Gene therapies


  • Gene therapy
  • human gene transfer

In DrugBank, biotech drugs are subcategorized according to their intrinsic properties. The gene therapy subcategory represents nucleotide sequences and vectors used for the genetic correction of pathological conditions inside cells.1 These types of therapies act either by promoting the production of certain proteins, inhibiting the expresion of a gene, or by correcting mutated pathological genetic sequences. Tisagenlecleucel is an example of an FDA approved gene therapy.


  1. Rosenberg SA, Aebersold P, Cornetta K, Kasid A, Morgan RA, Moen R, Karson EM, Lotze MT, Yang JC, Topalian SL, et al.: Gene transfer into humans--immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med. 1990 Aug 30;323(9):570-8. doi: 10.1056/NEJM199008303230904. [Article]