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Eupraxia Pharmaceuticals Announces Closing of Overnight Marketed Offering for Gross Proceeds of C$33.9 Million
Company to host conference call to discuss financial results and provide a corporate update on March 21, 2024 at 4:30 p.m. ET Company to host conference call to discuss financial results and provide a corporate update on March 21, 2024 at 4:30 p.m. ET
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Journey Medical Corporation to Announce Year End 2023 Financial Results on March 21, 2024
Live video webcast with Tom Equels, Chief Executive Officer of AIM ImmunoTech, on Wednesday, March 20th at 12:00 PM ET Live video webcast with Tom Equels, Chief Executive Officer of AIM ImmunoTech, on Wednesday, March 20th at 12:00 PM ET
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AIM ImmunoTech to Participate in the Virtual Investor Lunch Break: The AIM Opportunity
PALO ALTO, Calif., March 15, 2024 (GLOBE NEWSWIRE) -- Scilex Holding Company (Nasdaq: SCLX, “Scilex” or “Company”), an innovative revenue generating company focused on acquiring, developing and commercializing non-opioid pain management products for the treatment of acute and chronic pain, today announced that the U.S. Bankruptcy Court for the Southern District of Texas (the “Court”), in connection with the bankruptcy proceedings of Sorrento Therapeutics, Inc. (“Sorrento”), Scilex’s former controlling stockholder, approved the settlement and mutual release agreement (the “Definitive Settlement Agreement”) between Scilex’s wholly owned subsidiary, Scilex Pharmaceuticals Inc. (“Scilex Pharma”), and Sorrento, on the one hand, and Virpax Pharmaceuticals, Inc. (“Virpax”), on the other hand. The Definitive Settlement Agreement relates to the term sheet previously announced by Scilex on February 26, 2024, regarding a mutual release and settlement agreement between Scilex Pharma, Sorrento and Virpax in respect of the action (the “Action”) filed by Scilex Pharma and Sorrento (together, the “Plaintiffs”) against Anthony Mack, former President of Scilex Pharma and Virpax, a company founded and then headed by Mr. Mack. Pursuant to the Definitive Settlement Agreement, Virpax is obligated to make the following payments to the Company: (i) $3.5 million by March 18, 2024 (the “Initial Payment”); (ii) $2.5 million by July 1, 2024 (the “Second Payment”); and (iii) to the extent any of the following drug candidates are ever sold, royalty payments of (a) 6% of annual Net Sales (as defined in the Definitive Settlement Agreement) of Epoladerm; (b) 6% of annual Net Sales of Probudur; and (c) 6% of annual Net Sales of Envelta. Such royalty payments will end upon the later of (i) expiration of the last-to-expire valid patent claim of Virpax or its licensor covering the manufacture, use or sale of such product in such country; and (ii) expiration of any period of regulatory exclusivity for such product in such country.
SAN DIEGO, March 15, 2024 (GLOBE NEWSWIRE) -- Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced it will participate in a virtual fireside chat on the Treatment Landscape & New Treatment Options for Prostate Cancer.
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Oncternal Participating in Virtual Fireside Chat with Key Opinion Leader on Treatment Landscape & New Treatment Options for Prostate Cancer
PALO ALTO, Calif., March 15, 2024 (GLOBE NEWSWIRE) -- Scilex Holding Company (Nasdaq: SCLX, “Scilex” or “Company”), an innovative revenue generating company focused on acquiring, developing and commercializing non-opioid pain management products for the treatment of acute and chronic pain, today announced that it received Halal Certification of its commercial product ZTlido, indicating that ZTlido has undergone rigorous assessment to determine that it is permissible or acceptable in accordance with Islamic standards. The Halal certification was issued under the authority of Circle H International, Inc. (“Circle H”) and offers the Company the opportunity to provide ZTlido to Islamic markets globally. This announcement supports the global expansion strategy for Scilex, which Scilex anticipates will include a presence in the Middle East and North Africa (MENA) region with an initial priority focus on the UAE and Saudi Arabia.
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Scilex Holding Announces Issuance of Halal Certification for its ZTlido® product by Circle H International, Inc.
Information concerning the total number of voting rights and shares, provided pursuant to article L. 233-8 II of the Code de commerce (the French Commercial Code) and article 223-16 of the Règlement général de l’Autorité des Marchés Financiers (Regulation of the French stock market authority)
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Sanofi: Information concerning the total number of voting rights and shares - February 2024
ReOpen was the first ever large placebo-controlled clinical trial program to demonstrate statistically significant reduction of symptoms in chronic sinusitis patients without nasal polyps
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XHANCE Approved by FDA as First and Only Medication Indicated for Treatment of Adults with Chronic Rhinosinusitis without Nasal Polyps
SAN JOSE, Calif., March 15, 2024 (GLOBE NEWSWIRE) -- Rani Therapeutics Holdings, Inc. (“Rani Therapeutics” or “Rani”) (Nasdaq: RANI), a clinical-stage biotherapeutics company focused on the oral delivery of biologics and drugs, today announced that it plans to release financial results for the fourth quarter and full year ended December 31, 2023 and provide a business update on Wednesday, March 20 after the close of trading. Rani’s management team will host a conference call and webcast beginning at 4:30 p.m. ET.
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Rani Therapeutics to Report Fourth Quarter and Full Year 2023 Financial Results
TORONTO and HAIFA, Israel, March 15, 2024 (GLOBE NEWSWIRE) -- NurExone Biologic Inc. (TSXV: NRX) (Germany: J90) (the “Company” or “NurExone”), a pioneering biopharmaceutical company, developing regenerative medicine therapies, announces today its intention to broaden its market reach through a recently filed application for listing on the OTCQB® Venture Market (the "OTCQB") in the United States. Listing on the OTCQB is subject to approval of the OTC Markets Group.
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NurExone's Strategic Update: Submission of OTCQB Listing Application to Initiate US financial presence
NEW YORK, March 15, 2024 (GLOBE NEWSWIRE) -- Psyence Biomedical Ltd. (the “Company”) (Nasdaq: PBM) announced that on March 11, 2024, it received two letters from the listing qualifications department staff of The Nasdaq Stock Market (“Nasdaq”), one notifying the Company (the “MVLS Notice”) that for the last 30 consecutive business days, the Company’s Market Value of Listed Securities (“MVLS”) was below the minimum of $50 million required for continued listing on the Nasdaq Global Market pursuant to Nasdaq Listing Rule 5450(b)(2)(A) (the “Market Value Standard”), and the other notifying the Company (the “MVPHS Notice”) that for the last 30 consecutive business days, the Company’s Market Value of Publicly Held Shares (“MVPHS”) was below the minimum of $15 million required for continued listing on the Nasdaq Global Market pursuant to Nasdaq Listing Rule 5450(b)(2)(C) (the “MVPHS Standard”).
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Psyence Biomedical Ltd. Receives Nasdaq Notifications Regarding Market Value of Listed Securities and Market Value of Publicly Held Shares
FINANCIERE DE TUBIZE SA/NV
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Financière de Tubize - Annual report 2023
Agreement pairs Norgine’s commercial expertise and leading European footprint with PEDMARQSI®, the first and only approved therapy in the European Union and U.K. for reducing the risk of cisplatin-induced hearing loss in pediatric patients with localized, non-metastatic solid tumors
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Fennec Pharmaceuticals and Norgine Enter into Exclusive Licensing Agreement to Commercialize PEDMARQSI in Europe, Australia, and New Zealand
Ad hoc announcement pursuant to Art. 53 LR
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Idorsia and Viatris successfully close the transaction for the global research and development collaboration
Saint-Herblain (France), March 18, 2024 – Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company, today announced an agreement with funds managed by leading U.S. healthcare investment firms Deerfield Management Company and OrbiMed to extend the interest-only period of its existing loan by eighteen months.
Editors Note: This is the second article in a two-part series exploring the promise and limitations of the field of personalized medicine. The first part focused on advances and innovation in the field.
In the mid-1990s, researchers identified two gene mutations that are key to predicting genetic susceptibility to breast cancer: BRCA1 and BRCA2. In 1996, the BRCA1/2 mutation screening became the first genetic test for cancer risk available as a clinical service.
This genetic screening was an early innovation in a field that has come to be known as personalized medicine, which can be applied across a variety of medical specialties. Its defining characteristic is that a patients health care team takes into consideration a wide range of factors such as genetics, lifestyle, diet, specifics of disease presentation, and living environment when deciding on an individualized prevention or treatment plan.
With the advent of personalized medicine, including genetic screening as well as more targeted cancer drugs and therapies, the death rate for breast cancer in the United States declined by 43% from 1989 to 2020, according to the American Cancer Society (ACS). But even as mortality from breast cancer has decreased overall, there are statistics that highlight inequities in outcomes. Despite Black women having a lower incidence of breast cancer than non-Hispanic White women, Black women of all ages die from breast cancer at a 40% higher rate than non-Hispanic White women, and Black women under 50 years old die of breast cancer at twice the rate of non-Hispanic White women in the same age category.
Research shows that Black women get the BRCA1/2 screening less often than White women, at least in part because it is offered to them less frequently. One 2017 study found that, of women under 50 years old diagnosed with invasive breast cancer in Florida, 85.7% of the White women in the study were referred for genetic testing, while only 37% of the Black women were.
This is just one example of the inequities that some medical researchers and health equity advocates say severely limit the benefits of personalized medicine, even as technology advances.
[Personalized medicine] products are informative and are having an impact in certain communities, but its not equitable across all communities, says Rick Kittles, PhD, senior vice president for research at Morehouse School of Medicine, a historically Black medical college (HBCU) in Atlanta.
In the United States, people who are Black, Hispanic or Latino, American Indian or Alaska Native, people with low incomes, people who are uninsured or underinsured, and those who live in rural areas, as well as others who have been marginalized, face multiple barriers to personalized medicine. These barriers include a lack of inclusion of diverse genetics in research, the high cost of genetic testing and technology used in personalized medicine, and a lack of awareness of and education about personalized medicine among health care providers outside of urban medical centers. Some sociologists hypothesize that advances in medical innovation may, in fact, exacerbate existing inequities because people with economic and educational advantage are more likely to access care that improves lives and reduces mortality, while those from marginalized communities are left behind.
Its a problem that several academic medical centers are seeking to address with a range of strategies, from expanding personalized medicine research at HBCU medical schools to engaging community partners for research recruitment.
The field of human genetics has grown exponentially since the 2003 completion of the Human Genome Project, an international research effort that mapped the gene pairs that make up human DNA. The endeavor found that all humans share 99.9% of the same genome, with the other 0.1% accounting for all genetic diversity among individuals. Within that 0.1% are the wide variety of heritable traits, from physical characteristics to genetic mutations that cause or increase risk for certain diseases.
And yet, in the more than 6,000 genome-wide association studies (when researchers scan the genomes of large populations to try to identify genetic variations associated with diseases) that have been published internationally over the last two decades, 90% of all people analyzed were of European descent, according to a 2023 article in the Human Molecular Genetics journal.
This means that researchers have very little understanding of heritable disease risk for the vast majority of the worlds population when it differs from the variations seen in people of European descent.
Kittles, who is a genetic epidemiologist by trade, joined Morehouse in 2022 to lead the medical schools expanding efforts to advance medical research focusing on the inclusion of people from groups that have historically been excluded from clinical research and underserved in health care.
Among his faculty recruits is Melissa B. Davis, PhD, a genetics researcher focused on racial disparities in cancer who will lead the schools new Institute of Genomic Medicine. Davis previous work includes identifying two genes found in women of African ancestry that may increase their likelihood of developing an aggressive form of breast cancer, much like the BRCA1/2 gene.
For women of color who get tested [for BRCA 1/2], the benefit of that test is not equitable and in many cases the tests come back unknown, Kittles says. Thats because those variants [that are found in people of African descent] are not in databases Its a glaring, prime example of where we are in precision medicine right now.
The research expansion at Morehouse is funded by an $11.5 million grant from the Chan Zuckerberg Initiative (CZI, created by Facebook founder Mark Zuckerberg and his wife, Priscilla Chan) and is part of the charitable foundations larger Accelerate Precision Health program. CZI has granted equal funds to each of the nations three other HBCU medical schools: Charles Drew University College of Medicine in Los Angeles; Howard University College of Medicine in Washington, D.C.; and Meharry Medical College in Nashville.
When we think about the science we want to support, [we ask,] Who does the science? What science is being done? Who does the science serve? says Bil Clemons, PhD, science program officer for Diversity, Equity, and Inclusion in Science for CZI. Fundamentally, Is the science that were doing inclusive of everyone?
Most of the funding from CZI has gone to hiring faculty at HBCU medical schools to bolster their capacity to expand their research footprint over time, but its also funded the creation of new programs to train genetic counselors at Charles Drew University College of Medicine.
Kittles says that CZIs funding is instrumental to advancing research into genetic diversity and health disparities at HBCU medical schools, particularly because these institutions have often been overlooked for federal and philanthropic funding in the past.
That creates a disparity that not only limits the research impact of those institutions, but also the health of the communities that they serve, says Kittles. So much so that while all HBCUs have strong teaching experience, the development of research has been hampered because of the lack of funding and the ability to bring in talent who want to do research. The sustainability of research is limited because of that history.
In turn, thats set back progress in reducing health disparities, especially in Black communities and other communities of color, Kittles says, because HBCU medical schools tend to have more trust and access to those communities than many other medical centers.
Many academic medical centers historically have had a very strong disconnect with disparate communities, Kittles explains. The bulk of their research and the bulk of their patients are not diverse And so, when they do research, theyre limited in terms of their touch.
In addition to the efforts at the HBCU medical schools, dozens of medical centers are participating in the National Institutes of Health (NIH) All of Us research program, the goal of which is to build one of the largest and most diverse health databases in the world.
The All of Us program is studying patients social determinants of health, a phrase that refers to the various factors such as environment, socioeconomic status, access to healthy food, and access to health care that can affect health.
The NIH has funded and partnered with more than a dozen organizations to expand their reach into the communities that are historically underrepresented in biomedical research, including the American Association on Health and Disability; the National Alliance for Hispanic Health; and the National Baptist Convention, USA Inc.; among others. These organizations use their connections within marginalized communities to enroll and retain participants in the program. As of September 2021, the partners had helped enroll more than 400,000 participants, 80% of whom are from communities that are historically underrepresented in research. The study aims to provide a holistic picture of health by collecting samples of blood, urine, and saliva; physical measurements; electronic health records; health and family medical histories; information about lifestyles and communities; and data from wearable technologies, such as smartwatches, according to the NIH.
And while this and other endeavors are a step forward, Kittles says that all academic medical centers have a responsibility to resolve inequities in their own communities in order to truly make progress in advancing accessibility to personalized medicine.
In my career, Ive been at resource-rich [institutions], and resource-poor [institutions], and what I call community-rich and community-poor. Some had strong relationships with the community, and others had no trust from communities around them, says Kittles. When we talk about health equity, there has to be a commitment that goes beyond the window dressings and the social media tags that you see Part of that is bringing individuals into the institution that represent the communities that you want to benefit.
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Personalized medicine is coming, but who will have access to it?
Also called: precision medicine or individualized medicine(Showmore)
personalized medicine, field of medicine in which decisions concerning disease prevention, diagnosis, and treatment are tailored to individual patients based on information derived from genetic and genomic data. Personalized medicine centres on the concept that information about a patients genes and genome allows physicians to make more informed and effective decisions about a patients care.This idea essentially is an extension of conventional medicine, in which one strategy is applied across all patients, without tailoring to personal genetic and genomic information.
The concept of personalized medicine, although not novel at the time, materialized in the 1990s, following advances in DNA sequencing technology, including automation and increased throughput. Out of those advances came efforts such as the Human Genome Project (HGP; 19902003), in which sequences of more than three billion base pairs of the human genome were elucidated and made available to researchers worldwide. Likewise, the International HapMap Project (200210), which identified genetic variations that contribute tohuman disease, provided researchers with the information needed to associate gene variants with specific diseases and disorders.
Those advances cast light on phenomena in medicine that had been observed for yearsfor example, that certain drugs are more effective in some patients and that, in response to certain medications, some patients experience unusually severe side effects. Progress in understanding the molecular factors underlying the influence of individual genetic constitution on disease and therapeutics was greatly aided by developments in pharmacogenetics and pharmacogenomicsthe study of genetic causes behind differences in how individuals respond to drugs and the study of how multiple variations within the genome affect responses to drug treatments, respectively. Using data derived from pharmacogenetics and pharmacogenomics, researchers were able to develop more objective and accurate tests fordisease diagnosis and for predicting how patients would respond to therapeutic agents. In some cases, researchers found, using genetic and other molecular data to inform diagnosis and treatment, that the development or outcome of certain diseases could be modified.
The emergence of personalized medicine was further facilitated by developments in the area of health information technology, which entails electronic processing and storage of patient data, and in the clinical uptake of personalized medicine, particularly through translational and clinical research. Advances in those areasespecially the implementation of electronic health records (EHRs), which store data on patient history, medications, test results, anddemographicswere critical to the integration of data derived from genetics and genomics research and clinical settings.
Personalized medicine is used in various ways to facilitate the prevention, diagnosis, and treatment of disease. For example, physicians can use information on family history of disease to assess a patients risk for a disease. In certain instances, family history can be used to determine whether a patient should undergo genetic testing and, based on that information, whether the individual would benefit from specific preventive measures. In the case of individuals with a family history of Lynch syndrome (a cause of hereditary colorectal cancer), for instance, detection of the causative mutation through genetic testing can be used to inform decisions about screening. For persons who carry the mutation, frequent and routine screening for evidence of precancerous lesions in the colon allows for early disease detection, which can be a lifesaving measure. Similarly, tests capable of detecting mutations in multiple genes at one time can assist in the early diagnosis of hereditary forms of breast cancer, ovarian cancer, and prostate cancer.
The term personalized medicine is sometimes considered to be synonymous with targeted therapy, a form of treatment centred on the use of drugs that target specific molecules involved in regulating the growth and spread of cancer.Among the first successful targeted therapies was the anticancerdrug imatinib, which istailored to patients with chronic myelogenousleukemia(CML) who carry anenzymecalled BCR-ABLtyrosinekinase, a protein produced by a cytogenetic abnormality known as the Philadelphiachromosome. Imatinib blocks the proliferation of CML cells that possess themutated kinase, effectively reversing the abnoramalitys cancerous effects.
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Another example of personalized medicine applied to therapeutics is the use of genotyping to identify variations in enzymes that alter a patients sensitivity to the commonly prescribed anticoagulant drug warfarin. Information about variations in warfarin-metabolizing enzymes can be used to help guide decisions about the amount of the drug that a patient needs to receive in order to achieve the desired effect.
Personalized medicine faces significant challenges. For example, compared with the HGP reference sequence of the human genome, each individual persons genome houses roughly three to five million variations. Thus, attributing disease causation or therapeutic response to a given genetic variant requires careful analysis and interpretation across multiple disciplines. Moreover, genomes vary across geographic and ethnic populations and are influenced by environmental factors; thus, an individual variation identified within a given population may have very different impacts on disease in another population, based on ethnic or geographic factors.
Technological issues also continue to challenge advances in personalized medicine. The structure of EHR data, for example, can impact its utility. Access to and analysis of genomic data in EHRs may be limited by the presentation of genomic test results as a summary that includes relevant observations but excludes raw data and by the lack of information on details such as patient lifestyle and behaviour, which are essential to the accurateinterpretation of genomic information.
Various ethical issues are associated with personalized medicine. Of particular concern is that the majority of genomic studies historically have focused on populations of European descent, with significant underrepresentation of racial and ethnic minorities. This unevenness in representation can impact algorithms used to guide decisions about drug selection and dosing regimens, potentially resulting in ineffective treatment and poorer outcomes for patients whose genetic backgrounds and lifestyles differ from more thoroughly studied groups.
Other ethical issues surround privacy and security concerns, mainly involving the use of EHRs. For example, a breachin an EHR system could result in the release of personal information and health data as well as information about health care providers.Personalized medicine also carries high costs and therefore is potentially inaccessible for patients who lack health insurance and financially out of reach for less-developed countries with limited health resources.
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Personalized medicine | Definition, Origins, Examples, & Ethical ...
In 2022, personalized medicines (PM) made up more than 34% of new therapeutics approved by the Food and Drug Administration (FDA). PM is defined by the Personalized Medicines Coalition as the use of diagnostic tests to determine which medical treatments will work best for each patient or [the] use [of] medical interventions to alter molecular mechanisms that impact health. This emerging approach to healthcare is growing rapidly and having an important impact on patients, practitioners, and health systems. While many healthcare providers have already discovered the value of PM for their patients, others may remain unsure or unconvinced. The primary rationale for using PM is that the standard-of-care therapy may work well for some patients, but for others, it may have lower efficacy or higher risk for side effects due to patients biological differences. , This is the subset of patients who could benefit the most from PM.
At USP, the Healthcare Quality & Safety (HQS) Center of Excellence has developed a science-based roadmap for personalized medicine that considers how USP can evolve standards to address new modalities of medicine and close important gaps in these treatments that help ensure quality patient care. Waypoints on this roadmap include examining established standards, building collaborations with key stakeholders, identifying volunteers for future DTx standards work at USP, holding roundtable discussions, drafting Stimuli articles, and developing workplan focus areas for the HQS Expert Committees. USP has a long-standing history of healthcare standards including those for compounding preparations that are tailored to meet the unique needs of patients who may not otherwise have access to their medications. Currently, USP is exploring other specialized areas within PM, including pharmacogenomics, digital therapeutics, and 3D-printed medications to name a few.
Pharmacogenomics
Pharmacogenomics (PGx) is the study of how a patients genes can affect drug therapy. From a sample of saliva, cheek cells, or blood, scientists can extract a patients DNA and sequence it to understand how that individuals genes are similar to or different from genes of other patients. The individuals genetic results are then considered by healthcare providers, in combination with other data about the patient and their medical condition, to select the most appropriate drug therapy for them.
Based on decades of research with populations around the globe, healthcare providers can now use PGx to make predictions of an individuals personalized response to medications. For example, PGx can help predict the amount of drug available in the patients body, which can determine both its therapeutic effect(s) and likelihood to cause side effects. This is based on the observation that multiple people who take the same dose of the same medication may metabolize, transport, bind, or otherwise interact with drugs differently, leading to different amounts of drug in their bodies.
PGx presents opportunities for USP to collaborate with key stakeholders who are already developing PGx standards and guidelines and to apply its standards-setting process to establish alignment and consistency in PGx standards. Specifically, some of the standards and guidelines that USP could help develop are the 1) naming of genetic biomarkers and PGx terminology, 2) labeling of medicines to incorporate PGx information, 3) incorporation of PGx into healthcare information technology such as electronic health records and clinical decision support, and 4) diversification of clinical trials so that PGx information is not limited to patients of common ancestries, such as those of European ancestry. USP is well-positioned to engage a wide audience while increasing the reliability of and confidence in the utilization of PGx. This will help support PGx implementation, including payment and reimbursement, both nationally and globally.
Digital Therapeutics
Digital therapeutics (DTx) are defined by the International Organization for Standardization as health software intended to treat or alleviate a disease, disorder, condition, or injury by generating and delivering a medical intervention that has a demonstrable positive therapeutic impact on a patients health. The DTx landscape is evolving rapidly, with more than 40 prescription digital therapeutics already available in the U.S. DTx is projected to have a cumulative annual growth rate of up to 25.4% in the U.S. market by 2030, and this growth is not limited to the U.S., as the global DTx market is expected to expand by 31.6% by 2027. Due to this burgeoning expansion, DTx products are increasingly in need of robust standards to underpin the creation of high-quality products and the delivery of comprehensive care on a large scale.
USP is actively engaged in researching the landscape of DTx and has started an investigation into opportunities for DTx standards to improve the quality of care provided to patients. USP has identified areas for potential standards that include establishing, or supporting the establishment of, global DTx definitions including outcomes used in clinical studies of DTx products. These potential standards may also include key aspects such as security and privacy, promoting awareness and education among healthcare providers and patients, adopting consistent labeling practices, setting standard technology proficiency requirements, addressing issues related to data integrity and code authenticity to deter counterfeit DTx products, facilitating interoperability among the various clinical systems used in healthcare around the world, and integrating DTx into healthcare delivery systems such as existing software and devices.
These elements would create a comprehensive framework for seamlessly integrating DTx within the healthcare industry, as well as for its development and regulation. USP is also maintaining awareness of the current position of DTx, understanding the potential role of standards in DTx product growth, identifying factors that can expedite progress, recognizing and addressing barriers, and determining the competencies needed to navigate the ever-changing, technology-driven market.
What comes next
USP is currently engaging with stakeholder leaders to develop Stimuli Articles related to its research involving potential standards for PGx and DTx. These future articles will feature in the USP-PF and will solicit public comment to promote stakeholder engagement. In the meantime, USP encourages interested parties to reach out for more information as USP approaches its new 2025-2030 cycle.
For further information about USPs work on personalized medicine, visit our webpage, sign up for the HQS newsletter, or contact:
Blaine GroatEmail: blaine.groat@usp.org
Yasmin HaidarbaigiEmail: Yasmin.haidarbaigi@usp.org
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i Personalized Medicine Coalition. Personalized Medicine at FDA: the Scope & Significance of Progress in 2022.report.pdf (personalizedmedicinecoalition.org) Accessed November 10, 2023. Personalized Medicine Coalition.ii Personalized Medicine 101.https://www.personalizedmedicinecoalition.org/personalized-medicine-101/. Accessed November 6, 2023.iii Schork, N. Personalized medicine: Time for one-person trials. Nature 520, 609611 (2015).https://doi.org/10.1038/520609a.iv US Department of Health and Human Services. National Action Plan for Adverse Drug Event Prevention.Washington (DC): 2014; pharmacogenomics working group whose mission is to develop a 56.v International Organization for Standardization. "Health Informatics Personalized Digital Health DigitalTherapeutics Health Software Systems." ISO/TR 11147:Edition 1, 2023, https://www.iso.org/obp/ui/#iso:std:iso:tr:11147:ed-1:v1:en.vi Liesch J, Murphy D, Singh V. Under Pressure: Prescription Digital Therapeutics - How an analysis of the U.S. PDTlandscape indicates mounting pressure for a make-or-break next 3 years. Blue Matter. 2022.vii Digital Therapeutics Market Size, Share, and Analysis Report. Grand View Research, 2023. Accessed viahttps://www.grandviewresearch.com/industry-analysis/digital-therapeutics-market on Sep 05, 2023.viii Digital Therapeutics Market Revenue Forecast: Latest Industry Updates. Markets and Markets, 2023. Accessed via https://www.marketsandmarkets.com/Market-Reports/digital-therapeutics-market-51646724.html on Sep 05, 2023.
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Innovating for Individual Care: The Impact of USP on Personalized Medicine
Where Does Capricor Therapeutics Inc (CAPR) Stock Fall in the Biotechnology Field After It Has Risen 24.73% This Week? InvestorsObserver
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Where Does Capricor Therapeutics Inc (CAPR) Stock Fall in the Biotechnology Field After It Has Risen 24.73% This Week? - InvestorsObserver
Stem Cell Investig. 2019; 6: 19.
1Stem Cell Research Center, Tabriz University of Medical Sciences, Tabriz, Iran;
2Department of Clinical Sciences, Faculty of Veterinary Medicine, University of Tabriz, Tabriz, Iran;
2Department of Clinical Sciences, Faculty of Veterinary Medicine, University of Tabriz, Tabriz, Iran;
3Hematology and Oncology Research Center, Tabriz University of Medical Sciences, Tabriz, Iran
1Stem Cell Research Center, Tabriz University of Medical Sciences, Tabriz, Iran;
2Department of Clinical Sciences, Faculty of Veterinary Medicine, University of Tabriz, Tabriz, Iran;
3Hematology and Oncology Research Center, Tabriz University of Medical Sciences, Tabriz, Iran
Contributions: (I) Conception and design: E Fathi, R Farahzadi; (II) Administrative support: E Fathi, R Farahzadi; (III) Provision of study materials or patients: None; (IV) Collection and assembly of data: R Farahzadi, N Rajabzadeh; (V) Data analysis and interpretation: All authors; (VI) Manuscript writing: All authors; (VII) Final approval of manuscript: All authors.
#These authors contributed equally to this work.
Received 2018 Nov 11; Accepted 2019 Mar 17.
Recent developments in the stem cell biology provided new hopes in treatment of diseases and disorders that yet cannot be treated. Stem cells have the potential to differentiate into various cell types in the body during age. These provide new cells for the body as it grows, and replace specialized cells that are damaged. Since mesenchymal stem cells (MSCs) can be easily harvested from the adipose tissue and can also be cultured and expanded in vitro they have become a good target for tissue regeneration. These cells have been widespread used for cell transplantation in animals and also for clinical trials in humans. The purpose of this review is to provide a summary of our current knowledge regarding the important and types of isolated stem cells from different sources of animal models such as horse, pig, goat, dog, rabbit, cat, rat, mice etc. In this regard, due to the widespread use and lot of attention of MSCs, in this review, we will elaborate on use of MSCs in veterinary medicine as well as in regenerative medicine. Based on the studies in this field, MSCs found wide application in treatment of diseases, such as heart failure, wound healing, tooth regeneration etc.
Keywords: Mesenchymal stem cells (MSCs), animal model, cell-based therapy, regenerative medicine
Stem cells are one of the main cells of the human body that have ability to grow more than 200 types of body cells (1). Stem cells, as non-specialized cells, can be transformed into highly specialized cells in the body (2). In the other words, Stem cells are undifferentiated cells with self-renewal potential, differentiation into several types of cells and excessive proliferation (3). In the past, it was believed that stem cells can only differentiate into mature cells of the same organ. Today, there are many evidences to show that stem cells can differentiate into the other types of cell as well as ectoderm, mesoderm and endoderm. The numbers of stem cells are different in the tissues such as bone marrow, liver, heart, kidney, and etc. (3,4). Over the past 20 years, much attention has been paid to stem cell biology. Therefore, there was a profound increase in the understanding of its characteristics and the therapeutic potential for its application (5). Today, the utilization of these cells in experimental research and cell therapy represents in such disorders including hematological, skin regeneration and heart disease in both human and veterinary medicine (6).The history of stem cells dates back to the 1960s, when Friedenstein and colleagues isolated, cultured and differentiated to osteogenic cell lineage of bone marrow-derived cells from guinea pigs (7). This project created a new perspective on stem cell research. In the following, other researchers discovered that the bone marrow contains fibroblast-like cells with congenic potential in vitro, which were capable of forming colonies (CFU-F) (8). For over 60 years, transplantation of hematopoietic stem cells (HSCs) has been the major curative therapy for several genetic and hematological disorders (9). Almost in 1963, Till and McCulloch described a single progenitor cell type in the bone marrow which expand clonally and give rise to all lineages of hematopoietic cells. This research represented the first characterization of the HSCs (10). Also, the identification of mouse embryonic stem cells (ESCs) in 1981 revolutionized the study of developmental biology, and mice are now used extensively as one of the best option to study stem cell biology in mammals (11). Nevertheless, their application a model, have limitations in the regenerative medicine. But this model, relatively inexpensive and can be easily manipulated genetically (12). Failure to obtain a satisfactory result in the selection of many mouse models, to recapitulate particular human disease phenotypes, has forced researchers to investigate other animal species to be more probably predictive of humans (13). For this purpose, to study the genetic diseases, the pig has been currently determined as one the best option of a large animal model (14).
Stem cells, based on their differentiation ability, are classified into different cell types, including totipotent, pluripotent, multipotent, or unipotent. Also, another classification of these cells are based on the evolutionary stages, including embryonic, fetal, infant or umbilical cord blood and adult stem cells (15). shows an overview of stem cells classifications based on differentiation potency.
An overview of the stem cell classification. Totipotency: after fertilization, embryonic stem cells (ESCs) maintain the ability to form all three germ layers as well as extra-embryonic tissues or placental cells and are termed as totipotent. Pluripotency: these more specialized cells of the blastocyst stage maintain the ability to self-renew and differentiate into the three germ layers and down many lineages but do not form extra-embryonic tissues or placental cells. Multipotency: adult or somatic stem cells are undifferentiated cells found in postnatal tissues. These specialized cells are considered to be multipotent; with very limited ability to self-renew and are committed to lineage species.
Toti-potent cells have the potential for development to any type of cell found in the organism. In the other hand, the capacity of these cells to develop into the three primary germ cell layers of the embryo and into extra-embryonic tissues such as the placenta is remarkable (15).
The pluripotent stem cells are kind of stem cells with the potential for development to approximately all cell types. These cells contain ESCs and cells that are isolated from the mesoderm, endoderm and ectoderm germ layers that are organized in the beginning period of ESC differentiation (15).
The multipotent stem cells have less proliferative potential than the previous two groups and have ability to produce a variety of cells which limited to a germinal layer [such as mesenchymal stem cells (MSCs)] or just a specific cell line (such as HSCs). Adult stem cells are also often in this group. In the word, these cells have the ability to differentiate into a closely related family of cells (15).
Despite the increasing interest in totipotent and pluripotent stem cells, unipotent stem cells have not received the most attention in research. A unipotent stem cell is a cell that can create cells with only one lineage differentiation. Muscle stem cells are one of the example of this type of cell (15). The word uni is derivative from the Latin word unus meaning one. In adult tissues in comparison with other types of stem cells, these cells have the lowest differentiation potential. The unipotent stem cells could create one cell type, in the other word, these cells do not have the self-renewal property. Furthermore, despite their limited differentiation potential, these cells are still candidates for treatment of various diseases (16).
ESCs are self-renewing cells that derived from the inner cell mass of a blastocyst and give rise to all cells during human development. It is mentioned that these cells, including human embryonic cells, could be used as suitable, promising source for cell transplantation and regenerative medicine because of their unique ability to give rise to all somatic cell lineages (17). In the other words, ESCs, pluripotent cells that can differentiate to form the specialized of the various cell types of the body (18). Also, ESCs capture the imagination because they are immortal and have an almost unlimited developmental potential. Due to the ethical limitation on embryo sampling and culture, these cells are used less in research (19).
HSCs are multipotent cells that give rise to blood cells through the process of hematopoiesis (20). These cells reside in the bone marrow and replenish all adult hematopoietic lineages throughout the lifetime of the human and animal (21). Also, these cells can replenish missing or damaged components of the hematopoietic and immunologic system and can withstand freezing for many years (22).The mammalian hematopoietic system containing more than ten different mature cell types that HSCs are one of the most important members of this. The ability to self-renew and multi-potency is another specific feature of these cells (23).
Adult stem cells, as undifferentiated cells, are found in numerous tissues of the body after embryonic development. These cells multiple by cell division to regenerate damaged tissues (24). Recent studies have been shown that adult stem cells may have the ability to differentiate into cell types from various germ layers. For example, bone marrow stem cells which is derived from mesoderm, can differentiate into cell lineage derived mesoderm and endoderm such as into lung, liver, GI tract, skin, etc. (25). Another example of adult stem cells is neural stem cells (NSCs), which is derived from ectoderm and can be differentiate into another lineage such as mesoderm and endoderm (26). Therapeutic potential of adult stem cells in cell therapy and regenerative medicine has been proven (27).
For the first time in the late 1990s, CSCs were identified by John Dick in acute myeloid diseases. CSCs are cancerous cells that found within tumors or hematological cancers. Also, these cells have the characteristics of normal stem cells and can also give rise to all cell types found in a particular cancer sample (28). There is an increasing evidence supporting the CSCs hypothesis. Normal stem cells in an adult living creature are responsible for the repair and regeneration of damaged as well as aged tissues (29). Many investigations have reported that the capability of a tumor to propagate and proliferate relies on a small cellular subpopulation characterized by stem-like properties, named CSCs (30).
Embryonic connective tissue contains so-called mesenchymes, from which with very close interactions of endoderm and ectoderm all other connective and hematopoietic tissues originate, Whereas, MSCs do not differentiate into hematopoietic cell (31). In 1924, Alexander A. Maxi mow used comprehensive histological detection to identify a singular type of precursor cell within mesenchyme that develops into various types of blood cells (32). In general, MSCs are type of cells with potential of multi-lineage differentiation and self-renewal, which exist in many different kinds of tissues and organs such as adipose tissue, bone marrow, skin, peripheral blood, fallopian tube, cord blood, liver and lung et al. (4,5). Today, stem cells are used for different applications. In addition to using these cells in human therapy such as cell transplantation, cell engraftment etc. The use of stem cells in veterinary medicine has also been considered. The purpose of this review is to provide a summary of our current knowledge regarding the important and types of isolated stem cells from different sources of animal models such as horse, pig, goat, dog, rabbit, cat, rat, mice etc. In this regard, due to the widespread use and lot of attention of MSCs, in this review, we will elaborate on use of MSCs in veterinary medicine.
The isolation method, maintenance and culture condition of MSCs differs from the different tissues, these methods as well as characterization of MSCs described as (36). MSCs could be isolated from the various tissues such as adipose tissue, bone marrow, umbilical cord, amniotic fluid etc. (37).
Diagram for adipose tissue-derived mesenchymal stem cell isolation (3).
Diagram for bone marrow-derived MSCs isolation (33). MSC, mesenchymal stem cell.
Diagram for umbilical cord-derived MSCs isolation (34). MSC, mesenchymal stem cell.
Diagram for isolation of amniotic fluid stem cells (AFSCs) (35).
Diagram for MSCs characterization (35). MSC, mesenchymal stem cell.
The diversity of stem cell or MSCs sources and a wide aspect of potential applications of these cells cause to challenge for selecting an appropriate cell type for cell therapy (38). Various diseases in animals have been treated by cell-based therapy. However, there are immunity concerns regarding cell therapy using stem cells. Improving animal models and selecting suitable methods for engraftment and transplantation could help address these subjects, facilitating eventual use of stem cells in the clinic. Therefore, for this purpose, in this section of this review, we provide an overview of the current as well as previous studies for future development of animal models to facilitate the utilization of stem cells in regenerative medicine (14). Significant progress has been made in stem cells-based regenerative medicine, which enables researchers to treat those diseases which cannot be cured by conventional medicines. The unlimited self-renewal and multi-lineage differentiation potential to other types of cells causes stem cells to be frontier in regenerative medicine (24). More researches in regenerative medicine have been focused on human cells including embryonic as well as adult stem cells or maybe somatic cells. Today there are versions of embryo-derived stem cells that have been reprogrammed from adult cells under the title of pluripotent cells (39). Stem cell therapy has been developed in the last decade. Nevertheless, obstacles including unwanted side effects due to the migration of transplanted cells as well as poor cell survival have remained unresolved. In order to overcome these problems, cell therapy has been introduced using biocompatible and biodegradable biomaterials to reduce cell loss and long-term in vitro retention of stem cells.
Currently in clinical trials, these biomaterials are widely used in drug and cell-delivery systems, regenerative medicine and tissue engineering in which to prevent the long-term survival of foreign substances in the body the release of cells are controlled (40).
Today, the incidence and prevalence of heart failure in human societies is a major and increasing problem that unfortunately has a poor prognosis. For decades, MSCs have been used for cardiovascular regenerative therapy as one of the potential therapeutic agents (41). Dhein et al. [2006] found that autologous bone marrow-derived mesenchymal stem cells (BMSCs) transplantation improves cardiac function in non-ischemic cardiomyopathy in a rabbit model. In one study, Davies et al. [2010] reported that transplantation of cord blood stem cells in ovine model of heart failure, enhanced the function of heart through improvement of right ventricular mass, both systolic and diastolic right heart function (42). In another study, Nagaya et al. [2005] found that MSCs dilated cardiomyopathy (DCM), possibly by inducing angiogenesis and preventing cardial fibrosis. MSCs have a tremendous beneficial effect in cell transplantation including in differentiating cardiomyocytes, vascular endothelial cells, and providing anti-apoptotic as well angiogenic mediators (43). Roura et al. [2015] shown that umbilical cord blood mesenchymal stem cells (UCBMSCs) are envisioned as attractive therapeutic candidates against human disorders progressing with vascular deficit (44). Ammar et al., [2015] compared BMSCs with adipose tissue-derived MSCs (ADSCs). It was demonstrated that both BMSCs and ADSCs were equally effective in mitigating doxorubicin-induced cardiac dysfunction through decreasing collagen deposition and promoting angiogenesis (45).
There are many advantages of small animal models usage in cardiovascular research compared with large animal models. Small model of animals has a short life span, which allow the researchers to follow the natural history of the disease at an accelerated pace. Some advantages and disadvantages are listed in (46).
Despite of the small animal model, large animal models are suitable models for studies of human diseases. Some advantages and disadvantages of using large animal models in a study protocol planning was elaborated in (47).
Chronic wound is one of the most common problem and causes significant distress to patients (48). Among the types of tissues that stem cells derived it, dental tissuederived MSCs provide good sources of cytokines and growth factors that promote wound healing. The results of previous studies showed that stem cells derived deciduous teeth of the horse might be a novel approach for wound care and might be applied in clinical treatment of non-healing wounds (49). However, the treatment with stem cells derived deciduous teeth needs more research to understand the underlying mechanisms of effective growth factors which contribute to the wound healing processes (50). This preliminary investigation suggests that deciduous teeth-derived stem cells have the potential to promote wound healing in rabbit excisional wound models (49). In the another study, Lin et al. [2013] worked on the mouse animal model and showed that ADSCs present a potentially viable matrix for full-thickness defect wound healing (51).
Many studies have been done on dental reconstruction with MSCs. In one study, Khorsand et al. [2013] reported that dental pulp-derived stem cells (DPSCs) could promote periodontal regeneration in canine model. Also, it was shown that canine DPSCs were successfully isolated and had the rapid proliferation and multi-lineage differentiation capacity (52). Other application of dental-derived stem cells is shown in .
Diagram for application of dental stem cell in dentistry/regenerative medicine (53).
As noted above, stem cells have different therapeutic applications and self-renewal capability. These cells can also differentiate into the different cell types. There is now a great hope that stem cells can be used to treat diseases such as Alzheimer, Parkinson and other serious diseases. In stem cell-based therapy, ESCs are essentially targeted to differentiate into functional neural cells. Today, a specific category of stem cells called induced pluripotent stem (iPS) cells are being used and tested to generate functional dopamine neurons for treating Parkinson's disease of a rat animal model. In addition, NSC as well as MSCs are being used in neurodegenerative disorder therapies for Alzheimers disease, Parkinsons disease, and stroke (54). Previous studies have shown that BMSCs could reduce brain amyloid deposition and accelerate the activation of microglia in an acutely induced Alzheimers disease in mouse animal model. Lee et al. [2009] reported that BMSCs can increase the number of activated microglia, which effective therapeutic vehicle to reduce A deposits in AD patients (55). In confirmation of previous study, Liu et al. [2015] showed that transplantation of BMSCs in brain of mouse model of Alzheimers disease cause to decrease in amyloid beta deposition, increase in brain-derived neurotrophic factor (BDNF) levels and improvements in social recognition (56). In addition of BMSCs, NSCs have been proposed as tools for treating neurodegeneration disease because of their capability to create an appropriate cell types which transplanted. kerud et al. [2001] demonstrated that NSCs efficiently express high level of glial cell line-derived neurotrophic factor (GDNF) in vivo, suggesting a use of these cells in the treatment of neurodegenerative disorders, including Parkinsons disease (57). In the following, Venkataramana et al. [2010] transplanted BMSCs into the sub lateral ventricular zones of seven Parkinsons disease patients and reported encouraging results (58).
The human body is fortified with specialized cells named MSCs, which has the ability to self-renew and differentiate into various cell types including, adipocyte, osteocyte, chondrocyte, neurons etc. In addition to mentioned properties, these cells can be easily isolated, safely transplanted to injured sites and have the immune regulatory properties. Numerous in vitro and in vivo studies in animal models have successfully demonstrated the potential of MSCs for various diseases; however, the clinical outcomes are not very encouraging. Based on the studies in the field of stem cells, MSCs find wide application in treatment of diseases, such as heart failure, wound healing, tooth regeneration and etc. In addition, these cells are particularly important in the treatment of the sub-branch neurodegenerative diseases like Alzheimer and Parkinson.
The authors wish to thank staff of the Stem Cell Research Center, Tabriz University of Medical Sciences, Tabriz, Iran.
Funding: The project described was supported by Grant Number IR.TBZMED.REC.1396.1218 from the Stem Cell Research Center, Tabriz University of Medical Sciences, Tabriz, Iran.
Ethical Statement: The authors are accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.
Conflicts of Interest: The authors have no conflicts of interest to declare.
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