Science at Genzyme

It's only when you combine all the areas of expertise from different individuals that you can really make an impact and drive a project sufficiently forward.
— Andrew Leger
Senior Scientist

Inspired by the potential to improve patients' lives, Genzyme's scientists work at the forefront of health care research and technology. We collaborate with a global network of experts to discover and develop therapies that have the potential to change patients' lives.
The Path to Treatment

At Genzyme, science is at the core of what we do. It informs every step on the path to an approved treatment, from the initial discovery of a promising therapeutic candidate through the rigorous preclinical and clinical research and development necessary to deliver a marketable product.

We take a cross-functional approach, involving staff members from medical, regulatory, and sales and marketing early in the research process to help maximize our chances of both clinical and commercial success. With expertise across every step of the process, we have the capabilities to advance many kinds of research programs:

  • Internal discoveries – Our researchers in molecular biology and chemistry discover promising candidates that show potential to go all the way from the lab to the patient—such as the small molecule we've recently identified to target polycystic kidney disease (PKD), a rare genetic disorder that can affect the lungs, liver, pancreas, heart, and brain.
  • Developing external discoveries – Some of our greatest successes have been in realizing the full potential of outside discoveries. Several of our products began as joint ventures or partnerships that we developed further to bring to market.
  • Maximizing existing products – We're constantly exploring new opportunities to maximize the potential of our current products, whether enhanced formulations to better address patient needs or label expansions to treat entirely new indications. For example, one of our former oncology products, now a part of the Sanofi portfolio, is in late-stage development as a potentially promising new treatment for multiple sclerosis.

Success Story: Our First Therapy

Developing a new therapy is a challenging process – it takes many years, costs millions of dollars, and has a low rate of success. Persistence is a hallmark of good science, and a fundamental element of Genzyme's research philosophy. It took us 10 years to bring our first therapeutic product to market, a first-of-its-kind enzyme replacement to treat the rare genetic disorder Gaucher disease. Yet we persevered out of a commitment to a small group of patients that had no other alternative. Our success changed the lives of these patients – and established a business model for treatment of orphan diseases.

A Therapeutic Breakthrough
During his acceptance speech for Babson College's 2010 Distinguished Entrepreneur award, Genzyme's former CEO Henri Termeer tells the story of the challenges we faced on the road to launching our first therapy.

A Broad-Based Approach

Genzyme's research is oriented around medical areas where we feel new therapies can have a significant impact, emphasizing our primary areas of focus – rare diseases and neuroimmunological disorders such as multiple sclerosis. Our science organization includes researchers that are among the world's leading experts in these disease areas, bringing knowledge, resources, and credibility within the scientific community to our efforts.

To explore solutions to our targeted medical areas, we have developed a diverse array of cutting-edge technology platforms that include both biologic and chemical approaches. Our product development efforts emerge from a "matrix" of our research areas crossed with these technologies.

How We Work

Genzyme's scientists and researchers are fully integrated into our entire operations. They communicate regularly with our clinical, commercial, and regulatory experts and with outside clinicians to fully understand the needs that drive our business and to discuss new opportunities. These synergies have allowed us to innovate not only in our scientific discoveries, but also in the processes that lead to a marketable product. For example, we brought the first cell therapy to market largely by pioneering the regulatory path for its approval – and opened the door for a new class of treatments.

Discovering Hope
At Genzyme, science is at the core of what we do. Find out how we successfully developed cutting-edge therapies for rare diseases and multiple sclerosis, and how our scientists continue to innovate on behalf of patients today.

Our scientists are also part of a broader Sanofi research organization known as the Boston R&D Hub. In this highly collaborative environment, scientists with a variety of different areas of expertise share ideas freely and openly.  

Looking Outward: Global Collaborations

Every year, Genzyme invests hundreds of millions of dollars into research and development, yet that is only a fraction of what is spent globally in the field. We recognize that to have a meaningful impact, we need to reach beyond the bounds of our company and access an even more extensive base of scientific knowledge and resources.

Our research facilities are located in Massachusetts, home to some of the world’s most renowned scientific communities, and our scientists collaborate frequently with researchers from MIT, Harvard, Massachusetts General Hospital, and other organizations in the area. But we have also built strong relationships with universities, hospitals, companies, disease-associated foundations, and patient organizations around the world. Our scientists stay connected to the larger scientific community through conferences, symposiums, and personal networking. These activities and relationships help us stay abreast of the latest technologies and better understand regional medical needs, practices, and regulatory requirements.

Staying involved also helps us identify potential collaborators – a critical part of our business. We actively pursue partnerships, joint ventures, licensing opportunities, co-sponsored trials, and other activities to help advance our research efforts. Over the years, we've collaborated with investigators at universities, research institutions, private companies, and government organizations to put science to work for patients in need around the world.


Learn more about Genzyme's support for investigator-sponsored studies

Partnership with Alnylam

We recently entered into a partnership with Alnylam Pharmaceuticals to co-develop and co-commercialize several potential treatments for rare genetic diseases. These products are a new class of therapeutics based on RNA interference. RNAi, also known as “gene silencing,” works by inhibiting the production of proteins that cause or contribute to the targeted disease.

Learn more.

Last Updated: 6/30/2015
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Genzyme's R&D Brochure

Learn more about the diseases Genzyme is researching, our recent breakthroughs and promising programs, and our long history of successful innovation on behalf of patients.

Collaborative Alliances

Learn about Genzyme's strategies for external partnerships, based on collaboration, shared goals, and complementary strengths.

IRDiRC Member
Rare Disease Research Consortium

Genzyme is a proud member of the International Rare Disease Research Consortium (IRDiRC), which teams up researchers and organizations investing in rare disease research in order to achieve two main objectives by the year 2020, namely to deliver 200 new therapies for rare diseases and means to diagnose most rare diseases.