Institutional Preparedness for Handling In Vivo Gene Therapy

An overview of the key biosafety considerations associated with handling of in vivo gene therapy products and establishing protocols in your institutions


It is recommended that gene therapy products are treated as biohazards; therefore, their handling requires specific policies and protocols1–3

  • Experts recommend that institutions create their own standard operating procedures (SOPs) to cover the handling of gene therapy
  • Institutions should evaluate each gene therapy individually via an assessment of the risk group and biosafety level of each viral vector, including the potential risk associated with the encoding transgene2

Development of SOPs

SOPs are important for ensuring the safety of healthcare professionals (HCPs) and patients, in addition to maintaining the integrity of the gene therapy and preserving the environment2

Institutional Prepardness 1

Clinical Biosafety Committee

Experts recommend that a clinical biosafety committee be formed within each institution with the aim of developing and standardising safe-handling protocols and evaluating approved gene therapy products1. This is a clinician- or pharmacist-led cross-departmental committee that would include the following members2:


*The inclusion of an additional pharmacist specialising in gene therapy products is optional.

Operations Requiring SOPs

A number of operations will require the development of SOPs, as shown below2,3

BSC, biosafety cabinet; SOP, standard operating procedure.


Institutional Training

  • With the introduction of gene therapy into institutions, educational gaps among staff members may emerge, which would need to be addressed2,3
  • Training should be tailored to the needs of different staff members, e.g., pharmacists or infusion nurses1
  • HCPs involved in the handling and/or administration of such products may require training on gene therapy molecular biology principles2
  • Additional product-specific training on the potential risks and hazards associated with a gene therapy, such as the risk of an immune response or seroconversion, is also recommended2

Specific Training Areas Include1–5

SOP, standard operating process.

Caregiver Training

Institutions will also need to be able to provide training to caregivers on product-specific information, which would include2:

  • Administration issues

  • Waste handling

  • Viral vector shedding

  • Immune response

  • Seroconversion


To allow for the safe handling and administration of gene therapy, institutions should ensure that the proper infrastructure is in place. Examples of infrastructural requirements are shown in the figure2


BSC, biosafety cabinet; USP, US Pharmacopeia.


  1. Petrich J, et al. J Pharm Pract 2020;33:846–855.
  2. Armitstead JA, et al. Hosp Pharm 2001;36:56–66.
  3. Vulto A, et al. EJHP Practice 2007; 13: 29–39.
  4. Blind JE, et al. Am J Health-Syst Pharm 2020;76:795–802.
  5. World Health Organization. Laboratory biosafety manual. 4th edn. 2020. Available at: Accessed 16 July 2021.