Highlights of the 12th Japan Bioanalysis Forum Symposium

Bioanalysis ◽  
2021 ◽  
Author(s):  
Hitoshi Uchiyama ◽  
Takeru Yamaguchi ◽  
Koji Arai ◽  
Tomoko Arakawa ◽  
Harue Igarashi ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Clinical Trials ◽  
Cancer Genome ◽  
Genome Medicine ◽  
Middle Molecules ◽  
Biomarker Analysis ◽  
Key Issues ◽  
Pharmaceutical Industries ◽  
Young Researchers ◽  
Patient Centric

Approximately 300 people associated with pharmaceutical industries, contractors, academic institutions and regulatory authorities attended the 12th Japan Bioanalysis Forum Symposium. The webinar was conducted from 9 to 11 March 2021. The theme of the symposium was ‘for the next generation’, and the event provided ‘an opportunity for young researchers in bioanalysis (including students)’ and ‘an opportunity to discuss new frontiers of bioanalysis’. The speakers focused on hot topics of bioanalysis, including biomarker analysis, patient centric sampling, virtual clinical trials, gene therapy, cancer genome medicine and therapeutic middle molecules. The symposium presented a platform for the discussion of the prospects and challenges facing bioanalysts working in the field of pharmacokinetics. This report presents the key issues discussed.

scholarly journals The Application of Adeno-Associated Viral Vector Gene Therapy to the Treatment of Fragile X Syndrome

2019 ◽  
Vol 9 (2) ◽  
pp. 32 ◽  
Author(s):  
David Hampson ◽  
Alexander Hooper ◽  
Yosuke Niibori
Keyword(s):  
Gene Therapy ◽  
Clinical Trials ◽  
Fragile X Syndrome ◽  
Viral Vectors ◽  
Viral Vector ◽  
Fragile X ◽  
Animal Models Of Disease ◽  
Key Issues ◽  
Full Correction ◽  
Models Of Disease

Viral vector-mediated gene therapy has grown by leaps and bounds over the past several years. Although the reasons for this progress are varied, a deeper understanding of the basic biology of the viruses, the identification of new and improved versions of viral vectors, and simply the vast experience gained by extensive testing in both animal models of disease and in clinical trials, have been key factors. Several studies have investigated the efficacy of adeno-associated viral (AAV) vectors in the mouse model of fragile X syndrome where AAVs have been used to express fragile X mental retardation protein (FMRP), which is missing or highly reduced in the disorder. These studies have demonstrated a range of efficacies in different tests from full correction, to partial rescue, to no effect. Here we provide a backdrop of recent advances in AAV gene therapy as applied to central nervous system disorders, outline the salient features of the fragile X studies, and discuss several key issues for moving forward. Collectively, the findings to date from the mouse studies on fragile X syndrome, and data from clinical trials testing AAVs in other neurological conditions, indicate that AAV-mediated gene therapy could be a viable strategy for treating fragile X syndrome.

Towards More Successful Gene Therapy Clinical Trials for β-Thalassemia

2013 ◽  
Vol 13 (8) ◽  
pp. 1314-1330 ◽  
Author(s):  
E. Drakopoulou ◽  
E. Papanikolaou ◽  
M. Georgomanoli ◽  
N. Anagnou
Keyword(s):  
Gene Therapy ◽  
Clinical Trials

scholarly journals The United States’ Regulatory Environment Is Evolving to Accommodate a Coming Boom in Gene Therapy Research

2019 ◽  
Vol 24 (3) ◽  
pp. 147-152 ◽  
Author(s):  
Daniel Eisenman
Keyword(s):  
United States ◽  
Gene Therapy ◽  
Clinical Trials ◽  
The United States ◽  
Regulatory Environment ◽  
Evidence Based ◽  
Regulatory Structure ◽  
Regulatory Changes ◽  
Current State ◽  
Therapy Field

Introduction: A dramatic increase in the number of clinical trials involving gene-modified cell therapy and gene therapy is taking place. The field is on the verge of a boom, and the regulatory environment is evolving to accommodate the growth. Discussion: This commentary summarizes the current state of the field, including an overview of the growth. The United States (US) regulatory structure for gene therapy will be summarized, and the evolution of the oversight structure will be explained. Conclusion: The gene therapy field has recently produced its first FDA-approved therapeutics and has a pipeline of other investigational products in the final stages of clinical trials before they can be evaluated by the FDA as safe and effective therapeutics. As research continues to evolve, so must the oversight structure. Biosafety professionals and IBCs have always played key roles in contributing to the safe, evidence-based advancement of gene therapy research. With the recent regulatory changes and current surge in gene therapy research, the importance of those roles has increased dramatically.

scholarly journals Progress in Gene Therapy to Prevent Retinal Ganglion Cell Loss in Glaucoma and Leber’s Hereditary Optic Neuropathy

2018 ◽  
Vol 2018 ◽  
pp. 1-11 ◽  
Author(s):  
Sara E. Ratican ◽  
Andrew Osborne ◽  
Keith R. Martin
Keyword(s):  
Gene Therapy ◽  
Clinical Trials ◽  
Optic Nerve ◽  
Genome Editing ◽  
Optic Neuropathy ◽  
Single Gene ◽  
Leber’S Hereditary Optic Neuropathy ◽  
Leber's Hereditary Optic Neuropathy ◽  
Potential Use ◽  
Hereditary Optic Neuropathy

The eye is at the forefront of the application of gene therapy techniques to medicine. In the United States, a gene therapy treatment for Leber’s congenital amaurosis, a rare inherited retinal disease, recently became the first gene therapy to be approved by the FDA for the treatment of disease caused by mutations in a specific gene. Phase III clinical trials of gene therapy for other single-gene defect diseases of the retina and optic nerve are also currently underway. However, for optic nerve diseases not caused by single-gene defects, gene therapy strategies are likely to focus on slowing or preventing neuronal death through the expression of neuroprotective agents. In addition to these strategies, there has also been recent interest in the potential use of precise genome editing techniques to treat ocular disease. This review focuses on recent developments in gene therapy techniques for the treatment of glaucoma and Leber’s hereditary optic neuropathy (LHON). We discuss recent successes in clinical trials for the treatment of LHON using gene supplementation therapy, promising neuroprotective strategies that have been employed in animal models of glaucoma and the potential use of genome editing techniques in treating optic nerve disease.

scholarly journals Progress and prospects of gene therapy clinical trials for the muscular dystrophies

2015 ◽  
Vol 25 (R1) ◽  
pp. R9-R17 ◽  
Author(s):  
Niclas E. Bengtsson ◽  
Jane T. Seto ◽  
John K. Hall ◽  
Jeffrey S. Chamberlain ◽  
Guy L. Odom
Keyword(s):  
Gene Therapy ◽  
Clinical Trials ◽  
Muscular Dystrophies

scholarly journals Novel Marine Compounds: Anticancer or Genotoxic?

2004 ◽  
Vol 2004 (2) ◽  
pp. 93-98 ◽  
Author(s):  
Jamal M. Arif ◽  
Amal A. Al-Hazzani ◽  
Muhammed Kunhi ◽  
Fahad Al-Khodairy
Keyword(s):  
Clinical Trials ◽  
Anticancer Agents ◽  
Screening Tests ◽  
Tumor Xenograft ◽  
Anticancer Compounds ◽  
Early Exit ◽  
Marine Compounds ◽  
Xenograft Models ◽  
Pharmaceutical Industries ◽  
Carcinogenesis Models

In the past several decades, marine organisms have generously gifted to the pharmaceutical industries numerous naturally bioactive compounds with antiviral, antibacterial, antimalarial, anti-inflammatory, antioxidant, and anticancer potentials. But till date only few anticancer drugs (cytarabine, vidarabine) have been commercially developed from marine compounds while several others are currently in different clinical trials. Majority of these compounds were tested in the tumor xenograft models, however, lack of anticancer potential data in the chemical- and/or oncogene-induced pre-initiation animal carcinogenesis models might have cost some of the marine anticancer compounds an early exit from the clinical trials. This review critically discusses importance of preclinical evaluation, failure of human clinical trials with certain potential anticancer agents, the screening tests used, and choice of biomarkers.

scholarly journals EMEA and Gene Therapy Medicinal Products Development in the European Union

2003 ◽  
Vol 2003 (1) ◽  
pp. 3-8 ◽  
Author(s):  
Marisa Papaluca Amati ◽  
Francesco Pignatti ◽  
Alexis Nolte ◽  
Nirosha Amerasinghe ◽  
Daniel Gustafsson ◽  
...  
Keyword(s):  
Gene Therapy ◽  
European Union ◽  
Clinical Trials ◽  
Good Clinical Practice ◽  
Evaluation Agency ◽  
The European Union ◽  
Medicinal Products ◽  
Regulatory Processes ◽  
National Competent Authorities ◽  
Practice Implementation

The evaluation of quality, safety, and efficacy of medicinal products by the European Medicines Evaluation Agency (EMEA) via the centralized procedure is the only available regulatory procedure for obtaining marketing authorization for gene therapy (GT) medicinal products in the European Union. The responsibility for the authorization of clinical trials remains with the national competent authorities (NCA) acting in a harmonized framework from the scientific viewpoint. With the entry into force of a new directive on good clinical practice implementation in clinical trials as of 1 May 2004, procedural aspects will also be harmonized at EU level. Scientifically sound development of medicinal products is the key for the successful registration of dossiers and for contributing to the promotion and protection of public health. The objective of this paper is to introduce the EMEA regulatory processes and scientific activities relevant to GT medicinal products.

scholarly journals Transforming clinical trials in rheumatology: towards patient-centric precision medicine

2020 ◽  
Vol 16 (10) ◽  
pp. 590-599 ◽  
Author(s):  
Costantino Pitzalis ◽  
Ernest H. S. Choy ◽  
Maya H. Buch
Keyword(s):  
Clinical Trials ◽  
Precision Medicine ◽  
Patient Centric

scholarly journals Use of Research Biopsies in Clinical Trials: Are Risks and Benefits Adequately Discussed?

2013 ◽  
Vol 31 (1) ◽  
pp. 17-22 ◽  
Author(s):  
Michael J. Overman ◽  
Janhavi Modak ◽  
Scott Kopetz ◽  
Ravi Murthy ◽  
James C. Yao ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Tumor Tissue ◽  
Eligibility Criterion ◽  
Solid Organ ◽  
Complication Rates ◽  
Limited Information ◽  
Risks And Benefits ◽  
Study Protocols ◽  
Primary Indication ◽  
Biomarker Analysis

Purpose Although the incorporation of research biopsies into clinical trials is increasing, limited information is available about how study protocols and informed consents integrate and describe their use. Methods All therapeutic clinical trials in which image-guided research biopsies were performed from January 1, 2005, to October 1, 2010, were identified from an interventional radiology database. Data from study protocols and informed consents were extracted and analyzed. Procedural complications were recorded. Results A total of 57 clinical trials were identified, of which 38 (67%) contained at least one mandatory biopsy. The analysis of the research biopsy tumor tissue was a study end point in 95% of trials. The primary indication for a research biopsy was for integral biomarker analysis in 32% and for correlative science in 68% of trials. A statistical analytic plan for the correlative science research biopsy tumor tissue was mentioned in 26%, described as exploratory in 51%, and not mentioned in 23% of trials. For studies with mandatory biopsies, biopsy was an eligibility criterion in 71% of trials, and a statistical justification for the research biopsy sample size was present in 50% of trials. A total of 745 research biopsies were performed on 576 patients. Overall and major complication rates were 5.2% (39 of 745 biopsies) and 0.8% (six of 745 biopsies), respectively. Complication rates for intrathoracic and abdominal/pelvic solid organ biopsies were 17.1% (36 of 211 biopsies) and 1.6% (three of 189 biopsies), respectively. Site-stratified research biopsy–related risks were discussed in five consents. Conclusion A better representation of the risks and benefits of research biopsies in study protocols and informed consents is needed.

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