Main Conference - Day 3 - GMT (Greenwich Mean Time, GMTZ)
Main Conference - Day 3 - GMT (Greenwich Mean Time, GMTZ)
- Darrin Morrissey - Chief Executive Officer, National Institute for Bioprocessing Research and Training
Gene therapy may be one of the biggest paradigm shifts in modern medicine. The patient journey to achieving a haemophilia free mind isn’t something that can happen overnight. Join us as we unravel the lessons learned from the launch of a first-in-class, one-time gene therapy for haemophilia B. will start by understanding why haemophilia B is an ideal target for gene therapy and review the clinical development programme. We will then hear practical examples on what this treatment option means for patients and HCPs along the disease and gene therapy journey, looking at both the opportunities and potential challenges. Together we will not just discuss lessons learned, but form habits to help improve the future management of this condition.
- Karen Pinachyan - Head of Global Medical Affairs Hematology, CSL Behring
- Alex Templar - Senior Business Development Manager - Manufacturing, Cell and Gene Therapy Catapult
- Rob Noel - Head of Consultancy, Biopharm Services Ltd
- Alex Templar - Senior Business Development Manager - Manufacturing, Cell and Gene Therapy Catapult
Whilst advanced therapies (AT’s) are at the cutting edge of medicine, there is a disparity between these products and their raw materials. Serum-derived human and animal components are often utilized throughout the AT manufacturing process. Albumin is a natural multitool, its binding properties, tendency to act as an antioxidant, ability to reduce surface adsorption and mitigate aggregation make it useful across multiple unit applications. The variability in serum-derived albumins directly results from albumin's innate binding properties. This variability can directly impact your process and product. Join Dr Phil Morton, CTO of Albumedix, to discover how recombinant albumin could better support your AT’s process consistency.
- Phil Morton - Chief Technology Officer, Albumedix
- Nasser Sadr - Senior Director Global Analytical Development, uniQure
- Nasser Sadr - Senior Director Global Analytical Development, uniQure
- Jonathan Bones - Principal Investigator and Director, National Institute for Bioprocessing Research and Training
Over the past decade, gene therapy has revolutionized medicine for human diseases. In particular, recombinant adeno-associated virus (rAAV) has been used extensively for transgene delivery in clinical studies. One of the biopharmaceutical industry's biggest challenges in this regard is establishing production processes that can keep up with the increasing demand for volume and consistent quality of viral vectors.
ELEVECTA™ stable producer cell lines enable high yield, scalability, and robustness in the manufacturing process. All components necessary for vector production are stably integrated into the genome, which avoids the need for transient transfection or helper virus.
With current AAV processes, impurities are a major challenge for chemistry, manufacturing, and controls (CMC). Unpackaged host cell components like DNA, RNA, proteins, and lipids (collectively “process-related impurities”) can be removed by conventional downstream methods. However, product-related impurities involve host cell components that are incorporated into the viral capsid and are therefore resistant to classic purification methods. Among these encapsidated impurities, host cell DNA (hcDNA) is the most problematic. Safety concerns associated with encapsidated hcDNA relate to both potential genotoxicity and immunotoxicity.
We have developed a technology that reduces hcDNA encapsidation during both transient and stable rAAV production. In combination with our stable producer cell line, the new technology helps meet the steadily growing demand for rAAV while enhancing product quality.
- Vanessa Kress, PhD - Business Development Manager, CEVEC – now part of Cytiva
- Jahid Hasan - Lead Technical Scientist, The Cell and Gene Therapy Catapult
- Melody Dai - Executive Director, Regulatory Affairs, Adverum Biotechnologies
- Guido Moll - Co-Principle Investigator & Deputy Group Leader, Charité Universitätsmedizin Berlin
- Christopher Bravery, Ph.D. - Consulting Regulatory Scientist, Advbiols
- Madison Lee - Associate Director, CMC Program Management, Regeneron