Cell and gene therapies are changing the face of modern medicine – But how to find the right dose?

by Diane Seimetz

Finding the right dose for the first in human study can be a challenging undertaking for biopharmaceuticals. For cell and gene therapy products (commonly referred to as ATMPs), this is even more challenging. In this article, Diane Seimetz reminds us what Paracelsus’ toxicology principle has taught us, and she explains what the key questions for ATMPs are to guide a tailored dose-finding exercise.

Cutting costs for cutting edge therapeutic products: ATMP process optimization

by Elena Meurer

The affordability of ATMPs for the health care system and patients lies among the important aspects that clearly require further improvement. The overall high price of cell-based therapeutics, as known today, is driven by specific factors involved in development, manufacturing and clinical logistics. In this article, Elena Meurer explains where to cut costs in the manufacturing process of ATMPs.

What ATMP developers should know when looking for a CMO

by Elena Meurer

As ATMP start-ups mature, they will face the following challenging decision: whether to develop, test, and manufacture its products in-house or to outsource the activities to a contract manufacturer (CMO). ATMP developers who do not have their own GMP facility or plans to set-up their own plant, will need to look for a CMO. In this article, Elena Meurer provides a comprehensive and structured guide how to select a CMO for the manufacture of ATMPs.

The European GMO legislation for gene therapy products in clinical trials and its pitfalls

Advanced Therapy Medicinal Products (ATMPs) such as Gene and Cell Therapy medicinal products are innovative biopharmaceuticals with the potential to bring high therapeutic value. Especially the new gene editing tools such as CRISPR-Cas9 are expected to shift the focus of medicine from treating symptoms to curing diseases. However, especially their clinical development faces specific challenges. For products containing genetically modified organisms (GMOs), an additional risk assessment step in the clinical trial authorisation (CTA) procedure is required. Although a European regulatory framework for this matter exists, it has been implemented differently into the Member State (MS) national legislation, leading to different documentation requirements and review and approval frameworks between the Member States.

In this article, Theodor Tiko and Annegret Vaggelas review the current status of the European GMO legislation for gene therapy products in clinical trials, including its pitfalls for clinical trial applicants.

Hospital Exemption for ATMPs: Perspectives & Regulatory Trends Under Discussion

Advanced Therapy Medicinal Products (ATMPs) bear game changing potential for the treatment of severe conditions for which we have no appropriate therapies today. The product class of ATMPs comprises gene therapy medicinal products (GTMPs), somatic cell therapy medicinal products (sCTMPs), tissue-engineered products (TEPs) as well as combined ATMPs. ATMPs are complex products, and ATMP developing companies face diverse challenges during development as well as for commercialization.