Biotechnology Awards 2021

GHP / Biotechnology Awards 2021 9 Improving Cell and Gene Therapy Treatments Worldwide been yielding lucrative results, including a major licensing deal with Sanofi. Novel AAV capsids with optimized tissue tropism and enhanced delivery have been developed using technologies such as directed evolution through peptide insertion, and shuffled and barcoded AAV libraries. In tandem with AAV capsid development, SIRION has been upgrading and updating its vector design services in line with the latest industry standards, regulations, and innovations. This includes promotor choices and enhancer elements which provide optimal expression in the targeted tissues and vector optimization to decrease contamination and increase the number of undamaged genomes in the batches. Additionally, as part of ongoing optimization of its manufacturing processes, it is currently beta- testing an improved Lentivirus purification protocol. In this way, it does not just benefit from industry innovations, but it actively contributes to them, making itself a lynchpin of forward momentum for its sector. The overarching mission for SIRION is to to help cell and gene therapy companies get the best treatments to their patients as efficiently and cost-effectively as possible. To this end, it is committed to expanding its value chain, having recently opened a new Cell and Gene Therapy Clinical Support department to help customers with regulatory frameworks around EMA and FDA guidelines at a very early stage in vector development, saving both time and money at the clinical trial stage. SIRION innovations are forever striving to make genetic therapies safer and more effective for its clients and their patients. For example, lowering the amount of viral vector needed for the therapy reduces the risk of harm, so SIRION is actively working on increasing expression levels, improving targeting, and decreasing immunogenicity. One of the most challenging elements of viral vector production is upscaling the manufacturing process for pre- clinical and clinical trials. Large scale, high quality, and cost Adeno-Associated Virus Dr. Christian Thirion, CEO effective manufacture is essential for the industry’s success. SIRION’s Process Development Department is committed to increasing production capacity by expanding procedures in line with GMP requirements whilst maintaining vector quality. In addition, the newly established GMP Alliances Department works with clients to ensure smooth transfer of protocols and analytics to certified GMP manufacturers. This saves clients time and reduces risk. SIRION Biotech’s proprietary developments also include its product LentiBOOST ® , a highly effective and non- cytotoxic transduction enhancer for preclinical and clinical applications of lentiviral vector technology. It is universally acting, receptor independent, and can be applied to a wide range of cell types in modern clinical work. The technical efficacy of LentiBOOST ® makes it a promising candidate for the improvement of clinical transduction products for ex vivo gene therapies and CAR-T cell therapies. LentiBOOST ® has undergone rigorous testing and enhancement since it was first developed, and is currently in use in more than 20 phase 3 and phase 1/2 clinical trials. It has become an industry staple, with scientific institutions and businesses incorporating it in trials all around the world. The licensing strategy for LentiBOOST ® is another factor that has enhanced its popularity. SIRION supplies its licensees with GMP-grade material and allows research hospitals to apply its technology on a royalty free basis, to give a tangible example of its dedication towards promoting the betterment of its field. This operational model has allowed it to help patients in all corners of the globe as well as to further spread awareness of this amazing technology and its use for enhancing public health. In a recent update to LentiBOOST ® making waves in the world, Mustang Bio have announced the use of LentiBOOST ® in a crucial phase 2 trial for MB-207 in X-linked severe combined immunodeficiency. SIRION acknowledges that Europe’s funding opportunities for clinical stage companies is more limited than countries such as the US, and so it works hard to support its licensees in attaining funding. This has not just helped increase the overall use of LentiBOOST ® , but to better the European biological technology sphere in general. LentiBOOST ® is a product that complies with Eur. Pharm and USP. Of course, working within such a dynamic industry that is constantly in flux, SIRION’s LentiBOOST has competitors – however, it sets itself apart from them by way of LentiBOOST ® ’s ability to increase overall transduction efficiency at a lower vector dose. Non-toxic, effective, and coming with the benefit of SIRION providing identical GMP and non-GMP product, LentiBOOST ® has quickly risen to first place in the race for continued progress. With this in mind, it is easy to see how SIRION has made itself a standout voice in its field. Company: SIRION Biotechnology Contact: Joanne Crowe Web: