Monthly Archives: December 2020

ROCKVILLE, Md., Dec. 22, 2020 /PRNewswire/ --Synthetic Biologics, Inc.(NYSE American: SYN),a diversified clinical-stage company leveraging the microbiome to develop therapeutics designed to prevent and treat gastrointestinal (GI) diseases in areas of high unmet need,today announced it has received approval from the Institutional Review Board (IRB) at Washington University School of Medicine in St. Louis (Washington University), to commence the Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic hematopoietic cell transplant (HCT) recipients. As a result, the Company expects to commence patient enrollment for the Phase 1b/2a clinical trial in the first quarter of 2021.

The IRB-approved Phase 1b/2a clinical program will be conducted by Washington University and comprise a randomized, double-blinded, placebo-controlled clinical trial of oral SYN-004in up to 36 evaluable adult allogeneic HCT recipients. The goal of this study is to evaluate the safety, tolerability and potential absorption into the systemic circulation (if any) of 150 mg oral SYN-004 administered to allogeneic HCT recipients who receive an IV beta-lactam antibiotic to treat fever. Study participants will be enrolled into three sequential cohorts, with each receiving a different study-assigned IV beta-lactam antibiotic. Eight participants in each cohort will receive SYN-004 and four will receive placebo.

Safety and pharmacokinetic data for each cohort will be reviewed by an independent Data and Safety Monitoring Committee that will make a recommendation on whether to proceed to the next IV beta-lactam antibiotic. The study will also evaluate potential protective effects of SYN-004 on the gut microbiome as well as generate preliminary information on potential therapeutic benefits and patient outcomes of SYN-004 in allogeneic HCT recipients. Enrollment is expected to begin during the first quarter of 2021, pandemic conditions permitting.

"Approval of the Phase 1b/2a clinical protocol by Washington University's IRB is an important step in pursuing a potentially more cost-effective development strategy for SYN-004, targeting a highly specialized patient population," stated Steven A. Shallcross, Chief Executive and Financial Officer of Synthetic Biologics. "Broad spectrum IV beta-lactam antibiotics used to treat infection following conditioning chemotherapy for allogeneic HCT patients is a necessary and oftentimes lifesaving intervention. However, this may have the unintended consequence of causing dysbiosis of the microbiome which may lead to infection by opportunistic pathogens such as C. difficile (CDI) and vancomycin-resistant enterococci (VRE). In previously completed clinical trials, SYN-004 demonstrated a significant reduction in the incidence of CDI and VRE in treated patients versus placebo and has the potential to significantly improve outcomes for allogenic HCT recipients. We expect to commence enrollment of patients in the first quarter of 2021 and look forward to providing further updates as we advance this exciting program."

The Washington University IRB is a committee established to review and approve research involving human subjects. The purpose of the IRB is to ensure that all human subject research is conducted in accordance with all federal, institutional, and ethical guidelines. The primary goal of an IRB is to safeguard the rights, safety and welfare of participants in research studies.

About SYN-004 (ribaxamase)

SYN-004 (ribaxamase) is an oral prophylactic therapy designed to degrade certain IV beta-lactam antibiotics within the GI tract and maintain the natural balance of the gut microbiome for the prevention of Clostridioides difficile infection (CDI), overgrowth of pathogenic organisms, the emergence of antimicrobial resistance (AMR) and acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients. Allogeneic HCT recipients routinely receive long courses of IV beta-lactam antibiotics to treat infection. Antibiotic-mediated damage of the gut microbiome in allogeneic HCT recipients has been strongly associated with adverse outcomes including CDI, vancomycin-resistant enterococci (VRE) colonization and potentially fatal bacteremia and aGVHD. A previously completed placebo-controlled Phase 2b clinical trial of 412 patients demonstrated SYN-004 protected the gut microbiome from antibiotic-mediated dysbiosis. Patients receiving SYN-004 also demonstrated significantly better maintenance and recovery of the gut microbiome as well as lower incidences of new colonization by opportunistic and potentially pathogenic microorganisms such as VRE.

About Synthetic Biologics, Inc.

Synthetic Biologics, Inc. (NYSE American: SYN) is a diversified clinical-stage company leveraging the microbiome to develop therapeutics designed to prevent and treat gastrointestinal (GI) diseases in areas of high unmet need. The Company's lead candidates are: (1) SYN-004 (ribaxamase) which is designed to degrade certain commonly used intravenous (IV) beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent (a) microbiome damage, (b) Clostridioides difficile infection (CDI), (c) overgrowth of pathogenic organisms, (d) the emergence of antimicrobial resistance (AMR) and (e) acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients, and (2) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases. For more information, please visit Synthetic Biologics' website at http://www.syntheticbiologics.com.

This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases forward-looking statements can be identified by terminology such as "may," "should," "potential," "continue," "expects," "anticipates," "intends," "plans," "believes," "estimates," and similar expressions, and includes statements regarding the Company commencing patient enrollment for the Phase 1b/2a clinical trial in the first quarter of 2021, the potential of SYN-004 to significantly improve outcomes for allogenic HCT recipientand the intended benefits to be derived from SYN-004 and SYN-020. These forward-looking statements are based on management's expectations and assumptions as of the date of this press release and are subject to a number of risks and uncertainties, many of which are difficult to predict that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, Synthetic Biologics' ability to commence patient enrollment for thePhase 1b/2a clinical trial in the first quarter of 2021, the ability to continue to comply with continued listing requirements of the NYSE American, the ability of its product candidates to demonstrate safety and effectiveness, as well as results that are consistent with prior results, Synthetic Biologics' clinical trials continuing and/or beginning enrollment as expected, a failure to receive the necessary regulatory approvals for commencement of clinical trials and commercialization of Synthetic Biologics' therapeutics, including approval of proposed trial designs, a failure of Synthetic Biologics' clinical trials, and those conducted by investigators, for SYN-004 and SYN-020 to be commenced or completed on time or to achieve desired results and benefits, a failure of Synthetic Biologics' clinical trials to continue enrollment as expected or receive anticipated funding, a failure of Synthetic Biologics to successfully develop, market or sell its products, Synthetic Biologics' inability to maintain its material licensing agreements, or a failure by Synthetic Biologics or its strategic partners to successfully commercialize products, Synthetic Biologics' ability to achieve acceptance of its product candidates in the marketplace and the successful development, marketing or sale of Synthetic Biologics' products by competitors that render Synthetic Biologics' products obsolete or non-competitive, the continued maintenance and growth of Synthetic Biologics' patent estate, Synthetic Biologics becoming and remaining profitable, Synthetic Biologics' ability to obtain or maintain the capital or grants necessary to fund its research and development activities, a loss of any of Synthetic Biologics' key scientists or management personnel and other factors described in Synthetic Biologics' Form 10-K and 10-K/A for the year ended December 31, 2019 and its other filings with the SEC, including subsequent periodic reports on Forms 10-Q and 8-K. The information in this release is provided only as of the date of this release, and Synthetic Biologics undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.

SOURCE Synthetic Biologics, Inc.

http://www.syntheticbiologics.com

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Synthetic Biologics Announces Washington University School of Medicine in St. Louis IRB Approval of the SYN-004 (ribaxamase) Phase 1b/2a Clinical...

DUBLIN, Dec. 21, 2020 /PRNewswire/ -- The "Global CAR T-Cell Therapy Market: Focus on Product, Mechanism, Application, Target Antigen, Country Data (14 Countries), and Competitive Landscape - Analysis and Forecast, 2020-2030" report has been added to ResearchAndMarkets.com's offering.

The scientific community has been pursuing research for the development of CAR T-cell therapy for over a century. Immunotherapy has emerged as an innovative treatment option over decades, as it harnesses the patient's immune system to attack cancer. The global market for CAR T-cell therapy is predicted to grow at a CAGR of 44.79% over the forecast period 2020-2030. The market is driven by certain factors, which include increasing global geriatric population, increasing number of lymphoma and leukemia cases, rising number of relapsed or refractory cancer cases showing response failure to alternative treatments such as chemotherapy and radiation therapy, focus on research and development of novel immunotherapies, and strong product pipeline of global CAR T-cell therapy.

The market is favored by the development of CAR T-cell therapy for several clinical areas such as diffuse large B-cell lymphoma (DLBCL), acute lymphoblastic leukemia (ALL), multiple myeloma (MM), chronic lymphocytic leukemia (CLL), follicular lymphoma (FL), mantle cell lymphoma (MCL), and other cancers or indications. The increase in the geriatric population and the increasing number of lymphoma and leukemia cases across the globe are expected to translate into significantly higher demand for the CAR T-cell therapy market.

Furthermore, the companies are investing huge amounts in research and development of CAR T-cell therapy either as a monotherapy or as combination therapy. The clinical trial landscape of various hematologic cancers has been on the rise in recent years, and this will fuel the CAR T-cell therapy market in the future.

Within the research report, the market is segmented based on application, target antigen, and region. Each of these segments covers the snapshot of the market over the projected years, the inclination of the market revenue, underlying patterns, and trends by using analytics on the primary and secondary data obtained.

Competitive Landscape

The exponential rise in the application of precision medicine on a global level has created a buzz among companies to invest in the development of novel CAR T-cell therapy. Due to the diverse product portfolio and intense market penetration, Novartis AG. has been a pioneer in this field and has been a significant competitor in this market.

The pharmaceutical leading manufacturer, Kite Pharma, Inc. (a Gilead Company), has launched its CAR T-cell therapies into the market such as Yescarta (axicabtagene ciloleucel), and Tecartus (brexucabtagene autoleucel) to compete with Novartis AG's Kymriah (tisagenlecleucel) therapy market dominance.

Based on region, North America holds the largest share of the CAR T-cell therapy market due to improved healthcare infrastructure, rise in per capita income, early availability of approved therapies, and availability of state-of-the-art research laboratories and institutions in the region. Apart from this, the Asia-Pacific region is anticipated to grow at the fastest CAGR during the forecast period.

Growth Drivers

Market Challenges

Market Opportunities

Key Questions Answered in this Report:

Key Topics Covered:

1 Technology Definition

2 Research Scope

3 Research Methodology

4 Market Overview4.1 Timeline of CAR T-Cell Therapy Development4.2 CAR T-Cell Therapy Market and Growth Potential, 2019-20304.3 CAR T-Cell Therapy and Clinical Trials Landscape4.3.1 Overview4.3.2 Clinical Trials in China4.3.3 Clinical Trials in Rest-of-the-World

5 Global CAR T-Cell Therapy Market, $Million, 2019-20305.1 Commercialized Therapeutics5.1.1 Tisagenlecleucel (Kymriah)5.1.2 Axicabtagene Ciloleucel (Yescarta)5.1.3 Brexucabtagene Autoleucel (Tecartus)5.2 Pipeline Therapeutics5.2.1 BB2121 (Idecaptagene Cicleucel)5.2.2 JCAR017 (Lisocabtagene Maraleucel)5.2.3 KTE-C19 (Axicabtagene Ciloleucel)5.2.4 CTL019 (Tisagenlecleucel)5.2.5 JNJ-682845285.3 Impact of COVID-19 - CAR T-Cell Therapy5.3.1 Impact of COVID-19 on Global CAR T-Cell Therapy Market Growth Rate5.3.2 Clinical Trial Disruptions and Resumptions5.3.3 COVID-19 Impact on Supply Chain of CAR T-Cell Therapy Market5.3.4 Reduced Diagnosis of Cancer5.3.5 Impact on Commercial Operations

6 CAR T-Cell Therapy Patent Landscape

7 Epidemiology of CAR T-Cell Therapy7.1 Overview7.2 CAR T-Cell Therapy Side-Effects7.2.1 Cytokine Release Syndrome (CRS)7.2.2 CAR T-Cell-Related Encephalopathy Syndrome (CRES)7.2.3 Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS)7.3 Management of CAR T-Cell Therapy Side-Effects

8 Industry Insights8.1 Overview8.2 Regulatory Scenario of CAR T-Cell Therapy8.3 Legal Requirements and Frameworks in U.S.8.3.1 Clinical Trial Authorization8.3.2 Marketing Authorization8.3.3 USFDA Guidelines for BLA Submission8.3.4 Post-Authorization Regulations8.4 Legal Requirements and Frameworks in Europe8.4.1 EMA Biologics License Application Process8.4.2 Centralized Procedure8.4.3 Decentralized Procedure8.4.4 Mutual-Recognition Procedure8.4.5 National Procedure8.5 Legal Requirements and Frameworks in Asia-Pacific8.5.1 Regulation of Cell Immunotherapy in China8.5.2 Legal Requirements and Frameworks in Japan8.6 Expedited Regulatory Designations Around the World8.7 Pricing of CAR T-Cell Therapy8.8 Reimbursement of CAR T-Cell Therapy

9 CAR T-Cell Therapy Development and Manufacturing9.1 Overview9.2 Challenges in CAR T-Cell Therapy9.2.1 Therapy Approval9.2.2 Manufacture Quality Control9.2.3 Efficacy and Quality-Adjusted Life-Year (QALY) Assessment9.2.4 Safety Assessment9.2.5 Pricing and Patient Access9.2.6 Reimbursement

10 Market Dynamics10.1 Impact Analysis10.2 Market Drivers10.2.1 Rising Number of Patients with Hematologic Cancers10.2.2 Dramatic Rise in Global CAR T-Cell Trials10.2.3 Landmark Approvals of CAR T-Cell Therapies by USFDA and EMA10.3 Market Restraints10.3.1 High Treatment Cost of CAR T-Cell Therapy10.3.2 Side-Effects of CAR T-Cell Therapy10.4 Market Opportunities10.4.1 Opportunities for Immunotherapy

11 Global CAR T-Cell Therapy Market (by Application)11.1 Overview11.2 Diffuse Large B-Cell Lymphoma (DLBCL)11.3 Acute Lymphoblastic Leukemia (ALL)11.4 Multiple Myeloma (MM)11.5 Chronic Lymphocytic Leukemia (CLL)11.6 Follicular Lymphoma (FL)11.7 Mantle Cell Lymphoma (MCL)11.8 Others

12 Global CAR T-Cell Therapy Market (by Target Antigen)12.1 Overview12.2 CD19/CD2212.3 BCMA (B-Cell Maturation Antigen)12.4 Other Antigens

13 Global CAR T-Cell Therapy Market (by Region)13.1 Introduction13.2 North America13.2.1 U.S.13.2.1.1 U.S. CAR T-Cell Therapy Market (by Application), 2019-203013.2.2 Canada13.2.2.1 Canada CAR T-Cell Therapy Market (by Application), 2019-203013.3 Europe13.3.1 Germany13.3.1.1 Germany CAR T-Cell Therapy Market (by Application), 2019-203013.3.2 U.K.13.3.2.1 U.K. CAR T-Cell Therapy Market (by Application), 2019-203013.3.3 France13.3.3.1 France CAR T-Cell Therapy Market (by Application), 2019-203013.3.4 Italy13.3.4.1 Italy CAR T-Cell Therapy Market (by Application), 2019-203013.3.5 Spain13.3.5.1 Spain CAR T-Cell Therapy Market (by Application), 2019-203013.3.6 Rest-of-Europe13.3.6.1 Rest-of-Europe CAR T-Cell Therapy Market (by Application), 2019-203013.4 Asia-Pacific13.4.1 China13.4.2 Japan13.4.2.1 Japan CAR T-Cell Therapy Market (by Application), 2019-203013.4.3 India13.4.4 South Korea13.4.5 Australia13.4.5.1 Australia CAR T-Cell Therapy Market (by Application), 2019-203013.4.6 Rest-of-APAC13.4.6.1 Rest-of-APAC CAR T-Cell Therapy Market (by Application), 2019-203013.5 Latin America13.5.1 Brazil13.5.2 Mexico13.5.3 Rest-of-Latin America13.6 Rest-of-the-World

14 Competitive Landscape14.1 Key Developments and Strategies14.1.1 Overview14.1.1.1 Regulatory and Legal Developments14.1.1.2 Partnerships, Alliances, and Business Expansions14.1.1.3 M&A Activities14.1.1.4 Funding Activities14.2 Market Share Analysis

15 Company Profiles15.1 Overview15.2 Amgen, Inc.15.2.1 Company Overview15.2.2 Role of Amgen, Inc. in Global CAR T-Cell Therapy Market15.2.3 Financials15.2.4 R&D Expenditure, 2017-201915.2.5 SWOT Analysis15.3 Autolus Therapeutics plc15.4 Bellicum Pharmaceuticals, Inc.15.5 Bluebird Bio Inc15.6 Bristol-Myers Squibb Company15.7 CARsgen Therapeutics, Ltd.15.8 Cartesian Therapeutics, Inc.15.9 Cellectis S.A.15.1 Celyad Oncology SA15.11 Fortress Biotech, Inc15.12 Janssen Pharmaceuticals, Inc.15.13 Kite Pharma, Inc (A Gilead Company)15.14 Legend Biotech Corporation15.15 Novartis AG15.16 Pfizer Inc.

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

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Worldwide CAR T-Cell Therapy Industry to 2030 - Players Include Legend Biotech, Novartis & Pfizer Among Others - PRNewswire

By Sophie Holman-Jones, Third Year, Biomedical Sciences

Researchers at the University of Bristol have successfully manipulated cell-like micro-bots in the form of synthetic protocells to generate nitric oxide (NO) gas in blood vessels of rabbits. Developing micro-bots capable of integration within human cells could give rise to many possibilities with clinical benefits in the near future.

When biomedical engineering was in its infancy, the possibility of the use of artificial models (bots) to regulate disease management would have seemed impossible. This month, thanks to a team from the University of Bristol, we took a step closer to making this a reality. The ground-breaking research was headed by Professor Stephen Mann and Dr Mei Li, from the Department of Chemistry, in collaboration with colleagues from Hunan University and Central South University in China.

The team innovatively managed to develop bots (which they refer to as protocells) by deriving fragments of red-blood cells from sheep blood and manipulating them to assemble themselves around tiny droplets of an enzyme known as glucose oxidase. After assembly, the protocells were injected into rabbits, which is where a series of chemical reactions was observed.

In the presence of glucose, which occurs naturally in the blood, the enzyme glucose oxidase produces the chemical compound hydrogen peroxide. Red blood cells also contain a protein called haemoglobin, which mainly functions to transport oxygen around the body. In this case, haemoglobin recruits the hydrogen peroxide in order to aid the break-down of hydroxyurea (an oral drug commonly prescribed for certain cancers) into NO. The production of NO via this series of reactions is extremely rapid and was shown to increase twenty-one-fold in ten minutes.

NO is naturally produced in the body in order to relax smooth muscle and thus stimulate blood vessel dilation. The profound effect of protocell injection on NO production in sheep caused a 1.2-fold increase in blood vessel diameter compared to an unrelaxed vessel.

Manns team also noted a significant reduction in heart rate and blood pressure. The effect of blood vessel size can be thought of similarly to drinking through a straw; the smaller the diameter of the straw, the harder you need to suck to drink through it. Likewise, the wider the vessel diameter, the less force needs to be generated by the heart to pump the blood around the body.

High blood pressure is associated with a plethora of health complications, including heart attacks, strokes and heart failure. According to the World Health Organisation cardiovascular disease is the single biggest killer globally and costs the NHS 7 billion each year. The reduction in blood pressure seen by Manns team in rabbits means that a similar effect could potentially be replicable in humans. This method could have real therapeutic potential, in terms of preventing health problems associated with high blood pressure and relieving some of the financial burden on our health systems.

Even more exciting are the many other potential applications this pioneering work could have in the wider clinical field, which have not yet been elucidated. For example, it could allow for drugs to circulate in the body for extended periods of time or be administered to specific therapeutic targets that are otherwise difficult to reach via conventional methods. Medical drugs could also be designed to be more compatible with the patient to ensure they are more tolerable by the body.

In short, micro-bots could be an invaluable tool to manage cardiovascular disease and many other related illnesses. Using bots in this way is still in its early stages and should be corroborated by other studies before extensive clinical use. Nevertheless, this is still an exciting prospect and certainly one to watch for in the field of biologics.

Featured Image: Flickr / Mike Goad

Which new promising clinical therapies have you heard about? Can protocells be an answer to many critical illnesses?

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Bristol University's micro-bots could change the face of medicine - Epigram

-- Initiate Phase 1b randomized, placebo-controlled clinical trial of novel chemoprotective agent ALRN-6924 in patients with advanced non-small cell lung cancer (NSCLC) receiving first-line carboplatin doublet chemotherapy in second quarter 2021, advancing ALRN-6924 clinical development into a large cancer indication-- Report initial results from Phase 1b NSCLC clinical trial in fourth quarter 2021-- Report additional results from ongoing Phase 1b proof-of-concept clinical trial of ALRN-6924 in patients with small cell lung cancer receiving topotecan in first quarter 2021

WATERTOWN, Mass., Dec. 21, 2020 (GLOBE NEWSWIRE) --Aileron Therapeutics (NASDAQ:ALRN) today provided a business update and outlined its strategic priorities for 2021, including announcing more details about the design and conduct of a Phase 1b randomized, double-blind, placebo-controlled clinical trial of ALRN-6924 in patients with advanced non-small cell lung cancer (NSCLC) undergoing treatment with first-line carboplatin doublet chemotherapy (with or without immune checkpoint inhibitors), planned to begin enrolling in the second quarter of 2021. Aileron is developing ALRN-6924 as a novel medicine to selectively protect healthy cells in patients with cancers that harbor p53 mutations to reduce or eliminate chemotherapy-induced side effects while preserving chemotherapys activity on cancer cells, a concept known as chemoprotection.

2020 has been a year of important progress for Aileron, most notably achieving clinical proof-of-concept for ALRN-6924 as a chemoprotective agent in patients with p53-mutated small cell lung cancer (SCLC) undergoing treatment with topotecan, said Manuel Aivado, M.D., Ph.D., President and Chief Executive Officer of Aileron. In 2021, we aim to make further strides in our clinical development efforts by initiating a randomized, placebo-controlled trial of ALRN-6924 in patients with advanced NSCLC. This trial represents a key step toward advancing our vision to bring chemoprotection to all patients with p53-mutant cancer regardless of cancer type or chemotherapy. We believe chemoprotection has the potential to transform chemotherapy similar to the way anesthesia transformed surgery.

Dr. Aivado further commented, February 2021 will mark a critical milestone for the emerging chemoprotection field the first PDUFA date of a chemoprotective agent. While that therapeutics focus is myelopreservation, or the concept of protecting bone marrow cells from chemotherapy-induced toxicities, we view chemoprotections potential through a broader lens. We believe that, due to its mechanism of action, ALRN-6924 may have the potential not only to protect bone marrow cells from chemotherapy but also to protect other cell types, such as hair follicle cells and cells lining the oral cavity, among others. Currently there are no therapies to prevent hair loss or the painful mouth sores that patients undergoing chemotherapy often experience due to chemotherapys destruction of healthy cells.

Aileron Strategic Priorities and Anticipated Key Value Drivers in 2021

Initiate Phase 1b Randomized, Double-Blind, Placebo-Controlled Clinical Trial in First-Line NSCLC and Report Initial Data

Today, Aileron announced additional details about its planned Phase 1b clinical trial of ALRN-6924 in patients with advanced NSCLC. The company plans to begin patient enrollment in the second quarter of 2021, subject to obtaining funding for the trial, and expects that the randomized, double-blind, placebo-controlled trial will be part of a registration program designed to ultimately support approval for ALRN-6924 in NSCLC. Aileron anticipates enrolling approximately 40 patients with stage IV NSCLC undergoing treatment with first-line carboplatin doublet chemotherapy with or without an immune checkpoint inhibitor. Patients will be randomized 1:1 to receive either 0.3 mg/kg of ALRN-6924 or placebo. Endpoints will include the effect of ALRN-6924 to limit chemotherapy-induced bone marrow toxicities. Aileron anticipates reporting initial results from the trial late in the fourth quarter of 2021 and full results mid-2022.

In the U.S. alone, there are nearly 200,000 new cases of NSCLC diagnosed each year, and an estimated 50% or more of patients with NSCLC have p53-mutated cancer.1,2 Across all cancer types, there are an estimated 1.8 million patients in the U.S. diagnosed each year, and an estimated 50% of all cancer patients have p53-mutated cancer.1,3

Report Data Readouts from Ongoing Phase 1b Clinical Trial of ALRN-6924 in SCLC and Ongoing Healthy Volunteer Study

In 2021, Aileron plans to report additional results from its ongoing Phase 1b clinical trial in patients with SCLC. In October 2020, Aileron presented positive clinical data from the trial demonstrating clinical proof-of-concept that treatment with ALRN-6924 resulted in a protective effect against severe anemia, thrombocytopenia and neutropenia in patients with p53-mutated SCLC treated with topotecan. The data set from this trial, which Aileron plans to announce in the first quarter of 2021, will include results across all dose levels evaluating ALRN-6924 administered 24 hours prior to topotecan administration (24-hr schedule), including an exploratory 0.2 mg/kg dose level, as well as results from a cohort evaluating 0.3 mg/kg ALRN-6924 administered six hours prior to topotecan administration (6-hr schedule), which has now completed enrollment, with a total of six patients. In addition, Aileron initiated a healthy volunteer study in November 2020 to characterize the time to onset, and magnitude and duration of cell cycle arrest in human bone marrow relative to ALRN-6924 administration. Due to COVID-19-related delays, Aileron is updating its guidance on the readout of the healthy volunteer study from the second quarter of 2021 to mid-2021.

We expect that, taken together, these additional results from the Phase 1b clinical trial of ALRN-6924 in patients with small cell lung cancer and the results from the healthy volunteer study, will further inform and support future randomized, controlled trials of ALRN-6924 when given prior to various chemotherapies, said Dr. Aivado.

How ALRN-6924 Is Designed to Protect Healthy Cells from Chemotherapy

ALRN-6924 is being developed by Aileron as a novel chemoprotective medicine to selectively protect healthy cells in patients with cancers that harbor p53 mutations to reduce or eliminate chemotherapy-induced side effects.

Chemotherapy preferentially acts on cells that are cycling or undergoing the process of cell division. In cancer cells, the cell cycle is unchecked, which leads to uncontrolled cell proliferation, a hallmark of cancer. Certain types of healthy cells also naturally need to cycle, such as bone marrow cells, hair follicle cells, skin cells, and cells lining the oral cavity and the gastrointestinal tract. As a result, chemotherapy preferentially targets and kills both cycling healthy cells and cycling cancer cells. This, in turn, can lead to a spectrum of chemotherapy-induced side effects, from unpleasant to life-threatening and fatal.

ALRN-6924, an investigational first-in-class MDM2/MDMX dual inhibitor, is administered prior to chemotherapy to patients with p53-mutant cancers. ALRN-6924 is designed to activate normal p53 protein in patients healthy cells, temporarily and reversibly pausing cell cycling to selectively shield the patients healthy cells from chemotherapy. The protection is limited to healthy cells, as ALRN-6924 cannot work in p53-mutated cancer cells given that p53 has lost its function in those cells. Therefore, cancer cells continue to cycle uninterrupted and remain fully susceptible to destruction by chemotherapy.

About Aileron Therapeutics

At Aileron, we are focused on transforming the experience of chemotherapy for cancer patients, enabling them to fight cancer without the fear or burden of chemotherapy-induced side effects. ALRN-6924, our first-in-class MDM2/MDMX dual inhibitor activating p53, is the only reported chemoprotective agent in clinical development to employ a biomarker strategy, in which we exclusively focus on treating patients with p53-mutated cancers. With this unique, targeted strategy, ALRN-6924 is designed to protect multiple healthy cell types throughout the body from chemotherapy while chemotherapy continues to destroy cancer cells.

In addition to potentially reducing or eliminating multiple side effects, ALRN-6924 may also improve patients quality of life and help them better tolerate chemotherapy, potentially allowing patients to complete their treatment without dose reductions or delays. Our vision is to bring chemoprotection to patients with p53-mutated cancers approximately 50% of cancer patients regardless of cancer type or chemotherapy. Visit us at aileronrx.com to learn more.

Forward-Looking Statements

Statements in this press release about Aileron's future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements about the Companys strategy and clinical development plans. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, would and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including whether Ailerons cash resources will be sufficient to fund its continuing operations for the periods anticipated; whether the Company will obtain sufficient cash resources to conduct its planned clinical trials; whether initial results of clinical trials will be indicative of final results of those trials or results obtained in future clinical trials; whether Ailerons product candidates will advance through the clinical trial process on a timely basis, or at all; whether the results of such trials will be accepted by and warrant submission for approval from the United States Food and Drug Administration or equivalent foreign regulatory agencies; whether Aileron's product candidates will receive approval from regulatory agencies on a timely basis or at all; whether, if product candidates obtain approval, they will be successfully distributed and marketed; what impact the coronavirus pandemic may have on the timing of our clinical development, clinical supply and our operations; and other factors discussed in the "Risk Factors" section of Aileron's quarterly report on Form 10-Q for the period ended September 30, 2020, filed on November 12, 2020, and risks described in other filings that Aileron may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Aileron specifically disclaims any obligation to update any forward-looking statement, whether because of new information, future events or otherwise.

Investor Contacts: Media Contact:Richard Wanstall, SVP Chief Financial Officer Liz MeloneAileron Therapeutics 617-256-6622617-995-0900 lmelone@aileronrx.comrwanstall@aileronrx.com

Hans C. VitzthumLifeSci Advisors, LLC.617-430-7578hans@lifesciadvisors.com

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1 American Cancer Society. Cancer Facts & Figures 2020. Atlanta: American Cancer Society.2 The AACR Project GENIE Consortium. AACR Project GENIE: powering precision medicine through an international consortium. Cancer Discovery. 2017;7(8):818-831.3 Hoe, K., Verma, C., and Lane, D.P., Drugging the p53 pathway: understanding the route toclinical efficacy. Nature Reviews Drug Discovery. 2014;13:218-236.

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Aileron Therapeutics Provides Business Update and Outlines 2021 Strategic Priorities - BioSpace

Hui Wang, Shaoxia Du, Jun Cai, Juan Wang, Xiaohong Shen

School of Medicine, Nankai University, Tianjin 300071, Peoples Republic of China

Correspondence: Xiaohong ShenSchool of Medicine, Nankai University, Nankai District, Tianjin 300071, Peoples Republic of ChinaTel +86-22-23501649Email zebial2014@163.com

Background: Apolipoprotein E2 (ApoE2) is reported to be essential for cell metastasis and proliferation and has been considered a potential diagnostic marker in many cancers. However, the function of ApoE2 in the metastasis of pancreatic cancer, as well as the underlying mechanism, remain unclear.Purpose: In this study, we explored the effect of ApoE2 on the migration and invasion abilities of pancreatic cancer cells and explored the underlying molecular mechanism.Methods and Results: Wound healing and Matrigel Transwell assays were used to investigate the role of ApoE2 in cell migration and invasion. Western blotting analysis showed that ApoE2 was overexpressed in pancreatic cancer tissues. Additionally, the overexpression of ApoE2 promoted the process of epithelialmesenchymal transition (EMT) and enhanced the expression of MMP-2/9 in pancreatic cancer cells. Mechanistically, we found that inhibition of ERK1/2 signaling with PD98059 impaired the ApoE2-mediated promotion of cell migration, invasion and EMT.Conclusion: This study demonstrated that ApoE2/ERK1/2 signaling promoted the migration and invasion of pancreatic cancer cells. ApoE2 might be a potential therapeutic target for the treatment of pancreatic cancer metastasis.

Keywords: apolipoprotein E2, ERK1/2, migration, invasion, pancreatic cancer

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Apolipoprotein E2 Promotes the Migration and Invasion of Pancreatic Ca | CMAR - Dove Medical Press

OSLO, Norway--(BUSINESS WIRE)--Ultimovacs ASA ("Ultimovacs", ticker ULTI), today announced the initiation of FOCUS, a Phase II randomized clinical trial that will evaluate the Companys proprietary universal cancer vaccine, UV1, in 75 patients with recurrent or metastatic head and neck cancer who will be treated with standard of care therapy pembrolizumab. The trial will be conducted at 10 sites across Germany and led by principal investigator Prof. Mascha Binder, M.D., Medical Director and Head of the Immunological Tumor Group at University Medicine Halle, Germany, who is a renowned oncology clinician and researcher specializing in the analysis of immuno-oncology treatments and their interaction with tumor tissues. The FOCUS trial is Ultimovacs fourth Phase II clinical trial with UV1 and comes in addition to the collaboration Phase II trial announced in May 2020.

We continue to strategically expand our clinical development program for our proprietary cancer vaccine, not only by testing UV1 in additional cancer indications with critical need, such as head and neck cancer, but also by collaborating with clinicians like Professor Binder who can provide us with unique insights, stated Jens Bjrheim, Chief Medical Officer at Ultimovacs. Professor Binder and her team are well-recognized experts who will increase our understanding of UV1s mechanism of action and contribute data on anti-tumor activity induced by UV1 vaccination.

The FOCUS (First-line metastatic Or recurrent HNSCC/Checkpoint inhibitor UV1 Study) Phase II trial is an investigator-sponsored, randomized Phase II clinical trial that will recruit patients with recurrent or metastatic PD-L1 positive head and neck squamous cell carcinoma across 10 clinical sites in Germany. The trial will evaluate the addition of UV1 to a standard of care treatment with PD-1 checkpoint inhibitor pembrolizumab as compared to pembrolizumab monotherapy. A total of 75 patients indicated for treatment with pembrolizumab will be enrolled in the FOCUS study, randomized 2-to-1 so that 50 patients will receive UV1 and pembrolizumab and 25 patients will receive pembrolizumab alone. The primary endpoint of the study is the progression-free survival rate at 6 months, and planned readout of topline results is expected in 2023. The FOCUS Phase II trial is partially supported through an innovation grant of up to NOK 16 million from the Norwegian Research Council and will not require Ultimovacs to secure additional financing at this stage.

This clinical trial is the extension of an earlier collaboration with Ultimovacs that introduced me to the therapeutic potential of the UV1 approach, commented Professor Mascha Binder, M.D., Medical Director and Head of the Immunological Tumor Group at University Medicine Halle. I am excited to apply my own insights as well as my teams expertise to this trial with the goal of potentially providing head and neck cancer patients with better treatment options.

We have continued to build momentum in our broad clinical development program with the start of the FOCUS study, representing the important opportunity to test UV1 in combination with pembrolizumab in a randomized setting for the first time, said Carlos de Sousa, Chief Executive Officer at Ultimovacs. We welcome the funding provided by the Norwegian Research Council, which supports our ability as a small biotechnology company to conduct four Phase II trials in parallel and evaluate UV1 in different indications with more than 450 patients and as part of various treatment combinations.

Dr. de Sousa added: Although we have signed our agreement with the lead investigator for our previously announced third Phase II clinical trial, we will need to wait for the investigator and the pharmaceutical partner to finalize the signature process for their agreement before we can provide full details, which we expect to do before year end.

About UV1UV1 is a peptide-based vaccine inducing a specific T cell response against the universal cancer antigen telomerase. UV1 is being developed as a therapeutic cancer vaccine which may serve as a platform for use in combination with other immunotherapy which requires an ongoing T cell response for their mode of action. To date, UV1 has been tested in four phase I clinical trials in a total of 82 patients and maintained a positive safety and tolerability profile as well as encouraging signals of efficacy.

About UV1 Clinical ProgramsAs a universal cancer vaccine, UV1s unique mechanism of action has the potential to be applicable across most cancer types. The clinical development of the UV1 vaccine includes four randomized, multinational, Phase II combination trials recruiting more than 450 patients in total. The INITIUM trial is an Ultimovacs-sponsored clinical trial recruiting 154 patients with metastatic malignant melanoma to evaluate UV1 in combination with ipilimumab and nivolumab as first-line treatment. The NIPU study is testing UV1 in combination with checkpoint inhibitors ipilimumab and nivolumab as second-line treatment in 118 patients with advanced malignant pleural mesothelioma, a rare lung cancer. The study is sponsored by Oslo University Hospital and Bristol-Myers Squibb is providing the checkpoint inhibitors for this study. Ultimovacs anticipates announcing data on the primary endpoints for the NIPU and INITIUM studies in 2022. A third Phase II clinical trial will evaluate UV1 in a new cancer indication in combination with indication-specific standard of care cancer therapies different from those to be tested in INITIUM and NIPU. In this new collaboration, Ultimovacs will supply UV1 and a big pharma company will supply its proprietary cancer treatment to the clinical trial group, which will sponsor the trial. The fourth trial, FOCUS, is an investigator-sponsored, randomized clinical trial, enrolling 75 patients with metastatic head and neck cancer receiving pembrolizumab as standard of care, to evaluate the impact of adding UV1 to this regimen.

About UltimovacsUltimovacs UV1 universal cancer vaccine candidate leverages the high prevalence of the human telomerase (hTERT) to be effective across the dynamic stages of the tumors growth and its microenvironment. By directing the immune system to hTERT antigens that are present in over 80% of all cancers, UV1 drives CD4 helper T cells to the tumor with the goal of activating an immune system cascade to increase anti-tumor responses. Ultimovacs strategy is to clinically demonstrate UV1s impact in a range of cancers and in several immunotherapy combinations while expanding our pipeline of cancer vaccine therapies, convinced that a universal approach may be the key to achieving better outcomes for patients.

For further information, please see http://www.ultimovacs.com

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Ultimovacs ASA Initiates FOCUS Phase II Trial for Universal Cancer Vaccine, UV1, in Head and Neck Cancer Patients Receiving Pembrolizumab - Business...

Immunai, a company specializing in comprehensive mapping of the human immune system, is joining forces with 10x Genomics. The latter will leverage its single-cell technologies to map hundreds of cell types and states. By applying its artificial intelligence (AI) and machine learning (ML) algorithms, Immunai supports biomarker discovery and insight generation to help power new therapeutic discoveries and accelerate drug development.

Outsourcing-Pharma (OSP) discussed the partnership with Luis Voloch (LV), CTO and co-founder of Immunai, and how the map generated through the collaborative effort stands to benefit drug developers.

OSP: Please tell us a bit about Immunai.

LV: Immunai is comprehensively mapping the immune system to power new therapeutic discoveries, accelerate drug development, and improve patient outcomes. Leveraging single-cell technologies to profile cells and machine learning to map incoming data to hundreds of cell types and states, Immunai supports biomarker discovery and insight generation to better detect, diagnose, and treat disease.

The immune system is an incredibly complex, distributed system that researchers have been trying to understand with limited success for years. Immunai is the first company to fully map the immune system, generating the largest proprietary database for immunology.

Were disrupting legacy companies by analyzing 10,000 times more data from each cell than they are. No one is doing exactly what were doing.

OSP: How did you come to partner with 10x Genomics?

LV: There is an undeniable fit between the goals and capabilities of our two companies. At Immunai, we want to use AI to identify and understand novel elements within hundreds of different cell types to inform drug development, and we have been leveraging 10xs products to do that at a granular level from the start.

Through our initial work together, we identified even more mutually beneficial applications of our technologies for pharma companies and academic institutions alike. So we most recently applied to 10xs Certified Service Provider Program to give 10xs customers access to our advanced immune profiling solutions.

OSP: What does each of you bring to the table in this partnership, and how will the collaboration work?

LV: With this collaboration, we will pair our immune cell atlas with the phenotypic clinical data that hospitals, biopharma, and biotech companies derive from 10xs technology. With Immunais end-to-end computational AI pipeline customized for single-cell methods, researchers at pharmaceutical and cell therapy companies can better understand how immune cells operate with both granularity and scale. In turn, we will help 10xs customers answer clinical and translational questions related to the immune response to therapies.

OSP: Could you please talk a bit about the evolution of AI and how drug discovery professionals have made use of it to date?

LV: An analysis published earlier this year in the Journal of the American Medical Association found that the median cost of R&D for a new drug in the years between 2009 and 2018 was $985 million. This ever-increasing cost forces pharma companies to search for innovative means to create efficiencies in drug development.

Pharma companies are catching on to what Immunai already knows: AI can maximize our ability to layer data points, uncover deep insights, and advance research.

We envision AIin conjunction with human intelligenceas the major component to understanding and curing cancer. AI will increasingly have a tremendous impact on pharma. Pharma has traditionally had to experiment by testing out different compounds in a dish or in animals.

With more biological data available, AI provides a partial alternative to this that allows us to predict (without actual experiments) the impact of compounds in different biological systems. This ability has increased the speed in which we can profile and improve compounds.

OSP: What is particularly novel and noteworthy about this projectwhat do you hope to accomplish that hasnt been accomplished before?

LV: Until now, no one has been able to uncover the complexities of the immune system in the way that Immunai has. Current single-cell approaches generally operate at the scale of small academic studies because they suffer from the problem of batch effects, where noise from variation in biological samples quickly washes out any real biological signal as scale grows.

Immunais end-to-end platform is designed to manage batch effects through both proprietary lab methods and advanced AI, allowing us to build a large multi-omic single-cell database that we pair with clinical context. We train our proprietary neural network models on this data to surface insights about immune responses and facilitate the development of better therapies.

This lack of understanding of the immune system contributes to inefficiencies in drug R&D. Developing immunotherapies based on information provided by only two cells doesnt give researchers a view of the entire picture.

We believe that this collaboration will help to drastically improve the development of therapies and answer some of the biggest questions about cancer.

OSP: Is there anything youd like to add that we didnt touch upon?

LV: Our work with 10x is the second official collaboration weve announced over the past few months. In November, we announced a collaboration with Baylor College of Medicine to drive forward the development of novel NKT cell therapies. As our database continues to grow with these partnerships, we can apply learnings around immune response across different diseases from cancer to autoimmune disorders to cardiovascular diseases as well.

Continued here:
Collaboration to chart AI-generated map of the immune system - OutSourcing-Pharma.com

By Frances D'Emilio, The Associated Press on December 21, 2020.

VATICAN CITY The Vatican on Monday declared that it is morally acceptable for Roman Catholics to receive COVID-19 vaccines based on research that used cells derived from aborted fetuses, guidance that came after some churchmen in the United States argued that such products were immoral.

The Congregation for the Doctrine of the Faith, the Vaticans watchdog office for doctrinal orthodoxy, said it had received several requests for guidance during recent months. The doctrinal office pointed out that bishops, Catholic groups and experts have offered diverse and sometimes conflicting pronouncements on the matter.

Drawing on Vatican pronouncements in past years about developing vaccines prepared from cells derived from aborted fetuses, the watchdog offices statement was examined by Pope Francis, who ordered it to be made public.

The Catholic Churchs teaching says that abortion is a grave sin.

The Vatican concluded that it is morally acceptable to receive COVID-19 vaccines that have used cell lines from aborted fetuses in the research and production process when ethically irreproachable vaccines arent available to the public. But it stressed that the licit uses of such vaccines does not and should not in any way imply that there is a moral endorsement of the use of cell lines proceeding from aborted fetuses.

The Vatican didnt name any of the COVID-19 vaccines already being given to people in some countries or authorized to be used soon.

In its statement, the Vatican explained that obtaining vaccines that do not pose an ethical dilemma is not always possible. It cited circumstances in countries where vaccines without ethical problems are not made available to physicians and patients or where special storage or transport conditions make their distribution more difficult.

Much of the Vaticans pronouncement had echoes in a statement last week by officials of the U.S. Conference of Catholic Bishops. The U.S. conference officials said that in view of the gravity of the current pandemic and the lack of availability of alternative vaccines, receiving the vaccines being distributed in the United States is justified despite their remote connection to morally compromised cell lines.

Getting vaccinated against the coronavirus ought to be understood as an act of charity toward the other members of our community, the U.S. bishops conference officials said.

Weeks earlier, two U.S. bishops, one in Texas and one in California, had denounced vaccines using cell lines from the tissue of aborted fetuses as immorally produced. One of the bishops said he refused to receive such a vaccine and encouraged rank-and-file Catholics to follow his lead.

The Vatican, in reassuring faithful Catholics that getting a COVID-19 vaccine would not violate the churchs moral teaching, noted that health authorities do not allow citizens to choose the vaccine with which to be inoculated. Given such circumstances, it is morally acceptable to receive vaccines that have used cell lines from aborted fetuses, the Vatican said.

The Vatican said the COVID-19 vaccines that are getting rolled out or are expected to be soon used cell lines drawn from tissue obtained from two abortions that occurred in the last century.

The Vatican hasnt said if and when Francis would be vaccinated against the coronavirus. The 84-year-old pontiff has a pilgrimage to Iraq planned for early March, and its widely expected that he and the aides accompanying him would get vaccinated ahead of travel abroad.

The Roman Catholic churchs doctrinal orthodoxy office said vaccination is not, as a rule, a moral obligation and must be voluntary. Still, it said, from an ethical point of view, the morality of vaccination depends not only on the duty to protect ones own health but also on the duty to pursue the common good.

Those for reasons of conscience opting not to receive vaccinations produced by cell lines from aborted fetuses, must do their utmost to avoid, by appropriate behaviour and preventive means, becoming vehicles for transmission, the congregation said.

In any case, there is also a moral imperative for the pharmaceutical industry, governments and international organizations to ensure that safe, effective and ethically acceptable vaccines are accessible to the poorest countries and not too costly for them, the Vaticans doctrinal office said.

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Vatican: OK to get virus vaccines using abortion cell lines - Medicine Hat News

New York, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Cell Isolation/Cell Separation Market by Product, Cell Type, Cell Source, Technique, Application, End-User - Global Forecast to 2025" - https://www.reportlinker.com/p04315097/?utm_source=GNW With the rising focus on the development of personalized medicine, the number of personalized medications available in the market has steadily increased over the last decade, and this trend is expected to continue in the coming years.

The consumablesaccounted for the highest growth rate in thecell isolationmarket, by productduring the forecast periodBased on product, the cell isolation market is segmented into consumables and instruments.The consumables segment accounted for the largest share in the cell isolation market in the forecasted period.

The increasing investments by companies to develop technologically advanced products as well as the repetitive use of consumables as compared to instruments are the major factors driving the growth of this segment.

Human cells segment accounted for the highest CAGRBased on cell type, the cell isolation market is segmented into human cells and animal cells.The human cells segment accounted for the largest share of the global cell isolation market in the forecasted period.

The increasing investments by public and private organizations for research on human cells, growing application areas of human stem cells, and the high and growing incidence of diseases such as cancer are the major factors driving this segments growth.

Biotechnology and biopharmaceutical companiessegment accounted for the highest CAGRThe cell isolation market is segmented into hospitals and diagnostic laboratories, biotechnology and biopharmaceutical companies, research laboratories and institutes, and other end users based on end users.In 2019, the biotechnology and biopharmaceutical companies segment accounted for the largest share.

The widespread adoption of advanced instruments in cell-based experiments and cancer research in biotechnology and biopharmaceutical companies, as well as the increasing number of R&D facilities globally are the major factors driving this segments growth.

Asia Pacific: The fastest-growing regioncell isolation marketThe global cell isolation market is segmented into North America, Europe, Asia Pacific, and Rest of the world.The Asia Pacific region is projected to register the highest CAGR during the forecast period.

Growth in this region is expected to be centered on China and Japan. Factors such as the expansion by key market players in emerging Asian countries and the increasing trend of pharmaceutical outsourcing to Asian countries like India and China are driving the growth of the cell isolation market in this region.

The primary interviews conducted for this report can be categorized as follows: By Company Type: Tier 1 - 20%, Tier 2 - 45%,and Tier 3 -35% By Designation: C-level - 30%, D-level - 20%, and Others - 50% By Region: North America -35%, Europe - 24%, Asia Pacific - 25%, Rest of the world 16%

Lits of Companies Profiled in the Report: Thermo Fisher Scientific, Inc. (US) Becton, Dickinson and Company Limited (US) Beckman Coulter Inc. (US).Merck KGaA (Germany) Terumo BCT (Japan), GE Healthcare (US) Bio- Rad Laboratories Inc. (US) Corning Inc. (US) Roche Diagnostics (Switzerland) Alfa Laval (Sweden) Miltenyl Biotech (Germany) pluriSelect Life Science (Germany) STEMCELL Technologies Inc. (Canada) Akadeum Life Sciences, Inc (US) Bio- Techne (US), Bio Legend (US) Invent Biotechnologies (US)

Research Coverage:This report provides a detailed picture of the global cell isolation market.It aims at estimating the size and future growth potential of the market across different segments, such as product, celltype, cell source, technique, application, end user, and region.

The report also includes an in-depth competitive analysis ofthe key market players, along with their company profiles, recent developments, and key market strategies.

Key Benefits of Buying the Report:The report will help market leaders/new entrants by providing them with the closest approximations of the revenue numbers for the overall cell isolation market and its subsegments.It will also help stakeholders better understand the competitive landscape and gain more insights to better position their business and make suitable go-to-market strategies. This report will enable stakeholders to understand the markets pulse and provide them with information on the key market drivers, restraints, trends, and opportunities.

Read the full report: https://www.reportlinker.com/p04315097/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The global cell isolation market size is projected to reach USD 15.0 billion by 2025 from USD 6.9billion in 2020, at a CAGR of 16.8% - GlobeNewswire

BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) expanded the eligibility for TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include people with cystic fibrosis (CF) ages 12 years and older with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that are responsive to TRIKAFTA based on in vitro data. SYMDEKO (tezacaftor/ivacaftor and ivacaftor) and KALYDECO (ivacaftor) also received approvals to include additional responsive mutations in people with CF ages 6 years and older and age 4 months and older, respectively. These approvals allow more than 600 people with CF not previously eligible for these medicines an opportunity to potentially benefit from treatment that targets the underlying cause of their disease.

The approval for expanded use of three of our CF medicines based on our well-established in vitro model is a testament to the relentless commitment of our scientists to reach our goal of developing treatments for all people with CF, said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. We remain as committed today to reaching every patient who might benefit from our medicines as when we first started out on this journey 20 years ago, and this important milestone now enables hundreds of people with CF access to a treatment option to address the underlying cause of their disease many for the first time.

TRIKAFTA was previously approved for people with at least one F508del mutation and is now approved for 177 additional mutations; SYMDEKO is now approved for 127 additional mutations, for a total of 154 SYMDEKO-responsive mutations; and KALYDECO is now approved for an additional 59 mutations, for a total of 97 KALYDECO-responsive mutations. In addition, for certain people with CF who are currently eligible for KALYDECO, this approval allows them to also be eligible for SYMDEKO or TRIKAFTA; and similarly, for those who are currently eligible for SYMDEKO, this approval allows them to also be eligible for TRIKAFTA.

The full list of mutations for TRIKAFTA, SYMDEKO and KALYDECO can be found within the updated full Prescribing Information for each respective product. In addition, people with CF and their families can search eligibility for Vertex CF medicines through vertextreatments.com.

About Cystic Fibrosis

Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes one from each parent to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO (ivacaftor), SYMDEKO (tezacaftor/ivacaftor and ivacaftor), and TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor)

What is KALYDECO?

KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients

age 4 months and older who have at least one mutation in their CF gene that is responsive to KALYDECO. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if KALYDECO is safe and effective in children under 4 months of age.

What is SYMDEKO?

SYMDEKO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have two copies of the F508del mutation, or who have at least one mutation in the CF gene that is responsive to treatment with SYMDEKO. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if SYMDEKO is safe and effective in children under 6 years of age.

What is TRIKAFTA?

TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 12 years of age.

Patients should not take KALYDECO, SYMDEKO, or TRIKAFTA if they take certain medicines or herbal supplements, such as: the antibiotics rifampin or rifabutin; seizure medications such as phenobarbital, carbamazepine, or phenytoin; or St. Johns wort.

Before taking KALYDECO, SYMDEKO, or TRIKAFTA, patients should tell their doctor about all of their medical conditions, including if they: have kidney problems; have or have had liver problems; are pregnant or plan to become pregnant because it is not known if KALYDECO, SYMDEKO, or TRIKAFTA will harm an unborn baby; or are breastfeeding or planning to breastfeed because it is not known if KALYDECO, SYMDEKO, or TRIKAFTA passes into breast milk. Before taking KALYDECO, patients should tell their doctor if they drink grapefruit juice or eat grapefruit.

KALYDECO, SYMDEKO, or TRIKAFTA may affect the way other medicines work, and other medicines may affect how KALYDECO, SYMDEKO, or TRIKAFTA work. Therefore, the dose of KALYDECO, SYMDEKO, or TRIKAFTA may need to be adjusted when taken with certain medications. Patients should especially tell their doctor if they take antifungal medications such as ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; or antibiotics such as telithromycin, clarithromycin, or erythromycin.

KALYDECO, SYMDEKO, or TRIKAFTA can cause dizziness in some people who take it. Patients should not drive a car, use machinery, or do anything that needs them to be alert until they know how KALYDECO, SYMDEKO, or TRIKAFTA affects them.

Patients should avoid food or drink containing grapefruit while taking KALYDECO, SYMDEKO or TRIKAFTA.

KALYDECO, SYMDEKO, and TRIKAFTA can cause serious side effects, such as:

High liver enzymes in the blood have been reported in patients receiving KALYDECO, SYMDEKO, or TRIKAFTA. The patient's doctor will do blood tests to check their liver before starting treatment with KALYDECO, SYMDEKO, or TRIKAFTA, every 3 months during the first year of treatment, and every year while on treatment. Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of their skin or the white part of their eyes; loss of appetite; nausea or vomiting; or dark, amber-colored urine.

Abnormality of the eye lens (cataract) in some children and adolescents treated with KALYDECO, SYMDEKO, or TRIKAFTA. If the patient is a child or adolescent, their doctor should perform eye examinations before and during treatment with KALYDECO, SYMDEKO, or TRIKAFTA to look for cataracts.

The most common side effects of KALYDECO include headache; upper respiratory tract infection (common cold), which includes sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness.

The most common side effects of SYMDEKO include headache, nausea, sinus congestion, and dizziness.

The most common side effects of TRIKAFTA include headache, diarrhea, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, inflamed sinuses, increase in liver enzymes, increase in a certain blood enzyme called creatine phosphokinase, rash, flu (influenza), and increase in blood bilirubin.

These are not all the possible side effects of KALYDECO, SYMDEKO, or TRIKAFTA. Please click product link to see the full Prescribing Information for KALYDECO, SYMDEKO or TRIKAFTA.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Reshma Kewalramani in this press release, statements regarding the eligible patient population for TRIKAFTA, SYMDEKO and KALYDECO, our expectations regarding the number of patients newly eligible for TRIKAFTA, SYMDEKO and KALYDECO, and statements regarding the potential benefits of TRIKAFTA, SYMDEKO and KALYDECO. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under the heading Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission (SEC) and available through the company's website at http://www.vrtx.com and on the SECs website at http://www.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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Vertex Announces FDA Approvals of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO (tezacaftor/ivacaftor and ivacaftor) and KALYDECO...