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Drugs already in medicine cabinets may fight dementia, early data suggests – Ars Technica

Posted: April 26, 2017 at 3:42 pm

Enlarge / Oh, there's that cure I was looking for.

Tried, true, and FDA-approved drugs for cancer and depressionalready in medicine cabinetsmay also be long-sought treatments for devastating brain diseases such as Alzheimers, Parkinsons, and other forms of dementia, according to a new study in Brain, a Journal of Neurology.

The research is still in early stages; it only involved mouse and cell experiments, which are frequently not predictive of how things will go in humans. Nevertheless, the preliminary findings are strong, and scientists are optimistic that the drugs couldone day help patients with progressive brain disease. Researchers are moving toward human trials. And this process would be streamlined because the drugs have already cleared safety tests. But even if the early findings hold up, it would still take years to reach patients.

In the preliminary tests, the two drugstrazodone hydrochloride, used to treat depression and anxiety, and dibenzoylmethane (DBM), effective against prostate and breast tumorscould shut down a devastating stress response in brain cells, known to be critical for the progression of brain diseases. The drugs both protected brain cells and restored memory in mice suffering from brain diseases.

"We're excited by the potential of these findings from this well-conducted and robust study, Doug Brown, of the Alzheimer's Society, told the BBC.

David Dexter, from Parkinson's UK, added that if these studies were replicated in human clinical trials, both trazodone and DBM could represent a major step forward.

For years, researchers have known that a stress response in cells, called unfolded protein response, or UPR, is involved in a bunch of neurodegenerative diseases. The response kicks in when theres a buildup of unfolded or misfolded proteins. Typically, protein chains are folded into specific 3D structures that are often critical for their function in the body. But this folding goes awry in some neurodegenerative conditions, such as prion diseases, Alzheimer's disease, Parkinson's disease, and other forms of dementia.

When this happens, UPR kicks in. It shuts down protein production, tries to junk the botched proteins, and gets protein production machinery back in order. If all goes well, the cell can resume normal protein production. But if it doesnt, UPR initiates apoptosis, aka cell suicide.

In neurodegenerative diseases, things dont go well; UPR is over-activated, and brain cells start dying off. Scientists know that hampering UPR can protect brain cells and restore memory in mice engineered to mimic having Alzheimer's disease. But so far, all the compounds found to knock back UPR were highly toxic or highly insoluble (they dont work as medicine).

For a drug discovery shortcut, researchers at the University of Cambridge wondered: do we already have drugs that can interfere with UPRbut just dont know it? They screened a library of 1,040 FDA-approved drugs to find out.

Because UPR is highly conserved across animals, the researchers could use worms to screen the drugs. They initially found 20 drugs that seemed to have UPR-dampening effects. Upon further testing, they whittled down the list to five, then to two.

In further cell experiments, both trazodone and DBM inhibited a specific step in UPR and restored protein production. In mice, the drugs traversed the blood-brain barrier. When the researchers infected mice with a prion disease, clinically relevant doses of either drug held back neurological symptoms, boosted survival, and substantially reduced loss of brain cells in most of the infected mice. In mice that modeled a type of dementia, called frontotemporal dementia, both drugs could rescue the rodents memory and restore protein synthesis in brain cells.

The two drugs were markedly neuroprotective, the authors conclude. These drugs therefore represent an important step forward in the pursuit of disease-modifying treatments for Alzheimers and related disorders.

Trazodone, the authors note, is even already approved for use in the elderly. Clinical trials are the next step.

Brain, 2017. DOI: 10.1093/brain/awx074 (About DOIs).

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Henrietta Lacks And Modern Medicine’s Greatest Unpaid Debt – Huffington Post

Posted: April 26, 2017 at 3:42 pm

On Saturday evening I, along with millions of other people, watched the premiere of Auntie Oprahs film, The Immortal Life of Henrietta Lacks, on HBO. At the beginning of the film, I was nestled in my bed, eating a bowl of popcorn. I knew the premise of the movie and seriously, who doesnt love a good Oprah Winfrey performance (and let me just say, as usual, she delivered). But by the middle of the film, I was no longer lying comfortably, instead I was sitting upright, slightly teary-eyed...and by the end, there was popcorn on the floor and I was standing in front my television, gripping my remote tightly and shaking my head in disbelief. I immediately scrolled through Facebook to see if I was alone, and just as I imagined, I wasnt.

Several people with skin like mine who watched the film felt just like I did. Dont get me wrong, the movie was well done and the acting was superb, but it did something that I wasnt expecting. It evoked emotions that are still somewhat difficult for me to articulate. Simply put, I hurt for Henrietta Lacks and her family.

As I watched the movie, I thought to myself, why didnt I learn more about Henny (Henny because shes now my auntie in my head) in school? From grades 7-12, I attended a health sciences magnet school. In college, I was a biology major. To say I know a little something about HeLa is an understatement, but I honestly dont recall hearing much about the owner of those magical cells. Yes, magical. Black girl magic isnt just a hashtag, you know. But I digress. Like many Black women, Henrietta Lacks was an unknown hero who, thanks to the book and subsequent film about her life, is no longer an obscure entity. However, unlike the three heroines in one of my favorite movies, Hidden Figures, Henriettas story did not have such a happy ending.

Henrietta was diagnosed with cervical cancer and died from the disease at the age of 31. As a result, she left a special legacy and five children whose lives would never be the same. But it wasnt her untimely death that shocked me as that was quite common back in the day. No, it was the blatant disregard for her basic human rights that I found so appalling. To which human right am I referring? Henrietta was not given the right to consent, and even though it was not required at that time, ethically, she should have been asked for the use of her cells.

Picture this. Youre a young woman, lying in a hospital bed, gravely ill and undergoing treatment for a disease you dont fully understand. There are doctors and nurses coming in and out of your room. They are talking to you, smiling at you, and poking you with needles. They spread your legs to take a piece of your cervix so they can figure out how to fix you. Its ok because you trust them. But not one time do they ask you if they can use the cells from your body to learn more about your disease. No one takes the time to explain to you how the use of your cells could possibly benefit others for years to come. And after you pass away, no one consults your grieving family members to obtain permission to continue the use of your cells.

Mrs. Lacks, we need your permission for something. Can we use the cells from your biopsy for research? Theres a possibility that you could help us understand this disease better. You could also be helping others.

I imagine a conversation like that with Henrietta would have sufficed, but that didnt happen. So lets not sugarcoat it. Henriettas cells were stolen. Her cells were taken and used to advance research and medical practices and she went virtually unknown for years. And to make matters worse, her family still has not been properly compensated. Unbelievable, I know. Now, some may wonder whats so great about Henriettas cells anyway. Why does her family deserve anything after all of this time? Well, let me explain.

Henrietta cells, i.e., HeLa, were unique. You may say she was an ordinary woman with extraordinary cells. HeLa cells are an immortalized cell line, meaning the cells can be reproduced infinitely in a lab. Her cells have aided in the advancement of science and modern medicine tremendously. For example, the cells were used to develop the polio vaccine. In addition, they have been instrumental in HIV and cancer research. Oh, but it doesnt stop there. These cells even paved the way for in vitro fertilization, and the list goes on and on. And while I am thankful for the progress Henriettas cells have afforded medical research, the treatment of Blacks as guinea pigs in the past has left a very bitter taste in my mouth.

Real talk. What occurred to Henrietta wasnt an isolated incident. We all know about the Tuskegee Experiment the time scientists used Black men as lab animals to study the effects of Syphilis but I wonder just how many stories like Henriettas and the Tuskegee Experiment are still untold? And why have the contributions of so many Blacks been undervalued or erased from the history books? And lastly, why is health equity still so difficult to attain in 2017. What do I mean by that last question? Well, for example, Black women are still dying from breast and cervical cancers at higher rates than other ethnic/racial groups. The same goes for Black men and prostate cancer. I could go on but you get the point. The fact is, I dont know the answers to these questions, and as a public health professional, I am working everyday to identify solutions. But what I do know is this, modern medicine has a huge unpaid debt. The creditors are the descendants of Mrs. Henrietta Lacks, and I, for one, think its time to pay up.

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Kelly Osbourne campaigning to make stem cell therapy affordable in America – Hollywood.com

Posted: April 26, 2017 at 3:41 pm

Singer-turned-TV personality Kelly Osbourne wants to help cure fellow Lyme disease sufferers by making stem cell therapy available for all in the U.S. Ozzy and Sharon Osbournes daughter contracted the condition after she was bitten by a tick during a party for the rockers 56th birthday back in 2004, when her mother had a reindeer sanctuary installed at their Los Angeles home. However, Kelly wasnt properly diagnosed until 2014, months after suffering a seizure while filming an episode of E!s Fashion Police show in 2013, when doctors claimed her collapse had been caused by epilepsy. She did some research into her ailments and discovered she was actually struggling with Lyme disease, and promptly sought out alternative treatment to help her overcome the illness. I started to actually do the one thing doctors tell you not to do and thats to go online and look it up, she explained on U.S. breakfast show Good Morning America, and all roads pointed to Lyme disease so I found a doctor through my mum. I went to Frankfurt, Germany, and I did stem cell (therapy) and I got cured, Kelly claimed. The 32-year-old is lucky to have been in a position to afford the treatment, which involves the transplant of stem cells to heal those damaged by the disease, and now she is looking to get involved in making the therapy more widely available and affordable to others less fortunate. It sickens me that thats not available to everyone and that you have to be considered lucky or privileged to get that sort of treatment, she said. I want to make sure and I will do anything that I can do to make sure that that treatment is available in this country. Kelly details her experience with the bacterial infection in her new memoir There Is No F**king Secret: Letters from a Bada** B**ch. She isnt the only celebrity to open up about her struggles with Lyme disease pop star Avril Lavigne, and veteran model Yolanda Hadid and her runway star kids Bella and Anwar Hadid have also been battling the illness.

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Are baby, wisdom teeth the next wave in stem cell treatment? – CNN

Posted: April 26, 2017 at 3:41 pm

It's based on experimental research that suggests stem cells extracted from the pulp of these teeth might someday regrow a lost adult tooth or offer other regenerative medicine benefits -- some potentially life-saving.

"So I'll try not to get emotional here, but my husband was diagnosed with acute myeloid leukemia in 2011," said Bassetto, of Naperville, Illinois, head of a sales team at a software company.

In 2012, her husband, James, had a stem cell transplant to restore his bone marrow and renew his blood.

"He was very fortunate. He was one of six kids, and his brother was a perfect match," she said. She noted that her two children, Madeline, 23, and Alex, 19, may not be so lucky if they develop health problems, since they have only each other; the chance of two siblings being a perfect stem cell match is only 25%.

Unfortunately, her husband's stem cell transplant was not successful. He developed graft-versus-host disease, where his brother's donated stem cells attacked his own cells, and he died shortly afterward.

However, she says, the transplant had given him a chance at a longer life.

Last year, when her son saw a dentist for wisdom tooth pain, a brochure for dental stem cell storage caught Bassetto's eye and struck a chord.

"I know stem cells have tremendous health benefits in fighting disease, and there's a lot ways they're used today," she said. "Had my husband had his own cells, potentially, his treatment could have been more successful."

Medical breakthroughs happen all the time, said Bassetto. "Who knows what potential there is 20 years, 40 years down the road, when my son is an adult or an aging adult?

"Almost like a life insurance policy, is how I viewed it," she said.

Some scientists see storing teeth as a worthwhile investment, but others say it's a dead end.

"Research is still mostly in the experimental (preclinical) phase," said Ben Scheven, senior lecturer in oral cell biology in the school of dentistry at the University of Birmingham. Still, he said, "dental stem cells may provide an advantageous cell therapy for repair and regeneration of tissues," someday becoming the basis for reconstructing bone tissue, retinas and even optic neurons.

Dr. Pamela Robey, chief of the craniofacial and skeletal diseases branch of the National Institute of Dental and Craniofacial Research, acknowledges the "promising" studies, but she has a different take on the importance of the cells.

"There are studies with dental pulp cells being used to treat neurological disorders and problems in the eye and other things," Robey said. The research is based on the idea that these cells "secrete factors that encourage local cells to begin the repair process."

"The problem is, these studies have really not been that rigorous," she said, adding that many have been done only in animals and so provide "slim" evidence of benefits. "The science needs a lot more work."

Robey would know. Her laboratory discovered dental stem cells in 2003.

"My fellows, Songtao Shi and Stan Gronthos, did the work in my lab," Robey said. "Songtao Shi is a dentist, and basically he observed that, when you get a cavity, you get what's called 'reparative dentin.' In other words, the tooth is trying to protect itself from that cavity, so it makes a little bit of dentin to kind of plug the hole, so to speak."

Dentin is the innermost hard layer of tooth that lies beneath the enamel. Underneath the dentin is a soft tissue known as pulp, which contains the nerve tissue and blood supply.

Observing dentin perform reparative work, Shi hypothesized that this must mean there's a stem cell within the tooth that's able to activate and make dentin. So if you wanted to grow an adult tooth instead of getting an implant, knowing how to make dentin would be the start of the process, explained Robey.

Pursuing this idea, Shi, Gronthos and the team conducted their first study with wisdom teeth. They discovered that pulp cells in these third molars did indeed make dentin, but the cells found in baby teeth, called SHED (stem cells from human exfoliated deciduous teeth), had slightly different properties.

"The SHED cells seem to make not only dentin but also something that is similar to bone," Robey said. This "dentin osteogenic material" is a little like bone and a little like dentin -- "unusual stuff," she said.

There is a meticulous process for extracting stem cells from the pulp.

"We very carefully remove any soft tissue that's adhering to the tooth. We treat it with disinfectant, because the mouth is not really that clean," Robey said, laughing.

Scientists then use a dental drill to pass the enamel and dentin -- "kind of like opening up a clam," said Robey -- to get to the pulp. "We take the pulp out, and we digest it with an enzyme to release the cells from the matrix of the pulp, and then we put the cells into culture and grow them."

According to Laning, even very small amounts of dental pulp are capable of producing many hundreds of millions of structural stem cells.

Harvesting dental stem cells is not a matter of waiting for the tooth to fall out and then quickly calling your dentist. When a baby tooth falls out, the viability of the pulp is limited if it's not preserved in the proper solution.

American Academy of Pediatric Dentistry President Dr. Jade Miller explained that "it's critical that the nerve tissue in that pulp tissue, the nerve supply and blood supply, still remain intact and alive." Typically, the best baby teeth to harvest are the upper front six or lower front six -- incisors and cuspids, he said.

For a child between 5 and 8 years of age, it's best to extract the tooth when there's about one-third of the root remaining, Miller said: "It really requires some planning, and so parents need to make this decision early on and be prepared and speak with their pediatric dentist about that."

Bassetto found the process easy. All it involved was a phone call to the company recommended by her dentist.

"They offer a service where they grow the cells and save those and also keep the pulp of the tooth without growing cells from it," she said. "I opted for both." From there, she said, the dentist shipped the extracted teeth overnight in a special package.

Bassetto said she paid less than $2,000 upfront, and now $10 a month for continued storage.

So is banking teeth something parents should be doing?

In a policy statement, the American Academy of Pediatric Dentistry "encourages dentists to follow future evidence-based literature in order to educate parents about the collection, storage, viability, and use of dental stem cells with respect to autologous regenerative therapies."

"Right now, I don't think it is a logical thing to do. That's my personal opinion," said Robey of the National Institute of Dental and Craniofacial Research. As of today, "we don't have methods for creating a viable tooth. I think they're coming down the pike, but it's not around the corner."

Science also does not yet support using dental pulp stem cells for other purposes.

"That's not to say that in the future, somebody could come up with a method that would make them very beneficial," Robey said.

Still, she observed, if science made it possible to grow natural teeth from stem cells and you were in a car accident, for example, and lost your two front teeth, you'd probably be "very happy to give up a third molar to use the cells in the molar to create new teeth." Third molars are fairly expendable, she said.

Plus, Robey explained, it may not be necessary to bank teeth: Another type of stem cell, known as induced pluripotent stem cells, can be programmed into almost any cell type.

"It's quite a different story than banking umbilical cord blood, which we do know contains stem cells that re-create blood," Robey said.

"So cord blood banking -- and now we have a national cord blood bank as opposed to private clinics -- so there's a real rationale for banking cord blood, whereas the rationale for banking baby teeth is far less clear," Robey said.

And there's no guarantee that your long-cryopreserved teeth or cells will be viable in the future. Banking teeth requires proper care and oversight on the part of cryopreservation companies, she said. "I think that that's a big question mark. If you wanted to get your baby teeth back, how would they handle that? How would they take the tooth out of storage and isolate viable cells?"

Provia's Laning, who has "successfully thawed cells that have been frozen for more than 30 years," dismissed such ideas.

"Cryopreservation technology is not the problem here," he said. "Stem cells from bone marrow and other sources have been frozen for future clinical use in transplants for more than 50 years. Similarly, cord blood has a track record of almost 40 years." The technology for long-term cryopreservation has been refined over the years without any substantial changes, he said.

Despite issues and doubts, Miller, of the pediatric dentistry academy, said parents still need to consider banking baby teeth.

A grandparent, he is making the decision for his own family.

"It's really at its infancy, much of this research," he said. "There's a very strong chance there's going to be utilization for these stem cells, and they could be life-saving."

He believes that saving baby teeth could benefit not only his grandchildren but also their older siblings and various other family members if their health goes awry and a stem cell treatment is needed.

"The science is strong enough to show it's not science fiction," Miller said. "There's going to be a significant application, and I want to give my grandkids the opportunity to have those options."

Aside from cost, Miller said there are other considerations: "Is this company going to be around in 30, 40 years?" he asked. "That's not an easy thing to figure out."

Having taken the leap, Bassetto doesn't worry.

"In terms of viability, you know, if something were to happen with the company, you could always get what's stored and move it elsewhere, so I felt I was protected that way," she said. She feels "pretty confident" with her decision and plans to store her grandchildren's baby teeth.

Still, she concedes that her circumstances may be rare.

"Not everybody's going to be touched by some kind of disease where it just hits home," Bassetto said. "For me, that made it a no-brainer."

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Cellectis to Present at the American Society of Gene & Cell Therapy … – Yahoo Finance

Posted: April 26, 2017 at 3:41 pm

NEW YORK--(BUSINESS WIRE)--

Regulatory News:

Cellectis (ALCLS.PA) (CLLS) (Alternext: ALCLS; Nasdaq: CLLS), a biopharmaceutical company focused on developing immunotherapies based on gene edited CAR T-cells, today announced that data on its gene-edited allogeneic off-the-shelf CAR T-cell immunotherapies (UCART) will be presented at the ASGCT 20th Annual Meeting. The meeting will be held from May 10th to 13th, 2017 in Washington, D.C., USA.

Oral presentation:

Development of Gene Edited Allogeneic CAR T-Cell Therapy Philippe Duchateau, PhD. Chief Executive Officer, Cellectis

Session: 300 - Clinical Advancement of Gene Editing-Moving New Science to the Clinic - Organized by the Clinical Trials and Regulatory Affairs Committee

Friday, May 12, 2017 from 8:35 AM to 9:10 AM EST Lincoln 2, 3, 4

Poster presentations:

176 - Genome-Wide Analysis of TALEN Activity in Primary Cells Brian Busser, Sonal Temburni, Aymeric Duclert, Philippe Duchateau and Laurent Poirot

Session: Gene Targeting and Gene Correction I Wednesday May 10, 2017 at 5:30 PM EST Exhibit Hall A & B South

114 - UCART22: An Allogeneic Adoptive Immunotherapy for Leukemia Targeting CD22 with CAR T-cells Anne-Sophie Gautron, Ccile Schiffer-Mannioui, Alan Marechal, Severine Thomas, Agnes Gouble, Laurent Poirot, Julianne Smith

Session: Cancer-Immunotherapy, Cancer Vaccines I Wednesday May 10, 2017 from 5:30 PM to 7:30pm EST Exhibit Hall A & B South

372 - Manufacturing of Gene-Modified Mouse CAR T-Cells Laurent Poirot, Brian Busser, Sonal Temburni, Philippe Duchateau

Session: Gene Targeting and Gene Correction II Thursday May 11, 2017 from 5:15 PM to 7:15 PM EST Exhibit Hall A & B South

About Cellectis

Cellectis is a biopharmaceutical company focused on developing immunotherapies based on gene-edited CAR T-cells (UCART). The companys mission is to develop a new generation of cancer therapies based on engineered T-cells. Cellectis capitalizes on its 17 years of expertise in genome engineering - based on its flagship TALEN products and meganucleases as well as its pioneering electroporation PulseAgile technology - to create a new generation of immunotherapies. CAR technologies are designed to target surface antigens expressed on cells. Using its life-science-focused, pioneering genome-engineering technologies, Cellectis goal is to create innovative products in multiple fields and with various target markets.

Cellectis is listed on the Nasdaq market (CLLS) and on the NYSE Alternext market (ALCLS.PA). To find out more about us, visit our website: http://www.cellectis.com

Talking about gene editing? We do it. TALEN is a registered trademark owned by the Cellectis Group

Disclaimer

This press release and the information contained herein do not constitute an offer to sell or subscribe, or a solicitation of an offer to buy or subscribe, for shares in Cellectis in any country. This press release contains forward-looking statements that relate to the Companys objectives based on the current expectations and assumptions of the Companys management only and involve risk and uncertainties that could cause the Company to fail to achieve the objectives expressed by the forward-looking statements above.

View source version on businesswire.com: http://www.businesswire.com/news/home/20170425006830/en/

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SAVSU and TrakCel announce collaboration to integrate Cell … – PR Newswire (press release)

Posted: April 26, 2017 at 3:41 pm

TrakCel's Cellular Orchestration platform facilitates efficiency and compliance to quality standards in cell therapy manufacturing by enabling program-critical information to be collated, tracked and and documented. Detailed processes and managed workflows for every participant in the value chain enable safe and efficient scaling outward and upward as production increases. Conditional logic workflows for all participants including clinicians, logistics and manufacturing partners ensure standardization andcompliance with validated procedures.

Bruce McCormick, President of SAVSU Technologies, remarked, "Our optimized technologies represent the next generation of integrated, cloud-connected cold chain technologies. We are very pleased to collaborate with TrakCel and look forward to integrating our respective data platforms to provide even more value to our mutual customers. We are committed to driving a paradigm shift away from traditional, risk-laden cold chain practices based on the use of poor performing shipping containers and disconnected data systems."

Ravi Nalliah, CEO at TrakCel, commented, "We scoped the packaging and cold chain technologies space to identify best of breed ecosystem partners. SAVSU is so far ahead of traditional container and data logger suppliers, and we are very keen to integrate their innovations into our Cell Orchestration Platform. The timing of this collaboration is perfect as the cell therapy market continues to mature and awareness is peaked on the need to use better tools to enable clinical trial success, commercial scale-out, and reimbursement supported by evidence of compliance throughout manufacturing, distribution and patient administration."

Chain Link Research and Pharmaceutical Commerce market research estimate that there are 80 million to 130 million annual temperature sensitive pharmaceutical shipments requiring cold chain management, resulting in $12 billion spent annually on cold chain logistics, with $9 billion for transportation and $3 billion for specialized tertiary packaging and instrumentation such as insulated boxes, blankets, phase change materials, temperature sensors and data loggers. The use of currently available cold technologies results in $15 billion to $35 billion spent annually replacing products lost due to temperature excursions.

About SAVSU Technologies SAVSU is a leading designer and manufacturer of innovative, high performance, cloud-connected passive storage and transport containers for temperature-sensitive biologics and pharmaceuticals. Our mission is to improve global health by greatly reducing the waste and risks associated with the improper freezing and overheating of thermal-sensitive medicines and biologics. SAVSU has developed proprietary state-of-the-art technology to ultimately lower costs and improve delivery of these most essential materials.

For more information please visit http://www.savsu.com.

About TrakCel TrakCel's cell, gene and immunotherapy management solution improves clinical study efficacy and accelerates global scale-up and scale-out by implementing communications technology to integrate the delivery path from needle-to-needle. The technology provides interactive instructions to professionals across the supply chain and gives stakeholders on-demand visibility of procedural results and chain-of-custody data for immediate traceability, validation and compliance audits.

TrakCel technology is increasingly being adopted by leaders in the cell, gene and immunotherapy industry. TrakCel is headquartered in Cardiff, Wales with a U.S. offices in Newport Beach, California and Bridgewater, NJ.

For more information please visitwww.trakcel.com.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/savsu-and-trakcel-announce-collaboration-to-integrate-cell-therapy-manufacturing-and-cold--chain-data-management-platforms-300444497.html

SOURCE SAVSU Technologies

http://www.savsu.com

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Regenerative cell therapy, tissue engineering are future treatments – ModernMedicine

Posted: April 26, 2017 at 3:41 pm

Regenerative cell therapy, tissue engineering are future treatments
ModernMedicine
There have been major changes in the way keratoplasty has been performed over the past 15 years. Data from the Singapore Corneal Transplant Study shows there have been improved outcomes in the lamellar surgical techniques compared to the full ...

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TxCell to Provide CAR-Treg Update in Upcoming Cell Therapy Conferences – Business Wire (press release)

Posted: April 26, 2017 at 3:41 pm

VALBONNE, France--(BUSINESS WIRE)--Regulatory News:

TxCell SA (Paris:TXCL) (FR0010127662 TXCL), a biotechnology company developing innovative, personalized cellular immunotherapies using regulatory T cells (Treg) to treat severe inflammatory and autoimmune diseases as well as transplant rejection, today announces that Stphane Boissel, CEO of TxCell, will present in upcoming cell therapy conferences.

Mr. Boissel will present an update on TxCells ENTrIA CAR-Treg platform at the 5th Alliance for Regenerative Medicine (ARM) Annual Cell & Gene Therapy Investor Day which will be held on April 27, 2017 in Boston (US). The TxCell presentation will be coupled with an interview by Joshua Schimmer, MD, Managing Director and Senior Biotech Equity Research Analyst at Piper Jaffray & Co. The presentation and interview will take place at 5:00pm local time (Harborside Room).

Mr. Boissel will also be participating to a panel discussion entitled Biotech Perspective: The opportunities of cell & gene therapy and why now? at the Investment for Advanced Therapies Summit which will be held on May 3, 2017 in London (UK). This panel discussion will take place at 9:15am local time.

In addition, Mr. Boissel and other members from TxCell will attend the International Society for Cellular Therapy (ISCT) 2017 Annual Meeting which will be held on May 3-6, 2017, in London.

About TxCell http://www.txcell.com

TxCell is a biotechnology company that develops platforms for innovative, personalized T cell immunotherapies for the treatment of severe inflammatory and autoimmune diseases with high unmet medical need. TxCell is targeting a range of autoimmune diseases (both T-cell and B-cell-mediated) including Crohns disease, lupus nephritis, bullous pemphigoid and multiple sclerosis, as well as transplant rejection.

TxCell is the only clinical-stage cellular therapy company fully dedicated to the science of regulatory T lymphocytes (Tregs). Tregs are a recently discovered T cell population for which anti-inflammatory properties have been demonstrated. Contrary to conventional approaches based on non-specific polyclonal Tregs, TxCell is exclusively developing antigen-specific Tregs. This antigen specificity may either come from genetic modifications with Chimeric Antigen Receptor (CAR) or from pre-existing Treg cell T-Cell Receptor (TCR). TxCell is developing two proprietary technology platforms, ENTrIA, which is composed of genetically-engineered Tregs, and ASTrIA, which is composed of non-modified naturally antigen-specific Tregs.

Based in Sophia-Antipolis, France, TxCell is listed on Euronext Paris and currently has 46 employees.

Next events

Financial and business conferences

5th Annual Cell & Gene Therapy Investor Day (ARM)

Scientific and medical conferences

22e congrs Nantes Actualits Transplantation (NAT)

Forward-Looking Statements - TxCell

This press release contains certain forward-looking statements relating to the business of TxCell, which shall not be considered per se as historical facts, including TxCells ability to develop, market, commercialize and achieve market acceptance for specific products, estimates for future performance and estimates regarding anticipated operating losses, future revenues, capital requirements, needs for additional financing. In addition, even if the actual results or development of TxCell are consistent with the forward-looking statements contained in this press release, those results or developments of TxCell may not be indicative of their in the future.

In some cases, you can identify forward-looking statements by words such as "could," "should," "may," "expects," "anticipates," "believes," "intends," "estimates," "aims," "targets," or similar words. Although the management of TxCell believes that these forward-looking statements are reasonably made, they are based largely on the current expectations of TxCell as of the date of this press release and are subject to a number of known and unknown risks and uncertainties and other factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievement expressed or implied by these forward-looking statements. In particular, the expectations of TxCell could be affected by, among other things, uncertainties involved in the development of the Companys products, which may not succeed, or in the delivery of TxCells products marketing authorizations by the relevant regulatory authorities and, in general, any factor that could affects TxCell capacity to commercialize the products it develops, as well as, any other risk and uncertainties developed or identified in any public documents filed by TxCell with the AMF, included those listed in chapter 4 Risk factors of the 2015 document de rfrence (registration document) approved by the AMF on May 24, 2016 under number R.16-048 and in the section 5.1 of the update of the registration document filed with the AMF on January 24, 2107 under number D.16-0346-A01. In light of these risks and uncertainties, there can be no assurance that the forward-looking statements made in this press release will in fact be realized. Notwithstanding the compliance with article 223-1 of the General Regulation of the AMF (the information disclosed must be accurate, precise and fairly presented), TxCell is providing the information in these materials as of this press release, and disclaims any intention or obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise.

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TxCell to Provide CAR-Treg Update in Upcoming Cell Therapy Conferences - Business Wire (press release)

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Global Stem Cell Therapy Market – Analysis, Technologies and Forecasts to 2021 – Increasing Demand to Develop … – PR Newswire (press release)

Posted: April 26, 2017 at 3:41 pm

The global stem cell therapy market to grow at a CAGR of 36.52% during the period 2017-2021.

The report, Global Stem Cell Therapy Market 2017-2021, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the market landscape and its growth prospects over the coming years. The report also includes a discussion of the key vendors operating in this market.

The latest trend gaining momentum in the market is evolution of new destinations in the development of stem cell therapies. Traditionally, the US and European countries are the key destinations for clinical trials for stem cell therapy products. However, the transformation of regulatory landscape in countries such as Japan and South Korea has made these regions as attractive destinations for the development of stem cell therapy products.

According to the report, one of the major drivers for this market is increase in federal funding in stem cell therapy. Research and clinical trials of stem cell therapy require huge investment, which many research institutes and small companies cannot afford. Therefore, many federal organizations provide funding to these institutes and small companies to help their innovative ideas in the development of stem cell therapies. Worldwide, many government organizations have noticed the importance of regenerative medicine, and thus they have allocated funds and grants in that area. For instance, in the US, the NIH and CIRM provide most of the funds.

Key vendors

Key Topics Covered:

Part 01: Executive summary

Part 02: Scope of the report

Part 03: Research Methodology

Part 04: Introduction

Part 05: Understanding of stem cell therapy

Part 06: Ethical issues and regulatory landscape

Part 07: Key clinical trials

Part 08: Market landscape

Part 09: Market segmentation by therapy

Part 10: Market segmentation by applications

Part 11: Geographical segmentation

Part 12: Market drivers

Part 13: Impact of drivers

Part 14: Market challenges

Part 15: Impact of drivers and challenges

Part 16: Market trends

Part 17: Vendor landscape

Part 18: Key vendor analysis

For more information about this report visit http://www.researchandmarkets.com/research/gdv8s6/global_stem_cell

Media Contact:

Laura Wood, Senior Manager press@researchandmarkets.com

For E.S.T Office Hours Call 1-917-300-0470 For U.S./CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/global-stem-cell-therapy-market---analysis-technologies-and-forecasts-to-2021---increasing-demand-to-develop-effective-drugs-for-cardiology-and-degenerative-disorders---research-and-markets-300444066.html

SOURCE Research and Markets

http://www.researchandmarkets.com

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Global Stem Cell Therapy Market - Analysis, Technologies and Forecasts to 2021 - Increasing Demand to Develop ... - PR Newswire (press release)

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Lilly Assures Market As Diabetes Stumbles – Seeking Alpha

Posted: April 26, 2017 at 3:41 pm

As the diabetes wars have raged, Eli Lilly's (NYSE:LLY) balanced portfolio has helped insulate it from the share price losses dealt to competitor Novo Nordisk (OTCPK:NONOF). But even Lilly can have an off quarter.

Trulicity's growth was more than offset by below-consensus sales for Basaglar and Jardiance - the underperformance of the last of these was surprising given that it has begun marketing on its cardiovascular benefit. When combined with last week's US rejection of rheumatoid arthritis entry Olumiant, these disappointments forced Lilly executives to reassure investors that the company's medium-term guidance of 5% growth could still be met.

Lilly shares fell 3% today following the release of first-quarter results, removing $2.7bn in market valuation. Since the Olumiant complete response letter was announced, shares have fallen 6% (Olumiant setback opens the door to rivals, April 18, 2017).

Bernstein analyst Tim Anderson chalked up some of the losses to profit-taking - before the Olumiant news, shares were up 16% on the year - but acknowledged the effect of Olumiant and diabetes revenue on investor sentiment.

Topline good

In the long view, the Indiana-based group had a positive quarter, with revenue of $5.2bn and earnings per share of $0.98 in line with investor expectations. However, having just got over the news of Olumiant - rejected because of questions over the most efficacious dose and safety characterization - news that diabetes was not necessarily firing on all cylinders was not taken well.

Expectations for Jardiance remain high since it is the first of the SGLT2 class to be allowed to market on the basis of averting cardiovascular death in diabetics, although it could very well be chased by competitors Invokana, from Johnson & Johnson, and Farxiga, from Astrazeneca (NYSE:AZN) (ACC - Jardiance heart benefit looks like a class effect, March 20, 2017).

Lilly diabetes chief Enrique Conterno characterized the SGLT2 class as having flat growth before the cardiovascular data was included on the Jardiance label, but said that since marketing on that claim has begun new patient starts have risen 70% - the miss on Jardiance sales may simply reflect a mismatch between investor expectations and market realities.

Olumiant's bigger setback has investors questioning the 5% revenue growth forecast given that the drug is one of four new products that will post annual sales growth of $1bn or more to counteract losses due to patent expiries. Finance chief Derica Rice offered some comfort: "We've factored in that we won't have 100% success on every molecule" when preparing that forecast, he said.

Olumiant's failure to add US revenue does, however, increase the pressure on the rest of the portfolio to meet sales expectations. And Lilly's exposure to the volatile diabetes space raises the risk that that will not happen.

Editor's Note: This article discusses one or more securities that do not trade on a major U.S. exchange. Please be aware of the risks associated with these stocks.

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Lilly Assures Market As Diabetes Stumbles - Seeking Alpha

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