Category Archives: Cell Therapy

A perfect mix of quantitative & qualitative Cell Therapy Technologiesmarket information highlighting developments, industry challenges that competitors are facing along with gaps and opportunities available and would trend in Cell Therapy Technologiesmarket. The study bridges the historical data from 2014 to 2019 and estimated until 2025.

The Cell Therapy TechnologiesMarket report also provides the market impact and new opportunities created due to the COVID19/CORONA Virus Catastrophe The total market is further divided by company, by country, and by application/types for the competitive landscape analysis. The report then estimates 2020-2025 market development trends of Cell Therapy TechnologiesIndustry.

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The Top players are Danaher, Thermo Fisher Scientific, Merck, Terumo, BD, Lonza Group, GE Healthcare, Sartorius, Stemcell Technologies, Miltenyi Biotec, .

Market Segmentation:

Cell Therapy Technologies Market is analyzed by types like Equipment, Consumables, Others

On the basis of the end users/applications, Human, Animal

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Be the first to knock the door showing the potential that Cell Therapy Technologiesmarket is holding in it. Uncover the Gaps and Opportunities to derive the most relevant insights from our research document to gain market size.

A major chunk of this Global Cell Therapy TechnologiesMarket research report is talking about some significant approaches for enhancing the performance of the companies. Marketing strategies and different channels have been listed here. Collectively, it gives more focus on changing rules, regulations, and policies of governments. It will help to both established and new startups of the market.

The study objectives of this report are:To analyze global Cell Therapy Technologiesstatus, future forecast, growth opportunity, key market, and key players.To present the Cell Therapy Technologiesdevelopment in the United States, Europe, and China.To strategically profile the key players and comprehensively analyze their development plan and strategies.To define, describe and forecast the market by product type, market, and key regions.

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Major Points from Table of Contents

1 Cell Therapy Technologies Cell Therapy Technologies Market Overview2 Cell Therapy Technologies Market Competition by Manufacturers3 Production Capacity by Region4 Global Cell Therapy Technologies Market by Regions5 Production, Revenue, Price Trend by Type6 Global Cell Therapy Technologies Market Analysis by Application7 Company Profiles and Key Figures in Cell Therapy Technologies Business8 Cell Therapy Technologies Manufacturing Cost Analysis9 Marketing Channel, Distributors and Customers10 Market Dynamics11 Production and Supply Forecast12 Consumption and Demand Forecast13 Forecast by Type and by Application (2021-2026)14 Research Finding and Conclusion15 Methodology and Data Source.

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Latest Update 2020: Cell Therapy Technologies Market by COVID19 Impact Analysis And Top Manufacturers: Danaher, Thermo Fisher Scientific, Merck,...

Basel, June 2, 2020 Novartis today announced that it has received notice from the US Food and Drug Administration (FDA) that the agency has extended its review of the Supplemental Biologics License Application (sBLA) for ofatumumab (OMB 157), a self-administered, targeted B-cell therapy for patients with relapsing multiple sclerosis. Regulatory action is now expected in September 2020.

Novartis will continue to work with the FDA to complete the review as soon as possible, said Marie-France Tschudin, President, Novartis Pharmaceuticals. We are well prepared and ready to launch ofatumumab upon approval. We are committed to the MS community and look forward to bringing this important advancement to patients with MS.

Additional regulatory filings are currently underway and regulatory approval for ofatumumab in Europe is expected by Q2 2021.

About ofatumumabOfatumumab (OMB 157) is a fully human anti-CD20 monoclonal antibody (mAb) in development for RMS that is self-adminstered by a once-monthly injection, delivered subcutaneously1,2,7. As shown in preclinical studies, ofatumumab is thought to work by binding to a distinct epitope on the CD20 molecule inducing potent B-cell lysis and depletion3. The selective mechanism of action and subcutaneous administration of ofatumumab allows precise delivery to the lymph nodes, where B-cell depletion in MS is needed, and may preserve the B-cells in the spleen, as shown in preclinical studies4. Once-monthly dosing of ofatumumab also allows fast repletion of B-cells and offers more flexibility5. Ofatumumab was originated by Genmab and licensed to GlaxoSmithKline; Novartis obtained rights for ofatumumab from GlaxoSmithKline in all indications, including RMS, in December 20156.

DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as potential, can, will, plan, may, could, would, expect, anticipate, seek, look forward, believe, committed, investigational, pipeline, launch, or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AGs current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About NovartisNovartis is reimagining medicine to improve and extend peoples lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the worlds top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 145 nationalities work at Novartis around the world. Find out more athttps://www.novartis.com.

Novartis is on Twitter. Sign up to follow @Novartis at https://twitter.com/novartisnewsFor Novartis multimedia content, please visit https://www.novartis.com/news/media-libraryFor questions about the site or required registration, please contact media.relations@novartis.com

References1. Hauser S.Efficacy and safety of ofatumumab versus teriflunomide in relapsing multiple sclerosis: results of the phase 3 ASCLEPIOS I and II trials. Presented at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Annual Conference; September 1113, 2019; Stockholm, Sweden.2. Bar-Or A, Fox E, Goodyear A, et al.Onset of B-cell Depletion with Subcutaneous Administration of Ofatumumab in Relapsing Multiple Sclerosis: Results from the APLIOS Bioequivalence Study. Presented at Americas Committee for Treatment and Research in Multiple Sclerosis Forum; February 2729, 2020.3. Smith P, Kakarieka A, Wallstroem E.Ofatumumab is a fully human anti-CD20 antibody achieving potent B-cell depletion through binding a distinct epitope. Poster presented at ECTRIMS; September 2016; London, UK.4. Smith P, Huck C, Wegert V, et al.Low-dose, subcutaneous anti-CD20 therapy effectively depletes B-cells and ameliorates CNS autoimmunity. Poster presented at ECTRIMS; September 1417, 2016; London, UK.5. Savelieva M, Kahn J, Bagger M, et al.Comparison of the B-Cell Recovery Time Following Discontinuation of Anti-CD20 Therapies. ePoster presented at ECTRIMS; October 2528, 2017; Paris, France.6. GSK press release.GSK completes divestment of rights to ofatumumab for auto-immune indications to Novartis. December 21, 2015. Available from:https://www.gsk.com/en-gb/media/press-releases/gsk-completes-divestment-of-rights-to-ofatumumab-for-auto-immune-indications-to-novartis/%5BLast accessed: June 2020].7. Efficacy and Safety of Ofatumumab Compared to Teriflunomide in Patients With Relapsing Multiple Sclerosis (ASCLEPIOS I & II clinical trials), data on file, Novartis. https://clinicaltrials.gov/ct2/show/NCT02792218; https://clinicaltrials.gov/ct2/show/NCT02792231.

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Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com

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Novartis provides update on FDA review of ofatumumab, a self-administered, targeted B-cell therapy for patients with relapsing multiple sclerosis -...

The dying patient just needed to hold on a little while longer.

Within a few hours, she would receive the new treatment. The placenta cells a therapy that showed promise in boosting the immune system were on their way to Holy Name Medical Center from another state.

It was April, and the coronavirus crisis was intensifying. The patient, an unidentified woman, lay dying of COVID-19 at the Teaneck facility, one of New Jerseys hardest-hit hospitals.

Everyone was anxious and was excited that we would treat her, said Dr. Ravit Barkama, a clinical researcher at the hospital. And the cells were basically on the way to Holy Name from Maryland.

It was an example of the hope and heartache doctors and nurses faced in the early days of the unfolding medical crisis. They rushed to treat a crush of patients flooding the emergency room, while clinical researchers studied the latest data and searched for anything in their arsenals to keep the infected alive.

While the first phase of the COVID-19 crisis is winding down, the pandemic and the health care establishments work are far from over. Holy Name is taking a hard look at everything its medical personnel learned in enduring those treacherous first three months.

The clinical researchers recently shared a report with NJ Advance Media outlining a series of medical takeaways: Everything theyve learned after treating hundreds of coronavirus patients since the crisis began.

Its an orderly account with dense detail and precise explanations a dramatic contrast to March and April, when more than 200 patients were dying on ventilators, one after the other. When the intensive care units were filled.

When there seemed to be no clear answers.

The researchers learned the ways COVID-19 affects the body, the various stages in which it manifests and consumes patients, the medications and therapies that show promise and the treatments that proved ineffective.

Like every hospital in the state, Holy Name was desperately looking for ways to combat the disease after the pandemic hit New Jersey.

Treatment was trial and error. It was a scramble, often necessitating learning on the fly.

The first few weeks, basically, almost every night was ... working with a lot of frustration, Barkama said.

She and her counterpart, Dr. Thomas Birch, have been working as a research duo of sorts, investigating possible therapies for a deluge of COVID-19 patients.

"How are we going to treat the patients? What can we give them? How can we make this better? How can we keep people from dying? How can we keep people off of the ventilator?" Birch said.

It was an exhausting sprint of confusion and dashed hopes.

Barkama and Birch recalled late nights poring over research and data. Barkama called counterparts in Israel and Europe asking what was working.

They found insights, if not epiphanies.

One of the hard-won lessons was COVID-19 is a disease of stages.

It follows a largely defined progression in the human body, from exposure and incubation (phase 1), to the showing of symptoms (phase 2), to the virus impact on the lungs (phase 3) to the decrease of lung inflammation (phase 4).

We basically realized certain interventions might be effective in certain phases and not effective in others. And so thats the key, said Dr. Suraj Saggar, infectious disease specialist at Holy Name.

Early intervention is crucial. Antiviral drugs likely work best during the first phase when used as a pre-emptive therapy, the report said.

The antiviral drug, remdesivir, seems most effective during phase 2, the stage when the initial symptoms emerge. Convalescent plasma may also have some benefit during this stage.

The third phase is what makes COVID-19 different, the report said. This is when symptoms like shortness of breath, persistent fever and pneumonia develop. Severe inflammation begins to batter the lungs, blood vessels and organs.

The bodys overreactive immune system not the virus does most of the damage. It is why inflammatory drugs and immunosuppressive appear to help during this period.

The final stage, or the convalescent phase, is when the inflammation begins to subside and the lungs and other tissues clear the debris from inflammation, injured tissues and fluid, the report said. The stage can span three to six weeks, delaying the time when the patient can be weaned from the ventilator.

It is possible placenta-derived cells may further reduce inflammation, according to the report.

It was a tremendous amount of reading, a tremendous amount of reviewing patient cases and seeing what was going on with them clinically, their symptoms, their physical exam findings, the laboratory and imaging, said Birch, who is also an infectious disease specialist. How they responded to some of the therapies that were being used, and how we might use them more rationally.

When potential treatments emerged, such as placenta cell therapy or remdesivir, it offered hope after weeks of anguish at Holy Name.

It was no different in April as they tried to help the dying woman.

The doctors and researchers waited April 11.

The cells would arrive in just a few more hours, they were told.

The findings on placenta cell therapy were still preliminary, but the treatment championed by a company in Israel was showing promise. Six critically ill coronavirus patients in Israel had shown signs of improvement after taking it.

Some saw it as a possible dream treatment.

But then an alarm blared over Holy Names loudspeakers. It was a Code Blue, an emergency calling for the CPR team.

"It goes 'Code Blue ICU,' and then, of course, I knew that something was very wrong," Barkama said.

Her heart dropped. She rushed to see who it was.

It was the dying woman.

"A few hours before we were supposed to give the treatment, she desaturated and passed away," Barkama said.

The loss devastated her.

"When I saw it was her I must say that it was emotionally very difficult to lose a patient a few hours before the so-called 'dream treatment,'" Barkama said.

While the researchers remain hopeful about placenta-derived cell therapy, only four patients have been treated at the hospital thus far. Its too early to assess the results.

Remdesivir has also shown promise, but research remains ongoing. The National Institutes of Health has found positive results, and Gilead, the maker of the drug, said in a statement earlier this week that it found encouraging data from a recent trial, especially if used as an intervention therapy.

Holy Names staff hope the lessons pay dividends for New Jersey hospitals in case they again encounter chaos like they did in March.

Looming in the back of everyones mind is the possible resurgence of the virus in the fall, after already claiming 11,880 lives and causing 162,068 infections in New Jersey.

There is, of course, a good chance that there will be [another phase], and were not looking at this as a historical episode, Barkama said.

The research continues. Hospitals were caught flat-footed, often forced to learn and sift out new techniques on the fly, revamp old ones and apply them in new ways.

We obviously hope, just like everyone else, that it will never hit us so hard again, Barkama said. But were going to be prepared in case it does.

Spencer Kent may be reached at skent@njadvancemedia.com. Follow him on Twitter @SpencerMKent. Find NJ.com on Facebook.

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Lessons from the epicenter: How one N.J. hospital endured the worst of the pandemic and learned from it - nj.com

The world isnt just battling a health pandemic yet in addition a economic one, as the Novel Coronavirus (COVID-19) throws its long shadow over economies around the world. The whole lockdown circumstance in a few nations has legitimately or indirectly affected numerous ventures causing a move in activities like gracefully chain operations, seller tasks, product commercialization, and so forth. The Report on Adoptive Cell Therapy For Cancer Treatment Market which give detailed investigation of impact of the novel Coronavirus (COVID-19) pandemic on the historical and present/future market information.

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GlobalAdoptive Cell Therapy For Cancer Treatment market research reportprovides the latest manufacturing data and business coming trends, providing you to recognize the outcomes and end-users offering Revenue growth and profitability. The Adoptive Cell Therapy For Cancer Treatment industry report lists the leading competitors and provides the game-changing strategic analysis of the key factors driving the market. The report includes the forecasts by 2026, analysis by 2019, and discussion of significant trade, market volume, market share evaluations and outlines of the top Adoptive Cell Therapy For Cancer Treatment industry players.

Major Players in Adoptive Cell Therapy For Cancer Treatment market are:GlaxoSmithKlineGenentechMerckRocheAmgenEli LillyJanssenBristol-Myers Squibb

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Market Segmentation:

Market Segmentation by Type:

SurgeryChemotherapyRadiotherapy

Market Segmentation by Application:

Autologous adoptive T cell therapyAllogeneic Adoptive T cell therapy

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The COVID-19 Shuffle: Adoptive Cell Therapy For Cancer Treatment Market Size, Share, Applications, Regions, Top Companies, Trends, Market Drivers and...

Tumors shrank in one quarter of patients with advanced lung cancer who received a bespoke cell therapy made by doctors at H. Lee Moffitt Cancer Center in Tampa, Fla. In two patients, the researchers found, lung tumors disappeared completely.

Though the study was small, the results are impressive because the patients had tumors in their lungs that were not responding to Opdivo, one of the checkpoint-inhibitor immunotherapies that are now standard of care. That makes these patients particularly difficult to treat effectively.

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Custom cell therapy shrinks lung tumors in advanced cancer cases - STAT

On April 14, shares of Iovance Biotherapeutics jumped 17%, thanks to three magic words in the title of an abstract the company said will be presented at this weeks virtual meeting of the American Association for Cancer Research (AACR): durable complete responses.

The study, which will be discussed Tuesday morning, is a phase 1 clinical trial of Iovances highly anticipated tumor-infiltrating lymphocyte (TIL) product in non-small cell lung cancer (NSCLC). How durable were those responses? The abstract, released over the weekend, provides some clues.

Data from the study, being conducted at the H. Lee Moffitt Cancer Center, included results from 18 of 20 NSCLC patients treated with Iovances TILs after having received Bristol Myers Squibbs checkpoint inhibitor Opdivo. Two of the patients had complete responses to the therapy that are ongoing one year later, according to the abstract. Three other patients developed new lesions after undergoing the TIL therapy but went into remission after they received tumor-directed treatments.

Virtual Clinical Trials Online

This virtual event will bring together industry experts to discuss the increasing pace of pharmaceutical innovation, the need to maintain data quality and integrity as new technologies are implemented and understand regulatory challenges to ensure compliance.

TILs are personalized cell treatments made from immune cells that have a natural ability to invade and kill cancer cells. Iovances technology involves taking T lymphocytes from patients, expanding them into the billions, and then infusing them back into the body.

Iovances most advanced TIL, called lifileucel, is in pivotal trials in melanoma and cervical cancer, with FDA filings expected sometime this year. The company has simultaneously been investigating the potential of TILs in lung cancer and several other tumor types, including cervical cancer and head and neck tumors.

Because Iovance is considered a leading player in TIL technology, its investors tend to jump on any news that hints at the effectiveness of the technology. Last year, its shares rocketed up on news that its TILs produced a 44% response rate and 89% disease control in ovarian cancer. Earlier this year, Iovances stock rose 40% on rumors that it was exploring acquisition offers.

RELATED: Iovance shares jump on buyout rumors

The small study of Iovances TILs released at AACR has already raised some questions that will be hard to answer until data emerge from larger trials. Among the enrolled patients, 13 progressed on Opdivo and were given subsequent treatments combining two chemotherapy drugs with IL-1. Thus, it seems difficult to put any benefit specifically down to Iovances TILs, said an analyst from Evaluate Vantage in a recent posting.

Analysts from Jefferies pointed out in a note issued Monday that the median shrinkage of tumors observed in the study to be presented at AACR was 38%, but that the researchers did not disclose the objective response rate (ORR). "We think this means that least half of the patients observed a partial response, but we caution that we don't know if these are confirmed responses. We think anything greater than 20% ORR would be clinically meaningful," Jefferies said.

Iovances technology was originally developed at the National Cancer Institute and has so far shown the most promise in melanoma. Some patients involved in early trials have continued to respond to the therapy for more than a decade. So its no surprise Iovance has been investing heavily to prepare for the launch of lifileucel in melanoma: Last year, it announced a $75 million investment in a manufacturing facility in Philadelphia.

Meanwhile, Iovance continues to work with research partners to investigate the potential of TILs in other cancers and to improve upon the first generation of the product. Iovance teamed up with the University of Texas MD Anderson Cancer Center in 2017 to study TILs in several tumor types. And in January of this year, it licensed technology from Cellectis that it will use to genetically modify TILs in the hopes of improving their potency, the company said at the time.

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AACR: Iovance details early responses to TIL cell therapy in lung cancer - FierceBiotech

Ventilator-dependent COVID-19 patients with moderate/severe acute respiratory distress syndrome (ARDS) who were treated with intravenous infusions of an allogeneic mesenchymal stem cell product candidate had a survival rate of 83%.

The allogeneic mesenchymal stem cell product candidate remestemcel-L from Mesoblast Limited (Melbourne, Australia) was administered within the first five days under emergency compassionate use at New York Citys Mt Sinai hospital during the period March-April 2020. Patients received a variety of experimental agents prior to remestemcel-L. All patients were treated under an emergency Investigational New Drug (IND) application or expanded access protocol at Mt Sinai hospital. 75% of the patents successfully came off ventilator support within a median of 10 days, in contrast to only 9% of ventilator-dependent COVID-19 patients being able to come off ventilators with standard of care treatment and only 12% survival in ventilator-dependent COVID-19 patients at two major referral hospital networks in New York during the same time period. These poor outcomes are consistent with earlier published data from China where mortality rates of over 80% were reported in patients with COVID-19 and moderate/severe ARDS.

The remarkable clinical outcomes in these critically ill patients continue to underscore the potential benefits of remestemcel-L as an anti-inflammatory agent in cytokine release syndromes associated with high mortality, including acute graft versus host disease and COVID-19 ARDS, said Mesoblast Chief Executive Dr. Silviu Itescu. We intend to rapidly complete the randomized, placebo-controlled Phase 2/3 trial in COVID-19 ARDS patients to rigorously confirm that remestemcel-L improves survival in these critically ill patients.

There is a significant need to improve the dismal survival outcomes in COVID-19 patients who progress to ARDS and require ventilators, said Mesoblast Chief Medical Officer Dr Fred Grossman. We have implemented robust statistical analyses in our Phase 2/3 trial as recommended by the US Food and Drug Administration (FDA) in order to maximize our ability to evaluate whether remestemcel-L provides a survival benefit in moderate/severe COVID-19 ARDS.

Related Links:Mesoblast Limited

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Stem Cell Therapy Shows 83% Survival in COVID-19 Patients Dependent on Ventilator - HospiMedica

27 April 2020

Genome-edited human stem cells from diabetic patientshavebeen shown to successfully reverse diabetes in mice.

Researchers at Washington University School of Medicine in St Louis, Missouri, used CRISPR/Cas9 to correct a genetic defect in human stem cells and then converted them into cells capable of producing insulin. When these edited insulin-producing cells were implanted into diabetic mice, they were able to effectively control blood sugar levels for six months.

'We're excited about the fact that we were able to combine these two technologies - growing beta cells from induced pluripotent stemcells and using CRISPR to correct genetic defects,' said corresponding author Dr Jeffrey Millman. 'This is the first time CRISPR has been used to fix a patient's diabetes-causing genetic defect and successfully reverse diabetes.'

The human cells used were from a patient with a rare genetic type of diabetes called Wolfram syndrome, which develops during childhood and typically requires affected patients to inject insulin multiple times each day.

'For this study, we used cells from a patient with Wolfram syndrome because, conceptually, we knew it would be easier to correct a defect caused by a single gene. But we see this as a stepping-stone toward applying gene therapy to a broader population of patients with diabetes,' said Dr Millman.

The same team previously developed a new technique to more efficiently convert human stem cells into insulin-producing cells, allowing them to 'functionally cure' diabetes in mice for the first time (see BioNews1037). In the current study, they went one step further, adding the genome editing step to make cells from a diabetic personproduce insulin.

'We basically were able to use these cells to cure the problem, making normal beta cells by correcting this mutation,' said Dr Fumihiko Urano, who co-led the study. 'In fact, we found that corrected beta cells were indistinguishable from beta cells made from the stem cells of healthy people without diabetes,' added Dr Millman.

'It's also possible that by correcting the genetic defects in these cells, we may correct other problems Wolfram syndrome patients experience, such as visual impairment and neurodegeneration' said Dr Urano.

The study was published in the journal Science Translational Medicine.

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Diabetes reversed in mice using CRISPR and stem cell therapy - BioNews

I dont know why people pay attention only to vaccines and treatments against the new coronavirus. Stem cell therapies are more useful to treat Covid-19.

So claimed Lee Hee-young, president of the Korean Association of Stemcell Therapy, at a news conference in Seoul, Monday. He called for active use of stem cell therapies to treat Covid-19 patients.

Several studies have proved the effects of autologous stem cells in treating acute respiratory distress syndrome (ARDS), which is the leading cause of death in Covid-19 patients, Lee said. The concept of stem cell therapy is the same as that of blood transfusion or bone marrow transplantation. Decades of cell therapies have proved that stem cell therapy is safe.

While the development of a treatment or a vaccine against Covid-19 takes a long time and it may not be able to treat patients immediately because of virus mutation possibilities, stem cell therapies can restore damaged lungs directly, Lee claimed.

It is more important to restore damaged lungs than to fight the virus. Stem cell therapy restores the lungs, giving patients time to beat the virus, he went on to say. However, people are paying attention to vaccine or treatment candidates only. This is why I am holding a news conference.

Lee pointed out that the local environment makes it difficult to use stem cell therapies. Thus, the government should ease regulations on the management and use of cell culture facilities so that doctors can perform stem cell therapies with simple cell culture, he said.

As long as physicians have a positive pressure facility and a culture kit, they can separate and culture cells with simple training, he said. If the authorities allow doctors to perform stem cell therapies with a disposable mobile culture autonomously, the cost of stem cell therapies will go down significantly.

Lee added that he asked related officials to include such rules in the Act on Safety and Support for Advanced Regenerative Medicine and Advanced Biopharmaceuticals, which is to take effect in the second half of the year.

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'Stem cell therapy more effective on Covid-19' - Korea Biomedical Review - Korea Biomedical Review

CRANFORD, N.J., April 27, 2020 /PRNewswire/ --Citius Pharmaceuticals, Inc.. ("Citius" or the "Company") (Nasdaq: CTXR), a specialty pharmaceutical company focused on developing and commercializing critical care drug products, today announced that it submitted a pre-IND meeting request and supporting briefing documents to the Center for Biologics Evaluation and Research ("CBER") of the FDA under the Coronavirus Treatment Acceleration Program (CTAP) on April 24. The Company has requested the Division's feedback to support the most expeditious pathway into the clinic to evaluate a novel cell therapy in patients suffering from COVID-19-related ARDS.

The cells, called NoveCite Cells or NC-MSCs, are made by Novellus, Inc. ("Novellus"), a Cambridge-based biotechnology company, using its patented mRNA-based cell-reprogramming process. NC-MSCs are mesenchymal stem cells derived from a single donor's fibroblasts that have been dedifferentiated into an induced pluripotent stem cell (iPSC) master cell bank, thereby avoiding the need to source additional donor cells. The iPSCs are then further differentiated into a mesenchymal stem cell (MSC) therapy. Citius and Novellus plan to develop NC-MSCs for the treatment of ARDS, and last month the companies signed an exclusive option agreement.

The Company plans a multi-center randomized placebo-controlled dose-finding study followed by an expansion phase to assess the safety, tolerability, and efficacy of NC-MSCs in patients with moderate to severe ARDS due to COVID-19. The proposed trial, a Phase 1b/2 clinical trial, is titled "A Randomized Placebo-Controlled Dose-Finding Study Followed by a Dose Level Expansion to Assess the Safety and Efficacy of NoveCite MSCs in Subjects with Acute Respiratory Distress Syndrome (ARDS) Due to SARS-CoV-2 Disease (COVID-19)," or "MARCO". The primary objectives of this study are to evaluate the safety and efficacy of NoveCite cells as a treatment for subjects with moderate-to-severe ARDS due to COVID-19 and to identify therapeutic doses.

"MSCs have an established track-record of clinical safety, and have shown promise in the treatment of inflammatory lung disease," said Matt Angel, PhD, co-founder and Chief Science Officer at Novellus, Inc. "Our research has shown that the NoveCite cells, being derived from mRNA-reprogrammed iPSCs, secrete higher levels of immunomodulatory proteins than donor-derived MSCs, and have unique manufacturing advantages."

"We believe we have the key elements in place from a clinical design and manufacturing point of view to evaluate this novel cell therapy approach to deal with the current pandemic," said Myron Holubiak, Chief Executive Officer of Citius. "ARDS is a very serious complication for many patients suffering from COVID-19, and is believed to account for about 80% of the deaths in ventilated patients. There is no proven or FDA-approved treatment for it, other than oxygen therapy, including use of mechanical ventilation, and fluid management. Literature from previous investigational studies with MSCs in the treatment of lung injuries support the idea that MSCs could prove effective in treating COVID-19-related ARDS. We look forward to our FDA discussions and are excited to be at the cusp of what could be a novel and effective therapy for ARDS."

About Acute Respiratory Distress Syndrome (ARDS)ARDS is a type of respiratory failure characterized by rapid onset of widespread inflammation in the lungs. ARDS is a rapidly progressive disease that occurs in critically ill patients most notably now in those diagnosed with COVID-19. ARDS affects approximately 200,000 patients per year in the U.S., exclusive of the current COVID-19 pandemic, and has a 30% to 50% mortality rate. ARDS is sometimes initially diagnosed as pneumonia or pulmonary edema (fluid in the lungs from heart disease). Symptoms of ARDS include shortness of breath, rapid breathing and heart rate, chest pain (particularly while inhaling), and bluish skin coloration. Among those who survive ARDS, a decreased quality of life is relatively common.

About Coronavirus Treatment Acceleration Program (CTAP)In response to the pandemic, the FDA has created an emergency program called the Coronavirus Treatment Acceleration Program (CTAP) to accelerate the development of treatments for COVID-19. By redeploying staff, the FDA is responding to COVID-19-related requests and reviewing protocols within 24 hours of receipt. The FDA said CTAP "uses every available method to move new treatments to patients as quickly as possible, while at the same time finding out whether they are helpful or harmful." In practice, that means developers of potential treatments for COVID-19 will benefit from an unusually faster track at the FDA to shorten wait times at multiple steps of the process.

About Citius Pharmaceuticals, Inc.Citius is a late-stage specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives and cancer care. For more information, please visit http://www.citiuspharma.com.

About Novellus, Inc.Novellus is a pre-clinical stage biotechnology company developing engineered cellular medicines using its non-immunogenic mRNA, nucleic-acid delivery, gene editing, and cell reprogramming technologies. Novellus is privately held and is headquartered in Cambridge, MA. For more information, please visit http://www.novellus-inc.com.

Safe HarborThis press release may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements are made based on our expectations and beliefs concerning future events impacting Citius. You can identify these statements by the fact that they use words such as "will," "anticipate," "estimate," "expect," "should," and "may" and other words and terms of similar meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition, and stock price. Factors that could cause actual results to differ materially from those currently anticipated are: the risk of successfully negotiating a license agreement with Novellus within the option period; our need for substantial additional funds; the ability to access the FDA's CTAP program for the MARCO trial; the estimated markets for our product candidates, including those for ARDS, and the acceptance thereof by any market; risks associated with conducting trials for our product candidates, including those expected to be required for any treatment for ARDS and our Phase III trial for Mino-Lok; risks relating to the results of research and development activities; risks associated with developing our product candidates, including any licensed from Novellus, including that preclinical results may not be predictive of clinical results and our ability to file an IND for such candidates; uncertainties relating to preclinical and clinical testing; the early stage of products under development; risks related to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; our ability to identify, acquire, close, and integrate product candidates and companies successfully and on a timely basis; our ability to attract, integrate, and retain key personnel; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions, or circumstances on which any such statement is based, except as required by law.

Contact:Andrew ScottVice President, Corporate Development(O) 908-967-6677 x105ascott@citiuspharma.com

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Citius Announces Pre-IND Submission to FDA Under the Coronavirus Treatment Acceleration Program for a Novel Stem Cell Therapy for Acute Respiratory...