European Commission approves Roche’s Polivy for people with previously treated aggressive lymphoma – GlobeNewswire

Polivy approval is based on a phase Ib/II study, the first and only study showing improved response rates and overall survival in patients with this aggressive lymphoma who are not candidates for a haematopoietic stem cell transplant, compared to a commonly used regimen

Basel, 21 January 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Commission has granted conditional marketing authorisation for Polivy (polatuzumab vedotin), in combination with bendamustine plus MabThera (rituximab) (BR), for the treatment of adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) who are not candidates for a haematopoietic stem cell transplant.

With this approval, people in the EU with relapsed or refractory diffuse large B-cell lymphoma will have the opportunity to benefit from this new Polivy combination, said Levi Garraway, M.D., Ph.D., Roches Chief Medical Officer and Head of Global Product Development. For patients battling this aggressive disease, the prognosis is poor and few treatments are available. We are proud to bring this first-in-class treatment option to those who need it most.

The conditional approval is based on the results from the phase Ib/II GO29365 study, the first and only clinical trial to show higher response rates and improved overall survival (OS) compared to BR, a commonly used regimen, in people with R/R DLBCL who are not candidates for a haematopoietic stem cell transplant. Results of the study showed that 40% of people treated with Polivy plus BR achieved a complete response (n=16/40), meaning no cancer could be detected at the time of assessment, compared to 17.5% (n=7/40) with BR alone. Complete response rates were assessed by an independent review committee. The study also showed that OS more than doubled, with a median of 12.4 months in the Polivy arm vs. 4.7 months in the BR alone arm (HR=0.42). Furthermore, patients treated with Polivy plus BR showed a longer time between first response to treatment and disease worsening than those receiving BR alone (investigator assessed median duration of response: 10.3 months vs. 4.1 months; HR=0.44). The most commonly reported adverse events in people treated with Polivy in combination with BR included anaemia, thrombocytopenia, neutropenia, fatigue, diarrhoea, nausea, and pyrexia.

Conditional approval is granted to a medicinal product that fulfils an unmet medical need where the benefit of immediate availability outweighs the risk of less comprehensive data than normally required.

Todays conditional EU approval follows the US Food and Drug Administrations (FDA) accelerated approval of Polivy in combination with BR for the treatment of people with R/R DLBCL who have received at least two prior therapies, in June 2019. Polivy was granted Breakthrough Therapy Designation by the FDA and PRIME (PRIority MEdicines) designation by the European Medicines Agency (EMA) for the treatment of people with R/R DLBCL in 2017, the first PRIME designation for a Roche medicine. Additional submissions of the GO29365 data to health authorities around the world are ongoing with the goal of bringing this new treatment option to more patients as soon as possible.

About the GO29365 studyGO29365 is a global, phase Ib/II study evaluating the safety, tolerability and activity of Polivy (polatuzumab vedotin) in combination with bendamustine and MabThera (rituximab) (BR) or Gazyvaro (obinutuzumab) in relapsed or refractory (R/R) follicular lymphoma or diffuse large B-cell lymphoma (DLBCL). Eligible patients were not candidates for a haematopoietic stem cell transplant at study entry. The phase II part of the study randomised 80 patients with heavily pre-treated R/R DLBCL to receive either BR, or BR in combination with Polivy for a fixed duration of six 21-day cycles. Of the patients enrolled, 80% had refractory disease. The primary endpoint was complete response (CR) at the end of treatment, as measured by positron emission tomography and assessed by an independent review committee (IRC). Secondary endpoints included overall response rate (ORR; CR and partial response) by investigator assessment and best ORR at the end of treatment by investigator and IRC assessment. Exploratory endpoints included duration of response, progression-free survival, event-free survival and overall survival.

About Polivy (polatuzumab vedotin)Polivy is a first-in-class anti-CD79b antibody-drug conjugate (ADC). The CD79b protein is expressed specifically in the majority of B-cells (an immune cell impacted in some types of non-Hodgkin lymphoma (NHL)), making it a promising target for the development of new therapies.1,2 Polivy binds to CD79b and destroys these B-cells through the delivery of an anti-cancer agent, which is thought to minimise the effects on normal cells.3,4 Polivy is being developed by Roche using Seattle Genetics ADC technology and is currently being investigated for the treatment of NHL. Polivy is marketed in the US by Genentech as Polivy (polatuzumab vedotin-piiq), with piiq as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the US Food and Drug Administration.

About diffuse large B-cell lymphomaDiffuse large B-cell lymphoma (DLBCL) is the most common form of non-Hodgkin lymphoma (NHL), accounting for about one in three cases of NHL.5 DLBCL is an aggressive (fast-growing) type of NHL, which is generally responsive to treatment in the frontline.6 However, as many as 40% of patients will relapse, at which time salvage therapy options are limited and survival is short.6 Approximately 150,000 people worldwide are estimated to be diagnosed with DLBCL each year.7

About Roche in haematologyRoche has been developing medicines for people with malignant and non-malignant blood diseases for over 20 years; our experience and knowledge in this therapeutic area runs deep. Today, we are investing more than ever in our effort to bring innovative treatment options to patients across a wide range of haematologic diseases. Our approved medicines include MabThera/Rituxan (rituximab), Gazyva/Gazyvaro (obinutuzumab), Polivy (polatuzumab vedotin), Venclexta/Venclyxto (venetoclax) in collaboration with AbbVie, and Hemlibra (emicizumab). Our pipeline of investigational haematology medicines includes idasanutlin, a small molecule which inhibits the interaction of MDM2 with p53; T-cell engaging bispecific antibodies targeting both CD20 and CD3, Tecentriq (atezolizumab), a monoclonal antibody designed to bind with PD-L1; and crovalimab, an anti-C5 antibody engineered to optimise complement inhibition. Our scientific expertise, combined with the breadth of our portfolio and pipeline, also provides a unique opportunity to develop combination regimens that aim to improve the lives of patients even further.ounced that

About RocheRoche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve peoples lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare a strategy that aims to fit the right treatment to each patient in the best way possible.

Roche is the worlds largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. More than thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the eleventh consecutive year, Roche has been recognised as one of the most sustainable companies in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2018 employed about 94,000 people worldwide. In 2018, Roche invested CHF 11 billion in R&D and posted sales of CHF 56.8 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit http://www.roche.com.

All trademarks used or mentioned in this release are protected by law.

References[1] Dornan D, et al. Therapeutic potential of an anti-CD79b antibody-drug conjugate, anti-CD79b-vc-MMAE, for the treatment of non-Hodgkin lymphoma. Blood 2009; 114:2721-2729.[2] Pfeifer M, et al. Anti-CD22 and anti-CD79B antibody drug conjugates are active in different molecular diffuse large B-cell lymphoma subtypes. Leukemia 2015; 29:1578-1586.[3] Ducry L, Stump B. Antibody-drug conjugates: linking cytotoxic payloads to monoclonal antibodies. Bioconjug Chem. 2010; 21:5-13.[4] ADC Review. What are antibody-drug conjugates? [Internet; cited December 2019]. Available from: https://adcreview.com/adc-university/adcs-101/antibody-drug-conjugates-adcs/.%5B5%5D Lyon, France. World Health Organization Classification of Tumors of Haematopoietic and Lymphoid Tissues. IARC Press; 2008.[6] Maurer, JM et al. Event-free survival at 24 months is a robust end point for disease-related outcome in diffuse large B-cell lymphoma treated with immunochemotherapy. J Clin Oncol 2014; 32:1066-73.[7] Numbers derived from GLOBOCAN 2018: Estimated cancer incidence, mortality and prevalence worldwide in 2018. [Internet; cited December 2019]. Available from: http://globocan.iarc.fr.

Roche Group Media RelationsPhone: +41 61 688 8888 / e-mail: media.relations@roche.com- Nicolas Dunant (Head)- Patrick Barth- Daniel Grotzky- Karsten Kleine- Nathalie Meetz- Barbara von Schnurbein

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European Commission approves Roche's Polivy for people with previously treated aggressive lymphoma - GlobeNewswire

Europe’s guardian of stem cells and hopes, real and unrealistic – Yahoo Singapore News

Poland has emerged as Europe's leader in stem cell storage, a billion-dollar global industry that is a key part of a therapy that can treat leukaemias but raises excessive hopes.

Submerged in liquid nitrogen vapour at a temperature of minus 175 degrees Celsius, hundreds of thousands of stem cells from all over Europe bide their time in large steel barrels on the outskirts of Warsaw.

Present in blood drawn from the umbilical cord of a newborn baby, stem cells can help cure serious blood-related illnesses like leukaemias and lymphomas, as well as genetic conditions and immune system deficits.

Polish umbilical cord blood bank PBKM/FamiCord became the industry's leader in Europe after Swiss firm Cryo-Save went bankrupt early last year.

It is also the fifth largest in the world, according to its management, after two companies in the United States, a Chinese firm and one based in Singapore.

Since the first cord blood transplant was performed in France in 1988, the sector has significantly progressed, fuelling hopes.

- Health insurance -

Mum-of-two Teresa Przeborowska has firsthand experience.

At five years old, her son Michal was diagnosed with lymphoblastic leukaemia and needed a bone marrow transplant, the entrepreneur from northern Poland said.

The most compatible donor was his younger sister, Magdalena.

When she was born, her parents had a bag of her cord blood stored at PBKM.

More than three years later, doctors injected his sister's stem cells into Michal's bloodstream.

It was not quite enough for Michal's needs but nicely supplemented harvested bone marrow.

As a result, Michal, who is nine, "is now flourishing, both intellectually and physically," his mum told AFP.

A cord blood transplant has become an alternative to a bone marrow transplant when there is no donor available, with a lower risk of complications.

Stem cells taken from umbilical cord blood are like those taken from bone marrow, capable of producing all blood cells: red cells, platelets and immune system cells.

When used, stem cells are first concentrated, then injected into the patient. Once transfused, they produce new cells of every kind.

At the PBKM laboratory, "each container holds up to 10,000 blood bags... Safe and secure, they wait to be used in the future," its head, Krzysztof Machaj, said.

The bank holds around 440,000 samples, not including those from Cryo-Save, he said.

If the need arises, the "blood will be ready to use without the whole process of looking for a compatible donor and running blood tests," the biologist told AFP.

For families who have paid an initial nearly 600 euros ($675) and then an annual 120 euros to have the blood taken from their newborns' umbilical cords preserved for around 20 years, it is a kind of health insurance promising faster and more effective treatment if illness strikes.

But researchers also warn against unrealistic expectations.

- Beauty products -

Haematologist Wieslaw Jedrzejczak, a bone marrow pioneer in Poland, describes promoters of the treatment as "sellers of hope", who "make promises that are either impossible to realise in the near future or downright impossible to realise at all for biological reasons."

He compares them to makers of beauty products who "swear their cream will rejuvenate the client by 20 years."

Various research is being done on the possibility of using the stem cells to treat other diseases, notably nervous disorders. But the EuroStemCell scientist network warns that the research is not yet conclusive.

"There is a list of almost 80 diseases for which stem cells could prove beneficial," US haematologist Roger Mrowiec, who heads the clinical laboratory of the cord blood programme Vitalant in New Jersey, told AFP.

"But given the present state of medicine, they are effective only for around a dozen of them, like leukaemia or cerebral palsy," he said.

"It's not true, as it's written sometimes, that we can already use them to fight Parkinson's disease or Alzheimer's disease or diabetes."

EuroStemCell also cautions against private blood banks that "advertise services to parents suggesting they should pay to freeze their child's cord blood... in case it's needed later in life."

"Studies show it is highly unlikely that the cord blood will ever be used for their child," the network said.

Story continues

It also pointed out that there could be a risk of the child's cells not being useable anyway without reintroducing the same illness.

Some countries, such as Belgium and France, are cautious and ban the storage of cord blood for private purposes. Most EU countries however permit it while imposing strict controls.

- Rapid growth -

In the early 2000s, Swiss company Cryo-Save enjoyed rapid growth.

Greeks, Hungarians, Italians, Spaniards and Swiss stored blood from their newborns with the company for 20 years on payment of 2,500 euros upfront.

When the firm was forced to close in early 2019, clients were left wondering where their stem cells would end up.

Under a kind of back-up agreement, the samples of some 250,000 European families were transferred for storage at PBKM.

The Polish firm, founded in 2002 with two million zlotys (around 450,000 euros, $525,000), has also grown quickly.

Present under the FamiCord brand in several countries, PBKM has some 35 percent of the European market, excluding Cryo-Save assets.

Over the last 15 months, outside investors have contributed 63 million euros to the firm, PBKM's chief executive Jakub Baran told AFP.

But the company has not escaped controversy: the Polityka weekly recently published a critical investigative report on several private clinics that offer what was described as expensive treatment involving stem cells held by PBKM.

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Europe's guardian of stem cells and hopes, real and unrealistic - Yahoo Singapore News

Pain treatment using human stem cells a success – News – The University of Sydney

Chronic pain cost an estimated $139.3 billion in 2018. Image: iStock, Top image: Pexels

Researchers at the University of Sydney have used human stem cells to make pain-killing neurons that provide lasting relief in mice, without side effects, in a single treatment. The next step is to perform extensive safety tests in rodents and pigs, and then move to human patients suffering chronic pain within the next five years.

If the tests are successful in humans, it could be a major breakthrough in the development of new non-opioid, non-addictive pain management strategies for patients, the researchers said.

Thanks to funding from the NSW Ministry of Health, we are already moving towards testing in humans, said Associate Professor Greg Neely, a leader in pain research at the Charles Perkins Centre and the School of Life and Environmental Sciences.

Nerve injury can lead to devastating neuropathic pain and for the majority of patients there are no effective therapies. This breakthrough means for some of these patients, we could make pain-killing transplants from their own cells, and the cells can then reverse the underlying cause of pain.

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Pain treatment using human stem cells a success - News - The University of Sydney

PRP/Stem Cell Treatment Has Both Functional and Cosmetic Benefits – Haute Living

Dr. Jeffrey Baker based his medical career on continual research and clinical spectrum in Regenerative Medicine since graduating from Medical school at NOVA Southeastern University College of Osteopathic Medicine in South Florida. Osteopathic Medicine emphasizes helping each patient achieve a high level of wellness and quality of life by focusing on health promotion. Haute Beauty catches up with Dr. Baker to learn more about PRP/Stem Cell Therapy.

Photo Credit: https://www.instagram.com/drjeffbaker/

PRP and Mesenchymal Stem Cell Treatment and Therapies are terms for regenerative medicine. Platelet Rich Plasma (PRP) is a concentrate of platelet-rich plasma protein derived from whole blood, centrifuged to remove red blood cells. This can then be injected back into the areas of injury or for cosmetic needs in order to increase cell turnover, promote recovery, as well as improve skins overall appearance and elasticity.

When PRP is used in conjunction with Mesenchymal Stem Cells, we are able to regenerate and restore normal function of musculoskeletal injuries without the need for surgery. This type of treatment works best for acute injuries such as tendinitis shoulder, elbow, wrist, hip, knees, and ankles. It is also best suited for chronic injuries, which are secondary pain injuries due to arthritis in shoulders, elbows, wrists, hands, fingers, lower back, hips, knees, ankles, and toes.

The process of PRP/Mesenchymal Stem Cell treatment begins by drawing blood from the patient and then centrifuging the blood in order to remove red blood cells. Once that process is complete, we combine what is now PRP with the Mesenchymal Stem Cells. This is then injected directly into the site of injury. Depending on the location of the injury, the use of an ultrasound may be useful in ensuring the accuracy of the injection site.

Remember, this type of treatment works best for people with tendinitis, tendon tears, ligamentous tears, and sprains/strains and also people with secondary pain due to arthritis.

These regenerative treatments of PRP and Mesenchymal Stem Cells are ideal for boosting general health. Many of my patients who come to me are looking for a way to reduce their daily aches and pains due to the natural aging process so that they are able to feel younger and live more active and fulfilling lifestyles. PRP and Mesenchymal Stem Cells can provide the relief these patients are seeking.

Cosmetically, PRP is used to treat conditions such as acne, acne scars, and melasma among other skin conditions. I often inject PRP to add plumpness to the face, under eyes, and cheeks. I also use PRP as a natural approach to increase cell turnover and elasticity in order to achieve a more youthful look. Then, for optimal benefit, I can micro-needle the PRP all over the face to give an overall glowing appearance.

The majority of my patients start to see results within the first two weeks. The results get increasingly better with time.

Remember, you have undergone regenerative injection treatments in or around injured and weakened ligaments, tendons or joints. The solution injected is designed to heal and strengthen these areas, improve stability and reduce pain. It is NORMAL and expected to experience some pain and swelling at the injection site for a few days after

treatment due to the expected inammatory response that starts the healing process. You will be able to resume light activity as soon as possible such as walking and stretching. It is important to keep the area treated mobilized, if pain tolerates, in order to encourage healing blood ow to the area. This will help to decrease some of your pain and will help improve your range of motion. Inactivity will decrease optimal results. It is vital that you adhere to activity recommendations after your procedure as improper or over strenuous activity may inhibit the healing process or result in re-injury.

I highly recommend my patients to continue to follow up care with not only myself but also with physical therapy and chiropractic treatment. In my experience, these practices used in conjunction with the PRP and Mesenchymal Stem Cell treatment, my patients are able to yield optimal results.

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PRP/Stem Cell Treatment Has Both Functional and Cosmetic Benefits - Haute Living

Gene Therapy for Sickle Cell Disease Receives Orphan Drug Designation – Monthly Prescribing Reference

Home News Drugs in the Pipeline

The Food and Drug Administration has granted Orphan Drug designation to ARU-1801 (Aruvant) for the treatment of sickle cell disease.

The investigational gene therapy is expected to increase functioning red blood cells through proprietary technology that inserts a modified fetal hemoglobin gene into autologous stem cells via a lentiviral vector. A phase 1/2 clinical study in 10 individuals with sickle cell disease is currently examining the efficacy and safety of ARU-1801.

For patients suffering from sickle cell disease, we believe the ultimate promise of gene therapy is a one-time cure without the side effect profile of high intensity myeloablative conditioning. We are committed to providing patients with that option and look forward to presenting more data on our Reduced Intensity Conditioning (RIC) approach, said Will Chou, MD, Chief Executive Officer of Aruvant.

Orphan Drug status is granted to new therapies that treat diseases impacting 200,000 individuals in the US.

For more information visit aruvant.com.

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Gene Therapy for Sickle Cell Disease Receives Orphan Drug Designation - Monthly Prescribing Reference

Cynata Therapeutics to talk stem cell therapies at Proactive"s CEO Sessions – Proactive Investors Australia

Cynatas managing director Dr Ross Macdonald will present at Proactives CEO Sessions in Sydney on February 3 and Melbourne on February 4.

() is focused on using its proprietary Cymerusplatform technology to develop commercially scalable cellular therapeutic products to treat serious chronic disorders.

The trademark Cymerus refers to the patented process of generating cell-based products from intermediate cells

At present, Cynata is focused on the production of mesenchymal stem cell (MSC)-based products using the Cymerus technology.

An example of an MSC product is CYP-001, targeting graft-versus-host-disease (GvHD) for which a Phase I clinical trial has been completed.

During 2019, Fujifilm did a licensing deal with Cynata for CYP-001 and GvHD for a US$3 million upfront payment and future payments of up to US$43 million.

Cynata continues to focus on early commercialisation of Cynatas Cymerus MSC products and is in active commercial discussions for numerous therapeutic targets.

During calendar year 2020, Cynata plans tobeprogressing to three Phase II trials in for GvHD (funded by Fujifilm), CLI, and osteoarthritis.

The company also continues to investigate the therapeutic potential in numerous additional target areas.

It is worth noting the Cymerus process has a number of key advantages including being only platform in the world able to produce commercial quantities of MSCs from a single source.

Five companies will present at the Sydney and Melbourne events, including (), () and ().

The Sydney session will also include ().

Register now for the CEO Sessions

Sydney event, Monday, February 3

Melbourne event, Tuesday, February 4

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Cynata Therapeutics to talk stem cell therapies at Proactive"s CEO Sessions - Proactive Investors Australia

High-Definition View of Diabetes-Related Proteins Obtained, Opening the Door to Future Treatments – SciTechDaily

GLP1R visualized in insulin-secreting beta cells at super-resolution. Credit: University of Birmingham

Scientists have examined a key receptor for the first time at high resolution broadening understanding of how it might function, and opening the door to future improvements in treating conditions such as type 2 diabetes.

Glucagon-like peptide-1 receptors (GLP1R) are found on insulin-producing beta cells of the pancreas and neurons in the brain. The receptor encourages the pancreas to release more insulin, stops the liver from producing too much glucose, and reduces appetite. This combination of effects can helps to control blood sugar levels.

As such, GLP1R has become a significant target for the treatment of type 2 diabetes, and a range of drugs are now available that are based on it. But much remains unknown about GLP1R function because its small size makes it difficult to visualize.

An international group of scientists led by experts at the University of Birmingham and the Max Planck Institute for Medical Research, Heidelberg, have now conducted a detailed examination of the receptor in living cells.

Researchers used a number of techniques including synthesis of marker compounds, immunostaining, super-resolution microscopy, as well as in vivo examination of mice. They were able to label GLP1R with their developed fluorescent probes so as to show its location in the cells and its response to signal molecules.

Publishing their findings inNature Communications, the researchers who were partly funded by Diabetes UK note that they now provide a comprehensively tested and unique GLP1R detection toolbox, which has updated our view of this receptor, with implications for the treatment of conditions such as obesity and type 2 diabetes.

GLP1R visualized in insulin-secreting beta cells at super-resolution. Credit: University of Birmingham

David Hodson, Professor of Cellular Metabolism, at the University of Birmingham, commented: Our research allows us to visualize this key receptor in much more detail than before. Think about watching a movie in standard definition versus 4k, thats how big the difference is. We believe this breakthrough will give us a much greater understanding of GLP1R distribution and function. Whilst this will not immediately change treatment for patients, it might influence how we design drugs in the future.

Johannes Broichhagen, Departmental Group Leader of the Max-Planck Institute for Medical Research, commented: Our experiments, made possible by combining expertise in chemistry and cell biology, will improve our understanding of GLP1R in the pancreas and the brain. Our new tools have been used in stem cells and in the living animal to visualize this important receptor, and we provide the first super-resolution characterization of a class B GPCR. Importantly, our results suggest a degree of complexity not readily appreciated with previous approaches.

Dr. Elizabeth Robertson, Director of Research at Diabetes UK commented: The effects of type 2 diabetes are serious and widespread, so finding more effective treatments to help people manage their condition and reduce their risk of its potentially devastating complications is absolutely vital.

Through innovative research like this, we can get to grips with key aspects of type 2 diabetes in unprecedented detail, and blaze a trail towards better treatments.

GLP1R is a member of the so-called G protein-coupled receptors (GPCRs), which play a role in many of the bodys functions. An increased understanding of how they work has greatly affected modern medicine, and today, it is estimated that between one-third and one-half of all marketed drugs act by binding to GPCRs.

Reference: Super-resolution microscopy compatible fluorescent probes reveal endogenous glucagon-like peptide-1 receptor distribution and dynamics by Julia Ast, Anastasia Arvaniti, Nicholas H.F. Fine, Daniela Nasteska, Fiona B. Ashford, Zania Stamataki, Zsombor Koszegi, Andrea Bacon4 , Ben J. Jones, Maria A. Lucey, Shugo Sasaki, Daniel I. Brierley, Benoit Hastoy, Alejandra Tomas, Giuseppe DAgostino, Frank Reimann, Francis C. Lynn, Christopher A. Reissaus, Amelia K. Linnemann, Elisa DEste, Davide Calebiro, Stefan Trapp, Kai Johnsson, Tom Podewin, Johannes Broichhagen and David J. Hodson, 24 January 2020, Nature Communications.DOI: 10.1038/s41467-020-14309-w

The research was funded by Diabetes UK, Medical Research Council (MRC) and European Research Council (ERC).

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High-Definition View of Diabetes-Related Proteins Obtained, Opening the Door to Future Treatments - SciTechDaily

Ncardia and BlueRock Therapeutics Announce Collaboration Agreement and Licensing of Process Development Technologies for the Manufacture of…

Ncardia and BlueRock Therapeutics today announced an agreement covering process development technologies for the manufacture of induced pluripotent stem cell (iPSC)-derived cardiomyocytes. Under the terms of the agreement, Bluerock gains access to Ncardias large-scale production processes and intellectual property for the production of iPSC-derived cardiomyocytes for therapeutic use.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200121005200/en/

"BlueRock is a leader in the field of cell therapy and our collaboration is a perfect match of mission and capabilities. This relationship allows us to utilize our experience in iPSC process development to help advance potential cell therapies for cardiac diseases," said Stefan Braam, CEO of Ncardia.

"There are hundreds of millions of people worldwide that suffer from degenerative cardiovascular disease where the root cause is the loss of healthy heart muscle cells, and where medical treatment options are limited. BlueRocks authentic cellular therapy is a novel approach that has the potential to transform the lives of patients, but will require the manufacture of our cell therapies at unprecedented scale. The Ncardia team has developed key technologies related to this scale-up challenge, and we are pleased to work with them as we advance BlueRocks novel CELL+GENE platform towards the clinic and those patients in need," said Emile Nuwaysir, President and CEO, BlueRock Therapeutics.

About BlueRock Therapeutics

BlueRock Therapeutics, a wholly owned and independently operated subsidiary of Bayer AG, is a leading engineered cell therapy company with a mission to develop regenerative medicines for intractable diseases. BlueRock Therapeutics CELL+GENE platform harnesses the power of cells for new medicines across neurology, cardiology and immunology indications. BlueRock Therapeutics cell differentiation technology recapitulates the cells developmental biology to produce authentic cell therapies, which are further engineered for additional function. Utilizing these cell therapies to replace damaged or degenerated tissue brings the potential to restore or regenerate lost function. BlueRocks culture is defined by scientific innovation, highest ethical standards and an urgency to bring transformative treatments to all who would benefit. For more information, visit http://www.bluerocktx.com.

About Ncardia

Ncardia believes that stem cell technology can deliver better therapies to patients faster. We bring cell manufacturing and process development expertise to cell therapy by designing and delivering human induced pluripotent stem cell (iPSC) solutions to specification. Our offerings extend from concept development to pre-clinical studies, including custom manufacturing of a range of cell types, as well as discovery services such as disease modelling, screening, and safety assays. For more information, visit http://www.ncardia.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200121005200/en/

Contacts

BlueRock:media@bluerocktx.com

Ncardia:Steven Dublinmedia@ncardia.com

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Ncardia and BlueRock Therapeutics Announce Collaboration Agreement and Licensing of Process Development Technologies for the Manufacture of...

Stem cell treatment off the table – Zululand Observer

Rhyah Govender with mom Pamela at her 7th birthday party last year

OWING to recurring infection and organ issues, Richards Bays Rhyah Sai Govender will no longer undergo stem cell treatment as planned in India.

The seven-year-old, diagnosed with cerebral palsy, was scheduled for treatment this month but recurrent urinary tract infections and problems with her kidneys have forced her family to explore other treatment avenues.

They have contacted the Bostons Children Hospital in the United States in an effort to find a panel of doctors who specialise in this condition.

Rhyahs mother, Pamela, said the family has overcome the initial disappointment and is now focused on finding another way forward.

While searching for a panel of local doctors who specialise in assisting children with cerebral palsy, a professor at one of the academic hospitals told us such a panel does not exist in our country.

Over the past seven years we have had our fair share of nightmare experiences with some doctors in the country, and we felt many used our daughter as a test subject only.

Despite the poor prognosis, Rhyah Sais family has never given up in their efforts to improve the quality of her life and she went on to defy the medical odds.

While stem cell therapy treatment will still be in the grand plan of things, acquiring a team of doctors who specialise in treating cerebral palsy children who can assess and draw up a treatment plan for Rhyah Sai, is without a doubt the best course of action.

I would like to thank everyone who helped and supported our fund-raising drives last year and for all the love and encouragement shown towards our family, said Pamela.

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UK biotech PhoreMost to work with Otsuka on gene therapy projects – – pharmaphorum

Building on an R&D tie-up with an Indian stem cell institution last year, UK biotech PhoreMost has begun a collaboration with Japans Otsuka working on several gene therapy projects.

Cambridge-based PhoreMost said it will use its next-generation phenotypic screening platform Siteseeker to identify novel targets for Otsukas therapeutics discovery programmes.

Novel targets identified will be further validated and characterised by Otsuka as part of its internal development pipeline, with an initial focus on gene therapy applications of identified targets.

Siteseeker looks at different protein shapes to find functionally active peptides that can be targeted by new therapies.

The technology looks at the entire proteome all of the proteins expressed in a live cell environment looking for druggable targets for a chosen disease.

Financial details of the agreement were not disclosed.

Dr Chris Torrance, CEO of PhoreMost, said: This collaboration with Otsuka is further recognition of the power of the Siteseeker approach to drive the identification of novel, druggable targets.

We are particularly excited to be exploring not only small molecule therapeutics but also gene therapy applications of our platform.

PhoreMost was one of two UK-based companies to receive funding from the government-backed agency Innovate UK to receive funding for small molecule research.

The 1 million funding was announced in 2018, and supported PhoreMost and the immune-oncology firm NeoPhore.

The companies won the funding as part of a competition organised by Innovate UK and funded by the UKs Biomedical Catalyst.

PhoreMost is also working with Indias Centre for Chemical Biology and Therapeutics, part of the Institute for Stem Cell Science and Regenerative Medicine (inStem).

The project began in July last year and, with funding from the Indian government, aims to create chemical tools that modulate novel classes of drug targets.

InStem is researching the genetic mechanisms of potency, differentiation and proliferation in human pluripotent cells.

It aims to examine diseases that can potentially be treated by stem cells.

Originally posted here:
UK biotech PhoreMost to work with Otsuka on gene therapy projects - - pharmaphorum