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Archive for the ‘Regenerative Medicine’ Category

Leading Researchers to Unite at Texas State Capitol for Regenerative Medicine and Stem Cell Research

Saturday, October 13th, 2012

AUSTIN, Texas, Oct. 12th, 2012 /PRNewswire-USNewswire/ -- Prominent stem cell scientists, physicians, and advocates from leading medical facilities and research institutions across Texas and California will highlight the 3rd Annual Stem Cell Research Symposium: Spotlight on Texas, on October 19, 2012, at the Texas State Capitol.

This free, public symposium, produced and co-hosted by the Austin-based nonprofit Texas Cures Education Foundation (Texas Cures), is designed to educate the public about the exciting stem cell research andclinical trials currently under way in Texas.The event will also include a discussion of recent Texas laws affecting stem cell research, the potential economic impact of stem cell research and highlight the current progress in one of the most promising areas of medicine.

This year, more than a dozen local and national advocacy groups, institutions and foundations showed their support for the efforts of the hosting organizations Texas Cures and Texans for Stem Cell Research including the Genetics Policy Institute, Alliance for Regenerative Medicine and Texans for Advancement of Medical Research.

The symposium begins at 8:30 a.m. in the Capitol Extension Auditorium (E1.004), located at the Texas State Capitol Building. Admission is free and open to the public.Registration is recommended.

This program unites the diverse stem cell research and regenerative medicine community to provide a unified voice for promising science that holds unmatched potential to benefit patients. Leading speakers at the event will include:

For additional details about the program and presentation topics, please visit TexasCures.org.

The 3rd Annual Stem Cell Research Symposium: Spotlight on Texas is an official World Stem Cell Awareness Day Event. Follow @TexasCures and #stemcellday for live Twitter updates and announcements.

Texas Cures Education Foundation (Texas Cures) TexasCures.orgis a non-partisan, nonprofit 501(c)3] organization based in Austin, Texas. It was founded for the purpose of advancing knowledge of the life-saving work that doctors and researchers perform every day on behalf of patients and their families. Texas Cures facilitates stem cell public education for the betterment of healthcare and the growth of companies, research hospitals, and institutions, charities, and volunteer patient group organizations that include a broad range of regenerative medicine stakeholders. Texas Cures advocates for responsible public policy and encourages legislative and regulatory proposals that expand access to stem cell clinical applications.

SOURCE Texas Cures Education Foundation

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Scientists Identify Mammal Model of Bladder Regeneration

Saturday, October 13th, 2012

Newswise WINSTON-SALEM, N.C. While it is well known that starfish, zebrafish and salamanders can re-grow damaged limbs, scientists understand very little about the regenerative capabilities of mammals. Now, researchers at Wake Forest Baptist Medical Centers Institute for Regenerative Medicine report on the regenerative process that enables rats to re-grow their bladders within eight weeks.

In PLOS ONE, a peer-reviewed, online publication, the scientists characterize this unique model of bladder regeneration with the goal of applying what they learn to human patients.

A better understanding of the regenerative process at the molecular and cellular level is a key to more rapid progress in applying regenerative medicine to help patients, said George Christ, Ph.D., senior researcher and professor of regenerative medicine at Wake Forest Baptist.

In a previous study by Christs team, research in rats showed that when about 75 percent of the animals bladders were removed, they were able to regenerate a complete functional bladder within eight weeks. The current study focused on how the regeneration occurs.

There is very little data on the mechanisms involved in organ regeneration in mammals, said Christ. To our knowledge, bladder regeneration holds a unique position there is no other mammalian organ capable of this type of regeneration.

The ability of the liver to grow in size when lobes are removed is sometimes referred to as regeneration, but this is a misnomer, said co-author Bryon Petersen, Ph.D., who was a professor of regenerative medicine at Wake Forest Baptist during the period the research occurred. Instead, through a proliferation of cells, the remaining tissue grows to compensate for the lost size. In contrast, the hallmark of true regeneration is following natures pattern to exactly duplicate size, form and function, Petersen said.

If we can understand the bladders regenerative process, the hope is that we can prompt the regeneration of other organs and tissues where structure is important from the intestine and spinal cord to the heart, said Petersen.

The current study showed that the animals bodies responded to injury by increasing the rate at which certain cells divided and grew. The most notable proliferative response occurred initially in the urothelium, the layer of tissue that lines the bladder.

As the proliferative activity in the bladder lining waned, it continued elsewhere: in the fibrous band (lamina propria) that separates the bladder lining from the bladder muscles and in the bladder muscle itself.

The researchers have several theories about how the process works, said Christ. One possibility is that cells in the bladder lining transition and become a type of stem cell that can proliferate throughout the bladder. Other theories are that cells in the bladder lining signal other cells to replicate and that injury prompts stem cells to arrive through the blood stream to repair the bladder damage.

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Scientists Identify Mammal Model of Bladder Regeneration

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Stem Cell Scientists Awarded Nobel Prize in Physiology and Medicine

Tuesday, October 9th, 2012

Kyodo / Reuters

Kyoto University Professor Shinya Yamanaka (left) and John Gurdon of the Gurdon Institute in Cambridge, England, at a symposium on induced pluripotent stem cells in Tokyo in April 2008

In a testament to the revolutionary potential of the field of regenerative medicine, in which scientists are able to create and replace any cells that are at fault in disease, the Nobel Prize committee on Monday awarded the 2012 Nobel in Physiology or Medicine to two researchers whose discoveries have made such cellular alchemy possible.

The prize went to John B. Gurdon of the University of Cambridge in England, who was among the first to clone an animal, a frog, in 1962, and to Shinya Yamanaka of Kyoto University in Japan who in 2006 discovered the four genes necessary to reprogram an adult cell back to an embryonic state.

Sir John Gurdon, who is now a professor at an institute that bears his name, earned the ridicule of many colleagues back in the 1960s when he set out on a series of experiments to show that the development of cells could be reversed. At the time, biologists knew that all cells in an embryo had the potential to become any cell in the body, but they believed that once a developmental path was set for each cell toward becoming part of the brain, or a nerve or muscle it could not be returned to its embryonic state. The thinking was that as a cell developed, it would either shed or silence the genes it no longer used, so that it would be impossible for a cell from an adult animal, for example, to return to its embryonic state and make other cells.

(MORE: Stem Cell Miracle? New Therapies May Cure Chronic Conditions Like Alzheimers)

Working with frogs, Gurdon proved his critics wrong, showing that some reprogramming could occur. Gurdon took the DNA from a mature frogs gut cell and inserted it into an egg cell. The resulting egg, when fertilized, developed into a normal tadpole, a strong indication that the genes of the gut cell were amenable to reprogramming; they had the ability to function as more than just an intestinal cell, and could give rise to any of the cells needed to create an entirely new frog.

Just as Gurdon was facing his critics in England, a young boy was born in Osaka, Japan, who would eventually take Gurdons finding to unthinkable extremes. Initially, Shinya Yamanaka would follow his fathers wishes and become an orthopedic surgeon, but he found himself ill-suited to the surgeons life. Intrigued more by the behind-the-scenes biological processes that make the body work, he found himself drawn to basic research, and began his career by trying to find a way to lower cholesterol production. That work also wasnt successful, but it drew him to the challenge of understanding what makes cells divide, proliferate and develop in specific ways.

In 2006, while at Kyoto University, Yamanaka stunned scientists by announcing he had successfully achieved what Gurdon had with the frog cells, but without using eggs at all. Yamanaka mixed four genes in with skin cells from adult mice and turned those cells back to an embryo-like state, essentially erasing their development and turning back their clock. The four genes reactivated other genes that are prolific in the early embryo, and turned off those that directed the cells to behave like skin.

(MORE: Ovary Stem Cells Can Produce New Human Eggs)

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Stem Cell Scientists Awarded Nobel Prize in Physiology and Medicine

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ACT Announces Approval to Treat Additional Stargardt’s Disease Patients with Higher RPE Dosage in Both U.S. and …

Tuesday, October 9th, 2012

MARLBOROUGH, Mass.--(BUSINESS WIRE)--

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that the Data and Safety Monitoring Board (DSMB), an independent group of medical experts closely monitoring the Companys three ongoing clinical trials, has authorized the Company to move forward with enrollment and treatment of second and third additional patients with Stargardts macular dystrophy (SMD) in the second patient cohort of its U.S. trial for the condition. Additionally, the DSMB has authorized the Company to treat all three patients in the second cohort of its European trial for SMD.

The UK Medicines and Healthcare products Regulatory Agency (MHRA) recently approved a protocol modification to the DSMB review, streamlining the process, allowing the company to treat the first patient in a new cohort if the DSMB has allowed this in the US study, and once clearance has been received in the US trial to treat the next two patients in the US cohort. This would also allow for treatment of the UK patients without an additional review by the DSMB. Moreover, according to the protocol for both trials, each patient in the second cohort will be injected with 100,000 human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells, up from 50,000 in the first cohort.

This authorization to treat the next five patients in the second, higher-dosage cohort in both our clinical trials for SMD represents a significant step forward for our clinical programs, commented Gary Rabin, chairman and CEO of ACT. We are also encouraged with the MHRAs approval of the DSMBs streamlined review process. Clearly this has the potential to help accelerate the pace of our European trial.

ACT is conducting three clinical trials in the U.S. and Europe using hESC-derived RPE cells to treat forms of macular degeneration, SMD and dry age-related macular degeneration (dry AMD). Each trial will enroll a total of 12 patients, with cohorts of three patients each in an ascending dosage format, from 50,000 hESC-derived RPE cells in the first patient cohort to 200,000 in the last and final cohort. These trials are prospective, open-label studies, designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with dry-AMD or SMD at 12 months, the studys primary endpoint.

We are eagerly anticipating treating these final two patients in the second cohort of our U.S. trial for SMD, and all three patients in the second cohort of our E.U. trial, commented Robert Lanza, M.D., ACTs chief scientific officer. We are encouraged by the preliminary data in the first patient in this second, higher-dosage cohort and look forward to gathering more data.

Further information about patient eligibility for ACTs SMD studies in the U.S. and E.U. as well as its dry AMD study are available atwww.clinicaltrials.gov,with the following Identifiers: NCT01345006 (U.S. SMD), NCT01469832 (E.U. SMD), and NCT01344993 (dry AMD).

About Advanced Cell Technology, Inc.

Advanced Cell Technology, Inc., is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.

Forward-Looking Statements

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Regenerative medicine could be 'next big thing' for Va. biotech

Tuesday, October 9th, 2012

Regenerative medicine, a field that didn't exist 20 years ago and contains techniques seemingly straight out of science fiction, could be the next big thing in Virginia's biotechnology sector.

That's the opinion of Roy Ogle, an expert in the field who works at Old Dominion University as head of its new school of Medical Diagnostic and Translational Sciences.

So what is regenerative medicine? Simply put, it's the process of re-growing human cells to repair damaged tissues and organs.

In a meeting Thursday hosted by the Virginia Biotechnology Association, Ogle and Brian Pollok, principal of Rapidan BioAdvisors, discussed one of the field's newest developments: induced pluripotent stem cells, or iPSCs.

Let's go back to high school biology: Perhaps you remember embryonic stem cells. These cells can differentiate into different types of cells skin, blood, bone, muscle before a baby is born. But their use in scientific research has become controversial and difficult.

So scientists needed a new way to develop stem cells. iPSCs are already formatted cells that are "induced," or returned, to their original state as a stem cell. Then that stem cell can be reprogrammed to become a different type of cell. For example, a researcher can take a red blood cell, turn it into an iPSC, and then turn that into a muscle cell. (Yeah, our jaw dropped at this point, too). So you get most of the benefits of an embryonic stem cell without the controversy.

What's that mean for the business community?

"Ten or 20 years from now, we could have a way to do cell replacements and make a new spinal cord or new and healthy muscles," Ogle said. "But right now, there are genetic discoveries and methods of development with a giant potential that a small company can sell to (pharmaceutical giants such as) Roche or Sanofi-Aventis."

Ogle said this sort of intermediate work after invention but before the science is proven enough for big pharma to get involved is the perfect space for startups, especially those affiliated with research universities. He said small companies are best placed to do this work and sell the results to big companies because a startup is better suited to tolerate the risk and uncertainty.

"While we think about the long-term development as scientists, there are applications right now where we could serve society and make a lot of money," he said.

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K computer may be used in regenerative medicine

Saturday, October 6th, 2012

The Yomiuri Shimbun/Asia News Network Friday, Oct 05, 2012

The K supercomputer, which once held the world's fastest computing speed, may be used to shorten the time needed in regenerative medicine from several months, or even years, to several hours, according to the Riken Center of Developmental Biology and other institutions.

Researchers aim to create organs from human embryonic stem cells (ES cells) or induced pluripotent stem cells (iPS cells), but the length of time normally needed to accomplish this task is a problem.

The institutions hope to put regenerative medicine into practical use as soon as possible using iPS cells, a Japanese technology, and other cells, and this is where the supercomputer will come in.

Yoshiki Sasai, group director at the Riken Center, and other researchers are planning to use the K supercomputer to determine the best method to create organs from these cells.

The researchers successfully developed an optic cup, a basic part of the eye, from ES cells for the first time in the world. While it takes about six months to transform ES cells into an optic cup, the researchers spent about three years to find how to do this through trial and error.

Currently, it takes several years to complete basic experiments to transform ES cells or iPS cells into target organs, and in many cases the experiments fail to achieve their purpose.

Plans are under way to use the K supercomputer to develop new medicines, work out disaster prevention measures and conduct research on cosmic evolution and for other purposes.

Sasai and the other researchers, therefore, decided the supercomputer, which performs 10 quadrillion (or one kei in Japanese) calculations per second, would be ideal in completing basic experiments in a fraction of the time it now takes.

If the K supercomputer calculates mathematized data on divisions, growth and internal changes of iPS cells to which protein or certain kinds of genes are added, it will become possible to create target organs more effectively, according to the researchers.

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K computer may be used in regenerative medicine

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Histogenics Honored as a 2012 “Fierce 15” Company by FierceMedicalDevices

Wednesday, October 3rd, 2012

WALTHAM, Mass.--(BUSINESS WIRE)--

Histogenics, a regenerative medicine company combining cell therapy and tissue engineering technologies to develop highly innovative products for tissue repair and regeneration, announced today that it has been named to the FierceMedicalDevices Fierce 15 list, designating it as one of the leading medical device and diagnostic companies of 2012. FierceMedicalDevicesEditors Mark Hollmer and Damian Garde, in conjunction with Editor-in-Chief John Carroll and Executive Editor Ryan McBride, chose this years winners based on their top management teams, notable financial backing, and promising technologies and market opportunities.

We have worked hard over the past year, securing $49 million in financing and adding key new staff, investors and board members, so that we are now in the position to focus our full attention on continued successful clinical and regulatory execution for NeoCart cartilage regeneration implant, which is currently enrolling patients into the Phase 3 IND clinical study, and the EU regulatory development of our VeriCart cartilage repair scaffold, said Patrick ODonnell, President and Chief Executive Officer of Histogenics. We believe our product candidates have the potential to transform the treatment of cartilage injury with the goal of returning some of the estimated 1.8 million patients each year in the U.S. and E.U. that undergo arthroscopy for knee cartilage defects to their pre-injury level of activity.

Nailing down $49 million in financing in July reinforces the notion that this regenerative medicine company stands out for doing things differently.One example how: The company is well underway enrolling patients in a Phase 3 trial for NeoCart, a cartilage implant that uses a patients own cells to build it before treating cartilage lesions in the knee, said Hollmer.

NeoCart is an autologous neocartilage tissue implant in an ongoing Phase 3 clinical program that utilizes the patients own cells to regenerate cartilage in patients suffering from cartilage lesions in the knee.VeriCart, is a single-step, cell-free collagen scaffold uniquely designed to be used in conjunction with the patients own stem cells to repair small cartilage defects frequently observed in meniscal and anterior cruciate ligament repair procedures. Histogenics is seeking regulatory clearance in the European Union for VeriCart.

An internationally recognized e-newsletter reaching more than 34,000 medical device and diagnostic industry professionals, FierceMedicalDevices provides subscribers with a quick authoritative briefing on the days top stories, with a special focus on clinical studies, FDA/EMEA regulations and post-marketing. Sign up is free at http://www.fiercemedicaldevices.com/signup.

About FierceMarkets

FierceMarkets, a wholly owned subsidiary of Questex Media Group, is a leader in B2B emedia, providing information and marketing services in the telecommunications, life sciences, healthcare, IT, energy, government and finance industries through its portfolio of email newsletters, websites, webinars and live events. Every business day, FierceMarkets wide array of publications reaches more than 1.3 million executives in more than 100 countries.

About Histogenics

Histogenics is a leading regenerative medicine company that combines cell therapy and tissue engineering technologies to develop highly innovative products for tissue repair and regeneration. In May of 2011, Histogenics acquired Israeli cell-therapy company ProChon BioTech. Histogenics flagship products focus on the treatment of active patients suffering from articular cartilage derived pain and immobility. The Company takes an interdisciplinary approach to engineering neocartilage that looks, acts and lasts like hyaline cartilage. It is developing new treatments for sports injuries and other orthopedic conditions, where demand is growing for long-term alternatives to joint replacement. Histogenics has successfully completed Phase 1 and Phase 2 clinical trials in which the NeoCart autologous tissue implants effectiveness is compared to that of standard microfracture surgery. Based in Waltham, Massachusetts, the company is privately held. For more information, visitwww.histogenics.com.

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Histogenics Honored as a 2012 “Fierce 15” Company by FierceMedicalDevices

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Translational Regenerative Medicine: Market Prospects 2012-2022

Tuesday, October 2nd, 2012

NEW YORK, Oct. 1, 2012 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:

Translational Regenerative Medicine: Market Prospects 2012-2022

http://www.reportlinker.com/p0595030/Translational-Regenerative-Medicine-Market-Prospects-2012-2022.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Blood_Supply,_Tissue_Banking_and_Transplantation

Report Details

New study shows you commercial potential of regenerative treatments

See what the future holds for translational regenerative medicine. Visiongain's updated report lets you assess forecasted sales at overall world market, submarket, product and regional level to 2022.

There you investigate the most lucrative areas in that research field, industry and market. Discover prospects for tissue-engineered products, stem cell treatments and gene therapy.

We pack our study with information and analysis to help your work and save you time:

Access to present and predicted trends, with commercial opportunities and prospects revealed

Data and discussions - including our revenue forecasts to 2022 - for your research, analyses and decision making

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Translational Regenerative Medicine: Market Prospects 2012-2022

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Anticipated short-term cell therapy industry clinical milestones

Sunday, September 30th, 2012
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What follows is an interesting but not exhaustive list of cell therapy industry clinical milestones we anticipate in the next 3-9 months as selected from the list of cell therapy products we are tracking in late-stage or post-commercial development.  


There are other commercial milestones we are monitoring as well as other clinical milestones we expect to see related to cell therapy products in earlier stages of the development pipeline that are not included below.


CellCoTec (http://www.cellcotec.com)
  • Having completed a trial in Europe of their device to enable POC production of an autologous chondrocyte cellular product in/with a biodegradable, load-bearing scaffold for the treatment of articular cartilage defects, they have now submitted their CE market application.  The CE mark application is under review and they anticipate a response in October.  
  • This device and the potential emergence of Sanofi's MACI in the European market next year may have an impact on Tigenix (EBR:TIG) most directly.



ERYtech Parma (http://www.erytech.com)

  • Their 'pivotal' phase 2/3 trial in Europe of lead product, GRASPA, for the treatment of Acute Lymphoblastic Leukemia (ALL) is scheduled for completion 2H 2012. 


GamidaCell (http://www.gamidacell.com)

  • Their 'pivotal' phase 2/3 trial in the US, Israel, and Europe of lead product, StemEx, for the treatment of leukemia and lymphoma, in joint development with Teva, completed enrollment in February and is scheduled for completion 2H 2012.  They have not been shy about the fact they expect to be in the market in 2013.


Innovacell (http://www.innovacell.com)

  • They raised over 8m Euro in April for a phase 3 trial in Europe for their lead product, ICES13, for the treatment of stress-urinary incontinence which was scheduled for a preliminary clinical data readout in Q4 2012 and be ready for market authorization in 2013. Since announcing the capital raise the company has been stone silent and no clinical trial registry has been filed.  Status unknown.


Miltenyi Biotec (www.miltenyibiotec.com)

  • Their phase 3 trial in Germany of CD133+ cells as an adjunct to CABG surgery for myocardial ischemia or coronary artery disease is scheduled for completion in January.


NovaRx (http://www.novarx.com)

  • Their phase 3 trial in US, Europe, and India of their lead product, Lucanix, for the treatment of advanced Non-small Cell Lung Cancer (NSCLC) following front-line chemotherapy is scheduled in clnicaltrials.gov for completion in October but we have learned they expect their next 'interim analysis' in February.


NuVasive (http://www.nuvasive.com)

  • They have a series of trials scheduled to complete 2H 2012 intended to provide additional clinical data to support its marketing of Osteocel Plus for the treatment of a growing number of orthopedic applications.


Sanofi's Genzyme (http://www.genzyme.com)

  • Having completed their phase 3 trial in Europe of MACI for knee repair (symptomatic articular cartilage defects of the femoral condyle including the trochlea), they expect to file their market authorization application (MAA) in 1H 2013.


Hope that's helpful and gives you a sense some of the late-stage things to watch for in the coming weeks and months.  



--Lee

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

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The cost of clinical trial data bias/loss, FDA’s new job and the need for bold leadership.

Sunday, September 30th, 2012
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The scandal of clinical trial data loss is eroding the fundamentals of evidence-based research and clinical medicine.


Before you right this post off as the stuff of conspiracy theories, fear-mongering, and 'alternative world views' consider that this view is shared by the likes of the FDA, the International Committee of Medical Journal Editors, the Cochrane Collaboration, and researchers at institutions like Johns Hopkins School of Medicine.


Here's the underlying premise as succinctly described by author Ben Goldacre:

"Drugs are tested by the people who manufacture them, in poorly designed trials, on hopelessly small numbers of weird, unrepresentative patients, and analysed using techniques that are flawed by design, in such a way that they exaggerate the benefits of treatments. Unsurprisingly, these trials tend to produce results that favour the manufacturer.

When trials throw up results that companies don't like, they are perfectly entitled to hide them from doctors and patients, so we only ever see a distorted picture of any drug's true effects. Regulators see most of the trial data, but only from early on in a drug's life, and even then they don't give this data to doctors or patients, or even to other parts of government. This distorted evidence is then communicated and applied in a distorted fashion."

Authors M. Todwin and J. Abramson summarize it thusly:

"Trials with positive results generally are published more frequently than studies that conclude that a new drug poses greater risks or is no more effective than standard therapy or a placebo. Furthermore, some articles may distort trial findings by omitting important data or by modifying prespecified outcome measures. Lack of access to detailed information about clinical trials can undermine the integrity of medical knowledge."

Here is a great list of very recent resources that may convince you of the merits of this concern:

Yesterday, the US Secretary of Health and Human Services announced (in an FR notice) that the FDA was now charged with ensuring all organizations comply with the heretofore enacted but relatively unenforced  requirement to submit all relevant clinical trial data to http://www.clinicaltrials.gov

For further commentary on this move see the following reports from:
What is abundantly clear to me is that the FDA is left almost powerless - and if not powerless than certainly without sufficient resources - to successfully enforce its new power.  This requires collective industry leadership.  Bold, industry-initiated standards, infrastructure and old-fashioned peer pressure.

Here's what I wish.  

I wish that as a cell therapy industry we - through organizations like ISSCR, ARM, ISCT, etc and leading publishers of some of our leading journals like Regenerative Medicine, Cytotherapy, Cell Stem Cell, Stem Cells, etc - would take a leadership position on an issue like this.

I believe that as a relatively small and nascent sector of the biopharma industry we are more likely capable of collaborating on something important like this than larger, more established [entrenched] and diverse sectors.  Of course it requires the political will and cajones.

The payoff from our sector in taking a leadership role on this issue could potentially be enormous in terms of providing our sector with truly transparent and useful data.  Perhaps even more important would be the public profile such leadership would provide the sector.  Such a move requires bold leadership, pain, and cost but this is the kind of stuff that moves the needle and goes down as critical pivot points in history. 

Just my thought for the day...

--Lee

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feeds.feedburner.com/CellTherapyBlog

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The cost of clinical trial data bias/loss, FDA’s new job and the need for bold leadership.

Sunday, September 30th, 2012
Tweet 

The scandal of clinical trial data loss is eroding the fundamentals of evidence-based research and clinical medicine.


Before you right this post off as the stuff of conspiracy theories, fear-mongering, and 'alternative world views' consider that this view is shared by the likes of the FDA, the International Committee of Medical Journal Editors, the Cochrane Collaboration, and researchers at institutions like Johns Hopkins School of Medicine.


Here's the underlying premise as succinctly described by author Ben Goldacre:

"Drugs are tested by the people who manufacture them, in poorly designed trials, on hopelessly small numbers of weird, unrepresentative patients, and analysed using techniques that are flawed by design, in such a way that they exaggerate the benefits of treatments. Unsurprisingly, these trials tend to produce results that favour the manufacturer.

When trials throw up results that companies don't like, they are perfectly entitled to hide them from doctors and patients, so we only ever see a distorted picture of any drug's true effects. Regulators see most of the trial data, but only from early on in a drug's life, and even then they don't give this data to doctors or patients, or even to other parts of government. This distorted evidence is then communicated and applied in a distorted fashion."

Authors M. Todwin and J. Abramson summarize it thusly:

"Trials with positive results generally are published more frequently than studies that conclude that a new drug poses greater risks or is no more effective than standard therapy or a placebo. Furthermore, some articles may distort trial findings by omitting important data or by modifying prespecified outcome measures. Lack of access to detailed information about clinical trials can undermine the integrity of medical knowledge."

Here is a great list of very recent resources that may convince you of the merits of this concern:

Yesterday, the US Secretary of Health and Human Services announced (in an FR notice) that the FDA was now charged with ensuring all organizations comply with the heretofore enacted but relatively unenforced  requirement to submit all relevant clinical trial data to http://www.clinicaltrials.gov

For further commentary on this move see the following reports from:
What is abundantly clear to me is that the FDA is left almost powerless - and if not powerless than certainly without sufficient resources - to successfully enforce its new power.  This requires collective industry leadership.  Bold, industry-initiated standards, infrastructure and old-fashioned peer pressure.

Here's what I wish.  

I wish that as a cell therapy industry we - through organizations like ISSCR, ARM, ISCT, etc and leading publishers of some of our leading journals like Regenerative Medicine, Cytotherapy, Cell Stem Cell, Stem Cells, etc - would take a leadership position on an issue like this.

I believe that as a relatively small and nascent sector of the biopharma industry we are more likely capable of collaborating on something important like this than larger, more established [entrenched] and diverse sectors.  Of course it requires the political will and cajones.

The payoff from our sector in taking a leadership role on this issue could potentially be enormous in terms of providing our sector with truly transparent and useful data.  Perhaps even more important would be the public profile such leadership would provide the sector.  Such a move requires bold leadership, pain, and cost but this is the kind of stuff that moves the needle and goes down as critical pivot points in history. 

Just my thought for the day...

--Lee

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feeds.feedburner.com/CellTherapyBlog

Read More...

Anticipated short-term cell therapy industry clinical milestones

Sunday, September 30th, 2012
Tweet 

What follows is an interesting but not exhaustive list of cell therapy industry clinical milestones we anticipate in the next 3-9 months as selected from the list of cell therapy products we are tracking in late-stage or post-commercial development.  


There are other commercial milestones we are monitoring as well as other clinical milestones we expect to see related to cell therapy products in earlier stages of the development pipeline that are not included below.


CellCoTec (http://www.cellcotec.com)
  • Having completed a trial in Europe of their device to enable POC production of an autologous chondrocyte cellular product in/with a biodegradable, load-bearing scaffold for the treatment of articular cartilage defects, they have now submitted their CE market application.  The CE mark application is under review and they anticipate a response in October.  
  • This device and the potential emergence of Sanofi's MACI in the European market next year may have an impact on Tigenix (EBR:TIG) most directly.



ERYtech Parma (http://www.erytech.com)

  • Their 'pivotal' phase 2/3 trial in Europe of lead product, GRASPA, for the treatment of Acute Lymphoblastic Leukemia (ALL) is scheduled for completion 2H 2012. 


GamidaCell (http://www.gamidacell.com)

  • Their 'pivotal' phase 2/3 trial in the US, Israel, and Europe of lead product, StemEx, for the treatment of leukemia and lymphoma, in joint development with Teva, completed enrollment in February and is scheduled for completion 2H 2012.  They have not been shy about the fact they expect to be in the market in 2013.


Innovacell (http://www.innovacell.com)

  • They raised over 8m Euro in April for a phase 3 trial in Europe for their lead product, ICES13, for the treatment of stress-urinary incontinence which was scheduled for a preliminary clinical data readout in Q4 2012 and be ready for market authorization in 2013. Since announcing the capital raise the company has been stone silent and no clinical trial registry has been filed.  Status unknown.


Miltenyi Biotec (www.miltenyibiotec.com)

  • Their phase 3 trial in Germany of CD133+ cells as an adjunct to CABG surgery for myocardial ischemia or coronary artery disease is scheduled for completion in January.


NovaRx (http://www.novarx.com)

  • Their phase 3 trial in US, Europe, and India of their lead product, Lucanix, for the treatment of advanced Non-small Cell Lung Cancer (NSCLC) following front-line chemotherapy is scheduled in clnicaltrials.gov for completion in October but we have learned they expect their next 'interim analysis' in February.


NuVasive (http://www.nuvasive.com)

  • They have a series of trials scheduled to complete 2H 2012 intended to provide additional clinical data to support its marketing of Osteocel Plus for the treatment of a growing number of orthopedic applications.


Sanofi's Genzyme (http://www.genzyme.com)

  • Having completed their phase 3 trial in Europe of MACI for knee repair (symptomatic articular cartilage defects of the femoral condyle including the trochlea), they expect to file their market authorization application (MAA) in 1H 2013.


Hope that's helpful and gives you a sense some of the late-stage things to watch for in the coming weeks and months.  



--Lee

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

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BioTime Forms BioTime Acquisition Corporation

Friday, September 28th, 2012

ALAMEDA, Calif.--(BUSINESS WIRE)--

BioTime, Inc. (NYSE MKT: BTX), an Alameda-based company engaged in research and development of innovative new products in the field of regenerative medicine utilizing stem cells and related technology, announced today that it has formed a new wholly owned subsidiary, BioTime Acquisition Corporation, to pursue opportunities and acquire assets and businesses in the fields of stem cells and regenerative medicine. Thomas Okarma, PhD, MD, will serve as the Chief Executive Officer and as a member of the board of directors of BioTimes new subsidiary. Dr. Okarma is the former President and Chief Executive Officer of Geron Corporation and served on that companys board of directors.

Since 2010, BioTime has expanded the scope of its business through strategic acquisitions and has been continually exploring other acquisition opportunities in its fields of interest. BioTimes strategic acquisitions include:

Global advances on multiple fronts of stem cell biology have established the foundation for an integrative business approach to consolidate and translate these discoveries into products that may revolutionize clinical medicine, said Thomas Okarma, the new companys CEO. Living cell therapies can now be scalably manufactured, efficiently distributed to points of care, and tested in controlled clinical trials.The goal of regenerative medicine is to go beyond the reach of pills and scalpels to achieve a new level of healing that may, after a single administration of therapeutic cells, permanently restore function to tissues and organs damaged by chronic disease or injury. BioTime Acquisition Corporation intends to build its business by identifying, consolidating, and commercially developing the best available cell therapy technologies to realize the potential of regenerative medicine. Ultimately, the goal is to bring these new therapies to the many millions of patients who need them.

The breadth of Dr. Okarmas experience in the field of cell-based therapeutics is simply spectacular, said Michael D. West, PhD, BioTimes Chief Executive Officer. We look forward to working together with him to translate these new scientific advances into commercial products for the large and growing markets driven by age-related degenerative diseases.

Dr. Okarma has had a distinguished career as a physician and an innovator and executive in the biotechnology industry. Dr. Okarma served as Gerons President, Chief Executive Officer, and as a member of its board of directors from July 1999 until February 2011, after having previously served as that companys Vice President of Research and Development and Vice President of Cell Therapies. In 1985, Dr. Okarma founded Applied Immune Sciences, Inc. (AIS) and served initially as its Vice President of Research and Development and subsequently as Chairman and Chief Executive Officer and as a director until that company was acquired by Rhone-Poulenc Rorer in 1995. After that acquisition, Dr. Okarma served as a Senior Vice President at Rhone-Poulenc Rorer until December 1996. From 1980 to 1992, Dr. Okarma was a member of the faculty of the Department of Medicine at Stanford University School of Medicine. Dr. Okarma holds an AB from Dartmouth College, an MD and PhD from Stanford University, and is a graduate of the Executive Education program of the Stanford Graduate School of Business.

About BioTime, Inc.

BioTime, headquartered in Alameda, California, is a biotechnology company focused on regenerative medicine and blood plasma volume expanders. Its broad platform of stem cell technologies is enhanced through subsidiaries focused on specific fields of application. BioTime develops and markets research products in the fields of stem cells and regenerative medicine, including a wide array of proprietary ACTCellerate cell lines, HyStem hydrogels, culture media, and differentiation kits. BioTime is developing Renevia (formerly known as HyStem-Rx), a biocompatible, implantable hyaluronan and collagen-based matrix for cell delivery in human clinical applications. BioTime's therapeutic product development strategy is pursued through subsidiaries that focus on specific organ systems and related diseases for which there is a high unmet medical need. BioTime's majority owned subsidiary Cell Cure Neurosciences Ltd. is developing therapeutic products derived from stem cells for the treatment of retinal and neural degenerative diseases. BioTime's subsidiary OrthoCyte Corporation is developing therapeutic applications of stem cells to treat orthopedic diseases and injuries. Another subsidiary, OncoCyte Corporation, focuses on the diagnostic and therapeutic applications of stem cell technology in cancer, including the diagnostic product PanC-Dx currently being developed for the detection of cancer in blood samples. ReCyte Therapeutics, Inc. is developing applications of BioTime's proprietary induced pluripotent stem cell technology to reverse the developmental aging of human cells to treat cardiovascular and blood cell diseases. BioTime's subsidiary LifeMap Sciences, Inc. markets GeneCards, the leading human gene database, and is developing an integrated database suite to complement GeneCards that will also include the LifeMap database of embryonic development, stem cell research and regenerative medicine, and MalaCards, the human disease database. LifeMap will also market BioTime research products. BioTime's lead product, Hextend, is a blood plasma volume expander manufactured and distributed in the U.S. by Hospira, Inc. and in South Korea by CJ CheilJedang Corporation under exclusive licensing agreements. Additional information about BioTime can be found on the web at http://www.biotimeinc.com.

Forward-Looking Statements

Statements pertaining to future financial and/or operating results, future growth in research, technology, clinical development, and potential opportunities for BioTime and its subsidiaries, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the ability to identify and complete potential acquisitions, the ability to realize anticipated benefits of and achieve expected financial performance following completed acquisitions, the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of BioTime and its subsidiaries, particularly those mentioned in the cautionary statements found in BioTime's Securities and Exchange Commission filings. BioTime disclaims any intent or obligation to update these forward-looking statements.

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Discovery of reprogramming signature may help further stem cell-based regenerative medicine research

Wednesday, September 19th, 2012

Public release date: 18-Sep-2012 [ | E-mail | Share ]

Contact: Andy Hoang ahoang@salk.edu 619-861-5811 Salk Institute

LA JOLLA, CA---- Salk scientists have identified a unique molecular signature in induced pluripotent stem cells (iPSCs), "reprogrammed" cells that show great promise in regenerative medicine thanks to their ability to generate a range of body tissues.

In this week's Proceedings of the National Academy of Sciences, the Salk scientists and their collaborators at University of California, San Diego, report that there is a consistent, signature difference between embryonic and induced pluripotent stem cells. The findings could help overcome hurdles to using the induced stem cells in regenerative medicine.

"We believe that iPSCs hold a great potential for the treatment of human patients," says Juan Carlos Izpisua Belmonte, a professor in Salk's Gene Expression Laboratory and the senior author on the paper. "Yet we must thoroughly understand the molecular mechanisms governing their safety profile in order to be confident of their function in the human body. With the discovery of these small, yet apparent, epigenetic differences, we believe that we are now one step closer to that goal."

Embryonic stem cells (ESCs) are known for their "pluripotency," the ability to differentiate into nearly any cell in the body. Because of this ability, it has long been thought that ESCs would be ideal to customize for therapeutic uses. However, when ESCs mature into specific cell types, and are then transplanted into a patient, they may elicit immune responses, potentially causing the patient to reject the cells.

In 2006, scientists discovered how to revert mature cells, which had already differentiated into particular cell types, such as skin cells or hair cells, back into a pluripotent state. These "induced pluripotent stem cells" (iPSCs), which could be developed from the patient's own cells, would theoretically carry no risk of immune rejection.

However, scientists found that iPSCs had molecular differences from embryonic stem cells. Specifically, there were epigenetic changes, chemical modifications in DNA that might alter genetic activity. At certain points in the iPSC's genome, scientists could see the presence of different patterns of methyl groups when compared to the genomes of ESCs. It seemed these changes occurred randomly.

Izpisua Belmonte and his colleagues wanted to understand more about these differences. Were they truly random, or was there a discernable pattern?

Unlike previous studies, which had primarily analyzed iPSCs derived from only one mature type of cells (mainly connective tissue cells called fibroblasts), the Salk and UCSD researchers examined iPSCs derived from six different mature cell types to see if there were any commonalities. They discovered that while there were hundreds of unpredictable changes, there were some that remained consistent across the cell types: the same nine genes were associated with these common changes in all iPSCs.

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Discovery of reprogramming signature may help further stem cell-based regenerative medicine research

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Cytomedix to Present at BIOX; Noble Financial Capital Markets' Life Sciences Exposition

Wednesday, September 19th, 2012

GAITHERSBURG, MD--(Marketwire - Sep 19, 2012) - Cytomedix, Inc. ( OTCQX : CMXI ), a fully integrated regenerative medicine company commercializing and developing innovative platelet and adult stem cell technologies, today announced that Martin P. Rosendale, Chief Executive Officer of Cytomedix, will present a corporate update at BIOX; Noble Financial Capital Markets' Life Sciences Exposition to be held at the University of Connecticut, Stamford Campus on September, 24-25, 2012.Mr. Rosendale's presentation will take place on Monday, September 24th at 8:00 a.m. Eastern time.

In addition to the corporate presentation, Mr. Rosendale will be a participant on the panel presentation titled "Advancements in Cell Therapy & Regenerative Medicine," on September 24th at 11:45 a.m.

Following the event, a high-definition video webcast of the Company's presentation and a copy of the presentation materials will be available on the Company's web site at http://www.cytomedix.com, or through the Noble Financial websites: http://www.noblefcm.com, or http://www.nobleresearch.com/BioExposition.htm. Microsoft SilverLight viewer (a free download from the presentation link) is required to participate. The webcast will be archived on Cytomedix's website for 90 days following the event.

About Noble Financial Noble Financial Capital Markets was established in 1984 and is an equity research driven, full-service, investment banking boutique focused on life sciences, technology and media, emerging growth, companies. The company has offices in New York, Boston, New Jersey, Los Angeles, and Boca Raton, FL. In addition to non-deal road shows and sector-specific conferences throughout the year, Noble Financial hosts its large format annual equity conference in January in South Florida featuring 150 presenting companies from across North America and total attendance of close to 600. For more information: http://www.noblefcm.com.

About Cytomedix, Inc. Cytomedix, Inc. is a fully integrated regenerative medicine company commercializing and developing innovative platelet and adult stem cell separation products that enhance the body's natural healing processes. The Company's advanced autologous technologies offer clinicians a new treatment paradigm for wound and tissue repair. The Company's patient-derived PRP systems are marketed by Cytomedix in the U.S. and distributed internationally.Our commercial products include the AutoloGel System, cleared by the FDA for wound care and the Angel Whole Blood Separation System. The Company is developing novel regenerative therapies using our proprietary ALDH Bright Cell ("ALDHbr") technology to isolate a unique, biologically active population of a patient's own stem cells.A Phase 2 trial evaluating the use of ALDHbr for the treatment of ischemic stroke is underway. For additional information please visit http://www.cytomedix.com.

Safe Harbor Statement Statements contained in this press release not relating to historical facts are forward-looking statements that are intended to fall within the safe harbor rule for such statements under the Private Securities Litigation Reform Act of 1995. The information contained in the forward-looking statements is inherently uncertain, and Cytomedix' actual results may differ materially due to a number of factors, many of which are beyond Cytomedix' ability to predict or control, including among many others, risks and uncertainties related to the Company's reimbursement related efforts,the Company's ability to capitalize on the benefits of the above-referenced CMS determination, the Company's ability to successfully and favorably conclude the negotiations and related discussions with the above-referenced global pharmaceutical company, the Company's ability to successfully integrate the Aldagen acquisition, to successfully manage contemplated clinical trials, to manage and address the capital needs, human resource, management, compliance and other challenges of a larger, more complex and integrated business enterprise, viability and effectiveness of the Company's sales approach and overall marketing strategies, commercial success or acceptance by the medical community, competitive responses, the Company's ability to raise additional capital and to continue as a going concern, and Cytomedix's ability to execute on its strategy to market the AutoloGel System as contemplated. To the extent that any statements made here are not historical, these statements are essentially forward-looking. The Company uses words and phrases such as "believes," "forecasted," "projects," "is expected," "remain confident," "will" and/or similar expressions to identify forward-looking statements in this press release. Undue reliance should not be placed on forward-looking information. These forward-looking statements are subject to known and unknown risks and uncertainties that could cause actual events to differ from the forward-looking statements. More information about some of these risks and uncertainties may be found in the reports filed with the Securities and Exchange Commission by Cytomedix, Inc. Cytomedix operates in a highly competitive and rapidly changing business and regulatory environment, thus new or unforeseen risks may arise. Accordingly, investors should not place any reliance on forward-looking statements as a prediction of actual results. Except as is expressly required by the federal securities laws, Cytomedix undertakes no obligation to update or revise any forward-looking statements, whether as a result of new information, changed circumstances or future events or for any other reason. Additional risks that could affect our future operating results are more fully described in our U.S. Securities and Exchange Commission filings, including our Annual Report on Form 10-K for the year ended December 31, 2011 and other subsequent filings. These filings are available at http://www.sec.gov.

Excerpt from:
Cytomedix to Present at BIOX; Noble Financial Capital Markets' Life Sciences Exposition

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BioTime CEO Michael D. West to Present at Stem Cells USA & Regenerative Medicine Congress 2012

Wednesday, September 19th, 2012

ALAMEDA, Calif.--(BUSINESS WIRE)--

BioTime, Inc. (NYSE MKT: BTX) announced that Chief Executive Officer Michael D. West, Ph.D. will present at the Stem Cells USA & Regenerative Medicine Congress 2012 in Cambridge, MA on Thursday, September 20, 2012. Dr. West will speak on Second Generation hES Cell-Based Therapies: Achieving Purity and Scalability in the Midst of Diversity in the session Developments in Novel Therapeutics. The presentation will be made available on BioTime's website at http://www.biotimeinc.com.

The Stem Cells USA & Regenerative Medicine Congress 2012, September 20-21, is North Americas leading commercial stem cell event. This years conference will focus on strategies and business models for navigating the stem cell and regenerative medicine marketplace for pharma, biotech, and investors.

About BioTime, Inc.

BioTime, headquartered in Alameda, California, is a biotechnology company focused on regenerative medicine and blood plasma volume expanders. Its broad platform of stem cell technologies is enhanced through subsidiaries focused on specific fields of application. BioTime develops and markets research products in the field of stem cells and regenerative medicine, including a wide array of proprietary ACTCellerate cell lines, HyStem hydrogels, culture media, and differentiation kits. BioTime is developing Renevia (formerly known as HyStem-Rx), a biocompatible, implantable hyaluronan and collagen-based matrix for cell delivery in human clinical applications. BioTime's therapeutic product development strategy is pursued through subsidiaries that focus on specific organ systems and related diseases for which there is a high unmet medical need. BioTime's majority owned subsidiary Cell Cure Neurosciences Ltd. is developing therapeutic products derived from stem cells for the treatment of retinal and neural degenerative diseases. BioTime's subsidiary OrthoCyte Corporation is developing therapeutic applications of stem cells to treat orthopedic diseases and injuries. Another subsidiary, OncoCyte Corporation, focuses on the diagnostic and therapeutic applications of stem cell technology in cancer, including the diagnostic product PanC-Dx currently being developed for the detection of cancer in blood samples. ReCyte Therapeutics, Inc. is developing applications of BioTime's proprietary induced pluripotent stem cell technology to reverse the developmental aging of human cells to treat cardiovascular and blood cell diseases. BioTime's subsidiary, LifeMap Sciences, Inc., markets GeneCards, the leading human gene database, and is developing an integrated database suite to complement GeneCards that will also include the LifeMap database of embryonic development, stem cell research and regenerative medicine, and MalaCards, the human disease database. LifeMap will also market BioTime research products. BioTime's lead product, Hextend, is a blood plasma volume expander manufactured and distributed in the U.S. by Hospira, Inc. and in South Korea by CJ CheilJedang Corporation under exclusive licensing agreements. Additional information about BioTime can be found on the web at http://www.biotimeinc.com.

Forward-Looking Statements

Statements pertaining to future financial and/or operating results, future growth in research, technology, clinical development, and potential opportunities for BioTime and its subsidiaries, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of BioTime and its subsidiaries, particularly those mentioned in the cautionary statements found in BioTime's Securities and Exchange Commission filings. BioTime disclaims any intent or obligation to update these forward-looking statements.

To receive ongoing BioTime corporate communications, please click on the following link to join our email alert list:

http://phx.corporate-ir.net/phoenix.zhtml?c=83805&p=irol-alerts

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BioTime CEO Michael D. West to Present at Stem Cells USA & Regenerative Medicine Congress 2012

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Developments of Stem Cell Therapy and Regenerative Medicine

Wednesday, September 19th, 2012

Queenstown Regenerative Medicine - http://www.queenstownRM.co.nz

Professor Richard Boyd and Dr Dan Bates Latest developments of Stem Cell Therapy and Regenerative Medicine

Queenstown Regenerative Medicine, in association with Monash University Immunology and Stem Cell Centre (MISCL), has the pleasure of requesting your attendance at an evening lecture by Prof Richard Boyd, Head of MISCL and Dr Dan Bates, Sports Medicine Physician from Melbourne AFL Club.

Professor Richard Boyd is a world leader in the research and development of potential uses of stem cells to treat disease in both human and animal. He is the Director of Australia's largest and most prestigious Stem Cell Laboratory and a recipient of numerous International Awards for unique research into how stem cells and the immune system develop and how they have their effects in the body.

Professor Boyd's talk will give an overall background to stem cells and the work going on around the world to put these cellular therapies and regenerative medicine into the clinic.

Doctor Dan Bates is a Sports Medicine Physician working with Professor Boyd in the development and use of cellular medicine applications in the field of Sports Medicine and musculoskeletal injuries. Dan is the current team doctor of the Melbourne AFL club and will speak on his experiences using Platelet Rich Plasma to treat musculoskeletal injuries and the opening of stem cell treatment centres in conjunction with MISCL in Australia.

This is a unique opportunity to get first- hand knowledge from some of the best people in the field. These talks will be aimed at the practical applications of how you can use these therapies currently, as well as giving an idea of what the near future holds.

Date: Friday 21 September 2012 Time: from 6 pm 7.30 pm Location: Heritage Hotel, 91 Fernhill Road, Queenstown (Icon Conference Room) Cost: Free of charge

Scoop Media

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Developments of Stem Cell Therapy and Regenerative Medicine

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Discovery of reprogramming signature may help overcome barriers to stem cell-based regenerative medicine

Wednesday, September 19th, 2012

ScienceDaily (Sep. 18, 2012) Salk scientists have identified a unique molecular signature in induced pluripotent stem cells (iPSCs), "reprogrammed" cells that show great promise in regenerative medicine thanks to their ability to generate a range of body tissues.

In this week's Proceedings of the National Academy of Sciences, the Salk scientists and their collaborators at University of California, San Diego, report that there is a consistent, signature difference between embryonic and induced pluripotent stem cells. The findings could help overcome hurdles to using the induced stem cells in regenerative medicine.

"We believe that iPSCs hold a great potential for the treatment of human patients," says Juan Carlos Izpisua Belmonte, a professor in Salk's Gene Expression Laboratory and the senior author on the paper. "Yet we must thoroughly understand the molecular mechanisms governing their safety profile in order to be confident of their function in the human body. With the discovery of these small, yet apparent, epigenetic differences, we believe that we are now one step closer to that goal."

Embryonic stem cells (ESCs) are known for their "pluripotency," the ability to differentiate into nearly any cell in the body. Because of this ability, it has long been thought that ESCs would be ideal to customize for therapeutic uses. However, when ESCs mature into specific cell types, and are then transplanted into a patient, they may elicit immune responses, potentially causing the patient to reject the cells.

In 2006, scientists discovered how to revert mature cells, which had already differentiated into particular cell types, such as skin cells or hair cells, back into a pluripotent state. These "induced pluripotent stem cells" (iPSCs), which could be developed from the patient's own cells, would theoretically carry no risk of immune rejection.

However, scientists found that iPSCs had molecular differences from embryonic stem cells. Specifically, there were epigenetic changes, chemical modifications in DNA that might alter genetic activity. At certain points in the iPSC's genome, scientists could see the presence of different patterns of methyl groups when compared to the genomes of ESCs. It seemed these changes occurred randomly.

Izpisua Belmonte and his colleagues wanted to understand more about these differences. Were they truly random, or was there a discernable pattern?

Unlike previous studies, which had primarily analyzed iPSCs derived from only one mature type of cells (mainly connective tissue cells called fibroblasts), the Salk and UCSD researchers examined iPSCs derived from six different mature cell types to see if there were any commonalities. They discovered that while there were hundreds of unpredictable changes, there were some that remained consistent across the cell types: the same nine genes were associated with these common changes in all iPSCs.

"We knew there were differences between iPSCs and ESCs," says Sergio Ruiz, first author of the paper, "We now have an identifying mark for what they are."

The therapeutic significance of these nine genes awaits further research. The importance of the current study is that it gives stem cells researchers a new and more precise understanding of iPSCs.

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Discovery of reprogramming signature may help overcome barriers to stem cell-based regenerative medicine

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Leading stem cell scientists to focus on diabetes, eye diseases at Cedars-Sinai symposium

Tuesday, September 18th, 2012

Public release date: 17-Sep-2012 [ | E-mail | Share ]

Contact: Nicole White nicole.white@cshs.org 310-423-5215 Cedars-Sinai Medical Center

LOS ANGELES Sept. 17, 2012 Leading scientists and clinicians from across the nation will discuss the latest findings on potential stem cell treatments for diabetes and eye diseases at the second Cedars-Sinai Regenerative Medicine Scientific Symposium.

WHO: Stem cell scientists, clinicians and industry leaders.

The symposium is being hosted by the Cedars-Sinai Regenerative Medicine Institute, led by Clive Svendsen, PhD. The institute brings together basic scientists with specialist clinicians, physician scientists and translational scientists across multiple medical specialties to convert fundamental stem cell studies to therapeutic regenerative medicine.

FEATURED RESEARCH: The symposium's morning session will feature an overview of the current state of stem cells and diabetes, including efforts to start the first clinical trials with stem cells for the treatment of diabetes. Other research to be presented includes an update on regenerative medicine approaches to treating macular degeneration, a progressive deterioration of the eye that causes gradual loss of vision. This will include an update from Gad Heilweil , MD, on a key, stem-cell clinical trial on macular degeneration at the University of California Los Angeles.

WHEN: Sept. 21, 2012 8:30 a.m. to 6 p.m. Thomson's lecture begins at 8:40 a.m.

WHERE: Harvey Morse Auditorium Cedars-Sinai Medical Center 8700 Beverly Boulevard Los Angeles, CA 90048

How to register: http://www.cedars-sinai.edu/RMI

###

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Cytomedix Shares Now Trading on OTCQX

Monday, September 17th, 2012

GAITHERSBURG, Md., Sept. 17, 2012 /PRNewswire/ -- Cytomedix, Inc. (CMXI), a fully integrated regenerative medicine company commercializing and developing innovative platelet and adult stem cell technologies, today announced that its common shares are now trading on the highest OTC marketplace, OTCQX, under the ticker symbol "CMXI."

Cytomedix upgraded from OTCQB today to trade on the OTC market's prestigious marketplace, OTCQX. Investors can find current financial disclosure and real-time Level 2 quotes for the Company's common shares at http://www.otcmarkets.com.

"We believe investors will appreciate the quality-controlled admission process, the transparent trading and easy access to company information that are hallmarks of the OTCQX," said Martin P. Rosendale, Chief Executive Officer of Cytomedix.

C. K. Cooper & Company will serve as Cytomedix's Designated Advisor for Disclosure ("DAD") on OTCQX, and will be responsible for providing guidance on OTCQX requirements.

About Cytomedix, Inc. Cytomedix, Inc. is a fully integrated regenerative medicine company commercializing and developing innovative platelet and adult stem cell separation products that enhance the body's natural healing processes. The Company's advanced autologous technologies offer clinicians a new treatment paradigm for wound and tissue repair. The Company's patient-derived PRP systems are marketed by Cytomedix in the U.S. and distributed internationally. The Company's commercial products include the AutoloGel System, cleared by the FDA for wound care and the Angel Whole Blood Separation System. The Company is developing novel regenerative therapies using the proprietary ALDH Bright Cell ("ALDHbr") technology to isolate a unique, biologically active population of a patient's own stem cells. A Phase 2 trial evaluating the use of ALDHbr for the treatment of ischemic stroke is underway. For additional information please visit http://www.cytomedix.com.

About OTC Markets Group Inc. OTC Markets Group Inc. (OTCM) operates Open, Transparent and Connected financial marketplaces for investors to easily trade almost 10,000 equity and debt securities through the broker of their choice. Our OTC Link ATS directly links a diverse network of broker-dealers that provide liquidity and execution services for a wide spectrum of securities. We organize these securities into tiered marketplaces to inform investors of opportunities and risks including OTCQX - The Intelligent Marketplace for the Best OTC Companies; OTCQB - The Venture Marketplace; and OTC Pink - The Open Marketplace. Our data-driven platform enables efficient trading through any broker at the best possible price and empowers a broad range of companies to improve the quality and availability of information for their investors. To learn more about how we create smarter financial marketplaces, visit http://www.otcmarkets.com.

OTC Link ATS is operated by OTC Link LLC, member FINRA/SIPC and SEC registered ATS.

About Cytomedix, Inc.Cytomedix, Inc. is a fully integrated regenerative medicine company commercializing and developing innovative platelet and adult stem cell separation products that enhance the body's natural healing processes. The Company's advanced autologous technologies offer clinicians a new treatment paradigm for wound and tissue repair. The Company's patient-derived PRP systems are marketed by Cytomedix in the U.S. and distributed internationally. Our commercial products include the AutoloGel System, cleared by the FDA for wound care and the Angel Whole Blood Separation System. The Company is developing novel regenerative therapies using our proprietary ALDH Bright Cell ("ALDHbr") technology to isolate a unique, biologically active population of a patient's own stem cells. A Phase 2 trial evaluating the use of ALDHbr for the treatment of ischemic stroke is underway. For additional information please visit http://www.cytomedix.com.

Safe Harbor Statement Statements contained in this press release not relating to historical facts are forward-looking statements that are intended to fall within the safe harbor rule for such statements under the Private Securities Litigation Reform Act of 1995. The information contained in the forward-looking statements is inherently uncertain, and Cytomedix' actual results may differ materially due to a number of factors, many of which are beyond Cytomedix' ability to predict or control, including among many others, risks and uncertainties related to the Company's reimbursement related efforts, the Company's ability to capitalize on the benefits of the above-referenced CMS determination, the Company's ability to successfully and favorably conclude the negotiations and related discussions with the above-referenced global pharmaceutical company, the Company's ability to successfully integrate the Aldagen acquisition, to successfully manage contemplated clinical trials, to manage and address the capital needs, human resource, management, compliance and other challenges of a larger, more complex and integrated business enterprise, viability and effectiveness of the Company's sales approach and overall marketing strategies, commercial success or acceptance by the medical community, competitive responses, the Company's ability to raise additional capital and to continue as a going concern, and Cytomedix's ability to execute on its strategy to market the AutoloGel System as contemplated. To the extent that any statements made here are not historical, these statements are essentially forward-looking. The Company uses words and phrases such as "believes", "forecasted," "projects," "is expected," "remain confident," "will" and/or similar expressions to identify forward-looking statements in this press release. Undue reliance should not be placed on forward-looking information. These forward-looking statements are subject to known and unknown risks and uncertainties that could cause actual events to differ from the forward-looking statements. More information about some of these risks and uncertainties may be found in the reports filed with the Securities and Exchange Commission by Cytomedix, Inc. Cytomedix operates in a highly competitive and rapidly changing business and regulatory environment, thus new or unforeseen risks may arise. Accordingly, investors should not place any reliance on forward-looking statements as a prediction of actual results. Except as is expressly required by the federal securities laws, Cytomedix undertakes no obligation to update or revise any forward-looking statements, whether as a result of new information, changed circumstances or future events or for any other reason. Additional risks that could affect our future operating results are more fully described in our U.S. Securities and Exchange Commission filings, including our Annual Report on Form 10-K for the year ended December 31, 2011 and other subsequent filings. These filings are available at http://www.sec.gov.

Contacts:

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