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A predator’s world? Top analyst sees the ‘haves’ and the ‘haven’ts’ diverge as biotech bubbles form and collapse – Endpoints News

Count Pfizer in as a top player in the blockbuster game of JAK1 inhibitors.

Over the weekend the pharma giant posted some stellar Phase III efficacy data for their heavyweight contender abrocitinib in atopic dermatitis (eczema) that lines up ahead of a booming Dupixent (dupilumab), a blockbuster in the portfolios of Regeneron and Sanofi. And they put some real distance ahead of Eli Lillys trailing Olumiant, which made a delayed initial arrival on the market for rheumatoid arthritis after the FDA hobbled it with some additional hurdles on safety concerns.

JADE-MONO-1 scores well for Pfizer, teeing up what will be an intensely followed breakdown of the JADE MONO-2 data, which the pharma giant recently top-lined as similar to the first Phase III when tested against a placebo a control group that has been easily outclassed by all the drugs in this market niche.

As of now, Pfizer looks to be equipped to run into the review stage advantaged by a breakthrough therapy designation that is intended to speed up the regulatory process.

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A predator's world? Top analyst sees the 'haves' and the 'haven'ts' diverge as biotech bubbles form and collapse - Endpoints News

The global wound care market is growing at a CAGR of over 5% during the forecast period20182024 – Yahoo Finance

The adoption of wound care biologics is augmenting the growth of the global wound care market. The commercial availability of a wide array of wound biologics is likely to encourage many end-users to use them in treating wounds as they are clinically proven, safe, and effective than other products.

New York, Oct. 14, 2019 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Wound Care Market - Global Outlook and Forecast 2019-2024" - https://www.reportlinker.com/p05822880/?utm_source=GNW The growing incidence of infections caused in lesions is another factor accelerating the growth of anti-microbial dressings market segment. Anti-microbial agents such as chlorhexidine, maggots, silver, iodine, and honey are increasingly becoming important in the global wound care market. Therefore, the incorporation of anti-microbial agents in wound dressing products is improving clinical outcomes for the treatment of wounds, thereby driving the global wound care market.

The following factors are likely to contribute to the growth of the wound care market during the forecast period: Growing Focus on the Development and Commercialization of Wound Biologics The emergence of Stem Cell Therapy for Wound Healing New Product Approvals/Launches Increasing Number of Surgical Procedures

This research report on the global wound care market covers sizing and forecast, market share, industry trends, growth drivers, and vendor analysis. The market study includes insights on segmentation by products (advanced wound care products, sutures and stapling devices, traditional wound care products, and hemostats and surgical sealants), wound type (acute wound and chronic wound), end-users(hospitals and specialty wound care clinics, long-term care facilities, home healthcare,and others), and geography (North America, APAC, Europe, Latin America, and MEA).

The study considers the present scenario of the wound care market and its market dynamics for the period 2018?2024. It covers a detailed overview of several market growth enablers, restraints, and trends. The study covers both the demand and supply aspect of the market. The report profiles and examines leading companies and other prominent companies operating in the wound care market.

Wound Care Market: SegmentationThis research report includes detailed market segmentation by products, wound type, end-users, and geography. The increase in the geriatric population is a major contributing factor for the growth of the advanced wound care segment as the prevalence of diabetes and other diseases is more common in the elderly age group than youth. The advanced segment is also growing as the majority of market players are offering innovative products to meet the demand for wound care worldwide. The rising incidence of diabetes and associated diabetic foot ulcers in the elderly population globally is fueling steady growth for traditional products. The market is also growing steadily as products such as gauze bandages and adhesive bandages witness sustainable demand for small cuts, bruises as well as for chronic wounds and burns, especially in developing countries. Developing regions such as Africa, Asia, and Latin America are the largest contributors to the traditional products.

The acute wound market is growing mainly due to the rise in surgical site infections (SSI) and the increase in the number of burn cases worldwide. Chronic wounds do not heal through the normal healing process. The segment is growing due to the growing burden of diabetic foot ulcers, venous leg ulcers, pressure ulcers, and some surgical site infections that do not heal naturally or with medicines. Further, about 70% of lower-extremity ulcers are caused by chronic venous insufficiency. People aged 65 years or above are vulnerable to venous ulcers. The overweight and obese population also develop a high risk for diabetes and associated chronic wounds. Therefore, the growing incidence of several chronic wounds is driving the growth of the segment.

The shift from traditional lower technology wound care treatments to the adoption of advanced treatments is a major factor for the high share of the hospitals and specialty wound clinic segment. Long-term care facilities segment is growing at a steady pace because of the growing incidence of chronic wounds due to the increase in chronic diseases such as diabetes. The growing elderly population is contributing to the growth of the segment as they are more prone to chronic diseases.

Market Segmentation by Products Advanced Wound Care Products Sutures and Stapling Devices Traditional Wound Care Hemostats and Surgical SealantsMarket Segmentation by Wound Type Acute Wounds Chronic WoundsMarket Segmentation by End-users Hospitals and Specialty Wound Care Clinics Long-term Care Facilities Others (ASCs, solo practitioners, and other acute care settings) Home Healthcare

Wound Care Market: Geography

The growth in the number of people suffering from several acute and chronic wounds in the US is the primary factor for the high market share of the North American market. The rise in the geriatric population is another key factor driving the market growth of the wound care market in the region.Germany, France, the UK, Italy, and Spain are witnessing significant growth in the wound care market. Factors such as the presence of highly sophisticated healthcare infrastructure, the increase in the patient population, and high awareness regarding the availability of advanced treatment options are the major growth factors.

Market Segmentation by Geography North Americao USo Canada APACo Japano Chinao Indiao South Koreao Australia Europeo Germanyo Franceo UKo Spaino Italy Latin Americao Brazilo Mexicoo Argentine MEAo Turkeyo Saudi Arabiao South Africao Iran

Key Vendor AnalysisThe global wound care market is highly competitive and dynamic characterized by the presence of several global, regional, and local vendors, offering a diverse range of products and devices for treating various acute and chronic wounds. There are approximately more than 400 vendors providing a wide array of wound care products worldwide. Multiple product launches, strategic acquisitions, and differentiated products have fueled the growth in recent years. New product launches and strategic acquisitions, collaborations will be crucial for companies to maintain revenue growth in the coming years. Large and diversified companies account for nearly 48% of the market share. Also, the majority of key players have demonstrated consistent growth in the last three years. Moderate to the high growth of major players will continue to boost the market growth. In addition, both established and emerging players are developing or commercializing wound care products and devices. Major players are focusing on technological innovations, thereby investing substantial amounts on R&D activities for offering advanced wound care products such as advanced wound dressings, skin grafts, and wound biologics.

Key Vendors 3M Acelity Cardinal Health Coloplast ConvaTec Medtronic Mlnlycke Health Care PAUL HARTMANN Smith & Nephew

Prominent Vendors Acell Adhesys Medical Advanced Medical Solutions Group Advancis Medical AediCell AlloSource AMERX Health Care AOTI AxioBiosolution Baxter B. Braun Melsungen BD BSN Medical BTI Biotechnology Institute Carilex Medical Cohera Medical CONMED Cork Medical Covalon Technologies CryoLife DeRoyal Industries Ethicon Grena Hollister Integra LifeSciences Intuitive Surgical LOHMANN & RAUSCHER (L&R) Medline Industries MiMedx MPM Medical MTF Biologics Ocular Therapeutix Organogenesis Osiris Therapeutics Pters Surgical Purple Surgical Reach surgical Sealantis Sechrist Sonoma Pharmaceuticals Sutures India Synovis MCA Talley Group TRIAGE MEDITECH Tricol Biomedical URGO Group Vivostat Waston Medical Appliance

Key Market InsightsThe report provides the following insights into the wound care market for the forecast period 20192024. Offers market sizing and growth prospects of the wound care market for the forecast period 20192024. Provides comprehensive insights on the latest industry trends, market forecast, and growth drivers in the wound care market. Includes a detailed analysis of market growth drivers, challenges, and investment opportunities. Delivers a complete overview of market segments and the regional outlook of the wound care market. Offers an exhaustive summary of the vendor landscape, competitive analysis, and key market strategies to gain a competitive advantage in the market.Read the full report: https://www.reportlinker.com/p05822880/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Clare: clare@reportlinker.comUS: (339)-368-6001Intl: +1 339-368-6001

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The global wound care market is growing at a CAGR of over 5% during the forecast period20182024 - Yahoo Finance

UK biotech startup Mogrify injects $16M to get novel cell therapies to market soon – TechCrunch

Cambridge, UK-based biotech startup Mogrify, which is working on systematizing the development of novel cell therapies in areas such as regenerative medicine, has closed an initial $16 million Series A.

The raise from investors Ahren Innovation Capital,Parkwalkand24Haymarketfollows a $4M seed in February taking its total raised to date to $20M.

Put simply, Mogrifys approach entails analysis of vast amounts of genomic data in order to identify the specific energetic changes needed to flip an adult cell from one type to another without having to reset it to a stem cell state with huge potential utility for a wide variety of therapeutic use-cases.

What were trying to do with Mogrify is systematize that process where you can say heres my source cell, heres my target cell, here are the differences between the networks and here are the most likely points of intervention that were going to have to make to drive the fate of an adult cell to another adult cell without going through a stem cell stage, says CEO and investor Dr Darrin Disley.

So far he says its successfully converted 15 cells out of 15 tries.

Were now rapidly moving those on through our own programs and partnership programs, he adds.

Mogrifys business has three main components: Internal program development of cell therapies (current cell therapies its developing include enhancing augmented cartilage implantation; non-invasive treatment of ocular damage; and for blood disorders). Its also developing a universal source of cells for use in immunotherapy to act as disease-eaters, asDisley puts it.

Speculative IP development is another focus. Because of the systematic nature of the technology were in a position very rapidly to identify areas of therapy that have particular cell conversions at their essence and then drive that IP generation around those cells very quickly and create an IP footprint, he says.

Partnering deals is the third piece. Mogrify is also working with others to co-develop and bring targeted cell therapies to market. Disley says its already closed some partnerships, though its not announcing any names yet.

The startup is drawing on around a decades worth of recent work genomics science. And specifically on a data-set generated by an international research effort, called Fantom 5, which its founders had early access to.

We started with that massive Fantom data-set. Thats the baseline, the background if you like. Think of it like two cities in America: Chicago and New York. Theres your source cell, theres your target cell. And because you have all the background data of every piece of the network every building, every skyscraper if you look at the two you can identify the difference in the gene expression, therefore you can identify which factors will regulate a wide array of those genes. So you can start identifying the differences between the two, explainsDisley.

Weve then added to that massive data sets in DNA-protein and protein-protein interactions so you start to now overlay all of that data. And then weve added on top of that new next-gen sequencing data and epigenetic data. So youve now got this massive data-set. Its like having a network map between all the different cell types. So youre therefore then able to make predictions on how many interventions, what interventions are needed to drive that change of state and its systematic. It doesnt just recommend one set. Theres a ranking. It can go down to hundreds. And there is some overlap and redundancies, so for example if one youre preferred thing doesnt work the way you wanted it to you can go back and select another.

Or if theres an IP issue around that factor you can ignore that piece of the network and use an alternative route. And once youve got to your target cell, if it needs to some tweaking you can actually re-sequence it and take that back and thats your starting cell again. And you can go through this optimization process. So what comes out at the other end youve got a patent that it like a small molecule composition of matter patent; its the therapeutic. So youre not coming out with the target, youre actually coming out with here is the composition of matter on the cell.

In terms of timeframe for getting novel cell therapies from concept to market Disley suggests a range of between four and seven years.

Once youve identified the cell type that can be be the basis of your GMP manufacturable process and then you can tweak that to take it to the therapeutic indication you can develop a cell therapy and bring that to market in five years, he says. Its not like the old days with small molecules where it can take ten, 15, 20 years to get a serious therapy on the market.

When youre treating patients is because there are no other treatments for them, when you go into phase two and do your safety study [and] efficacy study youre actually treating patients already in terms of their disease. And if you get it right you can get a fast track approval. Or a conditional approval so that you may not even have to do a phase 3 [testing].

Were not using any artificial intelligence here, he also emphasizes, pointing to his experience investing in companies in the big extreme data space which he argues do best by using unbiased approaches.

AI I think is still trying to find its way, he continues. Because in its essence it will be able to get to answers with smaller amounts of data but its only as good as the data you train it on. And the danger with AI it just learns to recognize what you want it to recognize. It doesnt know what it doesnt know.

In combination, once you continue to generate this massive cell network data etc you can start applying aspects of machine learning and AI. But you couldnt do Mogrify with AI without the data. You have to do it that way. And the data is so complex and combinatorial 2,000 transcription factors, in terms of regulation of those genes, they then interact in network to do the protein-protein interactions, youve got epigenetic aspects of that, you could even start adding cell microbiome effects to that later so youve got a lot of factors that could influence the phenotype of the cell thats coming out the other end.

So I think with AI you have to be a little careful. I think it will be a more optimizing tool once youve got sufficient confidence in your system.

The plan for the Series A funding is to ramp up Mogrifys corporate operations and headcount including bringing in senior executives and expertise from industry as well as spending to fund its therapy development programs.

Disley notes its recent appointment of Dr Jane Osbourn as chair as one example.

Were bringing in more people with a lot of cell therapy experience from big pharma, around then more on the manufacturing and delivery of that so really building so that were not just a tech company, he says. Weve very strong already, were already 35 people on the tech and early stage drug discovery side were going to add another 30 to that. But thats going to be increasingly more people with big pharma, cell therapy development, manufacturing experience to get products on to market.

Partner search is another focus for the Series A. Were trying to find the right strategy partners. Were not doing services, were not doing products so we want to find the right strategic partners in terms of doing multi-programs in a partnership, he adds. And then a series of more tactical deals where people have got a specific problem with a cell conversion. These more turnkey deals, if you like. We still get up-fronts, milestones and royalties but theyre smaller.

Despite now having enough money for the next two to two and half years its also leaving the Series A open to continue expanding the round over the next 12 months up to a maximum of another $16M.

We have so many interested investors, Disley tells us. This round we didnt actually open our round. We did it with internal investors and people were very close to who weve worked with before, and there were investors lining up [so] we are leaving it open so that in these next 12 months we may choose to increase the amount we bring in.

It would be a maximum of another $16M if it was an A round but we may decide just to go straight forward if we progress very fast to a much bigger B round.

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UK biotech startup Mogrify injects $16M to get novel cell therapies to market soon - TechCrunch

Ridgefield woman, her mother write book about battle with MS – The Columbian

It hurt Margaret Godfrey to see daughter Rachel Carter in pain, so she painted.

And once the worst pain had passed, Carter needed a way to move forward, so she wrote.

In 2000, at age 24, Carter was diagnosed with multiple sclerosis, a disease where the immune system eats away at the lining of nerves and impairs or disables the brain and central nervous system.

Carter is now a married 43-year-old mother of three who lives in Ridgefield. But when she was diagnosed with MS, it marked a significant shift in her life. The woman who ran marathons, rode a motorcycle to high school and was once a deckhand on a small riverboat cruise ship to prove a point because she was told a woman couldnt hold that job, slowly morphed as her disease altered life.

It started with a fall on vacation during a run. Then falling on runs became more frequent. Gone went the marathons. In came the treadmill. Slowly, over the course of 12 years, the MS progressed and symptoms became debilitating. Running left completely. Neuropathic pain caused her to feel like she was sunburned in the morning, and by the afternoon or evening it felt like third degree burns. Carter left her job in sales in 2013.

I would always end up in tears in the bed, Carter said.

Carter has chronicled her battle with MS, and a stem cell treatment she credits with turning her prognosis around, in a new book called Enduring the Cure: My MS Journey to the Brink of Death and Back. Her mother helped her edit the book, and provided the books cover art. Adrienne van Der Valk also edited the book.

Through pain, Carter and her mother have created art. About five years ago, Carter underwent an experimental stem cell transplant in Seattle to help her rebuild her immune system. Carter lived in Seattle for about three months, while her parents watched two of her children, and the other child lived with an aunt.

As part of the procedure, Carters own stem cells were harvested and then reintroduced to her body after chemotherapy depleted most of her immune system. Its what she described as a rebooting of her whole immune system, in hopes that it would stop attacking her brain and spinal cord. Carter still has her previous brain damage, which impacts her memory and brain power; but so far, the stem cell transplant has improved her condition greatly, she said.

Carter has more energy she used to sometimes not be able to leave bed some days. She also can stand upright and walk easily she used to rely on a walker. And her pain is minimal now, the biggest improvement.

Carters family kept notes during her treatment, and Carter decided to use those, and the memories of her family, to help her write the book with van Der Valk.

Thinking was very fatiguing for Carter, since she still has the brain damage from MS. She could only work about one to two hours on the book at a time. She said writing was frustrating and hard in many ways. Parts of my brain are gone, Carter explained. She said it requires her about 10 times as much brain matter as the average person to process something simple.

Its so much pressure to have something you feel like you have to do, and then you cant do it. I had so little energy, Carter said. I cant explain very well because so many words dont come to me.

In spite of the challenges, Carter feels like sharing her experience will help others because, in life, we all have hard situations, she said.

Godfrey, a 70-year-old painter who lives in Blue River, Ore., found art in the familys experience. She drew inspiration from Gustav Klimt, an Austrian symbolist, who painted in the late 1800s and early 1900s, and created symbolic paintings of her daughter. Godfrey said art makes her an intellectual because of how she has to think about what shes painting. This time she was researching her own child.

Godfrey did a painting of Carter surrounded by old running shoes, a life she had to give up. She did a painting of Carter without her hair, which centered on Carters worries. She did a painting of Carter covered by a quilt with her three kids on it, what kept her going and allowed her to recover. She did a painting of Carter lying in a hospital bed, looking exhausted and resigned, which is the books cover. The final painting in the series, called The Journey Is Not Done, features Carter with hair, months after treatment. It has an unfinished mosaic because Carters journey isnt over.

Creating art is a very meditative thing, Godfrey said. Rather than getting wrapped up in emotions, once I start painting the world is me and paper. All the worries of life disappear. It was a way to enjoy that experience of just being able to throw out my expression, without getting too emotionally tangled up.

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Ridgefield woman, her mother write book about battle with MS - The Columbian

How the bodys cells can prevent the need for surgery – ABC 4

Posted: Oct 8, 2019 / 09:45 AM GMT-0600 / Updated: Oct 10, 2019 / 01:15 PM GMT-0600

Stem cell therapy is one of the most successful procedures ever for solving joint pain. Your own bodys cells are injected back into the area where healing is needed: back, neck, hip, shoulder, etc. The new cells signal to body the need for regeneration, which starts to take place.

The treatment is a one-and-done injection, no surgery. Regenerative Medicine of Utah successfully treats many with stem cell therapy. One patient, John Dunn, was scheduled for knee replacement because of bone on bone pain. Before surgery he received an injection. Three months later and regenerated tissue was found in his knee joint. Six months later his knee was even better than that.

In just four weeks John was out of pain, but the body wasnt done healing. The cells continue signaling to the body the need for healing. Its usual to allow 12 months for the regeneration process to complete and reach its limit.

Making sure youre a candidateis important. Not everyone needs it. Many patients that end up being a candidateinitially thought they werent eligible. To know if youre a candidate schedulea consultation.

Right now, at Regenerative Medicine ofUtah an evaluation is $49 for the first 25 callers to (800)453-0286.

This article contains sponsored content.

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How the bodys cells can prevent the need for surgery - ABC 4

ReNeuron cell-based treatment hailed by leading non-profit group funding research into treatments for retinal degenerative diseases – Proactive…

Benjamin Yerxa, chief executive of the Foundation Fighting Blindness, said results from a recent clinical trial represented a major step forward for sufferers of a degenerative disease called retinitis pigmentosa

A leading non-profit group funding research into treatments for retinal degenerative diseases has hailed as major step forward a potential therapy developed by ().

The comments from Benjamin Yerxa, chief executive of the Foundation Fighting Blindness, followed the presentation of data from a phase I/IIa clinical study by trial investigator Pravin Dugel.

He has been working on ReNeurons human retinal progenitor (hRPC) stem cell line, which has shown early promiseimproving, or stabilising clarity of vision in people with the degenerative disease retinitis pigmentosa.

Dugel's address to the American Academy of Ophthalmology Annual Meeting in San Francisco was based on the latest results from the clinical assessment of the drug candidate, published on October 2.

After the presentation, Yerxa said: "We're excited by the progress of ReNeuron's hRPC therapy.

From the Foundation's perspective, any gain in vision, or even stabilisation, is a major step forward for patients with RP as currently it is a condition where progressive loss of vision leads to blindness."

Earlier this month ReNeuron said it had seen rapid and profound results in the first three patients of the second phase of clinical studies of its human retinal progenitor cells.

The phase IIa trials saw noticeable improvements in visual acuity as measured by the number of letters that could be read on a standard eye chart.

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

With 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 three months after treatment.

I believe that we are seeing a clear signal of efficacy in this patient population where any gain in vision, let alone the levels seen in some of these patients, is so hard to come by and so very much appreciated," said Dr Pravin Dugel, managing partner at Retinal Consultants of Arizona and clinical professor at the Roski Eye Institute in Los Angeles.

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ReNeuron cell-based treatment hailed by leading non-profit group funding research into treatments for retinal degenerative diseases - Proactive...

Gay Couple Adopt HIV-Positive Child After She Was Rejected By Ten Other Families – UNILAD

A gay couple have spoken out about the immediate connection they felt after adopting a HIV-positive child who had been rejected by 10 other families.

Damian Pighin, 42, and Ariel Vijarra, 39, were the first gay couple to get married in the north-eastern Argentine province of Santa Fe, and made the decision to expand their family in 2011.

The married couple, from Rosario, had several years of disappointment before receiving a call in 2014 about a newborn baby who was ready to be adopted.

The baby girl, who was born with HIV, was 28 days old when Damian and Ariel received the call and were informed 10 families had previously rejected her.

However, they did not hesitate to accept her into their family, with Ariel telling local media:

As soon as I saw her, I felt that she was part of my life. The connection was immediate. We held her in our arms, gave her the bottle and she looked at us with her eyes wide open without crying.

The little girl, who has been named Olivia, responded very well to her HIV treatment and she immediately started to gain weight. A couple of years ago, the family received brilliant news when tests showed the virus was no longer detectable in Olivia.

The family grew by one more in 2015, when a woman who heard about the couples story wanted them to adopt her baby girl. They did, and so Damian and Ariel welcomed Victoria into their lives.

The two siblings are similar in age, with Olivia turning five years old later this month and Victoria celebrating the same birthday in February.

Ariel and Damien work for non-governmental organisation Acunar Familias, which roughly translates to Cradle Families, helping couples adopt unwanted children.

Although there is currently no cure for HIV, medical treatment is available that significantly reduces the amount of the virus in the body. As per Medical News Today, this can be to the point the virus becomes undetectable in the blood.

An undetectable viral load means the person living with HIV is no longer infectious and the virus is no longer able to damage their immune system.

Earlier this year, a third person appeared to have been cured from the virus when they underwent a stem cell transplant to treat cancer, rather than HIV. The transplant involved killing most of their immune cells with radiotherapy or drugs, and then replacing them with cells from a donor.

Although this provides hope a cure is close to being confirmed, bone marrow transplants reportedly wont be used for people with HIV who dont have cancer because of the risks involved in the procedure namely graft versus host disease, infection, organ damage, and death.

Regardless, Doctor Annemarie Wensing, a virologist at the University Medical Center Utrecht, told The New York Times she hoped news of the cases will inspire a generation of doctors to keep striving for what was previously believed to be impossible.

She concluded by saying this will inspire people to believe the cure is not a dream but is, in fact, reachable.

If you have a story you want to tell, send it to UNILAD via [emailprotected]

A Broadcast Journalism Masters graduate who went on to achieve an NCTJ level 3 Diploma in Journalism, Lucy has done stints at ITV, BBC Inside Out and Key 103. While working as a journalist for UNILAD, Lucy has reported on breaking news stories while also writing features about mental health, cervical screening awareness, and Little Mix (who she is unapologetically obsessed with).

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Gay Couple Adopt HIV-Positive Child After She Was Rejected By Ten Other Families - UNILAD

Cell therapy startup raises $16 million to fund its quest for the Holy Grail in regenerative medicine – Endpoints News

In 2006, Shinya Yamanaka shook stem cell research with his discovery that mature cells can be converted into stem cells, relieving a longstanding political-ethical blockage and throwing open medical research on everything from curbing eye degeneration to organ printing.

But that process still has pitfalls, including in risk and scalability, and some researchers are exploring another way first hinted at years ago: new technology to convert mature cells directly into other mature cells without the complex and time-consuming process of first making them into stem cells.

One of those companies, Mogrify, just raised $16 million in Series A financing to bring its overall funding to over $20 million since its February launch. Led by CEO Darrin Disley, the funding will help expand their new base in Cambridge to a 60-strong staff and push forward their direct-conversion approach to cell therapy through research and licensing. Investors include Parkwalk Advisors and Ahren Innovation Capital.

They list potential applications as treatments for musculoskeletal and auto-immune disorders, cancer immunotherapy, and therapies for ocular and respiratory diseases. For example, you could use it regenerate cartilage in arthritis patients.

If you could take a cell from one part of the body and turn it into any other cell at any other stage of development for another part of the body, you effectively have the Holy Grail of regenerative medicine, Disley told Labiotech.eu in April.

Mogrifys advantage over the Yamanaka method called induced pluripotent stem cells (iPS), is that in theory it can be more scalable and avoid the problems associated with iPS. These include instabilities arising from the induced immature state and an increased risk of cancer if any pluripotent cells remain in the body.

The concept behind Mogrify actually predates, by nearly 19 years, Yamanakas discovery, which fast won him the 2012 Nobel Prize in Medicine. A 2017 Nature study on transdifferentiation, as the process is called, of fibroblasts into cardiac tissue traced the idea to a 1987 findingthat a master gene regulator could convert mice fibroblasts into skeletal muscle.

The problem though, according to Mogrify, is that most current efforts rely on an exhausting guess-and-check process. With hundreds of cell types and an even greater number of transcription factors the program that recodes the cell finding the right factor for the right cell can be like a custodian with a jangling, unmarked key ring trying to get into a building with thousands of locks.

Mogrifys key tech is a computer model they say can predict the right combination. The scientists behind the platform published a 2016 study in Nature applying the model to 173 human cell types and 134 tissues.

Before Mogrify, Disley led the Cambridge-based gene-editing company Horizon Discovery.

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Cell therapy startup raises $16 million to fund its quest for the Holy Grail in regenerative medicine - Endpoints News

Some cases of SIDS may have this genetic cause – Futurity: Research News

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New research links a genetic anomaly and some forms of SIDS, or sudden infant death syndrome, which claims the lives of more than 3,000 infants a year.

The research, published in Nature Communications, focuses on mitochondrial tri-functional protein deficiency, a potentially fatal cardiac metabolic disorder caused by a genetic mutation in the gene HADHA.

Newborns with this genetic anomaly cant metabolize the lipids found in milk, and die suddenly of cardiac arrest when they are a couple months old. Lipids are a category of molecules that include fats, cholesterol, and fatty acids.

There are multiple causes for sudden infant death syndrome, says Hannele Ruohola-Baker, professor of biochemistry at the University of Washington School of Medicine, who is also associate director of the Medicine Institute for Stem Cell and Regenerative Medicine.

There are some causes which are environmental. But what were studying here is really a genetic cause of SIDS. In this particular case, it involves defect in the enzyme that breaks down fat.

Lead author Jason Miklas, who earned his PhD at the University of Washington and is now a postdoctoral fellow at Stanford University, says he first came up with the idea while researching heart disease and noticed a small research study that had examined children who couldnt process fats and who had cardiac disease that was not readily explained.

So he and Ruohola-Baker started looking into why heart cells, grown to mimic infant cells, died in the petri dish where they were growing.

If a child has a mutation, depending on the mutation the first few months of life can be very scary as the child may die suddenly, Miklas says. An autopsy wouldnt necessarily pick up why the child passed but we think it might be due to the infants heart stopping to beat.

Were no longer just trying to treat the symptoms of the disease, Miklas says. Were trying to find ways to treat the root problem. Its very gratifying to see that we can make real progress in the lab toward interventions that could one day make their way to the clinic.

In MTP deficiency, the heart cells of affected infants dont convert fats into nutrients properly, resulting in a build-up of unprocessed fatty material that can disrupt heart functions. More technically, the breakdown occurs when enzymes fail to complete a process known as fatty acid oxidation. It is possible to screen for the genetic markers of MTP deficiency; but effective treatments remain a ways off.

Ruohola-Baker says the latest laboratory discovery is a big step towards finding ways to overcome SIDS.

There is no cure for this, she says. But there is now hope, because weve found a new aspect of this disease that will innovate generations of novel small molecules and designed proteins, which might help these patients in the future.

One drug the group is focusing on is Elamipretide, used to stimulate hearts and organs that have oxygen deficiency, but barely considered for helping infant hearts, until now. In addition, prospective parents can undergo screening to see if there is a chance that they could have a child who might carry the mutation.

Ruohola-Baker has a personal interest in the research: one of her friends in Finland, her home country, had a baby who died of SIDS.

It was absolutely devastating for that couple, she says. Since then, Ive been very interested in the causes for sudden infant death syndrome. Its very exciting to think that our work may contribute to future treatments, and help for the heartbreak for the parents who find their children have these mutations.

The National Institutes of Health, the Academy of Finland, Finnish Foundation for Cardiovascular Research. Wellstone Muscular Dystrophy Cooperative Research Center, Natural Sciences and Engineering Research of Canada, an Alexander Graham Bell Graduate Scholarship, and the National Science Foundation funded the work.

Source: University of Washington

Original Study DOI: 10.1038/s41467-019-12482-1

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Some cases of SIDS may have this genetic cause - Futurity: Research News

More Studies Needed to Assess Effects of Cord Blood Transplants in CP, Review Finds – Cerebral Palsy News Today

More controlled clinical studies are needed to investigate the effects of umbilical cord blood transplants as a form of treatment for cerebral palsy (CP) and other diseases, according to a recent literature review.

The study, Systematic review of controlled clinical studies using umbilical cord blood for regenerative therapy: Identifying barriers to assessing efficacy, was published in Cytotherapy.

There has been an increasing use of umbilical cord blood (UCB) for different therapeutic purposes in regenerative medicine in recent years.

Cord blood is a long-known source of different cell types, including stem cells a type of cell capable of promoting the repair of damaged tissues. These cells represent a promising treatment for numerous conditions, including cerebral palsy. However, the usefulness of UCB transplants for new indications, including for the treatment of CP, type 1 diabetes, liver diseases, and congestive heart failure, is still unclear.

Clinical studies have been conducted in many different countries and have described cord blood therapy for a broad array of indications, the researchers said. However, these studies are often heterogeneous in nature, and study designs are variable and describe outcomes using a range of measures at various time-points.

In addition, many of these studies lacked a proper control group, which made estimations of therapeutic efficacy impossible for these new indications, they said.

In this review study, the researchers focused on summarizing the main findings of controlled clinical studies investigating the usefulness of UCB for CP, type 1 diabetes, and nine other new indications.

Literature searches in two online databases Medline and Embase yielded a total of 360 potentially relevant studies published between June 2016 and April 2018. After further screenings, a total of 16 controlled studies four on CP, three on type 1 diabetes, and nine on other medical conditions were selected for inclusion in this review.

Three of the four studies performed in CP, involving 247 children with the disease, were based on the use of allogeneic cells that is, UCB cells that had been obtained from a matching donor.

Only one of the studies investigated the effect of banked UCB cells that had been collected from the children at birth and then re-transplanted back to them at some point a procedure called autologous treatment.

Six months after treatment, children with CP who received allogeneic cells had a significant improvement in gross motor function, as measured by the Gross Motor Performance Measure (GMPM) and by the Gross Motor Function Measure (GMFM), compared with those who did not receive UCB cells (controls).

Mental and motor function scores at other time points were highly variable between studies.

The one study in which children received autologous treatment also reported a significant improvement in the participants GMFM scores after one year, compared with controls. This positive effect on motor function was proportional to the number of cells these children had received, known as a dose-dependent effect.

Although studies of cerebral palsy appear promising, the use of different scoring systems at varied time points following transplantation limits our ability to determine whether the intervention is beneficial, particularly at later time points beyond 12 months, the researchers said.

Of the three studies that focused on patients with type 1 diabetes, one evaluated the effects of allogeneic treatment, and two the effects of autologous treatment. All three failed to detect any positive effects of the therapy on daily insulin requirements, or on glycated hemoglobin A1c (HbA1c) the fraction of hemoglobin that is bound to glucose, or sugar, in the blood compared with controls.

Only one of the remaining nine controlled studies, which focused on investigating the effects of UCB cells in adults with optic nerve hypoplasia, reported a positive effect of treatment.

More controlled studies are needed that use similar approaches regarding cell source and outcome measures at similar time points. Pooled estimates of results from multiple studies will be essential as published studies remain modest in size, the researchers said.

Patients should continue to be enrolled in clinical trials because there are no novel potential indications remain unproven, they concluded.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that make up the lining of blood vessels found in the umbilical cord of newborns.

Total Posts: 70

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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More Studies Needed to Assess Effects of Cord Blood Transplants in CP, Review Finds - Cerebral Palsy News Today