Gene Therapy

Harnessing the power of the immune system: from bispecific antibodies to CAR-Ts and beyond

by Diane Seimetz

The first class of therapeutic products harnessing the power of T cells for target-specific treatment of oncological diseases was bispecific antibodies. Eight years later, the first autologous chimeric antigen receptor (CAR)-T cells received regulatory approval. In this article, Diane Seimetz reviews the current landscape of CAR-Ts, recent approvals  and further important developments in the cell- and gene therapy space.

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.

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.