Stem Cell Clinic

Cord blood banks sell parents on promising stem cell research, but with no guarantees – The Arizona Republic

Stem cell treatment promise sells pregnant parents on cord blood banks Arizona Republic

Moments after Linda Buzans son Luca is born, her OB-GYN goes to work. She carefully cuts the white umbilical cord, then clamps it shut before any blood can escape. Once the cord is cleaned, she carefully inserts a needle with a long tube attached and lets the umbilical cord blood pump into a collection bag.

From there, the sample will travel in a labeled collection box to Tucson, where a laboratory for one of the oldest and biggest private cord blood banking companies nationwide is located. There, at the Cord Blood Registry laboratory, or CBR,baby Lucas umbilical cord blood will be frozen in a metal tank at less than minus 320 degrees, joining almost 900,000 other samples in storage,including that of his older sister, Lola.

Umbilical cord blood is full of stem cells, so it can be transplanted into patients to treat certain types of cancers, diseases and blood disorders. Umbilical cord blood works as an alternative for many patients who cant find bone marrow transplants.

Butthe odds that either Lola or Luca will develop a disease like cancer that would require an umbilical cord blood transplant are slim,about one in 1,000 or one in 2,000, according to University of Arizona umbilical cord blood stem cell researcher David Harris.

Its difficult to reconcile saving for yourself because youre afraid of cancer, Harris said. Do it based on facts, not fear.

Stem cells in umbilical cord blood could have another purpose: regenerative medicine. Current clinical trials show promise for the use of umbilical cord blood to treat a host of conditions such as neurological disorders, orthopedic injuriesand even diabetes. These potential usages are a new draw for parents to bank their childs umbilical cord blood.

The odds of use for these regenerative medicine applications is much, much higher, Harris said.

He estimates the odds of developing a disease that could be treated by umbilical cord blood stem cells is about one in ten.

Butthe science is still developing, which meanscompanies are selling parents ona product they may never be able to use.

In the past two decades, Harris has been involved with studies to treat kids with conditions like cerebral palsy, strokes, traumatic brain injuries and diabetes.

When you now start to talk about being able to treat a knee injury or a heart attack, or a stroke the ability to actually do that and then to see that it works is very exciting, he said. And thats really where the use of cord blood is going.

So far, Harris said hes seen the most success with orthopedic injuries and in treating children that have suffered from strokes.

With strokes, Harris said he observed children go from being completely paralyzed on one side to being fully functioning.

DONATIONS:Donated cord blood saved Sophie Lee's life, but most parents throw it away

Though some initial results show promise, Harris acknowledges that to move forward with any of these treatments, researchers need to demonstrate a good success rate.

The big question when it comes to using cord blood for regenerative medicine is when it will be incorporated into actual medical practice. For safety reasons, FDA approval for new treatments can take years,if not decades.

Currently, the only way to use umbilical cord blood stem cells for FDA-approved regenerative medicine is to qualify for and register in clinical trials to treat specific conditions. Harris has banked his owns sons cord blood on his belief that more and more umbilical cord blood treatments will become commercially available in the future. Buzan has done the same.

Brandon Buzan packages up his newborn son's umbilical cord blood to be shipped to the Cord Blood Registry lab for storage.(Photo: Amanda Morris)

Its not something that you want to say I wish I had done this, because you cant do it later.You have this one shot,"Buzan said."Even its like one percent of saving their life or helpingI mean for your child, youd do anything as a parent.

In Arizona, obstetrics health providers are required by law to educateexpectant parents about the options to publicly donate or privately bank cord blood. YetDr. Jaime Shamonki, the Chief Medical Officer of CBR, estimates that less than 5% of the population chooses to bank cord blood. A small percentage donate the blood, but a much larger percentage simply throws it away.

Kelly Helms, a Scottsdale-based OB-GYN, said the most common reasonher patients give for not banking their childs cord blood is the cost.

Buzan, who is one of her patients, said she got a discount to bank her childrens samples with CBR because of a connection her familyhad with the company. She paid a little over $1,000 for the initial processing and storage fee for both samples, and continues to pay an annual storage fee of about $120 for each one.

To bank one sample of cord blood and cord blood tissue, the initial cost is$2,830, according to CBR, with a $360 annual storage fee after that. To bank just the cord blood, not the tissue, the initial cost is $1,680, with a $180annual storage fee. Both cord blood and cord tissue have different types of stem cells that are thought to potentially repair the body in different ways.

The stem cells and potential treatments for both sources arent fully understood yet, so there are no guarantees that parents or children will actually be able to use the samplesthey pay to store.

Of the nearly 900,000 samples CBR keeps in storage, Shamonki estimates about 600 have been released for customers to use,representing a usage rate of less than 1 percent. According to Shamonki, the low sample release rate is due to FDA regulations, which stipulate that umbilical cord blood can only be used in approved treatments or clinical trials.

If we didnt have the FDA, then I would be able to release like thousands of units, she said. Its really a regulatory problem, its not a utility problem.

To boost usage of the samples, CBR maintains a registry to match eligible customers to clinical trials and has partnered with research institutions to sponsor clinical trials.

Despite FDA regulations, which Shamonki acknowledges are important to protect public health, she said CBR is releasing more and more units every year. Of the samples taken out, about 83% are used for regenerative medicine purposes, according to CBR.

What I know is that in the next fiveyears, next 10 years, there will be so many more opportunities, Shamonki said. So just because you dont have 100 different clinical trials you can sign up for tomorrow doesnt mean that these applications wont exist in fiveor 10 years, and your child might need it.

CBR is one of many companies that market cord blood banking to new parentsand is one of the biggest. Helms said she always recommends her patients to do their research and pick one of the larger, more established cord blood banking companies. Such companies might includeCBR, Cryo-cell, or ViaCord.

I've had patients lose their cord blood, privately banked blood, because they went with a small company and now they closed down, Helms said.

Even with some of the larger companies, the process of cord blood banking doesnt always run smoothly.

Although she couldn't have her daughter's cord blood stored, Chelsea Radford says she paid over $1,000 to ViaCord for processing fees.(Photo: Amanda Morris)

The first time Phoenix-resident Chelsea Radford heard about cord blood banking, she was already pregnant and facingmyriaddecisions that were sometimes overwhelming. She had never heard of it before reading some pamphlets from her gynecologist, but she was quickly sold on the idea of banking her daughters cord blood and tissue with ViaCord.

Radford has a history of Alzheimers disease in her family, and said she was initially interested in what potential treatments cord blood and tissue might offer for the disease. In 2015, one study suggested that human umbilical cord blood cells could have therapeutic benefitsin mice with Alzheimers disease.

In addition to researching studies, Radford said she spent hours looking at different cord blood banking companies and asking representatives questions about the process. Of all the companies, she found ViaCord to be the most responsive and willing to answer her questions in-depth.

Having the communication and the availability that is what sold me on ViaCord. But that really quickly ended there with the sale, she said.

On the day of her daughter Brylees birth in July2018,Radford went to a hospital that ViaCord had assured her was familiar with collecting cord blood. Soon after, she got a call from the company saying her sample couldnt be stored because not enough blood was collected. Shestill owed ViaCord over $1,000 for a lab processing fee.

Radford wanted more information before she would agree to pay, and said she spent monthscalling, leaving the company messages, and getting no response.

After the birth, nobody responds to anything, she said.

The company called her three months later to tell her that if she didnt pay her bill, they would send it to collections.

I got pissed! Radford said. The only thing they seem to care about is the moneythey dont care about is having a conversation with me about why and how this sample didnt turn out the way it shouldve. All they want to talk to me about is the money.

Still, Radford said she refused to pay a dime until she got an explanation. She contacted the doctor who delivered her baby and said she learned the doctor had never done a cord blood collection before and had never been trained on how to do one.

Finally, she spoke with a ViaCord representative, who she saidtold her this sometimes happens, butthe company wasnt responsible for the fact that the doctor who took her sample didnt take enough blood.

Frustrated and inundated with other responsibilities that come with caring for a newborn, Radford said she decided to pay the fee so that she could move on.

We paid a company to do nothing for us just to get them to leave us alone and not send a bill to collections that I dont feel like we shouldve had to foot in the first place, she said.

If she had a second child, Radford said she wouldnt choose to cord blood bank again, and doesnt recommend it to other moms.

You can still get stem cell help without using your own banked blood and tissueThis is just a costly option that is not a given that its going to work out 100% in your favor, she said. You could have a newborn and be responsible to pay thousands of dollars for nothing.

ViaCord did not respondto multiple requests for comment.

If parents decide to pay for private banking, Radford said they should be careful about making sure the doctors delivering their children know how to collect the samples. Shesaid blood banking companies should be more responsible in making sure that doctors are trained in blood collection.

While Helms is comfortable doing cord blood collection, she was never formally taught how to do it while studying and training to be an OB-GYN.

It was basically taught by the company, she said. Each kits a little different.

Helms said the procedure is fairly simple, but every once in a while, she comes across a company shes never heard of, and a kit she is unfamiliar with. Sometimes she needs to take on the extra responsibility of making sure she understands the directions for that particular kit.

Each company really should take on the responsibility, she said. I can't surf the Internet and look for every YouTube video on every cord blood banking company, she said.

Another potential complication in banking cord blood orblood tissue is that the blood or tissue can become infected.

Birth is not that clean of a process and ideally when you take that needle and you drain the umbilical cord, youll have cleaned that umbilical cord first and you hope that no bacteria get in to the cord blood unit, but its possible, it does happen on a number of occasions, Shamonki said.

Shamonki says CBR tests for any bacterial contamination before storing the tissue and works with parents who have infected samples to discuss possibilities of being able to use the unit in the future.

A Cord Blood Registry worker processes cord blood for storage in the company's Tucson laboratory.(Photo: Amanda Morris)

Nobody knows when regenerative medicine applications for cord blood will become readily available as FDA-approved mainstreamtreatments. New applications for cord blood are being tested every year and new technologies to expand and utilize stem cells in cord blood are constantly being developed.

We dont really know what the limits are, but there are limits to what (umbilical cord blood stem cells) can do, Harris said.

Because cord blood banking is so new it has only been around since 1989 its unclear how long samples can be stored and remain effective.

According to Harris, cord blood samples can still work after being stored for about two decades.

We recently took one out that was 24, 25 years old, he said.

He speculates that properly stored cord blood samples could probably work throughout a person's lifetime, if not longer.

Buzan is aware that stem cells and cord blood treatments are still a new science with no guarantees, but she also believes in the treatments future potential.

Every month, she and her husband receive email updates from CBR that explain some of the new clinical trials and research discoveries involving cord blood.

To be honest the most exciting thing is the unknown the unknown of what the cord blood could do, what theyre looking into now, thats fascinating, she said. Im just so glad we live in a time that where this is available to use this was not an option for my parents or my grandparents.

Amanda Morris covers all things bioscience, which includeshealth care,technology, new researchand the environment. Send her tips, story ideas, or dog memes at amorris@gannett.com and follow her on Twitter @amandamomorris for the latest bioscience updates.

Independent coverage of bioscience in Arizona is supported by a grant from the Flinn Foundation.

Support local journalism.Subscribe to azcentral.com today.

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Cord blood banks sell parents on promising stem cell research, but with no guarantees - The Arizona Republic

Genetic engineering transformed stem cells into working mini-livers that extended the life of mice with liver disease – The Conversation US

Takeaways

Scientists have made progress growing human liver in the lab.

The challenge has been to direct stems cells to grow into a mature, functioning adult organ.

This study shows that stem cells can be programmed, using genetic engineering, to grow from immature cells into mature tissue.

When a tiny lab-grown liver was transplanted into mice with liver disease, it extended the lives of the sick animals.

Imagine if researchers could program stem cells, which have the potential to grow into all cell types in the body, so that they could generate an entire human organ. This would allow scientists to manufacture tissues for testing drugs and reduce the demand for transplant organs by having new ones grown directly from a patients cells.

Im a researcher working in this new field called synthetic biology focused on creating new biological parts and redesigning existing biological systems. In a new paper, my colleagues and I showed progress in one of the key challenges with lab-grown organs figuring out the genes necessary to produce the variety of mature cells needed to construct a functioning liver.

Induced pluripotent stem cells, a subgroup of stem cells, are capable of producing cells that can build entire organs in the human body. But they can do this job only if they receive the right quantity of growth signals at the right time from their environment. If this happens, they eventually give rise to different cell types that can assemble and mature in the form of human organs and tissues.

The tissues researchers generate from pluripotent stem cells can provide a unique source for personalized medicine from transplantation to novel drug discovery.

But unfortunately, synthetic tissues from stem cells are not always suitable for transplant or drug testing because they contain unwanted cells from other tissues, or lack the tissue maturity and a complete network of blood vessels necessary for bringing oxygen and nutrients needed to nurture an organ. That is why having a framework to assess whether these lab-grown cells and tissues are doing their job, and how to make them more like human organs, is critical.

Inspired by this challenge, I was determined to establish a synthetic biology method to read and write, or program, tissue development. I am trying to do this using the genetic language of stem cells, similar to what is used by nature to form human organs.

I am a researcher specializing in synthetic biology and biological engineering at the Pittsburgh Liver Research Center and McGowan Institute for Regenerative Medicine, where the goals are to use engineering approaches to analyze and build novel biological systems and solve human health problems. My lab combines synthetic biology and regenerative medicine in a new field that strives to replace, regrow or repair diseased organs or tissues.

I chose to focus on growing new human livers because this organ is vital for controlling most levels of chemicals like proteins or sugar in the blood. The liver also breaks down harmful chemicals and metabolizes many drugs in our body. But the liver tissue is also vulnerable and can be damaged and destroyed by many diseases, such as hepatitis or fatty liver disease. There is a shortage of donor organs, which limits liver transplantation.

To make synthetic organs and tissues, scientists need to be able to control stem cells so that they can form into different types of cells, such as liver cells and blood vessel cells. The goal is to mature these stem cells into miniorgans, or organoids, containing blood vessels and the correct adult cell types that would be found in a natural organ.

One way to orchestrate maturation of synthetic tissues is to determine the list of genes needed to induce a group of stem cells to grow, mature and evolve into a complete and functioning organ. To derive this list I worked with Patrick Cahan and Samira Kiani to first use computational analysis to identify genes involved in transforming a group of stem cells into a mature functioning liver. Then our team led by two of my students Jeremy Velazquez and Ryan LeGraw used genetic engineering to alter specific genes we had identified and used them to help build and mature human liver tissues from stem cells.

The tissue is grown from a layer of genetically engineered stem cells in a petri dish. The function of genetic programs together with nutrients is to orchestrate formation of liver organoids over the course of 15 to 17 days.

I and my colleagues first compared the active genes in fetal liver organoids we had grown in the lab with those in adult human livers using a computational analysis to get a list of genes needed for driving fetal liver organoids to mature into adult organs.

We then used genetic engineering to tweak genes and the resulting proteins that the stem cells needed to mature further toward an adult liver. In the course of about 17 days we generated tiny several millimeters in width but more mature liver tissues with a range of cells typically found in livers in the third trimester of human pregnancies.

Like a mature human liver, these synthetic livers were able to store, synthesize and metabolize nutrients. Though our lab-grown livers were small, we are hopeful that we can scale them up in the future. While they share many similar features with adult livers, they arent perfect and our team still has work to do. For example, we still need to improve the capacity of the liver tissue to metabolize a variety of drugs. We also need to make it safer and more efficacious for eventual application in humans.

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Our study demonstrates the ability of these lab livers to mature and develop a functional network of blood vessels in just two and a half weeks. We believe this approach can pave the path for the manufacture of other organs with vasculature via genetic programming.

The liver organoids provide several key features of an adult human liver such as production of key blood proteins and regulation of bile a chemical important for digestion of food.

When we implanted the lab-grown liver tissues into mice suffering from liver disease, it increased the life span. We named our organoids designer organoids, as they are generated via a genetic design.

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Genetic engineering transformed stem cells into working mini-livers that extended the life of mice with liver disease - The Conversation US

Procyon Technologies LLC and Novo Nordisk A/S to Collaborate on the Development of a Stem-Cell Based Therapy for Type 1 Diabetes – PRNewswire

TUCSON, Ariz., Dec. 8, 2020 /PRNewswire/ --Procyon Technologies LLC today announced that it has entered into an exclusive research collaboration and license agreement with Novo Nordisk A/S to develop an implantable cell encapsulation device to be used in Novo Nordisk's development of a novel therapy for Type 1 diabetes.

The collaboration brings together Procyon Technologies' expertise with development of oxygen enabled implantable cell encapsulation devices and Novo Nordisk's expertise in stem cell-derived insulin-secreting cells.

The partners will work together to further optimize the device and cells for clinical testing and accelerate the path to First Human Dose with the joint vision of delivering a functional cure for people living with Type 1 diabetes.

Under the terms of the agreement, Procyon Technologies, a startup founded to commercialize innovations developed at the University of Arizona College of Medicine Tucson, will receive an upfront license fee and will be eligible for further payments relating to preclinical, clinical and regulatory milestones. In addition, Procyon Technologies will receive tiered sales milestones and royalties on the annual net sales of the products resulting from the collaboration.

Novo Nordisk will be responsible for the development, manufacturing and commercialization of the products resulting from the collaboration for Type 1 diabetes.

The right cells and the right device

Type 1 diabetes is an autoimmune disease in which insulin-producing beta cells in the pancreas are mistakenly destroyed by the body's immune system. For people with Type 1 diabetes, life-long daily administration of insulin to control their blood sugar and constant blood glucose monitoring is the burden of reality.

"If we are able to offer a treatment that safely and effectively replaces the insulin-producing cells that people with Type 1 diabetes have lost, we could essentially offer them a functional cure for their disease," said Jacob Sten Petersen,DMSc, corporate vice president and head of stem cell research and development for Novo Nordisk.

Since 2008, Novo Nordisk has invested in human stem cell technology and worked on generating a protocol for stem cell-derived insulin producing islet-like clusters for the treatment of Type 1 diabetes.

But having the right cells is only half the solution; the cells also need to be protected from the recipient's immune system to avoid rejection, as well as from the autoimmunity of Type 1 diabetes.

For the last two decades, Procyon Technologies co-founder Klearchos Papas, PhD, a professor in the Department of Surgery and director of the Institute for Cellular Transplantation at the University of Arizona College of Medicine Tucson, has been working on a solution to the second part of that challenge.

"As a pancreas transplant surgeon, the idea of replacing beta cell function in a diabetic patient to prevent progression of diabetic complications makes perfect sense," said Michael M.I. Abecassis, MD, MBA, dean of the UArizona College of Medicine Tucson and professor of surgery and immunobiology. "Therefore, the notion of doing this without the need for major surgery and without the need for anti-rejection drugs by leveraging the assets of academia with those of industry represents the next frontier in curing Type 1 diabetes and preventing its complications."

With support from JDRF International and the National Institute of Diabetes and Digestive and Kidney Diseases, and utilizing key University of Arizona facilities and infrastructure (such as the BIO5 Institute device prototyping lab), Dr. Papas and his team developed oxygen enabled implantable immuno-isolation device technology with a focus on safety, practicality, and the maintenance of viability and functionality of encapsulated cells.

"We are delighted and excited to join forces with Novo Nordisk and provide the 'implantable encapsulation device' part of the functional cure for people suffering from Type 1 diabetes. Novo Nordisk is a leader in the development of stem cell-derived insulin producing islet-like clusters, has demonstrated strong commitment, and has the capacity, infrastructure and most importantly, the shared vision and interest in seeking to bring this functional cure to patients," said Dr. Papas.

"The combination of the implantable encapsulation device with islet-like clusters provides a unique opportunity to develop a novel cell therapy for diabetes. This announcement reinforces the value of JDRF in supporting science and technologies that can be further advanced in partnerships," said Esther Latres, PhD, assistant vice president of research at JDRF.

"Dr. Papas' work exemplifies our research mission in the Department of Surgery. The collaboration between our investigators and clinicians allows for the development of innovative, cutting-edge solutions to the clinical problems we treat every day," said Taylor S. Riall, MD, PhD, chair of the UArizona Department of Surgery. "The partnership between Procyon Technologies and Novo Nordisk represents the culmination of years of hard work and will revolutionize the care of people with Type 1 diabetes."

A therapeutic implant

The Procyon cell encapsulation device is a small, flat, thin, highly durable, flexible implantable chamber. It mitigates foreign body response, promotes the formation of vascular structures on its surface enabling the rapid diffusion of nutrients to the cells inside and the rapid absorption of insulin (or other therapeutic molecules) secreted by the encapsulated cells while providing a barrier protecting them from attacks by the body's immune system without the need for immunosuppressive drugs. The Procyon technology, designed with practical clinical use as a driver, includes integration of oxygen delivery to the implantable device, which enables tighter packing of cells while maintaining their viability and functionality.

About Procyon Technologies LLC:

Procyon Technologies LLC (https://procyon-technologies.com) was founded in Arizona in 2016. Klearchos Papas, PhD, Allison F. Corkey, MS, Thomas Loudovaris, PhD, and Robert C. Johnson, PhD, are co-founders and worked with Tech Launch Arizona, the University of Arizona commercialization arm, to protect the intellectual property and license the platform technology suitable for the implantation of a variety of therapeutic cells aimed at treating a number of disorders. In addition to being highly respected researchers in the field of diabetes and encapsulation therapy for decades, Dr. Johnson, a part-time research professor in the Department of Surgery at the University of Arizona, has had Type 1 diabetes for nearly 51 years and Dr. Loudovaris is the father of two children with the disease.

Contact: Allison F. Corkey [emailprotected] 520-329-1425

SOURCE Procyon Technologies LLC

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Procyon Technologies LLC and Novo Nordisk A/S to Collaborate on the Development of a Stem-Cell Based Therapy for Type 1 Diabetes - PRNewswire

Hadassah Medical Center and Neurogenesis Announce Groundbreaking Results from a Phase 2 Study in Progressive Multiple Sclerosis treated with NG-01…

JERUSALEM, Dec. 8, 2020 /PRNewswire/ --NeuroGenesis, a clinical-stage biopharmaceutical company advancing innovative cell therapies to combat myelin-related neurodegenerative diseases, and Hadassah Medical Center announced today highly positive results from a placebo-controlled Phase 2 clinical trial, headed by Prof. Dimitrios Karussis, together with Dr. Petrou Panayiota and Dr. Ibrahim Kassis from Hadassah Medical Center in Jerusalem, assessing the impact of NG-01 autologous proprietary subpopulation of mesenchymal stem cells (MSCs) on patients with progressive multiple sclerosis (MS).

The results, recently published in Brain, a prestigious peer-reviewed journal published by Oxford University, and highlighted in the "Editor's Choice", show that:

"The treatment was well tolerated and the trial met all of its primary endpoints," said Professor Dimitrios Karussis, lead principle investigator and Director of MS Center at Hadassah Medical Center, Jerusalem. "The patients' improvement was in many cases quite remarkable and included regain of motor function and noticeable effects on their cognitive abilities."

Prof Karussis added, "Although we currently have several good treatment options for relapsing remitting MS, we fall short in providing effective treatment for progressive MS that could substantially suppress the progression of disability. This trial provides encouraging results and suggests a potential for a new approach that may not only slow down the progression of the disease but even induce improvement and promote repair mechanisms in progressive MS."

The technology is now further developed by NeuroGenesis, following a license from Hadasit, Hadassah Medical Center Technology Transfer Company.

Neurogenesis' technology entails collecting bone marrow from the patient. Then by utilizing a proprietary process, a unique subpopulation of bone marrow cells is identified, cultured and enhanced towards remyelinating biofactory cells (NG-01) that also possess neurotrophic immunolatory and neuroprotective properties. The NG-01 cell population is injected directly into the central nervoussystem (through the cerebrospinal fluid), where the cells home-in on the damaged area, take up residence and produce significant amounts of neurotrophic factors.

"Progressive MS is a chronic, debilitating disease with no satisfactory treatment to improve or reverse established disability," said Tal Gilat, CEO of NeuroGenesis. "We are therefore extremely pleased to witness the significant positive effect of our NG-01 cells. Following recent interactions with the FDA, we look forward to confirming and expanding these findings in a large multi-center MS trial, and continuing advanced studies in additional indications such as ALS."

About the Phase 2 trial of NG-01

The Phase 2, randomized, double-blind, placebo-controlled, clinical trial assessed the safety, tolerability and efficacy of transplantation of NG-01 in people with progressive MS. The study enrolled 48 participants with progressive MS which were randomized into 3 groups, receiving either an intrathecal or intravenous NG-01 injection, or a placebo injection.

The two predetermined primary endpoints of the trial were: (i) the safety of the intrathecal and intravenous NG-01 treatments assessed by incidence of adverse events versus those in the placebo-treated group; and (ii) the differences among the three groups in the Expanded Disability StatusScale(EDSS) score changes and the proportion of patients with treatment failure, as evidenced by an increase in EDSS (disease progression) score, at 6 and 12 months. Overall, the study duration was 14 months.

About Multiple Sclerosis

Multiple sclerosis (MS) is an autoimmune disease that causes damage in the myelin and the nerve cells of the central nervous system (demyelinating plaques in brain and spinal cord), resulting in cumulating neurological disability. The destruction of the myelin (the covering that protects nerves and promotes the efficient transmission of nerve impulses) causes secondary damage to the nerve cells and progressive atrophy. MS often causes sensory disturbances in the limbs, including a prickling or tingling sensation (paresthesia), numbness, pain, and itching. Motor problems are common in people with MS. Affected individuals may have tremors, muscle stiffness (spasticity), exaggerated reflexes (hyperreflexia), weakness or paralysis of the muscles of the limbs, difficulty in walking, and poor sphincter control. The condition is also associated with visual problems, such as blurred or double vision or partial or complete vision loss. There is no known cure for multiple sclerosis.The existing treatments are mostly aimed to reduce the incidence of relapses of the disease and slow down the rate of neurological deterioration.

About NeuroGenesis

Neurogenesis is developing cell therapy for neurodegenerative diseases based on a unique approach for sustained delivery of high levels of remyelinating growth factors using the patient's own stem cells. The technology for this unique approach was licensed from Hadasit, theTechnology TransferCompany of Hadassah Medical Organization in Jerusalem, Israel. The Company's lead product is NG-01 for the treatment of progressive Multiple Sclerosis, (in which a placebo-controlled Phase 2 study has been completed and recently published). NG-01 were also tested in two successful Phase 2a trials in ALS patients. Up to today, more than 150 progressive MS and ALS patients from around the world have been treated with Neurogenesis'products via clinical trials (Phase 1 and Phase 2) and compassionate use treatments.

About Hadassah and Hadasit

For more than a century, Hadassah has set the standard of excellence for medical care and research in Israel. Our doctors and scientists are on the frontlines, uniquely positioned to pinpoint ever-evolving medical needs. Their experience and ingenuity have yielded new ideas with huge potential in all areas of medicine, including therapeutics, diagnostic medical devices, and digital health. Hadasit is the technology transfer company of Hadassah Medical Center in Jerusalem. We transform the cutting-edge research coming out of Hadassah into marketable medical technologies. We turn ground-breaking ideas into viable products and services that can change the world and better humanity.

NeuroGenesiscontact:Tsipi Haitovsky Global Media Liaison NeuroGenesis +972-52-5989-892 [emailprotected]

Hadassah contact:Hadar Elboim spokeswoman Hadassah Medical Organization + 972- 2-6776079 [emailprotected]

SOURCE NeuroGenesis

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Hadassah Medical Center and Neurogenesis Announce Groundbreaking Results from a Phase 2 Study in Progressive Multiple Sclerosis treated with NG-01...

Worldwide Cell Culture Industry to 2025 – Featuring Thermo Fisher Scientific, Corning Incorporated and Eppendorf Among Others – GlobeNewswire

December 08, 2020 08:18 ET | Source: Research and Markets

Dublin, Dec. 08, 2020 (GLOBE NEWSWIRE) -- The "Global Cell Culture Market by Product (Consumables [Media, Serum, Vessels], Equipment [Bioreactor, Centrifuge, Incubator, Autoclave]), Application (Therapeutic Proteins, Vaccines, Diagnostics, Stem Cells), End-user (Pharma, Biotech) and Region - Forecast to 2025" report has been added to ResearchAndMarkets.com's offering.

The global cell culture market is projected to reach USD 33.1 billion by 2025 from USD 19 billion in 2020, at a CAGR of 11.8% during the forecast period.

The growth of this market is majorly driven by the growing awareness about the benefits of cell culture-based vaccines, increasing demand for monoclonal antibodies (mAbs), funding for cell-based research, growing preference for single-use technologies, and the launch of advanced cell culture products. On the other hand, the high cost of cell biology research and the lack of proper infrastructure for cell-based research activities are the major factors restraining this market's growth.

Based on product, the consumables segment holds the largest market share during the forecast period

Based on product, the cell culture market is segmented into equipment and consumables. The consumables segment accounted for the largest market share in 2019 and is expected to register the highest CAGR during the forecast period. The dominant share and high growth of the consumables segment can be attributed to the repeated purchase of consumables and increased funding for cell-based research.

Based on application, the biopharmaceutical production segment is expected to register the highest CAGR during the forecast period

Based on application, the cell culture market is categorized into biopharmaceutical production, stem cell research, diagnostics, drug screening & development, tissue engineering and regenerative medicine, and other applications. The biopharmaceutical production application segment is expected to register the highest CAGR during the forecast period.The high growth of this segment is attributed to the commercial expansion of major pharmaceutical companies, growing regulatory approvals for the production of cell culture-based vaccines, and the increasing demand for monoclonal antibodies (mAbs).

Pharmaceutical & Biotechnology companies end-user segment is expected to grow at the highest CAGR in the cell culture market during the forecast period

Based on end-users, the cell culture market is segmented into pharmaceutical & biotechnology companies, academic & research institutes, hospitals and diagnostic centers, and cell banks. The pharmaceutical & biotechnology companies segment is expected to witness the highest growth during the forecast period. The growing use of single-use technologies, the increasing number of regulatory approvals for cell culture-based vaccines, and the presence of a large number of pharmaceutical players in this market are some of the factors driving the cell culture market for this end-user segment.

North America is expected to account for the largest share of the cell culture market in 2019

In 2019, North America accounted for the largest share of the cell culture market, followed by Europe, the Asia-Pacific, Latin America, and the Middle East & Africa. The large share of this market segment can be attributed to the growing regulatory approvals for cell culture-based vaccines, technological advancements, and growth in the biotechnology & pharmaceutical industries in the region are the key factors driving the growth of the cell culture market in North America.

Key Topics Covered:

1 Introduction

2 Research Methodology 3 Executive Summary

4 Premium Insights 4.1 Cell Culture: Market Overview 4.2 Asia-Pacific: Cell Culture Market Share, by End-user and Country (2019) 4.3 Cell Culture Market: Geographic Growth Opportunities 4.4 Regional Mix: Cell Culture Market 4.5 Cell Culture Market: Developed Vs. Developing Markets

5 Market Overview 5.1 Introduction 5.2 Market Dynamics 5.2.1 Market Drivers 5.2.1.1 Growing Awareness About the Benefits of Cell Culture-Based Vaccines 5.2.1.2 Increasing Demand for Monoclonal Antibodies 5.2.1.3 Funding for Cell-Based Research 5.2.1.4 Growing Preference for Single-Use Technologies 5.2.1.5 Launch of Advanced Cell Culture Products 5.2.1.6 Growing Focus on Personalized Medicine 5.2.2 Market Restraints 5.2.2.1 High Cost of Cell Biology Research 5.2.2.2 Lack of Infrastructure for Cell-Based Research in Emerging Economies 5.2.3 Market Opportunities 5.2.3.1 Growing Demand for 3D Cell Culture 5.2.3.2 The Growing Risk of Pandemics and Communicable Diseases 5.2.3.3 Emerging Economies 5.3 COVID-19 Impact on the Cell Culture Market 5.4 Value Chain Analysis 5.5 Supply Chain Analysis 5.6 Ecosystem Analysis 5.7 Regulatory Analysis

6 Cell Culture Market, by Product 6.1 Introduction 6.2 Impact of the COVID-19 on the Cell Culture Market 6.3 Consumables 6.3.1 Sera, Media, and Reagents 6.3.1.1 Media 6.3.1.1.1 Serum-Free Media 6.3.1.1.1.1 Lack of Sera Eliminates the Risk of Contamination by Viruses 6.3.1.1.2 Classical Media & Salts 6.3.1.1.2.1 Classical Media is Commonly Used in Virology, Vaccine Production, and Primary Tissue Explant Culture 6.3.1.1.3 Stem Cell Culture Media 6.3.1.1.3.1 Stem Cell Culture Media to Witness the Highest Growth in the APAC Market During the Forecast Period 6.3.1.1.4 Specialty Media 6.3.1.1.4.1 Specialty Media is Suitable for the Growth of Selective Cell Types 6.3.1.2 Reagents 6.3.1.2.1 Growth Factors 6.3.1.2.1.1 Growth Factors are Unique Cell Signaling Molecules That Help in Cell Proliferation & Development 6.3.1.2.2 Supplements 6.3.1.2.2.1 Supplements Such as Amino Acids Play a Key Role in Inducing Cell Growth 6.3.1.2.3 Buffers & Chemicals 6.3.1.2.3.1 Chemical Buffers are Widely Used, But It Can be Toxic at Higher Concentrations 6.3.1.2.4 Cell Dissociation Reagents 6.3.1.2.4.1 Dissociation Reagents Can be Enzymatic or Non-Enzymatic 6.3.1.2.5 Balanced Salt Solutions 6.3.1.2.5.1 Balanced Salt Solutions Find Wide Applications in Life Sciences 6.3.1.2.6 Attachment & Matrix Factors 6.3.1.2.6.1 The Development of Cells is Dependent on Attachment Factors 6.3.1.2.7 Antibiotics/Antimycotics 6.3.1.2.7.1 The Possibility of Contamination Risks Make the Long-Term Use of Antibiotics/Antimycotics Conditional 6.3.1.2.8 Contamination Detection Kits 6.3.1.2.8.1 Contamination Detection Kits Provide Rapid Results 6.3.1.2.9 Cryoprotective Reagents 6.3.1.2.9.1 Cryoprotective Reagents Protect Tissues/Cells from Damage due to Freezing 6.3.1.2.10 Other Cell Culture Reagents 6.3.1.3 Sera 6.3.1.3.1 Fetal Bovine Sera (FBS) 6.3.1.3.1.1 Use of FBS is Now Restricted due to Regulatory Guidelines 6.3.1.3.2 Adult Bovine Sera (ABS) 6.3.1.3.2.1 ABS is a Cost-Effective Alternative to FBS and is Used as a Biochemical Reagent in IVD 6.3.1.3.3 Other Animal Sera 6.3.2 Vessels 6.3.2.1 Roller/Roux Bottles 6.3.2.1.1 Roller Bottles Offer an Economical Means of Cultivating Large Cell Volumes 6.3.2.2 Cell Factory Systems/Cell Stacks 6.3.2.2.1 Cell Stacks Require Special Handling Equipment and Skilled Expertise 6.3.2.3 Multiwell Plates 6.3.2.3.1 Larger Well Formats Allow for Greater Culture Volumes 6.3.2.4 Flasks 6.3.2.4.1 Disposable Flasks are in Greater Demand Among End-users 6.3.2.5 Petri Dishes 6.3.2.5.1 The Wide Usage of Petri Dishes is Attributed to Ease of Use 6.3.3 Bioreactor Accessories 6.4 Equipment 6.4.1 Supporting Equipment 6.4.2 Bioreactors 6.4.3 Storage Equipment

7 Cell Culture Market, by Application 7.1 Introduction 7.2 Impact of the COVID-19 on the Cell Culture Market 7.3 Biopharmaceutical Production 7.3.1 Therapeutic Proteins 7.3.1.1 Growing Applications of Recombinant Proteins to Drive the Growth of this Segment 7.3.2.1 Rising Incidence of Disease Outbreaks to Drive the Market Growth for Vaccine Production 7.4 Diagnostics 7.4.1 The Growing Risk of Viral Infections Drives the Uptake of Cell Culture Products for Diagnostics 7.5 Drug Screening & Development 7.5.1 Increasing Adoption of Cell-Based Assays in R&D Activities to Drive Segment Growth 7.6 Stem Cell Research 7.6.1 Increasing Stem Cell Research Activities & Investments Drive Segment Growth 7.7 Tissue Engineering and Regenerative Medicine 7.7.1 Increasing Funding for Regenerative Medicine Boosts Segment Growth 7.8 Other Applications

8 Cell Culture Market, by End-user 8.1 Introduction 8.2 Impact of the COVID-19 on the Cell Culture End-User Market 8.3 Pharmaceutical & Biotechnology Companies 8.3.1 Growing Regulatory Approvals for Cell-Culture Based Vaccines Drives Segment Growth 8.4 Hospitals and Diagnostic Laboratories 8.4.1 Increasing Applications of Cell Culture for the Diagnosis of Various Diseases to Drive Segment Growth 8.5 Research & Academic Institutes 8.5.1 Increasing Government Funding for Research Projects and the High Prevalence of Cancer to Drive Growth for this End-User Segment 8.6 Cell Banks 8.6.1 Increasing Awareness of Preserving Stem Cells to Drive Segment Growth

9 Cell Culture Market, by Region 9.1 Introduction 9.2 COVID-19 Impact on the Cell Culture Market 9.3 North America 9.5 Asia-Pacific 9.6 Latin America 9.7 Middle East and Africa

10 Competitive Landscape 10.1 Overview 10.2 Competitive Scenario 10.2.1 Partnerships, Agreements, and Collaborations (2020) 10.2.2 Product Launches & Upgrades (2020) 10.2.3 Expansions (2020) 10.2.4 Acquisitions (2019-2020)

11 Company Evaluation Matrix and Company Profiles 11.1 Company Evaluation Matrix Definition & Methodology 11.2 Competitive Leadership Mapping (2019) 11.2.1 Stars 11.2.2 Emerging Leaders 11.2.3 Pervasive Companies 11.2.4 Emerging Companies 11.3 Market Share Analysis, 2019 11.4 Company Profiles 11.4.1 Thermo Fisher Scientific Inc. 11.4.2 Merck KGaA 11.4.3 Becton, Dickinson & Company 11.4.4 Corning Incorporated 11.4.5 Danaher Corporation 11.4.6 Eppendorf AG 11.4.7 Fujifilm Irvine Scientific, Inc. (Acquired by Fujifilm Corporation) 11.4.8 Lonza Group AG 11.4.9 Sartorius AG 11.4.10 Cellgenix GmbH 11.4.11 Miltenyi Biotec 11.4.12 Stemcell Technologies, Inc. 11.4.13 Himedia Laboratories 11.4.14 Invivogen 11.4.15 Infors AG 11.4.16 Promocell 11.4.17 Pan Biotech GmbH 11.4.18 Seracare Life Sciences Incorporation 11.4.19 Caisson Labs 11.4.20 Solida Biotech GmbH

12 Appendix 12.1 Insights from Industry Experts 12.2 Discussion Guide 12.3 Knowledge Store: The Subscription Portal 12.4 Available Customizations

For more information about this report visit https://www.researchandmarkets.com/r/wtzt47

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Worldwide Cell Culture Industry to 2025 - Featuring Thermo Fisher Scientific, Corning Incorporated and Eppendorf Among Others - GlobeNewswire

The cell culturemarket is projected to reach USD 33.1 billion by 2025 from USD 19.0 billion in 2020, at a CAGR of 11.8% – GlobeNewswire

December 08, 2020 08:52 ET | Source: ReportLinker

New York, Dec. 08, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Cell Culture Market by Product, Application, End User - Global Forecast to 2025" - https://www.reportlinker.com/p03665912/?utm_source=GNW On the other hand, the high cost of cell biology research and the lack of proper infrastructure for cell-based research activities are the major factors restraining this markets growth.

Based on product, the consumables segment holds the largest market share during the forecast period. Based on product, the cell culture market is segmented into equipment and consumables.The consumables segment accounted for the largest market share in 2019 and is expected to register the highest CAGR during the forecast period.

The dominant share and high growth of the consumables segment can be attributed to the repeated purchase of consumables and increased funding for cell-based research.

Based onapplication, thebiopharmaceutical productionsegment is expected to register the highest CAGR during the forecast period. Based on application, the cell culture market is categorized into biopharmaceutical production, stem cell research, diagnostics, drug screening & development, tissue engineering and regenerative medicine, and other applications. The biopharmaceutical production application segment is expected to register the highest CAGR during the forecast period.The high growth of this segment is attributed to the commercial expansion of major pharmaceutical companies, growing regulatory approvals for the production of cell culture-based vaccines, and the increasing demand for monoclonal antibodies (mAbs).

Pharmaceutical &Biotechnology companies end user segment is expected to grow at the highest CAGR in the cell culture market during the forecast period Based on end users, the cell culture market is segmented into pharmaceutical & biotechnology companies, academic & research institutes, hospitals and diagnostic centers, and cell banks. The pharmaceutical & biotechnology companies segment is expected to witness the highest growth during the forecast period. The growing use of single-use technologies, the increasing number of regulatory approvals for cell culture-based vaccines, and the presence of a large number of pharmaceutical players in this market are some of the factors driving the cell culture market for this end user segment

North America is expected to account for the largest share of the cell culture market in 2019 In 2019, North America accounted for the largest share of the cell culture market, followed by Europe, the Asia Pacific, Latin America, and the Middle East & Africa. The large share of this market segment can be attributed to thegrowing regulatory approvals for cell culture-based vaccines, technological advancements, and growth in the biotechnology & pharmaceutical industries in the region are the key factors driving the growth of the cell culture market in North America

Break of primary participants was as mentioned below: By Company Type Tier 136%, Tier 245%, and Tier 319% By Designation C-level33%, Director-level40%, Others27% By Region North America36%, Europe28%, AsiaPacific19%, Latin America-9%, Middle East and Africa8%

Key players in the cell culturemarket The key players operating in the cell culturemarketincludeThermo Fisher Scientific (US), Merck KGaA (Germany), GE Healthcare (US), Danaher Corporation (US), Lonza Group AG (Switzerland), Becton, Dickinson and Company (US), Corning Incorporated (US), Eppendorf (Germany), HiMedia Laboratories (India), Sartorius AG (Germany), PromoCell GmbH (Germany), FUJIFILM Irvine Scientific (US), InvivoGen (US), CellGenix GmbH (Germany), SeraCare Life Sciences Incorporation (US), Miltenyi Biotec (Germany), STEMCELL Technologies, Inc. (Canada), Solida Biotech GmBH (Germany), Caisson Labs Inc. (US), Cellexus Ltd, (UK), PIERRE GURIN (France), Applikon Biotechnology BV (Netherlands), WISENT Inc. (Canada), Koh Jin-Bio Co., Ltd. (Japan), PAN-Biotech GmbH (Germany), and Infors AG (Switzerland).

Research Coverage: The report analyzes the cell culturemarketand aims at estimating the market size and future growth potential of this market based on various segments such as product, application, end user, andregion.The report also includes aproduct portfolio matrix of various cell cultureproductsavailable in the market.

The report also providesa competitive analysis of the key players in this market, along with their company profiles, product offerings, and key market strategies.

Reasons to Buy the Report The report will enrich established firms as well as new entrants/smaller firms to gauge the pulse of the market, which in turn would helpthem, garner a more significant share of the market. Firms purchasing the report could use one or any combination of the below-mentioned strategies tostrengthen their position in the market.

This report provides insights into the following pointers: Market Penetration: Comprehensive information on product portfolios offered by the top players in the global cell culturemarket. The report analyzes this marketby product, application, and end user Product Enhancement/Innovation: Detailed insights on upcoming trends and productlaunches in the global cell culturemarket Market Development: Comprehensive information on the lucrative emerging markets by product, application, and end user Market Diversification: Exhaustive information about new products or product enhancements, growing geographies, recent developments, and investments in theglobal cell culturemarket Competitive Assessment: In-depth assessment of market shares, growth strategies, product offerings, competitive leadership mapping, and capabilities of leading players in theglobal cell culturemarket.

Read the full report: https://www.reportlinker.com/p03665912/?utm_source=GNW

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

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The cell culturemarket is projected to reach USD 33.1 billion by 2025 from USD 19.0 billion in 2020, at a CAGR of 11.8% - GlobeNewswire

Treatment with Investigational LentiGlobin Gene Therapy for Sickle Cell Disease (bb1111) Results in Complete Elimination of SCD-Related Severe…

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) announced that new data from Group C of its ongoing Phase 1/2 HGB-206 study of investigational LentiGlobin gene therapy (bb1111) for adult and adolescent patients with sickle cell disease (SCD) show a complete elimination of severe VOEs and VOEs between six and 24 months of follow-up. These data are being presented at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place virtually from December 5-8, 2020.

Now with more than two years of data, we continue to observe promising results in our studies of LentiGlobin for SCD that further illustrate its potential to eliminate the symptoms and devastating complications of sickle cell disease. Consistently achieving the complete resolution of severe vaso-occlusive events (VOEs) and VOEs between Month 6 and Month 24 follow-up is unprecedented other than with allogeneic stem cell transplantation. Importantly, our data show the potential for LentiGlobin for SCD to produce fundamentally disease-modifying effects with sustained pancellular distribution of gene therapy-derived anti-sickling HbAT87Q and improvement of key markers of hemolysis that approach normal levels, said David Davidson, M.D., chief medical officer, bluebird bio. In addition to these clinical outcomes, for the first time with a gene therapy we now have patient-reported outcomes through the validated PROMIS-57 tool, showing reduction in pain intensity at 12 months after treatment with LentiGlobin for SCD. These results provide insight into the potential real-life impact LentiGlobin for SCD may offer patients.

SCD is a serious, progressive and debilitating genetic disease. In the U.S., the median age of death for someone with sickle cell disease is 43 46 years. SCD is caused by a mutation in the -globin gene that leads to the production of abnormal sickle hemoglobin (HbS). HbS causes red blood cells to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and unpredictable, painful VOEs.

In the HGB-206 study of LentiGlobin for SCD, VOEs are defined as episodes of acute pain with no medically determined cause other than a vaso-occlusion, lasting more than two hours and severe enough to require care at a medical facility. This includes acute episodes of pain, acute chest syndrome (ACS), acute hepatic sequestration and acute splenic sequestration. A severe VOE requires a 24-hour hospital stay or emergency room visit or at least two visits to a hospital or emergency room over a 72-hour period, with both visits requiring intravenous treatment.

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 (HbAT87Q) that decreases the proportion of HbS, with the goal of reducing sickled red blood cells, hemolysis and other complications.

As a hematologist, I regularly see the debilitating effects of pain events caused by sickle cell disease. Pain has an overwhelmingly negative impact on many facets of my patients lives and can lead to prolonged hospitalizations, said presenting study author Alexis A. Thompson, M.D., professor of pediatrics at Northwestern University Feinberg School of Medicine and head of hematology at Ann and Robert H. Lurie Childrens Hospital of Chicago. The results observed with LentiGlobin gene therapy for SCD include the complete elimination of severe vaso-occlusive pain episodes, which is certainly clinically meaningful, but also for the first time, we have documented patients reporting that they are experiencing improved quality of life. This degree of early clinical benefit is extraordinarily rewarding to observe as a provider."

As of the data cut-off date of August 20, 2020, a total of 44 patients have been treated with LentiGlobin for SCD in the HGB-205 (n=3) and HGB-206 (n=41) clinical studies. The HGB-206 total includes: Groups A (n=7), B (n=2) and C (n=32).

HGB-206: Group C Updated Efficacy Results

The 32 patients treated with LentiGlobin for SCD gene therapy in Group C of HGB-206 had up to 30.9 months of follow-up (median of 13.0; min-max: 1.1 30.9 months).

In patients with six or more months of follow-up whose hemoglobin fractions were available (n=22), median levels of gene therapy-derived anti-sickling hemoglobin, HbAT87Q, were maintained with HbAT87Q contributing at least 40% of total hemoglobin at Month 6. At last visit reported, total hemoglobin ranged from 9.6 15.1 g/dL and HbAT87Q levels ranged from 2.7 8.9 g/dL. At Month 6, the production of HbAT87Q was associated with a reduction in the proportion of HbS in total hemoglobin; median HbS was 50% and remained less than 60% at all follow-up timepoints. All patients in Group C were able to stop regular blood transfusions by three months post-treatment and remain off transfusions as of the data cut-off.

Nineteen patients treated in Group C had a history of severe VOEs, defined as at least four severe VOEs in the 24 months prior to informed consent (annualized rate of severe VOE min-max: 2.0 10.5 events) and at least six months follow-up after treatment with LentiGlobin for SCD. There have been no reports of severe VOEs in these Group C patients following treatment with LentiGlobin for SCD. In addition, all 19 patients had a complete resolution of VOEs after Month 6.

Hemolysis Markers

In SCD, red blood cells become sickled and fragile, rupturing more easily than healthy red blood cells. The breakdown of red blood cells, called hemolysis, occurs normally in the body. However, in sickle cell disease, hemolysis happens too quickly due to the fragility of the red blood cells, which results in hemolytic anemia.

Patients treated with LentiGlobin for SCD in Group C demonstrated near-normal levels in key markers of hemolysis, which are indicators of the health of red blood cells. Lab results assessing these indicators were available for the majority of the 25 patients with 6 months of follow-up.

The medians for reticulocyte counts (n=23), lactate dehydrogenase (LDH) levels (n=21) and total bilirubin (n=24) continued to improve compared to screening values and stabilized by Month 6. In patients with Month 24 data (n=7), these values approached the upper limit of normal by Month 24. These results continue to suggest that treatment with LentiGlobin for SCD may improve biological markers to near-normal levels for SCD.

Pancellularity

As previously reported, assays were developed by bluebird bio to enable the detection of HbAT87Q and HbS protein in individual red blood cells, as well as to assess if HbAT87Q was pancellular, or present throughout all of a patients red blood cells. In 25 patients with at least six months of follow-up, on average, more than 80% of red blood cells contained HbAT87Q, suggesting near-complete pancellularity of HbAT87Q distribution and with pancellularity further increasing over time.

HGB-206: Improvements in Health-Related Quality of Life

Health-related quality of life (HRQoL) findings in Group C patients treated with LentiGlobin for SCD in the HGB-206 study were generated using the Patient Reported Outcomes Measurement Information System 57 (PROMIS-57), a validated instrument in SCD.

Data assessing pain intensity experienced by nine Group C patients were analyzed according to baseline pain intensity scores relative to the general population normative value: 2.6 on a scale of 0-10, where 10 equals the most intense pain. Data were assessed at baseline, Month 6 and Month 12.

Of the five patients with baseline scores worse than the population normative value average, four demonstrated clinically meaningful reductions in pain intensity at Month 12; the group had a mean score of 6.0 at baseline and a mean score of 2.4 at Month 12. Of the four patients with better than or near population normative values at baseline, two reported improvement and two remained stable with a mean score of 2.3 at baseline and 0.8 at Month 12.

HGB-206: Group C Safety Results

As of August 20, 2020, the safety data from Group C patients in HGB-206 remain generally consistent with the known side effects of hematopoietic stem cell collection and myeloablative single-agent busulfan conditioning, as well as underlying SCD. One non-serious, Grade 2 adverse event (AE) of febrile neutropenia was considered related to LentiGlobin for SCD. There were no serious AEs related to LentiGlobin for SCD.

One patient with significant baseline SCD-related and cardiopulmonary disease died 20 months post-treatment; the treating physician and an independent monitoring committee agreed his death was unlikely related to LentiGlobin for SCD and that SCD-related cardiac and pulmonary disease contributed.

LentiGlobin for SCD Data at ASH

The presentation of HGB-206 Group C results and patient reported outcomes research are now available on demand on the ASH conference website:

About HGB-206

HGB-206 is an ongoing, Phase 1/2 open-label study designed to evaluate the efficacy and safety of LentiGlobin gene therapy for sickle cell disease (SCD) that includes three treatment cohorts: Groups A (n=7), B (n=2) and C (n=32). A refined manufacturing process designed to increase vector copy number (VCN) and further protocol refinements made to improve engraftment potential of gene-modified stem cells were used for Group C. Group C patients also received LentiGlobin for SCD made from HSCs collected from peripheral blood after mobilization with plerixafor, rather than via bone marrow harvest, which was used in Groups A and B of HGB-206.

About LentiGlobin for SCD (bb1111)

LentiGlobin gene therapy for sickle cell disease (bb1111) is an investigational treatment being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the completed Phase 1/2 HGB-205 study, the ongoing Phase 1/2 HGB-206 study, and the ongoing Phase 3 HGB-210 study.

The U.S. Food and Drug Administration 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 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.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-307) 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 NCT04628585 for LTF-307.

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

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene and cell therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders: cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using gene and cell therapy technologies including gene addition, and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

LentiGlobin and bluebird bio are trademarks of bluebird bio, Inc.

Forward-Looking Statements

This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: regarding the potential for LentiGlobin for Sickle Cell Disease to treat SCD; the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing or planned clinical trials; the risk that the current or planned clinical trials of our product candidates will be insufficient to support regulatory submissions or marketing approval in the United States and European Union; the risk that regulatory authorities will require additional information regarding our product candidates, resulting in delay to our anticipated timelines for regulatory submissions, including our applications for marketing approval; and the risk that any one or more of our product candidates, will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

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Treatment with Investigational LentiGlobin Gene Therapy for Sickle Cell Disease (bb1111) Results in Complete Elimination of SCD-Related Severe...

New DARZALEX (daratumumab) Data from GRIFFIN Study Show Deeper and Longer Responses in Patients with Newly Diagnosed Multiple Myeloma – BioSpace

HORSHAM, Pa., Dec. 7, 2020 /PRNewswire/ --The Janssen Pharmaceutical Inc. Companies of Johnson & Johnson announced new data from the randomized Phase 2 GRIFFIN study showing that the addition of DARZALEX (daratumumab) to lenalidomide (Revlimid), bortezomib (VELCADE) and dexamethasone (D-RVd), followed by DARZALEXplus lenalidomide (D-R) maintenance therapy, resulted in deeper and improved responses, including minimal residual disease (MRD) negativity, compared to RVd followed by R alone in newly diagnosed, stem cell transplant-eligible patients with multiple myeloma.1These data investigating the use of DARZALEX in combination with RVd, which were shared in separate oral and poster presentations at the American Society of Hematology (ASH) 2020 Annual Meeting, provide further evidence that this regimen may provide greater efficacy for transplant-eligible, newly diagnosed multiple myeloma(NDMM) than standard therapy. The oral presentation (Abstract #549) shared longer-term follow-up data, and the poster presentation (Abstract #3243) featured additional data from the safety run-in cohort.1,2

"The long-term GRIFFIN data show that maintenance therapy with DARZALEX in combination with lenalidomide (D-R) resulted in deeper responses compared to R alone in patients with multiple myeloma who are newly diagnosed and transplant-eligible," said Peter Voorhees, M.D., Atrium Health's Levine Cancer Institute and GRIFFIN study investigator. "These data indicate that the addition of DARZALEX to RVd followed by R maintenance results in improved response rates and depth of response during induction, consolidation and maintenance treatment cycles."

Key Findings from GRIFFIN (Abstract #549): The GRIFFIN oral presentation featured updated safety and efficacy data based onlonger follow-up for D-RVd and evaluated the potential role of D-R for maintenance therapy in patients with NDMM.1

Key Findings from GRIFFIN (Abstract #3243): The poster presentation shared final results of the safety run-in cohort (n=16 patients) from the GRIFFIN study. Theseadditional data showed that maintenance therapy with DARZALEX and lenalidomide (D-R) improved both the sCR rate and MRD negativity rate in patients with NDMMwho underwent D-RVd induction, autologous stem cell transplant (ASCT) and D-RVd consolidation. This deepening of responses was associated with durable remissions, and no new safety signals were observed with maintenance therapy.2

"We continue to be encouraged by the GRIFFIN data showing deeper and improved responses in patients with newly diagnosed, ASCT-eligible multiple myeloma," said Andree Amelsberg, M.D., MBA, Vice President, Oncology Medical Affairs, Janssen Scientific Affairs, LLC. "These data show promising results for patients with newly diagnosed multiple myeloma, and we remain committed to exploring the full potential of DARZALEX and DARZALEX FASPRO."

About the GRIFFIN Study4 The Phase 2 GRIFFIN (NCT02874742) study has enrolled and treated more than 200 adults ages 18-70 years with NDMM and who are eligible for high-dose therapy/ASCT.

In the safety run-in cohort, patients received 25 mg of lenalidomide orally on Days 1-14; 1.3 mg/m2 of bortezomib subcutaneously on Days 1, 4, 8 and 11; and 20 mg of dexamethasone on Days 1, 2, 8, 9, 15 and 16, every 21 days during the induction and consolidation phases (Cycles 1-6). DARZALEX 16 mg/kg IV was given on Days 1, 8 and 15 of Cycles 1-4 and on Day 1 of Cycles 5-6.

During maintenance phase (Cycles 7-32), patients received 10 mg daily of lenalidomide (15 mg beginning at Cycle 10 if tolerated) on Days 1-21 every 28 days and DARZALEX 16 mg/kg IV every 56 days; this was amended to every 28 days based upon emerging clinical pharmacokinetic data demonstrating improved target saturation with every-4-week maintenance dosing. Maintenance therapy with lenalidomide may be continued beyond Cycle 32 in both arms, per local standard of care.

In the subsequent randomized Phase 2 portion of the study, approximately 200 patients were randomized and received treatment with RVd, induction and consolidation, ASCT and maintenance therapy with lenalidomide; or DARZALEX and RVd, ASCT and maintenance therapy with DARZALEX and lenalidomide.

About DARZALEX Janssen is committed to exploring the potential of DARZALEX (daratumumab) for patients with multiple myeloma across the spectrum of the disease. DARZALEX has been approved in eight indications, three of which are in the frontline setting, including newly diagnosed patients who are transplant eligible and ineligible.

DARZALEX has become a backbone therapy in the treatment of multiple myeloma, having been used in the treatment of more than 150,000 patients worldwide and more than 68,000 patients in the U.S. alone since its U.S. FDA approval in 2015. DARZALEX is the first CD38-directed antibody approved globally to treat multiple myeloma.

CD38 is a surface protein that is present in high numbers on multiple myeloma cells, regardless of the stage of disease.3 DARZALEX binds to CD38 and inhibits tumor cell growth causing myeloma cell death.4 DARZALEX may also have an effect on normal cells.5 Data across eight Phase 3 clinical trials, in both the frontline and relapsed settings, have shown that DARZALEX-based regimens resulted in significant improvement in progression-free survival and/or overall survival.5,6,7,8,9,10,11,12

About Multiple Myeloma Multiple myeloma is an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.13,14When damaged, these plasma cells rapidly spread and replace normal cells with tumors in the bone marrow. In 2020, it is estimated that more than 32,000 people will be diagnosed and close to 13,000 will die from the disease in the U.S.15 While some patients with multiple myeloma have no symptoms, most patients are diagnosed due to symptoms, which can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems or infections.15

DARZALEXINDICATIONS

DARZALEX(daratumumab) is indicated for the treatment of adult patients with multiple myeloma:

DARZALEXIMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

DARZALEX is contraindicated in patients with a history of severe hypersensitivity (eg, anaphylactic reactions) to daratumumab or any of the components of the formulation.

WARNINGS AND PRECAUTIONS

Infusion-Related Reactions

DARZALEX can cause severe and/or serious infusion-related reactions including anaphylactic reactions. In clinical trials (monotherapy and combination: N=2066), infusion-related reactions occurred in 37% of patients with the Week1 (16mg/kg) infusion, 2% with the Week2 infusion, and cumulatively 6% with subsequent infusions. Less than 1% of patients had a Grade3/4 infusion-related reaction at Week 2 or subsequent infusions. The median time to onset was 1.5hours (range: 0 to 73hours). Nearly all reactions occurred during infusion or within 4hours of completing DARZALEX. Severe reactions have occurred, including bronchospasm, hypoxia, dyspnea, hypertension, laryngeal edema, and pulmonary edema. Signs and symptoms may include respiratory symptoms, such as nasal congestion, cough, throat irritation, as well as chills, vomiting, and nausea. Less common symptoms were wheezing, allergic rhinitis, pyrexia, chest discomfort, pruritus, and hypotension.

When DARZALEX dosing was interrupted in the setting of ASCT (CASSIOPEIA) for a median of 3.75months (range: 2.4 to 6.9months), upon re-initiation of DARZALEX, the incidence of infusion-related reactions was 11% for the first infusion following ASCT. Infusion-related reactions occurring at re-initiation of DARZALEX following ASCT were consistent in terms of symptoms and severity (Grade 3 or 4: <1%) with those reported in previous studies at Week 2 or subsequent infusions. In EQUULEUS, patients receiving combination treatment (n=97) were administered the first 16mg/kg dose at Week 1 split over two days, ie, 8mg/kg on Day1 and Day2, respectively. The incidence of any grade infusion-related reactions was 42%, with 36% of patients experiencing infusion-related reactions on Day1 of Week1, 4% on Day2 of Week1, and 8% with subsequent infusions.

Pre-medicate patients with antihistamines, antipyretics, and corticosteroids. Frequently monitor patients during the entire infusion. Interrupt DARZALEX infusion for reactions of any severity and institute medical management as needed. Permanently discontinue DARZALEX therapy if an anaphylactic reaction or life-threatening (Grade 4) reaction occurs and institute appropriate emergency care. For patients with Grade 1, 2, or 3 reactions, reduce the infusion rate when re-starting the infusion.

To reduce the risk of delayed infusion-related reactions, administer oral corticosteroids to all patients following DARZALEX infusions. Patients with a history of chronic obstructive pulmonary disease may require additional post-infusion medications to manage respiratory complications. Consider prescribing short- and long-acting bronchodilators and inhaled corticosteroids for patients with chronic obstructive pulmonary disease.

Interference With Serological Testing

Daratumumab binds to CD38 on red blood cells (RBCs) and results in a positive Indirect Antiglobulin Test (Indirect Coombs test). Daratumumab-mediated positive Indirect Antiglobulin Test may persist for up to 6months after the last daratumumab infusion. Daratumumab bound to RBCs masks detection of antibodies to minor antigens in the patient's serum. The determination of a patient's ABO and Rh blood type is not impacted. Notify blood transfusion centers of this interference with serological testing and inform blood banks that a patient has received DARZALEX. Type and screen patients prior to starting DARZALEX.

Neutropenia and Thrombocytopenia

DARZALEX may increase neutropenia and thrombocytopenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer's prescribing information for background therapies. Monitor patients with neutropenia for signs of infection. Consider withholding DARZALEX until recovery of neutrophils or for recovery of platelets.

Interference With Determination of Complete Response

Daratumumab is a human IgG kappa monoclonal antibody that can be detected on both the serum protein electrophoresis (SPE) and immunofixation (IFE) assays used for the clinical monitoring of endogenous M-protein. This interference can impact the determination of complete response and of disease progression in some patients with IgG kappa myeloma protein.

Embryo-Fetal Toxicity

Based on the mechanism of action, DARZALEX can cause fetal harm when administered to a pregnant woman. DARZALEX may cause depletion of fetal immune cells and decreased bone density. Advise pregnant women of the potential risk to a fetus. Advise females with reproductive potential to use effective contraception during treatment with DARZALEX and for 3 months after the last dose.

The combination of DARZALEX with lenalidomide, pomalidomide, or thalidomide is contraindicated in pregnant women, because lenalidomide, pomalidomide, and thalidomide may cause birth defects and death of the unborn child. Refer to the lenalidomide, pomalidomide, or thalidomide prescribing information on use during pregnancy.

ADVERSE REACTIONS

The most frequently reported adverse reactions (incidence 20%) were: upper respiratory infection, neutropenia, infusionrelated reactions, thrombocytopenia, diarrhea, constipation, anemia, peripheral sensory neuropathy, fatigue, peripheral edema, nausea, cough, pyrexia, dyspnea, and asthenia. The most common hematologic laboratory abnormalities (40%) with DARZALEX are: neutropenia, lymphopenia, thrombocytopenia, leukopenia, and anemia.

Please click hereto see the full Prescribing Information.

DARZALEX FASPRO INDICATIONS DARZALEXFASPRO is indicated for the treatment of adult patients with multiple myeloma:

DARZALEX FASPROIMPORTANT SAFETY INFORMATION CONTRAINDICATIONS

DARZALEX FASPRO(daratumumab and hyaluronidase-fihi) is contraindicated in patients with a history of severe hypersensitivity to daratumumab, hyaluronidase or any of the components of the formulation.

WARNINGS AND PRECAUTIONS

Hypersensitivity and Other Administration Reactions

Both systemic administration-related reactions, including severe or life-threatening reactions, and local injection-site reactions can occur with DARZALEX FASPRO.

Systemic Reactions

In a pooled safety population of 490patients who received DARZALEX FASPROas monotherapy or in combination, 11% of patients experienced a systemic administration-related reaction (Grade 2: 3.9%, Grade 3: 1.4%). Systemic administration-related reactions occurred in 10% of patients with the first injection, 0.2% with the second injection, and cumulatively 0.8% with subsequent injections. The median time to onset was 3.7hours (range: 9minutes to 3.5days). Of the 84systemic administration-related reactions that occurred in 52patients, 73(87%) occurred on the day of DARZALEX FASPROadministration. Delayed systemic administration-related reactions have occurred in less than 1% of the patients.

Severe reactions included hypoxia, dyspnea, hypertension and tachycardia. Other signs and symptoms of systemic administration-related reactions may include respiratory symptoms, such as bronchospasm, nasal congestion, cough, throat irritation, allergic rhinitis, and wheezing, as well as anaphylactic reaction, pyrexia, chest pain, pruritis, chills, vomiting, nausea, and hypotension.

Pre-medicate patients with histamine-1 receptor antagonist, acetaminophen and corticosteroids. Monitor patients for systemic administration-related reactions, especially following the first and second injections. For anaphylactic reaction or life-threatening (Grade 4) administration-related reactions, immediately and permanently discontinue DARZALEX FASPRO.Consider administering corticosteroids and other medications after the administration of DARZALEX FASPROdepending on dosing regimen and medical history to minimize the risk of delayed (defined as occurring the day after administration) systemic administration-related reactions.

Local Reactions

In this pooled safety population, injection-site reactions occurred in 8% of patients, including Grade2 reactions in 0.6%. The most frequent (>1%) injection-site reaction was injection site erythema. These local reactions occurred a median of 7minutes (range: 0minutes to 4.7days) after starting administration of DARZALEX FASPRO. Monitor for local reactions and consider symptomatic management.

Neutropenia Daratumumab may increase neutropenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer's prescribing information for background therapies. Monitor patients with neutropenia for signs of infection. Consider withholding DARZALEX FASPROuntil recovery of neutrophils. In lower body weight patients receiving DARZALEX FASPROhigher rates of Grade 3-4 neutropenia were observed.

Thrombocytopenia Daratumumab may increase thrombocytopenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer's prescribing information for background therapies. Consider withholding DARZALEX FASPROuntil recovery of platelets.

Embryo-Fetal Toxicity Based on the mechanism of action, DARZALEX FASPROcan cause fetal harm when administered to a pregnant woman. DARZALEX FASPROmay cause depletion of fetal immune cells and decreased bone density. Advise pregnant women of the potential risk to a fetus. Advise females with reproductive potential to use effective contraception during treatment with DARZALEX FASPROand for 3months after the last dose.

The combination of DARZALEX FASPROwith lenalidomide is contraindicated in pregnant women, because lenalidomide may cause birth defects and death of the unborn child. Refer to the lenalidomide prescribing information on use during pregnancy.

Interference with Serological Testing Daratumumab binds to CD38 on red blood cells (RBCs) and results in a positive Indirect Antiglobulin Test (Indirect Coombs test). Daratumumab-mediated positive indirect antiglobulin test may persist for up to 6months after the last daratumumab administration. Daratumumab bound to RBCs masks detection of antibodies to minor antigens in the patient's serum. The determination of a patient's ABO and Rh blood type are not impacted.

Notify blood transfusion centers of this interference with serological testing and inform blood banks that a patient has received DARZALEX FASPRO.Type and screen patients prior to starting DARZALEX FASPRO.

Interference with Determination of Complete Response

Daratumumab is a human IgG kappa monoclonal antibody that can be detected on both the serum protein electrophoresis (SPE) and immunofixation (IFE) assays used for the clinical monitoring of endogenous M-protein. This interference can impact the determination of complete response and of disease progression in some DARZALEX FASPROtreated patients with IgG kappa myeloma protein.

ADVERSE REACTIONS The most common adverse reaction (20%) with DARZALEX FASPROmonotherapy is: upper respiratory tract infection. The most common adverse reactions with combination therapy (20% for any combination) include fatigue, nausea, diarrhea, dyspnea, insomnia, pyrexia, cough, muscle spasms, back pain, vomiting, upper respiratory tract infection, peripheral sensory neuropathy, constipation, and pneumonia.

The most common hematology laboratory abnormalities (40%) with DARZALEX FASPROare decreased leukocytes, decreased lymphocytes, decreased neutrophils, decreased platelets, and decreased hemoglobin.

Please see full Prescribing Information atwww.DARZALEX.com.

About the Janssen Pharmaceutical Companies of Johnson & Johnson At Janssen, we're creating a future where disease is a thing of the past. We're the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular & Metabolism, Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and Pulmonary Hypertension.

Learn more at http://www.janssen.com. Follow us at http://www.twitter.com/JanssenGlobal and http://www.twitter.com/JanssenUS. Janssen Research & Development, LLC, Janssen Scientific Affairs, LLC and Janssen Biotech, Inc. are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.

Cautions Concerning Forward-Looking Statements This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding DARZALEX. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Biotech, Inc., Janssen Research & Development, LLC, or any of the other Janssen Pharmaceutical Companies, and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended December 29, 2019, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in the company's most recently filed Quarterly Report on Form 10-Q, and the company's subsequent filings with the Securities and Exchange Commission. Copies of these filings are available online at http://www.sec.gov, http://www.jnj.comor on request from Johnson & Johnson. None of the Janssen Pharmaceutical Companies nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.

1 Kaufman, JL et al. Daratumumab (DARA) Plus Lenalidomide, Bortezomib, and Dexamethasone (RVd) in Patients with Transplant-eligible Newly Diagnosed Multiple Myeloma (NDMM): Updated Analysis of GRIFFIN After 12 Months of Maintenance Therapy. Abstract #549. To be presented at 2020 American Society of Hematology Annual Meeting. 2Voorhees, PM et al. Daratumumab (DARA) Plus Lenalidomide, Bortezomib, and Dexamethasone (RVd) in Patients with Transplant-eligible Newly Diagnosed Multiple Myeloma (NDMM): Updated Efficacy and Safety Analysis of the Safety Run-in Population of GRIFFIN. Abstract #3243. To be presented at 2020 American Society of Hematology Annual Meeting. 3Janssen Research & Development, LLC. Study Comparing Daratumumab, Lenalidomide, Bortezomib, and Dexamethasone (D-RVd) Versus Lenalidomide, Bortezomib, and Dexamethasone (RVd) in Subjects With Newly Diagnosed Multiple Myeloma In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2016 August 22]. Available at: https://clinicaltrials.gov/ct2/show/NCT02874742 Identifier: NCT02874742. 4Fedele G et al. CD38 Ligation in Peripheral Blood Mononuclear Cells of Myeloma Patients Induces Release of Protumorigenic IL-6 and Impaired Secretion of IFN Cytokines and Proliferation. Mediators Inflamm. 2013;564687. 5Janssen Research & Development, LLC. A Study Comparing Daratumumab, Lenalidomide, and Dexamethasone With Lenalidomide and Dexamethasone in Relapsed or Refractory Multiple Myeloma. In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT02076009?term=mmy3003&rank=1 Identifier: NCT02136134. 6Janssen Research & Development, LLC. Addition of Daratumumab to Combination of Bortezomib and Dexamethasone in Participants With Relapsed or Refractory Multiple Myeloma. In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT02136134?term=mmy3004&rank=1 Identifier: NCT02076009. 7Janssen Research & Development, LLC. A Study to Evaluate Daratumumab in Transplant Eligible Participants With Previously Untreated Multiple Myeloma (Cassiopeia). In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT02541383?term=mmy3006 Identifier: NCT02541383. 8Janssen Research & Development, LLC. A Study of Combination of Daratumumab and Velcade (Bortezomib) Melphalan-Prednisone (DVMP) Compared to Velcade Melphalan-Prednisone (VMP) in Participants With Previously Untreated Multiple Myeloma In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT02195479?term=mmy3007&rank=1 Identifier: NCT02195479. 9 Janssen Research & Development, LLC. Study Comparing Daratumumab, Lenalidomide, and Dexamethasone With Lenalidomide and Dexamethasone in Participants With Previously Untreated Multiple Myeloma. In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT02252172?term=mmy3008&rank=1 Identifier: NCT02252172. 10Janssen Research & Development, LLC. A Study of VELCADE (Bortezomib) Melphalan-Prednisone (VMP) Compared to Daratumumab in Combination With VMP (D-VMP), in Participants With Previously Untreated Multiple Myeloma Who Are Ineligible for High-Dose Therapy (Asia Pacific Region). In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT03217812?term=MMY3011&rank=1 Identifier: NCT03217812. 11European Myeloma Network. Compare Progression Free Survival Btw Daratumumab/Pomalidomide/Dexamethasone vs Pomalidomide/Dexamethasone (EMN14). In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24] Available at: https://clinicaltrials.gov/ct2/show/NCT03180736?term=MMY3013&rank=2 Identifier: NCT03180736. 12Amgen. Study of Carfilzomib, Daratumumab and Dexamethasone for Patients With Relapsed and/or Refractory Multiple Myeloma. (CANDOR). In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24] Available at: https://clinicaltrials.gov/ct2/show/NCT03158688?term=NCT03158688&rank=1 Identifier: NCT03158688. 13Kumar, SK et al. Risk of progression and survival in multiple myeloma relapsing after therapy with IMiDs and bortezomib: a multicenter international myeloma working group study. Leukemia. 2012 Jan; 26(1):149-57. 14American Cancer Society. "What Is Multiple Myeloma?" Available at: http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-what-is-multiple-myeloma. Accessed June 2019. 15American Cancer Society. "Key Statistics About Multiple Myeloma." Available at: https://www.cancer.org/cancer/multiple-myeloma/about/key-statistics.html. Accessed January 2020.

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New DARZALEX (daratumumab) Data from GRIFFIN Study Show Deeper and Longer Responses in Patients with Newly Diagnosed Multiple Myeloma - BioSpace

Anatomy of a vaccine: What it takes to create a safe, effective COVID shot – University of California

Shawn stepped into the UCLA Vine Street Clinic in Hollywood with confidence. He offered up his arm. The UCLA doctor injected him. It took seconds; there was barely a sting.

Twenty-four hours after the first of two shots, given 28 days apart, he suffered the headaches and fatigue associated with a milder case of COVID-19. But Shawn remained calm, resolved to honor the memory of his mother, a nurse who had died in May 2020 from an unrelated cause.

The 57-year-old nonprofit worker had been thinking about the challenges of COVID-19 for a long time, and he decided to go through the lengthy consent process for the medical trial. It gave me something to do with my anger that was so much better than yelling at someone for not wearing a mask, he says. And [at UCLA] I felt I was in good hands.

Shawn is one of many volunteers who have stepped up to participate in medical trials at UCLA, which is part of a global network thats determined to help find a vaccine against the novel coronavirus.

The stakes are huge. More than 250,000 Americans have already died, and there have been more than 1 million deaths around the world. Economies have been brought to their knees, social tensions have disrupted communities and emotional maladies are on the rise.

In response, doctors and scientists have been challenged to be resilient and ingenious. Theyre taking an array of different approaches, knowing that public confidence in vaccines hangs in the balance.

In addition, it has been a challenge to create a vaccine in such a short amount of time similar efforts have taken five to 10 years. Pharmaceutical giant Pfizer and biotech firm Moderna have both reported remarkable progress, announcing in November that their vaccine candidates were more than 90% effective. All of which has raised questions about the next steps, such as how the vaccines will be distributed.

I dont want to make a vaccine to protect against mild disease, says Dr. Marcus Horwitz, distinguished professor of medicine and microbiology, immunology and molecular genetics at the David Geffen School of Medicine at UCLA. I want to protect people who are going to get severe disease.

Horwitz has already developed vaccines against the bacteria behind tuberculosis, anthrax and the tick-borne disease tularemia, but he has never tried to create a vaccine against a virus. When faced with a worldwide pandemic, we thought we might be able to make a contribution, he says.

Vaccines work by training the immune system to recognize and fight disease-causing pathogens, such as viruses or bacteria. Doctors introduce the bodys immune system to antigens, which are molecules from the virus or bacteria, and the immune system responds by making proteins called antibodies and immunity-building T cells, which both neutralize the pathogen.

The delivery of these antigens requires a delicate calculus: It must provoke the immune system, but not go so far as to make the patient ill. You need a vector that will wake up the immune system of the host, but not cause any further harm, Horwitz says.

The vaccine approach by Horwitz and his team, including lead investigator Qingmei Jia, is a medical outlier: They adapted an existing antibacterial platform to build protection against SARS-CoV-2, the virus that causes COVID-19. The team has shown that their vaccine candidate protects hamsters, which develop severe disease in a way similar to humans.

Some of the potential vaccines for SARS-CoV-2 use a weakened form of an adenovirus, which causes the common cold, to deliver the S protein that is found on the surface of the SARS-CoV-2 virus. Horwitzs vaccine stands out from the pack because it uses a weakened bacterium to deliver two SARS-CoV-2 proteins, the M and N proteins.

That difference could have a tremendous impact. Billions of COVID-19 vaccine doses are needed, and bacteria, unlike viruses, are easy and cheap to produce and transportable.

The success of a COVID-19 vaccine also depends on the immune system, which can be less robust in older people.

This is a problem that has driven Song Li, chair of the bioengineering department at the UCLA Samueli School of Engineering, who has focused his career on cell and tissue engineering. Adapting a concept from cancer immunotherapy, Li is developing a biomaterial vaccine booster using artificial cells that could improve the immune systems ability to generate long-term protection.

When the immune system encounters a destructive pathogen, it produces cells that are designed to attack the invader. A small number of those cells, called T memory stem cells, can stay in the system for years ready for a future invasion. Unfortunately, our ability to produce T memory stem cells declines as we get older. Li hopes his booster, in combination with a vaccine, can help fragile immune systems effectively fight against the SARS-CoV-2 virus.

My goal at the outset was to help the elderly population, Li says. But it could be useful for any person whose immune system needs help generating protection from the virus.

Another UCLA team led by Bogdan Pasaniuc, Dr. Manish Butte and Dr. Daniel Geschwind, the Gordon and Virginia MacDonald Distinguished Professor of Human Genetics at the Geffen School of Medicine is trying to find out why the virus significantly impacts some, but leaves others relatively unscathed.

We know age is a major factor, but we see older people who get infected and do quite well, Geschwind says. We have a limited ability to predict how sick someone will get. His team hopes that studying whole-genome sequences from thousands of COVID-19 patients will reveal hidden factors that make some more vulnerable than others. The research could help identify people who are at higher risk for infection as well as develop new treatment and prevention strategies.

Dr. Brigitte Gomperts, professor of pediatrics and pulmonary medicine and a member of the UCLA Broad Stem Cell Research Center, is studying how COVID-19 affects lung tissue. By using stem cellderived clusters of lung cells, known as organoids, she can rapidly screen thousands of prospective treatments. Because the organoids are grown from human cells and reflect the cell types and architecture of the lungs, they can offer insights into how the virus infects and damages the organ.

At UCLA medical centers around Los Angeles County, physicians are ensuring that their medical trials include diverse groups of people and women of all ages.

COVID-19 has hit the African American and Latino communities particularly hard, says Dr. Jesse Clark, associate professor-in-residence in the department of medicine at the Geffen School of Medicine. We have to make sure that any vaccine has been determined to be safe and effective in all populations that will receive it.

COVID-19 has hit the African American and Latino communities particularly hard. We have to make sure that any vaccine has been determined to be safe and effective in all populations that will receive it.

Dr. Jesse Clark, associate professor-in-residence in the department of medicine at the David Geffen School of Medicine at UCLA

Clark is medical director of the UCLA Vine Street Clinic, which is involved in the Moderna clinical trial. Notably, Modernas vaccine works differently from a typical vaccine, because it doesnt contain the virus at all. Instead, it uses messenger RNA, or mRNA, which uses the bodys genetic code to produce antibodies against the virus.

CNN mentioned that the vaccine trials were having trouble finding minorities to participate, says Roderick, a 37-year-old IT manager and father of two, who is participating in the Moderna trial. Being Black and Mexican, and knowing how hard my demographic has been hit, I just went ahead and signed up online. Its worth doing to help out.

Meanwhile, Dr. Katya Corado, an infectious disease specialist at Harbor-UCLA Medical Center in Torrance, has been enrolling patients in a phase 3 clinical trial of an adenovirus vector vaccine thats under development by the University of Oxford and the biopharmaceutical company AstraZeneca.

All vaccines undergo three phases of clinical trials, according to rules set by the Food and Drug Administration. Phase 1, which involves 20 to 100 volunteers, tests the safety and dosage of the vaccine. Phase 2 tests the drugs efficacy and side effects among several hundred participants, and phase 3 gathers more information about a vaccines safety and effectiveness by studying thousands of volunteers.

In the phase 3 trial, we focus on studying how effective the vaccine is in populations that need it most, Corado says.

Clark and Corado are both hopeful that their work can protect the most vulnerable, which includes people over 65, patients with chronic conditions, those facing economic disadvantages and essential workers.

Inoculations have eradicated past epidemics, such as smallpox. But public faith in vaccines has wavered, especially when a now-disproven report in 1998 suggested that the measles, mumps and rubella vaccine was linked to autism spectrum disorder. That has led to U.S. outbreaks of measles, which had been previously eliminated. So scientists recognize the importance of getting the COVID-19 vaccine right.

There are other factors to consider as well. Vaccine distribution will be high on the agenda of the incoming White House administration, but if supply is limited, the Centers for Disease Control and Prevention recommends prioritizing certain groups, such as medical workers.

Also, some vaccines currently in development need to be stored in ultra-cold conditions. For example, Pfizers vaccine must be stored at minus 70 degrees Celsius, while Modernas vaccine must be kept at minus 20 degrees Celsius the temperature of a regular freezer. These factors will affect how the vaccines are distributed.

Some lawmakers have advocated letting the virus run its course in the hopes of achieving herd immunity, which is when enough people have become immune to an infectious disease, either through being infected or vaccination. Since the COVID-19 vaccine is still pending, a majority of people will need to be infected in order to achieve herd immunity and that comes at a terrible cost.

According to Dr. Robert Kim-Farley, professor-in-residence of epidemiology at the UCLA Fielding School of Public Health, up to 2 million Americans would have to die before the country reached herd immunity.

He argues that vaccines work, even if they are not perfectly safe or perfectly effective, as proven by the near-eradication of polio. But approving vaccines prematurely to buckle under the pressure of politics or profit could cause a terrible backlash against being vaccinated, which could lead to future outbreaks.

We want to make sure we are not cutting corners, Kim-Farley says, that we are getting the best vaccine that has the highest efficacy, the longest duration, the fewest number of side effects [with] the fewest number of doses.

This is a very high-stakes game, and its important to get it right, without recalls or playing into the [anti-vaccination] narrative. What still concerns me is the equitable distribution of vaccines to make sure that countries that are not as wealthy as us have access to these life-saving vaccines. We are all members of one global community.

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Anatomy of a vaccine: What it takes to create a safe, effective COVID shot - University of California