Archive for the ‘Bone Marrow Stem Cells’ Category

Anadrole Reviews Does CrazyBulks Anadrole (Anadrol) Supplement Really Work? Do the ingredients have any negative side effects? Read its price, results, dosage & customer reviews!

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What is Anadrole?

Anadrole is a muscle-building supplement developed by CrazyBulk that imitates the effects of the anabolic steroid Anadrol and androgen oxymetholone, which treat low levels of red blood cells.

Its primary function is to stimulate the synthesis of red blood cells in your body, which in turn helps to reduce the symptoms of anemia, including lethargy, muscle weakness, and weariness, among other symptoms.

Anadrole from CrazyBulk contains high concentrations of various proteins, amino acids, and ergogenic herbs.

These ingredients all work together to produce the same results as oxymetholone without harming your health.

It is designed to offer all of the advantages that its equivalent, available only by prescription, does, but without any potentially harmful effects.

Because the steroid Anadrol increases your stamina and assists you in fighting exhaustion, the Anadrole supplement makes it possible for you to attain bigger improvements in muscle mass.

It can effectively assist in growing muscle and bolstering muscle strength, and it speeds up the healing of muscles after exercise.

Your workouts will be more effective if you include them in your routine, and it is simple to use Anadrole in any fitness program.

Improve your physical performance, maintain your muscle mass, and shed excess weight with the help of this cutting-edge muscle-building supplement Anadrole.

It provides sustained energy surges, enabling you to work better and for longer at the gym. As a result, you can break through muscle-building plateaus more easily.

You will be able to achieve your fitness objectives more quickly and easily if you consistently apply this powerful method.

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How does Anadrole supplement work?

It accomplishes this by utilizing two metabolic processes stimulated by its proprietary formulas. To begin, taking this Anadrole can enhance a process known as erythropoiesis, which takes place in the bone marrow.

To put it another way, the stimulatory impact that the capsules have on the bone marrow stem cells causes an increase in the creation of red blood cells.

Because of this increased RBC, more oxygen will be carried into organs, particularly the muscles.

As a consequence, the muscles may be able to recover from weariness more quickly.

The second impact is known as the androgenic effect, which plays a role in both the manufacture of testosterone and the improvement of the bioavailability of male hormones.

Testosterone plays a significant role in the initiation of fat burning, which is beneficial to the process of creating lean muscle.

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Benefits of consuming Anadrole Capsules:

The Anadrole supplement sold by CrazyBulk is comprised of a potent composition that helps maintain your muscle health in a variety of ways.

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What are the Ingredients used in Anadrole Supplement?

Anadrole has appropriate quantities of natural substances that improve muscle strength, growth, and recovery and is packed full of these nutrients.

They cooperate to alleviate the symptoms associated with iron deficiency and low numbers of red blood cells.

In light of scientific research, the following is everything you need to know about the components that are found in Anadrole and the benefits that come from them:

Tribulus Terrestris, a plant native to the Mediterranean region that bears fruit and is a member of the caltrop family, is traditionally employed in herbal medicine as an aphrodisiac.

It is loaded with compounds that improve sexual performance in both men and women and has these substances in plenty.

The compounds found in the fruit of the Tribulus Terrestris plant have the power to interfere with the function of particular hormones in your body beneficially, which results in the production of ergogenic effects.

Anadrole from CrazyBulk has a total of 500 mg of Tribulus Terrestris fruit extract packed into every serving.

Soy protein is a type of protein that can be acquired from soybeans. It is unique in that it contains all of the essential amino acids your body cannot produce independently.

Because it is a complete protein, it stimulates muscle protein synthesis in your body by supplying your body with significant quantities of all of the required amino acids.

Losing weight, maintaining good cholesterol levels, and gaining muscle are all benefits. The Anadrole sold by CrazyBulk includes 200 mg of soy protein extract in every serving.

Whey protein, the primary protein found in dairy products, encourages the development of new proteins within your body by providing it with all of the needed amino acids.

It is a protein that is broken down very quickly and is an essential component in the process of creating new muscle protein.

Anadrole sold by CrazyBulk comes with 200 milligrams of whey protein concentrate in every single serving.

Nitric oxide can be produced from the amino acid L-arginine when it is metabolized in the body.

L-arginine has a role in maintaining healthy blood flow by contributing to the production of nitric oxide, which is important for keeping your blood vessels relaxed.

In addition, it influences particular hormones in your body, such as insulin and growth hormone, amongst others.

L-carnitine is a naturally occurring amino acid derivative that aids in synthesizing energy and enhances thermogenesis and encourages body fat reduction.

Your endurance will improve due to acetyl-l-role carnitine in the mitochondria, and you will be able to push yourself further during your workouts.

Acetyl l-carnitine is the primary factor responsible for Anadroles capacity to increase ones energy levels. Anadrole sold by CrazyBulk contains 50 mg of acetyl l-carnitine in every serving.

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Pros of Anadrole:

Cons of Anadrole:

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Side effects of Anadrole Crazy Bulk

Anadrole does not contain an API, which stands for active pharmaceutical ingredient, or, to put it another way, the chemical compound responsible for carrying out all of the necessary functions.

The so-called beneficial effects are caused by certain herbs and the extracts of those herbs, similar to the way that Ayurvedic medications and other traditional treatments function.

Because you are genuinely consuming certain plant components in capsule form, there are no adverse consequences, unlike those caused by steroids, such as kidney failure, liver damage, high blood pressure, or organ damage.

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Pricing of Anadrole and Return Policy

One bottle of CrazyBulks Anadrole costs $64.99 and includes 60 individual capsules in each bottle.

If you buy two bottles at once, you will receive a free bottle as a bonus, and the total cost of 3 bottles is $129.98.

When you place an order straight through the official CrazyBulks Anadrole supplement website, you get free shipping on all of your purchases, no matter where you are.

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How to use Anadrole?

To experience the maximum potential of Anadrole, the manufacturer suggests taking two capsules each day for at least two months.

Because it requires the body a little bit more time to digest it, it is recommended that the Anadrole be taken first thing in the morning.

It is recommended that you take both capsules at the same time, at least twenty minutes before breakfast, to ensure that the Anadrole capsules are adequately absorbed along with the food.

When taking capsules, it is advisable to consume them with water or fruit juice; alcohol of any kind should be avoided at all costs.

The body is better able to properly distribute the tablets contents when it is given for 60 days consecutively followed by 10 days off cycle.

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Anadrole Reviews Final Verdict

Anadrole is a great option for beginners and intermediates looking for a supplement that will help them gain muscle, increase their endurance and stamina, and improve their lean body mass.

This one, in contrast to other legal steroids, has a price tag that is slightly more reasonable, and its impact is comparable to that of the original steroid.

This products capacity to increase RBC counts assists in the fight against fatigue and shortens the time needed for recovery after exercise.

As a result of the herbal formulation, Crazy Bulk products do not cause any unfavorable health effects and do not cause any side effects.

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Disclaimer: We are a professional product review website. We might receive compensation when you buy through our website, we may earn a small affiliate commission. The information contained on this website is provided for informational purposes only and is not meant to substitute for the advice provided by your doctor or other healthcare professional. The products have not been evaluated by the Food and Drug Administration and are not intended to diagnose, treat, cure, or prevent any disease.

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Anadrole Reviews - Best Alternative to Anadrol Steroid? Any Side Effects? - MarylandReporter.com - MarylandReporter.com

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CHENNAI: In 2004, flight attendant Kaveri Mandanna was battling frequent fevers, cold, and dipping haemoglobin. Life was an endless series of check-ups and blood transfusions. After consultations with a haematology specialist, tests and procedures ensued. Kaveri's condition was found to be leukaemia -- it meant that her bone marrow tissue was rapidly producing abnormal numbers of a certain type of blood cell, crowding out the others.

Although shaken, she didnt want to lose hope. "I had to be alive for my daughter -- that was the only thought pushing me to fight," says Kaveri who was in her 30s then.

A bone marrow transplant, also called a stem cell transplant, was recommended as a recourse. That gave Kaveri additional succour.

"Today, people suffering from rare diseases like certain types of cancer, sickle cell anaemia, immune conditions, and other conditions affecting the production of blood, have a shot at being cured through such stem cell transplants," says Dr G Buvaneswari, infertility specialist, Rela and GBR Hospitals.

Stem cells are special cells which can create copies of themselves. "These are the bodys raw cells -- they keep dividing and evolving into other cell types such as the bones, heart muscle, nerves, other organs and tissue," explains Dr Buvaneswari.

Its a matchThe tricky part of such transplants? Finding an immunologically compatible donor. As Kaveri had two siblings, who are natural genetic matches, they were HLA-typed.

Explaining this, Dr Buvaneswari says, "This procedure identifies protein markers on cells and tissues. The markers help determine whether or not a donor and a beneficiary match."

The more the markers, the fewer the risks of infection. To Kaveri's luck, her younger brother was found to be eligible to donate.

Uthappa, Kaveris brother and donor, was glad that he could potentially save his sister's life. However, he was apprehensive, too.

"Blood from one of my arms was collected every day, for five days, for a few hours. It was a smooth process, but, still, I was worried I'd experience side effects after giving away my stem cells," says Uthappa. But there was no cause for concern, he found, as donors are given injections to replicate extra bone marrow stem cells.

On D-day of the transplant process, Uthappa's stem cells were introduced into Kaveri's body via a catheter. Meanwhile, Kaveri had undergone chemotherapy which emptied her bone marrow of all its blood cells, including the healthy ones. This is done to make space for the donor's stem cells, says Dr Buvaneswari.

With time, the transplanted stem cells travelled to Kaveris bone marrow, where it started producing healthy red blood cells, white blood cells, and platelets of its own.

Kaveri still needed to do check-ups at regular intervals. "At first, I had to meet my specialists every week. Then, the frequency reduced to quarterly sessions, and later, once a year," Kaveri recounts. Subsequent signs of her cell count returning to the normal number indicated disease remission.

Almost six years later, from the day of the transplant in 2004, Kaveri's cancer was cured. "The transplant's success and the blessing of being declared cancer-free gave me the confidence to resume my globe-trotting life," she exclaims.

Banking for the futureWhile Kaveri was lucky enough to find a biologically-related donor, the odds in general are not quite high. Contacting a registry of voluntary unrelated donors is the only option then.Healthy individuals between the ages of 18 to 60 can register to donate their blood stem cells, says Sumati Misra, head, counselling and transplant centre management at DATRI blood stem cell registry. Having a wide pool of registered donors increases the odds of finding HLA matches for those needing transplants, she says.

"At DATRI, we have 1,800 patients registered with us who havent found a match yet from our registry of almost four lakh registered donors," shares Sumati.

Another factor in HLA typing is ethnicity, thus, it's hard to find a European registry match for an Indian. A large Indian donor registry solves that problem, she suggests.

Did you know that umbilical cord blood is a rich source of stem cells?

"The cord and the placenta, generally discarded as medical waste. They can be saved, and the stem cells from the tissue and the blood extracted, and stored for future transplant use," says Dr Buvaneswari.

As the blood is tested for infections, the likelihood of a person's body rejecting stem cells from cord blood is lesser than from bone marrow.

Some parents pay for their children's cord blood cells to be cryo-preserved in private banks for an extended period of time. They can be used in the rare event of a child developing a condition, in the future, that can be treated with stem cell therapy. The child's siblings stem cells, too, if they are HLA matches, can prove helpful.

"If the child is found to be an HLA match with another child, alternatively, parents can take a call on donating the banked cord blood. As cord blood banking is an expensive undertaking, donating them may be ideal," suggests Dr Buvaneswari.

Research on stem cells is underway to see if they can treat various conditions that affect different systems and parts of the body. Stem cell therapy could be a prospective game-changer in not just regenerating tissues but also organs, says Dr Buvaneswari, who is currently researching the use of stem cells in infertility treatment.

Sharing a recent trend from the field, the doctor says, "After pre-clinical trials with animals, it was observed that stem cell therapy could restore their ovarian and even testicular functions."

"After more phases of the trials with human cells, the results, if favourable, could lead to advancements in reproductive care," she concludes.

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The gift of life: How stem cell transplant helped a flight attendant trump Cancer - The New Indian Express

Thalassemia is an inherited blood disorder that cannot be treated with medication. But stem cell therapy may be able to help. Know if stem cell therapy will work for the disease.

Written by Editorial Team | Updated : May 18, 2022 10:56 AM IST

Haemoglobin is the oxygen-carrying protein in the blood. In the absence or reduced amount of haemoglobin, the red cells do not survive for a long period and there is a diminished oxygen supply to the tissues causing a variety of symptoms. Thalassemia is an inherited blood disorder in which there is less haemoglobin than normal. There are two parts to the haemoglobin molecule alpha and beta. The disease is named based on the subunit that is not being made by the body alpha or beta-thalassemia.

Thalassemia runs in families; however, not everyone who has a dysfunctional gene for haemoglobin will have the symptoms of the disease. Those with moderate-severe disease experience symptoms such as breathlessness, weakness etc. (symptoms of anaemia), all associated with low levels of oxygen, from childhood. However, those with the mild disease might incidentally be diagnosed with thalassemia, mostly during investigations for other conditions or for anaemia.

Unfortunately, thalassemia cannot be prevented. Patients with the severe disease receive frequent blood transfusions, in an attempt to replenish a pool of healthy red blood cells. However, this procedure is associated with several issues such as iron overload that can deposit in different organs over time, immune system reactions, and risk of blood-related infections such as hepatitis, HIV etc. Folic acid is also prescribed to patients, which can help in the development of red blood cells.

Considering the issues with the conventional treatment of thalassemia, stem cell therapy can be an attractive choice for the condition. Allogeneic hematopoietic stem cell (blood stem cells) transplantation has been considered the only curative treatment for thalassemia. This treatment works by replenishing a healthy pool of blood-forming stem cells (master cells of the body) in the bone marrow consider the bone marrow as a factory of stem cells and specialized blood and tissue forming cells. However, considering that the source of stem cells is from a donor (usually a first-degree relative) because a patient's own blood-forming stem cells would be affected by the genetic mutation there are chances of certain side effects. One way to manage these issues is by the use of mesenchymal stem cells, which are known to have immune system regulating properties. By this, the rejection-related issues with hematopoietic stem cells as well as reduced immunity-related side effects can be handled efficiently. Considering that thalassemia is a disease that affects blood cells, mesenchymal cells (tissue forming cells) from the patient's own body are not affected.

Through this combined approach, a patient can achieve long-term remission from the symptoms of thalassemia without side effects. "Stem cell therapy for thalassemia is not a new treatment, only the approach by using a combination of stem cells to achieve holistic outcomes is novel.

(The article is contributed by Dr Pradeep Mahajan, Regenerative Medicine Researcher, StemRx Bioscience Solutions Pvt. Ltd., Navi Mumbai/Mumbai)

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How Does Stem Cell Transplant Work In Thalassemia? Know Everything | TheHealthSite.com - TheHealthSite

Named for their ability to branch out to become cells that build multiple kinds of tissue, mesenchymal stem cells (MSC) and mesenchymal stromal cells appear in embryos and adult bone marrow, respectively. In 2019 scientists suggested redefining the MSC acronym as medicinal signaling cells, because the therapeutic cells usually come from bone marrow rather than the mesenchymea part of embryos. In this article well refer to all these similar cell types as stem cells.

Twenty years ago biologists had successfully developed stem cells into cartilage, tendon, heart, bone, and other kinds of tissues in their laboratoriesspurring hope for unprecedented tissue repair therapies in humans and animals.

Scientists now know stem cells use a very different healing mechanism than previously suspected, Oosterlinck says. In fact, recent research has revealed its not what these cells become that matters, but what they doand, more specifically, how they do it, in large part through a process known as paracrine signaling.

Basically, theres a lot of crosstalk that happens between stem cells and the injured cells, Schnabel says. That crosstalk seems to recruit special cells within the injured tissue called progenitor cellsand those appear to develop into the original local tissue cells, such as tendon cells.

Some research groups are looking at ways to predifferentiate cells in a laboratorypreparing them to go into tendon versus cartilage tissue, for exampleto help encourage them to be more effective in their therapeutic environment, Oosterlinck says.

Meanwhile, other research teams are showing how stem cells trigger specific cytokines (cell-signaling proteins) and growth factors that contribute to better tissue healing, he says.

The cells also encourage vascularization, says Schnabel. They actually bring blood vessels into the area, promoting angiogenesis, she says.

These recent discoveries about stem cell functions are major breakthroughs, says Schnabel. Her team currently focuses on optimizing the use of these cells. One thing theyre homing in on is dosing, which has always been complex, especially in horses, because its impossible, she says, to achieve the per-pound dose of cells recommended in human medicine.

Theyre also fine-tuning the question of treatment timing, she says. Traditionally, clinicians have treated horses with stem cells once the initial inflammatory response from the injury subsides. The idea was that you dont want to cause more inflammation, potentially, and have an even lamer horse, and you dont want the stem cells to get killed by the inflammation, she explains.

Ultimately, that might not be the right approach. All the studies weve been doing actually suggest the opposite, that having them in an inflammatory environment is good, because it further primes the cells to secrete the things you want, she says.

Then that also begs the question, if you get a horse after the time of acute inflammation, could you prime the cells first in the lab so that theyre ready to go when you put them in the horse? she adds. And thats been a major focus of our work. We have a lot of strong preliminary data suggesting that thats true.

Schnabels team, as well as other research groups, have looked specifically at tendon healing, revealing that horses treated with stem cells have significantly reduced re-injury rates, especially in the superficial digital flexor tendon (SDFT)which has a traditional re-injury rate of up to 70% in racehorses (RK Smith, et al.). This is huge, she says.

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What's New in Equine Orthobiologics? The Horse - TheHorse.com

Theglobal cell therapy marketsize was valued atUSD 8.1 billion in 2021and is estimated to reachUSD 23.9 billion by 2030, growing at a CAGR of 14.5% over the forecast period. The development of precision medicine and advancements in cellular therapies in context to their efficiency & manufacturing are expected to be major drivers for the market. Moreover, the development of stem cell banking facilities and resultant enhancement of stem cells production, storage, and characterization are also expected to improve the volumetric capabilities of the market at a global level, which is anticipated to directly translate into revenue for this market at a larger level. Ongoing technological advancements in the parent and ancillary markets for stem and non-stem cells usage are expected to reinforce the demand over the forecast period. There are fewer commercialized cellular therapy products in the current market than the number of research products. This is partly due to stringent regulations and the high cost of stem cells.

Cell lines, such as Induced Pluripotent Stem Cells (iPSC) and human Embryonic Stem Cells (hESC) are recognized as having high growth potential; as a result, many research entities and companies are making significant investments in R&D pertaining to iPSC- and hESC-derived products.

Pricing of stem cell transplantation varies from region to region. For instance, the cost of transplantation in the U.S. is higher than that in Germany or China. In March 2018, Alofisel by TiGenix received approval for marketing in Europe. This was the first allogeneic stem cell therapy to be approved in Europe. Furthermore, revenue for certain products varies for the country; for instance, products like INVOSSA received approval for marketing in Korea but have yet to receive marketing authorization in the U.S. Growth is also influenced by the commercialization of unauthorized stem cell treatments revenue generation.

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Global Cell Therapy Market Definition

Therapy in which viable cells are injected, grafted, or implanted into a patient to effectuate a medicinal effect is known ascell therapy; for instance, In immunotherapy, T-cells capable of fighting cancer cells via cell-mediated immunity are transplanted, and stem cells are grafted to regenerate diseased tissues.

Cellular therapies hold a great therapeutic promise across various clinical applications. This has resulted in substantial global investments in research and their clinical translation. Rapid advances in stem cell research have the potential to fulfill the unmet demand of pharmaceutical entities, biotech entities, and doctors in disease management. Several unknown therapies are in clinical development.

Furthermore, government and private funding agencies are constantly offering grants to support projects at various stages of clinical trials, increasing the number of ongoing clinical trials.

Research on human embryonic stem cells is ethically controversial. Harvesting embryonic stem cells involves the destruction of human embryos, raising a moral concern. In addition, stringent regulations for obtaining Intellectual Property Rights (IPR) for products or materials used in research are major restraints for commercializing these services. Ethical approval should be obtained to store cell lines and tissues in biorepositories to avoid the usage of tissue for illegal purposes or to identify proxy diseases to claim insurance. Moreover, controversies surrounding the use of embryonic stem cells for research impede the market growth in several regions

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The study categorizes the cell therapy market based on use type and therapy type at the regional and global levels.

The analysis of the cell therapy market is based on the use of stem cells for clinical and research purposes. The research-use segment dominated the market for the global cell therapy market and accounted for the largest revenue share of 58.3% in 2021. Currently, cell therapies (stem & non-stem cells) are majorly being used for research projects, which in turn, has led to a large revenue share of this segment in 2021. Cell-based therapies are all possibilities for the replacement, repair, restoration, and regeneration of damaged tissues, cells, and organs. As an alternative to traditional treatment strategies, researchers are investing heavily in developing effective and safe cell-based treatments.

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As per the CGT Catapult database of clinical trials, 59 cell and gene therapy trials are ongoing in the UK. Out of all therapeutic areas, oncology has the highest number of ongoing clinical trials. T cells, CD34+ and CD133+ stem cells, mesenchymal stem/stromal cells are some predominantly employed cell types for clinical investigation. Neural cells, bone marrow mononuclear cells, fibroblasts, cornea cells, antigen-presenting cells, epithelial cells, and chondrocytes are some other cells that are being explored for the development of cell therapies.

Asia Pacificaccounts for the highestCAGR during the forecast period

Based on the regions, the global cell therapy market has been segmented across North America, AsiaPacific, Europe, South America, and the Middle East & Africa.In the Asia Pacific, the market for cell therapy is anticipated to witness a lucrative growth rate of 15.5% over the forecast period. Advancements in stem cell therapy in Asian countries are observed to be better than those in the U.S. This has resulted in Asia leading stem cell research. Several stem cell consortiums in Asian countries aim to ensure coordinated and focused R&D programs. Moreover, patients from western countries migrate to Asian countries for treatment, owing to the flexible legal framework.

Companies from Japan, South Korea, India, China, Taiwan, Singapore, and the rest of Asia were active participants in the conference. In addition, the large regional population and untapped potential present in the region have resulted in global firms entering the market. Moreover, this region offers relatively inexpensive manufacturing & operating units for conducting research. These factors are expected to play a major role in expanding the stem cell market in this region.

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The cell therapy market is mildly concentrated in nature with few numbers of global players operating in the market such as Kolon TissueGene, Inc., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., Castle Creek Biosciences, Inc., MEDIPOST, Osiris Therapeutics, Inc., PHARMICELL Co., Ltd, Tameika Cell Technologies, Inc., Cells for Cells, NuVasive, Inc., Vericel Corporation, and Celgene Corporation

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Cell Therapy Market Status and Size Report 2030 The Daily Vale - The Daily Vale

personalized-cell-therapy-market

Global Personalized Cell Therapy Market is growing at a High CAGR during the forecast period 2022-2028. The increasing interest of the individuals in this industry is that the major reason for the expansion of this market and This has brought along several changes in This report also covers the impact ofCOVID-19on the global market.

The latest research report, titled Personalized Cell TherapyMarket Added by Market Info Reports, provides the reader with a comprehensive overview of the Personalized Cell Therapy Industry and familiarizes them with the latest market trends, industry information, and market share. The report content includes technology, industry drivers, geographic trends, market statistics, market forecasts, producers, and raw material/equipment suppliers. Global Personalized Cell Therapy market size was xx million US$ and it is expected to reach xx million US$ by the end of 2028, with a CAGR of XX between 2022 and 2028.

Personalized Cell Therapy Market competition by top manufacturers as follow:Novartis AG, Vericel Corporation, Bellicum Pharmaceuticals, MolMed SpA, Cytori Therapeutics Inc, Gilead Sciences, Inc, Celgene Corporation, Bluebird Bio, Aurora Biopharma Inc, Saneron CCEL Therapeutics?Inc, Kuur Therapeutics, MediGene AG, Sangamo Therapeutics and More

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Dont miss the trading opportunities on Personalized Cell Therapy Market. Talk to our analyst and gain key industry insights that will help your business grow as you create PDF sample reports.

The global Personalized Cell Therapy market has been segmented on the basis of technology, product type, application, distribution channel, end-user, and industry vertical, along with the geography, delivering valuable insights.

Market split by Type, can be divided into:By Cell TypeHematopoietic Stem CellSkeletal Muscle Stem Cell/Mesenchymal Stem Cells/LymphocytesBy TechniquePlatelet Transfusions/Bone Marrow Transplantation/Packed Red Cell Transfusions/Organ Transplantation

Market split by Application, can be divided into:Cardiovascular DiseasesNeurological DisordersInflammatory DiseasesDiabetesCancer

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Market segment by Regions/Countries, this report covers:North AmericaEuropeChinaRest of Asia PacificCentral & South AmericaMiddle East & Africa

Years Considered to Estimate the Market Size:History Year: 2015-2019Base Year: 2019Estimated Year: 2022Forecast Year: 2022-2028

Major factors covered in the report:

Detailed TOC of Personalized Cell Therapy Market Report 2022-2028:Chapter 1: Personalized Cell Therapy Market OverviewChapter 2: Economic Impact on IndustryChapter 3: Market Competition by ManufacturersChapter 4: Production, Revenue (Value) by RegionChapter 5: Supply (Production), Consumption, Export, Import by RegionsChapter 6: Production, Revenue (Value), Price Trend by TypeChapter 7: Market Analysis by ApplicationChapter 8: Manufacturing Cost AnalysisChapter 9: Industrial Chain, Sourcing Strategy and Downstream BuyersChapter 10: Marketing Strategy Analysis, Distributors/TradersChapter 11: Market Effect Factors AnalysisChapter 12: Personalized Cell Therapy Market ForecastContinued

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The analysis objectives of the report are:

Does this report provide customization?Customization helps organizations gain insight into specific market segments and areas of interest. Therefore, Market Info Reports provides customized report information according to business needs for strategic calls.

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Personalized Cell Therapy Market 2022 Strategic Analysis, Growth Drivers, Industry Trends, Demand and Future Opportunities till 2028 |Novartis AG,...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--May 24, 2022--

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that new data from clinical trials of 18 approved and investigational medicines across more than 20 cancer types will be presented at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting, which will be held June 3-7, 2022. Genentech and its partners will present clinical studies across medicines, comprehensive genomic tests, and real-world data at this years meeting.

At ASCO this year, progress from our portfolio, partnerships and collaborations showcase our commitment to advance innovation in cancer care, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. Were especially pleased to present data from our broad hematology portfolio, including pivotal data for glofitamab, a potential first-in-class bispecific antibody that may improve the lives of people with heavily pre-treated aggressive lymphoma.

Focusing on improving outcomes in non-Hodgkin lymphoma

New and updated data in non-Hodgkin lymphoma will be presented at ASCO. This includes pivotal data from the Phase II NP30179 study investigating glofitamab, an investigational CD20xCD3 T-cell engaging bispecific antibody, in heavily pre-treated patients with diffuse large B-cell lymphoma (DLBCL). DLBCL is an aggressive form of lymphoma, where as many as 40% of patients will relapse, at which point treatment options are limited and survival is shortened. Glofitamab is part of Genentechs broad bispecific antibody development program, which may offer a new immunotherapy-based approach to tackle a range of blood cancers. It is being investigated in several clinical trials including the STARGLO Phase III study, evaluating glofitamab in combination with gemcitabine and oxaliplatin (GemOx) versus MabThera/Rituxan (rituximab) in combination with GemOx in autologous stem-cell transplant ineligible relapsed or refractory DLBCL. In addition, key findings from an analysis of the Asia subpopulation from the pivotal Phase III POLARIX study investigating Polivy (polatuzumab vedotin) in combination with MabThera/Rituxan plus cyclophosphamide, doxorubicin and prednisone (R-CHP) in people with newly diagnosed DLBCL will be featured. Polivy plus R-CHP is the first treatment regimen to significantly improve outcomes in previously untreated DLBCL in more than 20 years, potentially transforming treatment for people with this disease.

Driving innovation in personalized cancer care

More than 20 new pieces of research from partnerships with Foundation Medicine will be presented, which continue to support innovation as well as progress in personalized cancer care. This includes new data from the Phase II Profiler02 study,* which investigates the use of a comprehensive genomic profiling testing panel from Foundation Medicine, with the aim of informing possible treatment decisions for patients based on their tumors unique genomic information.

Data from the imCORE network

Additionally, three abstracts from the Immunotherapy Centers Of Research Excellence (imCORE) Network will be presented at ASCO: a Phase I study** investigating autogene cevumeran (an mRNA-based individualized neoantigen-specific immunotherapy [iNeST]***) in the adjuvant setting of pancreatic ductal adenocarcinoma; a data mining study** evaluating intermediate endpoints for survival in metastatic breast cancer in the real-world setting; and a study identifying mechanisms of acquired resistance to immune checkpoint blockade.**

imCORE is an academic-industry network for scientific collaboration. Established by Genentech and connecting experts from 26 leading institutions around the globe, imCORE is committed to advancing and accelerating cancer immunotherapy research. imCORE is an example of Genentechs dedication to collaborating with the global cancer community to further understand cancer biology and immunology, help inform the development of potential future treatment, and transform patients lives.

Genentechs data presented at ASCO will feature its efforts to drive innovation and commitment to health equity through delivery of pioneering medicines and personalized cancer care that together improve outcomes for every patient while reducing the cost to society, inclusive clinical trials that remove barriers to participation, partnerships that multiply our ability to address challenges in cancer care, and bringing innovation into earlier stages of disease to maximize a chance of cure.

Overview of key presentations featuring Genentech medicines

Medicine

Abstract title

Abstract number

Blood cancer

Glofitamab

Glofitamab in patients (pts) with relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL) and 2 prior therapies: Pivotal Phase II expansion results

#7500

Mosunetuzumab

CELESTIMO: a Phase III trial evaluating the efficacy and safety of mosunetuzumab plus lenalidomide versus rituximab plus lenalidomide in patients with relapsed or refractory follicular lymphoma who have received 1 line of systemic therapy

#TPS7588

Polivy

Asia subpopulation analysis from the Phase III POLARIX trial

#7558

Initial safety run-in results of the Phase III POLARGO trial: polatuzumab vedotin plus rituximab, gemcitabine, and oxaliplatin in patients (pts) with relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL)

#7551

Lung cancer

Tiragolumab

SKYSCRAPER-02: primary results of a Phase III, randomized, double-blind, placebo-controlled study of atezolizumab (atezo) plus carboplatin plus etoposide (CE) with or without tiragolumab (tira) in patients (pts) with untreated extensive-stage small cell lung cancer (ES-SCLC)

#LBA8507

Breast cancer

Giredestrant

Neoadjuvant giredestrant (GDC-9545) plus palbociclib (P) versus anastrozole (A) plus P in postmenopausal women with estrogen receptor-positive, HER2-negative, untreated early breast cancer (ER+/HER2 eBC): final analysis of the randomized, open-label, international Phase 2 coopERA BC study

#589

Inavolisib

Long-term safety of inavolisib (GDC-0077) in an ongoing Phase 1/1b study evaluating monotherapy and in combination (combo) with palbociclib (palbo) and/or endocrine therapy in patients (pts) with PIK3CA-mutated, hormone receptor-positive/HER2-negative (HR+/HER2) metastatic breast cancer (BC)

#1052

Tumor agnostic treatment and personalized healthcare

Rozlytrek

Efficacy/safety of entrectinib in patients (pts) with ROS1-positive (ROS1+) advanced/metastatic NSCLC from the Blood First Assay Screening Trial (BFAST)

#LBA9023

Rozlytrek

Trial in progress: a randomized Phase 3 study of entrectinib vs crizotinib in patients (pts) with locally advanced/metastatic ROS1 fusion-positive (fp) NSCLC with or without baseline CNS metastases (mets)

#TPS9141

Comprehensive genomic profiling

(IIS, Centre Lon Brard)

Increasing targeted therapy options for patients with relapsed cancer with broader somatic gene panel analysis from the primary tumor: The Profiler02 randomized Phase II trial*

#3130

Comprehensive genomic profiling

Clinical and genomic characteristics of patients with durable benefit from immune checkpoint inhibitors (ICI) in advanced non-small cell lung cancer (aNSCLC)

#9048

Comprehensive genomic profiling

ctDNA Shed as a Tool to Select Immune Checkpoint Inhibitors (ICPI) with or without Chemotherapy for Patients (pts) with advanced Non-small Cell Lung Cancer (aNSCLC)

#9045

Comprehensive genomic profiling

Trial in progress: LCMC LEADER Neoadjuvant Screening Trial: LCMC4 Evaluation of Actionable Drivers in Early Stage Lung Cancers

#TPS8596

Real world data

A real world (rw) evidence study quantifying the clinical value of multi-gene testing in early-stage lung adenocarcinoma (LUAD)

#8525

Real world data

Real world analysis of quantitative MET copy number (CN) as a biomarker in advanced NSCLC (aNSCLC)

#9123

Real world data

Ancestry-based differences in gene alterations in non-small cell lung cancer: real-world data using genetic ancestry analysis

#9125

imCORE,

ISR, Genentech

Identifying mechanisms of acquired immune escape from sequential, paired biopsies**

#2519

imCORE

ISR, Dana-Farber Cancer Institute

Real-World Progression-Free Survival (rwPFS) and Time to Next Line of Therapy (TTNT) as Intermediate Endpoints for Survival in Metastatic Breast Cancer: A Real World Experience**

#6520

imCORE

ISR, Memorial Sloan Kettering Cancer Center

Phase I Trial of Adjuvant Autogene Cevumeran, an Individualized mRNA Neoantigen Vaccine, for Pancreatic Ductal Adenocarcinoma**

#2516

* IIS, investigator-initiated study

** ISR, institution-sponsored research

*** jointly developed by Genentech and BioNTech

About Polivy (polatuzumab vedotin-piiq)

Polivy is a first-in-class anti-CD79b antibody-drug conjugate (ADC). The CD79b protein is expressed specifically in the majority of B cells, an immune cell impacted in some types of non-Hodgkins lymphoma (NHL), making it a promising target for the development of new therapies. Polivy binds to CD79b and destroys these B cells through the delivery of an anti-cancer agent, which is thought to minimize the effects on normal cells. Polivy is being developed by Genentech using Seagen ADC technology and is currently being investigated for the treatment of several types of NHL.

Polivy U.S. Indication

Polivy is a prescription medicine used with other medicines, bendamustine and a rituximab product, to treat diffuse large B-cell lymphoma in adults who have progressed after at least two prior therapies.

The accelerated approval of Polivy is based on a type of response rate. There are ongoing studies to confirm the clinical benefit of Polivy.

Important Safety Information

Possible serious side effects

Everyone reacts differently to Polivy therapy, so its important to know what the side effects are. Some people who have been treated with Polivy have experienced serious to fatal side effects. A patients doctor may stop or adjust a patients treatment if any serious side effects occur. Patients must contact their healthcare team if there are any signs of these side effects.

Nerve problems in arms and legs: This may happen as early as after the first dose and may worsen with every dose. If a patient already has nerve pain, Polivy may make it worse. The patients doctor will monitor for signs and symptoms, such as changes in sense of touch, numbness or tingling in hands or feet, nerve pain, burning sensation, any muscle weakness, or changes to walking patterns

Side effects seen most often

The most common side effects during treatment were:

Polivy may not be for everyone. A patient should talk to their doctor if they are:

These may not be all the side effects. Patients should talk to their healthcare provider for more information about the benefits and risks of Polivy treatment.

Report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch. Report side effects to Genentech at (888) 835-2555.

Please visit http://www.Polivy.com for the full Prescribing Information for additional Important Safety Information.

About Tecentriq (atezolizumab)

The rest is here:
Data at the 2022 ASCO Annual Meeting Highlight Genentech's Continued Commitment to Innovation in Oncology and Personalized Healthcare - Valdosta Daily...

Forward-looking Statements

Inadequate capital and barriers to raising the additional capital or to

Litigation with or legal claims and allegations by outside parties; and

Insufficient revenues to cover operating costs.

The Company is expanding its efforts to attract and cultivate collaborativepartners to accelerate its product development efforts, harnessing itsmanufacturing platform and tissue processing platforms. The R&D collaborationsare to discover, develop, and commercialize cellular therapies, laboratoryproducts and combinations thereof with synergistic technologies to createregenerative medicine applications and develop new intellectual property.

1. The Company filed its first Investigational New Drug Application (IND) with

the US Food and Drug Administration (FDA) for the ATCELL cellular therapy

product. The IND filing is titled "ATCell Expanded Autologous Adipose Derived

Mesenchymal Stem Cells deployed via Intravenous Infusion for the Treatment of

Post-Concussion Syndrome (PCS) in Retired Athletes and Military Personnel",

File number 19089, which was approved by the FDA on September 17, 2020. The

Company intends to invite additional developers of cellular therapies to

initiate additional arms of the clinical study focused on the use of ATCELL

for use in systemic inflammatory response relief for patient suffering from

systemic diseases. A number of these additional study targets have been

identified and ongoing discussions support the Company's belief that clinical

investigations can be developed and rapidly added upon completion of the new

2. DMD - The Company has completed the protocol for the treatment of Duchene

Muscular Dystrophy and is in final discussions with its Collaborative partner,

to select the principal investigator (PI) and clinical trial site selection.

3. Long COVID - The Company has completed the protocol for treatment of Long

COVID and is currently finalizing its FDA filings. We have completed, along

with a government partner the clinical protocol for a new Investigational New

Drug (IND) application to be filed with FDA within the next 90 days for Long

COVID. According to the Centers for Disease Control and Prevention,

"post-COVID conditions can be considered a lack of return to a usual state of

health following acute COVID-19 illness." In the US, following COVID recovery,

it is reported that up to 30% of those afflicted, diagnosed, or treated for

COVID-19 have continuing symptoms and medical complications following recovery

4. Wound Healing - the creation of topical applications and ingredients used by

physicians in the wound care and cosmetic industries as well as therapeutic

cellular applications and bio-materials development. An initial pilot study

involving a minimum of 10 participants for the assessment of its autologous

tissue products for the wound healing market is underway. The Company is

combining its tissue products, which do not require FDA approval, with current

standard-of-care methods to accelerate and improve the healing of diabetic and

5. ATGRAFT - products include adipose tissue and cell sample processing and

storage as a form of personal "bio-insurance", and adipose tissue (fat)

storage for cosmetic fat engraftment procedures. High demand for pure and

natural aesthetics in fast growing cosmetic industry with non-FDA required

Tissue Processing and Storage Services:

Laboratory Products; Culture Medium, and Manufacturing Services

The Company has created several versions of its ACSelerate cell culture mediaincluding:

ACSelerate-MAX - xeno serum free cell culture media,

ACSelerate-SFM - animal serum free cell culture media,

ACSelerate-LSM - low FBS (0.05%) cell culture media,

ACSelerate-CY- for differentiation of ATCELLinto chondrocytes (ATCELL-CY),

ACSelerate-OB- for differentiation of ATCELLinto osteoblasts (ATCELL-OB)

ACSelerate-AD - for differentiation of ATCELLinto adipocytes (ATCELL-AD)

ACSelerate-MY- for differentiation of ATCELL into myocytes (ATCELL-MY)

ACSelerate-CP- non-DMSO (Dimethyl Sulfoxide) cellular cryopreservation media

ACSelerate-TR- sterile transportation medium designed to maintain the

viability of the tissue during the shipment of adipose tissue to our processing

International Licensing Program - COVID RISK FACTOR

(https://www.transparencymarketresearch.com/pressrelease/stem-cells-market.htm)

China

Thailand

Japan

Collaborations / Partnering Opportunities / Acquisitions

WRNMMC

WRNMMC is part of The Military Health System (MHS) which is the enterprisewithin the United States Department of Defense that provides health care toactive duty, Reserve component and retired U.S. Military personnel and theirdependents.

To ensure that all active and reserve medical personnel in uniform are trained

and ready to provide medical care in support of operational forces around the

world.

To provide a medical benefit commensurate with the service and sacrifice of

more than 9.5 million active-duty personnel, military retirees, and their

families.

The MHS also provides health care, through the TRICARE health plan, to:

Active-duty service members and their families,

Retired service members and their families,

Reserve component members and their families,

Surviving family members,

Medal of Honor recipients and their families

Some former spouses, and

Others identified as eligible in the Defense Enrollment Eligibility Reporting

Intellectual Property

Systems and Methods for the Adipose Tissue Digestion U.S. Serial No.Digestion of Adipose Tissue Laboratory Processing 13/646,647 filed OctoberSamples Obtained From a Client Methods

Additionally, the Company has in-licensed the following IP:

Wound healing compositions and Protein Genomics andmethods using tropoelastin and American CryoStem USPTO: #6,808,707lysyl oxidase

Market Size and Opportunities

Regenerative Medicine Market

Development of Regional U.S. Markets

Physician Network

Development of International Markets

Corporate Information

Available Information

Results of Operations - Three Months

The Company's revenue for the quarter ended March 31, 2022, increased to $31,935versus $1,935 in the same period of Fiscal 2021.

Liquidity and Capital Resources

Accounts Receivable decreased to $65,680 at March 31, 2022 from $78,782 atSeptember 30, 2021.

There was no significant impact on the Company's operations as a result ofinflation for the six months ended March 31, 2022.

The Company leases Equipment at its laboratory from NFS Leasing, Inc. Leasepayments are $2,993.62 per month for eighteen (18) months. The final leasepayment is scheduled for January 1, 2023. When the final payment is made, theCompany will own the equipment. See Note 11 . Leases in the FinancialStatements.

The Company was not party to any litigation against it and is not aware of anylitigation contemplated against it as of March 31, 2022. See also LegalProceedings below.

From time to time the Company takes advances makes principal payments on thenote. During the six months ended March 31, 2022, the Company was advanced$1,729 and made payments of $18,000 on the note.

The principal balance of the Note is $131,505 at March 31, 2022 and 147,775 atSeptember 30, 2021.

Terms of the Series A Voting Convertible Preferred Shares are as follows:

1. Each Series A Share is convertible into 20 shares of American CryoStem common

stock $0.001 par value, subject to any recapitalization event.

2. Stated annual dividend of $0.20 per share payable quarterly in cash or stock

at the discretion of the Company's Board of directors.

The rest is here:
AMERICAN CRYOSTEM CORP MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND PLAN OF OPERATIONS (form 10-Q) - Marketscreener.com

RANDY HUTCHINSON| Better Business Bureau

The FDA says that stem cell products may offer the potential to treat many medical conditions and diseases for which few alternative treatments exist, but almost all of them have yet to be proven to be effective or safe. That hasnt stopped unscrupulous medical professionals and other promoters from touting them as miracle cures.

Stem cells are sometimes called the bodys master cells because they develop into blood, brain, bones and the bodys other organs. Stem cells that come from bone marrow or blood are routinely used to treat cancer and other disorders of the blood and immune system. But other uses, including treating COVID-19, have not been properly studied under an FDA Investigational New Drug Application, which includes providing sufficient data from human clinical testing to help evaluate a products potential risks.

The FDA cites these potential safety concerns for unproven treatments:

There are safety risks even with your own stem cells and contamination can occur if cells are manipulated after removal.

The FTC and Georgia Attorney General sued the co-founders of the Stem Cell Institute of America for allegedly targeting seniors with bogus claims that its therapy could treat arthritis, joint pain and other orthopedic ailments. The agencies said the company also claimed its stem cell therapy is comparable or superior to surgery, steroid injections and painkillers. A related company trained chiropractors and other healthcare practitioners in making similar claims. They also hosted free educational seminars for consumers at which they promoted injections that cost approximately $5,000 per joint, with many patients receiving multiple injections.

This wasnt the FTCs first enforcement action against deceptive stem cell therapy claims. In 2018, a California physician and his companies settled with the FTC over claims their amniotic stem cell therapy could treat Parkinsons disease, autism, macular degeneration, cerebral palsy, multiple sclerosis, heart attacks and a host of other serious ailments. The defendants earned at least $3.3 million offering injections that cost up to $15,000.

The only stem cell products approved by the FDA for use in the United States consist of blood-forming stem cells derived from umbilical cord blood. Theyre approved for limited use in patients with disorders that affect the production of blood.

In bringing the most recent action, the Acting Director of the FTCs Bureau of Consumer Protection said, At best, the use of unproven products or therapies can cost consumers thousands of dollars without affording them any results. At worst, it can be harmful to their health.

If youre considering a stem cell treatment, the FDA says to make sure its FDA-approved or being studied under an Investigational New Drug Application. The FTC and BBB offer these additional tips:

Randy Hutchinson is the president of the Better Business Bureau of the Mid-South. Reach the BBB at 800-222-8754.

See the original post:
Beware of unproven stem cell therapies - The Jackson Sun

New Jersey, United States,-ThisStem Cell TherapyMarketreport provides a clear picture of key players growth as well as the qualitative aspects of business in each area. ThisStem Cell TherapyMarket Report provides a current report on revenue generation, recent trends, financial status, and costing, as well as business profiles and financial status. The competitive landscape and potential growth factors are presented in thisStem Cell TherapyMarket Report.This way market report buyers can get a clear picture of the significant growth and the resulting market strategy. Granular market data would help monitor potential profitability and make critical growth decisions.

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Key Players Mentioned in the Stem Cell Therapy Market Research Report:

Osiris Therapeutics Medipost Co. Ltd., Anterogen Co. Ltd., Pharmicell Co. Ltd., HolostemTerapieAvanzateSrl, JCR Pharmaceuticals Co. Ltd., Nuvasive RTI Surgical Allosource

Stem Cell TherapyMarket Segmentation:

Stem Cell Therapy Market, By Cell Source

Adipose Tissue-Derived Mesenchymal Stem CellsBone Marrow-Derived Mesenchymal Stem CellsCord Blood/Embryonic Stem CellsOther Cell Sources

Stem Cell Therapy Market, By Therapeutic Application

Musculoskeletal DisordersWounds and InjuriesCardiovascular DiseasesSurgeriesGastrointestinal DiseasesOther Applications

Stem Cell Therapy Market, By Type

Allogeneic Stem Cell Therapy Market, By ApplicationAutologous Stem Cell Therapy Market, By Application

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Stem Cell Therapy Market Size, Business Opportunities and Forecast to 2029 The Oxford Spokesman - The Oxford Spokesman

DuBOIS Cayleigh Walkers hopes for a successful basketball season were almost benched when she tore her ACL playing summer league basketball at the YMCA in July 2021. However, the Clearfield High School sophomore resumed training just three weeks following her August surgery, fast tracking her return to the court with the Lady Bison varsity basketball team.

I was going up the court and felt it pop, Cayleigh recalled. I was in so much pain. I had to sit out the rest of the game while some of the parents tried to stretch it out. I was eager to have it examined because we were leaving for the beach two days later. Fortunately I was able to get an appointment to have it examined the next morning.

Cayleigh and her parents chose Penn Highlands Orthopedics and Sports Medicine for her care. At Penn Highlands, she was examined by Tyler Beers, PA-C, who tested her range of motion and felt around the injured area. He gave her a brace to wear and ordered an MRI which was scheduled the following week when the Walkers returned from their vacation.

The MRI revealed the torn ACL. The next day, Cayleigh met with Dr. Matthew A. Varacallo, medical director of Orthopedic Robotic Surgery at Penn Highlands Healthcare. The orthopedic surgeon specializes in sports medicine, total joint reconstruction, accelerated rehabilitation protocols and functional return to sports after surgeries and procedures.

Named to the Top 65 Total Knee Replacement Surgeons to Know by Beckers ACS Review, Dr. Varacallo pioneered the innovative Fertilized ACL technique along with Dr. Chad Lavender, an orthopedic surgeon at Marshall University in West Virginia. Currently, they are the only two surgeons in the US using the technique when performing ACL reconstruction surgery.

Typically, when an ACL tear occurs, one third of the athletes re-tear the same side or injure the other knee; but, the theory behind the Fertilized ACL technique is to improve and expedite bone tunnel healing rates, followed by graft revascularization and ligamentization (the process by which the tendon becomes a ligament) to improve graft function and incorporation into the knee joint, explained Dr. Varacallo.

The Fertilized ACL procedure begins when bone marrow is removed from the tibia the long bone on the inside of the lower leg which is rich in growth hormone and stem cells. A specialized perfusionist takes 60 milliliters of the bone marrow and spins it down to bone marrow aspiration concentrate (BMAC) which is used to initiate healing. The BMAC is mixed with bone graft. Small tunnels are then made in the femur and tibia to place the graft during the procedure. The bone graft mixture and stem cells/growth factors are then injected into the tunnels to help stimulate them to heal faster. Once the tunnels heal, the tendon can in theory possibly transform into a ligament faster.

This is truly a cutting edge procedure, explained Dr. Varacallo. In traditional ACL surgeries, the pressurized tunnels can be risk factors for re-injury because they can take up to six or seven months to heal. However, with the Fertilized ACL procedure, the tunnels heal faster because the graft starts to incorporate into the body faster. In fact, four weeks post surgery, you cannot even see the bone tunnels, the surgeon added.

Dr. Varacallo considers Cayleigh the poster child for successful ACL reconstruction because she is achieving milestones sooner than expected, and Samantha (Sam) Morgan, MS ATC PES, a certified athletic trainer with Penn Highlands Healthcare as well as with the Clearfield Area School District agrees.

Three days following surgery, Cayleigh began rehab, and at three and one half weeks post op she was running, and six to seven weeks following surgery she was punting a soccer ball. In contrast, with traditional ACL surgery, we would not see that type of progress until three or four months post op, Ms. Morgan detailed.

According to Dr. Varacallo, Cayleigh is several months ahead of where she should be in terms of her progress based on the functional testing ACL Report Card, The Report Card, which is used to measure how well a patient is meeting 15 different functional test milestones at two, four, six and eight months post op, was developed at the University of Kentucky where Dr. Varacallo completed his fellowship in Orthopedic Surgery and Sports Medicine.

The ACL Report Card is a great benchmark tool because it not only shows the medical team the patients progress, it gives the athlete something to shoot for in their rehab, said Dr. Varacallo.

I had four months of rehab five days a week, Cayleigh said, The first few weeks were challenging because I was in some pain, but Sam pushed me and helped me get through it.

At two months after surgery, Cayleigh was testing stronger on the surgery side than on the other non-injured side. One of the factors that contributed to her faster recovery was that prior to surgery she and the other patients who have Fertilized ACL reconstruction surgery have prehab.

We cannot operate on a stiff and weak knee, so prior to surgery, we have the patient undergo motion and strengthening exercises to improve outcomes following surgery, Dr. Varacallo explained. The stronger the knee heading into surgery, the faster the recovery.

In addition to enhancing flexibility, prehab teaches patients the exercises they will be doing during post-surgery rehab so that they are already familiar with what their physical therapy will entail.

Cayleigh had aggressive prehab, said Morgan. In addition to promoting flexibility of the knee, it is an educational tool that helps the patient understand the how and why of the injury and healing. Three days following surgery, Cayleigh started rehab, and because she had the prehab, she already knew how to perform her exercises.

The prehab, surgery and rehab are all interdependent on each other. In fact, Dr. Varacallo uses an analogy to tie the process together. I think of the reconstruction and healing process as a house the prehab is the foundation, surgery is the main living level and rehab is the attic.

According to Cayleighs mother, Tammy Walker, her daughters fast recovery is a combination of three factors, Dr. Varacallos innovative Fertilized ACL technique, the aggressive rehabilitation and equally important the determination by Cayleigh and Morgan to get her back to where she could play again.

We are very impressed with Dr. Varacallo, said Walker. He was very thorough and went through everything with us and even called to check on her progress following the surgery.

More here:
Penn Highlands Healthcare surgeon pioneers innovative ACL surgery offering faster recovery - The Courier-Express

Exclusive:

Football ace Ashley Cain and his girlfriend Safiyya Vorajee have been supporting parents of children battling cancer. The couple's baby girl Azaylia died last April

Image: Instagram)

Footballer Ashley Cain has been offering hope and solace to parents of a little girl battling the cancer that claimed the life of his own precious daughter.

Reality star Ashley and girlfriend Safiyya Vorajee have been supporting dozens of sick childrens families since the death of their baby girl.

Beloved Azaylia died last April after a courageous fight against the blood cancer acute myeloid leukaemia.

The ex-Coventry City winger and his partner have now struck up a special friendship with the family of five-year-old Esha Nadeswaran, who has spent eight months fighting the same disease at Londons Great Ormond Street Hospital.

Her dad Rishya, 45, of Gants Hill, North East London, said: Esha is our absolute hero and she is incredible. With our journey, it is like a marathon with no finish. We dont know when it is going to end and so we cant even pace ourselves.

There have been days where Ive gone to bed, crawled up and cried alone. But no matter how bad a day you are having and how much you hurt inside, Ashley reminded me you must stay positive and happy for your child.

He told me not to think too far ahead and do whatever I needed to do to support Esha. Its horrible to be told your child has cancer.

Listening to Ashley, and how vulnerable he felt, really struck a chord. Our chat has kept me going.

Former Ex On The Beach star Ashley, 31, and Safiyya, 34, were devastated to lose Azaylia when she was just eight months old.

She had been given stem cell treatment, which involves replacing damaged cells with healthy ones from a blood-match donor.

Ashley said Azaylia had the heart of a lion and he was given precious extra months with her after 100,000 people registered to be a donor.

The couple got involved in Eshas battle after Rishya and wife Kavitha, 41, appealed to members of the South Asian community to volunteer to be stem cell donors.

Esha has been in hospital since last May. She has had gruelling chemo and, in October, an emergency stem cell transplant. With her parents at her bedside, constant care includes regular platelet and red blood cell transfusions.

Rishya recalls the day they received the diagnosis. He says: She had been having tummy issues. Doctors ran tests and we were told wed receive the results in a fortnight. Two hours after returning home, they rang and we knew something was badly wrong. We rushed back and were told she had an acute form of leukaemia. It has been utterly heartbreaking.

Eshas Sri Lankan heritage meant it was hard to find a donor match and the family launched a social media campaign. Safiyya then reached out.

Rishya says: I arranged a Zoom chat with Safiyya and Ashley and I spoke father to father about what it feels like to be told your child has cancer. It was very emotional.

Ashley and Safiyya also recorded a video message on Eshas birthday.

Rishya, who works in banking, says: Esha really liked it. She is such a caring little girl and the way she has bounced back to defy doctors is something of a miracle.

She is always the one who goes from room to room cheering up the other children.

Rishya and his family now have 10,000 Instagram followers. But what they havent shared is their sacrifices to give her the best possible chance of survival.

Forming a Covid bubble, Rishya and his wife have had to live apart from elder daughter Ria, 10, who is with her grandparents.

They see her once a week at arms length and were apart at Christmas and on both girls birthdays. Its been really tough, says Rishya. My wife and I take it in turns to stay with Esha. The Young Lives vs Cancer charity has kindly been able to offer accommodation for the other to stay nearby.

We have been locked in this bubble for months now and its exhausting.

Ashley and Safiyya have helped dozens of parents. They set up a foundation raising money for treatment that may not be available on the NHS.

Image:

He says: No matter how painful it is, our mission is to really help and battle for these kids fighting cancer.

We speak to parents privately and try to guide them through the experience. We know first hand the stress and trauma parents have to go through.

Eshas dad and I talked about appreciating every single moment, making sure your child thinks theres nothing wrong. You need to stay positive and be the best you can be.

The pain of losing Azaylia is with us every minute. We go to her resting place every day and I want to hold this pain forever to use it effectively to help other children have a better chance.

Safiyya adds: So many children are suffering and we want to help.

The couple are planning a fundraising cycling event in June in conjunction with the Tour of Cambridgeshire.

Ashley says: Wed like everyone to wear orange and create a sea of orange so that Azalyia can look down from heaven and see what an amazing effect she has had on this world.

To join the cycle event, go to cycleforazaylia.com. Get updates on Esha at instagram.com/for_esha

Esha has the illness that cost Azaylia her life acute myeloid leukaemia, cancer of the white blood cells.

It starts in the bone marrow and spreads to the bloodstream and is very rare in young children.

The risk increases with age and it is most common in people aged over 75.

Acute leukaemia means it progresses quickly and chemotherapy is the main treatment, killing off as many unhealthy cells as possible and reducing the risk of the patient suffering a relapse.

Stem cell therapy involves removing a persons unhealthy cells and replacing them with healthy ones from a donor.

The new cells travel in the blood of the patient to the bone marrow, where they attach and grow.

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Ashley Cain supports parents of girl, 5, battling same cancer which killed his baby - The Mirror

This article was originally published here

J Oncol Pharm Pract. 2022 Jan 27:10781552221074269. doi: 10.1177/10781552221074269. Online ahead of print.

ABSTRACT

INTRODUCTION: Plasma cell leukemia (PCL) is a rare but aggressive variant of multiple myeloma (MM) with a poor prognosis. Due to the limited number of prospective clinical trials studying PCL, treatment options are often extrapolated from data available for the treatment of MM. Venetoclax has recently demonstrated antimyeloma activity in patients with relapsed/refractory MM carrying the t(11;14) translocation. However, few cases have reported the analogous efficacy of venetoclax in PCL.

CASE REPORT: A 64-year-old Caucasian male developed relapsed PCL despite treatment with hyperCD (hyperfractionated cyclophosphamide and dexamethasone) and Dara-KRd (daratumumab, carfilzomib, lenalidomide, dexamethasone). Due to the refractory nature of his disease and the presence of a t(11:14) translocation, the patient was subsequently initiated on venetoclax 400 mg daily and dexamethasone 4 mg once weekly.

MANAGEMENT AND OUTCOME: The patient achieved a complete response by International Myeloma Working Group criteria three months after initiating venetoclax-dexamethasone, including a repeat bone marrow biopsy that showed no abnormal plasma cells. He successfully underwent consolidation with melphalan-based autologous stem cell transplantation. He remains disease-free 9 months after venetoclax initiation.

DISCUSSION: Combination all-oral therapy with venetoclax and dexamethasone can induce deep hematologic responses in patients with relapsed/refractory PCL carrying the t(11;14) translocation.

PMID:35084252 | DOI:10.1177/10781552221074269

Original post:
Complete response following treatment of plasma cell leukemia with venetoclax and dexamethasone: A case report - DocWire News

In 2017, the Food and Drug Administration closed a loophole exploited by clinics pitching unproven, ineffective and potentially hazardous stem cell therapies directly to consumers.

Those treatments were illegal, the FDA ruled. That was the good news. The agency, however, suspended its enforcement for three years to give these operators time to get right with its regulations. During the pandemic, the FDA added six months to the deadline, so its period of regulatory forbearance expired on May 31.

What happened in the meantime? Instead of stem cell purveyors reaching out to the FDA to work out how to meet federal regulations, a torrent of shady operations poured into the field so many that the task of protecting the public from them may now exceed the FDAs capabilities.

Dont believe the hype.

The FDAs warnings against unlicensed stem cell clinics

Thats the concern of Leigh Turner, a public health expert at UC Irvine, longtime critic of stem cell treatment claims and author of a new study that tracked the explosion of businesses offering purported stem cell treatments and cures during the FDAs hands-off period.

The paper is essentially a follow-up to a seminal study Turner conducted with Paul Knoepfler of UC Davis in 2016, which identified 351 businesses hawking stem cell treatments directly to consumers through 540 clinics.

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Turners new study, which was published Thursday in the peer-reviewed journal Cell Stem Cell, identified 1,480 businesses operating 2,754 clinics nationwide.

That hardly seems like progress, Turner told me. Now the problem the FDA faces is four times larger than what existed in 2016. The FDA only has so many employees and so many inspectors. They dont really have enough inspectors to send them to 1,480 businesses.

The FDA hasnt been entirely inactive. Over the last 3 years it issued more than 400 warning letters to stem cell marketers, clinics and healthcare providers, notifying them that they may be operating outside the law. But it hasnt done much more than that.

The proliferation of stem cell claims points to a major breakdown in Americas healthcare regulatory system generally.

Given the size of this clinic industry the risk to the public is huge, Knoepfler says. The industry threatens the authority of the FDA itself. It might be one of the largest and most serious noncompliance challenges that the FDA has ever faced in its history. What would the FDA do if pharmaceutical firms were selling unapproved drugs at 2,700 clinics all over the country? It would be considered a national emergency.

State medical boards, which have at least nominal authority to ride herd on the practices of licensed physicians in this field, have done little of note. The Medical Board of California, a state that with 347 clinics is the largest center of facilities in Turners database, created a two-member Stem Cell and Regenerative Therapy Task Force in 2018.

The task force hasnt met since 2019, according to a board spokesman, and hasnt issued any reports. Board records indicate that it received 33 complaints about stem cell treatments from 2018 through this year, and has taken no disciplinary or administrative actions in response.

California hasnt done much to rise to the challenge, Turner says.

A few words may be useful about the regulatory environment. The chief targets of the FDAs enforcement program, such as it is, have been clinics that extract fat cells from customers through liposuction and then supposedly extract stem cells from the fat and inject them back into the customers bodies as treatments. Some purportedly extract stem cells from customers bone marrow.

The conditions for which these treatments are commonly offered include pain, sports injuries, heart and lung disease, multiple sclerosis, Parkinsons, Alzheimers, autism, diabetes, vision loss and erectile dysfunction.

No scientifically validated evidence exists for any of these claims, the FDA notes. The only stem cell products approved by the FDA are a few derived from umbilical cord blood, and then only for a very limited category of blood system diseases.

Beyond that, the agency advises consumers, Dont believe the hype.

The purveyors of unproven and unlicensed treatments identified by Turner charge as much as $28,000 for their services, with an average of more than $5,000 often ponied up by unwary customers seduced by advertising and irresponsibly credulous reports in the press.

The treatments typically are not covered by insurance, so customers are paying out of pocket.

Under FDA regulations, most stem cell treatments being sold to customers are illegal. Thats because the products are deemed to be unlicensed drugs. Exceptions exist for some surgical procedures and in cases in which almost identical cells are reinjected into patients, but the FDA says few of the targeted clinics qualify.

The FDAs position was endorsed by a federal judge in Miami in 2019, when she shut down a clinic that the FDA had sued for offering unapproved stem cell procedures. The clinic lost an appeal of her ruling in June. A separate lawsuit the FDA filed against California Stem Cell Treatment Center and associated businesses is awaiting a verdict from U.S. Judge Jesus G. Bernal in Riverside following a trial he conducted in May.

The offered treatments are not only unproven, but potentially hazardous. Reports of adverse outcomes from unlicensed treatments have proliferated, some of them gruesome. In a report issued in June, researchers at the Pew Charitable Trusts documented reports of adverse outcomes from 360 patients between 2004 and mid-2020.

These figures are almost certainly conservative, as clinics operating outside the law are highly unlikely to follow rules mandating that they report adverse reactions among their customers. Indeed, the FDA in its lawsuit against the California defendants asserted that numerous adverse outcomes experienced by their patients were not reported to the agency.

A note cited by the FDA from the file of one patient who was unable to walk for six months after receiving a stem cell injection in her knee from a clinic associated with the defendants, read, Not all treatments are successful. Not really adverse event due to the treatment. At trial, defendant Mark Berman said the clinics product has had very rare adverse events.

FDA officials have said that their expectations that stem cell treatment purveyors would engage with the agency during the forbearance period to work out how to come into compliance with its regulations proved wildly optimistic. We have been very disappointed in the number of clinics that have come in, Wilson Bryan, a top FDA official, told a law conference in June.

The FDA may not have recognized that the clinics they were targeting never had any intention of meeting its regulations.

Were talking about a huge number of businesses that are failing to comply with federal law, but have no reasonable prospects of coming into compliance, Turner says. They dont have clinical research programs. They dont have qualified stem cell researchers. Theyre just peddlers putting out a shingle on the internet.

Instead of using that three-year period to change their practices and comply with the law, lots of businesses stayed in the marketplace and did nothing to change what they were doing, and a huge number of other operators poured into the marketplace.

Over the last five years or so, or since Turner and Knoepfler published their original report, stem cell treatment claims have become just one more offering by practitioners advertising other services of dubious effectiveness.

Stem cell treatment has become just a routine claim, Turner says, from all kinds of orthopedic clinics, sports medicine clinics, podiatrists, chiropractors, naturopaths, wellness clinics. They market an array of services and stem cells are just something else they can sell, like cryotherapy or acupuncture.

Its unclear whether some of these operations really even use stem cells some may be offering just costly placebos, Turner conjectures.

The threat to public health from unlicensed and unproven stem cell claims will only get worse if the FDA fails to act forcefully.

People make the mistake of thinking that these are businesses that will go under if they get a bit of a push from the FDA or FTC [Federal Trade Commission] Turner says. The truth is that some of them are quite well capitalized and have the resources to go out and join these battles.

The FDA may be waiting for Judge Bernals ruling before taking the next step but thats dependent on his finding in the FDAs favor. It could seek mass injunctions, admittedly a big practical challenge, Knoepfler says. He adds, Looking ahead, the agency should take quick, large-scale, and even creative actions if it has any hope to make a dent in this clinic industry.

Read more from the original source:
Hiltzik: The battle against unlicensed stem cell clinics - Los Angeles Times

PlateletBio, a company developing a new class of cell therapies based on the biology of platelets, has raised $75.5 million to advance its drug pipeline, including a lead candidate for a rare bleeding disorder on track to reach the clinic next year.

Platelets are components of blood best known for their role forming clots that stop bleeding. But Watertown, Massachusetts-based PlateletBio notes that platelets have other properties, including a role delivering growth factors and proteins throughout the body. PlateletBio is developing therapies that take advantage of these properties, but rather than using platelets from a patient or healthy donors, the startup makes them.

In the body, platelets are formed in bone marrow. PlateletBio produces its platelet-like cells, or PLCs, inside a bioreactor that mimics bone marrow conditions. The source material for its PLCs are stem cells, which have the ability to become almost any cell or tissue in the body.

Platelets are technically not cells. They dont have a nucleus, but thats an advantage for therapeutic applications. Since a PlateletBio therapy wont introduce DNA into a patients body, the potential risks that come from introducing foreign genetic material are avoided. PlateletBio says it can produce PLCs with new features and therapeutic payloads that include antibodies, signaling proteins, therapeutic proteins, and nucleic acids.

PlateletBios lead cell therapy candidate is being developed to treat immune thrombocytopenia, a blood disorder in which the immune system mistakenly sees a patients platelets as foreign and destroys them. Immune thrombocytopenia patients have dangerously low platelet counts that make them susceptible to bleeding.

There is no FDA-approved treatment for the underlying cause of immune thrombocytopenia, but corticosteroids are used to try to dampen the immune systems attack on platelets. Platelet transfusions are another option, but the National Organization for Rare Disorders notes that these treatments are usually reserved for emergencies because the platelets are likely to be destroyed by antibodies produced by the patient.

Patients who have not responded to earlier treatments have two FDA-approved small molecule options: Tavalisse, from Rigel Pharmaceuticals, and the Swedish Orphan Biovitrum drug Doptelet. Sanofi aims to treat the disease with a small molecule called rilzabrutinib. That drug is designed to block Brutons tyrosine kinase, a protein that plays a role in the development of a B cells, a type of immune cell. Sanofi acquired the molecule last year via its $3.7 billion acquisition of Principia Biopharma.

The lead disease target for the Principia drug was multiple sclerosis. In September, Sanofi reported that rilzabrutinib failed that Phase 3 study. A separate Phase 3 test in immune thrombocytopenia is ongoing, as is a mid-stage clinical trial in another autoimmune condition called IgG4-related disease.

PlateletBio isnt the only company trying to turn a component of the blood into a new type of cell therapy. Cambridge, Massachusetts-based Rubius Therapeutics is developing cell therapies based on red blood cells. After disappointing early clinical trial results in the rare disease phenylketonuria last year, Rubius shifted its focus to cancer and immune system disorders. PlateletBios PLCs would represent an entirely new approach to treating immune thrombocytopenia. According to PlateletBios website, the company plans to file an investigational new drug application for its therapeutic candidate in the first half of next year.

PlateletBio is based on the research of Harvard University scientist Joseph Italiano, who co-founded the company under the name Platelet BioGenesis. When the startup emerged in 2017, it was developing platelets that could address the platelet shortage problem facing blood donation centers. Two years ago, the startup expanded its Series A round with $26 million in additional financing and plans to make its platelets into cell therapies. Besides immune thrombocytopenia, other diseases the biotech aims to treat include osteoarthritis and liver fibrosis.

PlateletBios latest financing, a Series B round, adds new investors SymBiosis, K2 HealthVentures, and Oxford Finance. Earlier investors Ziff Capital Partners and Qiming Venture Partners also participated in the new round.

This is a major milestone for PlateletBio, adding capital and resources needed to advance our innovative platelet-like cell therapy science and manufacturing platform and support key corporate initiatives over the next 18 to 24 months, Sam Rasty, the startups president and CEO, said in a prepared statement.

Photo by Flickr user Marco Verch via a Creative Commons license

See the original post:
Cell therapy biotech PlateletBio reels in $75M as it looks ahead to first clinical test - MedCity News

TEL AVIV, Israel, Nov. 4, 2021 /PRNewswire/ --BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical Company focused on oncology, today announced an oral presentation and three poster presentations at the 63rd American Society of Hematology (ASH) Annual Meeting & Exposition, which is being held December 11-14, 2021 in Atlanta, GA, and virtually.

The oral presentation will elaborate on the successful results of the Company's GENESIS Phase 3 pivotal trial. The study showed highly significant and clinically meaningful results supporting the use of Motixafortide on top of G-CSF for mobilization of stem cells for subsequent collection and transplantation in patients with multiple myeloma. In addition, the poster presentations will show that extended inhibition of the CXCR4 receptor by Motixafortide results in the mobilization of high numbers of stem cells, including specific sub-populations, which were correlated with reduced time to engraftment when infused in high numbers.

The Company is also presenting findings from in-vivo and in-vitro pre-clinical studies demonstrating that Motixafortide acts as an immunomodulator by affecting the biology of regulatory T cells (Tregs), supporting biomarker findings from the Company's COMBAT Phase 2 study in pancreatic cancer patients.

"We are very pleased with the breadth of our oral and poster presentations at this year's ASH meeting, which reflect the versatility of Motixafortide as the potential backbone of promising new treatments for both hematological and solid tumor cancers," stated Philip Serlin, Chief Executive Officer of BioLineRx. "Of particular note is the oral presentation on the outstanding results from our GENESIS Phase 3 pivotal study in stem cell mobilization demonstrating that Motixafortide effectively mobilizes a high number of cells enabling ~90% of patients to undergo transplantation following a single administration of Motixafortide and a single apheresis session. In addition, the high number of cells mobilized by Motixafortide enables infusion of an optimal number of cells, which could result in faster time to engraftment, and also allows for cryopreservation for future transplantation(s). These results, together with our recently completed successful pharmacoeconomic study, strongly support our view that Motixafortide on top of G-CSF can become the new standard of care in SCM, if approved, to the benefit of patients and payers alike. We look forward to submitting an NDA in the first half of next year, as previously communicated."

Further details of the presentations are provided below.

Oral Presentation

Title: Motixafortide (BL-8040) and G-CSF Versus Placebo and G-CSF to Mobilize Hematopoietic Stem Cells for Autologous Stem Cell Transplantation in Patients with Multiple Myeloma: The GENESIS Trial

Date: Sunday, December 12, 2021

Time: 12:00 PM

Location: Georgia World Congress Center, Hall A1

This oral presentation describes the GENESIS Phase 3 pivotal trial design, endpoints and results. The GENESIS study was a double blind, placebo controlled, multicenter trial, in which 122 patients were randomized (2:1) to receive either Motixafortide + G-CSF or placebo + G-CSF for stem cell mobilization prior to stem cell transplant in multiple myeloma patients. Total CD34+ cells/kg were analyzed on site to determine if patients mobilized to the goal and all samples were subsequently sent for assessment by a central laboratory. The number of CD34+ cells infused was determined independently by each investigator according to local practice.

The study concluded that a single administration of Motixafortide on top of G-CSF significantly increased the proportion of patients mobilizing 6x106 CD34+ cells/kg for stem cell transplantation (92.5%) vs G-CSF alone (26.2%) in up to two apheresis days (p<0.0001), while enabling 88.8% to collect 6x106 CD34+ cells/kg in just one apheresis day (vs 9.5% with G-CSF alone; p<0.0001). In addition, the median number of hematopoietic stem cells mobilized in one apheresis day with Motixafortide + G-CSF was 10.8x106 CD34+cells/kg vs 2.1x106 CD34+ cells/kg with G-CSF alone.

Poster Presentations

Title:Autologous Hematopoietic Cell Transplantation with Higher Doses of CD34+ Cells and Specific CD34+ Subsets Mobilized with Motixafortide and/or G-CSF is Associated with Rapid Engraftment A Post-hoc Analysis of the GENESIS Trial

Date: Sunday, December 12, 2021

Time: 6:00 PM - 8:00 PM

The CD34+ hematopoietic stem and progenitor cell (HSPC) dose infused during stem cell transplantation remains one of the most reliable clinical parameters to predict quality of engraftment. A minimum stem cell dose of 2-2.5x106 CD34+ cells/kg is considered necessary for reliable engraftment, while optimal doses of 5-6x106 CD34+ cells/kg are associated with faster engraftment, as well as fewer transfusions, infections, and antibiotic days.

An analysis was performed using pooled data from all patients in the GENESIS trial to evaluate time to engraftment based on the total number of CD34+ cells/kg infused, as well as specific numbers of CD34+ cell sub-populations infused.

The addition of Motixafortide to G-CSF enabled significantly more CD34+ cells to be collected in one apheresis (median 10.8x106 CD34+ cells/kg) compared to G-CSF alone (2.1x106 CD34+ cells/kg), as well as 3.5-5.6 fold higher numbers of hematopoietic stem cells (HSCs), multipotent progenitors (MPPs), common myeloid progenitors (CMPs) and granulocyte and macrophage progenitors (GMPs) (all p-values <0.0004). A dose response was observed with a significant correlation between faster time to engraftment and infusion of higher number of total CD34+ HSPC doses (6x106 CD34+ cells/kg) and combined HSC, MPP, CMP and GMP subsets. The high number of CD34+ cells/kg mobilized with Motixafortide on top of G-CSF enables the potential infusion of 6x106 CD34+ cells/kg, as well as cryopreservation of cells for later use.

Title: Immunophenotypic and Single-Cell Transcriptional Profiling of CD34+ Hematopoietic Stem and Progenitor Cells Mobilized with Motixafortide (BL-8040) and G-CSF Versus Plerixafor and GCSF Versus Placebo and G-CSF: A Correlative Study of the GENESIS Trial

Date: Monday, December 13, 2021

Time: 6:00 PM - 8:00 PM

CD34 expression remains the most common immunophenotypic cell surface marker defining human hematopoietic stem and progenitor cells (HSPCs). The addition of CXCR4 inhibitors to G-CSF has increased mobilization of CD34+ HSPCs for stem cell transplantation; yet the effect of CXCR4 inhibition, with or without G-CSF, on mobilization of specific immunophenotypic and transcriptional CD34+ HSPC subsets is not well-characterized.

Motixafortide is a novel cyclic peptide CXCR4 inhibitor with a low receptor-off rate and extended in vivo action when compared to plerixafor. GENESIS Phase 3 trial patients were prospectively randomized (2:1) to receive either Motixafortide + G-CSF or placebo + G-CSF for HSPC mobilization. Demographically similar multiple myeloma patients undergoing mobilization with plerixafor + G-CSF prior to stem cell transplant were prospectively enrolled in a separate tissue banking protocol.

Extended CXCR4 inhibition with Motixafortide + G-CSF mobilized significantly higher numbers of combined CD34+ HSCs, MPPs and CMPs compared to plerixafor + G-CSF or G-CSF alone (p<0.05). Additionally, Motixafortide + G-CSF mobilized a 10.5 fold higher number of immunophenotypically primitive CD34+ HSCs capable of broad multilineage hematopoietic reconstitution compared to G-CSF alone (p<0.0001) and similar numbers compared to plerixafor + G-CSF. Furthermore, lack of CXCR4 inhibition resulted in mobilization of more-differentiated HCSs, whereas extended CXCR4 inhibition with Motixafortide + G-CSF (but not plerixafor + G-CSF) mobilized a unique MPP-III subset expressing genes specifically related to leukocyte differentiation.

Title: The High Affinity CXCR4 Inhibitor, BL-8040, Impairs the Infiltration, Migration, Viability and Differentiation of Regulatory T Cells

Date: Sunday, December 12, 2021

Time: 6:00 PM - 8:00 PM

This poster describes results of pre-clinical in-vivo and in-vitro studies demonstrating that Motixafortide potentially acts as an immunomodulator by affecting the biology of regulatory T cells. Motixafortide reduced the amount of infiltrating Tregs into the tumors, impaired the migration of Tregs toward CXCL12 and induced Tregs cell death. Furthermore, Motixafortide was found to inhibit the differentiation of nave CD4 T cells toward Tregs.

About BioLineRx

BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a late clinical-stage biopharmaceutical company focused on oncology. The Company's business model is to in-license novel compounds, develop them through clinical stages, and then partner with pharmaceutical companies for further clinical development and/or commercialization.

The Company's lead program, Motixafortide (BL-8040), is a cancer therapy platform that was successfully evaluated in a Phase 3 study in stem cell mobilization for autologous bone-marrow transplantation, has reported positive results from a pre-planned pharmacoeconomic study, and is currently in preparations for an NDA submission. Motixafortide was also successfully evaluated in a Phase 2a study for the treatment of pancreatic cancer in combination with KEYTRUDA and chemotherapy under a clinical trial collaboration agreement with MSD (BioLineRx owns all rights to Motixafortide), and is currently being studied in combination with LIBTAYO and chemotherapy as a first-line PDAC therapy.

BioLineRx is also developing a second oncology program, AGI-134, an immunotherapy treatment for multiple solid tumors that is currently being investigated in a Phase 1/2a study.

For additional information on BioLineRx, please visit the Company's website at http://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Factors that could cause BioLineRx's actual results to differ materially from those expressed or implied in such forward-looking statements include, but are not limited to: the initiation, timing, progress and results of BioLineRx's preclinical studies, clinical trials and other therapeutic candidate development efforts; BioLineRx's ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; BioLineRx's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of BioLineRx's therapeutic candidates; BioLineRx's ability to establish and maintain corporate collaborations; BioLineRx's ability to integrate new therapeutic candidates and new personnel; the interpretation of the properties and characteristics of BioLineRx's therapeutic candidates and of the results obtained with its therapeutic candidates in preclinical studies or clinical trials; the implementation of BioLineRx's business model and strategic plans for its business and therapeutic candidates; the scope of protection BioLineRx is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; estimates of BioLineRx's expenses, future revenues, capital requirements and its needs for additional financing; risks related to changes in healthcare laws, rules and regulations in the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; risks related to the COVID-19 pandemic; and statements as to the impact of the political and security situation in Israel on BioLineRx's business. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on February 23, 2021. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contact:

Tim McCarthyLifeSci Advisors, LLC+1-212-915-2564[emailprotected]

or

Moran MeirLifeSci Advisors, LLC+972-54-476-4945[emailprotected]

SOURCE BioLineRx Ltd.

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BioLineRx Announces an Oral Presentation and Three Poster Presentations at the 63rd American Society of Hematology (ASH) Annual Meeting &...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines designed to bring the curative power of stem cell transplants to more patients, today announced positive top-line results from an investigator-initiated Phase 2 clinical trial of MGTA-145 stem cell mobilization in multiple myeloma. The data were accepted for a poster presentation at the 2021 American Society of Hematology (ASH) Annual Meeting, to be held in Atlanta and virtually from December 11-14, 2021. Oral and poster presentations of preclinical data related to the companys CD117 targeted conditioning program will also be made at the ASH Annual Meeting.

We have made significant progress with our mobilization and targeted conditioning programs and we look forward to the presentation of the data that have been generated to support both programs, said Jason Gardner, D.Phil., President and Chief Executive Officer, Magenta Therapeutics.

Stem Cell Mobilization and Collection Program (MGTA-145)

Poster Presentation Highlighting Investigator-Initiated Phase 2 Clinical Data of MGTA-145 Stem Cell Mobilization in Multiple Myeloma:

Title: MGTA-145 + Plerixafor Provides G-CSF-Free Rapid and Reliable Hematopoietic Stem Cell Mobilization for Autologous Stem Cell Transplant in Patients with Multiple Myeloma: A Phase 2 Study (Poster #3888)

Date and Time to View Poster Presentation: Monday, December 13, 2021, 6:00pm 8:00pm ET

Trial Design

Surbhi Sidana, M.D., Assistant Professor of Medicine in the Division of Blood and Marrow Transplantation and Cellular Therapy at Stanford University School of Medicine led this investigator-initiated, Phase 2 open-label clinical trial. The trial evaluated the ability of MGTA-145, in combination with plerixafor, to mobilize stem cells for autologous stem cell transplantation in patients with multiple myeloma. This trial had broad inclusion criteria and included the transplant-eligible population of patients with multiple myeloma who may have a variety of risk factors for mobilization.

Topline Clinical Data

Next Steps in Multiple Myeloma

As described in the companys third quarter earnings release, the results from this investigator-initiated trial represent a positive step forward in the development of MGTA-145, in combination with plerixafor, as a potential first line stem cell mobilization regimen. Based on the encouraging collection and engraftment data, the company intends to explore further development of MGTA-145 in a Phase 2b clinical setting. This approach would enable a comprehensive evaluation of the multiple myeloma patient population and allow for adjustments of dosing and administration which the company, in both cases, has identified as opportunities for optimization as a result of this investigator-initiated study and the companys other MGTA-145 development efforts.

While Dr. Sidana and her team are collecting and analyzing additional patient-level data, these topline results are encouraging and support further development of MGTA-145. commented Dr. Jeffrey Humphrey, M.D., the companys Chief Medical Officer. We believe this novel mobilization regimen has the potential to replace G-CSF regimens and to enable reliable, predictable, rapid and well-tolerated mobilization of stem cells for both transplant and gene therapies.

MGTA-145 is also being evaluated for its ability to mobilize stem cells for collection from donors for allogenic transplantation in patients with acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) in a Phase 2 clinical trial. The company is planning to open an additional Phase 2 clinical trial for mobilization and collection of stem cells for patients with sickle cell disease in December 2021.

Antibody-Drug Conjugate (ADC) Targeted Conditioning Program

Oral Presentation Showcasing Non-human Primate Data of Targeted ADC Conditioning for Gene Therapy

Title: CD117 Antibody Drug Conjugate-Based Conditioning Allows for Efficient Engraftment of Gene-Modified CD34+ Cells in a Rhesus Gene Therapy Model (Oral Abstract #560)

Presenting Author: Naoya Uchida, M.D., National Institutes of Health

Date: Sunday, December 12, 2021, 4:45pm ET

This preclinical study evaluated escalating doses of a tool CD117-ADC. As monotherapy conditioning, a single dose of the CD117-ADC allowed for efficient engraftment of gene-modified autologous stem cells in a rhesus model of gene therapy, without chemotherapy or radiation conditioning. Engraftment of gene-modified stem cells achieved with monotherapy CD117-ADC was robust and durable, equivalent to that achieved with four doses of myeloablative busulfan conditioning. Sustained gene expression of hemoglobin F was confirmed at the protein level in this CD117-ADC-conditioned rhesus transplant model of gene therapy for sickle cell disease. Compared to chemotherapy or radiation-based conditioning regimens, conditioning with monotherapy CD117-ADC could be both sufficiently potent and well tolerated to improve the safety and risk benefit profile for gene therapies that require stem cell transplantation.

Poster Presentation Highlighting Preclinical Data of Targeted ADC Conditioning Program:

Title: CD117-Targeted ADC, in Combination with Lymphodepleting Antibodies, Enables Allogeneic Hematopoietic Stem Cell Transplantation in Mice without Chemotherapy or Radiation (Poster #1682)

Presenting Author: Leanne Lanieri, M.S., Magenta Therapeutics, Inc.

Date to View Poster Presentation: Saturday, December 11, 2021, 5:30pm 7:30pm ET

This study evaluated the combination of a tool CD117-ADC with lymphodepleting antibodies as the conditioning regimen in a murine model of allogeneic HSC transplantation. The targeted conditioning regimen enabled complete donor chimerism in a fully mismatched allogeneic HSC transplant murine model, without use of chemotherapy or radiation. Antibody-based targeted conditioning regimens could offer a more favorable risk-benefit profile over chemotherapy and radiation-based conditioning regimens. An improved risk benefit profile, in turn, could extend the curative potential of allogeneic HSC transplantation to more patients with malignant and non-malignant diseases who otherwise would not be eligible for HSC transplantation.

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines designed to bring the curative power of stem cell transplants to more patients with blood cancers, genetic diseases and autoimmune diseases. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise and broad networks in the stem cell transplant community to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Massachusetts. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking Statements

This press release may contain forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Magentas future expectations, plans and prospects, including, without limitation, statements regarding expectations and plans for presenting pre-clinical and clinical data, the initiation of clinical trials or the results of ongoing and planned clinical trials, the development of product candidates and advancement of preclinical programs, projections regarding future revenues and financing performance, long-term growth, cash, cash equivalents and marketable securities, the anticipated timing of clinical trials and regulatory filings, the potential benefits of product candidates, the timing, progress and success of collaborations, as well as other statements containing the words anticipate, believe, continue, could, endeavor, estimate, expect, intend, may, might, plan, potential, predict, project, seek, should, target, will or would and similar expressions that constitute forward-looking statements under the Private Securities Litigation Reform Act of 1995. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical studies and in the availability and timing of data from ongoing clinical studies; whether interim results from a clinical trial will be predictive of the final results of the trial; whether results from preclinical studies or earlier clinical studies will be predictive of the results of future trials; the expected timing of submissions for regulatory approval or review by governmental authorities; discussions with governmental agencies such as the FDA; regulatory approvals to conduct trials or to market products; whether Magenta's cash resources will be sufficient to fund Magenta's foreseeable and unforeseeable operating expenses and capital expenditure requirements; risks, uncertainties and assumptions regarding the impact of the continuing COVID-19 pandemic on Magentas business, operations, strategy, goals and anticipated timelines, Magentas ongoing and planned preclinical activities, Magentas ability to initiate, enroll, conduct or complete ongoing and planned clinical trials, Magentas timelines for regulatory submissions and Magentas financial position; and other risks concerning Magenta's programs and operations are described in additional detail in its Annual Report on Form 10-K filed on March 3, 2021, as updated by Magentas most recent Quarterly Report on Form 10-Q, and its other filings made with the Securities and Exchange Commission from time to time. Although Magenta's forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Magenta. As a result, you are cautioned not to rely on these forward-looking statements. Any forward-looking statement made in this press release speaks only as of the date on which it is made. Magenta undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise.

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Magenta Therapeutics Announces Data Presentations Related to its Mobilization and Conditioning Programs at the 2021 American Society of Hematology...

Two kidney transplant patients who received a stem cell therapy developed by Talaris Therapeutics were able to come off all immunosuppressant drugs within a year, without any evidence of graft rejection.

The first findings from Talaris phase 3 trial of the cell therapy called FCR001 suggest it may be possible to eliminate the need entirely for patients to take what may be dozens of tablets daily after organ transplants, according to the US biotech.

While still preliminary, the experience with the two patients back up Talaris hope that giving a one-shot dose of FCR001 the day after an organ transplant could stimulate immune tolerance in the recipient, and avoid the side effects of current drug treatments such as infections, heart disease, and some forms of cancer.

The companys approach relies on administering haematopoietic stem cells from the individual who donated the organ, in order to generate what Talaris refers to as chimerism, with both donor and recipient cells present in the bone marrow. That allows the immune system to see the transplanted organ as self rather than foreign.

The first two recipients in Talaris FREEDOM-1 phase 3 trial had received FCR001 at least 12 months earlier, and showed stable kidney function, according to Talaris.

A larger group of five patients who were at least three months from the cell therapy maintained more than 50% chimerism in their T cells, which the biotech said was a sign of long-term, immunosuppression-free tolerance to the donated kidney in its phase 2 trials.

The FREEDOM-1 results reported at the American Society of Nephrology (ASN) meeting this week were accompanied by updated results from Talaris phase 2 study, in which all 26 patients originally weaned off immunosuppressants have continued to remain off them without rejecting their donated kidney.

Some transplant patients treated with Talaris therapy in earlier trials have now been off all immunosuppression for more than 12 years without signs of kidney rejection.

Talaris intends to enrol 120 subjects into the phase 3 trial, which is scheduled to generate results in 2023.

Earlier this year, Talaris raised $150 million via a Nasdaq listing that will be used to take FCR001 through the phase 3 programme in organ transplantation and as a treatment for rare autoimmune disease scleroderma.

It also recently started a phase 2 trial of the cell therapy to see if it is able to induce immune tolerance to a transplanted kidney in patients who received the transplant from a living donor up to a year prior to administration of FCR001.

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Talaris therapy ends need for immune drugs in transplant patients - - pharmaphorum

DUARTE, Calif.--(BUSINESS WIRE)--City of Hope announced today that it will present new research on bispecific antibodies at a press briefing during the ASH 63rd Annual Meeting and Exposition on Dec. 11 to 14 in Atlanta.

Other innovative City of Hope research on stem cell transplants and blood cancer treatments will also be presented during the conference organized by ASH. ASH is the world's largest professional society for clinicians and scientists around the world who are working on blood cancers and other hematological diseases.

City of Hope is finding new treatments for some of the hardest to treat cancers by accelerating innovative clinical research and therapies. The comprehensive cancer centers bone marrow transplant program is one of the largest and most successful in the nation and its chimeric antigen receptor (CAR) T cell therapy program is also focused on finding new therapies. City of Hope is leading other innovative immunotherapy treatments for blood cancers.

Elizabeth Budde, M.D, Ph.D., associate professor, City of Hope Division of Lymphoma, Department of Hematology & Hematopoietic Cell Transplantation, will present research on a bispecific antibody mosunetuzumab for relapsed/refractory follicular lymphoma at an ASH press briefing titled Immune System 1, Cancer 0: Advances in Immunotherapy on Saturday, Dec. 11 at 8:30 a.m. ET Follicular lymphoma is associated with frequent relapses and decreasing progression-free intervals with successive lines of conventional therapy. Later-line treatments may be less effective due to refractory disease. Mosunetuzumab is a CD20xCD3 bispecific antibody that redirects T cells to eliminate malignant B cells. In the dose-escalation phase of an ongoing Phase I/II study (NCT02500407), Mosunetuzumab was highly active and well tolerated in R/R FL patients (pts).

Dr. Budde will also present the research at a plenary session.

Title: Mosunetuzumab Monotherapy Is an Effective and Well-Tolerated Treatment Option for Patients with Relapsed/Refractory (R/R) Follicular Lymphoma (FL) Who Have Received 2 Prior Lines of Therapy: Pivotal Results from a Phase I/II Study Publication Number: 127Type: OralSession Name: 623. Mantle Cell, Follicular and Other B Cell Lymphomas: Clinical and Epidemiological: Evolution of Immunotherapeutic Regimens in B Cell LymphomasSession Date and Time: Saturday, Dec. 11, 2021, Noon to 1:30 p.m. ETPresentation Time: Saturday, Dec. 11, 2021, Noon ET

During the ASH conference, additional City of Hope researchers will also make presentations onsite or virtually:

Title: Pembrolizumab Plus Vorinostat Induces Responses in Patients with Hodgkin Lymphoma Who Are Refractory to Prior PD-1 Blockade Publication Number: 234Type: Oral.Session Name: 624. Hodgkin Lymphomas and T/NK cell Lymphomas: Hodgkin Lymphoma Clinical TrialsSession Date and Time: Saturday, Dec. 11, 2021, 2 to 3:30 p.m. ET Presentation Time: Saturday, Dec. 11, 2021, 3:15 p.m. ETPresenter: Alex Herrera, M.D., associate professor, City of Hope Division of Lymphoma, Department of Hematology & Hematopoietic Cell Transplantation

Herrera will also introduce an abstract at a plenary scientific session on Sunday, Dec. 12, 2 to 4 p.m. ET

Title: A Randomized Open Label Pilot Study of Clostridium Butyricum Miyairi 588 (CBM588) in Recipients of Allogeneic Hematopoietic Cell Transplantation Publication Number: 334Type: OralSession Name: 722. Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution: Infection and Immune ReconstitutionSession Date and Time: Saturday, Dec. 11, 2021, 4 to 5:30 p.m. ET Presentation Time: Saturday, Dec. 11, 2021, 4:45 p.m. ETPresenter: Karamjeet S. Sandhu, M.D., assistant professor, City of Hope Division of Leukemia, Department of Hematology & Hematopoietic Cell Transplantation

Title: The Impact of Somatic Mutations on Allogeneic Hematopoietic Cell Transplantation in Chronic Myelomonocytic Leukemia: A Center for International Blood and Marrow Transplant Research (CIBMTR) Analysis Publication Number: 417Type: OralSession Name: 732. Allogeneic Transplantation: Disease Response and Comparative Treatment Studies: Prognostic Biomarkers for Donor Selection and Recipient OutcomesSession Date and Time: Sunday, Dec. 12, 2021, 9:30 a.m. to 11 a.m. ET Presentation Time: Sunday, Dec. 12, 2021, 10 a.m. ETPresenter: Matthew G. Mei, M.D., associate professor, City of Hope Division of Lymphoma, Department of Hematology & Hematopoietic Cell Transplantation

In addition, Andrew Artz, M.D., M.S., professor, City of Hope Division of Leukemia, Department of Hematology & Hematopoietic Cell Transplantation, will speak at an education session titled How Can We Ensure That Everyone Who Needs a Transplant Can Get One? about allogeneic hematopoietic cell transplantation for older adults and will also give opening remarks a scientific workshop on hematology and aging.

About City of Hope

City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin, monoclonal antibodies and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope is ranked among the nations Best Hospitals in cancer by U.S. News & World Report. Its main campus is located near Los Angeles, with additional locations throughout Southern California and in Arizona. Translational Genomics Research Institute (TGen) became a part of City of Hope in 2016. AccessHope, a subsidiary launched in 2019, serves employers and their health care partners by providing access to NCI-designated cancer center expertise. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

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City of Hope Doctors Present New Research on Cancer Immunotherapies - Business Wire

Success Story of Extracorporeal Shock Wave Therapy (ESWT)

Successful for over 40 years in urology for the disintegration of kidney stones, with high efficiency and hardly any side effects worth mentioning.

How does the shock wave work?

Without causing mechanical damage, shockwaves trigger a biological response in the treated tissue through their compressive, tensile and shear forces (mechanotransduction). Genes are activated in the cell nucleus starting to produce proteins (including growth factors) responsible for the healing process. This also causes increased ingrowth of newly formed blood vessels, which improves local metabolism. The additional modulation of the inflammation necessary for healing enables regeneration of pathological tissue.

Recent studies prove.

Shockwaves also trigger the production of messenger substances to the cell nucleus, which mobilize the body's own stem cells from the bone marrow, stimulating them to migrate to the treated tissue, settle there and develop into the required tissue (e.g. heart muscle cells). Instead of conventional stem cell transplantation shockwaves make it possible to initiate the body's own regeneration without risk of complications.

Therapy for a wide variety of tissues.

Since the underlying pathology can be treated with these methods, shockwave therapy is being used in more and more medical disciplines.

This creates a tool that opens up completely new possibilities in tissue regeneration without triggering significant side effects. Since conventional medicine has not been able to offer any significant therapeutic options to date, the present results of shockwave therapy are of particular importance and are therefore applied in the following areas. It can be assumed that shockwave therapy can be used in practically all medical specialties.

Spinal cord injury/cross-sectional lesion.

What was long considered unthinkable is now one of the major hopes for causal therapy: shockwave has also made great progress in the treatment of paraplegia. Since November 2020, the first patients have been included in an Austria-wide study. Due to the COVID pandemic, the initiation of the individual study centers has been somewhat delayed, but so far eight patients have already been enrolled in the study. In addition, the Unfallkrankenhaus Berlin, one of the most important centers for spinal cord injury in Germany, will participate in the study.

Dr. Wolfgang Schaden, adj. Prof., President of ISMST, Ludwig Boltzmann Institute for Experimental and Clinical Traumatology, Deputy Medical Director of AUVA, Austria

Cardiac Surgery.

Regeneration of heart muscle after myocardial infarction has long remained a dream of modern medicine. Despite extensive efforts to develop stem cell and gene therapies, none of these methods could be brought into clinical routine. Cardiac shockwave therapy brings a scientific breakthrough: Cardiac function is improved, and impressive results show the increase of patients' quality of life. Shockwave therapy in cardiac surgery has a favorable side effect profile and is on the verge of bringing cardiac regeneration into daily clinical practice.

PD Dr. Johannes Holfeld, University Department of Cardiac Surgery, Innsbruck Medical University, Austria.

Sexual Medicine.

Low-energy shockwave therapy has been a fabulous addition to sexual medicine armamentarium for men and women with various forms of sexual dysfunction, e.g. erectile dysfunction, premature ejaculation, persistent genital arousal disorder PGAD/genito-pelvic dysesthesia GPD. Many patients (and their partners) describe these comfortable and quick shockwave treatments as life changing.

Prof. Dr. Irwin Goldstein, Alvarado Hospital, San Diego, CA, USA

Aesthetic-, hand-, burn- and reconstructive surgery.

Shockwave medicine can support these four pillars of surgery noninvasively. Two significant examples: In aesthetic surgery with significant improvement in cellulite with shockwave therapy after six to eight sessions, lasting for a period of about one year. In burns, shockwave therapy can accelerate epithelialization (healing) of superficial burn injuries clinically relevant by three days, with a significant reduction in infections and hospitalization.

Prof. Dr. Karsten Knobloch, SportPraxis Prof. Knobloch, Hanover, Secretary General of the German Shockwave Society DIGEST.

Sports Medicine.

After more than 30 years of experience, shock wave treatment is now a standard in sports medicine and rehabilitation facilities worldwide.

Leprosy.

Shockwaves used in a similar way as for diabetic foot ulcers have also led to the healing of wounds in leprosy patients and significantly improved the quality of life of these patients. This work, carried out in Agua de Dios, Colombia, by the Bosque University group in Bogot, is now being used in several medical centers around the world with very positive results.

Prof. Dr. Carlos Leal, Bosque University, Fenway Medical, Bogot, Colombia.

Wound healing.

Chronic wounds are challenging for patients concerned and practitioners and will have an increasing impact on health care systems. Treatment with shockwaves has a positive conditioning influence on the wounds and in a high proportion for healing, independent of otherwise aggravating factors (e.g. diabetes mellitus, immunosuppression, cortisone therapy, and other exacerbating factors).

With an average treatment frequency of one treatment every second week, in addition to the established wound therapy, healing was observed in more than 70% of the cases of ulcers and other wound healing disorders.The therapy is free of side effects and helps to reduce the burden of the health care system due to the enormous savings potential.

Rainer Mittermayr, MD, MBA, assoc. Prof., Treasurer of the ISMST and Conference Secretary, Senior Surgeon Orthopedic and Traumatology, AUVA (Austrian Workers Compensation Board, Vienna, Austria)

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Shockwave therapy brings new healing opportunities for heart attack patients and hope for people with spinal cord injuries - KULR-TV

Common stock will begin trading on The Nasdaq Capital Market under the ticker symbol BRTX November 5, 2021

MELVILLE, N.Y., Nov. 04, 2021 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (the Company") (NASDAQ:BRTX), a life sciences company focused on adult stem cell-based therapies, today announced the pricing of the underwritten public offering of 2,300,000 units, each consisting of one share of its common stock and a warrant to purchase one share of its common stock at a per unit price of $10.00. The warrants have a per share exercise price of $10.00, are exercisable immediately, and expire five years from the date of issuance. The aggregate gross proceeds from the offering are expected to total $23 million, before deducting the underwriting discounts and commissions and estimated offering expenses payable by the Company and without giving effect to proceeds from any subsequent exercise of warrants.

As a result of the offering, the Companys common stock will become listed on the Nasdaq Capital Market and will trade under the ticker symbol BRTX beginning November 5, 2021. The offering is expected to close on or about November 9, 2021, subject to customary closing conditions. In addition, the Company has granted to the underwriters of the offering a 45-day option to purchase up to 345,000 additional shares and/or additional warrants to purchase up to 345,000 shares of common stock to cover over-allotments, if any.

Roth Capital Partners is acting as sole manager for the offering.

BioRestorative Therapies advancement to The Nasdaq Capital Market continues a year of growth and accomplishment for our company during which time we emerged from Chapter 11 reorganization, transformed our business, strengthened our financial position and enhanced our IP position said Lance Alstodt, President and Chief Executive Officer of BioRestorative.

The securities described above are being sold by BioRestorative Therapies pursuant to a registration statement on Form S-1 (Registration No. 333-258611) that was previously filed by BioRestorative Therapies with the Securities and Exchange Commission (the SEC) and declared effective on November 4, 2021 and an additional registration statement filed pursuant to Rule 462(b), which became effective upon filing. This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

The offering is being made only by means of the written prospectus forming part of the effective registration statement. Electronic copies of the accompanying prospectus may be obtained, when available, by contacting Roth Capital Partners, 888 San Clemente, Newport Beach, CA 92660, Attn: Prospectus Department, telephone: 800-678-9147, or email at rothecm@roth.com, or by visiting the SECs website at http://www.sec.gov.

About BioRestorative Therapies, Inc.BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

Disc/Spine Program (brtxDISC): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders or as a complementary therapeutic to a surgical procedure. The BRTX-100 production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat chronic lower back pain arising from degenerative disc disease.

Metabolic Program (ThermoStem): We are developing a cell-based therapy candidate to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in animals may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Company's latest Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

CONTACT:

Email: ir@biorestorative.com

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BioRestorative Therapies Prices $23 Million Public Offering - GlobeNewswire

Updates from OTL-201 Clinical Proof-of-Concept Study in MPS-IIIA and OTL-204 Preclinical Study for GRN-FTD at ESGCT Showcase Potential for HSC Gene Therapy in Multiple Neurodegenerative Disorders

Launch Activities for Libmeldy Across Key European Countries, including Reimbursement Discussions, Progressing in Anticipation of Treating Commercial Patients

Frank Thomas, President and Chief Operating Officer, to Step Down Following Transition in 2022; Search for a Chief Financial Officer Initiated

Cash and Investments of Approximately $254M Provide Runway into First Half 2023

BOSTONandLONDON, Nov. 04, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today reported financial results for the quarter ended September 30, 2021, as well as recent business updates and upcoming milestones.

This quarter, we are pleased by the progress demonstrated by our investigational neurometabolic HSC gene therapy programs with promising preclinical and clinical updates at ESGCT, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With follow-up in OTL-201 for MPS-IIIA patients now ranging between 6 and 12 months, biomarker data remain highly encouraging, showing supraphysiological enzyme activity and corresponding substrate reductions in the CSF and urine. The launch strategy for Libmeldy is also advancing in Europe with momentum building on reimbursement discussions and patient finding activities.

Recent Presentations and Business Updates

Data presentations at ESGCT

Clinical and pre-clinical data from across the companys investigational hematopoietic stem cell (HSC) gene therapy portfolio were featured in two oral and seven poster presentations at the European Society of Gene & Cell Therapy Congress (ESGCT) on October 19-22. Highlights from key presentations are summarized below:

OTL-201 for Mucopolysaccharidosis type IIIA (MPS-IIIA): A poster presentation featured supportive biomarker data from the first four patients with evaluable results, with duration of follow-up ranging from 6 to 12 months. The treatment has been generally well-tolerated in all enrolled patients (n=5) with no treatment-related serious adverse events (SAEs). Supraphysiological N-sulphoglucosamine sulphohydrolase ( SGSH) enzyme activity above the normal range was seen in leukocytes and plasma within one to three months in all evaluable patients (n=4).A greater than 90% reduction in urinary glycosaminoglycans (GAGs) was seen within three months in all evaluable patients (n=4).SGSH activity in the cerebrospinal fluid (CSF) increased from undetectable at baseline to within or above the normal range by six months in all patients with available data (n=3).CSF GAGs decreased from baseline in patients with available data (n=3).OTL-204 for Progranulin-mutated Frontotemporal Dementia (GRN-FTD): Preliminary in vivo data from the preclinical proof-of-concept study showed that murine GRN -/- HSPCs, transduced with an LV expressing progranulin under the control of a novel promoter, are able to engraft and repopulate the brain myeloid compartment of FTD mice and to locally deliver the GRN enzyme.

R&D Investor Event Summary

In September, Orchard hosted an R&D investor event highlighting its discovery and research engine in HSC gene therapy, including an update on the OTL-104 program in development for NOD2 Crohns disease (NOD2-CD) and potential new applications in HSC-generated antigen-specific regulatory T-cells (Tregs) and HSC-vectorization of monoclonal antibodies (mAbs).

The discussion also covered the differentiated profile of Orchards HSC gene therapy approach, which has exhibited favorable safety, long-term durability and broad treatment applicability.

In particular, Orchards lentiviral vector-based HSC gene therapy programs have shown no indication of insertional oncogenesis and no evidence of clonal dominance due to integration into oncogenes. Importantly, the promoters and regulatory elements of Orchard vectors are derived from human (not viral) sequences and are specifically designed to have limited enhancer activity on neighboring genes thereby mitigating the potential for safety concerns.In addition, because of the fundamental biological differences between the HSC and adeno-associated virus (AAV) gene therapy approaches, Orchards programs have not, to date, seen the safety and durability concerns experienced by the AAV gene therapy field.

Libmeldy (atidarsagene autotemcel) launch in Europe

Orchard is providing an update on the following key launch activities for Libmeldy in Europe:

Discussions with health authorities and payors are underway across Europe in key markets including Germany, the UK, France and Italy.Qualification of treatment centers is progressing with The University of Tbingen in Germany ready to treat commercial patients and other centers in the final stages of qualification and treatment readiness.Disease awareness and patient identification activities continue and have supported patient referrals in major European centers. Orchards partnerships in the Middle East and Turkey allow for opportunities to treat eligible patients from these territories at qualified European centers.Orchard is providing sponsorship for an ongoing newborn screening pilot in Germany and is working with laboratories to implement pilots in Italy, the UK, France and Spain.

Executive organizational update

The company also announced that Frank Thomas will step down from his role as president and chief operating officer, following a transition in 2022. A search for a chief financial officer is underway. Mr. Thomas other responsibilities will be assumed by existing members of the leadership team in commercial and corporate affairs. Orchard recently strengthened the executive team with the appointments of Nicoletta Loggia as chief technical officer and Fulvio Mavilio as chief scientific officer and the promotion of Leslie Meltzer to chief medical officer.

I want to extend my gratitude to Frank Thomas for his immense contributions to Orchard, said Gaspar. During his tenure, Frank oversaw the transition of the organization to a publicly traded company and has managed operations with a focus on cross-company innovation, including his role as a key architect in creating and executing the focused business plan we rolled out in 2020. Along with the entire board of directors and leadership team, I appreciate Franks commitment to facilitate a smooth transition during this time.

Gaspar continued, Our search is focused on a CFO to lead the broad strategic planning efforts necessary to capitalize on the full potential of our hematopoietic stem cell gene therapy platform. We have a strong team in place to aid Orchards success in this next phase of growth and are well capitalized through the anticipated completion of several value-creating milestones.

Upcoming Milestones

In June 2021, Orchard announced several portfolio updates following recent regulatory interactions for the companys investigational programs in metachromatic leukodystrophy (MLD), Mucopolysaccharidosis type I Hurler syndrome (MPS-IH) and Wiskott-Aldrich syndrome (WAS).

OTL-200 for MLD in the U.S: Based on feedback received from the U.S. Food and Drug Administration (FDA), the company is preparing for a Biologics License Application (BLA) filing for OTL-200 in pre-symptomatic, early-onset MLD in late 2022 or early 2023, using data from existing OTL-200 patients. This approach and timeline are subject to the successful completion of activities remaining in advance of an expected pre-BLA meeting with FDA, including future CMC regulatory interactions and demonstration of the natural history data as a representative comparator for the treated population.OTL-203 for MPS-IH: Orchard is incorporating feedback from FDA and the European Medicines Agency (EMA) into a revised global registrational study protocol, with study initiation expected to occur in 2022.OTL-201 for MPS-IIIA: Additional interim data from this proof-of-concept study are expected to be presented at medical meetings in 2022, including early clinical outcomes of cognitive function.OTL-103 for WAS: The company expects a MAA submission with EMA for OTL-103 in WAS in 2022, subject to the completion of work remaining on potency assay validation and further dialogue with EMA. The company will provide updated guidance for a BLA submission in the U.S. following additional FDA regulatory interactions.

Third Quarter 2021 Financial Results

Revenue from product sales of Strimvelis were $0.7 million for the third quarter of 2021 compared to $2.0 million in the same period in 2020, and cost of product sales were $0.2 million for the third quarter of 2021 compared to $0.7 million in the same period in 2020. Collaboration revenue was $0.5 million for the third quarter of 2021, resulting from the collaboration with Pharming Group N.V. entered into in July 2021. This revenue represents expected reimbursements for preclinical studies and a portion of the $17.5 million upfront consideration received by Orchard under the collaboration, which will be amortized over the expected duration of the agreement.

Research and development (R&D) expenses were $20.8 million for the third quarter of 2021, compared to $14.7 million in the same period in 2020. The increase was primarily due to higher manufacturing and process development costs for the companys neurometabolic programs and lower R&D tax credits as compared to the same period in 2020. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and development milestone payments under the companys agreements with third parties, and personnel costs to support these activities.

Selling, general and administrative (SG&A) expenses were $13.0 million for the third quarter of 2021, compared to $13.0 million in the same period in 2020. SG&A expenses are expected to increase in future periods as the company builds out its commercial infrastructure globally to support additional product launches following regulatory approvals.

Net loss was $36.4 million for the third quarter of 2021, compared to $20.3 million in the same period in 2020. The increase in net loss as compared to the prior year was primarily due to higher R&D expenses as well as the impact of foreign currency transaction gains and losses. The company had approximately 125.5 million ordinary shares outstanding as of September 30, 2021.

Cash, cash equivalents and investments as of September 30, 2021, were $254.1 million compared to $191.9 million as of December 31, 2020. The increase was primarily driven by net proceeds of $143.6 million from the February 2021 private placement and $17.5 million in upfront payments from the July 2021 collaboration with Pharming Group N.V., offset by cash used for operating activities and capital expenditures. The company expects that its cash, cash equivalents and investments as of September 30, 2021 will support its currently anticipated operating expenses and capital expenditure requirements into the first half of 2023. This cash runway excludes an additional $67 million that could become available under the companys credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers granted by the FDA following future U.S. approvals.

About Libmeldy / OTL-200 Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability. For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website. Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. About Orchard

At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S. headquarters inBoston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website ( http://www.orchard-tx.com ), the investor relations website ( ir.orchard-tx.com ), and on social media ( Twitter and LinkedIn ), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, the therapeutic potential of Libmeldy (OTL-200) and Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding its ongoing preclinical and clinical trials, including the timing of enrollment for clinical trials and release of additional preclinical and clinical data, the likelihood that data from clinical trials will be positive and support further clinical development and regulatory approval of Orchard's product candidates, and Orchards financial condition and cash runway into the first half of 2023. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchards ability to commercialize its product candidates, if approved, or Libmeldy, including the risk that Orchard may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2021, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com

Media Benjamin Navon Director, Corporate Communications +1 857-248-9454 Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Reports Third Quarter 2021 Financial Results and Highlights Recent ... - KULR-TV

Myles Glass has spent the past several years living life on the sidelines in a wheelchair, wishing for a better day. That day came in November 2020.

INDIANAPOLIS A 13-year-old boy living with sickle cell disease has been given a second chance at life, thanks to his mother.

Myles Glass has been through more in his young life than most adults. For the past few years, Glass has spent his days in and out of Riley Hospital for Children.

"[I] kind of have to look on the bright side of things. Being in the hospital, I meet new nurses and kids who go through what I go through. It's kind of hard to go through that at my age," Glass said.

He was diagnosed with sickle cell disease as a newborn. According to the Centers for Disease Control and Prevention, African Americans make up the largest number of people with the disease in the U.S.

Sickle cell disease is an inherited condition that impacts red blood cells and causes pain, infections and extreme fatigue. These symptoms keep Glass from doing things he loves.

"For him, it's kind of like we have to have him in a bubble," said his mother, Melissa Sanders.

Glass has spent the past several years living life on the sidelines in a wheelchair, wishing for a better day.

"[I would] hope that one day, I can do what kids do, like playing football and basketball," Glass said.

That day came in November 2020 when his mother donated bone marrow for a stem cell transplant, curing him of sickle cell disease.

"I was able to give him a second life with being a donor so that he can somewhat be a normal kid," Sanders said.

Riley Hospital for Children Dr. Seethal Jacob, who has been working with Glass and his family, said one baby every two minutes is born with sickle cell disease. She also said studies show there is a clear disparity for funding for this disease.

"There's been a lot of neglect when it comes to the disease itself. I think it's important to pay attention to the population it affects. I think that likely tells the story why sickle cell disease has been a neglected disease for so long," Jacob said.

Despite his challenges, Glass is staying positive and making strides in his physical therapy at Riley Hospital for Children.

"He's already been through harder things than most people will ever go through. I think anything else in life is going to be a piece of cake," said his physical therapist, Sarah Johnson.

"This gives me a glimpse of hope that even though you may have been diagnosed with this disease, it's not the end of the world," Sanders said.

For Glass, this is just the beginning. He hopes his story encourages other people living with sickle cell disease to keep moving forward.

"I know it's hard now, but you'll get through it. You'll be able to do what kids do your own age," Glass said.

Click here for more information on sickle cell disease and treatment options.

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Indianapolis mother gives 13-year-old son with sickle cell disease a 2nd chance at life - WTHR

SYDNEY, Oct. 5, 2021 /PRNewswire/ -- Clinical stage drug development company Pharmaxis Ltd (ASX: PXS) today announced further positive results of data analysis from a phase 1c clinical trial (MF-101) studying its drug PXS-5505 in patients with the bone marrow cancer myelofibrosis for 28 days at three dosage levels.

Assessment with Pharmaxis' proprietary assays of the highest dose has shown inhibition of the target enzymes, LOX and LOXL2, at greater than 90% over a 24-hour period at day 7 and day 28. The trial safety committee has reviewed the results and having identified no safety signals, has cleared the study to progress to the phase 2 dose expansion phase where 24 patients will be treated at the highest dose twice a day for 6 months.

Pharmaxis CEO Gary Phillips said, "We are very pleased to have completed the dose escalation phase of this study with such clear and positive findings.We will now immediately progress to the phase 2 dose expansion study where we aim to show PXS-5505 is safe to be taken longer term with the disease modifying effects that we have seen in the pre-clinical models. The trial infrastructure and funding is in place and we are on track to complete the study by the end of 2022."

Independent, peer-reviewed research has demonstrated the upregulation of several lysyl oxidase family members in myelofibrosis.The level of inhibition of LOX achieved in the current study at all three doses significantly exceeds levels that caused disease modifying effects with PXS-5505 in pre-clinical models of myelofibrosis with improvements in blood cell count, diminished spleen size and reduced bone marrow fibrosis. LOXL2 was inhibited to a similar degree and based on pre-clinical work such high inhibition is likely replicated for other LOX family members (LOXL1, 3 and 4).[1] Study data can be viewed in the full announcement.

Commenting on the results of the trial, Dr Gabriela Hobbs, Assistant Professor, Medicine, Harvard Medical School & Clinical Director, Leukaemia, Massachusetts General Hospital said, "Despite improvements in the treatment of myelofibrosis, the only curative therapy remains an allogeneic stem cell transplantation, a therapy that many patients are not eligible for due to its morbidity and mortality. None of the drugs approved to date consistently or meaningfully alter the fibrosis that defines this disease. PXS-5505 has a novel mechanism of action by fully inhibiting all LOX enzymes. An attractive aspect of this drug is that so far in healthy controls and in this phase 1c study in myelofibrosis patients, the drug appears to be very well tolerated. This is meaningful as approved drugs and those that are undergoing study, are associated with abnormal low blood cell counts. Preliminary data thus far, demonstrate that PXS-5505 leads to a dramatic, >90% inhibition of LOX and LOXL2 at one week and 28 days. This confirms what's been shown in healthy controls as well as mouse models, that this drug can inhibit the LOX enzymes in patients. Inhibiting these enzymes is a novel approach to the treatment of myelofibrosis by preventing the deposition of fibrosis and ultimately reversing the fibrosis that characterizes this disease."

The phase 1c/2a trial MF-101 cleared by the FDA under the Investigational New Drug (IND) scheme aims to demonstrate that PXS-5505, the lead asset in Pharmaxis' drug discovery pipeline, is safe and effective as a monotherapy in myelofibrosis patients who are intolerant, unresponsive or ineligible for treatment with approved JAK inhibitor drugs. Trial sites will now open to recruit myelofibrosis patients into the 6-month phase 2 study in Australia, South Korea, Taiwan and the USA.

An effective pan-LOX inhibitor for myelofibrosis would open a market that is conservatively estimated at US$1 billion per annum.

While Pharmaxis' primary focus is the development of PXS-5505 for myelofibrosis, the drug also has potential in several other cancers including liver and pancreatic cancer where it aims to breakdown the fibrotic tissue in the tumour and enhance the effect of chemotherapy treatment.

Trial Design

Name of trial

PXS5505-MF-101: A phase 1/2a study to evaluate safety, pharmacokinetic and pharmacodynamic dose escalation and expansion study of PXS-5505 in patients with primary, post-polycythaemia vera or post-essential thrombocythemia myelofibrosis

Trial number

NCT04676529

Primary endpoint

To determine the safety of PXS-5505 in patients with myelofibrosis

Secondary endpoints

Blinding status

Open label

Placebo controlled

No

Trial design

Randomised, multicentre, 4 week duration phase 1 (dose escalation) followed by 6 month phase 2 (dose expansion)

Treatment route

Oral

Treatment frequency

Twice daily

Dose level

Dose escalation: three escalating doses

Dose expansion: one dose

Number of subjects

Dose escalation: minimum of three patients to maximum of 18 patients

Dose expansion: 24 patients

Subject selection criteria

Patients with primary or secondary myelofibrosis who are intolerant, unresponsive or ineligible for treatment with approved JAK inhibitor drugs

Trial locations

Dose escalation: Australia (2 sites) and South Korea (4 sites)

Dose expansion: Australia, Korea, Taiwan, USA

Commercial partners involved

No commercial partner

Reference: (1) doi.org/10.1002/ajh.23409

AUTHORISED FOR RELEASE TO ASX BY:

Pharmaxis Ltd Disclosure Committee. Contact: David McGarvey, Chief Financial Officer and Company Secretary: T +61 2 9454 7203, E [emailprotected]

Join the Pharmaxis mailing listhere

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About Pharmaxis

Pharmaxis Ltd is an Australian clinical stage drug development company developing drugs for inflammatory and fibrotic diseases, with a focus on myelofibrosis. The company has a highly productive drug discovery engine built on its expertise in the chemistry of amine oxidase inhibitors, with drug candidates in clinical trials. Pharmaxis has also developed two respiratory products which are approved and supplied in global markets, generating ongoing revenue.

Pharmaxis is developing its drug PXS-5505 for the bone marrow cancer myelofibrosis which causes a build up of scar tissue that leads to loss of production of red and white blood cells and platelets. The US Food and Drug Administration has granted Orphan Drug Designation to PXS-5055 for the treatment of myelofibrosis and permission under an Investigational Drug Application (IND) to progress a phase 1c/2 clinical trial that began recruitment in Q1 2021. PXS5505 is also being investigated as a potential treatment for other cancers such as liver and pancreatic cancer.

Other drug candidates being developed from Pharmaxis' amine oxidase chemistry platform are targeting fibrotic diseases such as kidney fibrosis, NASH, pulmonary fibrosis and cardiac fibrosis; fibrotic scarring from burns and other trauma; and inflammatory diseases such as Duchenne Muscular Dystrophy.

Pharmaxis has developed two products from its proprietary spray drying technology that are manufactured and exported from its Sydney facility; Bronchitol for cystic fibrosis, which is approved and marketed in the United States, Europe, Russia and Australia; and Aridol for the assessment of asthma, which is approved and marketed in the United States, Europe, Australia and Asia.

Pharmaxis is listed on the Australian Securities Exchange (PXS). Its head office, manufacturing and research facilities are in Sydney, Australia. http://www.pharmaxis.com.au

About PXS-5505

PXS-5505 is an orally taken drug that inhibits the lysyl oxidase family of enzymes, two members LOX and LOXL2 are strongly upregulated in human myelofibrosis. In pre-clinical models of myelofibrosis PXS-5505 reversed the bone marrow fibrosis that drives morbidity and mortality in myelofibrosis and reduced many of the abnormalities associated with this disease. It has already received IND approval and Orphan Drug Designation from the FDA.

About Myelofibrosis

Myelofibrosis is a disorder in which normal bone marrow tissue is gradually replaced with a fibrous scar-like material. Over time, this leads to progressive bone marrow failure. Under normal conditions, the bone marrow provides a fine network of fibres on which the stem cells can divide and grow. Specialised cells in the bone marrow known as fibroblasts make these fibres.

In myelofibrosis, chemicals released by high numbers of platelets and abnormal megakaryocytes (platelet forming cells) over-stimulate the fibroblasts. This results in the overgrowth of thick coarse fibres in the bone marrow, which gradually replace normal bone marrow tissue. Over time this destroys the normal bone marrow environment, preventing the production of adequate numbers of red cells, white cells and platelets. This results in anaemia, low platelet counts and the production of blood cells in areas outside the bone marrow for example in the spleen and liver, which become enlarged as a result.

Myelofibrosis can occur at any age but is usually diagnosed later in life, between the ages of 60 and 70 years. The cause of myelofibrosis remains largely unknown. It can be classified as either JAK2 mutation positive (having the JAK2 mutation) or negative (not having the JAK2 mutation).

Source: Australian Leukemia Foundation: https://www.leukaemia.org.au/disease-information/myeloproliferative-disorders/types-of-mpn/primary-myelofibrosis/

Forward-looking statements

Forwardlooking statements in this media release include statements regarding our expectations, beliefs, hopes, goals, intentions, initiatives or strategies, including statements regarding the potential of products and drug candidates. All forward-looking statements included in this media release are based upon information available to us as of the date hereof. Actual results, performance or achievements could be significantly different from those expressed in, or implied by, these forward-looking statements. These forward-looking statements are not guarantees or predictions of future results, levels of performance, and involve known and unknown risks, uncertainties and other factors, many of which are beyond our control, and which may cause actual results to differ materially from those expressed in the statements contained in this document. For example, despite our efforts there is no certainty that we will be successful in developing or partnering any of the products in our pipeline on commercially acceptable terms, in a timely fashion or at all. Except as required by law we undertake no obligation to update these forward-looking statements as a result of new information, future events or otherwise.

CONTACT:

Media: Felicity Moffatt: T +61 418 677 701, E [emailprotected]

Investor relations:Rudi Michelson (Monsoon Communications) T +61 411 402 737, E [emailprotected]

SOURCE Pharmaxis Limited

http://www.pharmaxis.com.au

Read more:
Pharmaxis Cleared To Progress To Phase 2 Bone Marrow Cancer Trial - PRNewswire

TUCSON, Ariz., Oct. 5, 2021 /PRNewswire/ --On November 15th, 2021, healthcare professionals and the general public are invited to participate in World Cord Blood Day 2021 (www.WorldCordBloodDay.org) via a free online conference and live educational events being held around the globe. Registration is now open (free, public welcome).

Cord blood is the blood left in the umbilical cord and placenta following the birth of a child. It is rich in life-saving stem cells. While cord blood has been used for over 30 years, Covid-19 has renewed interest in this medical resource given its unique regenerative qualities and the fact that most cord blood currently stored was collected prior to the pandemic. These units are naturally Covid-free, an advantage over many other stem cell sources. Yet, cord blood is still thrown away as medical waste in the majority of births worldwide. Education is key to changing this practice and World Cord Blood Day 2021 will provide the perfect opportunity for OBGYNs, midwives, transplant doctors, nurses, parents and students to learn about this vital medical resource.

During World Cord Blood Day 2021, participants will learn how cord blood is used to treat over 80 life-threatening diseases such as leukemia and lymphoma, bone marrow failure, immune deficiency diseases and inherited blood disorders such as thalassemia and sickle cell disease. Leading transplant doctors and researchers will also highlight cord blood's role in the emerging fields of gene therapy and regenerative medicine to potentially treat cerebral palsy, autism, stroke and more.

Organized by Save the Cord Foundation, a 501c3 non-profit, World Cord Blood Day 2021 is officially sponsored by QuickSTAT Global Life Science Logistics, recognized leader in medical shipping and healthcare logistics. Inspiring Partners include Be the Match (NMDP), World Marrow Donor Association (WMDA-Netcord), AABB Center for Cellular Therapies, Cord Blood Association, and the Foundation for the Accreditation of Cellular Therapy (FACT).

"QuickSTAT, part of Kuehne+Nagel, is proud to sponsor the 5th annual World Cord Blood Day to help support and educate the healthcare community and expectant parents about the life-saving value of cord blood stem cells. We're excited to play a role in the research and development of cord blood derivative therapies by providing logistics supply chain solutions to cord blood, biotech and pharmaceutical companies worldwide," said Monroe Burgess, VP Life Science Commercial Marketing, QuickSTAT.

Visit http://www.WorldCordBloodDay.org to learn how you can participate. Show your support on social media: @CordBloodDay, #WorldCordBloodDay, #WCBD21

About Save the Cord FoundationSave the Cord Foundation (a 501c3 non-profit) was established to advance cord blood education providing non-commercial information to health professionals and the public regarding methods for saving cord blood, as well as current applications and the latest research. http://www.SaveTheCordFoundation.org.

About QuickSTAT Global Life Science LogisticsEvery day, QuickSTAT, a part of Kuehne+Nagel, safely and reliably moves thousands of critical shipments around the world. For over forty years, QuickSTAT has been entrusted with transporting human organs and tissue for transplant or research, blood, blood products, cord blood, bone marrow, medical devices, and personalized medicine, 24/7/365. QuickSTAT's specially trained experts work with hospitals, laboratories, blood banks and medical processing centers, and utilize the safest routes to ensure integrity, temperature control and chain of custody throughout the transportation process. Learn more at http://www.quickstat.aero.

Contact:Charis Ober(520) 419-0269[emailprotected]

SOURCE Save the Cord Foundation

http://www.SaveTheCordFoundation.org

Link:
Ready to Treat Over 80 Life-Threatening Diseases, Discover the Potential of Cord Blood during World Cord Blood Day 2021 - PRNewswire