The missing piece of the puzzle

After decades of efforts, recent years have seen significant progress in research and development (R&D) in the field of cell and gene therapy. Following approvals of several therapies in haemato-oncology since 2017, developers across the globe have now turned their attention to opening entirely new treatment pathways for a variety of diseases beyond haematological cancers.

If proved effective, patients across Europe living with a range of auto-immune diseases, as well central nervous system diseases such as multiple sclerosis, could be set to benefit. To realise this opportunity though, we must ensure the European Union is putting innovation, and the patients it serves, at the forefront.

Against this backdrop, the announcement of a revision to the European Union's general pharmaceutical legislation was welcome news to European-based advanced therapy medicinal product (ATMP) developers and marked a potential once-in-a-lifetime opportunity for Europe to close the gap with global pioneers in the field of cell and gene therapy.

In the past, the European legislative environment has been complex and burdensome, putting ATMP developers in Europe at a disadvantage compared with those in other regions. Today, most innovations in cell and gene therapy are being pioneered in the USA or China. The regulatory frameworks and authorities in both countries have allowed for faster development and commercialisation of promising new therapies for patients.

The good news? Europe already has leaders in the ATMP field. A revision of European pharmaceutical legislation that includes long-term oriented research funding dedicated to cell and gene therapy products and a flexible and risk-adapted legislative framework will support these European developers to increase access to, and improve affordability of, new, life-saving treatments for European patients. The Miltenyi Group is one such company that has been at the heart of global R&D in cell and gene therapy for many years.

Miltenyi Biomedicine is a biopharmaceutical company whose mission is to bring treatments to patients living with cancer and autoimmune diseases. The company is building on the experience of Miltenyi Biotec, founded in Germany more than 30 years ago. Despite this heritage in cell and gene therapy, the company, along with other European-based biotech companies, needs more support to remain competitive.

"We are a highly specialised company with truly world-leading expertise in cell and gene therapy, and the potential to bring personalised new therapies to patients in need. While operating globally, our roots are in Germany and we have significant R&D operations in the EU. In order to bring faster new treatments to patients in need, we need a more supportive regulatory environment," said Toon Overstijns, CEO Miltenyi Biomedicine.

"In a complex and rapidly evolving scientific field, innovative companies like us need a European regulatory environment that allows for improved interactions with authorities by accelerating and intensifying the mutual feedback process in order to advance treatments that will help patients who fight serious diseases every day," he continued.

A particularly welcome aspect of the new package for the development of cell and gene therapies has been the clarification of the decentralised manufacturing-related requirements. This has the potential to democratise access to high-quality, effective, and vitally, safe ATMPs for patients in European countries that are typically forgotten. Likewise, the introduction of regulatory sandboxes could support the testing and implementation of new innovative technologies, such as point-of-care manufacturing, to further increase access to personalised ATMPs across the continent.

"Personally, I was always driven by the vision to reinvent medicine with new ways to tackle diseases through cell and gene therapies. We are fully committed at Miltenyi Biomedicine to making this change happen, and fast, but we need the legal and political environment to do this from a European base while ensuring we are competitive with companies from the USA or China," said Stefan Miltenyi, founder and owner of Miltenyi Biotec and Miltenyi Biomedicine.