disorders."

Professor Currie said he specialises in muscle stem cell biology and accidentally came across the discovery while studying muscle stem cells in zebrafish.

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