Biosafety Considerations for Viral Vector Gene Therapy: An Explanation and Guide for the Average Everyday-Hero Pharmacist

Abstract

Purpose An overview of the multi-faceted biosafety points that must be taken into consideration by pharmacists and pharmacies in order to provide viral vector gene therapy to their practice site. Summary As science and medicine evolves, pharmacists and other healthcare workers are continually faced with unique challenges in the workplace. They are expected to be informed and proficient on new therapies and standards of practice, and be able to apply this knowledge appropriately for their patients. One such advancement that seems to be picking up speed in recent years is gene therapy, which is often achieved with the assistance of a viral vector. As these viral vector doses move closer to mainstream medicine, a host of issues and concerns for the pharmacists, nurses, and caregivers that are involved in the process begin to rise to the surface, often rooted in the critical concern: “How do we dispense, utilize, and administer these doses safely?” Unfortunately, there is no singular, concise source of information for addressing biosafety with viral vector products, and guidance must be gathered from a variety of resources in order to mesh together a reasonable working process. Conclusion: While this may seem to be a daunting task, facilities that already meet USP 797 and USP 800 guidelines are well on their way to being ready to provide viral vector doses. By incorporating additional steps and reviewing biosafety specific resources, these sites can easily adapt to provide these new and novel therapies for their patient population.

A comprehensive literature scoping review of infection prevention and control methods for viral-mediated gene therapies

“… 8 , 9 , 42 When working with viral vector gene therapies, clinical staff should wear appropriate personal protective equipment, including gowns, gloves, and eye or respiratory protection, and should further be educated on the potential risks of percutaneous exposure through accidental needlestick. 7 , 9 …”

“…Viral vector-mediated gene therapies use modified viruses as drug-delivery vehicles to introduce specific DNA sequences, regulatory RNAs, or other therapeutic substrates into cells 5 , 6 Commercial gene therapy agents, such as onasemnogene abeparvovec and voretigene neparvovec, are comprised of an adeno-associated virus (AAV) vector, whereas the oncolytic virus, talimogene laherparepvec, is comprised of a modified herpes simplex virus. 7 Nearly all gene therapies commercially available use one of three vector types: AAV, adenovirus (AV), or lentivirus (LV). 4 , 8 Adeno-associated virus and AV vectors are typically used in gene therapies directly administered to patients by infusion or local administration, with AAV being the most popular vector for areas outside of oncology and vaccines.…”

European Association of Hospital Pharmacists (EAHP) Guidance on the Pharmacy Handling of <em>In Vivo</em> Gene Therapy Medicinal Products

“…Considering this lack of experience, many institutions are expected to need to procure additional equipment and/or develop new facilities, such as ultra-low temperature freezers and biosafety devices, to provide infrastructure for the safe handling of GTMPs (1). Different set-ups can be implemented depending on individual institution's requirements and in accordance with local regulations.…”

“…Experts from the USA and Australia have also produced opinion and review articles providing guidance on the handling of GTMPs in a hospital pharmacy setting (1,(19)(20)(21)(22)(23)(24). Common recommendations across documents include limiting access to GTMPs to trained personnel, using appropriate biosafety containment equipment and personal protective equipment (PPE), using appropriate containers for transport, developing institutional procedures and policies, and ensuring appropriate decontamination and waste management (19)(20)(21)(22)(23)(24).…”

“…The development of institutional SOPs should also consider local regulations, policies, and proceduresfor example, in Denmark, each facility must go through a formal application process before using any GTMP, to ensure safety for personnel, patients and the environment. However, it has been noted that, as of 2022, few national and/or industry-wide guidelines relating to GTMPs had been developed because of the novelty of these therapies (1). All the information required for each step is included in the appropriate chart or table (or their accompanying footnotes).…”