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 authorised, trained staff.2,3 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 Vulto AG, et al. 20073. *When gene medicines are delivered, the secondary packaging is guaranteed as not contaminated; †If the vial is damaged, the primary packaging should follow the decontamination process; ‡Location of storage must ensure that no unauthorised 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 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 present2

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The following should also be provided at the entrance2,3

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Preparation

 

Personal Protective Equipment (PPE)

  • The level of PPE will depend on the biosafety level (BSL) required to handle a particular viral vector
  • Most modern viral vectors are considered appropriate for use under BSL-13,4
  • However, many institutional biosafety committees require adherence to BSL-2 due to the unknown effects of accidental exposure4

BSL-13,5

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BSL-23,5

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

Compounding

Product-specific instructions from the product information, should be followed

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/regulations2,3,6

Disposable chuck pads 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 preparation6

Class II Type B2 BSC

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Figure adapted from Centers for Disease Control and Prevention5.
HEPA, high-efficiency particulate air.

Dispensing

A gene therapy product prescription should be checked based on normal pharmacy procedure3

When a therapy is new to the institution, an accountability log can be a useful means for tracing steps in the handling of the gene therapy product3

References

  1. Petrich J, et al. J Pharm Pract 2020;33:846-855.
  2. Blind JE, et al. Am J Health-Syst Pharm 2019;76:795–802.
  3. Vulto AG, et al. EJHP Pract 2007;13:29–39.
  4. Swindle S. Curr Protoc Hum Genet 2018;96:2.1.1–12.1.17.
  5. 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 20 July 2021.
  6. Armitstead JA, et al. Hosp Pharm 2001;36:56–66.