Biotechnology Research Articles

The winding journey of biotech capital raising: deciphering the jargon

Ensuring biotech companies are sufficiently capitalized to propel innovation and development remains a central focus for management. In this article, we draw on our broad perspective interacting with venture capitalists to offer thoughts on investor feedback. Understanding venture capitalists’ mindsets and investment theses will increase the probability of securing needed capital.

Predictions for the Revenue Status and Valuation of Biotechnology Companies Based on PEG Model

By the time of 2023, the world is recovering from the pandemic of COVID-19. The infection of COVID-19 has become regular and under governments’ control, the world is entering post-pandemic era. During the time of the pandemic, the crisis had led to a significant increase in the revenue and valuation of biotechnology companies. However, the peak of demand has been reduced, the market is concerning about the future of these companies. This study will investigate the revenue status and valuation of biotechnology companies now, by using PEG model. According to the current situations and data, the predictions of the future will be presented as well. Based on the analysis, the peak of revenue gained and valuation of biotechnology companies have passed. The pandemic has laid an excellent foundation for them, some of the firms may successfully transfer their production capacity into other medical products also demanded in post-pandemic era, maintaining their level of revenue and valuation. However, for the most companies in this industry, it is expected that they will experience a downward trend. Overall, these results shed light on guiding further exploration of significance for the prediction and analysis of the future development of biotechnology companies and the further exploration of the way that investors choose good biotechnology companies as value investment targets.

Two biotech leather companies struggle

Two biotech leather companies struggle

Magnetic Analysis of MgFe Hydrotalcites as Powder and Dispersed in Thin Films within a Keratin Matrix.

Hydrotalcites (HTlcs) are a class of nanostructured layered materials that may be employed in a variety of applications, from green to bio technologies. In this paper, we report an investigation on HTlcs made of Mg and Fe, recently employed to improve the growth in vitro of osteoblasts within a keratin sponge. We carried out an analysis of powder materials and of HTlcs dispersed in keratin and spin-coated on a Si/SiO2 substrate at different temperatures. A magnetic study of the powders was carried out with a Quantum Design Physical Property Measurement System equipped with a Vibrating Sample Magnetometer. The data gathered prove that these HTlcs are fully paramagnetic, and keratin showed a very small magnetic response. Optical and Atomic Force Microscopy analyses of the thin films provide a detailed picture of clusters randomly dispersed in the films with various dimensions. The magnetic properties of these films were characterized using the Nano Magneto Optical Kerr Effect (NanoMOKE) down to 7.5 K. The data collected show that the local magnetic properties can be mapped with a micrometric resolution distinguishing HTlc regions from keratin ones. This approach opens new perspectives in the characterization of these composite materials.

Ethics of research

Ethics of research

Bioconjugation Technology Selection during Early-Stage Development – A Strategy to Streamline IND and Manufacturing Timelines

Bioconjugate therapeutics comprise a fast-growing class of drugs with applications in various disease areas. In the past years, particularly antibody-drug conjugates (ADCs) have undergone a period of great success with eight products approved by the Food and Drug Administration (FDA) between 2019-2022 alone. The rapid emergence of novel bioconjugation technologies, payloads and linkers as well as their application on different protein modalities has made bioconjugate development and manufacturing increasingly complex. Clinical success of bioconjugates depends on the delicate interplay between the protein, linker and payload, which is often not readily predictable. Therefore, it is important to consider a range of different technologies when designing a bioconjugate drug. Since navigation of a constantly evolving technology landscape is challenging, it may be beneficial for many biotech companies to enter partnerships with a contract development and manufacturing organization (CDMO) that can help realize the drug concept by providing access to a range of tools for the development of bioconjugates. Partnering with a CDMO at an early-stage of drug development can greatly de-risk the program as the right CDMO can give guidance on how to design a conjugation process that not only fulfills the requirements in terms of product attributes but that is also robust enough to streamline further process development and upscale for moving towards an IND filing.

Stress, microRNAs, and stress-related psychiatric disorders: an overview.

Stress is a major risk factor for psychiatric disorders. During and after exposure to stressors, the stress response may have pro- or maladaptive consequences, depending on several factors related to the individual response and nature of the stressor. However, the mechanisms mediating the long-term effects of exposure to stress, which may ultimately lead to the development of stress-related disorders, are still largely unknown. Epigenetic mechanisms have been shown to mediate the effects of the environment on brain gene expression and behavior. MicroRNAs, small non-coding RNAs estimated to control the expression of about 60% of all genes by post-transcriptional regulation, are a fundamental epigenetic mechanism. Many microRNAs are expressed in the brain, where they work as fine-tuners of gene expression, with a key role in the regulation of homeostatic balance, and a likely influence on pro- or maladaptive brain changes. Here we have selected a number of microRNAs, which have been strongly implicated as mediators of the effects of stress in the brain and in the development of stress-related psychiatric disorders. For all of them recent evidence is reported, obtained from rodent stress models, manipulation of microRNAs levels with related behavioral changes, and clinical studies of stress-related psychiatric disorders. Moreover, we have performed a bioinformatic analysis of the predicted brain-expressed target genes of the microRNAs discussed, and found a central role for mechanisms involved in the regulation of synaptic function. The complex regulatory role of microRNAs has suggested their use as biomarkers for diagnosis and treatment response, as well as possible therapeutic drugs. While, microRNA-based diagnostics have registered advancements, particularly in oncology and other fields, and many biotech companies have launched miRNA therapeutics in their development pipeline, the development of microRNA-based tests and drugs for brain disorders is comparatively slower.

제약·바이오 기업의 연구개발비 회계처리 감독지침이 신규상장 기업들의 공모가 저평가에 미치는 영향

[Purpose]This study analyzes the impact of the supervisory guidance issued in 2018 on the accounting treatment of research and development expenses on firm valuations.
 [Methodology]We empirically analyze the impact of the guidance on uncertainty in firm valuations by examining the change in the underpricing of initial public offerings before and after the issuance of the supervisory guidance on accounting for research and development expenses for firms that went public between 2005 and 2020.
 [Findings]We find that the public offering undervaluation of pharmaceutical and biotechnology companies decreased after the issuance of the supervisory guidance. This result appears to be due to the fact that the issuance of the supervisory guidance on accounting for R&D expenses reduced the uncertainty of accounting for R&D expenses in pharmaceutical and biotechnology companies, which led to a decrease in the discount in the public offering price. In addition, the increase in IPO undervaluation due to impairment losses also decreased after the introduction of the guideline, which can be interpreted as the guideline reducing managerial discretion in recognizing impairment losses and reducing the uncertainty of firm value.
 [Implications]The finding that supervisory guidance on accounting for R&D expenses in the pharmaceutical and biotechnology industries mitigates IPO underpricing has policy implications that regulations that limit managerial discretion in accounting for these industries can enhance market efficiency.

100-OR: A Novel Macrophage-Regulation Drug for Treating Diabetic Foot Ulcer—A Phase III MRCT Study and Taiwan Experience

Macrophage regulation plays an essential role in Diabetic foot ulcer (DFU) healing. By recognizing the therapeutic potential in M1/M2-macrophage-regulation for DFUs, we aim to investigate the efficacy and safety of ON101, a topical new drug with a M1/M2-macrophage-regulating mechanism in phase 3 MRCT across the US, China, and Taiwan (NCT01898923). There are 236 eligible subjects with Wagner Grade 1 or 2 DFU(s), randomized 1:1 to receive ON101 or comparator for up to 16 weeks, followed by a 12-week follow-up. The incidence of complete healing was 60.7% by ON101 and 35.1% by the comparator (P=0.0001). The time to complete ulcer healing was faster in the ON101 group (P=0.002). Since the high levels of HbA1C, ulcer persistence, ulcer location, and amputation history as the risk factors leading to poor DFU outcomes, ON101 also showed demonstrated robust efficacy and safety in hard-to-heal ulcers. ON101, approved by Taiwan FDA in 2021, was shown with excellent therapeutic efficacy and safety in the treatment of DFUs. In a real-world scenario, case collation from 133 patients, ulcer severity with Wagner grade 1 to 4, after applying ON101, the average ulcer complete healing rate is 71%, no adverse events were related to ON101, which validates the drug safety of ON101. ON101 further supports its excellent ability to promote wound healing in DFU patients with various severities (Wagner grade 1 to 4). All those patients who applied ON101 alone or with other therapies reached ulcer healing under infection control and with necessary debridement. Macrophage-regulating drug achieved superior therapeutic efficacy in a large Phase 3 MRCT and has demonstrated excellent therapeutic efficacy in the real-world scenario where Wagner grade 3 and 4 DFUs reached ulcer healing when ON101 was combined with other therapies. This enlightens the future treatment in Wagner grade 1 to 4 DFUs by macrophage immunomodulation new drug as a promising solution. Disclosure S.Chen: Employee; Oneness Biotech Co., Ltd. S.Chu: None. C.Pu: None. J.Chen: Employee; Oneness Biotech Company. Y.Wu: Employee; Oneness Biotech Co., Ltd.

1583-P: Dynamic Tracking and Identification of Liver-Specific Secretory Proteins in the Circulation of Live Mice

Secretory proteins are an essential component of interorgan communication networks that regulate animal physiology. Current approaches for identifying secretory proteins from specific cell and tissue types are largely limited to in vitro or ex vivo models which often fail to recapitulate in vivo biology. As such, there is mounting interest in developing in vivo analytical tools that can provide accurate information on the origin, identity, and spatiotemporal dynamics of secretory proteins. Here, we describe iSLET (in situ Secretory protein Labeling via ER-anchored TurboID) which selectively labels proteins that transit through the classical secretory pathway via catalytic actions of Sec61b-TurboID, a proximity labeling enzyme anchored in the ER lumen. To validate iSLET in a whole-body system, we express iSLET in the mouse liver and demonstrate efficient labeling of liver secretory proteins which could be tracked and identified within circulating blood plasma. Furthermore, proteomic analysis of the labeled liver secretome enriched from liver iSLET mouse plasma is highly consistent with previous reports of liver secretory protein profiles. Taken together, iSLET is a versatile and powerful tool for studying spatiotemporal dynamics of secretory proteins, a valuable class of biomarkers and therapeutic targets. Disclosure K.Kim: None. J.Suh: None. Funding National Research Foundation of Korea (2018R1A2A3075389, 2016M3A9B6902871, 2017K1A1A2013124, 2021R1A2C2007573, 2020R1C1C1013927); KAIST Key Research Institutes Project; Bio and Medical Technology Development Program (2019M3E5D3073104); Ministry of Science

IPO as a Key Process for a Successful Company

IPO is the most popular business practice today, which can be seen as the key to a successful company. IPO could bring a lot of benefits to the company like additional capital raised, a better international reputation, deeper consumer recognition, liquidate benefits for the shareholders and investors, greater employee benefits, and higher valuation because of transparency, compared to a private business. During the crisis, there was less focus on maximizing IPO proceeds due to both an increased emphasis on research coverage and allocations of hot IPOs to the personal brokerage accounts of issuing firm executives. But the company should consider the costs of IPOs carefully, initial underpricing is likely to cause underperformance in the long run, mandatory disclosures may increase the competitive cost, especially for biotech companies, and the substantial fees of an IPO have to be considered. In this paper, a literature study of the pros and cons of an IPO and IPOs are crucial to the development of a business.

The Medical Science Liaison Role in Spain: Opinion of the Commercial Department Personnel.

The Medical Science Liaison (MSL) works in a cross-functional way, especially with the commercial department. The aim of the present study was to evaluate the knowledge of these positions about the MSL role in their companies and describe the grade of internal interaction between them in daily practice. An online survey was completed by 151 employees from commercial departments between January and April 2020. It consisted of 29 or 31 items, depending on answers. Of participants, 22.5% and 77.5% occupied management or non-management positions, respectively. Most respondents stated that the MSL role belongs primarily to the medical department (94.6%), considered important that promotional materials are elaborated or supported by the medical department (95.4%), highlighted the importance of sharing their daily activity with MSLs (77.8%) and vice versa (89.3%). The most valuable activity of MSLs was clinical sessions (55.3%), followed by speaker briefings (16.0%), and data discussions (14.7%). Most useful activities of MSLs for participant's day to day were external training (sessions to healthcare providers, HCPs, 34.9%), support of key opinion leaders' (KOLs) unmet needs (22.1%), and feedback from fieldwork aimed at redefining new strategies of the company (15.4%). The mean overall assessment (0-10) of the MSL was 8.1. The MSL represents a key role inside pharmaceutical and biotechnological companies, providing scientific value. The members of the commercial departments interact with the MSL on a daily basis and consider that it is a strategic position with a great future that adds value within the company.

An Overview of the 2022 Update of Good Publication Practice (GPP) Guidelines: A Medical Writer’s Perspective

The Good Publication Practice (GPP) guidelines are a set of principles and recommendations pertaining to peer-reviewed or peer-orientated publications arising from research funded by or conducted in collaboration with industry sponsors, typically pharmaceutical, biotechnology, and medical device companies.
 This year marks the 20-year anniversary of the first GPP guidelines, which were published in 2003.1 These initial GPP guidelines were developed to promote transparency and to establish standards for publication practice used by pharmaceutical companies. Since then, GPP guidelines have undergone several updates to reflect changes within the publication field and to provide additional practical guidance on publication processes and procedures. These updates include GPP22 published in 2009 and GPP33 published in 2013. Good Practice for Conference Abstracts and Presentations (GPCAP)4 was published in 2019 to provide guidance specifically for congress abstract and presentations. The latest update of the GPP guidelines, the GPP 2022 update, was published in August 20225.

Advances in Animal Models and Cutting-Edge Research in Alternatives: Proceedings of the Third International Conference on 3Rs Research and Progress, Vishakhapatnam, 2022.

Animal experimentation has been integral to drug discovery and development and safety assessment for many years, since it provides insights into the mechanisms of drug efficacy and toxicity (e.g. pharmacology, pharmacokinetics and pharmacodynamics). However, due to species differences in physiology, metabolism and sensitivity to drugs, the animal models can often fail to replicate the effects of drugs and chemicals in human patients, workers and consumers. Researchers across the globe are increasingly applying the Three Rs principles by employing innovative methods in research and testing. The Three Rs concept focuses on: the replacement of animal models (e.g. with in vitro and in silico models or human studies), on the reduction of the number of animals required to achieve research objectives, and on the refinement of existing experimental practices (e.g. eliminating distress and enhancing animal wellbeing). For the last two years, Oncoseek Bio-Acasta Health, a 3-D cell culture-based cutting-edge translational biotechnology company, has organised an annual International Conference on 3Rs Research and Progress. This series of global conferences aims to bring together researchers with diverse expertise and interests, and provides a platform where they can share and discuss their research to promote practices according to the Three Rs principles. In November 2022, the 3rd international conference, Advances in Animal Models and Cutting-Edge Research in Alternatives, took place at the GITAM University in Vishakhapatnam (AP, India) in a hybrid format (i.e. online and in-person). These conference proceedings provide details of the presentations, which were categorised under five different topic sessions. It also describes a special interactive session on in silico strategies for preclinical research in oncology, which was held at the end of the first day.

Endpoints reported in phase 3 randomized clinical trials at ASCO 2022.

1570 Background: The goal of phase 3 randomized clinical trials (RCTs) is to show clinically meaningful benefit for patients. An analysis of phase 3 RCTs presented at the ASCO 2022 Annual Meeting (ASCO22) was undertaken to assess which endpoints were evaluated. Methods: A systematic analysis was undertaken of ASCO22 abstracts from phase 3 RCTs reporting primary, secondary, interim, updated, and subgroup analyses, as well as trials reporting methodology of currently enrolling studies. Trials that reported posthoc, exploratory, biomarker, and retrospective analyses of RCTs were excluded. Information from ASCO22 materials and clinical trial registration websites was recorded by two independent researchers. Results: In total, 166 RCTs were identified: 93 trials with 50,781 enrolled patients and 73 trials expected to enroll 52,098 patients. More trials reported on patients with locally advanced/metastatic cancer (64.5%) compared to localized cancer (35.5%). Funding source was pharmaceutical or biotechnology company in 62%. Within locally advanced/metastatic trials, overall survival (OS) was a primary endpoint in 40%, while progression-free survival (PFS) was a primary or co-primary endpoint in 45% and 13% respectively. Other primary endpoints included (invasive) disease-free survival ((i)DFS), event free survival (EFS), overall response rate and pathological complete response. In trials that did not include OS as a primary endpoint, OS was a secondary endpoint in 88% (n = 56/64). Within the localized group, trials were subdivided into adjuvant, neo-adjuvant, definitive and other categories. Within the adjuvant trials (n = 27), the most common primary endpoint was (i)DFS (37%), followed by OS (15%). Other primary endpoints included recurrence-free survival, invasive breast cancer free survival, metastasis-free survival, EFS, and PFS. Across all trials, quality of life (QOL) was never reported as a primary endpoint, however it was as a secondary or other outcome in 53% for locally advanced/metastatic trials and 42% for localized trials. When comparing the endpoints reported in the ASCO22 abstract and meeting materials with those listed on the trial registration website, discordance was noted in 14%. Conclusions: Phase 3 RCTs presented at ASCO22 represented participation from over 100,000 patients, however, fewer than half in locally advanced/metastatic cancer and fewer than one quarter in localized disease utilized OS as a primary endpoint. QOL was listed as an endpoint in roughly one half of all trials. Endpoints reported did not match registration data in a significant minority. Debate over which endpoints should be used for cancer RCTs should be informed by this data.

Beyond Busulfan: Building a Better Conditioner for Bone Marrow Transplantation

Beyond Busulfan: Building a Better Conditioner for Bone Marrow Transplantation

Top 25 Biotech Companies of 2023

Genetic Engineering & Biotechnology NewsVol. 43, No. 6 A-ListTop 25 Biotech Companies of 2023Combined market cap soared 17% over the past year, with most of the largest public companies gaining value and biopharma outperforming the S&P 500Alex PhilippidisAlex PhilippidisSearch for more papers by this authorPublished Online:5 Jun 2023https://doi.org/10.1089/gen.43.06.08AboutSectionsView articleView Full TextPDF/EPUB Permissions & CitationsPermissionsDownload CitationsTrack CitationsAdd to favorites Back To Publication ShareShare onFacebookTwitterLinked InRedditEmail View articleFiguresReferencesRelatedDetails Volume 43Issue 6Jun 2023 Information© 2023 by GEN PublishingTo cite this article:Alex Philippidis.Top 25 Biotech Companies of 2023.Genetic Engineering & Biotechnology News.Jun 2023.18-19.http://doi.org/10.1089/gen.43.06.08Published in Volume: 43 Issue 6: June 5, 2023PDF download

HPR83 Coming to America: How Ex-US Drug Companies May Best Prepare for the Complexities of a US Market Product Launch

To determine the unique US policy and market access nuances which must be addressed for ex-US biotech companies or startups to enter the US market. The aim of this study is to determine what ex-US biotech startups must understand and focus on within the US market access space to best position new drug launches, given the differences among landscapes. Additionally, this work provides a framework of required issues to explore during early decision-making process in a pre-launch setting.

PRIORITY AREAS OF INNOVATION IN CORPORATE STRATEGIES OF MULTINATIONAL PHARMACEUTICAL COMPANIES IN GLOBAL COORDINATES

The purpose of the article is to substantiate global trends in the research and innovation activities of multinational pharmaceutical companies and to identify key directions of transformation in corporate innovation strategies in the context of increased international competition in the global pharmaceutical market. Methodology. The methodological basis of the work is the following methods of scientific research: dialectical, historical-logical, system-structural analysis of economic processes and phenomena, analysis and synthesis, the method of quantitative and qualitative comparisons. Results. One of the key global sustainable development goals of the United Nations for the period up to 2030 is to ensure the health and well-being of nations, which in global terms can be achieved primarily on the basis of the large-scale research activities of multinational pharmaceutical companies and the systematic commercialisation of the results of corporate research and development. In recent decades, under the influence of technoglobalisation processes, the innovatisation of pharmaceutical companies' activities has accelerated. It finds its concentrated expression in the steady increase of R&D costs, diversification of their structure, mass commercialisation of innovative developments and strengthening of their transdisciplinarity, accelerated capitalisation of R&D costs, etc. According to the world experience, the most effective innovative strategies of pharmaceutical companies today are such as mergers and acquisitions, strategic alliances of pharmaceutical and biotechnology companies, innovative outsourcing and insourcing, cointegration and pseudo-concentration, corporatisation of patent portfolios and research technologies, venture financing of R&D of pharmaceutical companies. At the same time, mergers and acquisitions are increasing the capitalisation of the global pharmaceutical industry, strengthening its expansion into global markets, redistributing its regional segments and diversifying the sources of funding for pharmaceutical companies' research activities. Value/оriginality. Current trends and features of corporate strategy formation in multinational pharmaceutical companies are identified. Strategic orientations of research activities of pharmaceutical companies in specialised areas of clinical research in the context of therapeutic categories are determined. The process of capitalisation of pharmaceutical companies is analysed and the tendency to activate the use of corporate mergers and acquisitions (M&A) strategy in the pharmaceutical business is revealed. It is proposed to create strategic alliances of multifunctional pharmaceutical companies with biotech companies using the M&A strategy. It is recommended that pharmaceutical companies strengthen their innovation orientation in the design of their strategies by combining patent portfolios and research technologies, as well as jointly implementing large-scale innovation programmes and projects.

Review of Recent Advances in the Physiology of the Regulation of Cellulase and Xylanase Production by Basidiomycetes

The potential of wood-rotting and litter-deconstructing basidiomycetes to convert lignocellulose into a wide variety of products has been extensively studied. In particular, wood-rotting basidiomycete secretomes are attracting much attention from researchers and biotechnology companies due to their ability to produce extracellular hydrolytic and oxidative enzymes that effectively degrade cellulose, hemicellulose, and lignin of plant biomass. An analysis of the available literature data shows that Basidiomycota fungi, which are most adapted to the depolymerization of plant polysaccharides, are promising but so far unexploited sources of new hydrolytic enzymes. The review summarizes the latest data on the great variety, common features, and unique properties of individual fungi and the production of cellulases and xylanases by various physiological and ecological groups of basidiomycetes. The most important microbial cellulase-producing strains for submerged and solid-phase fermentation, as well as the main substrates, including the use of agro-industrial waste, are considered. It highlights ways to increase both cellulase and xylanase expression levels and the cost-effectiveness of producing these enzymes for various biotechnological applications. It is anticipated that this review will be particularly useful to novice scientists working in the lignocellulose biorefinery, as it describes current knowledge and issues related to the production and regulation of polysaccharide hydrolyzing enzyme synthesis.