Category Archives: Stell Cell Research


Evotec and Medical Center Hamburg-Eppendorf Enter Partnership to Develop iPSC-Based Tissue Therapy for Heart Failure – Yahoo Finance UK

Bloomberg

(Bloomberg) -- Stocks extended their rally into a fourth day as traders parsed through a flurry of corporate results amid signs the U.S. labor market may be gradually improving. The dollar climbed.The S&P 500 headed toward a record, led by banks and tech shares, as the Russell 2000 Index jumped 1.8%. EBay Inc. and PayPal Holdings Inc. surged on upbeat forecasts, while Netflix Inc. gained on news its raising the price of its service in Japan. Meanwhile, GameStop Corp. plunged as day traders flocked to other stocks like drug developers, and Nordstrom Inc. slid after announcing a strategy to win back customers -- a sign that Wall Street may have expected a more comprehensive overhaul. A widely watched segment of the Treasury yield curve steepened to levels last seen in 2015.The bull market in U.S. stocks remains on solid footing as the rebound in activity and corporate profits alongside an accommodative Federal Reserve create a supportive environment for equities, according to UBS Group AG. A report Thursday showed jobless claims fell to the lowest since November, and the next major update on the economy comes on Friday -- with analysts forecasting the labor market added about 100,000 jobs in January after a 140,000 drop in December. Selected high-frequency data, such as weekly consumer-confidence readings, also point to some strengthening.We certainly seem to have shifted our focus back to fundamentals, said Arthur Hogan, chief market strategist at National Securities Corp. The virus news is getting incrementally better at the very same time that the earnings season and economic data seem to be showing some improvement. Markets are actually focusing on what were supposed to be focused on and less concerned about the machinations of getting fiscal policy out and whats going on in Reddit-land.The Reddit-fueled rumble in the U.S. stock market may have heightened fears of another burst of volatility, according to options data tracked by Bloomberg. Over the last two weeks, the Cboe Volatility Indexs futures curve has shifted markedly higher, showing a pronounced peak in April before a gradual decline. The move suggests investors expect more volatility in the short-term amid concerns about extended valuations, the pace of the vaccine rollout and the impact of retail-trading activity.These are some of the main moves in markets:StocksThe S&P 500 increased 0.8% as of 3:34 p.m. New York time.The Stoxx Europe 600 Index rose 0.6%.The MSCI Asia Pacific Index decreased 0.7%.CurrenciesThe Bloomberg Dollar Spot Index rose 0.4%.The euro dipped 0.6% to $1.1965.The Japanese yen depreciated 0.5% to 105.55 per dollar.BondsThe yield on 10-year Treasuries fell less than one basis point to 1.14%.Germanys 10-year yield climbed one basis point to -0.45%.Britains 10-year yield jumped seven basis points to 0.44%.CommoditiesWest Texas Intermediate crude rose 1% to $56.26 a barrel.Gold lost 2.1% to $1,794.80 an ounce.Silver fell 2% to $26.35 per ounce.For more articles like this, please visit us at bloomberg.comSubscribe now to stay ahead with the most trusted business news source.2021 Bloomberg L.P.

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Evotec and Medical Center Hamburg-Eppendorf Enter Partnership to Develop iPSC-Based Tissue Therapy for Heart Failure - Yahoo Finance UK

Biobanks Market | Prominent Factors Analysis that will Help in Reshaping the Market Growth – BioSpace

Biobanks are essentially repositories for storing biological substances derived from humans, which may include organs, bio-specimens, plasma, saliva, and blood. With a vast rise in the number of organ replacement surgeries globally and significant advancements made in the fields of transfusion and replacement technologies, the role of the biobanks market has become more concrete in the healthcare sector in recent years.

The global biobanks market has witnessed expansion at a significant pace in the recent years owing to the vast rise in prevalence of a number of chronic diseases and the increased demand for personal medicine. The massive rise in incidence rate of conditions such as diabetes, cancer, neurovascular diseases, cardiovascular diseases, and respiratory diseases has compelled government bodies to take stronger actions in terms of investments in biobanks and increasing awareness about them. The global market for biobanks works in coherence with these efforts by adding pace to the process of drug discovery and the treatment of chronic diseases that are caused by activities such as smoking, consumption of alcohol, obesity, and unhealthy lifestyles.

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Heavy investment by a proactive government has boosted the North America biobanks market to the leading position in the global biobanks market, followed closely by Europe. Both regions are home to several bioscience companies and host a large number of drug discovery and research activities. Asia Pacific still remains the region with the most promising growth potential as the region shown high promise owing to an increase in investments from both government and non-government organizations, along with a large and increasingly affluent population base that is gaining awareness about the market.

Global Biobanks Market: Overview

The global biobanks market owes its conception to the rising need for replacement organs and the steady advancement in the replacement and transfusion technologies regarding a number of bodily substances. Though the technology and need to store organs and other bio-entities had been available for a long time, the global biobanks market took on a more important role in the healthcare sector following the increasing research in genomics. In the new millennium, the development of the personalized medicine field has been the vital driver for the global biobanks market. The likely advancement of the latter, thanks to helpful government regulations, is likely to make the crucial difference for the global biobanks market in the near future.

The steady technological advancement in the healthcare sector in the last few decades has now led to a scenario where the full potential of biobanks can be harnessed. As a result, the global biobanks market is projected to exhibit steady growth over the coming years.

Global Biobanks Market: Key Trends

The rising interest in personalized medicine is the prime driver for the global biobanks market. Personalized medicine has only become a viable branch of modern medicine after steady research in genomics and the way various patients react to various medicines. The biobanks market has thus come into the spotlight thanks to their role as a steady supplier of human biomaterials for research as well as direct application. The increasing research in genomics following the findings of the Human Genome Project is thus likely to remain a key driver for the global biobanks market in the coming years.

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The utilization of biobanks in stem cell research has been hampered in several regions by ethical concerns regarding the origin of stem cells. Nevertheless, the potential of stem cells in the healthcare sector is immense, and is likely to have a decisive impact on the trajectory of the global biobanks market in the coming years. Many countries have, in recent years, adopted a supportive stance towards stem cell research, aiding the growth of the biobanks market. Continued government support is thus likely to remain vital for the global biobanks market in the coming years.

Global Biobanks Market: Market Potential

The leading role of the U.S. in the global biobanks market is unlikely to change in the coming years. The easy availability of government-supported healthcare infrastructure and the presence of several industry giants in the region has driven the biobanks market in the U.S.

Northwell Health, the largest healthcare provider in New York State, earlier in 2017 initiated a new biobank aimed at creating precision therapies against various types of cancer. Launched in collaboration with Indivumed, the biobank will provide catalogued biomaterials for research into lung, colorectal, breast, and pancreatic cancer. This would enable targeted, gene-specific studies of a variety of cancer samples, leading to a more comprehensive understanding of cancer. Such well-funded collaboration efforts are crucial for the developing biobanks market.

Global Biobanks Market: Geographical Dynamics

Led by the fertile healthcare research scenario in the U.S., North America is likely to retain a dominant share in the global biobanks market in the coming years. Steady support from institutes such as the NIH is likely to be vital for the North America biobanks market.

Emerging Asia Pacific economies such as India and China could emerge with a significant share in the global biobanks market in the coming years. The healthcare sector in both countries has received steady public or private funding in the last few years. India is also a global leader in medical tourism and is likely to receive an increasing number of patients in the coming years, leading to promising prospects for the global biobanks market in the region.

Global Biobanks Market: Competitive Dynamics

Due to the dynamic nature of the global biobanks market, with advancements in diagnostic fields often determining the direction of the market, the market is heavily fragmented. It is likely to retain a significant degree of fragmentation in the coming years thanks to the diversity in the application segments of the biobanks market. The leading players in the global biobanks market include BioCision, Tecan Group, VWR, Beckman Coulter Inc., and Thermo Fisher Scientific.

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Biobanks Market | Prominent Factors Analysis that will Help in Reshaping the Market Growth - BioSpace

Alexion Reports Fourth Quarter and Full Year 2020 Results – BioSpace

Feb. 4, 2021 13:00 UTC

BOSTON--(BUSINESS WIRE)-- Alexion Pharmaceuticals Inc.. (NASDAQ:ALXN) today announced financial results for the fourth quarter and full year of 2020. Total revenues for the full year of 2020 were $6,069.9 million, a 22 percent increase compared to the same period in 2019. The negative impact of foreign currency on total revenues year-over-year was 1 percent, or $52.0 million, inclusive of hedging activities. On a GAAP basis, diluted EPS for the full year of 2020 was $2.72, compared to $10.70 in the prior year. Full year 2020 includes impairment charges of $2,053.3 million primarily relating to the KANUMA intangible asset and a related deferred tax benefit of $377.3 million. Full year 2019 includes one-time tax benefits of $382.2 million related to intra-entity asset transfers of intellectual property. Non-GAAP diluted EPS for the full year of 2020 was $12.51, a 19 percent increase versus the prior year.

Total revenues in the fourth quarter were $1,591.8 million, a 15 percent increase compared to the same period in 2019. The positive impact of foreign currency on total revenues year-over-year was less than 1 percent, or $0.1 million, inclusive of hedging activities. On a GAAP basis, diluted EPS in the quarter was $2.42, compared to $4.00 in the prior year, inclusive of one-time tax benefits of $382.2 million related to intra-entity asset transfers of intellectual property in the fourth quarter of 2019. Non-GAAP diluted EPS for the fourth quarter of 2020 was $2.96, a 9 percent increase versus the fourth quarter of 2019.

"In 2020, we delivered on our LEAD-EXPAND-DIVERSIFY strategy - progressing our commercial portfolio with multiple global regulatory approvals, and further building our pipeline, which now spans more than 20 development programs. I am so proud of our team's remarkable execution and perseverance amidst the uncertainties of COVID-19," said Ludwig Hantson, Ph.D., Chief Executive Officer of Alexion. "We enter 2021 with significant momentum, a strong foundation and a promising future. I am confident we are well positioned to build on our success and further advance our mission of delivering life-changing medicines to people with rare diseases and devastating conditions in the coming months and once we become part of AstraZeneca."

Full Year 2020 Financial Highlights

Fourth Quarter 2020 Financial Highlights

COVID-19

We continue to take steps to proactively respond to the evolving COVID-19 pandemic and to plan for related uncertainties. We remain focused on continuing to serve patients, protecting the health and safety of our employees and the communities in which we live and work, and supporting patients in clinical trials. We are also focused on minimizing potential interactions that could contribute to the spread of the virus and put additional strain on healthcare systems through the use of innovative virtual means where possible.

Research and Development

PHASE 3/4

PHASE 1/2

Conference Call/Earnings Materials:

Given the recently announced agreement for Alexion to be acquired by AstraZeneca, Alexion will not be hosting a conference call. Earnings materials are available publicly on the Investor Relations page of our website at http://ir.alexion.com. Questions may be directed to the Investor Relations team via e-mail at InvestorRelations@Alexion.com or the contact information below.

About Alexion

Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. As a leader in rare diseases for more than 25 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D) as well as the first and only approved Factor Xa inhibitor reversal agent. In addition, the company is developing several mid-to-late-stage therapies, including a copper-binding agent for Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D inhibitor as well as several early-stage therapies, including one for light chain (AL) amyloidosis, a second oral Factor D inhibitor and a third complement inhibitor. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on hematology, nephrology, neurology, metabolic disorders, cardiology, ophthalmology and acute care. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: http://www.alexion.com.

[ALXN-E]

Additional Information and Where to Find It

In connection with the proposed transaction, AstraZeneca PLC (AstraZeneca) intends to file with the SEC a registration statement on Form F-4 that will include a proxy statement of Alexion and that also constitutes a prospectus of AstraZeneca. Each of Alexion and AstraZeneca may also file other relevant documents with the U.S. Securities and Exchange Commission (SEC) regarding the proposed transaction. This document is not a substitute for the proxy statement/prospectus or registration statement or any other document that Alexion or AstraZeneca may file with the SEC. The definitive proxy statement/prospectus (if and when available) will be mailed to stockholders of Alexion. INVESTORS AND SECURITY HOLDERS ARE URGED TO READ THE REGISTRATION STATEMENT, PROXY STATEMENT/PROSPECTUS AND ANY OTHER RELEVANT DOCUMENTS THAT MAY BE FILED WITH THE SEC, AS WELL AS ANY AMENDMENTS OR SUPPLEMENTS TO THESE DOCUMENTS, CAREFULLY AND IN THEIR ENTIRETY IF AND WHEN THEY BECOME AVAILABLE BECAUSE THEY CONTAIN OR WILL CONTAIN IMPORTANT INFORMATION ABOUT THE PROPOSED TRANSACTION. Investors and security holders will be able to obtain free copies of the registration statement and proxy statement/prospectus (if and when available) and other documents containing important information about Alexion, AstraZeneca and the proposed transaction, once such documents are filed with the SEC through the website maintained by the SEC at http://www.sec.gov. Copies of the documents filed with the SEC by Alexion will be available free of charge on Alexions website at http://www.alexion.com or by contacting Alexions Investor Relations Department by email at InvestorRelations@alexion.com. Copies of the documents filed with the SEC by AstraZeneca will be available free of charge on AstraZenecas website at https://www.astrazeneca.com/investor-relations.html or by contacting AstraZenecas Investor Relations department by email at global-mediateam@astrazeneca.com.

Participants in the Solicitation

Alexion, AstraZeneca, their respective directors and certain of their executive officers and other employees may be deemed to be participants in the solicitation of proxies from Alexions stockholders in connection with the proposed transaction. Information regarding the persons who may, under the rules of the SEC, be deemed participants in the solicitation of Alexion stockholders in connection with the proposed mergers, including a description of their direct or indirect interests, by security holdings or otherwise, will be set forth in the proxy statement/prospectus when it is filed with the SEC. Information about Alexions directors and executive officers is available in Alexions proxy statement for its 2020 annual meeting of stockholders, which was filed with the SEC on March 26, 2020, Alexions Annual Report on Form 10-K for the fiscal year ended December 31, 2019, which was filed with the SEC on February 4, 2020, and other documents subsequently filed by Alexion with the SEC. Information about AstraZenecas directors and executive officers is available in AstraZenecas Form 20-F filed with the SEC on March 3, 2020, and other documents subsequently filed by AstraZeneca with the SEC.

No Offer or Solicitation

This communication is not intended to and shall not constitute an offer to buy or sell or the solicitation of an offer to buy or sell any securities, or a solicitation of any vote or approval, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such jurisdiction. No offering of securities shall be made, except by means of a prospectus meeting the requirements of Section 10 of the U.S. Securities Act of 1933, as amended.

Forward Looking Statements

This communication contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. You can generally identify forward-looking statements by the use of forward-looking terminology such as anticipate, believe, continue, could, estimate, expect, explore, evaluate, intend, may, might, plan, potential, predict, project, seek, should, or will, or the negative thereof or other variations thereon or comparable terminology. These forward-looking statements are only predictions and involve known and unknown risks and uncertainties, many of which are beyond Alexions control. Statements in this communication regarding Alexion, AstraZeneca and the combined company that are forward-looking, including anticipated benefits of the proposed transaction, the impact of the proposed transaction on Alexions and AstraZenecas businesses, that Alexion is well positioned to build on its success, the anticipated timing of initiation, enrollment, reporting results of clinical trials, the timing of filing for regulatory approvals and receipt of approvals are based on managements estimates, assumptions and projections, and are subject to significant uncertainties and other factors, many of which are beyond Alexions and AstraZenecas control. These factors include, among other things, market factors, completion of the audit of Alexions fiscal year 2020 financial results, competitive product development and approvals, pricing controls and pressures (including changes in rules and practices of managed care groups and institutional and governmental purchasers), economic conditions such as interest rate and currency exchange rate fluctuations, judicial decisions, claims and concerns that may arise regarding the safety and efficacy of in-line products and product candidates, changes to wholesaler inventory levels, variability in data provided by third parties, changes in, and interpretation of, governmental regulations and legislation affecting domestic or foreign operations, including tax obligations, changes to business or tax planning strategies, difficulties and delays in product development, manufacturing or sales including any potential future recalls, patent positions and the ultimate outcome of any litigation matter. Additional information concerning these risks, uncertainties and assumptions can be found in Alexions filings with the SEC, including the risk factors discussed in Alexions most recent Annual Report on Form 10-K, as updated by its Quarterly Reports on Form 10-Q, and in Alexions future filings with the SEC. Important risk factors could cause actual future results and other future events to differ materially from those currently estimated by management, including, but not limited to, the risks that: a condition to the closing the proposed acquisition may not be satisfied; a regulatory approval that may be required for the proposed acquisition is delayed, is not obtained or is obtained subject to conditions that are not anticipated; managements time and attention is diverted on transaction related issues; disruption from the transaction makes it more difficult to maintain business, contractual and operational relationships; the credit ratings of the combined company declines following the proposed acquisition; legal proceedings are instituted against Alexion, AstraZeneca or the combined company; Alexion is unable to retain key personnel; and the announcement or the consummation of the proposed acquisition has a negative effect on the market price of the capital stock of Alexion or on Alexions operating results. No assurances can be given that any of the events anticipated by the forward-looking statements will transpire or occur, or if any of them do occur, what impact they will have on the results of operations, financial condition or cash flows of Alexion. Should any risks and uncertainties develop into actual events, these developments could have a material adverse effect on the proposed transaction and/or Alexion. You are cautioned not to rely on Alexions forward-looking statements. These forward-looking statements are and will be based upon managements then-current views and assumptions regarding future events and operating performance, and are applicable only as of the dates of such statements. Alexion assumes no duty to update or revise forward-looking statements, whether as a result of new information, future events or otherwise, as of any future date.

In addition to financial information prepared in accordance with GAAP, this press release also contains non-GAAP financial measures that Alexion believes, when considered together with the GAAP information, provide investors and management with supplemental information relating to performance, trends and prospects that promote a more complete understanding of our operating results and financial position during different periods. Alexion also uses these non-GAAP financial measures to establish budgets, set operational goals and to evaluate the performance of the business. The non-GAAP results, determined in accordance with our internal policies, exclude the impact of the following GAAP items (see reconciliation tables below for additional information): share-based compensation expense, fair value adjustment of inventory acquired, amortization of purchased intangible assets, changes in fair value of contingent consideration, restructuring and related expenses, upfront payments related to licenses and other strategic agreements, acquired in-process research and development, impairment of purchased intangible assets, gains and losses related to strategic equity investments, litigation charges, gain or loss on sale of a business or asset, gain or loss related to modification of purchase options, contingent milestone payments associated with acquisitions of legal entities accounted for as asset acquisitions, acquisition-related costs and certain adjustments to income tax expense. These non-GAAP financial measures are not intended to be considered in isolation or as a substitute for, or superior to, the financial measures prepared and presented in accordance with GAAP, and should be reviewed in conjunction with the relevant GAAP financial measures. Please refer to the attached Reconciliation of GAAP to non-GAAP Financial Results for explanations of the amounts adjusted to arrive at non-GAAP net income and non-GAAP earnings per share amounts for the three and twelve month periods ended December 31, 2020 and 2019.

(Tables Follow)

ALEXION PHARMACEUTICALS, INC.

TABLE 1: CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

(in millions, except per share amounts)

(unaudited)

Three months ended

Twelve months ended

December 31,

December 31,

2020

2019

2020

2019

Net product sales

$

1,591.7

$

1,384.2

$

6,069.1

$

4,990.0

Other revenue

0.1

0.1

0.8

1.1

Total revenues

1,591.8

1,384.3

6,069.9

4,991.1

Costs and expenses:

Cost of sales (exclusive of amortization of purchased intangible assets)

152.2

114.3

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Alexion Reports Fourth Quarter and Full Year 2020 Results - BioSpace

Autologous Stem Cell and Non-Stem Cell Based Therapies Market 2020 Size, Share, Analysis, Growth Driver and Industry Segments by 2027 KSU | The…

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Autologous Stem Cell and Non-Stem Cell Based Therapies Market 2020 Size, Share, Analysis, Growth Driver and Industry Segments by 2027 KSU | The...

Transfection Technologies Market Size 2020 | Global Business Strategies, Growing CAGR of 20.0%, Industry Revenue, Opportunities-Aldrich Co Thermo…

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Market Definition

Transfection is an approach to produce exogenous nucleic acids such as DNA, RNA or oligonucleotide into cells. Such nucleic acids can be transferred by polymeric or lipid transfection reagents which promote the cellular absorption. This method is widely used for genomic studies (cell representation, testing, RNA interference, in vitro research) but can be conducted for bio-production (vaccine and protein manufacturing) or medicinal reasons (animal cell treatment). Nucleic acid delivery to cells can be accomplished by distinct physical techniques, such as electroporation, sonoporation or microinjection; however, these procedures are comparatively hazardous to cells. Transfection with chemical substances is a better option for maintaining healthy cell feasibility.

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List of Companies Profiled in the Transfection Technologies Market Report are:

Lonza Promega Corporation Sigma-Aldrich Co. Thermo Fisher Scientific Inc Bio-Rad Laboratories, Roche Molecular Systems QIAGEN Inovio Pharmaceuticals POLYPLUS TRANSFECTION Complete Report is Available (Including Full TOC, List of Tables & Figures, Graphs, and Chart) @https://www.databridgemarketresearch.com/toc/?dbmr=global-transfection-technologies-market&ab

Transfection Technologies Report displays data on key players, major collaborations, merger & acquisitions along with trending innovation and business policies. The report highlights current and future market trends and carries out analysis of the effect of buyers, substitutes, new entrants, competitors, and suppliers on the market. The key topics that have been explained in this Transfection Technologies market report include market definition, market segmentation, key developments, competitive analysis and research methodology. To accomplish maximum return on investment (ROI), its very essential to be acquainted with market parameters such as brand awareness, market landscape, possible future issues, industry trends and customer behavior where this Transfection Technologies report comes into play.

The Segments and Sub-Section of Transfection Technologies Market are shown below:

Segmentation: Global Transfection Technologies Market

By Transfection Method

Cotransfection

Electroporation

Cationic Lipid Transfection

In Vivo Transfection

By Applications

Virus Production

Protein Production

Gene Silencing

Stem Cell Reprogramming & Differentiation

Stable Cell Line Generation

Market Size Segmentation by Re gion & Countries (Customizable):

North America (Canada, United States & Mexico)

Europe (Germany, the United Kingdom, BeNeLux, France, Russia & Italy)

Asia-Pacific (Japan, South Korea, China, India & Southeast Asia)

South America (Argentina, Brazil, Peru, Colombia, Etc.)

Middle East & Africa (United Arab Emirates, Egypt, Saudi Arabia, Nigeria & South Africa)

For More Information or Query or Customization Before Buying,Visit@https://www.databridgemarketresearch.com/inquire-before-buying/?dbmr=global-transfection-technologies-market&ab

Market Drivers

Surge in research & development in the field of cell based therapies is contributing to the growth of the market

Massive funds by government and private players is boosting the growth of the market

Growing occurrences of cancer diseases is propelling the growth of the market

Increasing number of obese and overweight population is driving the growth of the market

Market Restraints

Cost of transfection technology instruments is hampering the growth of the market

Hazard of negative reaction with the cell is hindering the growth of the market

Home brew reagents restricts sale of business supply which is restricting the growth of the market

Strategic Points Covered in Table of Content of Global Transfection Technologies Market:

Chapter 1: Introduction, market driving force product Objective of Study and Research Scope the Transfection Technologies market

Chapter 2: Exclusive Summary the basic information of the Transfection Technologies Market.

Chapter 3: Displaying the Market Dynamics- Drivers, Trends and Challenges of the Transfection Technologies

Chapter 4: Presenting the Transfection Technologies Market Factor Analysis Porters Five Forces, Supply/Value Chain, PESTEL analysis, Market Entropy, Patent/Trademark Analysis.

Chapter 5: Displaying market size by Type, End User and Region 2010-2019

Chapter 6: Evaluating the leading manufacturers of the Transfection Technologies market which consists of its Competitive Landscape, Peer Group Analysis, BCG Matrix & Company Profile

Chapter 7: To evaluate the market by segments, by countries and by manufacturers with revenue share and sales by key countries (2020-2027).

Chapter 8 & 9: Displaying the Appendix, Methodology and Data Source

Finally, Transfection Technologies Market is a valuable source of guidance for individuals and companies in decision framework.

Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, Europe or Asia.

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Transfection Technologies Market Size 2020 | Global Business Strategies, Growing CAGR of 20.0%, Industry Revenue, Opportunities-Aldrich Co Thermo...

Global Apoptosis Assays Market Report 2020: Apoptosis Assay Kits Lead the Demand with 42.5% Share – Forecast to 2026 – GlobeNewswire

February 01, 2021 04:08 ET | Source: Research and Markets

Dublin, Feb. 01, 2021 (GLOBE NEWSWIRE) -- The "Global Apoptosis Assays Market - Products, Detection Technologies, Applications and End-Use Markets" report has been added to ResearchAndMarkets.com's offering.

Accounting for an estimated share of 42.5% equating to US$1.7 billion in 2019, Apoptosis Assay Kits lead the demand for Apoptosis Assays globally, which is expected to maintain a 2019-2026 CAGR of 9.9% and reach a projected US$3.6 billion by 2026. The overall Apoptosis Assays market, globally, expected to touch US$4.5 billion in 2020.

Drug Discovery & Development is the principal application for Apoptosis Assays, which, however, is anticipated to maintain the slowest CAGR over the analysis period. In contrast, the market for these assays in Stem Cell Research is poised to grow at a rapid pace, owing to the increasing number of stem cell research activities and an increasing investment in the same.

Further growth in the market can be expected from government initiatives aimed at increasing the number of cell-based research activities, all of which point to a market for Apoptosis Assays that is flourishing.

While COVID-19 may not have an adverse impact on the market for Apoptosis Assays as anticipated, there is a definite slowdown in demand, which has to be addressed with requisite measures.

Research Findings & Coverage

Key Topics Covered:

PART A: GLOBAL MARKET PERSPECTIVE

1. INTRODUCTION 1.1 Product Outline 1.1.1 An Overview of Apoptosis 1.1.1.1 Phases of Apoptosis 1.1.1.1.1 Initiation Phase 1.1.1.1.2 Execution Phase 1.1.1.1.3 Elimination Phase 1.1.1.2 Morphological Changes During Apoptosis 1.1.1.3 Participants in Apoptosis 1.1.1.3.1 Caspases (Cysteine Protease) 1.1.1.3.2 Bcl-2 Family Proteins 1.1.1.3.3 P53 (Tumor Suppressor Protein) 1.1.1.4 Mechanism of Apoptosis 1.1.1.4.1 Extrinsic Pathway of Apoptosis 1.1.1.4.2 Intrinsic Pathway of Apoptosis 1.1.1.4.3 Perforin/Granzyme Pathway 1.1.1.4.4 Common Pathway/Execution Pathway 1.1.1.5 Induction of Apoptosis through Reactive Oxygen Species (ROS) 1.1.1.6 Regulation of Apoptosis 1.1.2 Apoptosis Detection Technologies 1.1.2.1 Flow Cytometry 1.1.2.1.1 The Principle of Flow Cytometry 1.1.2.2 Cell Imaging & Analysis System 1.1.2.3 Spectrophotometry 1.1.2.4 Other Detection Technologies 1.1.3 Apoptosis Product Types 1.1.3.1 Assay Kits 1.1.3.1.1 Annexin V Assay 1.1.3.1.2 DNA Condensation/Fragmentation (TUNEL assays) 1.1.3.1.3 Active Caspase Detection 1.1.3.1.4 Mitochondrial Membrane Potential-Dependent Dyes 1.1.3.1.5 Cytochrome C Release 1.1.3.2 Reagents 1.1.3.2.1 Classic DNA Dyes 1.1.3.2.2 Amine Dyes 1.1.3.2.3 Vital Dyes 1.1.3.3 Instruments 1.1.3.4 Microplates 1.1.4 Apoptosis Applications 1.1.4.1 Drug Discovery & Development 1.1.4.2 Clinical & Diagnostic Applications 1.1.4.3 Basic Research 1.1.4.4 Stem Cell Research 1.1.5 End-Use Markets for Apoptosis Products

2. KEY MARKET TRENDS 2.1 Detection of Early Apoptosis Being Enabled by Flow Cytometry and FTIR Spectroscopy 2.2 Targeted Apoptosis Initiators Developed for Treating Cancer 2.3 TRAIL-Induced Apoptosis in Glioma Using Lestaurtinib More Effective than CHOP-dependent DR5 induction 2.4 Kinetic Imaging and Fluorescent Probes Enable Live Cell Imaging of Apoptosis and Necrosis 2.5 Combination of Live Cell Imaging with Cellular Impedance Allows Real-Time Monitoring of Apoptotic Cell Death 2.6 Assessment of Apoptosis Facilitated by Real-Time, Bioluminescent Annexin V Assay 2.7 Morphological and Biochemical Characteristics of Apoptosis Imaged Using Optimized Optogenetic Actuators 2.8 Detection of Apoptosis Using Automated Algorithms Gains Traction 2.9 Advancements in Understanding Inhibitor of Apoptosis Proteins 2.10 Hydrazones Demonstrate Importance in Apoptosis 2.11 Rapid Detection and Real-Time Tracking of Apoptosis in Live Animals Enabled by Engineered Sensor Zebrafish

3. KEY GLOBAL PLAYERS

4. KEY BUSINESS AND PRODUCT TRENDS

5. GLOBAL MARKET OVERVIEW 5.1 Global Apoptosis Assays Market Overview by Product Type 5.1.1 Apoptosis Product Types Market Overview by Global Region 5.1.1.1 Assay Kits 5.1.1.2 Reagents 5.1.1.3 Instruments 5.1.1.4 Microplates 5.2 Global Apoptosis Assays Market Overview by Detection Technology 5.2.1 Apoptosis Detection Technologies Market Overview by Global Region 5.2.1.1 Flow Cytometry 5.2.1.2 Cell Imaging & Analysis System 5.2.1.3 Spectrophotometry 5.2.1.4 Other Detection Technologies 5.3 Global Apoptosis Assays Market Overview by Application 5.3.1 Apoptosis Applications Market Overview by Global Region 5.3.1.1 Drug Discovery & Development 5.3.1.2 Clinical & Diagnostic Applications 5.3.1.3 Basic Research 5.3.1.4 Stem Cell Research 5.4 Global Apoptosis Assays Market Overview by End-Use Market 5.4.1 Apoptosis Assays End-Use Market Overview by Global Region 5.4.1.1 Pharmaceutical & Biotechnology Companies 5.4.1.2 Hospital & Diagnostic Laboratories 5.4.1.3 Academic & Research Institutes

PART B: REGIONAL MARKET PERSPECTIVE

REGIONAL MARKET OVERVIEW

6. NORTH AMERICA

Major Market Players

7. EUROPE

Major Market Players

8. ASIA-PACIFIC

Major Market Players

9. REST OF WORLD

PART C: GUIDE TO THE INDUSTRY 1. North America 2. Europe 3. Asia-Pacific

PART D: ANNEXURE 1. Research Methodology 2. Feedback

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Global Apoptosis Assays Market Report 2020: Apoptosis Assay Kits Lead the Demand with 42.5% Share - Forecast to 2026 - GlobeNewswire

Global Hematopoietic Stem Cells Transplantation Market 2020 Industry Scenario, Strategies, Growth Factors and Forecast to 2025 KSU | The Sentinel…

MarketQuest.biz offers a detailed research study on Global Hematopoietic Stem Cells Transplantation Market 2020 by Company, Type and Application, Forecast to 2025 which is a professional detailing of the important elements that drive the market growth rate and revenue statistics. The report exhibits comprehensive information that is a valuable source of insightful data for business strategists during the decade 2015-2025. The report focuses on the possible requirements of the clients and assisting them to make the right decision about their business investment plans and strategies. It analyzes key segments and their sub-segments, revenue and demand & supply data. The report demonstrates many comprehensive factors including the globla Hematopoietic Stem Cells Transplantation market share, supply chain, trends, revenue graph, market size, and application spectrum.

Specialized Information And Analysis of The Market:

The report provides an accurate competitive analysis of the market that highlights the expansion tactics adopted by key players of the industry. It explains the scope of global Hematopoietic Stem Cells Transplantation market size, industry growth opportunities and challenges, current market trends, potential players, and expected performance of the market in regions for the forecast period from 2020 to 2025. The report includes the dynamics of the market, future business impact, competition landscape of the companies, and the flow of the global supply and consumption. Major companies in the market are covered with information about their sales data, upcoming innovations and development, revenue margins, investments, business models, strategies, and business estimations.

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NOTE: Our analysts monitoring the situation across the globe explains that the market will generate remunerative prospects for producers post COVID-19 crisis. The report aims to provide an additional illustration of the latest scenario, economic slowdown, and COVID-19 impact on the overall industry.

Topmost leading manufacturer covered in this report: CBR Systems, Lonza Group, Thermo Fisher Scientific, China Cord Blood Corporation, Merck Millipore, ViaCord, ThermoGenesis, Vcanbio, STEMCELL Technologies, CellGenix Technologie Transfer

Market segment considering production, revenue (value), price trend by type: Stem Cells Storage, Stem Cells Consumables

The market segment by consumption growth rate and market share by application: Leukemia, Lymphoproliferative Disorders, Solid Tumors, Non-Malignant Disorders

Identify the opportunities in the market by region: North America (United States, Canada and Mexico), Europe (Germany, France, United Kingdom, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina), Middle East & Africa (Saudi Arabia, UAE, Egypt and South Africa)

The study includes substantial portions, such as type and end-user, and a number of segments that evaluate the outlook of the global Hematopoietic Stem Cells Transplantation market. During the speculation time frame, the report offers data relevant to market appreciation. The application area also offers details within the projected time period according to volume and usage. This segments understanding guides readers to interpret the value of variables that form the growth of the industry.

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Mustang Bio Provides Updates on its Lentiviral Gene Therapies for the Treatment of X-linked Severe Combined Immunodeficiency (XSCID) – GlobeNewswire

February 02, 2021 07:30 ET | Source: Mustang Bio, Inc.

FDA removes clinical hold for pivotal Phase 2 MB-107 clinical trial

Company plans to enroll first patient in MB-107 pivotal trial in the second quarter of 2021

Clinical outcomes in investigator-IND XSCID trials continue to show compelling efficacy

WORCESTER, Mass., Feb. 02, 2021 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today provided updates on MB-107 and MB-207, its lentiviral gene therapies for the treatment of X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease. On January 28, 2021, the U.S. Food and Drug Administration (FDA) removed the clinical hold on the MB-107 pivotal Phase 2 clinical trial Investigational New Drug (IND) application after reviewing a comprehensive CMC package that was submitted by Mustang in late December 2020. Mustang will proceed with its plans to initiate the pivotal Phase 2 trial in newly diagnosed XSCID patients.

The same lentiviral vector used in MB-107 is currently being assessed in a Phase 1/2 clinical trial for XSCID in newly diagnosed infants under the age of two at St. Jude Childrens Research Hospital (St. Jude), UCSF Benioff Childrens Hospital in San Francisco and Seattle Childrens Hospital. Additionally, it is being assessed in a Phase 1/2 clinical trial at the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, for XSCID patients who have been previously treated with hematopoietic stem cell transplantation (HSCT) and for whom re-treatment is indicated.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, We are pleased to have resolved the clinical hold on the MB-107 IND with the FDA, enabling us to move forward with initiating the pivotal Phase 2 clinical trial. The clinical outcomes observed in XSCID patients in the ongoing Phase 1/2 clinical trials continue to be encouraging. It is especially gratifying to see the consistent safety and efficacy of our lentiviral vector over the course of more than eight years since the first patient was treated at NIAID in 2012. In 2021, we look forward to the anticipated initiation of our pivotal MB-107 and MB-207 clinical trials as we work to bring potential new treatment options for this devastating rare disease to patients and their families.

MB-107 UpdateData from the Phase 1/2 clinical trial led by St. Jude that were presented at the 61st American Society of Hematology (ASH) Annual Meeting in December 2019 included 11 newly diagnosed XSCID patients who had been treated with a median follow-up at data cut-off of 23.6 months (range 1.5 to 33.9 months).No serious adverse events related to treatment were reported other than hematologic ones related to low-dose busulfan conditioning. Nine patients, with a follow up of greater than 3 months, achieved normal-for-age T-cell and natural killer (NK)-cell numbers within 3-4 months post treatment with MB-107. Five patients were off intravenous immunoglobulin (IVIG) therapy, of whom 3 responded to vaccines.

To date, all 11 patients have continued to do well, and 5 additional patients were enrolled at the time of the most recent analysis in early September 2020.At that time, follow-up for these 16 patients ranged from 3 months to 47 months. Similar to previous reports, the therapy continued to be well tolerated in all patients, and stable vector marking was noted in all lineages, with successful engraftment of genetically-modified T-, B-, & NK-cells.All patients cleared pre-existing infections, no new severe infections were noted, and all patients were outpatients.Finally, there was no evidence of malignant transformation at a median follow up of 2 years. Enrollment will continue at all three clinical sites until Mustang initiates its multicenter pivotal Phase 2 trial of MB-107.

In September 2020, The Journal of Allergy and Clinical Immunology: In Practice published a case study of one patient with XSCID and disseminated Bacille Calmette-Gurin (BCG) infection, who was enrolled in the clinical trial at St. Jude. After 2.5 years of treatment, the patient has remained clinically well with a stable, functional immune system and protective vaccine titers, despite the complication of the disseminated BCG infection.

MB-207 UpdateThe ongoing Phase 1/2 clinical trial being conducted by the NIH is treating older XSCID patients, all of whom had previously received haplo-identical HSCT as infants and were subsequently noted to be experiencing declining immune function with symptomatic infections. At the time of the most recent NIH data presentation at ASH in 2019, 8 patients had been treated without transduction enhancers (referred to as Cohort A) and had been followed for 3 to 7 years. Seven of these 8 patients experienced gradual clinical benefit in terms of clearance of chronic norovirus and associated improved abdominal complaints, malabsorption, growth and IgG production. One of these 7 patients died 27 months after gene therapy of a pulmonary bleed related to chronic bronchiectasis that predated the therapy and was deemed to be unrelated to therapy.

In an attempt to address the relatively slow resolution of chronic norovirus observed in most of these 7 patients and the delayed immune cell recovery and persistent clinical disease in the eighth patient, transduction enhancers were introduced in the cell processing for the subsequent 6 patients (referred to as Cohort B), which included retreatment of the eighth patient in Cohort A who had delayed immune recovery and persistent clinical disease. This enhanced transduction procedure achieved much greater transduction efficiencies than were observed in Cohort A, with greater than 10-fold less vector, and resulted in faster immune reconstitution and more significant clinical benefit by 3 months. As a result, Mustang has licensed Sirion Biotechs Lentiboost and will include transduction enhancement in its pivotal Phase 2 clinical trial for MB-207 in this patient population.

To date, of the 6 Cohort A patients who were alive at the time of the 2019 NIH data readout and who did not undergo repeat therapy, 3 patients have been able to discontinue chronic intravenous immunoglobulin (IVIG) and have experienced sustained restoration of humoral responses to immunization.The remaining 3 patients have had reduced IVIG requirements.All chronic norovirus infections have resolved, and the quality of life of all patients has improved significantly.

The original 6 patients in Cohort B also continue to do well, with longest follow-up now 22 months.Two additional patients have been successfully treated with transduction enhancers, for a total of 8 patients in Cohort B. As was the case in Cohort A, no serious adverse events related to treatment were reported other than hematologic related to low-dose busulfan conditioning, and there was no evidence of malignant transformation. Further enrollment at NIH is now limited pending Mustangs initiation of its pivotal multicenter Phase 2 clinical trial, and the company expects to submit an Investigational New Drug (IND) application for this trial in the second quarter of 2021.

About X-linked Severe Combined Immunodeficiency (XSCID) X-linked severe combined immunodeficiency is a rare genetic disorder that occurs in approximately 1 per 225,000 births. It is characterized by the absence or lack of function of key immune cells, resulting in a severely compromised immune system and death by 1 year of age if untreated. Patients with XSCID have no T-cells or natural killer (NK)-cells. Although their B-cells are normal in number, they are not functional. As a result, XSCID patients are usually affected by severe bacterial, viral or fungal infections early in life and often present with interstitial lung disease, chronic diarrhea and failure to thrive. Among patients who receive HSCT, many are unable to establish adequate T-cell immunity or lose T-cell immunity over time. Further, approximately two-thirds of patients who receive HSCT lack sufficient B-cell immunity and need lifelong immunoglobulin replacement therapy.

The specific genetic disorder that causes XSCID is a mutation in the gene coding for the common gamma chain (c), a protein that is shared by the receptors for at least six interleukins. These interleukins and their receptors are critical for the development and differentiation of immune cells. The gene coding for c is known as IL-2 receptor gamma, or IL2RG. Because IL2RG is located on the X-chromosome, XSCID is inherited in an X-linked recessive pattern, resulting in almost all patients being male.

About Mustang Bio Mustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR-T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission (SEC). Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.

Company Contacts: Jaclyn Jaffe and William Begien Mustang Bio, Inc. (781) 652-4500 ir@mustangbio.com

Investor Relations Contact: Daniel Ferry LifeSci Advisors, LLC (617) 430-7576 daniel@lifesciadvisors.com

Media Relations Contact: Tony Plohoros 6 Degrees (908) 591-2839 tplohoros@6degreespr.com

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Mustang Bio Provides Updates on its Lentiviral Gene Therapies for the Treatment of X-linked Severe Combined Immunodeficiency (XSCID) - GlobeNewswire

Medexus Pharmaceuticals and medac GmbH enter into a License Agreement for First-in-Class Conditioning Agent for Hematopoietic Stem Cell…

February 02, 2021 14:21 ET | Source: Medexus Pharmaceuticals Inc

Orphan Designated Drug with August 2021 PDUFA date

Management to host conference call at 10:00 AM Eastern Time on February 3, 2021 and

Key Opinion Leader webinar to be held at 2:00 PM Eastern Time on February 5, 2021

TORONTO and CHICAGO and MONTREAL and WEDEL, Germany, Feb. 02, 2021 (GLOBE NEWSWIRE) -- Medexus Pharmaceuticals Inc. (Medexus) (TSXV: MDP) (OTCQX: MEDXF)(Frankfurt: P731) is pleased to announce that it and its wholly-owned United States-based subsidiary, Medexus Pharma, Inc. (Medexus Pharma and together with Medexus, the Company) entered into a Commercialization and Supply Agreement with medac Gesellschaft fr klinische Spezialprparate m.b.H. (medac), pursuant to which medac has granted Medexus Pharma an exclusive license to commercialize treosulfan, a bifunctional alkylating agent, in the United States (the License Agreement).

Treosulfan is an innovative, orphan-designated agent developed for use as part of a conditioning treatment for patients undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). If approved by the U.S. Food and Drug Administration (FDA), the Company expects that a treosulfan-based regimen will be the first in a new conditioning treatment class, Reduced Toxicity Conditioning, resulting in a unique combination of improved survival outcomes compared to reduced-intensity regimens and decreased toxicity compared to standard myeloablative regimens. A Prescription Drug User Free Act (PDUFA) date to review the New Drug Application (NDA) in respect of treosulfan by the FDA has been scheduled for August 2021.

The Company intends to leverage its strong, existing commercial infrastructure in the United States to address the underserved allo-HSCT market through its commercialization of treosulfan. medac conducted a phase III randomized study (the Phase III Study) comparing the results of treosulfan-based therapy with busulfan-based reduced intensity conditioning in advance of allo-HSCT for adult patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) who were considered ineligible for standard myeloablative conditioning regimens. The planned confirmatory interim analysis of the Phase III Study demonstrated that non-inferiority was achieved in the treosulfan group compared to the busulfan group in two-year event-free survival with 64.0% (95% CI 56.070.9) in the treosulfan group and 50.4% (95% CI 42.857.5) in the busulfan group (HR 0.65 [95% CI 0.470.90]); p=0.0000164 (adjusted p-value for testing non-inferiority of treosulfan compared to busulfan).1Despite lacking indications for use in patients with AML or MDS, busulfan is the current market leading alkylating agent for allo-HSCT. Prior to genericization in 2016, busulfan reached peak annual sales of U.S. $126 million in the United States.2

The NDA in respect of treosulfan was filed by medac in August 2020 and seeks FDA approval for use of treosulfan as part of a conditioning regimen for allo-HSCT for adults with AML and MDS. The NDA is supported by the completed follow-up results from the Phase III Study covering all 570 randomized patients including superiority testing, which may result in even stronger claims than non-inferiority in a final label for treosulfan, if approved by the FDA.3

On April 8, 2015, the FDA granted medac Orphan Drug Designation for treosulfan as a conditioning treatment prior to allo-HSCT in malignant and non-malignant disease in adults and pediatric patients. In accordance with the Orphan Drug Act, seven years of exclusivity for this indication is expected upon FDA approval. According to the most recent data from the Center for International Blood & Marrow Transplant Research (CIBMTR), there were an estimated 9,028 allo-HSCT procedures in the United States in 2018, growing at about 3% year over year. Another 14,006 autologous-HSCT (auto-HSCT) procedures, which also routinely feature conditioning regimens that include alkylating agents, were completed that same year.4

Treosulfan was granted marketing authorization in combination with fludarabine by the European Commission in June 2019, indicated for use in combination with fludarabine as part of a conditioning treatment prior to allo-HSCT in (i) adult patients with both malignant and non-malignant diseases, and (ii) pediatric patients older than one month with malignant diseases. In Canada, Medexus is currently distributing treosulfan via the Special Access Program.

H. Joachim Deeg, MD, Professor of Medical Oncology at the University of Washington School of Medicine, Professor of Clinical Research at the Fred Hutchinson Cancer Research Center, and Physician at the Seattle Cancer Care Alliance, commented, Treosulfan has proven to be a potent drug for transplant conditioning in several phase II trials for both malignant and non-malignant disorders, conducted at our own Center and several other institutions, earning the label high intensity, low toxicity. Of note, clinically meaningful improvements in favor of the treosulfan group for event-free survival, overall survival, and transplant-related mortality were seen in medacs study, and a treosulfan-based regimen promises to be the preferred standard conditioning therapy for this study population, which represents the growing population of older and comorbid patients with AML or MDS, and beyond.

Mary Horowitz, MD,MS,Professor of Hematologic Research at the Medical College of Wisconsin andScientific Director for the CIBMTR, commented, It is incredibly important for clinicians to have more options for patients undergoing allo-HSCT. I am very happy to see that medac and Medexus have teamed up to work towards bringing treosulfan to the U.S. market. The data on treosulfan thus far is highly encouraging, suggesting it could fill an important gap for higher risk patients who cannot tolerate the typical toxicity profile of currently available high-intensity conditioning regimens.

The License Agreement

Upon entering into the License Agreement, Medexus Pharma paid medac a non-refundable upfront payment of U.S. $5 million. Under the terms of the License Agreement, Medexus Pharma must also pay medac (i) up to an aggregate of U.S. $55 million in non-refundable regulatory milestone payments, contingent upon the achievement of certain regulatory events in connection with the FDAs review process (the Regulatory Milestone Payments), and (ii) up to an aggregate of U.S. $40 million in non-refundable sales milestone payments, contingent upon Medexus Pharmas achievement of certain net sales goals (the Sales Milestone Payments, and together with the Regulatory Milestone Payments, the Milestone Payments). In addition, Medexus Pharma will pay medac a low single-digit royalty on its net sales of treosulfan in the United States.

The License Agreement is effective as of today and continues until the 10th anniversary of FDA approval of the initial NDA, unless earlier terminated by either the Company or medac in accordance with their respective rights under the License Agreement. Going forward, medac will continue with primary responsibility for development and regulatory matters in respect of treosulfan, including preparing and obtaining FDA approval of the initial NDA. After such FDA approval, Medexus Pharma will maintain regulatory approval of treosulfan in the United States and leverage its significant commercial experience in leading the commercialization effort for treosulfan. medac will also be responsible for the manufacturing and supply of treosulfan to Medexus Pharma in accordance with the terms of the License Agreement. The Company and medac will work together to finalize the preparations for commercialization of treosulfan ahead of the PDUFA date and expect to launch shortly after FDA approval.

Ken dEntremont, Chief Executive Officer of Medexus, stated, We are pleased to execute another transformative transaction with medac. In 2018, when we acquired medacs U.S. affiliate, we anticipated that treosulfan could be a significant advancement in HSCT. This transaction marks another major milestone for Medexus and is indicative of our continued effort to further expand into the U.S. through what we believe will be a highly accretive transaction for the Company. Given the drugs therapeutic profile and the data generated to date, we believe that treosulfan could exceed peak sales of busulfan of U.S. $126 million from use in allo-HSCT alone. This belief is re-enforced by the fact that that busulfan is currently being used off-label for the indications for which treosulfan has Orphan Drug Designation. Importantly, we believe there is a large unmet need as the current standard of care is not suitable for numerous at-risk groups, due to the high toxicity effects. Treosulfan has demonstrated excellent event-free survival and overall survival among such groups and as a result, should be well positioned to become the new standard of care in the U.S., with more than 100 publications supporting the safety and efficacy of treosulfan. We are proud to be working towards providing patients with a new solution that could have a very meaningful impact on their lives.

Jrg Hans, Chief Executive Officer of medac, emphasizes, This licensing deal with Medexus offers us the unique opportunity of providing patients and physicians with our very promising new treatment option in the area of allogeneic hematopoietic stem cell transplantation now also in the United States. The treosulfan-based conditioning regimen stands out for its combination of being highly effective - similar to the potency of the myeloablative procedure - while simultaneously exhibiting significantly reduced toxicity. We at medac are very proud of our first-in-class conditioning agent as it addresses a huge need in the area of conditioning treatments especially with regard to high-risk patients. Therefore, this product fully meets our company goals of improving patients quality of life and supporting healthcare professionals in the best possible way. As a shareholder in Medexus we see the expansion of our relationship as a true win-win.

Medexus and Medexus Pharma were represented by Munsch Hardt Kopf and Harr, P.C. and medac was represented by Baker & McKenzie LLP with respect to the License Agreement.

Conference Call Details

Medexus will host a conference call on February 3, 2021 at 10:00 AM Eastern Time (U.S. and Canada) to discuss the License Agreement and to provide an operational update.

The conference call will be available via telephone by dialing toll free 888-506-0062 for Canadian and U.S. callers or 973-528-0011 for international callers, or on the Medexus Investor Events section of the website: https://www.medexus.com/en_US/investors/news-events.

A webcast replay will be available on Medexus Investor Events section of the website (https://www.medexus.com/en_US/investors/news-events) through May 3, 2021. A telephone replay of the call will be available approximately one hour following the call, through February 10, 2021 and can be accessed by dialing 877-481-4010 for Canadian and U.S. callers or 919-882-2331 for international callers and entering conference ID: 39898

Key Opinion Leader Webinar

Medexus will be hosting a Key Opinion Leader webinar to discuss treosulfan on February 5, 2021 at 2:00 PM Eastern Time (U.S. and Canada), followed by a question-and-answer period. Ken dEntremont, CEO, will be joined by H. Joachim Deeg, MD to discuss the clinical data supporting treosulfan.

To join the webinar, please register here: Treosulfan Key Opinion Leader Webinar. After registering, you will receive a confirmation email containing information about joining the webinar. The webinar will also be live streamed on YouTube for those who are unable to use Zoom: YouTube Live Stream.

Questions may be asked during the webinar or can be emailed ahead of time to info@adcap.ca. A replay will be made available on the Medexus website.

H. Joachim Deeg, MD H. Joachim Deeg, MD, is a Physician at the Seattle Cancer Care Alliance, a Professor of Medical Oncology at the University of Washington School of Medicine, and a Professor of Clinical Research at the Fred Hutchinson Cancer Research Center. He currently holds the Miklos Kohary and Natalia Zimonyi Kohary Endowed Chair for Cancer Research. He is an expert in bone marrow transplantation, myelodysplastic syndromes, and myeloproliferative neoplasms. Dr. Deeg is a board-certified oncologist with more than 40 years of experience treating blood-disorders. He has a medical degree from the University of Bonn School of Medicine. Dr. Deeg completed his residency at the University of Rochester, NY and did a fellowship in Hematology/Oncology at the Fred Hutchinson Cancer Research Center/ University of Washington, Seattle.

Mary Horowitz, MDDr. Horowitz is the Robert A. Uihlein Professor of Hematologic Research and Deputy Cancer Center at the Medical College of Wisconsin in Milwaukee. She is also Scientific Director Emeritus of the Center for International Blood and Marrow Transplant Research (CIBMTR). The CIBMTR is a research collaboration between the National Marrow Donor Program(NMDP)/Be The Matchand the Medical College of Wisconsin. The CIBMTR collaborates with the global scientific community to advance hematopoietic cell transplantation and cellular therapy worldwide to increase survival and enrich quality of life for patients. The CIBMTR facilitates critical observational and interventional research through scientific and statistical expertise, a large network of transplant centers, and a unique and extensive clinical outcomes database. Dr. Horowitz also leads the Coordinating Center of the U.S. Blood and Marrow Clinical Trials Network, a multicenter group funded by the National Institutes of Health to test new therapies to improve the safety and effectiveness of transplantation. She has co- authored more than 400 publications addressing diverse issues in clinical BMT.

1Beelen, DW et al., Final Results of a Prospective Randomized Multicenter Phase III Trial Comparing Treosulfan / Fludarabine to Reduced Intensity Conditioning with Busulfan / Fludarabine Prior to Allogeneic Hematopoietic Stem Cell Transplantation in Elderly or Comorbid Patients with Acute Myeloid Leukemia or Myelodysplastic Syndrome. Blood. 2017;130 (Suppl 1):521

2Symphony Health PHAST Data 2020

3Beelen, DW et al. Final Evaluation of a Clinical Phase III Trial Comparing Treosulfan to Busulfan-Based Conditioning Therapy Prior to Allogeneic Hematopoietic Stem Cell Transplantation of Adult Acute Myeloid Leukemia and Myelodysplastic Syndrome Patients Ineligible to Standard Myeloablative Regimens. Biol Blood Marrow Transplant 25 (2019) S1-S6, p. 53, Abstract No. 04.

4D'Souza, A, Fretham C, Lee SJ, et al. Current Use of and Trends in Hematopoietic Cell Transplantation in the United States. Biol Blood Marrow Transplant. 2020 May 11: S1083-8791(20)30225-1

About medac GmbH

medac GmbH is a privately held, global pharmaceutical company with a growing pharmaceutical and diagnostics business. Since its foundation in Germany in 1970, medac has been specializing in the treatment of diseases within the indication areas oncology, hematology, urology and autoimmune disorders. medac is committed to the refinement of existing and the development of new therapeutic products always with the focus on improving patients quality of life. medac has become known for developing innovative products also in less common indications. This dedication has resulted in a comprehensive portfolio of pharmaceutical products that help make a difference in the lives of patients. medac continually invests in its product development and manufacturing as well as logistic capacities to meet both patients needs and the demands of healthcare professionals.

About Medexus Pharmaceuticals Inc.

Medexus is a leading innovative and rare disease company with a strong North American commercial platform. From a foundation of proven best in class products we are building a highly differentiated company with a portfolio of innovative and high value orphan and rare disease products that will underpin our growth for the next decade. The Companys vision is to provide the best healthcare products to healthcare professionals and patients, through our core values of Quality, Innovation, Customer Service and Teamwork. Medexus Pharmaceuticals is focused on the therapeutic areas of auto-immune disease, hematology, and allergy. The Companys leading products are: Rasuvo and Metoject, a unique formulation of methotrexate (auto-pen and pre-filled syringe) designed to treat rheumatoid arthritis and other auto-immune diseases; IXINITY, an intravenous recombinant factor IX therapeutic for use in patients 12 years of age or older with Hemophilia B a hereditary bleeding disorder characterized by a deficiency of clotting factor IX in the blood, which is necessary to control bleeding; and Rupall, an innovative prescription allergy medication with a unique mode of action.

For more information, please contact:

Ken dEntremont, Chief Executive Officer Medexus Pharmaceuticals Inc. Tel.: 905-676-0003 E-mail:ken.dentremont@medexus.com

Roland Boivin, Chief Financial Officer Medexus Pharmaceuticals Inc. Tel.: 514-334-8765 E-mail:roland.boivin@medexus.com

Investor Relations (U.S.): Crescendo Communications, LLC Tel: +1-212-671-1020 Email:mdp@crescendo-ir.com

Investor Relations (Canada): Tina Byers Investor Relations Tel: 905-330-3275 E-mail:tina@adcap.ca

Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

READER ADVISORIES

Forward Looking Statements

Certain statements made in this press release contain forward-looking information within the meaning of applicable securities laws (forward-looking statements). The words anticipates, believes, expects, should, will, and similar expressions are often intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Specific forward-looking statements contained in this press release include, but are not limited to, statements with respect to the August 2021 PDUFA date, expectations for treosulfan to be the first in a new conditioning treatment class, the Companys intention to leverage its commercial infrastructure in the United States to commercialize treosulfan, the expectation for exclusivity for treosulfan upon FDA approval, the results of the Phase III Study and the possibility of non-inferiority or stronger claims in the final label for treosulfan, the expected launch of treosulfan, the accretive nature of the transaction, the potential for treosulfan to exceed peak sales of busulfan and the anticipated growth in sales of, the market for and distribution of, treosulfan. These statements are based on factors or assumptions that were applied in drawing a conclusion or making a forecast or projection, including assumptions based on historical trends, current conditions and expected future developments. Since forward-looking statements relate to future events and conditions, by their very nature they require making assumptions and involve inherent risks and uncertainties. The Company cautions that although it is believed that the assumptions are reasonable in the circumstances, these risks and uncertainties give rise to the possibility that actual results may differ materially from the expectations set out in the forward-looking statements. Material risk factors include those set out in the Companys materials filed with the Canadian securities regulatory authorities from time to time, including the Companys most recent annual information form and managements discussion and analysis; future capital requirements; intellectual property protection and infringement risks; competition (including potential for generic competition); reliance on key management personnel; the Companys ability to implement its business plan; the Companys ability to leverage its United States and Canadian infrastructure to promote additional growth, including with respect to the infrastructure of Medexus Pharma, and the potential benefits the Company expects to derive therefrom; regulatory approval by the FDA; litigation risk; and government regulation. Given these risks, undue reliance should not be placed on these forward-looking statements, which apply only as of the date hereof. Other than as specifically required by law, the Company undertakes no obligation to update any forward-looking statements to reflect new information, subsequent or otherwise.

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Medexus Pharmaceuticals and medac GmbH enter into a License Agreement for First-in-Class Conditioning Agent for Hematopoietic Stem Cell...

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Adipose Tissue-Derived Stem Cells (ADSCS) Market is Projected to Grow Massively in Near Future The Courier - The Courier