Terminology: Gene Therapy

The question here is whether it’s correct—as was done by article writers in 2019—to treat gene silencing as not a kind of gene therapy.

For example, 2019-05-24: Zolgensma (based on proprietary [secret] viral gene therapy). Claimed selling point: first and only gene therapy for SMA. First and only? Only if ASO gene silencing doesn’t count as gene therapy:

2016-12-23: Spinraza (a non-viral ASO). Selling point: first FDA-approved drug for SMA.

Here’s what I (and Wikipedia) think: therapeutic gene silencing and gene editing are examples of gene therapy. It’s so obvious, it doesn’t even need to be explicitly stated. Here’s an example of explicitly coming to this conclusion.

Pharmaceutical companies want very much to make themselves sound new and fresh, working on the first of something. Twenty-one years ago (1998) was already the year of gene silencing in the form of ASOs, and the first RNAi drugs in 2018 already sounded vaguely like they were based on ancient technology. If you are working on ASO gene silencing in 2019, you risk sounding like you are working with ancient technology.

The ironic thing is that the concept of gene therapy has been around longer (since 1972) than all the specific techniques being FDA approved now. I think this will all settle out accurately eventually, without anybody admitting mistakes.

2021-04-16 update. This article pulls a surprise out of the hat and says:

ASOs are not usually considered to be “gene therapy” because they must be injected at regular intervals, whereas gene therapy is a one-time delivery.

In other words, by their definition, gene therapy delivery must be viral.


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2 responses to “Terminology: Gene Therapy”

  1. MICAEL HEBERR MATEUS Avatar
    MICAEL HEBERR MATEUS

    Jens,
    In your opinion, the path from which we can expect the treatment of SCA3 comes from ASO?

    1. Jens-Ingo Farley Avatar
      Jens-Ingo Farley

      I think that an ASO for Huntington’s disease will be developed first, then for SCA3 probably by the same company or partner. The main ASO for HD is being developed by Ionis/Roche. Somewhat related (gene silencing but not an ASO) is the solution being developed by Uniqure. Details.

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