An Interview with Denis Bedoret of MaSTherCell
We recently sat down with Denis Bedoret, Chief BD Officier at MaSTherCell in Amsterdam at the Cell Therapy Manufacturing & Gene Therapy Congress conference to discuss the critical issues and opportunities present in the cell & gene therapy industry today.
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By Manuel J. T. Carrondo, Prof. Chem & Biochem Eng., FCT/UNL & Vice-president, iBET
Since the early nineties iBET has been involved in production and purification of viruses for gene therapy. Early on, enveloped retroviruses and non-enveloped adenoviruses where the targets; from late nineties onward lentivirus and baculovirus were added to the portfolio of enveloped viruses and AAV to the non enveloped viruses.
Although originally meant for monogenetic diseases, now some are also produced for cancer treatment (ex. oncolytic adenoviruses made in A547 cells, so replicative) or as reagents for cell therapies (also known as ex-vivo gene therapies).
Having developed scaled down tests and analyticals (including surface plasmom resonance, dynamic light scattering) coupled with its chemical engineering model competencies, iBET has designed membrane or media materials for which our key partners MERCK Millipore, SARTORIUS, GE HealthCare have developed the prototypes tested on our biologies and equipments. In this way, improved DSP processes have been created increasing viral yields and infective to total particle ratios or yields and viability for cell therapies.
Continue reading “Novel Strategies for Gene and Cell Therapies”
KNect365 is pleased to introduce a special Digital Week program, comprised of a week-long series of free webinars with live Q&A – connecting cell and gene therapy leaders throughout the year from the comfort of your desk.
Cell Therapy Manufacturing & Gene Therapy Digital Week connects cell and gene therapy leaders to drive manufacturing and commercialisation through direct access to innovative discovery, product development, and regulatory know-how.
Register now to watch free educational sessions presented by leading industry experts, get answers to your toughest questions, network with colleagues and partners, and download useful resources.
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Regulatory perspectives and considerations for cell & gene therapies
Japan has been at the forefront in the innovation and development cell and gene therapies. To accelerate the development, innovation and commercialization of regenerative medicine products, Japan has championed a radical approach by passing new legislations known as the act on the safety of regenerative medicine”(ASRM) and the pharmaceuticals, medical devices and other therapeutic products act (PMDA), which came into effect in November 2014. The PMDA and ASRM identify the need for collaboration between the industry and medical institutions and the need for guidelines securing the safety of new stem cell therapies respectively. The new PMDA includes drugs, medical devices as well as regenerative medical products defined as ‘processed live human/animal cells’ that are intended to be used for (1) the reconstruction, repair, or formation of structures or functions of the human body or (2) the treatment or prevention of human diseases, and gene therapy.
The ASRM regulates medical professionals’ practices and clinical studies related to regenerative medicine and charts the legal regulations for research and daily practice of cell therapy without approved regenerative medical products. The ASRM specifies manufacturing and quality requirements for cell and gene therapies, also called as good, gene, cellular and tissue-based product manufacturing practice (GCTP) to ensure safety of the manufacturing facilities and equipment, as well as the aspects of quality control. The PMDA enlists a new pathway to get conditional and time-limited approval for “regenerative medical products,” accounting for the high degree of variability in cell and therapies under the classification.
Continue reading “New Regulatory Framework in Japan”
With Nick Timmins, VP of Technology and Director of BridGE, CCRM
Nick Timmins, VP of Technology and Director of BridGE at CCRM sat down with us this October at Biotech Week Boston to discuss the collaboration efforts between CCRM and GE Healthcare, what issues have been encountered along the way, his experience working with start-ups, and much more. When initially outlining the GE collaboration, Nick mentions, “A key focus of this collaboration is really about identifying where gaps are in the field for cell and gene therapy manufacturing solutions both on the process side of things as well as the technologies that we utilize to enable those processes.” watch the full interview below to hear his complete thoughts on these pressing issues and the importance of this collaboration on the cell and gene therapy industry.
Recent clinical trials in the field of cell and gene therapy demonstrate remarkable therapeutic benefits with excellent safety. Despite demonstrated therapeutic effects, the commercialization of cell and gene therapies and their patient outreach remain scarce. Much of the research and development on cell and gene therapies is performed either on an academic level or by small and medium enterprises, largely excluding large pharmaceutical companies. Regulatory approval for cell and gene therapeutic products is performed on an individual product basis and is classified based on the degree of manipulation and intended end use. The primary deterrents to the lackluster commercialization of cell and gene therapies include the inherent complexities of the cells, issues with scalability for manufacturing and logistics and the complex regulatory requirements and time-consuming clinical trials. Cell and gene therapy products also have to navigate through the disparities in the regulatory requirements across regions. Furthermore, the complexity of product classification, extra requirements for combination of cell and gene therapies with a medical device, extensive paperwork surrounding the often ambiguous certification procedures and most importantly, the lack of harmonization of regulations across regions deter new investments and innovation in the field.
Limited understanding of the complex interactions of cell and gene therapeutics, absence of established standards and relatively scarce research data on the mechanism of action of these therapeutics make it difficult for stakeholders to navigate the complex and stringent regulatory requirements.
We elaborate the fundamental regulatory concerns associated with the development of cell and gene therapy products, and the need for international harmonization of regulatory requirement for approval of cell and gene therapies. The paper also addresses specific regulatory aspects in the EU and Japan as well as the roll-out of fast track mechanisms for market authorization in the EU and Japan. Finally, the paper addresses the urgent unmet need to provide regulatory certainty in the field of cell and gene therapeutics in the fast evolving global regulatory landscape.
By: Stephen Moore
IND to BLA: Regulatory Experiences with a Platform Cell and Therapy Autologous Fibroblast Program
John Maslowski, SVP Scientific Affairs at Fibrocell Scientific Inc., launched the morning session with a discussion on the FDA regulatory pathways a company can take when developing a new therapy. These include the Fast Track, Accelerated Approval, Priority Review, and Breakthrough Therapy pathways. He discussed the qualifying criteria and advantages for each pathway and emphasised that the Fast Track and Breakthrough Therapy pathways could be used with the investigational new drug (IND) program. He focussed on lessons learnt at Fibrocell when they sought IND approval for a biologic called FCX-007 and BLA approval for another therapeutic call LAVIV, including timeline considerations. One key point that was brought up was the possibility that the regulatory committee may require additional data, thus additional financial burden may be placed upon the submitter, which needed to considered. John then discussed in depth the key review focus of the FDA based on Fibrocell’s experience with an autologous cell product. Focus was placed on CMC, Clinical, Logistics, Labeling, and the Advisory Committee. For example, for Logistics the FDA was interested in logistical requirements that did not lie within manufacturing facility. These included the procedures to track the product throughout its entire life-cycle and customer service policies, including physician training and the handling of shipping errors. He finished his presentation by discussing what steps to take when changes to the manufacturing process are made after approval has been granted by the FDA.
Continue reading “The Regulatory Pathways and Breakthrough Designations for Cell & Gene Therapies”