Monthly Archives: June 2020

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

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Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

The regional analysis covers:

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Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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Stem Cell Therapy Market Analysis and Demand 2017 2025 - Cole of Duty

The current coronavirus pandemic clearly illustrates how dangerous viral infections can become for us. Independent of the present situation, there are people whose bodies are defenseless against infections because their immune systems are unable to combat them - they suffer from immunodeficiency diseases such as ADA-SCID (adenosine deaminase severe combined immunodeficiency) or Wiskott-Aldrich syndrome. Prof. Dr. Alessandro Aiuti, a physician and research scientist based in Milan who works at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) and at the Vita Salute San Raffaele University, is now being honored with the Else Krner Fresenius Prize for Medical Research 2020 for his groundbreaking successes in the development of gene therapies. The award is coupled to 2.5 million euros in prize money.

In the case of the rare immune disorder ADA-SCID, which exclusively afflicts young children and occurs about 15 times a year in Europe, a defective ADA gene within the genome disrupts lymphocyte development, leaving the young patient's body defenseless against infections. "Without effective therapy, the children rarely survive for more than 2 years because any infection can become perilous for them," Aiuti explains. Standard for this therapy is a bone marrow transplantation from a fully matched sibling. However, a suitable donor is available only for a minority of patients. "Meanwhile children with such a condition benefit from the advances we have made in the field of gene therapy. So far we have treated 36 children from 19 countries using the therapy we developed. In more than 80 percent of the cases, the treatment has had such an impact that no enzyme replacement therapy or transplantation is needed. This achievement has been made possible by the extraordinary effort and dedication of SR-Tiget researchers and clinical team throughout 25 years," Aiuti adds. All of the patients are still alive.

For these successes and his other work in the field of gene therapy, Alessandro Aiuti has now been honored with the Else Krner Fresenius Prize for Medical Research 2020 awarded by the Else Krner-Fresenius-Stiftung (EKFS) foundation. At 2.5 million euros, this award is one of the highest endowed prizes for medical research in the world. "Still young by comparison, this year the prize is being awarded for the third time. It honors research scientists for pioneering contributions in the areas of biomedical science. A major percentage of the prize money flows into the prizewinner's research and is supposed to contribute toward achieving further groundbreaking findings and medical breakthroughs in the future as well," emphasizes Prof. Dr. Michael Madeja, scientific director and member of the management board at EKFS.

The decision regarding the prize recipient was made by a ten-member international jury composed of renowned research scientists in the fields of genome editing and gene therapy along with delegates from the Scientific Commission at EKFS. Prof. Dr. Hildegard Bning, chairwoman of the jury and president of the European Society for Gene and Cell Therapy (ESGCT), substantiates the jury's decision: "Alessandro Aiuti is a truly outstanding physician and scientist. His work has decisively contributed to the development and successful treatment of rare, genetically caused disorders such as SCID. Thanks not least of all to the contributions he has made, even patients with other inheritable illnesses can presumably be treated successfully in the future."

After successful clinical trials, the gene therapy developed for ADA-SCID patients was approved as a pharmaceutical remedy in Europe. It is considered to be one of the key findings in the development of gene therapies worldwide. With this treatment certain blood stem cells (CD34+) are taken from the patient, then the cell DNA is modified. The cells are treated outside the body using a viral vector to accomplish this. The correct version of the gene for the ADA enzyme is introduced into the genome of the cells that were collected. The genetically modified cells are returned to the patient's bloodstream via intravenous infusion. A portion of the modified cells subsequently establish themselves in bone marrow again. The patient now has blood stem cells that function properly and produce lymphocytes to defend against infections - presumably on a life-long basis.

Alessandro Aiuti wants to utilize the prize money from EKFS to set the success story forth, to optimize the therapies further and map out the healing mechanisms involved in a better fashion. The scientist sees another major challenge in conveying the acquired knowledge beyond the successful gene therapies from Milan to as many other genetic disorders as possible. Alongside the therapy for ADA-SCID, the San Raffaele Telethon Institute for Gene Therapy has also developed gene therapies for four more hereditary diseases, among them the Wiskott-Aldrich syndrome and metachromatic leukodystrophy (MLD). To this day a total of more than 100 patients from 35 different countries have been treated.

Biography of Alessandro Aiuti

Alessandro Aiuti was born in Rome in 1966 and studied medicine there at Sapienza University. Following a stay at Harvard Medical School in Boston, Massachusetts in the USA, he received his doctorate in Human Biology in 1996 from Sapienza University. Since 1997 he has been active at the San Raffaele Scientific Institute in Milan, where he meanwhile also teaches as a professor at the Vita Salute San Raffaele University. He is furthermore Deputy Director of Clinical Research at the San Raffaele Telethon Institute for Gene Therapy and Head of the Pediatric Immunohematology Unit at San Raffaele Hospital.

Aiuti is the author of numerous and highly acclaimed publications. Over the course of his career he has received a number of prizes from national and international institutions. Aiuti is a member of the board of the European Society of Gene and Cell Therapy, and a member of the EMA Committee for Advanced Therapies since 2019.

The Else Krner Fresenius Prize for Medical Research

The international Else Krner Fresenius Prize for Medical Research came into existence in 2013 on the occasion of the 25th anniversary of Else Krner's death and is awarded in alternating fields of biomedical science. Endowed with 2.5 million euros, the prize is one of the most highly endowed medical research awards in the world. It honors and supports research scientists who have made significant scientific contributions in their fields and whose work can be expected to yield groundbreaking findings and medical breakthroughs in the future as well.

The Else Krner-Fresenius-Stiftung (EKFS) foundation - Advancing research. Helping people.

The Else Krner-Fresenius-Stiftung, a non-profit foundation, is dedicated to the funding of medical research and supports medical/humanitarian projects. The foundation was established in 1983 by entrepreneur Else Krner and appointed as her sole heir. EKFS receives virtually all of its income in dividends from the Fresenius healthcare group, in which the foundation is the majority shareholder. To date, the foundation has funded around 2,000 projects. With a current annual funding volume around 60 million euros the EKFS is one of the largest foundations for medicine in Germany. More information:www.ekfs.de.

The San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele and Fondazione Telethon. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases. Over the years, the Institute has given a pioneering contribution to the field with relevant discoveries in vector design, gene transfer strategies, stem cell biology, identity and mechanism of action of innate immune cells. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and storage disorders, which have already treated >115 patients and have led through collaboration with industrial partners to the filing and approval of novel advanced gene therapy medicines.

Fondazione Telethon

Fondazione Telethon is a non-profit organisation created in 1990 as a response to the appeals of a patient association group of stakeholders, who saw scientific research as the only real opportunity to effectively fight genetic diseases. Thanks to the funds raised through the television marathon, along with other initiatives and a network of partners and volunteers, Telethon finances the best scientific research on rare genetic diseases, evaluated and selected by independent internationally renowned experts, with the ultimate objective of making the treatments developed available to everyone who needs them. Throughout its 30 years of activity, Fondazione Telethon has invested more than EUR 528 million in funding more than 2.630 projects to study more than 570 diseases, involving over 1.600 scientists. Fondazione Telethon has made a significant contribution to the worldwide advancement of knowledge regarding rare genetic diseases and of academic research and drug development with a view to developing treatments. For more information, please visit:www.telethon.it

Issued by news aktuell/ots on behalf of Else Krner-Fresenius-Stiftung

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Groundbreaking Gene Therapies for Hereditary Diseases / Alessandro Aiuti, a physician and research scientist from Milan, receives the Else Krner...

What the company does

Human retinal progenitor cells (hRPC)

Human retinal progenitor cells differentiate into components of the retina.

Reneuron has developed the ability to scale up the manufacturing of hRPCs using a patented low-oxygen cell expansion technology.

The hRPC cell therapy candidate is being evaluated in an ongoing phase I/IIa clinical trial in the US in subjects with a blindness-causing inherited retinal disease, retinitis pigmentosa (RP).

CTX Cells

CTX cell therapy candidate is a treatment for patients left disabled by the effects of a stroke.

Reneurons product is a standardised, clinical and commercial-grade cell therapy product capable of treating all eligible patients presenting with the diseases targeted, without the need for additional immunosuppressive drug treatments.

Data from the Phase II PISCES trial indicated CTX therapy was safe and well-tolerated and produced clinically meaningful and sustained improvement in the level of disability and dependence as well as motor function.

Exosome platform

Exosomes are nanoparticles, released by cells, and contain a number of active proteins and micro RNAs, which are short non-coding RNAs capable of regulating gene expression, that arebelieved to play a key role in cell-to-cell communication.

ExoPr0, Reneurons first CTX-derived exosome therapeutic candidate, has demonstrated potential as both a novel therapeutic candidate as well as a drug delivery vehicle

hRPC

's ()human retinal progenitor cells (hRPC) have scored some early success.

A Phase I/II assessment of a very small group of sufferers of a blindness-causing disease called retinitis pigmentosa saw a significant improvement in vision after treatment.

Six months after treatment there was a mean improvement of 18.5per treated eye, with a mean improvement of 12 letters per treated eye after nine months, whereasinexorable disease progression is the norm for this disease.

With a total of 22 patients now treated and the study still ongoing, ReNeuron said the efficacy in subsequent patients was seen but at a lower rate and magnitude, with improvement in visual acuity ranging from +5 to +11 letters in the treated eye threemonths after treatment.

In February, clinicaldata from the PISCES II clinical trial were published in peer-reviewedJournal of Neurology, Neurosurgery, and Psychiatry.

CTX

A peer journal review published in May indicated a CTX human neural stem cell line can rescue deficits associated with an accepted animal model of Huntington's disease, a progressive genetic brain disorder.

ReNeuron has previously presented data demonstrating that its CTX stem cell line, currently undergoing clinical evaluation for the treatment of stroke disability, can cause functional and behavioural recovery in animal models of ischemic (restriction of blood supply) injury.

The new data showed that implantation of CTX cells into a model of Huntington's disease can reduce inflammation, glial scar formation and induce host neurogenesis (the generation of new brain cells) leading to a recovery in behavioural deficits.

Coronavirus

In April, Reneuron said it haddeveloped a line of the human exosomes that can deliver a medically relevant payload: Viral vaccines thatmight help in the fight against coronavirus.

The stem cell specialist added that the disruption from lockdowns would inevitably lead to delays in the recruitment of patients for trials of its treatments for stroke disability and retinitis pigmentosa (RP).

It said it will update on how this will affect the release of top-line data from the two studies once it knows the full impact of the restrictions.

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ReNeuron encouraged by progress in stroke and RP treatments - Proactive Investors Australia

Scientists have successfully transplanted functional miniature livers into rats, after growing the bioengineered organs in the lab from reprogrammed human skin cells.

The experiment, which gave the animals working liver organs, could lay the groundwork for future treatments to address terminal liver failure a disease that claims the lives of over 40,000 people in the US every year.

While there's still a lot of work to be done before the technique can directly aid human patients, the researchers say their proof of concept may help underpin a future alternative to liver transplants, which are often incredibly expensive procedures to perform, in addition to being strictly limited by donor supply.

Another positive outcome would be using the approach to temporarily augment failing liver function in patients, lengthening people's lives while they're on the waiting list for these vital organs: a situation facing about 14,000 Americans at any given moment, most of whom won't ever receive a transplant.

"The long-term goal is to create organs that can replace organ donation, but in the near future, I see this as a bridge to transplant," explains pathologist Alejandro Soto-Gutirrez from the University of Pittsburgh.

"For instance, in acute liver failure, you might just need hepatic boost for a while instead of a whole new liver."

To grow their mini livers, the researchers took human skin cells donated by volunteers and reverted them to a stem cell state, known as induced pluripotent stem cells, from which other kinds of cell types can be derived.

The researchers then induced differentiation in the cells with the aid of hormones and other chemicals, prompting them to become liver cells, which were cultured in the lab.

While it ordinarily takes two years for a human's liver to mature from the moment of their birth, the researchers were able to grow their miniature analogues in only a matter of weeks, seeding the grown cells on a rat liver scaffold that had been stripped of its rat cells.

While previous experimental liver graft research has incorporated rodent cells onto the scaffold, here the researchers used the human stem cells to populate the liver's functional tissue, along with its vascular system and bile duct network.

When transplanted into five rats, the mini livers appeared to be functional. After four days - at which point the animals were sacrificed and dissected - tests revealed that the bioengineered livers secreted bile acids and urea; human liver proteins in the animals' blood were another sign that the organs were working.

Not that the transplants functioned perfectly. Evidence of poor blood flow into the graft, in addition to thrombosis and ischemia, reveals there are still serious difficulties in properly connecting grafts like these to an animal's vascular network.

Nonetheless, it's still a remarkable achievement. For a short time, five rats lived their lives with miniature human livers, which isn't something that's ever been demonstrated before, and it could bring us closer to employing the same techniques for the benefit of human patients one day.

That day might be a long time away (perhaps a decade off, the researchers suggest), although it depends on a huge range of future experiments succeeding, including demonstrating that these kinds of engineered transplants are safe for humans, which remains to be seen.

In the meantime, methods like this including work pioneered by the same laboratory last year could enable the use of such mini organs to study simulated diseases and test different treatment options.

"I believe it's a very important step because we know it can be done," Soto-Gutirrez explained to Inverse. "You can make a whole organ that can be functional from one cell of the skin."

The findings are reported in Cell Reports.

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Tiny Human Livers Grown in The Lab Have Been Successfully Transplanted Into Rats - ScienceAlert