How does Genzyme's R&D strategy differ from that of its competitors?

Genzyme is a company that regards unmet medical needs as a top priority. So, we focus on areas where we think existing therapies do not provide satisfactory treatment. That's why Genzyme started focusing on rare diseases, such as genetic disorders like Gaucher's disease, about 15–20 years ago. At the time, this type of indication did not catch the attention of industry, but since the introduction of orphan drug legislation, R&D for niche diseases has become more attractive and many biotechnology companies have moved into this area. Of course we still also focus on major diseases, such as cancer and multiple sclerosis (MS), where there is a higher level of competition. In these markets, our aim is to develop products that are significantly better than existing therapies. The strategy here is to gain a competitive advantage by developing a product that is self-differentiating and presents a better risk–benefit profile. We feel we have done this with the development of our monoclonal antibody, alemtuzumab (Campath), which is about to enter Phase III trials for MS and works by targeting the immunological basis of the disease rather than its symptoms.boxed-text

How do you evaluate the risk of going into pioneering areas of R&D?

Risk taking is part of our culture as scientists. We are used to entering unchartered territories and to challenging regulators. Calculation of risk comes from our extensive experience of developing leading products in emerging areas. In particular, we always keep patients in mind when developing products. We tend to mitigate risk by working hand-in-hand with the regulator at an early stage, especially with pioneering products. This approach enabled us, a few years ago, to bring an autologous cartilage replacement therapy called Carticel to market by working with regulatory authorities to define a new regulatory framework for such products. The same is happening with the ongoing discussion on Advanced Therapy Regulation at the European Commission (EC), where Genzyme is actively collaborating with regulatory authorities and the EC to help design legislation that will enable innovative products in the area of somatic cell and gene therapy to be brought to market.

Does Genzyme prefer in-licensing over acquisition to broaden its product portfolio?

In the early days after Genzyme was founded, we enlarged our portfolio mainly through partnering rather than in-licensing or acquisition and got involved in many joint ventures. That's because Genzyme is not a big marketing machine, nor do we own a large capillary sales force. Our strength is in development with other companies.

Nowadays, however, in some cases, when we spot synergies in our product development philosophies with other companies, we might go ahead and make an acquisition. We would possibly leave a company's structure untouched as much as we can. That's what happened when we acquired GelTex Pharmaceuticals of Waltham, Massachusetts in 2000; the company has since become Genzyme's drug discovery facility. More recently, both the cancer diagnostics firm Impath and the drug development company ILEX Oncology were acquired in 2004 as a result of a strategic plan by Genzyme to move into the area of oncology. Genzyme's latest acquisition of AnorMED, in November 2006, also provides the company with additional innovative products for its oncology and transplants businesses.

How do you balance cutting edge research with maximizing return to investors?

The key to ensuring Genzyme is an attractive company for investors is sustainability. Our R&D strategy reflects this approach. We are careful to balance branching out into certain pioneering areas with consolidation of existing areas to provide steady revenue. Many people, including top-level scientific advisors, thought we were foolish when we started developing an enzyme replacement therapy (ERT) for Gaucher's disease. Today, more than 4,800 Gaucher patients in 90 countries are receiving our therapy. We have also developed three more ERTs for Fabry, mucopolysaccharidosis I and Pompe disease. We now have a system in place that enables us to look at technology platforms that offer significant improvement over existing treatments to try and find a business model to develop products on the basis of added value of this innovation. Our decision is science-based and relies on medical evidence.

A good example of how investment in a previously unexploited area can work is our experience with developing an adenoviral gene therapy vector for cystic fibrosis. More than 10 years ago, we started clinical trials to attempt the correction in the lungs of the genetic defect that causes cystic fibrosis, but because of the multi-factorial nature of the disease we were not as successful from a global clinical standpoint. However, we later used the enormous experience gained in the development of these adenoviral vectors and applied it to our R&D for other diseases such as heart disorders. As a result, we are in the process of developing a therapy that enables peripheral revascularization of the limbs to treat critical limb ailment and intermittent claudication. In this case, it was important to retain the technology platform we invested in to be able to apply it to a different disease. The secret is to be genuinely interested in the science and in treating patients while always keeping in mind the various potential applications of the products in your pipeline.

Interview by Sabine Louët