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Gene Therapy – Durability

February 27, 2021


So the durability or how long gene therapies will last is one of the biggest questions facing this new class of treatment. Unfortunately, the answer is complicated. We don't yet know how long even the two gene therapies, true viral vector deliver gene therapies that are available in the United States, how long those treatments will last, because the followup period in the patients who have been studied isn't long enough. Ultimately we think that for some gene therapies, there will be probably not a cliff, but a point at which either retreatment or some other way to boost expression of the non-mutated gene that's been injected into the patient already and has been proven to work, can be increased. So the real answer to the question of durability of gene therapy is that the jury is still out. We don't know how long most of these therapies, if any of them, will really last. In the payer, in the insurance coverage side of the healthcare stakeholder group, the hope is that we can come up with contracting paradigms and ways to be able to hold the manufacturer or developer of these treatments accountable. So that if the treatment doesn't work for as long as they originally intended and marketed and implied that it would for a given patient, there is a way to be able to adjust the price and allow that patient to be able to be retreated with the same therapy again, or given a different type of essentially a biological boost for gene expression to occur.

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