Gene therapy administration: A collaborative model for complex care
Author: Sarah Hale
Geisinger continues to expand its capabilities in delivering advanced therapies. Among the most notable are gene therapies for pediatric patients, including onasemnogene abeparvovec-xioi (Zolgensma) for spinal muscular atrophy (SMA) and delandistrogene moxeparvovec-rokl (Elevidys) for Duchenne muscular dystrophy (DMD). These treatments represent some of the most complex and costly interventions in modern medicine, with wholesale acquisition costs exceeding $2 million to $3 million per dose.
Successfully delivering these therapies requires seamless coordination across multiple disciplines, including pediatric neurology, pharmacy, informatics, formulary and procurement, finance and nursing. From securing prior authorizations to managing ultra-low temperature logistics and carefully timed dose preparation, each step demands meticulous planning, precision and teamwork. For example, Elevidys must be placed in an ultra-low freezer or refrigerator within 5 minutes of removal from its specialized shipping container. It must then follow controlled thawing instructions and adhere to a tightly regulated 6-hour timeline to begin infusion after preparation.
Zolgensma is supplied as a kit containing 5.5-mL or 8.3-mL vials, with each kit tailored to the patient’s weight — requiring between 2 and 14 vials in specific combinations. To support this complexity, the informatics team provided essential assistance in building customized, weight-based medication records that allow accurate and efficient preparation. Hazardous drug handling and waste disposal considerations were also critical components of our gene therapy readiness strategy, requiring specialized protocols and training.
These coordinated efforts across teams enabled the safe and timely administration of therapies with intricate handling and infusion requirements. By building robust infrastructure and fostering collaboration, Geisinger has positioned itself as a leader in offering transformative therapies that redefine possibilities for pediatric patients. The success of these programs reflects not only clinical excellence, but also a deep commitment to innovation and patient-centered care.
Looking ahead, Geisinger remains focused on expanding access to advanced therapies and refining operational workflows to support future treatments. As gene therapy continues to evolve, our teams are actively engaged in evaluating emerging products, updating protocols and staying ready for next-generation interventions. This forward-thinking approach means our youngest patients continue to benefit from the most cutting-edge care available.
