BioSpring Event: Genome Editing – Medicine of the Future

Frankfurt am Main, Germany, February 9, 2024. BioSpring GmbH, the world's leading manufacturer of genome editing active ingredients, hosted a specialist event on the 9^th of February that focused on the medical revolution sparked by the advancement of genome editing technology.

The outstanding importance of developing genomic editing therapeutics was emphasized by the Federal Minister of Education and Research, Bettina Stark-Watzinger, in her opening keynote speech at the Union Hall in Frankfurt. Distinguished researchers and industry experts delivered exclusive presentations to the attendees, a conglomeration of professionals from life science, business, and politics, offering profound insights into the groundbreaking advancements genome editing will bring in the near future.

Genome editing refers to the targeted modification of genes using molecular biological methods. This means that previously incurable diseases can be cured. Over 400 million people are affected by one of the approximately 7,000 diseases that have genetic origins.

An active pharmaceutical ingredient, based on a form of genome editing technology known as CRISPR/Cas9, commonly termed "gene scissors", received approval for the first time worldwide in the UK, the USA and the EU in November and December 2023. This new therapy has the potential to cure sickle cell anemia and ß-thalassemia, two serious hereditary blood diseases. With its unique ability to cure a disease with just one intervention, genome editing has the potential to fundamentally transform the approach to treatment of common diseases.

Of particular importance for genome editing technology is guide RNA, composed of synthetic nucleotide chains that act as a molecular "navigation system", guiding the CRISPR system to the DNA target where changes can be made to the genetic code. Utilizing high-tech production processes and analytical methods, BioSpring has developed into one of the world's leading companies for such therapeutic oligonucleotide active ingredients.

Achieving the highest quality is crucial in the production of these pioneering active pharmaceutical ingredients for both efficacy and safety. BioSpring’s commitment to highest quality has been thoroughly validated through numerous successful audits. BioSpring has been a commercial manufacturer of oligonucleotide active pharmaceutical ingredients approved by the US Food & Drug Administration (FDA) since 2020.

BioSpring's early investment in the development of manufacturing and analytical methods for genome editing compounds utilizing cutting-edge technologies has positioned the company as the global market leader in this category of compounds. With the commissioning of a new production site in Frankfurt am Main, Germany, specifically for the manufacture of these active pharmaceutical ingredients, BioSpring is rapidly expanding capacity to meet the high demand.

Genome Editing:  Special event hosted by BioSpring in Frankfurt am Main, Germany

In her welcoming speech, Managing Director Dr. Sylvia Wojczewski summed up the enormous potential of genome editing technology: “Highly effective treatment options in record time for both large and very small patient groups: that is the promise of genome editing. Our therapeutic ingredients have the potential to cure even serious diseases that were previously considered incurable.“

Genome editing technology takes a revolutionary approach to treat diseases at their root cause. As Dr. Jörg Vogel, Director of the Helmholtz Institute for RNA-based Infection Research, summarized in his presentation, "Therapies based on genome editing start where the diseases arise, namely at disease-causing sites in our genome."

"Certain diseases cannot be treated at all with conventional drugs," explained Dr. Toni Cathomen, Director of the Institute of Transfusion Medicine and Gene Therapy at the Freiburg University Medical Center, Germany, "and this is where genome editing offers hope for these patients for the first time."

Daniela Karlstetter, Procurement Category Lead Cell and Gene Therapy at Bayer AG, presented her perspective on this new field of therapy.

In the concluding panel discussion, Dr. Jochen Maas, Vice President of the House of Pharma and Healthcare and long-standing Head of Research and Development at Sanofi Germany, emphasized the particular importance of Germany as a production location: “Strong research must be flanked by strong production in order to keep know-how and well-paid jobs in Germany in the long term.” Dr. Hüseyin Aygün, Scientific Managing Director of BioSpring, added: "BioSpring is a good example of how the production of complex biopharmaceuticals can succeed in Germany. It is now important that politicians set the right course so that the enormous potential for value creation can also be realized in Germany in the future."