Just as vaccines transformed medicine over a century ago, the rapid technological advances of the past decade are changing drug development as we know it. At Sanofi, industry-leading technology platforms are giving our R&D teams the tools they need to design pioneering therapeutics that would have been unimaginable just a few years ago. These technologies are also shedding new light on the biology behind many diseases, helping our teams target the root causes of disease with more precise medicines.
Small molecules–drugs typically taken as a pill and absorbed into the body via the gut–can enter cells easily and interact with biological targets to treat disease. Our small molecule platforms help our teams develop innovative treatments across therapeutic areas. One example is cancer, where many therapies in use today are small molecule drugs that can disrupt the functioning of cancer cells. Our teams are using protein degrader technology and other approaches to develop drugs that could destroy key proteins in several types of cancer, including breast cancer.
Our TAILORED COVALENCY® platform gives our scientists unprecedented control over how a drug can bind–reversibly or irreversibly–to its target protein. Our teams can design drug candidates that create a strong bond with two targets: the target site affecting the disease, and a unique site adjacent to it. This approach works like a "double lock and key". Small molecules can be designed to stay on their target for longer periods of time, which reduces their ability to interact with other, off-target molecules. This could reduce the need for the sustained, elevated drug levels that are typically required by standard drugs, and potentially minimize unintended side effects.
Novel Antibody Technologies
Monoclonal antibodies are highly specialized immune proteins that can be engineered to recognize and interact with specific targets in the body. Our teams are using several different monoclonal antibody technologies to develop new therapeutics against cancer, viral infections, and other diseases.
Antibodies typically recognize only one type of molecule. Our teams use proprietary technology to develop antibodies that can recognize two or three different targets. These "multi-specific" antibodies combine key features of several different antibodies into a single therapy. This powerful approach presents new ways that could combat cancer and immunological disorders on many fronts.
Antibodies are powerful immune proteins that recognize and bind to very specific target molecules. Our teams can link antibodies to cancer-fighting drugs, forming "antibody–drug conjugates" (ADCs) designed to deliver powerful drugs directly to tumor cells. The antibody can have many small-molecule drugs attached to it. Like a GPS, it can guide them to a specific destination on a tumor cell before releasing them. Our platform helps our researchers develop cytotoxic and immune-stimulating ADCs. Cytotoxic ADCs are made with highly potent chemotherapy drugs that kill cancer cells. Immune-stimulator ADCs deliver a drug that activates the immune system at the tumor site, where it can mobilize immune cells to attack the diseased cells. Our teams are designing ADCs for both solid and liquid tumors.
NANOBODY® molecules are derived from a special type of antibody produced naturally by llamas and other camelid species. At a tenth the size of conventional antibodies, NANOBODY molecules have the potential to reach disease targets in the human body that are inaccessible to conventional antibodies. They are composed of antibody fragments strung together, designed to bind with many targets at once. This presents the possibility of replacing complex treatment regimens with single, multi-action medicines for immune-mediated diseases, cancer, and rare blood disorders.
Enzymes are proteins that 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 is one reason enzymes have so much therapeutic potential. Our teams are designing enzymes to be used as treatments for a range of metabolic diseases.
Proteins are usually independent entities, but they can be fused together to create novel proteins designed to carry out therapeutic biological functions. Our R&D teams are pioneering the use of fusion proteins as novel disease therapies.
Peptides are short strings of amino acids that can move around the body, 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.
Our teams are working on ways to repair defective, disease-causing genes by adding or removing DNA directly. These techniques rely on specialized enzymes that cut DNA at defined locations, including (but not limited to) zinc finger nucleases (ZFNs), a class of DNA-binding proteins that facilitate targeted genome editing. Using Sangamo Therapeutics' advanced ZFN technology, Sanofi and Sangamo researchers are collaborating to develop gene-edited cell therapies that could treat hemoglobinopathies, such as sickle-cell disease.
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. Sanofi's integrated, cross-disciplinary genomic medicine unit is accelerating progress toward gene and cell therapies by combining an array of technologies, from nucleic acid nanostructures to viral and non-viral gene-delivery platforms.
Messenger RNA (mRNA) are incredibly small but essential molecules in our cells that serve as the link between our DNA and all biological activities in our bodies. We produce mRNA continually: its purpose is to help translate DNA, our genetic blueprint, into the proteins we need to live. Sanofi is harnessing this natural molecular machinery to develop new, life-saving vaccines, and to address long-standing challenges in cancer, immune-mediated diseases, and rare diseases. Our mRNA platform supports our scientists in identifying and developing therapeutic mRNA sequences and ensuring they remain stable until they reach their target cell.
RNA interference (RNAi) represents a promising new approach to disease treatment. It allows our scientists to potentially reduce the production of disease-causing proteins by interfering with gene activity. In collaboration with Alnylam, we are harnessing this natural biological process of RNA interference to explore new therapeutic approaches for rare blood disorders.
DNA is composed of four chemical bases, which provide instructions to create the 20 amino acids that make up all human proteins and protein therapeutics. Our Expanded Genetic Alphabet adds a new pair of bases (often called letters), to DNA. Equipped with six letters instead of four, our teams can build new proteins, called SYNTHORIN™ molecules. This gives them the tools and material they need to design and optimize protein-based treatments for cancer and other diseases.
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MAT-GLB-2100354 v 1.0 | November 2021 | Last updated November 2021