Life sciences company Bayer announced the launch of a new cell and gene therapy platform within its Pharmaceuticals Division in early December 2020. With this strategic platform, the company aims to build a leading position in this field while continuing to drive the transformation of its pharmaceuticals business. Wolfram Carius, Head of Cell and Gene Therapy in Bayer's Pharmaceuticals Division since October 2020, explains to CHEManager how the company is driving its strategy in this field as well as its promising development portfolio. The questions were asked by Ralf Kempf.

CHEManager: Mr. Carius, why are cell and gene therapies of such strategic importance as growth drivers for Bayer's pharmaceutical business?

Wolfram Carius: Our vision at Bayer is to transform patient health through breakthrough innovation, and cell and gene therapies offer an unprecedented opportunity to do so. For the first time, we can enable a paradigm shift from treating symptoms to potentially curative approaches, and we are committed to championing this area of innovation.

Cell and gene therapies already today account for hundreds of clinical trials, and it is a rapidly growing sector. Scientific knowledge in this field is growing exponentially, and this is a defining moment for the industry. Years ago, when the monoclonal antibodies that now play a very crucial role in many therapeutic areas entered clinical development, we saw US companies really seizing this opportunity, while European players missed it – at least to a large extent. This can’t be the case now, and we at Bayer are determined to go "all in" on cell and gene therapies and be a true leader in this field.

Is Bayer focusing on selected areas of cell and gene therapies?

W. Carius:  More than a year ago, we took a very strategic look at the different technologies available on the market. We carefully selected what to focus on in a way that all elements of our strategy complement each other, build on each other and have a big development potential for the future.

Strategically, Bayer is focusing on four selected areas of cell and gene therapy: stem cell therapies with a focus on induced pluripotent cells or iPSCs, gene augmentation, gene editing and allogeneic cell therapies. These areas have been selected so that each part of the strategy contributes to building a holistic Cell and Gene Therapy Platform. This goes beyond individual acquisitions, and the focus is equally on making new treatment options available to patients safely and as quickly as possible, as well as creating sustainable value for Bayer.

What are your goals with the newly launched cell and gene therapy platform at the end of last year?

W. Carius:  Our primary goal is to transform innovative ideas into real products and tangible therapies.

Bayer's cell and gene therapy platform encompasses the entire value chain, from research and development to market and patient. And we combine the best of the two worlds of biotech and pharma: BlueRock Therapeutics or AskBio, for example, can remain focused on generating and driving breakthrough innovation on both the product and technology sides, while we bring the experience, expertise and, for example, global reach of a pharmaceutical company. A common alignment in strategy and goals is of course essential.

BlueRock Therapeutics and AskBio, the companies you mentioned that were acquired in 2019 and 2020, are fully owned by Bayer and integrated into the new cell and gene therapy platform, but are managed independently. What do you expect to gain from this?

W. Carius: We set ourselves the goal of fostering innovation, accelerating its implementation and driving value creation through largely independent and fully responsible partners: That's why BlueRock and AskBio, for example, operate independently, with us supporting on the pharma side when required. In this way, we bring together the entrepreneurial, passionate "can do" spirit of biotech and the global experience of pharma in the best possible way.

What role do acquisitions and collaborations with other companies generally play in Bayer's strategy?

W. Carius: External innovation is an important value driver, especially in the highly dynamic and competitive field of cell and gene therapies. Small, agile companies are strongly driving innovation based on their world-class technology leadership, and we encourage these teams to continue doing what they do best. This is where the right partnering strategy becomes especially important: We want to leverage external innovation along with the expertise of our teams at Bayer to bring products to the market as quickly as possible.

How many compounds for cell and gene therapies are in development at Bayer? Which therapeutic areas do they cover?

W. Carius: Our development portfolio in this area already includes eight advanced assets in various stages of clinical development. These address multiple therapeutic areas with high unmet medical need, such as neurodegenerative, neuromuscular and cardiovascular indications – with leading programs in Pompe disease, Parkinson's disease, hemophilia A and congestive heart failure. With over fifteen preclinical projects, the pipeline will continue to expand in the coming years.

Treatments based on cell and gene therapy approaches are very expensive. Do you see any possibility of reducing the cost of these therapies in the future or finding new payment models?

W. Carius: Although cell and gene therapies are making a strong appearance in the portfolios of companies and regulators, it is too early to comment on the potential pricing of the new treatments, and it is expected that each candidate will be considered individually. Our market access approach will consider the potential value of cell and gene therapies, with the goal of providing patients with access to the medicines they need. We are committed to our ambition of "Health for All" and our technology platforms undoubtedly have huge potential here.

Innovations in digital health solutions and integrated care offerings are also a new pillar of Bayer's pharma business. Are some of these offerings also part of the strategy to expand cell and gene therapy?

W. Carius:  At Bayer, we want to play a central role in digital health, developing solutions that better serve patients and physicians and make healthcare systems more efficient. The current pandemic has driven digital health forward, and this interest will continue after the Covid-19 pandemic, likely boosting the field even more.

But digitalization is already transforming our business today. For example, artificial intelligence is changing aspects from how we identify new biological targets and develop medicines to how we engage with our customers. We are also using artificial intelligence to increase the speed and efficiency of clinical trials.

The potential is huge, and so is the impact that digital health solutions and integrated care offerings can have on patients. Bayer and One Drop are already building on their existing diabetes management platform to offer options that support patients in other areas.

It's about accompanying patients during their journey and shifting the vision from a pathology-centric to a person-centric approach. For cell and gene therapy, digitization is essential.