Category Archives: Stem Cell Treatments

MATFORS, Sweden, June 21, 2022 (GLOBE NEWSWIRE) -- Sweden-based NorthX Biologics (NorthX) is expanding into cell therapy manufacturing at its existing GMP-facility, as well as in premises at the Karolinska University Hospital campus in Stockholm. This initiative is part of NorthXs Innovation Hub, an Innovation Track designed to provide development and GMP-manufacturing services to the next generation of drug development companies and innovative research groups in need of NorthXs Good Manufacturing Practice (GMP) expertise.

NorthX has one of Northern Europes largest clinical-grade manufacturing capacities for plasmid DNA, recombinant proteins, cell banking and associated gene therapy services and this expansion into cell therapy is a major step to complete our offering for innovative clients. We are especially excited to work with Dr. Kristian Tryggvason, a leader in cell therapy technologies, and his team, said Dr. Ted Fjllman, CEO of NorthX.

After having built up BioLamina and its cell culture reagents that are used worldwide both in academia and industry, Dr. Tryggvason recently launched his latest venture: Alder Therapeutics. In addition to its own product development, the company now entered into an agreement to help NorthX expand its cell culture services to many new different cell types, including pluripotent stem cells. The Alder team will also help to design and validate NorthXs new process development and GMP-manufacturing labs in Matfors, alongside those being established at the Karolinska University Hospital campus in Stockholm.

Our goal is to be able to offer synergies to both cell and gene therapy clients and to collaborate with them through our Innovation Track, in which we work hand in hand with our clients regarding process development, manufacturing, and analytics to progress clinical programs and bring life-saving treatments to patients, added Aaron Small, NorthX VP of Global Sales and Corporate Development

Universities and cell therapy companies worldwide need GMP-grade development and manufacturing capacity, as it is complex and outside the scope of most biotech companies to build themselves. NorthX Biologics is already helping to translate cutting-edge gene therapy research into clinical development and now we will together build upon the existing broad cell therapy know-how in Sweden to do the same for cell therapies, said Dr. Kristian Tryggvason, CEO Alder Therapeutics.

About NorthX Biologics:

NorthX Biologics provides process development and manufacturing services with expertise in plasmids, proteins and other advanced biologics. NorthX Biologics sits in the heart of Sweden, and the team has been manufacturing biologics to GMP since 1988. In 2021 NorthX was recognized as a national innovation hub for advanced therapeutics and vaccines. NorthX has the ambition to become a leading cell and gene therapy manufacturer and partner of choice for innovative drug development companies. For more information see http://www.nxbio.com

About Alder Therapeutics:

Headquartered inStockholm, Alder Therapeutics AB is a novel development stage, cell therapy platform company aiming to develop and manufacture the best functional cell therapy products based on the most simple and robust processes. Alder Therapeutics unique cell therapy platform will allow manufacturing of better cells at a lower cost, which will make pluripotent cell therapy treatment available for ever more patients. Alder Therapeutics will play an important role in opening the next era in medicinal treatment. For more information and important updates, please visit. http://www.aldertx.com

For further information please email NorthX at:contact@nxbio.com

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NorthX Biologics expands to Cell Therapy: Partnership with Alder Therapeutics and new manufacturing site on Karolinska campus - GlobeNewswire

Sickle cell beta-thalassemia refers to an inherited condition that impacts hemoglobin. People with the condition have different changes in each copy of their hemoglobin gene. One causes red blood cells (RBCs) to form a sickle shape and another reduces the amount of hemoglobin.

Sickle cell beta-thalassemia is a type of RBC disorder known as a hemoglobinopathy. These are conditions that cause abnormal hemoglobin production or a change in its structure. Hemoglobin is the protein in RBCs responsible for carrying oxygen around the body.

Both sickle cell disease and beta-thalassemia are genetic conditions that affect hemoglobin. A person with sickle cell beta-thalassemia inherits a trait for both conditions, impacting the shape and number of hemoglobin.

In this article, we will discuss the causes and risk factors for sickle cell beta-thalassemia, as well as symptoms and treatment options for the condition.

Sickle cell beta-thalassemia is a genetic condition and a type of sickle cell disease that features symptoms of both sickle cell disease and beta-thalassemia. It causes RBCs to take on a sickle shape, making them unable to flow through the blood vessels as smoothly. It also affects the amount of normal hemoglobin a person has in their blood.

It is an inherited condition, meaning that parents pass it on to their children. Researchers estimate that 57% of the global population are carriers of a significant hemoglobin mutation. People with sickle cell beta-thalassemia inherit a sickle trait from one parent and a beta-thalassemia trait from the other.

There are two types of sickle cell beta-thalassemia: plus (HbS beta+) and zero (HbS beta0). The former is the milder variant. The plus indicates that the blood contains a lower-than-average amount of normal hemoglobin. This differs from the latter, in which a person has no normal hemoglobin.

Sickle cell beta-thalassemia results from a change in the beta-hemoglobin (HBB) gene. The beta-hemoglobin gene is responsible for forming the hemoglobin subunit beta component of the hemoglobin protein.

A person develops sickle cell beta-thalassemia when they inherit one sickle cell trait from one parent and one beta-thalassemia trait from the other. The beta-thalassemia gene can be either beta+, which results in a lower production of normal hemoglobin, or beta0, which leads to a complete absence of normal hemoglobin.

Risk factors for a person developing sickle cell beta-thalassemia include having parents that may be carriers of the sickle cell, HbS beta+, or HbS beta0 gene. The condition follows an autosomal recessive inheritance pattern. This means that if a person has a beta-thalassemia trait and their partner has a sickle cell trait, there is a 25% chance with each pregnancy that their child will have sickle cell beta-thalassemia.

The symptoms a person may experience will depend on whether they have HbS beta+ or HbS beta0. The amount of normal hemoglobin a person can produce largely determines the severity of the condition.

A person with HbS beta+ will likely have milder symptoms, as while they produce less functioning hemoglobin than is typical, they can still produce normal hemoglobin. Individuals with either type will produce sickle-shaped RBCs that can block circulation and cause cell damage and pain.

A person with HbS beta+ may experience:

The symptoms of HbS beta0 are typically similar but with a more severe case of anemia.

Some newborn screenings include testing for sickle cell disorders such as sickle cell beta-thalassemia. Screening involves taking a blood sample and looking at the types of hemoglobin present in the newborns blood. However, newborn screenings only suggest the possibility of sickle cell beta-thalassemia and require further confirmatory tests. This may include the parents and any siblings having tests.

These tests may include:

Treatment aims to prevent complications from occurring and treat the symptoms a person may experience. This generally involves continuous care to prevent and manage potential problems.

In addition to the treatments below, a person may receive pain relief medications and antibiotics to help reduce pain and infections. The spleen usually helps prevent infections, but many people with sickle cell beta-thalassemia may lose spleen function. As such, children may receive penicillin prophylaxis to prevent pneumococcal sepsis.

Treatment options for sickle cell beta-thalassemia may include:

People may require hydroxyurea if they experience frequent periods of pain. Hydroxyurea is a drug that makes RBCs bigger and changes their shape to the typical round and flexible composition. This can help slow or prevent complications.

Hydroxyurea increases the level of fetal hemoglobin (HbF) in the body. HbF is present in higher quantities in newborns and can help protect against sickle cell complications. With higher levels of HbF, RBCs are less likely to become sickle-shaped.

According to the American Society of Hematology, people with sickle cell beta-thalassemia who take hydroxyurea also have fewer:

Some people with sickle cell beta-thalassemia may require blood transfusions. This is when a healthcare professional infuses healthy donor blood into the body of a person with sickle cell beta-thalassemia via a tube. The donor blood will need to have matching antigens to the blood of the person receiving the transfusion. These antigens, known as human leukocyte antigens, are proteins present on the surface of RBCs.

If the antigens do not match, the immune system of the person receiving the blood donation is more likely to reject the transfusion, and it may lead to a reaction that can cause health problems.

The bone marrow in the body produces blood cells. A person with dysfunctional bone marrow, such as in sickle cell beta-thalassemia, may receive hematopoietic stem cells from a healthy donor. This may help improve bone marrow function and reduce the symptoms of sickle cell beta-thalassemia. A hematopoietic stem cell transplant is currently the only cure for this condition.

Sickle cell beta-thalassemia is a type of sickle cell disease. Some evidence suggests the life expectancy of a person living with sickle cell disease is reduced by 2030 years compared with a healthy individual. Similarly, a 2019 study suggests a person with sickle cell disease may live roughly 22 fewer years than a person without the condition.

Advances in therapy and treatment options are helping to improve the outlook of people with sickle cell disease. However, statistics like these highlight the necessity to further develop approaches to improve the underlying morbidity and mortality of individuals with the condition.

Most often, a person will receive a diagnosis of sickle cell beta-thalassemia shortly after birth. Later in life, if they believe their current treatment regime is not suitable or notice worsening symptoms, they should contact their doctor.

If a person is aware of a family history of sickle cell disease, they may wish to consider undergoing genetic screening before attempting to have children.

Sickle cell beta-thalassemia is a type of sickle cell disease. It occurs when a person inherits a sickle cell trait and a beta-thalassemia trait from their parents. It results in a person having sickle-shaped RBCs and either producing a low amount of hemoglobin or none at all.

Symptoms of the condition can include mild to severe anemia, tiredness, weakness, pain, and possible organ damage. Treatment may involve pain relief medications, antibiotics, hydroxyurea, and blood transfusions. Some people may also receive a bone marrow transplant to help them produce healthy hemoglobin.

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Sickle cell beta thalassemia: Causes, symptoms, and treatments - Medical News Today

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SEATTLE, June 16, 2022 (GLOBE NEWSWIRE) -- Umoja Biopharma, Inc., an immuno-oncology company pioneering off-the-shelf, integrated therapeutics that reprogram immune cells in vivo to treat patients with solid and hematologic malignancies, announced today that it will have a poster presentation at the 2022 International Society for Stem Cell Research (ISSCR) Annual Meeting, to be held June 15-18, 2022 in San Francisco, California.

On Wednesday, June 15th, Principal Scientist & iPSC Team Lead, Teisha Rowland, Ph.D., will give a poster presentation titled, A Synthetic Cytokine Receptor Platform for Producing Cytotoxic Innate Lymphocytes as Off-the-Shelf Cancer Therapeutics. The presentation will discuss Umojas engineered induced pluripotent stem cell (iPSC) platform, that incorporates the synthetic cytokine receptor system rapamycin-activated cytokine receptor (RACR) platform. Umojas engineered iPSCs that are modified to express RACR, called RACR-induced cytotoxic innate lymphoid (iCIL) cells, drive differentiation and expansion of the cells while eliminating the need for expensive cytokines and other raw materials. The RACR platform has the potential to enable cytokine-free manufacturing and engraftment of the engineered cells in the patient without the need for toxic lymphodepletion.

Despite the advances chimeric antigen receptor T cell therapies have provided to the oncology space, we continue to battle significant challenges that these therapies cannot address, like limited expansion capacity and scalability, manufacturing complexity, variability among patients, and the need for toxic chemotherapy administration to combat patients anti-allograft response, said Andy Scharenberg, M.D., co-founder and Chief Executive Officer of Umoja. We are developing an engineered iPSC platform, including the RACR platform, to address these challenges by enabling a scalable, virtually unlimited, and simplified manufacturing of engineered, cancer-fighting cytotoxic innate lymphocytes.

About Umoja Biopharma

Umoja Biopharma, Inc. is an early clinical-stage company advancing an entirely new approach to immunotherapy. Umoja Biopharma, Inc. is a transformative multi-platform immuno-oncology company founded with the goal of creating curative treatments for solid and hematological malignancies by reprogramming immune cells in vivo to target and fight cancer. Founded based on pioneering work performed at Seattle Childrens Research Institute and Purdue University, Umojas novel approach is powered by integrated cellular immunotherapy technologies including the VivoVec in vivo delivery platform, the RACR/CAR in vivo cell expansion/control platform, and the TumorTag targeting platform. Designed from the ground up to work together, these platforms are being developed to create and harness a powerful immune response in the body to directly, safely, and controllably attack cancer. Umoja believes that its approach can provide broader access to the most advanced immunotherapies and enable more patients to live better, fuller lives. To learn more, visit http://umoja-biopharma.com/.

About RACR

CAR T cells generated by the body with VivoVec can be expanded and sustained with the rapamycin activated cytokine receptor (RACR) system, an engineered signaling system designed to improve chimeric antigen receptor (CAR) T cell persistence and produce durable anti-tumor responses. The RACR/CAR payload is integrated into the genomic DNA of a patients T cells. Rapamycin activates the RACR system resulting in preferential expansion and survival of cancer-fighting T cells. The RACR technology enables a patients cells to expand in a manner that resembles a natural immune response that does not require lymphodepletion, promoting durable T cell engraftment. RACR/CAR technology can also be used to enhanceex vivomanufacturing in support of more traditional autologous or allogeneic cell therapy manufacturing processes. To learn more about Umojas RACR platform please visit https://www.umoja-biopharma.com/platforms/

Media Contact:Darren Opland, Ph.D.LifeSci Communications[emailprotected]

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Umoja Biopharma Presents Data on its Engineered Induced Pluripotent Stem Cell Platform at the 2022 International Society for Stem Cell Research Annual...

Stem cell treatment is an alternative medical approach for various health conditions. It involves using stem cells to prevent, treat, or manage different diseases. But before you consider stem cell treatment as a therapy option, here are six things to know about this approach.

Different types of stem cells have their own purposes

There are four types of stem cells, and each has a unique application in regenerative medicine. Embryonic stem cells and pluripotent stem cells, for example, are commonly used not for treatment purposes but for studying disease processes and testing new drugs. In contrast, tissue-specific and mesenchymal stem cells are adopted for therapy use.

Tissue-specific stem cells are known to differentiate into cells of the certain tissues, e.g., hematopoietic stem cells (HSCs) that act as the progenitor of the different blood cells of the body. These stem cells are supposed only to be used to treat health conditions affecting the tissues in which they are found.

Mesenchymal stem cells (MSCs) differ from other stem cells in that it gives rise to their kind of cells and a variety of body cells. They are used in regenerative medicine to treat various medical conditions. Mesenchymal stem cells can be used in stem cell treatment for strokes, spinal cord injury, Crons disease, arthritis, and other conditions. You can visit website for a further list of diseases and for more information.

Stem cells cannot treat multiple diseases at the same time

Although there is stem cell treatment for diabetes, Crohn's disease, fibromyalgia, and several other medical conditions, stem cells cannot be used to treat comorbidities at the same time. For example, you can't use stem cell treatment for diabetes as the same stem cell treatment for knee problems. One stem cell treatment cannot be used to treat two or more unrelated medical conditions due to different routes of administration of a cell-based product, depending on the disease, as well as different treatment programs that may involve additional therapies. Be careful when choosing a clinic for stem cell treatment, and be sure to visit an accredited stem cell therapy clinic that offers various stem cell treatments instead of just one.

An experimental treatment offered for sale is not the same as a clinical trial

Many alternative treatment approaches have not been subjected to clinical trials. So, although a procedure can be an experimental treatment, it doesn't mean it has been researched or placed in clinical trials. When clinical trials are done successfully, it leads to the development of new treatment procedures that conforms to health regulations. Before signing up for any medical treatment, verify that it has passed through clinical trials.

Cells from your own body are not automatically safe when used in treatments

Stem cell treatments procedures involve harvesting some stem cells from a part of your body, manipulating these cells in the lab, and then reintroducing them into your body. Normally, because doctors take these stem cells from your body, there shouldn't be any problem reintroducing them back into your body.

Some scenarios can make them unsafe. If the cells were contaminated before being injected into your body, it could lead to severe microbial infection. If doctors inappropriately manipulate the cells and unintentionally alter their functioning and growth, they could become tumor cells. Be aware of these risks; abstain from clinics that do not lessen the risks associated with stem cell treatments.

Patient testimonials and other marketing provided by clinics may be misleading

One of the ways clinics market their services is through patient testimonials. Although patient testimonials are great for reviewing feedback from people who have adopted a particular medical treatment, they can be misleading.

Some clinics promote all the benefits of their treatments while lessening the potential risks involved. Others offer unproven treatments and unreliable patient testimonials to convince people to opt-in for their services. You want to ensure that the treatment is science-based, approved for use after successful trials, and provided by an accredited clinic.

There is something to lose when you try an unproven treatment

Most unproven experimental treatments have no solid scientific evidence detailing their effectiveness and benefits. Subscribing to such unproven treatments places you at risk of complications resulting in short to long-term problems.

Asides from the health risks attached, the costs of these procedures can be enormous and might amount to waste as the procedure might be ineffective. If you believe the potential benefits outweigh the presented risks, discuss with your family and healthcare providers to assess the treatment before properly making your final decision.

Conclusion

Stem cell treatments are proven medical therapy for several health conditions. For managing selected diseases, stem cell-based therapy has been subjected to clinical trials successfully. However, not all clinics are accredited to provide this type of treatment. The best stem cell therapy center offers various treatment programs for different medical conditions, does not treat multiple diseases with the same option, and provides you with adequate information about the benefits and risks of stem cell treatment.

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Six Things to Know About Stem Cell Treatment - HealthTechZone

An existing center on the University of Colorados Anschutz Medical Campus that helps develop treatments based on patients own cells is getting a $200 million boost, with the hope of getting those treatments to the public faster.

Chancellor Don Elliman said the Anschutz campus and the Gates Frontiers Fund will each invest $20 million per year over the next five years to turn the existing Gates Center for Regenerative Medicine into the significantly larger Gates Institute.

The Gates Frontiers Fund is affiliated with the Gates Family Foundation, a Colorado-based nonprofit,and is not connected to Bill Gates foundation.

The fund and the campus in Aurora also have partnered on a manufacturing facility that reprograms patients immune cells to fight certain cancers. Elliman said they dont expect to need a new building for the institutes expanded work.

Regenerative medicine is a broad term for treatments that try to harness the bodys ability to fix itself. That could involve reprogramming cells to replace dying tissue or fight cancer, or therapies that insert a healthy gene to replace a defective version thats causing disease.

Its early enough in the process that the institutes leadership hasnt chosen specific focus areas under the regenerative medicine umbrella.

Most of the $200 million will go toward hiring scientists, as well as support personnel to help both the new researchers and those already working on campus, Elliman said.

Once the institute is up and running, it will bring in new federal grants to support research and investment from biotech firms that can bring the treatments to market, he said.

This investment is really a seed investment, he said.

Dr. Terry Fry, the institutes executive director, said its meant to help scientists with ideas that show promise in the lab to take the steps toward testing them in humans.

The process of manufacturing treatments and getting trials approved is more complex for biologic therapies than for standard drugs, he said.

Theres a stage in the development of that sort of project where investigator-scientists run up against a brick wall, he said. A lot of the role that I see the institute playing is removing those barriers.

Fry, a pediatric oncologist and head of T-Cell therapeutics at Sana Biotechnology, was one of the first researchers who worked on chimeric antigen receptor T-cell (CAR-T) therapy the immune cell reprogramming therapy. It was approved first for children with leukemia, but now is also used for adults and for other blood cancers, like lymphoma and myeloma. He declined to name specific projects the institute would work on, but said potential improvements to CAR-T could be within its scope.

The therapy takes a kind of T-cell that kills cells infected with viruses or bacteria, and gets it to attack cancerous cells instead. While it has improved survival for people with certain blood cancers, it doesnt work well against solid tumors at this point. It also requires taking T-cells from each patient to produce their own treatment, which is expensive and slows down the process. Researchers are working on how to make CAR-T work for more people, and to create an off the shelf option, Fry said.

Another general area the institute could work on is growing cells to replace ones that have died or are defective, Fry said. Much of that work involves adult stem cells that have been coaxed back into an earlier form, when they could become almost any type of cell under the right conditions.

For example, if the stem cells can be primed to turn into cells producing insulin, that could help patients with Type 1 diabetes, which is caused when the insulin-producers die, he said.

It is really remarkable technology, he said.

The institute wont take down every hurdle to bringing new treatments to patients, Fry said. Manufacturing and distributing at a large scale will require partnerships with biotech firms, which fortunately are setting up in the Denver area in increasing numbers, he said.

I think this is the right time and exactly the right part of the country for this type of institute, he said.

Diane Gates Wallach, one of the Gates funds co-trustees, said the new institute will further her fathers goal of speeding up the process of getting new discoveries into clinical practice, so patients can benefit. Since the Anschutz campus includes researchers and two hospitals, it made sense to invest there, she said in a news release.

It takes a dynamic, innovative medical ecosystem for an institute like this to thrive and be successful, she said.

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CU Anschutz center for cell-based therapy gets $200 million expansion - The Denver Post

Researchers believe they may have found a way to strengthen possible treatments for glioblastoma and reduce the speed at which the aggressive tumour progresses This new study published in the journal Oncogene,suggests that an inhibitor drug which targets a particular cell protein, could refine therapeutic strategies against glioblastoma, making them more effective.

In demonstrating the potential impacts of differentiation therapy their research focuses on using drugs to switch malignant cells into a more benign composition and then as these cells divide, they grow more slowly therefore limiting tumour growth.

The researchers tested different drugs which belong to a family of proteins called kinases. They identified an inhibitor which targets a particular protein (PDGFR) and by altering the expression of downstream targets, it is able to switch glioblastoma cancer cells, and glioblastoma cancer stem cells, into neuronal-like cells and ultimately reduce their proliferation and invasion abilities.Furthermore, through in-vivo studies, the team then showed that treatment with this particular drug improved the effect of temozolomide.

Findings from a seven-year research project suggest that there could be a new approach to treating glioblastoma. In a peer-reviewed study published byBMC Cancer,UK scientists have shown that a short chain of amino acids (the HTL-001 peptide) is effective at targeting and inhibiting the function of a family of genes responsible for the growth of glioblastoma Hoxgenes.

Scientists have identified a drug that inhibits growth of the most aggressive meningiomas and how to most accurately identify which meningiomas will respond to the drug. The drug is a newer cancer treatment called abemaciclib. The scientists demonstrated the effectiveness of the drug in select patients, mouse models, a 3D living tissue brain tumour (organoids) and cell cultures. Investigators discovered that meningiomas can be divided into molecular subgroups with different clinical outcomes and recurrence rates. This new method of classifying tumours allows scientists to predict recurrence more accurately than the current method of classifying the tumour.

A study has created a library of models to study brain metastases that recapitulate the disease in humans. These models can be a relevant tool to understand the disease and discover new therapeutic approaches tailormade to each patient.

This National Geographic article is titled New method delivers life-saving drugs to the brainusing sound waves and provides an overview of focused ultrasound.

EANO members are able to sign in and access a paper about understanding epilepsy in IDH-mutated gliomas: towards a targeted therapy, while this paper, which is only available as a pdf, looks at how CXCL14 promotes a robust brain tumor-associated immune response in glioma.

Finally this week the University of Nottingham are recruiting for a 3-yr PhD studentship to focus on recapitulating the post-surgical brain microenvironment of atypical teratoid/rhabdoid tumours to identify proteins for targeted therapy

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Differentiation therapy, Hox genes and Abemaciclib Research update 13th May - Brain Tumour Research

Vertex Pharmaceuticals (VRTX 2.44%) is known for its blockbuster cystic fibrosis treatments. The company generates billions of dollars in revenue and profit from them annually, and it looks as if that's set to continue for quite some time. The closest potential competitor for Vertex's top-selling product is actually a candidate in its own pipeline.

That may be a good reason to buy shares of this biotech company, but I've got an even better one. Vertex today has six programs that are in mid-to-late-stage clinical trials, and during its recent first-quarter earnings call, management said they represent "multibillion-dollar opportunities."

Image source: Getty Images.

The company's nearest-to-market candidate just so happens to be a potential game-changer. If it is approved, it is designed to be a one-time curative treatment for a pair of inherited blood disorders. Vertex and partner CRISPR Therapeutics are studying the gene-editing candidate -- CTX001 -- in a pivotal trial. So far, results have been positive in both beta-thalassemia and sickle cell disease patients. The companies plan to file for regulatory approval by the end of this year.

Today, there are about 32,000 sickle cell and beta-thalassemia patients in the U.S. and Europe, all of whom could be candidates for the treatment, according to Vertex. The company already is preparing for a potential launch.

CTX001 would represent a significant milestone for Vertex as the company's first marketed product outside of its cystic fibrosis specialty. The biggest worry investors have had about Vertex in recent times is its dependence on the cystic fibrosis business -- even though its leadership position in the indication is solid. Success here could prove that Vertex has what it takes to expand into new areas.

Another potentially key treatment on the horizon is Vertex's candidate for acute pain. What makes that candidate -- VX-548 -- special is that it isn't an opioid. While opioids are effective, use of them may lead to addiction, and there are few other options when potent painkillers are needed. If Vertex is successful in developing an alternative to opioids, it could be a major revenue generator. The company recently reported positive phase 2 data for VX-548, and aims to begin pivotal studies in the second half of the year.

A third candidate that I see as big is VX-800, which targets type 1 diabetes. It's a stem cell-derived treatment to restore the function of pancreatic islet cells, which regulate the body's glucose levels.

Vertex actually is facing some headwinds here at the moment. The Food and Drug Administration (FDA) placed a clinical hold on the phase 1/2 study of VX-880. The agency said there is "insufficient information" to support an increase to full dosage of the diabetes treatment in the next part of the trial. Vertex's data using a half dose were positive. The study actually treated one patient with a full dose -- and that patient's results were positive too. Vertex now is working with the FDA to address the agency's questions. Considering the data so far, I'm still very optimistic about this type 1 diabetes program, and don't view the pause as a threat to what could be another game-changing product.

Vertex also has several preclinical programs worth keeping an eye on, though any revenues they may generate would start rolling in farther in the future.

One early program that stands out is an mRNA therapy for cystic fibrosis patients who aren't candidates for Vertex's current treatments. That's being developed in partnership with Moderna. Their candidate would instruct the body to make a particular protein these patients lack. Vertex and Moderna plan to ask the FDA for authorization to begin clinical studies in the second half of this year.

All of these elements could be fuel for future earnings increases -- and eventually, improved share performance. Speaking of the shares, they've lost about 17% since they hit a 52-week peak last month. They're now trading at less than 17 times forward earnings estimates, down from more than 20 earlier this year. As such, the stock looks like a bargain considering the potential of the new products that Vertex could bring to market -- both in the next year or so and farther down the road.

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Here's the No. 1 Reason to Buy Vertex Pharmaceuticals Now - The Motley Fool

OSAKA, Japan A new biomaterial can help regenerate tissue in people dealing with chronic lower back pain and spinal issues. Researchers in Japan say the secret to this breakthrough therapy is all in the hiPS. Not those hips, but human induced pluripotent stem cells.

A team from Osaka and Kyoto Universities explain that a common cause of lower back pain is the degeneration of intervertebral discs (IVDs). These discs sit between the vertebrae in the spine and help give the spinal column its flexibility. Severe IVD degeneration eventually leads to spinal deformity without treatment. In the new study, scientists used cartilage tissue derived from stem cells to build back lost IVDs in lab rats.

Researchers believe this problem starts in the nucleus pulposus (NP) the inner core of the vertebral disc. NP cells produce the surrounding extracellular matrix (ECM), which supports these cells and gives the NP its elasticity.

Although other treatments which use viable NP cells to fix the ECM have been promising, they lose their effectiveness in advanced cases of IVD degeneration. In people with severe degeneration in their spines, there arent enough of these cells present to respond to treatment.

With that in mind, the team looked at creating an implant which carries the necessary cells already. From there, the implant can create and regenerate the ECM that the NP cells form.

The ECM of the NP is a network of collagen that acts as scaffolding for other important proteins. Interestingly, this composition is similar to the ECM of articular cartilage, says lead author Takashi Kamatani in a media release. Thus, we hypothesized that cell types that can produce and support cartilage could be useful for treating IVD degeneration.

Study authors used induced pluripotent stem cells (iPSCs) during their experiments. These cells are basically the building blocks of development, turning into other types of cells as a person grows from an infant to an adult. They also dont have the growth and division limits that NP cells do.

Importantly, scientists are capable of turning iPSCs into chondrocytes cells that produce and maintain cartilage. Previous studies have successfully used this same method to treat cartilage defects in animals.

In the new study, researchers created human iPSC-derived cartilaginous tissue (hiPS-Cart) that they implanted into rats with no NP cells in their intervertebral discs.

The hiPS-Cart implanted in these rats was able to survive and be maintained, explains senior author Noriyuki Tsumaki. IVD and vertebral bone degeneration were prevented. We also assessed the mechanics and found that hiPS-Cart was able to revert these properties to similar levels observed in the control rats.

The team also studied the gene expression of the hiPS-Cart six weeks after the procedure. Results show hiPS-Cart displayed the same characteristics of chondrocyte-like NP cells, instead of another type of NP cell called notochordal. Researchers say this means chondrocyte-like cells are able to restore spinal health and function on their own.

Our findings provide strong support for using this hiPS-Cart system in the development of treatments for human IVD degeneration, Kamatani concludes.

The study is published in the journal Biomaterials.

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Stem cell cure for lower back pain is all in the 'hiPS' - Study Finds

Tokyo This month, a team of researchers at Osaka University declared an experimental treatment involving four patients suffering from corneal disease a success. The patients, who ranged in age from their 30s to 70s, received transplanted stem cells grown in the lab, known as iPS cells. Three had improved sight, and all were free of side effects one year later.

"This could be a revolutionary treatment that could overcome the challenges that existing treatment has faced, such as a shortage of cornea donors or transplant rejection," Koji Nishida, an Osaka University professor of ophthalmology, said at a news conference.

It was the latest in a flurry of iPS-related announcements in Japan as the country tries to carve a niche in "regenerative medicine" by culturing healthy cells to replace diseased, injured or non-functioning ones.

Induced pluripotent stem (iPS) cells are altered to revert to a non-differentiated "stem" state the building blocks of most organs. The stem cells can then be used to repair human tissues or grow organs.

Japan has invested $970 million in regenerative medicine, focusing on iPS as a strategy for coping with the world's oldest population and as a source of future economic growth. iPS is particularly attractive for Japan, which has one of the lowest rates of organ donation in the industrialized world.

iPS stem cell research in Japan took off after 2012, when biologist Shinya Yamanaka received the Nobel Prize in physiology or medicine after he discovered how to transform mature skin or blood cells into immature stem cells, which can then become neurons, muscle, cartilage or heart muscle cells.

Yamanaka went into medicine after his own father contracted hepatitis C a disease that became treatable 20 years later. Had iPS cells been available as a testing medium, he said, that treatment could have been developed much faster.

In the time since, numerous small-scale trials have been conducted for diseases like age-related macular degeneration, Parkinson's disease and arthritic disorders. In 2020, a six-day-old infant with a liver disorder received an iPS treatment that enabled the child to survive until it was old enough for a liver transplant. A stem cell transplant treatment was approved in 2019 for spinal-cord injuries in Japan.

iPS also offers hope for treating intractable and rare diseases like ALS - or Lou Gehrig's disease - and Alzheimer's.

And Yamanaka's findings offer a solution to the politically divisive dilemma posed by the use of embryonic stem cells, which rely on fertilized human eggs.

The development also eliminates the risk of transplant rejection of donor stem cells, since infinite lines of stem cells can be grown in a lab from as little as about two teaspoons of a patient's own skin or blood cells.

To reduce the massive cost and time needed to create iPS cells from each patient, a donor bank stockpile was set up in conjunction with the Red Cross that identified a tiny population of "super donors" whose blood can be used for many immunological types.

The corneal patients in the Osaka University trial received donor-generated iPS cells.

"I couldn't say Japan is leading the way with iPS because everybody, everybody's using it," David Cyranoski, a science policy researcher at Kyoto University's Institute for the Advanced Study of Human Biology, told CBS News. "It's such a powerful technology and it's so easy to adapt."

But while treatments using iPS offer hope for the future, approval is years away. "Stem cells themselves are harder to use than people thought," Cyranoski said, adding the therapies "haven't really proven themselves yet."

Despite hundreds of clinics in the United States offering unproven stem-cell therapies, which have been implicated in scores of deaths and injuries, the FDA has approved only blood stem cell transplantations for cancers and disorders of the blood and immune systems.

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Stem cell transplants can effectively cure a wide range of diseases, from blood cancers to rare genetic disorders. They've been used for decades and are considered standard treatment for certain conditions.

But for a good number of patients, stem cell transplants are out of reach. Drug regimens used to prepare the body for a transplant are toxic and can cause serious side effects. The transplanted cells don't always "engraft," or take root in the bone marrow. Even when they do, patients' disease may linger or recur.

A biotech startup launching Wednesday with $50 million in funding hopes that, by combining cell, antibody and gene editing technologies, at least some of these problems can be overcome. Called Cimeio Therapeutics, the new company is led by a team of pharmaceutical industry veterans and an advisory board filled with scientific luminaries, including immunologist Jeffrey Bluestone and gene editing pioneer Fyodor Urnov.

Cimeio's approach involves "shielding" transplanted cells by genetically editing them in ways that allows paired immunotherapies to be safely used both before and after a transplant.

Thomas Fuchs

Courtesy of Cimeio Therapeutics

"We think that this can really unleash the power of hematopoietic stem cell transplant and make a lot more patients eligible for it," said Thomas Fuchs, Cimeio's CEO and a former Genentech executive.

The "shielding" technology used by Cimeio was developed in Switzerland at the laboratory of Lukas Jeker, a physician-scientist from Basel University Hospital who will join Cimeio as head of gene editing.

Jeker's lab discovered that protein receptors on the surface of cells could be genetically edited in such a way that prevented antibodies from binding to them, while leaving their function intact. In preclinical testing, these edits could cloak, or "shield," the cells from being depleted by antibody drugs and T cell therapies.

The work could have powerful implications for improving stem cell transplant and adoptive cell therapy, according to Fuchs.

Once a stem cell or T cell is shielded, a complementary immunotherapy could be used to either help ready patients for a transplant or to further treat disease afterwards, he said. "Maybe you could give a cycle or two of the paired immunotherapy, implant the shielded cells and then continue to administer the immunotherapy," he added.

If the shielding works as intended, Cimeio could develop treatments for conditioning that are more tolerable than the chemotherapy or radiation-based regimens currently in use. Shielding might also allow existing drugs that target cell proteins on healthy as well as diseased cells to be used more flexibly with transplants, such as to treat residual disease that lingers afterwards.

For example, Cimeio could engineer stem cells that are protected against binding via a protein called CD19 that's often the target for CAR-T therapies that treat lymphoma, but is also found on healthy B cells that help the immune system fight off threats.

"One benefit could be that you could prevent a lifetime of B cell depletion, which happens when you give a CAR-T," said Fuchs.

Alex Mayweg

Courtesy of Cimeio Therapeutics

Cimeio was built from Jeker's lab by Versant Ventures at the company's "Ridgeline" incubator in Basel, which has previously produced companies like Monte Rosa Therapeutics and Black Diamond Therapeutics. The initial $50 million Versant provided will fund Cimeio through next year, said Alex Mayweg, a managing director at the venture firm and a Cimeio board member. Additional investors will be brought on later this year or early next, Mayweg said.

Cimeio will need the money, as its research and development plans are expansive. The company has identified four drug candidates already and envisions a dozen more behind those, said Fuchs. Its research spans blood cancers, rare genetic diseases and autoimmune disorders.

In some cases, Cimeio will develop paired immunotherapies to go with the shielded cells. In others, it will use existing treatments. Three of the first four candidates involve protecting hematopoietic stem cells, while the fourth involves T cells. The company hopes to begin human testing next year.

Cimeio plans to choose gene editing technologies based on the type of alteration it needs to make to shield cells. "Rather than building up an internal editing capability," Mayweg said, "we wanted to stay as flexible as possible."

That might mean partnerships or alliances with other companies, some of which have reached out to Cimeio already, according to Mayweg.

Cimeio is aided by a group of scientific advisers notable for their work in areas the company is focusing on. Urnov, of the University of California, Berkeley, is well known for his research in gene editing using zinc finger nucleases and CRISPR. Bluestone previously led the Parker Institute for Cancer Immunotherapy and is CEO of the cell therapy-focused biotech Sonoma Biotherapeutics.

Suneet Agarwal, a co-program leader of the stem cell transplant center at Boston Children's Cancer and Blood Disorders Center, is also on the advisory board, while Cimeio has a research collaboration in place with Matthew Porteus, a gene editing specialist at Stanford University.

About 20 people currently work at Cimeio directly, a number Fuchs expects will grow as the company's research advances. Another 15 are currently supporting Cimeio from Versant's Ridgeline group.

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Versant-backed startup launches with plans to broaden cell therapy's reach - BioPharma Dive