“Technology platform" refers to the technologic approach upon which a treatment is based. This approach may be chemical synthesis, like a small molecule or therapeutic polymer drug. Or it may be biologic, like an engineered protein or antibody. Even whole cells grown in culture can form the basis of a therapy.
Genzyme has developed one of the most diverse arrays of technology platforms in the industry, including many proprietary approaches and associated patents. This broad base of potential treatment platforms allows us to explore multiple avenues for treating any particular disease. Our experts work together to identify the optimal format to meet physician and patient needs – which may be a combination of platforms. We have brought products to market in all of these technology areas except gene therapy, a still developing field in which we have industry-leading research and development efforts.
Explore our current technology platforms to find out more about our approach, achievements, and ambitions for each.
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Proteins are organic compounds, composed of amino acids, that perform many important functions in the body. Examples include enzymes that catalyze chemical reactions and antibodies that protect us from foreign substances. Protein-based therapies are biological products that may be developed from human, animal, or genetically engineered sources. They are specifically designed to provide a targeted therapeutic benefit in a given disease.
Genzyme has long been a global leader in protein therapeutics. Our research focuses on a variety of protein-based product platforms, including enzymes, polyclonal and monoclonal antibodies, hormones, and more. Our expertise covers the entire product development process, from molecular discovery and biochemical investigation to cell culture and manufacturing. The therapies we develop address a wide range of indications.
We pioneered the first enzyme replacement therapies for Gaucher disease – one of several lysosomal storage disorders (LSDs) caused by enzyme deficiencies – first using placenta-derived enzymes and then later recombinant DNA manufacturing. We have since leveraged this work into enzyme replacements for several other LSDs and continue to expand our efforts in this therapeutic area.
The antibodies Genzyme has developed serve various therapeutic purposes, from targeting cancer cells to suppressing organ transplant rejection. One of our most promising initiatives that has completed two Phase 3 clinical trials is a monoclonal antibody showing strong data for stopping progression of multiple sclerosis and even reducing the disabilities associated with the disease. And we've also developed a hormone product used as a diagnostic tool and adjunctive therapy to other treatments for thyroid cancer.
| Types of therapies developed: |
recombinant enzyme replacement therapies; humanized monoclonal antibodies; animal polyclonal antibodies; recombinant human hormones and growth factor |
| Most relevant research areas: |
genetic disease; renal disease; oncology; immunology |
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Therapeutic polymers – large (high molecular weight) chemically synthesized compounds formed of repeating structural units – are used for specialized medical purposes, such as helping eliminate unwanted substances from the body. Synthetic polymers can also be used to improve the activity of drugs, deliver drugs to a precise target in the body, or control and sustain their release.
Therapeutic polymers represent one of Genzyme's earliest research efforts into chemistry-based (rather than biologic) pharmaceuticals. Our scientists have developed novel polymer drugs that, taken orally, bind and eliminate substances from the gastrointestinal tract without the drugs themselves being absorbed. This therapeutic technology has led to approved treatments, now in the Sanofi portfolio, for reducing phosphate levels in chronic kidney disease patients on dialysis as well as lowering blood cholesterol.
| Types of therapies developed: |
phosphate binders; sequestrants |
| Most relevant research areas: |
renal disease; cardiovascular disease |
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Most traditional pharmaceutical drugs are small (low molecular weight) molecules that are prepared by chemical synthesis. Small molecules have numerous therapeutic applications and are used across every area of medicine.
While Genzyme's roots are in biologic therapies, we have also built expertise in traditional small molecule drugs, with a particular focus on certain niches of clinical need. We have an extensive preclinical program focused on the discovery and development of potential small molecule candidates, supported by our diverse library of proprietary compounds and expertise in automated synthesis, medicinal chemistry, high-throughput screening, fragment-based drug design and biochemical target validation.
One of our most promising small molecule developments, which recently completed late-stage clinical trials, is an oral drug for Gaucher disease. This promising candidate functions via a different mechanism of action than the enzyme replacement therapy currently used to treat the disorder. As a substrate reduction therapy, it blocks the biosynthesis and resulting accumulation of the lipid that causes the disease symptoms.
Our research has led to the approval of several hematologic oncology products that are now in the Sanofi portfolio, including chemotherapies for patients who have not responded to other treatments. We have ongoing research in an effort to expand these existing cancer therapy indications, as well as to target new areas, such as polycystic kidney disease.
| Types of therapies developed: |
chemotherapy drugs; lipid reduction/inhibition therapies |
| Most relevant research areas: |
genetic disease; renal disease |
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Biomaterials are manufactured substances that can perform, augment, or replace natural functions in the body. Composed of either biologically derived or biocompatible synthetic materials, they are designed to function safely when implanted in the body for a wide range of medical applications.
Since the mid-1980s Genzyme has been a leading producer of high-quality sodium hyaluronate (HA, hyaluronic acid), a naturally occurring biopolymer that has many medical uses. Building on this expertise, we've developed a suite of HA-based products that are now in the Sanofi portfolio, including injectable viscosupplements (lubricants) to alleviate knee pain related to osteoarthritis, and materials used during some types of abdominal surgery to help prevent the formation of adhesions (internal scarring). Our current research is increasingly focused on leveraging biomaterials to enable more accurate, localized delivery of cell, protein-based, and other therapies.
| Types of therapies developed: |
viscosupplements; cartilage repair; adhesion barriers |
| Most relevant research areas: |
orthopedics; surgery |
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In diseases caused by a specific, known gene abnormality, gene therapy aims to treat the disease by inserting a normally functioning version of the gene into the patient's cells. The technology is still relatively new; challenges still exist in identifying optimal gene delivery mechanisms, and there are very few products currently available. Yet with further development, gene therapy has great potential to treat the underlying cause of many serious diseases.
While Genzyme does not currently have any marketed gene therapy products, we have one of the industry's longest standing and largest research programs in the field. Our earliest efforts focused on a treatment for cystic fibrosis, and we have since expanded to explore gene therapy targeting cardiovascular disease, ocular disease, and several genetic diseases. Clinical trials are now underway for a treatment for Parkinson's disease and age-related macular degeneration. Genzyme also holds significant intellectual property around gene therapy delivery vehicles, including both viral and non-viral vectors, strengthening our ability to evolve our research into practicable therapies.
| Types of therapies developed: |
adeno-associated virus (AAV) vectors |
| Most relevant research areas: |
neurologic disease; ocular disease
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A Collaborative Model
Genzyme’s research facilities are clustered in Massachusetts; this close proximity enables our scientists to share ideas and collaborate easily. As part of the Sanofi Boston R&D Hub, our research teams can tap into a wide range of expertise both within the company and in the scientific community of the greater Boston area.
Genzyme's roots are in biotechnology – the field of health care that uses biology and biological (rather than chemical) resources for medical purposes. We pioneered the first marketed therapeutics in two major biologic areas: enzyme replacements for patients with certain rare genetic diseases and cell therapies to rebuild damaged tissues.
Over the years, we've continued to build on our biologic expertise, exploring the many ways that medicine can leverage natural substances such as proteins, cells, and genes. But we've also advanced research in synthetic, chemistry-based pharmaceuticals, developing state-of-the-art technology to investigate small molecule and polymer drugs for many medical conditions.
A Focus on Therapeutic Needs
The starting point of all our research efforts is a significant medical need. We focus on developing breakthrough therapies within therapeutic areas such as genetic diseases and multiple sclerosis.
Find out more about our research areas