Category Archives: Stell Cell Research


VERTEX PHARMACEUTICALS INC / MA Management’s Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) – Marketscreener.com

OVERVIEW

Financial Highlights

Revenues In the first quarter of 2023, our net product revenues increased to $2.4 billion

primarily due to the

internationally and

Expenses Our total research and development ("R&D"), acquired in-process research and

administrative ("SG&A") expenses

compared to $818.3

primarily due to increased

in mid- to late-stage

net product revenues in

increased to $11.5

as of December 31, 2022

cash flows partially

Inc. ("Entrada") and

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TRIKAFTA/KAFTRIO is approved and reimbursed or accessible in more than 30countries outside the U.S.

Potential Near-Term Launch Opportunities

We are preparing for the following near-term launches of potential new products:

Exa-cel in SCD and TDT

We recently completed rolling submissions of our biologics licensingapplications ("BLAs") for exa-cel in the U.S. Exa-cel has been granted FastTrack, Regenerative Medicine Advanced Therapy, Orphan Drug and Rare PediatricDisease designations in the U.S.

Vanzacaftor/tezacaftor/deutivacaftor in CF

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Pipeline

Cystic Fibrosis

Beta Thalassemia and Sickle Cell Disease

We are evaluating the use of exa-cel, a non-viral ex vivo CRISPR gene-editingtherapy, for the treatment of SCD and TDT.

Dosing in the Phase 3 CLIMB-111 and CLIMB 121 clinical trials evaluatingexa-cel continues, as does the CLIMB 131 long-term follow-up clinical trial inpatients 12 years of age and older.

Two additional Phase 3 clinical trials evaluating exa-cel in children with SCDor TDT 5 to 11 years of age are ongoing.

Neuropathic Pain

APOL1-Mediated Kidney Disease

The FDA granted inaxaplin Breakthrough Therapy designation for APOL1-mediatedFSGS and the EMA granted inaxaplin Orphan Drug and PRIME designations for AMKD.

Type 1 Diabetes

Our hypoimmune cell research program continues to progress.

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Alpha-1 Antitrypsin Deficiency

Additional Earlier Stage R&D Programs

Investments in External Innovation

Recent investments in external innovation are included below:

We announced a new licensing agreement for the use of CRISPR's gene-editingtechnology, known as CRISPR/Cas9, to accelerate the development of ourhypoimmune cell therapies for T1D.

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Sales of our products depend, to a large degree, on the extent to which ourproducts are reimbursed by third-party payors, such as government healthprograms, commercial insurance and managed health care organizations.Reimbursement for our products, including our potential pipeline therapies,cannot be assured and may take significant periods of time to obtain. Wededicate substantial management and other resources to obtain and maintainappropriate levels of reimbursement for our products from third-party payors,including governmental organizations in the U.S. and ex-U.S. markets.

We expect to continue to identify and evaluate potential acquisitions and mayinclude larger transactions or later-stage assets.

Collaboration and In-Licensing Arrangements

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In February 2023, we closed our strategic collaboration and licensing agreementwith Entrada. Upon closing, we made an upfront payment of $225.1 million toEntrada, and purchased $24.9 million of Entrada's common stock.

Acquired In-Process Research and Development Expenses

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millions, except percentages and per share amounts)Product revenues, net

millions, except percentages)

millions, except percentages)

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Research and Development Expenses

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Our research expenses have been increasing over the last several years as wehave invested in our pipeline and expanded our cell and genetic therapycapabilities, resulting in increased headcount, outside services and otherdirect expenses and infrastructure costs associated with our researchfacilities. We expect to continue to invest in our research programs with afocus on creating transformative medicines for serious diseases.

Our development expenses increased by $118.5 million, or 26%, in the firstquarter of 2023 as compared to the first quarter of 2022, primarily due toincreased costs to support clinical trials associated with our advancingpipeline programs, including pain, our CF triple combination ofvanzacaftor/tezacaftor/deutivacaftor, exa-cel and T1D. We are significantlyinvesting in internal headcount, leveraging outsourced services, and investingin infrastructure to support these programs.

Acquired In-process Research and Development Expenses

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to another due to upfront, contingent milestone, and other payments pursuant toour existing and future business development transactions, includingcollaborations, licenses of third-party technologies, and asset acquisitions.

Selling, General and Administrative Expenses

Selling, general and administrative expenses increased by 12% in the firstquarter of 2023 as compared to the first quarter of 2022, primarily due to thecontinued investment to support the commercialization of our medicines andincreased support for our pipeline product candidates.

Contingent Consideration

The fair value of our contingent consideration decreased by $1.9 million and$7.5 million in the first quarter of 2023 and 2022, respectively.

Other Non-Operating Income (Expense), Net

Interest Income

Interest Expense

Interest expense was $11.4 million and $14.9 million in the first quarter of2023 and 2022, respectively. The majority of our interest expense in theseperiods was related to imputed interest expense associated with our leasedcorporate headquarters in Boston.

Other Income (Expense), Net

Income Taxes

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LIQUIDITY AND CAPITAL RESOURCES

The following table summarizes the components of our financial condition as ofMarch 31, 2023 and December 31, 2022:

Net cash provided by (used in):

Investing Activities

Financing Activities

Sources and Uses of Liquidity

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Credit Facilities & Financing Strategy

Future Capital Requirements

We have significant future capital requirements, including:

Facility and finance lease obligations.

Royalties we pay to the Cystic Fibrosis Foundation on sales of our CF products.

Cash paid for income taxes.

In addition, we have significant potential future capital requirementsincluding:

To the extent we borrow amounts under our existing credit agreement, we wouldbe required to repay any outstanding principal amounts in 2027.

As of March 31, 2023, we had $2.9 billion remaining authorization availableunder our Share Repurchase Program.

There have not been any material changes to our future capital requirementsdisclosed in our Annual Report on Form 10-K for the year ended December 31,2022, which was filed with the Securities and Exchange Commission, or SEC, onFebruary 10, 2023.

CRITICAL ACCOUNTING POLICIES AND ESTIMATES

Our discussion and analysis of our financial condition and results of operationsare based upon our condensed consolidated financial statements prepared inaccordance with generally accepted accounting principles in the U.S. The

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RECENT ACCOUNTING PRONOUNCEMENTS

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VERTEX PHARMACEUTICALS INC / MA Management's Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) - Marketscreener.com

Global Cell and Tissue Analysis Market Research Report 2023 – Benzinga

DUBLIN, May 4, 2023 /PRNewswire/ --The "Global Markets and Technologies for Cell and Tissue Analysis" report has been added to ResearchAndMarkets.com's offering.

The global market for cell and tissue analysis was estimated to be $16.7 billion in 2021 and is expected to increase to $28.9 billion in 2027, growing at a CAGR of 9.6% during the forecast period. In this report the cell and tissue analysis market is segmented based on technology type, end user and region.

This report focuses on the global market of cell and tissue analysis (CTA) products and provides an updated review, including basic design and applications in various arenas of biomedical and life science research. The report deals with CTA products covering the total market, which includes three main areas of applications.

Cell and tissue analysis products are playing pivotal roles in biomedical research. In the expanding field of biomedical research and development researchers and scientists employ a variety of cell and tissue analysis products. These products play important roles in the diagnosis of various forms of cancer and other diseases and also assume crucial roles when it comes to understanding diverse cellular activities. With the rise of chronic and infectious diseases, there is an urgent need for efficient diagnostics and better healthcare conditions to treat diseases. Along with the recent intensification of research in academia and the biomedical industry, demand is increasing significantly for advanced technology for drug development and screening.

Personalized drug treatment is becoming a reality. In response to the rise in the incidence of a number of diseases and an ageing population, the drug discovery industry is developing new and more efficacious drugs based on specific biomarker signatures and hence cell and tissue analysis technologies will also help in personalized drug development.

Apart from this the increasing demand for single-cell analysis and the extensive research ongoing in this area will further contribute to the market. Other drivers contributing to the market include advances in technologies and product launches, increasing prevalence of chronic diseases, an ageing population, and rising investments and funding. However, the market is facing some challenges such as the high cost of instruments, lack of skilled labor and stringent regulations.

Major players in the market are Thermo Fisher Scientific, Danaher, Becton, Dickinson & Co., Illumina, and Agilent Technologies. Technology types covered are biospecimen technology, cell separation, and cell and tissue characterization. Based on type, the cell and tissue characterization segment has the highest share. Each type is further categorized into subtypes.

North America has the highest share of the market by region, followed by Europe. Extensive R&D activities take place in the region, there are major players present, there is plentiful funding, and there is an increasing prevalence of chronic diseases. All these factors will contribute to growth in the market

Report Includes

Key Topics Covered:

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Market and Technology Background3.1 Introduction3.1.1 History3.1.2 Cells3.1.3 Animal Tissue3.2 Classification of Techniques Used in Cell and Tissue Analysis3.3 Biospecimen Techniques3.3.1 Microarray3.3.2 Tissue Microarrays3.3.3 Cellular Microarray3.3.4 Dna Microarray or Dna Chip3.3.5 Protein Microarray or Peptide Chip3.4 Cell-Separation Techniques3.4.1 Mechanical and Physical Dissociation3.4.2 Based on Adherence3.4.3 Based on Size and Density3.4.4 Based on Affinity3.4.5 Lab-On-Chip Techniques3.5 Cell and Tissue Characterization3.5.1 Cell-Based Assays3.5.2 Histology and Immunohistochemistry3.5.3 Flow Cytometry3.5.4 Genotyping and Expression Analysis3.5.5 Western Analysis3.5.6 Components of Cell-Based Assays

Chapter 4 Market Dynamics4.1 Factors Affecting the Market4.1.1 Market Drivers4.1.2 Challenges for the Market for Cell and Tissue Analysis4.1.3 Impact of the Covid-19 Pandemic

Chapter 5 Emerging Technologies

Chapter 6 Market Breakdown by Technology6.1 Cell and Tissue Analysis Technologies6.1.1 Biospecimen Technology6.1.2 Cell Separation Technology6.1.3 Cell and Tissue Characterization

Chapter 7 Market Breakdown by End-user7.1 End-users7.1.1 Biopharmaceutical Companies7.1.2 Academic and Research Institutes7.1.3 Healthcare and Clinical

Chapter 8 Market Breakdown by Region8.1 North America8.2 Europe8.3 Emerging Markets

Chapter 9 Regulatory Aspects9.1 New Approvals of Cell and Tissue Analysis Products9.2 Recalls and Safety Alerts

Chapter 10 Patent Analysis10.1 Patent Activity on Cell and Tissue Analysis10.1.1 Patent Review by Year10.1.2 Patent Review by Country10.1.3 Patent Review by Company, University and Institute

Chapter 11 Competitive Landscape11.1 Mergers and Acquisitions11.2 Competitive Analysis11.2.1 Biospecimen Technology11.2.2 Cell Separation Technology11.2.3 Cell and Tissue Characterization Technology

Chapter 12 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/l2g1qi

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Global Cell and Tissue Analysis Market Research Report 2023 - Benzinga

Editas Medicine Announces First Quarter 2023 Results and … – GlobeNewswire

Company to provide a clinical update on the EDIT-301 Phase 1/2 RUBY trial for SCD in June at the European Hematology Association Congress (EHA) and in a Company-sponsored webinar

On track to dose 20 total patients by year-end in the RUBY trial

First patient in EDIT-301 EDITHAL trial for TDT dosed with successful neutrophil and platelet engraftment; Company on track to provide clinical update by year-end

Appointed Emma Reeve as Chair of the Board, effective at Annual Meeting of Stockholders, and Elliott Levy, M.D. as an Independent Director

CAMBRIDGE, Mass., May 05, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical stage genome editing company, today reported business highlights and financial results for the first quarter 2023.

I am energized by our strong start to the year. We entered 2023 with the objective of accelerating the development of EDIT-301 and positioning Editas as a leader in programable in vivo gene editing. Following on our December EDIT-301 clinical data showing a competitive and potentially differentiated product, we have built considerable momentum with our EDIT-301 program, including dosing and engraftment of the first patient in our EDITHAL trial. We look forward to disclosing the clinical progress of EDIT-301 when we provide a RUBY trial update with safety and efficacy data from multiple patients next month in an oral presentation at the European Hematology Association Congress and in a Company-sponsored webinar. I am pleased with the progress weve made against our strategic plan, commented Gilmore ONeill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine. Alongside our newly sharpened strategic focus are our world-class scientists and employees who are committed to our strategic direction and are building on the momentum from our clinical milestones to date and driving execution towards our goals.

Recent Achievements and Outlook

Ex Vivo Hemoglobinopathies

Business Development & Other Corporate Highlights

Elliott Levy, M.D., appointed to the Editas Board of Directors as an independent directorDr. Levy is an accomplished biopharmaceutical executive with more than 20 years of global research and development expertise, including leading clinical strategy and development for multiple programs at all stages of development at global biopharmaceutical companies Amgen and Bristol Myers Squibb.

Linea Aspesi joined Editas as Chief People OfficerMs. Aspesi brings to Editas more than 25 years experience, including 15 years in the life sciences sector, aligning talent plans to company vision, mission, and values, and partnering with senior leaders to define and drive cultural transformation strategies.

First Quarter 2023 Financial Results

Cash, cash equivalents, and marketable securities as of March 31, 2023, were $401.8 million compared to $437.4 million as of December 31, 2022. The Company expects existing cash, cash equivalents and marketable securities to fund operating expenses and capital expenditures into 2025.

Upcoming Events

Editas Medicine plans to participate in the following scientific and medical conference:

Editas Medicine plans to participate in the following investor events:

Conference CallThe Editas Medicine management team will host a conference call and webcast today at 8:00 a.m. ET to provide and discuss a corporate update and financial results for the first quarter of 2023. To access the call, please dial 1-877-407-0989 (domestic) or 1-201-389-0921 (international) and ask for the Editas Medicine earnings call. A live webcast of the call will also be available on the Investors section of the Editas Medicine website at http://www.editasmedicine.com, and a replay will be available approximately two hours after its completion.

AboutEditas MedicineAs a clinical stage genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute and Harvard Universitys Cas9 patent estates and Broad Institutes Cas12a patent estate for human medicines. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

Forward-Looking StatementsThis press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, target, should, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the initiation, timing, progress and results of the Companys preclinical and clinical studies and its research and development programs, including dosing 20 total patients by year-end in the RUBY trial, the timing for the Companys receipt and presentation of data from its clinical trials and preclinical studies, including a clinical update for the RUBY trial in June 2023 and an additional clinical update by year-end and a clinical update from the EDITHAL trial by year-end, potential of, and expectations for, the Companys product candidates, the timing or likelihood of regulatory filings and approvals, and the Companys expectations regarding cash runway. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of pre-clinical studies and clinical trials, including the RUBY and EDITHAL trials, and clinical development of the Companys product candidates, including EDIT-301; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption Risk Factors included in the Companys most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release represent the Companys views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, the Company explicitly disclaims any obligation to update any forward-looking statements.

EDITAS MEDICINE, INC.Consolidated Statement of Operations(amounts in thousands, except share and per share data)(Unaudited)

EDITAS MEDICINE, INC.Selected Consolidated Balance Sheet Items(amounts in thousands)(Unaudited)

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Editas Medicine Announces First Quarter 2023 Results and ... - GlobeNewswire

Promising Future In Live Cell Imaging Market, Analysis By … – Digital Journal

PRESS RELEASE

Published May 3, 2023

The research elaborates on the growth rate of the Live Cell Imaging Market and examines it using a complete and trustworthy examination of the company profile. The research offers a thorough analysis, market size, share, insights, evaluation for developing segments, and many other important market aspects.

The global live cell imaging market was worth USD 2.35 billion in 2021 and is expected growing at a 9.2% CAGR from 2021 to 2030.

Live cell imaging is a technique used to observe and study biological processes at the cellular level in real-time using microscopy. It allows scientists to visualize and monitor dynamic cellular events such as cell division, migration, and death. The global Holotomography (HT)market is expected to grow significantly in the coming years due to the increasing demand for high-resolution imaging techniques in life sciences research and the development of new drugs.

The live cell imaging market is primarily driven by the increasing demand for advanced imaging techniques in the life sciences industry. The growing need for high-resolution imaging techniques to understand the complex biological processes at the cellular level is expected to boost the market growth. In addition, the development of new drugs and therapies for various diseases, such as cancer, is also driving the demand for Holotomography techniques.

The market is also expected to benefit from technological advancements in live cell imaging systems. The introduction of new imaging techniques such as confocal microscopy, two-photon microscopy, and super-resolution microscopy is expected to increase the adoption of Holotomography systems in research and development activities. However, the high cost of live cell imaging systems and the lack of skilled professionals to operate the systems are expected to restrain the market growth to some extent.

Read Report here: https://www.factualmarketresearch.com/Reports/Live-Cell-Imaging-Market

Key Players

Market Segmentation

Live Cell Imaging Market, Based onProduct

Live Cell Imaging Market, Based onApplication

Live Cell Imaging Market, Based onTechnology

Live Cell Imaging Market, Based on Regions

Report customized for your company: https://www.factualmarketresearch.com/Reports/Live-Cell-Imaging-Market

Brief Table of Contents: Live Cell Imaging Market

Introduction

Market Overview

Technology Overview

Market Segmentation

Competitive Landscape

Future Outlook

Conclusion

Read Detailed TOC here: https://www.factualmarketresearch.com/Reports/Live-Cell-Imaging-Market

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Cellectis Provides Business Update and Reports Financial Results … – GlobeNewswire

NEW YORK, May 04, 2023 (GLOBE NEWSWIRE) -- Cellectis (the Company) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today provided a business update and announced its results for the three-month period ending March 31, 2023.

Cellectis took a notable step forward this quarter with the first patient being dosed in France with our in-house manufactured product candidate UCART22 in the BALLI-01 clinical study. UCART22 is currently the most advanced allogeneic CAR T-cell product in development for relapsed or refractory B-cell acute lymphoblastic leukemia. We believe that our off-the-shelf treatment approach, coupled with our ability to manufacture UCART product candidates entirely in-house, gives us a main advantage on the market: it potentially maximizes the chances for eligible patients to be treated without delay, said Andr Choulika, Ph.D., CEO of Cellectis.

Cellectis also announced last month that it implemented the use of Sanofis alemtuzumab as a Cellectis Investigational Medicinal Product, coded as CLLS52, as part of the lymphodepletion regimen for UCART22 in the BALLI-01 clinical trial, for UCART123 in the AMELI-01 clinical trial, and for UCART20x22 in the NATHALI-01 clinical trial. This follows the partnership and supply agreements we entered with Sanofi regarding alemtuzumab.

This quarter, Cellectis announced the closing of the global offering of 25 million dollars of its Depository Shares, launched in February the net proceeds of the global offering and option of the Company is 22.8 million dollars and in April, the drawdown of the 20 million euros under the Finance Contract for up to 40 million euros credit facility made with the European Investment Bank in December 2022. Cellectis plans to use the net proceeds of the funds to focus on the development of its pipeline of allogeneic CAR T-cell product candidates UCART22, UCART20x22 and UCART123, the Company decided to stop enrollment and treatment of patients with UCARTCS1. Indeed, to accelerate the speed of enrollment of patients in the MELANI-01 study, evaluating UCARTCS1, the Company would have had to invest meaningful amount of resources. To optimize its resources, Cellectis decided to focus its development efforts on the BALLI-01, AMELI-01 and NATHALI-01 studies.

We are excited about the drive in our clinical trials, building on the momentum of our lead product candidates in our pipeline, and the upcoming milestones for 2023.

Pipeline Highlights

UCART Clinical Developments Programs

BALLI-01 (evaluating UCART22) in relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL)

NATHALI-01 (evaluating UCART20x22) in relapsed or refractory B-cell non-Hodgkin lymphoma (r/r NHL)

AMELI-01 (evaluating UCART123) in relapsed or refractory acute myeloid leukemia (r/r AML)

MELANI-01 (evaluating UCARTCS1) in relapsed or refractory multiple myeloma (r/r MM)

Research Data & Preclinical Programs

TALEN-edited MUC1 CAR T-cells

Multiplex engineering for superior generation of efficient CAR T-cells

Licensed Allogeneic CAR T-cell Development Programs

Servier and Allogene: anti-CD19 programs

Allogene continues to enroll patients in the industrys first potentially pivotal Phase 2 allogeneic CAR T clinical trial with ALLO-501A. Allogene announced that the single-arm ALPHA2 trial will enroll approximately 100 r/r large B cell lymphoma (LBCL) patients who have received at least two prior lines of therapy and have not received prior anti-CD19 therapy. Allogene expects to complete enrollment in H1 2024.After the close of the quarter, Allogene announced that pooled data from the Phase 1 ALPHA/ALPHA2 trials of ALLO-501/501A, in r/r LBCL would be presented at the American Society of Clinical Oncology (ASCO) Annual Meeting June 2 6, 2023 in Chicago, Illinois.

Allogene: anti-BCMA and anti-CD70 programs

Partnerships

Cytovia Therapeutics, Inc. (Cytovia)

Corporate Updates

Global offering and American Depositary Shares (ADS)

Calyxt and Cibus Merger Agreement

Warrant agreement with the European Investment Bank

Financial results

The interim condensed consolidated financial statements of Cellectis, which consolidate the results of Calyxt, Inc. of which Cellectis owned approximately 48.2% of outstanding shares of common stock (as of March 31, 2023), have been prepared in accordance with International Financial Reporting Standards, as issued by the International Accounting Standards Board (IFRS).

We present certain financial metrics broken out between our two reportable segments Therapeutics and Plants in the appendices of this Q1 2023 financial results press release.

On January 13, 2023, Calyxt, Cibus Global LLC (Cibus) and certain other parties named therein, entered into an Agreement and Plan of Merger (the Merger Agreement), pursuant to which, subject to the terms and conditions thereof, Calyxt and Cibus will merge in an all-stock transaction (the Calyxt Merger). As a consequence of the foregoing, Calyxt meets the held-for-sale" criteria specified in IFRS 5 and has been classified as a discontinued operation.

Cash: As of March 31, 2023, Cellectis, excluding Calyxt, had $88 million in consolidated cash, cash equivalents, and restricted cash. This compares to $95 million in consolidated cash, cash equivalents and restricted cash as of December 31, 2022. This difference mainly reflects $30 million of cash out, which include $6 million of payments for R&D expenses, $4 million for SG&A suppliers, $15 million for staff costs, $4 million for rents and taxes, $1 million of reimbursement of the PGE loan and a $23 million net cash inflow from the capital raise closed in February.

Based on the current operating plan, Cellectis (excluding Calyxt) anticipates that the cash and cash equivalents as of March 31, 2023 will fund Cellectis operations into the third quarter of 2024.

Revenues and Other Income: Consolidated revenues and other income were $3.6 million for the three months ended March 31, 2023 compared to $3.8 million for the three months ended March 31, 2022. The slight decrease of $0.2 million between the three months ended March 31, 2023, and 2022 reflects the recognition of two milestones related to Cellectis agreement with Cytovia for $1.5million in 2022 while recognition of revenues in 2023 is not material and was almost fully offset by an increase of the research tax credit for $1.0 million in addition to the recognition of a BPI R&D grant of $0.3 million.

R&D Expenses: Consolidated R&D expenses were $21.1 million three months ended March 31, 2023 compared to $26.6 million for the three months ended March 31, 2022. The $5.5 million decrease was primarily attributable to (i) a $2.6 million decrease in personal expenses due to departures not replaced (ii) a $3.0 million decrease in purchases, external expenses and other (from $13.8 million in 2022 to $10.8 million in 2023) mainly explained by internalization of our manufacturing and quality activities to support our R&D pipeline.

SG&A Expenses: Consolidated SG&A expenses were $5.0 million for the three months ended March 31, 2023 compared to $6.1 million for the three months ended March 31, 2022. The $1.1 million decrease primarily reflects (i) a $0.9 million decrease in purchases, external expenses and other (from $3.7 million in 2022 to $2.9 million in 2023) mainly explained by the implementation of our new enterprise resource planning (ERP) software in 2022 (ii) a $0.2 million decrease in personal expenses.

Net income (loss) from discontinued operations: The $1.7 million decrease of net loss from discontinued operations between the three-month period ended March 31, 2022 and 2023 is primarily driven by (i) the decrease of $2.6 million of R&D expenses (from $3.2 million in 2022 to $1.3 in 2023) and SG&A expenses (from $2.9 million in 2022 to $2.2 million in 2023) partially offset by (i) the increase of $0.7 million of net financial loss and (ii) the increase of $0.2 million of other operating expenses.

Net Income (loss) Attributable to Shareholders of Cellectis including Calyxt: The consolidated net loss attributable to shareholders of Cellectis was $30.1 million (or $0.58 per share) for the three months ended March 31, 2023, of which $27.8 million was attributed to Cellectis continuing operations, compared to $31.9 million (or $0.70 per share) for the three months ended March 31, 2022, of which $28.3 million was attributed to Cellectis continuing operations. This $1.8 million decrease in net loss between the three months of 2023 and 2022 was primarily driven by (i) a $5.3 million decrease of research and development, (ii) a decrease of $1.7 million of loss from discontinued operations, (iii) a $1.3 million decrease of SG&A expenses partially offset by (i) an increase in net financial loss of $5.3 million primarily due to the decrease of the fair value of Cytovias convertible note on March 31, 2023 of $4.6 million compared to a $7.9 million on December 31, 2022, (ii) a decrease of $0.2 million of revenues and other income, (iii) an increase of other operating expenses of $0.6 million, (iv) a decrease of $0.4 million in loss attributable to non-controlling interests due to the decrease in Calyxts net loss.

Adjusted Net Income (Loss) Attributable to Shareholders of Cellectis: The consolidated adjusted net loss attributable to shareholders of Cellectis was $28.1 million (or $0.55 per share) for the three months ended March 31, 2023, of which $26.2 million is attributed to Cellectis, compared to a net loss of $29.3 million (or $0.64 per share) for the three months ended March 31, 2022, of which $26.0 million was attributed to Cellectis.

Please see Note Regarding Use of Non-IFRS Financial Measures for reconciliation of GAAP net income (loss) attributable to shareholders of Cellectis to adjusted net income (loss) attributable to shareholders of Cellectis.

We currently foresee focusing our cash spending at Cellectis for 2023 in the following areas:

* These amounts reflect adjustments made in connection with the presentation of the discontinued operation

Note Regarding Use of Non-IFRS Financial Measures

Cellectis S.A. presents adjusted net income (loss) attributable to shareholders of Cellectis in this press release. Adjusted net income (loss) attributable to shareholders of Cellectis is not a measure calculated in accordance with IFRS. We have included in this press release a reconciliation of this figure to net income (loss) attributable to shareholders of Cellectis, which is the most directly comparable financial measure calculated in accordance with IFRS. Because adjusted net income (loss) attributable to shareholders of Cellectis excludes Non-cash stock-based compensation expensea non-cash expense, we believe that this financial measure, when considered together with our IFRS financial statements, can enhance an overall understanding of Cellectis financial performance. Moreover, our management views the Companys operations, and manages its business, based, in part, on this financial measure. In particular, we believe that the elimination of Non-cash stock-based expenses from Net income (loss) attributable to shareholders of Cellectis can provide a useful measure for period-to-period comparisons of our core businesses. Our use of adjusted net income (loss) attributable to shareholders of Cellectis has limitations as an analytical tool, and you should not consider it in isolation or as a substitute for analysis of our financial results as reported under IFRS. Some of these limitations are: (a) other companies, including companies in our industry which use similar stock-based compensation, may address the impact of Non-cash stock- based compensation expense differently; and (b) other companies may report adjusted net income (loss) attributable to shareholders or similarly titled measures but calculate them differently, which reduces their usefulness as a comparative measure. Because of these and other limitations, you should consider adjusted net income (loss) attributable to shareholders of Cellectis alongside our IFRS financial results, including Net income (loss) attributable to shareholders of Cellectis.

*These amounts reflect adjustments made in connection with the presentation of the discontinued operation

About Cellectis

Cellectis is a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies. Cellectis utilizes an allogeneic approach for CAR-T immunotherapies in oncology, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients, and a platform to make therapeutic gene editing in hemopoietic stem cells for various diseases. As a clinical-stage biopharmaceutical company with over 23 years of experience and expertise in gene editing, Cellectis is developing life-changing product candidates utilizing TALEN, its gene editing technology, and PulseAgile, its pioneering electroporation system to harness the power of the immune system in order to treat diseases with unmet medical needs. Cellectis headquarters are in Paris, France, with locations in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). For more information, visit http://www.cellectis.com. Follow Cellectis on social media: @cellectis, LinkedIn and YouTube

Forward-looking Statements

This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by words such as anticipate, believe, intend, expect, plan, scheduled, could, would and will, or the negative of these and similar expressions. These forward-looking statements, which are based on our managements current expectations and assumptions and on information currently available to management, including information provided or otherwise publicly reported by our licensed partners. Forward-looking statements include statements about advancement, timing and progress of clinical trials (including with respect to patient enrollment and follow-up), the timing of our presentation of data and submission of regulatory filings, the adequacy of our supply of clinical vials, the operational capabilities at our manufacturing facilities, the sufficiency of cash to fund operations, the adequacy and continuity of supply of clinical supply and alemtuzumab, the ability of an anti-CD52 as alemtuzumab to improve any efficacy and the potential benefit of UCART product candidates. These forward-looking statements are made in light of information currently available to us and are subject to numerous risks and uncertainties, including with respect to the numerous risks associated with biopharmaceutical product candidate development. With respect to our cash runway, our operating plans, including product development plans, may change as a result of various factors, including factors currently unknown to us. Furthermore, many other important factors, including those described in our Annual Report on Form 20-F and the financial report (including the management report) for the year ended December 31, 2022 and subsequent filings Cellectis makes with the Securities Exchange Commission from time to time, as well as other known and unknown risks and uncertainties may adversely affect such forward-looking statements and cause our actual results, performance or achievements to be materially different from those expressed or implied by the forward-looking statements. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.

For further information, please contact:

Media contact:

Pascalyne Wilson, Director, Communications, +33 (0)7 76 99 14 33, media@cellectis.com

Investor Relation contacts:

Arthur Stril, Chief Business Officer, +1 (347) 809 5980, investors@cellectis.com

Ashley R. Robinson, LifeSci Advisors, +1 617 430 7577

1 Cash position includes cash, cash equivalents and restricted cash. Restricted cash was $5 million as of March 31, 2023.

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Cellectis Provides Business Update and Reports Financial Results ... - GlobeNewswire

An inducible model for genetic manipulation and fate-tracing of … – Nature.com

The role of mesenchymal cell populations in the development of liver fibrosis has been extensively studied in the last decades15,16,17,18,19, but oftentimes interpretation of the results is limited by the models used.

Both the Lrat- and the PDGFR promoters were shown to label HSCs in the context of liver fibrosis of different etiologies3,7. However, both the previously described LratCre and PDGFR-Cre reporter mice have a constitutively active Cre recombinase, which limits their use. This can result in unspecific activity if the promoter is only temporarily active in the course of cell differentiation, but not specific for the differentiated cell20. Furthermore, the time point of Cre activity can be a concern once a specific deletion results in cell lethality during early development.

Those concerns could be addressed by using a transgenic mouse model with inducible Cre expression. However, no such model that targets mesenchymal cell populations has been published so far. We therefore investigated whether the inducible PDGFR-P2A-CreERT2 mouse11 can be used as a reliable tool to express transgens in mesenchymal cell populations in the liver.

To address this question, we generated a triple transgenic mouse model containing the PDGFR-P2A-CreERT211, a red fluorescent tdTomato Cre reporter21, and a Col1a1-driven GFP22. Our data revealed that tamoxifen-induced activation of PDGFR-P2A-CreERT2 efficiently induced reporter gene expression in pericytes of the liver, which lasted up to one year after activation. Experiments with vehicle-treated mice showed practically no reporter expression, demonstrating no significant leakiness of the PDGFR-P2A-CreERT2 construct which has been observed for other CreER transgenic mice, such as the RipCreER, a beta-cell specific mouse line that can be used to manipulate gene expression in insulin-producing cells of the endocrine pancreas23. PDGFR-P2A-CreERT2 induced reporter expression was tested via immunostaining for markers of different liver resident cell types, in which fluorescent reporter expression only overlapped with desmin, a marker commonly used to stain pericytes in different organs, including HSCs3,24,25,26. Staining for the portal fibroblast marker Thy1.2 revealed overlap with PDGFR-P2A-CreERT2 induced reporter expression, which is in line with previous data showing that fibroblasts and VSMC express Thy1 to a certain extent5. It has been suggested that HSCs and Thy1.2 positive cells are two distinct cell populations13,27, however it cannot be excluded that also some HSCs express Thy15.

Furthermore, PDGFR-P2A-CreERT2 induced reporter expression remained specific for mesenchymal cells even under fibrogenic conditions in the CCl4 toxic liver fibrosis model. Overlap of alpha smooth muscle actin, a common myofibroblast marker, and overlap with endogenous collagen 1a1 driven GFP further confirmed that fibrogenic cells in the liver are PDGFR-P2A-CreERT2 derived. As we achieve a high recombination of PDGFR-P2A-CreERT2 in retinoid positive HSCs of over 90% and a similarly high percentage of pericyte derived myofibroblasts in three different liver fibrosis models, the inducible PDGFR-P2A-CreERT2 model can be used once an inducible Cre mouse model for liver mesenchymal cell populations is required with a similar efficiency as the constitutive PDGFRCre7 or the well accepted LratCre transgenic mouse model3.

However, PDGFRCre as a marker for fibrogenic cells in the liver has some limitations. In recent years, single cell RNA sequencing studies using a Pdgfrb-GFP transgenic mouse have revealed a spatial zonation of HSCs with central-vein-associated HSCs and portal vein-associated HSCs, whereby central-vein-associated HSCs were the dominant collagen-producing cells in CCl4 induced toxic liver injury5. Without other markers, PDGFRCre cannot distinguish between these different HSC populations with distinct functions and the different PDGFR-positive cell populations including fibroblasts, HSCs and VSMC5. Furthermore, another study identified several clusters of fibroblasts in the liver, with some of them (Fib-3 and Fib-4 clusters) expressing low levels of Pdgfrb and thus being underestimated in studies using Pdgfrb as a promoter for Cre recombinases or GFP14. We also performed immunohistochemistry for Slit2, a marker for portal fibroblasts with mesenchymal stem cell features (PMSCs)14. In contrast to this study, we observed Slit2 expression not only restricted to the portal area, but also in the liver parenchmya. Of note, PDGFR-P2A-CreERT2 driven tdTomato expression overlapped with Slit2, both in normal and fibrotic liver indicating that Slit2 might not only be a precursor for PMSCs but also for HSCs. Further studies need to address this finding.

Nevertheless, our data demonstrates that the tamoxifen inducible PDGFR-P2A-CreERT2 mouse model is similarly efficient to the established constitutive LratCre and PDGFR-Cre mouse models and can be applied once an inducible Cre recombinase is required to study liver fibrogenesis.

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An inducible model for genetic manipulation and fate-tracing of ... - Nature.com

Global Hematological Cancers Therapeutics Market to Reach $122.75 Billion by 2032, Driven by Rising Prevalence – EIN News

The global hematological cancers therapeutics market size was USD 66.77 Billion in 2022 and is expected to reach USD 122.75 Billion in 2032

Although the development of novel treatments is leading to increased demand for hematological cancer therapies, the high cost of medicines, negative effects of therapies, and availability of alternative treatments remain major challenges to the growth of the market. Despite these challenges, rising R&D investments by government entities, commercial businesses, and academic institutions are expected to drive revenue growth.

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Segments Covered in the Report:

The global therapy market is expected to witness significant growth in the coming years. The therapy market is categorized into different types, including chemotherapy, targeted therapy, immunotherapy, and stem cell transplantation. The revenue generated by each therapy type is expected to increase from USD billion in 2019 to USD billion by 2032. Chemotherapy is a type of cancer treatment that uses drugs to destroy cancer cells. It is one of the most common cancer treatments and is expected to contribute significantly to the therapy market's revenue. The increasing incidence of cancer worldwide is a major driving factor for the growth of the chemotherapy market.

Targeted therapy is a type of cancer treatment that targets specific genes or proteins that contribute to cancer cell growth. It is expected to witness significant growth in the coming years, owing to its increasing effectiveness in cancer treatment. Immunotherapy is a type of cancer treatment that uses the body's immune system to fight cancer cells. It is a relatively new type of cancer treatment and is expected to witness significant growth in the coming years, owing to its potential to treat a wide range of cancers. Stem cell transplantation is a type of cancer treatment that involves replacing diseased bone marrow with healthy bone marrow stem cells. It is primarily used to treat blood cancers such as leukemia, lymphoma, and multiple myeloma. The increasing incidence of these types of cancers is expected to contribute significantly to the growth of the stem cell transplantation market.

The therapy market is also categorized based on the indication, including leukemia, lymphoma, and multiple myeloma. The revenue generated by each indication is expected to increase from USD billion in 2019 to USD billion by 2032. Leukemia is a type of blood cancer that affects the bone marrow and blood cells. The increasing incidence of leukemia worldwide is a major driving factor for the growth of the leukemia market. Lymphoma is a type of cancer that affects the lymphatic system. The increasing incidence of lymphoma worldwide is a major driving factor for the growth of the lymphoma market. Multiple myeloma is a type of blood cancer that affects the plasma cells in the bone marrow. The increasing incidence of multiple myeloma worldwide is a major driving factor for the growth of the multiple myeloma market.

The therapy market is also categorized based on the regional outlook, including North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa. The revenue generated by each region is expected to increase from USD billion in 2019 to USD billion by 2032.

North America is expected to dominate the therapy market owing to the increasing incidence of cancer and the high cost of cancer treatment in the region. Europe is also expected to witness significant growth owing to the increasing investment in cancer research and development. The Asia Pacific region is expected to witness significant growth owing to the increasing healthcare infrastructure and the rising prevalence of cancer in the region. Latin America and the Middle East and Africa are also expected to witness significant growth owing to the increasing government initiatives and investments in the healthcare sector.

Strategic Development:

Strategic advancements in the healthcare industry include significant developments in various drug treatments for different types of cancers. AbbVie Inc. declared positive outcomes from a Phase 3 clinical trial in September 2021 that assessed the drug, VENCLYXTO (venetoclax), in combination with a Hypomethylating Agent (HMA) to treat newly diagnosed Acute Myeloid Leukemia (AML) in elderly patients or those ineligible for intensive chemotherapy.

AstraZeneca plc announced that its drug, CALQUENCE (acalabrutinib), was granted regulatory approval by the European Commission in May 2021 for the treatment of adults with chronic lymphocytic leukemia (CLL). Additionally, Bristol-Myers Squibb Company stated that its drug, REBLOZYL (luspatercept-aamt), was granted regulatory approval by the European Commission in March 2021 to treat anemia in adults with beta-thalassemia who need regular Red Blood Cell (RBC) transfusions.

Furthermore, in November 2020, GlaxoSmithKline plc partnered with iTeos Therapeutics Inc. to develop and market iTeos's cancer immunotherapy, EOS-448, together with GlaxoSmithKline's hematological cancer therapies.

Access Full Report Description with Research Methodology and Table of Content @ https://www.reportsanddata.com/report-detail/hematological-cancers-therapeutics-market

Competitive Landscape:

The global hematological cancers therapeutics market is highly competitive, with several key players operating in the industry. These major players are AbbVie Inc., Amgen Inc., AstraZeneca plc, Bristol-Myers Squibb Company, Celgene Corporation, F. Hoffmann-La Roche Ltd., GlaxoSmithKline plc, Johnson & Johnson, Novartis International AG, and Pfizer Inc. AbbVie Inc. is a leading player in the global hematological cancers therapeutics market, with a strong portfolio of products, including Imbruvica and Venclexta. Amgen Inc. is another major player in the industry, offering a range of products for the treatment of hematological cancers, including Neulasta, Kyprolis, and Blincyto.

AstraZeneca plc is a multinational pharmaceutical company that offers a range of drugs for the treatment of hematological cancers, including Calquence and Lumoxiti. Bristol-Myers Squibb Company is another key player in the market, offering a portfolio of products for the treatment of hematological cancers, including Opdivo and Empliciti. Celgene Corporation is a biotechnology company that focuses on the discovery, development, and commercialization of therapies for the treatment of cancer and other diseases. Its portfolio of products for the treatment of hematological cancers includes Revlimid and Pomalyst.

Hoffmann-La Roche Ltd. is a multinational healthcare company that offers a range of products for the treatment of hematological cancers, including Rituxan and Gazyva. GlaxoSmithKline plc is another major player in the market, offering a portfolio of products for the treatment of hematological cancers, including Zejula and Arzerra. Johnson & Johnson is a multinational healthcare company that offers a range of products for the treatment of hematological cancers, including Darzalex and Imbruvica. Novartis International AG is another key player in the market, offering a portfolio of products for the treatment of hematological cancers, including Kymriah and Tasigna.

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Pfizer Inc. is a multinational pharmaceutical company that offers a range of products for the treatment of hematological cancers, including Besponsa and Mylotarg. These major players are expected to continue to dominate the global hematological cancers therapeutics market in the coming years, owing to their strong product portfolio and focus on innovation and research and development.

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Global Hematological Cancers Therapeutics Market to Reach $122.75 Billion by 2032, Driven by Rising Prevalence - EIN News

Recombinant proteins market is projected to grow at a CAGR of … – Benzinga

Visiongain has published a new report:Recombinant Proteins Market: Forecasts by Products & Services (Cytokines & Growth Factors (Interferons (IFNs), Interleukins (ILS), Others), Antibodies, Enzymes (Kinases Enzymes, Metabolic Enzymes, Others), Recombinant Regulatory Protein, Hormones, Virus Antigens, Immune Checkpoint Proteins, Others)), Services), by Application (Biopharmaceutical Production (Biologics, Vaccines, Cell & Gene Therapy, Others), Drug Discovery & Development, Research, Diagnostics, Others)), by Host Cell (Mammalian Systems, Bacterial Cells, Yeast & Fungi, Insect Cells, Others), by End-users (Biopharmaceutical and Biotechnology Companies, CROs, Academic & Research Institutes, Diagnostic Laboratories, Others), AND Regional and Leading National Market Analysis PLUS Analysis of Leading Companies AND COVID-19 Impact and Recovery Pattern Analysis.

The recombinant proteins market was valued at US$3,079.9 million in 2023 and is projected to grow at a CAGR of 11.14% during the forecast period 2023-2033.

The field of biologics within biotechnology has been recognised to be rapidly growing. Diabetes, autoimmune diseases, and cancer therapies account for more than 60% of the biologics industry. As a result, the cost is exorbitant and growing on a regular basis. According to the World Health Organisation (WHO), these high prices impede healthcare systems from providing affordable, universal access to such treatments. This creates a slew of opportunities for generic drugmakers to create biosimilar copies of original biologics whose patents have lapsed. As a result, generic medication manufacturers are continuously on the lookout for biologics with expiring labels. There will be 230 medicinal patents expiring between 2021 and 2025. PERJETA (Pertuzumab), VYROLOGIX (Leronlimab), and STELARA (Ustekinumab) are just a handful of the biologics that will be phased out in 2023. Biologics already account for half of the cancer pharmaceutical business, according to the Centre for Biosimilars, but their high price is a barrier to access. By 2023, around 20 oncology biologics' patents will have expired, potentially resulting in more biosimilars being utilised in cancer therapy and lower overall prices.

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Recombinant Proteins Market Report 2023-2033

How has COVID-19 had a Significant Impact on the Recombinant Proteins Market?

The growth of the recombinant protein market was impacted by the COVID-19 pandemic. The advent of COVID-19 has led to a surge in the need for therapeutic and vaccination interventions to counteract its effects. Numerous market participants and public institutions have redirected their attention towards research and development endeavours aimed at addressing COVID-19, with recombinant proteins serving as a crucial component in these initiatives. An article published in the JCLA journal in May of 2022 details how researchers and scientists focused their efforts on recombinant proteins as a means of developing a COVID-19 vaccine. The outbreak of COVID-19 has resulted in an increase in the need for pharmaceuticals based on recombinant proteins. The present circumstances suggest that the studied market is likely to experience substantial growth during the projected timeframe, owing to the widespread occurrence of chronic ailments and coexisting infectious diseases, as well as the effectiveness of recombinant protein-based medication in addressing them.

How will this Report Benefit you?

Visiongain's 358-page report provides 146 tables and 248 charts/graphs. Our new study is suitable for anyone requiring commercial, in-depth analyses for the recombinant proteins market, along with detailed segment analysis in the market. Our new study will help you evaluate the overall global and regional market for Recombinant Proteins. Get financial analysis of the overall market and different segments including product & services, application, cell source, and end-user, and capture higher market share. We believe that there are strong opportunities in this fast-growing recombinant proteins market. See how to use the existing and upcoming opportunities in this market to gain revenue benefits in the near future. Moreover, the report will help you to improve your strategic decision-making, allowing you to frame growth strategies, reinforce the analysis of other market players, and maximise the productivity of the company.

What are the Current Market Drivers?

Growing Demand for Vaccine Production

Recombinant proteins are required for the development and production of vaccines. The rapid development of the COVID-19 vaccine, as well as the amount of immunisation demand, is unprecedented in modern medicine. To accommodate this demand, stakeholders have had to rethink their manufacturing methods, make large investments, repurpose existing facilities, and develop sophisticated supply networks. The R&D reaction to COVID-19 has been spectacular. This required a far faster ramp-up of manufacturing than is customary in pharmaceutical development, a difficulty exacerbated by the global nature of vaccine demand.

Emerging Economies and Prioritisation of R&D to Boost Industry Growth

Some of the most rapidly rising economies in the pharmaceutical sector are Brazil, Russia, India, China, South Africa, Mexico, South Korea, Indonesia, and Turkey. In five years, pharmaceutical sales in these countries more than doubled, obtaining a market share of roughly 20% in the worldwide pharma industry. Furthermore, firms are dealing with flattening growth in developed nations, patent expiration, which leads to increased sales of less expensive generic drugs, and strict regulations enforced in mature markets.

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Recombinant Proteins Market Report 2023-2033

Where are the Market Opportunities?

Rising Significance of Genomics and Proteomics

Proteins perform critical roles in biological systems. A better knowledge of illnesses, health, and, by extension, health outcomes is increasingly seen as a difficult, but necessary, precondition. The recent spread of genome sequencing, powered by a steep decline in sequencing costs, has propelled the rapid rise in the identification of possible genetic targets and associated protein biomarkers for sickness diagnosis and more personalised therapies. To capitalise on this market potential in protein research, the recombinant protein market is seeing high rates of innovation and capital investment in support of several innovative solutions to uncover and analyse protein biomarkers.

Competitive Landscape

The major players operating in the recombinant proteins market are Abcam Plc; Abnova Corporation; Avantor Inc.; Bio-Rad Laboratories, Inc.; Bio-Techne; Enzo Biochem Inc.; GenScript; Merck KGaA; PerkinElmer, Inc; Proteintech Group, Inc.; RayBiotech Life, Inc.; Sino Biological Inc.; STEMCELL Technologies; and Thermo Fisher Scientific Inc. These market's top players have pursued a variety of strategies, including M&A, R&D investment, collaborations, partnerships, regional company development, and new product launches.

Recent Developments

To access the data contained in this document please emailoliver.davison@visiongain.com

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Do you have any custom requirements we can help you with?Any need for a specific country, geo region, market segment or specific company information? Contact us today, we can discuss your needs and see how we can help:oliver.davison@visiongain.com

About Visiongain

Visiongain is one of the fastest-growing and most innovative independent market intelligence providers around, the company publishes hundreds ofmarket research reportswhich it adds to its extensive portfolio each year. These reports offer in-depth analysis across 18 industries worldwide. The reports, which cover 10-year forecasts, are hundreds of pages long, with in-depth market analysis and valuable competitive intelligence data. Visiongain works across a range of vertical markets with a lot of synergies. These markets include automotive, aviation, chemicals, cyber, defence, energy, food & drink, materials, packaging, pharmaceutical and utilities sectors. Our customised and syndicatedmarket research reportsoffer a bespoke piece of market intelligence customised to your very own business needs.

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Recombinant proteins market is projected to grow at a CAGR of ... - Benzinga

Citius Pharmaceuticals Announces $15 Million Registered Direct … – PR Newswire

CRANFORD, N.J., May 4, 2023 /PRNewswire/ -- Citius Pharmaceuticals Inc. (Nasdaq: CTXR) ("Citius" or the "Company"), a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products, today announced that it has entered into definitive agreements with certain healthcare-focused and institutional investors for the purchase of an aggregate of 12,500,001 shares of its common stock and accompanying warrants to purchase up to an aggregate of 12,500,001 shares of its common stock, at a purchase price of $1.20 per share and accompanying warrant in a registered direct offering. The closing of the offering is expected to occur on or about May 8, 2023, subject to the satisfaction of customary closing conditions.

H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

The warrants have an exercise price of $1.50 per share, will be exercisable six months from the date of issuance, and will expire five years from the date of issuance.

The aggregate gross proceeds to the Company from the offering are expected to be approximately $15 million, before deducting the placement agent fees and other offering expenses payable by the Company. Citius currently intends to use the net proceeds from the offering for general corporate purposes, including pre-clinical and clinical development of our product candidates and working capital and capital expenditures.

The securities described above are being offered pursuant to a "shelf" registration statement (File No. 333-255005) filed with the Securities and Exchange Commission (SEC) and declared effective on April 16, 2021. The offering is being made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and the accompanying prospectus relating to the securities being offered will be filed with the SEC and be available at the SEC's website at http://www.sec.gov. Electronic copies of the final prospectus supplement and the accompanying prospectus relating to the securities being offered may also be obtained, when available, by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rdFloor, New York, NY 10022, by calling (646) 975-6996 or emailing [emailprotected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

About Citius Pharmaceuticals, Inc.

Citius is a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products, with a focus on oncology, anti-infectives in adjunct cancer care, unique prescription products, and stem cell therapies. For more information, please visit http://www.citiuspharma.com.

Forward Looking Statements

This press release may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements are made based on our expectations and beliefs concerning future events impacting Citius. You can identify these statements by the fact that they use words such as "will," "anticipate," "estimate," "expect," "should," and "may" and other words and terms of similar meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price, and includes all statements related to the completion of the registered direct offering, the satisfaction of customary closing conditions related to the registered direct offering and the intended use of net proceeds from the registered direct offering. Factors that could cause actual results to differ materially from those currently anticipated, include, without limitation: risks related to the closing of the offering; market and other conditions; our ability to successfully undertake and complete clinical trials and the results from those trials for our product candidates; our need for substantial additional funds; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; the early stage of products under development; the estimated markets for our product candidates and the acceptance thereof by any market; risks related to our growth strategy; patent and intellectual property matters, our ability to attract, integrate, and retain key personnel; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; our ability to identify, acquire, close and integrate product candidates and companies successfully and on a timely basis; our dependence on third-party suppliers; government regulation; 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.

Investor Contact:Ilanit Allen[emailprotected]908-967-6677 x113

Media Contact:STiR-communicationsGreg Salsburg[emailprotected]

SOURCE Citius Pharmaceuticals, Inc.

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Citius Pharmaceuticals Announces $15 Million Registered Direct ... - PR Newswire

HKUs innovative research novelties excel at – EurekAlert

image:Artificial Intelligence MGF Network for Anomalies Detection, developed by Dr Wilton Fok's team, wins two special grand prizes Invention & Innovation CAI Award (China Delegation) and Prize of the Delegation of Malaysia, and a gold medal view more

Credit: The University of Hong Kong

The University of Hong Kong (HKU) triumphed at the 48th International Exhibition of Inventions of Geneva, winning a total of 19 awards, including two special grand prizesInvention & Innovation CAI Award (China Delegation), andPrize of the Delegation of Malaysia.The results were announced yesterday (April 28).

Research teams from Faculty of Architecture, Faculty of Engineering, Faculty of Science,LKS Faculty of Medicine, and two HKU Inno Laboratories, established under the Hong Kong Government's InnoHK programme,garnered two special grand prizes, oneGold Medal with the Congratulations of the Jury, sixGold Medals, sixSilver Medals and fourBronze Medals with their innovations in the event.

TheArtificial Intelligence MGF Network for Anomalies Detection,developed byDr Wilton Fok's team from the Department of Electrical and Electronic Engineering, was winner of the two grand prizes and a gold medal. The system usesAI to analyse in real time human posture and movements in a video to identify anomaly scenarios such as abuse, drowning, and criminal behaviour.

TheProgrammable Cell Niche Engineering Platform,developed byProfessor Barbara Chan's team from the Department of Mechanical Engineering, wonaGold Medal with the Congratulations of the Jury. The system ensures proper cellular functions and results that can enable predictable clinical studies by mimicingthe biological environment for cells.

HKU has showcased this year a total of 17 research inventions that provide solutions to pressing human problems, spanning diverse areas such as biomedicine, vaccine development, artificial intelligence, and aging. Notably, the Advanced Biomedical Instrumentation Centre and the Centre for Virology, Vaccinology and Therapeutics, established by HKU in 2021 under the Hong Kong Government's InnoHK program, have demonstrated exceptional ability in successfully translating their biomedical research achievements into practical applications within a relatively short timeframe. Each have two research inventions selected, including an aptasensor for sepsis diagnosis, a capillary microfluidic platform for point-of-care testing, an interferon-integrated coronavirus vaccine, and NanoComplex vaccine technology.

HKU takes immense pride in the recognition our researchers have received for their innovative research efforts. We are also dedicated to facilitating the transfer of our research findings into tangible applications in the market. Through the powerful synergies generated from our collaborations with global research and industry partners, we strive to facilitate the emergence of pioneering innovations and their transformation into products that improve the well-being of humanity, said Professor Max Shen, Vice-President and Pro-Vice-Chancellor (Research).

Renowned as one of the most distinguished innovation exhibitions in the world, the International Exhibition of Inventions of Geneva (IEIG) is an extraordinary annual global event devoted exclusively to inventions and innovations.

Since first participating in the event in 2018, HKU has received a total of 60 awards including 4 prestigious Gold Medals with Congratulations of the Jury and 21 other gold medals.

HKUs award-winning inventions in 2023:

INVENTION & INNOVATION CAI AWARD (China Delegation) & PRIZE OF THE DELEGATION OF MALAYSIA & GOLD MEDAL

Artificial Intelligence MGF Network for Anomalies DetectionThe invention can be deployed in children's centres, swimming pools, public transport and exhibition centres to detect abnormal behaviours and situations for public safety and security. Using AI to detect human skeletons in a video and analyse their posture and movements, it is able to capture the context information in a long video and address specific portions for detecting multiple anomaly scenarios in real time such as abuse, drowning, terrorist attacks, traffic accidents, fighting and criminal behaviour, more accurately and efficiently.

The invention was developed by the research team of Dr Wilton Fok from the Department of Electrical and Electronic Engineering, The University of Hong Kong.

GOLD MEDALS WITH CONGRATULATIONS OF THE JURY

Programmable Cell Niche Engineering PlatformOne of the reasons many drugs that work in the lab do not work as expected in the clinical trial stage is that scientists grow cells on flat and rigid culture dishes for drug research, which are not biomimetic -- leading to cell responses in lab testing that do not represent what would happen in native tissues of human bodies. The Programmable Cell Niche Engineering Platform mimics the biological environment for cells, which in turn ensures proper cellular functions and results that can enable predictable clinical studies. The multifactorial biochips invented identify the ideal niche for different cell types, equipping scientists with a comprehensive library of individual niche factors to develop optimal culture environments for different types of cells of specialised applications.

The platform was developed by Professor Barbara Chan and her team from the Department of Mechanical Engineering, Biomedical Engineering Programme, The University of Hong Kong.

GOLD MEDALS

1. Respiratory OrganoidsBiomedical research mainly relies on cell lines and animal models for experimentation, which has limited physiological relevance to human biology and pathology. Under this project, a complete respiratory organoid culture system has been the first of its kind to efficiently reconstruct and reproducibly expand the entire human respiratory epithelium in culture plates. All the differentiated respiratory organoids, including nasal, airway and alveolar organoids, adequately mimic the multicellular composition of native respiratory epithelium and phenocopy the functionality. Superior to most existing in vitro models, these organoids represent universal tools with biological relevance for diverse biomedical and pharmaceutical applications, including disease modeling, development of therapeutics, toxicological evaluation and personalised medicine.

The project was originated by Dr Jie Zhou, Dr Man Chun Chiu, Dr Cun Li and Professor Kwok Yung Yuen from the Department of Microbiology, The University of Hong Kong.

2. Bismuth-based Cocktail Therapy for CoronavirusThere is an urgent need for safe and effective therapeutic options for COVID-19, with the SARS-CoV-2 variants associated with enhanced transmissibility, reduced vaccine-induced protection and emerging drug resistance. Under this project, the orally administrated bismuth drug colloidal bismuth subcitrate together with N-acetyl cysteine has been found to be a broad-spectrum anti-coronavirus cocktail therapy against multiple SARS-CoV-2 variants and different coronaviruses. Mixing two US Food and Drug Administration (FDA) approved over counter drugs, 1 molar equivalent of colloidal bismuth subcitrate and 3 molar equivalents of a mucoactive drug N-acetyl cysteine, is a safe and highly potent therapy through oral administration and 10 times more selective in killing the virus than the first FDA approved drug. This therapy is now in phase II/III clinical trial in Hong Kong, with a phase III trial underway in China.

The invention was developed by Professor Sun Hongzhe, Professor Yuen Kwok-yung, Dr Yuan Shuofeng, Dr Wang RunMing, Dr Li Hongyan, Dr Jasper Chan Fuk-Woo, Dr Cheng Tianfan, Mrs Wang SuYu, and Mr Chan Chun Lung of The University of Hong Kong.

3. PERfECT Wearables for Digital HealthEndorsed by over 100 leading scientists worldwide on social media, "HKU PERfECT" is the first wearable platform combining the distinctive features of being highly sensitive, energy efficient, lightweight and ultracompact, enabling decentralised medical technologies for remote and personalised healthcare to cope with an aging population, quarantine and the shortage of medical instrumentation. Compared to current wearable devices that can only measure vital signals (e.g. heart rate, breathing rate, blood pressure, etc.), PERfECT is able to detect molecular indicators in body fluids, which are more informative for fatal/chronic disease screening and monitoring.

The invention was developed by the WISE Research Group from The University of Hong Kong and SESIC LIMITED.

4. Method of Developing a Peptide-based Vaccine Conjugated with 1V209This invention describes the development of a method to conjugate 1V209, a toll-like receptor 7 (TLR7) agonist, to antigens to induce antigen-specific responses. Past synthetic peptide vaccines must be used together with vaccine adjuvants to stimulate T cells against specific antigens to treat cancer, but a simple mixture of antigens and adjuvants can hardly induce enough specific immune responses. To address this issue, we designed bioinspired nanoparticles mimicking the structure of the virus to enhance the antigen-specific immune response. Through safer and greener methods, it used the TLR7 agonist 1V209 to generate peptide-drug conjugates and form a self-adjuvating vaccine to deliver antigens and adjuvants at the same time, inducing a stronger T cell response with improved stability and successful suppression of tumour growth.

The invention was developed by Professor Jiandong Huang and Dr Yefan Hu from the School of Biomedical Sciences, The University of Hong Kong.

5. DipChip Capillary Microfluidic Platform for Point-of-care DiagnosticsDipChip is an automated capillary microfluidic-based point-of-care (POC) microsystem allowing rapid and portable detection of various high-impact and mortality diseases. It is designed and fabricated using state-of-the-art molecular biology and microfluidic technologies, utilising capillary pressure and surface-activating treatments that allow laboratory-level analytics to be conducted in an accessible manner. This microsystem paves the way for a versatile array of clinical and academic applications that require multiple conjugations and washings. End users of DipChip include clinics, hospitals, homes and assisted living healthcare facilities, democratising access to adequate clinical care, and saving precious lives of patients in need.

The platform was developed by Professor Anderson Shum, Dr. Hassan Sammer Ul and Mr. Nicky Lee from Department of Mechanical Engineering and Advanced Biomedical Instrumentation Centre.

SILVER MEDALS

1. Smart Address Plates for Pedestrian Indoor Navigation and Location-Based Services and ManagementTraditionally, pedestrian navigation uses Location Positioning System (LPS) with trilateration to find the users position, which have very high positioning error for outdoor GPS and indoor positioning. This new solution adopts a cost-effective innovative Location Confirmation System (LCS) to accurately locate and guide the user to the destination by using Smart Address Plates (SAP) that transmit stored geographic coordinates with innovative 3D Smart Address (SA) codes to the users even without WiFi or telephone signals. This SAP system can help to find shops/ offices/ restaurants/ car parking spaces inside a multi-storey building accurately while providing location-based services and management for precise target marketing. It is highly scalable, connecting shops/rooms on a floor to a building, then to a district and to the whole city through a Smart Address Plate Management System (SAP-MS).

The Smart Address Plates and Location Confirmation System were developed by Professor Anthony Yeh, Dr Zhong Teng and Dr Run Shi from the Department of Urban Planning and Design, The University of Hong Kong.

2. A Novel Stem Cell-based Platform for Antiviral Discovery, Vaccine and Healthy Natural ProductsThere is an urgent need for the vaccine industry to establish a new type of human cell model for anti-virus drug development in order to enhance sensitivity and efficiency. The human Expanded Potential Stem Cells (hEPSCs) developed under this project help generate trophoblast stem cells (TSCs) and syncytiotrophoblasts (STBs), which express high levels of SARS-CoV-2 host factors and are highly susceptible to the viral infection. Remarkably, the infected eSTBs are 1,000 times more sensitive to antiviral drugs, making the TSC-STB platform a more ideal human cell source for viral production and performing antiviral discovery, including SARS-CoV-2, MERS, their variants, and the FDA approved anti-virus drugs and natural products.

The platform was developed by Professor Pengtao Liu from the Li Ka Shing Faculty of Medicine, and Dr Degong Ruan and Professor Fang Liu from the Centre for Translational Stem Cell Biology, The University of Hong Kong.

3. Ultra-thin Flexible Robotic Instruments for Endoluminal SurgeryUnder this project, a robotic system with highly miniaturised and flexible instruments has been developed to address challenges in the endoluminal treatment of early-stage gastrointestinal and urinary tract cancers. In current practice, there is a steep learning curve for resecting tumours in their entirety due to a lack of instrument dexterity and poor tissue retraction. The developed system provides two robotic instruments as small as 2.5 mm with 5 degrees of freedom each, enabling enhanced endoluminal surgery with conventional rigid and flexible endoscopes. Overall, the system will help to flatten the learning curve so that more clinicians can provide patients with effective early-stage care.

The system was developed by the research team of Dr Ka-Wai Kwok from the Department of Mechanical Engineering, The University of Hong Kong.

4. Automatic Alignment of MicroparticlesThis groundbreaking chip-size invention revolutionises the way to align micro-particles in liquids. Using a microchannel with specifically arranged roadblocks that effortlessly guides disordered particles into an ultrafast-flowing chain, it provides outstanding performance at a low cost. As compact as a credit card and priced at less than USD10, each device can align up to one million particles per minute with precision down to 0.001 mm. This game-changer bypasses the limitations of current methods, thereby enhancing the efficiency of applications that involve microparticle analysis and processing, particularly blood cell screening and water filtration.

The invention was developed by Professor Kevin Tsia and Dr Kelvin Lee from the Department of Electrical and Electronic Engineering, The University of Hong Kong.

5. Interferon-integrated coronavirus vaccine: a next-generation universal live vaccine protects against pan beta-coronavirus infectionIn the wake of the COVID-19 pandemic, it is imperative to develop a next-generation coronavirus vaccine that can offer protection against current and potential future beta-coronavirus infections. However, there are several technical hurdles that must be overcome in order to create an ideal vaccine that has three key properties: 1) universal protection against a broad range of beta-coronaviruses, 2) enhanced mucosal T cell immunity, and 3) activated innate anti-viral mechanisms to ensure safety. The team has developed an innovative immunological design and genetic engineering technology that has resulted in the creation of a universal beta-coronavirus mucosal vaccine and a streamlined production pipeline. Preclinical studies in two animal models have demonstrated the efficacy of the vaccine in terms of the three key properties.

The technology was developed by Dr KH Kok and the team from the Department of Microbiology of The University of Hong Kong and the Centre for Virology, Vaccinology and Therapeutics.

6.NanoComplexNanoComplex is a next-generation vaccine technology designed for nasal use and carries the potential to target all kinds of viruses. Viruses have proteins as antigens and nucleic acids as genomes, and both are critical components to alert our immune system and generate strong protection. The NanoComplex technology takes full advantage of these components, combines viral proteins and nucleic acids with innovative methods, and creates a nano-sized complex that can be used as a nasal vaccine. Since all viruses have the same components, the NanoComplex can switch its internals for different viruses, and therefore can potentially defend against all known viruses.

The technology was developed by a research team lead by Dr Kin Hang Kok from the Department of Microbiology of The University of Hong Kong, and the Centre for Virology, Vaccinology and Therapeutics.

BRONZE MEDALS

1. Remote e-Inspection System for the Manufacturing and Delivery of Offsite Modular ConstructionThis e-Inspection System is a Modular Construction Supply Chain Quality Assurance system that includes i-Core (an loT device attached to each MC module to monitor the position, humidity, temperature and collision data), e-InStar (an App for uploading the checking result of each production step in a remote factory to the block chain), e-TranStar (an App for monitoring the location and condition of the MC module in the transport process) and a blockchain-based backend. Designed for offsite modular construction use to help solve housing problems, it reduces resources required for supervision and paperwork while ensuring tamper-proof data, helping overcome the current difficulties in monitoring the quality of production and transportation from remote sites. The system has been piloted in an HKU project in Hong Kong with two 17-storey buildings using 952 MiC modules.

The system was developed by Prof Wilson Lu, Director of the iLab of the Faculty of Architecture, together with Professor Anthony Yeh, Chair Professor of the Department of Urban Planning and Design, and Mr KL Tam, Former Director of the Estates Office of The University of Hong Kong.

2. On-site Fertilizer Production for Perpetual FarmingThe EC Flow device developed under this project is an energy-neutral system that fully integrates with solar panels and battery storage systems to detect and remove numerous toxic residual nitrates and nitrites (NOx) in soil using renewable energy. It can further upcycle NOx and feed fertilizer back into urban farming sites, achieving waste remediation, pollution mitigation and resource recovery to reach nitrogen neutrality. This solution can be applied to plantation areas of any scale and type, including farms, lawns, roof gardens and balconies. The goals are lowering toxin levels in municipal waste and alleviating ecological strains on urban sewage systems, ultimately improving the urban municipal discharge situation and local marine environment.

The device was innovated by Dr Edmund Tse, Wanying Wang from the Department of Chemistry, The University of Hong Kong, and commercialised with the help of Yip Jackson Ho So.

3. Smart Elderly WalkerThis is a smart and handy device pairing advanced Artificial Intelligence with a home-friendly design, helping the elderly stay healthy, active and independent while making their lives easier and more secure. It brings together several advanced features, including a stable mechanical structure with smooth omnidirectional mobility, dual-mode actuation and control for simultaneous walking/standing support and fall prevention/recovery, soft robotic handles and privacy-friendly sensors for event detection and control and fall detection, as well as specialised sound-source localisation to locate the user quickly and effectively, providing enhanced safety protection and superior walking assistance for older adults.

The device was developed by the research team of Professor Chuan Wu, Dr Hao Luo, Dr Zhao Chongyu and Dr Wen Rongwei from the Department of Computer Science of The University of Hong Kong; and Professor Wang Zheng from the Department of Mechanical and Energy Engineering of The Southern University of Science and Technology in Shenzhen.

4. Aptasensor for Sepsis DiagnosticDiagnosing sepsis in a timely manner to ensure optimal patient survival with prompt treatment remains a major challenge. This invention utilises a novel DNA-based biosensor that integrates aptamers into a point-of-care platform to advance sepsis diagnosis. Aptamers recognise biomarkers by specifically binding to unique structures on their surface. Unlike antibody-based tools, aptamers provide multiple advantages in terms of stability, production cost, reproducibility and flexibility that can readily be integrated into various platforms, such as electrochemical platforms (quantitative device) and/or colorimetric assays (rapid test). The aptasensor provides a quick and highly sensitive solution for sepsis detection that can help revolutionise point-of-care sepsis detection.

The invention was developed by Dr. Louisa Hoi-Ying Lo, Dr. William Whitehouse, supervised by Professor Julian Tanner, from the Advanced Biomedical Instrumentation Centre.

The Universitys participation in this years competition was coordinated by the Technology Transfer Office of HKU.

For details about the International Exhibition of Inventions of Geneva, please visit:https://inventions-geneva.ch/en/home/.

About Technology Transfer Office, HKUThe Technology Transfer Office (TTO) manages the use of HKUs intellectual property assets by providing patenting, licensing and other commercialisation support to the Universitys researchers and inventors. Acting as the bridge linking HKU to society in the area of technology commercialisation, TTO helps industries and businesses to access HKUs powerhouse of knowledge, innovation and expertise through close collaboration. Website:http://www.tto.hku.hk.

For media enquiries, please contact:Communications and Public Affairs Office, HKUMs Melanie Wan (Email: melwkwan@hku.hk)Ms Jaymee Ng (Email:ngjaymee@hku.hk)Mr Kenneth Choi (Email:khkchoi@hku.hk)

Technology Transfer Office, HKUMs Joy Ma (Email: joy@tto.hku.hk)

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