Bit Bio Secures Distribution Agreement with Abcam to Democratize Access to Human Cells for Global Life Science Research – Gooruf

Article summary

Bit Bio announces agreement with Abcam, a global innovator in life science reagents and tools, to make Bit Bios iPSC derived functional human cells widely available to the global life science community. The first product available are brain cells (ioNEURONS/glutTM, glutamatergic neurons) serving the neuroscience community. [] Access to human cells is a significant bottleneck in the field of medical research and drug development. [] Bit Bio is commercializing opti-ox TM , a precise reprogramming proprietary technology platform that enables uniquely efficient and consistent production of human cells for use in research, drug discovery, and cell therapy. [] Abcams reputation as a disruptive innovator in the field of biological reagents and dedicated global commercialization infrastructure make them the ideal partner. In line with our core value of democratizing access to human cells for research and drug development, our ioNEURONS/glut are offered at a highly competitive price point.

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Bit Bio Secures Distribution Agreement with Abcam to Democratize Access to Human Cells for Global Life Science Research - Gooruf

uniQure Presents Multiple New Preclinical Data on AMT-130 at the CHDI’s 15th Annual Huntington’s Disease Therapeutics Conference – GlobeNewswire

~ Up to Two Years of Follow-up in Large Transgenic Huntingtons Disease Model Demonstrates Stable mHTT Protein Lowering ~

~ Novel Preclinical Data Demonstrates Successful Lowering of Pathogenic Exon 1 Fragment ~

~ Additional Data Demonstrates the Potential of MRS as Imaging Biomarker for Huntingtons Disease Gene-Therapy Studies ~

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Feb. 27, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the presentation of multiple new preclinical data on AMT-130, its investigational AAV gene therapy for the treatment of Huntingtons disease (HD), at the 15th Annual CHDI Huntingtons disease Therapeutics Conference in Palm Springs, California.

Our data presentations at CHDI illustrate the increasing potential of AMT-130 to target the highly toxic exon 1 protein fragment, achieve broad vector biodistribution across several animal species and show meaningful activity using the presence of extracellular vesicles as a potential biomarker, stated Sander van Deventer, M.D., Ph.D., executive vice president, research & product development of uniQure. In addition, we highlight the use of magnetic resonance spectroscopy as a potentially important imaging biomarker to measure the restoration of target tissue. Collectively, these findings represent a robust package of new preclinical data to better inform how researchers and clinicians pursue a much-needed treatment for this devastating disease.

Four scientific abstracts submitted by uniQure researchers were accepted for presentation at the conference, of which one is an oral presentation to be featured today. Important findings across several preclinical studies presented at the conference include the following:

Translatable Biomarkers in Gene Therapy for Huntington Disease: Learnings from Pre-clinical Studies

Secreted Therapeutics: Monitoring Durability of microRNA-based Gene Therapies in Huntingtons disease

Lowering the Pathogenic Exon 1 HTT Fragment by AAV5-miHTT Gene Therapy

Exploring the Effects of Intrastriatal AAV5-miHTT Lowering Therapy on Neuronal Function, MRS Signal and Mutant Huntingtin Levels in the Q175FDN Mouse Model of Huntingtons disease

The uniQure data presentations featured at CHDI are available on the investor page of the Companys website, http://www.uniQure.com

About Huntingtons DiseaseHuntingtons disease is a rare, inherited neurodegenerative disorder that leads to loss of muscle coordination, behavioral abnormalities and cognitive decline, resulting in complete physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene, that leads to the production and aggregation of abnormal protein in the brain. Despite the clear etiology of Huntingtons disease, there are no therapies to delay the onset or to slow the diseases progression.

About uniQure uniQure is delivering on the promise of gene therapy single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. http://www.uniQure.com

uniQure Forward-Looking StatementsThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, whether AMT-130 will target the highly toxic exon 1 protein fragment, achieve broad vector biodistribution across several animal species or show meaningful activity using the presence of extracellular vesicles as a potential biomarker, and whether magnetic resonance spectroscopy will be an important imaging biomarker to measure the restoration of target tissue. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our and our collaborators clinical development activities, clinical results, collaboration arrangements, corporate reorganizations and strategic shifts, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQures Quarterly Report on Form 10-Q filed on October 28, 2019. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

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uniQure Presents Multiple New Preclinical Data on AMT-130 at the CHDI's 15th Annual Huntington's Disease Therapeutics Conference - GlobeNewswire

CAR T-Cell Therapy Improves Health-Related QOL in Adult Patients With R/R DLBCL – AJMC.com Managed Markets Network

Maggie L. Shaw

Health-related quality of life (QOL) improved in 54% of patients 18 years or older with relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL) after therapy with tisagenlecleucel (Kymriah; Novartis), one of 2 FDA-approved chimeric antigen receptor (CAR) T-cell therapies. The patients general health, vitality, physical function, and social function improved the most.

This population consisted of patients 18 years or older with R/R DLBCL following 2 or more lines of therapy who also had a failed autologous stem cell transplant (ASCT) or were deemed ineligible for ASCT. Their HRQOL was measured through the Functional Assessment of Cancer Therapy-Lymphoma (FACT-Lym) and Short Form-36 (SF-36) Health Survey, at baseline screening and at months 3, 6, 12, and 18. The lower the score, the worse the HRQOL. The data cutoff was May 21, 2018, by which time 115 patients had been infused with tisagenlecleucel, and the median follow-up was 19 months.

Overall, 40% of these patients also achieved a complete response (CR), and the 54% (15/28) who had an initial partial response (PR) eventually had a CR. The median duration of response was not reached (95% CI, 10.0 monthsnot evaluable [NE]); however, patients 65 years or older and those younger than 65 years had similar durations of response. In addition, median overall survival was 11.1 months (95% CI, 6.6 months-NE), but was not reached in patients with a CR (95% CI, 21.1 months-NE). Cytopenias longer than 28 days (34%), cytokine release syndrome (23%), infections (19%), febrile neutropenia (15%), and neurologic events (11%) were the most common adverse effects, and all were deemed manageable.

The HRQOL responses to the questionnaires were as positive. Per FACT-Lym, there was an overall improvement in HRQOL from the baseline screening through month 18, as well as in the patients with a CR or PR (n = 57), with the greatest improvement seen at month 18 for functional, physical, and social/family domains. And after the SF-36 was administered, a positive mean change was seen overall, signifying meaningful improvements in HRQOL. Measurements at months 3, 6, 12, and 18, in particular, showed increases above the minimally clinically important differences for general health, vitality, physical functioning, role-physical, and social functioning.

Broken down at each HRQOL assessment timepoint, the questionnaire completion rates were as follows:

Going forward, the study authors suggest adding the Patient-Reported Outcomes Measurement Information System to future CAR T-cell trials, as well as establishing optimal times at which to measure HRQOL following this gene therapy, especially because of the dearth of HRQOL data on patients with R/R DLBCL.

Reference

Maziarz RT, Waller EK, Jaeger U, et al. Patient-reported long-term quality of life after tisangenlecleucel in relapsed/refractory diffuse large B-cell lymphoma. Blood Adv. 2020;4(4):629-637. doi: 10.1182/bloodadvances.2019001026.

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CAR T-Cell Therapy Improves Health-Related QOL in Adult Patients With R/R DLBCL - AJMC.com Managed Markets Network

Bucks County Surgeon Battling ALS To Receive Special Honor At 4th Annual Gala Of Hope – CBS Philly

BUCKS COUNTY, Pa. (CBS) A story of hope and courage will be recognized this weekend with a special honor for a Bucks County surgeon who is sidelined with a devastating diagnosis. But he and his family have found a way to feel grateful.

ALS, or Lou Gehrigs Disease, is one of the most feared as its a slow, difficult death.

For patients and their families, its a race against time with American research being so slow, but the theme for this family is broken-hearted but not broken.

Bob Sinnott checks email with a voice-activated phone because the hands of this one-time surgeon dont work anymore.

This is one of many crushing realities that come with ALS.

The hardest part for me when I got diagnosed was telling my kids, Sinnott said. Ive given people bad news a thousand times in my life about cancer diagnoses and different things but telling them was really hard.

(credit: CBS3)

Since he was diagnosed two years ago, his family in Bucks County has watched the progressive neurodegenerative disease slowly rob the doctors ability to move.

Sinnott is facing a slow, crippling death. Theres sadness and fear but thats not all.

Im grateful for what I have and I think gratitude is a fuel, his wife, Trish Sinnott, said.

If you look at the blessing in your life every day, thats what keeps me going, Bob Sinnott said.

Family and friends have had a variety of events to fight ALS and support the Hope Foundation at Temple University.

(credit: CBS3)

Because the currently approved FDA medications arent very effective, Sinnott is taking dozens of supplements and trying different therapies.

I want to do everything I can to slow progression as long as I canto be as functional as I can, he said.

That includes going to South Korea for stem cell treatments which they think have helped.

While theres exciting U.S. research underway, its notoriously slow and time isnt on their side.

If Im ever having a bad day, I just have to look at all the cards Ive received from friends, family and patients. It just helps me get up and try to work as hard as I can, Sinnott said.

(credit: CBS3)

He will be honored at the upcoming Gala of Hope for the ALS Hope Foundation at Temple University.

The foundation is doing ALS research and provides patient support.

For more information on the fourth annual Gala of Hope, click here.

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Bucks County Surgeon Battling ALS To Receive Special Honor At 4th Annual Gala Of Hope - CBS Philly

2020 Cell Therapy Bioprocessing Conference (Boston, MA, United States – June 25-26, 2020) – ResearchAndMarkets.com – Business Wire

DUBLIN--(BUSINESS WIRE)--The "2nd Annual Cell Therapy Bioprocessing Conference" conference has been added to ResearchAndMarkets.com's offering.

Over the last decade, the field of cell therapy has rapidly grown, and it holds enormous promise for treating many diseases. In the 2017 conference, the specific risks and benefits of the cell culture and cell therapy bio-manufacturing for the cure of these diseases was assessed.

There are still factors like manufacturing maze, investment, logistics and regulatory challenges which prevents the cell and gene therapies to be widely used. An unique platform to provide the exact solutions to these robust manufacturing and bioprocessing challenges is presented at the 2nd Annual Cell Therapy Bioprocessing Conference, taking place at Boston-USA on 25th & 26th June 2020.

Key Highlights

Why Attend

Who Should Attend:

From Therapeutics and Pharmaceuticals, Cell & Gene therapy-based companies:

Engineers/ Scientists/ Researchers/ Project leaders in:

Agenda:

Day 1: Thursday June 25th

CELL CULTURE TO CELL THERAPY

CELL THERAPY BIOPROCESSING AND DEVELOPMENT

PROCESS MONITORING & QUALITY CONTROL

Day 2: Friday June 26th

LOGISTICS, REGULATORY & INVESTMENT

Speakers:

For more information about this conference visit https://www.researchandmarkets.com/r/xj2lwk

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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2020 Cell Therapy Bioprocessing Conference (Boston, MA, United States - June 25-26, 2020) - ResearchAndMarkets.com - Business Wire

Humans soon able to regrow spines as body given ‘new power to heal itself’ – Daily Star

Scientists believe that humans will soon be able to recover from injuries such as broken spines, as treatment looks to boost the body's ability to heal itself.

A new study in the journal Regenerative Medicine describes how scientists were able to stimulate the self-repair response in rats.

Rats in the study were given two drugs, one of which is usually given to bone marrow transplant patients, and another which is used for bladder control.

This cocktail caused the rats' bone marrow to produce a greater number of mesenchymal stem cells, the cells which can develop into bone tissue.

As a result, enhanced calcium binding was seen at the site of the rats' spinal injuries, speeding up the production of new bone as well as healing wounds.

The study's authors hope that one day, such treatments will work on humans.

"We know that when bones break they will heal, and this requires the activation of stem cells in the bone," study co-author Sara Rankin from the National Heart and Lung Institute at Imperial College London, said in a statement.

"However, when the damage is severe, there are limits to what the body can do of its own accord.

"We hope that by using these existing medications to mobilise stem cells, as we were able to do in rats in our new study, we could potentially call up extra numbers of these stem cells, in order to boost our bodies' own ability to mend itself and accelerate the repair process."

Both drugs tested on rats are already widely used, so researchers are hopeful human trails can begin soon.

If these trials produce the same results as those seen in rats, then it's hoped the treatment could help to not only repair spinal injuries but also speed up the rate at which broken bones heal and mend damaged tissues in other organs.

Dr Tariq Fellous, first author of the research, said: "We first need to see if these medications release the stem cells in healthy volunteers before we can test them in patients with fractures.

"We have the drugs and know they are safe to use in humans we just need the funding for the human trials."

Dr Andia Redpath, who also co-authored the paper, added that repurposing existing medicines - so-called Regenerative Pharmacology - could have major potential as an efficient and cheaper way of treating diseases.

"Rather than devising new stem cell treatments from scratch that involve lengthy and expensive trials, our approach harnesses the power of the body's own stem cells, using existing drugs.

"We already know the treatments in our study are safe, it's now just a matter of exploring further if they help our bodies heal."

Stem cells are providing incredible new medical breakthroughs all the time.

Earlier this month, scientists trialled 3D-printed skin containing stem cells to treat burns victims .

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Humans soon able to regrow spines as body given 'new power to heal itself' - Daily Star

Radiation Kills Cancer Cells, But May Also Protect Some Tumor Stem Cells That Can Spread – MedicalResearch.com

MedicalResearch.com Interview with:

Jennifer Sims-Mourtada, Ph.D.Senior Rsearch ScientistDirector of Translational Breast Cancer ResearchCenter for Translational Cancer ResearchChristianaCare

MedicalResearch.com: What is the background for this study?

Response: Cancer stem cells are resistant cancer cells that are able to continuously grow and are very resistant to radiation and chemotherapy. Cancer stem cells can also escape to the blood stream and travel to another site causing metastasis.

MedicalResearch.com: What are the main findings?

Response: In this study we show that cancer stem cells depend on radiation induced inflammatory responses to survive radiation. Additionally we show that activation of IL-6-STAT3 pathway after radiation can make even non-cancer stem cells look like and behave as cancer stem cells. Thus any cell that survives radiation in the presence of certain inflammatory signals may be converted to a cancer stem cell.

MedicalResearch.com: What should readers take away from your report?

Response: Although radiation kills tumor cells, it can also activate inflammatory responses that may protect some tumor cells. Co-treatment with anti-inflammatory agents such as inhibitors to the IL-6 STAT 3 pathway may sensitize cancer stem cells to radiation induced death, and may prevent generation of new cancer stem cancer stem cells and improve outcomes in triple negative breast cancer.

MedicalResearch.com: What recommendations do you have for future research as a result of this work?

Response: Agents inhibiting the IL-6-STAT3 pathway are currently in clinical trials for breast cancer. Future work should focus on understanding radiation-induced inflammation in the context of both the immune response and tumor cells, as radiation induced inflammation can activate anti-tumor immunity as well as killing tumor cells. Some studies suggest that inhibition of IL-6 STAT3 signaling may also improve anti-tumor immunity, and thus these agents may target cancer cells by multiple mechanisms. It will be important to understand how these agents work to select which patients will respond to inhibition of this pathway and which ones will not.

MedicalResearch.com: Is there anything else you would like to add?

Response: This is the good and the bad of radiation. We know radiation induced inflammation can help the immune system to kill tumor cells thats good but also it can protect cancer stem cells in some cases, and thats bad. Whats exciting about these findings is were learning more and more that the environment the tumor is in its microenvironment is very important. Historically, research has focused on the genetic defects in the tumor cells. Were now also looking at the larger microenvironment and its contribution to cancer.

There are no disclosures to report

Citation:

Kimberly M. Arnold et al, Radiation induces an inflammatory response that results in STAT3-dependent changes in cellular plasticity and radioresistance of breast cancer stem-like cells,International Journal of Radiation Biology(2019).DOI: 10.1080/09553002.2020.1705423

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The information on MedicalResearch.com is provided for educational purposes only, and is in no way intended to diagnose, cure, or treat any medical or other condition. Always seek the advice of your physician or other qualified health and ask your doctor any questions you may have regarding a medical condition. In addition to all other limitations and disclaimers in this agreement, service provider and its third party providers disclaim any liability or loss in connection with the content provided on this website.

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Radiation Kills Cancer Cells, But May Also Protect Some Tumor Stem Cells That Can Spread - MedicalResearch.com

Musculoskeletal Disorder Stem Cell Therapy Market Report 2019: With Essential Analysis of Market, Industry News and Policies by Regions, Top…

Musculoskeletal Disorder Stem Cell Therapy Market Scope of the Report:

The worldwide market for Musculoskeletal Disorder Stem Cell Therapy is expected to grow at a CAGR of roughly xx% over the next five years, will reach xx million US$ in 2025, from xx million US$ in 2018, according to a new study.

This report focuses on the Musculoskeletal Disorder Stem Cell Therapy in global market, especially in North America, Europe and Asia-Pacific, South America, Middle East and Africa. This report categorizes the market based on manufacturers, regions, type and application.

Request Sample Report @ https://www.researchmoz.com/enquiry.php?type=S&repid=2040936&source=atm

Musculoskeletal Disorder Stem Cell Therapy Market Segment by Manufacturers, this report covers

The key players covered in this studyOsiris TherapeuticsNuVasiveTakeda (TiGenix)Medi-post

Market segment by Type, the product can be split intoAllogeneicAutologous

Market segment by Application, split intoMuscle diseaseSkeletal disease

Market segment by Regions/Countries, this report coversUnited StatesEuropeChinaJapanSoutheast AsiaIndiaCentral & South America

Make An EnquiryAbout This Report @ https://www.researchmoz.com/enquiry.php?type=E&repid=2040936&source=atm

Reasons to Purchase this Musculoskeletal Disorder Stem Cell Therapy Market Report:

* Analyzing the outlook of the market with the recent trends and SWOT analysis

* Market dynamics scenario, along with growth opportunities of the market in the years to come

* Market segmentation analysis including qualitative and quantitative research incorporating the impact of economic and non-economic aspects

* Regional and country level analysis integrating the demand and supply forces that are influencing the growth of the market.

* Market value (USD Million) and volume (Units Million) data for each segment and sub-segment

* Competitive landscape involving the market share of major players, along with the new projects and strategies adopted by players in the past five years

* Comprehensive company profiles covering the product offerings, key financial information, recent developments, SWOT analysis, and strategies employed by the major market players

* 1-year analyst support, along with the data support in excel format.

You can Buy This Report from Here @ https://www.researchmoz.com/checkout?rep_id=2040936&licType=S&source=atm

The Musculoskeletal Disorder Stem Cell Therapy Market report has 150 tables and figures browse the report description and TOC:

Table of Contents

1 Study Coverage

1.1 Musculoskeletal Disorder Stem Cell Therapy Product

1.2 Key Market Segments in This Study

1.3 Key Manufacturers Covered

1.4 Market by Type

1.4.1 Global Musculoskeletal Disorder Stem Cell Therapy Market Size Growth Rate by Type

1.5 Market by Application

1.5.1 Global Musculoskeletal Disorder Stem Cell Therapy Market Size Growth Rate by Application

2 Executive Summary

2.1 Global Musculoskeletal Disorder Stem Cell Therapy Market Size

2.1.1 Global Musculoskeletal Disorder Stem Cell Therapy Revenue 2014-2025

2.1.2 Global Musculoskeletal Disorder Stem Cell Therapy Production 2014-2025

2.2 Musculoskeletal Disorder Stem Cell Therapy Growth Rate (CAGR) 2019-2025

2.3 Analysis of Competitive Landscape

2.3.1 Manufacturers Market Concentration Ratio (CR5 and HHI)

2.3.2 Key Musculoskeletal Disorder Stem Cell Therapy Manufacturers

2.3.2.1 Musculoskeletal Disorder Stem Cell Therapy Manufacturing Base Distribution, Headquarters

2.3.2.2 Manufacturers Musculoskeletal Disorder Stem Cell Therapy Product Offered

2.3.2.3 Date of Manufacturers Enter into Musculoskeletal Disorder Stem Cell Therapy Market

2.4 Key Trends for Musculoskeletal Disorder Stem Cell Therapy Markets & Products

3 Market Size by Manufacturers

3.1 Musculoskeletal Disorder Stem Cell Therapy Production by Manufacturers

3.1.1 Musculoskeletal Disorder Stem Cell Therapy Production by Manufacturers

3.1.2 Musculoskeletal Disorder Stem Cell Therapy Production Market Share by Manufacturers

3.2 Musculoskeletal Disorder Stem Cell Therapy Revenue by Manufacturers

3.2.1 Musculoskeletal Disorder Stem Cell Therapy Revenue by Manufacturers (2019-2025)

3.2.2 Musculoskeletal Disorder Stem Cell Therapy Revenue Share by Manufacturers (2019-2025)

3.3 Musculoskeletal Disorder Stem Cell Therapy Price by Manufacturers

3.4 Mergers & Acquisitions, Expansion Plans

More Information.

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Musculoskeletal Disorder Stem Cell Therapy Market Report 2019: With Essential Analysis of Market, Industry News and Policies by Regions, Top...

Consistent Outcomes Using Ryoncil as First-Line Treatment or Salvage Therapy in 309 Children With Steroid-Refractory Acute GVHD – BioSpace

NEW YORK, Feb. 24, 2020 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB) today announced that aggregated results from 309 children treated with Ryoncil (remestemcel-L) were presented atthe American Society for Transplantation Cellular Therapy and the Center for International Blood & Bone Marrow Transplant Research (TCT) meeting in Orlando, Florida on February 22. The data showed that treatment with RYONCIL across three separate trials resulted inconsistent treatment responses and survival outcomesinchildren with steroid-refractory acute graft versus host disease (SR-aGVHD).

Key findings and conclusions were:

Mesoblast Chief Medical Officer Dr Fred Grossman said: These aggregated data from three studies demonstrate consistent efficacy and safety of RYONCIL in children suffering from steroid refractory acute graft versus host disease. If approved, RYONCIL has the potential to be an effective and safe therapy to improve survival outcomes in the most vulnerable population of children with severe forms of this disease who can have mortality rates as high as 90 percent.

In January, Mesoblast filed a Biologics License Application (BLA) to the United States Food and Drug Administration (FDA) for RYONCIL for the treatment of children with steroid-refractory aGVHD. The Company has requested Priority Review of the BLA by the FDA under the product candidates existing Fast Track designation. If approved, RYONCIL is expected to be launched in the US in 2020.

About Acute GVHDAcute GVHD occurs in approximately 50% of patients who receive an allogeneic bone marrow transplant (BMT). Over 30,000 patients worldwide undergo an allogeneic BMT annually, primarily during treatment for blood cancers, and these numbers are increasing.1 In patients with the most severe form of acute GVHD (Grade C/D or III/IV) mortality is as high as 90% despite optimal institutional standard of care.2,3. There are currently no FDA-approved treatments in the US for children under 12 with SR-aGVHD.

About Ryoncil Mesoblasts lead product candidate, RYONCIL, is an investigational therapy comprising culture- expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. It is administered to patients in a series of intravenous infusions. RYONCIL is believed to have immunomodulatory properties to counteract the inflammatory processes that are implicated in SR- aGVHD by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.

References1. Niederwieser D, Baldomero H, Szer J. (2016) Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and Marrow Transplantation Group including the global survey.2. Westin, J., Saliba, RM., Lima, M. (2011) Steroid-refractory acute GVHD: predictors and outcomes. Advances in Hematology.3. Axt L, Naumann A, Toennies J (2019) Retrospective single center analysis of outcome, risk factors and therapy in steroid refractory graft-versus-host disease after allogeneic hematopoietic cell transplantation. Bone Marrow Transplantation.

About MesoblastMesoblast Limited (Nasdaq: MESO; ASX: MSB) is a world leader in developing allogeneic (off-the-shelf) cellular medicines. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platforms to establish a broad portfolio of commercial products and late-stage product candidates. Mesoblasts proprietary manufacturing process yields industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblast has filed a Biologics License Application to the United States Food and Drug Administration (FDA) to seek approval of its product candidate Ryoncil (remestemcel-L) for steroid-refractory acute graft versus host disease (acute GvHD). Remestemcel-L is also being developed for other rare diseases. Mesoblast is completing Phase 3 trials for its rexlemestrocel product candidates for advanced heart failure and chronic low back pain. If approved, RYONCIL is expected to be launched in the United States in 2020 for pediatric steroid-refractory acute GVHD. Two products have been commercialized in Japan and Europe by Mesoblasts licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see http://www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast

Mesoblasts Forward-Looking StatementsThis announcement includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about the timing, progress and results of Mesoblasts preclinical and clinical studies; Mesoblasts ability to advance product candidates into, enroll and successfully complete, clinical studies; the timing or likelihood of regulatory filings and approvals; and the pricing and reimbursement of Mesoblasts product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblasts actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

Release authorized by the Chief Executive.

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Consistent Outcomes Using Ryoncil as First-Line Treatment or Salvage Therapy in 309 Children With Steroid-Refractory Acute GVHD - BioSpace

Mice with diabetes "functionally cured" using new stem cell therapy – New Atlas

Diabetes is characterized by trouble producing or managing insulin, and one emerging treatment involves converting stem cells into beta cells that secrete the hormone. Now, scientists have developed a more efficient method of doing just that, and found that implanting these cells in diabetic mice functionally cured them of the disease.

The study builds on past research by the same team, led by Jeffrey Millman at Washington University. The researchers have previously shown that infusing mice with these cells works to treat diabetes, but the new work has had even more impressive results.

These mice had very severe diabetes with blood sugar readings of more than 500 milligrams per deciliter of blood levels that could be fatal for a person and when we gave the mice the insulin-secreting cells, within two weeks their blood glucose levels had returned to normal and stayed that way for many months, says Millman.

Insulin is normally produced by beta cells in the pancreas, but in people with diabetes these cells dont produce enough of the hormone. The condition is usually managed by directly injecting insulin into the bloodstream when its needed. But in recent years, researchers have found ways to convert human stem cells into beta cells, which can pick up the slack and produce more insulin.

In the new study, the team improved that technique. Usually when converting stem cells into a specific type of cell, a few random mistakes are made, so some other types of cells end up in the mix. These are harmless, but arent exactly pulling their weight for the job at hand.

The more off-target cells you get, the less therapeutically relevant cells you have, says Millman. You need about a billion beta cells to cure a person of diabetes. But if a quarter of the cells you make are actually liver cells or other pancreas cells, instead of needing a billion cells, youll need 1.25 billion cells. It makes curing the disease 25 percent more difficult.

So, the new method was focused on reducing those unwanted extras. By targeting the cytoskeleton, the underlying structure that gives cells their shape, the team was able to not only produce a higher percentage of beta cells, but they also functioned better.

Its a completely different approach, fundamentally different in the way we go about it, said Millman. Previously, we would identify various proteins and factors and sprinkle them on the cells to see what would happen. As we have better understood the signals, weve been able to make that process less random.

When these new-and-improved beta cells were infused into diabetic mice, their blood sugar levels were stabilized, rendering the diabetes functionally cured for up to nine months.

Of course, at this stage its just an animal trial, so the results may not translate to humans any time soon, if ever. But the researchers plan to continue the work by testing the cells in larger animals over longer periods, with hopes of one day getting the treatment ready for human clinical trials.

The research was published in the journal Nature Biotechnology.

Source: Washington University

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Mice with diabetes "functionally cured" using new stem cell therapy - New Atlas