Ifeyinwa Osunkwo, MD, MPH: Pat, can you describe the steps people go through to do a bone marrow transplant to gene therapy? Set the stage to help people understand why busulfan and why were talking about mutations. Can you walk us through the whole gene therapy process?

Patrick McGann, MD, MS: With transplant or gene therapy, the term transplant could be autologous, meaning your own cell gene therapy transplant. Its not as if youre transplanting a solid organ or a kidney. Sometimes patients get confused about this. Its looks like a blood transfusion hanging when it eventually goes in. For a bone marrow transplant, the donorwhoever that iseither gets a bone marrow aspiration, where they get bone marrow cells taken from their bone marrow, or a medicine to rev up their blood cells and get blood taken just from their vein. The patient needs to get prepared because they need to get rid of all their sickle cells. They need to suppress their immune system, so they dont reject this foreign cell, which is someone elses.

We use strong chemotherapy. If you have leukemia, as a comparison, you need to kill every last leukemia cell, and you get blasted with really strong chemotherapy agents and strong immunosuppressive agents. This is usually a week or so before; the days are counted backward. The cells that go in your body are most vulnerable to infection to everything. Its a dangerous time. Thats when complications come in. If its a transplant, you get infused with that donors bone marrow cells and hope it takes. It takes some weeks time for your body to take those new cells, and youre often receiving antibiotics and getting transfused and sustaining it, because your bone marrow is still not working. Basically, your immune system is suppressed. Its a tough time.

Transplant conditioning, as this regimen is called, has gotten a little less toxicreduced conditioning is the term. But thats still serious conditioning. Even though its reduced from what it used to be, its a relative term. Gene therapy is a little different because youre giving back your own cell. The way gene therapy happens is its ex vivo, meaning they take it out of your body. There are different ways that this is being done. Many patients need to have a bone marrow aspiration or many procedures to take enough cells out of their bone marrow to take them to the lab to fix them. There have been new ways to do this with peripheral blood and a medication called plerixafor, which is much better than going to the operating room for these horrible procedures.

Those cells are then taken to the lab and edited, or whatever the mechanism of gene therapy is. You still need to ablate your bone marrow to get rid of all your sickle cells. Because if you have any or many sickle cells in there, when you give back your edited gene cells, those will just take over. You still need to suppress that bone marrow. Because its your own cells, the immune suppression isnt as much of a problem as it is with transplant. Still, its a week of pretty serious medicinebusulfan, traditionallyand youre in the hospital for less of a period of time than transplant. Its quite an ordeal. Similarly, it takes or doesnt, and you monitor over time if that gene therapy has worked and whether its sustainablecross your fingersin the long term.

Ifeyinwa Osunkwo, MD, MPH: Basically, you have 2 options. The first option, you have to kill off their own bone marrow cells using chemotherapy. Then you give them somebody elses bone marrow, like a blood transfusion. The stem cells from the other person finds its way into their bone marrow and then grows. Then you wait and see what happens. Do you fight it? Do you accept it? We know if it takes or not. For gene therapy, we take out the patient stem cells, take it to a laboratory. Its usually in New Jerseydont ask me why. They manipulate it to pick out the gene they dont want. Then they give that patient back their own modified stem cells and wait for it to grow. But you still have to wipe out that persons bone marrow, so you dont have this fight going on. Even though theyre your cells, theyre a little different with the new gene change that has been made. Its a complicated process, and its really the only way to cure your disease. Either stem cell or gene therapy. We have had some setbacks in the past and more recently, but I believe that science is going to prevail. Over time were going to get to the point where we figure out the way to do this in the safest way to make it available to the most people with sickle cell disease and other blood disorders.

Thank you so much for watching this HCPLive Peer Exchange. If you enjoyed the content, please subscribe to the e-newsletter to receive upcoming Peer Exchanges and other great content right in your in-box.

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Stem Cell vs Gene Therapy Processes in SCD - MD Magazine

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Fighter: A year long battle but Rory is still smiling

In January 10-year-old Aurora Pile-Grays family were told they may lose their little girl as a rare and aggressive cancer took its devastating toll.

The previous November Aurora affectionately known as Rory had been declared in remission from the disease that she had been fighting since May 2020 but a cruel blow saw a severe relapse with the cancer progressing to her skull, eyes, neck, spine, liver, kidneys, lungs, abdomen and pelvis.

A discussion with the consultant over going home from hospital and preparing for end of life care took place but lion-hearted Rory was not ready to give up her fight against Burkitts Lymphoma which affects blood and bone marrow and her family were not ready to let go.

After a year-long battle with the disease, seven rounds of intensive chemotherapy, stays in Royal Marsden and Great Ormond Street hospitals and targeted therapy with trial drug Inotuzumab, Aurora was declared cancer free again at the end of April.

The Inotuzumab drug also offered the family, who live in Garlinge, a chance for Aurora to be at home despite treatment.

Mum Keisha, 28, said: We chose Inotuzumab because it meant a better quality of life for Rory, she could come home to us and her brother and sister rather than having to be in hospital.

The trial drug, and cancer all clear, opened the way for the youngster to undergo a bone marrow transplant on May 13 all the more vital as chemo had wiped out her immune system.

First there were 10 radiotherapy sessions to get through involving cranial boost, where Auroras face was bolted to the bed so she couldnt move.

The transplant which is universally referred to as a new birthday to signal a second chance at life took place at the Marsden and involved replacing old bone marrow cells that are failing to produce healthy new cells, with cells from a donor. The aim is to create a new immune system and hopefully prevent cancer returning or mutations occurring.

The stem cells Aurora now carries should begin to reproduce in her own body and allow her bone marrow to work as normal in producing healthy red cells, white cells and platelets, since her body is no longer capable of doing so after the effects of both the cancer and the treatment.

Auroras donor cells were frozen in December which meant on the day they had to be defrosted and infused within 20 minutes. Auroras donor produced nine bags of stem cells. Only four were required, leaving 5 for future use if needed.

Each bag is defrosted in a water bath around 38 degrees so that by the time its infused its not too different to body temperature. They are then put into separate syringes and pushed through a central line into the body.

The family have been told there is just a 10-20% chance that the transplant will achieve long lasting remission, but the St Crispins school youngster has so far beaten the odds and mum Keisha says her little girl is a fighter.

In her blog Growing Pains and Paper Planes, Keisha says: Shes been amazing throughout this entire journey and Im unbelievably proud to call her my daughter. Im in awe of her strength, determination and resilience and shes shown us all that sometimes the smallest hearts overcome the biggest battles.

So far Aurora has responded well to the transplant and there is evidence of engraftment where the donor cells find their way to her bone marrow and begin to make new blood cells. White cells are the first to engraft which include neutrophils, then red cells and then platelets.

Keisha said: We were worried the effects would be awful as Rory was so sensitive to the chemo but it is going really well.

Rory is up every day, listening to audio books and doing lots of colouring, cracking jokes and just being Rory.

It is such a relief. In January when we realised the cancer had spread and she could lose the use of her legs, bladder and bowel we had to talk about making her comfortable at home. But we were not ready to give up. I said it wasnt that time yet, not all the time that Rory was laughing and joking and looking forward to seeing her brother and sister. She wasnt giving up and we werent.

Now there has been this complete 360 turnaround. Everything still depends on how she takes to the transplant. On day 28 a sample will hopefully show what percentage are donor (cells) and what percentage are her own. Hopefully it will show primarily donor.

Rory is on immunosuppressants for up to a year but potentially could be able to return to school in six months time.

Keisha said: Shell go back as a Year 6 and has missed two years but has been doing schoolwork for an hour each day.

She is looking forward to it, more the social side than the work!

Rory has won the hearts of the Thanet community during her battle with cancer with many following the familys progress through Keishas blog and also donating to a fundraiser for life-saving treatment. People also signed up on the bone marrow register following Keishas highlighting of the desperate need for donors, especially people of mixed ethnicity.

Keisha said: We are unbelievably grateful and humbled by the support people have shown us over the past year. The kind words, the gestures, the gifts, the donations, the shares, the marrow registrations and the sense of community has helped us more than you will ever know.

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'Lion-hearted' ten-year-old Aurora's 'second chance' at life after cancer remission and stem cell transplant - The Isle of Thanet News

Bone Marrow Stem Cells Comments Off on ‘Lion-hearted’ ten-year-old Aurora’s ‘second chance’ at life after cancer remission and stem cell transplant – The Isle of Thanet News | May 28th, 2021

Magenta Therapeutics is losing its chief medical officer and head of R&D John Davis, M.D., with his last day coming July 30.

Davis helped steer the biotechs early path as well as deals with Avrobio and base editing biotech Beam Therapeutics around its lead stem cell conditioning program, MGTA-117, and MGTA-145 as a potentially new first-line standard of care for stem cell mobilization in a broad range of diseases. His departure comes three years after he joined the company from Pfizer, where he led its early R&D.

The biotech was keen to stress in an 8-K SEC filing (but not a press release) that his departure was not related to any disagreements with the Company on any matter relating to its operations, policies, practices or any issues regarding financial disclosures, accounting or legal matters.

It will now look for a new CMO, while Davis will become an adviser to the company.

RELATED: Magenta CSO Cooke jumps to IFM Therapeutics

This isnt the first move within the R&D ranks: Magenta started 2020 by losing its chief scientific officer when Michael Cooke, Ph.D., hopped over to IFM Therapeutics. In the fall, though, it nabbed Lisa Olson, Ph.D., who previously led immunology discovery at AbbVie, as his replacement.

Conditioning is a necessary step for some gene therapies, but one that can cause side effects like nausea, hair loss and mouth sores or make patients more vulnerable to infection. Magentas platform is based on looking to improve on current methods with an antibody-drug conjugate.

MGTA0117 is made up of an anti-CD117 antibody linked to amanitin, a cell-killing toxin. It is designed to target only hematopoietic, or blood-forming, stem cells and progenitor cells.

Animal studies suggest it could clear space in bone marrow for gene-modified stem cells to take root, Magenta said. The company plans to wrap IND-enabling studies for the antibody-drug conjugate this summer.

MGTA-145, meanwhile, just finished off a midstage test earlier this month, hitting its primary endpoint in a small multiple myeloma study.

Davis' replacement will already have a lot of clinical work on their hands.

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Magenta Therapeutics' research lead Davis hits the exit for family reasons - FierceBiotech

Bone Marrow Stem Cells Comments Off on Magenta Therapeutics’ research lead Davis hits the exit for family reasons – FierceBiotech | May 28th, 2021

Chronic lymphocytic leukemia (CLL) is a type of blood cancer. It affects lymphocytes, a type of immature white blood cell that the body produces in the bone marrow.

People with CLL may experience discomfort, swelling, and pain in the abdomen if their spleen enlarges. In rare cases, CLL can also affect mucosal tissues, such as those lining the gastrointestinal (GI) tract.

This article discusses the link between CLL and stomach pain in more detail. It also looks at ways to prevent and treat CLL, the other possible symptoms, and the outlook for people with this condition.

People with CLL may experience various symptoms involving the abdomen or stomach, such as:

In most cases, people with CLL do not experience abdominal symptoms due to the disease until it progresses and becomes more severe.

Research suggests that CLL infiltrates and affects the GI tract in about 5.713% of cases. When CLL affects the GI tract, doctors may call it Richters syndrome.

People with CLL may experience abdominal swelling, discomfort, and tenderness as a result of their spleen becoming larger. Less commonly, they may also feel full after eating small amounts of food, as the spleen can press on the stomach, making it smaller and able to hold less.

In rare cases, CLL infiltrates the lining of the GI tract, causing inflammation and ulcers or open wounds. People may experience symptoms similar to those of inflammatory bowel disease (IBD) and malabsorption disorders. These symptoms may include diarrhea, nausea, vomiting, abdominal pain and cramping, and unintentional weight loss.

CLL is cancer that develops in lymphocytes, which are white blood cells that form in the bone marrow and help fight infection.

Lymphocytes make up most of the lymph tissues in the lymph nodes, thymus gland, adenoids, tonsils, and spleen. They are also present in the GI tract, bone marrow, and respiratory system.

CLL is a type of leukemia that develops gradually over time.

About 5075% of people with CLL do not experience noticeable symptoms. Due to this, doctors diagnose most people with CLL during routine blood work.

The symptoms of CLL often begin when the cancerous cells crowd out healthy cells in the bone marrow or migrate to other organs or tissues. When symptoms first appear, they are typically mild, but they then become increasingly severe. CLL can cause many nonspecific symptoms, so a person may feel as though they have a cold or the flu.

Possible symptoms of CLL that do not relate to the abdominal area include:

Doctors do not yet have a way to prevent leukemia. However, some types of leukemia, including CLL, may have links to toxins, such as herbicides, pesticides, radon, and tobacco exposure. People can help reduce the risk of CLL by avoiding or practicing extreme caution around these toxins.

Many people do not experience symptoms of CLL for years and do not require treatment. However, as the disease progresses, these individuals may need treatment to extend their lives.

When and how a doctor treats someones CLL depends on a few factors, including:

Chemotherapy is typically the first-line therapy for CLL.

Doctors may use chemotherapy in conjunction with other treatment options, such as monoclonal antibody therapy. This therapy binds antibodies to cancer cells and destroys them. Treatment can also include medications to treat or prevent infections or improve low blood cell levels.

For instance, some people may take a combination of the monoclonal antibody rituximab and the chemotherapy drugs fludarabine and cyclophosphamide. Alongside the oral chemotherapy medication chlorambucil, doctors use obinutuzumab or ofatumumab, which have the same drug target as rituximab.

Small molecule inhibitors, such as bendamustine hydrochloride, idelalisib, and ibrutinib, can also sometimes form part of a CLL treatment regimen.

In 2017, the Food and Drug Administration (FDA) approved the combination medication Rituxan Hyecela (rituximab and hyaluronidase human) for CLL treatment.

In recurring or aggressive cases of CLL, an individual may have a blood or bone marrow stem cell transplant. This procedure replaces diseased cells with healthy blood cells that are able to mature into bone marrow cells.

Doctors may treat CLL until the symptoms lessen and then stop treatment until the symptoms worsen again.

Many people with CLL live for many years with a high quality of life.

There is no cure for CLL, so treatment aims to extend and improve someones life by reducing their symptoms. Doctors treat many people intermittently as their symptoms reoccur.

A persons outlook depends on their age, overall health, underlying conditions, and stage of CLL. Typically, people who are over the age of 65 years or have a more advanced stage of CLL have a less positive outlook.

Genetic changes in CLL cells and increased beta-2 microglobulin protein levels in the blood can make CLL more challenging to treat, potentially affecting a persons outlook.

Doctors classify people with CLL into different risk groups depending on certain factors. Based on these risk groups, the estimated percentages of people surviving 5 years or more after their diagnosis is:

People with more advanced or severe CLL may experience abdominal swelling, discomfort, tenderness, and pain. They may also feel full after eating small amounts. More rarely, someone with CLL may develop GI tract inflammation or ulcers, which can cause symptoms such as diarrhea, nausea, vomiting, cramping, and unexplained weight loss.

Anyone who thinks that they may have CLL should speak with a doctor. People with a confirmed diagnosis who experience symptoms of more advanced or severe CLL, such as abdominal pain, should also seek medical care.

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Chronic lymphocytic leukemia and stomach pain: What is the link? - Medical News Today

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REDWOOD CITY, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced the initiation of a Phase 1/2 clinical trial to evaluate JSP191, the companys first-in-class anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning regimen prior to allogeneic transplant for chronic granulomatous disease (CGD). Jasper Therapeutics and the National Institute of Allergy and Infectious Diseases (NIAID) have entered into a clinical trial agreement in which NIAID will serve as the Investigational New Drug (IND) sponsor for this study.

CGD is a rare, inherited disease of the immune system that develops in infancy or early childhood and results in severe and sometimes life-threatening infections. Allogeneic hematopoietic stem cell transplant is a proven cure for CGD. However, its use is limited because current conditioning agents used to deplete stem cells in preparation for transplantation are genotoxic and associated with limited efficacy and serious adverse effects, including veno-occlusive disease, long-term infertility and secondary malignancies.

We look forward to collaborating with NIAID on this Phase 1/2 clinical trial, which should provide important information about the potential of JSP191 as a safer and more effective conditioning agent for patients with CGD undergoing hematopoietic stem cell transplant, said Kevin N. Heller, M.D., Executive Vice President, Research and Development, of Jasper Therapeutics. Through this clinical trial agreement with NIAID, as well as others with the National Institutes of Health and academic centers, we are continuing to develop JSP191 for additional pretransplant conditioning regimens beyond severe combined immunodeficiency and acute myeloid leukemia/myelodysplastic syndromes, which have demonstrated safety and efficacy in early-stage clinical trials to date.

About JSP191

JSP191 is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow, creating an empty space for donor or gene-corrected transplanted stem cells to engraft. While hematopoietic cell transplantation can be curative for patients, its use is limited because standard high dose myeloablative conditioning is associated with severe toxicities and standard low dose conditioning has limited efficacy. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients. It is currently enrolling in two clinical trials for acute myeloid leukemia (AML)/myelodysplastic syndromes (MDS) and severe combined immunodeficiency (SCID) and is scheduled to begin enrollment in three additional studies in 2021 for severe autoimmune disease, sickle cell disease and Fanconi anemia patients undergoing hematopoietic cell transplantation.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The company is advancing two potentially groundbreaking programs. JSP191, a first-in-class anti-CD117 monoclonal antibody, is in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplantation. It is designed to enable safer and more effective curative allogeneic and autologous hematopoietic cell transplants and gene therapies. In parallel, Jasper Therapeutics is advancing its preclinical engineered hematopoietic stem cell (eHSC) platform, which is designed to overcome key limitations of allogeneic and autologous gene-edited stem cell grafts. Both innovative programs have the potential to transform the field and expand hematopoietic stem cell therapy cures to a greater number of patients with life-threatening cancers, genetic diseases and autoimmune diseases than is possible today. For more information, please visit us at jaspertherapeutics.com.

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Jasper Therapeutics Announces New Clinical Trial with the National Institute of Allergy and Infectious Diseases to Evaluate JSP191 in Chronic...

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KILLEEN, TX As blankets of snow covered the frigid ground and ice sheets laid atop of pavement, Michael or MJ Dixon was due for a sports physical at his normal pediatrician.

The doctor gave him a clearance, Chaundra Dixon, his mother explained. [The doctor] gave him a clearance and said, oh, he's healthy. He's fine.

MJ was used to getting hurt.

As a basketball, football, baseball and tennis star, its only a matter of time before one accumulates bruises and sore muscles.

However, MJ kept saying he was hurt.

His mom was convinced it was just growing pains, and so the duo worked on his stretching.

Until February 24, 2021.

She was at work on Fort Hood when MJs babysitter gave her a call.

She was just like, 'hey, I think you need to come home and get Michael, we need to get him to the hospital,' she recalled, as tears built up in her eyes thinking back to that day. He's crying non stop; he's saying he can't walk.

The panicked mother immediately picked up her son from their Killeen home and braved the icy road conditions, making it to Baylor Scott and White before EMS got to her house.

As they arrived at the hospital, they were met with COVID-19 tests. From there, physicians tested MJ for the basics, which all looked OK.

It wasnt until they ran blood work and found an abnormal amount of white blood cells in MJs body when they determined it was leukemia

About seven o'clock that night, I will never forget that, Dixon said. He was like, you're a great mom and I'm sorry, that I have to tell you, your son has leukemia, and I said leukemia? I lost it.

Living in this new reality brought on by his cancer, MJ and his family began looking for bone marrow donors.

The problem? He has less than a 25% chance of finding one due to his ethnicity.

http://www.bethematch.org

This is simply because we don't have the donor pool to pull from, Tressa Malone, a spokesperson with the Be The Match organization said. What that means is, we just need more people to join the registry. It's as simple as that.

Once you register to see if you can become a match, Malone explained that the following procedures are quite simple.

She said that if you match with a patient in need, 80% of their matches donate in a process similar to donating plasma.

You go and they take out the blood, separate the cells they need, then put it back in you.

She explained that this process is usually 3-4 hours.

The second procedure to donate is by retrieving the blood near your pelvic bone, the doctors separate the stem cells, all while youre under anesthesia.

Dr. Amy Mersiovsky, the director of nursing at Texas A&M Central Texas explained that she spent countless days caring for young kids with cancer as a pediatric nurse.

Realizing the low odds African Americans face when it comes to these donors, she explained that shes not necessarily surprised after the generations of mistrust that community typically has toward the medical field.

However, the Dixons dont go down without a fight because they know God is on their side.

Now, the family organizes bone marrow drives not only here in Central Texas, but across the county, hoping to find MJ a donor or anyone whos walking the same journey the 9-year-old boy is.

The family has set up a GoFundMe page to cover various expenses, and to see if you can be a match, you can text MJ to 61474 and a swab kit will be sent to your home.

To follow along with MJ's story, head over to his Facebook page.

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Killeen boy with leukemia needs a bone marrow donor, but the odds of finding one are slim - KXXV News Channel 25

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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Moderna, Inc., (Nasdaq: MRNA) a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced new research being highlighted as part of the Companys fourth annual Science Day. Modernas Science Day is designed to provide insight into the continued diverse efforts underway at Moderna to better understand how to use mRNA as medicines and vaccines and underscores the Companys continued commitment to basic science and innovation.

Science Day gives us an opportunity to provide insights into the advancements in our platform science and our further understanding of how to use mRNA as both a vaccine and a medicine. Our investments in basic science continue to result in major steps forward in our platforms capabilities, and these have allowed us to open new therapeutic areas and new scientific directions, said Stephen Hoge, M.D., President of Moderna. Our investments in platform research along with our digital backbone and manufacturing plants have enabled us to create first-in-class mRNA medicines and vaccines. Today, we're excited to highlight our work to identify and address SARS-CoV-2 variants of concern, optimize our proprietary lipid nanoparticle (LNP) technology, and deliver mRNA to hematopoietic stem cells.

Moderna currently has 24 mRNA development programs in its portfolio with 14 having entered clinical studies. The Companys updated pipeline can be found at http://www.modernatx.com/pipeline. Moderna and collaborators have published more than 65 peer-reviewed papers.

At this years Science Day, Moderna will present new platform science and preclinical research, including:

mRNA Delivery to Hematopoietic Stem and Progenitor Cells (HSPC)

Of the nearly 30 trillion cells in the body, approximately 90% are of hematopoietic origin. Hematopoietic lineages originating in the bone marrow are intimately involved in maintaining homeostasis and human health. Nonetheless, there are hundreds of hematologic or immune-related disorders caused or exacerbated by cells of the hematopoietic lineage. In a disease setting, cells of hematopoietic origin interact with host tissues to drive chronic inflammatory and immune disorders. Because some hematopoietic stem and progenitor cells (HSPC) have self-renewal and pluripotent properties, targeting HSPC has the potential to modulate underlying chronic inflammation and immune-related disorders.

Advances in lipid nanoparticle (LNP) technology has allowed for delivery to the bone marrow following systemic LNP administration in vivo. This achievement has enabled Moderna scientists to deliver mRNA directly to bone marrow HSPC in vivo, leading to HSPC transfection and long-term modulation of all hematopoietic lineages. This represents a major milestone in impacting chronic inflammatory and immune related disease.

mRNA Engineering: Optimizing Ribosome Load

The ribosome plays a central role in orchestrating the life of mRNA inside the cell. Understanding how to modulate translation by changing mRNA sequence could enable a powerful lever to control the performance of an mRNA drug. To develop such a lever, the Moderna's scientists strove to characterize mechanistically how differences in translation drive differences in protein expression. In this research, an unexpected relationship emerged where mRNAs with low translation initiation rates conferred the highest and most durable levels of protein expression. By understanding the mechanisms that drive this behavior, the Company is taking mRNA design from a guess-and-check discipline into an engineering discipline. With tools in place, and more under development, Moderna is improving its ability to make an mRNA that generates the right amount of the right protein, for the right amount of time, in the right cell type. As Moderna builds these levers into its mRNA drugs, the Company believes it will be able to target more indications with more precision.

Addressing SARS-CoV-2 Variants of Concern

One part of the Companys strategy to remain ahead of the SARS-CoV-2 virus is to closely monitor and address emerging variants of concern and waning immunity. Moderna is using artificial intelligence (AI) and machine learning to predict escape risk. This involves developing statistical models to understand and predict escape risk, including identifying breakthrough sequences from clinical trials and real-world data, examining spike protein biophysical and pseudovirus neutralization data, studying spike mutations and lineage info, and time tracking.

Science Day Webcast Information

Moderna will host its annual Science Day for analysts and investors at 8:00 a.m. ET on Thursday, May 27. A live webcast will be available under Events and Presentations in the Investors section of the Moderna website at investors.modernatx.com. A replay of the webcast will be archived on Modernas website for one year following the presentation.

About Moderna

In 10 years since its inception, Moderna has transformed from a science research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across six modalities, a broad intellectual property portfolio in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that allows for both clinical and commercial production at scale and at unprecedented speed. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing. Most recently, Modernas capabilities have come together to allow the authorized use of one of the earliest and most-effective vaccines against the COVID-19 pandemic.

Modernas mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Today, 24 development programs are underway across these therapeutic areas, with 14 programs having entered the clinic. Moderna has been named a top biopharmaceutical employer by Science for the past six years. To learn more, visit http://www.modernatx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: the potential for delivery of mRNA to hematopoietic stem and progenitor cells (HSPC) in vivo; methods of detecting and interrogating HSPC in vivo; the potential for delivering LNPs to hematopoietic stem cells in vivo; the ability to optimize codons and mRNA structures to increase total protein outputs; the potential for the Company to develop processes for controlling protein expression by modifying ribosomal loads; the Companys ability to engineer LNPs capable of accessing difficult-to-transfect primary cells with efficient endosomal escape and high functional mRNA delivery; the Companys strategy for combatting COVID-19, including processes for monitoring emerging variants and waning immunity; and strategies for modeling viral escape. In some cases, forward-looking statements can be identified by terminology such as will, may, should, could, expects, intends, plans, aims, anticipates, believes, estimates, predicts, potential, continue, or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Modernas control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include, among others: the fact that there has never been a commercial product utilizing mRNA technology approved for use; the fact that the rapid response technology in use by Moderna is still being developed and implemented; the safety, tolerability and efficacy profile of the Moderna COVID-19 Vaccine observed to date may change adversely in ongoing analyses of trial data or subsequent to commercialization; the Moderna COVID-19 Vaccine may prove less effective against variants of the SARS-CoV-2 virus, or the Company may be unsuccessful in developing future versions of its vaccine against these variants; despite having ongoing interactions with the FDA or other regulatory agencies, the FDA or such other regulatory agencies may not agree with the Companys regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted; Moderna may encounter delays in meeting manufacturing or supply timelines or disruptions in its distribution plans for the Moderna COVID-19 Vaccine; whether and when any biologics license applications and/or additional emergency use authorization applications may be filed in various jurisdictions and ultimately approved by regulatory authorities; potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and clinical trials, supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; and those other risks and uncertainties described under the heading Risk Factors in Modernas most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SECs website at http://www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Modernas current expectations and speak only as of the date hereof.

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Moderna Highlights Advances in Platform Science and Innovative Vaccine Research at Fourth Annual Science Day - Business Wire

Bone Marrow Stem Cells Comments Off on Moderna Highlights Advances in Platform Science and Innovative Vaccine Research at Fourth Annual Science Day – Business Wire | May 28th, 2021

For many years now, the topic of senescent cells has been the subject of plenty of research work. Back in the 1960s the Hayflick limit was noticed in cell culture: there was an apparent limit to the number of cell divisions that could take place before the cells just sort of stalled out. For human fibroblasts, that kicks in at around fifty divisions. Over time it was worked out that a primary mechanism involved is the shortening of telomeres with each cell division, specialized nucleotide sequences out at the ends of the chromosomes, and this cellular clock phenomenon has been making its way into the public consciousness ever since.

Its strange to think, but before these experiments human cells were considered to be more or less immortal and capable of unlimited numbers of divisions. Now, there are cells like that, but that (outside of some stem cell populations and a few other special cases) is a very short working definition of cancer. Those cells do indeed seem to be able to carry on for as long as conditions permit which in the artificial world of cell culture labs, means apparently forever. Henrietta Lacks died in 1951, but HeLa cells are still with us, and can be all too vigorous when they contaminate other lines. Tumor cells can pile up mutations that will make them die off, but short of that the jams have indeed been kicked out.

Its gradually become apparent that many aging or damaged tissues have a (sometimes substantial) population of cells that have reached their limit. Theyre alive and metabolically active but not really contributing much, in a stage of permanent growth arrest. Cellular senescence is a complex phenomenon, but its importance in aging, cancer, and tissue damaged by other factors (radiation, oxygen stress, etc.) is by now undeniable. Many of these non-aging states can be traced back to early telomere damage by other mechanisms, emphasizing that as a key countdown mechanism. But its clear that senescent have a different secretory profile (cytokines, growth factors and more) from the more vigorous cells around them and a number of other protein expression differences that can be used the characterize them.

Naturally enough, thoughts have turned to targeting such cells for therapy. There are a couple of very easy-to-picture hypotheses: first, could you keep telomeres from shortening (or shortening so much) and therefore keep cells in a non-senescent state for longer, potentially delaying biological aging? And second, could you somehow target cells that have already become senescent, and would doing so improve the health of the surrounding tissue? Though pretty obvious ideas, both of these are still very much in play. For now, Im going to talk about the second one, in light of a new paper.

That ones on the kidney. Younger people can regain some kidney function after an injury, but that ability goes down with aging, as youd imagine. It also goes down in states of chronic kidney disease, or after radiation damage. This new paper shows that targeting and removing senescent cells actually starts to reverse this phenotype once youve done that, the kidney tissue after injury shows increased function, increased regenerative ability, and less development of fibrosis. This is demonstrated both in aged tissue and in younger tissue exposed to radiation damage, in human cell culture and in mouse animal models.

You may well ask: how exactly does one target senescent cells? That takes us to ABT-263 (navitoclax), shown at right. This rather hefty molecule is part of a series of AbbVie protein-protein inhibitors for the Bcl-2 (B-cell-lymphoma) family. There are several of those, and navitoclax inhibits the function of Bcl-2, Bcl-xL, and Bcl-w. All of these proteins are intimately tied up in the pathways of apoptosis, programmed cell death, which is another monstrously huge pathway all its own. But one of the questions about senescent cells is why they dont go down some apoptotic pathway and just fall on their on cellular swords, instead of hanging around forever gumming up the works.

This one, like the others in its class, was developed to cause this to happen to tumor cells as an adjunct to other types of chemotherapy, but these have also turned out to be useful against senescent cells (although not all types of them). Similar to the kidney results reported in the new paper linked above, there have been reports in lung, CNS, muscle and other tissues of broadly similar enhancements (many of these summarized in this paper). So at this point you might be wondering why we dont just go ahead and put these things into the water supply already.

Theres a problem, unfortunately. It was clear from the clinical studies of the AbbVie compounds that platelet effects were dose-limiting. Cells in that pathway are sensitive to messing with these apoptosis pathways, and while you might be able to deal with that side effect in a chemotherapy situation, it doesnt exactly make for a good-for-what-ails-you drug. Navitoclax has alsorecently been shown to have profoundly bad effects on bone density and deposition, which is the exact opposite of what youd want for an aging population.

AbbVies next generation of such compounds, though, includes venetoclax, at right, also a lunker of a molecule and now approved for several types of leukemia. It still has platelet effects, but they arent nearly as disastrous as with navitoclax, thanks to deliberately lower binding to Bcl-xL. That also makes it a bit less of a mighty sword across senescent cell types for example, it appears that you need that pathway for activity against glioblastoma cells. But it has been reported to show strong protective effects against the development of Type I diabetes through the elimination of senescent cells in the islets of Langerhans. Meanwhile, other groups are looking at turning these ligands into targeted protein degraders, which (at least in some cases) seems to decrease the platelet problems and increase senolytic activity.

And before leaving the topic, it has to be noted that there are plenty of other ways to target these cells other than the Bcl pathway (although that one seems to be one of the most developed so far). What can I say? Im 59, and I doubtless have more senescent cells than I want or need, so I (and plenty of others) are interested in the idea. The whole cellular senescence pathway presumably developed as a way to avoid slipping into a tumor phenotype the more cellular divisions, the greater the chance of something going wrong along the way. Its a tradeoff, and evolution seems more than willing to shortchange older members of the species who have generally passed on their genes to all the offspring that theyre going to. But humans have other goals. We are looking at a rather rapidly aging planet, if current demographic trends hold up, and it would be extremely desirable to have that associated with less of a disease burden. Can we split the difference?

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Clearing Cellular Dead Wood | In the Pipeline - Science Magazine

Bone Marrow Stem Cells Comments Off on Clearing Cellular Dead Wood | In the Pipeline – Science Magazine | May 28th, 2021

REGULATED INFORMATION

Strong clinical progress especially in JTA-004 Phase III study thanks to high patient compliance and retention

Process development partnership and appointment of cell therapy expert Anthony Ting as CSO to further strengthen product pipeline

Gosselies, Belgium, 26May 2021, 7am CEST BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the cell therapy company addressing unmet medical needs in orthopedics and other diseases, today announces its business update for the first quarter, ended 31 March 2021.

Bone Therapeutics has continued the strong momentum into 2021, said Miguel Forte, MD, PhD, CEO of Bone Therapeutics. Bone Therapeutics mid-late stage clinical programs continue to advance largely on schedule, including the Phase IIb trial of the allogenic cell therapy platform ALLOB in difficult-to-heal tibial fractures and the Phase III trial of the enhanced viscosupplement JTA-004 in knee osteoarthritic pain. Alongside this, Bone Therapeutics has strengthened its manufacturing and R&D capabilities by signing a process development partnership with Rigenerand. It has also appointed the industry veteran Tony Ting as our new Chief Scientific Officer. Building on these achievements, Bone Therapeutics will be able to continue significant clinical and commercial advancements as we move towards the topline data of our JTA-004 Phase III study; a potential key inflection point for Bone Therapeutics.

Operational highlights

Financial highlights

Outlook for the remainder of 2021

(1) Unaudited number

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and other diseases. The Company has a, diversified portfolio of cell and biologic therapies at different stages ranging from pre-clinical programs in immunomodulation to mid-to-late stage clinical development for orthopedic conditions, targeting markets with large unmet medical needs and limited innovation.

Bone Therapeutics is developing an off-the-shelf next-generation improved viscosupplement, JTA-004, which is currently in Phase III development for the treatment of pain in knee osteoarthritis. Consisting of a unique combination of plasma proteins, hyaluronic acid - a natural component of knee synovial fluid, and a fast-acting analgesic, JTA-004 intends to provide added lubrication and protection to the cartilage of the arthritic joint and to alleviate osteoarthritic pain and inflammation. Positive Phase IIb efficacy results in patients with knee osteoarthritis showed a statistically significant improvement in pain relief compared to a leading viscosupplement.

Bone Therapeutics core technology is based on its cutting-edge allogeneic cell therapy platform with differentiated bone marrow sourced Mesenchymal Stromal Cells (MSCs) which can be stored at the point of use in the hospital. Currently in pre-clinical development, BT-20, the most recent product candidate from this technology, targets inflammatory conditions, while the leading investigational medicinal product, ALLOB, represents a unique, proprietary approach to bone regeneration, which turns undifferentiated stromal cells from healthy donors into bone-forming cells. These cells are produced via the Bone Therapeutics scalable manufacturing process. Following the CTA approval by regulatory authorities in Europe, the Company has initiated patient recruitment for the Phase IIb clinical trial with ALLOB in patients with difficult tibial fractures, using its optimized production process. ALLOB continues to be evaluated for other orthopedic indications including spinal fusion, osteotomy, maxillofacial and dental.

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

For further information, please contact:

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

For Belgian Media and Investor Enquiries:BepublicCatherine HaquenneTel: +32 (0)497 75 63 56catherine@bepublic.be

International Media Enquiries:Image Box CommunicationsNeil Hunter / Michelle BoxallTel: +44 (0)20 8943 4685neil.hunter@ibcomms.agency / michelle@ibcomms.agency

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

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

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Bone Therapeutics Provides First Quarter 2021 Business Update - GlobeNewswire

Bone Marrow Stem Cells Comments Off on Bone Therapeutics Provides First Quarter 2021 Business Update – GlobeNewswire | May 28th, 2021

A five-year-old boy has returned from New York where he is taking part in a pioneering medical trial aimed at preventing his cancer from returning.

Oliver Stephenson from West Yorkshire was given the all-clear earlier this year after battling neuroblastoma a rare cancer originating from immature nerve cells throughout 2020.

His family crowdfunded 270,000 for treatment as the schoolboy underwent chemotherapy, surgery, stem cell treatment and a bone marrow transplant.

At one point, Oliver and his dad James had to isolate together in a 15sqft hospital room for seven weeks due to the coronavirus pandemic.

Oliver has spent the past month at New York's Memorial Sloan Kettering Cancer Center, where he is on a clinical trial vaccine called Bivalent.

It is hoped the treatment will train his immune system to identify and destroy neuroblastoma cells lurking in his body to prevent the disease from returning.

The vaccine, which costs 210,000, will require Oliver to make four more trips to the US this year.

He returned from the four-week trip to New York around a week ago with his mum Laura, dad James and little brother Alfie.

Mrs Stephenson said: "Everything went great. The doctors were all amazing and Oliver has responded well so far.

"It is a clinical trial so we don't know what will happen but we are hoping that this will prevent Oliver from suffering as he has done again.

"After everything he has been through it feels great to be on a positive track."

She added the treatment was "quick and easy", explaining Oliver had three vaccines and that, other than some discomfort, he had no obvious side effects.

Due to the length of their stay, Mrs Stephenson said the family were able to make a holiday out of the trip.

She added: "Oliver and Alfie had a brilliant time in New York, it was amazing.

"Because of the pandemic everywhere was really quiet, which meant we felt very safe all the time and didn't have to queue for anything."

The pioneering treatment comes almost 18 months after Oliver was diagnosed with neuroblastoma, which affects around 100 children annually.

The coronavirus pandemic complicated the family's ordeal as Oliver and his dad had to isolate together in hospital during chemotherapy.

Mr Stephenson was at his son's bedside throughout, but the rest of his family were unable to visit due to restrictions and had to make do with video calls.

Crowdfunding pages were set up to help the family pay for treatment once options on the NHS were exhausted, as neuroblastoma has a significant chance of returning.

Mrs Stephenson added: "The incredible support and donations not just from our friends and family but around the world has been overwhelming.

"We are so grateful to everyone who has helped.

"It's been a really difficult year for us and back in 2020 Oliver's prognosis wasn't good at all.

"But he has battled through everything is doing well now, he's fit and strong and even back at school.

Despite Oliver getting the all-clear, figures show 60% of people who recovered from neuroblastoma end up relapsing.

Mrs Stephenson said: "We want to everything we can to stop that happening."

Throughout Oliver's treatment, his family has been supported by the Solving Kids Cancer charity.

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Five-year-old boy returns home after first round of cancer treatment in New York - Surrey Live

Bone Marrow Stem Cells Comments Off on Five-year-old boy returns home after first round of cancer treatment in New York – Surrey Live | May 28th, 2021

bluebird bios cerebral adrenoleukodystrophy (CALD) gene therapy Skysona has moved closer towards EU approval after gaining a positive opinion from the European Medicines Agencys Committee for Medicinal Products for Human Use (CHMP).

The CHMP has recommended marketing authorisation for Skysona (elivaldogene autotemcel, Lenti-D) for the early treatment of CALD in patients under 18 years old with an ABCD1 genetic mutations, and who do not have a matched sibling haematopoietic stem cell (HSC) donor.

bluebird bio's Skysona is a potential one-time gene therapy designed to add functional copies of the ABCD1 gene into a patients hematopoietic stem cells.

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

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

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

Currently, the only treatment for the disease is a stem cell transplant, although this carries a significant risk from the high-dose chemotherapy used to prepare patients for the procedure.

Other complications include graft-versus-host (GvHD) disease, which occurs when the transplanted cells recognise the recipients cells as foreign and attack them.

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

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

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

The CHMPs positive opinion is now due to be reviewed by the European Commission, with a final decision for Skysona expected in mid-2021.

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bluebird bio's CALD gene therapy Skysona gains positive opinion from CHMP - PMLiVE

Spinal Cord Stem Cells Comments Off on bluebird bio’s CALD gene therapy Skysona gains positive opinion from CHMP – PMLiVE | May 28th, 2021

NEARLY 10,000 has been raised in less than a week to help pay for a Dunfermline mum to have life-changing treatment in Russia.

Lynda Hogg was first diagnosed with Primary Progressive Multiple Sclerosis (PPMS) seven years ago and since then, she has seen her health deteriorate.

She has been forced to give up her job as a theatre nurse at Queen Margaret Hospital and faces a daily battle with pain from a condition for which there is no cure and no treatment on the NHS.

Lynda, 58, and her family are now pinning their hopes on travelling to Russia for Hematopoietic Stem Cell Transplantation which, it is hoped, will stop the progression of her MS.

Within a week of being set up, a GoFundMe page has already raised nearly a quarter of the required 45,000, something which Lynda said she was overwhelmed and humbled by.

It was my son who said how do you feel in 10 years time if you are wheelchair-bound knowing you had this opportunity and didnt take it? she explained.

We have considered selling the house if we have to.

Daughter Popsi is hopeful that the GoFundMe page will mean such a drastic step is not necessary.

Before the GoFundMe, these were all considerations, she said. Shall we sell the house, will my dad have to give up his job and become a carer?

After we found out about this treatment years ago, I dont think we ever really considered it a possibility. It has been quite tough. I have three brothers. Speaking on behalf of the younger ones, I want them to remember them as I remember her, full of life.

This treatment is kind of a possibility that it would not get any worse. It is a pipe dream. We never thought it could happen. The support and kindness of others has been so touching.

Multiple Sclerosis (MS) is a disabling disease of the brain and spinal cord which results in muscle and nerve damage, ongoing pain and fatigue.

Since her diagnosis, Lynda, who is married to Murray and has four children, Adam, 29, Popsi, 23, Mitchell, 18, and Charlie, 15, has seen her health go downhill with her mobility decreasing.

She has been shocked at the lack of available medication.

Basically, I get co-codamol, she said. Progressive Primary MS is the worst form of it. There is no treatment and there is really little in the way of emotional support. It is not a fault of the NHS. There is nothing and there needs to be something.

PPMS is 15 per cent of people diagnosed. It is horrible to think there is nothing out there for them.

Trials for the treatment which Lynda hopes to have in Russia have taken place in the UK but, to date, are not available on the NHS for patients like herself.

They give you medication to increase the quality of stem cells. They are harvested and then you are given chemo then the stem cells are reintroduced to you. They can stop MS in its tracks. It will stop the progression. It wont give you functions back.

Even just to stop it progressing; I can cope with the disabilities I have but I dont know if I can cope with them moving forward and getting worse.

Popsi and her siblings are planning to hold further fundraisers when coronavirus restrictions allow while also raising awareness of MS.

We have had to watch our mum struggle with the disease and battle with pain every day, she added. We feel that its time to give back to our mum, and everything that she has done for us over the years.

Anyone wanting to help can do so by visiting https://gofund.me/61401f6e.

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Dunfermline mum "overwhelmed" by fundraiser aiming to fund vital treatment in Russia - Dunfermline Press

Spinal Cord Stem Cells Comments Off on Dunfermline mum "overwhelmed" by fundraiser aiming to fund vital treatment in Russia – Dunfermline Press | May 28th, 2021

NEW ORLEANS (WGNO) Several contract workers were burned during a gas well explosion on Belle Isle along the Gulf of Mexico on Tuesday.

Of those wounded, three were reported taken to University Medical Center the regions only Level I verified trauma and verified burn center.

According to Dr. Jeffrey Carter, Medical Director of the UMC Burn Center and professor of surgery at LSU Health-New Orleans, speed is of the essence when it comes to successful treatment of burn victims.

At 2:56 pm, Acadian received a report of a well fire. We sent 4 helicopters and 5 ground units to Morgan City (LA). We transported two patients by air (1 to NOLA and 1 to Lafayette area) and 2 patients by ground (both to NOLA)

Delays in medical treatment can result in increased amounts of resuscitation, increased length of hospital stays, increased infection risks and increased kidney problems.

Its important to realize here in Louisiana, we have a fair number of risks. About 85 percent of all hazardous waste travels through our port or rail here in New Orleans, said Dr. Carter.

There are over 3,000 rigs, gas and pipeline areas here that are at risk along the gulf. When we have injuries that occur from industrial accidents, what we find is that the mortality is increased by about 20 percent if there is a delay of about two hours in transporting the patient to a center where they can get definitive care.

Dr. Carter says being an academic medical research center, UMC is able to offer the latest technology in the treatment of severe burns.

On the forefront of burn care is the use of RECELLs spray-on skin by Avita Medical, in which the doctors take a small portion of the patients own skin, dissolve it and pull the stem cells, and then -apply it at the time of surgery.

Dr. Carter explained and demonstrated the use of RECELL during a Zoom interview with WGNOs Aaron S. Lee on Wednesday.

He also discussed how the use of artificial intelligence will soon revolutionize how burn victims are treated and how quickly they recover.

That video clip can be seen below:

According to Dr. Carter, half of his patients suffer burns to their hands and face as these are areas not covered, especially in the deep south. This can be worrisome to the victim after recovery as those are places people notice first when interacting with another person.

Not to mention, the face is extremely complex with the amount of movement and how it changes over time.

The use of RECELL on a burn victims face is making skin grafts a thing of the past.

A skin graft is like putting a piece of plywood on your roof when it has a hole in it, explained Dr. Carter. It doesnt look the same. It doesnt act the same. It doesnt behave the same.

While its OK for some types of injuries, said Dr, Carter, its not necessarily the best thing.

Dr. Carter goes on to explain the benefits of using of the RECELL system and ultimately allowing burn patients to heal themselves using their own cells during recovery.

The FDA is considering RECELL for pediatric burns, with Dr. Carter serving as one of the clinical trial investigators.

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Burn victims like the wounded Belle Isle contractors in good hands at UMC, spray-on skin at forefront of care - ArkLaTexHomepage

Skin Stem Cells Comments Off on Burn victims like the wounded Belle Isle contractors in good hands at UMC, spray-on skin at forefront of care – ArkLaTexHomepage | May 28th, 2021

Aging is the ultimate risk factor for most diseases, such as cancer, neurodegenerative, cardiovascular, diabetes, degenerative fibrosis and many others. When we are young, we are typically healthy, despite a predisposition that will lead inevitably to a specific degenerative condition. However, the degenerative processes do not kick in until a certain age, when we are older. It looks like when we are younger, the body can compensate cumulative stress and damage caused to our cells in the tissues, allowing to maintain that equilibrium, called homeostasis, that keeps our organs functional and healthy. However, over time this buffering capacity becomes thinner and thinner, until things wear off: our tissues stop working as they used to. These changes are typically caused by an initial small number of rare but bad cells, that progressively increase over time, causing additional damage to the good cells that eventually stop working efficiently, causing a vicious cycle. Eventually the bad cells take over leading to the onset of a disease.

Our body is equipped with a number or regenerative and healing functions. Some are intrinsic in every cell, such as DNA repair mechanisms that are triggered when something compromises the integrity of our genomic structures. These are important functions that enable a cell, for example when it replicates, to repair errors and other damages that might have happened to our DNA. For example, two large proteins called ATM and ATR, involved in the cellular response to DNA damage, are responsible to maintain genomic instability caused by intrinsic and external DNA-damaging agents, such as UV light or various chemicals and toxins. A lack of functions of these proteins results in progressive neurodegeneration, immunodeficiency, predisposition to malignancy or radiation sensitivity. Mutations on the genes encoding these proteins can cause premature aging and premature development of these diseases, but this occurs also naturally, over time.

Cells also have an intrinsic immune system, producing factors called interferons employed by the cells as antiviral agents and to modulate other immune functions. It can be triggers by a viral infection so when a cell is infected will release interferons, protecting the neighbor cells against potential infection. Interferons can also suppress growth of blood vessels preventing tumors to get nutrients and growing. They can also activate immune cells so they can better fight viruses, tumors and others agents. Unfortunately, an age-related decline or impaired innate interferon functions in the cells results in a number of negative consequences in the body, such as increased susceptibility of the elderly to infections, tumors and damage.

In the body there are several cell types responsible to keep the tissues in check. The immune system is specialized to recognize remove and remember damaging agents. Those could be external, such as virus, bacteria or parasites, or internal, such as tumorigenic cells or senescent cells (see below). The immune system is a very sophisticated network of cell types, intercommunicating with each other to maintain the body clean from damaging factors. As we age the immune system also ages and loses capacity to recognize or responding to these damaging agents. It also become exhausted by an increasing chronic inflammation that progressively accumulate as we age, phenomenon also called inflammaging.

Another important repairing mechanism is the regenerative tissue functions, driven by the stem cells. Those cells are progenitor cells, often dormant in a quiescent state in the tissue and waiting to be activated by some damage. Stem cells are critical because once activated they can generate a progeny of daughter cells capable of re-growing the damaged tissue back to its original structure and function. Stem cells have another important function: they can regenerate themselves, in a process called self-renewal. This is important so that the new repaired tissue can repeat the process if a new damage occurs. The regenerative capacity of our body is remarkable, allowing our tissues to keep their integrity, health and functions. However, over time also stem cells age or respond to the aged microenvironment where they live (called the niche), and they become less efficient to repair tissues or to self-renewing. As a result, our tissues change, become atrophic, fibrotic or dysfunctional leading eventually to diseases.

In regenerative medicine, the application of stem cells resulted of the generation of multiple new therapeutic opportunities. A promising area uses stem cells to generate bioengineering strategies to grow new tissues in a petri dish to be then transplanted in the body to repair damaged tissues. Some applications are already in clinical use, such as for skin grafts. Many others are on their way, either in preclinical development or in clinical trials for many different tissue types and for different clinical indications.

Another promising stem cells application is the direct transplantation into damaged tissues, where they can grow and engraft repairing. However, as we age stem cells become less efficient. What if we If we could rejuvenate them? We could restore their capacity to repair our tissues and maintain homeostasis. Promising and exciting strategies are advancing in that direction. For example, we and others showed that it is possible to reprogram epigenetically a cell so it can become the younger and healthier version of itself (Sarkar et al., 2020). This is a mechanism that every cell has encoded in its DNA, but normally works only in the germline (the sperm and the egg) during the embryogenesis to make sure that the cellular clock is turned back to zero, before initiating the cellular programs to generate the embryo. This important for example to prevent making old newborn babies. This intrinsic rejuvenative mechanism is locked in the other somatic cells of the body. We found it is possible to re-activate it transiently and safely, without changing the identity of the cell, enabling to push back the cellular clock of aged human cells to make them healthier and restore their functions. These technologies are under development to be translated into therapeutics with the promise that one day could rejuvenate the aged cells in the body so they can become the younger version of themselves, repeating the process over time when needed.

Among many of the drivers of the aging process, there is one that seems to stands out as the lower hanging fruit among the emerging space of the longevity therapeutics. This is cellular senescence. Every damage that occurs to the cells in our body can push the cells to stop what they are doing and activate a safety mechanism that locks them into an arrested state called cellular senescence. Senescent cells cannot replicate anymore preventing them to cause additional damage, such as becoming cancer cells. All sort of damage can trigger this response leading to cellular senescence such as, oxidative stress, mitochondrial dysfunctions, DNA damage, viral infection, cigarette smoking, pollutions, chemicals, etc. They all can induce that safety lock and push damage cells to become senescent.

Senescent cells dont die easily but they stick around in the tissue, accumulating slowly over time. Importantly, cellular senescence is a pleiotropic mechanism, meaning it can be both good or bad. When we are young, we can efficiently get rid of senescent cells. The body uses them positively such as for tissue repair, wound healing or tissue remodeling. However, as we age, and our immune system ages (partially trough cellular senescence, a phenomenon called immune-senescence), our body become less efficient in removing senescent cells, which then start to accumulate.

Being able to make a new generation of drugs that are very selective for senescent cells, will enable the promise to achieve rejuvenative clinical results in humans similarly to what we found in preclinical results. On that end, we recently published a targeted strategy with the goal to advance the field in that direction (Doan et al., 2020). Using a prodrug, we engineered a small molecule to generate a selective senolytic compound to develop a targeted therapy. This prodrug is inactive in non-senescent cells but activated by senescent cells, taking advantage of an enzymatic function of those cells. In geriatric mice this prodrug showed to be well tolerated but also efficacious to clear senescent cells, resulting in restored cognitive functions, muscle functions, stem cells functions, vitality and overall health. As we advance senolytic drugs to the clinic to treat age-related diseases, it is very important to be mindful that elderly individuals, who are frail, with co-morbidities and exposed to multiple medications, will not well tolerate drugs that are not safe and effective. Importantly, not all senescent cells are the same. They are rare, interspersed in the tissues but are also very heterogeneous. Being able to hit the right senescent cells, in the right diseased tissue will be key to enable effective therapies. Developing drugs that are very potent, selective and potent and safe will be pivotal.

The longevity therapeutics space is emerging, but is already disrupting the medical industry. The goal of longevity therapeutics is not just to add years to life, extending lifespan. The true goal is to add life to years and extend health span. A target that gets closer every day.

Marco Quarta is CEO, Rubedo Life Sciences.

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The Rise of Longevity Therapeutics - Pharmaceutical Executive

Skin Stem Cells Comments Off on The Rise of Longevity Therapeutics – Pharmaceutical Executive | May 28th, 2021

CRANFORD, N.J., May 25, 2021 /PRNewswire/ --Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products with a focus on anti-infective products in adjunct cancer care, unique prescription products and stem cell therapy, today announced that it has received the Best Poster Award at the prestigious International Society for Cell and Gene Therapy (ISCT) 2021 Annual Meeting.

The poster, titled "Novel Induced-Mesenchymal Stem Cells (i-MSCs) Attenuate Severity of ARDS in Septic Sheep," will be presented today, May 25, 2021 by Dr. Perenlei Enkhbaatar, Professor and Director of the Translational Intensive Care Unit at The University of Texas Medical Branch.

"The ISCT annual meeting brings together the brightest minds in cell and gene therapy and highlights cutting edge research in the field," stated Dr. Myron Czuczman, Chief Medical Officer and Executive Vice President of Citius. "We are honored to be selected for the Best Poster Award from among this distinguished peer group. The interim results demonstrate a marked improvement in i-MSC treated animals over control animals in key clinical parameters including: improved oxygenation, less systemic shock, and reduced bacterial burden and vascular injury to the lungs. We are encouraged by the data and welcome the support and engagement of the scientific research community," concluded Dr. Czuczman.

Myron Holubiak, President and Chief Executive Officer of Citius added, "We are grateful to be recognized by our peers for this award as we advance our novel stem cell program for the treatment of ARDS. In parallel to the expansion of our proof-of-concept ARDS sheep study, we are following guidance from the U.S. Food and Drug Administration (FDA) in the development of a cGMP Master Cell Bank of i-MSCs. I am pleased to report that we have completed the development of an i-MSC Accession Cell Bank (ACB) which is to serve as the basis for a scalable cGMP compliant manufacturing capability to support all of our planned pre-clinical and clinical trials. Compared with donor-derived cells that require a continuous supply of new donors, we believe our i-MSCs,derived from a single clonal induced pluripotent stem cell (iPSC), offer multiple advantages including consistent and scalable manufacturing and a potentially limitless supply of i-MSCs to meet our future needs. Moreover, we believe that our i-MSC stem cell program has the potential to meaningfully impact the treatment of ARDS and we appreciate the recognition received from the cell and gene therapy community as we advance our program."

Citius' i-MSCs are derived from iPSCs originating from a qualified single-donor dermal fibroblast, resulting in one homogeneous, validated source for all future cells. A patented synthetic, non-immunogenic mRNA high efficiency cell reprogramming technique is applied to create a clonal iPSC Master Cell Bank from which our i-MSCs are differentiated and expanded to create an i-MSC Accession Cell Bank. Citius has completed the development of its i-MSC ACB and is currently testing (as per FDA guidance) and expanding the cells to create an allogeneic cGMP i-MSC Master Cell Bank to support all future i-MSC needs.

The poster will be available to conference attendees via the conference website. The poster will be available on Citius' website once the event commences.

Conference Details:

Abstract Title:

"Novel Induced-Mesenchymal Stem Cells (i-MSCs) Attenuate Severity of ARDS in Septic Sheep"

Authors:

K. Hashimoto, N. Bazhanov, P. Enkhbaatar, M. Angel, A. Lader, M. Czuczman, and M. Matthay

Abstract Number:

100

Date and Time:

May 25, 2021

Session I

12:30 2:00 PM EDT

Session II

8:00 9:30 PM EDT

About Acute Respiratory Distress Syndrome (ARDS)

ARDS is an inflammatory process leading to build-up of fluid in the lungs and respiratory failure. It can occur due to infection, trauma and inhalation of noxious substances. ARDS accounts for approximately 10% of all ICU admissions and almost 25% of patients requiring mechanical ventilation. Survivors of ARDS are often left with severe long-term illness and disability. ARDS is a frequent complication of patients with COVID-19. ARDS is sometimes initially diagnosed as pneumonia or pulmonary edema (fluid in the lungs from heart disease). Symptoms of ARDS include shortness of breath, rapid breathing and heart rate, chest pain (particularly while inhaling), and bluish skin coloration. Among those who survive ARDS, a decreased quality of life is relatively common.

About Citius Pharmaceuticals, Inc.

Citius is a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products, with a focus on anti-infectives in adjunct cancer care, unique prescription products, and stem cell therapy. The Company's lead product candidate, Mino-Lok, an antibiotic lock solution for the treatment of patients with catheter-related bloodstream infections (CRBSIs), is currently enrolling patients in a Phase 3 pivotal superiority trial. Mino-Lok was granted Fast Track designation by the U.S. Food and Drug Administration (FDA). Through its subsidiary, NoveCite, Inc., Citius is developing a novel proprietary mesenchymal stem cell treatment derived from induced pluripotent stem cells (iPSCs) for acute respiratory conditions, with a near-term focus on Acute Respiratory Distress Syndrome (ARDS) associated with COVID-19. For more information, please visit http://www.citiuspharma.com.

Safe Harbor

This press release may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements are made based on our expectations and beliefs concerning future events impacting Citius. You can identify these statements by the fact that they use words such as "will," "anticipate," "estimate," "expect," "plan," "should," and "may" and other words and terms of similar meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated are: risks relating to the results of research and development activities, including those for our NoveCite stem cell therapy; uncertainties relating to preclinical and clinical testing; the early stage of products under development; our dependence on third-party suppliers; our ability to successfully undertake and complete clinical trials and the results from those trials for our product candidates; the estimated markets for our product candidates and the acceptance thereof by any market; the ability of our product candidates to impact the quality of life of our target patient populations; our need for substantial additional funds; market and other conditions; risks related to our growth strategy; patent and intellectual property matters; our ability to attract, integrate, and retain key personnel; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; our ability to identify, acquire, close and integrate product candidates and companies successfully and on a timely basis; our ability to procure cGMP commercial-scale supply; government regulation; competition; as well as other risks described in our SEC filings. These risks have been and may be further impacted by Covid-19. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings which are available on the SEC's website at http://www.sec.gov, including in our Annual Report on Form 10-K for the year ended September 30, 2020, filed with the SEC on December 16, 2020 and updated by our subsequent filings with the SEC. These forward-looking statements speak only as of the date hereof, and we expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Investor Relations for Citius Pharmaceuticals:

Andrew ScottVice President, Special ProjectsT: 908-967-6677 x105E: [emailprotected]

Ilanit AllenVice President, Corporate Communications and Investor RelationsT: 908-967-6677 x113E: [emailprotected]

SOURCE Citius Pharmaceuticals, Inc.

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Citius Pharmaceuticals Selected to Receive Best Poster Award at the International Society for Cell and Gene Therapy 2021 Annual Meeting - PRNewswire

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If you have certain types of cancer, your doctor might suggest Keytruda (pembrolizumab) as a treatment option for you.

Keytruda is a prescription medication thats used to treat certain advanced forms of the following kinds of cancer in adults and some children:

Keytruda can also be used to treat these kinds of cancer in some children as well as adults:

Keytruda comes as a solution a healthcare professional injects into your vein over a period of time. This is called an intravenous infusion.

Keytruda is a biologic, which is a treatment made from parts of living organisms. It isnt available in a biosimilar form. Biosimilars are like generic drugs. But unlike generics, which are made for non-biologic drugs, biosimilars are made for biologic drugs.

For more information about Keytruda, including details about its uses, see this in-depth article on the drug.

Like other drugs, Keytruda can cause mild and serious side effects. Keep reading to learn more.

Some people may experience mild or serious side effects during their Keytruda treatment. These side effects can vary depending on whether Keytruda is used alone or with other cancer drugs.

Examples of Keytrudas commonly reported side effects include:

* To learn more about this side effect, see Side effects explained below.

Read on to learn about other possible side effects of Keytruda.

Keytruda may cause mild side effects. These side effects can vary depending on whether Keytruda is used alone or with other cancer drugs.

Examples of mild side effects that have been reported with Keytruda include:

* To learn more about this side effect, see Side effects explained below.

In most cases, these side effects should be temporary. And some may be easily managed, too. But if you have any symptoms that are ongoing or that bother you, talk with your doctor or pharmacist. And dont stop using Keytruda unless your doctor tells you to.

Keytruda may cause mild side effects other than the ones listed above. See the Keytruda medication guide for more information.

Note: After the Food and Drug Administration (FDA) approves a drug, it tracks side effects of the medication. If youd like to notify the FDA about a side effect youve had with Keytruda, visit MedWatch.

Serious side effects may occur with Keytruda. These side effects can vary depending on whether Keytruda is used alone or with other cancer drugs.

Many of Keytrudas serious side effects happen because of an overactive immune system. These are called immune-mediated side effects, and they often cause inflammation (damage and swelling) to tissues. Examples include:

Other serious side effects that have been reported with Keytruda include:

* To learn more about this side effect, see Side effects explained below.

If you develop serious side effects while using Keytruda, call your doctor right away. If the side effects seem life threatening or if you think youre having a medical emergency, immediately call 911 or your local emergency number.

Get answers to some frequently asked questions about Keytrudas side effects.

In most cases, Keytrudas side effects should be temporary. Most should go away soon after you start or stop the drug.

But Keytruda can cause serious side effects that may lead to long-term problems. In some cases, these problems can take many weeks or months to resolve. Here are some examples, all of which cause inflammation (damage and swelling) in different parts of the body:

If you have questions about what to expect long term while using Keytruda, talk with your doctor or pharmacist. But dont stop using Keytruda unless your doctor recommends it.

Yes, in rare cases, Keytruda may cause serious eye side effects (sometimes called ocular side effects).

Examples of eye problems that may happen while using Keytruda include:

Symptoms of eye side effects from Keytruda will depend on the exact eye problem you have. But possible symptoms that may happen with one or both eyes include:

Tell your doctor right away if you have any symptoms of eye problems while using Keytruda.

Keytruda is prescribed to treat many types of cancer, including non-small cell lung cancer and small cell lung cancer. The side effects of Keytruda are expected to be the same regardless of the type of cancer its treating. For a full list of the cancers Keytruda is used to treat, see this in-depth article on the drug.

To learn more about possible side effects of Keytruda, see the What are the mild side effects of Keytruda? and What are the serious side effects of Keytruda? sections above.

If you have questions about what to expect when using Keytruda to treat lung cancer, talk with your doctor.

Yes, confusion is a possible side effect of Keytruda. In fact, confusion was a common side effect of Keytruda in studies of the drug.

Confusion can make you feel as though you cant think clearly. You may also have problems making decisions or focusing on a task. This side effect can also lead to abnormal or slurred speech.

Its important to remember that encephalitis (inflammation of your brain) may cause confusion. Encephalitis is a rare but serious side effect of Keytruda. For this reason, you should tell your doctor right away if you experience confusion while using Keytruda. Theyll likely check you for signs of encephalitis.

Learn more about some of the side effects Keytruda may cause.

Muscle pain or bone pain are common side effects of Keytruda.

You can relieve muscle or bone pain by:

Before using OTC drugs with Keytruda, talk with your doctor or pharmacist. And ask your doctor about other ways to relieve bothersome muscle or bone pain that Keytruda may cause.

In rare cases, some people may have hair loss while using Keytruda. In studies, hair loss was more common when Keytruda was used with chemotherapy drugs than when used alone.

Hair loss as a side effect of Keytruda is usually temporary. If you have hair loss from using Keytruda, your hair should start growing back several weeks after your last dose.

Cooling caps, which are caps designed to keep your scalp cold, might help prevent hair loss. Cooling caps lessen the blood flow to your scalp, which may decrease the effect of Keytruda or chemotherapy on your hair. Ask your doctor if a cooling cap is right for you.

When your hair does start to return, dont overuse hair styling tools that are harsh on hair. These include blow dryers and hair straighteners. You should also avoid bleaching or coloring your hair so it stays healthy enough to grow.

If you experience bothersome hair loss while using Keytruda, talk with your doctor about ways to help with this side effect.

You may have itchy skin or rash from using Keytruda. Itchy skin and mild rashes are common side effects of the drug.

In rare cases, Keytruda may also cause severe rashes and other skin reactions. These include Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN). With SJS and TEN, you have a rash along with painful sores on your eyes, genitals, mouth, or throat.

Here are a few tips for helping relieve itching and rash:

If you have a severe skin reaction to Keytruda, youll likely need treatment in a hospital. If you have severe skin peeling or blisters after using the drug, call 911 or your local emergency number right away. These may be signs of a serious skin reaction, which can be life threatening.

If youre concerned about your risk for a severe skin reaction from using Keytruda, talk with your doctor.

Like most drugs, Keytruda can cause an allergic reaction in some people.

Symptoms can be mild or serious and can include:

If you have mild symptoms of an allergic reaction, such as a mild rash, call your doctor right away. They may suggest an over-the-counter antihistamine you can take by mouth, such as diphenhydramine (Benadryl), or a product you can apply to your skin, such as hydrocortisone cream, to manage your symptoms.

If your doctor confirms you had a mild allergic reaction to Keytruda, theyll decide if you should continue using it.

If you have symptoms of a severe allergic reaction, such as swelling or trouble breathing, call 911 or your local emergency number right away. These symptoms could be life threatening and require immediate medical care.

If your doctor confirms you had a serious allergic reaction to Keytruda, they may have you switch to a different treatment.

During your Keytruda treatment, consider keeping notes on any side effects youre having. Then, you can share this information with your doctor. This is especially helpful to do when you first start taking new drugs or using a combination of treatments.

Your side effect notes can include things like:

Keeping notes and sharing them with your doctor will help your doctor learn more about how Keytruda affects you. And your doctor can use this information to adjust your treatment plan if needed.

Keytruda is used to treat certain types of cancer in some children. (For information about the cancers Keytruda can treat in children, see this detailed article on the drug.)

Most side effects that occur in children receiving Keytruda are similar to those that adults experience. However, some side effects of Keytruda are more common in children. These include:

Talk with your childs doctor about their risk for side effects from Keytruda.

Keytruda may not be right for you if you have certain medical conditions or other factors that affect your health. Talk with your doctor about your health history before you take Keytruda. Factors to consider include those in the list below.

Allergic reaction. If youve had an allergic reaction to Keytruda or any of its ingredients, you shouldnt take Keytruda. Ask your doctor what other medications are better options for you.

Receiving certain other treatments for multiple myeloma. Using Keytruda with certain other treatments for multiple myeloma can be fatal. (Multiple myeloma is a cancer that affects a type of white blood cell called a plasma cell.) Before using Keytruda, tell your doctor if youre taking any treatments for multiple myeloma.

Received an organ transplant. Before using Keytruda, tell your doctor if youve had an organ transplant. Keytruda can raise the risk for your immune system attacking the transplanted organ. If youve had a transplant, your doctor will tell you what symptoms of organ rejection you should watch for while using Keytruda.

Received or plan to receive a stem cell transplant. Before using Keytruda, tell your doctor if youve received stem cells from a donor in the past or plan to do so. You may be at a higher risk for graft-versus-host disease. This condition causes your immune system to attack the transplant stem cells. Talk with your doctor about whether Keytruda is safe for you to use.

It should be safe to drink alcohol while using Keytruda.

But be aware that alcohol can cause side effects that are similar to some of Keytrudas. These include diarrhea, fatigue (lack of energy), and nausea. If you drink alcohol during Keytruda treatment, it may make these side effects worse.

Talk with your doctor about the amount of alcohol thats safe for you to drink while using Keytruda.

You shouldnt use Keytruda while pregnant or breastfeeding.

Keytruda hasnt been studied during pregnancy. But based on how the drug works, Keytruda may cause harm to infants born to pregnant females* who used the drug during pregnancy.

For this reason, you should use birth control while taking Keytruda if you or your partner can become pregnant. And you should continue to use birth control for at least 4 months after your last dose.

It isnt known if Keytruda can pass into breast milk. To be safe, you shouldnt breastfeed while using Keytruda and for at least 4 months after your last dose.

Before starting Keytruda treatment, tell your doctor if youre pregnant or planning to become pregnant. Also tell them if youre breastfeeding or planning to breastfeed. They can discuss your options with you.

* In this article, we use the term female to refer to someones sex assigned at birth. For information about the difference between sex and gender, see this article.

Keytruda is a drug used to treat certain types of cancer in adults and some children.

Some people who use Keytruda may have mild side effects. Although rare, serious side effects can occur with Keytruda. Many of these happen because of an overactive immune system. Keep in mind that the side effects of Keytruda can vary depending on whether Keytruda is used alone or with other cancer drugs.

Talk with your doctor or pharmacist if you have questions about Keytrudas side effects. Here are a few questions you may want to ask:

Disclaimer: Healthline has made every effort to make certain that all information is factually correct, comprehensive, and up to date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or other healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.

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Keytruda Side Effects: What They Are and How to Manage Them - Healthline

Skin Stem Cells Comments Off on Keytruda Side Effects: What They Are and How to Manage Them – Healthline | May 28th, 2021

Founded to meet the future protein demands of an expanding global population, Australias Magic Valley is developing cell-cultured lamb products including mince, strips, steaks and chops. With lambs currently slaughtered at an incredibly young age using traditional farming methods, its founder tells us this particular meat became the obvious choice for the companys first product range.

There is absolutely no need for the mass slaughter of animals for food and hopefully intensive animal agriculture will soon be a thing of the past

Vegconomist spoke with Founder Paul Bevan, who says that he had become frustrated by the pace of change and effectiveness of his own activism so he turned his attention to technology, specifically the development of slaughter-free cultured meat, beginning with lamb.

Utilising induced pluripotent stem-cells and FBS-free media, Magic Valley is able to grow real animal meat from animal cells, using animals such as Lucy, who Paul refers to as cell volunteers.

Eventually we would like to expand into developing cultured meat products for all other animal species

Lucy the lamb is our very special cell donor. From just a tiny skin biopsy less than 4mm in diameter we are able to generate an infinite number of muscle and fat cells without ever having to interfere with an animal again. That is one of the distinct advantages of our technology and using induced pluripotent stem cells.

Meanwhile, Lucy gets to live out the entirety of her natural life (up to 20 years of age) happy and unharmed, blissfully unaware that her cell donation has potentially saved the lives of billions of lambs that would otherwise have been slaughtered at just 6 months of age.

Magic Valleys team consisting of Australias leading scientists have extensive experience in both stem cell biology and livestock production. As part of its ambitions to become a leader in the field, the company also announced this week the onboarding of industry pioneer Dr. Sandhya Sriram, PhD, Co-Founder & CEO of the cell-based crustacean producers Shiok Meats, to its advisory board.

Eventually we would like to expand into developing cultured meat products for all other animal species that have traditionally been farmed for human consumption. With the advancement of this technology, there is absolutely no need for the mass slaughter of animals for food and hopefully intensive animal agriculture will soon be a thing of the past, Bevan commented to vegconomist.

Our immediate goal is to develop the safest, healthiest and tastiest cultured lamb products possible. We know that to be successful, cultured meat products have to become the obvious choice for consumers and that means taste, price & convenience are paramount. We know that ethical or environmental concerns alone are not enough to change consumer behaviour it has to be a better product.

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DUBLIN--(BUSINESS WIRE)--The "Global Stem Cell Therapy Market by Type (Allogeneic, Autologous), Therapeutic Application (Musculoskeletal, Wound & Injury, CVD, Autoimmune & Inflammatory), Cell Source (Adipose tissue, Bone Marrow, Placenta/Umbilical Cord) - Forecasts to 2026" report has been added to ResearchAndMarkets.com's offering.

The global stem cell therapy market is projected to reach USD 401 million by 2026 from USD 187 million in 2021, at a CAGR of 16.5% during the forecast period.

Growth in this market is majorly driven by the increasing investment in stem cell research and the rising number of GMP-certified stem cell manufacturing plants. However, factors such as ethical concerns and the high cost of stem cell research and manufacturing process likely to hinder the growth of this market.

The allogeneic stem cell therapy segment accounted for the highest growth rate in the stem cell therapy market, by type, during the forecast period

The stem cell therapy market is segmented into allogeneic and autologous stem cell therapy. Allogeneic stem therapy segment accounted for the largest share of the stem cell therapy market. The large share of this segment can be attributed to the lesser complexities involved in manufacturing allogeneic-based therapies.

This segment is also expected to grow at the highest growth rate due to the increasing number of clinical trials in manufacturing allogeneic-based products.

Bone Marrow-derived MSCs segment accounted for the highest CAGR

Based on the cell source from which stem cells are obtained, the global stem cell therapy market is segmented into four sources. These include adipose tissue-derived MSCs (mesenchymal stem cells), bone marrow-derived MSCs, placenta/umbilical cord-derived MSCs, and other cell sources (which include human corneal epithelium stem cells, peripheral arterial-derived stem cells, and induced pluripotent stem cell lines).

The bone marrow-derived MSCs segment is expected to witness the highest growth rate during the forecast period, owing to an increasing number of clinical trials focused on bone marrow-derived cell therapies and the rising demand for these cells in blood-related disorders.

Asia Pacific: The fastest-growing country in the stem cell therapy market

The stem cell therapy market is segmented into North America, Europe, Asia Pacific, RoW. The stem cell therapy market in the Asia Pacific region is expected to grow at the highest CAGR during the forecast period.

Factors such as the growing adoption of stem cell-based treatment in the region and the growing approval & commercialization of stem cell-based products for degenerative disorders drive the growth of the stem cell therapy market in the region.

Market Dynamics

Drivers

Restraints

Opportunities

Challenges

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/qiagh1

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Bone Marrow Stem Cells Comments Off on Stem Cell Therapy Market by Type, Therapeutic Application and Cell Source – Global Forecasts to 2026 – ResearchAndMarkets.com – Business Wire | May 15th, 2021

Expansion and persistence of UCARTCS1A was observed and was found to correlate with clinically meaningful antimyeloma activity and serum cytokine changes in very heavily pretreated patients with multiple myeloma. Also, the CAR T-cell product was noted to be detectable in patients, regardless of donor and batch.

These preliminary data validate CS1 as a target for CAR T-cell products in multiple myeloma and that UCARTCS1A is a promising potential therapy for [those with this disease], Krina K. Patel, MD, MSc, an associate professor of the Department of Lymphoma/Myeloma, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, said during a presentation on the results.

One of the benefits that comes with utilizing an allogeneic CAR T-cell approach over an autologous approach is that it affords the opportunity for off-the-shelf product availability, according to Patel. Patients are able to avoid a prolonged wait for the CAR T cells to be manufactured; the cells are able to be administered within a couple of weeks, Patel explained. In contrast, it can take 4 to 5 weeks to bring an autologous product to a treatment center.

Scalable manufacturing is another benefit of allogeneic approaches, and this can reduce costs and yield 100 or more doses from 1 batch of donor cells. Also, for allogeneic approaches, T cells are collected from healthy donors; these patients have not been given many steroids, chemotherapy, or have undergone autologous transplant. As such, their T cells will likely be more potent, Patel explained. Lastly, more flexible dosing is an option with allogeneic approaches; this allows for the possibility of redosing and alternate schedules.

UCARTCS1A is the first allogeneic CAR T-cell product developed to target CS1 and SLAMF7, both of which are highly and consistently expressed in multiple myeloma, according to Patel. The product knocks out the TRAC gene to avoid graft-versus-host disease through disruption of T-cell receptor (TCR) assembly. The product also knocks out CS1 to facilitate robust expansion and yield, while avoiding fratricide. Lastly, UCARTCS1A has a RQR8 safety switch, which is a CD20 mimotope that can use rituximab (Rituxan) to kill the cells, if necessary.

Previously, the CAR T-cell product demonstrated durable in vivo efficacy against MM1S tumors. Here, NSG mice were given a 5 x 105 MM1S myeloma cell line, which is known to be pretty aggressive, Patel noted; this was labeled with GFP and was given for 10 days. Subsequently, the mice received the CAR T cells. Investigators observed CAR-positive cells at day 4 and M protein, which is a surrogate marker for multiple myeloma in mice and patients.

We were able to see an early response, as well. However, eventually, the T cells went down, and the myeloma started to go back up, Patel added. Looking at the imaging, mice who [received] CAR T cells obviously did much better and lived longer and there was a dose-dependent response where the mice that got the higher dose did better, with a much longer survival. Investigators were also able to demonstrate that the mice that received the CAR T-cell therapy experienced improvement in lytic lesions over time.

MELANI-01 enrolled patients with confirmed multiple myeloma per International Myeloma Working Group criteria who relapsed following previous therapy for their disease. To be eligible for enrollment, patients needed to have an ECOG performance status of 0 to 2 and acceptable organ function. They could have not previously received an investigational drug or cell/gene therapy targeting CS1.

The key eligibility [for this trial] is similar to most cell therapy trials [that are done in] myeloma. However, for most of those trials, patients are not able to have previously received CAR T cells or BCMA-directed therapies, Patel said. In this trial, [those are not] ineligibility [criteria]. Our patients had really relapsed/refractory [disease.]

After going through screening, patients received lymphodepletion chemotherapy that was comprised of fludarabine at a daily dose of 30 mg/m2 for 3 days followed by cyclophosphamide at a daily dose of 1 g/m2, also for 3 days. The [cyclophosphamide] dose was 2 to 3 times higher than what [has been] used in most other trials, Patel noted.

Patients then received treatment with UCARTCS1A. Patients were started at dose level 1, where they received 1 x 106/kg. One patient went on to dose level 2, which was 3 x 106/kg. Patients underwent their first disease evaluation at day 28.

The primary and secondary objectives of the study included safety and tolerability of UCARTCS1A, as well as determining the maximum-tolerated dose and efficacy of the product. Exploratory end points are examining expression of CS1 on multiple myeloma cells, UCARTCS1A expansion and persistence, and changes in serum biomarkers or immune cell reconstitution.

Patel shared information on 5 patients who received treatment with UCARTCS1A to date; 4 of the patients received dose level 1 (102-101, 102-109, 102-107, and 102-111) and 1 patient (102-113) received dose level 2.

Four of the 5 patients (102-101, 102-109, 102-107, and 102-113) had previously received over 11 lines of therapy and had most had previously received a BCMA-directed therapy. Just to put this into context, most of the autologous CAR T-cell trials that are done have patients who had a median of 5 to 6 prior lines of treatment, Patel noted.

One patient (102-111) had received only 4 prior lines of therapy and was the only patient who had cells expand and responded on dose level 1. However, the patient had very high-risk disease with 90% plasma cells. He had the most myeloma going into the trial, Patel said.

Notably, patient 102-113 who had received dose level 2 and also experienced an expansion of cells at day 7 had received 13 prior lines of therapy, including 2 prior BCMA-targeted CAR T-cell therapies, the last of which was administered just 5 months prior to the study.

Patient 102-111 was 55 years of age, had 4 prior lines of therapy and 90% of bone marrow involvement. He had relapsed within 6 months of every prior line of therapy and he never experienced more than a partial response (PR) to any of his prior treatments, according to Patel. When looking at his peripheral blood at day 28, investigators noted that the CD45+ CAR-positive lymphocytes was almost 72% and a subgroup of CD8+ effector cells that are TCRnegative CAR-positive cells, were about 46%.

[Some might] think that allogenic cells would not last very long, but for this patient, we definitely saw the majority of T cells still there that were CAR positive, Patel said. For him, we were able to get a bone marrow [sample] at month 3, where we could also see CD45+ CAR-positive cells at 60% in the bone marrow of all CD45+ cells. The CD8+ effector [cells] were at 92%.

Moreover, CAR-positive cells were observed in the patients peripheral blood starting at day 14; they peaked at day 21, and then started to decrease. However, some of these cells were still observed at day 80 to 86, according to Patel. The patients white blood cell count was low, while peripheral blood was high, until approximately day 28, before it started decreasing. However, the patients bone marrow remained high, even at day 77, in terms of the vector copy number of the CAR T cells.

This patient experienced grade 2 cytokine release syndrome (CRS) within the first week of cell infusion. The patient also developed hemophagocytic lymphohistiocytosis (HLH), which has previously been observed with other autologous CAR T-cell products in multiple myeloma. Investigators treated the patients with anakinra (Kineret), dexamethasone, etoposide, and the rituximab kill switch. The rationale for triggering the kill switch was because the patient had reactivation of HHV6, which developed into HHV6 encephalitis.

Per the FDA, we were monitoring HHV6 and HHV7 levels, as we do for most of our CAR T-cell therapy trials. We were monitoring this [and when his levels were high enough that we decided to treat], the patient got admitted for antivirals, improved, went home, and then came back with an encephalitis picture. Initially, we treated him dexamethasone and gave the rituximab kill switch thinking that if it was immune effector cell-associated neurotoxicity, we could kill off some of the cells. But in the end, it was HHV6 encephalitis.

Although the patient did improve, and he had double antiviral coverage, he eventually passed away on day 109 from organizing pneumonia in the context of prolonged lymphopenia in the absence of multiple myeloma progression.

At the time, he did not have any myeloma and he had [experienced] this response that he had never had before, a near complete response Patel explained. We looked at his bone marrow, which was minimal residual diseasenegative at the 10-5 level. However, because of the prolonged lymphopenia, he ended up with this infection.

Multiple factors may have contributed to the prolonged lymphopenia, including viral reactivation, concomitant antivirals, and recent prior stem cell transplant, Patel explained.

The other patient with expansion, patient 102-113, was observed to have 25% CD45+ CAR-positive lymphocytes in the peripheral blood at day 9, 77% of which were CD8+ effector cells, according to Patel. Notably, investigators were unable to collect a bone marrow sample from the patient. In the peripheral blood, investigators observed expansion at day 7 and then a peak, and then the vector copy number persisted over the time the blood samples were obtained.

This patient had previously received 14 lines of therapy, including 2 previous BCMA-directed CAR T-cell therapies and associated lymphodepleting regimens, autologous transplant, and venetoclax (Venclexta), as his last line of therapy. The patient did not have any options left and we saw this fantastic response, where the lambda light chains had gone done by almost 90%; his M protein had at least a PR by just day 14.

However, this patient had CRS and HLH, as well. We treated him with etoposide, anakinra, dexamethasone, and the rituximab kill switch and he had improvement in his platelet and his liver function tests, Patel added. The HLH clinically improved for him. However, at day 25, he passed away.

An autopsy revealed G5 hemorrhagic pancreatitis, although he had not exhibited any clinical signs of this condition during his hospital stay. Investigators also found disseminated mucormycosis and pseudomonal pneumonia.

Select serum cytokine changes over time were found to correlate with expansion of the CAR T-cell product. Cytokines were increased much more in the patients who expanded vs those who did not expand at all, Patel noted.

MELANI-01 is currently enrolling patients with protocol modifications, including restarting at dose level -1 (3 x 105). Moreover, lower doses of lymphodepleting chemotherapy are being administered now in an attempt to address lymphopenia and lead to added expansion. The trial will also have additional requirements for monitoring and managing patients with regard to opportunistic infections, as well as CRS and HLH.

Patel KK, Bharathan M, Siegel D, et al. UCARTCS1A, an allogeneic CAR T-cell therapy targeting CS1 in patients with relapsed/refractory multiple myeloma (RRMM): preliminary translational results from a first-in-human phase I trial (MELANI-01). 2021 American Society of Gene and Cell Therapy Annual Meeting; May 11-14, 2021; Virtual. Accessed May 13, 2021. Abstract 118.

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News Release

Tuesday, May 11, 2021

An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to research supported in part by the National Institutes of Health. The researchers found that 48 of 50 children who received the gene therapy retained their replenished immune system function two to three years later and did not require additional treatments for their condition, known as severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID. The findings were published today inthe New England Journal of Medicine.

ADA-SCID, which is estimated to occur in approximately 1 in 200,000 to 1,000,000 newborns worldwide, is caused by mutations in theADAgene that impair the activity of the adenosine deaminase enzyme needed for healthy immune system function. This impairment leaves children with the condition highly susceptible to severe infections. If untreated, the disease is fatal, usually within the first two years of life.

These findings suggest that this experimental gene therapy could serve as a potential treatment option for infants and older children with ADA-SCID, said Anthony S. Fauci, M.D., director of NIHs National Institute of Allergy and Infectious Diseases (NIAID). Importantly, gene therapy is a one-time procedure that offers patients the hope of developing a completely functional immune system and the chance to live a full, healthy life.

People with ADA-SCID can be treated with enzyme replacement therapy, but this treatment does not fully reconstitute immune function and must be taken for life, usually once or twice weekly. Transplants of blood-forming stem cells, ideally from a genetically matched sibling donor, can provide a more lasting solution. However, most people lack such a donor. Additionally, stem cell transplants carry risks such asgraft-versus-host disease and side effects from chemotherapy medications given to help the donor stem cells establish themselves in the patients bone marrow.

The new research evaluated an experimental lentiviral gene therapy designed to be safer and more effective than previously tested gene-therapy strategies for ADA-SCID. This gene therapy involves inserting a normal copy of theADAgene into the patients own blood-forming stem cells. First, stem cells are collected from the patients bone marrow or peripheral blood. Next, a harmless virus is used as a vector, or carrier, to deliver the normalADAgene to these cells in the laboratory. The genetically corrected stem cells then are infused back into the patient, who has received a low dose of the chemotherapy medication busulfan to help the cells establish themselves in the bone marrow and begin producing new immune cells.

The experimental gene therapy, developed by researchers from the University of California, Los Angeles (UCLA) and Great Ormond Street Hospital (GOSH) in London, uses a modified lentivirus to deliver the ADA gene to cells. Previous gene-therapy approaches for ADA-SCID have relied on a different type of virus called a gamma retrovirus. Some people who have received gamma retroviral gene therapies have later developed leukemia, which scientists suspect is due to the vector causing activation of genes that control cell growth.The lentiviral vector is designed to avoid this outcome and to enhance the effectiveness of gene delivery into cells.

The results come from three separate Phase 1/2 clinical trials, two conducted in the United States and one in the United Kingdom. The U.S. trials, led by principal investigator Donald Kohn, M.D., of UCLA, enrolled 30 participants with ADA-SCID ranging in age from 4 months to 4 years at UCLA Mattel Childrens Hospital and the NIH Clinical Center in Bethesda, Maryland. The U.K. study, conducted at GOSH and led by principal investigator Claire Booth, M.B.B.S., Ph.D., enrolled 20 participants ranging in age from 4 months to 16 years. Most participants acquired and retained robust immune function following gene therapy 96.7% after two years in the U.S. studies and 95% after three years in the U.K. study and were able to stop enzyme replacement therapy and other medications. Of the two participants for whom gene therapy did not restore lasting immune function, one restarted enzyme replacement therapy and later received a successful stem cell transplant from a donor, and the other restarted enzyme replacement therapy. The lentiviral gene therapy appeared safe overall, although all participants experienced some side effects. Most of these were mild or moderate and attributable to the chemotherapy that the participants received.

Researchers observed similar outcomes in all three trials, although there were some differences between the studies. Stem cells were collected from bone marrow in the U.S. trials and from peripheral blood in the U.K. trial. In one of the U.S. trials, 10 children were treated with genetically corrected stem cells that had been frozen and later thawed. The two other trials used fresh stem cell preparations. In the future, the freezing procedure known as cryopreservation may allow stem cells to be more easily transported and processed at a manufacturing facility far from the patients home and shipped back to a local hospital, reducing the need for patients to travel long distances to specialized medical centers to receive gene therapy. A trial of the cryopreserved treatment is now underway at the Zayed Centre for Research into Rare Diseases in Children in London, in partnership with GOSH.

For more information about the trials described in the New England Journal of Medicine paper, visit ClinicalTrials.gov under identifiers NCT01852071, NCT02999984 and NCT01380990. The investigational lentiviral gene therapy, which is licensed to Orchard Therapeutics, has not been approved for use by any regulatory authority.

The research was funded in part by three NIH Institutes: NIAID; the National Heart, Lung and Blood Institute; and the National Human Genome Research Institute. Additional funding was provided by the California Institute for Regenerative Medicine, the Medical Research Council, the National Institute for Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children National Health Service Foundation Trust and University College London, and Orchard Therapeutics.

NIAID conducts and supports research at NIH, throughout the United States, and worldwide to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses. News releases, fact sheets and other NIAID-related materials are available on the NIAID website.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

DB Kohn, C Boothet al. Autologousex vivolentiviral gene therapy for adenosine deaminase deficiency.New England Journal of MedicineDOI: 10.1056/NEJMoa2027675 (2021).

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