Gene Therapies in Rare Disease

I’m sure most of you have seen the movie Field of Dreams, starring Kevin Costner as Ray Kinsella, an inexperienced, struggling farmer in Iowa. Early on in the movie, Kinsella hears a disembodied voice coming from his crops declaring, “If you build it, he will come.” The “it” in this scenario is a baseball field and the “he” is Kinsella’s father, who passed away some years prior. Kinsella does indeed build a baseball diamond; his father does comes back to play one last game of catch. If you have not seen the movie, I highly recommend it. It’s a feel-good yarn about lost chances, following your dreams, and the intricacy of human choices.

This e-book is obviously not about Field of Dreams, but the movie is a useful analogy for when we think about the challenges the pharmaceutical industry faces when launching a gene therapy into a rare disease space. Costner’s character took a giant leap of faith spending a large sum of money building a baseball field on top of his crops when financial ruin seemed a possibility. What if it was built but he never showed? What is more nuanced is how the movie revealed the profound complexities of how people behave unpredictably when they hope for a better, yet unknown future, particularly when their lives are rife with adversity.

We’re seeing incredible advancements in the development of medicines to treat people living with rare diseases. Enormous sums of money are being spent investigating regenerative medicines, like gene therapy. Analysts estimate that the global cell and gene therapy market size will top nearly $58B by year 2028. [citation: PR Newswire, Sept 2021] Despite significant growth opportunities in disruptive technologies, there are numerous risks and unknowns associated with developing gene therapies for rare diseases. And the industry needs to ask itself one very important question: if I build it, will they come?

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