Antibodies are powerful immune proteins that recognize and bind to specific molecules (called “antigens”), marking them for destruction. Because antibodies bind very specifically to their target, they are efficacious and safe, and often with reduced side effects. In addition, due to their large size, antibodies stay in the human body and are active for a long time. By coupling antibodies to drugs, Sanofi is developing novel medicines that can specifically target tumor cells, while sparing healthy ones—a key strategy for designing more effective cancer therapies.
Peptides are short strings of amino acids that can move around the body, including in and out of cells, more readily than their larger protein counterparts. Sanofi is exploiting this property to develop new peptide-based treatments, including experimental trigonal peptides as well as other novel entities.
Enzymes work as catalysts, accelerating biochemical processes by binding to specific targets. Just as only one key can fit precisely into a lock, only certain compounds can be bound by a particular enzyme. This specificity is part of what makes enzymes such useful therapeutics. Sanofi is currently pursuing the use of enzymes as treatments for metabolic diseases.
Proteins typically act as separate, independent entities. However, in the laboratory, these individual units can be fused together, creating novel proteins that possess new and important biological functions. Sanofi is pioneering ways to harness fusion proteins as novel disease therapies.
Typically, antibodies recognize only one type of molecule, but in the laboratory, they can be engineered to recognize two or three different targets. Such bi- and tri-specific antibodies represent a powerful new weapon in the therapeutic arsenal, and Sanofi has custom-made technology platforms capable of engineering and producing these multi-faceted therapeutic agents.
Small molecules are chemical substances that are typically taken as a pill and absorbed into the body via the intestines. Small molecules can also enter cells easily because of their small size relative to other biological entities, such as antibodies which are much larger. Small molecule drugs can interact with a variety of biological targets to achieve a positive pharmacological treatment of disease. For example, in the case of cancer, small molecules can disrupt the functioning of the cancer cell, causing tumor cells to die. Many of the targeted cancer therapies in use today are small molecule drugs. With its legacy of innovation in cancer, Sanofi is now working to discover the next generation of cancer treatments. Through a partnership with REVOLUTION Medicines, Sanofi is developing inhibitors of enzymes that regulate critical cellular proteins, targeting therapies against difficult-to-treat cancers. Within Sanofi, there are also many small molecule programs targeting treatments in oncology, immunology, neuroscience, rare diseases, and rare blood disorders.
Nanobodies® - Size matters
Immunoglobulin single variable domains Nanobodies® are molecules derived from a very special type of antibodies produced naturally by llamas and other camelid species. Nanobodies® are just a tenth the size of conventional antibodies. This small size gives them the potential to reach disease targets in the human body that are inaccessible to conventional antibodies. Nanobodies® can easily be connected like beads on a string. Using state-of-the-art technology, our scientists can create molecules able to reach multiple disease targets at the same time. Nanobodies® hold therapeutic promise for a wide range of human diseases, and with our proprietary platform we can generate these rapidly and in quantities to facilitate large-scale production.
Gene therapy is an emerging form of disease treatment that involves introducing a gene into a patient’s cells, often to compensate for one that is missing or defective. This approach holds extraordinary potential for a variety of illnesses, and Sanofi is currently pursuing gene therapy programs in multiple therapeutic areas.
Instead of replacing a faulty gene, it is now possible to repair it—by adding or removing DNA directly from the defective gene itself. Such gene editing relies on specialized enzymes that cut DNA at defined locations, including zinc finger nucleases (ZFNs), a class of engineered DNA-binding proteins that facilitate targeted editing of the genome. In collaboration with Sangamo Therapeutics, which has developed an advanced zinc finger protein platform for developing genomic medicines, Sanofi is exploring ways of harnessing ZFNs as novel disease treatments.
Sanofi, in close partnership with BioNTech, is discovering and developing a new class of experimental immunotherapies that use messenger RNA (mRNA), a molecular “script” that codes for the generation of a specific protein in a cell to promote an immune response to an individual patient’s tumor. BioNTech has advanced technological platforms for engineering therapeutic mRNAs that enable the body to make its own medicine—a revolutionary approach that is poised to transform the treatment of diseases, ranging from cancer to infectious disease to heart disease.
RNA interference (RNAi) represents a promising new approach to disease treatment. It works by interfering with gene activity, making it possible to reduce the production of disease-causing proteins. Sanofi, in collaboration with Alnylam, is harnessing this natural biological process of RNAi to explore novel therapeutic approaches.
Expanded Genetic Alphabet*
DNA is composed of two pairs of chemical bases: adenine (A) and thymine (T), guanine (G) and cytosine (C). These letters make up the “genetic alphabet”, which provides all the instructions needed to create life. They provide instructions to create the 20 amino acids that make up all the proteins in our bodies, which do everything from holding cell walls together to catalyzing biological reactions. These amino acids also provide the raw material for creating protein therapeutics, for example interleukin-2 (IL-2) for treating cancers, and antibiotics to treat infections.
Scientists invented a new pair of DNA letters, nicknamed X and Y, that can function in living systems. This invention led to the creation of an Expanded Genetic Alphabet by Synthorx, now part of Sanofi. Using six DNA letters instead of four, the Expanded Genetic Alphabet allows scientists to generate novel amino acids that can be used to build entirely new proteins. These new proteins, called Synthorins®, fill important gaps in protein therapeutics by vastly expanding the variety of materials available to bioengineers. This opens the door to creating new medicines, e.g. to re-program protein interactions that cause immune cells to attack healthy ones.
Used on its own or in combination with other Sanofi platforms, such as Nanobody® technology, the Expanded Genetic Alphabet is enabling the company’s scientists and bioengineers to develop novel biologics for cancer and other diseases.
* MAT-GLB-2001338 V1.0 08/2020