Stem Cell Clinic

Researching the Safety and Effectiveness of Stem Cells to Treat ‘Long Covid’ – Entrepreneur

Opinions expressed by Entrepreneur contributors are their own.

A representative from CNN reached out to us recently with some questions about stem cells as a potential treatment for so-called long Covid. As they were working on their story, they learned about an interventional clinical trial BioXcellerator has designed to study stem-cell therapy as a possible treatment for Covid-19.

Yes, we are conducting a studyon the use of stem cells to treat cases of acute respiratory distress syndrome (ARDS) that can develop in many Covid-19 patients and cause even higher mortality rates.

But this was a study we designed back in January of 2020. Thats when the global pandemic was in its infancy. The medical community was squarely focused on coping with acute disease, not chronic symptoms that might linger long after a patient recovers. The word Covid was still unfamiliar to many people, and the term long Covid wasn't used until months later.

As we explained to CNN, theres a big difference between acute cases of a disease and chronic conditions that may require different treatment approaches. So such a study would need to be a different one with its own set of research criteria. We pointed out that few, if any, such studies have begun by any research organization, but that didnt reflect a lack of interest.

In fact, quite the contrary. Our medical team is hard at work evaluating just such a study because we need more research to determine whether stem-cell therapy may indeed be a safe and effective treatment for the long-term lingering impact of this disease.

Related:High-Potency 'GoldenCells' Offer Hope to Those With Severe Chronic Back and Neck Pain

In the scientific community, clinical trials are designed to test the safety and efficacy of various treatments using strict controls to measure results. Im proud of our companys participation with leading scientists in a wide variety of studies.

To design these studies, scientists will often publish reviews of prior research to help guide the development of future research. I also take great pride in our participation in these types of reviews. Back in February, BioXcellerator Chief Medical Officer Dr. Karolynn Halpert and our epidemiologist Dr. Santiago Saldarriaga were coauthors of a review on this topic published in the Journal of Stem Cells Research Development & Therapy.

That review, Regenerative Rehabilitation for COVID 19 Sequelae, discusses thevarious disease processes that can impair lung function, prior research on regenerative rehabilitative approaches to treating patients and the potential for stem cells to improve treatment outcomes.

One major theme of this review is how controlling inflammation that results from an overactive immune response might help more patients recover lung function and promote healing of damaged tissue caused by the Covid virus.

Related:This Is HowStem-CellTherapy Treats Serious Brain Injuries

The study we are currently working on is based on treating acute Covid-19 using a specific type of stem cell:mesenchymal stem cells (MSCs) from the Whartons Jelly in donated umbilical cords. Many years of research haveshown the potential of this type of stem cell for reducing harmful inflammation. Earlier research demonstrates how stem cells can modulate the bodys immune response, which may help prevent the production of excess cytokines that can trigger serious inflammation and ARDS. As that journal review explains, MSCs release anti-inflammatory signals and growth factors that may help prevent cell death by reducing that serious inflammation.

And other studies not of stem cells show that reducing systemic inflammation through other treatments may reduce plasma levels of these harmful cytokines and, in turn, may prevent the onset of ARDS or help more patients recover from it.

Whats more, weve developed proprietary protocols for enhancing the potency of the stem cells we use for treatment through a process that include screening stem cells from donated umbilical cords for specific biomarkers that indicate the highest possible potency, selecting only those cells that meet strict criteria for potency and quality, and refining and purifying these cells before reproducing them into infusions of millions of high-potency stem cells for treatment.

This approach has led to treatments for a wide range of diseases and disorders where reducing inflammation and modulating the bodys immune response can be effective at promoting healing.

So back to the question CNN asked. Can stem cells be effective at treating long Covid? Obviously, it will take far more research by many organizations to reach any definitive conclusion, but the question itself demonstrates the importance of understanding that while there are differences between acute disease and chronic conditions, in some cases, results from one study can influence the direction of later studies.

Its also a reflection of how the value of all of our research may be uncertain now, but often becomes clear in the future. Indeed, as Soren Kierkegaard once pointed out, life must be lived looking forward, but it can only be understood by looking back.

Im not sure exactly what well understand when we look back at this unprecedented global pandemic, but we know far more about the role of regenerative medicine and stem-cell therapy in treating a wide range of diseases and how all of usentrepreneurs, scientists and physicians can work together to make even more discoveries to improve the quality of our lives.

Related:The Future of Health: Why Age 100 Will Soon Become 'the New 60'

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Researching the Safety and Effectiveness of Stem Cells to Treat 'Long Covid' - Entrepreneur

StemExpress Partners with the Alliance for Regenerative Medicine to Provide COVID-19 Testing for the Cell and Gene Meeting on the Mesa – WSAW

StemExpress to use utilize the Thermo Fisher Accula rapid PCR testing system to provide event attendees with accurate results in 30 minutes.

Published: Oct. 5, 2021 at 2:33 PM CDT|Updated: 3 hours ago

SACRAMENTO, Calif., Oct. 5, 2021 /PRNewswire/ --StemExpress is proud to announce that they will be the official COVID-19 testing provider for 2021's Meeting on the Mesa, a hybrid event bringing together great minds in the cell and gene biotech sphere. It has partnered with Alliance for Regenerative Medicine to comply with the newly implemented California state COVID-19 vaccination and testing policy regarding gatherings with 1,000 or more attendees. This partnership will allow the vital in-person networking aspect of the event to commence while protecting the health and safety of participants and attendees.

In-person networking commences at the 2021 Cell and Gene Meeting on the Mesa with COVID-19 testing options provided by StemExpress.

As a leading global provider of human biospecimen products, StemExpress understands the incredible impact that Meeting on the Mesa has on the industry and has been a proud participant for many years. For over a decade, StemExpress has provided the cell and gene industry with vital research products and holds valued partnerships with many of this year's participants. As such, it understands the immense value that in-person networking provides and is excited to help bring this element back to the meeting safely and responsibly.

StemExpress has been a trusted provider of widescale COVID-19 testing solutions since early 2020 - providing testing for government agencies, public health departments, private sector organizations, and the public nationwide. For Meeting on the Mesa, StemExpress is offering convenient testing options for unvaccinated attendees and those traveling from outside of the country. Options will include take-home RT-PCR COVID Self-Testing Kits and on-site, rapid PCR testing for the duration of the event. The self-testing kit option allows attendees to test for COVID in the days leading up to the event for a seamless admission and the days following the event to confirm they haven't been exposed. The on-site rapid testing option utilizes the new Thermo Fisher Accula, offering in-person testing at the event with results in around 30 minutes. StemExpress is excited to bring these state-of-the-art COVID testing solutions to the frontlines of the Cell & Gene industry to allow for safe in-person connections.

The StemExpress partnership with Alliance for Regenerative Medicine seeks to empower the entire cell and gene industry with a long-awaited opportunity to return to traditional networking practices. It is well known that innovation doesn't exist in a vacuum - allowing great minds to come together is a sure way to spur scientific growth and advance cutting-edge research, giving hope for future cures.

Cell and Gene Meeting on the Mesa will take place October 12th, 2021, through October 14th, 2021, at Park Hyatt Aviara,7100 Aviara Resort Drive Carlsbad, CA 92011. To learn more about the event, please visit MeetingOnTheMesa.com.

For more information about COVID testing solutions for businesses and events, visit https://www.stemexpress.com/covid-19-testing/.

About StemExpress:

Founded in 2010 and headquartered in Sacramento, California, StemExpress is a leading global biospecimen provider of human primary cells, stem cells, bone marrow, cord blood, peripheral blood, and disease-state products. Its products are used for research and development, clinical trials, and commercial production of cell and gene therapies by academic, biotech, diagnostic, pharmaceutical, and contract research organizations (CRO's).

StemExpress has over a dozen global distribution partners and seven (7) brick-and-mortar cellular clinics in the United States, outfitted with GMP certified laboratories. StemExpress runs its own non-profit supporting STEM initiatives, college and high school internships, and women-led organizations. It is registered with the U.S. Food and Drug Administration (FDA) and is continuously expanding its network of healthcare partnerships, which currently includes over 50 hospitals in Europe and 3 US healthcare systems - encompassing 31 hospitals, 35 outpatient facilities, and over 200 individual practices and clinics.

StemExpress has been ranked by Inc. 500 as one of the fastest-growing companies in the U.S.

About the Alliance for Regenerative Medicine:

The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies. ARM promotes legislative, regulatory, reimbursement and manufacturing initiatives to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-based therapies. Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing regenerative medicines and advanced therapies. In its 12-year history, ARM has become the voice of the sector, representing the interests of 400+ members worldwide, including small and large companies, academic research institutions, major medical centers and patient groups. To learn more about ARM or to become a member, visit http://www.alliancerm.org.

Media Contact: Anthony Tucker, atucker@stemexpress.com

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StemExpress Partners with the Alliance for Regenerative Medicine to Provide COVID-19 Testing for the Cell and Gene Meeting on the Mesa - WSAW

Faster healing of wounds can decrease pain and suffering and save lives – KY3

Published: Oct. 4, 2021 at 3:02 PM CDT

ORLANDO, Fla., Oct. 4, 2021 /PRNewswire/ --Billions of dollars are spent every year because of complications of wound healing. Researchers at the College of Medicine at the University of Central Florida (UCF) in Orlando have discovered a new technology to accelerate wound healing. Their research is published in the Life Cell Biology and Tissue Engineering Journal (https://pubmed.ncbi.nlm.nih.gov/34575027/). The UCF lab's research focus is to develop stem cell therapies for neurodegenerative diseases, including Alzheimer's disease, Parkinson's disease, wound healing and ALS.

Researchers at the College of Medicine at UCF in Orlando have discovered a new technology to accelerate wound healing.

Dr. Frederick R Carrick, Professor of Neurology at the College of Medicine at UCF, reported that animals with wounds and injured stem cells that were placed on a special ceramic blanket healed much faster than controls. Gladiator Therapeutics manufactured the therapeutic ceramic blanket that was used in this research. The researchers reported that wounds in animals and in stem cells were both repaired significantly faster when they treated them with the ceramic blankets.

This research was designed and accepted for presentation at the USA Department of Defense's premier scientific meeting, the Military Health System Research Symposium (MHSRS). Dr Carrick stated that the new ceramic blankets do not need a power supply and are ideally suited for use in both combat and civilian wound treatments. Large wounds, such as those suffered in combat are easily infected and may result in increased suffering, disability and death amongst Warfighters. Faster healing of wounds can decrease pain and suffering and save lives.

The UCF College of Medicine research team is conducting ongoing research on the use of the Gladiator ceramic blanket in animal models of Alzheimer's and Parkinson's disease, traumatic brain injury and wound care. They have recently developed a new Alzheimer's therapy combining drugs that affect stem cells that increase the development of brain cells and improve brain function. The UCF lab is also the first to transplant stem cells isolated from the human brain to aged rats where they showed increased development of new brain cells and improvement of cognition.

Dr. Kiminobu Sugaya, Professor of Medicine at the UCF College of Medicine is excited about their findings. Dr. Sugaya stated that the benefits of using the Gladiator ceramic blanket are that it can be used anywhere without a power supply or the side effects that are commonly found when injecting chemicals or drugs.

Further information about this study:

drfrcarrick@post.harvard.edu 321-868-6464

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Faster healing of wounds can decrease pain and suffering and save lives - KY3

Global Hemoglobinopathies Markets, 2021-2028 – High Unmet Needs / Increasing R&D Investment / Initiatives to Improve Disease Awareness -…

DUBLIN--(BUSINESS WIRE)--The "Global Hemoglobinopathies Market Size, Share & Trends Analysis Report by Type (Thalassemia, Sickle Cell Disease, Other Hemoglobin (Hb) Variants), by Diagnosis, by Therapy, by Region, and Segment Forecasts, 2021-2028" report has been added to ResearchAndMarkets.com's offering.

The global hemoglobinopathies market size is expected to reach USD 15.7 billion by 2028, expanding at a CAGR of 10.8%

Increasing awareness regarding hemoglobinopathies and government initiatives to diagnose the diseases at an early stage are expected to propel market growth over the forecast period. Moreover, increasing R&D investment, the presence of a promising drug pipeline, and technologically advanced diagnostics platforms are expected to boost the growth of the market.

The development of novel curative technologies, such as CRISPR/Cas9 and hematopoietic stem cell transplantation, coupled with a promising pipeline, is expected to propel market growth. Moreover, the presence of regulatory agencies, such as the FDA, which are working toward improving drug approval rate by granting accelerated approval for hemoglobinopathies drugs, is expected to drive the market.

Initiatives such as the Sickle Cell Awareness Initiative (SCAI) are working toward educating the people about the disease, which will increase the diagnosis and treatment rate. SCAI also raises funds for individuals affected with sickle cell diseases (SCDs) and provides research funding.

Moreover, governments of Middle Eastern and Asian countries provide funds for R&D of hemoglobinopathies treatment. For instance, various programs undertaken by governments, such as thalassemia screening in neonates and providing medicines, are impacting the market growth positively.

The high patient population in low-income countries has encouraged market players and non-profit organizations to launch several initiatives to improve the access to the therapy.

For instance, in February 2019, the Access to Excellent Care for Sickle Cell Patients Pilot Program (ACCEL) was launched by Global Blood Therapeutics, Inc. in order to provide research funding for novel SCD projects, with an aim to expand the access to optimal healthcare for sickle cell disease.

Biopharmaceutical companies are collaborating with nonprofit organizations to promote public awareness about hemoglobinopathies.

An increase in investment and funding for the development of novel therapies to treat hemoglobinopathies will further boost the market growth over the forecast period. The National Heart, Lung, and Blood Institute (NHLBI) has significantly invested in research & development in sickle cell disease.

The NHLBI supports research work through various initiatives. Furthermore, private funding such as the Bronx Blood Research Fund (BBRF) provides a platform for research and management of thalassemia and other hemoglobinopathies.

Gene therapy has emerged as a promising treatment option for managing hemoglobin disorder as it targets the underlying genetic cause of the condition through the administration of one-time gene therapy and significantly reduces the need for blood transfusions.

The current pipeline of gene therapy products includes CTX001 (CRISPR Therapeutics), BIVV003 (Sangamo Therapeutics, Inc. & Bioverativ Inc), and HGB-206 (bluebird bio, Inc).

Hemoglobinopathies Market Report Highlights

Global Hemoglobinopathies Market Variables, Trends & Scope

Penetration and Growth Prospect Mapping

Epidemiology Assessment of Hemoglobinopathies

Market Variable Analysis

Market Restraint Analysis

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/8dphl0

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Global Hemoglobinopathies Markets, 2021-2028 - High Unmet Needs / Increasing R&D Investment / Initiatives to Improve Disease Awareness -...

Stanford neuroscientist’s ‘assembloids’ pave the way for innovative brain research – Scope

A Stanford neuroscientist has led the development of a novel brain research tool for understanding diseases of brain development.

A recent article in the journal Nature credits Stanford physician-neuroscientist Sergiu Pasca, MD, with blazing a trail toward a more profound understanding of early brain development, and of what can go wrong in the process, using a cell-based research innovation he named "assembloids."

In 2015, Pasca and his colleagues published a paper in Nature Methods describing a fascinating feat: His tinkering with induced pluripotent stem cells, or iPS cells -- former skin cells transformed so that they've acquired an almost magical capacity to generate all the tissues in the body -- had borne a three-dimensional product. From these "magic" iPS cells grew a complex conglomerate of cells capable of modeling specific organs.

Pasca's particular interest was in the brain, and in the experiments detailed in the study, his lab had caused human iPS cells to multiply and differentiate into small spherical clusters of brain tissue suspended in laboratory glassware.

These clusters recapitulated the architecture and physiology of the human cerebral cortex -- the outermost layer of brain tissue, critical to perception, cognition and action. Pasca named these clusters, which grew to several millimeters in diameter and contained millions of cells, "cortical spheroids." Today, researchers around the world are using similar methodology to create models, broadly known as "organoids," to study other parts of the human body.

Two years later, in a study published in Nature, Pasca upped the ante by, first, generating a second kind of neural spheroid -- this time, representative of a deeper part of the developing forebrain called the subpallium -- and, second, by growing this kind of spheroid in conjunction with cortical spheroids, in the same dish.

To the researchers' amazement, spheroids of both types fused together, with nerve cells from subpallial spheroids migrating and poking extensions into the cortical spheroids and establishing working connections with nerve cells of a different type in the latter spheroids, just as occurs in fetal development.

"It's amazing that these cells already self-organize and know what they need to do," Pasca marveled in "Brain Balls," an article I wrote for our magazine, Stanford Medicine, a few years ago.

Pasca sensibly dubbed the two-fused-spheroid combos "assembloids," the Nature recap notes.

But why stop at two? Pasca has since created three-element assembloids composed of spheroids representative of cerebral cortex, spinal cord and skeletal muscle in order to model the circuitry of voluntary movement. He's also shown that stimulating the "cerebral cortex" spheroid can result in contraction of the "muscle" spheroid. (This accomplishment was published in Cell in late 2020.) He has explored other assembloid combinations, as well, such as the fusing of cortical spheroids with spheroids representing the striatum, a brain structure implicated in regulating our movements and responses to rewarding and aversive stimuli.

Because each spheroid begins with skin cells, they can be grown on a personalized basis -- and can therefore be extracted from patients with neurological disorders known or suspected to spring from early developmental aberrations (such as autism or schizophrenia). The cells can then be used to create models to probe these disorders' molecular, cellular and circuit-based deviations from the pathways of normal brain development, allowing scientists to study the brain in way they could never do with a living patient.

From the Nature article:

Assembloids are now at the leading edge of stem-cell research. Scientists are using them to investigate early events in organ development as tools for studying not only psychiatric disorders, but other types of disease as well.

An assembloid is by no means a complete, working brain. But, the article notes, "Pasca stands by the aphorism that all models are wrong, and some are useful. 'There's been important progress in the field in a short period of time,' he says."

Photo courtesy of the Pasca laboratory

Global health

Stanford researchers find that "entertainment education" helps teach new mothers about the importance of breastfeeding.

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Stanford neuroscientist's 'assembloids' pave the way for innovative brain research - Scope

BrainStorm to Present at the 2021 Cell & Gene Meeting on the Mesa – WWNY

Published: Oct. 4, 2021 at 6:00 AM EDT

NEW YORK, Oct. 4, 2021 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, announced today that Stacy Lindborg, Ph.D., Executive Vice President and Head of Global Clinical Research, will deliver a presentation at the2021 Cell & Gene Meeting on the Mesa, being held as a hybrid conferenceOctober 12-14, and October 19-20, 2021.

Dr. Lindborg's presentation highlights the expansion of Brainstorm's technology portfolio to include autologous and allogeneic product candidates, covering multiple neurological diseases. The most progressed clinical development program, which includes a completed phase 3 trial of NurOwn in ALS patients, remains the highest priority for Brainstorm. Brainstorm is committed to pursuing the best and most expeditious path forward to enable patients to access NurOwn.

Dr. Lindborg's presentation will be in the form of an on-demand webinar that will be available beginning October 12. Those who wish to listen to the presentation are required to registerhere. At the conclusion of the 2021 Cell & Gene Meeting on the Mesa, a copy of the presentation will also be available in the "Investors and Media" section of the BrainStorm website underEvents and Presentations.

About the 2021 Cell & Gene Meeting on the Mesa

The meeting will feature sessions and workshops covering a mix of commercialization topics related to the cell and gene therapy sector including the latest updates on market access and reimbursement schemes, international regulation harmonization, manufacturing and CMC challenges, investment opportunities for the sector, among others. There will be over 135 presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering and broader regenerative medicine technologies.

The conference will be delivered in a hybrid format to allow for an in-person experience as well as a virtual participation option. The in-person conference will take place October 12-14 in Carlsbad, CA. Virtual registrants will have access to all content via livestream during program dates. Additionally, all content will be available on-demand within 24 hours of the live program time. Virtual partnering meetings will take place October 19-20 via Zoom.

About NurOwn

The NurOwntechnology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwntechnology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive multiple sclerosis (MS) and was supported by a grant from the National MS Society (NMSS).

For more information, visit the company's website atwww.brainstorm-cell.com.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future NurOwnmanufacturing and clinical development plans, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect,""likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, the prospects for regulatory approval of BrainStorm's NurOwntreatment candidate, the initiation, completion, and success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwntreatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture, or to use third parties to manufacture, and commercialize the NurOwntreatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

ContactsInvestor Relations: Eric Goldstein LifeSci Advisors, LLC Phone: +1 646.791.9729 egoldstein@lifesciadvisors.com

Media:Paul Tyahla SmithSolve Phone: + 1.973.713.3768 Paul.tyahla@smithsolve.com

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BrainStorm to Present at the 2021 Cell & Gene Meeting on the Mesa - WWNY

Exosome therapeutic Market 2021 is Showing Impressive Growth by 2028 | Trends, Share, Size, Top Key Players Analysis and Forecast Research EcoChunk -…

DBMR has added another report named Exosome therapeutic Market with information Tables for recorded and figure years addressed with Chats and Graphs spread through Pages with straightforward definite examination. The a-list report concentrates on broad assessment of the market development expectations and limitations. The systems range from new item dispatches, extensions, arrangements, joint endeavors, organizations, to acquisitions. This report includes profound information and data on what the markets definition, characterizations, applications, and commitment and furthermore clarifies the drivers and restrictions of the market which is gotten from SWOT investigation. Worldwide market examination report serves a great deal for the business and presents with answer for the hardest business questions. While making Exosome therapeutic Market report, examination and investigation has been completed with one stage or the mix of a few stages relying on the business and customer necessities.

Market definition canvassed in the predominant Exosome therapeutic Market advertising report investigates the market drivers that show factors causing ascend in the market development and market limitations which demonstrate the components causing fall in the market development. It helps clients or other market members to know about the issues they might confront while working in this market throughout a more extended timeframe. This statistical surveying report additionally concentrates on utilization of market, central participants included, deals, value, income and portion of the overall industry with volume and an incentive for every area. The greatness and straightforwardness proceeded in xyz business research report makes acquire the trust and dependence of part organizations and clients.

Exosome therapeutic market is expected to gain market growth in the forecast period of 2019 to 2026. Data Bridge Market Research analyses that the market is growing with a CAGR of 21.9% in the forecast period of 2019 to 2026 and expected to reach USD 31,691.52 million by 2026 from USD 6,500.00 million in 2018. Increasing prevalence of lyme disease, chronic inflammation, autoimmune disease and other chronic degenerative diseases are the factors for the market growth.

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Exosomes are used to transfer RNA, DNA, and proteins to other cells in the body by making alteration in the function of the target cells. Increasing research activities in exosome therapeutic is augmenting the market growth as demand for exosome therapeutic has increased among healthcare professionals.

Increased number of exosome therapeutics as compared to the past few years will accelerate the market growth. Companies are receiving funding for exosome therapeutic research and clinical trials. For instance, In September 2018, EXOCOBIO has raised USD 27 million in its series B funding. The company has raised USD 46 million as series a funding in April 2017. The series B funding will help the company to set up GMP-compliant exosome industrial facilities to enhance production of exosomes to commercialize in cosmetics and pharmaceutical industry.

Increasing demand for anti-aging therapies will also drive the market. Unmet medical needs such as very few therapeutic are approved by the regulatory authority for the treatment in comparison to the demand in global exosome therapeutics market will hamper the market growth market. Availability of various exosome isolation and purification techniques is further creates new opportunities for exosome therapeutics as they will help company in isolation and purification of exosomes from dendritic cells, mesenchymal stem cells, blood, milk, body fluids, saliva, and urine and from others sources. Such policies support exosome therapeutic market growth in the forecast period to 2019-2026.

This exosome therapeutic market report provides details of market share, new developments, and product pipeline analysis, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, product approvals, strategic decisions, product launches, geographic expansions, and technological innovations in the market. To understand the analysis and the market scenario contact us for anAnalyst Brief, our team will help you create a revenue impact solution to achieve your desired goal.

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Competitive Landscape and Exosome Therapeutic Market Share Analysis

Global exosome therapeutic market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, company strengths and weaknesses, product launch, product trials pipelines, concept cars, product approvals, patents, product width and breadth, application dominance, technology lifeline curve. The above data points provided are only related to the companys focus related to global exosome therapeutic market.

The major players covered in the report are evox THERAPEUTICS, EXOCOBIO, Exopharm, AEGLE Therapeutics, United Therapeutics Corporation, Codiak BioSciences, Jazz Pharmaceuticals, Inc., Boehringer Ingelheim International GmbH, ReNeuron Group plc, Capricor Therapeutics, Avalon Globocare Corp., CREATIVE MEDICAL TECHNOLOGY HOLDINGS INC., Stem Cells Group among other players domestic and global. Exosome therapeutic market share data is available for Global, North America, Europe, Asia-Pacific, and Latin America separately. DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.

Many joint ventures and developments are also initiated by the companies worldwide which are also accelerating the global exosome therapeutic market.

For instance,

Partnership, joint ventures and other strategies enhances the company market share with increased coverage and presence. It also provides the benefit for organisation to improve their offering for exosome therapeutics through expanded model range.

Global Exosome Therapeutic Market Scope and Market Size

Global exosome therapeutic market is segmented of the basis of type, source, therapy, transporting capacity, application, route of administration and end user. The growth among segments helps you analyse niche pockets of growth and strategies to approach the market and determine your core application areas and the difference in your target markets.

Based on type, the market is segmented into natural exosomes and hybrid exosomes. Natural exosomes are dominating in the market because natural exosomes are used in various biological and pathological processes as well as natural exosomes has many advantages such as good biocompatibility and reduced clearance rate compare than hybrid exosomes.

Exosome is an extracellular vesicle which is released from cells, particularly from stem cells. Exosome functions as vehicle for particular proteins and genetic information and other cells. Exosome plays a vital role in the rejuvenation and communication of all the cells in our body while not themselves being cells at all. Research has projected that communication between cells is significant in maintenance of healthy cellular terrain. Chronic disease, age, genetic disorders and environmental factors can affect stem cells communication with other cells and can lead to distribution in the healing process. The growth of the global exosome therapeutic market reflects global and country-wide increase in prevalence of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases, along with increasing demand for anti-aging therapies. Additionally major factors expected to contribute in growth of the global exosome therapeutic market in future are emerging therapeutic value of exosome, availability of various exosome isolation and purification techniques, technological advancements in exosome and rising healthcare infrastructure.

Rising demand of exosome therapeutic across the globe as exosome therapeutic is expected to be one of the most prominent therapies for autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases treatment, according to clinical researches exosomes help to processes regulation within the body during treatment of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases. This factor has increased the research activities in exosome therapeutic development around the world for exosome therapeutic. Hence, this factor is leading the clinician and researches to shift towards exosome therapeutic. In the current scenario the exosome therapeutic are highly used in treatment of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases and as anti-aging therapy as it Exosomes has proliferation of fibroblast cells which is significant in maintenance of skin elasticity and strength.

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Huge Investment by Automakers for Exosome Therapeutics and New Technology Penetration

Global exosome therapeutic market also provides you with detailed market analysis for every country growth in pharma industry with exosome therapeutic sales, impact of technological development in exosome therapeutic and changes in regulatory scenarios with their support for the exosome therapeutic market. The data is available for historic period 2010 to 2017.

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Exosome therapeutic Market 2021 is Showing Impressive Growth by 2028 | Trends, Share, Size, Top Key Players Analysis and Forecast Research EcoChunk -...

Advancing the Development of Innovative Veterinary Products – FDA.gov

By: Steven M. Solomon, D.V.M., M.P.H., Director, Center for Veterinary Medicine

The U.S. Food and Drug Administrations Center for Veterinary Medicine (CVM) plays a vital role in facilitating the development and availability of innovative medical products for animals. Our role in promoting public health drives us to take steps that can help facilitate access to safe, effective, and innovative products to address existing, new, and emerging animal health challenges. The FDA takes a proactive approach, using all of our available tools and flexible processes, to provide timely and efficient review approaches to ensure safety and effectiveness and help bring these innovations to the market.

Today the FDA released four final guidance documents that demonstrate our commitment to innovative approaches. The guidance documents describe pathways for animal drug sponsors to use new approaches like adaptive study designs, real world evidence and biomarkers to establish drug effectiveness and more detailed guidance on how to leverage data collected from foreign countries to support approval of their products in the U.S. These guidance documents encourage animal drug sponsors to consider these innovative approaches as they prepare data submissions related to new animal drug products for agency evaluation and review, as part of the FDAs new animal drug approval process. The recommendations in these guidance documents closely align with those already issued by the FDAs other medical product centers.

CVM is also committed to the identification and use of alternative methods to bring innovative products to the market by encouraging use of novel approaches to meet the regulatory standards. The typical process for demonstrating drug effectiveness is conducting a clinical trial; however, there are other ways to prove an animal drug works and alternative study designs to use when the standard design doesnt fit the situation.

For example, CVM evaluated a range of data sources that demonstrate the safety of ThyroKare in dogs, a drug used to treat hypothyroidism, as an alternative to conducting laboratory animal studies. These sources included (1) a comprehensive review of publicly available literature on the use of levothyroxine in dogs; (2) pharmacovigilance data for ThyroKare that were voluntarily reported to the sponsor when it was previously marketed as an unapproved drug; and (3) reports to an animal poison control center of accidental overdoses of natural or synthetic thyroid hormone products in dogs.

In 2018, CVM approved Experior (lubabegron Type A medicated article), the first animal drug that when fed to beef cattle under specific conditions results in less ammonia gas released as a by-product of their waste. The drug sponsor collected data in special environmental chambers where gas emissions could be contained and accurately measured. These studies indicated that the product partially reduces ammonia gas emissions from manure from an individual animal or a pen of animals in semi-controlled conditions in enclosed housing.

Additionally, novel products, such as intentional genomic alterations (IGAs) in animals and animal cell and tissue-based products, offer the potential to treat or even cure diseases or conditions for which limited treatment options exist. The fields of veterinary regenerative medicine (e.g., stem cells) and IGAs in animals are fast-growing sectors of the industry and an active area of animal health research. While the same laws and regulations that apply to all other animal drugs also apply to these rapidly developing products, the FDA initiated the Veterinary Innovation Program (VIP) in 2019 to facilitate advancements in development of innovative animal products. The goals of the VIP are to provide greater certainty in the regulatory process, encourage development and research, and support an efficient and predictable pathway to approval for cellular products and IGAs in animals. As of September 2021, there were more than 40 animal drug products enrolled in the VIP program. IGA animals have potentially significant and broad applications to advance animal and human health including development of animal models of human disease, human pharmaceutical production, production of organs/tissues for xenotransplantation, improved animal health/husbandry and enhanced food production. Given the unique nature and evolving science associated with these products, the FDA uses a science- and risk-based review approach to assess the type and amount of information needed to support approval.

In December 2020, CVM approved a first-of-its-kind IGA in a line of domestic pigs, referred to as GalSafe pigs, which may be used for food or human therapeutics. This is the first IGA in an animal that the CVM has approved for both human food consumption and as a source for potential therapeutic uses. This approval underscores the success of the FDA in modernizing our scientific processes to optimize a risk-based approach that advances cutting-edge innovations in which consumers can have confidence. The developer of GalSafe pigs was the first product approved under the VIP program, demonstrating the impact of this initiative in supporting the sponsor throughout the product review process.

The FDA also helps support innovative product development by using legislative authorities designed to increase drug availability for minor animal species or minor uses in major animal species while ensuring critical standards are still met. One way the agency does this is through the conditional approval pathway for new animal drugs. Established as part of the Minor Use and Minor Species (MUMS) Animal Health Act in 2004, conditional approval allows a drug company to legally market a drug for use in a minor species, such as ornamental fish, ferrets, sheep, goats or honey bees, or for a use in a major species (horses, dogs, cats, cattle, pigs, turkeys and chickens) afflicted with an uncommon disease or condition (minor use) after proving the drug is safe and that there is reasonable expectation that it will be effective.

Recently, this program has helped facilitate development and approval of certain innovative canine cancer drugs. For example, in January 2017, the FDA conditionally approved the first drug to treat lymphoma in dogs, and this drug became the first conditionally-approved companion animal drug to achieve full approval in July of this year. In January 2021, the FDA conditionally approved the second drug and first oral tablet to treat lymphoma in dogs. The ability to utilize the MUMS conditional approval pathway enabled access to critical treatments for lymphoma in dogs before evaluation of all of the effectiveness data typical for a full approval had been completed.

In keeping with the goals of reducing, replacing, and/or refining the use of animals in research, CVM is also conducting a study at its research facility to validate an alternative in-vitro dissolution model approach for bioequivalence studies for certain animal drugs. If this study validates the bioequivalence model, this alternative approach will provide animal drug sponsors with a scientifically sound method to demonstrate bioequivalence of certain drugs that does not require that dogs be euthanized as part of the study.

CVMs flexible, collaborative and proactive approach to the regulation of animal drugs has facilitated the availability of critically needed drugs for conditions like heart failure, hypertension, cancer, epilepsy and pain and brought a number of generic animal drugs to market to enhance competition and drive innovation. These approaches have also facilitated the introduction of novel technologies with One Health impacts beyond animal health like reduction in gas emissions from an animals waste and IGAs in animals that produce biopharmaceuticals used in the production of human drugs. We are committed to continuing our collaborations with a variety of stakeholders to ensure the efficiency of our review and approval process for new animal drugs, and that we are supporting innovative approaches that ultimately help safe and effective animal drugs reach the marketplace.

For more information: Innovation in Animal Drug Development

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Advancing the Development of Innovative Veterinary Products - FDA.gov

Cell Based Assay & High Content Screening Market Report 2021: The Workhorse of the Pharmaceutical Industry is Becoming a Central Player in…

DUBLIN, Oct. 4, 2021 /PRNewswire/ -- The "Cell Based Assay & High Content Screening Markets" report has been added to ResearchAndMarkets.com's offering.

Research and Markets Logo

Cell-Based Assays are a mainstay of drug development and scientific research, but growth is now accelerating as the race for a COVID-19 cure gains speed.

On top of this, new technology is allowing Cell-Based Assays to be used to measure any aspect of cell function. This market just keeps on growing with no end in sight. The workhorse of the pharmaceutical industry is becoming a central player in biotechnology.

This is a complex area but this readable report will bring the entire management team up to speed, on both the technology and the opportunity.

The technology is moving faster than the market. Genomics and Immunology are playing a role too. Find the opportunities and pitfalls. Understand growth expectations and the ultimate potential market size.

Industry Overview

Players in a Dynamic Market

Academic Research Lab

Contract Research Organization

Genomic Instrumentation Supplier

Cell Line and Reagent Supplier

Pharmaceutical Company

Audit Body

Certification Body

Market Trends

Factors Driving Growth

Candidate Growth

Immuno-oncology

Genomic Blizzard

Technology Convergence

The Insurance Effect

Factors Limiting Growth

Technology Development

Cell Based Assays Recent Developments

Axxam and FUJIFILM Cellular Dynamics Announce Strategic Alliance

Cancer Genetics to Acquire Organoid Startup Stemonix

Curi Bio Acquires Artificial Intelligence Firm Dana Solutions

CRISPR Screens Uncover Novel Cancer Therapy Targets

ERS Genomics Licenses CRISPR-Cas9 Patents to Axxam

New Transcriptomics Assay Facilitates Compound Screens

Carta Biosciences Betting on Gene Interaction Mapping

High-throughput Identifies cancer drug candidates

Velabs Therapeutics partners with Alytas Therapeutics to develop a novel immune-based therapy for obesity

InSphero platform selected to test Cyclerion's sGC stimulator technology

OcellO to provide in vitro research services to Merus

Charles River Laboratories to acquire Citoxlab

Reaction Biology Corporation Purchases ProQinase GmbH

Cisbio extends its assay portfolio for immuno-oncology drug discovery

STEMCELL Technologies Launches Next-Generation Culture System

Abcam Acquires Calico Biolabs

Evotec announces achievement in Celgene alliance utilizing IPSC screening

Fujifilm Cellular Dynamics Inc. launches iCELL Microglia

Cisbio and Excellerate Bioscience partner

Horizon Discovery extends CRISPR Screening Service to primary human T cells

Profiles of Key Cell Based Assay Companies

Story continues

Abcam

Agilent

Aurora Instruments Ltd

Axxam

Beckman Coulter Diagnostics

Becton, Dickinson and Company

BioIVT

Bio-Rad Laboratories, Inc.

BioTek Instruments

BioVision, Inc.

BMG Labtech

Cell Biolabs, Inc

Cell Signaling Technology, Inc.

Charles River Laboratories

Cisbio Bioassays

Corning, Inc

Cytovale

Enzo Life Sciences, Inc

Eurofins DiscoverX Corporation

Evotec AG

Excellerate Bioscience

Fujifilm Cellular Dynamics International

Genedata

Hemogenix

Horizon Discovery

Invivogen

Leica Biosystems

Lonza Group Ltd.

Luminex Corp

Merck & Co., Inc

Miltenyi Biotec

Molecular Devices

Nanion

Ncardia

New England Biolabs, Inc

Olympus

Origene Technologies

Perkin Elmer

Promega

Qiagen Gmbh

Reaction Biology

Recursion Pharma

Roche Diagnostics

Sartorius

Sartorius-ForteBio

Sartorius-IntelliCyt

Sony Biotechnology

SPT Labtech

Stemcells Technologies Canada Inc

Tecan

Thermo Fisher Scientific Inc.

Vitro Biopharma

For more information about this report visit https://www.researchandmarkets.com/r/olr3vr

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Cell Based Assay & High Content Screening Market Report 2021: The Workhorse of the Pharmaceutical Industry is Becoming a Central Player in...

Pharmaxis Cleared To Progress To Phase 2 Bone Marrow Cancer Trial – WBKO

SYDNEY, Oct. 5, 2021 /PRNewswire/ -- Clinical stage drug development company Pharmaxis Ltd (ASX: PXS) today announced further positive results of data analysis from a phase 1c clinical trial (MF-101) studying its drug PXS-5505 in patients with the bone marrow cancer myelofibrosis for 28 days at three dosage levels.

Assessment with Pharmaxis' proprietary assays of the highest dose has shown inhibition of the target enzymes, LOX and LOXL2, at greater than 90% over a 24-hour period at day 7 and day 28. The trial safety committee has reviewed the results and having identified no safety signals, has cleared the study to progress to the phase 2 dose expansion phase where 24 patients will be treated at the highest dose twice a day for 6 months.

Pharmaxis CEO Gary Phillips said, "We are very pleased to have completed the dose escalation phase of this study with such clear and positive findings.We will now immediately progress to the phase 2 dose expansion study where we aim to show PXS-5505 is safe to be taken longer term with the disease modifying effects that we have seen in the pre-clinical models. The trial infrastructure and funding is in place and we are on track to complete the study by the end of 2022."

Independent, peer-reviewed research has demonstrated the upregulation of several lysyl oxidase family members in myelofibrosis.The level of inhibition of LOX achieved in the current study at all three doses significantly exceeds levels that caused disease modifying effects with PXS-5505 in pre-clinical models of myelofibrosis with improvements in blood cell count, diminished spleen size and reduced bone marrow fibrosis. LOXL2 was inhibited to a similar degree and based on pre-clinical work such high inhibition is likely replicated for other LOX family members (LOXL1, 3 and 4).[1] Study data can be viewed in the full announcement.

Commenting on the results of the trial, Dr Gabriela Hobbs, Assistant Professor, Medicine, Harvard Medical School & Clinical Director, Leukaemia, Massachusetts General Hospital said, "Despite improvements in the treatment of myelofibrosis, the only curative therapy remains an allogeneic stem cell transplantation, a therapy that many patients are not eligible for due to its morbidity and mortality. None of the drugs approved to date consistently or meaningfully alter the fibrosis that defines this disease. PXS-5505 has a novel mechanism of action by fully inhibiting all LOX enzymes. An attractive aspect of this drug is that so far in healthy controls and in this phase 1c study in myelofibrosis patients, the drug appears to be very well tolerated. This is meaningful as approved drugs and those that are undergoing study, are associated with abnormal low blood cell counts. Preliminary data thus far, demonstrate that PXS-5505 leads to a dramatic, >90% inhibition of LOX and LOXL2 at one week and 28 days. This confirms what's been shown in healthy controls as well as mouse models, that this drug can inhibit the LOX enzymes in patients. Inhibiting these enzymes is a novel approach to the treatment of myelofibrosis by preventing the deposition of fibrosis and ultimately reversing the fibrosis that characterizes this disease."

The phase 1c/2a trial MF-101 cleared by the FDA under the Investigational New Drug (IND) scheme aims to demonstrate that PXS-5505, the lead asset in Pharmaxis' drug discovery pipeline, is safe and effective as a monotherapy in myelofibrosis patients who are intolerant, unresponsive or ineligible for treatment with approved JAK inhibitor drugs. Trial sites will now open to recruit myelofibrosis patients into the 6-month phase 2 study in Australia, South Korea, Taiwan and the USA.

An effective pan-LOX inhibitor for myelofibrosis would open a market that is conservatively estimated at US$1 billion per annum.

While Pharmaxis' primary focus is the development of PXS-5505 for myelofibrosis, the drug also has potential in several other cancers including liver and pancreatic cancer where it aims to breakdown the fibrotic tissue in the tumour and enhance the effect of chemotherapy treatment.

Trial Design

Name of trial

PXS5505-MF-101: A phase 1/2a study to evaluate safety, pharmacokinetic and pharmacodynamic dose escalation and expansion study of PXS-5505 in patients with primary, post-polycythaemia vera or post-essential thrombocythemia myelofibrosis

Trial number

NCT04676529

Primary endpoint

To determine the safety of PXS-5505 in patients with myelofibrosis

Secondary endpoints

Blinding status

Open label

Placebo controlled

No

Trial design

Randomised, multicentre, 4 week duration phase 1 (dose escalation) followed by 6 month phase 2 (dose expansion)

Treatment route

Oral

Treatment frequency

Twice daily

Dose level

Dose escalation: three escalating doses

Dose expansion: one dose

Number of subjects

Dose escalation: minimum of three patients to maximum of 18 patients

Dose expansion: 24 patients

Subject selection criteria

Patients with primary or secondary myelofibrosis who are intolerant, unresponsive or ineligible for treatment with approved JAK inhibitor drugs

Trial locations

Dose escalation: Australia (2 sites) and South Korea (4 sites)

Dose expansion: Australia, Korea, Taiwan, USA

Commercial partners involved

No commercial partner

Reference: (1) doi.org/10.1002/ajh.23409

AUTHORISED FOR RELEASE TO ASX BY:

Pharmaxis Ltd Disclosure Committee. Contact: David McGarvey, Chief Financial Officer and Company Secretary: T +61 2 9454 7203, E david.mcgarvey@pharmaxis.com.au

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About Pharmaxis

Pharmaxis Ltd is an Australian clinical stage drug development company developing drugs for inflammatory and fibrotic diseases, with a focus on myelofibrosis. The company has a highly productive drug discovery engine built on its expertise in the chemistry of amine oxidase inhibitors, with drug candidates in clinical trials. Pharmaxis has also developed two respiratory products which are approved and supplied in global markets, generating ongoing revenue.

Pharmaxis is developing its drug PXS-5505 for the bone marrow cancer myelofibrosis which causes a build up of scar tissue that leads to loss of production of red and white blood cells and platelets. The US Food and Drug Administration has granted Orphan Drug Designation to PXS-5055 for the treatment of myelofibrosis and permission under an Investigational Drug Application (IND) to progress a phase 1c/2 clinical trial that began recruitment in Q1 2021. PXS5505 is also being investigated as a potential treatment for other cancers such as liver and pancreatic cancer.

Other drug candidates being developed from Pharmaxis' amine oxidase chemistry platform are targeting fibrotic diseases such as kidney fibrosis, NASH, pulmonary fibrosis and cardiac fibrosis; fibrotic scarring from burns and other trauma; and inflammatory diseases such as Duchenne Muscular Dystrophy.

Pharmaxis has developed two products from its proprietary spray drying technology that are manufactured and exported from its Sydney facility; Bronchitol for cystic fibrosis, which is approved and marketed in the United States, Europe, Russia and Australia; and Aridol for the assessment of asthma, which is approved and marketed in the United States, Europe, Australia and Asia.

Pharmaxis is listed on the Australian Securities Exchange (PXS). Its head office, manufacturing and research facilities are in Sydney, Australia. http://www.pharmaxis.com.au

About PXS-5505

PXS-5505 is an orally taken drug that inhibits the lysyl oxidase family of enzymes, two members LOX and LOXL2 are strongly upregulated in human myelofibrosis. In pre-clinical models of myelofibrosis PXS-5505 reversed the bone marrow fibrosis that drives morbidity and mortality in myelofibrosis and reduced many of the abnormalities associated with this disease. It has already received IND approval and Orphan Drug Designation from the FDA.

Myelofibrosis is a disorder in which normal bone marrow tissue is gradually replaced with a fibrous scar-like material. Over time, this leads to progressive bone marrow failure. Under normal conditions, the bone marrow provides a fine network of fibres on which the stem cells can divide and grow. Specialised cells in the bone marrow known as fibroblasts make these fibres.

In myelofibrosis, chemicals released by high numbers of platelets and abnormal megakaryocytes (platelet forming cells) over-stimulate the fibroblasts. This results in the overgrowth of thick coarse fibres in the bone marrow, which gradually replace normal bone marrow tissue. Over time this destroys the normal bone marrow environment, preventing the production of adequate numbers of red cells, white cells and platelets. This results in anaemia, low platelet counts and the production of blood cells in areas outside the bone marrow for example in the spleen and liver, which become enlarged as a result.

Myelofibrosis can occur at any age but is usually diagnosed later in life, between the ages of 60 and 70 years. The cause of myelofibrosis remains largely unknown. It can be classified as either JAK2 mutation positive (having the JAK2 mutation) or negative (not having the JAK2 mutation).

Source: Australian Leukemia Foundation: https://www.leukaemia.org.au/disease-information/myeloproliferative-disorders/types-of-mpn/primary-myelofibrosis/

Forward-looking statements

Forwardlooking statements in this media release include statements regarding our expectations, beliefs, hopes, goals, intentions, initiatives or strategies, including statements regarding the potential of products and drug candidates. All forward-looking statements included in this media release are based upon information available to us as of the date hereof. Actual results, performance or achievements could be significantly different from those expressed in, or implied by, these forward-looking statements. These forward-looking statements are not guarantees or predictions of future results, levels of performance, and involve known and unknown risks, uncertainties and other factors, many of which are beyond our control, and which may cause actual results to differ materially from those expressed in the statements contained in this document. For example, despite our efforts there is no certainty that we will be successful in developing or partnering any of the products in our pipeline on commercially acceptable terms, in a timely fashion or at all. Except as required by law we undertake no obligation to update these forward-looking statements as a result of new information, future events or otherwise.

CONTACT:

Media: Felicity Moffatt: T +61 418 677 701, E felicity.moffatt@pharmaxis.com.au

Investor relations:Rudi Michelson (Monsoon Communications) T +61 411 402 737, E rudim@monsoon.com.au

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Pharmaxis Cleared To Progress To Phase 2 Bone Marrow Cancer Trial - WBKO