Role of Pharmacy Professionals in Gene Therapy Based on Adeno-Associated Viruses: Treatment of Hemophilia as a Template of Care.

Background: Gene therapy using adeno-associated viruses (AAVs) has emerged as a promising approach for the treatment of genetic disorders, including hemophilia, a rare bleeding disorder. Recently approved AAV-based treatments for hemophilia, such as etranacogene dezaparvovec and fidanacogene elaparvovec, could provide long-term benefits by targeting the genetic basis of the condition. Nonetheless, the application of gene therapies presents several challenges that necessitate a multidisciplinary approach. Objectives: To explore the challenges associated with AAV-based gene therapy and the role of pharmacy professionals in ensuring its safe and effective application, using treatment of hemophilia as a care model. Data Sources: Relevant literature was identified through searches in the PubMed, Embase, and Google Scholar databases, with a focus on publications related to AAV-based gene therapy, hemophilia, and the involvement of pharmacy professionals. Study Selection and Data Extraction: Peer-reviewed articles, clinical guidelines, and regulatory documents were selected based on their relevance to the clinical, logistical, and ethical dimensions of AAV-based gene therapy. Data Synthesis: Pharmacy professionals are integral to the continuum of care for AAV-based gene therapy. Their responsibilities include product handling and preparation, patient education, medication management, and long-term safety monitoring. Furthermore, pharmacy professionals can help address challenges such as financial obstacles, regulatory adherence, and ethical issues. Conclusions: Using their expertise in medication management, patient education, and health system processes, pharmacy professionals can enhance the safety, effectiveness, and accessibility of AAV-based gene therapies for hemophilia, ultimately leading to better patient outcomes.