1、SPECIAL REPORTViral-Based Gene Therapy:Key Challenges and Bioprocessing Innovations2 L.E.K.ConsultingSPECIAL REPORTContentsAbout L.E.K.ConsultingWere L.E.K.Consulting,a global strategy consultancy working with business leaders to seize competitive advantage and amplify growth.Our insights are cataly

2、sts that reshape the trajectory of our clients businesses,uncovering opportunities and empowering them to master their moments of truth.Since 1983,our worldwide practice spanning the Americas,Asia-Pacific and Europe has guided leaders across all industries from global corporations to emerging entrep

3、reneurial businesses and private equity investors.Looking for more?Visit .L.E.K.Consulting is a registered trademark of L.E.K.Consulting LLC.All other products and brands mentioned in this document are properties of their respective owners.2022 L.E.K.Consulting LLCIntroduction.3Key challenges and bi

4、oprocessing innovations.4-12Conclusion.13Endnotes.143 L.E.K.ConsultingSPECIAL REPORTIntroductionIn recent years viral vector gene therapies reentered the advanced modality spotlight notably with the 2017 approval of Sparks Luxturna and the subsequent approval of Novartis/AveXis Zolgensma.Both of the

5、se therapies conveyed transformational potential for patients in high morbidity or mortality indications with significant unmet medical need.The immense promise of the budding adeno-associated virus(AAV)gene therapy field drove a flurry of M&A deal activity,with large biopharma and global contract d

6、evelopment and manufacturing organizations participating in many billion-dollar-plus deals.1 While the field has experienced several setbacks related to safety and efficacy,the global pipeline continues to grow,reaching hundreds of preclinical assets and more than 100 clinical assets by May 2022,2 a