Angioblast Systems - FAQ. Adult stem cells, repair, regeneration of blood vessels, adult stem cells technology, cardiovascular disease,

Section 1 - Scientific

What are adult stem cells?

Adult stem cells retain the ability to differentiate into various tissues of the body. Unlike embryonic stem cells, those from adult tissues are restricted in the number of different tissues that they can become. A subset of adult stem cells termed mesenchymal precursor cells (MPCs) is able to transform into tissues including heart muscle, blood vessels, bone, cartilage, and. MPCs have been shown in animal models to improve function in a variety of cardiovascular diseases.

Which stem cells are used for Angioblast's technology?

Angioblast uses a type of stem cell called a mesenchymal precursor cell (MPC) which has the ability to differentiate into a wide variety of tissues including heart muscle, blood vessels, bone cartilage and other mesenchymal lineages.

Why do adult stem cells offer certain advantages over embryonic stem cells?

Adult stem cells are more differentiated, meaning that they have less capacity than embryonic stem cells to grow and divide out of control (and possibly lead to the formation of tumors). Furthermore, they are less likely to transform spontaneously into unwanted tissue types. Importantly, the use of adult stem cells overcomes the ethical concerns associated with embryonic stem cells.

How do adult stem cells become specialized?

The adult stem cells become specialized by responding to local stimuli and signals from the injured tissue. In addition, MPCs secrete biochemical factors that induce the body's own tissue or cells to grow and regenerate, effectively shrinking the injury.

What are the potential therapeutic applications for Angioblast's technology?

Initially, Angioblast will examine the use of MPCs to treat heart failure, damage done by heart attacks, and peripheral artery disease.

Does the patient have to use their own MPCs for treatment?

No, although use of the patient's own cells is one way of delivering the treatment. One of the problems with this approach is the time taken to grow enough of the patient's cells from the relatively small number that can be collected from the body. A second disadvantage would be the cost of individualised therapy. For these reasons, Angioblast is developing ‘off-the-shelf' products based on MPCs.

What is an ‘off-the-shelf' product based on adult stem cells?

MPCs from unrelated donors have the unique ability of not stimulating an immune reaction when introduced into the patient. MPCs from donors can be grown in large numbers and made available for treatment in much the same way as a drug.

What is the source of MPCs?

MPCs are found in small numbers in various organs and tissues, including dental pulp, skin, fat and bone marrow. They make up less than 0.01% of the cells in bone marrow.

How are MPCs harvested?

Cells are removed from bone marrow. MPCs are then purified from the mixed bone marrow population by mixing them with magnetic monoclonal antibodies that only binds to MPCs. The MPCs can be separated from other cell types by a magnet.

What happens to the MPCs next?

To obtain enough cells for a therapeutic effect, the MPCs are grown in the laboratory in a special medium that allows them to divide, but not to differentiate. Mesoblast is able to grow billions of cells from a single donor within six weeks.

How are MPCs administered?

The stem cells suspended in saline can be given via injection (into heart muscle). Alternatively, they can be delivered via a tube to the heart or an arthroscopic procedure for bone repair

How do MPCs recognize the damaged tissue?

The MPCs have certain innate homing abilities due to their ability to move towards signals that are secreted following injury.

What are the potential uses for Angioblast's adult stem cells, MPCs?

MPCs have the ability to differentiate into a variety of tissues, create their own blood supply and induce the body's own healthy cells to regenerate and proliferate. At this stage, their potential is unlimited; however Angioblast will initially concentrate on the development of MPC technology for the treatment of cardiovascular diseases including heart attacks, heart failure, stroke, diabetes and peripheral vascular disease.

Which patients are most likely to benefit from MPC therapy in the future?

The aging population and active lifestyles among all age groups inevitably result in increased numbers of heart attacks, heart failure, peripheral artery disease, diabetes, bone breaks, knee injuries, strokes and so on. Advances in medicine have meant that many patients survive what would once have been a fatal condition, but very often, their quality of life suffers after such acute injuries. In addition, the first heart attack or severe fracture can put the patient at an increased risk of developing secondary complications. MPC therapy could lower the risk of such complications and improve the quality of life.

Are there ethical or moral issues associated with adult stem cell therapy?

There are no ethical or moral issues surrounding embryo creation and destruction associated with adult stem cells. Adult stem cells are obtained in a safe manner from adults and do not pose any significant risks.

Has Angioblast completed any pre-clinical validation of its technology on humans and animals and if so, what were the outcomes?

Angioblast's MPC technology for bone regeneration has already been successfully tested with great success in animal studies. Pre-clinical animal studies using both rodents and sheep have demonstrated the ability of the MPCs to differentiate into a variety of tissue types, increase new blood vessel formation, induce the animal's own ability to regenerate and significantly improve function in cardiovascular models.

Who is responsible for this technology?

The technology was based on 10 years' pioneering work conducted at the Institute of Medical and Veterinary Science (IMVS) and the Hanson Institute in South Australia . Angioblast's founder and chief scientific adviser, Professor Silviu Itescu, conducted a worldwide search to locate the best technology and successfully licensed it in 2004.

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Section 2 –Business

What is the company's business strategy?

Angioblast's business strategy is to produce an “off-the-shelf” adult stem cell product, similar to that of the pharmaceutical companies' business model with drug therapies. MPCs will be taken from ‘universal' donors and grown in the laboratory in large numbers under GMP conditions. This will enable low cost of goods sold and high margin returns. Importantly, the universal donor model will allow easy access to source material and will allow multiple recipients to benefit, which is in contrast to other cell therapies.

What is the company's commercial strategy?

Angioblast's approach to commercialization will be focus driven and outcomes focused. As various stages milestones are attained in the next 2 to 2.5 years, Angioblast will seek to collaborate with international pharmaceutical companies to generate early revenues and maximize returns for shareholders.

What is a realistic time to achieve the major clinical and commercial milestones?

Angioblast is in the process of finalizing GMP production of MPCs for allogeneic clinical trials, and initiated an autologous human trial for victims of a heart attack, which should be complete in the first quarter of 2007. Angioblast also expects to file and gain approval of an Investigational New Drug (IND) application for clinical trials using allogeneic MPCs for cardiovascular conditions by the end of 2006.

What differentiates Angioblast's technology from that of its competitors?

Angioblast's technology ensures that its starting population of MPCs is more than 1000-fold purer than that of its competitors. Angioblast's MPCs have been shown in animal models to both create new blood vessels and stimulate cells at the site of damage to repair themselves, as well as providing functional cells to the region of tissue in need of repair. Because MPCs do not cause an immune reaction, MPCs for the treatment of different individuals can be grown from universal donors. This is not the case with other stem cell companies.

What is Angioblast's relationship with Mesoblast?

In 2004 Mesoblast had an IPO and committed to invest AUS$10,000,000 (in milestone-linked tranches) into Angioblast for a 33% equity interest in Angioblast. Angioblast and Mesoblast have and will continue to share certain costs relating to pre-clinical and clinical development, patent management and technology development. Angioblast was assigned all intellectual property for MPC-related discoveries from the Institute of Medical and Veterinary Science and the Hanson Institute in South Australia , covering composition of matter and enablement of MPCs. Mesoblast has a worldwide exclusive license for the use of MPCs in all orthopedic applications, and Angioblast has such a license for all other applications.



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	9 April 2010 - ANGIOBLAST’S MARKET EXCLUSIVITY FOR ITS BIOLOGIC PRODUCTS STRENGTHENED BY NEW UNITED STATES HEALTH CARE ACT 18 December 2009 - SINGLE DOSE OF PROPRIETARY ADULT STEM CELLS REGENERATES DAMAGED PANCREAS AND REDUCES BLOOD GUCOSE LEVELS IN DIABETES 18 November 2009 - SUSTAINED SIX-MONTH IMPORVEMENT IN HEART MUSCLE FUNCTION BY “OFF-THE-SHELF” ADULT STEM CELLS 06 November 2009 - SUCCESSFUL BONE MARROW REGENERATION IN CANCER PATIENTS USING CORD BLOOD EXPANDED BY PROPRIETARY STEM CELLS 25 August 2009 - ANGIOBLAST CLOSES $10M EQUITY-BASED FINANCINGANGIOBLAST CLOSES $10M EQUITY-BASED FINANCING		Section 1 - Scientific What are adult stem cells? Adult stem cells retain the ability to differentiate into various tissues of the body. Unlike embryonic stem cells, those from adult tissues are restricted in the number of different tissues that they can become. A subset of adult stem cells termed mesenchymal precursor cells (MPCs) is able to transform into tissues including heart muscle, blood vessels, bone, cartilage, and. MPCs have been shown in animal models to improve function in a variety of cardiovascular diseases. Which stem cells are used for Angioblast's technology? Angioblast uses a type of stem cell called a mesenchymal precursor cell (MPC) which has the ability to differentiate into a wide variety of tissues including heart muscle, blood vessels, bone cartilage and other mesenchymal lineages. Why do adult stem cells offer certain advantages over embryonic stem cells? Adult stem cells are more differentiated, meaning that they have less capacity than embryonic stem cells to grow and divide out of control (and possibly lead to the formation of tumors). Furthermore, they are less likely to transform spontaneously into unwanted tissue types. Importantly, the use of adult stem cells overcomes the ethical concerns associated with embryonic stem cells. How do adult stem cells become specialized? The adult stem cells become specialized by responding to local stimuli and signals from the injured tissue. In addition, MPCs secrete biochemical factors that induce the body's own tissue or cells to grow and regenerate, effectively shrinking the injury. What are the potential therapeutic applications for Angioblast's technology? Initially, Angioblast will examine the use of MPCs to treat heart failure, damage done by heart attacks, and peripheral artery disease. Does the patient have to use their own MPCs for treatment? No, although use of the patient's own cells is one way of delivering the treatment. One of the problems with this approach is the time taken to grow enough of the patient's cells from the relatively small number that can be collected from the body. A second disadvantage would be the cost of individualised therapy. For these reasons, Angioblast is developing ‘off-the-shelf' products based on MPCs. What is an ‘off-the-shelf' product based on adult stem cells? MPCs from unrelated donors have the unique ability of not stimulating an immune reaction when introduced into the patient. MPCs from donors can be grown in large numbers and made available for treatment in much the same way as a drug. What is the source of MPCs? MPCs are found in small numbers in various organs and tissues, including dental pulp, skin, fat and bone marrow. They make up less than 0.01% of the cells in bone marrow. How are MPCs harvested? Cells are removed from bone marrow. MPCs are then purified from the mixed bone marrow population by mixing them with magnetic monoclonal antibodies that only binds to MPCs. The MPCs can be separated from other cell types by a magnet. What happens to the MPCs next? To obtain enough cells for a therapeutic effect, the MPCs are grown in the laboratory in a special medium that allows them to divide, but not to differentiate. Mesoblast is able to grow billions of cells from a single donor within six weeks. How are MPCs administered? The stem cells suspended in saline can be given via injection (into heart muscle). Alternatively, they can be delivered via a tube to the heart or an arthroscopic procedure for bone repair How do MPCs recognize the damaged tissue? The MPCs have certain innate homing abilities due to their ability to move towards signals that are secreted following injury. What are the potential uses for Angioblast's adult stem cells, MPCs? MPCs have the ability to differentiate into a variety of tissues, create their own blood supply and induce the body's own healthy cells to regenerate and proliferate. At this stage, their potential is unlimited; however Angioblast will initially concentrate on the development of MPC technology for the treatment of cardiovascular diseases including heart attacks, heart failure, stroke, diabetes and peripheral vascular disease. Which patients are most likely to benefit from MPC therapy in the future? The aging population and active lifestyles among all age groups inevitably result in increased numbers of heart attacks, heart failure, peripheral artery disease, diabetes, bone breaks, knee injuries, strokes and so on. Advances in medicine have meant that many patients survive what would once have been a fatal condition, but very often, their quality of life suffers after such acute injuries. In addition, the first heart attack or severe fracture can put the patient at an increased risk of developing secondary complications. MPC therapy could lower the risk of such complications and improve the quality of life. Are there ethical or moral issues associated with adult stem cell therapy? There are no ethical or moral issues surrounding embryo creation and destruction associated with adult stem cells. Adult stem cells are obtained in a safe manner from adults and do not pose any significant risks. Has Angioblast completed any pre-clinical validation of its technology on humans and animals and if so, what were the outcomes? Angioblast's MPC technology for bone regeneration has already been successfully tested with great success in animal studies. Pre-clinical animal studies using both rodents and sheep have demonstrated the ability of the MPCs to differentiate into a variety of tissue types, increase new blood vessel formation, induce the animal's own ability to regenerate and significantly improve function in cardiovascular models. Who is responsible for this technology? The technology was based on 10 years' pioneering work conducted at the Institute of Medical and Veterinary Science (IMVS) and the Hanson Institute in South Australia . Angioblast's founder and chief scientific adviser, Professor Silviu Itescu, conducted a worldwide search to locate the best technology and successfully licensed it in 2004. -------------------------------------------------------------------------------- Section 2 –Business What is the company's business strategy? Angioblast's business strategy is to produce an “off-the-shelf” adult stem cell product, similar to that of the pharmaceutical companies' business model with drug therapies. MPCs will be taken from ‘universal' donors and grown in the laboratory in large numbers under GMP conditions. This will enable low cost of goods sold and high margin returns. Importantly, the universal donor model will allow easy access to source material and will allow multiple recipients to benefit, which is in contrast to other cell therapies. What is the company's commercial strategy? Angioblast's approach to commercialization will be focus driven and outcomes focused. As various stages milestones are attained in the next 2 to 2.5 years, Angioblast will seek to collaborate with international pharmaceutical companies to generate early revenues and maximize returns for shareholders. What is a realistic time to achieve the major clinical and commercial milestones? Angioblast is in the process of finalizing GMP production of MPCs for allogeneic clinical trials, and initiated an autologous human trial for victims of a heart attack, which should be complete in the first quarter of 2007. Angioblast also expects to file and gain approval of an Investigational New Drug (IND) application for clinical trials using allogeneic MPCs for cardiovascular conditions by the end of 2006. What differentiates Angioblast's technology from that of its competitors? Angioblast's technology ensures that its starting population of MPCs is more than 1000-fold purer than that of its competitors. Angioblast's MPCs have been shown in animal models to both create new blood vessels and stimulate cells at the site of damage to repair themselves, as well as providing functional cells to the region of tissue in need of repair. Because MPCs do not cause an immune reaction, MPCs for the treatment of different individuals can be grown from universal donors. This is not the case with other stem cell companies. What is Angioblast's relationship with Mesoblast? In 2004 Mesoblast had an IPO and committed to invest AUS$10,000,000 (in milestone-linked tranches) into Angioblast for a 33% equity interest in Angioblast. Angioblast and Mesoblast have and will continue to share certain costs relating to pre-clinical and clinical development, patent management and technology development. Angioblast was assigned all intellectual property for MPC-related discoveries from the Institute of Medical and Veterinary Science and the Hanson Institute in South Australia , covering composition of matter and enablement of MPCs. Mesoblast has a worldwide exclusive license for the use of MPCs in all orthopedic applications, and Angioblast has such a license for all other applications.