Lee Rubin,

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In the presentation, Lee gives an overview on:

• Using stem cell-based screens to study the biology of orphan nervous system disorders and identify therapeutic targets
• How to address diseases without a strong genetic component
• How to address genetic variability
• Implications for the pharmaceutical industry: how does iPS disease modeling hold the potential to drastically improve drug pipelines for neurodegenerative diseases?

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For up to date information on the stem cells and regenerative medicine market, register now for Stem Cells USA & Regenerative Medicine Congress.

Brock Reeve, Executive Director at the Harvard Stem Cell Institute, will be participating in a panel session at this year’s conference on the topic, ‘Looking to alternative funding sources for the RM field.’

Kristin Comella,

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In her presentation, Kristin gives an overview on:

• Clinical development case study for MyoCell: challenges encountered and solutions for negotiating them
• Working with KOLs, regulatory authorities and advocacy groups: what is the respective involvement of each of these partners in improving clinical trials?
• Implications for the industry: what can other drug companies learn from the MyoCell story to date?

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For up to date information on the stem cells and regenerative medicine market, register now for Stem Cells USA & Regenerative Medicine Congress.

Natalie Mount,

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In her presentation, Natalie gives an overview on:

• What approach is Pfizer taking to developing innovative regenerative medicine projects?
• Cell therapy portfolio update and progress including the Athersys MultiStem Project and the London AMD Project
• Challenges and opportunities for Big Pharma in cell therapy

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For up to date information on the stem cells and regenerative medicine market, register now for Stem Cells USA & Regenerative Medicine Congress.

Download the presentation slides here.

In his presentation, David looks at biotech capital strategies, including government, public-private finance and entrepreneurial activities, and ways to drive innovation in regenerative medicine.

He gives an overview on:

• British Government investment in short-term feasibility studies to stimulate innovation by small companies in regenerative medicine
• The responsibility of funders: encouraging important initial feasibility work required to see if new ideas are workable
• Update on progress: who is getting funding? What results have been seen to date and how will this impact the field?

Get the presentation slides here.

For up to date information on the stem cells and regenerative medicine market, register now for Stem Cells USA & Regenerative Medicine Congress.

Yael Margolin

Each year, approximately 60,000 patients with leukemia or lymphoma can potentially be cured by a bone marrow transplantation. Of these, only approximately 25,000 are transplanted, primarily because they could not find a fully matched bone marrow donor in time. Cord blood transplantation (CBT) does not require full tissue matching and is considered a good alternative for transplantation. However, CBT is limited by a low number of TNC and CD34+ cells, influencing the incidence and rate of hematopoietic recovery and risk of early transplant-related mortality. Ex-vivo expansion is a strategy employed to increase the number of progenitor cells and improve clinical outcomes.

StemEx is a graft of stem/progenitor cells isolated and expanded from a portion of a single unit of umbilical cord blood (CBU) and transplanted in combination with non-expanded cells from the same unit. The portion of umbilical stem cells is expanded using Gamida Cell’s proprietary copper chelator technology.

StemEx is poised to answer the dire unmet clinical need in the field of bone marrow transplantation by supplying alternative cell grafts to 50,000 patients with hematological malignancies, who are indicated for transplantation but do not have a matched family related bone marrow donor. The market potential of StemEx is estimated at more than one billion USD.

StemEx is now being studied as a treatment for hematological malignancies in an international, Phase III, pivotal registration clinical trial. This is an open study with a historical control cohort: the design is approved by the FDA under SPA. The pivotal study has completed recruitment. Safety and efficacy clinical outcomes of the study will be available in Q4/2012. Gamida Cell

More than 100 StemEx batches were manufactured in 3 centralized GMP facilities. Data available for the first 88 batches shows a median fold expansion of TNC, CD34+ cells and CFU over culture input were 399 (range 52-764), 75 (6-280) and 107 (43-662), respectively. Since only a portion of the CBU isexpanded, patients were infused with a median 8.4 fold increase (0.8-90.3) in the number of CD34+ cells infused over the number that would be infused from the entire CBU without expansion. The CFU potential of culture seeded CD133+ cells measured at day-0 of production indicates the expansion potential of the cryopreserved CB cells. In all six batches which failed to expand, day-0 CFU was low, while day-0 CFU of all successfully expanded batches was within specification ranges. This information, available before patient myeloablation, strengthens the clinical applicability of StemEx.

Gamida Cell has developed a robust logistical infrastructure to support the clinical implementation of StemEx. All StemEx batches were successfully delivered and infused to the patients.

StemEx is developed in a 50/50 Joint Venture between Gamida Cell and Teva Pharmaceutical Industries. The JV is now seeking a strategic partner to support the global commercialization of StemEx.

Dr. Margolin will be presenting at this year’s Stem Cells USA & Regenerative Medicine Congress, on the topic, ‘StemEx pivotal trial completed – preparing for commercialization.’ Register now to hear her share her insights on the stem cells sector.

"With her body rejecting all possible treatment – and with no other options – we finally turned to Pluristem’s PLX cells, which literally saved her life," said Professor Reuven Or, Director of Bone Marrow Transplantation, Cell Therapy and Transplantation Research Center at Hadassah Medical Center and the child’s physician. The results of this unique case indicate that PLX cells may be effective in treating other diseases that affect the bone marrow."

The patient has been hospitalized at the Hadassah Hebrew University Medical Center, Jerusalem since August 2011. Her aplastic bone marrow had been refractory to treatment and, therefore, she underwent allogeneic stem cell transplantation from a matched unrelated donor. The first transplant was unsuccessful and the patient remained with bone marrow failure. Therefore, the patient underwent a second allogeneic stem cell transplantation from a second donor. Unfortunately, the bone marrow function was very poor and the patient suffered from recurrent infections. Approximately two months after the patient’s second bone marrow transplant, the child received PLX cells intramuscularly in two doses approximately one week apart. Approximately 10 days after the last administration of PLX cells, the patient’s hematological parameters began to significantly increase, an effect that has persisted to date. Additionally, the patient’s general clinical status has improved. Subsequent analysis has indicated that the PLX cells worked by stimulating the recovery of the hematopoietic stem cells contained in the
second bone marrow transplant that she had received over two months earlier.

"Pluristem is extremely happy that our PLX cells have helped this little girl," said Zami Aberman, Chairman and CEO of Pluristem. "Remarkably, these beneficial effects were seen in the patient after our PLX cells were administered intramuscularly (IM) and correlates with the positive effects on the bone marrow when we administered our PLX cells IM in animals exposed to toxic levels of radiation. Pluristem now has several data points to indicate that our PLX cells may work for systemic diseases when given locally, away from the target organ, and without a need to give cells intravenously.”

In February 2012, Pluristem announced the results of animal studies suggesting PLX cells can be potentially effective in treating the life threatening ematopoietic
complications associated with Acute Radiation Syndrome (ARS). In these experiments, animals given PLX cells IM up to 24 hours post irradiation demonstrated a recovery of their red cells, white cells, platelets and bone marrow to almost normal levels. It was that announcement, and the significant deterioration of the patient following two bone marrow transplants, that led Professor Reuven Or to contact Pluristem about the possible compassionate use of PLX cells to treat his young patient.

Pluristem recently received U.S. FDA Clearance to begin a Phase II clinical trial using the company’s proprietary PLX-PAD cell product candidate intramuscularly for the treatment of Intermittent Claudication (IC), a subset of peripheral artery disease (PAD). In April, the Company was awarded a $3.1 Million grant by the Israeli Government, which will be used to help fund R&D and clinical trials.

Dr. William R. Prather, Senior VP of Corporate Development at Pluristem, will be speaking at this year’s Stem Cells USA & Regenerative Medicine Congress on the topic ‘Treating acute radiation syndrome with PLX stem cells.’ Register now to hear him speak.

Today at World Stem Cells & Regenerative Medicine Congress 2012, Gil Van Bokkelen, CEO, at Athersys, delivered a presentation about clinical trials in stem cell.

Gil Van Bokkelen congratulated Osiris on approval of Prochymal for treating pediatric GvHD in Canada. This is an important event, because it demonstrates the growing regulatory acceptance of cell therapy and provides an example of how the field is progressing and maturing for the investors. He also highlighted that over 325, 000 patients have been treated with FDA approved cell therapy products.

Gil mentioned that not all of the programmes will be successful, so we need to be prepared that some clinical experiences will fail. However it is important to learn from the mistakes that have been made along the way.

Gil highlighted some major areas of medical need & opportunity:

• Cardiovascular disease
• Neurological injury & disease
• Inflammatory & immune disease
• Pulmonary disease
• Hematopoietic conditions
• Renal disease
• Orthopedic conditions

Some of the key clinical development goals that Gil identified are:

• What type of data do we need?
• What are the potential problems to overcome?
• What is in place to ensure failure at the late-stage clinical trial is avoided?
• How are the clinical end-points established to depict significant clinical added value?
• How to achieve a better regulatory transparency for pre-clinical development process?
• How to enhance the efficiency of clinical development?

Gil Van Bokkelen covered some of the ways to achieve these goals in his presentation. In conclusion Gil stressed the importance of establishing a clearly defined list of priorities: i.e. explicitly recognised unmet medical needs, recognised by appropriate bodies such as Health & Human Services and FDA, National Institute for Health & Clinical Excellence, European Medicines Agency. Therapies designed to specifically address these needs should be priority status & ideally list acceptable clinical end-points.

Gil enjoyed the Congress, he commented: “World Stem Cells & Regenerative Medicine Congress is Big enough – to represent all the leaders from the industry, but small enough to see and meet all the old friends.”

Check back here in a couple of days for the presentation. Excellent presentation Gil Van!

Today at World Stem Cells & Regenerative Medicine Congress 2012, Frank-Roman Lauter, Head of Business Development at Berlin-Brandenburg Centre for Regenerative Therapies, delivered a presentation about advancing pre-clinical research through to the clinic

Frank-Roman Lauter spoke about the foundation of the RMC – Berlin-Brandenburg Centre for Regenerative Therapies. He mentioned that sharing ideas and experience is much more frequent and free these days, than it used to be in the past. In his experience – openness really pays off. This positive experience of collaboration, motivated Frank-Roman to work on this project. Frank-Roman together with the experts from the industry, academic, government bodies and investors have established the RMC.
 
Here is what steps they are planning to take to achieve their goals:

- Foster communication

- Promote collaboration

- Enable accelerated delivery of new RM solutions

The structure of the consortium:
 
-       Core members: RM translational centres
-       Industry & associate members
-       RMC management office(s)
 
Among the benefits to the members are:
 
-       Capital efficiency
-       Access to larger network & experience pool
-       Critical mass for projects and funding
-       Influence on translation
-       First look at IP  technologies
-       Resources, opinion & opinion leadership
-       Opportunities of economic development
 
The foundation of RMC will be on the 25th May 2012!

Check back here in a couple of days for the presentation. Excellent presentation Frank-Roman!

Panel discussion included Michael May, Zahid Latif, Todd McAllister, Ruth McKernan and Greg Bonfiglio. The panel discussed the importance of global translation centers collaborating to advance stem cell therapies.

Michael May, the CEO of Centre for Commercialization of Regenerative Medicine (CCRM) told about his company. CCRM was set up relatively recently, and know all about the set up challenges. In Canada there have been 100’s of dollars invested in collaborating centers. CCRM brought together 6 institutions that represent more than 90% of regenerative medicine and cell therapy. Michael highlighted that their focus is product development technologies & combining stem cell technology with engineering. Another strategic development that is important for CCRM is to integrate excellence in science with business leadership: new employees have been hired, various industry projects have been launched, more than 35 inventions were made, first company creation was made and 3 other company creations have been identified. CCRM are looking at assets that they have, and thinking of what they could create collaboratively. Last week Pfizer-CCRM innovation fund was launched, as well as various discussions on collaboration outside Canada are being undertaken.

Zahid Latif spoke about the Technology Strategy Board, which is the UK’s business innovation agency and they try to identify and support those companies who are in particular need for it. They are a knowledge resource, which helps people address those challenges that they haven’t been able to tackle due to lack of finance.

To further highlight the importance of translation centers, Ruth has commented that successful projects need to be stimulated by investments: “We need to see the project succeed on a bigger scale. The investors need to be convinced that the value is there.”

All money that you spend don’t really move the technology forward. Todd believes that at the earliest stages when you are an academic fresh out of the lab – translation centers are incredibly important because of the funding they provide. In addition to that, in Greg’s opinion -  you need to find the technology and move it forward as quickly and efficiently as possible, you don’t need to build your own lab, as technology centers offer you the opportunity to do that without having to set up your own lab. In addition to that, you can get support from the experts in the technology lab.

Not only translation centers are working with researchers and SME’s , but they also collaborate with each other. There is a coalition of translation centers as they are trying to knit the existing centers into one powerful coalition.

To summarise – the most important for everyone on the panel is to drive the technology forward.

Check back here in a couple of days for the presentations.

Today at World Stem Cells & Regenerative Medicine Congress 2012, Keith Thompson, CEO at Cell Therapy Catapult Centre, delivered a presentation about the vision for the Cell Therapy Catapult

Some Key points raised in the presentation:

•    Establishing the Catapult in London
•    Experience in bringing cell therapies into clinical use
•    How will the Catapult work?
•    How will it impact the Cell Therapy industry?

Check back here in a couple of days for the presentation. Excellent presentation Keith!