The path to an approved treatment is never easy. It can take many years and millions of dollars. And, even with all the time and money in the world, success is never assured.
From initial discovery, through pre-clinical and clinical research, tremendous commitment is required to deliver a product to patients. Many treatment options and approaches are often explored: enzyme replacement, small molecule, antibody, RNA interference, messenger RNA, gene and cell therapy.
These techniques are complex and require specialized knowledge, but we have committed ourselves to developing expertise in all of them, enabling us to advance a diverse set of research programs and therapy options for patients.
This commitment, however, comes from something deeper than just scientific discovery. The desire to find and advance new therapies - to provide hope and improve the lives of patients - is led by our dedicated employees. These employees, located in offices and laboratories around the world, demonstrate a passion to help patients and the spirit to innovate.
Seng Cheng, Global Head of Research, Rare Diseases is one of those employees. He explains, “The culture has always been about our commitment to patients. We’re not only satisfied with a drug or the first drug. We’re always looking and finding if there are other aspects of the disease that are unmet.”
The culture has always been about our commitment to patients. We’re not only satisfied with a drug or the first drug. We’re always looking and finding if there are other aspects of the disease that are unmet.
For our researchers, cutting-edge science is the path to improving patients’ lives. Employees put our patients’ faces and names to the diseases they are working on, making research more than just an intellectual pursuit—it's a personal quest.
As part of the Sanofi North American R&D Hub, our researchers work in highly collaborative networks within the company as well as with external world-class scientific communities in the area and around the world.
Specialized in developing breakthrough therapies for patients who have few options in the areas of rare diseases, multiple sclerosis, immunology and oncology, our scientists are currently researching treatments for diseases including multiple myeloma, polycystic kidney disease, Parkinson’s disease, Huntington’s disease, and cystic fibrosis.
To guide today’s explorations in the laboratory, researchers call upon Sanofi Genzyme’s experience developing the world’s first enzyme replacement to treat Gaucher disease. Seng recalled, “We collaborated with the National Institutes of Health and the National Gaucher Foundation, leading to the development of a therapy for this patient population. That model still resonates today, and it’s how we continue to work in the rare disease space.”
Our research in Gaucher disease has led to our current clinical development programs for ASMD, or Niemann-Pick Disease Type B – an enzyme deficiency disorder similar to Gaucher disease, and Parkinson’s disease. Seng added, “The work we have done in rare diseases has provided us with the inspiration and vision for what we can do for patients living with difficult conditions like Niemann-Pick B and other diseases.”
Innovation means doing something that no one else has done before. Sometimes innovation means breaking new ground and sometimes it means unlocking new potential in a product that already exists.
“We’re not looking for things that provide an incremental improvement to patients,” said Seng. “We’re looking for drugs that truly transform the lives of patients. That’s a doctrine that we practice here in R&D. And that’s something we’re all very proud of. And we believe it.”
Learn more about Sanofi Genzyme’s research pipeline.