Receipt, Storage, Preparation, and Dispensing of In Vivo Gene Therapy

An overview of the key considerations associated with receiving, storing, preparing, and dispensing in vivo gene therapies

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Introduction

An institution should have standard operating procedures in place for the receipt, storage, preparation, and dispensing of gene therapy products1

Receipt and Storage

A gene therapy product should be received and stored in a specifically assigned room by authorized, trained staff1,2.  An example of the workflow is shown in the figure

Procedure for Receiving and Storing Gene Therapy Products

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Figure adapted from Chart 1 in Vulto AG, et al2. *When gene medicines are delivered, the secondary packaging is guaranteed as not contaminated; †If vial is damaged, the primary packaging should follow the decontamination process; ‡Location of storage must ensure that no unauthorized person can gain access and there is no undue exposure of hospital staff to the product. A biohazard label may be required on the door to the storage room, depending on the biosafety level (BSL) of the stored gene medicine.

The universal biohazard warning symbol should be included on all gene therapy products, and posted on storage units and at the entrance to any room where the agent is present1

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The following should also be provided at the entrance1,2

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Preparation

The person preparing the gene therapy product should follow U.S. Pharmacopeia (USP) 800 standards for safe handling of sterile preparations1–5

Personal Protective Equipment (PPE)1–7

  • The level of PPE will depend on the BSL required to handle a particular viral vector
  • Most modern viral vectors are considered appropriate for use under BSL-16,7
  • However, many institutional biosafety committees require adherence to BSL-2 due to the unknown effects of accidental exposure7

BSL-16

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BSL-22,6

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Click here for information on the PPE requirements for BSL-3 and BSL-4

Compounding

USP 800 standards on PPE and BSL requirements, in addition to product-specific instructions from the product information, should be followed1–3

Compounding should occur inside a biosafety device, such as a validated (minimum Class II, Type B, see figure) biosafety cabinet (BSC) or isolator, with a background grading per local guidance/regulations1–3

Disposable “chucks pad” of plastic-backed absorbent paper may be placed on the BSC surface to reduce the potential for dispersion of droplets/spills and facilitate clean-up; the paper should be removed after each preparation3

Class II Type B2 BSC

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Figure adapted from Centers for Disease Control and Prevention6. HEPA, high-efficiency particulate air.

Dispensing

A gene therapy product prescription should be checked based on normal pharmacy procedure2

When a therapy is new to the institution, an accountability log can be a useful means of tracing steps in the handling of the gene therapy product2

References

  1. Blind JE, et al. Am J Health Syst Pharm 2019;76:795–802.
  2. Vulto AG, et al. EJHP Pract 2007;13:29–39.
  3. Armitstead JA, et al. Hosp Pharm 2001;36(1):56–66.
  4. Petrich J, et al. J Pharm Pract 2020;33(6):846-855.
  5. Stanford University. Adeno-associated virus fact sheet. Available at: https://ehs.stanford.edu/reference/adeno-associated-virus-fact-sheet. Accessed March 23, 2020.
  6. Centers for Disease Control and Prevention. Biosafety in Microbiological and Biomedical Laboratories. 2009. Available at: https://www.cdc.gov/labs/pdf/CDC-BiosafetyMicrobiologicalBiomedicalLaboratories-2009-P.PDF. Accessed July 17, 2020.
  7. Swindle S. Curr Protoc Hum Genet 2018;96:2.1.1–12.1.17.