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

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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SOURCE Citius Pharmaceuticals, Inc.

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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...

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

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

Orchard Therapeutics Announces First Patient Dosed with OTL-201 Gene Therapy in Proof-of-Concept Clinical Trial for Sanfilippo Syndrome (MPS-IIIA) -…

BOSTON and LONDON, April 27, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the first patient has been dosed in an open-label, proof-of-concept investigational study of OTL-201, an ex vivo autologous hematopoietic stem cell (HSC) gene therapy for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA). The study is designed to evaluate safety, tolerability and clinical efficacy and is intended to enroll up to five patients between three months and 24 months of age who will be followed for three years. The study also contains a number of key secondary outcome measures such as overall survival, cognition and behavior to help inform future clinical development of HSC gene therapy in this indication.

MPS-IIIA, also known as Sanfilippo syndrome type A, is a rare, inherited neurometabolic disorder caused by genetic mutations that leads to the buildup of sugar molecules called mucopolysaccharides in the body, resulting in progressive intellectual disability and loss of motor function. Children born with MPS-IIIA rarely live past adolescence or early adulthood, and no approved therapies currently exist to treat the disease.

I am very encouraged that we, together with our research and clinical collaborators in Manchester, could achieve this important milestone in our efforts to develop a gene therapy for MPS-IIIA despite the current, challenging global health circumstances, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. It is a testament to the dedication of our collective teams and underscores the truly dire, life-limiting nature of the disease for affected children and their families. This study adds to Orchards clinical pipeline of HSC gene therapies for the treatment of severe neurometabolic disorders and further demonstrates the potential of our platform approach.

About MPS-IIIAMucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A) is a rare and life-threatening metabolic disease. People with MPS-IIIA are born with a mutation in the N-sulphoglucosamine sulphohydrolase (SGSH) gene, which, when healthy, helps the body break down sugar molecules called mucopolysaccharides. The buildup of mucopolysaccharides in the brain and other tissues leads to intellectual disability and loss of motor function. MPS-IIIA occurs in approximately one in every 100,000 live births. Life expectancy of children born with MPS-IIIA is estimated to be between 10-25 years.

About OrchardOrchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically-modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. The company has one of the deepest gene therapy product candidate pipelines in the industry and is advancing seven clinical-stage programs across multiple therapeutic areas, including inherited neurometabolic disorders, primary immune deficiencies and blood disorders, where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About OrchardInvestors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (twitter.com/orchard_tx and http://www.linkedin.com/company/orchard-therapeutics), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals and the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development and commercial programs; the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards annual report on Form 10-K for the year ended December 31, 2019, as filed with the U.S. Securities and Exchange Commission (SEC) on February 27, 2020, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

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Orchard Therapeutics Announces First Patient Dosed with OTL-201 Gene Therapy in Proof-of-Concept Clinical Trial for Sanfilippo Syndrome (MPS-IIIA) -...

Impact of COVID-19 on Stem Cell Therapy Market Investment Opportunities And Forecast 2020-2027 | By Top Key Players Magellan, Medipost Co., Ltd,…

The Stem Cell Therapy Market Has Witnessed Continuous Growth In The Past Few Years And Is Projected To Grow Even Further During The Forecast Period (2020-2027). The Assessment Provides A 360 View And Insights, Outlining The Key Outcomes Of The Industry. These Insights Help The Business Decision-makers To Formulate Better Business Plans And Make Informed Decisions For Improved Profitability. In Addition, The Study Helps Venture Or Private Players In Understanding The Companies More Precisely To Make Better-informed Decisions. Some Of The Prominent Key Players Covered In The Stem Cell Therapy Market Are Magellan, Medipost Co., Ltd, Osiris Therapeutics, Inc., Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., Anterogen Co. Ltd., Pharmicell Co., Inc., and Stemedica Cell Technologies, Inc.

Whats Keeping Magellan, Medipost Co., Ltd, Osiris Therapeutics, Inc., Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., Anterogen Co. Ltd., Pharmicell Co., Inc., and Stemedica Cell Technologies, Inc. Ahead In The Market? Benchmark Yourself With Strategic Steps And Conclusions Recently Published By Coherent Market Insights

Type Segmentation:

By Cell Source:Adult Stem CellsInduced Pluripotent Stem CellsEmbryonic Stem CellsOthersGlobal Stem Cell Therapy Market, By Application:Musculoskeletal DisordersWounds and InjuriesCancerAutoimmune disordersOthers

Consumer Traits (If Applicable)

The Stem Cell Therapy Market Study Covers Current Status, % Share, Future Patterns, Development Rate, Swot Examination, Sales Channels, To Anticipate Growth Scenarios For Years 2020-2027. It Aims To Recommend Analysis Of The Market With Regards To Growth Trends, Prospects, And Players Contribution To Market Development. The Report Size Market By 5 Major Regions, Known As, North America, Europe, Asia Pacific (Includes Asia & Oceania Separately), Middle East And Africa (Mea), And Latin America.

The Stem Cell Therapy Market Factors Described In This Report Are:-key Strategic Developments In Stem Cell Therapy Market: The Research Includes The Key Strategic Activities Such As R&d Plans, M&a Completed, Agreements, New Launches, Collaborations, Partnerships & (Jv) Joint Ventures, And Regional Growth Of The Key Competitors Operating In The Market At A Global And Regional Scale.

Key Market Features In Stem Cell Therapy Market: The Report Highlights Stem Cell Therapy Market Features, Including Revenue, Weighted Average Regional Price, Capacity Utilization Rate, Production Rate, Gross Margins, Consumption, Import & Export, Supply & Demand, Cost Bench-marking, Market Share, Cagr, And Gross Margin.

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Stem Cell Therapy Market Study Coverage: It Includes Major Manufacturers, Emerging Players Growth Story, Major Business Segments Of Stem Cell Therapy Market, Years Considered, And Research Objectives. Additionally, Segmentation On The Basis Of The Type Of Product, Application, And Technology.

Stem Cell Therapy Market Executive Summary: It Gives A Summary Of Overall Studies, Growth Rate, Available Market, Competitive Landscape, Market Drivers, Trends, And Issues, And Macroscopic Indicators.Stem Cell Therapy Market Production By Region Stem Cell Therapy Market Profile Of Manufacturers-players Are Studied On The Basis Of Swot, Their Products, Production, Value, Financials, And Other Vital Factors.

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Stem Cell Therapy Capacity, Production, Revenue (Value) By Region (2019-2027)

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Stem Cell Therapy Production, Revenue (Value), Price Trend By Type {strip Sensors, Invasive Sensors, Ingestible Sensors, Implantable Sensors, Wearable Sensors}

Stem Cell Therapy Market Analysis By Application {hospitals, Ambulatory Surgical Centers, Diagnostic Centers}

Stem Cell Therapy Manufacturers Profiles/analysis Stem Cell Therapy Manufacturing Cost Analysis, Industrial/supply Chain Analysis, Sourcing Strategy And Downstream Buyers, Marketing Strategy By Key Manufacturers/players, Connected Distributors/traders Standardization, Regulatory And Collaborative Initiatives, Industry Road Map And Value Chain Market Effect Factors Analysis.

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Impact of COVID-19 on Stem Cell Therapy Market Investment Opportunities And Forecast 2020-2027 | By Top Key Players Magellan, Medipost Co., Ltd,...

Molecules identified that reverse cellular aging process – New Atlas

Central to a lot of scientific research into aging are tiny caps on the ends of our chromosomes called telomeres. These protective sequences of DNA grow a little shorter each time a cell divides, but by intervening in this process, researchers hope to one day regulate the process of aging and the ill health effects it can bring. A Harvard team is now offering an exciting pathway forward, discovering a set of small molecules capable of restoring telomere length in mice.

Telomeres can be thought of like the plastic tips on the end of our shoelaces, preventing the fraying of the DNA code of the genome and playing an important part in a healthy aging process. But each time a cell divides, they grow a little shorter. This sequence repeats over and over until the cell can no longer divide and dies.

This process is linked to aging and disease, including a rare genetic disease called dyskeratosis congenita (DC). This is caused by the premature aging of cells and is where the Harvard University team focused its attention, hoping to offer alternatives to the current treatment that involves high-risk bone marrow transplants and which offers limited benefits.

One of the ways dyskeratosis congenita comes about is through genetic mutations that disrupt an enzyme called telomerase, which is key to maintaining the structural integrity of the telomere caps. For this reason, researchers have been working to target telomerase for decades, in hopes of finding ways to slow or even reverse the effects of aging and diseases like dyskeratosis congenita.

Once human telomerase was identified, there were lots of biotech startups, lots of investment, says Boston Childrens Hospital's Suneet Agarwal, senior investigator on the new study. But it didnt pan out. There are no drugs on the market, and companies have come and gone.

Agarwal has been studying the biology of telomerase for the past decade, and back in 2015 he and his team discovered a gene called PARN that plays a role in the action of the telomerase enzyme. This gene normally processes and stabilizes an important component of telomerase called TERC, but when it mutates, it results in less of the enzyme being produced and, in turn, the telomeres becoming shortened prematurely.

For the new study, Harvard researchers screened more than 100,000 known chemicals in search of compounds that could preserve healthy function of PARN. This led them to small handful that seemed capable of doing so by inhibiting an enzyme called PAPD5, which serves to unravel PARN and destabilize TERC.

We thought if we targeted PAPD5, we could protect TERC and restore the proper balance of telomerase, says Harvard Medical Schools Neha Nagpal, first author on the new paper.

These chemicals were tested on stem cells in the lab, made from the cells of patients with dyskeratosis congenita. These compounds boosted TERC levels in those stem cells and restored telomeres to their normal length. However, rather than a scattergun approach, the team really wanted to test for safety and see if the treatment could precisely target stem cells carrying the right ingredients for telomerase formation.

More specifically, the team wanted to see if this could be achieved by having the PAPD5-inhibiting drugs recognize and respond to another important component of telomerase, a molecule called TERT. To do so, in the next round of experiments the team used human blood stem cells and triggered mutations in the PARN gene that give rise to dyskeratosis congenita. These were then implanted into mice that were treated with the compounds, with the team finding the treatment boosted TERC, restored telomere length in the stem cells and had no ill effects on the rodents.

This provided the hope that this could become a clinical treatment, says Nagpal.

The team will now continue its work in an effort to prove these small molecules are a safe and effective way to apply the brakes to dyskeratosis congenita, other diseases, and possibly aging more broadly.

We envision these to be a new class of oral medicines that target stem cells throughout the body, Agarwal says. We expect restoring telomeres in stem cells will increase tissue regenerative capacity in the blood, lungs, and other organs affected in DC and other diseases.

The research was published in the journal Cell Stem Cell.

Source: Boston Childrens Hospital via Harvard University

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Molecules identified that reverse cellular aging process - New Atlas

Breakthrough to halt premature aging of cells – ScienceBlog.com

Capping decades of research, a new study may offer a breakthrough in treatingdyskeratosis congenitaand other so-called telomere diseases, in which cells age prematurely.

Using cells donated by patients with the disease, researchers at theDana-Farber/Boston Childrens Cancer and Blood Disorders Centeridentified several small molecules that appear to reverse this cellular aging process.Suneet Agarwal, the studys senior investigator, hopes at least one of these compounds will advance toward clinical trials. Findings werepublished Tuesday in the journal Cell Stem Cell.

If so, it could be the first treatment for dyskeratosis congenita, or DC, that could reverse all of the diseases varying effects on the body. The current treatment, bone marrow transplant, is high-risk, and only helps restore the blood system, whereas DC affects multiple organs.

The compounds identified in the study restore telomeres, protective caps on the tips of our chromosomes that regulate how our cells age. Telomeres consist of repeating sequences of DNA that get shorter each time a cell divides.

The bodys stem cells, which retain their youthful qualities, normally make an enzyme called telomerase that builds telomeres back up again. But when telomeres cant be maintained, tissues age before their time. A spectrum of diseases can result not just DC, but also aplastic anemia, liver cirrhosis, and pulmonary fibrosis.

The discovery of telomerase 35 years ago, earninga Nobel Prize in 2009, galvanized the scientific world. Subsequent studies suggested the enzyme could be a key to reversing aging, as well as treating cancer, in which malignant cells become immortal and divide indefinitely.

For years, researchers have tried to find a simple and safe way to manipulate telomerase, preserve telomeres, and create cures for telomere diseases.

Once human telomerase was identified, there were lots of biotech startups, lots of investment, says Agarwal, who has researched the biology of telomerase for the past decade. But it didnt pan out. There are no drugs on the market, and companies have come and gone.

DC can be caused by mutations in any of multiple genes. Most of these mutations disrupt telomerase formation or function in particular, by disrupting two molecules called TERT and TERC that join together to form telomerase. TERT is an enzyme made in stem cells, and TERC is a so-called non-coding RNA that acts as a template to create telomeres repeating DNA sequences. Both TERT and TERC are affected by a web of other genes that tune telomerases action.

One of these genes is PARN. In 2015, Agarwal and colleagues showed inNatureGeneticsthat PARN is important for processing and stabilizing TERC. Mutations in PARN mean less TERC, less telomerase, and prematurely shortened telomeres.

Thenew study, led by Harvard Medical School postdoctoral fellow Neha Nagpal, delved further, focusing on an enzyme that opposes PARN and destabilizes TERC, called PAPD5.

We thought if we targeted PAPD5, we could protect TERC and restore the proper balance of telomerase, says Nagpal, first author on the paper.

Nagpal and her colleagues first conducted large-scale screening studies to identify PAPD5 inhibitors, testing more than 100,000 known chemicals. They got 480 initial hits, which they ultimately narrowed to a small handful.

They then tested the inhibitors in stem cells made from the Martins cells and those of other patients with DC. To the teams delight, the compounds boosted TERC levels in the cells and restored telomeres to their normal length.

But the real challenge was to see if the treatment would be safe and specific, affecting only the stem cells bearing TERT. To test this, the team introduced DC-causing PARN mutations into human blood stem cells, transplanted those cells into mice, then treated the mice with oral PAPD5 inhibitors. The compounds boosted TERC and restored telomere length in the transplanted stem cells, with no adverse effect on the mice or on the ability to form different kinds of blood cells.

This provided the hope that this could become a clinical treatment, says Nagpal.

In the future, Agarwal, Nagpal, and colleagues hope to validate PAPD5 inhibition for other diseases involving faulty maintenance of telomeres and perhaps even aging itself. They are most excited about two compounds, known as BCH001 and RG7834 that are under further development.

We envision these to be a new class of oral medicines that target stem cells throughout the body, Agarwal says. We expect restoring telomeres in stem cells will increase tissue regenerative capacity in the blood, lungs, and other organs affected in DC and other diseases.

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Breakthrough to halt premature aging of cells - ScienceBlog.com

World coronavirus Dispatch: Animal Stem Cell Therapy Market Forecasted To Surpass The Value Of US$ XX Mn/Bn By 2036 2017 2025 – Latest Herald

Given the debilitating impact of COVID-19 (Coronavirus) on the Animal Stem Cell Therapy market, companies are vying opportunities to stay afloat in the market landscape. Gain access to our latest research analysis on COVID-19 associated with the Animal Stem Cell Therapy market and understand how market players are adopting new strategies to mitigate the impact of the pandemic.

Analysis of the Global Animal Stem Cell Therapy Market

Persistence Market Research (PMR) recently published a market study which provides a detailed understanding of the various factors that are likely to influence the Animal Stem Cell Therapy market in the forecast period (20XX-20XX). The study demonstrates the historical and current market trends to predict the roadmap of the Animal Stem Cell Therapy market in the coming years. Further, the growth opportunities, capacity additions, and major limitations faced by market players in the Animal Stem Cell Therapy market are discussed.

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Regional Overview

Our team of analysts at PMR, trace the major developments within the Animal Stem Cell Therapy landscape in various geographies. The market share and value of each region are discussed in the report along with graphs, tables, and figures.

Competitive Outlook

This chapter of the report discusses the ongoing developments, mergers and acquisitions of leading companies operating in the Animal Stem Cell Therapy market. The product portfolio, pricing strategy, the regional and global presence of each company is thoroughly discussed in the report.

Product Adoption Analysis

The report offers crucial insights related to the adoption pattern, supply-demand ratio, and pricing structure of each product.

Key Participants

The key participants in the animal stem cell therapy market are Magellan Stem Cells, ANIMAL CELL THERAPIES, Abbott Animal Hospital, VETSTEM BIOPHARMA, Veterinary Hospital and Clinic Frisco, CO, etc. The companies are entering into the collaboration and partnership to keep up the pace of the innovations.

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Israeli scientists begin trials into the effectiveness of cannabis in treating Covid-19 – Euro Weekly News

RESEARCHERS want to determine if CBDs anti-inflammatory properties can slow or stop the virus.

Tests at Rabin Medical Centre in Petah Tikva will be led by partnerships with Stero Biotech, an Israeli CBD company, and Clalit, Israels largest HMO.

We estimate that our CBD-based treatment can enhance the current treatment of those patients who are in life-threatening conditions, said David Bass, Stero Biotechs founder and CEO, in a statement.

Hospitalised Covid-19 patients are mostly being treated with steroids, and our study is planned to demonstrate the benefit of a combined solution with steroid treatments.

Bass added that the company is hopeful that this study will lead to faster benefit for the growing number of Covid-19 patients in Israel and around the world.

Last week, InnoCan Pharma announced a collaboration with Tel Aviv University to instil CBD medicine through exosomes the small cell structures created when stem cells multiply.

The unconventional method will utilise the exosomes as homing missiles, as they can uniquely target cell organs damaged by Covid-19.

Researchers then believe CBDs anti-inflammatory properties will repair the damaged cells through a synergistic effect.

However, health experts have warned smoking marihuana could have the opposite effect, and make the condition worse.

All the same, scientists are carrying out studies, alongside existing treatments.

Steros 10-patient study, now underway, will seemingly be a small-scale clinical trial, which pending final approval by the final Helsinki Committee, will be a proof of concept study the first step before a clinical study.

It is likely to take a couple of months.

Read more from the original source:
Israeli scientists begin trials into the effectiveness of cannabis in treating Covid-19 - Euro Weekly News

Stem Cell Alopecia Treatment Market Segmentation, Application, Technology, Analysis Research Report and Forecast to 2026 – Cole of Duty

Sanford Burnham Prebys Medical Discovery Institute

Global Stem Cell Alopecia Treatment Market Segmentation

This market was divided into types, applications and regions. The growth of each segment provides an accurate calculation and forecast of sales by type and application in terms of volume and value for the period between 2020 and 2026. This analysis can help you develop your business by targeting niche markets. Market share data are available at global and regional levels. The regions covered by the report are North America, Europe, the Asia-Pacific region, the Middle East, and Africa and Latin America. Research analysts understand the competitive forces and provide competitive analysis for each competitor separately.

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Stem Cell Alopecia Treatment Market Region Coverage (Regional Production, Demand & Forecast by Countries etc.):

North America (U.S., Canada, Mexico)

Europe (Germany, U.K., France, Italy, Russia, Spain etc.)

Asia-Pacific (China, India, Japan, Southeast Asia etc.)

South America (Brazil, Argentina etc.)

Middle East & Africa (Saudi Arabia, South Africa etc.)

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Table of Contents:

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Stem Cell Alopecia Treatment Market Size by Manufacturer: Here, the report concentrates on revenue and production shares of manufacturers for all the years of the forecast period. It also focuses on price by manufacturer and expansion plans and mergers and acquisitions of companies.

Production by Region: It shows how the revenue and production in the global market are distributed among different regions. Each regional market is extensively studied here on the basis of import and export, key players, revenue, and production.

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Tags: Stem Cell Alopecia Treatment Market Size, Stem Cell Alopecia Treatment Market Trends, Stem Cell Alopecia Treatment Market Growth, Stem Cell Alopecia Treatment Market Forecast, Stem Cell Alopecia Treatment Market Analysis

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Stem Cell Alopecia Treatment Market Segmentation, Application, Technology, Analysis Research Report and Forecast to 2026 - Cole of Duty

Global trade impact of the Coronavirus Animal Stem Cell Therapy Market Size Overview, Top Companies, Inventive Trends and Forecast to 2032 – Germany…

Companies in the Animal Stem Cell Therapy market are vying suggestive steps to tackle the challenges resulting from the COVID-19 (Coronavirus) pandemic. Exhaustive research about COVID-19 is providing present-day techniques and alternative methods to mitigate the impact on Coronavirus on the revenue of the Animal Stem Cell Therapy market.

The report on the Animal Stem Cell Therapy market provides a birds eye view of the current proceedings and advancements within the Animal Stem Cell Therapy landscape. Further, the report ponders over the various factors that are likely to impact the overall dynamics of the Animal Stem Cell Therapy market over the forecast period (20XX-20XX) including the current trends, business expansion opportunities and restraining factors amongst others.

As per the market report suggested by ResearchMoz.us, the global Animal Stem Cell Therapy market is expected to register a CAGR growth of ~XX% during the forecast period and attain a value of ~US$XX by the end of 20XX. Further, the report suggests that the growth of the Animal Stem Cell Therapy market is largely influenced by a range of factors including, emphasis on R&D innovations by market players, surging investments to increase product portfolio, and favorable regulatory policies among others.

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Questions Related to the Animal Stem Cell Therapy Market Explained:

Competitive Landscape

The report provides critical insights related to the leading players operating in the Animal Stem Cell Therapy market. The revenue generated, market presence, product range, and financials of each company are enclosed in the report.

Segment by Type, the Animal Stem Cell Therapy market is segmented intoDogsHorsesOthers

Segment by Application, the Animal Stem Cell Therapy market is segmented intoVeterinary HospitalsResearch Organizations

Regional and Country-level AnalysisThe Animal Stem Cell Therapy market is analysed and market size information is provided by regions (countries).The key regions covered in the Animal Stem Cell Therapy market report are North America, Europe, Asia Pacific, Latin America, Middle East and Africa. It also covers key regions (countries), viz, U.S., Canada, Germany, France, U.K., Italy, Russia, China, Japan, South Korea, India, Australia, Taiwan, Indonesia, Thailand, Malaysia, Philippines, Vietnam, Mexico, Brazil, Turkey, Saudi Arabia, UAE, etc.The report includes country-wise and region-wise market size for the period 2015-2026. It also includes market size and forecast by Type, and by Application segment in terms of sales and revenue for the period 2015-2026.Competitive Landscape and Animal Stem Cell Therapy Market Share AnalysisAnimal Stem Cell Therapy market competitive landscape provides details and data information by players. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on revenue (global and regional level) by players for the period 2015-2020. Details included are company description, major business, company total revenue and the sales, revenue generated in Animal Stem Cell Therapy business, the date to enter into the Animal Stem Cell Therapy market, Animal Stem Cell Therapy product introduction, recent developments, etc.The major vendors covered:Medivet Biologics LLCVETSTEM BIOPHARMAJ-ARMU.S. Stem Cell, IncVetCell TherapeuticsCelavet Inc.Magellan Stem CellsKintaro Cells PowerAnimal Stem CareAnimal Cell TherapiesCell Therapy SciencesAnimacel

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Regional Landscape

The regional landscape of the report provides resourceful insights related to the revenue share analysis of the Animal Stem Cell Therapy market in different regions. Further, the market attractiveness of each region provides players a clear understanding of the overall growth potential in each regional market.

End-User Analysis

The report provides an in-depth understanding of the various end-users of the Animal Stem Cell Therapy along with the market share, size, and revenue generated by each end-user.

Important Information that can be extracted from the Report:

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Global trade impact of the Coronavirus Animal Stem Cell Therapy Market Size Overview, Top Companies, Inventive Trends and Forecast to 2032 - Germany...