Classification of Borderline Products in the EU: Are all nucleic acid-based products gene therapies?Regulatory, Nucleic Acid-Based TherapiesBy Anika Schroeter
Nucleic Acid-based products are gene therapies, aren’t they? The answer to this question is not as straight forward as one might think, but will substantially influence the overall development strategy of a product candidate. Indeed,
in the past, European regulators classified nucleic acid-based products either as gene therapy or as small molecule / biologic or as vaccine based on the current definition laid out in EU Directive 2001/83/EC. However, a discussion on the
European level is ongoing indicating a change and streamlining in the current thinking.
A Frequently Asked Question in Non-Clinical Development: Is there a Need for (Bio)Analytical Method Validation under GLP Conditions?Regulatory, Method ValidationBy Roman Goetz
Bioanalytical methods (as well as dose formulation assays) used in GLP-compliant nonclinical studies (as well as dose formulation assays) should be validated. Regulatory requirements on GLP-compliance of method validation are different
in the ICH geographies. Here, we are discussing the requirements and their implications on method validation.
Establishing a Safe and Efficacious Human Starting Dose: A Hurdle for Cell and Gene TherapiesHuman Starting Dose, Cell and Gene Therapy ProductsBy Anika Schroeter and Marko Repic
Definition of a human starting dose is a challenging step in every development process, representing a particular hurdle for gene and cell therapy products. In contrast to small molecules or biologics, there is no specific guidance,
providing clear recommendations on how to determine a human starting dose. Individual, product-tailored and innovative approaches are needed, which require a thorough understanding of the indication, mode of action and potential risks related
to the product. Read further to learn more about specific approaches and our experience with different cell and gene therapy products.