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Colin Jackson health: Im in constant pain Athlete to undergo stem cell therapy to help – Express

Colin Ray Jackson, CBE, 54, is a Welsh former sprinter and hurdling Olympic silver medal champion. Colins world record for his 60 metres hurdles stood for an incredible 27 years. As with most athletes of his calibre, Colin suffers with ongoing injuries from his sports days and will be undergoing a treatment to reduce the pain which Mike Tyson recently underwent too.

In recent years, stem cell therapy has been hailed as a miracle cure for many conditions, from wrinkles to spinal repair.

A stem cell is an immature, basic cell that has not yet developed to become, say, a skin cell or a muscle cell or a nerve cell.

There are different types of stem cells that the body can use for different purposes.

There is evidence that stem cell treatments work by triggering damaged tissues in the body to repair themselves, often referred to as regenerative therapy.

In animal studies, stem cell treatments have shown promise for various diseases, including heart disease, Parkinsons disease and muscular dystrophy.

A study undertaken by Dr Timothy McGuine found that 34 percent of athletes involved in the one-year study were more likely to report a history of knee and hip injuries.

Additionally, he found that specialised athletes, such as those competing in the Olympic games, were twice as likely to sustain a gradual onset or repetitive use injuries than athletes who did not specialise.

Dr McGuine also found that these athletes who find themselves competing year-round, stressing the same muscles and movements, and predisposed to the symptoms of burnout are at higher risk of long-term injuries.

Many doctors and athletes use stem cell therapy to treat sports injuries, such as Achilles tendinopathy or damaged knee ligaments, said Sports Health.

The site continued: While increasing in popularity, stem cell therapy is not considered standard practice by sports medicine doctors and not covered by most insurance companies.

The process of collecting stem cells is often called harvesting. Physicians usually harvest stem cells from the patients fat, blood, or bone marrow.

Many physicians who use stem cell therapy hypothesize that, when placed into a certain environment, stem cells can transform to meet a certain need.

Other sports stars who underwent stem cell therapy for long-term injuries included Cristiano Ronaldo, Rafael Nadal and most recently Mike Tyson.

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Colin Jackson health: Im in constant pain Athlete to undergo stem cell therapy to help - Express

The Basics of MDS: Diagnosis and Staging – Curetoday.com

Myelodysplastic syndromes, known commonly as MDS, are a group of bone marrow diseases characterized by bone marrow failure, or an inadequate production of blood counts called cytopenia.

In a presentation at the CURE Educated Patient Leukemia Summit, Dr. Rami Komrokji, section head for Leukemia and MDS and Vice Chair of the Department of Malignant Hematology at Moffit Cancer Center, gave a run down on the diagnosis and staging process for MDS.

Diagnosing MDS

Komrokji explained that the myelo- prefix means bone marrow, and -dysplasia means abnormal-looking cells. When a patient has cytopenia, they may experience certain symptoms.

If patient is anemic, they will have shortness of breath, fatigue, palpitations, said Komrokji in an interview with CURE. If they have low platelets, they will have bleeding tendency, bruising. If they have low white blood cell counts, they will have maybe infections. So usually, either some of those symptoms will prompt blood testing, or on routine physical exam, the patients are found to have low blood counts. So that's usually the initial step.

Doctors will usually look into nutritional deficiencies such as B12, folate and ferritin, said Komrokji. Eventually, the patients will get a bone marrow aspirate and biopsy to diagnose their disease, which includes several parts.

There is the morphologic part, which means the pathologists are looking at the cells under a microscope, explained Komrokji. And then there is also some genetic testing. We look at cytogenetics nowadays, we look at gene mutations. So we put all of this information together to make the diagnosis.

The hematopathologist must see dysplasia, increased myeloblasts (immature cells known as blasts within the bone marrow) or certain cytogenetic abnormalities to make their diagnosis, Komrokji said.

Sometimes the diagnosis is straightforward, but sometimes it could be challenging, he added. It truly depends on an experienced hematopathologist to make the diagnosis.

Staging and Risk Stratification

Once a patient receives an MDS diagnosis, their doctor will go over risk stratification, or understanding what the risk of their disease is, which is what they consider staging, Komrokji said.

Now in MDS, its not like a lung cancer or colon cancer, he said. The disease does not spread around. The staging is based on the blood counts, on the percentage of those myeloblasts or immature cells (and) the chromosomal makeup of the cells. And nowadays, we sometimes also incorporate the presence of gene mis-happenings as well. So we get a lump score to estimate the risk.

Doctors typically use the International Prognostic Scoring System (IPSS) to categorize patients into one of five categories very low, low, intermediate, high and very high. The risk is the impact on survival and whether the disease will transform to leukemia, Komrokji explained. The disease risk must be known in order to tailor the patients treatment to them.

I always advise patients to see a specialized center in MDS, because obviously, those are not that common diseases, he said. A community oncologist could see a few (cases) per year, while an experienced center like in our place, we see like 15 to 20 per week.

Gene Mutations

Komrokji said that understanding gene mutations is an evolving field that is slowly becoming routine.

I advise all patients to inquire if theyve gotten genetic testing or not, he said. This sort of testing will help them understand any abnormalities. Doctors can look at a patients individual gene levels and detect for mutations, of which at least one was identified in 90% of patients with MDS.

Understanding the patients mutation(s) helps them tell whether there is a clonal hematopoiesis or mis-happening that occurred. It can also impact prognosis and allow them to further understand the disease risk.

And finally, some of them are targetable or important to follow through the treatment, said Komrokji. So patients should probably definitely have a genetic testing done. And sometimes after a treatment failure, we repeat it because we see other mutations that we could target with new drugs.

What Causes MDS?

In most cases, the cause of a patients MDS diagnosis is unknown.

We think it's phenomena of senescence or aging of those stem cells in the bone marrow that produces the blood, said Komrokji. Obviously, the process is very complicated. We have billions and billions of cells divide billions of times a day. So you know, as those cells age, mistakes can happen in them.

In most cases, he said, the mis-happenings which lead to the disease are random and at no fault of the patient. It is extremely rare for MDS to be inherited through familial genes.

There are, however, several known risk factors of MDS. If someone has history of another form of cancer and has received chemotherapy or radiation therapy, they may have possible stem cell damage and can develop MDS this is called therapy-related MDS. There has also been association of the disease with benzene exposure, chemical exposure and radiation exposure. Patients who have connective tissue diseases such as rheumatoid arthritis and lupus are at a slightly higher risk of getting MDS due to inflammation in the body and certain medications used to treat those diseases.

I would say there's a lot of better understanding of the disease in the past several years, of genetic mutation testing and incorporating them in practice, Komrokji said. And I think, you know, there are a lot of new treatments on the horizon for patients; there are several clinical trials in advanced phase.

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The Basics of MDS: Diagnosis and Staging - Curetoday.com

Pharming Group reports financial results for the first half of 2021 – PRNewswire

Recovery continued into Q2 2021 as patient enrollment and product demand increases

LEIDEN, The Netherlands, Aug. 5, 2021 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (Euronext Amsterdam: PHARM/NASDAQ: PHAR) presents its preliminary (unaudited) financial report for the first six months of 2021 ended June 30, 2021.

Financial Summary

Amounts in US$m except per share data

H1 2021

H1 2020

% Change

Income Statement

Revenues

93.2

97.8

(5)%

Gross profit

83.8

86.9

(4)%

Operating profit

17.2

35.7

(52)%

Profit for the year

14.4

20.3

(29)%

Balance Sheet

Cash & marketable securities

189.8

173.8

9%

Share Information

Basic earnings per share (US$)

0.022

0.032

(31)%

Diluted earnings per share (US$)

0.019

0.028

(32)%

Financial highlights

Operational highlights

Post-period operational highlights

Chief Executive Officer, Sijmen de Vries, commented:

"As expected, revenue growth during Q2 2021 has continued to recover, following the impact of COVID-19 on Q1 2021, as underlying demand and patients benefiting from RUCONEST treatments for their HAE increases. We are confident this positive trend will continue for the remainder of the year and, supported by our strong cash position, will enable us to continue our planned investment in R&D and the ongoing preparations for the launch of leniolisib, which, subject to regulatory approval, is on track for the end of 2022 following the completion of patient enrollment in the potentially registration enabling study in APDS.

In our earlier pipeline, we initiated enrollment of patients in a multi-center Phase IIb clinical trial of rhC1INH for the prevention of acute kidney injury after myocardial infarction. In addition, post period, we delivered on one of our strategic objectives to strengthen our longer-term HAE pipeline, through a collaboration with Orchard Therapeutics, to develop and commercialize the pre-clinical ex-vivo autologous hematopoietic stem cell therapy product OTL-105, which has the potential to become a curative treatment for HAE. We remain focused on the positive progress against our three-pillar strategy of sales, R&D and acquisitive growth."

Outlook

For the remainder of 2021, we expect:

No further specific financial guidance for 2021 is provided.

About Pharming Group N.V.

Pharming Group N.V. is a global, commercial stage biopharmaceutical company developing innovative protein replacement therapies and precision medicines for the treatment of rare diseases and unmet medical needs.

The flagship of our portfolio is our recombinant human C1 esterase inhibitor (rhC1INH) franchise. C1INH is a naturally occurring protein that down regulates the complement and contact cascades in order to control inflammation in affected tissues.

Our lead product, RUCONEST, is the first and only plasma-free rhC1INH protein replacement therapy. It is approved for the treatment of acute hereditary angioedema (HAE) attacks. We are commercializing RUCONEST in the United States, the European Union and the United Kingdom through our own sales and marketing organization, and the rest of the world through our distribution network.

In addition, we are investigating the clinical efficacy of rhC1INH in the treatment of further indications, including pre-eclampsia, acute kidney injury and severe pneumonia as a result of COVID-19 infections.

We are also studying our oral precision medicine, leniolisib (a phosphoinositide 3-kinase delta, or PI3K delta, inhibitor), for the treatment of activated PI3K delta syndrome, or APDS, in a registration enabling Phase 2/3 study in the United States and Europe.

Furthermore, we are leveraging our transgenic manufacturing technology to develop next-generation protein replacement therapies, most notably for Pompe disease, which is currently in preclinical development.

Forward-looking Statements

This press release contains forward-looking statements, including with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, Pharming's ability to overcome the challenges posed by the COVID-19 pandemic to the conduct of its business, and Pharming's expectations regarding its projected working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2020 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2020 filed with the U.S. Securities and Exchange Commission, the events and circumstances discussed in such forward-looking statements may not occur, and Pharming's actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Pharming as of the date of this release.

Inside Information

This press release relates to the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation.

For further public information, contact:Pharming Group, Leiden, The NetherlandsSijmen de Vries, CEO: T: +31 71 524 7400 Susanne Embleton, Investor Relations Manager: T: +31 71 524 7400 E: [emailprotected]

FTI Consulting, London, UKVictoria Foster Mitchell/Alex Shaw T: +44 203 727 1000

LifeSpring Life Sciences Communication, Amsterdam, The NetherlandsLeon Melens T: +31 6 53 81 64 27 E: [emailprotected]

Conference call dial-in informationThursday August 5, 2021 13:00CET/07:00ETPlease note, the Company will only take questions from dial-in attendees.

Dial-in details:

Netherlands (Local) 085 888 7233

United Kingdom 0800 640 6441

United Kingdom (Local) 020 3936 2999

All other locations +44 20 3936 2999

Access code: 914296

Webcast Link:https://webcast.openbriefing.com/pharming-aug21/

Pharming Group N.V.Condensed Consolidated Interim Financial Statements in US Dollars (unaudited)For the period ended 30 June 2021

Appendix: Main condensed consolidated Interim Financial Statements reported in Euros

(This appendix is not part of the Condensed Consolidated Financial Statements)

CONDENSED CONSOLIDATED STATEMENT OF PROFIT AND LOSS

For the 6-month period ended 30 June

Amounts in $ '000

notes

HY2021

HY 2020

Revenues

7

93,237

97,827

Costs of sales

8

(9,487)

(10,885)

Gross profit

83,750

86,942

Other income

1,354

525

Research and development

(24,206)

(17,658)

General and administrative

(15,060)

(9,846)

Marketing and sales

(28,686)

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Pharming Group reports financial results for the first half of 2021 - PRNewswire

Blood donation clinic will honour life of Jocelyn McGlynn – Chatham Daily News

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A blood donation clinic is being held Aug. 10 in Chatham to honour the life and memory of the late Jocelyn McGlynn, whose courageous battle with leukemia inspired many to register to become stem cell donors.

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A blood donation clinic is being held Aug. 10 in Chatham to honour the life and memory of the late Jocelyn McGlynn, whose courageous battle with leukemia inspired many to register to become stem cell donors.

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McGlynn, who was a medical science student at Western University in London with dreams of becoming a doctor, became an advocate for stem cell and blood donations while she battled leukemia, which took her life on Aug. 15, 2020, at age 23.

McGlynn was first diagnoses in the fall of 2018 when she went to see doctors for what appeared to be a cold she could not shake. This was the beginning of a tough medical battle that included multiple rounds of chemotherapy, radiation and two bone marrow transplants.

Joc was selfless and strong, caring and courageous, wonderful and wise, said McGlynns mother, Jacqueline McGlynn, about her daughter becoming a vocal champion for those fighting similar medical battles.

She loved to live and did not want others to suffer as she did.

During her own fight, Jocelyn McGlynn continued to encourage stem cell and blood donations, which included taking part in Walk the Night for the Leukemia and Lymphoma Society of Canada and creating awareness videos for Blood Sweat Spin and Canadian Blood Services.

McGlynn used her creative talents to envision and bring to reality her Lets All Go Get Swabbed music video. She wrote the lyrics, played every note, directed, edited and starred in the video while recovering from chemotherapy.

People can help continue McGlynns efforts by scheduling an appointment for the upcoming blood donor clinic being held Tuesday at the Chatham YMCA from noon to 7 p.m. Donations can be booked online at blood.ca, through the GiveBlood App or by calling 1-888 2 DONATE (236-6283).

A single donation of blood is equal to one unit of blood, and a leukemia patient can require up to eight units of blood per week, stated the release.

As McGlynn wrote: Everybody join the team, theres a cure in your bloodstream.

Maureen Macfarlane, event co-ordinator with Canadian Blood Services, said there is an increased need for blood donations as hospital procedures, which were previously on hold during the COVID-19 pandemic, are being scheduled.

Another blood donor clinic is also being held in Chatham on Aug. 31 from 1 p.m. to 7 p.m. at the Retro Suites Hotel.

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Blood donation clinic will honour life of Jocelyn McGlynn - Chatham Daily News

Symptoms of seizures: Early signs, post-seizure, and more – Medical News Today

A person having a seizure may experience muscle spasms, blink rapidly, fall to the ground, or lose consciousness. They will require medical diagnosis of and treatment for the underlying cause of seizures.

Seizures may occur as a side effect of a health condition that originates from the brain, such as epilepsy.

Nonepileptic seizures may occur as well. These vary in type and may affect individuals with curable or reversible health conditions.

Other catalysts for seizures include:

This article will discuss in further detail the symptoms and types of seizures, their causes, the aftereffects, and more.

Sometimes, people will experience certain sensations, called an aura, prior to a seizure. These symptoms result from electrical changes in the brain that occur when the seizure starts.

They act as a warning that a seizure will happen. Yet the exact symptoms will depend on which part of the brain the seizure starts in.

According to John Hopkins Medicine, an aura typically occurs before a focal seizure, especially a complex focal seizure. The most common aura involves feelings such as:

People may also experience visual changes, hearing abnormalities, or changes in their sense of smell prior to having a seizure.

If a person notices any of these symptoms, they may want to keep track of them. Recording details of symptoms and when they happen can help a doctor diagnose and treat seizures.

Symptoms can vary depending on the type of seizure an adult has, but they may include:

Seizures may last a few minutes. However, the Centers for Disease Control and Prevention (CDC) encourage people to call 911 if:

Babies can also have seizures. However, a person may have more difficulty recognizing them because a babys healthy, typical responses may look similar to symptoms of a seizure.

Newborns commonly jerk, twitch, or contort their faces into grimacing expressions. So, a person can easily mistake these movements for seizures or not recognize them as seizure symptoms at all.

Seizure symptoms may include any or all of the following:

According to the University of Chicago Medicine, seizures are particularly likely if a baby repeats the same movements for approximately the same duration at the same time of day, usually when waking up or going to sleep. For babies prone to seizures, symptoms commonly occur every day.

In general, research suggest that genetics play an important role in whether a baby will experience seizures.

According to a 2018 research review, one-third of children with febrile seizures have a family history of seizures. The risk of a baby having febrile seizures is around 20% with an affected sibling and 33% with affected parents.

After a seizure, a person may experience a variety of effects that may persist for several minutes or even hours.

According to the National Institute of Neurological Disorders and Stroke, people may feel tired, weak, and confused after a seizure has ended. Other common symptoms include headaches or pain in the muscles that contracted during the seizure.

The specific aftereffects that a person experiences can help a healthcare professional determine which part of the brain seizure originated in.

One example is Todds paralysis, a temporary paralysis in part of the body due to the area of the brain the seizure originated in. If the seizure stemmed from the temporal lobe, a person is more likely to experience language or behavioral changes.

According to the CDC, two main types of seizures exist: focal onset and generalized onset.

Generalized seizures affect both sides of the brain, and they include absence seizures and tonic-clonic seizures. These types of seizures produce many common seizure symptoms, such as:

Focal seizures, or partial seizures, originate in just one area of the brain. Subcategories of focal seizures include:

People with epilepsy may experience all these types of seizures.

Psychogenic nonepileptic seizures may also occur, due to psychological distress. Though these are not epileptic seizures, they have similar symptoms.

These symptoms include:

Research suggests the most common cause is a conversion disorder. This occurs when a person has the symptoms of a health condition without an actual underlying condition or injury.

Nerve cells in the brain send electrical signals to communicate. Any change to this electrical activity can cause seizures. Factors that can cause abnormal electrical activity include:

According to a 2021 article, genetics play a role in whether a person will experience epilepsy. The article also lists other potential causes for seizures:

With a treatable underlying cause, such as hypoglycemia, seizures are not likely to reoccur after treatment.

For epilepsy specifically, the CDC states that the condition is most likely to develop in children under age 2 and adults over age 65.

Not every seizure is an emergency, and while they can be fatal, the CDC states that most people can live safely with seizures. Seizures may turn life threatening if:

Although very rare, sudden unexpected death in epilepsy (SUDEP) does occur. This refers to deaths that have no other cause than the seizure itself.

SUDEP is the cause of death in roughly 1.16 out of every 1,000 people with epilepsy.

If a person experiences a seizure for the first time, they will need prompt medical attention. A doctor can then diagnose the underlying cause and suggest appropriate treatment.

If a person experiences subsequent, more severe seizures, they should also seek medical help. A doctor can analyze patterns, discuss any aftereffects, and reevaluate treatment to help reduce the severity of seizures.

People who experience seizures may find it helpful to record the details of each episode to report to their doctor.

If people witness someone having a seizure, they will need to call 911 for any seizure that lasts longer than 5 minutes, according to the CDC, or if any injury occurs during the seizure.

Seizures may be epileptic or nonepileptic, and they stem from a disruption in nerve cell activity in different parts of the brain. Seizures typically last for only a few minutes at a time.

A person may experience various symptoms before, during, and after a seizure.

Tracking specific symptoms of a seizure may help a doctor diagnose or adjust treatment for seizures. Learning to recognize the warning signs of a seizure can also help people get into a safe space before a seizure occurs.

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Symptoms of seizures: Early signs, post-seizure, and more - Medical News Today

Global Induced Pluripotent Stem Cells Market Industry Analysis, Post COVID-19 Impact, Emerging Trends, Business Growth by 2027||Fate Therapeutics,…

Induced Pluripotent Stem Cells market research report comprises of several parameters which are thoroughly studied by the experts. Market research analysis and data lend a hand to businesses for the planning of production, product launches, costing, inventory, purchasing and marketing strategies. This market study considers a market attractiveness analysis, where each segment is benchmarked based on its market size, growth rate, and general attractiveness. Market info can be explained more specifically in terms of breakdown of data by manufacturers, region, type, application, market status, market share, growth rate, future trends, market drivers, opportunities, challenges, emerging trends, risks and entry barriers, sales channels, and distributors.

Induced pluripotent stem cells (iPSCs) marketis expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to account to USD 2,442.97 million by 2027 growing at a CAGR of 7.5% in the above-mentioned forecast period. Increasing R&D investment activities is expected to create new opportunity for the market.

Get Sample Copy Of This Report @https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-induced-pluripotent-stem-cells-market

Few of the major competitors currently working in global induced pluripotent stem cells market areFUJIFILM Holdings Corporation, Astellas Pharma Inc, Fate Therapeutics, Bristol-Myers Squibb Company, ViaCyte, Inc., CELGENE CORPORATION, Vericel Corporation, KCI Licensing, Inc, STEMCELL Technologies Inc., Japan Tissue Engineering Co., Ltd., Organogenesis Holdings Inc, Lonza, Takara Bio Inc., Horizon Discovery Group plc, Thermo Fisher Scientific.

Global Induced Pluripotent Stem Cells Market Drivers:

Increasing R&D investment activities is expected to create new opportunity for the market.

Increasing demand for personalized regenerative cell therapies among medical researchers & healthcare is expected to enhance the market growth. Some of the other factors such as increasing cases of chronic diseases, growing awareness among patient, rising funding by government & private sectors and rising number ofclinical trialsis expected to drive the induced pluripotent stem cells (iPSCs) market in the forecast period of 2020 to 2027.

High cost of the induced pluripotent stem cells (iPSCs) and increasing ethical issues & lengthy processes is expected to hamper the market growth in the mentioned forecast period.

Global Induced Pluripotent Stem Cells (iPSCs) Market Scope and Market Size

Induced pluripotent stem cells (iPSCs) market is segmented of the basis of derived cell type, application and end- user. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Inquiry For Customize Report With Discount at :https://www.databridgemarketresearch.com/inquire-before-buying/?dbmr=global-induced-pluripotent-stem-cells-market

In-depth analysis of the market

The core objectives of this report are:

TOC of Induced Pluripotent Stem Cells Market Report Contains:

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Report points with potential

About Data Bridge Market Research

An absolute way to forecast what future holds is to comprehend the trend today!

Data Bridge set forth itself as an unconventional and neoteric Market research and consulting firm with unparalleled level of resilience and integrated approaches. We are determined to unearth the best market opportunities and foster efficient information for your business to thrive in the market. Data Bridge endeavors to provide appropriate solutions to the complex business challenges and initiates an effortless decision-making process.

Data bridge is an aftermath of sheer wisdom and experience which was formulated and framed in the year 2015 in Pune. We ponder into the heterogeneous markets in accord with our clients needs and scoop out the best possible solutions and detailed information about the market trends. Data Bridge delve into the markets across Asia, North America, South America, Africa to name few.

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Global Induced Pluripotent Stem Cells Market Industry Analysis, Post COVID-19 Impact, Emerging Trends, Business Growth by 2027||Fate Therapeutics,...

Bayer’s BlueRock Therapeutics gains FDA fast track for Parkinson’s disease cell therapy – PMLiVE

The US Food and Drug Administration (FDA) has granted BlueRock Therapeutics a fast track designation for its cell therapy candidate DA01 for advanced Parkinsons disease.

BlueRock a Bayer subsidiary is currently evaluating the pluripotent stem cell-derived dopaminergic neuron therapy in a phase 1 study.

This early-stage trial is set to enrol ten patients across the US and Canada, with its primary objective to assess the safety and tolerability of DA01 cell transplantation at one-year post-transplant.

As a secondary objective, BlueRock will assess the evidence of transplanted cell survival and motoreffects at one- and two-years post-transplant and evaluate the continued safety and tolerability at two years, as well as the feasibility of transplantation.

Receiving fast track designation from the FDA is an important step, which will help us further accelerate clinical development of our DA01 cell therapy approach for Parkinsons disease, said Joachim Fruebis, chief development officer of BlueRock.

This is another critical step in the BlueRock mission to create authentic cellular medicines to reverse devastating diseases, with the vision of improving the human condition, he added.

In 2019, Bayer bought out its private equity partner Versant Ventures and founders in BlueRock Therapeutics for $240m, three years after setting up the company.

The decision gave Bayer complete control of BlueRocks cell therapy pipeline, headed by DA01 and spanning various diseases in the neurology, cardiology and immunology categories.

BlueRock was set up in late 2016 with $225m in start-up funding from Bayer and investment firm Versant, shortly after Bayer backed gene-editing specialist Casebia via its Leaps by Bayer investment arm.

BlueRocks induced pluripotent stem cells (iPSCs) platform is designed to encourage PSCs to differentiate into the dopaminergic neurons that are progressively destroyed in Parkinsons disease.

The hope is that introducing these neurons into areas of the brain where neurons are depleted will lead to increased dopamine release, restoring motor function.

The company is also developing iPSCs that differentiate into microglia, oligodendrocytes and eneic neurons for neurology applications, cardiomyocytes for heart failure, as well as macrophages and T regulatory cells for immunology applications including immune tolerance, fibrosis and graft-versus-host disease (GvHD).

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Bayer's BlueRock Therapeutics gains FDA fast track for Parkinson's disease cell therapy - PMLiVE

$500K Grant Supports Research on Muscle Growth in Pigs, With Broader Health Implications – Maryland Today

A $500,000 award from the U.S. Department of Agriculture National Institute of Food and Agriculture is supporting University of Maryland-led research to enhance pork production through improved muscle growth in pigs.

While early life nutrition is especially important for how muscles grow and develop, less is known about how these benefits can be passed from mother to offspring during pregnancy. For the $20 billion U.S. pork industry, increased and faster muscle growth would result in healthier animals, less feed and waste to raise that animal, and ultimately a more competitive and sustainable pork industry.

In partnership with the Uniformed Services University of the Health Sciences (USUHS), this work could also have future applications beyond the pork industry to optimize human performance and treat wounded service members.The key to these applications could lie in the epigenetic changes (or changes to how genes are expressed) and stem cell activity caused by a simple supplementbutyric acid.

Over the last decade, weve published some nutritional work in this area showing the impact of butyric acid and other dietary components on the activity of tissue-specific stem cells, and feeding butyric acid to pigs resulted in faster muscle growth, said Chad Stahl, professor and chair in the Department of Animal and Avian Sciences who is leading the research. If we are able to make the muscle fibers grow bigger because of the activity of these muscle stem cells, we want to see what happens if we are giving these compounds to the pregnant sow during fetal development.

Stahl conducted previous research in this area with his former student, Robert Murray Ph.D. 18. Now an assistant professor with USUHS and a lieutenant in the U.S. Navy, he is a co-investigator on this grant.

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$500K Grant Supports Research on Muscle Growth in Pigs, With Broader Health Implications - Maryland Today

EU approves bluebird bio’s CALD gene therapy Skysona – PMLiVE

The European Commission (EC) has approved bluebird bios gene therapy Skysona for the rare inherited neurological disease cerebral adrenoleukodystrophy (CALD).

The EC has cleared Skysona (elivaldogene autotemcel) for the treatment of early CALD in patients under the age of 18 years old with an ABCD1 gene mutation who do not have a matched sibling blood stem cell donor.

The approval is supported by data from the phase 2/3 Starbeam study as well as the ongoing phase 3 ALD-104 study.

In the phase 2/3 Starbeam study evaluating Skysona, 90% of CALD patients met the month 24 major functional disability- (MFD) free survival endpoint as of the last data cutoff date.

MFDs are the six severe disabilities commonly attributed to CALD, which have the most severe effect on a patients ability to function independently.

In addition, 26 out of 28 evaluable patients maintained a neurologic function score (NFS) less than or equal to one until month 24, with 24 of those patients having no change in their NFS.

All the patients who completed the Starbeam study enrolled for long-term follow-up in the LTF-304 study. The majority of patients that enrolled in LTF-304 96.3% - remained alive and maintained their MFD-free status through their last follow-up on study.

The median duration of follow-up was 3.2 years and 14 patients reached at least their year five follow-up visit.

bluebird bio was founded with the mission of developing a therapy to recode CALD on the genetic level, and todays announcement represents over twenty years of research and development that has laid the groundwork for future gene therapies to be possible, said Andrew Obenshain, president of severe genetic diseases at bluebird bio.

CALD is a progressive and fatal neurodegenerative disease that overwhelmingly affects males. It involves the breakdown of myelin the protective sheath of nerve cells in the brain that is responsible for muscle control and thinking.

The condition is caused by mutations in the ABCD1 gene that affect the production of ALDP which eventually causes damage to the adrenal cortex and white matter of the brain and spinal cord.

Skysona is designed to add functional copies of the ABCD1 gene into a patients hematopoietic stem cells (HSC).

Once this functional gene is added to a CALD patients stem cells, the patient's body can produce the adrenoleukodystrophy protein (ALDP), which is believed to allow for the breakdown of very-long-chain fatty acids that build up to toxic levels in the brain.

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EU approves bluebird bio's CALD gene therapy Skysona - PMLiVE

Secrets and pies: the battle to get lab-grown meat on the menu – The Guardian

Not a week goes by without Elliot Swartz receiving at least one request from researchers asking him where they can find cell lines (a cell culture developed from a single cell) for use in cellular agriculture an essential tool for creating lab-grown meat. One of the most important things that cell lines offer is that they enable researchers to just get started in this new field, says Swartz, who works in New York as a senior scientist at the Good Food Institute (GFI) a nonprofit focused on advancing cellular agriculture and bringing its products to our shelves and stomachs as quickly as possible. Helping researchers is a core part of his role. In the case of cell lines, however, theres very little he can do.

Swartzs response to the researchers is unfortunately always the same: at the moment, publicly available cell lines relevant for cellular agriculture dont really exist. That doesnt mean that theyre nowhere to be found. Upside Foods (previously Memphis Meats) has submitted several patents to protect cell lines it has developed, and companies such as Cell Farm Food Tech have built a business around selling cell lines for profit. Keeping discoveries behind closed doors is a pattern of behaviour found in private companies across the industry, which many believe is slowing down innovation.

Cellular agriculture is the use of animal cells or microbes to grow animal products, such as meat or milk, in bioreactors. The field gained prominence after Dutch scientist Mark Post unveiled the first cultured meat burger in 2013. Since then, cultured meats have been touted as a sustainable alternative to livestock farming, which is the leading cause of habitat destruction. Global demand for burgers and bacon is to increase over the coming decades, meaning more ecosystems will be bulldozed to accommodate the expanding market. This, in turn, will increase the risk of future pandemics, as biodiversity loss is linked to the emergence of new diseases. Moreover, efforts to cut carbon emissions will also fall short of Paris targets if we dont reduce our meat consumption, according to a special report published by the Intergovernmental Panel on Climate Change in 2019.

There is some progress. In Singapore last year, Eat Just became the first cultured meat company to gain regulatory approval to sell its product. But many technological, social, and economic hurdles remain before our supermarkets are filled with a variety of cultured cutlets. To surpass these hurdles, organisations including the GFI are pushing for a more public exchange of data, tools and ideas. As it stands, most research in the field is done by private companies which seem keen to protect their intellectual property.

Swartz says the lack of publicly available cell lines is a gatekeeper in getting people into the field, even though theres a lot of interest, adding that this isnt really an issue in other industries. Scientists looking for stem cells for research or clinical purposes can go to the government-funded UK Stem Cell Bank, and across the Atlantic, the nonprofit American Type Culture Collection hosts a reserve of cell lines that are mainly open access. Although repositories like this do include animal cells, that doesnt mean theyre suitable for generating meat.

What makes cell lines themselves so useful is that they are immortal and can multiply indefinitely, so they can be used as a standard model across the industry. Were not going to understand if our findings are true if different groups are using different cells with different features, Swartz continues. So cell lines are the first piece of the puzzle for getting cultivated meat to become an actual field of study. The GFI is filling the cell-line-shaped hole in cellular agriculture by funding the creation of lines that will be openly accessible, and making a repository to store them in. Kerafast a Boston-based bioresearch company will maintain this repository. Researchers not involved with the GFI are welcome to deposit cell lines too, as are private companies; anyone looking to use the cells must pay a small fee to cover the costs of storing and maintaining them. So far, only one academic group has deposited a cell line. The lines being worked on in academic groups are still in development, which is why we havent got that many yet, Swartz says.

The reluctance of private companies to share their cell lines may in part be because of how they are financed a GFI report found that of the $366m invested in cultured meat in 2020, only around $12m came from public sources. Controlling the vast majority of the capital in the industry means that the private sector can comfortably dictate the pace and direction of innovation, which the Breakthrough Institutes food and agriculture analyst Saloni Shah sees as an issue. With the government and public sector funding research you can set criteria and standards, and make sure the right kinds of technologies get funded so that the development of the sector accelerates and improves, says Shah.

The complaint that governments need to start investing in more sustainable food options is echoed by Isha Datar, the executive director of New Harvest another nonprofit focused on advancing cellular agriculture. She thinks one of the reasons the field lacks government funding is that it is a mix of tissue engineering, which is medically oriented, and food science. Cellular agriculture is kind of homeless and so it falls in between the cracks of the existing pillars of funding and how we think about science being separated, she says. Swartz also agrees that more public funding is needed, but he thinks it will only come after the technology has been scaled up. Does this industry scale? is going to be the key to opening the floodgate for governments funding this technology, he says. Open source research is going to be really important for bringing new ideas on how to scale this technology or lower costs.

Swartz also complains that secrecy is holding up the industry-wide adoption of other cheaper, more efficient materials. For example, all of the nutrients needed for animal cells to grow into chunks of meat are contained in the cell culture medium, but the industry standard foetal bovine serum is expensive, and must be extracted from the foetus of a slaughtered cow. Many startups claim to have developed alternatives, but they remain trade secrets. Companies tend not to patent these things, because by patenting a cell culture medium you have to include everything thats in there, which is open sourcing what the ingredients are, says Swartz.

Even if the cell line problem were solved, there would still be technological hurdles holding the field back from large-scale commercialisation. Using computer modelling to address these hurdles and accelerate the intensification of cultured meat production is a central goal of the Cultivated Meat Modeling Consortium (CMMC).

Modelling is a useful tool that allows researchers to simulate experiments before entering a laboratory. This helps to save on time and resources. In order to run more complicated simulations, however, modellers first need data from simpler experiments that detail the fundamental biological processes behind cultured meat production to understand the sum of the whole, we must first analyse the parts. Were experiencing quite some difficulty in getting the information we need to actually build models, says Jaro Camphuijsen, a researcher associated with the CMMC. Private companies they work with have shown resistance to sharing data and running certain experiments. We have been talking to a cultivated meat company quite a lot, and we often ask: What happens if you do this experiment? The answer is usually: We dont know, and We arent going to do that because the cells will die, Camphuijsen explains. But failed experiments, he says, can provide useful data points that often reveal more than successful tests. Experiments that go wrong actually provide lots and lots of information if you want to find out how these tiny systems of cells are behaving.

When asked to respond to accusations that industry secrets were slowing down innovation in the field, Uma Valeti, the CEO of Upside Foods, wrote in an email that the firm kickstarted the cultured meat movement when we were founded in 2015. Without that, the industry wouldnt be in the place it is today, where there are hundreds of companies, NGOs, academic groups and government institutions focusing on cultured meat, across every continent but Antartica. He adds that Upside is actively supportive of more open access research on cultured meat, and it has actively supported the development of public research institutions like the Cultured Meat Consortium.

What's the point of lab-grown meat when we can simply eat more vegetables? | Jenny Kleeman

Responding to the same accusations, Robert E Jones, head of public affairs at Mosa Meat, wrote: Few companies have done more than Mosa Meat to contribute to the open advancement of cellular agriculture. He adds that Mosa hoped the 2013 burger would trigger a moonshot level of public investment in research, and that there is something to be said for an innovation ecosystem that includes both private capital and public investments for a challenge as big as reforming the food system.

The idea that governments need to start investing in more sustainable food options is echoed by Datar. She has concerns about a field that lacks an academic basis and publicly accessible information. It means cellular agriculture is going to have to be more transparent than other industries, says Datar. I think we need a lot more data sharing and a lot more transparency if we are to create a better food system. Will private companies heed this call for more transparency and build on their claims that they are supportive of more open access research, or will they follow the approach in other sectors where financial gain has been prioritised over societal benefits? Campaigners hope the answer is one that puts the planet before profit margins.

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Secrets and pies: the battle to get lab-grown meat on the menu - The Guardian