My summary of an article from BCG titled "From Lab to Marketplace, Succeeding with Gene Therapies", which taught me that I should look at the gene therapy "from lab to marketplace," not just the science of it.
Point 1. Put "a high premium on speed and creativity in clinical and regulatory approaches": Most of gene therapies are one-and-done treatments; the genetic cause of the disease is fixed, so you don't need any future hospital visits or medicine at all. In addition, many gene therapies target rare diseases, meaning that the patient populations are small to begin with. Therefore, the market shrinks rapidly once the initial drugs successfully treat the patients. In other words, there won't be much of the pie left for the late comers.
Point 2. Approval is only the first step: In addition to investing in R&D, biotech and pharmaceutical companies should find a way to manufacture the necessary components fast and deliver the therapies to patients. I wonder how the unprecedented rate of production and delivery of COVID-19 vaccines would change the manufacturing strategy of gene therapy in the future.
Point 3. Too expensive to get?: A one-and-done treatment leads to a life-changing result, but it also means a high cost. Thus, pharma companies should come up with creative coverage and payment models in order to sell their gene therapies. "It [Pharma] needs to be flexible in how it derives its revenue and profit streams," the article added.