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

J&J wins another label expansion for Darzalex in Europe – PMLiVE

Johnson & Johnson, through its Janssen pharmaceutical division, has scored another label expansion in Europe for Darzalex in newly diagnosed multiple myeloma patients.

This time round, the newly approved formulation is comprised of anti-CD38 Darzalex (daratumumab) in combination with bortezomib, thalidomide and dexamethasone (VTd),a triple regimen which is commonly used in the treatment of multiple myeloma.

The European Commission has granted a marketing authorisation for the Darzalex-VTd combination as a treatment of newly diagnosed patients who are eligible for autologous stem cell transplant (ASCT). J&J was granted FDA approval for this Darzalex regimenlast September, in the same patient population.

The new expansion authorisation was based on results from J&Js phase 3 CASSIOPEIA trial, in which Darzalex and VTd significantly improved progression-free survival by 93% compared to 85% of those treated with VTd alone.

We asked ourselves can we improve the standard of care that is bortezomib, thalidomide and dexamethasone (VTd) to provide patients with valuable extra time? said Philippe Moreau, principal investigator and head of the haematology department at the University Hospital of Nantes, France.

The CASSIOPEIA study answered that question definitively, demonstrating that the addition of daratumumab in combination with VTd can lead to very deep remissions and also prolong PFS, he added.

The drug, which was originally developed by Danish biotech Genmab, is a monoclonal antibody (mAb) that targets the CD38 protein, which is highly expressed on multiple myeloma cells.

J&J has largely had the anti-CD38 category to itself since Darzalex was first approved in 2015, and has chalked up a number of label expansions during that time, importantly in the early stages of the disease.

This includes anothercombination regimen, with Celegenes Revlimid (lenalidomide) and dexamethasone (DRd), for the treatment of newly diagnosed multiple myeloma patients who are ineligible for ASCT.

This approval represents our commitment to investigate daratumumab in earlier disease stages of multiple myeloma and to develop more effective frontline treatment options for newly diagnosed patients who are eligible for transplantation, said Craig Tendler, vice president, clinical development and global medical affairs, oncology, Janssen Research & Development.

However, J&J could see its exclusivity disappear as Sanofis rival anti-CD38 mAb isatuximab is currently being reviewed by the FDA for the treatment of relapsed/refractory multiple myeloma with a target action date for a decision due on30 April.

J&Js expansion into the earlier settings will see it retain a large share of the market, but the encroachment of another therapy could still damage sales of the blockbuster therapy.

Originally posted here:
J&J wins another label expansion for Darzalex in Europe - PMLiVE

Mayo woman raising funds for niece, 4, who is losing her sight from three different eye conditions – Extra.ie

A Mayo woman is raising funds for her niece who is losing her sight from three different eye conditions.

Magdalena Krawiec, originally from Poland, wants to help her goddaughter Zuzia Krawiec to get the best care possible.

Zuzia, four, was born with an eye disease called congenital nystagmus, which can be caused with the eye itself or by a problem with the visual pathway from the eye to the brain.

Around four months after she was born, Zuzia was then diagnosed with hyperopia astigmatism.

The condition affects Zuzias sight so that objects close to her are out of focus.

This along with her mcular hypoplasia, which she was only diagnosed with recently, make it impossible for her to see anything in detail.

Her aunt told Extra.ie: The macular hypoplasia is the worst because theres not much we can do about it.

It affects the vision clarity and because of it she will never be able to see clearly. She can only see as far as one metre and its basically shadows and lights

Were working on her sight so that she will eventually be able to see 10 or 15 metres in front of her but thats going to be over many years.

Zuzia has to take medication and do exercises to help her eyes improve but shes also had to have surgeries.

Shes had one of ten surgeries so far and the family are looking into stem cell treatment, which more people are interested in in recent years.

The family hope to schedule her second operation soon as the masses are really loose behind her eye and they need to tighten those muslces.

This operation will held Zuzias eyeball become more steady and so help her look straight because right now theyre all over the place.

Theyre kind of shaky, Magdalena continued. Thats the problem.

Zuzia is currently in play school but once a week she goes to a special school where she does two hours of different exercises for children with disabilities.

One hour she learns how to use her hands to recognise objects and another hour Zuzia uses an optical enlarger, something her family hopes to buy her so she can see things better.

It will be able to help her see better as it makes pictures at least 50 times larger, Magdalena said.

Zuzia needs constant care, her aunt tells Extra.ie, so that she doesnt hurt herself as she walks around.

Magdalena said: She needs help going to the toilet or going outside, anything where there may be obstacles in her way.

She cant see so she could walk into a wall or something, she could hurt herself.

Zuzia will never have the same sight the average person has, but with the operations she may gain better sight.

Magdalena revealed that she and her family dont know the root of the problem and hope genetic tests will help them get an idea of what is going on.

The genetic tests have to be done through the national health service and can take two to three years, but another huge hit is the cost of the tests.

Its around 2,500, Magdalena said. Its ridiculous because shes young, we need to do it as quick as possible.

The brain is getting used to the eyes not working, so the quicker we get it fixed the better. We want her to actually see something.

Despite her eyesight, Zuzia is a happy child and a big sister to her baby brother.

However, Zuzia has started asking about her brothers sight, wondering: How come he can see and I cant?

Its heartbreaking, Magdalena admitted. Its hard to explain to her that her eyes are sick.

To help her family, Magdalena set up a GoFundMe page to raise money for Zuzias operation and her care, which you can donate to here.

Magdalena has also has a collection point in The Breadski Brothers store in Westport to help raise funds.

She really needs specialised care, especially during these early years in her development, to improve her chances of avoiding total blindness and giving her the childhood she deserves.

Read more here:
Mayo woman raising funds for niece, 4, who is losing her sight from three different eye conditions - Extra.ie

Global Circulating Tumor Cells (CTCs) and Cancer Stem Cells (CSCs) Market Predicted to Grow by 2028 with Competitive Situation and Business…

The research report on Global Circulating Tumor Cells (CTCs) and Cancer Stem Cells (CSCs) Market offers the effective objectives of the market share, growth aspects, and market segmentation. In addition, the report includes the detailed analysis for significant designing of innovative strategies for service providers. Likewise, this report also provides the collection of information which is gathered for qualitative methodologies as well as survey studied by an experienced analyst. Moreover, the Circulating Tumor Cells (CTCs) and Cancer Stem Cells (CSCs) market report includes the comprehensive details of the market comprising market trends, growth paths, market opportunities, limitations, challenges, and leading service providers of the respective market for the prediction period.

Request a sample of this report @ https://www.orbisresearch.com/contacts/request-sample/4155718

In addition, the Circulating Tumor Cells (CTCs) and Cancer Stem Cells (CSCs) market report delivers the fundamental analysis which is carried out by experienced analysts with a perspective of the Circulating Tumor Cells (CTCs) and Cancer Stem Cells (CSCs) industry. Furthermore, the Circulating Tumor Cells (CTCs) and Cancer Stem Cells (CSCs) market report also covers the structures of the industries across the globe. The research report is designed with the help of proficient standardized tools such as SWOT analysis and others. Additionally, the Circulating Tumor Cells (CTCs) and Cancer Stem Cells (CSCs) report offers a brief judgement of the global Circulating Tumor Cells (CTCs) and Cancer Stem Cells (CSCs) market. The Circulating Tumor Cells (CTCs) and Cancer Stem Cells (CSCs) industry report offers a broad estimation of the prediction period that will guide the users to take significant decisions over the forecasted chart. This report contain the overall analysis of the Circulating Tumor Cells (CTCs) and Cancer Stem Cells (CSCs) market.

Key Players Analysis:

JanssenQiagenAdvanced Cell DiagnosticsApoCellBiofluidicaClearbridge BiomedicsCytoTrackCelseeFluxionGilupiCynvenioOn-chipYZY BioBioViewFluidigmIkonisysAdnaGenIVDiagnosticsMiltenyi BiotecScreenCellSilicon Biosystems

Browse the complete report @ https://www.orbisresearch.com/reports/index/2013-2028-report-on-global-circulating-tumor-cells-ctcs-and-cancer-stem-cells-cscs-market-by-player-region-type-application-and-sales-channel

Market split by Type:

CellSearchOthers

Market split by Application:

Breast Cancer Diagnosis and TreatmentProstate Cancer Diagnosis and TreatmentColorectal Cancer Diagnosis and TreatmentLung Cancer Diagnosis and TreatmentOther Cancers Diagnosis and Treatment

Market segment by Region/Country including:

North America (United States, Canada and Mexico)Europe (Germany, UK, France, Italy, Russia and Spain etc.)Asia-Pacific (China, Japan, Korea, India, Australia and Southeast Asia etc.)South America Brazil, Argentina, Colombia and Chile etc.)Middle East & Africa (South Africa, Egypt, Nigeria and Saudi Arabia etc.)

Major Points from Table of Content:

Chapter 1 Market OverviewChapter 2 Market Segment Analysis by PlayerChapter 3 Market Segment Analysis by TypeChapter 4 Market Segment Analysis by ApplicationChapter 5 Market Segment Analysis by Sales ChannelChapter 6 Market Segment Analysis by RegionChapter 7 Profile of Leading PlayersChapter 8 Upstream and Downstream Analysis of Scar DressingChapter 9 Development Trend of (2019-2028)Chapter 10 Appendix

Place a purchase order of this report @ https://www.orbisresearch.com/contact/purchase-single-user/4155718

About Us:

Orbis Research (orbisresearch.com) is a single point aid for all your market research requirements. We have vast database of reports from the leading publishers and authors across the globe. We specialize in delivering customized reports as per the requirements of our clients. We have complete information about our publishers and hence are sure about the accuracy of the industries and verticals of their specialization. This helps our clients to map their needs and we produce the perfect required market research study for our clients.

Contact Us:

Hector CostelloSenior Manager Client Engagements4144N Central Expressway,Suite 600, Dallas,Texas 75204, U.S.A.Phone No.: +1 (972)-362-8199 ; +91 895 659 5155

Original post:
Global Circulating Tumor Cells (CTCs) and Cancer Stem Cells (CSCs) Market Predicted to Grow by 2028 with Competitive Situation and Business...

Michael Schumacher health update: Neurosurgeon claims F1 legend ‘nothing like we remember’ – Express.co.uk

The Formula One legend suffered a brain injury in December 2013 and has only just come out of an induced coma. Due to the amount of time since his injury, leading surgeon, Nicola Acciari, has warned the Formula One driver is now a different person.

The surgeon told Contro Copertina: "We must imagine a person very different from the one we remember on the track, with a very altered and deteriorated organic, muscular and skeletal structure.

"All as a result of the brain trauma he suffered."

Updates on the health of the racing legend have been kept to a minimum over the last few years.

What is known is that Michael Schumacher has been receiving groundbreaking treatment from French surgeon, Philippe Menasche.

Mr Menasche is a leading light in stem cell therapy and was reportedly using surgery to transfer cells from his heart to the brain according to Spanish publication 20minutos.

The treatment took place at the Georges-Pompidou Hospital and according to Schumacher's doctor, professor Jean-Francois Payen, he has returned this month for the same treatment.

He said: "There is a one to three-year plan for the regeneration period.

I visit Schumacher frequently and talk to his family about the progress I see.

JUST IN:Michael Schumacher latest: Fan club to mark anniversary of accident

"I saw the race together withMichael Schumacherat his home in Switzerland.

"Michael is in the best hands and is well looked after in his house.

"He does not give up and keeps fighting.

"His family is fighting just as much and of course our friendship cannot be the same as it once was just because there's no longer the same communication as before."

During his stem cell treatment, Mr Menasche was forced to defend himself over accusations he was using Schumacher as an experiment as opposed to a patient to operate on.

The professor at the University of Alabama and leader of the National Institute for Medical and Health research hit back and said: There was great attention to our department, but it has already returned to normal.

There has been great progress in the last 20 years, but the truth is that we still know little.

To mark the six-year anniversary of his injury, his wife Corinna, issued a message to his fans.

Continue reading here:
Michael Schumacher health update: Neurosurgeon claims F1 legend 'nothing like we remember' - Express.co.uk

Bone Therapeutics announces 2020 business outlook and reports year-end 2019 cash position – GlobeNewswire

Press release Regulated information

Phase III study of JTA-004 in patients with knee osteoarthritis on track to start patient enrollment early 2020

CTA submission initiated for Phase IIb with ALLOB in difficult tibial fractures

Two-year follow-up Phase IIa data for ALLOB in spinal fusion expected in H2 2020

Cash burn for the full year 2019 below guidance

Gosselies, Belgium, 22January 2020, 7am CET BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the leading biotech company focused on the development of innovative cell and biological therapies to address high unmet medical needs in orthopedics and bone diseases, today provides its business outlook for 2020 and reports its cash position for the year ending December31, 2019.

Outlook for 2020

Clinical Trial Application (CTA) for the knee osteoarthritis pivotal Phase III study with the Companys enriched protein solution, JTA-004, has been submitted to regulatory authorities in 5 European countries and Hong Kong Special Administrative Region, with patient enrollment expected to start early 2020. The JTA-004 PhaseIII study is a placebo and active-controlled, randomized, double-blind study to evaluate the potential of a single, intra-articular injection of JTA-004 to reduce osteoarthritic pain in the knee compared to placebo or Hylan G-F 20, the leading osteoarthritis treatment on the market. The first results of the study are anticipated in the second quarter of 2021 after afollow-up period of 3 months.

The Company is initiating the CTA submission process in Europe for a PhaseIIb clinical trial with its allogeneic bone cell therapy product, ALLOB, in patients with tibial fractures at risk for delayed healing, using its optimized production process. The first patients are expected to be enrolled in Q2 2020.

In the second half of 2020, the Company expects to report results from the 2-year follow-up period of thePhaseIIa study with ALLOB in patients undergoing a spinal fusion procedure following the successful completion of the 12-months period for which positive results were published in July 2019. These results demonstrated that ALLOB in addition to the standard of care procedure was well-tolerated and resulted in significant clinical and radiological improvements.

Miguel Forte, Chief Executive Officer of Bone Therapeutics commented: I am very pleased to see that we continue to make strong progress in executing on our business strategy to become a global innovator in bone related diseases. In the next coming months, we expect to run a pivotal trial with our novel protein solution, JTA-004, with the goal to provide relevant regulatory and clinical evidence about the potential of JTA-004 as a treatment option for the many patients suffering from knee osteoarthritic pain. The anticipated start of the PhaseIIb study in patients with difficult to heal fractures and the 24months results from the PhaseIIa trial in patients undergoing a spinal fusion procedure, which we expect later this year, could further underscore the unique potential of ALLOB to bring much needed innovation to the treatment of severe bone disorders."

Cash position for the full year ended December 31, 2019

The continued strong focus on cash management resulted in a net cash utilization of 11.5million(1) for the full year 2019 which was below the Companys guidance of 12-13million. The net cash position totaled 8.6million(1) for the year ended December 31, 2019. The Company anticipates having sufficient cash to carry out its business objectives into Q3 2020.

Following the previously announced discontinuation of the autologous cell therapy program, PREOB, end 2018, Asahi Kasei and the Company agreed to formally end the PREOB licensing agreement. As a result, Bone Therapeutics and the Walloon Region agreed to terminate the related reimbursable grant agreements under the form of Recoverable Cash Advances, thereby reducing cash reimbursements and associated interest payments that were due by the Company by 1.4M over the next 5 years.

Financial Calendar 2020

The financial calendar is communicated on an indicative basis and may be subject to change.

(1) Unaudited number

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and bone diseases. The Company has a broad, diversified portfolio of bone cell therapies and an innovative biological product in later-stage clinical development, which target markets with large unmet medical needs and limited innovation.

Bone Therapeutics is developing an off-the-shelf protein solution, JTA-004, which is entering Phase III development for the treatment of pain in knee osteoarthritis. Positive Phase IIb efficacy results in patients with knee osteoarthritis showed a statistically significant improvement in pain relief compared to a leading viscosupplement. The clinical trial application (CTA) to start the pivotal Phase III program has been submitted to the regulatory authorities in Europe and the trial is expected to start in Q1 2020.

Bone Therapeutics other core technology is based on its cutting-edge allogeneic cell therapy platform (ALLOB) which can be stored at the point of use in the hospital, and uses a unique, proprietary approach to bone regeneration, which turns undifferentiated stem cells from healthy donors into bone-forming cells. These cells can be administered via a minimally invasive procedure, avoiding the need for invasive surgery, and are produced via a proprietary, scalable cutting-edge manufacturing process. Following the promising Phase IIa efficacy and safety results for ALLOB, the Company has started the CTA submission procedure with the regulatory authorities in Europe to start the Phase IIb clinical trial with ALLOB in patients with difficult-to-heal fractures, using its optimized production process.

The ALLOB platform technology has multiple applications and will continue to be evaluated in other indications including spinal fusion, osteotomy and maxillofacial and dental applications.

Bone Therapeutics cell therapy products are manufactured to the highest GMP (Good Manufacturing Practices) standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. The Company is based in the BioPark in Gosselies, Belgium. Further information is available at http://www.bonetherapeutics.com.

Contacts

Bone Therapeutics SAMiguel Forte, MD, PhD, Chief Executive OfficerJean-Luc Vandebroek, Chief Financial OfficerTel: +32 (0) 71 12 10 00investorrelations@bonetherapeutics.com

International Media Enquiries:Consilium Strategic CommunicationsMarieke VermeerschTel: +44 (0) 20 3709 5701bonetherapeutics@consilium-comms.com

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: + 33 (0)1 44 71 94 94bone@newcap.eu

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors` current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such person`s officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

See original here:
Bone Therapeutics announces 2020 business outlook and reports year-end 2019 cash position - GlobeNewswire

Science Talk – Tell me more about telomeres: how ‘basic’ science can help us treat cancer – The Institute of Cancer Research

Image: Chromosomes and their telomeres (visualised in red). Credit: Thomas Ried, NCI Center for Cancer Research

You might not have heard of telomeres but theyre incredibly important they are the caps that protect the end of chromosomes. They work like the plastic tips that stop your shoelaces from fraying.

All cancers alter telomeres in order to survive, so by doing basic research to try to understand how telomere replication and processing works, Max and his team hope to identify possible new ways to target and treat cancer.

Having joined the Division of Cancer Biology in October 2019, Dr Max Douglasis now one of the newest Team Leaders at the ICR. I met him at our Chester Beatty Laboratories in Chelsea, where he told me more about his work.

Max studied for his PhD in biochemistry and cell biology at the University of Cambridge. He then joined Dr John Diffleys team in Londons Clare Hall Laboratories which later became part of the Francis Crick Institute where he focused on studying the early stages of DNA replication.

At the Crick, he helped establish in detail how a protein complex called the CMG replicative helicase that helps unwind DNA during replication, is assembled and activated.

Now at the ICR, Max leads his own research team studying DNA replication but in the context of telomeres and cancer.

My main project is to rebuild telomeres in the lab and then unpick how they work how they are replicated and how they are processed. This knowledge is generally useful, but we will focus on studying it in the context of cancer, explained Max.

Lets finish it:help us revolutionise cancer treatment. We aim to discover a new generation of cancer treatments so smart and targeted, that more patients will defeat their cancer and finish what they started.

Support our work

When a cell becomes cancerous, it divides more often and every time it divides, its telomeres become shorter and shorter. Once there is no telomere left, the DNA unravels, like a shoelace fraying, and the cell dies. This eventually happens in most healthy cells telomeres shorten over time until cell division is no longer possible, leading to cell death.

While this loss of telomere protection can cause cancer cells and healthy cells to die, it can also lead to a state of genome instability that helps cancer survive and spread.

We also know that cancer cells can escape death by making telomerase, an enzyme that prevents telomeres from getting short. Certain cells in our body, such as stem cells, are able to divide over and over again thanks to telomerase. Cancer cells take advantage of this enzyme and hijack it to maintain telomere length which enables them to continue to divide and spread.

In other words, telomeres seem to play a role in the death of cancer cells but theyre also crucial for their survival. However, the molecular steps that guide telomere replication and processing remain poorly understood.

By using genetics and replicating cellular processes in a test tube, through a technique known as reconstitution biochemistry, Max and his team hope to better understand how telomeres are processed, and how they are inherited from one generation of cells to the next.

If Max and his team can dissect how telomeres work and clarify their link to cancer, maybe well figure out new ways to treat it.

His research might seem quite distant from the clinic, but Max knows he belongs at the ICR, which has an exemplary track record in making discoveries that ultimately benefit people with cancer.

I really value the ICRs commitment to doing basic, laboratory science. Good basic science is necessary to understand cancer, and the ICR values that. Here, I can figure out how to use my findings to benefit people, and that, in turn, will also hugely benefit my work, Max said.

I feel very lucky to work at an institution with a mission, being able to do what I love while getting opportunities to make discoveries that could help people.

As a new Team Leader, Max is currently the only member of his team but a higher scientific officer will be joining this month, as well as a post-doctoral training fellow, who will be joining in March. They will also start recruiting for a PhD student. As he told me, he cant wait for the new team members to join him in January.

Im excited to supervise other people for the first time. I want to build a strong team and a good environment for them to thrive in.

See original here:
Science Talk - Tell me more about telomeres: how 'basic' science can help us treat cancer - The Institute of Cancer Research