Conference call and webcast on Friday, November 6 at 2:30 pm CET/8:30 am ET

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ERYTECH Provides Business Update and Reports Financial Results for the Third Quarter of 2020

Global News Feed Comments Off on ERYTECH Provides Business Update and Reports Financial Results for the Third Quarter of 2020 | November 5th, 2020

NEW YORK, Nov. 05, 2020 (GLOBE NEWSWIRE) -- Cellectis (Euronext Growth: ALCLS – Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on allogeneic gene-edited CAR T-cells (UCART), today announced its results for the three-month and nine-month periods ending September 30, 2020.

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Cellectis Provides Business Update and Reports Financial Results for Third Quarter and First Nine Months 2020

Global News Feed Comments Off on Cellectis Provides Business Update and Reports Financial Results for Third Quarter and First Nine Months 2020 | November 5th, 2020

WINNIPEG, Manitoba, Nov. 05, 2020 (GLOBE NEWSWIRE) -- Kane Biotech Inc. (TSX-V:KNE; OTCQB:KNBIF) (“Kane Biotech”) today announced that it has entered into a one year credit agreement (the “Credit Agreement”) with Pivot Financial Inc. (“Pivot”) for a non-revolving term loan in the aggregate amount of $1,480,000.00 (the “Credit Facility”).

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Kane Biotech Enters into Credit Facility with Pivot Financial

Global News Feed Comments Off on Kane Biotech Enters into Credit Facility with Pivot Financial | November 5th, 2020

VANCOUVER, British Columbia and SEATTLE, Nov. 05, 2020 (GLOBE NEWSWIRE) -- Chinook Therapeutics, Inc. (NASDAQ: KDNY), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of precision medicines for kidney diseases, today announced third quarter 2020 financial results and provided a business update.

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Chinook Therapeutics Reports Third Quarter 2020 Financial Results and Provides Business Update

Global News Feed Comments Off on Chinook Therapeutics Reports Third Quarter 2020 Financial Results and Provides Business Update | November 5th, 2020

BRIDGEWATER, N.J., Nov. 05, 2020 (GLOBE NEWSWIRE) -- Osmotica Pharmaceuticals plc (Nasdaq: OSMT) (“Osmotica” or the “Company”), a fully integrated biopharmaceutical company, today announced that the Company will release its 2020 third quarter financial results on Tuesday, November 10, 2020, after the close of the U.S. financial markets.

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Osmotica Pharmaceuticals plc to Provide Third Quarter 2020 Business and Financial Update on November 10, 2020

Global News Feed Comments Off on Osmotica Pharmaceuticals plc to Provide Third Quarter 2020 Business and Financial Update on November 10, 2020 | November 5th, 2020

Call scheduled for November 9th at 8:30 a.m. EST Call scheduled for November 9th at 8:30 a.m. EST

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Genocea to Host Investor Call Highlighting New GEN-009 Clinical and Immunogenicity Data to be Presented at Virtual SITC 2020

Global News Feed Comments Off on Genocea to Host Investor Call Highlighting New GEN-009 Clinical and Immunogenicity Data to be Presented at Virtual SITC 2020 | November 5th, 2020

Merger completed with Rexahn Pharmaceuticals creating a Nasdaq-listed Biopharmaceutical Company Focused on Advancing Ocuphire’s Late-Stage Clinical Pipeline of Ophthalmic Drug Candidates

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Ocuphire Pharma Completes Transactions and Begins Trading on Nasdaq as OCUP

Global News Feed Comments Off on Ocuphire Pharma Completes Transactions and Begins Trading on Nasdaq as OCUP | November 5th, 2020

MUSC Hollings Cancer Center researchers identified that blocking an alternative energy pathway for T-cells after hematopoietic stem cell transplant helps reduce graft-versus-host disease (GVHD) in an animal model of leukemia.

Xue-Zhong Yu, M.D., who also is associate director of Basic Science at Hollings, and collaborators at the Indiana University School of Medicine discovered that donor T-cells must have the key enzyme lysosomal acid lipase in order to induce GVHD.

The Yu laboratory focuses on understanding the biological balance between GVHD and graft-versus-leukemia effect. Hematopoietic stem cell transplantation is used as a treatment option for some leukemia patients. T-cells in stem cell grafts from a donor are given to a leukemia patient in order to kill the cancer and reboot the patient's immune system. GVHD is a big clinical challenge because the donor T-cells, which come from the bone marrow, can attack the patient's organs. Anywhere from 30% to 70% of patients develop acute GVHD after allogeneic bone marrow transplant and 15% die.

"When we deal with hematopoietic cell transplant, it is an important balance - blocking GVHD while still allowing T-cells to do their job and control the cancer," Yu said.

Each cell in our body has its own metabolic process. Cells convert the food that is eaten into energy in order to perform their intended functions. However, cellular metabolism is often altered in various diseases. Yu researches T-cell metabolism in order to understand the balance between graft-versus-host and graft-versus-leukemia responses.

Most cells in our body require oxygen to create energy efficiently. However, this research focused on lipid, or fat, metabolism. T-cells have special metabolic processes: Sometimes they multiply so rapidly that they need an extra source of energy from free fatty acids.

Lysosomal acid lipase is an enzyme that breaks the large lipids and cholesterol into individual free fatty acid building blocks. If that enzyme is missing, there are not enough free fatty acids for energy production. This changes the T-cell metabolism, which in turn changes T-cell function.

Clinically, broad spectrum immunosuppression drugs (steroids and rapamycin) are still used as the first line of care in patients with severe GVHD. However, Yu and collaborators hypothesized that changing T-cell metabolism could reduce GVHD after hematopoietic stem cell transplantation.

"We know that the gut is the primary organ affected by GVHD. Since the gut has less oxygen, the T-cells rely on free fatty acids and must use lysosomal acid lipase. We thought if we could remove or block the activity of that, we could reduce GVHD in the gut."

The Yu Laboratory collaborated with the Indiana University School of Medicine and used a lysosomal acid lipase-deficient mouse model. T-cells lacking lysosomal acid lipase were given to mice with leukemia. As a control, T-cells with lysosomal acid lipase from normal mice were given to another group of leukemia mice. Strikingly, the mice that received the T-cells without lysosomal acid lipase did not get severe GVHD. Additionally, the T-cells from the donor lysosomal acid lipase-deficient bone marrow still killed the leukemia cells.

To increase the clinical translational potential of the work, orlistat, the FDA-approved lysosomal acid lipase inhibitor was also tested in the leukemia model. Mice with leukemia were treated with orlistat every other day after receiving bone marrow from normal mouse donors. Similar to the first experiment with the lysosomal acid lipase-deficient bone marrow, blocking the activity of lysosomal acid lipase with orlistat greatly reduced GVHD while the graft-versus-leukemia effect was preserved.

Additionally, the researchers discovered that inhibiting the lysosomal acid lipase enzyme with orlistat reduced the number of pathogenic T-cells and increased the number of regulatory T-cells. The pathogenic T-cells are the ones that cause GVHD. Regulatory T-cells are one of the "braking mechanisms" of the immune system. They help to reduce the activity of the pathogenic T-cells and prevent GVHD damage.

Therefore, blocking lysosomal acid lipase activity with orlistat preferentially stopped the donor T-cells from damaging the gut but allowed the T-cells to function during circulation and kill the leukemia cells.

The researchers' future plan is to look deeper at the biological mechanisms. For example, it is not clear how the loss or inhibition of lysosomal acid lipase affects the other metabolites in T-cells. To move this finding closer to the clinic, Yu explained that human cells can be used in a special mouse model that recreates the human immune environment.

"Looking at the immune cells in the gut was technically challenging. However, the results were exciting because our hypothesis was validated. These results encourage us to continue studying this in order to provide better treatment options to patients."

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Blocking energy pathway reduces GVHD while retaining anti-cancer effects of T-cells - Science Codex

Bone Marrow Stem Cells Comments Off on Blocking energy pathway reduces GVHD while retaining anti-cancer effects of T-cells – Science Codex | November 5th, 2020

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Latest Study explores the Stem Cell Banking Market Witness Highest Growth in nea - GroundAlerts.com

Bone Marrow Stem Cells Comments Off on Latest Study explores the Stem Cell Banking Market Witness Highest Growth in nea – GroundAlerts.com | November 5th, 2020

David Shaw walks into the hospital room and takes a seat next to the bed. He does this nearly every day, right around lunchtime.

He looks at his younger brother, Eric, tubes snaking across his arms, machines beeping and whirring. Eric does not look like Eric anymore, his skin darkened, scars deepened, features altered. They both know this but never mention it.

Eric is dying, a rare, aggressive skin cancer rampaging through his body with such ferocity that his doctors are nearly out of options. Radiation failed. Chemotherapy failed. Two bone marrow transplants failed.

As Stanford's head football coach, David Shaw is relied on to always know what to say, how to say it and when to say it; but he cannot find the words now that he and his brother are staring down what seems to be an inevitable fate.

"What do you say, where you think you've pulled at the last thread and there are no more threads?" David said. "All I could tell him was that I loved him and that I was there for him. The rest of it was really just ... I thought it was only a matter of time before he passed away."

Two years later, what happened between David and Eric remains real, present and raw -- changing their entire relationship, redefining what it means to be a brother. The words are still difficult to say, so they tip-toe around the crushing physical and mental toll Eric's cancer took on them.

David and Eric are sure to think about it all this weekend, when Stanford opens its season at Oregon on Saturday. Because the last time the Cardinal visited Eugene, neither one knew whether Eric would live or die.

After Stanford came from behind to win that game 38-31 in overtime, David delivered a message at the end of his postgame television interview, looking at the camera and saying, "To my brotherEric: I love you." He tapped the lime green pin on his black Stanford sweatshirt before he left the screen.

When Shaw became head coach at Stanford in 2011, it was the culmination of a family journey. His father was a longtime coach there; David played receiver for the Cardinal and eventually returned as an assistant under Jim Harbaugh. The entire Shaw family -- parents Willie and Gay, along with David, Eric and their sister, Tawnya -- all call the Bay Area home.

To this day, David says the day he was introduced as coach was "one of the better days in all our lives."

Yet something started to happen to Eric that no one could quite figure out. That same year, Eric found strange looking spots on his torso. His wife, Crystal, noticed the first one under his arm. Maybe it was eczema, they thought. Then the spots started to spread. He went to the doctor. They prescribed an ointment, but the spots kept popping up, until they covered his entire body. Eventually, tumors started to grow. It looked as if someone had pushed marbles under his skin. Doctors remained confounded. Eric itched uncontrollably, insatiably. His skin itched so badly, it became difficult to put on clothes, shower, sleep and go to work. He eventually needed sleep medication so he could get uninterrupted rest.

Even then, he itched subconsciously, only realizing what happened when he woke up in the morning to find his arms and sheets covered in blood. Some nights, he tried to sleep on his forearms so his body wouldn't touch the sheets, because his skin grew too sensitive to any touch. At one point, he had more than 30 open wounds on his body.

"It's something that's so pervasive and so destructive that a lot of people have mental problems -- you can't do anything without extreme pain," Eric said. "You bleed a lot through the tumors, through the lesions, through the scratching. A lot of people don't survive, really, because of the mental stress that comes with it."

Doctors had a hard time diagnosing his disease because it is often confused with psoriasis, eczema or other skin conditions. Eventually, they determined he had a rare form of skin cancer called mycosis fungoides, a type of T-cell lymphoma that affects one in 6 million people in the United States and Europe. At the time, Eric Shaw was 38.

In 2013, he and Crystal pushed for a referral to Stanford Cancer Center, which has leading experts in the disease. Mycosis fungoides is so rare, it accounts for only 4% of all non-Hodgkin lymphoma cases; among those who suffer from it, only 20% have the type of itching Eric experienced. Rarer still is to find it in people under the age of 40, and African American men often end up with the worst prognosis. All the odds were firmly against him.

"When you first hear skin cancer, your mind doesn't go too far," David said. "So initially I was like, 'There are creams and other minor surgeries. I think it'll be OK.' And then Eric said, 'No, this is not the typical skin cancer. This is inside my body. This is inside the layers of my skin, and it's not one spot. It's everywhere.'

"I didn't really get it for weeks after that because, rectifying something that I didn't think was so serious to [then thinking] ... 'Oh my gosh. So this is really cancer. This is really scary now.' It took a long time for that to sink in."

David turned it over in his mind. He was the big brother, the protector, the one who always made sure Eric would be OK. They were supposed to raise their kids together, grow old together, and reminisce about the randomness of a life spent together.

He kept coming back to one thought: You're not supposed to lose your little brother.

David and Eric Shaw grew particularly close as children as they moved from place to place when their father, Willie, took new coaching jobs. Tawnya, their older sister, fit in anywhere socially. But David and Eric, who is two years younger, stuck together.

"Like a pair," David said.

They loved riding their bikes and, when they moved to Arizona, they took advantage of the wide-open spaces in the new development where they lived. They rode for miles and miles, setting up their own ramps and doing tricks and wheelies, visiting friends along the way before returning home after dark. They played sports, too, and though David loved football as much as their dad, the basketball court is where the brothers had their epic battles.

"I was always kind of a little bit stronger and I'll never forget the last time we played one-on-one basketball," David said. "He just got better than me, and he won, and once I got over the anger and disappointment, I was proud because my younger brother had grown and was gaining confidence."

Said Eric: "I wanted nothing more than to beat him, and he wanted nothing more than to keep beating me. But, during those times, it was just us, it was me and him. He was my best friend."

David went on to play at Stanford and eventually got into coaching, against his mother's best wishes. Eric did not pursue a career in athletics. He went to San Diego State and got into a career in marketing at a financial services company, where his gregarious nature, big smile and easy laugh made him a perfect fit. Though their personalities are different -- David is stoic and introspective, Eric makes anyone feel as if they have been friends forever -- they are grounded in the same values they learned at a young age: family and faith above everything else.

Those principles only grew stronger after they found themselves in the Bay Area as adults.

After David was hired by Stanford, the entire Shaw family made it clear it would always be around to support him. Family members all have a standing invitation to come for dinner on Tuesdays. And they always attend home football games, waving and hugging David during the team's pregame walk, cheering from the stands, and then waiting for some time together once the game ends.

Even as Eric grew sick, he made it a point to go cheer for his big brother. "It's not just the football game. Our family comes together," he said. "We celebrate, we come to watch the game and cheer the team on and support David. And then afterwards, win or lose, we all wait for him to come out. It's a family day. It's been wonderful to share that experience with David."

Stanford eventually drew them even closer, and it had nothing to do with football.

Eric did not understand the gravity of his situation until his first meeting in 2013 with the doctors at Stanford Cancer Center. They put it bluntly: He had such an aggressive form of the disease that he needed immediate treatment. They would start with total skin radiation, preparing Eric to lose his hair, eyebrows, eyelashes, fingernails and toenails.

If that did not work, they would try chemotherapy next.

"All these thoughts are running through your mind," Crystal said. "'Is he going to make it? Is it going to work? What's going to happen?' At the time, our youngest daughter was 3 months old, so it was pretty overwhelming. We were just putting our lives together and then boom: you're in the middle of this cancer war."

The next week, Eric took a leave of absence from work and began four-times-a-week trips from their home east of Palo Alto, California, to Stanford Hospital, often driving as many as three hours one way in traffic. When he arrived, he went into a box and his whole body was exposed to the radiation light for about an hour. Then, he would make the drive back home to see Crystal and their four kids -- Caleb Michael, Jared Spann-Shaw, Madison Shaw and Olivia Shaw.

The radiation charred his skin. He lost weight. When he looked in the mirror, Eric no longer recognized the man looking back at him.

"Nothing prepares you for something like this," he said. "Knowing that other people were looking at me and knowing that something was very wrong, that was a daily grind to get myself up out of bed and get ready for the day, knowing that that was going to be my life."

He did this for three straight months, all to keep the disease from growing to a point where it would kill him. It worked for a short time, but the disease came back more aggressively six months later. Doctors moved on to chemotherapy treatments, some of them experimental, but also began discussing the last-resort option: a bone marrow transplant.

David and Tawnya immediately volunteered to become donors, and underwent testing. In most cases, siblings are the best chance at a donor match. Unfortunately, in their case, neither was close. On a 10-point match scale, Tawnya registered a 3, David a 5. Neither qualified to donate.

"I wanted to jump to the front of the line and say, 'Whatever I have to do, whatever you have to take out of me, however you have to do it, just do it,'" David said. "For them to come back and say that you're not a strong enough match was disheartening. It hurt me. The fact that we had to put our trust and faith in people that we didn't know, and that we're going to have to go out to registries and try to find someone who was a better match than I was, that uncertainty, and that doubt, it's hard to keep it at bay at that point. It starts to creep in."

Doctors eventually found two donors whom they believed could work, but they were not perfect matches. In early 2018, Eric and his family moved into a two-bedroom apartment near Stanford Hospital to prepare for the transplant. For three months, he went through radiation, then chemotherapy to prepare his body to accept the donor cells.

He underwent the transplant in April, feeling confident and inspired it would work. After a month, doctors did an initial check to see how many of the donor cells had survived the transplant.

None survived.

"It was like I never even had the transplant," Eric said. "That was so devastating. We just knew it was going to work. I mean, we're people of faith, and we knew everybody was praying for us, and that we were praying that this six-year journey was going to finally be over. And it wasn't over. It was crushing for them to say, 'It didn't work. We're going to have to try again.'"

The second attempt happened in September. Crystal bought lime green pins for the family to wear for lymphoma awareness. Without telling Eric or Crystal, David decided he would wear his on his shirt for the 2018 football season. In addition to that, he had lime green and yellow ribbons placed on the back of Stanford helmets as a way to show support for both cancer patients and cancer survivors.

He told his team that his brother was fighting cancer, and briefly mentioned the helmet ribbons publicly during an early-season news conference. But beyond that, David kept the severity of what was happening to his brother to himself, masking his growing nervousness, fear and anxiety as the clock ticked toward the next transplant. He had a hard time processing what was happening. He did not want to put that at the feet of his players, or his staff.

The doctors used the same donor cells that failed the first time for the second transplant on Sept. 11, 2018, because that was the only option available. But this time, doctors used even stronger drugs to prepare Eric's body to receive the donor cells -- hoping that would do enough to stop his immune system from attacking them.

When Stanford played Oregon on Sept. 22, no one in the Shaw family knew whether the transplant had worked. But the situation was more dire than the first transplant. The stronger chemotherapy caused major complications, and Eric became severely ill.

David coached the game with this in the back of his mind. Stanford rallied from a 21-7 deficit to win an overtime thriller, moving to 4-0 on the season, with a top-10 matchup against Notre Dame the following week. Back in Palo Alto, Eric watched the entire game alone in an apartment he rented near the hospital, the comeback buoying his spirits.

He had no idea his brother would speak to him through the television until he heard the words, "To my brother Eric ..."

"In that moment, I didn't feel any sickness at all," Eric said. "I can't really describe what I felt, just how proud I am of him and how awesome it made me feel that he would do that for me."

Said David: "If that transplant didn't work, I didn't know how many more games he was going to be able to see. That was an opportunity for me on national TV to speak to him, to say to my brother that against the odds, we came back and throughout the entire game, I was thinking about him."

Eric soon returned to Stanford Hospital. The chemotherapy destroyed his blood system, so he needed daily blood transfusions to stay alive. It came as no surprise when doctors told him the second transplant had failed. They had no plan now, no other donor options. David came by to visit as often as he could, but he had a hard time finding the words to say to his dying brother.

"I thought about Crystal. I thought about their kids," Shaw said. "I thought about, 'How can we help?' And then I kept going, 'We just can't get there. There has to be something else.' And we all prayed and we all comforted each other and trusted the doctors and prayed for the doctors. And just kept saying, 'Just tell us whatever options there are. Just tell us what to do and we'll do it.'"

During the day, Eric had his mother, Crystal, David, or David's wife, Kori, at his side, helping to keep his mind off what was happening to him. But in the evenings, when he was alone in his hospital room, he couldn't help but think about the dwindling medical options and his own death, slowly accepting what he believed would inevitably come.

Over seven years, everything the doctors tried had failed, and the disease always came back more aggressively. He felt exhausted in every possible way, desperate to feel better. He didn't want to die. All he wanted to do was get better, and see his kids again, hug his wife and go home. But that possibility seemed as far off as the stars.

"The doctors couldn't help us," Eric said. "They had lost all hope. There was nothing left, but we were in the deepest part of the valley, and there was nobody there but God. I said, 'You're going to take me off this Earth.' And he told me, 'Eric, you're not going to die.' That was the point at which my faith really took over, and I really had true peace."

His team of doctors huddled together again and came up with a plan many of their colleagues questioned, simply because they had never attempted it. In mid-October of 2018, they told Eric they wanted to try a third transplant.

Only this time, they wanted David to be the donor and they had only weeks to make it happen.

Eric thought, "Are they trying to kill me?"

When David was initially rejected, doctors had worked for 25 years to find a way to do half-match transplants but had virtually no success. By 2018, doctors explained that a different way to do the transplant had emerged, opening up the potential to try it with Eric. These transplants, called haploidentical transplants, typically use donor cells from a family member.

Dr. Wen-Kai Weng, Eric's bone marrow transplant physician, explained, "It was relatively new at this time. We decided to go ahead, because we knew if we didn't do it, the disease would really come back with a vengeance."

No one had ever done a third transplant with donor cells at Stanford.

"If he didn't go for this risk, he wouldn't be here," said Dr. Youn Kim, who treated Eric and heads Stanford's multidisciplinary Cutaneous Lymphoma Clinic/Program. "He wouldn't be living."

Doctors told Shaw there was a 15% chance he would not survive the transplant itself. If he did survive it, there was only about a 30% to 40% chance the donor cells would work. Compared to much steeper survival odds with no transplant at all, the decision -- filled with multiple layers of danger -- did not feel risky at all.

They had to try.

"They might have told us what the odds were, and I honestly just pushed it out of my brain," David said. "If this is the Hail Mary, hey, we're going to drop back and throw it as far as we can and send prayers along with it and hope that it works."

Without hesitation, David said to his brother, "Tell me what I need to do."

Stanford gathered in its team hotel early on Oct. 27 to begin final preparations before hosting Washington State later that day. David checked in for a 9 a.m. meeting and when it finished, he checked out of the hotel without saying a word. He walked toward the back exit, careful to make sure no one saw him, and snuck out the door to a waiting car.

Shaw sat in the passenger seat, headed toward campus and Stanford Hospital, praying all the while that what he was about to do would work.

He arrived at the hospital and was hooked up to an IV for the first dose of medication. This would not be the more traditional bone marrow transplant, where cells are extracted with a needle through the hips. Rather, the medication flowing through the IV would stimulate his body to overproduce the stem cells needed for the transplant, flooding his blood with them. The cells would then be extracted from his blood, and transplanted into Eric.

Doctors told him to expect to start feeling joint pain and tiredness within 24 hours. Those symptoms would grow only stronger over the coming days, when he came in for more medication. They told him he should stay off his feet, rest and remain hydrated.

That would be nice, David thought. But he had a game to coach. Only two people inside the program knew he had gone that morning: assistant athletic director for football operations Callie Dale, who drove him to the hospital, and defensive coordinator Lance Anderson.

"The way that I do my job, I work really hard not to make it about me," David said. "Although I wanted my team to know what my family was going through, college football is about the student-athletes. I wanted them to focus on what they needed to do. I didn't want to pull from that. I didn't want to, all of a sudden, now make it about me and my family."

A few hours later, he returned to the team hotel and acted as if he had been there the entire day, speaking nothing about his trip to the hospital. Shaw put on his lime green pin and made his way toward the bus. The short ride to the stadium felt long that day. His mind wandered before returning to the flip card in front of him.

As he exited the bus and finished the walk to the stadium, his two young nieces ran up to him. They squeezed him, holding on longer than usual, as if they knew their Uncle David was their only option, too.

He worried players would notice him moving around so slowly. If they did, no one said a word. Shaw kept pushing the pain aside, shoving his emotions down deep, saying prayers every chance he got.

On Wednesday, Shaw woke up and was so lethargic, he felt as if he was moving like a sloth. He went to the hospital for the final procedure: extracting the cells from his blood. Shaw wore comfortable clothes, arranged his pillows and settled in for a long day ahead. Doctors hooked him up to a machine that would do the work through two IVs: One took his blood so the needed donor cells could be siphoned out; the other IV would put the blood back in his body.

Eric rested on another floor in the same hospital.

David worked on his game plan, watched a few movies and occasionally stared at his own blood in the IVs, willing it to save his brother. He kept saying to himself over and over again, "God, I hope this works."

After eight hours, he was finished. Shaw then went out to practice.

"I remember walking up to him and just asking him, 'How are you doing, how are you feeling?'" Anderson said. "I could see it in him that he wasn't his normal self. He paused for a little bit and then he's like, 'I'm OK. A little bit tired, but I'm OK.' You know, just trying to put the most positive light that he could on it."

The next day, Nov. 1, 2018, Shaw went back to the hospital. It was transplant day, and he had to be with Eric to witness what they hoped would be a miracle. David and Crystal watched as Eric received a transfusion of David's stem cells, a shimmering light pink fluid flowing into his body. They sang and prayed. Already, they had received one small bit of good news: Doctors extracted 28 million cells from David's blood, about 20 million more than what they had hoped to get.

Stanford traveled the following day to Seattle, for a game against Washington. David felt guilty for leaving, but he knew there was nothing else he could do. Eric struggled in the hospital, not only from the transplant, but from the heavy chemo and radiation doctors used to prepare his body for the new cells.

Eric ran a fever of 105 degrees and vomited for days. The pain grew so intense he was put on a morphine drip and was in and out of consciousness. In Seattle, Shaw remembers being locked into the game, "except for those little moments where my heart was with my brother."

Stanford lost another heartbreaker, 27-23.

"I know us losing had nothing to do with everything David was going through," Dale said. "But just piling that on with everything else he was dealing with, it was a lot for him. He brought that up many times, about how Eric would tell him the biggest excitement for him every week was watching us play and watching us win. I know David had a lot of pressure on himself, amongst the pressure he already has as a head coach, to win for Eric. And I know that every time he did, he really felt like it was for him. And when we came up short, I know he was probably even harder on himself than he normally would have been."

Back at Stanford, David visited Eric when he could. But the waiting game took an increasing mental toll. David prides himself on his ability to compartmentalize, to focus on the only thing in front of him. He never spaces out, and he rarely gets emotional. But Shaw was falling apart on the inside.

He often found himself staring at cut-ups of red zone plays, not realizing the film had been paused for 20 minutes while his mind drifted off. Whenever that happened, he would stop and call someone, either his brother, his wife, his mother or Crystal just to see how they were doing.

"There were times where I thought life was slow motion, but it was actually moving and I was the one who was in slow motion," David said. "I found myself sometimes saying, 'Is this real? Is this really happening? This shouldn't happen.'"

In the middle of every single meeting, in the middle of every single film session, he silently prayed, "God help my brother. Just please let this one work."

"I look back now and I know more of everything that was going on and the situation," Anderson said. "I realized how much he was dealing with and how much he had to bear that week. And it's amazing that he was able to go through that week without really letting any of us really know exactly what he was going through and what a big deal this really was."

Within a few weeks, Eric started to turn a corner. Though they did not know whether the transplant had worked just yet, he showed enough improvement to leave the hospital after 52 days. David arrived for the big day, and Eric slowly put on a protective mask before shuffling to a waiting wheelchair. Doctors, nurses and support staff lined the hallway, clapping and cheering.

David cries when recalling that moment, his pent-up emotions flooding out as he describes it publicly for the first time.

"This is my little brother, after years of cancer, getting to leave the hospital," Shaw said, his voice quavering. He pauses to wipe tears from his eyes. "The nurses were crying. The doctors were crying. Because a few months earlier, they were preparing us for him to die. And he got to go home."

Three days later, doctors met with Eric and Crystal to deliver the results from the transplant. After only 27 days, Eric had none of his own blood coursing through his body.

It was all David's.

Eric picked up the phone.

"Dave," Eric said. "You have a twin. We're truly blood brothers."

Eric, who turns 46 on Friday, has lived a fairly normal life since he was declared cancer free on Jan. 1, 2019, although the coronavirus pandemic has limited how often the Shaw family can see each other.

In September, they decided to get together to celebrate all of their recent birthdays at David's house. They stayed outdoors, socially distanced, with masks on. Eric and David allowed themselves a hug, their heads turned to the side.

"Every time I see him, I just smile, you know? Because he gets to be here," David said.

Link:
'We're truly blood brothers': Stanford coach David Shaw and his recent fight to save his brother, Eric - KGO-TV

Bone Marrow Stem Cells Comments Off on ‘We’re truly blood brothers’: Stanford coach David Shaw and his recent fight to save his brother, Eric – KGO-TV | November 5th, 2020

Taipei, Nov. 5 (CNA) An Indonesian migrant worker who received a stem cell transplant in Taiwan in June thanked the nation on Thursday for expediting her treatment by lifting travel restrictions for her family amid COVID-19 thereby facilitating the operation that saved her life.

At a press conference that day to celebrate being discharged from the hospital, 23-year-old Nina Herlina thanked Taiwan for giving her a new lease of life and said her treatment was a testament to Taiwan's healthcare capabilities. In November last year, Nina began suffering from bouts of menorrhagia that lasted for about 20 days and came with symptoms that included dizziness, tiredness, and fever.

In February, she turned to the Taiwan International Workers' Association (TIWA), a local NGO that promotes migrant workers' rights when she was fired, shortly after a doctor diagnosed her as suffering from aplastic anemia, an autoimmune disease in which the bone marrow stops making new blood cells. With the help of the TIWA, the young woman was allowed to remain in Taiwan, where she had worked as a caregiver since October 2018.

In March, she was confirmed as having severe aplastic anemia, requiring an allogeneic stem cell transplant to treat the disease, according to the TIWA. However, at that time the COVID-19 pandemic was worsening and Nina's family were in rural Indonesia and local medical institutions lacked the technology and techniques to identify a donor in time for a bone marrow transplant.

At that time she was being kept alive in Taiwan by weekly blood transfusions. However, frequent blood transfusions can have a detrimental effect on the success of a transplant.

In addition, she also had leukopenia, a condition when a person has a reduced number of white blood cells, which increases the risk of infection. As a result, doctors at Taipei Veterans General Hospital (TVGH) determined the patient was in urgent need of a transplant, according to TIWA.

With the assistance of TIWA, a TVGH medical team explained the condition to Herlina and her family members in Indonesia via video calls. Doctors said the healthy cells for the transplant should ideally come from a family member, making her two younger sisters, aged 5 and 14, the best candidates for the operation, TIWA said.

Based on humanitarian considerations, the Central Epidemic Command Center decided in June to lift travel restrictions for her mother and sisters to visit Taiwan.

After undergoing special blood tests arranged by TVGH, the 5-year-old sister was identified as a suitable donor for a transplant. The operation was carried out after the three family members completed their 21-day quarantine in Taiwan and provided two consecutive negative COVID-19 test results.

After having received medical treatment in Taiwan for nine months, Nina was discharged from the hospital Thursday, after doctors confirmed she had recovered from the life-threatening illness.

Read more here:
Critically ill Indonesian woman thanks Taiwan for saving life - Taiwan News

Bone Marrow Stem Cells Comments Off on Critically ill Indonesian woman thanks Taiwan for saving life – Taiwan News | November 5th, 2020

Several drugs that work by targeting genetic alterations in cancer cells have won recent approval from the Food and Drug Administration as treatments for patients with diffuse large B-cell lymphoma (DLBCL).

Dr. Germame Ajebo, assistant professor of medicine at Georgia Cancer Center at Augusta University, shared information on novel treatments for the disease during the recent virtual CURE Educated Patient Leukemia & Lymphoma Summit.

In his talk, Ajebo focused on drugs meant for use in disease that has recurred or become resistant to previous treatments.

DLBCL is a usually aggressive form of the blood cancer known as a B-cell non-Hodgkin lymphoma, which affects the immune system. The disease causes rapid growth of tumors in the lymph nodes, spleen, liver, bone marrow or other organs.

Approved by the FDA within the last two years to treat aggressive DLBCL are oral Xpovio (selinexor), Polivy (polatuzumab vedotin-piiq) and Monjuvi (tafasitamab-cxix). In addition, an immunotherapy, the chimeric antigen receptor (CAR)-T cell therapy Yescarta (axi-cel), was approved to treat the disease in 2017, Ajebo reported.

Read more: Monjuvi-Revlimid Combination Approval Fills Unmet Need for Certain Patients with DLBCL.

Xpovio is a nuclear export inhibitor, which prevents cancerous cells from pushing tumor-suppressing proteins out of their nuclei. This results in tumor suppressors accumulating in the nucleus, where they can work to kill the cell.

In the phase 2b clinical trial that led to its approval which administered Xpovio by itself to 134 previously treated older adult patients the partial response rate (including those with tumor shrinkage) was 16%, the complete response rate (including those with no sign of cancer remaining) was 13% and the rate of stable disease (including patients with no progression of cancer) was 8.2%, Ajebo reported. Looking at all patients who had partial or complete responses, 38% responded for at least six months and 15% for at least 12 months.

The most common side effects that were serious or worse were low blood counts, Ajebo summarized. Other serious side effects included nausea, vomiting, diarrhea, weight loss, dizziness and infections.

Polivy is an antibody-drug conjugate that uses a targeted drug to deliver a potent chemotherapy directly to cancer cells.

It was approved based on the results of a phase 2 study of 80 previously treated patients who were divided into equally sized groups to receive the chemotherapy Treanda (bendamustine) and the targeted drug Rituxan (rituximab) with or without Polivy every 21 days for six cycles. At the end of treatment, 40% of those receiving the triplet combination had experienced a complete response, compared with 18% of those receiving Treanda and Rituxan alone. In the 63% of patients who achieved a best overall response at any point in the study while receiving the drug triplet, 48% had a response that lasted at least 12 months and 64% responded for at least six months, Ajebo noted.

Major side effects, he said, included tingling or weakness in the extremities, low blood counts, liver toxicity and tumor lysis syndrome, a condition that can damage organs due to blood chemistry issues arising from the quick destruction of tumor cells. The most common side effects of any severity included low blood counts, fatigue, diarrhea and fever.

Monjuvi, a targeted drug that inhibits the activity of the DLBCL-fueling protein CD19, was approved based on results of the phase 2 L-MIND study that demonstrated a 43% complete response rate and an 18% partial response rate in 80 previously treated adult patients who were prescribed the drug along with the targeted medication Revlimid (lenalidomide), with a median duration of response of 21.7 months. The drug is approved in combination with Revlimid for patients with recurrent or resistant DLBCL who are not eligible for, or did not agree to undergo, bone marrow transplant using their own stem cells, Ajebo said.

Serious side effects occurred in 52% of patients, he said, and included low blood counts and infections. The drug caused fatal reactions in 5% of patients, including stroke, respiratory failure, progressive multifocal leukoencephalopathy (a virus that infects the brain) and sudden death.

For more news on cancer updates, research and education, dont forget tosubscribe to CUREs newsletters here.

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Novel Targeted Drugs are Changing the Treatment of Diffuse Large B-Cell Lymphoma - Curetoday.com

Bone Marrow Stem Cells Comments Off on Novel Targeted Drugs are Changing the Treatment of Diffuse Large B-Cell Lymphoma – Curetoday.com | November 5th, 2020

Of late, there has been an increasing awareness regarding the therapeutic potential of stem cells for management of diseases which is boosting the growth of the stem cell therapy market. The development of advanced genome based cell analysis techniques, identification of new stem cell lines, increasing investments in research and development as well as infrastructure development for the processing and banking of stem cell are encouraging the growth of the global stem cell therapy market.

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One of the key factors boosting the growth of this market is the limitations of traditional organ transplantation such as the risk of infection, rejection, and immunosuppression risk. Another drawback of conventional organ transplantation is that doctors have to depend on organ donors completely. All these issues can be eliminated, by the application of stem cell therapy. Another factor which is helping the growth in this market is the growing pipeline and development of drugs for emerging applications. Increased research studies aiming to widen the scope of stem cell will also fuel the growth of the market. Scientists are constantly engaged in trying to find out novel methods for creating human stem cells in response to the growing demand for stem cell production to be used for disease management.

It is estimated that the dermatology application will contribute significantly the growth of the global stem cell therapy market. This is because stem cell therapy can help decrease the after effects of general treatments for burns such as infections, scars, and adhesion. The increasing number of patients suffering from diabetes and growing cases of trauma surgery will fuel the adoption of stem cell therapy in the dermatology segment.

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

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Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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Stem Cell Therapy Market to Surge at a Robust Pace in Terms of Revenue Over2017 2025 - Royal Sutton News

Cardiac Stem Cells Comments Off on Stem Cell Therapy Market to Surge at a Robust Pace in Terms of Revenue Over2017 2025 – Royal Sutton News | November 5th, 2020

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Nov 5, 2020--

bluebird bio, Inc. (Nasdaq: BLUE) announced today that data from its gene and cell therapy programs for sickle cell disease (SCD), transfusion-dependent beta-thalassemia (TDT) and multiple myeloma (MM) will be presented, including seven oral presentations, at the 62 nd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place virtually from December 5-8, 2020.

Updated results from patients in Group C of the companys Phase 1/2 HGB-206 study of LentiGlobin for SCD gene therapy (bb1111) will be presented.

bluebird bio will also present updated long-term efficacy and safety results from the LTF-303 follow-up study; outcomes across genotypes; and outcomes in pediatric patients from Phase 3 studies HGB-207 and HGB-212 of betibeglogene autotemcel (beti-cel; formerly LentiGlobin for -thalassemia) in TDT.

Data from across the companys multiple myeloma program will be presented. Presentations will include updated safety and efficacy results from the Phase 1 CRB-401 clinical study of idecabtagene vicleucel (ide-cel, bb2121) and preliminary data from the ongoing Phase 1 CRB-402 clinical study of bb21217, as well as subgroup analyses of the pivotal Phase 2 KarMMa study of ide-cel. Ide-cel and bb21217 are investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immune therapies being studied, in partnership with Bristol-Myers Squibb, for the treatment of adult patients with MM.

Sickle Cell Disease Data at ASH

Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Presenting Author: Julie Kanter, MD, University of Alabama at Birmingham, Birmingham, AL

Date/Time: Oral #365, Sunday, December 6, 2020, 9:45 am PST

Resolution of Serious Vaso-occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Presenting Author: Alexis A. Thompson, MD, Hematology Section Head, Ann & Robert H. Lurie Childrens Hospital, Chicago, IL

Date/Time: Oral #677, Monday, December 7, 2020, 1:30 pm PST

The GRNDaD Registry: Contemporary Natural History data and an analysis of real-world patterns of use and limitations of Disease Modifying Therapy in adults with SCD

Presenting Author: Alexandra Boye-Doe, MD, University of North Carolina School of Medicine, Chapel Hill, NC

Date/Time: Poster #1730, Sunday, December 6, 2020, 7:00 am 3:30 pm PST

Transfusion-Dependent -Thalassemia Data at ASH

Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent -Thalassemia: Results in Patients with up to 6 Years of Follow-up

Presenting Author: Janet L. Kwiatkowski, MD, MSCE, Director, Thalassemia Center at Children's Hospital of Philadelphia, Philadelphia, PA

Date/Time: Oral #153, Saturday, December 5, 2020, 12:00 pm PST

Favorable Outcomes in Pediatric Patients in the Phase 3 HGB-207 (Northstar-2) and HGB-212 (Northstar-3) Studies of betibeglogene autotemcel Gene Therapy for the Treatment of Transfusion-dependent -thalassemia

Presenting Author: Alexis A. Thompson, MD, MPH, Hematology Section Head, Ann & Robert H. Lurie Childrens Hospital of Chicago, Chicago, IL

Date/Time: Oral #154, Saturday, December 5, 2020, 12:15 pm PST

Improvement in Erythropoiesis Following Treatment with Betibeglogene Autotemcel Gene Therapy in Patients with Transfusion-Dependent -Thalassemia in the Phase 3 HGB-207 Study

Presenting Author: John B. Porter, MA, MD, FRCP, FRCPath, Head of Red Cell Unit, University College London Hospital, London, UK

Date/Time: Poster #776, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Response of patients with transfusion-dependent -thalassemia (TDT) to betibeglogene autotemcel (beti-cel; LentiGlobin for -thalassemia) gene therapy based on HBB genotype and disease genetic modifiers

Presenting Author: Mark C. Walters MD, Medical Director, Jordan Family Center for BMT & Cellular Therapies Research, UCSF Benioff Childrens Hospital Oakland, Oakland, CA

Date/Time: Poster #1699, Sunday, December 6, 2020, 7:00 am 3:30 pm PST

Multiple Myeloma Data at ASH

Updated results from the Phase I CRB-402 study of anti-BCMA CAR-T cell therapy bb21217 in patients with relapsed and refractory myeloma: correlation of expansion and duration of response with T cell phenotypes

Presenting Author: Melissa Alsina, MD, Department of Blood and Marrow Transplantation and Cellular Immunotherapy, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL

Date/Time: Oral #130, Saturday, December 5, 2020, 9:45 am PST

Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-directed CAR T cell therapy, in patients with relapsed and refractory multiple myeloma: updated results from phase 1 CRB-401 study

Presenting Author: Yi Lin, MD, PhD, Division of Hematology, Mayo Clinic, Rochester, MN

Date/Time: Oral #131, Saturday, December 5, 2020, 10:00 am PST

Secondary Quality-of-Life Domains in Patients With Relapsed and Refractory Multiple Myeloma Treated With the BCMA-Directed CAR T Cell Therapy Idecabtagene Vicleucel (ide-cel; bb2121): Results from the KarMMa Clinical Trial

Author: Nina Shah, MD, University of California San Francisco, San Francisco, CA

Date/Time: Oral #437, Sunday, December 6, 2020, 12:15 pm PST

Efficacy and Safety of Idecabtagene Vicleucel (ide-cel, bb2121) in Elderly Patients with Relapsed/Refractory Multiple Myeloma: KarMMa Subgroup Analysis

Presenting Author: Jess Berdeja, MD, Sarah Cannon Research Institute and Tennessee Oncology, Nashville, TN

Date/Time: Poster #1367, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Characterization of Cytokine Release Syndrome in the KarMMa Study of Idecabtagene Vicleucel (ide-cel, bb2121) For Relapsed and Refractory Multiple Myeloma

Presenting Author: Ankit Kansagra, MD, Simmons Comprehensive Cancer Center, UT Southwestern Medical Center, Dallas, TX

Date/Time: Poster #1378, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Molecular and Phenotypic Profiling of Drug Product and Post-infusion Samples from CRB-402, an Ongoing: Phase I Clinical Study of bb21217 a BCMA-directed CAR T Cell Therapy

Presenting Author: Olivia Finney, PhD, Associate Director, Immunotherapy, bluebird bio

Date/Time: Poster #1401, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Effects of Prior Alkylating Therapies on Preinfusion Patient Characteristics and Starting Material for CAR T Cell Product Manufacturing in Late-Line Multiple Myeloma

Presenting Author: Julie Rytlewski, PhD, Bristol Myers Squibb, Princeton, NJ

Date/Time: Poster #1405, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

KarMMa-4: Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-Targeted CAR T Cell Therapy, in High-Risk Newly Diagnosed Multiple Myeloma

Presenting Author: Saad Z. Usmani, MD, Director, Clinical Research in Hematologic Malignancies, Levine Cancer Institute/Atrium Health, Charlotte, NC

Date/Time: Poster #1418, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Healthcare Resource Utilization and Cost of Cytokine Release Syndrome and Neurologic Events in Patients with Relapsed and Refractory Multiple Myeloma Receiving the BCMA-directed CAR T Cell Therapy Idecabtagene Vicleucel (ide-cel, bb2121) in the KarMMa Trial

Presenting Author: Parmeswaran Hari, MD, Medical College of Wisconsin, Milwaukee, WI

Date/Time: Poster #1598, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

A Matching-Adjusted Indirect Comparison of Efficacy Outcomes for Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-directed CAR T Cell Therapy Versus Conventional Care in Triple-Class Exposed Relapsed and Refractory Multiple Myeloma

Presenting Author: Nina Shah, MD, University of California San Francisco, San Francisco, CA

Date/Time: Poster #1653, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Idecabtagene Vicleucel (ide-cel, bb2121) Responses Are Characterized by Early and Temporally Consistent Activation and Expansion of CAR T Cells With a T Effector Phenotype

Presenting Author: Nathan Martin, PhD, Bristol Myers Squibb, Princeton, NJ

Date/Time: Poster #2315, Sunday, December 6, 2020, 7:00 am 3:30 pm PST

KarMMa-3: A Phase 3 Study of Idecabtagene Vicleucel (ide-cel,bb2121), a BCMA-Targeted CAR T Cell Therapy Versus Standard Regimens in Relapsed and Refractory Multiple Myeloma

Presenting Author: Michel Delforge, MD, PhD, University Hospital Leuven, Leuven, Belgium

Date/Time: Poster #2323, Sunday, December 6, 2020, 7:00 am 3:30 pm PST

Idecabtagene Vicleucel (ide-cel, bb2121) in Relapsed and Refractory Multiple Myeloma: Analyses of High-Risk Subgroups in the KarMMa Study

Presenting Author: Noopur S. Raje, MD, Massachusetts General Hospital, Boston, MA

Date/Time: Poster #3234, Monday, December 7, 2020, 7:00 am 3:00 pm PST

Health State Utility Valuation in Patients with Triple-Class Exposed Relapsed and Refractory Multiple Myeloma Treated with the BCMAdirected CAR T Cell Therapy, Idecabtagene Vicleucel (idecel, bb2121): Results from the KarMMa Trial

Presenting Author: Michel Delforge, MD, PhD, University Hospital Leuven, Leuven, Belgium

Date/Time: Poster #3465, Monday, December 7, 2020, 7:00 am 3:00pm PST

Abstracts outlining bluebird bios accepted data at ASH are available on the ASH conference website.

About LentiGlobin for SCD (bb1111)

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the -globin gene that leads to the production of abnormal sickle hemoglobin (HbS), causing red blood cells (RBCs) to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive events (VOEs). For adults and children living with SCD, this means unpredictable episodes of excruciating pain due to vaso-occlusion as well as other acute complicationssuch as acute chest syndrome (ACS), stroke, and infections, which can contribute to early mortality in these patients.

LentiGlobin for SCD (bb1111) is an investigational gene therapy being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study and the ongoing Phase 3 HGB-210 study.

LentiGlobin for SCD was designed to add functional copies of a modified form of the -globin gene ( A-T87Q -globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once patients have the A-T87Q -globin gene, their red blood cells can produce anti-sickling hemoglobin (Hb A-T87Q ) that decreases the proportion of HbS, with the goal of reducing sickled red blood cells, hemolysis and other complications.

As of March 3, 2020, a total of 37 patients have been treated with LentiGlobin for SCD to-date in the HGB-205 (n=3) and HGB-206 (n=34) clinical studies. The HGB-206 total includes: Group A (n=7), B (n=2) and C (n=25).

LentiGlobin for SCD received orphan medicinal product designation from the European Commission for the treatment of SCD, and Priority Medicines (PRIME) eligibility by the European Medicines Agency (EMA) in September 2020.

The U.S. Food and Drug Administration (FDA) granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation and rare pediatric disease designation for LentiGlobin for SCD. LentiGlobin for SCD continues to be evaluated in the ongoing Phase 1/2 HGB-206 and Phase 3 HGB-210 studies.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

LentiGlobin for SCD is investigational and has not been approved in any geography.

About betibeglogene autotemcel

Transfusion dependent beta-thalassemia (TDT) is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT require chronic blood transfusions to maintain adequate Hb levels. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

Betibeglogene autotemcel (beti-cel) adds functional copies of a modified form of the -globin gene ( A-T87Q -globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q -globin gene, they have the potential to produce HbA -T87Q, which is gene therapy-derived adult hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

The European Commission granted conditional marketing authorization (CMA) for beti-cel, marketed as ZYNTEGLO gene therapy, for patients 12 years and older with transfusion-dependent -thalassemia (TDT) who do not have a 0 / 0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

As of March 3, 2020, a total of 60 pediatric, adolescent and adult patients, including 11 patients with at least 5 years of follow-up, across genotypes of TDT have been treated with beti-cel in the Phase 1/2 Northstar (HGB-204) and HGB-205 studies, and the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies. In studies of beti-cel, patients were assessed for transfusion independence, defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average Hb of at least 9 g/dL.

Non-serious adverse events (AEs) observed during clinical studies that were attributed to beti-cel included abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnoea, pain in extremity, tachycardia and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to beti-cel.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease. On April 28, 2020, the European Medicines Agency (EMA) renewed the CMA for beti-cel. The CMA for beti-cel is valid in the 27 member states of the EU as well as UK, Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. FDA granted beti-cel orphan drug designation and Breakthrough Therapy designation for the treatment of TDT. Beti-cel is not approved in the United States. Beti-cel continues to be evaluated in the ongoing Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of beti-cel.

About idecabtagene vicleucel (ide-cel, bb2121)

Ide-cel is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T cell immunotherapy. The ide-cel CAR is comprised of a murine extracellular single-chain variable fragment (scFv) specific for recognizing BCMA, attached to a human CD8 hinge and transmembrane domain fused to the T cell cytoplasmic signaling domains of CD137 4-1BB and CD3- chain, in tandem. Ide-cel recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.

Ide-cel is being developed as part of a Co-Development, Co-Promotion and Profit Share Agreement between Bristol Myers Squibb and bluebird bio. Ide-cel was granted accelerated assessment by the European Medicines Agency (EMA) on March 26, 2020, and the Marketing Authorization Application (MAA) was validated by the EMA on May 20, 2020. The FDA accepted the ide-cel Biologics License Application (BLA) for priority review on September 22, 2020.

KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multicenter, multinational, Phase 2 study evaluating the efficacy and safety of ide-cel in adults with RRMM in North America and Europe. The primary endpoint of the study is overall response rate as assessed by an independent review committee (IRC) according to the International Myeloma Working Group (IMWG) criteria. Complete response rate is a key secondary endpoint. Other secondary endpoints include time to response, duration of response, progression-free survival, overall survival, minimal residual disease evaluated by Next-Generation Sequencing (NGS) assay and safety. The study enrolled 140 patients, of whom 128 received ide-cel across the target dose levels of 150-450 x 10 6 CAR+ T cells after receiving lymphodepleting chemotherapy. All enrolled patients had received at least three prior treatment regimens, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and were refractory to their last regimen, defined as progression during or within 60 days of their last therapy.

CRB-401 (NCT02658929) is an open-label Phase 1 study evaluating the preliminary safety and efficacy of ide-cel in patients with relapsed and refractory multiple myeloma (RRMM). The primary endpoint of the study is safety. CRB-401 was designed as a two-part (dose escalation and dose expansion) study to determine the maximum tolerated dose and further evaluate the safety, tolerability and clinical activity at the recommended Phase 2 dose; these findings established the recommended dose of the Phase 2 KarMMa trial. All patients have been treated in the study and follow-up is ongoing.

In addition to the pivotal KarMMa and CRB-401 trials, bluebird bio and Bristol Myers Squibbs broad clinical development program for ide-cel includes clinical studies (KarMMa-2, KarMMa-3, KarMMa-4) exploring ide-cel combinations and activity in earlier lines of treatment for patients with multiple myeloma, including newly diagnosed multiple myeloma. For more information visit clinicaltrials.gov.

Ide-cel is not approved for any indication in any geography.

About bb21217

bb21217 is an investigational BCMA-targeted CAR T cell therapy that uses the ide-cel CAR molecule and is cultured with the PI3 kinase inhibitor (bb007) to enrich for T cells displaying a memory-like phenotype with the intention to increase the in vivo persistence of CAR T cells. bb21217 is being studied for patients with multiple myeloma in partnership with Bristol Myers Squibb.

bluebird bios clinical development program for bb21217 includes the ongoing Phase 1 CRB-402 study. CRB-402 is the first-in-human study of bb21217 in patients with relapsed and refractory multiple myeloma (RRMM), designed to assess safety, pharmacokinetics, efficacy and duration of effect. CRB-402 is a two-part (dose escalation and dose expansion), open-label, multi-site Phase 1 study of bb21217 in adults with RRMM. For more information visit: clinicaltrials.gov using identifier NCT03274219.

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bluebird bio to Present Data from Gene and Cell Therapy Programs During the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition -...

Cardiac Stem Cells Comments Off on bluebird bio to Present Data from Gene and Cell Therapy Programs During the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition -… | November 5th, 2020

This article was originally published here

Stem Cell Res. 2020 Oct 15;49:102030. doi: 10.1016/j.scr.2020.102030. Online ahead of print.

ABSTRACT

Multiple myeloma (MM) is a hematological cancer characterized by an uncontrolled proliferation of antibody-secreting plasma cells within the bone marrow. Currently, cell therapy such as chimeric antigen receptor T-cell (CAR-T) based on induced pluripotent stem cells (iPSCs) has received attention for treating MM. However, the generation of iPSCs from MM patients appears to be very rarely reported. Here we generated an iPSC line from CD34+ bone marrow cells of a patient with MM using human placenta-derived cell conditioned medium (hPCCM), offering a relatively high efficiency in humanized conditions. This iPSC line might be a useful model for research on MM.

PMID:33142253 | DOI:10.1016/j.scr.2020.102030

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Generation of normal induced pluripotent stem cell line KUMCi002-A from bone marrow CD34+ cells of patient with multiple myeloma disease having 13q...

Bone Marrow Stem Cells Comments Off on Generation of normal induced pluripotent stem cell line KUMCi002-A from bone marrow CD34+ cells of patient with multiple myeloma disease having 13q… | November 5th, 2020

NEW YORK, Nov. 4, 2020 /PRNewswire/ --Actinium Pharmaceuticals, Inc.,(NYSE AMERICAN: ATNM) ("Actinium") today announced that two abstracts on the Company's Antibody Radiation Conjugate (ARC) Iomab-B were accepted for oral presentations at the 2020 American Society of Hematology (ASH) annual meeting that is being held virtually December 5-8, 2020.

"This is an exciting fourth quarter for the company and we are honored to have multiple oral presentations at this year's ASH conference. Our 3 oral presentations and one poster presentation demonstrate the clinical progress we have seen not only with Iomab-B, but our other programs including Actimab-A in combination with novel and approved therapeutic agents," stated Sandesh Seth, Actinium's Chairman and CEO. "We look forward to presenting the Iomab-B data in further detail during the two oral presentations on the Iomab-B SIERRA study at ASH in December. The company remains on track to report safety and feasibility data from 75% of the patients to be enrolled in SIERRA, as well as to complete the ad hoc interim analysis in the fourth quarter."

Mark Berger, Actinium's Chief Medical officer, said, "We are encouraged that we continue to see positive ongoing results from our Phase 3 pivotal SIERRA trial in relapsed or refractory Acute Myeloid Leukemia (R/R AML) patients. In the Iomab-B Phase 3 trial we continue to see 100% engraftment in patients receiving a therapeutic dose of Iomab-B in the treatment arm whereas 83% of control arm patients failed salvage therapy, which includes the recently approved targeting agents such as venetoclax. This high failure rate demonstrates the significant need that exists in R/R AML and represents the paradigm shift we are looking to initiate with Iomab-B. The strong safety and feasibility data we have seen thus far gives us confidence that these older patients with active AML may benefit by undergoing a potentially curative bone marrow transplant which they could not receive otherwise."

Details & Highlights for Oral Presentations

Note: The two abstractsincludeSIERRA Phase 3 trial data available to the company from its CRO prior to August 10, 2020, the ASH submission cutoff date. Per ASH rules, updated data sets are permitted to be included in the live presentations.

OralPresentationTitle:

Personalized Targeted Radioimmunotherapy with Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] in Patients with Active Relapsed or Refractory Acute Myeloid Leukemia Results in Successful Donor Hematopoietic Cells Engraftment with the Timing of Engraftment Not Related to the Radiation Dose Delivered

Publication Number:

193

Session Name:

721. Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities

Session Date:

Saturday, December 5, 2020

Presentation Time:

1:00 PM PT / 4:00 PM ET

Phase 3 SIERRA Preliminary Results

Baseline Characteristics

Randomized to Iomab-B(N=53)

Randomized to Conventional Care (CC)(N=53)

Age (yrs, median, range)

64 (55-77)

65 (55-77)

Molecular & Cytogenetic Risk

Favorable: 2%Intermediate: 33%

Adverse: 65%

Favorable: 4%

Intermediate: 30%

Adverse: 66%

% TransplantedIntent-to-Treat Group

87% (46/53)

17% (9/53)

59% (26/44)

Results

Received Therapeutic dose of Iomab-B & Transplanted (N=46)****

Eligible to Receive Std. of Care Transplant Post-Salvage (N=9)

Evaluated for Crossover (N=44)*****

Cross-over Rate

n/a

n/a

Received Therapeutic Dose of Iomab-B (N=26)

Transplanted (N=26)

59% (26/44)

% Transplanted

100% (46/46)

17% (9/53)

100% (26/26)

BM Blast % @ baseline (median, range)

26 (4-95)

14 (5-97)

30 (6-87)

BM Blast % pre-HCT (median, range)

26 (4-95)

1 (0-3)*

32.5 (2-75)

Days to ANC Engraftment

14 (9-22)***

17 (13-83)#

14 (10-37)**

Days to Platelet Engraftment

17 (4-39)***

22 (8-35)#

19 (1-38)**

Days to HCT (Post Randomization)

30 (23-60)

66 (51-86)

65 (36-161)^

Myeloablative Dose Delivered to Bone Marrow

14.7 (4.6-32) Gv

n/a

14.4 (6.3-30) Gv

540 (313-1008) mCi

632 (354-1027) mCi

Chimerism>/=95% by D100

91% (39/43^ Evaluable)

67% (4/6^^ Evaluable)

87% (20/23^^^ Evaluable)

100-day non-Relapse Transplant-Related Mortality

5% (2/40 Evaluable)

25% (2/8 Evaluable)

8% (2/24 Evaluable)

*1 pt with 8% BM blasts on D42 with CRp on D50, ** ANC engraftment data not available (N=3), platelet engraftment data not available (N=4); *** ANC engraftment data not available (N=4), platelet engraftment data not available (N=9), ^ 1 patient at 161 days had delayed transplant due to infection and respiratory failure, received Iomab & transplant when stable, # ANC and platelet engraftment data not available (N=2)

**** No Therapy Dose (7) due to: Declining KPS (4), Infusion Reaction (1), Unfavorable Biodistribution (1), Post-Randomization Eligibility (1); 1 Pending Treatment.

*****Ineligible for Iomab-B HCT after crossover evaluation - 13: due to Hospice Care/Progression (4), Declined/Ineligible for HCT (5), Died Pre-Crossover (4). 4 Received Dosimetry but No Therapy Dose due to Declining KPS; 2 Pending Evaluation for Crossover.

^ Did not achieve 95% chimerism (4); Data pending (2); Died (1); ^^ Did not achieve 95% chimerism (4); Data pending (1); ^^^Did not achieve 95% chimerism (4); Data pending (2);

Oral PresentationTitle:

High Doses of Targeted Radiation with Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] Do Not Correlate with Incidence of Mucositis, Febrile Neutropenia or Sepsis in the Prospective, Randomized Phase 3 Sierra Trial for Patients with Relapsed or Refractory Acute Myeloid Leukemia

Publication Number:

135

Session Name:

721. Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities

Session Date:

Saturday, December 5, 2020

Presentation Time:

9:30 AM PT / 12:30 PM ET

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Actinium Announces Two Oral Presentations Featuring Data and Findings from the Phase 3 SIERRA Trial of Iomab-B at the 62nd American Society of...

Bone Marrow Stem Cells Comments Off on Actinium Announces Two Oral Presentations Featuring Data and Findings from the Phase 3 SIERRA Trial of Iomab-B at the 62nd American Society of… | November 5th, 2020

Development of limited, chronic graft-vs-host disease (GVHD) following allogeneic hematopoietic stem cell transplantation (HSCT) was associated with a survival benefit for patients with high-risk myelodysplastic syndrome (MDS), according to results of a retrospective study published in Clinical Cancer Research.1

MDS is a heterogeneous group of clonal hematopoietic stem cell disorders characterized by an inadequate production of normal, mature blood cells in bone marrow.

At the present time, the only curative treatment for patients with MDS is allogeneic HSCT. Although risks associated with this approach include posttransplant relapse, as well as the development of acute and/or chronic GVHD, previous findings from studies of patients with acute leukemia treated with allogeneic HSCT have shown potent graft-vs-tumor effects manifesting as lower mortality in those patients who subsequently experienced chronic GVHD.

The clinical data used in this study were collected from more than 300 HSCT centers throughout Japan by the Japanese Data Center for Hematopoietic Cell Transplantation.

MDS was characterized as low- or high-risk disease according to the

French-American-British or World Health Organization classification schemes, depending on the year of diagnosis.2,3 Consensus criteria were used to assign GVHD severity and distinguish acute and chronic forms of the condition.

Study inclusion criteria dictated patients should be aged 16 through 70 years, had received first allogeneic HSCT between 2001 and 2017, had achieved neutrophil engraftment, and had a follow-up period of over 60 days.

Of the 3119 patients included in this study, 1193 and 1926 were classified as having low- and high-risk disease, respectively. In the overall group, the median patient age was 54 years, more than 90% of patients had an Eastern Cooperative Oncology Group (ECOG) performance status score of 0 or 1, and 85% were diagnosed with de novo disease.

At a median follow-up of 55 months, rates of 5-year overall survival (OS) were 63% and 48% for those with low- and high-risk disease, respectively (P <.001). The cumulative incidence of posttransplantation relapse at 5 years was approximately twice as high for those with high-risk disease (29%) compared with those with low-risk disease (15%; P <.001), although the cumulative incidence of nonrelapse mortality was similar in the 2 groups: 25% (low-risk disease) and 27% (high-risk disease; P =.338).

Multivariate analyses accounting for all confounding variables performed to evaluate these transplant outcomes in patient subgroups defined according to disease risk, as well the severity of GVHD and whether it was classified as acute or chronic, revealed the following:

In summarizing the results of this study, the study authors emphasized that these data demonstrated a survival benet of the graft-versus-MDS effect is present only in high-risk MDS patients with limited chronic GVHD.1

Authors of an accompanying editorial noted that in recent years, the mutational spectrum in MDS has become more associated with clinical phenotype and prognosis, and selection of a patient with MDS for HSCT has begun to incorporate the mutational landscape of the patients disease. Thus, one will have to be cautious in extrapolating the current data to ongoing and future trials, which have begun to incorporate molecular information.4

References

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Survival Benefit of GVHD in MDS Is Limited to Specific Patient Subgroup - Cancer Therapy Advisor

Bone Marrow Stem Cells Comments Off on Survival Benefit of GVHD in MDS Is Limited to Specific Patient Subgroup – Cancer Therapy Advisor | November 5th, 2020

Stem Cell Therapy Market Size And Forecast

A comprehensive overview of the Stem Cell Therapy Market is recently added by Verified Market Research to its humongous database. Furthermore, the Stem Cell Therapy Market report has been aggregated by collecting informative data of various dynamics such as market drivers, restraints, and opportunities. Furthermore, this innovative report makes use of SWOT, PESTLE, and Porters Five Forces analyses to get a closer outlook on the Stem Cell Therapy Market. Furthermore, the Stem Cell Therapy Market report offers a detailed analysis of the latest industry developments and trending factors in the market that are influencing the market growth. Furthermore, this statistical market research repository examines and estimates the Stem Cell Therapy Market at the global and regional levels. The study covers the impact of various drivers and manacles on the Stem Cell Therapy Market growth opportunities over the forecast period.

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The degree of competition among leading global companies has been elaborated by examining various leading key players operating across the global regions An expert team of research analysts sheds light on various attributes such as -global market competition, market share, latest industry developments, innovative product launches, partnerships, mergers or acquisitions by leading companies in the Stem Cell Therapy Market. The leading manufacturers have been analyzed by using research methodologies for getting insight views on global competition.

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1.Stem Cell Therapy Market, By Cell Source:

Adipose Tissue-Derived Mesenchymal Stem Cells Bone Marrow-Derived Mesenchymal Stem Cells Cord Blood/Embryonic Stem Cells Other Cell Sources

2.Stem Cell Therapy Market, By Therapeutic Application:

Musculoskeletal Disorders Wounds and Injuries Cardiovascular Diseases Surgeries Gastrointestinal Diseases Other Applications

3.Stem Cell Therapy Market, By Type:

Allogeneic Stem Cell Therapy Market, By Application Musculoskeletal Disorders Wounds and Injuries Surgeries Acute Graft-Versus-Host Disease (AGVHD) Other Applications Autologous Stem Cell Therapy Market, By Application Cardiovascular Diseases Wounds and Injuries Gastrointestinal Diseases Other Applications

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North America Latin America Middle East Asia-Pacific Africa Europe

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Verified Market Research is a leading Global Research and Consulting firm servicing over 5000+ customers. Verified Market Research provides advanced analytical research solutions while offering information enriched research studies. We offer insight into strategic and growth analyses, Data necessary to achieve corporate goals, and critical revenue decisions.

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Stem Cell Therapy Market Size, Key Development Trends, and Growth Projection to 2027 - Stock Market Vista

Bone Marrow Stem Cells Comments Off on Stem Cell Therapy Market Size, Key Development Trends, and Growth Projection to 2027 – Stock Market Vista | November 5th, 2020

NEW YORK, Nov. 4, 2020 /PRNewswire/ -- Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, announced that an abstract demonstrating clinical benefit of the company's lead product candidate ARU-1801 has been published online and will be the subject of an oral presentation at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition. The meeting will take place virtually from December 5 to 8. Michael S. Grimley, M.D., Professor of Clinical Pediatrics, Medical Director of the Division of Bone Marrow Transplantation and Immune Deficiency at the Cincinnati Children's Hospital Medical Center, will present the data at 2:15 PM ET on December 7, 2020. Today the abstract was published online and will be published in the upcoming supplemental issue of Blood.

Data presented at ASH is from the MOMENTUM study, an open label Phase 1/2 clinical trial examining ARU-1801 as a one-time potentially curative gene therapy for individuals with sickle cell disease (SCD). The MOMENTUM study, which continues to enroll patients, examines ARU-1801, an autologous cell therapy leveraging a modified gamma globin lentivirus vector, in individuals with severe SCD. Unlike investigational gene therapies that require fully myeloablative conditioning, ARU-1801 is given with reduced intensity conditioning (RIC), which is a lower dose chemotherapy. ARU-1801 is designed to address the limitations of current curative treatment options, such as low donor availability and the risk of Graft-versus-Host Disease (GvHD) seen with allogeneic stem cell transplants. The data to be presented at ASH highlights participants dosed with product manufactured with both the original academic manufacturing process and the enhanced Process II.

"The clinical results thus far demonstrate that ARU-1801 holds significant promise for achieving durable responses with a reduced intensity conditioning approach to gene therapy," said Dr. Grimley, a principle investigator in the MOMENTUM study. "Given the impact chemotherapy toxicity has on physician and patient decision making around treatment options, ARU-1801 has the potential to be a unique option for SCD patients seeking gene therapy."

Abstract and Oral Presentation Information

Title: Early Results from a Phase 1/2 Study of ARU-1801 Gene Therapy for Sickle Cell Disease (SCD): Manufacturing Process Enhancements Improve Efficacy of a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning TransplantPublication Number: 680Session Name: 114. Hemoglobinopathies, Excluding ThalassemiaClinical: Novel Treatments for Sickle Cell DiseaseDate:Monday, December 7, 2020Session Time:1:30 PM - 3:00 PMPresentation Time:2:15 PM

About ARU-1801ARU-1801 is a one-time potentially curativeinvestigational gene therapy for individuals living with sickle cell disease. This product candidate was designed to address the limitations of current treatment options including chemotherapy toxicity, donor availability, andchronic administration and replace it with a curative therapy. ARU-1801 incorporates a patented modified gamma-globin into autologous stem cells, with the aim of restoring normal red blood cell function through increased levels of fetal hemoglobin. The high potency of the modified gamma globin enables ARU-1801 engraftment with only reduced intensity conditioning (RIC). Preliminary clinical data from the MOMENTUMstudy, an ongoing Phase 1/2 trial in patients with sickle cell disease, demonstrate continuing durable reductions in disease burden.

The MOMENTUM StudyAruvant is currently conducting the MOMENTUM study, which is evaluating ARU-1801, a one-time only potentially curative gene therapy for patients with SCD. This Phase 1/2 study currently is enrolling individuals with SCD, and information may be found at http://www.momentumtrials.com.

About Aruvant SciencesAruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talentedteamwith extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an activeresearchprogram with a lead product candidate, ARU-1801, in development for individuals suffering fromsickle cell disease(SCD). ARU-1801 is an investigational lentiviral gene therapy currently in aclinical trial,the MOMENTUM study, as a potential one-time curative treatment for SCD. Preliminary clinical data from the ongoing Phase 1/2 study demonstrated engraftment of ARU-1801 and amelioration of SCD is possible with one dose of low intensity chemotherapy. For more information on the clinical study, please visit http://www.momentumtrials.com and for more on the company, please visitwww.aruvant.com.

SOURCE Aruvant Sciences

Index

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Aruvant Announces Updated Data to be Presented in Oral Presentation of ARU-1801 Data at the 62nd American Society of Hematology (ASH) Annual Meeting -...

Bone Marrow Stem Cells Comments Off on Aruvant Announces Updated Data to be Presented in Oral Presentation of ARU-1801 Data at the 62nd American Society of Hematology (ASH) Annual Meeting -… | November 5th, 2020

The ability to grow and reproduce is a fundamental property of living organisms. Whether an organism is composed of a single cell or trillions of cells, individual cells must be able to grow and divide in an appropriately regulated fashion.

Cell growth is accomplished through the synthesis of new molecules of proteins, nucleic acids, carbohydrates and lipids. As the accumulation of these molecules causes the volume of a cell to increase, the plasma membrane grows to prevent the cell from burst-ing.

But cells cannot continue to enlarge indefinitely; as a cell grows larger, there is an accompanying decrease in its surface area/volume ratio and hence in its capacity for ef-fective exchange with the environment.

Therefore, cell growth is generally accompanied by cell division, whereby one cell gives rise to two new daughter cells (The term daughter is used by convention and does not indicate that cells have gender).

For single-celled organisms, cell division increases the total number of individuals in a population. In multicellular organisms, cell division either increases the number of cells, leading to growth of the or-ganism, or replaces cells that have died. In an adult human, for example, about two million stem cells in bone marrow divide every second to maintain a constant num-ber of red blood cells in the body. When cells grow and divide, the newly formed daugh-ter cells are usually genetic duplicates of the parent cell, containing the same (or virtually the same) DNA sequences.

Therefore, all the genetic information in the nucleus of the parent cell must be duplicated and carefully distributed to the daughter cells during the division process.

In accomplishing this task, a cell passes through a series of discrete stages, collectively known as the cell cycle. The cell cycle begins when two new cells are formed by the division of a single parental cell and ends when one of those cells divides again into two cells.

To early cell biologists studying eukaryotic cells with the mi-croscope, the most dramatic events in the life of a cell were those associated with the point in the cycle when the cell actually divides. This division process, called the M phase, involves two overlapping events in which the nucleus divides first and the cytoplasm second. Nuclear division is called mitosis, and the division of the cytoplasm to pro-duce two daughter cells is termed cytokinesis.

The stars of the mitotic drama are the chromosomes.

The beginning of mitosis is marked by condensation (coiling and folding) of the cells chromatin, which generates chromosomes that are thick enough to be individually discernible under the micro-scope.

Because DNA replication has already taken place, each chromosome actually consists of two chromosome copies that remain attached to each other until the cell divides. As long as they remain attached, the two new chromosomes are referred to as sister chromatids.

As the chromatids become visible, the nuclear envelope breaks into fragments. Then, in a stately ballet guided by the mi-crotubules of the mitotic spindle, the sister chromatids separate and each now a full-fledged chromosome move to opposite ends of the cell.

By this time, cytokinesis has usually begun, and new nuclear membranes envelop the two groups of daughter chromosomes as cell division is completed. While visually striking, the events of the mitotic phase account for a relatively small portion of the total cell cycle; for a typical mammalian cell, the mitotic phase usually lasts less than an hour.

Cells spend the majority of their time in the growth phase between divisions, called interphase. Most cellular contents are synthesised continuously during interphase, so cell mass gradually increases as the cell approaches division.

During interphase the amount of nuclear DNA doubles, and experiments using radioactive DNA precursors have shown that the new DNA is synthesised during a particular portion of interphase named the S phase (S for synthesis). A time gap called G1 phase separates the S phase from the preceding M phase, and a second gap, the G2 phase, separates the end of S phase from the beginning of the next M phase.

Although the cells of a multicellular organism divide at varying rates, most studies of the cell cycle involve cells growing in culture, where the length of the cycle tends to be similar for different cell types. We can easily determine the overall length of the cell cycle the generation time for cultured cells by counting the cells under a microscope and determining how long it takes for the population to double.

In cultured mammalian cells, for example, the total cycle usually takes about 18-24 hours. Once we know the total length of the cycle, it is possible to determine the length of specific phases. To determine the length of the S phase, we can expose cells to a radioactively labelled DNA precursor for a short period of time and then examine the cells by autoradiography.

The fraction of cells with silver grains over their nuclei represents the fraction of cells that were somewhere in S phase when the radioactive compound was available. When we mul-tiply this fraction by the total length of the cell cycle, the result is an estimate of the average length of the S phase.

For mammalian cells in culture, this fraction is often around 0.33, which indicates that S phase is about six to eight hours in length. Similarly, we can estimate the length of the M phase by multiplying the generation time by the percentage of the cells that are actually in mitosis at any given time.

This percentage is called the mitotic index.

The mitotic index for cultured mammalian cells is often about three to five per cent, which means that M phase lasts less than an hour (usually 30-45 minutes). In contrast to the S and M phases, whose lengths tend to be quite similar for different mammalian cells, the length of G1 is quite variable, depending on the cell type. Although a typical G1 phase lasts 8-10 hours, some cells spend only a few minutes or hours in Gl, whereas others are delayed for long periods of time. During Gl, a major decision is made as to whether and when the cell is to divide again. Cells that become arrested in Gl, awaiting for a signal that will trigger re-entry into the cell cycle and a commitment to divide, are said to be in G0 (G zero).

Other cells exit from the cell cycle entirely and undergo terminal differentiation, which means that they are destined never to divide again; most of the nerve cells in our body are in this state. In some cells, a transient arrest of the cell cycle can also occur in G2. In general, however, G2 is shorter than Gl and is more uniform in duration among different cell types, usually lasting 4-6 hours.

The writer is associate professor and head, department of botany, Ananda Mohan College, Kolkata.

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Putting it all together - The Statesman

Bone Marrow Stem Cells Comments Off on Putting it all together – The Statesman | November 5th, 2020