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 protocols1,2

  • The National Institutes of Health (NIH) has stratified viral vectors into risk groups
  • However, specific clinical guidelines have not been provided by the NIH, Centers for Disease Control and Prevention (CDC), World Health Organization (WHO), or U.S. Food and Drug Administration (FDA)1
  • 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 transgene1

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 environment1

Institutional Prepardness 1

Clinical Biosafety Committee

Experts recommend that a clinical biosafety committee be formed within each institution with the aim of developing and standardizing safe handling protocols and evaluating approved gene therapy products1. This is a clinician- or pharmacist-led cross-departmental committee that would include the following members1:


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

Operations Requiring SOPs

The standards of the U.S. Pharmacopeia (USP) Chapters 797 and 800 should be followed when developing SOPs that cover all stages of gene therapy manipulation3

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

BSC, biosafety cabinet.


Institutional Training

  • With the introduction of gene therapy into institutions, educational gaps among staff members may emerge that would need to be addressed
  • 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 principles
  • 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 recommended1

Specific training areas include4

Caregiver training

Institutions will also need to be able to provide training to caregivers on product-specific information, which would include1:

  • 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 place1. Examples of infrastructural requirements are shown in the figure



  1. Petrich J, et al. J Pharm Pract 2020;33(6):846-855.
  2. Armitstead JA, et al. Hosp Pharm 2001;36(1):56–66.
  3. Blind JE, et al. Am J Health Syst Pharm 2019;76:795–802.
  4. U.S. Pharmacopoeia. General Chapter Hazardous Drugs – Handling in Healthcare Settings. Available at: Accessed July 13, 2020.