New gene editing, synthetic biology, and nanobody technologies are reinventing the landscape of medicine. Combined with genomics and machine learning, this new toolbox has released a flood of possibilities for medicine, inspiring research and giving new hope to patients with genetic diseases.
Genomic medicine researchers at Sanofi are accelerating progress toward gene and cell therapies using an array of technologies, from nucleic acid nanostructures to viral and non-viral gene-delivery platforms. This work builds on four of the company's core strengths:
- Deep research and translational expertise in gene therapy;
- Emerging leadership in immuno-oncology R&D;
- Proprietary technology platforms for delivering therapeutic genes into target cells; and
- Integrated, large-scale clinical manufacturing for gene and cell therapies.
"A major bottleneck in the delivery of gene therapies has been the capacity of companies to support strong genomic medicine R&D and generate the highly specialized materials needed for manufacture. Sanofi has both," said Christian Mueller, Head of Genomic Medicine at Sanofi. "We are one of very few companies with the in-house capability to discover, develop, and manufacture gene and cell therapies at the right scale. But it's about more than just capacity – our cross-functional teams are working as one, from early research through development and production, to create new medicines that are potentially curative."
A biotechnology playground
Sanofi's genomic medicine R&D teams collaborate across disease areas to address challenges in genetic rare and neurological diseases, as well as some common diseases.
"For a scientist, Sanofi is like a big playground where you have access to all these incredible tools for exploring and building," said Sourav Choudhury, who leads the capsid engineering and adeno-associated virus (AAV) immunology laboratories for genomic medicine. "Here, you can take an idea out of a completely non-related field of research, test it out and see if it works. We can collaborate easily with teams working on technology platforms like Synthorin® molecules and apply technologies like machine learning to accelerate discovery."
Removing bottlenecks in manufacturing
Sanofi researchers are deploying the full technology toolkit in their quest to repair faulty genes behind many genetic and chronic diseases. This includes AAVs, viral-free gene delivery technologies, and mRNA therapeutics. Building on its long-standing expertise in virus-based vaccine CMC (Chemistry, Manufacturing & Controls) and manufacturing, the company is well placed to accelerate the delivery of genomic medicines.
“Sanofi has decades of innovation experience in biologics and continuous biomanufacturing processes. We are meeting the challenges that are currently holding back viral-vector-based genomic medicine. Many of these challenges come down to having highly efficient, research-enabling technology platforms," said Bruno Figueroa, global head of genomic medicine CMC at Sanofi R&D. "Our proprietary Producer Cell Line technology is an important platform for manufacturing AAVs, removing one of the biggest bottlenecks in gene therapy."
Smoothing the path to discovery
Sanofi is strengthening operations by creating deeply cross-functional teams. Specialists in viral vector engineering, cell line development, cell culture, biochemistry, chemistry, toxicology, translational medicine, preclinical evaluation, Good Manufacturing Practices, and regulatory are dedicated to making genomic medicine a reality.