White Paper

Avoiding Costly And Time-Consuming Mistakes In Gene Therapy Process Development

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The application for approval of Zolgensma, a gene therapy for spinal muscular atrophy (SMA) was granted  was granted Fast Track, Breakthrough Therapy, Priority Review and Orphan Drug designations by the US FDA.i  Affecting about 1 in every 11,000 births, SMA is a genetic neuromuscular disease and the leading genetic cause of death in infants and toddlers.ii   Many gene therapies are in development for similar diseases – those which strike early in life, significantly impact the quality and length of life and affect small patient populations.  These factors combine to create a sense of urgency for both developers of these therapies and patients who anxiously monitor news from clinical trials and eagerly await access once approval is granted.  

Our collective responsibility as participants in the gene therapy industry is to discover, develop and bring these innovative therapeutics to patients in need with a similar sense of urgency, effectively balanced, of course, with quality and safety.  Success in the evolving and highly competitive gene therapy space, however, requires navigating many uncertainties related to process development and manufacturing of adeno-associated virus (AAV) and lentivirus vectors, especially when development and manufacturing teams are operating with compressed timelines.  

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