scholarly journals AMRAD: Pioneer of Australian biotechnology

2001 ◽  
Vol 7 (3) ◽  
Author(s):  
Amanda Caples ◽  
John Grace
Keyword(s):  
Clinical Trials ◽  
International Development ◽  
Service Provision ◽  
Healthcare Sector ◽  
Proof Of Concept ◽  
Publicly Funded ◽  
Biotechnology Company ◽  
Research Activities ◽  
Research Institutes ◽  
Human Healthcare

AMRAD is a biotechnology company in the human healthcare sector. Its drug discovery projects are focused on novel cytokine therapies and small molecule anti-viral treatments. The company has a portfolio of early- and late-stage projects and its strategy is to enter into collaborative arrangements with international development partners upon generating proof-of-concept data in clinical trials. AMRAD differs from the typical 'biotech model' insofar as it was initially established as a private technology transfer organisation to serve the commercialisation needs of the publicly funded founding medical research institutes. In return for shares in the company the Member Institutes gave AMRAD a first right of invitation to their research activities. Although the Member Institute arrangement remains an important asset in feeding AMRAD's R&D pipeline, AMRAD's business model has evolved from essentially a service provision role to being actively involved in the progress of new medicines through human clinical trials.

Knowledge transfer and dissemination: insights from the dairy sector in the UK and India

2003 ◽  
Vol 2003 ◽  
pp. 208-208
Author(s):  
C.J. Garforth
Keyword(s):  
Developing Countries ◽  
Technology Transfer ◽  
Knowledge Transfer ◽  
International Development ◽  
Publicly Funded ◽  
Methodological Issues ◽  
Research Institutes ◽  
New Knowledge ◽  
Research Programmes ◽  
The Uk

‘Knowledge transfer’ and ‘uptake’ are high on the agenda of government departments and research institutes. In the U.K., the Department for Environment, Food and Rural Affairs (DEFRA) is concerned that new knowledge generated by publicly funded research is not being taken up by the industry and has funded a series of knowledge transfer initiatives. On the international front, the Department for International Development (DFID) is pressing managers of its various research programmes to demonstrate that the outputs of research are being used by farmers in developing countries and are having an impact on their livelihoods. International institutes are increasing the proportion of their resources devoted to technology transfer and dissemination programmes. These concerns and initiatives raise important conceptual, institutional and methodological issues.

scholarly journals Targeting for Success: Demonstrating Proof-of-Concept with Mechanistic Early Phase Clinical Pharmacology Studies for Disease-Modification in Neurodegenerative Disorders

2021 ◽  
Vol 22 (4) ◽  
pp. 1615
Author(s):  
Maurits F. J. M. Vissers ◽  
Jules A. A. C. Heuberger ◽  
Geert Jan Groeneveld
Keyword(s):  
Clinical Trials ◽  
Failure Rate ◽  
Neurodegenerative Disorders ◽  
Early Phase ◽  
Proof Of Concept ◽  
Mesh Terms ◽  
Disease Modifying ◽  
Early Phase Clinical Trials ◽  
Pharmacodynamic Biomarkers ◽  
Response Biomarkers

The clinical failure rate for disease-modifying treatments (DMTs) that slow or stop disease progression has been nearly 100% for the major neurodegenerative disorders (NDDs), with many compounds failing in expensive and time-consuming phase 2 and 3 trials for lack of efficacy. Here, we critically review the use of pharmacological and mechanistic biomarkers in early phase clinical trials of DMTs in NDDs, and propose a roadmap for providing early proof-of-concept to increase R&D productivity in this field of high unmet medical need. A literature search was performed on published early phase clinical trials aimed at the evaluation of NDD DMT compounds using MESH terms in PubMed. Publications were selected that reported an early phase clinical trial with NDD DMT compounds between 2010 and November 2020. Attention was given to the reported use of pharmacodynamic (mechanistic and physiological response) biomarkers. A total of 121 early phase clinical trials were identified, of which 89 trials (74%) incorporated one or multiple pharmacodynamic biomarkers. However, only 65 trials (54%) used mechanistic (target occupancy or activation) biomarkers to demonstrate target engagement in humans. The most important categories of early phase mechanistic and response biomarkers are discussed and a roadmap for incorporation of a robust biomarker strategy for early phase NDD DMT clinical trials is proposed. As our understanding of NDDs is improving, there is a rise in potentially disease-modifying treatments being brought to the clinic. Further increasing the rational use of mechanistic biomarkers in early phase trials for these (targeted) therapies can increase R&D productivity with a quick win/fast fail approach in an area that has seen a nearly 100% failure rate to date.

Multi-disciplinarity as a way to strengthen the positions of Russian technical universities in international rankings

2021 ◽  
Vol 20 (10) ◽  
pp. 1798-1817
Author(s):  
Leonid B. SOBOLEV
Keyword(s):  
Research Universities ◽  
Multidisciplinary Research ◽  
Global Trends ◽  
Russian Academy Of Sciences ◽  
Research Activities ◽  
Research Institutes ◽  
Integrated Research ◽  
World Class ◽  
Academy Of Sciences ◽  
Russian Research

Subject. The article is devoted to the problem of improving the ranking of the leading Russian technical universities by transforming them into multidisciplinary research universities with the limitation of technical faculties (programs) to 40–50% of the admission plan of entrants. Objectives. The study focuses on improving the position of Russian technical universities in international rankings, providing an opportunity for students to change their specialty or shape it, according to the requirements of the employer. Methods. I analyzed the structure of world-class technical universities in terms of differentiation of specialties and methods of improving the rankings. Results. The analysis shows that the leading positions in international rankings belong to multidisciplinary research universities that provide training and conduct integrated research at the intersection of different sciences. Conclusions. There is a need for gradual reforms of Russian research technical universities, in terms of compliance with global trends in multidisciplinarity, differentiation of funding, and research activities. Such reforms can be carried out in the form of mergers and creation of network structures on the basis of agreements on cooperation between technical universities and research institutes of the Russian Academy of Sciences.

scholarly journals Hemophilia gene therapy: ushering in a new treatment paradigm?

Hematology ◽  
2021 ◽  
Vol 2021 (1) ◽  
pp. 226-233
Author(s):  
Lindsey A. George
Keyword(s):  
Clinical Trial ◽  
Gene Therapy ◽  
Clinical Trials ◽  
Hemophilia A ◽  
Full Potential ◽  
Proof Of Concept ◽  
Aav Vector ◽  
Treatment Paradigm ◽  
Clinical Trial Enrollment ◽  
New Treatment

Abstract After 3 decades of clinical trials, repeated proof-of-concept success has now been demonstrated in hemophilia A and B gene therapy. Current clinical hemophilia gene therapy efforts are largely focused on the use of systemically administered recombinant adeno-associated viral (rAAV) vectors for F8 or F9 gene addition. With multiple ongoing trials, including licensing studies in hemophilia A and B, many are cautiously optimistic that the first AAV vectors will obtain regulatory approval within approximately 1 year. While supported optimism suggests that the goal of gene therapy to alter the paradigm of hemophilia care may soon be realized, a number of outstanding questions have emerged from clinical trial that are in need of answers to harness the full potential of gene therapy for hemophilia patients. This article reviews the use of AAV vector gene addition approaches for hemophilia A and B, focusing specifically on information to review in the process of obtaining informed consent for hemophilia patients prior to clinical trial enrollment or administering a licensed AAV vector.

scholarly journals Web Based Data Capture for Clinical Research

10.28945/3201 ◽  
2008 ◽  
Author(s):  
Stephen Smith ◽  
Samuel Sambasivam
Keyword(s):  
Clinical Trials ◽  
Clinical Research ◽  
Medical Device ◽  
Rapid Development ◽  
Research Data ◽  
Data Capture ◽  
Electronic Data Capture ◽  
Proof Of Concept ◽  
Web Based ◽  
Tools And Techniques

Electronic Data Capture (EDC) is increasingly being used in the pharmaceutical, biotech and medical device industries to gather research data worldwide from doctors, hospitals and universities participating in clinical trials. In this highly regulated environment, all systems and software must be thoroughly tested and validated, a task that is burdensome in terms of time and cost. Starting with database structures that are designed to be copied easily, this paper proposes a simple framework that allows for rapid development and minimal testing. The framework includes tools for building modules, for copying modules from one trial to the next, and tools to validate that the modules are the same as modules that have been fully tested previously. A proof-of-concept prototype has been built to demonstrate certain tools and techniques that can be used when designing and building a simplified EDC interface.

scholarly journals Managing Clinical Trials for Alzheimer’s Disease During the COVID-19 Crisis: Experience at Fundació ACE in Barcelona, Spain

2020 ◽  
Vol 77 (4) ◽  
pp. 1805-1813
Author(s):  
Carla Abdelnour ◽  
Ester Esteban de Antonio ◽  
Alba Pérez-Cordón ◽  
Asunción Lafuente ◽  
Mar Buendía ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Ad Hoc ◽  
Strategic Plan ◽  
Lessons Learned ◽  
Drop Out ◽  
Rt Pcr ◽  
Serological Tests ◽  
Clinical Trial Research ◽  
Research Activities ◽  
The Impact

Background: The COVID-19 pandemic has brought great disruption to health systems worldwide. This affected ongoing clinical research, particularly among those most vulnerable to the pandemic, like dementia patients. Fundació ACE is a research center and memory clinic based in Barcelona, Spain, one of the hardest-hit countries. Objective: To describe the ad-hoc strategic plan developed to cope with this crisis and to share its outcomes. Methods: We describe participants’ clinical and demographic features. Additionally, we explain our strategic plan aimed at minimizing the impact on clinical trial research activities, which included SARS-CoV-2 RT-PCR and IgG serological tests to all participants and personnel. The outcomes of the plan are described in terms of observed safety events and drop-outs during the study period. Results: A total of 130 patients were participating in 16 active clinical trials in Fundació ACE when the lockdown was established. During the confinement, we performed 1018 calls to the participants, which led to identify adverse events in 26 and COVID-19 symptoms in 6. A total of 83 patients (64%) could restart on-site visits as early as May 11, 2020. All SARS-CoV-2 RT-PCR diagnostic tests performed before on-site visits were negative and only three IgG serological tests were positive. Throughout the study period, we only observed one drop-out, due to an adverse event unrelated to COVID-19. Discussion: The plan implemented by Fundació ACE was able to preserve safety and integrity of ongoing clinical trials. We must use the lessons learned from the pandemic and design crisis-proof protocols for clinical trials.

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