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EMA Publish Guideline on Quality, Non-Clinical and Clinical Aspects of Medicinal Products Containing Genetically Modified Cells

This guideline defines scientific principles and provides guidance for the development and evaluation of medicinal products containing genetically modified cells intended for use in humans and presented for marketing authorisation. Its focus is on the quality, safety and efficacy requirements of genetically modified cells developed as medicinal products.The quality section addresses the requirements specific for the genetic modification of the target cell population and for the transduced cell product resulting from the manufacturing process.

The non-clinical section addresses the non-clinical studies required with the aim at maximising the information obtained on dose selection for the clinical trials, to support the route of administration and the application schedule. Non-clinical studies should also allow determining whether the observed effect is attributable to the transduced gene, to the transduced cells or to both.

The clinical section addresses the requirements for studying as far as possible pharmacological properties of the cells itself and the transgene. The requirements for efficacy studies emphasises that the same principles apply as for the clinical development of any other medicinal product, especially those of current guidelines relating to specific therapeutic areas. The clinical section further addresses the safety evaluation of the product as well as the principles for follow up and the pharmacovigilance requirements.

For further details, please view the document below.

EMA, the European Drug Regulators, Publish Draft Guidance on Non-Clinical and Clinical Aspects of Medical Products Containing Geneticaly Modified Cells

EMA, the European Drug Regulators, Publish Draft Guidance on Non-Clinical and Clinical Aspects of Medical Products Containing Genetically Modified Cells.

Full Text Here

This guideline defines scientific principles and provides guidance for the development and evaluation of medicinal products containing genetically modified cells intended for use in humans. Its focus is on the quality, safety and efficacy requirements of genetically modified cells developed as medicinal products.

Genetically modified cells may be developed either for therapeutic use (gene therapy medicinal products) or to use the genetic modification in the manufacturing process of a cell therapy / tissue engineering product.
The following are some examples of medicinal products containing genetically modified cells (GMC) that have been used in clinical trials:

genetically modified cells for treatment of monogeneic inherited disease;

genetically modified dendritic cells and cytotoxic lymphocytes for cancer immunotherapy;

genetically modified autologous chondrocytes for cartilage repair; genetically modified progenitor  cells for cardio-vascular disease treatment or for in vivo marking studies, particularly for in vivo biodistribution or in vivo differentiation analysis;

genetically modified osteogenic cells for bone fractures repair; genetically modified cells for 66 infectious disease treatment.
This guideline defines scientific principles and provides guidance to applicants developing medicinal products containing genetically modified cells. It is recognised that this is an area under constant development and guidance should be applied to any novel procedures as appropriate.

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Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

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“Please note that the pages on this website are designed to provide you with general information only. We make no warranties, representations or undertakings about any of its content. This includes the completeness, accuracy and fitness for any particular purpose, or the content of any third party site referred to or accessed through it. You are personally responsible for ensuring that the information is correct and we will not be held liable or accountable for any mistakes that occur.”

EMA, the European Drug Regulator publishes, Draft Guideline on Quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells.

EMA, the European Drug Regulator publishes, Draft Guideline on Quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells.

Full text Here

This guideline defines scientific principles and provides guidance for the development and evaluation of medicinal products containing genetically modified cells intended for use in humans. Its focus is on the quality, safety and efficacy requirements of genetically modified cells developed as medicinal products.

Genetically modified cells may be developed either for therapeutic use (gene therapy medicinal products) or to use the genetic modification in the manufacturing process of a cell therapy / tissue engineering product.
The following are some examples of medicinal products containing genetically modified cells (GMC) that have been used in clinical trials:
− genetically modified cells for treatment of monogeneic inherited disease;
− genetically modified dendritic cells and cytotoxic lymphocytes for cancer immunotherapy;
− genetically modified autologous chondrocytes for cartilage repair; genetically modified progenitor cells for cardio-vascular disease treatment or for in vivo marking  studies, particularly for in vivo biodistribution or in vivo differentiation analysis;
− genetically modified osteogenic cells for bone fractures repair; genetically modified cells for  infectious disease treatment.
This guideline defines scientific principles and provides guidance to applicants developing medicinal products containing genetically modified cells. It is recognised that this is an area under constant development and guidance should be applied to any novel procedures as appropriate.

For Biotechnology Development Services Click Here

Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

Avoid Expensive Mistakes, Keep On Top of New and Changing Regulations for Free!

Sign up for the most value add free newsource you can get for free. We spend a huge amount of time and effort monitoring the main drug / device regulators websites for changes in the regulatory environment, and capture between 20 and 40 new regulations, rules and initiatives each month, and summaries them in a fantastic FREE monthly Regulatory and Market Round Up. You can Un-Subscribe at any time and we don not share your details with anybody. You can’t afford to miss out on this service. Just fill in the form below.

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“Please note that the pages on this website are designed to provide you with general information only. We make no warranties, representations or undertakings about any of its content. This includes the completeness, accuracy and fitness for any particular purpose, or the content of any third party site referred to or accessed through it. You are personally responsible for ensuring that the information is correct and we will not be held liable or accountable for any mistakes that occur.”

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