VistaGen Therapeutics' Largest Stockholder Signs 6-Month Lock-Up Agreement Yahoo Finance VistaStem Therapeutics is VistaGen's wholly owned subsidiary focused on applying human pluripotent stem cell technology, internally and with collaborators, to discover, rescue, develop and commercialize proprietary new chemical entities (NCEs ... and more »

Read the rest here:
VistaGen Therapeutics' Largest Stockholder Signs 6-Month Lock-Up Agreement - Yahoo Finance

Recommendation and review posted by Bethany Smith

Sacramento Bee

California stem cell agency president steps down as worries mount about its future Sacramento Bee Mills will leave at the end of June to become president of the National Marrow Donor Program in Minneapolis, the world's largest bone marrow donor program. Maria Millan, vice president of therapeutics at the agency, will become its interim president in ... CA Stem Cell Agency Chief Randy Mills to Leave After Three Years ...Xconomy all 4 news articles »

Go here to read the rest:
California stem cell agency president steps down as worries mount about its future - Sacramento Bee

Recommendation and review posted by simmons

TEL AVIV, Israel, May 3, 2017 /PRNewswire/ --

- Initiation of Phase 3 registrational study expected in second half of this year

- Study to focus on stem cell mobilization for autologous transplantation in multiple myeloma patients

BioLineRx Ltd. (NASDAQ/TASE: BLRX, BLRX.TA), a clinical-stage biopharmaceutical company focused on oncology and immunology, today announced that it has met with the U.S. Food and Drug Administration (FDA) and has gained clarity on the development program and the design of a Phase 3 pivotal study for BL-8040, its robust platform for multiple oncology indications, as a novel stem cell mobilization treatment for autologous bone-marrow transplantation. Following its successful meeting with the FDA, the Company anticipates the initiation of a registrational Phase 3 trial during the second half of 2017. The study will investigate BL-8040 in combination with granulocyte colony-stimulating factor (G-CSF) for mobilization of stem cells from the bone marrow to the peripheral blood, followed by collection and subsequent autologous transplantation in patients with multiple myeloma.

"BL-8040 given as a single injection in a Phase 1/2 study in multiple myeloma patients was previously shown to be highly effective in mobilizing stem cells in combination with G-CSF," said Philip Serlin, Chief Executive Officer of BioLineRx. "Following our recent successful meeting with the FDA, we believe we have a clear development path forward towards registration of BL-8040 as a novel stem cell mobilization treatment for autologous transplantation. We look forward to the initiation of the Phase 3 pivotal study later this year, which, if successful, could pave the way for future commercialization of BL-8040."

"We see clear potential for BL-8040 to benefit multiple myeloma patients undergoing autologous bone marrow transplantation. In parallel, we are continuing to expand the potential of our unique BL-8040 oncology platform, with multiple clinical studies for additional indications that are up and running or expected to commence during 2017. These include several combination studies with immune checkpoint inhibitors, a Phase 2b study in consolidation AML and a Phase 2 study in allogeneic stem-cell mobilization as a monotherapy with topline results expected by the end of 2017," added Mr. Serlin.

About BL-8040

BL-8040 is a short peptide for the treatment of acute myeloid leukemia, solid tumors, and stem cell mobilization. It functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity. In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of cancer cells from the bone marrow, thereby sensitizing these cells to chemo- and bio-based anti-cancer therapy, as well as a direct anti-cancer effect by inducing apoptosis. In addition, BL-8040 has also demonstrated robust stem-cell mobilization, including the mobilization of colony-forming cells, and T, B and NK cells. BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

About Stem Cell Mobilization

High-dose chemotherapy followed by stem cell transplantation has become an established treatment modality for a variety of hematologic malignancies, including multiple myeloma, as well as various forms of lymphoma and leukemia. Stem cells are mobilized from the bone marrow using granulocyte colony-stimulating factor (G-CSF), harvested from the peripheral blood by apheresis, and infused to the patient after chemotherapy. This type of treatment often replaces the use of traditional bone marrow transplantation, because the stem cells are easier to collect and the treatment allows for a quicker recovery time and fewer complications.

About BioLineRx

BioLineRx is a clinical-stage biopharmaceutical company focused on oncology and immunology. The Company in-licenses novel compounds, develops them through pre-clinical and/or clinical stages, and then partners with pharmaceutical companies for advanced clinical development and/or commercialization.

BioLineRx's leading therapeutic candidates are: BL-8040, a cancer therapy platform, which has successfully completed a Phase 2a study for relapsed/refractory acute myeloid leukemia (AML) and is in the midst of a Phase 2b study as an AML consolidation treatment and a Phase 2 study in stem cell mobilization for allogeneic transplantation; and AGI-134, an immunotherapy treatment in development for multiple solid tumors, which is expected to initiate a first-in-man study in the first half of 2018. In addition, BioLineRx has a strategic collaboration with Novartis Pharma AG for the co-development of selected Israeli-sourced novel drug candidates; a collaboration agreement with MSD (known as Merck in the US and Canada), on the basis of which the Company has initiated a Phase 2a study in pancreatic cancer using the combination of BL-8040 and Merck's KEYTRUDA; and a collaboration agreement with Genentech Inc., a member of the Roche Group, to investigate the combination of BL-8040 and Genentech's Atezolizumab in several Phase 1b studies for multiple solid tumor indications and AML.

For additional information on BioLineRx, please visit the Company's website at http://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events. BioLineRx industry updates are also regularly updated on Facebook, Twitter, and LinkedIn.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Some of these risks are: changes in relationships with collaborators; the impact of competitive products and technological changes; risks relating to the development of new products; and the ability to implement technological improvements. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 23, 2017. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contacts: PCG Advisory Vivian Cervantes Investor Relations +1-212-554-5482 [emailprotected]

or

Tsipi Haitovsky Public Relations +972-52-989892 [emailprotected]

SOURCE BioLineRx Ltd.

View original post here:
BioLineRx to Initiate Phase ' Study with BL-84 as Novel Stem Cell Mobilization Treatment Following Successful ... - GuruFocus.com

Recommendation and review posted by Bethany Smith

A 26-YEAR-OLD localwoman suffering acute leukemia received a novel stem cell transplant at Shanghai General Hospital today.

Yang Yingjie received the transplant of her fathers steam cell, which is only a half match of hers, and another unit of matched umbilical cord blood to reduce rejection.

The patient is also the 2500thpatient who received umbilical cord blood transfusion from Shanghai Cord Blood Bank, the only licensed umbilical cord blood bank in the city.

Yang was found to have acute leukemia in December last year. She received treatment at Shanghai General Hospital to stabilizeher condition for transplant.

However, she couldnt find a match at stem cell donors bank and had to use her fathersa half match.

We used a unified stem cell transplant therapy in this case. It is also a new breakthrough of leukemia treatmentin recent years,said Dr Wang Chun from Shanghai General Hospital. Each unit of umbilical cord blood is only 50 milliliters, which is not enough for an adult patient. However a unified transplantwith half matched stem cells from adult donor and a unit of fully matched umbilical cord blood can reduce rejection and streamline the transplant success of half-matched stem cells.

Stem cells from umbilical cord blood are less mature than those in adult bone marrow, so they are less prone to rejection by the recipient and more active in developing into different types of cells, experts explained.

Wang said his team has used this therapy to treat somecomplicated leukemia patients and received good results.

There are about 5 million leukemia patients in China and half of them are children. Stem cells have been used in the treatment of many diseases including leukemia, lymphoma and anemia. The most common disease category is leukemia.

Visit link:
Patient receives novel stem cell transplant - Shanghai Daily (subscription)

Recommendation and review posted by Bethany Smith

STANFORD Stanford scientists have grown and assembled parts of a humanbrainin a dish.

Heres what is even more remarkable: Their mini-brain forms mental circuitry and cells converse with each other.

There is cross-talk, said lead researcher Dr. Sergiu Pasca, assistant professor of psychiatry and behavioral sciences at Stanford University School of Medicine, whose study was published in the journal Nature.

The team did not build an entire brain, the stuff of sci-fi fantasy. It doesnt think; its not self-aware. Thats a far more complex and likely unattainable goal.

Instead, they made a tiny but powerful model of the cerebral cortex for the study of such devastating human disorders as schizophrenia, epilepsy and autism impairments not easily studied in people.

This mini-brain reveals how networks of our mind can grow, behave and communicate, giving scientists an unprecedented view of our most mysterious organ.

What goes wrong with the mental circuitry of people with disease or disorders? Thats what they hope to learn.

Their brain also can be used to test potential drugs, essential for improving the pharmaceuticals need by psychiatry.

Its the first example of assembling, in a 3D culture, this brain region, Pasca said. Essentially, we get a small cerebral cortex in a dish.

The neurobiology of the brain remains one of the great challenges of modern medicine. Thats because we havent had direct views of the brains cellular behavior. While we can watch mental function through tools like Magnetic Resonance Imaging, that doesnt show us whats happening at the most basic level.

And we havent been able to watch brain development in the lab, because it happens during the second and third trimester of pregnancy.

Other diseases, like cancer, dont have this problem. Thats because doctors can sample tumor cells and look at them under a microscope. The sampling and study of brain cells is much harder.

Recapitulation of a pivotal stage in the cortexs formation demonstrates the techniques promise for discovery and even for testing potential interventions, according to a statement by Dr. Joshua Gordon, director of the National Institute of Mental Health, which made a videoto explain theresearch. It moves us closer to realizing the goal of precision medicine for brain disorders.

Members of the Stanford team started with longstanding tried-and-true techniques. They took skin cells and turned them into stem cells. Then they used chemical prods to turn them into two different types of brain cells.

In one dish, they grew cells called glutamatergic neurons, because they secrete the chemical glutamate, responsible for sending excitatory messages in the brain. Too much cellular excitement is thought to underlie conditions ilke seizures in epilepsy.

In a second dish, they grew cells that secrete a different chemical, called GABA, which sends inhibitory messages in the brain. Their job is to apply the brakes.

These arent just flat garden-variety layers of cells. Rather, theyre brainballs. Each ball measures about 1/16 of an inch in diameter and consists of over 1 million cells each, living for up to two years. They dont adhere to the dish; rather, they float, like little bobbing pearls.

Then they were introduced to each other.

And heres the magic: Within three days, the two cell types fused into one big sphere and then started organizing.

The cells that make GABA cells migrated over to the cells that make glutamate and began forming the circuitry that is responsible for the brains most advanced cognitive activities, the team found.

They start moving, and keep moving, for months, making small hops in one direction, said Pasca. They move to the other side and make connections.

They grew long appendages called axons. They also grew little knobby spines that stick out like branches to receive chemical messages from other cells axons. It is this signaling that enables us to think and learn.

Using small electrodes, the team listened in on the fused cells, and heard communication. The GABA-making and glutamatergic cells were successfully forming circuits and signaling to each other.

To be sure, their brainball is an incomplete model. It lacks complexity and is missing other cells that are part of the cerebral cortex. There arent blood vessels. It will never grow large.

But it is a powerful platform for asking how the human brain develops, said Pasca. Can we find abnormalities that are associated with disease? If we do, can we test drugs? That is its potential.

Already, its taught them about a rare developmental disease called Timothy syndrome, which includes symptoms of autism and epilepsy. Growing brainballs from skin cells donated by three patients, they found that these cells dont migrate normally their hopping movements are too quick, and too small. Over time, they got left behind.

The same gene that causes Timothy syndrome is linked to schizophrenia, other types of autism and bipolar disease. Pasca suspects these conditions may also have flaws in the fusing and communication of cells.

In the future, the Stanford team hopes to study the cells of individual patients to see if they can detect problems with their ability to move, migrate and communicate.

Stanfords Office of Technology Licensing has filed for a patent on the intellectual property involving the generation of these spheres and their assembly for studying development and disease.

The exquisite timing and placement of these different neuron cell types is critical for establishing a balance between excitation and inhibition within brain circuits. This balance is thought to be disrupted in brain disorders, Dr. David Panchision, chief of the NIMH Developmental Neurobiology Program (which funded the research), said in a statement. Re-playing these developmental processes with a patients own cells can allow us to determine what distinguishes these different disorders at a molecular and cellular level.

Read this article:
Human brain in a dish: Stanford-grown brain cells fuse and chat - The Mercury News

Recommendation and review posted by simmons

With the advent of CRISPR , a new way to edit DNA, the field of genomic technology has never been more exciting. The implications have yet to be seen, but scientists could theoretically snip out a person's genetic risk for disease. But it's also never been a more anxiety-inducing time. Some experts argue innovations in genomics are moving forward at a pace faster than our ability to parse their potential consequences.

In a panel discussion at Fortune s Brainstorm Health conference in San Diego, scientists discussed the promises and perils of this breakthrough technologysome of which they're already starting to see.

I think CRISPR is a very exciting discovery, said J. Craig Venter, co-founder of the health company Human Longevity, Inc. and one of the first scientists to sequence the human genome. Venter is using genome sequencing as a way to help predict a persons risk for disease and offer more personalized treatment with a physical exam called the Health Nucleus : an eight-hour, $25,000 inside-and-out doctors appointment that includes whole-genome sequencing, high-tech scanning and early diagnostics.

So far the company has sequenced more than 40,000 human genomes. Of the people that complete the Health Nucleus, one in 40 will discover they have a serious cancer they didn't know about, he said.

Yet some experts are skeptical that exhaustive testing always translates to better health. Dr. Eric Topol, founder and director of Scripps Translational Science Institute, called for a more reserved way forward in his remarks at the conference, arguing that too much scanning can lead to more false positive results and potentially unnecessary interventions. We have to prove that doing tests are truly associated with positive outcomes, Topol said. We have to be much more discriminative about the tests that we do.

Some companies are taking a more tempered approach: inexpensive testing that looks for specific genes known to substantially increase a persons risk for disease. Color Genomics, a genetics company that has brought down the cost of genetic testing, focuses on cancer and offers affordable tests for the BRCA1 and BRCA2 genes, which can significantly raise a womans risk for breast and ovarian cancer. It changes the equation in terms of treating disease, said Othman Laraki, co-founder and CEO of Color Genomics.

As for finding and fixing genetic problems well before they even arise? The scientists on the panel agreed that they're not there yet, and that current iterations of CRISPR may not be quite as precise as the hype has claimed. For now, that may be for the best. Editing human embryos with CRISPR should be a long way off, said Venter. Not something we do next week.

More here:

How Scientists Think CRISPR Will Change Medicine - TIME

Recommendation and review posted by Bethany Smith

Taube Philanthropies, founded by US-based Jewish businessman Tad Taube, said it would donate $3 million to support a collaboration between Stanford University School of Medicine and the Gladstone Institutes in California focused on research related to Huntingtons disease.

The donation will support a new program with three medical research groups and, for the first time, introduce gene editing and stem cell therapies for treatments and, eventually, a potential cure for Huntingtons disease, the philanthropic group said in a statement.

Researchers from Stanford School of Medicine and the Gladstone Institutes Taube-Koret Center for Neurodegenerative Disease Research, will collaborate with clinical trials at the Memory and Aging Center of the University of California, San Francisco (UCSF).

Huntingtons disease is a cruel genetic disorder that robs its victims of physical and mental control, said Taube, the chairman of Taube Philanthropies. The time and research that Stanford, UCSF and Gladstone Institutes have committed to this cause is remarkable.

Huntingtons disease is a genetic neurodegenerative disease that causes the progressive breakdown of nerve cells in the brain. It causes physical and mental abilities to deteriorate, eventually leading to premature death. There is no cure or approved drugs to slow the progression of the disorder. Huntingtons disease belongs to a family of neurodegenerative diseases that includes Alzheimers, Parkinsons and ALS. Today, there are approximately 30,000 symptomatic Americans and more than 200,000 at risk of inheriting the disease.

We have made considerable progress in advancing drug therapy developments for patients with neurodegenerative diseases since Taube Philanthropies started supporting my research, nearly a decade ago, said Dr. Steve Finkbeiner, director of the Gladstone Institutes Taube-Koret Center. With this renewed commitment and concerted approach focusing on Huntingtons disease, I am optimistic that we will continue advancing toward our research goals.

The $3 million donation is a continuation of Taube Philanthropies aim to finding treatments and cures for neurodegenerative diseases. Over the last 12 years, the organization has donated over $5 million to Gladstone Institutes and $1 million to the Stanford University School of Medicine for related research. Taube Philanthropies started funding Huntingtons disease research under the direction of Nobel laureate Dr. Stanley Prusiner at UCSFs department of neurology.

We have been working to develop similar therapies for neurodegenerative diseases for many years with some promising results, said Dr. Matthew Porteus, associate professor of pediatrics at Stanford. Employing the latest technology, including CRISPR/Cas9 a powerful new tool that enables scientists to make precise genome edits I believe we have the opportunity to make great strides toward developing a therapy specifically for Huntingtons disease.

The funds will be distributed to the researchers in annual payments through 2021, to account for the long-term process of translating scientific discoveries into clinical trials and applications. The research team will take on a multi-year program to develop genetic therapy for the disease. Stanford is already doing stem cell research and hopes to begin conducting clinical trials with Huntingtons patients soon, the statement said.

Having studied Alzheimers for many years, I am all too familiar with the devastating effects of neurodegenerative diseases, said Dr. Frank Longo, professor of neurology at Stanford. As researchers with different area specialties convene for this unique joint project, I am optimistic about our capacity to come even closer to finding treatments and cures for these degenerative conditions.

Research into Huntingtons disease has until recently suffered from a lack of significant funding. Due to the progress made in the field by researchers, new US federal funding has become available and major pharmaceutical companies have started funding research to develop possible medications. However, collaborative efforts still rely on contributions from individuals and private foundations, the statement said.

View post:
Taube to fund $3m Huntington's disease research in US - The Times of Israel

Recommendation and review posted by simmons

Have researchers taken a step closer to developing an eventual cure for HIV? A Temple University-led team hopes so, by using a gene editing technique to successfully remove HIV infection from lab mice. The gene-editing tool calledCRISPR which allows scientists to basically cut out and insert specific portions of DNA was used to excise HIV DNA from the mice.

This was the first time CRISPR has been used to shut down HIV replication and eliminate the virus from animal cells. Think of CRISPR as working somewhat like microscopic scissors that snip out an unwanted piece of DNA and then replace that with a new piece. The research, published in the journal Molecular Therapy, involved three animal models, including a "humanized" model where human immune cells infected with the virus were transplanted in lab mice.

"Over our years of research, all of this was frankly a big surprise. This research, so far, has yielded all pleasant surprises, frankly. I never thought that this CRISPR system was going to be working out so beautifully with such efficiency and precision when it first came onto the scene," Kamel Khalili, director of Temple's center for neurovirology, told CBS News.

Khalili led the study along with Wenhui Hu, associate professor in Temple University School of Medicine's Center for Metabolic Disease Research and the Department of Pathology, and Won-Bin Young, who was at that time an assistant professor in the Department of Radiology at the University of Pittsburgh School of Medicine.

This work builds off the team's previously published research last year in which they introduced the HIV-1 DNA into the tissue of rat and mice subjects, and then removed these fragments using CRISPR. This new study is the first time this has been done in three animal models.

Play Video

CRISPR could help rid of diseases like cystic fibrosis, muscular dystrophy and even HIV and cancer. But many scientists, including Jennifer Doudn...

While the work signals progress, the medical community still sees years of work ahead before there's a reliable cure for HIV. According to the World Health Organization, 36.7 million people were reported to be living with HIV globally by the end of 2015. Since the start of the HIV/AIDS epidemic, more than 70 million people have been infected with the virus that has resulted in 35 million deaths.

The stakes are high, and the Temple team is one of many trying to find a cure for the virus, which has proven exceptionally difficult to eliminate from the body. While current drug treatments can reduce the virus to virtually undetectable levels enabling many patients to live longer, healthier lives HIV continues to lurk in hidden reservoirs and comes roaring back if treatment stops. In late 2015, theamfAR Institute for HIV Cure Research set the ambitious goal of developing a basis for cure for HIV by the end of 2020.

"The basic science community in HIV research is now very focused on finding a cure," Paul Voldberding, head of the institute, wrote in an email to CBS News. "It still feels a long way off but the tools we now have definitely including the gene editing used in this report is accelerating our work and raising optimism. The cure field is in very close contact and collaborations are active world wide. It's really quite exciting!"

Voldberding is also the director of the UCSF AIDS Research Institute and has a place in history for founding the first inpatient ward for people with AIDS at San Francisco General Hospital in 1983. How promising does he view this new research out of Temple?

"Gene editing is a potent and still rather new tool in HIV research and many other areas as well," he wrote. "It faces a challenge in scalability getting the technology simplified and inexpensive but is certainly worth following."

Play Video

Musician and activist Sir Elton John thanks the U.S. Congress for its prior efforts on HIV/AIDS relief and urges further action to end the epidem...

Since first being developed a mere five years ago, CRISPR has generated excitement and controversy in equal measures. While it was named "Breakthrough of the Year" in 2015 by Science magazine, ethical debate has swirled around CRISPR over how it could be used for good or ill to make changes to our DNA down the line.

Ellen Jorgensen, a molecular biologist and science communicator whose latest project is the yet-to-launch Biotech Without Borders, said she thinks it's important to focus on the potential of CRISPR, rather than feed into the "hysteria" that can surround such life-altering scientific technologies.

"I think CRISPR is an example of why the general public should embrace the chance to learn more about this sort of technology that will be more and more relevant to everyone's daily life as time goes on," Jorgensen told CBS News. "We are in an age of biotechnology as opposed to the last century, which was the 'age of physics.' There is an equal potential here to disrupt technologies, but it also creates ethical questions that the general public has to weigh in on. My thing is, I want them to weigh in on them, but have the understanding that this technology is something that is powerful and that can spur a lot of change moving forward."

Play Video

University of Pennsylvania researchers have made a potential breakthrough in the fight against HIV. As Bigad Shaban explains, one patient seeking...

In the case of this latest HIV research advance, Jorgensen, who cofounded Genspace, a nonprofit devoted to fostering better science literacy, said she believes there is "great potential" in finding a cure for something like HIV through gene editing technology.

Moving forward, Khalili and his team plan to try their technique on primate subjects, whose DNA is obviously closer to humans. He said they are working on securing more funding to move on to primate clinical trials.

Voldberding added that "primates are a very good model for human trials," and that research like this is promising in the continued fight against HIV.

"I think that CRISPR and tools like it are revolutionizing the medical field and will bring about new ways for the treatment and cure for a broad range of diseases," Khalili said. "When it comes to treating HIV or cancer or other genetic diseases, I think there are a lot of good things that will come out of this."

See more here:
Researchers use gene editing to eliminate HIV infection in mice - CBS News

Recommendation and review posted by sam

Research at the University of Illinois at Urbana-Champaign has provided the first evidence that viruses and hosts share highly similar regulatory sequences in their promoters -- the initiation sequences of human genes that code for functional proteins.

"To date viral-host networks include protein and mRNA interactions between viruses and their hosts at later stages of gene expression, but our discovery of genetically coupled promoters is novel. They present an additional layer of regulatory synchrony between virus and host, established and poised before either expresses their protein products," explained Roy Dar, an assistant professor of bioengineering at Illinois.

Latent or dormant HIV infected cell reservoirs have been identified as the major barrier towards a cure due to their ability to spontaneously reactivate after removal of antiretroviral therapy. Leading strategies for eradication of HIV attempt to reactivate the whole latent reservoir and clear it with current drug cocktails, a process referred to as 'shock and kill' therapy.

"Promoters of genes coded within our DNA and the HIV-1 viral promoter which initiates active replication of the virus are strongly coupled in their regulation leading to co-expression -- potentially for a viral fitness advantage. In this study, we investigated a specific T-cell migratory pathway that HIV has coupled to, gaining therapeutic insights currently unknown to the HIV cure research community," Dar added.

Promoter similarity of human immunodeficiency virus (HIV) and a human surface receptor allows shared activators to co-regulate viral-host gene expression (blue and red in the cell nucleus). Viral proteins bind cell surface receptors enabling viral control of host cell migration (right side). Those same viral proteins form viral offspring which are shed from the host cell and increase infectious risk to the moving cell's environment.

Promoter similarity of human immunodeficiency virus (HIV) and a human surface receptor allows shared activators to co-regulate viral-host gene expression (blue and red in the cell nucleus). Viral proteins bind cell surface receptors enabling viral control of host cell migration (right side). Those same viral proteins form viral offspring which are shed from the host cell and increase infectious risk to the moving cell's environment.

Within the systems and synthetic biology fields, the group's findings reveal an additional layer of regulation with which viruses co-evolve with coding-genes and interlace pathways in their hosts.

"The study also presents a mechanism for synchronizing initiation of gene expression in synthetic gene circuitry," stated Kathrin Bohn-Wippert, a postdoctoral researcher and first author of the paper, "Genetic coupling of viral-host gene expression presents migratory challenges in HIV therapies" (10.1038/NCOMMS15006), appearing in Nature Communications. "Specifically, in this framework of viral-host genetic coupling we found that the HIV and human CXCR4 promoters are co-regulated and co-expressed. CXCR4 is a chemokine receptor involved in one of the major migratory pathways throughout our body."

"We have demonstrated, for the first time, that the virus co-expresses with the receptor in order to control infected cell migration and its importance in HIV 'shock and kill' eradication strategies (therapies towards a cure). We also demonstrated how drug treatments can differentially control infected cell migration and/or reactivation of the virus from its latent and inactive state," she said.

According to the researchers, additional network mapping of the coevolution of virus and host-cell gene regulatory coupling will guide future therapeutic strategies, expand systems biology efforts on viral-host networks, and provide novel design principles to reverse bioengineer viral circuitry for synthetic biology and gene therapies.

"For the HIV Cure Community we hope this study will raise awareness to the added challenges facing leading strategies towards a cure," remarked Dar, who is also affiliated with the Carl R. Woese Institute for Genomic Biology and the Center for Biophysics and Quantitative Biology at Illinois. "We hope this study will provide new insights to exploit viral-host relationships and viral control of cell migration for advanced therapeutic strategies."

Co-authors include Melina Megaridis and Erin Tevonian, both bioengineering undergraduate researchers, in the Illinois Cancer Scholars Program, and in the Dar "Noise Biology" Lab.

Link:
Insights to redirect leading HIV cure strategy - Science Daily

Recommendation and review posted by Bethany Smith

Using the gene-editing system known as CRISPR, MIT researchers have shown in mice that they can generate colon tumors that very closely resemble human tumors. This advance should help scientists learn more about how the disease progresses and allow them to test new therapies.

Once formed, many of these experimental tumors spread to the liver, just like human colon cancers often do. These metastases are the most common cause of death from colon cancer.

"That's been a missing piece in the study of colon cancer. There is really no reliable method for recapitulating the metastatic progression from a primary tumor in the colon to the liver," says Omer Yilmaz, an MIT assistant professor of biology, a member of MIT's Koch Institute for Integrative Cancer Research, and the lead senior author of the study, which appears in the May 1 issue of Nature Biotechnology.

The study builds on recent work by Tyler Jacks, the director of the Koch Institute, who has also used CRISPR to generate lung and liver tumors in mice.

"CRISPR-based technologies have begun to revolutionize many aspects of cancer research, including building mouse models of the disease with greater speed and greater precision. This study is a good example of both," says Jacks, who is also an author of the Nature Biotechnology paper.

The paper's lead authors are Jatin Roper, a research affiliate at the Koch Institute and a gastroenterologist at Tufts Medical Center, and Tuomas Tammela, a research scientist at the Koch Institute.

Mimicking human tumors

For many years, cancer biologists have taken two distinct approaches to modeling cancer. One is to grow immortalized human cancer cells known as cancer cell lines in a lab dish. "We've learned a lot by studying these two-dimensional cell lines, but they have limitations," Yilmaz says. "They don't really reproduce the complex in vivo environment of a tumor."

Another widely used technique is genetically engineering mice with mutations that predispose them to develop cancer. However, it can take years to breed such mice, especially if they have more than one cancer-linked mutation.

Recently, researchers have begun using CRISPR to generate cancer models. CRISPR, originally discovered by biologists studying the bacterial immune system, consists of a DNA-cutting enzyme called Cas9 and short RNA guide strands that target specific sequences of the genome, telling Cas9 where to make its cuts. Using this process, scientists can make targeted mutations in the genomes of living animals, either deleting genes or inserting new ones.

To induce cancer mutations, the investigators package the genes for Cas9 and the RNA guide strand into viruses called lentiviruses, which are then injected into the target organs of adult mice.

Yilmaz, who studies colon cancer and how it is influenced by genes, diet, and aging, decided to adapt this approach to generate colon tumors in mice. He and members of his lab were already working on a technique for growing miniature tissues known as organoids -- three-dimensional growths that, in this case, accurately replicate the structure of the colon.

In the new paper, the researchers used CRISPR to introduce cancer-causing mutations into the organoids and then delivered them via colonoscopy to the colon, where they attached to the lining and formed tumors.

"We were able to transplant these 3-D mini-intestinal tumors into the colon of recipient mice and recapitulate many aspects of human disease," Yilmaz says.

More accurate modeling

Once the tumors are established in the mice, the researchers can introduce additional mutations at any time, allowing them to study the influence of each mutation on tumor initiation, progression, and metastasis.

Almost 30 years ago, scientists discovered that colon tumors in humans usually acquire cancerous mutations in a particular order, but they haven't been able to accurately model this in mice until now.

"In human patients, mutations never occur all at once," Tammela says. "Mutations are acquired over time as the tumor progresses and becomes more aggressive, more invasive, and more metastatic. Now we can model this in mice."

To demonstrate that ability, the MIT team delivered organoids with a mutated form of the APC gene, which is the cancer-initiating mutation in 80 percent of colon cancer patients. Once the tumors were established, they introduced a mutated form of KRAS, which is commonly found in colon and many other cancers.

The scientists also delivered components of the CRISPR system directly into the colon wall to quickly model colon cancer by editing the APC gene. They then added CRISPR components to also edit the gene for P53, which is commonly mutated in colon and other cancers.

"These new approaches reduce the time frame to develop genetically engineered mice from two years to just a few months, and involve very basic gene engineering with CRISPR," Roper says. "We used P53 and KRAS to demonstrate the principle that the CRISPR editing approach and the organoid transplantation approach can be used to very quickly model any possible cancer-associated gene."

In this study, the researchers also showed that they could grow tumor cells from patients into organoids that could be transplanted into mice. This could give doctors a way to perform "personalized medicine" in which they test various treatment options against a patient's own tumor cells.

Yilmaz' lab is now using these techniques to study how other factors such as metabolism, diet, and aging affect colon cancer development. The researchers are also using this approach to test potential new colon cancer drugs.

Read the original post:
New model could speed up colon cancer research: Introducing ... - Science Daily

Recommendation and review posted by sam

Jonathan Pitre received some good news Thursday when blood tests revealed the first sign of white blood cell growth. Tina Boileau / -

After three arduous and eventful weeks in a Minnesota hospital, Jonathan Pitre recorded his first white blood cells Tuesday in the first promising sign since his stem cell transplant.

Pitre, 16, has suffered a series of medical challenges infections, fevers, pain and kidney issues since the April 13 transplant that saw him infused with stem-cell rich blood and marrow drawn from his mother.

But on Tuesday, he finally received some good news when his blood test revealed the first sign of white blood cell growth. The test, which measures white blood cells in a unit of blood, registered 0.1.The normal range is 4.0 to 11.0

We still have to wait for skin biopsies to find out whose cells they are, but its definitely a step in the right direction, said his mother, Tina Boileau.

Last year, after his first stem cell transplant, Pitre and his mother were devastated when biopsies revealed that the white blood cells his body had started to produce were his own. It meant that the transplant had not worked, and that Pitres own stem cells had recolonized his bone marrow.

The family is taking a cautious approach this time, but Tuesdays news was much welcomed after 19 difficult days.

Today is a good day, Boileau tweeted.

A rise in Pitres white blood cell count is the first potential sign that his mothers donated stem cells have started to take root and grow in his bone marrow. But only a biopsy will be able to confirm the cells are being made by Boileaus stem cells, and not his own.

Meanwhile, Pitre will head to surgery again Wednesday to have two central intravenous lines placed in his chest. A third line, a peripherally inserted central catheter (known as a PICC line), will also be installed in case Pitre has to go on dialysis at some point to give his overworked kidneys a break.

During the past three weeks, Pitre has had a central line and a PICC line removed because of infection concerns. It has forced him to use three IVs to support all of his medications.

Boileau said it has been a trying few weeks with one medical problem being fixed only in time for another to appear. The three IVs have also complicated Pitres once-every-second-night bath routine, which is necessary to care for his damaged skin.

Jonathan and I have become very creative with all of his new lines, said Boileau, who is the only one Pitre trusts to remove and re-apply his complex welter of bandages. Its like the most complex puzzle.

Since its not a bath night, they plan to watch the Ottawa Senators play the New York Rangers on Tuesday evening.

Follow this link:
Some good news for Jonathan Pitre: the first white blood cells have appeared - Ottawa Citizen

Recommendation and review posted by simmons

Stem Cells 101, the Value Proposition

The Key purpose of stem cells is to maintain, heal and regenerate tissues wherever they reside in our body. This is a continuous process that occurs inside the human body throughout its life.

If we did not have stem cells, our lifespan would be about 1 hour, because there would be nothing to replace exhausted cells or damaged tissue. In addition, any time the body is exposed to any sort of toxin, the inflammatory process causes stem cells to swarm the area to repair the damage.

As an example: Say you went to the gym in the morning and did some squats. As a result of that, you would get tiny tears inside the muscle. The stem cells that reside beneath the muscle would come out and repair those little tears.

The reason that, if you continuously go to the gym, you would start to build new muscle, is because those stem cells, hard at work underneath your muscle, are helping to repair and build that new muscle. This would apply to all of the tissues inside your body.

Sure, it is easy to think of stem cell therapy as a magic bullet,but is wise to implement strategies that nourish and thereby help optimize the stem cells we already have in our body.

As noted by Kristin Comella, named # 1 on the Academy of Regenerative Practices list of Top 10 stem cell innovators, has been a stem cell researcher for nearly 20 years: You have to create an appropriate environment for these cells to function in. If you are putting garbage into your body and you are constantly burdening your body with toxins, your stem cells are getting too distracted trying to fight off those toxins.

By creating an appropriate environment, optimizing your diet and reducing exposure to toxins, that will allow the stem cells that were putting in to really home in and focus on the true issue that were trying to treat.

The other thing weve discovered over the years is that [stem cell therapy] is not the type of thing where you take one dose and youre cured forever. Our tissues are constantly getting damaged Youre going to have to repeat-dose and use those stem cells to your advantage.

When you think about a lizard that loses its tail, it takes two years to grow back the tail. Why would we put unrealistic expectations on the stem cells that were trying to apply to repair or replace damaged tissue? This is a very slow process. This is something that will occur over months and may require repeat dosing.

In the past, stem cells were isolated from bone marrow, and were used for bone marrow transplants for cancer patients since the 1930s. But, stem cells come from just about any tissue in the human body, as every tissue contains stem cells.

Human bone marrow has very low amounts of mesenchymal stem cells now believed to be the most important, from a therapeutic perspective.

Mesenchymal stem cells help trigger an immunomodulatory response or a paracrine effect, which means they send signals out to the rest of your body, calling cells to the area to help promote healing.

What researchers have discovered recently is that a more plentiful source of stem cells is actually your fat tissue. Body fat can contain up to 500X more cells than bone marrow, as far as these mesenchymal type stem cells go.

One thing that is also critically important when youre talking about isolating the cells is the number of other cells that are going to be part of that population.

When youre isolating a bone marrow sample, this actually is very high in white blood cells, which are pro-inflammatory.

White blood cells are part of your immune response. When an injury occurs, or a foreign body enters your system, white blood cells will attack. Unfortunately, white blood cells do not discriminate, and can create quite a bit of damage as they clean the area out, Ms. Comella says.

Stem cells, in particular the mesenchymal cells, quiet down the white blood cells and then start the regeneration phase, which leads to new tissue.

Bone marrow tends to be very high in white blood cells and low in the mesenchymal cells. Isolating stem cells from fat tissue is preferred not only because its easier on the patient, but fat also contains a higher population of mesenchymal cells and fewer white blood cells.

The benefit also of isolating [stem cells from] fat is that its a relatively simple procedure. Theres typically no shortage of fat tissue, especially in Americans.

Also, as you age, your bone marrow declines with regards to the number of cells in it, whereas the fat tissue maintains a pretty high number of stem cells, even in older individuals.

We can successfully harvest fat off of just about anyone, regardless of their age or how thin they are. The procedure is done under local [anesthesia], meaning that the patient stays awake. They dont have to go under general anesthesia. We can harvest as few as 15 cubic centimeters of fat, which is a very small amount of fat, and still get a very high number of stem cells, Ms. Comella says.

A stem cell procedure can cost anywhere from $5,000 to $15,000, depending on what is being done, and rarely if ever will insurance cover it.

Still, when compared it to the cost of long-term medications or the out-of-pocket cost of getting a knee replacement, stem cell therapy may still be a less expensive alternative.

Also, a single extraction will typically yield enough stem cells for 20 to 25 future treatments, should one decide to store stem cells for future needs.

I think it is accessible for patients, Ms. Comella says. Its an out-patient procedure. One should plan to be in clinic for about 2 hours; no real limitations afterwards, just no submerging in water, no alcohol, no smoking for a week. But other than that, patients can resume their normal activities and go about their regular daily lives.

Interestingly, Ms. Comella notes that patients who eat a very healthy diet, focusing on Organic and grass fed meat, have body fat that is very hearty and almost sticky, yielding high amounts of very healthy stem cells.

We can grow much better and faster stem cells from that fat than [the fat from] somebody who eats a grain-based diet or is exposed to a lot of toxins in their diet, she says. Their fat tends to be very fluffy, buttery yellow. The cells that come out of that are not necessarily as good a quality. Its just been very interesting. And of note, patients that are cigarette smokers, their fat is actually gray-tinged in color. The stem cells do not grow well at all.

The beauty of stem cell therapy is that it mimics a process that is ongoing in the human body all the time. Our stem cells are continuously promoting healing, and they do not have to be manipulated in any way. The stem cells naturally know how to hone in on areas of inflammation and how to repair damaged tissue.

All we are doing is harnessing the cells from one location where theyre sitting dormant and relocating them to exactly where we want them and we need them to work, Ms.Comella says. Basically, anything inside your body that is inflamed, that is damaged in some way, that is lacking blood supply, the [stem] cells can successfully treat.

That means orthopedics, knee injections, shoulder injections, osteoarthritis, acute injuries, anterior cruciate ligament tears in your back back pain associated with degenerative disc disease or damaged tendons or ligaments, herniated and bulging discs. You can also use it in systemic issues, everything from diabetes, to cardiac, to lungs any tissue organ inside your body thats been damaged.

Autoimmune diseases [can also be treated]. The stem cells are naturally immunosuppressant, meaning they can help quiet down an over reactive immune system and help the immune system function in a more normal way. Neurological diseases, traumatic brain injury, amyotrophic lateral sclerosis, Parkinsons. All of these have to do with tissue thats not functioning properly. The cells can be used to address that.

The list of different diseases that could benefit from this intervention is very impressive.

And one can dramatically improve the benefits of stem cell intervention by combining it with other healthy lifestyle factors that optimize mitochondrial function, such as eating a healthy Real Food diet, exercising, sleeping well, avoiding toxins and detoxifying from toxic influences.

Stem cells can be used as part of an anti-aging program. Ms. Comella has used stem cells on herself for several years, and report feeling better now than she did a decade ago.

The ability to reduce inflammation inside your body is basically making yourself live longer. Inflammation is what kills us all. Its what makes our telomeres shrink. Its what causes us pain and discomfort. Its what makes the tissues start to die. The ability to dose yourself with stem cells and bring down your inflammation, which is most likely caused by any sort of toxin that youve been exposed to breathing air is exposure to toxins this is going to lengthen your lifespan.

I typically will do a dose every 6 to 12 months, regardless of whats going on. If I have anything thats bothering me, if I tweak my knee at the gym, then I absolutely will come in and do an injection in my knee. I want to keep my tissue healthy for as long as possible.

I want to stay strong. I dont want to wait until something is wrong with me. I think that this is the future of medicine. This is what were going to start to see. People will begin to get their regular doses of [their own] stem cells and itll just be common practice.

Keep in mind there is a gradual and progressive decline in the quality and the number of stem cells as we age, so when considering this approach, it would be prudent and advantageous to extract and bank stem cells as early on as possible. There are stem cell banking services available.

Your stem cells are never as young as they are right now. Every minute that you live, your telomeres are shrinking. The ability to lock in the youth of your cells today can be very beneficial for you going forward, and for your health going forward. God forbid something happens. What if you have a heart attack? Youre not going to get clearance to get a mini-lipo aspirate procedure.

If you have your cells waiting in the bank, ready for you, it becomes very easy to pull a dose and do an IV delivery of cells. Its almost criminal that were not doing this for every single one of our cardiac patients. This should be standard practice. We should be having every single patient bank their stem cells at a young age and have them waiting, ready and available. The technology is there. We have it. Im not sure why this technology is not being made available to everyone,says.

I think stem cell therapy is very different than traditional medicine. Stem cell therapy may actually make it so that you dont have to be dependent on pharmaceutical medications. You can actually repair the tissue and thats it. This is a very different way of viewing medicine,Ms. Comella says.

The amniotic products available in the US are not so much stem cell products as they are growth factor products.

According to Ms. Comella, they can be useful in creating an immunomodulatory response, which can help to promote healing, but that differs from the living stem cell procedures that can be done by either isolating cells from body fat or bone marrow. As a general rule, clinical benefits are not achieved when using an amniotic product, primarily because they do not contain living stem cells.

I want to contrast that to what are called embryonic stem cells, Ms.Comella adds. The products obtained from cord blood, from women who are having babies, are not embryonic stem cells. Embryonic stem cells are when you are first bringing the egg and sperm together. Three days after that, you can isolate what is called an inner cell mass. This inner cell mass can be used to then grow cells in culture, or that inner cell mass could eventually lead to the formation of a baby.

Those are embryonic stem cells, and those are pluripotential, meaning that they have the ability to form an entire being, versus adult stem cells or stem cells that are present in amniotic tissue, [which] are multipotential, which only have the ability to form subsets of tissue.

When dealing with different diseases or damaged tissue or inflammation, mostly you want to repair tissue. If somebody has damage in their knee, they do nnot necessarily need embryonic cells because they do not need a baby in their knee. They need new cartilage in their knee.

Stem cell therapy is very different than traditional medicine. Stem cell therapy may actually make it so that we do have to depend on pharmaceutical medications. And we can actually repair the tissue and be done with it. This is a very different way of viewing medicine.

Eat healthy, Be healthy, Live lively

cells, damage, help, medication, medicine, muscle, patients, procedures, repair, stem, therapy, tissues, toxins

Paul A. Ebeling, polymath, excels in diverse fields of knowledge. Pattern Recognition Analyst in Equities, Commodities and Foreign Exchange and author of The Red Roadmasters Technical Report on the US Major Market Indices, a highly regarded, weekly financial market letter, he is also a philosopher, issuing insights on a wide range of subjects to a following of over 250,000 cohorts. An international audience of opinion makers, business leaders, and global organizations recognizes Ebeling as an expert.

Read the rest here:
Stem Cells 101, the Value Proposition - Live Trading News

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics, a biotechnology company developing therapies to improve and expand the use of curative stem cell transplantation for more patients, today announced rapid progress in advancing the companys strategic vision, including the completion of a $50 million Series B financing; in-licensing a clinical-stage program from Novartis to support the use of stem cell transplantation in a variety of disease settings; and a strategic partnership with Be The Match BioTherapiesSM, an organization offering solutions for delivering autologous and allogeneic cellular therapies.

The financing announced today is intended to fuel development of innovative product candidates across multiple aspects of transplantation medicine, including more precise preparation of patients, stem cell harvesting and stem cell expansion. The Series B round, which was oversubscribed, was led by GV (formerly Google Ventures), with participation from all existing investors, including Atlas Venture, Third Rock Ventures, Partners Innovation Fund and Access Industries. The financing also included Casdin Capital and other crossover investors, as well as Be The Match BioTherapies, a subsidiary of National Marrow Donor Program(NMDP)/Be The Match, the worlds leading organization focused on saving lives through bone marrow and umbilical cord blood transplantation.

Magenta has quickly established itself as a nexus of innovation in stem cell science, catalyzing interest in this area of medicine with the recognition that improvements will have profound impact on patients, said Jason Gardner, D. Phil., chief executive officer, president and cofounder of Magenta Therapeutics. We aspire to accelerate products that could unleash the potential of transplantation to more patients, including those with autoimmune diseases, genetic blood disorders and cancer. The resounding interest in Magenta from such a high-quality set of investors is a testament to our solid progress since launch, including building a world-class team and a robust pipeline, and generating promising early data.

MGTA-456: Investigational Product Addressing Significant Unmet Need in Stem Cell Transplant

The clinical-stage program in-licensed by Magenta from Novartis, MGTA-456 (formerly HSC835), aims to expand the number of cord blood stem cells used in transplants to achieve superior clinical outcomes compared to standard transplant procedures, and to enable more patients to benefit from a transplant. Under this agreement, Magenta gains rights to use MGTA-456 in selected applications and will develop MGTA-456 in multiple diseases, including immune and blood diseases.

Early results published in Science1 demonstrated the ability of MGTA-456 to significantly increase the number of umbilical cord blood stem cells. Clinical results reported in Cell Stem Cell2 demonstrated that this approach yielded an increased expansion of stem cells.

John E. Wagner, M.D., executive medical director of the Bone Marrow Transplantation Program at the University of Minnesota and the studys lead author, stated: MGTA-456 markedly shortens time to recovery, addressing one of the most significant challenges in stem cell transplantation today. MGTA-456 achieved a remarkable increase in the number of blood-forming stem cells, greater than that observed by all other methods that have been tested to date. This product has the potential to further improve cord blood transplant outcomes.

Be The Match BioTherapies Strategic Partnership Agreement

Magenta and Be The Match BioTherapies also announced today that in addition to the equity investment, the two organizations have initiated a collaboration to support their shared goals of improving transplant medicine. Magenta and Be The Match BioTherapies will explore opportunities to work together across all of Magentas research efforts, from discovery through clinical development. Under this agreement, Magenta may leverage Be The Match BioTherapies capabilities, including its cell therapy delivery platform, industry relationships, clinical trial design and management, and patient outcomes data derived from the NMDP/Be The Match, which operates the largest and most diverse marrow registry in the world. NMDP/Be The Match has a network of more than 486 organizations that support marrow transplant worldwide, including 178 transplant centers in the United States and more than 45 international donor centers and cooperative registries.

We are proud to have made our first equity investment as an organization in Magenta Therapeutics, and we share a vision to improve and advance the use of curative stem cell transplantation for patients with a wide range of diseases, said Amy Ronneberg, president of Be The Match Biotherapies.

About Magenta Therapeutics

Magenta Therapeutics is a biotechnology company harnessing the power of stem cell science to revolutionize stem cell transplantation for patients with immune- and blood-based diseases. By creating a platform focused on critical areas of transplant medicine, Magenta Therapeutics is pioneering an integrated, but modular approach to stem cell therapies to create patient benefits. Founded by internationally recognized leaders in stem cell transplant medicine, Magenta Therapeutics was launched in 2016 by Third Rock Ventures and Atlas Venture and is headquartered in Cambridge, Mass. For more information, please visitwww.magentatx.com.

About Third Rock Ventures

Third Rock Ventures is a leading healthcare venture firm focused on investing and launching companies that make a difference in peoples lives. The Third Rock team has a unique vision for ideating and building transformative healthcare companies. Working closely with our strategic partners and entrepreneurs, Third Rock has an extensive track record for managing the value creation path to deliver exceptional performance. For more information, please visit the firms website atwww.thirdrockventures.com.

About Atlas Venture

Atlas Venture is a leading biotech venture capital firm. With the goal of doing well by doing good, we have been building breakthrough biotech startups since 1993. We work side by side with exceptional scientists and entrepreneurs to translate high impact science into medicines for patients. Our seed-led venture creation strategy rigorously selects and focuses investment on the most compelling opportunities to build scalable businesses and realize value. For more information, please visitwww.atlasventure.com.

About GV

GV provides venture capital funding to bold new companies. In the fields of life science, healthcare, artificial intelligence, robotics, transportation, cyber security, and agriculture, GV's companies aim to improve lives and change industries. GV's team of world-class engineers, designers, physicians, scientists, marketers, and investors work together to provide these startups exceptional support on the road to success.

Launched as Google Ventures in 2009, GV is the venture capital arm of Alphabet, Inc. GV helps startups interface with Google, providing unique access to the worlds best technology and talent. GV has $2.4 billion under management and is headquartered in Mountain View, California, with offices in San Francisco, Boston, New York, and London. Launched as Google Ventures in 2009, GV is the venture capital arm of Alphabet, Inc. For more information, please visit http://www.gv.com.

About Be The Match BioTherapies

Be The Match BioTherapies partners with organizations pursuing new life-saving treatments in cellular therapy. Built on the foundation established over the last 30 years by theNMDP/Be The Match, the organization has unparalleled experience in personalized patient management with a single point of contact, cell sourcing and collection, cell therapy delivery platform, immunogenetics and bioinformatics, research and regulatory compliance. By leveraging proven capabilities and established relationships, Be The Match BioTherapies can bring customizable solutions to organizations in every stage of cellular therapy developmentfrom discovery through commercialization. Discover how Be The Match BioTherapies can assist your company atBeTheMatchBioTherapies.com.

For more information on todays announcement, see Jason Gardners post in the Life Sci VC blog: https://lifescivc.com/2017/05/building-a-bioteth-a-triple-play/.

1Science.2010 Sep 10;329(5997):1345-8. 2Cell Stem Cell.2016 Jan 7;18(1):144-55.

Read more here:
Magenta Therapeutics Advances Stem Cell Transplantation Strategy with $50 Million Series B Financing, Licensing of ... - Business Wire (press release)

Recommendation and review posted by Bethany Smith

Researchers have modified mouse stem cells to combat the kind of inflammation that arthritis and other conditions cause. The stem cells may one day be used in a vaccine that would fight arthritis and other chronic inflammation conditions in humans, a new paper suggests.

Such stem cells, known as SMART cells (Stem cells Modified for Autonomous Regenerative Therapy), develop into cartilage cells that produce a biologic anti-inflammatory drug that, ideally, will replace arthritic cartilage and simultaneously protect joints and other tissues from damage that occurs with chronic inflammation.

Researchers initially worked with skin cells from the tails of mice and converted those cells into stem cells. Then, using the gene-editing tool CRISPR in cells grown in culture, they removed a key gene in the inflammatory process and replaced it with a gene that releases a biologic drug that combats inflammation. The research is availablein the journal Stem Cell Reports.

Our goal is to package the rewired stem cells as a vaccine for arthritis, which would deliver an anti-inflammatory drug to an arthritic joint but only when it is needed, says Farshid Guilak, the papers senior author and a professor of orthopedic surgery at Washington University School of Medicine. To do this, we needed to create a smart cell.

Many current drugs used to treat arthritisincluding Enbrel, Humira, and Remicadeattack an inflammation-promoting molecule called tumor necrosis factor-alpha (TNF-alpha). But the problem with these drugs is that they are given systemically rather than targeted to joints. As a result, they interfere with the immune system throughout the body and can make patients susceptible to side effects such as infections.

We want to use our gene-editing technology as a way to deliver targeted therapy in response to localized inflammation in a joint, as opposed to current drug therapies that can interfere with the inflammatory response through the entire body, says Guilak, also a professor of developmental biology and of biomedical engineering and codirector of Washington Universitys Center of Regenerative Medicine.

If this strategy proves to be successful, the engineered cells only would block inflammation when inflammatory signals are released, such as during an arthritic flare in that joint.

As part of the study, Guilak and his colleagues grew mouse stem cells in a test tube and then used CRISPR technology to replace a critical mediator of inflammation with a TNF-alpha inhibitor.

We hijacked an inflammatory pathway to create cells that produced a protective drug.

Exploiting tools from synthetic biology, we found we could re-code the program that stem cells use to orchestrate their response to inflammation, says Jonathan Brunger, the papers first author and a postdoctoral fellow in cellular and molecular pharmacology at the University of California, San Francisco.

Over the course of a few days, the team directed the modified stem cells to grow into cartilage cells and produce cartilage tissue. Further experiments by the team showed that the engineered cartilage was protected from inflammation.

We hijacked an inflammatory pathway to create cells that produced a protective drug, Brunger says.

The researchers also encoded the stem/cartilage cells with genes that made the cells light up when responding to inflammation, so the scientists easily could determine when the cells were responding. Recently, Guilaks team has begun testing the engineered stem cells in mouse models of rheumatoid arthritis and other inflammatory diseases.

If the work can be replicated in animals and then developed into a clinical therapy, the engineered cells or cartilage grown from stem cells would respond to inflammation by releasing a biologic drugthe TNF-alpha inhibitorthat would protect the synthetic cartilage cells that Guilaks team created and the natural cartilage cells in specific joints.

When these cells see TNF-alpha, they rapidly activate a therapy that reduces inflammation, Guilak explains. We believe this strategy also may work for other systems that depend on a feedback loop. In diabetes, for example, its possible we could make stem cells that would sense glucose and turn on insulin in response. We are using pluripotent stem cells, so we can make them into any cell type, and with CRISPR, we can remove or insert genes that have the potential to treat many types of disorders.

With an eye toward further applications of this approach, Brunger adds, The ability to build living tissues from smart stem cells that precisely respond to their environment opens up exciting possibilities for investigation in regenerative medicine.

The National Institute of Arthritis and Musculoskeletal and Skin Diseases and the National Institute on Aging of the National Institutes of Health supported this work. The Nancy Taylor Foundation for Chronic Diseases; the Arthritis Foundation; the National Science Foundation; and the Collaborative Research Center of the AO Foundation in Davos, Switzerland, provided additional funding.

Authors Farshid Guilak and Vincent Willard have a financial interest in Cytex Therapeutics of Durham, North Carolina, which may choose to license this technology. Cytex is a startup founded by some of the investigators. They could realize financial gain if the technology eventually is approved for clinical use.

Source: Washington University at St. Louis

Visit link:
How 'smart' stem cells could lead to arthritis vaccine - Futurity - Futurity: Research News

Recommendation and review posted by simmons

"During acute infection, HIV actively replicates," explained co-senior study investigator Kamel Khalili, Ph.D., professor and chair of the department of neuroscience at LKSOM. "With EcoHIV mice, we were able to investigate the ability of the CRISPR/Cas9 strategy to block viral replication and potentially prevent systemic infection." The excision efficiency of their strategy reached 96% in EcoHIV mice, providing the first evidence for HIV-1 eradication by prophylactic treatment with a CRISPR/Cas9 system.

In the third animal model, a latent HIV-1 infection was recapitulated in humanized mice engrafted with human immune cells, including T cells, followed by HIV-1 infection. "These animals carry latent HIV in the genomes of human T cells, where the virus can escape detection, Dr. Hu explained. Amazingly, after a single treatment with CRISPR/Cas9, viral fragments were successfully excised from latently infected human cells embedded in mouse tissues and organs.

In all three animal models, the researchers employed a recombinant adeno-associated viral (rAAV) vector delivery system based on a subtype known as AAV-DJ/8. "The AAV-DJ/8 subtype combines multiple serotypes, giving us a broader range of cell targets for the delivery of our CRISPR/Cas9 system," remarked Dr. Hu. Additionally, the researchers re-engineered their previous gene-editing apparatus to now carry a set of four guide RNAs, all designed to efficiently excise integrated HIV-1 DNA from the host cell genome and avoid potential HIV-1 mutational escape.

To determine the success of the strategy, the team measured levels of HIV-1 RNA and used a novel and cleverly designed live bioluminescence imaging system. "The imaging system, developed by Dr. Won-Bin Young while at the University of Pittsburgh, pinpoints the spatial and temporal location of HIV-1-infected cells in the body, allowing us to observe HIV-1 replication in real time and to essentially see HIV-1 reservoirs in latently infected cells and tissues," stated Dr. Khalili.

The researchers were excited by their findings and are optimistic about their next steps. The next stage would be to repeat the study in primates, a more suitable animal model where HIV infection induces disease, in order to further demonstrate the elimination of HIV-1 DNA in latently infected T cells and other sanctuary sites for HIV-1, including brain cells," Dr. Khalili concluded. "Our eventual goal is a clinical trial in human patients."

See the original post here:

CRISPR Eliminates HIV in Live Animals - Genetic Engineering & Biotechnology News

Recommendation and review posted by simmons

Brian Nolan is a partner in the Life Sciences Group of Mayer Brown LLP.

The European Patent Office (EPO) and the United States Patent and Trademark Office (USPTO) have been thrust into a dispute pitting the University of California, Berkeley (UC) and the University of Vienna against the Broad Institute of MIT and Harvard University. The patent applications in the dispute disclose the genome editing tool called CRISPR/Cas9. Scientists and inventors have been watching the decisions of the EPO and USPTO because these decisions may leave one of these entities with a patent portfolio worth billions of dollars.

The Broad Institute scored the first victory at the USPTO, which held that the Institute is entitled to claims for the use of CRISPR-Cas9 in eukaryotic cells because that use is patentably distinct from UC's earlier disclosure showing the ability to use CRISPR-Cas9 in vitro. But just a few weeks later, the EPO informed the public that it intends to issue a patent to UC for the use of CRISPR-Cas9 in either prokaryotic or eukaryotic cells.

The CRISPR-Cas9 genome editing system finds its scientific roots in the observation that prokaryotes utilise CRISPR to identify and destroy viruses. Professor Jennifer Doudna of UC and Professor Emmanuelle Charpentier of the University of Vienna showed that the CRISPR-Cas9 system could use short strands of guide RNA that recognize and direct Cas9 to locate a specific target DNA sequence, and to cut it. In four preliminary patent applications filed between May 2012 and February 2013 and a related scientific paper published on 17 August, 2012, Professor Doudna and her colleagues Dr Martin Jinek of UC and Professor Charpentier disclosed a successful test of a prokaryotic CRISPR-Cas9 genome editing tool in vitro.

Around the same time, Dr Feng Zhang, a scientist at the Broad Institute, was working on creating and using various bacterial Cas9 in mammalian cells. Between December 2012 and June 2013, a few months after Professor Doudna and Dr Charpentier's publication, Professor Zhang and his colleagues filed several provisional patent applications showing the use of CRISPR-Cas9 with human and mouse cells. Professor Zhang and his colleagues requested and paid for expedited review. The expedited review resulted in US patents being issued to Professor Zhang and colleagues before the issuance of patents to Professor Doudna and her colleagues.

UC and the Broad Institute have been watching the progression of each other's patent applications through the USPTO and EPO. When UC saw that the USPTO had issued patents to the Institute for the use of CRISPR-Cas9 in eukaryotic cells, UC requested that the USPTO decide whether UC's patent disclosure showing the use of CRISPR-Cas9 genome editing system in vitro in prokaryotic cells evidenced that using CRISPR-Cas9 in eukaryotic cells was UC's invention. The USPTO instituted a proceeding called an 'interference' to review the parties' claims.

During this proceeding, UC alleged that Zhang's work was an obvious extension of that shown in Professor Doudna's article. Based upon an email from a former member of Professor Zhang's laboratory, Shuailiang Lin, to Professor Doudna, UC asserted that Professor Zhang began only meaningfully working on CRISPR-Cas9 after reading that article. The Institute responded by characterizing the email as that of a former employee seeking a new job and willing to tell a prospective employer what they wanted to hear.

In response to this, the Broad Institute responded that the USPTO should not have instituted this proceeding because its patent claims are distinct from UC's patent applications, which only show success of CRISPR-Cas9 in vitro. In February, the USPTO ruled in the Broad Institute's favour and held that 'the parties claim patentably distinct subject matter, rebutting the presumption created by declaration of this interference. [The] Broad [Institute] provided sufficient evidence to show that its claims, which are all limited to CRISPR-Cas9 systems in a eukaryotic environment, are not drawn to the same invention as UC's claims, which are all directed to CRISPR-Cas9 systems not restricted to any environment.'

The USPTO came to this conclusion because it determined that the evidence showed that a person of ordinary skill in the art would not have a reasonable expectation that CRISPR-Cas9 could be implemented successfully in a eukaryotic cell based upon the success UC achieved in vitro. To support its conclusion, the USPTO pointed to several statements by Professor Doudna and Dr Jinek, the UC inventors, that questioned whether the CRISPR-Cas9 genome editing system could be implemented in a eukaryotic environment. UC has appealed the USPTO's decision to a United States appellate court. Yet as the appellate court will make its decision on the same record presented to the USPTO, it may be difficult for UC to convince the appellate court that substantial evidence did not support the USPTO's determination. The appeal should be resolved with 10-12 months.

In parallel with the USPTO's review, the EPO was considering what, if any, patent protection it should grant UC. UC's European patent application was the subject of great interest, as several third-party observations were submitted to the EPO seeking to impede the issuance of the patent. These observations included arguments that the UC patent publications did not support a claim scope that encompassed CRISPR in a eukaryotic environment, similar to those presented in the United States. Despite these submissions, the EPO has notified the public that it intends to issue a patent to UC for the use of CRISPR-Cas9 in either prokaryotic or eukaryotic cells. Within nine months of issuance, this patent may be the subject of third-party oppositions that challenge whether the patent should have issued. If filed, an opposition will afford the third-party a substantive role in the arguments before the EPO than it had before, similar to the procedure in the US where a third-party cab remain involved during an interference proceeding. This may increase the likelihood of the EPO questioning a claim scope that includes use in a eukaryotic cell.

Either way, the EPO will be unlikely to resolve an opposition until late 2018 or 2019; so as with the patent situation in the United States, the dispute will continue in Europe. Unless the parties enter into a settlement, the industry should expect uncertainty with respect to ownership of the use of CRISPR-Cas9 in eukaryotic cells for the next two to four years, with the potential for various disputes to erupt in the patent offices and courts of the United States and Europe.

The decisions of the USPTO and the EPO to grant rights to the use of CRISPR-Cas9 systems in a eukaryotic cell to different parties has complicated commercialisation decisions for biopharma companies. It is thought that the more lucrative uses for CRISPR-Cas9 will be in a eukaryotic environment. Thus, if the status quo remains, throughout the duration of the patents that may last until 2033, companies will have to secure licensing rights from both UC and the Broad Institute if they plan on commercialising products in both the United States and Europe which is the likely plan for biopharma companies. Therefore, biopharma companies will continue to watch these CRISPR-Cas9 patent disputes until either the disputes are resolved - or a significantly different CRISPR system becomes the genome editing tool of choice.

View post:

The CRISPR patent dispute - Europe and the US - BioNews

Recommendation and review posted by simmons

They found that the protein kinase called Polo-like kinase 4 (PLK4), which is increased in AT/RT, regulates these tumors growth, survival and tendency to metastasize.

Interestingly, Sredni had performed a number of genomics studies previously, and this specific marker had never popped up before.

If I hadnt done this functional study, where, knocking out the PLK4 gene, resulted in the inhibition of cell proliferation I wouldnt have discovered that PLK4 is essential for AT/RT growth. PLK4 is only slightly elevated in these tumors. However, this is gene is tightly regulated and slight increases in its expression results in an aggressive tumor phenotype. This overexpression can be targeted by inhibitors opening a new therapeutic prospective for children with AT/RT. Significantly, we also found it elevated in other embryonal tumors of the brain, what may have a larger impact in patient care, Sredni said. The fact that we used CRISPR for the functional assay was key.

Even more promising, Sredni and her team were able to target the kinase with an available PLK4 inhibitor currently being tested for breast cancer that so far is providing promising results.

Srednis group experiments showed that the drug significantly impaired AT/RT cell proliferation, survival, migration and invasion.

More interestingly, it appears that the drug does not affect normal cells. The researchers tested the toxicity of the drug in normal human fibroblasts and in zebrafish larvae, exposing them to high doses of the drug for extended periods of time and found that it did not affect the fibroblasts and did not increase death or affected development of zebrafish larvae. This is a sign that the drug may be safe for use in pediatrics.

The team has now moved on to studies in mice with intracranial xenograft. While this is still under development, Sredni said it looks like tumors are actually growing significantly slower or even shrinking.

So Im knocking on wood here and hoping for good news soon she said.

CRISPR made a big impact on this research and Sredni, like many, think the tools reach will be broad.

The advent of CRISPR I think its going to change science from now on, in every aspect, she said. Its really user-friendly, robust and reliable.

Of course its not perfect, it still needs some optimization, Sredni acknowledged. But, it is powerful! For example in my hands, with a small team and the help of my summer students, we were able to mutate individual kinases, observe the cells phenotype and make significant new discoveries.

While she thinks progress in therapeutics will be more challenging than discovery, she added, CRISPR will really make a difference in what we will be doing from now on in cancer research.

Next, Sredni will perform high-throughput analyses on her mutated and treated cells in order to find downstream targets that will be adequate for combined therapies.

One drug alone wont cure cancer, she said.

The team is working on developing data for an application for federal funding in order to fully characterize the phenomenon observed so far. If all goes well, Sredni sees a Phase 1 clinical trial coming up soon.

The findings were published in Pediatric Blood & Cancer.

The rest is here:

Using CRISPR to Find Treatments for Aggressive Pediatric Brain Cancer - Bioscience Technology

Recommendation and review posted by simmons

Using CRISPR to editthe fusion genes that can cause or worsen cancer reduced the size of tumors and improved survival in mice, report researchers.

This is the first time that gene editing has been used to specifically target cancer fusion genes. It is really exciting because it lays the groundwork for what could become a totally new approach to treating cancer, explains lead study author Jian-Hua Luo, professor of pathology at University of Pittsburghs School of Medicine and director of its High Throughput Genome Center.

Fusion genes, which often are associated with cancer, form when two previously separate genes become joined together and produce an abnormal protein that can cause or promote cancer.

Luo and his team had previously identified a panel of fusion genes responsible for recurrent and aggressive prostate cancer. In a study published earlier this year in the journal Gastroenterology, the team reported that one of these fusion genes, known as MAN2A1-FER, also is found in several other types of cancer, including that of the liver, lungs, and ovaries, and is responsible for rapid tumor growth and invasiveness.

In the current study, published online in Nature Biotechnology, the researchers employed the CRISPR-Cas9 genome editing technology to target unique DNA sequences formed because of the gene fusion. The team used viruses to deliver the gene editing tools that cut out the mutated DNA of the fusion gene and replaced it with a gene that leads to death of the cancer cells.

Because the fusion gene is present only in cancer cells, not healthy ones, the gene therapy is highly specific. Such an approach could come with significantly fewer side effects when translated to the clinic, which is a major concern with other cancer treatments such as chemotherapy.

To conduct the study, the researchers used mouse models that had received transplants of human prostate and liver cancer cells. Editing the cancer fusion gene resulted in up to 30 percent reduction in tumor size. None of the mice exhibited metastasis and all survived during the eight-week observation period.

In contrast, in control mice treated with viruses designed to cut out another fusion gene not present in their tumors, the tumors increased nearly 40-fold in size, metastasis was observed in most animals, and all died before the end of the study.

The new findings suggest a completely new way to combat cancer. Other types of cancer treatments target the foot soldiers of the army. Our approach is to target the command center, so there is no chance for the enemys soldiers to regroup in the battlefield for a comeback, says Luo.

Another advantage over traditional cancer treatment is that the new approach is very adaptive. A common problem that renders standard chemotherapies ineffective is that the cancer cells evolve to generate new mutations. Using genome editing, the new mutations could be targeted to continue fighting the disease, Luo notes.

In the future, the researchers plan to test whether this strategy could completely eradicate the disease rather than induce the partial remission observed in the current study.

Grants from the National Institutes of Health, the Department of Defense, and the University of Pittsburgh Cancer Institute supported this work.

Source: University of Pittsburgh

Link:

Using CRISPR against cancer shows success in mice - Futurity - Futurity: Research News

Recommendation and review posted by simmons

Schulman IRBhas launched a new service to ensure sponsors, CROs, and others are compliant when conducting genetic engineering research given the diverse risks, says committee lead.

The institutional biosafety committee (IBC) service will be headed by Dr. Daniel Eisenman, Ph.D., RBP, SM(NRCM), CBSP, who told us the service provides a means of obtaining regulatory approvals to perform gene therapy research and research involving genetic engineering.

The field of gene therapy is no longer science fiction as the US, Europe, and China have started approving genetically modified therapeutics in recent years, Eisenman added.

Technological advances have created a wealth of potential genetically modified therapeutics that are either ready or on the verge of being ready for clinical trials, and many clients have been asking us to provide a commercial IBC service.

As Eisenman explained, IBC review often comes as a surprise to clinical researchers not familiar with gene therapy research requirements though IBC review shouldnt be an obstacle to conducting innovative research, he said.

Eisenman explained NIH guidelines require approval from both an IBC and an IRB given the diverse risks associated with gene therapy research. As such, Schulman will offer its IBC services as well as central IRB services for a coordinated review process.

Rather than having to wait for one committees approval before the other review can begin, conducting the reviews concurrently means the committees work together collaboratively, which accelerates the regulatory approval process, said Eisenman.

The new IBC service offers complete setup, registration support, and administrative resources for sites to operate an NIH-compliant IBC.

The service will support clinical, pre-clinical and non-clinical research, providing all components to complement an existing IBC or build and administer an entirely new IBC from the ground up, Eisenman added.

After conducting a risk assessment, the committee focuses on the associated containment and safety practices while looking at occupational safety as well as the protection of the community and environment surrounding the research site to ensure a comprehensive risk mitigation plan is in place.

See the original post here:
Gene therapy no longer science fiction: IRB launches biosafety committee service - OutSourcing-Pharma.com

Recommendation and review posted by simmons

Using the gene-editing tool CRISPR/Cas9, researchers at University of California San Diego School of Medicine and Shiley Eye Institute at UC San Diego Health, with colleagues in China, have reprogrammed mutated rod photoreceptors to become functioning cone photoreceptors, reversing cellular degeneration and restoring visual function in two mouse models of retinitis pigmentosa.

The findings are published in the April 21 advance online issue ofCell Research.

Retinitis pigmentosa (RP) is a group of inherited vision disorders caused by numerous mutations in more than 60 genes. The mutations affect the eyes' photoreceptors, specialized cells in the retina that sense and convert light images into electrical signals sent to the brain. There are two types: rod cells that function for night vision and peripheral vision, and cone cells that provide central vision (visual acuity) and discern color. The human retina typically contains 120 million rod cells and 6 million cone cells.

In RP, which affects approximately 100,000 Americans and 1 in 4,000 persons worldwide, rod-specific genetic mutations cause rod photoreceptor cells to dysfunction and degenerate over time. Initial symptoms are loss of peripheral and night vision, followed by diminished visual acuity and color perception as cone cells also begin to fail and die. There is no treatment for RP. The eventual result may be legal blindness.

In their published research, a team led by senior author Kang Zhang, MD, PhD, chief of ophthalmic genetics, founding director of the Institute for Genomic Medicine and co-director of biomaterials and tissue engineering at the Institute of Engineering in Medicine, both at UC San Diego School of Medicine, used CRISPR/Cas9 to deactivate a master switch gene calledNrland a downstream transcription factor calledNr2e3.

CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, allows researchers to target specific stretches of genetic code and edit DNA at precise locations, modifying select gene functions. Deactivating eitherNrlorNr2e3reprogrammed rod cells to become cone cells.

"Cone cells are less vulnerable to the genetic mutations that cause RP," said Zhang. "Our strategy was to use gene therapy to make the underlying mutations irrelevant, resulting in the preservation of tissue and vision."

The scientists tested their approach in two different mouse models of RP. In both cases, they found an abundance of reprogrammed cone cells and preserved cellular architecture in the retinas. Electroretinography testing of rod and cone receptors in live mice show improved function.

Zhang said a recent independent study led by Zhijian Wu, PhD, at National Eye Institute, part of the National Institutes of Health, also reached similar conclusions.

The researchers used adeno-associated virus (AAV) to perform the gene therapy, which they said should help advance their work to human clinical trials quicker. "AAV is a common cold virus and has been used in many successful gene therapy treatments with a relatively good safely profile," said Zhang. "Human clinical trials could be planned soon after completion of preclinical study. There is no treatment for RP so the need is great and pressing. In addition, our approach of reprogramming mutation-sensitive cells to mutation-resistant cells may have broader application to other human diseases, including cancer."

Go here to see the original:
Using CRISPR to Reverse Retinitis Pigmentosa, Restore Visual Function - Drug Discovery & Development

Recommendation and review posted by simmons

Be The Match BioTherapies announces collaboration agreement with Magenta Therapeutics.

MINNEAPOLIS, May 2, 2017 Be The Match BioTherapiesSM, an organization offering solutions for delivering autologous and allogeneic cellular therapies, today announced that it has entered into a strategic partnership with Magenta Therapeutics, a biotechnology company developing therapies to improve and expand the use of curative stem cell transplantation. The collaboration is intended to support efforts to improve transplant outcomes and expand the application of stem cell transplantation into disease indications that include autoimmunity, serious inherited immune and metabolic disorders, blood defects and blood cancers. Be The Match BioTherapies also announced today that it is participating in Magentas Series B financing round, its first equity investment as an organization.

Under the terms of the collaboration agreement, Be The Match BioTherapies and Magenta will explore opportunities to work together across Magentas discovery, clinical development and product delivery efforts. The collaboration leverages a wide range of Be The Match BioTherapies research assets and services, including the National Marrow Donor Program(NMDP)/Be The Match marrow registry, the largest in the world with nearly 16 million volunteer marrow donors. Magenta may also collaborate with Be The Match BioTherapies in the design of clinical trials and of its cell therapy delivery platform and services.

Partnering with Magenta in its efforts to revolutionize the current state of stem cell transplantation aligns with our core mission to help organizations deliver cellular therapies that save more lives and improve the quality of life for patients, said Amy Ronneberg, President of Be The Match BioTherapies. Our collaboration with Magenta exemplifies how cell and gene therapy companies can benefit from our robust network of products and services regardless of where they are in the development life cycle. We look forward to lending our expertise in cellular therapy and leveraging our deep-rooted relationships, partnerships and global infrastructure to support the development of powerful treatment options with great potential to improve patient outcomes in a range of disease areas.

Jason Gardner, D. Phil., Chief Executive Officer, President, and Cofounder of Magenta, added: We believe that Be The Match BioTherapies extensive experience and network in stem cell transplant medicine, coupled with Magentas work in patient conditioning and stem cell harvesting and growth, could accelerate our development path and ability to positively impact patients lives.

Be The Match BioTherapies launched in 2016 as a subsidiary of NMDP/Be The Match, the national organization with a 30-year history of connecting patients with their donor match for a life-saving bone marrow or umbilical cord blood transplant. As experts in providing services and expertise to organizations pursuing life-saving treatments in the cellular therapy space, Be The Match BioTherapies aims to help critically ill patients who can benefit from these treatments.

(Source:Business Wire)

Follow us onTwitterandFacebookfor updates on the latest pharmaceutical and biopharmaceutical manufacturing news!

Originally posted here:
Cell Therapy Company Joins Forces with Biotech in New Transplantation Research Alliance - Pharmaceutical Processing

Recommendation and review posted by Bethany Smith

High Point Observer

Dimension Therapeutics Inc (DMTX) Upgraded at Zacks Investment Research Markets Daily According to Zacks, Dimension Therapeutics, Inc. is a biotechnology company which focuses on developing novel, liver-directed gene therapy treatments for severe, rare genetic disorders. The company's pipeline of programs includes DTX101, a lead gene ... Dimension Therapeutics (DMTX) Earning Favorable News Coverage, Report ShowsSports Perspectives all 4 news articles »

See the original post:
Dimension Therapeutics Inc (DMTX) Upgraded at Zacks Investment Research - Markets Daily

Recommendation and review posted by sam

SOUTH SAN FRANCISCO, CA--(Marketwired - May 01, 2017) - VistaGen Therapeutics Inc. (NASDAQ: VTGN), a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders, announced today that its largest institutional stockholder, holding both common stock and substantially all (99.3%) of the Company's outstanding preferred stock, entered into a 6-month lock-up agreement. Under the agreement, the stockholder and its affiliates agreed to not enter into any transaction involving the Company's securities during the term of the agreement, which runs through late-October 2017 and covers approximately 36% of the Company's issued and outstanding equity securities on an as converted basis.

About VistaGen

VistaGen Therapeutics, Inc. (NASDAQ: VTGN), is a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders. VistaGen's lead CNS product candidate, AV-101, is in Phase 2 development as a new generation oral antidepressant drug candidate for major depressive disorder (MDD). AV-101's mechanism of action is fundamentally differentiated from all FDA-approved antidepressants and atypical antipsychotics used adjunctively to treat MDD, with potential to drive a paradigm shift towards a new generation of safer and faster-acting antidepressants. AV-101 is currently being evaluated by the U.S. National Institute of Mental Health (NIMH) in a Phase 2 monotherapy study in MDD being fully funded by the NIMH and conducted by Dr. Carlos Zarate Jr., Chief, Section on the Neurobiology and Treatment of Mood Disorders and Chief of Experimental Therapeutics and Pathophysiology Branch at the NIMH. VistaGen is preparing to launch a 180-patient Phase 2 study of AV-101 as an adjunctive treatment for MDD patients with inadequate response to standard, FDA-approved antidepressants. Dr. Maurizio Fava of Harvard University will be the Principal Investigator of the Company's Phase 2 adjunctive treatment study. AV-101 may also have the potential to treat multiple CNS disorders and neurodegenerative diseases in addition to MDD, including chronic neuropathic pain, epilepsy, and symptoms of Parkinson's disease and Huntington's disease, where modulation of the NMDAR, AMPA pathway and/or key active metabolites of AV-101 may achieve therapeutic benefit.

VistaStem Therapeutics is VistaGen's wholly owned subsidiary focused on applying human pluripotent stem cell technology, internally and with collaborators, to discover, rescue, develop and commercialize proprietary new chemical entities (NCEs), including small molecule NCEs with regenerative potential, for CNS and other diseases, and cellular therapies involving stem cell-derived blood, cartilage, heart and liver cells. In December 2016, VistaGen exclusively sublicensed to BlueRock Therapeutics LP, a next generation regenerative medicine company established by Bayer AG and Versant Ventures, rights to certain proprietary technologies relating to the production of cardiac stem cells for the treatment of heart disease.

For more information, please visit http://www.vistagen.com and connect with VistaGen on Twitter, LinkedIn and Facebook.

Forward-Looking Statements

The statements in this press release that are not historical facts may constitute forward-looking statements that are based on current expectations and are subject to risks and uncertainties that could cause actual future results to differ materially from those expressed or implied by such statements. Those risks and uncertainties include, but are not limited to, risks related to the successful launch, continuation and results of the NIMH's Phase 2 (monotherapy) and/or the Company's planned Phase 2 (adjunctive therapy) clinical studies of AV-101 in MDD, and other CNS diseases and disorders, protection of its intellectual property, and the availability of substantial additional capital to support its operations, including the Phase 2 clinical development activities described above. These and other risks and uncertainties are identified and described in more detail in VistaGen's filings with the Securities and Exchange Commission (SEC). These filings are available on the SEC's website at http://www.sec.gov. VistaGen undertakes no obligation to publicly update or revise any forward-looking statements.

Read more:
VistaGen Therapeutics' Largest Stockholder Signs 6-Month Lock-Up Agreement - Marketwired (press release)

Recommendation and review posted by Bethany Smith

Sat, Apr 29, 2017, 01:00 Updated: Sat, Apr 29, 2017, 10:12

Irish cardiologists have found a way to repair damaged cardiac muscle and reduce the risk of future heart failure by injecting a growth promoter into the hearts of heart attack sufferers. Photograph: Getty Images

A team of Irish cardiologists have shown that injecting an insulin-like growth promoter into the hearts of patients who have suffered a severe heart attack can repair damaged cardiac muscle and reduce the risk of future heart failure.

Prof Noel Caplice, Chair of Cardiovascular Sciences at University College Cork, and his cardiologist colleagues at Cork University Hospital successfully tested the growth factor in a clinical trial involving 47 patients who presented at the Cork hospital after experiencing heart attacks.

Prof Caplice said 20 per cent of people who suffer heart attacks have severe ongoing difficulties because of lasting damage to heart muscle even after the best current therapies.

After you have a heart attack, regardless whether you treat it with a stent or whatever, about 20 per cent of patients go on to have poor remodelling heart muscle cells die, you get scar tissue forming and the heart tends to expand and dilates, a bit like a balloon, and you get thinned-out heart muscle.

With that poor remodelling of the heart, the heart as a structure performs much worse, it doesnt work very well in terms of its function that leads to a substantial number of those patients going on to suffer heart failure with an increased risk of death, he said.

However, 10 years ago, Prof Caplice and his team began looking at using stem cells as a means of repairing damaged tissue and they found a protein within the stem cells, IGF 1, previously used to treat congenital dwarfism and growth problems, was leading to the repair of damaged heart muscle.

IGF 1 acts differently to insulin in that it acts on a different receptor in the body and when we inject it, it gets into the heart tissue and it basically stimulates receptors on the surface of the cardiac cells and in about 30 minutes, it sends a survival signal to the heart muscles cells, he said.

What we discovered from the stem cell study was that the concentration of the factor was extremely low so what we did was that we took the purified factor and in studies with pigs we injected them in the context of a heart attack and we found these major remodelling benefits.

Those animal tests were funded by Science Foundation Ireland but four years ago the Health Research Board came on board and the two bodies provided a 1 million grant to allow the treatment be trialled on humans.

Working with a 25-strong team incorporating cardiologists, radiologists, MRI specialists and nurses, Prof Caplice was able to incorporate the IGF 1 trials into the treatment of patients attending CUH with severe cardiac events and over the past three years have trialled it on 47 patients.

Patients received two different low-dose preparations of insulin-like growth factor or placebo in a randomised double-blinded clinical trial, with results showing those who received the higher dose had improved remodelling of their heart muscle in the two-month follow-up after their heart attack.

Prof Caplice said the CUH trials, the results of which he will present at a European Society of Cardiology conference in Paris on Saturday, were the first use of IGF 1 in human hearts and part of its attractiveness was its low dosage ensuring minimal side effects while improving cardiac structure.

Among the beneficiaries was John Nolan from New Ross who suffered a heart attack in December 2014. I feel I was blessed to be asked to be involved; I had confidence that good would come from it, in terms of how they explained it to me. Looking back on it now, I feel it was the right choice.

For Prof Caplice, the challenge now is to expand the trials to several hundred patients possibly across different countries and different healthcare systems to see if the IGF 1 treatment is globally applicable which, if proven to be the case, could lead to regulatory approval within five years.

Read the original post:
Irish researchers 'cut risk of heart failure with one injection' - Irish Times

Recommendation and review posted by simmons

Anna-Lena Ahlstrm, Royal Court, Sweden

Princess Christina of Sweden, the youngest of King Carl XVI Gustafs four older sisters, has successfully undergone a stem cell transplant.

Swedish newspaper Expressen first reported the news with a confirmation from the Swedish Royal Courts Director of Information and Press Department,Margareta Thorgren. She explained to them, The stem cell operation is completed. Princess Christina is well under the circumstances.

The Princess will remain at home during her recuperation. After such operations, the immune system is considerably weakened, and as a result, doctors commonly advise patients stay isolated while they heal.

It was just last month that the Court made the announcement of the pending transplant, which can be stressful on the body,saying, Princess Christina, Mrs Magnuson has, since October, been treated for blood cancer with regular chemotherapy. The treatment has gone well. But the Princesss blood cancer cannot be cured with this treatment because it occurred in bone marrow stem cells that are resistant to chemotherapy.

In consultation with the family and doctors, the Princess has decided to undergo a stem cell transplant.

She was diagnosed with chronicleukaemia in October of last year. At the time, the Swedish Royal Court said that she was feeling relatively good. It was stated that the73-year-old would scale back her royal duties during her treatmentbut would fulfil her commitments when her health allowed.They also asked that she be able to undergo her chemotherapy in peace.

In 2010, Christina announced that she had undergone treatment for breast cancer including three surgeries and had beaten the disease. After defeating breast cancer, Christina devoted much of her time to bringing attention to cancer issues.

The Princess was born on 3 August 1943 at Haga Palace in Solna, Sweden. She married Tord Magnuson in 1974 at the Royal Chapel in Stockholm Palace. They have three sons: Gustaf, Oscar, and Victor.

See the original post here:
Princess Christina of Sweden undergoes successful stem cell transplant - Royal Central

Recommendation and review posted by Bethany Smith