Receiving CAR-T therapy sooner improves lymphoma survival, according to study published in New England Journal of Medicine – Newswise

Newswise Astudy published this month in the New England Journal of Medicine (NEJM)may change the standard of care for adults who relapse from large B-cell lymphoma.

"This is a paradigm shift," saidJoseph McGuirk, D.O., medical director of the blood and marrow transplant program at The University of Kansas Cancer Center, which is one of the leading sites in enrolling participants in the trial. McGuirk is a co-author on the study.

According to the results of a clinical trial published Dec. 11 and presented at the American Society of Hematology (ASH) annual meeting the same day, a form of chimeric antigen receptor T-cell (CAR-T) therapy known by its brand name, Yescarta (axicabtagene ciloleucel), is significantly more effective than the current standard of care in treating people with large B-cell lymphoma (LBCL) who relapse after the first line of treatment.

Yescarta, made by Kite Pharmaceuticals, was first approved by the U.S. Food and Drug Administration in 2017 as a third-line treatment for adults with LBCL, i.e., for those who had already undergone two rounds of treatment that failed. The goal of the ZUMA-7 trial was to determine if a one-time infusion of Yescarta is superior to the existing, longstanding second-line standard of care, which is a stem cell transplant following high-dose chemotherapy to kill the lymphoma.

The two-year follow-up results indicate that it is. About 40% of people with LBCL need such second-line treatment, either because their cancer returns or does not respond adequately to frontline treatment.

"We're seeing upfront responses that are significantly better than what we see with autologous stem cell transplants, McGuirk said.

The two-year follow-up data shows that median event-free survival, defined as survival without disease progression or need for a new lymphoma treatment, for people receiving Yescarta was quadruple that of those receiving standard of care: 8.3 months for the Yescarta group versus 2 months for standard-of-care.

CAR-T therapy is a novel type of immunotherapy in which blood is drawn from a patient, and then T-cells (white blood cells that detect disease-causing organisms in the body) are extracted and genetically re-engineered. These turbocharged cells are used to create the drug that is injected back into the patient to attack cancer.

ZUMA-7 began in 2017 and enrolled 359 people in 77 trial sites around the world. Participants ranged in age from 22 to 81. Nearly one-third of them were 65 and older, an age that in some countries would render them ineligible for a stem cell transplant.

"I believe we are likely to see the field quickly move away from autologous stem cell transplant toward CAR-T cell therapy as a second-line therapy for relapsed or refractory diffuse large B-cell lymphoma," McGuirk said.

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Receiving CAR-T therapy sooner improves lymphoma survival, according to study published in New England Journal of Medicine - Newswise

Cell Therapy Market becoming larger and Massively Growing up with CAGR of 25.6% mainlander.nz – mainlander.nz

Cell Therapy Market

According to a new report published by Allied Market Research, titled, Cell Therapy Marketby Cell Type, Therapy Type, Therapeutic Area, End User, and Region: Opportunity Analysis and Industry Forecast, 20202027,The global cell therapy market accounted for $7,754.89 million in 2019, and is expected to reach $48,115.40 million by 2027, registering a CAGR of 25.6% from 2020 to 2027.

Cell therapy is a technology, which relies on replacing diseased or dysfunctional cells with healthy functioning ones. Cells mainly used for such advanced therapies are stem cells, owing to their ability to differentiate into specific cells required for repairing damaged or defective tissues or cells. Moreover, cell therapy finds its application in development of regenerative medicines, which is a multidisciplinary area aimed at maintenance, improvement, or restoration of cells, tissues, or organ functioning, using methods mainly related to cell therapy.

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Technological advancements for decentralizing manufacturing of cell therapy is anticipated to significantly benefit the market. Furthermore, MiltenyiBiotec is one of the 25 companies that has contributed to decentralization in manufacturing through its CliniMACS Prodigy device. The device is an all-in-one automated manufacturing system that exhibits capability of manufacturing various cell types.

By cell type, the stem cell segment is anticipated to dominate the global market during the forecast period, as these are gaining popularity from initiatives taken by various governments. The number of stem cell banks are increasing in developing nations, which further propels the market growth. In addition, rise in awareness regarding storage of stem cells also have a positive effect on the cell therapy market.

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3) Moreover, nationwide lockdowns forced manufacturing facilities to partially or completely shut their operations.

4) Adverse impacts of the COVID-19 pandemic have resulted in delays in activities and initiatives regarding development of reliable and innovative drone analytics systems globally.

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Allied Market Research (AMR) is a full-service market research and business-consulting wing of Allied Analytics LLP based in Portland, Oregon. Allied Market Research provides global enterprises as well as medium and small businesses with unmatched quality of Market Research Reports and Business Intelligence Solutions. AMR has a targeted view to provide business insights and consulting to assist its clients to make strategic business decisions and achieve sustainable growth in their respective market domain.

We are in professional corporate relations with various companies and this helps us in digging out market data that helps us generate accurate research data tables and confirms utmost accuracy in our market forecasting. Each and every data presented in the reports published by us is extracted through primary interviews with top officials from leading companies of domain concerned. Our secondary data procurement methodology includes deep online and offline research and discussion with knowledgeable professionals and analysts in the industry.

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Cell Therapy Market becoming larger and Massively Growing up with CAGR of 25.6% mainlander.nz - mainlander.nz

City of Hope presents leading-edge research on blood cancer therapies and its vaccine to reduce stem cell transplant complications at American Society…

DUARTE, Calif.--(BUSINESS WIRE)--City of Hope doctors presented data on an investigational bispecific antibody for multiple myeloma and the CMVPepVax, a City of Hope-developed vaccine against the cytomegalovirus, at this years ASH Annual Meeting.

City of Hope continues to be a leader in innovative research on investigational immunotherapies for blood cancers and improving stem cell transplants, said Eileen Smith, M.D., City of Hopes Francis & Kathleen McNamara Distinguished Chair in Hematology and Hematopoietic Cell Transplantation. New research at this years ASH conference includes promising investigational immunotherapies for lymphoma, multiple myeloma, leukemia and other blood cancers and an update on a City of Hope-developed vaccine to prevent a virus that can cause serious complications in stem cell transplant recipients.

Here are highlights of City of Hope research presented at the ASH conference:

Investigational bispecific antibody for multiple myeloma is well-tolerated and effective

Bispecific antibodies are an emerging immunotherapy against blood cancers. City of Hopes Elizabeth Budde, M.D., Ph.D., presented at this years ASH conference on mosunetuzumab. The research demonstrated that mosunetuzumab is a safe and effective investigational bispecific antibody for follicular lymphoma.

Talquetamab is an investigational therapy that is also demonstrating encouraging results for the treatment of relapsed multiple myeloma, according to a study led by Amrita Krishnan, M.D., director of the Judy and Bernard Briskin Center for Multiple Myeloma Research at City of Hope and chief, Division of Multiple Myeloma.

Talquetamab targets the G protein-coupled receptor family C group 5 member (GPRC5D) that has a high expression on malignant plasma cells and is limited on normal human tissue. The first-in-class bispecific antibody directs T cells to kill multiple myeloma cells by binding to both GPRC5D and CD3 receptors.

Patients with relapsed or difficult to treat multiple myeloma in the Phase 1 study received recommended Phase 2 doses as an injection on a weekly or biweekly basis. By increasing the doses slowly, researchers hope that will help to minimize the severity of cytokine release syndrome.

Krishnan presented data on 55 patients. For the study, 30 patients who received the therapy weekly (and their results were evaluable, meaning they could be included in the study) and 23 people who received it on a biweekly schedule were included. The study is ongoing.

In the weekly cohort, the overall response rate was 70% and there was a very good partial response or better in 57% of patients.

The response numbers are very strong and whats also remarkable is that the responses were durable and deepened over time in both groups, Krishnan said.

Cytokine release syndrome occurred in 73% of the weekly dose cohort, but only one patient had a severe case and it was treatable. Other side effects included neutropenia and dysgeusia.

We are excited that our results demonstrated that talquetamab is well-tolerated and highly effective at the Phase 2 dose level and with tolerable side effects, Krishnan said.

Further studies of the therapy on its own or in combination with other treatments for multiple myeloma are underway.

City of Hope-developed vaccine to prevent cytomegalovirus shows safety, tolerability

Despite therapies to help prevent the cytomegalovirus (CMV), which can flare up in blood marrow/stem cell transplant recipients who are immunocompromised, CMV infections are one of the most common complications in these patients. Furthermore, the antiviral drugs used to prevent flare-ups are toxic, expensive and increase the risk of other opportunistic infections.

City of Hope has developed an anti-CMV vaccine, known as CMVPepVax. At this years ASH conference, the results of a Phase 2 trial using CMVPepVax were reported by Ryotaro Nakamura, M.D., City of Hopes Jan & Mace Siegel Professor in Hematology & Hematopoietic Cell Transplantation in the Division of Leukemia.

The double blinded, placebo-controlled, randomized Phase 2 trial enrolled stem cell transplant recipients from four transplant centers, including City of Hope. Nakamura reported on data from 32 patients in the vaccine arm and 29 patients in the placebo arm.

CMVPepVax was delivered via injections 28 days after transplant and 56 days after the procedure.

Trial results demonstrated that there was no difference in CMV reactivation in both arms.

CMVPepVax was well-tolerated in patients with no increase in adverse side effects. Transplant outcomes were also similar between the two groups when comparing one-year overall survival, relapse-free survival, nonrelapse mortality, relapse and acute graft-versus-host disease (GVHD).

Significantly higher levels of CMV-targeting T cells were measured in patients in the vaccine arm who did not have CMV in their bloodstream. In patients who had the CMVPepVax injections, robust expansion of functional T cells also occurred.

Our results confirm that CMVPepVax is safe to use and provides an immune response, Nakamura said. Although the vaccine did not reduce the presence of CMV in the bloodstream, there were favorable CD8 T cell responses, which are protective in principle, but maybe didn't recover fast enough to prevent CMV from reactivating.

Next steps include researching whether stem cell donors who receive the vaccine can transfer immunity to patients, as well as providing a booster to patients. This may lead to faster immune responses after transplant.

Using probiotics for stem cell transplant patients

City of Hope is a leader in bone marrow and stem cell transplantation it was one of the first cancer centers nationwide to perform a bone marrow transplant and has performed more than 17,000 bone marrow/stem cell transplants since 1976. Because of this leadership, City of Hope doctors and scientists are investigating how to make the transplant process better, as well as how to deal with complications that may arise from the procedure, such as GVHD.

Led by Karamjeet S. Sandhu, M.D., an assistant professor in City of Hope's Division of Leukemia in the Department of Hematology & Hematopoietic Cell Transplantation, a City of Hope study examined how adding the probiotic CBM 588 to transplant recipients diets might decrease inflammation in the gut and lower the risk of GVHD. The results were discussed in an oral presentation at the ASH conference.

Sandhu explained that the body hosts microbial communities, known as the microbiome. These microbes help the body in several metabolic processes, such as digesting food, strengthening the immune system, protecting against other bacteria and producing vitamins, including B vitamins.

Recent studies have shown the microbiome can play a role in cancer risk and how a persons body responds to cancer treatment. In people with blood cancers who receive a transplant, there is a direct link between the health of microbiome and survival.

Imbalance among these microbial species have also been associated with several transplant complications including GVHD, said Sandhu, M.D. He added that the imbalance also contributes to morbidity and mortality.

For the study, Sandhu and his team used Clostridium Butyricum Miyairi 588 (CBM588), a probiotic strain that has been used in Japan for several decades to manage diarrhea caused by antibiotics or infections. CBM588 is a butyrate-producing bacteria present in the spore form in soil and food. Administration of CBM588 has shown anti-inflammatory and immune modulating effects, as well as evidence of anti-cancer activity.

This was the first study of CBM588 among bone marrow/stem cell transplant recipients. Fifteen patients received the current standard of care therapies to prevent GVHD and 21 received CBM588 in addition to standard of care for GVHD.

Our study demonstrated that CMB588 is safe and feasible to use in this patient population without increasing mortality, Sandhu said. We even noted an improvement in gastrointestinal GVHD, but further studies are needed to prove the effect and mechanism of action among recipients of bone marrow/stem cell transplantation.

Joint study examines somatic mutations in CMML patients, impact on stem cell transplants

Chronic myelomonocytic leukemia (CMML) is a rare form of leukemia that primarily affects older adults. The only potential cure at this time is allogeneic hematopoietic cell transplantation, also known as a stem cell transplant.

Research has shown that somatic mutations genetic changes that are acquired during life and not inherited are an important factor in determining prognosis for CMML patients. However, limited data are available regarding their impact on outcomes after CMML patients receive transplant.

A joint study between City of Hope and Center for International Blood and Marrow Transplant Research (CIBMTR) analyzed the relationship between somatic mutations in CMML and their impact on stem cell transplants.

Additionally, the study aimed to evaluate two separate scoring systems commonly used in nontransplant CMML patients, the CMML-specific prognostic scoring system (CPSS) and molecular CPSS (CPSS-Mol), which takes into account the somatic mutations, to find out if they can predict the results of a transplant.

Led by City of Hopes Matthew Mei, M.D., an associate professor in City of Hopes Division of Lymphoma, Department of Hematology & Hematopoietic Cell Transplantation, the study included 313 patients across 78 different transplant centers, all of whom underwent a comprehensive mutation analysis of 131 genes performed at City of Hope under the supervision of Raju K. Pillai, M.D., director of Pathology Core Laboratories in Beckman Research Institute of City of Hope.

The study found that 93% of patients had at least one mutation identified, and the median number of mutations was three. The most frequently mutated genes were ASXL1 (62%), TET2 (35%), KRAS/NRAS (33% combined) and SRSF2 (31%); TP53 was mutated in 3% of patients.

Both the CPSS and CPSS-Mol were predictive of overall survival after transplant; however, neither system was able to identify patients who were at an increased risk of relapse. Furthermore, the incorporation of somatic mutations did not appear to refine the prognosis.

Our study is the largest analysis of CMML patients who underwent a stem cell transplant with paired mutation analysis, Mei said. Overall, patients with CMML remain at high risk for relapse after transplant. Novel therapies aimed at decreasing relapse and making transplants safer, as well as improved methods of predicting outcomes of transplant in CMML, are still critically needed.

Additional research on chimeric antigen receptor (CAR) T therapy and stem cell transplantation presented at ASH

Tanya Siddiqi, M.D., director of City of Hope's Chronic Lymphocytic Leukemia Program, also presented a poster on the Transcend NHL 001 trial at the ASH conference, and Ibrahim Aldoss, M.D., associate professor, City of Hope's Division of Leukemia, presented a poster on the outcomes of allogeneic hematopoietic cell transplantation in adults with Ph-like acute lymphoblastic leukemia.

City of Hope is a leader in blood cancer research and treatment. The National Cancer Institute-designated comprehensive cancer center has performed more than 17,000 bone marrow/stem cell transplants and is a leader in chimeric antigen receptor (CAR) T therapy, with nearly 800 patients treated with immune effector cells, including CAR T therapy, and nearly 80 open or completed trials.

About City of Hope

City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin, monoclonal antibodies and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope is ranked among the nations Best Hospitals in cancer by U.S. News & World Report. Its main campus is located near Los Angeles, with additional locations throughout Southern California and in Arizona. Translational Genomics Research Institute (TGen) became a part of City of Hope in 2016. AccessHope, a subsidiary launched in 2019, serves employers and their health care partners by providing access to NCI-designated cancer center expertise. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

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City of Hope presents leading-edge research on blood cancer therapies and its vaccine to reduce stem cell transplant complications at American Society...

This Startup Is Makingand ProgrammingHuman Cells – WIRED

Our cells are packed with unrealized potential. Almost every human cell contains the genetic information it needs to become any other kind of cell. A skin cell, for example, has the same genes as a muscle cell or a brain neuron, but in each type of cell only some of those genes are switched on, while others remain silent. Its a little like making different meals out of the same ingredients cupboard. If we understand the recipe behind each type of cell, then theoretically we can use this information to engineer every single cell type in the human body.

That is Mark Kotters goal. Kotter is the CEO and cofounder of bit.bioa Cambridge, UK, based company that wants to revolutionize clinical research and drug discovery by producing precisely engineered batches of human cells. Basic scientific research into new drugs and treatments often starts with tests in mice, or in the most widely used human cell lines: kidney cells and cervical cancer cells. This can be a problem, because the cells being experimented on may have major differences to the cells that a candidate drug is supposed to target in the human body. A drug that works in a mouse may turn out not to work when it's tested in humans. There is no mouse on this planet that has ever suffered from Alzheimers, it just doesnt exist, Kotter says. But testing a potential Alzheimers drug on a human brain cell engineered to have signs of Alzheimers disease could give a much clearer indication of whether that drug is likely to be successful.

Every cell type has its own little program, or postcodea combination of transcription factors that defines it, says Kotter. By inserting the right program into a stem cell, researchers can activate genes that code for these transcription factors and turn a stem cell into a specific type of mature cell. Unfortunately, biology has a way of fighting back. Cells often silence these genes, stopping the transcription factors from being produced. Kotters solutiondiscovered as part of his research at the University of Cambridgeis to insert this program in a region of the genome thats protected against gene silencing, something Kotter refers to as a genetic safe harbor.

Bit.bio currently sells two different reprogrammed cell lines: muscle cells and a specific kind of brain neuron, but the plan is to create bespoke cell lines for use in the pharmaceutical industry and academic research. What were doing with our partners in the industry now is to create genetic modifications that are relevant for diseases, Kotter says. He compares this approach to running software on a computer. By inserting the right bit of code into a cells genome, you can control how that cell behaves. That means that we can now run programs, and we can reprogram human cells, Kotter says. The cell reprogramming technology could also go well beyond model cell lines and help develop whole new kinds of treatment, such as cell therapy.

In some cell therapies, a patients own immune cells are grown outside of their body before being modified and inserted back into it to help fight a diseasea long and expensive process. One kind of cell therapy used to treat young people with leukemia costs more than 280,000 ($371,400) per patient. Bit.bios chief medical officer Ramy Ibrahim says that the firms technology could help drive down the cost of cell therapy and make it easier to manufacture immune cells at a large scale. Having abundant numbers of the right cell types that we can now make edits to, I think will be transformational, he says.

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This Startup Is Makingand ProgrammingHuman Cells - WIRED

Daiichi Sankyo Authorizes the First YESCARTA (Axicabtagene Ciloleucel) CAR T-cell Therapy Treatment Site in Japan – Business Wire

SANTA MONICA, Calif. & TOKYO--(BUSINESS WIRE)--Kite, a Gilead Company, and Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) today announced that YESCARTA (axicabtagene ciloleucel), a chimeric antigen receptor (CAR) T-cell therapy, will be available to patients with relapsed or refractory large B-cell lymphomas in Japan through the first treatment center now authorized by Daiichi Sankyo. Kite and Daiichi Sankyo will also build on the exclusive licensing deal for commercialization rights for axicabtagene ciloleucel in Japan, formalized in January 2017. Both partners are pleased to agree on a broadening of their business collaboration in Japan.

We are pleased to bring the benefits of axicabtagene ciloleucel to eligible patients in Japan, in collaboration with Daiichi Sankyo, said Warner Biddle, Kites Global Head of Commercial. Japan has the second-largest number of people diagnosed with non-Hodgkin lymphoma globally1 and we remain committed to bringing our innovative CAR T-cell therapies to additional new markets.

We are pleased to be able to deliver axicabtagene ciloleucel, Daiichi Sankyo's first cell therapy product, to patients in Japan, said Akio Sakurai, Daiichi Sankyo Corporate Officer, Head of Sales Division. By strengthening our collaboration with Kite, the originator of axicabtagene ciloleucel and a world leader in cell therapy, we will strive to bring this innovative therapy to as many patients as possible.

CAR T-cell therapy is a complex immunotherapy, and all hospitals must complete a rigorous training process before administering axicabtagene ciloleucel to patients. These hospitals receive specific training in handling and risk minimization procedures in order to ensure that patient safety remains a priority.

Several factors are considered when qualifying a hospital, including their specialist skills and services, geographic coverage and experience in managing other complex procedures, such as stem cell transplantation and a co-located intensive care unit.

Axicabtagene ciloleucel has been approved in Japan for treatment of patients with relapsed or refractory diffuse large B-cell lymphoma, primary mediastinal B-cell lymphoma, transformed follicular lymphoma or high-grade B-cell lymphoma. The use of axicabtagene ciloleucel is limited to patients not previously treated with a CD-19 CAR-positive T-cell infusion; patients previously treated with two or more lines of treatment including chemotherapy or an autologous stem cell transplant; and, patients ineligible for an autologous stem cell transplant. In January 2017, Daiichi Sankyo received exclusive development, manufacturing and commercialization rights for axicabtagene ciloleucel in Japan from California-based Kite, a Gilead Company.

The approval of axicabtagene ciloleucel in Japan is based on data from the global pivotal trial conducted by Kite (ZUMA-1)2 and results of a Phase 2 study conducted by Daiichi Sankyo in Japan. In the Japanese Phase 2, open-label, single-arm study, the same dose (2.0 x 106 cells/kg) of axicabtagene ciloleucel as used in the ZUMA-1 study was administered to assess efficacy and safety in 16 Japanese patients with relapsed or refractory large B-cell lymphoma, including diffuse large B-cell lymphoma, primary mediastinal B-cell lymphoma, transformed follicular lymphoma or high-grade B-cell lymphoma. The study reached its primary endpoint, demonstrating an objective response rate (ORR) of 86.7% (95% CI: 59.5 98.3%).

The overall safety and tolerability profile of axicabtagene ciloleucel in the Japan trial was consistent with that observed in ZUMA-1. Dose limiting toxicity was not observed. Grade 3 treatment emergent adverse event occurred in all patients; most commonly neutropenia (81.3%), lymphopenia (81.3%) and thrombocytopenia (62.5%). Cytokine release syndrome (CRS), a typical CAR T-cell therapy-emergent adverse event, occurred in 13 patients (81.3%, all Grade), with Grade 3 CRS in one patient (6.3%). No neurological events, another CAR T-cell therapy-emergent adverse event, were observed.

About YESCARTA

YESCARTA (axicabtagene ciloleucel) is a CAR T-cell therapy directed against CD19 (a cell membrane protein), which harnesses a patients own immune system to fight cancer. Axicabtagene ciloleucel is made by removing a patients T cells and engineering them in the lab to express chimeric antigen receptors so that they can recognize and destroy cancer cells. The CAR T therapy is manufactured specifically for each patient and administered only once.3

Axicabtagene ciloleucel received Orphan Drug Designation from the Japan MHLW in 2018 for the treatment of diffuse large B-cell lymphoma, primary mediastinal B-cell lymphoma, transformed follicular lymphoma and high-grade B-cell lymphoma.

YESCARTA is approved in the U.S. and Europe for patients with certain types of relapsed or refractory B-cell lymphoma, where it is developed, manufactured and commercialized by Kite.

Please see full U.S. Prescribing Information, including BOXED WARNING and Medication Guide.

Yescarta is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:

Limitations of Use: Yescarta is not indicated for the treatment of patients with primary central nervous system lymphoma.

About Daiichi Sankyo

Daiichi Sankyo is dedicated to creating new modalities and innovative medicines by leveraging our world-class science and technology for our purpose to contribute to the enrichment of quality of life around the world. In addition to our current portfolio of medicines for cancer and cardiovascular disease, Daiichi Sankyo is primarily focused on developing novel therapies for people with cancer as well as other diseases with high unmet medical needs. With more than 100 years of scientific expertise and a presence in more than 20 countries, Daiichi Sankyo and its 16,000 employees around the world draw upon a rich legacy of innovation to realize our 2030 Vision to become an Innovative Global Healthcare Company Contributing to the Sustainable Development of Society. For more information, please visit: http://www.daiichisankyo.com.

About Kite

Kite, a Gilead Company, is a global biopharmaceutical company based in Santa Monica, California, with manufacturing operations in North America and Europe. Kites singular focus is cell therapy to treat and potentially cure cancer. As the cell therapy leader, Kite has more approved CAR T indications to help more patients than any other company. For more information on Kite, please visit http://www.kitepharma.com.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including Kites ability to realize the anticipated benefits from the collaboration with Daiichi Sankyo or other investments in cell therapy; Kites ability to initiate, progress or complete clinical trials or studies within currently anticipated timelines or at all, including those involving YESCARTA; the possibility of unfavorable results from ongoing or additional clinical trials or studies, including those involving YESCARTA; the risk that physicians may not see the benefits of prescribing YESCARTA; the possibility that the treatment center may experience disruptions or difficulties in delivering YESCARTA to patients; and any assumptions underlying any of the foregoing. These and other risks, uncertainties and factors are described in detail in Gileads Quarterly Report on Form 10-Q for the quarter ended September 30, 2021, as filed with the U.S. Securities and Exchange Commission. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The reader is cautioned that any such forward-looking statements are not guarantees of future performance and involve risks and uncertainties and is cautioned not to place undue reliance on these forward-looking statements. All forward-looking statements are based on information currently available to Gilead and Kite, and Gilead and Kite assume no obligation and disclaim any intent to update any such forward-looking statements.

U.S. Prescribing Information for YESCARTA including BOXED WARNING, is available at http://www.kitepharma.com and http://www.gilead.com.

Kite, the Kite logo, YESCARTA, and GILEAD are trademarks of Gilead Sciences, Inc. or its related companies.

For more information on Kite, please visit the companys website at http://www.kitepharma.com Follow Kite on social media on Twitter (@KitePharma) and LinkedIn.

_________

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Daiichi Sankyo Authorizes the First YESCARTA (Axicabtagene Ciloleucel) CAR T-cell Therapy Treatment Site in Japan - Business Wire

Communication between cells plays a major role in deciding their fate > News > USC Dornsife – USC Dornsife College of Letters, Arts and Sciences

Findings from a new study could point the way to new treatments for blood diseases including cancers such as leukemia and lymphoma. [3 min read]

By Darrin S. Joy - December 22, 2021

In this schematic, cells (black spheres) within each well are committed to a specific fate, but external stimuli, such as cell-to-cell communication, can force cells out of one state and into another. (Illustration: Courtesy of Adam MacLean.)

Scientists have found a way to prove that biochemical signals sent from cell to cell play an important role in determining how those cells develop.

The study from researchers at the USC Dornsife College of Letters, Arts and Sciences was published in the journal Development on Dec. 22.

A little background:

Whats new:

We discovered that the communication process can change the formation of blood cell types dramatically, and that cells that are closer to one another have a greater influence on each others fate, MacLean said.

A controversy resolved

Researchers trying to determine what early factors nudge a cell down one developmental path or another have wondered if random fluctuations within the cell are enough to decide which path is taken. Many models have suggested they were, but recent breakthrough studies showed that random fluctuations were not enough, that something else drives cells toward their fate.

The model MacLean and Rommelfanger have developed appears to put an end to the controversy altogether. They show that cell-to-cell communication can, in fact, be the deciding factor that sets cells along a certain path.

Why it matters:

By understanding how blood cell fate decisions are made, MacLean said, we get closer to being able to identify leukemia cells of origin, and in theory we can design strategies to control or alter cell fate decision-making and stop the development of cancer.

The research could help improve cancer therapies such as bone marrow transplant.

Better understanding stem cell fate decisions, as our study provides, could provide new insight to improve clinical outcomes for these diseases, MacLean said.

More than just blood

This new model has important implications beyond the blood system.

Our model is broadly applicable, so researchers working on other cell types can apply it to find out for those other cells how important cell-to-cell communication may be, said MacLean.

Whats next:

The role of cell-to-cell communication in determining cell fate is in its nascent stages, says MacLean, but further experiments and future technologies to integrate these new types of data with sophisticated models should help expand understanding.

In addition, the team is developing methods to study the regulation of key genes involved in cell fate decisions, which should further advance their overall theoretical model.

About the study

This work was supported by National Science Foundation grant DMS 2045327 and a USC Women in Science and Engineering Top-up Fellowship.

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Communication between cells plays a major role in deciding their fate > News > USC Dornsife - USC Dornsife College of Letters, Arts and Sciences

Emendo Biotherapeutics and Seattle Childrens Research Institute Announce Collaboration to Develop CRISPR-based Therapeutic Strategy for Severe…

Collaboration formed to conduct preliminary research to inform protocol development ahead of gene editing clinical trial

NEW YORK & SEATTLE, December 22, 2021--(BUSINESS WIRE)--Emendo Biotherapeutics, a next-generation CRISPR biotech expanding the reach of gene editing therapeutics, and Seattle Childrens Research Institute today announced a research collaboration to investigate how hematopoietic stem cells (HSCs) extracted from patients with severe congenital neutropenia (SCN) respond to priming treatments ahead of administering a CRISPR-based therapeutic.

ELANE-related SCN, also known as SCN1, is a rare, autosomal dominant disease in which a mutation occurs in one allele of the ELANE gene, thereby preventing HSCs from differentiating into white blood cells, specifically neutrophils, which leaves the patient highly susceptible to recurrent bacterial infections, osteoporosis, developmental delays and abnormalities.

"Patients with SCN often suffer from reduced quality of life due to the lack of improvements in the standard of care," said Dr. David Rawlings, Division Chief of Immunology at Seattle Children's Hospital and Director of the Center for Immunity and Immunotherapies at Seattle Childrens Research Institute. "These children are immunocompromised, and, as a result, we feel a great sense of urgency to ensure were exploring all possible avenues towards a solution."

"Seattle Childrens collaboration with Emendo, utilizing its unique approach to edit only the mutated allele with CRISPR, will enable us to address the unmet needs of SCN at the very core," added Rawlings, who also serves as a professor of pediatrics and adjunct professor in the Department of Immunology at the University of Washington School of Medicine. "Were excited about this opportunity, and look forward to continuing the collaboration beyond this initial study."

Editing the mutated ELANE gene with CRISPR first requires overcoming a technological hurdle: Only the mutated allele must be targeted, while the healthy allele remains intact. Emendo engineered its roster of next-generation CRISPR nucleases to be biologically active and so specific that they can differentiate between two alleles of the same gene. EMD-101, Emendos lead therapeutic candidate for SCN, was specifically engineered to target the mutant ELANE allele.

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HSCs have been widely studied as a treatment for sickle cell anemia and cancer, as well as a potential therapy to treat organ and tissue damage. However, HSCs require initial priming prior to stem cell transplantation, which is typically done by administering G-CSF (granulocyte colony stimulating factor). Yet, the same drug is also a short-term treatment for SCN patients.

To better understand how SCN patients would respond to a priming dose of G-CSF and plerixafor, Emendo will evaluate the mobilization of HSCs excised from a small group of patients with SCN, which would be gene-edited later. Concurrently, Seattle Childrens will evaluate the composition of the HSCs obtained from the same patients. Prior mouse studies conducted by Emendo have shown that human cells edited to excise the disease-causing ELANE allele sufficiently engrafted and replaced existing diseased cells, restoring proper neutrophil differentiation.

"By combining our allele-specific genome editing technology with Seattle Childrens renowned expertise in SCN spearheaded by Dr. Rawlings we are laying the foundation for future clinical trials that could lead to potential therapies to treat the disease," said David Baram, Ph.D., CEO of Emendo. "Our portfolio of engineered nucleases tailored to any gene or allele gives us the unique opportunity to tackle the inherent challenges of SCN. Through this collaboration well be able to provide stronger evidence and further proof points for the capabilities of our technology."

Based on the outcome of the research, a protocol for a clinical trial could be developed with an expected initiation in late 2022, pending regulatory approval. Seattle Children's has certain preferred rights to serve as a clinical trial site.

About Emendo Biotherapeutics

EmendoBio is a next generation CRISPR gene editing company leveraging dual proprietary technology platforms to enable high precision gene editing throughout the genome. EmendoBios novel nuclease discovery platform broadens the targetable range of the genome while its target-specific optimization platform enables highly precise editing, including allele specific editing, while maintaining high efficiencies. The capabilities of the OMNI technology platforms, along with deep expertise in genomic medicine, protein engineering and therapeutic development, provide EmendoBio with a unique advantage when addressing indications within hematology, oncology, ophthalmology and other disease areas. For more information please visit us at http://www.emendobio.com.

About Seattle Childrens

Seattle Childrens mission is to provide hope, care and cures to help every child live the healthiest and most fulfilling life possible. Together, Seattle Childrens Hospital, Research Institute and Foundation deliver superior patient care, identify new discoveries and treatments through pediatric research, and raise funds to create better futures for patients.

Ranked as one of the top childrens hospitals in the country by U.S. News & World Report, Seattle Childrens serves as the pediatric and adolescent academic medical center for Washington, Alaska, Montana and Idaho the largest region of any childrens hospital in the country. As one of the nations top five pediatric research centers, Seattle Childrens Research Institute is internationally recognized for its work in neurosciences, immunology, cancer, infectious disease, injury prevention and much more. Seattle Childrens Foundation works with the Seattle Childrens Guild Association, the largest all-volunteer fundraising network for any hospital in the country, to gather community support and raise funds for uncompensated care and research. Join Seattle Childrens bold initiative It Starts With Yes: The Campaign for Seattle Childrens to transform childrens health for generations to come.

For more information, visit seattlechildrens.org or follow us on Twitter, Facebook, Instagram or on our On the Pulse blog.

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National Advertising Division Recommends Cryo-Cell Discontinue or Modify Certain Health-Related Claims for Cord Blood Banking and Treatment Services -…

PR Newswire

NEW YORK, Dec. 21, 2021

NEW YORK, Dec. 21, 2021 /PRNewswire/ -- The National Advertising Division (NAD) of BBB National Programs determined that certain advertising claims made by Cryo-Cell International, Inc. reasonably conveyed the unsupported message that families must engage the advertiser's cord blood storage services to ensure access to the advertised cord blood infusion treatments. Therefore, NAD recommended that Cryo-Cell discontinue such claims, or modify its advertising to avoid conveying the message that consumers have exclusive or superior access to advertised cord blood infusion treatments by virtue of choosing Cryo-Cell for storage services.

National Advertising Division (NAD) (PRNewsfoto/National Advertising Division,B)

The advertising at issue was challenged by ViaCord, LLC, a provider of competing cord blood banking services.

The challenged claims, which appeared on the advertiser's website, included express claims stating:

"This partnership will benefit families who store with Cryo-Cell by allowing them to have foremost access to the infusion treatments that are currently only performed at Duke University to treat autism, cerebral palsy, and other neurologic diseases in accordance with the FDA expanded access rights granted to Duke."

"What Sets Us Apart Key Partnerships Patients will have access to investigational therapies for certain conditions through our partnership with Duke University.

"Additionally, families will benefit from access to investigational therapies through the arrival of a new infusion clinic, expected in January 2022."

"Treatment Access" Cryo-Cell "[w]ill offer families expanded treatment options under an FDA-approved IND for therapies using cord blood stem cells."

"New Treatment Possibilities: Receive access to innovative and quality care."

"Access to Innovative Treatments"

"Extended Benefits for Patients access to treatment in clinical trialsExpedited Participationcritically important to provide access to therapy before the child outgrows the number of cells available for treatment."

"NOT ALL CORD BLOOD BANKS ARE CREATED EQUALkey partnerships"

ViaCord also challenged implied claims that:

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Storing cord blood with Cryo-Cell provides consumers exclusive and/or better access to treatments than storing with other cord blood banks.

Such access is because competitors lack "key partnerships" relating to such treatments.

The advertiser offers services relating to the preservation, storage and transportation of cord blood stored on behalf of families for potential future medical use. Expectant parents who choose to bank cord blood with a private bank such as Cryo-Cell register with the bank before the birth of their child. The bank helps facilitate the collection of the blood at the time of birth and the blood is then immediately taken by courier to a facility where it is then processed and stored.

Duke University holds a patent on methods of treating autism with cord blood and has granted Cryo-Cell an exclusive license to practice those treatment methods. Clinical trials of cord blood as treatment for autism and other neurologic disorders are being conducted by Dr. Joanne Kurtzberg at Duke University under the FDA's Expanded Access Protocol (EAP). The cord blood used in these trials has come from numerous cord blood banks, including both ViaCord and Cryo-Cell. Cryo-Cell is planning on opening a clinic in 2022 to provide infusion treatments to patients in accordance with the patent license and the EAP to advance regenerative therapy research and "bring greater access to novel cord blood and cord tissue-based cellular therapies to treat conditions such as autism, cerebral palsy, and other neurological conditions."

Cord blood banking and cord blood-based treatments are distinct services. Families who have stored cord blood at an FDA-compliant bank are free to use that blood for treatments offered by any treatment provider, regardless of where the blood is stored. Cord blood used for treatments is often transferred from the bank to an infusion center, even when within the same facility, where it must be processed according to required protocols.

NAD determined that one reasonable takeaway from the context of the challenged advertising, on an audience of expectant parents under time pressure to make an important health care decision for their family, is that one must engage the advertiser's cord blood storage service to ensure access to the advertised cord blood infusion treatments.

NAD found that the advertiser did not substantiate the message reasonably conveyed that Cryo-Cell storage clients have exclusive access to the treatments. Nor did the Advertiser demonstrate that Cryo-Cell customers have "better" access to the treatments than those families who store cord blood at a different bank in the sense that Cryo-Cell storage customers will be at risk of losing access to the treatments because they store their cord blood elsewhere.

NAD recommended that the advertiser discontinue the challenged claims or modify its advertising to avoid conveying the message that consumers have exclusive or superior access to the advertised cord blood infusion treatments by virtue of choosing Cryo-Cell for storage services. NAD noted that the advertiser may make this modification by clearly and conspicuously disclosing that using Cryo-Cell's storage services will not impact eligibility for the treatments, or, by modifying its website to separate its claims related to exclusive benefits for Cryo-Cell customers and access to the treatments available to all eligible patients.

Finally, NAD noted that nothing in its decision precludes the advertiser from making truthful and non-misleading claims about any convenience benefit to Cryo-Cell storage customers when accessing cord blood treatments or from advertising the exclusive license it has with Duke University and the treatments that may be available to eligible patients as a result.

In its advertiser statement, Cryo-Cell stated that it "agrees to comply with NAD's recommendations" and that it "will indeed provide additional disclosures stating that access to treatments at the Cryo-Cell Institute for Cellular Therapies, including treatments under Cryo-Cell's exclusive license with Duke University and the FDA's Expanded Access Protocol, will be available to all eligible patients, including those that have their cord blood stored at another cord blood storage provider."

All BBB National Programs case decision summaries can be found in the case decision library. For the full text of NAD, NARB, and CARU decisions, subscribe to the online archive.

About BBB National Programs: BBB National Programs is where businesses turn to enhance consumer trust and consumers are heard. The non-profit organization creates a fairer playing field for businesses and a better experience for consumers through the development and delivery of effective third-party accountability and dispute resolution programs. Embracing its role as an independent organization since the restructuring of the Council of Better Business Bureaus in June 2019, BBB National Programs today oversees more than a dozen leading national industry self-regulation programs, and continues to evolve its work and grow its impact by providing business guidance and fostering best practices in arenas such as advertising, child-directed marketing, and privacy. To learn more, visit bbbprograms.org.

About the National Advertising Division: The National Advertising Division (NAD) of BBB National Programs provides independent self-regulation and dispute resolution services, guiding the truthfulness of advertising across the U.S. NAD reviews national advertising in all media and its decisions set consistent standards for advertising truth and accuracy, delivering meaningful protection to consumers and leveling the playing field for business.

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Seven tech trends to watch in 2022 | The Star – Toronto Star

With COVID resurging and the climate crisis intensifying, 2021 is ending on a precarious note. But there are also reasons to be optimistic (or at least somewhat optimistic). Across Canada, scientists and entrepreneurs are innovating in ways that will help people strike a better work-life balance, live sustainably and stay healthy. Here are seven trends in Canadas thriving innovation economy to watch in the year ahead.

Farm-to-table becomes lab-to-table

With the supply-chain crisis expected to push family grocery bills up by an estimated $966 next year, Canada needs to find new ways to sustainably feed itself. According to Dana McCauley of the Canadian Food Innovation Network, when it comes to food, the really exciting stuff right now is happening in labs.

She points to companies, such as Vancouvers Wamame, which has produced the worlds first plant-based version of Wagyu beef and is leading a consortium to create other meatless meats. Meanwhile, researchers at the University of Guelph have discovered how to make plant-based cheese stretch, which could soon put a tastier and oozier vegan mozzarella on the menu.

Companies are taking advantage of advances in engineering yeast, algae and other microbes to create foods like animal-free milk. But researchers are also working on technologies to grow real meat from cell cultures, which are touted as being cruelty-free and better for the environment. Scientists at McMaster University recently found a way to better control the fat and muscle content of cultured meat, bringing the possibility of lab-grown steak a step closer.

McCauley points out that these techniques can also be used to grow specialized plant products, cutting down food miles: In future, instead of coming from Madagascar, maybe my vanilla will come from Montreal.

Hybrid working becomes a test of corporate culture

When the Omicron wave recedes, executives will again be mulling their return-to-the-office plans. But with 80 per cent of workers not keen on returning to their desks full time, a period of experimentation with various forms of part-office part-remote hybrid working lies ahead. Kyra Jones, head of talent at Communitech, says that successful companies will use this as an inflection point to reimagine how work is done, where work is done and what work looks like.

Toronto-based enterprise software company Sensei Labs is among a growing number of tech companies that have adopted work-from-anywhere policies. Staff are free to log on from the office, at home, in a coffee shop or even further afield. We have a very international team, many of them have family in places like Brazil, or India or Ukraine and we encourage them to go work from there for an extended period, says CEO Jay Goldman. The company also suggests which days each team might go into the office, so staff who want to be in part-time know when theyll be most likely to see their closest colleagues.

But its not just HR policies that are evolving, technology is too. Tools are now emerging designed with long-term remote working in mind. Vizetto, for instance, has created a virtual chalkboard to encourage collaborative meetings. WorkTango, an employee feedback platform, has created quick pulse surveys to make it easier for managers to check how workers are feeling. Regardless of the technology, however, hybrid working will continue to be tricky terrain for managers to navigate. Unsurprisingly, Jones says demand for leadership training courses is surging.

Lithium is the new oil

After production delays in 2021, electric-vehicle manufacturers are hoping to make up lost ground and they are all racing to secure supplies of lithium for the batteries. Amanda Hall, CEO of lithium-extraction firm Summit Nanotech, predicts global production will increase by about a third next year. But suppliers will still struggle to keep up with demand.

The downside is that soaring lithium prices could lead to sticker shock at the dealership if costs are passed on to electric-vehicle buyers. The upside: the focus on lithium is putting pressure on miners to clean up their operations.

The major investors in the world are saying we wont support unsustainable practices anymore, says Hall, who recently won Canadas $1-million Women in Cleantech Challenge for developing an extraction system that generates 90 per cent less waste than traditional chemical methods. Australia, one of the worst polluters, is looking at using electric vehicles and renewable energy in its lithium mines. As Canada considers plans for developing mineral mining in places like northern Ontario, expect environmental safeguards to come under close scrutiny here, too.

Stem cell therapies advance in clinical trials

For two decades, researchers have been experimenting with stem cells to repair damaged tissues and treat diseases. Now, those studies are leaving the lab and moving into clinical trials to see how they perform in real patients.

The long-standing investment in regenerative medicine is going to start to bear fruit but to a limited extent for a limited number of patients, says Michael Sefton, executive director of the University of Torontos Medicine by Design program.

In Vancouver, biotech firm ViaCyte is testing a device that implants pancreatic stem cells into diabetes patients, which should grow into insulin-producing cells and reduce the need for injections. And BlueRock Therapeutics, which has labs in Toronto, has started a trial using stem cells to replace damaged neurons in patients with Parkinsons Disease. If successful, these therapies could transform patients lives.

But Sefton cautions that we are still some way from seeing stem-cell treatments like these in clinical use: I think in 10 years they may be common, at least in large academic centres.

Investment in biotech picks up

After record investment poured into biotech at the start of the pandemic, 2021 was a bumpy year for the sector. Stocks of major pharmaceutical companies languished even as the industry was cranking out billions of doses of life-saving vaccines in record time.

But according to Dan Legault, CEO of Toronto-based Antibe Therapeutics, investment in biotech should pick up again in the coming year.

The pullback was too fast and too strong, he says. Legault puts the investment chill down to a lack of major mergers or acquisitions of late but says that with several promising developments on the horizon there is still huge money looking to invest in the sector.

Self-driving trucks inch closer to the road

While driverless cars may be taking longer than expected, developers of autonomous freight vehicles are stepping on the gas.

Michael Tremblay, CEO of Invest Ottawa, which operates a testing centre for driverless vehicles called AreaX.O, says interest in self-driving delivery vehicles is growing as trucking companies struggle to recruit drivers. Theres a real business need for it, he says.

At facilities like AreaX.O, researchers are now connecting up convoys of small test vehicles under the supervision of single drivers, which is seen as a potential stepping stone to fully autonomous trucks. They are also adapting driverless systems to Canadas harsh climate, including looking at centimetre-accurate GPS to enable vehicles to navigate when snow covers road markings, and testing special coatings to prevent sensors getting iced up.

For now, in Ontario, autonomous vehicles are confined to study tracks and pilot projects, and regulatory changes will be needed before driverless trucks hit our highways. But Tremblay points out that the core technology is available right now. Its a question of having society accept it.

In the meantime, researchers have discovered another uniquely Canadian challenge: geese. They dont behave the way youd expect them to they go right out in front of the vehicle, says Tremblay.

The end of writers block (maybe)

AI assistants can already help organize your life. Soon, they may be able to unlock your artistic side, too.

AI platforms have advanced to the point where they are surprisingly creative Rolling Stone recently deemed a computer-generated rock song to have an anthemic chorus.

According to Olga Vechtomova, a researcher at the University of Waterloo, its now possible to channel that creativity into a kind of electronic muse for artists. Vechtomovas lab has produced an AI model that listens to music and suggests lyrics that fit its rhythm and style. The aim is less to write the song than it is to spark musicians imaginations.

Its power is in its ability to surprise and be unpredictable, says Vechtomova. Ive seen it come up with novel metaphors and turns of phrase that would never occur to me.

There are already several platforms like LyricStudio, which uses AI to generate song lines based on topics and rhymes. Such tools are likely to proliferate in the next year or two.

But will we ever see an AI produce a masterpiece? Vechtomova has her doubts, not least because wed first need to figure out what genius actually is.

Try to set the objective to generate something thats musically brilliant we just cant define that.

David Paterson writes about technology for MaRS. Torstar, the parent company of the Toronto Star, has partnered with MaRS to highlight innovation in Canadian companies.

Disclaimer This content was produced as part of a partnership and therefore it may not meet the standards of impartial or independent journalism.

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Systemic Mastocytosis Treatments Gain Hope Due To Increasing Novel Treatment Options – PRNewswire

PALM BEACH, Fla., Dec. 21, 2021 /PRNewswire/ -- FinancialNewsMedia.com News Commentary - Systemic mastocytosis is rare disease which affects very few people and it causes due to C-kit mutation which leads to higher number of mast cell production in the body resulting in accumulation of excessive mast cells in the internal body organs such as spleen, liver, bone marrow and small intestine etc. Recently, the World Health Organization (WHO) updated the prognosis, diagnosis and systemic mastocytosis treatment guidelines for the disease which in turn helped to bring uniformity in the approach by healthcare professionals. The manufacturers in the systemic mastocytosis treatment market are focusing on evaluating possible treatment options to cure the disease by investing heavily in the research & development. Various leading manufacturers are focusing on gaining FDA approval to respective drugs for the systemic mastocytosis treatment to enhance their revenue generation. A reportfrom Future Market Insights said:"Increasing awareness about the systemic mastocytosis treatment as well as symptoms of the disease due to extended effort by non-profit organizations, governmental associations and through other platforms expected to drive the growth of the systemic mastocytosis treatment market Increasing approvals and launches of drugs for the systemic mastocytosis treatment expected to drive the growth of the market Increasing spending on research & development by various pharmaceutical companies to develop novel systemic mastocytosis treatment expected to further fuel the growth of market. Increasing early diagnosis rate subsequently followed by increasing treatment seeking rate further expected to drive the growth of the systemic mastocytosis treatment market." Active companies in the markets today include: Hoth Therapeutics, Inc. (NASDAQ:HOTH), Longeveron Inc. (NASDAQ: LGVN), Bristol Myers Squibb (NYSE: BMY), Takeda Pharmaceutical Company Limited (NYSE: TAK), Amgen (NASDAQ: AMGN).

Future Market Insights continued:"The global systemic mastocytosis treatment market is expected to experience significant growth due to increasing novel treatment options. By drug class, systemic mastocytosis treatment market is expected to be dominated by the mast cell stabilizers due to superior efficacy. By indication, systemic mastocytosis treatment market is expected to be dominated by indolent systemic mastocytosis (ISM) due to higher prevalence. By route o administration, systemic mastocytosis treatment market is expected to be dominated by injectables. By distribution channel, systemic mastocytosis treatment market is expected to be dominated by the retail pharmacies due to higher patient footfall. The global systemic mastocytosis treatment market is expected to be dominated by the North America due to comparatively higher prevalence of the disease. Europe systemic mastocytosis treatment market is expected to be second most lucrative market due to higher treatment seeking rate. Latin America expected to show gradual growth in the systemic mastocytosis treatment market due to steadily increasing diagnosis. Asia-Pacific is emerging systemic mastocytosis treatment market due to increasing diagnosis subsequently followed by treatment. Middle East & Africa is the least lucrative systemic mastocytosis treatment market due to least diagnostic rate and lower awareness about the symptoms."

Hoth Therapeutics, Inc. (NASDAQ:HOTH) BREAKING NEWS: Hoth Therapeutics Announces Submission of Orphan Designation Application for HT-KIT to Treat Mastocytosis Hoth Therapeutics, Inc., a patient-focusedclinical-stage biopharmaceutical company, announced it submitted an Orphan Drug Designation Application to the US Food and Drug Administration (FDA) for HT-KIT for the treatment of mastocyctosis. HT-KIT is an antisense oligonucleotide that targets the proto-oncogene cKIT by inducing mRNA frame shifting, resulting in apoptosis of neoplastic mast cells. The KIT signaling pathway is implicated in multiple diseases, including all types of mastocytosis (such as aggressive systemic mastocytosis (ASM), mast cell leukemia (MCL), and systemic mastocytosis with associated hematological neoplasm (SM-AHN)), acute myeloid leukemia, gastrointestinal stromal tumors, and anaphylaxis.

Drugs intended to treat orphan diseases (rare diseases that affect less than 200,000 people in the US)are eligible to apply for Orphan Drug Designation (ODD), which provides multiple benefits to the sponsor during development and after approval. Hoth intends to pursue these benefits as part of the drug development for HT-KIT for treatment of mastocytosis, pending designation of the ODD application.

Benefits of Orphan Drug Designation - Under the Orphan Drug Act, drug companies can apply for ODD, and if granted, the drug will have a status which gives companies exclusive marketing and development rights along with other benefits to recover the costs of researching and developing the drug. A tax credit of 50% of the qualified clinical drug testing costs awarded upon drug approval is also possible. Regulatory streamlining and provide special assistance to companies that develop drugs for rare patient populations. In addition to exclusive rights and cost benefits, the FDA will provide protocol assistance, potential decreased wait-time for drug approval, discounts on registration fees, and eligibility for market exclusivity after approval.

Key benefits of ODD:

Hoth recently announcedthat its novelanti-cancer therapeuticexhibited highly positive results in humanized mast cell neoplasm models, representative in vitro and in vivo models for aggressive, mast cell-derived cancers such as mast cell leukemia and mast cell sarcoma. CONTINUED Read the Hoth Therapeutics full press release by going to: https://ir.hoththerapeutics.com/news-releases

In other news and developments of note in the markets this week:

Amgen (NASDAQ: AMGN) recently announced that the U.S. Food and Drug Administration (FDA) has approved Amgen and AstraZeneca'sTezspire (tezepelumab-ekko) for the add-on maintenance treatment of adult and pediatric patients aged 12 years and older with severe asthma.

Tezspirewas approved following a Priority Review by the FDA and based on results from the PATHFINDER clinical trial program. The application included results from the pivotal NAVIGATOR Phase 3 trial in whichTezspiredemonstrated superiority across every primary and key secondary endpoint in patients with severe asthma, compared to placebo, when added to standard therapy.

Longeveron Inc. (NASDAQ: LGVN), a clinical stage biotechnology company developing cellular therapies for chronic aging-related and certain life-threatening conditions, recently announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for Lomecel-B for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart defect in infants.

ODD is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases or conditions. ODD positions Longeveron to be able to potentially leverage a range of financial and regulatory benefits, including government grants for conducting clinical trials, waiver of FDA user fees for the potential submission of a marketing application, and certain tax credits. Receiving ODD may also result in the product receiving seven years market exclusivity upon approval for use in the rare disease or condition for which the product was designated if all of the statutory and regulatory requirements are met.

Bristol Myers Squibb (NYSE: BMY) recently announced thatOrencia(abatacept) was approved by the U.S. Food and Drug Administration (FDA) for the prophylaxis, or prevention, of acute graft versus host disease (aGvHD), in combination with a calcineurin inhibitor (CNI) and methotrexate (MTX), in adults and pediatric patients 2 years of age and older undergoing hematopoietic stem cell transplantation (HSCT) from a matched or 1 allele-mismatched unrelated donor (URD).

"Orenciais the first FDA-approved therapy to prevent acute graft versus host disease following hematopoietic stem cell transplant, a potentially life-threatening complication that can pose a comparatively higher risk to racial and ethnic minority populations in the U.S. due to difficulty finding appropriately matched donors," said Tina Deignan, senior vice president, U.S. Immunology, Bristol Myers Squibb. "With this fourth indication forOrencia,Bristol Myers Squibb draws on its legacy and expertise in both immunology and hematology to deliver an important treatment option for patients in a disease with high unmet need.

Takeda Pharmaceutical Company Limited (NYSE: TAK) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the approval of intravenous (IV) vedolizumab for the treatment of adult patients with moderately to severely active chronic pouchitis, who have undergone proctocolectomy and ileal pouch-anal anastomosis (IPAA) for ulcerative colitis (UC), and have had an inadequate response with or lost response to antibiotic therapy. The CHMP opinion will now be reviewed by the European Commission. If approved, vedolizumab will become the first treatment indicated for active chronic pouchitis across the European Union.

DISCLAIMER: FN Media Group LLC (FNM), which owns and operates Financialnewsmedia.com and MarketNewsUpdates.com, is a third- party publisher and news dissemination service provider, which disseminates electronic information through multiple online media channels. FNM is NOT affiliated in any manner with any company mentioned herein. FNM and its affiliated companies are a news dissemination solutions provider and are NOT a registered broker/dealer/analyst/adviser, holds no investment licenses and may NOT sell, offer to sell or offer to buy any security. FNM's market updates, news alerts and corporate profiles are NOT a solicitation or recommendation to buy, sell or hold securities. The material in this release is intended to be strictly informational and is NEVER to be construed or interpreted as research material. All readers are strongly urged to perform research and due diligence on their own and consult a licensed financial professional before considering any level of investing in stocks. All material included herein is republished content and details which were previously disseminated by the companies mentioned in this release. FNM is not liable for any investment decisions by its readers or subscribers. Investors are cautioned that they may lose all or a portion of their investment when investing in stocks. For current services performed FNM was compensated twenty five hundred dollars for news coverage of current press release issued by: Hoth Therapeutics, Inc. by a non-affiliated third party.

FNM HOLDS NO SHARES OF ANY COMPANY NAMED IN THIS RELEASE.

This release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E the Securities Exchange Act of 1934, as amended and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. "Forward-looking statements" describe future expectations, plans, results, or strategies and are generally preceded by words such as "may", "future", "plan" or "planned", "will" or "should", "expected," "anticipates", "draft", "eventually" or "projected". You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events, or results to differ materially from those projected in the forward-looking statements, including the risks that actual results may differ materially from those projected in the forward-looking statements as a result of various factors, and other risks identified in a company's annual report on Form 10-K or 10-KSB and other filings made by such company with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and FNM undertakes no obligation to update such statements.

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