Our approach

What is gene therapy?

A gene therapy is a medicine that delivers a genetic code to the body, typically via a one-time-only dose.

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Building blocks of a gene therapy

There are many parts to a gene therapy, but the key ones are the capsid, the promoter, and the therapeutic transgene.1

The capsid is the outer shell of the AAV that packages the genetic code in a gene therapy. We use our cART platform to develop new adeno-associated virus (AAV) capsids. We prefer AAVs, as they don’t replicate on their own and aren’t known to cause human disease.1

The promoter controls how the body reads the genetic code for the therapeutic transgene and what it does with it. The promoter can be chosen either to enable the genetic code to work in all cell types or in specific cell types more than others. We methodically select the promoter for each therapy using our pART platform so it may optimally manage what the body does with the therapeutic gene.1

The therapeutic transgene is the genetic code to treat the disease. The code can include instructions for the body to start making a new protein that it didn’t make before (gene replacement), or to interfere with the body’s ability to make a specific protein that’s causing disease (knockdown), or to change the genes of cells (gene editing).1 It can also be used to help your body manufacture its own medicines (e.g., vectorized antibody).2


  1. Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019;18(5):358-378.
  2. Robert MA, Gilbert R, Gaillet B. Antibody delivery mediated by recombinant adeno-associated virus for the treatment of various chronic and infectious diseases. Curr Gene Ther. 2017;16(6):363-374.