Monthly Archives: April 2022

Single-cell RNA sequencing analysis of human bone-marrow-derived mesenchymal stem cells and functional subpopulation identification – DocWire News

Posted: April 6, 2022 at 2:04 am

This article was originally published here

Exp Mol Med. 2022 Apr 1. doi: 10.1038/s12276-022-00749-5. Online ahead of print.

ABSTRACT

Mesenchymal stem cells (MSCs) are a common kind of multipotent cell in vivo, but their heterogeneity limits their further applications. To identify MSC subpopulations and clarify their relationships, we performed cell mapping of bone-marrow-derived MSCs through single-cell RNA (scRNA) sequencing. In our study, three main subpopulations, namely, the stemness subpopulation, functional subpopulation, and proliferative subpopulation, were identified using marker genes and further bioinformatic analyses. Developmental trajectory analysis showed that the stemness subpopulation was the root and then became either the functional subpopulation or the proliferative subpopulation. The functional subpopulation showed stronger immunoregulatory and osteogenic differentiation abilities but lower proliferation and adipogenic differentiation. MSCs at different passages or isolated from different donors exhibited distinct cell mapping profiles, which accounted for their corresponding different functions. This study provides new insight into the biological features and clinical use of MSCs at the single-cell level, which may contribute to expanding their application in the clinic.

PMID:35365767 | DOI:10.1038/s12276-022-00749-5

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Single-cell RNA sequencing analysis of human bone-marrow-derived mesenchymal stem cells and functional subpopulation identification - DocWire News

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We finally have a fully complete human genome – Science News Magazine

Posted: April 6, 2022 at 2:01 am

Researchers have finally deciphered a complete human genetic instruction book from cover to cover.

The completion of the human genome has been announced a couple of times in the past, but those were actually incomplete drafts. We really mean it this time, says Evan Eichler, a human geneticist and Howard Hughes Medical Institute investigator at the University of Washington in Seattle.

The completed genome is presented in a series of papers published online March 31 in Science and Nature Methods.

An international team of researchers, including Eichler, used new DNA sequencing technology to untangle repetitive stretches of DNA that were redacted from an earlier version of the genome, widely used as a reference for guiding biomedical research.

Deciphering those tricky stretches adds about 200 million DNA bases, about 8 percent of the genome, to the instruction book, researchers report in Science. Thats essentially an entire chapter. And its a juicy one, containing the first-ever looks at the short arms of some chromosomes, long-lost genes and important parts of chromosomes called centromeres where machinery responsible for divvying up DNA grips the chromosome.

Some of the regions that were missing actually turn out to be the most interesting, says Rajiv McCoy, a human geneticist at Johns Hopkins University, who was part of the team known as the Telomere-to-Telomere (T2T) Consortium assembling the complete genome. Its exciting because we get to take the first look inside these regions and see what we can find. Telomeres are repetitive stretches of DNA found at the ends of chromosomes. Like aglets on shoelaces, they may help keep chromosomes from unraveling.

Data from the effort are already available for other researchers to explore. And some, like geneticist Ting Wang of Washington University School of Medicine in St. Louis, have already delved in. Having a complete genome reference definitely improves biomedical studies. Its an extremely useful resource, he says. Theres no question that this is an important achievement.

But, Wang says, the human genome isnt quite complete yet.

To understand why and what this new volume of the human genetic encyclopedia tells us, heres a closer look at the milestone.

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Eichler is careful to point out that this is the completion of a human genome. There is no such thing as the human genome. Any two people will have large portions of their genomes that range from very similar to virtually identical and smaller portions that are wildly different. A reference genome can help researchers see where people differ, which can point to genes that may be involved in diseases. Having a view of the entire genome, with no gaps or hidden DNA, may give scientists a better understanding of human health, disease and evolution.

The newly complete genome doesnt have gaps like the previous human reference genome. But it still has limitations, Wang says. The old reference genome is a conglomerate of more than 60 peoples DNA (SN: 3/4/21). Not a single individual, or single cell on this planet, has that genome. That goes for the new, complete genome, too. Its a quote-unquote fake genome, says Wang, who was not involved with the project.

The new genome doesnt come from a person either. Its the genome of a complete hydatidiform mole, a sort of tumor that arises when a sperm fertilizes an empty egg and the fathers chromosomes are duplicated. The researchers chose to decipher the complete genome from a cell line called CHM13 made from one of these unusual tumors.

That decision was made for a technical reason, says geneticist Karen Miga of the University of California, Santa Cruz. Usually, people get one set of chromosomes from their mother and another set from their father. So we all have two genomes in every cell.

If putting together a genome is like assembling a puzzle, you essentially have two puzzles in the same box that look very similar to each other, says Miga, borrowing an analogy from a colleague. Researchers would have to sort the two puzzles before piecing them together. Genomes from hydatidiform moles dont present that same challenge. Its just one puzzle in the box.

The researchers did have to add the Y chromosome from another person, because the sperm that created the hydatidiform mole carried an X chromosome.

Even putting one puzzle together is a Herculean task. But new technologies that allow researchers to put DNA bases represented by the letters A, T, C and G in order, can spit out stretches up to more than 100,000 bases long. Just as childrens puzzles are easier to solve because of larger and fewer pieces, these long reads made assembling the bits of the genome easier, especially in repetitive parts where just a few bases might distinguish one copy from another. The bigger pieces also allowed researchers to correct some mistakes in the old reference genome.

For starters, the newly deciphered DNA contains the short arms of chromosomes 13, 14, 15, 21 and 22. These acrocentric chromosomes dont resemble nice, neat Xs the way the rest of the chromosomes do. Instead, they have a set of long arms and one of nubby short arms.

The length of the short arms belies their importance. These arms are home to rDNA genes, which encode rRNAs, which are key components of complex molecular machines called ribosomes. Ribosomes read genetic instructions and build all the proteins needed to make cells and bodies work. There are hundreds of copies of these rDNA regions in every persons genome, an average of 315, but some people have more and some fewer. Theyre important for making sure cells have protein-building factories at the ready.

We didnt know what to expect in these regions, Miga says. We found that every acrocentric chromosome, and every rDNA on that acrocentric chromosome, had variants, changes to the repeat unit that was private to that particular chromosome.

By using fluorescent tags, Eichler and colleagues discovered that repetitive DNA next to the rDNA regions and perhaps the rDNA too sometimes switches places to land on another chromosome, the team reports in Science. Its like musical chairs, he says. Why and how that happens is still a mystery.

The complete genome also contains 3,604 genes, including 140 that encode proteins, that werent present in the old, incomplete genome. Many of those genes are slightly different copies of previously known genes, including some that have been implicated in brain evolution and development, autism, immune responses, cancer and cardiovascular disease. Having a map of where all these genes lie may lead to a better understanding of what they do, and perhaps even of what makes humans human.

One of the biggest finds may be the structure of all of the human centromeres. Centromeres, the pinched portions which give most chromosomes their characteristic X shape, are the assembly points for kinetochores, the cellular machinery that divvies up DNA during cell division. Thats one of the most important jobs in a cell. When it goes wrong, birth defects, cancer or death can result. Researchers had already deciphered the centromeres of fruit flies and the human 8, X and Y chromosomes (SN: 5/17/19), but this is the first time that researchers got a glimpse of the rest of the human centromeres.

The structures are mostly head-to-tail repeats of about 171 base pairs of DNA known as alpha satellites. But those repeats are nestled within other repeats, creating complex patterns that distinguish each chromosomes individual centromere, Miga and colleagues describe in Science. Knowing the structures will help researchers learn more about how chromosomes are divvied up and what sometimes throws off the process.

Researchers also now have a more complete map of epigenetic marks chemical tags on DNA or associated proteins that may change how genes are regulated. One type of epigenetic mark, known as DNA methylation, is fairly abundant across the centromeres, except for one spot in each chromosome called the centromeric dip region, Winston Timp, a biomedical engineer at Johns Hopkins University and colleagues report in Science.

Those dips are where kinetochores grab the DNA, the researchers discovered. But its not yet clear whether the dip in methylation causes the cellular machinery to assemble in that spot or if assembly of the machinery leads to lower levels of methylation.

Examining DNA methylation patterns in multiple peoples DNA and comparing them with the new reference revealed that the dips occur at different spots in each persons centromeres, though the consequences of that arent known.

About half of genes implicated in the evolution of humans large, wrinkly brains are found in multiple copies in the newly uncovered repetitive parts of the genome (SN: 2/26/15). Overlaying the epigenetic maps on the reference allowed researchers to figure out which of many copies of those genes were turned on and off, says Ariel Gershman, a geneticist at Johns Hopkins University School of Medicine.

That gives us a little bit more insight into which of them are actually important and playing a functional role in the development of the human brain, Gershman says. That was exciting for us, because theres never been a reference that was accurate enough in these [repetitive] regions to tell which gene was which, and which ones are turned on or off.

One criticism of genetics research is that it has relied too heavily on DNA from people of European descent. CHM13 also has European heritage. But researchers have used the new reference to discover new patterns of genetic diversity. Using DNA data collected from thousands of people of diverse backgrounds who participated in earlier research projects compared with the T2T reference, researchers more easily and accurately found places where people differ, McCoy and colleagues report in Science.

The Telomere-to-Telomere Consortium has now teamed up with Wang and his colleagues to make complete genomes of 350 people from diverse backgrounds (SN: 2/22/21). That effort, known as the pangenome project, is poised to reveal some of its first findings later this year, Wang says.

McCoy and Timp say that it may take some time, but eventually, researchers may switch from using the old reference genome to the more complete and accurate T2T reference. Its like upgrading to a new version of software, Timp says. Not everyone is going to want to do it right away.

The completed human genome will also be useful for researchers studying other organisms, says Amanda Larracuente, an evolutionary geneticist at the University of Rochester in New York who was not involved in the project. What Im excited about is the techniques and tools this team has developed, and being able to apply those to study other species.

Eichler and others already have plans to make complete genomes of chimpanzees, bonobos and other great apes to learn more about how humans evolved differently than apes did. No one should see this as the end, Eichler says, but a transformation, not only for genomic research but for clinical medicine, though that will take years to achieve.

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Researchers identify regions of genome involved in Alzheimer’s – UPI News

Posted: April 6, 2022 at 2:01 am

New research has identified 75 regions of the human genome involved in Alzheimer's development. File photo by BillionPhotos.com/Shutterstock

April 4 (UPI) -- An international team of researchers has identified 75 regions of the human genome associated with the development of Alzheimer's disease, including 42 never before linked with the common form of dementia, they said Monday.

After analyzing the genomes, or complete genetic data, for thousands of people, the researchers found 75 loci, or regions, of DNA involved in Alzheimer's, they reported in an article published Monday by the journal Nature Genetics.

Several of the identified regions of the genome are involved in the accumulation of amyloid-beta in the brain, which is known to cause Alzheimer's, according to the researchers.

In addition, they identified genes that affect production of a protein called tau that is found in brain cells. Changes in tau production also have been linked with Alzheimer's disease, the researchers said.

Based on their findings, the researchers have developed a genetic "risk score" for Alzheimer's, though it is still in the draft stage and is not yet ready for use in clinical practice, they said.

"Our knowledge of the genetics of AD common forms cannot allow it to be used as an individual diagnostic tool yet," study co-author Jean-Charles Lambert told UPI in an email.

"On the other hand, we show in our paper that this knowledge makes it possible in populations to define groups of individuals more or less at risk of developing the [disease]," said Lambert, research director at Inserm in Lille, France.

On Thursday, researchers working with the National Human Genome Research Institute announced that they had mapped a complete human genome for the first time.

This map could serve as a "reference," or guide, for researchers seeking to identify the genetic component of various diseases and traits, they said.

Amyloid-beta and tau have both been linked with Alzheimer's, the most common form of dementia in the United States, affecting some 6 million people, most of whom are age 65 years and older, according to the Alzheimer's Association.

However, it is not yet fully understood why some people have higher levels of amyloid-beta in their brains than others, placing them at higher risk for cognitive, or brain function, decline, Lambert said.

Most cases of Alzheimer's are thought to be caused by the interaction of different genetic and environmental factors, the latter of which include air pollution, research suggests.

In addition to identifying the genome regions behind amyloid-beta and tau development, Lambert and his colleagues also noted that many people with Alzheimer's also have modifications, or changes, in the genome that impact immune response, they said.

These changes affect the function of microglia, or the immune cells in the central nervous system that play a "trash collector" role by eliminating toxic substances, the researchers said.

The analysis also revealed that the tumor necrosis factor alpha-dependent signaling pathway, which plays a role in cell development, according to researchers.

The findings suggest that future clinical trials of therapies designed to treat Alzheimer's should focus on targeting amyloid-beta, microglial cells and the tumor necrosis factor alpha signaling pathway, they said.

They plan to validate the accuracy of their genetic risk score in future studies.

"This genetic knowledge will be the basis of personalized medicine" for Alzheimer's patients, Lambert said.

"This research is important today for the development of therapeutic approaches but in the not-so-distant future, for the clinical management of patients at the earliest stage," he said.

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Phase 1 Trial Begins Dosing of TARA-002 Cell Therapy for NMIBC – Targeted Oncology

Posted: April 6, 2022 at 2:00 am

The phase 1 ADVANCED-1 trial of the biologic immunopotentiator agent TARA-002 began dosing patients with non-muscular invasive bladder cancer.

A clinical trial investigating the safety and toxicity of TARA-002, a cell therapy agent, for non-muscular invasive bladder cancer (NMIBC), has started dosing, according to a press release from Protara Therapeutics.1

The new immunopotentiator agent phase 1 ADVANCED-1 trial (NCT05085977) is now investigating a single-arm, open-label, dose-finding trial of TARA-002 for treatment-naive and previously treated patients with NMIBC with high-grade carcinoma in situ (CIS) and high-grade papillary tumors.

NMIBC is one of the most recurrent and difficult to treat cancers with very limited treatment options, Jesse Shefferman, chief executive officer of Protara Therapeutics, said in a statement. We are thrilled to have dosed the first patient in our phase 1 study in NMIBC and look forward to exploring TARA-002s full potential in this pressing area of high unmet need.

TARA-002 is an immunopotentiator cell therapy based on group A Streptococcus pyogenes that cause immune cells to produce a local inflammatory cytokine reaction that destroys abnormal cells in a cyst or tumor. It was developed from the same genetic cell bank as OK-432 (Picibanil), a broad immunopotentiator that is approved in Japan and Taiwan.

The phase 1a of the ADVANCED-1 trial is enrolling an estimated 18 patients with NMIBC to receive 6 weekly intravesical doses of TARA-002. Patients are eligible if they are unable to receive intravesical Bacillus Calmette-Gurin (BCG), have received at least 1 dose of BCG, or have received at least 1 dose of intravesical chemotherapy.

Patients will receive up to 3 dose levels: 10 KE, 20 KE, and 40 KE, which will be tested sequentially starting with the lowest dose. The goal of the study is to determine the agents safety and tolerability as well as observe for preliminary signs of antitumor activity. The primary end points for the dose escalation phase are dose-limiting toxicities, maximum tolerated dose, and recommended phase 2 dose (RP2D).

The phase 1b dose expansion part of the trial (NCT05085990) is planned to enroll patients with CIS NMIBC in 2023 to receive 6 weekly doses of the RP2D. Its primary end point is the incidence of adverse events in these patients.

In 2021, TARA-002 was granted a rare prediatric disease designation by the FDA for lymphatic malformations, a rare condition affecting young children.2 Protara stated its intention to begin a clinical trial of TARA-002 in a clinical trial in this setting. While OK-432 is the standard-of-care agent to treat lymphatic malformations in Japan and Taiwan, there is no FDA-approved agent for this disease.

While bladder cancer is the 6th most common cancer in the United States today, with NMIBC representing approximately 80% of diagnoses, treatment options for these patients remain limited, Edward M. Messing, MD, a principal investigator of the ADVANCED-1 study and a professor of urology, oncology, and pathology at the University of Rochester said in a statement.1 There is an urgent need for new therapeutic interventions for these patients, as there continues to be an increase in recurrence, progression and an escalated number of patients needing cystectomies.

References:

1. Protara Therapeutics doses first patient in ADVANCED-1 phase 1 study of TARA-002 in non-muscle invasive bladder cancer. Protara Therapeutics. March 24, 2022. Accessed April 1, 2022. https://bit.ly/376E9TY

2. Protara Therapeutics provides regulatory update for TARA-002 for the treatment of lymphatic malformations. Protara Therapeutics. April 23, 2021. Accessed April 1, 2022. https://bit.ly/37cE5lG

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Phase 1 Trial Begins Dosing of TARA-002 Cell Therapy for NMIBC - Targeted Oncology

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UAB has been recognized as a Program of Experience for CAR-T therapy – University of Alabama at Birmingham

Posted: April 6, 2022 at 2:00 am

UAB has been awarded for its superior CAR-T therapy program services and leadership.

UAB has been awarded for its superior CAR-T therapy program services and leadership. Photography: Steve WoodThe University of Alabama at Birmingham has been identified by Emerging Therapy Solutions as a Program of Experience for Chimeric antigen receptor T cell therapy.

According to the American Cancer Society, CAR-T therapy is a path for immune cells called T cells a type of white blood cell to fight cancer by changing them in the lab so they can find and destroy cancer cells. This form of treatment can be helpful for patients with various cancer types even when other types of treatments are no longer available.

CAR-T therapy is becoming an essential pillar of cancer care, joining the ranks of surgery, chemotherapy, radiation and other targeted treatments.

The UAB-BMT and Cell Therapy Program is the only state-of-the-art program in Alabama to offer CAR-T therapy for patients with some cancers, said Amitkumar Mehta, M.D., associate scientist in the ONeal Comprehensive Cancer Center at UAB, assistant professor in the UAB Division of Hematology and Oncology, Director of CAR-T Program and Medical Director of Clinical Trial Office. The program is certified for all commercial FDA-approved CAR-T products. The program also supports next generation cell therapy treatment through clinical trials. The CAR-T team is a highly specialized and experienced team that supports the patients who are on this treatment. This recognition is an appreciation of the dedicated and patient-centered cell therapy team at UAB-BMT. I am very proud to be part of the program.

ETS created this designation after evaluating centers across the United States that provide CAR-T services and identifying the leading CAR-T programs with the essential experience and resources, including volume of CAR-Ts administered.

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Nurix Therapeutics Doses First Patient in Phase 1 Clinical Trial of DeTIL-0255, a Drug-Enhanced Cell Therapy for the Treatment of Patients with Solid…

Posted: April 6, 2022 at 2:00 am

SAN FRANCISCO, April 05, 2022 (GLOBE NEWSWIRE) -- Nurix Therapeutics, Inc. (Nasdaq:NRIX), a clinical stage biopharmaceutical company developing targeted protein modulation drugs, today announced that the first patient has been dosed in its Phase 1 clinical trial of DeTIL-0255, a drug-enhanced tumor infiltrating lymphocyte therapy and the lead candidate in its cellular therapy portfolio. The trial is designed to evaluate the safety and efficacy of DeTIL-0255 in patients with advanced gynecological malignancies including ovarian cancer, cervical cancer, and endometrial cancer.

DeTIL-0255 is a cell therapy derived via ex-vivo treatment of patient-derived tumor infiltrating lymphocytes (TILs) with a potent, small-molecule inhibitor of Casitas B-lineage lymphoma proto-oncogene-B (CBL-B) called NX-0255, which was identified using Nurixs proprietary DELigase platform.

The initiation of our first cell therapy study is a major milestone for Nurix and the culmination of significant efforts across our clinical, regulatory and manufacturing teams. It is also the first time targeted protein modulation has been combined with cell therapy, marking the beginning of what we believe will be an important step forward in the treatment of solid tumors, said Robert J. Brown, M.D., executive vice president of clinical development of Nurix. Within the rubric of targeted protein modulation, Nurix has now moved three treatment modalities into the clinic including oral targeted protein degraders, an oral CBL-B inhibitor, and now a drug-enhanced cell therapy.

CBL-B is an E3 ligase that is expressed in immune cells, and in the context of cancer functions as an intracellular checkpoint that negatively regulates T cell activation, NK cell activity, and immune response through the degradation of specific intracellular signalling proteins. Inhibition of CBL-B with NX-0255 increases those protein levels and is used to generate a drug-enhanced cell therapy product with superior anti-tumor activity in animal models of adoptive cell therapy. Nurix expects to provide a clinical update from the safety run-in portion of the Phase 1 study in the second half of 2022.

Our preclinical models of adoptive T-cell therapy demonstrate that NX-0255 treatment of cells provides improved characteristics that have the potential to increase the success of manufacturing durable cells that can deliver significant anti-tumor effects, said Michael T. Lotze, M.D., chief cellular therapy officer of Nurix. DeTIL-0255 is an autologous TIL product that is designed to overcome the major limitations of current TIL therapy which include T cell exhaustion post expansion, suboptimal manufacturing success rates, and poor persistence of anti-tumor cells in the patient.

About DeTIL-0255The DeTIL-0255 investigational product under development is an autologous cell therapy consisting of T cells derived from a patients tumor expanded in culture with recombinant interleukin-2 and the small molecule CBL-B inhibitor NX-0255. DeTIL-0255 is designed to be a single administration autologous TIL therapy infused following non-myeloablative chemotherapy. Given the improved phenotypes of T cells produced with CBL-B inhibition, DeTIL-0255 could allow a broader application of TIL therapy, potentially providing long term benefit to patients with multiple types of cancer. Nurix is conducting the open label Phase 1 trial of DeTIL-0255 at multiple sites in the United States. Additional information on the clinical trial can be accessed at http://www.clinicaltrials.gov (NCT05107739).

About Nurix Therapeutics, Inc.Nurix Therapeutics is a clinical stage biopharmaceutical company focused on the discovery, development, and commercialization of small molecule and cell therapies based on the modulation of cellular protein levels as a novel treatment approach for cancer and other challenging diseases. Leveraging Nurixs extensive expertise in E3 ligases together with its proprietary DNA-encoded libraries, Nurix has built DELigase, an integrated discovery platform to identify and advance novel drug candidates targeting E3 ligases, a broad class of enzymes that can modulate proteins within the cell. Nurixs drug discovery approach is to either harness or inhibit the natural function of E3 ligases within the ubiquitin proteasome system to selectively decrease or increase cellular protein levels. Nurixs wholly owned pipeline includes targeted protein degraders of Brutons tyrosine kinase, a B-cell signaling protein, and inhibitors of Casitas B-lineage lymphoma proto-oncogene B, an E3 ligase that regulates T cell activation. Nurix is headquartered in San Francisco, California. For more information, please visit http://www.nurixtx.com/.

Forward Looking StatementThis press release contains statements that relate to future events and expectations and as such constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. When or if used in this press release, the words anticipate, believe, could, estimate, expect, intend, may, outlook, plan, predict, should, will, and similar expressions and their variants, as they relate to Nurix, may identify forward-looking statements. All statements that reflect Nurixs expectations, assumptions or projections about the future, other than statements of historical fact, are forward-looking statements, including, without limitation, statements regarding our future financial or business performance, conditions, plans, prospects, trends or strategies and other financial and business matters; our current and prospective drug candidates; the planned timing and conduct of our clinical trial programs for our drug candidates; the planned timing for the provision of clinical updates and initial findings from our clinical studies; the potential advantages of our DELigase platform and drug candidates; and the extent to which our scientific approach and DELigase platform may potentially address a broad range of diseases. Forward-looking statements reflect Nurixs current beliefs, expectations, and assumptions regarding the future of Nurixs business, future plans and strategies, its development plans, its preclinical and clinical results, future conditions and other factors Nurix believes are appropriate in the circumstances. Although Nurix believes the expectations and assumptions reflected in such forward-looking statements are reasonable, Nurix can give no assurance that they will prove to be correct. Forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and changes in circumstances that are difficult to predict, which could cause Nurixs actual activities and results to differ materially from those expressed in any forward-looking statement. Such risks and uncertainties include, but are not limited to: (i) risks and uncertainties related to Nurixs ability to advance its drug candidates, obtain regulatory approval of and ultimately commercialize its drug candidates; (ii) the timing and results of preclinical studies and clinical trials; (iii) Nurixs ability to fund development activities and achieve development goals; (iv) the impact of the COVID-19 pandemic on Nurixs business, clinical trials, financial condition, liquidity and results of operations; (v) Nurixs ability to protect intellectual property and (vi) other risks and uncertainties described under the heading Risk Factors in Nurixs Annual Report on Form 10-K for the fiscal year ended November 30, 2021 and other SEC filings. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. The statements in this press release speak only as of the date of this press release, even if subsequently made available by Nurix on its website or otherwise. Nurix disclaims any intention or obligation to update publicly any forward-looking statements, whether in response to new information, future events, or otherwise, except as required by applicable law.

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Nurix Therapeutics Doses First Patient in Phase 1 Clinical Trial of DeTIL-0255, a Drug-Enhanced Cell Therapy for the Treatment of Patients with Solid...

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Reimbursement price of CAR-T cell therapy Kymriah set at $296138 – KBR

Posted: April 6, 2022 at 2:00 am

The government set the maximum reimbursement price of the worlds first CAR-T cell therapy Kymriah (tisagenlecleucel), developed by Novartis, at 360,039,359 won ($296,138) per treatment.

It also newly established reimbursement for medical practices for cell collection, in vitro treatment, treatment preparation, and treatment injection that accompany CAR-T cell therapy.

On Thursday, the Ministry of Health and Welfare held a Health Insurance Policy Review Committee meeting and decided to revise the list of reimbursed drugs and the maximum reimbursement rates.

Kymriahs reimbursement will be valid from Friday.

Regarding the application of the risk-sharing system to Kymriah, the government decided to grant an increase in health insurance coverage for both two indications -- B-cell acute lymphoblastic leukemia and diffuse large B-cell lymphoma.

To indicate diffuse large B-cell lymphoma, the government decided to apply a performance-based risk-sharing agreement (RSA) on a patient-by-patient basis. Therefore, patients without any treatment effect will have an additional refund.

On Thursday, the Korea Leukemia Patients Organization (KLPO) welcomed the news of Kymriah's reimbursement.

There is no better news than this for some 200 patients who are at risk of dying within three to six months if they did not receive Kymriah treatment, the patient group said.

The government authorities must thoroughly manage Kymriahs effects and side effects so that the refund-type RSA and expenditure cap RSA of 70.9 billion won per year, which are conditions for Kymriah's health insurance listing, but the performance-based RSA applicable only to lymphoma are properly implemented, it added.

KLPO said Kymriah, which was the nations first high-tech biological drug to win the license and health insurance coverage, will become the role model in reimbursement for ultra-expensive CAR-T therapies.

KLPO predicted that there would be more CAR-T therapies available in the market, including Zolgensma (onasemnogene abeparvovec), a CAR-T treatment for spinal muscular atrophy (SMA) seeking reimbursement, and Luxturna (voretigene neparvovec), gene therapy for inherited retinal disease.

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Reimbursement price of CAR-T cell therapy Kymriah set at $296138 - KBR

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Partnership to advance cell therapy research – Scientist Live

Posted: April 6, 2022 at 2:00 am

Olympus, a leadingmanufacturer of optical and digital precision technology, has announced a partnership with evorion biotechnologies, a pioneer in advanced single-cell analysis using unique microfluidic systems. By merging evorions3D hydrogel bead technology and Olympusmicroscope systems, the collaboration introduces a seamless and largely automated workflow solution to capture functional phenotypes of individual cells behaviour over time. This workflow enables researchers to reveal novel insights into thousands of cell-cell interactions in a 3D microenvironment, facilitating advanced single-cell research across applications such as adoptive cell therapy, cancer research and immunology.

The combination of OlympusIXploremicroscope systems and cellSenssoftware with evorions innovative CellCity system and CellCity Scout-AI software makes high-resolution live-cell analysis applications both easy and accessible. By encapsulating thousands of single cells or cell pairs into hydrogel beads and immobilising them on evorions CellCity Array Chip, researchers can monitor and image the functional phenotypes of cells in a physiologically relevant 3D microenvironment. High-resolution images of thousands of individual live cells are made possible by Olympus IXplore microscope systems, and the workflow can accommodate the IXplore Live, IXplore Spin or IXplore SpinSR system for exceptional 3D super-resolution imaging. Fast data analysis is facilitated by theCellCity Scout-AI software, which uses AI algorithms to automatically select positions of interest on the CellCity Array Chip and send the trackable coordinates to the IXplore system for further in-depth imaging.

A powerful aspect of the workflow is the ability to perform multi-modal analyses at single-cell resolution, enabling researchersfor the first timeto correlate time-resolved functional phenotypes with immunostaining, single-molecule RNA FISH analysis in a highly parallelised manner. Biomedical researchers can now obtain a more holistic understanding of dynamic cell behavior to drive advances in therapeutics.

Dr. Sebastian Bhren, CEO of evorion biotechnologies, commented, Early on we recognised theimpact that the CellCity platform could have on research fields like adoptive cell therapy, cancer immunotherapy and solid tumor treatment. We are excited to partner with Olympus, a renowned company with a global reach. Our partnership will allow us to bring our innovative platform to a wider audience and work together in advancing single-cell research by integrating the leadingimaging systems of Olympus into our innovative CellCity workflow.

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New gene- and cell-therapy manufacturing facility to launch in Hamilton with $580-million commitment – The Globe and Mail

Posted: April 6, 2022 at 2:00 am

Ontario says it will provide a $40-million loan to the Centre for Commercialization of Regenerative Medicine for its planned $580-million Hamilton facility where life-science companies can develop and commercialize cell and gene therapies.

The CCRM, a non-profit industry group, said Thursday that the facility will be run by a new subsidiary called OmniaBio Inc., which will operate what it hopes to become the largest contract development and manufacturing facility for these therapies in Canada. Up to 2,000 people could be employed at the planned 400,000-square-foot facility by 2026, and it is expected to it take on life-sciences companies of all sizes as clients.

The Alliance for Regenerative Medicine, an international advocacy group for the sector, said in 2021 that there are nearly 1,200 cell- and gene-therapy developers worldwide with more than 1,300 continuing clinical trials. This growing field of therapies treats or prevents diseases with technologies that alter genes or cells in the human body.

CCRM said the new facility would help improve the supply of cells and other biological tools for these therapies and trials in a market where demand for them is five times greater than whats currently available.

Michael May, the chief executive officer of CCRM, said in an interview that his organization has been working toward such a facility since launching nearly a decade ago. From Day 1, we understood that to drive commercialization and create companies that stay in Ontario, we needed to build manufacturing capability and capacity, he said.

The organization has built that capacity gradually, including through a partnership with the MaRS Discovery District entrepreneurship centre and the University Health Network to manufacture therapeutics for use in clinical trials. CCRM has been working over the past three years on developing the Hamilton facility, which was first announced in 2020. There is already a pipeline of potential customers, added Mr. May, who is also OmniaBios chair.

Of the $580-million costs, he said that $480-million would come from the private sector for real estate and construction at the McMaster Innovation Park. The remaining $100-million would be directed toward OmniaBios operations, and includes the $40-million loan from the province and a further $60-million from the private sector.

Economic Development Minister Vic Fedeli said that OmniaBio was the first-ever client for the provinces new Invest Ontario agency, which has earmarked $400-million to encourage businesses to set up in the province over the next four years. He told The Globe and Mail that he hoped the loan would be a signal to other businesses that his government wants to establish Ontario as a biomanufacturing hub.

It tells all of the vaccine and medical manufacturers that were open for business, he said. But it also tells the Ontario patients that theyre going to be able to have access to breakthrough technology, innovative medicines, right here with a with a made-in-Ontario stamp on it.

The provincial Progressive Conservatives used the Thursday OmniaBio news to announce they would make efforts to encourage life-sciences companies to set up in Ontario, establishing a council of medical experts and private-sector leaders to guide its work. The government said it plans to bring more vaccine, medicine, personal-protective-equipment and medical-supply manufacturing to the province.

The province also said it would work to encourage more Ontario businesses to commercialize their research, and to more easily allow locally made innovations to be used in the health care system removing roadblocks that Mr. Fedeli acknowledged in March could be a problem for innovators in the province.

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New gene- and cell-therapy manufacturing facility to launch in Hamilton with $580-million commitment - The Globe and Mail

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Global CAR-T Cell Therapy Markets, Competition, Forecast and Opportunities, 2027 – Rapidly Growing Clinical Trial Activities, Proliferative M&A…

Posted: April 6, 2022 at 2:00 am

DUBLIN, April 4, 2022 /PRNewswire/ --The "Global CAR-T Cell Therapy Market, By Product Type, By Tumor Type, By Indication, By Treatment Type, By Targeted Antigen, By End User, By Region, Competition, Forecast and Opportunities, 2017-2027" report has been added to ResearchAndMarkets.com's offering.

The Global CAR-T Cell Therapy Market value in the year 2021 was valued at USD1709 million that is anticipated to grow with CAGR of 64.66% during the forecast period, 2023-2027 to achieve market value of USD33153.30 million by 2027F.

Increasing instances of cancer among the world population and thus increasing death rate due to cancer is driving the growth of the Global CAR-T Cell Therapy Market in the upcoming five years. Rapidly growing clinical trial activities, proliferative M & A activities, and lucrative IPOs are majorly responsible for the growth of the Global CAR-T Cell Therapy Market in the next five years.

Consistent researches on cancer and its treatment along with the advancement in the therapeutics are further substantiating the growth of the Global CAR-T Cell Therapy Market in the future five years. Increasing demand for the effective cancer treatment for the growing number of people suffering from cancer along with the flourishing pharmaceutical industry producing products and therapies for the cancer are also responsible for the growth of the Global CAR-T Cell Therapy Market in the forecast years through 2027.

Although side-effects of the CAR-T cells therapy and high cost of treatment making it unaffordable to the financially challenged population may restrain the growth of the market. Government schemes, tax incentives, along with insurance policies for the treatment and increasing number of NGOs working for providing in-expensive treatment to the patients lacking financial support may aid the growth of the Global CAR-T Cell Therapy Market in the forecast period.

The Global CAR-T Cell Therapy Market is segmented by product type, tumor type, indication, treatment type, targeted antigen, end-user, regional distribution, and competition landscape. Based on treatment type, the market is further segmented into single treatment and combination treatment.

Single treatment is anticipated to hold the largest revenue shares of the market and dominate the market segment in the upcoming five years on grounds of increasing cases of cancer among the population. Increasing awareness about the cancer diagnostics and early recognition of the tests are responsible for the patients opting for single treatment.

Cancer in early stages can be treated with single treatments. The combination treatments are recent developments where multiple therapies are involved together to stop the cancer tumor development, to remove the lumps, and even alter the genetic information.

Objective of the Study:

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Years considered for this report:

CAR-T Cell Therapy, By Product Type:

CAR-T Cell Therapy Market, By Tumor Type:

CAR-T Cell Therapy Market, By Indication:

CAR-T Cell Therapy Market, By Treatment Type:

CAR-T Cell Therapy Market, By Targeted Antigen

CAR-T Cell Therapy Market, By End-User:

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Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Global CAR-T Cell Therapy Market.

For more information about this report visit https://www.researchandmarkets.com/r/sesih9

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Global CAR-T Cell Therapy Markets, Competition, Forecast and Opportunities, 2027 - Rapidly Growing Clinical Trial Activities, Proliferative M&A...

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